Shares in Agios Pharmaceuticals (AGIO +0.37%) crashed by 26.8% in the week as of the time of writing. The move comes after a much larger pharmaceutical company, Novo Nordisk (NVO +0.58%), announced top-line results from a class of drug to treat sickle cell disease (SCD) that implied superiority over Agios' rival drug mitapavit.
Novo Nordisk's latest announcement
Both drugs, Novo's etavopivat and Agios' mitapivat, are in the same class of oral pyruvate kinase (PK) activators that affect red blood cell metabolism in patients with sickle cell disease. However, Novo describes etavopivat as a pyruvate kinase-R (PKR) activator, reflecting that it specifically targets PKR (Protein Kinase R).
NASDAQ: AGIO
Key Data Points
Novo's top-line results from its Phase 3 trial demonstrated that etavopivat met both its primary endpoints of improving hemoglobin response and reducing vaso-occlusive crises (VOCs).
This stands in contrast with the Phase 3 results for mitapivat, which also met its primary endpoint of achieving hemoglobin response, but failed its other primary endpoint of annualized rate of sickle cell pain crises (SCPCs) compared to placebo.
Image source: Getty Images.
Where next for Agios?
Agios is currently working with the FDA to "achieve alignment" on a mitapivat trial needed for submission under an accelerated approval process. However, the news from Novo casts some doubt on the market potential and regulatory pathway for mitapivat.
That said, Agios may well have another ace up its sleeve in 2026 with another PK activator, tebapivat, which will report results in the second half of the year. Something to look out for.
