Australian biotechnology company NeuroScientific Biopharmaceuticals (ASX:NSB) has reached another milestone in its clinical development program, with the commencement of treatment under the Special Access Scheme (SAS) for its patented StemSmart mesenchymal stem cell (MSC) therapy in a fourth patient.
The treatment marks continued momentum as the company prepares for a Phase 2 clinical trial, scheduled to begin in 2026, targeting patients with fistulising Crohn’s disease. It is one of the most severe and treatment-resistant forms of inflammatory bowel disease.
Under the Therapeutic Goods Administration’s Category B Special Access Scheme, patients approved for treatment are those with advanced disease who have exhausted standard therapeutic options. The program allows compassionate use of unapproved therapies and, in this case, will generate valuable real-world data to inform the design of the upcoming trial.
NeuroScientific Chief Executive Officer Nathan Smith said the commencement of the fourth patient represented meaningful progress for patients and clinicians dealing with this complex condition.
“Treating the fourth patient under the Special Access Program with StemSmart marks an important step for both the patients and clinicians in the Crohn’s disease field,” Mr Smith said.
“We believe StemSmart has the potential to offer a new therapeutic option in an area where there are limited effective treatments. The outcomes from this program will provide direct guidance towards our Phase 2 clinical trial design, which we anticipate initiating in the second half of 2026.”
Derived from adult human donor bone marrow, StemSmart uses a patented enhancement process that improves the therapeutic activity and clinical performance of MSCs. Previous clinical data suggest the therapy is potent, efficacious, and well-tolerated, with several patients already reporting positive outcomes under compassionate access programs.
Crohn’s disease remains an area of significant unmet need, particularly for those with fistulising disease, where inflammation leads to the development of abnormal tracts between organs. The real-world treatment results from NeuroScientific’s SAS program will help de-risk development and refine the next stage of clinical testing in refractory Crohn’s disease.
As part of the company’s broader StemSmart platform strategy, NeuroScientific also sees potential applications beyond Crohn’s disease, including in organ transplant immune tolerance, lung inflammation, and graft-versus-host disease.
The therapy is being manufactured through the East Metropolitan Health Service, a TGA-certified facility, with additional manufacturing support planned from the Queensland Institute of Medical Research.
NeuroScientific said the first round of treatment results from the program is expected in January 2026.