# Expedited Expansion Cohort Clinical Trial for Relapsed/Refractory 'no-option' Non-Hodgkin's Lymphoma/Leukemia Patients

> **NIH NIH R44** · AVM BIOTECHNOLOGY, LLC · 2022 · $999,999

## Abstract

ABSTRACT
Non-Hodgkin’s Lymphoma (NHL) is the 7th most common cancer in the U.S., with over half of the 77,240 people
diagnosed annually over age 65. In about 50% of cases, disease recurs or relapses, typically within two years
of initial treatment, and in some patients disease is refractory to additional treatment (Refractory/Relapsed, R/R).
Salvage therapy in these patients consists of stronger chemotherapy cocktails or more recently, cell therapy or
hematopoietic cell transplantation. Four-year survival rates are about 40% with salvage chemotherapy and 60%
for ASCT. Autologous Chimeric Antigen Receptor T (CarT) cell products have triggered great response rates in
clinical trials and real-world use for some types of R/R NHL. However, the therapies are not curative with up to
46% relapsing within 2 weeks to 8.5 months even with negative minimal residual disease (MRD); they are
associated with significant toxicities and are restricted to administration at accredited treatment centers.
AVM Biotechnology’s FDA-approved trial, NCT04329728, is currently ongoing. This clinical trial includes a dose-
escalation study of AVM0703 and a pivotal adaptive-design expansion cohort study in no-option patients with
lymphoid malignancies who failed at least 1 prior regimen, who do not qualify for any other approved therapy,
and who need treatment due to progressive disease. Currently City of Hope, UCLA, Norton Cancer Institute,
Levine Cancer Institute, and University of Texas Southwestern are actively enrolling patients. Illinois Cancer
Care University of Illinois and Holden Comprehensive Cancer Center at University of Iowa are being activated.
Indiana University Melvin and Bren Simon Comprehensive Cancer Center, University of New Mexico
Comprehensive Cancer Center, CHI Health St. Francis Hospital, Advent Health Hendersonville, Moffitt Cancer
Center and MD Anderson Cancer Center will participate in the expansion cohort phase. Patients dosed to date,
in compassionate use and in enrolled dose-escalation cohorts, demonstrate that AVM0703 is distinguished from
other investigative drugs in this ‘terminal no-option’ patient population by its absence of safety concerns, and the
sites report that their patients feel great, regain appetite and energy, and durable response has been recorded.
This proposal requests funding for the adaptive design expansion cohort study. Once the expansion phase dose
has been determined (projected for Nov 2021 based on enrollment, safety and response to date) patients will be
enrolled in 5 NHL sub-indication specific cohorts, requiring about 18 patients per arm to be able to see significant
overall response rates. Adaptive design expansion studies offer a faster way to bring drugs to no-option patients
and this expansion study has been specifically designed to meet FDA guidance for a marketing application.
In the future AVM0703 will be studied in clinical trials in combination with earlier lines of therapy to determine
the potential synergy ...

## Key facts

- **NIH application ID:** 10482324
- **Project number:** 1R44CA272096-01
- **Recipient organization:** AVM BIOTECHNOLOGY, LLC
- **Principal Investigator:** Theresa Deisher
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $999,999
- **Award type:** 1
- **Project period:** 2022-06-10 → 2024-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10482324

## Citation

> US National Institutes of Health, RePORTER application 10482324, Expedited Expansion Cohort Clinical Trial for Relapsed/Refractory 'no-option' Non-Hodgkin's Lymphoma/Leukemia Patients (1R44CA272096-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10482324. Licensed CC0.

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