Investigational New Drug (IND) application to be filed in the fourth quarter of 2024
Phase 1 clinical trial in children with DIPG to be initiated in early 2025
RALEIGH, N.C., July 16, 2024 (GLOBE NEWSWIRE) -- FLAG Therapeutics, Inc. today announced preclinical data from the company’s lead program, FLAG-003, an investigational small molecule therapy for the treatment of diffuse intrinsic pontine glioma (DIPG) and glioblastoma multiforme (GBM). Data from this study, which was conducted by researchers from Dana-Farber Cancer Institute, showed that single agent FLAG-003 resulted in potent anti-tumor activity in all eight DIPG cell lines tested. Moreover, in all cell lines evaluated, FLAG-003 demonstrated greater potency than ONC201, an investigational compound in clinical-stage development. These data were presented at the recent 21st International Symposium on Pediatric Neuro-Oncology (Abstract/Poster DIPG-68) held in Philadelphia, Pennsylvania.
Study Overview
Eight DIPG cell lines hosted at the Center for Patient Derived Models were selected based on genomic and growth characteristics. Cells were plated on 384-well plates; 24 hours after plating, cells were dosed with 1 nM to 50 µM (12 step log curve) of FLAG-003. Three technical replicates were performed per step. Controls consisted of positive kill control Staurosporine, negative growth control of 0.5% dimethyl sulfoxide (DMSO), or no DMSO for baseline growth. ONC201, an investigational small molecule antagonist of DRD2/3 currently in clinical trials for DIPG, was used as a comparator. Two GBM patient-derived models were also evaluated. FLAG-003 demonstrated efficacy in all DIPG and GBM cell lines tested with an IC-50 less than 100nM. FLAG-003 was more potent compared to ONC201 in all models evaluated.
“The positive results from the Dana-Farber Research team supports earlier preclinical in vitro and in vivo (including completed toxicology) studies that show the robust anti-tumor activity of FLAG-003 against this highly aggressive, deadly form of pediatric brain cancer,” stated Frank Sorgi, Ph.D., President and Chief Executive Officer of FLAG Therapeutics. Dr. Sorgi continued, “With these encouraging results, we look forward to our continued collaboration with Dr. Warren and her team at Dana-Farber and remain focused on our goal to initiate a Phase 1 clinical study in children with DIPG in early 2025.”
Dr. Katherine E. Warren, Pediatric Neuro-Oncologist, Faculty, Dana-Farber Cancer Institute, Professor of Pediatrics, Harvard Medical School, stated, “These preclinical in vitro studies of FLAG-003 are promising and I believe FLAG-003 could be a game-changer in this disease.”
The U.S. Food and Drug Administration (FDA) has granted FLAG-003 Rare Pediatric Disease Designation and Orphan Drug Designation for DIPG and for gliomas, respectively. In May 2024, FLAG Therapeutics conducted a successful Type B meeting with the FDA regarding FLAG-003 and intends to file an Investigational New Drug (IND) application in the fourth quarter of 2024 and to initiate Phase 1 clinical studies in DIPG in early 2025.
About Childhood DIPG
Diffuse intrinsic pontine glioma (DIPG) is a highly aggressive, difficult to treat malignant brain tumor that is usually diagnosed in children between the ages of five and nine. It accounts for nearly 10 percent of all childhood central nervous system tumors. In the United States, approximately 300 children are diagnosed with DIPG each year. Due to its location in the brain stem, surgery is not an option; results of first line treatment with radiation are typically short lived, lasting on average approximately six to nine months. 1 At present, there are no approved drug therapies for the treatment of DIPG.
About RPD Designation
The Rare Pediatric Disease (RPD) designation program is offered by the FDA to encourage the development of new drugs for rare pediatric diseases (diseases affecting children 18 years of age and younger and fewer than 200,000 people in the United States). Under the RPD program, a sponsor who receives approval for a drug or biologic for a rare pediatric disease for which the RPD designation has been granted, may qualify for a priority review voucher (PRV) at the time of market approval. Holders of a PRV can redeem the voucher to obtain priority review, which shortens review from 10-months to 6-months, for any subsequent marketing application, or they can sell or transfer it to other developers. Recently, PRVs have commanded sale prices between $95 million to $125 million.
About FLAG Therapeutics, Inc.
FLAG Therapeutics is a near-term clinical stage biopharmaceutical company focused on the development of novel small molecule therapeutics for difficult-to-treat cancers. The company leverages its three proprietary drug platforms to develop novel multi-specific small molecule therapeutics designed to specifically target, bind, and kill cancer cells through well-established mechanisms of action. FLAG Therapeutics has a robust intellectual property (IP) portfolio surrounding all of its drug discovery platforms as well as the potential for extended market exclusivity offered through the U.S Food and Drug Administration’s Orphan Drug Designation and Rare Pediatric Drug Designation programs for qualifying indications. To date, FLAG’s IP portfolio includes over 100 issued patents and over 30 pending applications covering both composition of matter and method of use. For more information, please visit the company’s website at www.flagtherapeutics.com.
1 Source https://www.dana-farber.org/childhood-diffuse-intrinsic-pontine-glioma
Contact:
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FLAG Therapeutics, Inc.
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