Just as artificial intelligence (AI) becomes an ever-more common part of drug discovery, its potential role in clinical trials is slowly becoming more visible, too. Efforts to improve trial recruitment, efficiency and decision-making are underway at companies of all sizes as organizations look to better the oft-daunting odds of clinical success, industry executives told BioWorld.
Top-line results from The Medicines Co.'s first pivotal phase III trial of the cholesterol-lowering candidate inclisiran met all primary and secondary efficacy endpoints, with safety "at least as favorable" as in two recent studies, the company said, addressing a top concern for investors tracking the program. Though final results of the study, Orion-11, won't be made public until next week's European Society of Cardiology Congress (ESC) in Paris, excitement about what Evercore ISI analyst Umer Raffat said could be the "first 'vaccine' for cholesterol management," dosed twice-yearly, lifted Medco shares (NASDAQ:MDCO) 13.8% to $39.85 on Monday.
Graybug Vision Inc., a Redwood City, Calif.-based company developing therapies for eye diseases, has secured $80 million in series C financing to support moving its lead candidate, GB-102, into midstage studies in both wet age-related macular degeneration (AMD) and macular edema.
IGM Biosciences Inc., a Mountain View, Calif.-based biotech developing engineered immunoglobulin M antibodies for the treatment of cancer, has filed to raise up to $100 million in a Nasdaq IPO little more than a month after closing a $102 million series C financing. The company's lead candidate, IGM-2323, is expected to enter phase I testing for the potential treatment of relapsed/refractory (r/r) B-cell non-Hodgkin lymphoma (NHL) this year.
Sarepta Therapeutics Inc. has received a complete response letter (CRL) from the FDA as the agency declined to issue a sought-after accelerated approval for the company's Duchenne muscular dystrophy (DMD) follow-on therapy, Vyondys 53 (golodirsen).
Nabriva Therapeutics plc has gained FDA approval of NDAs for both I.V. and oral formulations of lefamulin, a semisynthetic antibiotic for the treatment of community-acquired bacterial pneumonia (CABP) it will market as Xenleta. It's the first pleuromutilin for systemic use in humans to win FDA approval, leading the first new class of antibiotics for CABP in almost 20 years.
Following a priority review at the FDA, Abbvie Inc.'s oral rheumatoid arthritis (RA) drug, upadacitinib, has won U.S. approval at the agency, taking the first step into a market that analysts predict could eventually lead to peak annual sales of as much as $2.2 billion by 2023.
Three years after its European approval, U.S. clearance for the narcolepsy drug Wakix (pitolisant) has arrived. Privately held Harmony Biosciences LLC will market the once-daily medicine, licensed from Bioprojet Societe Civile de Recherche, for the treatment of excessive daytime sleepiness (EDS) in adults with the chronic sleep disorder.
Rare disease specialist Ultragenyx Pharmaceutical Inc. has agreed to pay $20 million for an exclusive option to acquire Genetx Biotherapeutics LLC, the developer of a University of Texas-sourced antisense oligonucleotide for the potential treatment of Angelman syndrome, GTX-102. An IND for the asset, now in late preclinical development, is expected to be filed with the FDA in the first half of 2020.
