miRagen Therapeutics, Inc. (NASDAQ: MGEN), a clinical-stage
biopharmaceutical company focused on the discovery and development
of RNA-targeted therapies, today announced data from its Phase 1
clinical trial evaluating the safety, tolerability and efficacy of
cobomarsen, an inhibitor of microRNA-155, in cutaneous T-cell
lymphoma (CTCL) and in adult T-cell leukemia/lymphoma (ATLL). The
Company will also discuss initial clinical experience in treating
diffuse large B-cell lymphoma (DLBCL) patients with cobomarsen. The
data will be presented at the 11th Annual T-Cell Lymphoma Forum,
which is being held in La Jolla, CA, from January 10th-12th.
“The ATLL clinical data shows that cobomarsen provided sustained
disease stabilization in five patients for up to 13 months after
completing chemotherapy or experimental treatment and an
improvement in the levels of normal circulating blood cells.
In addition, patients did not report any significant side effects
attributed to cobomarsen. We believe these data are particularly
encouraging, as the patients in this study had previously failed on
other therapies and the median survival for patients with acute
disease typically ranges from four to ten months after diagnosis,”
stated Paul Rubin M.D. Executive Vice President, R&D, at
miRagen. “We are encouraged by our early experience in the ABC
subtype of DLBCL, as we have observed an improvement in a patient
that had long-standing disease and had previously been on multiple
chemotherapeutic regimens.”
Phase 1 Data for Cutaneous T-cell Lymphoma
(CTCL)Updated durability data for the 300mg IV infusion
cohort of the Phase 1 cobomarsen clinical trial, which is the dose
and route of administration being used in the ongoing SOLAR Phase 2
clinical trial, showed that four of eight patients (50%) achieved
an objective response with greater than four months of durability
(ORR4).
The Phase 2 SOLAR trial will evaluate the safety and efficacy of
cobomarsen given by intravenous infusion in an active control
comparison trial versus ZOLINZA (vorinostat) in patients with
CTCL. ORR4 is the primary endpoint that will be used in the
SOLAR trial. Based on discussions with the U.S. Food and Drug
Administration, miRagen believes the results from the SOLAR trial
could allow the Company to apply for accelerated approval in the
United States.
Phase 1 Data for HTLV-1 Associated Adult T-cell
Leukemia/Lymphoma (ATLL) Data from the ongoing Phase 1
clinical trial in ATLL has shown that cobomarsen had a favorable
safety and tolerability profile with no serious adverse events
attributed to the drug candidate in the clinical trial and no
documented opportunistic infections, which are common in patients
with the disease. Four patients – two lymphomatous and two acute –
who demonstrated a partial response after chemotherapy have
maintained their responses while on cobomarsen monotherapy.
Two of these patients – one from each subtype – have been
stable for more than a year . There is evidence of disease
stabilization in five patients on cobomarsen, as shown in both
peripheral blood and lymph nodes, without negatively impacting the
number of normal immune cells. One of these lymphomatous
patients with significant adenopathy prior to enrollment has
remained stable on cobomarsen, as measured by CT scans, for six
months. This includes an objective improvement in three out of four
measurable abnormal nodes since initiating cobomarsen therapy.
Phase 1 Trial of Cobomarsen in Patients with Diffuse
large B-cell lymphoma (DLBCL)To date, three DLBCL patients
of the ABC subtype have received cobomarsen. One patient has
seen a complete reduction in one of two measured lymph nodes and
stabilization in the second lymph node after six weeks of therapy.
This patient remains on cobomarsen. Two of the three patients
discontinued therapy after less than one month due to lack of
immediate response. Previously, all three patients had
relapsed after multiple cycles of treatments with other therapies
received over 12-56 months from diagnosis. Prior treatments
for these patients ranged from standard of care to experimental
chemotherapy.
William S. Marshall, Ph.D., President and Chief Executive
Officer of miRagen Therapeutics, stated, “Cobomarsen continues to
be generally well-tolerated, has had durable clinical activity in
responding patients, and has the potential to improve the quality
of life for patients with a variety of hematological malignancies.
These clinical responses, combined with the favorable tolerability
profile observed across patients with three different types of
malignancy, suggests that cobomarsen may combat cancers that
overexpress microRNA-155 in a targeted manner.”
For additional information, please visit the T-Cell Lymphoma
Forum website: www.tcellforum.com
About CobomarsenCobomarsen is an inhibitor of
microRNA-155. In cutaneous T-cell Lymphoma (CTCL), as well as
certain other blood cancers, microRNA-155 is present at abnormally
high levels and may play a role in the proliferation of blood and
lymph cells. miRagen believes therapeutic inhibition of
microRNA-155 may reduce aberrant cell proliferation and tumor
growth characteristics of certain types of cancer. The
Company is currently evaluating cobomarsen in three oncology
indications within the current Phase 1 trial, including adult
T-cell leukemia/lymphoma (ATLL), diffuse large B-cell lymphoma
(DLBCL) and chronic lymphocytic leukemia (CLL).
About ATLLATLL is a blood cell malignancy that
develops in patients after prolonged infection with the virus,
HTLV1. Literature suggests that the infection with HTLV1 as
well as the subsequent malignancies may be associated with
elevation in the expression of microRNA-155, the target of
cobomarsen. The disease presents in multiple forms, but the
most lethal include the acute leukemic form and the lymphomatous
version. Although the disease is rare, these two
manifestations lack good treatment options, and once the diagnosis
is made, average life expectancy is approximately 4 months for the
acute leukemic form and approximately 10 months for the
lymphomatous variety.
About DLBCLAccording to the Lymphoma Research
Foundation, diffuse large B-cell lymphoma (DLBCL) is the most
common type of non-Hodgkin lymphoma (NHL) in the United States and
worldwide, accounting for up to one-third of patients with newly
diagnosed NHL in the United States. DLBCL is an aggressive
(fast-growing) NHL that affects B-lymphocytes. Lymphocytes are one
type of white blood cell. B-cells are lymphocytes that make
antibodies to fight infections and are an important part of the
lymphatic system. DLBCL can develop in the lymph nodes or in
“extranodal sites” (areas outside the lymph nodes) such as the
gastrointestinal tract, testes, thyroid, skin, breast, bone, brain,
or essentially any organ of the body. It may be localized (in one
spot) or generalized (spread throughout the body).
Approximately 40% of patients have refractory disease or disease
that will relapse after an initial response, and the majority of
patients with relapsed DLBCL will succumb to the disease. There are
two major biologically distinct molecular subtypes of DLBCL:
germinal center B-cell (GCB) and activated B-cell (ABC). ABC DLBCL
is associated with substantially worse outcomes when treated with
standard chemoimmunotherapy (Nowakowski & Czuczman 2015).
One key molecular distinction between the two subtypes is the
understanding that nuclear factor (NF)-κB, a prosurvival and
antiapoptotic molecule, is constitutively expressed and may be a
key contributor to chemotherapy resistance in the ABC subgroup
(Khan & Fisher, Blood 2015). Cobomarsen has been shown to
inactivate the (NF)-κB pathway in a Phase 1 human clinical
trial.
About miRagen Therapeutics, Inc.miRagen
Therapeutics, Inc. is a clinical-stage biopharmaceutical company
discovering and developing proprietary RNA-targeted therapies with
a specific focus on microRNAs and their role in diseases where
there is a high unmet medical need. miRagen has three clinical
stage product candidates, cobomarsen (MRG-106), remlarsen
(MRG-201), and MRG-110. miRagen’s clinical product candidate for
the treatment of certain cancers, cobomarsen, is an inhibitor of
microRNA-155, which is found at abnormally high levels in malignant
cells of several blood cancers, as well as certain cells involved
in inflammation. miRagen’s clinical product candidate for the
treatment of pathological fibrosis, remlarsen, is a replacement for
microRNA-29, which is found at abnormally low levels in a number of
pathological fibrotic conditions, including cutaneous, cardiac,
renal, hepatic, pulmonary and ocular fibrosis, as well as systemic
sclerosis. MRG-110, an inhibitor of microRNA-92, is being developed
under a license and collaboration agreement with Servier for the
treatment of heart failure and other ischemic disease. In addition
to these programs, miRagen is developing a pipeline of preclinical
product candidates. The goal of miRagen’s translational medicine
strategy is to progress rapidly to first-in-human studies once it
has established the pharmacokinetics, pharmacodynamic, safety and
manufacturability of the product candidate in preclinical studies.
For more information, please visit www.miragen.com.
For information on clinical trials please visit
www.clinicaltrials.gov.
Note Regarding Forward-Looking StatementsThis
press release may contain forward-looking statements that involve
substantial risks and uncertainties for purposes of the safe harbor
provided by the Private Securities Litigation Reform Act of 1995.
All statements contained in this press release other than
statements of historical fact, including statements regarding
miRagen’s strategy, future operations, future financial position,
future revenue, projected expenses, prospects, plans and objectives
of management or the expected features of or potential indications
for miRagen’s product candidates are forward-looking statements.
The words “believe,” “may,” “will,” “estimate,” “continue,”
“anticipate,” “intend,” “plan,” “expect,” “predict,” “potential,”
“opportunity,” “goals,” or “should,” and similar expressions are
intended to identify forward-looking statements. Such statements
are based on management’s current expectations and involve risks
and uncertainties. Actual results and performance could differ
materially from those projected in the forward-looking statements
as a result of many factors, including, without limitation: that
miRagen has incurred losses since its inception, and anticipates
that it will continue to incur significant losses for the
foreseeable future; future financing activities may cause miRagen
to restrict its operations or require it to relinquish rights;
miRagen may fail to demonstrate safety and efficacy of its product
candidates; miRagen’s product candidates are unproven and may never
lead to marketable products; miRagen’s product candidates are based
on a relatively novel technology, which makes it difficult to
predict the time and cost of development and of subsequently
obtaining regulatory approval, if at all; miRagen’s product
candidates may cause undesirable side effects or have other
properties that could delay or prevent the regulatory approval; and
the results of miRagen’s clinical trials to date are not sufficient
to show safety and efficacy of miRagen’s product candidates and may
not be indicative of future clinical trial results.
miRagen has based these forward-looking statements largely on
its current expectations and projections about future events and
trends. These forward-looking statements are subject to a number of
risks, uncertainties and assumptions, including those described
under the heading “Risk Factors” in miRagen’s Annual Report on Form
10-K and subsequent periodic reports filed with the Securities and
Exchange Commission. Moreover, miRagen operates in a very
competitive and rapidly changing environment. New risks emerge from
time to time. It is not possible for its management to predict all
risks, nor can it assess the impact of all factors on its business
or the extent to which any factor, or combination of factors, may
cause actual results to differ materially from those contained in
any forward-looking statements it may make. In light of these
risks, uncertainties and assumptions, the future events and trends
discussed in this press release may not occur and actual results
could differ materially and adversely from those anticipated or
implied in the forward-looking statements. miRagen undertakes no
obligation to revise or publicly release the results of any
revision to such forward-looking statements, except as required by
law. Given these risks and uncertainties, readers are cautioned not
to place undue reliance on such forward-looking statements. All
forward-looking statements are qualified in their entirety by this
cautionary statement.
Investor/Media Contact:Adam Levy, Chief
Business Officer(720) 407-4595alevy@miragen.com
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