Sobi and Apellis announce first patient dosed in potentially registrational ALS study of pegcetacoplan
STOCKHOLM and WALTHAM, Mass., Nov. 19, 2020 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (SobiTM) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that the first patient has been dosed in the potentially registrational phase 2 MERIDIAN study of pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with sporadic amyotrophic lateral sclerosis (ALS).
Studies suggest that elevated levels of C3 may play a role in the progression of ALS, a neurodegenerative disease that leads to progressive muscle weakness and paralysis. The MERIDIAN study will assess whether pegcetacoplan may offer a new treatment approach for people living with ALS by controlling complement activation at the level of C3. There are currently no treatments to slow the advance of ALS.
"ALS patients have a very high unmet need. They expect more and better treatment options," said Bashar Al-Nakhala, Chief Operations Officer of the ALS Therapeutic Development Institute. "We are pleased that Apellis and Sobi have joined the ALS clinical development community with our shared goal of halting the devastating progression of ALS."
"We are delighted that the first patient in the phase 2 clinical study has been dosed as there is an urgency for a treatment for patients with ALS" said Ravi Rao, Head of R&D and Chief Medical Officer at Sobi. "In collaboration with Apellis, we look forward to evaluating the potential of pegcetacoplan in patients with ALS."
"Based on the suspected role of C3 in ALS, we are working urgently to understand whether pegcetacoplan, a targeted C3 therapy, has the potential to slow disease progression and make a difference for the ALS community," said Federico Grossi, M.D., Ph.D, Chief Medical Officer of Apellis. "We designed the MERIDIAN study based on significant feedback from the community, and we are dedicated to continuing our partnership to one day bring a meaningful therapy to families living with ALS."
The phase 2 MERIDIAN study (APL2-ALS-206) is a potentially registrational, randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the efficacy and safety of pegcetacoplan in approximately 200 adults with sporadic ALS. Study participants will be randomized in a 2:1 ratio to receive pegcetacoplan or placebo while continuing to receive their existing standard of care treatment for ALS. After 52 weeks of blinded treatment, all patients in the study will 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, with approximately six clinic visits in the first year and four in the open-label second year.
The primary endpoint of the study is the Combined Assessment of Function and Survival (CAFS) rank scores at week 52. Key secondary endpoints include measures of lung function, muscle strength, and quality of life. For more information about the phase 2 MERIDIAN study, visit www.clinicaltrials.gov (NCT04579666).
About amyotrophic lateral sclerosis (ALS)
About pegcetacoplan (APL-2)
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Linda Holmström, Corporate Communication & Investor Relations
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
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SOURCE Swedish Orphan Biovitrum AB
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