- REGENXBIO grants Rocket exclusive, worldwide rights to NAV AAV9 for
the development and commercialization of treatments for Danon Disease -
- Investigational New Drug Application Expected to be Filed in 2019;
Clinical Trial to Follow -
NEW YORK & ROCKVILLE, Md.--(BUSINESS WIRE)--Nov. 26, 2018--
Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT), a leading U.S.-based
multi-platform gene therapy company, and 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 an exclusive,
worldwide license agreement for Rocket to develop and commercialize gene
therapy treatments for Danon disease using REGENXBIO’s NAV AAV9 vector,
as well as exclusive options for two additional NAV AAV vectors for the
treatment of Danon disease.
Under the terms of the agreement, REGENXBIO has granted Rocket an
exclusive, worldwide license, with rights to sublicense, to REGENXBIO’s
NAV AAV9 vector for the development and commercialization of gene
therapy treatments for Danon disease. REGENXBIO has also granted Rocket
exclusive options to two additional undisclosed NAV AAV vectors for the
treatment of Danon disease for up to four years. In return for these
rights, REGENXBIO will receive a $7 million upfront payment, ongoing
fees, milestone payments and high-single to low-double digit royalties
on net sales of products incorporating the licensed intellectual
property.
“Rocket is delighted to partner with REGENXBIO to advance our first gene
therapy product derived from the NAV Technology Platform, reinforcing
the potential of our disease-focused, multi-platform development
approach allowing for the potential first-mover advantage without being
limited by vector type,” said Gaurav Shah, M.D., Chief Executive Officer
and President of Rocket. “Danon disease represents a significant area of
unmet medical need as no specific therapies are available for the
patients and families suffering from the disease. We believe the
well-understood disease biology and severe patient need makes Danon
disease an ideal target for gene therapy and we expect to enter the
clinic next year."
“This license agreement provides further validation of the potential for
the diverse application of our NAV Technology Platform for the treatment
of severe diseases with high unmet medical,” said Kenneth T. Mills,
President and Chief Executive Officer of REGENXBIO. “We are pleased to
initiate our partnership with Rocket, an emerging leader in gene therapy
for rare diseases, via development of a gene therapy treatment for Danon
disease.”
About Danon Disease
Danon disease is a rare neuromuscular and cardiovascular disease
characterized by profound cardiomyopathy, skeletal myopathies, and mild
cognitive impairment. It is estimated to have a prevalence of 15,000 to
30,000 patients in the U.S. and the European Union. Danon disease is
caused by mutations in the gene encoding lysosome-associated membrane
protein 2 (LAMP-2), an important mediator of autophagy. The LAMP-2
protein has three distinct variants generated by alternative splicing:
LAMP-2A, LAMP 2B and LAMP-2C. Mutations resulting in LAMP-2B dysfunction
are associated with severe cardiac disease features. The disease is
often fatal in patients in the second or third decade of life due to
progressive heart failure unless treated with a cardiac transplantation,
which is nonetheless not considered curative. There are no specific
therapies available for the treatment of Danon disease.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) (“Rocket”) is an emerging,
clinical-stage biotechnology company focused on developing
first-in-class gene therapy treatment options for rare, devastating
diseases. Rocket’s multi-platform development approach applies the
well-established lentiviral vector (LVV) and adeno-associated viral
vector (AAV) gene therapy platforms. Rocket's lead clinical program is a
LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a
difficult to treat genetic disease that leads to bone marrow failure and
potentially cancer. Preclinical studies of additional bone
marrow-derived disorders are ongoing and target Pyruvate Kinase
Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile
Malignant Osteopetrosis (IMO). Rocket’s first AAV-based gene therapy
program targets Danon disease, a rare neuromuscular and cardiovascular
disease. For more information about Rocket, please visit www.rocketpharma.com.
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.
Rocket Cautionary Statement Regarding Forward-Looking Statements
Various statements in this release concerning Rocket's future
expectations, plans and prospects, including without limitation,
Rocket's expectations regarding the safety, effectiveness and timing of
product candidates that Rocket may develop, including in collaboration
with academic partners, to treat Fanconi Anemia (FA), Leukocyte Adhesion
Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Infantile
Malignant Osteopetrosis (IMO), and the safety, effectiveness and timing
of related pre-clinical studies and clinical trials, may constitute
forward-looking statements for the purposes of the safe harbor
provisions under the Private Securities Litigation Reform Act of 1995
and other federal securities laws and are subject to substantial risks,
uncertainties and assumptions. You should not place reliance on these
forward-looking statements, which often include words such as "believe",
"expect", "anticipate", "intend", "plan", "will give", "estimate",
"seek", "will", "may", "suggest" or similar terms, variations of such
terms or the negative of those terms. Although Rocket believes that the
expectations reflected in the forward-looking statements are reasonable,
Rocket cannot guarantee such outcomes. Actual results may differ
materially from those indicated by these forward-looking statements as a
result of various important factors, including, without limitation,
Rocket's ability to successfully demonstrate the efficacy and safety of
such products and pre-clinical studies and clinical trials, its gene
therapy programs, the preclinical and clinical results for its product
candidates, which may not support further development and marketing
approval, Rocket's ability to commence a registrational study in FA
within the projected time periods, the potential advantages of Rocket's
product candidates, actions of regulatory agencies, which may affect the
initiation, timing and progress of pre-clinical studies and clinical
trials of its product candidates, Rocket's and its licensors ability to
obtain, maintain and protect its and their respective intellectual
property, the timing, cost or other aspects of a potential commercial
launch of Rocket's product candidates, Rocket's ability to manage
operating expenses, Rocket's ability to obtain additional funding to
support its business activities and establish and maintain strategic
business alliances and new business initiatives, Rocket's dependence on
third parties for development, manufacture, marketing, sales and
distribution of product candidates, the outcome of litigation, and
unexpected expenditures, as well as those risks more fully discussed in
the section entitled "Risk Factors" in Rocket's Annual Report on Form
10-K for the year ended December 31, 2017. Accordingly, you should not
place undue reliance on these forward-looking statements. All such
statements speak only as of the date made, and Rocket undertakes no
obligation to update or revise publicly any forward-looking statements,
whether as a result of new information, future events or otherwise.
REGENXBIO 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 and clinical
trials. 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, 2017 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20181126005167/en/
Source: Rocket Pharmaceuticals, Inc.
Rocket:
Claudine Prowse, Ph.D.
SVP, Corporate Strategy
and IRO
Rocket Pharma, Inc.
The Empire State Building, Suite
7530
New York, NY 10118
investors@rocketpharma.com
www.rocketpharma.com
REGENXBIO:
Investors
Natalie
Wildenradt, 646-681-8192
natalie@argotpartners.com
Media
Adam
Pawluk, 202-591-4063
apawluk@jpa.com