The positive results of the second phase experiment were obtained in the treatment of FOP regenerating element by express delivery

On January 9, regeneron announced that its ongoing drug, garetosmab (regn 2477), reduced the formation of new lesions by nearly 90% and the total number of lesions (existing and newly formed) by 25% in the phase 2 trial lumina-1 of patients with progressive muscular ossification (FOP) compared with placebo group Zaharan said it would further discuss with regulators the submission of regulatory applications and prepare to conduct clinical studies for children Another common name of FOP is "Stoneman disease", which means that with the development of the disease, people will be unable to move like stones The main symptom of this rare disease is that the soft and elastic muscles and connective tissue in the human body will become inflamed after slight injury, and will become bones, which will lock the joints that can move freely in one place The locking of elbow joint and knee joint will make the patient's arm and leg fixed at the same angle forever The fusion of jawbone joint will affect their ability to eat and speak, and the ossification of the tissue around the chest and rib will lead to their dyspnea For patients with this rare disease, even with the most delicate protection, ossification will continue to occur, eventually growing a second set of bones in their body Their average life span is just over 50 According to the press release statistics, there are about 800 FOP patients around the world The pathogenic gene of FOP is acvr1, which encodes proteins that regulate bone formation Acvr1 gene mutation was carried in all FOP family patients, but there was no mutation in the healthy family members of FOP patients Garetosmab, a monoclonal antibody developed by regenerator, can be combined with the downstream target of acvr1 protein to prevent abnormal growth of bone Previously, FDA of the United States has granted garetosmab orphan drug qualification, as well as fast track qualification to prevent heterotopic ossification (HO) in FOP patients A total of 44 adult FOP patients participated in a randomized, double-blind, placebo-controlled phase 2 clinical study called lumina-1 The results showed that after 28 weeks of treatment, compared with placebo, the treatment of garetosmab reduced the formation of new lesions by nearly 90% and the total number of lesions by 25% The detailed results of the trial will be presented at a future medical conference ▲ bone specimen of FOP patients (photo source: m ü tter museum official website) "FOP is a devastating disease, which makes many patients have to be wheelchair bound or locked in an immovable position, and the life span of patients is also shortened," said Dr George D yancopoulos, President and chief scientific officer of Zaiban "We believe that garetosmab is expected to bring new hope to the treatment of FOP patients and promote the research and development process of FOP therapeutic drugs We look forward to working closely with FDA and other regulators to bring garetosmab to patients in need as soon as possible " Note: the purpose of this article is to introduce the progress of medical health research, not to recommend the treatment plan For guidance on treatment options, please visit a regular hospital reference material: [1] Regeneron Announces Encouraging Garetosmab Phase 2 Results in Patients with Ultra-Rare Debilitating Bone Disease，Retrieved January 09, 2020, from https://investor.regeneron.com/news-releases/news-release-details/regeneron-announces-encouraging-garetosmab-phase-2-results Original title: rapid delivery for the treatment of "petrochemical" disease, regenerating yuan obtained positive results of phase 2 trial A kind of