Hemihyperplasia, Isolated

Researchers explored innovative therapeutic strategies that could greatly advance the understanding and management of OSA-related morbidities. A groundbreaking study provides new insights into the treatment of obstructive sleep apnea (OSA) and its associated health impacts. A groundbreaking study published in the American Journal of Respiratory and Critical Care Medicine provides new insights into the treatment of obstructive sleep apnea (OSA) and its associated health impacts. Researchers at the University of Missouri School of Medicine and the Marshall University Joan C. Edwards School of Medicine led by David Gozal, M.D., M.B.A., Ph.D. (Hon), vice president of health affairs at Marshall University and dean of the Joan C. Edwards School of Medicine, explored innovative therapeutic strategies that could greatly advance the understanding and management of OSA-related morbidities. Obstructive sleep apnea is a widespread condition affecting nearly one billion people around the world. It is linked to accelerated biological aging and various end-organ health issues, such as cardiovascular, cognitive and metabolic morbidities. While current treatments like continuous positive airway pressure (CPAP) have demonstrated limited success in reversing such outcomes, this recent research delves into adjunct therapies, particularly the use of senolytics, to alleviate the burden of OSA. The study assessed whether targeting senescence, a treatment that targets aging cells, coupled with a simulated approach that imitates good adherence to the most common treatment for sleep apnea (CPAP) could improve physiological outcomes in mice exposed to chronic intermittent hypoxia (IH), a hallmark feature of OSA. The study found that combining partial normoxic recovery with the senolytic Navitoclax (NAV) significantly reduced sleepiness during the dark phase, the usual sleeping time for rodents. The combined therapy also showed notable improvements in cognitive function. Such findings were not apparent when only "CPAP" was administered. "Our findings suggest that the reversibility of end-organ morbidities induced by OSA goes beyond normalizing oxygenation patterns," said Gozal, corresponding author on the study. "Targeting accelerated senescence appears to be a promising avenue for improving treatment outcomes in individuals with OSA." "It is quite evident in our preclinical model of OSA that recovering normal oxygenation patterns seen in traditional treatments, namely continuous positive airway pressure, is ineffective in preventing or mitigating multiple end-organ dysfunctions" said Mohammad Badran, Ph.D., assistant professor at the University of Missouri School of Medicine. "Adjuvant therapies, in this case senolytics, have the potential of becoming valuable and effective treatments targeting OSA-induced morbidities." In addition, the therapy demonstrated positive effects on coronary artery function, glucose and lipid metabolism and reduced intestinal permeability. The combination of simulated adherent CPAP treatment and NAV effectively reduced senescence in multiple organs, indicating a potential reversal of OSA-induced cellular aging processes. This research could open new doors in understanding the deleterious processes involved in OSA-associated morbidities and formulate novel approaches aimed at reversing the multifaceted impact of OSA on health. Development of safe senolytics specifically focused on OSA along with clinical studies are warranted to validate these findings in human subjects and explore potential applications in the field of sleep medicine. In addition to Gozal and Badran, co-authors on the study from the University of Missouri include Clementine Puech, Ph.D., post-doctoral fellow; Abdelnaby Khalyfa, Ph.D., associate professor; Rene Cortese, Ph.D., assistant professor in the Department of Child Health and the Department of Obstetrics, Gynecology and Women's Health; Kylie Cataldo, research specialist; and Zhuanhong Qiao, senior research consultant.

CELEBRATION, Fla., Nov. 18, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a biotechnology company focused on the discovery, development and commercialization of novel treatments for rare central nervous system (CNS) and neurodegenerative diseases, lysosomal storage disorders and related treatment areas, today announced that the U.S. Food and Drug Administration (FDA) has granted the Orphan Drug Designation to serdexmethylphenidate (SDX), KemPharm’s proprietary prodrug of d-methylphenidate (d-MPH), for the treatment of idiopathic hypersomnia (IH), a rare neurological sleep disorder. SDX is the sole active pharmaceutical ingredient (API) in KP1077, KemPharm’s lead clinical candidate being developed as a treatment for IH and narcolepsy. KemPharm expects to initiate a Phase 2 clinical trial of KP1077 in patients with IH prior to year-end 2022 and a second trial in patients with narcolepsy in 2023. “We appreciate the FDA’s decision to grant Orphan Drug Designation to SDX for IH, a rare sleep disorder characterized by multiple, debilitating symptoms for which few treatment options exist,” stated Travis Mickle, Ph.D., President and Chief Executive Officer of KemPharm. “This regulatory milestone comes at an important juncture in the ongoing development of KP1077 as we prepare to initiate the Phase 2 clinical trial in IH before year-end 2022. We believe KP1077 could provide an improved treatment option for patients with IH and other sleep disorders by addressing the most debilitating symptoms of IH, including excessive daytime sleepiness, extreme difficulty waking up (sleep inertia), severe “brain fog,” and falling asleep unintentionally and/or at inappropriate times, even after adequate or prolonged nighttime sleep.” FDA Orphan Drug Designation may be granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States, or a patient population greater than 200,000 individuals in the United States and when there is no reasonable expectation that the cost of developing and making available the drug in the United States will be recovered from sales in the United States for that drug. If a product that has Orphan Drug Designation subsequently receives the first FDA approval for a particular active ingredient for the disease for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications, including a full NDA, to market the same drug for the same indication for seven years, except in limited circumstances. Orphan drug exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for a different disease or condition. Among the other benefits of Orphan Drug Designation are tax credits for certain research and a waiver of the NDA application user fee. About KemPharm: KemPharm is a biotechnology company focused on the discovery, development and commercialization of novel treatments for rare CNS and neurodegenerative diseases, lysosomal storage disorders and related treatment areas. KemPharm has a diverse product portfolio, combining a clinical-stage development pipeline with NDA-stage and commercial assets. The pipeline includes arimoclomol, an orally-delivered, first-in-class investigational product candidate for Niemann-Pick disease type C (NPC), and KP1077, which the Company is developing as a treatment for idiopathic hypersomnia (IH), a rare neurological sleep disorder, and narcolepsy. In addition, the U.S. Food and Drug Administration (FDA) has approved AZSTARYS®, a once-daily treatment for ADHD in patients age six years and older containing KemPharm’s prodrug, serdexmethylphenidate (SDX), which is being commercialized by Corium, Inc. in the U.S. The FDA has also approved 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 U.S. For more information on KemPharm and its pipeline of product candidates visit www.kempharm.com or connect with us on 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 published on its website at www.kempharm.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 discretion of the treating physician.