Tiziana Life Sciences Files Investigational New Drug Application with FDA for ALS Phase 2 Clinical Trial
Tiziana Life Sciences (Nasdaq: TLSA) has submitted an Investigational New Drug (IND) application to the FDA for a phase 2 clinical trial of intranasal foralumab in Amyotrophic Lateral Sclerosis (ALS) patients. The trial, supported by an ALS Association grant through the Hoffman ALS Clinical Trial Awards Program, will evaluate two doses of the company's intranasal foralumab in 20 patients.
Foralumab, a fully human anti-CD3 monoclonal antibody, represents Tiziana's lead development candidate. The company's intranasal foralumab programs now target three neurodegenerative diseases: ALS, Multiple Sclerosis, and Alzheimer's disease.
ALS, also known as Lou Gehrig's disease, is a fatal neurodegenerative condition affecting nerve cells in the brain and spinal cord, leading to muscle weakness and paralysis. Most patients survive less than five years after diagnosis, highlighting the urgent need for new treatment options.
Tiziana Life Sciences (Nasdaq: TLSA) ha presentato una domanda di Nuovo Farmaco Investigativo (IND) alla FDA per un trial clinico di fase 2 di foralumab intranasale in pazienti affetti da Sclerosi Laterale Amiotrofica (SLA). Lo studio, sostenuto da una sovvenzione dell'ALS Association tramite il Programma di Premi per Trial Clinici Hoffman ALS, valuterà due dosi di foralumab intranasale in 20 pazienti.
Foralumab, un anticorpo monoclonale anti-CD3 completamente umano, rappresenta il principale candidato allo sviluppo di Tiziana. I programmi di foralumab intranasale dell'azienda ora mirano a tre malattie neurodegenerative: SLA, Sclerosi Multipla e malattia di Alzheimer.
La SLA, nota anche come malattia di Lou Gehrig, è una condizione neurodegenerativa fatale che colpisce le cellule nervose nel cervello e nel midollo spinale, portando a debolezza muscolare e paralisi. La maggior parte dei pazienti sopravvive meno di cinque anni dopo la diagnosi, evidenziando l'urgente necessità di nuove opzioni terapeutiche.
Tiziana Life Sciences (Nasdaq: TLSA) ha presentado una solicitud de Nuevo Medicamento en Investigación (IND) a la FDA para un ensayo clínico de fase 2 de foralumab intranasal en pacientes con
Foralumab, un anticuerpo monoclonal anti-CD3 completamente humano, representa el principal candidato en el desarrollo de Tiziana. Los programas de foralumab intranasal de la compañía ahora se dirigen a tres enfermedades neurodegenerativas: ELA, Esclerosis Múltiple y enfermedad de Alzheimer.
La ELA, también conocida como enfermedad de Lou Gehrig, es una condición neurodegenerativa fatal que afecta las células nerviosas en el cerebro y la médula espinal, llevando a debilidad muscular y parálisis. La mayoría de los pacientes sobrevive menos de cinco años después del diagnóstico, lo que resalta la urgente necesidad de nuevas opciones de tratamiento.
티지아나 라이프 사이언스 (Nasdaq: TLSA)는 제2상 임상 시험을 위한 FDA에 대한 임상 시험 신약 신청서(IND)를 제출했습니다. 이 시험은 근위축성 측삭 경화증(ALS) 환자들을 대상으로 하는 것으로, ALS 협회에서 지원하는 호프만 ALS 임상 시험 보조금을 통해 진행됩니다. 이 시험에서는 20명의 환자에게 회사의 비강 투여용 포랄루맙의 두 가지 용량을 평가할 예정입니다.
포랄루맙은 완전 인간 항-CD3 단클론 항체로, 티지아나의 주요 개발 후보 물질을 나타냅니다. 회사의 비강 투여용 포랄루맙 프로그램은 현재 ALS, 다발성 경화증 및 알츠하이머병의 세 가지 신경퇴행성 질환을 목표로 하고 있습니다.
ALS는 루 게릭병으로도 알려져 있으며, 뇌와 척수의 신경 세포에 영향을 미치는 치명적인 신경퇴행성 질환으로, 근육 약화와 마비를 초래합니다. 대부분의 환자는 진단 후 5년을 채 채우지 못하고 생존하므로, 새로운 치료 옵션의 긴급한 필요성이 강조됩니다.
Tiziana Life Sciences (Nasdaq: TLSA) a soumis une demande de Nouveau Médicament Investigational (IND) à la FDA pour un essai clinique de phase 2 de foralumab intranasal chez des patients atteints de Sclérose Latérale Amyotrophique (SLA). L'essai, soutenu par une subvention de l'ALS Association à travers le Programme de Prix des Essais Cliniques Hoffman ALS, évaluera deux doses de foralumab intranasal chez 20 patients.
Foralumab, un anticorps monoclonal anti-CD3 entièrement humain, représente le principal candidat au développement de Tiziana. Les programmes de foralumab intranasal de l'entreprise ciblent désormais trois maladies neurodégénératives : SLA, Sclérose en Plaques et maladie d'Alzheimer.
La SLA, également connue sous le nom de maladie de Lou Gehrig, est une condition neurodégénérative fatale affectant les cellules nerveuses dans le cerveau et la moelle épinière, entraînant une faiblesse musculaire et une paralysie. La plupart des patients survivent moins de cinq ans après le diagnostic, soulignant le besoin urgent de nouvelles options de traitement.
Tiziana Life Sciences (Nasdaq: TLSA) hat einen Antrag auf ein Investigational New Drug (IND) bei der FDA für eine Phase-2-Studie zu intranasalem Foralumab bei Patienten mit Amyotropher Lateralsklerose (ALS) eingereicht. Die Studie, die durch einen Zuschuss der ALS Association im Rahmen des Hoffman ALS Clinical Trial Awards Program unterstützt wird, wird zwei Dosen des intranasalen Foralumab bei 20 Patienten evaluieren.
Foralumab, ein vollständig menschlicher anti-CD3 monoklonaler Antikörper, stellt den Hauptentwicklungskandidaten von Tiziana dar. Die intranasalen Foralumab-Programme des Unternehmens zielen nun auf drei neurodegenerative Erkrankungen ab: ALS, Multiple Sklerose und Alzheimer-Krankheit.
ALS, auch bekannt als Lou-Gehrig-Krankheit, ist eine tödliche neurodegenerative Erkrankung, die Nervenzellen im Gehirn und Rückenmark betrifft und zu Muskelschwäche und Lähmung führt. Die meisten Patienten überleben weniger als fünf Jahre nach der Diagnose, was den dringenden Bedarf an neuen Behandlungsoptionen unterstreicht.
- FDA IND application submitted for Phase 2 ALS trial
- Received ALS Association grant funding
- Development expanding to three major neurodegenerative diseases
- None.
Insights
Tiziana's IND submission for intranasal foralumab represents an important developmental milestone for their lead asset. The FDA filing for a Phase 2 ALS trial advances their regulatory pathway and expands their neurological disease portfolio which now targets three significant neurodegenerative conditions: ALS, Multiple Sclerosis, and Alzheimer's disease.
The grant from the ALS Association provides external validation and partial funding support, reducing capital requirements for this specific program. This industry-academia framework strengthens Tiziana's development approach through collaborative expertise.
The planned 20-patient clinical trial will evaluate two doses, focusing on safety and preliminary efficacy signals. This is a modest-sized study appropriate for this stage of development but will require larger trials for eventual approval considerations.
For a small-cap biotech (
ALS represents a severe unmet medical need with treatment options and poor outcomes. However, investors should recognize that neurodegenerative disease clinical programs face historically high failure rates, and this Phase 2 study represents just one step in a lengthy development process that will require additional capital and successful outcomes in larger studies.
The IND submission for intranasal foralumab in ALS represents a novel therapeutic approach targeting neuroinflammation through CD3 modulation. Foralumab's mechanism as a fully human anti-CD3 monoclonal antibody aims to regulate T-cell function and potentially moderate neuroinflammatory processes implicated in ALS pathology.
The intranasal delivery system is particularly noteworthy as it may facilitate greater central nervous system penetration while potentially minimizing systemic immunosuppressive effects common with systemic administration of immunomodulatory agents. This delivery innovation could address a critical challenge in neurological drug development.
ALS pathophysiology involves multiple cellular mechanisms including neuroinflammation, making immunomodulatory approaches mechanistically rational. However, the complex and multifactorial nature of ALS has historically complicated therapeutic development, with numerous failed clinical programs despite promising preclinical data.
The 20-patient trial design suggests an early Phase 2 approach focused primarily on safety, tolerability, and biomarker exploration rather than definitive efficacy. Given ALS's rapid progression (typically fatal within five years of diagnosis), the trial will likely incorporate functional assessment measures alongside safety endpoints.
The expansion to three neurodegenerative indications suggests Tiziana is pursuing a platform approach, leveraging potential common neuroinflammatory pathways across different conditions. This strategy could enhance pipeline value if positive signals emerge in any indication.
NEW YORK, March 04, 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, today announced the submission of its Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a phase 2 clinical trial in ALS. This pivotal step marks a significant advancement in the company's commitment to advance a new treatment approach for Amyotrophic Lateral Sclerosis (ALS) which is supported by the ALS Association.
This IND filing follows the prestigious award of a grant as part of the Hoffman ALS Clinical Trial Awards Program from the ALS Association. Tiziana’s intranasal foralumab development programs are now focused on 3 neurodegenerative diseases. ALS, Multiple Sclerosis, and Alzheimer’s disease. This grant provides a framework for industry-academia collaboration where new discoveries are translated to clinical studies through innovative research and development.
ALS, also known as Lou Gehrig's disease is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord, ultimately leading to muscle weakness and paralysis. ALS is an orphan disease, but its clinical course can be rapid with marked disability even at an early stage. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. The disease is always fatal, usually within five years of diagnosis. Despite its devastating impact, treatment options remain limited, highlighting the critical importance of advancing research efforts like those undertaken by Tiziana Life Sciences.
Ivor Elrifi, CEO of Tiziana Life Sciences, expressed enthusiasm about this milestone, stating, "We are excited to initiate this important clinical study with the support of the ALS Association grant. This filing represents a significant achievement for our team and underscores our commitment to addressing the urgent medical needs of ALS patients."
Upon FDA clearance of the IND application, Tiziana plans to commence a 20-patient clinical trial of two doses of Tiziana’s novel and patented therapeutic candidate, intranasal foralumab, aimed at evaluating the safety and early-stage parameters of disease improvement in Amyotrophic Lateral Sclerosis (ALS). The company remains dedicated to delivering innovative solutions that can potentially improve outcomes and quality of life for ALS patients worldwide.
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. The FDA has recently allowed an additional 20 patients to be enrolled in this EA program. 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).
Activated T cells play an important role in the inflammatory process. Foralumab, the only fully human anti-CD3 monoclonal antibody (mAb) currently in clinical development, binds to the T cell receptor and dampens inflammation by modulating T cell function, thereby suppressing effector features in multiple immune cell subsets. This effect has been observed in patients with COVID and with multiple sclerosis, as well as in healthy normal subjects. The non-active SPMS intranasal foralumab Phase 2 trial (NCT06292923) began screening patients in November of 2023. Immunomodulation by nasal anti-CD3 mAb represents a novel avenue for 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.
Forward-Looking Statements
Certain statements made in this announcement are forward-looking statements. These forward-looking statements are not historical facts but rather are based on the Company's current expectations, estimates, and projections about its industry, its beliefs, and assumptions. Words such as 'anticipates,' 'expects,' 'intends,' 'plans,' 'believes,' 'seeks,' 'estimates,' and similar expressions are intended to identify forward-looking statements. These statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties, and other factors, some of which are beyond the Company's control, are difficult to predict, and could cause actual results to differ materially from those expressed or forecasted in the forward-looking statements. The Company cautions security holders and prospective security holders not to place undue reliance on these forward-looking statements, which reflect the view of the Company only as of the date of this announcement. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Tiziana’s Annual Report on Form 20-F for the year ended December 31, 2023, and other periodic reports filed with the Securities and Exchange Commission.The forward-looking statements made in this announcement relate only to events as of the date on which the statements are made. The Company will not undertake any obligation to release publicly any revisions or updates to these forward-looking statements to reflect events, circumstances, or unanticipated events occurring after the date of this announcement except as required by law or by any appropriate regulatory authority.
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
What is the purpose of Tiziana's (TLSA) Phase 2 clinical trial for ALS?
How many patients will be enrolled in TLSA's ALS Phase 2 trial?
What diseases is TLSA targeting with intranasal foralumab?
What grant did TLSA receive for their ALS research?
