Bispecific Antibody Receives FDA Orphan Drug Designation for the Treatment of Multiple Myeloma

The FDA granted orphan drug designation to LBL-034 (Leads Biolabs), a humanized bispecific T-cell engaging antibody which targets GPRC5D and CD3 for the treatment of multiple myeloma (MM).¹

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"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 tumor relapses will become shorter and shorter, and eventually it will evolve into [relapsed/refractory] MM, which seriously threatens human life and health. It is urgent to develop new and more effective treatment options,” Charles Cai, MD, PhD, chief medical officer of Leads Biolabs, said in a news release.¹

MM met the FDA’s standards for defining a rare disease as 179,000 US individuals were diagnosed with the disease in 2021. MM is a malignant neoplasm of plasma cells that occurs due to abnormal production of clonal plasma cells.¹ Symptoms of MM include bone pain, nausea, constipation, loss of appetite, mental fogginess, tiredness, infections, weight loss, weakness, thirst, and frequent urination. Study authors noted that causes of MM remain unclear, and there is no way to prevent the disease.²

Despite recent advancements in treatment for MM using proteasome inhibitors, immunomodulatory drugs, selective nuclear export inhibitors, CD38-targeting antibodies, bispecific antibodies, and CAR-T cell therapies, study authors noted that there is still a need for further effective novel therapies. According to data from Surveillance, Epidemiology, and End Results Program, an estimated 35,780 individuals will be newly diagnosed with MM, with about 12,540 deaths connected to MM-related complications in 2024.¹

The study authors noted that LBL-034 was created using the company’s proprietary T cell engager antibody technology platform "LeadsBodyTM," which is the third GPRC5D-targeted CD3 T-cell engager to reach clinical stage. The role of LBL-034 is to bring T cells into range with cancer cells to effectively activate the T cells to attack and kill the targeted cancer cells. Study results displayed that LBL-034 exhibited greater GPRC5D binding affinity and potency with a smaller chance of inducing T cell exhaustion and cell death.¹

“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," said Cai in a news release.¹

The novel bispecific antibody previously received FDA Investigational new drug approvals in July of 2023. Additionally, researchers began a phase 1/2 first in human, open-label, multi-center, dose escalation/expansion study of LBL-034 among individuals with relapsed/refractory multiple myeloma in November of 2023. The study authors noted that preliminary data from the phase 1/2 clinical trials displayed favorable safety and significant efficacy.¹

REFERENCES

1. Leads Biolabs Receives Orphan Drug Designation from the US FDA for LBL-034, a Uniquely Designed, Highly Differentiated Anti-GPRC5D/CD3 Bispecific Antibody, for the Treatment of Multiple Myeloma. Leads Biolabs. News release. November 6, 2024. Accessed November 11, 2024. https://prnmedia.prnewswire.com/news-releases/leads-biolabs-receives-orphan-drug-designation-from-the-us-fda-for-lbl-034-a-uniquely-designed-highly-differentiated-anti-gprc5dcd3-bispecific-antibody-for-the-treatment-of-multiple-myeloma-302297296.html

2. Multiple myeloma. Mayo Clinic. July 30, 2024. Accessed November 11, 2024. https://www.mayoclinic.org/diseases-conditions/multiple-myeloma/symptoms-causes/syc-20353378.