Category: Health

Release – Tonix Pharmaceuticals Reports Third Quarter 2023 Financial Results and Operational Highlights

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

November 09, 2023 4:15pm EST

Topline Results from Phase 3 Potentially NDA-Enabling Study of TNX-102 SL in Fibromyalgia Expected Late December 2023: Centrally-Acting Non-Opioid Analgesic

Topline Results from Phase 2 Proof-of-Concept Study of TNX-1900 in Chronic Migraine Expected Early December 2023: Intranasal Potentiated Oxytocin

Meaningful Progress Made in Obtaining External Support for Clinical Trials from U.S. Government Agencies and Other Institutions

Revenue from Marketed Acute Migraine Products: Zembrace® SymTouch® (sumatriptan injection) and Tosymra® (sumatriptan nasal spray) Included in Third Quarter Financial Statements

CHATHAM, N.J., Nov. 09, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced financial results for the third quarter ended September 30, 2023, and provided an overview of recent operational highlights.

“Tonix expects topline results from its Phase 3 fibromyalgia study and Phase 2 chronic migraine study before year end,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “The Phase 3 RESILIENT trial in fibromyalgia, if successful, is expected to be the final efficacy trial required for submitting a New Drug Application (NDA) for approval by the U.S. Food and Drug Administration (FDA) for TNX-102 SL (cyclobenzaprine HCl sublingual tablets). In the Phase 2 proof-of-concept PREVENTION study in chronic migraine of TNX-1900 (intranasal potentiated oxytocin), all patients have completed their final visit and topline results are expected in early December 2023.”

Dr. Lederman continued, “We are continuing to shift the expense of clinical trials from our operating budget to U.S. government agencies and other institutions through partnerships. The U.S. Department of Defense (DoD) is supporting the upcoming Phase 2 study of TNX-102 SL in acute stress disorder, being conducted and sponsored by University of North Carolina (UNC). The U.S. National Institutes of Health (NIH) and National Institute of Allergy and Infectious Diseases (NIAID), through its Project NextGen, will conduct the Phase 1 study of our vaccine candidate TNX-1800 (modified recombinant horsepox virus, live vaccine). The National Institute of Drug Abuse (NIDA) is supporting our Phase 2 study of TNX-1300 (recombinant double mutant cocaine esterase) for cocaine intoxication. Massachusetts General Hospital (MGH) is conducting Phase 2 studies of TNX-1900 in binge eating disorder and pediatric obesity, and the University of Washington is conducting a Phase 2 study of TNX-1900 in social anxiety disorder. Finally, we continue to collaborate with MGH on several preclinical non-human primate studies for TNX-1500 (anti-CD40L Fc-modified humanized monoclonal antibody), currently in a Phase 1 study being conducted by Tonix. These outside collaborations leverage our internal resources and allow us to progress our clinical programs in a capital efficient manner.”

Partnerships with External Funding – Recent Highlights

  • NIH/NIAID selected Tonix’s vaccine candidate, TNX-1800, as part of Project NextGen; a Phase 1 study is expected to start in the second half of 2024. NIH/NIAID will cover the full cost of the clinical trial, while Tonix will supply the vaccine candidate.
  • NIDA is supporting a Phase 2 study on TNX-1300 for cocaine Intoxication; expected to start enrolling patients in the fourth quarter of 2023.
  • DoD is supporting a Phase 2 investigator-initiated study of TNX-102 SL for acute stress disorder at UNC for motor vehicle accident victims; expected to start enrolling patients in 2024.
  • MGH/Harvard Medical School is conducting Phase 2 investigator-initiated studies of TNX-1900 in binge eating disorder and adolescent obesity.
  • The University of Washington is conducting a Phase 2 investigator-initiated study of TNX-1900 in social anxiety disorder.

Marketed Products – Recent Highlights

  • In September 2023, Tonix announced that it is committed to meeting potential increased demand for Tosymra® (sumatriptan nasal spray) 10 mg after GlaxoSmithKline’s planned discontinuation of Imitrex® (sumatriptan) nasal spray 5 mg and 20 mg products after January 2024. Tonix is preparing for potential increased demand for Tosymra to help avoid possible drug shortages for patients who suffer from migraines. Tosymra nasal spray is approved on the basis of bioequivalence to Imitrex injection 4 mg.
  • Tonix completed the acquisition of Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra from Upsher-Smith Laboratories, LLC in June 2023. Both products are indicated for the treatment of acute migraine with or without aura in adults.

Key Product Candidates* — Recent Highlights

Central Nervous System (CNS) Pipeline

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

  • The Company announced in August 2023 that it completed enrollment of its potentially confirmatory Phase 3 RESILIENT trial of TNX-102 SL 5.6 mg in FM. 457 participants were randomized in the trial, which, if successful, is expected to serve as the final, well-controlled efficacy trial required for submission of NDA for approval by the FDA. RESILIENT is a registration-quality, double-blind, placebo-controlled study. Topline results from the RESILIENT trial are expected in late December of 2023.

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

  • In September 2023, the Company announced that the UNC Institute for Trauma Recovery has been awarded a $3 million grant from the DoD to investigate the potential of Tonix’s TNX-102 SL to reduce the frequency and severity of adverse effects of acute trauma, which include ASR and ASD, and development of PTSD. The proposed Phase 2, Optimizing Acute Stress Reaction Interventions with TNX-102 SL (OASIS) study will examine the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients presenting to the emergency department (ED) after a motor vehicle collision (MVC). The study will enroll approximately 180 MVC trauma survivors at ED study sites in the U.S. Participants will be randomized in the ED to receive a two-week course of either TNX-102 SL or placebo.
  • Initiation of patient enrollment in the proposed investigator-sponsored OASIS study is anticipated in the beginning of 2024, subject to clearance by the FDA of an investigator-initiated Investigational New Drug (IND) application.

TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)

  • In September 2023, the Company announced topline results from its Phase 2 PREVAIL proof-of-concept study of TNX-102 SL for fibromyalgia-type Long COVID. TNX-102 SL showed a robust Cohen’s d effect size of 0.5 in improving fatigue relative to placebo; and it showed consistent activity across secondary measures of sleep quality, cognitive function, disability and Patient Global Impression of Change, but did not meet the primary endpoint of multi-site pain reduction at week 14. TNX-102 SL was generally well tolerated and no new safety signals were observed.
  • The Company intends to request an End-of-Phase 2 meeting with the FDA to discuss a potential Phase 3 program based on a proposed primary outcome measure using the PROMIS Fatigue scale. The meeting is expected to take place in the first quarter of 2024.

TNX-1900 (intranasal potentiated oxytocin): small peptide for migraine, craniofacial pain, social anxiety disorder (SAD), insulin resistance and related disorders, and adolescent obesity and binge eating disorder

  • In October 2023, the Company announced it completed the clinical phase of the PREVENTION study, a Phase 2 proof-of-concept study of TNX-1900 for the prevention of migraine headaches in chronic migraineurs, as the last of 88 enrolled patients completed their final study visit. PREVENTION is a registration-quality, double-blind, placebo-controlled study.
  • Topline results from the PREVENTION Phase 2 trial are expected in early December 2023.
  • In September 2023, the Company announced that David C. Yeomans, Ph.D. presented data relevant to the proposed mechanism of TNX-1900 in treating chronic migraine in a poster and an oral presentation at the 2023 International Headache Congress (IHC) in Seoul, South Korea. The poster and oral presentation titled, “Human trigeminal ganglia possess oxytocin receptors on CGRP positive neurons: expression increased by inflammation,” include research sponsored by and licensed to Tonix. The presentations show that oxytocin receptors are co-expressed with calcitonin gene-related peptide (CGRP) on human trigeminal ganglia neurons, which is similar to Professor Yeomans’ previous findings in animal trigeminal ganglia. The inflammatory cytokine IL-6 upregulated expression of oxytocin receptors on human trigeminal neurons, consistent with the previously observed impact of inflammation on the potency of oxytocin on its receptor. In animals, oxytocin has been shown to functionally inhibit the excitability of trigeminal neurons, which is consistent with oxytocin inhibiting the release of CGRP at trigeminal nerve terminals.1
  • Tonix announced in July 2023 that the first participant was enrolled in the investigator-initiated Phase 2 STROBE Study of TNX-1900 for the treatment of binge-eating disorder at MGH. Tonix is supporting the STROBE study through a clinical trial agreement with MGH.
  • Tonix announced in July 2023 that the first participant was enrolled in a Phase 2 investigator-initiated, proof-of-concept study of TNX-1900 for enhancing social safety learning in SADSAD. Tonix entered into an agreement with the University of Washington to examine the potential role of TNX-1900 in enhancing vicarious extinction learning in SAD, compared to healthy controls.
  • Tonix announced in July 2023 that the first participant was enrolled in the Phase 2 POWER study of TNX-1900 for the treatment of pediatric obesity with MGH. MGH is the sponsor of the NIH-funded trial, being conducted under an investigator-initiated IND.

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 the fourth quarter of 2023. In 2022, Tonix was awarded a Cooperative Agreement grant from NIDA, part of the NIH, to support development of TNX-1300.
  • TNX-1300 has been granted Breakthrough Therapy designation by the FDA.

Rare Disease Pipeline

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

  • In October 2023, Herbert Harris, M.D., Ph.D., Executive Vice President, Translational Medicine of Tonix Pharmaceuticals, provided an overview of Tonix’s TNX-2900 program at the Foundation for Prader-Willi Research (FPWR) Family Conference in Denver, CO. The presentation highlights preclinical data showing the enhancing effects of magnesium (Mg2+) on the activation of oxytocin receptors. The Mg2+ enhanced formulation of intranasal oxytocin is the basis for TNX-2900, in development to treat hyperphagia, or pathological over-eating, in children and adolescents with PWS. In preclinical studies, Mg2+ increases the potency of oxytocin, which is a peptide hormone that reduces appetite and signals fullness, potentially improving receptor binding and resulting in improved therapeutic action.

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.

  • In October 2023, the Company announced data from two oral presentations which were delivered recently at the American College of Surgeons (ACS) Clinical Congress 2023, and The International Pancreas and Islet Transplant Association (IPITA), the International Xenotransplantation Association (IXA), and the Cell Transplant and Regenerative Medicine Society (CTRMS) Joint Congress by faculty at the Center for Transplantation Sciences, MGH. The oral presentations titled, “Pilot Evaluation of a Clinical Xeno Heart Transplant Regimen in a Preclinical Model” and “Extended Survival of 9- and 10-Gene Edited Pig Heart Xenografts with Ischemia Minimization and CD154 Costimulation Blockade-Based Immunosuppression” by Dr. Ikechukwu Ileka et al. include data demonstrating the use of TNX-1500 as maintenance therapy after xeno heart transplant in non-human primates. In both studies, genetically engineered (GE) pigs in baboon transplants were treated with cold-perfused ischemia minimization and a novel costimulation-based immunosuppressive regimen that includes TNX-1500.
  • In October 2023, Tonix announced that a study published in the Journal Nature2 by faculty at the Center for Transplantation Sciences, MGH in collaboration with biotechnology company, eGenesis, utilized TNX-1500 as part of the immune modulating regimen to prevent organ transplant rejection. The Nature article titled, “Design and testing of a humanized porcine donor for xenotransplantation” includes data that provide additional support for TNX-1500’s activity in preventing pig xenograft organ rejection and for its safety and tolerability in non-human primates.
  • In August 2023, Tonix announced the initiation of a 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.
  • The first indication for TNX-1500 will be prophylaxis of organ rejection in adult patients receiving a kidney transplant, but multiple additional indications are possible, including autoimmune diseases. Two peer reviewed publications described the work at the MGH on allogeneic transplants in animals were published.3,4

Infectious Disease Pipeline

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 November 2023, Tonix announced that NIAID, a part of the NIH, will conduct a Phase 1 clinical trial with TNX-1800 as part of Project NextGen. The Phase 1 trial of TNX-1800 is expected to start in the second half of 2024. NIAID will cover the full cost of the clinical trial, including operations and related analyses. Tonix will be responsible for providing clinical trial materials, and upon completion will have the right to rely on the findings in regulatory filings with the FDA to support the approval of its COVID-19 vaccine and other vaccines based on the RPV platform.

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

  • In August 2023, Tonix received the official written response from a Type B pre-IND meeting with the FDA to develop TNX-801 as a potential vaccine to protect against mpox disease (formerly known as monkeypox) and smallpox. Tonix believes the FDA feedback provides a path to agreement on the design of a Phase 1/2 study and the overall clinical development plan. The Phase 1/2 clinical trial will assess the safety, tolerability, and immunogenicity of TNX-801, following the submission and clearance of an IND.

*All of Tonix’s product candidates are investigational new drugs or biologics and none have been approved for any indication.

1Tzabazis A, et al. Cephalalgia. 2016. 36(10):943-50.

2Anand R.P., et al. Nature. 2023. 622, 393–401.

3Lassiter, G., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate renal allograft survival. American Journal of Transplantation. https://doi.org/10.1016/j.ajt.2023.03.022

4Miura, S., et al. (2023). TNX-1500, a crystallizable fragment–modified anti-CD154 antibody, prolongs non-human primate cardiac allograft survival. American Journal of Transplantation. https://doi.org/10.1016/j.ajt.2023.03.025

      Recent Highlights—Financial

As of September 30, 2023, Tonix had approximately $6.9 million of cash and cash equivalents, compared to $120.2 million as of December 31, 2022. Additionally, Tonix had inventory totaling approximately $13.3 million as of September 30, 2023. In August 2023, Tonix received net proceeds of approximately $6.3 million through a public offering of common stock, after deducting underwriting discount and other offering expenses. Cash used in operations was approximately $79.7 million for the nine months ended September 30, 2023, compared to $75.8 million for the same period in 2022. Cash used by investing activities for the nine months ended September 30, 2023 was approximately $28.6 million.

On September 28, 2023, the Company sold 4,050,000 shares of common stock, pre-funded warrants to purchase up to 4,950,000 shares of common stock, and accompanying common A warrants to purchase 9,000,000 shares of common stock and common B warrants to purchase up to 9,000,000 shares of common stock in a public offering for net proceeds of approximately $4.0 million, after deducting underwriting discount and other offering expenses. This public offering closed on October 3, 2023.

Third Quarter 2023 Financial Results

Net product revenue for the third quarter 2023 was approximately $4.0 million. As a reminder, Tonix completed the acquisition of two currently marketed products from Upsher-Smith Laboratories, LLC on June 30, 2023.

During the three months ended September 30, 2023, Tonix received $0.4 million from NIDA for the TNX-1300 study to treat cocaine intoxication.

R&D expenses for the third quarter 2023 were approximately $21.1 million, compared to $22.2 million for the same period in 2022. This decrease is predominantly due to decreased non-clinical and manufacturing expenses, offset by an increase in clinical, employee-related and professional expenses.

SG&A expenses for the third quarter 2023 were $8.7 million, compared to $7.4 million for the same period in 2022. The increase was primarily due to sales and marketing associated with the Company’s recently acquired marketed products.

Net loss available to common stockholders was $28.0 million, or $1.83 per share, basic and diluted, for the third quarter 2023, compared to net loss available to common stockholders of $29.0 million, or $4.24 per share, basic and diluted, for the same period in 2022. The basic and diluted weighted average common shares outstanding for the third quarter 2023 was 15,327,558 compared to 6,843,099 shares for the same period in 2022.

Tonix Pharmaceuticals Holding Corp.*

Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed enrollment of a potentially confirmatory Phase 3 study in the third quarter of 2023, with topline data expected in late December 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition, and topline results were reported in the third quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), is in development as a preventive treatment in chronic migraine, and enrollment has completed in a Phase 2 proof-of-concept study with topline data expected in early December 2023. TNX-1900 is also being studied in binge eating disorder, pediatric obesity and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the fourth quarter of 2023. Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes 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. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases, including TNX-1800, in development as a vaccine to protect against COVID-19. During the fourth quarter of 2023, TNX-1800 was selected by the U.S. National Institutes of Health (NIH), National Institute of Allergy and Infectious Diseases (NIAID) Project NextGen for inclusion in Phase 1 clinical trials. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.

*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. Intravail is a registered trademark of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc. 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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.

 TONIX PHARMACEUTICALS HOLDING CORP.   
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS  
(In Thousands, Except Share and Per Share Amounts)  
(unaudited)
              
   Three Months Ended
September 30,		  Nine Months Ended
September 30,		 
   2023  2022  2023  2022 
REVENUE:                 
Product revenue, net  $3,989  $—  $3,989  $— 
                  
COSTS AND EXPENSES:                 
Cost of revenue

  $2,374  $—  $2,374  $— 
Research and development   21,050   22,201   69,537   57,202 
Selling, general and administrative   8,712   7,390   23,129   22,161 
    32,136   29,591   95,040   79,363 
                  
Operating loss   (28,147)  (29,591)  (91,051)  (79,363)
                  
Interest income, net   172   610   1,715   825 
                  
Net loss   (27,975)  (28,981)  (89,336)  (78,538)
                  
Preferred stock deemed dividend   —   —   —   4,255 
                  
Net loss available to common stockholders  $(27,975) $(28,981) $(89,336) $(82,793)
                  
Net loss per common share, basic and diluted  $(1.83) $(4.24) $(7.40) $(18.58)
                  
Weighted average common shares outstanding, basic and diluted   15,327,558   6,843,099   12,079,583   4,455,943 

See the accompanying notes to the condensed consolidated financial statements

TONIX PHARMACEUTICALS HOLDING CORP.
CONDENSED CONSOLIDATED BALANCE SHEETS
(In Thousands)
(Unaudited)
 
 September 30, 2023 December 31, 20221
Assets  
Cash and cash equivalents$6,914  $120,229 
Inventory 13,317   – 
Receivables, net 1,562   – 
Prepaid expenses and other 9,544   10,548 
Total current assets 31,337   130,777 
Other non-current assets 107,945   94,913 
Total assets$139,282  $225,690 
   
Liabilities and stockholders’ equity  
Total liabilities$18,449  $18,508 
Stockholders’ equity 120,833   207,182 
Total liabilities and stockholders’ equity$139,282  $225,690 


1The condensed consolidated balance sheet for the year ended December 31, 2022 has been derived from the audited financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial 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

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
(919) 360-3039

Zembrace® SymTouch® (sumatriptan Injection): IMPORTANT SAFETY INFORMATION

Zembrace SymTouch (Zembrace) 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 is 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 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, dihydroergotamine.
  • 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

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

Zembrace 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 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, 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 include: pain and redness at injection site; 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.

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. For more information, ask your provider.

This is the most important information to know about Zembrace but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit www.upsher-smith.com or call 1-888-650-3789.

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

INDICATION AND USAGE

Zembrace is a prescription medicine used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine.

Zembrace is not used to prevent migraines. It is not known if it is safe and effective in children under 18 years of age.

Tosymra® (sumatriptan nasal spray): IMPORTANT SAFETY INFORMATION

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

Tosymra is 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 is done and shows no problem.

Do not use 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
  • severe liver problems
  • hemiplegic or basilar migraines. If you are not sure if you have these, ask your healthcare provider.
  • had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
  • taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider if you are not sure if your medicine is listed above.
  • 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 ingredient in Tosymra

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

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.

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 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 Tosymra include: tingling, dizziness, feeling warm or hot, burning feeling, feeling of heaviness, feeling of pressure, flushing, feeling of tightness, numbness, application site (nasal) reactions, abnormal taste, and throat irritation.

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 Tosymra. For more information, ask your provider.

This is the most important information to know about 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 www.upsher-smith.com or call 1-888-650-3789.

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

INDICATION AND USAGE
Tosymra is a prescription medicine used to treat acute migraine headaches with or without aura in adults.

Tosymra is not used to treat other types of headaches such as hemiplegic or basilar migraines or cluster headaches.

Tosymra is not used to prevent migraines. It is not known if Tosymra is safe and effective in children under 18 years of age.

Source: Tonix Pharmaceuticals Holding Corp.

Released November 9, 2023

Release – Eledon Pharmaceuticals Reports Third Quarter 2023 Operating and Financial Results

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ELDN

November 9, 2023

Reported updated data from ongoing Phase 1b trial further supporting the potential of tegoprubart as a novel kidney transplant immunosuppressive therapy to prevent rejection and better preserve organ function

First participant dosed in Phase 2 BESTOW trial evaluating tegoprubart for the prevention of rejection in kidney transplantation

IRVINE, Calif., Nov. 09, 2023 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today reported its third quarter operating and financial results and reviewed recent business highlights.

“We were thrilled recently to report updated results from our ongoing Phase 1b study that continue to validate tegoprubart’s potential as an immunosuppressive agent that can prevent the rejection of transplanted kidneys,” said David-Alexandre C. Gros, M.D., Chief Executive Officer. “Tegoprubart demonstrated not only the potential to preserve, but also to improve graft function in comparison to the current standard of care treatment. Additionally, during the quarter we initiated our Phase 2 BESTOW trial and had the historic opportunity to support the second ever transplant of a genetically modified heart from a pig to a human. We continue to make significant progress toward our mission of bringing a much-needed, new treatment option to the growing number of patients undergoing kidney transplantation,” Dr. Gros continued.

Recent Corporate Developments

  • Reported data from the ongoing Phase 1b open-label trial evaluating tegoprubart for the prevention of rejection in patients undergoing kidney transplantation at the American Society of Nephrology Kidney Week 2023 Annual Meeting that took place in Philadelphia, PA from November 2-5, 2023. Data from 11 participants demonstrated that tegoprubart successfully prevented kidney transplant rejection and was generally safe and well-tolerated. Aggregate mean eGFR was above 70 mL/min/1.73m2 at all reported time points after day 90 supporting tegoprubart’s potential to protect organ function in patients undergoing kidney transplantation.
  • Announced that tegoprubart was used as a cornerstone component of the chronic immunosuppressive regimen administered following the second-ever transplant of a genetically modified heart from a pig to a human. The procedure was completed on September 20th at University of Maryland Medical Center on a 58-year-old male suffering from heart failure.
  • Dosed the first participant in the Phase 2 BESTOW trial evaluating tegoprubart for the prevention of organ rejection in patients receiving a kidney transplant.
  • Enrolled the first participant in the Phase 2 open-label extension (OLE) study which will evaluate the long-term safety, pharmacokinetics, and efficacy of tegoprubart in participants who have completed one year of treatment in the ongoing Phase 1b study, or the Phase 2 BESTOW study.
  • Announced the publication of results from a study evaluating tegoprubart as an immunomodulatory monotherapy in nonhuman primate kidney and islet allotransplants in Science Translational Medicine. Results from the study showed that treatment with tegoprubart as a monotherapy promoted long-term kidney and islet allograft survival and function in nonhuman primates, indicating its potential as an immunomodulatory agent for organ transplantation.
  • Strengthened leadership team with appointment of Eliezer Katz, M.D., FACS as Chief Medical Officer.
  • Appointed industry veteran James Robinson and renowned transplant surgeon Allan Kirk, M.D., Ph.D., to its Board of Directors.

Upcoming Anticipated 2024 Milestones

  • First Half 2024: Report updated interim clinical data from the ongoing Phase 1b trial of tegoprubart in kidney transplantation.
  • End of 2024: Complete enrollment in the Phase 2 BESTOW trial of tegoprubart in kidney transplantation.

Third Quarter Financial Results

The company reported a net loss of $10.3 million, or $0.35 per share, for the three months ended September 30, 2023, compared to a net loss of $10.5 million, or $0.73 per share, for the same period in 2022.

Research and development expenses were $7.9 million for the three months ended September 30, 2023, compared to $7.5 million for the comparable period in 2022, an increase of $0.4 million. The increase in research and development expenses was primarily driven by an increase in expenses related to the production of clinical trial materials of $0.8 million. The increase was partially offset by a decrease in employee compensation and benefits primarily driven by lower non-cash stock-based compensation expenses and a decrease in clinical development expenses with external contract research organizations.

General and administrative expenses were $3.3 million for the three months ended September 30, 2023, compared to $3.1 million for the comparable period in 2022, a decrease of $0.2 million. The increase in general and administrative expenses was primarily driven by an increase in employee compensation and benefits primarily driven by higher non-cash stock-based compensation expenses.

The company had approximately $59.6 million in cash and cash equivalents and short-term investments as of September 30, 2023, compared to $56.4 million in cash and cash equivalents as of December 31, 2022.

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, 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-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft 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 planned clinical trials, the development of product candidates, expected timing for initiation of future clinical trials, expected timing for receipt of data from clinical trials, the company’s capital resources and ability to finance planned clinical trials, 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 safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sides, 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-Q, 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

Source: Eledon Pharmaceuticals, Inc.

PDS Biotechnology Corp. (PDSB) – Triple Therapy Data Shows Benefits That Far Exceed Published Studies

Posted on by cpereira@noblefcm.com


Friday, November 10, 2023

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune® and Infectimune™ T-cell activating technology platforms. Our Versamune®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate and ovarian cancers.

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.

Triple Therapy Trial Results Show Much Longer Survival In HPV-Positive Cancer. PDS announced data from the Triple Therapy Combination conducted by the NCI (National Cancer Institute of the National Institutes of Health). This trial tested the combination of PDS0101, the tumor-targeted IL-12 derivative PDS0301, and a checkpoint inhibitor to give three mechanisms of action against HPV-positive cancers. While the patient sample is small, these results far exceed survival seen in published studies.

Results Shows Significant Improvements Over Expectations. Final survival data from the patients that had no previous therapy with immune checkpoint inhibitors (ICI naïve patients) showed 6 out of 8 (75%) of the patients alive at 36 months, compared with published studies with about 30% to 50% alive at 12 months and less than 30% alive at 24 months. Median overall survival for the ICI naïve patients has not been reached. 


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. 

Ocugen (OCGN) – 3Q23 Reported With Several Products On Schedule To Reach Clinical Milestones In 2024

Posted on by cpereira@noblefcm.com


Friday, November 10, 2023

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.

Pipeline Is Set For 2024. Ocugen reported 3Q23 loss of $14.2 million or $(0.06) per share, compared with our estimated loss of $18.2 million or $(0.07) per share. Cash balance at the end of the quarter was $53.5 million. During 3Q23, data was presented for OCU400, INDs were approved for OCU410 and OCU410ST, and OCU500 was selected by the NIAID-NIH (National Allergy and Infectious Disease Institute of the National Institutes of Health) to begin clinical testing. NeoCart is also expected to begin a Phase 3 trial in 2H24. We see these pipeline events as important milestones for the company. 

OCU400 is on schedule to finalize the Phase 3 trial design. The Phase 1/2 trial has completed dosing of the RP patients and three LCA patients. The treatment protocols for the Phase 3 trial are expected to be finalized by year-end. Following FDA acceptance, the Phase 3 trial is expected to begin in early 2024 as expected. The final LCA patient data could allow FDA approval to include LCA patients in 2H24.


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. 

Eledon Pharmaceuticals (ELDN) – 3Q23 Reported With Review Of Clinical Progress

Posted on by cpereira@noblefcm.com


Friday, November 10, 2023

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.

3Q23 Loss Was Less Than Projected. Eledon reported 3Q23 loss of $10.3 million or ($0.35) per share, compared with our estimate of a loss of $14.5 million or $(0.57) per share. The difference was mostly due to R&D expense of $7.9 million compared with our estimate of $11.9 million. Cash and equivalents at the end of the quarter were $59.6 million.

Phase 1b Data Shows Protection and Strong Function. As discussed in our Research Note on November 3, data from the Phase 1b trial testing tegoprubart in prevention of renal transplant rejection was presented at the American Society of Nephrology Kidney Week. The data from 11 patients showed tegoprubart was safe, well-tolerated, and had no rejection. Importantly, all of the patients that were evaluated at least 90 days after transplantation had eGFR (estimated glomerular filtration rate) above 70 ml/min/1.72 m2. This is above 50th percentile of 50 ml/min/1.72 m2 average in published studies, and is a strong predicator of graft survival. One patient who completed the 1-year treatment period had a eGFR of 91 ml/min/1.72 m2 and went into the open-label extension study.


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 – PDS Biotech Announces Updated Survival Data from NCI-Led Phase 2 Clinical Trial of PDS0101-Based Triple Combination Therapy in Advanced HPV16-Positive Cancer Patients which Show 75% Survival of ICI Naïve Patients at 36 Months

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

  • 75% of immune checkpoint inhibitor (ICI) naïve patients remain alive at 36 months; published median overall survival (OS) in similar patients is 7-11 months
  • 12-month survival rate in ICI resistant patients is 72%
  • Median OS in ICI resistant patients is approximately 20 months; published median OS is 3.4 months

PRINCETON, N.J., Nov. 09, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary T cell activating platforms, today announced updated survival data from the Phase 2 clinical trial investigating the triple combination of PDS0101, PDS0301 (IL-12 antibody-drug conjugate) and an investigational immune checkpoint inhibitor (ICI) in two groups of advanced cancer patients with various types of human papillomavirus (HPV) 16-positive cancers. The ICI naïve group had not responded to standard-of-care treatments but had not yet been treated with an ICI. The ICI resistant group included patients who had not responded to multiple prior treatments, including ICI therapy. Investigators at the National Cancer Institute (NCI), part of the National Institutes of Health, have completed the primary endpoint analysis of the Phase 2 trial.

In the ICI naïve group, final survival data from the trial indicated that 75% (6/8) of these patients were still alive at 36 months, and the median overall survival (OS) has not yet been reached. Published data on standard-of-care ICIs report 30-50% of these patients typically remain alive at 12 months, and less than 30% of the patients remain alive at 24 months.

In the ICI resistant group, the 12-month OS rate was 72% and the triple combination achieved a median OS of approximately 20 months. In addition:

  • For PDS0101 plus high doses of ICI and PDS0301, the overall response rate (ORR) was 63% (5/8).
  • For PDS0101 plus low doses of ICI and/or PDS0301, the ORR was 5% (1/21).
  • The historical median survival for ICI therapy in HPV-positive cancer ICI resistant patients is reported to be 3.4 months.

“We are encouraged by the survival rates for both ICI naïve and ICI resistant patients with HPV16-positive cancers who were treated with the triple combination therapy,” said Frank Bedu-Addo, PhD, Chief Executive Officer of PDS Biotech. “The ICI resistant data from the VERSATILE-002 trial evaluating PDS0101 in combination with KEYTRUDA® (pembrolizumab) that were reported October 3, 2023, further clarify the path forward for a potential registrational clinical trial of PDS0101 and PDS0301 in combination with a commercial ICI. With this exciting information, we will be finalizing the regulatory and clinical pathway for the triple combination with OS as the primary endpoint.”

PDS0101, PDS Biotech’s lead candidate, is a Versamune® based investigational immunotherapy designed to stimulate a potent targeted T cell attack against HPV16-positive cancers. PDS0301 is a novel, proprietary investigational tumor-targeting IL-12 antibody-drug conjugate that enhances the proliferation, potency, and longevity of T cells in the tumor microenvironment formulated to overcome tumor immune suppression utilizing a different mechanism from checkpoint inhibitors. PDS Biotech has patented the combination of Versamune® and IL-12.   The investigational ICI used in the triple combination therapy is Bintrafusp alfa, a bifunctional fusion protein targeting two independent immunosuppressive pathways (PD-L1 and TGF-β).  

About PDS0101 
PDS0101, PDS Biotech’s lead candidate, is a novel investigational human papillomavirus (HPV)-targeted immunotherapy that stimulates a potent targeted T cell attack against HPV-positive cancers. PDS0101 is given by subcutaneous injection alone or in combination with other immunotherapies and cancer treatments. In a Phase 1 study of PDS0101 in monotherapy, the treatment demonstrated the ability to generate multifunctional HPV16-targeted CD8 and CD4 T cells with minimal toxicity. Interim data suggests PDS0101 generates clinically active immune responses, and the combination of PDS0101 with other treatments can demonstrate significant disease control by reducing or shrinking tumors, delaying disease progression and/or prolonging survival. The combination of PDS0101 with other treatments does not appear to compound the toxicity of other agents.

About PDS0301
PDS0301 is a novel investigational tumor-targeting IL-12 antibody-drug conjugate IL-12 that enhances the proliferation, potency and longevity of T cells in the tumor microenvironment. PDS0301 is given by a subcutaneous injection. PDS0301 is designed to improve the safety profile of IL-12 and to enhance the anti-tumor response.

About PDS Biotechnology 
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune®, Versamune® plus PDS0301, and Infectimune® T cell-activating platforms. We believe our targeted immunotherapies have the potential to overcome the limitations of current immunotherapy approaches through the activation of the right type, quantity and potency of T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the ability to reduce and shrink tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV16-associated cancers in multiple Phase 2 clinical trials and will be advancing into a Phase 3 clinical trial in combination with KEYTRUDA® for the treatment of recurrent/metastatic HPV16-positive head and neck cancer in 2023.   Our Infectimune® based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech. 

References:
Strauss J et al. Journal for ImmunoTherapy of Cancer 2020;8:e001395
Burtness B et al., Lancet. 2019; 394:1915-1928
Ferris RL, et al. NEJM. 2016;375:1856-67

Forward Looking Statements 
This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune® and Infectimune® based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune® and Infectimune® based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to the Company’s currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the other risks, uncertainties, and other factors described under “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the documents we file with the U.S. Securities and Exchange Commission. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.    

Versamune® is a registered trademark and Infectimune® is a trademark of PDS Biotechnology Corporation.

KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.

Investor Contact:
Rich Cockrell
CG Capital
Phone: +1 (404) 736-3838
Email: pdsb@cg.capital

Media Contact:
Gina Cestari
6 Degrees
Phone: +1 (917) 797-7904
Email: gcestari@6degreespr.com

Release – Cocrystal Pharma to Discuss Progress with Novel Inhaled and Oral Influenza A Antiviral CC-42344 at the World Vaccine Congress West Coast

Posted on by aweinsoff@channelchek.com

Research News and Market Data on COCP

NOVEMBER 09, 2023

 DOWNLOAD AS PDF

BOTHELL, Wash., Nov. 09, 2023 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”), announces that President and co-CEO Sam Lee, PhD will discuss progress in developing the novel, broad-spectrum PB2 inhibitor CC-42344 in an oral presentation, “Taking a new route: Development of novel inhaled and oral influenza antiviral, CC-42344” at the World Vaccine Congress West Coast on Tuesday, November 28, 2023 at 3:20 p.m. Pacific Time. The conference is being held November 27-30 at the Santa Clara Convention Center in Santa Clara, Calif.

“It is highly gratifying to be selected for an oral presentation at this prestigious gathering to discuss the significant progress we’ve made in developing CC-42344 for the treatment of pandemic and seasonal influenza A,” said Dr. Lee. “We recently announced authorization by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a Phase 2a human challenge trial with orally administered CC-42344 and we expect to begin treating influenza-infected patients later in this quarter. Additionally, preparations are underway to begin a Phase 1 clinical trial in the first half of 2024 with an inhaled CC-42344 formulation as a potential treatment and prophylaxis for influenza A.”

Following the presentation, Cocrystal will issue a press release with highlights from Dr. Lee’s discussion and the slide presentation will be available on the Company’s website.

About CC-42344
CC-42344 is an PB2 inhibitor that blocks an essential step of viral replication and was discovered using Cocrystal’s proprietary structure-based drug discovery platform technology. It is specifically designed to be effective against all significant pandemic and seasonal influenza A strains and to have a high barrier to resistance due to the way the virus’ replication machinery is targeted. CC-42344 targets the influenza polymerase, an essential replication enzyme with several highly essential regions common to multiple influenza strains. In vitro testing showed CC-42344’s excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as against strains resistant to certain approved influenza antivirals, while also demonstrating favorable pharmacokinetic and safety profiles.

About Seasonal Influenza
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. Influenza is responsible for approximately $10.4 billion in direct costs for hospitalizations and outpatient visits for adults in the U.S. annually.

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 the initiation and characteristics of a Phase 2a human challenge trial in 2023 for CC-42344 as a product candidate for oral treatment of influenza A, and a Phase 1 clinical trial in 2024 for CC-42344 as a product candidate for inhaled treatment of influenza A, and the potential efficacy and clinical benefits of, and market for, such product candidate. 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, risks relating to the manufacturing and research delays arising from labor shortages and other factors, the ability of our Clinical Research Organization partners to recruit volunteers for, and to proceed with, clinical trials, and general risks arising from or involved in conducting clinical studies for CC-42344, including the results of such studies. 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, 2022. 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

# # #

Source: Cocrystal Pharma, Inc.

Released November 9, 2023

Release – ZyVersa Therapeutics Announces Article Published in Peer-Reviewed Biomedical Journal Demonstrating That NLRP3 Inflammasome Inhibition Attenuates Inflammatory Bowel Disease Symptoms in Animal Model

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Nov 9, 2023

PDF Version

  • Inflammatory bowel disease (IBD) is a chronic inflammatory disease of the gastrointestinal tract affecting 1 in 100 Americans (around 2.4 million people) that can lead to disabling bowel symptoms and progressive bowel damage.
  • Study provides direct evidence that NLRP3 signaling is over-activated in IBD, and that its inhibition attenuates intestinal inflammation and tissue damage, leading to significant improvements in IBD symptoms, and restoration of normal intestinal microbial flora.
  • ZyVersa is developing Inflammasome ASC Inhibitor IC 100 which can inhibit up to 12 different inflammasomes (including NLRP3 inflammasomes) and their associated ASC specks which perpetuate damaging inflammation.

WESTON, Fla., Nov. 09, 2023 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of inflammatory and renal diseases, announces publication of an article in the peer-reviewed Biomedical Journal demonstrating that inhibiting NLRP3 inflammasomes in an IBD animal model attenuates intestinal inflammation and tissue damage, leading to significant improvements in IBD symptoms, and restoration of normal intestinal microbial flora.

In the paper titled, “Inhibition of NLRP3 attenuates sodium dextran sulfate-induced inflammatory bowel disease through gut microbiota regulation,” the authors evaluated human colon biopsy samples from patients with IBD and healthy controls, and conducted a study in an IBD mouse model. Following are key findings reported in the paper:

  • NLRP3 and IL-1β expression is increased in the colon of IBD patients.
  • NLRP3 inhibition in the IBD animal model:

  • Inhibited NLRP3 inflammasome signaling in the colon, resulting in significantly reduced levels of the pro-inflammatory cytokines IL-1b, IL-6, and TNF-α.
  • Alleviated severe diarrhea and significantly improved IBD symptoms, based on the disease activity index score.
  • Attenuated histopathological changes indicative of tissue damage (goblet cell reduction, crypt destruction, and epithelial barrier disruption).
  • Restored gut microbiota to normal.

The authors stated, “In conclusion, this study provides direct evidence that NLRP3 signaling is over-activated in IBD patients. The inhibition of NLRP3 reverses the IBD-like symptoms in DSS-induced mice, which the regulatory effects on gut microbiota might mediate. Overall, this present study provides a basis for the clinical application of NLRP3 as a target for IBD treatment.” To read the article, Click Here.

“Restoration of quality of life is the ultimate long-term goal in IBD management. Although disease remission can often be achieved with current therapies, bothersome symptoms can still prevail,” stated Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO and President. “The research published in the Biomedical Journal provides support for inflammasome inhibition as a promising treatment option for IBD. ZyVersa is developing Inflammasome ASC inhibitor IC 100. Unlike NLRP3 inhibitors, designed to inhibit formation of one inflammasome to block initiation of the inflammatory cascade, IC 100 was designed to inhibit multiple types of inflammasomes and their associated ASC specks to uniquely block both initiation and perpetuation of damaging inflammation, which we believe is necessary to control chronic inflammation.” To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.

About Inflammasome ASC Inhibitor IC 100

IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, Pyrin. Intracellularly, IC 100 binds to ASC monomers, inhibiting inflammasome formation, thereby blocking activation of IL-1β early in the inflammatory cascade. IC 100 also binds to ASC in ASC Specks, both intracellularly and extracellularly, further blocking activation of IL-1β and the perpetuation of the inflammatory response that is pathogenic in inflammatory diseases. Because active cytokines amplify adaptive immunity through various mechanisms, IC 100, by attenuating cytokine activation, also attenuates the adaptive immune response.

About ZyVersa Therapeutics, Inc.

ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and other inflammatory diseases. For more information, please visit www.zyversa.com.

Cautionary Statement Regarding Forward-Looking Statements

Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.

New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.

Corporate and IR Contact:
Karen Cashmere
Chief Commercial Officer
kcashmere@zyversa.com
786-251-9641

Media Contacts
Tiberend Strategic Advisors, Inc.
Casey McDonald
cmcdonald@tiberend.com
646-577-8520

Dave Schemelia
dschemelia@tiberend.com
609-468-9325

Release – Ocugen Provides Business Update With Third Quarter 2023 Financial Results

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

November 9, 2023

PDF Version

Conference Call and Webcast Today at 8:30 a.m. ET

  • OCU400 demonstrated favorable safety and tolerability profile in retinitis pigmentosa (RP) and Leber congenial amaurosis (LCA) subjects
  • Completed dosing of three LCA patients including a pediatric patient
  • OCU400 Phase 1/2 study results suggest stabilization or improvement of Best-Corrected Visual Acuity (BCVA) or Multi-Luminance Mobility Testing (MLMT) or Low-Luminance Visual Acuity (LLVA) in treated eyes of 83% (10/12) subjects
  • Stabilization or improvement in MLMT scores from baseline in 86% (6/7) of RHO subjects demonstrated gene-agnostic property of OCU400 modifier gene therapy and its potential benefits in broader RP and LCA subjects
  • Ocugen’s inhaled mucosal vaccine candidate for COVID-19—OCU500—selected by National Institutes of Health (NIH)/National Institute of Allergy and Infectious Diseases’ (NIAID) Project NextGen for inclusion in clinical trials

MALVERN, Pa., Nov. 09, 2023 (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 reported third quarter 2023 financial results along with a general business update.

“Ocugen has made significant pipeline progress in the third quarter,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “In particular, based on the OCU400 data presented in September, I am as enthusiastic as ever regarding the potential for our modifier gene therapy approach to make an important difference in the lives of people living with blindness diseases. OCU400—our lead candidate—has the potential to address multiple genetic mutations with a single product compared to traditional gene therapies that target one gene at a time.”

This clinical efficacy update provided in September 2023 included data for 12 subjects who have completed a minimum of 6 months follow up. 83%, 83%, and 75% of subjects demonstrated stabilization or improvements in OCU400 treated eyes on BCVA, LLVA, and MLMT scores, respectively from baseline. 86% of subjects with the RHO gene mutation experienced either stabilization or increase in MLMT scores from baseline, including a subset of 29% that demonstrated a three Lux luminance level improvement. Preservation of remaining vision, slowing disease progression, or improving the vision can significantly impact patients’ quality of life.

“The improvements in BCVA, LLVA and MLMT in RHO patients—a disease affecting more than 10,000 people in the U.S. alone—supports the gene-agnostic mechanism of action for OCU400,” said Dr. Musunuri.

In October 2023, OCU500, was selected by the NIAID Project NextGen for inclusion in clinical trials. OCU500 will be tested via two different mucosal routes, inhalation into the lungs and as a nasal spray. Currently used injected vaccines, including mRNA vaccines, are not effective in preventing infection and spread although effective against severe infection. Generating mucosal immunity in nasal and respiratory airways could help block the infection at its origin, thus limiting spread.

“NIAID support for our mucosal vaccine platform is the result of many months of hard work and dedicated effort by our Ocugen team and is a first step in potentially expanding the platform to flu and other respiratory viral diseases and infections,” said Dr. Musunuri. “Additionally, this funding makes it possible to focus the majority of Ocugen’s R&D and clinical resources on our first-in-class gene and cell therapies.”

As Ocugen prepares to start Phase 3 for OCU400, begin dosing patients for OCU410 and OCU410ST, and initiate the Phase 1 trial for OCU500 in collaboration with NIAID, the company is making meaningful progress toward its long-term strategy and delivering on each of its scientific platforms in the near-term.

Ophthalmic Gene Therapies—First-in-class

  • OCU400 – Completed dosing adult RP patients in the dose-escalation and dose-expansion portions of the trial; completed dosing three LCA patients including a pediatric patient. Phase 3 clinical trial for the treatment of RP to be initiated in early 2024 following FDA concurrence on study design. Subsequently, the Company is expecting to expand the OCU400 Phase 3 clinical trial for LCA patients in the second half of 2024 based on Phase 1/2 study results in LCA patients and alignment with the FDA.
  • OCU410 and OCU410ST – IND applications to initiate Phase 1/2 trials for both OCU410 and OCU410ST were cleared by the FDA and the Company intends to dose patients in Phase 1/2 trials by the end of 2023.

Ophthalmic Biologic Product

  • OCU200 – Continuing to work on the Company’s response to the FDA regarding the IND application and expect to initiate the Phase 1 clinical trial in the first half of 2024, contingent on the lift of the FDA hold and adequate availability of funding.

Regenerative Cell Therapies—First-in-class

  • NeoCart® – Ocugen’s autologous regenerative cell therapy (using patients’ own cartilage cells) remains on track to begin its Phase 3 clinical trial in the second half of 2024. A cGMP facility for manufacturing NeoCart is expected to be completed at the end of 2023 and qualification is expected in the first half of 2024.  

Vaccines Portfolio—First-in-class

  • Mucosal Vaccine Platform – The Company is collaborating with NIAID to initiate clinical trials of OCU500 in early 2024.

Third Quarter 2023 Financial Results

  • The Company’s cash, cash equivalents, and investments totaled $53.5 million as of September 30, 2023, compared to $90.9 million as of December 31, 2022. The Company had 256.5 million shares of common stock outstanding as of September 30, 2023.
  • Total operating expenses for the three months ended September 30, 2023 were $15.4 million and included research and development expenses of $6.3 million and general and administrative expenses of $9.1 million. This compares to total operating expenses for the three months ended September 30, 2022 of $23.1 million that included research and development expenses of $15.6 million and general and administrative expenses of $7.5 million.
  • Ocugen reported a $0.06 net loss per common share for the three months ended September 30, 2023 compared to a $0.10 net loss per common share for the three months ended September 30, 2022.

Conference Call and Webcast Details

Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s senior management team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call or webcast using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 1787631
Webcast: Available on the events section of the Ocugen investor site

A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.

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, 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 include, but are not limited to, statements regarding the Company’s clinical development activities and related anticipated timelines; strategy, business plans and objectives for its clinical stage programs; plans and timelines for the preclinical and clinical development of its 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; and Ocugen’s financial condition. 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, as well as discussions of potential risks, uncertainties, and other important factors in Ocugen’s subsequent filings with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. 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
IR@ocugen.com 

Release – GeoVax Reports Third Quarter 2023 Financial Results and Provides Business Update

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

 

  • Last updated: 08 November 2023 21:03
  • Created: 08 November 2023 15:32
  • Hits: 505

Significant Progress towards data milestones in Phase 2 Program of GEO-CM04S1, Next-generation COVID-19 vaccine

Gedeptin® clinical data from Phase 1/2 study presented at AACR-AHNS Head and Neck Cancer Conference showing safe administration and tumor reduction

Multi-Product License Secured for ProBioGen’s AGE1.CR.pIX® suspension cell line to enhance manufacturing capabilities of MVA-based vaccine portfolio

Company to host conference call and webcast today at 4:30 p.m. ET

ATLANTA, GA, November 8, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced financial results for the third quarter ended September 30, 2023 and provided a business update.

“We are thrilled to see material advancements across all aspects of our business. Through the third quarter, we are delivering on our promise to bring transformative change to the treatment of COVID-19 by completing enrollment in our healthy volunteer Phase 2 clinical trial evaluating GEO-CM04S1 as a potentially more robust, more durable booster vaccine. In addition, with two additional Phase 2 trials ongoing in immunocompromised population, and recent publication of positive safety and potent immunogenicity data in this population, we have strengthened our conviction in the potential of our next-generation COVID-19 vaccine (GEO-CM04S1) for immunocompromised patients. We believe the need for new treatment options for immunocompromised patients in the fight against COVID-19 is significant, particularly as we begin to enter the winter surges of this diseases, where these patients will be left most vulnerable,” said David Dodd, Chief Executive Officer of GeoVax.

“We are preparing to announce initial data readouts from two of our Phase 2 GEO-CM04S1 trials as well as updates from our Gedeptin® clinical trial against head and neck cancer in the coming months. Earlier this summer, we were delighted to present clinical data which revealed the safe and feasible administration of Gedeptin®. We believe the Gedeptin mechanism of action to be solid tumor agnostic, and therefore offers the potential as a therapeutic option for multiple cancer types and positions the product for long-term growth. The third quarter of 2023 also saw new patents issued for our Ebola, Marburg, Malaria, and HIV vaccines, demonstrating the strength of our team’s ability to execute on our broader strategy. In addition, we advanced our manufacturing process through the signing of a landmark multi-product commercial license agreement with ProBioGen for the use of their AGE1.CR.PIX®suspension cell line. We believe that these catalysts altogether will have a profound impact on patients in need and all our stakeholders.”

Recent Business Achievements

GEO-CM04S1

  • Enrollment completed for Phase 2 clinical trial evaluating GEO-CM04S1 as a booster for healthy patients who have previously received the Pfizer or Moderna mRNA vaccine in September of 2023. An initial data readout is expected soon.
  • GEO-CM04S1 demonstrated potent antibody and cellular immunity in immunocompromised patients in a recent publication in the journal, Vaccines, in August 2023. GeoVax’s Phase 2 clinical trial evaluated the safety and immunogenicity of GEO-CM04S1, compared to either the Pfizer/BioNTech or Moderna mRNA-based vaccine in patients who have previously received an allogeneic hematopoietic cell transplant, an autologous hematopoietic cell transplant or CAR-T cell therapy. Most promising in the results published was the demonstration of the potential of GEO-CM04S1 as a variant agnostic COVID-19 vaccine, providing potent immunity from the Wuhan through Delta and Omicron strains.
    • In October 2023, GeoVax commenced the planned site expansion for this trial to accelerate patient enrollment. In addition to study enrollments completed at the City of Hope Medical Center (Duarte, California), the trial is now open at Wake Forest Baptist Medical Center (Winston Salem, North Carolina), the University of Massachusetts Medical Center (Worcester, Massachusetts), and Fred Hutchinson Cancer Center (Seattle, Washington).
  • Commenced investigator-initiated randomized observer-blinded Phase 2 clinical trial of COVID-19 boosters with GEO-CM04S1 or Pfizer-BioNTech bivalent vaccines in patients with chronic lymphocytic leukemia (CLL) in July of 2023. The trial is rapidly enrolling patients and progressing towards an interim data review. 
  • Presented data for GEO-CM02 at the Keystone Symposia Conference in an abstract titled, MVA-vector multi-antigen COVID-19 vaccines induce protective immunity against SARS-CoV-2 variants spanning Alpha to Omicron in preclinical animal models in September of 2023. The data revealed that the GEO-CM02 vaccine induced immune responses that were efficacious against the original Wuhan strain and Omicron variants with a single dose. This effort expands upon GeoVax’s hypothesis that vaccines designed to induce both antibodies and T-cell responses to multiple viral structural proteins can address the issue of viral variation and escape from the immune system without the need for repeated seasonal adjustments.

Gedeptin®

  • Gedeptin® clinical data presented at the AACR-AHNS Head and Neck Cancer Conference in an abstract titled, Phase 1/2 study of Ad/PNB with Fludarabine for the Treatment of Head and Neck Squamous Cell Carcinoma (HNSCC) in July 2023. The FDA-funded study revealed that the administration of Gedeptin® is safe and feasible.

Advanced Vaccine Manufacturing Process

  • GeoVax and ProBioGen announced the signing of a landmark multi-product commercial license agreement for ProBioGen’s groundbreaking AGE1.CR.PIX® suspension cell line in September 2023. The agreement enhances the manufacturing capabilities of GeoVax’s entire Modified Vaccinia Ankara (MVA)-based vaccine portfolio with respect to both scale and flexibility. This follows the May 2023 agreement with Advanced Bioscience Laboratories, Inc. (ABL) to support current Good Manufacturing Practices (cGMP) production of the Company’s vaccine candidates. These agreements move the Company toward fully implementing a continuous cell line manufacturing system that will provide lower-cost, scalable versatility for our entire MVA-based vaccine portfolio.

Intellectual Property Developments

  • GeoVax recently secured multiple positive patent decisions covering notice of allowances for Ebola, Marburg, Malaria and HIV vaccines.
    • GeoVax announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for Patent Application No. 17/584,231 titled patent titled “Replication Deficient Modified Vaccina Ankara (MVA) Expressing Marburg Virus Glycoprotein (GP) and Matrix Protein (VP40).”
    • GeoVax announced that the U.S Patent and Trademark Office has issued a Notice of Allowance for Patent Application No. 17/409,574 titled “Multivalent HIV Vaccine Boost Compositions and Methods of Use.”
    • GeoVax announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for Patent Application No. 17/726,254 titled “Compositions and Methods for Generating an Immune Response to Treat or Prevent Malaria.”
    • GeoVax announced that the U.S. Patent and Trademark Office has issued Patent No. 11,701,418 B2 to GeoVax, pursuant to the Company’s patent application No. 15/543,139 titled “Replication-Deficient Modified Vaccinia Ankara (MVA) and Matrix Protein (VP40).”

Upcoming Events

  • GeoVax will participate in upcoming conferences and industry events:
    • Vaccines Summit 2023, November 13-15, 2023, Boston, MA
    • World Vaccine Congress, West Coast, November 27-30, 2023, Santa Clara, CA
    • NobleCon19, December 3-5, Boca Raton, FL
    • Emerging Growth Conference, December 6-7, Virtual

Third Quarter 2023 Financial Results

Net Loss: Net loss for the three months ended September 30, 2023, was $8,408,818 ($0.32 per share) as compared to $3,968,102 ($0.17 per share) for the comparable period in 2022. For the nine months ended September 30, 2023, the Company’s net loss was $18,374,354 ($0.69 per share) as compared to a net loss of $8,637,316 ($0.63 per share) in 2022.

R&D Expenses: Research and development expenses were $6,947,979 and $14,486,896 for the three-month and nine-month periods ended September 30, 2023, compared to $2,721,196 and $5,358,917 for the comparable periods in 2022, with the increase primarily due to the cost of conducting clinical trials for GEO-CM04S1 and Gedeptin, costs of manufacturing materials for use in our clinical trials, additional personnel costs, technology license fees, costs of preclinical research activities and a generally higher level of activity.

G&A Expenses: General and administrative expenses were $1,651,775 and $4,562,293 for the three-month and nine-month periods ended September 30, 2023, compared to $1,249,337 and $3,363,672 for the comparable periods in 2022, with the increase primarily due to higher personnel costs, investor relations consulting costs, patent costs and other expenses supportive of a higher level of activity.

Cash Position: GeoVax reported cash balances of $12,687,041 at September 30, 2023, as compared to $27,612,732 at December 31, 2023.

Further information is included in the Company’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission.

Conference Call Details

Management will host a conference call scheduled to begin at 4:30 p.m. ET today, November 8, 2023, to review financial results and provide an update on corporate developments. A question-and-answer session will follow management’s formal remarks.

Domestic: +1-800-715-9871

International: +1-646-307-1963

Conference ID: 7519986

Webcast: https://edge.media-server.com/mmc/p/r4i9d5j7

A webcast replay of the call will be available for three months via the same link as the live webcast approximately two hours after the end of the call.

About GeoVax

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. For more information, visit our website: www.geovax.com.

Forward-Looking Statements

This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “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 and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.

Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. 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. 

Company Contact:        Investor Relations Contact:        Media Contact:
info@geovax.com paige.kelly@sternir.com sr@roberts-communications.com
678-384-7220 212-698-8699 202-779-0929

GeoVax Labs, Inc. (GOVX) – 3Q23 Quarterly Report Includes 2024 Milestones

Posted on by cpereira@noblefcm.com


Thursday, November 09, 2023

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.

GeoVax Announces 2024 Milestones and 3Q23 Financial Results. GeoVax reported a 3Q23 loss of $8.4 million or $(0.32) per share. The company reviewed the progress made during the quarter, including completion of enrollment for the Phase 2 trial testing CM04S1 as a booster in healthy patients, presentation of data from the Phase 1/2 data for Gedeptin showing safety and tumor reduction, and addition of 3 new clinical sites for the Phase 2 trial for CM04S1 in immunocompromised patients.

CM04S1 Has Made Progress In Three Trials. Enrollment has been completed in the Phase 2 trial testing CM04S1 as a booster for healthy patients that had received the Pfizer or Moderna mRNA vaccines. An announcement of the top-line data is expected shortly. As discussed in our Research Note on September 25, a publication of data from the Phase 2 trial in immuno-compromised patients showed both humoral and cellular immune responses in patients that have difficulty responding to vaccination. Three new clinical sites were also added to the trial. An Investigator Initiated Trial (IIT) in chronic lymphocytic leukemia (CLL) is expected to announce interim data in the coming months. 


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. 

Apollo Medical Scales Medicaid Presence with Purchase of CFC

Posted on by slightbourne@noblecapitalmarkets.com

Healthcare investor favorite Apollo Medical Holdings (NASDAQ: AMEH) is expanding its presence in value-based care arrangements through the acquisition of Community Family Care (CFC), a large independent physician association based in Los Angeles. The all-cash deal worth up to $202 million reflects ApolloMed’s strategy of targeting risk-bearing providers as a high-growth segment of the health biotech sector.

CFC manages care for over 200,000 members via its network of more than 350 primary care physicians and 500 specialists. The group has a strong presence in Medicaid, with a Restricted Knox Keene (RKK) license enabling it to take on full risk for this population in California. CFC also serves Medicare and commercial members under value-based contracts that incentivize providers to control costs and improve health outcomes.

For ApolloMed, acquiring CFC significantly boosts its portfolio of managed lives, particularly under global capitation arrangements that cover total cost of care. Additionally, CFC’s long track record of profitability in Medicaid provides ApolloMed with demonstrated capabilities to take on risk successfully in a population that is often challenging for providers.

The deal exemplifies a unique competitive edge for ApolloMed – its end-to-end platform spanning technology, management services, and risk contracting. CFC has utilized ApolloMed’s care enablement tools since 2020, allowing it to perform well under value-based care. Now, full acquisition enables tight integration and aligned incentives as CFC transitions to an ApolloMed care partner responsible for total cost and outcomes.

For 2023, CFC is expected to generate $190 million in revenue with $25 million in adjusted EBITDA, an impressive 13% margin for a risk-bearing provider group. ApolloMed has agreed to an acquisition price of up to $202 million, including $152 million in cash, $20 million in ApolloMed stock, and $30 million in potential milestone payments. The deal is anticipated to close in Q1 2024 after clearing regulatory reviews.

Rapid Growth in Value-Based Care

The acquisition comes at a time of rapid expansion in value-based care, where providers take on financial accountability for cost and quality of healthcare for a population of patients. Government and commercial payers are pushing providers into these arrangements to incentivize focus on preventative care and eliminating wasteful spending.

Analysts size the global market for value-based care at $3.1 billion by 2030, reflecting a blistering compound annual growth rate of 21.7% from 2022. In the United States, value-based care penetration is expected to reach 65% of healthcare payments by 2025.

ApolloMed is positioned at the forefront of this transformation with its integrated platform and focus on enabling providers, such as CFC, to take on risk successfully. The CFC deal adds a sizable value-based care presence to ApolloMed’s portfolio, demonstrating the appetite to aggressively scale its model with like-minded partners.

Targeting High-Growth Segments

The CFC acquisition also highlights ApolloMed’s focus on areas of healthcare with strong secular tailwinds: government programs and risk-based arrangements.

Medicaid represents a massive $650 billion total addressable market, and ApolloMed is specifically targeting expansion in California, which has among the most progressive Medicaid programs in supporting coordinated care models. CFC’s strong capabilities in the Medicaid population will be a key strategic asset.

Likewise, ApolloMed’s thesis of investing behind risk-bearing providers is underscored by CFC’s demonstrated ability to generate double-digit margins while managing patients under capitated contracts. As fee-for-service reimbursement models decline, providers capable of taking on risk will be increasingly valuable.

Wall Street’s View

ApolloMed has been a darling of growth investors, with shares up 270% over the past five years. Revenue and EBITDA have compounded annually at 50% and 90%, respectively, as the company scales its platform.

The market continues to reward this rapid expansion, with ApolloMed trading at a premium valuation of 50x P/E. Bulls believe the company’s strategy and competitive moats position it for continued acceleration as value-based care proliferates.

Meanwhile, bears argue that ApolloMed’s nosebleed valuation leaves little room for error. There are also concerns around rising fragmentation in California’s Medicaid market challenging operators.

However, with impressive unit economics and strong execution thus far, ApolloMed has many convinced it can maintain momentum. The CFC deal offers further validation of the company’s model and market opportunity. Investors will be watching closely for successful integration and financial accretion.

If ApolloMed can effectively leverage CFC to penetrate Medicaid and value-based arrangements more deeply, the deal may be looked back upon as an inflection point in the company’s growth story. At minimum, it provides another data point for ApolloMed’s ability to execute on acquisitions rapidly expanding its national footprint – a core piece of the bull thesis.

MustGrow Biologics Corp. (MGROF) – Receiving More Approval

Posted on by cpereira@noblefcm.com


Wednesday, November 08, 2023

Joe Gomes, 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.

New Approval. Yesterday, MustGrow announced the Company received Health Canada’s Pest Management Regulatory Agency (PMRA) approval to commence large-scale field trials via NexusBioAg’s 2024 BioAdvantage Trials Program (BAT Program). The program will focus on the Company’s TerraMG mustard-derived soil biopesticide technology for use in Canadian canola and pulse crop markets.

NexusBioAg BAT Program. The BAT Program is an industry leading field trialing program with an established process to gather data from large field scale trials across Canada. NexusBioAg, a partner of MustGrow and the operator of the program, validates product efficacy and establishes the product value and opportunity. Through the BAT Program, NexusBioAg farm customers will have access to MustGrow’s mustard plant-based product.


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.