KemPharm Doses First Subject in Phase 1 Clinical Trial Evaluating Cardiovascular Safety of Serdexmethylphenidate

KemPharm, Inc. recently announced the first subject has been dosed in a Phase 1 clinical trial designed to assess the relative cardiovascular safety of serdexmethylphenidate (SDX) compared to immediate-release and long-acting formulations of Ritalin (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, which KemPharm is developing as a treatment for idiopathic hypersomnia (IH), a rare sleep disorder.

“The intent of this clinical trial is to collect additional data to support the ongoing development of our SDX-based product candidates, in particular, KP1077, and further differentiate SDX from other methylphenidate-based products,” said Travis Mickle, PhD, President and Chief Executive of KemPharm. “The off-label use of stimulant based treatments in IH and other sleep disorders is growing but with an number of significant limitations based on patient comorbidities and demographics as well as patient dissatisfaction due to inadequate efficacy. Regarding cardiovascular safety, it is estimated that roughly 50% of the US population suffers from high blood pressure, which is also a primary side-effect of stimulant treatment and as a result, many IH patients are either contraindicated, discontinued or limited in the dosage of stimulant that can be prescribed for treatment. We believe this limitation can be overcome by demonstrating an improved cardiovascular safety profile compared to current stimulants, which would potentially allow SDX to be dosed higher than current products resulting in improved efficacy. As we hope to demonstrate in this trial, we believe that observing limited cardiovascular side-effects represents the key product differentiator for both the safety and potential efficacy of KP1077 compared to existing stimulant treatments.”

KemPharm anticipates filing an Investigational New Drug (IND) application for KP1077 with the US FDA as early as this quarter. Upon acceptance of the IND, KemPharm plans to initiate a Phase 2 clinical trial of KP1077 in patients with IH later this year and a second trial in patients with narcolepsy as early as the second half of 2022.

The Phase 1 open-label trial will enroll up to 15 volunteers, each randomly receiving a series of four oral treatments in a crossover design: single doses of 80 mg and 200 mg of SDX, two doses of 40 mg of immediate-release Ritalin, or a single dose of 80 mg of Ritalin LA, with each dose spaced at least seven days apart. The immediate-release Ritalin total dose (2 x 40 mg), the 80 mg Ritalin LA and 80 mg of SDX represent approximately the same amount of d-MPH, the active ingredient of interest, in each dose. The primary objective of the study is to evaluate cardiovascular response including heart rate, blood pressure and electrocardiogram (ECG), and any potential correlation with the exposure to d-MPH after administration of each treatment. Secondary endpoints will include pharmacokinetics, and overall safety and tolerability of SDX. KemPharm expects to receive topline results from this cardiovascular safety trial as early as the third quarter of 2022.

KemPharm is a specialty pharmaceutical company focused on the discovery and development of novel treatments for rare central nervous system (CNS) diseases through its proprietary LAT (Ligand Activated Therapy) platform technology. KemPharm utilizes its proprietary LAT platform technology to generate improved prodrug versions of FDA-approved drugs as well as to generate prodrug versions of existing compounds that may have applications for new disease indications. KemPharm’s prodrug product candidate pipeline is focused on the high need areas of idiopathic hypersomnia (IH) and other CNS/rare diseases. In addition, the US FDA has approved AZSTARYS, a new once-daily treatment for ADHD in patents age 6 years and older containing KemPharm’s prodrug, serdexmethylphenidate (SDX), which is being commercialized by Corium, Inc. in the US, and APADAZ, an immediate-release combination product containing benzhydrocodone, KemPharm’s prodrug of hydrocodone, and acetaminophen, which is being commercialized by KVK-Tech, Inc. in the US. For more information, visit www.kempharm.com.