christopher boyer, executive director,...
TRANSCRIPT
August 10 2018
wwwbio-linkcom108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Director Bio-Link
De-risking preclinical assets with early human insights recent advances in human tissue and disease models
August 10 2018
Bio-Link
o Professional business development and corporate advisory services
o Focused on facilitating commercial partnerships in the biopharma diagnostics nutraceuticals and medical devices industries
o Privately owned Sydney and Melbourne offices multi-disciplinary team of 10 employees
o Australian and international clients include biotech and medical device companies and medical research institutions
August 10 2018
Consulting and Technology AnalysisStrategic reviews competitive landscapes
IP development plans
Proactive Business DevelopmentFocused on identifying engaging with and closing deals with strategic partners on optimum terms
Scouting Targeted scouting and triage of
partnering and in-licensing opportunities
Project ManagementCoordination of commercially focused RampD collaborations
Sourcing of FundingFacilitating access to funding grants (eg EP NHMRC NIH SBIR MJFF NIH TRND) high net worth investors venture capital
Commercialisation services
August 10 2018
Type of investment Average returnyear Risk
Property 8 Medium
Shares 8 Medium
Bonds 6 Low
Savings 28 Very low
Biotech Equity 25 High
Source Canstar Investment AUS 2018 ForbesThe Biotech Venture Capital Math Problem
Biotech one of the best returns
hellip and one of the highest risks
August 10 2018
3 main business models in biotech
3rd type Big Pharma
bull Major buyers bull Lots of cashbull Many assetsbull Late stage assetsbull Bring to market
2nd type Medium-sized Pharma bull Buy cheap opportunistic bull 6-20 assetsbull Early ndash mid stage Clinical stagebull Sells to Big Pharma
eg Morphosys Mallinckrodt Pierre Fabre Molnlycke
1st type Small biotech
bull Early stagebull 1-3 assetsbull Little cashbull Tries to sell ASAP
Sell for $
Sell for $$$
Aussie Biotech- Seller-
Pharma- Buyer-
August 10 2018
Lead Optimisation
In vivoPD
ADME TOXIND-enabling
Phase 1 Phase 2-3NDA
approval Market
RISK VALUE
Earliest Pharma buysWhere you want to sell
Pharma- Buyer-
Aussie Biotech- Seller-
Looking for best return on investment
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Typical chat with PharmahellipHey Pharma we have great in vivo data massive indication wanna license
Interesting cool approach right up our alleyhellip and wersquore totally prepared to jump in early
Doesnrsquot seem to hurt the mice
Hmmn pityhellip just a bit too risky
What about tox
Come on matehellip we canrsquot afford that
ADME Tox package
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Bio-Link
o Professional business development and corporate advisory services
o Focused on facilitating commercial partnerships in the biopharma diagnostics nutraceuticals and medical devices industries
o Privately owned Sydney and Melbourne offices multi-disciplinary team of 10 employees
o Australian and international clients include biotech and medical device companies and medical research institutions
August 10 2018
Consulting and Technology AnalysisStrategic reviews competitive landscapes
IP development plans
Proactive Business DevelopmentFocused on identifying engaging with and closing deals with strategic partners on optimum terms
Scouting Targeted scouting and triage of
partnering and in-licensing opportunities
Project ManagementCoordination of commercially focused RampD collaborations
Sourcing of FundingFacilitating access to funding grants (eg EP NHMRC NIH SBIR MJFF NIH TRND) high net worth investors venture capital
Commercialisation services
August 10 2018
Type of investment Average returnyear Risk
Property 8 Medium
Shares 8 Medium
Bonds 6 Low
Savings 28 Very low
Biotech Equity 25 High
Source Canstar Investment AUS 2018 ForbesThe Biotech Venture Capital Math Problem
Biotech one of the best returns
hellip and one of the highest risks
August 10 2018
3 main business models in biotech
3rd type Big Pharma
bull Major buyers bull Lots of cashbull Many assetsbull Late stage assetsbull Bring to market
2nd type Medium-sized Pharma bull Buy cheap opportunistic bull 6-20 assetsbull Early ndash mid stage Clinical stagebull Sells to Big Pharma
eg Morphosys Mallinckrodt Pierre Fabre Molnlycke
1st type Small biotech
bull Early stagebull 1-3 assetsbull Little cashbull Tries to sell ASAP
Sell for $
Sell for $$$
Aussie Biotech- Seller-
Pharma- Buyer-
August 10 2018
Lead Optimisation
In vivoPD
ADME TOXIND-enabling
Phase 1 Phase 2-3NDA
approval Market
RISK VALUE
Earliest Pharma buysWhere you want to sell
Pharma- Buyer-
Aussie Biotech- Seller-
Looking for best return on investment
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Typical chat with PharmahellipHey Pharma we have great in vivo data massive indication wanna license
Interesting cool approach right up our alleyhellip and wersquore totally prepared to jump in early
Doesnrsquot seem to hurt the mice
Hmmn pityhellip just a bit too risky
What about tox
Come on matehellip we canrsquot afford that
ADME Tox package
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Consulting and Technology AnalysisStrategic reviews competitive landscapes
IP development plans
Proactive Business DevelopmentFocused on identifying engaging with and closing deals with strategic partners on optimum terms
Scouting Targeted scouting and triage of
partnering and in-licensing opportunities
Project ManagementCoordination of commercially focused RampD collaborations
Sourcing of FundingFacilitating access to funding grants (eg EP NHMRC NIH SBIR MJFF NIH TRND) high net worth investors venture capital
Commercialisation services
August 10 2018
Type of investment Average returnyear Risk
Property 8 Medium
Shares 8 Medium
Bonds 6 Low
Savings 28 Very low
Biotech Equity 25 High
Source Canstar Investment AUS 2018 ForbesThe Biotech Venture Capital Math Problem
Biotech one of the best returns
hellip and one of the highest risks
August 10 2018
3 main business models in biotech
3rd type Big Pharma
bull Major buyers bull Lots of cashbull Many assetsbull Late stage assetsbull Bring to market
2nd type Medium-sized Pharma bull Buy cheap opportunistic bull 6-20 assetsbull Early ndash mid stage Clinical stagebull Sells to Big Pharma
eg Morphosys Mallinckrodt Pierre Fabre Molnlycke
1st type Small biotech
bull Early stagebull 1-3 assetsbull Little cashbull Tries to sell ASAP
Sell for $
Sell for $$$
Aussie Biotech- Seller-
Pharma- Buyer-
August 10 2018
Lead Optimisation
In vivoPD
ADME TOXIND-enabling
Phase 1 Phase 2-3NDA
approval Market
RISK VALUE
Earliest Pharma buysWhere you want to sell
Pharma- Buyer-
Aussie Biotech- Seller-
Looking for best return on investment
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Typical chat with PharmahellipHey Pharma we have great in vivo data massive indication wanna license
Interesting cool approach right up our alleyhellip and wersquore totally prepared to jump in early
Doesnrsquot seem to hurt the mice
Hmmn pityhellip just a bit too risky
What about tox
Come on matehellip we canrsquot afford that
ADME Tox package
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Type of investment Average returnyear Risk
Property 8 Medium
Shares 8 Medium
Bonds 6 Low
Savings 28 Very low
Biotech Equity 25 High
Source Canstar Investment AUS 2018 ForbesThe Biotech Venture Capital Math Problem
Biotech one of the best returns
hellip and one of the highest risks
August 10 2018
3 main business models in biotech
3rd type Big Pharma
bull Major buyers bull Lots of cashbull Many assetsbull Late stage assetsbull Bring to market
2nd type Medium-sized Pharma bull Buy cheap opportunistic bull 6-20 assetsbull Early ndash mid stage Clinical stagebull Sells to Big Pharma
eg Morphosys Mallinckrodt Pierre Fabre Molnlycke
1st type Small biotech
bull Early stagebull 1-3 assetsbull Little cashbull Tries to sell ASAP
Sell for $
Sell for $$$
Aussie Biotech- Seller-
Pharma- Buyer-
August 10 2018
Lead Optimisation
In vivoPD
ADME TOXIND-enabling
Phase 1 Phase 2-3NDA
approval Market
RISK VALUE
Earliest Pharma buysWhere you want to sell
Pharma- Buyer-
Aussie Biotech- Seller-
Looking for best return on investment
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Typical chat with PharmahellipHey Pharma we have great in vivo data massive indication wanna license
Interesting cool approach right up our alleyhellip and wersquore totally prepared to jump in early
Doesnrsquot seem to hurt the mice
Hmmn pityhellip just a bit too risky
What about tox
Come on matehellip we canrsquot afford that
ADME Tox package
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
3 main business models in biotech
3rd type Big Pharma
bull Major buyers bull Lots of cashbull Many assetsbull Late stage assetsbull Bring to market
2nd type Medium-sized Pharma bull Buy cheap opportunistic bull 6-20 assetsbull Early ndash mid stage Clinical stagebull Sells to Big Pharma
eg Morphosys Mallinckrodt Pierre Fabre Molnlycke
1st type Small biotech
bull Early stagebull 1-3 assetsbull Little cashbull Tries to sell ASAP
Sell for $
Sell for $$$
Aussie Biotech- Seller-
Pharma- Buyer-
August 10 2018
Lead Optimisation
In vivoPD
ADME TOXIND-enabling
Phase 1 Phase 2-3NDA
approval Market
RISK VALUE
Earliest Pharma buysWhere you want to sell
Pharma- Buyer-
Aussie Biotech- Seller-
Looking for best return on investment
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Typical chat with PharmahellipHey Pharma we have great in vivo data massive indication wanna license
Interesting cool approach right up our alleyhellip and wersquore totally prepared to jump in early
Doesnrsquot seem to hurt the mice
Hmmn pityhellip just a bit too risky
What about tox
Come on matehellip we canrsquot afford that
ADME Tox package
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Lead Optimisation
In vivoPD
ADME TOXIND-enabling
Phase 1 Phase 2-3NDA
approval Market
RISK VALUE
Earliest Pharma buysWhere you want to sell
Pharma- Buyer-
Aussie Biotech- Seller-
Looking for best return on investment
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Typical chat with PharmahellipHey Pharma we have great in vivo data massive indication wanna license
Interesting cool approach right up our alleyhellip and wersquore totally prepared to jump in early
Doesnrsquot seem to hurt the mice
Hmmn pityhellip just a bit too risky
What about tox
Come on matehellip we canrsquot afford that
ADME Tox package
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Typical chat with PharmahellipHey Pharma we have great in vivo data massive indication wanna license
Interesting cool approach right up our alleyhellip and wersquore totally prepared to jump in early
Doesnrsquot seem to hurt the mice
Hmmn pityhellip just a bit too risky
What about tox
Come on matehellip we canrsquot afford that
ADME Tox package
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Unto the valley of death
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Pharma- Buyer-
Aussie Biotech- Seller-
Scenario 1 ADMETOXLarge animal model
Cost asymp $1MTime asymp 9-12 months
Scenario 2 TOXEfficacy Artificial human model
Cost asymp $30K ndash 100KTime asymp 1-3 months
Pharma- Buyer-
New options emerging
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Aussie Biotech- Seller-
Pharma- Buyer-
Letrsquos try that againhellip
OK letrsquos take baby steps
Hmmn pityhellip just a bit too risky
Simulated human tox studies
What do you have in mind
Humanised 3D models
Option amp eval $40K 2 months You pay
Irsquom listening
Could work can we see proposal
Now wersquore talking
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
ldquoTraditional preclinical disease models fail to accurately inform the potential efficacy [and toxicity] of
compounds transitioning into Phase I especially those with novel mechanisms of actionrdquo
- PAREXEL
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
How to de-risk preclinical investment
Show that a drug candidate will likely translate to humans before testing it in humans
o Safetytoxicity
o Efficacy
o Termination of deficient projectscompounds ()
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Humanised 3D cultures - The paradigm shift
o Spheroids
o Organoids
o Organs on a chip
o Bio-hybrid systems
o Disease modeling
o Robust and reproducible
o Detailed target validation
o Early translation to human physiology
o Strong IRB support to replace reduce amp refine (3Rs)
o Rapid and cost-effective
o Scalability - Low to High throughput
TechnologiesBenefits
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Human iPSC derived NSPCs DAPI GFAP B3 Tubulin
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Example Humanised 3D model of Alzheimerrsquos Disease
1mm
Model of CNS Model of Alzheimerrsquos (AD) Drug screening
Validation in AD animal model
Papadimitriou et al Developmental Cell 2018
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Companies investing in early human insights
Big Pharmas
Novartis
JampJ
Merck
Roche
Sanofi
GSK
Takeda
CROs offering 3D humanised assays
Charles River
Evotec
StemoniX
Ocello
Organovo
Creative BioLabs
InSphero
3D cell culture market to reach USD $47 bn in 2022 (Allied Research)
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Company Service offerings Clients and deals
Evotec
High-content screening
Target validation amp identification
Celgene $45M upfront
Sanofi euro3M upfront euro300M milestones
Bayer euro14M upfront euro300M milestones
Charles River
Laboratories
Target discovery
Hit ID
Academics
Biotechpharma
Governmentfoundations
Organovo
3D tissues for screening
therapeutics
3D bioprinter
NIH US$17M
LOreacuteal
Yale
CROs servicing the market
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
Alternative approaches for early human insights
Toxicity and efficacy screening in
o Human heart tissue
o Human DRG tissue
o Provides insights in aged cells
In vivo drug screening in
o Hollow fiber model
o Multiple human xenografts
o Mouse PKPD
Toxicity and efficacy screening in
o Bioprinted human tissues
o Measures liver metabolismtoxicity
o Measures excretion from kidneys
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
In vivo dataSmall animal model
Phase IHuman data
Scenario 1ADMETOX
Large animal model
Scenario 2TOXEfficacy
Artificial human model
What ifhellip
66 failure rateAnimals poor predictors of human physiology- Chocolate is toxic to dogs- Paracetamol is toxic to cats- Mice donrsquot develop Alzheimerrsquos
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom
August 10 2018
wwwbio-linkcom
108 South Parade Blackburn VIC 3130 Australia
Christopher Boyer Executive Directorcboyerbio-linkcom