In its financial results for the third quarter of 2020 Ionis Pharmaceuticals, Inc. reports the advance in the IONIS-PKK-LRx program: Proof-of-concept data from the PKK development program in patients with HAE were reported in the New England Journal of Medicine Enrollment completed in the IONIS-PKK-LRx Phase 2 study in patients with HAE IONIS-PKK-LRx advanced into [...]
The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted oral, once-daily berotralstat from BioCryst Pharmaceuticals, Inc. a positive scientific opinion through the Early Access to Medicines Scheme (EAMS). Under the EAMS, HAE patients in the UK aged 12 years and older can gain access to berotralstat for the routine prevention of recurrent [...]
At the presentation of the financial report for the nine months and third quarter ended 30 September 2020 Pharming Group N.V. CEO Sijmen de Vries says: “We are pleased to announce continued growth, demonstrating consistent progress, despite the impact of the COVID-19 pandemic. We remain focused on, and are delivering against, our strategy for long-term growth. [...]
In its annual R&D briefing to investors CSL Limited demonstrates how the company is advancing a novel research portfolio across four strategic scientific platforms (Plasma Fractionation, Recombinant Technology, Cell and Gene Therapy, Adjuvanted Cell and Egg-based Vaccines) across six therapeutic areas (Immunology, Hematology, Respiratory, Cardiovascular and Metabolic, Transplant, Influenza Vaccines) and two businesses (CSL Behring [...]
The Journal of Allergy and Clinical Immunology (JACI) publishes data from the first 24 weeks of the APeX-2 trial of oral, once-daily berotralstat in patients with HAE. APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group trial that evaluated the efficacy and safety of oral, once-daily berotralstat versus placebo over 24-weeks in 121 HAE patients ages 12 [...]
IONIS-PKK-LRx from Ionis Pharmaceuticals, Inc. is being evaluated in an investigator-initiated Phase 2 clinical study to determine its effectiveness in reducing the severity of respiratory complications in patients with COVID-19. The trial coordinators are Fernando G. Zampieri, M.D., Ph.D., and Alexandre Biasi Cavalcanti, M.D., Hospital do Coracao (HCor Research Institute), Sao Paulo, Brazil. The study [...]
KalVista Pharmaceuticals, Inc. provides an update on its franchise of oral therapies for treatment of HAE. “We have completed enrollment of our Phase 2 trial for our oral on-demand HAE treatment, KVD900, and remain on track to deliver data before the end of this year. We are also pleased to announce data from KVD824, our oral [...]
The U.S. Food and Drug Administration (FDA) has approved an expanded indication for HAEGARDA for routine prophylaxis to prevent HAE attacks in patients 6 years of age and older. HAEGARDA, the HAE therapy that reduces attacks by a median of 95%, is now the first and only subcutaneous treatment option for prevention of HAE attacks [...]
Bridge Medicines and The Rockefeller University has signed an exclusive license agreement to further develop a series of small molecule inhibitors of activated Factor XII (FXIIa) discovered in the laboratory of Jan L. Breslow, M.D. Breslow and his colleague Manish Ponda, M.D., illuminated a novel role for FXIIa as a key target for HAE and [...]
At the presentation of the operational update and released financial results for the first fiscal quarter ended 31 July 2020 KalVista Pharmaceuticals, Inc. CEO Andrew Crockett says: “We are pleased that our KVD900 Phase 2 trial has met its enrollment target and data is expected before the end of this year. We believe KVD900 can bring the [...]
