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 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.
- 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.
- 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 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
- 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)
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.
Dicerna Pharmaceuticals, Inc. Consolidated Balance
Sheet Information (In thousands)
June 30, December 31, 2017
2016 Cash and cash equivalents $ 38,777 $ 20,865
Held-to-maturity investments $ 49,953 $ 25,009 Total assets $
94,723 $ 51,252 Total liabilities $ 9,583 $ 10,044 Redeemable
convertible preferred stock $ 71,872 $ - Total stockholders’ equity
$ 13,268 $ 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 Six Months Ended June 30,
June 30, 2017 2016 2017 2016
Revenue $ 252 - $ 385 - Operating expenses: Research
and development 9,320 11,032 18,196 22,296 General and
administrative 6,300 4,656 11,796 9,140
Total operating expenses 15,620 15,688 29,992 31,436 Loss
from operations (15,368 ) (15,688 ) (29,607 ) (31,436 )
Interest income 143 66 181 121
Net loss $ (15,225 ) $ (15,622 ) $ (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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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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