AnnJi Pharmaceutical has received US FDA approval to proceed with the US arm of its pivotal Phase 3 study of AJ201 (rosolutamide) to treat spinal and bulbar muscular atrophy (SBMA or Kennedy’s disease). AJ201 has both US and EU orphan drug designation.

The ROMA-KD Phase 3 is a global, multi-centre, randomised, double-blind, placebo-controlled study, to evaluate the efficacy and safety of AJ201 in around 200 ambulatory individuals with SBMA.

AJ201 is an oral, small molecule that promotes the clearance of pathogenic mutant androgen receptor (mAR) protein by activating cellular stress-response pathways such as Nrf1, Nrf2, and HSF1.

SBMA is a rare, X-linked inherited neuromuscular disorder caused by a trinucleotide CAG repeat expansion of the androgen receptor gene, resulting in motor neuron and muscular degeneration. AnnJi proposes that SBMA is likely under-diagnosed, as symptoms are similar to other neuromuscular conditions, such as ALS, and with no currently approved drug for SBMA, genetic testing for the disease is not routine.