NeuroScientific’s StemSmart™ Shows Early Promise in Tough Crohn’s Cases
NeuroScientific Biopharmaceuticals has reported promising initial clinical responses from its StemSmart™ Special Access Program treating Fistulising Crohn’s Disease, while preparing for a pivotal Phase 2 trial in 2026.
- Positive clinical responses observed in 3 of 4 patients treated under Special Access Program
- Phase 2 clinical trial design informed by real-world treatment data
- Manufacturing technology transfer progressing to support scale-up
- New Clinical & Scientific Advisory Board appointed to guide development
- Strong cash position of approximately $6.4 million to fund ongoing activities
Early Clinical Progress with StemSmart™
NeuroScientific Biopharmaceuticals Limited (ASX – NSB) has taken a significant step forward in its development of StemSmart™, a mesenchymal stem cell therapy targeting Fistulising Crohn’s Disease, a severe and often treatment-resistant form of the condition. The company’s Special Access Program (SAP), launched under Australia’s Therapeutic Goods Administration Special Access Scheme, has treated four patients to date, with three showing a clear clinical response. This response is defined by a reduction of at least 50% in fistula openings or discharge, marking an encouraging early validation of the therapy’s potential.
These initial outcomes, announced shortly after the December 2025 quarter, provide NeuroScientific with valuable real-world data that is directly informing the design and regulatory planning of its upcoming Phase 2 clinical trial, expected to commence later this year. The patient-centric approach underscores the company’s commitment to addressing unmet medical needs in a population with limited treatment options.
Operational and Manufacturing Readiness
Alongside clinical progress, NeuroScientific has advanced its manufacturing capabilities by transferring its proprietary StemSmart™ production technology to its contract manufacturing partner, Q-Gen. This transfer is critical to establishing Good Manufacturing Practice (GMP)-compliant production necessary for the scale and quality demands of clinical trials and eventual commercialisation. Ensuring supply chain readiness remains a key operational focus as the company aligns manufacturing capacity with anticipated Phase 2 trial requirements.
Strengthening Clinical Expertise
To support its clinical development strategy, NeuroScientific has appointed a new Clinical & Scientific Advisory Board chaired by Chief Medical Officer Dr Catherine Cole. The board brings together specialists across gastroenterology, nephrology, respiratory medicine, and cell biology, including notable figures such as Dr Lena Thin and Professor Yuben Moodley. This assembly of expertise is expected to provide robust guidance on scientific innovation, clinical trial design, and patient treatment pathways as the company advances its StemSmart™ platform.
Progress on EmtinB Program
In parallel, NeuroScientific continues to develop EmtinB™, a novel peptide therapeutic targeting retinal degeneration conditions such as glaucoma. The program is progressing through formulation optimisation and non-clinical translational studies aligned with FDA pre-Investigational New Drug (IND) guidance. The company is evaluating potential partnerships to accelerate clinical development, signalling a strategic approach to maximise value from this pipeline asset.
Financial Position and Outlook
NeuroScientific ended the quarter with a solid cash balance of approximately $6.4 million, supporting its clinical and operational activities. The company has maintained disciplined expenditure, with research and development costs around $315,000 for the quarter. This financial footing provides a runway to advance the Phase 2 trial preparations and manufacturing scale-up, although further funding rounds may be anticipated as clinical programs intensify.
Bottom Line?
With early clinical signals and operational foundations in place, NeuroScientific is poised for a pivotal year ahead as it transitions StemSmart™ into Phase 2 trials.
Questions in the middle?
- Will the positive clinical responses in the Special Access Program translate into statistically significant outcomes in Phase 2?
- How will manufacturing scale-up impact timelines and costs for the upcoming clinical trial?
- What partnership or funding strategies will NeuroScientific pursue to support expanded clinical development?
