Agios Reports First Quarter 2017 Financial Results
– IDHIFA® (enasidenib) Granted Priority Review by
– Ivosidenib Phase 1 125-Patient Expansion Cohort in IDH1m R/R AML Fully Enrolled; NDA Submission on Track for Year End 2017 –
– AG-348 Granted Fast Track Designation in PK Deficiency by
“Our team made significant progress during the first quarter advancing our late-stage pipeline and preparing for the first product launch from our research and discovery engine, which is an important milestone for our organization,” said
FIRST QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS
IDH Mutant Inhibitors:
- A New Drug Application (NDA) was filed with the
U.S. Food and Drug Administration( FDA) for IDHIFA® (enasidenib) in relapsed and/or refractory (R/R) acute myeloid leukemia (AML) with an isocitrate dehydrogenase 2 (IDH2) mutation; the NDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of August 30, 2017.
- Completed enrollment of the 125-patient expansion cohort for the Phase 1 trial of ivosidenib in patients with IDH1m positive R/R AML.
April 26, 2017, the FDAgranted ivosidenib Orphan Drug Designation for the treatment of cholangiocarcinoma.
Rare Genetic Diseases:
April 27, 2017, the FDAgranted AG-348 Fast Track Designation for the treatment of patients with pyruvate kinase (PK) deficiency. Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
- Completed enrollment of 258 patients in the PK deficiency Natural History Study being conducted with Boston Children’s Hospital.
- Presented preclinical data on molecules with potential for the treatment of methylthioadenosine phosphorylase (MTAP) deleted tumors, at the Keystone Tumor Metabolism Meeting in
Whistler, British Columbia. Celgenedesignated AG-270 for the treatment of MTAP-deleted cancers as a development candidate under the master research and collaboration agreement dated May 17, 2016, triggering an $8 millionmilestone payment to Agios.
- In April,
Agios and Aurigene Discovery Technologies Limitedentered into a global license agreement to research, develop and commercialize small molecule inhibitors of an undisclosed cancer metabolism target.
- Also in April, Agios completed an underwritten public offering of 5,808,080 shares of common stock, which includes the full exercise of the underwriters’ option to purchase an additional 757,575 shares, at the offering price of
$49.50per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $270.2 million.
ANTICIPATED 2017 DATA PRESENTATIONS
- Updated data from the Phase 1/2 trial evaluating IDHIFA® (enasidenib) in patients with IDH2m positive R/R AML at the
American Society of Clinical Oncology( ASCO) Annual Meeting being held June 2-6, 2017in Chicago
- First data from the cholangiocarcinoma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at
- Updated data from the AG-348 Phase 2 DRIVE PK trial in PK deficiency at the 22nd Congress of the European Hematology Association (EHA) taking place June 22-25, 2017 in
- Updated data from DRIVE PK, including longer follow-up and secondary analyses, and updated data from the PK deficiency Natural History Study in the second half of 2017
- First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in R/R AML in the second half of 2017
- First data from the ongoing Phase 1b combination trial of IDHIFA® (enasidenib) or ivosidenib with standard-of-care intensive chemotherapy in newly diagnosed AML in the second half of 2017
- Updated data from the glioma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors in the second half of 2017
KEY UPCOMING MILESTONES
IDH Mutant Inhibitors in Hematologic Malignancies
- Potential approval and co-commercialization of IDHIFA® (enasidenib) in the U.S. for IDH2m positive R/R AML in collaboration with
Celgenewith a PDUFA action date of August 30, 2017.
- Initiate 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 in the first half of 2017.
- Submit an NDA to the U.S.
FDAfor ivosidenib for IDH1m positive R/R AML by the end of 2017.
IDH Mutant Inhibitors in Solid Tumors
- Complete enrollment of the dose-escalation phase of the ongoing Phase 1 study of AG-881 in IDHm positive glioma in the first half of 2017.
Rare Genetic Diseases
- Finalize design for a global pivotal trial of AG-348 in PK deficiency in the third quarter of 2017.
- Initiate a global pivotal trial 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.
FIRST 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 Guidance
Cash, cash equivalents and marketable securities as of
In April, Agios completed an underwritten public offering of 5,808,080 shares of common stock, which includes the full exercise of the underwriters’ option to purchase an additional 757,575 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 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||$||503,181||$||573,564|
|Collaboration receivable – related party||11,265||4,886|
|Deferred revenue – related party||188,286||190,210|
|Consolidated Statements of Operations Data
(in thousands, except share and per share data)
|Three Months Ended March 31,|
|Collaboration revenue – related party||$||10,508||$||31,281|
|Research and development||62,732||44,038|
|General and administrative||14,823||10,837|
|Total operating expenses||77,555||54,875|
|Loss from operations||(67,047)||(23,594)|
|Net loss per share – basic and diluted||$||(1.56)||$||(0.61)|
|Weighted-average number of common shares used in net loss per share applicable to common stockholders – basic and diluted||42,280,525||37,864,084|
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