ROCKVILLE, Md., Feb. 26, 2020 /PRNewswire/ --
- Additional data from RGX-314 Phase I/IIa trial for wet AMD
expected in 1H 2020
- RGX-314 clinical trials for treatment of wet AMD and
diabetic retinopathy expected to advance in 2020 using subretinal
and suprachoroidal delivery approaches
- Additional interim data from RGX-121 Phase I/II trial for
MPS II expected in 2020
- Plans to expand rare disease pipeline into neuromuscular
disorders in 2H 2020
- $400 million in cash, cash
equivalents and marketable securities as of December 31, 2019
- Conference call Wednesday, February
26th at 4:30 p.m.
ET
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 fourth quarter
and full year ended December 31,
2019, and recent operational highlights.
"In 2020, we are broadening our internal gene therapy pipeline
using our proprietary NAV Technology Platform, and advancing key
programs including RGX-314 for the treatment of retinal diseases,"
said Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "As we drive the RGX-314
program forward with new trials in wet AMD and diabetic retinopathy
planned for later this year, we are also advancing our RGX-121
program in MPS II based on encouraging data from the first cohort
of patients. We are pleased with the advancement of the RGX-111 and
RGX-501 programs, and look forward to submitting an IND for RGX-181
and providing updates for our research programs in HAE and
neurodegenerative diseases later this year."
Mr. Mills added: "Based on our in-house research and development
team's work, we also plan to expand our rare disease platform to
include a potential treatment for a neuromuscular disorder using
NAV AAV8, which we believe has the potential to treat patients who
lack treatments or who are currently underserved by existing
therapies. I look forward to providing further updates on this work
in the second half of the year."
Recent Operational Highlights
Gene Therapy Using NAV Vectors for AAV-Mediated Antibody
Delivery
- RGX-314 for the Treatment of Wet AMD
-
- REGENXBIO expects to report additional data from the RGX-314
Phase I/IIa trial using subretinal delivery in 2020
-
- One-year data from Cohorts 4 and 5 is expected in mid-2020. As
announced in January 2020, six-month
data from Cohort 5 demonstrated meaningful reductions in
anti-vascular endothelial growth factor (anti-VEGF) treatment
burden following one-time administration of RGX-314. Eight out of
11 (73%) patients remained anti-VEGF injection-free, and
demonstrated a mean improvement in vision of +5 ETDRS letters and a
mean improvement in retinal thickness of -83 microns.
- REGENXBIO expects to provide two-year data from Cohorts 1-3 in
the first quarter of 2020.
- REGENXBIO expects to initiate a pivotal program for the
subretinal delivery of RGX-314 for the treatment of wet AMD in the
second half of 2020.
-
- REGENXBIO plans to finalize the design of the trial based on
the one-year assessment of patients in Cohort 5 in the Phase I/IIa
trial and expects to begin dosing patients in a pivotal trial in
the second half of 2020.
- REGENXBIO plans to initiate the Phase II trial for the
suprachoroidal delivery of RGX-314 using the SCS Microinjector™ for
the treatment of wet AMD in the first half of 2020.
-
- Interim data is expected from the first cohort by the end of
2020.
- RGX-314 for the Treatment of Diabetic Retinopathy (DR)
-
- REGENXBIO expects to submit an IND for a Phase II trial for the
treatment of DR in the first half of 2020. This trial will
evaluate RGX-314 using the SCS Microinjector to deliver RGX-314 to
the suprachoroidal space.
-
- The trial is expected to begin in the second half of 2020 and
enrollment of the first cohort is expected to be complete by the
end of 2020, with interim data expected in 2021.
- Research Program for the Treatment of Hereditary Angioedema
(HAE)
-
- Lead product candidate selection is expected in the first half
of 2020 and REGENXBIO will provide a program update in the second
half of 2020.
- Research Program for the Treatment of Neurodegenerative
Diseases
-
- REGENXBIO recently announced the expansion of the exclusive
collaboration with Neurimmune AG to design and develop vectorized
antibody therapies targeting both alpha synuclein and tau.
REGENXBIO expects to provide a program update in the second half of
2020.
Gene Therapy Using NAV Vectors for Rare Genetic
Diseases
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS
II)
-
- Initial data from Cohort 1 of the Phase I/II trial was
presented at the WORLDSymposium™ conference in February 2020. As of January 27, 2020, RGX-121 was well-tolerated with
no drug-related or procedure-related serious adverse events
assessed. As reported in December
2019, patients had demonstrated consistent and sustained
reduction in heparan sulfate (HS) in the cerebral spinal fluid
(CSF) and available data supported early signs of neurocognitive
stability.
- REGENXBIO expects to provide additional data from Cohort 1 in
mid-2020.
- The first patient of Cohort 2 has been dosed at an increased
dose level and REGENXBIO expects to complete enrollment of the
cohort in the first half of 2020, with interim data available in
second half of 2020.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS
I)
-
- Recruitment, screening and additional site activations are
ongoing in the Phase I/II clinical trial evaluating RGX-111 for the
treatment of MPS I. Recruitment in this trial was previously
focused on an initial patient over 18 years of age; however, the
protocol has recently been amended to include patients 4 months of
age and older, given REGENXBIO's clinical experience with the
direct-to-CNS delivery platform and accumulating data from the
ongong Phase I/II trial of RGX-121.
- REGENXBIO expects to provide a program update in the second
half of 2020.
- RGX-181 for the Treatment of Late-infantile Neuronal Ceroid
Lipofuscinosis Type 2 (CLN2) Disease
-
- REGENXBIO is conducting ongoing preclinical development of
RGX-181, including assessment of unmet clinical needs such as
neurologic and ophthalmologic manifestations of the disease.
REGENXBIO expects to provide a program update in mid-2020 and
submit an IND for a first-in-human trial in the second half of
2020.
- RGX-501 for the Treatment of Homozygous Familial
Hypercholesterolemia (HoFH)
-
- As previously announced, REGENXBIO completed dosing of an
expanded Cohort 2 in the Phase I/II trial of RGX-501 and plans to
assess low-density lipoprotein (LDL-C) levels after all patients
have completed steroid prophylaxis treatment. REGENXBIO expects to
provide interim data in the first half of 2020.
- Research Program for the Treatment of Neuromuscular
Disorders
-
- REGENXBIO expects to announce plans for clinical development of
a potential treatment for a neuromuscular disorder using NAV AAV8
in the second half of 2020.
Operational Updates and Anticipated Milestones in
2020
- 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 late 2020.
- 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 fully operational
starting in 2021.
NAV Technology Licensee Program Highlights
As of December 31, 2019,
REGENXBIO's NAV Technology Platform was being applied in one
marketed product and more than 20 partnered product candidates in
development. Fifteen of these partnered product candidates are in
active clinical development. REGENXBIO's NAV Technology Licensees
are advancing product candidates in a broad range of therapeutic
areas and disease indications. Recent updates from NAV Technology
Licensees include:
Marketed NAV Technology Products
- On January 29, 2020, Novartis AG
reported fourth quarter 2019 U.S. Zolgensma® sales revenue of
$186 million, and REGENXBIO
recognized royalty revenue of $10.7
million in the fourth quarter of 2019 as a result of these
sales.
- Novartis has reported that Zolgensma is currently under
regulatory review in Europe with
an anticipated regulatory decision in the first quarter of 2020 and
in Japan with an anticipated
regulatory decision in the first half of 2020. Zolgensma uses the
NAV AAV9 vector.
Financial Results
Cash Position: Cash, cash equivalents and marketable
securities were $400.0 million as of
December 31, 2019, compared to
$470.6 million as of December 31, 2018. The decrease was primarily
attributable to $107.7 million of net
cash used in operating activities during 2019, partially offset by
realized and unrealized gains of $37.8
million related to our marketable equity securities of
Prevail Therapeutics Inc.
Revenues: Revenues were $11.8
million and $35.2 million for
the three months and year ended December 31,
2019, respectively, compared to $40.8
million and $218.5 million for
the three months and year ended December 31,
2018, respectively. The decreases were primarily
attributable to $176.1 million of
non-recurring revenue recognized in 2018 under REGENXBIO's
March 2014 license agreement with
AveXis, Inc., as amended, as well as $35.6
million of non-recurring revenue recognized in the fourth
quarter of 2018 under REGENXBIO's license agreement with Abeona
Therapeutics Inc. The decreases in revenue were partially offset by
$10.7 million and $20.8 million of royalty revenue recognized
during the three months and year ended December 31, 2019, respectively, related to net
sales of Zolgensma. Commercial sales of Zolgensma commenced in the
second quarter of 2019, and we are eligible to receive a milestone
payment of $80.0 million from AveXis
upon the achievement of $1.0 billion
in cumulative net sales of Zolgensma.
Research and Development Expenses: Research and
development expenses were $33.8
million and $124.2 million for
the three months and year ended December 31,
2019, respectively, compared to $24.3 million and
$83.9 million for the three months
and year ended December 31, 2018,
respectively. The increases were primarily attributable to
personnel costs as a result of increased headcount, laboratory and
facilities costs, 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 $14.5
million and $51.8 million for
the three months and year ended December 31,
2019, respectively compared to $11.1 million and
$36.9 million for the three months
and year ended December 31, 2018,
respectively. The increases were primarily attributable to
personnel costs as a result of increased headcount and professional
fees for advisory and other services.
Net Loss: Net loss was $26.5
million, or $0.72 basic and
diluted net loss per share, and $94.7
million, or $2.58 basic and
diluted net loss per share, for the three months and year ended
December 31, 2019, respectively,
compared to net income of $4.3
million, or $0.12 basic and
$0.11 diluted net income per share,
and $99.9 million, or $2.99 basic and $2.73 diluted net income per share, for the three
months and year ended December 31,
2018, respectively.
Financial Guidance
Based on its current operating plan, REGENXBIO expects its
balance in cash, cash equivalents and marketable securities of
$400 million to fund the completion
of its internal manufacturing capabilities and clinical advancement
of its product candidates into 2022.
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 2439067. To access a live or recorded webcast of the call
and accompanying slides, 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, 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,
which will be filed with the U.S. Securities and Exchange
Commission (SEC) in the first quarter of 2020, and comparable "risk
factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and
other filings, which have been filed with the 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.
Zolgensma® is a registered trademark of AveXis. All other
trademarks referenced herein are registered trademarks of
REGENXBIO.
REGENXBIO
INC.
CONSOLIDATED
BALANCE SHEETS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
December 31,
2019
|
|
|
December 31,
2018
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
69,514
|
|
|
$
|
75,561
|
|
Marketable
securities
|
|
|
226,696
|
|
|
|
244,200
|
|
Accounts
receivable
|
|
|
38,148
|
|
|
|
8,587
|
|
Prepaid
expenses
|
|
|
6,475
|
|
|
|
5,734
|
|
Other current
assets
|
|
|
4,199
|
|
|
|
3,831
|
|
Total current
assets
|
|
|
345,032
|
|
|
|
337,913
|
|
Marketable
securities
|
|
|
103,785
|
|
|
|
150,819
|
|
Accounts
receivable
|
|
|
4,155
|
|
|
|
23,012
|
|
Property and
equipment, net
|
|
|
28,973
|
|
|
|
28,702
|
|
Operating lease
right-of-use assets
|
|
|
10,078
|
|
|
|
—
|
|
Restricted
cash
|
|
|
1,330
|
|
|
|
1,053
|
|
Other
assets
|
|
|
4,555
|
|
|
|
2,315
|
|
Total
assets
|
|
$
|
497,908
|
|
|
$
|
543,814
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
6,409
|
|
|
$
|
4,412
|
|
Accrued expenses and
other current liabilities
|
|
|
24,846
|
|
|
|
17,164
|
|
Deferred
revenue
|
|
|
—
|
|
|
|
600
|
|
Operating lease
liabilities
|
|
|
2,421
|
|
|
|
—
|
|
Total current
liabilities
|
|
|
33,676
|
|
|
|
22,176
|
|
Deferred
revenue
|
|
|
3,333
|
|
|
|
3,333
|
|
Operating lease
liabilities
|
|
|
8,874
|
|
|
|
—
|
|
Deferred
rent
|
|
|
—
|
|
|
|
1,098
|
|
Financing lease
obligations
|
|
|
—
|
|
|
|
5,854
|
|
Other
liabilities
|
|
|
1,828
|
|
|
|
2,505
|
|
Total
liabilities
|
|
|
47,711
|
|
|
|
34,966
|
|
Stockholders' equity
|
|
|
|
|
|
|
|
|
Preferred stock;
$0.0001 par value; 10,000 shares
authorized, and no shares issued and
outstanding
at
December 31, 2019 and December 31, 2018
|
|
|
—
|
|
|
|
—
|
|
Common stock; $0.0001
par value; 100,000 shares
authorized at December 31, 2019 and
December 31, 2018;
36,992 and 36,120 shares issued and
outstanding at December 31, 2019 and
December 31, 2018, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
627,810
|
|
|
|
592,580
|
|
Accumulated other
comprehensive income (loss)
|
|
|
205
|
|
|
|
(720)
|
|
Accumulated
deficit
|
|
|
(177,822)
|
|
|
|
(83,016)
|
|
Total
stockholders' equity
|
|
|
450,197
|
|
|
|
508,848
|
|
Total liabilities and
stockholders' equity
|
|
$
|
497,908
|
|
|
$
|
543,814
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME
(LOSS)
(unaudited)
(in thousands,
except per share data)
|
|
|
|
Three Months Ended
December 31,
|
|
|
Years Ended
December 31,
|
|
|
|
2019
|
|
|
2018
|
|
|
2019
|
|
|
2018
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
|
$
|
11,768
|
|
|
$
|
40,777
|
|
|
$
|
35,233
|
|
|
$
|
218,505
|
|
Total
revenues
|
|
|
11,768
|
|
|
|
40,777
|
|
|
|
35,233
|
|
|
|
218,505
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
|
3,791
|
|
|
|
2,843
|
|
|
|
8,241
|
|
|
|
9,640
|
|
Research and
development
|
|
|
33,807
|
|
|
|
24,329
|
|
|
|
124,185
|
|
|
|
83,873
|
|
General and
administrative
|
|
|
14,450
|
|
|
|
11,144
|
|
|
|
51,815
|
|
|
|
36,850
|
|
Other operating
expenses (income)
|
|
|
44
|
|
|
|
11
|
|
|
|
(10)
|
|
|
|
42
|
|
Total operating
expenses
|
|
|
52,092
|
|
|
|
38,327
|
|
|
|
184,231
|
|
|
|
130,405
|
|
Income (loss) from
operations
|
|
|
(40,324)
|
|
|
|
2,450
|
|
|
|
(148,998)
|
|
|
|
88,100
|
|
Other
Income
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
|
860
|
|
|
|
584
|
|
|
|
2,951
|
|
|
|
8,946
|
|
Investment
income
|
|
|
10,609
|
|
|
|
2,893
|
|
|
|
48,559
|
|
|
|
7,070
|
|
Total other
income
|
|
|
11,469
|
|
|
|
3,477
|
|
|
|
51,510
|
|
|
|
16,016
|
|
Income (loss) before
income taxes
|
|
|
(28,855)
|
|
|
|
5,927
|
|
|
|
(97,488)
|
|
|
|
104,116
|
|
Income Tax Benefit
(Expense)
|
|
|
2,391
|
|
|
|
(1,621)
|
|
|
|
2,755
|
|
|
|
(4,179)
|
|
Net income
(loss)
|
|
$
|
(26,464)
|
|
|
$
|
4,306
|
|
|
$
|
(94,733)
|
|
|
$
|
99,937
|
|
Other
Comprehensive Income (Loss)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale
securities, net
|
|
|
(158)
|
|
|
|
154
|
|
|
|
885
|
|
|
|
(5)
|
|
Total other
comprehensive income (loss)
|
|
|
(158)
|
|
|
|
154
|
|
|
|
885
|
|
|
|
(5)
|
|
Comprehensive income
(loss)
|
|
$
|
(26,622)
|
|
|
$
|
4,460
|
|
|
$
|
(93,848)
|
|
|
$
|
99,932
|
|
Net income (loss) per
share:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
$
|
(0.72)
|
|
|
$
|
0.12
|
|
|
$
|
(2.58)
|
|
|
$
|
2.99
|
|
Diluted
|
|
$
|
(0.72)
|
|
|
$
|
0.11
|
|
|
$
|
(2.58)
|
|
|
$
|
2.73
|
|
Weighted-average
common shares outstanding:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
|
36,905
|
|
|
|
35,951
|
|
|
|
36,690
|
|
|
|
33,427
|
|
Diluted
|
|
|
36,905
|
|
|
|
38,933
|
|
|
|
36,690
|
|
|
|
36,648
|
|
Contacts:
Tricia Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Heather Savelle, 212-600-1902
heather@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
View original content to download
multimedia:http://www.prnewswire.com/news-releases/regenxbio-reports-fourth-quarter-and-full-year-2019-financial-results-and-operational-highlights-301011953.html
SOURCE REGENXBIO Inc.