Apellis Pharmaceuticals’ APL-2 Receives Fast Track Designation from FDA for the Treatment of Patients with Geographic Atrophy
July 24, 2018
The FDA’s Fast Track program facilitates the development and expedites the review of drugs to treat serious conditions and fill an unmet medical need, allowing important new drugs to reach the patient earlier. Filling an unmet medical need is defined as providing a therapy where none exists or providing a therapy which may have potential benefits over existing therapies. Drugs that receive a Fast Track designation are eligible for more frequent meetings and written communication with the
“The Fast Track designation represents an important recognition by the
Apellis plans to initiate a Phase 3 trial for patients with GA later this year which will consist of two identical, 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.
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 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.
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. PNH Phase 3 study, PEGASUS commenced in June and a Phase 3 in GA in anticipated later this year.
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.
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
Source: Apellis Pharmaceuticals, Inc.