Sobi bets $250M cash, about $1B in milestones for rights to a C3 therapy being pushed through 5 pivotal trials
A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.
The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.
The trials, to run in parallel, include two new registrational programs in cold agglutinin disease (CAD) and hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA), both slated to begin next year. The biotechs will also push forward with existing programs in paroxysmal nocturnal hemoglobinuria (PNH); immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G); and ALS.
In addition to the upfront and milestones, Sobi is sweetening the deal with $80 million in reimbursement payments over a four-year period for R&D. Plus, Apellis co-founder and CEO Cedric Francois said there are “double-digit” royalties on the table.
“This collaboration further strengthens our already strong balance sheets, which we believe will extend our cash runway into the second half of 2022,” Francois said in a conference call on Tuesday morning. He added that the company plans to leverage Sobi’s international infrastructure, and their “strong presence in key global markets.”
Apellis $APLS is keeping US commercialization rights for all indications, and worldwide rights for what Francois said is their “largest indication”: geographic atrophy (GA). Pegcetacoplan is currently in two Phase III trials for GA.
A joint development committee will oversee progress on the other indications, Francois said during the call. PNH is the most advanced of the 5 indications — Apellis has already submitted an NDA, and expects to hear about a PDUFA date by the end of this year.
“Overall, we believe this deal with SOBI is net-net a positive one for APLS, as it should support the robust commercial launch of systemic pegcetacoplan in PNH and beyond,” Baird analysts Madhu Kumar and Robert Palermo wrote in a note.
The upfront, they wrote, will likely allow for “rapid expansion” into CAD and HSCT-TMA, “where reviews suggest complement activation via C3 is a key activation node and clinical case studies suggest C5 mAbs like (Alexion’s) Soliris might not work where there are alternative pathway mutations.”
Francois said Apellis chose Sobi, which he called a “global leader in hematology and rare diseases,” because the collaboration “will be as important to them as it will be to us.”
Two years ago, Sobi put down $50 million upfront and up to $400 million in milestones for the rights to emapalumab — one of its stars after restructuring to focus on hematology and immunology. It later acquired the whole program, with milestones adding up to $518 million. The drug, marketed as Gamifant, is approved in the US for hemophagocytic lymphohistiocytosis (HLH), and raked in $26.7 million in the first half of 2020. But in July, the EMA rejected Sobi’s pitch for approval for reasons CEO and president Guido Oelkers didn’t elaborate on.
Regarding the prospects for pegcetacoplan, Oelkers said in a statement:
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.