On August 8, 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 second quarter ended June 30, 2017 (Filing, Q2, Agios Pharmaceuticals, 2017, AUG 8, 2017, View Source [SID1234520080]). In addition, Agios highlighted select corporate milestones and preclinical and clinical data from our clinical development programs .

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"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 David Schenkein, M.D., chief executive officer at Agios. "We are pursuing a similar regulatory strategy for ivosidenib, our wholly owned IDH1m inhibitor, and we remain on track to submit our NDA by year-end. With the pivotal program designed for AG-348 in PK deficiency, we are working to initiate two trials in the first half of 2018 for this rare anemia, which currently has no approved therapies."

SECOND QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS

IDH Mutant Inhibitors:

On August 1, 2017, the U.S. Food and Drug Administration (FDA) granted Celgene full 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 FDA approved 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) (Free ASCO Whitepaper) Annual Meeting and updated data from the Phase 1/2 trial of IDHIFA in IDH2m R/R AML at the 22ndCongress of the European Hematology Association (EHA) (Free EHA Whitepaper). 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 ASCO (Free ASCO Whitepaper) data here and the EHA (Free EHA Whitepaper) 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 (Free ASCO Whitepaper). Read the ASCO (Free ASCO Whitepaper) data 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 (Free EHA Whitepaper) showing consistent safety and efficacy data. Read the EHA (Free EHA Whitepaper) data here.
Discovery Research

In April, we entered into a new global license agreement with Aurigene Discovery Technologies Limited to research, develop and commercialize small molecule inhibitors for an undisclosed cancer metabolism target.
Corporate:

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 (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in 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 NeuroOncology Annual 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 Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper) 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 (Free ASH Whitepaper).
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 (Free ASH Whitepaper).
Updated data from the PK Deficiency Natural History Study being conducted with Boston Children’s Hospital have been submitted for presentation at ASH (Free ASH Whitepaper).
KEY UPCOMING MILESTONES

The company expects to achieve the following key milestones:

Submit an NDA (New Drug Application) to the U.S. FDA for 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 $11.3 million for the quarter ended June 30, 2017, compared to $7.0 million for the comparable period in 2016. Collaboration revenue increased compared to the prior year period partially due to reimbursement by Celgene of our share of the commercialization effort for IDHIFA.

Research and development (R&D) expense was $79.8 million, including $8.2 million of stock-based compensation expense, for the quarter ended June 30, 2017, compared to $50.8 million, including $6.6 million in stock-based compensation expense, for the quarter ended June 30, 2016. The increase in R&D expense was primarily attributable to activities related to the ivosidenib program, including manufacturing-related activities needed to prepare for a potential NDA submission in 2017, 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. In addition, Celgene was responsible for approximately half of the development costs for ivosidenib during the quarter ended June 30, 2016. As of August 2016, Agios is responsible for all ivosidenib development costs. R&D expense also increased compared to the quarter ended June 30, 2016 due to preparations for initiation of the AG-348 pivotal program in the first half of 2018, including manufacturing-related activities. Lastly, the $3.0 million upfront payment as part of the Aurigene license agreement was included in R&D during the quarter ended June 30, 2017.

General and administrative (G&A) expense was $16.1 million, including $4.0 million stock-based compensation expense, for the quarter ended June 30, 2017, compared to $12.6 million, including $4.4 million of stock-based compensation expense, for the quarter ended June 30, 2016. The increase in G&A expense was attributed to an increase of $1.0 million in personnel costs related to an increase in our internal headcount and an increase of $1.9 million related to support our growing commercial organization.

Net loss for the quarter ended June 30, 2017 was $83.1 million, compared to a net loss of $56.0 million for the quarter ended June 30, 2016.

Cash, cash equivalents and marketable securities as of June 30, 2017 were $715.9 million, compared to $573.6 million as of December 31, 2016. The increase in cash was driven by net proceeds of $270.2 million from the April financing, $8.1 million of program funding received under our collaboration agreements with Celgene and $6.8 million received from employee stock award transactions. This was offset by expenditures to fund operations of $142.5 million during the six months ended June 30, 2017.

The company expects that its cash, cash equivalents and marketable securities as of June 30, 2017, together with anticipated interest income, and anticipated expense reimbursements 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.