Agio Pharma Sinks on Midstage Trial

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Agio Pharmaceuticals Inc. (NASDAQ: AGIO) saw its shares sink on Monday after the firm announced results from its midstage trial for the treatment of relapsed or refractory acute myeloid leukemia (R/R AML). Unfortunately, results did not live up to expectations.

Specifically, the firm reported results from its Phase 1/2 study evaluating investigational oral IDHIFA (enasidenib). IDHIFA is in development in collaboration with Celgene Corp. (NASDAQ: CELG).

Data in an oral session at the 22nd Congress of the European Hematology Association (EHA) demonstrated an overall response rate of 37%, including a complete response rate of 20.1% in 214 patients with R/R AML who received enasidenib.

Unfortunately, a maximum tolerated dose was not reached. Although, the overall safety profile observed for enasidenib was consistent with previously reported data. The most common treatment-emergent adverse effects were nausea (48%), diarrhea (41%), fatigue (41%), decreased appetite (34%) and blood bilirubin increased (33%). For all patients in the study, 26.1% had treatment-related serious adverse events, notably IDH differentiation syndrome (7%), leukocytosis (4%), tumor lysis syndrome (3%) and hyperbilirubinemia (2%).

Chris Bowden, M.D., chief medical officer of Agios, commented:

With data from an additional 105 patients and the first look at data from the Phase 2 expansion in R/R AML patients treated at the recommended Phase 2 starting dose of 100 mg once daily, these updated results underscore the consistency and durability of response for enasidenib as a potential first-in-class therapy for patients with relapsed or refractory AML and an IDH2 mutation. We are working with our partner Celgene to quickly bring this oral, targeted therapy to patients with limited treatment options.

Shares of Agio were trading down about 10% at $53.20 on Monday, with a consensus analyst price target of $61.89 and a 52-week range of $35.84 to $67.74.

Celgene was down about 0.7% at $133.33, with a consensus price target of $142.04 and a 52-week trading range of $94.42 to $135.18.