On May 2, 2024 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, reported business highlights and financial results for the first quarter ended March 31, 2024 (Press release, Agios Pharmaceuticals, MAY 2, 2024, View Source [SID1234642572]).
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"We were delighted to report positive data from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia and look forward to announcing topline data from the Phase 3 ENERGIZE-T study in transfusion-dependent thalassemia in the second quarter of this year," said Brian Goff, chief executive officer at Agios. "Mitapivat has the potential to become the first therapy approved for all thalassemia subtypes, and our commercial organization is actively preparing for a potential launch next year. Beyond thalassemia, we look forward to the RISE UP Phase 3 readout in sickle cell disease next year, with potential for approval in 2026, as we progress toward our vision of becoming a leading rare disease company with a potential multi-billion-dollar franchise in PK activation."
First Quarter 2024 and Recent Highlights
PYRUKYND Revenues: Generated $8.2 million in net revenue for the first quarter of 2024, a 15 percent sequential increase from the fourth quarter of 2023, primarily driven by increased patient demand. A total of 188 unique patients have completed prescription enrollment forms, representing an increase of 6 percent over the fourth quarter of 2023. A total of 120 patients are on PYRUKYND therapy, a 10 percent increase from the fourth quarter of 2023.
Thalassemia: Announced positive results from the Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent alpha- or beta-thalassemia.
Earlier-Stage Pipeline: Dosed the first participants in the Phase 1 study of AG-181 for the treatment of phenylketonuria (PKU).
Environment, Social, and Governance (ESG): Published 2024 ESG Report, which provides corporate sustainability disclosures for the period Jan. 1, 2023 to Dec. 31, 2023.
Other: Servier announced FDA filing acceptance and priority review for a new drug application (NDA) for vorasidenib for the treatment of IDH-mutant diffuse glioma. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of August 20, 2024. 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 2024:
Thalassemia: Two key milestones for the year, including reporting topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (Q2) and filing 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 (now mid-year).
Lower-risk Myelodysplastic Syndromes: Dose first patient in Phase 2b study of AG-946 (mid-year).
Other: Potential approval of Servier’s vorasidenib for the treatment of IDH-mutant diffuse glioma. The FDA has assigned a PDUFA action date of August 20, 2024. Agios retains certain economic rights, as described above.
First Quarter 2024 Financial Results
Revenue: Net product revenue from sales of PYRUKYND for the first quarter of 2024 was $8.2 million, compared to $5.6 million for the first quarter of 2023.
Cost of Sales: Cost of sales for the first quarter of 2024 was $0.6 million.
Research and Development (R&D) Expenses: R&D expenses were $68.6 million for the first quarter of 2024, compared to $67.3 million for the first quarter of 2023. The year-over-year comparison reflects an increase in process development expenses, offset by a decrease in workforce-related expenditures.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $31.0 million for the first quarter of 2024 compared to $28.4 million for the first quarter of 2023. The year-over-year increase was primarily attributable to an increase in commercial-related activities as we prepare for the potential approval of PYRUKYND in thalassemia.
Net Loss: Net loss was $81.5 million for the first quarter of 2024 compared to $81.0 million for the first quarter of 2023.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2024, were $714.3 million compared to $806.4 million as of December 31, 2023. 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 first quarter 2024 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.