1. Pharma interactions: 25
Chagas KOL interactions: 25
Veterinary interactions: 20
Diagnostic interactions: 5
TOTAL interactions: 75
December 10, 2013

2. Mentors:
Steve Richards
Karl Handelsman
Jair L. Siqueira-Neto, PhD
UCSF Specialist, Kinetoplastid Core Director
Patrick Symmonds
UCSF/UC Berkeley Translational Medicine Candidate
Paul Novick
Stanford PhD Candidate
Susanna Wen
Stanford PhD Candidate

3. INSPIRATION
Identifying/Defining the Pain
1 million
250 K
10 K
8-10
million
10 K

4. Business Model Canvas
WEEK 1

5. Ecosystem: Wk1
Direct sales to
pet owners
IP for
human
mkt
Initial
Funding
IP to
developing
countries
Pharma
(human; developed country)
Traveler’s Medicine
Initial
Funding
Initial
Funding
Military
Initial
Funding
IP for
human
mkt
Endemic
Country Gov’t
(in endemic areas)
END USER

6. Ecosystem: Wk1
Direct sales to
pet owners
IP for
human
mkt
Initial
Funding
IP to
developing
countries
Pharma
(human; developed country)
Traveler’s Medicine
Initial
Funding
Initial
Funding
Military
Initial
Funding
IP for
human
mkt
Endemic
Country Gov’t
(in endemic areas)
END USER

7. Ecosystem: Wk10
Access to
cmpd libraries
PARTNER
IP for
human
mkt
PARTNER: Human Pharma
END USER
PR
IP to
developing
countries
PR
Initial
Funding
~$2-$3mil
to s
s rie
s a
ce libr
Ac d
p
cm
PARTNER:
Dx Company
Increase value
of product
Create mkt
Initial
Funding
~$600,000
IP
Initial
Funding
~$1-$2mil
IP for
human
mkt
PARTNER
Endemic
Country
Gov’t
for
An
im
Co
al
Ma
lla
bo
rk e
$2
t
0-3 rat
0m ion
il
PARTNER:
Vet Pharma

8. PRODUCT
Chemotherapy vs Vaccine
VS
● Could repurpose existing drug
● Existing pharma infrastructure
● Easily distributed
● Lifetime immunity
● Fewer doses

9. PRODUCT
Chemotherapy vs Vaccine
VS
● Could repurpose existing drug
● Existing pharma infrastructure
● Easily distributed
Funder Preference
● Lifetime immunity
● Fewer doses
Scientifically Risky

10. MARKET
Developed vs Developing World
Developing
● Majority of patients found here
● High awareness & treatment
● PR for developed country-based
pharma
Developed
● Human market would drive value of drug
● Traveller/tourist market
● Military use

11. MARKET
Developed vs Developing World
Developing
Developed
● Majority of patients found here
● Human market would drive value of drug
● High awareness & treatment
● Traveller/tourist market
● PR for developed country-based
● Military use
pharma
Lack of approved Dx & low
Developing country gov’t as customer
awareness in primary care MD

12. Veterinary Market
● Peripheral/supplementary market
● Hampered by low awareness
● Infection rates uncertain

13. Veterinary Market
● Peripheral/supplementary market
● Hampered by low awareness
● Infection rates uncertain (~5-15%)
Main driver of company value

14. Business Model Canvas
WEEK 5

15. KEY PARTNERS
Sources of Non-dilutive Funding
Generating Awareness to Build Market
R&D Collaborator
Bringing Product to Market

16. Business Model Canvas
WEEK 10

17. Strategy
2014
Q1
Q2
Q3
$ 800 K
2015
Q4
Q1
Q2
Q3
2016
Q4
$ 2.6 M
Q1
Q2
Q3
2017
Q4
Q1
Q2
Q3
2018
Q4
Q1
Q2
Q3
TAM = $500 Million
$ 3.6 M
SAM = $100 Million
Vet Clinical Trials
$ 1.0 M
Discovery
(Screening)
Development
(Lead Opt & DMPK-Tox)
PCC
Human Clinical Trials
$ 100 M
$ 600 K
Q4
$ 3.8 M
$ 2.6 M
SAM = $20 Million

18. Ecosystem: Wk10
PARTNER
PARTNER
Access to
cmpd libraries
IP for
human
mkt
END USER
PR
IP to
developing
countries
PR
Initial
Funding
~$2-$3mil
to s
s rie
s a
ce libr
Ac d
p
cm
PARTNER
Increase value
of product
Create mkt
Initial
Funding
~$600,000
IP
Initial
Funding
~$1-$2mil
IP for
human
mkt
PARTNER
Endemic
Country
Gov’t
for
An
im
al
Co
lla
$2 bor
0-3 at
0m ion
il
Ma
rk e
t
CUSTOMER

19. INVESTOR READINESS
“Proof of Relevance” Path to Revenue Fully Articulated
High Fidelity MVP
Business Case Defined
“Left Side of the Canvas”
Build KOL network
Deeper IP review and strategy
Due diligence on partners
Deeper cost analysis to each data point
Team talent gaps identified
“Right Side of the Canvas”
Product Market Fit
Problem/Solution Validation
Low Fidelity MVP
First Pass Canvas
IRL6

22. Multi-Sided Market
Philanthropic Organizations
Endemic Countries (Gov)
Patient
Heart Failure
Life Threatening
Global Health; Reduce Care Costs
$
Pet well-being
Life Threatening
$
Pet Owner
Pet well-being
Life Threatening
$
Veterinary

23. NEXT STEPS
Eric Chatelain
Robert Don
Bernard Pécoul
Head of
Drug Discovery
Discovery &
Pre-Clinical Director
Discovery &
Executive Director
Talk to KOLs and apply for funding

24. Business Model Canvas
WEEK 1

25. Finance Operations
Year 1
Year 2
Year 3
Discovery Phase
$ 300 K
$ 400 K
$0
Hit to Lead
$ 200 K
-
-
Lead Optimization (exploratory SAR)
$ 100 K
$ 400 K
-
$0
$ 1.7 mil
$ 2 mil
in vivo studies
-
$ 200 K
$ 2 mil
Matter synthesis & Scale up
-
$ 1 mil
-
Formulation
-
$ 500 K
-
Others (People + Space + etc)
$ 500 K
$ 500 K
$ 500 K
TOTAL
$ 800 K
$ 2.6 mil
$ 2.5 mil
Development Phase

26. Finance Operations
Year 1
Year 2
Year 3
Total Cost
$ 800 K
$ 2.6 mil
$ 2.5 mil
Funding
$ 800 K
$ 2.6 mil
$ 3.6 mil
NIH & similiar (US Gov’t funding)
$ 300 K
$ 600 K
$ 600 K
DNDi
$ 500 K
$ 1 mil
$ 1 mil
Gates/Carlos Slim Foundation
-
$ 500 K
$ 1 mil
Brazilian Gov’t Partner
-
$ 500 K
$ 1 mil
Vet Pharma Partner
-
-
end of yr
$0K
$0K
$ 1.1 mil
WORKING CAPITAL
Viable business via non-dilutive funding!

27. Veterinary Market Size
TAM = $500 Million
50 million dogs in
endemic regions
SAM = $100 Million
10 million dogs in
USA endemic states
SAM = $20 Million
20% of the dogs
diagnosed and treated

29. Revenue model
Norm:
<
Investment
>
Revenue

30. Cost Breakdown
Research
Experiment
Preclinical
Cost
Experiment
Clinical
Cost
$100 k
in vivo PK (mice, dogs)
$500 k
$1 mil
in vivo tox. (fertility,
$2 mil
Lead optimization
$500 k
teratogenicity, carcin, etc)
in vitro PK
$500 k
Prelim. formulation
Hit/Lead identification
Human vet exposure
in vitro tox (AMES,
HERG, etc)
Rodent efficacy model
TOTAL
$500 k
$0 - 100k
$100 k
$2,200,000
($200,000)
TOTAL
$3,100,000
(< $600,000)

31. Metrics that Matter
Unit
Quantity
Total
$ 1 / compound
100K compounds
$ 100K
$ 1K / series
10 scaffolds
$ 10K
Lead Optimization
$ 500K / series
2 series
$ 1 mil
In vitro PK
$ 100K / series
2 series
$ 200K
In vivo PK & Toxicology
$ 100K / series
1 series
$ 100K
In vivo PD (rodent efficacy)
$ 100K / molecule
2 molecules
$ 200K
GMP Synthesis & scale up
$ 1 mil / molecule
1 molecule
$ 1 mil
In vivo Pre-clinical package
$ 2 mil / molecule
1 molecule
$ 1 mil
Formulation
$ 500K / molecule
1 molecule
$ 500K
Screening & Hits identification
Hit-to-Lead
IP
$ 100K
$ 100K
People (4 scientists)
$ 320K / year
3 years
$ 960K
Space & Infrastructure
$ 200K / year
3 years
$ 600K
TOTAL
~$ 6 mil

32. Key Partner Hypotheses
Partner
Why do we need?
Why will they
partner?
Cost of
partnership?
DNDi
Funds and network for
preclinical and clinical research
Currently looking for
additional anti-parasitic
Chagas scaffolds
IP for human patients in
developing nations
NIH, foreign similars
Need funds for preclinical and
clinical research
History of funding Chagas
disease research
None
Vet pharma companies
Distribution of therapy,
potential acquirer
Improve product pipeline
IP for animal market in
developed countries
Human pharma
companies
Access to compound libraries,
screening resources
History of partnerships in
Chagas disease research
Potential IP for human
partnerships developing
nations, developed nations
Diagnostics companies
Increased awareness and
diagnosis of disease
Opportunity to sell more
diagnostics
Exclusive partnership?
Academic epidemiology
researchers
Increased awareness and
market size validation
Looking for Chagas therapies
in the marketplace
None
Universities/CROs
R&D resources and manpower
Fee for service relationships
Price of R&D efforts
FDA
Insight into exactly what is
needed in IND package
Orphan, Breakthrough, etc
designations
None

33. Learning about partners
Partner
Hypothesis
Experiment
Result
DNDi
Our current
chemotherapeutics are an
attractive scaffold for DNDi
Data review with DNDi
influencers/directors
One of our projects is likely
fundable, and are desperate
for more pipeline projects
NIH, foreign similars
Our research projects are
attractive funding projects
Discussions with focus
group heads, applied for
SBIR
A promising Chagas
chemotherapeutic is a
fundable project
Vet pharma companies
Vet pharma would be
interested in licensing a
Chagas therapeutic
Discussions with Biz Dev
employees
Depends on prevalence/
market size, and which
company
Human pharma
companies
Big pharma will provide
research resources
Discussions with external
partnership coordinators
Compound libraries,
screening resources, etc
could be donated
Diagnostics companies
Interested in partnership with
therapeutics company
Attempted interviews with
4 quick test producers
Still scheduling interviews
Academic epidemiology
researchers
Can firmly estimate the
prevalence of Chagas disease
in canines
Discuss with leading
researchers
Not a consensus on how
prevalent disease is
Universities/CROs
These partners will operate on
a fee for service basis
Interviews and quotes for
projects
These are reliable,
inexpensive research option
FDA

34. ASTMH Conference Notes
● DNDi is interested
o Currently reviewing data internally
o Profile of project matches what they are looking for
● GSK is ready to partner
o Analogue screening can begin soon
o Encouraging us to apply to Open Lab Foundation
● Neither would be interested in retaining vet IP, developed world IP
o GSK might not even want Brazil, Mexico (WIPO guidelines)
● US vet market may be more primed than previously thought
o Higher awareness than we believed
o Louisiana, in addition to Texas (and likely other states)

35. Early Stage Cash Flow
Year 1
Year 2
Year 3
NIH & similar
$300k
$300k
$1 million
DNDi
$500 k
$1 million
$1 million
Vet Pharma Partnership
end of year
Total Revenue
$800k
$1.3 million
$2 million
G&A
$300k
$400k
$500k
Rodent IND Study
$400k
Large Mammal IND Study
$1.3 million
Formulation & Manufacture
$400k (?)
$400k (?)
Total Costs
($700 k)
($1.2 million)
($1.8 million)
Working Capital
$100k
$200k
$400k

36. Veterinary Market
Vet. Pharma
● Min market size: $5-8mil annually (no risk, must produce results <6mo)
● IP: only license animal health rights
● What is the demand? Profit margin?
Veterinarians
●
●
●
●
Difficult to assess incidence & prevalence of Chagas - low awareness
AVMA: guidelines for recommended vaccines & treatments for animals
Currently no FDA-approved treatment options available
Modes of transmission - congenital seems to be a big problem
Diagnostic
● Collaboration/partnership? (est $30-50K to develop simple Dx w/ vet pharma)

37. Human Market
Pharma Companies
● Chagas chemotherapeutic DOES represent acquisition target
● DNDi TPP is basis of most company’s TPPs
Clinicians
●
●
●
●
●
Who pays for Chagas?
Need a better active infection Dx
High prevalence in select US populations
Poor compliance (toxicity)
Need to increase awareness among 1’ care and ID doctors
○ Often too late to treat by the time cardiologists see patients
Insurance Companies
● Provide data showing superiority to benznidazole
● Physician required to submit positive tests before authorization of charge