WALTHAM, Mass., May 25, 2023 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the Phase 2 MERIDIAN study investigating systemic pegcetacoplan for the treatment of amyotrophic lateral sclerosis (ALS) did not meet its primary endpoint of the Combined Assessment of Function and Survival (CAFS) rank score at Week 52. The study also did not meet key secondary efficacy endpoints. Systemic pegcetacoplan was well tolerated in the study, and the data were consistent with the established safety profile.
Based on the lack of efficacy, Apellis and Sobi plan to discontinue development of systemic pegcetacoplan for ALS. In April, Apellis and Sobi discontinued treatment in the open-label portion of the study, following a recommendation from an independent data monitoring committee.
“We are disappointed in the outcome of the MERIDIAN study, especially on behalf of the ALS community who has been waiting for new treatments for this complex and unrelenting disease. We would like to sincerely thank the study participants and their caregivers from around the world who contributed to this important research,” said Jeffrey Eisele, Ph.D., chief development officer, Apellis. “Our hope is that the data generated from this study will continue to support future research and development in ALS.”
The full MERIDIAN dataset is being analyzed, and detailed data is expected to be presented at a future medical meeting.
About the Phase 2 MERIDIAN Study
The Phase 2 MERIDIAN study (NCT04579666) is a randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of systemic pegcetacoplan in approximately 250 adults with sporadic amyotrophic lateral sclerosis (ALS). Study participants were randomized in a 2:1 ratio to receive pegcetacoplan or placebo while continuing to receive their existing standard of care treatment for ALS for 52 weeks. The primary endpoint of the study is the Combined Assessment of Function and Survival (CAFS) rank scores. Key secondary endpoints include measures of overall function, survival, lung function, and muscle strength. After 52 weeks, all study participants were to receive pegcetacoplan. To reduce the burden on people living with ALS and their caregivers, the study has been designed to minimize the number of in-clinic visits.
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a devastating neurodegenerative disease that results in progressive muscle weakness and paralysis due to the death of nerve cells, called motor neurons, in the brain and spinal cord.1,2 The death of motor neurons leads to the progressive loss of voluntary muscle movement required for speaking, walking, swallowing, and breathing.1,2 There are currently no approved treatments that stop or reverse the progression of ALS, which impacts ~225,000 patients worldwide.3
About Pegcetacoplan in Rare Diseases
Pegcetacoplan is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, a part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is under investigation for several rare diseases across hematology, nephrology, and neurology. Pegcetacoplan is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) as EMPAVELI® in the United States, Australia, Canada, and Saudi Arabia and in the European Union and the United Kingdom as Aspaveli®.
About the Apellis and Sobi Collaboration
Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, and Apellis has exclusive U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, including for geographic atrophy.
Apellis Pharmaceuticals, Inc. is a global biopharmaceutical company that combines courageous science and compassion to develop life-changing therapies for some of the most challenging diseases patients face. We ushered in the first new class of complement medicine in 15 years and now have two approved medicines targeting C3. These include the first and only therapy for geographic atrophy, a leading cause of blindness around the world. With nearly a dozen clinical and pre-clinical programs underway, we believe we have only begun to unlock the potential of targeting C3 across many serious diseases. For more information, please visit http://apellis.com or follow us on Twitter and LinkedIn.
Apellis Forward-Looking Statement
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. 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 discussed in the “Risk Factors” section of Apellis’ Annual Report on Form 10-K with the Securities and Exchange Commission on February 21, 2023 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.
1 National Institute of Neurological Disorders and Stroke. (2020). Amyotrophic Lateral Sclerosis Fact Sheet. Retrieved from https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-lateral-Sclerosis-ALS-Fact-Sheet
2 ALS Association. What is ALS? Retrieved June 2020 from https://www.als.org/understanding-als/what-is-als
3 Arthur K et al. Nat Commun, 2016, Vol 7, article 12408