Tiziana Life Sciences Receives FDA Approval for Phase 2a Trial of Foralumab in Multiple System Atrophy

Tiziana Life Sciences announces that the FDA has approved the IND for its Phase 2a clinical trial of intranasal foralumab in patients with Multiple System Atrophy, an orphan designated disease with no FDA-approved therapies. The trial aims to treat this life-limiting disease, which is in need of effective treatments.

Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) has announced that the U.S. Food & Drug Administration (FDA) has approved an Investigational New Drug (IND) application for its Phase 2a clinical trial of intranasal foralumab in patients with Multiple System Atrophy (MSA). This approval marks a significant milestone for the biotechnology company as it seeks to address the unmet medical need for effective treatments in MSA, a life-limiting neurodegenerative disease with no FDA-approved therapies.

The Phase 2a clinical trial, identified as NCT06868628, is a six-month, open-label study designed to evaluate the effects of intranasal foralumab on microglial activation, clinical outcomes, and safety in MSA patients. Foralumab, a fully human anti-CD3 monoclonal antibody, targets T-cell mediated neuroinflammation and is administered via nasal spray in eight 3-week dosing cycles.

Multiple System Atrophy (MSA) is an orphan disease, with a mean incidence in the US of 0.6:100,000 person-years, increasing with ≥50 years of age to 3:100,000 person-years. Prevalence estimates for MSA range from 1.9-4.9 per 100,000 worldwide, suggesting that environmental, genetic, and epigenetic influences contribute to disease pathogenesis. MSA is a rapidly progressive neurodegenerative disorder affecting autonomic functions (such as blood pressure, bladder control) and motor control, leading to severe disability and shortened life expectancy. Increasing evidence implicates neuroinflammation and microglial activation as key drivers in MSA pathogenesis, contributing to neuronal degeneration. Disease progression is assessed using the unified MSA rating scale (UMSARS), which rates activities of daily life, autonomic and motor impairment, as well as overall disability. Disease prognosis is poor with a median survival of 6-9 years.

"We are pleased that the FDA has approved the IND to treat MSA patients with nasal foralumab," said Vikram Khurana, MD, PhD, Tracy T. Batchelor Endowed Chair in Neurology, and Division Chief of Movement Disorders and Director of the MSA Center of Excellence at Brigham and Women’s Hospital and Principal Investigator of the MSA trial. "Neuroinflammation plays a significant role in MSA’s rapid progression. Intranasal foralumab offers a novel therapeutic pathway by targeting T-cell mediated immune activation in the brain. We aim to observe whether this therapy can meaningfully impact microglial activity and important clinical outcomes over six months in patients with MSA."

Ivor Elrifi, Chief Executive Officer of Tiziana Life Sciences, commented on the rationale of foralumab for the treatment of MSA: "Foralumab administered intranasally induces regulatory T cells and modulates T-cell-driven inflammation. Its ability to reduce microglial activation presents a promising therapeutic strategy to slow neuronal damage in neuroinflammatory and degenerative diseases such as Multiple Sclerosis, Alzheimer’s Disease, ALS, and MSA. This novel, non-systemic approach is designed to engage regulatory T cells, promoting immune tolerance while minimizing systemic immune suppression."

Foralumab, a fully human anti-CD3 monoclonal antibody, is a biological drug candidate that has been shown to stimulate T regulatory cells when dosed intranasally. At present, 10 patients with Non-Active Secondary Progressive Multiple Sclerosis (na-SPMS) have been dosed in an open-label intermediate sized Expanded Access (EA) Program (NCT06802328) with either an improvement or stability of disease seen within 6 months in all patients. In addition, intranasal foralumab is currently being studied in a Phase 2a, randomized, double-blind, placebo-controlled, multicenter, dose-ranging trial in patients with non-active secondary progressive multiple sclerosis (NCT06292923).

Tiziana Life Sciences is a clinical-stage biopharmaceutical company developing breakthrough therapies using transformational drug delivery technologies to enable alternative routes of immunotherapy. Tiziana’s innovative nasal approach has the potential to provide an improvement in efficacy as well as safety and tolerability compared to intravenous (IV) delivery. Tiziana’s lead candidate, intranasal foralumab, which is the only fully human anti-CD3 mAb currently in clinical development, has demonstrated a favorable safety profile and clinical response in patients in studies to date.

For more information about Tiziana Life Sciences and its innovative pipeline of therapies, please visit www.tizianalifesciences.com.

References:
[1] https://www.quiverquant.com/news/Tiziana+Life+Sciences+Announces+FDA+Approval+of+IND+for+Phase+2a+Clinical+Trial+of+Intranasal+Foralumab+in+Multiple+System+Atrophy+Patients
[2] https://www.pnas.org/doi/10.1073/pnas.2220272120
[3] https://www.pnas.org/doi/10.1073/pnas.2309221120