Agios' top cancer drug notches more remissions on the way to Phase III

Agios CEO David Schenkein

Agios Pharmaceuticals' lead cancer drug extended its streak of promising clinical results, racking up remissions in an expanded Phase I trial as the company ($AGIO) prepares for late-stage testing.

In data from patients with tough-to-treat blood cancers, Agios' AG-221 charted a measurable response in 63 of 158 patients, a 40% rate. Among those patients, 16% went into complete remission, the company said, added to 19 remissions with various degrees of hematologic recovery and 18 partial remissions. And Agios continues to be pleased with the drug's durability, as 76% of responses lasted more than 6 months, and some patients stayed on the drug beyond 15 months.

The latest efficacy data, presented at the annual European Hematology Association meeting in Vienna, build on positive results from the same program disclosed in December. Previously, AG-221 came through with a 56% response rate and 6 complete remissions. Agios has since added more than 100 patients to the trial, and the biotech believes AG-221's continued results suggest a strong clinical profile.

The company is enrolling yet another expansion group to the study, recruiting 125 more patients with serious acute myeloid leukemia to add to its existing results in AML, myelodysplastic syndrome and chronic myelomonocytic leukemia. Throughout AG-221's development, Agios has employed a series of small, blended trials to quickly gather safety and efficacy on its lead drug, a process designed to shave months off of development and establish AG-221's wide potential in blood cancers.

Now Agios has its eye on Phase III, working with partner Celgene ($CELG) to design a late-stage trial and begin enrollment in the second half of this year. The biotech has yet to disclose just which patient populations it'll target.

Meanwhile, at the weekend's EHA meeting, Agios is making 8 total presentations, providing a glimpse at early data on the Celgene-partnered AG-120, also for blood cancers, and unveiling healthy-patient safety results for AG-348, a treatment for a rare disease called rare pyruvate kinase deficiency. The biotech has already kicked off a Phase II trial on AG-348 and plans to get AG-120 into Phase III studies in early 2016.

Those ambitions make 2015 a pivotal year for Agios, CEO David Schenkein said. And, in addition to executing on its three clinical assets, the company is preparing itself for the long haul, he said, investing in the scientific, regulatory and clinical capacity it will need to follow through on inventions to come.

"Agios at its heart and soul is a research organization," Schenkein said. "Every molecule is homemade and discovered at our laboratories. And behind these molecules are the second, third and fourth wave of what we think will be important programs."

- read the AG-221 results

Special Reports: The 25 most influential people in biopharma in 2015 - David Schenkein - Agios Pharmaceuticals | FierceBiotech's 2009 Fierce 15 - Agios Pharmaceuticals

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