Agios Reports Business Highlights and Second Quarter 2022 Financial Results
– Actively Enrolling Patients in Five PYRUKYND® Pivotal Studies Across Thalassemia, Sickle Cell Disease and Pediatric Pyruvate Kinase (PK) Deficiency –
“As we look ahead to the next chapter for the company, Agios is operating from a position of strength. We are executing our commercial launch of PYRUKYND®, the first therapy for a rare, debilitating, lifelong blood disorder; we also have five pivotal trials underway, multiple early-stage studies planned or ongoing, a promising preclinical pipeline and a strong balance sheet providing optionality for the future growth of the business,” said
Second Quarter 2022 & Recent Highlights
- Continued to execute
U.S.launch of PYRUKYND®, generating $3.1 millionin U.S.net revenue for the second quarter of 2022, the first full quarter following FDA approval.
- Initiated Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND® in pediatric patients with PK deficiency who are not regularly transfused and who are regularly transfused, respectively.
- Announced that effective
August 8, 2022, Dr. Fousewill transition to the role of chair of Agios’ board of directors and Brian Goffwill assume the role of Agios’ chief executive officer and member of the board of directors.
- Evolved Agios’ research approach to focus on advancing the company’s existing validated preclinical programs and in-licensing or acquiring well-characterized, high-potential assets.
- Expanded role of
Sarah Gheuens, M.D., Ph.D., to chief medical officer and head of research and development, incorporating research and discovery sciences in addition to her existing chief medical officer responsibilities.
- Presented clinical and translational data at the 2022
European Hematology Association (EHA) Congress, including new data supporting the potential benefits of PYRUKYND® treatment in adults with PK deficiency.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following key milestones and priorities in 2022:
- Adult PK Deficiency: Receive
European Medicines Agency(EMA) regulatory decision for PYRUKYND® in adults with PK deficiency by year-end.
- Thalassemia: Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassemia, respectively, by year-end.
- Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND® in sickle cell disease by year-end.
- Myelodysplastic Syndrome: Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk MDS by year-end.
- Data Presentations: Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease.
Second Quarter 2022 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.
Cost of Sales: Cost of sales for the second quarter of 2022 was
Non-Operating Income: Non-operating income included approximately
Research and Development (R&D) Expenses: R&D expenses were $74.5 million for the second quarter of 2022 compared to $62.0 million for the second quarter of 2021. The year-over-year increase in R&D was driven primarily by increased headcount and workforce-related expenses, planned increased activity associated with the PAH preclinical program, start-up costs for the AG-946 Phase 2a MDS study, increased spend for the AG-946 Phase 1 trial, and start-up costs for the PYRUKYND pivotal studies in sickle cell disease and pediatric PK deficiency.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $28.3 million for the second quarter of 2022 compared to $29.2 million for the second quarter of 2021. The year-over-year decrease in SG&A expenses was primarily attributable to the completion of the reimbursable transition services Agios provided to
Net Loss from Continuing Operations: Net loss from continuing operations was $91.8 million for the second quarter of 2022 compared to a net loss of $82.8 million for the second quarter of 2021.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2022, were $1.1 billion compared to $1.7 billion as of June 30, 2021. The year-over-year decrease is attributable to operating expenses and 5.7 million shares of common stock that the company repurchased for
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 2022 financial results and recent business activities. 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 a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for genetically defined diseases. In the
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 PYRUKYND® (mitapivat) and AG-946; the expected benefits of Agios’ chief executive officer succession plan; the potential benefits of Agios’ products and product candidates; Agios’ key milestones and guidance for 2022; its financial guidance regarding the period in which it will have capital available to fund its operations; 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 impact of the COVID-19 pandemic on Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of future approved products, and launching, marketing and selling future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the
|Consolidated Balance Sheet Data
|June 30, 2022||December 31,
|Cash, cash equivalents, and marketable securities|
|Accounts receivable, net||1,598||—|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended
||Six Months Ended
|Product revenue, net||$—||$—|
|Cost and expenses:|
|Cost of sales||$—||$—|
|Research and development||74,523||62,007||144,646||119,674|
|Selling, general and administrative||28,264||29,215||59,779||62,765|
|Total cost and expenses||103,222||91,222||205,199||182,439|
|Loss from operations||(97,640)||(91,222)||(198,785)||(182,439)|
|Royalty income from gain on sale of oncology business||2,704||2,000||5,408||2,000|
|Interest income (expense), net||1,793||(92)||2,487||248|
|Other income, net||1,337||6,524||4,310||6,524|
|Net loss from continuing operations||(91,806)||(82,790)||(186,580)||(173,667)|
|Net (loss) income from discontinued operations, net of tax||—||(3,427)||—||1,961,775|
|Net (loss) income|
|Net loss from continuing operations per share - basic and diluted|
|Net (loss) income from discontinued operations per share - basic and diluted||$—||$—|
|Net (loss) income per share - basic and diluted|
|Weighted-average number of common shares used in computing net loss per share from continuing operations, net (loss) income from discontinued operations and net (loss) income per share – basic and diluted||54,799,680||61,066,977||54,678,249||65,281,827|
Senior Director, Investor Relations
Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.