ROCKVILLE, Md., Aug. 6,
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 June
30, 2020, and recent operational highlights.
"Now halfway through 2020 and with the backdrop of the
challenging COVID-19 pandemic, I am pleased that we have made
significant progress in our gene therapy programs and continue to
make important decisions with respect to our pipeline and plans. We
expect to initiate the RGX-314 wet
AMD pivotal program later this year based on the latest data from
our RGX-314 Phase I/IIa trial, which has demonstrated durable
treatment effect, with stable to improved visual acuity and retinal
thickness. We also look forward to dosing patients in our RGX-314
trials using the suprachoroidal delivery approach for the treatment
of wet AMD and diabetic retinopathy later this year," said
Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "Our team has also been hard
at work moving our CNS-focused gene therapy programs forward. We
have completed dosing of patients in the second cohort of our Phase
I/II study of RGX-121 for the treatment of MPS II, which includes
one patient dosed in Brazil at our
first ex-U.S. gene therapy site. We look forward to providing
additional updates for this program, as well as our RGX-111 program
for the treatment of MPS I, later this year."
Mr. Mills continued: "We are committed to continuously
evaluating our portfolio of gene therapy candidates, with
innovation and patient needs in mind. Today, we have announced the
expansion of our pipeline to include RGX-381 for the treatment
of ocular manifestations of CLN2 disease, also known as Batten
disease. This will complement our existing RGX-181 program for the
treatment of CLN2 disease in the central nervous system. I look
forward to providing additional details and updates for RGX-381 and
RGX-181 in the coming months."
"Finally, we have made the difficult decision to discontinue the
development of RGX-501 for the treatment of HoFH. This program was
one of our first clinical-stage gene therapy programs, and we are
grateful for the support of the HoFH patient community over the
years. We will look for opportunities to support the continued
advancement of this program through business
development."
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)
-
- On August 4, 2020, REGENXBIO
reported data from the RGX-314 Phase I/IIa trial using subretinal
delivery. RGX-314 was generally well-tolerated as of July 13, 2020. For Cohorts 4 and 5:
-
- Durable treatment effect was observed with stable to improved
visual acuity and retinal thickness at one year
- Meaningful reductions in anti-VEGF treatment burden were
demonstrated over one year
- Dose-dependent intraocular RGX-314 protein expression levels
were observed over one year
- REGENXBIO expects to initiate a pivotal program for the
subretinal delivery of RGX-314 for the treatment of wet AMD by the
end of 2020.
- REGENXBIO plans to dose patients in AAVIATE, the Phase II trial
for the suprachoroidal delivery of RGX-314 using the SCS
Microinjector™ for the treatment of wet AMD, in the third quarter
of 2020.
-
- Interim data is expected to be reported from the first cohort
by the end of 2020.
- RGX-314 for the Treatment of Diabetic Retinopathy (DR)
-
- REGENXBIO expects to initiate a Phase II trial in the second
half of 2020 and interim data is expected to be reported in
2021.
- Research Program for the Treatment of Hereditary Angioedema
(HAE)
-
- Preclinical data from the HAE program was presented at the
American Society of Gene and Cell Therapy (ASGCT) 23rd Annual
Meeting in May 2020, demonstrating
biological activity in a model of inflammatory edema and inhibition
of kallikrein comparable to other anti-kallikrein treatments.
- REGENXBIO expects to provide a program update in the second
half of 2020.
- Research Program for the Treatment of Neurodegenerative
Diseases
-
- REGENXBIO continues to collaborate 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)
-
- In July, REGENXBIO announced the completed dosing of three
patients in Cohort 2 of its Phase I/II trial. As of June 24, 2020, RGX-121 was reported to be
well-tolerated in patients across two dose levels, with no
drug-related serious adverse events (SAEs).
- The Company expects to provide a data and program update in the
second half of 2020.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS
I)
-
- In July, REGENXBIO reported data under a single-patient
investigator-initiated Investigational New Drug application (IND)
conducted at CHOC Children's, which demonstrated encouraging
biomarker activity and continued progression of neurocognitive
development. As of June 9, 2020,
RGX-111 was reported to be well-tolerated in this patient, with no
drug-related SAEs.
- Recruitment and patient screening are ongoing in the Company'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
-
- RGX-381 is a new program targeting the ocular manifestations of
CLN2 disease in patients and is designed to use the AAV9 vector to
deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the
retina. REGENXBIO believes that one-time administration of RGX-381
could provide a durable source of TPP1 activity in the retina,
thereby potentially preventing visual decline. There is currently
no available treatment for ocular manifestations of CLN2
disease.
- REGENXBIO expects to submit an IND for a Phase I/II study of
RGX-381 in patients with CLN2 disease in the second half of 2020
and plans to initiate enrollment in the first half of 2021.
- The company has initiated two non-interventional natural
history studies to assess ocular manifestations in CLN2
disease.
- The Orphan Drug Designation and Rare Pediatric Disease
Designation granted to the RGX-181 program also apply to the
RGX-381 program.
- RGX-181 for the Treatment of CLN2 Disease
-
- REGENXBIO expects to submit an IND for the intracisternal
delivery of RGX-181 by the end of 2020, and plans to initiate
enrollment in a Phase I/II trial in the first half of 2021.
- RGX-501 for the Treatment of Homozygous Familial
Hypercholesterolemia (HoFH)
-
- REGENXBIO has discontinued internal clinical development of
RGX-501 for the treatment of HoFH in order to focus on furthering
other gene therapy programs in its pipeline. The Company plans to
evaluate strategic alternatives to support the continued
advancement of this program, including through partnering.
- 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 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 late 2021.
Marketed NAV Technology Product Highlights
REGENXBIO's NAV Technology Platform is being applied in one
marketed product, Zolgensma®. On July 21, 2020, Novartis AG reported second
quarter 2020 U.S. Zolgensma sales revenue of $205 million, and REGENXBIO recognized royalty
revenue of $12 million in the second
quarter of 2020 as a result of these sales. Total net sales of
Zolgensma since launch in May 2019
are more than $735 million. REGENXBIO
is eligible to receive a milestone payment of $80.0 million from Novartis upon the achievement
of $1.0 billion in cumulative net
sales of Zolgensma.
Financial Results
Cash Position: Cash, cash equivalents and marketable
securities were $339.2 million as of
June 30, 2020, compared to
$400.0 million as of December 31, 2019. The decrease was
primarily attributable to net cash flows used in operating
activities of $57.1 million.
Revenues: Revenues were $16.6
million for the three months ended June 30, 2020, compared to $7.9 million for the three months ended
June 30, 2019. The increase was
primarily attributable to an $11.0
million increase in royalty revenue recognized on net sales
of Zolgensma in the second quarter of 2020 as compared to the
second quarter of 2019.
Research and Development Expenses: Research and
development expenses were $38.1
million for the three months ended June 30, 2020, compared to $29.5 million for the three months ended
June 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, laboratory and facilities
costs, and externally sourced services for preclinical, regulatory
and manufacturing-related activities.
General and Administrative Expenses: General and
administrative expenses were $15.6
million for the three months ended June 30, 2020, compared to $13.4 million for the three months ended
June 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 Loss: Net loss was $33.8
million, or $0.91 basic and
diluted net loss per share, for the three months ended June 30, 2020, compared to net loss of
$1.5 million, or $0.04 basic and diluted net loss per share, for
the three months ended June 30,
2019.
Financial Guidance
Based on its current operating plan, REGENXBIO expects its
balance in cash, cash equivalents and marketable securities of
$339.2 million to fund the completion
of its internal manufacturing capabilities and clinical advancement
of its product candidates into 2022.
Q2 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 (800)
708-4539 (domestic) or (847) 619-6396 (international) and enter the
passcode 49857700. 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 trademark of Clearside Biomedical, Inc.
Zolgensma® is a registered trademark of AveXis, Inc. All
other trademarks referenced herein are registered trademarks of
REGENXBIO.
REGENXBIO
INC.
CONSOLIDATED
BALANCE SHEETS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
|
June 30,
2020
|
|
December 31,
2019
|
Assets
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
Cash and cash
equivalents
|
$
|
94,222
|
|
$
|
69,514
|
Marketable
securities
|
|
174,964
|
|
|
226,696
|
Accounts
receivable
|
|
42,876
|
|
|
38,148
|
Prepaid
expenses
|
|
9,749
|
|
|
6,475
|
Other current
assets
|
|
7,271
|
|
|
4,199
|
Total current
assets
|
|
329,082
|
|
|
345,032
|
Marketable
securities
|
|
70,054
|
|
|
103,785
|
Accounts
receivable
|
|
3,618
|
|
|
4,155
|
Property and
equipment, net
|
|
32,696
|
|
|
28,973
|
Operating lease
right-of-use assets
|
|
8,635
|
|
|
10,078
|
Restricted
cash
|
|
1,330
|
|
|
1,330
|
Other
assets
|
|
4,323
|
|
|
4,555
|
Total
assets
|
$
|
449,738
|
|
$
|
497,908
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
Accounts
payable
|
$
|
10,901
|
|
$
|
6,409
|
Accrued expenses and
other current liabilities
|
|
25,839
|
|
|
24,846
|
Deferred
revenue
|
|
450
|
|
|
—
|
Operating lease
liabilities
|
|
3,013
|
|
|
2,421
|
Total current
liabilities
|
|
40,203
|
|
|
33,676
|
Deferred
revenue
|
|
4,007
|
|
|
3,333
|
Operating lease
liabilities
|
|
7,085
|
|
|
8,874
|
Other
liabilities
|
|
582
|
|
|
1,828
|
Total
liabilities
|
|
51,877
|
|
|
47,711
|
Stockholders' equity
|
|
|
|
|
|
Preferred stock;
$0.0001 par value; 10,000 shares
authorized, and no
shares issued and outstanding
at June 30, 2020 and
December 31, 2019
|
|
—
|
|
|
—
|
Common stock; $0.0001
par value; 100,000 shares
authorized at June
30, 2020 and December 31, 2019;
37,291 and 36,992
shares issued and outstanding at
June 30, 2020 and
December 31, 2019, respectively
|
|
4
|
|
|
4
|
Additional paid-in
capital
|
|
648,729
|
|
|
627,810
|
Accumulated other
comprehensive income
|
|
750
|
|
|
205
|
Accumulated
deficit
|
|
(251,622)
|
|
|
(177,822)
|
Total
stockholders' equity
|
|
397,861
|
|
|
450,197
|
Total liabilities and
stockholders' equity
|
$
|
449,738
|
|
$
|
497,908
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
|
Three Months Ended
June 30,
|
|
Six Months Ended
June 30,
|
|
2020
|
|
|
2019
|
|
2020
|
|
|
2019
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
$
|
16,566
|
|
|
$
|
7,881
|
|
$
|
34,210
|
|
|
$
|
8,765
|
Total
revenues
|
|
16,566
|
|
|
|
7,881
|
|
|
34,210
|
|
|
|
8,765
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
4,684
|
|
|
|
1,927
|
|
|
8,093
|
|
|
|
1,956
|
Research and
development
|
|
38,111
|
|
|
|
29,483
|
|
|
75,146
|
|
|
|
54,686
|
General and
administrative
|
|
15,554
|
|
|
|
13,405
|
|
|
30,387
|
|
|
|
24,963
|
Other operating
expenses (income)
|
|
50
|
|
|
|
(62)
|
|
|
117
|
|
|
|
(62)
|
Total operating
expenses
|
|
58,399
|
|
|
|
44,753
|
|
|
113,743
|
|
|
|
81,543
|
Loss from
operations
|
|
(41,833)
|
|
|
|
(36,872)
|
|
|
(79,533)
|
|
|
|
(72,778)
|
Other
Income
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
1,849
|
|
|
|
762
|
|
|
2,697
|
|
|
|
1,375
|
Investment
income
|
|
5,722
|
|
|
|
34,524
|
|
|
2,536
|
|
|
|
37,519
|
Total other
income
|
|
7,571
|
|
|
|
35,286
|
|
|
5,233
|
|
|
|
38,894
|
Loss before income
taxes
|
|
(34,262)
|
|
|
|
(1,586)
|
|
|
(74,300)
|
|
|
|
(33,884)
|
Income Tax
Benefit
|
|
500
|
|
|
|
129
|
|
|
500
|
|
|
|
199
|
Net loss
|
$
|
(33,762)
|
|
|
$
|
(1,457)
|
|
$
|
(73,800)
|
|
|
$
|
(33,685)
|
Other
Comprehensive Income
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain on
available-for-sale securities, net
|
|
1,330
|
|
|
|
530
|
|
|
545
|
|
|
|
1,151
|
Total other
comprehensive income
|
|
1,330
|
|
|
|
530
|
|
|
545
|
|
|
|
1,151
|
Comprehensive
loss
|
$
|
(32,432)
|
|
|
$
|
(927)
|
|
$
|
(73,255)
|
|
|
$
|
(32,534)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share
|
$
|
(0.91)
|
|
|
$
|
(0.04)
|
|
$
|
(1.98)
|
|
|
$
|
(0.92)
|
Weighted-average
basic and diluted common shares outstanding
|
|
37,257
|
|
|
|
36,669
|
|
|
37,180
|
|
|
|
36,518
|
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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SOURCE REGENXBIO Inc.