Apellis expects to restart enrollment in DERBY, OAKS trials in 2Q19

Apellis provided an update on the status of its Phase 3 program for APL-2 in patients with Geographic Atrophy, or GA. Apellis expects to be able to restart enrollment of its two Phase 3 GA trials in Q2 2019 and to have fully enrolled both studies by the end of Q1 2020, within the originally planned timeline for completion. As previously disclosed, the company voluntarily implemented a temporary pause in dosing in the DERBY and OAKS Phase 3 trials due to observed cases of non-infectious inflammation in patients treated from a single manufacturing lot of APL-2 intravitreal drug product. The company believes the source of inflammation in the Phase 3 trials resides in an impurity or contaminant in the active pharmaceutical ingredient, or API. Inflammation in all affected patients has resolved. In the Phase 2 FILLY trial, where more than 1,500 intravitreal injections of APL-2 were given, APL-2 was well-tolerated with only a single case of non-infectious inflammation reported. The source of inflammation has had no known impact on the studies utilizing systemic administration of APL-2. Based on Apellis’ investigation, which included a full manufacturing process review, six non-clinical studies in two species and an ongoing Phase 1b clinical study in patients with GA with low vision, the company does not believe that the following were the root causes for the observed inflammation in the Phase 3 GA program: the pharmacology or chemical composition of APL-2, a formulation change that was implemented between the Phase 2 FILLY trial and the Phase 3 trials, and the fill and finish process for the APL-2 intravitreal drug product. In order to resume the Phase 3 GA program in a safe and expeditious manner, the company has introduced improvements to the manufacturing process in order to eliminate impurities and potential contaminants like those that are believed to have caused inflammation in the Phase 3 trials. A new lot of API incorporating these improvements has been manufactured and is ready for release in a quantity sufficient to complete both the DERBY and OAKS Phase 3 clinical trials. The company believes that the improved manufacturing process can be used to supply API at a scale required for global commercialization. In non-clinical testing, treatment with API manufactured through the improved manufacturing process did not cause inflammation. Finally, prior to introduction in the Phase 3 clinical trials, new lots of API may first be introduced in a small Phase 1b study of patients with GA with low vision.

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