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Leads Biolabs' LBL-024 Receives FDA Orphan Drug Designation for Neuroendocrine Cancer

8 months ago3 min read

Key Insights

  • LBL-024, a bispecific antibody targeting PD-L1 and 4-1BB developed by Leads Biolabs, has been granted Orphan Drug Designation by the FDA for neuroendocrine cancer.

  • This designation follows a Breakthrough Therapy Designation from China's NMPA, highlighting LBL-024's potential to improve outcomes for patients with advanced extrapulmonary neuroendocrine cancer.

  • Clinical data suggest LBL-024 monotherapy has more than doubled both the Objective Response Rate (ORR) and Overall Survival (OS) compared to existing treatments for this disease.

Nanjing Leads Biolabs Co., Ltd. has announced that its investigational drug, LBL-024, a bispecific antibody targeting both PD-L1 and 4-1BB, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of neuroendocrine cancer. This regulatory milestone follows the Breakthrough Therapy Designation granted to LBL-024 by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) earlier this year.

Clinical Promise of LBL-024

LBL-024 is a tetravalent bispecific antibody designed to simultaneously block the PD-1/PD-L1 immunosuppressive pathway and selectively co-stimulate 4-1BB in the tumor microenvironment. This dual-action mechanism aims to enhance anti-tumor immune responses by lifting PD-1/PD-L1 immune inhibition and intensifying 4-1BB-modulated T cell activation.
Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, stated, "Our clinical data to date has been very encouraging, suggesting that LBL-024 could meaningfully improve outcomes of patients living with this devastating disease. The grant of ODD from FDA further underscores the exceptional potential of LBL-024 to address a critical gap in this therapeutic area."

Clinical Trial Data

LBL-024 received IND approvals from both the FDA and NMPA in 2021 to conduct phase I/II clinical research. Current clinical data, from studies sponsored by Leads Biolabs and led by Professor Shen Lin from Beijing Cancer Hospital, indicate that LBL-024 monotherapy has more than doubled both the Objective Response Rate (ORR) and Overall Survival (OS) compared to existing treatments for advanced extrapulmonary neuroendocrine carcinomas. Based on these results, LBL-024 has entered a single-arm pivotal trial for extrapulmonary neuroendocrine carcinomas in July 2024.

Neuroendocrine Cancer: An Unmet Need

Neuroendocrine carcinoma (NEC) is a high-grade neuroendocrine neoplasm that originates in the diffuse neuroendocrine cell system. NECs are divided into pulmonary and extrapulmonary types, with an estimated 127,652 new cases occurring annually in the United States. The overall survival rate for NEC is low due to limited effective treatment options; the 5-year survival rate of pulmonary NECs is 5.6%, digestive tract NECs is 13.1%, and other primary NECs are 26.0%.

Benefits of Orphan Drug Designation

The FDA's Orphan Drug Designation provides several incentives to encourage the development of drugs for rare diseases, including tax credits for clinical trial expenses, specific guidance from the FDA on clinical research, exemption of application fees for new drug registration, and seven years of market exclusivity upon approval.
Dr. Xiaoqiang Kang, founder, chairman and CEO of Leads Biolabs, noted that the ODD "not only enables LBL-024 to receive additional policy support and resource allocation during its development, accelerating its path to market and positioning it as a potential first-in-class therapeutic antibody targeting 4-1BB worldwide, but also provides us with greater market opportunities and avenues for growth on a global scale."
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