FDA gives positive Pre-IND feedback on StemSmart™ Crohn’s trial
NeuroScientific Biopharmaceuticals (ASX:NSB) has received positive feedback from the United States Food & Drug Administration (FDA) following its Pre-Investigational New Drug (Pre-IND) meeting for a planned Phase 2 trial in refractory Crohn’s disease.
After reviewing NSB’s clinical program briefing package, the FDA provided favourable responses to 6 of the 7 questions posed by the company, including agreement with the proposed Phase 2 clinical design.
The planned trial targets patients in the United States and Australia. Trial initiation is targeted by the end of CY2026, subject to completion of regulatory, manufacturing and operational requirements.
For investors, the outcome offers early regulatory feedback ahead of a planned Investigational New Drug (IND) submission. The announcement does not state that an IND has been filed or approved.
Nathan Smith, Chief Executive Officer
“The excellent outcome from our Pre-IND meeting with the US FDA demonstrates the exceptional work the NSB team has done to ensure StemSmart™ is suitable for use in the planned Phase 2 clinical trial in treatment-resistant Crohn’s disease in the US…”
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What the FDA agreed to
The FDA’s feedback covered the trial design, the existing human data package and the product release testing approach. One manufacturing-related item remains open.
Three key endorsements
Among the key positive responses provided by the FDA were:
- Agreement with the design of NSB’s Phase 2 clinical trial in refractory Crohn’s disease.
- Agreement that current weight-of-evidence data in humans previously treated with StemSmart™ was sufficient and did not require additional non-clinical studies.
- Agreement with the release testing strategy for the StemSmart™ product.
Non-clinical studies are laboratory and animal-based work conducted before or alongside human trials. The FDA’s view that none are required beyond the existing human data may help avoid additional pre-trial work, although the announcement does not quantify any time or cost effect.
The one outstanding item
The one outstanding matter the FDA requires to be addressed relates to the control strategy for manufacturing, to ensure the product is free from viral contamination. NSB agrees with the FDA’s feedback and, according to the company, is implementing the required testing with minimal impact to timelines.
Feedback from NSB’s regulatory consultants highlighted that the FDA’s response on the briefing package was very positive. The consultants also remarked that NSB was fortunate the FDA had granted the face-to-face Pre-IND meeting, as the agency has been very selective in granting these meetings in lieu of written responses in recent months.
| Area | FDA response | Investor relevance |
|---|---|---|
| Trial design | Agreed | Clear path for the Phase 2 protocol |
| Human weight-of-evidence data | Sufficient, no additional non-clinical studies required | Fewer pre-trial hurdles |
| Release testing strategy | Agreed | Manufacturing pathway validated |
| Viral contamination control strategy | Outstanding, testing being implemented | Only open item |
Pre-IND meetings and StemSmart™ explained
A Pre-IND meeting is a regulatory milestone where the FDA reviews the clinical, scientific and product development plans of a prospective treatment. It then provides guidance ahead of a future IND submission.
This matters to investors because it allows regulatory feedback to be received before the formal filing is made.
The Pre-IND meeting date was confirmed by the FDA for 31 August 2026, and the meeting itself is the step that precedes any formal IND filing for the US Phase 2 program.
StemSmart™ is derived from adult human donor bone marrow-sourced mesenchymal stem cells (MSCs). It is produced using a patented manufacturing process developed at Royal Perth Hospital, Western Australia, which has been designed to improve therapeutic activity and clinical response.
The technology targets immune-mediated severe inflammatory diseases with a large treatment-resistant population. Refractory Crohn’s disease is the focus of NSB’s initial clinical trial.
Secondary points from the announcement include:
- StemSmart™ is a patented stem cell technology platform.
- The manufacturing process is designed to modulate pathological immune responses often associated with severe and chronic inflammatory conditions.
- The goal is to make the treatment available to patients for whom current therapies remain inadequate.
Investment thesis and next steps
The FDA’s feedback on the trial design and existing data package supports NSB’s progress toward a Phase 2 trial in a hard-to-treat patient group. The outstanding viral contamination item is the remaining matter the FDA requires to be addressed.
The company states that early indications from a previous Phase 2 trial in refractory Crohn’s disease suggest StemSmart™ MSCs are “potent, efficacious and safe.” These are early indications only, and the planned Phase 2 trial is intended to further evaluate safety and preliminary efficacy.
Next steps outlined or implied by the announcement include:
- Implementing the required viral contamination testing.
- Proceeding with the planned IND submission.
- Completing regulatory, manufacturing and operational requirements.
- Initiating the Phase 2 trial in Australia and the United States, targeted by the end of CY2026.
The announcement does not disclose trial size, funding or approval timing. NSB’s stated focus is to initiate the trial by the end of CY2026, subject to the requirements above.
Investors tracking how the trial will be run on the ground can read our detailed coverage of NSB’s Phase 2 trial execution, which covers the appointment of Southern Star Research as CRO and the separate data and safety management arrangements.
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