Why This PNH Study Could Set the Standard for Treatment

Health and Healthcare

Published: January 7, 2020 9:05 am

Apellis Pharmaceuticals Inc. (NASDAQ: APLS) shares jumped on Tuesday after the firm announced positive results from its late-stage study in adults with paroxysmal nocturnal hemoglobinuria (PNH). Specifically, the data came from the Phase 3 Pegasus study evaluating pegcetacoplan (APL-2).

Ultimately, the top-line data showed that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a statistically significant improvement of hemoglobin at week 16.

Additionally, pegcetacoplan showed promising results in key secondary endpoints. Pegcetacoplan met non-inferiority on transfusion avoidance and absolute reticulocyte count. Pegcetacoplan also showed positive trends on lactate dehydrogenase and fatigue as measured by the Functional Assessment of Chronic Illness Therapy fatigue score.

In this study, the safety profile of pegcetacoplan was comparable to eculizumab. Seven of 41 patients (17.1%) in the pegcetacoplan group experienced a serious adverse event, and six of 39 patients (15.4%) in the eculizumab group experienced serious adverse events. No cases of meningitis and no deaths were reported in either treatment group.

Peter Hillmen, M.B., Ch.B., Ph.D., Professor of Experimental Hematology at the University of Leeds and an investigator in the Pegasus study, commented:

The majority of patients with PNH currently receiving treatment with eculizumab have continuing anemia. The Pegasus results show that pegcetacoplan has the potential to become a new standard of care for patients with PNH.

Shares of Apellis closed Monday at $30.19, in a 52-week range of $12.50 to $33.18. The consensus price target is $41.56. Following the announcement, the stock was up about 50% at $45.05, in early trading indications Tuesday.

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