- Continued progress on '5x'25' strategy to advance five AAV
Therapeutics from REGENXBIO's internal pipeline and licensed
programs into pivotal-stage or commercial products by 2025
- ABBV-RGX-314 program for the treatment of wet AMD and
diabetic retinopathy, being developed in collaboration with
AbbVie
-
- Interim results from Phase II AAVIATE® and
ALTITUDE® trials of dose level 3 cohorts with
short-course prophylactic steroid eye drops resulted in zero cases
of intraocular inflammation
- Additional interim efficacy data from the Phase II AAVIATE
and ALTITUDE trials expected at upcoming medical conferences in
second half 2023 and first half 2024
- Interim data from Phase II Bridging Study evaluating
subretinal delivery of ABBV-RGX-314 produced by the
NAVXpress™ platform process demonstrated the anticipated
clinical profile in the low dose cohort: well-tolerated and with
the majority of subjects (11/15, 73%) injection-free through
six months
- Initial safety results from Cohort 1 of the Phase I/II
AFFINITY DUCHENNETM trial of RGX-202
support well-tolerated profile; initial microdystrophin protein
expression levels from Cohort 1 to be presented at World Muscle
Society meeting in October
- Completed enrollment of the Phase III part of CAMPSIITE™
clinical trial of RGX-121 for treatment of MPS II; remains on track
to file BLA in 2024 using the accelerated approval pathway
- $415 million in cash, cash
equivalents and marketable securities as of June 30, 2023; operational runway into
2025
- Conference call Wednesday, August 2,
2023, at 4:30 PM
(EDT)
ROCKVILLE, Md., Aug. 2, 2023
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
financial results for the second quarter ended June 30, 2023, and recent operational
highlights.
"We continue to stay on track advancing our '5x'25' vision
to have five gene therapies either on the market or in late-stage
development by 2025," said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO.
"We are executing against a transformational year for REGENXBIO,
with multiple clinical milestones and updates anticipated in 2023.
The encouraging safety and tolerability data for the high-dose
cohorts of our in-office wet AMD and DR programs further validate
the potential of the suprachoroidal space for AAV delivery to treat
chronic retinal diseases. We successfully reached our goal of
enrolling patients in CAMPSIITE clinical trial of RGX-121 for
treatment of MPS II and reported initial safety results from the
first cohort of the AFFINITY DUCHENNE study and expect to present
initial efficacy data later this year. Overall, we're making
excellent progress advancing our '5x'25' strategy to bring leading,
ground-breaking AAV Therapeutics to patients in need."
PROGRAM HIGHLIGHTS AND MILESTONES
ABBV-RGX-314: ABBV-RGX-314 is an investigational one-time
AAV therapeutic being developed in collaboration with AbbVie for
the treatment of wet age-related macular degeneration (wet AMD),
diabetic retinopathy (DR) and other additional chronic retinal
conditions. ABBV-RGX-314 uses the NAV® AAV8 vector to deliver a
gene encoding a therapeutic antibody fragment to inhibit vascular
endothelial growth factor (VEGF).
ABBV-RGX-314 is currently being evaluated in nine ongoing
clinical trials, including two pivotal trials, a Phase II bridging
study, a Long-term Follow-up study, and a Fellow Eye treatment
study in patients with wet AMD, all utilizing subretinal delivery,
as well as two Phase II clinical trials in patients with wet AMD
and DR, and two corresponding Long-term Follow-up studies, all
utilizing in-office suprachoroidal delivery.
- ABBV-RGX-314 Subretinal Delivery for the Treatment of Wet
AMD
-
- Enrollment is ongoing in ATMOSPHERE® and
ASCENTTM pivotal trials and the Fellow Eye treatment
study for the treatment of patients with wet AMD using subretinal
delivery. Material from REGENXBIO's NAVXpressTM platform
process has been incorporated in these pivotal trials and is
expected to be produced at REGENXBIO's Manufacturing Innovation
Center (RMIC) for future commercialization of ABBV-RGX-314. These
trials are expected to support global regulatory submissions with
the U.S. Food and Drug Agency (FDA) and the European Medicines
Agency (EMA) in late 2025 through the first half of 2026.
- A Phase II pharmacodynamic study designed to evaluate the same
dose levels being used in the two pivotal trials is now fully
enrolled. In July 2023, updated
interim data was presented at the American Society of Retina
Specialists annual meeting, demonstrating that ABBV-RGX-314
manufactured using REGENXBIO's NAVXpress platform process was well
tolerated at both dose levels, initial data in the low dose cohort
through 6 months exhibited expected protein levels along with
stable to improved best corrected visual acuity and central retinal
thickness, as well as meaningful reductions in anti-VEGF burden,
with most subjects (11/15, 73%) remaining injection-free.
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD
and DR
-
- In July 2023, REGENXBIO presented
interim data from the Phase II AAVIATE® &
ALTITUDE® trials demonstrating that ABBV-RGX-314
suprachoroidal delivery administered to patients in cohorts at dose
level 3 (1.0x1012 genome copies per eye) with
short-course (seven-week) prophylactic topical steroid eye drops
(N=39) resulted in zero cases of intraocular inflammation. Time of
post-administration follow up ranged from six weeks to six
months.
- REGENXBIO expects to report additional interim data from the
ALTITUDE trial, including full twelve-month results from Cohorts
1-3, at the American Academy of Ophthalmology meeting (November 3-6, 2023) and from the AAVIATE trial,
including full six-month results from Cohorts 5 and 6, at the
Hawaiian Eye and Retina meeting (January
13-19, 2024).
RGX-202: RGX-202 is an investigational one-time AAV
therapeutic for Duchenne Muscular Dystrophy (Duchenne), using the
NAV AAV8 vector to deliver a transgene for a novel microdystrophin
that includes the functional elements of the C-Terminal domain as
well as a muscle-specific promoter to support a targeted therapy
for improved resistance to muscle damage associated with
Duchenne.
- In July 2023, REGENXBIO reported
that two Duchenne patients have received doses of RGX-202. As of
July 6, 2023, RGX-202 was reported to
be well-tolerated in the patients dosed, aged 4 and 10 years, with
no drug-related serious adverse events. Time of post-administration
follow up was 45 days and more than three months. The Phase I/II
AFFINITY DUCHENNE™ trial continues to recruit patients (aged 4 to
11 years). REGENXBIO is using commercial-scale cGMP material from
the RMIC in the clinical trial.
- REGENXBIO expects to report additional interim data of the
AFFINITY DUCHENNE trial, including longer-term safety and
microdystrophin protein expression levels in muscle at three
months, at the World Muscle Society Congress (October 3-7, 2023).
RGX-121: RGX-121 is an investigational one-time AAV
therapeutic for the treatment of mucopolysaccharidosis Type II (MPS
II), also known as Hunter syndrome, using the NAV AAV9 vector to
deliver the gene that encodes the iduronate-2-sulfatase enzyme.
- Today REGENXBIO announced it has completed enrollment of 10 MPS
II patients in the Phase I/II/III CAMPSIITE™ trial to support a
Biologics License Application filing in 2024 using the accelerated
approval pathway.
- In May 2023, REGENXBIO announced
that the FDA granted regenerative medicine advanced therapy (RMAT)
designation for RGX-121. RMAT designation is designed to expedite
the drug development and review processes for promising new
treatments, including gene therapies, and recognizes that the
preliminary clinical evidence from RGX-121 indicates its potential
to address unmet medical needs for MPS II.
RGX-111: RGX-111 is an investigational one-time AAV
therapeutic for the treatment of severe Mucopolysaccharidosis Type
I (MPS I), also known as Hurler syndrome, using the NAV AAV9 vector
to deliver the α-l-iduronidase gene.
- A Phase I/II trial of RGX-111 for the treatment of MPS I is
fully enrolled with follow-up ongoing.
- REGENXBIO continues to expect to complete analytical
characterization of recently manufactured commercial-scale cGMP
material and share additional updates on program plans by the end
of 2023.
RGX-181: RGX-181 is an investigational one-time
AAV therapeutic for the treatment of late-infantile neuronal ceroid
lipofuscinosis type 2 (CLN2) disease, a form of Batten disease,
using the NAV AAV9 vector to deliver the tripeptidyl peptidase
1 (TPP1) gene directly to the central
nervous system.
- Physician investigators in Brazil continue with follow up for the first
child with CLN2 disease dosed with RGX-181 under a single-patient
investigator-initiated study.
- REGENXBIO expects investigators to report initial interim data
from the single-patient investigator-initiated study, including
6-month results, at the Society for the Study of Inborn Errors of
Metabolism Annual Symposium meeting being held August 29 to September 1, 2023.
RGX-381: RGX-381 is an investigational one-time AAV
Therapeutic for the treatment of the ocular manifestations of CLN2
disease, using the NAV AAV9 vector to deliver the TPP1 gene
directly to the retina.
- Today, REGENXBIO announced dosing of the first patient in the
Phase I/II trial of RGX-381.
- Initial data from this trial is expected to be shared in
2024.
RESEARCH & OPERATIONAL UPDATES
New Exon-Skipping Therapy for Duchenne Muscular Dystrophy
- In July 2023, REGENXBIO announced
the development of a potential one-time gene therapy for Duchenne,
which is based on a novel exon-skipping construct targeting exon 53
of the DMD gene. Company scientists have demonstrated that
AAV-mediated exon skipping by the expression of antisense
ribonucleic acid has the potential to restore high and sustained
levels of near full-length dystrophin in Duchenne patients with
relevant mutations in the DMD gene.
- REGENXBIO is initiating Investigational New Drug (IND)
application-enabling studies and expects to submit an IND to the
FDA in the first half of 2025.
- The RMIC in Maryland is fully
operational, producing cGMP bulk substance lots to support programs
using the NAVXpress platform process. REGENXBIO is one of only a
few gene therapy companies worldwide with a cGMP facility capable
of production at scales up to 2,000 liters. The company continues
to use the facility to produce commercial-scale cGMP material for
its entire clinical pipeline and performance qualification lots to
support planned regulatory filings for ABBV-RGX-314 and
RGX-121.
NAV TECHNOLOGY PLATFORM LICENSEE PROGRAM HIGHLIGHTS
As of June 30, 2023, REGENXBIO's
NAV Technology Platform was being applied in one marketed product
and multiple clinical-stage partnered programs, with the potential
to impact a broad range of therapeutic areas and disease
indications.
- Zolgensma®, a one-time AAV Therapeutic for the
treatment of spinal muscular atrophy, is a marketed product
utilizing REGENXBIO's NAV AAV9 vector. In July 2023, Novartis AG reported second quarter
2023 global sales of Zolgensma of $311
million.
- In May 2023, Rocket
Pharmaceuticals announced that the EMA has granted Priority
Medicines designation to RP-A501 for the treatment of Danon
Disease. RP-A501 is being developed as a one-time gene therapy
utilizing REGENXBIO's NAV AAV9 vector.
- In May 2023, Ultragenyx
Pharmaceutical Inc. announced the completion of dosing in the Phase
III study of DTX401 for the treatment of Glycogen Storage Disease
Type Ia and expects data in the first half of 2024. DTX401 is being
developed as a one-time gene therapy utilizing REGENXBIO's NAV AAV8
vector.
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable
securities were $415.4 million as of
June 30, 2023, compared to
$565.2 million as of December 31, 2022. The decrease was primarily
driven by cash used to fund operating activities during the first
half of 2023.
Revenues: Revenues were $20.0
million for the three months ended June 30, 2023, compared to $32.6 million for the three months ended
June 30, 2022. The decrease was
primarily attributable to Zolgensma royalty revenues, which
decreased from $28.4 million in the
second quarter of 2022 to $19.0
million in the second quarter of 2023.
Research and Development Expenses: Research and
development expenses were $59.9
million for the three months ended June 30, 2023, compared to $61.0 million for the three months ended
June 30, 2022. The decrease was
primarily attributable to clinical trial and manufacturing expenses
for ABBV-RGX-314 resulting from an increase in development cost
reimbursement from AbbVie under our eye care collaboration, and was
partially offset by an increase in clinical trial expenses for our
other lead product candidates.
General and Administrative Expenses: General and
administrative expenses were $23.7
million for the three months ended June 30, 2023, compared to $20.8 million for the three months ended
June 30, 2022. The increase was
primarily attributable to expenses for professional services and
other corporate overhead costs.
Net Loss: Net loss was $72.1
million, or $1.66 basic and
diluted net loss per share, for the three months ended June 30, 2023, compared to a net loss of
$68.2 million, or $1.58 basic and diluted net loss per share, for
the three months ended June 30,
2022.
FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and
marketable securities of $415.4
million as of June 30, 2023,
to fund its operations into 2025. This cash runway guidance is
based on the Company's current operational plans and excludes the
impact of any payments that may be received from AbbVie upon the
achievement of development or commercial milestones under our
ABBV-RGX-314 collaboration.
CONFERENCE CALL
In connection with this announcement, REGENXBIO will host a
conference call and webcast today at 4:30
p.m. ET. A live audio webcast will be available at
HERE. Interested parties may also pre-register for the
earnings conference call HERE. Once registration is
completed, participants will be provided a dial-in number with a
personalized conference code to access the call. Those who plan on
participating are advised to dial in 15 minutes prior to the start
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, 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, 2022, 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.
Zolgensma® is a registered trademark of Novartis Gene
Therapies. All other trademarks referenced herein are registered
trademarks of REGENXBIO.
REGENXBIO
INC.
CONSOLIDATED BALANCE
SHEETS
(unaudited)
(in
thousands)
|
|
|
|
|
|
June 30,
2023
|
|
|
December 31,
2022
|
|
Assets
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
68,600
|
|
|
$
|
96,952
|
|
Marketable
securities
|
|
|
251,482
|
|
|
|
267,690
|
|
Accounts
receivable
|
|
|
21,380
|
|
|
|
28,082
|
|
Prepaid
expenses
|
|
|
15,112
|
|
|
|
13,900
|
|
Other current
assets
|
|
|
22,235
|
|
|
|
9,352
|
|
Total current
assets
|
|
|
378,809
|
|
|
|
415,976
|
|
Marketable
securities
|
|
|
95,302
|
|
|
|
200,560
|
|
Accounts receivable,
net
|
|
|
1,143
|
|
|
|
1,504
|
|
Property and equipment,
net
|
|
|
138,680
|
|
|
|
141,685
|
|
Operating lease
right-of-use assets
|
|
|
62,436
|
|
|
|
65,116
|
|
Restricted
cash
|
|
|
2,255
|
|
|
|
2,030
|
|
Other assets
|
|
|
3,833
|
|
|
|
6,397
|
|
Total
assets
|
|
$
|
682,458
|
|
|
$
|
833,268
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
11,639
|
|
|
$
|
27,213
|
|
Accrued expenses and
other current liabilities
|
|
|
50,517
|
|
|
|
46,794
|
|
Deferred
revenue
|
|
|
448
|
|
|
|
1,829
|
|
Operating lease
liabilities
|
|
|
6,326
|
|
|
|
5,997
|
|
Liability related to
sale of future royalties
|
|
|
48,963
|
|
|
|
48,601
|
|
Total current
liabilities
|
|
|
117,893
|
|
|
|
130,434
|
|
Operating lease
liabilities
|
|
|
85,254
|
|
|
|
88,802
|
|
Liability related to
sale of future royalties
|
|
|
67,377
|
|
|
|
89,005
|
|
Other
liabilities
|
|
|
6,079
|
|
|
|
8,832
|
|
Total
liabilities
|
|
|
276,603
|
|
|
|
317,073
|
|
Stockholders'
equity
|
|
|
|
|
|
|
Preferred stock; no
shares issued and outstanding
at June 30, 2023 and December 31, 2022
|
|
|
—
|
|
|
|
—
|
|
Common stock; 43,621
and 43,299 shares issued
and outstanding at June 30, 2023 and
December 31, 2022, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
996,239
|
|
|
|
973,145
|
|
Accumulated other
comprehensive loss
|
|
|
(10,098)
|
|
|
|
(15,401)
|
|
Accumulated
deficit
|
|
|
(580,290)
|
|
|
|
(441,553)
|
|
Total stockholders'
equity
|
|
|
405,855
|
|
|
|
516,195
|
|
Total liabilities and
stockholders' equity
|
|
$
|
682,458
|
|
|
$
|
833,268
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
Three
Months
|
|
|
Six
Months
|
|
|
Ended June
30,
|
|
|
Ended June
30,
|
|
|
2023
|
|
|
2022
|
|
|
2023
|
|
|
2022
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
$
|
19,977
|
|
|
$
|
32,649
|
|
|
$
|
39,115
|
|
|
$
|
54,867
|
|
Total
revenues
|
|
19,977
|
|
|
|
32,649
|
|
|
|
39,115
|
|
|
|
54,867
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
9,475
|
|
|
|
12,951
|
|
|
|
13,587
|
|
|
|
28,668
|
|
Research and
development
|
|
59,886
|
|
|
|
61,008
|
|
|
|
118,402
|
|
|
|
116,635
|
|
General and
administrative
|
|
23,698
|
|
|
|
20,832
|
|
|
|
46,332
|
|
|
|
43,150
|
|
Other operating
expenses
|
|
26
|
|
|
|
391
|
|
|
|
59
|
|
|
|
474
|
|
Total operating
expenses
|
|
93,085
|
|
|
|
95,182
|
|
|
|
178,380
|
|
|
|
188,927
|
|
Loss from
operations
|
|
(73,108)
|
|
|
|
(62,533)
|
|
|
|
(139,265)
|
|
|
|
(134,060)
|
|
Other Income
(Expense)
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
40
|
|
|
|
153
|
|
|
|
110
|
|
|
|
247
|
|
Investment
income
|
|
2,127
|
|
|
|
1,061
|
|
|
|
4,293
|
|
|
|
1,860
|
|
Interest
expense
|
|
(1,120)
|
|
|
|
(6,860)
|
|
|
|
(3,875)
|
|
|
|
(12,990)
|
|
Total other income
(expense)
|
|
1,047
|
|
|
|
(5,646)
|
|
|
|
528
|
|
|
|
(10,883)
|
|
Loss before income
taxes
|
|
(72,061)
|
|
|
|
(68,179)
|
|
|
|
(138,737)
|
|
|
|
(144,943)
|
|
Income Tax
Benefit
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
41
|
|
Net loss
|
$
|
(72,061)
|
|
|
$
|
(68,179)
|
|
|
$
|
(138,737)
|
|
|
$
|
(144,902)
|
|
Other Comprehensive
Income (Loss)
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities, net
|
|
1,524
|
|
|
|
(2,813)
|
|
|
|
5,303
|
|
|
|
(12,194)
|
|
Total other
comprehensive income (loss)
|
|
1,524
|
|
|
|
(2,813)
|
|
|
|
5,303
|
|
|
|
(12,194)
|
|
Comprehensive
loss
|
$
|
(70,537)
|
|
|
$
|
(70,992)
|
|
|
$
|
(133,434)
|
|
|
$
|
(157,096)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(1.66)
|
|
|
$
|
(1.58)
|
|
|
$
|
(3.19)
|
|
|
$
|
(3.37)
|
|
Weighted-average common
shares outstanding, basic and diluted
|
|
43,531
|
|
|
|
43,111
|
|
|
|
43,491
|
|
|
|
43,028
|
|
CONTACTS:
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.Brinzey@westwicke.com
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SOURCE REGENXBIO Inc.