steenbjere

Astria Therapeutics, Inc. initiates the Phase 1a clinical trial of STAR-0215 in healthy subjects. STAR-0215 is a monoclonal antibody inhibitor of plasma kallikrein designed to provide long-acting, effective attack prevention for HAE, with dosing once every three months or longer. Astria anticipates preliminary results from the Phase 1a trial by year-end. The Phase 1a trial [...]

Intellia Therapeutics, Inc. reports operational highlights and financial results for the second quarter ended 30 June 2022. In vivo program update regarding HAE: NTLA-2002 leverages Intellia’s proprietary in vivo LNP delivery technology to knock out the KLKB1gene in the liver with the potential to permanently reduce total plasma kallikrein protein and activity, a key mediator [...]

At the presentation of the BioCryst Pharmaceuticals, Inc. financial results for the second quarter ended 30 June 2022, Jon Stonehouse, President and CEO of BioCryst, says: “With ORLADEYO firmly established in the marketplace as it steadily grows each quarter and our pipeline of other oral drugs for rare diseases, BioCryst is uniquely positioned to bring multiple [...]

Strategic highlights from the presentation of Pharming Group N.V.'s preliminary (unaudited) financial report for the first six months of 2022 ended 30 June 2022: During the first half of 2022, we continued to execute on our strategic objectives of building a sustainable business by focusing on RUCONEST® sales, the approval, launch and commercialization of leniolisib, [...]

The U.S. Food and Drug Administration (FDA) gives clearance of Astria Therapeutics, Inc.'s Investigational New Drug (IND) application for STAR-0215, which the company is developing for the treatment of HAE. A Phase 1a trial of STAR-0215 in healthy volunteers is expected to initiate in the coming weeks, with preliminary results anticipated by year-end. “The acceptance of [...]

CSL Behring has received the first Patient Access Award for HAE, which recognizes companies that go above and beyond to ensure that much-needed therapies get into the hands of the patients who need them. Awarded by Managed Markets Insight & Technology (MMIT), the honor is determined by a survey of payer and physician stakeholders who participate in MMIT’s quarterly [...]

KalVista Pharmaceuticals, Inc. provides an operational update and released financial results for the fiscal year ended April 30, 2022. “We are pleased with the progress we have made over the last fiscal year in the development of the candidates in our oral HAE franchise,” says Andrew Crockett, CEO of KalVista. “We are meeting our enrollment [...]

KalVista Pharmaceuticals, Inc. presents data for its oral on-demand plasma kallikrein (PKa) inhibitor and Factor XIIa (FXIIa) programs at the EAACI2022 conference in Prague, Czech Republic. Data shows that sebetralstat was an effective treatment for both peripheral and abdominal attacks with 80% of attacks achieving symptom relief within 12 hours, regardless of attack location. Population pharmacokinetic [...]

Takeda announces late-breaking data from the Phase 3 SPRING study (NCT04070326) at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2022, demonstrating positive results of TAKHZYRO®(lanadelumab) for preventing HAE attacks in patients 2 to <12 years of age, which were consistent with earlier studies in adult and adolescent patients. There are currently [...]

BioCryst Pharmaceuticals, Inc. announces new long-term efficacy and safety data from the APeX-2 and APeX-S clinical trials evaluating oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of HAE showing sustained reductions in attack rates and improvement in quality of life (QoL) among patients living with HAE, and improved management of symptoms after switching to ORLADEYO [...]