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)