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, 2018.
“During the second quarter and subsequent period, we took a
significant step forward in the development of our lead program,
DCR-PHXC, as well as our broad pipeline of novel GalXCTM-based
therapeutics,” said Douglas M. Fambrough, Ph.D., president and
chief executive officer of Dicerna. “Our PHYOX Phase 1 trial of
DCR-PHXC for primary hyperoxaluria is progressing efficiently, with
10 of 16 PH type 1 (PH1) and PH type 2 (PH2) patients in the Group
B portion of the trial now dosed, and we expect to report interim
results later in the third quarter and publicly present trial
results in the fourth quarter of 2018. We continue to expect that
we will initiate a pivotal registration trial of DCR-PHXC in the
first quarter of 2019. In parallel, we continued to advance our
development program for DCR-HBVS for the treatment of chronic
hepatitis B virus toward clinical trials and in the fourth quarter
intend to file regulatory clearances that, if granted, will permit
us to commence human clinical trials for DCR-HBVS. Finally, with
the Alnylam litigation now behind us, we have seen a notable
increase in active discussions with potential partners and
collaborators. With these advancements, we remain on track to have
three programs in clinical trials in the first half of 2019 and
expect to achieve other development milestones in the coming
months.”
GalXC™ Pipeline Update
- Primary Hyperoxaluria (PH): Dicerna
continued to advance the PHYOX Phase 1 clinical trial of DCR-PHXC,
which is in development to treat all forms of PH. PHYOX is a
single-ascending dose study of DCR-PHXC in normal healthy
volunteers (NHVs) and patients with PH. PH is a family of severe,
rare, genetic liver disorders characterized by overproduction of
oxalate that often results in kidney failure.
- In May 2018, Dicerna dosed the first PH
patient with DCR-PHXC in the Group B portion of the PHYOX study.
Group B is an open-label, multi-center study enrolling up to 16
patients with PH1 and PH2. Group B consists of three cohorts of
patients with PH1 dosed at 1.5, 3 and 6 mg/kg, and an additional
fourth cohort that consists of patients with PH2 dosed at flexible
dosing levels. The fourth cohort runs in parallel to the other
three cohorts. Dicerna has dosed 10 out of 16 patients in Group
B—four PH1 patients in Cohort 1, four PH1 patients in Cohort 2, one
PH1 patient in Cohort 3, and one PH2 patient in Cohort 4.
- Dicerna completed the Group A portion
of the study in NHVs. Group A is a placebo-controlled,
single-blind, single center study that enrolled 25 NHVs. While the
study remains blinded to treatment assignment, topline results from
Group A evidence no serious adverse events (SAEs). 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 primary objective of PHYOX is to
evaluate the safety and tolerability of single doses of DCR-PHXC in
NHVs and patients with PH. The secondary objectives are to evaluate
the pharmacodynamic effect of single doses of DCR-PHXC on urinary
oxalate concentrations, as well as other biochemical markers, and
to characterize the pharmacokinetics of single doses of DCR-PHXC in
both groups.
- Dicerna expects to report interim
results from the PHYOX trial later in the third quarter and
publicly present trial results in the fourth quarter of 2018. The
Company intends to initiate a multi-dose Phase 2/3 study of
DCR-PHXC in the first quarter of 2019, pending positive
proof-of-concept data and regulatory feedback.
- In May 2018, the U.S. Food and Drug
Administration granted Orphan Drug Designation to DCR-PHXC for the
treatment of PH.
- In July 2018, the European Medicines
Agency’s Committee for Orphan Medicinal Products recommended
designating DCR-PHXC as an orphan medicinal product for the
treatment of PH in the European Union and the recommendation was
adopted by the European Commission in August 2018.
- Chronic Hepatitis B Virus (HBV):
Dicerna expects to file regulatory clearances for human clinical
trials in New Zealand and Australia for DCR-HBVS, in development
for the treatment of chronic HBV, during the fourth quarter of
2018. If the clearances are granted, the Company intends to begin
clinical studies shortly thereafter.
- Undisclosed Rare Disease Involving the
Liver: Dicerna is actively seeking a risk-sharing collaborator for
this second program focused on a serious rare disease, before it
files regulatory clearances to initiate a clinical trial. For
competitive reasons, the Company has not yet publicly disclosed the
target gene or disease.
- NASH Collaboration with Boehringer
Ingelheim (BI): Dicerna continued to advance the program, initially
focused on nonalcoholic steatohepatitis (NASH), in accordance with
the work plan for this collaboration.
Conclusion of Litigation
- On April 18, 2018, Dicerna and Alnylam
Pharmaceuticals 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 and neither party admitted wrongdoing as part
of the Settlement Agreement. Pursuant to the terms of the
Settlement Agreement, Dicerna paid to Alnylam an upfront payment of
$2.0 million, agreed to a future payment of $13.0 million and
issued 983,208 shares of common stock to Alnylam.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 June 30, 2018, which was filed with the Securities and
Exchange Commission (SEC) on August 8, 2018.
Financial Condition and Operating Results
- Cash Position – As of June 30,
2018, Dicerna had $82.3 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 June 30, 2018 and December 31,
2017, which reflects collateral securing the Company’s operating
lease obligation.
- Revenue – Dicerna recognized
$1.5 million and $3.1 million of revenue associated with the BI
Agreement, during the three and six months ended June 30, 2018,
respectively. The revenue 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 were $10.3 million and $20.2
million for the three and six months ended June 30, 2018,
respectively, as compared to $9.1 million and $17.8 million for the
same periods in 2017. The increase was due to higher direct R&D
expenses, partially offset by a reduction in platform-related
expenses.The increase in the three months ended June 30, 2018 was
primarily due to an increase in clinical study costs and an
increase in R&D salaries due to an increase in R&D
headcount compared to the same period in 2017. The increase in the
six months ended June 30, 2018 was primarily due to an increase in
toxicology studies and an increase in clinical studies.The decrease
in platform-related expenses for the three and six months ended
June 30, 2018 was primarily due to lab material and supply costs
and contract research costs associated with applying the Company’s
GalXC platform against gene targets.
- General and Administrative (G&A)
Expenses – G&A expenses were $4.8 million and $9.1 million
for the three and six months ended June 30, 2018, respectively, as
compared to $4.1 million and $8.2 million for the same periods in
2017. The increase is predominantly related to higher consulting
and corporate legal expenses.
- Litigation Expenses – Litigation
expenses, all related to the litigation with Alnylam, were $22.2
million and $25.4 million for the three and six months ended June
30, 2018, respectively, as compared to $2.2 million and $3.6
million for the same periods in 2017. The increase is predominantly
due to the $21.0 million of settlement expenses related to the
litigation settlement recorded in the second quarter of 2018.
- Net Loss Attributable to Common
Stockholders – Net loss attributable to common stockholders was
$35.6 million and $51.2 million for the three and six months ended
June 30, 2018, respectively, as compared to $24.0 million and $38.2
million for the same periods in 2017. The increase is attributable
to higher operating expenses, including the $21.0 million in
litigation settlement expenses, partially offset by higher
revenues, interest income and a decrease in dividends as a result
of the conversion of the redeemable convertible preferred stock in
December 2017.
For more detailed information and analysis, see Dicerna’s
Quarterly Report on Form 10-Q for the quarter ended June 30, 2018,
which was filed with the SEC on August 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 advancing DCR-PHXC through proof-of-concept trials
and into late-stage clinical development and initiating
proof-of-concept studies of DCR-HBVS in patients with HBV. 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 second 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 6588739 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 6588739. 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 the treatment
of 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.
The 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, expectations for discussions
and possible opportunities with potential partners and
collaborators, 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. Condensed
Consolidated Balance Sheet Information (Unaudited, in
thousands) June 30,
December 31, 2018 2017 Cash and cash
equivalents $ 42,426 $ 68,789 Held-to-maturity investments $ 39,875
$ 44,889 Total assets $ 89,715 $ 121,002 Total liabilities $ 25,140
$ 19,916 Total stockholders’ equity $ 64,575 $ 101,086
Dicerna Pharmaceuticals, Inc. Condensed
Consolidated Statements of Operations Information
(Unaudited, in thousands, except share and per share data)
Three Months Ended
June 30, Six Months Ended June 30, 2018
2017 2018 2017 Revenue from collaborative
arrangements $ 1,545 $ — $ 3,090 $ —
Operating expenses:
Research and development
10,339 9,068 20,232 17,811
General and administrative
4,760 4,066 9,095 8,188
Litigation expense
22,244 2,234 25,428
3,608
Total operating expenses
37,343 15,368 54,755
29,607
Loss from operations
(35,798 ) (15,368 ) (51,665 ) (29,607 )
Other income (expense):
Interest income
330 143 619 181
Interest expense
(176 ) — (176 ) —
Total other income, net
154 143 443 181
Net loss
(35,644 ) (15,225 ) (51,222 ) (29,426 )
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
$
(35,644
)
$ (23,991 ) $ (51,222 ) $ (38,192 )
Net loss per share attributable to
common stockholders— basic
and diluted
$ (0.68 ) $ (1.15 ) $ (0.98 ) $ (1.84 )
Weighted average common shares
outstanding—basic and diluted
52,555,751 20,794,193 52,141,849 20,792,925
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Investor:Rx Communications GroupPaula Schwartz,
917-322-2216pschwartz@rxir.comorMedia:SmithSolveAlex Van
Rees, 973-442-1555 ext. 111alex.vanrees@smithsolve.com
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