• 25 Nov 2024
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• News Article

Leads Biolabs Receives US FDA Orphan Drug Designation for LBL-024

Leads Biolabs receives US FDA orphan drug designation for anti-PD-L1/4-1BB bispecific antibody, LBL-024 for treatment of neuroendocrine cancer

Overview

Nanjing Leads Biolabs Co., Ltd. (Leads Biolabs), a clinical-stage biotechnology company founded in Nanjing by a team of senior US-trained antibody drug developers, announced that LBL-024, an anti-PD-L1/4-1BB bispecific antibody independently developed by the company with global intellectual property rights for the treatment of neuroendocrine cancer, has obtained Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA). This marks another significant milestone following the Breakthrough Therapy Designation granted to LBL-024 by the Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) in China.

Words from the CMO: Leads Biolabs

• Dr Charles Cai, chief medical officer of Leads Biolabs, said “LBL-024 obtained Breakthrough Therapy Designation from CDE in October this year for the treatment of advanced extrapulmonary neuroendocrine cancer. 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. These policy supports for obtaining Orphan Drug qualification will greatly expedite the commercialization of LBL-024, potentially changing the treatment landscape for patients with advanced neuroendocrine cancer who currently have few therapeutic options.”

Words from the CEO: Leads Biolabs

Dr. Xiaoqiang Kang, founder, chairman and CEO of Leads Biolabs, said “The receipt of ODD for LBL-024 from FDA represents a pivotal milestone in our global strategy. This designation 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.”

About the Antibody: LBL-024

• LBL-024 is a tetravalent bispecific antibody that simultaneously targets PD-L1 and 4-1BB, serving dual functions: blocking the immunosuppressive PD-1/PD-L1 pathway, and selectively co-stimulating 4-1BB in the tumour microenvironment to enhance immune responses. 
• The dual functions of LBL-024—lifting PD-1/PD-L1 immune inhibition and intensifying 4-1BB modulated T cell activation—synergistically enhance the anti-tumour immune response.
• LBL-024 approvals
• LBL-024 received IND approvals from both FDA and NMPA on July 30, 2021 and September 9, 2021 respectively to conduct phase clinical research, and has achieved outstanding results. 
• Sponsored by Leads Biolabs, led by Professor Shen Lin from Beijing Cancer Hospital with participation of multiple clinical trial centres, the current clinical data demonstrate 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 has entered into a single-arm pivotal trial for extrapulmonary neuroendocrine carcinomas in July 2024 and stands as the globally first 4-1BB-targetd drug candidates to have reached pivotal stage, according to Frost & Sullivan.

The Neuroendocrine Carcinoma

• Neuroendocrine carcinoma (NEC) is a class of poorly differentiated, high-grade neuroendocrine neoplasms (NEN), which originate in the diffuse neuroendocrine cell system and may occur in many different sites. 
• Its molecular characteristics are significantly different from those of neuroendocrine tumours (NET). 
• NEC can be divided into pulmonary NEC and extrapulmonary NEC, among which pulmonary NEC includes small cell lung cancer (SCLC) and pulmonary large cell neuroendocrine carcinoma (p-LCNEC). 
• According to previous data collected, the number of new cases of SCLC is about 98,193, p-LCNEC is about 19,639, and extrapulmonary NECs is about 9,820 in the United States every year. 
• As a result, there are an estimated 127,652 new cases of NECs in the United States each year, meeting the FDA's definition of a rare disease.

Symptoms of Neuroendocrine Carcinoma

• Symptoms of NEC can vary depending on the type of tumour, its location in the body and the hormone released. 
• The survival rate of NEC varies according to the type of cancer and whether it spreads. 
• Specifically, the 5-year survival rate of pulmonary NECs is 5.6%, digestive tract NECs is 13.1%, and other primary NECs are 26.0%. 
• Due to the limited effective treatment options available for patients, the overall survival rate of NEC is low, and more effective new treatment options are urgently needed.

All About the Orphan Drugs

• Orphan drugs are medications used for the prevention, treatment, and diagnosis of rare diseases. According to the Orphan Drug Act of the United States, Orphan Drug Designation is established to encourage the development of drugs for treating rare diseases. 
• It provides a series of incentives for new drug development, including but not limited to: 

(1) tax credits for clinical trial expenses; 
(2) specific guidance from FDA on all stages of clinical research; 
(3) exemption of the application fee for new drug registration; and 
(4) 7 years of market exclusivity after listing.