LEXINGTON, Mass., Dec. 8, 2020 /PRNewswire/ -- Curis, Inc.
(NASDAQ: CRIS), a biotechnology company focused on the development
of innovative therapeutics for the treatment of cancer, today
announced positive preliminary data from its ongoing open-label,
single arm Phase 1 dose escalation study of CA-4948, a novel, small
molecule IRAK4 kinase inhibitor, in patients with acute myeloid
leukemia (AML) or high-risk myelodysplastic syndromes (MDS). IRAK4
plays an essential role in the toll-like receptor (TLR) and
interleukin-1 receptor (IL-1R) signaling pathways, and these
pathways are frequently dysregulated in patients with AML and MDS.
Third parties have recently discovered that the long form of IRAK4
(IRAK4-L) is oncogenic and preferentially expressed in over half of
patients with AML and MDS. A variety of drivers are believed to
cause this, including specific spliceosome mutations.
"We are highly encouraged by the breadth of clinical activity
with CA-4948 seen with this early data, especially as this study is
both monotherapy and in a late line, relapsed/refractory
population. Historically, monotherapy studies in AML and MDS have
proven underwhelming; monotherapy studies in a relapsed/refractory
setting have been especially challenging," said James Dentzer, President and Chief Executive
Officer of Curis. "We also have been pleased by the pace at which
our trial partners have been able to enroll patients. We look
forward to continuing to advance CA-4948 and reporting additional
Phase 1 data in the second half of 2021."
"As a clinician intimately familiar with the treatment
challenges faced by patients with AML or high-risk MDS, I am very
encouraged by the early data coming out of this study," said Dr.
Guillermo Garcia-Manero, Chief of
the Section of Myelodysplastic Syndromes within the Department of
Leukemia at The University of Texas MD
Anderson Cancer Center. "While there have been important
advancements in the development of new therapeutics for patients
with previously untreated AML or MDS in recent years, relapsed and
refractory patients are still in need of better treatment options.
These preliminary data suggest, for the first time in a clinical
setting, that successfully targeting the long isoform of IRAK4,
which we know to be preferentially expressed in over half of AML
and MDS patients, could be the first major breakthrough in over a
decade for patients with these diseases."
The reported preliminary data are from Curis's ongoing
open-label, single arm Phase 1 dose escalation 3+3 study of orally
administered CA-4948 monotherapy in adult patients with AML or
high-risk MDS. A minimum of 3 patients will be enrolled in each
cohort of the two-part study, starting with 200 mg BID, which was
demonstrated to be well-tolerated, capable of achieving relevant
levels of drug exposure and has demonstrated signs of biologic
activity in Curis's ongoing Phase 1 study of CA-4948 for the
treatment of patients with relapsed or refractory non-Hodgkin's
lymphoma. The primary objective of the study is to determine the
maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D)
for CA-4948 based on safety and tolerability, dose-limiting
toxicities (DLT), and any biologic activity, pharmacokinetic and
pharmacodynamic findings from the study trial population.
Additional objectives include characterization of CA-4948's
pharmacokinetic parameters, and biomarker correlations. As of
November 23, 2020, 4 AML
patients and 3 high-risk MDS patients had been enrolled in the
first 2 study cohorts and no DLTs had been observed. The data being
reported from this ongoing trial are preliminary and subject to
Key findings include:
- Marrow blast reductions observed in all evaluable patients (6
- 6 of 7 patients enrolled remain on study.
- Patients enrolled experienced a median of 3 prior lines of
treatment (range 1-4).
- Two patients experienced a marrow complete response, one with
blast count going from 23% pretreatment to 1% on treatment, and the
other going from 11% pretreatment to 2% on treatment.
- No DLTs observed in 7 DLT-evaluable patients in the 200 mg BID
and 300 mg BID cohorts.
- Enrollment has begun in the 400 mg BID cohort.
Webcast Event Information
Curis management will host a virtual KOL event today,
December 8, 2020 at 8:00 am ET to discuss these results with Dr.
Amit Verma, Professor of
Medicine-Oncology at Albert Einstein College of Medicine, and
Director of the MDS Program at Montefiore Medical Center located in
Bronx, NY. To access the webcast,
please visit the Events and Presentations section of the Curis
website at https://www.curis.com/.
About Curis, Inc.
Curis is a biotechnology company focused on the development of
innovative therapeutics for the treatment of cancer. In 2015, Curis
entered into a collaboration with Aurigene in the areas of
immuno-oncology and precision oncology. As part of this
collaboration, Curis has exclusive licenses to oral small molecule
antagonists of immune checkpoints including the VISTA/PDL1
antagonist CA-170, and the TIM3/PDL1 antagonist CA-327, as well as
the IRAK4 kinase inhibitor, CA-4948. CA-4948 is currently
undergoing testing in a Phase 1 trial in patients with
non-Hodgkin's lymphoma and in a Phase 1 trial in patients with
acute myeloid leukemia and myelodysplastic syndromes. In addition,
Curis is engaged in a collaboration with ImmuNext for development
of CI-8993, a monoclonal anti-VISTA antibody, which is currently
undergoing testing in a Phase 1a/1b
trial in patients with solid tumors. Curis is also party to a
collaboration with Genentech, a member of the Roche Group, under
which Genentech and Roche are commercializing Erivedge®
for the treatment of advanced basal cell carcinoma. For more
information, visit Curis' website at www.curis.com.
This press release contains forward-looking statements within
the meaning of the U.S. Private Securities Litigation Reform Act of
1995, including, without limitation, statements regarding any
expectations of the potential for the Company's proprietary drug
candidate CA-4948, including with respect to the activity and
tolerability of CA-4948, future studies with respect to CA-4948,
the potential advantages and benefits of CA-4948 and small molecule
checkpoint antagonists, statements with respect to the timing of
the Company's studies, including enrollment and reporting of data,
and the Company's plans to advance its development programs.
Forward-looking statements may contain the words "believes,"
"expects," "anticipates," "plans," "intends," "seeks," "estimates,"
"assumes," "will," "may," "could" or similar expressions. These
forward-looking statements are not guarantees of future performance
and involve risks, uncertainties, assumptions and other important
factors that may cause actual results to be materially different
from those indicated by such forward-looking statements. For
example, Curis may experience adverse results, delays and/or
failures in its drug development programs and may not be able to
successfully advance the development of its drug candidates in the
time frames it projects, if at all. Curis's drug candidates may
fail to demonstrate sufficient safety and efficacy in clinical
studies and/or may never receive regulatory approval. Favorable
results seen in preclinical studies and early clinical trials of
Curis's drug candidates may not be replicated in later trials.
There can be no guarantee that Curis's collaborations with Aurigene
and ImmuNext will continue for their full terms and receive
sufficient financing and other resources, or that the parties will
successfully discover, develop or commercialize drug candidates
under the collaborations. Regulatory authorities may delay or
restrict Genentech's and/or Roche's ability to continue to develop
or commercialize Erivedge in BCC. Erivedge may not merit further
development in disease indications other than BCC. Competing drugs
may be developed that are superior to Erivedge. Curis faces
risks relating to the transfer and encumbrance of certain royalty
and royalty-related payments on commercial sales of Erivedge, which
could have a material adverse effect on its business, financial
condition and stock price. Based on its available cash resources,
Curis does not have sufficient cash on hand to support current
operations for the next 12 months. If it is not able to obtain
sufficient funding, it will be forced to delay, reduce in scope or
eliminate some of its research and development programs, including
related clinical trials and operating expenses, potentially
delaying the time to market for, or preventing the marketing of,
any of its product candidates, which could adversely affect its
ability to continue operations and pursue its business strategies.
Curis faces substantial competition. Curis also faces the risk of
potential adverse decisions made by the FDA and other regulatory
authorities, investigational review boards, and publication review
bodies. Curis may not obtain or maintain necessary patent
protection and could become involved in expensive and
time-consuming patent proceedings. Unstable market and economic
conditions, natural disasters, public health crises, political
crises and other events outside of Curis's control could
significantly disrupt its operations or the operations of third
parties on which Curis depends. For example, the COVID-19 pandemic
may result in closures of third-party facilities, impact clinical
trial enrollment or impact sales of Erivedge. The extent to
which the COVID-19 pandemic may impact Curis's business is
uncertain. Other important factors that may cause or contribute
to actual results being materially different from those
indicated by forward-looking statements include the factors set
forth under the caption "Risk Factors" in Curis's most recent Form
10-K and Form 10-Q and the factors that are discussed in other
filings that Curis periodically makes with the Securities and
Exchange Commission ("SEC"). In addition, any forward-looking
statements represent the views of Curis only as of today and should
not be relied upon as representing Curis's views as of any
subsequent date. Curis disclaims any intention or obligation to
update any of the forward-looking statements after the date of this
press release whether as a result of new information, future events
or otherwise, except as may be required by law.
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SOURCE Curis, Inc.