Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company’s deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera.
For more information, please visit the company’s website at www.agios.com.
Featured Clinical Trial
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
This study is currently enrolling in the Phase 3 portion. For 52 weeks (1 year), participants will randomly receive one of the following two treatment options twice daily:
- 100mg of mitapivat, or
- a placebo (100mg) tablet
The goal of this Phase 3 study is to look at the percentage of how many participants’ hemoglobin increased by 1g/dL or more within 52 weeks. This study will also look at mitapivat’s effect on pain crises. If you choose, you can continue taking mitapivat for up to four years, as part of a study extension.
www.riseupstudy.com | www.clinicaltrials.gov/ct2/show/NCT05031780
Point of Contact:
Agios Medical Information | medinfo@agios.com | 833-228-8474
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The SCD C.A.R.E.S. Consortium aims to change the perception of clinical trials and increase the participation of sickle cell patients in clinical trials to ultimately provide more therapies to the SCD community. Together, the consortium works together to educate and encourage more trial participation while providing opportunities and better options for the treatment of sickle cell disease.
