Nanjing Leads Biolabs Co., Ltd. announced that its novel bispecific antibody, LBL-034, targeting GPRC5D and CD3 for the treatment of multiple myeloma (MM), has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA). This designation aims to accelerate the clinical development and marketing of LBL-034, recognizing its potential to address a critical unmet need in MM treatment.
LBL-034: A Novel Bispecific Antibody
LBL-034 is a humanized bispecific T-cell engaging antibody developed using Leads Biolabs' proprietary LeadsBodyTM technology. It is designed to simultaneously bind to CD3 on T cells and GPRC5D on multiple myeloma cells, bringing T cells into close proximity with cancer cells to facilitate targeted killing. According to Frost & Sullivan, LBL-034 is the third GPRC5D-targeted CD3 T-cell engager to enter clinical development. Preclinical studies suggest that LBL-034 exhibits higher GPRC5D binding affinity and potency, with a reduced propensity for T cell exhaustion and cell death, indicating its potential as a best-in-class therapy.
Clinical Development and Early Data
Leads Biolabs initiated a Phase I/II, first-in-human, open-label, multi-center, dose escalation/expansion study of LBL-034 in patients with relapsed/refractory multiple myeloma (RRMM) in China in November 2023, following IND approvals from both the NMPA and FDA in July 2023. Preliminary data from this study, sponsored by Leads Biolabs and led by Professor Lu Jin from Peking University People's Hospital, has demonstrated a favorable safety profile and robust efficacy. These findings are scheduled for presentation at the 66th ASH Annual Meeting in San Diego, USA, on December 9, 2024.
Implications of Orphan Drug Designation
The ODD granted by the FDA provides several key benefits to support the development of LBL-034, including tax credits for clinical trial expenses, specific guidance from the FDA on clinical research stages, exemption from new drug registration application fees, and seven years of market exclusivity upon approval. These incentives are designed to encourage the development of treatments for rare diseases, such as multiple myeloma, which affects approximately 179,000 patients in the US as of 2021, according to the SEER registry database.
The Unmet Need in Multiple Myeloma
Multiple myeloma, a malignancy of plasma cells, accounts for 1% of neoplastic diseases and about 10% of hematological cancers globally. While advancements in treatment, including proteasome inhibitors, immunomodulatory drugs, CD38-targeting antibodies, bispecific antibodies, and CAR-T cell therapies, have improved outcomes, a significant unmet clinical need remains for more effective therapies, especially for patients with relapsed/refractory disease. In 2024, an estimated 35,780 new MM cases will be diagnosed in the US, with approximately 12,540 deaths, underscoring the urgency for novel treatment options.
Leadership Perspective
Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, emphasized the importance of developing new medications for rare diseases like MM, noting the potential of LBL-034's unique molecular design and promising early clinical results. Dr. Xiaoqiang Kang, founder, chairman, and CEO of Leads Biolabs, highlighted the ODD as a significant milestone for the company and a step towards providing innovative solutions for unmet medical needs.
