ROCKVILLE, Md., Nov. 4, 2020 /PRNewswire/ -- 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 announced financial results for the quarter ended
September 30, 2020, and recent
operational highlights.
"We have made several important advancements in our clinical
pipeline during the third quarter of 2020, with continued focus on
execution during the COVID-19 pandemic. I'm pleased that two Phase
II trials are underway for the treatment of wet AMD and diabetic
retinopathy using the suprachoroidal approach for the delivery of
RGX-314. In addition, we have expanded our RGX-121 program for
patients with MPS II to gain additional insight into the potential
treatment effects of this one-time gene therapy candidate in more
patients, and we have dosed additional patients in the second
cohort of the ongoing Phase I/II trial. I look forward to providing
additional updates on all of our clinical programs in 2021, as we
continue to focus on the significant unmet medical needs in both
large indications and rare diseases," said Kenneth T. Mills, President and Chief Executive
Officer of REGENXBIO.
"The overall progress of gene therapy treatments continues to be
encouraging, as we see additional patients around the world treated
with Zolgensma®, the first approved gene therapy based
on REGENXBIO's NAV Technology Platform," continued Mr. Mills. "We
maintain our focus on our important relationships with partners and
licensees in the gene therapy space, with a strong focus on patient
needs and innovative approaches to treat disease."
Recent Operational Highlights
Gene Therapy Using NAV Vectors for AAV-Mediated Antibody
Delivery
- RGX-314 for the Treatment of Wet Age-related Macular
Degeneration (wet AMD)
-
- REGENXBIO expects to initiate the pivotal program for the
subretinal delivery of RGX-314 for the treatment of wet AMD in the
first quarter of 2021.
- On September 9, 2020, REGENXBIO
announced that the first patient had been dosed in the Phase II
AAVIATE trial to evaluate the suprachoroidal delivery of RGX-314
using the SCS Microinjector® for the treatment of wet
AMD. REGENXBIO expects to complete enrollment of the first cohort
by the end of 2020, and report initial safety data from the first
cohort in early 2021.
- RGX-314 for the Treatment of Diabetic Retinopathy (DR)
-
- The Phase II trial, ALTITUDE, to evaluate the targeted,
in-office suprachoroidal delivery of RGX-314 in patients with DR is
active and REGENXBIO expects to begin enrolling patients by the end
of 2020. REGENXBIO plans to report interim data from this trial in
2021.
- Research Program for the Treatment of Hereditary Angioedema
(HAE)
-
- REGENXBIO expects to provide a program update in 2021.
- Research Program for the Treatment of Neurodegenerative
Diseases
-
- REGENXBIO continues to collaborate with Neurimmune AG on
research programs targeting both alpha synuclein and tau, and
REGENXBIO expects to provide program updates in 2021.
Gene Therapy Using NAV Vectors for Rare Genetic
Diseases
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS
II)
-
- Eight patients have now been dosed across two dose cohorts in
the ongoing Phase I/II trial of RGX-121 in severe MPS II patients
under the age of 5 years old. The first two patients in the
expanded Cohort 2 were dosed in October
2020, via intracisternal delivery of RGX-121 at a dose of
6.5x1010 genome copies per gram (GC/g) of brain mass.
REGENXBIO anticipates further updates from this trial by the end of
2020.
- On September 30, 2020, REGENXBIO
announced the expansion of the RGX-121 program for MPS II to gain
additional insight into the neurodegenerative manifestations of the
disease and evaluate RGX-121 in a broader patient population:
-
- REGENXBIO plans to begin a second Phase I/II multicenter,
open-label trial of RGX-121 for the treatment of pediatric patients
with severe MPS II over the age of 5 years old. Up to six patients
will be enrolled, and RGX-121 will be administered at a dose level
of 6.5x1010 GC/g of brain mass.
- REGENXBIO also announced a new prospective observational study
designed to provide detailed characterization of neurocognitive
development and key biomarkers in patients with severe MPS II.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS
I)
-
- Recruitment and patient screening are ongoing in REGENXBIO's
Phase I/II clinical trial evaluating RGX-111 for the treatment of
MPS I.
- REGENXBIO expects to provide a program update by the end of
2020.
- RGX-381 for the Treatment of Ocular Manifestations of
Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
Disease
-
- REGENXBIO is on track to submit an Investigational New Drug
(IND) application for a Phase I/II study of RGX-381 in patients
with CLN2 disease by the end of 2020 and plans to initiate
enrollment in the first half of 2021.
- Data from non-human primates demonstrate elevated and sustained
levels of TPP1, the enzyme deficient in patients with CLN2 disease,
in the vitreous following a single subretinal injection of
RGX-381.
- RGX-181 for the Treatment of CLN2 Disease
-
- REGENXBIO expects to submit an IND for the intracisternal
delivery of RGX-181 in the first quarter of 2021, and plans to
initiate enrollment in a Phase I/II trial in the first half of
2021.
- Research Program for the Treatment of Neuromuscular
Disorders
-
- REGENXBIO expects to announce a program update in 2021.
Operational Updates
- 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 the first half of 2021.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 operational starting in the first half of
2022.
NAV Technology Licensee Program Highlights
As of September 30, 2020,
REGENXBIO's NAV Technology Platform was being applied in one
marketed product, and multiple clinical stage programs, with over
20 partnered programs in total. REGENXBIO's NAV Technology
Licensees are advancing product candidates in a broad range of
therapeutic areas and disease indications.
REGENXBIO recently collaborated with Kurma Partners, a leading
European venture capital firm, to form Corlieve Therapeutics, a new
company focused on severe neurological conditions. Under the
collaboration and license agreement with Corlieve, REGENXBIO has
licensed Corlieve rights to the NAV AAV9 vector for the treatment
of refractory temporal lobe epilepsy. In return for these rights,
REGENXBIO received equity in Corlieve and is eligible to receive
milestone payments and royalties. In addition, REGENXBIO's Chief
Scientific Officer, Olivier Danos,
Ph.D., has joined Corlieve's Board of Directors. Idinvest Partners
and Pureos Bioventures are also investors in Corlieve.
Marketed NAV Technology Product Highlights
REGENXBIO's NAV Technology Platform is being applied in one
marketed product, Zolgensma. On October 27,
2020, Novartis AG reported third quarter 2020 global
Zolgensma sales revenue of $291
million, and REGENXBIO recognized royalty revenue of
$18.8 million in the third quarter of
2020 as a result of these sales. Novartis AG has recorded over
$1.0 billion in cumulative net sales
of Zolgensma since launch in 2019.
Financial Results
Cash Position: Cash, cash equivalents and marketable
securities were $289.8 million as of
September 30, 2020, compared to
$400.0 million as of December 31, 2019. The decrease was
primarily attributable to net cash used in operating activities of
$93.5 million and cash used to
purchase property and equipment of $14.0
million. Cash, cash equivalents and marketable securities
reported as of September 30, 2020
excludes the $80.0 million milestone
payment received from Novartis AG in October
2020, which was recorded as accounts receivable as of
September 30, 2020.
Revenues: Revenues were $98.9
million for the three months ended September 30, 2020, compared to $14.7 million for the three months ended
September 30, 2019. The increase was
primarily attributable to a $9.6
million increase in Zolgensma royalty revenue for the third
quarter of 2020 as compared to the third quarter of 2019, as well
as an $80.0 million milestone fee
recognized as revenue in the third quarter of 2020 upon the
achievement of $1.0 billion in
cumulative net sales of Zolgensma. Net sales of Zolgensma for the
third quarter of 2020 increased by 82% as compared to the third
quarter of 2019.
Research and Development Expenses: Research and
development expenses were $44.0
million for the three months ended September 30, 2020, compared to $35.7 million for the three months ended
September 30, 2019. The increase was
primarily attributable to personnel-related costs as a result of
increased headcount, expenses associated with conducting clinical
trials for our lead product candidates, and externally sourced
services for preclinical, regulatory and manufacturing-related
activities.
General and Administrative Expenses: General and
administrative expenses were $15.9
million for the three months ended September 30, 2020, compared to $12.4 million for the three months ended
September 30, 2019. The increase was
primarily attributable to personnel-related costs as a result of
increased headcount, and professional fees for advisory and other
services.
Net Income (Loss): Net income was $8.8 million, or $0.24 basic and $0.23 diluted net income per share, for the three
months ended September 30, 2020,
compared to net loss of $34.6
million, or $0.94 basic and
diluted net loss per share, for the three months ended September 30, 2019. Net income for the three
months ended September 30, 2020
includes a non-cash charge of $7.7
million for a provision for credit losses recognized related
to accounts receivable during the period.
Financial Guidance
Based on its current operating plan, REGENXBIO expects its
balance in cash, cash equivalents and marketable securities of
$289.8 million as of September 30, 2020, in addition to the
$80.0 million received from Novartis
AG in October 2020, to fund its
operations, including the completion of its internal manufacturing
capabilities and clinical advancement of its product candidates,
until mid-2022.
Q3 Earnings Conference Call
In connection with this announcement, REGENXBIO will host a
conference call and webcast today at 4:30
p.m. ET. To access the live call by phone, dial (855)
422-8964 (domestic) or (210) 229-8819 (international) and enter the
passcode 4107819. To access a live or recorded webcast of the call,
please visit the "Investors" section of the REGENXBIO website at
www.regenxbio.com. The recorded webcast will be available for
approximately 30 days following the call.
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,
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, 2019, 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.
SCS Microinjector® is a registered trademark of
Clearside Biomedical, Inc. 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,
except per share data)
|
|
|
|
September 30,
2020
|
|
|
December 31,
2019
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
93,220
|
|
|
$
|
69,514
|
|
Marketable
securities
|
|
|
148,305
|
|
|
|
226,696
|
|
Accounts receivable,
net
|
|
|
122,116
|
|
|
|
38,148
|
|
Prepaid
expenses
|
|
|
16,112
|
|
|
|
6,475
|
|
Other current
assets
|
|
|
7,197
|
|
|
|
4,199
|
|
Total current
assets
|
|
|
386,950
|
|
|
|
345,032
|
|
Marketable
securities
|
|
|
48,272
|
|
|
|
103,785
|
|
Accounts receivable,
net
|
|
|
3,564
|
|
|
|
4,155
|
|
Property and
equipment, net
|
|
|
38,871
|
|
|
|
28,973
|
|
Operating lease
right-of-use assets
|
|
|
57,827
|
|
|
|
10,078
|
|
Restricted
cash
|
|
|
1,330
|
|
|
|
1,330
|
|
Other
assets
|
|
|
4,360
|
|
|
|
4,555
|
|
Total
assets
|
|
$
|
541,174
|
|
|
$
|
497,908
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
15,244
|
|
|
$
|
6,409
|
|
Accrued expenses and
other current liabilities
|
|
|
44,356
|
|
|
|
24,846
|
|
Deferred
revenue
|
|
|
449
|
|
|
|
—
|
|
Operating lease
liabilities
|
|
|
3,564
|
|
|
|
2,421
|
|
Total current
liabilities
|
|
|
63,613
|
|
|
|
33,676
|
|
Deferred
revenue
|
|
|
3,895
|
|
|
|
3,333
|
|
Operating lease
liabilities
|
|
|
57,461
|
|
|
|
8,874
|
|
Other
liabilities
|
|
|
545
|
|
|
|
1,828
|
|
Total
liabilities
|
|
|
125,514
|
|
|
|
47,711
|
|
Stockholders' equity
|
|
|
|
|
|
|
|
|
Preferred stock;
$0.0001 par value; 10,000 shares authorized, and no shares issued and
outstanding at September 30, 2020
and December 31, 2019
|
|
|
—
|
|
|
|
—
|
|
Common stock; $0.0001
par value; 100,000 shares authorized at September 30, 2020 and December 31,
2019; 37,404 and 36,992
shares issued and outstanding at September 30, 2020 and December 31, 2019,
respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
658,224
|
|
|
|
627,810
|
|
Accumulated other
comprehensive income
|
|
|
263
|
|
|
|
205
|
|
Accumulated
deficit
|
|
|
(242,831)
|
|
|
|
(177,822)
|
|
Total
stockholders' equity
|
|
|
415,660
|
|
|
|
450,197
|
|
Total liabilities and
stockholders' equity
|
|
$
|
541,174
|
|
|
$
|
497,908
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME
(LOSS)
(unaudited)
(in thousands,
except per share data)
|
|
|
|
Three
Months
|
|
|
Nine
Months
|
|
|
|
Ended September
30,
|
|
|
Ended September
30,
|
|
|
|
2020
|
|
|
2019
|
|
|
2020
|
|
|
2019
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
|
$
|
98,912
|
|
|
$
|
14,700
|
|
|
$
|
133,122
|
|
|
$
|
23,465
|
|
Total
revenues
|
|
|
98,912
|
|
|
|
14,700
|
|
|
|
133,122
|
|
|
|
23,465
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
|
17,364
|
|
|
|
2,494
|
|
|
|
25,457
|
|
|
|
4,450
|
|
Research and
development
|
|
|
43,968
|
|
|
|
35,692
|
|
|
|
119,114
|
|
|
|
90,378
|
|
General and
administrative
|
|
|
15,859
|
|
|
|
12,402
|
|
|
|
46,246
|
|
|
|
37,365
|
|
Provision for credit
losses and other
|
|
|
7,770
|
|
|
|
8
|
|
|
|
7,887
|
|
|
|
(54)
|
|
Total operating
expenses
|
|
|
84,961
|
|
|
|
50,596
|
|
|
|
198,704
|
|
|
|
132,139
|
|
Income (loss) from
operations
|
|
|
13,951
|
|
|
|
(35,896)
|
|
|
|
(65,582)
|
|
|
|
(108,674)
|
|
Other Income
(Loss)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
|
1,444
|
|
|
|
716
|
|
|
|
4,141
|
|
|
|
2,091
|
|
Investment income
(loss)
|
|
|
(6,607)
|
|
|
|
431
|
|
|
|
(4,071)
|
|
|
|
37,950
|
|
Total other income
(loss)
|
|
|
(5,163)
|
|
|
|
1,147
|
|
|
|
70
|
|
|
|
40,041
|
|
Income (loss) before
income taxes
|
|
|
8,788
|
|
|
|
(34,749)
|
|
|
|
(65,512)
|
|
|
|
(68,633)
|
|
Income Tax
Benefit
|
|
|
3
|
|
|
|
165
|
|
|
|
503
|
|
|
|
364
|
|
Net income
(loss)
|
|
$
|
8,791
|
|
|
$
|
(34,584)
|
|
|
$
|
(65,009)
|
|
|
$
|
(68,269)
|
|
Other
Comprehensive Income (Loss)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities, net
|
|
|
(487)
|
|
|
|
(108)
|
|
|
|
58
|
|
|
|
1,043
|
|
Total other
comprehensive income (loss)
|
|
|
(487)
|
|
|
|
(108)
|
|
|
|
58
|
|
|
|
1,043
|
|
Comprehensive income
(loss)
|
|
$
|
8,304
|
|
|
$
|
(34,692)
|
|
|
$
|
(64,951)
|
|
|
$
|
(67,226)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net income (loss) per
share:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
$
|
0.24
|
|
|
$
|
(0.94)
|
|
|
$
|
(1.75)
|
|
|
$
|
(1.86)
|
|
Diluted
|
|
$
|
0.23
|
|
|
$
|
(0.94)
|
|
|
$
|
(1.75)
|
|
|
$
|
(1.86)
|
|
Weighted-average
common shares outstanding:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
|
37,342
|
|
|
|
36,813
|
|
|
|
37,234
|
|
|
|
36,618
|
|
Diluted
|
|
|
38,877
|
|
|
|
36,813
|
|
|
|
37,234
|
|
|
|
36,618
|
|
Contacts:
Tricia
Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Eleanor Barisser, 212-600-1902
eleanor@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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