An interim analysis from Fulcrum Therapeutics Inc.’s phase II study of losmapimod for treating facioscapulohumeral muscular dystrophy (FSHD) produced data that pleased the CEO and displeased investors.
Shares of Sweden-based Hansa Biopharma AB (NASDAQ:HNSA) jumped 32.7% to SEK53.90 (US$5.81) on July 2 as Sarepta Therapeutics Inc. became the first partner to license its lead asset, imlifidase, to enable gene therapies to treat muscular dystrophy patients harboring neutralizing antibodies to the adeno-associated virus (AAV) vectors the medicines employ.
Shares of Entera Bio Ltd. (NASDAQ:ENTX), a lightly traded Israeli drug delivery specialist, fell 27% on May 21 after the company said an ongoing phase II trial of its oral parathyroid hormone (PTH) candidate in osteoporosis patients found the two lowest doses tested demonstrated "suboptimal increases" in P1NP, an important biomarker of bone formation.
HONG KONG – South Korea’s Daewon Pharmaceutical Co. Ltd. has launched Terrosa, a teriparatide biosimilar for the treatment of osteoporosis, acquired from Hungarian firm Richter-Helm Biotec GmbH & Co KG. It’s the first biosimilar launch for the company.
Though Quralis Corp., of Cambridge, Mass., has been developing its pipeline for more than three years, the company now has a $42 million series A in hand to continue researching and developing therapies for amyotrophic lateral sclerosis and frontotemporal dementia.
DUBLIN – Dyno Therapeutics Inc., an early stage gene therapy firm applying artificial intelligence to advanced capsid engineering, has entered partnerships with Novartis AG and Sarepta Therapeutics Inc., in ophthalmic indications and muscle diseases, respectively, which have over $2 billion in biobucks attached.
Weeks after raising an oversubscribed $60 million series A, Affinia Therapeutics Inc., of Waltham, Mass., is collaborating with Vertex Pharmaceuticals Inc. in a far larger deal, one potentially worth more than $1.6 billion.
The allure of gene therapy was proved yet again as Waltham, Mass.-based Affinia Therapeutics Inc. bagged an oversubscribed $60 million series A financing to boost the push for drugs to benefit people affected by muscle and central nervous system conditions.
Design Therapeutics Inc., a San Diego startup developing new therapies for degenerative disorders caused by nucleotide repeat expansions, has raised $45 million in series A financing.
With a phase IIb readout coming in the third quarter of 2020, Cambridge, Mass.-based Fulcrum Therapeutics Inc. might be set up for a win in facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder for which there’s no treatment.
RSS
