RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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CONFIDENTIAL AND PROPRIETARY – DRAFT for discussion purposes only Application to OIS Summit 2016 CONFIDENTIAL AND PROPRIETARY – for discussion purposes only

Transcript of RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

Page 1: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

Application to OIS Summit 2016

CONFIDENTIAL AND PROPRIETARY – for discussion purposes only

Page 2: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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 and COPD

Broad, global IP protection

Experienced management with deep understanding complement and immunology

Strong investor syndicate

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Page 3: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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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Page 4: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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

Page 5: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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=8)

Ph 1b Tx Naïve (n=6)

Ph 2/3 (TBD)

Ph 2 (n=240)

Ph 1 HV (n=20)

Ph 2a PoC (TBD)

Page 6: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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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Page 7: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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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Page 8: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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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.

Page 9: RETINA COMPANY SHOWCASE - Apellis Pharmaceuticals

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(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

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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

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