Agios Announces Final Overall Survival Data from Phase 3 ClarIDHy Study of TIBSOVO (ivosidenib tablets) in Previously Treated IDH1-Mutant Cholangiocarcinoma Patients

– Supplemental New Drug Application Planned for Submission in Q1 2021 –

CAMBRIDGE, Mass., Sept. 21, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced the results of the final overall survival (OS) analysis from its global Phase 3 ClarIDHy trial of TIBSOVO (ivosidenib tablets) in previously treated cholangiocarcinoma patients with an isocitrate dehydrogenase 1 (IDH1) mutation. A consistent trend in improved OS was observed in patients treated with TIBSOVO compared to those randomized to placebo, but was not statistically significant. The OS endpoint can be affected by crossover, so these results should be taken in the context of the large proportion (70%) of patients in the placebo arm who crossed over to receive TIBSOVO following radiographic disease progression; additional analyses performed to take crossover into account further support that TIBSOVO may improve OS. The safety profile observed in the study was consistent with previously published data. OS was a secondary endpoint in the ClarIDHy study; as previously announced, the study met its primary endpoint of progression-free survival (HR 0.37, p-value < 0.0001).

“Advanced cholangiocarcinoma is a rapidly progressing, aggressive disease with a grim prognosis for patients,” said Chris Bowden, M.D., chief medical officer at Agios. “The data from the ClarIDHy Phase 3 study show that treatment with TIBSOVO has the potential to lengthen time to disease progression and have a clinically meaningful impact on life expectancy for patients with IDH1-mutant cholangiocarcinoma. We will collaborate closely with regulators to advance this potential new oral, targeted treatment option for patients.”

“We are tremendously grateful to the patients who participated in this study,” continued Dr. Bowden. “Part of their legacy is their commitment to contributing to medical advances on behalf of others who will face this devastating disease and who currently have limited treatment options.”

The company plans to submit a supplemental new drug application for TIBSOVO in previously treated IDH1-mutant cholangiocarcinoma in the first quarter of 2021 and intends to work closely with regulators on next steps. A full analysis of the ClarIDHy OS data will be submitted for presentation at a future medical meeting.

ClarIDHy Phase 3 Trial
The ClarIDHy trial is a global, randomized Phase 3 trial in previously treated IDH1-mutant cholangiocarcinoma patients who have documented disease progression following one or two systemic therapies in the advanced setting. Patients were randomized 2:1 to receive either single-agent TIBSOVO 500 mg once daily or placebo with crossover to TIBSOVO permitted at the time of documented radiographic progression per RECIST 1.1. As of the May 30, 2020 data cutoff, 185 patients were randomized, with 124 patients in the TIBSOVO arm and 61 patients in the placebo arm. Forty-three patients randomized to placebo (70%) crossed over to open-label TIBSOVO upon radiographic disease progression and unblinding.