# Targeted Lymphoablation as an alternative to HSCT to cure T1D

> **NIH NIH R44** · AVM BIOTECHNOLOGY, LLC · 2022 · $667,733

## Abstract

PROJECT SUMMARY
With insulin being the only approved treatment of Type 1 Diabetes (T1D), there is a very large unmet medical
need for a definitive cure for pediatric and adult patients. In the U.S., diabetes currently affects 14% of the
population of all ages, and it is the seventh leading cause of death, with an estimated cost of $237 billion every
year to the healthcare system. The medical community has recognized T1D as an autoimmune disease and
proposed different immunotherapeutic approaches to cure it. However, only hematopoietic stem cell
transplantation (HSCT) after reset of the immune system with toxic chemotherapy, has so far shown temporary
restoration of insulin independence. Antigen-specific immunotherapies (Teplizumab) are offering partial
therapeutic benefits with only 5-6% of the patients showing two-year insulin independence. AVM Biotechnology
(AVM) is developing a new approach for T1D based on a novel immunomodulatory formulation (AVM0703) that
mobilizes “Supercharged” Natural Killer T-Cells for safe removal of autoreactive lymphocytes responsible for
T1D. Results from Phase I activities show that AVM0703 administered alone can prevent diabetes in 45%, or
delay its onset by 20-31 weeks in 55% of mice in the NOD model of T1D. AVM0703 may represent a safe
standalone curative option for 50% of the patients based on preclinical mouse data, providing them an expected
window of insulin independence of 3-30 years. In case of relapse, the safety profile of AVM0703 will allow
repetitive dosing up to 8 times as currently approved by the FDA for an ongoing clinical trial in relapsed refractory
lymphoid malignancies (NCT04329728). For patients not showing remission, AVM0703 is expected to reinforce
other immunotherapies allowing a wider range of T1D patients to achieve insulin independence, for instance,
combinatorial therapy with Teplizumab (anti-CD3). This SBIR Phase II project has been designed as a
randomized, placebo-controlled multi-center pre-clinical trial to obtain solid data which, with the already available
toxicology information for AVM0703 and potential companion agents such as Teplizumab, will expedite IND
approval and pave the way to clinical trials. Activities in Aim 1 will be directed to perform a pre-clinical dose-
finding and mechanism of action study in NOD mice in three scenarios: pre-diabetic, new-onset, and established
diabetes. Results from Aim 1 will be used to determine the AVM0703 dose to be used in the pivotal efficacy
study for reversal of new-onset diabetes and established diabetes in Aims 2 and 3. An IND application will be
filed at the end of the project.

## Key facts

- **NIH application ID:** 10484003
- **Project number:** 2R44DK121634-02A1
- **Recipient organization:** AVM BIOTECHNOLOGY, LLC
- **Principal Investigator:** Theresa Deisher
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $667,733
- **Award type:** 2
- **Project period:** 2019-09-20 → 2024-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10484003

## Citation

> US National Institutes of Health, RePORTER application 10484003, Targeted Lymphoablation as an alternative to HSCT to cure T1D (2R44DK121634-02A1). Retrieved via AI Analytics 2026-05-29 from https://api.ai-analytics.org/grant/nih/10484003. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*