- RGX-314 program for the treatment of wet AMD and diabetic
retinopathy, being developed in collaboration with AbbVie, remains
on track for first BLA filing in 2024
-
- Enrollment ongoing in the pivotal ATMOSPHERE® and
ASCENT™ clinical trials of RGX-314 for the treatment of wet
AMD using subretinal delivery
- Completed enrollment in Cohort 5 of the AAVIATE®
trial of RGX-314 for the treatment of wet AMD using suprachoroidal
delivery
- Completed enrollment in ALTITUDE® trial of
RGX-314 for the treatment of diabetic retinopathy using
suprachoroidal delivery
- AFFINITY DUCHENNE™ Phase I/II trial of RGX-202
remains on track for dosing in the first half of 2023
- Announced intention to file a BLA in 2024 using the
accelerated approval pathway for RGX-121 for the treatment of MPS
II; pivotal program is active and enrolling patients
- $682 million in cash, cash
equivalents and marketable securities of as of June 30, 2022; operational runway into
2025
- Conference call Wednesday, August
3rd at 4:30 p.m.
ET
ROCKVILLE, Md., Aug. 3, 2022
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced
financial results for the second quarter ended June 30, 2022, and recent operational
highlights.
"Our '5x'25' strategy to have five gene therapies either on the
market or in late-stage development by 2025 is progressing well,"
said Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "Earlier today, we announced
that the pivotal program for RGX-121, our AAV Therapeutic for the
treatment of MPS II, is active and enrolling patients, making this
our second program to enter this stage of development. We believe
RGX-121 will advance rapidly through the clinic, supporting our
intention to file a BLA in 2024 using the accelerated approval
pathway. RGX-314, being developed in collaboration with AbbVie, is
also progressing well. We have two ongoing pivotal trials
evaluating RGX-314 for the treatment of wet AMD and remain on track
for a BLA filing in 2024. As previously announced, we also have
progressed our Phase II trials evaluating suprachoroidal delivery
of RGX-314. With our significant progress over the first half
of the year along with a strong balance sheet to support the
advancement of our leading AAV-gene therapy pipeline, we reman
confident in our ability to deliver meaningful therapies to
patients."
Program Highlights and Milestones
RGX-314: 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. RGX-314 uses the NAV® AAV8 vector to deliver
a gene encoding a therapeutic antibody fragment to inhibit vascular
endothelial growth factor (VEGF).
- RGX-314 Subretinal Delivery for the Treatment of Wet AMD
-
- Enrollment is ongoing in ATMOSPHERE® and ASCENT™,
two pivotal clinical trials to evaluate the efficacy and safety of
RGX-314 in patients with wet AMD using the subretinal delivery
approach. The ASCENT trial is the first trial to be initiated by
REGENXBIO under the eye care collaboration with AbbVie.
- Pivotal trials are expected to support Biologics Licensing
Application (BLA) submission for RGX-314 in 2024.
- RGX-314 Suprachoroidal Delivery for the Treatment of Wet
AMD
-
- Completed enrollment of Cohort 5 of the Phase II
AAVIATE® trial of RGX-314 for the treatment of wet AMD,
which is evaluating RGX-314 at a third dose level of
1x1012 genomic copies per eye (GC/eye) in patients who
are neutralizing antibody (NAb) positive. As in previous cohorts,
patients did not receive prophylactic immune suppressive
corticosteroid therapy before or after administration of
RGX-314.
- RGX-314 Suprachoroidal Delivery for the Treatment of DR
-
- Enrollment is complete in the Phase II ALTITUDE®
trial for the treatment of DR. Cohorts 2 and 3 are evaluating
RGX-314 at an increased dose level of 5x1011 GC/eye,
with Cohort 3 evaluating RGX-314 in patients who are NAb positive.
As in Cohort 1, patients did not receive prophylactic immune
suppressive corticosteroid therapy before or after administration
of RGX-314.
RGX-202: RGX-202 is an investigational one-time AAV
Therapeutic for the treatment of 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 (CT) domain as well as a muscle specific promoter to
support a targeted therapy for improved resistance to muscle damage
associated with Duchenne.
- Preparation for the initiation of the AFFINITY DUCHENNE™ Phase
I/II trial continues, including readying clinical trial sites and
manufacturing additional clinical supply for the trial.
- REGENXBIO anticipates dosing the first patient in this trial in
the first half of 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 (I2S)
enzyme.
- REGENXBIO has announced that, following discussions with the
FDA, it intends to file a BLA in 2024 using the accelerated
approval pathway for RGX-121 for the treatment of MPS II.
-
- The ongoing Phase I/II trial of RGX-121 in children up to five
years old has been expanded into a pivotal Phase I/II/III trial,
called CAMPSIITE™.
- CAMPSIITE, a multicenter, open-label trial, is active and
enrolling patients. The trial is expected to enroll up to 10 MPS II
patients using commercial-scale cGMP material to support the BLA
filing, with the potential to enroll additional patients.
- Glycosaminoglycans (GAGs) in the cerebrospinal fluid (CSF) have
the potential to be considered a surrogate biomarker that is
reasonably likely to predict clinical benefit in MPS II disease
under the accelerated approval pathway, as buildup of GAGs in the
CSF of MPS II patients correlates with clinical manifestations,
including neurodevelopmental deficits.
- The Phase I/II trial of RGX-121 for the treatment of pediatric
patients with MPS II over the age of five years old is also
ongoing.
RGX-111: RGX-111 is an investigational one-time AAV
Therapeutic for the treatment of severe Mucopolysaccharidosis Type
I (MPS I), using the NAV AAV9 vector to deliver the α-l-iduronidase
(IDUA) gene.
- REGENXBIO continues with plans to enroll additional patients in
a Cohort 2 expansion arm of the Phase I/II trial.
Operational Updates
- Opened state-of-the-art gene therapy manufacturing
facility
-
- REGENXBIO's cGMP facility, called the REGENXBIO Manufacturing
Innovation Center, is designed to meet global clinical and
commercial regulatory standards and enable the Company to
efficiently advance its AAV-based gene therapy pipeline from
research and early development to clinical programs to commercial
readiness.
- REGENXBIO is one of only a few gene therapy companies worldwide
with a GMP facility capable of production at scales up to 2,000
liters.
NAV Technology Licensee Program Highlights
As of June 30, 2022, 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 (SMA), is a marketed product
utilizing REGENXBIO's NAV AAV9 vector. In July 2022, Novartis AG reported second quarter
global sales of Zolgensma of $379
million (>2,300 patients treated worldwide.)
- In May 2022, Rocket
Pharmaceuticals, Inc. announced RP-A501 for the treatment of Danon
disease was well-tolerated in both patients from the low-dose (6.7
x 1013GC/kg; n=2) pediatric cohort. RP-A501 is being developed as a
one-time gene therapy utilizing REGENXBIO's NAV AAV9 vector.
- In May 2022, Prevail Therapeutics
Inc., a wholly owned subsidiary of Eli Lilly, announced that the
FDA accepted an IND application to study PR001 (LY3884961) for Type
1 Gaucher disease (GD1) in a Phase 1/2 clinical trial. PR001
(LY3884961) is being developed as a one-time gene therapy utilizing
REGENXBIO's NAV AAV9 vector.
- In May 2022, Ultragenyx
Pharmaceutical Inc. announced dosing and enrollment of the Phase 3
study of DTX401 for Glycogen Storage Disease Type Ia (GSDIa) is
ongoing. Ultragenyx expects to initiate the Phase 3 eNH3ance study
of DTX301 for Ornithine Transcarbamylase (OTC) Deficiency in
mid-2022, and is dosing patients in the Phase 1/2 stage of the
seamless Phase 1/2/3 Cyprus2+ study of UX701 for Wilson Disease.
DTX401 and DTX301 are both being developed as one-time gene
therapies utilizing REGENXBIO's NAV AAV8 vector. UX701 is being
developed as a one-time gene therapy utilizing REGENXBIO's NAV AAV9
vector.
Financial Results
Cash Position: Cash, cash equivalents and marketable
securities were $682.0 million as of
June 30, 2022, compared to
$849.3 million as of December 31, 2021. The decrease was primarily
driven by cash used to fund operating activities and capital
expenditures and temporary unrealized losses on marketable debt
securities during the six months ended June
30, 2022.
Revenues: Revenues were $32.6
million for the three months ended June 30, 2022, compared to $22.0 million for the three months ended
June 30, 2021. The increase was
primarily attributable to Zolgensma royalty revenues, which
increased by $10.0 million, from
$18.4 million for the second quarter
of 2021 to $28.4 million for the
second quarter of 2022. As reported by Novartis, sales of Zolgensma
for the second quarter of 2022 increased by 20% as compared to the
second quarter of 2021, driven by geographic expansion of product
access outside the United
States.
Research and Development Expenses: Research and
development expenses were $61.0
million for the three months ended June 30, 2022, compared to $45.9 million for the three months ended
June 30, 2021. The increase was
primarily attributable to personnel costs as a result of increased
headcount, and costs associated with clinical trials and
manufacturing-related activities for our lead product
candidates.
General and Administrative Expenses: General and
administrative expenses were $20.8
million for the three months ended June 30, 2022, compared to $18.4 million for the three months ended
June 30, 2021. The increase was
primarily attributable to personnel costs and corporate overhead
expenses.
Net Loss: Net loss was $68.2
million, or $1.58 basic and
diluted net loss per share, for the three months ended June 30, 2022, compared to a net loss of
$57.6 million, or $1.36 basic and diluted net loss per share, for
the three months ended June 30,
2021.
Financial Guidance
Based on its current operating plan, REGENXBIO expects its
balance in cash, cash equivalents and marketable securities of
$682.0 million as of June 30, 2022 to fund its operations into
2025.
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
regenxbio.com/investors. 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, 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.
REGENXBIO
INC.
CONSOLIDATED BALANCE
SHEETS
(unaudited)
(in
thousands)
|
|
|
|
|
|
|
|
|
|
June 30,
2022
|
|
|
December 31,
2021
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
121,374
|
|
|
$
|
345,209
|
|
Marketable
securities
|
|
|
281,588
|
|
|
|
112,230
|
|
Accounts receivable,
net
|
|
|
37,500
|
|
|
|
32,439
|
|
Prepaid
expenses
|
|
|
16,245
|
|
|
|
18,752
|
|
Other current
assets
|
|
|
8,717
|
|
|
|
10,196
|
|
Total current
assets
|
|
|
465,424
|
|
|
|
518,826
|
|
Marketable
securities
|
|
|
279,073
|
|
|
|
391,907
|
|
Accounts receivable,
net
|
|
|
2,034
|
|
|
|
2,262
|
|
Property and equipment,
net
|
|
|
138,815
|
|
|
|
131,547
|
|
Operating lease
right-of-use assets
|
|
|
60,163
|
|
|
|
60,904
|
|
Restricted
cash
|
|
|
2,030
|
|
|
|
2,030
|
|
Other assets
|
|
|
5,905
|
|
|
|
6,428
|
|
Total
assets
|
|
$
|
953,444
|
|
|
$
|
1,113,904
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
16,985
|
|
|
$
|
11,387
|
|
Accrued expenses and
other current liabilities
|
|
|
47,014
|
|
|
|
76,111
|
|
Deferred
revenue
|
|
|
6,636
|
|
|
|
3,333
|
|
Operating lease
liabilities
|
|
|
2,832
|
|
|
|
1,752
|
|
Liability related to
sale of future royalties
|
|
|
41,089
|
|
|
|
37,889
|
|
Total current
liabilities
|
|
|
114,556
|
|
|
|
130,472
|
|
Operating lease
liabilities
|
|
|
85,672
|
|
|
|
84,929
|
|
Liability related to
sale of future royalties
|
|
|
114,171
|
|
|
|
133,460
|
|
Other
liabilities
|
|
|
8,526
|
|
|
|
745
|
|
Total
liabilities
|
|
|
322,925
|
|
|
|
349,606
|
|
Stockholders'
equity
|
|
|
|
|
|
|
|
|
Preferred stock; no
shares issued and outstanding
at June
30, 2022 and December 31, 2021
|
|
|
—
|
|
|
|
—
|
|
Common stock; 43,171
and 42,831 shares issued
and
outstanding at June 30, 2022 and
December
31, 2021, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
951,412
|
|
|
|
928,095
|
|
Accumulated other
comprehensive loss
|
|
|
(14,763)
|
|
|
|
(2,569)
|
|
Accumulated
deficit
|
|
|
(306,134)
|
|
|
|
(161,232)
|
|
Total
stockholders' equity
|
|
|
630,519
|
|
|
|
764,298
|
|
Total liabilities and
stockholders' equity
|
|
$
|
953,444
|
|
|
$
|
1,113,904
|
|
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,
|
|
|
|
2022
|
|
|
2021
|
|
|
2022
|
|
|
2021
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
|
$
|
32,649
|
|
|
$
|
22,035
|
|
|
$
|
54,867
|
|
|
$
|
40,919
|
|
Total
revenues
|
|
|
32,649
|
|
|
|
22,035
|
|
|
|
54,867
|
|
|
|
40,919
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
|
12,951
|
|
|
|
9,819
|
|
|
|
28,668
|
|
|
|
14,670
|
|
Research and
development
|
|
|
61,008
|
|
|
|
45,882
|
|
|
|
116,635
|
|
|
|
85,604
|
|
General and
administrative
|
|
|
20,832
|
|
|
|
18,425
|
|
|
|
43,150
|
|
|
|
36,263
|
|
Credit losses and
other
|
|
|
391
|
|
|
|
135
|
|
|
|
474
|
|
|
|
650
|
|
Total operating
expenses
|
|
|
95,182
|
|
|
|
74,261
|
|
|
|
188,927
|
|
|
|
137,187
|
|
Loss from
operations
|
|
|
(62,533)
|
|
|
|
(52,226)
|
|
|
|
(134,060)
|
|
|
|
(96,268)
|
|
Other Income
(Expense)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
|
153
|
|
|
|
554
|
|
|
|
247
|
|
|
|
583
|
|
Investment
income
|
|
|
1,061
|
|
|
|
399
|
|
|
|
1,860
|
|
|
|
979
|
|
Interest
expense
|
|
|
(6,860)
|
|
|
|
(6,366)
|
|
|
|
(12,990)
|
|
|
|
(13,068)
|
|
Total other income
(expense)
|
|
|
(5,646)
|
|
|
|
(5,413)
|
|
|
|
(10,883)
|
|
|
|
(11,506)
|
|
Loss before income
taxes
|
|
|
(68,179)
|
|
|
|
(57,639)
|
|
|
|
(144,943)
|
|
|
|
(107,774)
|
|
Income Tax Benefit
(Expense)
|
|
|
—
|
|
|
|
—
|
|
|
|
41
|
|
|
|
(4)
|
|
Net loss
|
|
$
|
(68,179)
|
|
|
$
|
(57,639)
|
|
|
$
|
(144,902)
|
|
|
$
|
(107,778)
|
|
Other Comprehensive
Income (Loss)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities, net
|
|
|
(2,813)
|
|
|
|
113
|
|
|
|
(12,194)
|
|
|
|
(895)
|
|
Total other
comprehensive income (loss)
|
|
|
(2,813)
|
|
|
|
113
|
|
|
|
(12,194)
|
|
|
|
(895)
|
|
Comprehensive
loss
|
|
$
|
(70,992)
|
|
|
$
|
(57,526)
|
|
|
$
|
(157,096)
|
|
|
$
|
(108,673)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(1.58)
|
|
|
$
|
(1.36)
|
|
|
$
|
(3.37)
|
|
|
$
|
(2.56)
|
|
Weighted-average common
shares outstanding, basic and diluted
|
|
|
43,111
|
|
|
|
42,510
|
|
|
|
43,028
|
|
|
|
42,170
|
|
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey, ICR Westwicke
339-970-2843
Chris.brinzey@westwicke.com
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multimedia:https://www.prnewswire.com/news-releases/regenxbio-reports-second-quarter-2022-financial-results-and-recent-operational-highlights-301599291.html
SOURCE REGENXBIO Inc.