Company announced first patient dosed in its
Phase 1b Multiple-Ascending Dose
(MAD) study of RGLS8429 in patients with Autosomal Dominant
Polycystic Kidney Disease (ADPKD)
Company announced positive topline safety and
pharmacokinetic (PK) data from its Phase 1 Single-Ascending Dose
(SAD) study of RGLS8429 in healthy volunteers for the
treatment of ADPKD
$4.5 million in net proceeds in
At-the-Market (ATM) sale to a new institutional investor
SAN
DIEGO, Nov. 10, 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 third quarter ended September 30, 2022 and provided a corporate
update.
"We are pleased with the positive safety and PK data in healthy
volunteers in our Phase 1 SAD study and are delighted to have the
first patient dosed in our ongoing Phase 1b MAD study, which brings us a step forward in
investigating RGLS8429 as a potential treatment for ADPKD,"
commented Jay Hagan, CEO of Regulus.
"We anticipate topline data from the first cohort of patients in
mid-2023."
Program Updates
RGLS8429 for ADPKD: In early November, the Company
announced the dosing of the first patient in the Phase 1b MAD study of RGLS8429 for the treatment of
ADPKD. The Phase 1b MAD study
is a double-blind, placebo-controlled trial to assess safety,
tolerability, and pharmacokinetics of RGLS8429 in adult patients
with ADPKD. The study will evaluate the safety and efficacy of
RGLS8429 treatment across three different dose levels, including
measuring changes in polycystins, cystic kidney volume (htTKV), and
overall kidney function. The first cohort is being dosed at 1 mg/kg
of RGLS8429 or placebo every other week for three months.
In September 2022, the Company
announced positive topline safety and PK data from its Phase 1 SAD
clinical trial of RGLS8429. RGLS8429 was well-tolerated, with no
serious adverse events reported. Among the 32 subjects treated with
RGLS8429 or placebo, there were nine adverse events, all of which
were mild, except one (sinus infection), which was graded moderate
in severity. Preliminary results suggest plasma exposure is
approximately linear across the four doses tested and is similar to
the PK data from the first-generation compound, RGLS4326.
Corporate Highlights
Raised $4.5 Million in Net Proceeds Through its ATM
Facility: A total of 2,205,100 shares were sold and
settled for net proceeds of $4.5 million under the ATM
facility during the three months ended September 30, 2022.
Almost all the shares were sold to a new institutional investor.
The Company believes its existing cash, cash equivalents, and
short-term investments as of September 30,
2022 will now provide cash resources to fund current planned
activities through 2023.
Company Presented Recent Data at the American Society of
Nephrology Kidney Week 2022, which took place
in Orlando, November 3-6, 2022: The poster,
titled "Discovery of Next-generation Anti-miR-17 Oligonucleotide
RGLS8429 for Treatment of Autosomal Dominant Polycystic Kidney
Disease (ADPKD)," provides an overview of the data supporting the
potential of RGLS8429 as a treatment for ADPKD.
Financial Results
Cash, Cash Equivalents and Marketable Securities: As of
September 30, 2022, Regulus had
$45.3 million in cash, cash
equivalents and short-term investments.
Research and Development (R&D)
Expenses: Research and development expenses were
$5.3 million and $13.7 million for the three and nine months ended
September 30, 2022, respectively,
compared to $5.9 million and
$13.4 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
$2.3 million and $7.6 million for the three and nine months
ended September 30, 2022,
respectively, compared to $2.5
million and $7.5 million for the
same periods in 2021, respectively. These amounts reflect
personnel-related and ongoing general business operating costs.
Net Loss: Net loss was $7.6
million, or $0.50 per share
(basic and diluted), and $21.5
million, or $1.46 per share (basic and diluted),
for the three and nine months ended September 30, 2022, compared to $8.6 million, or $0.99 per share (basic
and diluted), and $20.7 million, or $2.63 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 Standard Time to discuss its third
quarter 2022 financial results and corporate update. To
access the call, please dial (866) 652-5200 (domestic) or (412)
317-6060 (international) and use the conference ID
10171026. 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 5033234. 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, RGLS326. Regulus
announced completion of the Phase 1 SAD study in September 2022 and dosed the first patient in the
Phase 1b MAD study in early
November. The Phase 1 SAD study demonstrated that RGLS8429
has a favorable safety and PK profile. RGLS8429 was
well-tolerated with no serious adverse events reported.
Preliminary results suggest plasma exposure is approximately linear
across the four doses evaluated and is similar to the PK data from
the first-generation compound.
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 expected timing for initiating clinical
studies, 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,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
5,310
|
|
5,915
|
|
13,697
|
|
13,385
|
General and
administrative
|
|
2,253
|
|
2,504
|
|
7,610
|
|
7,471
|
Total operating
expenses
|
|
7,563
|
|
8,419
|
|
21,307
|
|
20,856
|
Loss from
operations
|
|
(7,563)
|
|
(8,419)
|
|
(21,307)
|
|
(20,856)
|
Other income (expense),
net
|
|
12
|
|
(209)
|
|
(220)
|
|
182
|
Loss before income
taxes
|
|
(7,551)
|
|
(8,628)
|
|
(21,527)
|
|
(20,674)
|
Income tax
expense
|
|
|
-
|
|
|
-
|
|
(1)
|
|
(1)
|
Net loss
|
|
$
|
(7,551)
|
|
$
|
(8,628)
|
|
$
|
(21,528)
|
|
$
|
(20,675)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.50)
|
|
$
|
(0.99)
|
|
$
|
(1.46)
|
|
$
|
(2.63)
|
Weighted average shares
used to compute basic and diluted net loss per share:
|
|
|
14,969,574
|
|
|
8,703,637
|
|
|
14,727,591
|
|
|
7,855,479
|
|
|
September 30,
2022
|
|
December 31,
2021
|
|
|
(Unaudited)
|
Cash and cash
equivalents
|
|
$
|
45,324
|
|
$
|
60,383
|
Total assets
|
|
52,127
|
|
68,454
|
Term loan, less debt
issuance costs
|
|
4,478
|
|
4,673
|
Stockholders'
equity
|
|
|
39,670
|
|
|
54,958
|
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SOURCE Regulus Therapeutics Inc.