The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be archived on the company's website for at least two weeks following the presentation. Investor Contact Morgan Sanford, Vice President, Investor Relations Agios Pharmaceuticals IR@agios.com Media Contact Eamonn Nolan, Senior Director, Corporate Communications Agios Pharmaceuticals Media@agios.com About Agios Agios is the pioneering leader ...

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]

Core Insights - Agios Pharmaceuticals is scheduled to present at the BofA Securities 2025 Health Care Conference on May 14, 2025 [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]

Financial Performance - The net loss for the three months ended March 31, 2025, was $89.3 million, compared to a net loss of $81.5 million for the same period in 2024, resulting in an accumulated deficit of $238.2 million as of March 31, 2025[89]. - Total revenue for the three months ended March 31, 2025, was $8.726 million, an increase of $0.5 million compared to $8.189 million in the same period of 2024[120]. - Total operating expenses for the three months ended March 31, 2025, were $115.355 million, an increase of $15.1 million compared to $100.261 million in the same period of 2024[121]. - Research and development expenses for the three months ended March 31, 2025, totaled $72.743 million, an increase of $4.1 million compared to $68.620 million in the same period of 2024[123]. - Net cash used in operating activities for the three months ended March 31, 2025, was $111.5 million, compared to $99.9 million for the same period in 2024[132][133]. - Cash provided by investing activities was $112.6 million for the three months ended March 31, 2025, compared to $124.6 million in 2024[134]. - Interest income, net for the three months ended March 31, 2025, was $16.087 million, an increase from $8.889 million in the same period of 2024[124]. - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $1.4 billion[131]. Product Development and Approvals - PYRUKYND® is approved by the FDA for treating hemolytic anemia in adults with PK deficiency, and a supplemental new drug application was submitted for treating alpha- or beta-thalassemia, with a PDUFA goal date of September 7, 2025[81]. - The company is evaluating PYRUKYND® in a phase 3 clinical trial for sickle cell disease and developing other candidates like tebapivat for lower-risk myelodysplastic syndromes[82]. - PYRUKYND® received FDA approval for treating hemolytic anemia in adults with PK deficiency and marketing authorization in the EU and Great Britain[99]. - The company submitted an sNDA to the FDA for PYRUKYND® for treating non-transfusion dependent and transfusion-dependent alpha- or beta-thalassemia, with a PDUFA goal date of September 7, 2025[99]. - Tebapivat has been granted orphan drug designation for the treatment of MDS by the FDA, with a phase 2 clinical trial expected to begin in mid-2025[114]. Clinical Trials and Results - In the ENERGIZE trial, 42.3% of patients on PYRUKYND® achieved a hemoglobin response compared to 1.6% in the placebo group, demonstrating statistical significance (p<0.0001) with 194 patients enrolled[102]. - In the ENERGIZE-T trial, 30.4% of patients on PYRUKYND® achieved a transfusion reduction response compared to 12.6% in the placebo group (p=0.0003), with 258 patients enrolled[105]. - The RISE UP study showed that 46.2% and 50.0% of patients in the 50 mg and 100 mg arms, respectively, achieved a hemoglobin response compared to 3.7% in the placebo arm (p=0.0003 and 0.0001) with 79 patients enrolled[109]. - 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[111]. - In the ACTIVATE-kids study, 31.6% of patients in the mitapivat arm achieved a hemoglobin response, while 0% of patients in the placebo arm achieved this response[113]. - The company is evaluating PYRUKYND® in clinical trials for SCD and pediatric patients with PK deficiency[99]. - The company has updated clinical trial protocols to include monthly monitoring of liver tests for the first six months of treatment due to potential hepatocellular injury risks[107]. - 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[109]. Business Transactions and Collaborations - The sale of the oncology business to Servier was completed for approximately $1.8 billion in cash, with additional contingent payments based on future approvals and sales[83]. - In September 2024, the company recognized income of $200.0 million from the Vorasidenib Milestone Payment following FDA approval for vorasidenib[86]. - The company recognized income of $889.1 million from the sale of Vorasidenib Royalty Rights to Royalty Pharma, net of fees[87]. - The company entered into a distribution agreement with NewBridge Pharmaceuticals for PYRUKYND® in the GCC region, expanding its market reach[91]. - The company entered a distribution agreement with NewBridge Pharmaceuticals for PYRUKYND® commercialization in the GCC region[100]. Future Outlook and Risks - The company expects to continue generating revenue from product sales and potential milestone payments from collaborations or licensing agreements in the future[92]. - Research and development expenses are expected to increase as product candidate development programs progress, with significant uncertainty regarding costs and timelines[95]. - The company has historically incurred significant operating losses and expects to continue incurring significant expenses until achieving profitable results[89]. - The company expects expenses to increase as it continues research, development, and commercialization of its product candidates, including PYRUKYND®[136]. - Future capital requirements will depend on various factors, including the success of product sales and potential royalty payments from Retained Earn-Out Rights[139]. - The company is exposed to market risks related to interest rates and foreign currency exchange rates, with minimal liabilities in foreign currencies as of March 31, 2025[142][145]. - The company anticipates financing cash needs primarily through cash on hand, potential royalty payments, and product sales until substantial revenue is generated[139]. - The company may need additional capital resources to fund operating plans and capital expenditures, which could involve dilution of stockholder ownership[139].

Agios Pharmaceuticals (AGIO) Q1 2025 Earnings Call May 01, 2025 08:00 AM ET Company Participants Christopher Taylor - VP - IR & Corporate CommunicationsBrian Goff - CEOSarah Gheuens - Chief Medical Officer and Head of Research & DevelopmentTsveta Milanova - CCOCecilia Jones - CFOHiromichi Nagayumi - Associate - Biotech Equity ResearchAmanda Acosta-Ruiz - Equity Research Associate - Biotechnology Conference Call Participants Gregory Renza - Director & Senior Analyst of Biotechnology Equity ResearchAlec Stran ...

Q1 2025 Financial Results and Business Update May 1, 2025 1 Agios Conference Call Participants | TOPIC | PARTICIPANT | | --- | --- | | Introduction | Chris Taylor, VP Investor Relations and Corporate | | | Communications | | Business Update | Brian Goff, Chief Executive Officer | | R&D Update | Sarah Gheuens, M.D., Ph.D., Chief Medical Officer, | | | Head of R&D | | Commercial Update | Tsveta Milanova, Chief Commercial Officer | | First Quarter 2025 Financial Results | Cecilia Jones, Chief Financial Officer ...