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Regeneron Reports Positive Data on Rare Blood Disorder Drug

Regeneron Pharmaceuticals, Inc. REGN announced top-line data from a mid-stage study on pipeline candidate pozelimab (REGN3918) for the treatment of rare blood disorder — paroxysmal nocturnal hemoglobinuria (PNH).

Pozelimab, an investigational, fully-human monoclonal antibody, is designed to block complement factor C5 and prevent the destruction of red blood cells (hemolysis) that cause the symptoms of PNH and other diseases mediated by complement pathway activity.

The results showed that pozelimab reduced the abnormal destruction of red blood cells (known as "hemolysis), with patients in the initial cohort achieving normal levels of a blood biomarker of elevated hemolysis called lactate dehydrogenase (LDH). Patients in the trial initially received a 30-mg/kg IV loading dose of pozelimab, followed by weekly 800-mg subcutaneous injections. All 6 patients in the initial treatment cohort treated with pozelimab experienced rapid and sustained reductions in LDH up to week 8.

No adverse events were serious or led to the discontinuation of the study.

The ongoing open-label, single-arm, two-part trial will enroll patients with active symptomatic PNH, who are naïve to complement inhibitors or who have not received treatment with a complement inhibitor within 6 months prior to entering the trial. It consists of two cohorts — cohort A (n=6), which is complete and has achieved its objective of dose confirmation, and cohort B (n=approx. 30), which is ongoing and will focus on further evaluating efficacy and safety in a larger PNH population.

Plans for a phase III program are underway as well.

Regeneron’s shares have lost 1.4% in the year so far against the industry’s  growth of 6.2%.


The company’s lead drug, Eylea, continues to drive growth. Regeneron co-developed Eylea with the HealthCare unit of Bayer AG BAYRY. Meanwhile, sales of asthma drug Dupixent have been strong as well since approval. Regeneron has a collaboration agreement with Sanofi SNY for this drug.

We are impressed by Regeneron’s efforts to develop its pipeline. The successful development and commercialization of other drugs will reduce its dependence on Eylea, which is likely to face stiff competition from Novartis AG’s NVS Beovu.

Regeneron currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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