Zevra Therapeutics reports developments as a commercial-stage biopharmaceutical company focused on therapies for rare diseases. Recurring updates center on MIPLYFFA (arimoclomol), its U.S.-approved treatment for Niemann-Pick disease type C, including commercialization, patient access, regulatory activity and diagnostic-support initiatives tied to NPC.
Zevra news also covers financial results, pipeline programs in rare diseases such as vascular Ehlers-Danlos syndrome, and corporate actions that shape its portfolio. Recent company updates include the completed sale of the serdexmethylphenidate, or SDX, portfolio, including AZSTARYS and KP1077, as well as governance and executive-compensation announcements under Nasdaq rules.
Zevra Therapeutics (ZVRA) announced its financial results for Q4 and FY 2022, revealing a net revenue of $2.3 million for Q4, down from $2.6 million in 2021. The company reported a net loss of $9 million, primarily due to R&D expenses of $6.4 million. For FY 2022, net revenue was $10.5 million, significantly down from $28.7 million in 2021, attributed to a lack of one-time regulatory milestone payments. Zevra holds $102.9 million in cash, extending its runway into 2026. The company is focused on advancing its arimoclomol NDA resubmission and KP1077 Phase 2 trial, with key data expected in Q3 and year-end 2023.
Zevra Therapeutics, previously known as KemPharm, announced that CEO Richard W. Pascoe will participate in a fireside chat at the 35th Annual Roth Conference on March 13, 2023, at 2:00 p.m. PT. During this session, Pascoe will outline the company’s innovative clinical, regulatory, and commercialization strategies aimed at advancing therapies for rare diseases. The chat will be accessible in-person and online, with a replay available for 90 days. Investors can also schedule one-on-one meetings with Zevra management from March 12-14. More details are available on Zevra's corporate website.
Zevra Therapeutics (Nasdaq: ZVRA), previously KemPharm, has begun trading under its new ticker symbol on the Nasdaq Global Select Market as of March 1, 2023. The company's rebranding reflects its commitment to the rare disease community. Zevra is developing arimoclomol, an investigational treatment for Niemann-Pick type C disease, which currently has no approved U.S. therapies. The FDA has granted arimoclomol orphan drug designation, Fast Track designation, and rare pediatric disease designation. Zevra is also progressing KP1077, aimed at treating idiopathic hypersomnia, now in a Phase 2 trial. Key milestones for 2023 include a planned resubmission of the NDA for arimoclomol.