Tag: Biotechnology

Investors Take Interest in MAIA Biotechnology as FDA Clears Path for Cancer Drug Trial

Posted on by slightbourne@noblecapitalmarkets.com

Shares of MAIA Biotechnology were trading higher Tuesday after the company announced that the FDA has cleared its application to test its experimental cancer therapy THIO in patients in the United States.

MAIA Biotech is developing THIO as a novel immunotherapy approach for advanced non-small cell lung cancer (NSCLC). With FDA clearance of its Investigational New Drug (IND) application, the company can now include U.S. cancer patients in its ongoing mid-stage trial evaluating THIO’s safety and efficacy.

This regulatory win is driving investor enthusiasm and higher trading volume for MAIA stock today.

About MAIA Biotech and THIO

MAIA Biotech is a clinical-stage immunotherapy company aiming to improve cancer treatment by targeting telomeres. Telomeres play an important role in cancer cell survival and resistance to standard therapies.

The company’s lead therapy THIO represents a first-in-class telomere-targeting agent for NSCLC. Early preclinical research indicates THIO can induce cancer cell death and stimulate anti-tumor immune responses.

MAIA is positioning THIO as a second or third line of treatment for NSCLC patients who have stopped responding to initial immunotherapy. The company sees THIO’s novel approach as a way to improve outcomes in this hard-to-treat population.

Phase 2 Trial Details

THIO is currently being tested in a Phase 2 clinical trial involving sites across Europe, Asia Pacific, and now with FDA clearance, the United States.

The trial is evaluating THIO in combination with the PD-1 checkpoint inhibitor Libtayo in advanced NSCLC patients. Researchers want to see if giving THIO first to “prime” the immune system, followed by Libtayo, can enhance and prolong the immune response against cancer cells.

The primary goal is assessing THIO’s safety and antitumor activity based on overall response rates. Secondary goals include evaluating biomarkers and overall survival. The trial expects to enroll approximately 90 patients total.

Next Steps for MAIA

While still early stage, the FDA clearance represents an important milestone for MAIA as it works to expand THIO’s potential reach. Being able to include U.S. sites should support faster enrollment and generate data from a larger, more diverse patient population.

Positive Phase 2 results would support advancing to a pivotal Phase 3 study, which the company hopes could lead to regulatory approval. MAIA sees a multi-billion dollar market opportunity in later-line NSCLC treatment.

The company is also exploring THIO’s potential in other cancer types like melanoma, prostate cancer, and multiple myeloma.

Why Investors are Excited

FDA clearance of THIO’s IND removes a key regulatory hurdle for MAIA. Being able to test the therapy in the major U.S. market is critical for the smaller biotech company.

Today’s stock move reflects investors’ increased confidence in THIO’s outlook and MAIA’s ability to execute on development plans. If the Phase 2 trial goes well, it would further validate THIO’s novel approach and cancer-fighting potential.

While still highly speculative given the early stage, MAIA represents an intriguing immunotherapy play for investors interested in emerging approaches for hard-to-treat cancers. The company’s focus on telomere biology and unique combination strategy with Libtayo differentiate it from other biotechs.

MAIA stock could continue to be volatile in the months ahead as Phase 2 data approaches. But the FDA clearance has put a spotlight on this previously lesser known name. For investors open to some risk, MAIA may be a cancer immunotherapy stock to have on the radar.

Take a look at more research on MAIA Biotechnology by Noble Capital Markets Senior Research Analyst Robert LeBoyer.

Biotech Poised for a Powerful Comeback

Posted on by slightbourne@noblecapitalmarkets.com

After a fallow period, signs point to the biotech sector regaining its prior momentum. Several factors indicate a pending return to rapid growth and prolific innovation for biotech companies. This prospective resurgence could replicate the boom years of the 1980s and 1990s.

The first driver is scientific advancements that open new possibilities. CRISPR gene editing has revolutionized biotech, allowing cheaper and easier manipulation of genetic code. This enables creation of novel treatments and cures previously out of reach. Other breakthroughs like mRNA vaccines have proven the ability to rapidly develop radically new therapeutic approaches.

Vast amounts of genomic data generated in recent decades have also unlocked new understandings of biologically rooted diseases. By identifying key genetic drivers, drug targets can be validated to produce higher success rates in clinical trials. Failed drug candidates have historically been a major drag on biotech.

Substantial investment capital is also lining up behind biotech again after the sector fell out of favor. While biotech IPOs dropped sharply in 2022, venture funding actually rose to its second highest level ever at $32 billion. Investor appetite remains strong, especially for companies with promising new platforms.

Large cash piles among pharmaceutical giants could further bolster biotech. Big pharma companies have routinely turned to buying biotech firms to fill product pipelines. With major players like Pfizer and Merck holding over $25 billion in deployable cash reserves, expect more dealmaking ahead.

Take a look at several emerging growth biotech companies by looking at Noble Capital Market’s Senior Research Analyst Robert LeBoyer’s coverage list.

Regulatory incentives additionally sweeten the proposition of getting back into biotech. The FDA is actively supporting development in areas like gene therapy, rare diseases, and certain cancers through its pilot programs and priority reviews. This guidance can derisk investments.

The maturing ecosystem around biotech also fuels its potential rebound. Experienced veteran executives can now be tapped to steer startups. Clustering in hubs like Boston and San Francisco persists to provide concentration of talent, capital, and resources.

While risks like high failure rates remain, the ingredients are aligning for biotech’s next generation. Comparisons to the internet boom of the late 1990s resonate, with biotech representing a similar pivotal platform shift. The world’s demographics also underpin demand for new therapies – aging populations in developed nations will drive need.

This fertile environment parallels periods that produced prior biotech booms. In the late 1970s and 80s, the industry arose virtually from scratch around pioneering companies like Genentech and Amgen. The advent of genetic engineering allowed creation of the first biologic drugs.

Another surge came through the 90s as enabling technologies like high throughput screening scaled up the drug discovery process. The mapping of the human genome unleashed another wave of possibilities.

Today’s scientific advances pose an even greater leap, allowing drug development and treatment paradigms hardly imaginable just decades ago. The bounds of human understanding keep expanding.

The stars are aligning for biotech 2.0, an evolution building on past successes but catalyzed by new potentials. It promises to usher in an era of curative therapies, genomic precision, and accelerated innovation. The post-pandemic landscape offers the ideal springboard for this biotech revival.

With therapeutic bottlenecks getting cleared, investor interest rekindling, and confidence restored, expect biotech to reclaim its prior growth trajectory. The lessons of past booms were well learned, leaving companies better positioned to capitalize. Weapons to fight disease grow more powerful by the month.

The public expects and demands new treatments for pressing needs, from cancer to neurological conditions. Demographics favor biotech’s prospects as populations age. An energized ecosystem stands ready to nourish exciting science from lab to market.

The pieces are in place for biotech liftoff. As science unlocks new horizons, investor insight aligns with public health demands, fueling momentum. Biotech’s foundational role in driving modern medical progress is poised to only accelerate. The cycle of innovation spins up again.

Release – PDS Biotech Announces PDS0101 Combined with Chemoradiotherapy Associated with Rapid Decline in Circulating Tumor DNA (ctDNA/cfDNA)

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

  • Study evaluated levels of circulating cell-free HPV DNA (cfDNA) in patients with locally advanced cervical cancer in the IMMUNOCERV Phase 2 clinical trial and patients receiving standard of care (SOC) treatment
  • Patients treated with PDS0101 had greater clearance of cfDNA as compared to those treated with SOC (at week 5, 91.7% vs. 53.1%, P=0.0179)
  • Data presented during oral presentation at ASTRO 2023 Annual Meeting

PRINCETON, N.J., Oct. 02, 2023 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (PDS Biotech or the Company), a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary T cell activating platforms, today announced data demonstrating lead candidate PDS0101 in combination with standard-of-care (SOC) chemoradiotherapy (CRT) was associated with a rapid decline in human papillomavirus (HPV) circulating cell-free DNA (cfHPV-DNA), a potential predictive biomarker of treatment response. The data from the IMMUNOCERV Phase 2 clinical trial were featured in an oral presentation by Aaron Seo, MD, PhD, of The University of Texas MD Anderson Cancer Center, at the American Society for Radiation Oncology (ASTRO 2023) Annual Meeting in San Diego, CA.

The IMMUNOCERV Phase 2 trial is investigating PDS0101 in combination with SOC CRT in the treatment of cervical cancer patients with large tumors over 5 cm in size and/or cancer that has spread to the lymph nodes. HPV is the primary cause of cervical cancer with over 99% caused by HPV infection, and cfHPV-DNA can be detected in the blood of patients with cervical cancer. HPV type 16 (HPV16) is the most prominent subtype associated with cervical cancer. The study presented at ASTRO 2023 evaluated the relationship between the levels of circulating cfHPV-DNA and the extent of disease, clinical staging, and treatment response in patients with HPV-positive cervical cancer. 

“We are encouraged by this data from the IMMUNOCERV trial, which highlight the potential of PDS0101 to positively impact cfDNA, an emerging biomarker of clinical response in cervical and other HPV-related cancers,” said Lauren V. Wood, MD, Chief Medical Officer of PDS Biotech. “The findings complement previously presented IMMUNOCERV data which suggested PDS0101 promotes the induction of multifunctional CD8 killer T cells that were associated with declines in circulating tumor DNA and a clinical response with greater than 60% tumor shrinkage at mid-point evaluation in 100% of high-risk cervical cancer patients on the trial. We look forward to continuing to address the unmet needs of patients suffering from HPV-positive cancers such as cervical cancer.”

Sixty-one patients with cervical cancer were included in the analysis either as part of a SOC treatment banking protocol (n=44) or as part of the IMMUNOCERV Phase 2 clinical trial combining PDS0101 with SOC (n=17). Longitudinal plasma samples were collected from each patient at baseline, during weeks 1, 3, and 5, and at 3-4 months after CRT.

In the study, HPV16 was detected in 59% of tumors and 70% of cfDNA. The median cfDNA at baseline was 28.15 copies/mL, with a range of 0 to 206,030 copies/mL.

The presentation at ASTRO 2023 highlighted the following data:

  • Earlier and greater proportion of cfDNA clearance with PDS0101 plus chemoradiation (CRT) vs. SOC CRT alone (81.3% clearance after 3 weeks vs. 30.3% with SOC (p=0.0018), and 91.7% of clearance at 5 weeks vs. 53.1% with SOC (p=0.0179)
  • Baseline cfDNA levels correlated with the International Federation of Gynecology and Obstetrics (FIGO) stage and lymph node involvement; 100% of patients treated with PDS0101 had cancer that had spread to the lymph nodes

“HPV16 cfDNA represents a novel biomarker with the potential to help oncologists make more informed treatment decisions for their patients with HPV16-positive cancers. This early data are encouraging, and we will continue to evaluate patients to determine any potential correlations between cfDNA clearance and clinical outcomes. Further analysis of cfDNA kinetics could provide valuable information on the relationship between cfDNA levels, treatment response, and ongoing clinical outcomes,” said study principal investigator Ann Klopp, MD, PhD, Professor of Radiation Oncology at MD Anderson. “I look forward to the continued evaluation of PDS0101 in combination with standard-of-care chemoradiotherapy.”

About Versamune®
Versamune® is a novel investigational T cell activating platform which effectively stimulates a precise immune system response to a cancer-specific protein. Versamune® based investigational immunotherapies promote a potent targeted T cell attack against cancers expressing the protein. They are given by subcutaneous injection and can be combined with standard of care treatments. Clinical data suggest that Versamune® based investigational immunotherapies, such as PDS0101, demonstrate meaningful disease control by reducing and shrinking tumors, delaying disease progression and/or prolonging survival. Versamune® based immunotherapies have demonstrated minimal toxicity to date that may allow them to be safely combined with other treatments. We believe Versamune® based investigational immunotherapies represent a transformative treatment approach for cancer patients to provide improved efficacy, safety and tolerability.

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

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

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

Versamune® and Infectimune® are registered trademarks of PDS Biotechnology Corporation. KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.

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

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

Release – Eledon Pharmaceuticals Appoints Kidney Transplant Specialist Dr. Allan Douglas Kirk to Board of Directors

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ELDN

IRVINE, Calif., Oct. 02, 2023 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced the appointment of Allan Douglas Kirk, M.D., Ph.D., to its Board of Directors. Dr. Kirk is internationally recognized for his work in organ transplantation, with a specialized focus on kidney transplants and transplant immunology. He is currently Chairman of the Department of Surgery and Surgeon-in-Chief at Duke University School of Medicine. He is also the David C. Sabiston, Jr. Professor of Surgery, a Professor of Integrative Immunobiology, and a Professor of Pediatrics at Duke.

“We are pleased to have Dr. Kirk join our Board of Directors at a pivotal time at the company following initiation of our Phase 2 BESTOW trial evaluating tegoprubart for the prevention of organ rejection in patients receiving a kidney transplant,” said David-Alexandre C. Gros, M.D., Eledon Chief Executive Officer. “Dr. Kirk’s renowned expertise in the field of organ transplantation, including kidney transplantation, will be incredibly valuable as we advance tegoprubart in clinical development on our mission to fulfill an urgent unmet need for the thousands of patients in need of an organ transplant. I look forward to working closely together.”

“Organ transplantation remains an area of significant unmet need – a need that is often overlooked or underrecognized – and my decades of research have been driven by my desire to improve immunosuppression regimens for transplant patients. Transplant recipients deserve new treatment options with better efficacy and fewer side-effects to help protect their transplanted organs,” said Dr. Kirk. “I am proud to join the Eledon team on their mission to bring patients a novel treatment option that has the potential to transform immunosuppression and improve the lives of patients.”

Dr. Kirk served as the inaugural Chief of the Transplantation Branch for the National Institute of Diabetes and Digestive and Kidney Diseases and has been a principal investigator for multiple clinical trials, including the first-in-human experience with novel immunosuppressive agents. Dr. Kirk has helped pioneer the use of co-stimulation pathway blockade to prevent organ rejection in transplant patients. He has a special interest in immune management of transplant recipients, including therapies that influence T cell co-stimulation pathways and adjuvant therapies that facilitate co-stimulation blockade to prevent the rejection of transplanted organs without undue suppression of protective immunity. His research is directed toward understanding transplant rejection and translating this understanding into improved therapies for transplant recipients. He has co-authored hundreds of peer-reviewed journal publications.

Dr. Kirk is a member of the Duke Transplant Center and is a core faculty member of the Duke Innovation & Entrepreneurship program. He also previously served as Editor-in-Chief for the American Journal of Transplantation. Dr. Kirk earned an M.D. from Duke University School of Medicine, a Ph.D. from Duke University and a B.S. from Old Dominion University.

About Eledon Pharmaceuticals and tegoprubart (formerly AT-1501)

Eledon Pharmaceuticals is a clinical stage biotechnology company with immunology expertise that is developing therapies to protect and prevent rejection of transplanted organs, as well as to treat amyotrophic lateral sclerosis (ALS). The Company’s lead compound in development is tegoprubart, an anti-CD40L antibody with high affinity for CD40 Ligand (also called “CD154”), a well-validated biological target with broad therapeutic potential. 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 planned clinical trials and the Company’s other future expectations, plans and prospects, 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

Cocrystal Pharma (COCP) – CDI-988 Begins Phase 1a/b Clinical Trial

Posted on by cpereira@noblefcm.com


Monday, October 02, 2023

Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

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.

CDI-988 Begins Clinical Studies. Cocrystal announced that CDI-988 has begun Phase 1a/b studies, meeting our expected timeframe. CDI-988 is a 3CL protease inhibitor that interferes with an enzyme early in the viral lifecycle, stopping a virus from reproducing. This early action contrasts with other approaches that seek to kill the virus after it reaches the bloodstream. The study objective is to establish safety, dosing, and pharmacokinetics for design of a Phase 2 study.

Phase 1a/b Study Will Test Ascending Dose Cohorts. The study will have a Phase 1a single ascending-dose stage (SAD) in which patients receive a single administration of CDI-988. As safety is established for each dose, subsequent groups receive higher doses. The Phase 1b stage will have multiple administrations of CDI-988 at ascending doses (MAD). Since CDI-988 is an orally administered vaccine, pharmacokinetic data will include the effect of food drug adsorption. The results of the study will be used to design Phase 2 studies in both norovirus, a frequent cause of acute gastroenteritis, and COVID-19.


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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. 

Baudax Bio (BXRX) – Orphan Drug Designation Granted By FDA for TI-168

Posted on by cpereira@noblefcm.com


Friday, September 29, 2023

Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. ANJESO is the first and only 24-hour, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.

Gregory Aurand, Senior Vice President, Equity Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

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

Treatment of Hemophilia A with inhibitors.  As announced yesterday, the FDA granted Orphan Drug Designation to the Company’s novel treatment. TI-168 is the IND-cleared Treg therapy to address Factor VIII (FVIII) inhibitors in Hemophilia A patients.  Baudax Bio expects to initiate Phase 1/2a trial enrollment early 2024.

Important FDA acknowledgement.  The FDA has authority to grant orphan drug status for new treatments in rare diseases affecting fewer than 200,000 patients in the United States.  The designation qualifies sponsoring companies with qualified clinical trial tax credits, exemption from user fees, potential seven years of market exclusivity after approval, and other benefits. It is estimated that 105,000-165,000 Hemophilia A patients are affected with autoantibody inhibitors in the U.S.


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

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

Release – Baudax Bio Announces Orphan Drug Designation Granted by U.S. FDA for TI-168 for the Treatment of Hemophilia A with Inhibitors

Posted on by aweinsoff@channelchek.com

Research News and Market Data on BXRX

September 28, 2023 8:00am EDT

MALVERN, Pa., Sept. 28, 2023 (GLOBE NEWSWIRE) — Baudax Bio, Inc. (the “Company” or “Baudax Bio”) (NASDAQ: BXRX), a biotechnology company focused on developing T cell receptor (“TCR”) therapies utilizing human regulatory T cells (“Tregs”), as well as a portfolio of clinical stage Neuromuscular Blocking Agents (“NMBs”) and an associated reversal agent, today announced that U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead clinical candidate TI-168 for the treatment of Hemophilia A with inhibitors. TI-168 is the Company’s next-generation, FVIII specific Treg therapy designed to reliably and effectively address Hemophilia A patients with FVIII inhibitors.

“We are very pleased with the FDA’s decision to grant orphan drug designation to TI-168, which we believe highlights the urgent need for innovation and new therapeutic options for Hemophilia A patients,” said Gerri Henwood, President and Chief Executive Officer of Baudax Bio. “We believe this is an important therapeutic area, with established preclinical proof of concept in TI-168 through successes observed in Hemophilia A with inhibitors in animal models. With an Investigational New Drug (IND) application already FDA-cleared, we believe we can activate the Phase 1/2a Clinical Trial of TI-168 for Treatment of hemophilia A with inhibitors with a modest initial budget, and advance this therapy to further clinical investigation in early 2024.”

The FDA’s Office of Orphan Products Development grants orphan status to drugs being developed to treat, diagnose, or prevent a rare disease or condition affecting fewer than 200,000 people in the United States. Orphan Drug Designation is designed to provide drug developers with various benefits to support the development of novel drugs, including the potential for market exclusivity for seven years upon FDA approval, eligibility for tax credits for qualified clinical trials, waiver of application fees, reduced annual product fees, clinical protocol assistance and potential qualification for expedited development programs.

About Baudax Bio

Baudax Bio is a biotech company focused on innovative products for certain auto-immune conditions, of which many but not all, are orphan drug conditions as well as acute care and related settings. The combined company will further the development of Treg therapy specific to HA (pipeline candidate TI-168). TI-168 is a next-generation, FVIII specific Treg therapy designed to reliably and effectively address Hemophilia A patients with FVIII inhibitor. By combining the patented Treg culture method and TeraImmune designed FVIII-specific TCR, the Company has successfully demonstrated the therapeutic concept of FVIII TCR-Treg therapy in controlling of FVIII ADA in a hemophilic animal model. The lead program TI-168 has shown encouraging pre-clinical data and the FDA has cleared an IND to commence a Phase 1/2a clinical trial for the treatment of Hemophilia A with inhibitors.

In addition, over time, the combined company will advance the development of TeraImmune’s innovative immune-cell therapies, leveraging a dual Treg manufacturing platform consisting of both natural regulatory Tregs isolated from patients and induced Tregs converted from a patient’s T-effector (“Teff”) cells. This Treg platform technology is designed for conditions that suppress unwanted immune reactions and includes the allogenic, or off-the-shelf, Tregs obtained from Umbilical Cord Blood for the treatment of skin diseases such as Atopic Dermatitis. For more information, please visit www.baudaxbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements reflect Baudax Bio’s expectations about its future performance and opportunities that involve substantial risks and uncertainties. When used herein, the words “anticipate,” “believe,” “estimate,” “may,” “upcoming,” “plan,” “target,” “goal,” “intend,” and “expect,” and similar expressions, as they relate to Baudax Bio, are intended to identify such forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on Baudax Bio’s current beliefs, expectations and assumptions regarding the future of its business, future plans and strategies, clinical results and other future conditions. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption “Risk Factors” in Baudax Bio’s most recent Annual Report on Form 10-K filed with the SEC and its subsequent filings with the SEC. Any forward looking statement speaks only as of the date on which it was made. Neither Baudax Bio, nor any of its affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing Baudax Bio’s views as of any date subsequent to the date hereof.

Investor Relations Contact:

Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com

Source: Baudax Bio

Released September 28, 2023

Eledon Pharmaceuticals (ELDN) – Initiating Coverage With An Outperform Rating and $10 Price Target

Posted on by cpereira@noblefcm.com


Wednesday, September 27, 2023

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

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

We Are Initiating Coverage Of EledonPharmaceuticals. Eledon is developing tegoprubart, an anti-CD40L antibody to modulate the immune response. Phase 1b and Phase 2 clinical trials in progress for prevention of kidney transplant rejection. The clinical trial program is designed to show superiority to the immunosuppressive drugs that are currently the standard of care after organ transplants.

Tegoprubart Blocks CD40/CD40L. Tegoprubart blocks the step in the immune response where the CD40 ligand (CD40L, also known as CD154) on T cells interacts with the CD40 receptor on either B cells or antigen-presenting cells. This binding connects the innate and adaptive immune systems and leads to a full immune response. Tegoprubart binds to the CD40L to block this step, inhibiting both B cell maturation and T-cell proliferation that leads to cellular immune responses.


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Alfasigma Makes Big Bet on Liver Disease With $1.25 Billion Intercept Buyout

Posted on by slightbourne@noblecapitalmarkets.com

Italian pharma Alfasigma is expanding its gastroenterology portfolio in a major way with the proposed $1.25 billion acquisition of Intercept Pharmaceuticals. The all-cash deal provides Alfasigma with Intercept’s leading drug Ocaliva and a strengthened pipeline in progressive liver diseases.

Alfasigma will pay $19 per share to acquire Intercept, representing an 82% premium over Intercept’s share price before the deal announcement. The purchase price reflects a big bet on Ocaliva’s growth prospects and Intercept’s broader capabilities in rare liver conditions.

Ocaliva is the key asset Alfasigma gains from the deal. It’s the only FDA approved second-line treatment for primary biliary cholangitis (PBC), a progressive autoimmune disorder that damages the bile ducts in the liver. Ocaliva hit $152 million in sales over the first half of 2023 alone, underscoring its rapid growth trajectory.

Beyond Ocaliva, Alfasigma also adds Intercept’s emerging pipeline of novel therapies for PBC and other liver diseases. The crown jewel is a promising fixed-dose combination regimen that could transform the PBC treatment paradigm.

Take a look at Noble Capital Markets Senior Life Sciences Analyst Robert LeBoyer’s coverage universe.

The deal dramatically expands Alfasigma’s presence in the high-value U.S. pharma market. Previously focused primarily on the Italian market, the Intercept acquisition gives Alfasigma an anchor asset and commercial team in the U.S.

Strategically, the move aligns with Alfasigma’s vision to build up its gastroenterology and hepatology business. CEO Francesco Balestrieri highlighted Intercept’s compelling strategic fit with Alfasigma’s focus in these therapeutic areas.

Expect Alfasigma to invest heavily to maximize Ocaliva’s potential. The company sees major commercial expansion opportunities to extend Ocaliva’s reach across PBC patient populations. Alfasigma also gains Intercept’s seasoned specialty sales force to drive prescription growth.

With Intercept operating as a wholly-owned subsidiary once the buyout closes, Alfasigma is well-positioned to become a global force in progressive liver diseases. The deal enhances Alfasigma’s standing as an emerging player in the U.S. pharma market.

Look for Alfasigma to continue seeking acquisition targets to accelerate its growth. The company has the financial firepower to pursue additional deals that build up its portfolio. If the Intercept acquisition is any indication, Alfasigma has appetite for bold, transformative M&A.

The proposed buyout still requires regulatory and shareholder approval. But with a massive 82% premium offered, Intercept shareholders are likely to approve the $19 per share deal price. Expect Alfasigma to move rapidly to complete the acquisition by the end of 2023.

Soleno Therapeutics Stock Skyrockets 505% on Hunger Pill Promise

Posted on by slightbourne@noblecapitalmarkets.com

Shares of Soleno Therapeutics (SLNO) catapulted an astonishing 505% higher last week after positive late-stage clinical results for the company’s experimental obesity and hunger control pill. The massive stock surge demonstrates investor enthusiasm around Soleno’s DCCR pill as a potential new medicine for Prader-Willi syndrome (PWS).

DCCR aims to treat hyperphagia, or abnormally increased hunger and food drive, in patients with PWS. The rare genetic disorder can lead to excessive eating, obesity, and other complications. Soleno’s pill showed promising ability to control hunger signals in PWS patients during a 4-month placebo-controlled withdrawal study.

The trial enrolled 77 PWS patients who had been taking DCCR for 2-4 years. Half the participants continued their DCCR regimen, while the other half were switched to placebo. After just one month, the placebo group showed a rapid return of hyperphagia symptoms. On a 9-point clinical scale, their hunger scores increased by a significant 5 points on average.

In contrast, PWS patients who remained on DCCR maintained stable, controlled hunger levels throughout the 4-month trial. The results provide strong evidence that DCCR specifically curbs abnormal hunger drive in PWS, rather than just having a placebo effect.

Beyond hunger control, PWS patients taking placebo also showed worsening in overall PWS symptoms and severity. No new safety issues emerged during the 4 months on placebo, indicating the hunger control benefits of DCCR remained after stopping treatment.

Armed with the successful phase 3 results, Soleno now plans to submit a New Drug Application for DCCR to the FDA in mid-2024. The company is seeking an initial approval in PWS, which would make DCCR the first ever pharmacotherapy for hyperphagia associated with the condition.

The FDA previously designated DCCR an Orphan Drug for treating PWS. This status provides incentives to develop medicines for rare diseases, since PWS affects only about 15,000-16,000 individuals in the U.S. The new trial results could support approval based on the FDA’s prior guidance.

If greenlighted, analysts project DCCR peak sales could reach $500 million – $1 billion annually. The pill would provide a novel, non-surgical therapeutic option to manage hyperphagia and obesity in PWS. DCCR’s unique clinical benefits could command premium pricing above $100,000 per patient annually.

Soleno still has work ahead to realize commercialization, including securing FDA approval and payer coverage. But the company now has a de-risked late stage asset and clear regulatory path forward. The 505% share price spike last week reflects renewed investor confidence in DCCR’s commercial potential.

Despite the rally, Soleno still trades 70% below its 2018 highs. The stock has suffered from past clinical setbacks that stoked skepticism around DCCR. But with a successful phase 3 trial now completed, the tide may be turning.

Soleno could seek to expand DCCR’s use into additional patient populations with uncontrolled hunger and obesity issues beyond PWS. The pill works by modulating metabolic pathways that drive hyperphagia symptoms.

While gains may moderate going forward, last week’s huge rally kicks off a new chapter for Soleno stock. Look for the company to shift focus from pivotal trial execution to commercial planning. Investors cheered the pivotal data, signaling confidence in DCCR’s prospects. Soleno still has work left to turn its hunger-controlling pill into an FDA-approved medicine, but the 505% surge shows just how much upside investors anticipate.

Release – Tonix Pharmaceuticals Announces Initiation of Enrollment in the MGH Phase 2 ‘POWER’ Study of TNX-1900 (Intranasal Potentiated Oxytocin) for the Treatment of Pediatric Obesity

Release – GeoVax Secures Multi-Product License for ProBioGen’s AGE1.CR.pIX® Suspension Cell Line to Bolster MVA-Based Vaccine Development

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

ATLANTA, GA and BERLIN, Germany, September 26, 2023 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing vaccines and immunotherapies against infectious diseases and cancers, and ProBioGen announce the signing of a landmark commercial license agreement for ProBioGen’s groundbreaking AGE1.CR.pIX® suspension cell line. The agreement will empower GeoVax to enhance the manufacturing capabilities of its entire Modified Vaccinia Ankara (MVA) based vaccine portfolio.

ProBioGen’s AGE1.CR.pIX suspension cell line is an innovative and proven platform that enables high-yield and scalable production, ensuring efficient industrial manufacturing processes. This translates to cost-effectiveness and increased productivity for vaccine developers. The AGE1.CR.pIX cell line’s versatility allows it to support a wide range of viruses and vaccine types, enhancing its suitability for various vaccines in development and as a replacement for traditional production systems. Additionally, AGE1.CR.pIX’s robust growth and genetic stability ensure consistent and reliable production, leading to the delivery of safe and effective vaccines. MVA grows particularly well on this cell line, making it even more advantageous for vaccine development. By leveraging this modern production technology, GeoVax aims to accelerate the manufacturing of its entire vaccine pipeline. This multi-product license represents a strong commitment to improving global access to life-saving vaccines.

“We are thrilled to enter into this licensing agreement with ProBioGen”, said David Dodd, Chairman & CEO of GeoVax. “The AGE1.CR.pIX suspension Cell Line is a game-changer for our vaccine production, allowing us to streamline our manufacturing processes while maintaining the highest quality standards. Development of a high-yield, high-capacity process to produce MVA-based vaccines and immunotherapies is nothing short of transformational, and by advancing our MVA manufacturing to a modern, interchangeable process, we are on course to expand MVA applications from stockpile-based solutions for niche medical markets to respond to world needs on a timely basis, whenever and wherever they arise. This partnership with ProBioGen aligns perfectly with GeoVax’s mission of developing safe and effective vaccines to protect public health.”

“ProBioGen is equally excited about the collaboration, recognizing the potential impact of their technology on global health. GeoVax, as one of the key MVA companies, will strengthen its innovative vaccines with the benefit of a straightforward manufacturing solution”, said Dr. Volker Sandig, CSO of ProBioGen. “We believe this partnership will significantly contribute to GeoVax’s ability to accelerate vaccine production and distribution, ultimately benefiting communities worldwide.”

Financial terms of the commercial license to use the AGE1.CR.pIX, including potential clinical milestones and future royalties were not disclosed.

About ProBioGen

ProBioGen is a premiere, Berlin-based specialist for developing and manufacturing biopharmaceutical active ingredients, viral vectors and vaccines with applying proprietary technologies to improve product quality and features. Combining both state-of-the-art development services, together with intelligent product-specific technologies yields biologics with optimized properties. Rapid and integrated cell line and process development, comprehensive analytical development and GMP-compliant manufacturing is performed by a highly skilled and experienced team. All services and technologies are embedded in a total quality management system to assure compliance with international ISO and GMP standards (EMA/FDA).

ProBioGen has been operational for almost 30 years. At three locations in Berlin, over 300 employees contribute to the creation of new therapies in medicine and groundbreaking innovations worldwide through their creative and meticulous work. ProBioGen’s growth strategy is driven by the expansion of the service value chain through organic growth and potential acquisition. Diversification is a complement driver, while the focus is strict on enabling the development of biopharmaceuticals for tomorrow.

ProBioGen’s AGE1.CR.pIX cell line is derived from primary cells of a duck embryo and was designed to comply with health authority guidelines and the concept of “defined risk”. It was developed as an alternative to the use of chicken eggs for large-scale vaccine production. The AGE1.CR.pIX cell line grows in true suspension and has been optimized for viral vaccine production and stability. It grows in a commercially available, chemically defined medium without animal components and is an excellent host for a variety of different virus strains.

For more information about ProBioGen, follow us on LinkedIn.

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.

GeoVax 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.

ProBioGen Contact:         ProBioGen Press Contact:
Dr. Gabriele SchneiderLisa Petereit
Chief Business Officer Corporate Communications
cdmo@probiogen.de press@probiogen.de
   
   
GeoVax Contact: GeoVax Press Contact:
info@geovax.com  Susan Roberts
678-384-7220 sr@roberts-communications.com
  202-779-0929

Regeneron Strengthens Gene Therapy Pipeline Through Acquisition of Decibel Therapeutics

Posted on by slightbourne@noblecapitalmarkets.com

Regeneron Pharmaceuticals has expanded its gene therapy programs by acquiring Decibel Therapeutics, a biotech company focused on developing treatments for hearing loss. The $1.1 billion deal provides Regeneron with three promising gene therapy candidates that use adeno-associated virus (AAV) vectors to restore hearing.

The most advanced asset is DB-OTO, an AAV-based gene therapy designed to provide long-term hearing to individuals with profound congenital hearing loss caused by otoferlin gene mutations. DB-OTO is currently being evaluated in a Phase 1/2 clinical trial known as CHORD. The gene therapy aims to deliver a functional copy of the otoferlin gene to inner ear hair cells, potentially enabling hearing restoration.

The acquisition also includes two earlier-stage gene therapies, AAV.103 and AAV.104, targeting other genetic forms of hearing loss – GJB2 and STRC respectively. Both utilize a similar AAV gene delivery approach to DB-OTO.

According to Regeneron, the addition of Decibel’s pipeline and capabilities will strengthen its genetic medicines portfolio. Gene therapy has become a major focus for Regeneron beyond its foundational expertise in antibodies. The company is exploring gene silencing, gene editing and gene therapy technologies across a range of therapeutic areas.

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

Hearing loss represents a new area for Regeneron, building on an existing collaboration with Decibel. Integration of Decibel’s team and experience in inner ear biology and AAV gene therapy for hearing disorders will be invaluable as Regeneron advances the acquired programs.

Gene therapy aims to address disease at its genetic root cause by introducing functional genes into cells. The goal is to durably restore protein expression and correct the downstream impacts of gene mutations. Gene therapy has shown promise for treating rare monogenic disorders like certain forms of inherited hearing loss.

Both Regeneron and Decibel have utilized AAV vectors to deliver gene therapy payloads. AAV is considered one of the most effective vehicles for gene delivery and has an established safety profile. The viruses can be engineered to target specific cell types following injection into the body.

For DB-OTO, the AAV vector carries a functional copy of the otoferlin gene. Inner ear hair cells are the targets for gene transduction. Otoferlin protein is critical for hearing signal transduction, but mutations in the encoding gene cause profound congenital deafness. Gene therapy aims to restore otoferlin expression and regain hearing function.

Regeneron’s push into gene therapy aligns with its mission of tackling serious diseases with novel technologies. Gene-based treatments have potential for one-time curative therapies. The acquisition of Decibel’s pipeline further diversifies Regeneron’s genetic medicine capabilities as it aims to help patients worldwide.

Release – Eledon Pharmaceuticals Announces Use of Tegoprubart anti-CD40L Antibody in Second-ever Transplant of Genetically Modified Heart from a Pig to a Human

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ELDN

Landmark cardiac xenotransplantation procedure conducted at University of Maryland Medical Center

Tegoprubart, administered investigationally to prevent organ rejection post-transplant, targets the CD40L pathway known to play an essential role in both innate and adaptive immune cell activation and function.

IRVINE, Calif., Sept. 25, 2023 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that tegoprubart, the company’s investigational anti-CD40 antibody, was used as a cornerstone component of the chronic immunosuppressive regimen administered following the second-ever transplant of a genetically modified heart from a pig to a human. The procedure was completed on September 20th at University of Maryland Medical Center on a 58-year-old male suffering from heart failure.

“This is a momentous milestone for the transplant community and serves as a testament to the continued progress being made towards advancing novel treatment options for patients requiring an organ transplant,” said David-Alexandre C. Gros, M.D., Eledon Chief Executive Officer. “As the field of organ transplantation continues to make important scientific advances, Eledon is dedicated to delivering a novel immunosuppressive regimen with the potential to protect and prevent rejection of transplanted organs, and we are honored to play a role in this historic development.”

Following the successful transplantation, tegoprubart was administered to the patient as a novel, key component of a chronic immunosuppressive regimen designed to suppress the immune system and prevent the body from rejecting the implanted organ. In prior clinical research, tegoprubart has demonstrated a favorable safety and tolerability profile across multiple indications, including kidney transplantation, as well as clinical benefit in the prevention of rejection and the protection of organs after transplantation.

“The historic procedure we conducted on our courageous patient brings us to a pivotal moment in the history of organ transplantation. It is also a critical step in our ongoing mission to address the growing shortage of available organs,” said Muhammad M. Mohiuddin, MD, professor of surgery at University of Maryland School of Medicine (UMSOM) and scientific/program director of its Cardiac Xenotransplantation Program. “The ability to expand options in all areas including access to available organs and strategies to reduce the risk of rejection means that we are getting closer to realizing the full potential of transplantation for patients. I look forward to continued advancements so that we can hopefully make xenotransplantation an available organ source for patients in the years ahead.”

Eledon is advancing multiple research efforts related to the use of tegoprubart to reduce the risk of rejection in organ transplant. In collaboration with eGenesis, the company is currently advancing preclinical studies in which tegoprubart will be administered as part of an immunosuppression regimen to reduce the risk of rejection in nonhuman primate recipients in xenotransplant procedures. The company recently announced initiation of patient dosing in the phase 2 BESTOW clinical trial to further assess the use of tegoprubart in kidney transplantation and expects to present an updated readout from its ongoing phase 1b kidney transplantation study in November 2023. BESTOW is a head-to-head superiority study evaluating tegoprubart vs. standard of care in kidney transplantation, with a primary endpoint assessment of kidney graft function (eGFR) at 52 weeks.

The Risk of Organ Failure in Transplantation

In transplantation procedures, organ rejection is a major cause of graft failure which can be a life-threatening condition. To reduce the risk of organ damage and rejection, patients are typically treated with immunosuppressive therapies including calcineurin inhibitors (CNIs) such as tacrolimus. Strategies to better and more safely protect transplanted organs and thus increase how long they function represent a significant area of unmet need in organ transplantation.

About Eledon Pharmaceuticals and Tegoprubart (formerly AT-1501)

Eledon Pharmaceuticals is a clinical stage biotechnology company with immunology expertise that is developing therapies to protect and prevent rejection of transplanted organs, as well as to treat amyotrophic lateral sclerosis (ALS). The Company’s lead compound in development is tegoprubart, an anti-CD40L antibody with high affinity for CD40 Ligand (also called “CD154”), a well-validated biological target with broad therapeutic potential. 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 planned clinical trials and the Company’s other future expectations, plans and prospects, 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