LA JOLLA, Calif., March 12, 2020 /PRNewswire/ -- Regulus
Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company
focused on the discovery and development of innovative medicines
targeting microRNAs (the "Company" or "Regulus"), today reported
financial results for the fourth quarter and year ended
December 31, 2019 and provided a
corporate update.
"We have made significant progress over the past few months,
including receiving notification from FDA of their decision to lift
the partial clinical hold on our Phase 1 multiple ascending dose
clinical study for RGLS4326, enabling the initiation
of dosing of the second cohort of that study for the treatment
of autosomal dominant polycystic kidney disease this past
February," said Jay Hagan, CEO of Regulus. "This important
milestone, coupled with the closing of the second tranche of
financing, enables us to advance the program toward key data
read-outs."
Corporate Highlights
Closed $26 Million Second
Tranche of Private Financing: In December 2019, following the announcement of the
Company's plan to recommence the Phase 1 Multiple Ascending Dose
("MAD") clinical study of RGLS4326 for the treatment of autosomal
dominant polycystic kidney disease ("ADPKD") in the first quarter
of 2020, the Company completed a second and final closing under the
May 2019 securities purchase
agreement, pursuant to which the Company sold and issued 3,288,390
shares of non-voting Class A-2 convertible preferred stock, in lieu
of shares of common stock, at a price of $6.66 per share, and accompanying warrants to
purchase an aggregate of 32,883,900 shares of common stock at a
price of $0.125 for each share of
common stock underlying such warrants. Each share of the non-voting
Class A-2 convertible preferred stock is convertible into 10 shares
of common stock, subject to certain beneficial ownership conversion
limitations. The warrants are exercisable for a period of five
years following the date of issuance and have an exercise price of
$0.666 per share, pursuant to
proportional adjustments in the event of stock splits or
combinations or similar events. Together with the first tranche,
which closed in May 2019, the Company
raised a total of $42.7 million from
the private financing, which the Company expects will provide cash
resources to fund planned activities into mid-2021.
Program Highlights
Initiated Dosing of the Second Cohort in RGLS4326 Phase 1
for ADPKD: In February
2020, the Company initiated dosing of the second cohort of
the MAD clinical study of RGLS4326, a novel oligonucleotide
designed to inhibit miR-17 for the treatment of ADPKD. The Company
expects to complete this study in mid-2020 with topline results
available thereafter. The Company is also planning to initiate a
Phase 1b short-term dosing study in
patients with ADPKD in the second half of 2020 to evaluate RGLS4326
for safety, pharmacokinetics, and biomarkers of pharmacodynamic
activity. The re-initiation of the MAD study was allowed following
a satisfactory complete response provided to FDA in November 2019 to address the partial clinical
hold placed on the clinical program in July
2019. A partial clinical hold on studies of RGLS4326 dosed
for extended durations remains in effect until the second set of
requirements outlined by FDA have been satisfactorily addressed.
Information from the Phase 1 clinical studies, together with
information from additional nonclinical studies, will be used to
address the second set of requirements to support studies of
extended duration.
Financial Results
Cash Position: As of December
31, 2019, Regulus had $34.1
million in cash and cash equivalents.
Research and Development (R&D) Expenses:
R&D expenses were $2.1 million
and $12.3 million for the quarter and
year ended December 31, 2019,
respectively, compared to $5.3
million and $34.0 million for
the same periods in 2018. The decreases were largely driven by
decreases in external development expenses, primarily attributable
to the pause of the RGLS4326 MAD study in the third quarter of 2018
and transfer of the RG-012 program to Sanofi beginning in the
fourth quarter of 2018. Additionally, the decreases were driven by
reductions in personnel and internal expenses, primarily
attributable to a reduction in costs subsequent to our corporate
restructuring in the third quarter of 2018.
General and Administrative (G&A) Expenses:
G&A expenses were $2.4 million
and $11.3 million for the quarter and
year ended December 31, 2019,
respectively, compared to $2.7
million and $12.9 million for
the same periods in 2018. The decreases were mostly driven by a
reduction in costs subsequent to our corporate restructuring in the
third quarter of 2018.
Revenue: Revenue was less than $0.1 million and $6.8
million for the quarter and year ended December 31, 2019, respectively, compared to less
than $0.1 million and $0.1 million for the same periods in 2018. The
increase for the year ended December 31,
2019 was attributable to revenue recognition of the upfront
payments received under the 2018 Sanofi Amendment related to the
transfer of the RG-012 program to Sanofi.
Net Loss: Net loss was $4.9 million and $18.6 million for the quarter and year ended
December 31, 2019, respectively, compared to a net loss of
$8.6 million and $48.7 million for the same periods in 2018.
Basic and diluted net loss per share was $0.23 and $1.08 for
the quarter and year ended December 31, 2019, respectively,
compared to $0.98 and $5.59 for the same periods in 2018.
About ADPKD
ADPKD, caused by the mutations in the PKD1 or PKD2 genes, is
among the most common human monogenic disorders and a leading cause
of end-stage renal disease. The disease is characterized by the
development of multiple fluid filled cysts primarily in the
kidneys, and to a lesser extent in the liver and other organs.
Excessive kidney cyst cell proliferation, a central pathological
feature, ultimately leads to end-stage renal disease in
approximately 50% of ADPKD patients by age 60.
About RGLS4326
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17
and designed to preferentially target the kidney. Preclinical
studies with RGLS4326 have demonstrated direct regulation of PKD1
and PKD2 in human ADPKD cyst cells, a reduction in kidney cyst
formation, improved kidney weight/body weight ratio, decreased cyst
cell proliferation, and preserved kidney function in mouse models
of ADPKD. The RGLS4326 IND is currently on a Partial Clinical
Hold for treatment of extended duration by the U.S. Food and Drug
Administration.
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.
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 completion of
preclinical and clinical activities concerning the RGLS4326
program, the sufficiency of the data resulting from the ongoing or
planned preclinical studies required to recommence clinical studies
for extended duration dosing and the timing of preclinical and
clinical activities. 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, and
feedback from the FDA. These and other risks 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
December
31,
|
|
Year ended
December
31,
|
|
2019
|
|
2018
|
|
2019
|
|
2018
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
$
|
18
|
|
$
|
18
|
|
$
|
6,832
|
|
$
|
72
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
2,090
|
|
5,255
|
|
12,349
|
|
33,975
|
General and
administrative
|
2,363
|
|
2,745
|
|
11,317
|
|
12,860
|
Total operating
expenses
|
4,453
|
|
8,000
|
|
23,666
|
|
46,835
|
Loss from
operations
|
(4,435)
|
|
(7,982)
|
|
(16,834)
|
|
(46,763)
|
Other expense,
net
|
(458)
|
|
(520)
|
|
(1,757)
|
|
(1,884)
|
Loss before income
taxes
|
(4,893)
|
|
(8,502)
|
|
(18,591)
|
|
(48,647)
|
Income tax
expense
|
|
-
|
|
|
(61)
|
|
(1)
|
|
(62)
|
Net loss
|
$
|
(4,893)
|
|
$
|
(8,563)
|
|
$
|
(18,592)
|
|
$
|
(48,709)
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(0.23)
|
|
$
|
(0.98)
|
|
$
|
(1.08)
|
|
$
|
(5.59)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
20,950,602
|
|
|
8,780,779
|
|
|
17,260,176
|
|
|
8,718,563
|
|
December
31,
2019
|
|
December 31,
2018
|
|
|
Cash and cash
equivalents
|
$
|
34,121
|
|
$
|
13,935
|
Total
assets
|
42,081
|
|
27,927
|
Term loan, less debt
issuance costs
|
14,631
|
|
16,575
|
Stockholders' equity
(deficit)
|
|
20,015
|
|
|
(5,854)
|
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SOURCE Regulus Therapeutics Inc.