Stealth BioTherapeutics has started a Phase 1/2 clinical trial to investigate the safety and effectiveness of SBT-20 in patients with early-stage Huntington’s disease in the Netherlands.
SBT-20 was designed to restore the proper activity of mitochondria, the cell’s powerhouses, in Huntington’s patients. Mitochondria organelles become faulty as the disease progresses.
“Huntington’s disease is a fatal genetic disorder in which nerve cells in the brain deteriorate over time, causing patients to have progressively limited physical and mental abilities,” Jim Carr, Stealth’s chief clinical development officer, said in a press release. “Research shows that mitochondrial [impairment] may play a central role in this deterioration, making the mitochondria a prime target for study.
“This trial examines the safety and tolerability of SBT-20 at various doses and begins to explore the possible benefit of the compound in addressing mitochondrial dysfunction in Huntington’s disease,” he added.
The 24 patients in the CHALLENGE-HD trial will receive daily injections of SBT-20. Researchers will assess its safety, tolerability, and ability to improve mitochondrial and motor functions, compared with a placebo. It will also evaluate the drug’s behavior in the body, or pharmacokinetics.
There will be two parts of the study, which will be done at the Centre for Human Drug Research in Leiden. In the first part, patients will receive one of three doses of SBT-20 for seven days: 5, 15 or 25 mg. The initial results will lead to the team selecting a final dose for the second part of the study and administering it for 28 days.
“The initiation of CHALLENGE-HD is a significant milestone for Stealth as our second drug candidate enters human trials in a new therapeutic area,” Stealth’s CEO, Reenie McCarthy, said. “As a leader in mitochondrial medicine, we want to pursue the full potential of mitochondria-targeted therapies, in rare primary mitochondrial diseases, common diseases of aging and now in neurodegenerative disorders. We plan to use the results from this trial to better inform our SBT-20 pipeline development plan and the broader potential of our platform in neurodegenerative disorders.”