=0.0002).Mitapivat demonstrated a statistically significant increase in hemoglobin response rate compared to placebo.Mitapivat also showed statistically significant improvements in the key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration.These improvements were observed across all pre-specified subgroups.In the safety population, the most common adverse events (‰¥10%) in the mitapivat arm were headache (13.8%), diarrhea (10.8%), nausea (10.8%), and upper respiratory tract infection (10.0%).The majority of adverse events were mild to moderate in severity and transient, with no new or unexpected safety findings identified during the study.The detailed results from the ENERGIZE study will be submitted for publication in a peer-reviewed journal and presented at upcoming scientific congresses. Agios plans to host a virtual investor event on June 16, 2024, at 10 a.m. Eastern Time or 4 p.m. Central European Summer Time to discuss the ENERGIZE data and provide an update on the company’s pipeline and business strategy. Interested parties can access the live webcast on the Agios website at www.agios.com in the Investors section. A replay of the webcast will be available on the company’s website for at least 30 days following the event. About MitapivatMitapivat is an investigational oral small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes. PKR enzyme activators are believed to work by increasing the affinity of the PKR enzyme for its substrate, phosphoenolpyruvate (PEP), resulting in increased enzymatic activity. In vitro studies have shown that mitapivat increases enzyme activity in PK-deficient red blood cells, restores red blood cell metabolism, and decreases 2,3-DPG levels. In preclinical and clinical studies, mitapivat has demonstrated an increase in hemoglobin and a decrease in hemolysis. Mitapivat is being evaluated in several ongoing clinical trials for the treatment of thalassemia and other hemolytic anemias. About AgiosAgios is focused on discovering and developing novel investigational medicines to treat patients with genetically defined diseases. The company’s core focus is in the field of cellular metabolism, where it is pioneering the development of potential therapeutics targeting altered metabolism in oncology and rare genetic diseases. For more information, please visit the company’s website at www.agios.com. Forward-Looking StatementsThis press release contains forward-looking statements based on Agios’ current plans, estimates, and expectations. These statements are forward-looking and involve risks and uncertainties. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, but not limited to, the risks related to the impact of general economic, industry or political conditions, the impact of the COVID-19 pandemic, the risks associated with Agios’ ability to implement its business strategy, manage its growth, and execute on its business plans, the risks associated with Agios’ product candidate development, regulatory approvals and changes to the regulatory environment, the risks associated with Agios’ ability to obtain and maintain intellectual property protection for its product candidates, the risks related to the possible approval of mitapivat for thalassemia, the risks related to Agios’ ability to continue to build its product pipeline and commercialize its products, the risks associated with Agios’ dependence on third parties for development, manufacturing and distribution of its products, and the risks related to the potential impact of the COVID-19 pandemic on Agios’ business and operations. These and other risks are described in greater detail under the caption “Risk Factors” included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. ContactsInvestors: Holly Manning, 617-844-6630, [email protected]: Jessica Rennekamp, 857-209-3286, [email protected]\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”\”
