Taipei – October 05, 2026 -- AnnJi Pharmaceutical Co., Ltd. (TPEx: 7754) is proceeding with the U.S. portion of its pivotal Phase 3 ROMA-KD trial for AJ201, an oral small molecule targeting spinal and bulbar muscular atrophy (SBMA), also known as Kennedy's disease, a rare inherited neuromuscular disorder with no FDA-approved treatment.
AJ201 becomes the only investigational SBMA therapy in Phase 3 development worldwide
AnnJi submitted the Phase 3 protocol to the U.S. FDA under its active Investigational New Drug application and will now move to activate U.S. trial sites. The global, multicenter, randomized, double-blind, placebo-controlled ROMA-KD study is designed to evaluate efficacy and safety in ambulatory patients with symptomatic SBMA, with approximately 200 patients expected to enroll worldwide and the United States serving as a key region.
Regulatory designations accelerate the drug's pathway in two major markets
AJ201, also known as rosolutamide or JM17, has received Fast Track Designation from the U.S. FDA and Orphan Drug Designation in both the U.S. and the European Union. The molecule works by promoting clearance of the pathogenic mutant androgen receptor protein while activating cellular stress-response pathways including Nrf1, Nrf2 and HSF1, positioning it as a potential first-in-class disease-modifying treatment.
Phase 3 program builds on Phase 2 data announced in May 2025
The pivotal trial design was informed by subsequent interactions with regulatory authorities following the completed Phase 2 study results. AnnJi stated it will coordinate site activation across regions in line with its global development and partnering strategy, with the trial intended to support potential regulatory submissions worldwide.
AnnJi CEO cites patient impact as rationale for accelerated development
"For people living with SBMA, the disease is felt in everyday moments: a staircase that becomes harder to climb, a meal that becomes harder to swallow, a voice that becomes harder to use," said Dr. Wendy Huang, Chairperson and CEO of AnnJi, noting the company is now focused on activating U.S. sites and enrolling patients.
SBMA Patient and Care Partner Advisory Council established with Kennedy's Disease Association
AnnJi formed the Council in collaboration with the Kennedy's Disease Association, first announced at the 2026 KDA International Patient and Scientific Conference. The group provides a structured advisory forum for dialogue between AnnJi and the SBMA community on study design and patient-facing materials, though it operates independently of the clinical trial and has no bearing on enrollment eligibility.
SBMA is an X-linked disorder caused by a CAG repeat expansion in the androgen receptor gene, contributing to motor neuron and muscle degeneration through cellular toxicity, oxidative stress and neuroinflammation. The disease is considered substantially underdiagnosed, partly because symptoms are often mistaken for other neuromuscular conditions such as ALS.