Shares of Marinus Pharmaceuticals, Inc. (MRNS) plummeted on Thursday after the company unveiled disappointing results from its Phase 3 TrustTSC trial for oral ganaxolone. The trial, designed to evaluate the efficacy of ganaxolone for treating seizures associated with tuberous sclerosis complex (TSC) in children and adults, failed to reach its primary endpoint.
Tuberous sclerosis complex is a rare genetic disorder that causes non-cancerous tumors to develop in various parts of the body. The TrustTSC trial, the first of its kind to enroll patients already taking other anti-seizure medications, including mTOR inhibitors and cannabidiol, aimed to assess the efficacy of ganaxolone in conjunction with these standard-of-care treatments.
While the trial demonstrated a median reduction in seizure frequency of 19.7% for ganaxolone compared to 10.2% for placebo, this difference did not reach statistical significance. This outcome indicates that the drug’s effect on seizure reduction, although observed, was not sufficiently pronounced to meet the trial’s primary endpoint.
Despite the disappointing outcome, ganaxolone was generally well-tolerated with a safety profile consistent with previous clinical trials. The most frequent adverse event was somnolence, occurring in 28.1% of patients receiving ganaxolone and 16.9% of those receiving placebo.
In light of these results, Marinus has made the decision to discontinue further clinical development of ganaxolone and implement cost-cutting measures, including workforce reductions. The company has also initiated a process to explore strategic alternatives and has engaged Barclays as an advisor to assist in reviewing its strategic options.
The news of the TrustTSC trial’s failure has sent shockwaves through the market, resulting in a significant decline in MRNS stock. As of Thursday’s closing bell, the stock had fallen by 82.80% to $0.29.
This setback underscores the inherent risks associated with clinical trials and the unpredictable nature of drug development. Investors and stakeholders will be closely watching Marinus’s strategic decisions as the company navigates this challenging period.