Agios Presents Updated PYRUKYND® (mitapivat) Long-term Extension Data Demonstrating Sustained Clinical Benefits in Adults with Pyruvate Kinase (PK) Deficiency at 64th ASH Annual Meeting and Exposition
– Data Suggest Long-term Treatment with PYRUKYND® in Adults with PK Deficiency is Associated with Improvements in Hemoglobin, Iron Overload, Transfusion Burden and Patient-reported Outcomes, Regardless of Transfusion Status –
– Additional Data Presented at ASH Characterize Disease Complications and Co-morbidities of PK Deficiency in Pediatric Populations, Supporting Ongoing Pivotal ACTIVATE-Kids and ACTIVATE-KidsT Studies of PYRUKYND® –
– Agios to Host Live and Webcast Investor Event on
Long-term extension data (abstract # 2328) show that previously reported effects of PYRUKYND® on hemoglobin and transfusion burden were maintained over time. As of the
“PYRUKYND® is the first oral agent that has the potential to improve symptoms and long-term complications of PK deficiency in adult patients,” said
“Collectively, the data we have presented at ASH continue to demonstrate the benefits of long-term treatment with PYRUKYND® for adults with PK deficiency, including improvements in hemoglobin, transfusion burden, iron overload and patient-reported outcomes,” said
Agios also presented data at ASH further supporting the potential of PYRUKYND® to address hallmark symptoms and complications of PK deficiency. More details on the presentations are provided below and on the ASH 2022 page on Agios.com.
Long-term Improvements in Patient-reported Outcomes in Patients with Pyruvate Kinase Deficiency Treated with Mitapivat (Abstract #506)
In an oral presentation, data from ACTIVATE, ACTIVATE-T and the long-term extension study were reported, showing that treatment with PYRUKYND® was associated with long-term, durable and clinically meaningful improvements in signs, symptoms and functional impacts, irrespective of transfusion status. Patient-reported outcome (PRO) improvements among patients treated with PYRUKYND® were sustained over time in the long-term extension (LTE) study through Week 84. At Week 84 of the LTE study, clinically meaningful improvements in PROs mean scores were achieved in more than half of patients. These results suggest that by improving health-related quality of life, treatment with PYRUKYND® may provide meaningful patient-centric benefits.
Mitapivat Improves Iron Overload in Patients with Pyruvate Kinase Deficiency (Abstract #1021)
In a poster presentation, data from ACTIVATE and the long-term extension study were reported that showed meaningful long-term improvements in key systemic regulators of iron homeostasis and measures of iron overload – including erythroferrone, soluble transferrin receptor (sTfR) and hepcidin – continued up to 96 weeks in patients treated with PYRUKYND®. Additionally, patients treated with PYRUKYND® who had evidence of iron overload at baseline showed clinically meaningful and continued improvements in iron overload over time as measured by liver iron concentration (median [Q1, Q3] decrease from baseline to Week 96 of PYRUKYND® treatment of –1.95 [–4.85, –0.70] mg Fe/g dw). Ferritin levels remained stable across both patient groups treated with PYRUKYND® or placebo.
Mitapivat Improves Markers of Hemolysis and Erythropoiesis in Patients with Pyruvate Kinase Deficiency Irrespective of Hemoglobin Response (Abstract #3644)
In a separate poster presentation, data from the ACTIVATE study were reported showing that treatment with PYRUKYND® improved markers of hemolysis and ineffective erythropoiesis in adults with PK deficiency. The analysis also shows that directional improvements occur even in patients who did not achieve the clinical trial definition of hemoglobin response.
PYRUKYND® was approved in February 2022 by the U.S. Food and Drug Administration (FDA) and received marketing authorization in
Conference Call Information
Agios will host a live investor event on Dec. 12, 2022, at 7:00 a.m. CT in New Orleans to review the key clinical oral and poster presentations from this year’s ASH meeting. The event will be webcast live and can be accessed under “Events & Presentations” in the Investors and Media 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.
About PK Deficiency
Pyruvate kinase (PK) deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells. The inherited mutation in the PKLR gene can cause a deficit in energy within the red blood cell, as evidenced by lower PK enzyme activity, a decline in adenosine triphosphate (ATP) levels and a build-up of upstream metabolites, including 2,3-DPG (2,3-diphosphoglycerate).
PK deficiency is associated with serious complications, including gallstones, pulmonary hypertension, extramedullary hematopoiesis, osteoporosis and iron overload and its sequelae, which can occur regardless of the degree of anemia or transfusion burden. PK deficiency can also cause quality of life problems, including challenges with work and school activities, social life and emotional health. Current management strategies for PK deficiency, including red blood cell transfusions and splenectomy, are associated with both short- and long-term risks. For more information, please visit www.knowpkdeficiency.com.
About PYRUKYND® (mitapivat)
PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the
IMPORTANT SAFETY INFORMATION
Acute 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.
Adverse Reactions: Serious adverse reactions occurred in 10% of patients receiving PYRUKYND in the ACTIVATE trial, including atrial fibrillation, gastroenteritis, rib fracture, and musculoskeletal pain, each of which occurred in 1 patient. In the ACTIVATE trial, 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.
Drug Interactions:
- Strong CYP3A Inhibitors and Inducers: Avoid concomitant use.
- Moderate CYP3A Inhibitors: Do not titrate PYRUKYND beyond 20 mg twice daily.
- Moderate CYP3A Inducers: Consider alternatives that are not moderate inducers. If there are no alternatives, adjust PYRUKYND dosage.
- Sensitive CYP3A, CYP2B6, CYP2C Substrates Including Hormonal Contraceptives: Avoid concomitant use with substrates that have narrow therapeutic index.
- UGT1A1 Substrates: Avoid concomitant use with substrates that have narrow therapeutic index.
- P-gp Substrates: Avoid concomitant use with substrates that have narrow therapeutic index.
Hepatic Impairment: Avoid use of PYRUKYND in patients with moderate and severe hepatic impairment.
Please see full Prescribing Information and Summary of Product Characteristics for PYRUKYND.
About Agios
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); Agios’ plans regarding future data presentations; and the potential benefit of its strategic plans and focus. The words “anticipate,” “expect,” “intend,” “potential,” “milestone,” “goal,” “will,” “on track,” “upcoming,” 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 or its collaborators 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. Moreover, there can be no guarantee that any medicines ultimately commercialized by Agios will receive commercial acceptance. 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
Investor and Media Contact:
Senior Director, Corporate Communications
Jessica.Rennekamp@agios.com
![](https://ml.globenewswire.com/media/NGYzNDE0NTgtNzJjMy00MjY5LWE2ZTQtZDQyYjNiMGI4MjJjLTEwMTk3OTE=/tiny/Agios-Pharmaceuticals-Inc-.png)
Source: Agios Pharmaceuticals, Inc.