Spinal and bulbar muscular atrophy (SBMA) is a progressive neuromuscular disorder caused by the degeneration of lower motor neurons, resulting in muscle weakness and fasciculations. Since patients with SBMA have low concentrations of insulin-like growth factor-1 (IGM-1), treating the patients with an IGF-1 mimetic may help improve the condition. This study aims to evaluate the safety, tolerability, and preliminary efficacy of BVS857, an IGF-1 mimetic, in patients with SBMA.

This is a double-blind, placebo-controlled trial that included a total of 27patients aged 18 years or older with a confirmed diagnosis of SBMA. The participants were randomly assigned in a 2:1 ration to receive BVS857 (n=18) or placebo (n=9) for 12 weeks. The primary outcome of the study was safety, tolerability, and the effects of BVS857 as per the ANCOVA per protocol, and thigh muscle volume (TMV).

The researchers found BVS857 to be generally safe with no serious adverse events. However, no significant differences were identified between the BVS857 and the placebo groups. 13 of 18 patients (72%) in the BVS857 group showed signs of immunogenicity. TMV decreased in the placebo group but not in the BVS857 group.

The research concluded that TMV remained unchanged in SBMA patients who received BVS857, as compared with those who received a placebo. However, the intervention did not improve muscle strength or function.

Ref: https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(18)30320-X/fulltext