ZEVRA THERAPEUTICS, INC. has recently released its 10-Q report, detailing its focus on addressing unmet needs for the treatment of rare diseases in the United States. The company's lead product candidate is KP1077, currently in Phase 2 clinical trial for idiopathic hypersomnia and Phase 1/2 clinical trial for narcolepsy. Additionally, the company is developing Celiprolol, a prodrug product candidate under Phase 3 clinical trial for the treatment of vascular Ehlers Danlos syndrome. Zevra Therapeutics, Inc. also offers AZSTARYS for attention deficit and hyperactivity disorder, OLPRUVA for urea cycle disorders, and MIPLYFFA for niemann pick disease type C.
In the 10-Q report, ZEVRA THERAPEUTICS, INC. discussed its financial condition and results of operations. The company reported net proceeds of $148.3 million from the sale of a transferable rare pediatric disease priority review voucher (PRV) in connection with the approval of MIPLYFFA for the treatment of neurological manifestations of Niemann-Pick disease type C. Additionally, the company received an upfront payment of $250,000 and potential future regulatory milestones of up to $8.45 million from the licensing of certain intellectual property related to its pre-clinical stage prodrug of dextrorphan.
The report also highlighted the commercial progress of MIPLYFFA, which has been granted orphan medical product designation for the treatment of NPC by the European Commission. MIPLYFFA demonstrated halting of disease progression, ease of flexible administration as an oral treatment, extensive clinical experience with favorable safety data, and advantageous regulatory designations. The company is focusing on preparing for the submission of a Marketing Authorisation Application (MAA) in Europe in the second half of 2025.
Furthermore, ZEVRA THERAPEUTICS, INC. discussed the commercial progress of OLPRUVA, which is approved in the U.S. as adjunctive therapy for the chronic management of urea cycle disorders. The company initiated the commercial launch of OLPRUVA in the U.S. and has a partnership with Relief Therapeutics for commercialization rights in various EU countries. The company also sold the rights to royalty payments for OLPRUVA to Soleus Capital Management L.P.
The market has reacted to these announcements by moving the company's shares -1.52% to a price of $7.75. For the full picture, make sure to review ZEVRA THERAPEUTICS, INC.'s 10-Q report.
