NeuroScientific Biopharmaceuticals (ASX: NSB) has confirmed that 75% of patients in the first cohort of a study into StemSmart mesenchymal stem cell (MSC) therapy to treat fistulising Crohn’s disease have demonstrated a positive clinical response following treatment.
The patients were approved by the Therapeutic Goods Administration under Australia’s Special Access Scheme (SAS) Category B pathway.
This pathway provides potential treatment options for those with serious and life-threatening conditions where no effective conventional therapies exist.
Three out of a total four patients in the SAS cohort achieved a clinical response—defined as either closure of ≥50% of fistula openings or a decrease in fistula discharge of ≥50%.
The fourth showed a partial response, providing critical validation for the StemSmart platform in a real-world setting.
Debilitating Bowel Complication
Fistulising Crohn’s disease represents one of the most severe and debilitating complications of inflammatory bowel disease, and is often resistant to currently available approved therapies.
StemSmart uses adult human donor bone marrow MSCs produced via a patented manufacturing process designed to improve therapeutic activity and clinical response.
Early indications from a previous Phase 2 trial on patients with the broader indication of refractory Crohn’s disease suggest StemSmart MSCs to be potent, efficacious, and safe.
NeuroScientific is also positioning StemSmart as a platform cell therapy to meet other chronic inflammation conditions such as organ transplant immune tolerance, lung inflammatory disease, and graft-vs-host disease.
Critical StemSmart Validation
“The treatment results provide critical validation of our StemSmart MSC platform in presenting a potential therapeutic solution to these patients, who currently have limited effective treatment options,” chief executive officer Nathan Smith said.
“These early outcomes provide a strong foundation for our commercialisation plans and allow us to advance the development of a novel therapeutic in a responsible, informed, and patient-centred fashion, as well as accelerate our progress toward clinical trial work later this year.”
NeuroScientific intends to conduct further trials into fistulising and refractory Crohn’s disease in the second half of this year, with manufacturing technology transfer, clinical development, and regulatory planning already underway.
The company expects the results to set the foundation for its entry into the $19.3 billion global Crohn’s disease market.
