On January 11, 2021 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, reported its key 2021 milestones that will drive its recently announced strategic pivot to focus on developing and commercializing innovative treatments for genetically defined diseases, as well as its five-year vision for the company (Press release, Agios Pharmaceuticals, JAN 11, 2021, View Source [SID1234573824]). Agios will present at the virtual 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11 at 10:50 a.m. ET, and a live webcast will be available at investor.agios.com.
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"Agios is at an exciting inflection point as we prepare to move forward with a singular focus on genetically defined diseases," said Jackie Fouse, Ph.D., chief executive officer of Agios. "2021 will be a year of significant momentum and further evidence of our potential to meaningfully impact the lives of patients with unmet needs in pyruvate kinase deficiency, thalassemia, sickle cell disease and other genetically defined diseases. This year, we expect to file for the approval of mitapivat in pyruvate kinase deficiency, which currently has no disease-modifying treatment options, and to initiate pivotal development programs in thalassemia and sickle cell disease. We also expect to further unlock the potential of PK activation across a range of genetically defined diseases by advancing our rich and sustainable research pipeline. As we reimagine the future of Agios, we look forward to building on our core values and pioneering leadership in cellular metabolism to expand and accelerate our work on behalf of patients."
Anticipated 2021 Key Milestones
Agios expects to achieve the following key milestones in 2021:
Corporate
Complete sale of oncology portfolio to Servier, in a transaction worth up to $2 billion plus royalties, in the second quarter of 2021 subject to shareholder approval and satisfaction of regulatory conditions, and commence return of at least $1.2 billion to shareholders post-closing
Genetically Defined Disease Program Milestones
File for regulatory approval for mitapivat in adults with PK deficiency: submit new drug application (NDA) in the U.S. in the second quarter of 2021 and marketing authorization application (MAA) in the EU in mid-2021
Initiate two Phase 3 studies of mitapivat, ENERGIZE and ENERGIZE-T, in not regularly transfused and regularly transfused adults with thalassemia in the second half of 2021
Announce pivotal development plan for mitapivat in adults with sickle cell disease in the first half of 2021 and initiate pivotal development program by year-end
Prioritize new indications for pyruvate kinase R (PKR) and pyruvate kinase M2 (PKM2) activator clinical development by year-end
Genetically Defined Disease Data Presentations
Report topline data from the Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions in the first quarter of 2021
Submit data from the following clinical studies for presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Virtual Congress, which will be held June 9-17, 2021:
Phase 3 ACTIVATE study of mitapivat in adults with PK deficiency who do not receive regular transfusions
Phase 3 ACTIVATE-T study of mitapivat in adults with PK deficiency who receive regular transfusions
Phase 2 study of mitapivat in adults with α- and β-thalassemia who do not receive regular transfusions
Submit data from ongoing clinical studies of mitapivat in sickle cell disease for presentation at medical meetings throughout 2021
Present data from the single ascending dose (SAD) and multiple ascending dose (MAD) cohorts of the Phase 1 study of AG-946, the company’s next-generation PKR activator, in healthy volunteers by year-end
Oncology Milestones & Data Presentations
Present mature overall survival data from the Phase 3 ClarIDHy study of TIBSOVO (ivosidenib tablets) in patients with previously treated IDH1-mutant cholangiocarcinoma at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO-GI), which will be held virtually January 15-17, 2021
Submit supplemental new drug application (sNDA) in the U.S. for TIBSOVO in patients with previously treated IDH1-mutant cholangiocarcinoma in the first quarter of 2021
Enrollment in the Phase 3 AGILE trial of TIBSOVO in combination with azacitidine in adult patients with previously untreated IDH1-mutant acute myeloid leukemia is expected to be complete by year-end
Enrollment in the relapsed or refractory myelodysplastic syndrome arm of the TIBSOVO Phase 1 study of IDH1-mutant advanced hematologic malignancies is expected to be complete by year-end
Full-year net product revenue for TIBSOVO is expected to be $160-170 million
Agios 2025 Strategic Vision
The Agios 2025 strategic vision reflects the company’s expected evolution over the next five years in light of its singular focus on genetically defined diseases. As part of this vision, Agios expects to achieve the following milestones by the end of 2025:
Receive regulatory approval for mitapivat in three initial indications: 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
2020 Year-End Cash and Guidance
Agios ended 2020 with approximately $670.5 million of cash, cash equivalents and marketable securities. The company expects that its cash, cash equivalents and marketable securities as of December 31, 2020, together with anticipated product and royalty revenue, interest income and expense reimbursements under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its planned operating expenses and capital expenditure requirements to the end of 2022. Following the completion of the transaction with Servier and the subsequent shareholder returns, Agios expects its cash runway to extend to cash-flow positivity in 2025.
Presentation at 39th Annual J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation from the virtual 39th Annual J.P. Morgan Healthcare Conference on Monday, January 11, 2021 at 10:50 a.m. ET (7:50 a.m. PT). 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.