The Therapeutic Goods Administration has completed its inspection of Q-Gen Cell Therapeutics, the Brisbane contract manufacturer that will make Miro Sciences' StemSmart stem cell product for a planned Phase 2 trial in refractory Crohn's disease.
Miro Sciences, formerly NeuroScientific Biopharmaceuticals, said the inspection was a major operational milestone in supplying StemSmart for the trial. The company plans to run the trial in Australia and the United States, subject to regulatory approvals, and is aiming to start it by the end of 2026.
The inspection is the latest of several steps the company has announced in recent weeks. In October, it appointed Southern Star Research as the clinical research organisation for the trial. It also reported positive feedback from a meeting with the US Food and Drug Administration held ahead of an investigational new drug application.
Chief Scientific Officer Dr Marian Sturm, who invented the StemSmart technology, said the result showed the manufacturing transfer was on track.
"The TGA inspection of StemSmart manufacturing at Q-Gen demonstrates that we are on target with our technology transfer and represents an important step towards TGA licensure of our MSC product. It is a significant milestone in our preparations for the upcoming clinical trial in refractory Crohn's disease and was only achievable through the hard work of both the MSX and Q-Gen teams, along with ongoing communications with the TGA."
The TGA audits manufacturing procedures and facilities to ensure they meet regulatory standards for supplying therapeutic goods to Australian patients in clinical trials. Miro Sciences said the audit was a critical step in moving StemSmart manufacturing to Q-Gen, which will scale up clinical product supply for patients in both countries.
Q-Gen operates within QIMR Berghofer in Brisbane and is one of Australia's largest cell therapy contract manufacturers. It has 13 cleanrooms dedicated to cell manufacturing and quality control, holds a TGA licence for cell therapy manufacture and has more than 25 years of experience making cell therapies for industry.
StemSmart is made from mesenchymal stem cells taken from the bone marrow of adult human donors. Developed at Royal Perth Hospital in Western Australia, it is produced using a patented process. The company says the process is designed to improve therapeutic activity and clinical response and to modulate the harmful immune responses often seen in severe chronic inflammatory conditions.
Miro Sciences is focusing first on refractory Crohn's disease, a severe form of the condition that does not respond to existing treatments. The company said early indications from an earlier Phase 2 trial in refractory Crohn's disease suggest StemSmart cells are potent, efficacious and safe. It plans further trials to assess safety and early efficacy in refractory and fistulising Crohn's disease, with the aim of offering treatment to patients whose current therapies are inadequate.