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WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a nonprofit biotechnology organization developing genetic therapies for ultra-rare diseases, today announced the successful completion of a pre-Investigational New Drug (pre-IND) meeting with the U.S. Food and Drug Administration (FDA) regarding its investigational gene therapy program for Limb-Girdle Muscular Dystrophy type 2I/R9 (LGMDR9). The LGMDR9 program, known as CRD-003, utilizes a novel, engineered AAVMYO2 capsid to deliver a...

SANTA CLARA, Calif.--(BUSINESS WIRE)--Ancora Heart has reached the first enrollment milestone in the CORCINCH-HF pivotal trial of the AccuCinch® System in patients with heart failure....

PASADENA, Calif.--(BUSINESS WIRE)--Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that it has completed enrollment in SHASTA-3, SHASTA-4, and MUIR-3, the company’s global Phase 3 clinical studies designed to support regulatory submissions for marketing approval of investigational plozasiran in the treatment of severe hypertriglyceridemia. Arrowhead anticipates completing the primary portion of these studies in mid-2026 with topline data expected shortly thereafter and planned su...

LONDON & NEW YORK--(BUSINESS WIRE)--Compass Pathways plc (Nasdaq: CMPS), a biotechnology company dedicated to accelerating patient access to evidence-based innovation, announced today the successful achievement of the primary endpoint in the ongoing Phase 3 COMP005 trial, the first of two Phase 3 trials evaluating COMP360, a synthetic, proprietary formulation of psilocybin, for treatment-resistant depression (TRD). The primary endpoint is the difference in change from baseline in the Montgomery...

WILMINGTON, Del.--(BUSINESS WIRE)--Update on FDA Review of Ruxolitinib Cream (Opzelura) for Children Ages 2-11 with Atopic Dermatitis...

MADISON, Wis.--(BUSINESS WIRE)--Archeus Technologies, a company developing radiopharmaceutical therapies for the treatment of patients with cancer, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for ART-101, a novel receptor-based targeting small molecule that Archeus has developed for the imaging and treatment of prostate cancer. This IND clearance enables Archeus to initiate a Phase 1 clinical trial in men with metas...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), presented today results from the Phase III SUNMO [NCT05171647] study showing Lunsumio® (mosunetuzumab-axgb) administered subcutaneously in combination with Polivy® (polatuzumab vedotin-piiq) demonstrated a clinically meaningful and statistically significant improvement in its primary endpoints of progression-free survival (PFS) and objective response rate (ORR) compared to Rituxan®...

ALAMEDA, Calif.--(BUSINESS WIRE)--Exelixis, Inc. (Nasdaq: EXEL) today announced that its partner Ipsen received a positive opinion from the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for CABOMETYX® (cabozantinib) for adult patients with unresectable or metastatic, well-differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumors who have progressed following at least one prior systemic therapy other than somatostatin analogues....

BOSTON & CAMBRIDGE, England--(BUSINESS WIRE)--Cycle Pharmaceuticals announced that the FDA has approved HARLIKU (nitisinone) Tablets for the reduction of urine homogentisic acid (HGA) in adult patients with AKU.1 Launching in July 2025, HARLIKU will be the first and only FDA-approved treatment for AKU,1 an ultra-rare genetic metabolic disorder in which patients have a buildup of HGA that leads to osteoarthritis, ochronosis, and complications in the kidneys, and heart.2 Patients with AKU often d...

NEW YORK--(BUSINESS WIRE)--FDA APPROVES SMILE Dx....