On May 4, 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 first quarter ended March 31, 2023 (Press release, Agios Pharmaceuticals, MAY 4, 2023, View Source [SID1234630996]).
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"In the first quarter of the year, Agios made significant progress executing across our industry-leading pipeline of PK activators, with clinical studies spanning thalassemia, sickle cell disease, lower-risk MDS and pediatric PK deficiency," said Brian Goff, chief executive officer at Agios. "We closed screening of the Phase 3 studies of PYRUKYND in thalassemia with enrollment expected to be complete later this month, and look forward to the data readout of the Phase 2 portion of the RISE UP study of PYRUKYND in sickle cell disease in the middle of this year."
First Quarter 2023 & Recent Highlights
PYRUKYND U.S. Launch: Generated $5.6 million in U.S. net revenue for the first quarter of 2023, the fourth full quarter following FDA approval. A total of 127 unique patients have completed prescription enrollment forms, representing an increase of 21 percent over the fourth quarter of 2022. A total of 89 patients are on PYRUKYND therapy, representing a 14 percent increase over the fourth quarter of 2022.
Thalassemia: Closed screening of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively.
Leadership: Appointed Jeffrey Capello to the board of directors. Paul Clancy will step down from the board of directors at the end of his term, effective June 13, 2023.
Environmental, Social, and Governance (ESG): Published 2023 ESG Report, which provides corporate sustainability disclosures for the period January 1, 2022 to December 31, 2022.
Other: Servier’s Phase 3 trial of vorasidenib in patients with residual or recurrent IDH mutant low-grade glioma met both its primary endpoint and key secondary endpoints. As part of the divestiture of Agios’ oncology business to Servier, Agios retains rights to a potential $200 million milestone upon FDA approval of vorasidenib and 15% royalties on potential U.S. net sales.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following additional key milestones and priorities by the end of 2023:
Thalassemia: Complete enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND in not regularly transfused and regularly transfused adults with thalassemia, respectively, by mid-year.
Sickle Cell Disease: Announce data readout from the Phase 2 portion of the RISE UP study of PYRUKYND 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.
First Quarter 2023 Financial Results
Revenue: Net U.S. product revenue from sales of PYRUKYND for the first quarter of 2023 was $5.6 million. This revenue reflects the fourth full quarter of PYRUKYND launch, following FDA approval on February 17, 2022.
Cost of Sales: Cost of sales for the first quarter of 2023 was $0.6 million.
Research and Development (R&D) Expenses: R&D expenses were $67.3 million for the first quarter of 2023 compared to $70.1 million for the first quarter of 2022. The year-over-year decrease was primarily driven by the $1.5 million of reimbursable transition-related expenses provided to Servier in the first quarter of 2022 related to the sale of the oncology business.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $28.4 million for the first quarter of 2023 compared to $31.5 million for the first quarter of 2022. The year-over-year decrease was primarily attributable to a reduction in workforce-related expenses.
Net Loss: Net loss was $81.0 million for the first quarter of 2023 compared to $94.8 million for the first quarter of 2022.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2023, were $1.0 billion compared to $1.1 billion as of December 31, 2022. 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.
Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss first quarter 2023 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.