Regulus Therapeutics Inc. (NASDAQ:RGLS) Files An 8-K Termination of a Material Definitive Agreement

Regulus Therapeutics Inc. (NASDAQ:RGLS) Files An 8-K Termination of a Material Definitive Agreement

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Item1.02

Termination of a Material Definitive
Agreement.

On June9, 2017, AstraZeneca AB (AstraZeneca) delivered written
notice to Regulus Therapeutics Inc. (the Company) of AstraZenecas
election to terminate the collaboration and license agreement
entered into between AstraZeneca and the Company on August5,
2012, as amended (the Agreement). In accordance with the
Agreement, the termination will become effective on June9, 2018,
which is 12 months following the date of delivery of the notice
by AstraZeneca.

Under the terms of the Agreement, the Company agreed to
collaborate with AstraZeneca to identify, research and develop
compounds targetingthreemicroRNA alliance targets primarily in
the fields of cardiovascular diseases, metabolic diseases and
oncology. to the Agreement, the Company granted AstraZeneca an
exclusive, worldwide license to develop, manufacture and
commercialize lead compounds designated by AstraZeneca in the
course of the collaboration activities against the alliance
targets for all human therapeutic uses. Under the terms of the
Agreement the Company was required to use commercially reasonable
efforts to perform all research, development and manufacturing
activities described in the research plan, at the Companys cost,
until the acceptance of an investigational new drug application
or the end of the research term, which expired in August 2016.
AZD4076(RG-125) was jointly identified and selected as a clinical
candidate inApril 2015by AstraZeneca to the Agreement. Effective
upon the termination of the Agreement, AstraZenecas rights with
respect to AZD4076(RG-125) will revert to the Company.

Item8.01 Other Events.

On June12, 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 todiscontinueclinical
development of RG-101upon completion of the one remaining
clinical study, which is expected to occurinJuly 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
compoundstargetingmiR-122have been identifiedthat maintain potent
HCVantiviralactivitywhile lackinginhibition ofthe
bilirubintransporter. These compounds have the potential for
rapidclinical proof-of-concept of a novel, markedly shortened
treatment regimen for HCV andwill 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 inNature
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
theSecurities and Exchange Commission, including under the
caption Risk Factors in Regulus Quarterly Report on Form 10-Q for
the quarter ended March31, 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.


About Regulus Therapeutics Inc. (NASDAQ:RGLS)

Regulus Therapeutics Inc. is a biopharmaceutical company focused on discovering and developing drugs that target microRNAs to treat a range of diseases. The Company uses its microRNA product platform to develop chemically modified, single-stranded oligonucleotides that the Company calls anti-miRs to modulate microRNAs and return diseased cells to their healthy state. The Company’s Regulus microMarkers division is focused on identifying microRNAs as biomarkers of human disease. It has a research collaboration with Biogen focused on the discovery of microRNAs as biomarkers for multiple sclerosis and has completed research for another pharmaceutical company to explore microRNAs as biomarkers for specific patient populations. The Company is developing RG-101, an N-Acetylgalactosamine (GalNAc)-conjugated anti-miR targeting miR-122; RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, and RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107.

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