Nanjing-based Leads Biolabs Co., Ltd., a clinical-stage biotechnology firm established by a team of experienced antibody drug developers trained in the United States, has announced a major advancement in the development of its proprietary bispecific antibody, LBL-024. The United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to LBL-024, which aims to treat neuroendocrine cancer by targeting PD-L1 and 4-1BB pathways. This significant recognition follows the antibody’s acquisition of Breakthrough Therapy Designation by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China.
Dr. Charles Cai, Leads Biolabs’ Chief Medical Officer, commented on the promising clinical data supporting LBL-024, which earned the Breakthrough Therapy Designation in October for combating advanced extrapulmonary neuroendocrine cancer. Cai stated, “Our findings have shown that LBL-024 holds great promise in improving patient outcomes in this challenging therapeutic area. FDA’s grant of ODD further validates the antibody’s potential impact and aids in fast-tracking its commercialization, potentially transforming treatment paradigms for those with few available options.”
Dr. Xiaoqiang Kang, the Founder, Chairman, and CEO of Leads Biolabs, highlighted the global strategic importance of receiving the ODD designation from the FDA. “This designation attracts additional development support and resources, accelerating LBL-024’s market entry and enhancing its prospects as the first-in-class therapeutic antibody focusing on 4-1BB worldwide. It significantly boosts our global market presence and potential for growth,” Kang stated.
The unique tetravalent bispecific antibody LBL-024 provides dual action by inhibiting the PD-1/PD-L1 pathway and selectively stimulating 4-1BB in the tumor microenvironment, thereby reinforcing anti-tumor immune responses. Having received Investigational New Drug (IND) approvals from both the FDA and NMPA in mid-2021, LBL-024 has produced exceptional clinical results. Under the leadership of Professor Shen Lin at Beijing Cancer Hospital, alongside multiple clinical trial centers, LBL-024’s monotherapy has substantially improved both Objective Response Rate (ORR) and Overall Survival (OS) compared to existing treatment options. Entering a landmark single-arm pivotal trial for extrapulmonary neuroendocrine carcinomas in July 2024, LBL-024 stands as a pioneering 4-1BB-targeted drug candidate in the pivotal development stage, according to Frost & Sullivan.
Neuroendocrine carcinoma (NEC), originates within the diffuse neuroendocrine cell system and varies widely based on tumor site and hormone release. The survival rate of NEC is notably low, with a particularly poor prognosis for lifesaving treatments, illustrating the urgent need for innovative therapeutic options.
Orphan drugs address rare disease challenges and, as mandated by the U.S. Orphan Drug Act, provide a framework to encourage development through various incentives. These incentives may include clinical trial tax credits, FDA developmental guidance, application fee exemptions, and exclusive marketing rights for seven years post-approval.