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New Drug for the Treatment of 
High Risk Neuroblastoma 
Total interviews: 116 to date 
(90 FACE TO FACE) 
4 more scheduled to year end 
(1 interview on Dec 24th 12.30 pm!!!) 
ICORPS: FINAL 2014 
David Bettoun Ph.D. 
C-level, Founder 
Pharmaceutical preclinical expert transitioned 
to C level biotech executive 
Azriel Schmidt Ph.D. 
P.I 
Pharmaceutical distinguished senior investigator 
and project manager for 25 years 
Lisa Malseed, Esq 
IE 
Biotech Executive 20 years
In the beginning… we thought multiple 
indications with a market of 10B
HaRo Pharmaceutical, Inc. 
Team 13 
Bifunctional compounds relevant to treatment of 
certain cancers (TNBC and NEUROBLASTOMA), 
autoimmunity, cachexia as well as live stock 
development 
ICORPS: FINAL 2014 
BEFORE ICORPS
Our first canvas
So for 120 hours… 
…We listened to experts from 
Academic Pediatric Research Hospitals 24 (41 interviews) 
Pharmaceutical and Biotech 11 (21 interviews) 
Clinical Trial Consortia 4 (12 interviews) 
CROs 6 (12 interviews)
About some of the little pain and 
HaRo 
needs 
- Regulatory 
- Manufacturing 
- Distribution 
Pharma 
need 
- Pipeline 
- R&D 
- Shorter 
development 
time 
Clinical oncology 
consortia / 
advocacy groups 
Have a mandate 
to bring novel medicine to 
patients 
HaRo 
needs to : 
access to 
Patients 
pediatric 
clinical expertise
About some of the little pain and 
KOLs 
need 
Novel mechanisms 
Novel compounds 
HaRo 
Needs 
• Primary cells 
• Animal models 
• Recognition
Medical Relevant Key activity Met with Inventor and Users (5 interviews) 
KOLs (15 interviews) are split on whether 
this model is still appropriate for new 
compound testing 
We asked KOL/CRO for comparison 
Unclear whether BOTH targets 
Transgenic 
TH-MYCN 
Traditionial xenografts for are well expressed 
Neuroblastoma are 
sufficient 
Other 
Models 
Patient Derived human cell lines are 
the new gold standard (Kol/CRO) 
Jackson Lab has orphan group with 
xenograph models of neuroblastoma 
under development 
UCSF lab has another xenograph 
model with both targets 
1. 2. 
Hypothesis 
We HEARD
At week 6
At week 6
At week 6
What we have they don’t-what they have we don’t” 
Relationship that crosses the canvas
Who is HaRo’s 
Customer 
Archetype? 
How does HaRo 
fit?
Hypothesis of Development Path 
Lead 
Optimization 
• Med Chem 
• In Vitro Efficacy 
• Primary cells 
based efficacy 
and targets 
engagement. 
• Benchmarking 
against known 
therapies in 
primary cells. 
Animal 
Modeling 
• Xenograft 
• Orthotopic 
• Genetically 
modified 
• Standard model 
Clinical 
• Toxicology 
• Engage clinical 
partners 
In House/ 
CRO/ 
Academic 
$100/$150 
Academic collaborators/ 
CRO/Pharma 
$50/$200 
NCI/ 
Pharma/ 
Foundations 
Still working 
Key Activities 
Key 
Resources/ 
partners 
In thousands, $Haro Has $ Needed
Advocacy groups and Pharma all go 
through KOL to carry efficacy studies
Cost vs. potential revenue 
Costs 
• COG will pay for IND filing and for phase-1/2 cost. Company provides testing material ($250K-$500K per kg) 
(Medical Director, Pediatric Cancer Foundation Developmental Therapeutics Program) 
• Orphan status filing: $40K (Orphan consultant) 
• Compound testing PDX mice $1,100 per mouse (Jackson Lab) 
• Social media are fundamental game changers but companies must be careful in handling them. Global Director 
Bio-CSL President IMPAX 
Revenue 
• Market potential for orphan drugs can be estimated by using cost of current care in view of clinical efficacy 
• Current cost of treatment: 
 Families with debts in the $1M range in the US. 
 $250K Israel 
 €180K France 
 1 course of mAb treatment $250K-cash for non-US patients in US 
• Find early on whether costs of drug development will match the potential revenues. Determine the potential 
revenues by estimating the number of paying patients in the Western World and finding the prices of orphan 
drugs with equivalent therapeutic efficacies. Professor of Economics, Temple University 
• Multiple indications is a financial advantage. (Partner, Third Rock Ventures)
Keys to HaRo’s success are: 
Key Activities 
• Clear achievable 
clinical development 
path. Bristol Meyer Squibb 
– GCT, Oncology 
• PoC in disease 
relevant animal 
model. Endo Pharma–Busi 
Develop 
• Trial must be 
designed for an 
existing and 
available patient 
population. Lankenau 
Institute– Manager, Clinical 
Trials 
Key Resources 
• Standard 
neuroblastoma 
animal model has 
limitations with 
respect to human 
disease relevance. 
Jackson Laboratories – 
modeling 
• Medically relevant 
models are essential. 
Endo, R&D Oncology 
• Primary cell based 
assay can go a long 
way in generating 
convincing data. NCI NIH 
– cell based researcher 
Value Proposition 
• A functional proven 
R&D team is a value 
proposition. ICORP 
experienced, Business 
Development 
• TNBC better VP for 
business purpose. 
Neuroblastoma 
pending institutional 
support and KOL 
buying. Endo, R&D 
Oncology. 
• Value in 
developing 
bifunctional 
compounds. Molecular 
Genetic Pathologist, Genentech
Our Customers want and value Orphan 37 
indication in pediatric oncology 
We have defined the key partners and activities 
which will deliver the data that our Customers 
have identified as important to them 
48 
We have identified NCI-funded clinical development 
partners leading to substantially reduced costs and a 
timeline acceptable to our Customers 
24 
81 
Pediatric oncologists are enthusiastic about the 
potential of our technology
What’s NEXT….
• Phase II SBIR application

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Haro Pharmaceutical I-Corps@NIH 121014

  • 1. New Drug for the Treatment of High Risk Neuroblastoma Total interviews: 116 to date (90 FACE TO FACE) 4 more scheduled to year end (1 interview on Dec 24th 12.30 pm!!!) ICORPS: FINAL 2014 David Bettoun Ph.D. C-level, Founder Pharmaceutical preclinical expert transitioned to C level biotech executive Azriel Schmidt Ph.D. P.I Pharmaceutical distinguished senior investigator and project manager for 25 years Lisa Malseed, Esq IE Biotech Executive 20 years
  • 2. In the beginning… we thought multiple indications with a market of 10B
  • 3. HaRo Pharmaceutical, Inc. Team 13 Bifunctional compounds relevant to treatment of certain cancers (TNBC and NEUROBLASTOMA), autoimmunity, cachexia as well as live stock development ICORPS: FINAL 2014 BEFORE ICORPS
  • 5. So for 120 hours… …We listened to experts from Academic Pediatric Research Hospitals 24 (41 interviews) Pharmaceutical and Biotech 11 (21 interviews) Clinical Trial Consortia 4 (12 interviews) CROs 6 (12 interviews)
  • 6. About some of the little pain and HaRo needs - Regulatory - Manufacturing - Distribution Pharma need - Pipeline - R&D - Shorter development time Clinical oncology consortia / advocacy groups Have a mandate to bring novel medicine to patients HaRo needs to : access to Patients pediatric clinical expertise
  • 7. About some of the little pain and KOLs need Novel mechanisms Novel compounds HaRo Needs • Primary cells • Animal models • Recognition
  • 8. Medical Relevant Key activity Met with Inventor and Users (5 interviews) KOLs (15 interviews) are split on whether this model is still appropriate for new compound testing We asked KOL/CRO for comparison Unclear whether BOTH targets Transgenic TH-MYCN Traditionial xenografts for are well expressed Neuroblastoma are sufficient Other Models Patient Derived human cell lines are the new gold standard (Kol/CRO) Jackson Lab has orphan group with xenograph models of neuroblastoma under development UCSF lab has another xenograph model with both targets 1. 2. Hypothesis We HEARD
  • 12. What we have they don’t-what they have we don’t” Relationship that crosses the canvas
  • 13. Who is HaRo’s Customer Archetype? How does HaRo fit?
  • 14. Hypothesis of Development Path Lead Optimization • Med Chem • In Vitro Efficacy • Primary cells based efficacy and targets engagement. • Benchmarking against known therapies in primary cells. Animal Modeling • Xenograft • Orthotopic • Genetically modified • Standard model Clinical • Toxicology • Engage clinical partners In House/ CRO/ Academic $100/$150 Academic collaborators/ CRO/Pharma $50/$200 NCI/ Pharma/ Foundations Still working Key Activities Key Resources/ partners In thousands, $Haro Has $ Needed
  • 15. Advocacy groups and Pharma all go through KOL to carry efficacy studies
  • 16. Cost vs. potential revenue Costs • COG will pay for IND filing and for phase-1/2 cost. Company provides testing material ($250K-$500K per kg) (Medical Director, Pediatric Cancer Foundation Developmental Therapeutics Program) • Orphan status filing: $40K (Orphan consultant) • Compound testing PDX mice $1,100 per mouse (Jackson Lab) • Social media are fundamental game changers but companies must be careful in handling them. Global Director Bio-CSL President IMPAX Revenue • Market potential for orphan drugs can be estimated by using cost of current care in view of clinical efficacy • Current cost of treatment:  Families with debts in the $1M range in the US.  $250K Israel  €180K France  1 course of mAb treatment $250K-cash for non-US patients in US • Find early on whether costs of drug development will match the potential revenues. Determine the potential revenues by estimating the number of paying patients in the Western World and finding the prices of orphan drugs with equivalent therapeutic efficacies. Professor of Economics, Temple University • Multiple indications is a financial advantage. (Partner, Third Rock Ventures)
  • 17. Keys to HaRo’s success are: Key Activities • Clear achievable clinical development path. Bristol Meyer Squibb – GCT, Oncology • PoC in disease relevant animal model. Endo Pharma–Busi Develop • Trial must be designed for an existing and available patient population. Lankenau Institute– Manager, Clinical Trials Key Resources • Standard neuroblastoma animal model has limitations with respect to human disease relevance. Jackson Laboratories – modeling • Medically relevant models are essential. Endo, R&D Oncology • Primary cell based assay can go a long way in generating convincing data. NCI NIH – cell based researcher Value Proposition • A functional proven R&D team is a value proposition. ICORP experienced, Business Development • TNBC better VP for business purpose. Neuroblastoma pending institutional support and KOL buying. Endo, R&D Oncology. • Value in developing bifunctional compounds. Molecular Genetic Pathologist, Genentech
  • 18. Our Customers want and value Orphan 37 indication in pediatric oncology We have defined the key partners and activities which will deliver the data that our Customers have identified as important to them 48 We have identified NCI-funded clinical development partners leading to substantially reduced costs and a timeline acceptable to our Customers 24 81 Pediatric oncologists are enthusiastic about the potential of our technology
  • 20. • Phase II SBIR application