Harbour BioMed Resubmits Batoclimab BLA to NMPA for Generalized Myasthenia Gravis

Harbour BioMed (HKEX: 02142), a global biopharmaceutical enterprise focusing on oncology and immunology, has taken a significant step by resubmitting its Biologics License Application (BLA) for batoclimab (HBM9161) to China’s National Medical Products Administration (NMPA). This submission aims to obtain approval for batoclimab as a treatment for generalized myasthenia gravis (gMG).

The resubmission follows the completion of a Phase III clinical trial, during which an extension period was utilized to gather additional long-term safety data without recruiting new participants. Harbour BioMed voluntarily included this new safety information in the renewed BLA submission to align with regulatory expectations.

Expressing optimism, Dr. Jingsong Wang, Founder, Chairman, and CEO of Harbour BioMed, affirmed the company’s commitment to collaborating closely with the NMPA to expedite the review process. He emphasized the promising results from the Phase III trial, which indicated batoclimab's efficacy in addressing both primary and secondary endpoints. Dr. Wang believes that batoclimab can significantly enhance the treatment landscape for gMG and offer substantial benefits to patients.

Batoclimab has already garnered significant accolades. In 2021, it received the "Breakthrough Therapy Certificate" from the NMPA, following a successful proof-of-concept study for treating Chinese gMG patients. The Phase III clinical trial, completed in March 2023, further reinforced its potential with positive topline results.

In a strategic move, Harbour BioMed partnered with NBP Pharma, a subsidiary of the CSPC Group, in October 2022 to co-develop batoclimab in Greater China. Under this collaboration, Harbour BioMed is responsible for conducting the comprehensive clinical trial process for batoclimab in gMG and will receive tiered royalties based on annual net sales in the region.

Generalized myasthenia gravis (gMG) is an autoimmune disorder characterized by the presence of antibodies such as anti-acetylcholine receptor (AChR) immunoglobulin G (IgG) and anti-muscle-specific tyrosine kinase (Anti-MuSK) IgG. These antibodies disrupt the neuromuscular junction's normal function, leading to weakened skeletal muscle contractions. Symptoms often include ocular manifestations like eyelid ptosis and diplopia, but the disease can progress to affect other muscles, severely impacting patients' quality of life and work capabilities. In severe cases, it can lead to life-threatening myasthenic crises.

Current treatments for MG, including cholinesterase inhibitors and immunosuppressive drugs like glucocorticoids, often fall short in addressing all clinical needs due to limited efficacy and safety. Alternative therapies targeting pathogenic IgG autoantibodies, such as plasmapheresis and intravenous immunoglobulin, show promise but still face challenges related to accessibility, safety, and cost.

Batoclimab (HBM9161) represents a novel therapeutic approach. As a fully human anti-FcRn monoclonal antibody (mAb), it disrupts FcRn-IgG interactions, thereby promoting the degradation of autoantibodies and addressing autoimmune diseases mediated by pathogenic IgG. The Phase III study results underscored batoclimab's ability to rapidly, significantly, and safely alleviate symptoms in gMG patients, enhancing their quality of life.

Harbour BioMed continues to pioneer advancements in biopharmaceuticals, leveraging its proprietary antibody technology platforms, such as Harbour Mice®, which generate fully human monoclonal antibodies. The company’s pipeline is further bolstered by innovative technologies like the HCAb-based immune cell engagers (HBICE®), capable of delivering unparalleled tumor-killing effects. Through these innovations, Harbour BioMed aims to develop next-generation therapeutic antibodies, solidifying its position at the forefront of antibody therapeutics in immunology and oncology.