- ARQ 531 demonstrates substantial anti-tumor activity and
manageable safety profile
- Eight of nine evaluable CLL patients initially dosed at ≥65 mg
experienced a Partial Response (PR)
- Five of five CLL patients that were evaluable at the third
scan (cycle 9) are durable PRs and continue on therapy
- Three of six evaluable Richter’s patients dosed at 65 mg
experienced a PR
- Call with management scheduled for today, December 9, at 8:15
am EST to discuss these results
ArQule, Inc. (Nasdaq: ARQL) today announced final results from
the phase 1 study for ARQ 531, an orally bioavailable, potent and
reversible dual inhibitor of both wild type and C481S-mutant
Bruton’s tyrosine kinase (BTK) in patients with relapsed or
refractory hematologic malignancies at the American Society of
Hematology (ASH) 2019 Annual Meeting & Exposition in Orlando,
Florida.
“The final phase 1 data set confirms the potential utility of
ARQ 531 for the treatment of these heavily pretreated CLL patients.
We were excited to observe such deep and durable responses at a
well-tolerated dose in this highly refractory population,”
commented Dr. Brian Schwartz, Chief Medical Officer of ArQule. “In
addition, the three responses we observed in Richter’s
Transformation patients were a welcome outcome and allowed several
patients to transition to potentially curative therapies.”
“ARQ 531 was selected and extensively tested preclinically to
address the emerging therapeutic needs of patients who have become
resistant to covalent BTK inhibitors in a broad set of hematologic
malignancies,” commented Dr. Jennifer Woyach, Associate Professor
of Medicine at The Ohio State University and the Principal
Investigator of the study. “It is tremendously gratifying to
witness the emergence of a potential therapeutic for patients with
such a high degree of unmet need, such as C481S-mutant CLL and
Richter’s Transformation, and beyond. The data presented in this
poster provide compelling proof-of-concept for this novel class of
reversible BTK inhibitors.”
The reported data are from the phase 1, open label, single arm
dose escalation study and include patients (n=47) initially dosed
at levels of 5, 10, 15, 20, 30, 45, 65 and 75 mg once a day with
relapsed or refractory chronic lymphocytic leukemia (CLL), small
lymphocytic leukemia (SLL), Richter’s Transformation, Waldenstr�m
macroglobulinemia and other B-cell Non-Hodgkin lymphomas.
Key Findings:
- 65 mg QD was selected as the Recommended Phase 2 Dose (RP2D)
for further studies
- Across all disease subsets, ARQ 531 showed a low incidence of
associated toxicities, including one grade three DLT and no atrial
fibrillation or bleeding observed
- At 65 mg QD, ARQ 531 has a steady state mean Cmin above 1 µM
and long plasma half-life of 56 hours resulting in complete pBTK
inhibition
- Clinical Anti-Tumor Activity:
- In CLL, an Overall Response Rate (ORR) of 89% (8/9 responses in
evaluable patients) was achieved in heavily pretreated R/R CLL
patients (7/8 harboring BTK-C481S mutation) dosed at ≥65 mg QD.
Eleven of 19 patients treated at 65 mg QD remain on study
- In Richter’s Transformation, an ORR of 50% (3/6 responses in
evaluable patients) was achieved at 65 mg QD
- Two additional PRs were observed including one patient with
Follicular Lymphoma (FL) and 1 patient with Diffuse Large B-cell
Lymphoma (DLBCL)
- Durability:
- 100% (5/5) evaluable CLL patients that received a third scan
(cycle 9) are durable confirmed PRs and remain on therapy
- 67% (2/3) of Richter’s patients that achieved PRs came off
study after becoming eligible for CAR-T therapy
- The Follicular Lymphoma patient that achieved a PR has been on
study for 120 weeks and remains a PR and on therapy
The poster at ASH presenting these data entitled, “Final Results
of Phase 1, Dose Escalation Study Evaluating ARQ 531 in Patients
with Relapsed or Refractory B-Cell Lymphoid Malignancies,” is
available on the company’s website at
www.arqule.com/publications-presentations/.
In addition, earlier today Merck and ArQule announced that the
companies have entered into a definitive agreement under which
Merck, through a subsidiary, will acquire ArQule. A copy of the
joint press release is available on ArQule’s website,
www.arqule.com.
ArQule will host a conference call and webcast for investors on
Monday, December 9, 2019 at 8:15 a.m. EST to discuss the ARQ 531
clinical data. The live webcast can be accessed in the “Investors
and Media” section of our website, www.arqule.com, under “Events
& Presentations” or by visiting click here. You may also listen
to the call by dialing (877) 868-1831 within the U.S. or (914)
495-8595 outside the U.S. and providing conference ID 4573858. A
replay will be available two hours after the completion of the call
and can be accessed in the “Investors & Media” section of our
website, www.arqule.com, under “Events and Presentations.”
ArQule management will host an Investor Event to answer
questions and discuss these data in Orlando tonight, Monday,
December 9, 2019 from 8:00–10:00 p.m. EST. Investors, sell side
analysts, and industry representatives are welcome to attend. For
event details and to RSVP, please email ir@arqule.com.
About BTK and ARQ 531
Bruton’s tyrosine kinase, BTK, is a therapeutic target that has
been clinically proven to inhibit B-cell receptor signaling in
blood cancers. ARQ 531 is an orally bioavailable, potent and
reversible dual inhibitor of both wild type and C481S-mutant BTK.
The C481S-mutation is a known resistance mechanism for first
generation irreversible BTK inhibitors. ARQ 531 has demonstrated a
manageable safety profile, predictable PK, convincing
pharmacodynamic effects and signs of clinical activity.
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 four 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 pipeline includes: ARQ 531, an orally
bioavailable, potent and reversible dual inhibitor of both wild
type and C481S-mutant BTK, in phase 2 for patients with B-cell
malignancies refractory to other therapeutic options; miransertib
(ARQ 092), a potent and selective inhibitor of the AKT
serine/threonine kinase, in a registrational trial with cohorts in
Proteus syndrome and PROS; ARQ 751, a next generation highly potent
and selective AKT inhibitor, in phase 1 for patients with solid
tumors with AKT1 and PI3K mutations; and derazantinib, a
multi-kinase inhibitor designed to preferentially inhibit the
fibroblast growth factor receptor (FGFR) family, in a
registrational trial for iCCA in collaboration with Basilea and
Sinovant. ArQule’s current discovery efforts are focused on the
identification and development of novel kinase inhibitors,
leveraging the Company’s proprietary library of compounds.
Forward Looking Statements
This press release contains forward-looking statements,
including without limitation those regarding the ongoing clinical
trial with ARQ 531. 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
from those set forth in this press release. Positive information
about early stage clinical trial results does not ensure that later
stage or larger scale clinical trials will be successful. For
example, ARQ 531 may not demonstrate adequate therapeutic effect;
in addition, it may not demonstrate an appropriate safety profile
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 current or 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 that could lead the Company
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 collaborators’ view of data or require
additional data or information or additional studies. In addition,
the planned timing of completion of clinical trials is subject to
the ability of the Company and, in certain cases, its collaborators
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 expect to utilize diagnostic
tools in ongoing and future biomarker-guided clinical trials with
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 or
intellectual property, selectivity/specificity, analytical
validation, reproducibility, or clinical validation. Any delay or
failure by our collaborators or us to develop or obtain regulatory
approval of companion diagnostic could delay or prevent approval of
ARQ 531. Only a small number of research and development programs
result in the commercialization of a product. Furthermore, the
Company may not have the financial or human resources to
successfully pursue drug discovery 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20191209005198/en/
Kathleen Farren Communications Specialist, IR/PR and Executive
Assistant to the CFO ir@arqule.com
Media: Cait Williamson, Ph.D. LifeSci Public Relations
(646) 751-4366 cait@lifescipublicrelations.com
www.ArQule.com
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