AGIOS PHARMACEUTICALS (AGIO) Company Report

Agios announced the FDA approval of Acvezmi for thalassemia on December 23rd last year, with a U.S. price of $425,000 per patient per year and an addressable U.S. launch population of approximately 4,000 adult patients. The worldwide market potential for Acvezmi is over $1 billion.
The company is pursuing regulatory clarity for mitapivat in sickle cell disease with a pre-SNDA meeting with the FDA in Q1 2026, following positive Phase 3 RISE UP study results.
Key pipeline readouts for 2026 include Phase 2b results for tevapivat in low-risk MDS in the first half and Phase 2 data for tevapivat in sickle cell disease in the second half.
Agios projects a total estimated global market size for its current pipeline indications of over $10 billion by 2030, with a commitment to financial discipline by keeping 2026 operating expenses flat to 2025 and a clear path to profitability.

2 days ago

Transcript

Agios Discusses Recent Thalassemia Approval, Pipeline Advancements, and 2026 Outlook

AGIO

Product Launch

New Projects/Investments

Guidance Update

Agios announced the recent FDA approval of Aqvesme for thalassemia at the end of 2025, with a U.S. price point of $425,000 per patient per year and an addressable market of approximately 4,000 adult patients in the U.S.. The company is focused on a high-impact launch, with drug availability expected by the end of January 2026.
The company's pyruvate kinase (PK) activator franchise is expanding, with mitapivat (Pyrukynd) already approved for pyruvate kinase deficiency and thalassemia, and pursuing sickle cell disease. The RISE UP Phase 3 study for mitapivat in sickle cell disease showed a strong anti-hemolytic profile, and Agios plans to discuss this data with the FDA in a pre-sNDA meeting in Q1 2026.
Agios is also advancing tebapivat, a more potent PK activator, for sickle cell disease (Phase 2 data expected in H2 2026) and low-risk Myelodysplastic Syndromes (MDS) (Phase 2b results expected in H1 2026). The total market potential for these pipeline indications (thalassemia, sickle cell disease, and low-risk MDS) is estimated to exceed $7 billion worldwide by 2030.
The company projects a clear path to profitability based on its current commercial portfolio (Pyrukynd and Aqvesme) and is guiding towards keeping 2026 operating expenses flat to 2025.
Key 2026 catalysts include the pre-sNDA meeting with the FDA for mitapivat in sickle cell disease, Phase 2b readout for tebapivat in low-risk MDS, Phase 1 healthy volunteer data for AG236 (polycythemia vera) in H1 2026, and tebapivat Phase 2 data for sickle cell disease and Phase 1b proof of mechanism data for AG-181 (phenylketonuria) in H2 2026.

2 days ago

Transcript

Agios Provides 2026 Outlook and Pipeline Updates at J.P. Morgan Healthcare Conference

AGIO

Product Launch

Guidance Update

New Projects/Investments

Agios announced the FDA approval of Acvezmi for thalassemia on December 23, 2025, with a worldwide market potential exceeding $1 billion. The U.S. launch is underway, targeting 4,000 adult patients at a price of $425,000 per patient per year.
The company is expanding its PK activation franchise into sickle cell disease and low-risk MDS, which together represent a worldwide market potential exceeding $7 billion by 2030.
Key pipeline milestones for 2026 include a pre-SNDA meeting with the FDA for mitapivat in sickle cell disease in Q1, Phase 2b data for tevapivat in low-risk MDS in H1, and Phase 2 data for tevapivat in sickle cell disease in H2.
Agios is committed to financial discipline, guiding towards keeping 2026 operating expenses flat to 2025, and anticipates a clear path to profitability from its current commercial portfolio.

2 days ago

Transcript

Agios Announces AQVESME Approval and Pipeline Advancements

AGIO

Product Launch

New Projects/Investments

Guidance Update

Agios received FDA approval for AQVESME™ (mitapivat) in thalassemia in December 2025, with a broad label for alpha- or beta-thalassemia, targeting 4,000 addressable U.S. patients at launch and an annual U.S. WAC of $425k.
The company anticipates a pre-sNDA meeting with the FDA for mitapivat in Sickle Cell Disease in Q1 2026, following positive Phase 3 RISE UP data in Q4 2025.
Key pipeline catalysts for 2026 include tebapivat Phase 2b topline data for Lower-Risk MDS in H1 2026 and tebapivat Phase 2 topline data for Sickle Cell Disease in H2 2026.
Agios aims for a clear path to profitability with its existing commercial portfolio, having generated approximately $35M Net Revenues in 2025 as of Q3 from PYRUKYND in PK Deficiency.
AQVESME carries a boxed warning for hepatocellular injury and requires liver monitoring at baseline and every 4 weeks for the first 24 weeks of treatment.

2 days ago

Presentation

Agios Pharmaceuticals Outlines 2026 Strategic Priorities and Key Milestones

AGIO

Product Launch

New Projects/Investments

Guidance Update

Agios Pharmaceuticals is executing the U.S. commercial launch of AQVESME™ (mitapivat) for thalassemia, following its FDA approval in December 2025, with the medicine expected to be available in late January 2026.
The company projects a clear path to profitability, with the potential to achieve over $1 billion in peak global sales from its existing commercial presence in thalassemia and PK deficiency.
Key pipeline milestones for 2026 include a pre-sNDA meeting with the FDA for mitapivat in sickle cell disease in Q1 2026, topline results for tebapivat in lower-risk myelodysplastic syndromes in H1 2026, and topline results for tebapivat in sickle cell disease in H2 2026.
Agios anticipates Operating Expenses in 2026 to be flat compared to 2025.

4 days ago

8-K

Agios receives FDA approval for AQVESME

AGIO

Product Launch

New Projects/Investments

Agios has received FDA approval for AQVESME (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.
AQVESME is the first FDA-approved medicine for anemia in both non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia, and the first oral medicine for this condition.
The U.S. launch targets an initial 4,000 addressable patients with an annual U.S. Wholesale Acquisition Cost (WAC) of $425,000, and the company anticipates $1 billion in global peak-year sales across pyruvate kinase deficiency (PKD) and thalassemia indications.
A REMS program will be implemented, requiring liver monitoring and certification for prescribers, patients, and pharmacies due to the risk of hepatocellular injury.

Dec 24, 2025, 1:00 PM

Presentation

Agios Pharmaceuticals Receives FDA Approval for Axazeni

AGIO

Product Launch

Guidance Update

New Projects/Investments

Agios Pharmaceuticals (AGIO) announced the FDA approval of Axazeni (mitapivat) for the treatment of anemia in adults with alpha or beta thalassemia, regardless of transfusion burden. This marks the first and only medicine for this broad indication and the first disease-modifying treatment for non-transfusion-dependent thalassemia.
Axazeni will be introduced at approximately $425,000 per patient per year on a WAC basis. The initial addressable U.S. patient population at launch is estimated at 4,000 patients out of a total of 6,000 diagnosed, actively managed adult thalassemia patients.
The drug has an associated Risk Evaluation and Mitigation System (REMS) requiring liver testing at baseline and every four weeks for the first 24 weeks of treatment. The REMS program is expected to be fully operational in late January, with no prescriptions fulfilled until then.
Due to the REMS implementation and patient onboarding, the time from prescription to treatment initiation is expected to be 10-12 weeks initially, leading to a lag between prescription demand and revenue recognition in the early quarters of the launch.

Dec 24, 2025, 1:00 PM

Transcript

Agios Pharmaceuticals Announces FDA Approval of Aqneusa

AGIO

Product Launch

New Projects/Investments

Agios Pharmaceuticals announced the FDA approval of Aqneusa (mitapivat) for the treatment of anemia in adults with alpha or beta thalassemia, regardless of transfusion burden. This marks the first and only medicine for this broad patient population and the first disease-modifying treatment for non-transfusion-dependent thalassemia.
Aqneusa is an oral, twice-daily pyruvate kinase activator. It demonstrated a hemoglobin response in 42.3% of non-transfusion-dependent patients and a transfusion reduction response in 30.4% of transfusion-dependent patients in pivotal trials.
The U.S. market opportunity is approximately 4,000 addressable patients at launch, with a target price of approximately $425,000 per patient per year on a WAC basis.
Aqneusa is expected to be available in late January , with an initial estimated 10-12 weeks from prescription to treatment initiation due to the required REMS program.
The company plans to engage with the FDA in Q1 next year to discuss the regulatory path for mitapivat in sickle cell disease based on phase 3 RISE UP data.

Dec 24, 2025, 1:00 PM

Transcript

Agios Pharmaceuticals Announces FDA Approval of Axazeni for Thalassemia

AGIO

Product Launch

New Projects/Investments

Revenue Acceleration/Inflection

Agios Pharmaceuticals announced the FDA approval of Axazeni (mitapivat) for the treatment of anemia in adults with alpha or beta thalassemia, making it the first and only medicine indicated for both non-transfusion-dependent and transfusion-dependent forms of the disease.
Axazeni is an oral, twice-daily pyruvate kinase activator that demonstrated significant clinical benefits, including being the first disease-modifying treatment for non-transfusion-dependent thalassemia and showing durable reductions in transfusion burden for transfusion-dependent patients.
The drug's label includes a Risk Evaluation and Mitigation Strategy (REMS) program with a black box warning for potential hepatocellular injury, requiring liver testing for the first six months of treatment.
Agios expects the REMS program to be fully operational in late January, with initial prescriptions anticipated to be fulfilled in Q1. The initial time from prescription to treatment initiation is estimated at 10-12 weeks.
The initial addressable U.S. patient population is approximately 4,000 adults, with Axazeni priced at approximately $425,000 per patient per year on a WAC basis.

Dec 24, 2025, 1:00 PM

Transcript

Agios Announces Positive Mitapivat Phase III Trial Results for Sickle Cell Disease

AGIO

Product Launch

New Projects/Investments

The RISE UP phase III trial of mitapivat in sickle cell disease achieved statistical significance on the primary endpoint of hemoglobin response, with 40.6% of patients in the mitapivat arm achieving this endpoint.
While mitapivat showed a trend towards reduction in the annualized rate of sickle cell pain crisis, this trend did not achieve statistical significance.
In hemoglobin responders, mitapivat demonstrated clinically meaningful benefits, including a 1.6 grams per deciliter mean increase in hemoglobin, a 26% reduction in vaso-occlusive crises, and a 34% reduction in hospitalizations for sickle cell pain crises.
The safety profile of mitapivat was favorable, with no observed drug-induced hepatocellular injury similar to what was seen in thalassemia trials.
Agios intends to submit a marketing application for mitapivat in the US for sickle cell disease after a pre-SNDA meeting with the FDA in Q1 2026.