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Haro Pharmaceutical I-Corps@NIH 121014

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I-Corps @ NIH

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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
  4. Our first canvas
  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
  9. At week 6
  10. At week 6
  11. At week 6
  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
  19. What’s NEXT….
  20. • Phase II SBIR application

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