Agios Pharmaceuticals (AGIO) Q3 2024 earnings summary

Executive summary

• Achieved net income of $947.9 million in Q3 2024, primarily from $1.1 billion in milestone and royalty payments after FDA approval of vorasidenib and the sale of royalty rights.

• PYRUKYND®/mitapivat revenue rose to $9.0 million in Q3 2024, up 22% year-over-year, with 127 patients on therapy and strong clinical progress in thalassemia, sickle cell disease, and pediatric PK deficiency.

• Completed enrollment in Phase 3 RISE UP study for sickle cell disease; positive Phase 3 data in thalassemia and pediatric PKD; initiated Phase 2b tebapivat study in MDS with orphan drug designation.

• Entered distribution agreement for PYRUKYND® in the GCC region, expanding international reach and targeting high-prevalence markets.

• Positioned for multi-billion-dollar rare disease markets with a robust pipeline and differentiated PK activation franchise.

Financial highlights

• Q3 2024 net income was $947.9 million, compared to a net loss of $91.3 million in Q3 2023, driven by $1.1 billion in vorasidenib-related payments.

• PYRUKYND® net revenue reached $9.0 million in Q3 2024, up from $7.4 million in Q3 2023; cost of sales was $0.8 million.

• R&D expenses were $72.5 million, down from $81.8 million in Q3 2023; SG&A expenses rose to $38.5 million from $25.8 million due to commercial launch preparations.

• Cash, cash equivalents, and marketable securities totaled $1.7 billion as of September 30, 2024, up from $806.4 million at year-end 2023.

• Net income margin and EPS for Q3 2024 were exceptionally high due to one-time vorasidenib payments; Q3 2024 EPS was $16.22 diluted.

Outlook and guidance

• sNDA submission for mitapivat in thalassemia expected by year-end 2024, with anticipated U.S. launches in thalassemia (2025) and sickle cell disease (2026).

• Cash position expected to fund operations, launches, and pipeline expansion for at least the next twelve months.

• Expects continued commercial expansion of PYRUKYND® into new geographies and patient populations.

• Additional clinical data to be presented at ASH Congress in December 2024.