Regeneron releases positive results from Phase 3 Trial in Generalized Myasthenia Gravis

Regeneron Pharmaceuticals have recently announced that the primary and key secondary endpoints were met in the Phase 3 NIMBLE trial assessing investigational cemdisiran monotherapy in adults with generalized myasthenia gravis. Cemdisiran is an siRNA that reduces circulating levels of complement factor 5 and, as monotherapy in this trial, was associated with an average of 74% inhibition of complement activity. Note that the trial also assessed a combination of cemdisiran and pozelimab, a C5 antibody; this combination, which resulted in nearly 99% inhibition of complement activity, also met the primary and key secondary endpoints, though cemdisiran monotherapy was numerically better across these endpoints.

“Our pipeline approach to treating complement-mediated diseases allows us to tailor treatment to the underlying disease biology,” said L. Andres Sirulnik, M.D., Ph.D., Senior Vice President and Hematology Clinical Development Unit Head at Regeneron. “The results of the NIMBLE trial confirm that, in myasthenia gravis, robust efficacy can be achieved without complete complement blockade, whereas in other diseases such as paroxysmal nocturnal hemoglobinuria, complete inhibition is likely to be necessary. We have previously released data from the lead-in portion of our PNH Phase 3 trial, supporting the potential for the cemdi-poze combination to deliver best-in-class efficacy in PNH. We are also investigating systemic administration of both cemdisiran monotherapy and the cemdi-poze combination in our Phase 3 program for geographic atrophy secondary to age-related macular degeneration.”

“The NIMBLE trial results underscore the potential for cemdisiran to offer a best-in-class profile for those suffering with myasthenia gravis, providing for robust efficacy with a convenient quarterly subcutaneous administration,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron. “The potential for best-in-class efficacy with less than complete complement blockade with cemdisiran monotherapy may also provide for a more favorable safety profile. These exciting results highlight the transformative potential of our siRNA and genetic medicines pipeline to deliver paradigm-changing therapies for patients.”

 

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Related Topics and Keywords

batura phase 3 trial, generalized myasthenia gravis, Regeneron Pharmaceuticals