AGIO Shares Sink Nearly 7% After Scrapping the Tebapivat SCD Program

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AGIO’s Phase II Data Validates PK Activation, Lacks Differentiation

Tebapivat is a next-generation oral PK activator that uniquely targets both PKR and PKM2 isoforms, aiming to improve outcomes in rare blood disorders. In the phase II study, Agios evaluated three once-daily dose levels (2.5 mg, 5.0 mg and 7.5 mg) in patients aged 16 years and older with SCD to assess whether the therapy could offer a clinically meaningful advantage over existing PK activators.

The 12-week study data demonstrated that tebapivat improved hemoglobin levels and reduced markers of hemolysis across all dose groups, confirming its expected mechanism of PK activation. The drug also exhibited a favorable safety and tolerability profile. However, management concluded that the efficacy data did not show sufficient clinical differentiation from existing PK activators to justify further development, resulting in the discontinuation of the SCD program.

Tebapivat Development Terminated Across All Indications

The SCD decision follows Agios' earlier move in May to discontinue development of tebapivat for lower-risk myelodysplastic syndromes (LR-MDS) after a mid-stage dose-finding study failed to demonstrate compelling clinical benefit. With the termination of the SCD program, Agios has discontinued the development of tebapivat across all indications.

Agios will instead focus on mitapivat, its lead oral PK activator, which is currently under regulatory review in the United States for the SCD indication.

Agios’ Focus Shifts to Mitapivat for SCD Program

Mitapivat is marketed in the United States as Pyrukynd for adults with pyruvate kinase deficiency and as Aqvesme, for the treatment of anemia in adults with alpha- or beta-thalassemia. Aqvesme was approved by the FDA in December 2025. Outside the United States, the therapy is marketed as Pyrukynd for PK deficiency and thalassemia. The European Union expanded the drug's label to include thalassemia in May 2026, adding to its existing PK deficiency approval.

Earlier this month, the FDA accepted its supplemental new drug application (sNDA) seeking approval for mitapivat in SCD.The sNDA for mitapivat in SCD was submitted under the FDA’s accelerated approval pathway.

With the FDA granting a priority review to the sNDA, a decision from the regulatory body is expected on Nov. 1, 2026. If approved, mitapivat is likely to become the first approved oral PK activator for patients with SCD.

AGIO Zacks Rank & Stocks to Consider

Agios currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Neurocrine Biosciences NBIX, Amarin Corporation AMRN and Liquidia Corporation LQDA, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Neurocrine Biosciences’ 2026 earnings per share have risen from $9.15 to $9.44. Over the same period, EPS estimates for 2027 have increased from $10.23 to $10.79. NBIX shares have gained 26.2% year to date.

Neurocrine Biosciences’ earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 9.08%.

Over the past 60 days, estimates for Amarin Corporation’s 2026 bottom line have declined from earnings per share of 92 cents to a loss of 65 cents per share. Over the same period, estimates for earnings per share decreased from $1.61 to a loss per share of 51 cents for 2027. AMRN shares have lost 0.1% year to date.

Amarin Corporation’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 50.02%.

Over the past 60 days, estimates for Liquidia’s 2026 earnings per share have increased to $3.02 from $2.97. Over the same period, EPS estimates for 2027 have risen to $4.92 from $4.81. LQDA shares have gained 134.6% year to date.

Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.

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This article originally published on Zacks Investment Research (zacks.com).

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