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.
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.
Hemispherian AS has received IND clearance for GLIX-1 for the treatment of glioblastoma from the U.S. FDA. GLIX-1 targets DNA repair mechanisms, specifically the enzyme TET2, selectively in tumor cells while sparing healthy tissues.
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.
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