Abeona Therapeutics
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Employees
- 84
- Market Cap
- $242.5M
- Introduction
Abeona Therapeutics, Inc. is a clinical stage biopharmaceutical company, which engages in the development of gene therapy for severe and life-threatening rare diseases. It programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA), and ABO-101 (AAV NAGLU), an AAV based gene therapy for Sanfilippo syndrome type B (MPS IIIB). The company was founded in 1974 and is headquartered in Cleveland, OH.
US Orphan Drug Market Set to Exceed $190 Billion by 2030 as FDA Designations Accelerate
• The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval.
• Since 2020, more than half of all new drug approvals by the FDA's Center for Drug Evaluation and Research have been granted orphan status, highlighting the growing importance of rare disease treatments in pharmaceutical development.
• Despite criticism over high pricing, exemplified by Abeona Therapeutics' Zevaskyn at $3.1 million per treatment, the orphan drug model has evolved into a sound business strategy offering fewer competitors, faster approvals, and seven-year market exclusivity.
iTeos and GSK Halt Development of TIGIT Antibody Belrestotug After Phase II Trial Failures in Lung and Head & Neck Cancers
• iTeos Therapeutics and GlaxoSmithKline have discontinued development of their TIGIT inhibitor belrestotug after Phase II clinical trials failed to meet efficacy endpoints in non-small cell lung cancer and head and neck squamous cell carcinoma.
• The setback represents another blow to the TIGIT inhibitor class, which has faced multiple clinical disappointments despite initial promise as a potential breakthrough in immuno-oncology combination therapies.
• The companies will now redirect resources toward other pipeline assets, while researchers continue to investigate whether specific biomarkers might identify patient subgroups who could benefit from TIGIT-targeted therapies.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
Continuity Biosciences Expands into Aesthetics with Strategic Investment in 3D-Printed Microneedle Technology
• Continuity Biosciences has made a strategic investment in PinPrint, Inc., expanding its drug delivery portfolio from implanted nanofluidic systems to 3D-printed microneedle technology for aesthetic and cosmetic applications.
• PinPrint's breakthrough platform, developed by Stanford's Dr. Joseph DeSimone, enables ultra-precise microchannels in microneedle patches that can deliver vaccines and active agents intradermally without traditional injections.
• The partnership aims to revolutionize dermatologic and cosmetic agent delivery directly into the dermis, offering improved precision, personalization, and patient comfort across therapeutic categories.
FDA Misses Deadline for Stealth Biotherapeutics' Rare Barth Syndrome Drug Elamipretide
• The FDA has missed its April 29 deadline to decide on Stealth Biotherapeutics' elamipretide for Barth syndrome, with no new decision date announced yet.
• Elamipretide aims to become the first approved treatment for Barth syndrome, an ultra-rare condition affecting approximately 150 people in the U.S. that causes muscle weakness, heart failure, and often early death.
• The delay comes amid thousands of FDA layoffs, raising concerns about the agency's capacity to meet drug review timelines despite assurances that drug reviewers would not be affected.
Trump's Executive Order Delays Medicare Drug Price Negotiations, Sparking Industry and Policy Debate
• President Trump signed an executive order extending the exemption period for small-molecule drugs from Medicare price negotiations by four years, a move criticized by advocacy groups as favoring pharmaceutical industry interests.
• The order aims to address what the administration calls the "pill penalty" - the current policy where small-molecule drugs (90% of medications) face negotiations after 9 years while biologics have longer exemption periods.
• The Medicare Drug Price Negotiation Program, established under the Biden administration, had already achieved price reductions of 38-79% on 10 high-cost drugs with projected savings of $6 billion if applied in 2023.
FDA Approves Abeona's Novel Cell Therapy for Rare Skin Disease
• The FDA has granted approval for Abeona Therapeutics' cell therapy, marking a significant advancement in treatment options for patients with a rare genetic skin condition.
• The therapy represents the first FDA-approved treatment specifically targeting the underlying genetic cause of the disease, potentially offering substantial quality of life improvements for affected patients.
• Clinical trials demonstrated meaningful efficacy with a favorable safety profile, addressing a critical unmet need in this patient population with limited therapeutic alternatives.
FDA Accepts Teva's Application for AJOVY in Pediatric Migraine Prevention, Potentially First CGRP Antagonist for Children
• The FDA has accepted Teva Pharmaceuticals' supplemental Biologics License Application for AJOVY (fremanezumab) to expand its indication to include prevention of episodic migraine in children and adolescents aged 6-17 years weighing at least 45 kg.
• The application is supported by positive Phase 3 SPACE trial results, which demonstrated statistically significant reductions in monthly migraine days and monthly headache days compared to placebo, with a safety profile consistent with adult populations.
• If approved, AJOVY would become the first calcitonin gene-related peptide (CGRP) antagonist indicated for migraine prevention in both adults and pediatric patients, addressing a significant unmet need in pediatric migraine care.
Next-Generation Gene Therapies: Evolving Beyond Viral Vectors Towards More Affordable, Sustainable Solutions
• Despite 32 approved gene therapies globally, the industry faces significant challenges in safety, efficacy, and affordability, prompting development of novel delivery systems beyond traditional viral vectors.
• Companies are advancing non-viral delivery platforms including exosomes, lipid nanoparticles, and hydrophilic nanoparticles that offer cost-effective alternatives with reduced immunogenicity and potential for repeat dosing.
• Next-generation gene editing technologies like Prime Editing and CRISPR variants are emerging as more precise alternatives to traditional CRISPR-Cas9, with Prime Medicine's PM359 for chronic granulomatous disease advancing to clinical trials.
Newron Pharmaceuticals Advances Pivotal Phase III Program for Treatment-Resistant Schizophrenia
• Newron Pharmaceuticals has received regulatory approval for its ENIGMA-TRS Phase III program evaluating Evenamide as an add-on therapy for treatment-resistant schizophrenia, with patient recruitment beginning immediately.
• The pivotal program consists of two studies designed to meet requirements for marketing authorization in major markets including the USA and Europe, with results expected by late 2026.
• Evenamide, a first-in-class glutamate modulator, has shown promising results in earlier trials with 70% of TRS patients experiencing clinically significant benefits and 25% achieving remission during one-year treatment.
FDA Grants Breakthrough Therapy Status to Radiprodil for GRIN-Related Seizure Treatment
• GRIN Therapeutics' radiprodil receives FDA Breakthrough Therapy designation for treating seizures in patients with GRIN-related neurodevelopmental disorder, showing 86% median reduction in seizure frequency during Phase 1b trials.
• The investigational drug demonstrated significant efficacy in Phase 1b Honeycomb study, with 71% of patients achieving over 50% reduction in countable motor seizures and most patients showing behavioral improvements.
• The company plans to initiate Phase 3 pivotal trials in mid-2025, backed by a $200M capital commitment from Blackstone Life Sciences, to evaluate radiprodil's impact on seizures and behavioral outcomes.
Abeona Therapeutics' Pz-cel BLA Resubmission Accepted by FDA for RDEB Treatment
• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to treat recessive dystrophic epidermolysis bullosa (RDEB).
• The FDA has set a PDUFA target action date of April 29, 2025, for its decision on pz-cel, an autologous cell-based gene therapy.
• Abeona Therapeutics has made significant strides toward the potential commercialization of pz-cel in 2025, including discussions with payers and target treatment centers.
FDA Accepts Aldeyra's Reproxalap NDA for Dry Eye Disease with AbbVie Collaboration
• The FDA has accepted Aldeyra Therapeutics' New Drug Application (NDA) for reproxalap, a topical ocular therapy for dry eye disease, with a PDUFA date of April 2, 2025.
• Reproxalap, a first-in-class RASP modulator, has demonstrated significant clinical efficacy and safety in reducing the signs and symptoms of dry eye disease in multiple late-stage trials.
• Aldeyra has expanded its collaboration with AbbVie to accelerate pre-commercial activities, with AbbVie covering 60% of the costs and a potential $100 million upfront payment upon exercising its option.
• The partnership includes up to $300 million in milestone payments and a profit-sharing model for U.S. commercialization, positioning reproxalap as a potential game-changer in dry eye disease treatment.
FDA Accepts Abeona Therapeutics' BLA Resubmission for Prademagene Zamikeracel in RDEB Treatment
• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB).
• Pz-cel, an autologous cell-based gene therapy, aims to address the unmet needs of RDEB patients by providing collagen VII expression at wound sites.
• The BLA is supported by data from the Phase 3 VIITAL study and a Phase 1/2a study with up to 8 years of follow-up, showcasing clinical efficacy and safety.
• The FDA has set a PDUFA target action date of April 29, 2025, with potential for Abeona to receive a Priority Review Voucher upon approval.
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