AGIOS PHARMACEUTICALS, INC. Q1 FY2025 earnings call

Product Focus - Brian Goff highlighted Agios' mission to develop transformative medicines for rare diseases, with multiple late-stage programs near critical readouts and a strong balance sheet. - Sarah Gheuens discussed thalassemia and sickle cell disease programs, including positive results from ENERGIZE trials and the RISE UP study for sickle cell. - Tsveta Milanova detailed commercial preparations for thalassemia launch, including disease education campaigns, team sizing, and payer engagement. - Cecilia Jones provided financial details, noting $1.4 billion in cash on hand and focus on disciplined cash allocation for future launches.

In the first quarter of 2025, Agios generated $8.7 million in net PYRUKYND revenue, an increase of 6% compared to the first quarter of 2024. The lead product PYRUKYND (mitapivat) is the focus, with potential launches in thalassemia and sickle cell disease. R&D expenses for the first quarter were $72.7 million, an increase of $4.1 million compared to the first quarter of 2024, primarily due to workforce and trial-related expenses. SG&A expenses were $41.5 million, an increase of $10.5 million, driven by commercial activities in preparation for PYRUKYND's potential approval in thalassemia.

2025 Priorities - Maximize the PYRUKYND franchise, including pursuing new indications. - Advance and diversify key pipeline programs. - Strategically deploy capital. ### Milestones - Anticipates potential U.S. approval and launch of PYRUKYND in thalassemia in September 2025, followed by sickle cell disease in 2026. - Progress expected in mid and early stage pipeline, such as tebapivat Phase 2 study in sickle cell and AG-236 IND filing in mid-2025.

Risks - Regulatory review uncertainties for thalassemia program. - Potential impact of new tariffs on operating expenses. - Market dynamics affecting trial recruitment and product adoption in rare diseases.

Q: Gregory Renza asked about mid cycle review for thalassemia and next steps with FDA.

A: Brian Goff and Sarah Gheuens responded that there's ongoing FDA engagement, no advisory committee planned, and looking forward to the September 7 PDUFA date.

Q: Gregory Renza followed up on sickle cell community and tebapivat enrollment.

A: Sarah Gheuens said there are no changes in trial conduct due to vox withdrawal, and Tsveta Milanova mentioned the sickle cell market need and partnership for ex-U.S. launch.

Q: Alec Stranahan asked about ex-U.S. launches for thalassemia and Krishnan's impact.

A: Tsveta Milanova talked about ex-U.S. plans with partners like Newbridge in GCC region, Cecilia Jones mentioned revenue split partnership, and Brian Goff discussed Krishnan's role in strengthening corporate strategy.

Q: Divya Rao asked about FDA communication changes.

A: Sarah Gheuens said FDA communication is normal, with ongoing review and PDUFA date set for September 7.

Q: Hiro Nagayumi asked about tebapivat Phase 2 start.

A: Sarah Gheuens explained the Phase 2 is a dose-finding study for proof-of-concept, and Tsveta Milanova discussed the patient population approach in sickle cell.

Q: Emily Bodnar asked about thalassemia marketing for non-transfusion dependent patients.

A: Tsveta Milanova discussed initial launch focus on both transfusion dependent and non-transfusion dependent patients with complications.

Q: Unidentified Analyst asked about tebapivat study details and liver impact.

A: Sarah Gheuens and Brian Goff explained Phase 2 design and safety monitoring for tebapivat, noting no liver signal observed yet.

Q: Tessa Romero asked about mid cycle meeting and labeling.

A: Sarah Gheuens said it's a normal FDA engagement process with ongoing review, and labeling negotiations are too early to discuss.

Q: Unidentified Analyst asked about thalassemia pricing.

A: Sarah Gheuens said pricing will be anchored in label value, with positive payer feedback on the product's unmet need