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AnnJi Pharmaceutical Company Announces Positive Phase 1/2a Results for AJ201 in Spinal and Bulbar Muscular Atrophy (SBMA) Patients

  • On May 21, 2025, AnnJi Pharmaceutical reported favorable outcomes from its Phase 1/2a clinical study of AJ201, conducted in adult patients diagnosed with Spinal and Bulbar Muscular Atrophy.
  • The randomized, double-blind, placebo-controlled study in six US sites tested AJ201’s safety, pharmacokinetics, and pharmacodynamics in SBMA patients.
  • After 12 weeks of treatment, participants receiving AJ201 demonstrated notable enhancements in muscle and physical function, with an average improvement of 17.6 meters on the 6-Minute Walk Test and a 0.8-point rise in their SBMA Functional Rating Scale scores.
  • Dr. Christopher Grunseich highlighted that the observed results provide strong evidence supporting the effectiveness of AJ201, noting improvements that were not seen in the placebo group.
  • These results support AJ201’s continued development, with AnnJi planning Phase 3 trials to deliver a much-needed treatment for SBMA, which presently lacks FDA-approved options.
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AnnJi Pharmaceutical Company Announces Positive Phase 1/2a Results for AJ201 in Spinal and Bulbar Muscular Atrophy (SBMA) Patients

TAIPEI, May 21, 2025 /PRNewswire/ -- AnnJi Pharmaceutical Co., Ltd., a clinical-stage Taiwanese biotechnology company focused on addressing unmet medical needs in dermatology, neurology, and rare diseases, today announced positive results from its Phase 1/2a randomized, double-blind, placebo-controlled, first-in-patient clinical…

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Charleston Gazette-Mail broke the news in Charleston, United States on Thursday, May 22, 2025.
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