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.

PERTH, Australia – Telix Pharmaceuticals Ltd. raised AU$40 million as it prepares regulatory packages and commercial strategies in Europe, the U.S. and Australia.

What the phase III multiple sclerosis (MS) win scored by Biogen Inc. and partner Alkermes plc might mean in the marketplace for diroximel fumarate – tested against their approved MS therapy Tecfidera (dimethyl fumarate) – became fodder for speculation as investors weighed the fine points of side-effect profiles and would-be reimbursement hitches.

The failure of Novartis AG's Entresto in a phase III clinical trial staggered the stock (NASDAQ:NVS) somewhat Monday, down just 1.14%, but the real trauma may well be the loss of roughly $2.5 billion in anticipated sales.

In late June, when Pfizer Inc. unveiled the first phase Ib data, mixed safety signal and all, for its Duchenne muscular dystrophy (DMD) gene therapy, PF-06939926, investors in Sarepta Therapeutics Inc. as well as Solid Biosciences Inc. watched with particular interest. The latter firm seems none the worse for wear, though, raising $60 million in a private placement.

Following more than a year of restructuring and recalibration, Regulus Therapeutics Inc. is facing a new partial clinical hold placed by the FDA on its phase I test of RGLS-4326, an oligonucleotide it's developing for the treatment of autosomal dominant polycystic kidney disease (ADPKD). 

MEXICO CITY – Merck Sharp & Dohme's MK-8591, a nucleoside reverse transcriptase translocation inhibitor (NRTTI), impressed in both treatment and prevention studies at the 2019 International AIDS Society (IAS) Conference on HIV Science.

Although the placebo response proved better than expected in Chiasma Inc.'s phase III trial of Mycapssa (octreotide) capsules for acromegaly, "we believe that the response of the patients on Mycapssa is the clinically relevant indicator," said William Ludlam, the Waltham, Mass.-based firm's senior vice president of clinical development and medical affairs.

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.