20150924 smb cristal therapeutics jeroen tonnaer

S
From Molecule to Business
Health Valley/SMB
September 24, 2015
Nijmegen
CriPec® Nanomedicines for Superior
Therapeutic Performance
Dr. Jeroen Tonnaer, CBO
2
Introduction
• Pharmaceutical company developing nanomedicines
• Founded in April 2011
• Located in Maastricht, The Netherlands
• Headcount: 10 fte
• Private - VC funded:
2
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
3
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
4
PATENT PROTECTED BY 5 PATENT FAMILIES
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
5
Therapeutic effect CriPec® Docetaxel
Breast xenograft model
6
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
)
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
7
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)
8
Pipeline
9
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
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
10
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
11
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
12
www.cristaltherapeutics.com
1 of 13

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20150924 smb cristal therapeutics jeroen tonnaer

  • 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: 2
  • 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 3
  • 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 4 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 5
  • 6. Therapeutic effect CriPec® Docetaxel Breast xenograft model 6 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 7
  • 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) 8
  • 9. Pipeline 9 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 10
  • 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 11
  • 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 12