Apellis Pharma Flunks On Primary, Secondary Endpoints In Amyotrophic Lateral Sclerosis Study

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  • Apellis Pharmaceuticals Inc APLS announced that the Phase 2 MERIDIAN study investigating systemic pegcetacoplan for amyotrophic lateral sclerosis (ALS) did not meet its primary endpoint of the Combined Assessment of Function and Survival (CAFS) rank score at Week 52
  • CAFS ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score.
  • The study also did not meet key secondary efficacy endpoints. Systemic pegcetacoplan was well tolerated in the study, and the data were consistent with the established safety profile.
  • Based on the lack of efficacy, Apellis and Swedish Orphan Biovitrum AB BIOVF plan to discontinue the development of systemic pegcetacoplan for ALS. 
  • In April, Apellis and Sobi discontinued treatment in the open-label portion of the study following a recommendation from an independent data monitoring committee.
  • In April, a Bloomberg report noted that large drugmakers were interested in Apellis Pharmaceuticals.
  • The report added that Apellis might seek partnerships or licensing agreements for some of its ophthalmology products. Deliberations are ongoing, and there's no certainty they will lead to a transaction.
  • Price Action: APLS shares are down 2.05% at $84.79 on the last check Thursday.
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