Agios Presents Final Data from Phase 3 ClarIDHy Study of TIBSOVO® (ivosidenib tablets) in Patients with Previously Treated IDH1-Mutant Cholangiocarcinoma
January 17, 2021 at 10:00 am EST
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Agios Pharmaceuticals, Inc. reported a full analysis of the final data, including mature overall survival (OS) results, from its global Phase 3 ClarIDHy trial of TIBSOVO® (ivosidenib tablets) in patients with previously treated isocitrate dehydrogenase 1 (IDH1) mutated cholangiocarcinoma. Data from the study were featured in an oral presentation at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO-GI), which is being held virtually January 15-17, 2021. The final analysis showed an improvement in the secondary endpoint of OS favoring patients randomized to TIBSOVO® compared to those randomized to placebo; however, statistical significance was not reached. The median OS for patients randomized to TIBSOVO was 10.3 months compared to 7.5 months for patients randomized to placebo (hazard ratio [HR]=0.79; 95% CI [0.56–1.12], 1-sided p=0.093). The protocol specified that patients randomized to placebo could cross over to TIBSOVO at the time of disease progression, and a high proportion of patients in the placebo arm (70.5%) crossed over to TIBSOVO. The results of a pre-specified analysis to adjust for crossover, based on the rank-preserving structural failure time (RPSFT) model, showed a median OS for patients in the placebo arm of 5.1 months (HR=0.49, 95% CI 0.34–0.70, 1-sided p<0.0001). The safety profile observed in the study was consistent with previously published data. As previously announced, the study demonstrated a statistically significant improvement in the primary endpoint of progression-free survival (PFS) by independent radiology review.
Agios Pharmaceuticals, Inc. is a biopharmaceutical company. The Company is focused on developing and delivering transformative therapies for patients living with rare diseases. It markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for debilitating hemolytic anemia. Its lead product candidate in its genetically defined disease (GDD) portfolio, PYRUKYND (Mitapivat), is an activator of both wild-type and a variety of mutant pyruvate kinase (PK) enzymes, for the treatment of hemolytic anemias. The Mitapivat is an orally available small molecule and a potent activator of the pyruvate kinase R (PKR) enzymes. In addition, it is also evaluating mitapivat for the treatment of alpha- and beta- thalassemia and sickle cell disease (SCD) in ongoing clinical trials. The Company is also developing AG-946, a clinical-stage oral activator of PKR enzymes for the treatment of hemolytic anemias and other indications, including SCD.
Agios Presents Final Data from Phase 3 ClarIDHy Study of TIBSOVO® (ivosidenib tablets) in Patients with Previously Treated IDH1-Mutant Cholangiocarcinoma
AGIOS PHARMACEUTICALS, INC. 
