Phase 3 Trial of Oral Ganaxolone Fails to Meet Primary Endpoint in Tuberous Sclerosis Complex
Key Insights
Marinus Pharmaceuticals' Phase 3 TrustTSC trial evaluating oral ganaxolone for tuberous sclerosis complex (search) (TSC (search))-associated seizures did not meet its primary endpoint.
The trial aimed to demonstrate a statistically significant reduction in 28-day frequency of TSC (search)-associated seizures with ganaxolone compared to placebo.
Ganaxolone treatment resulted in a 19.7% median reduction in seizure frequency, versus 10.2% with placebo, a difference that was not statistically significant.
Marinus Pharmaceuticals' Phase 3 TrustTSC study of oral ganaxolone for seizures associated with tuberous sclerosis complex (search) (TSC (search)) has failed to meet its primary endpoint. The results have led the company to discontinue further development of the drug and explore strategic alternatives.
The TrustTSC trial aimed to assess the efficacy and safety of oral ganaxolone in reducing the frequency of TSC (search)-associated seizures. The primary endpoint was the reduction in 28-day seizure frequency. Patients treated with ganaxolone experienced a median reduction of 19.7% in TSC-associated seizure frequency, compared to a 10.2% reduction in the placebo group. This difference did not reach statistical significance.
Impact and Future Plans
Following the disappointing results, Marinus Pharmaceuticals is taking immediate steps to reduce costs, including workforce reductions. The company has also engaged Barclays as an advisor to assist in exploring strategic alternatives. The failure of the TrustTSC trial represents a significant setback for Marinus, particularly given the unmet need for effective treatments for TSC (search)-associated seizures.
About Tuberous Sclerosis Complex
Tuberous sclerosis complex (search) (TSC (search)) is a rare genetic disorder that causes tumors to form in various organs, including the brain, skin, kidneys, heart, and lungs. Seizures are a common and often debilitating symptom of TSC, significantly impacting the quality of life for affected individuals and their families. Current treatment options for TSC-associated seizures are limited, highlighting the need for novel therapeutic approaches.