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 first quarter ended March 31, 2018.
“Dicerna is off to a strong start in 2018 with the achievement
of a number of important milestones for our lead GalXC™ program,
DCR-PHXC, currently in development for primary hyperoxaluria,” said
Douglas M. Fambrough, Ph.D., president and chief executive officer
of Dicerna. “With the healthy volunteer phase of the PHYOX Phase 1
study now complete, we have initiated the second phase of the
trial, and dosing of the first primary hyperoxaluria patient with
DCR-PHXC is imminent. While the PHYOX study remains blinded to
treatment assignment, the early safety and tolerability data in
healthy volunteers are very encouraging. The FDA’s acceptance of
our investigational new drug application (IND) for DCR-PHXC gives
us the greenlight to expand the trial into the U.S. We expect 2018
to be an eventful year for Dicerna as we hope to achieve clinical
proof-of-concept results from this multi-center study in the second
half of 2018. Additionally, we look forward to achieving key
milestones for our other priority programs, DCR-HBVS for chronic
hepatitis B virus and an undisclosed program for a rare disease of
the liver, including the filing of regulatory clearances to
initiate clinical trials later this year.
“On an important note, we recently announced that we have
resolved all ongoing litigation between Dicerna and Alnylam
Pharmaceuticals. This settlement allows us to advance all of our
existing and currently anticipated pipeline programs while
maintaining a strong balance sheet. With the litigation now behind
us, we are able to focus all of our resources on progressing our
clinical programs, discovery programs, and partnering initiatives
in accordance with our strategy and timelines. In a separate
disclosure, we reported that Bruce Peacock, a member of the Board
of Directors, will be stepping down from his board position at
Dicerna’s Annual Meeting of Stockholders in June. I would like to
thank Bruce for his service and wise counsel to the Company over
the past four years and wish him well in his future endeavors.”
GalXC™ Pipeline Update
- GalXC Priority Programs: During the
first quarter of 2018, Dicerna continued to actively progress the
development of its three priority programs and remains on track to
advance these programs into clinical development by early 2019.
- Primary Hyperoxaluria (PH): During the
first quarter of 2018, Dicerna advanced its PHYOX Phase 1 clinical
trial of DCR-PHXC in healthy volunteers and patients with PH.
DCR-PHXC is the only potential treatment in development for all
forms of the disease. PH is a family of severe, rare, genetic liver
disorders characterized by overproduction of oxalate that often
results in kidney failure.
- Dicerna has now completed dosing of all
normal healthy volunteers (NHVs) in the Group A portion of the
PHYOX study and dosing of the first PH patient with DCR-PHXC in the
Group B portion of the study is imminent. While the study remains
blinded to treatment assignment, topline results from Group A show
there were no serious adverse events (SAEs) and no
discontinuations. There have been two mild-to-moderate transient
injection site reactions at doses of 6 and 12 mg/kg involving
erythema and tenderness, lasting no more than 36 hours.
- The PHYOX trial is a Phase 1
single-ascending dose study of DCR-PHXC in NHVs and patients with
PH. The study is divided into two groups: Group A is a
placebo-controlled, single-blind, single-center study that has
enrolled 25 NHVs; Group B is an open-label, multi-center study
enrolling up to 16 patients with primary hyperoxaluria type 1 (PH1)
and type 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 in NHVs and patients
with PH, and to evaluate the pharmacodynamic effect of single doses
of DCR-PHXC on biochemical markers, including but not limited to,
changes in urine oxalate concentrations.
- On March 30, 2018, Dicerna received a
notice from the U.S. Food and Drug Administration (FDA) indicating
the acceptance of its IND to conduct the DCR-PHXC Phase 1 study in
the U.S. In addition to this active IND, the Company has active
CTAs in the United Kingdom, France and Germany, having received the
appropriate regulatory and ethical approvals for the trial in these
countries. A CTA has been submitted and is pending approval in the
Netherlands.
- Dicerna is on track to have clinical
proof-of-concept (POC) data from the PHYOX trial 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 feedback.
- Chronic Hepatitis B Virus (HBV):
Dicerna expects to file regulatory clearances to initiate a
clinical trial for its DCR-HBVS program in development for chronic
HBV, during the fourth quarter of 2018, and expects to begin
clinical studies shortly thereafter.
- Undisclosed Rare Disease Involving the
Liver: Dicerna continued to prepare for its regulatory filing for
its second GalXC-based clinical candidate targeting a liver
expressed gene involved in a serious rare disease. The Company is
seeking a risk-sharing collaborator for this program before it
files regulatory clearances to initiate a clinical trial, likely in
the second half of 2018. For competitive reasons, the Company has
not yet publicly disclosed the target gene or disease.
- NASH Collaboration with Boehringer
Ingelheim (BI): During the first quarter of 2018, Dicerna continued
to successfully execute on the program in accordance with the work
plan for this collaboration. The collaboration is focused on
chronic liver diseases, with an initial focus on nonalcoholic
steatohepatitis (NASH).
- GalXC Platform Improvement: During the
first quarter of 2018, Dicerna continued to optimize its GalXC
technology platform, which has enabled the development of next
generation GalXC molecules that can be applied to any target gene.
Next generation GalXC improvements have yielded a longer duration
of action and higher potency of target gene silencing against
multiple targets in animal models. Dicerna anticipates utilizing
its next generation GalXC molecules in its DCR-PCSK9 program for
the treatment of hypercholesterolemia as well as in additional
programs.
Conclusion of Litigation
- On April 18, 2018, Dicerna and Alynlam
entered into a Confidential Settlement Agreement and General
Release (the Settlement Agreement), resolving all ongoing
litigation between the two companies. The terms of the Settlement
Agreement include mutual releases and dismissals with prejudice of
all claims and counterclaims in the litigation between Dicerna and
Alnylam. Dicerna denies wrongdoing and did not admit wrongdoing as
part of the Settlement Agreement. Dicerna has agreed to make the
following payments to Alnylam: (i) a $2.0 million upfront payment
in cash; (ii) an additional $13.0 million in cash paid over the
next four years, to be paid as 10% of any upfront or first year
cash consideration that the Company receives pursuant to future
GalXC technology-based collaborations (excluding any amounts
received or to be received by the Company from its existing
collaboration with BI); (iii) issuance of 983,208 shares of Dicerna
common stock. The Settlement Agreement does not include any
licenses to any intellectual property from either party and does
not include any royalties or milestones related to product
development. For additional detail, please see Dicerna’s Quarterly
Report on Form 10-Q for the quarter ended March 31, 2018, which was
filed with the Securities and Exchange Commission (SEC) on May 14,
2018.
Financial Condition and Operating Results
- Cash Position – As of March 31,
2018, Dicerna had $97.8 million in cash and cash equivalents and
held-to-maturity investments, as compared to $113.7 million in cash
and cash equivalents and held-to-maturity investments as of
December 31, 2017. Additionally, the Company had $0.7 million of
restricted cash equivalents as of March 31, 2018, which reflects
collateral securing the Company’s operating lease obligation.
- Revenue – For the three-month
period ended March 31, 2018, Dicerna recognized $1.5 million of
revenue associated with the BI Agreement. This amount primarily
represents partial amortization of the $10.0 million non-refundable
upfront payment from BI, as well as certain reimbursable
third-party research expenses which are billable to BI. Dicerna
expects to recognize the remainder of the initial transaction price
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 first quarter of 2018 were
$9.9 million, as compared to $8.7 million for the same quarter in
2017. The increase was predominantly due to higher direct research
and development and employee-related expenses, partially offset by
lower platform-related expenses.
- General and Administrative (G&A)
Expenses – G&A expenses for the first quarter of 2018 were
$7.5 million, as compared to $5.5 million for the same quarter in
2017. The increase was predominantly related to higher legal costs
associated with the litigation with Alnylam, and to higher
corporate legal expenses, partially offset by lower consulting
expenses. We expect our G&A expenses to increase significantly
in the second quarter of 2018, as compared to the three-month
period ended March 31, 2018, as a result of the one-time charges
associated with the Settlement Agreement.
- Net Loss – Net loss was $15.6
million for the first quarter of 2018, as compared to a net loss of
$14.2 million for the same quarter in 2017. This increase is
attributable to higher operating expenses, offset by higher
revenues and interest income.
For more detailed information and analysis, see Dicerna’s
Quarterly Report on Form 10-Q for the quarter ended March 31, 2018,
which was filed with the Securities and Exchange Commission (SEC)
on May 14, 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 first quarter 2018 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 9666285 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 9666285. 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 GalXCTM RNAi Technology Platform
GalXCTM 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, viral infectious
diseases, chronic liver diseases, and cardiovascular 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 seen in preclinical studies
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-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, 2018
2017 Cash
and cash equivalents $ 43,046 $ 68,789
Held-to-maturity investments $ 54,770 $ 44,889 Total assets $
104,074 $ 121,002 Total liabilities $ 16,188 $ 19,916 Total
stockholders’ equity $ 87,886 $ 101,086
Dicerna
Pharmaceuticals, Inc. Consolidated Statements of Operations
Information (In thousands, except share and per share
data)
For the Three Months Ended March
31,
2018 2017 Revenue $ 1,545 - Operating
expenses: Research and development 9,893 8,743 General and
administrative 7,519 5,496 Total operating expenses 17,412 14,239
Loss from operations (15,867) (14,239) Interest
income 288 38 Net loss $ (15,579) $ (14,201) Net loss
per share - basic and diluted $ (0.30) $ (0.68) Weighted
average shares outstanding - basic and diluted 51,723,349
20,791,644
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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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