GRIA1

STOCKHOLM, Jan. 4, 2023 /PRNewswire/ -- Sedana Medical AB (publ) (SEDANA: FN Stockholm) today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) for the evaluation of isoflurane via the Sedaconda ACD-S device for sedation of mechanically ventilated patients in the intensive care (ICU) setting. Fast Track is a process designed to facilitate the development, and expedite the review of therapies that treat serious conditions and fill an unmet medical need. The purpose is to get important new therapies to the patient earlier. Clinical programs with Fast Track Designation may benefit from frequent communication with the FDA throughout the development and review process and may be eligible to Accelerated Approval and Priority Review if relevant criteria are met. Another possible benefit may be a Rolling Review, which means that completed sections of the New Drug Application (NDA) can be submitted for review by FDA, rather than waiting until every section of the NDA is completed. "The FDA's decision to grant Fast Track Designation underscores the potential for our product candidates to address a serious unmet need and bring meaningful benefits to ventilated patients in intensive care. We are fully committed to work even more closely with FDA to bring our therapy to US patients as soon as possible," said Johannes Doll, CEO of Sedana Medical. "This excellent news represents an important step forward in our highest potential market." Sedana Medical is aiming for a combination registration of the medical device Sedaconda ACD and the pharmaceutical isoflurane for sedation of mechanically ventilated intensive care patients in the United States. Two identical Phase III studies, INSPiRE-ICU 1 and 2, are ongoing with the objective to confirm the efficacy and safety of inhaled sedation with isoflurane delivered via Sedaconda ACD in intensive care. Sedana Medical will engage in discussions with the FDA on how the development program can benefit from the FTD. At present, the guidance regarding the timeline is unchanged: assuming rapid enrolment of patients and successful trials, Sedana Medical expects the NDA submission in 2024 and a launch in early 2025. About the studies INSPiRE-ICU 1 and INSPiRE-ICU 2 (SED003 and SED004) The INSPiRE-ICU (Inhaled Sedation vs Propofol in Respiratory failure) studies are two identical phase III studies aiming to confirm the efficacy and safety of inhaled isoflurane, delivered via the Sedaconda ACD, for the sedation of adult mechanically ventilated ICU patients, in comparison to intravenous infusion of propofol. The studies will enrol a total of 470 adult patients across 25-30 sites in the US. Patient enrolment is expected to be completed in 2023. The primary endpoint is the proportion of time spent within the target range of sedation depth in absence of rescue sedation, as assessed according to the Richmond Agitation Sedation Scale (RASS). In addition, the studies will investigate several secondary endpoints, including the use of opioids, the wake-up time, the cognitive recovery after end of sedation, and the spontaneous breathing effort. The assessments will be performed by blinded assessors to meet the requirements of the FDA. Further information on the studies is available at (NCT05312385 for SED003 and NCT05327296 for SED004). For additional information, please contact: Johannes Doll, CEO, +46 76 303 66 66 Johan Spetz, CFO, +46 730 36 37 89 [email protected] Sedana Medical is listed on Nasdaq First North Growth Market in Stockholm. The company's Certified Adviser is Erik Penser Bank, +46 8 463 83 00, [email protected] About Sedana Medical Sedana Medical AB (publ) is a pioneer medtech and pharmaceutical company focused on inhaled sedation to improve the patient's life during and beyond sedation. Through the combined strengths of the medical device Sedaconda ACD and the pharmaceutical Sedaconda (isoflurane), Sedana Medical provides inhaled sedation for mechanically ventilated patients in intensive care. Sedana Medical has direct sales in Benelux, France, Germany, Great Britain, the Nordic, and Spain. In other parts of Europe as well as in Asia, Australia, Canada, and South- and Central America, the company works with external distributors. Sedana Medical was founded in 2005, is listed on Nasdaq First North Growth Market (SEDANA) and headquartered in Stockholm, Sweden. The following files are available for download: SOURCE Sedana Medical

Scientists have identified a new genetic disease, which causes some children's brains to develop abnormally, resulting in delayed intellectual development. The majority of patients with the condition, that is so new it doesn't have a name yet, have severe learning difficulties which affect their quality of life. Scientists have identified a new genetic disease, which causes some children's brains to develop abnormally, resulting in delayed intellectual development. The majority of patients with the condition, that is so new it doesn't have a name yet, have severe learning difficulties which affect their quality of life. An international team of researchers from the universities of Portsmouth, Southampton and Copenhagen found that changes in a protein coding gene called Glutamate Ionotropic Receptor AMPA Type Subunit 1 (GRIA1) caused this rare genetic disease. Now the variant has been identified, it will help clinicians come up with targeted interventions to help patients and their families, also opening the door to screening and prenatal diagnosis. The GRIA1 gene helps to move electrical signals around the brain. However, if this process is interrupted or made less efficient, it can cause a reduction in the brain's capacity to retain information. The research team, made up of frog geneticists, biochemists and clinical geneticists, used tadpoles in which the human gene variants were mimicked using gene editing to show that GRIA1 changes are the underlying cause of the behaviour-altering disease. The biochemical analysis of the variants was also performed in frog oocytes. The findings are published in the American Journal of Human Genetics. Study co-author Professor Matt Guille, who leads a laboratory in the Epigenetics and Developmental Biology research group at the University of Portsmouth, said: "Next generation DNA sequencing is transforming our ability to make new diagnoses and discover new genetic causes of rare disorders. "The main bottleneck in providing diagnoses for these patients is linking a change discovered in their genome firmly to their disease. Making the suspect genetic change in tadpoles allows us to test whether it causes the same illness in humans. "The resulting data allow us to support our colleagues in providing the more timely, accurate diagnosis that patients and their families so desperately need." Co-author Dr Annie Goodwin, Research Fellow at the University of Portsmouth who performed much of the study, said: "This was a transformational piece of work for us; the ability to analyse human-like behaviours in tadpoles with sufficient accuracy to detect genetic disease-linked changes opens the opportunity to help identify a huge range of diseases. This is particularly important given that so many neurodevelopmental diseases are currently undiagnosed." Co-author Professor Diana Baralle, Professor of Genomic Medicine and Associate Dean (Research) in the Faculty of Medicine at the University of Southampton, added: "Discovering these new causes for genetic disorders ends our patients' diagnostic odyssey and this has been made possible by collaborative interdisciplinary working across universities." One in 17 people will suffer from a rare disease at some time in their lives. Most of these rare diseases have a genetic cause and often affect children, but proving which gene change causes a disease is a huge challenge. Professor Guille said that previously, while studies connecting a gene and a disease were mainly performed in mice; several labs, including his own at the University of Portsmouth, have recently shown that experiments in tadpoles can also provide very strong evidence about the function of variant human genes. The process of re-creating some gene variants in tadpoles is straightforward and can be done in as little as three days. Professor Guille added: "We are currently extending and improving our technology in a programme funded by the Medical Research Council; this is making it applicable to the wider range of disease-related DNA changes provided to us by our clinical collaborators. "If the clinical researchers find the information sufficiently useful, then we will continue to work together to scale up the pipeline of gene function analysis so it can be used to direct effective interventions for a significant number of patients."