LA JOLLA, Calif., Nov. 12, 2019 /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 third quarter ended September 30, 2019 and provided a summary of
recent events.
"We recently received the final reports from the chronic
toxicity studies in mice and non-human primates and are preparing
our submission of our complete response to Food and Drug
Administration ("FDA") in our efforts to address the requirements
to reinitiate the Multiple Ascending Dose ("MAD") clinical study
for RGLS4326," said Jay Hagan, CEO
of Regulus. "We look forward to FDA's feedback and continued
productive dialogue."
Third Quarter 2019 Corporate Highlights and Recent
Updates
- Management Transition to New Chief Financial Officer: In
July 2019, the Company appointed
Cris Calsada as its new Chief
Financial Officer, effective August 30,
2019. Ms. Calsada's appointment follows the resignation of
the Company's previous Chief Financial Officer, Dan Chevallard, in July
2019.
- New Lease Agreement Significantly Reduces Contractual Lease
Obligations: In June 2019, the
Company entered into an amendment of its lease (the "Lease
Amendment") of 24,562 square feet located at 10628 Science Center
Drive Suite 100, San Diego,
California 92121. Under the terms of the Lease Amendment,
the expiration of the lease was accelerated from June 30, 2023 to June 30,
2019, and the lease terminated on July 1, 2019. Concurrently with the Lease
Amendment, the Company entered into a new lease agreement for 8,727
square feet located at 10628 Science Center Drive, Suite 225,
San Diego, California, 92121,
which it uses as its new principal offices and laboratory for
research and development. This relocation reduced the Company's
facility size by approximately 65% and reduced its future
contractual lease obligations by approximately 78%.
Program Updates
- RGLS4326 for Autosomal Dominant Polycystic Kidney Disease
("ADPKD"): RGLS4326, a novel oligonucleotide designed to
inhibit miR-17, is in Phase 1 clinical development for the
treatment of ADPKD and currently on a partial clinical hold. In
January 2019, the Company submitted a
comprehensive data package for RGLS4326 to FDA that included the
results from the planned 13-week interim analysis of the repeat
mouse chronic toxicity study, as well as results from additional
investigations, analytical testing, additional data from the
previously terminated mouse chronic toxicity study, data from the
completed Phase I single ascending dose ("SAD") study and data from
the first cohort of the Phase I MAD study, to support its plan to
resume the Phase I MAD study. After review of the requested
submission, FDA notified the Company in July
2019 of additional nonclinical data requirements and placed
the IND on a partial clinical hold, formalizing the specific
requirements to initiate the MAD study and further proceed into
chronic dosing in ADPKD patients. The additional data requirements
have been outlined in two parts. In order to resume the MAD study,
FDA has requested the final reports from the chronic toxicity
studies in both mice and non-human primates and satisfactory
related analyses to ensure subjects can be safely dosed. Additional
information and analyses from new nonclinical studies, planned to
be generated over the next several quarters, are required for
chronic dosing and will be submitted at a later date. The Company
recently received the final reports from the chronic toxicity
studies in mice and non-human primates and is preparing to submit a
complete response to the partial clinical hold in order to be able
to resume the MAD study, pending agreement with FDA. Regulus
anticipates a response from FDA before the end of the year. Regulus
is allowed to proceed with additional SAD clinical studies as part
of the process to gather additional supporting information to guide
the future development of the program.
In September 2019, the Company
announced that Nature Communications published an article entitled,
"Discovery and preclinical evaluation of anti-miR-17 for the
treatment of polycystic kidney disease." The article
highlights preclinical research that identifies RGLS4326, a
first-in-class anti-miR-17 oligonucleotide, as a potential
disease-modifying treatment for ADPKD. In addition, a poster
highlighting this work was presented at the Oligonucleotide
Therapeutics Society (OTS) Annual Meeting held October 13-16, 2019 in Munich, Germany.
Third Quarter 2019 Financial Results
Cash Position: As of September 30, 2019, Regulus had $14.6 million in cash and cash equivalents.
Revenue: Revenue was less than $0.1 million and $6.8
million for the three and nine months ended September 30, 2019, respectively, compared to
less than $0.1 million and
$0.1 million for the three and nine
months ended September 30, 2018. The
increase for the nine months ended September
30, 2019 was attributable to revenue recognition of the
upfront payments received under the 2018 Sanofi Amendment related
to the transfer of RG-012.
Research and Development (R&D) Expenses:
R&D expenses were $2.4 million
and $10.3 million for the three and
nine months ended September 30, 2019,
compared to $6.9 million and
$28.7 million for the same periods in
2018. The decreases were driven by decreases in external
development expenses, primarily attributable to the voluntary pause
of the RGLS4326 Phase 1 MAD clinical study in the third quarter of
2018 and commencement of the transfer of the RG-012 program to
Sanofi 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 $2.6 million
and $9.0 million for the three and
nine months ended September 30, 2019,
compared to $3.0 million and
$10.1 million for the same periods in
2018. These amounts reflect personnel-related and ongoing general
business operating costs. The decreases were driven by a reduction
in costs subsequent to our corporate restructuring in the third
quarter of 2018.
Net Loss: Net loss was $5.4
million, or $0.26 per share
(basic and diluted), and $13.7
million, or $0.86 per share
(basic and diluted), for the three and nine months ended
September 30, 2019, respectively,
compared to $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 same
periods in 2018. Historical and current period net loss per share
values have been retroactively adjusted to reflect our October 2018 reverse stock split.
About Autosomal Dominant Polycystic Kidney Disease
(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
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,
California. 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, RGLS5579 or its other preclinical
programs), its estimated cash runway, the projected timeline of
clinical development activities, the sufficiency of data supporting
its complete response and the timing of regulatory submissions
regarding RGLS4326 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,
|
|
|
2019
|
|
2018
|
|
2019
|
|
2018
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
18
|
|
$
|
18
|
|
$
|
6,814
|
|
$
|
54
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
2,440
|
|
6,879
|
|
10,259
|
|
28,720
|
General and
administrative
|
|
2,571
|
|
2,993
|
|
8,954
|
|
10,115
|
Total operating
expenses
|
|
5,011
|
|
9,872
|
|
19,213
|
|
38,835
|
Loss from
operations
|
|
(4,993)
|
|
(9,854)
|
|
(12,399)
|
|
(38,781)
|
Other expense,
net
|
|
(430)
|
|
(419)
|
|
(1,299)
|
|
(1,365)
|
Loss before income
taxes
|
|
(5,423)
|
|
(10,273)
|
|
(13,698)
|
|
(40,146)
|
Income tax
expense
|
|
|
-
|
|
|
-
|
|
(1)
|
|
-
|
Net loss
|
|
$
|
(5,423)
|
|
$
|
(10,273)
|
|
$
|
(13,699)
|
|
$
|
(40,146)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.26)
|
|
$
|
(1.18)
|
|
$
|
(0.86)
|
|
$
|
(4.62)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
|
20,849,083
|
|
|
8,703,626
|
|
|
16,016,515
|
|
|
8,688,831
|
|
|
September 30,
2019
|
|
December 31,
2018
|
|
|
(Unaudited)
|
Cash and cash
equivalents
|
|
$
|
14,646
|
|
$
|
13,935
|
Total
assets
|
|
21,410
|
|
27,927
|
Term loan, less debt
issuance costs
|
|
14,626
|
|
16,575
|
Stockholders' equity
(deficit)
|
|
|
(143)
|
|
|
(5,854)
|
View original content to download
multimedia:http://www.prnewswire.com/news-releases/regulus-therapeutics-reports-third-quarter-2019-financial-results-and-recent-updates-300956684.html
SOURCE Regulus Therapeutics Inc.