Agios Reports First Quarter 2015 Financial Results and Outlines Late-Stage Clinical Development for AG-221 and AG-120
- AG-221: Ongoing Phase 1 study expanded by an additional 125 patients in selected AML (acute myeloid leukemia) populations
- AG-120: Ongoing Phase 1 hematologic malignancy study expanded by 175 patients in three cohorts, including 125 patients in a defined AML population
EHA: Abstracts for three lead clinical programs accepted for presentation at the 20th
Congressof the European Hematology Association(EHA)
"Leveraging the foundation we built in 2014 and our unique scientific approach, the expansion of the AG-221 and AG-120 trials in hematologic malignancies positions Agios as a late-stage clinical development company," said
Agios today provided the following updates on its clinical development programs in collaboration with
AG-221: a first-in-class, oral, selective, potent inhibitor of the mutated IDH2 protein
Added Additional Cohort to AG-221's Ongoing Phase 1 Expansion Study in Hematologic Malignancies
AG-221 is currently being evaluated in an ongoing Phase 1 trial that includes a dose escalation phase and four expansion cohorts of 25 patients each evaluating patients with relapsed or refractory AML who are 60 years of age and older and transplant ineligible, 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. Based on encouraging data from the dose escalation phase,
Celgeneand Agios have expanded the Phase 1 trial to add an additional more homogenous 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. Consistent with the ongoing expansion cohorts, AG-221 will be administered at a dose of 100 mg once daily. This multicenter, single-arm, open-label, expanded clinical trial will be conducted at the current treatment centers in the U.S. and France. The primary objectives of the study are to confirm the safety and clinical activity of AG-221 in a select, highly resistant AML population.
- Agios also continues to conduct a Phase 1/2 clinical trial evaluating AG-221 in IDH2-mutant positive advanced solid tumors, including gliomas, as well as angioimmunoblastic T-cell lymphoma (AITL).
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein
Initiated Three Expansion Cohorts in Ongoing Phase 1 Study in Hematologic Malignancies
AG-120 is currently being evaluated in an ongoing Phase 1 trial in patients with advanced hematologic malignancies. Three expansion cohorts have been added to this study and will evaluate AG-120 in 175 patients with IDH1 mutated advanced hematologic malignancies at approximately 15 clinical trial sites in the U.S. and
France. The first cohort will evaluate a more homogenous population of 125 AML patients who relapsed after bone marrow transplantation, are in second or later relapse, refractory to second line induction or reinduction treatment. The second cohort will evaluate 25 untreated AML patients, and the third cohort will evaluate 25 patients with IDH1 mutated advanced hematologic malignancies not eligible for cohorts one or two. AG-120 will be administered at a 500 mg once daily oral dose, in 28-day cycles. The study's primary objectives are to confirm the safety and clinical activity of AG-120.
- Agios also continues to advance a Phase 1 clinical trial evaluating AG-120 in patients with IDH1-mutant positive advanced solid tumors, including glioma.
"Our novel IDH inhibitors are advancing at an important time for cancer patients, as many do not respond to current standard of care chemotherapy," said
ADDITIONAL CLINICAL DEVELOPMENT AND BUSINESS HIGHLIGHTS
Cancer Metabolism: IDH Mutant Inhibitors in Collaboration with
AG-120: a first-in-class, oral, selective, potent inhibitor of the mutated IDH1 protein
Celgeneexercised its option to obtain an exclusive license outside the United Statesfor AG-120 in the first quarter.
AG-881: a brain-penetrant, pan-IDH mutant inhibitor
In April, Agios announced that it selected its third IDH mutant inhibitor, AG-881, for clinic development and entered into a new joint worldwide development and profit share collaboration with
Celgenefor this investigational medicine.
Rare Genetic Disorders of Metabolism: Wholly Owned PKR Activator
AG-348: a novel, first-in-class, oral activator of pyruvate kinase-R (PKR) for the treatment of pyruvate kinase (PK) deficiency
March, AG-348 was granted orphan drug designation for the treatment of PK deficiency by the U.S. Food and Drug Administration( FDA).
- A natural history study of PK deficiency is also ongoing. 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.
AG-221 Clinical development milestones in collaboration with
Present first data from Phase 1 hematological malignancy expansion cohorts at EHA in
Vienna, June 11-14, 2015.
- Initiate combination trials to evaluate AG-221 as a potential frontline treatment for patients with AML and a broad range of hematologic malignancies in the second half of 2015.
- Initiate a global Phase 3 registration-enabling study in relapsed/refractory AML patients that harbor an IDH2 mutation in the second half of 2015.
- Continue dose escalation in the Phase 1/2 trial in patients with advanced solid tumors or AITL that carry an IDH2 mutation in 2015.
AG-120 Clinical development milestones in collaboration with
- Present new data from the ongoing Phase 1 study evaluating patients with IDH1 mutant positive advanced hematologic malignancies at EHA.
- Present first data from the Phase 1 trial in advanced solid tumors at a medical conference in the second half of 2015.
- Begin combination trials to evaluate AG-120 as a potential frontline treatment of AML and a broad range of hematologic malignancies in the second half of 2015.
- Intend to initiate a global registration-enabling Phase 3 study in AML patients that harbor an IDH1 mutation in the first half of 2016.
AG-881: Clinical development milestones in collaboration with
- Initiate Phase 1 clinical development of AG-881 in the second quarter of 2015.
AG-348: Clinical development milestones for wholly owned PKR activator
- Present final data for the Phase 1 multiple ascending dose (MAD) clinical trial of AG-348 in healthy volunteers and the first data from a natural history study of PK deficiency, a rare hemolytic anemia at EHA.
Two abstracts submitted by
Boston Children's Hospitalon the natural history study have also been accepted for presentation at EHA.
- Expect to initiate a Phase 2 trial for AG-348 in the first half of 2015 in patients with PK deficiency.
FIRST QUARTER 2015 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of
Total revenue was
Research and development (R&D) expense was
General and administrative (G&A) expense was
Net loss for the first 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
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|
|March 31,||December 31,|
|Cash, cash equivalents and marketable securities||$ 439,964||$ 467,447|
|Collaboration receivable – related party||7,017||6,492|
|Deferred revenue – related party||7,019||38,411|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended March 31,|
|Collaboration revenue – related party (1)||$ 34,202||$ 8,411|
|Research and development (2)||32,443||17,407|
|General and administrative||6,954||3,288|
|Total operating expenses||39,397||20,695|
|Loss from operations||(5,195)||(12,284)|
|Net loss||$ (4,957)||$ (12,248)|
|Net loss per share– basic and diluted||$ (0.13)||$ (0.39)|
|Weighted-average number of common shares used in net loss per share – basic and diluted||37,214,747||31,394,563|
Note 1 (Collaboration revenue): The majority of the collaboration revenue increase was due to the application of new accounting guidance to the Company's 2010 collaboration agreement 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 Relations and Public Relations Renee.Leck@agios.com