AGIO Agios Pharmaceuticals Inc.

31
-0.52  -2%
Previous Close 31.52
Open 31.59
52 Week Low 28.96
52 Week High 62.155
Market Cap $1,683,562,694
Shares 54,308,474
Float 31,072,673
Enterprise Value $525,873,134
Volume 438,295
Av. Daily Volume 659,274
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Upcoming Catalysts

Drug Stage Catalyst Date
Mitapivat (AG-348)
Pyruvate kinase deficiency
PDUFA priority review
PDUFA priority review
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Drug Pipeline

Drug Stage Notes
AG-946
Sickle cell disease
Phase 1
Phase 1
Phase 1 study expected to initiate 1H 2022, noted January 10, 2021.
Mitapivat (AG-348) - (RISE UP)
Sickle cell disease
Phase 1
Phase 1
Phase 1 primary and secondary endpoints were met with no AEs led to drug discontinuation, (9/16) patients (56.3%) achieved a hemoglobin response, noted December 13, 2021. Phase 2/3 trial initiated, phase 2 portion of study enrollment to be completed by year end, noted January 10, 2021.
AG-946
Myelodysplastic syndrome (MDS)
Phase 2a
Phase 2a
Phase 1 data reported 6 early discontinuations unrelated to study treatment. 4 of 39 (10.3%) subjects experienced ≥ 1 treatment-emergent AE (TEAE); all TEAEs were assessed as mild (Grade [Gr] 1). In the multiple ascending oral doses 4 of 17 (23.5%) subjects experienced ≥ 1 TEAE; the majority of the TEAEs were mild (Gr 1), with 1 subject experiencing a serious AE (Gr 2) of exercise-induced rhabdomyolysis 14 days after last dose, noted December 13, 2021. Phase 2a study in adults with low- to intermediate-risk myelodysplastic syndrome (MDS) to initiate by year-end, noted January 10, 2021.
Mitapivat (ENERGIZE)
Thalassemia
Phase 3
Phase 3
Phase 3 trial to be initiated, noted November 3, 2021. Phase 3 enrollment ongoing until the end of the year, noted January 10, 2022.
Mitapivat (AG-348) - (ACTIVATE)
Pyruvate Kinase Deficiency
Phase 3
Phase 3
Phase 3 long-term extension study showed that treatment improved markers of ineffective erythropoiesis and iron metabolism in adults with PK deficiency, regardless of transfusion status, noted December 14, 2021.
Mitapivat (AG-348) - ESTIMATE open label
Sickle Cell Disease
Phase 2
Phase 2
Phase 2 data demonstrated the potential to provide clinically meaningful outcomes for patients, including improvements in anemia, hemolysis and red blood cell sickling. No SAEs occurred, and all AEs were mild and mostly transient. One vaso-occlusive crisis (VOC) occurred without hospital admission and did not require dose reduction or discontinuation, noted December 13, 2021.
TIBSOVO (ivosidenib) - (ClarIDHy)
IDH1 mutant cholangiocarcinoma - cancer
sNDA Filing
sNDA Filing
sNDA filing announced March 1, 2021.
TIBSOVO (ivosidenib)
Frontline AML with IDH1 mutation
Approved
Approved
FDA Approval announced May 2, 2019.
TIBSOVO (ivosidenib)
IDH1m Relapsed/Refractory AML - cancer
Approved
Approved
FDA Approval announced July 20, 2018.
AG-120 and VIDAZA - AGILE
Frontline Acute myeloid leukemia (AML) harboring an IDH1 mutation - cancer
Phase 3
Phase 3
Phase 3 enrolment to be completed 2021.
AG-270
Solid tumors
Phase 1
Phase 1
Phase 1 enrolment has slowed due to COVID-19. A go/no-go decision is expected no later than 2022.
Vorasidenib (AG-881)
IDHm low-grade glioma
Phase 1
Phase 1
Phase 1 presentation at ASCO May 29, 2020. Median Progression-free Survival of 31.4 months.
Vorasidenib (AG-881)
Glioma
Phase 3
Phase 3
Phase 3 enrolment has slowed as a result of COVID-19.
IDHIFA (enasidenib) - AG-221
Advanced hematologic malignancies with an IDH2 mutation
Approved
Approved
Approval announced August 1, 2017.

Latest News

  1. – Company Expects to Receive FDA Regulatory Decision for Mitapivat as a Potential Treatment for Adults with PK Deficiency in February –

    – Five Pivotal Clinical Trials are Planned and Ongoing in Thalassemia, Sickle Cell Disease and Pediatric PK Deficiency –

    – Agios is Expanding Clinical Portfolio with Trials of Novel PK Activator AG-946 –

    – Strong Cash Position Expected to Enable Execution of Robust Operating Plan to Cash-Flow Positivity –

    CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, today announced its anticipated 2022 key milestones and priorities that will drive its transition to a commercial-stage…

    – Company Expects to Receive FDA Regulatory Decision for Mitapivat as a Potential Treatment for Adults with PK Deficiency in February –

    – Five Pivotal Clinical Trials are Planned and Ongoing in Thalassemia, Sickle Cell Disease and Pediatric PK Deficiency –

    – Agios is Expanding Clinical Portfolio with Trials of Novel PK Activator AG-946 –

    – Strong Cash Position Expected to Enable Execution of Robust Operating Plan to Cash-Flow Positivity –

    CAMBRIDGE, Mass., Jan. 10, 2022 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, today announced its anticipated 2022 key milestones and priorities that will drive its transition to a commercial-stage genetically defined disease company and the expansion of its robust clinical and research portfolio. Agios will present at the virtual 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12 at 7:30 a.m. ET, and a live webcast will be available at investor.agios.com.

    "We are beginning an exciting and defining year for Agios as we prepare for our first genetically defined disease product launch, execute on five pivotal clinical trials, continue to expand our PK activation clinical portfolio and foster our innovative research engine," said Jackie Fouse, Ph.D., chief executive officer of Agios. "We are already making strong progress with mitapivat being evaluated under FDA Priority Review in the U.S. as a potential treatment for adults with PK deficiency and executing our Phase 3 ENERGIZE and ENERGIZE-T studies in thalassemia and Phase 2/3 RISE UP study in sickle cell disease. In 2022, we expect to further drive our PK activation expansion strategy by initiating our pivotal trials in pediatric PK deficiency, completing enrollment in the Phase 2 portion of the RISE UP study and advancing our novel PK activator AG-946 in sickle cell disease and low to intermediate risk myelodysplastic syndrome. At Agios, we are fueled by connections – with patient communities, a world-class network of healthcare providers and researchers, and each other – and these collaborative partnerships will drive our key priorities and advance care for people with genetically defined diseases."

    Anticipated 2022 Key Milestones & Priorities

    Agios expects to execute on the following key milestones and priorities in 2022:

    Pyruvate Kinase (PK) Deficiency

    • Receive U.S. Food and Drug Administration (FDA) regulatory decision for mitapivat in adults with PK deficiency in Q1
    • Receive European Medicines Agency (EMA) regulatory decision for mitapivat in adults with PK deficiency by year-end
    • Initiate Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies of mitapivat in not regularly transfused and regularly transfused pediatric patients with PK deficiency, respectively, in mid-2022

    Thalassemia

    • Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of mitapivat in not regularly transfused and regularly transfused adults with thalassemia, respectively, by year-end

    Sickle Cell Disease

    • Complete enrollment in the Phase 2 portion of the RISE UP study of mitapivat in sickle cell disease by year-end
    • Initiate the sickle cell disease cohort of the Phase 1 study of novel PK activator AG-946 in the first half of 2022

    Expansion and Acceleration of PK Activation Portfolio

    • Initiate Phase 2a study of AG-946 in adults with low- to intermediate-risk myelodysplastic syndrome (MDS) by year-end
    • Continue to publish clinical and translational data supporting the utility of PK activators across key disease areas and elucidating the burden of disease for PK deficiency, thalassemia and sickle cell disease

    Agios Five-Year Strategic Vision

    The Agios five-year strategic vision reflects the company's expected evolution as a leader in developing genetically defined disease therapies based on its scientific expertise in cellular metabolism. As part of this vision, Agios expects to achieve the following milestones by the end of 2026:

    • Receive regulatory approvals globally for mitapivat in three initial indications: adult PK deficiency, thalassemia and sickle cell disease
    • Advance a broad clinical pipeline of at least 5 molecules exploring at least 10 indications
    • Foster a robust research pipeline poised to deliver an investigational new drug (IND) every 12-24 months
    • Achieve cash-flow positivity

    Presentation at 40th Annual J.P. Morgan Healthcare Conference

    Agios will webcast its corporate presentation from the virtual 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12 at 7:30 a.m. ET. A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's website at agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

    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 genetically defined diseases. Building on the company's leadership in the field of cellular metabolism, Agios is advancing a robust pipeline of investigational medicines, including a first-in-class pyruvate kinase (PK) activator that is being evaluated for the treatment of three distinct hemolytic anemias – pyruvate kinase deficiency, alpha- and beta-thalassemia and sickle cell disease. In addition to its active late-stage clinical pipeline, Agios has multiple novel investigational therapies in clinical and preclinical development. 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 Agios' plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs, including mitapivat and AG-946; the potential benefits of Agios' products and product candidates; Agios' key milestones and guidance for 2022 and its strategic vision for 2026; its financial guidance regarding the period in which it will have capital available to fund its operations; 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. 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 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 Servier; the impact of the COVID-19 pandemic on 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 future approved products, and launching, marketing and selling 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, including with respect to the regulatory submissions for mitapivat, 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 and 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 collaborations; 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, or SEC, including the risks and uncertainties set forth under the heading Risk Factors in our filings with the SEC. While the list of factors presented here is considered representative, this list should not be considered to be a complete statement of all potential risks and uncertainties. Any forward-looking statements contained in this communication are made only as of the date hereof, and we undertake no obligation to update forward-looking statements to reflect developments or information obtained after the date hereof and disclaim any obligation to do so other than as may be required by law.

    Contacts

    Investors:

    Holly Manning, 617-844-6630

    Senior Director, Investor Relations

    Media:

    Jessica Rennekamp, 857-209-3286

    Director, Corporate Communications

     



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  2. CAMBRIDGE, Mass., Dec. 22, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced that the company is scheduled to present at the virtual 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022, at 7:30 a.m. ET.

    A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

    About Agios
    Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through…

    CAMBRIDGE, Mass., Dec. 22, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced that the company is scheduled to present at the virtual 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022, at 7:30 a.m. ET.

    A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

    About Agios

    Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. The company's most advanced drug candidate is a first-in-class pyruvate kinase (PK) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias. In addition to its active late-stage clinical pipeline, Agios has multiple novel, investigational therapies in clinical and preclinical development. For more information, please visit the company's website at www.agios.com.

    Contacts

    Investors:

    Holly Manning, 617-844-6630

    Senior Director, Investor Relations

    Media:

    Jessica Rennekamp, 857-209-3286

    Director, Corporate Communications

     



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  3. – Additional Data Presented at ASH Support the Potential of Mitapivat to Improve Ineffective Erythropoiesis and Iron Overload and Stabilize Bone Mineral Density in PK Deficiency Patients –

    – Mitapivat Is Under Regulatory Review in the U.S. and EU as a Potential Treatment for Adults with PK Deficiency –

    – Agios to Host Investor Webcast on Dec. 14, 2021, at 7:30 a.m. ET –

    CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported new data from the ongoing long-term extension study assessing the duration of effects of mitapivat on hemoglobin response and transfusion burden reduction in adults with pyruvate…

    – Additional Data Presented at ASH Support the Potential of Mitapivat to Improve Ineffective Erythropoiesis and Iron Overload and Stabilize Bone Mineral Density in PK Deficiency Patients –

    – Mitapivat Is Under Regulatory Review in the U.S. and EU as a Potential Treatment for Adults with PK Deficiency –

    – Agios to Host Investor Webcast on Dec. 14, 2021, at 7:30 a.m. ET –

    CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported new data from the ongoing long-term extension study assessing the duration of effects of mitapivat on hemoglobin response and transfusion burden reduction in adults with pyruvate kinase (PK) deficiency who had participated in one of the pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively. Data from the study were featured in an oral presentation at the American Society of Hematology (ASH) Annual Meeting and Exposition, hosted virtually and in person from Dec. 11-14, 2021, in Atlanta. Mitapivat is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase enzymes.

    Long-term extension data (abstract #848) demonstrate that previously reported effects of mitapivat on hemoglobin and transfusion burden were maintained over time. Non-regularly transfused patients randomized to mitapivat in ACTIVATE maintained hemoglobin response for up to 19.5 months. Similarly, 35 percent of ACTIVATE patients who switched from placebo to mitapivat in the extension study achieved a hemoglobin response, which was maintained for the duration of follow-up. All regularly transfused patients who achieved transfusion-free status in ACTIVATE-T with mitapivat treatment maintained the status through the extension study for up to 21.9 months. Mitapivat was well tolerated, and the safety profile was consistent with the core period of ACTIVATE and ACTIVATE-T, as well as previous studies.

    "The Phase 3 long-term extension data demonstrate that mitapivat's clinically meaningful effects on hemoglobin response and reduction in transfusion burden can be maintained over time, sustaining its potential impact on patients' lives," said Rachael Grace, M.D., MMSc, director of hematology clinical research at Boston Children's Hospital and investigator on the study. "Treatment with mitapivat has resulted in improvements in many of the most challenging manifestations of PK deficiency in a broad spectrum of patients, and I believe it has the potential to be an important treatment option for this community."

    In addition, Agios presented data at ASH further supporting the potential of mitapivat to address hallmark symptoms and complications of PK deficiency.

    Mitapivat Improves Ineffective Erythropoiesis and Reduces Iron Overload in Patients with Pyruvate Kinase Deficiency (Abstract #757)

    In an oral presentation, data from ACTIVATE, ACTIVATE-T and the Phase 3 long-term extension study were reported, showing that treatment with mitapivat improved markers of ineffective erythropoiesis and iron metabolism in adults with PK deficiency, regardless of transfusion status. Through this mechanism, mitapivat may have the potential to improve iron homeostasis, thereby reducing iron overload.

    Bone Mineral Density Remains Stable in Pyruvate Kinase Deficiency Patients Receiving Long-term Treatment with Mitapivat (Abstract #924)

    In a separate poster presentation, data from a large, pooled analysis of the DRIVE-PK Phase 2 study, ACTIVATE and ACTIVATE-T Phase 3 studies and the Phase 3 long-term extension study were reported, assessing the impact of mitapivat treatment on bone mineral density in a broad population of non-regularly transfused and regularly transfused adults with PK deficiency for up to 5.5 years. Among the 64 patients who had low bone mineral density at baseline, 62 patients remained stable or improved while being treated with mitapivat.

    "PK deficiency is characterized by serious symptoms and complications, including anemia, iron overload and osteoporosis, regardless of transfusion status, and there are currently no approved therapies for people living with this disease," said Sarah Gheuens, M.D., Ph.D., chief medical officer of Agios. "Collectively, the data we've presented at ASH continue to support the potential of mitapivat to revolutionize care for PK deficiency patients as the first disease-modifying therapy for this chronic, debilitating disease. We look forward to working with regulators as they continue their review of mitapivat for potential approval in the U.S. and EU next year."

    Mitapivat is not approved for use by any regulatory authority.

    Conference Call Information

    Agios will host a virtual investor event at 7:30 a.m. ET on Dec. 14, 2021, 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. There are no currently approved therapies for PK deficiency. For more information, please visit www.knowpkdeficiency.com.

    Agios, in partnership with PerkinElmer Genomics, launched the Anemia ID program to offer no-cost genetic testing to eligible patients in the U.S with suspected hereditary anemias, including PK deficiency. The program was created in response to feedback from patients, advocates and physicians about the need for improved diagnosis to inform disease management decisions. To learn more, please visit www.AnemiaID.com.

    Agios also launched the myAgios® patient support services program for people living with PK deficiency and their caregivers. After enrolling in the program, patients and caregivers are connected with a dedicated Patient Support Manager (PSM) with a clinical background to provide tailored support, educational resources and opportunities to connect with other patients and caregivers in the community. To learn more or enroll, please visit www.myagios.com.

    About Agios

    Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. The company's most advanced drug candidate is a first-in-class pyruvate kinase (PK) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias. In addition to its active late-stage clinical pipeline, Agios has multiple novel, investigational therapies in clinical and preclinical development. 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 Agios' plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs, including mitapivat; the potential benefits of Agios' products and product candidates, including mitapivat; 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. 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 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; 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 future approved products, and launching, marketing and selling 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 and 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 collaborations; 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, or SEC, including the risks and uncertainties set forth under the heading Risk Factors in our filings with the SEC. While the list of factors presented here is considered representative, this list should not be considered to be a complete statement of all potential risks and uncertainties. Any forward-looking statements contained in this communication are made only as of the date hereof, and we undertake no obligation to update forward-looking statements to reflect developments or information obtained after the date hereof and disclaim any obligation to do so other than as may be required by law.

    Contacts

    Investors:

    Holly Manning, 617-844-6630

    Senior Director, Investor Relations

    Media:

    Jessica Rennekamp, 857-209-3286

    Director, Corporate Communications



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  4. – Global Phase 3 ENERGIZE and ENERGIZE-T Studies of Mitapivat in Transfusion-dependent and Non-transfusion-dependent α- and β-Thalassemia Initiated –

    – Agios to Host Investor Webcast on Dec. 14, 2021, at 7:30 a.m. ET –

    CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported data for the first time from the ongoing long-term extension period of the Phase 2 open-label study of mitapivat, a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults with non-transfusion dependent α- or β-thalassemia. Data from the…

    – Global Phase 3 ENERGIZE and ENERGIZE-T Studies of Mitapivat in Transfusion-dependent and Non-transfusion-dependent α- and β-Thalassemia Initiated –

    – Agios to Host Investor Webcast on Dec. 14, 2021, at 7:30 a.m. ET –

    CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported data for the first time from the ongoing long-term extension period of the Phase 2 open-label study of mitapivat, a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults with non-transfusion dependent α- or β-thalassemia. Data from the study were featured in an oral presentation at the American Society of Hematology (ASH) Annual Meeting and Exposition, hosted virtually and in person from Dec. 11-14, 2021, in Atlanta.

    Consistent with previously reported data, durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis, were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients. Mitapivat was well tolerated, and the safety profile was consistent with previous studies.

    "The data presented today continue to demonstrate that chronic treatment with mitapivat is well tolerated and has the potential to meaningfully improve hallmarks of thalassemia, including hemolysis and ineffective erythropoiesis. I am particularly excited by the data generated in α-thalassemia, as there are no currently approved therapies for this subtype," said Kevin Kuo, M.D., hematologist at University Health Network, University of Toronto, and an investigator in the study. "Mitapivat has the potential to be an important treatment option for people with this lifelong disease characterized by severe complications, and I look forward to its continued advancement in pivotal clinical trials."

    Long-term Efficacy and Safety of the Oral Pyruvate Kinase Activator Mitapivat in Adults with Non-transfusion-dependent Alpha- or Beta-Thalassemia (Abstract #576)

    The open-label Phase 2 study evaluated the efficacy, safety, pharmacokinetics and pharmacodynamics of mitapivat treatment in adults with either non-transfusion-dependent α- or β-thalassemia who have a baseline hemoglobin concentration of ≤10 g/dL. The trial enrolled 20 patients. All patients were treated with an initial dose of 50 mg mitapivat twice daily followed by a dose-level increase to 100 mg twice daily at the Week 6 visit based on safety evaluations and hemoglobin concentrations. Following the completion of the 24-week core period, patients had the opportunity to enroll in an optional 10-year extension period to evaluate long-term efficacy and safety of mitapivat in this population. As of the data cut-off date of March 27, 2021, 17 of the 20 patients remain in the extension phase with a median treatment duration of 70.9 weeks (range 54.7-105.6).

    As of the data cut-off, efficacy results were as follows:

    • Mean hemoglobin increase from baseline to Week 60 (α-thalassemia, n = 4; β-thalassemia, n = 9) was 1.5 g/dL.
    • Mean hemoglobin increase from baseline to Week 72 (β-thalassemia, n = 8) was 1.7 g/dL.
    • Improvements in markers of hemolysis and ineffective erythropoiesis achieved during the core period were sustained among both α- and β-thalassemia patients, up to Week 72.

    Adverse events (AEs) for patients who continued in the study (n=17) were comparable in the core and extension periods. No new safety signals were identified in the extension period.

    "Following encouraging results from our Phase 2 trial of mitapivat – the first clinical study of a PK activator in thalassemia and the first drug trial in α-thalassemia – we are now focused on advancing the development of mitapivat for patients as quickly and efficiently as possible," said Sarah Gheuens, M.D., Ph.D., chief medical officer at Agios. "Our two global, placebo-controlled pivotal trials of mitapivat – ENERGIZE and ENERGIZE-T – have been initiated, and we look forward to enrolling the first patients soon."

    Mitapivat is not approved for use by any regulatory authority.

    ENERGIZE Trial Design

    ENERGIZE is a Phase 3, double-blind, randomized, placebo-controlled multicenter study evaluating the efficacy and safety of mitapivat as a potential treatment for adults with non-transfusion-dependent α- or β-thalassemia, defined as ≤5 red blood cell units during the 24-week period before randomization and no red blood cell transfusions ≤8 weeks before providing informed consent or during the screening period.

    The primary endpoint of the trial is percentage of patients with hemoglobin response, defined as a ≥1.0 g/dL increase in average hemoglobin concentration from Week 12 through Week 24 compared with baseline. Secondary endpoints include markers of hemolysis and ineffective erythropoiesis, as well as patient-reported outcome (PRO) measures.

    ENERGIZE-T Trial Design

    ENERGIZE-T is a Phase 3, double-blind, randomized, placebo-controlled multicenter study evaluating the efficacy and safety of mitapivat as a potential treatment for adults with transfusion-dependent α- or β-thalassemia, defined as 6 to 20 red blood cell units transfused and ≤6-week transfusion-free period during the 24-week period before randomization.

    The primary endpoint of the trial is percentage of patients with transfusion reduction response, defined as a ≥50% reduction in transfused red blood cell units with a reduction of ≥2 units of transfused red blood cells in any consecutive 12-week period through Week 48 compared with baseline. Secondary endpoints include additional transfusion reduction measures and percentage of participants with transfusion-independence.

    Conference Call Information

    Agios will host a virtual investor event at 7:30 a.m. ET on Dec. 14, 2021, 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 Thalassemia

    Thalassemia is a rare, inherited blood disorder caused by mutations in either alpha (α)- or beta (β)-globin genes, resulting in excessive destruction of red blood cells. Globin precipitates in thalassemia cause oxidative damage, leading to hemolytic anemia, ineffective erythropoiesis and iron overload.

    Thalassemia is associated with serious complications, including fatigue, jaundice, facial bone deformities, delayed growth and development, abdominal swelling, dark urine and reduced life expectancy. Current management strategies for β-thalassemia can include ​​red blood cell transfusions, splenectomy and stem cell transplant, which are associated with short- and long-term risks. There are no currently approved therapies for α-thalassemia.

    About Agios

    Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. The company's most advanced drug candidate is a first-in-class pyruvate kinase (PK) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias. In addition to its active late-stage clinical pipeline, Agios has multiple novel, investigational therapies in clinical and preclinical development. 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 Agios' plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs, including mitapivat; the potential benefits of Agios' products and product candidates, including mitapivat; 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. 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 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; 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 future approved products, and launching, marketing and selling 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 and 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 collaborations; 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, or SEC, including the risks and uncertainties set forth under the heading Risk Factors in our filings with the SEC. While the list of factors presented here is considered representative, this list should not be considered to be a complete statement of all potential risks and uncertainties. Any forward-looking statements contained in this communication are made only as of the date hereof, and we undertake no obligation to update forward-looking statements to reflect developments or information obtained after the date hereof and disclaim any obligation to do so other than as may be required by law.

    Contacts

    Investors:

    Holly Manning, 617-844-6630

    Senior Director, Investor Relations

    Media:

    Jessica Rennekamp, 857-209-3286

    Director, Corporate Communications

     



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  5. – Data Underscore Potential of Mitapivat to Provide Clinically Meaningful Outcomes for Patients –

    – Safety Profile Consistent with Previously Reported Clinical Data –

    – Agios Announces Initiation of Phase 2/3 RISE UP Study of Mitapivat in Adults with Sickle Cell Disease –

    – Agios to Host Investor Webcast on Dec. 14, 2021, at 7:30 a.m. ET –

    CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced new data from investigator-led studies of mitapivat, a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults…

    – Data Underscore Potential of Mitapivat to Provide Clinically Meaningful Outcomes for Patients –

    – Safety Profile Consistent with Previously Reported Clinical Data –

    – Agios Announces Initiation of Phase 2/3 RISE UP Study of Mitapivat in Adults with Sickle Cell Disease –

    – Agios to Host Investor Webcast on Dec. 14, 2021, at 7:30 a.m. ET –

    CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced new data from investigator-led studies of mitapivat, a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults with sickle cell disease. The data, reported by the National Institutes of Health (NIH) and University Medical Center (UMC) Utrecht, were featured in two presentations at the American Society of Hematology (ASH) Annual Meeting and Exposition, hosted virtually and in person from Dec. 11-14, 2021, in Atlanta. The data demonstrate the potential of mitapivat to provide clinically meaningful outcomes for patients, including improvements in anemia, hemolysis and red blood cell sickling.

    Consistent with previously reported proof-of-concept data, mitapivat reduced 2,3-diphosphoglycerate (2,3-DPG) and increased adenosine triphosphate (ATP), and through this mechanism, may reduce hemoglobin S polymerization and red blood cell sickling. The safety profile observed in both studies was also generally consistent with previously published clinical data, including Phase 3 data in adults with pyruvate kinase deficiency.

    "Results reported from these investigator-led studies provide additional efficacy, safety and translational data that continue to support the clinical development of mitapivat in people with sickle cell disease, a lifelong, debilitating condition with few treatment options," said Sarah Gheuens, M.D., Ph.D., chief medical officer at Agios. "We'd like to thank our collaborators at the NIH and UMC Utrecht and look forward to building upon their contributions through our recently initiated Phase 2/3 RISE UP trial. In collaboration with the sickle cell disease community, we designed this trial to have a broad global reach, reduce barriers to participation and understand how mitapivat can impact the aspects of the disease that patients indicated were of greatest importance to them."

    Safety and Efficacy of Mitapivat (AG-348), An Oral Activator of Pyruvate Kinase-R, in Patients with Sickle Cell Disease: A Phase 2, Open-label Study (ESTIMATE) (Abstract #2005)

    The Phase 2 ESTIMATE study being conducted by UMC Utrecht is designed to assess safety and efficacy of mitapivat in patients with sickle cell disease. The primary endpoints of the study are safety and point of sickling (point on the deoxygenation curve when sickling begins), and key secondary endpoints include changes in hemoglobin levels and clinical complication rates.

    Key findings from six patients who completed the eight-week dose-finding period, all reaching 100 mg twice daily dosing, include:

    • No serious adverse events (AEs) occurred, and all AEs were mild and mostly transient. One vaso-occlusive crisis (VOC) occurred without hospital admission and did not require dose reduction or discontinuation.
    • All six patients had improvements in point of sickling.
    • Observed changes in 2,3-DPG and ATP levels were consistent with proposed mechanism of action.
    • Mitapivat increased hemoglobin and decreased hemolysis and sickling parameters.
      • Five out of six patients (83.3%) achieved hemoglobin response (≥ 1g/dL increase from baseline).
    • Early improvements in albumin-to-creatinine ratio were observed.
    • Follow-up data from the ongoing study will be reported at a later stage.

    Mitapivat (AG-348) Demonstrates Safety, Tolerability and Improvements in Anemia, Hemolysis, Oxygen Affinity and Hemoglobin S Polymerization Kinetics in Adults with Sickle Cell Disease: A Phase 1 Dose Escalation Study (Abstract #10)

    The Phase 1 study, which is being conducted in collaboration with the NIH as part of a cooperative research and development agreement, is designed to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with mitapivat in adults with sickle cell disease. The primary endpoint of the study is safety and tolerability, as assessed by frequency and severity of AEs and laboratory parameters. Secondary endpoints include changes in hemoglobin, markers of hemolysis, 2,3-DPG and ATP levels and hemoglobin S polymerization.

    Key findings from the 16 evaluable patients who completed the core period of the study, including up to eight weeks of dose escalation, dose taper and four-week safety follow-up, include:

    • No AEs led to drug discontinuation.
      • No VOCs occurred during dose-escalation. Two VOCs occurred during drug taper, which were deemed possibly drug-related, and two VOCs occurred during the 28-day safety follow-up.
    • Treatment with mitapivat demonstrated improvement in anemia and decreases in markers of hemolysis, including lactate dehydrogenase, total bilirubin, absolute reticulocyte count and aspartate aminotransferase.
      • Nine out of 16 patients (56.3%) achieved a hemoglobin response (≥1g/dL increase from baseline).
    • Mitapivat reduced 2,3-DPG and increased ATP, with expected increase in oxygen affinity and decreased sickling rate.
    • Long-term disease modifying effects being evaluated in an ongoing extension study.

    Mitapivat is not approved for use by any regulatory authority.

    Overview of Phase 2/3 RISE UP Study

    The Phase 2/3 RISE UP study will evaluate the efficacy and safety of mitapivat in sickle cell disease patients who are 16 years of age or older, have had between two and 10 sickle cell pain crises in the past 12 months, and have hemoglobin within the range of 5.5 to 10.5 g/dL during screening.

    The Phase 2 portion, which was recently initiated, includes a 12-week randomized, placebo-controlled period in which participants will be randomized in a 1:1:1 ratio to receive 50 mg mitapivat twice daily, 100 mg mitapivat twice daily or matched placebo. The primary endpoints are hemoglobin response, defined as ≥1 g/dL increase in average hemoglobin concentration from Week 10 through Week 12 compared to baseline, and safety. These data will be used to establish a clear dosing paradigm for the Phase 3 portion.

    The Phase 3 portion includes a 52-week randomized, placebo-controlled period in which participants will be randomized in a 2:1 ratio to receive the recommended mitapivat dose level or placebo. The primary endpoints are hemoglobin response, defined as ≥1 g/dL increase in average hemoglobin from baseline to Week 52, and annualized rate of sickle cell pain crises. Participants who complete either the Phase 2 or Phase 3 portion will have the option to move into a 216-week open-label extension period to receive mitapivat.

    Conference Call Information

    Agios will host a virtual investor event at 7:30 a.m. ET on Dec. 14, 2021, 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 Sickle Cell Disease

    Sickle cell disease is caused by inherited mutations in the beta-globin gene, leading to sickle-shaped red blood cells that are rigid and prone to getting trapped in small vessels, slowing or stopping the flow of blood. People with sickle cell disease have inherited mutations in the beta-globin gene, which lead to elevated levels of the metabolite 2,3-DPG (2,3-diphosphoglycerate) and decreased adenosine triphosphate (ATP) levels.

    Sickle cell disease can cause pain and is associated with serious complications, including anemia, increased risk of infection, acute chest syndrome and stroke. Current management strategies for sickle cell disease can include ​​red blood cell transfusions, stem cell transplant and pain medications, which are associated with short- and long-term risks. In addition, some disease-modifying therapies are available, but significant unmet need remains. Although sickle cell disease is considered a rare disease in the U.S. and EU, it is one of the most common genetic disorders in the world.

    About Agios

    Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. The company's most advanced drug candidate is a first-in-class pyruvate kinase (PK) activator, mitapivat, that is currently being evaluated for the treatment of three distinct hemolytic anemias. In addition to its active late-stage clinical pipeline, Agios has multiple novel, investigational therapies in clinical and preclinical development. 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 Agios' plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs, including mitapivat; the potential benefits of Agios' products and product candidates, including mitapivat; 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. 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 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; 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 future approved products, and launching, marketing and selling 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 and 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 collaborations; 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, or SEC, including the risks and uncertainties set forth under the heading Risk Factors in our filings with the SEC. While the list of factors presented here is considered representative, this list should not be considered to be a complete statement of all potential risks and uncertainties. Any forward-looking statements contained in this communication are made only as of the date hereof, and we undertake no obligation to update forward-looking statements to reflect developments or information obtained after the date hereof and disclaim any obligation to do so other than as may be required by law.

    Contacts

    Investors:

    Holly Manning, 617-844-6630

    Senior Director, Investor Relations

    Media:

    Jessica Rennekamp, 857-209-3286

    Director, Corporate Communications

     



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