Solid Biosciences Inc.'s chief technology officer, Joel Schneider, said the company is "not going to leave any stone unturned" as investigators begin to explore the severe adverse event (SAE) that led to the FDA's clinical hold on the phase I/II study with SGT-001 in Duchenne muscular dystrophy (DMD).
LONDON – Sanifit Therapeutics SA has delivered positive phase IIb data demonstrating SNF-472 significantly reduces progression of cardiovascular calcification (CVC) in patients with end-stage kidney disease who are on dialysis.
Roche Holding AG and PTC Therapeutics Inc. together with their partner, the SMA Foundation, reported success in part two of the pivotal Sunfish study testing their survival motor neuron-2 splicing modifier risdiplam in patients with type 2 or 3 spinal muscular atrophy (SMA).
Shares of Nextcure Inc. (NASDAQ:NXTC) have alternately been up sharply lately or down dramatically. Following the weekend release of updated positive clinical data from its NC-318 phase I/II study, the share price nosed down 52.9% Monday.
Within the first few minutes of a key opinion leader webinar on Alzheimer's disease sponsored by AC Immune SA, CEO Andrea Pfeifer brought up the decision by Biogen Inc. to file for regulatory approval of amyloid beta targeter aducanumab in early AD, based on results from a subset of patients in the phase III study called Engage.
In the wake of each dosing arms' failure to hit the primary endpoint in Anaptysbio Inc.'s phase IIb study of etokimab to treat moderate to severe atopic dermatitis (AD), the stock dropped steeply and the company postponed initiating its phase IIb etokimab trial in eosinophilic asthma until it analyzes the phase IIb result.
Allena Pharmaceuticals Inc.'s phase III URIROX-1 trial of reloxaliase hit its primary endpoint, with a statistically significant reduction in urinary oxalate (Uox) in patients with enteric hyperoxaluria (EH), while its phase II Study 206 trial showed reduced plasma oxalate reduction in patients with EH and advanced chronic kidney disease (CKD).
DUBLIN – Obseva SA's stock plunged 65% Thursday on news that it was terminating development of its in vitro fertilization (IVF) drug, nolasiban, after it missed the primary endpoint of a confirmatory phase III trial. The result blindsided both Obseva and investors, as the outcome was at odds with that of a previous phase III study, Implant 2, which had demonstrated a statistically significant and clinically relevant improvement in the rate of successful pregnancy at 12 weeks.
Emerging from the tsunami of abstracts from the nearly here American Society of Hematology (ASH) annual meeting in Orlando, Fla., were data that sent some stocks upward on Wednesday. Most notably, Constellation Pharmaceuticals Inc. (NASDAQ:CNST) was a huge winner as its shares nearly doubled in value as they closed at $28,10, up $13.33, for a gain of 90.25%.
Supernus Pharmaceuticals Inc. CEO Jack Khattar said the phase III failure in the study called P301 with SPN-810 (molindone hydrochloride) was "very puzzling" to the Rockville, Md.-based firm, which will be "digging very deep into the data" with hopes of figuring out what went wrong. Shares (NASDAQ:SUPN) closed Wednesday at $19.93, down $9.20, or 31.6%.
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