Marinus Pharmaceuticals (MRNS) Investor Presentation summary

Product and clinical overview

• Ganaxolone is a neuroactive steroid that modulates both synaptic and extrasynaptic GABA receptors, offering a differentiated mechanism of action for epilepsy treatment.

• ZTALMY (ganaxolone) is FDA-approved for CDKL5 deficiency disorder (CDD) and has a well-characterized safety profile, addressing a significant unmet need.

• In the Phase 3 Marigold trial, ganaxolone reduced major motor seizure frequency by a median of 30.7% versus 6.9% for placebo; sustained reductions of ~50% were observed over two years in the open-label extension.

• The most frequent adverse events were somnolence, pyrexia, and upper respiratory tract infection, with safety findings consistent across studies.

Commercial performance and strategy

• Net product revenue for ZTALMY reached $8.5M in Q3 2024, a 56% increase from Q3 2023, with full-year revenue expected at $33–34M.

• Achieved profitability on ZTALMY commercial investment in Q1 2024, ahead of target, driven by strong patient enrollments and prescriber growth.

• Commercial strategy includes focused education for healthcare providers, enhanced patient support, and increased investment in patient identification.

• Global access is expanding through partnerships in Europe (Orion), China (Tenacia), MENA (Biologix), and a distribution agreement with Uniphar Group.

Clinical pipeline and trial results

• In the Phase 3 TrustTSC trial for TSC-associated seizures, ganaxolone showed a 19.7% reduction in seizure frequency versus 10.2% for placebo; the primary endpoint was not statistically significant (p=0.0904).

• The RAISE Phase 3 trial in refractory status epilepticus (SE) showed IV ganaxolone led to SE cessation within 30 minutes in 80% of patients versus 13% for placebo (p<0.0001), with similar rates of serious adverse events.

• Median time to SE cessation was 4.2 minutes for IV ganaxolone versus 307.2 minutes for placebo; EEG seizure burden reduction was 93% for ganaxolone versus 36% for placebo (p=0.003).