US FDA Grants Orphan Drug Status to Leads Biolabs' LBL-034 for Multiple Myeloma

NANJING, China, Nov. 6, 2024 – On November 1, Nanjing Leads Biolabs Co., Ltd. (Leads Biolabs) announced that their humanized bispecific T-cell engaging antibody, LBL-034, has been granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of multiple myeloma (MM). LBL-034 is a novel antibody that targets both GPRC5D and CD3, developed independently by Leads Biolabs with global intellectual property rights.

LBL-034 stands out as the third GPRC5D-targeted CD3 T-cell engager to reach the clinical stage worldwide, according to Frost & Sullivan. This antibody, designed using Leads Biolabs' proprietary T cell engager technology platform "LeadsBodyTM", works by binding to CD3 on T cells and GPRC5D on cancer cells. This dual binding brings T cells into close proximity with cancer cells, activating the T cells to destroy the cancer cells. LBL-034 has shown superior GPRC5D binding affinity and potency while being less likely to cause T cell exhaustion and death. Extensive preclinical and early clinical research suggests that LBL-034 could potentially be best in class.

In July 2023, Leads Biolabs obtained Investigational New Drug (IND) approvals from both the National Medical Products Administration (NMPA) of China and the FDA. The company then started a phase I/II clinical trial in November 2023 in China, focusing on patients with relapsed/refractory multiple myeloma (RRMM). This first-in-human, open-label, multi-center study includes dose escalation and expansion phases. Sponsored by Leads Biolabs and led by Professor Lu Jin from Peking University People's Hospital, the preliminary data from this study has shown a favorable safety profile and strong efficacy. The preliminary findings are scheduled to be presented at the 66th ASH Annual Meeting in San Diego, USA, on December 9, 2024.

The FDA's approval of ODD for LBL-034 acknowledges the significant potential of this drug in treating MM. This designation will facilitate the global clinical development and marketing of LBL-034. The Orphan Drug Act in the United States encourages development of drugs for rare diseases by offering numerous incentives, such as tax credits for clinical trial expenses, specific FDA guidance at all stages of clinical research, exemption of new drug registration fees, and seven years of market exclusivity upon approval.

Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, expressed his satisfaction with the progress in developing new medications for rare diseases like MM. He emphasized the need for new, effective treatment options for MM, an incurable malignancy that evolves into RRMM, posing severe threats to human life and health. Dr. Cai highlighted the unique molecular design of LBL-034 and its promising antitumor efficacy and safety demonstrated in preliminary clinical results. He assured that Leads Biolabs would expedite the clinical development of LBL-034 to provide MM patients worldwide with safe and effective treatment options as soon as possible.

Dr. Xiaoqiang Kang, founder, chairman, and CEO of Leads Biolabs, remarked that receiving the ODD for LBL-034 marks a significant milestone for the company. He noted that this achievement would further optimize Leads Biolabs' pipeline, expand their exploration in the biopharmaceutical field, and deliver innovative solutions for more unmet medical needs.

Multiple myeloma (MM) is a malignant neoplasm of plasma cells, caused by the abnormal proliferation of clonal plasma cells. It accounts for 1% of neoplastic diseases and about 10% of hematological cancers globally. According to the latest SEER registry data, there were approximately 179,000 MM patients in the US in 2021, fitting the FDA's rare disease criteria. Despite advancements in MM treatment with various drug developments, there remains a need for more effective options. In 2024, it is estimated that approximately 35,780 new MM cases will be diagnosed in the US, comprising about 1.8% of all new cancer cases. Additionally, around 12,540 deaths are projected due to MM-related complications, making up roughly 2.0% of all cancer deaths. Therefore, developing novel therapies to address this medical challenge is imperative.

Nanjing Leads Biolabs Co., Ltd is a clinical-stage biotechnology company focused on creating innovative therapies for underserved medical needs in oncology, autoimmune, and other severe diseases globally. With their proprietary technology platforms, Leads Biolabs has developed a diverse pipeline of 12 innovative drug candidates, six of which are in clinical stages. The company is dedicated to providing safe, effective, and affordable new drugs to address unmet patient needs worldwide.