On March 20, 2023 FORE Biotherapeutics (FORE Bio) a precision stage oncology company dedicated to developing innovative treatments that provide a better outcome for cancer patients reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead program, FORE8394, for the treatment of primary brain and central nervous system (CNS) malignancies (Press release, Fore Biotherapeutics, MAR 20, 2023, View Source [SID1234629068]). This is the first orphan drug designation received by FORE Bio and the FORE8394 program.

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FORE8394 is an investigational, novel, small-molecule, next-generation, orally available selective inhibitor of mutated BRAF being evaluated in FORTE, a global Phase 2 clinical study in adult and pediatric participants with advanced unresectable solid or primary CNS tumors harboring BRAF alterations (NCT05503797).

"The receipt of Orphan Drug Designation is another important regulatory achievement that reinforces the FDA’s recognition of the potential of FORE8394 to improve clinical outcomes in patients with BRAF-altered brain tumors," said Stacie Shepherd, M.D., Ph.D., Chief Medical Officer of Fore Biotherapeutics. "This designation will help us continue to expedite the development of our novel BRAF inhibitor, and we look forward to working closely with the global investigator community supporting FORTE and to advancing the development of FORE8394 for patients in need."

Matthew Ros, FORE’s CEO, commented, "available treatments are limited for patients with primary recurrent CNS tumors or those patients living with solid tumors with a BRAF fusion. With its unique mechanism of action as a paradox breaker, we believe we have a significant opportunity to deliver a novel and best in class potential therapeutic option with FORE8394. We look forward to accelerating enrollment in the Phase 2 Forte master protocol throughout 2023."

Orphan Drug Designation is granted to drugs or biological products for the treatment of rare diseases or conditions that impact fewer than 200,000 people in the United States. FORE8394 was granted Fast Track Designation by the U.S. Food and Drug Administration in September 2022 for the treatment of patients with cancers harboring BRAF Class 1 (V600) and Class 2 (including fusions) alterations who have exhausted prior therapies.

About FORE8394

FORE8394 is an investigational, novel, small-molecule, next-generation, orally available selective inhibitor of mutated BRAF. It was designed to target a wide range of BRAF mutations while sparing wild-type forms of RAF. Preclinical studies and clinical trials have shown that its unique mechanism of action effectively inhibits not only the constitutively active BRAFV600 monomers targeted by first-generation RAF inhibitors but also disrupts constitutively active dimeric BRAF class 2 mutants, fusions, splice variants and others. Unlike first-generation RAF inhibitors, FORE8394 does not induce paradoxical activation of the RAF/MEK/ERK pathway. As a "paradox breaker," FORE8394 could therefore treat acquired resistance to current RAF inhibitors and, more generally, yield improved safety and more durable efficacy than first-generation RAF inhibitors. The company previously announced interim data from the ongoing Phase 1/2a clinical trial evaluating FORE8394 in advanced solid and CNS tumors with activating BRAF alterations, providing evidence of durable anti-tumor activity in patients with BRAF-mutated (V600+) cancers. The data were presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress (ESMO) (Free ESMO Whitepaper) in September 2022. Mature data from the Phase 1/2a clinical trial is expected in mid-2023.