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 operational results for
the first quarter ended March 31, 2017.
“While continuing to focus on our research and development
activities during the first quarter, we announced a major $70.0
million convertible preferred stock financing, which subsequently
closed on April 11, 2017,” said Douglas Fambrough, Ph.D., president
and chief executive officer of Dicerna. “The financing, led by Bain
Capital Life Sciences and a syndicate of current and new investors,
lends an additional level of validation to the potential of our
proprietary GalXC™ RNAi technology platform. Furthermore, the
funds, when combined with cash already on-hand, provide us with the
necessary resources to execute our stated strategy, which includes
pursuing the advancement of our core therapeutic programs.
Specifically, as we look forward from today and into 2019, we
expect to be able to advance our first three development programs,
including DCR-PHXC, a second undisclosed rare disease program and
DCR-HBV, into proof of concept studies, while progressing DCR-PCSK9
toward formal preclinical development, resulting in a number of
key, value-creating inflection points. In the nearer term, I look
forward to presenting new preclinical data for DCR-PHXC later this
year and to our expected Investigational New Drug (IND) or Clinical
Trial Application (CTA) filing at the end of 2017.”
GalXC™ Program Update
- During the first quarter of 2017,
Dicerna continued to advance its preclinical pipeline of
liver-targeted GalXC product candidates. These long-acting,
subcutaneously delivered therapeutic candidates are within the
Company’s strategic focus areas of rare diseases, chronic liver
diseases, cardiovascular diseases and viral infectious diseases.
Dicerna has prioritized four therapeutic programs that the Company
believes have favorable probabilities of success, including
DCR-PHXC, an undisclosed rare disease program, DCR-HBV and
DCR-PCSK9.
- Primary Hyperoxaluria Type 1 (PH1):
Dicerna continued to progress IND-enabling studies for its lead
product candidate, DCR-PHXC, which is being evaluated for the
treatment of patients with PH1. PH1 is a rare, inborn error of
metabolism in which the liver produces excessive levels of oxalate,
which in turn causes damage to the kidneys and other tissues in the
body. Dicerna plans to announce additional details about its
DCR-PHXC program at the Oxalosis and Hyperoxaluria Foundation’s
12th International Workshop on Primary Hyperoxaluria in Tenerife,
Spain, in July 2017, and is on track to file an IND submission or
CTA in late 2017. The Company plans to commence Phase 1 clinical
trials in the first quarter of 2018.
- Undisclosed Rare Disease Involving the
Liver: During the period, Dicerna initiated IND-enabling activities
for a second GalXC clinical candidate targeting an undisclosed rare
disease. The Company expects to file an IND application or CTA for
this program in the second quarter of 2018.
- Chronic Hepatitis B Virus (HBV):
Dicerna has recently initiated formal IND-enabling studies for
DCR-HBV, which targets HBV directly. Current therapies for HBV
rarely lead to a long-term immunological cure as measured by the
clearance of HBV surface antigen (HBsAg). 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 or CTA for this
program around the end of 2018.
- Cardiovascular Disease: 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 properties such as small subcutaneous injection volumes
and relatively infrequent dosing.
Financial Condition and Operating Results
- Cash Position – As of March 31,
2017, Dicerna had $32.9 million in cash and cash equivalents and
held-to-maturity investments, as compared to $45.9 million in cash
and cash equivalents and held-to-maturity investments as of
December 31, 2016. In addition, the Company had $1.1 million of
restricted cash equivalents as of March 31, 2017, which reflects
collateral securing the Company’s operating lease obligation.
- Research and Development (R&D)
Expenses – R&D expenses for the first quarter of 2017 were
$8.9 million, as compared to $11.3 million for the same period in
2016. The decrease was due primarily to a reduction in clinical and
manufacturing activities related to the Company’s now discontinued
PH1 and MYC programs, both of which will be fully wound down before
the end of the current year, offset by an overall increase in
manufacturing activities related to new candidates under the GalXC
platform; a decrease in platform-related expenses; and a decrease
in employee-related expenses, due largely to an overall decrease in
headcount from the prior year and slightly lower stock-based
compensation costs.
- General and Administrative (G&A)
Expenses – G&A expenses for the first quarter of 2017 were
$5.5 million, compared to $4.5 million for the same period in 2016.
The increase was primarily attributable to higher legal costs,
partially offset by a slight reduction in stock-based compensation
costs.
- Net Loss – Net loss for the
first quarter of 2017 was $14.2 million compared to a net loss of
$15.7 million for the same period in 2016.
- Financing – On March 30, 2017,
Dicerna entered into a stock purchase agreement with a syndicate of
current and new investors, led by Bain Capital Life Sciences, for
the sale of 700,000 shares of redeemable convertible preferred
stock (Preferred Stock) at a purchase price of $100.00 per share,
for total gross proceeds of $70.0 million. Participants in the
financing include Cormorant Asset Management, Domain Associates,
EcoR1 Capital, RA Capital and Skyline Ventures, among others. The
Preferred Stock is convertible into common shares at a conversion
price of $3.19 per share.
For more detailed information and analysis, see Dicerna’s
Quarterly Report on Form 10-Q for the quarter ended March 31, 2017,
which was filed with the Securities and Exchange Commission (SEC)
on May 8, 2017.
Subsequent Event
- On April 11, 2017, Dicerna announced
that it closed its stock purchase transaction for the sale of
redeemable convertible preferred stock, under which the Company
received gross proceeds of $70.0 million. Under 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.
Guidance
With the closing of its Preferred Stock transaction, Dicerna
believes that it has sufficient cash to fund the execution of its
current operating plan into 2019, which includes focusing its
resources on advancing its first three development programs into
proof of concept studies and a fourth program into formal
preclinical development. This estimate assumes no additional
funding from new partnership agreements or from additional
financing events.
Conference Call
Management will host a conference call at 4:30 p.m. ET today to
review the Company's first 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 7812180 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
May 8, 2017. To access the replay, please dial (855) 859-2056 or
(404) 537-3406, and refer to conference ID 7812180. The webcast
will also be archived on the Company's website.
About Dicerna Pharmaceuticals, Inc.
Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company
focused on the discovery and development of innovative RNAi-based
therapeutics for diseases involving the liver, including rare
diseases, chronic liver diseases, cardiovascular diseases, and
viral infectious diseases. The Company 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. The Company 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
(GalNAc) 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 we believe will enable subcutaneous delivery of
Dicerna's 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 anticipated use of proceeds from the
Preferred Stock transaction, expected timeline and plans for
development, potential collaborations, 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.
Dicerna Pharmaceuticals, Inc. Consolidated Balance
Sheet Information (In thousands) March
31, December 31, 2017 2016 Cash and
cash equivalents $ 22,873 $ 20,865 Held-to-maturity investments $
10,002 $ 25,009 Total assets $ 39,436 $ 51,252 Total liabilities $
10,347 $ 10,044 Total stockholders’ equity $ 29,089 $ 41,208
Dicerna Pharmaceuticals, Inc. Consolidated
Statements of Operations Information (In thousands, except
share and per share data)
For the Three Months EndedMarch
31,
2017 2016 Revenue $ 133 $ -
Operating expenses: Research and development 8,876 11,264
General and administrative 5,496 4,484 Total
operating expenses 14,372 15,748 Loss from
operations (14,239 ) (15,748 ) Interest income 38 55
Net loss $ (14,201 ) $ (15,693 ) Net loss per share -
basic and diluted $ (0.68 ) $ (0.76 ) Weighted average
shares outstanding - basic and diluted 20,791,644 20,646,793
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170508006191/en/
Investor:Rx Communications GroupPaula Schwartz,
917-322-2216pschwartz@rxir.comorMedia:SmithSolveAlex Van
Rees, 973-442-1555 ext. 111alex.vanrees@smithsolve.com
Dicerna Pharmaceuticals (NASDAQ:DRNA)
Historical Stock Chart
From Apr 2024 to May 2024
Dicerna Pharmaceuticals (NASDAQ:DRNA)
Historical Stock Chart
From May 2023 to May 2024