· 52-week phase 2 MERIDIAN study to evaluate pegcetacoplan, a targeted C3 therapy, in approximately 200 adults with ALS
Stockholm, (https://www.sobi.com/)
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
"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
"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
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 (https://www.clinicaltrials.gov/ct2/show/NCT04579666?term=NCT04579666&draw=2&rank=1) (NCT04579666).
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] In individuals with ALS, high levels of C3 are present at the neuromuscular junction[3] where motor neurons communicate directly to muscle cells. Numerous studies suggest that elevated levels of C3 present throughout the motor system of ALS patients are likely to contribute to chronic neuroinflammation and the death of motor neurons.[3,4,5] There are currently no approved treatments that stop or reverse the progression of ALS, which impacts ~225,000 patients worldwide.[6]
About pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, 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. Pegcetacoplan is being evaluated in several clinical studies across haematology, ophthalmology, nephrology, and neurology. Marketing applications for pegcetacoplan for paroxysmal nocturnal haemoglobinuria (PNH) are under review by the
About Apellis
About Sobi™
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,500 people across
For more information please contact
Sobi
Paula Treutiger, Head of Communication & Investor Relations
+ 46 733 666 599
paula.treutiger@sobi.com
+ 46 708 734 095
linda.holmstrom@sobi.com
Apellis
Media
media@apellis.com
+1 617 420 4839
Investors
sam@argotpartners.com / maghan@argotpartners.com
+1 212 600 1902
[1 ]
[2]
[3 ]
[4 ]Woodruff, et al., PNAS
[5 ]Lee, et al
[6 ]Arthur K et al.
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Phone: 46 8 697 20 00 www.sobi.com
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