Agios Reports Q1 2024 Highlights and Financial Results

Agios Pharmaceuticals, Inc., a pioneer in cellular metabolism and PK activation for rare diseases, released its business highlights and financial results for the first quarter ending March 31, 2024. The company reported positive data from the Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent thalassemia. Chief Executive Officer Brian Goff emphasized mitapivat's potential to be the first therapy approved for all thalassemia subtypes, with plans for a potential launch next year. The company also anticipates results from the RISE UP Phase 3 study in sickle cell disease next year, aiming for approval in 2026.

First Quarter 2024 and Recent Highlights

Sales of PYRUKYND® (mitapivat) generated $8.2 million in net revenue for the first quarter of 2024, marking a 15 percent increase from the previous quarter, driven by higher patient demand. The number of unique patients completing prescription enrollment forms rose by 6 percent from the last quarter of 2023. Currently, 120 patients are on PYRUKYND® therapy, a 10 percent increase from the fourth quarter of 2023.

Agios disclosed positive results from the Phase 3 ENERGIZE study involving adults with non-transfusion-dependent alpha- or beta-thalassemia. Additionally, the company has initiated a Phase 1 study of AG-181 for treating phenylketonuria (PKU). The 2024 ESG Report was also released, covering corporate sustainability disclosures for the past year.

Furthermore, Servier announced that the FDA had accepted a new drug application for vorasidenib, targeting IDH-mutant diffuse glioma, with a Prescription Drug User Fee Act (PDUFA) action date set for August 20, 2024. As part of Agios' previous oncology business divestment to Servier, the company retains rights to a potential $200 million milestone upon FDA approval and 15% royalties on potential U.S. net sales.

Key Upcoming Milestones & Priorities

Agios expects to achieve several key milestones by the end of 2024. These include reporting topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia and filing for FDA approval of mitapivat in thalassemia by year-end. The company aims to complete enrollment in the Phase 3 RISE UP study of mitapivat for sickle cell disease by year-end. Another milestone includes completing enrollment in the Phase 3 ACTIVATE-kids study of mitapivat for pediatric PK deficiency by mid-year and reporting topline data from the Phase 3 ACTIVATE kids-T study around the same time. Additionally, Agios plans to dose the first patient in a Phase 2b study of AG-946 for lower-risk myelodysplastic syndromes by mid-year.

First Quarter 2024 Financial Results

Net product revenue from PYRUKYND® sales for the first quarter of 2024 was $8.2 million, a significant increase from $5.6 million in the first quarter of 2023. The cost of sales for the quarter was $0.6 million. Research and development expenses totaled $68.6 million, compared to $67.3 million in the first quarter of 2023, reflecting increased process development expenses but reduced workforce-related costs. Selling, general, and administrative expenses were $31.0 million, up from $28.4 million in the same period last year, mainly due to increased commercial activities in preparation for potential PYRUKYND® approval in thalassemia. The net loss for the quarter was $81.5 million, compared to $81.0 million in the first quarter of 2023.

Cash, cash equivalents, and marketable securities as of March 31, 2024, stood at $714.3 million, down from $806.4 million as of December 31, 2023. Agios anticipates that its current cash reserves, coupled with expected product revenue, interest income, and milestones from vorasidenib, will fund its operations and capital expenditures at least into 2026. This projection does not include potential cash inflows from royalties or monetization of royalties from vorasidenib, commercializing mitapivat outside the U.S., or other strategic business or financial agreements.

Overall, Agios Pharmaceuticals continues to make strides in its rare disease therapies, focusing on achieving significant milestones and preparing for future product launches.