Core Insights - Agios Pharmaceuticals announced topline results from the RISE UP Phase 3 trial of mitapivat, an oral pyruvate kinase activator, for patients with sickle cell disease, demonstrating significant improvements in hemoglobin response and hemolysis markers [1][5][6] Trial Results - The trial met its primary endpoint of hemoglobin response, with 40.6% of patients in the mitapivat arm achieving a significant increase in hemoglobin levels compared to 2.9% in the placebo group [12] - Mitapivat showed a statistically significant improvement in average hemoglobin concentration and indirect bilirubin levels, but the reduction in annualized rate of sickle cell pain crises did not achieve statistical significance [2][3][12] - The safety profile of mitapivat was consistent with previous trials, with a similar proportion of adverse events reported in both mitapivat and placebo groups [19] Patient Outcomes - Patients who achieved hemoglobin response experienced clinically meaningful benefits in the annualized rate of sickle cell pain crises and hospitalizations, as well as improvements in fatigue scores [4][12][13] - The average change in PROMIS Fatigue score was -5.19 for hemoglobin responders, indicating a clinically meaningful improvement [13] Regulatory and Future Plans - Agios plans to engage with the FDA to discuss the findings and intends to submit a marketing application for mitapivat for sickle cell disease in early 2026 [15][16] - The company is also focused on the potential U.S. approval of PYRUKYND for thalassemia, anticipated in December 2025, and will take steps to reduce operating expenses [16] About Sickle Cell Disease - Sickle cell disease is a rare inherited blood disorder characterized by abnormal hemoglobin production, leading to severe complications and increased healthcare needs [18]

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Core Insights - Agios Pharmaceuticals reported a GAAP EPS of -$1.78 for Q3 2025, surpassing estimates by $0.12 [1] - The company achieved revenue of $12.88 million, reflecting a year-over-year increase of 43.8% [1] Company Overview - Agios Pharmaceuticals is focused on drug development, leveraging scientific expertise in cell biology and therapeutic research [1] - The company aims to innovate through unique mechanisms of action and first-in-class therapies, targeting the biotechnology sector [1] Financial Performance - The reported EPS of -$1.78 indicates a loss, but it is an improvement over analyst expectations [1] - Revenue growth of 43.8% year-over-year highlights the company's strong performance in the market [1]

CAMBRIDGE, Mass., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that new data on mitapivat, an oral pyruvate kinase (PK) activator, will be featured in oral and poster presentations during the 67th American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2025) in Orlando, Florida, December 6-9, 2025. “This year’s presentations ...

Key Takeaways Agios reported a Q3 loss of $1.78 per share, beating expectations of a loss of $1.93.AGIO's total revenues climbed 44% year over year to $12.9 million, fueled by Pyrukynd sales.The FDA decision on Pyrukynd's thalassemia indication is due on Dec. 7, as the EMA gives a positive opinion.Agios Pharmaceuticals (AGIO) incurred a loss of $1.78 per share in the third quarter of 2025, narrower than the Zacks Consensus Estimate of a loss of $1.93. In the year-ago quarter, the company had reported earnin ...

Financial Performance - The net loss for the nine months ended September 30, 2025, was $304.7 million, compared to a net income of $770.2 million for the same period in 2024, primarily due to the sale of Vorasidenib Royalty Rights[100] - As of September 30, 2025, the accumulated deficit stood at $453.7 million[100] - The net loss for the three months ended September 30, 2025, was $103.4 million, compared to a net income of $947.9 million for the same period in 2024, primarily due to the sale of Vorasidenib Royalty Rights in 2024[138] - Total revenue for the nine months ended September 30, 2025, was $34.06 million, an increase of $8.3 million compared to the same period in 2024[130] - Total product revenue for the three months ended September 30, 2025, was $12.88 million, an increase of $3.9 million compared to the same period in 2024[129] - Interest income for the nine months ended September 30, 2025, was $44.0 million, compared to $30.1 million for the same period in 2024, indicating an increase in returns on investments[137] Research and Development - The company expects to continue incurring significant expenses as it advances clinical development and commercialization activities for PYRUKYND® and other products[100] - Research and development expenses are expected to increase as product candidate development programs progress, with costs primarily incurred for drug discovery and clinical trials[106] - Research and development expenses for the nine months ended September 30, 2025, rose by $33.0 million to $251.5 million compared to $218.5 million for the same period in 2024, driven by increased costs associated with clinical trials[135] - Total research and development expenses for the three months ended September 30, 2025, were $86.8 million, up from $72.5 million in 2024, reflecting a $10.8 million increase in direct expenses[134] Product Development and Regulatory Milestones - PYRUKYND® received FDA approval on February 17, 2022, and began generating product revenue from sales shortly thereafter[101] - The company achieved a regulatory milestone that triggered a $10.0 million payment to Alnylam in the nine months ended September 30, 2025[93] - In August 2024, the FDA approved vorasidenib for adult and pediatric patients, leading to a recognized income of $200.0 million from the Vorasidenib Milestone Payment[96] - The European Commission is expected to make a final decision on the new indication for PYRUKYND® by early 2026 following a positive opinion from the CHMP[111] - The company has submitted regulatory applications for PYRUKYND® in multiple jurisdictions, including the FDA and EMA, highlighting hepatocellular injury as a potential risk[116] Clinical Trials and Results - In the ENERGIZE trial, 42.3% of patients treated with PYRUKYND® achieved a hemoglobin response compared to 1.6% in the placebo group, demonstrating statistical significance (p<0.0001)[113] - The ENERGIZE-T trial showed that 30.4% of patients on PYRUKYND® achieved a transfusion reduction response compared to 12.6% in the placebo group (p=0.0003)[114] - The ENERGIZE trial enrolled 194 patients, with 93.8% in the PYRUKYND® arm completing the study[113] - In the phase 2 portion of the RISE UP study, 46.2% of patients in the 50 mg twice daily mitapivat arm and 50.0% in the 100 mg twice daily mitapivat arm achieved a hemoglobin response, compared to 3.7% in the placebo arm[117] - The average change from baseline in hemoglobin levels from week 10 to week 12 was 1.11 g/dL for the 50 mg arm and 1.13 g/dL for the 100 mg arm, while the placebo arm showed a change of 0.05 g/dL[117] - In the ACTIVATE-kidsT study, 28.1% of patients in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 11.8% in the placebo arm[120] - In the ACTIVATE-kids study, 31.6% of patients in the mitapivat arm achieved a hemoglobin response, while 0% in the placebo arm achieved this[121] - The company expects to announce topline data for the phase 3 portion of the RISE UP trial in late 2025, with a potential U.S. commercial launch in 2026 if approved[118] Cash Flow and Financial Position - Cash used in operating activities for the nine months ended September 30, 2025, was $276.8 million, compared to $256.7 million for the same period in 2024, largely due to increased operating expenses[145] - The company reported cash, cash equivalents, and marketable securities of $1.3 billion as of September 30, 2025[144] - Existing cash, cash equivalents, and marketable securities as of September 30, 2025, are projected to support potential commercial launches of PYRUKYND® in thalassemia and SCD[151] - Future capital requirements will depend on revenue from commercial sales of PYRUKYND® and costs associated with ongoing commercialization activities[152] - The company plans to finance cash needs primarily through cash on hand, potential royalty payments, and future sales of PYRUKYND®[153] - The company may need to relinquish rights to technologies or revenue streams if it raises funds through collaborations or licensing arrangements[154] - As of September 30, 2025, the company had cash, cash equivalents, and marketable securities of $1.3 billion, down from $1.5 billion as of December 31, 2024[156] Market Risks - The company is exposed to market risk related to interest rate changes, particularly due to investments in short-term marketable securities[156] - The company is also exposed to foreign currency exchange rate fluctuations due to contracts with CROs and manufacturing organizations in Asia and Europe[157] Commercialization Efforts - A distribution agreement was established with NewBridge Pharmaceuticals for PYRUKYND® in the Gulf Cooperation Council region[102] - The company entered into a distribution agreement with Avanzanite Bioscience for commercialization of PYRUKYND® in the European Economic Area, Switzerland, and the U.K.[102] - The company is exploring potential partnership opportunities for the commercialization of PYRUKYND® outside the United States[112] - The company anticipates an increase in selling, general and administrative expenses to support ongoing commercialization activities related to PYRUKYND®[127] - The increase in selling, general, and administrative expenses for the three months ended September 30, 2025, was $2.7 million, driven by preparations for the potential approval of PYRUKYND®[131]

Financial Data and Key Metrics Changes - In Q3 2025, Agios Pharmaceuticals reported net revenue of $12.9 million, a 44% increase compared to $9 million in Q3 2024, and a 3% increase from $12.5 million in Q2 2025 [8][6][9] - The cost of sales for the quarter was $1.7 million, while R&D expenses rose to $86.8 million, an increase of $14.3 million compared to the previous year [9][10] - SG&A expenses were $41.3 million, up $2.7 million year-over-year, reflecting disciplined investment ahead of potential product launches [9] Business Line Data and Key Metrics Changes - PYRUKYND net revenue was $12.9 million, reflecting strong commercial execution in PK deficiency ahead of potential U.S. approval for thalassemia [8][12] - 262 patients completed prescription enrollment forms, with 149 currently on therapy, marking a 5% increase from the second quarter [12][13] Market Data and Key Metrics Changes - Agios received approval for PYRUKYND in adults with thalassemia in Saudi Arabia, marking its first global regulatory approval for this indication [6][18] - A positive CHMP opinion was received for PYRUKYND in Europe, with a regulatory decision anticipated in early 2026 [18][19] Company Strategy and Development Direction - The company is focused on unlocking long-term shareholder value through multiple high-value catalysts, including the upcoming PDUFA date for PYRUKYND in thalassemia [5][6] - Agios is pursuing a capital-efficient global commercialization strategy through partnerships, retaining full rights to PYRUKYND while minimizing capital investment for U.S. launches [10][13] - The company aims to expand and diversify its pipeline through both internal efforts and external assets [10][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ability to deliver a successful launch for PYRUKYND in thalassemia, emphasizing the critical need for treatment innovation in the community [7][16] - The company anticipates continued revenue growth in 2025, driven by strong execution of its sales force [9][10] Other Important Information - Agios has approximately $1.3 billion in cash and investments, positioning it to invest in potential U.S. launches and advance its rare disease pipeline [6][10] - The company completed enrollment in the Phase 2b trial of tebapivat for lower-risk myelodysplastic syndromes, with top-line data expected early next year [9][24] Q&A Session Summary Question: What type of REMS program is the FDA interested in for thalassemia? - Management indicated that the REMS program will include monitoring and some form of education, but specific details cannot be disclosed at this time [33] Question: What type of business development makes sense for the company at this stage? - The company is looking for transformative therapies for rare diseases and is not tied to any specific timeline for transactions [34] Question: What are the liver monitoring requirements in Saudi Arabia and Europe? - In Saudi Arabia, monitoring is required once a month for the first six months, while the European label is still pending [41] Question: How is the commercial approach changing due to monitoring requirements? - Management does not anticipate REMS to be a barrier to prescribing, as both academic and community practices have experience with such programs [43] Question: What is the strategy for the European thalassemia launch with Avanzanite? - The pricing and reimbursement process in Europe can take 12 to 18 months post-approval, and the company is working closely with Avanzanite to refine its strategy [51]

Core Insights - Agios Pharmaceuticals reported a quarterly loss of $1.78 per share, better than the Zacks Consensus Estimate of a loss of $1.93, representing an earnings surprise of +7.77% [1] - The company generated revenues of $12.88 million for the quarter ended September 2025, exceeding the Zacks Consensus Estimate by 27.69% and up from $8.96 million a year ago [2] - Agios Pharmaceuticals shares have increased by approximately 27.7% year-to-date, outperforming the S&P 500's gain of 17.2% [3] Financial Performance - Over the last four quarters, Agios Pharmaceuticals has surpassed consensus EPS estimates two times and topped consensus revenue estimates three times [2] - The current consensus EPS estimate for the upcoming quarter is -$2.04 on revenues of $10.91 million, and for the current fiscal year, it is -$7.47 on revenues of $42.13 million [7] Industry Context - The Medical - Biomedical and Genetics industry, to which Agios Pharmaceuticals belongs, is currently ranked in the top 38% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact investor sentiment [5]

Financial Performance - Q3 2025 net revenues reached $12.9 million, a 44% increase compared to $9.0 million in Q3 2024 [12] - Cash on hand remains strong at $1.3 billion, intended for investment in U S launches and pipeline development [10, 12, 15] - Net loss for Q3 2025 was $103.4 million, compared to a net income of $947.9 million in Q3 2024 [15] PYRUKYND® Commercial Progress - PYRUKYND net sales were $12.9 million in Q3 2025, compared to $12.5 million in Q2 2025 and $9.0 million in Q3 2024 [15, 20] - In the U S, 262 unique PK deficiency patients have completed prescription enrollment forms since launch [20] - There are 149 net patients on PYRUKYND treatment in the U S, including new prescriptions and treatment continuations [20] - PYRUKYND has been prescribed by 227 unique prescribers in the U S [20] Regulatory and Pipeline Advancements - PYRUKYND received SFDA approval in Saudi Arabia for thalassemia [12, 27, 28] - A positive CHMP opinion in Europe was received for PYRUKYND in thalassemia [12, 27, 28] - Enrollment is complete in the tebapivat Phase 2b trial for lower-risk MDS, with top-line data anticipated in early 2026 [12, 27]

Financial Performance - PYRUKYND net revenues for Q3 2025 were $12.9 million, a 44% increase from $9.0 million in Q3 2024 and a 3% increase from $12.5 million in Q2 2025[6] - The company reported a net loss of $103.4 million for Q3 2025, compared to a net income of $947.9 million in Q3 2024, which included milestone payments and royalty sales[9] - As of September 30, 2025, Agios had $1.3 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion at the end of 2024[12] Drug Development and Trials - The PDUFA goal date for PYRUKYND's supplemental New Drug Application in thalassemia is set for December 7, 2025[5] - Enrollment in the Phase 2b tebapivat trial for lower-risk MDS is fully completed, with topline results expected in early 2026[5] - The RISE UP Phase 3 trial topline results in sickle cell disease are expected by year-end 2025, potentially leading to a U.S. commercial launch in 2026[5] Patient Enrollment and Therapy - 262 unique patients completed prescription enrollment forms for PYRUKYND, representing a 6% increase over Q2 2025[6] - 149 patients are currently on therapy in the U.S., reflecting a 5% increase from Q2 2025[6] Expenses - Research and Development expenses for Q3 2025 were $86.8 million, an increase of $14.3 million compared to Q3 2024[12] - Selling, General and Administrative expenses for Q3 2025 were $41.3 million, up $2.7 million from Q3 2024, driven by preparations for the potential U.S. launch of PYRUKYND[12]