Neuroscientific Biopharmaceuticals Reports 80% Response Rate in Crohn’s SAS Program
StemSmart™ delivers 80% clinical response rate in fistulising Crohn’s disease patients
NeuroScientific Biopharmaceuticals Limited (ASX: NSB) has reported that 4 out of 5 patients (80%) achieved a Clinical Response in its Special Access Program using StemSmart™ MSC therapy for fistulising Crohn’s disease, results that prompted the lifting of the company’s trading halt applied on 25 May 2026.
The five-patient cohort, aged 18 to 49 (3 male), had all failed or were resistant to currently available approved therapies. Critically, all five patients showed measurable improvement; four met the formal Clinical Response threshold, while one demonstrated a partial response.
What the clinical data showed
Clinical Response is defined as the closure of ≥50% of fistula openings, OR a ≥50% decrease in fistula discharge, as assessed by the treating physician or qualified investigator.
Supporting evidence from the program included:
- All patients improved across the Crohn’s Disease Activity Index (CDAI), Perianal Disease Activity Index (PDAI), and Inflammatory Bowel Disease Questionnaire (IBDQ) Quality of Life Index.
- MRI imaging showed a trend toward fistula healing across all patients, though it was considered too early in treatment to confirm fistula closure.
- All patients had at least one seton removed, which the company describes as a significant indicator of fistula healing. Setons are surgical implants that maintain drainage and help prevent fistula infection.
- No serious adverse events were observed across all five patients.
Dr Cathy Cole, Chief Medical Officer
“A clinical response rate of 80% to a novel treatment for a serious, debilitating and long-standing medical condition, that largely affects younger adults, is exceptional. Considering these fistula patients receiving StemSmart™ were treated in a real-world setting and had limited treatment options available, the response is outstanding and offers hope for clinical recovery when there was previously little. We can confidently proceed to our phase 2 trials informed on frequency and timing of doses of MSC and MRI assessments. In keeping with our total experience to date, there are no safety concerns.”
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Understanding fistulising Crohn’s disease and why StemSmart™ matters
Fistulising Crohn’s disease is one of the most severe complications of inflammatory bowel disease. It involves the formation of abnormal tunnels (fistulas) in tissue near the digestive tract, causing chronic pain, infection risk, and significant impairment to quality of life. The condition disproportionately affects younger adults and is frequently resistant to conventional therapies, leaving many patients with limited treatment pathways.
NSB accessed this patient group through the Therapeutic Goods Administration’s (TGA) Special Access Scheme (SAS) Category B pathway. In plain terms, this pathway allows patients with serious conditions and very few remaining treatment options to receive an unapproved therapy on a patient-by-patient basis, following individual review and approval by the TGA. The program began treating patients from October CY2025.
StemSmart™ is derived from adult human donor bone marrow-sourced mesenchymal stem cells (MSCs) and is produced using a patented manufacturing process designed to enhance therapeutic activity and clinical response. MSCs are a class of adult stem cell with established anti-inflammatory properties.
For investors, outcomes generated under a real-world SAS setting carry particular weight. These are patients who had exhausted conventional options, making an 80% Clinical Response rate in this cohort a materially significant result.
Phase 2 trial initiation and the path to Marketing Authorisation
Following the SAS outcomes, NSB has initiated development of a pivotal Phase 2 clinical trial specifically for fistulising Crohn’s disease patients in Australia, with commencement anticipated in 2H CY2026. The company is pursuing a dual-track clinical strategy:
- Australia-only Phase 2 trial targeting fistulising Crohn’s disease, designed to support Marketing Authorisation in Australia.
- US & Australia Phase 2 trial targeting the broader refractory Crohn’s disease indication, previously announced on 13 January 2026, running in parallel.
Data from the SAS program will directly inform the emerging Phase 2 study design, including induction considerations, dosing schedules, and the timing of MRI scans. NSB also confirmed that the SAS outcome satisfies the Performance Shares Milestone outlined in the ASX Announcement dated 16 April 2025.
Nathan Smith, Chief Executive Officer
“The outstanding results from the initial patients in the Special Access Program have highlighted the opportunity for NSB to conduct an Australian clinical trial for fistulising Crohn’s disease; an incredibly debilitating condition with a patient population that has very few effective treatment options available to them. The pivotal data generated from this planned trial will assist NSB in its aim to obtain Marketing Authorisation for StemSmart™ to potentially benefit Australian patients and provide a pathway to commercialisation of our technology earlier than previously forecasted.”
Robert McKenzie, Non-Executive Chairman
“Our medical & scientific teams’ response to these initial results indicates that NSB is making momentous progress. The clinical success of StemSmart™ in this difficult to treat patient group is significant and reinforces NSB’s commitment to the development of StemSmart™ for the benefit of patients.”
StemSmart™ addressable market snapshot
Beyond fistulising Crohn’s disease, StemSmart™ is positioned as a platform cell therapy with potential applications across multiple inflammatory conditions. The table below summarises the addressable market figures across NSB’s key target indications. These are global market size estimates, not revenue projections for NSB.
| Indication | Global Market Size | Target Year | Status |
|---|---|---|---|
| Crohn’s Disease | ~US$13.8 billion | 2026 | Phase 2 trials initiated |
| Kidney Transplant (organ transplant immuno-suppressants) | ~US$7.2 billion | 2030 | Pipeline opportunity |
| Lung Disorders | ~US$33 billion | 2034 | Pipeline opportunity |
| Graft-versus-Host Disease (GvHD) | ~US$5.31 billion | 2032 | Pipeline opportunity |
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What investors should watch next
Key milestones to monitor as NSB advances its clinical programme include:
- Phase 2 fistulising Crohn’s disease trial (Australia-only): anticipated initiation 2H CY2026.
- Phase 2 refractory Crohn’s disease trial (US & Australia): running in parallel with the Australia-only trial.
- Marketing Authorisation application in Australia: a downstream objective contingent on pivotal Phase 2 data from the fistulising Crohn’s disease trial.
NSB is an Australian biotechnology company with a patented, platform cell therapy technology that management believes is applicable across a range of inflammatory disease indications. While the Phase 2 data required to support regulatory applications is still to come, the 80% Clinical Response rate observed under real-world SAS conditions represents a meaningful early signal. Investors should note that clinical and regulatory outcomes remain subject to the inherent uncertainties of drug development.
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