- REGENXBIO to receive $110
million upfront, potential milestone payments of up to
$700 million and meaningful
double-digit royalties on net sales, and lead
manufacturing
- Nippon Shinyaku to lead commercialization of first potential
gene therapies for Mucopolysaccharidosis II (MPS II) and
Mucopolysaccharidosis I (MPS I) in U.S. and Asia
- REGENXBIO retains rights to RGX-121 Priority Review Voucher
(PRV) with potential accelerated approval expected in 2025;
rolling BLA submission underway
ROCKVILLE, Md., Jan. 14,
2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
and Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) today announced a
strategic partnership for the development and commercialization of
RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II),
also known as Hunter syndrome, and RGX-111 for
Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome.
Under the terms of the agreement, REGENXBIO will receive
$110 million at closing and up to an
additional $700 million if certain
milestones are achieved, consisting of $40
million in potential development and regulatory milestones
and $660 million in potential sales
milestones. REGENXBIO will also receive meaningful double-digit
royalties on net sales in the U.S. and Asia (collectively, the "Licensed
Territory").
"This partnership with Nippon Shinyaku is exciting in that it
maximizes our collective strengths and enables access of two
potentially transformational medicines to key markets," said
Curran M. Simpson, President and
Chief Executive Officer, REGENXBIO. "The structure of the agreement
allows us to leverage our expertise in gene therapy manufacturing
while also capturing milestones and a meaningful share of future
product revenues. RGX-121 is poised to be the first gene therapy
for MPS II with potential FDA approval as early as late 2025, and
RGX-111 has demonstrated very promising results in Phase 1/2 study.
With Nippon Shinyaku's expertise in rare disease and strong
commercial capabilities, we look forward to working together to get
both of these promising candidates across the finish line for
patients."
"RGX-121 and RGX-111 represent one-time gene therapies that can
potentially change the course of MPS disease, and we are very
pleased to be partnering with REGENXBIO, experts in gene therapy
development and manufacturing," said Toru
Nakai, President and Representative Director of Nippon
Shinyaku. "We are confident these therapies can bring tremendous
value to those living with MPS II and I."
Per the agreement, Nippon Shinyaku will commercialize both
products in the Licensed Territory and future clinical development
of RGX-121 and RGX-111 will be led by REGENXBIO. REGENXBIO retains
all rights to, and 100 percent of any proceeds related to the sale
of, the Priority Review Voucher (PRV) for RGX-121 received upon
potential approval.
REGENXBIO will lead the manufacturing of both products for
clinical and commercial supply in the Licensed Territory. REGENXBIO
reserves the right to develop and commercialize these products in
countries outside of the Licensed Territory.
The transaction is expected to close by the end of the first
quarter of 2025, subject to the customary conditions, including
applicable regulatory approvals.
About RGX-121
RGX-121 is a potential one-time AAV
therapeutic for the treatment of boys with MPS II. RGX-121
expressed protein is structurally identical to normal I2S. Delivery
of the IDS gene within cells in the CNS could provide a permanent
source of secreted I2S beyond the blood-brain barrier, allowing for
long-term cross correction of cells throughout the CNS.
RGX-121 has received Orphan Drug Product, Rare Pediatric
Disease, Fast Track and Regenerative Medicine Advanced Therapy
designations from the U.S. Food and Drug
Administration and advanced therapy medicinal products (ATMP)
classification from the European Medicines Agency.
About RGX-111
RGX-111 is designed to use the AAV9
vector to deliver the α-l-iduronidase (IDUA) gene to the central
nervous system (CNS). By providing rapid IDUA delivery to the
brain, RGX-111 could potentially help prevent the progression of
cognitive deficits that otherwise occurs in MPS I patients.
Positive interim data from a Phase I/II trial of RGX-111 were
reported in February 2023. RGX-111
has received orphan drug product, rare pediatric disease and Fast
Track designations from the U.S. Food and Drug Administration
(FDA).
ABOUT REGENXBIO Inc.
REGENXBIO is a leading
clinical-stage biotechnology company seeking to improve lives
through the curative potential of gene therapy. Since its founding
in 2009, REGENXBIO has pioneered the development of AAV
Therapeutics, an innovative class of gene therapy medicines.
REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal
and rare diseases, including ABBV-RGX-314 for the treatment of wet
AMD and diabetic retinopathy, being developed in collaboration with
AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the
treatment of MPS II. Thousands of patients have been treated with
REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA
for children with spinal muscular atrophy. Designed to be one-time
treatments, AAV Therapeutics have the potential to change the way
healthcare is delivered for millions of people. For more
information, please visit www.regenxbio.com.
ABOUT NIPPON SHINYAKU
Based on Nippon
Shinyaku's business philosophy, "Helping people lead healthier,
happier lives," we aim to be an organization trusted by the
community through creating unique medicines that will bring hope to
patients and families suffering from illness. Please visit our
website (www.nippon-shinyaku.co.jp/english/) for products or
detailed information.
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, and
regulatory plans. 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 anticipated completion of REGENXBIO's proposed
transaction with Nippon Shinyaku, the outcome of REGENXBIO's
proposed collaboration with Nippon Shinyaku, whether the milestones
contemplated by the proposed transaction will be achieved, 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, 2023, 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.
Contacts:
Dana
Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com
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