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Release – Cadrenal Therapeutics Announces Upcoming Type-B FDA Meeting in September to Discuss Tecarfarin Trial in LVAD Patients

Posted on August 22, 2024August 22, 2024 by dburczyk@noblecapitalmarkets.com

PONTE VEDRA, Fla., Aug. 22, 2024 — Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage, next-generation Vitamin K Antagonist (VKA) oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices and those with rare cardiovascular conditions, announced today that it will be engaging with the U.S. Food and Drug Administration (FDA) in early September for a Type-B meeting to discuss its clinical trial for tecarfarin in LVAD patients.

“This upcoming meeting with the FDA is a crucial step in developing tecarfarin as we prepare for our pivotal trial. We look forward to discussing the development program for tecarfarin in LVAD patients,” said Quang Pham, Chief Executive Officer of Cadrenal Therapeutics.

ABOUT LVAD PATIENTS

Left Ventricular Assist Devices (LVADs) are mechanical pumps to support heart function in patients with advanced heart failure. These devices are vital for patients awaiting heart transplants or those who are ineligible for transplants. However, LVAD patients face an increased risk of thromboembolic events, such as strokes, which necessitates ongoing anticoagulation therapy. The current anticoagulation therapy, warfarin, presents challenges, including variability in dosing, a narrow therapeutic window, and potential interactions with other medications, making effective management crucial to reducing complications and ensuring patient safety.

ABOUT CADRENAL THERAPEUTICS, INC.
Cadrenal Therapeutics is developing tecarfarin for unmet needs in anticoagulation therapy. Tecarfarin is a late-stage novel oral and reversible anticoagulant (blood thinner) to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices and those with rare cardiovascular conditions. Tecarfarin has orphan drug designation for the prevention of thrombosis and thromboembolism in patients with ventricular assist devices. Tecarfarin also has orphan drug and fast-track designations from the FDA for the prevention of systemic thromboembolism (blood clots) of cardiac origin in patients with end-stage kidney disease and atrial fibrillation. Cadrenal is also pursuing additional regulatory strategies for unmet needs in anticoagulation therapy for patients with thrombotic antiphospholipid syndrome (APS). Tecarfarin is specifically designed to leverage a different metabolism pathway than the oldest and most commonly prescribed Vitamin K Antagonist (warfarin). Tecarfarin has been evaluated in 11 human clinical trials and more than 1,000 individuals. In Phase 1, Phase 2, and Phase 2/3 clinical trials, tecarfarin has generally been well-tolerated in both healthy adult subjects and patients with chronic kidney disease. For more information, please visit: www.cadrenal.com.

SAFE HARBOR STATEMENT

Any statements contained in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding the Company engaging with the FDA in early September for a Type-B meeting to discuss its clinical trial for tecarfarin in LVAD patients and the planned pivotal trial. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to improve anticoagulation treatment in patients, the success of the Type-B meeting, the ability of the Company to commence and complete a pivotal trial and commercialize tecarfarin with patients with left ventricular assist devices (LVADs), and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s subsequent filings with the SEC, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.

For more information, please contact:

Cadrenal Therapeutics:
Matthew Szot, CFO
858-337-0766
press@cadrenal.com

Investors:
Lytham Partners, LLC
Robert Blum, Managing Partner
602-889-9700
CVKD@lythampartners.com

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SOURCE Cadrenal Therapeutics, Inc.

Release – Published Findings Highlight Tecarfarin’s Potential and Reinforce Need for Better Anticoagulation Therapy in LVAD Patients

Posted on August 20, 2024August 20, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on CVKD

Analysis features the growing need to evolve anticoagulation therapy beyond warfarin to avoid gastrointestinal bleeding, a significant and common complication for left ventricular assist device (LVAD) patients resulting in expensive hospitalizations
Tecarfarin, a novel vitamin K antagonist (VKA), uses a different metabolic pathway than warfarin, the most commonly used anticoagulant for LVAD patients, and may offer more stable and effective anticoagulation

PONTE VEDRA, Fla., Aug. 20, 2024 — Cadrenal Therapeutics, Inc., (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage, next-generation VKA oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices or rare cardiovascular conditions, today highlighted a manuscript that was recently peer-reviewed and published in the Journal of Cardiac Failure evaluating the relationship between time in therapeutic range, or TTR, management quality and LVAD patient clinical outcomes. These findings focus on the potential role of investigating new VKA agents beyond warfarin in improving clinical outcomes in LVAD patients.

In the manuscript co-authored by Mandeep R. Mehra, MD, MSc, FRCP, Brigham and Women’s Hospital Heart and Vascular Center, Center for Advanced Heart Disease, titled “Antithrombotic Strategies with Left Ventricular Devices,” the findings conclude that although the current generation of LVAD pumps has largely overcome hemocompatibility-related adverse events there is a continuing need to evolve the anticoagulant therapy to avoid persistent gastrointestinal bleeding, which leads to frequent hospital admissions, procedures, blood transfusions, decreased quality of life and increased cost of care.¹

Dr. Mandeep Mehra, who chaired the ARIES-HM3 study, commented, “Tecarfarin could potentially be an important therapy for patients with LVADs who all require chronic anticoagulation since it does not get affected by drug-drug interactions or changes in kidney function like warfarin and deserves further study.”

A secondary analysis of the ARIES-HM3 trial, sponsored by Abbott, evaluated the relationship between VKA management quality and clinical outcomes, wherein a median TTR for VKAs of 56% was achieved. This analysis found an inverse relationship between TTR and bleeding events, with a 47% reduction in bleeding risk for patients with a TTR above the median. Lower TTRs were associated with threefold more sub-therapeutic INRs than supra-therapeutic INRs, with no clear correlation between higher INRs and bleeding events.

“These findings provide us with increased confidence that tecarfarin has the potential to address the LVAD patients’ critical unmet anticoagulation needs more effectively,” said Quang X. Pham, Chairman and Chief Executive Officer of Cadrenal Therapeutics. “We are excited to work with the LVAD community to potentially bring our next-generation VKA to patients.”

About Tecarfarin
Tecarfarin is the only oral anticoagulant in development worldwide for patients with implanted cardiac devices and other rare cardiovascular conditions. It has been uniquely designed to overcome many of the challenges patients experience with warfarin. Tecarfarin is metabolized using carboxyl esterase rather than the cytochrome P450 system, which allows the drug to avoid interactions with many other commonly used medications and may offer more stable anticoagulation, including those patients with renal dysfunction, which is common in LVAD patients. In a Phase II study with 66 patients with atrial fibrillation switched from warfarin to tecarfarin, the mean interpolated TTR was 71.4% within three weeks, with minimal time spent in extreme INR ranges (<1.5 and >4.0). If approved, tecarfarin has the potential to be the only on-label drug for LVAD patients in the U.S.

In addition, tecarfarin may prove valuable for other patients where warfarin is not providing recommended anticoagulation because of genetic warfarin resistance or renal impairment making warfarin metabolism difficult. These include individuals with end-stage kidney disease and atrial fibrillation or those with mechanical heart valves and hard-to-control International Normalized Ratio, which measures how long it takes the blood to clot.

On August 6, 2024, Cadrenal Therapeutics announced that it had been in discussions with Abbott about Cadrenal’s planned pivotal study of tecarfarin in patients with recently implanted LVADs. LVAD patients require lifelong anticoagulation to protect against thromboembolic events. The only LVAD available in the U.S. is Abbott’s HeartMate 3^TM.

On April 9, 2024, Cadrenal Therapeutics announced that the U.S. Food and Drug Administration (FDA) had granted tecarfarin Orphan Drug Designation for the prevention of thromboembolism and thrombosis in patients with an implanted mechanical circulatory support device, which includes the LVAD.

About Cadrenal Therapeutics, Inc.
Cadrenal Therapeutics Is Developing Tecarfarin for unmet needs in anticoagulation therapy. Tecarfarin is a late-stage novel oral and reversible anticoagulant (blood thinner) to prevent heart attacks, strokes, and deaths due to blood clots in patients with implanted cardiac devices and those with rare cardiovascular conditions. Tecarfarin has an orphan drug designation for the prevention of thrombosis and thromboembolism in patients with ventricular assist devices (VADs). Tecarfarin also has orphan drug and fast-track designations from the FDA for the prevention of systemic thromboembolism (blood clots) of cardiac origin in patients with end-stage kidney disease and atrial fibrillation. Cadrenal is also pursuing additional regulatory strategies for unmet needs in anticoagulation therapy for patients with thrombotic antiphospholipid syndrome. Tecarfarin is specifically designed to leverage a different metabolism pathway than the oldest and most commonly prescribed Vitamin K Antagonist (warfarin). Tecarfarin has been evaluated in 11 human clinical trials and more than 1,000 individuals. In Phase 1, Phase 2, and Phase 2/3 clinical trials, tecarfarin has generally been well-tolerated in both healthy adult subjects and patients with chronic kidney disease. For more information, please visit: www.cadrenal.com.

Safe Harbor Statement
Any statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding tecarfarin offering more stable and effective anticoagulation than warfarin, the potential role of investigating new VKA agents beyond warfarin in improving clinical outcomes in LVAD patients, the continuing need to evolve the anticoagulant therapy to avoid persistent gastrointestinal bleeding and tecarfarin potentially being an important therapy for patients with LVADs who all require chronic anticoagulation since it does not get affected by drug-drug interactions or changes in kidney function like warfarin, tecarfarin having the potential to address the LVAD patients’ critical unmet anticoagulation needs more effectively and potentially bringing the Company’s r next-generation VKA to patients The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to improve anticoagulation treatment in patients, the ability of the Company to advance tecarfarin with patients with left ventricular assist devices (LVADs), and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s subsequent filings with the SEC, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.

For more information, please contact:

Cadrenal Therapeutics:
Matthew Szot, CFO
858-337-0766
press@cadrenal.com

Investors:
Lytham Partners, LLC
Robert Blum, Managing Partner
602-889-9700
CVKD@lythampartners.com

View original content to download multimedia:https://www.prnewswire.com/news-releases/published-findings-highlight-tecarfarins-potential-and-reinforce-need-for-better-anticoagulation-therapy-in-lvad-patients-302225967.html

SOURCE Cadrenal Therapeutics, Inc.

Release – Tonix Pharmaceuticals Announces First Patient Enrolled in Phase 2 CATALYST Study of TNX-1300 for the Treatment of Cocaine Intoxication

Posted on August 20, 2024August 20, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on TNXP

CATALYST is a Phase 2 single-blind, placebo-controlled, proof-of-concept study in patients presenting to the emergency department

More than 27,569 individuals in the U.S. died from drug overdose deaths involving cocaine in 2022; there is currently no FDA-approved product for cocaine intoxication

Topline results are expected in the first half of 2025

CHATHAM, N.J., Aug. 20, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the first patient has been dosed in the Phase 2, single-blind, placebo-controlled, proof of concept trial of TNX-1300 (double-mutant cocaine esterase 200 mg, i.v. solution) for the treatment of acute cocaine intoxication in the emergency department (ED). TNX-1300 is a recombinant enzyme that rapidly and efficiently degrades and metabolizes cocaine in cocaine users, as demonstrated in a prior Phase 2a randomized, double-blind, placebo-controlled, laboratory-based clinical study, providing support for the use of TNX-1300 as a treatment for life-threatening cocaine intoxication.¹

Tonix has been awarded a Cooperative Agreement Grant from National Institute on Drug Abuse (NIDA), part of the National Institutes of Health (NIH), to support development of TNX-1300 for the treatment of cocaine intoxication. In addition, TNX-1300 has been granted Breakthrough Therapy designation by the U.S Food and Drug Administration (FDA).

“Cocaine abuse and dependence are major problems in the U.S. However, there is currently no FDA-approved treatment indicated for cocaine intoxication, a life-threatening state characterized by acute symptoms including agitation, hyperthermia, tachycardia, arrhythmias, hypertensive crisis, myocardial infarction, stroke, and seizures,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “In 2022, the number of overdose deaths involving cocaine reached 27,569 individuals.² With approximately 505,000 emergency room visits annually involving cocaine use and approximately 61,000 of the visits involving detox services to treat cocaine overdose,^3,4 we believe TNX-1300 has the potential to help address the morbidity and mortality caused by cocaine intoxication. By targeting the cause rather than the symptoms of cocaine intoxication, TNX-1300 may offer significant advantages to the current standard of care for cocaine overdose.”

The Phase 2 trial is a single-blind, open-label, placebo-controlled, randomized study comparing the safety of a single 200 mg dose of TNX-1300 to placebo injection plus standard of care alone for the treatment of signs and symptoms of acute cocaine intoxication. The study is being conducted in the EDs of six academic medical centers in the U.S. It will include approximately 60 subjects presenting to the ED with cocaine intoxication. During the treatment period, subjects randomized to receive TNX-1300 will receive a single i.v. injection of TNX-1300 administered over two minutes or less; whereas subjects randomized to receive standard of care alone will receive a single i.v. saline injection over two minutes or less. For both study arms, signs and symptoms of cocaine intoxication will be assessed at pre-determined time points after treatment. After randomization, blood samples will be drawn at specific time points to assess the pharmacokinetics of TNX-1300 and levels of cocaine and its metabolites in the plasma. The primary endpoint of the study is reduction of systolic blood pressure associated with acute cocaine intoxication identified at study baseline comparing TNX-1300 to placebo with standard of care after 60 minutes. A variety of secondary endpoints will be measured, including reduction of circulating cocaine and levels of its metabolites at multiple post-baseline timepoints.

For more information, see ClinicalTrials.gov Identifier: NCT06045793

About TNX-1300

TNX-1300 (T172R/G173Q double-mutant cocaine esterase 200 mg, i.v. solution) is being developed under an Investigational New Drug application (IND) for the treatment of cocaine intoxication. TNX-1300 is a recombinant protein enzyme produced through recombinant DNA technology in a non-disease-producing strain of E. coli bacteria. Cocaine esterase (CocE) was identified in bacteria (Rhodococcus) that uses cocaine as its sole source of carbon and nitrogen and that grows in soil surrounding coca plants.⁵ The gene encoding CocE was identified and the protein was extensively characterized.⁵^–⁸ CocE catalyzes the breakdown of cocaine into metabolite ecgonine methyl ester and benzoic acid. Wild-type CocE is unstable at human body temperature, so targeted mutations were introduced in the CocE gene and resulted in the T172R/G173Q double-mutant CocE, which is active for approximately 6 hours at body temperature.⁸ In a Phase 2 laboratory-based study in volunteers who use cocaine, TNX-1300, at 100 mg or 200 mg i.v. doses, was well tolerated and rapidly reduced cocaine effects after cocaine 50 mg i.v. challenge.¹

About Cocaine Intoxication and Overdose

Cocaine is an illicit recreational drug which is taken for its pleasurable effects and associated euphoria. In 2022, over 5 million individuals in the U.S. reported current cocaine use, almost 2% of the population.⁹ Pharmacologically, cocaine blocks the reuptake of the neurotransmitter dopamine from central nervous system synapses, resulting in the accumulation of dopamine within the synapse and an amplification of dopamine signaling and its capacity to produce euphoric mood states. With the continued use of cocaine, however, intense cocaine cravings occur resulting in a high potential for abuse and addiction (dependence), as well as the risk of acute cocaine intoxication. Cocaine intoxication refers to the deleterious effects on several body systems, especially those involving the cardiovascular system. Common symptoms of cocaine intoxication include tachyarrhythmias and elevated blood pressure, either of which can be life-threatening. As a result, individuals with known or suspected cocaine intoxication are sent immediately to the emergency department (ED), preferably by ambulance in case cardiac arrest occurs during transit. The standard of care for treating cocaine intoxication in the ED focuses on symptom management, preventing complications, and supporting cardiovascular, respiratory, and neurological function, e.g. benzodiazepines for agitation, seizures, and sympathetic overdrive; antihypertensives for extremely elevated blood pressure; aspirin and nitroglycerine for cardiac ischemia. There are approximately 505,000 emergency room visits for cocaine abuse each year in the U.S., of which 61,000 require detoxification services.^3,⁴ According to the National Institute on Drug Abuse, in 2022 the number of overdose death involving cocaine reached 27,569 individuals.² In 2019, Black Americans experienced the highest death rate for overdoses involving cocaine, at 10.7 per 100,000.¹⁰

References

¹ Nasser et al. A randomized, double-blind, placebo-controlled trial of RBP-8000 in cocaine abusers: pharmacokinetic profile of rbp-8000 and cocaine and effects of RBP-8000 on cocaine-induced physiological effects. J Addict Dis. 2014;33(4):289-302.

² https://nida.nih.gov/research-topics/trends-statistics/overdose-death-rates; August 18, 2024

³ Substance Mental Health Services Administration, Drug Abuse Warning Network, 2011: National Estimates of Drug- Related Emergency Department Visits. HHS Publication No. (SMA) 13-4760, DAWN Series D-39. Rockville, MD: Substance Abuse and Mental Health Services Administration, 2013.

⁴Drug Abuse Warning Network, 2011: Selected Tables of National Estimates of Drug-Related Emergency Department Visits. Rockville, MD: Center for Behavioral Health Statistics and Quality, SAMHSA, 2013.

⁵ Bresler, et al. Gene cloning and nucleotide sequencing and properties of a cocaine esterase from Rhodococcus sp. strain MB1. Appl Environ Microbiol. 2000. 66(3):904-8.

⁶ Larsen, et al. Crystal structure of a bacterial cocaine esterase. Nat Struct Biol. 2002. 9(1):17-21.

⁷ Turner, et al. Biochemical characterization and structural analysis of a highly proficient cocaine esterase. Biochemistry. 2002. 41(41):12297-307.

⁸ Gao, et al. Thermostable variants of cocaine esterase for long-time protection against cocaine toxicity. Mol Pharmacol. 2009. 75(2):318-23.

⁹ https://www.cdc.gov/drugoverdose/deaths/other-drugs.html; accessed August 18, 2024

¹⁰ Kariisa, et al. Drug overdose deaths involving cocaine and psychostimulants with abuse potential among racial and ethnic groups – United States, 2004-2019. Drug Alcohol Depend. 2021. 1;227:109001.

Tonix Pharmaceuticals Holding Corp.^*

Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA) to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD, instrumental in progressing this development. Tonix Medicines, our commercial subsidiary, markets Zembrace^® SymTouch^® (sumatriptan injection) 3 mg and Tosymra^® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.

*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Peter Vozzo
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Media Contact

Ray Jordan
Putnam Insights
ray@putnaminsights.com
(949) 245-5432

Source: Tonix Pharmaceuticals Holding Corp.

Released August 20, 2024

Release – Tonix Pharmaceuticals Reports Second Quarter 2024 Financial Results and Operational Highlights

Posted on August 19, 2024August 19, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on TNXP

On track to submit NDA in second half 2024 for TNX-102 SL for fibromyalgia; completed successful pre-NDA meetings with FDA in second quarter 2024

FDA granted Fast Track designation for TNX-102 SL for fibromyalgia

Commercial planning continues for U.S. launch of TNX-102 SL, a potential new first-line, centrally-acting, non-opioid analgesic for the management of fibromyalgia

U.S. Department of Defense contract awarded for up to $34 million over 5 years to develop a broad-spectrum antiviral drug

World Health Organization (WHO) recently declared spread of mpox in multiple African countries a public health emergency of international concern – Tonix’s TNX-801 is an mpox vaccine in development which protects animals against lethal challenge of monkeypox virus

CHATHAM, N.J., Aug. 19, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced financial results for the second quarter ended June 30, 2024, and provided an overview of recent operational highlights.

“In the second quarter of 2024, we made significant progress advancing our New Drug Application (NDA) and market access strategy for TNX-102 SL (cyclobenzaprine HCl sublingual tablets),” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “We are working diligently on the regulatory submission and are pleased to have been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) and to be in alignment with the agency regarding the content of our proposed NDA package. Fast Track is designed to facilitate the development and expedite FDA review of important new drugs to treat serious conditions and fill an unmet medical need. We are hopeful that TNX-102 SL can be the first new drug to treat fibromyalgia in more than 15 years.”

Dr. Lederman continued, “Additionally, we continue to advance other key pipeline candidates through a capital efficient strategy, including TNX-4200, our broad-spectrum antiviral program for which we were awarded up to $34 million over five years from the U.S. Department of Defense (DoD) to advance its development.”

Recent Highlights – Key Product Candidates*

Central Nervous System (CNS) Pipeline

TNX-102 SL (cyclobenzaprine HCl sublingual tablets): a centrally-acting, non-opioid, analgesic taken once-daily at bedtime for the management of fibromyalgia.

In July 2024, the FDA granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. The designation validates that fibromyalgia is a serious condition and that TNX-102 SL has the potential to address this unmet medical need. The Company continues to guide for submission of the NDA for TNX-102 SL in the second half of 2024 which would allow for a potential FDA approval in 2025.
In July 2024, Tonix announced that, based on the new definition of Long COVID by the U.S. National Academies of Sciences, Engineering and Medicine (NASEM), fibromyalgia is a ‘diagnosable condition’ in people suffering from Long COVID. The Company believes that diagnosing fibromyalgia in Long COVID patients will increase the potential market for TNX-102 SL following approval as compared to market estimates from before the COVID-19 pandemic.
During the second quarter, Tonix successfully completed two positive pre-NDA meetings with the FDA for TNX-102 SL for the management of fibromyalgia. The first, with minutes announced in June 2024, was a Type B Chemistry, Manufacturing, and Controls (CMC) meeting to seek alignment and agreement with the FDA on key CMC topics to support the planned NDA submission for TNX-102 SL. Based on formal meeting minutes, the Company believes it is aligned with the FDA on proposed drug substance and drug product commercial specifications, shelf life assignment, manufacturing and commercial drug packaging. At the second pre-NDA meeting announced in July 2024, the Company and the FDA aligned on nonclinical, clinical pharmacology and clinical matters and agreed that the proposed data package is sufficient to support the NDA submission.
In June 2024, at the American Society of Clinical Psychopharmacology (ASCP) meeting the Company presented new, additional data on TNX-102 SL, highlighting improvement in depressive symptoms as measured by the Beck Depression Inventory-II (BDI-II) as well as improvements in anxiety, memory and energy items on the Fibromyalgia Impact Questionnaire-Revised (FIQR). Depression was frequent among patients enrolled in the Phase 3 RESILIENT study, as ~47% reported experiencing depression within the past six months upon fibromyalgia diagnosis and ~25% of the intent-to-treat (ITT) population had experienced a lifetime major depressive episode (MDE). However, by Week 14 of the trial, the total BDI-II score in the TNX-102 SL group improved over placebo with a nominal p-value of 0.005 and an effect size of 0.27. On the FIQR, exploratory analyses uncorrected for multiplicity demonstrated improvement in the TNX-102 SL group over placebo in depression (p < 0.001), anxiety (p = 0.001), sensitivity (p = 0.020), memory problems (p = 0.001) and energy (p < 0.001). TNX-102 SL was well tolerated and the most common adverse events were transient sensations in the mouth corresponding with the disintegration of the tablet under the tongue. Together these findings provide further support that TNX-102 SL has broad-spectrum activity against fibromyalgia symptoms and may improve fibromyalgia at the syndromal level.
EVERSANA® Life Science Services, LLC, a leading provider of commercialization services to the global life sciences industry, completed the initial phase of an assessment of the U.S market opportunity for TNX-102 SL. Tonix had previously announced that EVERSANA was selected to support the launch strategy and commercial planning of TNX-102 SL. Specifically, EVERSANA is working with Tonix to assess the fibromyalgia landscape and help plan an efficient go-to-market strategy. EVERSANA conducted primary research, analyzed potential market size, surveyed currently marketed treatment options and their market shares and interviewed physicians for feedback regarding the currently prescribed treatments and their interest in TNX-102 SL as a potential new treatment option. This physician feedback demonstrated high levels of dissatisfaction with currently prescribed drugs, a high unmet need for their fibromyalgia patients, and high levels of interest in TNX-102 SL’s favorable activity and tolerability profile. Further analysis showed that addictive opioids are prescribed more frequently than the currently approved drugs following fibromyalgia diagnosis. Physicians also indicated they would intend to use TNX-102 SL in 40% of their fibromyalgia patients.¹

TNX-102 SL for the treatment of acute stress reaction (ASR) and acute stress disorder (ASD), and prophylaxis against development of posttraumatic stress disorder (PTSD)

In May 2024, Tonix announced a plan for a Phase 2, investigator-initiated OASIS trial, designed to examine the safety and efficacy of TNX-102 SL in treating Acute Stress Disorder (ASD) after motor vehicle collision. The trial is sponsored by the University of North Carolina Institute for Trauma Recovery and supported by a $3 million contract from the DoD, which was awarded in September 2023. TNX-102 SL will be evaluated for the reduction in severity of acute stress reaction (ASR) and the frequency of acute stress disorder (ASD) and posttraumatic stress disorder (PTSD) in civilians after a motor vehicle collision. Previous trials of TNX-102 SL showed that it reduced military PTSD symptoms within two weeks while evidencing similar favorable tolerability as in fibromyalgia patients.
Tonix expects to enroll the first patient in the Phase 2 OASIS trial in the third quarter of 2024.

TNX-1300 (recombinant double mutant cocaine esterase): biologic for life-threatening cocaine intoxication

Tonix expects to initiate a Phase 2 clinical study of TNX-1300 for the treatment of cocaine intoxication in emergency rooms in the third quarter of 2024. In 2022, Tonix was awarded a Cooperative Agreement grant from the National Institutes of Health (NIH)’s National Institute of Drug Abuse (NIDA) to support development of TNX-1300.
TNX-1300 has been granted Breakthrough Therapy designation by the FDA.

TNX-1900 (intranasal potentiated oxytocin): small peptide in development through investigator-initiated studies for adolescent obesity, binge eating disorder (BED), bone health in autism and social anxiety disorder (SAD).

TNX-1900 continues to be studied in four ongoing investigator-initiated Phase 2 studies. There are three studies at Massachusetts General Hospital: the POWER study for the treatment of adolescent obesity, the STROBE study for the treatment of BED, and the BOX study for the treatment of bone health in pediatric autism. In addition, Tonix is conducting an investigator-initiated study of TNX-1900 for the treatment of SAD at the University of Washington.

Rare Disease Pipeline

TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)

In March 2024, Tonix announced that it received Rare Pediatric Disease designation from the FDA for TNX-2900 for the treatment of PWS. Tonix has an investigational new drug (IND) to support clinical development of TNX-2900 to treat PWS in children and adolescents, including a planned Phase 2, dose-finding study involving approximately 36 PWS patients. TNX-2900 for the treatment of PWS was granted Orphan Drug designation by the FDA in 2022. PWS is a rare genetic disorder which causes cognitive and behavioral symptoms including pathological over-eating in childhood, which leads to severe metabolic sequelae in adolescence and adulthood.

Immunology Pipeline

TNX-1500 (anti-CD40L Fc-modified humanized monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.

The first proposed indication for TNX-1500 is prophylaxis of organ rejection in adult patients receiving a kidney transplant; but multiple additional indications are possible, including autoimmune diseases. Preclinical studies have shown that TNX-1500 maintains the activity of first-generation monoclonal antibodies (mAbs), yet with reduced risk of thrombotic complications.^2-4 Modeling studies from animal pharmacokinetic data² predict a half-life of greater than three weeks for TNX-1500 in humans, which supports a monthly i.v. dosing regimen^5,6. This analysis together with TNX-1500’s activity and tolerability in animals, suggests that the protein engineering of TNX-1500’s Fc region has achieved its design goals.
In June 2024, at the American Transplant Congress 2024, Tonix announced data demonstrating the combined use of TNX-1500 and anti-CD28 monoclonal antibody, VEL-101 is associated with durable protection and graft survival and function in a nonhuman primate model. Further data demonstrated that TNX-1500 has promise to prevent rejection of 9-, or 10-gene-edited (GE) pig hearts.⁷^,⁸ All research has been directed by the faculty of the Center for Transplantation Sciences at Massachusetts General Hospital.
Tonix completed the clinical stage of its Phase 1 single ascending dose study of TNX-1500 in healthy volunteers. The primary objectives of the study are to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of intravenous TNX-1500. This first-in-human study is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients.

Infectious Disease Pipeline

TNX-4200 (orally available CD45 antagonist), TNX-3900 (cathepsin inhibitor), TNX-4000 (glycan-targeted biologic)

Tonix is developing potential broad-spectrum antiviral drugs in three programs: CD45-targeted therapeutics (TNX-4200), cathepsin inhibitors (TNX-3900) and viral glycan-targeted engineered biologics (TNX-4000).
In July 2024, the Company announced that the DoD’s Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA). The objective of the contract is to develop small molecule broad-spectrum antiviral agents for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. The $34 million five-year contract will help fund and accelerate the development of Tonix’s broad-spectrum antiviral program, which has the potential to reduce viral load and allow the adaptive immune system to alert the other arms of the immune system to mount a protective response. The Company’s program will focus on optimization and development of its TNX-4200 program, to develop an orally available CD45 antagonist, with broad-spectrum efficacy against a range of viral families through preclinical evaluation. The program is expected to establish physicochemical properties, pharmacokinetics, and safety attributes to support an IND submission and to fund a first-in-human Phase 1 clinical study.

TNX-801 (recombinant horsepox virus, live vaccine): potential vaccine to protect against mpox disease and smallpox.

In June 2024, Tonix announced data at an oral keynote presentation at the Vaccine Congress 2024, detailing the company vaccine platform, including TNX-801 (horsepox, live virus) vaccine for preventing mpox (formerly known as monkeypox). TNX-801 is a live replicating attenuated vaccine based on horsepox that is believed to provide immune protection with better tolerability than modern vaccinia viruses. In the data, Tonix highlighted positive preclinical efficacy, demonstrating that TNX-801 protected non-human primates against lethal challenge with intratracheal Clade 1 monkeypox virus.9 After a single dose vaccination, TNX-801 prevented clinical disease and lesions and also decreased shedding in the mouth and lungs of non-human primates. These findings are consistent with mucosal immunity and suggest the ability to block forward transmission.
The WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern, the second such declaration in the past two years called in response to transmission of the virus.
Mpox is epidemic in Central Africa and experts fear the new Clade 1 strain may spread to the U.S.
The company’s Good Manufacturing Practice (GMP)-capable advanced manufacturing facility in Dartmouth, MA was purpose-built to manufacture TNX-801 and the GMP suites are ready to be reactivated in case of a national or international emergency.

TNX-1800 (modified recombinant horsepox virus, live vaccine): potential vaccine to protect against COVID-19 designed to express the SARS-CoV-2 spike protein

In June 2024, the Company announced preclinical data, demonstrating immunity and tolerability, during an oral keynote talk at the Vaccine Congress 2024.¹⁰^,¹¹ Like TNX-801, TNX-1800 is a live replicating attenuated vaccine based on horsepox that is believed to provide immune protection with better tolerability than modern vaccinia viruses. TNX-1800 was selected by the NIH’s Project NextGen for inclusion in clinical trials as part of a select group of next generation COVID-19 vaccine candidates with the intent to identify promising vaccine platforms. NIH plans to conduct a Phase 1 trial and cover the full cost, while Tonix provides the vaccine candidate.

Marketed Products – Recent Highlights

As of April 1, 2024, Tonix completed the transition to becoming a fully integrated biopharmaceutical company. Tonix Pharmaceuticals has implemented personnel, systems and contracts required to support a commercial organization and has assumed responsibility for distribution, selling and marketing of Zembrace SymTouch® and Tosymra®, as well as supply chain, regulatory and quality control of the two products.
In June 2024, the Company presented data at the 66^th Annual Scientific Meeting of the American Headache Society (AHS) comparing real-world data with real-world usage of non-oral migraine products with the most recent AHS consensus statement. This data stressed the need for customizing treatment of migraine headaches to the needs of patients. Thus far, real world data show that conformity with the guidelines and the consensus statement have yet to be achieved but has the potential to be increased. The data show the use of non-oral drugs for treating an acute migraine attack was only 7% in 2012 and has decreased to below 4% in 2023, when the potential need for such drugs is anticipated to be a more substantial percentage of migraineurs based on epidemiological data.

TNX-102 SL has not been approved for any indication.

¹EVERSANA primary physician research, May 2024; commissioned by Tonix

²Lassiter G., et al. Am J Transplantation. 2023. https://doi.org/10.1016/j.ajt.2023.03.022

³Miura S., et al. Am J Transplantation. 2023. https://doi.org/10.1016/j.ajt.2023.03.025

⁴Anand RP., et al. Nature. 2023:622, 393–401. https://doi.org/10.1038/s41586-023-06594-4

⁵Deng R., et al. Mabs. 2011. https://doi.org/10.4161/mabs.3.1.13799

⁶Tonix Pharmaceuticals – Data on File

⁷Revivicor 9-GE pigs: GalKO.β4GalNT2KO.GHRKO.hCD46.hCD55.hTBM.hEPCR.hCD47.hHO-1

⁸ Revivicor 10-GE pigs: GalKO.β4GalNT2KO.CMAHKO.GHRKO.hCD46.CD55.hTBM.hEPCR.hCD47.hHO-1.

⁹Noyce RS, et al. Viruses. 2023;15(2):356. doi:10.3390/v15020356.

¹⁰Awasthi M., et al. Viruses. 2023;15(10):2131. doi:10.3390/v15102131.

¹¹Awasthi M., et al. Vaccines (Basel). 2023;11(11):1682. doi:10.3390/vaccines11111682.

Recent Highlights – Financial

As of June 30, 2024, Tonix had $4.2 million of cash and cash equivalents, compared to $24.9 million as of December 31, 2023. Net cash used in operations was approximately $30.5 million for the six months ended June 30, 2024, compared to $56.3 million for the same period in 2023.

Subsequent to the quarter ending June 30, 2024, Tonix received net proceeds of approximately $3.5 million in a securities purchase agreement with certain institutional and retail investors, and sold 0.8 million shares of common stock under the ATM Sales Agreement, for net proceeds of approximately $0.4 million.

Second Quarter 2024 Financial Results

Net product revenue for the second quarter 2024 was approximately $2.2 million. Net product revenue consisted of combined net sales of Zembrace® SymTouch® and Tosymra®, which were acquired from Upsher-Smith Laboratories, LLC on June 30, 2023. Cost of Sales for the second quarter 2024 was approximately $3.4 million, which included a write-down related to Tosymra and Zembrace finished goods inventory of approximately $1.7 million based on an assessment of inventory on hand and projected sales prior to the respective expiration dates.

Research and development expenses for the second quarter 2024 were $9.7 million, compared to $22.0 million for the same period in 2023. This decrease is predominantly due to decreased clinical, non-clinical and manufacturing expenses aligned with the Company’s capital efficient strategy.

Selling, general and administrative expenses for the second quarter 2024 were $7.5 million, compared to $7.0 million for the same period in 2023. The increase was primarily due to sales and marketing and the transition services expenses associated with the Company’s recently acquired marketed products offset by a decrease in financial reporting expenses.

Net loss available to common stockholders was $78.8 million, or $19.28 per share, basic and diluted, for the second quarter 2024, compared to net loss of $28.4 million, or $49.23 per share, basic and diluted, for the same period in 2023. Included in the net loss for the three months ended June 30, 2024, are non-cash asset impairment charges totaling $58.9 million. The basic and diluted weighted average common shares outstanding for the second quarter 2024 was 4,085,132 compared to 576,047 shares for the same period in 2023.

The impairment of the Tosymra and Zembrace inventory, intangibles and goodwill was driven by our delayed investment in the sales personnel required to drive growth in the business as we are focusing our cash resources to further our efforts to bring TNX-102 SL through the FDA approval process and to market. However, we believe that the benefits and long-term value proposition of the 2023 acquisition of Tosymra and Zembrace remain, in that we now have the infrastructure to be ready to manufacture and sell TNX-102 SL under an expedited timeline pending FDA approval for which we expect an FDA decision in 2025.

Tonix Pharmaceuticals Holding Corp.^*

Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA) to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD, instrumental in progressing this development. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.

*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Click here for full report

Release – Tonix Pharmaceuticals Provides Update on the Development of Its Single Dose Live Attenuated Virus Vaccine Candidate for Mpox, TNX-801, as WHO Declares Mpox Outbreak a Global Health Emergency

Posted on August 16, 2024August 16, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on TNXP

World Health Organization (WHO) has declared spread of mpox in multiple African countries a public health emergency of international concern (PHEIC) ¹

Tonix’s live virus vaccine candidate, TNX-801, is designed to provide long-term protection from mpox and smallpox with one dose

TNX-801 vaccination demonstrated efficacy in protecting animals from lethal challenge with intratracheal monkeypox virus

Clade II mpox is now endemic in the U.S. with >30,000 cases reported since May 2022² and Clade I mpox is endemic in the Democratic Republic of the Congo³

Tonix’s vaccine platform has been selected by NIH’s Project NextGen for clinical testing

CHATHAM, N.J., Aug. 16, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, has reiterated its commitment to advance development of its live attenuated virus vaccine, TNX-801 (recombinant horsepox virus), for preventing mpox (formerly known as monkeypox) and other infectious diseases. On August 14, 2024, the World Health Organization (WHO) determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern, the second such declaration in the past two years called in response to an mpox outbreak. The current outbreak was caused by Clade 1 monkeypox virus, while the 2022 outbreak was Clade 2 monkeypox virus.

TNX-801 is a live replicating attenuated vaccine candidate based on horsepox that is believed to provide immune protection with better tolerability than 20^th century vaccinia viruses. The same vaccine platform upon which TNX-801 is based was selected by the U.S. National Institutes of Health (NIH) for Project NextGen for an engineered version that expresses the spike protein of SARS-CoV-2.

As previously disclosed, TNX-801 protected animals against lethal challenge with intratracheal Clade 1 monkeypox virus⁴. After a single-dose vaccination, TNX-801 prevented clinical disease and lesions and also decreased shedding in the mouth and lungs of non-human primates⁴. These findings are consistent with mucosal immunity and suggest the ability to block forward transmission, similar to Dr. Edward Jenner’s vaccinia vaccine, which eradicated smallpox and kept mpox out of the human population.

“The recent WHO declaration underscores the urgent need for additional treatments to stop these outbreaks and save lives,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “We are motivated to advance development for our mpox vaccine with urgency given the global public health emergency. Preclinical trials demonstrate that TNX-801 combines immune protection with improved tolerability and safety compared to other vaccines based on orthopoxviruses, and is administered with a single dose which has advantages over two-dose regimens. The durability of protection from 19^th century live virus vaccinia vaccines suggests that our attenuated TNX-801 will not require multiple repeated doses at six-month intervals like mRNA vaccines. Also, the stability of live virus vaccines eliminates the need for ultra-cold storage which complicates the widespread use of mRNA vaccines in Africa, where they are needed most right now.”

The global mpox outbreak, which commenced in 2022, has affected over 90,000 persons in countries where mpox had previously not been endemic, including Europe and the U.S. The spread of Clade IIb strain mpox in 2022 underscores the pandemic potential of mpox. Unlike Clade IIb mpox, the Clade I strain of mpox appears to be spreading to countries neighboring the Democratic Republic of the Congo. Clade I mpox is typically associated with approximately 20 times the case fatality rates than Clade IIb mpox in Africa. According to the U.S. Centers for Disease Control and Prevention (CDC), and other experts, there is a significant risk that the deadlier Clade I strain may appear in the U.S.²^,⁸

Further, the Bipartisan Commission on Biodefense recently highlighted the renewed dual threats of a more virulent mpox epidemic and a smallpox re-introduction from lab accidents or bad actors⁹. The National Academies of Science, in its review of smallpox preparedness, highlighted the need for new single dose vaccines, like TNX-801 against smallpox¹⁰.

About TNX-801* and Tonix’s RPV Platform

TNX-801 (recombinant horsepox virus) is a live virus vaccine for percutaneous administration that is being developed to target smallpox, and mpox (monkeypox). TNX-801 is also the basis of the RPV platform based on a horsepox vector, which is being adapted as a COVID-19 vaccine, term TNX-1800*. Horsepox is a live replicating, attenuated virus that has been shown to be >1,000-fold more attenuated than 20^th century vaccinia (VACV) strains in immunocompromised mice. Horsepox and the vaccinia vaccine viruses are closely related orthopoxviruses that are believed to share a common ancestor. Molecular analysis shows that horsepox is closer than modern vaccinia vaccines in DNA sequence to the vaccine discovered and disseminated by Dr. Edward Jenner. Live replicating orthopoxviruses, like vaccinia or horsepox, can be engineered to express foreign genes and have been explored as platforms for vaccine development because they possess; (1) large packaging capacity for exogenous DNA inserts, (2) precise virus-specific control of exogenous gene insert expression, (3) lack of persistence or genomic integration in the host, (4) strong immunogenicity as a vaccine, (5) ability to rapidly generate vector/insert constructs, (6) readily manufacturable at scale, and (7) ability to provide direct antigen presentation. Relative to vaccinia, horsepox has substantially decreased virulence in mice. The current formulation is a frozen liquid, but we believe that future lyophilized versions can be stored and shipped at standard refrigeration. Horsepox-based vaccines are designed to be single dose, vial-sparing vaccines that can be administered without sterile injection, manufactured using conventional cell culture systems with the potential for mass scale production, and packaged in multi-dose vials.

Tonix Pharmaceuticals Holding Corp.^*

Tonix is a fully-integrated biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA) to develop TNX-4200 small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. The company’s Good Manufacturing Practice (GMP)-capable advanced manufacturing facility in Dartmouth, MA was purpose-built to manufacture TNX-801 and the GMP suites are ready to be reactivated in case of a national or international emergency. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace^® SymTouch^® (sumatriptan injection) 3 mg and Tosymra^® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.

*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.

This press release and further information about Tonix can be found at www.tonixpharma.com.

¹WHO Press Release August 14, 2024. “WHO Director-General declares mpox outbreak a public health emergency of international concern”. URL: www.who.int/news/item/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern (accessed 8-15-24)
²McQuiston JH, et al. U.S. Preparedness and Response to Increasing Clade I Mpox Cases in the Democratic Republic of the Congo. 2024, MMWR Morb Mortal Wkly Rep: United States. p. 435-440
³CDC. 2022-2023 Mpox: US Map and Case Count. https://www.cdc.gov/poxvirus/mpox/response/2022/us-map.html
⁴Noyce RS, et al. Viruses. 2023 Jan 26;15(2):356. doi: 10.3390/v15020356
⁵Trefry, SV et al. bioRxiv 2023.10.25.564033; https://doi.org/10.1101/2023.10.25.564033
⁶Awasthi M, et al. Viruses. 2023 Oct 21;15(10):2131. doi: 10.3390/v15102131.
⁷Awashti M et al Vaccines (Basel). 2023 Nov 2;11(11):1682. doi:10.3390/vaccines11111682.
⁸World Health Organization SAGE meeting highlights on updated mpox vaccine recommendations. 2024, March
⁹Bipartisan Commission on Biofense. Box the Pox: Reducing the risk of Smallpox and Other Orthopoxviruses, Washington: 2024
¹⁰U.S. National Academies of Science. Future State of Smallpox Medical Countermeasures. Washington: 2024

Forward Looking Statements

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Peter Vozzo
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Media Contact

Lisa DeScenza
LaVoieHealthScience
ldescenza@lavoiehealthscience.com
(617) 351-0243

Source: Tonix Pharmaceuticals Holding Corp.

Released August 16, 2024

Release – LakeShore Biopharma Announces Financial Results for Fiscal Year 2024

Posted on August 15, 2024August 15, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on LSB

Gross margin increased to 79.5%; product pipeline continues to advance

Company anticipates double-digit year-over-year revenue growth and bottom line breakeven for Fiscal Year 2025

GAITHERSBURG, Md., Aug. 15, 2024 /PRNewswire/ — LakeShore Biopharma Co., Ltd. (Nasdaq: LSB) (“LakeShore Biopharma” or the “Company”), a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer, today announced its financial results for the fiscal year ended March 31, 2024 (“Fiscal Year 2024”).

Mr. Dave Chenn, Chairman and Interim Chief Executive Officer of the Company, commented, “In Fiscal Year 2024, our revenue was affected by a shortage of finished product inventory, resulting from earlier supply chain disruptions at our YSJA rabies vaccine production facilities. Despite these difficulties, we have successfully implemented operational improvements that have normalized production, and our inventory challenges are now resolved as we enter Fiscal Year 2025. We have maintained our position as a leading rabies vaccine producer in China, and continued to improve our gross margin by emphasizing operational efficiency. We also made significant advancements in our product pipeline during Fiscal Year 2024, progressing our next-generation PIKA rabies vaccine through its ongoing Phase III clinical trial and meeting significant developmental milestones for other pipeline products.”

Mr. Dave Chenn continued, “Looking ahead, our Fiscal Year 2025 strategy will focus on driving revenue growth, controlling expenses, and enhancing profit margins. We are implementing a comprehensive plan that includes cost reductions, organizational restructuring, strengthened internal controls, and strategic resource allocation to key growth areas. Our goal will be to foster innovation, efficiency, stability, and sustainable profitability as we work to build value for our shareholders.”

Ms. Rachel Yu, Interim Chief Financial Officer of the Company, added, “For Fiscal Year 2024, we recorded revenues of RMB573.4 million (US$80.8 million), representing a decrease compared to Fiscal Year 2023 due to persistent supply chain issues affecting raw material availability, manufacturing processes, and overall output capacity which impacted our topline. We recorded a gross profit of RMB455.7 million (US$64.2 million), and our gross margin increased by 1.8 percentage points to 79.5% compared to Fiscal Year 2023. With cash and cash equivalents of RMB246.6 million (US$34.7 million), we will maintain our focus on maximizing long-term shareholder returns by refining our business strategies and leveraging new market opportunities. Looking ahead to Fiscal Year 2025, we anticipate double-digit year-over-year growth in revenues, and expect to achieve breakeven on our bottom line by the time our fiscal year concludes on March 31, 2025.”

Business Updates

YSJA^TM Rabies Vaccine

LakeShore Biopharma’s marketed vaccine product, YSJA^TM rabies vaccine, is the first aluminum-free lyophilized rabies vaccine launched in China. Since the Company commenced production at its current GMP-compliant facilities in February 2020, and commercialization of the product in late 2020, market intake of the Company’s YSJA rabies vaccine has been consistent and strong. As of March 31, 2024, LakeShore Biopharma maintained its leadership position as one of the top rabies vaccine producers in China and has sold more than 27.3 million doses of YSJA^TM rabies vaccines to approximately 1,767 Chinese Center(s) for Disease Control and Prevention (“CDC”) customers, which represents 61.3% of CDC customers in China since October 2020.

Clinical Pipeline

LakeShore Biopharma continues to prioritize and advance its portfolio of innovative product candidates under various clinical development stages, including PIKA rabies vaccine, PIKA YS-ON-001, and PIKA YS-HBV-002.

PIKA Rabies Vaccine

In April 2024, the Company announced positive interim results from the ongoing Phase III clinical trial of its next-generation PIKA rabies vaccine in the Philippines and Pakistan. The interim results indicate that the PIKA rabies vaccine has successfully met the primary endpoints of the trial and has the potential to achieve best-in-class accelerated protection and meet the World Health Organization’s goal of a one-week rabies vaccine regimen to replace the conventional three- or four-week regimens. Subject to successful completion of the Phase III clinical trial, the Company plans to submit the New Drug Applications/Biologics License Applications to relevant regulatory authorities throughout Asia, Africa, Europe, and the Americas.

PIKA YS-ON-001

PIKA YS-ON-001 is designed as an immunological therapeutical agent against cancers. In 2023, the Company completed the Phase I clinical trial of PIKA YS-ON-001 in China.

PIKA YS-HBV-002

In April 2024, the Company announced that its YS-HBV-002, the second generation of immunotherapeutic vaccine designed to treat patients suffering from chronic hepatitis B virus infection, had been granted clinical trial approval by the Philippine Food and Drug Administration. In light of the approval, the Company is preparing to initiate a Phase I clinical trial for YS-HBV-002 in the Philippines.

Fiscal Year 2024 Financial Results

Total Revenue

Total revenue was RMB573.4 million (US$80.8 million) in Fiscal Year 2024, compared to RMB687.2 million in the same period of 2023, representing a decrease of 16.6%. This was primarily due to COVID-related disruptions affecting the Company’s manufacturing operations and production, which reduced batch approvals and doses available for sale. This impact was partially offset by an increase in the product price of the YSJA rabies vaccine of approximately RMB2.9 per dose.

Gross Profit

Gross profit in Fiscal Year 2024 was RMB455.7 million (US$64.2 million), representing a 79.5% gross margin, compared to RMB533.8 million, or a 77.7% gross margin, in the same period of 2023. The improvement in gross margin was primarily due to the increase in unit price of the YSJA rabies vaccine, and a decrease in unit cost resulting from lowered delivery costs and an increase in production batches.

Selling and Marketing Expenses

Selling and marketing expenses in Fiscal Year 2024 were RMB301.3 million (US$42.5 million), compared to RMB272.9 million in the same period of 2023. This change was primarily due to an increase in promoting and marketing service fees to continuously promote the YSJA rabies vaccine.

General and Administrative Expenses

General and administrative expenses in Fiscal Year 2024 were RMB140.1 million (US$19.7 million), compared to RMB72.9 million in the same period of 2023. This change was primarily attributable to increases in legal fees, auditing fees, directors & officers liability insurance costs, and employee benefits.

Research and Development Expenses

Research and development expenses in Fiscal Year 2024 were RMB302.8 million (US$42.7 million), compared to RMB318.7 million in the same period of 2023. The change was primarily driven by decreases in testing fees, clinical trial fees, and consulting service fees related to the development of the Company’s COVID-19 vaccine, and decreases in employee benefits as a result of staffing optimizations.

Impairment Loss on Inventory, Property, Plant and Equipment, and Other Assets

Impairment loss on inventory, property, plant and equipment (“PP&E”), and other assets in Fiscal Year 2024 was RMB157.4 million (US$22.2 million), compared to RMB8.7 million in the same period of 2023. The change was primarily attributable to 1) impairment loss on inventory impacted by COVID-related disruptions affecting the Company’s manufacturing operations and production and raw materials used in the research and development of the Company’s COVID-19 vaccine, and 2) impairment loss on PP&E related to COVID-19 vaccine equipment.

Net Loss

Net loss for Fiscal Year 2024 was RMB433.5 million (US$61.1 million), compared with RMB145.5 million in the same period of 2023.

Balance Sheet

As of March 31, 2024, the Company had cash and cash equivalents of RMB246.6 million (US$34.7 million), compared with RMB370.4 million as of March 31, 2023.

Business Outlook

The Company anticipates double-digit year-over-year revenue growth and expects to achieve breakeven in Fiscal Year 2025.

The above outlook is based on information available as of the date of this press release and reflects the Company’s current and preliminary expectations regarding its business situation and market conditions. The outlook is subject to changes, especially given uncertainties and situations related to market competitive dynamics and regulatory policies, etc.

About LakeShore Biopharma

LakeShore Biopharma, previously known as YS Biopharma, is a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer. It has developed a proprietary PIKA® immunomodulating technology platform and a new generation of preventive and therapeutic biologics targeting Rabies, Coronavirus, Hepatitis B, Influenza, Shingles, and other virus infections. The Company operates in China, the United States, Singapore, and the Philippines, and is led by a management team that combines rich local expertise and global experience in the biopharmaceutical industry. For more information, please visit https://investor.lakeshorebio.com/.

Exchange Rate Information

This announcement contains translations of certain RMB amounts into U.S. dollars at a specified rate solely for the convenience of the reader. Unless otherwise noted, all translations from RMB to U.S. dollars are made at a rate of RMB7.095 to US$1.00, the exchange rate set forth in the central parity rate release of the People’s Bank of China on March 31, 2024.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical or current fact included in this press release are forward-looking statements, including but not limited to statements regarding the expected growth of LakeShore Biopharma, the development progress of all product candidates, the progress and results of all clinical trials, LakeShore Biopharma’s ability to source and retain talent, and the cash position of LakeShore Biopharma. Forward-looking statements may be identified by the use of words such as “estimate,” “plan,” “project,” “potential,” “forecast,” “intend,” “will,” “expect,” “anticipate,” “believe,” “goal,” “seek,” “target” or other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These statements are based on various assumptions, whether identified in this press release, and on the current expectations of LakeShore Biopharma’s management and are not predictions of actual performance.

LakeShore Biopharma cannot assure you the forward-looking statements in this press release will be accurate. These forward-looking statements are subject to a number of risks and uncertainties, including those included under the heading “Risk Factors” in the company’s Annual Report on Form 20-F filed with the Securities and Exchange Commission, or SEC, and other risks described in documents subsequently filed by the company from time to time with the SEC. There may be additional risks that LakeShore Biopharma does not presently know or that LakeShore Biopharma currently believes are immaterial that could also cause actual results to differ from those contained in the forward-looking statements. In light of the significant uncertainties in these forward-looking statements, nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. The forward-looking statements in this press release represent the views of LakeShore Biopharma as of the date of this press release. Subsequent events and developments may cause those views to change. However, while LakeShore Biopharma may update these forward-looking statements in the future, there is no current intention to do so, except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing the views of LakeShore Biopharma as of any date subsequent to the date of this press release. Except as may be required by law, LakeShore Biopharma does not undertake any duty to update these forward-looking statements.

Investor Relations Contact

Robin Yang
Partner, ICR, LLC
Tel: +1 (212) 537-4035
Email: LakeShoreBiopharma.IR@icrinc.com

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Release – Traws Pharma Reports Q2 2024 Financial Results, Provides Recent Business Highlights

Posted on August 15, 2024August 15, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on TRAW

Merger with Trawsfynydd Therapeutics, Inc (“Trawsfynydd”) and concurrent private placement of $14 million (cash runway to support planned operations through year end), with recently achieved clinical milestones, put Traws on track to achieve key readouts for the clinical pipeline in H2 2024 and beyond

Lead antiviral program, tivoxavir marboxil, for influenza, including avian flu, is dosing the first cohort in the Phase 1 dose extension study in Australia
COVID-19 program, ratutrelvir, completed the Phase 1 single-ascending dose (SAD) and multiple-ascending dose (MAD) study in Australia
Oncology strategy includes investigator-sponsored trials (ISTs), planned to begin in H2 2024, adding a new dimension to the narazaciclib program, with continued investigator support for rigosertib
Corporate update call planned for August 15, 2024 at 8:00 AM ET

NEWTOWN, Pa., Aug. 15, 2024 (GLOBE NEWSWIRE) — Traws Pharma, Inc. (“Traws” or “Traws Pharma”), a clinical stage biopharmaceutical company developing oral small molecules for respiratory viral diseases and cancer, today outlined recent business highlights and reported unaudited financial results for the second quarter ended June 30, 2024. The Company intends to host a conference call and live webcast on Thursday, August 15, 2024 at 8:00 AM ET.

“The last several months have been transformational for Traws Pharma, with strategic and product development progress and an increase in capital,” said Werner Cautreels, PhD, Chief Executive Officer of Traws Pharma. “Completion of the merger agreement that created Traws Pharma expanded our investor base to include recognized healthcare investors, Orbimed and Torrey Pines, while broadening our portfolio to include compounds that we believe have best-in-class potential for influenza (flu), including bird flu, and COVID-19. In addition, ISTs are enhancing our multi-kinase inhibitors for cancer, narazaciclib and rigosertib.”

“We expect the antiviral program to advance over the next 6+ months as we complete preparations to report Phase 1 results and initiate Phase 2 studies in flu and COVID-19. Preclinical data and topline data from SAD studies from our influenza candidate, tivoxavir marboxil, suggest that it has the potential to achieve our target product profile as a single dose treatment that is active against pandemic-potential viruses as well as oseltamivir and baloxavir resistant viruses,” continued Dr. Cautreels. “In addition, clinical-trial enabling studies for our COVID-19 candidate, ratutrelvir, suggest that it has the potential to be effective without the requirement for co-administration of a CYP-inhibitor, and active against virus strains that may be resistant to other approved agents such as nirmatrelvir. In the coming months, we look forward to completing the Phase 1 studies, finalizing the dosing plan and initiating the Phase 2 program for both tivoxavir marboxil and ratutrelvir. We believe these data could pave the way for key readouts in 2025.”

Upcoming Milestones

Tivoxavir marboxil — Targets the influenza cap-dependent endonuclease, which is highly conserved across flu strains, including avian flu. The compound is intended to be administered as a single dose per treatment

Q4 2024: Announcement of topline Phase 1 dose extension results from Australia
Q4 2024/Q1 2025: Initiation of Phase 2 efficacy study

Ratutrelvir – Targets Mpro (3CL protease). The compound does not require combination use of a CYP-inhibitor such as ritonavir

Q4 2024: Announcement of topline results from Phase 1 SAD and MAD studies
Q4 2024/Q1 2025: Initiation of Phase 2 efficacy study

Narazaciclib – our multi-kinase inhibitor, including CKD4+, with potential for use in multiple solid tumors

H2 2024: Release topline results from our Phase 1/2 study at an upcoming medical meeting. In addition, we plan to announce the recommended Phase 2 dose (RP2D) and initiate ISTs in multiple myeloma, breast cancer and other indications

Rigosertib – our multi-kinase inhibitor targeting cell cycle proteins including PLK-1, with potential for use in the ultra-rare disease, advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC, RDEB)

Continue to support the IST-led program, including compassionate use for patients with RDEB

Recent Developments

Start of dosing in Phase 1 dose extension study for tivoaxavir marboxil, a placebo-controlled trial designed to evaluate three doses, including two doses from the successful Phase 1 dose escalation study (80 and 120 mg) and one new, increased dose (240 mg) in healthy volunteers. The study was designed to assess pharmacokinetics, pharmacodynamics and safety, to define the dosing plan for the upcoming Phase 2 efficacy study. The expected dosing schedule will be one single oral dose.
Completion of a Phase 1 dose escalation study for ratutrelvir in Australia, a SAD/MAD study conducted in healthy volunteers. The study was designed to assess SAD (5 doses ranging from 15 mg to 600 mg) and MAD (2 doses, 150 mg, and 600 mg, daily for 10 days) and provide pharmacokinetics, pharmacodynamics and safety data to guide the dosing plan for the upcoming Phase 2 efficacy study. The expected dosing schedule will be one oral dose per day for 10 days.
Completion of Phase 1/2 dose escalation studies for narazaciclib, evaluated as a monotherapy and in combination with letrozole in patients with recurrent metastatic low-grade endometrioid endometrial cancer and other gynecologic malignancies. The studies were designed to define the dose limiting toxicity (DLT) and maximally tolerated dose (MTD) of the combination, and the RP2D for further clinical trials. Topline data are are being analyzed.
Presentation of further data from ongoing ISTs for rigosertib, shared at the Society for Investigative Dermatology (SID) held in July, 2024 which highlighted ongoing studies for RDEB-associated SCC, conducted at the University Hospital in Salzburg, Austria and Thomas Jefferson University, Philadelphia, PA.
Completion of the merger with Trawsfynydd and concurrent $14 million private placement in April expanded the clinical portfolio of the combined entity to include two potentially best-in-class oral small molecule programs, for influenza and COVID-19, as well as oral two agents for solid tumor cancers. The transaction also enhanced the Company’s investor base, with the addition of Orbimed and Torrey Pines.

Financial Results:

Traws financial results for the quarter ended June 30, 2024 represent the first post-transaction quarterly report of the combined company. The financial results for the comparable period in 2023 represent a consolidated summary of the combined entity. For simplicity, the explanatory statements below provide a high-level summary of expenses for the quarter ended June 30, 2024.

Cash, cash equivalents and short-term investments: As of June 30, 2024, the Company had cash, cash equivalents, and short-term investments of approximately $16.9 million, compared to cash, cash equivalents, and short-term investments of approximately $20.8 million at December 31, 2023. The company believes its cash balance is adequate to support planned operations through year end 2024.

Acquired in-process R&D related to our transaction early in the second quarter resulted in a non-cash charge of $117.5 million.

Research and development (R&D) expense for the three months ended June 30, 2024, totaled $4.0 million, compared to $2.5 million for the comparable period in 2023. Q2 2024 R&D expenses mainly reflect the cost of the Phase 1 SAD/MAD COVID-19 study, the Phase 1 extension study in flu, completion of the narazaciclib Phase 1/2 dose escalation study, regulatory expenses to support planned upcoming studies, and expenses related to stock-based compensation and restructuring costs related to the recent transaction.

General and administrative (G&A) expense for the three months ended June 30, 2024, totaled $2.0 million compared to $2.2 million for the comparable period in 2023. Q2 2024 G&A expenses include stock-based compensation and restructuring costs related to the merger.

Net loss: The net loss for the three months ended June 30, 2024 was $123.1 million, or $4.87 per basic and diluted common share, which reflects a non-cash charge of $117.5 million related to in-process R&D from Onconova’s April 2024 acquisition of Trawsfynydd. This compares with a net loss of $4.3 million, or $0.20 per basic and diluted common share, for the same period in 2023.

Conference Call and Webcast Information

Traws Pharma will host a conference call and webcast today, August 15, 2024, at 8:00 AM ET to discuss recent business progress and second quarter financial results. To access the call, please dial:
1 (877) 407-0789 (United States) or 1 (201) 689-8562 (International) and reference the conference ID “13748066”. To access the webcast, please click: Traws Pharma Corporate Update Call. The live and archived webcast can also be accessed by visiting the “Corporate Events & Presentations” tab of the Events and Presentations section of the Investor Relations page. A replay of the webcast will be archived for 90 days.

About Traws Pharma, Inc.

Traws Pharma is a clinical stage biopharmaceutical company developing oral small molecule therapies for the treatment of respiratory viral diseases and cancer. The viral respiratory disease program includes two potentially best-in-class oral small molecules in Phase 1 studies: tivoxavir marboxil, a novel oral antiviral drug candidate for influenza and avian flu, targeting the influenza cap-dependent endonuclease, and ratutrelvir, targeting Mpro (3CL protease).

In the cancer program, Traws is developing the novel, proprietary multi-kinase CDK4-plus inhibitor narazaciclib, with potential for refractory endometrial cancer and potentially other solid tumor cancers, and rigosertib, multi-kinase inhibitor targeting cell cycle proteins including PLK-1, with potential for use in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC, RDEB).

Traws Pharma is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a focus on product safety and a commitment to patients in need or that are specifically vulnerable, we aim to build solutions for important medical challenges and alleviate the burden of viral infections and cancer.

Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties including statements regarding Traws Pharma, its respective businesses, the merger agreement with Trawsfynydd Therapeutics, Inc. and concurrent private placement and the use of proceeds from such financing and the Company’s financing strategies, as well as statements regarding the milestones, preclinical studies and clinical studies for its four product candidates, tivoxavir marboxil for influenza including avian flu, ratutrelvir for COVID-19, and narazaciclib and rigosertib for cancer, related to the design, timing and potential results and the timing of next steps. Traws has attempted to identify forward-looking statements by terminology including “believes”, “estimates”, “anticipates”, “expects”, “plans”, “intends”, “may”, “could”, “might”, “will”, “should”, “preliminary”, “encouraging”, “approximately” or other words that convey uncertainty of future events or outcomes. Although Traws believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Traws’ clinical trials, collaborations, merger integration, market conditions and those discussed under the heading “Risk Factors” in Traws’ filings with the Securities and Exchange Commission. Any forward-looking statements contained in this release speak only as of its date. Traws undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Traws Pharma Contact:
Mark Guerin
Traws Pharma, Inc.
267-759-3680
www.trawspharma.com

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
bmackle@lifesciadvisors.com

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Release – Eledon Pharmaceuticals Reports Preliminary Second Quarter 2024 Operating Results

Posted on August 15, 2024August 15, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on ELDN

Presented updated data on 13 participants from ongoing Phase 1b trial evaluating tegoprubart for prevention of rejection in kidney transplantation

80 participants (two-thirds of projected recruitment) enrolled in Phase 2 BESTOW trial

Completed an oversubscribed $50 million private placement; Company expects sufficient liquidity through December 2025

IRVINE, Calif., Aug. 14, 2024 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (Nasdaq: ELDN) today reported recent business highlights for its second quarter 2024.

“We have entered the second half of the year with a strong balance sheet following our oversubscribed $50 million private placement and we are highly encouraged by the progress and reception from the transplant community for our Phase 2 BESTOW trial, which remains on track to complete enrollment by the end of this year,” said David-Alexandre C. Gros, M.D., Chief Executive Officer of Eledon. “Looking at this progress and the data we presented in June, we continue to believe that tegoprubart has the potential to displace calcineurin inhibitors, the current standard of care, as a first-line immunosuppression agent for patients undergoing kidney transplant.”

Second Quarter 2024 and Recent Corporate Developments

Enrolled the 80th participant in July 2024 in the ongoing Phase 2 BESTOW trial assessing tegoprubart head-to-head with tacrolimus for the prevention of organ rejection in kidney transplantation.
Presented updated data at the American Transplant Congress (ATC) in June 2024 from the ongoing Phase 1b open-label trial evaluating tegoprubart for the prevention of organ rejection in kidney transplant patients. Updated data from 13 participants demonstrated that tegoprubart was generally safe and well tolerated, with an overall mean estimated glomerular filtration rate (eGFR) of all reported time points after day 30 post-transplant of 70.5 mL/min/1.73m2. Two participants completed over 12 months on therapy post-transplant, and both demonstrated mean eGFRs above 90 mL/min/1.73m2 at one-year post-transplant.
Completed an oversubscribed private placement financing for total gross proceeds of $50.0 million, before deducting any offering related expenses.

Anticipated Upcoming Milestones

End of 2024: Complete enrollment in the Phase 2 BESTOW trial of tegoprubart in kidney transplantation.
Mid-2025: Report updated interim clinical data from the ongoing Phase 1b and long-term safety and efficacy extension studies of tegoprubart in kidney transplantation.

Financial Results

In the course of preparing the Company’s financial statements as of and for the three and six months ended June 30, 2024, the Company, in consultation with Crowe LLP, the Company’s independent registered public accounting firm, determined that a reclassification was necessary with respect to the Company’s reporting and recording of the fair value of certain common stock warrants and pre-funded warrants associated with the Company’s Securities Purchase Agreement dated as of April 28, 2023 (and the potential second and third closings thereof), resulting in a reclassification of these warrants as liabilities on the Company’s balance sheet, on a mark-to-market basis.

The Company expects to restate its audited consolidated financial statements that appeared in its Annual Report on Form 10-K filed with the Securities and Exchange Commission (the “SEC”) on March 28, 2024, as amended on April 26, 2024, and its unaudited condensed consolidated financial statements that appeared in the Company’s Quarterly Report on Form 10-Q filed with the SEC on May 15, 2024 (together, the “Impacted Reports”). As previously disclosed on the Company’s Form 12b-25 Notification of Late Filing filed with the SEC today, the Company also expects to delay the filing of its Form 10-Q for the three and six months ended June 30, 2024 in light of the time and resources needed to prepare a complete and accurate Form 10-Q in light of the restatement process . See also the Company’s Current Report on Form 8-K filed today for additional information.

This accounting reclassification is non-cash and is not expected to have an economic impact on the Company’s operations or on the Company’s cash, cash equivalents and short-term investments, or cash runway.

Eledon ended the second quarter with approximately $83.6 million in cash and cash equivalents, which includes the $50.0 million received in the private placement financing transaction during the second quarter.

About Eledon Pharmaceuticals and tegoprubart

Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for CD40 Ligand (CD40L), a well-validated biological target within the costimulatory CD40/CD40L cellular pathway. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40L biology to conduct preclinical and clinical studies in allogeneic kidney transplantation, xenotransplantation, and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com.

Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. Any statements about the company’s future expectations, plans and prospects, including statements about enrollment in our clinical trials, the development and future success of product candidates, the company’s capital resources and ability to finance operations through December 2025, our filing of amendments to the Impacted Reports and our Form 10-Q for the three and six months ended June 30, 2024, as well as other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “estimates,” “intends,” “predicts,” “projects,” “targets,” “looks forward,” “could,” “may,” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are inherently uncertain and are subject to numerous risks and uncertainties, including: risks relating to the completion of our financial closing procedures; final adjustments; completion of the review by our independent registered public accounting firm; the safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sites, as well as patient enrollment; risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials; and risks associated with the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the company’s actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Qs, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Contact:

Stephen Jasper
Gilmartin Group
(858) 525 2047
stephen@gilmartinir.com

Media Contact:

Jenna Urban
Berry & Company Public Relations
(212) 253 8881
jurban@berrypr.com

Source: Eledon Pharmaceuticals

Release – Cocrystal Pharma Reports Second Quarter 2024 Financial Results and Provides Updates on its Antiviral Drug-Development Programs

Posted on August 14, 2024August 14, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on COCP

Expects to report topline results in 2025 from Phase 2a influenza A human challenge study with oral CC-42344, including initial indication of virology
In vitro testing shows CC-42344 inhibits the avian influenza A (H5N1) PB2 protein recently identified in U.S. dairy cows
Expects to report topline results in late 2024 or early 2025 from Phase 1 study with oral CDI-988, the first potential pan-coronavirus/pan-norovirus oral antiviral
Plans to initiate Phase 1 study in 2025 with inhaled CC-42344, a potential influenza treatment and post-exposure prophylactic

BOTHELL, Wash., Aug. 14, 2024 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) reports financial results for the three and six months ended June 30, 2024, and provides updates on its antiviral product pipeline, upcoming milestones and business activities.

“We are rapidly approaching major inflection points in our clinical programs,” said Sam Lee, Ph.D., President and co-CEO of Cocrystal. “In the coming months we expect to report topline results from our Phase 2a study with PB2 inhibitor CC-42344 including an initial indication of virology in humans infected with the influenza A virus. Our plan is to file an Investigational New Drug (IND) application in 2025 to conduct our next study in the U.S. We further validated CC-42344’s broad-spectrum activity through in vitro testing demonstrating it inhibits the new, highly pathogenic avian flu PB2 protein identified as infecting U.S. dairy cows. We also are preparing to initiate a Phase 1 study in healthy volunteers as the first clinical step in evaluating inhaled CC-42344 as a potential prophylactic and therapeutic for influenza A.

“Preparations are underway to begin the multiple-ascending dose portion of the first-in-human study with our pan-norovirus/pan-coronavirus oral protease inhibitor CDI-988, following favorable safety and tolerability data from the single-ascending dose (SAD) portion of this study,” said Dr. Lee. “We expect to report topline results from the full study in late 2024 or early 2025.”

“I’m pleased to report that through our cost-efficient business model, we expect our cash to be sufficient to advance our planned development programs through the coming 12 months,” said James Martin, CFO and co-CEO of Cocrystal.

Antiviral Product Pipeline Overview

We apply our proprietary structure-based drug discovery platform technology for developing broad-spectrum antivirals that inhibit viral replication. By designing and selecting antiviral drug candidates that target the highly conserved regions of the viral enzymes, we seek to develop drugs that are effective against the virus and mutations of the virus, and also have reduced off-target interactions that may cause undesirable side effects. Our drug discovery process differs from traditional, empirical medicinal chemistry approaches that often require iterative high-throughput compound screening and lengthy hit-to-lead processes.

Influenza Programs
Influenza is a major global health threat that may become more challenging to treat due to the emergence of highly pathogenic avian influenza viruses and resistance to approved influenza antivirals. Each year there are approximately 1 billion cases of seasonal influenza worldwide, 3-5 million severe illnesses and up to 650,000 deaths, according to the World Health Organization. On average, about 8% of the U.S. population contracts influenza each season. In addition to the health risk, influenza is responsible for approximately $10.4 billion in direct costs for hospitalizations and outpatient visits for adults in the U.S. annually.

Oral CC-42344 for the treatment of pandemic and seasonal Influenza A infections
- Our novel PB2 inhibitor CC-42344 showed excellent in vitro antiviral activity against pandemic and seasonal influenza A strains, as well as strains that are resistant to Tamiflu^® and Xofluza^®.
- In March 2022 we initiated enrollment in a randomized, double-blind, dose-escalating Phase 1 study to evaluate the safety, tolerability and pharmacokinetics (PK) of oral CC-42344 in healthy adults.
- In July 2022 we reported PK results from the SAD portion of the study that support once-daily dosing.
- In December 2022 we reported favorable safety and tolerability results from the oral CC-42344 Phase 1 study.
- In April 2023 we received authorization from United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) for an oral CC-42344 Phase 2a human challenge study.
- In December 2023 we began a randomized, double-blind, placebo-controlled Phase 2a study to evaluate the safety, tolerability, viral and clinical measurements of CC-42344 in influenza A-infected subjects.
- In March 2024 we received feedback from the FDA on a Pre-IND package improving clarity on clinical study design, drug manufacturing and nonclinical studies necessary to file a Phase 2b study design.
- In May 2024 we completed enrollment in the Phase 2a human challenge study.
- In June 2024 we reported that in vitro testing showed CC-42344 inhibited the activity of the highly pathogenic avian influenza A (H5N1) PB2 protein that was identified as infecting U.S. dairy cows.
- We expect to report topline results from the Phase 2a human challenge study in 2024 and to plan to file an IND application in 2025 to conduct a late-stage study in the U.S.
Inhaled CC-42344 for the treatment of pandemic and seasonal Influenza A infections
- GLP toxicology study is underway with inhaled CC-42344 as a potential therapeutic and post-exposure prophylaxis for influenza A. CC-42344 has exhibited superior pulmonary exposure in preclinical studies.
- We expect to begin a Phase 1 study with inhaled CC-42344 in Australia in 2025.
Influenza A/B Program
- Preclinical lead development of novel influenza replication inhibitors is underway.

Norovirus Program
Norovirus is a highly contagious infection and is the most common cause of acute gastroenteritis, accounting for nearly one in five cases. According to the Centers for Disease Control and Prevention (CDC), an estimated 685 million cases and an estimated 50,000 child deaths are attributed to norovirus each year worldwide, with an estimated societal cost of $60 billion. By targeting viral replication, we believe it is possible to develop an effective treatment and/or short-term prophylactic for closed environments for all genogroups of norovirus.

Oral pan-viral protease inhibitor CDI-988 for the treatment of norovirus and coronavirus infections
- Our novel broad-spectrum protease inhibitor CDI-988 is being evaluated as a potential oral treatment for noroviruses and coronaviruses.
- CDI-988 has shown pan-viral activity against multiple norovirus strains, including the genogroup II, genotype 4 (GII.4) norovirus strain that is responsible for major norovirus outbreaks.
- In May 2023 we announced approval of our application to the Australian regulatory agency for a randomized, double-blind, placebo-controlled Phase 1 study to evaluate the safety, tolerability and PK of oral CDI-988 in healthy volunteers.
- In August 2023 we announced our selection of CDI-988 as our lead for the oral treatment for norovirus, in addition to coronavirus.
- In September 2023 we began dosing subjects in a first-in-human study in healthy volunteers in Australia with oral CDI-988.
- In July 2024 we reported favorable safety and tolerability results from the SAD cohorts in the Phase 1 study.
- We expect to report topline results from the CDI-988 Phase 1 study in late 2024 or early 2025.

COVID-19 and Other Coronavirus Programs
By targeting viral replication enzymes and protease, we believe it is possible to develop effective treatments for all diseases caused by coronaviruses including COVID-19, Severe Acute Respiratory Syndrome (SARS) and Middle East Respiratory Syndrome (MERS). CDI-988 showed potent in vitro pan-viral activity against common human coronaviruses, rhinoviruses and respiratory enteroviruses, as well as against noroviruses. The global COVID-19 therapeutics market is estimated to exceed $16 billion by the end of 2031.

Oral pan-viral protease inhibitor CDI-988 for the treatment of coronaviruses and noroviruses
- CDI-988 exhibited superior in vitro potency against SARS-CoV-2 and demonstrated a favorable safety profile and PK properties.
- In September 2023 we dosed the first subject in our dual norovirus/coronavirus oral CDI-988 study, which is expected to serve as a Phase 1 study for both indications.
- In July 2024 we reported favorable safety and tolerability results from the SAD cohorts in the Phase 1 study.
- We expect to report topline results from the CDI-988 Phase 1 study in late 2024 or early 2025.

Second Quarter Financial Results

Research and development (R&D) expenses for the second quarter of 2024 were $4.3 million, compared with $2.8 million for the second quarter of 2023. The increase was primarily due to CC-42344 entering into a Phase 2a clinical study and norovirus and coronavirus candidate CDI-988 entering into a Phase 1 clinical study. General and administrative (G&A) expenses for the second quarter of 2024 were $1.1 million, compared with $1.5 million for the second quarter of 2023, with the decrease mainly due to lower legal expenses.

The net loss for the second quarter of 2024 was $5.3 million, or $0.54 per share, compared with a net loss for the second quarter of 2023 of $4.2 million, or $0.41 per share.

Six Month Financial Results

R&D expenses for the first six months of 2024 were $7.3 million, compared with $6.7 million for the first six months of 2023. G&A expenses for the first six months of 2024 were $2.3 million, compared with $2.7 million for the first six months of 2023.

The net loss for the first six months of 2024 was $9.3 million, or $0.91, per share, compared with a net loss for the first six months of 2023 of $9.4 million, or $1.03 per share.

Cocrystal reported unrestricted cash as of June 30, 2024 of $18.1 million, compared with $26.4 million as of December 31, 2023. Net cash used in operating activities for the first six months of 2024 was $8.2 million, compared with $8.7 million for the first six months of 2023. The Company had working capital of $17.0 million and 10.2 million common shares outstanding as of June 30, 2024.

About Cocrystal Pharma, Inc.

Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), noroviruses and hepatitis C viruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding our plans for the future development of preclinical and clinical drug candidates, our expectations regarding future characteristics of the product candidates we develop, the expected time of achieving certain value-driving milestones in our programs, including preparation, commencement and advancement of clinical studies for certain product candidates in 2024 and 2025, the viability and efficacy of potential treatments for diseases our product candidates are designed to treat, expectations for the markets for certain therapeutics, our ability to execute our clinical and regulatory goals and deploy regulatory guidance towards future studies, and the expected sufficiency of our cash balance to advance our programs and fund our planned operations. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, the risks and uncertainties arising from the high interest rates in response to inflation, uncertainty in the financial markets, the possibility of a recession, and geopolitical conflict in Ukraine and Israel on our Company, our collaboration partners, and on the U.S., UK, Australia and global economies, including manufacturing and research delays arising from raw materials and labor shortages, supply chain disruptions and other business interruptions on our ability to proceed with studies as well as similar problems with our vendors and our current and any future clinical research organization (CROs) and contract manufacturing organizations (CMOs), the ability of our CROs to recruit volunteers for, and to proceed with, clinical studies, our and our collaboration partners’ technology and software performing as expected, financial difficulties experienced by certain partners, the results of any current and future preclinical and clinical studies, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes, and potential development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government, potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2023. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100
jcain@lhai.com

Media Contact:
JQA Partners
Jules Abraham
917-885-7378
Jabraham@jqapartners.com

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Release – Unicycive Announces Second Quarter 2024 Financial Results and Provides Business Update

Posted on August 14, 2024August 14, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on UNCY

– On Track to Submit OLC New Drug Application (NDA) by End of August 2024 –

LOS ALTOS, Calif., Aug. 14, 2024 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY) (the “Company” or “Unicycive”), a clinical-stage biotechnology company developing therapies for patients with kidney disease, today announced its financial results for the three months ended June 30, 2024, and provided a business update.

“Achieving successful results from our oxylanthanum carbonate (OLC) pivotal trial was a significant milestone for the company and brings us one step closer to becoming a commercial organization,” said Shalabh Gupta, M.D., Chief Executive Officer of Unicycive. “Importantly, the trial confirmed tolerability of OLC in patients with hyperphosphatemia on dialysis which is the final data component needed to support submission of a New Drug Application (NDA) to the FDA utilizing the 505(b)(2) regulatory pathway. In addition, we were able to achieve phosphate control in 90% of patients at the end of their titration. Our recent pharmacokinetic analysis of samples from the pivotal study revealed that the systemic exposure of our drug is minimal and, as expected, the serum lanthanum levels are similar to that seen with Fosrenol^®. With this data, we believe that we have completed all the necessary requirements from this pivotal clinical trial to fulfill the FDA’s requests. We remain on track to submit our NDA by the end of this month, and we maintain a high degree of confidence in the potential for OLC to be a best-in-class commercial product, if approved.”

“In July 2024 we were granted a new patent for UNI-494 by the USPTO which is an important component of our development strategy to target patients with acute kidney injury (AKI), a serious condition resulting from a sudden loss of kidney function. We have completed enrollment in the UNI-494 Phase 1 dose-ranging study and expect to report results in the third quarter of this year. With assets targeting both chronic and acute kidney conditions, we remain steadfastly focused on improving treatment options and overall quality of life for patients living with renal diseases,” concluded Dr. Gupta.

Key Highlights

Reported positive topline data from the pivotal clinical trial of OLC with regard to both safety and tolerability endpoints. The study established promising tolerability of OLC at clinically effective doses in chronic kidney disease (CKD) patients on hemodialysis. In terms of tolerability, OLC had a low rate of discontinuation due to adverse events (AEs) with only 5/86 patients (6%) discontinuing from the Study. The primary endpoint was defined as the rate of discontinuations due to treatment-related AEs leading to discontinuation in the maintenance period. In the UNI-OLC-201 trial, the discontinuation rate was 1.4%, as there was only 1 discontinuation due to a treatment-related AE in the Evaluable Population (n=71). In the full Safety Population (n=86), a total of 3 patients discontinued due to treatment-related AEs, a rate of 3.5%. There were no treatment-related serious adverse events (SAEs).
Announced initial results from the patient reported outcome survey conducted during the UNI-OLC-201 pivotal clinical trial. In the survey, OLC consistently outperformed the other phosphate binders in all categories: 79% of patients preferred OLC while 18% preferred their prior therapy, 98% of patients said that OLC was easy to take compared to 55% for their prior therapy, 89% of patients said they were satisfied with OLC while 49% were satisfied with their prior therapy.
Enrollment in the UNI-494 Phase 1 study is complete, and the Company expects to present the data in Q3 2024.
Granted a patent on UNI-494 to treat AKI by the United States Patent and Trademark Office (USPTO). The patent, valid until 2040, secures protection of a method of treating a disease or a condition selected from AKI or contrast induced nephropathy by administering the UNI-494 compound.
Included in the Russell Microcap^® Index effective July 1, 2024. Membership in the Russell Microcap^® Index, which remains in place for one year, means automatic inclusion in the appropriate growth and value style indexes.
Delivered multiple presentations on OLC and UNI-494 at the 61^st European Renal Association (ERA) Congress including two oral presentations and trial-in-progress posters on OLC and UNI-494. An oral presentation demonstrated a significant reduction in urinary phosphate excretion for OLC compared to vehicle treated animals. A second oral presentation evaluated the in vivo efficacy of UNI-494 and showed that a single oral dose of UNI-494 significantly reduced important kidney functional markers.
Presented two posters related to OLC at the National Kidney Foundation (NKF) Spring Clinical Meeting. Importantly, it was demonstrated that OLC is bioequivalent to lanthanum carbonate from the Phase 1, single-center, randomized 1:1, open-label, controlled, 2-way crossover study. In addition, a poster presentation on the findings of a survey of 100 renal dieticians concluded that strategies that reduce pill burden and increase ease of use for patients are needed. This poster was among the top-rated submissions to the Meeting.

Financial Results for the Quarter Ended June 30, 2024

Research and Development (R&D) expenses were $4.9 million for the three months ended June 30, 2024, compared to $2.3 million for the three months ended June 30, 2023. The increase in research and development expenses was primarily due to increased drug development costs.

General and Administrative (G&A) expenses were $2.5 million for the three months ended June 30, 2024, compared to $2.1 million for the three months ended June 30, 2023. The increase was primarily due to increased non-cash stock compensation costs.

Other Income (Expense) was $17.3 million for the three months ended June 30, 2024 compared to $0.5 million in the three months ended June 30, 2023, due primarily to a decrease in the fair value of our warrant liability.

Net income attributable to common stockholders for the three months ended June 30, 2024 was $3.0 million, and basic earnings per share was $0.08. On a diluted basis, we reported a loss per share for the same period of $0.15. The net income for the three-month period ended June 30, 2024 was attributable to a decrease in the fair value of our warrant liability. For the three months ended June 30, 2023, we reported a net loss of $4.4 million, and basic loss per share of $0.29. On a diluted basis, we reported a loss per share for the same period of $0.29.

As of June 30, 2024, cash and cash equivalents totaled $41.8 million. The Company believes that it has sufficient resources to fund planned operations into 2026.

About Unicycive Therapeutics

Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug candidate, oxylanthanum carbonate (OLC), is a novel investigational phosphate binding agent being developed for the treatment of hyperphosphatemia in chronic kidney disease patients on dialysis. UNI-494 is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. For more information, please visit Unicycive.com and follow us on LinkedIn and YouTube.

Forward-looking statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified using words such as “anticipate,” “believe,” “forecast,” “estimated” and “intend” or other similar terms or expressions that concern Unicycive’s expectations, strategy, plans or intentions. These forward-looking statements are based on Unicycive’s current expectations and actual results could differ materially. There are several factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions which could seriously harm our financial condition and increase our costs and expenses; dependence on key personnel; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Unicycive’s Annual Report on Form 10-K for the year ended December 31, 2023, and other periodic reports filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Unicycive specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Fosrenol^® is a registered trademark of Shire International Licensing BV.

Investor Contact:

ir@unicycive.com
(650) 900-5470

SOURCE: Unicycive Therapeutics, Inc.

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Release – Schwazze Announces Second Quarter 2024 Financial Results

Posted on August 14, 2024August 14, 2024 by dburczyk@noblecapitalmarkets.com

Research News and Market Data on SHWZ

Growth and Restructuring Initiatives Lead to Quarter-over-Quarter Growth Across all Key Financial Metrics in Q2

Schwazze Management to Host Conference Call Today at 5:00 p.m. Eastern Time

DENVER, Aug. 13, 2024 (GLOBE NEWSWIRE) — Medicine Man Technologies, Inc., operating as Schwazze, (OTC: SHWZ) (Cboe CA: SHWZ) (“Schwazze” or the “Company”), today announced financial and operational results for the second quarter ended June 30, 2024.

“We made solid progress on our growth and optimization initiatives in Q2 and generated sequential quarterly growth across all key financial metrics while advancing our retail strategy,” said Forrest Hoffmaster, Interim CEO of Schwazze. “During the quarter, we continued to deepen our customer understanding, sharpen our pricing and promotional strategy, enhance the in-store experience, and improve our assortment and in-stock positions. These efforts drove increased store traffic and market share expansion in both Colorado and New Mexico. In our wholesale business, we generated our second consecutive period of quarter-over-quarter growth in both states with penetration growth and catalog expansion while improving wholesale margins.”

“To drive growth in the competitive Colorado environment, we continued to elevate the retail experience and loyalty offerings to improve customer acquisition and retention. As a result, we outpaced the market on a year-over-year basis and generated 6% growth in a market that declined 11% during the same period. As part of our restructuring initiative, we shuttered our non-plant touching wholesale operation in Denver and eliminated three underperforming stores that no longer met our high-margin expectations. We continue to evaluate our asset base to ensure we’re running as efficiently as possible while maximizing output.”

“In New Mexico, state cannabis sales were up 7% across a store base that was 20% higher year-over-year in Q2. The state’s regulatory body continued to increase its enforcement, helping lead to a reduction in net new store openings, which we anticipate will flip from positive to negative in the back half of the year. Our consistent efforts to optimize our pricing and promotional strategy, expand assortment with high-quality flower, and deliver an enhanced customer experience is generating momentum. In the second quarter, we grew revenue 9% sequentially compared to the state’s 2%, demonstrating the effectiveness of our operating playbook to compete in challenging environments.”

“Looking ahead, we will continue to refine our retail strategy while further driving operating efficiencies across our retail, cultivation, and manufacturing assets. Our recent debt restructuring provides us with the financial flexibility to execute our strategic growth initiatives in Colorado and New Mexico. Over the past year, our consistent efforts to optimize operations have established a solid foundation, positioning us for continued growth and stronger levels of profitability in the second half of 2024.”

Recent Highlights

In July 2024, Schwazze extended the maturities of its original $15.0 million Altmore, LLC Loan Agreement and its $17.0 million Reynold Greenleaf & Associates LLC Promissory Note to November 2025 (both previously due in February 2025) in a step toward addressing future debt obligations.
Announced the grand opening of a medical and recreational dispensary in June under the R. Greenleaf banner in Bernalillo, New Mexico, increasing the Company’s retail footprint to 35 stores across the state.
Closed the Company’s Colorado distribution center and shuttered its non-plant touching wholesale operations, The Big Tomato, in Colorado to concentrate on core business operations.
Closed three underperforming Colorado dispensaries and streamlined the Company’s corporate office support structure to strengthen its retail forward strategy.
Increased wholesale penetration during the quarter to approximately 34% and 35% of total doors in Colorado and New Mexico, respectively.
Expanded wholesale catalog in New Mexico with the launch of Lowell Farms pre-rolls.
Generated 28% sequential wholesale unit growth in New Mexico with Wana gummies.

¹ Adjusted EBITDA is a non-GAAP measure as defined by the SEC, and represents earnings before interest, taxes, depreciation, and amortization, adjusted for other income, non-cash share-based compensation, one-time transaction related expenses, or other non-operating costs. The Company uses Adjusted EBITDA as it believes it better explains the results of its core business. See “ADJUSTED EBITDA RECONCILIATION (NON-GAAP)” section herein for an explanation and reconciliations of non-GAAP measure used throughout this release.

Second Quarter 2024 Financial Results

Total revenue in the second quarter of 2024 increased 2% to $43.2 million compared to $42.4 million for the same quarter last year. The increase was primarily due to growth from new stores compared to the prior year period, partially offset by lower wholesale revenue and continued pricing pressure from the proliferation of new licenses in New Mexico.

Gross profit for the second quarter of 2024 was $19.0 million or 44.0% of total revenue, compared to $23.0 million or 54.4% of total revenue for the same quarter last year. The decrease in gross margin was primarily driven by the aforementioned pricing pressure and greater mix of third-party purchasing in New Mexico to broaden assortment in the state, as well as higher medical sales mix in Colorado.

Operating expenses for the second quarter of 2024 were $21.8 million compared to $18.1 million for the same quarter last year. The increase was primarily driven by four-wall SG&A costs associated with five additional stores in Colorado and New Mexico, as well as non-recurring professional service fees related to prior period workpaper review stemming from work required to comply with SEC based on their Order against BF Borgers.

Loss from operations for the second quarter of 2024 was $2.7 million compared to income from operations of $5.0 million in the same quarter last year. Net loss was $13.9 million for the second quarter of 2024 compared to $6.6 million for the same quarter last year.

Adjusted EBITDA for the second quarter of 2024 was $9.0 million compared to $13.8 million for the same quarter last year. The decrease in Adjusted EBITDA was primarily driven by lower gross margin and higher operating expenses.

As of June 30, 2024, cash and cash equivalents were $12.3 million compared to $19.2 million on December 31, 2023. Total debt as of June 30, 2024, was $163.4 million compared to $156.8 million on December 31, 2023.

Conference Call

The Company will conduct a conference call today, August 13, 2024, at 5:00 p.m. Eastern time to discuss its results for the second quarter ended June 30, 2024.

Schwazze management will host the conference call, followed by a question-and-answer period. Interested parties may submit questions to the Company prior to the call by emailing ir@schwazze.com.

Date: Tuesday, August 13, 2024
Time: 5:00 p.m. Eastern time
Toll-free dial-in: (844) 825-9789
International dial-in: (412) 317-5180
Conference ID: 10191294
Webcast: SHWZ Q2 2024 Earnings Call

The conference call will also be broadcast live and available for replay on the investor relations section of the Company’s website at https://ir.schwazze.com.

Toll-free replay number: (844) 512-2921
International replay number: (412) 317-6671
Replay ID: 10191294

If you have any difficulty registering or connecting with the conference call, please contact Elevate IR at (720) 330-2829.

About Schwazze

Schwazze (OTC: SHWZ) (Cboe CA: SHWZ) is building a premier vertically integrated regional cannabis company with assets in Colorado and New Mexico and will continue to explore taking its operating system to other states where it can develop a differentiated regional leadership position. Schwazze is the parent company of a portfolio of leading cannabis businesses and brands spanning seed to sale.

Schwazze is anchored by a high-performance culture that combines customer-centric thinking and data science to test, measure, and drive decisions and outcomes. The Company’s leadership team has deep expertise in retailing, wholesaling, and building consumer brands at Fortune 500 companies as well as in the cannabis sector.

Medicine Man Technologies, Inc. was Schwazze’s former operating trade name. The corporate entity continues to be named Medicine Man Technologies, Inc. Schwazze derives its name from the pruning technique of a cannabis plant to enhance plant structure and promote healthy growth. To learn more about Schwazze, visit https://schwazze.com/.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements include financial outlooks; any projections of net sales, earnings, or other financial items; any statements of the strategies, plans and objectives of our management team for future operations; expectations in connection with the Company’s previously announced business plans; any statements regarding future economic conditions or performance; and statements regarding the intent, belief or current expectations of our management team. Such statements may be preceded by the words “may,” “will,” “could,” “would,” “should,” “expect,” “intends,” “plans,” “strategy,” “prospects,” “anticipate,” “believe,” “approximately,” “estimate,” “predict,” “project,” “potential,” “continue,” “ongoing,” or the negative of these terms or other words of similar meaning in connection with a discussion of future events or future operating or financial performance, although the absence of these words does not necessarily mean that a statement is not forward-looking. We have based our forward-looking statements on management’s current expectations and assumptions about future events and trends affecting our business and industry. Although we do not make forward-looking statements unless we believe we have a reasonable basis for doing so, we cannot guarantee their accuracy. Therefore, forward-looking statements are not guarantees of future events or performance, are based on certain assumptions, and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company’s control and cannot be predicted or quantified. Consequently, actual events and results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) regulatory limitations on our products and services and the uncertainty in the application of federal, state, and local laws to our business, and any changes in such laws; (ii) our ability to manufacture our products and product candidates on a commercial scale on our own or in collaboration with third parties; (iii) our ability to identify, consummate, and integrate anticipated acquisitions; (iv) general industry and economic conditions; (v) our ability to access adequate capital upon terms and conditions that are acceptable to us; (vi) our ability to pay interest and principal on outstanding debt when due; (vii) volatility in credit and market conditions; (viii) the loss of one or more key executives or other key employees; and (ix) other risks and uncertainties related to the cannabis market and our business strategy. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company’s filings with the Securities and Exchange Commission (SEC), including the Company’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Investors and security holders are urged to read these documents free of charge on the SEC’s website at http://www.sec.gov. The Company assumes no obligation to publicly update or revise its forward-looking statements as a result of new information, future events or otherwise except as required by law.

Investor Relations Contact
Sean Mansouri, CFA or Aaron D’Souza
Elevate IR
(720) 330-2829
ir@schwazze.com

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GoHealth, Inc. (GOCO) – Is there a Tailwind in the Forecast?

Posted on August 9, 2024August 9, 2024 by slightbourne@noblecapitalmarkets.com

Friday, August 09, 2024

Patrick McCann, CFA, Research Analyst, Noble Capital Markets, Inc.

Michael Kupinski, Director of Research, Equity Research Analyst, Digital, Media & Technology , Noble Capital Markets, Inc.

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Q2 results below estimates. The company reported Q2 revenue and adj. EBITDA of $105.9 million and a loss of $12.3 million, respectively, below our estimates. Our revenue and adj. EBITDA estimates were $144.0 million and a loss of $6.3 million, respectively.

Preparing for AEP. The company has been testing Plan Fit Save, an initiative whereby it is compensated by health plan carriers for improving customer retention when GoHealth recommends consumers to keep their existing plans. We believe the combination Plan Fit Save, as well as the prospect for disruption to health plan benefits in the upcoming Annual Enrollment Period, could set the company up for a strong finish to the year.

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Ocugen (OCGN) – 2Q24 Reported With Summary Of Progress In Clinical Trials

Posted on August 9, 2024August 9, 2024 by slightbourne@noblecapitalmarkets.com

Friday, August 09, 2024

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

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Ocugen reported a 2Q24 loss of $10.3 million or $(0.04) per share. During the quarter, the first patient was treated in the Phase 3 liMeliGhT (pronounced “limelight”) trial for OCU400 in retinitis pigmentosa (RP). Separately, the Phase 1/2 OCU410 trial for geographic atrophy in dry AMD completed dosing of its third cohort and began Phase 2. Cash on June 30 was $15.7 million, excluding $32.6 million raised in a public offering on August 2. Based on our quarterly estimates, we project cash to last until 2H25 with about $40 million in cash at the end of 3Q24.

OCU400 Began The Phase 3 liMeliGht Trial and Expanded Access. During the quarter, the first patient in the Phase 3 liMeliGhT trial testing OCU400 in retinitis pigmentosa (RP) was treated. The trial has one arm testing OCU400 in patients that have the RHO mutation and another arm with any of several gene mutations associated with RP. Each arm will have 75 patients for a total target enrollment of 150 patients.

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