# Completion of Non-clinical long-term GLP safety and GMP manufacture for first-in-class neuron regenerative therapy, NNI-362 Phase 2 POC AD trial

> **NIH NIH U01** · NEURONASCENT, INC. · 2024 · $350,000

## Abstract

Project Summary
Neuronascent (NNI) discovered a unique therapy for Alzheimer’s, NNI-362, that promotes adult-
born neurons to replace lost neurons, while protecting from degeneration. In NNI’s completed
Phase1a trial, NNI-362 demonstrated safety and significant reduction of brain phosphoTau181.
The aim is to follow up the Ph1a with a Phase 2 trial in AD, for which the following 4 specific
aims are necessary. 1) NNI requires further GMP manufacture and 2) liquid-gel cap formulation
of NNI-362; 3) the long-term (6-months) safety in M/F rats and dogs; and 4) regular IND updates
and a meeting with FDA to discuss the Phase 2 clinical plan. This unique drug could be
beneficial for AD by limiting phosphoTau pathology and promoting new neurons to reverse
disease over a six-month period.
In the first aim, Onyx Scientific will scale-up NNI-362 from the 1 kg cGMP method used for
Phase 1a, to the >5 kg cGMP required for completion of Phase 2-3. Secondly, NNI-362 clinical
product will then be formulated into a liquid-gel capsule prepared at 120 mg/capsule, already
shown safe and potentially efficacious in aged subjects. Patheon prepared the formulation used
in Phase1a, and determined the practicality of the liquid-gel cap with NNI-362.
Thirdly, Charles River will complete a dog and rat (M/F) GLP safety testing for 6-months, as
required by FDA for treatment of humans for the same 6-month period. The doses will be the
same as the completed 14day/14day washout study in rats at 10, 50 and 150 mg/kg and in dogs
at 15, 45 and 210 mg/kg po daily. Charles River has considerable experience in carrying out
FDA-approved GLP safety studies. Lastly, regulatory contractors CCS Associates will provide
yearly regulatory IND-update filings and will request a FDA meeting to discuss the Phase 2
clinical trial plan for mild to moderate AD patients.
With decades of AD drug failures, NNI used human neural cell screen to discover a “neuron
generating and neuroprotective” drug. Pre-clinical studies of NNI-362 demonstrated new
hippocampal neurons in aging and disease models, and neuroprotection to allow new neuron
integration that reversed behavioral deficits. Phase 1a in healthy aged subjects demonstrated
safety and indicated that NNI-362, provided significant reduction of AD-progression marker
phosphoTau181 after only 16 days of treatment period in human aged subjects. With longer term
treatment of AD patients, NNI-362 has the potential to not only slow progression of AD over the
short-term, but promote new neurons to fully reverse disease, long-term.

## Key facts

- **NIH application ID:** 11110628
- **Project number:** 3U01AG082687-02S1
- **Recipient organization:** NEURONASCENT, INC.
- **Principal Investigator:** JUDITH A KELLEHER-ANDERSSON
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $350,000
- **Award type:** 3
- **Project period:** 2023-09-01 → 2026-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11110628

## Citation

> US National Institutes of Health, RePORTER application 11110628, Completion of Non-clinical long-term GLP safety and GMP manufacture for first-in-class neuron regenerative therapy, NNI-362 Phase 2 POC AD trial (3U01AG082687-02S1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/11110628. Licensed CC0.

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