FLAG Therapeutics Reports Promising Preclinical Data on FLAG-003 for DIPG Treatment

FLAG Therapeutics, Inc., based in Raleigh, N.C., recently revealed promising preclinical data for its leading therapy, FLAG-003. This investigational small molecule is being developed to treat diffuse intrinsic pontine glioma (DIPG) and glioblastoma multiforme (GBM), two aggressive brain tumors. Conducted by researchers at Dana-Farber Cancer Institute, the study demonstrated that FLAG-003 exhibited potent anti-tumor activity in eight DIPG cell lines.

The research involved eight DIPG cell lines chosen based on their genomic and growth properties. These cells were placed in 384-well plates and treated with varying concentrations of FLAG-003, ranging from 1 nM to 50 µM. Positive and negative controls were used for comparison, including Staurosporine and dimethyl sulfoxide (DMSO). The study also evaluated FLAG-003 against ONC201, another investigational compound currently in clinical trials for DIPG. Results showed that FLAG-003 was more effective than ONC201 across all tested cell lines, displaying an IC-50 of less than 100nM.

Dr. Frank Sorgi, President and CEO of FLAG Therapeutics, expressed optimism about these findings, affirming the robust anti-tumor activity of FLAG-003 in preclinical studies. Dr. Sorgi emphasized the company's commitment to advancing this therapy in collaboration with Dr. Katherine E. Warren and her team at Dana-Farber, with plans to initiate a Phase 1 clinical trial in early 2025.

Dr. Warren, a Pediatric Neuro-Oncologist at Dana-Farber and Professor of Pediatrics at Harvard Medical School, also expressed hope, describing FLAG-003 as potentially transformative for treating DIPG. The U.S. Food and Drug Administration (FDA) has recognized FLAG-003 with both Rare Pediatric Disease Designation and Orphan Drug Designation for DIPG and gliomas. FLAG Therapeutics successfully engaged in a Type B meeting with the FDA in May 2024 and aims to file an Investigational New Drug (IND) application by the fourth quarter of 2024.

Diffuse intrinsic pontine glioma (DIPG) is a malignant brain tumor that primarily affects children aged five to nine. This aggressive cancer accounts for about 10 percent of all childhood central nervous system tumors, with around 300 cases diagnosed annually in the United States. Its location in the brain stem makes surgical intervention impossible, and radiation therapy provides only temporary relief, typically extending survival by six to nine months. Currently, no approved drug therapies exist for DIPG.

The Rare Pediatric Disease (RPD) designation by the FDA encourages the development of drugs for rare pediatric diseases, affecting fewer than 200,000 people in the U.S. and children aged 18 and under. This program allows sponsors who gain approval for a drug to qualify for a priority review voucher (PRV), which can expedite future drug reviews or be sold to other developers for significant sums.

FLAG Therapeutics is a biopharmaceutical firm nearing clinical-stage development, focusing on small molecule therapeutics for challenging cancers. The company utilizes three proprietary drug platforms to create multi-specific small molecules targeting cancer cells. FLAG Therapeutics holds a robust intellectual property portfolio, including over 100 issued patents and 30 pending applications, covering both the composition and usage of its drugs. The company benefits from the FDA’s Orphan Drug and Rare Pediatric Drug Designation programs, which offer extended market exclusivity.