CRESTWOOD, Ky. and CAMBRIDGE, Ma., Dec. 20, 2017- 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, following discussions with the FDA, it has finalized the trial design for its planned Phase 3 program evaluating APL-2 for the treatment of patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD).
The Phase 3 program, planned to begin in the second half of 2018, will consist of two identical 600-patient prospective, multicenter, randomized, double-masked, sham-injection controlled studies to assess the efficacy and safety of multiple intravitreal (IVT) injections of APL-2 in patients with GA. The Phase 3 trials will be substantially similar in design to Apellis’ ongoing Phase 2 FILLY trial, which, at 12 months, showed a 29% reduction in the growth of GA lesions in the monthly treatment group (p=0.008) and a 20% reduction in the every other month treatment group (p=0.067). The primary endpoint, change in GA lesion size from baseline to month 12 as compared to sham, is unchanged from Phase 2, as are the enrollment criteria, which will include patients with a history of wet AMD in the fellow eye. Patients whose treatment eye develops wet AMD will continue to be treated with APL-2 along with anti-VEGF therapy, the current standard of care for wet AMD.
“Our Phase 3 trial design is intended to address a patient population similar to the one we studied in Phase 2, which we believe is representative of the general population of patients with GA in the United States,” said Cedric Francois, MD, PhD, founder and chief executive officer of Apellis. “Currently there are no approved treatments for the approximately one million patients suffering from GA in the US. We believe that by slowing down the rate of degeneration of retinal tissue through broad C3 inhibition, we may be able to delay or prevent the progression to blindness in these patients.”
About the FILLY trial
The FILLY trial is a 246-patient Phase 2 multicenter, randomized, single-masked, sham-controlled clinical trial of APL-2 in patients with GA conducted at over 40 clinical sites, located in the United States, Australia and New Zealand. APL-2 was administered as an intravitreal injection in the study eye monthly or every other month for 12 months, followed by six months of monitoring after the end of treatment. Eyes were evaluated for GA by fundus autofluorescence photographs (FAF). The rate of GA area growth was measured by mean change in square root area of GA lesion from baseline to month 12. The primary endpoint was the change in GA lesion size from baseline to month 12, compared to sham.
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 FILLY trial in GA, Apellis is currently evaluating APL-2 in two clinical trials for systemic administration in paroxysmal nocturnal hemoglobinuria (PNH). Interim data from these trials demonstrated meaningful improvements in lactate dehydrogenase and hemoglobin levels in previously untreated patients as well as patients who are suboptimal responders to eculizumab, the current standard of care in the treatment of PNH. Phase 3 studies are planned in GA and PNH, and future clinical studies of APL-2 are anticipated in other diseases in which complement is implicated.
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, we estimate that approximately one million people have GA in the United States alone. There are currently no approved treatments for GA.
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.
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 implications of preliminary clinical data. 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 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 such as the results referenced in this release 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, and receive approval from the United States Food and Drug Administration or equivalent foreign regulatory agencies; whether, if Apellis’ products receive approval, they will be successfully distributed and marketed; and other factors discussed in the “Risk Factors” section of Apellis’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on December 20, 2017, 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.
Click here to view the original press release.