The BioWorld Drug Developers Index (BDDI) continued to track closely with the Nasdaq Biotechnology Index (NBI) and the Dow Jones Industrial Average (DJIA), maintaining its trend upward after a decline at the end of April. BDDI increased by 2.14% through the end of June, following a 1.82% rise through May, after ending April down 4.8% from the start of the year.
Flamingo Therapeutics BV and Ionis Pharmaceuticals Inc.’s FTX-001 (also known as FLM-7523) is a potential first-in-class antisense oligonucleotide (ASO) targeting the human metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) long noncoding RNA (lncRNA); it is being developed for the treatment of cancer.
Researchers from Yale School of Medicine, Ionis Pharmaceuticals Inc. and the National Institutes of Health have published data from a study that aimed to identify the signaling pathways involved in cilia-dependent cyst activation (CDCA) in autosomal dominant polycystic kidney disease (ADPKD).
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.
Alnylam Pharmaceuticals Inc.’s decision to change its analysis plan for the phase III Helios-B trial of RNAi therapeutic Amvuttra (vutrisiran) to treat transthyretin amyloidosis with cardiomyopathy, pushing top-line results back by three months, pressured its share price (NASDAQ:ALNY) down by 10% on Feb. 15, while also boosting shares of competitor Bridgebio Pharma Inc. (NASDAQ:BBIO) by 14%.
Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
Armed with strong phase III safety data in Japanese patients, Palo Alto, Calif.-based Bridgebio Pharma Inc. is planning to file for Japan approval of its investigational drug acoramidis for a rare heart disorder.
Vect-Horus SAS has entered into a license agreement providing Ionis Pharmaceuticals Inc. with a worldwide, exclusive license for a specified number of targets using Vect-Horus’ platform technology Vectrans for systemic delivery of RNA-targeted therapeutics that can cross the blood-brain barrier and address targets in the central nervous system.
Travere Therapeutics Inc. inked a licensing deal with Tokyo-based Renalys Pharma Inc. to develop sparsentan in 13 Asian countries, becoming the latest news to heat up the immunoglobulin A nephropathy (IgAN) therapy space.
Travere Therapeutics Inc. inked a licensing deal with Tokyo-based Renalys Pharma Inc. to develop sparsentan in 13 Asian countries, becoming the latest news to heat up the immunoglobulin A nephropathy (IgAN) therapy space.
