# Development of a novel small molecule MDM2 inhibitor for innovative sarcoma treatment

> **NIH NIH R44** · LAMASSU BIO INC · 2024 · $723,169

## Abstract

Project summary/abstract
This proposal advances the development of a novel therapeutic for p53 wild type sarcomas. Our team has
identified a novel MDM2 inhibitor, SA53, with best-in-class potency, bioavailability and in vivo efficacy across a
broad range of preclinical models. It has also undergone extensive safety and pharmacokinetic testing, suitable
for support of an IND submission, with an identified starting dose in humans. SA53 has the potential to
facilitate apoptosis in p53 wild-type cancers and to spare normal tissue. To drive proof of concept, we will
conduct a first in human trial to 1) identify a safe dose for future clinical trials, 2) establish the pharmacokinetic
profile and appropriate safety endpoints, 3) in an expansion cohort, establish feasibility and early indication of
efficacy in treating p53 wild type soft tissue sarcoma. Our primary objective is to rapidly and efficiently advance
SA53 through clinical trials to implement a novel and innovative treatment for p53 wild type soft tissue
sarcoma. Our approach is first establishing the recommended phase 2 dose in a trial for p53 wild type
refractory solid tumors, then to apply this novel therapy in an expansion cohort targeting a pre-surgical window
in soft tissue sarcoma patients. To achieve these goals, we will complete the following specific aims:
Aim 1: We will complete GMP manufacturing of the drug and formulated clinical product suitable to complete a
phase 1 clinical trial.
Aim 2: We will conduct a phase 1 clinical trial to establish the recommended dose for phase 2 and early
indication of feasibility and efficacy in soft tissue sarcoma patients.
At the completion of this proposal, we will have completed a phase 1 trial to have established safety and
preliminary efficacy, which will serve as the foundation for registration directed studies. In light of our
exceptional in vivo efficacy data, we further believe that this can be a breakthrough paradigm-changing therapy
for cancer treatment.

## Key facts

- **NIH application ID:** 10900571
- **Project number:** 5R44CA281622-02
- **Recipient organization:** LAMASSU BIO INC
- **Principal Investigator:** Gabi Hanna
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $723,169
- **Award type:** 5
- **Project period:** 2023-08-07 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10900571

## Citation

> US National Institutes of Health, RePORTER application 10900571, Development of a novel small molecule MDM2 inhibitor for innovative sarcoma treatment (5R44CA281622-02). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10900571. Licensed CC0.

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