Agios Pharmaceuticals Inc Stock Price, News & Analysis

Agios Pharmaceuticals (AGIO) announced the FDA's acceptance of their supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) for treating adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The FDA assigned a Standard review classification with a PDUFA goal date of September 7, 2025.

The sNDA submission is supported by results from two Phase 3 trials: ENERGIZE and ENERGIZE-T, which evaluated mitapivat versus placebo in adults with non-transfusion-dependent and transfusion-dependent thalassemia. The ENERGIZE trial results were presented at the European Hematology Association 2024 Congress, while the ENERGIZE-T results were presented at the American Society of Hematology Annual Meeting in December 2024.

Agios Pharmaceuticals (Nasdaq: AGIO), a company specializing in cellular metabolism and PK activation therapies for rare diseases, has announced its upcoming participation at the 43rd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled for Wednesday, January 15, 2025, at 7:30 a.m. PT / 10:30 a.m. ET.

The company will provide a live webcast of the presentation, which will be available through the Investors section of Agios's website under the Events & Presentations tab. Interested parties can access the replay of the webcast on the company's website, where it will remain available for a minimum of two weeks following the presentation.

Agios Pharmaceuticals (AGIO) has received orphan medicinal product designation from the European Commission for mitapivat, their oral PK activator, for treating sickle cell disease. This follows the FDA's orphan drug designation granted in November 2020. The European designation provides benefits including reduced fees and 10-year market exclusivity for conditions affecting fewer than 5 in 10,000 EU individuals.

The company's Phase 3 RISE UP study evaluating mitapivat's efficacy and safety in sickle cell disease is fully enrolled, with results expected in late 2025. This designation highlights the urgent need for new sickle cell disease treatments and mitapivat's potential to provide meaningful benefits to patients.

Agios Pharmaceuticals (AGIO) announced positive results from its Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia. The study achieved its primary endpoint with 30.4% of patients on mitapivat showing significant reduction in transfusion burden compared to 12.6% on placebo.

The trial also met all key secondary endpoints, demonstrating sustained transfusion reduction responses. Notably, 9.9% of mitapivat patients achieved transfusion independence versus 1.1% in the placebo group. Safety profile showed similar adverse event rates between mitapivat (90.1%) and placebo (83.5%).

Based on these results, Agios has filed regulatory applications for mitapivat (PYRUKYND®) in the U.S., European Union, Saudi Arabia, and UAE for treating both transfusion-dependent and non-transfusion-dependent thalassemia patients.

Agios Pharmaceuticals will present new data on their PK activators mitapivat and tebapivat at the 66th ASH Annual Meeting in San Diego from December 7-10, 2024. Key highlights include results from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia, Phase 1 data on tebapivat in sickle cell disease, and a Phase 2b trial update in myelodysplastic syndromes.

The presentations will feature 16 abstracts led by Agios and external collaborators, including oral presentations on thalassemia research and poster presentations on sickle cell disease treatments. The company will host a live investor event with leadership and medical experts on December 9.

Agios Pharmaceuticals reported Q3 2024 financial results and business highlights. The company achieved $9.0 million in PYRUKYND® net revenue, a 4% increase from Q2. Significant developments include completing enrollment for Phase 3 RISE UP study in sickle cell disease and receiving $1.1 billion in payments following FDA approval of vorasidenib. The company reported net income of $947.9 million and ended the quarter with $1.7 billion in cash and equivalents. R&D expenses decreased to $72.5 million, while SG&A expenses increased to $38.5 million.

Agios Pharmaceuticals (AGIO) has completed enrollment for its Phase 3 RISE UP study evaluating mitapivat in sickle cell disease patients aged 16 and older. The global trial enrolled over 200 patients, with topline results expected in late 2025. The study's primary endpoints focus on hemoglobin response and annualized rate of sickle cell pain crises. Mitapivat, an oral PK activator, is designed to optimize the glycolytic pathway by increasing ATP levels and decreasing 2,3-DPG concentrations in red blood cells, potentially addressing the underlying mechanisms of sickle cell disease.

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, has announced a conference call and live webcast scheduled for Thursday, October 31, 2024, at 8:00 a.m. ET. The purpose of this event is to report the company's third quarter 2024 financial results and business highlights.

Interested parties can access the live webcast of the presentation under the "Events & Presentations" section in the Investors area of Agios' official website at www.agios.com. For those unable to attend the live event, a replay of the webcast will be available on the company's website for a minimum of two weeks following the presentation.

Agios Pharmaceuticals (Nasdaq: AGIO) has received FDA orphan drug designation for its novel pyruvate kinase activator tebapivat (AG-946) in the treatment of myelodysplastic syndromes (MDS). This designation supports the development of medicines for rare disorders affecting fewer than 200,000 people in the U.S. and offers incentives such as tax credits and potential market exclusivity.

Tebapivat aims to be the first oral therapy addressing anemia due to ineffective erythropoiesis in lower-risk MDS, which affects 75,000-80,000 patients in the U.S. and EU5. Agios has completed a Phase 2a study and is initiating a Phase 2b study of tebapivat in lower-risk MDS. The company's lead PK activator, mitapivat, has previously received FDA orphan drug designation for other rare blood disorders.