The higher of two doses of Ionis Pharmaceuticals Inc.’s drug olezarsen for treating adults with familial chylomicronemia syndrome reduced fasting triglyceride levels by 44% from baseline at the six-month mark in a phase III study, but one case of pancreatitis in the trial paints a less attractive picture than top-line data the firm reported in September.
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.
Bolden Therapeutics Inc. has closed a $1.5 million pre-seed convertible note financing. This financing, together with National Institutes of Health (NIH) small business grants, will support preclinical development of Bolden's antisense oligonucleotides to promote neurogenesis.