Our Pipeline
At Apellis, we aim to transform treatment across a broad range of debilitating diseases driven by complement.
We brought forward the first new class of complement medicine in 15 years with the approval of the first targeted C3 therapy. Now, we are advancing that science in multiple clinical and pre-clinical programs across many serious diseases.
Ophthalmology
Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
SYFOVRE®
(pegcetacoplan injection)
(pegcetacoplan injection)
GA
Approved Marketed in the US
Approved Marketed in the US
APL-3007 + SYFOVRE
GA
Phase I Plan to initiate Ph2 in Q2 2025
Phase I Plan to initiate Ph2 in Q2 2025
RARE DISEASE
Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
EMPAVELI®
(pegcetacoplan)*
(pegcetacoplan)*
PNH
Approved Marketed in the US
Approved Marketed in the US
C3G & IC-MPGN
Phase III PDUFA date: July 28, 2025
Phase III PDUFA date: July 28, 2025
HSCT-TMA
Phase II
Phase II
FSGS
Phase II Plan to initiate pivotal study in H2 2025
Phase II Plan to initiate pivotal study in H2 2025
DGF
Phase II Plan to initiate pivotal study in H2 2025
Phase II Plan to initiate pivotal study in H2 2025
Neurology
Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
RNA therapies
Undisclosed
Preclinical
Preclinical
MULTIPLE THERAPEUTIC AREAS
Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
Gene-edited FcRn therapy (Beam)
Undisclosed
Preclinical
Preclinical
Gene-edited complement therapies (Beam)
Undisclosed
Preclinical
Preclinical
Oral complement inhibitor
Undisclosed
Preclinical
Preclinical
*Sobi has global co-development and ex-U.S. commercialization rights for systemic pegcetacoplan
**Initial IND for siRNA
Potential to be registrational
The safety and efficacy of the agents for the indications under investigation have not been established.