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New designation now includes all PNH patients
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 diseases through inhibition of the complement system, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the Company’s APL-2, a novel inhibitor of complement factor C3, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). APL-2 is currently being studied in a Phase 3 active comparator trial in patients with PNH who remain anemic despite treatment with eculizumab.
This new Fast Track designation for APL-2 for all patients with PNH, supersedes the prior Fast Track designation granted on December 15, 2016 for APL-2 for the subset of PNH patients who continue to experience hemolysis requiring blood transfusions despite receiving therapy with eculizumab.
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 FDA to discuss development plans and clinical trial design. In addition, if criteria are met, Fast Track-designated drugs are eligible for Rolling Review, where a drug company can separately submit sections of its New Drug Application to the FDA, and Accelerated Approval and Priority Review, in which drugs for serious conditions fulfilling an unmet medical need can be approved based on a surrogate endpoint.
“We are very pleased that the FDA recognizes the potential for APL-2 to help every patient with PNH. We believe that APL-2 has the potential to be a best in class treatment for all PNH patients,” said Pascal Deschatelets, PhD, co-founder and chief operating officer of Apellis.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. A significant subset of patients treated with the current standard of care still suffer from debilitating anemia and transfusion dependence.
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). To date, APL-2 has generally been well-tolerated. No significant infections have been observed in trials involving the systemic administration of APL-2, including the trials in PNH, AIHA or other trials.
In hematologic diseases, Apellis is currently evaluating APL-2 in PEGASUS, a Phase 3 trial for patients with PNH as well as in two Phase 1b trials (PHAROAH and PADDOCK) for systemic administration. Previously reported interim data from these 1b trials showed improvements in lactate dehydrogenase and hemoglobin levels in patients who are suboptimal responders to eculizumab and untreated patients, respectively. Apellis is also testing APL-2 for systemic administration in a Phase 2 clinical trial in autoimmune hemolytic anemia (AIHA) and a Phase 2 clinical trial in complement dependent nephropathies. For additional information regarding our clinical trials, visit www.apellis.com/clinical-trials.html.
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 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; 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, C3G 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’ Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 13, 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.