Zevra Therapeutics, Inc. (NASDAQ: ZVRA) Q2 2025 Earnings | 08/13/2025

7 days ago

51:55

Inside Ticker

Earnings Calls

AI Summary

Key Highlights:

1. Financial Performance

Q2 2025 Net Revenue: $25.9M
- MyKliefa (NPC treatment): $21.5M (+26% QoQ growth)
- Olpruva (urea cycle disorders): $300K (slow uptake, $58.7M non-cash impairment charge)
- Other: $2.6M (French EAP for arimoclomol), $1.2M (royalties), $300K (Dextropin licensing)
Cash Position: $217.7M (up from $68.7M in Q1), bolstered by $150M PRV sale in April.
Adjusted Net Loss: $3.2M ($0.06 per share) excluding one-time charges.

2. MyKliefa (Niemann-Pick Disease Type C – NPC) Launch Update

Strong U.S. Launch:
- 129 total prescription enrollments (7 new in Q2), reaching ~1/3 of diagnosed U.S. patients (300-350).
- Revenue Growth: $21.5M in Q2, up 26% from Q1.
- Patient Segments Focus:
  1. EAP conversions (long-term patients, high retention).
  2. Diagnosed but untreated (engaging broader prescribers beyond centers of excellence).
  3. Undiagnosed patients (awareness campaigns, genetic testing initiatives).
- Payer Coverage: 52% covered lives, with exceptions for others.
European Expansion:
- MAA submitted to EMA (ahead of schedule), targeting ~1,100 NPC patients in Europe.
- EAP program expanded to 89 patients (up from 70-80 last year).
New Data:
- Published 48-month open-label extension data showing sustained efficacy/safety.
- Mechanistic data supports CLEAR network activation, targeting NPC pathology.

3. Olpruva (Urea Cycle Disorders) Challenges

Slow Adoption: Only 1 prescription in Q2 due to market maturity and competition.
Strategic Shift: Focused on niche positioning, reimbursement (79% coverage), but cautious outlook.

4. Pipeline Updates

Solipulol (VEDS – Phase 3 DISCOVER Trial):
- 39 patients enrolled (of 150 planned), with genetic testing initiatives accelerating recruitment.
Arimoclomol (Europe MAA submitted).

5. Outlook & Strategy

Priorities:
- Drive MyKliefa adoption (U.S. & Europe).
- Advance Solipulol and arimoclomol regulatory progress.
- Maintain strong balance sheet ($217.7M cash).
Long-Term Vision: Build a leading rare disease company through commercial execution, pipeline innovation, and patient-centric focus.

Q&A Highlights:

MyKliefa Trends: Early prescribers include neurologists, pediatricians, and geneticists.
Europe EAP Conversion: Expected to mirror U.S. success post-approval, country-by-country reimbursement.
Diagnosed but Untreated Patients: Efforts to re-engage via awareness and clinical data.
Solipulol Enrollment: Genetic testing initiatives may accelerate recruitment, but timelines remain fluid.

Conclusion: Zevra delivered a strong Q2, driven by MyKliefa’s launch momentum and strategic financial moves (PRV sale). Challenges persist with Olpruva, but the company remains focused on rare disease leadership with a robust pipeline and cash runway.

About this video

Zevra Therapeutics, Inc. (NASDAQ: ZVRA), a commercial-stage biopharmaceutical company focused on rare diseases, reported robust second quarter 2025 financial results highlighted by significant revenue growth and a strong balance sheet boost from a major one-time transaction. The company posted net revenue of $25.9 million, a sharp increase from $4.4 million in Q2 2024, driven primarily by $21.5 million in MIPLYFFA product sales, alongside contributions from OLPRUVA, French Early Access Program reimbursements for arimoclomol, royalties from AZSTARYS®, and upfront licensing income. Zevra completed the sale of its Priority Review Voucher (PRV) for $150 million earlier in the year, providing a strong capital infusion that significantly strengthened its financial position. This one-time event contributed to GAAP net income of $74.7 million, or $1.24 per basic share, reversing a net loss in the prior year period. Excluding the PRV sale and significant non-cash charges, including a $58.7 million impairment related to intangible assets and an $11.7 million inventory write-down associated with OLPRUVA, the company reported an adjusted net loss of $3.2 million, or $0.06 per share. Operating expenses totaled $24.2 million, with R&D costs decreasing substantially to $3.4 million due to fewer clinical trial costs, while SG&A expenses increased to $20.8 million reflecting expanded commercial, medical, and launch activities. The quarter marked continued momentum in the U.S. launch of MIPLYFFA for Niemann-Pick disease type C (NPC), with the company also submitting a Marketing Authorization Application to the European Medicines Agency to expand access in the EU. Risks remain around OLPRUVA’s underperformance, reflected in the impairment charges, inventory obsolescence, and an uncertain competitive and market landscape. The company highlighted that its strong cash position of approximately $217.7 million as of June 30, 2025, offers financial flexibility to execute its strategic priorities including expanding MIPLYFFA's market penetration, advancing regulatory submissions, and managing assets proactively. About Inside Ticker: Inside Ticker delivers timely, in-depth financial news and earnings analysis to empower investors with actionable insights on global companies across sectors. For comprehensive video analysis and real-time updates, visit https://www.insideticker.com/. Hashtags: #ZevraTherapeutics #ZVRA #q22025 #Earnings #FinancialResults #revenuegrowth #netincome #MIPLYFFA #PriorityReviewVoucher #PRV #NiemannPickDisease #RareDiseases #Biopharma #ImpairmentCharge #InventoryObsolescence #operatingexpenses #RandD #SGA #MarketingAuthorization #EuropeanMedicinesAgency #ProductLaunch #CommercialMomentum #cashposition #StrategicUpdate #InvestorRelations #nasdaq #earningsreport #biotech #stockmarket #RareDiseaseTreatment #pipeline #pharmaceuticals #medicalinnovation #earningsmiss #q2results

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