Management to Host Conference Call Today at
4:30 p.m. ET
Dicerna Pharmaceuticals, Inc. (NASDAQ:DRNA), a leading developer
of investigational ribonucleic acid interference (RNAi)
therapeutics, today reported financial and operating results for
the third quarter ended September 30, 2017.
“We are extremely pleased with the progress we made during the
third quarter, and the fourth quarter is off to a great start,
highlighted by the achievement of two key milestones for the
Company,” said Douglas M. Fambrough, Ph.D., president and chief
executive officer of Dicerna. “These include the submission of a
Clinical Trial Application (CTA) in the United Kingdom for our lead
program, DCR-PHXC, which we are developing for primary
hyperoxaluria (PH), and the announcement of a strategic research
collaboration with Boehringer Ingelheim International GmbH (BI),
which, not only expands our GalXC™ pipeline to include a program
for nonalcoholic steatohepatitis (NASH), but is reflective of the
strength of our proprietary technology platform and our underlying
intellectual property.
“As we approach year end and look ahead to 2018, we are working
toward a number of other important catalysts for the Company. These
include CTA submissions in additional European countries and an
investigational new drug (IND) application filing in the U.S. for
DCR-PHXC, as well as the commencement of our Phase 1 clinical trial
of DCR-PHXC in the United Kingdom early in the first quarter of
2018. We also anticipate reporting human proof-of-concept data for
this program in the second half of 2018. At the same time, we are
focused on advancing our undisclosed GalXC program for a second
rare disease, for which we plan to file an IND application and/or
CTA in mid-2018, as well as our continued focus on DCR-HBVS for
chronic hepatitis B virus, DCR-PCSK9 for hypercholesterolemia, and
additional GalXC programs. Lastly, we are eager to embark on our
new, joint GalXC collaboration with BI in chronic liver diseases,
and we look forward to working closely with the company as we seek
to discover and develop RNAi therapeutics to treat NASH.”
GalXC™ Research Collaboration
On November 2, 2017, Dicerna announced a research collaboration
and license agreement with BI to discover and develop novel GalXC
RNAi therapeutics for the treatment of chronic liver diseases. The
collaboration will initially focus on NASH, a devastating, chronic
liver disease for which there is no approved treatment option. NASH
is caused by the buildup of fat in the liver, potentially leading
to liver fibrosis and cirrhosis. It has an especially high
prevalence among obese and diabetic patients, and is an area of
high unmet medical need. NASH is expected to soon become the most
common cause of advanced liver disorders, and it often necessitates
liver transplantation. Under the terms of the agreement, Dicerna
may receive more than $200 million from BI, including an upfront
payment, development and commercial milestone payments, and
research and development reimbursement for a GalXC candidate
product addressing an undisclosed NASH target. Dicerna is also
eligible to receive royalties tiered up to double-digits on
worldwide net sales.
GalXC™ Pipeline Program Update
- During the third quarter of 2017,
Dicerna continued to progress preclinical activities for its three
priority programs, which include DCR-PHXC for the treatment of PH,
a program for an undisclosed rare disease, and DCR-HBVS for the
treatment of chronic hepatitis B virus (HBV) infection. Dicerna’s
programs in clinical candidate selection include: DCR-PCSK9 for the
treatment of hypercholesterolemia, and multiple programs targeting
undisclosed targets in chronic liver diseases, cardiovascular
diseases, and additional rare diseases.
- Primary Hyperoxaluria: On October 16,
2017, Dicerna announced it had submitted a CTA for DCR-PHXC to the
Medicines and Healthcare products Regulatory Agency (MHRA) in the
United Kingdom. DCR-PHXC is in development for the treatment of all
types of PH. PH is a family of severe, rare, genetic liver
disorders characterized by overproduction of oxalate that often
results in kidney failure. Once the CTA is authorized by the MHRA
and Ethics Committee approval has been obtained, Dicerna plans to
conduct a Phase 1 trial as a randomized, single-blind,
placebo-controlled, single-ascending dose study, enrolling up to 25
healthy volunteer subjects and up to 16 patients with PH type 1
(PH1) and PH type 2 (PH2). The primary objective of the study is to
evaluate the safety and tolerability of single doses of DCR-PHXC,
with participants being enrolled into as many as five sequential
cohorts of increasing doses. Secondary endpoints include the
pharmacokinetics of DCR-PHXC and its pharmacodynamic effects on
oxalate biomarkers in plasma and urine. Dicerna plans to commence
human clinical trials in the first quarter of 2018 and expects to
have human proof of concept data in the second half of 2018.
Dicerna also plans to submit additional CTAs in other European
countries later this year and plans to file an IND in the U.S. in
the first quarter of 2018.On July 15, 2017, in a series of
presentations at the 12th International Workshop on Primary
Hyperoxaluria for Professionals, Patients and Families in Tenerife,
Spain, Dicerna presented new preclinical data suggesting the
potential utility of DCR-PHXC for treating all forms of PH. In
particular, Dicerna presented research from animal models
demonstrating how DCR-PHXC inhibits the lactate dehydrogenase A
(LDHA) gene, which Dicerna has identified as potentially being an
optimal therapeutic target in patients with PH. LDHA inhibition was
shown in animal models to reduce oxalate to normal or near-normal
levels in PH1, PH2 and ethylene glycol-induced hyperoxaluria (a
model for idiopathic PH. In animal studies, DCR-PHXC was shown to
be well tolerated with no adverse effects in the liver.During the
workshop, Dicerna also reported interim data from its Primary HYperoxaluria Observational Study (PHYOS), an international,
multicenter, observational study in patients with a genetically
confirmed diagnosis of PH1. PHYOS collected data on key biochemical
parameters, including changes in oxalate, glycolate, and other
metabolites, implicated in the pathogenesis of the disease. Twenty
(20) patients were enrolled in the study with a median age at
screening of 21 years (range 12-61 years). Over the six-month
observation period, the variability (coefficient of variation)
between 24-hour urine measurements of oxalate at different time
points was 28%. Dicerna has now completed PHYOS and hopes to use
the data to better understand the baseline PH1 disease state, which
will help guide long-term DCR-PHXC drug development plans. These
data will help the clinical team design future clinical studies
using 24-hour urinary oxalate excretion as a surrogate marker for
clinical benefits.
- 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.
- Chronic Hepatitis B Virus (HBV):
Dicerna declared a product candidate for DCR-HBVS, which targets
HBV directly, and is conducting formal IND application-enabling
work for this program. Current therapies for HBV rarely lead to a
long-term functional cure as measured by the clearance of HBV
surface antigen (HBsAg) and sustained HBV DNA 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
approximately at the end of 2018.
- Hypercholesterolemia: Dicerna continued
to develop its DCR-PCSK9 program, which targets the PCSK9 gene for
the treatment of hypercholesterolemia. The Company has selected a
provisional clinical candidate for the program and is exploring
ways to further optimize the program. 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.
Financial Condition and Operating Results
- Cash Position – As of September
30, 2017, Dicerna had $75.9 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 September 30, 2017, which
reflects collateral securing the Company’s operating lease
obligation.
- Research and Development (R&D)
Expenses – R&D expenses were $9.0 million and $27.2 million
for the three and nine months ended September 30, 2017, as compared
to $10.1 million and $32.4 million for the same periods in 2016,
respectively. The decrease in overall R&D expenses in both the
three and nine month periods ended September 30, 2017, is primarily
attributable to a reduction in employee-related expenses due to an
overall decrease in headcount from the same periods in 2016, along
with a decrease in non-cash stock-based compensation costs, as well
as a substantial decrease in platform-related expenses and to a
reduction in clinical manufacturing activities related to the
Company’s now discontinued DCR-PH1 and DCR-MYC programs, which were
not based on Dicerna’s GalXC platform. These decreases were
partially offset by an increase in direct research and development
expenses in both the three and nine month periods ended September
30, 2017, attributable to an overall increase in manufacturing
activities and in toxicology study costs related to Dicerna’s
product candidates under its GalXC platform. Dicerna expects
overall R&D expenses to increase during the fourth quarter of
2017, as compared to the third quarter of 2017, primarily as the
Company ramps up clinical initiatives associated with
DCR-PHXC.
- General and Administrative (G&A)
Expenses – G&A expenses were $6.7 million and $18.5 million
for the three and nine months ended September 30, 2017, compared to
$4.3 million and $13.5 million for the same periods in 2016,
respectively. The increases are predominantly related to higher
litigation costs 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
$19.1 million and $57.3 million for the three and nine months ended
September 30, 2017, as compared to a net loss of $14.2 million and
$45.5 million for the same periods in 2016, respectively. The
overall increases in net loss attributable to common stockholders
are due to the aforementioned changes in R&D and G&A
expenses, as well as to the recording of non-cash dividends in 2017
on the Redeemable Convertible Preferred Stock.
For more detailed information and analysis, see Dicerna’s
Quarterly Report on Form 10-Q for the quarter ended September 30,
2017, which was filed with the Securities and Exchange Commission
(SEC) on November 2, 2017.
Guidance
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. This
estimate assumes no new funding from additional collaboration
agreements or from external financing events.
Conference Call
Management will host a conference call at 4:30 p.m. ET today to
review Dicerna's third 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 95620574 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 approximately two hours after the
completion of the call and will remain available for seven days. To
access the replay, please dial (855) 859-2056 or (404) 537-3406,
and refer to conference ID 95620574. 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, Dicerna’s expected timeline and plans for
development of DCR-PHXC and other pipeline programs, expectations
related to the collaboration with BI, and guidance related to the
anticipated availability of current liquidity. 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 Dicerna’s clinical and preclinical research and
other risks identified under the heading "Risk Factors" included in
the Company’s 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.
Dicerna Pharmaceuticals,
Inc. Consolidated Balance Sheet Information (In
thousands) September 30, December 31,
2017 2016 Cash and cash equivalents $
30,960 $ 20,865 Held-to-maturity investments $ 44,959 $ 25,009
Total assets $ 82,726 $ 51,252 Total liabilities $ 10,522 $ 10,044
Redeemable convertible preferred stock $ 75,983 $ - Total
stockholders’ (deficit) equity $ (3,779 ) $ 41,208
Dicerna
Pharmaceuticals, Inc. Consolidated Statements of Operations
Information (In thousands, except share and per share
data)
For the Three Months
EndedSeptember 30,
For the Nine Months
EndedSeptember 30,
2017 2016 2017 2016 Revenue $
474 162 $ 859 162 Operating expenses: Research and
development 9,001 10,061 27,197 32,357 General and administrative
6,685 4,338 18,481 13,478 Total
operating expenses 15,686 14,399 45,678 45,835
Loss from operations (15,212 ) (14,237 ) (44,819 )
(45,673 ) Interest income 179 61 360
182 Net loss $ (15,033 ) $ (14,176 ) $ (44,459 )
$
(45,491 )
Dividends on redeemableconvertible
preferred stock
(4,111 ) - (6,733 ) -
Deemed dividend related tobeneficial
conversion featureof redeemable convertiblepreferred stock
- - (6,144 ) -
Net loss attributable tocommon
stockholders
$ (19,144 ) $ (14,176 ) $ (57,336 )
$
(45,491 ) Net loss per share - basic and diluted $ (0.92 ) $
(0.68 ) $ (2.76 )
$
(2.20 )
Weighted average sharesoutstanding - basic
and diluted
20,841,728 20,752,416 20,809,372 20,708,600
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Investors:Rx Communications GroupPaula Schwartz,
917-322-2216pschwartz@rxir.comorMedia:SmithSolveAlex Van
Rees, 973-442-1555 ext. 111alex.vanrees@smithsolve.com
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