Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

A Two-part Placebo-controlled Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)

The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).

Key Inclusion Criteria:

• Genetic diagnosis of SBMA with symptomatic muscle weakness
• Able to complete 2 minute timed walk
• Serum IGF-1 level less than or equal to 170 ng/mL

Key Exclusion Criteria:

• Medically treated diabetes mellitus or known history of hypoglycemia
• History of Bell's palsy
• Treatment with systemic steroids > 10 mg/day (or equivalent dose); androgens or androgen reducing agents; systemic beta agonists; or other muscle anabolic drugs within the previous 3 months
• History of cancer, other than non-melanomatous skin cancer
• Retinopathy
• Papilledema Other protocol defined inclusion/exclusion criteria may apply