Positive phase 1 results in trial of novel therapy for spinal and bulbar muscular atrophy

Nido Biosciences has announced positive results from a phase 1 study of its novel, small molecule therapeutic to treat spinal and bulbar muscular atrophy in healthy male participants.

Data from the study were presented at the 2023 International Kennedy’s Disease Patient Conference, held in London from Nov. 4 to Nov. 5.

According to a company release, spinal and bulbar muscular atrophy (SBMA) is caused by an X-linked genetic mutation on the androgen receptor that results in the progressive loss of skeletal muscle and motor neuron function. The developing therapy works by binding to a distinct site on the androgen receptor and corrects transcriptional dysregulation to restore healthy cell function, per the release.

The randomized, double-blind, placebo-controlled, phase 1 study evaluated safety, tolerability and the pharmacokinetic profile of NIDO-361 in 64 individuals as a single-dose, oral treatment in dose-escalated cohorts followed by multiple doses in dose-escalated cohorts. Results showed that the drug was generally safe and well-tolerated when administered as a single or as multiple doses, with no serious treatment-emergent adverse events.

“We are excited by the potential of NIDO-361 to be a medicine that matters for SBMA patients as we continue our efforts to bring transformative therapies to patients suffering from debilitating neurological diseases,” Vissia Viglietta, MD, PhD, chief medical officer of Nido Biosciences, said in the release.

Nido plans to commence a phase 2 trial with enrollment anticipated to begin during the first quarter of 2024, according to the release.