LA JOLLA, Calif., March 2, 2017 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
leading the discovery and development of innovative medicines
targeting microRNAs, today reported financial results for the
fourth quarter and full year ended December
31, 2016 and provided a summary of corporate highlights and
pipeline update.
"2016 was a year of accomplishments and important key learnings
that position us for meaningful advancements in our clinical and
pre-clinical programs in 2017 and beyond. Based on these learnings,
I am even more convinced that targeting microRNAs offer significant
advancements in therapeutic potential for many diseases for which
there are currently no or limited treatment options," said
Paul Grint, M.D., President and
Chief Executive Officer of Regulus.
Fourth Quarter 2016 Highlights
- Initiated a Phase I MAD study for RG-012: Data from this
study in healthy volunteers will provide additional PK and safety
data and will be available in the second quarter 2017. The Phase II
HERA study is expected to resume in the second half of 2017
following completion of the MAD study.
- Announced two new clinical candidates at the Company's first
R&D Day: RGLS5040 (anti-miR-17) for the treatment of
cholestatic diseases; and RGLS4326 (anti-miR-27) for the treatment
of autosomal dominant polycystic kidney disease (ADPKD).
IND-enabling activities are currently underway. Regulus expects to
file the INDs in the second half of 2017.
- Attracted Key Talent: Dr. Timothy Wright joined Regulus in October as its
Chief R&D Officer.
Pipeline Update
RG-012 for treatment in patients with Alport syndrome
- Today, Regulus announced that it has modified the Phase II
RG-012 clinical development program in patients with Alport
syndrome to accelerate patient enrollment and potentially achieve
early proof of mechanism/proof of concept. The Phase II
program will include the HERA study, a randomized, double-blind,
placebo-controlled 48-week study in 30 patients with rapid renal
function decline. The objective of the HERA study will be to
evaluate the safety and efficacy of RG-012 for the treatment of
renal function decline in patients with Alport syndrome. In
parallel, a separate Phase I/II renal biopsy study in Alport
patients will be initiated to evaluate RG-012 tissue PK, target
engagement of miR-21, and effects on downstream genomic markers of
disease. Both studies are anticipated to begin enrollment
following the completion of the Phase I MAD study in Q2 2017.
Based on projected enrollment rates, data from the renal biopsy
study are expected in Q4 2017 and interim data from HERA are
expected in mid-2018.
RG-101 for the treatment of HCV
- Today, Regulus reported top-line results from the primary
endpoint analysis of its completed Phase II study of RG-101 in
combination with 4 weeks of once/daily approved anti-viral agents
Harvoni®, Olysio®, or Daklinza™. The
study enrolled 79 treatment naïve genotype 1 and 4 HCV patients
(Harvoni® combination arm, n=27, Olysio®
combination arm, n=27, Daklinza™ combination arm, n=25). The
results from this final analysis demonstrated significant virologic
response through 48 weeks of follow-up: RG-101 plus Harvoni
demonstrated 100% SVR48; RG-101 plus Olysio demonstrated 77% SVR48;
and RG-101 plus Daklinza™ demonstrated 84% SVR48. There were 10
relapses: six in the Olysio® combination arm (weeks 8,
20, 24 and 32); and four in the Daklinza™ combination arm (weeks 8,
12 and 24). RG-101 in combination with four weeks of
oral DAA therapy was generally well tolerated with the majority of
adverse events (AEs) considered mild or moderate with no AE-related
discontinuations. Commonly reported AEs included fatigue,
headache, and injection site reactions. There were four patients
across all arms that experienced adverse events of asymptomatic
transient hyperbilirubinemia (greater than or equal to two times
the upper limit of normal). No cases met criteria for Hy's law.
Over the course of the one-year study, five subjects reported
serious adverse events (SAE). As previously reported, there
was a single SAE of jaundice in a patient at seven weeks in the
Daklinza™ combination arm of the study. Since the interim
analysis in June 2016, there were two
additional SAEs: one was a trauma-related knee injury; and the
other was an upper respiratory infection.
- In January, the Company reported that it received written
communication from the FDA that the clinical development program
from RG-101 remains on clinical hold. The FDA has requested the
final safety and efficacy data from all on-going RG-101
pre-clinical and clinical studies before reconsidering the clinical
hold. These data are anticipated to be available in Q4 2017.
RGLS5040 for the treatment of cholestatic diseases
- IND-enabling activities are currently underway, and an IND
filing is currently on track for the second half of 2017.
RGLS4326 for the treatment of ADPKD
- IND-enabling activities are currently underway, and an IND
filing is currently on track for the second half of 2017.
Financial Results
Cash Position: Cash, cash equivalents and
short-term investments were $76.1
million at December 31, 2016,
compared with $115.3 million at
December 31, 2015.
Research and Development (R&D) Expenses:
R&D expenses were $15.0 million
and $64.3 million for the quarter and
year ended December 31, 2016,
respectively, compared to $12.8
million and $56.4 million for
the same periods in 2015. The increases in R&D expenses were
primarily driven by the advancement of our clinical programs and
increased investment in our preclinical pipeline.
General and Administrative (G&A)
Expenses: G&A expenses were $4.8 million and $18.4
million for the quarter and year ended December 31, 2016, respectively, compared to
$5.4 million and $19.1 million for the same periods in 2015. The
decreases in G&A expenses were principally driven by a decrease
in personnel costs, including non-cash stock-based
compensation.
Revenue: Revenue was less than $0.1 million and $1.2
million for the quarter and year ended December 31, 2016, respectively, compared to
$10.9 million and $20.8 million for the same periods in 2015.
Net Loss: Net loss was $20.0
million, or $0.38 per share,
and $81.8 million, or $1.55 per share, for the quarter and year ended
December 31, 2016, respectively,
compared to a net loss of $7.2
million, or $0.14 per share,
and $55.7 million, or $1.08 per share, for the same periods in
2015.
Conference Call Details
Regulus will host a conference call and webcast at 5:00 p.m. Eastern Time today to discuss third
quarter financial results and provide a general business
update. A live webcast of the call will be available online
at www.regulusrx.com. To access the call, please dial (877)
257-8599 (domestic) or (970) 315-0459 (international) and refer to
conference ID 75961316. To access the replay of the call, dial
(855) 859-2056 (domestic) or (404) 537-3406 (international),
passcode 75961316. The webcast and telephone replay will be
archived on the company's website following the call.
About Regulus
Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical
company leading the discovery and development of innovative
medicines targeting microRNAs. Regulus has leveraged its
oligonucleotide drug discovery and development expertise to develop
a well-balanced microRNA therapeutics pipeline complemented by a
rich intellectual property estate to retain its leadership in the
microRNA field. Regulus is advancing several programs in
renal, hepatic and central nervous systems diseases, both
independently and with our strategic alliance partners, Sanofi and
AstraZeneca. Regulus maintains its corporate headquarters in
La Jolla, CA. For more
information, please visit http://www.regulusrx.com.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are "forward-looking statements"
within the meaning of the Private Securities Litigation Reform Act
of 1995, including statements associated with the expected ability
of Regulus to undertake certain activities and accomplish certain
goals (including with respect to development and other activities
related to RG-101, RG-012, RGLS426 and RGLS456070), the projected
timeline of clinical development activities, and expectations
regarding future therapeutic and commercial potential of Regulus'
business plans, technologies and intellectual property related to
microRNA therapeutics and biomarkers being discovered and developed
by Regulus. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Words such
as "believes," "anticipates," "plans," "expects," "intends,"
"will," "goal," "potential" and similar expressions are intended to
identify forward-looking statements. These forward-looking
statements are based upon Regulus' current expectations and involve
assumptions that may never materialize or may prove to be
incorrect. Actual results and the timing of events could
differ materially from those anticipated in such forward-looking
statements as a result of various risks and uncertainties, which
include, without limitation, risks associated with the process of
discovering, developing and commercializing drugs that are safe and
effective for use as human therapeutics, and in the endeavor of
building a business around such drugs. These and other risks
concerning Regulus' financial position and programs are described
in additional detail in Regulus filings with the Securities and
Exchange Commission. All forward-looking statements contained
in this press release speak only as of the date on which they were
made. Regulus undertakes no obligation to update such statements to
reflect events that occur or circumstances that exist after the
date on which they were made.
Regulus
Therapeutics Inc.
|
Selected Financial
Information
|
Statement of
Operations
|
(In thousands,
except share and per share data)
|
|
|
|
Three months
ended
December
31,
|
|
Year
ended
December
31,
|
|
|
2016
|
|
|
2015
|
|
|
2016
|
|
2015
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
18
|
|
$
|
10,860
|
|
$
|
1,194
|
|
$
|
20,759
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
14,979
|
|
12,794
|
|
64,305
|
|
56,387
|
General and
administrative
|
|
4,783
|
|
5,427
|
|
18,391
|
|
19,130
|
Total operating
expenses
|
|
19,762
|
|
18,221
|
|
82,696
|
|
75,517
|
Loss from
operations
|
|
(19,744)
|
|
(7,361)
|
|
(81,502)
|
|
(54,758)
|
Other (expense)
income, net
|
|
(271)
|
|
139
|
|
(338)
|
|
(1,008)
|
Loss before income
taxes
|
|
(20,015)
|
|
(7,222)
|
|
(81,840)
|
|
(55,766)
|
Income tax (expense)
benefit
|
|
|
(5)
|
|
|
(4)
|
|
4
|
|
18
|
Net loss
|
|
$
|
(20,020)
|
|
$
|
(7,226)
|
|
$
|
(81,836)
|
|
$
|
|
(55,748)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.38)
|
|
$
|
(0.14)
|
|
$
|
(1.55)
|
|
$
|
|
(1.08)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
|
52,923,713
|
|
|
52,477,489
|
|
|
52,813,474
|
|
|
51,411,353
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Regulus
Therapeutics Inc.
|
Balance
Sheets
|
(In
thousands)
|
|
|
|
|
December
31,
2016
|
|
December 31,
2015
|
|
|
|
Assets
|
|
|
|
|
Cash, cash
equivalents, short-term investments and restricted cash
|
|
$
|
76,111
|
|
$
|
115,319
|
Other current
assets
|
|
11,363
|
|
18,939
|
Non-current
assets
|
|
13,187
|
|
6,825
|
Total
assets
|
|
$
|
100,661
|
|
$
|
141,083
|
Liabilities and
stockholders' equity
|
|
|
|
|
Current
liabilities
|
|
$
|
13,735
|
|
$
|
11,438
|
Total deferred
revenue
|
|
2,065
|
|
3,259
|
Term Loan, less debt
issuance costs
|
|
19,802
|
|
—
|
Other long-term
liabilities
|
|
8,984
|
|
2,308
|
Stockholders'
equity
|
|
56,075
|
|
124,078
|
Total
liabilities and stockholders' equity
|
|
$
|
100,661
|
|
$
|
141,083
|
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visit:http://www.prnewswire.com/news-releases/regulus-reports-fourth-quarter-2016-financial-results-and-pipeline-update-300417255.html
SOURCE Regulus Therapeutics Inc.