Agios Pharmaceuticals, Inc. - AGIO STOCK NEWS

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.

Agios Pharmaceuticals (Nasdaq: AGIO), a leader in cellular metabolism and PK activation therapies for rare diseases, has announced its participation in the 2024 Cantor Global Healthcare Conference. The company is scheduled to present on Wednesday, September 18, 2024, at 10:20 a.m. ET.

Investors and interested parties can access a live webcast of the presentation through the 'Events & Presentations' section of Agios' 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 at least two weeks following the presentation, ensuring widespread access to the information shared during the conference.

Agios Pharmaceuticals (Nasdaq: AGIO) is set to receive $1.1 billion in milestone payments following the FDA approval of vorasidenib for Grade 2 astrocytoma or oligodendroglioma. This includes $905 million from Royalty Pharma and $200 million from Servier, expected in Q3 2024. The payments will increase Agios' pro-forma cash position to $1.7 billion as of June 30, 2024.

This strong financial position will enable Agios to prepare for potential PYRUKYND® (mitapivat) launches in thalassemia (2025) and sickle cell disease (2026), while driving pipeline progress. The approval marks the first for Grade 2 glioma in over 20 years, highlighting Agios' expertise in discovering novel therapies for high unmet needs.

Agios Pharmaceuticals (NASDAQ: AGIO) reported its Q2 2024 financial results and business highlights. Key points include:

1. Positive topline data from Phase 3 ENERGIZE-T study for thalassemia treatment.

2. $905 million purchase agreement with Royalty Pharma for vorasidenib royalty.

3. Results from Phase 3 ACTIVATE-KidsT study of mitapivat in children with PK deficiency.

4. PYRUKYND® net revenue of $8.6 million in Q2, a 5% increase from Q1.

5. Cash position of $645.3 million as of June 30, 2024.

6. Net loss of $96.1 million for Q2 2024.

7. R&D expenses increased to $77.4 million, while SG&A expenses rose to $35.5 million.

8. Plans to file sNDA for mitapivat in thalassemia by year-end 2024.