Apellis and Sobi Enter Potential Blockbuster Collaboration to Develop Drug for Multiple Rare Diseases

Rare Daily Staff

Apellis Pharmaceuticals and Swedish Orphan Biovitrum entered a strategic collaboration to accelerate the global advancement of systemic pegcetacoplan for the treatment of multiple rare diseases with high unmet need.

By controlling complement activation centrally, pegcetacoplan offers the potential to become a transformative new therapy in several rare diseases where patients have few or no treatment options today.

Under the terms of the deal, which is valued at $1.2 billion including an upfront payment of $250 million to Apellis, Sobi will receive global co-development and exclusive ex-U.S. commercialization rights for systemic pegcetacoplan. Apellis retains U.S. commercialization rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, which is being evaluated by Apellis in two ongoing phase 3 studies in geographic atrophy (GA).

Sobi will also contribute $80 million in reimbursement payments over a four-year period for research and development to support the initial development plan, which includes ongoing studies in PNH, IC-MPGN/C3G, and ALS and new studies in CAD and HSCT-TMA. Apellis will also be eligible for tiered double-digit royalties on sales ranging from high teens to high twenties. Sobi will receive reimbursement payments for the costs incurred by Sobi in connection with the CAD and HSCT-TMA trials that Sobi will conduct. The parties have agreed to split costs 50/50 for any future global studies beyond the initial development plan.

Per the terms of the agreement, Apellis will be responsible for all regulatory and commercial activities in the United States and the ongoing MAA review for PNH in the European Union, which will be subsequently transferred to Sobi. Sobi will be responsible for regulatory and commercial activities for systemic pegcetacoplan in ex-US markets. A joint development committee will oversee the co-development of systemic pegcetacoplan, and a joint commercial committee will oversee the commercial strategy.

Pegcetacoplan is an experimental synthetic cyclic peptide conjugated to a polyethylene glyocol polymer that binds specifically to C3 and C3b, targeting excessive activation of C3 in the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases.

Apellis and Sobi plan to jointly advance the clinical development of systemic pegcetacoplan in five parallel registrational programs across hematology, nephrology, and neurology, which include cold agglutinin disease (CAD) and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA), both of which are expected to start in 2021

“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, co-founder and chief executive officer of Apellis. “We evaluated numerous companies, medium and large, and chose Sobi because of their global leadership in hematology and rare diseases, track record of successful product launches, and deep commitment to patients. Financially, this transaction also strengthens our position, with our cash runway expected to extend into the second half of 2022.”

Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Together, the companies can quickly advance systemic pegcetacoplan in multiple registrational programs across hematology, nephrology, and neurology. The company is also preparing for its first potential U.S. launch in paroxysmal nocturnal hemoglobinuria (PNH), a rare, chronic, life-threatening blood disorder characterized by the destruction.

The U.S. Food and Drug Administration granted pegcetacoplan Fast Track designation for the treatment of PNH and the treatment of geographic atrophy and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. The NDA and MAA submissions for pegcetacoplan for the treatment of PNH are based on positive results from the phase 3 PEGASUS study, a multi-center, randomized, active-comparator controlled study in 80 adults with PNH. Both agencies are expected to make a decision on marketing approval over the next few months. Top-line results from the Phase 3 PRINCE study, which is evaluating pegcetacoplan in treatment-naïve patients with PNH, are expected in the first half of 2021.

“The collaboration will significantly strengthen and broaden our late-stage R&D portfolio and be a catalyst for further internationalization. The products have an excellent fit with our strategic focus on hematology and immunology,” said Guido Oelkers, president and CEO 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. With positive phase 3 data in PNH, pegcetacoplan can elevate the standard of care for this debilitating blood disorder.”

Photo: Guido Oelkers, president and CEO of Sobi