Neuromuscular

The director of Child and Adolescent Psychiatry at Mount Sinai Health System spoke about the results of the phase 2 study of OV101 (gaboxadol), and the next steps for the phase 3 trial.

The investigators observed a dose-dependent reduction of mutant huntingtin concentration in the cerebrospinal fluid of patients who received HTTRX, supporting the launch of a confirmatory phase 3 trial.

The treatment was efficacious in patients with AQP4-IgG-positive NMOSD, which accounts for nearly three-quarters of the population with the disorder, for which there is currently no approved treatment.

The phase 2 STARS trial was the first time in 50 years that an overall improvement in patients with Angelman syndrome was observed, in this instance with a once-daily dose of gaboxadol, also known as OV101.

In a pair of phase 2 trials and a phase 1 study, patients with SMA types 1 and 2 treated with the gene therapy displayed a number of motor milestone achievements and a prolonged event-free survival rate.

Interim results of the phase 1/2 study show statistically significant reductions in SOD1 protein levels in cerebrospinal fluid, suggesting lower concentrations in the brain and spinal cord, as well.

Changes in MS diagnostic criteria and the availability of highly effective DMTs has deemed the risk for postpartum relapse nearly irrelevant for the majority of patients with MS.

Although highly efficacious DMTs have improved outcomes for a growing number of patients, the majority of people with MS are still subjected to the day-to-day consequences of the disease’s symptoms, which often have a more debilitating effect on the patient than the disease itself.

The investigational candidate is the subject of the 2-part phase 2/3 SUNFISH trial in young adult and pediatric patients with type 2 and type 3 SMA.