ROCKVILLE, Md., May 5, 2021 /PRNewswire/ --
- Enrollment ongoing in ATMOSPHERE™, the first of two planned
pivotal trials for the subretinal delivery of RGX-314 for the
treatment of wet AMD
- Continued progress and expansion of Phase II
AAVIATE® trial of RGX-314 utilizing in-office
suprachoroidal delivery for the treatment of wet AMD
- Began dosing in Phase I/II trial of RGX-121, a one-time gene
therapy for MPS II, in pediatric patients over 5 years old
- Completed dosing in Cohort 1 of ongoing trial of RGX-111, a
one-time gene therapy for MPS I
- IND filing expected in mid-2021 for RGX-202, a novel,
advanced microdystrophin gene therapy for treatment of Duchenne
Muscular Dystrophy
- $657 million in cash, cash
equivalents and marketable securities as of March 31, 2021
- Conference call Wednesday, May
5th at 4:30 p.m.
ET
REGENXBIO Inc. (Nasdaq: RGNX) today announced financial results
for the first quarter ended March 31,
2021, and recent operational highlights.
"In the first quarter of 2021, we were able to build upon the
important clinical advancements we made in 2020, including
continued enrollment of patients in the Company's first pivotal
trial for the treatment of wet AMD," said Kenneth T. Mills, President and Chief Executive
Officer of REGENXBIO. "Additionally, we are pleased to announce the
advancement of our Phase II AAVIATE® trial for the
suprachoroidal delivery of RGX-314 for wet AMD where we have
completed enrollment in the second cohort of patients and expanded
the trial to evaluate suprachoroidal delivery of RGX-314 in
patients who are positive for neutralizing antibodies. This cohort
of patients may provide us additional information about the effects
of RGX-314 in neutralizing antibody positive patients, potentially
broadening the patient population that could be treated with this
in-office delivery approach."
Mr. Mills added: "Beyond RGX-314, we have continued to advance
our MPS II clinical program, enrolling additional patients in our
ongoing Phase I/II trial of RGX-121 in younger patients, as well as
dosing the first patient in our Phase I/II trial of RGX-121 in
older patients. The ongoing Phase I/II trial of RGX-111 in patients
with MPS I also continues to advance, having completed dosing in
Cohort 1. We are also on track to file an IND for RGX-202 in
mid-2021. We look forward to providing additional updates from our
programs in 2021."
Recent Operational Highlights
Gene Therapy Using NAV® Vectors for
AAV-Mediated Antibody Delivery
- Pivotal Program for RGX-314 for the Treatment of Wet
Age-related Macular Degeneration (wet AMD)
-
- Enrollment is ongoing in ATMOSPHERE™, the first of two planned
pivotal trials to evaluate the efficacy and safety of RGX-314 in
patients with wet AMD using the subretinal delivery
approach.
-
- The randomized, well-controlled trial will enroll approximately
300 patients across two RGX-314 dose arms versus ranibizumab. The
primary endpoint of the trial is non-inferiority to ranibizumab
based on change from baseline in Best Corrected Visual Acuity
(BCVA) at one year.
- The second pivotal trial is expected to be similar in design to
ATMOSPHERE and REGENXBIO plans to initiate the trial in
the second half of 2021.
- The manufacturing bridging study is now active to align with
plans to incorporate REGENXBIO's scalable suspension cell culture
manufacturing process to support potential future commercialization
of RGX-314.
-
- The open-label study will enroll approximately 60 patients to
evaluate two manufacturing process formulations of RGX-314. The
primary endpoint of the trial is RGX-314 protein concentration in
the aqueous humor at six months.
- Suprachoroidal Delivery of RGX-314 for the Treatment of Wet
AMD
-
- REGENXBIO plans to report interim data from Cohort 1 of
AAVIATE, a Phase II trial of RGX-314 for the treatment of wet AMD,
in the third quarter of 2021.
- REGENXBIO has completed dosing of patients in Cohort 2 of
AAVIATE and expects to report interim data from Cohort 2 in the
second half of 2021.
- In addition, REGENXBIO has expanded AAVIATE, and began dosing
in a third cohort of patients.
-
- Cohort 3 will evaluate the efficacy, safety and tolerability of
RGX-314 in up to 20 patients who are neutralizing antibody (NAb)
positive.
- The same dose evaluated in Cohort 2, 5.0x1011
genomic copies per eye (GC/eye) of RGX-314, will be delivered to
patients in Cohort 3 via a single injection. As with Cohorts 1 and
2, patients in Cohort 3 will not receive prophylactic immune
suppressive corticosteroid therapy before or after administration
of RGX-314.
- Suprachoroidal Delivery of RGX-314 for the Treatment of
Diabetic Retinopathy (DR)
-
- REGENXBIO continues to enroll patients in Cohort 1 in
ALTITUDE™, a Phase II trial for the treatment of DR, and expects to
report initial data in 2021.
- Research Program for the Treatment of Hereditary Angioedema
(HAE)
-
- REGENXBIO expects to provide a program update in 2021.
- Research Program for the Treatment of Neurodegenerative
Diseases
-
- REGENXBIO continues to collaborate with Neurimmune
AG on research programs targeting both alpha synuclein and tau
and expects to provide a program update in 2021.
Gene Therapy Using NAV Vectors for Rare Genetic
Diseases
- RGX-202 for the Treatment of Duchenne Muscular Dystrophy
(DMD)
-
- REGENXBIO expects to submit an Investigational New Drug
application (IND) to the FDA for RGX-202 for the treatment of DMD
in mid-2021.
- RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS
II)
-
- In April 2021, REGENXBIO
announced that the first patient had been dosed in Cohort 3 of the
ongoing Phase I/II trial of RGX-121 for the treatment of MPS II in
patients up to 5 years old. Patients in the third cohort will
receive a dose of 2.0x1011 GC/g of brain mass of
RGX-121, delivered directly to the cerebrospinal fluid (CSF).
- In addition, the first patient has been dosed in a second Phase
I/II trial of RGX-121 for the treatment of pediatric patients with
MPS II over the age of 5 years old.
-
- Up to six patients are expected to be enrolled in the
multicenter, open-label trial, and RGX-121 will be administered at
a dose level of 6.5x1010 GC/g of brain mass, delivered
directly to the CSF.
- RGX-111 for the Treatment of Mucopolysaccharidosis Type I (MPS
I)
-
- REGENXBIO has completed dosing of patients in Cohort 1 of the
Phase I/II trial of RGX-111 for the treatment of MPS I.
- RGX-181 for the Treatment of Late-infantile Neuronal Ceroid
Lipofuscinosis Type 2 (CLN2) Disease
-
- An IND was submitted to the U.S. Food and Drug Administration
(FDA), after which the FDA notified REGENXBIO in a letter that its
proposed trial had been placed on clinical hold and the agency
requested more information to support the initial dose selection
and certain study drug administration procedures. REGENXBIO is
evaluating the FDA's requests and plans to provide an update on the
program in the second half of 2021.
- RGX-381 for the Treatment of Ocular Manifestations of CLN2
Disease
-
- Based on communication with the FDA and the update from the
RGX-181 program, REGENXBIO now expects to provide a program
update for RGX-381 in the second half of 2021.
Operational Updates
- Current Good Manufacturing Practice (cGMP) Manufacturing
Facility
-
- REGENXBIO expects to begin utilizing its new headquarters in
Rockville, Maryland in the first
half of 2021, and the cGMP facility which is expected to allow for
production of NAV vectors at scales up to 2,000 liters using
REGENXBIO's platform suspension cell culture process is on track to
be operational starting in the first half of 2022.
NAV Technology Licensee Program Highlights
As of March 31, 2021, REGENXBIO's
NAV Technology Platform was being applied in one marketed product
and multiple clinical stage programs, 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:
- In January 2021, Ultragenyx
Pharmaceutical Inc. announced FDA Clearance of an IND for UX701, a
gene therapy for the treatment of Wilson Disease. Ultragenyx is
expected to begin enrolling patients in a single-protocol Phase
1/2/3 study in the second half of 2021. UX701 uses the NAV AAV9
vector.
- In April 2021, Astellas Gene
Therapies, formerly Audentes Therapeutics, Inc., announced that the
first patient has been dosed in its Phase I/II FORTIS trial
evaluating AT845 in patients with late-onset Pompe disease. AT845
uses the NAV AAV8 vector.
Marketed NAV Technology Products
REGENXBIO's NAV Technology Platform is being applied in one
marketed product, Zolgensma®. On April 27, 2021, Novartis AG reported first
quarter 2021 global Zolgensma sales revenue of $319 million.
Financial Results
Cash Position: Cash, cash equivalents and marketable
securities were $656.5 million as of
March 31, 2021, compared to
$522.5 million as of December 31, 2020. The increase was primarily
attributable to $216.1 million of
aggregate net proceeds received from the Company's follow-on public
offering of common stock completed in January 2021, including the full exercise of the
underwriters' option to purchase additional shares in connection
with the offering. The increase was partially offset by net cash
used in operating activities of $41.1
million, cash used to purchase property and equipment of
$31.0 million, and Zolgensma
royalties paid to Healthcare Royalty Management, LLC of
$9.5 million during the three months
ended March 31, 2021.
Revenues: Revenues were $18.9
million for the three months ended March 31, 2021, compared to $17.6 million for the three months ended
March 31, 2020. The increase was
primarily attributable to Zolgensma royalty revenues, which
increased by $8.3 million, from
$10.0 million for the first quarter
of 2020 to $18.3 million for the
first quarter of 2021. As reported by Novartis, sales of Zolgensma
for the first quarter of 2021 increased by 88% as compared to the
first quarter of 2020. The increase in revenues was partially
offset by non-recurring revenue recognized during the three months
ended March 31, 2020 related to new
licenses to the NAV Technology Platform granted by REGENXBIO during
the period.
Research and Development Expenses: Research and
development expenses were $39.7
million for the three months ended March 31, 2021, compared to $37.0 million for the three months ended
March 31, 2020. The increase was
primarily attributable to personnel costs as a result of increased
headcount, laboratory and facilities costs, and clinical trial
expenses for our lead product candidates.
General and Administrative Expenses: General and
administrative expenses were $17.8
million for the three months ended March 31, 2021, compared to $14.8 million for the three months ended
March 31, 2020. The increase was
primarily attributable to personnel costs as a result of increased
headcount and professional fees for advisory and other
services.
Net Loss: Net loss was $50.1
million, or $1.20 basic and
diluted net loss per share, for the three months March 31, 2021, compared to net loss of
$40.0 million, or $1.08 basic and diluted net loss per share, for
the three months ended March 31,
2020.
Financial Guidance
Based on its current operating plan, REGENXBIO expects its
balance in cash, cash equivalents and marketable securities of
$656.5 million as of March 31, 2021, to fund its operations, including
the completion of its internal manufacturing capabilities and
clinical advancement of its product candidates, into the second
half of 2023.
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 7044809. 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," "assume," "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, 2020, 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. Except as required by law,
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.
Zolgensma® is a registered trademark of Novartis Gene
Therapies. All other trademarks referenced herein are registered
trademarks of REGENXBIO.
REGENXBIO
INC.
CONSOLIDATED
BALANCE SHEETS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
|
March 31,
2021
|
|
|
December 31,
2020
|
|
Assets
|
|
|
|
|
|
|
|
|
Current
assets
|
|
|
|
|
|
|
|
|
Cash and cash
equivalents
|
|
$
|
291,482
|
|
|
$
|
338,426
|
|
Marketable
securities
|
|
|
149,398
|
|
|
|
137,314
|
|
Accounts receivable,
net
|
|
|
41,039
|
|
|
|
42,999
|
|
Prepaid
expenses
|
|
|
13,839
|
|
|
|
10,505
|
|
Other current
assets
|
|
|
2,880
|
|
|
|
1,953
|
|
Total current
assets
|
|
|
498,638
|
|
|
|
531,197
|
|
Marketable
securities
|
|
|
215,598
|
|
|
|
46,809
|
|
Accounts receivable,
net
|
|
|
2,859
|
|
|
|
3,267
|
|
Property and
equipment, net
|
|
|
89,342
|
|
|
|
56,467
|
|
Operating lease
right-of-use assets
|
|
|
62,607
|
|
|
|
63,815
|
|
Restricted
cash
|
|
|
1,330
|
|
|
|
1,330
|
|
Other
assets
|
|
|
9,068
|
|
|
|
5,279
|
|
Total
assets
|
|
$
|
879,442
|
|
|
$
|
708,164
|
|
Liabilities and
Stockholders' Equity
|
|
|
|
|
|
|
|
|
Current
liabilities
|
|
|
|
|
|
|
|
|
Accounts
payable
|
|
$
|
11,311
|
|
|
$
|
10,622
|
|
Accrued expenses and
other current liabilities
|
|
|
41,605
|
|
|
|
49,082
|
|
Deferred
revenue
|
|
|
395
|
|
|
|
449
|
|
Operating lease
liabilities
|
|
|
1,843
|
|
|
|
2,500
|
|
Liability related to
sale of future royalties
|
|
|
28,807
|
|
|
|
18,794
|
|
Total current
liabilities
|
|
|
83,961
|
|
|
|
81,447
|
|
Deferred
revenue
|
|
|
3,729
|
|
|
|
3,783
|
|
Operating lease
liabilities
|
|
|
75,078
|
|
|
|
70,153
|
|
Liability related to
sale of future royalties
|
|
|
161,722
|
|
|
|
174,504
|
|
Other
liabilities
|
|
|
448
|
|
|
|
524
|
|
Total
liabilities
|
|
|
324,938
|
|
|
|
330,411
|
|
Stockholders' equity
|
|
|
|
|
|
|
|
|
Preferred stock; no
shares issued and outstanding
at
March 31, 2021 and December 31, 2020
|
|
|
—
|
|
|
|
—
|
|
Common stock; 42,505
and 37,476 shares issued
and
outstanding at March 31, 2021 and
December 31, 2020, respectively
|
|
|
4
|
|
|
|
4
|
|
Additional paid-in
capital
|
|
|
895,079
|
|
|
|
667,181
|
|
Accumulated other
comprehensive loss
|
|
|
(1,368)
|
|
|
|
(360)
|
|
Accumulated
deficit
|
|
|
(339,211)
|
|
|
|
(289,072)
|
|
Total
stockholders' equity
|
|
|
554,504
|
|
|
|
377,753
|
|
Total liabilities and
stockholders' equity
|
|
$
|
879,442
|
|
|
$
|
708,164
|
|
REGENXBIO
INC.
CONSOLIDATED
STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(unaudited)
(in thousands,
except per share data)
|
|
|
|
|
|
Three Months Ended
March 31,
|
|
|
|
2021
|
|
|
2020
|
|
Revenues
|
|
|
|
|
|
|
|
|
License and royalty
revenue
|
|
$
|
18,884
|
|
|
$
|
17,644
|
|
Total
revenues
|
|
|
18,884
|
|
|
|
17,644
|
|
Operating
Expenses
|
|
|
|
|
|
|
|
|
Cost of
revenues
|
|
|
4,851
|
|
|
|
3,409
|
|
Research and
development
|
|
|
39,722
|
|
|
|
37,035
|
|
General and
administrative
|
|
|
17,838
|
|
|
|
14,833
|
|
Provision for credit
losses and other
|
|
|
515
|
|
|
|
67
|
|
Total operating
expenses
|
|
|
62,926
|
|
|
|
55,344
|
|
Loss from
operations
|
|
|
(44,042)
|
|
|
|
(37,700)
|
|
Other Income
(Expense)
|
|
|
|
|
|
|
|
|
Interest income from
licensing
|
|
|
29
|
|
|
|
848
|
|
Investment income
(loss)
|
|
|
580
|
|
|
|
(3,186)
|
|
Interest
expense
|
|
|
(6,702)
|
|
|
|
—
|
|
Total other income
(expense)
|
|
|
(6,093)
|
|
|
|
(2,338)
|
|
Loss before income
taxes
|
|
|
(50,135)
|
|
|
|
(40,038)
|
|
Income Tax
Expense
|
|
|
(4)
|
|
|
|
—
|
|
Net loss
|
|
$
|
(50,139)
|
|
|
$
|
(40,038)
|
|
Other
Comprehensive Loss
|
|
|
|
|
|
|
|
|
Unrealized loss on
available-for-sale securities, net
|
|
|
(1,008)
|
|
|
|
(785)
|
|
Total other
comprehensive loss
|
|
|
(1,008)
|
|
|
|
(785)
|
|
Comprehensive
loss
|
|
$
|
(51,147)
|
|
|
$
|
(40,823)
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(1.20)
|
|
|
$
|
(1.08)
|
|
Weighted-average
common shares outstanding, basic and diluted
|
|
|
41,819
|
|
|
|
37,104
|
|
Contacts:
Tricia
Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Brendan Burns, 212-600-1902
brendan@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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SOURCE REGENXBIO Inc.