Agios Reports Second Quarter 2015 Financial Results and Provides Updates on Clinical Development Progress
Global Phase 3 Registration-enabling Studies on Track to Begin in Second Half of 2015 for AG-221 & First Half of 2016 for AG-120
Phase 1 Expansion Studies for AG-221 & AG-120 Programs Ongoing
"In the first half of 2015, we continued to drive rapid and meaningful progress across our development programs, as well as selecting our fourth molecule for development, AG-881, and advancing it into two Phase 1 studies," said
KEY UPCOMING MILESTONES
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
- Initiate a global Phase 3 registration-enabling study in relapsed/refractory acute myeloid leukemia (AML) patients who harbor an IDH2 mutation by the end of 2015.
- Initiate combination trials to evaluate AG-221 as a potential frontline treatment for patients with AML who harbor an IDH2 mutation by the end of 2015.
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein
- Begin combination trials to evaluate AG-120 as a potential frontline treatment for AML patients who harbor an IDH1 mutation by the end of 2015.
- Initiate a global registration-enabling Phase 3 study in AML patients who harbor an IDH1 mutation in the first half of 2016.
- Present data on the ongoing Phase 1 study of AG-120 in IDH1 mutant-positive advanced solid tumors at a medical meeting in the fourth quarter of 2015.
RECENT DEVELOPMENT UPDATES IN CANCER METABOLISM
Agios has provided the following updates on its clinical development programs in collaboration with
New data from the dose-escalation phase and expansion cohorts from the ongoing Phase 1 study evaluating single agent AG-221 were presented in June at the 20th
Congressof the European Hematology Association(EHA). Read the full AG-221 data here.
- As of July, Agios completed the dose-escalation portion of the Phase 1 study of AG-221 in IDH2 mutant-positive hematologic malignances. The expansion cohorts are on track, including the fifth expansion cohort of 125 patients with IDH2 mutant-positive AML who are in second or later relapse, refractory to second-line induction or re-induction treatment, or have relapsed after allogeneic transplantation.
- Dose escalation continues in the Phase 1/2 trial in patients with advanced solid tumors and angioimmunoblastic T-cell lymphoma (AITL) who carry an IDH2 mutation.
- New data from the dose-escalation phase of the ongoing Phase 1 study evaluating single agent AG-120 in advanced hematologic malignancies were presented in June at EHA. Read the full AG-120 data here.
Also announced in June, the
U.S. Food and Drug Administration( FDA) granted Agios orphan drug designation for AG-120 for the treatment of patients with IDH1 mutant-positive AML.
In May, Agios announced that the
FDAgranted Fast Track designation to AG-120 for the treatment of patients with IDH1 mutant-positive AML.
- As of July, Agios completed the dose-escalation portion of the Phase 1 study of AG-120 in IDH1 mutant-positive hematologic malignances. The three expansion cohorts to evaluate AG-120 in 175 patients with IDH1-mutated advanced hematologic malignancies, including one cohort with 125 patients with relapsed and/or refractory AML, are on track.
AG-881: a brain-penetrant, first-in-class, oral, potent pan-inhibitor of the mutated IDH1 and IDH2 proteins
- In June, Agios announced that the first patient was dosed in a Phase 1, open-label, dose-escalation and expansion study of single agent AG-881 in patients with IDH mutant-positive advanced solid tumors, including gliomas.
- In August, the first patient was dosed in a second dose-escalating and expansion trial for patients with IDH mutant-positive advanced hematologic malignancies, whose cancer has progressed on a prior IDH inhibitor therapy.
RECENT DEVELOPMENT UPDATES 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
- In June, final data from the Phase 1 multiple-ascending dose (MAD) study in healthy volunteers were presented at EHA, establishing clear proof-of-mechanism for AG-348. Read the full AG-348 data, as well as data from Boston Children's natural history study, here.
- Also in June, Agios initiated DRIVE PK, a global Phase 2, open-label safety and efficacy trial in adult, transfusion-independent patients with PK deficiency. The first patient was dosed in July.
Agios plans to host and webcast an investor event in
SECOND 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 second quarter of 2015 was
ADJUSTED FINANCIAL GUIDANCE FOR THE FULL YEAR 2015
Today Agios raised its previous year end cash guidance and now expects to end 2015 with more than
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
About Agios/Celgene Collaboration
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.
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|
|June 30,||December 31,|
|Cash, cash equivalents and marketable securities||$ 434,037||$ 467,447|
|Collaboration receivable – related party||10,474||6,492|
|Deferred revenue – related party||31,997||38,411|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended June 30,||Six Months Ended June 30,|
|Collaboration revenue – related party (1)||$13,219||$8,411||$47,421||$16,822|
|Research and development (2)||36,423||22,576||68,866||39,982|
|General and administrative||8,929||4,165||15,883||7,454|
|Total operating expenses||45,352||26,741||84,749||47,436|
|Loss from operations||(32,133)||(18,330)||(37,328)||(30,614)|
|Net loss per share– basic and diluted||$ (0.85)||$ (0.54)||$ (0.99)||$ (0.94)|
|Weighted-average number of common shares used in net loss per share applicable to common stockholders – basic and diluted||37,329,220||33,602,472||37,272,300||32,506,739|
Note 1 (Collaboration revenue): The collaboration revenue increase was primarily due to the application of new accounting guidance to the Company's collaboration arrangements with
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