NeuroScientific Biopharmaceuticals reports encouraging clinical responses in fistulising Crohn’s disease patients treated under Australia’s Special Access Scheme, setting the stage for pivotal Phase 2 trials later this year.
- 3 of 4 patients achieved significant clinical response with StemSmart™ therapy
- 1 patient showed partial improvement, with ongoing assessments
- Results validate StemSmart™ platform in real-world treatment of severe Crohn’s disease
- Phase 2 clinical trials planned for second half of 2026
- StemSmart™ targets a global Crohn’s disease market valued at around US$13 billion
Promising Clinical Outcomes in a Challenging Condition
NeuroScientific Biopharmaceuticals Limited (ASX, NSB) has announced encouraging clinical results from its StemSmart™ mesenchymal stem cell therapy in patients suffering from fistulising Crohn’s disease, a severe and notoriously difficult-to-treat form of inflammatory bowel disease. Under the Therapeutic Goods Administration’s Special Access Scheme (SAS), four patients received treatment, with three achieving a clear clinical response and a fourth showing partial improvement.
Clinical response was defined as either closure of at least half of the fistula openings or a reduction in fistula discharge by 50% or more, as assessed by treating physicians. These outcomes are particularly notable given the limited treatment options available for this patient group, many of whom have exhausted conventional therapies.
Validation of StemSmart™ in Real-World Use
The positive responses observed provide critical validation for NeuroScientific’s StemSmart™ platform outside of controlled clinical trial settings. The therapy utilises patented manufacturing processes to enhance the therapeutic activity of adult human donor bone marrow-derived mesenchymal stem cells, aiming to modulate the immune system and reduce chronic inflammation.
Chief Executive Officer Nathan Smith highlighted the significance of these results, noting that they lay a strong foundation for the company’s commercialisation plans and upcoming clinical trials. Chief Medical Officer Dr Cathy Cole described the response rate as exceptional, especially given the severity and chronic nature of the patients’ conditions.
Next Steps, Phase 2 Trials and Market Potential
Building on these findings, NeuroScientific is preparing to launch Phase 2 clinical trials in the second half of 2026, targeting both fistulising and refractory Crohn’s disease across multiple jurisdictions including Australia and the United States. Preparations are underway for commercial manufacturing scale-up, clinical trial design, and regulatory submissions.
The global market for Crohn’s disease therapies is projected to reach approximately US$13.8 billion by 2026, underscoring the significant commercial opportunity if StemSmart™ can deliver consistent clinical benefits. Moreover, the platform’s potential extends beyond Crohn’s disease to other immune-mediated inflammatory conditions such as organ transplant immune tolerance, lung inflammatory diseases, and graft-versus-host disease.
A Cautious Optimism Amid Early Data
While the small patient cohort limits broad conclusions, these early real-world results offer a compelling glimpse into StemSmart™’s potential. The company’s transparent communication and regulatory engagement suggest a measured but confident approach to advancing this novel therapy through the clinical pipeline.
Bottom Line?
NeuroScientific’s next phase of clinical trials will be pivotal in confirming StemSmart™’s role in transforming treatment for severe Crohn’s disease.
Questions in the middle?
- Will Phase 2 trials replicate these early clinical response rates in larger patient populations?
- How will regulatory authorities in key markets respond to the data from the Special Access Program and upcoming trials?
- What timelines and strategies will NeuroScientific adopt for commercial manufacturing scale-up?