FDA Grants Fast Track Designation to Cothera Bioscience’s Zotiraciclib for Treatment of Recurrent High-Grade Glioma with IDH1/2 Mutations

San Mateo, CA and Bethesda, MD — November 26, 2025 — Cothera Bioscience today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to zotiraciclib (ZTR/TG02) for the treatment of patients with recurrent high-grade gliomas (rHGG) harboring IDH1 or IDH2 mutations (IDHmut rHGG). These aggressive brain tumors have no established standard of care, limited treatment options, and poor prognoses. The designation highlights the urgent need for new therapeutic approaches and provides an expedited regulatory pathway to accelerate the development and review of zotiraciclib.

Zotiraciclib is a potent, oral, brain-penetrant multi-kinase inhibitor that primarily targets CDK9. Zotiraciclib suppresses the transcription of short-lived anti-apoptotic proteins, disrupting tumor cell survival. Additionally, zotiraciclib impairs mitochondrial function and suppresses stress-response pathways, creating a synthetic-lethal vulnerability in IDH-mutant gliomas. This dual mechanism supports zotiraciclib as a monotherapy option for IDHmut rHGG, potentially eliminating the need for combination with cytotoxic chemotherapies and improving quality of life for patients. Zotiraciclib represents an important therapeutic advance for devastating brain cancers with urgent unmet needs.

The FDA’s decision was supported by the successful completion of a Phase 1 clinical trial sponsored by the U.S. National Cancer Institute (NCI), part of the National Institutes of Health (NIH), which demonstrated the safety and preliminary efficacy of zotiraciclib as a single-agent therapy for patients with IDHmut rHGG (NCT05588141). Results from this study were presented on November 22–23, 2025, at the 2025 WFNOS and SNO Annual Meeting in Honolulu, Hawaii, in a presentation titled “Phase I/II study of zotiraciclib for recurrent malignant gliomas with IDH mutations: results of the phase I study.”

Presentation Details:
Location: Hawaii Convention Center, Kamehameha Exhibit Hall II & III
Rapid Oral Session: Saturday, November 22, 11:45 am–12:45 pm
Poster Session: Friday, November 21, 11:30 am–12:45 pm
Abstract ID: CTNI-27

“Receiving FDA Fast Track Designation for zotiraciclib marks a pivotal moment in our mission to improve outcomes for patients with IDHmut rHGG,” said Vernon Jiang, Ph.D. EVP of R&D at Cothera Bioscience. “This designation accelerates our efforts to deliver the first breakthrough targeted therapy for this patient population since the approval of temozolomide in 1999.”

“We are grateful to the patients and families who participated in these studies, as well as to our clinical and research teams at the NCI,” said Jing Wu, MD, PhD, Acting Deputy Chief, Neuro-Oncology Branch, NCI, Principal Investigator of the ongoing Phase I/II study. “The monotherapy study data gathered to date provide important insights into the safety and biological activity of zotiraciclib.  We remain committed to advancing zotiraciclib into subsequent phases of development with the goal of bringing a new therapeutic option to patients with IDH-mutant recurrent high-grade gliomas.”

Forward-Looking Statements

Cothera Bioscience will continue to work closely with the FDA and its clinical partners to advance the development of zotiraciclib, including the initiation of additional clinical studies and preparation for regulatory submissions.

Contact Information

For more information, please visit: https://cotherabio.com/

Media Contact:
Keiko Mitsunobu
Head of Business Development
Cothera Bioscience, Inc.
kmitsunobu@cotherabio.com
(650) 773-5690