Agios Reports Business Highlights and Second Quarter 2021 Financial Results
– Completed Regulatory Submissions for Mitapivat for the Treatment of Adults with Pyruvate Kinase (PK) Deficiency in the
– Presented Data from ACTIVATE and ACTIVATE-T Phase 3 Studies and Thalassemia Phase 2 Study at
“Our first quarter as a company solely focused on genetically defined diseases was marked by several significant milestones, most notably our
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) 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 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
$50.41per share. This accounts for $529 millionof the up to $1.2 billionof 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.
- Submit data from ongoing collaborator-led clinical studies of mitapivat in sickle cell disease for presentation at the 63rd
American Society of Hematology(ASH) 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 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
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.
Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. The company’s most advanced drug candidate is a first-in-class pyruvate kinase R (PKR) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias. In addition to its active late-stage clinical pipeline, Agios has multiple novel, investigational therapies in clinical and preclinical development. For more information, please visit the company’s website at www.agios.com.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Agios’ plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs, including mitapivat and AG-946; the potential benefits of Agios’ products and product candidates; Agios’ key milestones and guidance for 2021; its financial guidance regarding the period in which it will have capital available to fund its operations; expectations regarding the return of capital to shareholders following the sale of Agios’ oncology business; its chief medical officer transition plan and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation risks and uncertainties related to: the failure of Agios to receive milestone or royalty payments related to the sale of its oncology business, the uncertainty of the timing of any receipt of any such payments, and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with
|Consolidated Balance Sheet Data|
|Cash, cash equivalents and marketable securities||$||1,734,301||$||670,537|
|Assets held for discontinued operations||-||50,460|
|Liabilities held for discontinued operations||-||299,728|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
||Six Months Ended
|Cost and expenses:|
|Research and development||$||62,007||$||54,086||$||119,674||$||109,445|
|Selling, general and administrative||$||29,215||$||29,178||$||62,765||$||60,849|
|Total cost and expenses||$||91,222||$||83,264||$||182,439||$||170,294|
|Loss from operations||$||(91,222||)||$||(83,264||)||$||(182,439||)||$||(170,294||)|
|Gain on sale of oncology business||$||2,000||$||-||$||2,000||$||-|
|Interest (expense) income, net||$||(92||)||$||1,769||$||248||$||4,705|
|Other income, net||$||6,524||$||-||$||6,524||$||-|
|Net loss from continuing operations||$||(82,790||)||$||(81,495||)||$||(173,667||)||$||(165,589||)|
|Net income (loss) from discontinued operations, net of tax||$||(3,427||)||$||(8,983||)||$||1,961,775||$||34,855|
|Net income (loss)||$||(86,217||)||$||(90,478||)||$||1,788,108||$||(130,734||)|
|Net loss from continuing operations per share - basic and diluted||$||(1.36||)||$||(1.18||)||$||(2.66||)||$||(2.41||)|
|Net income (loss) from discontinued operations per share - basic and diluted||$||(0.06||)||$||(0.13||)||$||30.05||$||0.51|
|Net income (loss) per share - basic and diluted||$||(1.41||)||$||(1.31||)||$||27.39||$||(1.90||)|
|Weighted-average number of common shares used in computing net income (loss) per share – basic and diluted||61,066,977||68,958,091||65,281,827||68,784,109|
Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.