The prospects for Ultragenyx Pharmaceutical Inc.'s adeno-associated virus (AAV) gene therapy, DTX-301, in patients with ornithine transcarbamylase (OTC) deficiency weren't looking too great after treatment of the first two cohorts in a phase I/II study.
Applied Therapeutics Inc. (ATI) CEO Shoshana Shendelman said positive top-line findings with aldose reductase inhibitor AT-007 in galactosemia were “the data we needed, and we’re going to move as quickly as we can to get in and speak to the FDA about this, not only to firm up our NDA filing but also to get into the pediatric study.”
While the disappearing drug-eluting stent seems to have faded from view for the time being, several manufacturers have invested in fabrication of drug-eluting stents with polymers that absorb once the drug of elution has done its work. A new study indicates that patients who have already had a myocardial infarction fare better on stents made with these biodegradable polymers, an outcome that may soon push second-generation DES devices into med-tech history.
Apellis Pharmaceuticals Inc.’s CEO, Cedric Francois, said his firm’s phase III study called Pegasus testing pegcetacoplan, or APL-2, in paroxysmal nocturnal hemoglobinuria (PNH) “finally established that there is an important unmet medical need in this disease.”
Saluda Medical Pty. Ltd., of Artarmon, Australia, made waves in 2019, winning the CE mark for its Evoke closed-loop spinal cord stimulation (SCS) system. The good news kept coming following the publication of results from a pivotal study in The Lancet Neurology.
LONDON – A high level U.K. group, involving industry, academia, patients, health technology assessment and regulatory representatives, has set out guidelines for designing complex cancer trials and is calling for their rapid implementation to reduce clinical development timelines.
A closely watched bid by Incyte Corp. to tackle acute graft-vs.-host-disease (GVHD) with the JAK1 inhibitor itacitinib has fallen flat in pivotal testing, leaving analysts skeptical about the drug's future and pessimistic about the company's R&D chops.
While Novan Inc.’s phase III failure in treating molluscum contagiosum for patients ages 6 months and older battered its stock Friday, the company intends to take the positive data it gleaned to the FDA to discuss an NDA submission and a possible phase III trial for next year.