Asclepix I-Corps@NIH 121014
•Download as PPTX, PDF•
1 like•2,281 views
The document summarizes the findings from 102 customer interviews conducted by an I-Corps team exploring a novel cancer therapy. Based on the interviews, the team pivoted from targeting head and neck cancer to glioblastoma, identified their customers as pharmaceutical licensing scouts and biotech executives rather than patients or doctors, and adjusted their preclinical strategy and partnerships to focus on demonstrating safety and efficacy while mitigating risks related to their nanotechnology approach.
More Related Content
What's hot
What's hot (20)
Viewers also liked
Viewers also liked (20)
Similar to Asclepix I-Corps@NIH 121014
Similar to Asclepix I-Corps@NIH 121014 (20)
More from Stanford University
More from Stanford University (20)
Recently uploaded
Recently uploaded (20)
Asclepix I-Corps@NIH 121014
- 1. Total Interviews: 102 Initial Proposition: A novel therapy for head and neck cancer Jordan J. Green- CEO Eric Bressler- PI Niranjan Pandey- Industry Expert Dec 10, 2014 NIH I-CORPS Team 6
- 2. AsclepiX I-Corps Team 6 2 Jordan J. Green CEO Niranjan Pandey Industry Expert Eric Bressler PI Total Interviews: 102
- 3. Original Business Model Canvas 3 Key Partners Key Activities Value Props. Customer Rel. Customer Seg. • KOLs • Peptide GMP • Particle GMP • Preclinical CRO • Increase survival vs. SOC Key Resources • HNSCC patients • Low tox • Continued drug development Channels • MOA • Efficacy Data • IP • Grants/Fundi ng • Personnel • Reputable GMP • Publications • KOLs • HNSCC patients • Neurosugeon s • Oncologists • Pharma licensing scouts Cost Structure Revenue Streams • Toxicology • Preclinical efficacy • Salaries • Rent/in-house R&D • Legal/Patent • Sale of drug • Acquisition of assets by outside company • Co-development w/ Big Pharma
- 4. What we did: Interviews 4 : 31 : 35 : 17 : 15 : 4
- 5. Finding Our Real Customers 5 Key Partners Key Activities Value Props. Customer Rel. • KOLs • Peptide GMP • Particle GMP • Preclinical CRO • Increase survival vs. SOC Key Resources • HNSCC patients • Low tox • Continued drug development Channels • MOA • Efficacy Data • IP • Grants/Fundi ng • Personnel • Reputable GMP • Publications • KOLs Customer • HNSCC patients • Neurosugeon s • Oncologists • Pharma licensing scouts Cost Structure Revenue Streams • Toxicology • Preclinical efficacy • Salaries • Rent/in-house R&D • Legal/Patent Seg. • Sale of drug • Acquisition of assets by outside company • Co-development w/ Big Pharma
- 6. Finding Our Real Customers 6 Customer Segments • HNSCC patients • Neurosugeon s • Oncologists • Pharma licensing scouts • Biotech BD • Scientific heads of R&D Our customers aren’t the patients. Our customers aren’t the physicians We ourselves will not sell a physical drug Our product is data, IP, and a signal of efficacy from our drug • Recommenders are Pharma licensing scouts • Decision makers are BD Execs at Pharma/Biotech • Influencers are Scientific heads of R&D at Pharma/Biotech Who did we learn this from? • Big pharma business development • Small biotech C-level executives • Peer learning
- 7. A Pivot from HNSCC to ? 7 Key Partners Key Activities Value Props. Customer Rel. Customer Seg. • KOLs • Peptide GMP • Particle GMP • Preclinical CRO • Increase survival vs. SOC Key Resources • HNSCC patients • Low tox • Continued drug development Channels • MOA • Efficacy Data • IP • Grants/Fundi ng • Personnel • Reputable GMP • Publications • KOLs • Pharma licensing scouts • Pharma licensing scouts • Biotech BD • Specific people in executive-level Cost Structure Revenue Streams positions (7 contacts) • HNSCC patients • Neurosugeons • Oncologists • Toxicology • Preclinical efficacy • Salaries • Rent/in-house R&D • Legal/Patent • Sale of drug • Acquisition of assets by outside company • Co-development w/ Big Pharma
- 8. Hypotheses and Experiments 8 Hypothesis Experiment Results: Knowledge gained Head and Which cancer Glioblastoma : 19 neck cancer is indications are best Breast : 2 our target targeted by anti-angiogenic HCC: 2 market for our agents? HNSCC : 0 technology Glioblastoma is our target market for our technology Is an anti-angiogenic agent such as our compound best for glioblastoma? Thumbs up: 19 Thumbs down: 7 Glioblastoma has no good treatment, no cure Pre-operative MRI (August 8th, 2014) Immediate post-operative MRI (August 10th, 2014) Most-recent MRI (October 10th, 2014)
- 9. A Pivot to Glioblastoma, Revised VP 9 Value Propositions • Double progression-free survival vs. SOC • Delay in adverse events (seizures) • Improved QoL (reduced edema) “Brain cancer (glioblastoma) has no cure and no significant competition on the market. It is a highly angiogenic cancer.” - Alfredo Quinones-Hinojosa, M.D. , JHU Medicine “Multiple growth factor inhibition is promising. There are 3 clinical endpoints. There is almost always space in clinical trials for glioblastoma” - W. D. Figg Sr., PharmD, NCI “Glioblastoma is an orphan disease and a good opportunity.” - Wendy Perrow, MBA, CEO, Alba Therapeutics
- 10. Preclinical Opportunities to Create Value 10 Key Partners Key Activities Value Props. Customer Rel. Customer Seg. • KOLs • Peptide GMP • Particle GMP • Preclinical CRO • Increase survival vs. SOC • Delay in adverse event (need metrics) • Increased QoL (need metrics) Key Resources • HNSCC patients • Low tox • Continued drug development Channels • MOA • Efficacy Data • IP • Grants/Fundi ng • Personnel • Reputable GMP • Publications • KOLs • Pharma licensing scouts • Pharma licensing scouts • Biotech BD • Specific people in executive-level (7 contacts) Cost Structure Revenue Streams • Toxicology • Preclinical efficacy • Salaries • Rent/in-house R&D • Legal/Patent positions • Sale of drug • Acquisition of assets by outside company • Co-development w/ Big Pharma
- 11. Preclinical Opportunities to Create Value 11 Hypothesis: There is a “Gold Standard Model” that will satisfy our customers Experiment: 20 interviews w/ KOLs, pharma, and physicians Results: 18 16 14 12 10 0 2 4 6 8 What is the most trusted preclinical model? What would indicate success to pharma/biotech? Interviews Supporting PDGX “The best model is human, but doubling survival time in orthotopic PDGX models would be exciting.” -Jan Sakaria, Mayo Clinic 8 confirm Vs. “Unbelievable amounts of money wasted on animal efficacy models for cancer.” -Ken Carter, Noble Life Sciences 12 confirm (no gold standard) Ask the right question
- 12. Preclinical Opportunities to Create Value 12 Key Activities • Mechanism of action • PDGX efficacy model, multiple models • Metabolism • Exposure “You can’t go to the FDA without metabolism and exposure data in several species.” -Barbara Slusher, Dir. Drug Disc., JHU; Ex Pharma VP >10 Confirm Moving Forward • Choose a PDGX model which has value to certain KOLs. Use it for toxicity, exposure, metabolism, & signal of efficacy (doubling length of survival) • In vitro data on MOA / target validation adds value - Don’t waste time/money on additional models
- 13. Are there challenges to “Nano?” 13 Key Partners Key Activities Value Props. Customer Rel. Customer Seg. • KOLs • Peptide GMP • Particle GMP • Preclinical CRO • Increase survival vs. SOC • Delay in adverse event (need metrics) • Increased QoL (need metrics) Key Resources • HNSCC patients • Low tox • Continued drug development Channels • MOA • PDGX efficacy model • Metabolism • Exposure • IP • Grants/Fundi ng • Personnel • Reputable GMP • Publications • KOLs • Pharma licensing scouts • Pharma licensing scouts • Biotech BD • Specific people in executive-level (7 contacts) Cost Structure Revenue Streams • Toxicology • Preclinical efficacy • Salaries • Rent/in-house R&D • Legal/Patent positions • Sale of drug • Acquisition of assets by outside company • Co-development w/ Big Pharma
- 14. Hypotheses and Experiments 14 Hypothesis Experiment Results: Knowledge gained Nanotechnolog Does Risk: 24 y adds nanotechnology Value: 11 significant add value or risk value to our to our program? product Manufacturing is the main risk with nanotechnolog y What are the types of risks inherent to nanotechnology-based therapy? Complicates development: 5 Potentially toxic: 2 Manufacturing challenges: 4 Technical issues: 3 Perception: 4 Our customers do not understand nano: 1 Customers regard new medical technology such as nanotechnology as neutral to negative, not intrinsically valuable What the technology enables, targeted delivery, is valuable
- 15. Challenges of “Nano” 15 Key Partners Key Activities • KOLs • Peptide GMP • Narrowed choices • Particle GMP • Preclinical CRO • Narrowed choices • Nano Characterization Laboratory • MOA • Efficacy Data • Metabolism • Exposure • Continuous flow manufacturing Key Resources • IP • Grants/Funding • Personnel “Every nanoparticle company is full of sh*t when they say they have manufacturing worked out. It is not trivial.” - CEO of a biotech “Items that cause failure are: Inability to scale up for clinical studies, reproducibility issues due often to incorrect choice of manufacturer, endotoxin/sterility issues, and stability/shelf-life.” -Nicholas Panaro, NCL Nano manufacturing problems can be overcome using continuous flow processes. -Judy Britz, MD Bio Center We need to demonstrate low risk through “pain relievers” such as validated QC and scale up.
- 16. Resources- -Partners Intellectual Property Scientists Capital Consultants Management Personnel MOA Signal Efficacy Manufact. Peptide Manufact. Particle/QC Clinical Trials Acquire Personnel JHU Labs (collab.) AsclepiX Lab Raise Funds GMP: Peptide GMP: Particle CROs KOLs NCL Foundations F T O 16
- 17. Revised Trajectory 102 Interviews later: Pivot from Head and Neck Cancer to Glioblastoma Customers from patients to BD executives in Pharma and Biotech. We add value for them through our data and IP, both of which we continue to develop Activities such as demonstrating preclinical safety and efficacy. We will choose a patient-derived glioblastoma animal model as we can link it to key KOL and customers New Partners, Resources, and Activities as “pain relievers” to reduce risks of our new technology 17
- 18. Our Team has had a Paradigm Shift 18 Know our customer first and have them in mind always We are already applying the same business canvas approach to our company’s other strategic goals to very good effect Think about learning opportunities (interviewing) whenever meeting with people Academics + journals see the newest thing as exciting, our customers see low risk + high reward as most exciting
- 19. Next Steps with SBIR Phase II Go, with a significant pivot Feasibility data from Phase I can still lead to the technical foundation for Phase II General drug and approach will stay the same Indication will switch to glioblastoma Commercial plan will be significantly improved Investment readiness level: 3 Problem / solution validation 19