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 and only targeted C3 therapy. Now, we are advancing that science by investing in over a dozen clinical and pre-clinical programs spanning rare disease, ophthalmology, and neurology.

RARE DISEASE

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
EMPAVELI®
(systemic pegcetacoplan)*
PNH
Approved Marketed in the US
IC-MPGN & C3G
Phase III
ALS
Phase II Completed enrollment in 1H'22
CAD
Phase II
HSCT-TMA
Phase II
siRNA + EMPAVELI**
Existing + new indications
Preclinical IND in 1H'23

Ophthalmology

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
Intravitreal
pegcetacoplan
GA
Phase III U.S. PDUFA Feb. 26 '23
APL-2006
GA & Wet AMD
Preclinical IND in 1H'23
Gene therapies
Wet AMD, Intermediate AMD & GA
Preclinical

Neurology

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
APL-1030
Undisclosed
Preclinical
Brain Shuttle
Undisclosed
Preclinical  
Gene therapies
Undisclosed
Preclinical  

MULTIPLE THERAPEUTIC AREAS

Product
Disease
Preclinical
Phase I
Phase II
Phase III
Approved
Systemic pegcetacoplan
Control of host attack
for gene therapies
Preclinica
Oral alternative pathway inhibitor
Mild C3G and other indications
Preclinical
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.