Zevra Therapeutics Reiterates Commitment to Rare Disease Community as New Corporate Council Member of the National Organization for Rare Disorders (NORD)

CELEBRATION, Fla., Feb. 28, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NASDAQ:KMPH) ("Zevra" or the "Company" and formerly KemPharm, Inc.), a rare disease therapeutics company, today announced that it has become a Corporate Council member of the National Organization for Rare Disorders (NORD) and reiterated its commitment to the rare disease community by celebrating Rare Disease Day 2023.

A rare disease is a condition that impacts fewer than 200,000 people in the United States, with over 7,000 known rare diseases nationally affecting nearly 1 in 10 people. NORD is dedicated to promoting research and access to therapies for the more than 300 million people worldwide with rare diseases, as well as raising awareness of this important community. As a company focused on advancing therapies for rare diseases, Zevra stands with NORD and the rare disease community today and every day.

"Zevra's dedication to the rare disease community is critical as we all work to bring much-needed therapies and relief to people affected by a rare condition without treatment options," said Peter L. Saltonstall, President and Chief Executive Officer of NORD. "We enthusiastically welcome Zevra as an important partner for our community and appreciate the symbolism of its new name inspired by the zebra – the international symbol of the rare disease community."

On February 22, 2023, Zevra announced its new corporate name to embody the Company's mission of innovating new therapies for people with rare conditions and the highest unmet needs. Zevra is Greek for zebra, the symbol of rare diseases.

"Rare Disease Day is both a reminder of the strides the rare disease community has made over the last four decades with the support of NORD's leadership, as well as a call to action to continue making meaningful progress in rare disease therapies," said Richard W. Pascoe, Chief Executive Officer of Zevra. "Zevra is proud to partner with NORD as we redouble our efforts to bring accessible treatments to patients with Niemann-Pick disease type C and idiopathic hypersomnia."

Visit Zevra's new corporate website at www.zevra.com to learn more.

About Zevra

Zevra Therapeutics is a rare disease company melding science, data, and patient need to create transformational therapies for diseases with limited or no treatment options. With unique, data-driven clinical, regulatory, and commercialization strategies, the Company is overcoming complex drug development challenges to bring much-needed therapies to patients.

Arimoclomol, Zevra's orally-delivered, first-in-class investigational product candidate for the treatment of Neimann-Pick disease type C ("NPC"), has been granted orphan drug designation, Fast Track designation and rare pediatric disease designation for the treatment of NPC by the US Food and Drug Administration ("FDA"), and orphan medicinal product designation for the treatment of NPC by the European Medicines Agency ("EMA").

KP1077 is Zevra's lead clinical candidate being developed to treat idiopathic hypersomnia ("IH") and narcolepsy. KP1077 is comprised solely of serdexmethylphenidate ("SDX"), Zevra's proprietary prodrug of d-methylphenidate ("d-MPH"). The FDA has granted KP1077 orphan drug designation for the treatment of IH, and the US Drug Enforcement Agency ("DEA") has classified SDX as a Schedule IV controlled substance based on evidence suggesting SDX has a lower potential for abuse when compared to d-MPH, a Schedule II controlled substance.

Early access programs are made available by Zevra Therapeutics, Inc. and its affiliates and are subject to the Company's Early Access Program ("EAP") policy as published on its website at zevra.com. Participation in these programs is subject to the laws and regulations of each jurisdiction under which each respective program is operated. Eligibility for participation in any such program is at the treating physician's discretion.

Caution Concerning Forward Looking Statements

This press release may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements that do not relate solely to historical or current facts, including without limitation and which can be identified by the use of words such as "may," "will," "expect," "project," "estimate," "anticipate," "plan," "believe," "potential," "should," "continue," "could," "intend," "target," "predict," or the negative versions of those words or other comparable words or expressions, although not all forward-looking statements contain these identifying words or expressions. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include statements regarding our efforts to bring accessible treatments to patients with Niemann-Pick disease type C disease and idiopathic hypersomnia, and our strategic and product development objectives. These forward-looking statements are based on information currently available to Zevra and its current plans or expectations and are subject to a number of known and unknown uncertainties, risks and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These and other important factors are described in detail in the "Risk Factors" section of Zevra's (formerly KemPharm's) Annual Report on Form 10-K for the year ended December 31, 2021, as updated by Zevra's (formerly KemPharm's) Quarterly Report on Form 10-Q for the three months ended September 30, 2022, and Zevra's (formerly KemPharm's) other filings with the Securities and Exchange Commission. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts:

Nichol Ochsner

+1 (732) 754-2545

[email protected]

Jennifer Arcure

+1 (917) 603-0681

[email protected]