Agios Pharmaceuticals, Inc. Q2 FY2025 earnings call

Key Points

• 2025 is a breakout year with PYRUKYND's derisked multibillion-dollar opportunity, near-term catalysts including PDUFA for thalassemia (less than 40 days from September 7 goal date), RISE UP Phase III sickle cell data by end of year/early next year, and Phase IIb data for tebapivat.
• Second quarter net revenue was $12.5 million. Ended Q2 with ~$1.3 billion in cash, etc. Entered agreements with Avanzanite and NewBridge for European and GCC commercialization.
• Dosed first patient in tebapivat Phase II trial for sickle cell and received IND clearance for AG-236. Strong balance sheet supports capital allocation to U.S. launches, pipeline investment, and potential pipeline expansion.
• Commercial highlights: 248 patients completed prescription enrollment forms in Q2, 142 on active PYRUKYND treatment; confident in U.S. launch of PYRUKYND for thalassemia with robust clinical data.
• R&D highlights: PYRUKYND's clinical data across hemolytic anemias, Phase III ENERGIZE studies for thalassemia, RISE UP Phase II data for sickle cell, and multiple abstracts at European Hematology Association Congress.

In the second quarter, Agios reported $12.5 million in net revenue, primarily from PYRUKYND. The company entered into agreements with Avanzanite Bioscience to commercialize and distribute PYRUKYND in Europe and with NewBridge Pharmaceuticals for the GCC, with these being capital-efficient revenue-sharing arrangements. Net revenue from PYRUKYND was $12.5 million in Q2 2025, a 45% increase from Q2 2024 and 44% increase from Q1 2025.

Forward-Looking Statements

• Anticipate PDUFA for thalassemia in the U.S. by September 7.
• Expect to read out RISE UP Phase III trial for PYRUKYND in sickle cell disease by end of 2025/early 2026.
• Anticipate Phase IIb data for tebapivat in lower-risk myelodysplastic syndromes.
• Well-capitalized to develop and launch PYRUKYND in thalassemia and sickle cell disease, and advance pipeline through internal efforts and business development.

Risks Discussed

• Potential variability in net revenues due to ordering patterns for rare disease medicines.
• Uncertainties related to clinical trial outcomes, including safety profiles like liver toxicity (though no recent updates reported).
• Market access challenges in ex-U.S. regions, such as fragmented access in the GCC and regulatory review timelines in Europe.

Q: Any update on mitapivat safety profile, including liver toxicity outside thalassemia?

A: Sarah Gheuens states no updates to the safety profile.

Q: Color on GCC approval date?

A: Brian M. Goff says team submitted simultaneously across 4 regions, actively in discussions, and Tsveta's team is ready commercially.

Q: Labeling discussions for thalassemia?

A: Sarah Gheuens says update to PKD label to reflect indication and dose, but final label at PDUFA date.

Q: SG&A spend for thalassemia launch?

A: Cecilia Jones says more growth expected on SG&A, with launch-related expenses upon approval; Tsveta says team is ready for potential launch.

Q: Pediatric opportunity for thalassemia?

A: Tsveta Milanova mentions ~2,000 pediatric patients in U.S., Sarah Gheuens says plan is to expand trials in pediatric population after adult data.

Q: PKU pipeline after PTC's approval?

A: Sarah Gheuens says no changes to plans, PKU program remains with novel mechanism addressing unmet need.

Q: Sickle cell trial protocol changes re: liver injury?

A: Sarah Gheuens says protocols aligned with monthly monitoring for first 6 months, including open-label extension; dosing in sickle cell trial lower due to metabolism matching healthy volunteers.

Q: Hepatocellular injury in label and open-label extension data?

A: Sarah Gheuens says open-label extension data to be published, HCI language in label under ongoing review with FDA.

Q: Prescriber base for thalassemia launch?

A: Sarah Gheuens says thalassemia patients have established ICD-10 codes, team focused on launch preparations, engaging prescribers in academic and community centers.