Key Points: – FDA advisory panel recommends approval of arimoclomol for Niemann-Pick disease type C (NPC). – If approved, arimoclomol would be the first FDA-approved treatment for NPC in the US. – Final FDA decision expected by September 21, 2024.
In a significant development for patients with a rare and devastating brain disease, an FDA advisory panel has recommended approving arimoclomol, a drug developed by Zevra Therapeutics. This decision marks a potential turning point in the treatment of Niemann-Pick disease type C (NPC), a condition that currently lacks FDA-approved therapies in the United States.
NPC is a serious genetic disorder that impairs the body’s ability to process and transport fats, leading to their accumulation in various organs, including the brain. This buildup causes progressive neurological damage, severely impacting patients’ quality of life. The disease is caused by mutations in either the NPC1 or NPC2 genes, which are responsible for producing proteins involved in cellular cholesterol transport.
Arimoclomol’s journey to potential approval has been marked by setbacks and perseverance. In 2021, the FDA initially rejected the drug, requesting additional evidence of its efficacy. However, under the new ownership of Zevra Therapeutics (formerly KemPharma), arimoclomol has found new life. The company submitted a reinforced New Drug Application (NDA) with additional long-term data, which seems to have addressed the FDA’s previous concerns.
The FDA’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted 11 to 5 in favor of approving arimoclomol. This recommendation is based on a comprehensive review of clinical data, including results from a pivotal trial and a four-year open-label extension study. These studies demonstrated a decrease in the NPC Clinical Severity Scale (NPCCSS) score compared to placebo, indicating a meaningful clinical benefit for patients.
Arimoclomol works by inducing the heat shock response in cells, which helps to correct the protein misfolding that contributes to NPC. This novel approach has earned the drug several FDA designations, including orphan drug, fast track, breakthrough therapy, and rare pediatric disease status, underscoring its potential significance in treating this devastating condition.
If approved, arimoclomol would become the first FDA-approved treatment for NPC in the United States. Currently, US patients rely on off-label use of miglustat (Zavesca), which is approved for NPC in some European countries. The FDA’s final decision on arimoclomol is expected by September 21, 2024, the Prescription Drug User Fee Act (PDUFA) action date for the NDA.
The market implications of arimoclomol’s potential approval are substantial. GlobalData forecasts that the NPC drug market could reach $220 million by 2031 across the US, Germany, and the UK. This represents a significant opportunity for Zevra Therapeutics and, more importantly, a beacon of hope for NPC patients and their families.
Zevra’s CEO, Neil McFarlane, expressed confidence in arimoclomol’s clinical benefit and optimism about its path to approval. The company’s persistence in addressing the FDA’s initial concerns and providing robust long-term data has seemingly paid off, potentially bringing a much-needed treatment option to a patient population with limited choices.
This story underscores the complex and often challenging path of drug development for rare diseases. It highlights the importance of persistence and comprehensive clinical data in addressing regulatory concerns and ultimately bringing innovative treatments to patients in need. If approved, arimoclomol could significantly improve the lives of people with NPC, offering hope to a community that has long awaited an effective treatment option.
This post was written and published as a collaboration between the in-house editorial team at Benzinga and Tonix Pharmaceuticals Holding Corp. with financial support from Tonix. The two organizations work to ensure that any and all information contained within is true and accurate as of the date hereof to the best of their knowledge and research. This content is for informational purposes only and not intended to be investing advice.
CHATHAM, NJ / ACCESSWIRE / August 1, 2024 / When it comes to chronic pain conditions, fibromyalgia is among the most common, afflicting more than ten million people in the United States. Globally, 3-6% of the world’s population suffers from this chronic disease that has no cure. While it is more prevalent in women, men and children across every race can also be afflicted with fibromyalgia.
With so many people suffering from a disease that brings widespread pain, fatigue and cognitive issues, it’s no wonder the market for symptom treatment has seen steady growth over the years and is poised for more. By 2031, the fibromyalgia treatment market is projected to reach $3.86 billion growing at a CAGR of 3.3% between now and then.
Fibromyalgia is a Serious Disease
This form of chronic pain has become such a problem in the United States that the Food & Drug Administration (FDA) considers the disease to be a serious condition, which means it is a disease associated with morbidity that has a substantial impact on day-to-day functioning. After all, 70% of sufferers have difficulty with daily activities, 90% report poor sleep quality and 20% file disability claims.
Most treatments on the market today are a combination of medication and self-care focused on reducing symptoms and improving the quality of life. Unfortunately, due to dissatisfaction with the available treatment options, many patients (at the direction of their medical providers) turn to opioids to relieve their pain. As it stands, more people diagnosed with fibromyalgia are prescribed opioids than the bestselling FDA-approved drug duloxetine (generic Cymbalta). That’s concerning, given opioids can be addictive and with time, life-threatening.
Of patients prescribed opioids for chronic pain, 21% to 29% misuse them while an average of 8% to 12% develop opioid use disorder. A U.S. policy to address opioid use, which killed more than 100,000 Americans in 2022, is aimed at curtailing imports of fentanyl and synthetics. Many experts believe a concurrent and perhaps more effective strategy is to provide pain sufferers relief with non-addictive products.
Fast Track Granted to TNX-102 SL*
The FDA has granted Tonix Pharmaceuticals (NASDAQ:TNXP) a fully integrated biopharmaceutical company Fast Track designation for TNX-102 SL (cyclobenzaprine HCl sublingual tablets), its drug candidate for the management of fibromyalgia. Tonix says the designation validates that fibromyalgia is a serious condition and that TNX-102 SL, which has no known addictive properties, has the potential to address this unmet medical need.
The FDA’s Fast Track process is designed to facilitate development and expedite the review of therapies intended to treat serious conditions and address unmet medical needs to potentially get new treatments to patients sooner. Companies whose programs are granted Fast Track designation are eligible for more frequent interactions with the FDA during clinical development.
“The designation underscores the importance of addressing the unmet needs of fibromyalgia patients, who report dissatisfaction with current treatment options,” said Seth Lederman, M.D., CEO of Tonix Pharmaceuticals. “If approved by the FDA, we expect TNX-102 SL to become the first new pharmacotherapy for fibromyalgia in over 15 years.”
TNX-102 SL is a sublingual formulation of cyclobenzaprine hydrochloride designed to improve sleep quality rather than quantity, setting it apart from existing treatments, which fail to manage sleep disturbances that exacerbate fibromyalgia symptoms, the company says.
In recent Phase 3 trials, TNX-102 SL showed a statistically significant improvement in fibromyalgia pain with a p-value of 0.00005. Tonix reports that significant results were also seen in improving sleep quality, reducing fatigue and improving overall fibromyalgia symptoms and function. TNX-102 SL was well tolerated and the most common adverse events were transient sensations in the mouth corresponding with the disintegration of the tablet under the tongue.
New Drug Application Coming Soon
In addition to receiving Fast Track status, Tonix announced it is making progress on its new drug application (NDA), which it plans to submit to the FDA in the second half of this year. Coming out of pre-NDA meetings, Tonix said it is aligned with the FDA regarding the application for TNX-102 SL.
Tonix plans to request Priority Review designation for TNX-102 SL, and if granted, the FDA may accelerate the review of the new drug application.
“The NDA being prepared supports TNX-102 SL’s potential position as a first-line non-addictive therapy for fibromyalgia, indicated for long-term daily use at bedtime,” said Lederman.
*TNX-102 SL is an investigational new drug and has not been approved for any indication.
Company to initiate Phase 3 VERSATILE-003 trial in Q4 2024
Conference Call Today at 8:00 a.m. Eastern Time
PRINCETON, N.J., Aug. 01, 2024 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, today announced that it has received the official minutes from its meeting with the U.S. Food and Drug Administration (“FDA”) regarding next steps in its planned Phase 3 clinical trial of its Versamune® based investigational immunotherapy designed to stimulate a targeted T cell attack against HPV16-positive head and neck squamous cell carcinoma (“HNSCC”). The Company will host a conference call today at 8:00 a.m. ET to discuss details of the anticipated Phase 3 clinical trial of Versamune® HPV (formerly PDS0101) in this indication.
PDS Biotech presented the FDA with recent data from both the VERSATILE-002 study of Versamune® HPV + pembrolizumab, and the triple combination of Versamune® HPV + PDS01ADC + bintrafusp alfa. The Company also provided an updated design of the Phase 3 VERSATILE-003 trial of Versamune® HPV + pembrolizumab which included updated statistical endpoints based on recent and more mature survival data. PDS Biotech proposed the addition of a third arm to the study which would be a triple combination of Versamune® HPV + PDS01ADC + pembrolizumab. The first part of the study would therefore involve a dose optimization of PDS01ADC in the novel combination.
The FDA supported the strategy and development of the double and triple combinations. Also, the FDA requested additional safety analysis in the lead-in PDS01ADC dose optimization part of the study. To avoid potential delays in initiating the randomized trial, the FDA agreed that the dose optimization should be done separately and the registrational trial of the revised 2-arm double combination trial, VERSATILE-003, should proceed. The Versamune® HPV + pembrolizumab combination has received Fast Track designation.
“We appreciate the FDA’s support in the development of both the double and triple Versamune® HPV-based combinations. We are also pleased to have aligned on initiating the updated VERSATILE-003 study,” said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. “The VERSATILE-002 results have matured significantly and positively over the last year, allowing us to revise the statistical endpoints of the study to provide additional robustness to the study design. We continue to believe that the combination, based on encouraging survival, disease control response rates and safety has the potential to significantly advance the treatment of HPV16-positive HNSCC. Our goal now is to investigate Versamune® HPV + pembrolizumab’s potential as the first targeted immunotherapy for HPV16-positive HNSCC. The addition of PDS01ADC in the future has the potential to provide further clinical benefit to an effective targeted immunotherapy.”
Kirk Shepard, MD, Chief Medical Officer, continued, “We have contracted with a clinical research organization and the preparatory work is advancing to begin enrollment in the VERSATILE-003 Phase 3 clinical trial in first-line treatment of patients with recurrent or metastatic HPV16-positive HNSCC, with overall survival as the study’s primary endpoint. Our VERSATILE-003 trial has significant key opinion leader support, including from the investigators involved in VERSATILE-002, and we have lined up a significant number of the target sites that have indicated strong interest in participating in the trial.”
Conference Call Details Date: August 1, 2024 Time: 8:00 a.m. ET Dial-in: 1-877-704-4453 or 1-201-389-0920
Webcast Registration: Click Here Call MeTM Registration: Click Here (Available 15 minutes prior to call)
About PDS Biotechnology
PDS Biotechnology is a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines. The Company plans to initiate a pivotal clinical trial in 2024 to advance its lead program in advanced HPV16-positive head and neck squamous cell cancers. PDS Biotech’s lead investigational targeted immunotherapy Versamune® HPV is being developed in combination with a standard-of-care immune checkpoint inhibitor, and also in a triple combination including PDS01ADC, an IL-12 fused antibody drug conjugate (ADC), and a standard-of-care immune checkpoint inhibitor.
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 PDS01ADC, 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 PDS01ADC, 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; the Company’s ability to continue as a going concern; 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.
MALVERN, Pa., July 31, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Nasdaq: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced the pricing of its underwritten public offering of 30,434,783 shares of its common stock at an offering price of $1.15 per share of common stock for gross proceeds of $35 million, before deducting the underwriting discounts and commissions and other estimated offering expenses payable by Ocugen. In addition, Ocugen has granted to the underwriter a 30-day option to purchase up to 4,565,217 additional shares of its common stock at the public offering price, less the underwriting discounts and commissions. The offering is expected to close on or about August 2, 2024, subject to the satisfaction of customary closing conditions.
Ocugen intends to use the net proceeds from the offering for general corporate purposes, capital expenditures, working capital, and general and administrative expenses.
Titan Partners Group, a division of American Capital Partners, is acting as the sole book-running manager for the offering.
The offering is being made by Ocugen pursuant to a shelf registration statement on Form S-3 (File No. 333-278774) previously filed with the Securities and Exchange Commission (the “SEC”) on April 18, 2024, which became effective on May 1, 2024. The securities may be offered only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement was filed with the SEC on July 31, 2024. The final prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website. Copies of the preliminary prospectus supplement and the accompanying base prospectus, and when available, copies of the final prospectus supplement and the accompanying base prospectus relating to the offering, may be obtained by visiting the SEC’s website at www.sec.gov or by contacting Titan Partners Group, LLC, a division of American Capital Partners, LLC, 4 World Trade Center, 29th Floor, New York, NY 10007, by phone at (929) 833-1246 or by email at prospectus@titanpartnersgrp.com.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction.
AboutOcugen,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.
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. Such forward-looking statements within this press release include, without limitation, statements regarding Ocugen’s expectations regarding the timing of the completion of the offering and the anticipated use of proceeds. 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, such as market and other conditions. These and other risks and uncertainties are more fully described in our periodic filings with the 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 applicable law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, changed circumstances or otherwise, after the date of this press release.
Joe Gomes, CFA, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.
Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Another State. MustGrow announced the Company has received the Idaho State Department of Agriculture approval for TerraSante, allowing the product to commence sales in the state. The state follows the existing Organic OMRI Listed certifications in Oregon and Washington. Idaho now joins the list of states to authorize product sales, including the aforementioned Oregon and Washington and California.
Market Size. Idaho provided approximately $1.3 billion in crop production from potatoes in 2023, an increase from $1.2 billion in 2022, as potatoes are the state’s top crop. Other commodities the state provides includes barley, alfalfa hay, peppermint oil, and food trout. Overall, the state’s crop production was $3.3 billion in 2021.
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.
GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation COVID-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Gedeptin Will Be Tested As A Neoadjuvent. GeoVax announced the design of the Phase 2 trial testing Gedeptin in head and neck cancer. As planned, a Clinical Advisory Panel completed its data review from the clinical trials and made its design recommendation. The trial will test Gedeptin in combination with an immune checkpoint inhibitor (ICI) before surgery in head and neck squamous cell carcinoma (HNSCC) patients after first recurrence. The trial is expected to start in 1H25.
Trial Will Use A Single Cycle Of Gedeptin Before Surgery. The trial will enroll patients with HNSCC after first relapse. Patients will be treated with a single cycle of Gedeptin/fludarabine and a checkpoint inhibitor, followed by surgery. This adds Gedeptin’s intracellular activation of a chemotherapy agent to kill cancer cells with the immune response of the checkpoint inhibitor. The planned enrollment is 36 patients with a primary endpoint of pathological response rate.
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.
PRINCETON, N.J., July 31, 2024 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, today announced that the Company will host a conference call to provide a clinical program update on Thursday, August 1, 2024, at 8:00 a.m. ET.
Conference Call Details Date: August 1, 2024 Time: 8:00 a.m. ET Dial-in: 1-877-704-4453 or 1-201-389-0920
Webcast Registration: Click Here Call Me™ Registration: Click Here (Available 15 minutes prior to call)
About PDS Biotechnology PDS Biotechnology is a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines. The Company plans to initiate a pivotal clinical trial in 2024 to advance its lead program in advanced HPV16-positive head and neck squamous cell cancers. PDS Biotech’s lead investigational targeted immunotherapy Versamune® HPV is being developed in combination with a standard-of-care immune checkpoint inhibitor, and also in a triple combination including PDS01ADC, an IL-12 fused antibody drug conjugate (ADC), and a standard-of-care immune checkpoint inhibitor.
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 PDS01ADC, 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 PDS01ADC, 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; the Company’s ability to continue as a going concern; 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.
MALVERN, Pa., July 31, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Nasdaq: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that it has commenced an underwritten public offering (the “offering”) of its common stock. In addition, Ocugen expects to grant the underwriter a 30-day option to purchase up to an additional 15% of the aggregate number of shares of its common stock to be sold in the public offering at the public offering price, less the underwriting discounts and commissions. All of the shares of common stock in the offering are to be sold by Ocugen. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.
Ocugen intends to use the net proceeds of the proposed offering for general corporate purposes, capital expenditures, working capital, and general and administrative expenses.
Titan Partners Group, a division of American Capital Partners, is acting as the sole book-running manager for the proposed offering.
The offering is being made by Ocugen pursuant to a shelf registration statement on Form S-3 (File No. 333-278774) previously filed with the Securities and Exchange Commission (the “SEC”) on April 18, 2024, which became effective on May 1, 2024. The securities may be offered only by means of a prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and the accompanying base prospectus relating to and describing the proposed terms of the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and accompanying base prospectus relating to this offering may be obtained, when available, from Titan Partners Group LLC, a division of American Capital Partners, LLC, 4 World Trade Center, 29th Floor, New York, NY 10007, by phone at (929) 833-1246 or by email at prospectus@titanpartnersgrp.com.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of such state or jurisdiction.
AboutOcugen,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.
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. Such forward-looking statements within this press release include, without limitation, statements regarding market conditions, completion, timing, size and use of proceeds of the proposed offering and our intention to grant the underwriter a 30-day option to purchase additional shares. There can be no assurance that we will be able to complete the proposed public offering on the anticipated terms, or at all. 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, such as market and other conditions. These and other risks and uncertainties are more fully described in our periodic filings with the 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 applicable law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, changed circumstances or otherwise, after the date of this press release.
Company plans Phase 2 trial in first-recurrence head & neck cancer,
in combination with immune checkpoint inhibitor
Atlanta, GA, July 31, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a clinical-stage biotechnology company developing immunotherapies and vaccines against cancer and infectious diseases, today announced the next steps for the clinical development of its Gedeptin® cancer therapy, following a clinical advisory committee review.
GeoVax and a specially convened clinical advisory committee have completed a comprehensive review of the recently completed PNP-002 Phase 1b/2a trial, together with the previously completed PNP-001 Phase 1 trial. The Phase 1b/2a trial evaluated the safety and efficacy of repeat cycles of Gedeptin therapy in patients with advanced head and neck squamous cell carcinoma (HNSCC), with tumor(s) accessible for injection and no curable treatment options. The protocol assessed up to five treatment cycles, each consisting of three intratumoral injections of Gedeptin over two days followed by infusion of a prodrug, fludarabine phosphate, once a day for three days. This review concluded that Gedeptin demonstrated acceptable safety and efficacy to support continued development. No new safety signals were identified in the PNP-002 trial in heavily pretreated head & neck cancer patients. The therapy demonstrated sufficient tumor stabilization/reduction activity to support plans to advance clinical development of Gedeptin in an expanded Phase 2 clinical trial.
The primary goal of the planned Phase 2 trial will be to establish efficacy of neoadjuvant Gedeptin therapy combined with an immune checkpoint inhibitor in first-recurrence squamous cell head and neck cancer. This trial is anticipated to be a single cycle trial with surgery to follow in approximately 36 patients with pathologic response rate as the primary endpoint. The Company has initiated the necessary planning activities, including protocol development, manufacturing, and CRO selection with the trial activation anticipated during the first half of 2025.
“Based on the review of the two clinical trials completed with Gedeptin, we believe that this therapy, combined with an immune checkpoint inhibitor, has the potential to represent an active therapy against solid tumors. As such, in alignment with the Gedeptin clinical advisory committee, we have initiated efforts in support of an expanded Phase 2 trial, combining Gedeptin with an immune checkpoint inhibitor as therapy in squamous cell head and neck cancer,” stated J. Marc Pipas, M.D., GeoVax Executive Medical Director, Oncology.
“We are pleased to announce the plans for this novel, expanded Phase 2 clinical trial of Gedeptin, seeking to demonstrate efficacy and safety of Gedeptin and an immune checkpoint inhibitor combination therapy in support of such neoadjuvant therapy. We look forward to activation of this trial and are pursuing development plans in additional solid tumor indications in partnership with leading academic oncology centers. Further updates will be provided during the remainder of 2024,” added David Dodd, GeoVax’s Chairman and CEO.
About Gedeptin®
Gedeptin is a novel patented product/technology for the treatment of solid tumors through a gene therapy strategy known as Gene-Directed Enzyme Prodrug Therapy (GDEPT). In GDEPT, a vector is used to selectively transduce tumor cells with a non-human gene, which expresses an enzyme that converts a nontoxic prodrug into a highly active antitumor compound in situ.
The Phase 1b/2a trial (ClinicalTrials.gov Identifier: NCT03754933) evaluated the safety and efficacy of repeat cycles of Gedeptin therapy in patients with recurrent head and neck squamous cell carcinoma (HNSCC), with tumor(s) accessible for injection and no curable treatment options. The protocol entailed up to five treatment cycles, each consisting of three intratumoral injections of Gedeptin over two days followed by infusion of a prodrug, fludarabine phosphate, once a day for three days. A previously completed Phase 1 dose-ranging study demonstrated that treating a tumor with a single cycle of Gedeptin, followed by fludarabine infusions, was well tolerated, with evidence of a reduction in tumor size in patients with solid tumors.
The current study is being funded in part by the FDA pursuant to its Orphan Products Clinical Trials Grants Program. The FDA has also granted Gedeptin orphan drug status for the intratumoral treatment of anatomically accessible oral and pharyngeal cancers, including cancers of the lip, tongue, gum, floor of mouth, salivary gland, and other oral cavities.
About GeoVax
GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel vaccines for many of the world’s most threatening infectious diseases and therapies for solid tumor cancers. The company’s lead clinical program is GEO-CM04S1, a next-generation COVID-19 vaccine for which GeoVax was recently awarded a BARDA-funded contract to sponsor a 10,000-participant Phase 2b clinical trial to evaluate the efficacy of GEO-CM04S1 versus an approved COVID-19 vaccine. In addition, GEO-CM04S1 is currently in three Phase 2 clinical trials, being evaluated as (1) 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, (2) a booster vaccine in patients with chronic lymphocytic leukemia (CLL) and (3) a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. In oncology the lead clinical program is evaluating a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax has a strong IP portfolio in support of its technologies and product candidates, holding worldwide rights for its technologies and products. The Company has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information about the current status of our clinical trials and other updates, 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.
Fibromyalgia is a “diagnosable condition” in people suffering from Long COVID according to NASEM1
Diagnosing fibromyalgia in Long COVID patients is expected to expand the potential addressable market for Tonmya relative to pre-COVID-19 pandemic estimates2
Tonmya is a potential new first-line non-opioid analgesic under development for the management of fibromyalgia with Fast Track designation: NDA submission target is second half of 2024, with potential for FDA approval in 2025
CHATHAM, N.J., July 31, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today provided an update on the potential impact on the size of the fibromyalgia market for its development candidate Tonmya (TNX-102 SL or cyclobenzaprine HCl sublingual tablets), based on the new definition of Long COVID by the U.S. National Academies of Sciences, Engineering and Medicine (NASEM) announced on June 11, 20241.
“The consensus report from the NASEM concludes that fibromyalgia is a ‘diagnosable condition’ in people suffering from Long COVID,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “It has long been recognized that fibromyalgia can arise after a viral illness and is considered an infection-associated chronic condition.2,3 The NASEM definition confirms that fibromyalgia occurs after infection with the SARS-CoV-2 virus in some patients with Long COVID. Tonix is planning to file a New Drug Application (NDA) for Tonmya in the second half of this year for the indication of fibromyalgia with the U.S. Food and Drug Administration (FDA). We believe that diagnosing fibromyalgia in Long COVID patients will increase the potential market for Tonmya following approval as compared to market estimates from before the COVID-19 pandemic.”
Dr. Lederman continued, “Prior to the COVID-19 pandemic, the prevalence of fibromyalgia was estimated to be more than 10 million adults in the U.S.4 The U.S. Census Bureau, the National Center for Health Statistics Household Pulse Survey and the Centers for Disease Control and Prevention (CDC) estimate that approximately 5.3% of U.S. adults suffer from Long COVID, or 14 million people.5 Fibromyalgia is commonly diagnosed in Long COVID patients6-8. Although it is unknown how many Long COVID patients meet the diagnostic criteria for fibromyalgia, the National Institutes of Health (NIH)-sponsored RECOVER study found many Long COVID patients suffer from pain at multiple sites9. The Company has previously presented its analysis of real-world evidence from the TriNetX claims database suggesting that over 40% of Long COVID patients present with a constellation of symptoms that overlap with fibromyalgia.10,11 The Company completed a proof-of-concept study in Long COVID patients who presented with multisite pain and found a nominal benefit in fatigue with TNX-102 SL treatment with no new safety signals. We hope this new definition of Long COVID will allow patients suffering from chronic pain and other debilitating symptoms to access the care they need.”
About Fibromyalgia
Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6 million to 12 million adults in the U.S., the majority of whom are women. Symptoms of fibromyalgia include chronic widespread pain, non-restorative sleep, fatigue, and brain fog (or cognitive dysfunction). Other associated symptoms include mood disturbances, including anxiety and depression, headaches, and abdominal pain or cramps. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.
About Tonmya* (also known as TNX-102 SL)
Tonmya™ (cyclobenzaprine HCl sublingual tablets) is a centrally acting, non-opioid, non-addictive, investigational drug being developed as a daily bedtime medication for the management of fibromyalgia, alcohol use disorder and agitation in Alzheimer’s disease. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride TNX-102 SL provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT2A-serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic receptors. The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TNX-102 SL composition. These patents are expected to provide TNX-102 SL, upon NDA approval, with U.S. market exclusivity until 2034/2035. In December 2023, the company announced highly statistically significant and clinically meaningful topline results in RESILIENT, the second pivotal Phase 3 clinical trial of Tonmya for the management of fibromyalgia. In the study, Tonmya met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all six key secondary endpoints related to improving sleep quality, reducing fatigue and improving overall fibromyalgia symptoms and function. RELIEF, the first statistically significant Phase 3 trial of Tonmya in fibromyalgia, was completed in December 2020. It met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010) and showed activity in key secondary endpoints. In both pivotal studies, the most common treatment-emergent adverse event was tongue or mouth numbness at the administration site, which was temporally related to dosing, self-limited, never rated as severe, and rarely led to study discontinuation (one participant in each study). Tonix has recently announced receipt of the formal minutes from both recent pre-New Drug Application (NDA) meetings with the U.S. Food and Drug Administration (FDA) for Tonmya for the management of fibromyalgia. At these meetings, the Company and the FDA agreed on the proposed content and timing of an NDA submission. The Company reaffirmed its guidance to submit the NDA for Tonmya to the FDA in the second half of 2024 which would allow for a potential FDA approval in 2025. Tonmya for the management of fibromyalgia has been granted FDA Fast Track designation.
About the Phase 2 PREVAIL Study
The Phase 2 PREVAIL study was a 14-week double-blind, randomized, multicenter, placebo-controlled study to evaluate the efficacy and safety of TNX-102 SL taken daily at bedtime in patients with multi-site pain associated with Long COVID. The trial was conducted at approximately 30 sites in the U.S. The study was not significant on the primary efficacy endpoint of the change from baseline in the weekly average of daily self-reported worst pain intensity scores at the Week 14 endpoint. Nominal activity of TNX-102 SL was shown in fatigue.
For more information, see ClinicalTrials.gov Identifier: NCT05472090.
About Long COVID
Long COVID is an infection-associated chronic condition (IACC) that occurs after SARS-CoV-2 infection and is present for at least 3 months as a continuous, relapsing and remitting, or progressive disease state that affects one or more organ systems. Long COVID is the term widely recognized for the post-COVID syndrome formerly known as Post-acute sequelae of COVID-19, or PASC. Although most people recover from COVID-19 within weeks of the acute illness, a substantial portion develops Long COVID. These individuals experience a constellation of disabling symptoms long past the time of recovery from acute COVID-19. Most Long COVID patients who have been studied appear to have cleared the SARS-CoV-2 infection from their systems.
Tonix Pharmaceuticals Holding Corp.**
Tonix is a fully-integrated biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.
**Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
Atlanta, GA, July 30, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a clinical-stage biotechnology company developing immunotherapies and vaccines against cancer and infectious diseases, today announced that it will report second quarter 2024 financial results on Tuesday, August 6, 2024, after the close of U.S. markets. Following the release, management will host a live conference call and webcast, including Q&A, at 4:30 p.m. ET to provide a corporate update and discuss financial results.
A webcast replay of the call will be available for three months via the same link as the live webcast approximately two hours after the end of the call.
About GeoVax
GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel vaccines for many of the world’s most threatening infectious diseases and therapies for solid tumor cancers. The company’s lead clinical program is GEO-CM04S1, a next-generation COVID-19 vaccine for which GeoVax was recently awarded a BARDA-funded contract to sponsor a 10,000-participant Phase 2b clinical trial to evaluate the efficacy of GEO-CM04S1 versus an approved COVID-19 vaccine. In addition, GEO-CM04S1 is currently in three Phase 2 clinical trials, being evaluated as (1) 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, (2) a booster vaccine in patients with chronic lymphocytic leukemia (CLL) and (3) a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. In oncology the lead clinical program is evaluating a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax has a strong IP portfolio in support of its technologies and product candidates, holding worldwide rights for its technologies and products. The Company has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information about the current status of our clinical trials and other updates, visit our website: www.geovax.com.
Presentation to highlight statistically significant Phase 3 results of Tonmya™ for the management of fibromyalgia, including improvement in pain and sleep quality
Tonix granted FDA Fast Track Designation for Tonmya™; NDA submission on track for the second half of 2024
CHATHAM, N.J., July 30, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced a poster presentation at the International Association for the Study of Pain (IASP) 2024 World Congress on Pain, being held August 5-9, 2024 in Amsterdam, Netherlands. Details on the presentation can be found below.
A copy of the Company’s poster presentations will be available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference. Additional meeting information can be found on the IASP website here.
Poster Presentation Details
Presenter:
Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals
Title:
Targeting Fibromyalgia Non-Restorative Sleep with Bedtime TNX-102 SL (Sublingual Cyclobenzaprine HCl): Results of the Positive Phase 3 RESILIENT Trial Consistent with Syndromic Improvement
Abstract:
#WE685
Date/Time:
Wednesday, August 7, 2024, 3:15 p.m. CEST
Tonix Pharmaceuticals Holding Corp.**
Tonix is a fully-integrated biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya*, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to Tonmya for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.
**Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
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.
Eledon Announced It Has Reached Two-Thirds Patient Enrollment Milestone. Eledon has enrolled its 80thpatient in the Phase 2 BESTOW trial testing tegoprubart against tacrolimus for prevention of kidney transplant rejection. The trial is expected to reach its full enrollment of 120 patients by YE2024 as expected.
The BESTOW Trial Is Designed To Compare Tegoprubart To Tacrolimus. The Phase 2 BESTOW trial is an open-label active-comparator trial comparing tegoprubart directly against tacrolimus, the standard of care in kidney transplantation. The primary endpoint is superior mean eGFR (a measure of kidney graft function) at 12 months post-transplant, with additional endpoints to provide clinically meaningful measures for comparison with tacrolimus.
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*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.
