Agios Pharmaceuticals (AGIO) Q4 2023 earnings summary

Executive summary

• All stated 2023 development goals were delivered; Phase 3 ENERGIZE in non-transfusion-dependent thalassemia met its primary and key secondary endpoints.

• PYRUKYND is approved for adult PK deficiency in the U.S., EU and Great Britain; development includes pediatric PK deficiency, thalassemia and sickle cell disease.

• Four additional Phase 3 mitapivat readouts are expected by end-2025, with two additional readouts expected in 2024.

Financial highlights

• PYRUKYND net U.S. sales were $7.1M in Q4 2023 vs. $4.3M in Q4 2022, and $26.8M for FY 2023 vs. $11.7M in FY 2022.

• R&D expense was $77.5M in Q4 2023 vs. $70.3M, and $295.5M for FY 2023 vs. $279.9M; the annual increase reflected mitapivat development and the Alnylam license payment, partly offset by lower other costs.

• SG&A expense was $35.3M in Q4 2023 vs. $32.8M, and $119.9M for FY 2023 vs. $121.7M.

• Net loss was $95.9M in Q4 2023 vs. net income of $36.5M; FY net loss was $352.1M vs. $231.8M, and FY net loss per share was $6.33 vs. $4.23.

• Cash, cash equivalents and marketable securities were $806.4M at Dec. 31, 2023 vs. $1.1B at Dec. 31, 2022.

Outlook and guidance

• ENERGIZE-T topline data in transfusion-dependent thalassemia expected mid-2024; FDA filing for mitapivat in thalassemia planned for year-end, with ENERGIZE and ENERGIZE-T data planned for joint submission and potential U.S. launch targeted for 2025.

• Phase 3 RISE UP enrollment expected to complete by year-end 2024; readout expected in 2025 and potential U.S. approval targeted for 2026.

• ACTIVATE-kids enrollment expected to complete mid-2024; ACTIVATE-kids-T topline data expected by year-end.

• AG-181 Phase 1 first patient dosing expected in early 2024; AG-946 Phase 2b lower-risk MDS first patient dosing expected mid-year.

• Cash, anticipated product revenue, interest income and a vorasidenib milestone expected to fund operating expenses and capital expenditures at least into 2026.