On November 3, 2016 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of cancer metabolism and rare genetic metabolic disorders, reported business highlights and financial results for the third quarter ended September 30, 2016 (Filing, Q3, Agios Pharmaceuticals, 2016, NOV 3, 2016, View Source [SID1234516180]).
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"We have made significant progress during 2016, establishing proof of concept with our lead pyruvate kinase-R (PKR) activator, executing late-stage clinical development for both of our lead isocitrate dehydrogenase (IDH) mutant inhibitors in hematologic malignancies and strengthening our balance sheet through our recent financing," said David Schenkein, M.D., chief executive officer at Agios. "As we head into year end, we are focused on supporting the enasidenib NDA submission with our partner Celgene and planning for our next steps in clinical development for our PKR and IDH portfolios based on data we will present at a number of upcoming medical meetings this quarter."
THIRD QUARTER 2016 HIGHLIGHTS & UPDATES
IDH Mutant Inhibitors in Hematologic Malignancies:
In September, Agios and Celgene announced plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration for enasidenib (AG-221) in relapsed and/or refractory (R/R) acute myeloid leukemia (AML) with an IDH2 mutation by year-end. The NDA will be based on data from an ongoing Phase 1/2 trial in patients with relapsed and/or refractory AML and other advanced hematologic malignancies with an IDH2 mutation.
Agios plans to explore a similar regulatory path for AG-120, its wholly owned, first-in-class, oral, potent inhibitor of mutant IDH1, which could lead to a NDA submission in 2017 in the U.S.
Corporate:
In September, Agios completed an underwritten public offering of common stock for 3,876,403 shares at the offering price of $44.50 per share, resulting in gross proceeds of approximately $173 million.
Agios recently announced the appointment of Andrew Hirsch to chief financial officer. Mr. Hirsch has more than 20 years of experience in a range of strategic and operating roles. He most recently served as president and chief executive officer of BIND Therapeutics. Prior to joining BIND, he was chief financial officer at Avila Therapeutics until its acquisition by Celgene and held roles of increasing responsibility during his nearly 10-year tenure at Biogen.
UPCOMING MEDICAL MEETING PRESENTATIONS
• First data from the expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive chondrosarcoma at the Connective Tissue Oncology Society (CTOS) Annual Meeting on November 10, 2016 in Lisbon, Portugal.
• Preliminary data from the expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive low-grade glioma at the Society for NeuroOncology (SNO) Annual Meeting on November 18, 2016 in Scottsdale, AZ.
• Updated data from the AG-348 Phase 2 DRIVE-PK study, the AG-519 Phase 1 healthy volunteer study, the Natural History Study of pyruvate kinase deficiency (PKD), and new AG-348 metabolic data in PKD patients at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on December 3-6, 2016 in San Diego, CA.
• Updated follow-up and molecular data from the completed dose escalation portion of the AG-120 Phase 1 study in R/R AML at ASH (Free ASH Whitepaper).
ADDITIONAL 2016 EXPECTED MILESTONES
IDH Mutant Inhibitors in Hematologic Malignancies:
• Complete enrollment of the 125-patient expansion cohort for the Phase 1 study of AG-120 in patients with R/R AML
IDH Mutant Inhibitors in Solid Tumors:
• Initiate a randomized study of AG-120 in IDH1 mutant positive cholangiocarcinoma
Cancer Metabolism Research:
• Initiate preclinical development activities for the first molecule in a program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers
Rare Genetic Metabolic Disorders:
• Provide a development strategy update for our PKR activator program, including molecule selection
• Outline the clinical development plans for our PKR activators in beta-thalassemia
THIRD QUARTER 2016 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of September 30, 2016 were $622.6 million, compared to $375.9 million as of December 31, 2015. The increase in cash was driven by cash received from Celgene totaling $251.5 million, which includes a $200 million upfront payment from the May 2016 collaboration agreement, $25 million related to initiation of the enasidenib Phase 3 IDHENTIFY study and $26.5 million of program funding related to our collaboration agreements, and net proceeds of $162.1 million received from the company’s September 2016 public offering. These items were offset by a decrease in cash related to expenditures to fund operating activities of $161.7 million and purchases of fixed assets, net of reimbursements, of $4.3 million during the nine months ended September 30, 2016.
Collaboration revenue was $9.0 million for the quarter ended September 30, 2016, compared to $5.5 million for the comparable period in 2015.
Research and development (R&D) expense was $60.6 million, including $7.9 million of stock-based compensation expense, for the quarter ended September 30, 2016, compared to $36.0 million, including $4.9 million in stock-based compensation expense, for the quarter ended September 30, 2015. 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 enasidenib and certain development costs for AG-881 and reimburses the company for development costs incurred for these investigational medicines.
General and administrative (G&A) expense was $11.9 million, including $4.2 million of stock-based compensation expense, for the quarter ended September 30, 2016, compared to $9.9 million, including $4.5 million of stock-based compensation expense, for the quarter ended September 30, 2015. The increase in G&A expense was largely due to increased headcount and other professional expenses to support growing operations.
Net loss for the quarter ended September 30, 2016 was $62.8 million, compared to a net loss of $40.3 million for the comparable period in 2015.
UPDATED FINANCIAL GUIDANCE FOR THE FULL YEAR 2016
As a result of the recent financing, Agios now expects to end 2016 with more than $550 million of cash, cash equivalents and marketable securities. The anticipated year-end 2016 cash position does not include any additional program-specific milestone payments from Celgene. Based on its current operating plans, the company expects that its existing cash, cash equivalents and marketable securities as of September 30, 2016, together with anticipated interest income, and anticipated expense reimbursements under our collaboration agreements with Celgene, but excluding any additional program-specific milestone payments from Celgene, will enable the company to fund its anticipated operating expenses and capital expenditure requirements through at least the end of 2018.