Ganaxolone

Stockholm, Sweden, October 26, 2026 - Immedica Pharma AB ("Immedica") today announced that it will advance the development of intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE) and plans to initiate a new phase 3 clinical study in 2027. The decision follows an extensive review of the available clinical data and productive interactions with the U.S. Food and Drug Administration (FDA), which have provided a clear regulatory path forward for the program. Immedica is now progressing the activities required to prepare and operationalize the new study, including finalization of the study design, manufacturing of clinical trial material and clinical site planning. RSE is a life-threatening neurological emergency in which ongoing seizure activity does not respond to at least two different antiseizure medications. Patients who continue to experience seizures after first- and second-line treatment may require intravenous anesthetics and medically induced coma, involving invasive treatment and intensive care. Despite the severity of the condition, treatment options remain limited, and there is currently no approved therapy specifically for the condition. There is a significant need for therapies that can rapidly control seizures and reduce the need for escalation to intravenous anesthesia. "Advancing IV ganaxolone into a new phase 3 study is an important step for Immedica and reflects our commitment to developing treatments for people affected by severe conditions with high unmet medical need," said Anders Edvell, CEO of Immedica. "The program also marks an important evolution for our company as we broaden our capabilities beyond commercialization and strengthen our focus on clinical development. Following a comprehensive evaluation of the clinical evidence and constructive discussions with the FDA, we believe there is a well-defined path forward, and we are now focused on preparing the study and bringing IV ganaxolone into clinical development." "Refractory status epilepticus is a serious medical emergency for which additional treatment options are urgently needed. IV ganaxolone works through a mechanism distinct from currently used therapies. A new phase 3 study is expected to elucidate the role of IV ganaxolone for patients whose seizures continue despite standard treatment", commented Dr Aatif M. Husain, Professor of Neurology and Division Chief, Epilepsy, Sleep, and Clinical Neurophysiology. Duke University School of Medicine, Durham, NC, USA Immedica acquired the global rights to ganaxolone through its acquisition of Marinus Pharmaceuticals in 2025. The company is building on the existing clinical and scientific evidence for the molecule and expanding its clinical development capabilities within rare neurology. About refractory status epilepticus (RSE) Status epilepticus (SE) is a life-threatening, neurological emergency characterized by prolonged, self-sustaining or repeated uncontrolled seizure activity without sufficient recovery between seizures. Management of SE centers around rapid and sustained seizure control. Refractory SE (RSE) is defined as SE resistant to treatment with one first-line ASM (e.g. benzodiazepines) and one second-line ASM. The condition is associated with a risk of neurological injury, including alterations of neuronal networks and neuronal death, complications from prolonged intensive care, and high morbidity and mortality. Current treatment may involve intravenous anesthetics and medically induced coma. These interventions have been associated with additional morbidity, highlighting the need for additional treatment options that can rapidly and durably control seizures. About IV ganaxolone Internalization of GABAA receptors occurs within minutes after SE onset. This results in functional loss of GABAergic inhibition and contributes to SE becoming treatment resistant. While extrasynaptic GABAA receptors are not internalized, they are insensitive to standard GABAergic agents. Ganaxolone, a neuroactive steroid and a synthetic analogue of allopregnanolone, positively modulates both synaptic and extrasynaptic GABAA receptors, thus enhancing inhibitory signaling in the central nervous system. The intravenous formulation of ganaxolone is being developed as an investigational treatment for refractory status epilepticus. IV ganaxolone is not approved for the treatment of refractory status epilepticus. About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica's capabilities cover marketing and sales, market access, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and clinical development, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica's therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE endocrinology and specialty care. Immedica was founded in 2018 and employs today around 200 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of HR & Communication linda.holmstrom@immedica.com We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com