IND accepted and first subject dosed in the
Phase 1 Single-Ascending Dose (SAD) study of RGLS8429 for
the treatment of Autosomal Dominant Polycystic Kidney Disease
(ADPKD)
RGLS8429 granted Orphan Drug Designation (ODD)
from the U.S. Food and Drug Administration (FDA)
Scientific leadership strengthened with the
appointment of Amin Kamel, Ph.D., as
Vice President, Drug Metabolism and Pharmacokinetics (DMPK)
SAN
DIEGO, Aug. 11, 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 second quarter ended June 30, 2022 and provided a corporate
update.
"This has been an exciting quarter, which culminated in the
achievement of multiple milestones. We continue to make steady
progress with RGLS8429, evidenced by the dosing of the first
subject in our Phase 1 SAD study," commented Jay Hagan, CEO of Regulus. "Moreover, we are
pleased to have Dr. Amin Kamel join
the Regulus team at this pivotal moment. We are proud of the
work our team is doing to advance our novel therapeutic candidate
for ADPKD patients, and we look forward to providing additional
updates on progress in the coming quarters."
Program Updates
RGLS8429 for ADPKD: In early June
2022, the Company announced the dosing of the first subject
in its Phase 1 SAD study of RGLS8429 for the treatment of ADPKD.
The study will assess the safety, tolerability, and
pharmacokinetics of RGLS8429 in healthy volunteers. The Company
subsequently plans to initiate a Phase 1b multiple ascending dose (MAD) study to assess
safety, tolerability, and pharmacokinetics of RGLS8429 in adult
patients with ADPKD, and evaluate the efficacy of RGLS8429
treatment across three different dose levels measuring changes in
polycystins, cystic kidney volume (htTKV), and overall kidney
function.
In late June, the Company announced that FDA had granted ODD to
RGLS8429 for the treatment of ADPKD. The FDA's Office of Orphan
Products Development grants orphan designation status to drugs and
biologics that are intended for the safe and effective treatment,
diagnosis or prevention of rare diseases, or conditions that affect
fewer than 200,000 people in the U.S. Orphan designation status is
intended to facilitate drug development for rare diseases and may
provide several benefits to drug developers, including financial
incentives, to support clinical development and the potential for
up to seven years of market exclusivity in the U.S. upon regulatory
approval.
Corporate Highlights
Expanded Team: In June
2022, the Company announced the appointment of Amin Kamel, Ph.D., as Vice President, DMPK. Most
recently, Dr. Kamel was the Scientific Director, DMPK at Takeda,
where he also served as a scientific advisor. Prior to Takeda, Dr.
Kamel held positions at Biogen, Novartis, and Pfizer.
Financial Results
Cash, Cash Equivalents and Marketable Securities: As of
June 30, 2022, Regulus had
$47.5 million in cash, cash
equivalents and marketable securities.
Research and Development (R&D)
Expenses: Research and development expenses were
$4.7 million and $8.4 million for the three and six months ended
June 30, 2022, respectively, compared
to $4.2 million and $7.5 million for the same periods in 2021,
respectively. These amounts reflect internal and external costs
associated with advancing our pipeline.
General and Administrative (G&A)
Expenses: General and administrative expenses were
$2.5 million and $5.4 million for the three and six months
ended June 30, 2022, respectively,
compared to $2.5 million and
$5.0 million for the same periods in
2021, respectively. These amounts reflect personnel-related and
ongoing general business operating costs.
Net Loss: Net loss was $7.3
million, or $0.50 per share
(basic and diluted), and $14.0
million, or $0.96 per share (basic and diluted),
for the three and six months ended June 30,
2022, compared to $6.0
million, or $0.78 per share (basic and diluted),
and $12.0 million, or $1.62 per share (basic and
diluted), for the same periods 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 second 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) and refer to the entry
replay code 5578933. 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
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 160,000 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 for the
treatment of ADPKD 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 have been demonstrated
along with a superior pharmacologic profile in preclinical studies
compared to Regulus' first-generation compound. Regulus is
currently conducting a Phase 1 single-ascending dose study in
healthy volunteers to assess safety, tolerability, and
pharmacokinetics of RGLS8429. In June
2022, FDA granted ODD for RGLS849 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
June
30,
|
|
Six months
ended
June
30,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
4,708
|
|
4,150
|
|
8,387
|
|
7,470
|
General and
administrative
|
|
2,467
|
|
2,488
|
|
5,357
|
|
4,967
|
Total operating
expenses
|
|
7,175
|
|
6,638
|
|
13,744
|
|
12,437
|
Loss from
operations
|
|
(7,175)
|
|
(6,638)
|
|
(13,744)
|
|
(12,437)
|
Other (expense) income,
net
|
|
(83)
|
|
605
|
|
(232)
|
|
391
|
Loss before income
taxes
|
|
(7,258)
|
|
(6,033)
|
|
(13,976)
|
|
(12,046)
|
Income tax
expense
|
|
|
-
|
|
|
(1)
|
|
(1)
|
|
(1)
|
Net loss
|
|
$
|
(7,258)
|
|
$
|
(6,034)
|
|
$
|
(13,977)
|
|
$
|
(12,047)
|
Other comprehensive
loss:
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized loss on
short-term investments, net
|
|
|
(36)
|
|
|
-
|
|
|
(36)
|
|
|
-
|
Comprehensive
loss
|
|
|
(7,294)
|
|
|
(6,034)
|
|
|
(14,013)
|
|
|
(12,047)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.50)
|
|
$
|
(0.78)
|
|
$
|
(0.96)
|
|
$
|
(1.62)
|
Weighted average shares
used to compute basic and diluted net loss per share:
|
|
|
14,612,312
|
|
|
7,716,982
|
|
|
14,604,594
|
|
|
7,424,371
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
June 30,
2022
|
|
December 31,
2021
|
|
|
|
Cash, cash equivalents
and short-term investments
|
|
$
|
47,534
|
|
$
|
60,383
|
Total assets
|
|
54,298
|
|
68,454
|
Term loan, less debt
issuance costs
|
|
4,446
|
|
4,673
|
Stockholders'
equity
|
|
|
42,367
|
|
|
54,958
|
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SOURCE Regulus Therapeutics Inc.