RDEB

Abeona Therapeutics Reports Full Year 2023 Financial Results and Announces Completion of FDA Inspections

Retrieved on: 
Monday, March 18, 2024
Research, AAV, Advisory Committee, Form FDA 483, PLI, Sale, EIR, University, Trial of the century, Rett syndrome, Concha bullosa, Priority review, Investment, IP, Therapy, Safety, REMS, EB, Food, FDA, BLA, RDEB, PDUFA, Pharmaceutical industry

On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.

Key Points: 
  • On March 15, 2024, the Company submitted a response to the FDA, outlining already implemented and ongoing steps toward resolution that follow FDA guidance provided during the audit.
  • In January 2024, the FDA completed both a Bioresearch Monitoring (BIMO) inspection of Abeona and the Mid-Cycle Meeting regarding the pz-cel BLA.
  • Following completion of the BIMO inspection, the FDA inspector did not issue any observations or FDA Form 483s.
  • In March 2024, Abeona received a written Establishment Inspection Report (EIR), the formal report from the FDA regarding the BIMO inspection.

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Therapy, Regenerative Medicine Advanced Therapy, FAP, Concha bullosa, Sulindac, Chemistry, Fast track (FDA), Muscular dystrophy, FDA, Sensation, News, Eflornithine, DMD, Prader–Willi syndrome, BLA, Chromosome, RDEB, PDUFA, Skin, Voyager, PRISM, Duchenne muscular dystrophy, Safety, Neurocrine Biosciences, Rare Disease Day, Genetics, Fatigue, Walking, PWS, Patient, DSM-IV codes, Medicine, Disease, Exosome, FA, CMC, Priority review, Pharmaceutical industry

All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.

Key Points: 
  • All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.
  • Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
  • RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds.
  • Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases.

Abeona Therapeutics Announces Progress Update on Pz-cel Biologics License Application (BLA)

Retrieved on: 
Thursday, February 1, 2024
REMS, Research, Patient, RDEB, FDA, Marketing, Advisory Committee, Biologics license application, Food, PDUFA, Concha bullosa, BLA, Medical device

CLEVELAND, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced, as part of the review process by the U.S. Food and Drug Administration (FDA) for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB), completion by the FDA of both a Bioresearch Monitoring (BIMO) inspection of Abeona and the BLA mid-cycle review meeting.

Key Points: 
  • The FDA inspector did not issue any observations or FDA Form 483s during the inspection.
  • The formal report from the FDA regarding the BIMO inspection will be received at a later date.
  • Following the BIMO inspection, the BLA mid-cycle review meeting took place on January 25, 2024.
  • The FDA reaffirmed its earlier indication that it does not currently plan to convene an Advisory Committee for pz-cel.

Abeona Therapeutics Announces $50 Million Credit Facility

Retrieved on: 
Monday, January 8, 2024
Marketing, Food, Concha bullosa, Priority review, FDA, PDUFA, PZ, RDEB, Biologics license application, Patient, COL7A1

CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.

Key Points: 
  • CLEVELAND, Jan. 08, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that it has entered into a $50 million credit facility with the Avenue Venture Opportunities Fund, L.P.
  • The credit agreement, which has a term of three and a half years, includes a first tranche of $20 million at closing, a second tranche of $10 million of committed capital, and an additional accordion option to upsize the credit facility by an additional $20 million upon satisfaction of certain terms and conditions.
  • The FDA has accepted and granted Priority Review with a PDUFA target action date of May 25, 2024 for the Biologics License Application for pz-cel.
  • “We are excited to enter into this relationship with Avenue Venture Fund and secure additional financial resources to further support our launch and commercialization efforts for pz-cel,” said Joe Vazzano, Chief Financial Officer of Abeona.

Eloxx Pharmaceuticals Reports Third Quarter 2023 Financial and Operating Results and Provides Business Update

Retrieved on: 
Monday, November 13, 2023
Sale, FSD, RDEB, ASN, Kidney, GBM, American Society of Nephrology, Patient, CFF, FDA, Glomerular basement membrane, G&A, Health, R, JEB, ATM, Society, TEM, OTC, RNA, Proteinuria, Biopsy, Pharmaceutical industry, Phosphorus, Microscope, Cryptocurrency, Alport syndrome

WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.

Key Points: 
  • WATERTOWN, Mass., Nov. 13, 2023 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial results for the three months ended September 30, 2023, and provided a business update.
  • Data from the Phase 2 study of ELX-02 for Alport Syndrome was included in two presentations at the American Society of Nephrology (ASN) Kidney Week 2023.
  • R&D expenses were $1.3 million for the three months ended September 30, 2023, which included $0.1 million in stock-based compensation.
  • Additionally, we sold shares of common stock through our ATM program during the quarter for gross proceeds of $1.8 million.

Abeona Therapeutics Reports Third Quarter 2023 Financial Results and Corporate Developments

Retrieved on: 
Monday, November 13, 2023
World Health Organization, Priority review, Bristol Myers Squibb, Safety, Business development, Food, Therapy, BLA, International, Investment, COL7A1, Research, FDA, EB, BMS, Indiana University, Concha bullosa, PZ, Pharmaceutical industry, Cryptocurrency, RDEB

CLEVELAND, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the third quarter of 2023 and provided corporate updates.

Key Points: 
  • The FDA also agreed that retroviral vector manufactured at Abeona and Indiana University appear comparable based on the data that Abeona provided in its briefing book.
  • If accepted with Priority Review, Abeona expects potential BLA approval in the second quarter of 2024.
  • Abeona estimates that its cash and cash equivalents, restricted cash and short-term investments as of September 30, 2023 are sufficient resources to fund operations into the fourth quarter of 2024.
  • Abeona Therapeutics will host a conference call and webcast today, November 13, 2023, at 8:30 a.m.

Encouraging Rigosertib Data Presented at EADV as Late Breaker

Retrieved on: 
Thursday, October 12, 2023
Professor, Cancer, Journal of the European Academy of Dermatology and Venereology, Late, RDEB, PMU, Carcinoma, Therapy, SCC, Concha bullosa, IST, Patient, Pharmaceutical industry, Dermatology, Venereology

“RDEB-associated SCC is a devastating disease with few if any treatment options for advanced patients.

Key Points: 
  • “RDEB-associated SCC is a devastating disease with few if any treatment options for advanced patients.
  • We are very pleased for additional data on the potential use of rigosertib in patients with RDEB-associated SCC to be presented as a ‘late breaker’ at EADV 2023,” said Steven Fruchtman, M.D., President and CEO of Onconova.
  • “We believe that rigosertib’s profile and impact on key cell signaling pathway targets, including PLK-1 kinase, could make rigosertib a very attractive approach for this indication and other cancers.
  • I am pleased to share the initial encouraging patient experience with rigosertib at this conference.

Onconova Therapeutics to Present a Late-Breaking Abstract on Rigosertib at the 2023 EADV Congress

Retrieved on: 
Thursday, September 28, 2023
SCC, Late, Journal of the European Academy of Dermatology and Venereology, RDEB, Cancer, Concha bullosa, Congress, Patient, Therapy, Pharmaceutical industry, Dermatology, Venereology

NEWTOWN, Pa., Sept. 28, 2023 (GLOBE NEWSWIRE) -- Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”, the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that a late-breaking abstract reporting the use of rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB) associated with advanced/metastatic squamous cell carcinoma (SCC) will be presented in the Late Breaking News session at the European Academy of Dermatology and Venereology (EADV) 2023 Congress taking place October 11-14 in Berlin, Germany.

Key Points: 
  • NEWTOWN, Pa., Sept. 28, 2023 (GLOBE NEWSWIRE) -- Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”, the “Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that a late-breaking abstract reporting the use of rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB) associated with advanced/metastatic squamous cell carcinoma (SCC) will be presented in the Late Breaking News session at the European Academy of Dermatology and Venereology (EADV) 2023 Congress taking place October 11-14 in Berlin, Germany.

Abeona Therapeutics Submits Biologics License Application to U.S. FDA Seeking Priority Review and Approval of EB-101 for the Treatment of Patients with Recessive Dystrophic Epidermolysis Bullosa

Retrieved on: 
Tuesday, September 26, 2023
Biologics license application, Priority review, ISID, RDEB, Quality of life, Concha bullosa, Regenerative medicine, Rare, Orphanet Journal of Rare Diseases, Patient, Tissue Engineering and Regenerative Medicine International Society, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Safety, Wound healing, Pharmaceutical industry, New Drug Application

As part of the submission, Abeona requested a Priority Review, which, if granted, would shorten the FDA’s review period to six months from the filing acceptance of the BLA, instead of 10 months under standard review.

Key Points: 
  • As part of the submission, Abeona requested a Priority Review, which, if granted, would shorten the FDA’s review period to six months from the filing acceptance of the BLA, instead of 10 months under standard review.
  • “We appreciate the FDA’s level of engagement and constructive guidance in the months leading up to the pre-BLA meeting.
  • If accepted with Priority Review, Abeona expects potential BLA approval in the second quarter of 2024, at which time, Abeona believes that it would be eligible to receive a Priority Review Voucher.
  • EB-101 has been granted Rare Pediatric Disease, Regenerative Medicine Advanced Therapy, Breakthrough Therapy and Orphan Drug designations.

Abeona Therapeutics Appoints Madhav Vasanthavada, Ph.D., M.B.A. as Chief Commercial Officer

Retrieved on: 
Tuesday, September 12, 2023
Business development, University, Doctor of Philosophy, Pharmacy, Marketing, RDEB, Bristol Myers Squibb, Concha bullosa, CCO, Radium, Bayer, Publication, Triple accreditation, Lisocabtagene maraleucel, Harvard Business School, BMS, BD, Pharmaceutical industry

CLEVELAND, Sept. 12, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the appointment of Madhav Vasanthavada, Ph.D., M.B.A. to the role of Chief Commercial Officer (CCO) and Head of Business Development (BD), effective immediately.

Key Points: 
  • CLEVELAND, Sept. 12, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the appointment of Madhav Vasanthavada, Ph.D., M.B.A. to the role of Chief Commercial Officer (CCO) and Head of Business Development (BD), effective immediately.
  • Over the past year, as Abeona’s Head of BD, Dr. Vasanthavada’s efforts to assess EB-101’s commercial opportunity informed the Company’s strategy to prepare for a U.S. commercial launch without depending on a partner.
  • “Madhav’s appointment as Chief Commercial Officer is timely as Abeona prepares for the transition into a commercial-stage organization with the potential approval and launch of EB-101 in the U.S. next year,” said Vish Seshadri, Chief Executive Officer of Abeona.
  • He began his career as a scientist in Novartis R&D where his work led to multiple patents and publications.