Agios Pharmaceuticals Inc. (NASDAQ:AGIO) stock is trading lower after the update on the sickle cell disease program.
The company shared top-line results from its Phase 2 study evaluating tebapivat in patients with sickle cell disease.
Despite seeing improvements in hemoglobin levels, the commercial-stage biopharmaceutical firm decided to halt further development of the oral pyruvate kinase activator for this condition after the drug failed to show sufficient clinical differentiation.
The Phase 2 clinical trial evaluated patients aged 16 years or older to characterize the dose-response relationship of tebapivat and determine how its profile compared to other pyruvate kinase activators.
The 12-week trial enrolled 59 participants who were randomized into four groups.
Participants received once-daily doses of 2.5 mg, 5.0 mg, or 7.5 mg of tebapivat, or a placebo.
Phase 2 Primary Endpoint Data
Throughout the 12-week treatment period, researchers noted improvements in hemolysis and hemoglobin levels across every tebapivat dosage, matching the established mechanism of pyruvate kinase activation.
The trial’s primary endpoint measured a hemoglobin response of at least a 1.0 g/dL increase in average hemoglobin concentration from Weeks 10 through 12 relative to baseline.
Results showed that 43.8% of patients in the 2.5 mg group (seven of 16), 47.1% in the 5.0 mg group (eight of 17), and 29.4% in the 7.5 mg group (five of 17) met the primary endpoint.
By comparison, 33.3% of participants (three of nine) in the placebo group achieved this response.
Development Discontinued Following Trial
Safety and tolerability observations for tebapivat aligned with previous trials involving sickle cell disease patients.
However, because the study did not show the required differentiation relative to other therapies, Agios chose to discontinue further development of tebapivat in sickle cell disease.
Earlier in July, the U.S. Food and Drug Administration (FDA) accepted Agios Pharmaceuticals’ supplemental New Drug Application (sNDA) for mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease with a Priority Review.
The Prescription Drug User Fee Act (PDUFA) goal date for this sNDA, submitted under the FDA’s accelerated approval pathway, is November 1.
AGIO Stock Price Activity: Agios Pharmaceuticals shares were down 7.11% at $37.21 at the time of publication on Tuesday, according to Benzinga Pro data.
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