NeuroScientific Biopharmaceuticals Locks In FDA Pre-IND Meeting for StemSmart in Refractory Crohn's Disease
核心洞察
NeuroScientific Biopharmaceuticals (搜索) has scheduled a Pre-IND meeting with the US FDA for August 31 to advance its StemSmart (搜索) therapy toward a Phase 2 trial in refractory Crohn's disease (搜索).
StemSmart (搜索), an off-the-shelf mesenchymal stem cell therapy, demonstrated a 78% clinical response rate in a prior Phase 2 trial, with 44 patients achieving remission.
Four of five patients in a Special Access Program for fistulizing Crohn's disease (搜索) achieved clinical response, with reduced fistula discharge and Seton removal indicating healing.
Perth-based biotech NeuroScientific Biopharmaceuticals (搜索) has secured a crucial Pre-Investigational New Drug (Pre-IND) meeting with the United States Food and Drug Administration (FDA), scheduled for August 31, as it advances its patented "StemSmart (搜索)" technology toward a Phase 2 clinical trial in the lucrative US market. The meeting represents a critical regulatory step toward testing the treatment for refractory Crohn's disease (搜索) on American patients.
The meeting will see the FDA review the clinical, scientific and product development plan for StemSmart (搜索) to ensure it is up to standard for a formal IND submission. NeuroScientific says it plans to submit its full IND application for approval by the end of this calendar year.
NeuroScientific Biopharmaceuticals (搜索) chief executive officer Nathan Smith said: "Our upcoming Pre-IND meeting with the US FDA marks an important milestone in NSB's clinical development for our patented StemSmart (搜索) technology platform. 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."
The StemSmart Platform
StemSmart (搜索) is a stem cell technology platform derived from adult human donor bone marrow-sourced mesenchymal stem cells. It is manufactured using a patented process designed to improve the cells' therapeutic activity and control the inflammatory immune responses often associated with severe and chronic inflammatory conditions such as Crohn's disease (搜索).
The therapy is off-the-shelf, does not require HLA matching, and has a strong safety profile with no serious adverse events recorded in over 200 patients. StemSmart (搜索)'s mechanism involves homing to inflamed tissue, modulating the immune response, and promoting tissue repair. It is administered intravenously for systemic effect, using a unique activated cell manufacturing process.
Clinical Evidence to Date
The company is not entering its FDA discussions without supporting data. Early indications from a previous Special Access Program (SAP) trial in fistulising Crohn's disease (搜索) suggested the StemSmart (搜索) product was potent, effective and safe. Four out of five patients in the Special Access Program for fistulizing Crohn's disease achieved a successful clinical response, with significant reduction in fistula discharge and Seton removal indicating healing.
A previous Phase 2 trial in refractory Crohn's disease (搜索) showed a 78% clinical response rate, with 44 patients achieving remission. The product has also demonstrated efficacy in other high-inflammation conditions, including graft-versus-host disease (搜索) and acute renal rejection.
Planned Clinical Development
Two Phase 2 clinical trials are planned: an open-label trial for fistulizing Crohn's in Australia and a double-blind, placebo-controlled trial for refractory Crohn's in the US and Australia, both targeting trial commencement by year-end. The refractory Crohn's trial will enroll approximately 65–66 patients, while the fistulizing Crohn's trial will enroll 20–25 patients. The total estimated cost for both trials is AUD 15–25 million, spread over several years.
The upcoming Phase 2 trial is planned to evaluate the safety and preliminary efficacy of the treatment in patients in both Australia and the US suffering from refractory and/or fistulising Crohn's disease (搜索).
Unmet Need and Commercial Opportunity
The initial target is refractory Crohn's disease (搜索), a severe and treatment-resistant form of inflammatory bowel disease. For patients who have exhausted other options, the condition can be seriously debilitating, creating a significant unmet clinical need. The global market for Crohn's disease treatments is estimated to be worth well in excess of US$10 billion (A$14.2 billion) annually, underscoring the commercial opportunity for new and effective therapies.
Manufacturing is being transferred to QGen Cell Therapeutics (搜索), with regulatory audit and licensure expected by September. Smith added: "Outcomes from the Pre-IND meeting will strengthen our ongoing Phase 2 clinical trial preparatory activities."