BridgeBio Pharma LLC Signs Joint Collaboration Agreement with Cincinnati Children’s to Accelerate Development of Genetic Disease Therapeutics

PALO ALTO, Calif. and CINCINNATI, Nov. 12, 2018 /PRNewswire/ — BridgeBio Pharma LLC and Cincinnati Children’s Hospital Medical Center today announced they are entering into a research collaboration to identify and develop genetic disease therapies. This partnership will combine BridgeBio’s strengths in therapeutic development for diseases of high unmet need with Cincinnati Children’s world-class researchers and clinicians. As a global research center of […]

Eidos Therapeutics Announces Positive Phase 2 Data for AG10 in Symptomatic Patients with Mutant or Wild-Type TTR Amyloid Cardiomyopathy

SAN FRANCISCO, Nov. 10, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced positive results of its Phase 2 clinical trial studying AG10 in subjects with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM). The data were presented in a late-breaking Featured Science oral presentation at the American Heart Association (AHA) Scientific Sessions. AG10 was […]

Eidos Therapeutics Receives Positive Opinion for Orphan Designation from the European Medicines Agency for AG10, a Potent Oral Stabilizer for the Treatment of Transthyretin Amyloidosis

SAN FRANCISCO, Oct. 26, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), announced today that the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency (EMA) adopted a positive opinion for the designation of Eidos’ product candidate, AG10, as an orphan medicinal product for the treatment of transthyretin (TTR) amyloidosis (ATTR). In […]

Data Published in Nature Communications Demonstrates CoA Therapeutics’ Potential to Treat Genetically Driven Pantothenate Kinase-Associated Neurodegeneration (PKAN)

PALO ALTO, Calif., Oct. 23, 2018 /PRNewswire/ — CoA Therapeutics, a company developing small molecules that target Coenzyme-A for patients with PKAN and other diseases of CoA sequestration, today announced key data published by researchers from St. Jude Children’s Research Hospital in Nature Communications demonstrating the potential of small molecule activators of pantothenate kinase for the treatment of pantothenate kinase-associated […]

QED Therapeutics Presents Data for Infigratinib in Cholangiocarcinoma in Late Breaking Abstract at the European Society of Medical Oncology 2018 Congress

SAN FRANCISCO, Oct. 19, 2018 /PRNewswire/ — QED Therapeutics today announced the presentation of positive, updated interim data of infigratinib (BGJ398), an orally administered, selective fibroblast growth factor receptor (FGFR) 1-3 tyrosine kinase inhibitor, for the treatment of advanced or metastatic cholangiocarcinoma (bile duct cancer) during a late-breaking poster discussion at the European Society for Medical Oncology (ESMO). […]

PellePharm Presents Updated Data from Two Phase 2 Studies Demonstrating the Potential of Patidegib Topical Gel to Treat Basal Cell Carcinomas in Patients with Gorlin Syndrome and Patients with Non-Gorlin Sporadic BCCs

SAN FRANCISCO–(BUSINESS WIRE)–PellePharm, a late clinical-stage biopharmaceutical company committed to targeting rare skin conditions at their source, today presented updated clinical data from two Phase 2 studies of patidegib topical gel in a poster session at NORD’s Rare Diseases and Orphan Products Breakthrough Summit 2018 in Washington, D.C. Results of the studies showed clinical clearance […]

Eidos Therapeutics to Present Phase 2 Data for AG10 in TTR Amyloidosis Cardiomyopathy at the AHA 2018 Scientific Sessions in a Late-Breaking Featured Science Oral Presentation

SAN FRANCISCO, Oct. 05, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), today announced that results of its Phase 2 clinical trial studying AG10 in subjects with symptomatic transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM) will be announced in a late-breaking featured science oral presentation at this year’s American Heart Association (AHA) Scientific Sessions. Daniel Judge, […]

FDA Grants Orphan Drug Designation to Eidos Therapeutics’ Product Candidate, AG10, for Treatment of Transthyretin Amyloidosis

SAN FRANCISCO, Oct. 03, 2018 (GLOBE NEWSWIRE) — Eidos Therapeutics, Inc. (Eidos) (Nasdaq:EIDX), a clinical stage biopharmaceutical company focused on addressing the large and growing unmet need in diseases caused by transthyretin (TTR) amyloidosis (ATTR), announced today that the U.S. Food and Drug Administration (FDA) has granted the company Orphan Drug Designation for AG10 for […]