Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase $(PK)$ activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. 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 the potential benefits of PYRUKYND$(R)$ (mitapivat), tebapivat (AG-946), AG-236 and AG-181, Agios' PAH stabilizer; Agios' plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND(R) , tebapivat, AG-236 and AG-181; Agios' use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios' strategic vision and goals, including its key milestones for 2025; 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 pandemics or other public health emergencies 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 establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios' cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios' public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Consolidated Balance Sheet Data
(in thousands)
(Unaudited)
December 31, December 31,
2024 2023
----------------------------------- -------------- --------------
Cash, cash equivalents, and marketable
securities $ 1,532,031 $ 806,363
Accounts receivable, net 4,109 2,810
Inventory 27,616 19,076
Total assets 1,663,199 937,118
Stockholders' equity 1,540,956 811,019
-------------------------------------- ---------- ----------
Consolidated Statements of Operations Data
(in thousands, except share and per share data)
(Unaudited)
Years Ended Dec 31,
------------------------------------------
2024 2023 2022
---------- ----------
Revenues:
Product revenue,
net $ 36,498 $ 26,823 $ 11,740
Milestone
revenue -- -- 2,500
-------------------- ---------- ---------- ----------
Total revenue 36,498 26,823 14,240
-------------------- ---------- ---------- ----------
Operating expenses
Cost of sales $ 4,165 $ 2,881 $ 1,704
Research and
development 301,286 295,526 279,910
Selling, general
and
administrative 156,784 119,903 121,673
-------------------- ---------- ---------- ----------
Total
operating
expenses 462,235 418,310 403,287
-------------------- ---------- ---------- ----------
Loss from
operations (425,737) (391,487) (389,047)
-------------------- ---------- ---------- ----------
Gain on sale of
contingent
payments 889,136 -- 127,853
Milestone
payment from
gain on sale of
oncology
business 200,000 -- --
Royalty income
from gain on
sale of
oncology
business -- -- 9,851
Interest income,
net 48,083 33,344 12,793
Other income,
net 6,487 6,055 6,749
-------------------- ---------- ---------- ----------
Net income
(loss) before
taxes 717,969 (352,088) (231,801)
Income tax
expense 44,244 -- --
-------------------- ---------- ---------- ----------
Net income
(loss) $ 673,725 $ (352,088) $ (231,801)
Net income (loss)
per share - basic $ 11.86 $ (6.33) $ (4.23)
Net income (loss)
per share -
diluted $ 11.64 $ (6.33) $ (4.23)
Weighted-average
number of common
shares used in
computing net
income (loss) per
share -- basic 56,807,415 55,651,487 54,789,435
Weighted-average
number of common
shares used in
computing net
income (loss) per
share -- diluted 57,889,255 55,651,487 54,789,435
Contacts:
Investor Contact
Chris Taylor, VP, Investor Relations and Corporate Communications
Agios Pharmaceuticals
IR@agios.com
Media Contact
Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
media@agios.com
(END) Dow Jones Newswires
February 13, 2025 06:31 ET (11:31 GMT)