LA JOLLA, Calif., Aug. 8, 2019 /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, 2019 and provided a summary of recent events.
"With the completed financing and restructuring of our term
loan, we believe we are in a good position to work expeditiously to
address the clear requirements outlined by FDA to reinitiate the
MAD clinical study for RGLS4326," said Jay
Hagan, CEO of Regulus. "We will also be evaluating potential
options to initiate single dose studies in order to further
characterize the molecule."
Second Quarter 2019 Corporate Highlights and Recent
Updates
- Private Financing: In May
2019, the Company closed the first tranche of its
$41.8 million private placement of
equity (the "Private Placement"). The Company received net proceeds
of approximately $15.7 million from
the first tranche, after deducting placement agent fees and other
offering expenses. Subject to the Company's public announcement on
or before December 31, 2019 of its
plan to recommence the Phase 1 multiple ascending dose ("MAD")
clinical trial for RGLS4326 based upon correspondence from the
United States Food and Drug Administration ("FDA"), the investors
who purchased securities in the first tranche of the Private
Placement have agreed to purchase shares of non-voting convertible
preferred stock and accompanying warrants to purchase shares of
common stock in a second closing (the "Milestone Closing"). If the
Milestone Closing occurs, the gross proceeds to the Company from
that closing will be approximately $25.1
million. The Company expects to use the proceeds from the
Private Placement primarily to advance RGLS4326 for the treatment
of autosomal dominant polycystic kidney disease ("ADPKD"), to
advance select programs from its pipeline of microRNA therapies and
for general corporate purposes. Excluding any potential proceeds
from the Milestone Closing, the Company believes it has sufficient
cash to fund operations into mid-2020.
- Term Loan Amendments: In May
2019, and concurrently with the Private Placement, the
Company amended its Term Loan with Oxford Finance to provide a new
twelve-month period of interest-only payments, commencing
May 2019, and a two-year extension of
its maturity date from June 2020 to
May 2022. Upon the closing of the
second tranche of the Company's Private Placement, the Company will
receive an additional twelve-month period of interest-only
payments, commencing May 2020.
- RG-012 Transition to Sanofi: In November 2018, the Company and Sanofi agreed to
transition further development activities of the miR-21 programs,
including the Company's RG-012 program, to Sanofi who will be
responsible for all costs incurred in the development of these
miR-21 programs (the "2018 Sanofi Amendment"). As of June 30, 2019, the transition activities,
including the transfer of the investigational new drug application
("IND"), were complete. The Company received a total of
$6.8 million in upfront and material
transfer-related payments, which were recognized as revenue in the
three months ended March 31, 2019.
Regulus is also eligible to receive up to $40 million in clinical milestone payments.
- Lease Agreement: In June
2019, the Company entered into an amendment of its lease
(the "Lease Amendment") of 24,562 square feet located at 10628
Science Center Drive Suite 100, San
Diego, California 92121. Under the terms of the Lease
Amendment, the expiration of the lease was accelerated from
June 30, 2023 to June 30, 2019, and the lease terminated on
July 1, 2019. Concurrently with the
Lease Amendment, the Company entered into a new lease agreement
(the "New Lease") for 8,727 square feet located at 10628 Science
Center Drive, Suite 225, San Diego,
California, 92121, which it uses as its new principal
offices and laboratory for research and development. This
relocation reduced the Company's facility size by approximately 65%
and reduced its future contractual lease obligations by
approximately 78%.
- Management Transition: In July
2019, the Company appointed Cris
Calsada as its new Chief Financial Officer, effective
August 30, 2019. Ms. Calsada joins
Regulus from Sanifit where she has served as Chief Financial
Officer since December 2017. Ms.
Calsada brings to Regulus' senior management team a unique
combination of financial, operational and managerial experience.
She has over 20 years of leadership experience in the life sciences
and technology industries. Prior to her employment with Sanifit,
Ms. Calsada was self-employed as a finance consultant to various
life sciences companies. From 2004 until its acquisition in 2015,
she served in positions of increasing responsibility with Ambrx,
most recently serving as its Chief Operating Officer and Vice
President of Finance. Prior to Ambrx, she worked for Sony Online
Entertainment as its Executive Director of Finance and Controller.
Earlier in her career, she practiced as a certified public
accountant. Ms. Calsada received a B.S. in Business Administration
with emphasis in Accounting from San Diego
State University and an M.B.A. from the University of Southern California Marshall School
of Business. Ms. Calsada's appointment follows the resignation of
the Company's previous Chief Financial Officer, Dan Chevallard, in July
2019.
Program Updates
- RGLS4326 for ADPKD: In January
2019, the Company announced data from a planned interim
analysis of a new mouse chronic toxicity study after 13 weeks of
dosing in which no adverse or other significant findings across the
range of doses tested were shown. In January
2019, the Company submitted a comprehensive data package for
RGLS4326 to FDA that included the results from the planned 13-week
interim analysis of the repeat mouse chronic toxicity study, as
well as results from additional investigations, analytical testing,
additional data from the previously terminated mouse chronic
toxicity study, data from the completed Phase I single ascending
dose ("SAD") study and data from the first cohort of the Phase I
MAD study, to support its plan to resume the Phase I MAD study.
After review of the requested submission, FDA notified the Company
of additional nonclinical data requirements and placed the IND on a
partial clinical hold, formalizing the specific requirements to
initiate the MAD study and further proceed into chronic dosing in
ADPKD patients. The additional data requirements have been outlined
in two parts. In order to resume the MAD study, FDA has requested
the final reports from the chronic toxicity studies in both mice
and non-human primates and satisfactory related analyses to ensure
subjects can be safely dosed. Additional data and analyses from new
nonclinical studies, planned to be generated over the next several
quarters, will be required for chronic dosing, and may also be used
to support the resumption of the MAD study. Regulus is allowed to
proceed with additional SAD clinical studies as part of the process
to gather additional supporting information to guide the future
development of the program.
- Anti-miR-132 for Nonalcoholic Steatohepatitis (NASH): In
April 2019, Regulus presented a late
breaker poster at the EASL International Liver Congress™ describing
the development of its lead anti-miR-132 for the treatment of NASH.
Across multiple animal models of NASH, the lead candidate
demonstrated improvement in key endpoints, including NAFLD Activity
Score (NAS), liver transaminases, hyperglycemia, and
disease-related gene expression. In the diet-induced NASH mouse
model (Amylin model) after two to four weekly doses, early onset of
improvement across multiple disease parameters including liver
triglycerides and blood levels of transaminases was observed. After
nine weeks of treatment, there was evidence of sustained benefit
with significant improvement of liver fibrosis and hyperglycemia
compared to control-treated animals. The Company believes that
targeting dysregulated microRNA in a complex disease like NASH may
offer a unique mechanism of action from other programs in
development. The Company plans to seek a partner to further advance
the development of this program.
Second Quarter 2019 Financial Results
Cash Position: As of June 30,
2019, Regulus had $19.6
million in cash and cash equivalents.
Revenue: Revenue was less than $0.1 million and $6.8
million for the three and six months ended June 30, 2019, respectively, compared to less
than $0.1 million for each the three
and six months ended June 30, 2018.
The increase for the six months ended June
30, 2019 was attributable to revenue recognition of the
upfront payments received under the 2018 Sanofi Amendment related
to the transfer of RG-012.
Research and Development (R&D) Expenses:
R&D expenses were $1.8 million
and $7.8 million for the three and
six months ended June 30, 2019,
compared to $10.0 million and
$21.8 million for the same periods in
2018. The decreases were driven by decreases in external
development expenses, primarily attributable to the voluntary pause
of the RGLS4326 Phase 1 MAD clinical study in the third quarter of
2018 and commencement of the transfer of the RG-012 program to
Sanofi in the fourth quarter of 2018. Additionally, the decreases
were driven by reductions in personnel and internal expenses,
primarily attributable to a reduction in costs subsequent to our
corporate restructuring in the third quarter of 2018.
General and Administrative (G&A) Expenses:
G&A expenses were $2.9 million
and $6.4 million for the three and
six months ended June 30, 2019,
compared to $3.3 million and
$7.1 million for the same periods in
2018. These amounts reflect personnel-related and ongoing general
business operating costs.
Net Loss: Net loss was $5.0
million, or $0.30 per share
(basic and diluted), and $8.3
million, or $0.61 per share
(basic and diluted), for the three and six months ended
June 30, 2019, respectively, compared
to $13.8 million, or $1.59 per share (basic and diluted), and
$29.9 million, or $3.44 per share (basic and diluted), for the same
periods in 2018. Historical and current period net loss per share
values have been retroactively adjusted to reflect our October 2018 reverse stock split.
About Autosomal Dominant Polycystic Kidney Disease
(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 in human ADPKD cyst cells, a reduction in kidney cyst
formation, improved kidney weight/body weight ratio, decreased cyst
cell proliferation, and preserved kidney function in mouse models
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 La Jolla,
California. For more information, please visit
http://www.regulusrx.com.
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 expected ability
of Regulus to undertake certain activities and accomplish certain
goals (including with respect to development and other activities
related to RG-012, RGLS4326, RGLS5579 or its other preclinical
programs), Regulus' sales of securities, including timing, size and
completion of the Milestone Closing, its estimated cash runway, the
projected timeline of clinical development activities, and
expectations regarding future therapeutic and commercial potential
of Regulus' business plans, technologies and intellectual property
related to microRNA therapeutics and biomarkers being discovered
and developed by Regulus. 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.
These and other risks concerning Regulus' financial position and
programs 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
June
30,
|
|
Six months
ended
June
30,
|
|
|
2019
|
|
2018
|
|
2019
|
|
2018
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Revenue under
strategic
alliances
|
|
$
|
18
|
|
$
|
18
|
|
$
|
6,796
|
|
$
|
36
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
Research and
development
|
|
1,836
|
|
10,013
|
|
7,819
|
|
21,841
|
General and
administrative
|
|
2,850
|
|
3,349
|
|
6,383
|
|
7,122
|
Total operating
expenses
|
|
4,686
|
|
13,362
|
|
14,202
|
|
28,963
|
Loss from
operations
|
|
(4,668)
|
|
(13,344)
|
|
(7,406)
|
|
(28,927)
|
Other expense,
net
|
|
(347)
|
|
(503)
|
|
(869)
|
|
(945)
|
Loss before income
taxes
|
|
(5,015)
|
|
(13,847)
|
|
(8,275)
|
|
(29,872)
|
Income tax
expense
|
|
|
(1)
|
|
|
-
|
|
(1)
|
|
(1)
|
Net loss
|
|
$
|
(5,016)
|
|
$
|
(13,847)
|
|
$
|
(8,276)
|
|
$
|
(29,873)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.30)
|
|
$
|
(1.59)
|
|
$
|
(0.61)
|
|
$
|
(3.44)
|
Weighted average
shares used to compute basic and diluted net loss per
share:
|
|
16,705,587
|
|
|
8,693,788
|
|
|
13,560,183
|
|
8,681,311
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
June
30,
2019
|
|
December 31,
2018
|
|
|
(Unaudited)
|
|
|
|
Cash and cash
equivalents
|
|
$
|
19,571
|
|
$
|
13,935
|
Total
assets
|
|
25,691
|
|
27,927
|
Term loan, less debt
issuance costs
|
|
14,621
|
|
16,575
|
Stockholders' equity
(deficit)
|
|
|
4,743
|
|
|
(5,854)
|
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SOURCE Regulus Therapeutics Inc.