Core Viewpoint - The announcement by He Yu-B (02256) regarding the acceptance of the New Drug Application (NDA) for the oral, highly selective, and effective small molecule CSF-1R inhibitor, Beijiemai (ABSK021), by the FDA marks a significant milestone for the treatment of TGCT patients [1][2]. Group 1: Drug Development and Approval - Beijiemai, developed by He Yu Pharmaceutical, has received FDA acceptance for its NDA, based on positive efficacy and safety results from the global Phase III MANEUVER study [1]. - The drug is expected to be commercially available globally through a partnership with Merck [1]. - Beijiemai was previously approved by the NMPA in China in December 2025 for symptomatic TGCT adult patients who may face functional limitations or severe complications from surgical removal [1]. Group 2: Clinical Efficacy and Safety - The Phase III study demonstrated statistically significant improvement in the objective response rate (ORR) for TGCT patients treated with Beijiemai, assessed at week 25 according to RECIST v1.1 standards [1]. - All secondary endpoints related to key clinical outcomes for TGCT patients showed significant and clinically meaningful improvements, including enhanced joint range of motion and physical function, as well as reduced stiffness and pain [2]. - Long-term data from a median follow-up of 14.3 months indicated a continuous increase in ORR for patients who received Beijiemai treatment from the start of the study [3]. Group 3: Market Potential and Clinical Need - TGCT is a rare locally aggressive tumor that can lead to joint swelling, stiffness, and limited mobility, significantly impacting patients' daily activities and quality of life [3]. - If untreated or recurrent, TGCT can cause irreversible damage to bones, joints, and surrounding tissues [3]. - The ongoing regulatory submissions for Beijiemai in major global markets position the drug as a promising daily oral treatment option that is effective and well-tolerated, addressing unmet clinical needs in the TGCT field [3].

Core Viewpoint - The announcement highlights the acceptance of the New Drug Application (NDA) for the novel oral, highly selective, and effective small molecule CSF-1R inhibitor, Beijimai (ABSK021), by the FDA for the treatment of TGCT patients, indicating a significant advancement in addressing an unmet clinical need in this rare tumor type [1][2]. Group 1 - Beijimai is developed by Shanghai Heyu Biopharmaceutical Technology Co., Ltd. and is licensed to Merck for global commercialization [1]. - The NDA acceptance is based on positive efficacy and safety results from the global multi-center, randomized, double-blind, placebo-controlled Phase III MANEUVER study [1]. - The study demonstrated a statistically significant improvement in the objective response rate (ORR) for TGCT patients treated with Beijimai, assessed at week 25 according to RECIST v1.1 criteria [1]. Group 2 - All secondary endpoints related to key clinical outcomes for TGCT patients showed significant and clinically meaningful improvements, including enhanced joint range of motion and physical function, as well as reductions in stiffness and pain [2]. - Long-term data with a median follow-up of 14.3 months indicated a sustained increase in ORR for patients who received Beijimai treatment from the start of the study [2]. - TGCT is a rare locally aggressive tumor that can lead to joint swelling, stiffness, and limited mobility, significantly impacting patients' daily activities and quality of life [2].

Core Viewpoint - The new drug application (NDA) for the oral, highly selective, and effective small molecule CSF-1R inhibitor, Beijiemai® (ABSK021), developed by the subsidiary of He Yu-B (02256.HK), has been officially accepted by the U.S. Food and Drug Administration (FDA) for the systemic treatment of patients with tenosynovial giant cell tumor (TGCT) [1]. Group 1 - The drug Beijiemai® is a novel oral treatment targeting CSF-1R, indicating a significant advancement in the therapeutic options for TGCT patients [1]. - The acceptance of the NDA by the FDA marks a critical milestone for the company and its product development pipeline [1].