ROCKVILLE, Md., Aug. 7, 2019 /PRNewswire/ --
- Reports further positive interim update from RGX-314 Phase
I/IIa trial for Wet AMD
-
- Well-tolerated at all doses
- Dose-dependent protein expression levels observed across all
five dose cohorts
- 50% of subjects treated in Cohort 3 remain free of anti-VEGF
injections at 18 months
- Company plans to present interim data update from all five
dose cohorts in October 2019
- Remains on-track to initiate Phase IIb trial in late
2019
- Continues dosing in the RGX-501 Phase I/II trial for HoFH
and the RGX-121 Phase I/II trial for MPS II
- Expanded pipeline using NAV® Vectors to deliver
therapeutic antibodies for the treatment of hereditary angioedema
and neurodegenerative diseases
- Launch of first FDA-approved gene therapy based on
REGENXBIO's NAV Technology Platform, Novartis'
Zolgensma® for the treatment of SMA
- $450 million in cash, cash
equivalents and marketable securities as of June 30, 2019
- Webcast and conference call scheduled for today 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 quarter ended June 30,
2019, and recent operational highlights.
"We are encouraged by the continued positive interim data from
the Phase I/IIa RGX-314 trial and the potential of NAV gene therapy
as a one-time treatment for wet AMD, and look forward to sharing
six-month results from all five cohorts in the trial in
October 2019," said Kenneth T. Mills, President and Chief Executive
Officer of REGENXBIO. "In our clinical trial for wet AMD, we
have seen dose-dependent increases in protein expression levels
across cohorts, and 50% of Cohort 3 subjects continued to be free
of anti-VEGF injections at 18 months following a single
administration of our gene therapy. These findings are particularly
encouraging, given the severity of the disease and the high
anti-VEGF injection treatment burden of these enrolled patients
prior to administration of RGX-314. With feedback from the FDA
provided at our recent Type B meeting, we also look forward to our
anticipated start of the Phase IIb trial in late 2019."
Mr. Mills added: "In addition to our excitement around RGX-314,
the past quarter was highlighted by important milestones for
REGENXBIO, including the FDA approval and launch of
Zolgensma®—the first FDA-approved gene therapy based on
REGENXBIO's NAV Technology Platform. We recently announced the
expansion of our pipeline using NAV Vectors to deliver therapeutic
antibodies, beginning with novel treatments for hereditary
angioedema and chronic neurodegenerative diseases; this approach
has enormous potential for patients who lack treatments or who are
currently underserved by existing therapies. We currently have an
internal pipeline of novel gene therapy candidates across large,
chronic diseases and rare, monogenic diseases, which positions us
to potentially develop meaningful treatments for a broad spectrum
of patients with significant unmet needs."
Lead Product Candidate Updates
Gene Therapy using NAV Vectors for AAV-Mediated Antibody
Delivery:
- RGX-314 for the Treatment of Wet AMD
-
- As of July 31, 2019, 42 subjects
across five dose cohorts have been treated in the Phase I/IIa trial
of RGX-314. RGX-314 continues to be well-tolerated across all five
dose cohorts, with no drug related serious adverse events (SAEs)
reported.
- Dose-dependent increases in RGX-314 protein expression levels,
as measured from aqueous samples by electrochemiluminescence
immunoassay (ECL) at approximately one month after administration
of RGX-314, have been observed across all doses.
- 50% of subjects (3/6) in Cohort 3 continue to remain
injection-free at 18 months.
- REGENXBIO expects to present long-term clinical data from
Cohort 3 out to 18 months and interim data from Cohort 4 and Cohort
5 in October 2019.
- Following the Type B meeting with the U.S. Food and Drug
Administration (FDA) held in July 2019, REGENXBIO remains
on track to initiate a Phase IIb trial for wet AMD in late
2019.
- RGX-314 for the Treatment of Diabetic Retinopathy (DR)
-
- REGENXBIO is on track to file a new Investigational New Drug
(IND) application for a Phase II trial evaluating RGX-314 in
subjects with DR in the second half of 2019.
- Hereditary Angioedema (HAE)
-
- REGENXBIO continues work on the HAE program and expects to
provide an update in early 2020 on the preclinical studies, as well
as plans for entering clinical trials.
- Neurodegenerative Disease
-
- REGENXBIO, in collaboration with Neurimmune AG, has initiated
work to jointly develop novel therapies using AAV vectors to
deliver human antibodies targeting abnormal tau and will provide
program updates as the collaboration advances.
Gene Therapy using NAV Vectors for Rare Genetic
Diseases:
- RGX-501 for the Treatment of Homozygous Familial
Hypercholesterolemia (HoFH)
-
- Dosing resumed and recruitment continues in the Phase I/II
clinical trial evaluating RGX-501 for the treatment of HoFH.
- REGENXBIO expects to report interim data from Cohort 2 with
corticosteroid prophylaxis from the Phase I/II clinical trial in
the second half of 2019.
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS
II)
-
- Recruitment, screening, and dosing continue in the first of two
dose cohorts in the Phase I/II clinical trial evaluating RGX-121
for the treatment of MPS II.
- REGENXBIO expects to present an interim data update from the
Phase I/II clinical trial in the second half of 2019.
- RGX-181 for the Treatment of Late-infantile Neuronal Ceroid
Lipofuscinosis Type 2 (CLN2) Disease
-
- REGENXBIO expects to file an IND or foreign equivalent for the
first-in-human clinical trial evaluating RGX-181 for the treatment
of CLN2 in the second half of 2019.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS
I)
-
- Recruitment, screening and additional site activations are
ongoing in the Phase I clinical trial evaluating RGX-111 for the
treatment of MPS I.
Other Recent Operational Highlights
- In May 2019, REGENXBIO announced
its plans to construct a current good manufacturing practice (cGMP)
production facility in Rockville,
Maryland. The new cGMP production facility will be
integrated into REGENXBIO's previously announced new 139,000 square
foot headquarters, for which construction is currently underway,
and will allow for production of NAV Technology-based vectors at
scales up to 2,000 liters using REGENXBIO's platform suspension
cell culture process. The facility will be designed to meet global
regulatory requirements and is expected to be operational in
2021.
NAV Technology Licensee Program Highlights
As of July 31, 2019, REGENXBIO's
NAV Technology Platform was being applied in one marketed product,
Novartis AG's Zolgensma, and more than 20 partnered product
candidates in development by NAV Technology Licensees. Fifteen of
these partnered product candidates are in active clinical
development. Over 200 subjects have been treated in clinical trials
sponsored by NAV Technology Licensees. 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 Products
- In May 2019, Novartis announced
the FDA approval of Zolgensma as a one-time infusion for pediatric
patients with spinal muscular atrophy (SMA) who are less than two
years of age. Novartis has stated that it
anticipates regulatory approvals of Zolgensma in Japan
and Europe in the second half of 2019. REGENXBIO earned a
$3.5 million milestone payment upon
Zolgensma's approval in the United
States and is eligible to receive an additional $80 million milestone payment upon the
achievement of $1 billion in
cumulative net sales of Zolgensma. REGENXBIO will receive tiered
royalties on sales of Zolgensma up to a low double-digit
percentage. Zolgensma uses the NAV AAV9 vector.
Partnered Product Candidates
- In July 2019, REGENXBIO announced
that it granted a non-exclusive worldwide license for its NAV AAV9
vector to Pfizer Inc. for the development and commercialization of
gene therapy for the treatment of Friedreich's ataxia, the most
common hereditary ataxia. In return, Pfizer will provide REGENXBIO
with an upfront payment, and REGENXBIO has the potential to receive
ongoing fees, development and commercial milestone payments and
royalties on net sales of products incorporating the NAV AAV9
vector.
- In July 2019, Abeona
Therapeutics, Inc. announced positive data from its ongoing Phase
I/II clinical trial evaluating ABO-102 for the treatment of
Sanfilippo syndrome type A (MPS IIIA). ABO-102 uses the NAV
AAV9 vector.
- In June 2019, Rocket
Pharmaceuticals, Inc. announced it dosed the first subject in its
Phase I clinical trial of RP-A501 for the treatment of Danon
disease. RP-A501 uses the NAV AAV9 vector.
Financial Results
Cash, cash equivalents and marketable securities were
$449.7 million as of June 30, 2019, compared to $470.6 million as of December 31, 2018. Cash, cash equivalents and
marketable securities as of June 30,
2019 include $32.1 million of
marketable equity securities of Prevail Therapeutics Inc. REGENXBIO
is a co-founder of Prevail and currently owns 2.43 million shares
of Prevail common stock. Prior to Prevail's initial public offering
(IPO) in June 2019, REGENXBIO's
equity interests in Prevail had a carrying value of $0.4 million and were included in other assets on
the consolidated balance sheet. Upon the completion of Prevail's
IPO in June 2019, the securities were
reclassified to marketable securities, and subsequently remeasured
at fair value. The decrease in cash, cash equivalents and
marketable securities during the six months ended June 30, 2019, was primarily attributable to
$53.0 million of net cash used in
operating activities during the period, partially offset by the
unrealized gain of $31.7 million
related to the marketable equity securities of Prevail.
Revenues were $7.9 million for the
three months ended June 30, 2019,
compared to $40.0 million for the
three months ended June 30, 2018. The
decrease was primarily attributable to $40.0
million of non-recurring license revenue recognized during
the three months ended June 30, 2018
under our amended license agreement with AveXis Inc. for the
development and commercialization of treatments for SMA. The
decrease was partially offset by revenue recognized during the
three months ended June 30, 2019
resulting from license options exercised and development milestones
achieved by licensees during the period, as well as royalties on
net sales of Zolgensma. Commercial sales of Zolgensma commenced in
the second quarter of 2019, and we expect royalties to increase in
future periods. We are also eligible to receive a milestone payment
of $80.0 million from AveXis upon the
achievement of $1 billion in
cumulative net sales of Zolgensma.
Research and development expenses were $29.5 million for the three months ended
June 30, 2019, compared to
$21.5 million for the three months
ended June 30, 2018. The increase was
primarily attributable to personnel costs as a result of increased
headcount, laboratory and facilities costs and external expenses
associated with conducting clinical trials and
manufacturing-related services.
General and administrative expenses were $13.4 million for the three months ended
June 30, 2019, compared to
$8.3 million for the three months
ended June 30, 2018. The increase was
primarily attributable to personnel costs as a result of increased
headcount and professional fees for advisory and other
services.
Net loss was $1.5 million, or
$0.04 basic and diluted net loss per
share, for the three months ended June 30,
2019, compared to net income of $10.6
million, or $0.33 basic and
$0.30 diluted net income per share,
for the three months ended June 30,
2018. Net loss for the three months ended June 30, 2019, includes the unrealized gain of
$31.7 million on REGENXBIO's
marketable equity securities of Prevail recognized during the
period.
Financial Guidance
Based on its current operating plan, REGENXBIO expects its
balance in cash, cash equivalents and marketable securities to be
at least $350 million as of
December 31, 2019.
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 6839996. 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,
research and development activities, preclinical studies, 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, 2018, 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.
REGENXBIO
INC.
|
CONSOLIDATED
BALANCE SHEETS
|
(unaudited)
|
(in thousands,
except per share data)
|
|
|
June 30,
2019
|
|
|
December 31,
2018
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
55,142
|
|
|
$
|
75,561
|
|
Marketable
securities
|
|
|
286,354
|
|
|
|
244,200
|
|
Accounts
receivable
|
|
|
9,679
|
|
|
|
8,587
|
|
Prepaid
expenses
|
|
|
6,036
|
|
|
|
5,734
|
|
Other current
assets
|
|
|
2,281
|
|
|
|
3,831
|
|
Total current
assets
|
|
|
359,492
|
|
|
|
337,913
|
|
Marketable
securities
|
|
|
108,194
|
|
|
|
150,819
|
|
Accounts
receivable
|
|
|
23,955
|
|
|
|
23,012
|
|
Property and
equipment, net
|
|
|
27,330
|
|
|
|
28,702
|
|
Operating lease
right-of-use assets
|
|
|
5,904
|
|
|
|
—
|
|
Restricted
cash
|
|
|
1,053
|
|
|
|
1,053
|
|
Other
assets
|
|
|
3,211
|
|
|
|
2,315
|
|
Total
assets
|
|
$
|
529,139
|
|
|
$
|
543,814
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
5,851
|
|
|
$
|
4,412
|
|
Accrued expenses and
other current liabilities
|
|
|
16,534
|
|
|
|
17,164
|
|
Deferred
revenue
|
|
|
—
|
|
|
|
600
|
|
Operating lease
liabilities
|
|
|
2,276
|
|
|
|
—
|
|
Total current
liabilities
|
|
|
24,661
|
|
|
|
22,176
|
|
Deferred
revenue
|
|
|
3,333
|
|
|
|
3,333
|
|
Operating lease
liabilities
|
|
|
4,654
|
|
|
|
—
|
|
Deferred
rent
|
|
|
—
|
|
|
|
1,098
|
|
Financing lease
obligations
|
|
|
—
|
|
|
|
5,854
|
|
Other
liabilities
|
|
|
1,899
|
|
|
|
2,505
|
|
Total
liabilities
|
|
|
34,547
|
|
|
|
34,966
|
|
Stockholders' equity
|
|
|
|
|
|
|
|
|
Preferred stock;
$0.0001 par value; 10,000 shares authorized,
and no shares issued and
outstanding at June 30, 2019 and December 31, 2018
|
|
|
—
|
|
|
|
—
|
|
Common stock; $0.0001
par value; 100,000 shares authorized at June 30, 2019 and December 31, 2018;
36,752 and
36,120 shares issued and outstanding at June 30,
2019 and
December 31, 2018, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
610,891
|
|
|
|
592,580
|
|
Accumulated other
comprehensive income (loss)
|
|
|
471
|
|
|
|
(720)
|
|
Accumulated
deficit
|
|
|
(116,774)
|
|
|
|
(83,016)
|
|
Total
stockholders' equity
|
|
|
494,592
|
|
|
|
508,848
|
|
Total liabilities and
stockholders' equity
|
|
$
|
529,139
|
|
|
$
|
543,814
|
|
REGENXBIO
INC.
|
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME
(LOSS)
|
(unaudited)
|
(in thousands,
except per share data)
|
|
|
|
|
|
|
|
|
|
Three Months Ended
June 30,
|
|
|
Six Months Ended
June 30,
|
|
|
|
2019
|
|
|
2018
|
|
|
2019
|
|
|
2018
|
|
Revenues
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
|
$
|
7,881
|
|
|
$
|
40,031
|
|
|
$
|
8,765
|
|
|
$
|
172,422
|
|
Total
revenues
|
|
|
7,881
|
|
|
|
40,031
|
|
|
|
8,765
|
|
|
|
172,422
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
|
1,927
|
|
|
|
3,872
|
|
|
|
1,956
|
|
|
|
6,280
|
|
Research and
development
|
|
|
29,483
|
|
|
|
21,486
|
|
|
|
54,686
|
|
|
|
41,036
|
|
General and
administrative
|
|
|
13,405
|
|
|
|
8,318
|
|
|
|
24,963
|
|
|
|
16,698
|
|
Other operating
expenses (income)
|
|
|
(62)
|
|
|
|
5
|
|
|
|
(62)
|
|
|
|
33
|
|
Total operating
expenses
|
|
|
44,753
|
|
|
|
33,681
|
|
|
|
81,543
|
|
|
|
64,047
|
|
Income (loss) from
operations
|
|
|
(36,872)
|
|
|
|
6,350
|
|
|
|
(72,778)
|
|
|
|
108,375
|
|
Other
Income
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
|
762
|
|
|
|
6,898
|
|
|
|
1,375
|
|
|
|
8,253
|
|
Investment
income
|
|
|
34,524
|
|
|
|
1,196
|
|
|
|
37,519
|
|
|
|
2,055
|
|
Total other
income
|
|
|
35,286
|
|
|
|
8,094
|
|
|
|
38,894
|
|
|
|
10,308
|
|
Income (loss) before
income taxes
|
|
|
(1,586)
|
|
|
|
14,444
|
|
|
|
(33,884)
|
|
|
|
118,683
|
|
Income Tax Benefit
(Expense)
|
|
|
129
|
|
|
|
(3,850)
|
|
|
|
199
|
|
|
|
(3,850)
|
|
Net income
(loss)
|
|
$
|
(1,457)
|
|
|
$
|
10,594
|
|
|
$
|
(33,685)
|
|
|
$
|
114,833
|
|
Other
Comprehensive Income (Loss)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities, net of reclassifications
and
income tax
expense
|
|
|
530
|
|
|
|
132
|
|
|
|
1,151
|
|
|
|
(56)
|
|
Total other
comprehensive income (loss)
|
|
|
530
|
|
|
|
132
|
|
|
|
1,151
|
|
|
|
(56)
|
|
Comprehensive income
(loss)
|
|
$
|
(927)
|
|
|
$
|
10,726
|
|
|
$
|
(32,534)
|
|
|
$
|
114,777
|
|
Net income (loss) per
share:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
$
|
(0.04)
|
|
|
$
|
0.33
|
|
|
$
|
(0.92)
|
|
|
$
|
3.60
|
|
Diluted
|
|
$
|
(0.04)
|
|
|
$
|
0.30
|
|
|
$
|
(0.92)
|
|
|
$
|
3.29
|
|
Weighted-average
common shares outstanding:
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic
|
|
|
36,669
|
|
|
|
32,082
|
|
|
|
36,518
|
|
|
|
31,858
|
|
Diluted
|
|
|
36,669
|
|
|
|
35,272
|
|
|
|
36,518
|
|
|
|
34,884
|
|
Zolgensma® is a registered
trademark of AveXis. All other trademarks referenced herein
are registered trademarks of REGENXBIO.
Contacts:
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-second-quarter-2019-financial-and-operating-results-and-additional-positive-interim-phase-iiia-trial-update-for-rgx-314-for-the-treatment-of-wet-amd-300898256.html
SOURCE REGENXBIO Inc.