uniQure and Apic Bio enter into global licensing agreement for APB-102, a clinical stage gene therapy for patients with ALS caused by mutations in SOD1
January 31 2023 - 7:05AM
uniQure N.V. (NASDAQ: QURE), a leading gene therapy company
advancing transformative therapies for patients with severe medical
needs, and Apic Bio, an innovative gene therapy company developing
novel treatment options for patients with rare genetic diseases,
today announced that they have entered into a global licensing
agreement for APB-102 to treat superoxide dismutase 1 (SOD1)
amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS.
Under the agreement, uniQure acquires global rights for the
development and commercialization of APB-102, adding to its
pipeline of gene therapies to treat neurological disorders. The
U.S. Food and Drug Administration has cleared the investigational
new drug (IND) application for APB-102 and has granted Orphan Drug
and Fast Track designations.
APB-102 is designed to be a novel, one-time,
intrathecally administered gene therapy for ALS caused by mutations
in SOD1, a rapidly progressing, rare motor neuron disease that
leads to loss of everyday functions and is uniformly fatal.
Mutations in the SOD1 gene of ALS account for approximately
one-fifth of all inherited forms of this fatal disease1. APB-102 is
comprised of a recombinant AAVrh10 vector that expresses a micro
ribonucleic acid (miRNA) designed to knock down the expression of
SOD1 with the goal of slowing down or potentially reversing the
progression of ALS in patients with SOD1 mutations.
“The licensing of APB-102 provides uniQure with
another clinical stage program that is strategically aligned with
our current pipeline and highly complementary with our AMT-161
program for the treatment of ALS caused by mutations in the c9orf72
gene,” stated Ricardo Dolmetsch, Ph.D. president of research and
development at uniQure. “Together, these ALS gene therapy
candidates have the potential to address most familial forms of ALS
and transform the lives of thousands of patients around the world
suffering from this devastating disease. We look forward to
initiating a Phase I/II clinical study of APB-102 in the second
half of 2023.”
The clinical development of APB-102 is based on
nearly 30 years of research demonstrating the link between the SOD1
gene mutation and ALS. Preclinical studies in a SOD1-ALS mouse
model demonstrated that APB-102 greatly enhanced survival in
affected mice. Relevant SOD1 reduction in spinal cord motor neurons
also was demonstrated in rodents, as well as in non-human primates
at proposed clinical doses.
“I am very proud of the contributions Apic Bio
has made to bring APB-102 to the cusp of clinical development,”
stated John Reilly, co-founder and chief executive officer of Apic
Bio. “uniQure is at the forefront of the field of miRNA gene
therapies for neurological disorders and is the ideal partner to
achieve the goal of rapidly advancing the clinical development of
APB-102 for the potential benefit of SOD1-ALS patients.”
Under the terms of the agreement, uniQure will
make an initial cash payment of $10 million. In addition, uniQure
will pay Apic Bio up to $45 million in milestones upon achievement
of regulatory approvals in the U.S. and Europe and pre-specified
annual net sales, and a tiered royalty on net sales ranging from
the mid-single digits to low double digits.
SVB Securities acted as exclusive financial
advisor to uniQure in the transaction. Torreya Partners acted as
exclusive financial advisor to Apic Bio in the transaction.
About SOD1-ALS
Amyotrophic lateral sclerosis (ALS) is a fatal
neurodegenerative disorder characterized by loss of motor neurons,
leading to muscle weakness and eventual paralysis. Most patients
face mortality within five years of disease onset due to
respiratory failure2. ALS can be caused by multiple genetic
mutations and can be sporadic (spontaneous mutations) or familial
(inherited mutations). Familial mutations account for approximately
ten percent of ALS cases, and of these, approximately twenty
percent are linked to a mutation in the SOD1 gene that codes for
the enzyme superoxide dismutase 11. SOD1-linked ALS is most likely
caused by toxic mutant forms of the superoxide dismutase 1 (SOD1)
protein (a gain-of-function mutation)1. Current approved ALS
treatments only delay disease progression without addressing the
underlying genetic causes of the disease.
About uniQure
uniQure is delivering on the promise of gene
therapy – single treatments with potentially curative results. The
recent approval of our gene therapy for hemophilia B – an historic
achievement based on more than a decade of research and clinical
development – represents a major milestone in the field of genomic
medicine and ushers in a new treatment approach for patients living
with hemophilia. We are now leveraging our modular and validated
technology platform to rapidly advance a pipeline of proprietary
gene therapies for the treatment of patients with Huntington's
disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and
other severe diseases. www.uniQure.com
About Apic Bio
Apic Bio is an innovative gene therapy company
focused on developing first-in-class treatment options for rare,
undertreated neurological and liver diseases. The Company’s
lead program to date has been an adeno-associated (AAV)-based gene
therapy for the treatment of SOD1 ALS. Preclinical studies of
additional genetic forms of ALS (C9orf72) and Alpha-1 Antitrypsin
Deficiency (Alpha-1) are ongoing. The Company is also advancing
discovery programs for two undisclosed CNS indications that
leverage its proprietary silence and replace THRIVE™ platform.
The Company is backed by leading and disease-centric investors,
including Morningside Venture Investments, ALS Investment Fund, and
The Alpha-1 Project (TAP). For more information, please
visit www.apic-bio.com.
uniQure Forward-Looking
Statements
This press release contains forward-looking
statements. All statements other than statements of historical fact
are forward-looking statements, which are often indicated by terms
such as "anticipate," "believe," "could," “establish,” "estimate,"
"expect," "goal," "intend," "look forward to", "may," "plan,"
"potential," "predict," "project," “seek,” "should," "will,"
"would" and similar expressions. Forward-looking statements are
based on management's beliefs and assumptions and on information
available to management only as of the date of this press release.
These forward-looking statements include, but are not limited to,
statements about whether uniQure’s ALS gene therapy candidates have
the potential to address most familial forms of ALS or transform
the lives of thousands of patients around the world, whether
uniQure will initiate a Phase I/II clinical study of APB-102 in the
second half of 2023, and whether uniQure will be able to rapidly
advance the clinical development of APB-102 for the potential
benefit of SOD1-ALS patients. The Company’s actual results could
differ materially from those anticipated in these forward-looking
statements for many reasons, including, without limitation, risks
associated with the regulatory approval and commercial launch of
HEMGENIX®, our clinical trial for Huntington’s disease, the impact
of financial and geopolitical events on our Company and the wider
economy and health care system, our Commercialization and License
Agreement with CSL Behring, our clinical development activities,
clinical results, collaboration arrangements, regulatory oversight,
product commercialization and intellectual property claims, as well
as the risks, uncertainties and other factors described under the
heading "Risk Factors" in the Company’s periodic securities
filings, including its Annual Report on Form 10-K filed February
25, 2022. Given these risks, uncertainties and other factors, you
should not place undue reliance on these forward-looking
statements, and the Company assumes no obligation to update these
forward-looking statements, even if new information becomes
available in the future.
References:
- Brown CA, Lally C, Kupelian V,
Flanders WD. Estimated Prevalence and Incidence of Amyotrophic
Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants.
Neuroepidemiology. 2021
- Brown RH, Al-Chalabi A. Amyotrophic
Lateral Sclerosis. N Engl J Med. 2017 Jul 13
uniQure Contacts:FOR
INVESTORS:Maria E. CantorDirect:
339-970-7536Mobile: 617-680-9452m.cantor@uniQure.comApic
Bio Contacts:FOR INVESTORS/MEDIA:David
Rosendavid.rosen@argotpartners.com646.461.6387 |
Chiara RussoDirect: 617-306-9137Mobile:
617-306-9137c.russo@uniQure.com |
FOR MEDIA:Tom MaloneDirect:
339-970-7558Mobile: 339-223-8541t.malone@uniQure.com |
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