Gammaglobulin I-Corps@NIH 121014

Copyright © 2014 GigaGen, Inc.

Team 11: Recombinant Gammaglobulin

Matt SpindlerPrincipal InvestigatorPhD UCSFPostdoc StanfordSpecialty is immune therapies

David JohnsonC-LevelMBA UC BerkeleyPhD StanfordSpecialty is genomics innovation

Jennifer KellerIndustry ExpertMBA UC IrvineMS UC BerkeleySpecialty is clinical marketing

The first pure antibody source for patients who can’t make their own antibodies ($7 billion existing market).We interviewed 163 people to test our business hypothesis.


Situation

• Millions of sick people can’t make their own antibodies, or they make faulty ones

• To stay healthy, these people need to get antibodies from another source• Now, plasma is collected from thousands of healthy people and fractionated to

isolate antibodies• The antibodies are then shot back into the sick people• This is called gammglobulin, or IVIG: a $7 billion market!

1000’s of peoplePlasma collection Plasma fractionation IVIg


Complication

Demand exceeds supply:•Three major shortages in the past 15 years•Strong market growth and off-label use will continue to stress the supply•Serum collection will hit upper limit

Quality problems:•Factor XIa contaminants disrupted supply in 2010 and caused significant morbidity•IgA contamination can cause anaphylactic shock•Batch-to-batch variation in genetic content limits efficacy•No ability to engineer isotype or genotype content for particular applications

IVIg Shortages

Year


Solution: Recombinant Gammaglobulin

Remember these guys?If nature doesn’t have enough, make it yourself!•More predictable•More engineerable•More scalable


Get out of the building? Get out of the country!

• Would anyone use recombinant?• Who is the first target patient population• Why would they use recombinant?• Who would we need to work with to make

the product?• What are the regulatory risks?• What would clinical trials look like?


Finding the Key First Customers

Primary immune deficiency patient

Immunologist

Cancer patient

Autoimmunity patient

Infectious disease doctor

Oncologist


Customers Teach Us What They Value


Immunologist

Cancer patient

Autoimmunity patient


Purity

Differentiating preclinical and clinical work

“Hyperimmune” targeting specific pathogens

Oncologist


Learning: Customer Value Proposition

Unreliable supply

Viral contamination

Convenience

Peace of mind

Live freely

Survive

Blood protein contamination

Consistency

Home infusion

Consistent product

Price uncertainty

Clear reimbursement Renewabl

e resource

In vitro production

In vitro productionrhIVIg

Clinical studies

Infusions every 3rd week

Longer ½ life in vivo

Don’t get sick

Save money

Feel healthy


Which Stakeholders Are Most Important?

Healthcare professionals

Patients

Pharmas

Payer/Regulatory

Distribution


Learning: Stakeholder Importance


Patients

Pharmas

Immunologists

IDF

Baxter/Grifols/CSL

Payer/Regulatory

Distribution

PIDautoimmunity

Infectious disease

CBERMedicare/private payer

pediatric

Specialty pharmaWholesale

Less value More value

Biotechs w/innovation programs


Learning: Stakeholder Importance


Patients

Pharmas

Immunologists

IDF

Baxter/Grifols/CSL

Payer/Regulatory

Distribution

PIDautoimmunity

Infectious disease

CBERMedicare/private payer

pediatric

Specialty pharmaWholesale

Less value More value

Biotechs w/innovation programs


Business Model Canvas: Partners


Immunologist


Purity



IDF

CBER


Learning: Ecosystem for Development

• Yeast library protocol development internal vs. external

• Small-scale vs. large-scale cGMP production

• cGMP vs. pilot production• US vs. Europe vs. Asia production


Business Model Canvas: Protein Partners


Immunologist


Purity



IDF

CBER

Small scale GMP (EU)

Large scale GMP (EU)

Protein library protocol development

Protein library protocols


Business Model Canvas: Channels


Immunologist


Purity



IDF

CBER





Distributer

Specialty pharma

Hospitals

GPO


Learning: Distribution Models

Sp Pharmacy

patient

Distributer

Lg Hosp Pharm

GPO(Manages pricing for Sp

P/Pharm)

Greatest volume

Some volume

Very little volume


Business Model Canvas: Channels


Immunologist


Purity



IDF

CBER





Distributer

Specialty pharma

Hospitals

Group purchasing organization


Learning: Ecosystem for Development

Resources Activities Partners

IP protecting in vitro production process

Physician channels for clinical studies

Pre-clinical assays SOPs

Regulatory Consultant

Microfluidic chip designs

Recombinant Ig library

Human Blood Source

Clin Study sites (PID)

GMP production facilities

Med Ad Board

Pre-clinical studies for IND

Build rIg library pipeline

<100pt study for launch

Informatics for library QC Microfluidic chip foundry

GLP rodent CRO

IDF

Distribution

Specialty Pharmacy or Wholesaler


Conclusion: Next Steps

First Pass Canvas

Problem/Solution validation

Low Fidelity MVP, Clinical Paths ID’d

Product/Market Fit

Left-side of the Canvas

Path to Revenues, OP Plan, Cust Data points

Right-side of the Canvas

High Fidelity MVP

Business Case Defined

Verdict on Phase II Grant:•NCI Phase I is a separate program from IVIG•Definitely moving forward on that separate program•Separate program supports big pharma partnering

Investment Readiness Verdict: 7•MVP is our big hurdle•Financing with pending Phase I SBIR and profit from two big pharma deals•Looking for $15-20MM Series A in 2015


Post-Script: Hyperimmune Directed IVIG


Immunologist


Purity



IDF

CBER





Distributer

Specialty pharma


Business Model Canvas: Week 1







Documents

Gammaglobulin I-Corps@NIH 121014