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
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