ROCKVILLE, Md., Jan. 9, 2020 /PRNewswire/ --
- RGX-314 programs for treatment of wet AMD and diabetic
retinopathy continue to advance
-
- Plans to initiate a pivotal program of subretinal delivery for
wet AMD in 2H 2020, following the 12-month assessment of Cohort 5
patients in the Phase I/IIa trial
- FDA removes partial clinical hold on Phase I/IIa trial of
subretinal delivery for wet AMD
- Phase II trials of suprachoroidal delivery for treatment of wet
AMD and diabetic retinopathy expected to begin in 2020
- Enrollment continues in Cohort 2 of RGX-121 Phase I/II trial in
MPS II, with additional interim data expected in 2020
- Interim update from RGX-501 Phase I/II trial in HoFH supports
Cohort 2 safety with steroid prophylaxis; LDL-C measures expected
in 1H 2020
- Utilization of new corporate, research and manufacturing
headquarters expected to begin in late 2020, and cGMP manufacturing
facility with capacity to produce NAV vectors at scales up to 2,000
liters expected to be fully operational in 2021
- Ended 2019 above guidance estimate with $400 million in cash, cash equivalents and
marketable securities
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 a year-end 2019 corporate
update and anticipated milestones for 2020.
"2019 was an important year for the advancement and broadening
of our internal gene therapy programs using NAV vectors for
AAV-mediated antibody delivery and monogenic gene replacement.
Importantly, we now have extensive clinical data from our RGX-314
program, which supports further development of our promising
ophthalmology platform, with additional studies planned in 2020 to
investigate subretinal and suprachoroidal approaches to treating
wet AMD and diabetic retinopathy. Our key objective is to make
RGX-314 available to patients as quickly as possible," said
Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "We also recently announced
interim data from our RGX-121 program for the treatment of MPS II,
which showed encouraging signs of enzyme activity in the central
nervous system, and we began construction of our new headquarters,
which will include additional cGMP manufacturing facilities to
support late-stage development and commercial scale needs."
"Our strategic partnerships with other leading gene therapy
developers continue to validate our NAV Technology Platform," Mr.
Mills continued. "Zolgensma® became the first FDA
approved gene therapy based on our proprietary NAV technology last
year, a significant milestone, and other strategic partners
continue to advance their clinical programs into later stages. We
have a strong team and resources in place to drive our business
forward to help realize the transformative potential that gene
therapy holds for patients."
Product Candidate Updates
Gene Therapy Using NAV Vectors for AAV-Mediated Antibody
Delivery:
- RGX-314 for the Treatment of Wet AMD
-
- REGENXBIO expects to initiate a pivotal program for the
subretinal delivery of RGX-314 for the treatment of wet AMD in the
second half of 2020.
-
- A pivotal clinical trial is expected to investigate the
one-time administration of RGX-314 at a single dose compared to
anti-VEGF injections, which is the current standard of care for
patients with wet AMD. The primary efficacy endpoint in the trial
will be the mean change in visual acuity from baseline assessed at
12 months after treatment with RGX-314.
- REGENXBIO plans to finalize the design of the trial based on
the 12-month assessment of patients in Cohort 5 in the Phase I/IIa
trial. This will allow for further characterization of
RGX-314-treated patients, enhancements of the trial design and the
potential acceleration of the clinical program.
- REGENXBIO intends to submit the design of the trial to the FDA
in mid-2020 and begin dosing patients in the second half of
2020.
- The U.S. Food and Drug Administration (FDA) has removed the
partial clinical hold on the Investigational New Drug (IND)
application for the Phase I/IIa trial of the subretinal delivery of
RGX-314 without modification to the surgical delivery system, after
reviewing information provided by the Company. The partial clinical
hold was not related to the gene therapy candidate.
- As of December 31, 2019, RGX-314
continued to be well-tolerated across all cohorts in the Phase
I/IIa trial, with no drug-related serious adverse events (SAEs)
reported. All patients in Cohort 5 (2.5 x 1011 GC/eye) have now
completed their 6-month follow-up.1
-
- Patients in Cohort 5 continue to demonstrate a meaningful
reduction in anti-vascular endothelial growth factor (anti-VEGF)
treatment burden at 6 months following administration of RGX-314,
with 8 out of 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
administration of RGX-314. Importantly, the Cohort 5 patients
continued to demonstrate a mean improvement in vision of +3 ETDRS
letters and mean improvement in retinal thickness of -83 microns,
while the 8 patients who were anti-VEGF injection-free after
administration of RGX-314 showed a mean improvement in vision of +5
ETDRS letters and mean improvement in retinal thickness of -83
microns.
- REGENXBIO plans to initiate the Phase II trial of the
suprachoroidal delivery of RGX-314 using the SCS Microinjector™ for
the treatment of wet AMD in the first half of 2020.
-
- The trial will build upon data from the Phase I/IIa trial of
RGX-314 and is expected to evaluate patients in two dose cohorts of
RGX-314 versus a control arm. Interim data is expected from Cohort
1 by the end of 2020.
- RGX-314 for the Treatment of Diabetic Retinopathy (DR)
-
- REGENXBIO expects to submit an IND in the first half of 2020
and plans to initiate a Phase II trial of the suprachoroidal
delivery of RGX-314 using the SCS Microinjector for the treatment
of DR in the second half of 2020.
-
- The trial is expected to evaluate patients in up to three dose
cohorts of RGX-314 versus a control arm. Enrollment of Cohort 1 is
expected to be complete by the end of 2020, with interim data
expected in 2021.
- Research Program for the Treatment of Hereditary Angioedema
(HAE)
-
- As first announced in July 2019,
REGENXBIO is developing a one-time gene therapy candidate to
deliver a gene encoding a therapeutic antibody against plasma
kallikrein, a key protein of the plasma contact pathway which is
left unregulated in patients with HAE.
- Preclinical animal studies conducted using NAV AAV8 indicate
the potential for a sustained and safe delivery of biologically
active antibody at therapeutic concentrations. REGENXBIO expects to
select a lead product candidate in the first half of 2020 and
provide a program update in the second half of 2020.
- Research Program for the Treatment of Neurodegenerative
Diseases
-
- REGENXBIO has expanded its exclusive collaboration program with
Neurimmune AG to include therapies targeting alpha synuclein, in
addition to tauopathies. The program uses Neurimmune's Reverse
Translational Medicine™ platform along with REGENXBIO's NAV
Technology Platform to design and develop vectorized antibody
therapies for neurodegenerative diseases. REGENXBIO expects to
provide a program update in the second half of 2020.
Gene Therapy Using NAV Vectors for Rare Genetic
Diseases:
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS
II)
-
- As previously reported, as of December
16, 2019, RGX-121 was well-tolerated in Cohort 1 of the
Phase I/II trial of RGX-121 delivered directly to the central
nervous system (CNS), and no drug-related SAEs were reported.
Patients in Cohort 1 demonstrated consistent and sustained
reduction in heparan sulfate (HS) in the cerebral spinal fluid
(CSF) and early signs of neurocognitive stability. HS is a key
biomarker of iduronate-2-sulfatase enzyme activity and high amounts
of HS accumulate in the CNS of MPS II patients, which closely
correlates with neurocognitive decline. Additional data from Cohort
1 are expected to be presented at an upcoming medical conference in
early 2020.
- Dosing in Cohort 2 has begun, at an increased dose level.
REGENXBIO expects to complete enrollment of Cohort 2 in the first
half of 2020 and provide interim data in mid-2020.
- REGENXBIO expects to provide additional details for a
potentially accelerated program pathway following evaluation of
interim data from Cohort 2 in the second half of 2020 and
subsequent interactions with the FDA, in accordance with the Fast
Track designation granted to RGX-121 in 2018.
- RGX-501 for the Treatment of Homozygous Familial
Hypercholesterolemia (HoFH)
-
- REGENXBIO previously announced the completed dosing of an
expanded Cohort 2 in the Phase I/II trial of RGX-501 at a dose of
7.5x1012 GC/kg and including steroid prophylaxis. Per
protocol, patients received at least a 13-week steroid
treatment.
- As of December 31, 2019, the
three patients in the expanded Cohort 2 have been followed for an
average of 17 weeks following administration of RGX-501, all beyond
the 3-6 week window during which previous transaminase elevations
have occurred at this dose level. No SAEs or significant elevations
in liver enzyme levels were reported in the expanded Cohort 2 and
all patients have completed their steroid treatment or initiated
the taper from steroid treatment.
- REGENXBIO plans to assess low-density lipoprotein (LDL-C)
levels in the expanded Cohort 2 after all patients have completed
their steroid treatment, and expects to provide interim data in the
first half of 2020.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS
I)
-
- Recruitment, screening and additional site activations are
ongoing in the Phase I clinical trial evaluating RGX-111 for the
treatment of MPS I. In November 2019,
REGENXBIO announced that RGX-111 was administered to a patient with
MPS I through an investigator-initiated study. REGENXBIO expects to
provide a program update in the second half of 2020.
- RGX-181 for the Treatment of Late-infantile Neuronal Ceroid
Lipofuscinosis Type 2 (CLN2) Disease
-
- REGENXBIO is conducting ongoing preclinical development of
RGX-181, including assessment of unmet clinical needs such as
neurologic and ophthalmologic manifestations of the disease.
REGENXBIO expects to provide a program update in mid-2020 and
submit an IND for a first-in-human trial in the second half of
2020.
Anticipated 2020 Milestones
REGENXBIO expects to meet the following milestones related to
the development of internal product candidates in 2020:
Gene Therapy Using NAV Vectors for AAV-Mediated Antibody
Delivery:
- RGX-314 for the Treatment of Wet AMD
-
- Subretinal delivery
-
- Complete 12-month assessments of Cohort 5 in the Phase I/IIa
trial in the first half of 2020.
- Initiate a pivotal trial in the second half of 2020.
- Suprachoroidal delivery
-
- Initiate Phase II trial in the first half of 2020.
- Report interim data from Cohort 1 of the Phase II trial in the
second half of 2020.
- RGX-314 for the Treatment of DR
-
- Submit IND for a Phase II trial of suprachoroidal delivery in
the first half of 2020.
- Initiate Phase II trial of suprachoroidal delivery in the
second half of 2020.
- Research Program for Treatment of HAE
-
- Select lead product candidate in first half of 2020.
- Provide program update in the second half of 2020.
- Research Program for Treatment of Neurodegenerative
Diseases
-
- Provide program update in the second half of 2020.
Gene Therapy Using NAV Vectors for Rare Genetic
Diseases:
- RGX-121 for the Treatment of MPS II
-
- Present additional interim data from Cohort 1 of the Phase I/II
trial in the early 2020.
- Provide interim data from Cohort 2 of the Phase I/II trial in
mid-2020.
- Provide program update in the second half of 2020.
- RGX-501 for the Treatment of Homozygous Familial
Hypercholesterolemia (HoFH)
-
- Present data from secondary endpoints of the expanded Cohort 2
of the Phase I/II trial, including changes in LDL-C, in the first
half of 2020.
- Provide program update in the first half of 2020.
- RGX-111 for the Treatment of MPS I
-
- Provide program update in the second half of 2020.
- RGX-181 for the Treatment of CLN2 Disease
-
- Provide program update in mid-2020.
- Submit IND for a first-in-human trial in the second half of
2020.
Operational Updates and Anticipated Milestones in
2020
- Current Good Manufacturing Practice (cGMP) Manufacturing
Facility
-
- Construction of a new corporate, research and manufacturing
headquarters in Rockville,
Maryland, continues, with plans to begin utilizing the new
headquarters in late 2020.
- The new cGMP production facility is expected to allow for
production of NAV vectors at scales up to 2,000 liters using
REGENXBIO's platform suspension cell culture process, which will
complement REGENXBIO's current external manufacturing network and
capabilities. The cGMP facility is expected to be fully operational
starting in 2021.
NAV Technology Licensee Program Highlights
As of December 31, 2019,
REGENXBIO's NAV Technology Platform was being applied in one
marketed product and more than 20 partnered product candidates in
development. Fifteen of these partnered product candidates are in
active clinical development. REGENXBIO's NAV Technology Licensees
are advancing product candidates in a broad range of therapeutic
areas and disease indications. Recent updates from NAV Technology
Licensees include:
Marketed NAV Technology Products
- Novartis AG's Zolgensma for the Treatment of Spinal Muscular
Atrophy (SMA)
-
- REGENXBIO receives tiered royalties on the worldwide sales of
Zolgensma up to a low double-digit percentage and is eligible to
receive an additional $80 million
milestone payment upon the achievement of $1
billion in cumulative net sales. As of October 22, 2019, Novartis reported U.S.
Zolgensma sales revenue of $175
million through the third quarter of 2019.
- Novartis announced in October
2019 that Zolgensma is currently under regulatory review in
Europe with an anticipated
regulatory decision in the first quarter of 2020 and in
Japan with an anticipated
regulatory decision in the first half 2020.
Late-stage NAV Technology Clinical Programs
- Audentes Therapeutics, Inc.'s AT132 for the Treatment of
X-Linked Myotubular Myopathy (XLMTM)
-
- In November 2019, Audentes stated
that the Biologics License Application submission for AT132 is
planned for mid-2020, and the Marketing Authorization Application
submission is planned for the second half of 2020. AT132 uses the
NAV AAV8 vector.
Financial Guidance
As of December 31, 2019, REGENXBIO had
$400 million in cash, cash
equivalents and marketable securities. REGENXBIO expects
these resources to fund the completion of its internal
manufacturing capabilities and clinical advancement of its product
candidates into 2022.
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,
research and development activities, preclinical studies, clinical
trials, costs 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.
Zolgensma® is a registered trademark of AveXis. All
other trademarks referenced herein are registered trademarks of
REGENXBIO.
1 One patient died 4.5 months after the
administration of RGX-314 as a result of the subject's underlying
disease, which was assessed to be unrelated to RGX-314. At the time
of the death, the subject was free of anti-VEGF injections.
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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