# BMX-001 as a Therapeutic Agent for Treatment of Multiple Brain Metastases.

> **NIH NIH R44** · BIOMIMETIX JV, LLC · 2020 · $1,000,000

## Abstract

PROJECT SUMMARY/ABSTRACT
In the United States an estimated 1.7 million patients will be newly diagnosed with cancer in 2017 and
approximately 15% of these patients will develop brain metastases. The most common treatment for
brain metastases is whole brain radiation therapy (WBRT). The primary limitation to WBRT is
neurotoxicity, including brain white matter damage, cognitive impairment and quality of life impairment.
BMX-001 is a new class of pharmaceutical (metalloporphyrin) that in preclinical studies has been
shown to provide marked protection against radiation-induced tissue damage and at the same time to
augment tumor growth inhibition. BMX-001 is redox-active, scavenges reactive oxygen species and
inhibits transcriptional activity of key inflammation-related nuclear transcription factors such as NFKB
and HIF-1. In animal models BMX-001 has been shown to prevent hippocampal stem cell loss and
white matter degradation after WBRT. BMX-001 is currently in a Phase 1 clinical trial of patients with
high-grade glioma undergoing primary chemoradiation therapy. No adverse drug-related events have
identified, and the Phase 1 dose escalation trial will be completed by December 2017 with identification
of a maximum tolerated dose (MTD) that can be used for a Phase 2 clinical trial. Chemoradiation of
high-grade glioma involves localized brain radiation therapy. In contrast, patients with multiple brain
metastases (MBM) receive whole brain irradiation under a different dose and protocol and have a
somewhat different toxicity profile. This proposed fast-track SBIR will support a Phase 1 safety lead-in
clinical trial of BMX-001 in combination with WBRT in 5 patients with MBM. The dose will be the MTD
selected from the current clinical trial of patients with high-grade glioma undergoing brain
chemoradiation therapy. Demonstrating safety of the selected MTD of BMX-001 in patients with MBM
undergoing standard protocol WBRT will be the “go/no-go” criteria for proceeding to Phase II of this
SBIR. Phase II will be a randomized open-label Phase 2 clinical trial of 50 patients with MBM, half
receiving BMX-001 in combination with WBRT and half receiving WBRT alone. The hypothesis being
tested is that BMX-001 is an effective radioprotector in WBRT of patients with MBM from extracranial
primary tumors. The primary proposed outcome is protection/improvement of cognition. Secondary
outcomes are 1) efficacy based on overall and progression-free survival, 2) median distant brain new
metastases rate, and 3) rate of death from neurologic causes. Exploratory outcomes are 1) quality of
life and 2) hair loss. The proposed randomized 50-patient trial will provide essential data on the efficacy
of BMX-001 in modifying the neurotoxicity of WBRT and will enable design and implementation of
future expanded trials with sufficient power to demonstrate drug efficacy and move this new class of
pharmaceuticals toward appropriate commercialization.

## Key facts

- **NIH application ID:** 10064765
- **Project number:** 4R44CA228694-02
- **Recipient organization:** BIOMIMETIX JV, LLC
- **Principal Investigator:** David S. Silberstein
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,000,000
- **Award type:** 4N
- **Project period:** 2018-07-06 → 2021-12-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10064765

## Citation

> US National Institutes of Health, RePORTER application 10064765, BMX-001 as a Therapeutic Agent for Treatment of Multiple Brain Metastases. (4R44CA228694-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10064765. Licensed CC0.

---

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