ROCKVILLE, Md., May 7, 2019 /PRNewswire/ --
- Reports positive interim update from RGX-314 Phase I/IIa
trial for wet AMD in Cohort 3 over one year
- Announces new IND submission for RGX-314 for a Phase II
trial in diabetic retinopathy; filing planned for second half
2019
- Continuing subject recruitment for RGX-121 Phase I/II trial
for MPS II, RGX-111 Phase I trial for MPS I and RGX-501 Phase I/II
trial for HoFH; interim updates for all trials expected in second
half 2019
- On-track to submit IND for first-in-human trial of RGX-181
for CLN2 form of Batten disease in second half 2019
- Anticipates FDA approval of NAV Technology licensee
Novartis' Zolgensma for SMA Type I
- $444 million in cash, cash
equivalents and marketable securities as of March 31, 2019
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,
2019, and recent operational highlights.
"We continue to be encouraged by the data from Cohort 3 of the
RGX-314 Phase I/IIa trial for wet AMD, which demonstrated sustained
protein expression and clinical effect on best corrected visual
acuity and central retinal thickness measures at one year," said
Kenneth T. Mills, President and
Chief Executive Officer of REGENXBIO. "These results further
support the potential of our NAV Technology gene therapy, and we
are excited to announce that we will expand clinical development of
RGX-314 later this year into diabetic retinopathy, the most common
cause of vision loss in people with diabetes, affecting
approximately 8 million people in the
United States alone."
Mr. Mills added: "This past quarter, we worked to drive
significant clinical and regulatory progress across our five
internal candidate programs for the treatment of retinal,
neurodegenerative and metabolic diseases. We continue to advance
our capabilities as a leader in the development and manufacture of
NAV product candidates and to enable our NAV Technology licensee
network to develop potentially life-changing treatments. As we
celebrate the 10th anniversary of REGENXBIO's founding, we remain
highly motivated and deeply committed to our mission of improving
the lives of patients through the curative potential of gene
therapy."
Interim Phase I/IIa Trial Update for RGX-314 for the
Treatment of Wet Age-Related Macular Degeneration
REGENXBIO announced today an update to its Phase I/IIa
trial of RGX-314 for the treatment of wet age-related macular
degeneration (wet AMD), since its previously reported interim
results. The interim data update announced today includes safety
assessments for all subjects enrolled as of April 18, 2019,
and new assessments of efficacy at one year after a single
administration of RGX-314 for Cohort 3 (6 x 10^10
GC/eye).
- As of April 18, 2019, 33 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
cohorts, with no drug related serious adverse events (SAEs)
reported.1
- In addition, the interim update announced today includes new
assessments of protein expression levels, reduction in
anti-vascular endothelial growth factor (VEGF) intravitreal
injections, change in central retinal thickness (CRT) by spectral
domain optical coherence tomography (SD-OCT), and Best Corrected
Visual Acuity (BCVA) at one year after a single administration of
RGX-314 for Cohort 3 (n=6). These new data are summarized
below.
-
- At one year after administration of RGX-314, Cohort 3 subjects
continued to demonstrate evidence of sustained RGX-314 intraocular
protein expression levels. Mean protein levels were 180.8 ng/ml one
year post-treatment.
- Mean BCVA improved by +5 letters and mean CRT decreased by 39
μm from baseline in Cohort 3 subjects at one year.
- Subjects upon enrollment in the study had received, on
average, more than 35 anti-VEGF injections since diagnosis,
prior to RGX-314 treatment. Cohort 3 subjects received a low number
of anti-VEGF injections following the administration of RGX-314,
with a mean of 2.3 injections over one year.
- 50% of subjects (3/6) in Cohort 3 continue to remain
injection-free at one year with persistent clinical durability of
effect observed on BCVA and CRT. Mean BCVA improved by +10 letters
and mean CRT decreased by 59 μm from baseline in these subjects at
one year. Evidence of durable protein expression was also observed
(see Table 1).
Table 1: RGX-314 Protein Expression Levels (ng/ml) of Cohort
3 Subjects with No Additional Anti-VEGF Injections through One Year
(N=3)
Visit
|
1 month
|
6 months
|
1 year
|
Mean Protein Level
(ng/ml)
|
236.2
|
274.9
|
260.5
|
"The sustained protein expression and durable clinical response
observed in Cohort 3 at one year after one-time administration of
RGX-314 in these previously treated subjects is promising," said
Dr. Jeffrey Heier, Co-President and
Director of Retina Research at Ophthalmic Consultants of
Boston and primary investigator
for the trial. "Most subjects in this study had received
frequent anti-VEGF eye injections over five years at entry into the
trial. The notable reduction in the number of anti-VEGF injections
combined with the sustained clinical effect at one year show the
potential of one-time gene therapy with RGX-314 to achieve
long-lasting clinical outcomes while alleviating the burden of
current treatment in wet AMD patients."
REGENXBIO has completed dosing subjects in Cohort 4 (1.6 x 10^11
GC/eye), is nearing completion of Cohort 5 (2.5 x 10^11 GC/eye)
enrollment and expects to present top-line data from
the Phase I/IIa clinical trial by the end of 2019. The company is
on track to initiate a Phase IIb trial for wet AMD in late
2019.
Expansion of RGX-314 Program for the Treatment of Diabetic
Retinopathy
Today, REGENXBIO announced it expects to file a new IND for a
Phase II trial evaluating RGX-314 in subjects with diabetic
retinopathy (DR) in the second half of 2019.
DR is the leading cause of vision loss in the working-age
population and affects approximately 8 million people in
the United States. DR is a
complication of diabetes and is a progressive retinopathy, the
severity of which ranges from mild non-proliferative diabetic
retinopathy to a more advanced proliferative diabetic retinopathy
(PDR). The main causes of vision loss secondary to DR are the
vision-threatening complications of PDR, marked by the growth of
new abnormal blood vessels onto the surface of the retina and
vitreous cavity causing severe vision loss and diabetic macular
edema (DME) leading to visual impairment. DME can occur at any
stage of DR as the blood vessels in the retina become increasingly
fragile and leak fluid.
"Diabetic retinopathy progression and its complications leading
to vision impairment are largely driven by excess intraocular
VEGF," said Dr. Stephen Pakola,
Senior Vice President and Chief Medical Officer of REGENXBIO. "The
standard of care for vision-threatening complications of diabetic
eye disease is frequent intraocular injections of drugs that target
VEGF on a long-term basis or panretinal laser treatment depending
on the stage of the disease. We are excited at the potential
applications of RGX-314 as a one-time therapy to address
significant unmet needs in diabetic eye disease."
"Diabetic retinopathy is a serious public health concern
affecting our growing diabetic patient populations, who often
present with vision loss at a younger age than patients with
macular degeneration," said Dr. Allen
Ho, Wills Eye Hospital Attending Surgeon and Director of
Retina Research and study investigator. "The opportunity to
administer a one-time therapy to potentially halt disease
progression and/or treat vision-threatening complications may offer
important and efficient treatment options for patients who are
otherwise burdened by numerous specialist visits and interventions
for their diabetes."
For the Phase II trial evaluating RGX-314 in subjects with DR,
REGENXBIO expects to administer the treatment using the same
subretinal approach as in the current trial of RGX-314 for
treatment of wet AMD. Safety and other data from the ongoing Phase
I/IIa trial of RGX-314 in wet AMD will also be used to support dose
selection. REGENXBIO anticipates many of the existing clinical
sites from the Phase I/IIa trial will participate in the DR study,
plus additional sites to be selected.
Manufacturing of Phase II product continues to be on track to
support enrollment in all trials of RGX-314 for wet AMD and DR.
REGENXBIO expects that its current manufacturing process is
capable of meeting future clinical program demand and anticipated
potential commercial demand.
Other Recent Operational Highlights
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS
II)
-
- At a recent 28-week safety assessment, RGX-121 continues to be
well-tolerated with no serious adverse events (SAEs) reported.
Additional recruitment and site activations are ongoing 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-111 for the Treatment of Mucopolysaccharidosis Type I (MPS
I)
-
- Recruitment and additional site activations are ongoing in the
Phase I clinical trial evaluating RGX-111 for the treatment of MPS
I.
- REGENXBIO expects to present an interim data update from the
Phase I clinical trial in the second half of 2019.
- RGX-501 for the Treatment of Homozygous Familial
Hypercholesterolemia (HoFH)
-
- The protocol has been amended and screening has re-initiated 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-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.
NAV Technology Licensee Program Highlights
As of March 31, 2019, REGENXBIO's
NAV Technology Platform was being applied in more than 20 partnered
product candidates in development by NAV Technology Licensees.
Fourteen of these partnered product candidates are in active
clinical development and one partnered product candidate has been
submitted for Biologics License Application (BLA) approval with
the FDA. 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:
- In April 2019, Novartis announced
that interim data from its Phase III STR1VE trial of Zolgensma in
SMA type I showed prolonged event-free survival, an early and rapid
increase in CHOP-INTEND scores and significant milestone
achievement compared to untreated natural history, consistent with
data from the pivotal Phase I START trial. Novartis has
reiterated that they remain on track to launch
Zolgensma in the United
States and Japan in first half of 2019
and Europe in second half of 2019 for the treatment of
spinal muscular atrophy (SMA) Type I, pending approval by the FDA.
REGENXBIO is eligible to receive $80 million in
potential future commercial milestone payments, in addition to
regulatory milestones and royalties on net sales of
Zolgensma. Zolgensma uses the NAV AAV9 vector.
- Earlier this week, Novartis presented interim data from their
STRONG Phase I study of Zolgensma in Type 2 SMA and SPR1NT Phase
III study, designed to evaluate Zolgensma in pre-symptomatic SMA
newborns. Intrathecal administration of Zolgensma in SMA Type
2 subjects led to improvement of motor function, as assessed by
HFMSE, and achievement of motor milestones following
treatment. Initial results of the SPR1NT study in
pre-symptomatic SMA newborns are encouraging, with subjects
demonstrating improvement in CHOP-INTEND scores.
- In May 2019, Audentes
Therapeutics, Inc. announced new positive data from ASPIRO, the
Phase I/II Clinical Trial of AT132 for X-linked Myotubular
Myopathy. The new data included up to 48 weeks of follow-up for
nine treated subjects in two dose cohorts and reported significant
and sustained improvements in neuromuscular and respiratory
function in both dose cohorts, with corresponding achievement of
clinically meaningful milestones. Audentes continues to plan for
interactions in the second half of 2019 to gain further alignment
on the license application submission pathways for AT132 in
the United States and
Europe. AT132 uses the NAV AAV8 vector.
Financial Results
Cash, cash equivalents and marketable securities were
$444.3 million as of March 31, 2019, compared to $470.6 million as of December 31, 2018. The decrease was primarily
attributable to net cash flows used in operating activities of
$29.3 million.
Revenues were $0.9 million for the
three months ended March 31, 2019,
compared to $132.4 million for the
three months ended March 31, 2018.
The decrease was primarily attributable to $132.1 million of non-recurring revenue
recognized during the three months ended March 31, 2018 under our amended license
agreement with AveXis for the development and commercialization of
treatments for SMA.
Research and development expenses were $25.2 million for the three months ended
March 31, 2019, compared to
$19.6 million for the three months
ended March 31, 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 $11.6 million for the three months ended
March 31, 2019, compared to
$8.4 million for the three months
ended March 31, 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 $32.2 million, or
$0.89 basic and diluted net loss per
share, for the three months ended March 31,
2019, compared to net income of $104.2 million, or $3.30 basic and $3.04 diluted net income per share, for the three
months ended March 31, 2018.
Financial Guidance
Upon regulatory approval and product launch, Zolgensma is
expected to provide REGENXBIO with its first revenue stream from
royalties on commercial product sales, adding commercial revenue to
its existing base of licensee revenue this year. Based on
REGENXBIO's current operating plan, and excluding any royalties
from Zolgensma, REGENXBIO reiterates that it expects its balance in
cash, cash equivalents and marketable securities to be between
$330 million and $350 million as of December 31, 2019, which will be used to support
the continued development of its lead product candidate
programs.
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.
About Diabetic Retinopathy
Diabetic retinopathy (DR) is the leading cause of vision loss in
adults between 25 and 74 years of age worldwide. DR affects
approximately 8 million people in the
United States alone. The spectrum of DR encompasses
nonproliferative diabetic retinopathy and proliferative diabetic
retinopathy, marked by the absence or presence of retinal
neovascularization, respectively. Macular edema, defined as
retinal thickening and edema involving the macula, can occur at any
stage of DR and is a significant complication.
Current treatment options include anti-VEGF injections and/or
panretinal laser treatment, depending on the stage of DR with and
without macular edema. Anti-VEGF therapy, however, requires
repetitive and inconvenient intraocular injections, typically
ranging from every four to eight weeks in frequency, to maintain
efficacy. Panretinal laser treatment cauterizes and destroys
diseased retina to preserve healthy retina. Side-effects of
panretinal laser include permanent decrease in peripheral and color
vision. One-time administration of RGX-314 can potentially provide
lasting treatment effect and halt progression of disease in DR by
preventing and treating vision threatening complications of the
disease.
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, 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.
1.
|
As previously
reported, one SAE was a procedure-related peripheral retinal
detachment that occurred, was repaired with a scleral buckle and
resolved without significant sequelae.
|
REGENXBIO
INC.
CONSOLIDATED
BALANCE SHEETS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
|
|
|
March 31,
2019
|
|
|
December 31,
2018
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
55,852
|
|
|
$
|
75,561
|
|
Marketable
securities
|
|
|
229,373
|
|
|
|
244,200
|
|
Accounts
receivable
|
|
|
8,372
|
|
|
|
8,587
|
|
Prepaid
expenses
|
|
|
6,292
|
|
|
|
5,734
|
|
Other current
assets
|
|
|
3,995
|
|
|
|
3,831
|
|
Total current
assets
|
|
|
303,884
|
|
|
|
337,913
|
|
Marketable
securities
|
|
|
159,083
|
|
|
|
150,819
|
|
Accounts
receivable
|
|
|
22,758
|
|
|
|
23,012
|
|
Property and
equipment, net
|
|
|
23,140
|
|
|
|
28,702
|
|
Operating lease
right-of-use assets
|
|
|
6,858
|
|
|
|
—
|
|
Restricted
cash
|
|
|
1,053
|
|
|
|
1,053
|
|
Other
assets
|
|
|
2,255
|
|
|
|
2,315
|
|
Total
assets
|
|
$
|
519,031
|
|
|
$
|
543,814
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
4,204
|
|
|
$
|
4,412
|
|
Accrued expenses and
other current liabilities
|
|
|
14,189
|
|
|
|
17,164
|
|
Deferred
revenue
|
|
|
600
|
|
|
|
600
|
|
Operating lease
liabilities
|
|
|
2,397
|
|
|
|
—
|
|
Total current
liabilities
|
|
|
21,390
|
|
|
|
22,176
|
|
Deferred
revenue
|
|
|
3,333
|
|
|
|
3,333
|
|
Operating lease
liabilities
|
|
|
5,483
|
|
|
|
—
|
|
Deferred
rent
|
|
|
—
|
|
|
|
1,098
|
|
Financing lease
obligations
|
|
|
—
|
|
|
|
5,854
|
|
Other
liabilities
|
|
|
1,772
|
|
|
|
2,505
|
|
Total
liabilities
|
|
|
31,978
|
|
|
|
34,966
|
|
Stockholders' equity
|
|
|
|
|
|
|
|
|
Preferred stock;
$0.0001 par value; 10,000 shares authorized,
and
no shares issued and outstanding at March 31, 2019
and
December 31, 2018
|
|
|
—
|
|
|
|
—
|
|
Common stock; $0.0001
par value; 100,000 shares authorized
at
March 31, 2019 and December 31, 2018; 36,611 and
36,120 shares issued and outstanding at March 31,
2019
and
December 31, 2018, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
602,425
|
|
|
|
592,580
|
|
Accumulated other
comprehensive loss
|
|
|
(59)
|
|
|
|
(720)
|
|
Accumulated
deficit
|
|
|
(115,317)
|
|
|
|
(83,016)
|
|
Total
stockholders' equity
|
|
|
487,053
|
|
|
|
508,848
|
|
Total liabilities and
stockholders' equity
|
|
$
|
519,031
|
|
|
$
|
543,814
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE INCOME
(LOSS)
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
Three Months Ended
March 31,
|
|
|
|
2019
|
|
|
2018
|
|
Revenues
|
|
|
|
|
|
|
|
|
License
revenue
|
|
$
|
884
|
|
|
$
|
132,391
|
|
Total
revenues
|
|
|
884
|
|
|
|
132,391
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
Costs of
revenues
|
|
|
|
|
|
|
|
|
Licensing
costs
|
|
|
29
|
|
|
|
2,408
|
|
Research and
development
|
|
|
25,203
|
|
|
|
19,550
|
|
General and
administrative
|
|
|
11,558
|
|
|
|
8,380
|
|
Other operating
expenses
|
|
|
—
|
|
|
|
28
|
|
Total operating
expenses
|
|
|
36,790
|
|
|
|
30,366
|
|
Income (loss) from
operations
|
|
|
(35,906)
|
|
|
|
102,025
|
|
Other
Income
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
|
613
|
|
|
|
1,355
|
|
Investment
income
|
|
|
2,995
|
|
|
|
859
|
|
Total other
income
|
|
|
3,608
|
|
|
|
2,214
|
|
Income (loss) before
income taxes
|
|
|
(32,298)
|
|
|
|
104,239
|
|
Income Tax
Benefit
|
|
|
70
|
|
|
|
—
|
|
Net income
(loss)
|
|
$
|
(32,228)
|
|
|
$
|
104,239
|
|
Other
Comprehensive Income (Loss)
|
|
|
|
|
|
|
|
|
Unrealized gain (loss)
on available-for-sale securities,
net of reclassifications and income
tax expense
|
|
|
621
|
|
|
|
(188)
|
|
Total other
comprehensive income (loss)
|
|
|
621
|
|
|
|
(188)
|
|
Comprehensive income
(loss)
|
|
$
|
(31,607)
|
|
|
$
|
104,051
|
|
Net income (loss) per
share:
|
|
|
|
|
|
|
|
|
Basic
|
|
$
|
(0.89)
|
|
|
$
|
3.30
|
|
Diluted
|
|
$
|
(0.89)
|
|
|
$
|
3.04
|
|
Weighted-average
common shares outstanding:
|
|
|
|
|
|
|
|
|
Basic
|
|
|
36,366
|
|
|
|
31,632
|
|
Diluted
|
|
|
36,366
|
|
|
|
34,275
|
|
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-first-quarter-2019-financial-and-operating-results-and-additional-positive-interim-phase-iiia-trial-update-for-rgx-314-for-the-treatment-of-wet-amd-300845423.html
SOURCE REGENXBIO Inc.