On January 8, 2023 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported its anticipated 2023 milestones and significant value-driving catalysts through 2026 that support the company’s mission to transform patient outcomes in rare diseases (Press release, Agios Pharmaceuticals, JAN 8, 2023, View Source [SID1234626022]). Agios will present at the 41st Annual J.P. Morgan Healthcare Conference on Wednesday, January 11, 2023, at 7:30 a.m. PT, and a live webcast will be available at investor.agios.com.

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"Agios is poised for significant growth with the potential for approvals in two additional PYRUKYND indications by 2026, and is well capitalized to advance its robust existing clinical pipeline and expand its portfolio within our core areas of expertise," said Brian Goff, chief executive officer at Agios. "As the pioneering leader in PK activation with more than seven years of clinical experience with PYRUKYND, we have generated an impressive body of consistent and compelling data across rare hematological diseases with shared underlying pathophysiology that further builds confidence in our five ongoing pivotal clinical trials in thalassemia, sickle cell disease and pediatric PK deficiency. Furthermore, we are executing our first rare disease U.S. product launch with PYRUKYND in adult PK deficiency, providing the first disease-modifying therapy for this patient community that previously had no treatment options and building the capabilities to set us up for success with our expected launches in meaningfully larger patient populations."

Recent Highlights

Adult PK Deficiency: Received marketing authorization for PYRUKYND in adults with PK deficiency in the EU and Great Britain

Thalassemia: Enrolled approximately half of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively

Sickle Cell Disease: Closed screening in the Phase 2 portion of the RISE UP study of PYRUKYND in adults with sickle cell disease in December and expect to complete enrollment in January

Data Presentations: Presented broad set of clinical and translational data at the 64th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, including long-term PYRUKYND data in adults with non-transfusion-dependent thalassemia and in adults with PK deficiency

Leadership: Appointed Tsveta Milanova to the role of chief commercial officer, bringing two decades of experience in rare disease commercial strategy and global market access

Anticipated 2023 Milestones

Thalassemia: Complete enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND by mid-year

Pediatric PK Deficiency: Enroll at least half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND by year-end

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

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

Earlier-stage Pipeline: File investigational new drug (IND) application for PAH stabilizer for the treatment of phenylketonuria (PKU) by year-end

Agios 2026 Vision

By 2026, Agios’ vision is to establish a classical hematology franchise with PYRUKYND approvals across PK deficiency, thalassemia and sickle cell disease; expand its portfolio by advancing AG-946 and the preclinical pipeline as well as through disciplined business development aligned with the company’s core therapeutic focus areas and capabilities; and achieve cash-flow positivity. Agios provided a roadmap of additional significant potential catalysts between 2024 and 2026 to enable the realization of this vision, as follows:

2024

Data readout from Phase 3 ENERGIZE study of PYRUKYND in adults with non-transfusion-dependent thalassemia (first half of 2024)

Data readout from Phase 3 ENERGIZE-T study of PYRUKYND in adults with transfusion-dependent thalassemia (second half of 2024)

Data readout from Phase 2a study of AG-946 in LR-MDS

2025

Potential FDA approval for PYRUKYND in thalassemia

Data readout from Phase 3 portion of RISE UP study of PYRUKYND in sickle cell disease, pending go/no-go decision in 2023

Data readouts from Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND in pediatric PK deficiency

2026

Potential FDA approval for PYRUKYND in sickle cell disease

Potential FDA approval for PYRUKYND in pediatric PK deficiency

Achieve cash-flow positivity

Presentation at 41st Annual J.P. Morgan Healthcare Conference

Agios will webcast its corporate presentation from the 41st Annual J.P. Morgan Healthcare Conference on Wednesday, January 11 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.