- Announces pipeline prioritization and corporate
restructuring to focus on clinical stage AAV Therapeutic product
candidates addressing large commercial opportunities and value
generation
- Highest priority programs are ABBV-RGX-314 for the treatment
of wet AMD and diabetic retinopathy, being developed in
collaboration with AbbVie, RGX-202 for the treatment of Duchenne,
and RGX-121 for the treatment MPS II
- Restructuring plan, including a 15% reduction in workforce,
expected to result in total savings of at least $100 million
- Anticipated cost savings from corporate restructuring, along
with $365 million in cash, cash
equivalents and marketable securities as of September 30, 2023, expected to fund operational
runway into the second half of 2025
- Conference call Wednesday, November
8, 2023, at 4:30 p.m.
(EST)
ROCKVILLE, Md., Nov. 8, 2023
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
financial results for the third quarter ended September 30, 2023, and recent operational
highlights, including a strategic pipeline prioritization and
corporate restructuring intended to significantly reduce operating
expenses while continuing to support meaningful value generation
from the Company's strong pipeline of AAV Therapeutics.
"In the past two months, we reported exciting, positive clinical
data from investigational treatments for diabetic retinopathy, and
Duchenne, as well as had a very encouraging RMAT meeting with the
FDA about expediting the BLA for our treatment for MPS II," said
Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "These milestones demonstrate
how our science is supporting avenues to accelerate development,
and, today, we are following the data and making necessary
decisions to focus our capabilities and resources on these
differentiated product candidates which represent opportunities to
bring ground-breaking AAV Therapeutics to millions of
patients."
PIPELINE PRIORITIZATION AND CORPORATE RESTRUCTURING
The following key strategic decisions support the pipeline
prioritization and corporate restructuring related to
product candidates that are differentiated, can be expedited,
and support near-term and long-term value generation.
- Prioritizing investigational, one-time AAV therapeutic clinical
stage programs:
- ABBV-RGX-314, 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
- RGX-202 for the treatment of Duchenne muscular dystrophy
(Duchenne)
- RGX-121 for the treatment of Mucopolysaccharidosis Type II
(MPS II)
- Pursuing strategic alternatives, including potential
partnering, for its other rare neurodegenerative disease clinical
stage programs: RGX-111 for the treatment of severe
Mucopolysaccharidosis Type I, RGX-181 for the treatment of
late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2)
disease, a form of Batten disease and RGX-381 for the treatment of
the ocular manifestations of CLN2 disease.
- Reducing its workforce by approximately 15%, primarily in rare
neurodegenerative disease development, early research, and other
general and administrative areas. REGENXBIO expects to incur
approximately $4 million in one-time
restructuring costs in the fourth quarter of 2023.
As a result of the portfolio prioritization and corporate
restructuring, REGENXBIO anticipates total savings of at least
$100 million over the next two years.
These anticipated cost savings along with $365 million in cash, cash equivalents and
marketable securities as of September 30,
2023 are now expected to fund operations into the second
half of 2025.
Mr. Mills added, "since our formation, REGENXBIO has been a
leader in the field because of our mission to improve lives through
the curative potential of gene therapy with our NAV®
Technology Platform. As we refocus our capital allocation in a
challenging economic environment and position the Company to meet
important business goals, I want to express my gratitude for the
commitment and dedication exhibited by our colleagues and partners
in support of this mission."
PROGRAM HIGHLIGHTS AND MILESTONES
ABBV-RGX-314: 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 continues to be on track in
ATMOSPHERE® and ASCENTTM pivotal
trials for the treatment of patients with wet AMD using subretinal
delivery.
- These trials are expected to support global regulatory
submissions with the U.S. Food and Drug Administration (FDA) and
the European Medicines Agency (EMA) in late 2025 through the first
half of 2026.
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD
and DR
- In November 2023, REGENXBIO
presented data from the Phase II ALTITUDE® trial
demonstrating that ABBV-RGX-314 administered using suprachoroidal
delivery was well tolerated in dose levels 1 and 2. At one year,
dose level 2 in non-proliferative DR patients prevented disease
progression as measured by the Early Treatment Diabetic Retinopathy
Study-Diabetic Retinopathy Severity Scale. Dose level 2 reduced the
risk of developing vision-threatening events by 89% in these
patients.
- REGENXBIO expects to report additional interim data from the
Phase II AAVIATE® trial of ABBV-RGX-314 using
suprachoroidal delivery for the treatment of wet AMD, 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, 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 October 2023, REGENXBIO
reported interim data from the Phase I/II AFFINITY
DUCHENNE® trial, demonstrating that RGX-202 continued to
be well tolerated with no drug-related serious adverse events in
three patients at dose level 1. Initial biomarker data in two
patients who completed three-month assessment demonstrate robust
RGX-202 microdystrophin expression with localization to the muscle
cell membrane.
- REGENXBIO plans to use RGX-202 microdystrophin expression as a
surrogate endpoint to support a Biologics License Application (BLA)
filing using the accelerated approval pathway.
- REGENXBIO reported that, based on these interim results,
reported from dose level 1, the FDA has supported amending the
trial protocol to accelerate development. Escalation to dose level
2 is expected by the end of 2023.
- REGENXBIO expects to share initial strength and functional
assessment data for both dose levels in 2024. Additionally,
REGENXBIO anticipates pivotal dose determination and the initiation
of a pivotal program in 2024.
RGX-121: RGX-121 is an investigational one-time AAV
therapeutic for the treatment of MPS II, also known as Hunter
syndrome, using the NAV AAV9 vector to deliver the gene that
encodes the iduronate-2-sulfatase enzyme.
- REGENXBIO continues to follow patients in the Phase I/II/III
CAMPSIITE® trial, with topline results from pivotal
phase expected in the first quarter of 2024.
- A recent positive Regenerative Medicine Advanced Therapy (RMAT)
meeting held in October with FDA confirmed alignment on
manufacturing strategy, adequacy of safety database, and
confirmatory study design, which are key elements for an expedited
plan for BLA filing using the accelerated approval pathway in
2024.
- Based on an expected priority review, potential approval of the
Company's planned BLA for RGX-121 could result in receipt of a Rare
Pediatric Disease Priority Review Voucher in 2025.
OPERATIONAL UPDATES
- The REGENXBIO Manufacturing Innovation Center in
Maryland is using the NAVXpress™
platform process to produce GMP lots intended for upcoming
regulatory submissions and initial launch supply for ABBV-RGX-314
and RGX-121. 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.
NAV® TECHNOLOGY PLATFORM LICENSEE PROGRAM
HIGHLIGHTS
As of September 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 October 2023, Novartis AG reported third quarter
2023 global sales of Zolgensma of $308
million.
- In November 2023, Rocket
Pharmaceuticals announced the initiation of the Phase II pivotal
trial of RP-A501 for Danon Disease. RP-A501 is being developed as a
one-time gene therapy utilizing REGENXBIO's NAV AAV9 vector.
- In October 2023, Ultragenyx
Pharmaceutical Inc. announced plans to provide preliminary Phase
III DTX401 data in the first half of 2024 and that the DTX301 Phase
III study is expected to complete enrollment in the first half of
2024. DTX401 for the treatment of Glycogen Storage Disease Type Ia
and DTX301 for the treatment of Ornithine Transcarbamylase
Deficiency are both being developed as one-time gene therapies
utilizing REGENXBIO's NAV AAV8 vector.
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable
securities were $364.5 million as of
September 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 nine
months ended September 30, 2023.
Revenues: Revenues were $28.9
million for the three months ended September 30, 2023, compared to $26.5 million for the three months ended
September 30, 2022. The increase was
primarily attributable to Zolgensma royalty revenues, which
increased from $25.2 million for the
third quarter of 2022 to $28.4
million for the third quarter of 2023.
Research and Development Expenses: Research and
development expenses were $58.2
million for the three months ended September 30, 2023, compared to $63.3 million for the three months ended
September 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 $23.1
million for the three months ended September 30, 2023, compared to $20.9 million for the three months ended
September 30, 2022. The increase was
primarily attributable to personnel-related costs, expenses for
professional services and other corporate overhead costs.
Net Loss: Net loss was $61.9
million, or $1.41 basic and
diluted net loss per share, for the three months ended September 30, 2023, compared to a net loss of
$75.5 million, or $1.75 basic and diluted net loss per share, for
the three months ended September 30,
2022.
FINANCIAL GUIDANCE
As a result of the portfolio prioritization and corporate
restructuring, REGENXBIO anticipates total savings of at least
$100 million over the next two years.
These anticipated cost savings along with $365 million in cash, cash equivalents and
marketable securities as of September 30,
2023 are now expected to fund operations into the second
half of 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. EST. 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 and AAV9. 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
strategic pipeline prioritization and corporate restructuring plan
or other cost-saving measures, and expected charges associated with
restructuring and any future cost reduction measures, 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)
|
|
|
|
|
|
September 30,
2023
|
|
|
December 31,
2022
|
|
Assets
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
53,045
|
|
|
$
|
96,952
|
|
Marketable
securities
|
|
|
248,842
|
|
|
|
267,690
|
|
Accounts
receivable
|
|
|
28,043
|
|
|
|
28,082
|
|
Prepaid
expenses
|
|
|
12,561
|
|
|
|
13,900
|
|
Other current
assets
|
|
|
23,347
|
|
|
|
9,352
|
|
Total current
assets
|
|
|
365,838
|
|
|
|
415,976
|
|
Marketable
securities
|
|
|
62,639
|
|
|
|
200,560
|
|
Accounts receivable,
net
|
|
|
1,078
|
|
|
|
1,504
|
|
Property and equipment,
net
|
|
|
135,534
|
|
|
|
141,685
|
|
Operating lease
right-of-use assets
|
|
|
61,773
|
|
|
|
65,116
|
|
Restricted
cash
|
|
|
2,255
|
|
|
|
2,030
|
|
Other assets
|
|
|
4,669
|
|
|
|
6,397
|
|
Total
assets
|
|
$
|
633,786
|
|
|
$
|
833,268
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
21,859
|
|
|
$
|
27,213
|
|
Accrued expenses and
other current liabilities
|
|
|
45,656
|
|
|
|
46,794
|
|
Deferred
revenue
|
|
|
442
|
|
|
|
1,829
|
|
Operating lease
liabilities
|
|
|
6,639
|
|
|
|
5,997
|
|
Liability related to
sale of future royalties
|
|
|
52,750
|
|
|
|
48,601
|
|
Total current
liabilities
|
|
|
127,346
|
|
|
|
130,434
|
|
Operating lease
liabilities
|
|
|
84,058
|
|
|
|
88,802
|
|
Liability related to
sale of future royalties
|
|
|
53,096
|
|
|
|
89,005
|
|
Other
liabilities
|
|
|
6,186
|
|
|
|
8,832
|
|
Total
liabilities
|
|
|
270,686
|
|
|
|
317,073
|
|
Stockholders'
equity
|
|
|
|
|
|
|
Preferred stock; no
shares issued and outstanding
at September 30, 2023 and December 31, 2022
|
|
|
—
|
|
|
|
—
|
|
Common stock; 43,991
and 43,299 shares issued
and outstanding at September 30, 2023 and
December 31, 2022, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
1,012,667
|
|
|
|
973,145
|
|
Accumulated other
comprehensive loss
|
|
|
(7,413)
|
|
|
|
(15,401)
|
|
Accumulated
deficit
|
|
|
(642,158)
|
|
|
|
(441,553)
|
|
Total stockholders'
equity
|
|
|
363,100
|
|
|
|
516,195
|
|
Total liabilities and
stockholders' equity
|
|
$
|
633,786
|
|
|
$
|
833,268
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
Three
Months
|
|
|
Nine
Months
|
|
|
Ended September
30,
|
|
|
Ended September
30,
|
|
|
2023
|
|
|
2022
|
|
|
2023
|
|
|
2022
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
$
|
28,914
|
|
|
$
|
26,512
|
|
|
$
|
68,029
|
|
|
$
|
81,379
|
|
Total
revenues
|
|
28,914
|
|
|
|
26,512
|
|
|
|
68,029
|
|
|
|
81,379
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
12,388
|
|
|
|
13,094
|
|
|
|
25,975
|
|
|
|
41,762
|
|
Research and
development
|
|
58,183
|
|
|
|
63,313
|
|
|
|
176,585
|
|
|
|
179,948
|
|
General and
administrative
|
|
23,083
|
|
|
|
20,921
|
|
|
|
69,415
|
|
|
|
64,071
|
|
Other operating
expenses
|
|
220
|
|
|
|
229
|
|
|
|
279
|
|
|
|
703
|
|
Total operating
expenses
|
|
93,874
|
|
|
|
97,557
|
|
|
|
272,254
|
|
|
|
286,484
|
|
Loss from
operations
|
|
(64,960)
|
|
|
|
(71,045)
|
|
|
|
(204,225)
|
|
|
|
(205,105)
|
|
Other Income
(Expense)
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
56
|
|
|
|
18
|
|
|
|
166
|
|
|
|
265
|
|
Investment
income
|
|
4,660
|
|
|
|
1,497
|
|
|
|
8,953
|
|
|
|
3,357
|
|
Interest
expense
|
|
(1,624)
|
|
|
|
(5,954)
|
|
|
|
(5,499)
|
|
|
|
(18,944)
|
|
Total other income
(expense)
|
|
3,092
|
|
|
|
(4,439)
|
|
|
|
3,620
|
|
|
|
(15,322)
|
|
Loss before income
taxes
|
|
(61,868)
|
|
|
|
(75,484)
|
|
|
|
(200,605)
|
|
|
|
(220,427)
|
|
Income Tax
Benefit
|
|
—
|
|
|
|
—
|
|
|
|
—
|
|
|
|
41
|
|
Net loss
|
$
|
(61,868)
|
|
|
$
|
(75,484)
|
|
|
$
|
(200,605)
|
|
|
$
|
(220,386)
|
|
Other Comprehensive
Income (Loss)
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities, net
|
|
2,685
|
|
|
|
(3,493)
|
|
|
|
7,988
|
|
|
|
(15,687)
|
|
Total other
comprehensive income (loss)
|
|
2,685
|
|
|
|
(3,493)
|
|
|
|
7,988
|
|
|
|
(15,687)
|
|
Comprehensive
loss
|
$
|
(59,183)
|
|
|
$
|
(78,977)
|
|
|
$
|
(192,617)
|
|
|
$
|
(236,073)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(1.41)
|
|
|
$
|
(1.75)
|
|
|
$
|
(4.60)
|
|
|
$
|
(5.11)
|
|
Weighted-average common
shares outstanding, basic and diluted
|
|
43,945
|
|
|
|
43,251
|
|
|
|
43,644
|
|
|
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43,103
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CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.Brinzey@westwicke.com
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SOURCE REGENXBIO Inc.