On January 11, 2016 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported the company’s 2016 strategy and expected clinical development and research milestones in conjunction with the 34th Annual J.P. Morgan Healthcare Conference in San Francisco (Press release, Agios Pharmaceuticals, JAN 11, 2016, View Source [SID:1234508737]). The presentation will outline three strategic priorities for 2016: continue rapid and broad late-stage clinical development for its lead isocitrate dehydrogenase (IDH) mutant inhibitors in hematologic malignancies and solid tumors; demonstrate clinical activity of its wholly owned, global pyruvate kinase-R (PKR) activators in patients; and advance research and initiate preclinical development of a program from the next wave of research.

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"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 David Schenkein, M.D., chief executive officer at Agios. "We believe these milestones, coupled with our growing late-stage development and commercial capabilities, set Agios firmly on the path to become a sustainable, multi-product biopharmaceutical company with a strong research core and broad pipeline of first-in-class medicines."

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."

AG-221, AG-120 and AG-881 are part of Agios’ global strategic collaboration with Celgene Corporation.

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 Scott Biller, Ph.D., chief scientific officer at Agios. "We are excited to move the first program in our next wave of investigational medicines into preclinical development this year."

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 J.P. Morgan Healthcare Conference
Agios will webcast its corporate presentation from the 34th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, January 11, 2016 at 3:30 p.m. PST (6:30 p.m. EST). A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors and Media 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.