Zevra Therapeutics (NASDAQ: ZVRA), a rare Disease therapeutics company, uniquely combines data-driven techniques, patient needs and outside the box strategies while challenging moribund drug development protocols to bring new therapeutic relief to millions of people suffering with rare diseases. With a team of expert scientists, patient advocates, development strategists, commercialization specialists, and business development gurus – Zevra intends to become a global leader in developing and commercializing treatments for rare diseases that have limited or no therapeutic options. The company’s recent Letter to Stockholders reveals what looks like a launch code for success and suggests the company is on a fast track to drive value for all its stakeholders – patients and caregivers, stockholders, investors, and employees.

In the letter, Neil F. McFarlane, President and CEO, highlighted recent achievements and, importantly, outlined the vision for the company’s future. McFarlane began his tenure by instilling a couple of prime directives that have become guiding principles for the Zevra team. The first directive has become the company mantra, “What’s best for the patient?”, and patient outcomes now guide daily decisions. The second directive McFarlane instilled, “We must execute.”, drives value not just for patients but for all stakeholders – he believes that by defining a clear strategy, creating effective tactics to reach strategic objectives, then executing at the highest level will create and drive value, and help propel Zevra into global leadership in developing and commercializing rare disease therapeutics. Hard to argue with his directives, Zevra is fast making huge strides in reaching objectives.

Zevra has already made significant progress advancing its rare disease portfolio and made demonstrable progress in adroitly executing key strategic priorities. In November 2023, the company completed a shrewd, capital-efficient acquisition of Acer Therapeutics. That acquisition allowed Zerva to immediately start delivering value to patients by commercializing OLPRUVA(R) for treatment of urea cycle disorders (“UCDs”). UCDs are related to defects of enzymes involved in the urea cycle which can lead to high toxic features of ammonia-like irreversible brain damage, coma, and death.

Immediately after the acquisition, Zerva deployed a systematic strategy to build awareness with physicians across the country about OLPRUVA for people living with UCDs. In the three months since launch, Zerva’s team also engaged with over 90% of its customers. The company concurrently achieved meaningful growth in reimbursement coverage, from ~55% of U.S. covered lives at the time of acquisition, to now more than 70%.

The full commercial launch of OLPRUVA at the end of January 2024 was designed to provide scale and cost synergies to support and accelerate the launch and commercialization of arimoclomol to treat Niemann-Pick Disease. Affecting mostly children, Niemann-Pick is a group of rare genetic conditions that affect the body’s ability to break down and use fats, such as cholesterol and lipids, inside cells, and over time, the cells die worsening the function of the nerves, brain, and other organs. With no known cure and sometimes fatal, Zerva’s arimoclomol could be the lifeline so many children need.

Exemplifying the prime directives, the entire Zerva team put forth enormous effort to submit a comprehensive data set to the U.S. Food and Drug Administration (“FDA”) supporting the resubmission of the New Drug Application (“NDA”) for arimoclomol. In March, Zerva announced that the FDA had extended the review period for the NDA, resulting in a revised Prescription Drug User Fee Act (“PDUFA”) date of September 21, 2024.

Also in March, Zerva reported positive top-line data from its Phase 2 study of KP1077 in patients with idiopathic hypersomnia (“IH”). Initial results show that KP1077 is well tolerated and demonstrates early signs of differentiated and meaningful clinical benefits. Topline data from the completed trial are expected in the first half of 2024 and represent another potential Zevra commercial candidate.

Zerva fully intends to bring life-changing therapeutics to people living with rare diseases through expertise, elegant solutions, and transparent data narratives which have already produced a late-stage rare disease clinical pipeline and two partnered commercial products.

But blockbusters and breakthroughs don’t happen in a vacuum. It takes a dedicated and committed team of expert scientists, patient advocates, development strategists, commercialization specialists, and business development gurus with a proven record of bringing new therapies to patients…. and Zerva is loaded with talent.

Zerva is building a team of talented professionals committed to using science and data-driven development approaches to create and deliver new therapies to address critical and often overlooked unmet needs. Since announcing the strategic objective of becoming a leading rare disease company in the first half of 2022, Zerva has grown its team of skilled professionals through both recruiting and acquisitions, from a team of 24 to 81. It’s important to note that this personnel ramp-up includes the buildout of targeted customer-facing commercial and medical affairs teams with decades of experience in rare diseases. The company also added Adrian Quartel, M.D. as Zevra’s Chief Medical Officer, who brings more than 20 years of experience with a track record of success in clinical development, pharmacovigilance and medical affairs addressing the needs of the rare disease community. Such powerful commercial and clinical muscle are likely to really drive value and bode well for the future of the company.

Zevra Therapeutics leverages decades upon decades of collective experience in drug development and regulatory machinations to develop therapies faster and overcome challenging regulatory situations. Rare diseases may finally be reigned-in and held at bay by combining science, data, and patient needs with a team of dedicated professionals to create transformational therapies and bring life-changing therapeutics to millions of people living with rare diseases. Look for more exciting news from Zerva in the months to come.

For more information, please visit www.Zevra.com.

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