Agios Reports Third Quarter 2015 Financial Results
– R&D Day Highlights Included Initiation of AG-221 Phase 3 "IDHENTIFY" Study, Design of AG-221 and AG-120 Frontline Combination Studies, and Selection of
"We have made significant progress this year toward realizing our goal of what's possible for patients with our IDH inhibitors, by deploying a comprehensive development strategy of speed and breadth with AG-221 and AG-120 in AML and other cancers," said
KEY UPCOMING MILESTONES IN CANCER METABOLISM
Agios anticipates the following milestones from its IDH clinical development programs in collaboration with
AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein
Present new data from the ongoing Phase 1 dose-escalation and expansion studies of AG-221 in advanced IDH2-mutant positive hematologic malignancies at the
American Society of Hematology(ASH) Annual Meeting taking place December 5-8, 2015in Orlando.
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein
Present first data from the ongoing Phase 1 dose-escalation trial of AG-120 in advanced IDH1-mutant positive solid tumors in an oral presentation at
AACR-EORTC-NCI International Conference on Molecular Targets and Cancer Therapeuticson November 8, 2015in Boston.
- Present new data from the ongoing Phase 1 dose-escalation and expansion studies of AG-120 in advanced IDH1-mutant positive hematologic malignancies at the ASH Annual Meeting.
- Initiate a global registration-enabling Phase 3 study in patients with acute myeloid leukemia (AML) harboring an IDH1 mutation in the first half of 2016.
AG-221 and AG-120 front-line AML combination trials
- Initiate a Phase 1b combination study of either AG-221 or AG-120 with standard induction (7+3, Ara-C and idarubicin/daunorubicin) and consolidation (Ara-C, or mitoxantrone with etoposide) chemotherapy in newly diagnosed AML patients eligible for intensive chemotherapy by the end of 2015.
- Initiate a Phase 1/2 combination study of either AG-221 or AG-120 with VIDAZA® (azacitidine) in newly diagnosed AML patients not eligible for intensive chemotherapy in the first quarter of 2016.
KEY UPCOMING MILESTONES IN RARE GENETIC METABOLIC DISORDERS
AG-348: a novel, first-in-class, oral activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency
Present data from the Phase 1 healthy volunteers study of AG-348 and new findings from the Natural History Study of PK deficiency (being conducted with
Boston Children's Hospital) at the ASH Annual Meeting.
AG-519: a novel, oral activator of PKR for the treatment of PK deficiency
- Initiate an integrated single ascending dose (SAD) and multiple ascending dose (MAD) placebo-controlled Phase 1 study in healthy volunteers in the first quarter of 2016.
RECENT DEVELOPMENT UPDATES IN CANCER METABOLISM
Agios has provided the following updates on its clinical development programs in collaboration with
IDHENTIFY, the Phase 3 study of AG-221, was initiated in October. This is an international, multi-center, open-label, randomized clinical trial designed to compare the efficacy and safety of AG-221 versus conventional care regimens in patients 60 years or older with IDH2 mutant-positive AML that is refractory to or relapsed after second- or third-line therapy. This study is being conducted by
- The expansion phase of the Phase 1 trial of AG-221 is on track and continues to enroll. It includes four cohorts with 25 patients each and a fifth expansion cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or reinduction treatment, or have relapsed after allogeneic transplantation.
- The ongoing Phase 1 trial of AG-221 in IDH2-mutated advanced solid tumors and angioimmunoblastic T-cell lymphoma continues to enroll patients.
- The expansion phase of the Phase 1 trial of AG-120 is on track and continues to enroll. It includes three expansion cohorts of a total of 175 patients with IDH1-mutated advanced hematologic malignancies, including one cohort with 125 patients with relapsed and/or refractory AML.
- The ongoing Phase 1 trial of AG-120 in IDH1-mutated advanced solid tumors continues to enroll.
AG-881: a brain-penetrant, first-in-class, oral, potent pan-inhibitor of the mutated IDH1 and IDH2 proteins
- Two Phase 1, open-label, dose-escalation and expansion studies are on track and continue to enroll – the first in advanced IDH mutant-positive solid tumors and the second in patients with advanced IDH mutant-positive hematologic malignancies whose cancer has progressed on a prior IDHm inhibitor therapy.
RECENT DEVELOPMENT UPDATES IN RARE GENETIC DISORDERS OF METABOLISM
- DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency, is on track and enrolling.
- A natural history study of PK deficiency is also ongoing and patient enrollment is on track.
- Agios selected a fifth molecule for clinical development, AG-519, a novel, oral activator of PKR for the treatment of PK deficiency.
THIRD QUARTER 2015 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of
Collaboration revenue was
Research and development (R&D) expense was
General and administrative (G&A) expense was
Net loss for the third quarter of 2015 was
FINANCIAL GUIDANCE FOR THE FULL YEAR 2015
Agios is reiterating that it expects to end 2015 with more than
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 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 agios.com.
About Agios/Celgene Collaboration
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, including
|Consolidated Balance Sheet Data|
|Cash, cash equivalents and marketable securities||$ 407,986||$ 467,447|
|Collaboration receivable – related party||9,078||6,492|
|Deferred revenue – related party||28,890||38,411|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Gross Collaboration revenue – related party (1)||$5,480||$33,900||$52,901||$50,722|
|Research and development (2)||36,028||25,526||104,894||65,509|
|General and administrative||9,927||5,166||25,809||12,619|
|Total operating expenses||45,955||30,692||130,703||78,128|
|Income (loss) from operations||(40,475)||3,208||(77,802)||(27,406)|
|Income (loss) before benefit for income taxes||(40,257)||3,256||(77,110)||(27,288)|
|Benefit for income taxes||--||(448)||--||(448)|
|Net loss per share– basic||$(1.07)||$0.11||$(2.06)||$(0.81)|
|Net loss per share– diluted||$(1.07)||$0.10||$(2.06)||$(0.81)|
|Weighted-average number of common shares used in net loss per share – basic||37,507,298||34,495,076||37,351,493||33,176,801|
|Weighted-average number of common shares used in net loss per share – diluted||37,507,298||36,592,683||37,351,493||33,176,801|
Note 1 (Collaboration revenue): The collaboration revenue decrease for the three months ended
Note 2 (R&D expense): During the first quarter of 2015, the Company began offsetting R&D expense for amounts received from
Agios Pharmaceuticals: Renee Leck, 617-649-8299 Senior Manager, Investor and Public Relations Renee.Leck@agios.com