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,...
treatment News
PHILADELPHIA, PA — Aro Biotherapeutics, a clinical-stage biotechnology company developing potent and tissue-targeted short-interfering RNA (siRNA) medicines in areas of high unmet need in rare muscle diseases, immunology, and oncology, today announced positive topline results from the Phase 1b portion of its study evaluating ABX1100, a novel investigational therapy for...
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...
PASADENA, Calif. — Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the Chinese National Medical Products Administration (NMPA) has approved REDEMPLO® (plozasiran) for the reduction of triglyceride levels in adult patients with familial chylomicronemia syndrome (FCS). FCS is a severe, rare disease characterized by triglyceride levels that can be 10 to...
PASADENA, Calif. — Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has dosed the first subjects in a Phase 1/2a clinical trial of ARO-DIMER-PA, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia. ARO-DIMER-PA is designed...