FDA Grants Orphan Drug Status to BioInvent's BI-1808 for T-cell Lymphoma

Lund, Sweden – March 20, 2025 – BioInvent International AB, a leading biotechnology company specializing in the discovery and development of innovative immune-modulatory antibodies for cancer immunotherapy, has made a significant advancement. The U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to BI-1808, an anti-TNFR2 antibody designed to treat T-cell Lymphoma (TCL), a rare and aggressive form of cancer. This acknowledgment by the FDA marks a pivotal step in addressing the pressing need for new treatment options for TCL, particularly cutaneous T-cell lymphoma (CTCL), a subtype originating from T-lymphocytes in the skin.

CTCL is characterized by persistent skin lesions, itching, and possible systemic complications, which can severely affect patients' quality of life. With roughly 3,000 new cases diagnosed annually in the United States, effective treatment options are limited, making this FDA designation a promising development for patients.

Martin Welschof, CEO of BioInvent, expressed enthusiasm about the FDA’s decision, emphasizing that this designation, along with recent positive Phase 2a data in CTCL, highlights the company's progress in developing BI-1808 as a novel class of immunomodulatory agents for TCL. He further stated BioInvent's eagerness to collaborate with regulatory bodies to expedite the development process and bring this innovative treatment to those in need.

Orphan Drug Designation by the FDA is granted to drugs intended to treat rare conditions affecting fewer than 200,000 people in the U.S. The designation offers several benefits to drug developers, such as support in the drug development process, tax credits for clinical costs, exemption from certain FDA fees, and seven years of marketing exclusivity. These incentives are designed to encourage the development of drugs for small patient populations with unmet medical needs.

BI-1808 is part of BioInvent’s tumor-associated regulatory T cells (Treg)-targeting program. It targets TNFR2, a receptor upregulated on Tregs within the tumor environment, crucial for tumor growth and survival. This makes TNFR2 a promising target for cancer immunotherapy. BI-1808, a first-in-class drug candidate, is under clinical development for both solid and blood cancers. The drug has demonstrated notable activity and tolerability as a standalone treatment in ongoing Phase 2a studies, with promising results in combination with pembrolizumab, an anti-PD-1 therapy, in Phase 1/2a trials.

The ongoing Phase 1/2a study evaluates the safety, tolerability, and potential efficacy of BI-1808 as a monotherapy and in conjunction with pembrolizumab in patients with advanced solid tumors and T-cell lymphoma. The study's first phase has completed dose escalation for the combination therapy, and the Phase 2a dose expansion is in progress. This expansion includes cohorts with ovarian cancer, various tumor types, and T-cell lymphomas, including CTCL.

Preliminary results from the CTCL cohort show that out of four evaluable patients, three demonstrated a partial response and one maintained stable disease, despite having previously deteriorated with standard treatments. These findings are consistent with earlier single-agent data presented at the American Society of Clinical Oncology conference in June 2024, which included one complete response, one partial response, and nine cases of stable disease. Additional data from the Phase 2a study of BI-1808 as a monotherapy are anticipated by mid-2025.

BioInvent International AB is a clinical-stage biotech firm dedicated to discovering and developing pioneering immune-modulatory antibodies for cancer treatment. The company is advancing various drug candidates through its clinical programs targeting hematological cancers and solid tumors. BioInvent’s proprietary F.I.R.S.T™ technology platform plays a crucial role in identifying both targets and corresponding antibodies, driving the company’s clinical development pipeline and creating opportunities for research collaborations and partnerships with leading pharmaceutical companies.