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Regeneron's Phase II Candidate Promising in Primary Analysis

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Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced encouraging results from the mid-stage study on pipeline candidate garetosmab (REGN2477) for an ultra-rare genetic bone disorder.

LUMINA-1, a 44-patient, phase II, double-blind, placebo-controlled study is evaluating garetosmab in patients with fibrodysplasia ossificans progressiva (FOP).

FOP is an ultra-rare genetic disorder that leads to abnormal bone formation resulting in skeletal deformities, progressive loss of mobility and premature death.

Garetosmab decreased total lesion activity (both new and existing lesions) compared to placebo by 25%, measured by PET bone scans as observed after 28 weeks of treatment. This result was driven by a nearly 90% decrease compared to placebo in the number of new lesions.

Correspondingly, there was an approximate 25% relative decrease in bone lesion volumes (both new and existing lesions), measured by CT scan, which was also driven by a nearly 90% decrease in the number of new bone lesions as measured by CT. Moreover, patient-reported flare-ups reduced 50%. Investigator-reported adverse events of flare-ups were 10% for garetosmab and 42% for placebo.

LUMINA-1 has a three-period trial design consisting of a randomized, double-blind, placebo-controlled treatment period (6 months), open-label treatment period, during which placebo-treated patients cross over to garetosmab treatment (6 months), and open-label follow-up treatment. The primary analysis was recorded at week 28.

Detailed results from this study will be used as the basis of regulatory submissions. Plans for a pediatric study are also underway. The data will also be submitted for presentation at a future medical congress.

In 2017, the FDA granted Fast Track designation to garetosmab for the prevention of heterotopic ossification in patients with FOP. Garetosmab also enjoys Orphan Designation in the United States and Europe.

Regeneron’s efforts to develop its pipeline are impressive. Its shares have lost 6.2% in the past year compared with the industry’s decline of 2.7%.

 

The company’s lead drug, Eylea, continues to drive growth. Regeneron co-developed Eylea with the HealthCare unit of Bayer AG (BAYRY - Free Report) . Meanwhile, sales of asthma drug, Dupixent, in collaboration with Sanofi (SNY - Free Report) , have been strong as well since the approval and has been boosting the top line.

The successful development and commercialization of other drugs will reduce the company’s dependence on Eylea, which is likely to face stiff competition from Novartis AG’s (NVS - Free Report) Beovu.

Regeneron currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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