The fast-shifting obesity space gained more clinical results as Viking Therapeutics Inc. shared data from its phase I, multiple ascending-dose trial with oral VK-2735, a dual agonist of the glucagon-like peptide 1 (GLP-1) and glucose-dependent insulinotropic polypeptide (GIP) receptors.
Updated results from Stoke Therapeutics Inc.’s studies of antisense oligonucleotide STK-001 showed a reduced convulsion seizure frequency in those ages 2 to 18 with Dravet syndrome. The new data, which also showed improved cognition and behavior in participants, substantially boosted the company’s stock value and caused analysts to applaud.
Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment, PRAX-628, resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%.
China’s National Medical Products Administration has cleared China Grand Pharmaceutical and Healthcare Holdings Ltd. to advance radiopharmaceutical agent ITM-11 (177Lu-edotreotide) to phase III trials in gastroenteropancreatic-neuroendocrine tumors
An asset that some investors may have overlooked came through for Axsome Therapeutics Inc., which rolled out phase III data from the trial called Symphony with AXS-12 (reboxetine), which hit the primary endpoint by significantly reducing cataplexy attacks in narcolepsy patients vs. placebo.
Benevolentai Ltd. is betting on its oral phosphodiesterase 10 inhibitor BEN-8744 as having first-in-class potential for moderate to severe ulcerative colitis, after healthy volunteers in a phase Ia trial remained free of central nervous system-associated side effects.
Neuromuscular disease specialist NMD Pharma A/S has been given U.S. FDA approval for a phase IIb trial of NMD-670, after demonstrating proof of mechanism for the orally-available chloride ion channel-1 inhibitor in generalized myasthenia gravis.
China’s National Medical Products Administration has cleared China Grand Pharmaceutical and Healthcare Holdings Ltd. to advance radiopharmaceutical agent ITM-11 (177Lu-edotreotide) to phase III trials in gastroenteropancreatic-neuroendocrine tumors
Eledon Pharmaceuticals Inc.’s tegoprubart, an investigational anti-CD40 ligand antibody, was used as part of the immunosuppressive regimen after the first-ever transplant of a kidney from a genetically modified pig to a human. The tegoprubart procedure was done March 16 at Massachusetts General Hospital on a 62-year-old man with end-stage renal disease.
The Hodgkin lymphoma space found itself in the spotlight late last year when Affimed NV rolled out updated data with lead innate cell engager acimtamig when combined with allogeneic natural killer cells. Findings from the investigator-initiated trial became the topic of talk at the American Society of Hematology meeting, when Yago Nieto, professor at the University of Texas MD Anderson Cancer Center – and principal investigator of the study – spoke.
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