Presentation Science Meets Business @ Health Valley From Molecule to Business 20150924

1. From Molecule to Business
Health Valley/SMB
September 24, 2015
Nijmegen
CriPec® Nanomedicines for Superior
Therapeutic Performance
Dr. Jeroen Tonnaer, CBO

2. 2
Introduction
• Pharmaceutical company developing nanomedicines
• Founded in April 2011
• Located in Maastricht, The Netherlands
• Headcount: 10 fte
• Private - VC funded:
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3. 3
CriPec® Nanomedicines
Improved efficacy and tolerability
CriPec® improves the therapeutic profile of drugs as a result of:
• Extended systemic circulation
• Improved disposition over the body
• Active or passive targeting to tissue, cellular and
molecular targets
• Tuneable and timely release
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4. CriPec® polymer
drug linker
entrapment of drug,
so inactive
release native drug &
degradation NP
coupling of drug
drug
linker
CriPec® polymer
incl. linker
drug lactic acid
Unique CriPec® Platform
Transiently stable nanomedicines
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PATENT PROTECTED BY 5 PATENT FAMILIES

5. range of therapeutic agents
(small molecules - peptides)
biodegradable and safe
fragments
multiple administration routes
controllable size
from 30 nm up to 100 nm
X
surface functionalisation for
cellular and molecular targeting
ease of manufacturing
customised drug release
Unique Features of CriPec®
Highly flexible nanomedicinal platform
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6. Therapeutic effect CriPec® Docetaxel
Breast xenograft model
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s.c. MDA-MB-231 xenografts in
nude mice (n=8±SD)
0 14 28 42 56
0
250
500
750
1000
1250
1500
single dose
vehicle
Taxotere 60 mg/kg
CriPec docetaxel 60 mg/kg
time (days)
tumourvolume(mm3
)
s.c. MDA-MB-231 xenografts in
nude mice (n=10±SD)
0 14 28 42 56
0
250
500
750
1000
1250
1500
1750
2000
vehicle
Taxotere 30 mg/kg
CriPec docetaxel 125 mg/kg
single dose
time (days)
tumourvolume(mm3
)

7. Unique Opportunity
• CriPec® is unique and competitive nanotechnology platform
that enables development of multiple (targeted) products
• CriPec® boosts efficacy and improves tolerability
• Applicable in disease areas with high unmet medical need
• First product started clinical development July 2015
• Attractive co-development potential
• CriPec® platform & products are protected by 5 patent
families
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8. Value Drivers
• Clinical
- Clinical phase I: safety of CriPec® docetaxel and PK profile
- Clinical phase IIa/IIb: efficacy of CriPec® docetaxel
• Platform
- In house PoC for CriPec® peptide, CriVac® antigen, and
CriPec® active targeting (ATN)
- A commercial viable, robust and generic cGMP-manufacturing process
• Business
- Successful feasibility studies for partners
- Successful (license) partnerships (full (co-)development programs)
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9. Pipeline
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indication discovery PoC preclinical phase I phase IIa
Proprietary programs
CriPec® docetaxel solid tumours
CriPec® dexamethasone inflammatory
CriPec® active targeting solid tumours
CriPec® oligonucleotides various
Collaborative programs
CriPec® peptides solid tumours
CriVac® antigens solid tumours
achieved ongoing

10. General BD&L Considerations
DO:
• Understand the candidate licensee’s strategy and needs
- Unlikely our opportunity will move the company’s strategy
- Look for gaps we can fill
- Ask for specific guidance on what the company is looking for
• Appeal to licensee’s core values, for example:
- Cutting-edge science
- Novel medicines that make a difference
• Demonstrate how our offering fits into their pipeline or
research framework
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11. General BD&L Considerations
DON’T:
• Forget that there is a fundamental difference between
research (which advances technology without being
constrained by business requirements) and product
development (which must satisfy customer needs with safe,
proven therapies that are competitive and cost-effective)
• Expect to successfully license technology with no proven
customer or market fit
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12. CriPec® Docetaxel
BD&L Considerations
• Concept of disruptive vs. incremental innovation
• Understanding stakeholders’ innovation preference
• Multinationals
• Regional players (e.g. Asian Pharma)
• Adopters (e.g. innovative generics manufacturers)
• Drafting CriPec® docetaxel licensing strategy
• Analysis of market gaps in oncology
• New application areas for CriPec® docetaxel
• Competitive landscape (USPs vs. taxotere & NM competition)
• Stakeholders landscape
• Valuation of CriPec® docetaxel
• Outline licensing deal
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13. www.cristaltherapeutics.com