Agios Reports Fourth Quarter and Full Year 2016 Financial Results and Highlights Key 2017 Milestones
- Ivosidenib (AG-120) NDA Submission for IDH1m R/R AML Planned by Year End 2017; First Phase 1 Expansion Data Expected in the Second Half of 2017 -
- AG-348 Pivotal Trial Design Update Expected in the Third Quarter of 2017; Updated DRIVE PK Data Planned for the First Half of 2017 -
- MTAP Pathway IND Submission Expected by Year End 2017; Updated Preclinical Data to be Presented at Keystone Tumor Metabolism Meeting in March -
- Company Ends 2016 in Strong Financial Position with
“Our 2016 accomplishments, including the enasidenib NDA submission with our collaboration partner
KEY UPCOMING MILESTONES
The company expects to achieve the following key milestones:
IDH Mutant Inhibitors in Hematologic Malignancies
- Potential approval of enasidenib in
the United Statesfor IDH2m positive relapsed/refractory (R/R) acute myeloid leukemia (AML) in collaboration with Celgeneby the end of 2017.
- Submit a new drug application (NDA) to the U.S.
FDAfor ivosidenib (AG-120) for IDH1m positive R/R AML by the end of 2017.
- Initiate a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib (AG-120) and VIDAZA® in newly diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy in the first half of 2017.
IDH Mutant Inhibitors in Solid Tumors
- Complete the dose-escalation phase of the ongoing Phase 1 study of AG-881 in IDHm positive glioma in the first half of 2017.
Rare Genetic Diseases
- Finalize design for a global pivotal trial of AG-348 in pyruvate kinase (PK) deficiency in the third quarter of 2017.
- Initiate a global pivotal trial of AG-348 in PK deficiency in the first half of 2018.
- Submit an Investigational New Drug (IND) application for the development candidate targeting methylthioadenosine phosphorylase (MTAP)-deleted tumors by the end of 2017. MTAP is a metabolic enzyme that is deleted in approximately 15 percent of all cancers.
ANTICIPATED 2017 DATA PRESENTATIONS
- First data from the expansion phase of the ongoing Phase 1 study of ivosidenib (AG-120) in R/R AML in the second half of 2017
- First data from the ongoing Phase 1b combination study of enasidenib or ivosidenib (AG-120) with standard-of-care intensive chemotherapy in newly diagnosed AML in the second half of 2017
- First data from the cholangiocarcinoma expansion cohort of the ongoing Phase 1 study of ivosidenib (AG-120) in advanced IDH1m positive solid tumors in the first half of 2017
- Updated data from the glioma expansion of the ongoing Phase 1 study of ivosidenib (AG-120) in advanced IDH1m positive solid tumors in the second half of 2017
- Updated data from AG-348 Phase 2 DRIVE PK study in PK deficiency in both the first and second half of 2017
- Updated preclinical data for the program targeting MTAP-deleted tumors at the Keystone Tumor Metabolism Meeting taking place
March 5-9, 2017in Whistler, British Columbia
FOURTH QUARTER 2016 HIGHLIGHTS
- Supported Celgene’s submission of an NDA for enasidenib in IDH2m positive R/R AML.
- Initiated a global, registration-enabling randomized Phase 3 study (ClarIDHy) for ivosidenib (AG-120) in IDH1m positive advanced cholangiocarcinoma. The
FDAalso granted ivosidenib Fast Track Designation for the treatment of patients with previously treated, unresectable or metastatic cholangiocarcinoma with an IDH1 mutation.
- Completed the dose-escalation phase of the Phase 1 study of AG-881 in IDHm positive hematologic malignancies. The study is now closed for enrollment.
- Selected a development candidate targeting MTAP-deleted tumors to enter IND-enabling studies.
FULL YEAR 2016 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of December 31, 2016 were $573.6 million, compared to $375.9 million as of December 31, 2015. This increase was driven by cash received under our collaboration agreements with
Collaboration revenue was $69.9 million for the year ended December 31, 2016, compared to $59.1 million for the prior year.
Research and development (R&D) expenses were $220.2 million, including $25.4 million of stock-based compensation expense, for the year ended December 31, 2016, compared to $141.8 million, including $17.4 million in stock-based compensation expense, for the year ended December 31, 2015. The increase in R&D expenses was primarily due to increased costs to support advancement of the company's lead investigational medicines toward later-stage development. Celgene is responsible for all development costs for enasidenib and certain development costs for AG-881 and reimburses the company for development costs incurred for these investigational medicines.
General and administrative (G&A) expenses were $50.7 million, including $16.7 million of stock-based compensation expense, for the year ended December 31, 2016, compared to $36.0 million, including $14.5 million of stock-based compensation expense, for the year ended December 31, 2015. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.
Net loss for the year ended December 31, 2016 was $198.5 million, compared to a net loss of $117.7 million for the year ended December 31, 2015.
Based on its current operating plans, the company expects that its existing cash, cash equivalents and marketable securities as of
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class investigational medicines in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company's website at www.agios.com.
About Agios/Celgene Collaboration
Enasidenib and AG-881 are part of Agios' global strategic collaboration with
Cautionary Note Regarding Forward-Looking Statement
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the Agios’ plans, strategies and expectations for its and its collaborator’s preclinical, clinical and commercial advancement of its drug development programs including enasidenib, ivosidenib (AG-120)
|Consolidated Balance Sheet Data
|Cash, cash equivalents and marketable securities||$||573,564||$||375,907|
|Collaboration receivable – related party||4,886||8,225|
|Deferred revenue – related party||190,210||24,364|
|Consolidated Statements of Operations Data
(in thousands, except share and per share data)
|Three Months Ended December 31,
||Years Ended December 31,|
|Collaboration revenue – related party||$||22,648||$||6,218||$||69,892||$||59,119|
|Research and development||64,678||36,933||220,163||141,827|
|General and administrative||15,379||10,182||50,714||35,992|
|Total operating expenses||80,057||47,115||270,877||177,819|
|Loss from operations||(57,409||)||(40,897||)||(200,985||)||(118,700||)|
|Net loss per share– basic and diluted||$||(1.34||)||$||(1.08||)||$||(5.07||)||$||(3.15||)|
|Weighted-average number of common shares used in net loss per share applicable to common stockholders – basic and diluted||42,110,541||37,660,033||39,126,400||37,429,262|
Kendra Adams, 617-844-6407 Senior Director, Investor & Public Relations Kendra.Adams@agios.com Renee Leck, 617-649-8299 Senior Manager, Investor & Public Relations Renee.Leck@agios.com Media: Holly Manning, 617-844-6630 Associate Director, Corporate Communications Holly.Manning@agios.com