On June 12, 2017, the Company announced in a press release the following updates
with respect to the Companys pipeline.
RG-012
for Alport syndrome
: Initiation of the Phase II
clinical programs for
RG-012
for the treatment of Alport syndrome is on track as planned. Important changes to the Phase II study design have been incorporated with the goal to accelerate patient
enrollment, improve statistical power, and potentially achieve proof of mechanism data by the end of 2017. HERA, the Phase II randomized (1:1), double-blinded, placebo-controlled study evaluating the safety and efficacy of
RG-012
in Alport syndrome patients, has been modified to increase enrollment to 40 patients to improve its statistical power. Dose frequency has also been adjusted to once every other week. The separate renal biopsy
study will evaluate
RG-012
renal tissue pharmacokinetics, target engagement and downstream effects on genomic disease biomarkers. Data from the renal biopsy study is anticipated by
year-end
and interim data from HERA is anticipated
mid-2018.
RG-101
(anti-miR122) for HCV:
The Company plans to discontinue clinical development of
RG-101 upon
completion of the one remaining
clinical study, which is expected to occur in July 2017. Comprehensive
pre-clinical
investigation and thorough evaluation of the clinical data from
RG-101
has
led to the identification of a bilirubin transport mechanism as the likely cause for the cases of hyperbilirubinemia in the
RG-101
program. The Company believes that a combination of factors including
inhibition of conjugated bilirubin transport by
RG-101,
impaired baseline bilirubin transport in HCV patients and the preferential uptake of
RG-101
by hepatocytes
contributed to this mechanism. Additional patient specific contributing factors cannot be excluded. Applying the learnings from the
RG-101
program, alternative
compounds targeting miR-122 have
been identified that maintain potent HCV antiviral activity while lacking inhibition of the bilirubin transporter. These
compounds have the potential for rapid clinical
proof-of-concept
of a novel, markedly shortened treatment regimen for HCV and will be considered for further
development pending an updated global commercial market assessment for HCV.
RGLS4326
(anti-miR-17)
for autosomal dominant polycystic kidney disease
(
ADPKD):
The IND for RGLS4326 is on track for filing by year end
2017. IND enabling toxicology, repeat pharmacology and manufacturing work have been completed as scheduled to support regulatory submissions. Data from the
pre-clinical
program have been recently
published in
Nature Communications
and support the rationale for targeting
miR-17
for the treatment of ADPKD, an orphan indication with no treatment options affecting approximately 600,000
people in the United States.
RGLS5040
(anti-miR-27)
for cholestatic
disease:
RGLS5040, an unconjugated inhibitor of microRNA27, has been discontinued based on a positioning of the compound with respect to the competitive landscape coupled with the results from repeat pharmacology studies as part of
IND-enabling
work. The Company continues to work on developing highly effective therapeutics for genetic forms of cholestatic disease as part of its overall research activities targeting unmet diseases of the
liver and kidney.
Forward-Looking Statements
Statements contained in this Current Report on Form
8-K
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 the Company to undertake certain activities and accomplish certain goals
(including with respect to development and other activities related to
RG-101
or
RG-012),
the projected timeline of clinical development activities, and expectations
regarding future therapeutic and commercial potential of the Companys business plans, technologies and intellectual property related to microRNA therapeutics and biomarkers being discovered
and developed by the Company. 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 the Companys 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 the Companys financial position and programs are described in additional detail in the Company filings with the Securities
and Exchange Commission, including under the caption Risk Factors in Regulus Quarterly Report on Form
10-Q
for the quarter ended March 31, 2017. All forward-looking statements
contained in this Current Report on Form
8-K
speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that
exist after the date on which they were made.