New Drug for the Treatment of

High Risk Neuroblastoma

ICORPS: FINAL 2014

Total interviews: 116 to date(90 FACE TO FACE)4 more scheduled to year end(1 interview on Dec 24th 12.30 pm!!!)

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

Bifunctional compounds relevant to treatment of

certain cancers (TNBC and NEUROBLASTOMA),

autoimmunity, cachexia as well as live stock

development

HaRo Pharmaceutical, Inc.Team 13

ICORPS: FINAL 2014

BEFORE ICORPS

Our first canvas

Clinical Trial Consortia 4 (12 interviews)

Pharmaceutical and Biotech 11 (21 interviews)

Academic Pediatric Research Hospitals 24 (41 interviews)

So for 120 hours…

…We listened to experts from

CROs 6 (12 interviews)

About some of the little pain and

HaRoneeds

- Regulatory- Manufacturing

- Distribution

Pharmaneed

- Pipeline- R&D- Shorter

developmenttime

Clinical oncology consortia /

advocacy groups

Have a mandateto bring novel medicine to

patients

HaRoneeds to :

access to Patients

pediatric clinical expertise

About some of the little pain and

KOLsneed

Novel mechanismsNovel compounds

HaRoNeeds

• 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

Unclear whether BOTH targets are well expressed Traditionial xenografts for

Neuroblastoma aresufficient

OtherModels

TransgenicTH-MYCN

Patient Derived human cell lines are the new gold standard (Kol/CRO)

UCSF lab has another xenographmodel with both targets

Jackson Lab has orphan group with xenograph models of neuroblastoma under development

1. Thisisanxampletext.

2.Goah

Hypothesis

We asked KOL/CRO for comparison

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?

HaRo provides

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/

FoundationsStill working

Key Activities

KeyResources/

partnersIn thousands, $Haro Has $ Needed

Advocacy groups and Pharma all go through KOL to carry efficacy studies

Cost vs. potential revenueCosts

• 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

37Our Customers want and value Orphan 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

81Pediatric oncologists are enthusiastic about the

potential of our technology

What’s NEXT….

• Phase II SBIR application