Astria Therapeutics (ATXS) 2025 Conference September 04, 2025 03:20 PM ET Speaker1Great. Good afternoon, everyone, and welcome to the next session at the Cantor Global Health Care Conference. I'm Steve Seedhouse on the Cantor Biotech team, really glad to be joined by our next participating company, Astria Therapeutics, an exciting company that we launched coverage on when we came to Cantor. It's been a privilege to cover so far, and looking forward to all the upcoming progress. I'm joined on stage by CEO Ji ...

Core Viewpoint - Jetabelin's innovative drug FXIIa inhibitor monoclonal antibody (garadacimab) has received acceptance for market application in China for the prevention of hereditary angioedema (HAE) attacks [1] Group 1: Product Information - Garadacimab is the world's first and currently the only drug targeting factor XIIa (FXIIa) for preventive treatment of HAE [1] - HAE is a rare, potentially life-threatening genetic disease characterized by recurrent and unpredictable swelling of the skin and/or submucosa [1] Group 2: Regulatory Approval - The drug has already obtained marketing authorization in several countries and regions, including the United States, European Union, United Kingdom, Japan, Australia, Switzerland, and the United Arab Emirates [1]

Summary of CalVista Pharmaceuticals FDA Approval Call Company and Industry - **Company**: CalVista Pharmaceuticals (KALV) - **Industry**: Hereditary Angioedema (HAE) Treatment Core Points and Arguments 1. **FDA Approval Announcement**: CalVista announced the FDA approval of sebitralstat, now branded as ECTRLY, as the first and only oral on-demand treatment for hereditary angioedema (HAE) attacks in patients aged 12 and older [3][6][39] 2. **Significance of Approval**: This approval marks a major milestone for both CalVista and the HAE community, being the first new on-demand therapy in over a decade [6][39] 3. **Treatment Landscape**: HAE is a rare genetic condition affecting approximately 8,000 people in the U.S. The current treatment landscape has been dominated by injectable therapies, which present barriers to timely treatment [9][10][13][14] 4. **Efficacy and Safety Profile**: ECTRLY is designed to be taken at the earliest recognition of an HAE attack, with a recommended dose of 600 mg. It has shown a rapid response time and a favorable safety profile, with headaches being the only notable adverse reaction [17][22][23] 5. **Market Opportunity**: The on-demand segment of the HAE market is expected to grow by 70%, reaching $1.2 billion by 2030, largely driven by the introduction of ECTRLY [39][40] 6. **Commercial Strategy**: CalVista plans to drive demand through a well-prepared sales force, patient education programs, and partnerships with healthcare professionals. The company has already engaged with key prescribers and plans to leverage patient summits for awareness [30][31][37][88] 7. **Patient Access Programs**: The Quick Start program allows patients immediate access to ECTRLY at no charge while their claims are processed, ensuring that they can treat attacks promptly [34][36] 8. **Global Expansion Plans**: Following U.S. approval, CalVista is preparing for global expansion, with plans to launch in Germany and Japan in 2026 [40] Important but Possibly Overlooked Content 1. **Patient Compliance**: The approval of ECTRLY is expected to improve patient compliance with treatment guidelines, as it overcomes barriers associated with injectable therapies [15][16] 2. **Real-World Data**: In the ongoing CONFIDENCE trial, a median time to treatment of 10 minutes and a median time to end of attack progression of 19.8 minutes were reported, indicating a rapid response to treatment [25][26] 3. **Physician Engagement**: The company has been actively engaging with physicians since February, ensuring a high level of awareness and readiness for the launch [84][85] 4. **Patient Satisfaction**: Early feedback from patients in the CONFIDENCE trial indicates high satisfaction levels with ECTRLY, which will be further analyzed and reported in future updates [90][91] This summary encapsulates the key points discussed during the call, highlighting the significance of ECTRLY's approval and the strategic plans for its commercialization and market penetration.

–FDA grants Priority Review of application, with PDUFA target action date of September 12, 2025– –ORLADEYO would be the first targeted oral prophylactic therapy for patients with HAE under the age of 12, if approved– RESEARCH TRIANGLE PARK, N.C., May 14, 2025 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for the use of oral, once-daily ORLADEYO (berotralstat) in pediatric patients ...