ROCKVILLE, Md., April 22, 2020 /PRNewswire/ --
- RGX-314 continues to be well-tolerated at all dose
levels
- Long-term, durable treatment effect demonstrated over two
years in Cohort 3
-
- Mean improvement in vision (+14 letters) and stable retinal
thickness (+2 µm)
- 50% of patients (3/6) remain anti-VEGF injection-free over
two years
- 67% of patients (4/6) are anti-VEGF injection-free from nine
months to two years
- Stable intraocular RGX-314 protein expression over two
years
- 73% of patients (8/11) in Cohort 5 remain anti-VEGF
injection-free over nine months
- Intraocular RGX-314 protein levels at six months demonstrate
dose-dependent expression across cohorts
- Company on track to provide one-year data from Cohorts 4
& 5 in mid-2020 and initiate RGX-314 subretinal delivery
pivotal program in 2H 2020
- Webcast conference call to be hosted Wednesday, April 22 at 8:30 a.m. ET featuring wet AMD Key Opinion
Leaders, Allen C. Ho, M.D.,
Robert Avery, M.D., and Peter Campochiaro, M.D.
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, today provided additional
long-term data from the ongoing Phase I/IIa trial of RGX-314 for
the treatment of wet age-related macular degeneration (wet
AMD).
"I am impressed by the overall outcomes in patients after a
one-time administration of RGX-314. I believe that RGX-314 is the
leading gene therapy program for a major retinal disease such as
wet AMD and could be an important potential one-time treatment
option for AMD patients who require frequent and burdensome
anti-VEGF injections. Real-world evidence demonstrates that
patients lose vision over time with our current standard of care
and incur significant treatment burden with frequent clinic visits
and injections," said Allen C. Ho,
M.D., Director of Retina Research at Wills Eye Hospital and Mid
Atlantic Retina and investigator surgeon in the RGX-314 trial.
"The clinical profile of RGX-314 appears very promising as a
one-time treatment strategy for wet AMD as we continue to learn
about the consistent and durable effects of our anti-VEGF gene
therapy," added Steve Pakola, M.D.,
Chief Medical Officer of REGENXBIO. "We are encouraged that
all patients in Cohort 5 who were anti-VEGF injection-free at six
months remained anti-VEGF injection-free at nine months. We also
have further evidence of dose-dependent intraocular protein levels
in this Phase I/IIa study. We are on track to have one-year data
from our later cohorts in mid-2020 and look forward to initiating a
pivotal program for the subretinal delivery of RGX-314 in the
second half of 2020. We are also continuing our preparations to
initiate a Phase II trial for the in-office suprachoroidal delivery
of RGX-314 in wet AMD patients in the first half of this year."
Study Design and Safety
In the Phase I/IIa trial of RGX-314, 42 patients with severe wet
AMD requiring frequent anti-vascular endothelial growth factor
(anti-VEGF) injections were treated across five dose cohorts, with
doses ranging from 3x109 GC/eye to 2.5x1011
GC/eye. Patients were enrolled into all dose cohorts independent of
their neutralizing antibody titers to AAV and did not receive
prophylactic immune suppressive oral corticosteroid therapy before
or after administration of RGX-314.
Patients in the study are being assessed each month for 24
months and will receive safety follow-up for five years after
RGX-314 administration. Efficacy assessments for the study include
reduction in anti-VEGF intravitreal injections, change in vision as
measured by Best Corrected Visual Acuity (BCVA), change in central
retinal thickness (CRT) as measured by spectral domain optical
coherence tomography (SD-OCT), and protein expression levels as
measured from aqueous samples by electrochemiluminescence
immunoassay (ECL).
As of April 6, 2020, RGX-314
continued to be well-tolerated across all cohorts, with no
drug-related serious adverse events (SAEs) reported. Sixteen SAEs
that were not related to RGX-314, including two ocular
procedure-related SAEs, were reported in ten patients. There have
been no reports of clinically-determined immune responses,
drug-related ocular inflammation, or post-surgical inflammation
beyond what is expected following routine vitrectomy.
Across all 42 patients in the study, the most common nonserious
adverse events in the study eye were generally assessed as mild
(90%). These included post-operative conjunctival hemorrhage (69%
of patients), post-operative inflammation (36% of patients), eye
irritation (17% of patients), eye pain (17% of patients), and
post-operative visual acuity reduction (17% of patients). In 67% of
patients across all cohorts, and in 83% of patients in Cohorts 3-5,
mild to moderate retinal pigmentary changes were observed on
imaging, the majority of which were in the peripheral inferior
retina. There was no evidence of clinical symptoms or changes to
visual acuity related to retinal pigmentary changes. Retinal
hemorrhage was observed in 17% of patients and is an anticipated
event in patients with severe wet AMD.
Summary of Long-term Data for Cohort 3 Over Two Years
Positive long-term potential efficacy signals were sustained
over two years in Cohort 3.The mean change in visual acuity across
all six patients in Cohort 3 was markedly improved over two years,
with a mean BCVA improvement of +14 letters, and the mean change in
CRT was stable, with an increase of +2 µm.
Patients in Cohort 3 also demonstrated long-term reductions in
anti-VEGF treatment burden over two years with a mean annualized
rate of 2.8 anti-VEGF injections after administration of RGX-314,
which is a reduction of over 60% from the mean annualized injection
rate during the twelve months prior to administration of RGX-314.
Three out of six (50%) patients received no anti-VEGF injections
over two years following one-time administration of RGX-314. One
patient received four anti-VEGF injections after RGX-314
administration and then did not receive anti-VEGF injections from
nine months through two years.
The four patients who did not receive anti-VEGF injections after
nine months demonstrated a mean BCVA improvement of +14 letters,
with a range of +6 letters to +25 letters. In addition, these
patients had stable retinal thickness with a mean change of +9
µm.
Additionally, long-term intraocular RGX-314 protein expression
was stable in patients in Cohort 3 over two years. The mean RGX-314
protein expression level in Cohort 3 was 227.2 ng/ml at two years,
compared to 217.8 ng/ml at six months. The mean RGX-314 protein
expression level in the four patients who did not receive anti-VEGF
injections after nine months was 291.7 ng/ml at two years, compared
to 273.6 ng/ml at six months.
Summary of Data for Cohorts 4 and 5
Consistent with previous results, intraocular RGX-314 protein
expression levels increased in a dose-dependent manner across
cohorts when measured at six months after administration of
RGX-314; the mean protein expression level in Cohort 4 and Cohort 5
was 653.6 ng/ml and 848.7 ng/ml, respectively.
Patients in Cohort 5 continued to demonstrate a meaningful
reduction in anti-VEGF treatment burden over nine months following
administration of RGX-314, with 8/11 (73%) patients remaining
anti-VEGF injection-free, and a reduction across the cohort of over
80% from the mean annualized injection rate during the 12 months
prior to RGX-314 administration.
Conference Call
In connection with this announcement, REGENXBIO will host a
webcast and conference call with accompanying slides today at
8:30 a.m. ET. This event will feature
study investigators Allen C. Ho,
M.D., Wills Eye Hospital and Mid Atlantic Retina, Robert Avery, M.D., Founder of California Retina
Consultants and Research Foundation, and Peter Campochiaro, M.D., Johns Hopkins Wilmer
Eye Institute.
To access a live or recorded webcast of the call and
accompanying slides, please visit the "Investors" section of the
REGENXBIO website at www.regenxbio.com. To access the live webcast
by phone, dial (855) 422-8964 (domestic) or (210) 229-8819
(international) and enter the passcode 6668158. The recorded
webcast will be available for approximately 30 days following the
call.
About RGX-314
RGX-314 is being developed as a potential one-time treatment for
wet AMD, diabetic retinopathy, and other additional chronic retinal
conditions treated with anti-VEGF. RGX-314 consists of the NAV AAV8
vector encoding an antibody fragment which is designed to inhibit
VEGF, modifying the pathway for formation of new leaky blood
vessels which lead to retinal fluid accumulation and vision
loss.
About the Phase I/IIa Clinical Trial of RGX-314
RGX‑314 is being evaluated in a Phase I/IIa, multi-center,
open-label, multiple-cohort, dose‑escalation study in adult
patients with wet AMD in the United
States. The study includes patients previously treated for
wet AMD who are responsive to anti-VEGF therapy. The study is
designed to evaluate five escalating doses of RGX-314, with six
patients in the first three dose cohorts and 12 patients in the
fourth and fifth dose cohorts. Patients were enrolled into all dose
cohorts independent of their neutralizing antibody titers to AAV
and did not receive prophylactic immune suppressive oral
corticosteroid therapy before or after administration of RGX-314.
The primary endpoint of the study is safety at 6 months following
administration of RGX-314. Secondary endpoints include visual
acuity, retinal thickness on SD‑OCT, ocular RGX-314 protein
expression, and the need for additional anti-VEGF therapy.
Following completion of the primary study period, patients enter a
follow-up period and will continue to be assessed until week 106
for long-term safety and durability of effect.
About Wet AMD
Wet AMD is characterized by loss of vision due to new, leaky
blood vessel formation in the retina. 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 alone. 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 patients.
These therapies, however, require life-long intraocular injections,
typically repeated every four to 12 weeks in frequency, to maintain
efficacy. Due to the burden of treatment, patients often
experience a decline in vision with reduced frequency of treatment
over time.
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.
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 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 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, the impact of the COVID-19 pandemic or similar public
health crises on REGENXBIO's business, 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, 2019, 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.
Contacts:
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
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