Zevra Therapeutics, a forward-looking pharmaceutical company, has taken an impressive stride toward establishing itself as a prominent player in the realm of rare diseases through a strategic acquisition and the addition of a novel marketed product. In a noteworthy development announced on Thursday, Zevra has committed a substantial $91 million investment to acquire Acer Therapeutics, a fellow rare disease-focused entity. This strategic move not only reflects Zevra’s commitment to expanding its portfolio but also underscores its dedication to addressing unmet medical needs in the rare disease landscape.
Under the terms of this significant acquisition, slated to conclude in the fourth quarter, shareholders of Acer Therapeutics stand to receive up to $76 million in potential cash payments linked to Contingent Value Rights (CVRs). These CVRs are contingent on achieving pivotal commercial and regulatory milestones for two rare disease drugs – Olpruva and Edsivo.
Acer’s pioneering drug, Olpruva, has garnered U.S. approval for the treatment of urea cycle disorders (UCDs) in December. This disorder entails the absence of a critical liver enzyme responsible for converting nitrogen into urea. Consequently, patients afflicted with UCDs experience an accumulation of toxic ammonia in their bloodstream, which poses severe health risks. The approval of Olpruva offers a ray of hope for patients grappling with this challenging condition.
Furthermore, Edsivo, also known as celiprolol, has emerged as the “standard of care” for the off-label treatment of Ehlers-Danlos syndrome in Europe. Ehlers-Danlos syndrome, or EDS, is an inherited disorder rooted in genetic mutations impacting collagen’s structure, production, or processing. Edsivo’s reputation as a treatment standard underscores Acer’s commitment to addressing critical unmet needs within the rare disease community.
Beyond the potential financial gains tied to commercial and regulatory milestones, Acer investors may also stand to receive additional cash payments connected to regulatory achievements within the company’s early-stage program, ACER-2820, which targets infectious diseases.
While the acquisition deal awaits confirmation by Acer’s shareholders, its implications are substantial for Zevra. The acquisition aligns perfectly with Zevra’s strategic objectives, enabling the company to swiftly bolster its position within the rare disease sector. Christal Mickle, Zevra’s interim CEO and chief development officer, emphasizes that the launch of Olpruva in the U.S. will be facilitated by a specialized, compact commercial team. This dovetails with Zevra’s broader plan, particularly in relation to arimoclomol, the company’s product candidate aimed at treating Niemann-Pick disease Type C (NPC).
Zevra already boasts another commercial drug, Azstarys, designed for the treatment of ADHD in patients aged six and above. With the potential to achieve significant milestones in 2023, along with ongoing royalty revenue generation, Azstarys augments Zevra’s diverse portfolio. The drug received its initial approval in March 2021 and is positioned to contribute to the company’s growth trajectory.
Zevra Therapeutics’ strategic acquisition of Acer Therapeutics marks a significant advancement in the field of rare diseases. This move aligns perfectly with Zevra’s mission to enhance patient care and underscores its commitment to pushing boundaries in the realm of pharmaceutical innovation.