Agios Announces Q3 2024 Financial Results and Business Highlights

Agios Pharmaceuticals, Inc., based in Cambridge, Massachusetts, has reported key business and financial results for the third quarter ending September 30, 2024. This period was marked by significant progress in their clinical programs and financial stability.

Agios has completed the enrollment for the Phase 3 RISE UP study of mitapivat in sickle cell disease, with more than 200 patients participating globally. The topline data from this 52-week study are anticipated in late 2025. Additionally, the Phase 2b study of tebapivat in patients with lower-risk myelodysplastic syndromes (LR-MDS) has commenced, following the FDA’s orphan drug designation granted for this indication.

The company reported $9.0 million in net revenue for PYRUKYND® (mitapivat) for the third quarter, reflecting a 4% increase from the previous quarter. This growth was driven by heightened patient demand, with 211 unique patients having completed prescription enrollment forms and 127 patients actively receiving PYRUKYND® therapy.

Agios also highlighted significant corporate developments, including receiving $1.1 billion in payments from Royalty Pharma and Servier following the FDA's approval of vorasidenib. This strengthens Agios' financial position and supports its growth and development initiatives. The company also entered a distribution agreement with NewBridge Pharmaceuticals to commercialize PYRUKYND® in the Gulf Cooperation Council (GCC) region, covering Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, and the United Arab Emirates.

In terms of upcoming milestones, Agios plans to file a supplemental New Drug Application (sNDA) for mitapivat in thalassemia based on positive results from the Phase 3 ENERGIZE and ENERGIZE-T clinical trials. The company also intends to present additional clinical data at the 66th Annual American Society of Hematology (ASH) Congress in December 2024.

Financially, Agios reported net product revenue from sales of PYRUKYND® rose to $9.0 million for the third quarter of 2024, compared to $7.4 million in the same period in 2023. The cost of sales was $0.8 million for the quarter. Research and development expenses were reported at $72.5 million, a decrease from $81.8 million in the third quarter of 2023, primarily due to a significant upfront payment made in the prior year. Selling, general, and administrative expenses rose to $38.5 million compared to $25.8 million in the same quarter of 2023, driven by increased commercial activities in preparation for the potential approval of PYRUKYND® in thalassemia.

Agios recorded a substantial net income of $947.9 million for the third quarter of 2024, a stark contrast to a net loss of $91.3 million in the same period the previous year. This remarkable turnaround is attributed to milestone and royalty agreement income recorded in the current quarter.

As of September 30, 2024, Agios had $1.7 billion in cash, cash equivalents, and marketable securities, up from $806.4 million as of December 31, 2023. The company expects that its financial reserves, combined with anticipated product revenue and interest income, will support its initiatives, including potential launches of PYRUKYND® in thalassemia and sickle cell disease, and the advancement of existing and new programs.

In summary, Agios Pharmaceuticals has made significant strides in its clinical programs and maintained a strong financial performance in the third quarter of 2024. The company remains focused on advancing its pipeline and bringing innovative therapies to patients with rare diseases.