NeuroScientific Biopharmaceuticals (ASX:NSB) has reached a significant milestone in its clinical development program, announcing TGA approval for the first three patients to receive its proprietary stem cell therapy, StemSmart, under Australia’s Special Access Program for the treatment of fistulising Crohn’s disease.
The approvals mark the formal commencement of Cohort 1 within the company’s Special Access Program. The company described it as a significant step forward following its acquisition of Isopogen WA and its patented StemSmart technology earlier this year.
StemSmart is a mesenchymal stem cell (MSC) therapy derived from adult human bone marrow. The company’s proprietary manufacturing process is designed to enhance the clinical performance of MSCs, addressing a persistent gap in treatment for patients with severe, treatment-resistant Crohn’s disease. Earlier studies have shown strong safety and early efficacy signals, supporting its potential as a new therapeutic option in one of the most challenging forms of inflammatory bowel disease.
NeuroScientific Chief Executive Officer Nathan Smith said the approvals represent both a clinical and strategic inflection point for the Company. “I am extremely excited with the TGA approval to treat patients with StemSmart as we advance the Special Access Program towards completion by mid-2026,” Mr Smith said. “This milestone, alongside our progress in transferring the manufacturing technology to Q-Gen in parallel, paves the way towards initiation of our Phase 2 clinical trial next year.”
The Special Access Program enables the use of unapproved therapeutic goods under Category B of the TGA scheme, providing physicians with access to innovative treatments for patients who have exhausted standard care options. NeuroScientific expects the data and patient outcomes generated through this program to directly inform regulatory pathways and trial design for a full Phase 2 study in refractory Crohn’s disease, planned for 2026.
Beyond Crohn’s disease, NeuroScientific sees broader clinical applications for the StemSmart technology platform, including kidney transplant immunosuppression, lung disorders, and graft-versus-host disease (GvHD).