Perth-based biotech NeuroScientific Biopharmaceuticals has bagged a crucial meeting with the United States Food & Drug Administration (FDA) as it looks to push its patented “StemSmart” technology into a Phase 2 clinical trial in the lucrative US market. The company says the Pre-Investigational New Drug, or Pre-IND, meeting has been scheduled for August 31 and represents a critical regulatory step towards testing its treatment for refractory Crohn’s disease on American patients.
The meeting will see the powerful US federal agency review the clinical, scientific and product development plan for StemSmart to ensure it is up to scratch for a formal IND submission. Getting early feedback from the FDA is a well-trodden path for biotech hopefuls as it can de-risk the expensive and lengthy approvals process and ensure the planned trial design meets the regulator’s exacting standards.
NeuroScientific says it plans to submit its full IND application for approval by the end of this calendar year.
The company’s StemSmart product 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.
The initial target is refractory Crohn’s disease, 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.
NeuroScientific 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™ 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 company is not going into its discussions with the FDA cold. It says early indications from a previous Special Access Program (SAP) trial in fistulising Crohn's disease suggested its StemSmart product was potent, effective and safe. All five patients in the trial showed improvement following treatment, with no serious adverse events recorded marking a strong outcome in a group typically resistant to conventional therapies.
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.
For any biotech company, getting a product in front of the US FDA is a significant moment. The American market is by far the largest and most valuable pharmaceutical market in the world, and a green light from its regulator is a globally recognised seal of approval.
With an August date now locked in, NeuroScientific is one step closer to potentially tapping that market. The outcome of the August meeting with the FDA will help set the agenda and requirements for the company’s up coming Phase 2 trial and importantly, its commercial pathway in the months ahead.
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