• Net Revenue: $8.7 million in Q1 2025, a 6% increase from $8.2 million in Q1 2024.
• Cash and Equivalents: Approximately $1.4 billion on hand.
• R&D Expenses: $72.7 million in Q1 2025, up $4.1 million from Q1 2024.
• SG&A Expenses: $41.5 million in Q1 2025, an increase of $10.5 million from the prior year quarter.
• Cost of Sales: $1.1 million for Q1 2025.
• Patient Enrollment: 234 patients completed prescription enrollment forms, with 136 net patients on therapy, both increasing by 5% from the prior quarter.

• Warning! GuruFocus has detected 5 Warning Signs with AGIO.

Release Date: May 01, 2025

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

Positive Points

• Agios Pharmaceuticals Inc (NASDAQ:AGIO) has a strong balance sheet with approximately $1.4 billion in cash, providing financial independence for future product launches and pipeline advancements.
• The company is preparing for potential commercial launches of PYRUKYND in thalassemia in 2025 and sickle cell disease in 2026, representing a multi-billion dollar opportunity.
• Positive top-line results were announced from the Activate-Kids Phase 3 trial of mitapivat in pediatric patients with PK deficiency, indicating progress in their pipeline.
• Agios Pharmaceuticals Inc (NASDAQ:AGIO) has a robust early and mid-stage pipeline poised for clinical advancement, offering a strong foundation for innovation and long-term growth.
• The company has a highly experienced team and has recently added Krishnan Viswanadhan as Chief Corporate Development and Strategy Officer to maximize asset potential and explore expansion opportunities.

Negative Points

• First quarter 2025 net revenue was $8.7 million, a decrease of 19% compared to the fourth quarter of 2024, primarily due to year-end stocking benefits and revenue reserve adjustments.
• R&D expenses increased by $4.1 million compared to the first quarter of 2024, driven by workforce-related expenses and clinical trial costs.
• SG&A expenses rose by $10.5 million compared to the prior year quarter, mainly due to increased commercial activities and headcount in preparation for potential PYRUKYND approval.
• The company anticipates relatively flat revenues for PK deficiency in 2025 compared to 2024, indicating limited growth in this area.
• There is uncertainty regarding the regulatory review process, as the FDA has not yet finalized labeling negotiations or confirmed the absence of a REMS requirement for PYRUKYND in thalassemia.

Q & A Highlights

Q: Has the mid-cycle review with the FDA for PYRUKYND in thalassemia occurred, and what are the next steps in the regulatory process? A: Brian Goff, CEO, confirmed that the process with the FDA is progressing well, with no advisory committee meeting planned at this time. Sarah Gheuens, Chief Medical Officer, added that they are engaged with the FDA and are looking forward to the PDUFA date of September 7th.

Q: How have current market conditions affected the recruitment of trials in the sickle cell community, particularly for Tebapivat? A: Sarah Gheuens stated that the withdrawal of VOCs was disappointing, but it has not impacted their clinical trial conduct. Tsveta Milanova, Chief Commercial Officer, emphasized the large market and unmet need in sickle cell disease, expressing excitement about the potential opportunity to serve the community with innovative therapies.

Q: What are Agios' plans for launching PYRUKYND outside the US, particularly in thalassemia? A: Tsveta Milanova explained that the Gulf region, particularly Saudi Arabia, is a priority for ex-US launches. They have a partnership with New Bridge to leverage regional expertise. Cecilia Jones, CFO, mentioned a revenue split model with New Bridge and plans for a similar structure in Europe.

Q: What is the rationale for starting the Phase 2 Tebapivat study in sickle cell disease before the Phase 3 readout of PYRUKYND? A: Sarah Gheuens highlighted the promising Phase 1 data for Tebapivat, justifying the move to Phase 2. Tsveta Milanova added that the large unmet need in sickle cell disease supports the development of multiple therapies, and they plan to position Tebapivat based on upcoming data.

Q: Can you comment on the anticipated pricing strategy for PYRUKYND in thalassemia? A: Tsveta Milanova stated that pricing decisions will be based on the product's value proposition and label. The payer feedback has been positive, recognizing the unmet need and product profile, and they are confident in navigating the pricing strategy effectively.

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