On February 23, 2023 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 Dec. 31, 2022 (Press release, Agios Pharmaceuticals, FEB 23, 2023, View Source [SID1234627595]).

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"Over the past year, Agios has made significant progress toward our vision of transforming the lives of patients with rare diseases as we build a hematology franchise focused on diseases that share a common underlying pathophysiology, limited treatment options and profound unmet need," said Brian Goff, chief executive officer at Agios. "In 2022, we received regulatory approvals in the U.S., EU and Great Britain for PYRUKYND as the first and only disease-modifying treatment for adults with pyruvate kinase (PK) deficiency. We achieved our ambitious enrollment targets for our pivotal trials in thalassemia and sickle cell disease. We strengthened our company leadership with the appointments of new management team and Board members with deep expertise in rare diseases and global commercial strategy. We are poised for significant near- and long-term growth and look forward to a productive 2023, anticipating the readout of our Phase 2 sickle cell disease study and the completion of enrollment in our Phase 3 thalassemia studies, driving toward two additional PYRUKYND indications by 2026."

Fourth Quarter 2022 & Recent Highlights

PYRUKYND U.S. Launch: Continued to execute launch, generating $4.3 million in U.S. net revenue for the fourth quarter of 2022, the third full quarter following FDA approval. A total of 105 unique patients have completed prescription enrollment forms, representing an increase of 25 percent over the third quarter. A total of 78 patients are on PYRUKYND therapy, representing a 39 percent increase over the third quarter.
PYRUKYND Global Approvals: Received marketing authorization for adults with PK deficiency in the EU and Great Britain.
Sickle Cell Disease: Completed enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND in adults with sickle cell disease.
Thalassemia: Enrolled more than 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.
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.
Sickle Cell Disease: Announce data readout from the Phase 2 portion of the RISE UP study of PYRUKUND 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.
Fourth Quarter 2022 Financial Results

The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

Revenue: Net U.S. product revenue from sales of PYRUKYND was $4.3 million for the fourth quarter of 2022, and $11.7 million for the full year ended Dec. 31, 2022. PYRUKYND was approved by the FDA on February 17, 2022.

Cost of Sales: Cost of sales was $0.4 million for the fourth quarter of 2022 and $1.7 million for the full year ended Dec. 31, 2022.

Non-Operating Income: Non-operating income included $127.9 million as gain on sale of contingent payments from the sale of TIBSOVO royalty rights to Sagard Healthcare Partners. Non-operating income also included approximately $9.9 million from TIBSOVO royalties for the full year ended Dec. 31, 2022, with royalty income ceasing after the third quarter of 2022 due to the sale of these rights to Sagard.

Research and Development (R&D) Expenses: R&D expenses were $70.3 million for the fourth quarter of 2022 compared to $73.3 million for the fourth quarter of 2021, and $279.9 million for the year ended Dec. 31, 2022 compared to $257.0 million for the year ended Dec. 31, 2021. The year-over-year increase in R&D expense was primarily driven by increased costs for PYRUKYND and AG-946 studies and increased workforce spend across R&D.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $32.8 million for the fourth quarter of 2022 compared to $31.5 million for the fourth quarter of 2021, and $121.7 million for the year ended Dec. 31, 2022 compared to $121.4 million for the year ended Dec. 31, 2021.

Net Income (Loss) from Continuing Operations: Net income was $36.5 million for the fourth quarter of 2022 compared to a net loss of $98.6 million for the fourth quarter of 2021, and net loss was $231.8 million for the year ended Dec. 31, 2022 compared to $356.5 million for the year ended Dec. 31, 2021.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of Dec. 31, 2022, were $1.1 billion compared to $1.3 billion as of Dec. 31, 2021. This cash position includes the receipt of a one-time payment of $131.8 million associated with the sale of our rights to 5% royalties on U.S. net sales of Servier’s TIBSOVO. Agios expects that its cash, cash equivalents and marketable securities together with anticipated product revenue and interest income will enable the company to execute its operating plan, including funding the currently planned development programs for mitapivat, AG-946 and PAH stabilization and commercializing mitapivat outside of the U.S. through one or more partnerships, to cash-flow positivity without the need to raise additional equity.

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 year end 2022 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.