AnHeart Therapeutics Announces Initiation of Global Phase 2 Trial Evaluating Safusidenib for IDH1-Mutant Glioma

NEW YORK--(BUSINESS WIRE)-- AnHeart Therapeutics (“AnHeart”), a global clinical-stage biopharmaceutical company developing novel precision therapies for people with cancer, today announced the first patient has been dosed in a Phase 2 clinical trial, G203, evaluating safusidenib in patients with Grades 2 or 3 recurrent or progressive mutant isocitrate dehydrogenase 1 (mIDH1) glioma, one of the most common types of adult primary brain cancer.

Oncogenic mutations in IDH1 are frequently found in gliomas and current treatment options, including chemotherapy and radiotherapy, can cause significant impact on neurocognition and quality of life. Safusidenib is a novel, selective, potent, oral mIDH1 inhibitor. Safusidenib has shown high blood-brain barrier penetration in both pre-clinical and clinical studies. Encouraging anti-tumor activity was observed in a Phase 1 clinical trial in patients with recurrent IDH1-mutant gliomas.

“IDH1 mutations are now known to be a key driver in the development of IDH-mutant gliomas and we are excited by the potential of IDH targeted therapy for our patients, who tend to be in the prime of their life at diagnosis,” said Professor David Reardon, the leading Principal Investigator of the G203 clinical trial and Clinical Director at the Center for Neuro-Oncology, Dana-Farber Cancer Institute, Boston. “Early data suggest safusidenib may have a role in IDH1-mutant gliomas, notably in high-risk populations, and we look forward to further evaluating this investigational therapy in the G203 trial.”

“This marks an important milestone for AnHeart, as we now have two investigational precision therapies in late-stage clinical trials,” said Jerry Wang, PhD, Chief Executive Officer of AnHeart. “Our mission is to improve the lives of people with cancer. Our strategy is to target known genetic drivers of cancer with the goal of bringing next-generation medicines that raise the bar for what patients should expect from cancer treatment. We look forward to sharing additional updates as we continue to progress our clinical programs.”

About the G203 Phase 2 Safusidenib Trial

G203 (NCT05303519) is a global Phase 2, multicenter, open-label, two-part clinical trial evaluating the efficacy and safety of safusidenib as a monotherapy in approximately 95 patients with recurrent or progressive WHO Grade 2 or Grade 3 IDH1-mutant glioma. The trial is divided into two parts. Part 1 is designed to confirm the optimal dose of safusidenib. Part 2 will evaluate the efficacy and safety of safusidenib in Grade 2 or Grade 3 glioma. Currently, patients are being enrolled in the United States, with expansion to other countries planned for Part 2 of the trial.

About Safusidenib

Safusidenib is a novel, selective, potent, oral mIDH1 inhibitor. Safusidenib has shown high blood-brain barrier penetration in both pre-clinical and clinical studies. Encouraging anti-tumor activity was observed in a Phase 1 clinical trial (NCT03030066) evaluating safusidenib in patients with recurrent or progressive WHO Grades 2 to 4 IDH1-mutant gliomas. In the trial, the objective response rate (ORR) was 33% (4/12) and 17% (6/35) in patients with contrast non-enhancing and enhancing tumors, respectively. Tumors that show enhancement on MRI scans tend to have more vascularization and disruption to the blood-brain barrier and are generally associated with a higher degree of malignancy compared with non-enhancing tumors.

Safusidenib demonstrated a tolerable safety profile. Most adverse events (AEs) were Grades 1 or 2. 43% of patients experienced at least one Grade 3 treatment emergent AE. No Grade 4 or 5 AEs or serious treatment-related AEs were reported.

AnHeart licensed safusidenib from Daiichi Sankyo in 2020 and owns global rights for safusidenib, excluding Japan where Daiichi Sankyo retains development and commercial rights.

About Glioma

Glioma is the most common type of adult brain cancer. Approximately 30,000 patients are diagnosed with IDH1-mutant gliomas in the United States, Europe, and China annually. Patients are typically diagnosed in the prime of their life in their mid-forties to early-fifties. IDH1 mutations are present in the majority of Grade 2 or 3 diffuse gliomas and IDH-mutant gliomas are recognized as distinct tumor subtypes by the World Health Organization (WHO).

There are no targeted treatments approved for IDH-mutant glioma. Current treatment options include surgery, radiation, and chemotherapy, which can cause significant impact on neurocognition and quality of life.

About AnHeart Therapeutics

AnHeart Therapeutics (“AnHeart”) is a global clinical-stage biopharmaceutical company developing novel precision therapies for people with cancer. Our lead investigational therapy, taletrectinib, is a next-generation ROS1-inhibitor currently in pivotal Phase 2 trials for ROS1-positive non-small cell lung cancer (NSCLC). Taletrectinib has been granted Breakthrough Therapy Designation by both the U.S. Food and Drug Administration and the China National Medical Products Administration. AnHeart’s second investigational therapy, safusidenib, is a mIDH1-inhibitor being evaluated in a Phase 2 trial for IDH1-mutant glioma.

Our mission is to improve the lives of people with cancer. We are supported by leading life sciences investors and have built an organization with deep oncology drug discovery and development expertise, with offices in New York and Shanghai. For more information, visit https://www.anhearttherapeutics.com/ or follow us on LinkedIn at https://www.linkedin.com/company/anheart-therapeutics-official/.

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