- Physician reports show signals of efficacy and safety consistent with previously disclosed data, as well as maintenance of clinical effect beyond 12 weeks and high retention rate - - Data to be presented at R&D Day today commencing at 10:00 a.m. ET, 15:00 BST -
Highlights
London, UK; 14 October 2014: GW Pharmaceuticals plc (Nasdaq: GWPH, AIM: GWP, “GW,” “the Company” or “the Group”), a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, today announced new physician reports of efficacy and safety associated with the Epidiolex® “expanded access” studies to be presented today at the Company’s R&D Day event in New York City. Physician reports of efficacy and safety data have been made available on 58 children and young adults with treatment-resistant epilepsy who have been treated with GW’s investigational cannabidiol (CBD) product candidate, Epidiolex, for a period of at least 12 weeks. This includes 27 patients on whom data was reported in June 2014 and an additional 31 patients on whom data has recently been made available to the Company. In addition, of the 58 total patients, 40 patients have been treated for at least 16 weeks and data has been available for this time period also. These data are from three hospital sites in the United States and were generated under expanded access Investigational New Drug applications (INDs) authorized by the U.S. Food and Drug Administration (FDA). In addition, physician reports of safety data were made available on 151 patients (58 patients with 12 weeks treatment effect data plus 93 additional patients for whom 12 week treatment effect data is not yet available).
The patients suffer from a range of treatment-resistant epilepsies. Many have extreme and rare forms of epilepsy including several patients with major congenital structural brain abnormalities.
“I am very encouraged with the preliminary results from our open-label study of Epidiolex. I think they show very promising signals of safety and efficacy in patients, which include some of the most difficult epilepsy cases we follow in our program,” stated Dr. Elizabeth Thiele, Director of the Pediatric Epilepsy Program at Massachusetts General Hospital and Harvard Professor of Neurology. “Based on my experience thus far, I believe that Epidiolex has the potential to be an important advance in treatment for these treatment-resistant children and will likely have a significant role as a future therapy. I believe these data fully support advancing into formal clinical development, and we are very excited to participate with GW in the upcoming placebo-controlled trials in Dravet and Lennox-Gastaut syndromes.”
“We are pleased to report that the data on additional children treated with Epidiolex is consistent with the promising signals of efficacy previously seen in our initial data released in June. It is also encouraging to see Epidiolex lead to a maintained reduction in seizure frequency beyond the initial 12 week treatment period and that approximately 95% of patients who have commenced treatment to date remain on therapy,” stated Justin Gover, GW’s Chief Executive Officer. “We believe that these signals of efficacy, together with the side effect profile observed to date, serve to reinforce our confidence in Epidiolex as we commence our placebo-controlled clinical trials in both Dravet syndrome and Lennox-Gastaut syndrome.”
Available Epidiolex Data
Clinical effect data were made available on 58 patients whom have been treated with Epidiolex for at least 12 weeks, of which 40 patients have been treated for at least 16 weeks. The 58 patients were predominately children with an average age of 11 years. In all cases, Epidiolex was added to current anti-epileptic drugs (AEDs). On average, patients were taking approximately 3 other AEDs.
The largest single type of epilepsy was Dravet syndrome (n=12) and data is presented separately for these patients. In addition, data is presented for all patients with drop seizures at baseline (n=12), the types of seizures considered by the FDA in assessing primary efficacy for Lennox-Gastaut syndrome (LGS) trials.
Treatment effect data have been presented to show median percent changes in seizure frequency during the first, second, third and fourth months of treatment compared with seizure frequency during a 4 week baseline observation period. Data are also presented which compares percent change in the average 4 week seizure frequency throughout the first 12 week treatment period with seizure frequency during a 4 week baseline observation period, a calculation that is consistent with the FDA’s recommended endpoint for evaluating efficacy. Additionally, data are presented in the form of responder analyses, showing the proportion of patients achieving responses of greater than or equal to 50%, 70%, 90% and those patients that are seizure-free.
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