SAN FRANCISCO (TheStreet) -- Agios Pharmaceuticals (AGIO) and partner Celgene (CELG) will start pivotal studies of their cancer metabolism pill AG-221 in 2015 designed to support worldwide approvals, the companies announced Sunday.
Updated results from an early-stage study of AG-221 continue to show strong and durable clinical activity. More than half of the patients with advanced blood cancers are responding to treatment with AG-221, including some responses lasting as long as eight months. One-third of patients in the study achieved a complete remission or a near-complete remission following treatment with AG-221. Researchers see two deaths in the trial possibly related to AG-221.
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A presentation of the AG-221 phase I study data is scheduled for later today at the American Society of Hematology annual meeting underway here.
Agios and Celgene are able to move quickly from a phase I study directly into studies designed for regulatory approval because AG-221, a pill taken once or twice per day, targets a specific genetic mutation found in some cancer cells. By identifying and enrolling only those patients with tumors expressing this mutation into its studies, response rates to AG-221 are significantly better than what's normally seen with traditional chemotherapy.
Agios developed AG-221 and has shepherded the drug through early clinical development. Celgene licensed worldwide rights to AG-221 from Agios in June 2014.
The "global registration program" for AG-221 will begin in 2015, but Agios and Celgene are not providing details on the studies or a more exact timeline.
In this latest update to the phase I study, 73 patients with advanced blood cancers, mostly acute myeloid leukemia no longer responding to current medicines, have been enrolled and treated with escalating doses of AG-221. All the patients have tumors containing a mutated, metabolic enzyme known as IDH2. Normally, cells use IDH2 to help break down nutrients and generate energy. When mutated, IDH2 alters the genetic programming of cells, preventing them from maturing and causing them to grow uncontrollably.
Patients with AML have bone marrow crowded with undifferentiated, rapidly dividing blast cells. AG-221 blocks the mutated IDH2 enzyme and allows the leukemic cells in the bone marrow to mature into normal blood cells.
Agios estimates that between 18-24% of AML patients harbor the IDH2 mutations or a similar IDH1 mutation.