Agios Reports Fourth Quarter and Full Year 2017 Financial Results
– All 2017 Key Milestones Achieved, Including First U.S. Product Approval from Agios’ Discovery Platform and NDA Submission for Second Product Candidate, Ivosidenib, for
IDH1m R/R AML –
– 2018 Priorities Focus on Expanding Clinical and Research Programs to Drive Long-Term Value –
– 2017 Year-End Cash,
“2017 was an extraordinary year for Agios with the U.S. approval of IDHIFA®, our first internally discovered and developed drug, the NDA submission for our wholly owned medicine ivosidenib and our sixth IND submission since the company’s inception,” said
KEY UPCOMING MILESTONES
The company plans to achieve the following key milestones in 2018:
- Potential approval and commercialization of ivosidenib in
the United Statesfor relapsed/refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase-1 (IDH1) mutation in the third quarter of 2018.
- Potential submission of a Marketing Authorization Application (MAA) to the
European Medicines Agency(EMA) for ivosidenib for IDH1m R/R AML in the fourth quarter of 2018.
- Support, in conjunction with
Celgene, the initiation of an intergroup sponsored, global, registration-enabling Phase 3 trial combining ivosidenib or enasidenib with standard induction and consolidation chemotherapy in frontline AML patients with an IDH1 or IDH2 mutation in the fourth quarter of 2018.
- Initiate a perioperative 'window' trial with ivosidenib and AG-881 in low-grade glioma in the first quarter of 2018 to further investigate their effects on brain tumor tissue.
- Initiate a Phase 1 dose-escalation trial for AG-270, a first-in-class methionine adenosyltransferase 2a (MAT2A) inhibitor, in patients with methylthioadenosine phosphorylase (MTAP)-deleted tumors in the first quarter of 2018.
Rare Genetic Diseases:
- Initiate two global pivotal trials for AG-348 in pyruvate kinase (PK) deficiency in the first half of 2018:
- ACTIVATE-T: A single arm trial of approximately 20 regularly transfused patients is expected to initiate in the first quarter of 2018.
- ACTIVATE: A placebo-controlled trial of approximately 80 patients who do not receive regular transfusions is expected to initiate in the second quarter of 2018.
- Initiate a global registry, known as PEAK, for adult and pediatric patients with PK deficiency in the first quarter of 2018.
- Initiate a Phase 2 proof of concept trial of AG-348 in thalassemia in the fourth quarter of 2018.
- Submit an investigational new drug (IND) application for our newest development candidate, an inhibitor of the metabolic enzyme dihydroorotate dehydrogenase (DHODH) for the treatment of hematologic malignancies in the fourth quarter of 2018.
ANTICIPATED KEY 2018 DATA PRESENTATIONS
- Updated data from the expansion phase of the ongoing Phase 1 study of ivosidenib in IDH1m R/R AML has been submitted to the 2018
American Society of Clinical Oncology( ASCO) Annual Meeting.
- Updated 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 has been submitted to
- First clinical data from the Phase 1 study of AG-881 in advanced IDHm positive solid tumors, including glioma, has been submitted to
- Updated data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH2 or IDH1 mutation to be submitted to the 2018
American Society of Hematology(ASH) Annual Meeting and Exposition.
FOURTH QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS
- Completed an underwritten public offering in January of 8,152,986 shares of common stock at the offering price of
$67.00per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $516.2 million.
- Submitted a new drug application (NDA) to the
U.S. Food and Drug Administration( FDA) for ivosidenib for the treatment of patients with R/R AML with an IDH1 mutation.
FDAclearance of an IND application for AG-270, a MAT2A inhibitor, for the treatment of MTAP-deleted tumors.
- Presented new and updated data from the IDH and PKR programs at the 2017
American Society of HematologyAnnual Meeting and Exposition (ASH):
- First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies
- First data from the ongoing Phase 1 combination trial of ivosidenib or enasidenib with standard-of-care intensive chemotherapy in patients with newly diagnosed AML with an IDH1 or IDH2 mutation
- First data from the ongoing Phase 1/2 combination trial of ivosidenib or enasidenib with VIDAZA® in patients with newly diagnosed AML with an IDH1 or IDH2 mutation ineligible for intensive chemotherapy
- Updated data from the AG-348 Phase 2 DRIVE PK study in PK deficiency
- Appointed Jacqualyn “Jackie” Fouse, Ph.D., former president and chief financial officer of
Celgene, to Agios’ board of directors.
- Presented 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.
FULL YEAR 2017 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of
Revenue for the year ended
Research and development (R&D) expenses were $292.7 million, including $30.8 million of stock-based compensation expense, for the year ended December 31, 2017, compared to $220.2 million, including $25.4 million in stock-based compensation expense, for the year ended December 31, 2016. The increase in R&D expense was primarily attributable to the ivosidenib program, including manufacturing and regulatory activities to prepare the NDA submission, start-up costs for the Phase 3 AGILE clinical trial, and on-going site activation and patient enrollment of the Phase 3 ClarIDHy clinical trial. R&D expense also increased compared to the prior year due to IND enabling activities for AG-270.
General and administrative (G&A) expenses were $71.1 million, including $17.0 million of stock-based compensation expense, for the year ended December 31, 2017, compared to $50.7 million, including $16.7 million of stock-based compensation expense, for the year ended December 31, 2016. The increase in G&A expense was primarily attributable to an increase of
Net loss for the year ended December 31, 2017 was $314.7 million, compared to a net loss of $198.5 million for the year ended December 31, 2016.
In January, Agios completed an underwritten public offering of 8,152,986 shares of common stock, which includes the full exercise of the underwriters’ option to purchase an additional 1,063,433 shares, at the offering price of
The company expects that its cash, cash equivalents and marketable securities as of
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at
Agios is focused on discovering and developing novel investigational medicines to treat cancer and rare genetic diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has an approved oncology precision medicine and multiple first-in-class investigational therapies in clinical and/or preclinical development. All Agios programs focus on genetically identified patient populations, leveraging our knowledge of metabolism, biology and genomics. For more information, please visit the company's website at www.agios.com.
About Agios/Celgene Collaboration
IDHIFA® (enasidenib) and AG-881 are part of Agios' global strategic collaboration with
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding Agios’ plans, strategies and expectations for its and its collaborator’s preclinical, clinical and commercial advancement of its drug development programs including IDHIFA®, ivosidenib,
|Consolidated Balance Sheet Data|
|December 31, 2017||December 31, 2016|
|Cash, cash equivalents and marketable securities||$||567,750||$||573,564|
|Collaboration receivable – related party||2,448||4,886|
|Royalty receivable – related party||1,222||—|
|Deferred revenue – related party||163,640||190,210|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended December 31,||Years Ended December 31,|
|Collaboration revenue – related party||$||8,577||$||22,648||$||41,074||$||69,892|
|Royalty revenue – related party||1,222||—||1,937||—|
|Research and development, net||77,216||64,678||292,681||220,163|
|General and administrative||22,713||15,379||71,124||50,714|
|Total operating expenses||99,929||80,057||363,805||270,877|
|Loss from operations||(90,130||)||(57,409||)||(320,794||)||(200,985||)|
|Net loss per share – basic and diluted||$||(1.81||)||$||(1.34||)||$||(6.75||)||$||(5.07||)|
|Weighted-average number of common shares used in computing net loss per share – basic and diluted||48,772,901||42,110,541||46,587,631||39,126,400|
Senior Manager, Investor Relations
Associate Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.