Reaffirming Buy on Zevra: Durable Miplyffa Efficacy and Annuity-Like NPC Revenue Support $26 Target

Analyst Brandon Folkes of H.C. Wainwright reiterated a Buy rating on Zevra Therapeutics, retaining the price target of $26.00.

Brandon Folkes has given his Buy rating due to a combination of factors that underscore the long-term commercial potential of Miplyffa in Niemann-Pick disease type C (NPC). The new poster data reinforce that Miplyffa not only slows disease progression over a four-year period but also shows a clinically meaningful effect as early as three months, distinguishing it from other options by stabilizing overall disease trajectory in both adult and pediatric patients. He views this early and durable efficacy, along with its status as the only therapy shown to halt disease progression at 12 months, as a key competitive advantage that should support sustained physician adoption.

At the same time, Folkes emphasizes the annuity-like revenue profile emerging from the Expanded Access Program: patients typically begin treatment in late adolescence, remain on therapy for extended periods, and show high continuation rates when transitioning from the access program to commercial use. The data highlight that NPC remains underdiagnosed and undertreated, which he believes gives Zevra a significant runway to expand the treated population over time. While recognizing that quarterly enrollment numbers can appear uneven in a rare disease setting, he argues that annual patient adds and high treatment persistence are the more relevant metrics, and together they support his view of a long-duration, profitable revenue stream that justifies maintaining a Buy rating and a $26 price target.