Avenue Therapeutics has completed subject enrolment in the Phase Ib/IIa clinical trial of its new drug candidate, AJ201, to treat spinal and bulbar muscular atrophy (SBMA), or Kennedy’s disease.

The multicentre, randomised, 12-week, double-blind trial is designed to analyse a 600mg daily dose of AJ201 or a placebo.

It enrolled a total of 25 subjects who received either AJ201 or a placebo.

The trial’s primary goal is to evaluate the tolerability and safety of AJ201 in individuals with clinically and genetically confirmed SBMA.

Despite the disease's slow progression, which may limit the ability to show clinical efficacy over 12 weeks, the company incorporated secondary endpoints. These include biomarkers for proof of target engagement.

The biomarkers comprise pharmacodynamic information that evaluates changes from baseline in mutant androgen receptor protein levels in skeletal muscles, as well as alterations in fat and muscle composition.

Topline findings from the trial are expected in the second quarter of this year.

AJ201 is currently the lead drug candidate in clinical trials for SBMA treatment.

A Phase I study involving 72 healthy subjects demonstrated AJ201’s promising safety and pharmacokinetic profile.

AJ201 received orphan drug designation from the US Food and Drug Administration (FDA) for several polyglutamine (polyQ) ailments, such as SBMA, Huntington’s disease, and spinocerebellar ataxia.

The drug was licensed by Avenue from AnnJi Pharmaceuticals in the US, Canada, the European Union, Great Britain, and Israel.

Avenue CEO Alexandra MacLean said: “We are pleased with the speed of enrolment in our Phase Ib/IIa study evaluating AJ201 for the treatment of SBMA, reflecting the large unmet need in this debilitating neurodegenerative disease.

“We are intently focused on advancing this much-needed drug for patients and their caregivers, and we look forward to reporting topline data in the second quarter of 2024.”