Positive Impacts on Non-Seizure Symptoms, Caregiver Experience Observed With Soticlestat


Using a caregiver questionnaire, noted improvements in verbal, non-verbal communication, and engagement were observed on soticlestat treatment.

Mahnaz Asgharnejad, PharmD, vice president, Global Program Leader, Takeda Pharmaceuticals

Mahnaz Asgharnejad, PharmD

New 2-year data from the open-label extension (OLE) of the phase 2 ENDYMION 1 study (NCT03635973) showed that treatment with soticlestat (Takeda Pharmaceutical), an antiseizure medication, was associated with high rates of caregiver-reported satisfaction. Across non-seizure symptoms/impacts faced by patients with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS), the highest levels of caregiver-reported improvement were in verbal and non-verbal communication.1

ENDYMION 1 is a multi-site, open-label, extension study assessing the long-term safety and tolerability of soticlestat, also known as TAK-935, in patients with developmental and epileptic encephalopathies like LGS and DS. In the trial, patients receive soticlestat twice a day, with site visits every 2-6 months throughout the study. The new analysis, presented at the 2023 American Epilepsy Society (AES) annual meeting, held December 1-5, in Orlando, Florida, evaluated caregiver experience of treatment satisfaction and impacts on non-seizure symptoms with long-term use of soticlestat.

Led by Mahnaz Asgharnejad, PharmD, vice president, Global Program Leader, Takeda Pharmaceuticals, 62 caregivers of patients with LGS or DS on the drug completed at least 1 questionnaire item of the Experience Questionnaire at 1 study visit. Patient and caregiver experience was evaluated every ~26 weeks, with the analysis focusing on responses after ~2 years of treatment.

At week 104, 44 caregivers responded to the questionnaire, with a majority reporting overall satisfaction with soticlestat (75.0%) and its administration (72.7%). More specifically, 42.4% (14 of 33) of those satisfied with treatment were reportedly extremely or very satisfied while 59.4% (19 of 32) of those satisfied with administration were considered extremely or very satisfied. Notably, one-fifth (20.5%) of caregivers reported improvements in alertness, and most reported no change or improvement across symptoms/impacts.

READ MORE: Long-Term CBD Treatment in Tuberous Sclerosis Complex Effective Regardless of Age

Based on questionnaire responses, caregivers noted improvements in verbal communication (46.9%), non-verbal communication (45.5%), and engagement (39.5%) at 104 weeks. Most caregivers who reported improvement in these 3 items rated the change as meaningful (verbal: 14 of 15; non-verbal: 17 of 20; engagement: 11 of 17). The study also allowed caregivers the option to include free-text responses. Using these responses, the most frequently mentioned positive impacts on day-to-day quality of life were on communication (17 mentions by 12 caregivers), and interaction and engagement (12 mentions by 8 caregivers).

ENDYMION is an open-label extension to ELEKTRA, a previously conducted phase 2 placebo-controlled study assessing 300 mg or less of twice daily, weight-adjusted soticlestat in children with DS and LGS. Published in Epilepsia in 2022, the trial enrolled 141 participants, 126 (86%) of which completed the study. ELEKTRA achieved its primary end point: the combined soticlestat-treated population demonstatrated a placebo-adjusted media reduction in seizure frequency of 30.21% during the maintenance period (n = 139; P = .0008).

Similar reductions were observed over the full 20-week treatment period, with soticlestat-treated patients demonstrating a placebo-adjusted median reduction in seizure frequency of 25.06% (95% CI, –42.48% to –10.69%; P = .0024). At the last study visit, responses on Clinical Global Impression-Clinician assessment indicated marked improvement in more patients on soticlestat than those on placebo (DS: 26.9% vs 8.0%; LGS: 27.9% vs 11.1%). These results were reflected by caregivers, with Care GI-C responses that indicated improvement in more patients on active treatment (DS: 57.7% vs 32.0%; LGS: 51.2% vs 28.9%).

Click here for more coverage of AES 2023.

1. Asgharnejad M, Andrews JS, Mitra P, et al. Caregiver-reported overall treatment experience and change in non-seizure symptoms/impacts in the ENDYMION1 study of soticlestat (TAK-935) in individuals with Dravet or Lennox-Gastaut syndrome. Presented at: 2023 AES annual meeting; December 1-5; Orlando, FL. Abstract 1.282
Related Videos
Shadi Yaghi, MD, associate professor of neurology at Brown University
Matthew B. Harms, MD, MDA Medical Advisor and Care Center Director, Associate Professor of Neurology at Columbia University Irving Medical Center - Eleanor and Lou Gehrig ALS Center.
Ava L. Liberman, MD, medical director of the Stroke Center at Weill Cornell Medicine
Svetlana Blitshteyn, MD, FAAN, director and founder of Dysautonomia Clinic
© 2024 MJH Life Sciences

All rights reserved.