Perth-based NeuroScientific Biopharmaceuticals has cleared a major US regulatory hurdle for its planned phase two trial of StemSmart cell therapy in patients with treatment-resistant Crohn's disease, with the US Food and Drug Administration backing the proposed study design and key parts of the therapy's development package.
The 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 regulator approved the company's proposed trial blueprint and accepted its existing StemSmart human clinical evidence, supported by encouraging results from an earlier phase two Crohn's trial.
The FDA also backed NeuroScientific's batch-testing plans for StemSmart before clinical use and agreed that no additional non-clinical studies were required. The company's regulatory consultants described the response as very positive, noting the agency had recently been selective in 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.
“The outcome from our Pre-IND meeting with the US FDA demonstrates StemSmart is suitable for use in the planned Phase 2 clinical trial in treatment-resistant Crohn’s disease in the US,” said chief executive officer Nathan Smith.
Crohn's disease is a lifelong, incurable condition involving chronic gut inflammation that affects an estimated 6 to 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.
Analysis
Why This Matters
- The FDA's positive pre-IND response is a significant derisking step for NeuroScientific's phase two trial, potentially bringing a new treatment option closer for Crohn's patients who no longer respond to conventional therapies.
- Treatment-resistant Crohn's disease affects a substantial share of the millions of people living with the condition worldwide, and a stem cell therapy could offer an alternative where existing drugs have failed.
- A successful US trial would open the door to a formal investigational new drug application and eventual commercialisation in the world's largest pharmaceutical market.
Background
Pre-IND meetings are an early consultation between a drug developer and the FDA, used to align on trial design, manufacturing standards and the evidence needed before a formal application. Positive feedback can reduce the risk of costly delays later in the regulatory process. NeuroScientific is developing StemSmart using mesenchymal stem cells derived from adult donor bone marrow, a field that has attracted growing interest for inflammatory conditions.
Key Perspectives
NeuroScientific Biopharmaceuticals: The company sees the FDA response as validation that StemSmart is suitable for its planned phase two trial in treatment-resistant Crohn's disease, and it is moving to address the remaining manufacturing question.
US FDA: The regulator has signalled broad support for the proposed study design and existing evidence, while requiring additional viral-contamination testing as a condition of proceeding.
Patients and clinicians: For the estimated 30 per cent of Crohn's patients who become refractory, a new therapy that targets the underlying inflammation could fill a significant gap, though phase two results are still needed to show whether StemSmart is safe and effective at scale.
What to Watch
- Implementation of the viral-contamination testing protocol and its effect on phase two trial timelines.
- Whether NeuroScientific proceeds to a formal IND submission and begins patient enrolment in the US.
- The release of further data from the earlier phase two Crohn's trial that the FDA cited in its positive response.