Financial Performance - PYRUKYND generated net revenue of $8.7 million in Q1 2025, up from $8.2 million in Q1 2024, representing a year-over-year increase of 6.1%[3] - The net loss for Q1 2025 was $89.3 million, compared to a net loss of $81.5 million in Q1 2024, reflecting an increase of 9.8%[7] - Cash, cash equivalents, and marketable securities totaled $1.4 billion as of March 31, 2025, down from $1.5 billion at the end of 2024[8] Expenses - Research and Development (R&D) expenses for Q1 2025 were $72.7 million, an increase of 5.2% compared to $68.6 million in Q1 2024[5] - Selling, General and Administrative (SG&A) expenses rose to $41.5 million in Q1 2025, compared to $31.0 million in Q1 2024, marking a 33.9% increase[7] Patient Enrollment - The total number of unique patients who completed prescription enrollment forms for PYRUKYND increased by 5% from Q4 2024, reaching 234 patients[3] Regulatory and Clinical Developments - The FDA accepted the supplemental New Drug Application (sNDA) for PYRUKYND for thalassemia treatment, with a PDUFA goal date of September 7, 2025[2] - The Phase 3 RISE UP study for mitapivat in sickle cell disease is on track, with topline results expected in late 2025 and a potential U.S. commercial launch in 2026[2] - Agios plans to file an Investigational New Drug Application for AG-236, targeting TMPRSS6 for polycythemia vera, in mid-2025[5] Strategic Focus - The company is advancing its pipeline with a focus on transformative therapies for rare diseases, aiming to create significant value for shareholders[2]

Financial Data and Key Metrics Changes - In Q4 2024, net revenue was $10.7 million, a 51% increase compared to $7.1 million in Q4 2023, primarily driven by year-end stocking and adjustments to revenue reserves [43][44]. - Cost of sales for the quarter was $1.3 million, with R&D expenses at $82.8 million, an increase of $5.3 million year-over-year, mainly due to workforce-related expenses [44]. - G&A expenses were $51.7 million for Q4, up $16.4 million compared to the prior year, driven by increased commercial activities in preparation for PYRUKYND's potential approval in thalassemia [45]. Business Line Data and Key Metrics Changes - PYRUKYND generated $10.7 million in net revenue in Q4 2024, up from $9 million in Q3 2024, with 223 patients completing prescription enrollment forms [38][39]. - The company anticipates a potential approval and launch of PYRUKYND in thalassemia in September 2025, followed by sickle cell disease in 2026 [9][14]. Market Data and Key Metrics Changes - The U.S. market for thalassemia is significant, with 67% of diagnosed patients having no approved therapies, presenting a substantial opportunity for PYRUKYND [22][63]. - The global prevalence of sickle cell disease exceeds 3 million, with an urgent need for novel therapeutic options [24][27]. Company Strategy and Development Direction - The company aims to maximize the potential of the PYRUKYND franchise, advance and diversify pipeline programs, and strategically focus capital deployment to sustain growth [12][55]. - The anticipated launch in thalassemia is expected to address a large patient population, with a robust disease state education campaign underway [33][40]. Management's Comments on Operating Environment and Future Outlook - Management expressed optimism for 2025, highlighting it as a breakout year with multiple regulatory and clinical milestones, including the PDUFA date for thalassemia [54][55]. - The company is well-prepared for the upcoming launch, with a strong balance sheet providing financial independence to support growth initiatives [47][49]. Other Important Information - The company received $1.1 billion in milestone payments related to the FDA approval of vorasidenib, enhancing its financial position [46]. - Dr. David Schenkein will step down from the Board of Directors effective February 28, 2025, but will continue as a strategic adviser [51][52]. Q&A Session Summary Question: What is the company's plan for updating the investment community on the safety profile of mitapivat? - Management stated they would update the community if there are any changes to the safety profile as they have done previously [61]. Question: How does the company view the peak sales potential in thalassemia or sickle cell disease? - Management highlighted the significant unmet need in thalassemia, with two-thirds of the patient population lacking approved therapies, and expressed confidence in the long-term potential [63][64]. Question: Can you provide details on changes to the sickle cell trial protocol after the liver toxicity disclosure? - The company confirmed that monitoring for liver test enzymes was already in place and adjustments were made to the open-label portion of the trial to align monitoring frequency [70]. Question: How does the company plan to develop the tevapivat program in sickle cell disease? - Management indicated that they are looking to build a sickle cell disease franchise across both PYRUKYND and tevapivat, guided by data and the competitive environment [75][76]. Question: How should investors model the launch trajectory for mitapivat in thalassemia? - Management expects to capture patients gradually as they come to their doctors, emphasizing disease state education to facilitate treatment decisions [92][93]. Question: What is the commercial potential in the Gulf region? - The company sees significant opportunities in the Gulf, particularly in Saudi Arabia, and is working closely with local teams to navigate the regulatory process [99][100].

Q4 2024 Financial Results and Business Update February 13, 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 | | Third Quarter 2024 Financial Results | Cecilia Jones, Chief Financial O ...

Financial Performance - The company reported a net income of $673.7 million for the year ended December 31, 2024, primarily due to the sale of Vorasidenib Royalty Rights and receipt of the Vorasidenib Milestone Payment[443]. - The company incurred a net loss of $352.1 million for the year ended December 31, 2023, and a net loss of $231.8 million for the year ended December 31, 2022[443]. - Total revenue for 2024 was $36.5 million, an increase of $9.7 million (26.5%) compared to 2023, driven by increased volume of PYRUKYND®[491]. - Total operating expenses for 2024 were $462.2 million, up $43.9 million (10.5%) from 2023, primarily due to a $36.9 million increase in selling, general and administrative expenses[493]. - Research and development expenses for 2024 totaled $301.3 million, an increase of $5.8 million (2.0%) compared to 2023, with significant costs associated with PYRUKYND®[495]. - The gain on sale of contingent payments in 2024 was $889.1 million, attributed to the sale of Vorasidenib Royalty Rights[498]. - Cash, cash equivalents, and marketable securities balance was $1.5 billion at December 31, 2024[507]. - Net cash used in operating activities for 2024 was $389.8 million, an increase from $296.1 million in 2023[508]. - Cash used in operating activities for the year ended December 31, 2024, was $389.8 million, primarily due to operating expenses driven by research and development costs, partially offset by $43.5 million in interest income and $37.8 million in product revenues[509]. - Cash used in operating activities for the year ended December 31, 2023, was $296.1 million, with $31.2 million in interest income and $28.6 million in revenues partially offsetting the operating expenses[510]. - Cash provided by investing activities for the year ended December 31, 2024, was $363.4 million, mainly from the Upfront Payment from Royalty Pharma and the Vorasidenib Milestone Payment from Servier[512]. - Cash provided by financing activities for the year ended December 31, 2024, was $14.4 million from stock option exercises and employee stock purchase plan[515]. - As of December 31, 2024, the company had cash, cash equivalents, and marketable securities of $1.5 billion, up from $0.8 billion as of December 31, 2023[527]. - The company may need to raise additional capital through equity or debt financing, which could dilute existing stockholders' ownership[519]. Product Development and Approvals - PYRUKYND® is approved for treating hemolytic anemia in adults with PK deficiency and has been submitted for additional indications, with a PDUFA goal date of September 7, 2025[433]. - The company has made an upfront payment of $17.5 million to Alnylam for the AG-236 siRNA development candidate and may pay up to $130.0 million in potential development and regulatory milestones[435]. - The company completed the sale of its oncology business to Servier for approximately $1.8 billion in cash, with additional contingent payments based on future approvals[436]. - In August 2024, the FDA approved vorasidenib for adult and pediatric patients with Grade 2 astrocytoma or oligodendroglioma, leading to a recognized income of $200.0 million from the Vorasidenib Milestone Payment[440]. - PYRUKYND® is approved by the FDA for treating hemolytic anemia in adults with PK deficiency and has received marketing authorization in the EU and Great Britain[451]. - The company submitted regulatory applications for PYRUKYND® for thalassemia treatment in December 2024, with a PDUFA goal date of September 7, 2025[451]. - The company is evaluating PYRUKYND® in clinical trials for SCD and pediatric patients with PK deficiency[451]. - The company updated clinical trial protocols to include monthly monitoring of liver tests for the first six months of treatment due to potential hepatocellular injury risks[458]. - The phase 3 portion of the RISE UP trial has enrolled over 200 patients, with topline data expected in late 2025 and a potential U.S. commercial launch in 2026 if approved[460]. - The company initiated a phase 1 clinical trial of AG-181 in healthy volunteers in Q1 2024, following the IND filing in December 2023[467]. - The company expects to file an IND for AG-236 for the treatment of PV in mid-2025[467]. Clinical Trial Results - The ENERGIZE trial enrolled 194 patients, with 42.3% of the PYRUKYND® arm achieving a hemoglobin response compared to 1.6% in the placebo arm (p<0.0001)[454]. - In the ENERGIZE-T trial, 30.4% of patients on PYRUKYND® achieved a transfusion reduction response compared to 12.6% in the placebo arm (p=0.0003) with 90.1% experiencing any treatment-emergent adverse events[456]. - The phase 2 portion of the RISE UP study showed 46.2% and 50.0% of patients in the 50 mg and 100 mg PYRUKYND® arms achieved a hemoglobin response, respectively, compared to 3.7% in the placebo arm (p=0.0003 and 0.0001)[460]. - 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[461]. - In the ACTIVATE-kids study, 31.6% of patients in the mitapivat arm achieved a hemoglobin response, while 0% in the placebo arm, with a 95% confidence interval for the difference in response rates between mitapivat and placebo being 10.8% to 52.7%[462]. - The phase 2a trial of tebapivat in adults with low transfusion burden MDS showed that 40% of patients achieved transfusion independence[466]. - The company achieved clinical proof-of-concept in the phase 2a trial of tebapivat, with one patient achieving the hemoglobin response endpoint[466]. - The company has completed enrollment in the phase 2a trial of tebapivat with 22 patients, including 10 classified as low transfusion burden[465]. Future Outlook and Strategic Plans - The company expects to continue incurring significant expenses as it advances clinical development and commercialization activities for PYRUKYND® and other product candidates[443]. - The company anticipates generating future revenue from product sales, milestone payments, and royalties under potential collaborations or licensing agreements[445]. - Selling, general and administrative expenses are anticipated to increase to support ongoing research and commercialization activities, including hiring additional personnel[469]. - The company plans to finance operations through cash on hand, potential royalty payments, and strategic collaborations[503]. - The company is exposed to market risk related to changes in interest rates and foreign currency exchange rates, with minimal liabilities in foreign currencies as of December 31, 2024[528].

Exhibit 99.1 Agios Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights – Filed for Regulatory Approval of Mitapivat (PYRUKYND ) for the Treatment of Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia in U.S., European Union, Kingdom of Saudi Arabia and United Arab Emirates; PDUFA Goal Date of September 7, 2025 – ® – Completed Enrollment for Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease; Topline Results Expecte ...

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) Q3 2024 Earnings Conference Call October 31, 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 Greg Harrison - Scotiabank Alex Nackenoff - Raymond James Greg Renza - RBC Capital Markets Eric Schmidt - Ca ...

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 September 30, 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 o ...

Exhibit 99.1 Agios Reports Business Highlights and Third Quarter 2024 Financial Results – Completed Enrollment of the Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease; Topline Data from 52-Week Study Expected in Late 2025 – – Received $1.1 Billion in Payments from Royalty Pharma and Servier, Following FDA Approval of Vorasidenib – – Commenced Enrollment of the Phase 2b Study of Tebapivat in Lower-Risk Myelodysplastic Syndromes (LR-MDS); Granted FDA Orphan Drug Designation for Treatment of MDS – – P ...

Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) Q2 2024 Results Conference Call August 1, 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 Eric Schmidt - Cantor Christopher Raymond - Piper Sandler Gregory Renza - RBC Capital Markets Divya Rao - TD Co ...

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 June 30, 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) (State or Other Jurisdiction of Incorporation or Organi ...