Praxis Precision Medicines Inc. is making a change to its upcoming registrational, 400-patient Power2 study – adding a dose arm plus a depression/mood endpoint – based on encouraging phase II data from the Radiant study with vormatrigine in focal onset seizure (FOS) patients over an eight-week period. Specifically, Power2 will test 20 mg, 30 mg, and 40 mg of the drug against placebo for 12 weeks.
With the U.S. FDA’s approval of Lenz Therapeutics Inc.’s Vizz (aceclidine ophthalmic solution), there is a third eye drop on the market for treating presbyopia. Vizz, however, is the first aceclidine-based eye drop for adults.
Madrigal Pharmaceuticals Inc.’s long-awaited business development pact became reality by way of an exclusive global license agreement that could be worth more than $2 billion with CSPC Pharmaceutical Group Ltd., of Shijiazhuang, China, for SYH-2086. The candidate is a preclinical oral, small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist and orforglipron derivative.
Alterity Therapeutics Ltd. helped develop a new neuroimaging biomarker called the multiple system atrophy index (MSA-AI), which looks to be a more reliable biomarker for tracking disease progression of MSA. Developed using deep learning methods, the MSA-AI offers a superior, objective and quantifiable measure of brain atrophy in MSA patients.
Cardiff Oncology Inc.’s mid-stage and ongoing clinical trial of its PLK1 inhibitor in colorectal cancer produced promising data but the company’s stock lost a quarter of its value on July 30 because of second-quarter losses. The randomized phase II study of onvansertib combined with a standard-of-care treatment in first-line, RAS-mutated metastatic colorectal cancer produced a 49% confirmed objective response rate at the 30-mg dose level compared to 30% in the control arm.
Madrigal Pharmaceuticals Inc.’s long-awaited business development pact became reality by way of an exclusive global license agreement that could be worth more than $2 billion with CSPC Pharmaceutical Group Ltd., of Shijiazhuang, China, for SYH-2086. The candidate is a preclinical oral, small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist and orforglipron derivative.
Suzhou, China-based Kintor Pharmaceutical Ltd.’s KX-826 met the primary endpoint in a phase II trial for treating male adult androgenetic alopecia (AGA). Kintor is developing KX-826 (pyrilutamide), an androgen receptor inhibitor, for topical treatment of AGA as well as acne vulgaris.
Amplia Therapeutics Ltd. is raising AU$27.5 million (US$18.12 million) to advance lead compound narmafotinib (AMP-945), a focal adhesion kinase (FAK) inhibitor, into new indications beyond pancreatic cancer.
PTC Therapeutics Inc. will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission, roughly three months after a thumbs up from the EMA’s Committee for Medicinal Products for Human Use.
With more-than-satisfying phase III data in hand, Minneapolis-based Celcuity Inc. is eyeing an NDA submission in the fourth quarter of this year for gedatolisib (geda) in breast cancer. Shares of the company (NASDAQ:CELC) closed July 28 at $36.79, up $23.02, or 167%, after Wall Street learned of stellar top-line results from the PIK3CA wild-type cohort of the phase III Viktoria-1 trial.
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