AnnJi Pharmaceutical Launches Phase 3 Trial for Kennedy's Disease

The ROMA-KD trial is a global, randomized, double-blind study designed to evaluate the safety and efficacy of AJ201 in approximately 200 ambulatory patients. By targeting the pathogenic mutant androgen receptor protein and activating cellular stress-response pathways, the drug seeks to mitigate the muscle degeneration and motor neuron damage characteristic of the disease. The development program follows encouraging results from a Phase 2 study completed in May 2025.

Dr. Wendy Huang, CEO of AnnJi, emphasized that the lack of existing FDA-approved therapies makes this trial a vital development for families affected by the condition. To ensure patient-centered research, the company has established an advisory council in collaboration with the Kennedy's Disease Association, providing a forum for those living with the disorder to share lived experiences that influence study design. AJ201 currently holds Fast Track and Orphan Drug designations in the U.S., as well as Orphan Drug status in the European Union.