Agios Pharmaceuticals Reports Second Quarter 2014 Financial Results
- AG-221: Company on track to start four Phase 1 expansion cohorts in second half of 2014; abstract of additional Phase 1 data submitted to the 2014 ASH Annual Meeting -
- AG-348: Phase 1 healthy volunteer studies met primary endpoints; abstract of Phase 1 data has been submitted to the 2014 ASH Annual Meeting -
"The first half of the year was defined by substantial progress in all three of our clinical development programs, and we anticipate this progress will continue through the remainder of 2014," said
"In the second half of the year, we submitted additional data for AG-221 and the first data for AG-348 for presentation at the 2014 Annual Meeting of the
SECOND QUARTER 2014 AND RECENT BUSINESS HIGHLIGHTS
Cancer Metabolism: IDH Mutant Inhibitors in Collaboration with
AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein
- Agios continues to enroll patients in the Phase 1 study of AG-221 with the primary goals of determining the maximum tolerated dose (MTD) and recommended Phase 2 dose.
Data presented in June at the 19th
Congressof the European Hematology Associationfrom the company's ongoing Phase 1 trial of AG-221 in 35 patients with IDH2-mutant positive advanced hematologic malignancies built upon those presented earlier this year at the American Association for Cancer Researchmeeting. Specifically, the data showed complete and durable remissions in patients with IDH2-mutant positive acute myelogenous leukemia (AML) and other advanced hematologic cancers, and a well-tolerated safety profile. Additional information on these data is available in a press release issued by Agios on June 14, 2014.
In June, Agios' collaboration partner
Celgeneexercised its option to an exclusive worldwide license to AG-221. Agios will continue to conduct early clinical development and regulatory activities within the development program in collaboration with Celgene.
Also in June, the
U.S. Food and Drug Administration( FDA) granted Agios Orphan Drug Designation for AG-221 for treatment of patients with AML. The FDAgrants Orphan Drug Designation to support development of medicines that affect fewer than 200,000 people in the U.S.
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein
- Agios continues to advance two separate Phase 1 trials evaluating AG-120 in patients with IDH1-mutant hematologic malignancies and IDH1-mutant advanced solid tumors. The Phase 1 trials are multicenter, open-label, dose-escalation clinical studies designed to assess the safety and tolerability of AG-120 as a single agent in these cancers.
- Agios' scientists and academic collaborators published nonclinical research in the July issue of the journal Nature showing that IDH mutations may drive tumorigenesis in a mouse model of intrahepatic cholangiocarcinoma (iCCA). iCCA is the second most common form of liver cancer.
Rare Genetic Disorders of Metabolism: Wholly Owned PKR Activator
AG-348: a novel, first-in-class, orally available activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency, a cause of hemolytic anemia
- Agios today announced that the Phase 1 single ascending dose (SAD) escalation trial for AG-348 in healthy volunteers (n=48) is complete and successfully met the primary endpoint of the study. The Phase 1 multiple ascending dose (MAD) escalation trial has also met its primary endpoint. Dose escalation is ongoing in healthy volunteers in the MAD study. The primary objectives as defined by the SAD and MAD study protocols are to identify a safe and pharmacodynamically active dose and schedule of AG-348 to be used in subsequent trials in patients with PK deficiency.
- A natural history study of PK deficiency is also ongoing with patient enrollment on track. Natural history studies are important to confirm and further understand clinical characteristics, symptoms and disease complications and potentially support the design of future clinical trials.
Agios submitted an abstract on its ongoing Phase 1 study of AG-221 for potential presentation at the upcoming 56th Annual Meeting of the
American Society of Hematology(ASH) in December. The company continues to enroll patients in the dose escalation portion of the Phase 1 study. The maximum tolerated dose has not been reached.
- Agios plans to select a dose and schedule from the ongoing Phase 1 study and initiate four expansion cohorts of 25 patients each in the second half of 2014. The expansion cohorts will evaluate relapsed or refractory AML patients 60 years of age and older and transplant in-eligible, relapsed or refractory AML patients under age 60, untreated AML patients who decline standard of care chemotherapy and patients with other IDH2-mutant positive hematologic malignancies.
- The company remains on track to initiate a Phase 1 study of AG-221 in patients with advanced solid tumors with an IDH2 mutation in the second half of 2014.
- The company expects to provide an update on the two Phase 1 studies evaluating patients with advanced hematologic malignancies and advanced solid tumors with an IDH1 mutation at medical conferences in 2015.
Rare Genetic Disorders of Metabolism
- Based on meeting the primary endpoints announced today in the Phase 1 healthy volunteer studies, the company believes it will be in a position to initiate a Phase 2 clinical trial for AG-348 by early 2015 in patients with PK deficiency.
- The company has submitted an abstract for the Phase 1 healthy volunteer studies for AG-348 to the ASH Annual Meeting in 2014.
- Agios expects to report initial data from its study of the natural history of PK deficiency at a medical conference in 2015.
Agios plans to host and webcast an investor event in
SECOND QUARTER 2014 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of
Collaboration revenue was
Research and development (R&D) expenses were
General and administrative (G&A) expenses were
Net loss for the second quarter of 2014 was
"Agios continues to maintain a strong balance sheet, which we strengthened in the second quarter by raising approximately
FINANCIAL GUIDANCE FOR THE FULL YEAR 2014
Agios is reiterating today that it expects to end 2014 with more than
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
About Acute Myelogenous Leukemia (AML)
AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. Undifferentiated blast cells proliferate in the bone marrow rather than maturing into normal blood cells. AML incidence significantly increases with age. Less than 10 percent of U.S. patients are eligible for bone marrow transplant, and the vast majority of patients do not respond to chemotherapy and progress to relapsed or refractory AML. AML prevalence is estimated to be approximately 115,000 to 160,000 patients worldwide, with approximately 20 percent of patients carrying an IDH mutation. The five-year survival rate for AML is approximately 20 to 25 percent.
About IDH Mutations and Cancer
IDH1 and IDH2 are two metabolic enzymes that are mutated in a wide range of hematologic and solid tumor malignancies. The prevalence of IDH mutations is expected to evolve as genomic analysis of tumors increase. Agios' research revealed the potential of IDH1 and IDH2 mutations as novel therapeutic targets in cancer, which may lead to clinical benefit for the subset of cancer patients whose tumors carry them. Patients carry either an IDH1 or IDH2 mutation, but not both.
Agios is developing two oral, first-in-class IDH mutant inhibitors: AG-221 is an IDH2 mutant inhibitor and AG-120 is an IDH1 mutant inhibitor. AG-221 is currently being evaluated in a Phase 1 dose-escalation study in patients with advanced hematologic malignancies. AG-120 is currently being evaluated in two Phase 1 trials, one in hematologic malignancies and another in solid tumors. Both compounds were discovered and developed in the laboratory of Agios.
About Pyruvate Kinase (PK) Deficiency, a Rare, Inherited Hemolytic Anemia
Pyruvate kinase (PK) deficiency, a rare, inherited hemolytic anemia affecting children and adults, is caused by mutations that affect the activity of the metabolic enzyme pyruvate kinase-R (PKR), the form of pyruvate kinase that is present in red blood cells. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, chelation therapy to address iron overload and/or interventions for other treatment, and disease-related morbidities. Currently, there is no approved therapy to treat the underlying cause of PK deficiency. AG-348 is a first-in-class orally available, potent, selective small molecule activator of PKR, which, when mutated, leads to PK deficiency. AG-348 was discovered in the laboratory of Agios, and the company retains worldwide development and commercialization rights.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements, including those regarding Agios' expectations and beliefs about: the potential of IDH1/IDH2 and pyruvate kinase-R mutations as therapeutic targets; the potential benefits of Agios' drug candidates targeting IDH1/IDH2 or pyruvate kinase-R mutations, including
|AGIOS PHARMACEUTICALS, INC.|
|Consolidated Balance Sheet Data (Unaudited)|
|June 30, 2014||December 31, 2013|
|Cash, cash equivalents and marketable securities||$ 261,111||$ 193,894|
|Deferred revenue – related party||60,341||57,639|
|Consolidated Statements of Operations Data (Unaudited)|
|(in thousands, except share and per share data)|
|Three Months Ended June 30,||Six Months Ended June 30,|
|Collaboration revenue – related party||$ 8,411||$ 6,268||$ 16,822||$ 12,536|
|Research and development||22,576||12,958||39,982||24,420|
|General and administrative||4,165||1,836||7,454||3,688|
|Total operating expenses||26,741||14,794||47,436||28,108|
|Loss from operations||(18,330)||(8,526)||(30,614)||(15,572)|
|Loss before provision for income taxes||(18,296)||(8,521)||(30,544)||(15,559)|
|Provision for income taxes||--||(99)||--||(289)|
|Cumulative preferred stock dividends||--||(1,798)||--||(3,595)|
|Net loss applicable to common stockholders||$ (18,296)||$ (10,418)||$ (30,544)||$ (19,443)|
|Net loss per share applicable to common stockholders – basic and diluted||$ (0.54 )||$ (2.80)||$ (0.94)||$ (5.27)|
|Weighted-average number of common shares used in net loss per share applicable to common stockholders – basic and diluted||33,602,472||3,722,963||32,506,739||3,690,669|
Agios Pharmaceuticals: Lora PikeSenior Director, Investor Relations and Public Relations Lora.Pike@agios.com 617-649-8608