ArQule Reports Third Quarter 2017 Financial Results
Conference call scheduled today at
For the quarter ended
Miransertib (ARQ 092), lead AKT inhibitor, has met its primary
endpoint of determining a biologically active dose in a phase 1 trial
for Proteus syndrome lead by the National Human Genome Research
Institute (NHGRI) of the National Institutes of Health (NIH).
NIHpresented preliminary data from the ongoing phase 1 clinical trial for Proteus syndrome at the Proteus Syndrome Foundation Family Conferencein September 2017. In five of the six patients, a reduction of a least 50% of phospho-AKT levels was reported. This met the primary objective of the study. Most significantly the NIHobserved disease modification in the cerebriform connective tissue nevus (CCTN) lesions which are considered one of the hallmarks of the disease.
Miransertib was granted by the
U.S. Food and Drug Administration(FDA) Rare Pediatric Disease Designation. Under the FDA's rare pediatric disease priority review voucher program, the sponsor may be eligible for a voucher that can be used to obtain a priority review for a subsequent human drug application.
Miransertib continues to dose in the phase 1/2 trial for Overgrowth
Diseases driven by PI3K and AKT mutations and opened its first
U.S.clinical trial site. This trial is part of the company's continued progress towards expanding its rare disease strategy in Overgrowth Diseases.
- ARQ 531, an orally bioavailable, potent and reversible BTK inhibitor, continues dosing as planned 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.
Derazantinib (ARQ 087), a pan-FGFR inhibitor, in a registrational
trial in FGFR2 fusion positive second-line intrahepatic
cholangiocarcinoma (iCCA) is recruiting with six active sites in the
U.S.The trial is planned to enroll up to 100 iCCA patients and provides an opportunity for a conditional approval as part of a fast-to-market strategy and includes an interim analysis that will be performed after the first 40 patients have been enrolled and evaluated for response.
Company raised approximately
$29 millionin capital through a private placement of $15.7 millionof common stock, a private placement of $9.5 millionof convertible preferred stock and an additional $4 millionthrough unrelated business development activities and other sources. Net proceeds will be used to advance ArQule'sproprietary pipeline and for general business purposes, including working capital.
"The ArQule clinical pipeline is the strongest it has ever been, and the most recent positive developments are the compelling clinical data in Proteus syndrome and the granting of Rare Pediatric Disease Designation for miransertib in this indication," said Paolo Pucci, Chief Executive Officer of ArQule. "We are now well capitalized to see our pipeline assets through major inflection points."
"Based on the preliminary results from the phase 1
Revenues and Expenses
Revenues for the quarter ended
Research and development expense in the third quarter of 2017 was
Research and development expense in the nine-months ended
General and administrative expense was
General and administrative expense was
2017 Financial Guidance
As a result of ArQule's recent stock offerings and business development
activities the company is updating 2017 guidance. For 2017,
Conference Call and Webcast
This press release contains forward-looking statements, including, without limitation, those related to clinical trials and the progress of the Company's clinical pipeline as well as the achievement of certain clinical and regulatory milestones with derazantinib (ARQ 087), miransertib (ARQ 092) and ARQ 531, use of offering proceeds and financial guidance with respect to cash at year end and the Company's ability to fund operations with current cash and marketable securities. These statements are based on the Company's current beliefs and expectations, and are subject to risks and uncertainties that could cause actual results to differ materially. Positive information about pre-clinical and early stage clinical trial results does not ensure that later stage milestones will be met or that later stage or larger scale clinical trials will be successful. Moreover, derazantinib, miransertib, and ARQ 531 or other programs may not demonstrate promising therapeutic effect; in addition, they may not demonstrate appropriate safety profiles in current or later stage or larger scale clinical trials as a result of known or as yet unanticipated side effects. The results achieved in later stage trials may not be sufficient to meet applicable regulatory standards or to justify further development. Problems or delays may arise prior to the initiation of planned clinical trials, during clinical trials or in the course of developing, testing or manufacturing these compounds that could lead the Company or its partners and collaborators to fail to initiate or to discontinue development. Even if later stage clinical trials are successful, unexpected concerns may arise from subsequent analysis of data or from additional data. Obstacles may arise or issues may be identified in connection with review of clinical data with regulatory authorities. Regulatory authorities may disagree with the Company's or its partners' view of data or require additional data or information or additional studies. In addition, the planned timing of completion of clinical trials for miransertib in Proteus syndrome is dependent in part on the National Institutes of Health, our collaborator responsible for the phase 1 trial in Proteus syndrome, to enroll patients, enter into agreements with clinical trial sites and investigators, and overcome technical hurdles and other issues related to the conduct of the trials for which each of them is responsible. There is a risk that these issues may not be successfully resolved. In addition, we are utilizing a companion diagnostic to identify patients in our registration trial with derazantinib in intrahepatic cholangiocarcinoma with FGFR2 fusions, and we are utilizing or expect to utilize diagnostic tools in our other biomarker-guided clinical trials with derazantinib, miransertib, ARQ 751 and ARQ 531; we or our collaborators may encounter difficulties in developing and obtaining approval for companion diagnostics, including issues relating to access to certain technologies, selectivity/specificity, analytical validation, reproducibility, or clinical validation. Any delay or failure by our collaborators or ourselves to develop or obtain regulatory approval of companion diagnostics could delay or prevent approval of our product candidates. Drug development involves a high degree of risk. Only a small number of research and development programs result in the commercialization of a product. Furthermore, the Company's expectations regarding its use of cash are subject to numerous risks and uncertainties, including, without limitation, those set forth above. The Company may not have the financial or human resources to successfully pursue all of its drug discovery programs in the future. For more detailed information on the risks and uncertainties associated with the Company's drug development, financial condition and other activities, see the Company's periodic reports filed with the Securities and Exchange Commission. The Company does not undertake any obligation to publicly update any forward-looking statements.
|Condensed Statement of Operations and Comprehensive Loss|
|(In Thousands, Except Per Share Amounts)|
THREE MONTHS ENDED
NINE MONTHS ENDED
|(IN THOUSANDS, EXCEPT PER SHARE DATA)|
|Research and development revenue||$||—||$||1,223||$||—||$||3,522|
|Costs and expenses:|
|Research and development||4,570||5,265||14,747||13,800|
|General and administrative||1,762||1,824||5,702||5,755|
|Total costs and expenses||6,332||7,089||20,449||19,555|
|Loss from operations||(6,332||)||(5,866||)||(20,449||)||(16,033||)|
|Unrealized gain (loss) on marketable securities||6||(10||)||(3||)||19|
|Basic and diluted net loss per share:|
|Net loss per share||$||(0.09||)||$||(0.08||)||$||(0.30||)||$||(0.23||)|
|Weighted average basic and diluted common shares outstanding||71,541||71,083||71,282||69,247|
|Balance sheet data (in thousands) (Unaudited):||
|Cash, equivalents and marketable securities- short term||$||27,603||$||31,126|
|Marketable securities-long term||-||-|
Vice President, Investor Relations/ Corp. Communications
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