Key Takeaways AGIO reported Q4 loss of $1.85 per share and $20M in revenues, topping estimates.Pyrukynd U.S. sales rose 49% year over year, while Aqvesme had a strong launch in December 2025.AGIO eyes FDA talks in Q1 2026 for mitapivat in SCD after mixed phase III RISE UP data.Agios Pharmaceuticals (AGIO) reported a loss of $1.85 per share in the fourth quarter of 2025, narrower than the Zacks Consensus Estimate of a loss of $1.97. In the year-ago quarter, the company had reported a loss of $1.74 per share. ...

Agios Pharmaceuticals (NasdaqGS:AGIO) Q4 2025 Earnings call February 12, 2026 08:00 AM ET Company ParticipantsBrian Goff - CEOCecilia Jones - CFOMark Breidenbach - Managing DirectorMorgan Sanford - Head of Investor RelationsSarah Gheuens - Chief Medical Officer and Head of Research and DevelopmentTsveta Milanova - Chief Commercial OfficerConference Call ParticipantsAlec Stranahan - Biotechnolology Equity Research AnalystEmily Bodnar - Biotechnolology Equity Research AnalystEric Schmidt - Biotechnology Analy ...

Agios Pharmaceuticals (NasdaqGS:AGIO) Q4 2025 Earnings call February 12, 2026 08:00 AM ET Company ParticipantsBrian Goff - CEOCecilia Jones - CFOMark Breidenbach - Managing DirectorMorgan Sanford - Head of Investor RelationsSarah Gheuens - Chief Medical Officer and Head of Research and DevelopmentTsveta Milanova - Chief Commercial OfficerConference Call ParticipantsAlec Stranahan - Biotechnolology Equity Research AnalystEmily Bodnar - Biotechnolology Equity Research AnalystEric Schmidt - Biotechnology Analy ...

Financial Data and Key Metrics Changes - In Q4 2025, Agios Pharmaceuticals reported worldwide PYRUKYND revenue of $20 million, an increase of 86% compared to Q4 2024, and a sequential increase of 55% from $13 million in Q3 2025 [9][10] - Full-year 2025 revenue for PYRUKYND reached $54 million, reflecting robust year-on-year growth [5][9] - The company ended Q4 2025 with approximately $1.2 billion in cash, cash equivalents, and marketable securities, providing flexibility for future investments [6][10] Business Line Data and Key Metrics Changes - U.S. fourth quarter revenues for PYRUKYND were $16 million, driven by commercial focus in PK deficiency and favorable growth adjustments [9] - Ex-U.S. revenue was $4 million in Q4, primarily reflecting inventory stocking ahead of demand [10] - Cost of sales for Q4 was $1.9 million, R&D expenses were $88.1 million, and SG&A expenses were $51.6 million, roughly flat year-on-year [10] Market Data and Key Metrics Changes - The ACTIVASE launch for thalassemia in the U.S. is underway, with expectations for U.S. PK deficiency revenues in 2026 to be in the range of $45-$50 million [9][10] - The company anticipates a sequential decline in ex-U.S. revenues into Q1 2026 due to inventory adjustments [10] Company Strategy and Development Direction - The company outlined its 2026 strategic priorities, focusing on executing the ACTIVASE launch, expanding the PK activation franchise, advancing early-stage pipeline programs, and maintaining financial discipline [4][5] - Agios aims to leverage its capital-efficient global commercial model to focus investments on the U.S. market, which presents the most significant revenue opportunity [16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the early demand for ACTIVASE, noting strong engagement from physicians and a healthy breadth of early prescribers [14][40] - The company sees a clear path to profitability through its existing commercial presence in thalassemia and PK deficiency, with operating expenses expected to remain flat in 2026 [11][68] Other Important Information - The company received FDA approval for ACTIVASE on December 23, 2025, and the U.S. launch is underway with positive early feedback from physicians [18][19] - Enrollment in the phase II sickle cell disease trial of tebapivat is complete, with top-line results expected in the second half of 2026 [6][20] Q&A Session Summary Question: How does the ACTIVASE launch translate to treatment initiation and revenue recognition? - Management indicated that prescriptions are expected to grow ahead of revenues due to a 10-12 week conversion time from prescription to treatment initiation [29][30] Question: What are the expectations for the phase II data in sickle cell disease? - Management expressed excitement about the trial, noting that it is a dose-finding study aimed at exploring hemoglobin response [34][35] Question: What is the payer mix for ACTIVASE and how does it affect revenue? - The payer mix is similar to PK deficiency, with most patients under commercial payers, and initial market access will occur through medical exceptions [90] Question: What is the rationale for higher dosing in lower-risk MDS compared to sickle cell disease? - Higher doses in MDS are due to faster metabolism in MDS patients, while sickle cell patients metabolize the drug similarly to healthy volunteers [98] Question: What is the expected timeline for profitability? - Management has not provided specific timing but indicated that thalassemia presents a meaningful opportunity for profitability [68][70]

Q4 and FY 2025 Financial Results and Business Highlights Conference call for investors and analysts 12 February 2026 4 2026 strategic priorities – driving long-term value creation | Execute high-impact launch for AQVESME | | --- | | (mitapivat) in thalassemia | | Potential to expand PK activation franchise into | | sickle cell disease and LR-MDS | | Unlock future value in hematology and other rare | | disease by advancing early-stage pipeline | | Ensure long-term sustainability through disciplined | | capit ...

Core Insights - Agios Pharmaceuticals reported strong execution in 2025, highlighted by the U.S. approval and launch of AQVESME, the first treatment for anemia in adults with thalassemia, which has received positive feedback from the community [2][5] - The company aims to expand its product portfolio and pipeline, focusing on high-value indications such as sickle cell disease and myelodysplastic syndromes, while maintaining disciplined capital allocation [2] Financial Performance - For Q4 2025, Agios reported net revenues of $20.0 million from PYRUKYND, with a full-year total of $54.0 million, marking a 49% increase from Q4 2024 [5][12] - The net loss for Q4 2025 was $108.0 million, compared to a net loss of $96.5 million in Q4 2024 [8] - Total revenues for the year 2025 reached $54.0 million, up from $36.5 million in 2024 [15] Research and Development Highlights - AQVESME received FDA approval in December 2025 and is now available in the U.S. [6][12] - A pre-sNDA meeting with the FDA for mitapivat in sickle cell disease is scheduled for Q1 2026, with plans to submit a marketing application thereafter [5][6] - The Phase 2 trial of tebapivat in sickle cell disease has been fully enrolled, with topline results expected in the second half of 2026 [7] Cash Position - As of December 31, 2025, Agios had $1.2 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion a year earlier [12][14] - The company anticipates that its financial resources will support the U.S. launch of AQVESME and the advancement of its clinical programs [12]

Core Thesis - Agios Pharmaceuticals, Inc. is positioned as a compelling investment opportunity due to its innovative therapies and strong financial standing, particularly with the recent FDA approval of its lead product, mitapivat, for thalassemia [1][4][5] Company Overview - Agios Pharmaceuticals is a clinical-stage biotech company focused on cellular metabolism through oral, small-molecule allosteric activators of the pyruvate kinase (PK) enzyme [2] - The lead product, mitapivat, is marketed as PYRUKYND for PK deficiency and received FDA approval in December 2025 under the brand name AQVESME for alpha- and beta-thalassemia [2] Clinical Trials and Efficacy - Phase 3 trials in thalassemia demonstrated strong efficacy, with ENERGIZE showing a 42.3% hemoglobin response in non-transfusion-dependent patients and ENERGIZE-T achieving a 30.4% transfusion reduction in dependent patients [3] - In sickle cell disease, the RISE UP trial met hemoglobin endpoints but showed only favorable trends in pain crisis reduction [3] - Agios is advancing tebapivat, which achieved proof-of-concept in lower-risk myelodysplastic syndromes, with 40% of patients reaching transfusion independence [3] Financial Position - As of Q3 2025, Agios holds approximately $1.3 billion in cash and marketable securities, sufficient to fund launches and clinical programs without dilutive financing [4] - PYRUKYND revenues grew 44% year-over-year, although net losses persist due to high R&D and SG&A expenses [4] Market Position and Competitive Advantage - The thalassemia approval provides a first-mover advantage in a larger patient population, while the upcoming sickle cell opportunity could further expand its market [4] - Agios differentiates from competitors like Reblozyl and gene therapies by offering a twice-daily pill with broad accessibility [4] Future Catalysts - Led by CEO Brian Goff, the company is positioned to scale globally, with upcoming catalysts in early 2026 including the AQVESME U.S. launch, FDA discussions for sickle cell indications, and Phase 2b tebapivat data [5] - Multiple approved indications and a strong balance sheet enhance Agios's potential for successful commercial execution and global expansion [5]

Core Insights - Agios Pharmaceuticals has outlined its strategic priorities and key milestones for 2026, focusing on the launch of AQVESME™ and expansion into additional indications for its PK activation franchise [1][3] Company Performance - In 2025, Agios achieved significant progress, highlighted by the U.S. approval of AQVESME™, the first medicine approved for treating anemia in adults with alpha- or beta-thalassemia [2][9] - The company aims to become a sustainable and diversified rare disease company, leveraging its existing commercial presence [2][3] 2026 Strategic Priorities - The company plans a high-impact U.S. launch of AQVESME in thalassemia and seeks to expand its PK activation franchise into sickle cell disease and lower-risk myelodysplastic syndromes [3][8] - Agios is committed to disciplined capital allocation and operational efficiency to support long-term sustainability [3] Anticipated Milestones - AQVESME's U.S. commercial launch is set for late January 2026, following FDA approval in December 2025 [9] - A pre-sNDA meeting with the FDA for mitapivat in sickle cell disease is anticipated in Q1 2026, with a regulatory submission to follow [8][9] - Topline results from the Phase 2b trial of tebapivat in lower-risk myelodysplastic syndromes are expected in the first half of 2026 [6][8] - Enrollment in the Phase 2 sickle cell disease trial of tebapivat was initiated in 2025, with topline results expected in the second half of 2026 [9] Pipeline Development - Agios is advancing its early- and mid-stage pipeline across multiple high-value indications, including a Phase 1 trial of AG-236 for polycythemia vera and a Phase 1b trial of AG-181 for phenylketonuria [10][11]

RISE UP Phase 3 Trial Results - Mitapivat met the primary endpoint with a 40.6% hemoglobin response rate, defined as ≥1.0 g/dL increase in average Hb from Week 24 through Week 52 compared to baseline, significantly higher than the 2.9% in the placebo arm[38] - There was a trend favoring mitapivat with a 14% reduction in the annualized rate of sickle cell pain crises (SCPCs) compared to placebo, although this did not reach statistical significance[39] - Mitapivat showed a statistically significant improvement in average change from baseline in hemoglobin concentration, with a 7.43 g/L difference compared to placebo[41] - Mitapivat significantly reduced the average change from baseline in indirect bilirubin compared to placebo, with a difference of -16.91 µmol/L[46] - The trial showed a trend in favor of mitapivat with a 14% reduction in the annualized rate of hospitalizations for SCPC compared to placebo[50] - Percent reticulocytes substantially decreased from baseline in the mitapivat arm vs placebo[52] Safety and Discontinuation - The safety profile observed in the RISE UP Phase 3 trial was consistent with previous mitapivat sickle cell disease trials, with no new safety signals identified[57] - The discontinuation rate in the double-blind treatment period was low, with 13% in the mitapivat arm and 18.8% in the placebo arm[34] Next Steps - The company 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[61, 68]

Core Insights - Agios Pharmaceuticals reported a narrower loss of $1.78 per share in Q3 2025, compared to the Zacks Consensus Estimate of a loss of $1.93, and a significant decline from earnings of $16.22 per share in the same quarter last year [1][6] - Total revenues for the third quarter reached $12.9 million, exceeding the Zacks Consensus Estimate of $10 million, and reflecting a 44% year-over-year increase [2][6] - The company's stock rose by 2.9% following the earnings report, and has increased by 31.5% year-to-date, outperforming the industry average increase of 10.6% [2] Financial Performance - Revenues were solely derived from product sales of Pyrukynd, Agios' only marketed drug, which is approved for treating hemolytic anemia in adults with pyruvate kinase deficiency [3] - Pyrukynd's sales increased by 3% sequentially, with 149 patients currently on the therapy in the U.S., marking a 5% increase from Q2 2025 [3] - Research and development expenses rose by approximately 19.7% year-over-year to $86.8 million, attributed to higher costs related to studies on the PK activation franchise [4] - Selling, general, and administrative expenses increased by 7.3% year-over-year to $41.3 million, driven by preparations for a potential approval and launch of Pyrukynd for thalassemia [7] Pipeline Developments - The FDA is expected to make a decision on Pyrukynd's thalassemia indication by December 7, 2025, following a positive opinion from the EMA [6][11] - The FDA extended the review timeline for Pyrukynd's supplemental new drug application by three months due to a request for a Risk Evaluation and Mitigation Strategy [9][10] - Agios has completed enrollment in the phase III RISE UP study for mitapivat in treating sickle cell disease, with top-line data expected by the end of 2025 [12] - The company is also developing tebapivat for myelodysplastic syndromes, with patient enrollment in a phase IIb study completed and top-line data expected in early 2026 [13]