US FDA Grants Orphan Drug Designation to Leads Biolabs’ LBL-034

US FDA grants orphan drug designation to Leads Biolabs’ LBL-034, a humanized bispecific T-cell engaging antibody targeting both GPRC5D & CD3 to treat multiple myeloma

Overview

Nanjing Leads Biolabs Co., Ltd. (Leads Biolabs), a clinical- stage biotechnology company, announced that LBL-034, a humanized bispecific T-cell engaging antibody targeting both GPRC5D and CD3 which is independently developed by Leads Biolabs with global intellectual property rights for treatment of multiple myeloma (MM), has been granted the Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA).

About the Molecule: LBL-034

• LBL-034, a novel bispecific antibody designed using Leads Biolabs' proprietary T cell engager antibody technology platform “LeadsBody”, is the world's third GPRC5D-targeted CD3 T-cell engager to enter clinical stage, according to Frost & Sullivan. 
• By simultaneously binding to CD3 on T cells and the tumour-associated antigen GPRC5D on cancer cells, LBL-034 brings T cells into close proximity with cancer cells, effectively activating the T cells to attack and kill the targeted cancer cells. 
• LBL-034 has exhibited higher GPRC5D binding affinity and potency while being less prone to inducing T cell exhaustion and cell death. 
• Extensive preclinical and early clinical research strongly indicates that LBL-034 has the potential to be best in class.

IND Approvals

• We received IND approvals from the NMPA and FDA in July 2023 and commenced a phase I/II first in human, open-label, multi-centre, dose escalation/expansion study of LBL-034 in patients with relapsed/refractory multiple myeloma (RRMM), in November 2023 in China. 
• Sponsored by Leads Biolabs, led by Professor Lu Jin from Peking University People's Hospital with participation of multiple clinical trial centres, the preliminary data of this study demonstrated favourable safety profile and robust efficacy. 
• The preliminary data will be presented at the 66th ASH Annual Meeting in San Diego, USA in December 9, 2024.

The ODD Apprvoals

• This ODD approval is a recognition by the FDA regarding the significant potential of Leads Biolabs' LBL-034 in the treatment of MM. 
• It will help accelerate the clinical development and marketing process of LBL-034 globally. 
• According to the Orphan Drug Act of the United States, ODD is established by FDA 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.

Words from the Leads Biolabs

• Dr. Charles Cai, chief medical officer of Leads Biolabs, said "" I am pleased to see the positive progress in our development of the new medications to benefit patients with rare diseases like MM. MM remains an incurable malignancy and with the increasing lines of treatment, the interval between the tumour relapses will become shorter and shorter, and eventually it will evolve into RRMM, which seriously threatens human life and health.
• It is urgent to develop new and more effective treatment options. LBL-034 adopts a unique molecular design, and the preliminary clinical results indicate its promising antitumor efficacy and safety. We will expedite the clinical development of LBL-034 and strive to bring safe and effective treatment options to MM patients around the world as early as possible.

Words from the CEO: Leads Biolabs

Dr. Xiaoqiang Kang, founder, chairman and CEO of Leads Biolabs, said "" LBL-034 is the first product from Lead Biolabs to receive Orphan Drug Designation from FDA, marking a successful step for us on this challenging but meaningful journey. Taking this opportunity, we will further optimize our pipeline layout, broaden our exploration boundaries in the biopharmaceutical field, and provide innovative solutions for more unmet medical needs.""

About the Diseases: Multiple Myeloma

• Multiple myeloma (MM) is a malignant neoplasm of plasma cells that is caused by abnormal proliferation of clonal plasma cells, accounting for 1% of neoplastic diseases and approximately 10% of haematological cancers globally. 
• The latest data from the SEER registry database showed that, in 2021, there were approximately 179,000 MM patients in the US, meeting the FDA's criteria for defining a rare disease. 
• In recent years, significantly advancement have been made in the treatment of MM through successful development of proteasome inhibitors, immunomodulatory drugs, selective nuclear export inhibitors, CD38-targeting antibodies, bispecific antibodies, and CAR-T cell therapies. 
• However, despite these achievements, there remains an unmet clinical need for more effective treatment options. 
• According to SEER data, in 2024, it is estimated that there will be around 35,780 newly diagnosed MM patients in the US, accounting for approximately 1.8% of all new cancer cases reported nationwide. 
• Additionally, it is projected that about 12,540 deaths will occur due to MM-related complications during this period—equating to roughly 2.0% of all cancer deaths recorded nationally. 
• Therefore, urgent attention needs to be given towards developing novel therapies to address this pressing medical challenge.

Nanjing Leads Biolabs Co, Ltd is a clinical- stage biotechnology company founded in Nanjing by a team of senior US-trained antibody drug developers.

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