Apellis and Sobi Enter Collaboration for Global Co-development and Ex-US Commercialization of Systemic Pegcetacoplan in Rare Diseases with Urgent Need for New Treatments - BiopharmaDirect

Apellis and Sobi Enter Collaboration for Global Co-development and Ex-US Commercialization of Systemic Pegcetacoplan in Rare Diseases with Urgent Need for New Treatments

27 October 2020

• Sobi obtains global co-development and exclusive ex-U.S. commercialization rights for systemic pegcetacoplan, a targeted C3 therapy.
• Apellis retains U.S. commercialization rights for systemic pegcetacoplan and worldwide commercialization rights for ophthalmological pegcetacoplan (geographic atrophy program in Phase 3)
• The companies will jointly advance systemic pegcetacoplan in five parallel registrational programs including two new hematological studies planned to start in 2021 (CAD and HSCT-TMA). These join ongoing registrational programs in hematology (PNH), nephrology (IC-MPGN/C3G) and neurology (ALS)
• Sobi will make an upfront payment of $250 million to Apellis and $80 million in committed development reimbursements over four years and up to $915 million in regulatory and commercial milestones plus tiered double-digit royalties

WALTHAM, Mass. and STOCKHOLM, Sweden, Oct. 27, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi^TM) (STO:SOBI) today announced a strategic collaboration to accelerate the advancement of systemic pegcetacoplan, a targeted C3 therapy, for the treatment of multiple rare diseases with high unmet need that impact more than 275,000 patients globally.

Sobi will receive global co-development and exclusive ex-US 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 fully enrolled Phase 3 studies in geographic atrophy (GA). Pegcetacoplan targets 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. These include new registrational programs in cold agglutinin disease (CAD) and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA), both of which are expected to start in 2021. 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.

"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, M.D., Ph.D., 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. Together, we will quickly advance systemic pegcetacoplan in multiple registrational programs across hematology, nephrology, and neurology while also preparing for our first potential U.S. launch in PNH. Financially, this transaction also strengthens our position, with our cash runway expected to extend into the second half of 2022."

"We are excited to collaborate with Apellis, a leader in targeted C3 therapies. 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, chief executive officer 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. With positive Phase 3 data in PNH, pegcetacoplan can elevate the standard of care for this debilitating blood disorder."

As part of the collaboration, Apellis and Sobi will co-develop systemic pegcetacoplan in the following rare diseases:

Hematology – Paroxysmal nocturnal hemoglobinuria (PNH), CAD, and HSCT-TMA
PNH represents the first potential indication to market for systemic pegcetacoplan. Marketing applications for pegcetacoplan for the treatment of PNH were submitted to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) based on positive results from the Phase 3 PEGASUS study. 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.

Sobi will lead development activities for the Phase 3 study in CAD and a potentially registrational Phase 2 study in HSCT-TMA, both planned to start in 2021.

Nephrology – Immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G)
Apellis has initiated and will continue to lead a registrational program in IC-MPGN and C3G, which includes Phase 2 and Phase 3 studies.

Neurology – Amyotrophic lateral sclerosis (ALS)
Apellis has initiated and will continue to lead a potentially registrational Phase 2 study in ALS. Multiple other neurological conditions are under consideration for future clinical development.

About the Transaction
Sobi will make an upfront payment of $250 million to Apellis and up to $915 million in other regulatory and commercial milestone payments, and will 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 intends to finance these payments with available funds. 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 Marketing Authorization Application (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. The co-development of systemic pegcetacoplan will be overseen by a joint development committee, and the commercial strategy will be overseen by a joint commercial committee.

Source: apellis.com