Sustainability: The R&D Pipeline for Neglected Disease Vaccines

Elizabeth L. Ponder, PhD

May 4, 2011

Global Vaccines 202X: Access, Equity, Ethics2-4 May 2011

The Franklin Institute Science MuseumPhiladelphia, USA

BIO Ventures for Global Health (BVGH)

We have a unique perspective. We look at health problems facing poor countries through a dual lens:

Expertise in Industry Expertise in Global Health

Mission: BIO Ventures for Global Health is an independent, non-profit organization whose mission is to save lives by accelerating the development of novel, biotechnology-based drugs, vaccines, and diagnostics that address the unmet medical needs of the developing world.

Mission: BIO Ventures for Global Health is an independent, non-profit organization whose mission is to save lives by accelerating the development of novel, biotechnology-based drugs, vaccines, and diagnostics that address the unmet medical needs of the developing world.

The Global Health Primer provides a landscape analysis for neglected disease products

• Updated online database for 17 neglected diseases: www.bvgh.org/globalhealthprimer.aspx

• Covers drugs, vaccines, and diagnostics

• Highlights product needs and opportunities

There are 12 approved and 160 vaccines in development for neglected diseases

106 Products in Clinical

Development

Preventive vaccines receive the largest portion of neglected disease R&D funding

G-FINDER ANALYSIS:

Mary Moran & Javier Guzman, Policy Cures

Neglected disease vaccines are in development by 169 organizations representing 35 countries

Developer Type Number of Organizations

Academic/Research Institution 67

Biopharmaceutical Company 52

Government 29

Product Development Partnership (PDP)

17

Other 4

Total 169

80

55

12

1210

6

Number of Developers per Country

Looking across the vaccine landscape, several trends emerge:

• Newer vaccines in development for neglected diseases are increasingly scientifically complex due to new technologies and targeting more challenging organisms

• Proof of concept trials for new vaccines are progressing, but scientific learning beyond efficacy from these efforts has been minimal

• A lack of financing options, clear policy statements, or WHO prequalification of an approved vaccine severely limits access

Early Innovation

Clinical Proof of Concept

Approval and Access

Early-stage neglected disease vaccines focus on newer technologies

No approved vaccines in humans

In use in approved vaccines

Parasitic diseases represent the majority of neglected diseases but the minority of products

Unicellular parasitic diseases:• Malaria• Sleeping sickness• Chagas disease• Leishmaniasis

Multicellular parasitic diseases:• Schistosomiasis• Soil transmitted helminthiasis• Onchocerciasis• Lymphatic filariasis

Proof of concept studies are advancing but still have a long way to go

Disease Proof of ConceptR&D Investment

(G-FINDER)

ETEC Partial protection from Cholera vaccine $15.6 million

Dengue Data expected Q4 2012 $115.7 million

HIV 30% efficacy in Phase III trial $1,374 million

Malaria30-50% efficacy in a series of Phase II trials

$116.4 million

SchistosomiasisPhase III trial as adjunct to MDA in progress

$9.5 million

ShigellaOn market vaccine in China provides ~60% efficacy

$14.0 million

Tuberculosis BCG protects against systemic TB $187.0 million

PATH and BVGH (2011) The Case for Investment in Enterotoxigenic Escheriscia coli Vaccines.Clemens JD et al. (1988) J Infect Dis 158: 372-377. Peltola H et al. (1991) Lancet 338: 1285-1289. Rerks-Ngarm S et al. (2009) NEJM 361: 2209-20. Casares S et al. (2010) Vaccine 28: 4880-94. PAHO et al. (2004) “Progress in Shigella vaccine development.” in Vaccines: preventing disease & protecting health. Colditz GA et al. (1994) JAMA 271: 698-702WHO (2004) Weekly Epidemiological Record 79: 27-38Moran M et al. (2011) Neglected Disease Research and Development: Is the Global Financial Crisis Changing R&D? Policy Cures.

Neglected disease vaccines challenge the traditional industry model for clinical evaluation

As products fail, or show only partial efficacy, how do we decide what to do next?

• Traditional Industry Model: How can we help vaccines that will fail, fail faster?

• For Neglected Diseases: How can we help vaccines that will fail, not just fail faster, but also fail smarter? How can we maximize learning from vaccine trials with partial efficacy?

Clear policy and financial support improve vaccine access

Disease Product (Company)Initial

ApprovalYear of WHO

Prequalification GAVI supported? Current UsePneumococcal Disease

Synfloris (GSK) Europe,

20092009

Yes, AMC44 countries as of

2009Prevnar 13 (PCV13 – replacement for PCV 7 which was approved in the U.S. in 2000; Pfizer)

U.S., 2010 2010

Rotavirus RotaTeq (Merck) U.S., 2006 2008Yes

23 countries as of 2009Rotarix (GSK) U.S., 2008 2009

Clear policy and financial support improve vaccine access

Disease Product (Company)Initial

ApprovalYear of WHO

Prequalification GAVI supported? Current UsePneumococcal Disease

Synfloris (GSK) Europe,

20092009

Yes, AMC44 countries as of

2009Prevnar 13 (PCV13 – replacement for PCV 7 which was approved in the U.S. in 2000; Pfizer)

U.S., 2010 2010

Rotavirus RotaTeq (Merck) U.S., 2006 2008Yes

23 countries as of 2009Rotarix (GSK) U.S., 2008 2009

Cholera Dukoral (SBL Vaccin, Sweden)

Sweden, 1991

2001

NoLimited, need

more data on roll in outbreaks

Shanchol (Shantha Biotechnics, India)

India, 2009 -

mORCVAC (VaBiotech, Vietnam)

Vietnam, 2009

-

TyphoidVivotif (Crucell)

Switzerland, 1981

-No, shortlisted for

future support

In travelers and select endemic

regionsTyphim Vi (Sanofi-Pasteur)

U.S., 1994 -

Key needs for future neglected disease vaccine development include:

• A focus on parasitic disease vaccine development to understand the technical feasibility and potential health impact

• Increased investment in the integration of scientific research questions with efficacy studies for vaccines to maximize return on public investment

• More operational and clinical research on existing vaccines to support the development of clear policy statements, accelerate WHO prequalification, and inform decisions on new product needs

Early Innovation

Clinical Proof of Concept

Approval and Access