On August 4, 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 second quarter ended June 30, 2016 (Press release, Agios Pharmaceuticals, AUG 4, 2016, View Source;p=RssLanding&cat=news&id=2192919 [SID:1234514229]).
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"Second quarter achievements marked tremendous progress towards our 2016 goals across our research and development programs," said David Schenkein, M.D., chief executive officer at Agios. "At EHA (Free EHA Whitepaper), we established clear proof-of-concept for AG-348 in pyruvate kinase deficiency, validating our novel approach to treat rare genetic metabolic disorders by correcting the underlying enzymatic defect and potentially establishing the first treatment for patients with this serious disease. This milestone sets the stage for pivotal development of our PKR activator program. In addition, our new strategic collaboration with Celgene enables Agios to expand into the emerging field of metabolic immuno-oncology, an important new field of cancer research. With full worldwide rights for AG-120, we now have another wholly owned investigational medicine we discovered with the potential to benefit patients waiting for better therapies. As we move into the second half of the year, our focus is on the execution of our late-stage programs and several clinical updates across both IDH and PKR portfolios."
SECOND QUARTER 2016 HIGHLIGHTS & UPDATES
PKR Activator Program for the Treatment of Pyruvate Kinase Deficiency:
Data presented at the 21st Congress of the European Hematology Association (EHA) (Free EHA Whitepaper) in June validated Agios’ novel approach to the treatment of rare genetic metabolic disorders by correcting the underlying enzymatic defect with a small molecule.
AG-348 achieved proof-of-concept with rapid and sustained hemoglobin increases in the ongoing Phase 2 DRIVE-PK study of patients with pyruvate kinase (PK) deficiency. Read the AG-348 data from EHA (Free EHA Whitepaper) here.
Initial data from the AG-519 Phase 1 integrated single ascending dose (SAD) and multiple ascending dose (MAD) clinical trial in healthy volunteers provided proof-of-mechanism. As reported at EHA (Free EHA Whitepaper), one subject from the MAD portion of the study experienced a reversible Grade 2 platelet reduction consistent with drug-induced immune thrombocytopenia. Since the EHA (Free EHA Whitepaper) presentation data cut-off, 18 additional healthy volunteers have been dosed with AG-519 for 14 days and there have been no additional cases of thrombocytopenia. Read the AG-519 data from EHA (Free EHA Whitepaper) here.
Corporate:
In May, Agios and Celgene announced a new global strategic collaboration focused on metabolic immuno-oncology, an emerging field of cancer research focused on altering the metabolic state of immune cells to enhance the body’s immune response to cancer.
Agios received an upfront cash payment of $200 million and as a result updated expected 2016 ending cash, cash equivalents and marketable securities to more than $390 million. This revised cash balance is expected to be sufficient to fund Agios’ operating expenses and capital expenditure requirements through mid-2018.
The companies also amended certain rights from their 2010 collaboration agreement, including the transfer of ex-U.S. development and commercialization rights of AG-120 to Agios. For more information on the May 2016 agreement, read here.
2016 EXPECTED MILESTONES IN CANCER METABOLISM
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 relapsed/refractory acute myeloid leukemia (R/R AML) in the second half of the year
Plan to present updated data from the completed dose escalation portion of the AG-120 Phase 1 study in R/R AML in the second half of the year
Initiate an expansion arm in high-risk myelodysplastic syndrome patients for AG-221 in the second half of the year
Continue to enroll patients in the following ongoing clinical trials:
Phase 3 IDHENTIFY study of AG-221 vs. standard of care chemotherapy in R/R AML
Phase 1b frontline combination study of AG-221 or AG-120 with standard-of-care intensive chemotherapy in AML
Phase 1/2 frontline combination study of AG-221 or AG-120 with VIDAZA in AML
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive hematologic malignancies
IDH Mutant Inhibitors in Solid Tumors:
Plan to present data from the expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive low-grade glioma in the second half of the year
Initiate a randomized Phase 2 study of AG-120 in IDH1 mutant positive cholangiocarcinoma in the second half of the year
Continue to enroll patients in the following ongoing clinical trials:
Expansion phase of the ongoing Phase 1 study of AG-120 in advanced IDH1 mutant positive solid tumors
Phase 1 dose-escalation and expansion study of AG-881 in IDH mutant positive solid tumors
Cancer Metabolism Research:
Initiate preclinical development activities for the first molecule in a program focused on MTAP (methylthioadenosine phosphorylase) deleted cancers
2016 EXPECTED MILESTONES IN RARE GENETIC METABOLIC DISORDERS
Plan to present updated data from the AG-348 Phase 2 DRIVE-PK study, the AG-519 Phase 1 healthy volunteer study, and the Natural History Study of PK deficiency in the second half of the year
Provide a development strategy update for our PKR activator program, including molecule selection, in the second half of the year
Outline the clinical development plans for our PKR activators in beta-thalassemia in the second half of the year
SECOND QUARTER 2016 FINANCIAL RESULTS
Cash, cash equivalents and marketable securities as of June 30, 2016 were $512.3 million, compared to $375.9 million as of December 31, 2015. The increase in cash was driven by cash received from Celgene totaling $243.5 million, which included a $200 million upfront payment from the May 2016 collaboration agreement, $25 million related to a substantive clinical development milestone for the AG-221 program and $18.5 million of program funding related to our collaboration agreements. The cash received from Celgene was offset by a decrease in cash related to expenditures to fund operating activities of $104.7 million during the six months ended June 30, 2016.
Collaboration revenue was $7.0 million for the quarter ended June 30, 2016, compared to $13.2 million for the comparable period in 2015. For the second quarter of 2015, collaboration revenue included $8.8 million related to the delivery of the U.S and ex-U.S. licenses for AG-881.
Research and development (R&D) expense was $50.8 million, including $6.6 million of stock-based compensation expense, for the quarter ended June 30, 2016, compared to $36.4 million, including $4.6 million in stock-based compensation expense, for the quarter ended June 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 AG-221 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 $12.6 million, including $4.4 million of stock-based compensation expense, for the quarter ended June 30, 2016, compared to $8.9 million, including $3.6 million of stock-based compensation expense, for the quarter ended June 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 June 30, 2016 was $56.0 million, compared to a net loss of $31.9 million for the comparable period in 2015.