Extremely rare bone disease! Regenerating element garetosmab in the treatment of progressive ossifying fibrous dysplasia (FOP) phase II clinical success!

January 12, 2020 / BIOON / -- regeneron recently announced the positive results of phase II lumina-1 study on the treatment of progressive ossifying fibrodysplasia (FOP) with garetosmab (regn 2477) Based on the complete results of the lumina-1 study, the company will submit an application for listing to the regulatory authority Garetosmab has the potential to change the process of FOP and bring important new hope to patients FOP is a very rare genetic disease, which can lead to abnormal bone formation, bone deformity, progressive disability and premature death At present, there is no drug approved to treat fop FOP is a merciless and devastating disease, many patients are bound by wheelchair or locked in a certain position can not move, life span is greatly shortened Lumina-1 is a double-blind, placebo-controlled study involving 44 adult patients (18-60 years old) from North America and Europe who were clinically diagnosed with FOP and recorded mutations in the acvr1 gene In the study, patients were randomly assigned to receive garetosmab or placebo In the main analysis after 28 weeks of treatment, using PET bone scan measurement, compared with placebo, garetosmab reduced total lesion activity (new and existing lesions) by 25%, driven by a nearly 90% reduction in new lesions compared with placebo Accordingly, with CT scan, the volume of osteopathy (including new and existing lesions) was reduced by about 25% (P = 0.37), which was also driven by the reduction of new bone lesions by nearly 90% Patients reported a 50% reduction in flare up (nominal P = 0.03) The incidence of acute adverse events reported by investigators was 10% in the garetosmab group and 42% in the placebo group In FOP patients, abnormal bone formation occurs in soft tissue outside normal bone, muscle, tendon and ligament are gradually replaced by bone, this process is called heterotopic ossification (HO) Around the world, about 800 patients have been diagnosed with FOP, but many have not been diagnosed or misdiagnosed Zaihuan has been engaged in FOP research for more than 20 years and has helped to provide significant insights into the biology and natural history of the disease The scientists of regenerator found that activin A plays a key role in FOP by driving Ho, which is the main pathology of FOP Galetosmab was developed by veloclmune technology of regenerator, which is a monoclonal antibody It can neutralize activin a protein to reduce the formation of heterotopic bone lesions In 2017, FDA of the United States has granted garetosmab fast track qualification to prevent ho in FOP patients In the United States and the European Union, garetosmab has been granted orphan drug status At present, the research on the treatment of FOP adult patients with garetosmab is in progress Frederick S Kaplan, MD, Issac & Rose Nassau professor of orthopaedic molecular medicine, Perelman School of medicine, University of Pennsylvania, who is the world's leading investigator of lumina-1 research, said: "prevention of new ectopic bone is essential for the treatment of FOP The preliminary results of the lumina-1 clinical trial are encouraging because they clearly show a significant reduction in the formation of new lesions, which is important when we consider future studies in the pediatric population " "Now we have the first promising results of placebo-controlled trials, showing that the incidence of new ectopic bone formation is reduced and the incidence rate of new HO has decreased by nearly 90%, which is a pioneering result for FOP patients," said LUMINA-1 investigator, Marelise Eekhoff medical director of the Amsterdam FOP center, University of Amsterdam medical center This trial has also significantly improved our understanding of the disease, demonstrating that the frequency and extent of new bone lesions in untreated FOP patients is much higher than previously thought, and that the formation of these new bone lesions seems to depend on activin A " Original source: regeneron announcements encouraginggaretosmab phase 2 results in patients with ultra rate identifying bone disease