A disease-modifying gene therapy for Sanfilippo syndrome type A has demonstrated reductions in heparin sulfate within cerebrospinal fluid, as well as increases in the cognitive function of young patients, arming Ultragenyx Pharmaceutical Inc. with data needed to support an accelerated BLA with the U.S. FDA.
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.
Mereo Biopharma plc is heading into phase III with a bone-building drug to treat the rare condition osteogenesis imperfecta – and CEO Denise Scots-Knight reckons the company has the financial backing to get setrusumab marketed thanks to a partnership with Ultragenyx Pharmaceutical Inc.
Immunogen Inc. leads a busy earnings season with numbers that pushed the stock (NASDAQ:IMGN) 30.3% higher on Feb. 12. The antibody-drug conjugates developer posted revenues of $132.3 million for 2020 compared to its $82.3 million in revenues for 2019, a 62.2% increase.
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Solid Biosciences Inc. already has its own Duchenne muscular dystrophy program, but with some new investment money it is plunging into a collaboration with Ultragenyx Pharmaceutical Inc. to co-create another program. The two will collaborate on developing and commercializing new gene therapies for treating Duchenne muscular dystrophy as Novato, Calif.-based Ultragenyx is investing $40 million in Solid.
Second-quarter earnings reports, flowing fast in the last days of July, delivered a bevy of contrasting results, from A to V. Stymied by the pandemic, Aimmune Therapeutics Inc. booked exactly zero net sales during the quarter for its expected blockbuster peanut allergy treatment, Palforzia. Vertex Pharmaceuticals Inc. and Seattle Genetics Inc. beat top-line expectations, driven primarily by strong sales of new products.
HONG KONG – Daiichi Sankyo Co. Ltd., Mitsubishi UFJ Capital Co. Ltd. and Nagoya Institute of Technology have kicked off research on a joint project for a gene therapy that could be applied to the restoration of vision.
Ultragenyx Pharmaceutical Inc.’s top-line win in January with DTX-301 gene therapy in ornithine transcarbamylase (OTC) deficiency seemed to presage even better things to come later this year, and analysts more recently hailed fourth-quarter earnings that showed satisfying progress with Crysvita (burosumab).
The prospects for Ultragenyx Pharmaceutical Inc.'s adeno-associated virus (AAV) gene therapy, DTX-301, in patients with ornithine transcarbamylase (OTC) deficiency weren't looking too great after treatment of the first two cohorts in a phase I/II study.
