Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26.
Beijing- and Shanghai-based Sperogenix Therapeutics Ltd. said that China’s regulatory agency accepted the NDA filing and granted priority review of Agamree (vamorolone) for Duchenne muscular dystrophy on March 26.
Genescience Pharmaceuticals Co. Ltd. has divulged MAP kinase-activated protein kinase 2 (MAPKAPK2; MK2) inhibitors reported to be useful for the treatment of rheumatoid arthritis and radiographic axial spondyloarthritis (ankylosing spondylitis).
Recent genome-wide association studies identified an association between low bone mineral density (BMD) and a single-nucleotide polymorphism (SNP) at the MALAT1 locus, but there is no functional evidence on the role of MALAT1 alterations in BMD or osteoporosis. Hence, scientists at MD Anderson Cancer Center aimed to assess the functional role of MALAT1 alterations in low BMD and osteoporosis.
The U.S. FDA has approved Duvyzat (givinostat), from Italfarmaco SpA, for treating Duchenne muscular dystrophy (DMD). It is the first oral, nonsteroidal drug for treating all of DMD’s genetic variants. The oral treatment is approved for those ages 6 and older.
Sudo Biosciences Ltd. has divulged non-receptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of scleroderma, radiographic ankylosing spondylitis, type 1 diabetes, idiopathic pulmonary fibrosis, multiple sclerosis, psoriasis, rheumatoid arthritis, Sjögren’s syndrome and systemic lupus erythematosus, among others.
Top-line data from Seelos Therapeutics Inc.’s phase II/III study of SLS-005 in amyotrophic lateral sclerosis (ALS) failed to meet statistical significance in its primary and secondary endpoints, continuing the stock’s nearly half-year downward trajectory.
Mutations in the gene encoding calpain-3, CAPN3, are causative for autosomal recessive limb-girdle muscular dystrophy-1 (LGMDR1), a type of muscular dystrophy characterized by muscle weakening around the shoulders and hips for which there is currently no treatment.
Tweaks made to the design of the phase III trial called Phoenix (vs. the narrowly positive phase II Centaur study) with Amylyx Pharmaceuticals Inc.’s amyotrophic lateral sclerosis (ALS) drug Relyvrio (sodium phenylbutyrate plus taurursodiol) didn’t work. Now, the Cambridge, Mass.-based firm is facing possible withdrawal of the treatment from the U.S. and Canada, where it’s known as Albrioza. Shares of Amylyx (NASDAQ: AMLX) closed March 8 at $3.36, down $15.61, or 82.3%, after the firm disclosed top-line results from Phoenix, a global, 48-week, randomized, placebo-controlled phase III effort with Relyvrio, also known as AMX-0035.
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