Tag: Biotechnology

Johnson & Johnson Spends $2 Billion to Buy Ambrx and Expand in Oncology

Posted on by slightbourne@noblecapitalmarkets.com

Johnson & Johnson announced Monday that it will acquire clinical-stage biotech Ambrx Biopharma for $2 billion, making a big bet on Ambrx’s proprietary platform for developing next-generation antibody drug conjugates (ADCs) to treat cancer.

The acquisition provides Johnson & Johnson access to Ambrx’s promising pipeline of ADC candidates, while also allowing the healthcare giant to leverage Ambrx’s novel conjugate technology that improves the efficacy and safety of ADCs. Ambrx’s proprietary platform incorporates synthetic amino acids to allow site-specific conjugation of antibodies to toxic payloads, creating more stable ADCs with less off-target effects.

Johnson & Johnson is particularly interested in Ambrx’s lead asset ARX517, an anti-PSMA ADC currently in Phase 1/2 development for metastatic castration-resistant prostate cancer (mCRPC). Prostate cancer has long been a focus for J&J and its Janssen pharmaceuticals unit, with blockbuster prostate cancer drug Zytiga bringing in over $2 billion in annual sales prior to losing patent protection in 2019.

The pressing need for improved mCRPC treatments provided additional impetus for the deal. Over 185,000 men in the U.S. currently have mCRPC, with a poor median overall survival of less than two years. The early data for ARX517 demonstrates promising anti-tumor activity, and Johnson & Johnson believes the drug could become a first-in-class targeted ADC therapy for mCRPC if approved.

“We see a unique opportunity to harness the potential of this innovative ADC platform, and with our deep understanding of prostate cancer, deliver a targeted PSMA therapeutic for addressing the growing needs of the more than 185,000 patients living with metastatic castration-resistant disease today,” said Dr. Yusri Elsayed, Global Therapeutic Area Head for Oncology at Johnson & Johnson.

Beyond ARX517, Ambrx has several other ADC candidates in its pipeline targeting cancer antigens like HER2 and CD70, providing Johnson & Johnson with a robust suite of new ADC therapies that can be optimized using Ambrx’s conjugate technology.

The acquisition reflects Johnson & Johnson’s strategy of using deals to access innovation, especially in high-potential areas like oncology. With in-house R&D productivity under scrutiny, major players like J&J and its pharma peers have turned to M&A to supplement pipeline development. Cancer has been the top therapy area target for M&A over the past 5 years, according to EY data, demonstrating the demand for innovative oncology drugs.

Ambrx was founded in 2003 as a spin-out from The Scripps Research Institute. The company raised over $200 million in venture capital and held its IPO in 2021, listing on the NASDAQ exchange. The $2 billion buyout price represents a nice return for Ambrx’s backers and shareholders.

The deal is expected to close in the first half of 2024, pending approval from Ambrx stockholders as well as regulatory clearance. Upon completion of the acquisition, Ambrx’s stock will be delisted and it will no longer be an independent public company.

Johnson & Johnson’s acquisition of Ambrx highlights the pharma industry’s race to find new modalities like ADCs that can precisely target cancer cells while minimizing side effects. With cancer poised to become the leading cause of death globally, the need for better tolerated treatments has never been more pressing. J&J is making a big bet that Ambrx’s next-gen ADC platform can yield breakthroughs in achieving that goal.

Take a moment to take a look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage universe.

Merck Acquires Harpoon Therapeutics for $680 Million To Diversify Cancer Immunotherapies

Posted on by slightbourne@noblecapitalmarkets.com

Merck has announced a definitive agreement to acquire clinical-stage biotech Harpoon Therapeutics for $23 per share in an all-cash deal valued at approximately $680 million. The acquisition provides Merck with Harpoon’s promising pipeline of novel T-cell engager immunotherapies that harness the body’s immune system to treat cancer.

Harpoon’s lead asset is HPN328, an investigational T-cell engager targeting delta-like ligand 3 (DLL3) for the treatment of small cell lung cancer (SCLC) and other neuroendocrine tumors expressing DLL3. HPN328 directs a patient’s T-cells to kill tumor cells displaying DLL3. In October 2022, Harpoon reported positive interim data from the ongoing Phase 1/2 trial showing encouraging tolerability and early signs of efficacy for HPN328.

The acquisition expands Merck’s burgeoning oncology portfolio, adding a new modality to its toolkit. “This agreement reflects the creativity and commitment of scientists and clinical development teams at Harpoon. We look forward to further evaluating HPN328 in innovative combinations with other pipeline candidates,” stated Dr. Dean Y. Li, President of Merck Research Laboratories.

Harpoon’s TriTAC and ProTriTAC Platforms

Beyond HPN328, Merck also gains Harpoon’s proprietary TriTAC and ProTriTAC platforms for developing novel T-cell engagers. TriTACs (tri-specific T-cell activating constructs) are engineered protein therapies designed to recruit a patient’s immune cells to attack tumor cells. The ProTriTAC platform applies a prodrug concept to remain inactive until reaching the tumor site.

Harpoon has an extensive pipeline of TriTAC candidates against various cancer targets, including:

  • HPN217: Targets B-cell maturation antigen (BCMA) for relapsed/refractory multiple myeloma, currently in Phase 1.
  • HPN601: Targets epithelial cell adhesion molecule (EpCAM) for solid tumors expressing EpCAM.
  • HPN424: Targets delta-like ligand 4 (DLL4) for solid tumors.
  • Other preclinical TriTACs targeting tumor antigens like NaPi2b, FLT3, and DLL3.

The platforms offer modular designs to quickly generate and test new immunotherapies directed to disease-specific targets. Merck can leverage these platforms to strengthen its immunotherapy pipeline in cancer and possibly other disease areas.

Merck Building an Oncology Powerhouse

Cancer immunotherapies represent the next wave of innovation in oncology drug development. The Harpoon acquisition aligns with Merck’s strategy to establish leadership in immuno-oncology.

Merck already markets the blockbuster PD-1 checkpoint inhibitor Keytruda, approved for 30 different cancer indications. Keytruda generated $17.2 billion in sales in 2021. Now with Harpoon, Merck adds T-cell engagers to its arsenal. These therapies provide another way to leverage the immune system against hard-to-treat tumors like SCLC.

Merck is also developing numerous other novel agents across various modalities:

  • Cancer vaccines targeting specific tumor mutations (Personalized Cancer Vaccine, V590, V591)
  • Antibody-drug conjugates (belantamab mafodotin, ladiratuzumab vedotin)
  • Bispecific fusion proteins targeting both PD-1 and LAG-3
  • First-in-class inhibitors (MK-6482, KL-A)

Combined with its extensive capabilities in discovery research and clinical development, Merck is positioning itself as an oncology powerhouse able to take on cancers from all angles.

The Harpoon acquisition provides another building block in this strategy. In Harpoon’s pipeline and platforms, Merck gains cutting-edge T-cell engager capabilities to complement internal immuno-oncology programs. Merck can advance Harpoon’s therapies into new combination regimens and indications to maximize their potential.

Deal Details

Under the terms of the agreement, Merck will acquire Harpoon through a subsidiary, purchasing all outstanding Harpoon shares for $23 each in cash. This represents a premium of 118% over Harpoon’s previous closing share price.

The deal has been approved by Harpoon’s Board of Directors and is expected to close in the first half of 2024, pending shareholder approval and regulatory clearances. It will be accounted for as an asset acquisition by Merck.

Harpoon shareholders will vote on the acquisition at a future shareholder meeting. The waiting period under the Hart-Scott-Rodino Antitrust Improvements Act will also need to expire.

Advisors on the deal include Evercore Group for Merck and Centerview Partners for Harpoon.

With promising new immunotherapies and platforms adding to its robust oncology pipeline, Merck strengthens its leadership in the high-growth cancer drug market. The Harpoon acquisition provides Merck with new T-cell engager capabilities to help develop life-changing medicines for patients with cancer worldwide.

Release – GeoVax Appoints Dr. Marc Pipas as Executive Medical Director, Oncology

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

 

 Strengthening GeoVax’s Team Advancing Gedeptin® Clinical Program

Atlanta, GA, January 8, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced the appointment of J. Marc Pipas, M.D., to serve as the Company’s Executive Medical Director, Oncology.

Kelly McKee, M.D., MPH, GeoVax Chief Medical Officer, commented, “Dr. Pipas’ addition to our team represents an important step forward in the strengthening of GeoVax’s organizational and operational resources to support advancement of our principal oncology asset, Gedeptin®, through clinical development and regulatory approval.  He is also well-positioned to guide the clinical development of GeoVax’s broader immune-oncology portfolio. We are excited to leverage the wealth of knowledge, expertise, and insights he will provide relative to the clinical developments in support of our oncology programs.”

“I am pleased to be joining the GeoVax team,” said Dr. Pipas. “Oncologic research and development is my passion and I look forward to engaging with our clinical researchers, regulatory authorities, and the medical community at large, to advance GeoVax’s mission.”

Dr. Pipas has extensive clinical, research, and leadership expertise in oncology, built on a long and successful academic career at Dartmouth-Hitchcock Medical Center/Norris Cotton Cancer Center, an NCI Comprehensive Cancer Center.  During his academic career, Dr Pipas led the GI Clinical Oncology Group for >10 years, served as Hematology/Oncology Fellowship Director, was Medical Director for the Office of Clinical Research in the Cancer Center, and served for more than a decade as a member of Human Subject Protection and Data Monitoring Boards. Following his academic career, he held positions of increasing responsibility in the biopharma industry, guiding the development of several novel oncology therapeutics through early- and late-stage clinical trials.

Dr. Pipas received his Doctor of Medicine degree from SUNY-HSC at Syracuse in 1989 and completed a residency in Internal Medicine at the Medical University of South Carolina in Charleston.  Following this, he completed a fellowship in Hematology/Oncology at Dartmouth-Hitchcock Medical Center in New Hampshire and was then invited to become a faculty member at Dartmouth Medical School where he achieved the rank of Full Professor. He has 40 scientific papers and more than 60 abstracts to his credit and has numerous nominations and awards for teaching and humanism in medicine. Dr Pipas brings a deep understanding of oncologic therapeutics and clinical trial management, as well as a network of research contacts and leadership skills honed by many years of experience.

About GeoVax

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

Forward-Looking Statements

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

Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law. 

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

Annual JPMorgan Conference Attracts Investors Seeking Insights Into Biotech’s Promising Pipeline

Posted on by slightbourne@noblecapitalmarkets.com

The buzz in biotech circles is building as the industry prepares to descend on San Francisco for the annual JPMorgan Healthcare Conference running January 8th through 11th. The high-profile event represents a prime opportunity for investors to gain valuable insights into the sector’s most promising up-and-comers.

Now in its 42nd year, the JPMorgan conference attracts leading biotech and pharmaceutical companies along with institutional investors, analysts, and dealmakers. Presenting firms range from massive big pharma players to small emerging growth biotechs.

Nearly 500 companies are slated to present this year, most running 30-minute Q&A sessions. These tightly packed presentations offer a wealth of intel for those looking to separate promising science from speculative hype.

The event also facilitates crucial networking and dealmaking. With so many industry leaders gathered in one place, the conference often catalyzes partnerships, financing deals, and even M&A activity.

For investors, the information bonanza can heavily influence trading decisions in the year ahead. The majority of presenting firms see significant stock volatility around their presentations as analysts and investors digest new details.

This is especially true for micro-cap biotechs developing novel platforms. The conference represents their best shot at introducing promising science to a captive audience.

Noble Capital Markets analyst Robert LeBoyer will be at the JPMorgan conference seeking hidden gems among early-stage drug developers to add to his coverage universe. His focus areas include oncology, rare diseases, and molecular diagnostics.

The four-day gathering kicks off Monday evening with keynote presentations from industry luminaries like Eli Lilly CEO Dave Ricks and CVS Health Executive Vice President Karen Lynch.

But the real action gets going Tuesday morning when company presentations start at 7:30am local time. With non-stop panels running through Thursday afternoon, the schedule stays jam-packed.

Much of the focus tends to fall on clinical trial data reveals and pipeline updates for major drug development programs. However, digging into the schedules of micro-cap presenters can pay off big for enterprising analysts and investors.

These small companies are often where the next generation of groundbreaking therapies get their start. Wall Street has seen many cases where a small or microcap biotech makes waves at JPMorgan only to become a mammoth player years later.

For instance, cancer therapy innovator Mirati Therapeutics has skyrocketed from a $200 million micro-cap at the 2012 conference to now boast a $10 billion valuation. The company’s promising clinical data updates year after year built significant investor enthusiasm.

Success stories like Mirati help explain why the JPMorgan conference receives such massive interest despite its insider feel. Registering to attend requires an invitation, and getting meetings with management teams can prove challenging given packed schedules.

But resourceful attendees find ways to build productive agendas even without formal presentations. The four-day stretch offers countless sidebar conversations and impromptu meetups.

The healthcare sector faces no shortage of complex challenges, from surging costs to ageing populations across the developed world. But the constant flow of biopharmaceutical innovation provides reason for long-term optimism.

Conferences like JPMorgan offer a window into the relentless progress companies of all sizes are making against the world’s most pressing health needs. For investors, finding the next breakthrough drug before it makes headlines could lead to substantial upside. That’s why analysts like LeBoyer eagerly make the trek each year.

The scope of the JPMorgan Healthcare Conference mirrors the diverse breadth of the wider industry. Oncology, rare diseases, neurology, infectious diseases – no area with unmet needs goes overlooked.

Both science and business play equal roles at a conference ultimately aimed at facilitating capital flows into the most promising research. The progress showcased reflects the entwinement of noble medical advancement and shrewd financial investment.

In that sense, JPMorgan offers the ideal backdrop for launching promising biotech companies into the public markets. The conference’s elevated stage has introduced scores of now-large firms over the years, and 2024 will undoubtedly add to that list.

For a list of emerging growth biotech companies, take a look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage universe.

Release – Eledon Pharmaceuticals Highlights Recent Business Milestones and Provides 2024 Outlook

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ELDN

January 4, 2024

PDF Version

Reported updated data from ongoing Phase 1b trial in kidney transplantation demonstrating tegoprubart treatment successfully prevented kidney transplant rejection and was generally safe and well-tolerated 

Dosed first participants in Phase 2 BESTOW trial in kidney transplantation 

Tegoprubart dosed in second-ever pig to human xenotransplant procedure

IRVINE, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced a summary of 2023 accomplishments and provided guidance for anticipated upcoming 2024 milestones.

2023 Key Highlights

  • Reported updated data from ongoing Phase 1b trial evaluating tegoprubart for prevention of rejection in kidney transplantation. Data from 11 trial participants demonstrated that tegoprubart was generally safe and well-tolerated in patients undergoing kidney transplantation, with aggregate mean estimated glomerular filtration rate (eGFR) above 70 mL/min/1.73m2 at all reported time points after 90 days post-transplant. Amended the Phase 1b trial protocol to add a second cohort, now allowing enrollment of up to 24 trial participants who are undergoing kidney transplantation.
  • Dosed first participants in Phase 2 BESTOW trial assessing tegoprubart head-to-head with tacrolimus for the prevention of rejection in kidney transplantation.
  • Dosed tegoprubart in second-ever transplant of genetically modified heart from a pig to a human.
  • Completed financing of up to $185 million, with $35 million in upfront funding and additional aggregate financing of up to $105 million, subject to achieving clinical development milestones, volume weighted share price levels, and trading volume conditions, as well as up to an additional $45 million upon exercise of warrants. If all commitments are met, the financing is expected to be sufficient to fund the Company through the completion of the Phase 2 BESTOW trial, subject to the achievement of specified milestones, including clinical development enrollment targets.
  • Partnered with the University of Chicago Transplantation Institute to secure financing from the Juvenile Diabetes Research Foundation (JDRF) and The Cure Alliance to fund an investigator sponsored study in pancreatic islet cell transplantation in participants with type 1 diabetes. Tegoprubart treatment will be evaluated for the prevention of transplant rejection.
  • Strengthened leadership team with appointment of Eliezer Katz, M.D., FACS as Chief Medical Officer and strengthened board of directors with appointment of Allan Kirk, M.D., Ph.D. and James Robinson.

“Eledon made significant progress in 2023 on multiple fronts, highlighted by the presentation of the first clinical evidence of tegoprubart’s potential to prevent organ rejection while producing robust improvements in eGFR and maintaining a favorable safety profile. We were also honored to play an important role in the second-ever transplant of a genetically modified heart from a pig to a human with the use of tegoprubart as part of the patient’s cornerstone immunosuppressive regimen,” said David-Alexandre C. Gros, M.D., Chief Executive Officer of Eledon. “In the coming year, we look forward to building upon the promising results from our tegoprubart kidney transplant Phase 1b trial, generating additional long-term data from the Phase 1b extension study, and completing enrollment in our Phase 2 BESTOW trial.”

Anticipated 2024 Milestones

  • First half of 2024: Report updated interim clinical data from the ongoing Phase 1b trial of tegoprubart in kidney transplantation.
  • First half of 2024: Dose the 12th participant in the Phase 2 BESTOW trial. Upon the dosing of the 12th participant, the Company will have completed both clinical development milestones related to the second financing tranche from the private placement announced on May 1, 2023.
  • End of 2024: Complete enrollment in the Phase 2 BESTOW trial of tegoprubart in kidney transplantation.
  • 2024: Dose the first islet cell transplant participant for the treatment of type 1 diabetes at the University of Chicago Transplantation Institute.

About Eledon Pharmaceuticals and tegoprubart

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

Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter

Forward-Looking Statements

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

Investor Contact:

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

Media Contact:

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

Source: Eledon Pharmaceuticals

Release – GeoVax Announces Issuance of Malaria Vaccine Patent

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

 

Patent Covers Multiple Component Vaccine for Both Prevention and Treatment

Atlanta, GA, January 3, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced that the U.S. Patent and Trademark Office has issued Patent No. 11,857,611 to GeoVax, pursuant to the Company’s patent application No. 17/726,254 titled “Compositions and Methods for Generating an Immune Response to Treat or Prevent Malaria.”

The claims granted by the patent generally cover compositions comprising GeoVax’s modified vaccinia Ankara (MVA) vector expressing Plasmodium antigens and methods of inducing an immune response to malaria utilizing the compositions. The compositions and methods covered in the claims are useful both prophylactically and therapeutically and may be used to prevent and/or treat malaria.

David Dodd, GeoVax President and CEO, commented, “During 2023, there were the first cases of locally transmitted malaria reported in the United States in 20 years, with cases reported in Florida, Maryland and Texas. Worldwide, there are over 600,000 deaths annually attributed to malaria. This patent represents a potentially significant advance against a most critical deadly threat worldwide. While our focus and priority is to support our clinical-stage programs, we also remain strongly committed to improving public health worldwide and developing vaccines against global public health threats, such as malaria, as a part of our long-term vision for the company.”

According to data from the World Health Organization, globally, malaria causes 227 million infections and 619,000 deaths annually. Despite decades of vaccine research, vaccine candidates have failed to induce substantial protection. Most of these vaccines are based on individual proteins that induce immune responses targeting only one stage of the malaria parasite’s life cycle. GeoVax’s MVA-VLP malaria vaccine candidates incorporate antigens derived from multiple stages of the parasite’s life cycle and are designed to induce an immune response with durable functional antibodies and CD4+ and CD8+ T cell responses, all hallmarks of an ideal vaccine-induced immune response.

About GeoVax

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

Forward-Looking Statements

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

Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law. 

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

ZyVersa Therapeutics, Inc. (ZVSA) – Novel Therapies For Renal Diseases and Inflammation

Posted on by cpereira@noblefcm.com


Wednesday, January 03, 2024

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.

Initiating Coverage With An Outperform Rating. ZyVersa Therapeutics is a biotechnology company focused on renal and inflammatory diseases. Its lead product, VAR 200, is in development to reduce renal cholesterol and lipid accumulation in the kidney. These accumulations damage the kidney and lead to loss of filtration in kidney diseases. Its second product in development, IC 100, is an inflammasome inhibitor to inhibit the inflammation that contributes to many chronic diseases.

Renal Diseases. Accumulation of renal cholesterol and lipids in the kidney leads to cellular damage that starts a decline in filtration function and leakage of protein into the urine. VAR 200 was developed to reduce levels of cholesterol and lipids in renal cells to reduce the damage, scarring, and disease progression. The first clinical trial in diabetic kidney disease (DKD) is planned for early 2024.


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Bristol Myers Squibb $4.1B RayzeBio Buyout

Posted on by slightbourne@noblecapitalmarkets.com

Pharma giant Bristol Myers Squibb (BMY) announced Tuesday that it will acquire clinical-stage biotech RayzeBio for $4.1 billion, continuing Bristol’s strategy of deals to refresh its drug pipeline amid upcoming patent expirations.

RayzeBio is developing a novel targeted radiotherapy called RYZ101 to treat multiple types of cancer. The company’s technology combines tumor-targeting antibodies with radioactive isotope payloads that selectively damage cancer cells’ DNA when delivered.

RYZ101 is currently in Phase 3 testing for treating metastatic castration-resistant prostate cancer. Early clinical data showed promising results with the drug demonstrating tumor response rates of 44-55%.

Bristol gains full rights to RYZ101 and RayzeBio’s broader platform for linking radioisotopes to cancer-fighting proteins. The deal gives Bristol a potential new blockbuster cancer treatment as competition intensifies in the immuno-oncology space.

Shoring Up the Cancer Business

Bristol already markets leading cancer immunotherapies Opdivo and Yervoy. However, Opdivo faces patent expiration in 2028/2031, forcing Bristol to find new long-term growth drivers.

The RayzeBio deal comes right after Bristol announced the $13.1 billion acquisition of schizophrenia drug developer Karuna Therapeutics last Friday. Karuna’s lead drug KarXT could generate peak annual sales of over $3 billion, analysts project.

These acquisitions help future-proof Bristol’s business as its top-selling drugs face new competition. Blood thinner Eliquis, which makes up over 30% of Bristol’s revenue, will see biosimilar rivals by 2026. Cancer drug Revlimid, acquired in Bristol’s 2019 buyout of Celgene, faces generics soon too.

“We are focused on strengthening our portfolio through a combination of internal programs and targeted business development,” said Bristol Myers CEO Giovanni Caforio. The RayzeBio and Karuna deals “complement our existing pillars of growth,” he added.

Betting Big on Radio-Pharmaceuticals

In addition to RYZ101’s potential, Bristol gains RayzeBio’s expertise with radio-pharmaceuticals. Attaching radioactive particles to antibodies allows them to precisely pinpoint tumor cells and kill them via DNA damage.

RayzeBio’s technology overcomes past challenges with radio-drugs such as lack of tumor specificity and rapid decay of radioisotopes. Linking radioisotopes to robust antibodies circumvents these issues and improves the drugs’ efficacy.

Analysts see radio-pharmaceuticals as an emerging trend in oncology. Radio-immunotherapies like RayzeBio’s could complement immuno-oncology drugs that activate the immune system against cancer.

By acquiring RayzeBio’s platform, Bristol can expand development of new radio-drug conjugates across its oncology pipeline. Bristol may also look to license out the technology to other companies given the heightened industry interest.

An Expensive Acquisition

Bristol is paying a huge premium to acquire RayzeBio before the biotech can prove RYZ101’s efficacy in late-stage testing. The $4.1 billion price tag works out to $62.50 per share, more than double RayzeBio’s prior closing price.

But Bristol likely wanted to preempt competition for the promising biotech asset. Amgen and Novartis are also developing radio-pharmaceutical drugs for cancer. And RayzeBio would have commanded an even higher valuation had RYZ101 succeeded in Phase 3.

Bristol expects the acquisition will reduce its adjusted earnings by about 13 cents per share in 2024. But Bristol maintained its existing profit guidance for 2022 and 2023, implying confidence the long-term benefits outweigh the near-term costs.

The company plans to finance the purchase using new debt. Bristol’s strong cash flows should allow it to service the additional debt load as it waits for RYZ101 to potentially reach the market around 2025.

Conclusion: Bolstering Its Firepower

The back-to-back deals for Karuna Therapeutics and RayzeBio showcase Bristol Myers Squibb’s strategy to acquire new therapies and drug platforms that can drive growth over the next decade. While expensive, these acquisitions reduce Bristol’s reliance on aging blockbuster drugs facing patent cliffs.

Gaining Karuna’s potential multi-billion dollar schizophrenia medicine and RayzeBio’s cutting-edge radio-pharmaceutical technology gives Bristol valuable new firepower to deploy in the fiercely competitive pharma market. If successful, the deals will ensure Bristol Myers remains an industry leader as it confronts upcoming challenges from biosimilar and generic competition.

Take a moment to take a look at emerging growth biotechnology companies by looking at Noble Capital Markets Senior Research Analyst Robert LeBoyer’s coverage universe.

Bristol Myers Drops $14 Billion to Acquire Karuna Therapeutics, Gaining Schizophrenia Drug

Posted on by slightbourne@noblecapitalmarkets.com

Pharmaceutical giant Bristol Myers Squibb made a bold move into neuroscience today, announcing the $14 billion acquisition of clinical-stage biotech Karuna Therapeutics. The massive deal provides Bristol Myers with Karuna’s lead drug candidate, KarXT, a potential new treatment for schizophrenia and other psychiatric disorders.

KarXT could be the first drug in its class approved for schizophrenia in decades. The market for schizophrenia drugs is estimated at over $7 billion globally. If approved, KarXT is projected to achieve multi-billion dollar peak sales. Bristol Myers is betting the experimental medicine could transform treatment for millions struggling with serious mental illness.

This acquisition is the latest in a wave of big pharma interest in the emerging neuroscience space. Companies are eager to find new approaches to historically hard-to-treat psychiatric conditions like schizophrenia, depression and Alzheimer’s disease.

Smaller biotechs like Karuna have led the charge, developing novel therapies targeting neurological mechanisms of psychiatric disorders. But larger players like Bristol Myers have taken notice of the promise of these new technologies.

Karuna’s KarXT combines xanomeline, a novel muscarinic receptor agonist, with trospium chloride, an FDA-approved muscarinic receptor antagonist. Early clinical results show this approach reduces side effects and improves efficacy compared to current schizophrenia drugs.

Take a look at other emerging growth biotechnology companies by taking a look at Noble Capital Markets’ Senior Research Analyst Robert Leboyer’s coverage list.

In late-stage clinical trials, KarXT demonstrated statistically significant and clinically meaningful improvements in schizophrenia symptoms. Patients experienced rapid reductions in hallucinations and delusions with far fewer problematic side effects like sedation.

Based on positive Phase 3 data, Karuna submitted a New Drug Application for KarXT in schizophrenia in mid-2022. The FDA accepted the application and set a PDUFA goal date of September 2023 for a potential approval.

Clearly Bristol Myers feels confident about KarXT’s chances, agreeing to pay $28.5 billion upfront in cash to finalize the acquisition. Karuna shareholders will also be eligible for up to $3.5 billion in milestone payments if KarXT reaches certain commercial goals.

For Bristol Myers, the move signals a push into neuroscience and psychiatric disease, an area it has not traditionally emphasized. But the company likely sees major growth potential, given the prevalence of mental illness and the need for better treatments.

Almost 3% of the U.S. population suffers from schizophrenia. Another 17% experience some other mental illness like depression, bipolar disorder or PTSD. Existing drugs fail to adequately manage symptoms for many patients and carry tolerability issues that lead to poor compliance.

Doctors and patients are eagerly awaiting novel therapies like KarXT that balance safety and efficacy. Karuna is also exploring KarXT’s potential in dementia-related psychosis and other indications beyond schizophrenia.

The lucrative deal builds on other recent big-ticket acquisitions for Bristol Myers as the company looks to expand its portfolio. Earlier this year, Bristol Myers acquired cancer biotech Turning Point Therapeutics for $3.2 billion and the oncology company MyoKardia for $13 billion.

But the Karuna purchase represents Bristol Myers’ biggest bet yet on the emerging neuroscience space. It’s the second largest biopharma acquisition announced in 2022 after Pfizer’s $43 billion buyout of cancer drugmaker Seagen.

Other large pharmaceutical companies have also signed deals to access neuropsychiatric drug candidates. AbbVie recently acquired an option to purchase Alector’s experimental Alzheimer’s therapy for up to $2.2 billion. And Eli Lilly collaborated with NextCure on novel immuno-oncology approaches for treating mental illness.

As more novel mechanisms like KarXT arrive, expect growing competition among pharma giants to capture market share. Bristol Myers struck first with today’s monumental acquisition, but likely won’t be the last looking to neuroscience for future growth.

Release – Ocugen Gains FDA Alignment on Key Aspects of OCU400—Modifier Gene Therapy—Pivotal Phase 3 Study Design

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

December 21, 2023

MALVERN, Pa., Dec. 21, 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 and vaccines, today announced that the Company received alignment from FDA on key aspects of the Phase 3 clinical trial design to assess the safety and efficacy of OCU400 in patients with RHO and other gene mutations associated with Retinitis Pigmentosa (RP).

“This news brings us even closer to fulfilling our mission to bring our first-in-class, gene-agnostic therapies to market and provide access to patients globally,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “We look forward to beginning the Phase 3 clinical trial, which we plan to initiate in early 2024.”

During a multidisciplinary meeting with FDA, based on preliminary results from an ongoing Phase 1/2 study, Ocugen received alignment on key aspects of the Phase 3 study design—including the study endpoint, patient enrollment strategy, and study duration of one year. The Phase 3 clinical trial will enroll a broader group of RP patients, including patients with the most common RHO gene mutation, based on OCU400’s potentially gene-agnostic mechanism of action.

With orphan drug and RMAT designations in place for OCU400, FDA’s alignment on key aspects of the Phase 3 study design positions Ocugen to confidently move forward in pursuing product development and licensure for OCU400.

Currently there are approximately 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation. Advancing OCU400 to Phase 3 clinical development will be an important step toward addressing unmet needs in the RP patient community.

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 X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential therapeutic and clinical benefits of our product candidates, expectations for clinical trial timing and results, anticipated timing of clinical trial updates and expectations for timing and outcome of regulatory interactions, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities; that receipt of orphan drug and RMAT designations may not lead to faster development or regulatory review; and that regulatory authorities may disagree with additional aspects of our clinical trial designs or may not approve our future IND applications on the anticipated timeline or at all. 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
IR@ocugen.com

Tonix Pharmaceuticals (TNXP) – Fibromyalgia Trial Meets All Endpoints

Posted on by cpereira@noblefcm.com


Thursday, December 21, 2023

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-15001 which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-29002 for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-8013, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-18504, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-35005 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL6, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.

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.

RESILIENT Study Shows Strong Results In Fibromyalgia. Tonix announced that the Phase 3 RESILIENT study testing TNX-102 SL in fibromyalgia met its primary endpoint of pain reduction and its six secondary endpoints. The fibromyalgia population is estimated at 6 to 12 million patients, making this a significant market for Tonix. The company plans to submit an application for FDA approval in 2H2024, consistent with our projected product launch in 2025.

Tonix Raised Funds With Stock and Warrants. The company also announced a $144 million Registered Direct offering that will raise $30 million upon closing and a potential $114 million through warrant exercise. We believe the offering offset the positive news from the RESILIENT clinical trial, causing the stock to decline despite good clinical news.


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

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

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

Ocugen (OCGN) – OCU400 Receives RMAT Designation, OCU410 Treated First Patient

Posted on by cpereira@noblefcm.com


Wednesday, December 20, 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.

OCU400 Received RMAT Designation. The FDA has designated OCU400 as a Regenerative Medicine Advanced Therapy (RMAT) for treating retinitis pigmentosa associated with RHO mutations. RMAT designation is intended to speed development of regenerative medicines, and is awarded to therapies that have potential treat, reverse, or modify a life-threatening disease. We see this as a validation of Ocugen’s clinical trial data. Additional data submissions to expand the designation to other gene mutations in RP and Leber congenital amaurosis (LCA) are planned.

Phase 3 Trial Should Benefit From RMAT Designation. RMAT designation includes the benefits of Fast Track and Breakthrough designations, with increased guidance from the FDA. It also allows the use of surrogate markers as endpoints in clinical trials, and accelerated review after the application is filed. Ocugen is currently finalizing the design of the Phase 3 trial for OCU400. We expect the trial to begin in early 2024 with data available in 2H24.


Get the Full Report

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

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

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

Release – Ocugen Announces OCU400 Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment Of Retinitis Pigmentosa Associated With Rho Mutations

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

December 19, 2023

  • RMAT designation intended to help expedite development of new regenerative medicines

MALVERN, Pa., Dec. 19, 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 and vaccines, today announced that the FDA has granted RMAT designation to Ocugen’s investigational product OCU400 for the treatment of retinitis pigmentosa (RP) associated with RHO mutations.

“RMAT designation is a significant accomplishment for the OCU400 clinical development program, as it validates the potential for our game-changing gene therapy approach to fulfill an unmet medical need for people who are facing blindness due to RP,” said Arun Upadhyay, PhD, Chief Scientific Officer and Head of R&D at Ocugen. “FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”

RMAT designation for OCU400 was based on preliminary clinical data supporting the maintenance and improvement of visual acuity and function in RP patients in the OCU400 -101 Phase 1/2 clinical trial as measured by Best Corrected Visual Acuity (BCVA), Low Luminance Visual Acuity (LLVA), and Multi-Luminance Mobility Test (MLMT).

RMAT designation is part of the 21st Century Cures Act. The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition. Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA. Ocugen is working with the FDA to finalize the Phase 3 protocol necessary to advance the clinical development for OCU400 to support an application for marketing authorization.

Current data support the gene-agnostic mechanism of action for OCU400, which suggests that it may be able to provide treatment benefit to a broader group of RP and Leber congenital amaurosis (LCA) patients. Ocugen intends to submit additional efficacy and safety data for OCU400 in RP and LCA patients to the FDA in the future to potentially expand this RMAT designation to broader RP and LCA patient populations.

RHO mutations affect more than 10,000 of the 110,000 people in the United States diagnosed with RP. In the latest clinical study update from the Phase 1/2 trial of OCU400, 86% (6/7) of RHO mutation subjects experienced either stabilization or improvement in MLMT scores from baseline, among which 29% (2/7) demonstrated 3 Lux luminance level improvement. There are currently no treatment options available for RP patients with RHO gene mutations.

OCU400 represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors (NHRs) that regulate diverse physiological functions, such as homeostasis, reproduction, development, and metabolism to potentially improve retinal health and function.

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 X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, the expected benefits of RMAT designation, and anticipated regulatory interactions, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our 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
IR@ocugen.com