ZEVRA THERAPEUTICS, INC.
ZVRA evidence brief
What we can confirm
What it means
What to watch
Prepared research question
What matters most for ZVRA—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · ZVRA
Zevra Therapeutics is a commercial-stage rare-disease company with approved products MIPLYFFA and OLPRUVA, a late-stage pipeline including celiprolol and arimoclomol, and a focus on expanding patient access globally.
Key risk: Operating expense volatility
BioSniper Score
Based on 6 of 6 signals
Financial data as of Jun 30, 2026.
Tracking 6 pipeline candidates · 1 clinical trial · 4 catalysts · 1 FDA decision for ZEVRA THERAPEUTICS, INC..
Zevra Therapeutics is a commercial-stage rare-disease company with approved products MIPLYFFA and OLPRUVA, a late-stage pipeline including celiprolol and arimoclomol, and a focus on expanding patient access globally.
- Generated $106.5 million in net revenue for full-year 2025, including $87.4 million from MIPLYFFA [sec · filed 2026-04-20].
- Cash and equivalents of $145.3 million as of 2026-06-30 [sec].
- Net income of $8.8 million for the period ended 2026-06-30 [sec].
- Shareholders' equity of $154.7 million as of 2026-06-30 [sec].
- Submitted EU Marketing Authorization Application (MAA) for arimoclomol in July 2025 and advanced Phase 3 DiSCOVER trial of celiprolol with approximately one-third of patients enrolled as of year-end 2025 [sec · filed 2026-04-20].
What works
- Zevra has a commercial-stage product, MIPLYFFA, generating significant revenue ($87.4M in 2025) for Niemann-Pick disease type C, providing a strong corporate foundation [sec · filed 2026-04-20].
- The company has a late-stage pipeline with arimoclomol (EU MAA submitted) and celiprolol (Phase 3 DiSCOVER trial enrolling), indicating potential for future growth [sec · filed 2026-04-20].
- Zevra has a solid cash position of $145.3M as of 2026-06-30, providing financial flexibility to support operations and pipeline development [sec].
- The company reported net income of $8.8M for the period ended 2026-06-30, demonstrating a path to profitability [sec].
What to weigh
- Zevra expects to continue incurring significant expenses and minimal positive or negative net cash flows from operations for the near future, with fluctuations quarter-to-quarter [sec_mda · as of 2026-05-06].
- The company's cash deposits at financial institutions exceed insured limits, posing a risk in the event of a bank failure [sec_mda · as of 2025-11-05].
- Zevra cannot guarantee sufficient proceeds from potential funding sources (e.g., product sales, ATM offerings) to cover operating expenses [sec_mda · as of 2026-08-05].
- The company's pipeline assets (arimoclomol, celiprolol) are still in development or regulatory review, with no guarantee of approval or commercial success [sec_mda · as of 2026-08-05].
From the filings
Quoted directly from source documents.
Full-year 2025 net revenue of $106.5 million, with $87.4 million from MIPLYFFA
“For the full year, Zevra generated $106.5 million in net revenue, including $87.4 million from MIPLYFFA.”
SEC filings · April 20, 2026
EU MAA for arimoclomol submitted in July 2025
“We submitted the EU Marketing Authorization Application (MAA) for arimoclomol in July of 2025”
SEC filings · April 20, 2026
Phase 3 DiSCOVER trial of celiprolol approximately one-third enrolled as of year-end 2025
“we advanced our Phase 3 DiSCOVER trial of celiprolol, our late-stage development asset, with approximately one-third of patients enrolled as of year‑end 2025.”
SEC filings · April 20, 2026
Catalyst timeline
- 2031-02-22 · Patent_Expiry · APADAZ — Patent/exclusivity expiry for APADAZ; may affect competitive landscape for that product.
- 2031-09-20 · Patent_Expiry · MIPLYFFA — Patent/exclusivity expiry for MIPLYFFA; potential for generic competition post-expiry.
- 2036-10-17 · Patent_Expiry · OLPRUVA — Patent/exclusivity expiry for OLPRUVA; long-term exclusivity protection.
- 2037-12-09 · Patent_Expiry · AZSTARYS — Patent/exclusivity expiry for AZSTARYS; extended exclusivity period.
Risks & what to watch (4)›
Key risks
- Operating expense volatility — Zevra expects significant fluctuations in expenses and minimal positive net cash flows from operations for the near future [sec_mda · as of 2026-05-06].
- Cash deposit concentration risk — Majority of cash held at institutions exceeding insured limits; failure could delay access to funds [sec_mda · as of 2025-11-05].
- Funding uncertainty — No guarantee of sufficient proceeds from product sales, ATM offerings, or other sources to fund operations [sec_mda · as of 2026-08-05].
- Pipeline development risk — Arimoclomol and celiprolol are in regulatory review or clinical trials; no assurance of approval or commercial success [sec_mda · as of 2026-08-05].
What to watch
- Regulatory decision on EU MAA for arimoclomol, submitted July 2025 [sec · filed 2026-04-20].
- Enrollment progress and data readouts from Phase 3 DiSCOVER trial of celiprolol [sec · filed 2026-04-20].
- Quarterly revenue trends for MIPLYFFA and OLPRUVA, and any new distribution agreements [sec · filed 2026-04-20].
- Cash runway and any additional financing activities, given expected operating expense fluctuations [sec_mda · as of 2026-05-06].
Sources: SEC filings · SEC MD&A · Generated August 29, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 6 of 6 signals · derived from public data
Profitable (positive operating cash flow)
D/E 0.39 · 4% dilution
Lead: Approved
6 candidates
4 upcoming, next in ~1637d
0 buys · 4 sells (180d)
Catalysts
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
145
Total value held
$369.1M
| Holder | Shares | Value |
|---|---|---|
| vanguard capital management llc | 4,938,656 | $46.0M |
| blackrock, inc. | 4,456,476 | $41.5M |
| fmr llc | 3,211,877 | $29.9M |
| caption management, llc | 2,353,136 | $21.9M |
| state street corp | 1,561,569 | $14.6M |
| geode capital management, llc | 1,371,857 | $12.8M |
| morgan stanley | 1,192,262 | $11.1M |
| legal & general group plc | 1,163,090 | $10.8M |
| point72 asset management, l.p. | 1,157,131 | $10.8M |
| royce & associates lp | 988,674 | $9.2M |
Showing top 10 of 145 institutional holders of ZEVRA THERAPEUTICS, INC.. Open the full 13F history after sign-in.
Short Interest
as of Apr 28, 2026- % of float
- 8.2%
- Days to cover
- 5.9
- Shares short
- 4,776,072
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
ZVRA short interestGovernance
Frequently asked questions
What is the AI investment thesis for ZEVRA THERAPEUTICS, INC.?
Zevra Therapeutics is a commercial-stage rare-disease company with approved products MIPLYFFA and OLPRUVA, a late-stage pipeline including celiprolol and arimoclomol, and a focus on expanding patient access globally.
What is ZEVRA THERAPEUTICS, INC.'s lead drug candidate?
ZEVRA THERAPEUTICS, INC.'s most advanced tracked candidate is apadaz (Approved).
What is ZEVRA THERAPEUTICS, INC.'s next catalyst?
ZEVRA THERAPEUTICS, INC.'s next tracked catalyst is a Patent_Expiry event — "Patent/Exclusivity Expiry: APADAZ" — expected February 22, 2031.
Who is the CEO of ZEVRA THERAPEUTICS, INC.?
Mr. Neil F. McFarlane is the chief executive officer of ZEVRA THERAPEUTICS, INC. (ZVRA).
Where is ZEVRA THERAPEUTICS, INC. headquartered?
ZEVRA THERAPEUTICS, INC. (ZVRA) is headquartered in Boston, MA, United States.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.