BIOCRYST PHARMACEUTICALS, INC.

Shionogi to Acquire Japan Tobacco's Pharmaceutical Subsidiaries for $1.1 Billion

13 days ago

• Shionogi & Co. plans to acquire Japan Tobacco's pharmaceutical subsidiaries Torii Pharmaceutical and Akros Pharma for ¥160 billion ($1.1 billion) to strengthen its global R&D capabilities. • The acquisition, negotiated since early 2024, will proceed through a tender offer beginning June 18 with a share price of ¥6,350 to acquire at least 11.89% of the minority stake. • This strategic move represents significant consolidation in the Japanese pharmaceutical sector and expands Shionogi's market presence amid increasing global competition.

Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments

3 months ago

• Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027. • KalVista Pharmaceuticals' sebetralstat receives Orphan Drug Designation in Japan, marking progress toward the first oral on-demand treatment for HAE attacks. • The global angioedema pipeline includes 20+ companies developing innovative therapies, with several promising candidates in late-stage development from companies like ADARx, BioMarin, and Astria Therapeutics.

BioCryst's Orladeyo Demonstrates Safety and Efficacy in Pediatric Hereditary Angioedema Trial

3 months ago

• Interim analysis of APeX-P trial shows Orladeyo (berotralstat) oral granule formulation is safe and well-tolerated in children aged 2-12 years with hereditary angioedema. • Real-world evidence demonstrates significant reduction in HAE attack rates across patients with both C1-INH deficiency and normal C1-INH levels after Orladeyo initiation. • Patient-reported outcomes indicate high treatment satisfaction and reduced attack frequency and severity, with 54% of U.S. participants maintaining Orladeyo treatment for over one year.

Clearside's CLS-AX Phase 2b Trial Data Shows Promise in Treatment-Naïve AMD Patients

3 months ago

• New subgroup analyses from Clearside Biomedical's ODYSSEY Phase 2b trial provide strategic insights for upcoming Phase 3 program in neovascular age-related macular degeneration. • Data presented at the Angiogenesis 2025 meeting supports focusing on treatment-naïve patients and implementing specific visual acuity criteria for future trials. • The 36-week ODYSSEY trial employed a robust design, being randomized, double-masked, and active-controlled across multiple centers.

Krystal Biotech and CRISPR Therapeutics Announce Key Updates in Gene Therapy Programs

4 months ago

• Krystal Biotech reported a 473% increase in full-year revenue, driven by VYJUVEK sales, and anticipates European and Japanese regulatory decisions in 2025. • Krystal Biotech's KB407 for cystic fibrosis received full sanctioning for its Phase 1 CORAL-1 study, with interim molecular data expected mid-2025. • CRISPR Therapeutics reported positive momentum in the CASGEVY launch, with over 50 activated treatment centers and patients initiating cell collection globally. • CRISPR Therapeutics expects key clinical data updates in 2025 for CTX112 in oncology and autoimmune diseases, and for CTX310 and CTX320 in cardiovascular programs.

Major Breakthroughs in HAE Treatment: 2024 Highlights Gene Editing and Novel Therapeutics

5 months ago

2024 marked significant advances in hereditary angioedema (HAE) treatment, with Intellia's gene-editing therapy NTLA-2002 showing a remarkable 95% reduction in monthly attacks. The year also saw important developments in multiple therapeutic candidates, including donidalorsen and garadacimab, while established treatments like Takhzyro demonstrated continued efficacy in adolescent populations.

Merus' Petosemtamab and Mersana's Emi-Le Show Promise in Cancer Treatment

6 months ago

• Merus' petosemtamab demonstrates a 36% overall response rate in recurrent/metastatic head and neck squamous cell carcinoma, with median overall survival of 11.4 months. • Mersana Therapeutics' Emi-Le receives Fast Track designation for HER2-negative breast cancer, showing promising monotherapy activity in multiple tumors. • Petosemtamab's clinical trials are ongoing, including phase 3 studies in HNSCC and expanded evaluation in metastatic colorectal cancer, with data updates planned for 2025.

BioCryst's Orladeyo® (berotralstat) Receives Recommendation in Ireland for HAE Prophylaxis

6 months ago

• The Health Services Executive (HSE) in Ireland has recommended Orladeyo® (berotralstat) for routine prevention of recurrent hereditary angioedema (HAE) attacks in eligible patients 12 years and older. • This recommendation grants HAE patients in Ireland access to the first oral, once-daily therapy aimed at reducing the frequency of HAE attacks. • Orladeyo® functions by decreasing the activity of plasma kallikrein, and it has already been licensed in 44 countries following its European Commission marketing authorization in April 2021.

Iptacopan Shows Promise in Reducing Proteinuria in IgA Nephropathy

7 months ago

• Phase III APPLAUSE-IgAN study demonstrates iptacopan significantly reduces proteinuria in patients with IgA nephropathy compared to placebo. • Iptacopan, an alternative pathway inhibitor, led to a 38.3% reduction in 24-hour urinary protein-to-creatinine ratio (UPCR) at 9 months. • The study also revealed significant changes in complement pathway biomarkers, indicating selective alternative pathway inhibition. • While showing promise, further data on eGFR is needed to confirm iptacopan's impact on slowing kidney function decline.

Clearside Biomedical's CLS-AX Phase IIb Trial Readout Anticipated in Q3 2024

a year ago

• Clearside Biomedical anticipates primary data readout from its Phase IIb ODYSSEY trial of CLS-AX for wet AMD in Q3 2024, assessing its potential as a maintenance therapy. • The company's SCS Microinjector continues to demonstrate benefits in suprachoroidal drug delivery, with multiple partnerships and clinical trials underway for various indications. • A new collaboration with BioCryst Pharmaceuticals will utilize Clearside's SCS Microinjector to deliver their compound aboard Elestat for treating diabetic macular edema. • Clearside Biomedical's cash balance, including proceeds from a recent offering, is expected to fund operations into Q3 2025, supporting the CLS-AX Phase III program.