PERTH, Australia – Stem cell therapy has failed to deliver on its promises, according to Exopharm Ltd. founder and CEO Ian Dixon, who said he believes that exosomes, or the extracellular vesicles released by stem cells, could be a disrupter in the regenerative medicine space.

Good news from San Diego-based Acadia Pharmaceuticals Inc.'s phase III trial with Nuplazid (pimavanserin) in dementia-related psychosis (DRP) had analysts trotting out music metaphors in reports about the study called Harmony, while talking already about would-be sales of the drug, a selective serotonin inverse agonist and antagonist that preferentially targets the 5-HT2A receptor.

Shares of New York-based Neurotrope Inc. (NASDAQ:NTRP) fell 77.3% to close at a record low of $1 Monday after a phase II study of its lead candidate, bryostatin-1, failed to outperform a placebo in helping people with moderate to severe Alzheimer's disease (AD) achieve improvement on a standardized measure of cognition.

As Wall Street ponders how pricing and reimbursement for gene therapies might shake out, companies in the field march intrepidly on, among them Orchard Therapeutics Inc., which early this month rolled out heartening results from an ongoing proof-of-concept trial evaluating the safety and efficacy of OTL-203 for mucopolysaccharidosis type I (MPS-I), a space that's heating up.

Findings disclosed from Synlogic Inc.'s phase Ib/IIa trial with SYNB-1020 in hyperammonemia surprised the company and Wall Street, dealing a blow to the compound but leaving in place the Synthetic Biotic platform, which genetically engineers probiotic microbes.

Abbott Laboratories has kicked off a pivotal study to evaluate its catheter-based Triclip transcatheter tricuspid valve repair system in patients with severe tricuspid regurgitation (TR). The first enrollments in the U.S. FDA-approved Triluminate study were performed at Abbott Northwestern Hospital in Minneapolis by a cardiac team led by Paul Sorajja, director of the Center for Valve and Structural Heart Disease at the Minneapolis Heart Institute and co-primary investigator for the trial.

Following its phase II stumble of AR-105, Aridis Pharmaceuticals Inc. is dropping its development of the IgG1 monoclonal antibody. The study failed to meet its primary endpoint of showing superiority in clinical cure rates compared to placebo for treating ventilator-associated pneumonia caused by gram-negative Pseudomonas aeruginosa.

DUBLIN – Shares in Pharnext SA plunged by more than 40% Friday on news that the FDA requires the company to undertake a second phase III trial of its Charcot-Marie-Tooth disease type 1A (CMT1A) therapy PXT-3003.

HONG KONG – Following a regulatory committee's ruling that three of four deaths previously reported during a phase II trial were unrelated to the study drug, South Korea's Ministry of Food and Drug Safety (MFDS) cleared Yuhan Corp. to continue testing lazertinib (YH-25448) in the non-small-cell lung cancer (NSCLC) study.