Developing multi-benefit
DARPin® therapies to
improve health and advance
modern medicine
Cowen and Company - 35th Annual Healthcare Conference
Boston, March 3, 2015
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Confidential - 2
Corporate overview
• Assets in ophthalmology, oncology and immunology
• Pipeline of 4 DARPin® product candidates and several research programs
• Abicipar (partnered with Allergan) is expected to enter Phase III in wet AMD in Q2 2015
• Proprietary assets in oncology and ophthalmology
• DARPin® technology platform
• Multiple partnerships with blue chip pharma companies
• c.CHF 161 million non-equity funding collected1
• c.CHF 3.0 billion milestone potential2
• Up to double-digit royalties in all partnerships
• Financials
• Second largest Biotech IPO in Europe in 2014: Raised CHF 106.2 million on SIX Swiss Exchange (MOLN)
• CHF 55.8 million venture capital raised pre-IPO
• Pre-IPO cash & cash equivalents of CHF 99.0 million with multiple years of runway3
1
up until June 30, 2014; 2 Assumes exercise of all options for additional programs and attainment of all research option fees, preclinical milestones, development milestones and full commercial
milestones for the maximum number of products; 3 as of June 30 2014;
Confidential - 3
Strong management team includes DARPin
creators
Christian Zahnd, CEO
• Founder, board member
• PhD in Molecular Biology from University of
Zurich
Patrick Amstutz, COO
• Founder
• PhD in Molecular Biology from University of
Zurich
Michael Stumpp, CSO
• Founder
• PhD in Molecular Biology, Postdoc from
University of Zurich
Andreas Emmenegger, CFO
• Previously with Roche and Glycart
• Ex-CFO of two listed companies
From left to right: Dr. Stumpp, Mr. Emmenegger, Dr. Amstutz, Dr. Zahnd
Confidential - 4
Experienced and independent board of directors
Jörn Aldag (Chairman)
Steve Holtzman (Non-executive director)
• CEO uniQure, Ex CEO Evotec
• EVP BD Biogen, Founder and Chairman of
Infinity, Ex CBO of Millenium
Christian Zahnd (CEO)
Francesco de Rubertis (Investor)
• CEO Molecular Partners, Founder
• Index Ventures
Göran Ando (Non-executive director)
Petri Vainio (Investor)
• Chairman Novo Nordisk, Ex CEO Celltech, Ex
CSO Pharmacia
• Essex Woodlands Ventures
Bill Lee (Non-executive director)
Andreas Plückthun (Founder)
• SVP Research Gilead, part of the Gilead team
from the start
• Professor UZH, co-founder of Morphosys
Confidential - 5
Broad pipeline addressing high value end markets
Immunology
Oncology
Ophthalmology
Description
Area
Target
Abicipar
wet AMD
VEGF
Abicipar
DME
VEGF
multi-DARPin®
VEGF/PDGF
wet AMD
3 programs
n.d.
n.d.
2 programs
n.d.
n.d.
MP0250
solid tumors
VEGF/HGF
MP0274
solid tumors
HER2
2 Programs
immunooncology
n.d.
Multiple
programs
DARPin®-Toxins
n.d.
Program
Immunology
n.d.
Partner
Preclinical development
Clinical development
Discovery
Phase I
Preclinics
Phase II
Phase III
Expected Phase III
start in Q2 2015
Phase II study
started in July 2014
VEGF/PDGF
Phase I study started
in July 2014
Lighter shading indicates research program
Confidential - 6
Unlocking value in partnerships
• Validation of the DARPin® approach
Comments
• All partnerships expanded over time
• Validation of the technological approach
• Upfront: CHF 99.7 million¹
Ophthalmology
• Milestones: c.CHF 1.8 billion2,3,4
• Attractive economical value
• Royalties: up to double-digit3
• Significant revenue potential
• Upfront: up to CHF 55 million
• Strong long-term value retention
• Milestones: c.CHF 1.0 billion4
• Development costs borne by partner
Oncology
• Significant upfront and milestones
• Potential for further partnerships
• Unlock the full potential of DARPins®
• Royalties: up to low double-digit
Immunology
• Royalties: up to low double-digit
• Option taken for program in Dec 14
• Use of DARPins® in novel fields
• Increase speed to market
c.CHF 3.0 billion4 in milestone potential
c.CHF 161 million in non-equity funding collected
1
as per audited accounts; USD 45 million (CHF 38.8 million) for Abicipar and USD 62.5 million (CHF 60.9 million) for multi-DARPin® VEGF/PDGF and discovery alliance; 2 Exchange rate as of 31 Dec 2014:
CHF/USD: 1.0064; USD 375 million (CHF 373 million) for Abicipar and USD 1.4 billion (CHF 1.4 billion) for multi-DARPin® VEGF/PDGF and discovery alliance; 3 Co-funding option for significant step-up in
royalties on multi-DARPin® VEGF/PDGF; 4 Assumes exercise of all options for additional programs and attainment of all research option fees, preclinical milestones, development milestones and full
commercial milestones for the maximum number of products
Confidential - 7
DARPin™ Technology
Platform
Confidential - 8
Mono-DARPins®: Next generation biologics
• Tailor-made binding proteins derived from natural ankyrin
repeat proteins
• Fast and robust DARPin® generation process
• Selection of mono-DARPins® from large DARPin® libraries (display
technologies)
mono-DARPin®
• Versatile building blocks with pronounced class behavior
• Small size (14-18 kDa)
good tissue penetration
• High potency (<5-100 pM)
active at low concentration
• High stability
long shelf-life
• High solubility (> 100 mg/ml)
ideal drug properties
• Bacterial production (7-15g/L)
rapid and low-cost production
• PK properties (min – weeks)
adjustable to patient need
• Class behavior
high developability
DARPin inhibiting a target
Confidential - 9
Abicipar
Retinal diseases – Unmet medical need remains
• Large and growing market opportunity in wet agerelated macular degeneration (wet AMD) and diabetic
macular edema (DME)
• Annual sales in 2013: USD 6.0 billion1
• Expected sales in 2016: USD 8.4 billion (12% CAGR)2
wet AMD
Normal vision
• Standard of care drugs administered by frequent
E
injection into the eye
• 4 wk dosing3 for Lucentis, Avastin4
• 8 wk
dosing5
for Eylea
• Unmet medical need for new wet AMD/DME
F P
therapeutics with:
T O Z
• Less frequent injections
L P E D
• Greater vision gains
P E C F D
E
• Quality of life issues (travel, lost wages)
D
F
C
Z
P
F E L O P Z D
Source: 1 Represents 2013A global sales of Eylea® (USD 2.0 billion) and Lucentis® (USD 3.8 billion) and other products (USD 0.3 billion) in wet AMD and DME as reported by EvaluatePharma® a service of
Evaluate Ltd. (UK), www.evaluategroup.com, accessed [28 Aug.2014]; 2 EvaluatePharma® a service of Evaluate Ltd. (UK), www.evaluategroup.com, accessed [28 Aug.2014]; Estimated expected sales in
2016 do not include off-label use of Avastin; 3 PRN dosing under some European labels; 4 off-label use; 5 Dosing interval following 3 initial loading doses
Confidential - 11
Abicipar – VEGF blocker with potential for less
frequent dosing and higher vision gain
Abicipar
Product
•
Mono-DARPin® blocking VEGF
•
Collaboration with Allergan
•
Low double digit to mid-teen royalties and up to USD 375 million in additional
milestones
• USD 225 million in development and regulatory milestones, USD 150 million in
sales milestones
•
Tested in more than 240 patients to-date
Indications and markets
•
Wet AMD and DME
Phase and timing
•
Wet AMD: Initiation of pivotal Phase III study expected in Q2 2015
•
DME: Phase II study initiated in July 2014
Differentiation
•
Potential for less frequent injections than competitors (long intra-vitreal half-life)
•
Potential for higher efficacy and higher vision gain
Confidential - 12
REACH, Stage 3 – Abicipar data presented at ASRS
Abicipar
Change of best-corrected visual acuity (BCVA) 1,2
Summary of efficacy and safety data
Vision gain
Safety
(letters)
# AEs3 of ocular
WK16 WK20 inflammation
n = 23
n = 25
Abicipar 2.0mg
8.2
9.0
2
Abicipar 1.0mg
6.3
7.1
3
Lucentis 0.5mg
5.3
4.7
0
n = 16
Dosing:
Source: Allergan, 12 August 2014
1 Study not powered to reach statistical significance; 2 Mean visual acuity improvement from baseline (letters); 3 AE: adverse event; 4 BCVA: best corrected visual acuity
Confidential - 13
Strong support by Allergan and Actavis
“I think as you look at the Allergan products specifically, probably DARPin is the one that I don't think gets
enough credit and perhaps that's because it comes in at the ending of the planning period and we're looking at”
“When I was at Bausch & Lomb, we tried to buy DARPin as well and missed out on it to [Allergan and its CEO
David Pyott]. But this is an area that is growing; [AMD is] a horrific disease and if we can reduce the injection
burden for patients, then you really could have a potential blockbuster"
Brent Saunders, CEO Actavis, 17 November 2014
“Mr. Saunders praised Allergan's R&D pipeline […]. He was particularly impressed with Allergan's
ophthalmology portfolio, including the DARPin program for age-related macular degeneration (AMD)“
Mandy Jackson, Scrip Intelligence, 18 November 2014
“DARPin® could provide the next leap in treatment duration & has c. $ 20 billion in potential cumulative 10 year
sales (excl. dual-DARPin®)”
David Pyott, CEO Allergan, 30 June 2014
Confidential - 14
Multi-Benefit
DARPins in
Oncology
MP0250 – Multi-benefit DARPin® blocking VEGF
and HGF (Phase I, proprietary)
MP0250
Product
• Multi-DARPin®: blocking VEGF and HGF and binding to human serum albumin
• Molecular Partners has full commercial rights
Indications and markets
• Solid tumors such as liver, renal and gastric cancers
Phase and timing
• Clinical development started in July 2014 in solid tumors (est. recruitment of 54 patients)
• Initial top-line safety and efficacy data expected in 2015
Differentiation
• First biologic targeting both VEGF and HGF – blocking tumor growth and spreading
• Low toxicity of MP0250 expected to allow for combination with chemotherapy or tyrosine
kinase inhibitors (TKIs)
• Potential PFS (progression free survival) benefit vs. anti-VEGF mAbs
Confidential - 16
MP0250 data is equal or superior to clinical
standard of care in all tested PDX models1,2
MP0250
Performance of MP0250 vs. Sorafenib as monotherapy
Performance of MP0250 in combination with
Paclitaxel
PDX: Renal cancer
PDX: Gastric cancer
RXF 2264
GXA 3027
Source: Presented by the company at ASCO, 2014
1 Company preclinical data. Antitumor activity of MP0250, a bispecific VEGF- and HGF-targeting DARPin, in Patient-Derived Xenograft models (Abstract), ASCO, 2014; 2 MP0250 also tested in
preclinical models in liver cancer and lung cancer, which are not depicted above
Confidential - 17
MP0274 – Multi-benefit DARPin® with broad antiHER activity (proprietary)
MP0274
Product
• Multi-DARPin® blocking HER2 and HER3 signaling
• Molecular Partners has full commercial rights
Indications and markets
• In HER2+ patients including those with low levels of HER2 expression
Phase and timing
• In preclinical development
• Expected to enter clinical development in 2016
Differentiation
• Inhibits downstream-signaling of HER2 and HER3
• Potentially induces tumor apoptosis and blocks proliferation
• Potentially higher cancer killing of MP0274 than Herceptin®
Confidential - 18
MP0274 – Differentiation potential based on preclinical studies1
MP0274
Tumor cell apoptosis
• Higher cell-killing than Herceptin
and Perjeta in preclinical models
• Increased potency relative to
current HER2-targeted therapies
• Potential to benefit a broader
spectrum of cancer patients
Tumor volume
• Treatment for low HER2 expressers
• Improved treatment for HER2
overexpressers
Source: Company information
1 Company preclinical data. Antitumor activity of MP0274, anti-HER2 VEGF- and HGF-targeting DARPin
Confidential - 19
DARPins® with potential to go beyond –
Expanding our reach into emerging areas
• Molecular Partners explores approaches with
Immune checkpoint modulators
game-changing potential with DARPins® in
oncology
• Potential to address fields alone or with partners
• Immune-oncology as key focus area
• Immune checkpoint modulators (ICM) show
potential to revolutionize oncology (e.g. monotargeting PD-1)
• Multi-benefit DARPin® approach could have the
potential to unlock the complex ICM signaling
network
• DARPin®-toxin fusions
• DARPins® allow combination with different toxins
DARPin®-toxin conjugate
Confidential - 20
Financials
Confidential - 21
Financial highlights
CHF 322 million funding since inception
Financial highlights
• CHF 161 million collected from partners so far2
• CHF 160 million equity raised so far2
• Multiple years of cash runway with CHF 104.1
million IPO and CHF 99.0 million pre-IPO cash3
• Debt free balance sheet
• Operating Cash Flow positive since inception on a
accumulated basis
• Net profit positive in the last two audited years
(2012/2013)
• c.CHF 3.0 billion4 (USD 3.2 billion) combined
milestone potential and up to double-digit
royalties in all partnerships
1 VC
funding as net proceeds; IPO as gross primary proceeds; 2 up until June 30, 2014; 3 as of June 30, 2014; 4 Exchange rate as of 31 Dec 2014: CHF/USD: 1.0064; Assumes exercise of all options
for additional programs and attainment of all research option fees, preclinical milestones, development milestones and full commercial milestones for the maximum number of products
Confidential - 22
Financial Summary
CHF million
FY 2011
FY 2012
FY 2013
HY 20144
Revenues1
18.1
35.6
32.4
13.2
R&D expenses2
(17.7)
(18.2)
(21.8)
(9.8)
G&A expenses3
(3.1)
(3.0)
(3.5)
(1.9)
Net Profit (Loss)
(0.7)
13.3
7.1
1.5
Net cash from (used in) operations4
28.5
54.0
(13.6)
3.1
Cash & cash equivalents
64.2
113.2
96.1
99.0
1
Accounting revenues from collaboration agreements with Allergan, Janssen and Roche. Effective cash payments received amounted to CHF 145.8 million over this entire 3.5y period (includes
upfront fees, milestone payments, FTE payments and R&D expense re-imbursements); 2 Thereof non cash costs of CHF 1.1 million in FY2011, CHF 0.9 million in FY2012, CHF 0.8 million in FY2013 and
CHF 0.5 million in HY2014; 3 thereof non cash costs of CHF 0.6 million in FY2011, CHF 0.2 million in FY2012, CHF 0.1 million in FY2013 and CHF 0.2 million in HY2014; 4 includes payments of CHF 145.8
million over this entire 3.5y period from Allergan, Janssen and Roche (upfront fees, milestone payments, FTE payments and R&D expense re-imbursements); 4 Unaudited
Confidential - 23
Shareholder structure
• Listed in SIX Swiss Exchange (Ticker: MOLN)
since November 2014
• Included in Swiss Performance Index (SPI)
• 19.6 million shares outstanding
24%
25%
• 2.7 million outstanding stock options (at IPO)
• 24% Free Float
• Customary lock-up for Management, Board of
Directors (12m) and VCs (6m)
51%
Free Float (IPO + non-lockup)
VC's
Management, Board, Founders
Confidential - 24
Summary
A strong track record of financial and clinical
success
2015
IPO on SIX Swiss Exchange
2014
Start Phase I for MP0250
Phase IIb data for Abicipar
Extension of Roche alliance
2013
Phase I/II data of Abicipar in DME
Extension of Allergan and JNJ alliance
2012
Alliance with Allergan
2011
2010
Series B financing
Start Phase I for Abicipar
2009
Alliance with Janssen
Roche partnership and
Series A Financing
First clinical data for Abicipar
2008
Other milestones
2007
Clinical milestones
Source: Company information
Confidential - 26
Key near term value catalysts
• Start of Abicipar Phase III in wet AMD expected in Q2 2015
• Abicipar Phase II data from BAMBOO/CYPRESS study (establish comparability between Japanese
and non-Japanese patients) in wet AMD
• Abicipar Phase II data from PALM study in DME
• Topline safety and initial efficacy data for MP0250 in solid tumors expected in 2015
• Expect to start clinical development for MP0274 in 2016
• Several upcoming milestone events from partnerships
Confidential - 27
Key investment highlights
• Most advanced product candidate Abicipar partnered with Allergan expected to enter
Phase III development in Q2 2015 in wet AMD
• Broad pipeline of 4 DARPin® product candidates and several research programs targeting high
value indications
• Proprietary portfolio of multi-benefit product candidates in oncology with MP0250 in
Phase I development for solid tumors
• Partnerships with blue chip pharma companies: Roche (oncology), Allergan (ophthalmology) and
Janssen (immunology)
• Robust compound engine based on highly efficient DARPin® discovery and preclinical
development
• Entrepreneurial and collaborative management team who are pioneers in the DARPin® space
Confidential - 28
Molecular Partners AG
Wagistrasse 14
8952 Zürich-Schlieren
Switzerland
www.molecularpartners.com