Corporate Presentation

Corporate Presentation
March 2017
© 2017 OvaScience, Inc. All rights reserved.
1
Forward-Looking Statements
This presentation includes forward-looking statements about the Company's plans for the OvaPrimeSM treatment, the OvaTureSM treatment and
the AUGMENTSM treatment, including the Company’s plans to increase its focus on OvaPrime and OvaTure; timing for the development process
involved with development and fertilization of bovine and human EggPCSM cell-derived eggs, and the receipt of related regulatory approvals; plans
to maintain the AUGMENT commercial footprint and slow the commercial expansion of AUGMENT; plans to continue to make AUGMENT available
to patients in partner clinics in Canada and Japan; plans to improve the Company’s cost structure through a corporate restructuring; plans to
extend the Company’s cash runway into the first quarter of 2019 without further financing; anticipated 2017 cash burn; anticipated cash items
and restructuring charges associated with the corporate restructuring; plans to reassess the ongoing and planned clinical studies of AUGMENT;
belief in the potential of the Company’s EggPC technology; plans to speak with the FDA in the first half of 2017; enrollment plans for, and timing of
enrollment and safety results from, the OvaPrime clinical study in Canada; plans to use the future data from ongoing OvaPrime studies to assess
the safety profile of the treatment, help define the patient population for the treatment and support OvaPrime commercial efforts; plans to
provide OvaPrime data in the future; plans to continue efforts in the OvaTure program relating to human egg maturation, characterization and
fertilization; size of the target markets for the Company’s treatments; and plans to fertilize a bovine EggPC cell-derived egg as proof of concept in
the OvaXonSM joint venture.
Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including
risks related to: the risks implicit in the development process of preparing bovine and human eggs for fertilization; regulatory risks associated with
obtaining authorization to fertilize human EggPC cells for research; the possibility that international IVF clinics that we work with may determine
not to provide or continue providing the AUGMENT treatment or OvaPrime treatment, or to delay providing such treatments, or to limit the
population of patients receiving the treatments based on clinical efficacy, safety or commercial, logistic, economic, available data, regulatory or
other reasons; challenges associated with enrolling and completing clinical trials, the science underlying our treatments (including the AUGMENT,
OvaPrime and OvaTure treatments), which is unproven; the risk that prior results with treatment of AUGMENT may not be replicated in future
treatments; scientific and regulatory challenges associated with characterizing and fertilizing an EggPC cell-derived egg; our ability to obtain
regulatory approval or licenses where necessary for our treatments; our ability to develop our treatments on the timelines we expect, if at all; our
ability to commercialize our treatments on the timelines we expect, if at all; as well as those risks more fully discussed in the "Risk Factors" section
of our most recently filed Quarterly Report on Form 10-Q and/or Annual Report on Form 10-K as filed with the Securities and Exchange
Commission. The forward-looking statements contained in the presentation reflect our current views with respect to future events. We anticipate
that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking
statements in the future, we specifically disclaim any obligation to do so. These forward-looking statements should not be relied upon as
representing our view as of any date subsequent to the date hereof.
© 2017 OvaScience, Inc. All rights reserved.
2
Agenda
 Company Background
 Our Markets
 Our Treatments
 Corporate
© 2017 OvaScience, Inc. All rights reserved.
3
Company Background
© 2017 OvaScience, Inc. All rights reserved.
4
Company Highlights
Novel Technology Platform
Large Market
New Frontier in Human
Reproductive Medicine
Discovery of mammalian egg precursor (EggPCSM) cells in 2004 provides new
options to improve female fertility
83 million women worldwide estimated to be infertile1
Portfolio of novel treatments offers significant advances for female fertility
R&D:
- OvaTureSM, in preclinical development, designed to eliminate hormones
from in vitro fertilization (IVF)
- OvaPrimeSM, in clinical development, developed to replenish egg reserve
Commercial:
- AUGMENTSM, autologous treatment designed to improve IVF success rates
Business Strategy
Focus on OvaTure and OvaPrime, while maintaining commercial footprint of
AUGMENT
Financial Position
$114 million in cash and short-term investments as of December 31, 2016;
sufficient to fund Company into 1Q19
Company estimates based on: 1 www.census.gov/popclock 2/23/17, Country Census Tables (2010-2015), Boivin 2007, Human Reproduction Vol. 22, No. 6 pp. 1510
© 2017 OvaScience, Inc. All rights reserved.
5
Our Mission
Transforming women’s fertility
Our Vision
We aspire to create a future
in which every woman
can have a child through
the innate power of her
own biology.
© 2017 OvaScience, Inc. All rights reserved.
6
OvaScienceSM: Built on Groundbreaking Science
2004
Discovery of
egg precursor
(EggPCSM) cells
in mice
Johnson, J. et al.
2009
Mouse oocytes
derived from
EggPC cells
generated in
vivo, resulting in
mouse live
births
Zou, et al.
2011
Core technology
licensed from
Harvard Medical
School and
Massachusetts
General Hospital
2012
Discovery of
EggPC cells
in humans
White, Y., et al.
Company founded
to translate the
groundbreaking
scientific discovery
of EggPC cells into
potential new
treatments for
female fertility
© 2017 OvaScience, Inc. All rights reserved.
7
From EggPCSM Cells to Treatments
Designed to
Eliminate
Hormones
from IVF
In vitro maturation
Mature Egg
ICSI*
Ovarian Cortex
Patient
Ovary
Pregnancy
Reintroduction
Hormone stimulation
Extract mitochondria
Hormone stimulation
Designed to
Replenish
Egg Reserve
EggPC Cells
ICSI
Live Birth
ICSI with mito transfer
Designed to
Improve IVF
Success
*ICSI: intracytoplasmic sperm injection
© 2017 OvaScience, Inc. All rights reserved.
8
Treatments Range From Preclinical to Commercial
Treatment
Preclinical
Clinical
Commercial*
Designed to
Eliminate
Hormones from IVF
Designed to
Replenish
Egg Reserve
Designed to
Improve IVF
Success
* The AUGMENTSM treatment
© 2017 OvaScience, Inc. All rights reserved.
is available in select international regions.
9
Our Markets
© 2017 OvaScience, Inc. All rights reserved.
10
Infertility Market
Worldwide Market
7.4B1
Target Markets*
Population
845M2
916M2
Women in reproductive age
132M2
83M3
Women estimated to be infertile
18M3
(20-42)
*Inclusive of Japan, US, Canada, Italy, Spain, France, Germany, UK, Australia, New Zealand, Benelux
Company estimates based on: 1 www.census.gov/popclock 2/23/17 2 Country Census Tables (2010-2015); 3 Boivin 2007, Human Reproduction Vol. 22, No. 6 pp. 1510
© 2017 OvaScience, Inc. All rights reserved.
11
Market Opportunities Among Multiple Conditions
Target Markets Population
Condition
7.4M1
Female infertility
(25-58% of infertile women are seeking
treatment)
2M2
(1.5% of women in reproductive age 20-42)
750K3
(10% of women seeking treatment)
550K4
Treatment
Designed to Eliminate
Hormones from IVF
Primary ovarian insufficiency
3.3M
Designed to Replenish
Egg Reserve
Diminished ovarian reserve
(32% of Assisted Reproductive
Technology cycles)
950K5
(57% of IVF*-ICSI**)
Failed one or more IVF cycle
Designed to Improve
IVF Success
Company estimates based on 1 NHS Statistics Report Number 73, Jan 2014, page 17; Women, married, infertility diagnosis 2 Greene 2007, J Assist Reprod Genet (2014) 31:935-946; Nelson LM. Clinical practice. Primary ovarian insufficiency. N
Engl J Med. 2009;360(6):606–14; Persani L, Rossetti R, Cacciatore C. Genes involved in human premature ovarian failure. J Mol Endocrinol. 2010;45(5):257–79 3 Greene 2014, J Assist Reprod Genet (2014) 31:935-946; Levi AJ, Reproductive
outcome in patients with diminished ovarian reserve. Fertil Steril. 2001;76(4):666–9 4 CDC.gov 2014 Annual ART Report page 21 5 CDC.gov 2013 ART Report, Figure 23 page 35 *in vitro fertilization **intracytoplasmic sperm injection
© 2017 OvaScience, Inc. All rights reserved.
12
In Vitro Fertilization (IVF) Market
$14.3B
2.6M Cycles
Total Market Size (Thousands of IVF Cycles, 2016)
Total Market Size (Millions of Dollars, 2016)
Middle East 167
U.S.
230
Japan
383
China
575
Canada 25
Italy 77
Spain 83
Rest of Asia 98
Turkey 98
Latin America/
South America 60
France 111
Rest of
Europe
392
U.S.
3,132
Middle East 767
1.2M
Rest of Asia 197
China
1,035
Turkey 373
Latin America/
South America 229
Rest of Europe
1,635
Canada 231
Italy 423
Germany 97
Spain 438
UK 75
Benelux 63
Aus/NZ 78
Benelux 14
$9B
Japan
1,991
Target Market
Aus/NZ 584
France 1,009
UK 576 Germany 876
Other Markets
Company estimates based on: SART; ESHRE (2012 – Present); ICMART; Country Registries; Latin America Registry; Israeli Ministry; Japan Society of Obstetrics and Gynecology; Allied Market Research report, 2014, 2015; Australia National Perinatal Epidemiology and Statistics Unit
2015; France agence de la biomedicine 2013; CATR Plus report Sep 12 2014, Canada CFAS (2012 - Present); UK Human Fertilisation and Embryology Authority (HFEA) 2013; Germany Reproduktionsmedizin und Endokrinologie, DIR Annual 2013 - German IVF-Registry; US CDC
Annual Report (2012-Present); Turkey & Other - L.E.K. Consulting; IVF aboard. Org; GLOBALSURGERYPROVIDERS.COM; Grand View Research In-Vitro Fertilization (IVF) 2015
© 2017 OvaScience, Inc. All rights reserved.
13
Our Treatments
© 2017 OvaScience, Inc. All rights reserved.
14
OvaTure
SM
Designed to Eliminate Hormones from IVF
OvaTureSM: Designed to Eliminate Hormones from IVF
OvaTure Treatment Procedure
In Preclinical Development
Patient ovary
Extracted
EggPCSM cells
Sperm
Ovarian Cortex
In vitro maturation
ICSI* egg fertilization
*ICSI: intracytoplasmic sperm injection
© 2017 OvaScience, Inc. All rights reserved.
16
Stages of Development from EggPCSM Cell to Embryo
Stage of Development
1
2
EggPC Cell
Cytoplasmic
Volume
 <10 microns
Structural
Characteristics
10µm
3
4
Immature Egg
Maturing Egg
5
Fertilization
 >35 microns
 >100 microns
 100-120 microns (human)
 120-150 microns (bovine)
 Zona pellucida
 Germinal vesicle
 Zona pellucida
 First polar body
 Two pronuclei
 Second polar body
 Euploid (normal number
chromosomes)
50µm
Embryo
Development
Targets
 Cleavage
 Blastocyst formation
50µm
Chromosomal
segregation
Functional Tests
 Positive BCB test (bovine and human)
 Parthenogenetic test (bovine only)
50µm
50µm
Asterisk
indicates
individual cell
As eggs mature, they begin to increase in cytoplasmic volume or size, and develop a thickened zona pellucida to protect the maturing egg and prevent fertilization from
multiple sperm. The nucleus enlarges and forms a germinal vesicle – which would then break down to allow the chromosomes to condense and separate, keeping half the
genetic material and the majority of the cytoplasm in the egg and placing the remainder in the polar body for disposal. At this point the egg would be ready to fertilize.
© 2017 OvaScience, Inc. All rights reserved.
17
Progress Made Growing EggPCSM Cells into Mature Eggs
Stage of Development
1
2
EggPC Cell
3
Immature Egg
4
Maturing Egg
5
Fertilization
Embryo
Development
Focus for 2017
Human & Bovine
 Robust process to
isolate EggPCs
Human & Bovine
 Robust process
to develop
immature eggs
Human & Bovine
 Observed
cytoplasmic
volume, zona
pellucida and
first polar body
 2 of 3 criteria
observed
simultaneously
in a single egg*
Bovine
 Fertilize bovine
EggPC cell-derived
egg
Not yet initiated
Human
 Optimizing process
to mature EggPC
cell-derived egg
 Working to secure
authorization to
fertilize for research
*Achieving sufficient cytoplasmic volume and a polar body (without zona pellucida) would be sufficient to attempt fertilization (Stanger, et al. Human Reproduction, 2001)
© 2017 OvaScience, Inc. All rights reserved.
18
Genetic and Morphological Criteria Consistent with a Maturing Egg
Genetic and Morphological Criteria
Cytoplasmic
volume
Human
Bovine
 Increase in cytoplasmic volume or size
Increased size
from 10 micron
to 120 micron
104µm
Increase size
from 10 micron
to 100 micron
Structural characteristics
Zona pellucida
Germinal vesicle
Chromosomal
segregation/first
polar body
 Thickened outer membrane that
protects the maturing egg and prevents
fertilization from multiple sperm
Zona pellucida
50µm
50µm
Condensed
Breakdown
Germinal vesicle
Separation
50µm
Condensed
Breakdown
First polar body
Separation
First polar body
Zona pellucida
50µm
© 2017 OvaScience, Inc. All rights reserved.
50µm
Germinal vesicle
 Enlarged nucleus of an egg before
meiotic division is completed
 Germinal vesicle breaks down to allow
chromosomes to condense and
separate, keeping half the genetic
material and the majority of the
cytoplasm in the egg, and placing the
remainder in the polar body for
disposal
Zona pellucida
Zona pellucida
50µm
19
Functional Tests Indicate Normal Maturation
Functional Tests
Positive Brilliant
Cresyl Blue (BCB)
test
 BCB is a blue dye which is metabolized
by glucose-6-phosphate
dehydrogenase
- In a cell that is still growing, BCB is
metabolized and the cell appears
clear
Human
Bovine
Developmentally
Competent
50µm
Developmentally
Incompetent
50µm
Developmentally
Competent
50µm
Developmentally
Incompetent
50µm
- In a cell that is no longer growing
and is mature, the cell remains blue
Parthenogenetic
activation
 Parthenogenic activation is the
chemical induction of egg cleavage,
and mimics the entry of sperm into
the egg
- A cell that is mature enough to be
fertilized is induced to divide into
two cells, then into four cells after
activation
 Test performed in bovine only
© 2017 OvaScience, Inc. All rights reserved.
Asterisk indicates
individual cell
50µm
Not performed in human
50µm
Multiple
cells identified after
chemical induction
20
OvaTureSM Milestones
2017
2018
2019
Fertilization of bovine EggPCSM cell-derived egg
Bovine pregnancy
Bovine
Embryo transfers
Bovine live birth from EggPC cell-derived egg
Six-month bovine
follow-up
Mature human EggPC cell-derived egg (for research)
Human
Authorization and fertilization of EggPC cell-derived egg (for research)
Submit for regulatory approval (clinical study)
© 2017 OvaScience, Inc. All rights reserved.
21
OvaXonSM: Proof of Concept for OvaTureSM
 Joint venture with Intrexon® established in 2013
 R&D costs and net financial results shared equally
 Focused on making significant improvements in human and animal health using
EggPCSM cell technology and Intrexon’s platform
 Status: Study ongoing to fertilize bovine EggPC cell-derived egg
“This EggPC platform therefore may substantially increase the availability of eggs from female cattle
thereby increasing the power of genetic selection to improve productivity in the dairy and beef industries.”
– Andrew Last, Chief Operating Officer, Intrexon (March 1, 2017)
© 2017 OvaScience, Inc. All rights reserved.
22
OvaPrime
SM
Designed to Increase Egg Reserve
OvaPrimeSM: Designed to Increase Egg Reserve
OvaPrime Treatment Procedure
In Clinical Development
Patient ovary
Extracted
EggPCSM cells
EggPC cells are injected into ovary;
patient undergoes standard IVF cycle
Ovarian Cortex
© 2017 OvaScience, Inc. All rights reserved.
24
OvaPrimeSM Process Per Patient
Month
1
2
3
4
5
6
7
8
9
10
11
12
Baseline tests – AMH, FSH, E2 and ultrasound
Scheduling/Performing Biopsy
SM
Reintroduction of EggPC
cells
Monthly Ovarian Monitoring: ultrasound and blood tests evaluating AMH, FSH and E2
Ovarian Hyperstimulation
Occurs after observation of 5-10 basal follicles
Egg Retrieval
Egg Fertilization
PGD*
Embryo Transfer
Monthly visit
*Preimplantation Genetic Diagnosis
© 2017 OvaScience, Inc. All rights reserved.
Pregnancy Test
25
OvaPrimeSM Clinical Trial
Single Center, Prospective, Blinded, Randomized (Ovaries), Controlled Company Sponsored Trial
Number of Subjects: 70
Key Inclusion Criteria:1
 Women with diminished ovarian reserve (DOR) who meet two of the following criteria2
- ≥40 years old
- A previous IVF cycle with ≤3 eggs with standard hyperstimulation
- An abnormal ovarian reserve test
 Women with primary ovarian insufficiency (POI) who meet the following criteria
- <40 years old
- Non-detectable AMH
- FSH >15 mIU/ml or symptoms of menopause (or both)
Control Procedure: Subjects will receive injection of EggPCSM cells into one ovary (based on a pre-defined randomization
schedule), and EggPC suspension fluid into the contralateral ovary (control ovary)
Primary Endpoint: To evaluate safety of all subjects regardless of pregnancy outcome
Secondary Endpoint: To assess changes in hormone levels and follicular development
Status: Enrollment ongoing
1 Full
inclusion criteria available on www.clinicaltrials.gov
© 2017 OvaScience, Inc. All rights reserved.
2
Based on European Society of Human Reproduction and Embryology (ESHRE) Guidelines
26
OvaPrimeSM Milestones
2017
2018
2019
2020
Complete enrollment of 70 patients
Clinical
Complete biopsies in all patients
Complete reintroduction in all patients
Complete embryo transfers in all patients
Last potential birth
12 months post birth monitoring
Initial readout: 6-months of post-EggPCSM reintroduction safety data for first 20 patients
Data
Initial presentation of 6-months post-EggPC reintroduction data for first 20 patients
Initial readout: 6-months of post-EggPC reintroduction safety
data for all patients
Initial readout: embryo transfers
of all patients
Final readout
© 2017 OvaScience, Inc. All rights reserved.
27
AUGMENTSM
Designed to Improve IVF Success
AUGMENTSM: Designed to Improve IVF Success
AUGMENT Treatment Procedure
Patient ovary
Extracted
EggPCSM cells
EggPC cell
Extracted
autologous
mitochondria
Inject mitochondria
into egg during
fertilization by ICSI*
Sperm
Ovarian Cortex
The AUGMENTSM treatment is not available in the U.S. *ICSI: intracytoplasmic sperm injection
© 2017 OvaScience, Inc. All rights reserved.
29
AUGMENTSM Results vs. Patient’s IVF History1
UAE
Canada
ICSI*
AUGMENT Treatment
% Success per Cycle
22%
4%
2%
9/257
prior
cycles
Pregnancy
4/257
prior
cycles
Live Birth




1Updated
18%
13/60
AUGMENT
11/60
cycles
AUGMENT
cycles
Pregnancy
Live Birth
59 patients
Average age: 37.3
4.3 average prior failed IVF cycles
257 total prior cycles
% Success per Cycle
ICSI*
AUGMENT Treatment
35%
12/34
cycles
26%
9/34
cycles
11%
8/71
prior
cycles
1.4%
1/71
prior
cycles
Pregnancy
Live Birth




Pregnancy
Live Birth
34 patients
Average age: 36.0
2 average prior failed IVF cycles
71 total prior cycles
data on live birth rates. Fakih, M. et al. Journal of Fertilization: In Vitro, IVF-Worldwide, Reproductive Medicine, Genetics & Stem Cell Biology, 2015 *ICSI: intracytoplasmic sperm injection
© 2017 OvaScience, Inc. All rights reserved.
30
AUGMENTSM Status
 Safety and efficacy supported by published, retrospective analysis1
 Offered at partner clinics in Canada and Japan
 First births in Japan in 1Q17
 Scheduled to meet with FDA in 1H17 to explore potential entry into U.S. market
1Fakih,
M. et al. Journal of Fertilization: In Vitro, IVF-Worldwide, Reproductive Medicine, Genetics & Stem Cell Biology, 2015
© 2017 OvaScience, Inc. All rights reserved.
31
Corporate
© 2017 OvaScience, Inc. All rights reserved.
32
Financials
Revenue
Cost of Revenue
R&D
SG&A
Restructuring
Net Loss
Operating cash burn
One-time cash expenditures resulting
from restructuring
Cash on-hand (no debt)
© 2017 OvaScience, Inc. All rights reserved.
Q416
2016
$121K
$1.4M
$653K
$5.4M
$4.7M
$10.9M
$5.4M
$21.6M
$49.2M
$5.4M
$(22.6)M
$(82.3)M
$16.6M
$66.2M
2017
Increase as % of
total costs
Absolute Decrease
$45M - $50M
$5.7M - $6.5M
over 2017 and 2018
$114.4M
$114.4M
Cash into 1Q19
33
Patent Portfolio
 Comprehensive IP portfolio; patent protection into 2035
─ 58 issued patents in 44 countries
─ Over 150 pending applications pending in more than 130 countries
EggPCSM (2025)



EggPC cells
EggPC isolation methods
OvaPrimeSM and OvaTureSM
related methods
Anti-VASA mAb (2035)


mAb variants
EggPC isolation methods
(AUGMENTSM, OvaPrime and
OvaTure)
AUGMENT Compositions &
Methods (2032-2035)
Platform Patents
Issued Patent(s)
Pending Application(s)
© 2017 OvaScience, Inc. All rights reserved.
34
Key Catalysts and Milestones
1Q17
2Q17
3Q17
4Q17
2018
Fertilize bovine EggPCSM cell-derived egg
Bovine embryo transfer
Mature human EggPC cell-derived egg (for research)
Receive authorization to fertilize and fertilize human EggPC cell-derived egg (for research)
Complete enrollment of 70 patients
Complete biopsies in all patients
Complete reintroduction in all patients
Initial data readout*
Initial presentation of data**
Engage with FDA
Reassess ongoing IVI-sponsored and planned multi-center clinical studies
Continue offering treatment at existing Canadian and Japanese partner clinics
*Initial
readout will include 6 month post EggPC cell reintroduction safety data from first 20 patients, as well as efficacy data, as measured by follicular development and changes in patients hormone levels.
presentation of data will include 6 month post EggPC cell reintroduction safety data from first 20 patients, as well as efficacy data, as measured by follicular development and changes in patients hormone levels.
**Initial
© 2017 OvaScience, Inc. All rights reserved.
35
Company Highlights
Novel Technology Platform
Large Market
New Frontier in Human
Reproductive Medicine
Discovery of mammalian egg precursor (EggPCSM) cells in 2004 provides new
options to improve female fertility
83 million women worldwide estimated to be infertile1
Portfolio of novel treatments offers significant advances for female fertility
R&D:
- OvaTureSM, in preclinical development, designed to eliminate hormones
from in vitro fertilization (IVF)
- OvaPrimeSM, in clinical development, developed to replenish egg reserve
Commercial:
- AUGMENTSM, autologous treatment designed to improve IVF success rates
Business Strategy
Focus on OvaTure and OvaPrime, while maintaining commercial footprint of
AUGMENT
Financial Position
$114 million in cash and short-term investments as of December 31, 2016;
sufficient to fund Company into 1Q19
Company estimates based on: 1 www.census.gov/popclock 2/23/17, Country Census Tables (2010-2015), Boivin 2007, Human Reproduction Vol. 22, No. 6 pp. 1510
© 2017 OvaScience, Inc. All rights reserved.
36
© 2017 OvaScience, Inc. All rights reserved.
37