Agios Pharmaceuticals Inc Stock Price, News & Analysis

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.

Agios Pharmaceuticals (Nasdaq: AGIO) announced topline results from the Phase 3 ACTIVATE-KidsT study of mitapivat in children with PK deficiency who are regularly transfused. While the prespecified statistical criterion for the primary endpoint was not met, the results were clinically meaningful. 28.1% of patients in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 11.8% in the placebo arm. Secondary endpoints of transfusion-free response and normal hemoglobin response were only observed in the mitapivat arm. The safety profile was consistent with previous observations in adults. Agios has also completed enrollment for the ACTIVATE-Kids study in non-regularly transfused children, with topline data expected in 2025.

Agios Pharmaceuticals (Nasdaq: AGIO), a leader in cellular metabolism and PK activation therapies for rare diseases, has announced a conference call and live webcast scheduled for August 1, 2024, at 8:00 a.m. ET. The event will cover Agios' second quarter 2024 financial results and other business highlights. Investors and interested parties can access the live webcast under the 'Events & Presentations' section of Agios' website. A replay will be available for at least two weeks following the presentation.

Agios Pharmaceuticals presented positive results from its Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia at the European Hematology Association 2024 Hybrid Congress. The study met its primary endpoint, showing a statistically significant increase in hemoglobin response compared to placebo. Key secondary endpoints, including improvements in FACIT-Fatigue Score and hemoglobin concentration, also achieved statistical significance. The study demonstrated clinically meaningful improvements in health-related quality of life measures and patient-reported outcomes. Agios plans to file for regulatory approval of mitapivat by the end of 2024.

Agios Pharmaceuticals (Nasdaq: AGIO) will present at the Goldman Sachs 45th Annual Global Healthcare Conference on June 10, 2024, at 8:40 a.m. ET. The company, known for its leadership in cellular metabolism and PK activation therapies for rare diseases, will provide a live webcast of the presentation, accessible via the 'Events & Presentations' section of its website. The webcast replay will be available on the company's site for two weeks.

Agios announced that its Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia met its primary endpoint of transfusion reduction response. The study achieved statistical significance in all key secondary endpoints.

Mitapivat demonstrated a significant reduction in transfusion burden compared to placebo, with 30.4% of patients achieving a transfusion reduction response versus 12.6% in the placebo group. Additionally, 9.9% of patients in the mitapivat arm achieved transfusion independence compared to 1.1% in the placebo arm. The U.S. marketing application for mitapivat will be submitted by the end of 2024.

Agios Pharmaceuticals (Nasdaq: AGIO) will present at the Goldman Sachs 45th Annual Global Healthcare Conference on June 10, 2024, at 1:20 p.m. ET. The company, known for its work in cellular metabolism and PK activation for rare diseases, will have its presentation webcast live on its website. The replay of this webcast will be available for at least two weeks post-event.

Agios Pharmaceuticals (Nasdaq: AGIO) announced a $905 million purchase agreement with Royalty Pharma for the acquisition of its 15% royalty on potential U.S. net sales of vorasidenib, an oral dual inhibitor for IDH-mutant diffuse glioma. Upon FDA approval of vorasidenib, Agios will receive an upfront payment of $905 million and retain rights to a $200 million milestone payment from Servier, totaling $1.1 billion. The PDUFA action date is set for August 20, 2024. Agios will maintain a 3% royalty on annual U.S. net sales over $1 billion. This transaction is expected to boost Agios' financial flexibility and support future product launches and pipeline expansion.