NEWS
13th C1-inhibitor deficiency and angioedema workshop
Diagnostic and therapeutic applications of genetic research results The 13th C1-INH Deficiency and Angioedema Workshop took place in Budapest from 4-7 May. Nearly 400 participants arrived in the Hungarian capital this year from 42 [...]
ORLADEYO® granted marketing authorization in Chile
The Public Health Institute (ISP) of Chile has granted marketing authorization for oral, once-daily ORLADEYO® (berotralstat) for the prophylaxis of HAE attacks in patients 12 years of age or older. “The announcement marks the first [...]
Astria expects initial proof-of-concept results for STAR-0215 mid-2024
Astria Therapeutics, Inc. reports financial results for the first quarter ended 31 March 2023 and provides a corporate update. “We are excited to be administering STAR-0215 to patients in the Phase 1b/2 ALPHA-STAR clinical trial. [...]
KalVista delivers new data
KalVista Pharmaceuticals, Inc. delivers two oral presentations and one poster presentation at the 13th C1-inhibitor Deficiency & Angioedema Workshop (C1 Workshop) in Budapest, Hungary. Rationale for the Short-term Prophylaxis Regimen with Sebetralstat in KONFIDENT-S: Michael [...]
Milestone of 1,000 U.S. patients on ORLADEYO surpassed
“The strong new patient growth in the first quarter, building on our large patient base with ORLADEYO, positions us very well to achieve our expectations for 2023. This growing revenue stream, alongside our robust balance [...]
New data from APeX-S clinical trial
BioCryst Pharmaceuticals, Inc. announces new data from the APeX-S clinical trial, which evaluated oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of HAE, showing sustained reduction in disease burden for patients across multiple subgroups through [...]
Pharvaris reaches significant milestone
At the presentation of the Pharvaris financial results for the fourth quarter and year ended 31 December 2022, CEO Berndt Modig says: “Our first in-patient data readout of deucrictibant in people living with HAE was [...]
FDA grants RMAT designation to Intellia’s NTLA-2002
The U.S. Food and Drug Administration (FDA) grants Regenerative Medicine Advanced Therapy (RMAT) designation to Intellia Therapeutics, Inc.'s NTLA-2002 for the treatment of HAE. NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to inactivate [...]
Results support Astria’s vision for STAR-0215
At the presentation of the company's financial results for the fourth quarter and full year ended 31 December 2022, Astria Therapeutics, Inc.'s CEO Jill C. Milne, Ph.D., says: “We made excellent progress with our STAR-0215 program [...]
Pharvaris presents positive study data
At the 2023 HAEi Regional Conference APAC in Bangkok, Thailand, Pharvaris presents positive data from its Phase 2 RAPIDe-1 study of PHVS416 for the on-demand treatment of HAE attacks. Marc A. Riedl, M.D., M.S., Professor of [...]
Pharming’s stem cell gene therapy makes good progress
At the presentation of the Pharming Group N.V. preliminary (unaudited) financial report for the full year ended 31 December 2022, CEO Sijmen de Vries comments: "2022 was a pivotal year for Pharming. It was a year [...]
KalVista makes significant advances
At the operational update and release of the financial results for the third fiscal quarter ended 31 January 2023, the KalVista Pharmaceuticals, Inc. CEO Andrew Crockett says: “We have made significant advances at KalVista over this [...]
FDA clearance enables Intellia to include USA in Phase 2 study
The U.S. Food and Drug Administration (FDA) has cleared Intellia Therapeutics, Inc.’s Investigational New Drug (IND) application for NTLA-2002 for the treatment of HAE, enabling the company to include the United States in the global [...]
Real-world data highlight potential for sebetralstat to improve treatment experience
KalVista Pharmaceuticals, Inc. presents five posters at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting in San Antonio, Texas, United States. “The real-world data presented at AAAAI highlight the significant treatment burden and [...]
Once-monthly subcutaneous injections of garadacimab significantly reduces attack rate
CSL presents results from the pivotal placebo-controlled Phase 3 VANGUARD clinical trial of garadacimab (CSL312), CSL's investigational first-in-class monoclonal antibody being developed as a long-term prophylactic treatment for patients with HAE. Results from the trial, [...]
Astria demonstrate early proof-of-concept for STAR-0215
Astria Therapeutics, Inc. presents new STAR-0215 data in two presentations at the American Academy of Allergy, Asthma, and Immunology Annual Meeting 2023, that demonstrate early proof-of-concept for STAR-0215’s profile as a long-acting preventative therapy for HAE. [...]
New data from ORLADEYO® clinical trials
BioCryst Pharmaceuticals, Inc. announces new data from the APeX-S and APeX-2 clinical trials which evaluated oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of HAE demonstrating sustained reductions in attack rates and improvement in quality [...]
Additional positive interim data from Phase 2 study of donidalorsen
Ionis Pharmaceuticals, Inc. announces additional positive interim data from a Phase 2 open-label extension (OLE) study of donidalorsen, an investigational antisense medicine for the treatment of patients with HAE. Positive interim data presented in November 2022 [...]
ORLADEYO build on strong initial launch
BioCryst Pharmaceuticals, Inc. reports financial results for the fourth quarter and full year ended 31 December 2022, and provides a corporate update. “It has been exciting to build on our strong initial launch by doubling [...]
KalVista Provides Progress Updates on Sebetralstat
KalVista Pharmaceuticals, Inc. provides multiple clinical trial and regulatory updates for its lead compound sebetralstat, as a potential oral on-demand therapy for HAE attacks. KalVista has enrolled more than 50% of the 114 targeted number [...]
Astria Initiates Clinical Trial Of STAR-0215
Astria Therapeutics, Inc. initiates the ALPHA-STAR Phase 1b/2 clinical trial of STAR-0215 in people living with HAE. Initial proof-of-concept results in HAE patients from single and multiple-dose cohorts are expected in mid-2024. “Our vision for [...]
The Lancet publishes Sebetralstat Phase 2 Data
The Lancet, the international weekly medical journal, publishes results from the phase 2 trial evaluating the efficacy and safety of KalVista Pharmaceuticals, Inc.'s oral sebetralstat for the on-demand treatment of HAE. The phase 2 study was [...]
KalVista Presents Data at WSAAI 60th Annual Scientific Session
KalVista Pharmaceuticals, Inc. presents three posters at the Western Society of Allergy, Asthma & Immunology 60th Annual Scientific Session in Kona, the United States. The first highlights that KONFIDENT-S (NCT05505916) is a prospective trial to [...]
FDA Approves TAKHZYRO® to Prevent HAE Attacks in Children 2 Years of Age and Older
The U.S. Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for the expanded use of Takeda's TAKHZYRO® (lanadelumab-flyo) for prophylaxis to prevent attacks of HAE in pediatric patients 2 to <12 [...]
BioCryst enrolling first patient in pediatric trial
BioCryst Pharmaceuticals, Inc. announces the enrollment of the first patient in the pivotal APeX-P trial evaluating oral, once-daily ORLADEYO® (berotralstat) in pediatric HAE patients who are 2 to <12 years of age. “Today’s announcement marks [...]
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