Core Insights - The Phase 3 Orbit study for UX143 (setrusumab) in pediatric and young adult patients with osteogenesis imperfecta (OI) is on track for final analysis by the end of the year [1][3] - The Data Monitoring Committee has confirmed an acceptable safety profile for UX143, allowing the study to proceed [2] - Ultragenyx and Mereo BioPharma are collaborating on the development of setrusumab, targeting OI sub-types I, III, and IV [5][12] Study Details - The Phase 3 Orbit study has enrolled 159 patients across 45 sites in 11 countries, with a primary efficacy endpoint focused on annualized clinical fracture rate [7] - The Cosmic study, which is also in Phase 3, has enrolled 69 patients aged 2 to <7 years, comparing setrusumab to intravenous bisphosphonates [8] - Both studies will conduct final analyses after patients have been on therapy for at least 18 months, with specific statistical thresholds set for each study [4] Background on Osteogenesis Imperfecta - OI is a genetic disorder affecting bone metabolism, primarily caused by mutations in the COL1A1 or COL1A2 genes, leading to increased bone brittleness and a high rate of fractures [9] - Approximately 60,000 individuals are affected by OI in commercially accessible regions, with no globally approved treatments available [9] Mechanism of Setrusumab - Setrusumab is a fully human monoclonal antibody that inhibits sclerostin, which negatively regulates bone formation, potentially increasing bone mass and strength [10] - Previous studies have shown that anti-sclerostin antibodies can significantly improve bone formation and density in OI models [11] Company Profiles - Ultragenyx is focused on developing therapies for rare genetic diseases, with a portfolio aimed at addressing high unmet medical needs [13] - Mereo BioPharma is also dedicated to innovative therapeutics for rare diseases, with setrusumab as one of its key candidates [15]

Zevra Therapeutics (ZVRA) Q1 2025 Earnings Call May 13, 2025 04:30 PM ET Company Participants Nichol Ochsner - Vice President of Investor Relations & Corporate CommunicationsNeil McFarlane - President & CEOJoshua Schafer - CCOR. LaDuane Clifton - CFO, Secretary & TreasurerSumant Kulkarni - Managing DirectorJason Butler - Managing Director & Biotechnology Equity ResearchEddie Hickman - Vice President Conference Call Participants Lachlan Hanbury-Brown - Biotech Equity Research Analyst Operator Good afternoon ...

Core Insights - Zevra Therapeutics reported Q1 2025 net revenue of $20.4 million, a significant increase from $3.4 million in Q1 2024, primarily driven by product net revenue of $17.2 million from MIPLYFFA [8][6][30] - The company completed the sale of its Pediatric Rare Disease Priority Review Voucher (PRV) for gross proceeds of $150 million, enhancing its financial position to support commercial launches and development programs [5][2] - Zevra aims to establish MIPLYFFA as a cornerstone treatment for Niemann-Pick disease type C (NPC) and maximize commercial opportunities with OLPRUVA [2][30] Financial Highlights - Total net revenue for Q1 2025 was $20.4 million, including $17.1 million from MIPLYFFA, $0.1 million from OLPRUVA, and $2.3 million in reimbursements from the French Expanded Access Program for arimoclomol [8][6] - Operating expenses for Q1 2025 were $22.8 million, with R&D expenses at $3.3 million, a decrease from the previous year, while SG&A expenses increased to $19.5 million [13][8] - The net loss for Q1 2025 was $3.1 million, or $0.06 per share, compared to a net loss of $16.6 million, or $0.40 per share in Q1 2024 [13][8] Commercial Highlights - MIPLYFFA had 13 new prescription enrollments in Q1 2025, totaling 122 prescriptions, with market access at 38% of covered lives [7] - OLPRUVA received 5 new patient enrollment forms in Q1 2025, bringing the total to 28, with market access increasing to 78% of covered lives [7] - The company plans to file the MIPLYFFA Marketing Authorization Application (MAA) with the European Medicines Agency in the second half of 2025 [7] Pipeline and Innovation Highlights - Zevra enrolled 5 additional patients in the Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome, bringing total enrollment to 32 [7] - The company out-licensed intellectual property related to a deprioritized pre-clinical prodrug, which may yield future regulatory milestones and royalties [7] Upcoming Events - Zevra will participate in the H.C. Wainwright 3rd Annual Bioconnect Investor Conference on May 20, 2025, and will host a conference call to discuss Q1 2025 results [9][10]

CELEBRATION, Fla., May 01, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage rare disease therapeutics company, today announced that members of Zevra’s executive leadership team will participate in a fireside chat at the Citizens Life Science Conference in New York, NY, on Wednesday, May 7, 2025, at 11:30 a.m. ET. Additionally, management will be available for one-on-one meetings with registered attendees at each conference. The live webcasts will ...