Data show SBT101 effectively delivered a functional copy of the ABCD1 gene to spinal cord tissues with resultant protein expression, addressing the underlying cause of disease and improving function in preclinical models
Highlighted in peer-reviewed journal Molecular Therapy Methods & Clinical Development, these data supported the advancement of SBT101 into a Phase 1/2 clinical trial
Expect to report updated safety data from the ongoing trial in first half of 2025
Spur Therapeutics today announced the publication of preclinical proof-of-concept data for SBT101, its potential first-in-class gene therapy program for the treatment of adrenomyeloneuropathy (AMN), in Molecular Therapy Methods & Clinical Development. AMN is a rare and devastating neurodegenerative disease caused by a mutation in the ABCD1 gene and characterized by progressive muscle weakness and sensory loss, leading to impaired mobility, increased risk of falls, incontinence and debilitating pain.
“People living with AMN currently have no treatment options to slow or alter the progression of this devastating disease, and gene therapy presents the opportunity to address the disease at its root cause,” said Pamela Foulds, M.D., Chief Medical Officer at Spur. “These data demonstrate SBT101’s potential to halt disease progression by delivering a functioning ABCD1 gene to generate the adrenoleukodystrophy protein (ALDP) lacking in AMN. We are currently advancing SBT101 in a Phase 1/2 trial with the aim of developing a life-changing gene therapy for people with AMN.”
The studies highlighted in the publication, titled “An in vitro and in vivo efficacy evaluation of a novel gene therapy candidate (SBT101) in mouse models of adrenomyeloneuropathy and in NHPs,” supported SBT101’s advancement into the PROPEL study[1].
Key findings from the studies include:
- Favorable safety and tolerability, with no treatment-related mortality or clinical signs observed in non-human primates (NHPs).
- Successful delivery of the ABCD1 gene to target tissues in the spinal cord in mouse models of the disease and NHPs.
- Increased ALDP protein production in the target tissues, resulting in reductions in the harmful buildup of substrates caused by the deficiency of the ABCD1 gene in AMN.
- Significant improvement or prevention of loss in grip strength, an established metric for assessing muscle strength, were seen in double knockout mouse models.
“These studies validate SBT101’s potential to be a first-in-class gene therapy for AMN, which could change the lives of thousands of people with this progressive and debilitating disease,” said Florian Eichler, M.D., Director of Leukodystrophy Service at Massachusetts General Hospital, a co-author of the publication. “These data demonstrate the ability of SBT101 to target the underlying disease mechanism of AMN and provide positive indications of preclinical efficacy.”
Spur expects to complete dosing in the Phase 1/2 PROPEL study of SBT101 early next year and report initial safety data from the high-dose cohort in the first half of 2025. More information about the trial can be found at ClinicalTrials.gov.
About Spur Therapeutics
Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine.
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For more information, visit www.spurtherapeutics.com or connect with Spur on LinkedIn and X.
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[1] Spur Therapeutics acquired SwanBio Therapeutics in 2024, adding SBT101 to its pipeline.