SAN DIEGO, Nov. 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 third quarter ended September 30, 2021 and provided a corporate
update.
"We are pleased with the progress we have made in advancing our
next generation candidate RGLS8429 as a potential treatment for
ADPKD, as the compound has been shown to have the favorable
properties of RGLS4326 without the limitations of the
first-generation compound," commented Jay
Hagan, CEO of Regulus Therapeutics. "RGLS8429 has
demonstrated comparable potency, as well as similar pharmacokinetic
and pharmacodynamic profiles, without the off-target CNS effects
seen in chronic preclinical toxicology studies with RGLS4326 at the
top doses tested. We look forward to our pre-IND meeting in
December with the FDA to help finalize our IND submission and reach
another important milestone in our mission to improve the lives of
ADPKD patients."
Program Updates
RGLS8429 for ADPKD: In October
2021, the Company discontinued development of its
first-generation compound, RGLS4326, to allocate resources and
efforts towards the development of its more promising
next-generation compound, RGLS8429. The Company believes RGLS8429
has demonstrated a superior pharmacological profile, with the
absence of the off-target central nervous system (CNS) effects that
were seen with RGLS4326 at the top doses tested in chronic
preclinical toxicology studies. RGLS8429 has also shown equal
potency to RGLS4326 for its molecular target (miR-17) in both
in-vitro and in-vivo efficacy studies. The Company expects to have
a pre-IND meeting with the U.S. Food and Drug Administration (FDA)
for RGLS8429 before year-end and is on track for an IND submission
and initiation of clinical development in the second quarter of
2022, subject to FDA clearance of the IND.
The Company's Phase 1 plans include a single dose escalation
study in healthy volunteers to enable a multi-dose escalation study
in ADPKD patients around the dose levels where robust clinical
biomarker effects were demonstrated with RGLS4326. The Company
anticipates reporting top-line biomarker data in the first cohort
of RGLS8429-treated patients in early 2023.
RGLS4326 for ADPKD: On November 4 and November
9, data from the first cohort of patients in the Phase
1b clinical trial of RGLS4326, the
Company's first-generation compound, for the treatment of ADPKD,
was presented at the American Society of Nephrology (ASN) Kidney
Week, and at the Biomarkers for Rare Diseases Summit. In the first
cohort, nine patients were enrolled and received 1 mg/kg of
RGLS4326 subcutaneously every other week for four doses. The mean
increase in polycystins 1 and 2 at the end of study compared to
baseline levels for all nine patients in the first cohort were 58%
(p=.0004) and 38% (p=.026), respectively. These data demonstrate
clinical evidence that treatment with RGLS4326 increased PC1 and
PC2, most likely through inhibition of miR-17 in the kidney of
patients with ADPKD. These results also imply that overexpressed
miR-17 in ADPKD patients represses Pkd1 and Pkd2 expression,
further validating miR-17 as a therapeutic target for ADPKD
treatment. The details for each presentation are below:
ASN Kidney Week ePoster:
Poster Title: RGLS4326 Increases Urinary PC1 and PC2 Levels in
Individuals with Autosomal Dominant Polycystic Kidney Disease
(ADPKD)
Poster Date and Time: Thursday, November 4,
2021, 10:00 AM PDT
Poster Number: PO1244
Biomarkers for Rare Diseases Summit Presentation:
Presentation Title: Results from the First Cohort of Phase1b
Clinical Trial of RGLS4326 for the Treatment of Patients with
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Presenter: Edmund Lee, PhD,
Executive Director, Biology, Regulus Therapeutics
Presentation Date and Time: Tuesday,
November 9, 2021, 9:30 AM
PDT
A copy of each presentation is available at
www.regulusrx.com/publications/
Financial Results
Cash Position: As of September 30, 2021, Regulus had $35.8 million in cash and cash equivalents.
Research and Development (R&D)
Expenses: Research and development expenses were
$5.9 million and $13.4 million for the three and nine months ended
September 30, 2021, respectively,
compared to $4.0 million and
$11.4 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 $7.5 million for the three and nine months
ended September 30, 2021,
respectively, compared to $2.1
million and $6.7 million for the
same periods in 2020, respectively. These amounts reflect
personnel-related and ongoing general business operating costs.
Net Loss: Net loss was $8.6
million, or $0.10 per share
(basic and diluted), and $20.7
million, or $0.26 per share (basic and diluted),
for the three and nine months ended September 30, 2021, compared to $1.5 million, or $0.04 per share (basic
and diluted), and $14.4 million, or $0.47 per share
(basic and diluted), for the same periods in 2020.
Conference Call and Webcast Information:
The Company
will host a conference call and live audio webcast today
at 5:00 p.m. Eastern Daylight Time to discuss its third
quarter 2021 financial results and corporate update. To
access the call, please dial (877) 257-8599 (domestic) or (970)
315-0459 (international) and refer to conference ID 2108429.
To access the telephone replay of the call, dial (855) 859-2056
(domestic) or (404) 537-3406 (international), passcode ID
2108429. The webcast and telephone replay will be archived on
the Company's website at www.regulusrx.com following the
call.
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 RGLS8429
RGLS8429 is a novel, second generation oligonucleotide designed
to inhibit miR-17 and to preferentially target the kidney.
Administration of RGLS8429 has shown robust data in preclinical
models, where clear improvements in kidney function, size, and
other measures of disease severity, as well as a superior
pharmacologic profile have been demonstrated. Regulus has
nominated RGLS8429 as a development candidate for the treatment of
ADPKD.
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 presentation 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 Company's RGLS8429
program, including the potential sufficiency of the preclinical
data required to support clinical studies, the expected timing for
submitting an IND and initiating a Phase 1 clinical study, 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, and the risk additional
toxicology data may be negative. 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
September
30,
|
|
Nine months
ended
September
30,
|
|
|
2021
|
|
2020
|
|
2021
|
|
2020
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic alliances
|
|
$
|
-
|
|
$
|
5,000
|
|
$
|
-
|
|
$
|
5,006
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
5,915
|
|
4,036
|
|
13,385
|
|
11,396
|
General and
administrative
|
|
2,504
|
|
2,059
|
|
7,471
|
|
6,736
|
Total operating
expenses
|
|
8,419
|
|
6,095
|
|
20,856
|
|
18,132
|
Loss from
operations
|
|
(8,419)
|
|
(1,095)
|
|
(20,856)
|
|
(13,126)
|
Other expense,
net
|
|
(209)
|
|
(428)
|
|
182
|
|
(1,289)
|
Loss before income
taxes
|
|
(8,628)
|
|
(1,523)
|
|
(20,674)
|
|
(14,415)
|
Income tax (expense)
benefit
|
|
|
-
|
|
|
(1)
|
|
(1)
|
|
7
|
Net loss
|
|
$
|
(8,628)
|
|
$
|
(1,524)
|
|
$
|
(20,675)
|
|
$
|
(14,408)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.10)
|
|
$
|
(0.04)
|
|
$
|
(0.26)
|
|
$
|
(0.47)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
|
87,042,437
|
|
|
38,137,281
|
|
|
78,560,760
|
|
|
30,695,137
|
|
|
September 30,
2021
|
|
December 31,
2020
|
|
|
(Unaudited)
|
|
|
|
Cash and cash
equivalents
|
|
$
|
35,848
|
|
$
|
31,087
|
Total
assets
|
|
43,042
|
|
37,604
|
Term loan, less debt
issuance costs
|
|
4,668
|
|
4,652
|
Stockholders'
equity
|
|
29,012
|
|
26,026
|
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SOURCE Regulus Therapeutics Inc.