AGIOS PHARMACEUTICALS (AGIO) Company Report

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.

4 hours ago

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.

5 hours ago

Transcript

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.

6 hours ago

Transcript

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.

6 hours ago

Presentation

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.

Nov 19, 2025, 1:00 PM

Transcript

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

AGIO

Product Launch

Agios Pharmaceuticals' mitapivat achieved statistical significance on the primary endpoint of hemoglobin response in the RISE UP phase III trial for sickle cell disease, with 40.6% of patients in the mitapivat arm showing at least a 1 gram per deciliter increase in average hemoglobin concentration.
The other primary endpoint, annualized rate of sickle cell pain crises, showed a 14% reduction for mitapivat versus placebo but did not achieve statistical significance in the total trial population (p-value 0.12).
In the 40.6% of patients who were hemoglobin responders, mitapivat demonstrated clinically meaningful benefits, including a 1.6 grams per deciliter mean increase in hemoglobin, a 26% reduction in annualized sickle cell pain crises, and a 34% reduction in annualized hospitalizations for pain crises.
The trial supported mitapivat's favorable safety profile, with adverse events generally balanced across treatment arms and no drug-induced liver injury observed similar to prior thalassemia trials.
Agios Pharmaceuticals plans to submit a marketing application for mitapivat in the U.S. for sickle cell disease after a pre-SNDA meeting with the FDA in Q1 2026.

Nov 19, 2025, 1:00 PM

Transcript

AGIO Reports Positive Phase 3 RISE UP Trial Results for Mitapivat in Sickle Cell Disease

AGIO

Product Launch

Guidance Update

New Projects/Investments

AGIO's mitapivat met its primary endpoint of hemoglobin (Hb) response in the Phase 3 RISE UP trial, with 40.6% of patients in the mitapivat arm achieving a response compared to 2.9% in the placebo arm.
The trial demonstrated statistically significant improvements in key secondary endpoints, including Hb concentration and indirect bilirubin, indicating a strong anti-hemolytic profile.
While there was a 14% reduction in the annualized rate of sickle cell pain crises (SCPCs) favoring mitapivat, this primary endpoint did not achieve statistical significance.
Mitapivat showed a favorable safety profile consistent with prior trials, with a low discontinuation rate of 13.0% in the mitapivat arm during the double-blind period.
AGIO intends to submit a marketing application for mitapivat in the U.S. for sickle cell disease following a pre-sNDA meeting with the FDA in Q1 2026.

Nov 19, 2025, 1:00 PM

Presentation

Agios Pharmaceuticals Reports Positive Phase III RISE UP Trial Results for Mitapivat in Sickle Cell Disease

AGIO

Product Launch

New Projects/Investments

Agios Pharmaceuticals' mitapivat Phase III RISE UP trial for sickle cell disease achieved statistical significance on its primary endpoint of hemoglobin response, with 40.6% of patients in the mitapivat arm showing a significant increase in hemoglobin.
The second primary endpoint, annualized rate of sickle cell pain crises, showed a 14% reduction in observed crises for mitapivat compared to placebo, but did not achieve statistical significance in the total trial population.
In the 40.6% of patients who were hemoglobin responders, mitapivat demonstrated clinically meaningful benefits, including a 1.6 grams per deciliter mean increase in hemoglobin, a 26% reduction in sickle cell pain crises, and a 34% reduction in hospitalizations for sickle cell pain crises.
The trial showed a favorable safety profile for mitapivat, with adverse events generally balanced across treatment arms and no similar cases of drug-induced hepatocellular injury as observed in thalassemia trials.
Agios intends to submit a marketing application for mitapivat in the U.S. for sickle cell disease after a pre-SNDA meeting with the FDA in Q1 2026.

Nov 19, 2025, 1:00 PM

Transcript

Agios Pharmaceuticals Reports Q3 2025 Financial Results and Provides Pipeline Updates

AGIO

Earnings

Guidance Update

Product Launch

Agios Pharmaceuticals reported $12.9 million in net PYRUKYND revenue for Q3 2025, representing a 44% increase compared to Q3 2024 and a 3% sequential increase from Q2 2025.
The company maintains a strong financial position with approximately $1.3 billion in cash, cash equivalents, and marketable securities at the end of Q3 2025.
Key regulatory and clinical milestones include the extended PDUFA date for PYRUKYND in thalassemia to December 7 and the anticipated top-line results from the RISE UP Phase 3 trial of PYRUKYND in sickle cell disease by year end.
Internationally, PYRUKYND received approval for adults with thalassemia in Saudi Arabia in August and a positive CHMP opinion for marketing authorization in Europe. Enrollment was also completed for the Phase 2b trial of tebapivat in lower-risk MDS, with top-line data expected early next year.

Oct 30, 2025, 12:00 PM

Transcript

Agios Pharmaceuticals Reports Q3 2025 Revenue and Provides PYRUKYND Updates

AGIO

Earnings

Guidance Update

Product Launch

Agios Pharmaceuticals reported $12.9 million in net revenue for PYRUKYND in Q3 2025, marking a 44% increase compared to Q3 2024 and a 3% sequential increase from Q2 2025.
The PDUFA date for PYRUKYND in thalassemia has been extended to December 7 , and top-line results from the Phase 3 RISE UP trial for PYRUKYND in sickle cell disease are expected by year-end.
The company ended Q3 2025 with a strong balance sheet, holding approximately $1.3 billion in cash and investments.
Enrollment for the Phase 2b trial of tebapivat in lower-risk myelodysplastic syndromes (MDS) was completed, with top-line data anticipated early next year.

Oct 30, 2025, 12:00 PM

Transcript

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