NeuroScientific Biopharmaceuticals (NSB) Status update summary

Clinical program and trial updates

• 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.

• Two phase II 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 about 65-66 patients, while the fistulizing Crohn's trial will enroll 20-25 patients.

• Total estimated cost for both trials is AUD 15–25 million, spread over several years.

• Manufacturing is being transferred to QGen Cell Therapeutics, with regulatory audit and licensure expected by September.

StemSmart technology and clinical data

• StemSmart is a mesenchymal stem cell therapy with a unique activated cell manufacturing process, administered intravenously for systemic effect.

• Previous phase II trial in refractory Crohn's showed a 78% clinical response rate, with 44 patients achieving remission.

• The therapy is off-the-shelf, does not require HLA matching, and has a strong safety profile with no serious adverse events in over 200 patients.

• StemSmart's mechanism involves homing to inflamed tissue, modulating immune response, and promoting tissue repair.

• The product has demonstrated efficacy in other high-inflammation conditions, including graft-versus-host disease and acute renal rejection.

Regulatory, IP, and commercialization strategy

• Patent protection for the manufacturing process extends to 2034, with potential for extension and additional provisional patents in progress.

• Plans for marketing authorization in Australia and the US, with 12-year exclusivity in the US upon approval.

• Pre-IND meeting with the FDA for the refractory Crohn's study is targeted for late July; TGA audit for manufacturing licensure is scheduled for September.

• Commercialization strategy includes seeking partners for phase III and global marketing, leveraging contacts with major pharma companies.

• Expansion into additional indications is planned once Crohn's trials are underway.