Three of four fistulising Crohn’s disease patients treated with NeuroScientific Biopharmaceuticals Ltd (ASX:NSB)'s StemSmart™ mesenchymal stem cell (MSC) therapy under the Therapeutic Goods Administration’s (TGA) Special Access Scheme (SAS) Category B pathway achieved a successful “Clinical Response”. A Clinical Response is defined as closure of at least 50% of fistula openings, or at least a 50% reduction in fistula discharge as assessed by the treating physician or qualified investigator.
The fourth patient recorded a partial response and improved, with further clinical assessment ongoing.
NeuroScientific is positioning StemSmart™ first into Crohn’s disease, citing a global market of US$13.8 billion by 2026.
Special Access setting used for patients with limited treatment options
Patients were approved to receive StemSmart™ through the SAS Category B pathway, which provides a route for accessing unapproved therapies where conventional options are unavailable or ineffective, aiming to address unmet needs in serious conditions.
CEO Nathan Smith said the outcomes support the company’s broader commercial direction for the platform.
“These treatment results provide critical validation of the StemSmart™ MSC platform in presenting a potential therapeutic solution to patients with debilitating fistulising Crohn’s disease that have limited effective treatment options.
"This data, along with our previous clinical trial results in refractory Crohn’s disease, provides a strong foundation for our commercialisation plans for StemSmart™ moving forward.
"Together, these early outcomes allow us to advance the development of a novel therapeutic in a responsible, informed, and patient-centred fashion as it supports and accelerates our progress toward clinical trial work later this year.”
Chief medical officer Dr Cathy Cole pointed to the severity of the cohort and the real-world context of the data.
“The response rate to StemSmart™ MSC treatment seen in these patients in a real-world setting is exceptional, given the serious, debilitating and long-standing adverse nature of their condition. If you consider that for these fistula patients treated with StemSmart™, there were limited treatment options available, then the response to treatment is truly outstanding and offers hope for clinical recovery when there was previously little.”
Product focus: StemSmart MSC therapy and planned later-stage trial work
StemSmart™ is an adult donor bone marrow-sourced MSC product manufactured using a patented process designed to improve therapeutic activity and clinical response, according to the company.
SAS program data will be used to inform the design of planned later-phase trials, with the company targeting commencement in 2H 2026 and noting Phase 2 start-up activities are underway, including commercial manufacturing establishment, clinical trial development and regulatory planning.
The program data will support development of a later-phase trial in fistulising Crohn’s disease and a Phase 2 trial in refractory Crohn’s disease, with the refractory Crohn’s trial planned to treat patients across multiple jurisdictions, including the US and Australia, to support commercialisation plans.
Addressable markets: Crohn’s and broader inflammation indications
Beyond Crohn’s, the company is also highlighting additional potential clinical opportunities where chronic inflammation is a feature, including organ transplant immune tolerance, lung inflammatory disease and graft-versus-host disease (GvHD).
It listed the following markets as part of its “StemSmart™ Key Addressable Markets”:
- Kidney transplant immuno-suppressants: global market forecast to US$7.2 billion by 2030 (majority renal)
- Lung disorders: global market US$33 billion by 2034
- GvHD: global market increasing to US$5.31 billion in 2032
NeuroScientific said the latest SAS outcomes “set the foundation” for entry into the Crohn’s disease market and that manufacturing technology transfer, clinical development and regulatory planning are already progressing ahead of its planned clinical trials.