Monday, in an interim analysis of Marinus Pharmaceuticals Inc's (NASDAQ:MRNS) phase 3 RAISE trial, evaluating intravenous (IV) ganaxolone as a treatment for refractory status epilepticus (RSE), failed to meet pre-defined "stopping criteria."
Marinus has completed RAISE enrollment at approximately 100 patients, with topline results expected in the summer of 2024.
The company said the future development of IV ganaxolone in refractory status epilepticus will be assessed following a review of the final RAISE study results.
An independent Data Monitoring Committee (DMC) recommended continuing the study.
Cantor Fitzgerald said it shifted focus to oral ganaxolone (O-GNX), which is progressing in the global Phase 3 TrustTSC study for tuberous sclerosis complex (TSC), and to a second-generation ganaxolone (2G-O-GNX) formulation aimed at enhancing safety, efficacy, and tolerability, potentially enabling less frequent dosing.
The analyst anticipates that further data analysis, once the study is complete, may provide clarity on statistical significance, but the potential commercial viability of IVGNX may be the "trump card" decision driver.
The analyst maintains the Overweight rating with a reduced price target of $13 from $28.
RBC Capital Markets says the IV ganaxolone study update is disappointing and somewhat surprising given prior data and a careful study design albeit in a challenging indication.
That said, capital constraints are placing more pressure on the upcoming Phase 3 TSC readout, and RBC views that the TSC study is a higher risk and <50/50 to hit; it is difficult to recommend buying ahead of that.
RBC downgraded to Sector Perform from Outperform, with a price target of $3, down from a prior target of $24.
Price Action: MRNS shares are down 8.07% at $1.1950 on the last check Tuesday.
Photo via Darko Stojanovic from Pixabay