miRagen Therapeutics, Inc. (NASDAQ: MGEN), a clinical-stage
biopharmaceutical company developing proprietary RNA-targeted
therapies with a specific focus on microRNAs, today reported
financial results for the first quarter ended March 31, 2019
and provided a corporate update.
“Execution on our strategy by the miRagen team
over the past few quarters has allowed us to make important
advances in the development of cobomarsen, remlarsen, and MRG-110
in 2019, including initial dosing in the SOLAR Phase 2 trial of
cobomarsen and the recent release of new data from our Phase 1
trial of cobomarsen in adult T-cell leukemia/lymphoma (ATLL)
patients,” said William S. Marshall, Ph.D., President and Chief
Executive Officer of miRagen Therapeutics.
“In addition to cobomarsen, we continue to make
progress advancing remlarsen and MRG-110. We look forward to
releasing additional data from three clinical trials during the
remainder of 2019, including our Phase 2 clinical trial of
remlarsen in keloid scarring and two Phase 1 trials for MRG-110,”
continued Dr. Marshall.
Recent Program Highlights
Cobomarsen
In April 2019, the first mycosis fungoides
patients were dosed in the SOLAR Phase 2 trial. The SOLAR trial is
designed to evaluate the safety and efficacy of cobomarsen given in
300 mg doses by intravenous infusion in an active control
comparison trial versus vorinostat. miRagen has opened a number of
clinical sites in the trial and is planning to initiate activities
at up to sixty clinical sites in eleven countries worldwide. The
primary endpoint of the SOLAR trial is the rate of an objective
response that is durable for four months, defined as 50% or greater
improvement in the severity of a patient’s skin disease over the
entire body with no evidence of disease progression in the blood,
lymph nodes, or viscera. Progression-free survival is a secondary
endpoint, and miRagen plans to use patient-reported outcomes as
additional endpoints to monitor quality of life improvements. Based
on discussions with the U.S. Food and Drug Administration (FDA),
miRagen believes that primary endpoint data from this clinical
trial could allow miRagen to apply for accelerated approval in the
United States. The Company expects to report primary endpoint data
from this clinical trial in the second half of 2020.
miRagen is also evaluating cobomarsen in certain
expansion indications where the disease process appears to be
correlated with an increase in miR-155 levels, the target of
cobomarsen. The Company recently announced new data from the ATLL
cohort of its Phase 1 clinical trial of cobomarsen in an oral
presentation at the 19th International Congress HTLV 2019. In the
updated data from the trial, five patients with aggressive ATLL
subtype in partial remission have been treated with cobomarsen for
up to 16 months. Four of the five patients remained stable for up
to 16 months and continued dosing. Biological activity of
cobomarsen in these patients is indicated by a significant decrease
in biomarkers of cell proliferation (e.g., Ki67) and activation
(e.g., HLD-R and CD69) in all patients, supporting the observed
clinical stabilization. Cobomarsen has been well tolerated over
prolonged treatment, with no deaths, dose limiting toxicities,
related serious adverse events, related Grade 3 or Grade 4 adverse
events, hematological events or discontinuation from trial due to
related adverse events.
Remlarsen
miRagen recently announced new preclinical data
from a study exploring the antifibrotic effects of remlarsen in the
cornea. In the study, remlarsen treatment appeared to accelerate
the healing of corneal injury, resulting in a more rapid
restoration of epithelial thickness and a reduction in stromal
thickness as compared to saline-treated injured eyes. Remlarsen
treatment also appeared to reduce the expression of multiple
collagens and fibrosis-associated genes from 7-14 days post-injury
and to reduce α-SMA protein expression in the epithelium and stroma
at 14 days. Lastly, remlarsen treatment appeared to reduce corneal
hazing and scarring beginning at 10 days post-burn. Dose and
treatment schedule were optimized in preparation for enabling
toxicology studies to support human clinical trials.
Based on this data, miRagen believes that
further exploration of the topical application of remlarsen as a
treatment to improve vision in patients suffering from multiple
conditions resulting in corneal scarring may be warranted. Corneal
scarring is one of the leading causes of blindness worldwide.
miRagen is currently conducting a Phase 2
clinical trial of remlarsen assessing the safety, tolerability, and
activity of remlarsen in the potential prevention or reduction of
keloid formation in subjects with a history of keloid scars, a form
of pathological scarring. The trial has completed its enrollment
and miRagen expects to report data from this clinical trial in the
second half of 2019.
MRG-110
MRG-110 is currently being evaluated in
collaboration with Servier in two Phase 1 clinical trials designed
to evaluate the safety, tolerability, and pharmacokinetics of
MRG-110. The data generated in these clinical trials is expected to
provide clinically translatable biomarkers that may support future
clinical trials for the treatment of heart failure and other
conditions where patients may benefit from increased vascular flow
and accelerated healing, such as complicated lacerations in high
risk patients and burns. Enrollment has been completed in both
Phase 1 clinical trials of MRG-110 and the Company expects to
report data in 2019.
Financial Condition and Operating
Results
Cash, cash equivalents, and short-term
investments were $51.0 million as of March 31, 2019, compared
to $62.5 million as of December 31, 2018. Cash used in
operating activities was $11.8 million for the first quarter of
2019. miRagen believes that its current cash, cash-equivalents, and
short-term investments will be sufficient to fund the Company’s
operations for the next twelve months.
Revenue was $0.4 million for the first quarter
of 2019, compared to $4.8 million for the first quarter of 2018.
The decrease in revenue is primarily due to a $3.7 million
development milestone payment miRagen earned under its
collaboration agreement with Servier during the first quarter of
2018.
Research and development expenses were $8.8
million for the first quarter of 2019, compared to $6.4 million for
the first quarter of 2018. The increase in research and development
expenses is primarily due to increases in clinical development
activities associated with the Phase 2 SOLAR clinical trial of
cobomarsen and personnel-related costs. These increases were
partially offset by lower technology license fees in 2019.
General and administrative expenses were $3.4
million for the first quarter of 2019, compared to $3.0 million for
the first quarter of 2018. The increase in general and
administrative expenses is primarily due to increases in
personnel-related costs resulting primarily from the growth of our
general and administrative team and higher share-based compensation
charges.
The Company’s net loss was $11.6 million, or
$0.38 per share, for the first quarter of 2019, compared to $4.7
million, or $0.18 per share, for the first quarter of 2018.
Conference Call Information
miRagen will host a conference call today at
4:30 p.m. ET to discuss its financial results for the first quarter
of 2019. Participants may access the call by dialing 877-407-0789
in the U.S. or 201-689-8562 outside the U.S. and providing the
conference ID number 13689475. The call will also be webcast and
can be accessed from the Investors and Media section of miRagen’s
website at www.miragen.com. A replay of this conference call will
be available on miRagen’s website approximately one hour after the
event.
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, remlarsen,
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. 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 in
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
Statements
This 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, anticipated clinical
development milestones, 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
Miragen Therapeutics,
Inc.Condensed Consolidated Statements of
Operations and Comprehensive Loss(in thousands,
except share and per share
data)(unaudited)
|
|
|
Three Months Ended March 31, |
|
2019 |
|
|
2018 |
|
Revenue: |
|
|
|
Collaboration revenue |
$ |
348 |
|
|
$ |
4,756 |
|
Grant revenue |
24 |
|
|
28 |
|
Total revenue |
372 |
|
|
4,784 |
|
Operating expenses: |
|
|
|
Research and development |
8,751 |
|
|
6,413 |
|
General and administrative |
3,357 |
|
|
2,990 |
|
Total operating expenses |
12,108 |
|
|
9,403 |
|
Loss from operations |
(11,736 |
) |
|
(4,619 |
) |
Other income (expense): |
|
|
|
Interest and other income |
339 |
|
|
167 |
|
Interest and other expense |
(232 |
) |
|
(209 |
) |
Net loss |
(11,629 |
) |
|
(4,661 |
) |
Change in unrealized gain on
investments |
5 |
|
|
— |
|
Comprehensive loss |
$ |
(11,624 |
) |
|
$ |
(4,661 |
) |
|
|
|
|
Net loss |
$ |
(11,629 |
) |
|
$ |
(4,661 |
) |
Net loss per share, basic and diluted |
$ |
(0.38 |
) |
|
$ |
(0.18 |
) |
Weighted-average shares used to compute basic and diluted net loss
per share |
30,886,085 |
|
|
26,483,112 |
|
|
|
|
|
|
|
Miragen Therapeutics,
Inc.Selected Financial
InformationCondensed Consolidated Balance Sheet
Data(amounts in
thousands)(unaudited)
|
|
|
|
|
March 31, 2019 |
|
December 31, 2018 |
Cash and cash
equivalents |
$ |
18,195 |
|
$ |
32,606 |
Short-term investments |
$ |
32,844 |
|
$ |
29,875 |
Total assets |
$ |
54,950 |
|
$ |
66,147 |
Note payable, inclusive of current portion |
$ |
10,392 |
|
$ |
10,298 |
Total liabilities |
$ |
13,994 |
|
$ |
14,803 |
Total stockholders’
equity |
$ |
40,956 |
|
$ |
51,344 |
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