NORTH CHICAGO, Ill. and
ROCKVILLE, Md., Sept. 13, 2021 /PRNewswire/ -- AbbVie (NYSE:
ABBV) and REGENXBIO Inc. (Nasdaq: RGNX) today announced a
partnership to develop and commercialize RGX-314, a potential
one-time gene therapy for the treatment of wet age-related macular
degeneration (wet AMD), diabetic retinopathy (DR) and other chronic
retinal diseases. RGX-314 is currently being evaluated in patients
with wet AMD in a pivotal trial utilizing subretinal delivery, and
in patients with wet AMD and DR in two separate Phase II clinical
trials utilizing in-office suprachoroidal delivery.
Under the collaboration, REGENXBIO will be responsible for
completion of the ongoing trials of RGX-314. AbbVie and REGENXBIO
will collaborate and share costs on additional trials of RGX-314,
including the planned second pivotal trial evaluating subretinal
delivery for the treatment of wet AMD and future trials. AbbVie
will lead the clinical development and commercialization of RGX-314
globally. REGENXBIO shall participate in U.S. commercialization
efforts as provided under a mutually agreed upon commercialization
"We are committed to finding solutions for patients living with
difficult-to-treat retinal diseases and to helping preserve and
protect our patients from visual impairment and devastating vision
loss," said Tom Hudson, MD, senior
vice president, R&D, chief scientific officer, AbbVie. "In
collaboration with REGENXBIO, we aim to make a remarkable impact
for the millions of patients suffering from vision loss associated
with retinal diseases."
"AbbVie is a strong, complementary partner for REGENXBIO. We
expect to leverage AbbVie's global developmental and commercial
infrastructure within eye care with our expertise in AAV gene
therapy clinical development and deep in-house knowledge of
manufacturing and production to continue the development of
RGX-314," said Kenneth T. Mills,
president and chief executive officer of REGENXBIO.
Under the terms of the agreement, AbbVie will pay REGENXBIO a
$370 million upfront payment with the
potential for REGENXBIO to receive up to $1.38 billion in additional development,
regulatory and commercial milestones. REGENXBIO and AbbVie will
share equally in profits from net sales of RGX-314 in the U.S.
AbbVie will pay REGENXBIO tiered royalties on net sales of RGX-314
outside the U.S. In addition, REGENXBIO will lead the manufacturing
of RGX-314 for clinical development and U.S. commercial supply, and
AbbVie will lead manufacturing of RGX-314 for commercial supply
outside the U.S.
The transaction is expected to close by the end of 2021, subject
to the satisfaction of customary closing conditions, including
applicable regulatory approvals.
REGENXBIO Conference Call
In connection with this
announcement, REGENXBIO will host a webcast and conference call
today at 8:00 a.m. ET. To access a
live or recorded webcast of the call, please visit the "Investors"
section of the REGENXBIO website at www.regenxbio.com. To access
the live call by phone, dial (855) 422-8964 (domestic) or (210)
229-8819 (international) and enter the passcode 6379638. The
recorded webcast will be available for approximately 30 days
following the call.
RGX-314 is being investigated as a
potential one-time treatment for wet AMD, diabetic retinopathy, and
other chronic retinal conditions. RGX-314 consists of the NAV AAV8
vector, which encodes an antibody fragment designed to inhibit
vascular endothelial growth factor (VEGF). RGX-314 is believed to
inhibit the VEGF pathway by which new, leaky blood vessels grow and
contribute to the accumulation of fluid in the
REGENXBIO is advancing research in two separate routes of
administration of RGX-314 to the eye, through a standardized
subretinal delivery procedure as well as delivery to the
suprachoroidal space. REGENXBIO has licensed certain exclusive
rights to the SCS Microinjector® from Clearside Biomedical, Inc. to
deliver gene therapy treatments to the suprachoroidal space of the
About Wet AMD
Wet AMD is characterized by loss of
vision due to new, leaky blood vessel formation in the
retina2. Wet AMD is a significant cause of vision loss
in the United States, Europe and Japan, with up to 2 million people living with
wet AMD in these geographies alone3. Current anti-VEGF
therapies have significantly changed the landscape for treatment of
wet AMD, becoming the standard of care due to their ability to
prevent progression of vision loss in the majority of
patients4. These therapies, however, require life-long
repeated intraocular injections, to maintain
efficacy5,6. Due to the burden of treatment,
patients often experience a decline in vision with reduced
frequency of treatment over time7.
About Diabetic Retinopathy
Diabetic retinopathy (DR)
is the leading cause of vision loss in adults between 24 and 75
years of age worldwide8. DR affects approximately eight
million people in the United
States alone9. The spectrum of DR severity ranges
from non-proliferative diabetic retinopathy (NPDR) to proliferative
diabetic retinopathy (PDR) and as DR progresses, a large proportion
of patients develop vision threatening complications, including
diabetic macular edema (DME) and neovascularization that can lead
to blindness10. Current treatment options for
patients with DR include "watchful waiting", anti-VEGF treatment,
retinal laser or surgical treatment8.
AbbVie's mission is to discover and
deliver innovative medicines that solve serious health issues today
and address the medical challenges of tomorrow. We strive to have a
remarkable impact on people's lives across several key therapeutic
areas: immunology, oncology, neuroscience, eye care, virology,
women's health and gastroenterology, in addition to products and
services across its Allergan Aesthetics portfolio. For more
information about AbbVie, please visit us at www.abbvie.com.
Follow @abbvie on Twitter, Facebook, Instagram,
YouTube and LinkedIn.
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.
AbbVie Forward-Looking Statements
in this news release are, or may be considered, forward-looking
statements for purposes of the Private Securities Litigation Reform
Act of 1995. The words "believe," "expect," "anticipate," "project"
and similar expressions, among others, generally identify
forward-looking statements. AbbVie cautions that these
forward-looking statements are subject to risks and uncertainties
that may cause actual results to differ materially from those
indicated in the forward-looking statements. Such risks and
uncertainties include, but are not limited to, failure to realize
the expected benefits from AbbVie's acquisition of Allergan plc
("Allergan"), failure to promptly and effectively integrate
Allergan's businesses, competition from other products, challenges
to intellectual property, difficulties inherent in the research and
development process, adverse litigation or government action,
changes to laws and regulations applicable to our industry and the
impact of public health outbreaks, epidemics or pandemics, such as
COVID-19. Additional information about the economic, competitive,
governmental, technological and other factors that may affect
AbbVie's operations is set forth in Item 1A, "Risk Factors," of
AbbVie's 2020 Annual Report on Form 10-K, which has been filed with
the Securities and Exchange Commission, as updated by its
subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no
obligation to release publicly any revisions to forward-looking
statements as a result of subsequent events or developments, except
as required by law.
REGENXBIO Forward-Looking Statements
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," "assume," "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 proposed collaboration with AbbVie and
REGENXBIO's future operations and clinical trials. 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 anticipated completion of
REGENXBIO's proposed transaction with AbbVie, the outcome of
REGENXBIO's proposed collaboration with AbbVie 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, 2020 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. Except as required by law,
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
Penn JS, Madan A,
Caldwell RB, et al. Vascular endothelial growth factor in eye
disease. Prog Retin Eye Res. 2008;27(4):331-71.
Angiogenesis in life, disease and medicine. Nature.
Alexandra NM. Wet age related macular degeneration management and
follow-up. Rom J Ophthalmol. 2016;60:9–13.
AAO PPP. Preferred
Practice Patterns: Age related macular degeneration. American
Academy of Ophthalmology. 2019.
Dugel PU, Koh A,
Ogura Y, et al. HAWK and HARRIER: phase 3, multicenter, randomized,
double-masked trials of brolucizumab for neovascular age-related
macular degeneration. Ophthalmology. 2020;127(1):72-84.
Holz FG et al. Br J
Cheung N, Mitchell P,
Wong TY. Diabetic retinopathy. Lancet.
Wittenborn, J.S. and
D.B. Rein. Cost of Vision Problems: The future of vision,
forecasting the prevalence and costs of vision problems. 2014. NORC
at the University of Chicago: Chicago
Alexandra Acabá. Current Management of Diabetic Retinopathy,
SOURCE AbbVie; REGENXBIO