Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and Abeona
Therapeutics Inc. (Nasdaq: ABEO) today announced an exclusive
license agreement for AAV gene therapy ABO-102 (now UX111) for the
treatment of Sanfilippo syndrome type A (MPS IIIA). Under the terms
of the agreement, Ultragenyx will assume responsibility for the
ABO-102 program and in return Abeona is eligible to receive tiered
royalties of up to 10% on net sales and commercial milestone
payments following regulatory approval.
“Based on promising data from Abeona’s clinical program,
regulatory feedback to date, and our experience developing
treatments for other MPS diseases, we believe ABO-102 has the
potential to be a transformative therapy for patients with MPS
IIIA,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer
and President of Ultragenyx. “Our team’s expertise in MPS and
gene therapy clinical development makes this program a seamless
integration, and it has the potential to be our first gene therapy
to market. The Sanfilippo community has been waiting too long for a
first treatment and we believe we can help accelerate this
program.”
“Data from the ongoing Transpher A trial demonstrate ABO-102
holds significant potential to improve outcomes for patients with
MPS IIIA who experience relentlessly progressing neurodevelopmental
and physical decline that is life-threatening at a very young age,”
said Vish Seshadri, Ph.D., Chief Executive Officer of Abeona. “We
believe that Ultragenyx, with deep expertise in rare, genetic,
metabolic lysosomal storage disorders and a demonstrated commitment
towards MPS diseases, is the ideal partner to eventually bring
ABO-102 to patients.”
Abeona has completed a successful Type B meeting with the U.S.
Food and Drug Administration (FDA) regarding the pivotal Transpher
A trial to support filing and approval for ABO-102 for the
treatment of patients with MPS IIIA. Interim results from the
Transpher A trial presented in an encore presentation at the
American Society of Gene & Cell Therapy (ASGCT) today
demonstrate that neurocognitive development was preserved in
children treated before 2 years old or with a development quotient
(DQ) > 60 (n=10) within normal range of a non-afflicted child
after treatment with ABO-102 (3x1013 vg/kg). The interim results
also showed continued or stabilized cognitive function and
behavioral progress using standard developmental assessments. Some
of these patients have reached 24-months post treatment and
stabilization or increase in cortical gray matter, total cerebral,
and amygdala volumes have been observed. Statistically significant
reduction in liver volume was seen with ABO-102 treatment.
Dose-dependent and statistically significant reductions in
cerebrospinal fluid and plasma heparan sulfate, demonstrating
replacement of enzyme activity consistent with levels required for
disease correction in the central nervous system, have been
sustained in treated patients for two years after treatment.
ABO-102 has been well-tolerated with no treatment-related serious
adverse events and no clinically meaningful adverse events
reported.
“MPS IIIA is characterized by severe neurodegeneration with
debilitating symptoms for which there is currently no treatment,”
said Kevin Flanigan, M.D., director of the Center for Gene Therapy
at Nationwide Children’s Hospital in Columbus, Ohio, and Transpher
A study principal investigator. “The promising results to date
suggest a single intravenous dose of ABO-102 AAV-based gene therapy
has the potential to help children with MPS IIIA sustain
neurocognitive development when they are treated during early
stages of their disease.”
About ABO-102 / UX111ABO-102 (now UX111), is a
novel gene therapy in Phase 1/2 development for Sanfilippo syndrome
type A (MPS IIIA), a rare lysosomal storage disease with no
approved treatment that primarily affects the central nervous
system (CNS). ABO-102 is dosed in a one-time intravenous infusion
using a self-complementary AAV9 vector to deliver a functional copy
of the SGSH gene to cells of the CNS and peripheral organs. The
therapy is designed to address the underlying SGSH enzyme
deficiency responsible for abnormal accumulation of
glycosaminoglycans in the brain and throughout the body that
results in progressive cell damage and neurodevelopmental and
physical decline. The ABO-102 program has received Regenerative
Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease, and
Orphan Drug designations in the U.S., and PRIME and Orphan
medicinal product designations in the EU.
About the Transpher A StudyThe Transpher A
Study (ABT-001) is an ongoing, two-year, open-label,
dose-escalation, Phase 1/2 global clinical trial assessing ABO-102
for the treatment of patients with Sanfilippo syndrome type A (MPS
IIIA). The study is intended for patients from birth to 2 years of
age, or patients older than 2 years with a cognitive developmental
quotient of 60% or above. ABO-102 gene therapy is delivered using
AAV9 technology via a single-dose intravenous infusion. The study
primary endpoints are neurodevelopment and safety, with secondary
endpoints including behavior evaluations, quality of life, enzyme
activity in cerebrospinal fluid (CSF) and plasma, heparan sulfate
levels in CSF, plasma and urine, and brain and liver volume.
Further details can be referenced here:
https://clinicaltrials.gov/ct2/show/NCT02716246
About Sanfilippo syndrome type A (MPS
IIIA)Sanfilippo syndrome type A (MPS IIIA) is a rare,
fatal lysosomal storage disease with no approved treatment that
primarily affects the CNS and is characterized by rapid
neurodevelopmental and physical decline, often by age three. MPS
IIIA has a global incidence of one in 100,000 with a median life
expectancy of 15 years.
Children with MPS IIIA present with progressive language and
cognitive decline and behavioral abnormalities. Other symptoms
include sleep problems and frequent ear infections. Additionally,
distinctive facial features with thick eyebrows or a unibrow, full
lips and excessive body hair for one’s age, and liver/spleen
enlargement are also present in early childhood. MPS IIIA is caused
by genetic mutations that lead to a deficiency in the SGSH enzyme
responsible for breaking down glycosaminoglycans, which accumulate
in cells throughout the body resulting in rapid health decline
associated with the disorder.
About Ultragenyx Pharmaceutical Inc.Ultragenyx
is a biopharmaceutical company committed to bringing novel
therapies to patients for the treatment of serious rare and
ultra-rare genetic diseases. The company has built a diverse
portfolio of approved medicines and treatment candidates aimed at
addressing diseases with high unmet medical need and clear biology,
for which there are typically no approved therapies treating the
underlying disease.
The company is led by a management team experienced in the
development and commercialization of rare disease therapeutics.
Ultragenyx’s strategy is predicated upon time- and cost-efficient
drug development, with the goal of delivering safe and effective
therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's
website at: www.ultragenyx.com.
About Abeona TherapeuticsAbeona Therapeutics
Inc. is a clinical-stage biopharmaceutical company developing cell
and gene therapies for serious diseases. Abeona’s lead clinical
program is EB-101, its investigational autologous, gene-corrected
cell therapy for recessive dystrophic epidermolysis bullosa in
Phase 3 development. The Company’s development portfolio also
features AAV-based gene therapies for ophthalmic diseases with high
unmet medical need. Abeona’s novel, next-generation AAV capsids are
being evaluated to improve tropism profiles for a variety of
devastating diseases. Abeona’s fully integrated gene and cell
therapy cGMP manufacturing facility produces EB-101 for the pivotal
Phase 3 VIITAL™ study and is capable of clinical and potential
commercial production of AAV-based gene therapies. For more
information, visit www.abeonatherapeutics.com.
Ultragenyx Forward-Looking Statements and Use of Digital
MediaExcept for the historical information contained
herein, the matters set forth in this press release, including
statements related to Ultragenyx's expectations and projections
regarding its business plans and objectives, the therapeutic
potential and clinical benefits of its products and product
candidates, expectations regarding the safety and tolerability of
its products and product candidates, and future clinical
developments or commercial success for its products or product
candidates are forward-looking statements within the meaning of the
"safe harbor" provisions of the Private Securities Litigation
Reform Act of 1995. Such forward-looking statements involve
substantial risks and uncertainties that could cause our clinical
development programs, collaboration with third parties, future
results, performance or achievements to differ significantly from
those expressed or implied by the forward-looking statements. Such
risks and uncertainties include, among others, the ability of
Ultragenyx and its third party partners to successfully develop
product candidates, including ABO-102 / UX111, the effects from the
COVID-19 pandemic on the company’s clinical and commercial
activities and business and operating results, risks related to
reliance on third party partners to conduct certain activities on
the company’s behalf, the potential for any license or
collaboration agreement, including the company’s license agreement
with Abeona as described in this press release, to be terminated,
uncertainty and potential delays related to clinical drug
development, the company’s ability to achieve its projected
development goals in its expected timeframes, risks and
uncertainties related to the regulatory approval process, smaller
than anticipated market opportunities for the company’s products
and product candidates, manufacturing risks, competition from other
therapies or products, and other matters that could affect
sufficiency of existing cash, cash equivalents and short-term
investments to fund operations, the company’s future operating
results and financial performance, the timing of clinical trial
activities and reporting results from same, and the availability or
commercial potential of Ultragenyx’s products and drug candidates.
Ultragenyx undertakes no obligation to update or revise any
forward-looking statements. For a further description of the risks
and uncertainties that could cause actual results to differ from
those expressed in these forward-looking statements, as well as
risks relating to the business of Ultragenyx in general, see
Ultragenyx's Quarterly Report on Form 10-Q filed with the
Securities and Exchange Commission (SEC) on May 5, 2022, and its
subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public
conference calls, Ultragenyx uses its investor relations website
and social media outlets to publish important information about the
company, including information that may be deemed material to
investors, and to comply with its disclosure obligations under
Regulation FD. Financial and other information about Ultragenyx is
routinely posted and is accessible on Ultragenyx’s investor
relations website (https://ir.ultragenyx.com/) and LinkedIn website
(https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).
Abeona Forward-Looking StatementsThis press
release contains certain statements that are forward-looking 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, and that involve risks and uncertainties. We have
attempted to identify forward-looking statements by such
terminology as “may,” “will,” “believe,” “estimate,” “expect,” and
similar expressions (as well as other words or expressions
referencing future events, conditions or circumstances), which
constitute and are intended to identify forward-looking statements.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, numerous risks and uncertainties, including but not
limited to the potential impacts of the COVID-19 pandemic on our
business, operations, and financial condition; continued interest
in our rare disease portfolio; our ability to commercialize our
EB-101 product candidate; obtaining a strategic partnership to take
over development activities for ABO-102; our ability to enroll
patients in clinical trials; the outcome of any future meetings
with the U.S. Food and Drug Administration or other regulatory
agencies; the impact of competition; the ability to secure licenses
for any technology that may be necessary to commercialize our
product candidates; the ability to achieve or obtain necessary
regulatory approvals; the impact of changes in the financial
markets and global economic conditions; risks associated with data
analysis and reporting; reducing our operating expenses and
extending our cash runway; our ability to execute our operating
plan and achieve important anticipated milestones; and other risks
disclosed in the Company’s most recent Annual Report on Form 10-K
and other periodic reports filed with the Securities and Exchange
Commission. The Company undertakes no obligation to revise the
forward-looking statements or to update them to reflect events or
circumstances occurring after the date of this press release,
whether as a result of new information, future developments or
otherwise, except as required by the federal securities laws.
ContactsUltragenyx Pharmaceutical Inc.
Investors Joshua Higair@ultragenyx.com
Media Jeff BlakeMedia@ultragenyx.com
Abeona Therapeutics Investors and Media Greg
Ginir@abeonatherapeutics.com
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