Enrollment progressing in Phase 1b Multiple-Ascending Dose (MAD) study of
RGLS8429 in patients with Autosomal Dominant Polycystic Kidney
Disease (ADPKD)
Top-line data in the first cohort of
RGLS8429-treated ADPKD patients anticipated in second half of
2023
Expansion of team with key leadership
appointments in clinical development, regulatory and clinical
operations
SAN
DIEGO, March 23, 2023 /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 fourth quarter and year ended
December 31, 2022, and provided a
corporate update.
"We ended 2022 in a position of strength, with enrollment in our
Phase 1b MAD study under way. This
momentum was reinforced by the appointments of Drs. Rekha Garg and Claire
Padgett, who bring significant depth of industry, clinical
and regulatory experience to help us advance development of
RGLS8429," commented Jay Hagan, CEO
of Regulus. "With three months of dosing and one month of follow-up
for each patient in the study, we would anticipate topline data
approximately five months after we complete enrollment of the first
cohort."
Program Updates
RGLS8429 for 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, height-adjusted total 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, with
top-line data anticipated in the second half of 2023. The company
also recently completed the in-life portion of the 27-week chronic
mouse toxicity study for RGLS8429. No CNS toxicity was observed at
all dose levels up to the top dose of 300 mg/kg administered every
other week.
Corporate Highlights
Expanded Team: In November
2022, the Company announced the appointments of Rekha
Garg, M.D., M.S., to Senior Vice President, Clinical Development
and Regulatory, and Claire Padgett, Ph.D., to Senior Vice
President, Clinical Operations. In addition, the company announced
the promotions of Morgan Carlson, Ph.D., to Vice President,
Biology and Edmund Lee, Ph.D., to Vice President,
Translational Medicine.
Presented Data at the 5th Chronic Kidney Disease Drug
Development Summit: On March 9,
2023, the Company presented preclinical data on RGLS8429 at
the 2023 CKD Summit, entitled "Targeting miR-17 to Address the
Genetic Cause of ADPKD." Drs. Edmund
Lee and Rekha Garg
represented the Company.
Financial Results
Cash, Cash Equivalents and Marketable Securities: As
of December 31, 2022, Regulus had
$39.2 million in cash, cash
equivalents and short-term investments.
Research and Development (R&D) Expenses: Research and
development expenses were $4.7
million and $18.4 million for
the fourth quarter and year ended December
31, 2022, respectively, compared to $4.4 million and $17.8
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
$2.2 million and $9.8 million for the fourth quarter and year
ended December 31, 2022,
respectively, compared to $2.6
million and $10.0 million for
the same periods in 2021, respectively. These amounts reflect
personnel-related and ongoing general business operating costs.
Net Loss: Net loss was $6.8
million, or $0.40 per share
(basic and diluted), and $28.3
million, or $1.86 per share (basic and diluted),
for the fourth quarter and year ended December 31, 2022, compared to $7.1 million, or $0.67 per share (basic
and diluted), and $27.8 million, or $3.24 per share
(basic and diluted), for the same periods in 2021.
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 and plasma exposure was approximately
linear across the four doses tested 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 filed annual report on Form 10-K. 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
December
31,
|
|
Year
ended
December
31,
|
|
|
2022
|
|
2021
|
|
2022
|
|
2021
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
4,713
|
|
4,409
|
|
18,410
|
|
17,794
|
General and
administrative
|
|
2,219
|
|
2,551
|
|
9,829
|
|
10,022
|
Total operating
expenses
|
|
6,932
|
|
6,960
|
|
28,239
|
|
27,816
|
Loss from
operations
|
|
(6,932)
|
|
(6,960)
|
|
(28,239)
|
|
(27,816)
|
Other income (expense),
net
|
|
137
|
|
(173)
|
|
(83)
|
|
9
|
Loss before income
taxes
|
|
(6,795)
|
|
(7,133)
|
|
(28,322)
|
|
(27,807)
|
Income tax
expense
|
|
|
-
|
|
|
-
|
|
(1)
|
|
(1)
|
Net loss
|
|
$
|
(6,795)
|
|
$
|
(7,133)
|
|
$
|
(28,323)
|
|
$
|
(27,808)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.40)
|
|
$
|
(0.67)
|
|
$
|
(1.86)
|
|
$
|
(3.24)
|
Weighted average shares
used to compute basic and diluted net loss per
share:
|
|
|
16,839,700
|
|
|
10,689,683
|
|
|
15,259,958
|
|
|
8,569,854
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31,
2022
|
|
December 31,
2021
|
|
|
|
Cash, cash equivalents
and short-term investments
|
|
$
|
39,160
|
|
$
|
60,383
|
Total assets
|
|
46,716
|
|
68,454
|
Term loan, less debt
issuance costs
|
|
4,511
|
|
4,673
|
Stockholders'
equity
|
|
|
33,291
|
|
|
54,958
|
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SOURCE Regulus Therapeutics Inc.