Brabant Pharma, a pharmaceutical company focused on advancing Brabafen, its novel, low-dose formulation of fenfluramine for the treatment of Dravet syndrome, announces that clinical data supporting the use of low-dose fenfluramine to treat Dravet syndrome will be highlighted in an oral presentation at the Dravet Syndrome Foundation's First Biennial Family and Professional Conference taking place June 26-29, 2014 at Lurie Children's Hospital in Chicago.
Lieven Lagae, M.D., Professor of Pediatric Neurology at the University of Leuven (Belgium) and current President of the European Pediatric Neurology Society, will present the latest follow-up results to a longitudinal analysis of up to 26 years of clinical data from a continuing prospective study of 15 children and adults with Dravet syndrome who were treated with low-dose fenfluramine for an average of 12.4 years. Prof. Lagae will host a session titled "Fenfluramine in the Treatment of Dravet Syndrome: Long-Term Data," on Friday, June 27 at 2:00 p.m. Central time at the Conference.
These data provide a further update to earlier published results (Epilepsia, 2012-V53: 1131-1139) from the same continuing prospective study of 12 children and adults with Dravet Syndrome, who were treated with low-dose fenfluramine. Highlights of the data include:
Clinical Effect Data - 12 Patients
-Longest duration of treatment - 26 years
-Average duration of treatment - 14.9 years
-83% of patients (n=10 out of 12) were seizure free at the latest assessment
-Average seizure free period - 6.6 years
-92% of patients (n=11 out of 12) had greater than 75% reduction in seizure frequency at last assessment
Highlights including three additional patients incorporated since 2011, include:
Clinical Effect Data - 15 patients
-Longest duration of treatment - 26 years
-Average duration of treatment - 12.4 years
-67% of patients (n=10 out of 15) were seizure free at the latest assessment
-Average seizure free period - 5.5 years
-87% of patients (n=13 out of 15) had greater than 75% reduction in seizure frequency at last assessment
Low-dose fenfluramine was shown to be well tolerated and side-effects of treatment were mild and transient for the entire 26-year study period. There were no reports of pulmonary hypertension and there were no deaths. Two patients showed sub-clinical evidence of cardiac valve thickening that was judged to be clinically insignificant by cardiologists. Similar findings spontaneously resolved in a third patient.
"We are especially pleased to be reporting continued positive and durable long-term results for low-dose fenfluramine to treat Dravet syndrome. Current treatment options, as well as treatments in development, have not demonstrated the ability to achieve the significant seizure-free status and reduction in seizure frequency demonstrated in this study. Importantly, these robust clinical outcomes have been maintained for many years, which is a critical achievement for patients suffering from this debilitating and life-threatening disease," stated Anthony Clarke, Ph.D., Chief Scientific Officer of Brabant Pharma.
"We are particularly pleased with the long-term safety profile of low-dose fenfluramine in this high-risk patient population, which has been shown to be safe and effective with no treatment-limiting adverse events. We are keenly aware of the historic risk associated with high doses of fenfluramine combined with phentermine for the treatment of obesity. Consequently, we have a rigorous risk-management program in place to closely monitor patients and will continue to maintain these programs post-marketing. Importantly, we have not seen any clinically-relevant cardiac events in our 26 years of clinical experience with low-dose fenfluramine in Dravet syndrome," added Dr. Clarke.
"These positive data and the durability of response are very compelling. We are fully committed to bringing Brabafen to market for the benefit of these children and their families," noted Rick Stewart, Chief Executive Officer of Brabant Pharma.
"Toward that end, we are actively engaged in dialogue with representatives at the U.S. Food and Drug Administration and the European Medicines Agency to establish a regulatory path forward for Brabafen to treat Dravet syndrome. Based on our decades of human clinical experience with Brabafen in this orphan indication, it is our hope, and the hope of those with Dravet syndrome and their families, that we can accelerate the initiation of Phase 3 studies in order to continue studying this promising therapeutic," concluded Mr. Stewart.
About the Dravet Syndrome Foundation's Conference
The Dravet Syndrome Foundation's First Biennial Family and Professional Conference brings together Dravet syndrome families and caregivers with leading clinicians and researchers to discuss new developments in the treatment of the syndrome and associated intractable childhood epilepsies. The event features more than a dozen clinical topics, as well as parent-to-parent roundtables and opportunities for families to socialize. Brabant Pharma has established a Scholarship Program to cover conference registration and hotel costs for families in need of financial assistance.
About Dravet Syndrome
Dravet syndrome (also known as Severe Myoclonic Epilepsy of Infancy) is a rare, severe and therapy-resistant form of epilepsy most often caused by an identifiable gene defect that results in abnormal functioning of a sodium channel in the brain. Children with Dravet's syndrome experience severe, long-lasting, fever-related seizures in the first year of life. Other seizures typically arise later, including myoclonus (involuntary muscle spasms) and status epilepticus (prolonged seizures), which oftentimes result in severe cognitive and developmental impairment. Episodes of status epilepticus require immediate emergency care and can be fatal. Individuals with Dravet syndrome face a higher incidence of SUDEP (sudden unexplained death in epilepsy). Children with Dravet syndrome do not outgrow this condition and it affects every aspect of their daily lives.
Unless a cure or better treatments for Dravet syndrome and related epilepsies are found, individuals with these disorders face a diminished quality of life. Current treatment options are limited and the prognosis for these children is poor. The constant care and supervision of an individual with such highly specialized needs is emotionally and financially draining on the family members who care for these individuals. For more information visit: www.dravetfoundation.org.
About Brabant Pharma
Brabant Pharma is a privately held company focused on advancing the development of Brabafen, a low-dose formulation of fenfluramine for the treatment of Dravet syndrome. The company was founded in 2013 by bringing together 25 years of research and long-term clinical experience in the treatment of Dravet syndrome by world-leading experts along with a highly experienced management team to deliver a transformational treatment for Dravet patients. Brabant is working closely with regulatory authorities and clinical experts to accelerate the clinical trial timetable and time to regulatory approval. Brabant has received Orphan Drug designation for Brabafen in both the U.S. and Europe. Brabant has met with representatives of the U.S. Food and Drug Administration and the European Medicines Agency, and expects to commence Phase 3 clinical trials in the U.S. and Europe. For more information visit: www.brabantpharma.com
Anne Marie Fields
Senior Vice President