Three-year ganaxolone data in CDD presented at EEC 2026

CHICAGO, Illinois, Sept 10, 2026 - Immedica Pharma US Inc. announced today that data evaluating the long-term efficacy of ganaxolone in patients with CDKL5 deficiency disorder (CDD) was presented at the 16th European Epilepsy Congress (EEC), taking place in Athens, Greece, September 5-9, 2026. The poster presentation featured results from a post-hoc analysis titled "Sustained clinical efficacy of ganaxolone in CDKL5 deficiency disorder: three-year follow-up." The analysis evaluated long-term seizure outcomes in patients who participated in the open-label extension phase following the pivotal, Phase III Marigold study (NCT03572933). 88 patients (aged 2 to 19) who completed the 17-week double-blind phase continued into the open-label extension study, where all participants received ganaxolone treatment. The study was completed after 233 weeks (~4.25 years; inclusive of the 17-week double blind phase) after FDA approval was granted in the United States in March of 2022. Results demonstrated sustained reductions in major motor seizure frequency (MMSF) throughout the open-label extension period, with the proportion of participants with ≥50% reduction from baseline in 28-day MMSF consistently having met or exceeded that achieved in the ganaxolone group during the double-blind phase (24.5%) at all 3-month intervals for the first 3 years of treatment in the open-label extension. After 163 weeks (~3 years) of ganaxolone treatment in the Marigold open label extension, the median change from baseline in the 28-day MMSF was 35.6%, and 42.1% of participants demonstrated ≥50% reduction in the 28-day MMSF. No new safety findings were identified during the open label extension. "Previously published data have demonstrated the long-term, 2-year clinical effectiveness of ganaxolone in people with CDD," said presenting author Professor Sam Amin, Bristol Royal Hospital for Children, United Kingdom. "The continued trends noted in these 3-year findings contribute additional evidence to support the long-term, clinical effectiveness of ganaxolone." Poster Details Title : Sustained clinical efficacy of ganaxolone in CDKL5 deficiency disorder: three-year follow-up Presentation date and time: Monday, September 7th from 2-3pm Poster number: P0262 Presenter : Sam Amin, Department of Paediatric Neurology, Bristol Royal Hospital for Children, United Kingdom Authors : Sam Amin, Department of Paediatric Neurology, Bristol Royal Hospital for Children, Bristol, United Kingdom, Michelle L. Shuffett (submitting author): Immedica Pharma US Inc., Chicago, IL, Jenny Samuelsson: Immedica Pharma AB, Stockholm, Sweden, Kristina Lindsten: Immedica Pharma AB, Stockholm, Sweden, Kenneth Johnson: Immedica Pharma US Inc., Chicago, IL, Christine Ochoa Escamilla: Immedica Pharma US Inc., Chicago, IL, Scott T. Demarest: Children's Hospital Colorado, Department of Pediatrics, University of Colorado, School of Medicine, University of Colorado, Aurora, CO, USA. Please see the INDICATION AND SAFETY SUMMARY WITH WARNINGS below, and the full Prescribing Information for ZTALMY® (ganaxolone) oral suspension, CV. About CDKL5 Deficiency Disorder CDKL5 deficiency disorder (CDD) is a serious and rare genetic disorder that is caused by a pathogenic variant in the cyclin-dependent kinase-like 5 (CDKL5) gene, located on the X chromosome. CDD is characterized by early-onset, difficult-to-control seizures and severe neurodevelopmental impairment. About the Marigold Trial ZTALMY was approved by the U.S. Food and Drug Administration in March 2022 based on data from the Phase 3 Marigold double-blind placebo-controlled trial of 101 patients with CDD, published in The Lancet Neurology , in which ZTALMY significantly reduced the frequency of monthly major motor seizures by a median of 30.7% compared with 6.9% for placebo (p=0.0036). In this trial, ZTALMY demonstrated efficacy, safety and tolerability with the most common adverse reactions (incidence ≥5% and at least twice the rate of placebo) in the ZTALMY group being somnolence, pyrexia, salivary hypersecretion and seasonal allergy. About ZTALMY® (ganaxolone) ZTALMY is the first FDA-approved treatment indicated specifically for seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two years of age and older. ZTALMY, a neuroactive steroid anticonvulsant that acts as a positive allosteric modulator of GABAA receptors in the central nervous system, is a sugar-free cherry-flavored oral suspension taken three times daily. IMPORTANT SAFETY INFORMATION AND INDICATION AND USAGE ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS · Somnolence and Sedation: ZTALMY can cause somnolence and sedation. In a clinical study somnolence and sedation appeared early during treatment and were generally dose related. Other CNS depressants, including opioids, antidepressants, and alcohol, could potentiate these effects. Monitor patients for these effects and advise them not to drive or operate machinery until they have gained sufficient experience on ZTALMY to gauge whether it adversely affects their ability to drive or operate machinery. · Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including ZTALMY, increase the risk of suicidal thoughts or behavior. Monitor patients taking ZTALMY for the emergence or worsening of depression, suicidal thoughts or behavior, or any unusual changes in mood or behavior. Advise patients, caregivers, and their families to be alert for these behavioral changes and report behaviors of concern immediately to healthcare providers. When considering ZTALMY, or any other AED, balance the risk of suicidal thoughts or behaviors with the risk of untreated illness. If these symptoms emerge during treatment, consider whether it may be related to the AED or the underlying illness. · Withdrawal of Antiepileptic Drugs: As with most AEDs, withdraw ZTALMY gradually to minimize the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse event, rapid discontinuation can be considered. ADVERSE REACTIONS The most common adverse reactions (incidence of at least 5% and at least twice the rate of placebo) were somnolence (38%), pyrexia (18%), salivary hypersecretion (6%), and seasonal allergy (6%). DRUG INTERACTIONS Cytochrome P450 inducers will decrease ganaxolone exposure. Avoid concomitant use with strong or moderate CYP3A4 inducers; if unavoidable, consider a dosage increase of ZTALMY, but do not exceed the maximum recommended dosage. UGT inhibitors may increase ganaxolone exposure. Consider a reduction of ZTALMY maintenance dose when initiating UGT inhibitors. USE IN SPECIFIC POPULATIONS · Pregnancy: Use caution when ZTALMY is administered to pregnant women as there are no adequate data on the developmental risk associated with use in pregnant women. In animal studies, developmental adverse effects were observed following exposure during organogenesis or throughout gestation and lactation. · Lactation: ZTALMY is excreted in human milk at concentrations resulting in a dose to the breastfed infant of less than 1% maternal dose. The effects of ZTALMY on milk production and the breastfed infant are unknown. · Hepatic Impairment: Administration of ZTALMY in patients with severe hepatic impairment (Child-Pugh class C) results in elevated ganaxolone plasma concentrations. Therefore, dosage adjustment in these patients during titration and maintenance is required. No dosage adjustment is necessary in patients with mild (Child-Pugh class A) or moderate (Child-Pugh class B) hepatic impairment. DRUG ABUSE AND DEPENDENCE ZTALMY contains ganaxolone, a Schedule V controlled substance (CV). Advise patients of the potential for abuse and dependence. It is recommended that ZTALMY be tapered according to the dosage recommendations unless symptoms warrant immediate discontinuation. Please see full Prescribing Information. About Immedica Pharma US Inc. Immedica Pharma US Inc. is an affiliate of Immedica Pharma AB (collectively, "Immedica"), a pharmaceutical company headquartered in Stockholm, Sweden. Immedica is focused on the commercialization of medicines for rare diseases and specialty care products. Immedica's capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, 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 180 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.ImmedicaUS.com . Immedica contact: Linda Holmström Head of HR & Communication [email protected] 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 and RARE neurology. Read our CEO statement.

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