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.
Lysophosphatidylcholine acyltransferase 3 (LPCAT3), highly expressed in the liver, intestine, and adipose tissues, is an enzyme that preferentially incorporates polyunsaturated fatty acyl chain into lysophospholipids. Previous work showed that LPCAT3 and phospholipid remodeling play a crucial role in regulating glucose metabolism and contribute to the development of insulin resistance in type 2 diabetes.
Vanda Pharmaceuticals Inc. and Olipass Corp. have entered into a research and development collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on Olipass' proprietary modified peptide nucleic acids.
Philadelphia-based Aro Biotherapeutics Co. CEO Sue Dillon told BioWorld her firm has “incoming interest from other companies to apply Centyrins to other kinds of drug conjugates,” which could mean more deals with the platform like the one sealed with Ionis Pharmaceuticals Inc.
It’s a big day for privately held Empirico Inc. as it embarks on a massive combo pharma deal with Ionis Pharmaceuticals Inc. while racking up a $17 million series A-2 financing.