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.
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.
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.
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.
Sarepta Therapeutics Inc.’s adventure with the Duchenne muscular dystrophy (DMD) AAV-based gene therapy Elevidys (delandistrogene moxeparvovec) continued as the firm said it would restart shipments of the compound for ambulatory patients “imminently,” with the U.S. FDA’s blessing.
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.
Neurogene Inc.’s disclosure June 30 of its registrational trial design in Rett syndrome (RS) added spice to the space, where Taysha Gene Therapies Inc. is another closely watched player. The New York-based firm said Embolden will test NGN-401, a gene therapy for the treatment of females age 3 and older with RS. U.S. regulators have signed off regarding key aspects of Embolden’s structure.
Astrazeneca plc seems on the way to expanding its presence in myasthenia gravis (MG) with positive “high-level” results from a global, randomized, double-blind, placebo-controlled phase III trial with once-weekly, self-administered gefurulimab in adults with anti-acetylcholine receptor antibody-positive, generalized disease.
Dispatch Biotherapeutics Inc. is taking aim at solid tumors with a new viral vector/antigen technology backed by major industry names such as Arch Venture Partners and Bristol Myers Squibb Co. With offices in Philadelphia and San Francisco, Dispatch has raised $216 million since its founding in 2022. The firm’s platform delivers a cell-specific viral vector carrying a novel, universal antigen called Flare that tags solid, epithelial-derived tumor cells. Acting as a beacon, the Flare antigen directs the immune system to find and clear the cancer cells without harming healthy tissue.
