Miro Sciences, formerly NeuroScientific Biopharmaceuticals, has announced that it plans to start a randomised Phase 2 trial of its StemSmart cell therapy for people with severe Crohn's disease who have failed existing treatments.
The company said it expects to treat the first Australian patient in the first quarter of 2027.
StemSmart is an activated mesenchymal stem cell therapy developed at Royal Perth Hospital. It targets patients who have failed at least two classes of therapy. The company's investor presentation says about 30 per cent of patients do not respond to standard therapies, and that there are 'Zero' approved treatment options once standard therapies fail.
In an open-label Phase 2 study without a placebo arm, 14 of 18 patients who were failing tumour necrosis factor inhibitors had a clinical response at day 42. Under the TGA Special Access Scheme, four of five patients with fistulising disease responded on the treating physician's assessment, and no serious adverse events were reported.
The new trial will randomise about 74 patients equally to StemSmart or placebo. Each patient will receive nine infusions over 20 weeks. Safety and clinical response at week 11 are the primary endpoints, with an interim analysis at week 24. The study is powered to detect a response rate of 55 per cent against 20 per cent for placebo.
Miro said the US Food and Drug Administration gave positive responses to six of its seven questions in October. The agency supported the trial design and asked for no further non-clinical studies. The outstanding item, additional virus testing, is described as "simple to implement and will not impact timelines".
The company said it plans to submit an investigational new drug application to the FDA in the second quarter of 2027 and to treat its first US patient later that year. It is targeting full enrolment by the second quarter of 2028 and results by the end of that year, and will then seek a partner for Phase 3.