Agios Outlines Key 2016 Goals and Priorities
- Complete Enrollment of 125-Patient Expansion Cohorts for AG-221 and AG-120 in Relapsed/Refractory Acute Myeloid Leukemia in Second Half of 2016 -
- Present First Data from Phase 2 DRIVE PK Study for AG-348 in PK Deficiency and Phase 1 Healthy Volunteer Study for AG-519 in First Half of 2016 -
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- Company to Present at the 34th Annual J.P. Morgan Healthcare Conference Today at
“We expect each of our programs to achieve important catalysts in 2016 that will bring us closer to our vision of helping people with cancer and rare genetic disorders,” said
IDH Mutant Inhibitors
Dr. Schenkein continued, “We remain focused on executing on our ‘speed and breadth’ clinical development strategy for AG-221 and AG-120 in hematological malignancies, with the intent to complete enrollment of both 125-patient expansion cohorts this year. Further understanding the potential of our IDH mutant inhibitors in solid tumors remains a priority with several new and ongoing trials in 2016.”
Expected 2016 milestones for IDH mutant inhibitors in hematologic malignancies:
- Complete enrollment of both 125-patient expansion cohorts for the Phase 1/2 study of AG-221 and Phase 1 study of AG-120 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) in the second half of 2016
- Initiate a global, registration-enabling Phase 3 study of AG-120 in frontline AML patients with an IDH1 mutation in the second half of 2016
- Initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in 2016
- Initiate a Phase 1/2 frontline combination study of AG-221 or AG-120 with VIDAZA® (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy in the first quarter of 2016
- Continue to enroll patients in the following ongoing clinical trials:
- Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML
- Phase 1b frontline combination study of AG-221 or AG-120 with standard-of-care intensive chemotherapy in AML
- Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies
Expected 2016 milestones for IDH mutant inhibitors in solid tumors:
- Initiate a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of 2016
- Continue to enroll patients in the following ongoing clinical trials:
- Expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive solid tumors
- Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive solid tumors
PKR Activators
“Having initiated dosing in the Phase 1 healthy volunteer study of AG-519, we've completed the first of several key clinical milestones expected from our PKR activators in the first half of this year,” said Dr. Schenkein. “Notably, we expect to present the first data from this study and the Phase 2 DRIVE PK study for AG-348 in PK deficiency patients. There are currently no approved or disease-modifying treatments for PK deficiency, which drives our focus on advancing potential new treatment options for these patients.”
Milestone announced today:
- Dosing was initiated in an integrated single ascending dose (SAD) and multiple ascending dose (MAD) placebo-controlled Phase 1 study of AG-519 in healthy volunteers
Expected 2016 milestones for PKR activators:
- Present the first data from DRIVE PK, a global Phase 2, open-label safety and efficacy trial of AG-348 in adult, transfusion-independent patients with PK deficiency in the first half of 2016
- Present data from Phase 1 study of AG-519 in healthy volunteers as well as preclinical findings about the molecule in the first half of 2016
- Outline the clinical development plans for Agios’ PKR activators in beta-thalassemia in the second half of 2016
- Present new findings from the Natural History Study of PK deficiency being conducted with Boston Children’s Hospital in the second half of 2016
Research Programs
“We continue to focus on discovering and validating first-in-class targets that meet our high bar for development and align with our precision medicine strategy,” said
- Agios scientists have discovered a novel pathway comprised of multiple targets with a shared vulnerability in MTAP-deleted tumors and have demonstrated that this pathway can be modulated by small molecule inhibitors, resulting in robust anti-tumor activity in animal models
- MTAP (methylthioadenosine phosphorylase) is a metabolic enzyme that is deleted in approximately 15 percent of all cancers. This deletion is readily detected by a simple genomic test, thus allowing the selection of patients predicted to be sensitive to the therapy.
Expected 2016 milestones for research:
- Publish preclinical findings on a new cancer metabolism program
- Initiate preclinical development activities for the first molecule in the next wave of novel investigational medicines
Presentation at 34th Annual
Agios will webcast its corporate presentation from the 34th Annual
About Agios
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic metabolic disorders through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class investigational medicines in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company's website at www.agios.com.
About Agios/Celgene Collaboration
VIDAZA® is a registered trademark of
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' product candidates targeting IDH1/IDH2 or pyruvate kinase-R mutations or other genetic mutations, including
Senior Manager, Investor Relations and Public Relations
Renee.Leck@agios.com