MELBOURNE, Australia – Neuren Pharmaceuticals Limited (ASX: NEU) has received clear and constructive guidance from the FDA Office of Neuroscience in pre-IND meetings for Phase 2 clinical trials of NNZ-2591 in Phelan-McDermid, Angelman and Pitt Hopkins syndromes. All three syndromes are serious neurological disorders that emerge in early childhood and have no...
treatment News
STOCKHOLM – Swedish Orphan Biovitrum AB (publ) (Sobi™) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) (GLOBE NEWSWIRE) today reported positive top-line results from the phase 3 PRINCE study evaluating the efficacy and safety of pegcetacoplan in adults with paroxysmal nocturnal haemoglobinuria (PNH) who are treatment naïve, meaning they had not received a...
ATLANTA — UCB, a global biopharmaceutical company, will present a post hoc analysis of the EXXELERATE trial examining the efficacy of CIMZIA® (certolizumab pegol) and adalimumab in patients with rheumatoid arthritis (RA) with high rheumatoid factor (RF) levels. The data are being presented at the American College of Rheumatology (ACR)...
JERSEY CITY, N.J. – Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced a post-hoc analysis of its Phase 3 edaravone study reviewing the results of intravenous (IV) edaravone treatment on disease progression milestones and events among people with amyotrophic lateral sclerosis (ALS). In the analysis, a risk reduction was observed...
HAIFA, Israel and MIAMI, FL — Insightec, a global healthcare company dedicated to using acoustic energy to transform patient care, today announced a promising clinical study in the treatment of Alzheimer’s disease leveraging focused ultrasound (FUS) technology, which has been reported in the latest issue of The New England Journal...
Pittsburgh, PA – In a Nature Metabolism paper published today, researchers from the University of Pittsburgh detail a potential new target and a small-molecule drug candidate for treating Barth syndrome, a rare, life-threatening and currently incurable genetic disease with devastating consequences. Barth syndrome affects about 1 in every 300,000 to...
Utrecht, Netherlands – The Organoid Group (Hubrecht Institute) and the Rare Cancers Genomics Team (IARC/WHO) found a way to grow samples of different types of neuroendocrine tumors (NETs) in the lab. While generating their new model, the researchers discovered that some pulmonary NETs need the protein EGF to be able...
NASHVILLE, Tenn. – A potential schizophrenia treatment discovered through the Warren Center for Neuroscience Drug Discovery has been cleared by the U.S. Food and Drug Administration for use in phase 1 clinical trials—the third WCNDD therapeutic to reach that benchmark. The drug is an allosteric modulator that works through a...
BIRMINGHAM, Ala. – It has been uncertain how Mycobacterium tuberculosis deflects the immune response in humans, though evidence has pointed to host immunometabolism — the intrinsic link between metabolism in immune cells and their immune function. The pathogen M. tuberculosis is known to disrupt a metabolic pathway called glycolysis in...
GERMANTOWN, Md. – Precigen, Inc., a biopharmaceutical company specializing in the development of innovative gene and cell therapies to improve the lives of patients, today announced that the first patient has been dosed in the Phase I study of PRGN-2012, a first-in-class, investigational off-the-shelf (OTS) AdenoVerse™ immunotherapy in adult patients with recurrent respiratory...