Agios Pharmaceuticals Reports First Quarter 2014 Financial Results
AG-221 Showed Promising Clinical Activity in AML; Additional Data to be Presented at EHA
Three First-in-Class Compounds now in Clinical Development
“In the first quarter, we made significant progress in the development
and advancement of all our programs,” said
Recent Business Highlights
Cancer Metabolism: IDH Mutant Inhibitors in Collaboration with
In April, during the clinical trial symposium at the
American Association of Cancer Research(AACR) Annual Meeting 2014, investigators presented preliminary data from the first two cohorts in the ongoing Phase 1 study of AG-221 in 10 patients with advanced IDH2 mutant positive hematologic malignancies. Initial data showed that AG-221 was well tolerated when dosed at 30 mg and 50 mg twice daily in 28 day continuous cycles. Promising efficacy was observed with six of seven evaluable patients having objective responses, including three complete remissions and two complete remissions with incomplete platelet recovery. Additional information on the preliminary data is available in a press release issued by Agios on April 6, 2014.
- In March, Agios announced initiation of two Phase 1 studies of AG-120, one in advanced hematologic malignancies and one in advanced solid tumors, including glioma. These multi-center, open-label, dose-escalation studies are evaluating the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in patients whose advanced cancer harbor an IDH1 mutation.
IEMs: Wholly Owned Asset Targeting PK Deficiency
- In April, Agios announced dose administration of AG-348, a first-in-class pyruvate kinase (PK) R activator, in a Phase 1 healthy volunteer study. The single-center, randomized, double-blind, placebo-controlled clinical trial is assessing the safety and tolerability, as well as pharmacokinetics and pharmacodynamics, of AG-348 through a single ascending dose escalation in healthy adults. This will be the first of two healthy volunteer studies designed to enable a proof of concept study in patients with pyruvate kinase deficiency.
- A natural history study of PK deficiency is also ongoing. Natural history studies represent an important tool for understanding clinical characteristics and disease progression and help support the design of future clinical trials.
- Agios continues to dose escalate its Phase 1 study of AG-221, and expects to initiate expansion cohorts by the end of 2014.
A late-breaker abstract on an update from the dose-escalation portion
of the ongoing Phase 1 study of AG-221 has been accepted for oral
presentation at the 19th
Congressof the European Hematology Association(EHA) in Milan, Italy, June 12-15, 2014.
- Enrollment remains on track for the AG-120 clinical trials, and the company anticipates providing updates on both trials at medical conferences in 2015.
- Agios expects to start a multiple-ascending dose escalation Phase 1 clinical trial of AG-348 in healthy volunteers in mid-2014, and to report data for the healthy volunteer trials of AG-348 and interim data from the natural history study at a medical conference in 2015.
First Quarter 2014 Financial Results
Cash, cash equivalents and marketable securities as of
Total revenue was
Research and development (R&D) expenses were
General and administrative (G&A) expenses were
Net loss for the first quarter of 2014 was
“Following the close of our recent equity offering, we believe we’re
well positioned financially to fund our current development and research
Financial Guidance for the Full Year 2014
Agios 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 mature 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 / 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 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 PK Deficiency
Pyruvate kinase (PK) deficiency, a rare, inherited hemolytic anemia, is caused by mutations that affect the activity of the metabolic enzyme pyruvate kinase-R (PK-R), the form of pyruvate kinase that is present in red blood cell counts. 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 PK-R, 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’ product candidates targeting IDH1/IDH2 or
pyruvate kinase-R mutations, including
AGIOS PHARMACEUTICALS, INC.
Consolidated Balance Sheet Data (Unaudited)
|Cash and cash equivalents, and marketable securities||
Consolidated Statements of Operations Data (Unaudited)
(in thousands, except share and per share data)
|Three Months Ended March 31,|
|Research and development||17,407||11,462|
|General and administrative||3,288||1,852|
|Total operating expenses||20,695||13,314|
|Loss from operations||(12,284)||(7,046)|
|Loss before provision for income taxes||(12,248)||(7,038)|
|Provision for income taxes||—||190|
|Cumulative preferred stock dividends||—||(1,797)|
|Net loss applicable to common stockholders||$||(12,248)||$||(9,025)|
|Net loss per share applicable to common stockholders – basic and diluted||$||(0.39)||$||(2.47)|
Weighted-average number of common shares used in net loss per share applicable to common stockholders – basic and diluted
Agios Pharmaceuticals, Inc.
Lora Pike, 617-649-8608
Senior Director, Investor Relations and Public Relations