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Agios Announces the Randomized Phase 3 ClarIDHy Trial of TIBSOVO (ivosidenib) Achieved its Primary Endpoint in Previously Treated IDH1 Mutant Cholangiocarcinoma Patients

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3.6 from 5 votes
Thursday, May 16, 2019

CAMBRIDGE, Mass. - Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, announced that the global Phase 3 ClarIDHy trial of TIBSOVO® (ivosidenib) in previously treated cholangiocarcinoma patients with an isocitrate dehydrogenase 1 (IDH1) mutation met its primary endpoint. Treatment with TIBSOVO® demonstrated a statistically significant improvement in progression-free survival (PFS) by independent radiology review compared with patients who received placebo. The safety profile observed in the study was consistent with previously published data.

A full analysis of the ClarIDHy trial will be submitted for presentation at the European Society for Medical Oncology Congress taking place in Barcelona, Spain from September 27-October 1, 2019. The company plans to submit a supplemental new drug application for TIBSOVO® in previously treated IDH1 mutant cholangiocarcinoma by the end of 2019.

“Advanced cholangiocarcinoma is a life-threatening disease with no currently approved treatment options,” said Chris Bowden, M.D., chief medical officer at Agios. “The data from the ClarIDHy Phase 3 trial demonstrate the clinically significant benefit of TIBSOVO® in patients with this challenging disease who harbor the IDH1 mutation. We are committed to working with regulators to bring this potential treatment option to patients as quickly as possible. We thank the patients and physicians who participated in the ClarIDHy study, without whom this important advancement would not be possible.”

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. As of the January 31, 2019 data cutoff, 185 patients were randomized.

  • 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.
  • The primary endpoint of the trial is PFS as evaluated by independent radiology review with secondary endpoints including investigator evaluated PFS, safety and tolerability, overall response rate, overall survival, duration of response, PK/PD and quality of life assessments.
  • The study was designed with 96% power to detect a hazard ratio of 0.5 for PFS (TIBSOVO® vs. placebo), with a one-sided alpha of 0.025.
  • Thermo Fisher Scientific is providing next-generation sequencing to detect IDH1 mutations for all tumor samples as inclusion criteria for enrollment in the study and will develop and commercialize the validated companion diagnostic.

TIBSOVO® is not approved in any country for the treatment of patients with advanced cholangiocarcinoma.

About TIBSOVO® (ivosidenib)

TIBSOVO® is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

  • Adult patients with newly-diagnosed AML who are ≥75 years old or who have comorbidities that preclude use of intensive induction chemotherapy
  • Adult patients with relapsed or refractory AML

    About Cholangiocarcinoma
    Cholangiocarcinoma (CC) is a rare cancer of the bile ducts within and outside of the liver. Cases that occur within the liver are known as intrahepatic cholangiocarcinoma (IHCC) and those that occur outside the liver are considered extrahepatic. Mutations in IDH1 occur in up to 20% of IHCC cases. Current treatment options for localized disease include surgery, radiation and/or other ablative treatments. There are no approved systemic therapies for cholangiocarcinoma and limited chemotherapy options are available in the advanced setting. Gemcitabine-based chemotherapy is often recommended for newly diagnosed metastatic disease.

    About Agios
    Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism and adjacent areas of biology. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has two approved oncology precision medicines and multiple first-in-class investigational therapies in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company's website at www.agios.com.

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Source: agios
3.25
3.6 from 5 votes
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