Manufacturing of New Batch AV-1959D Drug Product and Placebo for Phase 1 Trial

NIH RePORTER · NIH · R01 · $699,000 · view on reporter.nih.gov ↗

Abstract

Abstract Immunotherapy targeting pathological Aβ continues to be a promising therapeutic strategy for Alzheimer's Disease (AD) prevention. Published data suggested that inhibition of amyloid aggregation in people at risk of AD could delay AD onset. However, due to the treatment schedule and the cost of monoclonal antibodies, their passive administration may not be the best preventive treatment strategy. Therefore, we hypothesized that our universal MultiTEP-platform-based active vaccine, AV-1959D, could be an inexpensive alternative to the passive vaccination strategy. To move further with this program, our multidisciplinary team manufactured a clinical grade AV-1959D vaccine and completed comprehensive IND-enabling safety, efficacy, and immunogenicity studies in mouse models of AD, rabbits, and monkeys. As a result of these studies conducted within the scope of an NIA cooperative agreement (U01 AG048310), we generated data for the preparation of IND18953 for Phase 1 AV- 1959D clinical trials that the FDA cleared in 2020, prepared 200 mg (225 vials, 145 of which was used for release test and 3Y stability) cGMP AV-1959D for future phase 1 clinical trial and submitted the grant application for clinical trial "Determine the safety/tolerability and immunogenicity of ascending doses of AV-1959D in patients with early-stage AD" in February of 2019. We received funding from NIA only in 2022, after two years and three attempts, to begin the first human clinical trial of AV-1959D. Hence, we had to adjust our clinical program based on new information and regulatory recommendations outlined in this Administrative Supplement. More specifically, we extended the stability testing of the drug product up to five years and increased doses for the intradermal delivery of the AV-1959D vaccine based on new data obtained in COVID-19 trials with the first approved DNA vaccine delivered by Pharmajet Tropis®. These amendments required additional drug product vials for the Phase 1 trial. Accordingly, this administrative supplement program aims to justify manufacturing a new GMP batch of the AV-1959D vaccine as early as possible to initiate the Phase 1 trial in Y1 of this program.

Key facts

NIH application ID
10732215
Project number
3R01AG074983-02S1
Recipient
INSTITUTE FOR MOLECULAR MEDICINE
Principal Investigator
Michael G Agadjanyan
Activity code
R01
Funding institute
NIH
Fiscal year
2023
Award amount
$699,000
Award type
3
Project period
2022-02-15 → 2027-01-31