Goldman Sachs: 3 Questions For Agios Pharmaceuticals

Agios Pharmaceuticals Inc AGIO reported interim data from an ongoing open label Phase 2 trial of AG348 in pyruvate kinase deficiency [PKD], a rare genetic blood disorder, and from the Phase 1 trial of AG509 in healthy volunteers. Goldman Sachs’ Terence Flynn maintained a Neutral rating for the company, with a price target of $46, citing safety issues.

Unanswered Questions

Analyst Terence Flynn mentioned that the interim ‘348 data achieved proof ¬of ¬concept and offered additional validation of Agios Pharmaceuticals’ platform. The analyst believes that several questions still remain unanswered. These include:
Does treatment with ‘348 lead to a significant change in hemoglobin?
Does the Hb data correlate with the genotype data?
Does ‘519 have a better safety profile than ‘348?

AG¬519 had “comparable biochemical, cellular and in vivo activity to AG¬348 and no aromatase inhibitory activity has been observed pre¬-clinically and no sex hormone effects were reported in healthy volunteers,” the analyst said.

Flynn pointed out that the MAD portion of the trial was ongoing and thus there would be additional cohorts of patients offering further insight into the frequency of thrombocytopenia.

Agios Pharmaceuticals is expected to make a decision on the advancement of one of its drugs into a pivotal trial later this year. The company intends to take the decision on the basis of the totality of data, including additional data from the ongoing '348 Ph2 trial and from '519 Ph1 MAD trial.

“While we are incrementally more positive on the clinical proof¬ of¬ concept for AGIO's PKD program, the safety profile of both drugs leaves outstanding questions,” Flynn wrote.