FDA Acceptance of Investigational New Drug
(IND) Application for RGLS8429 for Autosomal Dominant Polycystic
Kidney Disease (ADPKD)
On track to initiate Phase 1 study in second
quarter 2022
SAN DIEGO, May 12, 2022 /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, 2022 and provided a corporate update.
"The year is off to a great start as we continue to advance our
pipeline, including the recent acceptance of our IND application
for RGLS8429. We are thrilled to begin clinical development where
we will investigate the safety, tolerability, pharmacokinetics, and
preliminary efficacy of RGLS8429 for the treatment of ADPKD,"
stated Jay Hagan, CEO of Regulus.
"We also entered into an exciting research collaboration with
Brigham and Women's Hospital, which will further our understanding
into broader applications of miR-155 inhibitors and potentially
bring us closer to improving outcomes for Amyotrophic Lateral
Sclerosis (ALS) patients. We look forward to providing updates in
the coming quarters regarding progress on these fronts."
Program Updates
RGLS8429 for ADPKD: As announced yesterday, the U.S. Food
and Drug Administration (FDA) recently accepted the Company's IND
for RGLS8429 for the treatment of ADPKD. The Company plans
to initiate a Phase 1 single-ascending dose (SAD) study in healthy
volunteers to assess safety, tolerability and pharmacokinetics of
RGLS8429. Following the SAD study, the Company plans to initiate a
Phase 1b multiple ascending dose
(MAD) study in adult patients with ADPKD to assess safety,
tolerability and pharmacokinetics of RGLS8429, and to evaluate the
dose response of RGLS8429 treatment on ADPKD biomarkers including
polycystins, cystic kidney volume (htTKV), and overall kidney
function. Top-line data from the healthy volunteer study are
expected in the second half of 2022, and top-line biomarker data
for the first cohort of RGLS8429-treated patients with ADPKD are
expected in the first half of 2023.
Lademirsen (RG-012) for Alport
syndrome: In February 2022, the Company announced
completion of enrollment by Sanofi in the Phase 2 HERA clinical
study evaluating lademirsen for the treatment of adult patients
with Alport Syndrome under the Company's Collaboration and License
Agreement with Sanofi. Final data are expected in the first
half of 2023 and, if successful, could provide further validation
of the Company's platform technology designed to address genetic
kidney diseases and earn the Company a $25
million milestone.
Corporate Highlights
Collaboration Agreement with Brigham and Women's
Hospital: In March
2022, the Company announced a collaboration agreement with
the laboratories of Oleg Butovsky, Ph.D., and Howard L.
Weiner, M.D., at Brigham and Women's Hospital to
investigate the biologic effects of miR-155 inhibitors in both in
vitro and in vivo models of ALS.
Financial Results
Cash Position: As of March
31, 2022, Regulus had $53.9
million in cash and cash equivalents.
Research and Development (R&D)
Expenses: Research and development expenses were
$3.7 million for the three months
ended March 31, 2022, compared to
$3.3 million for the same period in
2021. 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
$2.9 million for the three months
ended March 31, 2022, compared to
$2.5 million for the same period in
2021. These amounts reflect personnel-related and ongoing general
business operating costs.
Net Loss: Net loss was $6.7
million, or $0.05 per share
(basic and diluted), for the three months ended March 31, 2022, compared to $6.0 million, or $0.08 per share (basic and diluted), for the same
period in 2021.
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 first
quarter 2022 financial results and corporate update. To
access the call, please dial (866) 652-5200 (domestic) or
(412) 317-6060 (international). To access the telephone
replay of the call, dial (877) 344-7529 (domestic) or (412)
317-0088 (international), passcode ID 6812601. The
webcast and telephone replay will be archived on the Company's
website at www.regulusrx.com following the call.
About ADPKD
Autosomal Dominant Polycystic Kidney Disease (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. Approximately
140,000 individuals are diagnosed with the disease in the United States alone, with an estimated
global prevalence of 4 to 7 million.
About RGLS8429
RGLS8429 is a novel, next 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 and has demonstrated a superior
pharmacologic profile compared to Regulus' first generation
compound in preclinical studies. The U.S. Food and Drug
Administration (FDA) accepted the Company's IND for RGLS8429 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, the expected timing for initiating a Phase 1 clinical
study, the expected timing for reporting topline data, and the
timing and future occurrence of data concerning the Company's
preclinical programs. 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
March
31,
|
|
2022
|
|
2021
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
Research
and development
|
3,679
|
|
3,320
|
General
and administrative
|
2,890
|
|
2,478
|
Total operating
expenses
|
6,569
|
|
5,798
|
|
|
|
|
Loss from
operations
|
(6,569)
|
|
(5,798)
|
Other expense,
net
|
(149)
|
|
(215)
|
|
|
|
|
Loss before income
taxes
|
(6,718)
|
|
(6,013)
|
Income tax
expense
|
|
(1)
|
|
|
-
|
Net loss
|
$
|
(6,719)
|
|
$
|
(6,013)
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(0.05)
|
|
$
|
(0.08)
|
|
|
|
|
|
|
Weighted average shares
used to compute basic and diluted net loss per share
|
|
145,973,989
|
|
|
71,290,918
|
|
|
|
|
|
|
|
|
|
March 31,
2022
|
|
December 31,
2021
|
|
|
|
Cash and cash equivalents
|
$
|
53,902
|
|
$
|
60,383
|
Total assets
|
60,985
|
|
68,454
|
Term loan, less debt issuance costs
|
4,674
|
|
4,673
|
Stockholders' equity
|
|
49,241
|
|
|
54,958
|
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SOURCE Regulus Therapeutics Inc.