--- title: "賦能 SBMA 社區：AnnJi 在 2026 年 KDA 會議上突出患者與家庭論壇，並分享 AJ201 的科學進展" type: "News" locale: "zh-HK" url: "https://longbridge.com/zh-HK/news/277860992.md" description: "安吉製藥有限公司參加了 2026 年 KDA 會議，展示了 AJ201 在脊髓和延髓肌萎縮症（SBMA）方面的進展，這是一種潛在的首創療法。公司分享了令人鼓舞的二期臨牀結果，並公佈了全球三期試驗的設計，旨在評估 AJ201 的療效和安全性。安吉還舉辦了患者與家庭論壇，以與 SBMA 社區進行互動，並宣佈成立患者顧問委員會，以優化試驗設計。公司始終致力於滿足 SBMA 患者的未滿足需求" datetime: "2026-03-05T01:43:12.000Z" locales: - [zh-CN](https://longbridge.com/zh-CN/news/277860992.md) - [en](https://longbridge.com/en/news/277860992.md) - [zh-HK](https://longbridge.com/zh-HK/news/277860992.md) --- # 賦能 SBMA 社區：AnnJi 在 2026 年 KDA 會議上突出患者與家庭論壇，並分享 AJ201 的科學進展 TAIPEI, March 4, 2026 /PRNewswire/ -- AnnJi Pharmaceutical Co., Ltd. (AnnJi, TWSE: 7754) announced its participation in the 2026 International Patient and Scientific Conference hosted by the Kennedy's Disease Association (KDA). Held between February 27 and March 2, the conference served as a premier forum bringing together patients with Spinal and Bulbar Muscular Atrophy (SBMA), caregivers, clinicians and leading researchers to discuss advances in disease science, therapeutic development, and clinical care. Building on the recent U.S. FDA Fast Track Designation for AJ201(Rosolutamide) and encouraging Phase 2 clinical results, AnnJi presented two scientific abstracts spanning translational transcriptomic findings through highly anticipated Phase 3 trial. These presentations underscored AJ201's multi-modal mechanism of action and its potential as a first-in-class therapy within AnnJi's strategic roadmap paving the way for global Phase 3 trial. **Scientific Highlights and Abstract Presentations of AJ201 during the KDA Conference** - **Transcriptomic Insights:** Research titled _"Transcriptomic Evidence of AJ201 Target Engagement and Biological Response in SBMA Muscle"_ utilized RNA-seq to demonstrate that AJ201 engages the Nrf2 antioxidant pathway. The data confirmed that treatment modulates multiple biological axes rather than a single pathway, reinforcing AJ201's drug-specific biological engagement. - **Phase 3 Trial Design:** AnnJi unveiled the preliminary design of its global, multicenter Phase 3 trial. This proposed randomized, double-blind, placebo-controlled trial is designed to evaluate the efficacy and safety of AJ201 in ambulatory individuals with symptomatic SBMA. The trial represents a critical step toward potential global regulatory registration and aims to further validate AJ201's therapeutic benefit in this underserved patient population. Additionally, Dr. Christopher Grunseich, MD, the study's Principal Investigator, led a featured session titled _"Safety and Preliminary Efficacy Demonstrated in a Phase 1/2a Clinical Trial of AJ201 in Spinal and Bulbar Muscular Atrophy."_ During the presentation, he shared data highlighting AJ201's favorable safety profile, early signals of efficacy observed in the Phase 2 study, and key updates on the overall clinical development program. **Steadfast Commitment to the SBMA Community** "We are honored to stand alongside patients and advocates as we work to translate scientific progress into tangible hope," said Dr. Wendy Huang, CEO and Chairperson of AnnJi. "The positive clinical findings, together with the recent FDA Fast Track Designation, strengthen our confidence in AJ201's potential and affirm that we are advancing in the right direction." Amid recent clinical setbacks of other candidates in the field, AnnJi continues to lead SBMA drug development. The company has intensified preparations to initiate its multinational Phase 3 trial, with participant enrollment anticipated to commence in late 2026. This momentum reflects AnnJi's unwavering commitment to advancing AJ201 until it reaches the patients who need it most. To further this mission, AnnJi also hosted a "Patient & Family Forum" during the 2026 KDA Conference. Recognizing that patients and caregivers bring invaluable lived expertise beyond what scientific publications alone can capture, this forum provided a platform for open dialogue and transparent engagement. The event introduced the mechanism and development of AJ201, explored potential partnership opportunities, and announced the formation of a Patient Advisory Council in collaboration with KDA. AnnJi aims to partner with SBMA patients and families to help optimize the Phase 3 trial design, address potential challenges proactively, and accelerate the path toward delivering new hope to SBMA families worldwide. **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 developing 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 2 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 View original content to download multimedia:https://www.prnewswire.com/news-releases/empowering-the-sbma-community-annji-highlights-patient--family-forum-and-shares-aj201-scientific-advancement-at-the-2026-kda-conference-302704676.html SOURCE AnnJi Pharmaceutical Co., Ltd. ### 相關股票 - [4528.JP](https://longbridge.com/zh-HK/quote/4528.JP.md) - [4886.JP](https://longbridge.com/zh-HK/quote/4886.JP.md) - [3671.JP](https://longbridge.com/zh-HK/quote/3671.JP.md) ## 相關資訊與研究 - [港大團隊開創新型混合療法 提高急性骨髓性白血病患者存活率](https://longbridge.com/zh-HK/news/287004908.md) - [希瑪醫療逾 1 億人幣收購眼科基因療法新創企業北京中因科技逾 10% 股權](https://longbridge.com/zh-HK/news/287136053.md) - [數字療法：虛擬情感互動治癒社交焦慮](https://longbridge.com/zh-HK/news/287278193.md) - [國際關注 | FDA 推動實時臨牀試驗（RTCT）利好大型製藥公司，小型生物技術公司如何應對？](https://longbridge.com/zh-HK/news/287228700.md) - [Cell Rep：破解 CAR-T 療法失效之謎！患者 T 細胞 “衰老” 是罪魁禍首，新研究為精準篩選獲益人羣指明方向](https://longbridge.com/zh-HK/news/287171706.md)