posterior segment company showcase - apellis pharmaceuticals
TRANSCRIPT
OIS@AAO 2016
CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
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
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CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
APL-1 Nebulized for COPD Short-acting
Ac-IC*V(Me)WQDWGAHRC*T-NH2
NH
O
NH2
NH NH
NH
O
NH N
NH
O
NH
O
NH O
NH
NH
O
OOH
NH
O
NH2
O
NH
O
N
NH
O
NH
O
O
NH
O
NH
S
S
O
NH
OH
O
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
NH
O
NH 2
NH NH
NH
O
NH N
NH
O
NH
O
NH O
NH
NH
O
OOH
NH
O
NH 2
O
NH
O
N
NH
O
NH
O
O
NH
O
NH
S
S
O
NH
OH
O
NH 2
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
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CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Temporary courses of complement inhibition to correct auto-immunity in diseases like PNH, AMD and COPD
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Science: Complement Immunotherapy
STOP
CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Broad pipeline with near-term clinical milestones
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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)
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
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CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
Inhibition of complement at C3 may overcome limitations of partial inhibition
Potential Benefits APL-2 • Prevent/reduce rate of retinal cell death• Broad patient population• Local administration• Reduced frequency of injections• Disease modifying potential
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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)
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* 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.
CONFIDENTIAL AND PROPRIETARY – for discussion purposes only9
(SEOM)N=40
Sham Every Other
Month
APL-2 15 mgEvery Other Month
(AEOM)N=80
Sham Monthly
(SM)N=40
APL-2 15 mgMonthly
(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
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
CONFIDENTIAL AND PROPRIETARY – for discussion purposes only
FILLY: GA (Ophthalmology)
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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
CONFIDENTIAL AND PROPRIETARY – for discussion purposes only12