LA JOLLA, Calif., March 9, 2021 /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, 2020 and provided a
corporate update.
"Regulus finished the year on a high note", stated Jay Hagan, CEO of Regulus. "We advanced RGLS4326
into a Phase 1b study in ADPKD
patients, achieved an enrollment milestone for RG-012 with Sanofi
and its Phase 2 clinical study for the treatment of patients with
Alport syndrome, and completed a private financing. With the
recent progress, we believe we are well positioned to advance the
ADPKD program through Phase 1b while
also advancing our next generation ADPKD compound toward the
clinic."
Program Updates
RGLS4326 for ADPKD: In February
2021, the Company completed enrollment in the first cohort
of a Phase 1b clinical study for
RGLS4326 in patients with ADPKD (The "Phase 1b"). The Phase 1b
is an adaptive design, open-label, multiple dose study in up to
three cohorts of patients with ADPKD. The study is designed
to evaluate the safety, pharmacokinetics, and changes in levels of
polycystin 1 ("PC1") and polycystin 2 ("PC2") in patients with
ADPKD administered RGLS4326 every other week for a total of four
doses. The dose level for the first cohort is 1 mg/kg of
RGLS4326 and the dose level for the second cohort is 0.3
mg/kg. The third and final cohort will be dosed at a level to
be determined based on the results of the first two cohorts.
Concurrent with completion of enrollment in the first cohort and
based on the review of the available interim safety data, the first
patient of the second cohort has commenced dosing of RGLS4326. The
Company anticipates availability of results from the first cohort
in early Q2 2021.
RG-012 for Alport syndrome: In November of 2020, the
Company announced that it has achieved the remaining $5 million milestone associated with interim
enrollment under its Collaboration and License Agreement (the
"Milestone") with Sanofi for its development of miR-21 programs.
The Milestone was triggered upon achievement of an enrollment
metric by Sanofi in its Phase 2 clinical study evaluating RG-012
for the treatment of patients with Alport syndrome. Results from a
previously completed biopsy study demonstrated kidney tissue
concentrations that would be predictive of therapeutic benefit
based on animal disease models as well as engagement of the target,
miR-21. Over the course of the one year open-label study, promising
trends in disease markers were observed and RG-012 was generally
well tolerated with no serious adverse events. RG-012 has received
orphan designation in both the U.S. and Europe.
Corporate Highlights
Closed $19 Million Private
Financing: In December 2020, the
Company entered into a definitive securities purchase agreement
with certain existing and new institutional investors. The Company
received gross proceeds of approximately $19 million from
the sale of 24,341,607 shares of the Company's common stock
("Common Stock") and accompanying warrants to purchase up to an
aggregate of 18,256,204 shares of Common Stock at a purchase price
of $0.622 per share of Common Stock
and $0.125 for each share of Common Stock underlying such
warrants. In addition, the Company sold 272,970 shares of
non-voting Class A-3 convertible preferred stock, in lieu of shares
of Common Stock, at a price of $6.22 per share, and
accompanying warrants to purchase an aggregate of 2,047,276
shares of Common Stock at a price of $0.125 for each
share of Common Stock underlying these warrants. Each share
of non-voting Class A-3 convertible preferred stock is convertible
into 10 shares of Common Stock, subject to certain beneficial
ownership conversion limitations. Investors in the private
placement included existing institutional investors, New Enterprise
Associates (NEA) and BVF Partners L.P., and members of the
Company's Board and management. The financing also included
participation from several new institutional investors, including
RS Investments, Point72 and Asymmetry Capital. The Company expects
to use the net proceeds of approximately $18M from the transaction primarily to advance
RGLS4326 for the treatment of ADPKD and for general corporate
purposes, and the Company expects net proceeds, together with
existing cash, will provide cash resources to fund planned
activities through Q1 2022.
Expanded Board of Directors: In January 2021, Regulus announced the appointment
of Dr. Alice Huang to the Company's
Board of Directors. Dr. Huang brings an extensive scientific
background to the Board to help direct the company's drug discovery
and development efforts. Dr. Huang is currently Senior Faculty
Associate of Biology and Biological Engineering at
the California Institute of Technology having
joined Caltech in July 1997. Previous to her
tenure at Caltech she was Dean for Science and Professor
of Biology at New York University,
Professor of Microbiology and Molecular Genetics at Harvard
Medical School and Director, Laboratories of Infectious
Disease at Boston Children's Hospital.
Fourth Quarter 2020 Financial Results
Cash Position: As of December
31, 2020, Regulus had $31.1
million in cash and cash equivalents.
Revenue: Revenue was $5.0
million and $10.0 million for
the quarter and year ended December 31,
2020, respectively, compared to less than $0.1 million and $6.8
million for the same periods in 2019. Revenue recognized for
the quarter and year ended December 31,
2020 was attributable to the achievement of the enrollment
milestone and completion of transfer and verification of certain
materials to Sanofi under the August
2020 amendment to our collaboration agreement with
Sanofi related to RG-012, currently in Phase 2 for Alport
syndrome. Revenue recognized for the same periods in 2019 was
attributable to the upfront payments received under the 2018 Sanofi
amendment related to the transfer of the RG-012 program to
Sanofi.
Research and Development (R&D)
Expenses: Research and development expenses were
$4.0 million and $15.3 million for the quarter and year ended
December 31, 2020, respectively,
compared to $2.1 million and
$12.3 million for the same periods in
2019. The aggregate increase for the quarter ended December 31, 2020, as compared to the quarter
ended December 31, 2019, was driven
by an increase in external development expenses, primarily
attributable to start-up activities and initiation of enrollment in
our RGLS4326 Phase 1b study in
October 2020. The aggregate increase
for the year ended December 31, 2020,
as compared to the year ended December 31,
2019, was driven by an increase in external development
expenses, attributable to RGLS4326 MAD and Phase 1b study activities, partially offset by a
reduction in personnel and internal expenses.
General and Administrative (G&A)
Expenses: General and administrative expenses were
$2.1 million and $8.8 million for the quarter and year ended
December 31, 2020, compared to
$2.4 million and $11.3 million for the same periods in 2019. These
amounts reflect personnel-related and ongoing general business
operating costs. The decreases are primarily attributable to
continued cost reduction efforts.
Net Loss: Net loss was $1.3
million, or $0.03 per share
(basic and diluted), and $15.7
million, or $0.45 per share
(basic and diluted), for the quarter and year ended December 31, 2020, respectively, compared to
$4.9 million, or $0.23 per share (basic and diluted), and
$18.6 million, or $1.08 per share (basic and diluted), for the same
periods in 2019.
About ADPKD
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.
About RGLS4326
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17
and designed to preferentially target the kidney. Preclinical
studies with RGLS4326 have demonstrated direct regulation of
Pkd1 and Pkd2, reduction of cyst growth in human
in vitro ADPKD models, and attenuation of cyst proliferation
and improvement of kidney function in mouse models of
ADPKD. The RGLS4326 IND is currently on a Partial Clinical
Hold for treatment of extended duration by FDA until the
second set of requirements outlined by the agency have been
satisfactorily addressed. The Company will use information from the
Phase 1 clinical studies, including the first cohort of the Phase
1b together with information from the
recently completed additional nonclinical studies generated in
2020, in its plan to address the second set of requirements
outlined in the Partial Clinical Hold letter to support studies of
extended duration. Regulus plans to discuss its approach to
addressing the remaining Partial Clinical Hold requirements with
FDA in mid-2021. RGLS4326 received orphan drug designation
from FDA in July 2020.
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 La Jolla,
CA.
Forward-Looking Statements
Statements contained in this press release 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 completion of
preclinical and clinical activities concerning the RGLS4326 and
RG-012 programs, the sufficiency of the data resulting from the
ongoing or planned preclinical studies required to recommence
clinical studies for extended duration dosing and the timing of
preclinical, clinical activities and regulatory 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 feedback from the
FDA. 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. 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,
|
|
2020
|
|
2019
|
|
2020
|
|
2019
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
$
|
5,000
|
|
$
|
18
|
|
$
|
10,006
|
|
$
|
6,832
|
Operating
expenses:
|
|
|
|
|
|
|
|
Research and
development
|
3,951
|
|
2,090
|
|
15,347
|
|
12,349
|
General and
administrative
|
2,078
|
|
2,363
|
|
8,814
|
|
11,317
|
Total operating
expenses
|
6,029
|
|
4,453
|
|
24,161
|
|
23,666
|
Loss from
operations
|
(1,029)
|
|
(4,435)
|
|
(14,155)
|
|
(16,834)
|
Other expense,
net
|
(286)
|
|
(458)
|
|
(1,575)
|
|
(1757)
|
Loss before income
taxes
|
(1,315)
|
|
(4,893)
|
|
(15,730)
|
|
(18,591)
|
Income tax benefit
(expense)
|
|
(7)
|
|
|
-
|
|
0
|
|
(1)
|
Net loss
|
$
|
(1,322)
|
|
$
|
(4,893)
|
|
$
|
(15,730)
|
|
$
|
(18,592)
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(0.03)
|
|
$
|
(0.23)
|
|
$
|
(0.45)
|
|
$
|
(1.08)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
47,731,012
|
|
|
20,950,602
|
|
|
34,977,378
|
|
|
17,260,176
|
|
|
December 31,
2020
|
|
December 31,
2019
|
|
|
|
Cash and cash
equivalents
|
|
$
|
31,087
|
|
$
|
34,121
|
Total
assets
|
|
37,604
|
|
42,081
|
Term loan, less debt
issuance costs
|
|
4,652
|
|
14,631
|
Stockholders'
equity
|
|
|
26,026
|
|
|
20,015
|
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SOURCE Regulus Therapeutics Inc.