Agios Pharmaceuticals (AGIO) shares rallied 5.2% in the last trading session to close at $24.02. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 1.3% gain over the past four weeks.Share price rose after management reported encouraging results from a late-stage study, evaluating orally-administered mitapivat in non-transfusion-dependent (NTD) thalassemia patients. Patients treated with mitapivat achieved demon ...

–Mitapivat Demonstrated a Statistically Significant Increase in Hemoglobin Response Rate Compared to Placebo – – Statistical Significance Also Achieved for Key Secondary Endpoints of Change From Baseline in Both FACIT-Fatigue Score and Hemoglobin Concentration – – ENERGIZE is the First Phase 3 Study to Demonstrate Efficacy of an Oral Treatment for Non-Transfusion-Dependent Alpha- and Beta-Thalassemia – – Agios to Host Investor Webcast Event Today at 8:00 a.m. ET– CAMBRIDGE, Mass., Jan. 03, 2024 (GLOBE NEWSW ...

Agios Pharmaceuticals Inc.’s stock AGIO, +2.51% soared 19.6% early Wednesday, after the biotech announced positive results from a Phase 3 trial of a treatment for a rare blood disorder. The trial dubbed Energize sought to evaluate the company’s mitapivat therapy and met its primary endpoint of hemoglobin response in patients with non-transfusion-dependent (NTD) alpha- or beta-thalassemia. The trial also met secondary endpoints of change from baseline in FACIT-Fatigue Score and hemoglobin concentration. The ...

CAMBRIDGE, Mass., Jan. 02, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that the company is scheduled to present at the 42nd Annual J.P. Morgan Healthcare Conference on Wednesday, January 10, 2024, at 7:30 a.m. PT. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. A replay of the webcast ...

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].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 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, 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) (State or Other Jurisdiction of Incorporation or Organiza ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 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, 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) (State or Other Jurisdiction of Incorporation or Organiz ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 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, 2022 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 incorpora ...