Manufacturing platform for rapid, cost-effective, and scalable

production of therapeutics in tobacco

“insero” = to plant ”gen” = gene

Lucas Arzola (EL)Karen McDonald (PI)

Vasilis Voudouris (Mentor)

The Business Model Canvas

SpeedCost-EffectivenessRobustnessScalabilitySafetyEase of CustomizationU.S. Supply

R&DManufacturingRegulatory ApprovalLicensingMarketing

Tobacco SuppliersGene Synthesis CompaniesCMOs - Purification - Fill & Finish- Packaging- QA/QCCROs- Clinical TrialsFDA

IP – Patents, Trade SecretManufacturing Facility

Capital InvestmentsManufacturing CostsLicensing CostsMarketing

Contract Manufacturing Fully Integrated Manufacturing (Sales) Licensing (Royalties)

U.S. Government- CDC - HHS BARDA- DOD DARPAForeign GovernmentsNGOsVaccine Manufacturers- Established and

Emerging Biotech

Distribution through Government and Pharma Companies

PublicationsConferencesLong-Term Contracts with Government and Vaccine Manufacturers

Target Product – seasonal & pandemic flu vaccines alpha-1

antitrypsin

Customer

Dr. Jeffrey Almond – VP of External Discovery and R&D, Sanofi PasteurDr. Roman Chicz – Senior Director of External R&D, Sanofi Pasteur

Who is our customer? Biotechnology startups follow a licensing model, the goal is to develop a

working product and seek a big partner that will fund product development through clinical trials and regulatory approval (joint venture for product co-development, startup is acquired if product is successful)

Established companies – they rely on startups to broaden their product pipeline and find new technologies (70% of pipeline, 100% of preclinical projects are from partnerships)

Customer Relationships

How are they going to hear about us? Vaccine world is small! Conferences and publications – large biotech companies have “scout teams”

that attend conferences and read publications to find out about the latest technologies and products BIO (Biotech Industry Organization) International Convention World Congress of Vaccine ImVacs (Immunotherapeutics and Vaccine Summit) ISPE (Pharmaceutical Engineering) Annual Meeting BIO Smartbrief, BioPharm, GEN, Fierce Biotech

Direct contact – startups looking to partner with big biotech approach their ventures and partnering division directly

Customer Decision Making

Total time: 6-9 months

How do customers decide if they want our product?

Being Deal Ready

What are the terms for the partnership? Collaborations have clear go – no go milestones built into the agreement Customer appoints an alliance manager to work directly with the startup Customer agrees to pay a non-refundable upfront payment, royalties for the

technology, and milestones payments once those are achieved Startup agrees to provide an exclusive license to the customer, equity stake

depending on the valuation of the startup, stage of product development Terms may be included for acquisition depending on success

Being Deal Ready

How do customers want our product? As a biotech startup, we have only 1-2 products Must bring value to our customer – expand their pipeline, provide them with

new capabilities

What do they want to see in place? Proof-of-concept data IP and Freedom to Operate Preclinical Data – toxicology studies, animal studies Nice to have: Phase I and or Phase II clinical trial data

How do keep them wanting our product? Partnerships are win-win and long term Joint venture will transition into acquisition if product development is

successful

Initial Customer Feedback

Dr. Nancy Cox– Director of Influenza Division, CDC

What are the entry barriers in the flu vaccine market? For seasonal vaccines, process is established and supplies the 250M yearly

worldwide demand effectively Opportunity is in the lack of surge capacity of current platforms to deal with

the 2B doses needed for a pandemic – however, you need to have approval for seasonal to have a shot at the pandemic market

Unexpected market – pandemic once every 20-30 years Flu vaccines are commodities – low selling price, low profit margins, a lot of

competition

Leaning towards a pivot to the therapeutics market Pursue a high-value product that is not been made recombinantly, known

market, high selling price, high profit margin Possible new target: alpha-1 antitrypsin (AAT) 2 more meetings with vaccine customers

Prototype

What have we done in the lab? For vaccine: Proof of concept in progress For AAT: proven production, years of experience

What do we want to do? Product characterization Purification studies Perform pilot scale proof-of-concept