At the American College of Asthma, Allergy & Immunology (ACAAI) Annual Scientific Meeting, Ionis Pharmaceuticals, Inc. presents positive results from the Phase 2 study of its investigational antisense medicine, donidalorsen (formerly IONIS-PKK-LRx). The Phase 2 study results support the clinical profile of donidalorsen as a potential, best-in-class prophylactic treatment for patients with HAE, and underscore Ionis' [...]
At the American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, KalVista Pharmaceuticals, Inc. presents data on Phase 2 for KVD900, KalVista’s lead program for oral on-demand treatment of HAE attacks. “Current guidelines recommend effective on-demand therapy for every patient with HAE to reduce symptom severity and attack duration,” says Jonathan Bernstein, FAAAAI, FACAAI, FACP, [...]
At the 2021 Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), BioCryst Pharmaceuticals, Inc. presents new long-term efficacy and safety data from studies evaluating oral, once-daily Orladeyo (berotralstat) for the prophylactic treatment of HAE. “We continue to see improvement in key indicators that demonstrate the value of oral, once-daily Orladeyo as an [...]
From the Ionis Pharmaceuticals, Inc. financial results for the three and nine months ended 30 September 2021: "We are looking forward to multiple near-term catalysts, beginning with the presentation of donidalorsen Phase 2 data in patients with HAE this month. We anticipate initiating the donidalorsen Phase 3 study before year-end", says Brett P. Monia, Ph.D., [...]
Announcing the financial results for the third quarter ended 30 September 2021, BioCryst Pharmaceuticals, Inc. President and CEO Jon Stonehouse says: “With the significant revenue we expect to generate with Orladeyo - our oral, once-daily treatment for prevention of HAE attacks - in its first year of launch, BioCryst plans to repeat our clinical and commercial success with [...]
Adalvo is pleased to announce the successful closure on the DCP procedure for our Icatibant development, a complex injectable peptide product, forming part of our high-value product portfolio offering to our partners. Icatibant is an equivalent and more affordable version of Firazyr that will now be made more accessible to patients world-wide. The product is [...]
Plasma helps your body fight diseases, and it deserves greater recognition. Ask people who have certain rare and chronic complex diseases – they will tell you that therapies made from donated plasma help them manage conditions such as immunodeficiency disorders, hemophilia, and HAE. Plasma is a core substance within our blood and carries important cells, [...]
Intellia Therapeutics, Inc.'s Clinical Trial Application (CTA) has been authorized by the New Zealand Medicines and Medical Devices Safety Authority (MEDSAFE) to initiate a Phase 1/2 study evaluating NTLA-2002 for the treatment of adults with HAE. NTLA-2002 is a systemically administered single-dose CRISPR/Cas9-based therapeutic candidate designed to inactivate the target gene Kallikrein B1 (KLKB1) to [...]
Cycle Pharmaceuticals launces Sajazir (icatibant) Injection, a new treatment option for patients affected by HAE approved by the US Food and Drug Administration (FDA). Sajazir is a bradykinin B2 receptor antagonist indicated for treatment of acute attacks of HAE in adults 18 years of age and older. Sajazir is supplied in a prefilled syringe delivering 30 [...]
CAMP4 Therapeutics presents preclinical data demonstrating the utility of its RNA Actuating Platform (RAP) and the therapeutic potential of oligonucleotide-mediated gene upregulation targeting a new class of RNA known as regulatory RNAs (“regRNAs”). The data describe this novel treatment approach for two genetically defined diseases, one being HAE. “These data demonstrate that by using oligonucleotides [...]
