On January 8, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported its anticipated 2024 milestones and value-driving catalysts through 2026 that support the company’s mission to transform patient outcomes in rare diseases (Press release, Agios Pharmaceuticals, JAN 8, 2024, View Source [SID1234639052]). Agios will present at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 7:30 a.m. PT, and a live webcast will be available at investor.agios.com.
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"We were pleased to announce positive topline data from the Phase 3 study of our lead PK activator, mitapivat, in non-transfusion-dependent alpha- or beta-thalassemia last week, a segment of the population with no currently approved therapeutic options in the U.S. We look forward to data readouts from four additional Phase 3 studies across our industry-leading PK activator franchise by the end of 2025," said Brian Goff, chief executive officer at Agios. "This robust series of near-term catalysts positions Agios for potential launches of a first- and best-in-class therapy in thalassemia in 2025 and in sickle cell disease in 2026, and we look forward to maximizing the commercial opportunities ahead of us. Supported by our strong cash position, Agios is poised for significant progress in the next 12-24 months, and we look forward to the opportunity to deliver a novel oral treatment option for two additional hematologic diseases with high unmet need."
2023 Highlights
Thalassemia: Completed enrollment in the Phase 3 ENERGIZE and ENERGIZE-T studies of mitapivat in non-transfusion-dependent and transfusion-dependent thalassemia, respectively
Sickle Cell Disease: Announced positive data from the Phase 2 portion of the RISE UP study of mitapivat and dosed the first patients in the Phase 3 portion
Pediatric PK Deficiency: Completed enrollment in the Phase 3 ACTIVATE kids-T study of mitapivat in children with PK deficiency who are regularly transfused. Enrolled more than half of patients in the Phase 3 ACTIVATE-kids study of mitapivat in children with pediatric PK deficiency who are not regularly transfused
Lower-risk Myelodysplastic Syndromes (LR-MDS): Announced clinical proof-of-concept in Phase 2a study of AG-946, supporting continued development in Phase 2b
Earlier-stage Pipeline: Filed an Investigational New Drug Application (IND) for PAH stabilizer for the treatment of phenylketonuria (PKU)
Business Development: Announced exclusive worldwide license agreement with Alnylam for novel siRNA targeting TMPRSS6 for the potential treatment of polycythemia vera (PV)
Data Presentations: Presented broad set of clinical and translational data at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, including positive data from the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease
Anticipated 2024 Milestones
Thalassemia: Following the announcement of topline data from the Phase 3 ENERGIZE study last week, Agios plans to report topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year) and submit a New Drug Application (NDA) for mitapivat in thalassemia (year-end)
Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat (year-end)
Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat (mid-2024). Report topline data from Phase 3 ACTIVATE kids-T study (year-end)
Lower-risk Myelodysplastic Syndromes (LR-MDS): Dose first patient in Phase 2b study of AG-946 (mid-year)
Earlier-stage Pipeline: Dose the first patient in the Phase 1 study of PAH stabilizer for the treatment of PKU (H1 2024)
Four Additional Phase 3 Readouts and Two Potential New Indication Approvals Expected by End of 2026
2024
Data readout from Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia (announced January 3, 2024)
Data readout from Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year)
Data readout from Phase 3 ACTIVATE kids-T study of mitapivat in pediatric PK deficiency (year-end)
2025
Data readout from Phase 3 portion of the RISE UP study of mitapivat in sickle cell disease
Data readout from Phase 3 ACTIVATE kids study of mitapivat in pediatric PK deficiency
Potential FDA approval for mitapivat in thalassemia
2026
Potential FDA approval for mitapivat in sickle cell disease
Potential FDA approval for mitapivat in pediatric PK deficiency
Presentation at 42nd Annual J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation from the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10 at 7:30 a.m. PT. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.