AGIO Stock Hits 52-Week Low on Mixed Sickle Cell Disease Study Results

Shares of Agios Pharmaceuticals AGIO tanked about 51% on Wednesday after it reported mixed top-line results from the phase III RISE UP study, which evaluated orally administered Pyrukynd (mitapivat) in patients aged 16 years or older with sickle cell disease (SCD).

While the study did meet its primary endpoint of improving patients’ hemoglobin levels, it failed to achieve the other co-primary endpoint of reducing the annualized rate of sickle cell pain crises (SCPCs).

More on AGIO’s RISE UP Study Results

In terms of efficacy, data from the study showed that nearly 41% of Pyrukynd-treated patients achieved a hemoglobin response compared with around 3% in the placebo arm. While patients treated with the drug did show numerical reductions in SCPCs — 2.62 for Pyrukynd-treated vs. 3.05 for placebo — it failed to achieve statistical significance and therefore did not meet this co-primary endpoint.

The results were also mixed across the key secondary endpoints. While Pyrukynd-treated patients showed statistically significant improvements in average hemoglobin concentration and indirect bilirubin levels, it fell short in improving patient-reported fatigue.

Despite the mixed results, Agios is preparing to move forward with a regulatory filing for the drug. It emphasizes findings from a post hoc analysis of hemoglobin responders, a subgroup of SCD patients that not only achieved the primary endpoint but also showed clearer clinical benefits, including reduced SCPCs and improved patient-reported fatigue. The company intends to submit this regulatory filing after meeting with the FDA in the first quarter of 2026.

An oral pyruvate kinase (PK) activator, Pyrukynd is currently approved in the United States and Europe for treating hemolytic anemia in adults with PK deficiency. A regulatory filing is currently under FDA review, seeking label expansion for the drug across both alpha- or beta-thalassemia patients who are non-transfusion-dependent (NTD) and transfusion-dependent (TD), respectively. A final decision is expected by Dec. 7, 2025.

AGIO Stock’s Performance

Following the mixed study results, shares of Agios hit a 52-week low of $22.24. The latest announcement failed to impress investors, who had high hopes for Pyrukynd to meet both study goals. Some investors also questioned the drug’s future in SCD — an area with significant commercial potential that the company was planning to enter next year.

Year to date, the company’s shares have lost 32% against the industry’s 17% growth.

Rival Fulcrum Shares Gains on Agios’ Setback

Post the results announcement, shares of clinical-stage biotech Fulcrum Therapeutics FULC gained over 18% in one trading session. FULC is developing an investigational drug called pociredir, which is currently being evaluated in an early-stage study for SCD.

Wall Street expressed optimism around the Fulcrum Therapeutics drug, which utilizes a different mechanism to treat SCD.

AGIO’s Zacks Rank

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

Agios Pharmaceuticals, Inc. Price

 

Agios Pharmaceuticals, Inc. price | Agios Pharmaceuticals, Inc. Quote

Our Key Picks Among Biotech Stocks

Some better-ranked stocks from the sector are Alkermes ALKS and CorMedix CRMD, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Earnings per share (EPS) estimates for Alkermes’ 2025 have increased from $1.82 to $1.96, while those for 2026 have risen from $1.59 to $1.66 in the past 60 days. ALKS stock has registered breakeven growth year to date.

Alkermes’ earnings beat estimates in three of the trailing four quarters and missed the mark on one occasion, delivering an average negative surprise of 4.58%.

In the past 60 days, estimates for CorMedix’s EPS have increased from $1.24 to $2.87 for 2025. During the same time, EPS estimates for 2026 have increased from $2.09 to $2.88. Year to date, shares of CRMD have rallied 24%.

CorMedix’s earnings beat estimates in each of the trailing four quarters, the average surprise being 27.04%.

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