AGIO Agios Pharmaceuticals Inc.

46.84
-0.17  -0%
Previous Close 47.01
Open 47.21
52 Week Low 32.47
52 Week High 62.155
Market Cap $2,761,286,808
Shares 58,951,469
Float 35,715,668
Enterprise Value $1,147,173,235
Volume 104,063
Av. Daily Volume 632,662
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Upcoming Catalysts

Drug Stage Catalyst Date
AG-946
Healthy volunteers
Phase 1
Phase 1
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Mitapivat (AG-348)
Pyruvate kinase deficiency
PDUFA priority review
PDUFA priority review
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Drug Pipeline

Drug Stage Notes
Mitapivat (AG-348)
Sickle cell disease.
Phase 2/3
Phase 2/3
Phase 2/3 trial to be initiated by end of 2021.
Mitapivat (ENERGIZE )
Thalassemia
Phase 3
Phase 3
Phase 3 trial to be initiated in 2021.
Mitapivat (AG-348) - (ACTIVATE)
Pyruvate Kinase Deficiency
Phase 3
Phase 3
Phase 3 trial met primary endpoint - December 1, 2020. Data presented at EHA Virtual Congress, June 11, 2021. 40 percent (n=16) of patients randomized to mitapivat achieved a hemoglobin response, compared to 0 patients randomized to placebo (2-sided p<0.0001).
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. CAMBRIDGE, Mass., Aug. 30, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that the company is scheduled to present at the following September investor conferences:

    • Citi's 16th Annual Biopharma Virtual Conference 
      Panel: Sickle Cell, Beta-Thal, ITP & PKD - Measuring Industry Progress in Benign Hematology
      Friday, September 10 at 12:30 p.m. ET
    • 2021 Cantor Virtual Global Healthcare Conference 
      Monday, September 27 at 8:00 a.m. ET

    Live webcasts of the presentations can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. Replays…

    CAMBRIDGE, Mass., Aug. 30, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that the company is scheduled to present at the following September investor conferences:

    • Citi's 16th Annual Biopharma Virtual Conference 

      Panel: Sickle Cell, Beta-Thal, ITP & PKD - Measuring Industry Progress in Benign Hematology

      Friday, September 10 at 12:30 p.m. ET
    • 2021 Cantor Virtual Global Healthcare Conference 

      Monday, September 27 at 8:00 a.m. ET

    Live webcasts of the presentations can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. Replays of the webcasts will be archived on the Agios website for at least two weeks following each 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 R (PKR) 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:

    1AB

    Steve Klass

    steve@1abmedia.com

    Media:

    Jessi Rennekamp, 857-209-3286

    Director, Corporate Communications

    Jessica.Rennekamp@agios.com 



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  2. CAMBRIDGE, Mass., Aug. 17, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's New Drug Application (NDA) for mitapivat for the treatment of adults with pyruvate kinase (PK) deficiency. The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from 10 months to six months from the day of filing acceptance. The FDA's Priority Review designation is given to investigational medicines that treat a serious…

    CAMBRIDGE, Mass., Aug. 17, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's New Drug Application (NDA) for mitapivat for the treatment of adults with pyruvate kinase (PK) deficiency. The NDA was granted a Priority Review designation and has been given a Prescription Drug User Fee Act (PDUFA) action date of February 17, 2022, accelerating the review time from 10 months to six months from the day of filing acceptance. The FDA's Priority Review designation is given to investigational medicines that treat a serious condition and offer significant improvements in safety or effectiveness.

    "The acceptance of our NDA for mitapivat with priority review represents an important milestone on the path to expeditiously deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong hemolytic anemia characterized by serious complications affecting multiple organs," said Sarah Gheuens, M.D., Ph.D., senior vice president of clinical development and incoming chief medical officer at Agios. "We look forward to working with the FDA during the review process and will continue to execute on our global strategy to ensure we are well positioned to rapidly deliver mitapivat to patients and healthcare providers upon approval."

    Agios also submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) in June 2021 for mitapivat as a potential treatment for adults with PK deficiency. As announced on the company's second quarter 2021 earnings call, the MAA passed validation which triggered the start of the MAA review procedure.

    The NDA and MAA submissions are based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively. A full analysis of these data – including patient-reported outcomes (PRO) – was recently presented at the European Hematology Association (EHA) Virtual Congress. An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.

    Mitapivat is not currently approved for use in any country.

    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 mutations in PKR genes 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 pyruvate kinase (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 R (PKR) 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' expectations for the FDA's review of its NDA for mitapivat. The words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" 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, the FDA's acceptance of Agios's NDA for mitapivat does not represent evaluation of the efficacy and safety of mitapivat, and is not a guarantee of approval. 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: risks associated with the regulatory review process generally; the risk that the FDA may determine that the data included in the NDA are insufficient for approval and that the Company must conduct additional clinical trials, or nonclinical or other studies, before mitapivat can be approved; the risk that the results of previously conducted studies involving mitapivat will not be repeated or observed in ongoing or future studies or following commercial launch, if mitapivat is approved; and risks associated with the Company's dependence on third parties with respect to regulatory matters for mitapivat. 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. 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:

    1AB

    Steve Klass

    steve@1abmedia.com

    Media:

    Jessi Rennekamp, 857-209-3286

    Director, Corporate Communications

    Jessica.Rennekamp@agios.com



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  3. – Completed Regulatory Submissions for Mitapivat for the Treatment of Adults with Pyruvate Kinase (PK) Deficiency in the U.S. and EU –

    – Presented Data from ACTIVATE and ACTIVATE-T Phase 3 Studies and Thalassemia Phase 2 Study at European Hematology Association Virtual Congress, Supporting Potential of PK Activation for Underserved Patients with Hemolytic Anemias –

    – Chris Bowden, M.D., to Retire as Chief Medical Officer and Continue as Strategic Advisor; Vice President of Clinical Development Sarah Gheuens, M.D., Ph.D., to Assume Role –

    CAMBRIDGE, Mass., July 29, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported business…

    – Completed Regulatory Submissions for Mitapivat for the Treatment of Adults with Pyruvate Kinase (PK) Deficiency in the U.S. and EU –

    – Presented Data from ACTIVATE and ACTIVATE-T Phase 3 Studies and Thalassemia Phase 2 Study at European Hematology Association Virtual Congress, Supporting Potential of PK Activation for Underserved Patients with Hemolytic Anemias –

    – Chris Bowden, M.D., to Retire as Chief Medical Officer and Continue as Strategic Advisor; Vice President of Clinical Development Sarah Gheuens, M.D., Ph.D., to Assume Role –

    CAMBRIDGE, Mass., July 29, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported business highlights and financial results for the second quarter ended June 30, 2021.

    "Our first quarter as a company solely focused on genetically defined diseases was marked by several significant milestones, most notably our U.S. and EU regulatory submissions for mitapivat for the treatment of adults with PK deficiency, bringing us one step closer to potentially delivering the first disease-modifying treatment for people with this serious and underserved condition," said Jackie Fouse, Ph.D., chief executive officer at Agios. "We continue to unlock the potential of mitapivat for people with other chronic hemolytic anemias and are gaining momentum on our pivotal programs in thalassemia and sickle cell disease. This year, we look forward to initiating our two registrational Phase 3 trials – ENERGIZE and ENERGIZE-T – in not regularly transfused and regularly transfused adults with thalassemia, as well as our pivotal Phase 2/3 trial of mitapivat in sickle cell disease."

    SECOND QUARTER 2021 & RECENT HIGHLIGHTS

    • Completed two regulatory filings for approval of mitapivat in adults with PK deficiency; submitted new drug application (NDA) in the U.S. and marketing authorization application (MAA) in the EU.
    • Presented full analysis of data from Phase 3 ACTIVATE and ACTIVATE-T studies of mitapivat in adults with PK deficiency at European Hematology Association (EHA) Virtual Congress; studies met primary and secondary endpoints, including patient-reported outcome (PRO) measures that address symptom burden and quality-of-life impact in adults.
    • Presented data from Phase 2, open-label, multicenter study of mitapivat in adults with non-transfusion dependent α- or β-thalassemia at EHA Virtual Congress; study met its primary endpoint, with 16 of the 20 patients (80%) achieving a hemoglobin increase of ≥1.0 g/dL from baseline at one or more assessments during Weeks 4-12.
    • Launched myAgios® patient support services for people living with PK deficiency and their caregivers, providing tailored support, educational resources and opportunities to connect with other patients and caregivers in the community.
    • Completed hiring and training of customer-facing and patient support team that will support the U.S. launch of mitapivat in PK deficiency upon product approval.
    • Announced succession plan for Chris Bowden, M.D., who will transition from his role as chief medical officer to strategic advisor following his retirement on Sept. 1, at which time Sarah Gheuens, M.D., Ph.D., vice president of clinical development, will assume the role.
    • Repurchased approximately 10.5 million shares of Agios common stock, inclusive of shares acquired from Bristol-Myers Squibb Company (BMS) and its affiliates, at an average price of $50.41 per share. This accounts for $529 million of the up to $1.2 billion of share repurchases authorized by the Board of Directors following the company's sale of its oncology business to Servier.

    KEY UPCOMING MILESTONES

    • Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia, by year-end.
    • Initiate Phase 2/3 study of mitapivat in sickle cell disease by year-end.
    • Host investor day in fourth quarter to share more information about commercial launch planning for mitapivat in PK deficiency and research and development pipeline.

    Data Presentations

    • Submit data from ongoing collaborator-led clinical studies of mitapivat in sickle cell disease for presentation at the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition, to be held Dec. 11-14.
    • Submit data from the Phase 1 healthy volunteer study of AG-946, the company's next-generation PKR activator, for presentation at the 63rd ASH Annual Meeting & Exposition.

    SECOND QUARTER 2021 FINANCIAL RESULTS

    The financial results discussion compares Agios' continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.

    Research and Development (R&D) Expenses: R&D expenses for continuing operations were $62.0 million for the second quarter of 2021 compared to $54.1 million for the second quarter of 2020. The year-over-year increase in R&D was driven primarily by start-up costs associated with the Phase 3 studies of mitapivat in thalassemia and sickle cell disease, as well as the NDA and MAA filings for mitapivat in PK deficiency and launch preparation activities.

    Selling, General and Administrative (SG&A) Expenses: SG&A expenses for continuing operations were $29.2 million for both the second quarter of 2021 and the second quarter of 2020.

    Non-Operating Income: Non-operating income included approximately $2.0 million from TIBSOVO® (ivosidenib) royalties for the second quarter of 2021.

    Net Loss: Net loss was $86.2 million for the second quarter of 2021 compared to a net loss of $90.5 million for the second quarter of 2020.

    Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2021, were $1.7 billion compared to $794.4 million as of June 30, 2020. The company expects that its cash, cash equivalents and marketable securities as of June 30, 2021 will enable the company to execute its operating plan through major catalysts and to cash-flow positivity without the need to raise additional equity.

    CONFERENCE CALL INFORMATION

    Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2021 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 2663508. The live webcast can be accessed under "Events & Presentations" in the Investors 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 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 R (PKR) 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 and AG-946; the potential benefits of Agios' products and product candidates; Agios' key milestones and guidance for 2021; its financial guidance regarding the period in which it will have capital available to fund its operations; expectations regarding the return of capital to shareholders following the sale of Agios' oncology business; its chief medical officer transition plan 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.





    Consolidated Balance Sheet Data
    (in thousands)

    (Unaudited)
     
     June 30,

    2021
     December 31,

    2020
     
    Cash, cash equivalents and marketable securities$1,734,301 $670,537 
    Assets held for discontinued operations -  50,460 
    Total assets 1,874,603  852,952 
    Liabilities held for discontinued operations -  299,728 
    Stockholders' equity 1,721,816  399,500 





    Consolidated Statements of Operations Data
    (in thousands, except share and per share data)
    (Unaudited)
        
     Three Months Ended June 30, Six Months Ended June 30,
      2021   2020   2021   2020 
    Cost and expenses:       
    Research and development$62,007  $54,086  $119,674  $109,445 
    Selling, general and administrative$29,215  $29,178  $62,765  $60,849 
    Total cost and expenses$91,222  $83,264  $182,439  $170,294 
    Loss from operations$(91,222) $(83,264) $(182,439) $(170,294)
    Gain on sale of oncology business$2,000  $-  $2,000  $- 
    Interest (expense) income, net$(92) $1,769  $248  $4,705 
    Other income, net$6,524  $-  $6,524  $- 
    Net loss from continuing operations$(82,790) $(81,495) $(173,667) $(165,589)
    Net income (loss) from discontinued operations, net of tax$(3,427) $(8,983) $1,961,775  $34,855 
    Net income (loss)$(86,217) $(90,478) $1,788,108  $(130,734)
    Net loss from continuing operations per share - basic and diluted$(1.36) $(1.18) $(2.66) $(2.41)
    Net income (loss) from discontinued operations per share - basic and diluted$(0.06) $(0.13) $30.05  $0.51 
    Net income (loss) per share - basic and diluted$(1.41) $(1.31) $27.39  $(1.90)
    Weighted-average number of common shares used in computing net income (loss) per share – basic and diluted 61,066,977   68,958,091   65,281,827   68,784,109 



    Contacts

    Investors:

    Steve Klass, 1AB

    steve@1abmedia.com

    Media:

    Jessica Rennekamp, 857-209-3286

    Director, Corporate Communications

    Jessica.Rennekamp@agios.com 

     



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  4. CAMBRIDGE, Mass., July 15, 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 will host a conference call and live webcast on Thursday, July 29, 2021 at 8:00 a.m. ET to report its second quarter 2021 financial results and other business highlights.

    A live webcast can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. The conference call can be accessed by dialing 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 2663508. The webcast will be archived and made available for replay on the company's website beginning…

    CAMBRIDGE, Mass., July 15, 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 will host a conference call and live webcast on Thursday, July 29, 2021 at 8:00 a.m. ET to report its second quarter 2021 financial results and other business highlights.

    A live webcast can be accessed under "Events & Presentations" in the Investors section of the company's website at www.agios.com. The conference call can be accessed by dialing 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and referring to conference ID 2663508. The webcast will be archived and made available for replay on the company's website beginning approximately two hours after the event.

    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 R (PKR) 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:

    1AB

    Steve Klass

    steve@1abmedia.com

    Media:

    1AB

    Josie Butler

    josie@1abmedia.com



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  5. CAMBRIDGE, Mass., June 28, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that it has submitted a Marketing Authorisation Application (MAA) for mitapivat to the European Medicines Agency (EMA) for the treatment of adults with pyruvate kinase (PK) deficiency in the European Union. This submission follows the company's recent New Drug Application (NDA) submission of mitapivat to the U.S. Food and Drug Administration (FDA) for treatment of adults with PK deficiency in the U.S.

    "With both our NDA and MAA filings, we are poised to deliver the first potentially disease-modifying therapy…

    CAMBRIDGE, Mass., June 28, 2021 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that it has submitted a Marketing Authorisation Application (MAA) for mitapivat to the European Medicines Agency (EMA) for the treatment of adults with pyruvate kinase (PK) deficiency in the European Union. This submission follows the company's recent New Drug Application (NDA) submission of mitapivat to the U.S. Food and Drug Administration (FDA) for treatment of adults with PK deficiency in the U.S.

    "With both our NDA and MAA filings, we are poised to deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong hemolytic anemia characterized by serious complications affecting multiple organs," said Chris Bowden, M.D., chief medical officer at Agios. "There are no approved therapies for PK deficiency, and the current management strategies of blood transfusions and splenectomy are associated with both short- and long-term risks, including iron overload, blood clots and increased risk for infections. PK deficiency can lead to chronic fatigue, hemolytic crisis, gallstones, splenomegaly, liver cirrhosis, pulmonary hypertension and osteoporosis, and the burden of disease can take a toll on patients' ability to navigate work and other day-to-day activities, as well as on their mental health. We are looking forward to working with both the FDA and EMA to provide a potential new treatment option for this community that may help address the significant unmet needs of PK deficiency patients."

    Like the NDA, the MAA submission is based on results from two pivotal studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively. A full analysis of these data – including patient-reported outcomes (PRO) – was recently presented at the European Hematology Association (EHA) Virtual Congress. An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.

    Mitapivat is not approved for use by any regulatory authority.

    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 mutations in PKR genes 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.

    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 R (PKR) 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 the potential benefits of Agios' products and product candidates; Agios' plans for future regulatory submissions; 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 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:

    1AB

    Steve Klass

    steve@1abmedia.com

    Media:

    1AB

    Josie Butler

    josie@1abmedia.com



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