On November 2, 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 third quarter ended September 30, 2023 (Press release, Agios Pharmaceuticals, NOV 2, 2023, View Source [SID1234636722]).
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"Agios is approaching a catalyst-rich period, with three mid-to-late-stage data readouts expected by the end of next year, and a total of six by the end of 2025," said Brian Goff, chief executive officer at Agios. "We are excited to report dosing of the first patient in the Phase 3 portion of the pivotal RISE UP study of our leading PK activator, mitapivat, in sickle cell disease and look forward to sharing more detailed data from the positive Phase 2 portion of RISE UP at an upcoming medical meeting. We look forward to future data readouts across our industry-leading pipeline of PK activators, including the Phase 2a study of AG-946 in lower-risk MDS by the end of this year and both Phase 3 studies of mitapivat in thalassemia next year."
Third Quarter 2023 & Recent Highlights
PYRUKYND U.S. Launch: Generated $7.4 million in U.S. net revenue for the third quarter of 2023, a 10 percent increase over the second quarter of 2023. A total of 160 unique patients have completed prescription enrollment forms, representing an increase of 9 percent over the second quarter of 2023. A total of 100 patients are on PYRUKYND therapy.
Sickle Cell Disease: Dosed first patient in the Phase 3 portion of the RISE UP pivotal study of mitapivat.
Pediatric PK Deficiency: Completed enrollment in the Phase 3 ACTIVATE-kidsT study of mitapivat in regularly transfused pediatric patients with PK deficiency. Achieved goal of >50% enrollment in Phase 3 ACTIVATE-kids study.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following additional key milestones and priorities in the coming months:
Sickle Cell Disease: Present data from the positive Phase 2 portion of the RISE UP study of mitapivat at an upcoming medical meeting. Advance patient enrollment in the Phase 3 portion of RISE UP.
Lower-risk Myelodysplastic Syndromes (LR-MDS): Announce topline data from the Phase 2a study of novel PK activator AG-946 by year-end 2023.
Thalassemia: Announce topline data from the two Phase 3 studies of mitapivat in non-transfusion-dependent and transfusion-dependent thalassemia in the first and second halves of 2024, respectively.
Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat in non-regularly transfused pediatric PK deficiency next year.
Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU) by year-end 2023.
Data Presentations: Present broad set of clinical and translational data at the 65th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition; abstracts will be available at 9 a.m. ET today.
Third Quarter 2023 Financial Results
Revenue: Net U.S. product revenue from sales of PYRUKYND for the third quarter of 2023 was $7.4 million, compared to $3.5 million for the third quarter of 2022.
Cost of Sales: Cost of sales for the third quarter of 2023 was $0.6 million.
Research and Development (R&D) Expenses: R&D expenses were $81.8 million for the third quarter of 2023, compared to $65.0 million for the third quarter of 2022. The year-over-year increase was primarily driven by the $17.5 million upfront payment to Alnylam for the TMPRSS6 asset.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $25.8 million for the third quarter of 2023 compared to $29.1 million for the third quarter of 2022. The year-over-year decrease was primarily attributable to lower stock-based compensation expense and reduced professional fees.
Net Loss: Net loss was $91.3 million for the third quarter of 2023 compared to $81.7 million for the third quarter of 2022.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2023, were $872.4 million 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, 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 potential 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 third 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.