FDA Grants Orphan Drug Status to Leads Biolabs’ LBL-024 for Neuroendocrine Cancer

NANJING, China, Nov. 22, 2024 /PRNewswire/ -- Nanjing Leads Biolabs Co., Ltd. (Leads Biolabs) has announced that their anti-PD-L1/4-1BB bispecific antibody, LBL-024, developed to treat neuroendocrine cancer, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). This follows the Breakthrough Therapy Designation granted to LBL-024 by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China.

Dr. Charles Cai, Chief Medical Officer at Leads Biolabs, mentioned that LBL-024 was awarded Breakthrough Therapy Designation from the CDE in October for treating advanced extrapulmonary neuroendocrine cancer. He emphasized that the clinical data so far has been promising, indicating that LBL-024 could significantly improve outcomes for patients suffering from this severe disease. The ODD from the FDA highlights the considerable potential of LBL-024 to address an important need in this therapeutic area. The policy supports linked to obtaining Orphan Drug status will speed up the commercialization of LBL-024, potentially altering the treatment landscape for patients with advanced neuroendocrine cancer who have limited therapeutic options at present.

Dr. Xiaoqiang Kang, founder, chairman, and CEO of Leads Biolabs, stated that receiving ODD for LBL-024 from the FDA is a critical milestone in their global strategy. This designation not only allows LBL-024 to benefit from additional policy support and resource allocation during its development, accelerating its market path and positioning it as a potential first-in-class therapeutic antibody targeting 4-1BB worldwide, but also offers greater market opportunities and growth avenues on a global scale.

LBL-024 is a tetravalent bispecific antibody that targets PD-L1 and 4-1BB, performing dual functions: inhibiting the immunosuppressive PD-1/PD-L1 pathway and selectively co-stimulating 4-1BB in the tumor microenvironment to enhance immune responses. The dual functions of LBL-024, which involve lifting PD-1/PD-L1 immune inhibition and enhancing 4-1BB modulated T cell activation, work together to boost the anti-tumor immune response.

LBL-024 received IND approvals from both the FDA and NMPA on July 30, 2021, and September 9, 2021, respectively, to conduct phase Ⅰ/Ⅱ clinical research, and has achieved excellent results. Sponsored by Leads Biolabs and led by Professor Shen Lin from Beijing Cancer Hospital with the participation of various clinical trial centers, current clinical data show that LBL-024 monotherapy has more than doubled both the Objective Response Rate (ORR) and Overall Survival (OS) compared to existing treatments for this disease. Based on the current treatment status and the available safety and efficacy data, LBL-024 entered a single-arm pivotal trial for extrapulmonary neuroendocrine carcinomas in July 2024 and stands as the first 4-1BB-targeted drug candidate globally to have reached this pivotal stage, according to Frost & Sullivan.

Neuroendocrine carcinoma (NEC) is a type of poorly differentiated, high-grade neuroendocrine neoplasm originating in the diffuse neuroendocrine cell system and can occur in various body sites. Its molecular characteristics are markedly different from those of neuroendocrine tumors (NET). NEC can be categorized into pulmonary NEC and extrapulmonary NEC, with pulmonary NEC including small cell lung cancer (SCLC) and pulmonary large cell neuroendocrine carcinoma (p-LCNEC). According to previous data, there are approximately 127,652 new cases of NECs in the United States each year, fitting the FDA's definition of a rare disease.

Symptoms of NEC can vary depending on the tumor type, its location, and the hormone released. The survival rate of NEC differs according to the cancer type and its spread. Specifically, the 5-year survival rate for pulmonary NECs is 5.6%, digestive tract NECs is 13.1%, and other primary NECs is 26.0%. Given the limited effective treatment options available, the overall survival rate for NEC is low, emphasizing the urgent need for more effective new treatment options.

Orphan drugs are used for preventing, treating, and diagnosing rare diseases. The Orphan Drug Act in the United States was established to encourage the development of drugs for rare diseases. It offers several incentives for new drug development, including tax credits for clinical trial expenses, specific guidance from the FDA on all stages of clinical research, exemption of the application fee for new drug registration, and seven years of market exclusivity after approval.

Nanjing Leads Biolabs Co., Ltd. is a clinical-stage biotechnology company focused on discovering, developing, and commercializing innovative therapies for unmet medical needs in oncology, autoimmune, and other severe diseases both in China and globally. Backed by proprietary technology platforms and strong drug development capabilities, Leads Biolabs has created a differentiated pipeline of 12 innovative drug candidates, with six having successfully progressed to the clinical stage. The company is committed to providing safe, effective, accessible, and affordable new drugs to meet patients' unmet needs worldwide.