On October 31, 2019 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, 2019 (Press release, Agios Pharmaceuticals, OCT 31, 2019, View Source [SID1234550097]).
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"The third quarter was marked by strong commercial execution, now a full year since the U.S. launch of TIBSOVO, and several meaningful clinical updates spanning our early and late stage oncology programs that advance our strategy," said Jackie Fouse, Ph.D., chief executive officer at Agios. "As we look ahead to the remainder of 2019, we are focused on achieving our remaining key milestones, including completing enrollment in our mitapivat PK deficiency pivotal program, establishing proof of concept for mitapivat in thalassemia, initiating our pivotal trial of vorasidenib in low-grade glioma and submission of the TIBSOVO supplemental new drug application for IDH1 mutant cholangiocarcinoma. These milestones are critical steps toward realizing the value-creation potential for both our oncology and rare genetic disease portfolios in 2020 and beyond."
THIRD QUARTER 2019 HIGHLIGHTS & RECENT PROGRESS
Presented data from the single agent dose-escalation portion of the ongoing Phase 1 study of AG-270 in patients with methylthioadenosine phosphorylase (MTAP)-deleted tumors 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 demonstrated that AG-270 induces reductions in the biomarkers of methionine adenosyltransferase 2A (MAT2A) inhibition, notably plasma concentrations of S-adenosylmethionine (SAM) and tumor levels of symmetrically demethylated arginine (SDMA), at well tolerated doses.
Presented results from the Phase 3 ClarIDHy study of TIBSOVO in previously treated isocitrate dehydrogenase-1 (IDH1) mutant cholangiocarcinoma at the European Society for Medical Oncology Congress in September, demonstrating significant improvement in progression free survival compared to placebo.
Initiated two combination arms for the Phase 1 study of AG-270 in MTAP-deleted tumors, one evaluating AG-270 in combination with docetaxel in MTAP-deleted second-line non-small cell lung cancer and another in combination with nab-paclitaxel and gemcitabine in MTAP-deleted first or second-line pancreatic ductal adenocarcinoma.
Published new data from the core and extension phases of the DRIVE PK Phase 2 study of mitapivat in adults with pyruvate kinase (PK) deficiency in the September 5, 2019 issue of the New England Journal of Medicine, demonstrating sustained increases in hemoglobin for up to 35 months.
KEY UPCOMING MILESTONES
The company plans to achieve the following key milestones in the remainder of 2019:
Oncology:
Submit a supplemental new drug application to the FDA for TIBSOVO for previously treated IDH1 mutant cholangiocarcinoma by year-end.
Initiate the registration-enabling Phase 3 INDIGO study of vorasidenib in Grade 2 non-enhancing glioma with an IDH mutation by year-end. The study will evaluate 366 patients in 1:1 double-blind randomization to either 50 mg of vorasidenib once daily or placebo. The primary endpoint is progression free survival.
Rare Genetic Diseases:
Complete enrollment in two global pivotal trials for mitapivat in adults with PK deficiency by year-end:
ACTIVATE-T: A single-arm trial of up to 40 regularly transfused patients
ACTIVATE: A 1:1 randomized, placebo-controlled trial of up to 80 patients who do not receive regular transfusions
Achieve proof-of-concept for mitapivat in thalassemia in the second half of 2019.
REMAINING 2019 DATA PRESENTATIONS
Updated data from the perioperative study of TIBSOVO and vorasidenib in low-grade glioma have been accepted for presentation at the Society for Neuro-Oncology Annual Meeting taking place in Phoenix from November 22-24, 2019.
Data from the IDH and PKR programs have been accepted for presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting taking place in Orlando, Fla. from December 7-10, 2019, including new data from the extension phase of the Phase 2 DRIVE PK study of mitapivat in adults with PK deficiency and important translational data from the Phase 1 combination study of TIBSOVO and azacitidine in frontline acute myeloid leukemia (AML).
THIRD QUARTER 2019 FINANCIAL RESULTS
Revenue: Total revenue for the third quarter of 2019 was $26.0 million, which includes $17.4 million of net product revenue from U.S. sales of TIBSOVO, $5.5 million in collaboration revenue and $2.7 million in royalty revenue from net global sales of IDHIFA under our collaboration agreement with Celgene. This compares to total revenue of $15.2 million for the third quarter of 2018.
Cost of Sales: Cost of sales were $0.4 million for the third quarter of 2019.
Research and Development (R&D) Expenses: R&D expenses were $101.7 million for the third quarter of 2019 compared to $82.6 million for the third quarter of 2018. The increase in R&D expense was primarily attributable to start-up costs for the Phase 3 INDIGO study of vorasidenib in IDH mutated low-grade glioma, the mitapivat pivotal program in PK deficiency and Phase 2 study in thalassemia, and costs of the ongoing Phase 3 TIBSOVO combination trials in the frontline AML setting.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $33.0 million for the third quarter of 2019 compared to $31.1 million for the third quarter of 2018.
Net Loss: Net loss was $106.2 million for the third quarter of 2019 compared to $94.7 million for the third quarter of 2018.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2019 were $540.5 million compared to $805.4 million as of December 31, 2018. The net decrease of $264.9 million in cash position was primarily driven by net expenditures to fund operations. The company expects that its cash, cash equivalents and marketable securities as of September 30, 2019, together with anticipated product and royalty revenue, anticipated interest income, and anticipated expense reimbursements under our collaboration and license 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 2020.
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 2019 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 5996044. 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.