BRIDGEWATER, N.J. – Insmed Incorporated (Nasdaq:INSM), a global biopharmaceutical company on a mission to transform the lives of patients with serious and rare diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved ARIKAYCE®(amikacin liposome inhalation suspension) for the treatment of patients with nontuberculous mycobacterial (NTM)...
treatment News
Watertown, Mass. — Arkuda Therapeutics, a biotechnology company applying novel insights at the intersection of lysosomal biology and neuronal health to develop medicines that change the trajectory of neurodegenerative disease, announced Arkuda co-founder and previous Senior Vice President, Head of Discovery, Duane Burnett, PhD, has been appointed Chief Technology Officer,...
ARDSLEY, N.Y. – ARMGO Pharma Inc., a clinical stage biopharmaceutical company advancing a novel class of small molecule drugs known as Rycals®, announced today the publication of the results from a Phase 1b study of its Rycal ARM210 (also known as S48168), for the treatment of Ryanodine Receptor 1 Related...
PHILADELPHIA, PA — Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines, today announced that it has completed enrollment in its ongoing Phase 1b clinical trial of ABX1100, a novel investigational therapy for the treatment of late-onset Pompe disease (LOPD). The trial is evaluating safety,...
WALTHAM, Mass. — Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced the presentation of preclinical data demonstrating the progress of its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1),...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced positive interim 48-week liver biopsy results from the AROAAT2002 study, an open-label Phase 2 clinical study of ARO-AAT, the company’s second generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited (“Takeda”) as a treatment for the rare...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced positive interim results from the first two cohorts of AROHIF21001, a Phase 1b dose-finding clinical study of ARO-HIF2, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with clear cell renal cell carcinoma (ccRCC). ARO-HIF2...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced positive interim results from the first two cohorts of AROHIF21001, a Phase 1b dose-finding clinical study of ARO-HIF2, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with clear cell renal cell carcinoma (ccRCC). ARO-HIF2 is...
PASADENA, Calif. – Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today notified regulatory agencies, institutional review boards, and investigators that effective immediately it is voluntarily pausing AROENaC1001, a Phase 1/2 clinical study of ARO-ENaC, the company’s investigational RNA interference (RNAi) therapeutic being developed as a treatment for patients with cystic fibrosis (CF),...
PASADENA, Calif. – Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the U.S. Food and Drug Administration (FDA) has approved REDEMPLO (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). FCS is a severe, rare disease, with...