TAIPEI, Oct. 13, 2025 /PRNewswire/ -- AnnJi Pharmaceutical Co., Ltd. (AnnJi, TWSE: 7754) announced that the Phase 2a clinical result of AJ201 for spinal and bulbar muscular atrophy (SBMA, also known as Kennedy's disease) was selected as one of the seven Late-Breaking Abstracts at the World Muscle Society (WMS) 2025 International Congress in Vienna. Dr. Tahseen Mozaffar, an investigator of the study, presented the study outcome, emphasizing the therapeutic potential of AJ201 for SBMA patients.
AJ201, an oral suspension of a novel curcumin analog-Rosolutamide, offers ease of use for patients with swallowing difficulties. Mechanistically, AJ201 acts through multiple pathways, including Nrf2 activation, oxidative stress modulation, and induction of heat shock proteins to facilitate clearance of mutant protein aggregates. This multi-modal mechanism positions AJ201 as a pioneering small-molecule therapy for PolyQ neurodegenerative disorders, such as SBMA. In the Phase 2a trials, positive functional outcomes were reported in AJ201 treated SBMA patients, highlighting AJ201's potential as a first-in-class therapy for SBMA in two decades.
Dr. Andrew Pai presented the latest preclinical research of Rosolutamide (JM17), the active ingredient of AJ201. JM17 demonstrated multi-pathway benefits in SBMA mouse model, including enhanced mitochondrial function, reduced inflammation and fibrosis, as well as improved structural organization of muscle fibers. Upstream regulator analysis further confirmed the inhibition of pro-inflammatory mediators (TNF-a, IL-6, TGF-ß) alongside activation of protective signaling pathways. These findings underscore JM17's potential as a multi-functional therapeutic candidate, providing new mechanistic insights for SBMA treatment.
"We are very proud of the Late Breaking Abstract, as well as the preclinical research paper presented at the 2025 WMS, which highlighted the significant advancement we have made in the development of AJ201 toward the world's first effective therapy for Kennedy's disease." said Dr. Wendy Huang, CEO and Chairperson of the Board of AnnJi.
AJ201 has been granted Orphan Drug Designation by both the U.S. FDA and EMA. AnnJi has also built a robust global patent portfolio, securing strong market exclusivity for AJ201. Looking ahead, AnnJi continues advancing AJ201 into Phase 2/3 clinical development and global licensing partnerships, aiming to deliver new hope for patients worldwide and establish AJ201 as a landmark success for Taiwan's new drug industry on the international stage.
About Kennedy's Disease (SBMA)
Kennedy's disease, also known as Spinal and Bulbar Muscular Atrophy (SBMA), is a rare and serious inherited neuromuscular degenerative disorder. It is characterized by the progressive degeneration of lower motor neurons in the spinal cord, brainstem, and skeletal muscles. The disease primarily affects males between the ages of 30 and 40, with an estimated prevalence of 1 in 40,000. As the disease progresses-typically by age 50-patients often develop difficulties with chewing and swallowing, and ultimately, recurrent aspiration pneumonia becomes a common cause of death. Currently, there are no approved treatments for SBMA.
About AnnJi Pharmaceutical
AnnJi Pharmaceutical Co., Ltd. (TWSE: 7754) is a clinical-stage drug development company focused on the development of innovative small molecules (NCEs). The company is dedicated to addressing high unmet medical needs in the fields of neurology, dermatology, and immune-inflammatory diseases, including rare diseases, such as Kennedy's disease (SBMA) and Idiopathic Pulmonary Fibrosis (IPF).
AnnJi is committed to develop innovative therapies that improve the quality of life for patients suffering from neglected chronic diseases. We focus on advancing high-quality, differentiated, and innovative drug candidates, and collaborating with global pharmaceutical partners. Upon achieving proof-of-concept in phase II clinical trials, we aim to out-license our products for further co-development and commercialization, with the goal of bringing them to global markets and driving sustainable growth for the company.
Contact:
Anne Yu; anne.yu@ajpharm.com
Sylvia Lin; sylvia.lin@ajpharm.com
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