• Net Pyrukynd Revenue (Q4 2024): $10.7 million, a 51% increase from $7.1 million in Q4 2023.
• Cost of Sales (Q4 2024): $1.3 million.
• R&D Expenses (Q4 2024): $82.8 million, an increase of $5.3 million from Q4 2023.
• SG&A Expenses (Q4 2024): $51.7 million, an increase of $16.4 million from Q4 2023.
• Cash Equivalents and Marketable Securities (End of Q4 2024): Approximately $1.5 billion.
• Milestone Payments (2024): Total of $1.1 billion, including $905 million from Royalty Pharma and $200 million from Servier.

• Warning! GuruFocus has detected 4 Warning Signs with AGIO.

Release Date: February 13, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Agios Pharmaceuticals Inc (NASDAQ:AGIO) reported a 51% increase in net Pyrukynd revenue for Q4 2024 compared to the same quarter in 2023.
• The company is preparing for two additional commercial launches, with potential approval and launch of Pyrukynd in thalassemia expected in September 2025 and in sickle cell disease in 2026.
• Agios has a robust early and mid-stage pipeline poised for clinical advancement, including the completion of enrollment in the phase 3 rise up study for sickle cell disease.
• The company has a strong balance sheet with approximately $1.5 billion in cash, equivalents, and marketable securities, providing financial independence for future growth.
• Agios announced positive top-line results from the Activate Kids Phase 3 trial of Mitapivat in pediatric patients with PK deficiency, marking its first pediatric clinical program for a rare hemolytic anemia.

Negative Points

• Agios Pharmaceuticals Inc (NASDAQ:AGIO) expects flat revenues for PK deficiency in 2025 compared to 2024, indicating limited growth in this area.
• The company noted that Q4 2024 revenues were higher due to year-end stocking and adjustments, which are not expected to repeat in Q1 2025.
• There are concerns about liver toxicity in the sickle cell trial, leading to changes in the monitoring protocol.
• The launch in the Gulf region, particularly Saudi Arabia, is expected to take time due to the need for formulary access and healthcare system navigation.
• Agios faces challenges in pediatric development, which is complex and requires significant logistical considerations.

Q & A Highlights

Q: What is Agios Pharmaceuticals' strategy for updating the investment community on the safety profile of Mitapivat, especially concerning hepatocellular injury? A: Sarah Gheuens, Chief Medical Officer, stated that if there are any changes to the safety profile of Mitapivat, the company will update the investment community as they have done in the past when new safety information became available.

Q: How does Agios Pharmaceuticals view the peak sales potential for Mitapivat in thalassemia and sickle cell disease? A: Brian Goff, CEO, expressed confidence in the multi-billion-dollar potential of Pyrukynd, citing the significant unmet need in thalassemia, where two-thirds of the U.S. patient population has no approved therapy, and the high unmet need in sickle cell disease, which has increased due to limitations in available therapeutic options.

Q: What changes were made to the sickle cell trial protocol following the liver toxicity disclosure? A: Sarah Gheuens explained that monitoring for liver enzymes was already part of the core period of trials. The open-label extension (OLE) portion was adjusted to align with the core period's monthly monitoring for the first six months of exposure.

Q: How does Agios Pharmaceuticals plan to approach the development of Tepapivat in sickle cell disease? A: Brian Goff and Tsveta Milanova, Chief Commercial Officer, indicated that they are building a sickle cell disease franchise with both Pyrukynd and Tepapivat. The development path will be guided by data from ongoing studies, and they aim to provide multiple treatment options for this complex disease.

Q: How should investors model the launch trajectory for Mitapivat in thalassemia, considering potential pent-up demand? A: Tsveta Milanova noted that while there is a focus on educating and increasing urgency among physicians, they do not expect an initial bolus of patients. The launch will target about 65% of the U.S. thalassemia patient population, capturing patients as they visit their doctors.

For the complete transcript of the earnings call, please refer to the full earnings call transcript.