LEO Pharma and PellePharm Announce $760 Million Collaboration to Advance Innovative Therapies for Rare Skin Diseases

SAN FRANCISCO & BALLERUP, Denmark–(BUSINESS WIRE)– Danish dermatology specialists LEO Pharma and California rare disease pioneers PellePharm today announced a strategic development and commercialization collaboration to address unmet medical needs across various skin diseases with no approved treatments, advancing innovation and access to potential therapies for patients with life-altering conditions, such as Gorlin Syndrome and High Frequency Basal Cell […]

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). […]