Apellis, Sobi Partner on Systemic Pegcetacoplan for CAD, Other Rare Diseases

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by Forest Ray PhD |

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Apellis Pharmaceuticals and Swedish Orphan Biovitrum (Sobi) are collaborating to advance the development of systemic pegcetacoplan, or APL-2, in the treatment of cold agglutinin disease (CAD) and other disorders, the companies announced in a press release.

Pegcetacoplan is an investigational therapy designed to limit activation of the body’s complement system — a branch of the immune system — by blocking the activity of the C3 protein. C3 plays a central role in a cascade of events ending in the destruction of red blood cells, an event known as hemolysis.

In CAD, autoantibodies called cold agglutinins cause red blood cells to clump together at temperatures below 30 C, or 86 F. This activates the complement system, which destroys the clumped cells, leading to anemia.

With no approved treatments specific for CAD, a significant unmet need for new therapies exists.

Results of an ongoing Phase 2 clinical trial (NCT03226678) have shown that pegcetacoplan reduces hemolysis and increases levels of hemoglobin among CAD patients and those with a related disorder called warm antibody autoimmune hemolytic anemia.

Under the agreement, Sobi will take the lead on a planned Phase 3 trial testing pegcetacoplan for CAD, scheduled to begin in 2021.

Pegcetacoplan’s key role in regulating the complement response makes it a potential treatment for several rare diseases, for which patients have limited treatment options. In addition to CAD, the collaborators plan to develop pegcetacoplan in five parallel registrational programs involving disorders of the blood, kidneys, and nervous system.

“We are excited to collaborate with Apellis, a leader in targeted C3 therapies,” said Guido Oelkers, CEO and president of Sobi. “Given the central role of C3 in the complement cascade, pegcetacoplan has the potential to become the foundation for a broader platform in rare diseases.”

According to the terms of the agreement, Sobi will receive the rights to co-develop the medication globally and will obtain exclusive rights to commercialize it outside the U.S. Apellis will hold U.S. commercial rights for systemic pegcetacoplan and worldwide rights for the medication’s ophthalmological (for the eyes) formulation. Apellis is evaluating this formulation in two fully enrolled Phase 3 studies of geographic atrophy, an eye disease also known as age-related macular degeneration.

A joint development committee will oversee the co-development of systemic pegcetacoplan, and a joint commercial committee will oversee commercial strategy.

Pegcetacoplan has received fast track designation from the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria, another hemolytic disorder, and geographic atrophy. It also received orphan drug designation for the treatment of the kidney disease C3 glomerulopathy from the FDA and the European Medicines Agency.

“This collaboration enables us to further expand on the broad platform potential of targeting C3 for serious rare diseases that impact hundreds of thousands of patients around the world,” said Cedric Francois, MD, PhD, co-founder and CEO of Apellis.