The EMA is standing firm on its refusal to recommend approval of the amyotrophic lateral sclerosis (ALS) treatment Albrioza in Europe after re-examining Amylyx Pharmaceuticals Inc.’s marketing authorization application and remaining unconvinced that the main study demonstrated the drug effectively slows disease progression.
It has been previously demonstrated that therapeutic inhibition of the prostaglandin E2 (PGE2)-degrading enzyme, 15-prostaglandin dehydrogenase (15-PGDH), was able to improve muscle strength in aged mice. Researchers from Epirium Bio Inc. have now reported the discovery and preclinical characterization an orally bioavailable small molecule inhibitor of 15-PGDH – MF-300 – being developed for the treatment of neuromuscular dysfunction.
Researchers from Immunophage Biomedical Co. Ltd. have published details on the discovery and preclinical evaluation of a new CD38 inhibitor being developed for the treatment of mitochondrial myopathy.
Researchers from East China Normal University and Shanghai Jiao Tong University presented the discovery and preclinical evaluation of new potent antiosteoporosis agents. Synthesis and optimization of a series of heterocyclic ring-fused derivatives of 20(S)-protopanaxadiol (PPD) led to the identification of SH-491 as the lead candidate with the most potent inhibitory effects on RANKL-induced osteoclastogenesis (IC50=11.8 nM).
Brainstorm Cell Therapeutics Inc. said it’s exploring all its options in the wake of a Sept. 27 U.S. FDA advisory committee vote, in which the committee overwhelmingly disagreed with the company that the data it presented supported the effectiveness of Nurown (debamestrocel) for the treatment of mild to moderate amyotrophic lateral sclerosis.
Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
The autosomal dominant form of osteopetrosis, referred to as autosomal dominant osteopetrosis type 2 (ADO2), is caused by single allele dominant negative mutations of the CLCN7 gene. In a recent paper, researchers from Sisaf Ltd. detailed the development and preclinical evaluation of novel silicon stabilized hybrid lipid nanoparticles (sshLNPs), SIS-101-ADO, designed to deliver small interfering RNA (siRNA) specific against the human CLCN7 G215R mRNA.
How flexible should the U.S. FDA evidentiary standards be for a therapy addressing a significant unmet need in a disease such as amyotrophic lateral sclerosis (ALS)? That’s the question the agency’s Cellular, Tissue and Gene Therapies Advisory Committee will ponder Sept. 27 as it looks at the data for Brainstorm Cell Therapuetics Inc.’s Nurown (debamestrocel), a mesenchymal stromal cell therapy targeting ALS. Nurown is going into the adcom with a bit of a checkered history that includes a refuse-to-file letter and a single phase III trial that failed to demonstrate efficacy for the primary endpoint and all key secondary efficacy endpoints, according to the FDA briefing document.
Researchers from the University of Florida have published preclinical data for the novel indoleamine 2,3-dioxygenase (IDO) and galectin-3 (Gal3) fusion protein (IDO-Gal3), being evaluated for the treatment of osteoarthritis (OA).
Long bones, vertebrae and skull bones have distinct types of stem cells, and new insights into those stem cells could lead to new ways to treat both rare developmental disorders of skull formation and the all-too-common phenomenon of bone metastases. Scientifically, the work, which was published in two papers by Matthew Greenblatt and colleagues in Nature, adds to the increasing understanding of bone’s complexities. “Bone may serve as an endocrine organ that is secreting factors throughout the body,” Greenblatt said.
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