ROCKVILLE, Md. and ALPHARETTA, Ga., Sept.
4, 2019 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a
leading clinical-stage biotechnology company seeking to improve
lives through the curative potential of gene therapy based on its
proprietary NAV® Technology Platform, and Clearside
Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company
dedicated to developing and delivering treatments that restore and
preserve vision for people with serious back of the eye diseases,
today announced an option and license agreement for exclusive
worldwide rights to Clearside's proprietary, in-office SCS
Microinjector™ for the delivery of RGX-314 to the
suprachoroidal space to treat wet age-related macular degeneration
(wet AMD), diabetic retinopathy (DR) and other conditions for which
anti-vascular endothelial growth factor (anti-VEGF) treatment is
currently the standard of care. REGENXBIO plans to evaluate RGX-314
using Clearside's SCS Microinjector for in-office, non-surgical
delivery into the suprachoroidal space, while continuing to advance
its RGX-314 subretinal delivery program currently in development
for wet AMD and DR.
Delivery of NAV AAV8-based gene therapy through
the suprachoroidal space can potentially provide a targeted,
in-office, non-surgical approach to obtaining widespread transgene
expression in the retina without exposing the vitreous and the
anterior segment of the eye to the injected drug.
Clearside's patented SCS Microinjector is specifically
designed to allow for consistent injection into the suprachoroidal
space and has been tested in over 1,000 injections in
clinical trials to date.
"We are pleased to partner with Clearside in evaluating the use
of the SCS Microinjector to deliver RGX-314 to the suprachoroidal
space in the eye. We believe this approach can potentially allow
for the treatment of an expanded population of patients with wet
AMD and DR by providing access to gene therapy treatment in all
settings of patient care," said Kenneth T.
Mills, President and Chief Executive Officer of
REGENXBIO. "Delivery into the suprachoroidal space, which in
clinical trials has demonstrated minimal associated procedural
risks, may allow physicians to treat patients with diseases like DR
earlier in the disease course with RGX-314. We will continue to
advance the RGX-314 subretinal delivery clinical program, with a
Phase IIb clinical trial in wet AMD and a Phase II clinical trial
in DR expected to begin by the end of 2019, and we look forward to
moving both delivery approaches through development as part of our
deep commitment to patients."
"REGENXBIO is the ideal partner for us given its leadership,
expertise and pioneering work in the development and manufacturing
of AAV vectors for the delivery of anti-VEGF therapeutic antibodies
in the ophthalmology space," said George Lasezkay, Pharm.D., J.D.,
Chief Executive Officer of Clearside. "We are excited by the recent
positive interim data reported by REGENXBIO from the current
RGX-314 Phase I/IIa trial in wet AMD. This is an exciting time for
us to collaborate with REGENXBIO to evaluate the potential
application of our proprietary in-office SCS Microinjector for AAV
gene therapy."
A recent research collaboration with Johns
Hopkins School of Medicine published in The Journal of
Clinical Investigation1 highlights the potential of
RGX-314 gene therapy delivered into the suprachoroidal
space. In these preclinical studies, delivery of RGX-314 into
the suprachoroidal space resulted in similar expression of
anti-VEGF Fab and suppression of VEGF-induced vascular leakage
as subretinal delivery.
"Intravitreal delivery of AAV gene therapy can be limited by
ocular inflammation, immune responses in the vitreous and
restrictive physical transduction barriers due to the inner
limiting membrane on the retina. These hurdles can potentially be
overcome with alternative in-office delivery approaches such as
delivery to the suprachoroidal space," said Olivier Danos, Ph.D., Chief Scientific Officer
of REGENXBIO. "Preclinical suprachoroidal delivery studies
conducted with the Johns Hopkins University
School of Medicine demonstrated encouraging initial results
in multiple animal species, and we look forward to further
evaluation of this route of administration for RGX-314."
"Following on the positive long-term clinical results from
RGX-314 delivered subretinally, it is exciting to see that
preliminary preclinical studies with suprachoroidal delivery of
RGX-314 achieved widespread transgene expression in the retina,
similar to what was observed in RGX-314 preclinical studies using
subretinal delivery. Suprachoroidal delivery of gene therapy has
the opportunity for broad transduction of the retina and has the
potential to be the preferred choice for in-office treatment of wet
AMD, DR and other chronic retinal diseases," said Robert Avery, M.D., study investigator from
California Retina Consultants in Santa
Barbara, CA. "I am thrilled to see REGENXBIO evaluating an
in-office procedure in addition to the on-going RGX-314 subretinal
delivery program, which has the potential to provide multiple
treatment options to patients with significant unmet needs."
Under the terms of the agreement, Clearside has granted
REGENXBIO an option to receive an exclusive, worldwide commercial
license, with rights to sublicense, to Clearside's SCS
Microinjector for the delivery of AAV gene therapies for the
treatment of wet AMD, DR, and other conditions for which chronic
anti-VEGF treatment is currently the standard of care. In return
for these rights, Clearside will receive a fee upon REGENXBIO's
exercise of its option, as well as up to $34
million in total development milestones across multiple
indications, up to $102 million in
sales milestones and mid-single digit royalties on net sales of
products using the SCS Microinjector. REGENXBIO will be responsible
for all development and commercialization activities for gene
therapy product candidates. Clearside will be responsible for
supplying the SCS Microinjector and supporting REGENXBIO's
preclinical studies, clinical studies and commercial use.
About RGX-314
RGX-314 is being developed as a potential one-time treatment for
wet AMD, DR and other additional chronic retinal conditions treated
with anti-VEGF. RGX-314 consists of the NAV AAV8 vector encoding an
antibody fragment which inhibits VEGF, modifying the pathway for
formation of new leaky blood vessels which lead to retinal fluid
accumulation and vision loss. In REGENXBIO's on-going clinical
trial in subjects with wet AMD, dose-dependent increases in
protein expression levels across five cohorts have been seen, and
50% of Cohort 3 subjects continued to be free of anti-VEGF
injections at 18 months following a single subretinal
administration of RGX-314.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene
therapy. REGENXBIO's NAV Technology Platform, a proprietary
adeno-associated virus (AAV) gene delivery platform, consists of
exclusive rights to more than 100 novel AAV vectors, including
AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV
Technology Platform Licensees are applying the NAV Technology
Platform in the development of a broad pipeline of candidates in
multiple therapeutic areas. For more information, visit
http://www.regenxbio.com.
About Clearside Biomedical, Inc.
Clearside Biomedical, Inc. is a biopharmaceutical company
dedicated to developing and delivering treatments that restore and
preserve vision for people with serious back of the eye diseases.
Clearside's proprietary SCS Microinjector™ targeting
the suprachoroidal space (SCS) offers unique access to the macula,
retina and choroid where sight-threatening disease often occurs.
The Company's SCS injection platform is an inherently flexible,
in-office, non-surgical procedure, intended to provide targeted
delivery to the site of the disease and to work with both
established and new formulations of medications, as well as future
therapeutic innovations such as gene therapy. For more information,
please visit www.clearsidebio.com.
REGENXBIO Forward-Looking Statements
This press release includes "forward-looking statements," within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. These statements express a belief, expectation or
intention and are generally accompanied by words that convey
projected future events or outcomes such as "believe," "may,"
"will," "estimate," "continue," "anticipate," "design," "intend,"
"expect," "could," "plan," "potential," "predict," "seek,"
"should," "would" or by variations of such words or by similar
expressions. The forward-looking statements include statements
relating to, among other things, REGENXBIO's future operations,
preclinical studies, clinical trials and cash flow. REGENXBIO has
based these forward-looking statements on its current expectations
and assumptions and analyses made by REGENXBIO in light of its
experience and its perception of historical trends, current
conditions and expected future developments, as well as other
factors REGENXBIO believes are appropriate under the circumstances.
However, whether actual results and developments will conform with
REGENXBIO's expectations and predictions is subject to a number of
risks and uncertainties, including the timing of enrollment,
commencement and completion and the success of clinical trials
conducted by REGENXBIO, its licensees and its partners, the timing
of commencement and completion and the success of preclinical
studies conducted by REGENXBIO and its development partners, the
timely development and launch of new products, the ability to
obtain and maintain regulatory approval of product candidates, the
ability to obtain and maintain intellectual property protection for
product candidates and technology, trends and challenges in the
business and markets in which REGENXBIO operates, the size and
growth of potential markets for product candidates and the ability
to serve those markets, the rate and degree of acceptance of
product candidates, and other factors, many of which are beyond the
control of REGENXBIO. Refer to the "Risk Factors" and "Management's
Discussion and Analysis of Financial Condition and Results of
Operations" sections of REGENXBIO's Annual Report on Form 10-K for
the year ended December 31, 2018, and
comparable "risk factors" sections of REGENXBIO's Quarterly Reports
on Form 10-Q and other filings, which have been filed with the U.S.
Securities and Exchange Commission (SEC) and are available on the
SEC's website at www.sec.gov. All of the forward-looking statements
made in this press release are expressly qualified by the
cautionary statements contained or referred to herein. The actual
results or developments anticipated may not be realized or, even if
substantially realized, they may not have the expected consequences
to or effects on REGENXBIO or its businesses or operations. Such
statements are not guarantees of future performance and actual
results or developments may differ materially from those projected
in the forward-looking statements. Readers are cautioned not to
rely too heavily on the forward-looking statements contained in
this press release. These forward-looking statements speak only as
of the date of this press release. REGENXBIO does not undertake any
obligation, and specifically declines any obligation, to update or
revise any forward-looking statements, whether as a result of new
information, future events or otherwise.
Clearside Forward-Looking Statements
Any statements contained in this press release that do not
describe historical facts may constitute forward-looking statements
as that term is defined in the Private Securities Litigation Reform
Act of 1995. These statements may be identified by words such as
"believe", "expect", "may", "plan", "potential", "will", and
similar expressions, and are based on Clearside's current beliefs
and expectations. These forward-looking statements include
statements regarding the potential application of the SCS
Microinjector for AAV gene therapy and the potential benefits of
the SCS injection platform. These statements involve risks and
uncertainties that could cause actual results to differ materially
from those reflected in such statements. Risks and uncertainties
that may cause actual results to differ materially include
uncertainties inherent in the conduct of clinical trials,
Clearside's reliance on third parties over which it may not always
have full control, and other risks and uncertainties that are
described in Clearside's Annual Report on Form 10-K for the year
ended December 31, 2018, filed with
the U.S. Securities and Exchange Commission ("SEC") on March 15, 2019, Clearside's Quarterly Report on
Form 10-Q for the quarter ended June 30,
2019, filed with the SEC on August 8,
2019, and Clearside's other Periodic Reports filed with the
SEC. Any forward-looking statements speak only as of the date of
this press release and are based on information available to
Clearside as of the date of this release, and Clearside assumes no
obligation to, and does not intend to, update any forward-looking
statements, whether as a result of new information, future events
or otherwise.
- Ding, K., Shen, J., Hafiz, Z., Hackett, S. F., Silva, R. L.
E., Khan, M., … Campochiaro, P. A. (2019). AAV8-vectored
suprachoroidal gene transfer produces widespread ocular transgene
expression. Journal of Clinical Investigation. doi:
10.1172/jci129085
Contacts:
For REGENXBIO
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
For CLEARSIDE
Jenny Kobin
Remy Bernarda
ir@clearsidebio.com
(678) 430-8206
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SOURCE REGENXBIO Inc.; Clearside Biomedical, Inc.