Category: Health

Release – GeoVax Next-Generation COVID-19 Vaccine Demonstrates Potent Antibody and Cellular Immunity in Immunocompromised Patients

Posted on by aweinsoff@channelchek.com

Research News and Market Data GOVX

 

  • Last updated: 19 September 2023 13:26
  • Created: 19 September 2023 15:09
  • Hits: 72

Data Published from Phase 2 Open-Label Study of GEO-CM04S1

ATLANTA, GA, September 19, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing human vaccines and immunotherapies against infectious diseases and cancer, announced today the publication of data from the ongoing Phase 2 trial of its next-generation COVID-19 vaccine (GEO-CM04S1) in the journal Vaccines. The article, authored by researchers at the City of Hope National Medical Center, can be accessed here: GEO-CM04S1 Publication.

The Phase 2 clinical trial (ClinicalTrials.gov Identifier: NCT04977024) is evaluating the safety and immunogenicity of GEO-CM04S1, compared to either the Pfizer/BioNTech or Moderna mRNA-based vaccine, in patients who have previously received either an allogeneic hematopoietic cell transplant, an autologous hematopoietic cell transplant or chimeric antigen receptor (CAR) T cell therapy (i.e., patients who have reduced immune system function as a result of treatment). These patients have significantly compromised immune system function and generally respond at suboptimal levels after receiving currently available COVID-19 vaccines. As such, these patients are at greater risk for developing severe disease if infected and would likely benefit from the types of immune responses induced by the GEO-CM04S1 vaccine, which are more broadly specific and include activation of both the antibody and T cell arms of the immune system.

The article, titled “Stimulation of Potent Humoral and Cellular Immunity via Synthetic Dual-Antigen MVA-Based COVID-19 Vaccine COH04S1 in Cancer Patients Post Hematopoietic Cell Transplantation and Cellular Therapy”, describes the immune response analyses conducted on the open-label portion of the Phase 2 trial, indicating that GEO-CM04S1 (referred to as COH04S1 in the article) is highly immunogenic in these immunocompromised patients, inducing potent humoral (antibody) and cellular (T cell) responses, including neutralizing antibodies against SARS-CoV-2 ancestral virus and variants of concern, including the highly immune-evasive Omicron XBB 1.5 variant.

David Dodd, GeoVax Chairman and CEO, commented, “The data presented in this publication confirm our earlier findings in healthy adults and give us confidence that we are on the right path. Protecting individuals at highest risk in our society requires next-generation vaccines, specifically with more robust immune responses, durable protection, and the ability to address continued emerging variants. The mRNA vaccines and monoclonal antibody therapies have been shown to be inadequate in providing protective immunity in such immunocompromised patients. Variants will continue to emerge, and the public health community will continue to be challenged to minimize the infection impact, thereby reducing the risk of severe disease, hospitalizations and death. Vaccines that are “variant-agnostic” such as GEO-CM04S1 will increasingly be critical to reducing such risks, protecting the most vulnerable populations and improving our ability to overcome such infectious threats.”

About GEO-CM04S1

GEO-CM04S1 is based on GeoVax’s MVA viral vector platform, which supports the presentation of multiple vaccine antigens to the immune system in a single dose. GEO-CM04S1 encodes for both the spike (S) and nucleocapsid (N) antigens of SARS-CoV-2 and is specifically designed to induce both antibody and T cell responses to those parts of the virus less likely to mutate over time. The more broadly functional engagement of the immune system is designed to protect against severe disease caused by continually emerging variants of COVID-19. Vaccines of this format should not require frequent and repeated modification or updating.

In addition to the ongoing Phase 2 study in hematological cancer patients, GEO-CM04S1 is being evaluated in two other Phase 2 clinical trials:

  • As a booster vaccine for healthy patients who have previously received the Pfizer or Moderna mRNA vaccine. gov Identifier: NCT04639466. GeoVax recently announced that this trial has met its targeted patient enrollment.
  • As a booster vaccine in immunocompromised patients with chronic lymphocytic leukemia (CLL), a recognized high-risk group for whom current mRNA vaccines and monoclonal antibody (MAb) therapies appear inadequate relative to providing protective immunity. ClinicalTrials.gov Identifier: NCT05672355.

Further underscoring the need for next-generation COVID-19 vaccines such as GEO-CM04S1, GeoVax scientists co-authored an article titled, “MVA-Vectored Universal Beta-Coronavirus Vaccine Design & Development”, published in the June 2023 issue of the online journal Vaccine Insights. The article, accessible here, provides expert insight into the emergence of SARS-CoV-2 (COVID-19), the risk of new “spillover events” from animal hosts, and how this risk can be addressed proactively. Regarding COVID-19 and its continually evolving variants, the authors describe the limitations of first-generation vaccines and the potential for MVA-vectored vaccines such as GEO-CM04S1 to overcome these limitations.

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. 

Investor Relations Contact:

GeoVax Labs, Inc.

investor@geovax.com

678-384-7220

Media Contact:

Susan Roberts

sr@roberts-communications.com

202-779-0929

Release -Tonix Pharmaceuticals Announces Presentation of Non-Clinical Studies Supporting the Mechanism of TNX-1900 (Intranasal Potentiated Oxytocin) at the 2023 International Headache Congress

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

September 18, 2023 7:00am EDT

Oxytocin receptors and CGRP are co-expressed on human trigeminal ganglia neurons and their expression is increased in inflammation

Human tissue data support the proposed mechanism of action of TNX-1900 in treating headache: oxytocin treatment blocks the release of CGRP

CHATHAM, N.J., Sept. 18, 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 that David C. Yeomans, Ph.D. presented data relevant to the proposed mechanism of TNX-1900 (intranasal potentiated oxytocin) in treating chronic migraine in a poster and an oral presentation at the 2023 International Headache Congress (IHC), being held September 14-17, 2023, 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 Pharmaceuticals. Professor Yeomans was a founder of Trigemina, which Tonix acquired, and he remains a consultant to Tonix. A copy of the poster is available under the Scientific Presentations tab of the Tonix Pharmaceuticals corporate website at www.tonixpharma.com.

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. 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

“In animal studies, oxytocin has been shown to inhibit trigeminal neurons which we believe relates to its mechanism in preventing migraines,”1 said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “The data presented at 2023 IHC by Professor Yeomans shows that oxytocin receptors are present on human trigeminal ganglia neurons, and that these same neurons express CGRP, a key peptide in the pathogenesis of migraine. These data support the proposed mechanism of TNX-1900, which we believe inhibits the release of CGRP from trigeminal neurons that otherwise would trigger a cascade leading to migraine.”

“Similar to animals, human trigeminal ganglia neurons express oxytocin receptors and also co-express CGRP,” said Professor Yeomans. “In addition, the inflammatory mediator IL-6 induces robust upregulation of oxytocin receptors on human trigeminal ganglia neurons. In the presence of inflammation, which is persistently present in chronic migraine, there is a robust increase in the expression of both oxytocin receptors and CGRP in human trigeminal neurons.”

In February 2023, Tonix initiated enrollment in the Phase 2 ‘PREVENTION’ study of TNX-1900 for the prevention of migraine headache in chronic migraineurs. Topline results from the study are expected in the fourth quarter of 2023. In addition, TNX-1900 is also being evaluated in investigator-initiated Phase 2 trials in adolescent obesity (initiated July 2023), binge eating disorder and social anxiety disorder.

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

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 the fourth quarter of 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Topline results from a proof-of-concept Phase 2 study were reported in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily oral formulation being developed as a treatment for major depressive disorder (MDD), that completed enrollment in a Phase 2 proof-of-concept study in the third quarter of 2023, with topline results expected in the fourth quarter of 2023. TNX-4300 (estianeptine) is a single isomer version of TNX-601, a small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. Relative to tianeptine, estianeptine lacks activity on the µ-opioid receptor while maintaining activity in the rat Novel Object Recognition test in vivo and the ability to activate PPAR-β/δ and neuroplasticity in tissue culture. TNX-1900 (intranasal potentiated oxytocin), is in development for preventing headaches in chronic migraine, and has completed enrollment in a Phase 2 proof-of-concept study with topline data expected in the fourth quarter of 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. 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.

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

Source: Tonix Pharmaceuticals Holding Corp.

Released September 18, 2023

Release – Tonix Pharmaceuticals Announces Poster Presentation Involving TNX-1700 in Preclinical Colorectal Cancer Models at the Seventh International Cancer Immunotherapy Conference 2023

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

September 14, 2023 7:00am EDTDownload as PDF

CHATHAM, N.J., Sept. 14, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced a poster presentation at the Seventh International Cancer Immunotherapy Conference 2023 (CICON23): Translating Science into Survival. The Conference is being held September 20-23, 2023, in Milan, Italy. A copy of the poster will be made available on the Tonix Pharmaceuticals website following the presentation at www.tonixpharma.com.

Poster Presentation Details
    
Title:  mTFF2-MSA (mTNX-1700) Suppresses Tumor Growth and Increases Survival in Anti-PD-1 Treated CT26.WT Subcutaneous and CT26-Luciferase Orthotopic Syngeneic Colorectal Cancer Models by Targeting MDSCs in BALB/C Mice
    
Authors:  Bruce L. Daugherty, Rebecca J. Boohaker, Rebecca Johnstone, Karr Stinson, Grace Zhao, Mingfa Zang, Jin Qian, Timothy C. Wan, Seth Lederman
    
Session Date:  September 21, 2023
    
Session Time:  12:30 p.m. – 2:00 p.m
    
Poster Number:  P220


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 the fourth quarter of 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 proof-of-concept study has been completed, and topline results were reported in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily oral formulation being developed as a treatment for major depressive disorder (MDD), that completed enrollment in a Phase 2 proof-of-concept study in the third quarter of 2023, with topline results expected in the fourth quarter of 2023. TNX-4300 (estianeptine) is a single isomer version of TNX-601, small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer’s disease and Parkinson’s disease. Relative to tianeptine, estianeptine lacks activity on the µ-opioid receptor while maintaining activity in the rat Novel Object Recognition test in vivo and the ability to activate PPAR-β/δ and neuroplasticity in tissue culture. TNX-1900 (intranasal potentiated oxytocin), is in development for preventing headaches in chronic migraine, and has completed enrollment in a Phase 2 proof-of-concept study with topline data expected in the fourth quarter of 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 third 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. 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.

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

Source: Tonix Pharmaceuticals Holding Corp.

Released September 14, 2023

Ocugen (OCGN) – OCU400 Update Shows Improvement In Three Vision Measures

Posted on by cpereira@noblefcm.com


Thursday, September 14, 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.

Data Shows Clinically Meaningful Improvements. Ocugen provided an update to the Phase 1/2 clinical trial data for OCU400 in retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA), two rare diseases in which genetic mutations cause loss of vision and progress to blindness. We see these data as clinically meaningful for these indications as well as providing proof-of-concept for Ocugen’s method of targeting a “master control gene” that has downstream effects on other genes.

We See Proof-of-Concept For The “Master Control Gene” Approach. RP is a group of genetic disorders with over 100 possible genetic mutations that may be present and contributing to loss of vision. This makes the course of disease highly variable and difficult to target with a single therapy. Ocugen has been developing OCU400 to target the RHO and NR2E3 genes that control pathways and regulate other genes that act later in those pathways. The data show that OCU400 can affect several genetic pathways in eye, with improvements in three measures of vision.


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. 

Cancer Immunotherapy Developer Calidi Goes Public Via SPAC Merger

Posted on by slightbourne@noblecapitalmarkets.com

Calidi Biotherapeutics has completed its merger with special purpose acquisition company First Light Acquisition Group (FLAG), debuting as a publicly traded cancer immunotherapy company. The combined entity, now named Calidi Biotherapeutics, Inc., will commence trading on the NYSE American under ticker symbols “CLDI” and “CLDI WS” on September 13.

The merger provides Calidi with gross proceeds of approximately $28 million before expenses and debt repayments. This consists of $25 million raised in a concurrent private offering, $1 million in cash from FLAG’s trust, and $2 million in PIPE and non-redemption agreements.

Founded in 2014, Calidi is developing first-in-class immunotherapies using allogeneic stem cells to deliver targeted cancer treatments. The SPAC deal enables the company to continue advancing its pipeline as a publicly listed firm.

Calidi’s lead candidates CLD-101 and CLD-201 leverage proprietary platforms called NeuroNova and SuperNova. Both utilize allogeneic stem cells loaded with oncolytic viruses that directly infect and kill tumor cells.

CLD-101, which employs neural stem cells, is currently in a Phase 1 trial for recurrent high-grade glioma brain tumors. Interim data is expected in 2024. CLD-201 uses mesenchymal stem cells to treat advanced solid tumors, with a Phase 1/2 study slated for 2024.

Take a moment to take a look at PDS Biotechnology, a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies.

According to Calidi CEO Allan Camaisa, the IPO “will allow us to push the boundaries of cell-based virotherapies and continue to research novel ways to eradicate cancer.”

SPACs have become an increasingly popular alternative to traditional IPOs in the biotech sector. Also known as “blank check companies”, SPACs raise capital through an IPO and then merge with a private entity to take it public. This allows the operating company to avoid some of the uncertainty associated with a traditional public debut.

First Light Acquisition Group, led by CEO Tom Vecchiolla, raised $172.5 million in its own IPO in May 2021. The team then sought a merger target that could benefit from the injection of public capital. They ultimately settled on clinical-stage Calidi and its novel immunotherapy approach.

In addition to the SPAC proceeds, Calidi has secured a $10 million forward purchase agreement from several institutional investors. It also intends to execute a $50 million purchase agreement with Lincoln Park Capital Fund.

Between its strengthened balance sheet and non-dilutive financing options, Calidi believes it now has the runway to advance its programs into 2025 without need for further equity funding.

According to Vecchiolla, “We are excited to see Calidi continue to grow as they transition into a public company and look forward to their clinical pursuit of new treatment options for patients everywhere in need.”

The merger completes Calidi’s transformation into a publicly traded company. With shares soon to start trading on the NYSE American under ticker “CLDI”, the company is poised to continue developing its promising immunotherapy candidates for cancer patients in need of new treatment options.

Rocket Shares Soar as FDA Aligns on Danon Disease Gene Therapy Trial

Posted on by slightbourne@noblecapitalmarkets.com

Rocket Pharmaceuticals (NASDAQ: RCKT) announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the design of a global pivotal Phase 2 trial for its gene therapy RP-A501 in Danon Disease. This marks an important milestone on the path to delivering a potentially transformative treatment for this devastating inherited cardiomyopathy.

Danon Disease is caused by genetic mutations in the LAMP2 gene leading to fatal heart failure. There are currently no approved therapies. The disease affects an estimated 15,000 to 30,000 patients in the U.S. and Europe, taking the lives of most males by age 20 and females by 40. RP-A501 aims to be the first-ever therapy to change the course of Danon Disease.

The news sent Rocket’s stock price soaring 34% to $20.46 in after-hours trading on Tuesday, as investors welcomed the positive regulatory update.

The planned Phase 2 pivotal trial will be a global, single-arm, open label study enrolling 12 Danon Disease patients. This includes a pediatric safety lead-in of 2 patients. All participants will receive a dose of 6.7 x 1013 GC/kg of RP-A501 delivered through intravenous infusion.

To support potential accelerated approval by the FDA, the co-primary efficacy endpoints at 12 months are LAMP2 protein expression and reduction in left ventricular mass, a key measure of heart damage. Expression of LAMP2, which is deficient in Danon patients, and decreased cardiac hypertrophy would signal RP-A501 is restoring cardiac function at its root genetic cause.

Take a moment to look at Ocugen Inc., a biotechnology company focused on discovering, developing and commercializing novel gene and cell therapies and vaccines.

Secondary trial endpoints consist of the biomarker troponin, heart failure questionnaires, functional classification, event-free survival, and safety. These could support eventual full approval. A concurrent global natural history study will provide an external control arm for comparison.

According to Rocket CEO Dr. Gaurav Shah, “RP-A501 has the potential to restore normal cardiac function and provide a lifetime of benefit to patients with Danon Disease who have no other viable treatment options.” The company believes this pivotal trial design sets the most rapid path to deliver RP-A501 to patients in dire need.

Rocket’s gene therapy approach involves using an engineered virus called AAV9 to deliver a functional LAMP2 gene into patients’ heart cells. The gene insert encodes LAMP2B, a key protein involved in the cellular recycling process called autophagy. Restoring LAMP2B in the heart could potentially halt the accumulation of cellular debris and improve cardiac structure and function.

The company has already manufactured sufficient high-quality RP-A501 drug product at its in-house cGMP facility to supply the full pivotal Phase 2 trial. Qualified potency assays are also in place to support quality control and regulatory compliance.

Looking ahead, Rocket plans to file a Clinical Trial Application in the EU in Q3 2023 to initiate study activities abroad. The company also recently secured an ICD-10 code from the Centers for Medicare and Medicaid Services for LAMP2 deficiency, which will support diagnostic efforts.

This alignment with the FDA represents a major achievement for both Rocket and the Danon Disease community. RP-A501 would be the first-ever approved treatment for this deadly cardiovascular condition. The gene therapy aims to be a one-time curative infusion that could provide transformative and lifelong benefits to affected patients.

Beyond Danon Disease, Rocket believes this program paves the way for developing genetic medicines against other inherited heart diseases. Cardiac gene therapy has long faced hurdles, but the company is forging a new path for treating genetic cardiovascular conditions at their root.

With the pivotal Phase 2 trial design now locked in, Rocket can move full speed ahead on enrolling patients and gathering data to support potential approval. The company expects to release initial results from the trial evaluating RP-A501 in 2024. This could lead to a approved treatment for Danon Disease in the not too distant future—bringing tremendous hope to patients and families affected by this devastating illness.

Tonix Pharmaceuticals announces poster presentation involving TNX-1700 in preclinical colorectal cancer models at the 7th International Cancer Immunotherapy Conference 2023.

Release – PDS Biotech to Host Key Opinion Leader Roundtable Addressing Current and Future Treatments for Recurrent/Metastatic HPV-Positive HNSCC and the Potential Application of PDS0101 on September 27, 2023

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

Company to provide updated VERSATILE-002 data on combination of PDS0101 and KEYTRUDA®

PRINCETON, N.J., Sept. 13, 2023 (GLOBE NEWSWIRE) —

Company to provide updated VERSATILE-002 data on combination of PDS0101 and KEYTRUDA®

PRINCETON, N.J., Sept. 13, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (PDS Biotech or 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 it will host a Key Opinion Leader (KOL) Roundtable on Addressing Current and Future Treatments for Recurrent/Metastatic Human Papillomavirus (HPV)-Positive Head and Neck Squamous Cell Carcinoma (HNSCC) and the Potential Application of PDS0101, including an update from the Phase 2 VERSATILE-002 trial, for analysts, investors and the scientific community from 8:00 – 9:00 AM EDT on Wednesday, September 27, 2023.

The Company-sponsored event will focus on current and potential treatments for HPV16-positive head and neck cancer, including the emerging use of circulating tumor DNA (ctDNA), and unmet needs of this growing patient population. The event will also include a presentation on updated data from the VERSATILE-002 study of the combination of KEYTRUDA® (pembrolizumab) and PDS0101, PDS Biotech’s novel investigational HPV-targeted immunotherapy, as well as the design for the planned VERSATILE-003 Phase 3 clinical trial due to be initiated in the fourth quarter of 2023. The event will be moderated by PDS Biotech’s Chief Medical Officer, Dr. Lauren V. Wood, and will feature presentations from the following head and neck cancer KOLs:

  • Dr. Glenn Hanna, Assistant Professor, Harvard University and Medical Oncologist, Dana-Farber Cancer Institute
  • Dr. John Kaczmar, Associate Professor, Medical University of South Carolina
  • Dr. Ricard Mesía, Head of Medical Oncology, Catalan Institute of Oncology
  • Dr. Katharine Price, Associate Professor, Oncology Head and Neck Disease Group, Mayo Clinic Comprehensive Cancer Center

Registration of PDS Biotech’s KOL Roundtable is now open, and a live webcast of the event will be available online in the investor relations section of the Company’s website at https://www.pdsbiotech.com/index.php/investors. A replay will be available for 90 days following the webcast.

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. 

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 VERSATILE-003

VERSATILE-003 is a randomized, controlled Phase 3 trial evaluating the safety and efficacy of PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) compared with KEYTRUDA® monotherapy as the control arm. The primary end point for the study is patient overall survival (OS). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the Food and Drug Administration in June 2022.  

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® and Infectimune® are registered trademarks 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 Contacts:
Dave Schemelia
Tiberend Strategic Advisors
Phone: +1 (609) 468-9325
Email: dschemelia@tiberend.com

Eric Reiss
Tiberend Strategic Advisors
Phone: +1 (802) 249-1136
Email: ereiss@tiberend.com

(Nasdaq: PDSB) (PDS Biotech or 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 it will host a Key Opinion Leader (KOL) Roundtable on Addressing Current and Future Treatments for Recurrent/Metastatic Human Papillomavirus (HPV)-Positive Head and Neck Squamous Cell Carcinoma (HNSCC) and the Potential Application of PDS0101, including an update from the Phase 2 VERSATILE-002 trial, for analysts, investors and the scientific community from 8:00 – 9:00 AM EDT on Wednesday, September 27, 2023.

The Company-sponsored event will focus on current and potential treatments for HPV16-positive head and neck cancer, including the emerging use of circulating tumor DNA (ctDNA), and unmet needs of this growing patient population. The event will also include a presentation on updated data from the VERSATILE-002 study of the combination of KEYTRUDA® (pembrolizumab) and PDS0101, PDS Biotech’s novel investigational HPV-targeted immunotherapy, as well as the design for the planned VERSATILE-003 Phase 3 clinical trial due to be initiated in the fourth quarter of 2023. The event will be moderated by PDS Biotech’s Chief Medical Officer, Dr. Lauren V. Wood, and will feature presentations from the following head and neck cancer KOLs:

  • Dr. Glenn Hanna, Assistant Professor, Harvard University and Medical Oncologist, Dana-Farber Cancer Institute
  • Dr. John Kaczmar, Associate Professor, Medical University of South Carolina
  • Dr. Ricard Mesía, Head of Medical Oncology, Catalan Institute of Oncology
  • Dr. Katharine Price, Associate Professor, Oncology Head and Neck Disease Group, Mayo Clinic Comprehensive Cancer Center

Registration of PDS Biotech’s KOL Roundtable is now open, and a live webcast of the event will be available online in the investor relations section of the Company’s website at https://www.pdsbiotech.com/index.php/investors. A replay will be available for 90 days following the webcast.

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. 

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 VERSATILE-003

VERSATILE-003 is a randomized, controlled Phase 3 trial evaluating the safety and efficacy of PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab) compared with KEYTRUDA® monotherapy as the control arm. The primary end point for the study is patient overall survival (OS). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the Food and Drug Administration in June 2022.  

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® and Infectimune® are registered trademarks 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 Contacts:
Dave Schemelia
Tiberend Strategic Advisors
Phone: +1 (609) 468-9325
Email: dschemelia@tiberend.com

Eric Reiss
Tiberend Strategic Advisors
Phone: +1 (802) 249-1136
Email: ereiss@tiberend.com

Release – Ocugen Announces Positive Clinical Study Update From The Phase 1/2 Trial Of OCU400, A Modifier Gene Therapy Product Candidate, For The Treatment Of Retinitis Pigmentosa (RP) And Leber Congenital Amaurosis (LCA)

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

September 13, 2023

PDF Version

  • Favorable safety and tolerability profile of OCU400 investigational drug product in RP and LCA subjects to date
  • Clinical study update suggests continued positive trends in Best-Corrected Visual Acuity (BCVA) and Multi-Luminance Mobility Testing (MLMT), as well as positive trends in Low-Luminance Visual Acuity (LLVA) among treated eyes
  • 83% (10/12) of subjects demonstrated stabilization or improvement in treated eye either on BCVA or LLVA or MLMT scores from baseline
  • Notably, 86% (6/7) of RHO mutation subjects experienced either stabilization of or increase in MLMT scores from baseline including a subset of 29% (2/7) that demonstrated a 3 Lux luminance level improvement

MALVERN, Pa., Sept. 13, 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 announced a clinical study update for Retinitis Pigmentosa (RP) participants treated in the Phase 1/2 trial to assess the safety and efficacy of OCU400 for RP associated with NR2E3 and Rhodopsin (RHO) mutations and Leber congenital amaurosis (LCA) with mutation(s) in the CEP290 gene. This clinical study update is an extension of results provided by Ocugen on April 14, 2023, and includes additional subjects from the high dose group. The Company believes that OCU400—Ocugen’s therapeutic approach, utilizing a proprietary modifier gene therapy platform—has the potential to be a gene-agnostic therapeutic for RP and LCA patients with inherited retinal degeneration.

“This clinical study update supports our vision to help change the lives of patients suffering from inherited retinal diseases,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We remain dedicated to our mission of pioneering breakthroughs in biotechnology and believe that OCU400 has the potential to have an impact on the future treatment of patients with RP and LCA.” 

This Phase 1/2 trial is a multicenter, open-label, dose ranging study. A total of 18 subjects with vision impairment due to RP associated with RHO and NR2E3 gene mutations received a unilateral subretinal injection of either a low dose (1.66 x 1010 vg/mL), medium dose (3.33 x 1010 vg/mL), or high dose (1.66 x 1011 vg/mL) of OCU400. The study profile included a diverse group of subjects aged 18-77 years old, with varied disease stages, racial and ethnic profiles, medical histories, and mutation subgroups. Ocugen further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.

Inherited retinal diseases (IRDs) such as RP and LCA encompass a group of genetic disorders that affect the retina, the light-sensitive tissue at the back of the eye. These diseases often lead to a gradual loss of vision over time and can ultimately result in blindness. Stabilization of vision is crucial for patients with IRDs due to the progressive and degenerative nature of these conditions.

Preserving remaining vision, slowing disease progression, or improving the vision can significantly impact patients’ quality of life. It not only enhances the quality of life for affected individuals but also provides hope for future treatments that may ultimately lead to vision restoration. Comprehensive care, early diagnosis, and access to emerging therapies are essential components of a strategy to stabilize vision in IRD patients.

“I am gratified to see the progress we have made in our pursuit of developing a novel gene-agnostic therapy for RP and LCA. Our team’s unwavering dedication to advancing modifier gene therapy research demonstrated positive preliminary clinical results that offer renewed hope to patients and their families. We remain resolute in our mission and vision to bring a bright future to those with inherited or age-related retinal diseases through courageous innovation and unwavering determination,” said Dr. Arun Upadhyay, Chief Scientific Officer, Head of Research, Development and Medical at Ocugen.

This clinical study update is based on the currently available data from Phase 1 (dose-escalation: Cohort 1, 2 and 3) and the Phase 2 (open enrollment) portion of the study. The exploratory efficacy update includes data for 12 subjects who have completed a minimum of 6-month follow up. The data set comprised of 2 subjects [Cohort 1] with 12-month follow-up, 5 subjects [N=2 from Cohort 1 and N=3 from Cohort 2] with 9-month follow-up, and 5 subjects [N=2 from Cohort 3 and N=3 from Open Enrollment/Phase 2] with 6-month follow-up.

“It is an important steppingstone for Ocugen and its mission to help the nearly 1.6 million patients affected by RP and LCA worldwide. For those suffering from these IRDs, this clinical trial update provides hope. It is encouraging to see a favorable safety and tolerability profile and positive efficacy readout for OCU400 in RP patients,” said Dr. Lejla Vajzovic, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine and leader in gene-therapy research.

Key efficacy outcomes from 12 subjects demonstrated:
BCVA:

  • 83% (10/12) of subjects demonstrated stabilization or improvements in treated eyes in BCVA scores from baseline
  • 42% (5/12) of OCU400 treated eyes experienced 4-letter improvement and 33% (4/12) treated eyes experienced 7-letter improvement in BCVA from baseline
  • 57% (4/7) of RHO subjects’ treated eyes experienced 4-letter improvement and 43% (3/7) treated eyes experienced 7-letter improvement in BCVA scores from baseline

LLVA:

  • 83% (10/12) of subjects demonstrated stabilization or improvement in treated eyes in LLVA scores from baseline
  • 42% (5/12) of OCU400 treated eyes experienced 5-letter improvement (1 line) in LLVA from baseline, with 25% (3/7) increasing by 10 letters (2 lines)
  • 43% (3/7) of RHO subjects experienced 5-letter improvement (1 line) in treated eyes in LLVA scores from baseline, among which 29% (2/7) increased by 10 letters (2 lines)

MLMT:

  • 75% (9/12) of subjects demonstrated stabilization or improvement in treated eyes in MLMT scores from baseline
  • 33% (4/12) of subjects in the low, medium, and high dose cohorts experienced at least 1 Lux luminance level improvement from baseline in treated eyes, among which 17% (2/12) increased by 3 Lux luminance levels
  • 86% (6/7) of RHO subjects experienced either stabilization or increases in MLMT scores from baseline, among which 29% (2/7) improved by 3 lux levels

“The RHO mutation affects more than 10,000 people in the US,” said Dr. David Birch, Scientific Director, Retina Foundation of the Southwest and Principal investigator of the study. “In my view, the clinical study update supports the gene-agnostic mechanism of action of OCU400 in RHO patients. The improvements in BCVA, LLVA and MLMT in this patient population are very exciting and encouraging because stabilization alone could be considered as a treatment benefit.”

The clinical study update from the Phase 1/2 clinical trial demonstrated that OCU400 continued to be generally safe and well-tolerated in subjects across different mutations and dose levels. There were no serious adverse events (SAEs) related to the investigational product in the low and medium-dose cohorts. In the high-dose and open-enrollment cohorts, SAEs were reported for two subjects. Adverse events were mostly deemed related to the surgical procedure and resolved within a few days to weeks.

“The clinical study update released by Ocugen appears to have a tangible biological impact on Retinitis Pigmentosa associated with NR2E3 and RHO mutations,” said Dr. David Boyer, Clinical Professor of Ophthalmology USC/Keck School of Medicine Los Angeles, CA and Partner, Retina Vitreous Associates Medical Group. “These findings may indicate a huge step forward in the way we approach and treat this condition. We remain optimistic and eager to continue the trial and understand the full potential of OCU400.”

Ocugen will continue to monitor long-term safety and efficacy data from the treated patients and provide additional updates.

A webcast and conference call will take place today at 8:30 a.m. ET:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 7803227

Webcast: Available on the events section of the Ocugen investor site

About Modifier Gene Therapy
Modifier gene therapy is designed to fulfill unmet medical needs related to retinal diseases, including IRDs, such as RP, LCA, and Stargardt disease, as well as dry AMD. Our modifier gene therapy platform is based on the use of Nuclear hormone receptors (NHRs), master gene regulators, which have the potential to restore homeostasis — the basic biological processes in the retina. Unlike single-gene replacement therapies, which only target one genetic mutation, we believe that our modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and to address complex diseases that are potentially caused by imbalances in multiple gene networks. Currently Ocugen has three modifier gene therapy programs OCU400 (RP, LCA), OCU410 (dry AMD), OCU410ST (Stargardt disease).

About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter 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, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trial results, and anticipated timing of clinical trial updates and regulatory interactions. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward- looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in the Phase 1/2 clinical trial or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Release – MustGrow Announces Investor Relations Agreement with Harbor Access

Posted on by aweinsoff@channelchek.com

Research News and Market Data on MGROF

SASKATOON, Saskatchewan, Canada, September 12, 2023 – MustGrow Biologics Corp. (TSXV: MGRO) (OTC: MGROF) (FRA: 0C0) (the “Company” or “MustGrow”) is pleased to announce the engagement of Harbor Access LLC (“Harbor Access”) to perform investor relations services and activities, as defined in accordance with the policies of the TSX Venture Exchange (“TSXV”) and applicable securities laws.

“In connection with recent Company announcements including our significant project expansion with Janssen PMP, it is timely to refresh our investor relations and communications efforts, ” said Corey Giasson, CEO of MustGrow. “We believe that having an investor relations firm with broad experience in both the Canadian and US markets can provide strong support in creating wider awareness and expanding our investor base.”

“We are delighted to be working with Corey and the MustGrow team to enable more investors to be familiar with this unique and compelling investment opportunity given the growing importance of securing the global food supply,” stated Jonathan Paterson, Founder of Harbor Access

Pursuant to the agreement with Harbor Access, the Company will pay a cash fee of USD$8,000 per month over an initital period of 12 months, subject to renewal. Harbor Access does not control any common shares in the Company.

———

About MustGrow

MustGrow is an agriculture biotech company developing organic biocontrol, soil amendment and biofertility products by harnessing the natural defense mechanism and organic materials of the mustard plant to sustainably protect the global food supply and help farmers feed the world. MustGrow and its leading global partners — Janssen PMP (pharmaceutical division of Johnson & Johnson), Bayer, Sumitomo Corporation, and Univar Solutions’ NexusBioAg — are developing mustard-based organic solutions to potentially replace harmful synthetic chemicals. Concurrently, with new formulations derived from food- grade mustard, the Company is pursuing the adoption and use of its technology in the soil amendment and biofertily markets. Over 150 independent tests have been completed, validating MustGrow’s safe and effective approach to crop and food protection and yield enhancements. Pending regulatory approval, MustGrow’s patented liquid products could be applied through injection, standard drip or spray equipment, improving functionality and performance features. Now a platform technology, MustGrow and its global partners are pursuing applications in several different industries from preplant soil treatment and weed control, to postharvest disease control and food preservation, to soil amendment and biofertility. MustGrow has approximately 50.1 million basic common shares issued and outstanding and 56.3 million shares fully diluted. For further details, please visit www.mustgrow.ca.

Contact Information

Corey Giasson
Director & CEO
Phone: +1-306-668-2652
info@mustgrow.ca

About Harbor Access 

Harbor Access represents and advises micro to mega cap companies. The Company has a measured approach to creating a capital markets communications program, which helps optimize their clients’ visibility. Harbor Access brings extensive global experience and professionalism to each client and customizes every solution to meet their clients’ goals.

For more information about Harbor Access, please visit www.harbor-access.com

Contact Information

Jonathan Paterson
Founder & Managing Partner
Phone: +1-475-477-9401
Jonathan.Paterson@Harbor-access.com

MustGrow Forward-Looking Statements

Certain statements included in this news release constitute “forward-looking statements” which involve known and unknown risks, uncertainties and other factors that may affect the results, performance or achievements of MustGrow.

Generally, forward-looking information can be identified by the use of forward-looking terminology such as “plans”, “expects”, “is expected”, “budget”, “estimates”, “intends”, “anticipates” or “does not anticipate”, or “believes”, or variations of such words and phrases or statements that certain actions, events or results “may”, “could”, “would”, “might”, “occur” or “be achieved”. Examples of forward-looking statements in this news release include, among others, statements MustGrow makes regarding creating wider awareness and expanding its investor base.

Forward-looking statements are subject to a number of risks and uncertainties that may cause the actual results of MustGrow to differ materially from those discussed in such forward-looking statements, and even if such actual results are realized or substantially realized, there can be no assurance that they will have the expected consequences to, or effects on, MustGrow. Important factors that could cause MustGrow’s actual results and financial condition to differ materially from those indicated in the forward-looking statements include market receptivity to investor relations activities as well as those risks described in more detail in MustGrow’s Annual Information Form for the year ended December 31, 2022 and other continuous disclosure documents filed by MustGrow with the applicable securities regulatory authorities which are available at www.sedar.com. Readers are referred to such documents for more detailed information about MustGrow, which is subject to the qualifications, assumptions and notes set forth therein.

This release does not constitute an offer for sale of, nor a solicitation for offers to buy, any securities in the United States.

Neither the TSXV, nor their Regulation Services Provider (as that term is defined in the policies of the TSXV), nor the OTC Markets has approved the contents of this release or accepts responsibility for the adequacy or accuracy of this release.

© 2023 MustGrow Biologics Corp. All rights reserved.

Release – Ocugen To Host Virtual Investor & Analyst Event on September 13, 2023

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

September 12, 2023

PDF Version

COMPANY TO SHARE UPDATED RESULTS FROM ONGOING PHASE 1/2 TRIAL OF OCU400 FOR THE TREATMENT OF RETINITIS PIGMENTOSA AND LEBER CONGENITAL AMAUROSIS

MALVERN, Pa., Sept. 12, 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 announced that it will host an Investor and Analyst Event on September 13, 2023 at 8:30 a.m. ET. During the webcast and conference call, members of the Ocugen leadership team and key opinion leaders will discuss updated results from the Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA).

The event will feature:

Shankar Musunuri, PhD, MBA, Chairman, CEO and Co-founder, Ocugen

Arun Upadhyay, PhD, Chief Scientific Officer, Head of Research, Development & Medical, Ocugen

Huma Qamar, MD, MPH, Head of Clinical Development and Medical Affairs, Ocugen

David Birch, PhD, Scientific Director, Retina Foundation of the Southwest, Principal investigator of the study

Byron L. Lam, MD, Mark J. Daily Professor, Bascom Palmer Eye Institute, University of Miami, Principal investigator of the study

Lejla Vajzovic, MD, FASRS, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine and leader in gene-therapy research

Webcast and Conference Call Details

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 7803227

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 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 Twitter and LinkedIn.

Cautionary Note on Forward-Looking Statements
This presentation 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 our clinical development activities and related anticipated timelines. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this presentation speak only as of the date of this presentation. Except as required by law, we assume no obligation to update forward-looking statements contained in this presentation whether as a result of new information, future events, or otherwise, after the date of this presentation.

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

Mainz Biomed Reports Strong ColoAlert Data, Identifies Biomarkers for FDA Trial

Posted on by slightbourne@noblecapitalmarkets.com

Mainz Biomed (NASDAQ:MYNZ) announced highly positive results this week from its ColoFuture clinical trial evaluating the integration of novel mRNA biomarkers into its ColoAlert screening test for colorectal cancer. The results demonstrated ColoAlert’s strong performance for detecting colorectal cancer and advanced adenomas, while also identifying promising biomarkers to further improve early detection capabilities.

The ColoFuture study is a multi-center international trial assessing whether integrating recently acquired mRNA biomarkers from Université de Sherbrooke can enhance ColoAlert’s sensitivity and specificity profile. ColoAlert is Mainz’s flagship product, a simple yet highly accurate home-based test using stool samples to detect colorectal cancer and advanced precancerous adenomas.

The product is already commercialized across Europe and select international territories. However, ColoFuture aimed to identify ways to further extend ColoAlert’s technical capabilities in preparation for an upcoming U.S. clinical trial that could lead to FDA approval.

Interim analysis from the ColoFuture trial included 220 subjects across centers in Germany, Norway and Denmark. On the primary endpoints for colorectal cancer detection, ColoAlert achieved 94% sensitivity and 97% specificity after integrating the novel mRNA biomarkers.

For identifying advanced adenomas, a key precursor to cancer, the updated test demonstrated 81% sensitivity. According to Mainz Biomed CEO Guido Baechler, “The power to detect lesions in a pre-cancerous stage can change the entire CRC diagnostic landscape. If advanced adenomas are identified early, they are curable.”

The positive data catalyzed strong trading volume as Mainz Biomed’s stock price rose 15% on over 1.5 million shares traded. The market enthusiastically welcomed the results.

The mRNA biomarkers evaluated in ColoFuture were specifically selected from research at Université de Sherbrooke. Published analysis of the biomarkers showed ability to detect signals from patients with either colorectal cancer or advanced adenomas. Mainz Biomed acquired these biomarker rights in January 2022.

By treating patients before polyps progress to cancer, integrating these biomarkers into ColoAlert could help prevent colorectal cancer altogether. This could greatly improve patient outcomes and reduce the burden of this deadly disease.

The positive data provides validation of ColoAlert’s accuracy as a non-invasive screening tool. It also gives Mainz Biomed multiple novel mRNA biomarkers to integrate into its upcoming U.S. clinical trial, dubbed ReconAAsense, which could support FDA approval under the PMA pathway.

According to Baechler, “As we look forward to publishing and presenting the full dataset, we eagerly await the outcome from our ReconAAsense clinical trial which remains on track to report results in Q4 of this year.”

With colorectal cancer remaining a leading cause of cancer deaths, early detection is critical. ColoAlert offers a simple, at-home solution that people can easily incorporate into routine wellness screenings. The new biomarkers identified in ColoFuture could make the test accessible to even more patients.

Mainz Biomed continues to spearhead innovation in the field, leveraging the latest advances in genetics to improve detection. The impressive ColoFuture results provides further validation of ColoAlert’s accuracy, while also charting a path forward to commercialization in the U.S.

With pivotal FDA trial data on the horizon, Mainz Biomed is positioned to disrupt the market, offering an easy yet cutting-edge approach to potentially save lives through early colorectal cancer detection.

Take a look at more biotechnology companies by viewing Robert LeBoyer’s coverage list.

Release – GeoVax Presents Corporate Update at the H.C. Wainwright 25th Annual Global Investment Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

Updates Presented for Gedeptin® and GEO-CM04S1 Phase 2 Clinical Trials

ATLANTA, GA, September 12, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that its Chairman and CEO, David Dodd, presented a company overview during the H.C. Wainwright 25th Annual Global Investment Conference on September 11. A webcast replay of Mr. Dodd’s presentation can be viewed here.

During his presentation, Mr. Dodd reviewed the Company’s four ongoing Phase 2 clinical trials:

GEO-CM04S1 (Next-Generation COVID-19 Vaccine)

  • As a primary vaccine in immunocompromised patients (with hematologic cancers receiving cell transplants or CAR-T therapy). gov Identifier: NCT04977024.
  • As a booster vaccine in immunocompromised patients with chronic lymphocytic leukemia (CLL), a recognized high-risk group for whom current mRNA vaccines and monoclonal antibody (MAb) therapies appear inadequate relative to providing protective immunity. ClinicalTrials.gov Identifier: NCT05672355.
  • As a booster vaccine for healthy patients who have previously received the Pfizer or Moderna mRNA vaccine. gov Identifier: NCT04639466.

Gedeptin® (Gene Therapy for Solid Tumors)

  • Evaluating the safety and efficacy of repeat cycles of Gedeptin therapy in patients with recurrent head and neck cancers whose tumor(s) are accessible for injection and who have no curable treatment options.gov Identifier: NCT03754933.

Commenting on the GEO-CM04S1 clinical program, Mr. Dodd said, “Our next-generation COVID-19 vaccine is designed to provide critically needed protection to those individuals with depleted immune systems, and who are inadequately protected by the currently authorized vaccines and monoclonal antibody therapies. Using our MVA platform as its backbone, our vaccine candidate is “variant-agnostic” and we expect it to provide broader, more durable protection than existing authorized vaccines. We anticipate multiple significant catalyst events during the remainder of 2023. We recently completed patient enrollment in the healthy booster trial, and anticipate completion of enrollment in our CLL trial soon, while also accelerating enrollment in the cell transplant trial through multi-site expansion. We also believe our vaccine to be an excellent example of next-generation vaccines, providing more robust and durable protection, especially for immunocompromised individuals who don’t adequately respond to the currently authorized COVID-19 vaccines or monoclonal antibody therapies.”

Regarding the Gedeptin program, Mr. Dodd stated, “We expect to soon complete the first stage of this study, which is being funded in part by the U.S. Food and Drug Administration (FDA) pursuant to its Orphan Products Clinical Trials Grants Program, and then initiate an expanded Phase 2 trial focused on expedited FDA registration. We are excited about the potential for the Gedeptin “tumor-agnostic” platform, which may include expanded development, both as monotherapy and combination therapy in conjunction with therapies such as immune checkpoint inhibitors. Possible expansion indications include breast, prostate, and pancreatic cancers, among other solid tumors. This program also offers multiple opportunities for global strategic collaborations and partnerships.”

Mr. Dodd’s concluding comments focused on GeoVax’s advancements made in its MVA manufacturing process in transforming to a high-yield, high-capacity continuous cell line system, as well as the Company’s efforts for achieving expedited registration for the first U.S.-sourced vaccine against Mpox and smallpox. Mr. Dodd said, “Our progress in developing a continuous avian cell line system is nothing short of transformative for manufacturing our MVA-based vaccines and immunotherapies, and we recently partnered with Advanced Bioscience Laboratories, Inc. to support the production of our MVA-based products through late-stage development and eventual commercialization. Additionally, the recent expansion of our GEO-CM04S1 rights to include development for Mpox and smallpox adds to other rights we previously secured from the NIH covering preclinical, clinical, and commercial uses of the NIH-MVA. This provides a compelling opportunity to leverage our MVA-based vaccine expertise and help expand the global public health supply options available for the worldwide public health threats posed by SARS-CoV-2, Mpox, and smallpox.”

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. 

Contact:

GeoVax Labs, Inc.

investor@geovax.com

678-384-7220

Release – ZyVersa Therapeutics Announces Research Published in Frontiers in Immunology Reinforcing IC 100’s Rationale for Inhibiting Multiple Inflammasomes to Control Inflammation in Various Inflammatory Diseases

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Sep 12, 2023

PDF Version

  • Activation of multiple inflammasome pathways contributes to pathological inflammation in neurodegenerative (“CNS”) diseases, such as amyotrophic lateral sclerosis (“ALS”)
  • This study showed that NLRP3 inhibition alone was inadequate to address CNS inflammation in a mouse model of ALS
  • ZyVersa is developing Inflammasome ASC Inhibitor IC 100, designed to inhibit up to 12 different inflammasomes (including NLRP3 inflammasomes) and their associated ASC specks to control damaging inflammation in CNS and non-CNS inflammatory diseases

WESTON, Fla., Sept. 12, 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 a paper in the peer-reviewed journal, Frontiers in Immunology, highlighting potential limitations of NLRP3 inhibition in attenuating CNS Inflammation associated with activation of multiple inflammasome pathways. This paper supports ZyVersa’s rationale for targeting inflammasome ASC with IC 100 to inhibit multiple inflammasome pathways, not just NLRP3, to control inflammation in various inflammatory diseases.

In the paper titled, “Divergent functional outcomes of NLRP3 blockade downstream of multi-inflammasome activation: therapeutic implications for ALS,” the authors conducted studies in various human cell lines and in an animal model of ALS. Data demonstrate that NLRP3 inhibition alone is insufficient to attenuate proinflammatory cytokine release and inflammatory cell death associated with activation of multiple inflammasome pathways. Likewise, NLRP3 inhibition alone did not ameliorate spinal cord inflammation in an ALS model.

The authors stated, “we hypothesize that strategies that impact multiple inflammasome pathways may hold more promise in CNS disorders like ALS where multiple inflammasomes are dysregulated.” To read the article Click Here.

“The research published in Frontiers in Immunology reinforces the need to inhibit more than the NLRP3 inflammasome pathway to control inflammation associated with activation of multiple inflammasomes,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO and President. “ZyVersa’s Inflammasome ASC inhibitor IC 100 is designed to inhibit formation of multiple types of inflammasomes to attenuate initiation of the inflammatory cascade, and to inhibit ASC specks to attenuate perpetuation of damaging 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