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.

PERTH, Australia – San Diego-based Okogen Inc. is taking its clinical trial program to Australia where it will test its lead compound OKG-0301 for acute adenoviral conjunctivitis in the phase II (RUBY) trial.

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.

HONG KONG – China has approved the fourth homegrown CD47 antibody to start clinical trials, intensifying the local CD47 race. Chinese biologics developer I-Mab Biopharma (Shanghai) Co. Ltd. said it received the go-ahead for the clinical study of TJC-4, its differentiated fully human CD47 monoclonal antibody for treating advanced malignant tumors.

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.