Q1 2026 Earnings — Reported May 6, 2026 · After Market Close
Revenue +78% YoY to $36.2M — Debt-Free · Cash $236.8M · EPS beats by 757%
Zevra delivered its strongest quarter since MIPLYFFA's commercial launch. Revenue of $36.2M beat the $31.96M consensus by 13.3%. GAAP EPS of $0.60 crushed the $0.07 estimate — driven by a $43.3M gain on the $50M SDX portfolio sale and full repayment of $63.1M in term debt. The company enters Q2 2026 with a clean, debt-free balance sheet and $236.8M in cash. MIPLYFFA reached 170 total prescription enrollment forms since launch, 9 added in Q1 alone.
Key Metrics — Q1 2026 Actuals
Net Revenue
$36.2M
+78% YoY
MIPLYFFA Revenue
$24.6M
vs $17.1M Q1 2025
GAAP Net Income
$37.9M
vs loss Q1 2025
GAAP EPS (diluted)
$0.60
vs −$0.06 Q1 2025
Adj. EPS (non-GAAP)
$0.18
vs −$0.06 Q1 2025
Cash & Equivalents
$236.8M
Debt-free
GAAP EPS Beat
+757%
$0.60 actual vs $0.07 consensus
Revenue Beat
+13.3%
$36.2M vs $31.96M est.
SDX Portfolio Sale
$50.0M
To Commave · $43.3M gain recorded
Term Loan Repaid
$63.1M
Fully prepaid · zero debt remaining
Beat / Miss Matrix
Beats
Net RevenueEst. $31.96M$36.2M (+13.3%)
GAAP EPSEst. $0.07$0.60 (+757%)
Adj. EPS (non-GAAP)Est. $0.06 (Zacks)$0.18 (+200%)
MIPLYFFA Revenuevs $17.1M Q1 2025$24.6M (+44% YoY)
Net IncomeExpected loss$37.9M (profitable)
Cash PositionPrior quarter level$236.8M · debt-free
Concerns
GAAP EPS Quality—$43.3M gain = one-time event
Operating Expenses—$25.2M (+10.5% YoY)
DiSCOVER Trial Events28 events neededOnly 2 events recorded
OLPRUVA Revenue—$0.3M — minimal traction
No FY Guidance Issued—Management did not give FY guide
EMA Decision Pending—EU approval timing uncertain
P&L Summary — Q1 2026 vs Q1 2025 (USD millions)
Select Income Statement
Net Revenue$36.2M$20.4M+78%
MIPLYFFA Revenue$24.6M$17.1M+44%
EAP Reimbursements$10.2M—Global
OLPRUVA Revenue$0.3M—Minimal
AZSTARYS Royalties$1.1M—Stable
Total Operating Expenses$25.2M~$22.8M+10.5%
SDX Portfolio Sale Gain$43.3M—One-time
GAAP Net Income$37.9M−lossSwing
GAAP EPS (diluted)$0.60−$0.06+1,100%
Adj. EPS (non-GAAP)$0.18−$0.06Profitable
Cash & Equivalents$236.8M—Zero debt
Product Revenue & CEO Quote
Revenue Breakdown — Q1 2026
MIPLYFFA (arimoclomol) U.S.$24.6M · 68% of revenue
Global EAP Reimbursements$10.2M · 28% of revenue
AZSTARYS Royalties$1.1M · 3% of revenue
OLPRUVA (urea cycle disorders)$0.3M · 1% of revenue
MIPLYFFA Rx forms (total)170 since launch (9 in Q1)
EAP enrolled patients122 internationally
U.S. reimbursement coverage~38% of covered lives (growing)
Balance Sheet & Corporate Actions
SDX portfolio sold to Commave$50.0M proceeds
Gain on sale recorded$43.3M (one-time)
Term loan fully repaid$63.1M — debt-free
Cash at quarter end$236.8M
Interest expenses (forward)$0 — eliminated
Fast Company recognitionTop 10 innovative pharma companies
"We made meaningful progress across the business in the first quarter, led by continued performance for MIPLYFFA, underscoring its role in addressing the needs of patients with Niemann-Pick disease type C. We also completed the $50.0 million divestiture of the SDX portfolio, and we repaid our term loan debt, sharpening our strategic focus and strengthening our financial position as we execute on our 2026 priorities."
Neil F. McFarlane, President & CEO · Q1 2026 Earnings Call, May 6, 2026
Pipeline & Clinical Update
Late-Stage Pipeline — Status as of Q1 2026
MIPLYFFA
Niemann-Pick disease type C (NPC) — arimoclomol + miglustat combo
FDA Approved
MIPLYFFA (EU)
NPC — EMA Marketing Authorization Application under review. 120-day questions responded to.
EMA Review
Celiprolol
Vascular Ehlers-Danlos Syndrome (VEDS) — DiSCOVER Phase 3 trial. 62 patients enrolled, 10 added Q1. Only 2 of 28 required events recorded. FDA Type C meeting planned H2 2026.
Phase 3
KP1077
Idiopathic hypersomnia & narcolepsy — Orphan designation. Phase 2 completed. Phase 3 dosing informed.
Ph2 Complete
OLPRUVA
Urea cycle disorders (UCD) — Commercially available U.S. Slow uptake. 28 Rx forms since 2023 launch.
Approved
Positives & Concerns
Positives
▲MIPLYFFA U.S. revenue of $24.6M — +44% YoY — demonstrates sustained commercial execution for the first and only FDA-approved NPC therapy. 170 total Rx forms since launch, 9 added in Q1 alone.
▲Company achieved profitability on an adjusted basis ($0.18 non-GAAP EPS) for the first time — a structural milestone reflecting operating leverage as MIPLYFFA scales without proportional cost increases.
▲$236.8M cash with zero debt after full $63.1M term loan repayment — eliminates all future interest expenses and significantly de-risks the balance sheet for pipeline investment and geographic expansion.
▲Global EAP growing to 122 enrolled patients across multiple regions — validates international demand ahead of potential EMA approval, which would open a substantial new revenue territory.
▲SDX portfolio divestiture for $50M to Commave Therapeutics sharpens strategic focus on high-value rare disease assets — eliminates distraction and adds capital for core programs.
Concerns
▼GAAP net income of $37.9M was almost entirely driven by the $43.3M non-cash SDX sale gain — a one-time event. Without this, Q1 would have reflected an operating loss. Underlying profitability remains limited.
▼DiSCOVER trial (celiprolol for VEDS) has accumulated only 2 of the 28 required events for interim analysis — event accrual is significantly slower than expected. Timeline to pivotal readout is highly uncertain.
▼OLPRUVA generated only $0.3M in Q1 — minimal commercial traction more than two years post-launch. The urea cycle disorder market is proving harder to penetrate than initially anticipated.
▼Management did not issue formal full-year 2026 revenue guidance. Analyst consensus of $146.2M for FY2026 implies revenue below the Q1 run rate for the remainder of the year — a notable deceleration assumption.
▼U.S. reimbursement coverage for MIPLYFFA stood at only ~38% of covered lives at the end of Q1 2025 — market access remains a structural barrier that limits the addressable patient pool for near-term revenue growth.
Analyst Coverage — Post Q1 2026
Wall Street Ratings
| Firm | Rating | Price Target | Note |
|---|
| High End Consensus | Buy | $26.00 | Top analyst target — rare disease ramp conviction |
| Consensus (avg.) | Buy | $23.00 | Simply Wall St consensus — broadly unchanged post Q1 |
| Low End Consensus | Buy | $18.00 | More conservative — event accrual risk in DiSCOVER |
| FY 2026 EPS Consensus | — | $1.01 | Raised from $0.519 post Q1 beat |
| FY 2026 Revenue Consensus | — | $147.6M | Raised from $143.3M; implies ~$37M/qtr avg. |
| Stock (30D post Q1) | +6% AMC | ~$10.77–11.05 | +6.03% aftermarket · PT range $18–$26 |
Earnings Verdict
Commercial Momentum Intact — Pipeline Timeline the Key Risk
Zevra delivered a clean beat on every operational metric in Q1 2026. MIPLYFFA continues to scale at a healthy pace, the balance sheet is now fortress-like with $236.8M cash and zero debt, and the company crossed into adjusted profitability for the first time — a structural milestone. The SDX divestiture was a strategically sound decision that sharpens focus and funds the pipeline. The GAAP headline numbers are flattering due to the one-time $43.3M gain, and investors should anchor on the $0.18 non-GAAP EPS as the true operational picture. The main overhang is the DiSCOVER trial — only 2 of 28 required events recorded creates deep uncertainty around the celiprolol readout timeline, which is potentially the most important binary catalyst for the stock. EMA review of MIPLYFFA in Europe is the next meaningful near-term catalyst. At a consensus PT of $23 vs the current ~$11 price, the implied upside is significant, but patience on DiSCOVER is required.
Next Catalyst
EMA Decision