Apellis Pharmaceuticals and SFJ Pharmaceuticals are now collaborating on the development of investigational therapy APL-2 as a treatment for cold agglutinin disease (CAD) and warm antibody hemolytic anemia (wAIHA).
This follows the announcement of a newly established partnership to support the clinical program of APL-2 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).
Under the terms of the agreement, Appelis will retain exclusive global commercial rights over APL-2 in all indications upon regulatory approval.
“This innovative collaboration with SFJ provides Apellis with substantial non-dilutive funding to develop APL-2 in hematologic diseases of complement with serious unmet need,” Cedric Francois, CEO and co-founder of Apellis, said in a press release. “We are fortunate to partner with the highly experienced and qualified drug development professionals at SFJ Pharma, who have an outstanding track record of success.”
APL-2 was engineered to specifically inhibit the three immune complement pathways (classical, lectin, and alternative) by blocking the activity of one of its central players, the C3 protein. Given its mode of action, APL-2 has the potential to treat several complement-mediated disorders including PNH, CAD, wAIHA, and geographic atrophy.
According to Apellis, “APL-2 has the potential to be a best-in-class treatment and may address the limitations of existing treatment options or provide a treatment option where there is none.”
APL-2 received orphan drug designation by the U.S. Food and Drug Administration just last month for the treatment of autoimmune hemolytic anemia (AIHA), which includes both CAD and wAIHA. It has also been granted the FDA’s fast track designation for the treatment of PNH.
These designations are expected to support and speed up the clinical development, regulatory review, and approval of APL-2 for these indications.
Apellis is currently testing the safety and efficacy of the C3 inhibitor in patients with PNH in a Phase 3 trial (NCT03500549), called PEGASUS, and two Phase 1b studies, one called PHAROAH (NCT02264639) and another called PADDOCK (NCT02588833).
The company is also evaluating APL-2 in an ongoing Phase 2 open-label trial (NCT03226678), called PLAUDIT, in patients with CAD and wAIHA who are positive for the C3 protein.
This trial, which is still recruiting participants, is intended to evaluate the safety, tolerability, and efficacy of daily treatment with APL-2 in about 24 participants at sites across the United States. Participants will be randomly assigned to receive either 270 mg or 360 mg per day of APL-2 for up to 12 months.
For more information about the PLAUDIT trial, including its locations and contacts, visit the study’s webpage here.
Preliminary data suggests that APL-2 may be an effective strategy to improve the levels of hemoglobin, as well as to reduce hemolysis (red blood cell rupture), reticulocytes, bilirubin, and lactate dehydrogenase (LDH) levels in both CAD and wAIHA patients.
“The collaboration with Apellis is particularly exciting for SFJ as it expands our business model to include pre-revenue biopharma companies,” said Bob DeBenedetto, CEO of SFJ. “After performing an in-depth diligence review of Apellis’ clinical data in PNH, CAD, and wAIHA, as well as the PNH Phase 3 program design and commercial scale manufacturing capabilities, we believe that Apellis is the ideal partner with which to enter this novel agreement.”