We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
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.
Agios (AGIO) Up 21% on Upbeat Data From 2nd Thalassemia Study
Read MoreHide Full Article
Shares of Agios Pharmaceuticals (AGIO - Free Report) rose more than 20% on Monday after management reported that the phase III ENERGIZE-T study, evaluating its sole marketed drug, Pyrukynd (mitapivat) in certain thalassemia patients, achieved its primary and key secondary endpoints.
A total of 258 adult participants with transfusion-dependent (TD) alpha- or beta-thalassemia were enrolled in the ENERGIZE-T study. These patients were randomized into two treatment arms — 171 patients who received a 100mg oral dose of Pyrukynd and the remaining 87 patients who were administered placebo — over a 48-week treatment period.
Data from the study showed that patients treated with Pyrukynd demonstrated a statistically significant reduction in transfusion burden, the primary endpoint of the study. Per management, 30.4% of patients in the Pyrukynd arm achieved a transfusion reduction response, in contrast to 12.6% in the placebo arm.
The study also demonstrated statistically significant reductions on three key secondary endpoints, which evaluated additional measures of reduction of transfusion burden compared with a placebo. Around 10% of patients treated with Pyrukynd also achieved the secondary endpoint of transfusion independence (defined as being transfusion-free for at least eight consecutive weeks) compared with 1.1% in the placebo arm.
The results were cheered by investors, who believed that it bolsters the company’s plans to seek label expansion for Pyrukynd across all thalassemia sub-types. In January, Agios reported positive results from the phase III ENERGIZE study results, which evaluated Pyrukynd in non-transfusion-dependent (NTD) thalassemia patients. This study achieved its primary endpoint of hemoglobin response along with lower levels of fatigue.
There is no approved therapy to treat NTD thalassemia patients. Agios estimates that among the overall thalassemia patient population in the United States, more than half of it is represented by NTD patients. A potential approval in this indication will allow Agios to cater to a market with highly unmet needs.
Per Agios, results from the ENERGIZE and ENERGIZE-T studies strengthen Pyrukynd’s potential to become the first oral therapy for TD and NTD thalassemia patients. Based on data from these two studies, management plans to seek label expansion for Pyrukynd across all thalassemia sub-types before 2024-end. If approved, a commercial launch in this indication is expected next year.
We remind investors that the FDA approved Pyrukynd in November 2022 to treat adults with pyruvate kinase (PK) deficiency, a rare and debilitating blood disorder. The drug is currently the sole marketed product in the company’s portfolio.
Year to date, Agios’ shares have surged 96.8% compared with the industry’s 2.9% rise.
Image Source: Zacks Investment Research
Agios is also evaluating conducting the phase II/III RISE UP study evaluating mitapivat for sickle cell disease (SCD). Last year, management reported that the phase II portion of the RISE UP study achieved its primary endpoint of hemoglobin response, along with reductions in SCD pain. Based on this data, the company initiated the phase III portion of the study. The company intends to secure approval in SCD by 2026.
Last week, management entered into a deal with Royalty Pharma (RPRX - Free Report) to sell the royalty rights on potential net sales of vorasidenib, a brain cancer therapy. Per the deal, Agios will receive an upfront payment of $905 million if the FDA approves the drug. In exchange for this payment, RPRX will receive the entire 15% royalty on U.S. sales up to $1 billion. Post this threshold, Royalty Pharma will get a 12% royalty on sales.
Vorasidenib was a part of Agios’ oncology business that was sold to France-based pharmaceutical company Servier in 2021. Per the sale terms, the company is entitled to receive a 15% royalty on potential net sales of the drug in the United States following a possible FDA approval. The Servier drug is under the FDA’s review for a brain tumor called IDH-mutant diffuse glioma. If approved, it will be the first targeted therapy for this indication. A final decision is expected by Aug 20.
However, the company is not selling all of its rights. It will still be eligible to receive a royalty of 3% on U.S. sales of the drug exceeding $1 billion. Agios also retains the right to receive the milestone payment of $200 million from Servier upon the drug’s FDA approval.
The funds from the Royalty Pharma deal are expected to help Agios to expand its pipeline. The increased cash balance should also provide the company with financial independence when it launches Pyrukynd across thalassemia and SCD indications.
In the past 60 days, estimates for Arcutis Biotherapeutics’ 2024 loss per share have narrowed from $2.49 to $1.60. During the same period, the loss estimates per share for 2025 have improved from $1.77 to $1.14. Year to date, shares of Arcutis have surged 152.9%.
Earnings of Arcutis Biotherapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. Arcutis delivered a four-quarter average earnings surprise of 14.93%.
In the past 60 days, estimates for Heron Therapeutics’ 2024 losshave improved from 22 cents to 10 cents. During the same period, estimates for 2025 have improved from a loss of 9 cents to earnings of 1 cent. Year to date, HRTX’s shares have appreciated 127.1%.
Earnings of Heron Therapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. HRTX delivered a four-quarter average earnings surprise of 30.33%.
Unique Zacks Analysis of Your Chosen Ticker
Pick one free report - opportunity may be withdrawn at any time
Image: Bigstock
Agios (AGIO) Up 21% on Upbeat Data From 2nd Thalassemia Study
Shares of Agios Pharmaceuticals (AGIO - Free Report) rose more than 20% on Monday after management reported that the phase III ENERGIZE-T study, evaluating its sole marketed drug, Pyrukynd (mitapivat) in certain thalassemia patients, achieved its primary and key secondary endpoints.
A total of 258 adult participants with transfusion-dependent (TD) alpha- or beta-thalassemia were enrolled in the ENERGIZE-T study. These patients were randomized into two treatment arms — 171 patients who received a 100mg oral dose of Pyrukynd and the remaining 87 patients who were administered placebo — over a 48-week treatment period.
Data from the study showed that patients treated with Pyrukynd demonstrated a statistically significant reduction in transfusion burden, the primary endpoint of the study. Per management, 30.4% of patients in the Pyrukynd arm achieved a transfusion reduction response, in contrast to 12.6% in the placebo arm.
The study also demonstrated statistically significant reductions on three key secondary endpoints, which evaluated additional measures of reduction of transfusion burden compared with a placebo. Around 10% of patients treated with Pyrukynd also achieved the secondary endpoint of transfusion independence (defined as being transfusion-free for at least eight consecutive weeks) compared with 1.1% in the placebo arm.
The results were cheered by investors, who believed that it bolsters the company’s plans to seek label expansion for Pyrukynd across all thalassemia sub-types. In January, Agios reported positive results from the phase III ENERGIZE study results, which evaluated Pyrukynd in non-transfusion-dependent (NTD) thalassemia patients. This study achieved its primary endpoint of hemoglobin response along with lower levels of fatigue.
There is no approved therapy to treat NTD thalassemia patients. Agios estimates that among the overall thalassemia patient population in the United States, more than half of it is represented by NTD patients. A potential approval in this indication will allow Agios to cater to a market with highly unmet needs.
Per Agios, results from the ENERGIZE and ENERGIZE-T studies strengthen Pyrukynd’s potential to become the first oral therapy for TD and NTD thalassemia patients. Based on data from these two studies, management plans to seek label expansion for Pyrukynd across all thalassemia sub-types before 2024-end. If approved, a commercial launch in this indication is expected next year.
We remind investors that the FDA approved Pyrukynd in November 2022 to treat adults with pyruvate kinase (PK) deficiency, a rare and debilitating blood disorder. The drug is currently the sole marketed product in the company’s portfolio.
Year to date, Agios’ shares have surged 96.8% compared with the industry’s 2.9% rise.
Image Source: Zacks Investment Research
Agios is also evaluating conducting the phase II/III RISE UP study evaluating mitapivat for sickle cell disease (SCD). Last year, management reported that the phase II portion of the RISE UP study achieved its primary endpoint of hemoglobin response, along with reductions in SCD pain. Based on this data, the company initiated the phase III portion of the study. The company intends to secure approval in SCD by 2026.
Last week, management entered into a deal with Royalty Pharma (RPRX - Free Report) to sell the royalty rights on potential net sales of vorasidenib, a brain cancer therapy. Per the deal, Agios will receive an upfront payment of $905 million if the FDA approves the drug. In exchange for this payment, RPRX will receive the entire 15% royalty on U.S. sales up to $1 billion. Post this threshold, Royalty Pharma will get a 12% royalty on sales.
Vorasidenib was a part of Agios’ oncology business that was sold to France-based pharmaceutical company Servier in 2021. Per the sale terms, the company is entitled to receive a 15% royalty on potential net sales of the drug in the United States following a possible FDA approval. The Servier drug is under the FDA’s review for a brain tumor called IDH-mutant diffuse glioma. If approved, it will be the first targeted therapy for this indication. A final decision is expected by Aug 20.
However, the company is not selling all of its rights. It will still be eligible to receive a royalty of 3% on U.S. sales of the drug exceeding $1 billion. Agios also retains the right to receive the milestone payment of $200 million from Servier upon the drug’s FDA approval.
The funds from the Royalty Pharma deal are expected to help Agios to expand its pipeline. The increased cash balance should also provide the company with financial independence when it launches Pyrukynd across thalassemia and SCD indications.
Agios Pharmaceuticals, Inc. Price
Agios Pharmaceuticals, Inc. price | Agios Pharmaceuticals, Inc. Quote
Zacks Rank & Other Key Picks
Agios currently carries a Zacks Rank #2 (Buy). Some other top-ranked stocks in the overall healthcare sector include Arcutis Biotherapeutics (ARQT - Free Report) and Heron Therapeutics (HRTX - Free Report) , each carrying a Zacks Rank #2 at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Arcutis Biotherapeutics’ 2024 loss per share have narrowed from $2.49 to $1.60. During the same period, the loss estimates per share for 2025 have improved from $1.77 to $1.14. Year to date, shares of Arcutis have surged 152.9%.
Earnings of Arcutis Biotherapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. Arcutis delivered a four-quarter average earnings surprise of 14.93%.
In the past 60 days, estimates for Heron Therapeutics’ 2024 losshave improved from 22 cents to 10 cents. During the same period, estimates for 2025 have improved from a loss of 9 cents to earnings of 1 cent. Year to date, HRTX’s shares have appreciated 127.1%.
Earnings of Heron Therapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. HRTX delivered a four-quarter average earnings surprise of 30.33%.