Netherton syndrome is a rare disease caused by loss of activity of the lympho-epithelial Kazal-type-related inhibitor (LEKTI) protein, which in turn is caused by mutations in its encoding gene, SPINK5. This deficiency leads to the triggering of the kallikrein (KLK) signaling cascade resulting in skin barrier dysfunction, inflammation and atopy. At the recent Society for Investigative Dermatology meeting, Biocryst Pharmaceuticals Inc. presented early data on BCX-17725, a KLK5/KLK14 inhibitor fusion protein developed to restore LEKTI functioning in patients with Netherton syndrome.
Biocryst Pharmaceuticals Inc.’s near-term dilution should “pave the way for entrenchment” in the hereditary angioedema (HAE) space, thanks to the buyout of Astria Therapeutics Inc., RBC analyst Brian Abrahams said.
After delaying a June PDUFA date, the U.S. FDA has approved Kalvista Pharmaceuticals Inc.’s Ekterly (sebetralstat) for hereditary angioedema (HAE) in those aged 12 and older. The plasma kallikrein inhibitor now joins a market with previously approved drugs for the rare, genetic, life-threatening condition, as well as other companies with HAE drugs in development. Ekterly is the first orally-delivered on-demand treatment, as all others in the U.S. are intravenously or subcutaneously administered.
Biocryst Pharmaceuticals Inc. has described kallikrein 5 (KLK5; KLK-L2) inhibitors reported to be useful for the treatment of allergy, asthma, cancer, conjunctivitis, dermatological disorders, gastrointestinal conditions, inflammatory disorders and rhinitis, among others.
Biocryst Pharmaceuticals Inc.’s unveiling of real-world data Feb. 24 with its approved Orladeyo (berotralstat) for prophylactic treatment of hereditary angioedema (HAE) brought more attention to the space, due to yield findings from would-be competitors in the near term.
Biocryst Pharmaceuticals Inc. has disclosed imidazole derivatives acting as activin receptor-like kinase 2 (ALK-2; ActR-IA) inhibitors reported to be useful for the treatment of diffuse intrinsic pontine glioma, spondyloarthritis and fibrodysplasia ossificans progressiva.
Researchers from BioCryst Pharmaceuticals Inc. have developed a series of plasma kallikrein inhibitors with potential for the treatment of hereditary angioedema (HAE).
Elevated serum creatinine levels in some participants prompted Biocryst Pharmaceuticals Inc. to pause three clinical trials involving BCX-9930, an oral, small-molecule factor D inhibitor for treating rare diseases.
