Improving Global Health

Through Multidisciplinary Research

and Multi-Institutional Partnerships

Brett P. Giroir, M.D. Vice Chancellor for Strategic Initiatives

The Texas A&M University System

Presented to

The Texas Higher Education Coordinating Board

October 27, 2011

Desperately Seeking Cures

How the road from promising scientific

breakthrough to real-world remedy has

become all but a dead end.

“Basic research is healthy in America. But patients aren’t benefiting. Our understanding of diseases is greater than ever. But academics think, ‘We had three papers in Science or Nature, so that must have been [NIH] money well spent.’?”

Dr. John Adler Stanford University

May 15, 2010

The Texas Medical Center, Houston

The Pipeline of New Therapies is “Drying Up”

Critical Barriers for the Development of New Vaccines and Therapeutics

Translational Research:

Characterizing animal models,

particularly natural animal

models of disease

Developing diagnostic tests and

biomarkers

Researching and implementing

new ways to make vaccines and

biotherapeutics

Basic Science

Discoveries

Basic Science

Discoveries

Translational Research

Translational Research

Pivotal Clinical Trials

Pivotal Clinical Trials

>75% of the development costs (through Phase 2 clinical trials) for new

vaccines and biotherapeutics is on R&D for manufacturing

>75% of the development costs (through Phase 2 clinical trials) for new

vaccines and biotherapeutics is on R&D for manufacturing

Complexity of Biological Drugs Must be Manufactured in “Live” Organisms (adapted from Genentech)

“Small Molecule Drug” Aspirin

21 Atoms

“Biological Drug” Antibody

~25,000 atoms

Business Jet ~30,000 pounds

Bike ~20 pounds

SIZ

E

CO

MP

LE

XIT

Y

1950s chicken embryo technology

7-8 months to change vaccine strains

Limited by availability of fertilized eggs from pathogen-free hens, growth of flu strain, etc.

Biologics Manufacturing: Description

Inadequate Manufacturing Platforms

Cause Slow Response Times

Inadequate Facility Design Stifles

the Innovation Pipeline

$500M - $1B “up front” costs

5-7 years to design and construct

Single product, single technology ONLY

Must be completed before pivotal clinical

trials are completed

The National Center for Therapeutics Manufacturing (NCTM)

The NCTM was supported by a

$50 million grant from the State of

Texas Emerging Technology Fund

Requires only electrical hook-up and chilled water Air-bearings allow pod to be mobile

Superstructure 42 x 18 feet

Modular Biomanufacturing Units (“Pods”) Collaborative Development with Texas Based Commercial Partners

Redundant core systems with automatic failover

The National Center for Therapeutics Manufacturing A Disruptive Innovation for Public Health and Biosecurity

TAMUS-owned, privately-operated biopharmaceutical manufacturing research, development, and production facility

As many as six simultaneous products can be developed (vaccines and therapeutics)

Supports personalized therapeutics to national scale vaccines

Demonstrated a new architecture to lower capital costs by as much as 90%

Allows quick change over to new products, or surge response against pandemics

Completely integrated with educational programs and worker training courses

Has become a magnet for corporate relocation, research, education, and jobs

The NCTM is a prototype for a flexible multiproduct,

multi-technology strategic biosecurity facility

The NCTM is a prototype for a flexible multiproduct,

multi-technology strategic biosecurity facility

8

TAMUS – MD Anderson Cancer Center Collaboration Agreement for the NCTM

Edgar Hernandez Patient Zero, March 2009

April 27, 2009

U.S. Declares Public Health Emergency

Over Swine Flu

Pandemic Declared June 11, 2009

The Pandemic Wake-Up Call

1918

DARPA Blue Angel Program Also known as “Project GreenVax”

Objective: Perform the research and development required to deliver a candidate

pandemic influenza bulk antigen, expressed in plants, at a scale of 1 kilogram per

month (scalable to 10 kg per month)

Performer: Texas Plant Expressed Vaccine Consortium

(TAMUS and G-Con, LLC )

Start: February 1, 2010

Funding: DARPA $39,998,063

TPEVC $25,488,160

Plant Made Pharmaceuticals

• Plants are capable of making

complex proteins, including

vaccines and antibodies

• Process: – Nicotiana plants are seeded and

grown for 5-6 weeks

– Plants are infiltrated with bacteria

containing the gene(s) for the protein

of interest

– Infected plants synthesize desired

protein for 5-7 days

– Plants are harvested, protein

extracted, and then purified by

conventional pharmaceutical

processes

Project GreenVax Ongoing Collaboration between TAMUS and Caliber Biotherapeutics

13

• 2.2 million Nicotiana plants

– Fully contained, hydroponic, wavelength

optimized LED grow lights

• Up to 10 kg per month of purified

recombinant protein

– Equivalent of 100 million doses of vaccine per

month

• The facility was fully operational 15

months post federal award

• Initial research products

– New vaccine for tuberculosis

(2 billion people infected worldwide)

– New antibody therapies for cancer

(lymphoma, breast cancer, and others)

– Enzyme therapies for patients with metabolic

diseases

Photos excerpted from Houston Chronicle, October 10, 2011

Texas A&M Stakes Claim as Leader

in Pharmaceuticals

Friday, November 26, 2010

“…a confluence of factors had primed

Bryan and neighboring College Station…

to be the country’s “third coast” of

pharmaceutical manufacturing, with

dozens of companies cropping up in the

next 5 to 10 years …”

"They are building a little Sunnyvale down

there," Callahan said, referring to a city at

the center of California's Silicon Valley.

"I don't find that kind of sparkle anywhere

else. That is what we need for this big, big

problem."

Saturday, April 2, 2011

Biotechnology is new breed of

business at Texas A&M

There is an Urgent Need to Improve

U.S. Biosecurity Preparedness

Bioterrorism Report Card:

U.S. unprepared

October 12, 2011

April 27, 2009

U.S. Declares

Public Health

Emergency

Over Swine Flu

Edgar Hernandez Patient Zero, March 2009

The Movie: Contagion Almost everything is correct….

…except the happy ending

“And we are launching a new initiative

that will give us the capacity to respond

faster and more effectively to

bioterrorism or an infectious disease -–

a plan that will counter threats at home

and strengthen public health abroad.”

President Barack Obama State of the Union

January 2010

Texas is Uniquely Poised to Meet the New

National Biosecurity Agenda

“Our Nation must have the nimble, flexible

capacity to produce MCMs rapidly in the face

of any attack or threat, known or unknown,

including a novel, previously unrecognized,

naturally occurring emerging infectious disease.”

U.S. Department of Health and Human Services The Public Health Emergency Medical Countermeasures

Enterprise Review

August 2010

U.S. Department of Health and Human Services Request for Solutions

Centers of Innovation in Advanced Development and Manufacturing

– Objectives

• Develop a national response capability to develop and manufacture

pandemic and seasonal influenza vaccines for the US population

(50 million doses in 4 months)

• Develop and manufacture biothreat vaccines and countermeasures

for the U.S. Strategic National Stockpile

• Lead the development of new therapies from ~Pre-IND through FDA

approval

• Train the US workforce in all aspects of vaccine development,

including high tech manufacturing and clinical trials, etc.

The Texas A&M University System (TAMUS) Proposal Team

TAMUS COMPONENTS

Texas A&M University

TAMU Institute for Preclinical Studies (TIPS)

Texas Engineering Experiment Station (TEES)

Texas Engineering Extension Service (TEEX)

Texas Veterinary Medical Diagnostic Laboratory

(TVMDL)

Texas A&M Research Foundation (TAMRF)

TAMUS COMPONENTS

Texas A&M University

TAMU Institute for Preclinical Studies (TIPS)

Texas Engineering Experiment Station (TEES)

Texas Engineering Extension Service (TEEX)

Texas Veterinary Medical Diagnostic Laboratory

(TVMDL)

Texas A&M Research Foundation (TAMRF)

INTEGRATED BIOPHARMACEUTICAL

COMPANIES

GlaxoSmithKline Biologicals (Belgium)

Bavarian Nordic (Denmark)

Caliber Biotherapeutics (Texas)

INTEGRATED BIOPHARMACEUTICAL

COMPANIES

GlaxoSmithKline Biologicals (Belgium)

Bavarian Nordic (Denmark)

Caliber Biotherapeutics (Texas)

BIOPROCESS TECHNOLOGY PROVIDERS

Sartorius (Germany)

GE Healthcare (Sweden)

deltaDOT (UK)

BIOPROCESS TECHNOLOGY PROVIDERS

Sartorius (Germany)

GE Healthcare (Sweden)

deltaDOT (UK)

ACADEMIC INSTITUTIONS

Baylor College of Medicine (Texas) Sabin Vaccine Institute

Vaccine Research Unit

Texas Children’s Hospital

UTMB-Galveston National Laboratory

University of Florida

Blinn College (Texas)

ACADEMIC INSTITUTIONS

Baylor College of Medicine (Texas) Sabin Vaccine Institute

Vaccine Research Unit

Texas Children’s Hospital

UTMB-Galveston National Laboratory

University of Florida

Blinn College (Texas)

NON-PROFIT RESEARCH INSTITUTES

Lovelace Biomedical and Environmental Research

Institute (New Mexico)

Texas Biomedical Research Institute (Texas)

Mary Crowley Research Center (Texas)

NON-PROFIT RESEARCH INSTITUTES

Lovelace Biomedical and Environmental Research

Institute (New Mexico)

Texas Biomedical Research Institute (Texas)

Mary Crowley Research Center (Texas)

COMMERCIAL PARTNERS

• Kalon Biotherapeutics (Texas)

• Lonza Houston (Texas)

• PPD, Inc. (North Carolina)

• NDA Partners (California)

COMMERCIAL PARTNERS

• Kalon Biotherapeutics (Texas)

• Lonza Houston (Texas)

• PPD, Inc. (North Carolina)

• NDA Partners (California)

Texas versus “the others”

The New York Times

“The flying machine which will really fly

might be evolved by the combined and continuous efforts of mathematicians

and mechanicians in from one million to ten million years”

October 9, 1903

“We started assembly today”

Orville Wright’s Diary

October 9, 1903