GBR 1342, an investigational bispecific antibody developed by Glenmark Pharmaceuticals, was granted orphan drug designation by the U.S Food and Drug Administration (FDA) for the treatment of multiple myeloma patients who have received prior therapies.
The FDA’s Office of Orphan Products Development (OOPD) gives this designation to novel therapies intended to improve the treatment and management of rare diseases or disorders that affect fewer than 200,000 people in America. Manufacturers of medicines with orphan drug designation qualify for various incentives, including seven years of market exclusivity once the treatment is approved.
Glenmark created a spinoff company that will focus on the clinical development of GBR 1342. The name of the new company is not yet known.
“Patients who are struggling with diseases like multiple myeloma are seeking new treatment options and innovative approaches. We believe the focus of our new company is essential to these patients and to the healthcare system,” Alessandro Riva, MD, CEO of the new company, said in a press release.
“As one of our first important pipeline milestones, we are excited that the FDA has recognized the potential for GBR 1342 to offer a significant advancement for patients with multiple myeloma,” he said.
GBR 1342 is manufactured using Glenmark’s BEAT platform, which stands for bispecific engagement by antibodies based on the T-cell receptor. The BEAT technology develops therapeutic agents that mimic natural antibodies, but that can specifically bind two targets at the same time.
GBR 1342 binds to a molecule called CD3, which is located at the surface of the immune T cells, and CD38, a protein found at the surface of blood cancer cells, such as multiple myeloma. By interacting with both molecules at the same time, GBR 1342 activates T cells and brings them closer to myeloma cells, triggering an immune response against the tumor cells.
In preclinical studies, BEAT antibodies showed more localized response and less toxicity than other treatments based on antibodies, either mono or bispecific, the company says on its website.
An ongoing, open-label Phase 1 trial (NCT03309111), which is still recruiting patients at several locations in the U.S, is testing the safety, efficacy, and maximum tolerated dose of GBR 1342 in people with multiple myeloma who have received previous therapies.
The study expects to enroll 125 participants, and will also assess markers of disease activity such as the immune response against the tumor and the proportion of patients achieving stable disease or better.