Agios Pharmaceuticals Inc. said the patients’ deaths tied to anemia therapy Pyrukynd (mitapivat) that were announced in a securities analyst’s Aug. 4 note to investors hasn’t changed the drug’s already established benefit-risk profile on the drug’s U.S. prescribing information.
Vertex Pharmaceuticals Inc.’s bad news from a phase II pain study and separately on the regulatory front caused shares (NASDAQ:VRTX) to close Aug. 5 at $393.83, down $78.44, or 16%. Vertex rolled out top-line results from the phase II dose-ranging study to test the safety and efficacy of its NaV1.8 pain signal inhibitor, VX-993, in tackling acute pain after bunionectomy.
Novartis AG’s financial update included good news about Scemblix (asciminib), the first and only U.S. FDA-approved allosteric inhibitor for chronic myeloid leukemia (CML), which gained clearance in October 2021. The drug introduced a new mechanism of action by specifically targeting the ABL myristoyl pocket.
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.
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.
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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