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 by investing in nearly a dozen clinical and pre-clinical programs spanning ophthalmology, rare disease, and neurology.

Ophthalmology

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
SYFOVRE®
(pegcetacoplan injection)
GA
Approved Marketed in the US
Oral complement inhibitor
Undisclosed
Preclinical

RARE DISEASE

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
EMPAVELI®
(pegcetacoplan)*
PNH
Approved Marketed in the US
IC-MPGN & C3G
Phase III
CAD
Phase III
HSCT-TMA
Phase II

Neurology

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
Gene/RNA therapies
Undisclosed
Preclinical

MULTIPLE THERAPEUTIC AREAS

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
APL-3007
(siRNA silencing C3)
Undisclosed
Phase I
Gene-edited therapies (Beam)
Undisclosed
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.