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Experimental Gonococcal Vaccine

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 2R44AI115877-04
Agency Tracking Number: R44AI115877
Amount: $1,807,210.00
Phase: Phase II
Program: SBIR
Solicitation Topic Code: NIAID
Solicitation Number: PA19-272
Solicitation Year: 2019
Award Year: 2020
Award Start Date (Proposal Award Date): 2020-07-15
Award End Date (Contract End Date): 2023-06-30
Small Business Information
Louisville, KY 40204-2304
United States
DUNS: 145056607
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 (716) 829-2528
Business Contact
Phone: (716) 829-2528
Research Institution

Genital tract infection with Neisseria gonorrhoeae does not induce a state of specific protective immunity and it
can be acquired repeatedly. Despite public health measures, the disease persists at an unacceptably high
frequency; there is no vaccine against it, and resistance even to the latest generations of antibiotics continues
to emerge. Our SBIR Phase I work established proof-of-principle that intra-vaginal immunization with a
sustained-release interleukin-12 microsphere formulation plus proprietary gonococcal (GC) outer membrane
vesicles (NGoXIM, previously GvaX12) induces durable Th1-driven adaptive immune responses that protect
mice against genital tract infection with N. gonorrhoeae. Those studies demonstrated: (i) induction of GC-specific
Th1 cells, (ii) generation of anti-GC antibodies in serum and genital tract secretions, (iii) protection against
diverse strains of N. gonorrhoeae, (iv) recall of specific antibodies and reactivation of T cells after challenge
infection, and (v) duration of protection against infection for at least 6 months. Protection depended upon both
antibodies and IFNγ production. In SBIR Phase II, we established efficacy of intranasal (i.n.) immunization in
mice and, importantly, in non-human primates. We confirmed similar IFNγ production and generation of GC-
specific antibodies and by this route, demonstrated activity against additional antigenically diverse strains of N.
gonorrhoeae, including clinical isolates, and showed functional antibody activity against GC in bactericidal
assays. Finally, we concluded a type C pre-pre-IND meeting with the FDA, garnering Agency agreement with
our single species primate toxicology plans. The specific aims of this Phase IIb application are partially based
on Agency recommendations communicated during that meeting and the need to optimize NGoXIM for human
application. In Aim 1 NGoXIM will be further optimized in non-human primates using the i.n. route in preparation
for human trials. Aim 2 studies will conclude FDA-compliant pharmaceutical quality and development activity for
the vaccine components. Upon successful completion of Aims 1 and 2, Therapyx will request a type B meeting
with the FDA to confirm that the completed non-clinical IND-enabling program, including an NHP toxicology study
plan and preliminary clinical study design, is sufficient to support an IND (Aim 3). In Aim 4, following FDA
guidance and the results of our type B meeting, we will use validated products generated in Aim 2 to evaluate
initial serum pharmacokinetics (PK) and toxicity profiles of NGoXIM in cynomolgus monkeys. The overall goal
of this project is to produce the worldandapos;s first prophylactic vaccine against genital tract infection with Neisseria
Gonorrhea is the second-most-frequent, notifiable infectious disease in the United States. The Centers for
Disease Control report ~350,000 cases annually, and worldwide incidence is estimated at 78 million new
infections per year. No vaccine is available and the emergence of multiple-drug-resistant strains now raises
serious and urgent concerns over future treatment options. This proposal seeks to develop a novel strategy for
prophylactic vaccination against gonorrhea by directing the immune response to generate lasting protective

* Information listed above is at the time of submission. *

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