CAMBRIDGE, Mass. - Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a company focusing on cellular metabolism to develop treatments for rare diseases, has announced the completion of patient enrollment for its Phase 3 RISE UP study. The study is evaluating mitapivat, an investigational oral medication, for its effectiveness and safety in treating sickle cell disease in patients aged 16 and older.
The global, double-blind, randomized, placebo-controlled trial has enrolled over 200 patients. Agios anticipates reporting the topline results from this 52-week study in late 2025. The trial's primary endpoints include hemoglobin response and the annualized rate of sickle cell pain crises, which are significant indicators of the disease's impact on patient quality of life.
Mitapivat aims to address the heightened energy demand in red blood cells and the increased likelihood of sickling by optimizing the glycolytic pathway. This potentially could lead to increased ATP levels and decreased 2,3-DPG concentrations in red blood cells.
The company's Chief Medical Officer, Dr. Sarah Gheuens, expressed gratitude to patients, investigators, and advocates for their role in achieving full enrollment. The Phase 3 trial follows promising results from the RISE UP Phase 2 study, which were presented at the American Society of Hematology Annual Meeting in December 2023.
Agios markets PYRUKYND® (mitapivat) in the U.S. for adults with pyruvate kinase (PK) deficiency and in the European Union for adult patients with the same condition. The company cautions that the press release contains forward-looking statements regarding the potential benefits of mitapivat and its development and commercial advancement plans.
The completion of enrollment in the Phase 3 RISE UP study marks a milestone for Agios as it continues to explore the potential of mitapivat in addressing sickle cell disease. The information reported is based on a press release statement from Agios Pharmaceuticals, Inc.
In other recent news, Agios Pharmaceuticals has seen some significant developments. The company's sickle cell disease (SCD) treatment, mitapivat, has received attention due to the withdrawal of a competing drug, Oxbryta, from the market. This development has led to several analyst firms adjusting their ratings on Agios Pharma (NASDAQ:AGIO) shares. Leerink Partners maintained their Market Perform rating, while Raymond James upgraded the company to Outperform. However, Leerink Partners also highlighted potential safety concerns in the SCD treatment landscape, which could impact Agios Pharma's development program.
In other developments, Agios Pharma's candidate for the treatment of myelodysplastic syndromes, tebapivat, received an orphan drug designation from the U.S. Food and Drug Administration. The company also announced a deal with Royalty Pharma involving the sale of rights to a royalty on potential U.S. net sales of Vorasidenib, and a distribution agreement with NewBridge Pharmaceuticals for commercializing mitapivat outside the U.S. These are the recent developments in Agios Pharmaceuticals.
InvestingPro Insights
As Agios Pharmaceuticals (NASDAQ: AGIO) reaches this significant milestone in its Phase 3 RISE UP study for mitapivat, investors may be interested in the company's financial health and market performance. According to InvestingPro data, Agios has a market capitalization of $2.58 billion, reflecting substantial investor interest in its potential.
The company's revenue growth is noteworthy, with a 55.39% increase over the last twelve months as of Q2 2024. This robust growth aligns with the progress in its clinical trials and the potential for mitapivat to address multiple indications. An InvestingPro Tip highlights that net income is expected to grow this year, which could be linked to the anticipated commercialization of mitapivat for sickle cell disease if the Phase 3 trial proves successful.
Despite not being profitable over the last twelve months, Agios maintains a strong financial position. An InvestingPro Tip indicates that the company holds more cash than debt on its balance sheet, providing financial flexibility to support its ongoing research and development efforts, including the RISE UP study.
The market has responded positively to Agios' progress, with the stock showing a high return over the last year. The price has seen a large uptick over the last six months, with a total return of 42.66% during that period. This performance suggests investor optimism about the company's pipeline and the potential of mitapivat.
For readers interested in a more comprehensive analysis, InvestingPro offers additional tips and insights. There are 7 more InvestingPro Tips available for Agios Pharmaceuticals, providing a deeper understanding of the company's financial health and market position.
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