Regeneron Halts Garetosmab Clinical Trial Following Multiple Patient Deaths

Compiled by Fan Dongdong

Regeneron Halts LUMINA-1 Trial of Garetosmab (REGN2477) for Rare Bone Disease Following Multiple Patient DeathsRecently, after multiple patient deaths occurred, Regeneron announced the suspension of the LUMINA-1 trial of its rare bone disease drug, garetosmab (REGN2477). The company stated that it is currently reviewing relevant trial data to better understand the drug’s risk-benefit profile. Additionally, Regeneron has informed the independent data monitoring committee associated with the trial and regulatory authorities of the current situation and its decision to suspend the trial.

Regeneron’s decision to suspend the trial was primarily driven by multiple fatal adverse events observed in the open-label extension study. In a statement released on Friday, Regeneron Pharmaceuticals, Inc. reported that several patients receiving Garetosmab died during the open-label extension phase, whereas no deaths occurred among participants in the placebo-controlled arm of the trial.

To date, Regeneron has not specified the number of patient deaths. The company is conducting further investigations into these fatalities to determine whether they are directly related to garetosmab. In January this year, Regeneron announced positive results from the Phase II LUMINA-1 trial of garetosmab for the treatment of fibrodysplasia ossificans progressiva (FOP).

The trial primarily enrolled 44 patients with fibrodysplasia ossificans progressiva (FOP), aged 18 to 60 years, from North America and Europe. In the primary analysis after 28 weeks of treatment, results based on PET bone scans showed that garetosmab reduced overall lesion activity (new and existing lesions) by 25% compared with placebo, and reduced the formation of new lesions by nearly 90%.

In terms of safety, patients in both treatment arms of the trial experienced adverse events; however, all 24 patients in the placebo group and 19 of the 20 patients in the garetosmab group completed the 28-week treatment trial. The majority of patients experienced mild to moderate adverse events, whereas two patients developed more severe abscesses. In contrast, the incidence rate of acute exacerbation-related adverse events was 10% in the garetosmab group, compared with a striking 42% in the placebo group.

Fibrodysplasia ossificans progressiva (FOP) is an extremely rare genetic disorder that causes abnormal bone formation, leading to skeletal deformities, progressive loss of mobility, and premature death. The condition is also commonly known as “stone man syndrome,” meaning that as the disease progresses, patients become immobile like stone. Currently, there are no approved therapies for FOP on the market. Garetosmab is a monoclonal antibody developed using Regeneron’s VelocImmune technology, which holds promise for reducing the formation of heterotopic bone lesions by neutralizing activin A protein.

Despite the suspension of this trial, Dr. George D. Yancopoulos, President and Chief Scientific Officer of Regeneron, remains confident in garetosmab, stating, “We believe that garetosmab may offer important new hope and has the potential to transform the treatment landscape for fibrodysplasia ossificans progressiva (FOP). We look forward to working closely with the FDA and other regulatory authorities to facilitate the approval of garetosmab.”

It is worth noting that Regeneron is not the only company exploring treatments for fibrodysplasia ossificans progressiva (FOP). In August this year, Ipsen announced positive results from a multicenter Phase III trial of palovarotene for the treatment of FOP. The trial results showed that patients treated with palovarotene experienced a 62% reduction in the annual volume of new heterotopic ossification on average. A total of 107 participants were enrolled in the MOVE trial, and the incidence of adverse drug reactions was categorized as mild (32.2%), moderate (45.5%), and severe (22.2%).

References:

1.Regeneron Hits Pause on LUMINA-1 Trial Due to Concerns with Garetosmab

2.Regeneron slams the brakes on rare bone disease trial after patient deaths