Agios Q1, upcoming milestones, call

 On Track for PYRUKYND® (mitapivat) Data Readouts of the Phase 2 Portion of the RISE UP Study in Sickle Cell Disease in Mid-2023 and the Phase 3 ENERGIZE and ENERGIZE-T Studies in Thalassemia in 2024

– U.S. PYRUKYND® Net Revenue of $5.6 Million in Q1; $1.0 Billion of Cash, Cash Equivalents and Marketable Securities as of March 31, 2023

Agios expects to execute on the following additional key milestones and priorities by the end of 2023:

• Thalassemia: Complete enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassemia, respectively, by mid-year.

• Sickle Cell Disease: Announce data readout from the Phase 2 portion of the RISE UP study of PYRUKYND® and go/no-go to Phase 3 decision by mid-year.

• Pediatric PK Deficiency: Enroll more than half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND® by year-end.

• Lower-risk Myelodysplastic Syndromes (LR-MDS): Complete enrollment of the Phase 2a study of novel PK activator AG-946 by year-end.

• Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU) by year-end.

Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss first quarter 2023 financial results and recent business activities. The live webcast can be accessed under “Events & Presentations” in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company's website beginning approximately two hours after the event.

https://finance.yahoo.com/news/agios-reports-business-highlights-first-110000675.html