Agios Pharmaceuticals Inc - AGIO STOCK NEWS

Agios Pharmaceuticals (AGIO) reported its Q4 and full-year 2024 financial results, highlighting significant progress in its PYRUKYND franchise. The company generated $10.7 million in Q4 PYRUKYND net revenue, a 20% increase from Q3 2024. For the full year 2024, PYRUKYND revenue reached $36.5 million.

Key developments include regulatory filings for mitapivat (PYRUKYND) in thalassemia across four markets, with an FDA PDUFA date of September 7, 2025. The company completed enrollment for its Phase 3 RISE UP study in sickle cell disease, with topline results expected in late 2025.

Financial highlights show a Q4 net loss of $96.5 million, while the full year 2024 recorded a net income of $673.7 million. The company maintains a strong cash position of $1.5 billion as of December 31, 2024, supported by royalty monetization and milestone payments.

Agios Pharmaceuticals (AGIO) announced positive results from its Phase 3 ACTIVATE-Kids study of mitapivat in children with Pyruvate Kinase (PK) deficiency. The study met its primary endpoint of hemoglobin response in non-regularly transfused patients aged 1 to <18 years.

The trial enrolled 30 patients, with 19 receiving mitapivat and 11 receiving placebo. 31.6% (6/19) of patients in the mitapivat arm achieved hemoglobin response compared to 0% in the placebo arm. The safety profile was consistent with previous adult studies, with no treatment discontinuations due to adverse events.

This marks Agios' first pediatric clinical program for rare hemolytic anemia, following the previously reported positive results from ACTIVATE-KidsT study in regularly transfused children. Based on these results, Agios plans to submit a marketing application for mitapivat in pediatric PK deficiency patients.

Agios Pharmaceuticals (AGIO), a company focused on cellular metabolism and pyruvate kinase activation therapies for rare diseases, has announced it will host a conference call and live webcast to discuss its fourth quarter and full year 2024 financial results. The event is scheduled for Thursday, February 13, 2025, at 8:00 a.m. ET. The webcast will be available on the company's website under the Investors section, with a replay accessible approximately two hours after the event.

Agios announced key milestones for 2025, focusing on its rare disease portfolio. The FDA accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) targeting thalassemia, with a PDUFA goal date of September 7, 2025. Topline results from the Phase 3 RISE UP study on sickle cell disease are expected in late 2025, with a potential U.S. commercial launch in 2026. The company aims to leverage its strong financial position to maximize PYRUKYND launches, advance early- and mid-stage clinical programs, and expand its pipeline.

2024 highlights include positive results from the ENERGIZE and ENERGIZE-T Phase 3 trials for thalassemia, and completed enrollment for the Phase 3 RISE UP study on sickle cell disease. Agios also reported topline results from the Phase 3 ACTIVATE-KidsT trial on pediatric pyruvate kinase deficiency and initiated a Phase 2b study on lower-risk myelodysplastic syndromes.

In corporate development, Agios secured a $905 million purchase agreement with Royalty Pharma and a $200 million milestone payment from Servier. They also entered a distribution agreement with NewBridge Pharmaceuticals for PYRUKYND in the GCC region. Anticipated 2025 milestones include receiving an FDA decision for PYRUKYND in thalassemia, announcing topline results from the RISE UP study, and filing an Investigational New Drug Application for AG-236.

Cardurion Pharmaceuticals has appointed Charlotte (Charlie) Newman as Chief Business Officer. With over 25 years of industry experience, Newman joins from Agios Pharmaceuticals (NASDAQ: AGIO) where she served as CBO leading corporate strategy and business development. Previously, she held strategic roles at Biogen (NASDAQ: BIIB).

In her new role, Newman will lead corporate development, business development, portfolio strategy, and new product planning for Cardurion's cardiovascular drug candidates. At Agios, she was instrumental in portfolio management and focusing on first and best-in-class therapeutics in non-malignant hematology. Her previous experience includes executive positions at Biogen, Provensis , and G.D. Searle and Company.

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.