Executive Summary

Q1 2025 delivered a clean top- and bottom-line beat: net revenue $20.40M vs S&P Global consensus $16.96M* and EPS ($0.06) vs ($0.19)*, driven by $17.1M from MIPLYFFA and continued French EAP reimbursements ($2.3M).
Sequential momentum continued post-launch: product revenue scaled from $10.1M in Q4 (first commercial quarter for MIPLYFFA) to $17.1M in Q1; total net revenue rose ~70% q/q and ~6x y/y, while loss per share improved to ($0.06) from ($0.67) in Q4 and ($0.40) in Q1’24.
Balance sheet significantly strengthened: $68.7M cash/equivalents/investments at 3/31/25; PRV sale closed for $150M gross ($148.3M net) on 4/1/25, implying pro forma liquidity of ~$217.0M; debt ~ $60.1M.
Commercial KPIs: 122 cumulative MIPLYFFA prescription enrollment forms through Q1 (13 in Q1); payer coverage at 38% of covered lives; OLPRUVA coverage at 78% (5 new enrollments in Q1). EU MAA for MIPLYFFA remains on track for 2H 2025.
Near-term stock drivers: pace of MIPLYFFA payer policy formalization and pull-through, sustained new patient adds and refills, EU MAA filing/milestones, and VEDS (celiprolol) Phase 3 enrollment acceleration to de-risk development timelines.

What Went Well and What Went Wrong

What Went Well

Strong launch execution and revenue mix: MIPLYFFA contributed $17.1M; total net revenue $20.4M beat consensus, with majority of enrolled patients already on therapy and refilling. “The launch is progressing well and exceeding our expectations.”
Balance sheet de-risked with PRV sale: closed for $150M gross ($148.3M net), taking pro forma cash to ~$217.0M, enabling execution “independent from the capital markets.”
Clinical and pipeline progress: Celiprolol DiSCOVER enrollment increased to 32; EU MAA for MIPLYFFA remains on track for 2H 2025; out-licensed deprioritized preclinical IP.

What Went Wrong

Gross margin still weighed by non-cash amortization; operating margin negative: Q1 cost of goods sold included $1.6M intangible amortization; EBIT margin about -26% (improved but still negative).
SG&A elevated to support launches: Q1 SG&A $19.5M, up $9.6M y/y, reflecting personnel and commercial activity; operating expense base requires scaling revenue to reach breakeven.
OLPRUVA remains slow: only $0.1M net revenue and 5 enrollments in Q1 despite repositioning; management continues payer/formulary work to improve pull-through.

Financial Results

Headline P&L vs prior periods and estimates

Metric	Q3 2024	Q4 2024	Q1 2025
Net Revenue ($M)	$3.70	$12.04	$20.40
Diluted EPS ($)	($0.69)	($0.67)	($0.06)
Loss from Operations (EBIT) ($M)	($27.31)	($15.42)	($5.36)
EBIT Margin (%)	(calc) ~ -739%	(calc) ~ -128%	(calc) ~ -26.3%
Consensus Revenue ($M)*	—	—	$16.96*
Consensus EPS ($)*	—	—	($0.19)*

Notes: EBIT Margin is derived from loss from operations / revenue. Consensus estimates marked with asterisks are Values retrieved from S&P Global.

Q1 2025 vs expectations: Revenue beat by ~$3.44M (~20%); EPS beat by ~$0.13. Q1 y/y: revenue up from $3.43M to $20.40M; EPS improved from ($0.40) to ($0.06).

Revenue composition (by source)

Revenue Component ($M)	Q3 2024	Q4 2024	Q1 2025
MIPLYFFA Net Revenue	—	$10.10	$17.10
OLPRUVA Net Revenue	de minimis	$0.10	$0.10
French EAP (Arimoclomol)	$2.60	$1.10	$2.30
AZSTARYS Royalties/Other	$1.10	$0.70	$0.90
Total Net Revenue	$3.70	$12.04	$20.40

Operating expenses and key line items

Line Item ($M)	Q3 2024	Q4 2024	Q1 2025
Cost of Product Revenue (excl. amort.)	$2.30	$1.37 FY context; Q4 CoGS $3.0 incl amort	$1.35
Intangible Amortization	$1.55	$1.60	$1.62
R&D Expense	$10.95	$8.40	$3.26
SG&A Expense	$16.21	$16.10	$19.55
Loss from Operations	($27.31)	($15.42)	($5.36)

Balance sheet and liquidity

Cash, cash equivalents and investments: $68.7M at 3/31/25; pro forma $217.0M including PRV sale net proceeds received 4/1/25; Long-term debt ~$60.1M.
Shares outstanding: 54.68M basic; fully diluted 67.90M as of 3/31/25.

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
MIPLYFFA EU MAA filing	2025	Target 2H 2025 (Q4 remarks)	On track for 2H 2025	Maintained
Capital position/runway	2025+	No Going Concern; runway into 2029 (10-K context)	“Ample resources… independent from capital markets” (no numeric runway update)	Qualitatively maintained
Quantitative financial guidance (rev/EPS/OpEx)	2025	None provided	None provided	Unchanged
French EAP reimbursements	Near-term	~ $2.1M/quarter (Q4 call)	Not updated in Q1 call	N/A

Earnings Call Themes & Trends

Topic	Previous Mentions (Q3 2024, Q4 2024)	Current Period (Q1 2025)	Trend
MIPLYFFA launch metrics & payer access	90 enrollments by 10/31; ~30% approvals; initial shipment in Q4; payer policies pending	122 cumulative enrollments; 13 in Q1; 38% covered lives; majority of enrolled “on drug” with refills; payer access via formulary or medical exception	Improving coverage and patient pull-through
OLPRUVA repositioning	Strategy refined; 3 enrollments in Q3; 76% coverage	5 enrollments in Q1; coverage 78%; targeting adult/OTC-deficient segment	Gradual progress; still small base
EU MAA (MIPLYFFA)	Top priority; planning	2H 2025 filing “on track” reinforced	Maintained timeline
PRV monetization	Intent to monetize; PRV market pricing discussed	Agreement signed (Q4); closed $150M gross (Q1)	Achieved; bolstered liquidity
Celiprolol (VEDS) Phase 3	19 enrolled (Q3)	27 enrolled (Q4) → 32 (Q1); genetic testing & center outreach to accelerate	Steadily improving enrollment
KP1077 strategy	Exploring strategic alternatives (Q3)	Reiterated value-maximizing alternatives	Ongoing
Reimbursement dynamics	Early denials typical; medical exceptions effective (Q4)	Similar tone; broad success via formulary or exceptions; coverage building	Stable/Improving

Management Commentary

“We have exceeded our internal expectations for the MIPLYFFA launch and have enrolled over 1/3 of those diagnosed with Niemann-Pick Disease Type C or NPC in the United States.” — CEO Neil McFarlane
“Combined with the net proceeds of $148.3 million from the sale of the PRV… cash, cash equivalents and investments would be $217.0 million… ample resources… independent from the capital markets.” — CFO LaDuane Clifton
“Through the end of the first quarter, we have achieved 38% of covered lives… We have been able to secure reimbursement authorization for many of our patients through direct formulary coverage or via the medical exception process.” — CCO Josh Schafer
“Filing of the MIPLYFFA MAA with the European Medicines Agency remains on track for the second half of 2025.” — Company statement

Q&A Highlights

Reimbursement/coverage: Management emphasized early denials are typical but are largely overcome via medical exception; 38% covered lives achieved; majority of enrolled patients are already on drug, with high refill rates so far.
Cadence of new patient starts: No formal guidance; focus shifted from EAP conversion to diagnosed and undiagnosed cohorts; inventory maintained at target days on hand.
Net pricing/mix: Commercial dosing distribution mirrors EAP (more adult/heavier-weight dosing), supporting previously communicated average pricing assumptions.
Celiprolol timeline: Enrollment increased to 32; strategies (genetic testing, COL3A1 centers) expected to accelerate adds, but no specific completion timing provided.

Estimates Context

Q1 2025 actuals vs S&P Global consensus: Revenue $20.40M vs $16.96M*; EPS ($0.06) vs ($0.19)*; 7 estimates for both revenue and EPS. Beat driven by MIPLYFFA uptake and French EAP reimbursements.
Implications: Consensus likely to move higher on MIPLYFFA trajectory and payer access progress; watch for updates on coverage policy formalizations and EU catalysts.
Values retrieved from S&P Global.

Key Takeaways for Investors

MIPLYFFA is scaling faster than modeled: strong q/q product revenue build, majority of enrolled patients on therapy with refills, and 38% coverage provides a path to sustained pull-through.
Quality of beat matters: revenue and EPS beats were operational (commercial traction), not solely driven by one-time items; operating leverage improved as EBIT margin tightened to ~-26% from ~-128% in Q4.
Balance sheet a competitive advantage: PRV proceeds drive pro forma cash to ~$217M vs ~$60M debt, funding launches and pipeline without near-term equity needs.
Pipeline diversification: celiprolol enrollment progress de-risks VEDS program; EU MAA for MIPLYFFA in 2H 2025 offers ex-US optionality/catalysts.
OLPRUVA remains “prove-it”: modest contribution persists; management targeting specific adult/OTC segments and payer work—assume limited near-term revenue until evidence of inflection.
Watch the payer narrative: further increases in covered lives, speed of medical exception approvals, and refill persistence are key leading indicators for revenue durability.
Near-term setup: Continued commercial momentum into Q2, EU MAA progress, and updates on VEDS enrollment cadence are likely stock catalysts.

Appendix: Additional Detail

Q1 2025 revenue breakdown: MIPLYFFA $17.1M; OLPRUVA $0.1M; French EAP $2.3M; AZSTARYS $0.9M.
Non-cash items: Q1 COGS included $1.615M intangible amortization; stock-based comp within OpEx was $3.1M.
Share count: 54,679,363 basic; 67,897,631 fully diluted at 3/31/25.
Cash and debt: $68.7M cash/equivalents/investments at 3/31/25; pro forma $217.0M including PRV net; long-term debt ~$60.1M.

ZEVRA THERAPEUTICS, INC. (ZVRA)·Q1 2025 Earnings Summary