Biogen to acquire Apellis, adding PNH treatment Empaveli to portfolio
Deal adds approved therapies for rare blood, kidney, and eye diseases
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Biogen has entered into an agreement to acquire Apellis Pharmaceuticals, adding Empaveli (pegcetacoplan), marketed in Europe as Aspaveli, to its portfolio for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and two other rare diseases involving abnormal complement activation, which is part of the immune system.
For Biogen, acquiring Apellis means immediate access to revenue and growth potential from Empaveli. The company has agreed to acquire Apellis for about $5.6 billion in cash. The agreement also includes potential payments tied to annual global net sales thresholds for Syfovre (pegcetacoplan injection), which contains the same active ingredient as Empaveli and is approved for geographic atrophy, an immune-mediated retinal disease.
Acquisition expands Biogen’s rare disease and immunology portfolio
“Consistent with our strategy, this acquisition immediately advances Biogen’s ongoing transformation. The addition of Apellis expands our growth portfolio in immunology and rare disease with two approved, best-in-class medicines that complement our existing portfolio and bolsters our near-and long-term growth potential,” Christopher A. Viehbacher, Biogen’s president and CEO, said in a company press release.
PNH is most often caused by genetic mutations that make red blood cells vulnerable to a misdirected immune attack. As a result, red blood cells are destroyed too early by an overactive complement system, a cascade of proteins involved in the immune response. Symptoms may include blood in the urine, blood clots, and fatigue due to anemia (low red blood cell levels).
Developed by Apellis, with Sobi holding commercial rights outside the U.S., Empaveli targets C3, a complement protein. Because C3 plays a central role in the complement cascade, blocking it helps prevent the destruction of red blood cells. By acting at this point in the pathway, the therapy can help limit red blood cell destruction both inside and outside blood vessels.
Empaveli works by blocking complement protein C3
Because of this mechanism, Empaveli has been shown to increase hemoglobin levels, the protein that carries oxygen in red blood cells, and reduce the need for blood transfusions in adults with PNH. It is also approved for use in C3 glomerulopathy and primary immune-complex membranoproliferative glomerulonephritis, two rare kidney diseases, where it is used to reduce proteinuria (excess protein in the urine).
“I am incredibly proud of the Apellis team and what we have achieved, including bringing two transformational medicines — Syfovre and Empaveli — to patients and building an innovative pipeline leveraging our deep expertise in complement science,” said Cedric Francois, MD, PhD, Apellis’ co-founder and CEO. “With Biogen’s extensive experience with immunology and rare disease, we believe this transaction will accelerate our impact and enable us to reach more patients.”