Rare Daily Staff

Apellis Pharmaceuticals said it has begun a late-stage trial of APL-2, an experimental therapy to treat paroxysmal nocturnal hemoglobinuria, a rare, acquired, potentially life-threatening disease.

The study will compare the safety and efficacy of APL-2 to Soliris, the current standard of care for PNH.

PNH is characterized by a chronic, uncontrolled activation of the complement system, a component of the body’s immune system. It results in the destruction of red blood cells, which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath. The condition can also lead to the formation of blood clots, which can damage vital organs and cause premature death. A significant subset of patients treated with the current standard of care still suffer from debilitating anemia and transfusion dependence.

APL-2 is designed to inhibit the complement cascade and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with partial inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a PEG polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation. Apellis is also evaluating APL-2 in a variety of other conditions in which complement is implicated.

“We believe there’s an unmet medical need to bring patients with PNH a better treatment option that will provide broad hematological correction and better quality of life than the current standard of care,” said Cedric Francois, Apellis co-founder and CEO.

June 27, 2018
Photo: Cedric Francois, Apellis co-founder and CEO

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