Rosolutamide

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. Continue Reading 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-α, IL-6, TGF-β) 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; [email protected] Sylvia Lin; [email protected] SOURCE AnnJi Pharmaceutical Co., Ltd. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

TAIPEI, May 22, 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 trial of AJ201 in adults with Spinal and Bulbar Muscular Atrophy (SBMA). Conducted across six U.S. clinical sites, the study evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of AJ201 (ClinicalTrials.gov Identifier: NCT05517603). While not powered to assess efficacy, exploratory endpoints revealed meaningful treatment-related improvements that support continued clinical development. Safety and Pharmacokinetics The safety and pharmacokinetic profiles in SBMA patients were consistent with prior healthy volunteer data. AJ201 was generally well tolerated, and no systemic drug accumulation was observed. Positive Clinical Signals After 12 weeks of oral treatment, AJ201 recipients showed clinically meaningful improvements in physical and muscle function compared to placebo, including a 17.6-meter gain in the 6-Minute Walk Test (6MWT) and a 0.8-point increase in the SBMA Functional Rating Scale (SBMAFRS) on average, while the placebo group experienced slight declines. AJ201 also led to reduction in serum creatine kinase and myoglobin levels—suggesting a positive therapeutic effect. Most responders are in the AJ201 group: 11 of 15 for 6MWT, 6 of 7 for SBMAFRS, 14 of 14 for creatine kinase, and 11 of 12 for myoglobin, further supporting AJ201's benefit. Additionally, patients in AJ201 group reported significant improvement in physical function component of SF36v2 quality-of-life questionnaire, while placebo group a decline (p=0.026). Biomarker Findings and RNA Sequencing Reveal Mechanistic Support Mutant androgen receptor (mAR) levels, a proposed biomarker for SBMA, were assessed via muscle biopsies. Nuclear mAR levels were reduced by more than 50% in 53% of AJ201-treated patients, compared to 17% of placebo, indicating potential therapeutic activity. RNA sequencing of muscle biopsies from AJ201-treated patients revealed activation of the Nrf2 pathway, along with modulation of several disease-relevant signaling cascades. These changes, absent in the placebo group, offer support for AJ201's therapeutic mechanism of action. Conclusion and Expert Commentary Consistent improvements across functional, biochemical, and molecular markers observed in this study support continued development of AJ201 for SBMA. "The study results are highly encouraging. AJ201 has shown evidence of clinical benefit, demonstrated through improvements in functional assessments, positive shifts in serum biomarkers, and RNA sequencing data supporting activation of the Nrf2 pathway. Together, these findings reinforce the therapeutic potential of AJ201," said Dr. Christopher Grunseich, Principal Investigator of the study. Dr. Grunseich is a Lasker Clinical Research Scholar and Head of the Inherited Neuromuscular Diseases Unit at the National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH). "SBMA is a slowly progressing neuromuscular disorder, and I am greatly encouraged by the positive clinical outcomes observed after a relatively short course of AJ201 treatment," said Wendy Huang, Ph.D., Chief Executive Officer and Chairperson of the Board at AnnJi. "AnnJi is committed to advancing the program into Phase 3 clinical trials, with the aim of delivering a safe, effective, and much-needed therapeutic option for patients living with SBMA—a disease that currently lacks any FDA-approved treatments." About SBMA and AJ201 SBMA, or Kennedy's disease, is a rare X-linked inherited neuromuscular disorder caused by CAG repeat expansion in the androgen receptor (AR) gene. The resulting mutant AR protein contributes to muscle and neuron degeneration through mechanisms involving cellular toxicity, oxidative stress, and neuroinflammation. SBMA affects ~1 in 40,000 males globally and currently has no FDA-approved treatment. AJ201, also known as JM17, is a novel investigational compound that has shown potential in reducing mutant AR toxicity and improving motor function in preclinical SBMA models. At the molecular level, it promotes degradation of pathogenic mAR protein, induces expression of antioxidant enzymes, proteasome subunits, and heat shock proteins, all of which may slow disease progression (Bott et al., 2016). About AnnJi Pharmaceutical AnnJi Pharmaceutical Co., Ltd., founded in 2014, is a clinical-stage biotech company focused on developing first-in-class small molecule therapies for serious and underserved diseases. Our pipeline spans neurology, dermatology, and rare disorders like SBMA. AnnJi is committed to translating cutting-edge science into differentiated treatments and advancing them through global collaboration and commercialization. For more information, visit: . Forward-Looking Statements This press release contains forward-looking statements regarding AnnJi's plans, programs, and expected results. These statements are based on current assumptions and subject to risks and uncertainties that could cause actual outcomes to differ materially. AnnJi undertakes no obligation to update forward-looking statements except as required by law. Contact Anne Yu AnnJi Pharmaceutical Co., Ltd. (+886) 2-2365-5677 [email protected] WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED