BioInvent International AB announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to BI-1808, the company's first-in-class anti-TNFR2 antibody, for the treatment of T-cell Lymphoma (TCL).
The designation comes as a significant milestone for the Sweden-based biotech company, which focuses on developing novel immune-modulatory antibodies for cancer immunotherapy. This regulatory recognition provides BioInvent with several benefits designed to accelerate the development pathway for BI-1808, including development assistance, tax credits for qualified clinical costs, exemptions from certain FDA fees, and seven years of marketing exclusivity upon approval.
"We are excited to receive the FDA's Orphan Drug Designation for the treatment of TCL. This designation along with the recently announced positive Phase 2a data in CTCL reinforces our continued progress and commitment to developing BI-1808 as a potential novel class of immunomodulatory agents for TCL," said Martin Welschof, Chief Executive Officer of BioInvent. "We look forward to working closely with the regulatory agencies to accelerate the development of BI-1808 and bring this innovative treatment to patients who are in great need of new treatment options."
Understanding T-cell Lymphoma and the Unmet Need
T-cell lymphomas encompass several subtypes of T cell-derived non-Hodgkin's lymphoma, including cutaneous T-cell lymphoma (CTCL). CTCL is particularly challenging as it originates in T-lymphocytes residing in the skin, typically manifesting with persistent skin lesions, intense itching, and potential systemic complications that significantly impact patients' quality of life.
The disease affects approximately 3,000 new patients annually in the United States, with limited effective treatment options currently available. This represents a significant unmet medical need that BioInvent aims to address with BI-1808.
Promising Clinical Results
The Orphan Drug Designation follows encouraging clinical data from BioInvent's ongoing Phase 2a study. Recent results from the single-agent CTCL cohort demonstrated three patients with partial response (PR) and one with stable disease (SD) out of four evaluable patients. Notably, all these patients had previously deteriorated after standard treatment.
These new findings build upon earlier data presented at the American Society of Clinical Oncology conference (ASCO) in June 2024, which showed one complete response (CR), one partial response (PR), and nine patients with stable disease (SD).
The company is currently conducting a Phase 1/2a clinical trial (NCT04752826) evaluating BI-1808 both as a monotherapy and in combination with pembrolizumab, an anti-PD-1 therapy. The trial is investigating the safety, tolerability, and efficacy of BI-1808 in patients with advanced solid tumors and T-cell lymphoma.
Novel Mechanism of Action
BI-1808 represents a novel approach to cancer immunotherapy through its targeting of TNFR2 (Tumor Necrosis Factor Receptor 2). This receptor is particularly upregulated on regulatory T cells (Tregs) within the tumor microenvironment and has been shown to play a crucial role in tumor expansion and survival.
As part of BioInvent's tumor-associated regulatory T cells (Treg)-targeting program, BI-1808 aims to disrupt this mechanism, potentially offering a new therapeutic strategy for patients with T-cell lymphoma and other malignancies.
Dr. Björn Frendéus, Chief Scientific Officer at BioInvent, explained in a previous statement: "TNFR2 represents an exciting new target in cancer immunotherapy. By targeting this receptor, we aim to deplete immunosuppressive Tregs in the tumor microenvironment while simultaneously activating effector T cells, potentially creating a powerful dual mechanism of action."
Development Timeline and Future Directions
BioInvent continues to advance the clinical development of BI-1808, with additional data from the Phase 2a study of single-agent BI-1808 expected by mid-2025. The company is also exploring the potential of BI-1808 in other indications, with expansion cohorts including ovarian cancer and various tumor types.
The Phase 2a dose expansion study for the combination of BI-1808 with pembrolizumab is ongoing, with plans to include patients with ovarian cancer, multiple tumor types, and T-cell lymphoma (including CTCL).
Company Profile and Technology Platform
BioInvent International AB, listed on Nasdaq Stockholm (BINV), is a clinical-stage biotech company with a robust pipeline of immuno-modulatory antibodies. The company currently has five drug candidates in six ongoing clinical programs in Phase 1/2 trials for the treatment of hematological cancers and solid tumors.
The company's proprietary F.I.R.S.T™ technology platform enables the identification of both targets and the antibodies that bind to them, generating promising new immune-modulatory candidates for the company's clinical development pipeline and creating licensing and partnering opportunities.
BioInvent generates revenue through research collaborations and license agreements with pharmaceutical companies, as well as from producing antibodies for third parties in its integrated manufacturing unit.
The Orphan Drug Designation for BI-1808 represents an important validation of BioInvent's approach to developing novel cancer immunotherapies and strengthens the company's position in the competitive oncology landscape.
