On heels of a Celgene deal, Agios adds promising data on leukemia drug

Agios CEO David Schenkein

On Friday, Celgene ($CELG) followed up on its aggressive $130 million upfront deal to land an option on Agios' experimental therapies by locking in development and commercialization rights to its lead drug, AG-221. On Saturday, Agios ($AGIO) explained why, detailing Phase I data that showed an objective response among more than half of the evaluable patients and a complete response from 6 of 25.

The promising data builds on positive numbers gathered for the first evaluation of 7 patients two months ago, keeping the biotech and its big partner on track with a drug both believe could provide an important advance for leukemia patients..

Investigators examined the impact of AG-221 on IDH2-mutant positive hematologic malignancies, releasing the results at the European Hematology Association meeting in Milan. Lead investigator Stephane de Botton said that the 6 complete responders in the small bunch also exhibited some durability of response, ranging from one to four months. In addition, none of the patients dropped out due to adverse events in the study and the maximum tolerated dose has yet to be achieved--all positive signs early on in the clinical development of this drug.

One of the big questions lingering from the first look at the data was whether the drug's effect would last long, Agios CEO David Schenkein tells FierceBiotech. The new data "gives us a very encouraging look at durability." "The safety profile is excellent," he adds, "but were seeing a degree of efficacy that is really very encouraging to us."

Agios, a 2009 Fierce 15 company, isn't mapping out a public timeline on the development plan, but Schenkein says the Phase I results open the door to recruiting "multiple expansion cohorts in this study in more homogeneous patient populations. And that will set up faster development" of this drug. On Monday Agios followed up with the news that the FDA has granted 221 orphan drug status.

The early success of this drug is critical to Agios. The biotech's deal with Celgene gives it a shot at sharpening up its commercial skills as it preps for a solo U.S. rollout of a separate drug, AG-120, also in an early-stage study. Agios now has about 100 staffers, says Schenkein, who has been adding development staff as he builds a company that intends to build a commercial infrastructure in the not distant future.

The latest batch of data helps flesh out the preliminary glimpse of the drug's effect back in April. Agios reported then that of the 10 patients initially treated with AG-221, three died of their disease and 7 were evaluable. Of those 7, three achieved complete remission, erasing signs of the cancer, two patients achieved complete remission with incomplete platelet recovery and one patient demonstrated a partial response. And another data drop is due out when ASH rolls around.

"These data demonstrate that AG-221 treatment leads to a profound differentiation effect and is associated with durable complete remissions in patients who are extremely ill and have limited treatment options," said Dr. de Botton in a statement. "We believe these data support comprehensive investigation of AG-221 in IDH2 mutant positive cancers and look forward to participating in Agio's planned expansion cohorts and future clinical trials."

- here's the release

Special Report: 2009 Fierce 15 - Agios Pharmaceuticals