Enrollment completed in first cohort of
patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
in Phase 1b Multiple-Ascending Dose
(MAD) study of RGLS8429
Closed $15.0
million private placement of equity; expected to extend cash
runway into mid-2024
SAN
DIEGO, May 11, 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 first quarter ended March 31, 2023 and provided a corporate
update.
"Our work in ADPKD continues to progress, with enrollment now
complete in cohort 1 of our Phase 1b
MAD study of RGLS8429," stated Jay Hagan, CEO of Regulus. "We
look forward to sharing topline data around the end of the third
quarter of this year. We also appreciate the continued support of
our top shareholders who participated in our recently announced
private placement of $15 million in
April, which we anticipate will extend our cash runway into
mid-2024 and through several additional program milestones."
Program Updates
RGLS8429 for ADPKD: In April
2023, the company announced the completion of enrollment for
the first cohort of patients in the Phase 1b MAD study of RGSL8429 for the treatment of
ADPKD. The Phase 1b MAD study is a
double-blind, placebo-controlled trial evaluating the safety,
tolerability, pharmacokinetics and pharmacodynamics (PK/PD) 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), cyst architecture, 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 around the end of the third quarter of
2023. The Company expects to begin dosing the second cohort
following a review of all available cohort 1 safety data.
Corporate Highlights
Closed $15.0 Million Private
Placement: On April 13,
2023, the Company announced that it entered into a
definitive securities purchase agreement in connection with a
private placement to certain institutional and other accredited
investors. The financing was led by the Federated Hermes Kaufmann
Funds and New Enterprise Associates (NEA), with participation from
additional existing shareholders.
Financial Results
Cash Position: As of March
31, 2023, Regulus had $30.3
million in cash and cash equivalents. Combined with the
$15.0 million private placement in
April, the Company expects its cash runway to extend into
mid-2024.
Research and Development (R&D)
Expenses: Research and development expenses were
$4.9 million for the three months
ended March 31, 2023, compared to
$3.7 million for the same period in
2022. 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
$2.4 million for the three months
ended March 31, 2023, compared to
$2.9 million for the same period in
2022. These amounts reflect personnel-related and ongoing general
business operating costs.
Net Loss: Net loss was $7.1
million, or $0.42 per share
(basic and diluted), for the three months ended March 31, 2023, compared to $6.7 million, or $0.46 per share (basic and diluted), for the same
period in 2022.
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, RGLS4326. Regulus
announced completion of the Phase 1 SAD study in September 2022 and completed enrollment of the
first cohort of patients in the Phase 1b MAD study in April of this year. 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 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, the expected timing for initiating clinical studies, the
expected timing for reporting topline data, the timing and future
occurrence of other preclinical and clinical activities and the
expected length of our cash runway. 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.
Investor Relations Contact:
Cris Calsada
Chief Financial Officer
858-202-6376
ccalsada@regulusrx.com
Media Contact:
Sarah Sutton
Argot Partners
212-600-1902
regulus@argotpartners.com
Regulus Therapeutics
Inc.
Selected Financial
Information
Condensed Statement
of Operations
(In thousands,
except share and per share data)
|
|
|
|
Three months
ended
March
31,
|
|
|
2023
|
|
2022
|
|
|
|
|
|
|
Operating
expenses:
|
|
|
|
|
|
Research and
development
|
|
4,925
|
|
3,679
|
|
General and
administrative
|
|
2,444
|
|
2,890
|
|
Total operating
expenses
|
|
7,369
|
|
6,569
|
|
Loss from
operations
|
|
(7,369)
|
|
(6,569)
|
|
Other income (expense),
net
|
|
230
|
|
(149)
|
|
Loss before income
taxes
|
|
(7,139)
|
|
(6,718)
|
|
Income tax
expense
|
|
|
-
|
|
|
(1)
|
|
Net loss
|
|
$
|
(7,139)
|
|
$
|
(6,719)
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.42)
|
|
$
|
(0.46)
|
|
Weighted average shares
used to compute basic and diluted net loss per share:
|
|
|
16,844,243
|
|
|
14,596,789
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
March 31,
2023
|
|
December 31,
2022
|
|
|
|
Cash, cash equivalents
and short-term investments
|
|
$
|
30,308
|
|
$
|
39,160
|
Total assets
|
|
37,015
|
|
46,716
|
Term loan, less debt
issuance costs
|
|
3,716
|
|
4,511
|
Stockholders'
equity
|
|
|
26,595
|
|
|
33,291
|
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SOURCE Regulus Therapeutics Inc.