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
~ License of APB-102 further strengthens uniQure’s pipeline of innovative gene therapies to treat neurological disorders and miRNA-based gene silencing programs ~
~ APB-102 and uniQure’s c9orf72-ALS program have the potential to address most inherited forms of ALS ~
~ uniQure plans to initiate a Phase I/II trial of APB-102 in the second half of 2023 ~
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
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
Under the terms of the agreement, uniQure will make an initial cash payment of
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.
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
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,
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
- 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
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