[August 10, 2017] |
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Dicerna Reports Second Quarter 2017 Financial and Operating Results and Provides Corporate Update
Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA), a leading developer of
investigational ribonucleic acid interference (RNAi) therapeutics, today
reported financial and operating results for the second quarter ended
June 30, 2017.
"We are pleased to have reported a number of significant events during
this quarter that support the execution of our business strategy,"
stated Douglas Fambrough, president and chief executive officer of
Dicerna. "Specifically, the completion of our $70 million convertible
preferred stock financing and the key addition of Dr. Ralf Rosskamp as
chief medical officer, have served to further solidify our financial and
leadership capacity. As a result, we are in a strong position to
continue to advance DCR-PHXC, our lead GalXC™-based product candidate,
into Phase 1 clinical studies for primary hyperoxaluria (PH) early next
year and to continue to pursue Investigational New Drug (IND)
application-enabling activities for our undisclosed rare disease program
as well as for DCR-HBVS. More recently, during the 12th
International Workshop on Primary Hyperoxaluria held this past July, we
presented new preclinical data for DCR-PHXC demonstrating how inhibition
of the lactate dehydrogenase A (LDHA) gene reduced oxalate
production in multiple animal models of PH. These data highlight the
role of LDHA as an optimal therapeutic target and the potential
utility of DCR-PHXC to treat all forms of the disease. These findings
indicate that this novel target may offer the ability to treat an
expanded population of patients who currently have no other effective
options."
GalXC™ Program Update
-
During the second quarter of 2017, Dicerna continued to progress
preclinical activities for its four core therapeutic programs,
including DCR-PHXC for PH, an undisclosed rare disease program,
DCR-HBVS for hepatitis B virus, and DCR-PCSK9 for
hypercholesterolemia. Dicerna's development activities are focused in
the areas of rare diseases, chronic liver diseases, cardiovascular
diseases, and viral infectious diseases.
-
Primary Hyperoxaluria: On July 15, 2017, in a series of
presentations at the 12th International Workshop on
Primary Hyperoxaluria, Dicerna presented new preclinical data
showing DCR-PHXC's ability to inhibit LDHA resulting in
consistent and significant reduction in urinary oxalate levels in
animal models of PH type 1 (PH1), PH type 2 (PH2) and idiopathic
PH. PH is a family of severe, rare, genetic liver disorders
characterized by overproduction of oxalate that often results in
kidney failure.
Research from multiple animal models of PH demonstrated how DCR-PHXC
inhibits LDHA, which the Company has identified as potentially
being an optimal therapeutic target in patients with the disease. The
data highlights included:
-
LDHA inhibition reduces oxalate to normal or near-normal levels
in PH types 1, 2, and ethylene glycol-induced hyperoxaluria (a model
for idiopathic PH).
-
LDHA reduction has a near-linear correlation with oxalate
reduction and offers a minimal metabolic intervention. These benefits
of LDHA inhibition may translate into consistent therapeutic
activity even in the event of a missed dose. There are numerous case
reports of LDHA deficiency naturally occurring in healthy
humans, with no reported adverse effects due to deficiency in the
liver.
-
DCR-PHXC appeared to be well tolerated in these animal studies, with
no adverse effects in the liver. Formal animal toxicology studies are
ongoing.
During the workshop, Dicerna also reported data from its Primary
HYperoxaluria Observational
Study (PHYOS), an international,
multicenter, observational study in patients with a genetically
confirmed diagnosis of PH1. PHYOS is collecting data on key biochemical
parameters, including changes in oxalate, glycolate, and other
metabolites, implicated in the pathogenesis of the disease. Dicerna
continues to advance PHYOS to facilitate DCR-PHXC development and hopes
to use the data to better understand the baseline PH1 disease state,
which will help guide long-term drug development plans.
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Twenty (20) patients were enrolled in the study, with a median age at
screening of 21 years (range 12-61 years). The patients had been
diagnosed at a median age of 7 years (range 1-59 years), and 14
patients (74%) had a medical history of renal stones.
-
Over the six-month observation period, the variability (coefficient of
variation) between 24-hour urine measurements of oxalate at different
time points was 28%.
-
These data will be used by Dicerna's clinical team in the design of
future clinical studies using 24-hour urinary oxalate excretion as a
surrogate marker for clinical benefit.
Dicerna is on track to file a clinical trial application (CTA) in Europe
for DCR-PHXC in late 2017 and to commence human clinical trials in the
first quarter of 2018. During the workshop, Dicerna disclosed that the
DCR-PHXC clinical trial will be conducted at multiple sites in Europe
and will include both healthy volunteer and patient cohorts. The Company
anticipates that study participants will receive a single ascending dose
of DCR-PHXC via subcutaneous injection, transitioning, as appropriate,
to multiple ascending doses. The primary endpoints will include safety
and tolerability, urine and plasma biomarkers, and pharmacokinetics.
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Undisclosed Rare Disease Involving the Liver: Dicerna advanced IND
application-enabling activities for a second GalXC-based clinical
candidate targeting an undisclosed rare disease. For competitive
reasons, the Company has not yet publicly disclosed the target gene or
disease. Dicerna is on track to file an IND application in the U.S.
and/or CTA in Europe for this program in the second quarter of 2018.
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Chronic Hepatitis B Virus (HBV): Dicerna continued to progress its
DCR-HBVS program, which targets HBV directly, and has initiated formal
IND-enabling activities. Current therapies for HBV rarely lead to a
long-term immunological cure as measured by the clearance of HBV
surface antigen (HBsAg) and sustained HBV deoxyribonucleic acid
suppression. Based on findings from its preclinical studies, Dicerna
is evaluating whether its GalXC RNAi platform can produce an
experimental HBV-targeted therapy that significantly reduces HBsAg
expression in affected patients and that has the potential to be
delivered in a subcutaneous dosing paradigm. The Company expects to
file an IND application in the U.S. or CTA in Europe for this program
at approximately the end of 2018.
-
Hypercholesterolemia: Dicerna continued to develop its DCR-PCSK9
program, which targets the PCSK9 gene and will be evaluated for the
treatment of statin-refractory patients with hypercholesterolemia. The
Company is positioned to advance DCR-PCSK9 into formal preclinical
development. Based on preclinical studies, Dicerna believes that its
GalXC RNAi platform has the potential to produce a PCSK9-targeted
therapy with attractive commercial properties, such as small
subcutaneous injection volumes and less frequent dosing.
Financing Update
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As previously reported, on April 11, 2017, Dicerna closed a stock
purchase transaction for the sale of redeemable convertible preferred
stock (Preferred Stock) to a syndicate of current and new investors
led by Bain Capital Life Sciences, under which the Company received
gross proceeds of $70.0 million (Private Placement). At the closing,
Dicerna issued 700,000 shares of Preferred Stock, which are
convertible into common shares at an initial conversion price of $3.19
per share. In addition to the lead investor, other participants in the
Private Placement included Cormorant Asset Management, Domain
Associates, EcoR1 Capital, RA Capital and Skyline Ventures, among
others. Under the terms of the Preferred Stock purchase agreement,
Adam M. Koppel, M.D., Ph.D., a managing director of Bain Capital Life
Sciences, joined Dicerna's board of directors, which has been expanded
to nine seats.
Corporate Update
-
On June 8, 2017, Dicerna announced the appointment of industry veteran
Ralf Rosskamp, M.D., as chief medical officer. Dr. Rosskamp brings to
Dicerna more than 20 years of research and development experience
spanning the entire drug development cycle, from preclinical through
product commercialization. He has been responsible for numerous IND
applications, the design and execution of clinical development
programs, and new drug applications across multiple therapeutic areas
including diabetes, cardiovascular, respiratory, and orphan drugs.
Approved products for which Dr. Rosskamp was involved include
Natpara®, Amaryl®, Lantus®, Apidra® and Simcor®. Dr. Rosskamp is a
pediatric endocrinologist and received his M.D. from the University of
Bonn, Germany.
Financial Condition and Operating Results
-
Cash Position - As of June 30, 2017, Dicerna had $88.7 million
in cash and cash equivalents and held-to-maturity investments, as
compared to $45.9 million as of December 31, 2016. In addition, the
Company had $1.1 million of restricted cash equivalents as of June 30,
2017, which reflects collateral securing the Company's operating lease
obligation. The increase in cash and cash equivalents and
held-to-maturity investments was due chiefly to the addition of funds
generated by the Company's $70 million Private Placement, which closed
on April 11, 2017.
-
Research and Development (R&D) Expenses - R&D expenses were
$9.3 million and $18.2 million for the three and six months ended June
30, 2017, as compared to $11.0 million and $22.3 million for the same
periods in 2016, respectively. The decrease was due primarily: to a
reduction in platform-related expenses, resulting from the timing of
activities related to discovery and early development programs,
including supply and external study costs; to a decrease in
employee-related expenses, including non-cash stock-based compensation
costs; and to a reduction in clinical and manufacturing activities
related to the Company's now discontinued DCR-PH1 and DCR-MYC
programs, both of which Dicerna anticipates will be fully wound down
before the end of 2017. These decreases were partially offset by an
increase in direct R&D expenses due to an overall increase in
manufacturing activities and in toxicology study costs related to
Dicerna's new candidates under its GalXC platform.
-
General and Administrative (G&A) Expenses - G&A expenses
were $6.3 million and $11.8 million for the three and six months ended
June 30, 2017, compared to $4.7 million and $9.1 million for the same
periods in 2016, respectively. The increase was predominantly related
to higher litigation-related expenses as well as to higher salaries,
benefits and other employee-related expenses.
-
Net Loss Attributable to Common Stockholders - Net loss
attributable to common stockholders was $24.0 million and $38.2
million for the three and six months ended June 30, 2017, as compared
to a net loss of $15.6 million and $31.3 million for the same periods
in 2016, respectively. In addition to the aforementioned changes in
R&D and G&A expenses, net loss attributable to common stockholders
also increased as a result of the recording of Preferred Stock
dividends, which include a one-time non-cash deemed dividend charge of
$6.1 million.
For more detailed information and analysis, see Dicerna's Quarterly
Report on Form 10-Q for the quarter ended June 30, 2017, which was filed
with the Securities and Exchange Commission (SEC (News - Alert)) on August 10, 2017.
Guidance
With the closing of its Preferred Stock transaction, Dicerna believes
that it has sufficient cash to fund the execution of its current
clinical and operating plan into 2019, which includes focusing its
resources on advancing its first three development programs into
proof-of-concept clinical studies and a fourth program into formal
preclinical development. This estimate assumes no additional funding
from new collaboration agreements or from additional financing events.
Conference Call
Management will host a conference call at 4:30 p.m. ET today to review
Dicerna's second quarter 2017 financial results and provide a general
business update. The conference call can be accessed by dialing (855)
453-3834 or (484) 756-4306 (international), and referencing conference
ID 38183667 prior to the start of the call. The call will also be
webcast via the Internet and will be available under the "Investors &
Media" section of the Dicerna website, www.dicerna.com.
A replay of the call will be available beginning at 7:30 p.m. ET on
August 10, 2017. To access the replay, please dial (855) 859-2056 or
(404) 537-3406, and refer to conference ID 38183667. The webcast will
also be archived on Dicerna's website.
About Dicerna Pharmaceuticals, Inc.
Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company focused on
the discovery and development of innovative, subcutaneously delivered
RNAi-based therapeutics for diseases involving the liver, including rare
diseases, chronic liver diseases, cardiovascular diseases, and viral
infectious diseases. Dicerna is leveraging its proprietary GalXC™ RNAi
technology platform to build a broad pipeline in these core therapeutic
areas, focusing on target genes where connections between target gene
and diseases are well understood and documented. Dicerna intends to
discover, develop and commercialize novel therapeutics either on its own
or in collaboration with pharmaceutical partners. For more information,
please visit www.dicerna.com.
About GalXCTM RNAi Technology Platform
GalXCTM is a proprietary technology platform invented by
Dicerna to discover and develop next-generation RNAi-based therapies
designed to silence disease-driving genes in the liver. Compounds
produced via GalXC are intended to be broadly applicable across multiple
therapeutic areas, including rare diseases, chronic liver diseases,
cardiovascular disease and viral infectious diseases. Using GalXC,
Dicerna scientists attach N-acetylgalactosamine sugars directly to the
extended region of our proprietary Dicer substrate short-interfering RNA
molecules, yielding multiple proprietary conjugate delivery
configurations. Many of the conjugates produced via GalXC incorporate a
folded motif known as a tetraloop in the extended region. The tetraloop
configuration, which is unique to Dicerna's GalXC compounds, allows
flexible and efficient conjugation to the targeting ligands, and
stabilizes the RNAi duplex which the Company believes will enable
subcutaneous delivery of its RNAi therapies to hepatocytes in the liver,
where they are designed to specifically bind to receptors on target
cells, potentially leading to internalization and access to the RNAi
machinery within the cells. The technology may offer several distinct
benefits, as suggested by strong preclinical data. These benefits
include: potency that is on par with or better than comparable
platforms; highly specific binding to gene targets; long duration of
action; and an infrequent subcutaneous dosing regimen.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements, including, for
example, our expected timeline and plans for development of DCR-PHXC and
other pipeline programs, and potential therapeutic benefits. Such
forward-looking statements are subject to risks and uncertainties that
could cause actual results to differ materially from those expressed or
implied in such statements. Applicable risks and uncertainties include
risks relating to our clinical and preclinical research and other risks
identified under the heading "Risk Factors" included in our most recent
Form 10-Q filing and in other future filings with the SEC. The
forward-looking statements contained in this press release reflect
Dicerna's current views with respect to future events, and Dicerna does
not undertake and specifically disclaims any obligation to update any
forward-looking statements.
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Dicerna Pharmaceuticals, Inc.
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Consolidated Balance Sheet Information
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(In thousands)
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June 30,
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December 31,
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2017
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|
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2016
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Cash and cash equivalents
|
|
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$
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38,777
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|
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$
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20,865
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Held-to-maturity investments
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|
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$
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49,953
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$
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25,009
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Total assets
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$
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94,723
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$
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51,252
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Total liabilities
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|
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$
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9,583
|
|
|
$
|
10,044
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Redeemable convertible preferred stock
|
|
|
$
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71,872
|
|
|
$
|
-
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Total stockholders' equity
|
|
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$
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13,268
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|
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$
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41,208
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Dicerna Pharmaceuticals, Inc.
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Consolidated Statements of Operations Information
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(In thousands, except share and per share data)
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For the Three Months Ended
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For the Six Months Ended
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June 30,
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June 30,
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2017
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|
|
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2016
|
|
|
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2017
|
|
|
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2016
|
|
|
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|
|
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|
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|
|
|
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Revenue
|
|
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$
|
252
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|
|
|
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-
|
|
|
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$
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385
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|
|
|
-
|
|
|
|
|
|
|
|
|
|
|
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|
|
|
|
|
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Operating expenses:
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|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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Research and development
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|
|
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9,320
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|
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|
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11,032
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|
|
|
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18,196
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|
|
|
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22,296
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General and administrative
|
|
|
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6,300
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|
|
|
|
4,656
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|
|
|
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11,796
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|
|
|
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9,140
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Total operating expenses
|
|
|
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15,620
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|
|
|
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15,688
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|
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|
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29,992
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|
|
|
|
31,436
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|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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|
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Loss from operations
|
|
|
|
(15,368
|
)
|
|
|
|
(15,688
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)
|
|
|
|
(29,607
|
)
|
|
|
|
(31,436
|
)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
|
|
|
|
143
|
|
|
|
|
66
|
|
|
|
|
181
|
|
|
|
|
121
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|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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Net loss
|
|
|
$
|
(15,225
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)
|
|
|
$
|
(15,622
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)
|
|
|
$
|
(29,426
|
)
|
|
|
$
|
(31,315
|
)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Dividends on redeemable convertible preferred stock
|
|
|
|
(2,622
|
)
|
|
|
|
-
|
|
|
|
|
(2,622
|
)
|
|
|
|
-
|
|
Deemed dividend related to beneficial conversion feature of
redeemable convertible preferred stock
|
|
|
|
(6,144
|
)
|
|
|
|
-
|
|
|
|
|
(6,144
|
)
|
|
|
|
-
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss attributable to common stockholders
|
|
|
$
|
(23,991
|
)
|
|
|
$
|
(15,622
|
)
|
|
|
$
|
(38,192
|
)
|
|
|
$
|
(31,315
|
)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share - basic and diluted
|
|
|
$
|
(1.15
|
)
|
|
|
$
|
(0.75
|
)
|
|
|
$
|
(1.84
|
)
|
|
|
$
|
(1.51
|
)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average shares outstanding - basic and diluted
|
|
|
|
20,794,193
|
|
|
|
|
20,726,108
|
|
|
|
|
20,792,925
|
|
|
|
|
20,706,388
|
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