LA JOLLA, Calif., May 14, 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 first quarter ended March 31, 2020 and provided a corporate
update.
"We have completed the second cohort and have initiated the
dosing for the third and final cohort in the Phase 1 multiple
ascending dose ("MAD") clinical study for RGLS4326"
said Jay Hagan, CEO of Regulus. "Additionally, preparations
for the Phase 1b study in patients
with autosomal dominant polycystic kidney disease ("ADPKD") are
well underway with plans to initiate in the second half of
2020."
Program Highlights
Initiated Dosing of the Third and Final Cohort in RGLS4326
Phase 1 for ADPKD: In April 2020, the Company initiated dosing of the
third and final 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 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.
Financial Results
Cash Position: As of March
31, 2020, Regulus had $28.1
million in cash and cash equivalents.
Research and Development (R&D) Expenses: R&D
expenses were $3.1 million for the
three months ended March 31, 2020,
compared to $6.0 million for the same
period in 2019. The aggregate decrease was driven by a $1.5 million reduction in personnel and internal
expenses and a $1.0 million reduction
in external development expenses, both of which were primarily
attributable to a reduction in costs associated with the partial
clinical hold of the RGLS4326 MAD study. In December 2019, the U.S. Food and Drug
Administration ("FDA") lifted the partial clinical hold on the MAD
study and it was recommenced in February
2020.
General and Administrative (G&A) Expenses:
G&A expenses were $2.4 million
for the three months ended March 31,
2020 compared to $3.5 million
for the same period in 2019. These amounts reflect
personnel-related and ongoing general business operating costs. The
decrease is primarily attributable to continued cost reduction
efforts subsequent to our corporate restructuring in the third
quarter of 2018.
Revenue: Revenue was less than $0.1 million for the three months ended
March 31, 2020. Revenue was
$6.8 million for the three months
ended March 31, 2019, 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 $5.9
million, or $0.25 per share
(basic and diluted), for the three months ended March 31, 2020, compared to $3.3 million, or $0.31 per share (basic and diluted), for the same
period in 2019.
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 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.
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. In addition, while Regulus expects the
COVID-19 pandemic to adversely affect its business operations and
financial results, the extent of the impact on Regulus' ability to
achieve its preclinical and clinical development objectives and the
value of and market for its common stock, will depend on future
developments that are highly uncertain and cannot be predicted with
confidence at this time, such as the ultimate duration of the
pandemic, travel restrictions, quarantines, social distancing and
business closure requirements in the U.S. and in other countries,
and the effectiveness of actions taken globally to contain and
treat the disease. 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
March 31,
|
|
2020
|
|
2019
|
|
|
Revenues:
|
|
|
|
|
|
Revenue under
strategic
alliances
|
$
|
6
|
|
$
|
6,778
|
Operating
expenses:
|
|
|
|
Research and
development
|
3,119
|
|
5,983
|
General and
administrative
|
2,422
|
|
3,533
|
Total operating
expenses
|
5,541
|
|
9,516
|
Loss from
operations
|
(5,535)
|
|
(2,738)
|
Other expense,
net
|
(410)
|
|
(522)
|
Loss before income
taxes
|
(5,945)
|
|
(3,260)
|
Income tax
benefit
|
|
8
|
|
|
-
|
Net loss
|
$
|
(5,937)
|
|
$
|
(3,260)
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(0.25)
|
|
$
|
(0.31)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
24,064,373
|
|
|
10,379,830
|
|
|
|
March 31,
2020
|
|
December 31,
2019
|
|
|
Cash and cash
equivalents
|
$
|
28,052
|
|
$
|
34,121
|
Total
assets
|
35,006
|
|
42,081
|
Term loan, less debt
issuance costs
|
14,636
|
|
14,631
|
Stockholders' equity
(deficit)
|
|
14,902
|
|
|
20,015
|
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SOURCE Regulus Therapeutics Inc.