Tiziana Life Sciences gets FDA nod for foralumab trial in Multiple System Atrophy

Tiziana Life Sciences has received FDA approval for a Phase 2a clinical trial of intranasal foralumab in patients with Multiple System Atrophy (MSA). The trial aims to evaluate the effects of foralumab on microglial activation, clinical outcomes, and safety in MSA patients. Foralumab is a fully human anti-CD3 monoclonal antibody targeting T-cell mediated neuroinflammation. MSA is a rare, rapidly progressive neurodegenerative disorder with no FDA-approved therapies, and the trial will assess disease progression using the unified MSA rating scale (UMSARS). Shares of Tiziana climbed nearly 13% at Monday's open.

Tiziana Life Sciences has received FDA approval for a Phase 2a clinical trial of intranasal foralumab in patients with Multiple System Atrophy (MSA). The trial aims to evaluate the effects of foralumab on microglial activation, clinical outcomes, and safety in MSA patients. Foralumab is a fully human anti-CD3 monoclonal antibody targeting T-cell mediated neuroinflammation. MSA is a rare, rapidly progressive neurodegenerative disorder with no FDA-approved therapies, and the trial will assess disease progression using the unified MSA rating scale (UMSARS). Shares of Tiziana climbed nearly 13% at Monday's open.

The Phase 2a study (ClinicalTrials.gov Identifier: NCT06868628) is a six-month, open-label clinical trial 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 have 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 is the only fully human anti-CD3 monoclonal antibody currently in clinical development. The non-active SPMS intranasal foralumab Phase 2 trial (NCT06292923) began screening patients in November of 2023. Immunomodulation by intranasal foralumab represents a novel avenue for the treatment of neuroinflammatory and neurodegenerative human diseases.

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.

Shares of Tiziana Life Sciences (Nasdaq: TLSA) rose nearly 13% at Monday's open following the FDA approval, indicating investor confidence in the company's innovative approach to treating MSA and other neurodegenerative diseases.

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