Why This Analyst Is Awaiting More Efficacy Signals For Agios Pharma Candidates

The brokerage is waiting for the company to provide definitive efficacy signals on the rare genetic disease (RGD) program before becoming more bullish. The analyst thinks RGD will be a key value driver in the upcoming years. He also pointed to the company's impressive pipeline in cancer and RDG of metabolism with "five clinical stage compounds."

At this point, Agios is engaged in oncology studies like the AG-221 and AG-120, which have demonstrated preliminary anti-tumor activities in AML. BTIG believes there is limited market opportunity for these.

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Referring to AG-348, the brokerage said in a research note that "early efficacy in hemoglobin improvement in PKD patients was interesting but durability is yet to be seen. We have reservations about AG-348's offtarget aromatase inhibition and lack of a PD response in PKD patients."

The analyst also preferred to wait for clarification from Agios on the additional data and compound to remain bullish on the PKD program. Interestingly, the company would select a compound in PKD by the current year end.

At time of writing Friday, Agios was down 0.96 percent at $40.19.

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