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.
The biosimilars revolution continues with the U.S. FDA’s approval of the first denosumab biosimilars: Wyost (denosumab-bbdz) and Jubbonti (denosumab-bbdz) from Sandoz Inc. for treating osteoporosis and to prevent bone problems in cancer. The approval puts up a strong challenge to Amgen Inc.’s Prolia, the first biologic for osteoporosis, and Xgeva, for bone cancer.
The long-term use of corticosteroids for treating Duchenne muscular dystrophy (DMD) is tied to several undesired effects, compromising the patient’s quality of life; hence, the use of nonsteroidal drugs is highly desirable in the treatment of DMD. Metriopharm AG is investigating the nonsteroidal drug MP-1032 for DMD and recently presented data from studies in a model of DMD.
Pharmaust Ltd.’s monepantel met its primary safety endpoints and showed positive signals of potential efficacy in a phase I trial in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.
Psoriatic arthritis mutilans (PAM) is a rare variant and the most severe form of psoriatic arthritis where patients present erosions of the small joints and osteolysis leading to joint disruption and shortening of one or more digits. To date, no susceptibility genes have been determined.
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