NSB Secures FDA Pre-IND Meeting for StemSmart Phase 2 Trial in Refractory Crohn’s

FDA Pre-IND Meeting Marks Critical Regulatory Step

NeuroScientific Biopharmaceuticals (ASX:NSB) has secured a Pre-Investigational New Drug (Pre-IND) meeting with the US Food and Drug Administration (FDA) scheduled for 31 August 2026. This meeting is a pivotal regulatory checkpoint ahead of NSB’s planned Phase 2 clinical trial for StemSmart™, its proprietary stem cell therapy targeting refractory Crohn’s disease. The FDA’s review will focus on the clinical trial design to ensure it meets standards suitable for US patients, a crucial step before submitting a formal IND application later this year.

StemSmart™ Targets a Tough Crohn’s Subset

StemSmart™ leverages a patented manufacturing process using adult human donor bone marrow-derived mesenchymal stem cells (MSCs) designed to modulate pathological immune responses in severe inflammatory diseases. Refractory Crohn’s disease, the initial focus of NSB’s clinical program, represents a patient population with limited treatment options due to resistance to conventional therapies. Early data from a previous Phase 2 trial and Special Access Program indicate StemSmart™’s potential efficacy and safety, supporting the rationale for advancing to a larger clinical trial.

Clinical Development Progress and Strategic Planning

NSB’s CEO Nathan Smith emphasised the importance of this FDA engagement: “Interfacing with the FDA on the development of StemSmart at this time is critical to ensuring that the planned clinical trial design is of a standard to support clinical translation of the product.” Preparations for the Phase 2 trial are accelerating, with the company targeting IND approval by late 2026 to enable patient enrolment in both Australia and the US.

The upcoming Phase 2 trial aims to further evaluate StemSmart™’s safety and preliminary efficacy in refractory and fistulising Crohn’s disease, potentially paving the way for broader clinical use. The trial’s design will benefit from real-world treatment data collected under NSB’s Special Access Scheme, which previously reported an 80% clinical response rate in patients with fistulising Crohn’s disease, highlighting encouraging early signals for the therapy’s effectiveness.

Manufacturing and Market Potential

StemSmart™’s manufacturing process, developed at Royal Perth Hospital, aims to enhance therapeutic activity and clinical response consistency, addressing a common challenge in stem cell therapies. NSB is currently progressing technology transfer to support manufacturing scale-up, a necessary step to meet anticipated demand if clinical trials prove successful.

Given the global market for Crohn’s disease treatments is valued at approximately US$13 billion, NSB’s development of a novel therapy for treatment-resistant cases could represent a significant commercial opportunity, contingent on successful clinical outcomes and regulatory approvals.