Exhibit 99.1 Agios Reports Business Highlights and Second Quarter 2024 Financial Results – Reported Positive Topline Data from Phase 3 ENERGIZE-T Study; Expect to File sNDA Based on ENERGIZE and ENERGIZE-T Studies Encompassing All Thalassemia Subtypes by End of 2024 – – Announced $905 Million Purchase Agreement for Vorasidenib Royalty with Royalty Pharma; Agios to Receive a Total of $1.1 Billion in Payments Upon FDA Approval of Vorasidenib – – Reported Results from Phase 3 ACTIVATE-KidsT Study of Mitapivat ...

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 ...

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 ...

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]

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

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 Form 10-K (Mark One) ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 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 incorpo ...

Financial Performance - The company reported a net loss of $256.1 million for the nine months ended September 30, 2023, compared to a net loss of $268.3 million for the same period in 2022[91]. - As of September 30, 2023, the company had an accumulated deficit of $726.7 million[91]. - Net loss for the three months ended September 30, 2023, was $91.3 million, compared to a net loss of $81.7 million for the same period in 2022, driven by a $17.5 million up-front payment associated with the agreement with Alnylam[118]. - Total revenue for the nine months ended September 30, 2023, was $19.7 million, up $9.8 million from the same period in 2022, primarily due to increased product revenue from PYRUKYND®[109]. - PYRUKYND® product revenue for Q3 2023 was $7.4 million, an increase of $3.9 million compared to Q3 2022, attributed to its FDA approval in February 2022[108]. - Net cash used in operating activities was $223.6 million for the nine months ended September 30, 2023, compared to $243.3 million for the same period in 2022[124]. - Cash, cash equivalents, and marketable securities balance was $872.4 million at September 30, 2023, down from $1.1 billion at December 31, 2022[137]. Research and Development - The lead product candidate, PYRUKYND®, received FDA approval on February 17, 2022, for the treatment of hemolytic anemia in adults with PK deficiency[93]. - The company expects to incur significant expenses and net losses as it continues to advance clinical development activities for PYRUKYND® and AG-946[91]. - The company continues to invest in late-stage research programs, including a PAH stabilizer for the treatment of phenylketonuria[83]. - AG-946, a novel PK activator, is in a phase 1 trial with topline results expected by the end of 2023[103]. - The company expects to report topline data from the ENERGIZE trial in the first half of 2024, which evaluates PYRUKYND® for non-transfusion-dependent thalassemia[101]. - The ENERGIZE-T trial has completed enrollment, with topline data expected in the second half of 2024, focusing on transfusion-dependent thalassemia patients[101]. - Total research and development expenses increased by $8.4 million for the nine months ended September 30, 2023, primarily due to an $18.4 million increase in direct expenses, partially offset by a $9.9 million decrease in indirect expenses[114]. - Research and development expenses for Q3 2023 were $81.8 million, an increase of $16.9 million compared to Q3 2022, driven by higher direct expenses related to PYRUKYND® and in-process research and development[113]. Commercialization and Sales - The company anticipates significant commercialization expenses related to product sales, marketing, manufacturing, and distribution as it continues to develop and commercialize PYRUKYND®[130]. - The company has initiated a global managed access program for PYRUKYND® in the EU and Great Britain, providing free access to eligible patients[100]. - The company anticipates generating future revenue from product sales, royalties, and potential collaborations or licensing agreements[94]. - The company recognized income of $127.9 million from the sale of rights to future contingent payments related to its oncology business[86]. - The sale of the oncology business to Servier included a payment of approximately $1.8 billion in cash at closing, with additional contingent payments based on future approvals[84]. Agreements and Milestones - The company made an upfront payment of $17.5 million to Alnylam Pharmaceuticals for the development and commercialization rights of a novel siRNA targeting TMPRSS6[88]. - The company is responsible for up to $130.0 million in potential development and regulatory milestones related to the licensed product from Alnylam[88]. - A license agreement with Alnylam for the development of products targeting the TMPRSS6 gene was established in July 2023, with an IND filing expected by the end of 2023[104]. - The company entered into a license agreement with Alnylam, which may require up to $130.0 million in potential development and regulatory milestones[136]. Cash Flow and Investments - Cash provided by investing activities was $144.1 million for the nine months ended September 30, 2023, primarily due to higher proceeds from maturities and sales of marketable securities[126]. - The company expects to fund its operating expenses and capital expenditures through existing cash, anticipated product revenue, and potential milestone payments through at least 2026[131]. - Interest income increased significantly due to rising interest rates, with net interest income of $24.7 million for the nine months ended September 30, 2023, compared to $6.3 million in 2022[115]. - The company sold its rights to future contingent payments associated with royalties on U.S. net sales of TIBSOVO® for $131.8 million in October 2022[121]. Market Risks - The company is exposed to market risk related to changes in foreign currency exchange rates due to contracts with CROs in Asia and Europe[138]. - As of September 30, 2023, and December 31, 2022, liabilities denominated in foreign currencies were immaterial[138].

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=3&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Unaudited condensed consolidated financial statements are presented, detailing the company's financial position and performance [Condensed Consolidated Balance Sheets](index=3&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to **$1.09 billion** as of June 30, 2023, driven by reduced cash and marketable securities Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $84,753 | $139,259 | | Marketable securities | $862,170 | $957,734 | | Total current assets | $757,239 | $832,772 | | **Total assets** | **$1,085,153** | **$1,238,718** | | **Liabilities & Equity** | | | | Total current liabilities | $55,141 | $62,629 | | **Total liabilities** | **$120,917** | **$137,904** | | **Total stockholders' equity** | **$964,236** | **$1,100,814** | [Condensed Consolidated Statements of Operations](index=4&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Total revenue increased to **$12.3 million**, while net loss narrowed to **$164.8 million** for the six months ended June 30, 2023 Statement of Operations Summary (in thousands, except per share data) | Metric | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Product revenue, net | $6,712 | $3,082 | $12,321 | $3,914 | | Total revenue | $6,712 | $5,582 | $12,321 | $6,414 | | Research and development | $68,895 | $74,523 | $136,196 | $144,646 | | Total operating expenses | $100,412 | $103,222 | $196,634 | $205,199 | | Loss from operations | ($93,700) | ($97,640) | ($184,313) | ($198,785) | | Net loss | ($83,806) | ($91,806) | ($164,824) | ($186,580) | | Net loss per share | ($1.51) | ($1.68) | ($2.97) | ($3.41) | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities improved to **$161.9 million**, resulting in a **$54.5 million** net decrease in cash Cash Flow Summary (in thousands) | Activity | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($161,931) | ($171,964) | | Net cash provided by investing activities | $104,721 | $56,240 | | Net cash provided by financing activities | $2,704 | $1,141 | | **Net change in cash and cash equivalents** | **($54,506)** | **($114,583)** | | Cash and cash equivalents at end of period | $84,753 | $88,543 | [Notes to Condensed Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes provide detailed information on the company's operations, accounting policies, and key financial statement items - The company's lead product is **PYRUKYND® (mitapivat)**, an activator of pyruvate kinase enzymes, approved for hemolytic anemia in adults with **PK deficiency** in the U.S., EU, and Great Britain, and is also in clinical trials for **thalassemia**, **sickle cell disease (SCD)**, and **pediatric PK deficiency**[25](index=25&type=chunk) - The company is also developing **AG-946**, another **PK activator**, for **lower-risk myelodysplastic syndrome (LR MDS)** and **hemolytic anemias**, and has a late-stage research program for a **PAH stabilizer** for **phenylketonuria (PKU)**[25](index=25&type=chunk)[26](index=26&type=chunk) - On July 28, 2023, Agios entered into a license agreement with **Alnylam Pharmaceuticals** to develop and commercialize a preclinical **siRNA targeting TMPRSS6** for **polycythemia vera (PV)**, involving a **$17.5 million upfront payment** and up to **$130.0 million in potential development and regulatory milestones**[75](index=75&type=chunk) - As of June 30, 2023, the company had **$946.9 million in cash, cash equivalents, and marketable securities** and expects this to be **sufficient to fund operations for at least the next twelve months**[35](index=35&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=18&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial condition and results, highlighting revenue growth, expense control, and liquidity into 2026 [Results of Operations](index=23&type=section&id=Results%20of%20Operations) Total revenue increased, operating expenses decreased, and net loss narrowed to **$164.8 million** for the six months ended June 30, 2023 Revenue Comparison (in thousands) | Revenue Type | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | Product revenue, net | $12,321 | $3,914 | | Milestone revenue | $0 | $2,500 | | **Total revenue** | **$12,321** | **$6,414** | Operating Expense Comparison (in thousands) | Expense Category | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | Cost of sales | $1,662 | $774 | | Research and development | $136,196 | $144,646 | | Selling, general and administrative | $58,776 | $59,779 | | **Total operating expenses** | **$196,634** | **$205,199** | - The **$8.5 million decrease in R&D expenses** for the six-month period was primarily due to an **$8.7 million decrease in indirect expenses** (lower compensation and facilities costs), partially offset by a **$0.2 million increase in direct expenses** driven by higher costs for **PYRUKYND® trials** in **thalassemia** and **SCD**[108](index=108&type=chunk) - The **decrease in net loss to $164.8 million** for the first six months of 2023 from **$186.6 million** in 2022 was driven by **higher interest income**, **lower R&D expenses**, and **increased revenue**, partially offset by the **elimination of royalty income** from the sale of TIBSOVO® rights[113](index=113&type=chunk) [Liquidity and Capital Resources](index=25&type=section&id=Liquidity%20and%20Capital%20Resources) The company had **$946.9 million** in cash, cash equivalents, and marketable securities, expected to fund operations into 2026 - The company's cash, cash equivalents, and marketable securities balance was **$946.9 million** at June 30, 2023[116](index=116&type=chunk) - The company expects its **existing cash**, anticipated product revenue, interest income, and the **potential vorasidenib milestone payment** to **fund operating expenses and capital expenditures at least into 2026**[123](index=123&type=chunk) - Future capital requirements depend on factors like **PYRUKYND® sales**, **contingent payments from Servier**, **clinical trial costs**, and **potential in-licensing activities**, including **payments to Alnylam**[124](index=124&type=chunk)[203](index=203&type=chunk) Cash Flow Summary (in thousands) | Activity | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($161,931) | ($171,964) | | Net cash provided by investing activities | $104,721 | $56,240 | | Net cash provided by financing activities | $2,704 | $1,141 | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=27&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Primary market risk is interest rate sensitivity on the **$946.9 million** cash portfolio, with immaterial foreign currency risk - The primary market risk is **interest rate sensitivity** on the company's **$946.9 million portfolio of cash, cash equivalents, and marketable securities** as of June 30, 2023[129](index=129&type=chunk) - The company has some exposure to **foreign currency exchange rate fluctuations** due to contracts with CROs in Europe and Asia, but this risk is considered **immaterial** as of June 30, 2023[130](index=130&type=chunk) [Item 4. Controls and Procedures](index=27&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were **effective** as of June 30, 2023, with no material changes to internal control - Management concluded that as of June 30, 2023, the company's disclosure controls and procedures were **effective**[131](index=131&type=chunk) - **No changes** occurred during the fiscal quarter ended June 30, 2023, that have **materially affected**, or are reasonably likely to materially affect, the company's internal control over financial reporting[133](index=133&type=chunk) PART II. OTHER INFORMATION [Item 1A. Risk Factors](index=29&type=section&id=Item%201A.%20Risk%20Factors) Significant risks include heavy dependence on **PYRUKYND®**, intense competition, reliance on third parties, and healthcare reform impacts - The company's success is **heavily dependent** on the successful commercialization of **PYRUKYND®** and the clinical development of its other product candidates, where failure or significant delays would **materially harm the business**[137](index=137&type=chunk)[139](index=139&type=chunk) - The company faces **substantial competition** from major pharmaceutical and biotech companies like Merck, BMS, Novartis, and Vertex, which have **greater resources** and are developing therapies for the same or similar indications[168](index=168&type=chunk)[170](index=170&type=chunk) - The company **relies on third parties** (CROs and manufacturers) for **clinical trials** and **product supply**, which introduces risks related to performance, regulatory compliance, and potential **supply chain disruptions**[212](index=212&type=chunk)[219](index=219&type=chunk) - The business is subject to risks from **healthcare reform**, including the **Inflation Reduction Act (IRA)**, which could lead to **government price negotiations**, and other **downward pricing pressures** that could **harm profitability**[277](index=277&type=chunk)[286](index=286&type=chunk)[287](index=287&type=chunk) - As a **smaller, less diversified company** focused on rare diseases after selling its oncology business, Agios is **more susceptible to market fluctuations and risks** specific to this sector[197](index=197&type=chunk)[198](index=198&type=chunk) [Item 5. Other Information](index=61&type=section&id=Item%205.%20Other%20Information) This section details a significant exclusive worldwide **license agreement with Alnylam Pharmaceuticals** for a preclinical siRNA candidate - Agios entered into a **license agreement with Alnylam** on July 28, 2023, for an **exclusive worldwide license** to develop and commercialize an **siRNA candidate targeting the TMPRSS6 gene**[320](index=320&type=chunk)[321](index=321&type=chunk) Alnylam License Agreement Financial Terms | Payment Type | Amount | | :--- | :--- | | Upfront Payment | $17.5 million | | Potential Development & Regulatory Milestones | Up to $130.0 million | | Royalties | Tiered percentages from mid-single digits to mid-teens on annual net sales | [Item 6. Exhibits](index=62&type=section&id=Item%206.%20Exhibits) Exhibits filed include corporate governance documents, stock incentive agreements, and officer certifications - Exhibits filed include forms of **stock option** and **restricted stock unit agreements** under the **2023 Stock Incentive Plan**[328](index=328&type=chunk) - **Certifications from the CEO and CFO** pursuant to the **Securities Exchange Act of 1934** and the **Sarbanes-Oxley Act of 2002** are included as exhibits[328](index=328&type=chunk)