We use cookies to understand how you use our site and to improve your experience.
This includes personalizing content and advertising.
By pressing "Accept All" or closing out of this banner, you consent to the use of all cookies and similar technologies and the sharing of information they collect with third parties.
You can reject marketing cookies by pressing "Deny Optional," but we still use essential, performance, and functional cookies.
In addition, whether you "Accept All," Deny Optional," click the X or otherwise continue to use the site, you accept our Privacy Policy and Terms of Service, revised from time to time.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Novo Nordisk plans a 2026 filing, setting a higher efficacy bar that could challenge AGIO's approval path.
Shares of Agios Pharmaceuticals (AGIO - Free Report) declined sharply by 23% on Monday after Novo Nordisk (NVO - Free Report) announced positive top-line data for its investigational drug, etavopivat, for the treatment of sickle cell disease (SCD).
The decline reflects growing investor concerns over Agios’ competitive position, as both companies are developing therapies for SCD that work through a similar mechanism—pyruvate kinase (PK) activation. Agios is developing its lead drug, mitapivat, as a potential treatment for SCD, while Novo Nordisk is evaluating etavopivat in the phase III HIBISCUS study for the same indication.
Mitapivat is already marketed under two brand names, Pyrukynd and Aqvesme. Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with PK deficiency. Aqvesme is approved for the treatment of anemia in adults with alpha- or beta-thalassemia. Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications.
Mitapivat’s Phase III SCD Data Weighs on AGIO
AGIO reported top-line data from the late-stage RISE UP study evaluating mitapivat for SCD in November 2025. While the drug demonstrated some benefit in improving hemoglobin levels and a reduction in the annualized rate of sickle cell pain crises, it failed to achieve statistical significance.
The results were also mixed across the key secondary endpoints. Although the patients showed statistically significant improvements in average hemoglobin concentration and indirect bilirubin levels, mitapivat fell short of improving patient-reported fatigue.
These outcomes failed to impress investors, who had high hopes that the drug would meet all study goals and raised concerns about the drug’s commercial potential in the large SCD market.
Year to date, the stock has lost 0.6% against the industry’s 3.5% growth.
Image Source: Zacks Investment Research
NVO’s Phase III HIBISCUS Data Raises Competitive Bar in SCD
The phase III data from NVO’s HIBISCUS study demonstrated that etavopivat significantly reduced the annualized rate of vaso-occlusive crises (VOCs) by 27% compared to placebo and notably delayed the onset of the first VOC, with a median time of 38.4 weeks versus 20.9 weeks. The drug also demonstrated a strong improvement in hemoglobin response, along with a favorable safety profile. The study met its primary endpoints and positioned etavopivat as a potentially best-in-class therapy.
Novo Nordisk intends to file for the regulatory approval of etavopivat in the second half of 2026, potentially setting a higher efficacy benchmark for treatments in SCD.
This creates uncertainty for Agios as it works closely with the FDA to establish an accelerated approval pathway for mitapivat in SCD. The company has already submitted its proposed confirmatory study design for regulatory review and plans to file a supplemental new drug application in the coming months, depending on the FDA feedback. If regulatory feedback is not favorable, mitapivat may struggle to compete clinically, and its approval could become more challenging.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.87. Over the same period, EPS estimates for 2027 have surged from $2.85 to $3.25. CPRX shares have gained 11.7% year to date.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.89 to $3.08. Over the same period, EPS estimates for 2027 have surged from $3.22 to $3.41. INDV shares have lost 9.7% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Zacks' 7 Best Strong Buy Stocks (New Research Report)
Valued at $99, click below to receive our just-released report
predicting the 7 stocks that will soar highest in the coming month.
View the Zacks #1 Rank List
Image: Bigstock
Agios Stock Sinks 23% as NVO's PKR Activator Meets Goal in SCD Study
Key Takeaways
Shares of Agios Pharmaceuticals (AGIO - Free Report) declined sharply by 23% on Monday after Novo Nordisk (NVO - Free Report) announced positive top-line data for its investigational drug, etavopivat, for the treatment of sickle cell disease (SCD).
The decline reflects growing investor concerns over Agios’ competitive position, as both companies are developing therapies for SCD that work through a similar mechanism—pyruvate kinase (PK) activation. Agios is developing its lead drug, mitapivat, as a potential treatment for SCD, while Novo Nordisk is evaluating etavopivat in the phase III HIBISCUS study for the same indication.
Mitapivat is already marketed under two brand names, Pyrukynd and Aqvesme. Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with PK deficiency. Aqvesme is approved for the treatment of anemia in adults with alpha- or beta-thalassemia. Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications.
Mitapivat’s Phase III SCD Data Weighs on AGIO
AGIO reported top-line data from the late-stage RISE UP study evaluating mitapivat for SCD in November 2025. While the drug demonstrated some benefit in improving hemoglobin levels and a reduction in the annualized rate of sickle cell pain crises, it failed to achieve statistical significance.
The results were also mixed across the key secondary endpoints. Although the patients showed statistically significant improvements in average hemoglobin concentration and indirect bilirubin levels, mitapivat fell short of improving patient-reported fatigue.
These outcomes failed to impress investors, who had high hopes that the drug would meet all study goals and raised concerns about the drug’s commercial potential in the large SCD market.
Year to date, the stock has lost 0.6% against the industry’s 3.5% growth.
Image Source: Zacks Investment Research
NVO’s Phase III HIBISCUS Data Raises Competitive Bar in SCD
The phase III data from NVO’s HIBISCUS study demonstrated that etavopivat significantly reduced the annualized rate of vaso-occlusive crises (VOCs) by 27% compared to placebo and notably delayed the onset of the first VOC, with a median time of 38.4 weeks versus 20.9 weeks. The drug also demonstrated a strong improvement in hemoglobin response, along with a favorable safety profile. The study met its primary endpoints and positioned etavopivat as a potentially best-in-class therapy.
Novo Nordisk intends to file for the regulatory approval of etavopivat in the second half of 2026, potentially setting a higher efficacy benchmark for treatments in SCD.
This creates uncertainty for Agios as it works closely with the FDA to establish an accelerated approval pathway for mitapivat in SCD. The company has already submitted its proposed confirmatory study design for regulatory review and plans to file a supplemental new drug application in the coming months, depending on the FDA feedback. If regulatory feedback is not favorable, mitapivat may struggle to compete clinically, and its approval could become more challenging.
Agios Pharmaceuticals, Inc. Price and Consensus
Agios Pharmaceuticals, Inc. price-consensus-chart | Agios Pharmaceuticals, Inc. Quote
AGIO’s Zacks Rank & Stocks to Consider
AGIO currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals (CPRX - Free Report) and Indivior Pharmaceuticals (INDV - Free Report) , 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 Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.87. Over the same period, EPS estimates for 2027 have surged from $2.85 to $3.25. CPRX shares have gained 11.7% year to date.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.89 to $3.08. Over the same period, EPS estimates for 2027 have surged from $3.22 to $3.41. INDV shares have lost 9.7% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.