With no drugs approved by the U.S. FDA for treating Becker muscular dystrophy, Edgewise Therapeutics Inc. reported positive two-year, phase Ib data looking at patients’ ability to physically function, plus biomarker data.
BLR Bio LLC announced that the U.S. FDA has granted orphan drug designation to BLR-200, the company’s investigational therapy for the treatment of systemic sclerosis (SSc).
Enlivex Therapeutics Ltd. extolled the top-line “positive indication of effect and safety” from the phase II study of Allocetra in treating sepsis and sepsis shock, but the market took another view of the clinical trial. In an analysis of eligible adult patients from the cell therapy’s multicenter, randomized, placebo-controlled, dose-finding study of 120 enrolled patients, the company also reported low mortality rates.
Scientists at Ez Bioxcel Solutions Pvt Ltd., Invea Therapeutics Inc. and Inveniai LLC have identified apoptosis-associated speck-like protein containing a CARD (PYCARD; ASC) inhibitors reported to be useful for the treatment of primary sclerosing cholangitis, arthritis and inflammatory disorders.
Biosimilar competition to Amgen Inc.’s denosumab (Prolia/Xgeva) is rising globally, with Mabwell (Shanghai) Bioscience Co. Ltd. gaining the latest China NMPA approval of Maiweijian (TK-006) on April 8. Mabwell’s wholly owned subsidiary, Jiangsu T-mab Biopharma Co. Ltd., gained NMPA clearance of Maiweijian (120 mg) as the first denosumab biosimilar for the indications of U.S.-licensed Xgeva for bone-related diseases.
Researchers from Catholic University of Korea published data from a study that investigated the effect of miRNA-21a-5p on fibrosis development in systemic sclerosis (SSc). With the aim of assessing the pathological impact of miRNA-21a-5p on skin and lung fibrosis in vivo, a bleomycin-induced SSc murine model was developed, and the mice were hydrodynamically injected with plasmids containing pre-miRNA-21a-5p or anti-miRNA-21a-5p.
Less than a month after disclosing that its confirmatory phase III trial of Relyvrio (sodium phenylbutyrate plus taurursodiol) fell short of its endpoint, Amylyx Pharmaceuticals Inc. is withdrawing the amyotrophic lateral sclerosis (ALS) drug from the market.
About 90% of patients with multiple myeloma (MM) develop severe bone disease, known as myeloma bone disease (MBD). This occurs due to the ability of MM cells to disrupt bone homeostasis, leading to excessive bone resorption. Some current treatments are effective for treating MBD, but they are associated with undesired adverse events.
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.
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