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ZEVRA THERAPEUTICS, INC. (ZVRA)·Q4 2024 Earnings Summary

Executive Summary

  • Q4 2024 net revenue was $12.0M, driven by MIPLYFFA’s early U.S. launch ($10.1M), OLPRUVA ($0.1M), French EAP reimbursements ($1.1M), and AZSTARYS royalties ($0.7M). EPS was ($0.67), and operating expenses were $24.5M .
  • Management highlighted a “transformational year,” strong MIPLYFFA demand (109 enrollment forms by Dec 31), and rapid conversion of U.S. EAP patients in one quarter versus typical 12 months .
  • Guidance improved: cash runway extended into 2029 and “no Going Concern” disclosure; PRV sale agreement for $150M expected to yield ~$148.3M net proceeds at closing, taking pro forma cash/investments to ~$223.8M .
  • Near-term catalysts: ongoing payer coverage work for MIPLYFFA, U.S. EAP closure by end of Q2 2025, EU MAA targeted for H2 2025, and continued DiSCOVER Phase 3 enrollment for celiprolol (27 patients) .

What Went Well and What Went Wrong

What Went Well

  • Rapid MIPLYFFA launch momentum: 109 enrollment forms received through Dec 31; all active U.S. EAP patients enrolled by year-end. “The early days of the MIPLYFFA launch have exceeded our expectations” .
  • Record quarterly product revenue with $10.1M MIPLYFFA net revenue in Q4 (product available for last 5 weeks), including initial dispenses and refills for early enrollees .
  • Balance sheet strength and visibility: PRV sale agreement for $150M with expected ~$148.3M net proceeds; extended runway into 2029 and removal of Going Concern disclosure .

What Went Wrong

  • Q4 EPS ($0.67) and net loss ($35.7M) widened YoY, reflecting higher SG&A from full commercial build and launch activities; SG&A rose to $16.1M vs $14.7M in Q4 2023 .
  • OLPRUVA uptake slower than desired; only 4 new enrollments in Q4 despite 76% covered lives; strategy being refined toward adult segments to mitigate reimbursement hurdles .
  • French EAP reimbursements decreased sequentially due to a true-up in program access fees, and inventory obsolescence reserves impacted cost of goods in prior quarters .

Financial Results

Sequential performance (Q2–Q4 2024)

MetricQ2 2024Q3 2024Q4 2024
Revenue, Net ($USD Millions)$4.449 $3.695 $12.0
Cost of Goods Sold ($USD Millions)$3.573 cost of product + $1.546 intangible amortization $2.303 cost of product + $1.545 intangible amortization $3.0 (incl. $1.6 intangible amortization)
Operating Expense ($USD Millions)$23.125 $27.153 $24.5
Net Loss ($USD Millions)($19.925) ($33.225) ($35.7)
Diluted EPS ($USD)($0.48) ($0.69) ($0.67)

Notes: Q2 and Q3 statements present cost of product revenue and intangible amortization separately; Q4 “COGS” includes intangible amortization .

Year-over-year (Q4 2023 vs Q4 2024)

MetricQ4 2023Q4 2024
Revenue, Net ($USD Millions)$13.2 (incl. $10M one-time AZSTARYS milestone) $12.0
Net Loss ($USD Millions)($19.6) ($35.7)
Diluted EPS ($USD)($0.51) ($0.67)
Cost of Goods Sold ($USD Millions)Not disclosed in press release; see Q4 2023 true-up comment $3.0 (incl. $1.6 intangible amortization)

Q4 2024 revenue components

ComponentQ4 2024 ($USD Millions)
MIPLYFFA Net Revenue$10.1
OLPRUVA Net Revenue$0.1
French EAP Reimbursements (arimoclomol)$1.1
AZSTARYS Royalties/Other Reimbursements$0.7
Total Net Revenue$12.0

Commercial KPIs

KPIQ2 2024Q3 2024Q4 2024
MIPLYFFA Enrollment Forms (cumulative period-end)NDA pending; not launched90 as of Oct 31; ~30% approved/reimbursable 109 as of Dec 31; active U.S. EAP patients enrolled
OLPRUVA New Patient Enrollments (quarter)9 3 4
OLPRUVA Covered Lives (%)~75% Not updated~76%

Guidance Changes

MetricPeriodPrevious GuidanceCurrent GuidanceChange
Cash RunwayMulti-yearInto 2027 (Q3 guidance, subject to covenants) Into 2029; Form 10-K to exclude Going Concern disclosure Raised runway and removed going concern
PRV MonetizationNear-termPotential to monetize PRV (post-approval) Asset purchase agreement signed; $150M gross, ~$148.3M net expected; pro forma cash/investments ~$223.8M New/confirmed transaction details
MIPLYFFA EU FilingRegulatoryNot specified in Q2; planning in Q3 EU MAA targeted H2 2025 New specific timeline
U.S. EAP (NPC)ProgramEAP active / transition expected over ~12 monthsU.S. EAP anticipated to close by end of Q2 2025 New/accelerated closure timeline
French EAP Net ReimbursementsQuarterlyNot quantified~$2.1M per quarter expected (normalized post true-up) New numeric guidance

Earnings Call Themes & Trends

TopicPrevious Mentions (Q2 2024, Q3 2024)Current Period (Q4 2024)Trend
MIPLYFFA Launch & DemandQ2: Advisory committee favorable vote; launch readiness · Q3: 90 enrollment forms; ~30% approved prior to availability 109 enrollment forms by Dec 31; rapid EAP conversion in one quarter; payer engagement ongoing Strong acceleration; broadening beyond centers of excellence
Payer Coverage & ReimbursementQ3: Initial approvals (~30%); payer policy formation underway Most patients reimbursed via formulary or medical exception; coverage still formalizing; net pricing commentary conservative Improving approvals with ongoing payer dialogues
OLPRUVA StrategyQ2: Transition to Orsini; 75% covered lives; 9 enrollments · Q3: 3 enrollments; focus on adult segments 4 enrollments; 76% covered lives; sharpened targeting and field reimbursement support Gradual build; strategy refinement underway
Celiprolol (VEDS) DiSCOVER TrialQ2: Recruitment restarted · Q3: 19 patients enrolled +8 in Q4 to 27 total; tactics to accelerate enrollment Enrollment ramping; event-driven focus
KP1077 (IH/Narcolepsy)Q2: Phase 2 positive topline; EoP2 meeting planned Exploring strategic alternatives; Phase 3–ready asset Monetization/partnering focus
EU ExpansionQ2/Q3: Planning for ex-U.S. access; EMA orphan status EU MAA targeted for H2 2025; 1,100 prevalent patients estimated Concrete timeline; ex-U.S. growth vector
Patient Identification InitiativesQ2/Q3: AmplifyAssist support programs Media/genetic testing campaigns; 3 newly identified patients via awareness efforts Early wins; scalable HCP outreach

Management Commentary

  • “2024 was a transformational year for Zevra…The early days of the MIPLYFFA launch have exceeded our expectations.” — Neil F. McFarlane, President & CEO .
  • “This was a record level of quarterly product revenue for Zevra.” — R. LaDuane Clifton, CFO, on Q4 product revenue dynamics and French EAP normalization .
  • “We expect to receive net proceeds of approximately $148.3 million…our pro forma balance of cash, cash equivalents and investments will be approximately $223.8 million…our current operating forecast…extends our cash runway into 2029.” — R. LaDuane Clifton .

Q&A Highlights

  • EU Filing: EMA MAA planned for H2 2025; prior EMA feedback being addressed with long-term data and consultancy support; Europe seen as more mature given miglustat and broader diagnosed base .
  • Launch Trajectory: After rapid EAP conversion, focus shifts to diagnosed-but-untreated and newly diagnosed patients; management views momentum carrying into Q1/Q2 2025 .
  • Payer Dynamics: Most patients get reimbursement via formulary or medical exceptions; approvals improving; limited denials addressed via appeals and assistance programs .
  • Discontinuation: Too early to model; historical EAP/OLE experience suggests high continuity on MIPLYFFA + miglustat over multiple years .

Estimates Context

  • We attempted to retrieve S&P Global consensus for Q4 2024 revenue and EPS; data was unavailable due to request limits. As a result, consensus comparisons could not be made. We searched for: “Primary EPS Consensus Mean,” “Revenue Consensus Mean,” “# of Estimates” for Q4 2024 using GetEstimates; SPGI returned Daily Request Limit exceeded errors. If provided, we would anchor all comparisons on S&P Global consensus.
  • Implication: Without published consensus in this recap, we cannot formally classify Q4 as an estimate beat/miss.

Key Takeaways for Investors

  • MIPLYFFA’s launch showed strong early adoption with 109 enrollment forms and rapid EAP conversion, underpinning Q4 product revenue leadership ($10.1M) despite limited weeks of availability .
  • Cash runway extended into 2029 and “no Going Concern” disclosure signals improved financial durability; PRV monetization (~$148.3M net) adds non-dilutive capital for commercial and pipeline execution .
  • Near-term stock catalysts: payer policy formalization for MIPLYFFA, U.S. EAP closure (end of Q2 2025), EU MAA submission (H2 2025), and continued DiSCOVER enrollment progress .
  • OLPRUVA remains a strategic lever with targeted adult-segment focus and 76% covered lives; execution improvements (specialty pharmacy, field reimbursement) aim to enhance pull-through .
  • French EAP reimbursements expected to normalize at ~$2.1M per quarter, adding a recurring contribution alongside AZSTARYS royalties .
  • With payer engagements highlighting MIPLYFFA’s disease-modifying data and preference for multi-therapeutic NPC approaches, coverage should improve, supporting continued revenue ramp through 2025 .
  • Pipeline optionality (celiprolol VEDS Phase 3, KP1077 strategic alternatives) offers medium-term upside while commercial pillars drive near-term revenues .

Appendix: Additional Financial Detail (FY context)

  • FY 2024 net revenue $23.6M; net loss ($105.5M), EPS ($2.28); cash, cash equivalents, and investments $75.5M at Dec 31, 2024; shares outstanding 53.67M .
  • Q3 2024: net revenue $3.7M; net loss ($33.2M), EPS ($0.69); cash/cash equivalents/investments $95.5M .
  • Q2 2024: net revenue $4.449M; net loss ($19.925M), EPS ($0.48) .

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