Spur Therapeutics Releases Preclinical Data for SBT101 Gene Therapy in AMN

Spur Therapeutics has published promising preclinical data for its gene therapy program, SBT101, aimed at treating adrenomyeloneuropathy (AMN). AMN is a rare neurodegenerative disease caused by mutations in the ABCD1 gene, leading to progressive muscle weakness and sensory loss. These symptoms significantly impair mobility and quality of life, with no current treatment to slow the disease's progression.

The publication in Molecular Therapy Methods & Clinical Development highlights that SBT101 effectively delivers a functional ABCD1 gene to spinal cord tissues. This gene replacement results in the expression of the adrenoleukodystrophy protein (ALDP), which is deficient in AMN patients. The data showed improvements in function and addressed the underlying cause of the disease in preclinical models, paving the way for the initiation of a Phase 1/2 clinical trial known as the PROPEL study.

Key findings from the preclinical studies demonstrated that SBT101 has favorable safety and tolerability profiles in non-human primates (NHPs), with no treatment-related mortality or adverse clinical signs. Additionally, successful delivery of the ABCD1 gene to target tissues in the spinal cord was achieved in both mouse models and NHPs. This gene delivery led to increased ALDP production in the target tissues, reducing the harmful buildup of substrates associated with the ABCD1 gene deficiency. Significant improvements or prevention of loss in muscle grip strength, an important measure of muscle function, were observed in double knockout mouse models.

Pamela Foulds, M.D., Chief Medical Officer at Spur, emphasized the significance of these findings: “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.” She noted that the data demonstrate SBT101’s potential to halt disease progression by delivering a functioning ABCD1 gene, thereby generating the ALDP protein that AMN patients lack. Spur is advancing SBT101 in a Phase 1/2 trial with the goal of developing a transformative gene therapy for AMN patients.

Florian Eichler, M.D., Director of the Leukodystrophy Service at Massachusetts General Hospital and a co-author of the publication, commented on the study's impact: “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. The data demonstrate the ability of SBT101 to target the underlying disease mechanism of AMN and provide positive indications of preclinical efficacy.”

Spur Therapeutics expects to complete dosing in the Phase 1/2 PROPEL study early next year and plans to report initial safety data from the high-dose cohort in the first half of 2025.

Spur Therapeutics is dedicated to developing life-changing gene therapies for chronic conditions. By optimizing each component of its products, Spur aims to achieve significant clinical results. Besides SBT101 for AMN, Spur is also progressing a gene therapy candidate for Gaucher disease and exploring gene therapy applications for more prevalent diseases such as Parkinson’s, dementia, and cardiovascular disease.

The progress of SBT101 represents a significant advancement in the field of gene therapy, offering hope for AMN patients and potentially transforming the treatment landscape for other chronic conditions.