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Agios (AGIO) Up 5% on Upbeat Data From Thalassemia Study
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Shares of Agios Pharmaceuticals (AGIO - Free Report) were up 5.2% on Jan 3, after management reported that the phase III ENERGIZE study, evaluating orally-administered mitapivat in certain thalassemia patients, achieved its primary and key secondary endpoints.
A total of 194 adult participants with non-transfusion-dependent (NTD) alpha- or beta-thalassemia were enrolled in the ENERGIZE study. These patients were randomized into two treatment arms — 130 patients who received a 100mg dose of mitapivat and the remaining 64 patients who were administered placebo — over a 24-week treatment period.
Data from the ENERGIZE study showed that patients treated with mitapivat demonstrated a statistically significant increase in hemoglobin response, the primary endpoint of the study. Per management, 42.3% of patients in the mitapivat arm achieved a hemoglobin response in contrast to 1.6% in the placebo arm.
The study also demonstrated statistically significant improvements on both key secondary endpoints of average FACIT-Fatigue score and hemoglobin concentration. Improvement in the FACIT-Fatigue score suggests that mitapivat patients experienced lower levels of fatigue than the placebo recipients.
Per Agios, the above results exhibit mitapivat’s potential to become the first oral therapy for NTD thalassemia patients. Management estimates that the overall thalassemia patient population in the U.S. alone stands at around 8,000, more than half of which is represented by NTD patients. Currently, there is no approved therapy in the country to treat NTD patients.
We remind investors that mitapivat was approved by the FDA in November 2022 to treat adults with pyruvate kinase (PK) deficiency, a rare and debilitating blood disorder. The drug is being marketed under the trade name Pyrukynd.
In the past year, shares of Agios have lost 16.8% against the industry’s 1.7% rise.
Image Source: Zacks Investment Research
Alongside the ENERGIZE study, Agios is also evaluating mitapivat in the phase III ENERGIZE-T study in adults with transfusion-dependent alpha- or beta-thalassemia. Management expects to report topline data from this 48-week study in mid-2024.
Through mitapivat, Agios intends to seek regulatory approval for the drug as a potential treatment for all thalassemia sub-types, i.e., both alpha and beta thalassemia, regardless of whether patients are transfusion-dependent or not.
Following the data readout from the ENERGIZE-T study, management intends to start regulatory submissions with regulatory authorities for mitapivat in thalassemia indication supported by data from both the ENERGIZE and ENERGIZE-T studies.
Management expects a potential approval in the United States next year.
Currently, the only FDA-approved treatments for thalassemia include bluebird bio’s (BLUE - Free Report) gene therapy Zyntelgo and Bristol Myers’ (BMY - Free Report) prescription medication Reblozyl (luspatercept). However, the bluebird and Bristol Myers treatments are only approved to treat transfusion dependent beta-thalassemia patients.
If approved, Agios’ mitapivat is likely to carry an edge over both the above-approved medications. Unlike bluebird bio’s therapy, which requires a gene therapy infusion, or Bristol Myers’ Reblozyl, which is administered via an injection, mitapivat is an orally administered drug, which is likely to attract a patient’s attention.
Agios is also evaluating conducting the phase II/III RISE UP study evaluating mitapivat for sickle cell disease (SCD). In June 2023, 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.
In the past 60 days, Aquestive Therapeutics’ loss estimates for 2023 have improved from 25 cents per share to 7 cents. During the same period, loss estimates per share for 2024 have narrowed from 56 cents to 34 cents. Shares of Aquestive have surged 134.8% in the past year.
Aquestive Therapeutics’ earnings beat estimates in three of the last four quarters while missing the estimates on one occasion. On average, the company witnessed an average surprise of 70.58%. In the last reported quarter, Aquestive’s earnings beat estimates by 72.73%.
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Agios (AGIO) Up 5% on Upbeat Data From Thalassemia Study
Shares of Agios Pharmaceuticals (AGIO - Free Report) were up 5.2% on Jan 3, after management reported that the phase III ENERGIZE study, evaluating orally-administered mitapivat in certain thalassemia patients, achieved its primary and key secondary endpoints.
A total of 194 adult participants with non-transfusion-dependent (NTD) alpha- or beta-thalassemia were enrolled in the ENERGIZE study. These patients were randomized into two treatment arms — 130 patients who received a 100mg dose of mitapivat and the remaining 64 patients who were administered placebo — over a 24-week treatment period.
Data from the ENERGIZE study showed that patients treated with mitapivat demonstrated a statistically significant increase in hemoglobin response, the primary endpoint of the study. Per management, 42.3% of patients in the mitapivat arm achieved a hemoglobin response in contrast to 1.6% in the placebo arm.
The study also demonstrated statistically significant improvements on both key secondary endpoints of average FACIT-Fatigue score and hemoglobin concentration. Improvement in the FACIT-Fatigue score suggests that mitapivat patients experienced lower levels of fatigue than the placebo recipients.
Per Agios, the above results exhibit mitapivat’s potential to become the first oral therapy for NTD thalassemia patients. Management estimates that the overall thalassemia patient population in the U.S. alone stands at around 8,000, more than half of which is represented by NTD patients. Currently, there is no approved therapy in the country to treat NTD patients.
We remind investors that mitapivat was approved by the FDA in November 2022 to treat adults with pyruvate kinase (PK) deficiency, a rare and debilitating blood disorder. The drug is being marketed under the trade name Pyrukynd.
In the past year, shares of Agios have lost 16.8% against the industry’s 1.7% rise.
Image Source: Zacks Investment Research
Alongside the ENERGIZE study, Agios is also evaluating mitapivat in the phase III ENERGIZE-T study in adults with transfusion-dependent alpha- or beta-thalassemia. Management expects to report topline data from this 48-week study in mid-2024.
Through mitapivat, Agios intends to seek regulatory approval for the drug as a potential treatment for all thalassemia sub-types, i.e., both alpha and beta thalassemia, regardless of whether patients are transfusion-dependent or not.
Following the data readout from the ENERGIZE-T study, management intends to start regulatory submissions with regulatory authorities for mitapivat in thalassemia indication supported by data from both the ENERGIZE and ENERGIZE-T studies.
Management expects a potential approval in the United States next year.
Currently, the only FDA-approved treatments for thalassemia include bluebird bio’s (BLUE - Free Report) gene therapy Zyntelgo and Bristol Myers’ (BMY - Free Report) prescription medication Reblozyl (luspatercept). However, the bluebird and Bristol Myers treatments are only approved to treat transfusion dependent beta-thalassemia patients.
If approved, Agios’ mitapivat is likely to carry an edge over both the above-approved medications. Unlike bluebird bio’s therapy, which requires a gene therapy infusion, or Bristol Myers’ Reblozyl, which is administered via an injection, mitapivat is an orally administered drug, which is likely to attract a patient’s attention.
Agios is also evaluating conducting the phase II/III RISE UP study evaluating mitapivat for sickle cell disease (SCD). In June 2023, 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.
Agios Pharmaceuticals, Inc. Price
Agios Pharmaceuticals, Inc. price | Agios Pharmaceuticals, Inc. Quote
Zacks Rank & A Key Pick
Agios currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the overall healthcare sector is Aquestive Therapeutics (AQST - Free Report) , which sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, Aquestive Therapeutics’ loss estimates for 2023 have improved from 25 cents per share to 7 cents. During the same period, loss estimates per share for 2024 have narrowed from 56 cents to 34 cents. Shares of Aquestive have surged 134.8% in the past year.
Aquestive Therapeutics’ earnings beat estimates in three of the last four quarters while missing the estimates on one occasion. On average, the company witnessed an average surprise of 70.58%. In the last reported quarter, Aquestive’s earnings beat estimates by 72.73%.