Full Press Release Details
Agios Announces Key 2025 Milestones for Innovative Rare Disease Portfolio
FDA Accepted Agios Supplemental New Drug Application for PYRUKYND
(mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia; PDUFA Goal Date is September 7, 2025
Topline Results from Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease to be Announced in Late 2025, with Potential U.S.
Commercial Launch in 2026
Strong Financial Position Provides Opportunity to Maximize Potential PYRUKYND
Commercial Launches, Advance Early- and Mid-Stage Clinical Programs and Expand Pipeline
Mass., Jan. 13, 2025 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced its anticipated key 2025 milestones and
value-driving catalysts through 2026. The company s management team will present this information at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday,
January 15, 2025, at 7:30 a.m. PT / 10:30 a.m. ET.
2024 was marked by exceptional progress at Agios. We delivered on all our key
priorities, advanced our potential best- and first-in-class rare disease pipeline and further strengthened our financial position. Today, we are entering an era of
growth and expansion for the company, building on a strong foundation and focus, and are well-positioned for a sustained trajectory of success, said Brian Goff, chief executive officer at Agios. Our blueprint encompasses the
potential for two additional commercial launches of PYRUKYND in thalassemia and sickle cell disease in 2025 and 2026, respectively, along with an early- and mid-stage pipeline that offers a strong foundation
for innovation and growth, all supported by a highly experienced team with proven executional excellence and a strong balance sheet. Over the next 12 months, our priorities will be to maximize the potential of the PYRUKYND franchise, advance and
diversify our key pipeline programs, and strategically focus our capital deployment to sustain our growth. We are excited about the future and the meaningful impact we can have in addressing the critical needs of rare disease patients.
Anticipated 2025 Milestones:
Presentation at 43rd Annual J.P. Morgan Healthcare Conference
Agios management team will present at the 43rd Annual J.P. Morgan Healthcare Conference on
Wednesday, January 15, 2025, at 7:30 a.m. PT / 10:30 a.m. ET. The live webcast will be accessible on the Investors section of the company s website (www.agios.com) under the Events & Presentations tab. A
replay of the webcast will be archived on the company s website for at least two weeks following the presentation.
About PYRUKYND (mitapivat)
PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
U.S. IMPORTANT SAFETY INFORMATION
Hemolysis: Acute hemolysis with subsequent anemia has been observed following abrupt interruption or discontinuation of PYRUKYND in a dose-ranging study. Avoid abruptly discontinuing PYRUKYND. Gradually taper the dose of PYRUKYND to discontinue
treatment if possible. When discontinuing treatment, monitor patients for signs of acute hemolysis and anemia including jaundice, scleral icterus, dark urine, dizziness, confusion, fatigue, or shortness of breath.
Hepatocellular Injury in Another Condition: In patients with another condition treated with PYRUKYND at a higher dose than that recommended for
patients with PK deficiency, liver injury has been observed. These events were characterized by a time to onset within the first 6 months of treatment with peak elevations of alanine aminotransferase of >5 upper limit of normal (ULN) with
or without jaundice. All patients discontinued treatment with PYRUKYND, and these events improved upon treatment discontinuation.
prior to the initiation of PYRUKYND and monthly thereafter for the first 6 months and as clinically indicated. Interrupt PYRUKYND if clinically significant increases in liver tests are observed or alanine aminotransferase is >5x ULN. Discontinue
PYRUKYND if hepatic injury due to PYRUKYND is suspected.
Adverse Reactions: The most common adverse reactions including laboratory abnormalities
( 10%) in patients with PK deficiency were estrone decreased (males), increased urate, back pain, estradiol decreased (males), and arthralgia.
Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment.
Please see full Prescribing Information for PYRUKYND.
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
syndrome (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 (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 , tebapivat, AG-181 and AG-236; the submission of PYRUKYND
to regulators for approval in alpha-and-beta thalassemia; 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 , opportunity , 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
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.
Chris Taylor, VP, Investor Relations and Corporate Communications
Agios Pharmaceuticals
Eamonn Nolan, Senior Director, Corporate
Agios Pharmaceuticals