On November 1, 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 third quarter ended September 30, 2017. In addition, Agios highlighted select corporate milestones and preclinical and clinical data from its development programs (Press release, Agios Pharmaceuticals, NOV 1, 2017, View Source [SID1234521379]).
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!
"We achieved two key 2017 goals in the third quarter with the approval and launch of IDHIFA with our partner Celgene for patients with IDH2m R/R AML and the design completion of the AG-348 pivotal program in PK deficiency," said David Schenkein, M.D., chief executive officer at Agios. "We are now focused on completing the NDA for our first wholly owned product, ivosidenib for IDH1m R/R AML and will present the core data from the submission next month at ASH (Free ASH Whitepaper). In addition, the year-end submission of our IND for AG-270 targeting MTAP-deleted tumors remains on track, highlighting our commitment to remain a research-focused organization pursuing novel science with the potential to change patients’ lives."
THIRD QUARTER 2017 HIGHLIGHTS & RECENT PROGRESS
• The U.S. Food and Drug Administration (FDA) granted Celgene full approval of IDHIFA (enasidenib) for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved diagnostic 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.
• 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.
LOGO
• Presented the first preclinical data for AG-881 in IDHm solid and hematologic malignancies 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. The data support that AG-881 potently suppresses 2-hydroxyglutarate (2-HG) production by both IDH1 (isocitrate dehydrogenase-1) and IDH2 mutant proteins in biochemical, cell-based and in vivo systems.
FOURTH QUARTER 2017 DATA PRESENTATIONS
IDH Mutant Inhibitors:
• 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 NeuroOncology Annual Meeting on November 17 in San Francisco.
• First data from the expansion phase of the ongoing Phase 1 trial of ivosidenib in IDH1m R/R AML and advanced hematologic malignancies at the 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH) (Free ASH Whitepaper) on December 9-12 in Atlanta.
• First data from the ongoing Phase 1 combination trial of enasidenib or ivosidenib with standard-of-care intensive chemotherapy ("7 +3" and consolidation) in patients with newly diagnosed AML with an IDH2 or IDH1 mutation at ASH (Free ASH Whitepaper).
• First 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 at ASH (Free ASH Whitepaper).
Rare Genetic Diseases:
• Updated data from the AG-348 Phase 2 DRIVE PK study in PK deficiency at ASH (Free ASH Whitepaper).
• Updated data from the PK Deficiency Natural History Study being conducted with Boston Children’s Hospital at ASH (Free ASH Whitepaper).
KEY UPCOMING MILESTONES
The company expects to achieve the following 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.
• Submit an Investigational New Drug (IND) application for AG-270, the development candidate targeting MTAP-deleted tumors, by the end of 2017.
• Initiate two global, pivotal trials of AG-348 in PK deficiency in the first half of 2018.
• Initiate a global registry for adult and pediatric patients with PK deficiency in the first half of 2018. The registry will include approximately 60 sites in 20 countries and will follow patients for at least two years.
• Initiate a perioperative ‘window’ study with ivosidenib and AG-881 in low grade glioma to further investigate their effects on brain tumor tissue in the first half of 2018.
LOGO
THIRD QUARTER 2017 FINANCIAL RESULTS
Revenue for the quarter ended September, 30, 2017 was $11.4 million, which includes $10.7 million of collaboration revenue and $0.7 million of royalty revenue from net sales of IDHIFA. Revenue for the comparable period in 2016, was $9.0 million. Revenue increased compared to the prior year period primarily due to reimbursement by Celgene of our share of the commercialization effort for IDHIFA and the IDHIFA royalty revenue.
Research and development (R&D) expense was $72.9 million, including $7.6 million of stock-based compensation expense, for the quarter ended September 30, 2017, compared to $60.6 million, including $7.9 million in stock-based compensation expense, for the quarter ended September 30, 2016. The increase in R&D expense was primarily attributable to the ivosidenib program, including 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. R&D expense also increased compared to the quarter ended September 30, 2016 due to IND enabling activities for AG-270 as well as ongoing research efforts across our discovery platform programs.
General and administrative (G&A) expense was $17.5 million, including $4.6 million stock-based compensation expense, for the quarter ended September 30, 2017, compared to $11.9 million, including $4.2 million of stock-based compensation expense, for the quarter ended September 30, 2016. The increase in G&A expense was attributed to an increase of $5.7 million related to support our growing commercial organization for the launch of IDHIFA and the potential launch of ivosidenib in 2018.
Net loss for the quarter ended September 30, 2017 was $77.1 million, compared to a net loss of $62.8 million for the quarter ended September 30, 2016.
Cash, cash equivalents and marketable securities as of September 30, 2017 were $641.7 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, $12.3 million of cost reimbursements related to our collaboration agreements with Celgene and $12.4 million received from employee stock transactions. This was offset by expenditures to fund operations of $226.4 million during the nine months ended September 30, 2017.
The company expects that its cash, cash equivalents and marketable securities as of September 30, 2017, together with anticipated interest income, anticipated expense reimbursements, and royalty 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.
LOGO
CONFERENCE CALL INFORMATION
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss third quarter 2017 financial results and recent business activities. To participate in the conference call, please dial 1-877-377-7098 (domestic) or 1-631-291-4547 (international) and refer to conference ID 99771605. The live webcast can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company’s website beginning approximately two hours after the event.