FDA Approves Tiziana Life Sciences Phase 2 IND for Multiple System Atrophy
Tiziana Life Sciences (NASDAQ:TLSA) has received FDA approval for its Phase 2a Investigational New Drug (IND) application to study intranasal foralumab in Multiple System Atrophy (MSA) patients. The six-month, open-label clinical trial will evaluate foralumab, a fully human anti-CD3 monoclonal antibody, for its effects on microglial activation, clinical outcomes, and safety.
MSA is a rare, rapidly progressive neurodegenerative disorder with no FDA-approved treatments. The disease affects 1.9-4.9 per 100,000 people worldwide, with a median survival of 6-9 years. The trial (NCT06868628) will administer foralumab via nasal spray in eight 3-week dosing cycles, targeting T-cell mediated neuroinflammation.
Tiziana Life Sciences (NASDAQ:TLSA) ha ottenuto l'approvazione della FDA per la sua domanda IND di Fase 2a per studiare il foralumab per via intranasale in pazienti con atrofia multisistemica (MSA). Lo studio clinico open-label della durata di sei mesi valuterà il foralumab, un anticorpo monoclonale umano anti-CD3, per i suoi effetti sull'attivazione delle microglia, sugli esiti clinici e sulla sicurezza.
L'MSA è una rara e rapidamente progressiva malattia neurodegenerativa senza trattamenti approvati dalla FDA. La malattia colpisce 1,9-4,9 persone su 100.000 in tutto il mondo, con una sopravvivenza mediana di 6-9 anni. Lo studio (NCT06868628) somministrerà il foralumab tramite spray nasale in otto cicli di somministrazione da 3 settimane, mirati a contrastare l'infiammazione neuro-mediata dalle cellule T.
Tiziana Life Sciences (NASDAQ:TLSA) ha obtenido la aprobación de la FDA para su solicitud IND de fase 2a para estudiar foralumab por vía intranasal en pacientes con atrofia multisistémica (MSA). El ensayo clínico abierto de seis meses evaluará foralumab, un anticuerpo monoclonal humano anti-CD3, por sus efectos sobre la activación de la microglía, los resultados clínicos y la seguridad.
La MSA es un trastorno neurodegenerativo raro y de rápida progresión sin tratamientos aprobados por la FDA. La enfermedad afecta a 1,9-4,9 por cada 100.000 personas en todo el mundo, con una supervivencia media de 6-9 años. El ensayo (NCT06868628) administrará foralumab mediante spray nasal en ocho ciclos de dosificación de 3 semanas, con el objetivo de abordar la neuroinflamación mediada por células T.
Tiziana Life Sciences (NASDAQ:TLSA)는 비강 투여 포랄루맙을 다루는 2a상 IND 신청에 대해 FDA의 승인을 받았습니다. 6개월 간의 오픈라벨 임상시험은 완전 인간 유래 항-CD3 단클론항체인 포랄루맙이 미세아교세포 활성화, 임상적 결과 및 안전성에 미치는 영향을 평가합니다.
MSA는 희귀하고 빠르게 진행되는 신경퇴행성 질환으로 FDA 승인 치료제가 없습니다. 전 세계적으로 인구 10만 명당 1.9~4.9명이 영향을 받으며 중앙 생존 기간은 6~9년입니다. 해당 시험(NCT06868628)은 포랄루맙을 비강 스프레이로 3주 투여 주기 8회 투여하며 T세포 매개 신경염증을 표적으로 합니다.
Tiziana Life Sciences (NASDAQ:TLSA) a obtenu l'approbation de la FDA pour sa demande IND de phase 2a visant à étudier le foralumab par voie intranasale chez des patients atteints d'atrophie multisystémique (MSA). L'essai clinique ouvert de six mois évaluera le foralumab, un anticorps monoclonal anti-CD3 entièrement humain, pour ses effets sur l'activation des microglies, les résultats cliniques et la sécurité.
La MSA est une maladie neurodégénérative rare et rapidement progressive sans traitements approuvés par la FDA. La maladie touche 1,9 à 4,9 personnes pour 100 000 dans le monde, avec une survie médiane de 6 à 9 ans. L'essai (NCT06868628) administrera le foralumab par spray nasal en huit cycles de traitement de 3 semaines, visant l'inflammation neuro-médiée par les lymphocytes T.
Tiziana Life Sciences (NASDAQ:TLSA) hat die FDA-Zulassung für ihren Phase-2a-IND-Antrag erhalten, um intranasales Foralumab bei Patientinnen und Patienten mit multisystemischer Atrophie (MSA) zu untersuchen. Die sechsmonatige, offene klinische Studie wird Foralumab, einen voll-humanen Anti-CD3-Monoklonalantikörper, hinsichtlich seiner Wirkung auf die Aktivierung von Mikroglia, klinische Endpunkte und Sicherheit bewerten.
MSA ist eine seltene, schnell fortschreitende neurodegenerative Erkrankung ohne von der FDA zugelassene Behandlungen. Die Erkrankung betrifft weltweit 1,9–4,9 von 100.000 Menschen, mit einer medianen Überlebensdauer von 6–9 Jahren. Die Studie (NCT06868628) verabreicht Foralumab per Nasenspray in acht 3‑wöchigen Dosierungszyklen und zielt auf T‑Zell-vermittelte Neuroinflammation ab.
- FDA approval received for Phase 2a clinical trial in MSA patients
- Addresses an unmet medical need with no current FDA-approved treatments
- Novel non-systemic treatment approach targeting neuroinflammation
- Potential application in other neurological conditions like Multiple Sclerosis and Alzheimer's Disease
- Early-stage clinical trial with uncertain outcomes
- Target disease (MSA) has rapid progression with poor prognosis
- Small patient population due to rare disease status may limit market potential
Insights
FDA IND approval for Tiziana's Phase 2a trial in MSA represents significant regulatory progress for their lead candidate in an orphan disease with no approved treatments.
Tiziana Life Sciences has secured FDA approval for its Phase 2a Investigational New Drug (IND) application for intranasal foralumab targeting Multiple System Atrophy (MSA). This represents a significant regulatory milestone for several reasons.
MSA is an orphan disease with no FDA-approved treatments and poor prognosis (median survival 6-9 years). The disease affects approximately 1.9-4.9 per 100,000 people worldwide with incidence increasing in those over 50 years old. This creates a clear unmet medical need and potential market opportunity despite the small patient population.
The trial design shows scientific sophistication - it will evaluate foralumab's effects on microglial activation (a key neuroinflammatory marker) while measuring clinical outcomes over six months. Foralumab's mechanism as a fully human anti-CD3 monoclonal antibody that targets T-cell mediated neuroinflammation is particularly relevant as increasing evidence implicates neuroinflammation in MSA pathogenesis.
The intranasal delivery method is noteworthy as it potentially allows the drug to bypass the blood-brain barrier while minimizing systemic exposure. This approach could provide targeted neurological benefits with fewer systemic side effects typically associated with immunomodulatory therapies.
Notably, Tiziana is positioning foralumab broadly across several neuroinflammatory conditions including Multiple Sclerosis, Alzheimer's Disease, and ALS. This suggests a platform approach that could expand the potential market opportunity beyond MSA alone if the mechanism proves successful.
NEW YORK, Aug. 11, 2025 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3 monoclonal antibody, is pleased to announce that the U.S. Food & Drug Administration (FDA) have approved the IND for its Phase 2a clinical trial of intranasal foralumab in patients with Multiple System Atrophy (MSA). Multiple System Atrophy is an unmet medical need as there are currently no FDA approved therapies to treat this life limiting, orphan designated disease.
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 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.”
About Foralumab
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).
Foralumab is the only fully human anti-CD3 monoclonal antibody (mAb) 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.[1],[2]
About Tiziana Life Sciences
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. Tiziana’s technology for alternative routes of immunotherapy has been patented with several applications pending and is expected to allow for broad pipeline applications.
For more information about Tiziana Life Sciences and its innovative pipeline of therapies, please visit www.tizianalifesciences.com.
For further inquiries:
Tiziana Life Sciences Ltd
Paul Spencer, Business Development, and Investor Relations
+44 (0) 207 495 2379
email: info@tizianalifesciences.com
[1] https://www.pnas.org/doi/10.1073/pnas.2220272120
[2] https://www.pnas.org/doi/10.1073/pnas.2309221120
FAQ
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