A Phase 1/2 Randomized, Blinded, Dose-escalation Study to Evaluate the Safety and Efficacy of Intrathecal Administration of AAV9-ABCD1 Gene Therapy (SBT101) in Adult Patients with Adrenomyeloneuropathy

Adrenomyeloneuropathy

Percentage of patients with at least 1 Grade III or Grade IV AE that is at least possibly related to SBT101 at Month 24, as reported by the patients or observed by the Investigator, after SBT101 treatment

• Changes in the 6-minute Walk Test (6MWT) at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study., • Changes in the 2-minute Walk Test (2MWT) at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study., • Changes in the 5 times Sit-to-Stand Test (5XSST) at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study., • Changes in postural body sway amplitudes at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study., • Proportions of patients with Expanded Disability Status Score (EDSS) of ≥5.5 at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study., • Proportions of patients with increased anti-spasticity medication dose at Month 12 and Month 24 compared to controls, and compared to the reference changes determined by the prospective SBTNHX-CT901 study., • Changes in Timed Up and Go (TUG) at Month 12 and Month 24 compared to controls, and compared to the changes observed in the prospective SBTNHX-CT901 study., • Changes in Dual-task TUG (TUG-DT) at Month 12 and Month 24 compared to controls, and compared to the changes observed in the prospective SBTNHX-CT901 study., • Changes in Balance Evaluation System Test (miniBESTest) at Month 12 and Month 24 compared to controls, and compared to the changes observed in the prospective SBTNHX-CT901 study., • Changes in Clinical Global Impression (CGI; performed by a qualified professional) score at Month 12 and Month 24 compared to controls., • Changes in Patient Global Impression of Severity (PGI-S), Patient Global Impression of Severity – Balance (PGI-S Balance), and Patient Global Impression of Change – Balance (PGI-C Balance) scores at Month 12 and Month 24 compared to controls, and compared to the changes observed in the prospective SBTNHX-CT901 study., • Percentage of patients with at least 1 Grade III or Grade IV AE that is at least possibly related to SBT101 at Month 60, as reported by the patients or observed by the Investigator, after SBT101 treatment.

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