Avenue Therapeutics

AnnJi Pharmaceutical Reports Promising Phase 1/2a Results for AJ201 in SBMA, a Rare Neuromuscular Disease

13 hours ago

• AnnJi Pharmaceutical's AJ201 demonstrated positive results in a Phase 1/2a trial for Spinal and Bulbar Muscular Atrophy (SBMA), showing improvements in physical function and muscle biomarkers after 12 weeks of treatment. • The investigational drug reduced mutant androgen receptor levels by more than 50% in over half of treated patients and activated the Nrf2 pathway, supporting its proposed mechanism of action. • With no FDA-approved treatments currently available for SBMA, AnnJi plans to advance AJ201 to Phase 3 trials, potentially offering hope for approximately 1 in 40,000 males affected by this rare genetic disorder.

Fortress Biotech Reports Strong 2024 Results with Two FDA Approvals and Strategic Acquisition Deal

2 months ago

• Fortress Biotech secured FDA approvals for two key products in Q4 2024: Emrosi for inflammatory lesions of rosacea and UNLOXCYT for advanced cutaneous squamous cell carcinoma, with commercial launch of Emrosi already underway. • Subsidiary Checkpoint Therapeutics is being acquired by Sun Pharma in a deal valued at $4.10 per share plus potential CVR of $0.70, expected to generate approximately $28 million for Fortress plus a 2.5% royalty on UNLOXCYT sales. • FDA accepted New Drug Application for CUTX-101 for priority review with PDUFA date of September 30, 2025, potentially qualifying for a valuable Priority Review Voucher upon approval.

FDA Grants Priority Review to CUTX-101 for Menkes Disease, Offering Hope for Rare Pediatric Condition

5 months ago

• The FDA has accepted Sentynl Therapeutics' NDA for CUTX-101, granting priority review for the treatment of Menkes disease, a rare genetic disorder. • Clinical trials of CUTX-101 demonstrated an almost 80% reduction in mortality risk compared to untreated patients, significantly improving overall survival. • CUTX-101 has been granted multiple designations, including Breakthrough Therapy and Orphan Drug, highlighting its potential to address a critical unmet need. • Cyprium Therapeutics is eligible to receive up to $129 million in milestone payments and royalties, retaining ownership of a potential Priority Review Voucher.