Tuberculosis The Race for a Cure

Transcription

1 Tuberculosis The Race for a Cure Kari Stoever Faster Cures Annual Conference November 4, 2013

2 Aeras: A non profit biotech advancing TB vaccines for the world Founded in 2003 in-house capabilities in finance, portfolio management, GMP pilot manufacturing, translational product development and policy, advocacy and resource mobilization 501(c)(3) organization registered in Washington DC Offices Rockville, MD (headquarters) Cape Town, South Africa Beijing, China Executive leadership team with decades of experience developing and commercializing new vaccines/biologics ~ 160 employees with annual budget of approx. USD $55 million

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4 TB is Mother Nature s number one killer over the past 200 years TB is spread through the air like a common cold Nearly 8.5 million people become sick with TB each year. TB kills 1 in 4 people infected with HIV. 530,000 annual cases among children aged under ,000 women killed annually by the disease Source: Nature/ World Tuberculosis Report,

5 How TB Spreads SOURCE: O Garra Anne et al. Annu. Rev. Immunol :

6 TB decline in the pre-antibiotic era COMPLACENCY & A FAILURE TO INNOVATE led the World Health Organisation (WHO) to declare TB to be a global emergency: Tuberculosis today is humanity s greatest killer, and it is out of control in many parts of the world. The disease, preventable and treatable, has been grossly neglected and no country is immune to it. Arati Kochi, Manager WHO Tuberculosis Program,

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8 US Outbreaks in 2012 Nearly 10,000 cases annually 8

9 Antimicrobial Resistance is Confounding Global Efforts to Control the Global Epidemic ~ 500,000 drug resistant cases in in 5 individuals with drug resistant TB are being accurately diagnosed and even less are receiving appropriate treatment Current treatments for drug resistant strains require a minimum of 2 years using highly toxic drug regimens 92 countries have reported XDR-TB Evolving with some strains becoming virtually untreatable Source: WHO TB Report, Image courtesy of Nature/ World Tuberculosis Report,

10 Ebola with Wings: Coming to a town near you? 92 countries reporting at least one case of XDR-TB Source: WHO Global TB Report,

11 TB will not be eliminated by 2050 Current rate of decline - 2%/yr (globally) Today TB incidence >100x higher than elimination target for 2050 Elimination target:<1 / million / yr -20%/yr China, Cambodia -4%/yr (the best observed nowadays) W Europe after WWII - 10%/yr (Historical example)

12 Controlling and reducing TB incidence requires the application of transformational interventions Infectious TB B C A 1 Transmission cycle breakpoints Transmission A 2 Breakpoints Preventing A transmission and infection B Blocking progression to infectious TB C Treating and sterilizing active TB Infected individuals Courtesy of Gilla Kaplan, Bill & Melinda Gates Foundation

13 Ill-equipped for this Complex Epidemic THE NEED FOR A NEW VACCINE We are beginning to see the winds of change, but what we really need is a storm. It is imperative that we transform the way we diagnose, treat, prevent, and control TB through biomedical research and public health measures Anthony Fauci, Director of NIAID

14 90 year-old BCG vaccine is the most widely used vaccine in the world Reduces the risk of severe pediatric TB disease, but: Unreliable protection against adult pulmonary TB, which accounts for most TB worldwide No significant impact on the global TB epidemic Not known to protect against latent TB Not recommended for use in infants infected with HIV

15 The Global Pipeline of TB Vaccine Candidates PHASE I PHASE IIa PHASE IIb PHASE III Ad5 Ag85A VPM 1002 MVA85A/AERAS-485 M. Vaccae McMaster CanSino Max Planck, VPM, TBVI, SII Oxford, Aeras Anhui Zhifei Longcon, China MTBVAC H1 + IC31 M72 + AS01E TBVI, Zaragoza, Biofabri SSI, TBVI, EDCTP, Intercell GSK, Aeras ID93 + GLA-SE RUTI IDRI, Aeras Archivel Farma, S.L Crucell Ad35/MVA85A H4/AERAS IC31 Crucell, Oxford, Aeras SSI, Sanofi-Pasteur, Aeras, Intercell H56/AERAS IC31 SSI, Aeras, Intercell Crucell Ad35/AERAS-402 Crucell, Aeras VIRAL VECTOR PROTEIN/ADJUVANT IMMUNOTHERAPEUTIC: AERAS SPONSORED rbcg ATTENUATED M.Tb Mycobacterial Whole Cell or Extract

16 A Business Case for TB Vaccine R&D Evidence-Based Decision Making in TB Vaccine Research & Development Aeras, TuBerculosis Vaccine Initiative (TBVI), the European Commission, and the European Investment Bank have developed a TB Vaccine R&D Business Case that utilizes rigorous public health and financial modeling to: 1. identify a product development strategy to maximize public health impact; 2. Assess the commercial viability of new TB vaccines; 3. evaluate portfolio development costs to inform investment strategies by phase of development 4. Demonstrate the cost efficiencies of a portfolio management approach.

17 Global Market Segments Countries in analysis 197 countries included in the Applied Strategy Model 14 countries excluded (no TB and/or birth data 2009) 183 countries included for analysis 183 countries geographic segmentation Economies are divided according to 2011 GNI per capita, calculated using the World Bank Atlas method low income - $1,025 or less; middle income - $1,026 - $12,475 lower middle income, $1,026 - $4,035; upper middle income, $4,036 - $12,475 high income - $12,476 or more CONFIDENTIAL - NOT FOR CIRCULATION DATA ARE ESTIMATES AND SUBJECT TO CHANGE 17

18 Public Health Impact A new vaccine that could prevent adolescents and adults from developing and transmitting TB would be the single most cost-effective tool in mitigating the epidemic. A 60% efficacious adolescent and adult vaccine, delivered to 20% of the target population, could potentially avert million incident cases of TB by 2050.

19 Higher vaccine coverage rates with a 60% efficacious infant, adolescent & adult vaccine could significantly reduce transmission

20 Revenue Market Potential A conservative estimate of the market revenue potential for new TB vaccines is estimated to be $13-14 billion in the first 10 years. The majority of the revenue potential is from High Income Countries, Upper Middle Income Countries and China.

21 Public and Private Sector Risk-Sharing A blended-capital financing structure will enable appropriate risk-sharing between private and public sectors: with governments strong economic and public health interest in new TB vaccines, public funding will be required to support the earlier phases of vaccine development, where the scientific risk is the greatest in return, industrial partners will be expected to cost-share in the later, more expensive phases of development

22 Portfolio Evolution using Monte Carlo Simulations Number of Vaccines Number of Vaccines in Portfolio by Development Stage Over Time 25 Projected Annual De $120, $100,000 $80,000 $60,000 $40,000 $20,000 $0 Discovery / Pre-Clinical 1 Pre-Clinical 2 Phase 1 / 2A Phase 2B Phase 3 Pre-Commerce Post-Commerce Discove Phase 2 Projected Annual Development Costs by Stage $120,000 Total Development Costs $846,786 $100,000 $80,000 $60,000 $40,000 $20,000 $0 Discovery / Pre-Clinical 1 Pre-Clinical 2 Phase 1 / 2A Phase 2B Phase 3 Pre-Commerce

23 Portfolio Development Probability of Success Probability of 1 Vaccine Reaching Commercialization 90% 80% 70% 60% 55% 56% 70% 77% 79% 82% 83% 50% 40% 30% 20% 16% 16% 10% 0% By Year 2022 By Year 2023 By Year 2024 By Year 2025 By Year 2026 By Year 2027 By Year 2028 By Year 2029 By Year 2030 Based on the base assumptions and the initial portfolio, there is a 55% probability of having one vaccine commercialized by 2024 and greater than 80% chance of having one vaccine commercialized by 2030

24 Overall Portfolio Development Costs Total Portfolio Development Costs - 1 Vaccine Commercialized Average - 630,592 Std. Deviation - 97,254 Min - 435,250 Max - 1,050, $250 - $300 $300 - $350 $350 - $400 $400 - $450 $450 - $500 $500 - $550 $550 - $600 $600 - $650 $650 - $700 $700 - $750 $750 - $800 $800 - $850 $850 - $900 $900 - $950 $950 - $1,000 - $1,000 $1,050 As highlighted in the histogram above (based on Monte Carlo simulation of the various potential outcomes), the estimated cost range for developing one vaccines through commercialization can range from $435m to $1,050m, with and average development cost of $630m.

25 Business Case Key Messages TB vaccines are our best hope to end the TB epidemic Shared public and private sector investment at key stages will offset risk Even partially effective TB vaccines for adults and adolescents will have a significant public health impact A viable market for TB vaccines exists Greater diversification needed in TB vaccine portfolio A disciplined portfolio management approach is necessary

26 Source: Global Tuberculosis Report 2012, WHO (2012), Nature Vol 502, No Suppl, S2 (2013), Financing Global Health 2012, IHME Funding priorities have lagged relative to the morbidity and mortality of tuberculosis Tuberculosis has led to more deaths in the last 200 years than any other infectious disease but has received significantly less funding in the last 10 years as compared to HIV and malaria 1 1,000,000,000 Tuberculosis death $43 billion HIV/AIDS global funding Plague Influenza Cholera HIV/AIDS Small pox Malaria 30,000,000 HIV/AIDS death 1 Based on OECD and IHME Development Assistance for Health (DAH) funding data Malaria Tuberculosis $7 billion global funding

27 Since this presentation began ~200 people have become infected with TB ~32 have died

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29 Thank you.