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No inflammation observed in ten patients dosed in Phase 1b trial with new intravitreal APL-2 drug product from modified manufacturing process
CRESTWOOD, Ky. and WALTHAM, Mass., March 04, 2019 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals Inc. (Nasdaq:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds to treat disease through the inhibition of the complement system, today announced that with the agreement of the independent safety monitoring committee for the Company’s Phase 3 clinical program for APL-2 in patients with Geographic Atrophy (GA), the Company has resumed enrollment in its two Phase 3 GA trials (DERBY & OAKS) with intravitreal APL-2. Apellis continues to expect to have both trials fully enrolled by the end of the first quarter of 2020.
“Patient safety is our first priority and we feel that we can now reinitiate our Phase 3 program for patients with geographic atrophy with confidence following an in-depth investigation and modifications to the manufacturing process,” said Cedric Francois, CEO and co-founder of Apellis. “We remain on track for full enrollment by the end of the first quarter of 2020, as originally guided. We believe that APL-2 can offer significant benefits to patients with geographic atrophy and look forward to continuing our pivotal trials for this important potential first approved treatment for GA.”
In October 2018, Apellis communicated that the Company had voluntarily implemented a 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 investigational material. Inflammation in all affected patients resolved.
Based on its investigation, the Company believes that the likely source of inflammation resided in an impurity in the active pharmaceutical ingredient (API) that was introduced during the scale-up of the manufacturing process to produce commercial lot sizes. Apellis has modified its manufacturing process in order to eliminate the impurity and has manufactured sufficient supply of APL-2 utilizing the modified manufacturing process to conduct the entire Phase 3 GA program.
APL-2 intravitreal drug product produced from the modified manufacturing process was introduced into the Company’s ongoing Phase 1b trial in low vision patients with GA. Ten patients in the trial have received at least one intravitreal injection of APL-2 manufactured through the modified process and there has been no inflammation observed in any patient injected with APL-2 from this new manufacturing lot. Patients in the trial will continue to receive monthly APL-2 injections for two years.
The independent safety monitoring committee reviewed all non-clinical data and clinical data from the Phase 1b trial and agreed that Apellis could resume dosing in the Phase 3 program using APL-2 manufactured with the modified process.
“Geographic atrophy is a major cause of visual impairment across our society, profoundly and negatively impacting routine activities like driving and reading, leading to loss of independence and inability to perform every-day life activities,” said Charles Wykoff, MD, PhD (Director of Research, Retina Consultants of Houston and Investigator of Apellis Phase 1b, 2 and 3 trials). “It is incredibly frustrating to patients and physicians that there are no approved treatments for this common, blinding disease. It is an important and exciting step forward to be reinitiating these Phase 3 trials as it brings much needed hope to the GA community.”
About the DERBY and OAKS trials
The Derby and OAKS trials are 600-patient prospective, international, multicenter, randomized, double-masked, sham-injection controlled Phase 3 studies assessing the efficacy and safety of multiple intravitreal (IVT) injections of APL-2 in patients with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD). For more information, please visit https://gastudy.com/.
About Geographic Atrophy (GA)
GA is an advanced form of age-related macular degeneration (AMD), a disorder of the central portion of the retina, known as the macula, which is responsible for central vision and color perception. GA is a chronic, progressive condition that leads to central blind spots and permanent loss of vision. Based on published studies, the company estimates that approximately one million people have GA in the United States alone. There are currently no FDA-approved treatments for GA.
APL-2 is designed to inhibit the complement cascade centrally at C3 and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with partial inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative). In addition to the DERBY and OAKS trials, Apellis is currently evaluating APL-2 in a head-to-head Phase 3 clinical trial for systemic administration comparing APL-2 to Soliris in PNH patients (the PEGASUS trial), a Phase 1b clinical trial for systemic administration in treatment naïve PNH patients (the PADDOCK trial), and a Phase 1b clinical trial for systemic administration in eculizumab-treated PNH patients (the PHAROAH trial).
Apellis Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of a broad range of life-threatening or debilitating autoimmune diseases based upon complement immunotherapy through the inhibition of the complement system at the level of C3. Apellis is the first company to advance chronic therapy with a C3 inhibitor into clinical trials. For additional information about Apellis and APL-2, please visit http://www.apellis.com. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.
Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements” within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the timing of enrollment in the Company’s two Phase 3 GA trials and the potential benefits of APL-2. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: whether Apellis will be able to reinitiate dosing and enroll patients in the Phase 3 GA trials rapidly enough to meet the projected timelines; whether the new manufacturing process referenced in this release will provide the anticipated improvements and be scalable for global commercialization; whether preliminary or interim results from a clinical trial will be predictive of the final results of the trial; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether APL-2 will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of such clinical trials will warrant regulatory submissions and whether APL-2 will receive approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies for GA, PNH or any other indication; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 26, 2019 and the risks described in other filings that Apellis may make with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Apellis specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.