On July 29, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, reported business highlights and financial results for the second quarter ended June 30, 2021 (Press release, Agios Pharmaceuticals, JUL 29, 2021, View Source [SID1234585385]).
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"Our first quarter as a company solely focused on genetically defined diseases was marked by several significant milestones, most notably our U.S. and EU regulatory submissions for mitapivat for the treatment of adults with PK deficiency, bringing us one step closer to potentially delivering the first disease-modifying treatment for people with this serious and underserved condition," said Jackie Fouse, Ph.D., chief executive officer at Agios. "We continue to unlock the potential of mitapivat for people with other chronic hemolytic anemias and are gaining momentum on our pivotal programs in thalassemia and sickle cell disease. This year, we look forward to initiating our two registrational Phase 3 trials – ENERGIZE and ENERGIZE-T – in not regularly transfused and regularly transfused adults with thalassemia, as well as our pivotal Phase 2/3 trial of mitapivat in sickle cell disease."
SECOND QUARTER 2021 & RECENT HIGHLIGHTS
Completed two regulatory filings for approval of mitapivat in adults with PK deficiency; submitted new drug application (NDA) in the U.S. and marketing authorization application (MAA) in the EU.
Presented full analysis of data from Phase 3 ACTIVATE and ACTIVATE-T studies of mitapivat in adults with PK deficiency at European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress; studies met primary and secondary endpoints, including patient-reported outcome (PRO) measures that address symptom burden and quality-of-life impact in adults.
Presented data from Phase 2, open-label, multicenter study of mitapivat in adults with non-transfusion dependent α- or β-thalassemia at EHA (Free EHA Whitepaper) Virtual Congress; study met its primary endpoint, with 16 of the 20 patients (80%) achieving a hemoglobin increase of ≥1.0 g/dL from baseline at one or more assessments during Weeks 4-12.
Launched myAgios patient support services for people living with PK deficiency and their caregivers, providing tailored support, educational resources and opportunities to connect with other patients and caregivers in the community.
Completed hiring and training of customer-facing and patient support team that will support the U.S. launch of mitapivat in PK deficiency upon product approval.
Announced succession plan for Chris Bowden, M.D., who will transition from his role as chief medical officer to strategic advisor following his retirement on Sept. 1, at which time Sarah Gheuens, M.D., Ph.D., vice president of clinical development, will assume the role.
Repurchased approximately 10.5 million shares of Agios common stock, inclusive of shares acquired from Bristol-Myers Squibb Company (BMS) and its affiliates, at an average price of $50.41 per share. This accounts for $529 million of the up to $1.2 billion of share repurchases authorized by the Board of Directors following the company’s sale of its oncology business to Servier.
KEY UPCOMING MILESTONES
Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia, by year-end.
Initiate Phase 2/3 study of mitapivat in sickle cell disease by year-end.
Host investor day in fourth quarter to share more information about commercial launch planning for mitapivat in PK deficiency and research and development pipeline.
Data Presentations
Submit data from ongoing collaborator-led clinical studies of mitapivat in sickle cell disease for presentation at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition, to be held Dec. 11-14.
Submit data from the Phase 1 healthy volunteer study of AG-946, the company’s next-generation PKR activator, for presentation at the 63rd ASH (Free ASH Whitepaper) Annual Meeting & Exposition.
SECOND QUARTER 2021 FINANCIAL RESULTS
The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.
Research and Development (R&D) Expenses: R&D expenses for continuing operations were $62.0 million for the second quarter of 2021 compared to $54.1 million for the second quarter of 2020. The year-over-year increase in R&D was driven primarily by start-up costs associated with the Phase 3 studies of mitapivat in thalassemia and sickle cell disease, as well as the NDA and MAA filings for mitapivat in PK deficiency and launch preparation activities.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses for continuing operations were $29.2 million for both the second quarter of 2021 and the second quarter of 2020.
Non-Operating Income: Non-operating income included approximately $2.0 million from TIBSOVO (ivosidenib) royalties for the second quarter of 2021.
Net Loss: Net loss was $86.2 million for the second quarter of 2021 compared to a net loss of $90.5 million for the second quarter of 2020.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2021, were $1.7 billion compared to $794.4 million as of June 30, 2020. The company expects that its cash, cash equivalents and marketable securities as of June 30, 2021 will enable the company to execute its operating plan through major catalysts and to cash-flow positivity without the need to raise additional equity.
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2021 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 2663508. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.