On May 4, 2017 Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported business highlights and financial results for the first quarter ended March 31, 2017 (Press release, Agios Pharmaceuticals, MAY 4, 2017, View Source [SID1234518817]). In addition, Agios highlighted select corporate milestones and data presentations for its preclinical and clinical development programs.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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 David Schenkein, M.D., chief executive officer at Agios. "We are on track to execute against our remaining key 2017 goals, including finalizing the design of our global pivotal program for AG-348 in PK deficiency in the third quarter and submitting both an NDA for ivosidenib in R/R AML and our sixth IND, for AG-270, a development candidate focused on MTAP-deleted tumors, by the end of the year."

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.
On April 26, 2017, the FDA granted ivosidenib Orphan Drug Designation for the treatment of cholangiocarcinoma.
Rare Genetic Diseases:

On April 27, 2017, the FDA granted 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.
Research:

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.
Celgene designated 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 million milestone payment to Agios.
In April, Agios and Aurigene Discovery Technologies Limited entered into a global license agreement to research, develop and commercialize small molecule inhibitors of an undisclosed cancer metabolism target.
Corporate:

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 $49.50 per 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) (Free ASCO Whitepaper) Annual Meeting being held June 2-6, 2017 in Chicago
First data from the cholangiocarcinoma expansion cohort of the ongoing Phase 1 trial of ivosidenib in advanced IDH1m positive solid tumors at ASCO (Free ASCO Whitepaper)
Updated data from the AG-348 Phase 2 DRIVE PK trial in PK deficiency at the 22nd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) taking place June 22-25, 2017 in Madrid, Spain
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 Celgene with 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. FDA for 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.
Research:

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 $10.5 million for the quarter ended March 31, 2017, compared to $31.3 million for the comparable period in 2016. The decrease in revenue was primarily due to the fact that in the first quarter of 2016, the company recognized a $25 million milestone payment related to the initiation of the Phase 3 IDHENTIFY trial of IDHIFA (enasidenib).

Research and development (R&D) expense was $62.7 million, including $7.0 million of stock-based compensation expense, for the quarter ended March 31, 2017, compared to $44.0 million, including $5.5 million of stock-based compensation expense, for the quarter ended March 31, 2016. The increase in R&D expense was primarily due to increased costs to support advancement of the company’s lead investigational medicines toward later-stage development. Celgene is responsible for all development costs for IDHIFA (enasidenib) and certain development costs for AG-881, and reimburses Agios for development costs incurred for these investigational medicines. In addition, Celgene was responsible for approximately half of development costs for ivosidenib during the quarter ended March 31, 2016. As of August 2016, Agios is responsible for all ivosidenib development costs.

General and administrative (G&A) expense was $14.8 million, including $3.7 million of stock-based compensation expense, for the quarter ended March 31, 2017, compared to $10.8 million, including $3.6 million of stock-based compensation expense, for the quarter ended March 31, 2016. The increase in G&A expense was largely due to increased headcount and additional expenses to support our growing commercial organization.

Net loss for the quarter ended March 31, 2017 was $66.2 million, compared to a net loss of $23.2 million for the quarter ended March 31, 2016.

Cash & Cash Guidance

Cash, cash equivalents and marketable securities as of March 31, 2017 were $503.2 million, compared to $573.6 million as of December 31, 2016. The decrease in cash was driven by expenditures to fund operations of $79.4 million during the quarter ended March 31, 2017. These items were offset by an increase in cash of $5.0 million of program funding received under our collaboration agreements with Celgene and $4.1 million received from employee stock award transactions.

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 $49.50 per share, resulting in proceeds, net of underwriting discounts and commissions, of approximately $270.2 million.

The company expects that its cash, cash equivalents and marketable securities as of March 31, 2017, together with the net proceeds from the recent financing, anticipated interest income, and anticipated payments under our collaboration agreements, but excluding any additional program-specific milestone payments, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2019.