REGENXBIO Reports Second Quarter 2017 Financial Results and Recent Operational Highlights
August 08 2017 - 4:05PM
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 second quarter
ended June 30, 2017 and recent operational highlights.
“We continue to progress our lead product candidates, most
notably with the continued enrollment in our Phase I clinical trial
evaluating RGX-314 for the treatment of wet AMD and in our Phase
I/II clinical trial evaluating RGX-501 for the treatment of HoFH.
The active IND for RGX-111 for the treatment of MPS I – our third
clinical-stage lead product candidate – is another pipeline
achievement that demonstrates our strong internal capabilities and
commitment to the development of a robust clinical pipeline of gene
therapy product candidates in many diseases,” said Kenneth T.
Mills, President and Chief Executive Officer of REGENXBIO. "We
continue to advance our internal and partnered gene therapy product
candidates, leveraging our proprietary NAV Technology Platform to
demonstrate the curative potential of one-time administrations of
gene therapy. We look forward to sharing more pipeline updates,
including interim updates from the RGX-314 Phase I clinical trial
for wet AMD and the RGX-501 Phase I/II clinical trial for HoFH, by
year-end 2017.”
Recent Operational Highlights
- REGENXBIO initiated dosing in the Phase I clinical trial
evaluating RGX-314 for the treatment of wet age-related macular
degeneration (wet AMD). Patient recruitment and enrollment is
continuing and REGENXBIO expects to provide an interim update from
the RGX-314 clinical trial by the end of 2017.
- Patient recruitment and enrollment is continuing in the Phase
I/II clinical trial evaluating RGX-501 for the treatment of
homozygous familial hypercholesterolemia (HoFH). REGENXBIO expects
to provide an interim update from the RGX-501 clinical trial by the
end of 2017.
- REGENXBIO today announced that the Investigational New Drug
(IND) application for the Phase I clinical trial of RGX-111 for the
treatment of Mucopolysaccharidosis Type I (MPS I) is active. Site
activation in this planned multi-center, open-label,
multiple-cohort, dose-escalation trial is underway to support
recruitment and patient enrollment, with the first patient expected
to be dosed in the first half of 2018.
- REGENXBIO plans to file an IND application for RGX-121 for the
treatment of Mucopolysaccharidosis Type II (MPS II) in the second
half of 2017, which will incorporate feedback received from the
U.S. Food and Drug Administration (FDA) in its review of the IND
application for RGX-111.
- REGENXBIO has completed production of investigational product
for all four of its lead product candidates in an amount which is
expected to supply planned clinical trials through 2018 and has
established a supply chain to ensure effective distribution to
REGENXBIO’s planned clinical trial sites worldwide.
- As of June 30, 2017, REGENXBIO’s NAV Technology Platform was
being applied in the development of more than 20 partnered product
candidates by nine NAV Technology Licensees. Eight of these
partnered product candidates have advanced to clinical stages of
development.○ In June 2017, AveXis, Inc. announced alignment
with the FDA on its Good Manufacturing Practice (GMP) commercial
manufacturing process for AVXS-101 for the treatment of spinal
muscular atrophy (SMA) Type 1. AveXis announced that it plans to
initiate the pivotal trial later in the third quarter of 2017.
AVXS-101 uses the NAV AAV9 vector.○ Also in June 2017,
REGENXBIO and AveXis entered into an exclusive worldwide license
agreement for AveXis to develop and commercialize gene therapy
treatments using the NAV AAV9 vector to treat two rare neurological
monogenic disorders: Rett syndrome (RTT) and amyotrophic lateral
sclerosis (ALS).○ In July 2017, Shire plc announced the
submission of an IND application for SHP654, an investigational
factor VIII gene therapy for the treatment of hemophilia A. SHP654
uses the NAV AAV8 vector.
Financial Results
Cash, cash equivalents and marketable securities were $208.5
million as of June 30, 2017, compared to $159.0 million as of
December 31, 2016.
Revenues were $6.6 million for the three months ended June 30,
2017, compared to $2.4 million for the three months ended June 30,
2016.
Total operating expenses were $21.6 million for the three months
ended June 30, 2017, compared to $17.3 million for the three months
ended June 30, 2016.
Net loss was $14.5 million, or $0.47 net loss per basic and
diluted common share, for the three months ended June 30, 2017,
compared to $14.4 million, or $0.55 net loss per basic and diluted
share, for the three months ended June 30, 2016.
Financial Guidance
REGENXBIO now expects full-year 2017 cash burn to be between $75
million and $80 million, which will support the continued
development of its lead product candidate programs. Full-year 2017
cash burn guidance excludes the effect of REGENXBIO’s previously
announced underwritten public offering of common stock in March
2017 and the underwriters’ exercise of their option to purchase
additional shares in April 2017, which resulted in aggregate net
proceeds to REGENXBIO of approximately $81.5 million, after
deducting underwriting discounts and commissions and offering
expenses.
Conference Call Information
In connection with the earnings release, REGENXBIO will host a
conference call 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 53241624. 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 contains “forward-looking statements,” within
the meaning of the Private Securities Litigation Reform Act of
1995, regarding, among other things, REGENXBIO’s research,
development and regulatory plans in connection with its NAV
Technology Platform and gene therapy treatments, the research,
development and regulatory plans of REGENXBIO’s NAV Technology
Licensees and REGENXBIO’s cash burn. Such forward-looking
statements are based on current expectations and involve inherent
risks and uncertainties, including factors that could cause actual
results to differ materially from those projected by such
forward-looking statements. All of REGENXBIO’s development
timelines could be subject to adjustment depending on recruitment
rate, regulatory agency review and other factors that could delay
the initiation and completion of clinical trials. Meaningful
factors which could cause actual results to differ include, but are
not limited to, the timing of enrollment, commencement and
completion of REGENXBIO’s clinical trials; the timing and success
of preclinical studies and clinical trials conducted
by REGENXBIO and its development partners; the ability to
obtain and maintain regulatory approval of REGENXBIO’s product
candidates, and the labeling for any approved products; the scope,
progress, expansion, and costs of developing and commercializing
REGENXBIO’s product candidates; REGENXBIO’s ability to obtain and
maintain intellectual property protection for REGENXBIO’s product
candidates and technology; REGENXBIO’s growth strategies;
REGENXBIO’s competition; trends and challenges in REGENXBIO’s
business and the markets in which REGENXBIO operates;
REGENXBIO’s ability to attract or retain key personnel; the size
and growth of the potential markets for REGENXBIO’s product
candidates and the ability to serve those markets; the rate and
degree of market acceptance of any of REGENXBIO’s product
candidates; REGENXBIO’s ability to establish and maintain
development partnerships; REGENXBIO’s expenses and revenue;
regulatory developments in the United States and foreign
countries; the sufficiency of REGENXBIO’s cash resources and needs
for additional financing; and other factors discussed in 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, 2017 and
Quarterly Report on Form 10-Q for the quarter ended March 31, 2017,
which have been filed with the Securities and Exchange Commission
(SEC) and are available on the SEC’s website at www.sec.gov.
Additional factors may be set forth in those sections of
REGENXBIO’s Quarterly Report on Form 10-Q for the quarter
ended June 30, 2017, which will be filed with the SEC in the
third quarter of 2017. In addition to the risks described above and
in REGENXBIO’s filings with the SEC, other unknown or
unpredictable factors also could affect REGENXBIO’s results. There
can be no assurance that the actual results or developments
anticipated by REGENXBIO will be realized or, even if
substantially realized, that they will have the expected
consequences to, or effects on, REGENXBIO. Therefore, no
assurance can be given that the outcomes stated in such
forward-looking statements and estimates will be achieved.
All forward-looking statements contained in this press release
are expressly qualified by the cautionary statements contained or
referred to herein. REGENXBIO cautions investors not to
rely too heavily on the forward-looking
statements REGENXBIO makes or that are made on its
behalf. These forward-looking statements speak only as of the date
of this press release (unless another date is
indicated). REGENXBIO undertakes no obligation, and
specifically declines any obligation, to publicly update or revise
any such 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, 2017 |
|
|
December 31, 2016 |
|
Assets |
|
|
|
|
|
|
|
|
Current assets |
|
|
|
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
57,649 |
|
|
$ |
24,840 |
|
Marketable securities |
|
|
104,434 |
|
|
|
64,714 |
|
Accounts
receivable |
|
|
50 |
|
|
|
1,032 |
|
Prepaid
expenses |
|
|
2,432 |
|
|
|
1,775 |
|
Other
current assets |
|
|
1,252 |
|
|
|
1,010 |
|
Total
current assets |
|
|
165,817 |
|
|
|
93,371 |
|
Marketable
securities |
|
|
46,417 |
|
|
|
69,412 |
|
Property and equipment,
net |
|
|
11,524 |
|
|
|
9,324 |
|
Restricted cash |
|
|
225 |
|
|
|
225 |
|
Other assets |
|
|
393 |
|
|
|
400 |
|
Total
assets |
|
$ |
224,376 |
|
|
$ |
172,732 |
|
Liabilities and
Stockholders’
Equity |
|
|
|
|
|
|
|
|
Current
liabilities |
|
|
|
|
|
|
|
|
Accounts
payable |
|
$ |
3,948 |
|
|
$ |
1,543 |
|
Accrued
expenses and other current liabilities |
|
|
7,514 |
|
|
|
8,126 |
|
Total
current liabilities |
|
|
11,462 |
|
|
|
9,669 |
|
Deferred rent, net of
current portion |
|
|
1,217 |
|
|
|
1,326 |
|
Total
liabilities |
|
|
12,679 |
|
|
|
10,995 |
|
Stockholders’
equity |
|
|
|
|
|
|
|
|
Preferred
stock; $0.0001 par value; 10,000 shares authorized, and no
shares issued and outstanding at June 30, 2017 and December
31, 2016 |
|
|
— |
|
|
|
— |
|
Common
stock; $0.0001 par value; 100,000 shares authorized at June
30, 2017 and December 31, 2016; 30,853 and 26,477 shares
issued and outstanding at June 30, 2017 and December 31, 2016,
respectively |
|
|
3 |
|
|
|
3 |
|
Additional paid-in capital |
|
|
363,393 |
|
|
|
276,354 |
|
Accumulated other comprehensive loss |
|
|
(646 |
) |
|
|
(33 |
) |
Accumulated deficit |
|
|
(151,053 |
) |
|
|
(114,587 |
) |
Total
stockholders’ equity |
|
|
211,697 |
|
|
|
161,737 |
|
Total
liabilities and stockholders’ equity |
|
$ |
224,376 |
|
|
$ |
172,732 |
|
|
|
|
|
|
|
|
|
|
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, |
|
|
|
2017 |
|
|
2016 |
|
|
2017 |
|
|
2016 |
|
Revenues |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
License
revenue |
|
$ |
6,555 |
|
|
$ |
2,245 |
|
|
$ |
7,010 |
|
|
$ |
2,573 |
|
Reagent
sales |
|
|
— |
|
|
|
107 |
|
|
|
— |
|
|
|
166 |
|
Grant
revenue |
|
|
7 |
|
|
|
23 |
|
|
|
7 |
|
|
|
29 |
|
Total
revenues |
|
|
6,562 |
|
|
|
2,375 |
|
|
|
7,017 |
|
|
|
2,768 |
|
Expenses |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Costs of
revenues |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Licensing
costs |
|
|
1,311 |
|
|
|
449 |
|
|
|
1,402 |
|
|
|
515 |
|
Costs of
reagent sales |
|
|
6 |
|
|
|
49 |
|
|
|
6 |
|
|
|
79 |
|
Research
and development |
|
|
13,917 |
|
|
|
10,680 |
|
|
|
30,536 |
|
|
|
16,863 |
|
General
and administrative |
|
|
6,355 |
|
|
|
6,169 |
|
|
|
12,977 |
|
|
|
11,648 |
|
Other
operating expenses (income) |
|
|
29 |
|
|
|
(20 |
) |
|
|
74 |
|
|
|
(134 |
) |
Total
operating expenses |
|
|
21,618 |
|
|
|
17,327 |
|
|
|
44,995 |
|
|
|
28,971 |
|
Loss from
operations |
|
|
(15,056 |
) |
|
|
(14,952 |
) |
|
|
(37,978 |
) |
|
|
(26,203 |
) |
Other
Income |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Investment income |
|
|
583 |
|
|
|
515 |
|
|
|
1,512 |
|
|
|
998 |
|
Total
other income |
|
|
583 |
|
|
|
515 |
|
|
|
1,512 |
|
|
|
998 |
|
Net
loss |
|
$ |
(14,473 |
) |
|
$ |
(14,437 |
) |
|
$ |
(36,466 |
) |
|
$ |
(25,205 |
) |
Other
Comprehensive Income (Loss) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gain (loss) on available-for-sale securities, net
of reclassifications of $480 for the six months ended June 30,
2017 |
|
|
(74 |
) |
|
|
246 |
|
|
|
(613 |
) |
|
|
1,240 |
|
Total
other comprehensive income (loss) |
|
|
(74 |
) |
|
|
246 |
|
|
|
(613 |
) |
|
|
1,240 |
|
Comprehensive loss |
|
$ |
(14,547 |
) |
|
$ |
(14,191 |
) |
|
$ |
(37,079 |
) |
|
$ |
(23,965 |
) |
Basic and diluted net
loss per common share |
|
$ |
(0.47 |
) |
|
$ |
(0.55 |
) |
|
$ |
(1.27 |
) |
|
$ |
(0.96 |
) |
Weighted-average basic
and diluted common shares |
|
|
30,662 |
|
|
|
26,362 |
|
|
|
28,678 |
|
|
|
26,344 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
CONTACT:
Investors
Heather Savelle, 646-395-3734
heather@argotpartners.com
Media
Adam Pawluk, 202-591-4063
apawluk@jpa.com
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