Category: Health

Release – Tonix Pharmaceuticals Announces FDA Acceptance of the New Drug Application (NDA) for TNX-102 SL for Fibromyalgia

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

December 17, 2024 8:00am EST Download as PDF

FDA is expected to assign a Prescription Drug User Fee Act (PDUFA) target action date and announce whether Priority Review has been granted in the Day 74 Letter

TNX-102 SL is a non-opioid, centrally acting analgesic, granted Fast Track designation by FDA

Fibromyalgia affects more than 10 million adults in the U.S. who are mostly women

TNX-102 SL has the potential to be the first member of a new class of analgesic drugs for fibromyalgia and the first new drug for treating fibromyalgia in more than 15 years, if approved by FDA

NDA based on two statistically significant Phase 3 studies of TNX-102 SL for the management of fibromyalgia; in which TNX-102 SL was generally well tolerated

CHATHAM, N.J., Dec. 17, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for TNX-102 SL (cyclobenzaprine HCl sublingual tablets), a 5.6 mg, non-opioid, centrally-acting analgesic, for the management of fibromyalgia. The FDA is expected to assign the NDA a Prescription Drug User Fee Act (PDUFA) target action date in a Day 74 Letter. At that time, the FDA will also communicate to Tonix whether Priority Review has been granted. TNX-102 SL was granted Fast Track designation for fibromyalgia by the FDA in July of 2024. Fast Track is designed to expedite FDA review of important new drugs to treat serious conditions and fill an unmet medical need.

“The FDA’s acceptance of our NDA represents another step forward as we pursue our goal of delivering the first member of a new class of medicines for the management of fibromyalgia, a condition affecting over 10 million adults in the U.S.,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “The fibromyalgia community, comprised of patients and their families and support groups, has been waiting for a new drug for over 15 years. Analysis of insurance claims in the U.S., commissioned by Tonix, have shown that 18 months after diagnosis, fibromyalgia patients were more likely to be prescribed addictive opioids than all three of the FDA-approved drugs combined.”

Dr. Lederman continued, “We look forward to working closely with the FDA throughout the NDA review period with the goal of bringing TNX-102 SL to the market to address the significant unmet needs of the fibromyalgia community as quickly as possible. Furthermore, this is an important milestone as we advance our commercial preparations in anticipation of a potential approval in 2025 with an accomplished commercial leadership team already in place, supporting our marketed products 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.”

The NDA is supported by data from two 14-week double-blind, randomized, placebo-controlled Phase 3 clinical trials evaluating the safety and efficacy of TNX-102 SL as a bedtime treatment for fibromyalgia. The first Phase 3 trial, RELIEF, of TNX-102 SL 5.6 mg in fibromyalgia, completed in December 2020, met its pre-specified primary endpoint of significantly reducing daily pain compared to placebo (p=0.010). In the confirmatory Phase 3 RESILIENT study in fibromyalgia, completed in December 2023, TNX-102 SL again met the pre-specified primary endpoint of significantly reducing daily pain compared to placebo (p =0.00005). In both trials, TNX-102 SL was generally well tolerated with an adverse event profile comparable to prior studies and with no new safety signals observed. In both pivotal studies, the most common treatment-emergent adverse event was tongue or mouth numbness at the administration site, which was temporally related to dosing, self-limited, never rated as severe, and rarely led to study discontinuation (one participant in each study). Excluding COVID-19, systemic adverse events in each of the two studies was lower than 4.0%. Tonix believes the submitted dossier contains the requisite safety and efficacy data from two adequate and well-controlled studies to support NDA approval.

About Fibromyalgia

Fibromyalgia is a common chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system, called central sensitization. Brain imaging studies have localized the functional disorder to the brain’s insula and anterior cingulate cortex. Fibromyalgia afflicts more than 10 million adults in the U.S., the majority of whom are women. Symptoms of fibromyalgia include chronic widespread pain, non-restorative sleep, fatigue, and brain fog (or cognitive dysfunction). Other associated symptoms include mood disturbances, including depression, anxiety, headaches and abdominal pain or cramps. Individuals suffering from fibromyalgia often struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products. Fibromyalgia is now recognized as the prototypic nociplastic syndrome. Nociplastic pain is the third primary type of pain in addition to nociceptive pain and neuropathic pain. Many patients present with pain syndromes that are a spectrum of mixtures of the three primary types of pain. Nociplastic syndromes are associated with central and peripheral sensitization. Fibromyalgia can occur without any identifiable precipitating event. However, many fibromyalgia cases follow one or more precipitating event(s) including: post-operative pain, acute or chronic nociceptive or neuropathic pain states; recovery from an infectious illness; a cancer diagnosis or cancer treatment; a metabolic or endocrine stress; or a traumatic event. In the cases of recovery from an infectious illness, fibromyalgia is considered an Infection-Associated Chronic Condition. In addition to fibromyalgia cases associated with other conditions or stressors, the U.S. National Academies of Sciences, Engineering, and Medicine, has concluded that fibromyalgia is a diagnosable condition that can occur after recovery from COVID-19 in the context of Long COVID. Fibromyalgia is also recognized as a Chronic Overlapping Pain Condition, which is a group of related conditions including, chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME), irritable bowel syndrome, endometriosis, low back pain, post-concussive syndrome (also known as mild traumatic brain injury), chronic Lyme Disease, chronic diabetic neuropathy and chronic post-herpetic neuralgia.

About TNX-102 SL

TNX-102 SL is a centrally acting, non-opioid investigational drug, designed for chronic use. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride developed for bedtime dosing for the management of fibromyalgia. Cyclobenzaprine potently binds and acts as an antagonist at four different post-synaptic neuroreceptor subtypes: serotonergic-5-HT2A, adrenergic-α1, histaminergic-H1, and muscarinic-M1-cholinergic receptors. Together, these interactions are believed to target the non-restorative sleep characteristic of fibromyalgia that was identified by Professor Harvey Moldofsky in 1975. Cyclobenzaprine is not associated with risk of addiction or dependence. The TNX-102 SL tablet is based on a eutectic formulation of cyclobenzaprine HCl and mannitol that provides a stable product which dissolves rapidly and delivers cyclobenzaprine by the transmucosal route efficiently into the bloodstream. The eutectic protects cyclobenzaprine HCl from interacting with the basifying agent that is also part of the formulation and required for efficient transmucosal absorption. Patents based on TNX-102 SL’s eutectic composition and its properties have issued in the U.S., E.U., Japan, China and many other jurisdictions around the world and provide market protection into 2034. The European Patent Office’s Opposition Division maintained Tonix’s European Patent EP 2 968 992 in unamended form after an Opposition was filed against it by a Sandoz subsidiary, Hexal AG. Hexal AG did not appeal that decision. The formulation of TNX-102 SL was designed specifically for sublingual administration and transmucosal absorption for bedtime dosing to target disturbed sleep, while reducing the risk of daytime somnolence. Clinical pharmacokinetic studies indicated that relative to oral cyclobenzaprine, TNX-102 SL results in higher levels of exposure during the first 2 hours after dosing and in deceased levels of the long-lived active metabolite, norcyclobenzaprine in both single dose and multiple dose studies, consistent with bypassing first pass hepatic metabolism. At steady state after 20 days of dosing TNX-102 SL, the dynamic peak level of cyclobenzaprine is higher than the background level of norcyclobenzaprine. In contrast, after 20 days of dosing oral cyclobenzaprine, the simulated peak level of cyclobenzaprine is lower than the simulated background level of norcyclobenzaprine.

Tonix Pharmaceuticals Holding Corp.*

Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia. The FDA has accepted the NDA filing for TNX-102 SL for fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation, and its development is supported by a grant from the U.S. National Institute of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified 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. In July 2024, Tonix announced a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years 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. 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; their efficacy and safety have not been established 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 Healthcare
peter.vozzo@icrhealthcare.com
(443) 213-0505

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

Indication and Usage

Zembrace® SymTouch® (sumatriptan succinate) injection (Zembrace) and Tosymra® (sumatriptan) nasal spray are prescription medicines used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine.
Zembrace and Tosymra are not used to prevent migraines. It is not known if Zembrace or Tosymra are safe and effective in children under 18 years of age.

Important Safety Information

Zembrace and Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:

  • discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
  • severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
  • pain or discomfort in your arms, back, neck, jaw or stomach
  • shortness of breath with or without chest discomfort
  • breaking out in a cold sweat
  • nausea or vomiting
  • feeling lightheaded

Zembrace and Tosymra are not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.

Do not use Zembrace or Tosymra if you have:

  • history of heart problems
  • narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
  • uncontrolled high blood pressure
  • hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
  • had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
  • severe liver problems
  • taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider for a list of these medicines if you are not sure.
  • are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
  • an allergy to sumatriptan or any of the components of Zembrace or Tosymra

Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.

Zembrace and Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.

Zembrace and Tosymra may cause serious side effects including:

  • changes in color or sensation in your fingers and toes
  • sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
  • cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
  • increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
  • medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
  • serotonin syndrome, a rare but serious problem that can happen in people using Zembrace or Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
  • hives (itchy bumps); swelling of your tongue, mouth, or throat
  • seizures even in people who have never had seizures before

The most common side effects of Zembrace and Tosymra include: pain and redness at injection site (Zembrance only); tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired; application site (nasal) reactions (Tosymra only) and throat irritation (Tosymra only).

Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace and Tosymra. For more information, ask your provider.

This is the most important information to know about Zembrace and Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit https://www.tonixpharma.com or call 1-888-869-7633.

You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

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Source: Tonix Pharmaceuticals Holding Corp.

Released December 17, 2024

Release – Unicycive Therapeutics Announces Publication of Oxylanthanum Carbonate (OLC) Positive Bioequivalence Data in Clinical Therapeutics

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

December 17, 2024 7:00am EST Download as PDF

LOS ALTOS, Calif., Dec. 17, 2024 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company” or “Unicycive”), today announced that data from the Company’s oxylanthanum carbonate (OLC) bioequivalence study in healthy volunteers was published in the peer-reviewed journal, Clinical Therapeutics.

The publication, entitled, “Two-Way Randomized Crossover Study to Establish Pharmacodynamic Bioequivalence Between Oxylanthanum Carbonate and Lanthanum Carbonate” described the study that established pharmacodynamic (PD) bioequivalence of OLC to Fosrenol®. In the study, OLC was well tolerated and demonstrated bioequivalence to lanthanum carbonate (LC).

“Demonstrating bioequivalence was a key component of our OLC New Drug Application, and we are pleased that these positive data have been published in the peer-reviewed journal, Clinical Therapeutics,” said, Shalabh Gupta, MD, Chief Executive Officer of Unicycive. “With our NDA now under review, we are preparing for commercial launch of OLC in 2025.”

Phosphate binders are integral to hyperphosphatemia management in patients with end-stage kidney disease. This objective of the Unicycive study was to demonstrate the pharmacodynamic equivalence of orally administered OLC to LC in healthy participants. A total of 80 participants were randomized and 75 received all doses. Participants were treated with OLC swallowable 1000 mg tablets three times/day and LC chewable 1000 mg tablets three times/day in a two-way crossover design. The primary pharmacodynamic variable was the least squares mean (LSM) change in urinary phosphate excretion from baseline to the evaluation period (Days 1–4 of treatment). The LSM change in urinary phosphate excretion from Baseline to the Evaluation (Treatment) Period was similar for both OLC (–320.4 mg/day [90% CI: –349.7, –291.0]) and LC (–324.0 mg/day [90% CI: –353.3, –294.7]); the between-group LSM difference was 3.6 [90% CI: –37.8, 45.1] mg/day. Both drugs were well tolerated with an equal incidence of adverse events.

The full publication can be accessed here.

About Oxylanthanum Carbonate (OLC)

Oxylanthanum carbonate is a next-generation lanthanum-based phosphate binding agent utilizing proprietary nanoparticle technology being developed for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD). OLC has over forty issued and granted patents globally. Its potential best-in-class profile may have meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden for patients in terms of number and size of pills per dose that are swallowed instead of chewed. Based on a survey conducted in 2022, Nephrologists stated that the greatest unmet need in the treatment of hyperphosphatemia with phosphate binders is a lower pill burden and better patient compliance.1 The global market opportunity for treating hyperphosphatemia is projected to be in excess of $2.28 billion, with the North America accounting for more than $1 billion of that total.2 Despite the availability of several FDA-cleared medications, 75 percent of U.S. dialysis patients fail to achieve the target phosphorus levels recommended by published medical guidelines.3

Unicycive is seeking FDA approval of OLC via the 505(b)(2) regulatory pathway. The NDA submission package is based on data from three clinical studies (a Phase 1 study in healthy volunteers, a bioequivalence study in healthy volunteers, and a tolerability study of OLC in CKD patients on dialysis), multiple preclinical studies, and the chemistry, manufacturing and controls (CMC) data. OLC is protected by a strong global patent portfolio including issued patents on composition of matter with exclusivity until 2031, and with the potential for patent term extension until 2035.

About Hyperphosphatemia

Hyperphosphatemia is a serious medical condition that occurs in nearly all patients with End Stage Renal Disease (ESRD). If left untreated, hyperphosphatemia leads to secondary hyperparathyroidism (SHPT), which then results in renal osteodystrophy (a condition similar to osteoporosis and associated with significant bone disease, fractures and bone pain); cardiovascular disease with associated hardening of arteries and atherosclerosis (due to deposition of excess calcium-phosphorus complexes in soft tissue). Importantly, hyperphosphatemia is independently associated with increased mortality for patients with chronic kidney disease on dialysis. Based on available clinical data to date, over 80% of patients show signs of cardiovascular calcification by the time they become dependent on dialysis.4

Dialysis patients are already at an increased risk for cardiovascular disease (because of underlying diseases such as diabetes and hypertension), and hyperphosphatemia further exacerbates this. Treatment of hyperphosphatemia is aimed at lowering serum phosphate levels via two means: (1) restricting dietary phosphorus intake; and (2) using, on a daily basis, and with each meal, oral phosphate binding drugs that facilitate fecal elimination of dietary phosphate rather than its absorption from the gastrointestinal tract into the bloodstream.

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. Positive pivotal trial results were reported in June 2024 for OLC, and a New Drug Application (NDA) is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) Target Action Date of June 28, 2025. OLC is protected by a strong global patent portfolio including an issued patent on composition of matter with exclusivity until 2031, and with the potential patent term extension until 2035 after OLC approval. Unicycive’s second asset, UNI-494, is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. UNI-494 has successfully completed a Phase 1 trial. For more information, please visit Unicycive.com and follow us on LinkedIn, X, 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.
1Reason Research, LLC 2022 survey. Results here.
2 Fortune Business InsightsTM, Hyperphosphatemia Treatment Market, 2023-2030
3 US-DOPPS Practice Monitor, May 2021; http://www.dopps.org/DPM
4 Block GA, Klassen PS, Lazarus JM, Ofsthun N, Lowrie EG, Chertow GM. Mineral metabolism, mortality, and morbidity in maintenance hemodialysis. J Am Soc Nephrol. 2004 Aug;15(8):2208-18. doi: 10.1097/01.ASN.0000133041.27682.A2. PMID: 15284307.

Investor Contacts:

Kevin Gardner
LifeSci Advisors
kgardner@lifesciadvisors.com

Chris Calabrese
LifeSci Advisors
ccalabrese@lifesciadvisors.com

SOURCE: Unicycive Therapeutics, Inc.

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Source: Unicycive Therapeutics, Inc.

Released December 17, 2024

Quanterix Advances Scientific Innovation with Strategic Acquisition of EMISSION

Posted on by slightbourne@noblecapitalmarkets.com
Key Points
– Quanterix acquires EMISSION for $10M, expanding its technological capabilities and entering the OEM market.
– EMISSION’s bead technology enhances Quanterix’s Simoa platform for high-multiplex and multi-omic assays.
– The acquisition is expected to drive revenue growth and improve margins by 2026.

Quanterix Corporation (NASDAQ: QTRX), a company advancing scientific discovery through ultrasensitive biomarker detection, has announced the acquisition of EMISSION iNC., a Georgetown, TX-based manufacturer of proprietary magnetic beads and mid-plex assay platforms. The transaction, expected to close in January 2025, aims to vertically integrate EMISSION’s bead technology into Quanterix’s next-generation platform and drive a new multi-plex segment targeting OEM customers.

Masoud Toloue, Quanterix’s CEO, emphasized the importance of controlling core components to expand their technology stack and capabilities. “EMISSION’s proprietary bead technology has already been validated on our upcoming new Simoa platform and will enable us to provide OEM beads to other non-Quanterix platforms. We look forward to welcoming EMISSION’s innovations and colleagues to the Quanterix team,” he stated.

EMISSION’s magnetic beads are designed for low and mid-plex assays, offering high uniformity and scalability. Their integration into Quanterix’s platform will enhance multi-plex and multi-omic capabilities, ensuring greater control over critical components. EMISSION CEO Van Chandler expressed enthusiasm about the partnership, noting that their high-quality bead technology aligns with Quanterix’s vision to make advanced multi-plex assays accessible to all labs.

The acquisition involves an upfront cash payment of $10 million, with an additional $10 million contingent on the completion of technical milestones. EMISSION may also earn up to $50 million in performance-based payouts, expected to be funded through cash generated from meeting those milestones. Quanterix anticipates the deal will positively impact revenue and gross margins by 2026.

This strategic move reinforces Quanterix’s commitment to innovation in biomarker detection and diagnostics. By integrating EMISSION’s technology, the company strengthens its position in the multi-plex assay market while opening new revenue streams through OEM partnerships. With Simoa technology already setting industry standards for ultrasensitive biomarker detection, the acquisition marks a significant step toward broadening the reach of Quanterix’s tools and solutions.

Quanterix’s focus on neurology, oncology, immunology, and infectious disease research continues to fuel breakthroughs in disease understanding and management. With nearly two decades of experience, the company remains a trusted partner for researchers, boasting over 2,900 peer-reviewed publications featuring its technology. The integration of EMISSION’s beads is expected to enhance Quanterix’s ability to deliver precise, flexible solutions to researchers and clinicians worldwide, further cementing its leadership in the field.

Furthermore, the acquisition aligns with Quanterix’s strategy of vertical integration, which is increasingly critical in the competitive field of diagnostics. By bringing key components in-house, Quanterix not only enhances its technological control but also reduces dependence on external suppliers, paving the way for faster innovation cycles and cost efficiencies. This approach is expected to drive long-term growth and maintain the company’s edge in a rapidly evolving industry.

The addition of EMISSION’s proprietary bead technology also has implications for the broader scientific community. By targeting third-party OEM customers, Quanterix is fostering collaboration and expanding access to advanced diagnostic tools. This could accelerate the adoption of multi-plex assays across various laboratories and research institutions, driving progress in disease diagnostics and personalized medicine.

As the demand for high-sensitivity biomarker detection continues to grow, Quanterix’s ability to deliver scalable, high-quality solutions becomes increasingly vital. The integration of EMISSION’s technology not only reinforces Quanterix’s position as a market leader but also underscores its commitment to empowering scientists with cutting-edge tools to address complex healthcare challenges. With this acquisition, Quanterix is poised to play a pivotal role in shaping the future of diagnostics and research.

Release -MAIA Biotechnology Granted FDA Rare Pediatric Disease Designation for THIO as a Treatment for Pediatric High-Grade Gliomas

Posted on by aweinsoff@channelchek.com

Research News and Market Data on MAIA

December 16, 2024 8:37am EST Download as PDF

CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that the FDA has designated THIO for the treatment of pediatric-type diffuse high-grade gliomas (PDHGG) as a drug for a “rare pediatric disease.”

“THIO is a versatile anti-cancer agent that has demonstrated positive results in multiple difficult to treat cancer types, including pediatric high-grade glioma, which is among the most treatment-resistant cancers in children. THIO is shown to activate the immune system while evading tumor immunosuppression, a novel therapeutic approach for this devastating childhood disease,” said MAIA Chairman and Chief Executive Officer Vlad Vitoc, M.D. “We are proud to receive the FDA’s Rare Pediatric Disease designation for THIO, which significantly bolsters our plans for continuing research in the PDHGG indication.”

MAIA’s Vice President and Head of Regulatory and Quality K. Robinson Lewis added, “Rare pediatric disease designation also offers a highly valuable incentive for MAIA. Upon FDA approval of a future new drug application in PDHGG, MAIA would be eligible to receive a priority review voucher that can be redeemed or sold as an asset at a very high valuation.”

Rare pediatric disease priority review vouchers (PRVs) can be redeemed by drug developers for FDA priority review of a different product or transferred or sold to another sponsor. Since 2015, FDA priority review vouchers have sold as assets at an average amount of $100 million.1

Previous research showcased THIO’s potency as a treatment for a PDHGG subtype known as diffuse intrinsic pontine glioma (DIPG). A research collaboration between MAIA and Nationwide Children’s Hospital found that THIO combined with ionizing radiation (IR) resulted in significantly decreased cell proliferation and produced potent anticancer effects in highly aggressive DIPG. The data was presented in April 2024 at the American Association for Cancer Research (AACR) Annual Meeting.

MAIA collaborated with Only Orphans Cote for THIO’s designation request. Only Orphans Cote is a foremost provider of regulatory services and strategies for FDA orphan drug designations and marketing authorization.

In addition to its rare pediatric disease designation in PDHGG, THIO holds orphan drug designations (ODD) in three cancer types: hepatocellular carcinoma (HCC), small cell lung cancer (SCLC) and glioblastoma. MAIA believes that THIO is the only direct telomere-targeting agent currently in clinical development.

About THIO

THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.

About MAIA Biotechnology, Inc.

MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.

Forward Looking Statements

MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.

1 Pharmaceutical Technology, GlobalData Pharma Intelligence Centre, January 2024

Investor Relations Contact
+1 (872) 270-3518
ir@maiabiotech.com

Source: MAIA Biotechnology, Inc.

Released December 16, 2024

GeoVax Labs (GOVX) – GeoVax Receives Allowance For New Patent Covering Vaccine Platform Technologies

Posted on by cpereira@noblefcm.com


Tuesday, December 10, 2024

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation COVID-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades.

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

Refer to the full report for the price target, fundamental analysis, and rating.

Intellectual Property Estate Is Expanding. GeoVax announced that the US Patent and Trademark Office has issued a Notice of Allowance for its patent application covering the expression of antigens in virus-like particles delivered with a viral vector. This is a mechanism used to stimulate an immune response from the GeoVax MVA-based vaccines. The patent allowance is the final step in the USPTO application process, and the patent will be issued after payment of fees.

The Application Covers Expression Of Immune Stimulation Antigens. The patent titled “Vaccinia Viral Vectors Encoding Chimeric Virus Like Particles” includes claims for expressing a tumor associated antigen (TAA) in virus-like particles (VLPs) from a recombinant Modified Vaccinia Ankara (MVA) viral vector. This covers vaccines that use the MVA as a vector to deliver DNA that assembles into non-infectious virus-like constructs to simulate an immune response against the targeted virus. This mechanism is used in the MVA MUC-1 (cancer) and infectious disease vaccines. A detailed description of the MVA platform and VLPs begins on page 7 of our Initiation of Coverage report.


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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Release – Ocugen CEO to Present at NobleCon20 – Noble Capital Markets’ 20th Annual Emerging Growth Equity Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

November 27, 2024

PDF Version

MALVERN, Pa., Nov. 27, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen will present at NobleCon20 – Noble Capital Markets’ 20th Annual Emerging Growth Equity Conference at Florida Atlantic University, Executive Education Complex, in Boca Raton, FL—taking place December 3 – 4, 2024.

“Every year, NobleCon showcases emerging growth companies from a variety of industries to a relevant investor audience,” said Dr. Musunuri. “I look forward to sharing an update on our clinical programs for attendees who may not yet be familiar with the Ocugen story.”

Details regarding the presentation and moderated Q&A are as follows:

Date: Tuesday, December 3, 2024

Location: Presentation Room 3

Time: 1:30 p.m. ET  
 

In addition to Dr. Musunuri’s session, members of Ocugen’s executive team will conduct one-on-one meetings with investors to highlight the Company’s business and clinical development strategy across its unique modifier gene therapy platform.
 

A high-definition video webcast of the presentation will be available the following day in the Events section of the Company’s website, and as part of a complete catalog of presentations available at Noble Capital Markets’ Conference website and on Channelchek the investor portal created by Noble. The webcast will be archived on the Company’s website, the NobleCon website, and Channelchek.com for 90 days following the event. 
 

About Ocugen, Inc. 
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
 

About Noble Capital Markets, Inc. 
Established in 1984, Noble Capital Markets is an SEC / FINRA registered full-service investment bank and advisory firm with an award-winning research team and proprietary investor distribution platform. We deliver middle market expertise to entrepreneurs, corporations, financial sponsors, and investors. Over the past 40 years, Noble has raised billions of dollars for companies and published more than 45,000 equity research reports. Noble launched www.channelchek.com in 2018 – an investor community dedicated exclusively to public emerging growth and their industries. Channelchek is the first service to offer institutional-quality research to the public, for FREE at every level without a subscription. More than 7,000 public emerging growth companies are listed on the site, and content including equity research, webcasts, and industry articles.
 

Cautionary Note on Forward-Looking Statements 
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com

Release – Zomedica to Present at the NobleCon20 Twentieth Annual Emerging Growth Equity Conference December 4, 2024

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZOM

ANN ARBOR, MI / ACCESSWIRE / November 27, 2024 / Zomedica Corp. (NYSE American:ZOM) (“Zomedica” or the “Company”), a veterinary health company offering point-of-care diagnostics and therapeutic products for equine and companion animals, today announced that its Chief Executive Officer, Larry Heaton, will present and host one-on-one meetings with investors at NobleCon20 – Noble Capital Markets’ Twentieth Annual Emerging Growth Equity Conference at Florida Atlantic University, Executive Education Complex, in Boca Raton, Florida on Wednesday, December 4, 2025 at 11:00 am Eastern Standard Time.

A high-definition video webcast of the presentation will be available the following day on the Company’s website, www.zomedica.com, and as part of a complete catalog of presentations available at Noble Capital Markets’ Conference website, www.nobleconference.com, and on the investor portal created by Noble, Channelchek at www.channelchek.com. The webcast will be archived on the company’s website, at https://investors.zomedica.com/, and the NobleCon and Channelchek websites for 90 days following the event.

About Zomedica

Zomedica is a leading equine and companion animal healthcare company dedicated to improving animal health by providing veterinarians innovative therapeutic and diagnostic solutions. Our gold standard PulseVet® shock wave system, which accelerates healing in musculoskeletal conditions, has transformed veterinary therapeutics. Our suite of products also includes the Assisi® Loop line of therapeutic devices and the TRUFORMA® diagnostic platform, the TRUVIEW® digital cytology system, and the VetGuardian® no-touch monitoring system, all designed to empower veterinarians to provide top-tier care. In the aggregate, their total addressable market in the U.S. exceeds $2 billion. Headquartered in Michigan, Zomedica employs approximately 150 people and manufactures and distributes its products from its world-class facilities in Georgia and Minnesota. An NYSE American company, Zomedica grew revenue 33% in 2023 to $25 million and maintains a strong balance sheet with approximately $78 million in liquidity as of September 30, 2024. Zomedica is advancing its product offerings, leveraging strategic acquisitions, and expanding internationally as we work to enhance the quality of care for pets, increase pet parent satisfaction, and improve the workflow, cash flow and profitability of veterinary practices. For more information visit www.zomedica.com.

Follow Zomedica

About Noble Capital Markets, Inc.

Established in 1984, Noble Capital Markets is an SEC / FINRA registered full-service investment bank and advisory firm with an award-winning research team and proprietary investor distribution platform. We deliver middle market expertise to entrepreneurs, corporations, financial sponsors, and investors. Over the past 40 years, Noble has raised billions of dollars for companies and published more than 45,000 equity research reports. Noble launched www.channelchek.com in 2018 – an investor community dedicated exclusively to public emerging growth and their industries. Channelchek is the first service to offer institutional-quality research to the public, for FREE at every level without a subscription. More than 7,000 public emerging growth companies are listed on the site, and content including equity research, webcasts, and industry articles.

Investor Relations Contact:

Zomedica Investor Relations
investors@zomedica.com
1-734-369-2555

SOURCE: Zomedica Corp.

MustGrow Biologics Corp. (MGROF) – Sales are Trickling In

Posted on by cpereira@noblefcm.com


Wednesday, November 27, 2024

Joe Gomes, CFA, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.

Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

3Q Results. Revenue for the quarter totaled CAD$279,182, including the Company’s CAD$272,500 in deferred revenue. Excluding this, revenue was CAD$6,682, including TerraSante sales’ impact. We estimated revenue of CAD$15,000. Net loss was CAD$1.7 million, or a loss of $0.03/sh, compared to a loss of CAD$1.9 million last year, or  $0.04/sh. We estimated a net loss of CAD$1.9 million or $0.04/sh.

Sales Revenue is Here. Notably, the quarter recognized product sales revenue for the TerraSante product for the first time. We expect TerraSante revenue to begin to impact revenue in a meaningful way in the latter half of 2025. As the Company receives more approvals from different states, we believe the opportunity to showcase TerraSante to farmers can expand, along with revenue.


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Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Traws Pharma (TRAW) – Antiviral Pipeline Makes Progress and Moves Forward

Posted on by cpereira@noblefcm.com


Wednesday, November 27, 2024

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in a combination trial with estrogen blockade in advanced endometrial cancer. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also evaluating opportunities for combination studies with narazaciclib in additional indications. Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies. These studies include a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma.

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

Refer to the full report for the price target, fundamental analysis, and rating.

Traws Has Made Significant Progress During 2024. In May 2024, Onconova and Transfynydd combined to form Traws Pharmaceuticals. The new company brought together the Transfynydd small molecules for respiratory viral diseases with the Onconova clinical-stage oncology pipeline. The company is planning Phase 2 clinical trials for its antivirals while looking for collaborations and/or partnerships for the oncology pipeline.

Phase 2 Trials Are Planned For Two Antivirals. Tivoxavir marboxil (TRX100) is a protease inhibitor of enzyme needed for the reproduction of the virus that causes seasonal and pandemic influenza. Ratutrelvir (TRX01) inhibits the protease Mpro (the main protease or 3CL), the main protease of SAR-CoV-2 (the COVID-19 virus), but does not require co-administration of a metabolic inhibitor. This avoids the risk of drug-drug interactions and potential side effects. Phase 2 studies for both drugs are expected to begin in 1H24.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Release – Tonix Pharmaceuticals to Participate in the NobleCon20 Investor Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

November 26, 2024 7:00am EST

CHATHAM, N.J., Nov. 26, 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, announced today that Jessica Morris, Chief Operating Officer of Tonix Pharmaceuticals, will present and conduct investor meetings at NobleCon20, Noble Capital Markets’ Twentieth Annual Emerging Growth Equity Conference, being held December 3-4 in Boca Raton, Fla.

Investors interested in arranging a meeting with the Company’s management during the conference should contact the NobleCon conference coordinator. A video webcast of the presentation will be available the day following the presentation under the IR Events tab of the Tonix website at www.tonixpharma.com and as part of a complete catalog of presentations available at Noble Capital Markets’ Conference website at www.nobleconference.com and on Channelchek, the investor portal created by Noble Capital Markets, at www.channelchek.com.  

Details of the Tonix Pharmaceuticals Presentation

Event:NobleCon20, Noble Capital Markets’ Twentieth Annual Emerging Growth Equity Conference
  
Date:Tuesday, December 3, 2024
  
Time:12:00 p.m. ET
  
Location:Florida Atlantic University, College of Business Executive Education (COBEE) Complex
  
Track:Presentation Room 3
  

Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully-integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia, for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. We expect an FDA decision on the acceptance of the NDA for review and a PDUFA date in December and if accepted, a decision on NDA approval in 2025. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institute of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified 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 a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years 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. 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; their efficacy and safety have not been established 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 Healthcare
peter.vozzo@icrhealthcare.com
(443) 213-0505

Media Contact

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

Primary Logo

Source: Tonix Pharmaceuticals Holding Corp.

Released November 26, 2024

How Trump’s DOGE Initiative Could Reshape Biotech’s Future

Posted on by slightbourne@noblecapitalmarkets.com

The Department of Government Efficiency (DOGE), spearheaded by Donald Trump with the involvement of Elon Musk and Vivek Ramaswamy, promises sweeping changes to federal operations, including deregulation efforts targeting agencies like the FDA. For the biotech industry, these potential reforms could significantly impact drug development, approval processes, and market dynamics.

Faster Approvals: A Biotech Catalyst

The FDA’s rigorous drug approval process ensures safety and efficacy but often takes years and billions of dollars to navigate. By streamlining these regulations, DOGE could shorten timelines for bringing therapies to market. This shift would lower barriers for smaller biotech firms, enhance their ability to innovate, and reduce costs for large pharmaceutical companies.

For investors, expedited approvals would translate to quicker revenue streams for new drugs, driving valuations and fueling confidence in the sector. This environment could spur a rally in biotech stocks, particularly for companies with promising pipelines awaiting FDA clearance.

Risks of Deregulation

However, deregulation comes with significant challenges. The FDA exists to ensure public safety, and relaxing oversight could lead to unintended consequences. If subpar drugs enter the market, public trust in the industry might erode, and companies could face litigation or reputational damage. These risks could temper investor enthusiasm, particularly if deregulation is perceived as compromising safety.

Additionally, deregulation might benefit well-established players disproportionately. Large pharmaceutical firms with the resources to navigate even a reduced regulatory framework may thrive, while smaller biotech startups could struggle to compete despite faster approvals.

Investor Implications

DOGE’s initiatives might catalyze investment in biotechnology, particularly in areas like gene therapy, oncology, and rare diseases. Sectors with high unmet needs and long development cycles stand to gain the most from expedited approvals. Furthermore, the promise of reduced R&D costs could attract private equity and venture capital to early-stage biotech firms.

In the stock market, the potential for reform could trigger bullish sentiment in biotech ETFs and sector-specific indices. Companies with late-stage trials or pending FDA decisions might see the greatest benefit. However, volatility is likely as the industry adapts to new regulations, and investors should be cautious of overvaluations driven by speculative enthusiasm.

Industry Innovators at the Forefront

As these potential regulatory shifts emerge, a select group of biotech pioneers are positioning themselves to capitalize on the DOGE initiative’s promising landscape. At the upcoming NobleCon conference, a plethora of healthcare companies will showcase their innovative approaches and strategic vision, offering investors and industry observers a glimpse into how they might leverage potential FDA streamlining and accelerated approval processes. These organizations represent the cutting edge of biotech innovation, each poised to potentially benefit from the proposed regulatory reforms.

Broader Impacts on Healthcare

Beyond the biotech sector, DOGE’s focus on efficiency could reshape the broader healthcare landscape. Cheaper and faster drug approvals might improve patient access to innovative treatments. Yet, balancing speed with safety will be critical to achieving long-term success.

The initiative also highlights the growing intersection of politics and business, with leaders like Musk and Ramaswamy advocating for entrepreneurial approaches to governance. Whether DOGE delivers on its promises remains to be seen, but its potential to disrupt traditional industries, including biotechnology, is undeniable.

DOGE represents both opportunity and risk for biotech investors. If executed thoughtfully, its reforms could unlock unprecedented growth in the sector by fostering innovation and reducing costs. However, ensuring safety and maintaining public trust will be critical to its long-term success. Investors should monitor regulatory developments closely, as the outcomes of these reforms will shape the biotech landscape for years to come.

The biotech rally might already be on the horizon—if DOGE achieves its ambitious goals, this sector could see transformative growth.

Release – Ocugen Announces European Medicines Agency Grants Orphan Medicinal Product Designation for Modifier Gene Therapy Candidate OCU410ST for Treatment of ABCA4-Associated Retinopathies including Stargardt Disease

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

November 20, 2024

MALVERN, Pa., Nov. 20, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the European Medicines Agency (EMA) has granted orphan medicinal product designation for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3).

“We are deeply honored to receive orphan medicinal product designation from the EMA for OCU410ST. This recognition brings us one step closer to providing a much-needed option for Stargardt patients who currently have no therapies available,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen. “We are committed to advancing this treatment with urgency and dedication, with the hope of making a meaningful impact on the lives of those affected by this challenging disease.”  

The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation to OCU410ST in April 2023. Stargardt disease affects approximately 100,000 people in the U.S. and Europe combined.

Orphan medicinal product designation in Europe offers certain benefits to drug developers while they develop drugs intended for safe and effective treatment, diagnosis, or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Benefits include protocol assistance, reduced regulatory fees, research grants, and 10 years of market exclusivity following regulatory approval.

Dosing in the first phase of the Phase 1/2 OCU410ST GARDian trial for Stargardt disease is complete and the Data and Safety Monitoring Board (DSMB) has recommended moving forward with Phase 2. To date, the safety and tolerability profile of OCU410ST appears to be very favorable.

Preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410ST GARDian clinical trial was recently presented at Ocugen’s Clinical Showcase. Data from evaluable subjects at six months demonstrated a remarkable 84% reduction in atrophic lesion growth in treated eyes versus untreated fellow eyes.

“We are encouraged by the preliminary efficacy data showing stabilization or improvement in visual function and retinal structure outcomes in OCU410ST treated eyes,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “These positive clinical and regulatory milestones continue to support the potential for OCU410ST to address inherited retinal diseases with a one-time therapy for life.”

OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and further represents the impact of Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks.

Ocugen intends to pursue an accelerated marketing authorization application (MAA) for OCU410ST.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com

Ocugen (OCGN) – Interim Data From The Clinical Showcase Highlighted

Posted on by cpereira@noblefcm.com


Wednesday, November 20, 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.

Refer to the full report for the price target, fundamental analysis, and rating.

Summary Of Data Announced For Products In Clinical Trials. Ocugen summarized some of the major points presented at its Clinical Showcase on November 12. As discussed in our Research Note on November 13, speakers reviewed the products’ mechanisms of action, reviewed OCU400 data from the Phase 1/2 trial, the design of the OCU400 Phase 3 liMeliGhT trial, and presented new interim data from the Phase 1/2 ArMaDa trial for OCU410.

Mechanism of Action For The Modifier Gene Therapy (MGT) Detailed. Ocugen is developing gene therapies for retinal diseases that deliver a regulatory gene to control other genes and regulate downstream pathways. Products in development are for inherited diseases that result from multiple gene mutations or conditions that result from several dysfunctional pathways.


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