NeuroScientific Biopharmaceuticals (ASX:NSB) has reported significant outcomes from its Special Access Program treating fistulising Crohn’s disease with the company's StemSmart mesenchymal stem cell therapy.
StemSmart is derived from adult human donor bone marrow-sourced MSCs and is produced using a manufacturing process that has been designed to improve therapeutic activity and clinical response.
The company said that five adults aged 18 to 49 received treatment, three of them male, and all five showed improvement, with four achieving a Clinical Response, defined as closure of at least 50 per cent of fistula openings or a 50 per cent decrease in fistula discharge. No serious adverse events were observed.
All patients experienced reductions in fistula discharge and showed improvement on internationally recognised measures, including the Crohn Disease Activity Index CDAI the Perianal Disease Activity Index PDAI and the Inflammatory Bowel Disease Questionnaire IBDQ Quality of Life Index.
MRI imaging indicated a trend toward fistula healing, though it was considered too early to observe closure. Clinically meaningful progress was also shown by the removal of at least one seton in each patient, which the company notes is a significant indicator of healing. The treatments were delivered under the TGA Special Access Scheme Category B pathway beginning in October of last year.
NeuroScientific's Chief Medical Officer, Dr Cathy Cole, said, "A clinical response rate of 80 per cent 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."
On the back of the Special Access Program, NeuroScientific has initiated development of an Australia-only pivotal Phase 2 clinical trial for fistulising Crohn’s disease, planned to commence in the second half of calendar year 2026.
Chief Executive Officer Nathan Smith said, "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."