SAN DIEGO, May 13, 2021 /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, 2021 and provided a corporate
update.
"We are excited about the data we recently announced from the
first cohort of our phase 1b clinical
trial of RGLS4326 for the treatment of patients with ADPKD where we
saw mean increases of greater than 50% and 20% in Polycystin 1
(PC1) and Polycystin 2 (PC2), respectively. PC1 and PC2 are
the products of the PKD1 and PKD2 genes, and are depressed in
patients with this disease. The trends for both suggest that with
continued therapy, higher levels of these proteins may be
attainable and less frequent dosing required. Additionally,
RGLS4326 was well tolerated by all nine patients with no serious
adverse events reported", stated Jay
Hagan, CEO of Regulus. "Data from the first cohort provides
the safety and pharmacokinetic data needed to complete the modeled
safety margins. We plan to submit these data to FDA this
summer in the context of our discussions regarding the remaining
partial clinical hold requirements."
Program Updates
RGLS4326 for ADPKD: In February
2021, the Company completed enrollment in the first cohort
of a Phase 1b clinical study for
RGLS4326 in patients with ADPKD (The "Phase 1b"). The Phase 1b
is an adaptive design, open-label, multiple dose study in up to
three cohorts of patients with ADPKD. The study is designed
to evaluate the safety, pharmacokinetics, and changes in levels of
PC1 and PC2 in patients with ADPKD administered RGLS4326 every
other week for a total of four doses. The dose level for the
first cohort is 1 mg/kg of RGLS4326 and the dose level for the
second cohort is 0.3 mg/kg. The third and final cohort will
be dosed at a level to be determined based on the results of the
first two cohorts. In May 2021,
the Company announced top-line results from the first cohort of
patients with ADPKD in its ongoing Phase 1b clinical trial of RGLS4326.
In the first cohort, nine patients were enrolled and received 1
mg/kg of RGLS4326 subcutaneously every other week for four
doses. Safety, pharmacokinetics, and certain disease related
biomarkers were evaluated through the course of the study.
The biomarkers included: PC1 and PC2, kidney injury marker 1
(KIM-1), neutrophil gelatinase-associated lipocalin (NGAL), as well
as urea and creatinine and were chosen to evaluate changes in
disease related measures.
Measured levels of PC1 and PC2 increased greater than 50% and
20%, respectively by the end of study compared to baseline
levels. Regulus believes these initial data demonstrate that
RGLS4326 engages the target miR-17 leading to increased expression
of the PKD1 and PKD2 genes and the resultant increases in measured
polycystin levels. Measured levels of PC1 and PC2 have been
shown to inversely correlate with disease severity and are believed
to be directly linked to the underlying genetic drivers of the
disease. The overall trend in polycystin showed increasing levels
of both PC1 and PC2 over time with a sustained effect suggesting
less frequent dosing could be utilized. Importantly, at the
time of the analysis, patient mutational status was not known and
may further contribute to understanding differences in response
rates. Approximately 85% of patients with ADPKD are reported to
have a mutation in the PKD1 gene, while the remaining 15% have a
mutation in the PKD2 gene. Additionally, the PKD1 gene has
one predicted binding site for miR-17 while the PKD2 gene has two
predicted binding sites for miR-17, potentially contributing to
different response rates between the biomarkers.
RGLS4326 was well tolerated by all nine patients with no serious
adverse events reported. All reported adverse events were
mild and generally transient in nature.
Overall, the pharmacokinetic profile of RGLS4326 in patients
with ADPKD was similar to that observed in a prior healthy
volunteer study. Concentrations of RGLS4326 in plasma were
greater in patients (Cmax ~ 3 ug/mL) relative to healthy volunteers
(Cmax ~ 2 ug/mL), suggesting a lower dose in patients could achieve
the desired exposure in the kidney, the target organ of
interest.
Data from this first cohort is planned to be submitted for
presentation at PKD Connect in June
2021 and at Kidney Week, the American Society of Nephrology
annual meeting being held in November
2021.
Corporate Highlights
Lease Agreement for New Corporate Headquarters Executed and
Relocation Completed: In February 2021, we entered into a lease agreement
(the "New Lease") for 13,438 square feet located at 4224 Campus
Point Court, Suite 210, San Diego,
California, 92121, which is to be used as our new principal
offices and laboratory for research and development. The move
into the space leased under the New Lease was completed in
April 2021. Concurrently with the New
Lease, we assigned the lease to the space that had served as our
previous corporate headquarters, and have no remaining obligations
associated with the previous corporate headquarters lease after
April 2021.
Financial Results
Cash Position: As of March
31, 2021, Regulus had $31.6
million in cash and cash equivalents.
Research and Development (R&D)
Expenses: Research and development expenses were
$3.3 million for the three months
ended March 31, 2021, compared to
$3.1 million for the same period in
2020. These amounts reflect internal and external costs associated
with advancing our clinical and preclinical pipeline.
General and Administrative (G&A)
Expenses: General and administrative expenses were
$2.5 million for the three months
ended March 31, 2021, compared to
$2.4 million for the same period in
2020. These amounts reflect personnel-related and ongoing general
business operating costs.
Net Loss: Net loss was $6.0
million, or $0.08 per share
(basic and diluted), for the three months ended March 31, 2021, compared to $5.9 million, or $0.25 per share (basic and diluted), for the same
period in 2020.
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 preferentially target the kidney. Preclinical studies with
RGLS4326 have demonstrated direct regulation of Pkd1 and
Pkd2, reduction of cyst growth in human in vitro
ADPKD models, and attenuation of cyst proliferation and improvement
of kidney function in mouse models of ADPKD. The RGLS4326 IND
is currently on a partial clinical hold for treatment of extended
duration by FDA until the second set of requirements outlined
by the agency have been satisfactorily addressed. The Company will
use information from the Phase 1 clinical studies, including the
first cohort of the Phase 1b together
with information from the recently completed additional nonclinical
studies generated in 2020, in its plan to address the second set of
requirements outlined in the Partial Clinical Hold letter to
support studies of extended duration. Regulus plans to discuss its
approach to addressing the remaining Partial Clinical Hold
requirements with FDA in mid-2021. RGLS4326 received orphan
drug designation from FDA in July
2020.
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 clinical
activities concerning the RGLS4326 program, including the
preliminary biomarker, pharmacokinetic and safety data resulting
from the first cohort of patients from the ongoing clinical study,
the sufficiency of the data required to recommence clinical studies
for extended duration dosing, the timing of the Company's
interactions with FDA regarding the clinical hold and the timing
and of other 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,
|
|
|
|
2021
|
|
2020
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
-
|
|
$
|
6
|
|
Operating
expenses:
|
|
|
|
|
|
Research and
development
|
|
3,320
|
|
3,119
|
|
General and
administrative
|
|
2,478
|
|
2,422
|
|
Total operating
expenses
|
|
5,798
|
|
5,541
|
|
Loss from
operations
|
|
(5,798)
|
|
(5,535)
|
|
Other expense,
net
|
|
(215)
|
|
(410)
|
|
Loss before income
taxes
|
|
(6,013)
|
|
(5,945)
|
|
Income tax
benefit
|
|
|
-
|
|
|
8
|
|
Net loss
|
|
$
|
(6,013)
|
|
$
|
(5,937)
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.08)
|
|
$
|
(0.25)
|
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
|
71,290,918
|
|
|
24,064,373
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31,
2021
|
|
December 31,
2020
|
|
|
|
Cash
and cash equivalents
|
|
$
|
31,597
|
|
$
|
31,087
|
Total
assets
|
|
39,841
|
|
37,604
|
Term
loan, less debt issuance costs
|
|
4,657
|
|
4,652
|
Stockholders'
equity
|
|
|
27,111
|
|
|
26,026
|
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SOURCE Regulus Therapeutics Inc.