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].

Agios Pharmaceuticals (AGIO) came out with a quarterly loss of $1.74 per share versus the Zacks Consensus Estimate of a loss of $1.73. This compares to loss of $1.72 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -0.58%. A quarter ago, it was expected that this biopharmaceutical company would post earnings of $16.69 per share when it actually produced earnings of $4.20, delivering a surprise of -74.84%.Over the last four quar ...

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 ...

– 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 Expected in Late 2025, with Potential U.S. Commercial Launch in 2026 – – Early- and Mid-Stage Pipeline Offers Strong Fo ...

– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety Profile for Mitapivat Previously Observed in Adults with PK Deficiency Who Are Not Regularly Transfused – – First Mitapivat Pediatric Clinical Program for a Rare Hemolytic Anemia; Double-blind Period Completed for Both PK Deficiency Trials ACTIVATE-Kids and ACTIVATE-KidsT – CAMBRIDGE, Mass., Feb. 13, 2025 (GLOBE NEWSWIRE) -- ...

– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety Profile for Mitapivat Previously Observed in Adults with PK Deficiency Who Are Not Regularly Transfused – – First Mitapivat Pediatric Clinical Program for a Rare Hemolytic Anemia; Double-blind Period Completed for Both PK Deficiency Trials ACTIVATE-Kids and ACTIVATE-KidsT – CAMBRIDGE, Mass., Feb. 13, 2025 (GLOBE NEWSWIRE) -- ...

CAMBRIDGE, Mass., Feb. 06, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced the company will host a conference call and live webcast on Thursday, February 13, 2025, at 8:00 a.m. ET to report its fourth quarter and full year 2024 financial results and business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios ...

– FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia; PDUFA Goal Date is September 7, 2025 – – Topline Results from Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease to be Announced in Late 2025, with Potential U.S. Commercial Launch in 2026 – – Strong Financial Position Provides Opportunity to Maximize Potential PYRUKYND Commercial Launches, Advance Early- and Mid-Stage ...

Agios Pharmaceuticals (AGIO) announced that the FDA has accepted its supplemental new drug application (sNDA) seeking label expansion for its sole marketed drug, Pyrukynd, in thalassemia indication.A final decision is expected by Sep. 7, 2025. If approved, Pyrukynd will become the first oral therapy for use in all thalassemia subtypes. A commercial launch in this indication is expected before this year’s end.The sNDA is supported by data from two phase III studies, ENERGIZE and ENERGIZE-T, which evaluated P ...