funding in return for rights outside the developed world: public-private partnerships gerald j....
TRANSCRIPT
Funding in Return for RightsOutside the Developed World:
Public-Private PartnershipsGerald J. Siuta, Ph.D.
Consultant, Business Development
September 27, 2007
What is a Public-Private Partnership?
An organization that pursues a social mission by employing the best
practices of the private sector and drawing upon resources from the
public and private realms
Types of Public-Private Partnerships Basic Knowledge/Research
– SNP Consortium Improvement of Access to Health Products
– International Trachoma Initiate Global Coordinating/Funding Mechanisms
– Global Fund to Fight AIDS, Tuberculosis and Malaria Health Services Strengthening
– Global Campaign for Microbicides Public Education and Advocacy
– Corporate Council on Africa Regulation, Quality and Standards
– Anti-Counterfeit Drug Initiative Product Development Partnership (PDP)
– Global Alliance for TB Drug Development
PDP Operations Provide specific disease expertise Fill development gaps Have purchasing power – US$1B for TB alone Unique deals, not charity projects Undertake clinical development Build extensive networks for market access in
developing countries Provide credibility with advocates, NGOs and
activists
How PDPs Work
Who are PDPs?
Case Study:TUBERCULOSIS
Global Tuberculosis Epidemic One-third of the world’s population is infected
with Mycobacterium tuberculosis (M.tb)– 2 billion people
8-9 million develop active disease annually 2 million deaths occur each year
– 1 person dies every 15 seconds 400,000 cases of MDR-TB each year Leading cause of death in HIV-positive people
– 12 Million people are TB/HIV co-infected
TB’s economic toll: $16 billion a year
Current TB Drug Therapy Active TB
– Standard therapy – 4 drugs (isoniazid, rifampin, pyrazinamide & ethambutol) for 2 months, followed by isoniazid and rifampin for 4 months
Latent TB– Standard therapy – isoniazid for 9 months
Multi-Drug Resistant TB (MDR-TB)– Individualized, prolonged therapy, few available drugs, poorly tolerated
and difficult to administer TB/HIV Co-Infection
– Treatment as in active TB, but drug interactions with antiretroviral agents make simultaneous therapy difficult
Extensively Drug Resistant TB (XDR-TB)– No treatment available
The Need for New TB Drugs
Complex 6-9 months treatment with a 4 drug combination regimen
No new anti-TB drug in over 40 years TB/HIV co-infections fueling each other MDR-TB is on the rise Unattractive market for private sector No capitalization of public sector research
History of the TB Alliance
Cape Town Declaration – February 2000– Hosts: Rockefeller Foundation and the Medical
Research Council of South Africa– Over 120 organizations (health, science, philanthropy
and private industry) Results
– Support goals of Stop TB Initiative– Create Scientific Blueprint– Develop Pharmacoeconomic Analysis
Build a Global Alliance forTB Drug Development
The TB Alliance Independent, international Product Development
Partnership founded in October 2000 Non-profit organization Headquarters in New York City
– Offices in Brussels and Cape Town Entrepreneurial, virtual R&D approach
– Out-source R&D to public and private partners Pro-active fundraising
– Over US $200 million raised Support ~ 200 FTE worldwide and 35 FTE in-house
Our Mission
Develop an entirely new therapeutic regimen that will shorten or simplify the treatment of tuberculosis
Coordinate and act as catalyst for global TB drug development activities
Ensure Affordability, Adoption and Access (AAA Strategy)
AAA Strategy
Affordability– Appropriate pricing in developing countries
Adoption– Ensure that new drugs are incorporated into
existing treatment programs Access
– Procurement and distribution to those patients who need them most
Our VisionFDCs
6 Months
2 Months
10 Days
Profile of a New TB Drug
Shorten treatment to less than 2 months Novel mechanism of action (MDR/XDR-TB) Orally active Once daily or intermittent therapy Compatible with HIV treatment Low cost of goods
Financial Support
Bill and Melinda Gates Foundation Rockefeller Foundation Netherlands Ministry for Development
Cooperation United States Agency for International
Development (USAID) Governments of Great Britain and Ireland
Types of Deals
In-Licensing IP Assignment Sponsored R&D Collaborative R&D Freedom to Operate Clinical Trials
TB Alliance PortfolioDiscovery
Co
mp
ou
nd
s, A
nal
og
s an
d D
eriv
ativ
es
Nitroimidazole Analogs (U. of Auckland/U. of Illinois at Chicago)
Quinolones(KRICT/Yonsei University)
Multi-Functional Molecules(Cumbre)
Mycobacterial Gyrase Inhibitors(GlaxoSmithKline)
InhA Inhibitors(GlaxoSmithKline)
Screening and Target Identification(AstraZeneca)
Nitroimidazole PA-824 (Chiron/Novartis)
Clinical Development
Active TB Alliance program
TB Alliance in discussion
Focused Screening(GlaxoSmithKline)
Pleuromutilins(GlaxoSmithKline)
Moxifloxacin (Ethambutol Substitution)(Bayer)
Malate Synthase Inhibitors(GlaxoSmithKline/Rockefeller U./Texas A&M U.)
New Targets(University of Pennsylvania)
Riminophenazines(Institute of Materia Medica/BTTTRI)
Protease Inhibitors(Queen Mary, University of London)
Proteasome Inhibitors(Cornell University)
Moxifloxacin (Isoniazid Substitution)(Bayer)
Chiron/Novartis
PA-824 – A novel nitroimidazole Discovered by Pathogenesis, Inc. Distinct mechanism of action Potent activity against both active and
slow growing M.tb Possesses both bactericidal and sterilizing
activity
Chiron/Novartis
Worldwide exclusive license for the treatment of tuberculosis
Defined scientific milestones Grant-back option Manufacturing rights No royalties in developing world
Development of PA-824
Phase I clinical trials began June 3, 2005– Preclinical development completed in 3 years– Drug was well tolerated with no definitive
dose-limiting adverse events
Phase II extended Early Bactericidal Activity (EBA) study has begun in Cape Town, South Africa
University of Auckland
Synthesis of PA-824 analogs Identified many new pharmacophores,
several of which have demonstrated potent activity against TB
Optimization has led to nitroimidazole analogs that have in vitro activity greater than PA-824
GlaxoSmithKline
Joint drug discovery program at GSK’s Diseases of the Developing World facility in Tres Cantos, Spain
Four individual projects:– Mycobacterial gyrase inhibitors– InhA inhibitors– Pleuromutilins– Focused screening
GlaxoSmithKline
Project oversight by Joint Steering Committee TB Alliance helps to support 25 full-time
scientists at GSK working exclusively on the TB drug program
GSK absorbs all remaining overhead costs GSK contributes a matching number of staff Any resulting medicines will be made affordable
and accessible to those most in need
Korea Research Institute of Chemical Technology (KRICT)
Located in Daejeon, South Korea Synthesized more than 600 quinolones,
pyridones & quinolizines In vitro and in vivo biological testing at the
Yonsei University College of Medicine in Seoul, South Korea
Four lead compounds have been selected for further preclinical evaluation
Cumbre Pharmaceuticals
Joint program on the design, synthesis and optimization of multi-functional antibiotics
The TB Alliance has exclusive rights to these compounds for the treatment of tuberculosis and other neglected diseases
Cumbre retains rights to pursue the compounds for use in other infectious disease areas
Institute of Materia Medica
Joint research partnership for the design, synthesis and evaluation of a class of compounds known as riminophenazines– Class was discovered in the 1950s
The collaboration will utilize IMM's expertise and integrated capabilities in chemistry, pharmacology and manufacture
The TB Alliance-BayerMoxifloxacin Deal
Moxifloxacin
Fluoroquinolone antibiotic Orally active Once-a-day dosage Approved in 104 countries for the
treatment of bacterial respiratory and skin infections
Moxifloxacin for TB
Novel mechanism of action: kills M.tb by inhibition of DNA gyrase
In vivo studies showed moxifloxacin reduced treatment time by two months when substituted for isoniazid
Safe to use with antiretroviral agents since it is not metabolized by the cytochrome P-450 enzyme system
October 18, 2005
TB Alliance and Bayer HealthCare announced a partnership to coordinate a global clinical trial program to study
the potential of moxifloxacin to shorten the standard six-month treatment of TB
The Partnership
Clinically assess the efficacy and safety of moxifloxacin as a front-line agent for the treatment of TB
If clinical trials are successful, register moxifloxacin for a TB indication
Committed to making the product affordable and accessible to patients in the developing world
Moxifloxacin Clinical Trials
Evaluate whether substitution of moxifloxacin for one of the standard TB drugs (isoniazid or ethambutol) eliminates TB infection faster than current standard therapy
Trials to be run in Brazil, Canada, South Africa, Spain, Tanzania, Uganda, the United States and Zambia
More than 3,000 TB patients will be enrolled
Bayer Commitments
Donate moxifloxacin for each clinical trial site
Cover costs of regulatory filings Provide moxifloxacin at an affordable
price for patients with TB in the developing world
TB Alliance Commitments
Coordinate and help cover the costs of the clinical trials
Ensure coordination of information and results towards the goal of registration
Leverage substantial support from:– U.S. Centers for Disease Control and Prevention (CDC)– Orphan Products Development Center of the U.S. Food &
Drug Administration– European and Developing Countries Clinical Trials
Partnership (EDCTP)
Special Recognition
Licensing Executives Society
On September 13, 2006, the Licensing Executives Society Industry/University and Government
Laboratory Transactions Industry Sector presented the TB Alliance and Bayer its Deals of Distinction
Award which recognizes worthy transactions involving licensing and transfer of intellectual property and promote creative and innovative
solutions to business issues
Scrip – World Pharmaceutical News
The TB Alliance-Bayer deal was also one of six finalists for the Scrip 2006 Best Partnership
Alliance Award which recognizes the importance of partnerships involving pharmaceutical and/or
biotech companies, focusing on deals that require strong strategic input from both partners, are
mutually beneficial to both parties, hold promise to address an unmet medical need and demonstrate
strategic potential as well as an innovative business model
Global Alliance for TBDrug Development
www.tballiance.org