MARINUS PHARMACEUTICALS, INC. (MRNS)·Q4 2023 Earnings Summary

Executive Summary

• Q4 2023 delivered continued commercial traction for ZTALMY: net product revenue grew to $6.6M (vs. $5.4M in Q3; $2.3M in Q4’22), total revenue $7.2M, and management introduced FY24 U.S. ZTALMY guidance of $32–$34M .
• Pipeline execution advanced: RAISE Phase 3 interim enrollment threshold achieved (83-patient interim set; DMC decision and topline outcome targeted for 1H Q2 2024), while TrustTSC Phase 3 enrollment ~85% with topline now expected 1H Q4 2024 (slipped from prior Q3 timing) .
• OpEx rose on clinical and API onshoring investments; Q4 net loss was $(41.8)M (EPS $(0.74)), with cash/short-term investments at $150.3M and runway projected into Q4 2024; CFO targets >$100M cash at RAISE readout .
• Potential stock catalyst: RAISE interim decision (co-primary endpoints for onset/durability; p≤0.0293 at interim) with secondary/HEOR readouts planned for the fall; management plans NDA in early Q1 2025 if positive .

What Went Well and What Went Wrong

• What Went Well

  • ZTALMY commercial momentum: Q4 net product revenue $6.6M; >165 patients active at year-end; payer approvals near 100%; time from enrollment to fill ~2 weeks in 2H’23 .
  • RAISE Phase 3 progress: interim enrollment achieved (>90 patients randomized, interim dataset of 83); interim outcome expected 1H Q2 2024; database to ~100 patients supports robust HEOR .
  • Clear hospital-launch groundwork for IV ganaxolone and franchise-expansion strategy (RAISE II EU study; SRSE PoC planning) .
  • Quote: “We expect to deliver the interim results… and plan to announce the outcome within the first half of the second quarter [of 2024].” – CEO .

• What Went Wrong

  • TrustTSC topline delayed to first half of Q4 2024 from prior expectations (had been Q3 2024), reflecting minor screening/enrollment timing .
  • Continued operating losses driven by R&D scale-up; Q4 loss from operations $(35.4)M; net loss $(41.8)M; cash down to $150.3M at year-end (from $176.4M Q3) .
  • No formal EPS/Revenue consensus comparison disclosed; S&P Global consensus could not be retrieved via our tool this cycle (see Estimates Context) [GetEstimates attempt failed].
  • Analyst concern: trial execution/statistics (stopping criteria; placebo rates/power) required detailed clarification in Q&A, underscoring complexity of the RAISE dataset .

Financial Results

• Income statement comparison (USD Millions unless stated)

• Additional Q4 vs year-ago context: product revenue rose to $6.6M from $2.3M in Q4’22; BARDA revenue declined to $0.6M from $1.8M .

• Revenue breakdown (mix) is driven by ZTALMY U.S. sales plus BARDA contract revenue; no separate segments reported .

• Commercial KPIs

Guidance Changes

Earnings Call Themes & Trends

Management Commentary

• Strategic priorities: “2024 will be a pivotal year… CDD, RSE and TSC represent a multibillion-dollar opportunity” – CEO .
• RAISE interim details: co-primaries at p=0.0293 interim threshold; power >90% to detect 40% delta; minimum delta for significance ~19–27% .
• Commercial profitability: “Expect to achieve profitability on our ZTALMY commercial investment by the second quarter of 2024” – CEO .
• Hospital launch prep: NTAP strategy, formulary targeting, HEOR focus for IV ganaxolone .
• Cash/OpEx stance: runway into Q4’24; >$100M projected at RAISE readout; 2024 OpEx to depend on trial outcomes .

Q&A Highlights

• Reimbursement learnings for TSC: Positive CDD payer experience informs TSC expectations; payers recognize high unmet needs; strong prior-authorization outcomes .
• RAISE stopping rules and power: Both co-primaries must hit; p=0.0293 at interim; 94% power for 40% delta; significance possible at ~25–30% deltas .
• If interim doesn’t stop: Company will update the Street and continue blinded enrollment; data cleaning emphasized .
• SRSE dosing/label path: Pursuing higher-dose regimen safety via single-arm PoC; eventual goal to reflect safety in label subject to FDA discussions .
• Off-label dynamics: ~10% of ZTALMY sales from spontaneous refractory epilepsy use; ~two-thirds of such PA forms reimbursed; company focuses promotion on on-label CDD .

Estimates Context

• We attempted to pull S&P Global consensus for Q2–Q4 2023 EPS and revenue (Primary EPS Consensus Mean; Revenue Consensus Mean), but the CIQ mapping for MRNS was unavailable via our tool; therefore, we cannot present a definitive vs-consensus comparison for Q4 2023 at this time. Values would normally be retrieved from S&P Global; consensus was unavailable via our data connector during this analysis.
• The company did not provide a formal consensus comparison in its materials. Our tables therefore benchmark sequential and year-over-year trends using reported figures .

Key Takeaways for Investors

• Sequential commercial execution is intact: ZTALMY revenue rose to $6.6M in Q4, with FY24 guidance implying ~63–73% YoY growth (to $32–$34M) if achieved .
• The near-term stock driver is the RAISE interim DMC outcome (1H Q2’24); success accelerates NDA (targeted early Q1’25) and validates hospital launch plans, while secondary/HEOR endpoints in the summer/fall frame reimbursement/distribution arguments .
• TrustTSC delay modestly shifts the sNDA and launch curve to 1H 2025; low discontinuation (<7%) and OLE rollover (>85%) support data quality .
• Balance sheet supports catalysts (runway into Q4’24; >$100M expected at readout), but financing risk rises if interim or subsequent milestones underwhelm .
• SRSE pathway offers upside optionality (single-arm PoC; strong eIND signals) and may broaden the IV ganaxolone value proposition beyond RSE .
• Global partners (EU/China/MENA) can add ex-U.S. royalty/launch leverage over time, complementing U.S. growth .
• Execution check-points: DMC outcome (1H Q2’24), summer/fall secondary/HEOR detail, TrustTSC topline (1H Q4’24), and evolving 2024 OpEx as readouts crystallize .

Sources: Q4’23 8-K/press release, financial tables and corporate deck ; Q4’23 earnings call transcript ; Q3’23 8-K/press release and transcript ; Jan 4, 2024 preliminary revenue/guidance 8-K ; Q2’23 8-K and transcript .