Q1 2024 Financial Results May 2, 2024 1 Agios conference call participants TOPIC PARTICIPANT Chris Taylor, VP Investor Relations and Corporate Introduction Communications Business Update Brian Goff, Chief Executive Officer Sarah Gheuens, M.D., Ph.D., ...

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) Q1 2024 Earnings Conference Call May 2, 2024 8:00 AM ET Company Participants Chris Taylor - Vice President, Investor Relations and Corporate Communications Brian Goff - Chief Executive Officer Sarah Gheuens - Chief Medical Officer and Head, R&D Tsveta Milanova - Chief Commercial Officer Cecilia Jones - Chief Financial Officer Conference Call Participants Gregory Renza - RBC Capital Markets Divya Rao - TD Cowen Eric Schmidt - Cantor Alec Stranahan - Bank of America T ...

Agios Pharmaceuticals (AGIO) came out with a quarterly loss of $1.45 per share versus the Zacks Consensus Estimate of a loss of $1.64. This compares to loss of $1.47 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 11.59%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $1.64 per share when it actually produced a loss of $1.72, delivering a surprise of -4.88%.Over the last four quarters, ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number: 001-36014 AGIOS PHARMACEUTICALS, INC. (Exact Name of Registrant as Specified in Its Charter) Delaware 26-0662915 (State or Other Jurisdiction of In ...

Exhibit 99.1 Agios Reports Business Highlights and First Quarter 2024 Financial Results – Topline Data Readout from the Phase 3 ENERGIZE-T Study of Mitapivat in Adults with Transfusion- Dependent Alpha- or Beta-Thalassemia now expected in Q2 2024 – – Announced Positive Results from the Phase 3 ENERGIZE Study of Mitapivat in Adults with Non-Transfusion- Dependent Alpha- or Beta-Thalassemia in January – – Phase 3 Readouts from Mitapivat RISE UP Study in Sickle Cell Disease, ACTIVATE-KIDS and ACTIVATE KIDS-T i ...

– Report Highlights Company's Focus on Red Blood Cell Health; Diversity, Equity & Inclusion; and Strong Corporate Governance – – Aligns with the Sustainability Accounting Standards Board (SASB) Standards for the Biotechnology and Pharmaceuticals Industry and the United Nations Sustainable Development Goals (UN SDGs) – CAMBRIDGE, Mass., April 09, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today ...

Agios Pharmaceuticals, Inc. (AGIO) reported a loss of $1.72 per share in fourth-quarter 2023, wider than the Zacks Consensus Estimate of a loss of $1.64. In the year-ago quarter, the company posted a loss of 67 cents per share.AGIO reported revenues of $7.1 million, which also missed the Zacks Consensus Estimate of $8 million. In the year-ago quarter, the company recorded revenues of $4.3 million.Quarter in DetailIn the reported quarter, revenues were generated entirely from product revenues of Agios’ only ...

Financial Data and Key Metrics - Full year 2023 net PYRUKYND revenue was $26.8 million, compared to $11.7 million in 2022 [25] - Q4 2023 net PYRUKYND revenue was $7.1 million, a 4% reduction compared to Q3 2023, driven by lower inventory levels [25] - R&D expenses for Q4 2023 were $77 million, and $296 million for the full year 2023, an increase of $16 million compared to 2022 [26] - SG&A expenses for Q4 2023 were $35 million, and $120 million for the full year 2023, a decrease of $2 million compared to 2022 [27] - The company ended 2023 with approximately $806 million in cash, cash equivalents, and marketable securities [27] Business Line Data and Key Metrics - The PK activator franchise, led by mitapivat, reported three key data readouts in the last 12 months, including positive Phase 2 data in sickle cell disease and Phase 3 data in non-transfusion-dependent thalassemia [6][7] - The Phase 3 ENERGIZE study for mitapivat in non-transfusion-dependent thalassemia showed a 42.3% hemoglobin response rate in the treatment arm compared to 1.6% in the placebo arm [15] - The company is preparing for potential U.S. launches of mitapivat in thalassemia in 2025 and sickle cell disease in 2026 [8] - AG-946, another PK activator, showed positive Phase 2a data in lower-risk MDS, with 40% of patients achieving transfusion independence [6] Market Data and Key Metrics - In the U.S., there are approximately 6,000 diagnosed adult patients with thalassemia, with 4,000 being non-transfusion-dependent and 2,000 being transfusion-dependent [19] - The company estimates approximately 13,000 thalassemia patients in the EU5 and 70,000 in the Gulf region [22] - For sickle cell disease, the U.S. patient population is estimated at 100,000, with significant unmet needs for novel oral therapies [59] Company Strategy and Industry Competition - The company aims to position mitapivat as a foundational therapy for sickle cell disease, leveraging its unique mechanism of action and oral administration [57] - The company is focused on building a multi-billion dollar PK activation franchise, with potential launches in thalassemia and sickle cell disease [8][29] - The company is preparing for a potential U.S. launch of mitapivat in thalassemia in 2025, followed by sickle cell disease in 2026 [8][22] - The company is also advancing AG-946 in lower-risk MDS and exploring its potential in sickle cell disease [69] Management Commentary on Operating Environment and Future Outlook - The company is confident in the potential of its PK activation pipeline, with multiple near-term catalysts expected to drive significant value [7] - The company expects to deliver four additional Phase 3 readouts and two potential launches in the near term [8] - The company believes its strong cash position will enable it to fund operating expenses and capital expenditures through 2026 [27] Other Important Information - The company retains rights to a potential $200 million milestone upon FDA approval of vorasidenib and 15% royalties on potential U.S. net sales [27] - The company is conducting extensive market research and claims data analysis to inform its launch strategy for thalassemia [20][21] Q&A Session Summary Question: ENERGIZE-T Study Primary Endpoint - The primary endpoint for the ENERGIZE-T study is a 50% reduction in transfusions over any 12-week rolling period, which differs from Luspatercept's fixed-period endpoint [33] - The company believes this dynamic endpoint better reflects real-world patient experience [33] Question: ENERGIZE Data Transition from Phase 2 to Phase 3 - The Phase 3 ENERGIZE study incorporated a longer duration for hemoglobin response measurement, which differs from the single time point used in Phase 2 [36] - The company does not observe waning efficacy over time, with hemoglobin response maintained similarly to PKD [36] Question: Commercial Coexistence of Mitapivat and Luspatercept - Mitapivat is positioned as an oral therapy for non-transfusion-dependent thalassemia, where no current therapies exist, while Luspatercept is a subcutaneous option for transfusion-dependent patients [39] - The company emphasizes the different mechanisms of action, with mitapivat focusing on overall red blood cell health [40] Question: Powering Assumptions for ENERGIZE-T - The company has not disclosed specific powering assumptions but has studied internal programs to inform its development approach [44] - The focus is on reducing transfusions rather than increasing hemoglobin levels, reflecting the different mechanism of action [44] Question: Multibillion Dollar Opportunity in PK Activation - The company sees significant potential in moving from ultra-rare diseases like PKD to larger prevalent diseases like sickle cell and thalassemia [51] - The company is preparing for back-to-back launches in thalassemia and sickle cell disease, with a focus on pricing dynamics and commercial opportunities [52] Question: Differences Between Mitapivat and Other PK Activators - Mitapivat stimulates multiple PK isoenzymes, including PKR and PKM2, which are relevant for red blood cell health and other tissues affected by diseases like sickle cell and MDS [53] - The company is planning further clinical studies to explore the role of PKM2 in sickle cell disease, particularly in kidney health [53] Question: Sickle Cell Disease Commercial Landscape - The company believes mitapivat has the potential to be a foundational therapy for sickle cell disease, offering benefits in hemoglobin improvement and VOC reduction [57][59] - The company is preparing for a potential launch in 2026, following the thalassemia launch in 2025 [60] Question: PKD Commercial Opportunity - The company maintains its peak sales estimate for PKD at $200 million to $225 million in the U.S., with slow and steady growth expected [65] - The company is encouraged by patient persistency and is building capabilities for future launches [66] Question: AG-946 Development Strategy - The company is advancing AG-946 in lower-risk MDS and exploring its potential in sickle cell disease, with plans to test multiple dose levels in Phase 2b [69][74] - The company is incorporating patient voice and regulatory feedback into trial design [70] Question: ENERGIZE-T Primary Endpoint Design - The primary endpoint design for ENERGIZE-T was influenced by regulatory feedback, with the company believing the dynamic endpoint better reflects real-world patient experience [73] Question: AG-946 Phase 2b Design in MDS - The Phase 2b trial for AG-946 in MDS will test multiple dose levels, including higher doses than initially anticipated, based on Phase 2a learnings [74] - The trial will focus on patients with transfusion burden and will include a broad MDS population [74]

Agios Pharmaceuticals (AGIO) came out with a quarterly loss of $1.72 per share versus the Zacks Consensus Estimate of a loss of $1.64. This compares to earnings of $0.67 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -4.88%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $1.69 per share when it actually produced a loss of $1.64, delivering a surprise of 2.96%.Over the last four quarter ...

Q4 and Full Year 2022 Financial Results February 23, 2023 Agios conference call participants ...