Marinus discontinuing oral ganaxolone trial development to treat seizures in children
The TrustTSC trial did not achieve its primary endpoint of percent reduction in 28-day frequency of TSC-associated seizures.
Marinus discontinuing oral ganaxolone trial development for treatment of seizures in children | Image Credit: © SewcreamStudio - © SewcreamStudio - stock.adobe.com.
Per a press release from Marinus Pharmaceuticals, the company is discontinuing further clinical development of oral ganaxolone to treat seizure disorders associated with tuberous sclerosis complex (TSC) in children, after it did not achieve statistical significance on the primary endpoint of the phase 3 TrustTSC trail (NCT05323734).
According to Marinus, oral ganaxolone did not meet the primary endpoint of percent change in 28-day TSC-associated seizure frequency. In the phase 3, randomized, double-blind, placebo-controlled trial that included oral ganaxolone treatment among children and adults with TSC-related epilepsy (n = 129), median reduction was 19.7% for ganaxolone compared to 10.2% for placebo (P = 0.09). Ganaxolone featured a safety profile consistent with previous clinical trials, with the most frequent adverse even being somnolence (28.1% for ganaxolone vs 16.9% for placebo).
The pharmaceutical company stated it will continue to and invest in the commercial growth of ganaxolone (ZTALMY) oral suspension CV, currently FDA-approved to treat seizures associated with CDKL5 deficiency disorder in patients aged 2 years and older. Among this population, more than 200 patients are receiving treatment, stated Marinus.
"Ganaxolone is a neuroactive steroid GABAA receptor modulator that acts on a well-characterized target in the brain known to have anti-seizure effects," according to Marinus. "It is a prescription medicine that has been approved by the [FDA] and the European Commission for appropriate patients with CDKL5 deficiency disorder."
“As the first controlled trial in TSC that allowed enrollment of patients taking a range of concomitant antiseizure medications, which included mTOR inhibitors and cannabidiol, these data represent a significant advancement in our understanding of the use of ganaxolone with other standard of care treatments,” said Scott Braunstein, MD, chairman and CEO of Marinus, in a statement. “We are disappointed that the results of the TrustTSC trial are not likely to be sufficient for an sNDA filing. We are deeply grateful for the contributions of the clinical trial investigators, patients, and caregivers whose support and participation made the study possible.”
Reference:
Marinus Pharmaceuticals Announces Topline Results From Phase 3 TrustTSC Trial of Oral Ganaxolone in Tuberous Sclerosis Complex and Commences Process to Explore Strategic Alternatives. Marinus Pharmaceuticals. Press release. October 24, 2024. Accessed October 24, 2024. https://www.businesswire.com/news/home/20241024735870/en/Marinus-Pharmaceuticals-Announces-Topline-Results-From-Phase-3-TrustTSC-Trial-of-Oral-Ganaxolone-in-Tuberous-Sclerosis-Complex-and-Commences-Process-to-Explore-Strategic-Alternatives
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