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™
(intravitreal pegcetacoplan injection)
(intravitreal pegcetacoplan injection)
GA
Approved Marketed in the US
Approved Marketed in the US
APL-2006
GA & Wet AMD
Preclinical
Preclinical
Gene therapies
Wet AMD, Intermediate AMD & GA
Preclinical
Preclinical
RARE DISEASE
Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
EMPAVELI®
(systemic pegcetacoplan)*
(systemic pegcetacoplan)*
PNH
Approved Marketed in the US
Approved Marketed in the US
IC-MPGN & C3G
Phase III
Phase III
ALS
Phase II
Phase II
CAD
Phase II
Phase II
HSCT-TMA
Phase II
Phase II
siRNA + EMPAVELI**
Existing + new indications
Preclinical
Preclinical
Neurology
Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
APL-1030
Undisclosed
Preclinical
Preclinical
Brain Shuttle
Undisclosed
Preclinical
Preclinical
Gene therapies
Undisclosed
Preclinical
Preclinical
MULTIPLE THERAPEUTIC AREAS
Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
Systemic pegcetacoplan
Control of host attack
for gene therapies
Preclinica
for gene therapies
Preclinica
Oral alternative pathway inhibitor
Mild C3G and other indications
Preclinical
Preclinical
Gene-edited therapies (Beam)
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.