- No cases of inflammation observed after 12 months with Phase 3 formulation of pegcetacoplan in patients with advanced geographic atrophy (GA)
- Decrease in mean lesion growth in nine patients with bilateral GA consistent with Phase 2 FILLY study
- Phase 3 DERBY and OAKS studies expected to fully enroll in the first half of 2020
WALTHAM, Mass., April 28, 2020 (GLOBE NEWSWIRE) — Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company pioneering targeted C3 therapies, today announced early data from the Phase 1b APL2-103 study of pegcetacoplan (APL-2) in patients with advanced geographic atrophy (GA) and low vision. The study, which enrolled 12 patients, was initiated to assess the safety of the Phase 3 formulation of pegcetacoplan (15mg/0.1mL). Patients were dosed with pegcetacoplan in one eye using the fellow eye as untreated control. The analysis of APL2-103 has shown that the Phase 3 formulation was well tolerated over 12 months of treatment. Of the 12 patients, there were no cases of inflammation and one patient (8%) developed new-onset exudation. The formulation used in this Phase 1b study is the same as that being evaluated in the Phase 3 DERBY and OAKS GA studies, which are expected to be fully enrolled in the first half of 2020.
“Our Phase 1b analysis has provided robust safety data, along with a trend of decreased mean lesion growth, for pegcetacoplan in geographic atrophy patients who received treatment with the Phase 3 formulation over 12 months,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “We are encouraged by these results, which give us confidence in targeting C3 and advancing pegcetacoplan as a potential treatment for GA in our ongoing Phase 3 studies.”
Additional analysis at 12 months
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In the nine Phase 1b study patients who had bilateral GA (disease in both eyes), and for whom data were available for at least 12 months, the growth rate of GA lesions in the treated eye was on average 31.1% (mean square root) slower than the opposite untreated eye. This trend is consistent with the reduction seen in the post-hoc analysis of pegcetacoplan compared to the contralateral control eye in the monthly treated group in the completed Phase 2 FILLY study of pegcetacoplan in GA. This observation is noteworthy because lesions in both eyes of patients with bilateral GA are known to grow at similar rates.1 As in FILLY, GA lesions in the APL2-103 study were measured by blinded readers in a third-party reading center.
The ongoing pegcetacoplan development program in GA includes the Phase 1b APL2-103 study and the Phase 3 DERBY and OAKS studies. The patient population enrolled in the Phase 1b study is similar to DERBY and OAKS but allowed for more advanced disease with a wider range of lesion size and lower visual acuity.
In February 2018, Apellis announced positive 18-month results from the Phase 2 FILLY study that demonstrated pegcetacoplan significantly reduced the growth rate of GA lesions. The DERBY and OAKS studies were initiated in 2019 with the pegcetacoplan formulation tested in this Phase 1b study and are expected to fully enroll in the first half of 2020.
About the APL2-103 study
The APL2-103 study is a 12-patient Phase 1b, multicenter, open label, single arm, 24-month clinical trial to assess the safety of monthly intravitreal (IVT) injections of pegcetacoplan in patients diagnosed with advanced geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary outcome measures include incidence and severity of ocular and systemic treatment-emergent adverse events (TEAEs).
About the DERBY and OAKS studies
The DERBY and OAKS studies are 600-patient prospective, international, multicenter, randomized, double-masked, sham-injection controlled Phase 3 studies assessing the efficacy and safety of multiple IVT injections of pegcetacoplan in patients with GA secondary to AMD. For more information, please visit https://gastudy.com/.
About the FILLY study
The FILLY study was a 246-patient Phase 2 multicenter, randomized, single-masked, sham-controlled clinical trial of pegcetacoplan (APL-2) in patients with GA conducted at over 40 clinical sites, located in the United States, Australia and New Zealand. Pegcetacoplan 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 Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), and C3 glomerulopathy. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.i
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 global biopharmaceutical company that is committed to leveraging courageous science, creativity, and compassion to deliver life-changing therapies. By pioneering targeted C3 therapies, we aim to develop best-in-class and first-in-class therapies for a broad range of debilitating diseases that are driven by uncontrolled or excessive activation of the complement cascade, including those within hematology, ophthalmology, and nephrology. For more information, please visit http://apellis.com.
Apellis Forward-Looking Statement
Statements in this press release about future expectations, plans and prospects, such as the statements regarding the timing of enrollment of the DERBY and OAKS trials, 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 the company’s clinical trials will be fully enrolled and completed when anticipated; uncertainties regarding the impact of the Covid19 pandemic on the Company’s business and operations, including the timing and conduct of its ongoing clinical trials, including its DERBY and OAKS trials; whether preliminary or interim results from a clinical trial such as the results presented in this will be predictive of the final results of the trial or of other trials; whether results obtained in preclinical studies and clinical trials will be indicative of results that will be generated in future clinical trials; whether pegcetacoplan will successfully advance through the clinical trial process on a timely basis, or at all; whether the results of the company’s clinical trials will warrant regulatory submissions and whether pegcetacoplan will receive approval from the FDA or equivalent foreign regulatory agencies for GA, PNH, CAD, C3G or any other indication when expected or at all; 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 27, 2020 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.
i. Sunness JS, et al. The long-term natural history of geographic atrophy from age-related macular degeneration: enlargement of atrophy and implications for interventional clinical trials. Ophthalmology. 2007 Feb;114(2):271-7.
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