Latest news with #EDSS
Business Upturn
06-05-2025
- Health
- Business Upturn
Tiziana Life Sciences Announces Comprehensive Positive Results from Study of Nasal Foralumab in Patients with Multiple Sclerosis
Meaningful Fatigue score reduction seen in study is a critically important quality of life (QoL) measure for Multiple Sclerosis patients. All patients experienced stabilization of Expanded Disability Status Scale (EDSS) scores. TSPO-PET imaging showed significant reductions in microglial activation at six months (p<0.05). NEW YORK, May 06, 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 promising results from an open-label clinical study evaluating nasal foralumab, the world's only fully human anti-CD3 monoclonal antibody administered intranasally, for the treatment of non-active secondary progressive multiple sclerosis (na-SPMS). This comprehensive study demonstrated that nasal foralumab was safe, induced potent regulatory immune responses, reduced microglial activation, and stabilized clinical progression in patients suffering from progression independent of relapse activity (PIRA)—a major unmet need in the treatment of MS, and a key disease endpoint for intranasal foralumab development. The article is titled 'Nasal foralumab treatment of PIRA induces regulatory immunity, dampens microglial activation and stabilizes clinical progression in non-active secondary progressive MS.' This study is the first to integrate, F18TSPO PET, Proteomics, and Clinical assessments in na-SPMS. These findings in MFIS score reduction are a critically important Quality of Life (QoL) measure for patients with MS. Study Highlights: Ten patients with na-SPMS, who continued to progress despite B-cell therapy, were treated with nasal foralumab for a minimum of six months. No serious or severe treatment-related adverse events were reported. All patients experienced stabilization of their Expanded Disability Status Scale (EDSS) scores; three of four patients that were treated continuously for 12 months showed improvement. Fatigue, a major symptom burden in MS, improved in six out of ten patients, as measured by the Modified Fatigue Impact Scale (MFIS). Total MFIS scores correlated strongly with mGALP scores in the hippocampus (r=0.89, p=0.007) at baseline. No new T2 lesions were observed on MRI. TSPO-PET imaging showed significant reductions in microglial activation at six months (p<0.05). Single-cell RNA sequencing revealed early and sustained changes in peripheral immune cells, including increased regulatory T cells (Tregs) and expression of TGFβ across multiple cell types. 'Our findings mark a significant advancement for patients living with non-active Secondary Progressive MS, who currently have very limited treatment options,' said Tanuja Chitnis, M.D., Principal Investigator and Professor of Neurology at Harvard Medical School and senior neurologist at Brigham and Women's Hospital, a founding member of Mass General Brigham Healthcare System. 'Nasal administration of foralumab represents a novel, non-invasive approach that not only induces regulatory immunity but also reduces harmful CNS inflammation.' PIRA and progressive forms of MS are characterized by central nervous system-centric inflammation driven by microglial activation, processes not adequately addressed by existing therapies. Traditional MS treatments targeting B-cells and cell trafficking have limited efficacy in managing progression behind the blood-brain barrier. Nasal foralumab leverages the mucosal immune system to induce regulatory immune responses, offering a novel mechanism to suppress CNS inflammation without the systemic immunosuppression seen with intravenous therapies. Previous studies showed that nasal anti-CD3 could treat progressive MS in animal models by expanding LAP+ and IL-10+ Tregs, providing the scientific rationale for this human study. In parallel with these encouraging results, Tiziana Life Sciences has initiated a randomized, double-blind, placebo-controlled Phase 2 clinical trial to further assess the efficacy and safety of nasal foralumab in a larger cohort of patients with na-SPMS. This trial is expected to reach top line data read out at the end of 2025. 'We are incredibly excited by these results, which validate the potential of nasal foralumab to fundamentally shift the treatment paradigm for progressive MS,' said Ivor Elrifi, Chief Executive Officer of Tiziana Life Sciences. 'We are also committed to advancing this promising therapy into other larger clinical studies, such as Alzheimer's Disease and ALS as quickly as possible.' This article has been submitted for peer review, and the full preprint is available here: 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 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 LtdPaul Spencer, Business Development, and Investor Relations+44 (0) 207 495 2379 email: [email protected]
Yahoo
30-04-2025
- Business
- Yahoo
Immunic Announces Vidofludimus Calcium Reduced Risk of Disability Worsening by 30% in Primary Progressive Multiple Sclerosis Patients from Phase 2 CALLIPER Trial
– Reduced Relative Risk of 24-Week Confirmed Disability Worsening Events by 20% in Overall Study Population Compared to Placebo; Even More Prominent 30% Reduction in High Unmet Need Population of Primary Progressive Multiple Sclerosis – – Showed Consistent Reduction of Disability Worsening in Subpopulations Without Inflammatory Lesions at Baseline in Overall Study Population; Reduced Relative Risk of 24-Week Confirmed Disability Worsening Events in Patients Without Gadolinium-Enhancing Lesions at Baseline by 29% Compared to Placebo – – Reduced Annualized Rate of Thalamic Brain Volume Loss by 20% Compared to Placebo – – Confirmed Favorable Safety and Tolerability Observed in Previous Clinical Trials; No New Safety Signals Identified – – Webcast to be Held Today, April 30, at 8:00 am ET – NEW YORK, April 30, 2025 /PRNewswire/ -- Immunic, Inc. (Nasdaq: IMUX), a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases, today announced positive data from its phase 2 CALLIPER trial of nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), in patients with progressive multiple sclerosis (PMS). Clinical Endpoints In the overall PMS patient population (n=467), vidofludimus calcium reduced the relative risk of 24-week confirmed disability worsening (24wCDW) events based on changes in the expanded disability status scale (EDSS) by 20% compared to placebo. Further analyses by disease subtype demonstrated that vidofludimus calcium was associated with a 30% reduction in the relative risk of 24wCDW events in the primary progressive multiple sclerosis (PPMS) study population (n=152) compared to placebo and a respective 15% reduction in the non-active secondary progressive multiple sclerosis (naSPMS) study population (n=268). Reduction of confirmed disability worsening is widely considered to be the most recognized regulatory approval endpoint for registrational studies in progressive forms of multiple sclerosis (MS). Immunic believes that these reductions in 24wCDW events are remarkable in light of the overwhelming non-activity of the CALLIPER population. In support, the evidence of focal inflammatory disease (gadolinium-enhancing lesions at baseline in 6.8% of naSPMS and 17.8% of PPMS patients) and the number of on-study relapses (5.8% in the overall patient population) are lower than in most historical PMS trials. A consistent reduction of disability worsening was observed in the different subpopulations with or without inflammatory gadolinium-enhanced lesion activity at baseline and during the study. Vidofludimus calcium reduced the relative risk of 24wCDW events in patients without gadolinium-enhancing lesions at baseline by 29% compared to placebo. In historical studies, such patients were largely shown to not benefit from current anti-inflammatory therapies. Immunic believes the substantial effect in these patients, therefore, underlines the previously observed neuroprotective effect of Nurr1 activation by vidofludimus calcium. Magnetic Resonance Imaging (MRI) Endpoints While vidofludimus calcium had a modest benefit on the exploratory primary MRI endpoint (annualized rate of percent brain volume change: 5% improvement compared to placebo), vidofludimus calcium substantially reduced the annualized rate of thalamic brain volume loss by 20% in patients with PMS compared to placebo. Change in thalamic volume is considered a more sensitive MRI atrophy marker. Thalamic atrophy is prevalent in PMS and data has shown strong associations with clinical disability progression. The total volume of new or enlarging T2 lesions showed a substantial difference between vidofludimus calcium and placebo over time, with vidofludimus calcium decreasing and placebo increasing (mean percent change, 3.19% benefit for vidofludimus calcium (-0.22%) over placebo (+2.97%) at month 24). "The tremendous reduction of confirmed disability worsening in PMS patients is a wonderful confirmation of the objectives of this exploratory phase 2 trial. CALLIPER was designed to evaluate the clinical efficacy, safety and tolerability of vidofludimus calcium in a broad set of PMS patients to determine the suitability of advancing to a confirmatory phase 3 program," said Daniel Vitt, Ph.D., Chief Executive Officer of Immunic. "We are particularly thrilled to see such a clinically meaningful effect in the PPMS population, with a 30% reduction in the relative risk of 24-week confirmed disability worsening events, which would be the endpoint of a future phase 3 registration study, outperforming historic trials in PPMS regarding numerical reduction of disability progression events. We believe that vidofludimus calcium may represent a novel and exciting approach for people living with PMS, where there continues to be a huge unmet medical need given only one approved therapy. We look forward to discussing these results with healthcare authorities to determine appropriate next steps for vidofludimus calcium in PMS." "The most significant unmet need in MS continues to be the lack of safe and effective therapies that can slow or halt disease progression, especially in PPMS and non-active SPMS. Vidofludimus calcium is the only medicine in development for MS that has been shown to be a potent activator of Nurr1, which plays a key role in neuroprotection. With this unique mode of action, vidofludimus calcium could become the first real neuroprotective treatment option for patients with progressive forms of MS," added Andreas Muehler, M.D., M.B.A., Chief Medical Officer of Immunic. "In particular, the 29% reduction of 24-week confirmed disability worsening events for vidofludimus calcium over placebo in the 391 patients without gadolinium-enhancing lesions at baseline underlines the drug's breakthrough potential in addressing the high unmet need of slowing neurodegeneration in MS patients. We believe today's exciting results of the phase 2 CALLIPER trial further validate vidofludimus calcium's scientific rationale, specifically driven by the impressive numerical benefit in reducing disability worsening, which deserves to be further tested in a phase 3 registration trial. Since almost all disability events in a non-active PMS population are known to be progression independent of relapse activity (PIRA), we believe the CALLIPER results also corroborate the benefit on disability progression expected for people suffering from relapsing forms of MS, allowing for a beneficial read-through to our ongoing phase 3 ENSURE trials." Safety and Tolerability The top-line CALLIPER data set confirmed the favorable safety and tolerability profile of vidofludimus calcium already observed in previous clinical trials. No new safety signals were identified. The occurrence of treatment-emergent adverse events and serious adverse events showed a similar frequency between both treatment arms. The rate of treatment-emergent adverse events was 69.4% of vidofludimus calcium-treated patients compared with 68.5% of patients on placebo. Likewise, serious adverse events were rare and only observed in 8.1% of vidofludimus calcium-treated patients, and in 6.5% of patients on placebo. Additionally, no Hy's law range cases regarding elevations of liver enzymes were observed during the study. About CALLIPER CALLIPER is an international, multicenter, randomized, double-blind, placebo-controlled, exploratory phase 2 trial which enrolled 467 patients at more than 70 sites throughout North America as well as Western, Central and Eastern Europe. Patients, aged 18 to 65 years and without limitation on disease duration, were randomized to either 45 mg daily doses of vidofludimus calcium or placebo and treated for up to 120 weeks. CALLIPER enrolled mainly patients with PPMS (n=152/467) and naSPMS (n=268/467). All patients showed no evidence of relapse in the last 24 months before randomization. For more information, please visit: NCT05054140. Analysis of the full CALLIPER data set is ongoing and will be presented at upcoming scientific meetings. The company's phase 3 clinical trial program of vidofludimus calcium in relapsing multiple sclerosis is ongoing and expected to be completed in 2026. Webcast InformationImmunic will host a webcast today at 8:00 am ET to discuss these results. To participate in the webcast, please register in advance at: or on the "Events and Presentations" section of Immunic's website at: Registrants will receive a confirmation email containing a link for online participation or a telephone number for dial in access. An archived replay of the webcast will be available approximately one hour after completion on Immunic's website at: About Progressive Multiple SclerosisWhile multiple sclerosis (MS) in general is an autoimmune disease characterized by immune-mediated demyelination that affects the brain, spinal cord and optic nerve, the progressive phase of the disease seems to be dominated by significant neurodegenerative mechanisms. Progressive multiple sclerosis (PMS) is a clinical form of MS characterized by gradual accrual of disability independent of relapses over time. PMS includes both primary progressive MS (PPMS) and secondary progressive MS (SPMS). PPMS is characterized by steadily worsening neurologic function from the onset of symptoms without initial relapse or remissions. SPMS is identified following an initial relapsing-remitting course, after which the disease becomes more steadily progressive, with (active SPMS) or without (non-active SPMS) MRI lesions and/or relapses present. About Vidofludimus Calcium (IMU-838)Vidofludimus calcium is an orally administered investigational small molecule drug being developed for chronic inflammatory and autoimmune diseases, currently in late-stage clinical trials for multiple sclerosis (MS). Uniquely, vidofludimus calcium's first-in-class, dual mode of action combines neuroprotective, anti-inflammatory and anti-viral effects to target the complex pathophysiology of MS. As a selective immune modulator, it activates the neuroprotective transcription factor, nuclear receptor-related 1 (Nurr1), which provides direct and indirect neuroprotective effects. Additionally, vidofludimus calcium achieves anti-inflammatory and anti-viral effects through highly selective inhibition of the enzyme dihydroorotate dehydrogenase (DHODH). Vidofludimus calcium is currently being evaluated in phase 3 and phase 2 clinical trials for the treatment of relapsing and progressive MS, respectively. In a phase 2 clinical trial, it has shown therapeutic activity in patients suffering from relapsing-remitting MS, significantly reducing MRI lesions and demonstrating encouraging results in reducing confirmed disability worsening. Additionally, vidofludimus calcium has demonstrated clinical benefits in progressive MS patients by showing substantial reductions in the relative risks of 24-week confirmed disability progression events and in thalamic brain volume in a phase 2 clinical trial. To date, vidofludimus calcium has been exposed to approximately 2,700 individuals and has shown an attractive pharmacokinetic, safety and tolerability profile. Vidofludimus calcium is not yet licensed or approved in any country. About Immunic, Inc. (Nasdaq: IMUX) is a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases. The company's lead development program, vidofludimus calcium (IMU-838), is currently in phase 3 clinical trials for the treatment of relapsing multiple sclerosis, which are expected to be completed in 2026. It has already shown therapeutic activity in phase 2 clinical trials in patients suffering from relapsing-remitting multiple sclerosis and progressive multiple sclerosis. Vidofludimus calcium combines neuroprotective effects, through its mechanism as a first-in-class nuclear receptor related 1 (Nurr1) activator, with additional anti-inflammatory and anti-viral effects, by selectively inhibiting the enzyme dihydroorotate dehydrogenase (DHODH). IMU-856, which targets the protein Sirtuin 6 (SIRT6), is intended to restore intestinal barrier function and regenerate bowel epithelium, which could potentially be applicable in numerous gastrointestinal diseases, such as celiac disease as well as inflammatory bowel disease, Graft-versus-Host-Disease and weight management. IMU-381, which currently is in preclinical testing, is a next generation molecule being developed to specifically address the needs of gastrointestinal diseases. For further information, please visit: Cautionary Statement Regarding Forward-Looking StatementsThis press release contains "forward-looking statements" that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this press release regarding strategy, future operations, future financial position, future revenue, projected expenses, sufficiency of cash and cash runway, expected timing, development and results of clinical trials, prospects, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to Immunic's development programs and the targeted diseases; the potential for vidofludimus calcium to safely and effectively target diseases; preclinical and clinical data for vidofludimus calcium; the feasibility of advancing vidofludimus calcium to a confirmatory phase 3 clinical trial in progressive multiple sclerosis; the timing of current and future clinical trials and anticipated clinical milestones; the nature, strategy and focus of the company and further updates with respect thereto; and the development and commercial potential of any product candidates of the company. Immunic may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Such statements are based on management's current expectations and involve substantial risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, increasing inflation, tariffs and macroeconomics trends, impacts of the Ukraine – Russia conflict and the conflict in the Middle East on planned and ongoing clinical trials, risks and uncertainties associated with the ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the availability of sufficient financial and other resources to meet business objectives and operational requirements, the fact that the results of earlier preclinical studies and clinical trials may not be predictive of future clinical trial results, any changes to the size of the target markets for the Company's products or product candidates, the protection and market exclusivity provided by Immunic's intellectual property, risks related to the drug development and the regulatory approval process and the impact of competitive products and technological changes. A further list and descriptions of these risks, uncertainties and other factors can be found in the section captioned "Risk Factors," in the company's Annual Report on Form 10-K for the fiscal year ended December 31, 2024, filed with the SEC on March 31, 2025, and in the company's subsequent filings with the SEC. Copies of these filings are available online at or Any forward-looking statement made in this release speaks only as of the date of this release. Immunic disclaims any intent or obligation to update these forward-looking statements to reflect events or circumstances that exist after the date on which they were made. Immunic expressly disclaims all liability in respect to actions taken or not taken based on any or all of the contents of this press release. Contact Information Immunic, BreuVice President Investor Relations and Communications+49 89 2080 477 US IR ContactRx Communications GroupPaula Schwartz+1 917 633 7790immunic@ US Media ContactKCSA Strategic CommunicationsCaitlin Kasunich+1 212 896 1241ckasunich@ View original content to download multimedia: SOURCE Immunic, Inc. Sign in to access your portfolio
