UPDATED: Agios shares jump on another round of 'exciting' early data for cancer drug

Agios CEO Dr. David Schenkein

Following up on positive Phase I data for its lead therapy, Agios has come through with its first snapshot of promising early results on its second cancer drug, gaining some fresh proof-of-concept data on acute myeloid leukemia to help guide an expanding clinical program.

Cambridge, MA-based Agios ($AGIO) held on to U.S. commercial rights to AG-120, an IDH1 inhibitor, when the biotech forged its big partnership deal with Celgene ($CELG). And in the first glimpse at efficacy in the dose-escalating Phase I trial, investigators tracked an objective response in 7 of 14 patients with refractory or relapsed AML. Four of those 7 were in complete remission--an impressive achievement. The maximum tolerated dose has not yet been reached in the early study, though according to a company statement, one of the patients had a "dose limiting toxicity of asymptomatic grade 3 QT prolongation at the highest dose tested to date, which improved to grade 1 after AG-120 dose reduction according to treatment protocol." That is one of the patients in complete remission.

Agios' high-profile CEO, Dr. David Schenkein, says it's "exciting" to see these kinds of results, particularly considering that Agios was little more than a blank sheet of paper just 5 1/2 years ago--not long before it was named a Fierce 15 company in 2009. The biotech went on to score one of the hottest IPOs during the opening round of the biotech boom on Wall Street, due in part to the rep Schenkein brought to the company after completing a stint at Genentech.  

Investors were still enthusiastic on Wednesday. Agios' shares surged 17% as the news spread.

Of particular note in the data today, says Schenkein, is early evidence of durability in patients' response to the drug, with one patient with very limited life expectancy going 5.5 months on therapy. And that matches well with encouraging data gathered on the biotech's other closely watched development program for AG-221.

Investigators examined the impact of Agios' AG-221 on IDH2-mutant positive hematologic malignancies, releasing the results at the European Hematology Association meeting last summer. The Phase I data available showed an objective response among more than half of the evaluable patients and a complete response from 6 of 25 patients. Lead investigator Dr. Stéphane de Botton said at the time that the 6 complete responders in the small bunch also exhibited a durable response, ranging from one to four months. In addition, none of the patients had yet to drop out due to adverse events.

Agios is a specialist in cancer metabolism, focusing on targets that play a big role in feeding tumors. And Celgene's option deal on 221, which it exercised just before the Phase I results were released, was seen as a foundational relationship that tightly bound the biotech upstart to a major player in oncology.

"Together with the data we reported from our ongoing Phase I study of AG-221 in advanced hematologic cancers, our lead investigational medicine and an IDH2-mutant inhibitor, we believe IDH inhibitors could potentially change the treatment paradigm for AML patients with these mutations," said Dr. Chris Bowden, chief medical officer of Agios, in the release. "We look forward to moving rapidly into multiple expansion cohorts in the first half of 2015 to further characterize the potential of AG-120." 

- here's the release

Special Report: 2009 Fierce 15 - Agios Pharmaceuticals