Apellis, Sobi halt development of pegcetacoplan for CAD

Apellis said Enjaymo, other therapies 'decreased need' for new therapies

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

Share this article:

Share article via email
A woman shouts an announcement into a bullhorn.

Apellis Pharmaceuticals and Swedish Orphan Biovitrum (Sobi) have stopped the clinical development of the experimental therapy pegcetacoplan for cold agglutinin disease (CAD).

The decision, which includes discontinuing a Phase 3 clinical trial of the therapy in adults, was based on a realignment of the companies’ priorities in light of recent therapeutic advances, according to a recent Apellis corporate update. The placebo-controlled Phase 3 study, called CASCADE (NCT05096403) and started in late 2022, sought to enroll 57 patients in the U.S., Europe, and Japan who hadn’t recently received other CAD treatments.

In early 2022, Sanofi‘s Enjaymo (sutimlimab-jome) became the first therapy to be formally approved for CAD in the U.S. With Enjaymo’s approval and because other therapies are being developed, “there is a decreased medical need in CAD and therefore a limited number of patients eligible for the study,” Apellis said.

Sobi, which was sponsoring the CASCADE trial as per the 2020 collaboration agreement with Apellis to develop pegcetacoplan for CAD, is now working with trial investigators to help participants find an appropriate treatment option going forward.

Recommended Reading
A child pretends to be a sailor, sitting in a box on a blanket.

Epstein–Barr virus is cause of boy’s complicated secondary CAD

Phase 2 study showed pegcetacoplan reduced hemolysis

CAD is caused by antibodies that stick to red blood cells at low temperatures, which leads to their destruction, called hemolysis. When antibodies stick to cells, they activate a group of immune proteins called the complement cascade, whose activation plays a major role in CAD-related hemolysis.

Pegcetacoplan, previously known as APL-2, is designed to block the complement cascade’s activation by suppressing a complement protein called C3. It’s administered systemically via an infusion pump under the skin.

Data from an earlier Phase 2 pilot study (NCT03226678), which tested pegcetacoplan in 24 adults with CAD or a related disorder called warm antibody autoimmune hemolytic anemia, indicated it could reduce hemolysis and increase hemoglobin, the protein in red blood cells that carries oxygen through the body.

The Phase 3 CASCADE trial was testing whether pegcetacoplan was better than a placebo at increasing hemoglobin without the need for a blood transfusion. It’s effectiveness wasn’t being evaluated as a result of it being a blind study, meaning neither the patients nor investigators were aware of who was taking the therapy or a placebo.

No noteworthy safety issues were reported, however, Apellis stated.