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CRESTWOOD, Ky., and WALTHAM Mass. -- 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 it has commenced its Phase 3 clinical program for APL-2 in patients with geographic atrophy (GA), consisting of two Phase 3 trials (DERBY & OAKS.) The first patient has been dosed in the OAKS trial and the first patient has been enrolled in the DERBY trial. Both trials will assess the safety and efficacy of APL-2 in patients with GA, an advanced form of age-related macular degeneration. There are approximately 1 million patients in the United States with GA and there are currently no FDA-approved treatments for the disease, which leads to loss of visual function and blindness.
“Dosing the first patient in our Phase 3 program in geographic atrophy is an exciting milestone for Apellis, physicians, and most importantly patients suffering from GA,” said Cedric Francois, MD, PhD, co-founder and CEO of Apellis. “APL-2 offers hope for patients currently without an approved treatment option.”
“Patients living with GA over time lose their ability to drive, read and perform daily functions, so a treatment that could potentially reduce the rate of GA lesion growth and save visual function is important to this population,” said Nathan Steinle, MD, Retina Specialist at California Retina Consultants.
The Phase 3 program consists of two 600-patient prospective, international, multicenter, randomized, double-masked, sham-injection controlled studies (DERBY & OAKS) to assess the efficacy and safety of multiple intravitreal (IVT) injections of APL-2 in patients with GA. The Phase 3 trials are similar in design to Apellis’ Phase 2 FILLY trial, including the eligibility criteria and primary endpoint of change in GA lesion size from baseline to month 12, compared to sham. In DERBY and OAKS, patients will continue on therapy for an additional 12 months beyond the 12-month primary endpoint. APL-2 met its primary endpoint in the Phase 2 FILLY trial. At 12 months, APL-2, administered monthly via intravitreal injection, showed a 29% (p=0.008) reduction in the rate of GA lesion growth compared to sham. With every other month administration, a 20% (p=0.067) reduction compared to sham was observed.
Apellis recently received Fast Track Designation from the Food and Drug Administration (FDA) for APL-2 for the treatment of GA.
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).
About the FILLY trial
The FILLY trial was 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 without active treatment until Month 18. Eyes were evaluated for GA by fundus autofluorescence photographs (FAF). The rate of GA area growth was measured from baseline to Month 18. The primary efficacy endpoint was the change in GA lesion size from baseline to Month 12, compared to sham.
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. About APL-2APL-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 with hemoglobin levels less than 10.5g/dL (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 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 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 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 April 30, 2018, 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.