Analyst Conference Summary

biotechnology

conference date: November 5, 2026, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2026 (Q3, third quarter 2026)

Forward-looking statements

Overview: Record revenue, delay to FDA decision for Sickle Cell disease.

Basic data (GAAP):

Revenue was $55.4 million, up 23% sequentially from $45 million, and up 329% from $12.9 million year-earlier.

Net income was negative $65 million, up sequentially from negative $101 million, and up from negative $103 million year-earlier.

EPS (diluted GAAP) was negative $1.09, flat sequentially from negative $1.69, and up from negative $1.78 year-earlier.

Guidance:

Has cash to get to profitability in 2028.

Conference Highlights:

Brian Goff, CEO of Agios said "The third quarter was a record for our product revenue, outperforming our entire 2025 total in just three months. This growth was driven by the U.S. commercial launch of Aqvesme in thalassemia, where broadening demand is translating into a durable base of patients on therapy. This launch has built the commercial foundation for our largest indication opportunity with mitapivat: sickle cell disease. Our conviction in mitapivat for patients with sickle cell disease is grounded in the robust efficacy and safety data generated to date and we are launch-ready ahead of our PDUFA goal date of February 1, 2027. With this commercial infrastructure, alongside a strong balance sheet and a disciplined operating model, Agios is well positioned for sustained growth as a focused, rare hematology company." Goal is to achieve GAAP profitability in 2028. Confident in approval of mitapivat for Sickle Cell anemia.

Pyrukynd and Aqvesme (mitapivat) revenue was $55.4 million, up 23% sequentially from $44.7 million, and up 329% from $12.9 million year-earlier. U.S. revenue was $ million, driven by Aqvesme launch. Ex-US revenue was $ million. In Q2 approved in EU for anemia from transfusion-dependent and non-transfusion-dependent thalassemias. Sees the physician and patient reactioon to the launch as very strong. It typically takes 10 to 12 weeks to move from presciption to treatment; using a single specialty pharmacy. About 75% of patients remain on therapy after 6 months of treatment. Recent data has shown that about one-third of initial non-responders do respond with longer-term therapy.

Over the course of 2026, Agios reviewed its operating model, and will have reduced its R&D headcount by over 100 roles by year-end, representing a more than 35% decrease compared with 2025. Associated cost savings of these reductions to be realized in 2027.

Mitapivat for Sickle Cell sNDA submitted in Q2 2026. PDUFA November 1, 2026 delayed to February 1, 2027. The Phase 3 mitapivat sickle cell study topline results were reported November 2025. Met primary endpoint and two secondary endpoints, but missed two other secondary endpoints. See Agios Announced Topline Results. EU gave orphan status. In Q3 2026 Agios dosed the first patient in the Phase 3 confirmatory trial, required under the accelerated approval pathway. There are about 75 thousand potential patients.

in Q2 2026 licensed cevidoplenib for immune thrombocytopenia, from Oscotec. Expects to begin Phase 3 trial in 1H 2028.

In Q2 2026 advanced AG-236 into Phase 2/3 development for polycythemia vera. This follows positive Phase 1 results to be presented at AH. Should launch Phase 2/3 trial before the end of 2026.

The BCAT2 preclinical program targets acidemias.

AG-181 is a PAH stabilizer for phenylketonuria in Phase 1 trial. Positive data were presented at SSIEM 2026. Results support Phase 1b dosing.

Cash (including equivalents & securities) ended at $907 million, down sequentially from $965 billion. No debt.

GAAP operating expenses were $133 million, consisting of: Cost of goods $2.5 million; $73 million for R&D and $57 million for SG&A. Loss from operations was $77 million. Interest income was $9 million. Royalty income $3 million. Other income $0.27 million. Income tax $0 million.

Q&A selective summary:

PDUFA delay? REMS. Engagement is going well. Negotiating label. Delay should not affect commercial launch target. Prepared for multiple scenarios.

ASH presentation v. competing drugs? Incremental data very positive, can improve organ health in kidney and cardiac. PKN2 is a unique mechanism, no competitor with this.

Sickle Cell REMs scenarios? There is a large range. Could just be educational. Negotiations due include a potential REMS.

Mitapivat commercial position v. competitors? Baseline data can be looked at for cross trial comparison. Safety too. 25,000 potential initial patients. Will educate with clinical data, generating more open label data. Market experiencewith mitapivat safety helps. We have good communications with the community, which we will leverage.

We are reducing headcount by 100, will see a charge in q4, then reduced run rate in 2027. We will be disciplined manageing our operating expenses.

Brand name if different REMS? Too early to comment, depends on negotiations.

We have excellent payer coverage for Thalassemia, currently 75% of covered lives.

Pricing and brand strategy for sickel cell? Is a rare disease, high unmet need, no options. So believe payers will agree to rare disease pricing. There are many, however, on Medicaid.

Run rate for Aqvesme scripts? Q1 and Q2 benefitted from a bolus. 177 new per quarter is an appropriate run rate now. Revenue will depend on continuing patients as well.

Any Sickle Cell patients already (said a doctor reported this)? 619 and 48 are on label thalassemia prescriptions.

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