KemPharm Announces Positive Topline Data from Phase 1 Clinical Trial Evaluating Cardiovascular Safety of Serdexmethylphenidate (SDX)



SDX administered in single doses of 80 mg and 200 mg was well tolerated

CELEBRATION, Fla., Sept. 28, 2022 (GLOBE NEWSWIRE) — KemPharm, Inc. (NasdaqGS: KMPH) (“KemPharm” or the “Company”), a specialty pharmaceutical company focused on the discovery, development and commercialization of novel treatments for orphan diseases Nervous System (CNS), Neurodegenerative and Lysosomal Storage Diseases, today announced topline data from its exploratory phase 1 clinical study evaluating the relative cardiovascular effects and pharmacokinetics of serdex methylphenidate (SDX) compared to immediate-release and sustained-release Ritalin formulations confirm® (racemic methylphenidate), a commonly prescribed CNS stimulant. SDX, KemPharm’s proprietary prodrug of D-methylphenidate (d-MPH), is the sole active pharmaceutical ingredient (API) in KP1077, a potential treatment for idiopathic hypersomnia (IH), a rare sleep disorder.

Based on the data, KemPharm believes that the initial dosage levels for the planned Phase 2 clinical trial of KP1077 will be well tolerated while providing higher overall exposure to d-MPH compared to other methylphenidate products, which are often off-label to be used to treat ICH H. KemPharm expects to initiate a Phase 2 clinical trial of KP1077 in patients with IH before the end of 2022 and a second trial in patients with narcolepsy in 2023.

“We are pleased with the initial results from the Phase 1 cardiovascular safety study of SDX, which have demonstrated the potential for safety and good tolerability for ‘higher dose’ formulations of SDX. These data reinforce our confidence that the planned doses for the upcoming Phase 2 trial of KP1077 in IH will provide higher exposure to d-MPH while also offering the potential for greater cardiovascular safety risk compared to current methylphenidate products, which are off- label will be avoided,” said Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “Overall, the data suggest that SDX can be safely dosed at higher concentrations than current products, which should result in improved efficacy. We believe this could position KP1077 as an advance in the treatment of IH.”

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The phase 1 open-label study enrolled 15 volunteers, each of whom was randomly assigned to receive a series of four oral treatments in a crossover design: single doses of 80 mg and 200 mg SDX, two doses of 40 mg immediate-release Ritalin with an interval of 5 hours between dosing and a single dose of 80 mg Ritalin LA®, with each dosing period being at least seven days apart. The total Ritalin immediate-release dose (2 x 40 mg), the 80 mg Ritalin LA and 80 mg SDX represent approximately the same amount of d-MPH, the compound of interest, in each dose, while the 200 mg dose contains SDX approximately 2.5 times the amount of d-MPH as 80 mg Ritalin LA.

Maximum plasma concentrations (CMax) of d-MPH were similar for both Ritalin treatments administered at equal doses of 80 mg. The d-MPH CMax Values ​​for both SDX treatments were dose proportional to CMax of 200 mg SDX about half of the values ​​for Ritalin. Total plasma exposure to d-MPH was highest in the 200 mg SDX treatment group due to the prolonged-release profile unique to the prodrug that resulted in prolonged exposure to d-MPH compared to either Ritalin treatment group .

The data from the study also showed that the maximum drug-related change from baseline in the mean heart rate and systolic blood pressure curves was highly correlated with the maximum d-MPH exposure during the same period. As a result, the maximum changes from baseline in heart rate and systolic blood pressure were higher for both Ritalin treatments than for the SDX treatments. Overall, the pharmacokinetic and pharmacodynamic data confirmed that the two doses of SDX that released d-MPH are released as planned and in a manner expected to be well suited for the treatment of IH.

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About KemPharm:

KemPharm is a specialty pharmaceutical company focused on the discovery, development and commercialization of novel treatments for rare central nervous system (CNS), neurodegenerative and lysosomal storage disorders. KemPharm has a diverse product portfolio combining a clinical-stage development pipeline with NDA-stage and commercial assets. The pipeline includes arimoclomol, an orally administered, first-in-class investigational drug candidate for Niemann-Pick type C disease (NPC), and KP1077, which the company is developing to treat idiopathic hypersomnia (IH), a rare neurological disorder that causes sleep disorders and narcolepsy. In addition, the US Food and Drug Administration (FDA) has approved AZSTARYS®a once-daily treatment for ADHD in patients six years of age and older, containing KemPharm’s prodrug serdexmethylphenidate (SDX), marketed by Corium, Inc. in the US and APADAZ®an immediate-release combination product containing benzhydrocodone, KemPharm’s prodrug of hydrocodone, and acetaminophen, marketed by KVK-Tech, Inc. in the United States. For more information about KemPharm and its pipeline of product candidates, visit or contact us Twitter, LinkedIn, Facebook and youtube.

Early Access programs are made available by KemPharm, Inc. and its affiliates and are subject to the Company’s Early Access Program (EAP) policy as posted on its website at Participation in these programs is subject to the laws and regulations of the relevant jurisdiction in which the relevant program is operated. Eligibility to participate in such a program is at the discretion of the treating physician.

Caution Regarding 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 are not purely historical or current facts, including but not limited to, 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”, “aim”, “predict” or the negative versions of these words or other comparable word or phrase, although not all forward-looking statements contain those identifying words or phrases. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements include statements about the promise and potential impact of our data from preclinical or clinical studies, including but not limited to the timing and results of clinical studies or evaluations, the potential uses or benefits of KP1077, SDX or other product candidates for each specific disease indication or at any dosage, the potential benefits of any of KemPharm’s product candidates, our plans and our strategic and product development goals. These forward-looking statements are based on information currently available to KemPharm and its current plans or expectations and are subject to a number of known and unknown uncertainties, risks and other important factors that could cause our actual results, performance or achievements to differ materially 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 KemPharm’s annual report on Form 10-K for the year ended December 31, 2021, as updated by the Quarterly Report on Form 10-Q for the three months ended December 30 June 2022 and KemPharm’s other filings with the Securities and Exchange Commission. Although we may elect to update any such forward-looking statements at any time in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe that the expectations reflected in such forward-looking statements are reasonable, we cannot guarantee that such expectations will prove to be correct. These forward-looking statements should not be construed as representing our views at any time after the date of this press release.

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KemPharm contacts:

Tiberend Strategic Advisors, Inc.
Jason Rando/Daniel Kontoh-Boateng
[email protected]
[email protected]