A Chinese study has identified small-molecule compounds that can selectively reduce levels of the mutant Huntingtin (HTT) protein involved in the pathogenesis of Huntington's disease (HD), and possibly other neurodegenerative diseases involving mutant protein accumulation.

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.

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.

Given how many patients who completed the double-blind portion of the study opted into the extension segment and the quality of pharmacokinetic (PK) data, phase III testing of Intec Pharma Ltd.'s Accordion Pill (AP, carbidopa/levodopa [CD/LD]) against immediate-release CD/LD in Parkinson's disease (PD) seemed likely to ring the statistical significance bell.

Drug "re-innovation" specialist Bioxcel Therapeutics Inc. is gearing up to discuss a pivotal phase III trial for BXCL-501 with the FDA after top-line results of a phase Ib study showed it reduced schizophrenia-associated agitation while calming trial participants without excessive sedation. H.C. Wainwright & Co. analyst Raghuram Selvaraju called the results "highly favorable" and suggested the drug, a sublingual-formulated version of dexmedetomidine, could reach market as soon as 2021.

Rising from Case Western Reserve in Cleveland was science that Roche Holding AG's Genentech Inc. could not pass up, so it struck a deal with Convelo Therapeutics Inc. to discover and develop remyelinating medicines for patients with neurological disorders such as multiple sclerosis.

LONDON – Cytox Ltd. is launching a genetic test that can predict the risk of developing Alzheimer's disease and is able to single out which people presenting with symptoms of mild cognitive decline will go on to most rapidly develop Alzheimer's disease.

Prospects for the new wave of migraine therapies targeting calcitonin gene-related peptide (CGRP) – with three approved already and more in developers' pipelines – have been the topic of talk lately, with analysts such as Evercore ISI's Umer Raffat pointing to what some might see as a dispiriting level-out in sales among those treatments to reach the market so far.

Word from the FDA to Axsome Therapeutics Inc. about the effort with oral N-methyl-D-aspartate (NMDA) receptor antagonist AXS-05 in depression – and the company's accelerated push with the compound – had Wall Street watching the NMDA space with even more interest than usual.