AGIOS PHARMACEUTICALS (AGIO) Company Report

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

Agios reports Q3 2025 financial results and provides pipeline updates

AGIO

Earnings

Product Launch

Guidance Update

Agios reported PYRUKYND net revenues of $12.9 million for Q3 2025, a 44% increase compared to $9.0 million in Q3 2024, and maintained a strong financial position with $1.3 billion in cash on hand.
PYRUKYND achieved significant regulatory milestones, including a positive CHMP opinion in Europe for thalassemia and SFDA approval in Saudi Arabia for the same indication, with a new U.S. PDUFA goal date of December 7th for thalassemia.
The company advanced its pipeline with enrollment complete in the tebapivat Phase 2b trial for lower-risk MDS, with top-line data anticipated in early 2026, and PYRUKYND Phase 3 RISE UP trial data for Sickle Cell Disease is on track for late 2025.

Oct 30, 2025, 12:00 PM

Presentation

Agios Pharmaceuticals Reports Q3 2025 Financial Results and Key Pipeline Updates

AGIO

Earnings

Product Launch

New Projects/Investments

Agios Pharmaceuticals reported net revenue of $12.9 million for Q3 2025, marking a 44% increase compared to Q3 2024.
The PDUFA date for PYRUKYND's thalassemia supplemental New Drug Application (sNDA) has been extended to December 7th, following an FDA request for a REMS program.
Top-line results from the RISE UP phase III trial of PYRUKYND in sickle cell disease are anticipated by year-end.
The company ended Q3 2025 with a strong balance sheet, holding approximately $1.3 billion in cash and investments.
Enrollment was completed for the phase II-B trial of tebapivat in lower-risk myelodysplastic syndromes (MDS), with top-line data expected early next year.

Oct 30, 2025, 12:00 PM

Transcript

Agios Pharmaceuticals Reports Q3 2025 Financial Results and Business Updates

AGIO

Earnings

Product Launch

New Projects/Investments

Agios Pharmaceuticals reported $12.9 million in PYRUKYND net revenues for the third quarter ended September 30, 2025, representing a 44% increase from the third quarter of 2024.
The company recorded a net loss of $103.4 million for the third quarter of 2025, compared to a net income of $947.9 million in the third quarter of 2024, which included a milestone payment and the sale of royalty rights.
As of September 30, 2025, Agios held $1.3 billion in cash, cash equivalents, and marketable securities.
Key pipeline developments include a PDUFA goal date of December 7, 2025, for PYRUKYND's U.S. sNDA in thalassemia, and topline results from the RISE UP Phase 3 trial in sickle cell disease expected by year-end 2025.

Oct 30, 2025, 10:32 AM

8-K

Agios Pharmaceuticals Reports Q3 2025 Financial Results and Business Update

AGIO

Earnings

Product Launch

Revenue Acceleration/Inflection

Agios Pharmaceuticals reported $12.9 million in PYRUKYND net revenues for the third quarter of 2025, representing a 44% increase from the third quarter of 2024.
The company posted a net loss of $103.4 million for the third quarter of 2025, compared to a net income of $947.9 million in the third quarter of 2024, which included milestone payments and sale of royalty rights.
As of September 30, 2025, Agios held $1.3 billion in cash, cash equivalents, and marketable securities.
Key upcoming milestones include a PDUFA goal date of December 7, 2025, for PYRUKYND's U.S. sNDA in thalassemia and expected topline results from the RISE UP Phase 3 trial in sickle cell disease by year-end.

Oct 30, 2025, 10:30 AM

Press Release

Earnings Transcripts

Quarterly earnings call transcripts for AGIOS PHARMACEUTICALS.

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