Regenerative meta "stone man disease" new drug Phase 2 clinical success

, regenerative meta announced the results of a trial of clinical LULINA-1 phase 2 in the treatment of aggressive musculoskeletalization (Fibrodysplasia Ossificans Progressiva, FOP) in the new drug garetosmab. Preliminary analysis after 28 weeks of treatment showed that garetosmab reduced the total number of lesions (new and existing lesions) by 25% (measured by PET bone scan) (p -0.07) compared to placebo, reducing the number of new lesions in patients by nearly 90%.L
UMINA-1 was a randomized, double-blind, placebo-controlled study that recruited 44 FOP patients (18-60 years old) with varying severity of the disease to receive garetosmab or a placebo treatment at random to assess the efficacy of garetosmab in FOP patients.Regeneration said the detailed results of the study will serve as a basis for submitting FDA regulatory applications, and plans for child patients are under way.Dr George Yancopoulos, President and Chief Scientific Officer of Regeneron, said: "FOP is a cruel and devastating disease that has left many patients without even being able to travel in wheelchairs and their lifespans significantly reduced. We believe that garetosmab will bring new hope to FOP patients and hopefully change the process of FOP treatment. We look forward to working closely with the FDA and other regulators to get Garetosmab on the market as soon as possible., another commonly known as "stone man's disease," means that as the disease progresses, the patient becomes insulable like a stone, a very rare genetic disorder. About 800 people worldwide are currently diagnosed with FOP, but many remain undiagnosed or misdiagnosed, Regeneration said in a press release.For 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. After years of effort, researchers discovered in 2006 that the main cause of FOP was a mutation in the gene (ACVR1) on the long arm of the fourth pair of chromosomes. ACVR1-coded proteins regulate bone production, and all ACVR1 genes in FOP family patients carry gene mutations, and healthy family members in FOP patients do not have mutations in this gene.The finding of the cause inspired the development of FOP therapy. Currently, FOP has three companies in the clinical development phase of research therapy, in addition to regenerative elements, as well as Blueprint Medicine and Clementia, and the three therapies have different mechanisms of action. The regenerative metagaretosmab is able to be combined with downstream targets of the ACVR1 gene-coded protein to prevent abnormal bone growth; blueprint Medicine has developed a BLU-782 that directly targets mutations in ACVR1 to inhibit abnormal protein activity; and Clementia's therapy is the fastest progressing, completing Phase 3 clinical patient registration in August this year and is expected to be test results and approved for market approval in 2020. (Sina Pharmaceutical News)