Dear Patients, Caregivers, and Advocates,

In the rare disease community, progress is never measured only in milestones or data—it is measured in perseverance, hope, and the daily strength of patients and families. At AnnJi Pharmaceutical (AnnJi), we deeply understand that the journey toward an effective treatment for Spinal and Bulbar Muscular Atrophy (SBMA/Kennedy’s Disease) is a long and often uncertain marathon. We recognize the profound physical, emotional, and personal toll this disease places on individuals and their loved ones, and we want you to know that your resilience continually inspires our work. We share your urgency, your determination, and your hope for meaningful progress.

 

Since AJ201 first entered clinical development, we have been humbled by the trust you have placed in our science and in our team. Following the successful completion of our Phase 2a study, AJ201 has emerged as the most advanced first-in-class therapeutic candidate currently in development for SBMA. Our clinical results validated the proposed mechanism of action of AJ201—activating Nrf2 to reduce oxidative stress and promoting the degradation of toxic mutant androgen receptor (mAR). Importantly, we observed encouraging positive signals in both the 6-Minute Walk Test (6MWT) and the SBMA Functional Rating Scale (SBMAFRS) along with a manageable safety profile after only 12 weeks of treatment. These findings, together with the recent FDA Fast Track Designation, reinforce our belief that AJ201 holds real promise and that we are moving in the right direction.

 

In recent weeks, the broader SBMA clinical development landscape has changed significantly due to the latest setback against our peer. We know the news has been difficult and has left many in the community feeling uncertain and with fewer perceived options. We have heard your concerns and felt the increased urgency in your outreach to us. As the current forerunner in SBMA drug development, we do not take this responsibility lightly. We want to be clear and unequivocal: AnnJi remains steadfastly committed to seeing this program through, with the patients and families affected by SBMA at the center of every decision we make.

 

To ensure uninterrupted momentum, we are actively advancing the clinical study design and global infrastructure required to initiate a multinational pivotal Phase 3 trial—the final critical step toward regulatory approval. This rigorous early groundwork positions AJ201 for efficient late-stage development and future strategic partnerships. We will soon establish a dedicated communication cadence to share timely updates on trial readiness and key information.

Science advances most meaningfully when it addresses unmet medical needs and is grounded in strong partnership with the whole community. We are honored to walk alongside you—patients, caregivers, advocates, and clinicians—as we work to transform years of research into tangible hope. We remain committed to transparency, urgency, and compassion, and we will not stop until this medicine reaches those who need it most.

 

With deepest sincerity and unwavering resolve,

Dr. Wendy Huang

CEO 

AnnJi Pharmaceutical Co., Ltd.