Executive Summary

Q3 2025 net revenue was $26.1M, up ~606% YoY from $3.7M, led by MIPLYFFA® net revenue of $22.4M; GAAP diluted EPS was $(0.01) versus $(0.69) a year ago .
Versus consensus, revenue was slightly below ($26.06M actual vs $26.64M estimate; miss of ~$0.58M)* and EPS beat (−$0.01 actual vs −$0.034 estimate; beat of ~$$0.02); eight sell-side estimates supported the consensus.
Covered lives for MIPLYFFA reached 66% (52% in Q2; 38% in Q1), with strong reimbursement pull-through and shorter time-to-paid dispense; Medicare Part D rebate redesign drove a $1.2M gross-to-net true-up, impacting Q3 reported net sales .
Balance sheet strength remained notable: cash, cash equivalents, and investments were $230.4M, total debt ~$61.3M; management believes resources are sufficient to execute priorities independently of capital markets .
Near-term catalysts: EMA MAA for arimoclomol validated; first 120-day clock feedback expected before day 150; OLPRUVA promotion scaled back to focus resources on MIPLYFFA and patient services .

What Went Well and What Went Wrong

What Went Well

Commercial execution: “third quarter net revenue reached $26.1 million, driven by $22.4 million in net revenue for MIPLYFFA” .
Access expansion and execution: covered lives for MIPLYFFA increased to 66%, “in line with what we would expect one year into the launch,” with “high overall reimbursement rates…either through direct formulary coverage or…medical exception pathways” .
EU pathway advancing: “application has been validated and is under review by the EMA…highly confident in our submission,” supported by the largest NPC dataset and active EAP (92 patients) to seed launch .

What Went Wrong

Policy headwind: “Q3 MIPLYFFA net revenue was impacted by the redesign of Medicare Part D rebates…gross‑to‑net true‑up from prior quarters of $1.2 million,” a reduction to net sales that is now embedded in estimates going forward .
OLPRUVA underperformance: company “decided to scale back sales and marketing efforts for OLPRUVA” amid a “mature and well‑served UCD market” and prior quarter impairment/write-downs tied to slower adoption .
Non-cash volatility: warrant/CVR fair value expense of $5.5M and interest expense of $2.05M weighed on GAAP results despite operational improvement .

Financial Results

Quarterly P&L comparison (GAAP)

Metric	Q1 2025	Q2 2025	Q3 2025
Revenue, Net ($)	$20,401,000	$25,881,000	$26,063,000
MIPLYFFA Net Revenue ($)	$17,100,000	$21,500,000	$22,400,000
OLPRUVA Net Revenue ($)	$100,000	$300,000	$100,000
French EAP Net Reimbursements ($)	$2,300,000	$2,600,000	$2,400,000
AZSTARYS Royalties & Other ($)	$900,000	$1,200,000	$1,200,000
Cost of Product Revenue ($)	$1,345,000	$12,379,000	$1,238,000
Intangible Asset Amortization ($)	$1,615,000	$1,616,000	$315,000
Operating Expenses ($)	$22,803,000	$24,215,000	$20,367,000
R&D Expense ($)	$3,258,000	$3,433,000	$3,432,000
SG&A Expense ($)	$19,545,000	$20,782,000	$16,935,000
Income (Loss) from Operations ($)	$(5,362,000)	$(71,039,000)	$4,143,000
Net Income (Loss) ($)	$(3,099,000)	$74,707,000	$(544,000)
Diluted EPS ($)	$(0.06)	$1.21	$(0.01)

Notes: Q2 includes $148.3M PRV sale gain and OLPRUVA impairment/write‑down; Q3 includes $1.2M Medicare Part D gross‑to‑net true‑up and $5.5M warrant/CVR fair value expense .

YoY comparison (Q3 2025 vs Q3 2024)

Metric	Q3 2024	Q3 2025
Revenue, Net ($)	$3,695,000	$26,063,000
Cost of Product Revenue (ex‑amort.) ($)	$2,303,000	$1,238,000
Operating Expenses ($)	$27,153,000	$20,367,000
Net Loss ($)	$(33,225,000)	$(544,000)
Diluted EPS ($)	$(0.69)	$(0.01)

Q3 2025 Actual vs Consensus

Metric	Actual	Consensus	Surprise
Revenue ($)	$26,063,000	$26,641,880*	$(578,880)*
Primary EPS ($)	$(0.01)	$(0.03375)*	$0.02375*
Primary EPS – # of Estimates	—	8*	—
Revenue – # of Estimates	—	8*	—

Values retrieved from S&P Global.*

Segment/Source Breakdown (Q3 2025)

Source	Q3 2025 Amount ($)
MIPLYFFA Net Revenue	$22,400,000
OLPRUVA Net Revenue	$100,000
French EAP Net Reimbursements (arimoclomol)	$2,400,000
AZSTARYS Royalties & Other Reimbursements	$1,200,000
Total Net Revenue	$26,100,000

KPIs and Operating Metrics

KPI	Q1 2025	Q2 2025	Q3 2025
MIPLYFFA Prescription Enrollment Forms (New)	13	7	8
MIPLYFFA Total Enrollment Forms Since Launch	122	129	137
MIPLYFFA Covered Lives (%)	38%	52%	66%
OLPRUVA Covered Lives (%)	78%	79%	81%
Global EAP (arimoclomol) Patients	—	89	92
Celiprolol DiSCOVER Trial – Patients Enrolled	32	39	44
Cash, Cash Equivalents & Investments ($)	$68.7M	$217.7M	$230.4M
Total Debt ($)	~$60.1M	~$60.7M	~$61.3M
Shares Outstanding (Basic)	54,095,543	54,780,938	55,951,572

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Selling Expenses Outlook	FY 2025/near term	Not specified	“Meaningful change in selling expenses is not anticipated” despite reallocating OLPRUVA promotion	Maintained
OLPRUVA Commercial Strategy	Near term	Continued promotion	Sales & marketing scaled back; maintain availability/support while evaluating options	Lowered focus
Capital Needs	Near term	Not specified	Sufficient resources to execute priorities independent of capital markets	Maintained
EU MAA (Arimoclomol)	EMA process	Submission planned H2’25	MAA validated; 120-day clock underway; update expected in review timeline	Progressed

No formal numeric revenue/EPS guidance ranges were provided in Q3 materials .

Earnings Call Themes & Trends

Topic	Previous Mentions (Q1 & Q2)	Current Period (Q3 2025)	Trend
Market Access – MIPLYFFA Covered Lives	38% (Q1) ; 52% (Q2)	66%; high reimbursement via formulary or exceptions	Improving
Enrollment Forms – MIPLYFFA	13 (Q1) ; 7 (Q2)	8 new; 137 total since launch	Stable to improving
EU MAA Status	On track H2’25 filing (Q1) ; Submitted (Q2)	Validated; 120-day clock; confident in largest dataset	Advancing
OLPRUVA Strategy	Slow uptake; competitive shifts; coverage 79% (Q2)	Promotion scaled back; maintain access/support	Deprioritized
Medicare Part D Rebate Redesign	Not highlighted	$1.2M gross-to-net true-up; future estimates updated	New policy headwind embedded
Patient Identification Initiatives	Disease awareness; genetic testing (Q2)	Adds AI/ML models to find undiagnosed patients	Expanding tools
Celiprolol (VEDS) Enrollment	32 (Q1) ; 39 (Q2)	44 enrolled; 1 confirmed event; interim after 28 events	Progressing

Management Commentary

“Zevra is well‑positioned for continued growth, driven by the strong performance of MIPLYFFA…” .
“Q3 MIPLYFFA net revenue was impacted by the redesign of Medicare Part D rebates…gross‑to‑net true‑up…$1.2 million” .
“We increased the percent of covered lives to 66%…achieving high overall reimbursement rates…through direct formulary coverage or medical exception pathways” .
“Our Expanded Access Program…with 92 patients enrolled…lay[s] the groundwork for a potential commercial launch [in Europe]” .

Q&A Highlights

Newly diagnosed patient pull-through improving: disease awareness and genetic testing partnerships are identifying undiagnosed patients; majority of new forms still from previously diagnosed patients .
Reimbursement velocity: time from enrollment to paid dispense has compressed from months to weeks; some 24–72 hour turnarounds observed .
EU MAA timeline: first 120-day feedback; potential broader update closer to day 150; EAP conversion expected country-by-country with reimbursement .
Policy impact clarity: Medicare Part D redesign (catastrophic threshold and rebate increase to 20%) now embedded in gross‑to‑net assumptions .
Commercial focus: OLPRUVA promotion scaled back; resources prioritized to MIPLYFFA and patient services; selling expenses expected to be broadly stable .

Estimates Context

Q3 2025 revenue: actual $26.06M vs consensus $26.64M (miss ~$0.58M); Primary EPS: actual $(0.01) vs consensus $(0.0338) (beat ~$0.02); 8 estimates supported each consensus*.
Implications: modest top-line miss likely due to gross‑to‑net true-up; EPS beat driven by operating expense control and mix; consensus models should reflect updated Medicare Part D rebate rates and continued access gains*.