Agios Pharmaceuticals: Takeaways from our thalassemia expert call on AQVESME launch outlook

Research evidence excerpt

Agios Pharmaceuticals: Takeaways from our thalassemia expert call on AQVESME launch outlook

ed 12-15 adult patients, with the first infusion

expected next month, but emphasized that curative therapy is still not easy for everyone

to access. We think this supports the idea that AQVESME and gene therapy are more

complementary than mutually exclusive, particularly given access, patient preference,

and treatment burden.

SCD remains competitive with approval an open question

In SCD, the physician was familiar with the evolving pyruvate kinase activator landscape

and viewed Novo’s etavopivat as having potential advantages over mitapivat if the VOC

benefit and once-daily dosing are reflected on label (see our thoughts on the data here).

The KOL emphasized variability in VOC timing across patients and studies, but still saw a

positive VOC result as clinically meaningful because it is easy to communicate to

patients. The physician also noted Hb improvement and once-daily dosing as practical

advantages for etavopivat. So while there is a potential accelerated path for mitapivat in

SCD, the competitive bar for uptake has risen after HIBISCUS.

Tebapivat has to rebuild confidence

The physician noted upcoming tebapivat data in SCD as a point to watch, while our

published view is that the LR-MDS discontinuation narrows AGIO’s pipeline optionality

without changing the core stock debate (see our tebapivat data thoughts). The key SCD

question for tebapivat is whether more differentiated PK activation can translate into a

stronger or broader Hb response, and ideally clinical benefit beyond laboratory measures.

After the LR-MDS miss, a clean SCD dataset would help rebuild pipeline momentum,

while a weak readout would leave the story more dependent on AQVESME launch