Reduce the number of new lesions by 90%! Regenerative meta "stone man disease" new drug Phase 2 clinical success.

Recently, Regenerative Meta announced the results of the trial of the new osteopathic drug garetosmab (REGN2477) in the treatment of fibrodysplasia Ossificans Progressiva,FOP Phase 2 clinical LUMINA-1. Preliminary analysis after 28 weeks of
treatment showed that garetosmab reduced the total number of lesions (new and existing lesions) by 25% compared to placebos (measured by PET bone scans) (p s 0.07), reducing the number of new lesions by nearly 90%.
LUMINA-1 was a randomized, double-blind, placebo-controlled study that recruited 44 FOP patients (18-60 years of age) with varying severity of the disease and was randomly treated with garetosmab or placebo to assess the efficacy of garetosmab in FOP patients.
the study will form the basis for submitting an FDA regulatory application, and programs for children are underway, the agency said. Dr George Yancopoulos, President and Chief Scientific Officer of regeneron,
, said: "FOP is a very cruel and devastating disease that has left many patients wheelchair-dependent or inactive and life expectancy significantly shorter.
we believe that garetosmab will bring new hope to FOP patients and hopefully change the treatment process of FOP.
looking forward to working closely with the FDA and other regulators to achieve the early launch of Garetosmab.
" another commonly known term for muscular osteopathy is "stone man's disease", which means that as the disease progresses, the patient becomes inactive like a stone, a very rare genetic disease.
about 800 people worldwide are currently diagnosed with FOP, but many others have not been diagnosed or misdiagnosed, the company said in a press release.
decades, FOP has been a problem for patients and researchers, and there is no cure, and scientists can't even figure out how the disease occurs.
years of effort, researchers discovered in 2006 that the main cause of FOP was a mutation in the gene (ACVR1) on the fourth pair of chromosome long arms.
acVR1-coded protein regulates bone production, and all FOP family patients carry genetic mutations on the ACVR1 gene, and healthy family members of FOP patients do not have mutations in this gene.
found the cause that inspired the development of FOP therapy.
currently, FOP has three companies in the clinical development phase of research therapy, in addition to regenerative elements, there are Blueprint Medicine and Clementia, and the three therapeutic mechanisms are different.
-recycled metagaretosmab can be combined with downstream targets of proteins encoded in the ACVR1 gene to prevent abnormal bone growth; BLUEPRINT Medicine's BLU-782, which directly targets ACVR1 mutants to inhibit abnormal activity of proteins; and Clementia's therapy, which is the fastest-growing, has completed phase 3 clinical patient registration in August and is expected to be trial-approved by 2020.
sources: 1, Regeneron's bone disease drug delivers in phase 2, poised for FDA filing 2, REGENERON ANNOUNCES BINDING GARETOSMAB PHASE 2 RESULTS IN PATIENTS WITH-ULTRA RARE DEBILITATING BONE DISEASE.