- Focus on clinical stage AAV Therapeutic product candidates
addressing large commercial opportunities and value
generation
- Prioritized pipeline is expected to further progress to
pivotal stage and first BLA filing in 2024
- New updates planned for Duchenne and in-office delivery
retinal disease programs starting in March
- $314 million in cash, cash
equivalents and marketable securities as of December 31, 2023, expected to fund operational
runway into the second half of 2025
- Conference call Tuesday, February
27, at 4:30 p.m. ET
ROCKVILLE, Md., Feb. 27,
2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
today announced financial results for the fourth quarter and full
year ended December 31, 2023, and
recent operational highlights, including progress on the strategic
pipeline prioritization and corporate restructuring intended to
significantly reduce operating expenses and support meaningful
value generation from the Company's strong pipeline of AAV
Therapeutics.
"We started 2024 with amazing data from our AbbVie-partnered eye
care programs and our treatments for Duchenne and Hunter syndrome.
Our strategic pipeline prioritization at the end of 2023 created a
sharpened focus for us and today we are rapidly advancing products
through late-stage clinical trials. We believe this is the best way
to support the creation of meaningful value," said Kenneth T. Mills, President and Chief Executive
Officer of REGENXBIO. "We expect more important data readouts and
milestones to be achieved across all programs and the initiation of
new pivotal trials this year. 2024 will be a turning point in our
journey to bring ground-breaking AAV Therapeutics to millions of
patients."
PROGRAM HIGHLIGHTS AND MILESTONES
Retinal Disease: ABBV-RGX‑314, in collaboration with
AbbVie, is a potential one-time treatment for wet age-related
macular degeneration (wet AMD), diabetic retinopathy (DR) and other
chronic retinal disease that is designed to continually produce an
anti-VEGF protein in the eye. ABBV-RGX-314 is currently being
evaluated in patients with wet AMD and DR in nine ongoing clinical
trials, including two pivotal trials. A single ABBV-RGX-314
treatment has the potential to become a new standard-of-care option
among anti-VEGF treatments by sustaining vision health long term
and overcoming the clinical challenges of managing retinal disease
due to the treatment burden of chronic anti-VEGF
injections.
- ABBV-RGX-314 Subretinal Delivery for the Treatment of Wet AMD
- Enrollment is on track in ATMOSPHERE® and ASCENT™
pivotal trials and these trials are expected to support global
regulatory submissions with the U.S. Food and Drug Administration
and the European Medicines Agency in late 2025 through the first
half of 2026.
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD
- REGENXBIO expects to share new program and data updates for the
Phase II AAVIATE trial in mid-2024.
- In January 2024, REGENXBIO
presented data from the AAVIATE® trial demonstrating
that, at six months, patients treated with ABBV-RGX-314 continue to
demonstrate stable vision and retinal anatomy while a meaningful
reduction in anti-VEGF treatment burden was observed. The highest
reduction was seen in dose level 3, demonstrating an 80% reduction
in annualized injection rate with 50% of patients remaining
injection-free.
- ABBV-RGX-314 Suprachoroidal Delivery for Treatment of DR
- REGENXBIO expects to share new program and data updates for the
Phase II ALTITUDE® trial in Q2 2024.
- In November 2023, REGENXBIO
presented data from the ALTITUDE trial showing that, 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 patients developing vision-threatening events by 89%.
Neuromuscular Disease: RGX-202 is an investigational
one-time AAV therapeutic designed to meaningfully impact disease by
delivering a gene for a novel microdystrophin with important
biology most similar to naturally occurring dystrophin that
protects from the muscle degradation associated with Duchenne.
- REGENXBIO plans to share new updates from the Phase I/II
AFFINITY DUCHENNE® trial at the Muscular Dystrophy
Association Clinical and Scientific Meeting being held next week in
Orlando, FL and virtually.
- REGENXBIO expects to make a pivotal dose determination in
mid-2024. The Company also expects to share initial strength and
functional assessment data for both dose levels and the initiation
of a pivotal program in the second half of 2024.
- In February 2024, REGENXBIO
reported interim data from the AFFINITY DUCHENNE trial,
demonstrating that all three patients at dose level 1 indicate
encouraging increases in expression of RGX-202 microdystrophin at
three months and reduction from baseline in serum creatinine kinase
levels, supporting evidence of clinical improvement. In the third
patient, RGX-202 microdystrophin expression was measured to be
83.4%.
- 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.
Neurodegenerative Disease: RGX-121 is an
investigational one-time AAV therapeutic designed to change the
course of disease by restoring the gene missing in boys with MPS
II.
- On track to file a BLA in 2024 using the accelerated approval
pathway. Approval of the planned BLA could result in receipt of a
Priority Review Voucher in 2025.
- In February 2024, REGENXBIO
reported the pivotal phase of the CAMPSIITE® trial
achieved its primary endpoint, as treated patients achieved
decreased cerebrospinal fluid (CSF) levels of D2S6 below maximum
attenuated disease levels at 16 weeks (p value of 0.00016).
Patients treated with RGX-121 have showed continued improvement in
neurodevelopmental skill acquisition up to four years and
discontinued intravenous enzyme therapy.
NAV® TECHNOLOGY PLATFORM LICENSEE PROGRAM
HIGHLIGHTS
Novartis AG reported fourth quarter and full year 2023 global
sales of Zolgensma, for the treatment of spinal muscular atrophy,
of $286 million and $1.21 billion, respectively. Novartis, Rocket
Pharmaceuticals and Ultragenyx Pharmaceutical all have
investigational AAV Therapeutics in pivotal phase that have
multiple milestones expected throughout 2024. Eli Lilly is also
developing several AAV Therapeutics in Phase II for
neurodegenerative diseases using REGENXBIO NAV Technology.
FINANCIAL RESULTS
Cash Position: Cash, cash equivalents and marketable
securities were $314.1 million as of
December 31, 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 year
ended December 31, 2023.
Revenues: Revenues were $22.2
million and $90.2 million for
the three months and full year ended December 31, 2023, respectively, compared to
$31.3 million and $112.7 million for the three months and full year
ended December 31, 2022,
respectively. The decreases were primarily attributable to
Zolgensma royalty revenues, which decreased from $101.9 million for the year ended December 31, 2022 to $85.3
million for the year ended December
31, 2023.
Research and Development Expenses: Research and
development expenses were $55.7
million and $232.3 million for
the three months and full year ended December 31, 2023, respectively, compared to
$62.5 million and $242.5 million for the three months and full year
ended December 31, 2022,
respectively. The decreases were 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 were partially offset by increased
clinical trial expenses for our other lead product candidates.
General and Administrative Expenses: General and
administrative expenses were $19.1
million and $88.5 million for
the three months and full year ended December 31, 2023, respectively, compared to
$21.2 million and $85.3 million for the three months and full year
ended December 31, 2022,
respectively. The increase for the full year ended December 31, 2023 was primarily attributable to
personnel-related costs, expenses for professional services and
other corporate overhead costs, and was partially offset by a
decrease in these costs in the fourth quarter of 2023 as compared
to the fourth quarter of 2022.
Net Loss: Net loss was $62.9
million, or $1.43 basic and
diluted net loss per share, for the three months ended December 31, 2023, compared to a net loss of
$59.9 million, or $1.38 basic and diluted net loss per share, for
the three months ended December 31,
2022. Net loss was $263.5
million, or $6.02 basic and
diluted net loss per share, for the year ended December 31, 2023, compared to a net loss of
$280.3 million, or $6.50 basic and diluted net loss per share, for
the year ended December 31, 2022.
FINANCIAL GUIDANCE
REGENXBIO expects its balance in cash, cash equivalents and
marketable securities of $314.1
million as of December 31,
2023 to fund its 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. ET. 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 REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene
therapy. Since its founding in 2009, REGENXBIO has pioneered the
development of AAV Therapeutics, an innovative class of gene
therapy medicines. REGENXBIO is advancing a pipeline of AAV
Therapeutics for retinal and rare diseases, including 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 of MPS II. Thousands of
patients have been treated with REGENXBIO's AAV Therapeutic
platform, including Novartis' ZOLGENSMA for children with spinal
muscular atrophy. Designed to be one-time treatments, AAV
Therapeutics have the potential to change the way healthcare is
delivered for millions of people. For more information, please
visit www.regenxbio.com.
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, 2023,
which will be filed with the SEC in the first quarter of 2024 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)
|
|
|
December 31,
2023
|
|
|
December 31,
2022
|
|
Assets
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
34,522
|
|
|
$
|
96,952
|
|
Marketable
securities
|
|
|
240,736
|
|
|
|
267,690
|
|
Accounts receivable,
net
|
|
|
24,790
|
|
|
|
28,082
|
|
Prepaid
expenses
|
|
|
14,520
|
|
|
|
13,900
|
|
Other current
assets
|
|
|
20,403
|
|
|
|
9,352
|
|
Total current
assets
|
|
|
334,971
|
|
|
|
415,976
|
|
Marketable
securities
|
|
|
38,871
|
|
|
|
200,560
|
|
Accounts receivable,
net
|
|
|
701
|
|
|
|
1,504
|
|
Property and equipment,
net
|
|
|
132,103
|
|
|
|
141,685
|
|
Operating lease
right-of-use assets
|
|
|
60,487
|
|
|
|
65,116
|
|
Restricted
cash
|
|
|
2,030
|
|
|
|
2,030
|
|
Other assets
|
|
|
4,807
|
|
|
|
6,397
|
|
Total
assets
|
|
$
|
573,970
|
|
|
$
|
833,268
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
22,786
|
|
|
$
|
27,213
|
|
Accrued expenses and
other current liabilities
|
|
|
49,703
|
|
|
|
46,794
|
|
Deferred
revenue
|
|
|
148
|
|
|
|
1,829
|
|
Operating lease
liabilities
|
|
|
7,068
|
|
|
|
5,997
|
|
Liability related to
sale of future royalties
|
|
|
50,567
|
|
|
|
48,601
|
|
Total current
liabilities
|
|
|
130,272
|
|
|
|
130,434
|
|
Operating lease
liabilities
|
|
|
82,222
|
|
|
|
88,802
|
|
Liability related to
sale of future royalties
|
|
|
43,485
|
|
|
|
89,005
|
|
Other
liabilities
|
|
|
6,249
|
|
|
|
8,832
|
|
Total
liabilities
|
|
|
262,228
|
|
|
|
317,073
|
|
Stockholders'
equity
|
|
|
|
|
|
|
Preferred stock; no
shares issued and outstanding
at December 31, 2023 and 2022
|
|
|
—
|
|
|
|
—
|
|
Common stock; 44,046
and 43,299 shares issued
and outstanding at December 31, 2023 and
2022, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
1,021,214
|
|
|
|
973,145
|
|
Accumulated other
comprehensive loss
|
|
|
(4,429)
|
|
|
|
(15,401)
|
|
Accumulated
deficit
|
|
|
(705,047)
|
|
|
|
(441,553)
|
|
Total stockholders'
equity
|
|
|
311,742
|
|
|
|
516,195
|
|
Total liabilities and
stockholders' equity
|
|
$
|
573,970
|
|
|
$
|
833,268
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands,
except per share data)
|
|
|
Three
Months
|
|
|
Years
|
|
|
Ended December
31,
|
|
|
Ended December
31,
|
|
|
2023
|
|
|
2022
|
|
|
2023
|
|
|
2022
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
$
|
22,213
|
|
|
$
|
31,345
|
|
|
$
|
90,242
|
|
|
$
|
112,724
|
|
Total
revenues
|
|
22,213
|
|
|
|
31,345
|
|
|
|
90,242
|
|
|
|
112,724
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
11,238
|
|
|
|
12,783
|
|
|
|
37,213
|
|
|
|
54,545
|
|
Research and
development
|
|
55,681
|
|
|
|
62,505
|
|
|
|
232,266
|
|
|
|
242,453
|
|
General and
administrative
|
|
19,079
|
|
|
|
21,210
|
|
|
|
88,494
|
|
|
|
85,281
|
|
Other operating
expenses (income)
|
|
118
|
|
|
|
(7,382)
|
|
|
|
397
|
|
|
|
(6,679)
|
|
Total operating
expenses
|
|
86,116
|
|
|
|
89,116
|
|
|
|
358,370
|
|
|
|
375,600
|
|
Loss from
operations
|
|
(63,903)
|
|
|
|
(57,771)
|
|
|
|
(268,128)
|
|
|
|
(262,876)
|
|
Other Income
(Expense)
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
(141)
|
|
|
|
77
|
|
|
|
25
|
|
|
|
342
|
|
Investment
income
|
|
2,366
|
|
|
|
2,026
|
|
|
|
11,319
|
|
|
|
5,383
|
|
Interest
expense
|
|
(1,363)
|
|
|
|
(4,310)
|
|
|
|
(6,862)
|
|
|
|
(23,254)
|
|
Total other income
(expense)
|
|
862
|
|
|
|
(2,207)
|
|
|
|
4,482
|
|
|
|
(17,529)
|
|
Loss before income
taxes
|
|
(63,041)
|
|
|
|
(59,978)
|
|
|
|
(263,646)
|
|
|
|
(280,405)
|
|
Income Tax
Benefit
|
|
152
|
|
|
|
43
|
|
|
|
152
|
|
|
|
84
|
|
Net loss
|
$
|
(62,889)
|
|
|
$
|
(59,935)
|
|
|
$
|
(263,494)
|
|
|
$
|
(280,321)
|
|
Other Comprehensive
Loss
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities, net
|
|
2,984
|
|
|
|
2,855
|
|
|
|
10,972
|
|
|
|
(12,832)
|
|
Total other
comprehensive income (loss)
|
|
2,984
|
|
|
|
2,855
|
|
|
|
10,972
|
|
|
|
(12,832)
|
|
Comprehensive
loss
|
$
|
(59,905)
|
|
|
$
|
(57,080)
|
|
|
$
|
(252,522)
|
|
|
$
|
(293,153)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
$
|
(1.43)
|
|
|
$
|
(1.38)
|
|
|
$
|
(6.02)
|
|
|
$
|
(6.50)
|
|
Weighted-average common
shares outstanding, basic and diluted
|
|
44,001
|
|
|
|
43,296
|
|
|
|
43,734
|
|
|
|
43,152
|
|
CONTACTS:
Dana Cormack
Corporate Communications
Dcormack@regenxbio.com
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.Brinzey@westwicke.com
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