Analyst Conference Summary

biotechnology

conference date: February 12, 2026, @ 5:00 AM Pacific Time
for quarter ending: December 31, 2025 (Q4, fourth quarter 2025)

Forward-looking statements

Overview: Increasing Pyrukynd sales, but far from break-even.

Basic data (GAAP):

Revenue was $20.0 million, up 55% sequentially from $12.9 million, and up 87% from $10.7 million year-earlier.

Net income was negative $108 million, down sequentially from negative $103 million, and down from negative $96.5 million year-earlier.

EPS (diluted GAAP) was negative $1.86 [not stated, so my calculation], down sequentially from negative $1.78, and down from $na year-earlier.

Guidance:

In 2026 expects U.S. PK revenue to be between $45 and $50 million. Ex-US sales expected to decline in Q1 compared to Q4. Expects operating expenses in 2026 to be roughly flat compared to 2025.

Conference Highlights:

Brian Goff, CEO of Agios said "2025 was another year of continued execution across our portfolio, highlighted by the historic U.S. approval of Aqvesme – the only medicine approved to treat anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The U.S. launch is off to a strong start, with Aqvesme now available and earning an enthusiastic response from the thalassemia community. In 2026, we are focused on driving a high-impact U.S. launch of Aqvesme, expanding our PK activation franchise into additional high-value indications such as sickle cell disease and lower-risk myelodysplastic syndromes, and advancing our promising early-stage pipeline to further diversify across hematologic and other rare diseases. With disciplined capital allocation and strong operational execution, we are very well positioned at this critical inflection point to deliver a transformative medicine for the thalassemia community and advance our clinical programs as we work toward our goal of becoming a sustainable rare disease company."

Hopes to expand treatment into MDS (myelodysplastic syndrome) and sickle cell anemia.

Pyrukynd and Aqvesme (mitapivat) revenue was $20.0 million, up 55% sequentially from $12.9 million, and up 87% from $10.7 million year-earlier. U.S. revenue was $16.0 million, ex-US $4.0 million, reflecting mainly stocking. There were 44 presciptions of Aqvesme for thalassemia in the U.S. by the end of January 2026. 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.

Reported positive mitapivat full Phase 3 transfusion-dependent thalassemia results in December 2024. The FDA approved Aqvesme (mitapivat for thalassemia) in December 2025. Initial launch will target 4,000 U.S. patients with high frequency doctor visits. EU decision, following the positive CHMP recomendation in October 2025, likely in early 2026. In EU Agios is partnered with Avanzanite Bio. Was approved for sale in Saudi Arabia in August 2025. There are about 6,000 diagnosed adult thalassemia patients in the U.S.

Completed enrollment of Phase 3 mitapivat sickle cell study in Q3 2024, with topline results reported November 2025, possible sickle cell launch in 2026. EU gave orphan status.

Tebapivat (AG-946) for low-risk MDS Phase 2b completed enrollment in Q3 2025, topline data expected 1H 2026. In tebapivat for sickle cell the Phase 2 trial enrollment completed in Q4 2025.

Believes over $10 billion in potential global revenue by 2030 [with the big bump coming following sickle cell disease approval.]

Believes there could be 4,000 on-label Pyrukynd patients in the U.S., which could lead to annual revenue of $200 to $225 million. Thalassemia and sickle cell potential patient numbers are much larger [18,000 and 120,000].

The BCAT2 preclinical program targets acidemias.

AG-181 is a PAH stabilizer for phenylketonuria in an ongoing Phase 1 trial.

In Q3 2025 Phase 1 of AG-236 for polycemia vera was launched. It is an siRNA targetting TMPRSS6.

Cash (including equivalents & securities) ended at $1.16 billion, down sequentially from $1.26 billion. No debt.

GAAP operating expenses were $141.6 million, consisting of: Cost of goods $1.9 million; $88.1 million for R&D and $51.6 million for SG&A. Loss from operations was $101.6 million. Interest income was $6.4 million. Other income $0 million. Income tax $0 million.

Full year 2025 revenue was $54.0 million. Net income was negative $413 million. EPS negative $7.12.

Q&A selective summary:

Aqvesme launch, revenue recognition? Very encouraged by early demand. 44 prescriptions in first 5 weeks of launch. We expect prescriptions to grow in advance of revenue due to time lag.

Tebapivat sickle cell? Phase 2 is a dose finding trial. Community support shown by enrollment completion.

Aqvesme scripts since January? Not giving data past first 44. Educating physicians on the value proposition. Many prescriptions are in the community setting. 10 to 12 week lag is largely driven by need for insurance authorization, but also the liver test. Over time could shorten the lag, particularly if payers put Aqvesme on formulary.

Tebapivat in MDS? Looking at higher doses. Looking at subsets of MDS patients, like low and high burden. It has the advantages of being an oral therapy.

REMs requirement has not been a problem, it is quick once a physician wants to prescibe.

Ex-US revenue in Q4 was mainly driven by Europe. GCC (Saudi, etc.) is case by case, will take time to build traction.

Sickle cell launch costs? We will manage our spend, we already have an infrastructure, but we would need to scale it up.

We do not believe we will need a REMS in sickle cell disease. But if we do need one, we have the experience with thalassemia.

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