FLAG Therapeutics Announces Encouraging Preclinical Data from Studies of FLAG-003 for the Treatment of Diffuse Intrinsic Pontine Glioma (DIPG)
16 Julio 2024 - 8:00AM
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 DIPGDiffuse
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 DesignationThe 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:Investor RelationsFLAG
Therapeutics, Inc.+1 (919) 294-6472info@flagtherapeutics.com