Description:
043 Adjuvant Development
Fast-Track proposals will not be accepted.
Direct-to-phase II proposals will be accepted
Number of anticipated awards: 1-3
Budget (total costs):
Phase I: $225,000 for up to 1 year
Phase II: $1,500,000 for up to 3 years
Background:
Adjuvants stimulate innate and/or adaptive immune responses. For the purpose of this SBIR, adjuvants are defined according to the U.S. Food and Drug Administration (FDA) as “agents added to, or used in conjunction with, vaccine antigens to augment or potentiate (and possibly target) the specific immune response to the antigen.” Currently, only three adjuvants have been licensed as components of vaccines in the United States - aluminum hydroxide/aluminum phosphate (alum), 4’-monophosphoryl lipid A (MPL), adsorbed to alum as an adjuvant for an HPV vaccine, and the oil-in-water emulsion MF59 as part of the Fluad influenza vaccine for people age 65 years and older. Additional efforts are needed to more fully develop the potential capabilities of promising adjuvants, particularly for special populations such as the young, elderly and immune-compromised. In addition, adjuvants may facilitate the development of immunotherapeutics for immune-mediated diseases, such as allergen immunotherapy.
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Project Goal:
The goal of this project is to accelerate pre-clinical development and optimization of a single lead adjuvant candidate or a select combination-adjuvant for prevention of human disease caused by non-HIV infectious pathogens. For this solicitation, a combination-adjuvant is defined as a complex exhibiting synergy between individual adjuvants, such as: overall enhancement of the immune response; potential for adjuvant-dose sparing to reduce reactogenicity while preserving immunogenicity; or broadening of effector responses, such as through target-epitope spreading or enhanced antibody avidity. The adjuvant products supported by this program must be studied and further developed toward human licensure with currently licensed or new investigational vaccines, and may not be developed as stand-alone agents.
Phase I Activities
Depending on the developmental stage at which an adjuvant is entered into the
Program, the offeror may choose to perform one or more of the following:
• Optimization of one candidate compound for enhanced safety and efficacy. Studies may include:
• Structural alterations of the adjuvant or modifications to formulation; or
• Optimization of heterologous prime-boost-regimens.
• Development of novel combinations of previously described individual adjuvants, including the further characterization of an adjuvant combination previously shown to enhance immune responses synergistically.
• Establishment of an immunological profile of activity and immunotoxicity that can be used to evaluate the capability of the adjuvant to advance to human testing.
• Preliminary studies in a suitable animal model to evaluate the protective efficacy of a lead adjuvant:vaccine or adjuvant/immunotherapeutic combination.
• Analysis of vaccine efficacy through the use of a combination adjuvant and studies to evaluate the safety profile of the combination adjuvant:vaccine or adjuvant/immunotherapeutic formulation.
Phase II Activities
Extended pre-clinical studies that may include IND-enabling studies such as:
• Additional animal testing of the lead adjuvant:vaccine or adjuvant/immunotherapeutic combination to evaluate immunogenicity, protective efficacy and immune mechanisms of protection.
• Pilot lot or cGMP manufacturing of adjuvant or adjuvant:vaccine or adjuvant/immunotherapeutic compound.
• Advanced formulation and stability studies.
• Toxicology testing.
• Establishment of quality assurance and quality control protocols.
• Pharmacokinetics/absorption, distribution, metabolism and excretion studies.
This SBIR will not support:
• The further development of an adjuvant that has been previously licensed for use with any vaccine.
• The design and conduct of clinical trials (see http://www.niaid.nih.gov/researchfunding/glossary/pages/c.aspx#clintrial for the NIH definition of a clinical trial).
• The discovery and initial characterization of adjuvant candidates.
• The development of adjuvants or vaccines to prevent or treat cancer.
• Development of Platforms, such as vehicles, or Delivery Systems that have no immunostimulatory activity themselves.
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• The development and/or optimization of a pathogen-specific vaccine component.
