Phase 2 Study of Pegcetacoplan in Recently Diagnosed Patients Opens

Phase 2 Study of Pegcetacoplan in Recently Diagnosed Patients Opens
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A Phase 2 and potentially pivotal study investigating pegcetacoplan (APL-2) as a potential treatment for amyotrophic lateral sclerosis (ALS) is now enrolling patients, Apellis Pharmaceuticals, the therapy’s developer, announced in a press release.

The MERIDIAN trial (NCT04579666) is enrolling up to 228 adults with sporadic ALS and symptom onset within the previous 18 months. Recruitment is underway at the Austin Neuromuscular Center, in Texas, with other centers expected to join soon. Apellis is planning to dose a first patient before this year’s end.

Pegcetacoplan inhibits complement protein 3 (C3), a key component of the complement system — a part of the body’s immune system involved in fending off infections, as well as removing dead cells and foreign material.

Uncontrolled complement system activation can trigger excessive inflammatory responses that participate in the onset and progression of many serious diseases, including ALS.

In ALS, C3 is found at high levels in neuromuscular junctions, which function like communication hubs between the nerve cells and muscles. Chronic inflammation at the neuromuscular junction contributes to motor neuron death and to such ALS symptoms as a progressive loss of muscle control and muscle atrophy.

MERIDIAN aims to evaluate the safety and efficacy of pegcetacoplan in recently diagnosed patients. Participants will be randomly assigned to either pegcetacoplan or a placebo for one year. Those who complete this first year will enter the trial’s open-label second year, where all are treated with pegcetacoplan.

Treatment will be administered as an under-the-skin (subcutaneous) injection twice weekly, and patients may continue with their existing standard of ALS care throughout.

The trial’s main goal is to assess a combined score of physical function and survival after one year of treatment against placebo. Secondary measures of safety include side effects and suicidal thoughts throughout the trial’s two years; changes in daily life abilities, lung function, and muscle strength are other secondary goals.

According to the company, MERIDIAN is designed to minimize the number of clinic visits, with six visits required in the first year and four in the subsequent year.

MERIDIAN is a potentially registrational (or pivotal) trial, meaning it has the potential to support a submission to regulatory agencies requesting the approval of pegcetacoplan to treat ALS.

Apellis is also exploring pegcetacoplan as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy, and cold agglutinin disease.

The company also announced the launch of a registrational program in C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN), beginning with the NOBLE Phase 2 trial (NCT04572854) that is expected to open next month.

“People and families living with C3G, IC-MPGN, and ALS are in significant need of new medicines. We believe that controlling complement activation centrally, at the level of C3, has the potential to offer an important new therapeutic approach for each of these rare diseases,” said Cedric Francois, MD, PhD, co-founder and CEO of Apellis.

“Our data in C3G support continued advancement of pegcetacoplan, and numerous studies suggest that elevated levels of C3 may play a role in the progression of ALS. These new trials build on the broad platform potential of targeting C3, and we are working urgently to advance these programs for patients,” Francois added.

Pegcetacoplan has received fast track designation from the U.S. Food and Drug Administration (FDA) for the treatment of PNH and geographic atrophy, and has received orphan drug designation for the treatment of C3G from the FDA and the European Medicines Agency.

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Ciências e Tecnologias and Instituto Gulbenkian de Ciência. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
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