SAN DIEGO, Aug. 10, 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 second quarter ended June 30, 2021 and provided a corporate
update.
"The team at Regulus accomplished a lot over the last several
months, including the achievement of several major milestones in
our ADPKD program, the completion of meeting preparations with FDA
and extending our cash runway into Q4 of next year," commented
Jay Hagan, CEO of Regulus. "In
addition, we are pleased to have completed enrollment of the second
cohort of our Phase 1b trial of
RGLS4326 for ADPKD, for which we expect to report topline data in
the next several months. Additionally, we look forward to obtaining
FDA feedback on our approach for addressing the remaining partial
clinical hold requirements through our meeting request."
Program Updates
RGLS4326 for ADPKD: In June
2021, the Company presented additional data from the first
cohort of patients in its Phase 1b
clinical trial of RGLS4326 for the treatment of ADPKD, as well as
new preclinical data from relevant animal models of the disease, at
PKD Connect 2021. The additional data presented at PKD Connect
demonstrated clinical proof of mechanism by showing statistically
significant increases in polycystin levels in ADPKD patients by
targeting miR-17 in the kidneys. Levels of PC1 and PC2 have
previously been shown to be inversely correlated with disease
severity. RGLS4326 was well-tolerated with no serious adverse
events.
The Phase 1b study is an
adaptive design, open-label, multiple dose study in up to three
cohorts of patients with ADPKD evaluating the safety,
pharmacokinetics, and effects on pharmacodynamic biomarkers of
multiple doses of RGLS4326. In the first cohort of the study, nine
patients were enrolled and received four doses of 1mg/kg of
RGLS4326 administered every other week.
The Company also presented new data from relevant preclinical
models showing treatment with RGLS4326 resulted in increased gene
and polycystin levels in vitro. Improvements in
key disease markers including serum creatinine and BUN were
demonstrated in the Pkd1(F/RC) mouse model that harbors
a mutation in the Pkd1 gene equivalent to human ADPKD.
Mutation to the Pkd1 gene is reported for 85% of diagnosed patients
with ADPKD.
In late July 2021, the Company
completed enrollment of the second cohort of patients in its Phase
1b clinical study of RGLS4326. The
patients in the second cohort are being administered 0.3mg/kg of
RGLS4326 every other week for four doses. The dose of RGLS4326 for
the third and final cohort will be chosen based on the results of
this second cohort and enrollment is expected to commence
thereafter. The Company expects to report topline data from the
second cohort in the fourth quarter.
The Company requested a Type A meeting with FDA to discuss the
data supporting its approach to addressing the remaining partial
clinical hold requirements. FDA typically responds to a sponsor's
request with the meeting being scheduled within 30 days from the
receipt of the meeting request.
Corporate Highlights
Raised $15.4 Million in Gross
Proceeds Through its ATM Facility: A total of 12,007,546 shares
were sold and settled for gross proceeds of $15.4 million at a weighted average price
per share of $1.28 under the ATM
facility during the three months ended June
30, 2021. The Company expects its existing cash will provide
cash resources to fund planned activities into Q4 2022.
Financial Results
Cash Position: As of June 30,
2021, Regulus had $41.4
million in cash and cash equivalents.
Research and Development (R&D)
Expenses: Research and development expenses were
$4.2 million and $7.5 million for the three and six months ended
June 30, 2021, respectively, compared
to $4.2 million and $7.4 million for the same periods in 2020,
respectively. 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 and $5.0 million for the three and six months
ended June 30, 2021, respectively,
compared to $2.3 million and
$4.7 million for the same periods in
2020, respectively. 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), and $12.0
million, or $0.16 per share (basic and diluted),
for the three and six months ended June 30,
2021, compared to $6.9
million, or $0.23 per share (basic and diluted),
and $12.9 million, or $0.48 per share (basic and
diluted), for the same periods 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 the third quarter 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 San Diego,
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 regarding the Company's expected cash
runway and statements associated with the Company's RGLS4326
program, including the potential 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, the expected timing for reporting topline data, and
the timing and future occurrence 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, the risk that feedback from
the FDA may be negative or may not be provided when we expect,
risks associated with our need for additional capital. 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, including under the
"Risk Factors" heading of Regulus most recently quarterly report on
Form 10-Q. 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
June
30,
|
|
Six months
ended
June
30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
-
|
|
$
|
-
|
|
$
|
-
|
|
$
|
6
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
4,150
|
|
4,242
|
|
7,470
|
|
7,360
|
General and
administrative
|
|
2,488
|
|
2,254
|
|
4,967
|
|
4,676
|
Total operating
expenses
|
|
6,638
|
|
6,496
|
|
12,437
|
|
12,036
|
Loss from
operations
|
|
(6,638)
|
|
(6,496)
|
|
(12,437)
|
|
(12,030)
|
Other income
(expense), net
|
|
605
|
|
(451)
|
|
391
|
|
(862)
|
Loss before income
taxes
|
|
(6,033)
|
|
(6,947)
|
|
(12,046)
|
|
(12,892)
|
Income tax (expense)
benefit
|
|
|
(1)
|
|
|
-
|
|
(1)
|
|
8
|
Net loss
|
|
$
|
(6,034)
|
|
$
|
(6,947)
|
|
$
|
(12,047)
|
|
$
|
(12,884)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.08)
|
|
$
|
(0.23)
|
|
$
|
(0.16)
|
|
$
|
(0.48)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
|
77,175,831
|
|
|
29,801,974
|
|
|
74,249,631
|
|
|
26,933,173
|
|
|
June 30,
2021
|
|
December 31,
2020
|
|
|
|
Cash and cash
equivalents
|
|
$
|
41,440
|
|
$
|
31,087
|
Total
assets
|
|
49,516
|
|
37,604
|
Term loan, less debt
issuance costs
|
|
4,662
|
|
4,652
|
Stockholders'
equity
|
|
|
36,828
|
|
|
26,026
|
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SOURCE Regulus Therapeutics Inc.