LA JOLLA, Calif., Nov. 8, 2018 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
focused on the discovery and development of innovative medicines
targeting microRNAs, today reported financial results for the third
quarter ended September 30, 2018 and
provided a summary of recent events.
Third Quarter 2018 Corporate Highlights and Recent
Updates
- Amended and restructured its Collaboration and License
Agreement with Sanofi: In November, Regulus and Sanofi agreed
to restructure their Collaboration and License Agreement and
immediately transfer development responsibilities of RG-012 for the
treatment of Alport syndrome to Sanofi. Sanofi will assume all
future costs and development activities associated with the
advancement of RG-012, currently in Phase 2 for the treatment of
Alport syndrome. Under the terms of the Amendment, Regulus is
eligible to receive approximately $7
million in upfront and material transfer payments. Regulus
is also eligible to receive up to $40
million in development milestone payments, including a
$10 million payment for an interim
enrollment milestone. In addition, Sanofi will reimburse Regulus
for certain out-of-pocket expenses associated with transition
activities and assume Regulus' upstream license royalty
obligations.
- Initiated new chronic mouse toxicity study for RGLS4326;
data anticipated in Q1 2019: In September 2018, and in consultation with the FDA,
the Company initiated a new 27-week chronic mouse toxicity study
for RGLS4326 for the treatment of autosomal dominant polycystic
kidney disease (ADPKD), incorporating several changes believed to
address the unexpected findings in the earlier terminated chronic
mouse toxicity study. This study is ongoing, and data are
anticipated in Q1 2019. The Company anticipates the advancement of
the RGLS4326 clinical program upon successful resolution of the
unexpected findings.
- Completed reverse stock split and regained compliance with
NASDAQ listing requirements: In a special meeting of
stockholders, held September 28,
2018, stockholders voted to approve a proposal authorizing
the Board of Directors of the Company to amend the Company's
certificate of incorporation to affect a reverse stock split of
Regulus' outstanding common shares. Following the special meeting
of stockholders, the Board of Directors approved a 1-for-12 reverse
stock split. The Company's shares began trading on a split-adjusted
basis on October 4, 2018. On
October 18, 2018, The Nasdaq Stock
Market notified Regulus that it had regained compliance with the
minimum bid price requirement for continued listing on The Nasdaq
Global Market.
"The recently announced Sanofi restructuring represents a
significant achievement for Regulus, bringing non-dilutive capital
and eliminating future spend for this partnered program.
Importantly, we are eligible to receive approximately $17 million in upfront and milestone payments
anticipated over the next twelve months," said Jay Hagan, President and Chief Executive Officer
of Regulus. "In July, we established several near-term key
objectives, including reducing our cash burn, restructuring the
Sanofi collaboration, advancing our prioritized pipeline, including
ADPKD, and positioning other programs for business development
opportunities. Regulus has made significant progress towards
completing all of these objectives, and we look forward to the
continued advancement of our ADPKD and HBV programs."
Program Updates
- Presented preclinical data supporting RGLS4326 as a novel
therapeutic for the treatment of ADPKD at the American Society of
Nephrology's Kidney Week 2018: In October 2018, Regulus presented three posters
during the American Society of Nephrology's Kidney Week describing
the discovery and preclinical evaluation of RGLS4326, a novel
single-stranded, chemically-modified oligonucleotide designed to
preferentially target the kidney and inhibit miR-17 functions to
treat ADPKD. In preclinical studies, RGLS4326 inhibited miR-17
activity and reduced cyst formation and proliferation of primary
cyst cultures derived from human donors with ADPKD. The data
presented also demonstrated that RGLS4326 has favorable
pharmacokinetic and pharmacodynamic profiles in normal and
polycystic kidney disease (PKD) mouse models, where preferential
distribution to kidney and localization to collecting duct cysts
were evident. Furthermore, data demonstrated that RGLS4326 directly
modulates expression of genes implicated in ADPKD pathogenesis
including Pkd1 and Pkd2, and conferred efficacy in multiple PKD
mouse models following subcutaneous administration.
- Advancement of Hepatitis B virus (HBV) Programs: The
Company has identified multiple human microRNA targets that serve
as host factors for the virus. Compounds directed at modulating
these targets are under active in vitro and in vivo
preclinical investigation. The Company believes that targeting a
host factor in the liver represents a unique mechanism of action
for treatment of the virus compared to other programs in
development and holds the potential for achieving a functional
cure. The Company currently expects to file an IND for the HBV
program in the second half of 2019, with the potential of achieving
human proof-of-concept in a Phase 1 study.
- Additional Program Updates: In September 2018, the Company announced progress of
its glioblastoma multiforme (GBM) and non-alcoholic steatohepatitis
(NASH) preclinical programs. The Company's GBM program, targeting
microRNA-10b, demonstrated statistically significant
improvements in survival as both a monotherapy as well as in
combination with temozolomide (TMZ) in an orthotopic GBM animal
model. The Company's lead NASH candidate demonstrated improvement
in key endpoints, including NAFLD Activity Score (NAS), liver
transaminases, hyperglycemia, and disease-related gene expression.
In the diet-induced NASH mouse model (Amylin model) after 2-4
weekly doses, early onset of improvement across multiple disease
parameters including liver triglycerides and blood levels of
transaminases was observed. The Company plans to seek partners to
further advance these programs' development.
Third Quarter 2018 Financial Results
Cash Position: As of September 30, 2018, Regulus had $20.5 million in cash, cash equivalents and
short-term investments. Under the terms of the Sanofi
Amendment, Regulus is eligible to receive approximately
$7 million in upfront and material
transfer payments. Including these additional proceeds, the
Company expects its cash runway to extend through Q2 2019.
Research and Development (R&D) Expenses:
R&D expenses were $6.9 million
and $28.7 million for the three and
nine months ended September 30, 2018,
respectively, compared to $12.7
million and $42.7 million for
the same periods in 2017. The decreases were primarily attributable
to the pausing of the RG-012 and RGLS4326 programs early in the
third quarter of 2018, the discontinuation of the RG-101 and
RGLS5040 programs in 2017 and reductions in personnel-related
expenses primarily attributable to our corporate
restructurings.
General and Administrative (G&A)
Expenses: G&A expenses were $3.0 million and $10.1
million for the three and nine months ended September 30, 2018, respectively, compared to
$2.7 million and $13.8 million for the same periods in 2017. The
increase for the three months ended September 30, 2018 as compared to the three
months ended September 30, 2017 was
driven by non-recurring severance charges recorded in the third
quarter of 2018 in connection with our July
2018 corporate restructuring. The decrease for the nine
months ended September 30, 2018 as
compared to the nine months ended September
30, 2017 was driven by non-recurring severance charges and
non-recurring, non-cash stock-based compensation charges recorded
in connection with our May 2017
corporate restructuring.
Revenue: Revenue was less than $0.1 million for each of the three and nine
months ended September 30, 2018 and
2017.
Net Loss: Net loss was $10.3
million, or $1.18 per share
(basic and diluted), and $40.1
million, or $4.62 per share
(basic and diluted), for the three and nine months ended
September 30, 2018, respectively,
compared to $15.8 million, or
$2.11 per share (basic and diluted),
and $57.5 million, or $10.52 per share (basic and diluted), for the
same periods in 2017. Historical and current period net loss
per share values have been retroactively adjusted to reflect our
October 2018 reverse stock split.
Upcoming Events
- On November 14, 2018, Regulus
will present survival data on its lead anti-miR candidate targeting
microRNA-10b for glioblastoma multiforme at the Society for
Neuro-Oncology Meeting in New Orleans,
Louisiana.
- On November 14, 2018, Regulus
will present a corporate overview at the Stifel 2018 Healthcare
Conference in New York.
About Regulus
Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical
company focused on the discovery and development of innovative
medicines targeting microRNAs. Regulus has leveraged its
oligonucleotide drug discovery and development expertise to develop
a pipeline complemented by a rich intellectual property estate in
the microRNA field. 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-012, RGLS4326, or its Hepatitis B virus program),
Regulus' estimated cash runway and anticipated cost savings
associated with its planned reduction in workforce, 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
|
Condensed
Statement of Operations
|
(In thousands,
except share and per share data)
|
|
|
|
Three months
ended September
30,
|
|
Nine months
ended September
30,
|
|
|
2018
|
|
2017
|
|
2018
|
|
2017
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
18
|
|
$
|
18
|
|
$
|
54
|
|
$
|
54
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
6,879
|
|
12,697
|
|
28,720
|
|
42,727
|
General and
administrative
|
|
2,993
|
|
2,736
|
|
10,115
|
|
13,752
|
Total operating
expenses
|
|
9,872
|
|
15,433
|
|
38,835
|
|
56,479
|
Loss from
operations
|
|
(9,854)
|
|
(15,415)
|
|
(38,781)
|
|
(56,425)
|
Other expense,
net
|
|
(419)
|
|
(420)
|
|
(1,365)
|
|
(1,171)
|
Loss before income
taxes
|
|
(10,273)
|
|
(15,835)
|
|
(40,146)
|
|
(57,596)
|
Income tax
benefit
|
|
|
-
|
|
|
7
|
|
-
|
|
139
|
Net loss
|
|
$
|
(10,273)
|
|
$
|
(15,828)
|
|
$
|
(40,146)
|
|
$
|
(57,457)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(1.18)
|
|
$
|
(2.11)
|
|
$
|
(4.62)
|
|
$
|
(10.52)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
|
8,703,626
|
|
|
7,506,529
|
|
|
8,688,831
|
|
|
5,463,096
|
|
|
September
30, 2018
|
|
December 31, 2017
|
|
|
(Unaudited)
|
|
|
|
Cash, cash
equivalents and short-term investments
|
|
$
|
20,517
|
|
$
|
60,074
|
Total
assets
|
|
34,768
|
|
77,809
|
Term loan, less debt
issuance costs
|
|
19,069
|
|
19,859
|
Stockholders'
equity
|
|
|
1,578
|
|
|
35,216
|
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SOURCE Regulus Therapeutics Inc.