ArQule Reports Second Quarter 2017 Financial Results

Conference call scheduled today at 9:00 a.m. ET

BURLINGTON, Mass.--(BUSINESS WIRE)--ArQule, Inc. (Nasdaq: ARQL) today announced its financial results for the second quarter of 2017.

For the quarter ended June 30, 2017, the Company reported a net loss of $7,201,000 or $0.10 per share, compared with a net loss of $5,100,000 or $0.07 per share, for the second quarter of 2016. For the six-month period ended June 30, 2017, the Company reported a net loss of $14,777,000 or $0.21 per share, compared with a net loss of $10,081,000 or $0.15 per share, for the six-month period ended June 30, 2016.

At June 30, 2017, the Company had a total of approximately $31,007,000 in cash, equivalents and marketable securities.

Key Highlights

• Derazantinib (ARQ 087), a pan-FGFR inhibitor, has begun recruiting in a registrational phase 3 trial for FGFR2 fusion positive second-line intrahepatic cholangiocarcinoma (iCCA). Enrollment is planned to commence in the current quarter. In May, compelling data from the phase 1/2 trial in second-line iCCA was presented at the American Society of Clinical Oncology (ASCO) meeting highlighting a disease control rate of 83% and an objective response rate of 21%.
• ARQ 531, an orally bioavailable, potent and reversible BTK inhibitor, has been dosed in a phase 1a/b trial. The trial is enrolling patients with B-cell malignancies, including B-cell lymphomas, chronic lymphocytic leukemia, and Waldenstrom's macroglobulinemia, who are refractory to other therapeutic options, including ibrutinib. Up to 120 patients can be enrolled in the trial. The company also presented preclinical data for ARQ 531 in diffuse large B-cell lymphoma at the Annual Congress of the European Hematology Association which further strengthens the preclinical package for this molecule.
• ARQ 092, lead AKT inhibitor, has been dosed in a phase 1/2 company-sponsored trial in Overgrowth Diseases with genetic alterations of the PI3K/AKT1 pathway, including PROS (PIK3CA-related Overgrowth Spectrum) and Proteus syndrome. The trial is designed to enroll six patients in a dose escalation cohort as part of the phase 1 portion of the trial. An additional 10 patients will be enrolled in an expansion cohort as part of the phase 2 portion of the trial. The objective of this study is to determine a clinically meaningful endpoint to pursue in a registrational trial.

“We have made significant progress over the past few months by initiating phase 1 trials for ARQ 092 and ARQ 531 with the aim of achieving clinical proof of principle, and we are now poised to initiate a registrational trial with derazantinib,” said Paolo Pucci, Chief Executive Officer of ArQule. "We achieved all of our targeted pipeline milestones for the first half of 2017, most notably moving ARQ 531, our BTK inhibitor, into the clinic. We believe ARQ 531 was the first reversible BTK inhibitor to be dosed in patients with B-cell malignancies. With four programs in the clinic, including derazantinib, ARQ 092, ARQ 751, and ARQ 531, we are poised to continue to achieve our goals for 2017."

“Our pipeline achieved two important milestones with the dosing of the first patient in two biomarker driven clinical trials targeting patients in areas of high unmet need,” said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule. "In the first of these clinical trials, ARQ 531 aims to demonstrate its potential to address a large patient population with B-cell malignancies who become refractory to current therapies. This is a significant emerging clinical need, particularly in C481S-mutant patients. In the second trial, ARQ 092 is now being dosed in Overgrowth Diseases driven by the PI3K/AKT1 mutation targeting a completely unmet clinical need in a patient population comprised of multiple orphan diseases. Both programs have the potential to be transformational and represent well ArQule’s mission to bring life-changing therapies to address unmet medical needs."

Revenues and Expenses

Revenues for the quarter ended June 30, 2017, were zero compared with revenues of $1,072,000 for the quarter ended June 30, 2016. Revenues in the six-months ended June 30, 2017 were zero compared with revenues of $2,299,000 in the six-months ended June 30, 2016. Revenue in the three and six-month periods of 2016 is comprised of revenue from the Daiichi Sankyo tivantinib development agreement and the Kyowa Hakko Kirin exclusive license agreement. No further revenue is anticipated from these agreements.

Research and development expense in the second quarter of 2017 was $4,983,000, compared with $4,337,000 for the second quarter of 2016. Research and development expense increased $0.6 million in the second quarter of 2017 primarily due to higher outsourced preclinical, clinical and product development costs.

Research and development expense in the six-months ended June 30, 2017 was $10,177,000 compared with $8,535,000 in the six-months ended June 30, 2016. The $1.6 million increase in research and development expense in the six-months ended June 30, 2017 was primarily due to higher outsourced preclinical, clinical and product development costs.

General and administrative expense was $1,866,000 in the second quarter of 2017 compared with $1,887,000 in the second quarter 2016.

General and administrative expense was $3,940,000 in the six-months ended June 30, 2017 compared with $3,931,000 in the six-months ended June 30, 2016.

Conference Call and Webcast

ArQule will hold its second quarter 2017 financial results call today, August 4, 2017 at 9:00 a.m. ET. The live webcast can be accessed in the "Investors & Media" section of our website, www.arqule.com, under "Events & Presentations." You may also listen to the call by dialing (877) 868-1831 within the U.S. or (914) 495-8595 outside the U.S. A replay will be available two hours after the completion of the call and can be accessed in the "Investor and Media" section of our website, www.arqule.com, under "Events & Presentations."

About ArQule

ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and rare diseases. ArQule’s mission is to discover, develop and commercialize novel small molecule drugs in areas of high unmet need that will dramatically extend and improve the lives of our patients. Our clinical-stage pipeline consists of five drug candidates, all of which are in targeted, biomarker-defined patient populations, making ArQule a leader among companies our size in precision medicine. ArQule’s proprietary pipeline includes: ARQ 087, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family, in phase 2 for iCCA and in phase 1b for multiple oncology indications; ARQ 092, a selective inhibitor of the AKT serine/threonine kinase, in a phase 1/2 company sponsored study for Overgrowth Diseases, in a phase 1 study for ultra-rare Proteus syndrome conducted by the National Institutes of Health (NIH), as well as in multiple oncology indications; ARQ 751, a next generation AKT inhibitor, in phase 1 for patients with AKT1 and PI3K mutations; and ARQ 761, a ß-lapachone analog being evaluated as a promoter of NQO1-mediated programmed cancer cell necrosis, in phase 1/2 in multiple oncology indications in partnership with the University of Texas Southwestern Medical Center. In addition, we have advanced ARQ 531, an investigational, orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant BTK, in phase 1 for patients with B-cell malignancies refractory to other therapeutic options. ArQule’s current discovery efforts are focused on the identification and development of novel kinase inhibitors, leveraging the Company’s proprietary library of compounds. You can follow us on Twitter and LinkedIn.