Higher-than-expected serum bicarbonate values in the placebo group foiled Tricida Inc.’s effort in its phase III renal outcomes trial to compare an untreated acidotic chronic kidney disease (CKD) population with those given veverimer. The hitch meant the company could not measure the oral, non-absorbed polymer’s ability to slow the disease in patients with metabolic acidosis, an estimated market of several million people in the U.S.
Mirum Pharmaceuticals Inc. plans to “move rapidly toward regulatory filings” to expand use of IBAT inhibitor Livmarli (maralixibat) into progressive familial intrahepatic cholestasis (PFIC), President and CEO Chris Peetz told investors during a conference call Oct. 24 to discuss the positive top-line results from the phase III March study, which not only hit its primary endpoint in patients with PFIC2 but showed statistical improvements in pruritis across a range of PFIC subtypes.
A second death in the study of davoceticept (ALPN-202) has prompted Alpine Immune Sciences Inc. to stop enrolling participants in two phase I studies of the CD28 co-stimulator and dual checkpoint inhibitor, throwing the program’s future into doubt.
Immunic Inc. plans to forge ahead with oral psoriasis therapy IMU-935 despite stock-punishing results from a phase Ib trial that showed a higher placebo response than expected. Shares of the New York-based firm (NASDAQ:IMUX) closed at $2.08, down $7.12, or 77.4% after findings were made public from a preplanned interim group-level data analysis of the ongoing experiment, which is testing patients with moderate to severe disease. The group averages for Psoriasis Area and Severity Index (PASI) reductions in the two active arms failed to separate from placebo at four weeks, Immunic said. Although the active arms performed as intended, placebo decreases in PASI proved greater than hoped.
Galecto Inc.’s late-September news from its ongoing phase IIa trial with GB-2064 in myelofibrosis hiked shares of the Boston-based firm and piqued interest in the lysyl oxidase-like 2 (LOXL2) target, where such names as Gilead Sciences Inc. and Pharmaxis Ltd. also have been busy.
The U.S. FDA wants more data on PTC-518 before PTC Therapeutics Inc.'s phase II study of Huntington’s disease can continue enrollment. While stopped in the U.S., the study of the oral, small-molecule splicing modifier still is enrolling participants at sites in several European countries and in Australia.
Radiopharm Theranostics Ltd.’s phase II F-18 Pivalate positron emission tomography trial in brain metastases saw positive results with high uptake regardless of the origin of primary tumors, showing that Pivalate could be used to monitor brain metastases.
Milestone Pharmaceuticals Inc. saw a premarket surge in its share price value after a phase III trial of the company’s self-administered, heart rhythm-restoring nasal spray, etripamil, hit its target in a phase III trial, setting up a potential filing with the U.S. FDA in mid-2023.
Over the next two weeks, the team at NGM Biopharmaceuticals Inc. will be digging through the data from its phase II trial of complement 3 inhibitor NGM-621 in patients with geographic atrophy (GA) secondary to age-related macular degeneration, following reports of a top-line miss Oct. 17 and a severe reaction from investors, who sent shares (NASDAQ:NGM) plummeting 70.5% to close at $3.41.
GSK plc has announced results from its respiratory syncytial virus (RSV) vaccine in older people that look stronger than those from its rival, Pfizer Inc., as the race to develop a first ever vaccine against the common respiratory disease heats up.
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