Agios Reports Third Quarter 2017 Financial Results
IDHIFA® Approval for IDH2m R/R AML Validates Precision Medicine Approach; Ivosidenib NDA Submission for IDH1m R/R AML on Track for Year End 2017
First Expansion Data from Ivosidenib Phase 1 Trial in IDH1m R/R AML and
IND Submission for AG-270 Targeting MTAP-deleted Tumors on Track for Year End 2017
“We achieved two key 2017 goals in the third quarter with the approval and launch of IDHIFA® with our partner
THIRD QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS
The U.S. Food and Drug Administration( FDA) granted Celgenefull approval of IDHIFA® (enasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved diagnostic test. IDHIFA®, an oral targeted inhibitor of the IDH2 enzyme, is the first and only FDA-approved therapy for patients with R/R AML and an IDH2 mutation.
- Finalized two global, pivotal trial designs evaluating AG-348 in adults with pyruvate kinase (PK) deficiency:
- A randomized, placebo-controlled trial with a 1:1 randomization expected to enroll approximately 80-100 non-transfusion dependent patients. The primary endpoint of the study is the proportion of patients who achieve at least a 1.5 gram per deciliter (g/dL) increase in hemoglobin.
- A single arm trial of approximately 20 regularly transfused patients with a primary endpoint of reduction in transfusion burden over six months.
- Presented the first preclinical data for AG-881 in IDHm solid and hematologic malignancies at the
AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeuticsin October. The data support that AG-881 potently suppresses 2-hydroxyglutarate (2-HG) production by both IDH1 (isocitrate dehydrogenase-1) and IDH2 mutant proteins in biochemical, cell-based and in vivo systems.
FOURTH QUARTER 2017 DATA PRESENTATIONS
IDH Mutant Inhibitors:
- Updated data from the glioma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at the 2017
Society for NeuroOncologyAnnual Meeting on November 17in San Francisco.
- First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies at the 2017
American Society of HematologyAnnual Meeting and Exposition (ASH) on December 9-12in Atlanta.
- First data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy (“7 +3” and consolidation) in patients with newly diagnosed AML with an IDH2 or IDH1 mutation at ASH.
- First data from the ongoing Phase 1/2 combination trial of enasidenib or ivosidenib with VIDAZA® in patients with newly diagnosed AML with an IDH2 or IDH1 mutation ineligible for intensive chemotherapy at ASH.
Rare Genetic Diseases:
- Updated data from the AG-348 Phase 2 DRIVE PK study in PK deficiency at ASH.
- Updated data from the PK Deficiency Natural History Study being conducted with Boston Children’s Hospital at ASH.
KEY UPCOMING MILESTONES
The company expects to achieve the following milestones:
- Submit an NDA (New Drug Application) to the U.S.
FDAfor ivosidenib for IDH1m positive R/R AML by the end of 2017.
- Submit an Investigational New Drug (IND) application for AG-270, the development candidate targeting MTAP-deleted tumors, by the end of 2017.
- Initiate two global, pivotal trials of AG-348 in PK deficiency in the first half of 2018.
- Initiate a global registry for adult and pediatric patients with PK deficiency in the first half of 2018. The registry will include approximately 60 sites in 20 countries and will follow patients for at least two years.
- Initiate a perioperative ‘window’ study with ivosidenib and AG-881 in low grade glioma to further investigate their effects on brain tumor tissue in the first half of 2018.
THIRD QUARTER 2017 FINANCIAL RESULTS
Revenue for the quarter ended September, 30, 2017 was
Research and development (R&D) expense was
General and administrative (G&A) expense was
Net loss for the quarter ended
Cash, cash equivalents and marketable securities as of
The company expects that its cash, cash equivalents and marketable securities as of
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
IDHIFA® (enasidenib) is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.
Important Safety Information
|WARNING: DIFFERENTIATION SYNDROME|
|Patients treated with IDHIFA have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, lymphadenopathy, bone pain, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.|
WARNINGS AND PRECAUTIONS
Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 14% of patients treated with IDHIFA experienced differentiation syndrome. Differentiation syndrome has been observed with and without concomitant hyperleukocytosis, as early as 10 days and at up to 5 months after IDHIFA initiation. If differentiation syndrome is suspected, initiate systemic corticosteroids and hemodynamic monitoring until improvement. Taper corticosteroids only after resolution of symptoms. Differentiation syndrome symptoms may recur with premature discontinuation of corticosteroids. If severe pulmonary symptoms requiring intubation or ventilator support and/or renal dysfunction persist for more than 48 hours after initiation of corticosteroids, interrupt IDHIFA until signs and symptoms are no longer severe. Hospitalization for close observation and monitoring of patients with pulmonary and/or renal manifestation is recommended.
Embryo-Fetal Toxicity: Based on animal embryo-fetal toxicity studies, IDHIFA can cause embryo-fetal harm when administered to a pregnant woman. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with IDHIFA and for at least 1 month after the last dose. Pregnant women, patients becoming pregnant while receiving IDHIFA, or male patients with pregnant female partners should be apprised of the potential risk to the fetus.
- The most common adverse reactions (≥20%) included total bilirubin increased (81%), calcium decreased (74%), nausea (50%), diarrhea (43%), potassium decreased (41%), vomiting (34%), decreased appetite (34%), and phosphorus decreased (27%)
- The most frequently reported ≥Grade 3 adverse reactions (≥5%) included total bilirubin increased (15%), potassium decreased (15%), phosphorus decreased (8%), calcium decreased (8%), diarrhea (8%), differentiation syndrome (7%), non-infectious leukocytosis (6%), tumor lysis syndrome (6%), and nausea (5%)
- Serious adverse reactions were reported in 77.1% of patients. The most frequent serious adverse reactions (≥2%) were leukocytosis (10%), diarrhea (6%), nausea (5%), vomiting (3%), decreased appetite (3%), tumor lysis syndrome (5%), and differentiation syndrome (8%). Differentiation syndrome events characterized as serious included pyrexia, renal failure acute, hypoxia, respiratory failure, and multi-organ failure
Many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed infants, advise women not to breastfeed during treatment with IDHIFA and for at least 1 month after the last dose.
Please see full Prescribing Information, including Boxed WARNING
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has an approved oncology precision medicine and multiple first-in-class investigational therapies 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 www.agios.com.
About Agios/Celgene Collaboration
IDHIFA® (enasidenib) and AG-881 are part of Agios' global strategic collaboration with
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 Agios’ plans, strategies and expectations for its and its collaborator’s preclinical, clinical and commercial advancement of its drug development programs including IDHIFA® (enasidenib), ivosidenib,
|Consolidated Balance Sheet Data|
|September 30, 2017||December 31, 2016|
|Cash, cash equivalents, and marketable securities||$||641,732||$||573,564|
|Collaboration receivable – related party||3,489||4,886|
|Royalty receivable – related party||715||—|
|Deferred revenue – related party||170,069||190,210|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Collaboration revenue – related party||$||10,643||$||8,985||$||32,497||$||47,244|
|Royalty revenue – related party||715||—||715||—|
|Research and development||72,917||60,643||215,465||155,485|
|General and administrative||17,458||11,854||48,411||35,335|
|Total operating expenses||90,375||72,497||263,876||190,820|
|Loss from operations||(79,017||)||(63,512||)||(230,664||)||(143,576||)|
|Net loss per share – basic and diluted||$||(1.59||)||$||(1.63||)||$||(4.94||)||$||(3.72||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||48,459,424||38,548,153||45,851,203||38,124,425|
Senior Manager, Investor & Public Relations
Associate Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.