Rapid, cost-effective, and scalable manufacturing technologythat uses tobacco plants as biofactories of therapeutics

“insero” = to plant ”gen” = gene

I-Corps Contacts: 52

“Using tobacco to produce a life-enhancing treatment for emphysema disease”

Team Inserogen

Mentor Vasilis Voudouris, Ph.D., MBA

Managing Director, Vereniki Solutions

Lucas Arzola Ph.D. Candidate, Chemical

Engineering Emphasis in Biotechnology

Entrepreneurial Lead

Principal InvestigatorKaren McDonald, Ph.D.

UC Davis Professor, Chemical Engineering

1st Prize, 2010 Big Bang! Business Plan Competition at UC Davis

Elevator Pitch Award, 2011 UC Berkeley B-Plan Competition

E-Team Grant, National Collegiate Inventors and Innovators Alliance

25+ years of experience in Plant Made Proteins Research

Inventor in 1 issued patent and 4 patent applications

20 years of experience in Process Development Manufacturing and Strategic Planning

Animal and human vaccines

Personalized medicine/biosimilars

Proteins for research use and diagnostics

Industrial enzymes for bioenergy

Cosmetic and cosmeceutical

Nutraceutical/ functional food

A Green Multi-Product Manufacturing Platform

Harvested tobacco

> 90% reduction in capital costsReduction in operating costsLarge scale production in weeks, not months

The Business Model Canvas Target Product – seasonal & pandemic flu vaccines

Wholesalers

Tobacco Suppliers

Gene Synthesis Comp.

CMO

CRO

FDA

Intellectual Property

Manufacturing Facility

R&D

Manufacturing

Regulatory Approval

Marketing

Speed

Cost-Effectiveness

Scalability

Safety

Customization

U.S. Based Supply

Publications

Conferences

Long Term Contracts

Government

HHS BARDA

DOD DARPA

Vax Manufacturers

NGOs

Contract Manufacturing

Fully Integrated Manufacturing (Sales)

Licensing (Royalties)

CDC

Facility Investment

Manufacturing Costs

IP Licensing Costs

Marketing Costs

Version 1

The Business Model Canvas

Speed

Cost-Effectiveness

Scalability

Safety

Customization

U.S. Based Supply

Version 1

What we thought: these features can meet a need for the influenza vaccine market.

Target Product – seasonal & pandemic flu vaccines

Getting Out of the Laboratory!What we did: Contacted vaccine industry stakeholders

What we found:

Pandemic flu2B

Surge capacity Opportunity

DOSES

Seasonal flu

YEAR 1

Seasonal fluSeasonal flu250M

YEAR 2 YEAR 3

Unpredictable market Flu vaccines are commodities Large companies are not willing

to change their manufacturing platform

“This is a billion-dollar investment we are not willing to make”

PIVOT TO THERAPEUTIC M

ARKET

The Business Model Canvas Target Product – seasonal & pandemic flu vaccines

alpha-1 antitrypsin (AAT)

Tobacco Suppliers

CRO

FDA

Alpha-1 Foundation

CMO

Patients

Physicians

Pulmonary Labs

Large Biotech Comp.

Conferences

Long Term Contracts

Publications

Wholesalers

Direct to Patient

Speed

Cost-Effectiveness

Scalability

Safety

Reliable Supply

Intellectual Property

Manufacturing Facility

R&D

Manufacturing

Regulatory Approval

Marketing

Gene Synthesis Comp.

Government

Facility Investment

Manufacturing Costs

IP Licensing Costs

Marketing Costs

Grants

Upfront & Milestone Payments (from Partners)

Lean Biotech Company (Sales)

Licensing (Royalties)

Alpha-1 Project Customization

U.S. Based Supply

Version 2

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

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

Cost per patient: ~$100,000/year for life

Competitor Pricing Prolastin - $350/gram Zemaira - $430/gram Aralast - $510/gram Alfalastin - $450/gram

Value Proposition

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

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

Target Market

6,000 patients

AAT deficiency in the United States

100,000 patients

Millions of patients

Under treatment

Total

New indications for AATDiabetes, cystic fibrosis, others

$600M

$10B

Billions

Cost per patient: $100,000/year

“As diagnosis improves and new indications are found, we will need more AAT produced.” – Scientific Director, Alpha-1 Foundation

Business Model and Customer Archetype

Lean biotech business model

+

Core technology Outsource to CMO Finished Drug Product

“Starting out and for as long as you can, be a lean biotech company.” – CEO, Biotech Company

Patent protection

(for clinical development) Customer: Established biotech company 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

Customer Decision Making

• 400-500 opportunities per year• 1-2 weeksInitial Evaluation

• 80-100 opportunities• 2-3 monthsDetailed Analysis

• Internal innovation committee meeting• Once a monthGo – No Go Decision

• 25-30 opportunities• 2-3 monthsDue Diligence

• Agree to terms, business development team approval• 2-3 monthsBusiness Discussions

• Establish work plan and define milestones• Project startsDeal is signed

Total time: 6-9 months

How do customers decide if they want our product?

Revenue Model

= money= information

= relationship

Large Pharma

INFLUENCER

Hospital/Clinic

Physicians

Patient

Employer

Government

Taxpayer

Government Payor

Private Payor

Pulmonary Function Lab

Wholesalers

= AAT

The Business Model Canvas Target Product – seasonal & pandemic flu vaccines

alpha-1 antitrypsin (AAT)

Tobacco Suppliers

CRO

FDA

Alpha-1 Foundation

CMO

Patients

Physicians

Pulmonary Labs

Large Biotech Comp.

Conferences

Long Term Contracts

Publications

Wholesalers

Direct to Patient

Speed

Cost-Effectiveness

Scalability

Safety

Reliable Supply

Intellectual Property

Manufacturing Facility

R&D

Manufacturing

Regulatory Approval

Marketing

Gene Synthesis Comp.

Government

Facility Investment

Manufacturing Costs

IP Licensing Costs

Marketing Costs

Grants

Upfront & Milestone Payments (from Partners)

Lean Biotech Company (Sales)

Licensing (Royalties)

Alpha-1 Project Customization

U.S. Based Supply

Version 3

Next Steps

FundingAlpha-1 Project

Venture Philanthropy Grant

NSF SBIR Phase I Program

IP Licensing agreement of core technology

Freedom to Operate analysis

Business Team Development & Incorporation

Join Engineering Translational Technology Center