ROCKVILLE, Md., May 7, 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
March 31, 2020, and recent
operational highlights.
"Our overall focus at REGENXBIO during the COVID-19 pandemic is
on the health and safety of our people and our communities, as well
as the important work that we need to continue in order to help
patients," said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. "We recently
reported additional positive data from our RGX-314 Phase I/IIa wet
AMD trial which demonstrated long-term, sustained clinical outcomes
in patients after a one-time administration of RGX-314 gene
therapy. We look forward to initiating a pivotal program for the
subretinal delivery of RGX-314 for wet AMD as well as additional
studies using the suprachoroidal delivery approach in both wet AMD
and diabetic retinopathy in 2020. We also anticipate providing
updates throughout 2020 from our other programs, including the
Phase I/II trials of RGX-121 for the treatment of MPS II and
RGX-501 for the treatment of HoFH."
Mr. Mills added: "We continue to track the positive progress of
Novartis' Zolgensma which uses the NAV AAV9 vector for the
treatment of pediatric patients with spinal muscular atrophy (SMA).
We are encouraged by the recent regulatory updates from
Japan and Europe, and we look forward to continued sales
growth for Zolgensma. After more than a decade of steadfast effort
and focus, we remain dedicated and committed to improving lives
through the curative potential of gene therapy."
Recent Operational Highlights
Gene Therapy Using NAV Vectors for AAV-Mediated Antibody
Delivery
- RGX-314 for the Treatment of Wet AMD
-
- In April 2020, REGENXBIO reported
positive additional data from the RGX-314 Phase I/IIa trial using
subretinal delivery. As of April 6,
2020:
-
- RGX-314 continued to be well-tolerated at all dose levels.
- Long-term, durable treatment effect was demonstrated in Cohort
3:
-
- Mean improvement in vision (+14 letters) and stable retinal
thickness (+2 µm) observed at two years.
- 50% of patients (3/6) remained anti-VEGF injection-free over
two years.
- 67% of patients (4/6) were anti-VEGF injection-free from nine
months to two years.
- Stable intraocular RGX-314 protein expression observed over two
years.
- 73% of patients (8/11) in Cohort 5 were anti-VEGF
injection-free over nine months.
- Intraocular RGX-314 protein levels at six months demonstrated
dose-dependent expression across cohorts.
- 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 Cohorts 4 and 5 in the Phase
I/IIa trial and expects to begin dosing patients in the 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 plans to evaluate RGX-314 using the SCS Microinjector
for the treatment of DR, and expects to submit an investigational
new drug (IND) application for a Phase II trial in mid-2020.
-
- REGENXBIO plans to initiate the trial in the second half of
2020 and interim data is 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 expects to provide a program update in the
second half of 2020.
- Research Program for the Treatment of Neurodegenerative
Diseases
-
- REGENXBIO previously 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 were
presented at the WORLDSymposium conference in February 2020 and REGENXBIO expects to provide
additional data from this cohort in mid-2020.
- REGENXBIO expects to complete enrollment of patients in Cohort
2 in the first half of 2020, with interim data anticipated in the
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 trial.
- 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 and plans to assess
low-density lipoprotein (LDL-C) levels after all patients have
completed steroid prophylaxis treatment. REGENXBIO expects to
provide a program update 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
- 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 operational
starting in 2021.
NAV Technology Licensee Program Highlights
As of March 31, 2020, REGENXBIO's
NAV Technology Platform was being applied in one marketed product,
and the clinical development of 15 partnered product candidates,
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. Recent updates
from NAV Technology Licensees include:
- On March 31, 2020, REGENXBIO and
Ultragenyx Pharmaceutical Inc. announced a new exclusive, worldwide
license agreement to REGENXBIO's NAV AAV8 and AAV9 vectors for the
treatment of a rare metabolic disorder, extending the companies'
existing gene therapy partnership. In return for these rights,
REGENXBIO received an upfront payment of $7
million, and will receive ongoing fees, milestone payments,
and royalties on net sales of products incorporating the licensed
intellectual property.
Marketed NAV Technology Product Highlights
- On April 28, 2020, Novartis AG
reported first quarter 2020 Zolgensma® sales revenue of
$170 million, and REGENXBIO
recognized royalty revenue of $10.0
million in the first quarter of 2020 as a result of these
sales. Since launch in May 2019,
Zolgensma has recorded total net sales of over $530 million.
- On March 27, 2020, Novartis
announced that the Committee for Medicinal Products for Human Use
(CHMP) of the European Medicines Agency (EMA) adopted a positive
opinion recommending conditional marketing authorization of
Zolgensma.
- On March 19, 2020, Novartis
announced that Zolgensma received approval from the Japanese
Ministry of Health, Labour and Welfare (MHLW) for the treatment of
patients under the age of two with spinal muscular atrophy (SMA),
including patients who are pre-symptomatic at diagnosis.
Financial Results
Cash Position: Cash, cash equivalents and marketable
securities were $356.6 million as of
March 31, 2020, compared to
$400.0 million as of December 31, 2019. The decrease was primarily
attributable to net cash used in operating activities of
$35.6 million and cash used to
purchase property and equipment of $4.6
million.
Revenues: Revenues were $17.6
million for the three months ended March 31, 2020, compared to $0.9 million for the three months ended
March 31, 2019. The increase was
primarily attributable to $10.0
million of royalty revenue recognized on net sales of
Zolgensma in the first quarter of 2020, as well as $7.2 million of license revenue recognized from a
new license granted to Ultragenyx Pharmaceutical Inc. during the
period. Commercial sales of Zolgensma commenced in the second
quarter of 2019, and REGENXBIO is eligible to receive a milestone
payment of $80.0 million from AveXis,
Inc. upon the achievement of $1.0
billion in cumulative net sales of Zolgensma.
Research and Development Expenses: Research and
development expenses were $37.0
million for the three months ended March 31, 2020, compared to $25.2 million
for the three months ended March 31,
2019. The increase was primarily attributable to
personnel-related 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 $14.8
million for the three months ended March 31, 2020, compared to $11.6 million
for the three months ended March 31,
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 Loss: Net loss was $40.0
million, or $1.08 basic and
diluted net loss per share, for the three months March 31, 2020, compared to net loss of
$32.2 million, or $0.89 basic and diluted net loss per share, for
the three months ended March 31,
2019.
Financial Guidance
Based on its current operating plan, REGENXBIO expects its
balance in cash, cash equivalents and marketable securities of
$356.6 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 3893881. 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, 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 trademark of Clearside Biomedical, Inc.
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)
|
|
|
|
March 31,
2020
|
|
|
December 31,
2019
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
69,411
|
|
|
$
|
69,514
|
|
Marketable
securities
|
|
|
209,846
|
|
|
|
226,696
|
|
Accounts
receivable
|
|
|
44,522
|
|
|
|
38,148
|
|
Prepaid
expenses
|
|
|
7,422
|
|
|
|
6,475
|
|
Other current
assets
|
|
|
7,032
|
|
|
|
4,199
|
|
Total current
assets
|
|
|
338,233
|
|
|
|
345,032
|
|
Marketable
securities
|
|
|
77,361
|
|
|
|
103,785
|
|
Accounts
receivable
|
|
|
4,373
|
|
|
|
4,155
|
|
Property and
equipment, net
|
|
|
30,414
|
|
|
|
28,973
|
|
Operating lease
right-of-use assets
|
|
|
9,375
|
|
|
|
10,078
|
|
Restricted
cash
|
|
|
1,330
|
|
|
|
1,330
|
|
Other
assets
|
|
|
2,850
|
|
|
|
4,555
|
|
Total
assets
|
|
$
|
463,936
|
|
|
$
|
497,908
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
9,008
|
|
|
$
|
6,409
|
|
Accrued expenses and
other current liabilities
|
|
|
20,327
|
|
|
|
24,846
|
|
Operating lease
liabilities
|
|
|
2,454
|
|
|
|
2,421
|
|
Total current
liabilities
|
|
|
31,789
|
|
|
|
33,676
|
|
Deferred
revenue
|
|
|
3,333
|
|
|
|
3,333
|
|
Operating lease
liabilities
|
|
|
7,990
|
|
|
|
8,874
|
|
Other
liabilities
|
|
|
672
|
|
|
|
1,828
|
|
Total
liabilities
|
|
|
43,784
|
|
|
|
47,711
|
|
Stockholders' equity
|
|
|
|
|
|
|
|
|
Preferred stock;
$0.0001 par value; 10,000 shares authorized, and no shares issued and
outstanding at March
31, 2020 and December 31, 2019
|
|
|
—
|
|
|
|
—
|
|
Common stock; $0.0001
par value; 100,000 shares authorized at March 31, 2020 and December 31,
2019; 37,190 and 36,992
shares issued and outstanding at March 31, 2020 and December 31, 2019,
respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
638,588
|
|
|
|
627,810
|
|
Accumulated other
comprehensive income (loss)
|
|
|
(580)
|
|
|
|
205
|
|
Accumulated
deficit
|
|
|
(217,860)
|
|
|
|
(177,822)
|
|
Total
stockholders' equity
|
|
|
420,152
|
|
|
|
450,197
|
|
Total liabilities and
stockholders' equity
|
|
$
|
463,936
|
|
|
$
|
497,908
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
Three Months Ended
March 31,
|
|
|
|
2020
|
|
|
2019
|
|
Revenues
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
|
$
|
17,644
|
|
|
$
|
884
|
|
Total
revenues
|
|
|
17,644
|
|
|
|
884
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
|
3,409
|
|
|
|
29
|
|
Research and
development
|
|
|
37,035
|
|
|
|
25,203
|
|
General and
administrative
|
|
|
14,833
|
|
|
|
11,558
|
|
Other operating
expenses
|
|
|
67
|
|
|
|
—
|
|
Total operating
expenses
|
|
|
55,344
|
|
|
|
36,790
|
|
Loss from
operations
|
|
|
(37,700)
|
|
|
|
(35,906)
|
|
Other Income
(Loss)
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
|
848
|
|
|
|
613
|
|
Investment income
(loss)
|
|
|
(3,186)
|
|
|
|
2,995
|
|
Total other income
(loss)
|
|
|
(2,338)
|
|
|
|
3,608
|
|
Loss before income
taxes
|
|
|
(40,038)
|
|
|
|
(32,298)
|
|
Income Tax
Benefit
|
|
|
—
|
|
|
|
70
|
|
Net loss
|
|
$
|
(40,038)
|
|
|
$
|
(32,228)
|
|
Other
Comprehensive Income (Loss)
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities, net
|
|
|
(785)
|
|
|
|
621
|
|
Total other
comprehensive income (loss)
|
|
|
(785)
|
|
|
|
621
|
|
Comprehensive
loss
|
|
$
|
(40,823)
|
|
|
$
|
(31,607)
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
|
$
|
(1.08)
|
|
|
$
|
(0.89)
|
|
Weighted-average
basic and diluted common shares outstanding
|
|
|
37,104
|
|
|
|
36,366
|
|
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
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SOURCE REGENXBIO Inc.