- Company announced positive interim data from and the
expansion of Phase II AAVIATE® trial of RGX-314 for the
treatment of wet AMD using suprachoroidal delivery
-
-
- RGX-314 continues to be well tolerated in 85 patients from
Cohorts 1-5 with no drug-related serious adverse events
- Meaningful reduction in treatment burden at six months
across all dose levels, 85% reduction in treatment burden observed
at third dose level
- 67% of patients in Cohort 4 were injection-free
- No meaningful differences in outcomes at six months for
patients who are NAb positive
- Phase II trial expanded to include new cohort at third dose
level with short-course prophylactic ocular steroids following
RGX-314 administration
- Positive interim data presented at the AAO Annual Meeting
from the Phase I/IIa Long-term Follow-up study of RGX-314 for the
treatment of wet AMD using subretinal delivery
-
-
- RGX-314 continues to be well-tolerated and demonstrates
long-term, durable treatment effect up to four years
- Two pivotal trials, ATMOSPHERE® and
ASCENT™, are active and enrolling patients
- Pivotal trials are expected to support BLA submission in
2024
- New interim data from Phase II ALTITUDE® trial of
RGX-314 for the treatment of diabetic retinopathy using
suprachoroidal delivery expected at the Retina Society
55th Annual Scientific Meeting in November
- Conference call Monday, October
3 at 8:30 a.m. ET
ROCKVILLE, Md, Oct. 3, 2022
(PRNewswire) – REGENXBIO Inc. (Nasdaq: RGNX) today announced a
program update from its ongoing clinical investigation of RGX-314
for the treatment of wet AMD, a leading cause of vision loss
globally.
REGENXBIO announced positive interim data from the Phase II
AAVIATE® trial of RGX-314 for the treatment of wet AMD
using suprachoroidal delivery. REGENXBIO also presented positive
interim data from the Phase I/IIa long-term follow-up (LTFU) study
of RGX-314 for the treatment of wet AMD using subretinal delivery
at the American Academy of Ophthalmology (AAO) annual meeting this
past weekend in Chicago.
"Today's announcements come nearly a year into our collaboration
with AbbVie to advance RGX-314 in wet AMD and other retinal
diseases. These new subretinal and suprachoroidal data highlight
the potential impact of RGX-314 for the millions of patients facing
vision loss from wet AMD," said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO.
"I am pleased with the continued progress on and momentum for these
programs. We remain on track to submit a BLA for RGX-314 in 2024,
and we expect to provide an update on the ALTITUDE trial of RGX-314
for the treatment of diabetic retinopathy using suprachoroidal
delivery at the upcoming Retina Society Meeting in November."
Data Summary and Safety Update for the Phase II AAVIATE Trial
of RGX-314 using Suprachoroidal Delivery
The Phase
II AAVIATE trial of RGX-314 for the treatment of
wet AMD using in-office suprachoroidal
delivery continues to show positive interim results.
AAVIATE is a multi-center, open-label, randomized,
active-controlled, dose-escalation trial that is evaluating the
efficacy, safety and tolerability of suprachoroidal
delivery of RGX-314. The primary endpoint of the trial is
mean change in vision in patients dosed with RGX-314, as measured
by best corrected visual acuity (BCVA) at Week 40 from
baseline, compared to patients receiving monthly injections
of ranibizumab. Other endpoints include mean change in
central retinal thickness (CRT) and number of anti-vascular
endothelial growth factor (anti-VEGF)
intravitreal injections received following administration of
RGX-314.
As of August 1, 2022, RGX-314
suprachoroidal delivery was reported to be well tolerated across 85
patients dosed in Cohorts 1-5. Fifteen SAEs were reported, none of
which were considered related to RGX-314. For the total group of
Cohorts 1-4 (n=65), all common treatment emergent adverse events
(TEAEs) through 6 months in the study eye were mild or moderate and
included conjunctival hemorrhage, increased intraocular pressure,
episcleritis, and conjunctival hyperemia. Mild intraocular
inflammation was reported at similar incidence in the first and
second dose levels, with an increase in incidence in mild to
moderate inflammation seen at the third dose level (Cohort 4). All
intraocular inflammation resolved with topical corticosteroids.
Patients treated in the RGX-314 arms and the ranibizumab control
arm both continue to demonstrate stable BCVA and CRT at 6 months.
In addition, a meaningful reduction in anti-VEGF treatment burden
following administration of RGX-314 compared to mean annualized
injection rate during the 12 months prior to administration was
observed and ranged from -63.8% to -84.7% across all cohorts. The
highest reduction in treatment burden was observed in the third
dose level, with patients receiving a mean of 1.3 injections over
six months following administration of RGX-314, which represents an
84.7% reduction in anti-VEGF treatment burden. Ten out of 15
patients (67%) in the third dose level received no anti-VEGF
injections over six months following RGX-314 administration. In
these patients, visual acuity and CRT was observed to be stable
over six months.
Additionally, the interim data from the second dose level
(Cohorts 2 and 3) suggests there is no meaningful difference in
safety and vision outcomes for patients who are neutralizing
antibody (NAb) positive.
Phase II AAVIATE Trial Expansion to Cohort 6
REGENXBIO
announced today that the AAVIATE study has expanded, and an
additional cohort (Cohort 6) will be enrolled to evaluate RGX-314
at the third dose level of 1x1012 GC/eye with a
short course of prophylactic ocular steroids to evaluate the
ability to prevent or reduce the occurrence of the mild to moderate
intraocular inflammation seen to date. Patients will be enrolled in
Cohort 6 regardless of NAb status.
"We are pleased to share updated data from the AAVIATE trial,
including new 6-month data from Cohorts 1-4 which provides
continued evidence of the emerging clinical profile of RGX-314 for
the treatment of wet AMD using suprachoroidal delivery," said
Steve Pakola, M.D., Chief Medical
Officer of REGENXBIO. "RGX-314 continues to be well tolerated, with
emerging evidence of treatment effect, including meaningful
reduction in anti-VEGF treatment burden at all dose levels. We look
forward to expanding this trial to further explore the third dose
level."
Data Summary from Phase I/IIa Long-term Follow-up Study of
RGX-314 using Subretinal Delivery
The Phase I/IIa study
evaluated RGX-314 in 42 patients with wet AMD using subretinal
delivery and was designed as an open label, dose escalation study
evaluating five doses of RGX-314 over two years. Dose dependent
increases in treatment effect were observed, and doses similar to
those used in Cohort 3 and Cohort 4 of the Phase I/IIa trial were
advanced into the ongoing pivotal trials, ATMOSPHERE and ASCENT.
After the Phase I/IIa study's completion, patients were encouraged
to enroll into a long-term follow-up study for up to five years
after RGX-314 administration. Data presented at AAO highlights the
results of the 16 (out of 18) patients from Cohorts 3 and 4 who
enrolled in the LTFU study, with data now out to 4 years and 3
years, respectively. Data from this study demonstrating that
RGX-314 continues to be well-tolerated with long-term, durable
treatment effect up to four years is expected to support the
anticipated BLA filing for RGX-314 in 2024.
As of August 29, 2022, RGX-314
continues to be generally well-tolerated in the long-term follow-up
study (n=37). A total of nine serious adverse events (SAEs) were
reported in four patients, none of which were considered related to
RGX-314. No new drug-related ocular AEs were reported in the
long-term follow-up study for Cohorts 3 and 4. One patient in
Cohort 5 experienced a significant decrease of vision during the
long-term follow-up study who had macular pigmentary changes after
a superior bleb in the Phase I/IIa trial.
Patients treated with RGX-314 continue to demonstrate a
long-term, durable treatment effect in Cohort 3 up to 4 years and
Cohort 4 up to three years. Stable to improved visual acuity was
observed, with a mean BCVA of +12 letters from baseline at four
years for Cohort 3 patients and -5 letters from baseline at three
years for Cohort 4 patients following RGX-314 administration.
Patients also demonstrated meaningful long-term reductions in
anti-VEGF treatment burden following administration of RGX-314.
Patients in Cohort 3 received a mean annualized rate of 2.4
injections through 4 years following administration of RGX-314
(versus 6.8 injections in the 12 months prior to treatment),
representing a 67.0% reduction in mean annualized injection rate.
Patients in Cohort 4 received a mean annualized rate of 4.4
injections through 3 years following administration of RGX-314
(versus 10.2 injections in the 12 months prior to treatment),
representing a 58.4% reduction in mean annualized injection
rate.
"These positive interim data from the long-term follow-up and
AAVIATE trials continue to reinforce the potential clinical
benefit of a one-time administration of RGX-314 in the overall
management of patients with neovascular AMD," said Arshad M. Khanani, M.D., M.A., FASRS, Director
of Clinical Research at Sierra Eye Associates, Reno, NV. "I am extremely encouraged by this
long-term data up to four years showing durable treatment effect
and the potential of RGX-314 to meaningfully reduce injection
burden for patients while maintaining vision outcomes. I look
forward to the further investigation of RGX-314 in Cohort 6 of the
AAVIATE trial, as in-office suprachoroidal delivery has the
potential to be an important treatment option for patients."
These study findings are available under the Presentations &
Publications page in the Media section of REGENXBIO's website
located at www.regenxbio.com.
Conference Call
In connection with this announcement,
REGENXBIO will host a conference call and webcast today at
8:30 a.m. ET to discuss the new,
interim update from the ongoing Phase II AAVIATE® trial
of RGX-314 for the treatment of wet AMD using suprachoroidal
delivery. Listeners can register for the webcast via this link.
Analysts wishing to participate in the question and answer session
should use this link. A replay of the webcast will be available via
the Company's investor website approximately two hours after the
call's conclusion. Those who plan on participating are advised to
join 15 minutes prior to the start time.
About the AAVIATE® Trial
The multi-center,
open-label, randomized, active-controlled, dose-escalation Phase II
AAVIATE trial is evaluating the efficacy, safety and tolerability
of suprachoroidal delivery of RGX-314 in patients with wet AMD
using the Clearside SCS Microinjector®. Twenty patients
in Cohort 1 were randomized to receive RGX-314 at a dose level of
2.5x1011 genomic copies per eye (GC/eye) through one
injection versus monthly 0.5 mg ranibizumab intravitreal injection
at a 3:1 ratio. Twenty patients in Cohort 2 were randomized to
receive RGX-314 at a dose level of 5x1011 GC/eye through
two injections versus monthly 0.5 mg ranibizumab intravitreal
injection at a 3:1 ratio. Cohort 3 is evaluating RGX-314 at the
same dose level as Cohort 2 in 20 patients who are NAb positive.
Cohort 4 is evaluating RGX-314 in 15 patients at a dose level of
1x1012 GC/eye and Cohort 5 is evaluating the same dose
level of RGX-314 in 20 patients who are NAb positive. Cohort 6 is
evaluating patients at the same dose level as Cohorts 4 and 5 and
includes a short course of prophylactic steroids following
administration of RGX-314.
About RGX-314
RGX-314, being developed in
collaboration with AbbVie, 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
retina.
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 eye.
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 repeated intraocular
injections to maintain efficacy. Due to the burden of treatment, it
is difficult for patients to adhere to frequent injections, which
can lead to a decline in vision 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, including late-stage and commercial
programs, in multiple therapeutic areas. REGENXBIO is committed to
a "5x'25" strategy to progress five AAV Therapeutics from our
internal pipeline and licensed programs into pivotal-stage or
commercial products by 2025.
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," "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 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,
2021, 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 or otherwise.
SCS Microinjector® is a trademark of Clearside
Biomedical, Inc. All other trademarks referenced herein are
registered trademarks of REGENXBIO.
Contacts:
Dana
Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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