Posterior Segment Company Showcase - Apellis Pharmaceuticals at OIS@AAO 2016. Presenter: Cedric Francois, MD, PhD, CEO Powered by: Healthegy For more ophthalmology innovation Visit us at www.ois.net

1. OIS@AAO 2016
CONFIDENTIAL AND PROPRIETARY – for discussion purposes only

2. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Apellis investment highlights
 Pioneers in the development of C3 inhibitors
 Wholly-owned, lead product candidates APL-1 and APL-2
 Potent and selective C3 inhibitors
 Clinical pipeline targets autoimmune and inflammatory diseases
 Initially PNH, AMD and COPD
 Multibillion dollar global market opportunities
 Multiple near-term clinical milestones
 2016: Phase 1b results in PNH
 2017: Phase 2 results in GA
 Broad, global IP protection
 Experienced management with deep understanding complement and immunology
2

3. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
 APL-1
 Nebulized for COPD
 Short-acting
Ac-IC*V(Me)WQDWGAHRC*T-NH2
APL-1 and APL-2 are potent and selective
C3 inhibitors
 APL-2
 Long-acting version of APL-1
 Subcutaneous for PNH
 Intravitreal for GA and
intermediate AMD
APL-1Ac-IC*V(Me)WQDWGAHRC*T-NH2
Peptides of the APL-1 / APL-2 family bind
to a pocket of C3 and inhibit activation*
* Janssen, J. Biol. Chem., 282(40), 29241-29247, 2007
3

4. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Temporary courses of complement inhibition to correct auto-immunity in diseases like PNH, AMD
and COPD
4
Science: Complement Immunotherapy
Complement
ActivationPhagocyte Activation
(e.g. M1/ M2)
Oxidation of proteins /
phospholipids
Adduct formation
Cell Death
Tissue specific
Dendritic Cells
T Cells
B Cells
Low affinity
Polyclonal Antibodies
Th17
Polarization
Th17
Signaling
Fluid Phase Activation
Oxidative Damage, Allergens
Infections?
Break Self-Tolerance
Classical
Alternative
(MDA à CFH402)
LYSISIMMUNE
STOP

5. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Broad pipeline with near-term clinical milestones
5
2016 2017
1H 2H 1H 2H
Chronic Obstructive Pulmonary Disease (Pulmonary)
Paroxysmal Nocturnal Hemoglobinuria (Rare Indications)
Geographic Atrophy in AMD (Ophthalmology)
Ph 1b Soliris (n=12)
Ph 1b Tx Naïve (n=5)
Ph 2/3 (TBD)
Ph 2 (n=246)
Ph 1 HV (n=20)
Ph 2a PoC (TBD)

6. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Meaningful unmet need in
age-related macular degeneration
DISEASE
 Intermediate AMD: drusen but no serious vision loss /
precedes Wet AMD and Geographic Atrophy (GA)
 Wet AMD: blood leakage in retina  rapid blindness if
not treated
 GA: slow progressive retinal death  starts in periphery /
blind when central vision is affected
 ~15M patients with AMD, ~1M with GA in US*
STANDARD OF CARE
 Wet AMD: anti-VEGF (Lucentis, Avastin, Eylea)
 iAMD and GA: supportive care
UNMET NEED
 Intermediate AMD: no approved therapies to slow
progression to Wet AMD or GA
 GA: no approved therapies
* http://www.asrs.org/patients/retinal-diseases/2/agerelated-macular-degeneration
Intermediate AMD
Wet AMD
GA
6

7. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Inhibition of complement at C3 may overcome
limitations of partial inhibition
Activation
Pathways
Classical pathway
Activated by antibody-
antigen complex
Lectin pathway
Activated by lectin and
mannose complex
Alternative pathway
Spontaneous C3
convertase activation
C5
C5bC5a MACInflammation
Inflammation Cell removal
Lampalizumab
Cell destruction
C3
C3bC3a
Potential Benefits APL-2
• Prevent/reduce rate of retinal cell death
• Broad patient population
• Local administration
• Reduced frequency of injections
• Disease modifying potential
7

8. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Inhibiting complement in GA reduces
retinal cell death
LAMPALIZUMAB
 Only inhibits alternative pathway
 Reduced retinal death rate by 44% in CFI+
patients
 No effect in CFI- patients
 Monthly injections
POTENTIAL OPPORTUNITY FOR APL-2 IN GA
 Inhibits alternative and classical pathway
 Similar effect expected in CFI+ patients
 May reduce retinal cell death rate in CFI- patients
 Longer intravitreal half life may reduce frequency
of injections
Lampalizumab Phase 2
MAHALO Trial*
(CFI+ Patients)
(n=14,13,12) (n=17,17,16)
8
* Regillo CD. Lampalizumab (anti-factor D) in patients with geography atrophy: the MAHALO phase 2 results. Paper presented at: the 2013 Annual Meeting of the
American Academy of Ophthalmology; November 16-19, 2013; New Orleans, LA.

9. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only9
(SEOM)
N=40
Sham
Every Other
Month
APL-2 15 mg
Every Other Month
(AEOM)
N=80
Sham
Monthly
(SM)
N=40
APL-2 15 mg
Monthly
(AM)
N=80
Safety, Tolerability and Evidence of Activity N=240
Randomized 2:1:2:1
Phase 2 GA – Filly design
Treatment Period Follow up
AM=2 D0
AEOM=2
SM=1
SEOM=1
M1 M2 M3 M4 M5 M6 M7 M8 M9 M10 M11 M12
D0 M1 M2 M3 M4 M5 M6 M7 M8 M9 M10 M11 M12
D0 M2 M4 M6 M8 M10 M12
D0 M2 M4 M6 M8 M10 M12
Randomization
M15 M18
M15 M18
M15 M18
M15 M18

10. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Primary Efficacy Endpoint
 The primary endpoint is the change in square root geographic
atrophy (GA) lesion size from baseline at month 12 as measured by
FAF.
Primary Safety Endpoint
 Number and severity of local and systemic treatment emergent
averse events (TEAE).
Phase 2 GA – Filly Endpoints

11. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
FILLY: GA (Ophthalmology)
11
Screened Pass Enrolled Early Withdrawal
417 246 246 7
 Study is fully enrolled
 Last subject was randomized on July 18th 2016
 Twelve months read out is expected in September-October 2017

12. CONFIDENTIAL AND PROPRIETARY – for discussion purposes only12