Tiziana Life Sciences Seeks Orphan Drug Status for Intranasal Foralumab

On May 13, 2024, Tiziana Life Sciences, Ltd. (Nasdaq: TLSA), a biotechnology firm focused on innovative immunomodulation therapies through unique drug delivery methods, announced a significant step forward. The company has submitted a request to the FDA to obtain Orphan Drug Designation for intranasal foralumab aimed at treating non-active secondary progressive Multiple Sclerosis (na-SPMS). If approved, foralumab would be the first therapy for na-SPMS to receive this designation. The application is supported by extensive clinical and non-clinical data demonstrating the drug's effectiveness in treating na-SPMS, with prevalence estimates partly derived from a longitudinal study at Brigham & Women’s Hospital in Boston, Massachusetts.

Foralumab is a fully human anti-CD3 monoclonal antibody that, when administered intranasally, has been shown to activate T regulatory cells. Currently, 10 patients with na-SPMS have been treated under an open-label intermediate-size Expanded Access Program (EA), with an additional 20 patients recently authorized by the FDA to join. Results from this program are promising, with all patients either showing improvement or stabilization. Notably, 70% of the patients reported a reduction in fatigue, a major symptom of MS, after six months of treatment as measured by the Modified Fatigue Impact Scale (MFIS). Importantly, none of the patients experienced declines in key clinical measures. Besides the Expanded Access Program, intranasal foralumab is undergoing a Phase 2a trial, which is a randomized, double-blind, placebo-controlled, multicenter, dose-ranging study (NCT06292923) with data expected in 2025.

Gabriele Cerrone, Chairman, acting CEO, and founder of Tiziana Life Sciences, emphasized the importance of obtaining Orphan Drug Designation. He noted that this designation is granted by the FDA for drugs intended to treat rare diseases affecting fewer than 200,000 people in the U.S. It provides benefits such as up to seven years of market exclusivity if the drug is approved, along with other financial incentives to support its development, which would be highly valuable to the company and its shareholders.

Once submitted, the FDA's Orphan Drug Designation program will review the application to ensure all criteria are met, a process that takes approximately 90 days.

Activated T cells are crucial in the inflammatory process, and foralumab, as a fully human anti-CD3 monoclonal antibody, targets the T cell receptor to reduce inflammation by modulating T cell function. This suppression of inflammatory responses has been observed in patients with COVID, multiple sclerosis, and even in healthy individuals. The Phase 2 trial of intranasal foralumab for non-active SPMS began patient screening in November 2023. Utilizing nasal anti-CD3 mAb for immunomodulation represents a groundbreaking approach for treating neuroinflammatory and neurodegenerative diseases.

Tiziana Life Sciences is committed to advancing clinical-stage biopharmaceutical development through innovative drug delivery methods. The company's intranasal delivery system for foralumab presents a potential improvement in both efficacy and safety when compared to traditional intravenous methods. To date, foralumab has shown a favorable safety profile and significant clinical responses in various studies. Tiziana has patented its technology for alternative immunotherapy delivery routes and anticipates broad applications for its pipeline.

In summary, Tiziana Life Sciences' request for Orphan Drug Designation for intranasal foralumab marks a critical milestone in their efforts to develop transformative treatments for rare diseases like na-SPMS. The company's novel approach to drug delivery holds promise for improving patient outcomes and advancing the field of immunotherapy.