Agios Reports Second Quarter 2017 Financial Results
– IDHIFA® (enasidenib) Granted Approval from
– Trial Designs Finalized for Two Pivotal Studies for AG-348 in Pyruvate Kinase Deficiency; Studies on Track to Begin in 1H2018 –
– Initiated Ivosidenib Phase 3 Study (AGILE) in Newly Diagnosed IDH1m AML; NDA Submission for R/R AML on Track for Year End 2017 –
“Our second quarter progress was followed by a remarkable milestone for any biotechnology company, the full approval of our first product, IDHIFA®, a treatment for patients with IDH2m R/R AML developed in partnership with Celgene,” said
SECOND QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS
IDH Mutant Inhibitors:
August 1, 2017, the U.S. Food and Drug Administration( FDA) granted Celgenefull approval of IDHIFA® (enasidenib) for the treatment of patients with relapsed or refractory AML (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDAapproved 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.
- Presented updated data from the Phase 1 trial of IDHIFA® in IDH2m R/R AML at the
American Society of Clinical Oncology( ASCO) Annual Meeting and updated data from the Phase 1/2 trial of IDHIFA® in IDH2m R/R AML at the 22nd Congressof the European Hematology Association(EHA). The data presentations demonstrated durable complete responses in patients with IDH2m R/R AML and a safety profile consistent with previously reported data. Read the ASCOdata here and the EHA data here.
- Presented the first data from the cholangiocarcinoma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at
ASCO. Read the ASCOdata here.
- Initiated a global, registration-enabling Phase 3 study (AGILE) combining ivosidenib and VIDAZA® in newly diagnosed AML patients with an IDH1 mutation ineligible for intensive chemotherapy.
- Completed enrollment of the dose-escalation phase of the ongoing Phase 1 study of AG-881 in IDHm positive glioma.
Rare Genetic Diseases
- 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 updated data from the AG-348 Phase 2 DRIVE PK trial in PK deficiency at EHA showing consistent safety and efficacy data. Read the EHA data here.
- In April, we entered into a new global license agreement with
Aurigene Discovery Technologies Limitedto research, develop and commercialize small molecule inhibitors for an undisclosed cancer metabolism target.
- In April, Agios completed an underwritten public offering of 5,808,080 shares of common stock, which included the full exercise of the underwriters’ option to purchase 757,575 shares, at the offering price of $49.50 per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $270.2 million.
EXPECTED 2H 2017 DATA PRESENTATIONS
- First preclinical data of AG-881 in IDHm solid and hematologic malignancies have been submitted for presentation at the
AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeuticsin October.
- Updated data from the glioma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors have been submitted for presentation at the 2017
Society for NeuroOncologyAnnual Meeting in November.
- First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies have been submitted for presentation at the 2017
American Society of HematologyAnnual Meeting and Exposition (ASH) in December.
- First data from the ongoing Phase 1 combination trial of IDHIFA® or ivosidenib with standard-of-care intensive chemotherapy (“7 +3” and consolidation) in patients with newly diagnosed AML with an IDH2 or IDH1 mutation have been submitted for presentation at ASH.
- Updated data from the Phase 2 DRIVE PK trial with AG-348 in patients with PK deficiency, including longer follow-up and secondary analyses, have been submitted for presentation at ASH.
- Updated data from the PK Deficiency Natural History Study being conducted with Boston Children’s Hospital have been submitted for presentation at ASH.
KEY UPCOMING MILESTONES
The company expects to achieve the following key milestones:
- Submit an NDA (New Drug Application) to the U.S.
FDAfor ivosidenib for IDH1m positive R/R AML by the end of 2017.
- Initiate two global, pivotal trials of AG-348 in PK deficiency in the first half of 2018.
- Submit an Investigational New Drug (IND) application for AG-270, the development candidate targeting MTAP-deleted tumors, by the end of 2017.
SECOND QUARTER 2017 FINANCIAL RESULTS
Collaboration revenue 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 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 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 Statement
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
|Cash, cash equivalents and marketable securities||$||715,941||$||573,564|
|Collaboration receivable – related party||4,842||4,886|
|Deferred revenue – related party||179,026||190,210|
|Consolidated Statements of Operations Data
(in thousands, except share and per share data)
|Three Months Ended
|Six Months Ended
|Collaboration revenue – related party||$||11,346||$||6,978||$||21,854||38,259|
|Research and development||79,816||50,804||142,548||94,842|
|General and administrative||16,130||12,644||30,953||23,481|
|Total operating expenses||95,946||63,448||173,501||118,323|
|Loss from operations||(84,600||)||(56,470||)||(151,647||)||(80,064||)|
|Net loss per share – basic and diluted||$||(1.78||)||$||(1.47||)||$||(3.35||)||$||(2.09||)|
|Weighted-average number of common shares used in
computing net loss per share – basic and diluted
Kendra Adams, 617-844-6407 Senior Director, Investor & Public Relations Kendra.Adams@agios.com Renee Leck, 617-649-8299 Senior Manager, Investor & Public Relations Renee.Leck@agios.com Media: Holly Manning, 617-844-6630 Associate Director, Corporate Communications Holly.Manning@agios.com