Zogenix Presents New Data at Virtual AAN 2021 Showing Improved Executive Function in Lennox-Gastaut Syndrome (LGS) Patients Treated with FINTEPLA® (Fenfluramine) Oral Solution
- LGS is one of the most challenging developmental and epileptic encephalopathies to manage because of high seizure frequency and significant neurodevelopmental impairment.
- More LGS study patients treated with FINTEPLA (fenfluramine) showed improvement in each of the BRIEF 2 indexes that have been used to assess behavior, emotion, and cognitive function in intractable epilepsy and other developmental conditions.
- Improvement was statistically significant for Cognition (27% vs 13% placebo, p=0.046) and the Global Executive Composite score (25% vs 11% placebo, p=0.034), where approximately twice as many patients achieved clinically meaningful improvement.
The data, presented in an oral presentation and fireside chat at the virtual
"FINTEPLA-treated children and young adults with LGS showed improved overall self-regulation, with specific improvements in regulation of attention and cognition after 14 weeks in a Phase 3 double-blind randomized controlled trial,” said
The objective of this post-hoc analysis was to assess the impact of FINTEPLA (fenfluramine) on everyday executive function as captured using the Behavior Rating Inventory of Executive Function (BRIEF®) as part of the
This analysis included 137 patients (age 6-18) that had both baseline and end of-study BRIEF assessments (median age of 12 years), mapping the BRIEF ratings to the current BRIEF® 2 parent form, a shorter, 63-item version, which incorporates a large, 1,400-sample size normative population and statistics to support interpretation. This analysis did not include patients over 18 years of age because normative population data in adults is not yet available. The BRIEF 2 analysis utilizes the Reliable Change Index (RCI) for Behavior, Emotion, Cognition, and Global scores that represent a degree of confidence that the change represents a real effect (versus test-related errors and practice effects), and therefore is clinically meaningful. Associations between change in BRIEF 2 scores and combined active (0.2 and 0.7 mg/kg/day fenfluramine) versus placebo treatment groups were evaluated in cross tabulations using Somers’ D statistic.
Mean age of patients in the FINTEPLA (n=92) vs. placebo (n=45) groups was 12 ± 3.6, with 44% of the FINTEPLA patients being female vs. 53% in the placebo group. The frequency of clinically elevated T-scores at baseline, defined as a T-score ≥65, was substantial, suggesting frequent impairment in executive function, as would be expected for children with LGS. Subjects with greater improvement in seizure control were more likely to show clinically meaningful improvements in executive functioning after the 14-week study duration.
More patients on FINTEPLA (fenfluramine) than placebo showed improvement in each of the four BRIEF 2 indexes (Behavior, Emotions, Cognition, and Global Executive Composite). Improvement was significantly greater for the CRI (Cognition) and GEC (Global Executive Composite) scores, indicating clinically meaningful improvement in the FINTEPLA (fenfluramine)-treated patients.
|Percentage of Patients Showing Clinically Meaningful Improvement
(RCI ≥95% Certainty) in Active vs Placebo Treatment Groups
|BRIEF® 2 Index||FINTEPLA®
|GEC (Global Executive Composite overarching summary score)||25%||11%||0.034|
“Analyses from our separate Phase 3 trials and long-term open label studies in Dravet syndrome, another difficult to treat rare epilepsy, showed that FINTEPLA provided sustained, clinically meaningful improvements in seizure control and executive function,” said
About Lennox-Gastaut Syndrome
Lennox-Gastaut Syndrome (LGS) is a rare and devastating lifelong childhood-onset epilepsy that can arise from multiple different causes. LGS is characterized by many different seizure types, including many that result in frequent falls and injuries and that often don't respond to currently available seizure medications The intellectual and behavioral problems associated with LGS, as well as around-the-clock care requirements, add to the complexity of life with this disease.
Zogenix cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “indicates,” “will,” “intends,” “potential,” “suggests,” “assuming,” “designed,” and similar expressions are intended to identify forward-looking statements. These statements are based on Zogenix’s current beliefs and expectations. The inclusion of forward-looking statements should not be regarded as a representation by Zogenix that any of its plans will be achieved. Actual results may differ from those set forth in this release due to the risks and uncertainties inherent in patient outcomes and other risks described in Zogenix’s prior press releases as well as in public periodic filings with the U.S. Securities & Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Zogenix undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
Senior Director, Corporate Communications
+1 (510) 788-8732 | firstname.lastname@example.org
Managing Director, LifeSci Advisors LLC
+1 (212) 915-2578 | email@example.com
+1 (978) 390-1394 | firstname.lastname@example.org
Source: Zogenix, Inc