Perth-based NeuroScientific Biopharmaceuticals has cleared a key regulatory hurdle for its planned phase two trial of its innovative StemSmart cell therapy in patients with treatment-resistant Crohn’s disease, with US regulators backing the proposed study design and key elements of the therapy’s development package.
The US Food and Drug Administration (FDA) responded positively to six of seven questions at a pre-investigational new drug (Pre-IND) meeting, which guides development ahead of a future drug submission. The medicines watchdog gave the company’s proposed trial blueprint a resounding thumbs-up and waved through its existing StemSmart human clinical evidence, bolstered by encouraging results from an earlier phase two Crohn’s trial.
The FDA also backed NeuroScientific’s StemSmart batch-testing plans before clinical use and agreed no additional non-clinical studies were required. The company’s regulatory consultants called the response very positive, noting the agency had recently been selective, granting only face-to-face meetings instead of written responses.
The only outstanding matter is a manufacturing protocol to ensure the product is free from viral contamination. NeuroScientific has agreed to implement the required testing, which is expected to have minimal impact on trial timelines.
Crohn’s disease is a lifelong, incurable condition involving chronic gut inflammation that affects an estimated 6–8 million people worldwide. Around 30 per cent of patients become refractory, meaning their disease no longer responds to conventional therapies. A similar proportion develop fistulas — abnormal tracts that can form between the bowel and other organs or the skin.
StemSmart is NeuroScientific’s patented cell therapy, made up of mesenchymal stem cells (MSCs) sourced from adult donor bone marrow. Developed at Royal Perth Hospital, its manufacturing process is designed to boost the cells’ therapeutic activity and help regulate severe inflammation. The universal donor cells are being developed as an off-the-shelf intravenous infusion, requiring no patient matching or waiting list.
A previous StemSmart study of 18 patients with refractory Crohn’s disease reported a 78 per cent overall clinical response rate, with 44 per cent of patients achieving remission, offering an encouraging signal.
The new phase two trial will now build on those promising earlier results, putting the therapy’s safety and preliminary efficacy to the test in patients with refractory and/or fistulising Crohn’s disease. The FDA’s positive response marks an important step towards that next clinical test.
NeuroScientific Biopharmaceuticals chief executive officer Nathan Smith said: “The achievement of another pivotal milestone in NSB’s clinical development for our patented StemSmart™ technology platform takes us one step closer to providing a potential treatment option for patients with this debilitating disease.”
Subject to completing regulatory, manufacturing and operational requirements, NeuroScientific aims to start the trial in Australia and the US by the end of 2026. The company has already appointed Australian contract research organisation Southern Star Research to manage clinical sites, engage clinicians and investigators. The contractor will also coordinate the logistics of delivering StemSmart to enrolled patients.
In parallel, NeuroScientific has begun transferring its patented process to Brisbane-based Q-Gen Cell Therapeutics, a specialist cell-therapy manufacturer, to support clinical-grade StemSmart supply for the trial and potential future scale-up. The company expects to complete the transfer by Christmas, subject to a Therapeutic Goods Administration audit in Australia and the FDA-required viral-contamination testing.
Management says the new study will also strengthen the evidence base for StemSmart’s efficacy in patients with fistulising Crohn’s, building on an earlier Special Access Program which recorded no adverse events but was limited to a small cohort of just five patients.
The StemSmart acquisition in 2025 marked a sharp shift for NeuroScientific, which had spent years developing EmtinB for neurodegenerative conditions. Crohn’s disease is now its lead focus, with the long-term goal of building the evidence needed to secure approval for widespread use of the therapy in a global market the company values at a whopping US$13 billion (A$18.7 billion).
NeuroScientific retains EmtinB, its protein-based peptide program for neurodegenerative conditions and sees potential to explore further StemSmart applications in lung inflammation, kidney transplantation and graft-versus-host disease. However, each would require its own clinical evidence.
With the US regulator now largely on board and a clear path to a major international trial, NeuroScientific is rounding the corner with its plan to tackle one of medicine’s most stubborn inflammatory diseases.
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By Penny Taylor
