1. “insero” = to plant
”gen” = gene
Rapid, cost-effective, and scalable manufacturing technology
that uses tobacco plants as biofactories of therapeutics
“Using tobacco to produce a life-enhancing treatment for emphysema disease”
I-Corps Contacts: 52

2. Team Inserogen
Entrepreneurial Lead  1st Prize, 2010 Big Bang! Business Plan
Competition at UC Davis
Lucas Arzola  Elevator Pitch Award, 2011 UC
Ph.D. Candidate, Chemical Engineering Berkeley B-Plan Competition
Emphasis in Biotechnology
 E-Team Grant, National Collegiate
Inventors and Innovators Alliance
Principal Investigator  25+ years of experience in Plant
Made Proteins Research
Karen McDonald, Ph.D.
UC Davis Professor, Chemical Engineering  Inventor in 1 issued patent and 4
patent applications
Mentor  20 years of experience in Process
Development Manufacturing and
Vasilis Voudouris, Ph.D., MBA Strategic Planning
Managing Director, Vereniki Solutions

3. A Green Multi-Product Manufacturing Platform
Harvested tobacco
> 90% reduction in capital costs
Reduction in operating costs
Large scale production in
weeks, not months

4. The Business Model Canvas Target Product – seasonal & pandemic flu
Version 1 vaccines
Tobacco Suppliers R&D Speed Publications
HHS BARDA
Gene Synthesis Comp. Manufacturing Cost-Effectiveness Conferences
DOD DARPA
CMO Regulatory Approval Scalability Long Term Contracts
CDC
CRO Marketing Safety
Vax Manufacturers
FDA Customization
NGOs
Intellectual Property U.S. Based Supply Wholesalers
Government
Manufacturing Facility
Contract Manufacturing
Facility Investment IP Licensing Costs
Fully Integrated Manufacturing (Sales)
Manufacturing Costs Marketing Costs
Licensing (Royalties)

5. The Business Model Canvas Target Product – seasonal & pandemic flu
Version 1 vaccines
Speed
Cost-Effectiveness
Scalability
Safety
Customization
U.S. Based Supply
What we thought: these features can meet
a need for the influenza vaccine market.

6. Getting Out of the Laboratory!
What we did: Contacted vaccine industry stakeholders
What we found:
DOSES  Unpredictable market
2B Pandemic flu  Flu vaccines are commodities
 Large companies are not willing
Surge capacity Opportunity to change their manufacturing
platform
250M Seasonal flu Seasonal flu Seasonal flu
“This is a billion-dollar investment
we are not willing to make”
YEAR 1 YEAR 2 YEAR 3

8. The Business Model Canvas Target Product – seasonal & pandemic flu vaccines
Version 2 alpha-1 antitrypsin (AAT)
Tobacco Suppliers R&D Speed Publications
Patients
Gene Synthesis Comp. Manufacturing Cost-Effectiveness Conferences
Physicians
CMO Regulatory Approval Scalability Long Term Contracts
Pulmonary Labs
CRO Marketing Safety Large Biotech Comp.
FDA Reliable Supply
Alpha-1 Foundation Intellectual Property U.S. Based Supply Wholesalers
Government
Alpha-1 Project Manufacturing Facility Customization
Direct to Patient
Grants Licensing (Royalties)
Facility Investment IP Licensing Costs
Upfront & Milestone Payments (from Partners)
Manufacturing Costs Marketing Costs
Lean Biotech Company (Sales)

9. What are we selling?
 First target product: Alpha-1 antitrypsin (AAT)
 Indication: AAT Deficiency (AATD) –
hereditary disorder resulting in low levels of
AAT in lungs, leads to emphysema, often
before age 40
 Less than 10% of AAT Deficiency individuals
have been properly diagnosed
 Worldwide Estimates
 3.4 million with AATD
 116 million carriers
“AAT may be one of the most common serious hereditary disorders in the world”
- President, Alpha-1 Europe

10. AAT Treatment is Expensive
 There is no known cure for AAT Deficiency
 Treatment: Augmentation therapy – weekly AAT infusions
 5 g AAT per patient per week
 Annual: 260 g AAT per patient
Competitor Pricing
 Prolastin - $350/gram
 Zemaira - $430/gram
 Aralast - $510/gram
 Alfalastin - $450/gram
Cost per patient: ~$100,000/year for life

11. Value Proposition
Problem: Commercial AAT for augmentation therapy is
derived from donated human blood
 Cost
 Reliable Supply: “There are problems with supply.
We need new ways of making AAT.”
– Scientific Director, Alpha-1 Foundation
 Safety: “The risk of transmission of infectious
agents cannot be completely eliminated.” –
Prescribing information for Zemaira
Value proposition: We can provide recombinant AAT
(we’ve already produced it in the lab!)
 Reliable and scalable supply
 Safer production
 Cost reduction – we believe we can substantially reduce treatment cost

12. Target Market
AAT deficiency in the United States New indications for AAT
Diabetes, cystic fibrosis, others
Under Total
treatment
6,000 patients
$600M
100,000 patients
$10B
Cost per patient: $100,000/year Millions of patients
Billions
“As diagnosis improves and new indications are found, we will need more
AAT produced.” – Scientific Director, Alpha-1 Foundation

13. Business Model and Customer Archetype
Lean biotech business model
“Starting out and for as long as you can, be a lean biotech company.” – CEO, Biotech Company
Core technology Outsource to CMO Finished Drug
Product
+
Patent protection
Customer: Established biotech company (for clinical development)
 Interested in blood proteins and/or alternative
manufacturing platforms
 Looking to increase their pipeline, gain market share
 Hundreds of millions (not billions) in revenue
 Hundreds (not thousands) of employees

14. Customer Decision Making
How do customers decide if they want our product?
• 400-500 opportunities per year
Initial Evaluation • 1-2 weeks
• 80-100 opportunities
Detailed Analysis • 2-3 months
• Internal innovation committee meeting
Go – No Go Decision • Once a month
• 25-30 opportunities
Due Diligence • 2-3 months
• Agree to terms, business development team approval
Business Discussions • 2-3 months
• Establish work plan and define milestones
Deal is signed • Project starts
Total time: 6-9 months

15. Revenue Model
= money = relationship
= information = AAT
Patient
Large Pharma Private Payor
Employer
Government Payor
Wholesalers
Hospital/Clinic
Government
Physicians
Taxpayer
Pulmonary Function Lab INFLUENCER

16. The Business Model Canvas Target Product – seasonal & pandemic flu vaccines
Version 3 alpha-1 antitrypsin (AAT)
Tobacco Suppliers R&D Speed Publications
Patients
Gene Synthesis Comp. Manufacturing Cost-Effectiveness Conferences
Physicians
CMO Regulatory Approval Scalability Long Term Contracts
Pulmonary Labs
CRO Marketing Safety Large Biotech Comp.
FDA Reliable Supply
Alpha-1 Foundation Intellectual Property U.S. Based Supply Wholesalers
Government
Alpha-1 Project Manufacturing Facility Customization
Direct to Patient
Grants Licensing (Royalties)
Facility Investment IP Licensing Costs
Upfront & Milestone Payments (from Partners)
Manufacturing Costs Marketing Costs
Lean Biotech Company (Sales)

17. Next Steps
Alpha-1 Project
NSF SBIR Phase I
Funding Venture Philanthropy
Program
Grant
Licensing agreement Freedom to
IP of core technology Operate analysis
Join Engineering
Team Development & Translational
Business Incorporation Technology
Center