AGIOS PHARMACEUTICALS, INC. Q3 FY2024 earnings call

• Agios' mission is to develop transformative medicines for rare diseases, focusing on red blood cell-related diseases like PKD, thalassemia, sickle cell disease, and lower risk MDS.
• Lead product mitapivat has positive Phase 3 data in PKD and thalassemia, with full enrollment in the Phase 3 RISE UP sickle cell study. Received $1.1 billion in milestone payments from vorasidenib.
• Progress in mitapivat development: Phase 3 ACTIVATE-KidsT data in pediatric PK deficiency, filing sNDA for thalassemia by end 2024. Progress in tebapivat program: FDA orphan drug designation for MDS, Phase 2b study initiation in lower risk MDS.
• Commercial preparation: Actively preparing for potential U.S. launch of mitapivat in thalassemia in 2025, with market research, disease education, and team expansion.

Third quarter 2024 net PYRUKYND revenue was $9 million, an increase of 22% compared to the third quarter of 2023. R&D expenses were $72.5 million for the third quarter, a decrease of $9.4 million compared to the third quarter of 2023. SG&A expenses were $38.5 million for the third quarter, an increase of $12.7 million compared to the prior year quarter. Agios received a total of $1.1 billion in milestone payments following the FDA approval of vorasidenib, ending the quarter with approximately $1.7 billion in cash, cash equivalents and marketable securities.

• Aim to deploy cash for potential launches of mitapivat in thalassemia in 2025 and sickle cell disease in 2026.
• Intend to submit an sNDA for mitapivat in thalassemia by the end of 2024.
• Phase 3 RISE UP sickle cell study completed enrollment and expects top line data next year.

• Concerns within the sickle cell community regarding past drug withdrawals and trust issues. Potential challenges in trial conduct, especially in certain regions related to diseases like malaria. Regulatory considerations in relation to trial data submission and approval.

Q: How are you thinking about capital allocation and appetite for business development now that vorasidenib has been largely monetized?

A: Priorities are potential launches for thal and sickle cell, advancing pipeline, and expanding pipeline internally/externally, looking for rare, transformative, with clear regulatory path.

Q: Can you speak to expectations for the Phase 3 portion of the RISE UP trial and VOC powering?

A: Phase 3 RISE UP has two primary endpoints (hemoglobin response and annualized rate of sickle cell pain crises), powered to detect differences, and completed enrollment.

Q: What's the thinking on regulatory perspective after Oxbryta withdrawal?

A: Have not changed clinical design or approach to regulators; data package submission is a collaboration, and regulators decide on benefit-risk.

Q: Can you provide more specific quantitative detail on thalassemia patient groups for initial launch?

A: ~6,000 adults diagnosed with thalassemia in US, initial launch focus on 65% of adult thalassemia patients, including transfusion-dependent, non-transfusion-dependent with comorbidities, and non-transfusion-dependent with debilitating fatigue.

Q: How are you thinking about timing and design of pediatric trials for thalassemia?

A: Similar approach to PKD, with benefit-risk profile from adult thalassemia population, timing to be determined later.

Q: When will data from tebapivat Phase 2b be shared?

A: Will provide more detail and timing next year, focusing on transfusion independence in the trial.

Q: How quickly could payer access come online for thalassemia?

A: Market access team is engaging with payers, with experience from PK deficiency launch, expecting similar success.

Q: Any details on baseline VOCs in sickle cell RISE UP trial?

A: No details provided before full data readout.

Q: Color on ensuring similar trial conduct issues don't occur in Africa?

A: Trials have good geographical distribution, data will accrue over time to observe any issues.