New interim data from Ultragenyx Pharmaceutical Inc. and Genetx Biotherapeutics LLC’s phase I/II study of GTX-102 in Angelman’s syndrome didn’t do much to bolster investor confidence, as Ultragenyx shares sagged considerably July 19. The Novato, Calif.-based company’s stock (NADAQ:RARE) closed 13% downward at $52.89 per share. That is nearly half the price shares fetched in late August. At $102.40 per share, that was the stock’s highest value in the past 12 months.
Astrazeneca plc and Ionis Pharmaceuticals Inc. have unveiled some eye-catching figures from their cholesterol drug, AZD-8233, in a phase IIb trial, taking aim at the PCSK9 inhibitor market where Novartis AG, Sanofi SA/Regeneron Inc. and Amgen Inc. are vying for supremacy.
Secarna Pharmaceuticals GmbH & Co. KG and Denali Therapeutics Inc. have expanded a central nervous system research partnership as the gene silencing field heats up.
Times have changed at Ionis Pharmaceuticals Inc. since it spun out Akcea Therapeutics Inc. in 2015. The company’s evolution is such that Ionis is now ready to bring Akcea back into the fold by acquiring the remaining 24% of its affiliate.