Servier Expands Tibsovo Program for IDH1-Mutated Cancer Safety and Efficacy

Servier has recently provided updates on two of its key Phase 3 clinical trials involving TIBSOVO (ivosidenib tablets) for treating cancers with isocitrate dehydrogenase 1 (IDH1) mutations. The first patient has been enrolled in the CHONQUER study, which is a pivotal Phase 3 clinical trial examining the effectiveness and safety of TIBSOVO compared to a placebo in patients with IDH1-mutated conventional chondrosarcoma. Additionally, the Phase 3 PyramIDH clinical trial has commenced, with sites actively recruiting worldwide. This trial is focused on assessing TIBSOVO as a monotherapy and azacitidine monotherapy for treating patients with IDH1-mutated myelodysplastic syndromes (MDS), who have not yet received treatment with a hypomethylating agent.

Susan Pandya, MD, Vice President of Clinical Development and Global Head of Oncology LS/LCM at Servier Pharmaceuticals, noted the company's commitment to advancing treatment options for patients with newly diagnosed IDH1-mutated conventional chondrosarcoma and IDH1-mutated MDS. These trials mark a significant milestone in the TIBSOVO clinical development program, highlighting Servier's dedication to exploring the application of IDH1 inhibition across various malignancies to cater to patient needs. Pandya expressed gratitude to the patients, families, and healthcare providers participating in these crucial studies.

The use of TIBSOVO for patients with IDH1-mutated conventional chondrosarcoma and those with IDH1-mutated MDS who have not previously undergone treatment with a hypomethylating agent is currently under investigation. Its safety and efficacy in these specific cases have not yet been evaluated by any regulatory authority.

The PyramIDH trial (NCT06465953) is a pivotal Phase 3 multicenter, open-label, randomized clinical study of TIBSOVO and azacitidine monotherapy in adult patients with IDH1-mutated MDS who have not been treated with a hypomethylating agent. The primary objective is to achieve complete or partial remission as per the International Working Group 2006 criteria at four months. Secondary goals include overall response rate, event-free survival, and overall survival.

The CHONQUER trial (NCT06127407) is a pivotal Phase 3 multicenter, double-blind, randomized, placebo-controlled crossover trial evaluating TIBSOVO in adults with locally advanced or metastatic conventional chondrosarcoma with an IDH1 mutation. Participants either have not received treatment or were previously treated with one systemic regimen in the advanced or metastatic setting for conventional chondrosarcoma. The main endpoint is progression-free survival in Grades 1 and 2 participants, with secondary outcomes being progression-free survival and overall survival across all randomized participants. The trial will also assess the impact of TIBSOVO on health-related quality of life and health economic outcomes.

TIBSOVO is a precision medicine targeting a specific mutation type known as IDH1 and is approved for five indications globally, including in the U.S., EU, Australia, and China. In the U.S., it's approved for treating adults with IDH1-mutant relapsed or refractory AML and as a monotherapy or in combination with azacitidine for adults with newly diagnosed IDH1-mutant AML who are 75 years or older or have comorbidities preventing the use of intensive induction chemotherapy. It's also approved for treating adult patients with IDH1-mutant relapsed or refractory MDS and previously treated IDH1-mutated cholangiocarcinoma.

Chondrosarcoma is a group of bone tumors characterized by cells that produce excess cartilage and is the second most common bone sarcoma following osteosarcoma. Conventional primary chondrosarcoma is the most common variant, making up 85% of cases. IDH mutations are found in 50-70% of chondrosarcomas, and surgery remains the primary treatment, as radiation and chemotherapy have proven ineffective.

Servier, a global leader in oncology, is governed by a non-profit foundation. The company prioritizes innovation with a long-term vision, free from fiduciary influences, and dedicates over 65% of its research and development budget to oncology. Servier aims to advance targeted therapies by identifying mutations and understanding their impact on cancer and its progression.