On February 15, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, reported business highlights and financial results for the fourth quarter and year ended December 31, 2023 (Press release, Agios Pharmaceuticals, FEB 15, 2024, View Source [SID1234640134]).

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"The past 12 months have been remarkable for Agios. We reported positive data across our industry-leading pipeline of PK activators, including Phase 3 data in non-transfusion-dependent thalassemia, Phase 2 data in sickle cell disease and clinical proof-of-concept in lower-risk MDS, and expanded our preclinical pipeline by in-licensing a novel siRNA program from Alnylam," said Brian Goff, chief executive officer at Agios. "In 2024, we expect two additional Phase 3 readouts, including the Phase 3 study of mitapivat in transfusion-dependent thalassemia, and are actively preparing for a potential U.S. launch in thalassemia in 2025. Together with our strong cash position, Agios is poised for significant near- and long-term growth as we progress toward of our vision of becoming a leading rare disease company."

Fourth Quarter 2023 & Recent Highlights

PYRUKYND U.S. Launch: Generated $7.1 million in U.S. net revenue for the fourth quarter of 2023, a 4 percent decrease from the third quarter of 2023, primarily driven by lower customer inventory levels at the end of the fourth quarter of 2023, partially offset by favorable gross-to-net adjustments. A total of 178 unique patients have completed prescription enrollment forms, representing an increase of 11 percent over the third quarter of 2023. A total of 109 patients are on PYRUKYND therapy, a 9 percent increase from the third quarter of 2023.
Thalassemia: Announced positive topline data from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia. The study achieved its primary endpoint of hemoglobin response and achieved both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration.
Sickle Cell Disease: Presented positive results from the Phase 2 portion of the RISE UP pivotal study of mitapivat at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition. The study achieved its primary endpoint of hemoglobin response and an improvement in annualized rates of sickle cell pain crises was observed.
Lower-risk Myelodysplastic Syndromes: Announced clinical proof-of-concept data in the open-label Phase 2a study of AG-946 for the treatment of anemia in lower-risk myelodysplastic syndromes (LR-MDS).
Earlier-stage Pipeline: Filed an Investigational New Drug Application (IND) for AG-181, Agios’ PAH stabilizer for the treatment of phenylketonuria (PKU).
Data presentations: Presented broad set of clinical and translational data at the 65th 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, as noted above.
Anticipated 2024 Milestones

Thalassemia: Following the announcement of positive topline data from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia in January 2024, Agios plans to report topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year) and file for FDA approval of 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-year). Report topline data from Phase 3 ACTIVATE kids-T study (year-end)
Lower-risk Myelodysplastic Syndromes: 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 AG-181 for the treatment of PKU (early 2024)
Fourth Quarter and Full Year 2023 Financial Results

Revenue: Net U.S. product revenue from sales of PYRUKYND was $7.1 million for the fourth quarter of 2023 compared to $4.3 million for the fourth quarter of 2022, and $26.8 million for the year ended Dec. 31, 2023 compared to $11.7 million for the year ended Dec. 31, 2022.

Cost of Sales: Cost of sales was $0.6 million for the fourth quarter of 2023 and $2.9 million for the full year ended Dec. 31, 2023.

Research and Development (R&D) Expenses: R&D expenses were $77.5 million for the fourth quarter of 2023 compared to $70.3 million for the fourth quarter of 2022, and $295.5 million for the year ended Dec. 31, 2023 compared to $279.9 million for the year ended Dec. 31, 2022. These changes reflect an increase in development costs for mitapivat and the up-front payment associated with the license agreement with Alnylam, partially offset by a reduction in expenses associated with the evolution of our research organization and the sale of our oncology business to Servier.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $35.3 million for the fourth quarter of 2023 compared to $32.8 million for the fourth quarter of 2022, and $119.9 million for the year ended Dec. 31, 2023 compared to $121.7 million for the year ended Dec. 31, 2022.

Net Income (Loss): Net loss was $95.9 million for the fourth quarter of 2023 compared to a net income of $36.5 million for the fourth quarter of 2022, and net loss was $352.1 million for the year ended Dec. 31, 2023 compared to $231.8 million for the year ended Dec. 31, 2022. The increase in net loss is due to the $127.9 million sale to Sagard in the fourth quarter of 2022 of our rights to future contingent payments associated with royalties on U.S. net sales of TIBSOVO.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of Dec. 31, 2023, were $806.4 million compared to $1.1 billion as of Dec. 31, 2022. Agios expects that its cash, cash equivalents and marketable securities together with anticipated product revenue, interest income and vorasidenib milestone will enable the company to fund its operating expenses and capital expenditures at least into 2026. This does not include cash inflows which could extend runway beyond 2026 including potential royalties or monetization of royalties from vorasidenib, commercializing mitapivat outside of the U.S. through one or more partnerships, or other potential strategic business or financial agreements.

Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and full year 2023 financial results and recent business highlights. 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.