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]

Core Insights - Agios Pharmaceuticals reported financial results for Q3 2025, highlighting a focus on key milestones for its drug PYRUKYND in treating thalassemia and sickle cell disease [1][2]. Financial Performance - PYRUKYND generated $12.9 million in net revenues for Q3 2025, a 44% increase from $9.0 million in Q3 2024 and a 3% increase from $12.5 million in Q2 2025 [5][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 was primarily due to a milestone payment and sale of royalty rights recorded in the previous year [7][17]. Product Development and Regulatory Updates - The PDUFA goal date for PYRUKYND's supplemental New Drug Application (sNDA) for thalassemia has been extended to December 7, 2025, due to a request for a Risk Evaluation and Mitigation Strategy (REMS) [6][11]. - The European Medicines Agency's CHMP has adopted a positive opinion for PYRUKYND in thalassemia, with a final decision expected by early 2026 [5][6]. - The RISE UP Phase 3 trial for sickle cell disease is expected to provide topline results by year-end 2025, potentially leading to a U.S. commercial launch in 2026 [5][11]. Research and Development - The Phase 2b trial for tebapivat in lower-risk myelodysplastic syndromes (MDS) has completed enrollment, with topline results anticipated in early 2026 [5][11]. - Research and Development expenses for Q3 2025 were $86.8 million, an increase of $14.3 million compared to Q3 2024, driven by higher clinical trial costs [11][17]. Cash Position - 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 [5][11].

Core Viewpoint - Agios Pharmaceuticals, a partner of Avanzanite, has announced a regulatory update that may impact its operations and market position [1] Group 1: Company Updates - Agios Pharmaceuticals is actively engaging with regulatory bodies to ensure compliance and advance its product pipeline [1] - The regulatory update is expected to provide clarity on the approval process for Agios's key therapies, which could influence future revenue streams [1] Group 2: Industry Implications - The biopharma industry is closely monitoring regulatory changes as they can significantly affect drug development timelines and market entry [1] - Successful navigation of regulatory hurdles is critical for biopharma companies to maintain competitive advantage and investor confidence [1]

Core Viewpoint - Agios Pharmaceuticals has received a positive opinion from the CHMP of the EMA for the new indication of PYRUKYND® (mitapivat) to treat anemia associated with both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia in adults [1][2][3] Company Developments - The CHMP's opinion is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE-T and ENERGIZE Phase 3 trials [2] - Agios has entered into an exclusive agreement with Avanzanite Bioscience B.V. for the commercialization and distribution of PYRUKYND across the European Economic Area, the UK, and Switzerland [3] - PYRUKYND has already received approval in Saudi Arabia for treating adult patients with both types of thalassemia [4] Clinical Trials - The ENERGIZE trial involved 194 non-transfusion-dependent patients, with a primary endpoint of achieving a hemoglobin response defined as an increase of ≥1.0 g/dL [10] - The ENERGIZE-T trial included 258 transfusion-dependent patients, focusing on a transfusion reduction response defined as a ≥50% reduction in transfused RBC units [11] Disease Background - Thalassemia is a rare inherited blood disease affecting hemoglobin production, categorized into alpha-thalassemia and beta-thalassemia [6] - An estimated 18,000 to 23,000 individuals are living with thalassemia in the U.S. and the five largest European countries [8]

CAMBRIDGE, Mass., Oct. 16, 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 the company will host a conference call and live webcast on Thursday, October 30, 2025, at 8:00 a.m. ET to report its third quarter 2025 financial results and business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.co ...

Core Viewpoint - The Schall Law Firm is investigating Agios Pharmaceuticals, Inc. for potential violations of securities laws related to misleading statements and undisclosed information affecting investors [2]. Group 1: Investigation Details - The investigation centers on whether Agios issued false or misleading statements or failed to disclose critical information to investors [2]. - On September 4, 2025, Agios announced that the FDA extended the PDUFA goal date for the supplemental New Drug Application of PYRUKYND® by three months to December 7, 2025 [2]. - Following this announcement, Agios shares dropped by over 11% the next day [2]. Group 2: Legal Representation - The Schall Law Firm is representing investors globally and specializes in securities class action lawsuits and shareholder rights litigation [3]. - Shareholders who suffered losses are encouraged to contact the firm for a free discussion of their rights [3].