[March 08, 2018] |
|
Dicerna Reports Fourth Quarter and Year End 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 fourth quarter and
full-year ended December 31, 2017.
"2017 was a transformative year for Dicerna, as we successfully
transitioned to a clinical stage company advancing our growing pipeline
of GalXC™-based RNAi therapeutics, and signed our first GalXC platform
collaboration agreement with Boehringer Ingelheim," said Douglas M.
Fambrough, Ph.D., president and chief executive officer of Dicerna.
"With our lead candidate, DCR-PHXC for the treatment of primary
hyperoxaluria (PH), now in a Phase 1 clinical trial, and our other
priority programs, including DCR-HBVS for chronic hepatitis B and an
undisclosed program for a rare disease of the liver, following quickly
behind, we plan to have three GalXC product candidates in clinical
development by early 2019.
"We entered this year with a significantly stronger balance sheet and
simplified capital structure, which we anticipate permits the funding of
our development activities through 2019. Subsequent to our recent $46.0
million follow-on offering of common stock, at December 31, 2017, we had
$113.7 million in cash and cash equivalents, which we expect will be
sufficient to advance our DCR-PHXC development program through
proof-of-concept trials and into advanced clinical development and
advance our DCR-HBVS development program into clinical proof-of-concept
studies in HBV patients, and provides a runway for us to further develop
our earlier stage pipeline programs. As we advance through 2018, we are
focused on seeking to achieve the key clinical milestones ahead of us,
including dosing the first PH patient in our Phase 1 trial, the
submission of clinical trial applications (CTAs) and/or investigational
new drug (IND) applications for our other priority programs, and sharing
clinical proof-of-concept data from our DCR-PHXC Phase 1 study in the
second half of 2018."
GalXC™ Research Collaboration
-
On October 27, 2017, Dicerna entered into a research collaboration and
license agreement with Boehringer Ingelheim (BI) to discover and
develop novel GalXC™ RNAi therapeutics for the treatment of chronic
liver diseases. The collaboration initially focuses on nonalcoholic
steatohepatitis (NASH), a chronic liver disease for which there is no
approved treatment option. The BI agreement is for the development of
product candidates against one target gene with an option for BI to
add the development of product candidates against a second target
gene. Dicerna granted BI a worldwide license to the product candidates
in connection with the agreement. Under the terms of the agreement, BI
agreed to pay Dicerna a non-refundable upfront payment of $10.0
million for the first target. During the term of the research program,
BI will reimburse Dicerna the cost of materials and third-party
expenses that have been included in the preclinical studies up to an
agreed-upon limit. Dicerna is eligible to receive up to $191.0 million
in potential development and commercial milestones, as well as royalty
payments on potential global net sales, subject to certain
adjustments, tiered from high single digits up to low double-digits.
BI's option to add a second target would provide for an option fee
payment and success-based development and commercialization milestones
and royalty payments to Dicerna.
GalXC™ Pipeline Program Update
-
During the fourth quarter of 2017, Dicerna continued to progress
development activities for its three priority programs, which include
DCR-PHXC for the treatment of PH, DCR-HBVS for the treatment of
chronic hepatitis B virus (HBV) infection, and a program for an
undisclosed rare disease involving the liver.
Additionally,
the Company further optimized its GalXC™ technology platform, enabling
the development of next generation GalXC molecules that can be applied
to any target gene and program. Improvements to Dicerna's GalXC
compounds include modification of the tetraloop end of the molecule
resulting in a substantially longer duration of action and improved
potency in animal models. These modifications are unique to Dicerna's
GalXC technology platform and are not gene-specific, so they are
easily applied to GalXC molecules targeting any gene. Dicerna plans to
utilize its next generation GalXC molecules in its DCR-PCSK9 program
for the treatment of hypercholesterolemia, and in additional programs
targeting chronic liver diseases, cardiovascular diseases, and
additional rare diseases.
-
Primary Hyperoxaluria: 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.
-
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.
-
On December 7, 2017, the Company announced it dosed the first
healthy volunteer in a Phase 1 clinical trial of DCR-PHXC,
named PHYOX. PHYOX is a single-ascending dose study of
DCR-PHXC in normal healthy volunteers (NHVs) and patients with
PH. The study is divided into two groups: Group A is a
placebo-controlled, single-blind, single-center study
enrolling up to 25 NHVs; Group B is an open-label,
multi-center study enrolling up to 16 patients with PH types 1
(PH1) and 2 (PH2). The primary objective of the study is to
evaluate the safety and tolerability of single doses of
DCR-PHXC in both groups. The secondary objectives are to
characterize the pharmacokinetics of single doses of DCR-PHXC
and its pharmacodynamic effect on biochemical markers,
including changes in urine oxalate concentrations.
-
Dicerna has submitted CTAs for the PHYOX study in Germany,
France and the Netherlands, and intends to submit additional
CTAs in other European countries later this year.
-
Dicerna plans to dose the first patient with PH in the second
quarter of 2018, and expects to have clinical proof-of-concept
(POC) data from the PHYOX study in the second half of 2018.
-
Dicerna expects to initiate a multi-dose Phase 2/3 study of
DCR-PHXC in the first quarter of 2019, pending positive POC
data and regulatory approvals.
-
Chronic Hepatitis B Virus: Dicerna has declared a GalXC-based
product candidate, DCR-HBVS, which targets HBV directly, and is
conducting formal non-clinical development studies. The Company
expects to file an IND in the U.S. or CTA during the fourth
quarter of 2018.
-
Undisclosed Rare Disease Involving the Liver: Dicerna advanced IND
application-enabling activities for a second GalXC-based clinical
candidate targeting a liver expressed gene involved in a serious
rare disease. For competitive reasons, the Company has not yet
publicly disclosed the target gene or disease. Dicerna plans to
seek a risk-sharing collaborator for this program before it files
an IND in the U.S. and/or CTA in Europe.
Financing Update
-
On December 18, 2017, Dicerna closed a follow-on public offering of
6,571,428 shares of its common stock with aggregate gross proceeds
totaling $46.0 million. In connection with the offering, Dicerna
entered into a Letter Agreement with the holders of all of the
outstanding shares of the Company's Redeemable Convertible Preferred
Stock (RCPS), resulting in the conversion of the RCPS into an
aggregate of approximately 24.2 million shares of the Company's common
stock at the completion of the follow-on public offering. As a result,
Dicerna's capital structure is now comprised of approximately 51.6
million shares of all common stock, and no RCPS shares remain
outstanding.
Financial Condition and Operating Results
-
Cash Position - As of December 31, 2017, Dicerna had $113.7
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. Additionally,
the Company had $0.7 million of restricted cash equivalents as of
December 31, 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 primarily to the addition of
funds generated by the Company's $70.0 million Private Placement,
which closed on April 11, 2017, the $46.0 million follow-on public
offering of shares of its common stock, which closed on December 18,
2017, and to the upfront payment received from BI.
-
Revenue - As of December 31, 2017, Dicerna recognized $1.2
million of revenue associated with the BI Agreement. This amount
represents partial amortization of the $10.0 million upfront payment
received from BI, as well as reimbursable third-party research
expenses which are billable to BI. Dicerna currently expects to
recognize the remaining $9.0 million of the aforementioned
non-refundable upfront payment on a straight-line basis through June
30, 2019. Dicerna does not expect to generate any product revenue for
the foreseeable future.
-
Research and Development (R&D) Expenses - R&D expenses for
the fourth quarter of 2017 were $9.8 million, as compared to $9.3
million for the same quarter in 2016. The increase was due to higher
direct research and development expenses, including drug substance,
toxicology study and manufacturing activities associated with the
Company's GalXC platform product candidates, partially offset by a
decrease in platform-related expenses, which decreased primarily due
to lower spending in discovery and early development programs, and by
a decrease in employee-related expenses.
Total R&D expenses
for the year ended December 31, 2017, decreased by $4.7 million, as
compared to the same period in 2016, despite an overall increase in
direct research and development expenses, which was due to higher
development and manufacturing activities associated with Dicerna's
GalXC platform product candidates, partially offset by a decrease in
comparative clinical activities related to the Company's non-GalXC
platform clinical trials, which were discontinued during 2016.
Platform-related expenses decreased primarily as a result of lower
spending in discovery and early development programs, which advanced
in 2017 into manufacturing and clinical testing. Employee-related
expenses decreased due to an overall decrease in headcount from 2016,
along with a decrease in non-cash stock-based compensation costs.
Dicerna
expects its overall R&D expenses to increase in 2018, as compared to
2017, as the Company continues spending on its development programs
and related resources, including the continued advancement of its lead
product candidate, DCR-PHXC, through clinical trials.
-
General and Administrative (G&A) Expenses - G&A expenses
for the fourth quarter of 2017 were $7.4 million, as compared to $4.9
million for the same quarter in 2016. The increase was largely due to
higher legal costs, most notably those incurred in connection with
litigation.
Total G&A expenses were $25.9 million and $18.3
million for the years ended December 31, 2017 and 2016, respectively.
The increase was primarily due to higher litigation costs, in addition
to higher salaries, benefits and other employee-related expenses.
Dicerna expects G&A expenses to decrease in 2018, as compared to 2017,
largely as the Company expects to incur lower legal expenses.
-
Net Loss Attributable to Common Stockholders - Net loss
attributable to common stockholders was $22.8 million for the fourth
quarter of 2017, as compared to a net loss of $14.0 million for the
same quarter in 2016. The overall increase in net loss attributable to
common stockholders was due to higher R&D and G&A expenses incurred
during the fourth quarter of 2017 as compared to the same period in
2016, as well as to the recording, in 2017, of $7.2 million of
non-cash dividends and deemed dividends related to and upon conversion
of the Company's RCPS.
Total net loss attributable to
common stockholders was $80.1 million and $59.5 million for the years
ended December 31, 2017 and 2016, respectively. The overall increase
in net loss attributable to common stockholders was due to the
recording, in 2017, of $20.1 million of non-cash dividends and deemed
dividends related to and upon the conversion of the RCPS, as well as
to higher G&A expenses, partially offset by higher collaboration and
grant revenues and lower R&D expenses.
For more detailed information and analysis, see Dicerna's Annual Report
on Form 10-K for the year ended December 31, 2017, which was filed with
the Securities and Exchange Commission (SEC (News - Alert)) on March 8, 2018.
Guidance
Dicerna believes that it has sufficient cash to fund the execution of
its current clinical and operating plan through 2019, which includes
focusing its resources on advancing its DCR-PHXC development program
through proof-of-concept trials and into advanced clinical development,
and advancing its DCR-HBVS development program into proof-of-concept
studies in HBV patients. 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 fourth quarter and year end 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 9997829 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 9997829. 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, viral infectious diseases, chronic liver diseases, and
cardiovascular 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 GalXC™ RNAi Technology Platform
GalXC™ is a proprietary technology platform invented by Dicerna to
discover and develop 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 the
Company's proprietary RNAi 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 duration
and usage of current cash and cash equivalents. 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-K 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)
|
|
|
|
|
|
|
|
December 31,
|
|
December 31,
|
|
|
2017
|
|
2016
|
Cash and cash equivalents
|
|
$
|
68,789
|
|
$
|
20,865
|
Held-to-maturity investments
|
|
$
|
44,889
|
|
$
|
25,009
|
Total assets
|
|
$
|
120,884
|
|
$
|
51,252
|
Total liabilities
|
|
$
|
19,646
|
|
$
|
10,044
|
Total stockholders' equity
|
|
$
|
101,238
|
|
$
|
41,208
|
|
|
|
|
|
|
|
|
Dicerna Pharmaceuticals, Inc.
|
Consolidated Statements of Operations Information
|
(In thousands, except share and per share data)
|
|
|
|
|
For the Three Months Ended
|
|
|
For the Year Ended
|
|
|
|
December 31,
|
|
|
December 31,
|
|
|
|
2017
|
|
|
2016
|
|
|
2017
|
|
|
2016
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue
|
|
$
|
1,418
|
|
|
133
|
|
$
|
2,277
|
|
|
295
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
|
|
|
|
|
|
|
|
|
|
|
|
|
development
|
|
|
9,786
|
|
|
9,337
|
|
|
36,983
|
|
|
41,694
|
General and
|
|
|
|
|
|
|
|
|
|
|
|
|
administrative
|
|
|
7,400
|
|
|
4,871
|
|
|
25,881
|
|
|
18,349
|
Total operating
|
|
|
|
|
|
|
|
|
|
|
|
|
expenses
|
|
|
17,186
|
|
|
14,208
|
|
|
62,864
|
|
|
60,043
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from
|
|
|
|
|
|
|
|
|
|
|
|
|
operations
|
|
|
(15,768)
|
|
|
(14,075)
|
|
|
(60,587)
|
|
|
(59,748)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income
|
|
|
179
|
|
|
53
|
|
|
539
|
|
|
235
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
$
|
(15,589)
|
|
$
|
(14,022)
|
|
$
|
(60,048)
|
|
$
|
(59,513)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Dividends on
|
|
|
|
|
|
|
|
|
|
|
|
|
redeemable
|
|
|
|
|
|
|
|
|
|
|
|
|
convertible
|
|
|
|
|
|
|
|
|
|
|
|
|
preferred stock
|
|
|
(3,378)
|
|
|
-
|
|
|
(10,111)
|
|
|
-
|
Deemed dividend
|
|
|
|
|
|
|
|
|
|
|
|
|
related to
|
|
|
|
|
|
|
|
|
|
|
|
|
beneficial
|
|
|
|
|
|
|
|
|
|
|
|
|
conversion feature
|
|
|
|
|
|
|
|
|
|
|
|
|
of redeemable
|
|
|
|
|
|
|
|
|
|
|
|
|
convertible
|
|
|
|
|
|
|
|
|
|
|
|
|
preferred stock
|
|
|
-
|
|
|
-
|
|
|
(6,144)
|
|
|
-
|
Deemed dividend on
|
|
|
|
|
|
|
|
|
|
|
|
|
conversion of
|
|
|
|
|
|
|
|
|
|
|
|
|
redeemable
|
|
|
|
|
|
|
|
|
|
|
|
|
convertible
|
|
|
|
|
|
|
|
|
|
|
|
|
preferred stock
|
|
|
(3,837)
|
|
|
-
|
|
|
(3,837)
|
|
|
-
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss
|
|
|
|
|
|
|
|
|
|
|
|
|
attributable to
|
|
|
|
|
|
|
|
|
|
|
|
|
common
|
|
|
|
|
|
|
|
|
|
|
|
|
stockholders
|
|
$
|
(22,804)
|
|
$
|
(14,022)
|
|
$
|
(80,140)
|
|
$
|
(59,513)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share
|
|
|
|
|
|
|
|
|
|
|
|
|
- basic and
|
|
|
|
|
|
|
|
|
|
|
|
|
diluted
|
|
$
|
(0.90)
|
|
$
|
(0.68)
|
|
$
|
(3.66)
|
|
$
|
(2.87)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average
|
|
|
|
|
|
|
|
|
|
|
|
|
shares outstanding
|
|
|
|
|
|
|
|
|
|
|
|
|
- basic and
|
|
|
|
|
|
|
|
|
|
|
|
|
diluted
|
|
|
25,205,415
|
|
|
20,753,001
|
|
|
21,917,415
|
|
|
20,719,761
|
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