Tag: Biotech

Novartis to Acquire Regulus Therapeutics in $1.7 Billion Biotech Buyout

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– Novartis to acquire Regulus for up to $1.7B, including $7/share upfront and $7/share tied to farabursen approval.
– Farabursen, a potential first-in-class ADPKD treatment, heads into Phase 3 with FDA alignment.
– Boosts Novartis’s kidney disease pipeline and commitment to innovation in rare conditions.

Novartis AG announced plans to acquire Regulus Therapeutics Inc. in a transaction valued at up to $1.7 billion, reinforcing the Swiss pharmaceutical giant’s strategy to deepen its portfolio in renal and genetic disease treatments. The deal includes an upfront cash payment of $7.00 per share, representing approximately $800 million in equity value, and an additional $7.00 per share tied to a regulatory milestone via a contingent value right (CVR), pending approval of Regulus’s lead drug candidate, farabursen.

Farabursen is being developed as a novel treatment for autosomal dominant polycystic kidney disease (ADPKD), a condition with limited current options and significant unmet clinical need. If approved, farabursen could become the first systemic therapy of its kind in this indication, offering a potentially superior safety and efficacy profile compared to existing treatments.

The acquisition reflects a growing trend in the biopharma sector where large-cap pharmaceutical companies pursue innovative pipelines through targeted M&A. In recent quarters, the industry has seen an uptick in transactions focused on small to mid-sized biotech firms that specialize in high-impact therapies for rare or underserved diseases. Regulus’s focus on microRNA-based therapies, a field once viewed as experimental, is now receiving renewed attention as advances in RNA technology improve target precision and therapeutic delivery.

For Novartis, the move expands its nephrology franchise and bolsters its pipeline in genetic disorders, aligning with the company’s long-term innovation strategy. Financially, the deal signals confidence in both Regulus’s platform and farabursen’s development prospects. The 274% premium to Regulus’s 60-day volume-weighted average price underscores the strategic value Novartis sees in the program.

The transaction is expected to close in the second half of 2025, subject to regulatory approval and the successful tender of a majority of Regulus’s outstanding shares. Once finalized, Regulus will become a wholly owned subsidiary of Novartis, with its operations and development programs integrated into Novartis’s global R&D structure.

The deal may also serve as a bellwether for continued consolidation in biotech, particularly among companies advancing oligonucleotide or RNA-based therapeutics. Investors are likely to see the acquisition as further validation of microRNA platforms, potentially reinvigorating interest in similar early-stage biotech firms.

At a time when cost pressures and generic competition are accelerating across the pharmaceutical landscape, acquiring promising assets with a clear regulatory path remains a preferred strategy for growth. For Regulus, integration with Novartis offers the financial and operational muscle needed to take farabursen through the final stages of development and, if approved, to global markets.

As the biotech sector continues to recalibrate from recent valuation contractions, strategic acquisitions like this illustrate the enduring value of focused innovation, especially in areas with limited treatment alternatives and high unmet demand.

Release – Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2025 Annual Meeting and Retina World Congress

Posted on by slightbourne@noblecapitalmarkets.com

Research News and Market Data on Ocugen

April 29, 2025

MALVERN, Pa., April 29, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410ST for the treatment of Stargardt disease (Phase 2/3 pivotal confirmatory clinical trial), and OCU410 for the treatment of geographic atrophy (Phase 2 ArMaDa clinical trial), at The Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting at the Calvin L. Rampton Salt Palace Convention Center in Salt Lake City, Utah from May 4-8, 2025, and Retina World Congress at the Marriott Harbor Beach Resort in Ft. Lauderdale, Florida from May 8-11, 2025.

“We look forward to sharing more about the potential of our modifier gene therapy platform and the meaningful results we are seeing in the clinic during these two important meetings for the retina community,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “Ocugen remains on track to deliver on our commitment to file three Biologics License Applications (BLAs)/Marketing Authorization Applications (MAAs) in the next three years—potentially addressing significant unmet medical need for large patient populations through our gene-agnostic approach.”

The ARVO Annual Meeting is a premiere gathering for eye and vision scientists from across the globe, students, and those in affiliated fields to share the latest research findings and collaborate on innovative solutions. Retina World Congress brings together leading retina specialists from every continent to achieve a global scientific and clinical exchange in retinal health.

Ocugen’s presence in Utah kicks off with the Company Showcase at Eyecelerator, presented by Dr. Huma Qamar, Chief Medical Officer at Ocugen, and continues through presentations and thought leadership engagement at ARVO.

Eyecelerator @ Park City 2025

Session: Retina—Gene Therapy and Novel Mechanisms of Action Showcase
Location: Grand Hyatt Deer Valley, Strawberry Ballroom, Park City, UT
Date: Friday, May 2, 2025
Time: 2:06 p.m. MDT
Presenter: Dr. Huma Qamar

ARVO

Exhibitor Education Forum
Two-Year Follow-Up of a Phase 1/2 Clinical Trial for the Safety and Efficacy of OCU400 Novel Modifier Gene Therapy for Retinitis Pigmentosa
Location: Exhibitor Floor, Section 1037
Date: Monday, May 5, 2025
Time: 2 p.m. MDT 
Presenter: Benjamin Bakall, MD, Ph.D., Assistant Clinical Professor, University of Arizona, College of Medicine–Phoenix, and Director for Clinical Research, Director for The Inherited Retinal Disease and Visual Function Clinic, Associated Retina Consultants

An Evaluation of the Safety and Efficacy of Novel Modifier Gene Therapy OCU410 for the Treatment of Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration
Location: Exhibitor Floor, Section 1037
Date: Tuesday, May 6, 2025
Time: 2 p.m. MDT
Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin

Safety and Efficacy of OCU410ST: A Phase 1/2 Trial of a Novel Modifier Gene Therapy for Stargardt Disease (GARDian)
Location: Exhibitor Floor, Section 1037
Date: Wednesday, May 7, 2025
Time: 2 p.m. MDT
Presenter: Neena Haider, Ph.D., Faculty Harvard Medical School and Founder, CEO, Shifa Precision

Paper Session
Preliminary Safety and Efficacy of OCU410 for Treatment of Geographic Atrophy: Phase 1/2 OCU410: The Age-related Macular Degeneration (ArMaDa) Study Update
Presentation Number: 3675 
Session Number and Title: 358/AMD Clinical research II 
Location: Ballroom J 
Date: Tuesday, May 6, 2025
Time: 4:15 p.m. MDT
Presenter: Syed M. Shah, MD, FACS, Vice Chair for Research and Digital Medicine, Director of Retina Service at Gundersen Health System, La Crosse, Wisconsin

Poster Session
A0513: Safety and Efficacy of OCU410ST for the Treatment of Stargardt Disease: Phase 1/2 Study Update
Location: Hall A-E
Date: Thursday, May 8, 2025
Time: 2 p.m. MDT
Presenter: Ramiro Maldonado, MD, Duke Center for Ophthalmic Genetics, Duke Pediatric Retina, Adult vitreo-Retinal diseases

Dr. Qamar will represent Ocugen at Retina World Congress to share the Company presentation and serve alongside notable retinal surgeons and industry peers during a panel discussion.

Retina World Congress

Retina Unplugged
Inherited and Rare Retinal Diseases Session
Moderators: Rishi P. Singh, MD, FASRS and Kourous A. Rezaei, MD
Location: Grand Ballroom
Date: Thursday, May 8, 2025
Time: 10:35 am – 11:12 a.m. EDT

Ocugen is committed to bringing game-changing therapies to treat inherited retinal diseases as well as blindness diseases affecting millions to market and working even harder to provide access to patients globally.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Longevity Health Merges with 20/20 BioLabs in Bid to Redefine Healthy Aging

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– Longevity Health and 20/20 BioLabs to merge, forming a $99M company focused on diagnostics and healthy aging.
– 2025 revenue expected to double post-merger, driven by cross-sell opportunities and product synergies.
– Combined firm targets expanding into MedSpas, retail, and clinical settings, reflecting a hybrid approach to wellness and diagnostics.

Longevity Health Holdings (Nasdaq: XAGE) is doubling down on its ambition to lead the healthy aging and diagnostics market with the announcement of a strategic all-stock merger with 20/20 BioLabs, a provider of cutting-edge diagnostic tests for early cancer detection and chronic disease risk management. The deal, which is expected to close in Q3 2025, marks another step in Longevity’s pivot toward becoming a vertically integrated longevity-focused healthcare platform.

The merger comes just months after Longevity’s acquisition of Elevai Skincare and follows the company’s March 2025 announcement outlining a broader strategy to combine diagnostics, bio-aesthetics, and nutrition under the unifying theme “Healthy Aging, Inside and Out™.” With 20/20’s technology and distribution capabilities, Longevity is adding a diagnostics engine to its growing wellness infrastructure and positioning itself as a unique player at the intersection of science, skincare, and preventative healthcare.

Under the terms of the agreement, 20/20 shareholders will own approximately 50.1% of the combined company, with Longevity shareholders retaining 49.9%—a sign of parity and the significance of what 20/20 brings to the table. The merged company will continue to trade under the ticker “XAGE” on the Nasdaq.

Founded in Gaithersburg, Maryland, 20/20 operates a CLIA-licensed and CAP-accredited laboratory and has developed OneTest™, a multi-cancer early detection (MCED) blood test capable of identifying over a dozen tumor types for under $200. The company has already integrated its tests into wellness protocols for firefighters and military veterans and is preparing to launch a new “longevity test” this spring that evaluates inflammatory markers tied to aging and disease risk.

Financially, the merger is set to double Longevity’s expected revenue for fiscal year 2025 from $3–4 million to $7–8 million and deliver at least $1 million in operational synergies. The combined company’s equity valuation is pegged at $99 million, offering a promising growth profile in a market that increasingly values integrated health solutions.

For small-cap investors, the deal highlights an emerging investment theme: convergence in wellness, biotech, and diagnostics. Longevity is carving out a niche in a crowded but high-potential market by integrating scientific, consumer-facing products with medical-grade diagnostics. This cross-disciplinary approach could make it more resilient than standalone players focused solely on aesthetics or lab testing.

Beyond the numbers, Longevity plans to offer 20/20’s tests through its network of physicians to inform more personalized bio-aesthetic treatment plans. Conversely, 20/20 will gain access to Longevity’s customer base—including thousands of firefighters—to introduce its diagnostics in new environments, including MedSpas and retail.

Leadership will be shared post-merger. Longevity’s Rajiv Shukla will remain Chairman, while 20/20’s Jonathan Cohen will step in as CEO, underscoring a collaborative transition.

As Longevity eyes further acquisitions, this deal positions it as a unique micro-cap consolidator in the rapidly evolving healthy aging space. Investors should watch closely as the company scales up from niche science to potentially mass-market longevity solutions.

Release – Gyre Therapeutics Announces Publication of Protocol for Phase 3 Trial Evaluating F351 for CHB-Associated Liver Fibrosis in Journal of Clinical and Translational Hepatology

Posted on by slightbourne@noblecapitalmarkets.com

Research News and Market Data on Gyre Therapeutics

March 27, 2025

PDF Version

SAN DIEGO, March 27, 2025 (GLOBE NEWSWIRE) — Gyre Therapeutics (“Gyre”) (Nasdaq: GYRE), an innovative, commercial-stage biotechnology company with clinical development programs focusing on organ fibrosis, today announced the publication of the manuscript titled “Hydronidone for the Treatment of Liver Fibrosis Associated with Chronic Hepatitis B: Protocol for a Phase 3 Randomized Trial” in the Journal of Clinical and Translational Hepatology. This publication details the full protocol for the pivotal Phase 3 trial to support the use of hydronidone in Chinese patients with liver fibrosis associated with chronic hepatitis B (“CHB”). The published protocol outlines patient inclusion criteria, randomization and blinding processes, key assessments, and the statistical analysis plan.

The randomized, double-blind, placebo-controlled, multicenter Phase 3 trial (NCT05115942) completed the enrollment of 248 patients across 44 clinical research hospitals in the People’s Republic of China (“PRC”) in October 2024. Patients were randomized 1:1 to receive either F351 or placebo in addition to entecavir antiviral basic therapy for CHB. The primary endpoint is a decrease in liver fibrosis (as measured by the Ishak Scoring System) by at least one stage after 52 weeks of treatment relative to baseline.

The PRC’s National Medical Products Administration (“NMPA”) designated F351 as a “Breakthrough Therapy” in 2021. Gyre expects to report topline results from this Phase 3 trial in the second quarter of 2025.

About Hydronidone (F351)

F351 is a next-generation anti-fibrotic compound and a structural analogue of Pirfenidone, the first approved treatment for idiopathic pulmonary fibrosis (“IPF”) in Japan, the European Union, the United States and the PRC. F351’s dual mechanism has been shown to inhibit in vitro p38γ kinase activity and TGF-β1-driven collagen overproduction—both key drivers of liver fibrosis. F351 specifically targets hepatic stellate cells (“HSCs”), which are central to the progression of fibrosis. In preclinical studies, it has shown strong anti-proliferative and anti-fibrotic effects on HSCs. These effects have been validated across multiple in vivo models of liver fibrosis, including CCl4-induced liver fibrosis in mice, DMN- and HSA-induced liver fibrosis in rats, and a mouse model of MASH fibrosis (a form of MASH with fibrosis) induced by CCl4 plus a Western high-fat diet. Together, these results highlight F351’s potential as a differentiated treatment for liver fibrosis, with a unique mechanism and strong preclinical and clinical evidence supporting its advancement into later-stage development.

About Gyre Pharmaceuticals

Gyre Pharmaceuticals is a commercial-stage biopharmaceutical company committed to the research, development, manufacturing and commercialization of innovative drugs for organ fibrosis. Its flagship product, ETUARY® (Pirfenidone capsule), was the first approved treatment for IPF in the PRC in 2011 and has maintained a prominent market share (2024 net sales of $105.8 million). In addition, Gyre Pharmaceuticals is evaluating F351 in a Phase 3 clinical trial in CHB-associated liver fibrosis in the PRC, which is expected to readout topline data by Q2 2025. F351 received Breakthrough Therapy designation by the NMPA Center for Drug Evaluation in March 2021. Gyre Pharmaceuticals is also developing treatments for PD, DKD, COPD, PAH and ALF/ACLF. In October 2023, Gyre Therapeutics acquired an indirect majority interest in Gyre Pharmaceuticals (also known as Beijing Continent Pharmaceuticals Co., Ltd.).

About Gyre Therapeutics

Gyre Therapeutics is a biopharmaceutical company headquartered in San Diego, CA, with a primary focus on the development and commercialization of F351 (Hydronidone) for the treatment of MASH-associated fibrosis in the U.S. Gyre’s development strategy for F351 in MASH is based on the company’s experience in MASH rodent model mechanistic studies and CHB-induced liver fibrosis clinical studies. Gyre is also advancing a diverse pipeline in the PRC through its indirect controlling interest in Gyre Pharmaceuticals, including ETUARY therapeutic expansions, F573, F528, and F230.

Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, which statements are subject to substantial risks and uncertainties and are based on estimates and assumptions. All statements, other than statements of historical facts included in this press release, are forward-looking statements, including statements concerning: the expectations regarding Gyre’s research and development efforts and timing of expected clinical readouts, including timing of topline data from Gyre Pharmaceuticals’ Phase 3 clinical trial evaluating F351 for the treatment of CHB-associated liver fibrosis in the PRC. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements reflect our plans, estimates, and expectations, as of the date of this press release. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this press release. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: Gyre’s ability to execute on its clinical development strategies; positive results from a clinical trial may not necessarily be predictive of the results of future or ongoing clinical trials; the timing or likelihood of regulatory filings and approvals; competition from competing products; the impact of general economic, health, industrial or political conditions in the United States or internationally; the sufficiency of Gyre’s capital resources and its ability to raise additional capital. Additional risks and factors are identified under “Risk Factors” in Gyre’s Annual Report on Form 10-K for the year ended December 31, 2024 filed on March 17, 2025 and in other filings the Company may make with the Securities and Exchange Commission.

Gyre expressly disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

For Investors:
Stephen Jasper
stephen@gilmartinir.com

FOXO Technologies Signs Non-Binding Agreement to Acquire Vector Biosource

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– FOXO Technologies has signed a non-binding agreement to acquire Vector Biosource, a biospecimen sourcing provider.
– Vector is expected to generate $800,000 in revenue in 2025 without additional capital.
– The acquisition involves Series D Preferred Stock and milestone-based earnout payments.

FOXO Technologies Inc. (NYSE American: FOXO) has announced the execution of a non-binding agreement to acquire Vector Biosource Inc., a provider of information and biospecimen sourcing services for the biotechnology, clinical research, and pharmaceutical industries. The acquisition aligns with FOXO’s strategy of expanding its footprint in healthcare and biotechnology sectors.

The proposed transaction includes an initial payment of $750,000 in Series D Cumulative Redeemable Preferred Stock, with an additional $750,000 in Series D Preferred Stock contingent on Vector meeting specific revenue and cash collection milestones in 2025. Further earnout payments in Series D Preferred Stock are structured based on Vector’s performance in 2026 and 2027. The deal remains subject to definitive agreements, due diligence, and the provision of $1 million in working capital.

Seamus Lagan, CEO of FOXO Technologies, emphasized the strategic benefits of the deal, stating, “We are excited to have reached agreement with Vector to move forward with this strategic acquisition. We were attracted to Vector’s unique position in this healthcare sector and its growth profile, and we are focused on working closely with Vector senior leadership to aggressively expand the Vector platform.”

Vector’s CEO, Frank Dias, Jr., highlighted the advantages of the partnership, noting, “We believe the partnership with FOXO will allow Vector to achieve its near and long-term growth plans by providing growth capital, corporate infrastructure, and potential synergies with other FOXO subsidiaries. We anticipate a significant increase in expected revenues with the provision of growth capital and corporate infrastructure by FOXO.”

FOXO Technologies operates through three subsidiaries:

  • Rennova Community Health, Inc.: Owner and operator of Scott County Community Hospital (Big South Fork Medical), a critical access hospital in East Tennessee.
  • Myrtle Recovery Centers, Inc.: A 30-bed behavioral health facility offering inpatient detox, residential treatment, and outpatient services.
  • Foxo Labs, Inc.: A biotechnology company dedicated to advancing health and lifespan through innovative technology and product solutions.

The acquisition of Vector Biosource marks another step in FOXO’s broader growth strategy as it continues to integrate specialized healthcare and biotechnology services under its corporate umbrella. The deal is expected to close within the next 45 days, subject to regulatory approvals and standard closing conditions.

Take a moment to take a look at Noble Capital Markets’ biotechnology and life sciences research analyst Robert Leboyer’s coverage list.

AstraZeneca Strengthens Cell Therapy Portfolio with $1B EsoBiotec Acquisition

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– AstraZeneca is acquiring Belgium-based EsoBiotec for $425 million upfront, with an additional $575 million contingent on milestones.
– The deal enhances AstraZeneca’s cell therapy capabilities through EsoBiotec’s ENaBL platform, which enables in vivo immune cell engineering.
– The acquisition aligns with AstraZeneca’s broader strategy of leveraging cell therapies, gene editing, and radioconjugates for oncology and immune disorders.

AstraZeneca has announced a significant expansion of its cell therapy pipeline with the planned acquisition of EsoBiotec, a Belgium-based biotech firm specializing in immune cell engineering. The deal, valued at up to $1 billion, consists of a $425 million upfront payment with the potential for an additional $575 million based on development and regulatory milestones. The acquisition is expected to close in the second quarter of 2025.

EsoBiotec’s ENaBL platform represents a transformative approach to cell therapy. Unlike conventional ex vivo cell therapies that require the extraction, modification, and reinfusion of patient cells, ENaBL allows for direct genetic programming of immune cells within the body. This eliminates the need for invasive lymphodepletion procedures and could significantly lower costs while improving accessibility for patients.

AstraZeneca has not yet disclosed specific target indications for EsoBiotec’s platform but has emphasized its potential applications in oncology and immune-mediated diseases. The ENaBL technology could help develop novel cancer treatments or autoreactive cell therapies for conditions such as autoimmune disorders.

This acquisition marks another step in AstraZeneca’s aggressive expansion into the cell therapy space. The pharmaceutical giant has been actively pursuing high-value deals to strengthen its pipeline in this emerging field. In 2022, AstraZeneca acquired TeneoTwo for up to $1.27 billion, securing its T cell engager TNB-486, now renamed AZD0486, which is in Phase III trials for follicular lymphoma and Phase II trials for B cell non-Hodgkin lymphoma.

Further reinforcing its position, AstraZeneca made another major investment in December 2023 with the $1 billion purchase of Gracell Biotechnologies. This deal added GC012F, now known as AZD0120, an investigational CAR T therapy targeting CD19 and BCMA for multiple myeloma and systemic lupus erythematosus.

Beyond acquisitions, AstraZeneca has formed strategic collaborations in cell therapy, including a $245 million agreement with Cellectis in November 2023 and a potential $2 billion partnership with Quell Therapeutics in June 2023. These investments highlight the company’s commitment to leveraging cutting-edge biotechnologies to expand its capabilities in immune modulation and oncology.

As a relative latecomer to the cell therapy market, AstraZeneca is rapidly scaling its presence through acquisitions and partnerships. By integrating EsoBiotec’s ENaBL platform into its pipeline, AstraZeneca positions itself to compete with industry leaders in the race to develop next-generation cell therapies that offer improved efficacy, lower costs, and enhanced patient accessibility.

With this latest acquisition, AstraZeneca continues to build a robust portfolio of cell therapies that could redefine treatment approaches for cancer and immune-related diseases. Investors and industry analysts will be closely monitoring how effectively AstraZeneca integrates these new technologies and translates them into viable commercial therapies.

Release – Unicycive to Highlight Patient Reported Outcomes Data at Upcoming Medical Meetings Showing Oxylanthanum Carbonate Reduced Pill Burden and Improved Adherence in Treatment of Hyperphosphatemia

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

March 13, 2025 7:00am EDT Download as PDF

LOS ALTOS, Calif., March 13, 2025 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease, today announced that it will present patient reported outcomes data from its pivotal UNI-OLC-201 clinical study characterizing the potential impact of oxylanthanum carbonate (OLC) on the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis. These data will be presented at three medical meetings, including the 2025 Annual Dialysis Conference (ADC), March 13-16, 2025, National Kidney Foundation (NKF) Spring Clinical Meetings, April 10-13, 2025, and the 2025 American Nephrology Nurses Association (ANNA) National Symposium, being held on May 1-4, 2025.

Unicycive’s investigational drug OLC leverages proprietary nanoparticle technology to reduce the number and size of pills that patients must take. If approved, OLC may provide patients and their physicians with a welcome new option to control hyperphosphatemia. The New Drug Application (NDA) for OLC was accepted by the U.S. Food and Drug Administration (FDA) for the treatment of hyperphosphatemia in patients with chronic kidney disease on dialysis. The FDA set a Prescription Drug User Fee Act (PDUFA) Target Action Date of June 28, 2025.

“Despite the availability of several approved phosphate binders, hyperphosphatemia remains uncontrolled in 75% of people in the U.S. on dialysis due to challenges of insufficient potency, pill burden and unpalatable formulations. There is a critical need for more effective solutions,” said Shalabh Gupta, M.D., Chief Executive Officer of Unicycive. “Innovative solutions such as OLC that improve phosphate control and minimize pill size and count have the potential to significantly improve adherence, empowering those on dialysis to manage their treatment more effectively.”

Unicycive abstracts to be presented at the upcoming medical meetings include:

ADC

  • Title: Patient-Reported Outcomes in a Pivotal Clinical Study of Hyperphosphatemia: Oxylanthanum Carbonate Reduces Pill Burden by Half and Improves Adherence – Poster #: A-6870
  • Presentation Details: Friday, March 14, 5:30-7:30 p.m. PT
  • Presenting Author: Doug Jermasek

NKF Spring Clinical Meetings

  • Title: Patient-Reported Outcomes in a Pivotal Clinical Study of Hyperphosphatemia: Oxylanthanum Carbonate Reduces Pill Burden by Half and Improves Adherence – Poster #: G-018
  • Presentation Details: Thursday, April 10, from 5:15-7:30 p.m. ET
  • Presenting Author: Guru Reddy, PhD
  • Title: Pill Burden and Large Tablet Size Are Key Barriers to Phosphate Binder Adherence in Dialysis Patients – Poster #: G-297
  • Presentation Details: Thursday, April 10, from 5:15-7:30 p.m. ET
  • Presenting Author: Dr. Hill Gallant, PhD, RD, Associate Professor of Nutrition in the Department of Food Science and Nutrition at the University of Minnesota-Twin Cities

ANNA

  • Title: Pill Burden and Large Tablet Size Are Key Barriers to Phosphate Binder Adherence in Dialysis Patients
  • Presentation Details: Friday, May 2, starting at 8:45 a.m. PT

About Oxylanthanum Carbonate (OLC)

Oxylanthanum carbonate is a next-generation lanthanum-based phosphate binding agent utilizing proprietary nanoparticle technology being developed for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD). OLC has over forty issued and granted patents globally. Its potential best-in-class profile may have meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden for patients in terms of number and size of pills per dose that are swallowed instead of chewed. Based on a survey conducted in 2022, Nephrologists stated that the greatest unmet need in the treatment of hyperphosphatemia with phosphate binders is a lower pill burden and better patient compliance.1 The global market opportunity for treating hyperphosphatemia is projected to be in excess of $2.28 billion, with the North America accounting for more than $1 billion of that total.2 Despite the availability of several FDA-cleared medications, 75 percent of U.S. dialysis patients fail to achieve the target phosphorus levels recommended by published medical guidelines.3

Unicycive is seeking FDA approval of OLC via the 505(b)(2) regulatory pathway. The NDA submission package is based on data from three clinical studies (a Phase 1 study in healthy volunteers, a bioequivalence study in healthy volunteers, and a tolerability study of OLC in CKD patients on dialysis), multiple preclinical studies, and the chemistry, manufacturing and controls (CMC) data. OLC is protected by a strong global patent portfolio including issued patents on composition of matter with exclusivity until 2031, and with the potential for patent term extension until 2035.

About Hyperphosphatemia

Hyperphosphatemia is a serious medical condition that occurs in nearly all patients with End Stage Renal Disease (ESRD). If left untreated, hyperphosphatemia leads to secondary hyperparathyroidism (SHPT), which then results in renal osteodystrophy (a condition similar to osteoporosis and associated with significant bone disease, fractures and bone pain); cardiovascular disease with associated hardening of arteries and atherosclerosis (due to deposition of excess calcium-phosphorus complexes in soft tissue). Importantly, hyperphosphatemia is independently associated with increased mortality for patients with chronic kidney disease on dialysis. Based on available clinical data to date, over 80% of patients show signs of cardiovascular calcification by the time they become dependent on dialysis.4

Dialysis patients are already at an increased risk for cardiovascular disease (because of underlying diseases such as diabetes and hypertension), and hyperphosphatemia further exacerbates this. Treatment of hyperphosphatemia is aimed at lowering serum phosphate levels via two means: (1) restricting dietary phosphorus intake; and (2) using, on a daily basis, and with each meal, oral phosphate binding drugs that facilitate fecal elimination of dietary phosphate rather than its absorption from the gastrointestinal tract into the bloodstream.

About Unicycive Therapeutics

Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug candidate, oxylanthanum carbonate (OLC), is a novel investigational phosphate binding agent being developed for the treatment of hyperphosphatemia in chronic kidney disease patients on dialysis. Positive pivotal trial results were reported in June 2024 for OLC, and a New Drug Application (NDA) is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) Target Action Date of June 28, 2025. OLC is protected by a strong global patent portfolio including an issued patent on composition of matter with exclusivity until 2031, and with the potential patent term extension until 2035 after OLC approval. Unicycive’s second asset, UNI-494, is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. UNI-494 has successfully completed a Phase 1 trial. For more information, please visit Unicycive.com and follow us on LinkedIn, X, and YouTube.

Forward-looking statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified using words such as “anticipate,” “believe,” “forecast,” “estimated” and “intend” or other similar terms or expressions that concern Unicycive’s expectations, strategy, plans or intentions. These forward-looking statements are based on Unicycive’s current expectations and actual results could differ materially. There are several factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions, which could seriously harm our financial condition and increase our costs and expenses; dependence on key personnel; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Unicycive’s Annual Report on Form 10-K for the year ended December 31, 2023, and other periodic reports filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Unicycive specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

1Reason Research, LLC 2022 survey. Results here.
2 Fortune Business Insights™, Hyperphosphatemia Treatment Market, 2023-2030
3 US-DOPPS Practice Monitor, May 2021; http://www.dopps.org/DPM
4 Block GA, Klassen PS, Lazarus JM, Ofsthun N, Lowrie EG, Chertow GM. Mineral metabolism, mortality, and morbidity in maintenance hemodialysis. J Am Soc Nephrol. 2004 Aug;15(8):2208-18. doi: 10.1097/01.ASN.0000133041.27682.A2. PMID: 15284307.

Investor Contacts:

Kevin Gardner
LifeSci Advisors
kgardner@lifesciadvisors.com

Media Contact:

Rachel Visi
Real Chemistry
redery@realchemistry.com

Primary Logo

Source: Unicycive Therapeutics, Inc.

Released March 13, 2025

Eli Lilly Acquires Organovo’s FXR Program in Strategic Expansion

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– Eli Lilly (LLY) is acquiring Organovo’s (ONVO) FXR program, including lead drug candidate FXR314, for further development.
– Organovo will receive an upfront payment along with milestone-based regulatory and commercial payouts.
– ONVO stock surged over 200% following the announcement.

In a significant move for the biotechnology sector, Organovo Holdings, Inc. (Nasdaq: ONVO) announced the sale of its FXR program, including its lead candidate FXR314, to pharmaceutical giant Eli Lilly and Company (NYSE: LLY). The acquisition, disclosed on Tuesday, marks a pivotal moment for Organovo as it aligns its proprietary 3D human tissue technology with a global leader in drug development.

The FXR program, focused on inflammatory bowel disease (IBD), is a major step toward advancing novel treatment approaches. Organovo’s Executive Chairman, Keith Murphy, expressed confidence in Lilly’s ability to further develop FXR314, highlighting the company’s world-class expertise and commitment to patient care.

Under the agreement, Organovo will receive an upfront cash payment, with additional milestone payments contingent on regulatory approvals and commercial success. While the specific financial terms remain undisclosed, the market’s response has been overwhelmingly positive.

Following the announcement, ONVO shares skyrocketed by over 200%, reflecting investor optimism about the deal’s potential impact. Lilly’s stock also saw a modest gain of 2.32% as the acquisition strengthens its pipeline in the IBD treatment space.

For Organovo, this transaction reinforces its ability to leverage its cutting-edge 3D tissue technology for drug development partnerships. The company, known for pioneering bioprinting innovations, has been positioning itself as a key player in personalized medicine and regenerative therapies.

For Eli Lilly, the acquisition aligns with its broader strategy of expanding its immunology portfolio. FXR314’s development complements Lilly’s existing research efforts in inflammatory diseases, further cementing its position as a leader in next-generation therapeutics.

With FXR314 now under Lilly’s stewardship, the biotech industry will closely monitor its progression into Phase 2 trials. If successful, the drug could represent a breakthrough in IBD treatment, addressing a significant unmet medical need.

As Organovo pivots towards future innovation, and Lilly integrates this promising asset into its pipeline, investors and analysts alike will be watching closely to gauge the long-term benefits of this high-profile acquisition.

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

AbbVie and Xilio Therapeutics Collaborate to Develop Tumor-Activated Immunotherapies

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– AbbVie and Xilio Therapeutics announce a partnership to develop innovative tumor-activated immunotherapies, including masked T-cell engagers.
– Xilio will receive $52 million upfront and is eligible for up to $2.1 billion in milestone payments and royalties.
– The collaboration aims to enhance the effectiveness of immunotherapy while minimizing systemic side effects.

AbbVie and Xilio Therapeutics have entered a strategic collaboration to advance next-generation tumor-activated immunotherapies, a move that could significantly impact the oncology space. The partnership will focus on developing masked T-cell engagers (TCEs), a cutting-edge approach designed to precisely target tumors while reducing the systemic toxicity often associated with immunotherapies.

Under the terms of the agreement, Xilio will receive an upfront payment of $52 million, with the potential to earn up to $2.1 billion in milestone payments and royalties if the collaboration yields successful drug candidates. This deal highlights the growing interest in tumor-selective therapies as biopharmaceutical companies seek to refine cancer treatments for better efficacy and safety.

Immunotherapy has revolutionized cancer treatment over the past decade, with checkpoint inhibitors and CAR-T therapies offering promising results. However, many of these treatments come with serious side effects, such as cytokine release syndrome and immune-related toxicities, which can limit their widespread use. Tumor-activated therapies, like those being developed through the AbbVie-Xilio collaboration, aim to overcome these challenges by ensuring immune system activation occurs predominantly at the tumor site rather than throughout the body.

This strategy aligns with a broader industry trend where major pharmaceutical companies are investing heavily in precision oncology. Companies such as Bristol Myers Squibb, Merck, and Roche are also exploring targeted immune therapies, with some already advancing their own masked TCE platforms.

AbbVie’s decision to partner with Xilio follows similar collaborations between biotech startups and large pharmaceutical firms. Smaller biotech companies often bring innovative drug discovery capabilities, while established players like AbbVie provide the resources and expertise needed to navigate clinical development and regulatory approval.

The move also positions AbbVie competitively in the immuno-oncology space, where it faces increasing competition from global drugmakers. The company has been expanding its oncology pipeline following the success of Imbruvica and Venclexta, and this partnership could strengthen its position in the next generation of cancer therapeutics.

Meanwhile, Xilio Therapeutics, a biotech firm specializing in tumor-selective treatments, stands to gain significant financial backing and research support through this agreement. Its proprietary technology platform, which develops highly potent, tumor-activated biologics, has the potential to redefine immunotherapy approaches for solid tumors.

With oncology continuing to be one of the most lucrative and rapidly evolving fields in biotech, tumor-activated immunotherapies are poised to become a major focus of drug development. The potential to minimize toxicity while enhancing efficacy makes these therapies particularly appealing for both patients and healthcare providers.

If successful, the AbbVie-Xilio collaboration could lead to groundbreaking advancements in cancer treatment, opening doors for future partnerships and expanding the role of tumor-targeted biologics in oncology.

Take a moment to take a look at Noble Capital Markets Senior Research Analyst Robert LeBoyer’s life sciences and biotechnology coverage list.

Novo Nordisk Stock Soars After Groundbreaking Results for New Obesity Drug

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– Novo Nordisk’s amycretin led to 22% average weight loss in a 36-week trial.
– Shares rose 7.13%, marking the best single-day gain since March 2024.
– Amycretin targets dual hormones to tackle hunger and appetite, showcasing groundbreaking innovation.

Novo Nordisk shares surged Friday after the pharmaceutical giant announced promising early-stage trial results for amycretin, a groundbreaking weight-loss drug administered through a once-weekly injection. The Danish company revealed that the treatment led to an average weight reduction of 22% in overweight and obese patients over a 36-week trial, marking a significant advancement in the fight against obesity. This compares to a 2% weight gain observed in patients receiving a placebo, showcasing the drug’s potential to reshape the treatment landscape for weight management.

The trial involved 125 participants and highlighted amycretin’s innovative mechanism of action. The drug targets GLP-1, a gut hormone that regulates appetite, and amylin, a hormone produced by the pancreas that influences hunger. This dual-action approach is a step forward from Novo Nordisk’s flagship products, Wegovy, which mimics GLP-1, and Ozempic, its well-known diabetes treatment. Amycretin’s ability to address weight loss through multiple pathways underscores its potential to provide life-changing results for patients struggling with obesity.

Novo Nordisk’s stock rose by 7.13% on Friday, reaching its best single-day performance since March 2024. The initial gains peaked at nearly 14% before settling, reflecting strong investor confidence in the company’s ability to expand its market dominance in obesity therapeutics. Fellow Danish drugmaker Zealand Pharma also benefited from the announcement, with shares climbing 4.7%, while rival Eli Lilly, the maker of obesity drug Zepbound, saw a slight dip in premarket trading.

The pharmaceutical industry has been increasingly focused on developing more effective weight-loss solutions, with obesity affecting millions worldwide and posing significant health risks. Novo Nordisk’s continued innovation in this space has made it a frontrunner, and the results from the amycretin trial further solidify its position. The company is already exploring oral formulations of the drug, which, in a separate early-stage trial announced last September, demonstrated a 13.1% weight reduction over 12 weeks.

Safety and tolerability are key considerations for any obesity treatment, and amycretin appears to meet these benchmarks. The most common side effects observed during the trial were gastrointestinal issues, but most were mild to moderate in severity. These findings align with patient tolerability seen in previous trials for similar drugs, making amycretin a promising addition to Novo Nordisk’s portfolio.

Martin Lange, executive vice president for development at Novo Nordisk, expressed optimism about the trial results. “We are very encouraged by the subcutaneous phase 1b/2a results for amycretin in people living with overweight or obesity,” Lange said in a statement. “The results seen in the trial support the weight-lowering potential of this novel unimolecular GLP-1 and amylin receptor agonist.”

As Novo Nordisk invests further in amycretin, the drug has the potential to transform the obesity treatment market, which is projected to grow substantially in the coming years. The company’s strategic focus on innovative, science-driven solutions positions it to maintain a competitive edge while addressing a critical global health challenge.

Biotech Merger: Salarius and Decoy Unite to Advance AI-Driven Peptide Therapeutics

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– Combined company to focus on ML/AI-powered drug development platform
– Decoy shareholders to own 86% of merged entity
– Pipeline includes treatments for respiratory viruses and GI cancers

In a strategic move to accelerate the development of next-generation therapeutics, Salarius Pharmaceuticals (NASDAQ: SLRX) announced today its merger with privately-held Decoy Therapeutics in an all-stock transaction. The combined company, which will operate under the Decoy Therapeutics name, aims to leverage artificial intelligence and machine learning to revolutionize peptide conjugate drug development.

The merger brings together Decoy’s proprietary IMP3ACT™ platform, which has already attracted approximately $7 million in non-dilutive funding from prestigious organizations including The Bill & Melinda Gates Foundation, with Salarius’ clinical-stage pipeline and public market presence. Under the terms of the agreement, Decoy shareholders will own approximately 86% of the combined company, with Salarius stockholders retaining the remaining 14%.

“Peptide conjugates have become one of the most important drug classes as measured by prescription rates and revenue growth,” said Rick Pierce, Decoy’s Co-founder and CEO, who will lead the combined company. “Our highly experienced team is excited to be able to unlock significant shareholder value from our IMP3ACT platform, which can rapidly design new peptide conjugate drugs by applying ML and AI tools.”

The merged entity’s immediate focus includes advancing a pan-coronavirus antiviral toward an FDA Investigational New Drug (IND) application within the next 12 months. Additionally, the company plans to develop a broad-acting antiviral targeting flu, COVID-19, and respiratory syncytial virus (RSV), as well as a peptide drug conjugate for gastrointestinal cancers.

David Arthur, Salarius’ CEO, emphasized the strategic rationale: “The compelling science supporting Decoy’s peptide conjugate technology and the company’s management team are truly impressive. Based on our diligence, we believe Decoy is poised to advance multiple drug candidates that address significant unmet needs in numerous therapeutic areas.”

The combined company will maintain Salarius’ ongoing Phase 1/2 clinical trial at MD Anderson Cancer Center while exploring strategic alternatives for its seclidemstat program. The merger has received unanimous approval from both companies’ boards of directors and is expected to close following customary closing conditions.

Quanterix’s Game-Changing $220M Merger with Akoya Sets New Path for Disease Detection

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– All-stock merger creates first integrated blood and tissue biomarker detection platform
– Combined company projects $40M in annual cost savings by 2026
– Post-merger entity to maintain $175M cash position with zero debt

In a groundbreaking move that promises to revolutionize disease detection and monitoring, Quanterix Corporation announced today its acquisition of Akoya Biosciences in an all-stock transaction. The merger unites Quanterix’s ultra-sensitive biomarker detection capabilities with Akoya’s spatial biology expertise, creating the first integrated platform for comprehensive blood- and tissue-based protein biomarker analysis.

The strategic combination positions the merged entity at the forefront of liquid biopsy innovation, a market that Quanterix CEO Masoud Toloue believes will eventually eclipse all other diagnostic testing segments combined. “This transaction accelerates our progress by creating the first platform that lets researchers and clinicians track disease progression from tissue to blood,” said Toloue, who will continue as CEO of the combined company.

The deal structure gives Akoya shareholders 0.318 shares of Quanterix common stock for each Akoya share, representing a 19% premium to Akoya’s unaffected stock price from November 14, 2024. Post-merger, current Quanterix shareholders will hold approximately 70% of the combined company, with Akoya shareholders owning the remaining 30%.

Looking ahead, the merged company projects annual cost synergies of $40 million by the end of 2026, with half that amount expected within the first year post-closing. These savings will come from streamlined operations, improved commercial infrastructure, and optimized facilities. The combined entity will maintain a strong financial position with approximately $175 million in cash and no debt at closing.

Akoya CEO Brian McKelligon emphasized the strategic importance of the merger: “We are thrilled to be part of an established leader in the life science tools and diagnostics market that not only strengthens our presence in critical markets but also accelerates our ability to scale, innovate and ultimately bring to market products that impact human health.”

The transaction, expected to close in the second quarter of 2025, will create a powerhouse in biomarker detection with a combined installed base of 2,300 instruments and trailing 12-month revenue of approximately $220 million. The merger has already secured support from shareholders owning more than 50% of Akoya’s common stock.

Release – Data and Safety Monitoring Board Reviews Interim Safety Data of Phase 2 Subjects of OCU410 ArMaDa Clinical Trial for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

December 19, 2024

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  • OCU410 has a very favorable safety and tolerability profile
  • No serious adverse events related to the study drug have been reported, such as exudation, infectious endophthalmitis, intraocular Inflammation, anterior ischemic optic neuropathy, or vasculitis

MALVERN, Pa., Dec. 19, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU410 ArMaDa clinical trial recently convened and approved continuation of the second phase of the Phase 1/2 study. OCU410 (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD).

“The DSMB assessed data on 15 subjects from Phase 2. Initial data indicates that OCU410 appears to be safe and well-tolerated,” said Peter Chang, MD, FACS, Co-President and Partner of the Massachusetts Eye Research and Surgery Institution (MERSI). “No serious adverse events (SAEs) related to OCU410 have been reported to date.”

The ArMaDa clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410 in subjects with GA. Phase 2 is an ongoing, randomized, outcome assessor-blinded, dose-expansion study in which 45 subjects are randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups (5×1010 vg/mL or 1.5 ×1011 vg/mL) or an untreated control group.

“Currently approved treatments for GA require 6-12 intravitreal injections annually and frequent injections are a burden on patients and caregivers,” said Huma Qamar, MD, MPH, CMI, Chief Medical Officer of Ocugen. “We are very enthusiastic about the potential of OCU410 to serve as a game-changing, one-time treatment for life for patients with GA.”

Positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the ArMaDa clinical trial demonstrated: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA).

dAMD is a multifactorial disease involving genetic and environmental factors that is one of the world’s leading causes of blindness in people aged 50 years and older. Four cellular pathways drive the pathology of dry AMD: lipid metabolism, inflammation, oxidative stress, and complement. Currently approved therapies target only the latter, while OCU410 addresses all four and thereby helps reestablish retinal homeostasis.

The ArMaDa clinical trial is currently being performed at 13 leading retinal surgery centers across the U.S. Dosing in the OCU410 ArMaDa clinical trial will be completed in early 2025 and the Company will continue to provide 9- and 12-month efficacy updates from Phase 1.  

About dAMD and GA
dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.

About OCU410
OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in vitro and in vivo (animal model) studies. These results demonstrate the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV5–hRORA as a one-time gene therapy for the treatment of GA.

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, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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 annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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