[Business and Operational Highlights](index=1&type=section&id=Business%20and%20Operational%20Highlights) Agios reported strong PYRUKYND® commercial performance, advanced its R&D pipeline, and prepared for potential U.S. approval for thalassemia - The company is focused on the upcoming PDUFA goal date of September 7, 2025, for the potential U.S. approval of PYRUKYND for thalassemia[3](index=3&type=chunk) - Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease are expected by the end of the year, with a potential U.S. commercial launch in 2026[3](index=3&type=chunk)[5](index=5&type=chunk)[10](index=10&type=chunk) - Entered into a distribution agreement with Avanzanite Bioscience B.V. to commercialize PYRUKYND in the European Economic Area, the UK, and Switzerland[6](index=6&type=chunk) [Commercial Performance – PYRUKYND® (mitapivat)](index=1&type=section&id=Commercial%20Performance%20%E2%80%93%20PYRUKYND%C2%AE%20%28mitapivat%29) PYRUKYND® net revenue reached **$12.5 million** in Q2 2025, driven by steady growth in patient enrollment and therapy PYRUKYND® Q2 2025 Performance | Metric | Q2 2025 | Q2 2024 | QoQ Growth (vs Q1 2025) | | :--- | :--- | :--- | :--- | | Net Revenue | $12.5 million | $8.6 million | N/A | | Unique Patient Enrollments | 248 | N/A | 6% | | Patients on Therapy | 142 | N/A | 4% | [R&D Highlights](index=2&type=section&id=R%26D%20Highlights) R&D pipeline advanced with PYRUKYND's sNDA for thalassemia, progress in sickle cell and LR-MDS trials, and AG-236 IND clearance - **PYRUKYND (mitapivat):** - **Thalassemia:** sNDA under active FDA review with a PDUFA goal date of September 7, 2025 - **Sickle Cell Disease:** Topline Phase 3 (RISE UP) results on track for year-end 2025[10](index=10&type=chunk) - **Tebapivat:** - **Sickle Cell Disease:** Dosed the first patient in a Phase 2 trial - **Lower-risk Myelodysplastic Syndromes (LR-MDS):** Phase 2b trial enrollment is progressing, with completion targeted by the end of 2025[8](index=8&type=chunk)[10](index=10&type=chunk) - **Early Pipeline:** - Received Investigational New Drug (IND) clearance for AG-236, an siRNA targeting TMPRSS6 for treating polycythemia vera (PV)[10](index=10&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) Agios reported a **net loss of $112.0 million** in Q2 2025, driven by increased R&D and SG&A expenses, with **$1.3 billion** in cash Q2 2025 Key Financial Metrics (in millions, vs. Q2 2024) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Net Product Revenue | $12.5 million | $8.6 million | | R&D Expenses | $91.9 million | $77.4 million | | SG&A Expenses | $45.9 million | $35.5 million | | Net Loss | $112.0 million | $96.1 million | - The increase in R&D expenses was primarily due to a **$10.0 million** regulatory milestone payment to Alnylam for AG-236[13](index=13&type=chunk) - The increase in SG&A expenses was mainly attributable to increased commercial activities in preparation for the potential approval of PYRUKYND in thalassemia[13](index=13&type=chunk) - The company ended the quarter with **$1.3 billion** in cash, cash equivalents, and marketable securities, expected to fund operations, potential launches, and pipeline expansion[5](index=5&type=chunk)[13](index=13&type=chunk) [Financial Statements](index=5&type=section&id=Financial%20Statements) Consolidated financial statements show **$1.47 billion** in total assets and a **net loss of $112.0 million** for Q2 2025, or **($1.93)** per share [Consolidated Balance Sheet Data](index=5&type=section&id=Consolidated%20Balance%20Sheet%20Data) As of June 30, 2025, Agios reported **$1.34 billion** in cash, **$1.47 billion** in total assets, and **$1.37 billion** in stockholders' equity Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash, cash equivalents, and marketable securities | $1,339,404 | $1,532,031 | | Total assets | $1,471,237 | $1,663,199 | | Stockholders' equity | $1,369,555 | $1,540,956 | [Consolidated Statements of Operations Data](index=5&type=section&id=Consolidated%20Statements%20of%20Operations%20Data) For Q2 2025, net product revenue was **$12.5 million**, with loss from operations at **$127.1 million** and a **net loss of $112.0 million**, or **($1.93)** per share Statement of Operations Summary (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Product revenue, net | $12,455 | $8,615 | | Total operating expenses | $139,511 | $114,432 | | Loss from operations | $(127,056) | $(105,817) | | Net loss | $(112,020) | $(96,118) | | Net loss per share | $(1.93) | $(1.69) |

Core Viewpoint - Agios Pharmaceuticals reported its financial results for Q2 2025, highlighting progress in its drug pipeline, particularly with PYRUKYND for thalassemia and ongoing trials for sickle cell disease [1][2]. Financial Performance - The company generated $12.5 million in net revenue for Q2 2025, an increase from $8.6 million in Q2 2024 [6][11]. - The net loss for Q2 2025 was $112.0 million, compared to a net loss of $96.1 million in Q2 2024 [7][17]. - As of June 30, 2025, Agios had $1.3 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion at the end of 2024 [11][14]. Product Development and Pipeline - PYRUKYND's supplemental New Drug Application (sNDA) for thalassemia is under active review by the FDA, with a PDUFA goal date of September 7, 2025 [5][6]. - The topline results from the RISE UP Phase 3 trial for PYRUKYND in sickle cell disease are expected by year-end 2025, with a potential U.S. commercial launch in 2026 [5][6]. - The first patient has been dosed in the Phase 2 trial for tebapivat in sickle cell disease, which is currently enrolling patients [11]. Operational Highlights - There was a 6% increase in unique patients completing prescription enrollment forms compared to Q1 2025, totaling 248 patients [6]. - The number of patients on PYRUKYND therapy increased by 4% over Q1 2025, reaching 142 patients [6]. - Agios entered into a distribution agreement with Avanzanite Bioscience B.V. to commercialize PYRUKYND across the European Economic Area, the UK, and Switzerland [6]. Research and Development Expenses - R&D expenses for Q2 2025 were $91.9 million, up from $77.4 million in Q2 2024, primarily due to a $10 million regulatory milestone payment [11][17]. - Selling, General and Administrative (SG&A) expenses increased to $45.9 million in Q2 2025 from $35.5 million in Q2 2024, attributed to increased commercial activities [11][17]. Strategic Vision - Agios aims to redefine the future of rare disease treatment by developing innovative medicines that can transform patients' lives [9].

Core Insights - Agios Pharmaceuticals will host a conference call and live webcast on July 31, 2025, at 8:00 a.m. ET to report its Q2 2025 financial results and business highlights [1] - The live webcast will be accessible on the company's website, with a replay available approximately two hours after the event [2] Company Overview - Agios Pharmaceuticals is a leader in pyruvate kinase (PK) activation, focusing on developing therapies for rare diseases [3] - The company markets a first-in-class PK activator for adults with PK deficiency, which is the first disease-modifying therapy for this condition [3] - Agios is advancing a clinical pipeline that includes investigational medicines for alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndrome (MDS)-associated anemia, and phenylketonuria (PKU) [3] - Additionally, Agios is developing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera [3]

Core Insights - Agios Pharmaceuticals has appointed Dr. Jay Backstrom to its Board of Directors, which is expected to enhance the company's strategic direction and clinical program advancements [1][2][3] Company Overview - Agios Pharmaceuticals is a leader in cellular metabolism and pyruvate kinase (PK) activation, focusing on therapies for rare diseases [1][4] - The company markets a first-in-class PK activator for adults with PK deficiency, representing the first disease-modifying therapy for this condition [4] Leadership and Expertise - Dr. Backstrom brings extensive experience from his previous roles, including President and CEO of Scholar Rock and Executive Vice President of Research and Development at Acceleron Pharma [2][3] - His leadership has been instrumental in advancing clinical programs to regulatory approval, particularly in rare diseases [2][3] Strategic Focus - Agios is focused on maximizing the potential of its PK activator, PYRUKYND (mitapivat), and advancing its pipeline of rare disease medicines [2][3] - The company is developing investigational medicines for conditions such as alpha- and beta-thalassemia, sickle cell disease, and myelodysplastic syndromes [4]

Company Overview - Agios Pharmaceuticals, Inc. is a leader in cellular metabolism and pyruvate kinase (PK) activation, focusing on therapies for rare diseases [3] - The company markets a first-in-class PK activator for adults with PK deficiency, representing the first disease-modifying therapy for this rare condition [3] - Agios is advancing a clinical pipeline that includes investigational medicines for alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia, and phenylketonuria (PKU) [3] - Additionally, Agios is developing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera [3] Upcoming Events - The management team of Agios is scheduled to present at the Goldman Sachs 46th Annual Global Healthcare Conference on June 9, 2025, at 8:40 a.m. ET [1] - A live webcast of the presentation will be available on the company's website, with a replay archived for at least two weeks [2]

Agios Pharmaceuticals (AGIO) 2025 Conference May 21, 2025 08:00 AM ET Speaker0 Great. Good morning, everyone. We're going to get started, and welcome back to day two of the twenty twenty five RBC Global Healthcare Conference. My name is Greg Renzo, of the biotech analysts, and we're pleased to be joined today by Agios Pharmaceuticals. And and joining us from from the company is the chief executive officer, Brian Goff, as well as the chief financial officer, Cecilia Jones. Guys, it's great to great to have y ...

Summary of Agios Pharmaceuticals Conference Call Company Overview - **Company**: Agios Pharmaceuticals (AGIO) - **Focus**: Development of transformative medicines for patients with rare diseases, particularly those affecting red blood cells such as pyruvate kinase deficiency (PKD), thalassemia, sickle cell disease, and low-risk myelodysplastic syndromes (MDS) [6][7] Key Pipeline Developments - **Product Highlight**: PyraKine (Mitapivat) - **Mechanism**: Novel approach to enhance red blood cell metabolism, leading to improved energy and healthier cells [7] - **Current Status**: Commercialized for PKD; pursuing indications in thalassemia and sickle cell disease [7] Thalassemia - **Pivotal Studies**: Two phase three studies (ENERGIZE and ENERGIZE T) showed statistical significance on all primary and key secondary endpoints [8] - **Regulatory Filings**: Filed for approval in the US, Europe, Saudi Arabia, and UAE; FDA PDUFA goal date set for September 7, 2025 [8][14] - **Commercial Readiness**: Sales team fully prepared for the launch of PyraKine in thalassemia [9][35] Sickle Cell Disease - **Study Enrollment**: Fully enrolled the Rise Up phase three study, a 52-week trial [9] - **Data Readout**: Expected by year-end 2025 [9] - **Trial Design**: Focuses on hemolytic anemia and vaso-occlusion, with two primary endpoints [42][43] Financial Position - **Balance Sheet**: Approximately $1.4 billion in cash, positioning the company strongly in the current macroeconomic environment [10][12][61] - **Capital Allocation Strategy**: Focus on maximizing value from launches, advancing the pipeline, and exploring external opportunities for growth [64] Market Opportunities - **Thalassemia Patient Population**: Estimated 6,000 adult patients in the US and 70,000 in Saudi Arabia, with a prevalence rate significantly higher in the Middle East [32] - **Regulatory Engagement**: Positive interactions with regulatory bodies across multiple markets, indicating a smooth approval process [24][30] Competitive Landscape - **Treatment Paradigm**: Oral therapies like Mitapivat are positioned as accessible options compared to gene therapies, which may have more complex onboarding processes [54] - **Future Trials**: Plans to initiate a phase two trial for another PK activator, tebipivat, to provide additional treatment options for sickle cell disease [48][50] Upcoming Events - **EHA Presentations**: Agios will present data on pediatric PKD and sickle cell disease at the upcoming EHA conference, showcasing their commitment to advancing research and treatment options [58][59] Conclusion - **Strategic Vision**: Agios aims to build a multibillion-dollar franchise with PyraKine while maintaining a disciplined approach to capital allocation and exploring new opportunities for growth [64]

Core Insights - Agios Pharmaceuticals is presenting new data on its pyruvate kinase (PK) activators, mitapivat and tebapivat, at the upcoming European Hematology Association (EHA) Congress in Milan, Italy, from June 12-15, 2025 [1][2] Company Overview - Agios Pharmaceuticals is a leader in cellular metabolism and PK activation, focusing on therapies for rare diseases [1][17] - The company has developed PYRUKYND (mitapivat), a first-in-class pyruvate kinase activator for treating hemolytic anemia in adults with PK deficiency [9][17] Clinical Data Presentation - A total of 14 presentations and publications will be shared at EHA 2025, highlighting the efficacy and safety of PK activation in treating rare blood disorders [3][2] - Key presentations include: - Results from the ACTIVATE-KidsT Phase 3 study of mitapivat in children with PK deficiency, showing a clinically meaningful reduction in transfusion burden [6] - Long-term data from the ESTIMATE Phase 2 trial of mitapivat in sickle cell disease, demonstrating sustained efficacy and tolerability over three years [6] - Preclinical data on tebapivat's potential in reducing red blood cell sickling in sickle cell disease patients [6] Research Focus Areas - The presentations will cover serious conditions with limited treatment options, including sickle cell disease, thalassemia, PK deficiency, and myelodysplastic syndromes [2][6] - Ongoing investigations include the expression patterns of PKM2 in patients with myelodysplastic syndromes, supporting the potential of tebapivat in lower-risk MDS [6] Collaboration and Community Engagement - Agios aims to strengthen its collaboration with the global hematology community through these presentations at EHA [2]

Core Insights - Agios Pharmaceuticals is scheduled to present at the 2025 RBC Capital Markets Global Healthcare Conference on May 21, 2025, at 8:00 am ET [1] - The presentation will be accessible via a live webcast on the company's website, with a replay available for at least two weeks [2] Company Overview - Agios Pharmaceuticals is a leader in cellular metabolism and PK activation, focusing on therapies for rare diseases [3] - The company markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, which is the first disease-modifying therapy for this condition [3] - Agios is advancing a clinical pipeline that includes investigational medicines for alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia, and phenylketonuria (PKU) [3] - Additionally, Agios is developing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera [3]

Core Viewpoint - Agios Pharmaceuticals reported a narrower loss per share in Q1 2025 compared to estimates, but revenues fell short of expectations despite a year-over-year increase Financial Performance - Agios incurred a loss of $1.55 per share in Q1 2025, better than the Zacks Consensus Estimate of a loss of $1.80, but wider than the loss of $1.45 reported in the same quarter last year [1] - Revenues for Q1 2025 were $8.7 million, missing the Zacks Consensus Estimate of $10 million, but representing a 6.1% increase from $8.2 million in the year-ago quarter [1] - The company's stock has declined 5.8% year-to-date, while the industry has seen a decrease of 1.7% [2] Product Performance - All revenues in Q1 2025 were generated from Pyrukynd, Agios' only marketed drug, which is approved for treating hemolytic anemia in adults with PK deficiency [3] - Pyrukynd revenues declined 19% sequentially due to the absence of year-end stocking benefits seen in Q4 2024 [3] - As of Q1 2025, 136 patients are on Pyrukynd therapy, reflecting a 4.6% increase from Q4 2024 [4] Expenses - Research and development expenses rose approximately 6% year-over-year to $72.7 million, attributed to higher workforce-related costs [4] - Selling, general, and administrative expenses surged 33.9% year-over-year to $41.5 million, driven by preparations for potential approval and launch of Pyrukynd for thalassemia [4] Cash Position - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $1.4 billion, down from $1.5 billion as of December 31, 2024 [5] Pipeline Developments - The FDA accepted Agios' supplemental new drug application for Pyrukynd to treat adult patients with thalassemia, with a decision expected by September 7, 2025 [9] - Agios completed enrollment in the phase III RISE UP study for SCD, with top-line data expected in late 2025 and potential regulatory filing in 2026 [10] - The company is also conducting two phase III studies for Pyrukynd in pediatric patients with PK deficiency [10] - Agios is developing tebapivat for myelodysplastic syndromes and has initiated a phase IIb study, with enrollment expected to complete in late 2025 [12]