Agios Reports Fourth Quarter and Full Year 2022 Financial Results
– Completed Enrollment in Phase 2 RISE UP Study of PYRUKYND® (mitapivat) in Sickle Cell Disease –
– On Track to Complete Enrollment in Phase 3 ENERGIZE and ENERGIZE-T Studies of PYRUKYND® in Thalassemia by Mid-2023 –
– U.S. PYRUKYND® Launch Provides Capability Building Platform to Support Expected Future Product Growth and Expansion; Net Revenue of $4.3 Million in Q4 –
“Over the past year, Agios has made significant progress toward our vision of transforming the lives of patients with rare diseases as we build a hematology franchise focused on diseases that share a common underlying pathophysiology, limited treatment options and profound unmet need,” said
Fourth Quarter 2022 & Recent Highlights
U.S.Launch: Continued to execute launch, generating $4.3 millionin U.S.net revenue for the fourth quarter of 2022, the third full quarter following FDA approval. A total of 105 unique patients have completed prescription enrollment forms, representing an increase of 25 percent over the third quarter. A total of 78 patients are on PYRUKYND® therapy, representing a 39 percent increase over the third quarter.
- PYRUKYND® Global Approvals: Received marketing authorization for adults with PK deficiency in the EU and Great Britain.
- Sickle Cell Disease: Completed enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND® in adults with sickle cell disease.
- Thalassemia: Enrolled more than half of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassemia, respectively.
- Data Presentations: Presented broad set of clinical and translational data at the 64th American Society of Hematology (ASH) Annual Meeting & Exposition, including long-term PYRUKYND® data in adults with non-transfusion-dependent thalassemia and in adults with PK deficiency.
- Leadership: Appointed Tsveta Milanova to the role of chief commercial officer, bringing two decades of experience in rare disease commercial strategy and global market access.
Anticipated 2023 Milestones
- Thalassemia: Complete enrollment of the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® by mid-year.
- Sickle Cell Disease: Announce data readout from the Phase 2 portion of the RISE UP study of PYRUKYND® and go/no-go to Phase 3 decision by mid-year.
- Pediatric PK Deficiency: Enroll more than half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of PYRUKYND® by year-end.
- Lower-risk Myelodysplastic Syndromes (LR-MDS): Complete enrollment of the Phase 2a study of novel PK activator AG-946 by year-end.
- Pipeline: File investigational new drug (IND) application for phenylalanine hydroxylase (PAH) stabilizer for the treatment of phenylketonuria (PKU) by year-end.
Fourth 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 was
Non-Operating Income: Non-operating income included
Research and Development (R&D) Expenses: R&D expenses were $70.3 million for the fourth quarter of 2022 compared to $73.3 million for the fourth quarter of 2021, and
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were
Net Income (Loss) from Continuing Operations: Net income was $36.5 million for the fourth quarter of 2022 compared to a net loss of $98.6 million for the fourth quarter of 2021, and net loss was
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of Dec. 31, 2022, were $1.1 billion compared to $1.3 billion as of Dec. 31, 2021. This cash position includes the receipt of a one-time payment of
Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss fourth quarter and year end 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 rare 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 the potential benefits of PYRUKYND® (mitapivat), AG-946 and its PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, AG-946 and its PAH stabilizer; Agios’ strategic vision and goals, including its key milestones for 2023 and potential catalysts through 2026; 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. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. 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 to 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 current or future approved products, and launching, marketing and selling current or 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 U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to maintain key collaborations; 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||$||1,096,993||$||1,286,393|
|Accounts receivable, net||2,206||—|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Product revenue, net||$||11,740||$||—||$||—|
|Cost of sales||$||1,704||$||—||$||—|
|Research and development||279,910||256,973||220,811|
|Selling, general and administrative||121,673||121,445||115,105|
|Total operating expenses||403,287||378,418||335,916|
|Loss from operations||(389,047||)||(378,418||)||(335,916||)|
|Gain on sale of contingent payments||127,853||—||—|
|Royalty income from gain on sale of oncology business||9,851||6,639||—|
|Interest income, net||12,793||836||6,611|
|Other income, net||6,749||14,433||—|
|Net loss from continuing operations||(231,801||)||(356,510||)||(329,305||)|
|Net income from discontinued operations, net of tax||—||1,961,225||1,935|
|Net (loss) income||$||(231,801||)||$||1,604,715||$||(327,370||)|
|Net loss from continuing operations per share - basic and diluted||$||(4.23||)||$||(5.90||)||$||(4.77||)|
|Net income from discontinued operations per share - basic and diluted||$||—||$||32.45||$||0.03|
|Net (loss) income per share - basic and diluted||$||(4.23||)||$||26.55||$||(4.74||)|
|Weighted-average number of common shares used in computing net loss per share from continuing operations, net income per share from discontinued operations and net (loss) income per share – basic and diluted||54,789,435||60,447,346||68,997,879|
Investor & Media Contact
Senior Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.