Zogenix, Inc. (NASDAQ:ZGNX), a pharmaceutical company developing therapies for the treatment of central nervous system (CNS) disorders, announced new data which continues to demonstrate the sustained effectiveness and cardiovascular safety of ZX008 (low-dose fenfluramine) as an adjunctive therapy for seizures associated with Dravet syndrome. In addition, for the first time, data were presented on patient and caregiver sleep quality and quality of life. The podium presentation was given at the 14th International Child Neurology Congress (ICNC), taking place this week inAmsterdam, The Netherlands. ZX008 is designated as an orphan drug in both the U.S. and Europe, and recently received Fast Track designation in the U.S., for the treatment of Dravet syndrome.
The data presented highlighted the updated results from the new patient cohort, which now includes 9 Dravet syndrome patients. All of these patients began add-on treatment with low-dose fenfluramine (5 mg to 20 mg per day) at various starting points between 2010 and the end of January 2016. Median treatment duration was 1.5 years (range 0.3 to 5.1 years). During the 90-day run-in period prior to initiating low-dose fenfluramine treatment, the median frequency of major motor seizures (defined as tonic, clonic, tonic-clonic, atonic, and myoclonic seizures lasting >30 seconds) was 15.0 per month (range 0.4 to 39.7). Over the entire observation period, the median frequency of major motor seizures was reduced to 1.5 per month, and the median decrease was 75% (range 28-100%). Six of the 9 patients had at least a 70% reduction in major motor seizures.
In addition, parents/caregivers were asked to rate both their child’s and their own sleep quality and quality of life using 0-10 scales where 0 = extremely bad and 10 = very good. At the most recent visit, mean sleep quality reported for patients and parents was 8.1/10 and 7.9/10, respectively, while mean Quality of Life scores were 7.4/10 for both groups.
In this new cohort of patients, treatment with low-dose fenfluramine continued to be generally well-tolerated, and did not result in any echocardiographic or clinical signs of cardiac valve abnormalities, pulmonary hypertension or any other cardiovascular abnormalities. The most common treatment-emergent adverse events were mild-to-moderate somnolence (n=6), diminished appetite (n=4), mood changes (n=2), and non-convulsive status epilepticus (n=2). There were no fenfluramine discontinuations due to adverse events or lack of effect.
“The continued meaningful reduction in seizure frequency and sustained cardiovascular safety demonstrated in these updated results for the new cohort of patients further support our confidence in the potential of ZX008 as a safe and effective treatment for seizures associated with Dravet syndrome,” said Bradley Galer, M.D., Chief Medical Officer of Zogenix. “We believe the relatively high levels of sleep quality and quality of life reported reflect the seizure control in these patients. Our Phase 3 program for ZX008 in Dravet syndrome is currently enrolling patients in the U.S., and will begin enrolling patients in Europe shortly.”
The observed effectiveness, tolerability and cardiovascular-related safety with add-on, low-dose fenfluramine in this new cohort of Dravet syndrome patients further extends the findings initially reported for the original twelve subjects in 2012 and the initial report from this new cohort in December of 2015. (Original Source)
Shares of Zogenix closed yesterday at $9.91, down $0.37 or -3.60%. ZGNX has a 1-year high of $21.65 and a 1-year low of $7.90. The stock’s 50-day moving average is $10.16 and its 200-day moving average is $11.45.
On the ratings front, Brean Murray Carret analyst Difei Yang maintained a Buy rating on ZGNX, with a price target of $28, in a report issued on April 21. The current price target represents a potential upside of 182.5% from where the stock is currently trading. According to TipRanks.com, Yang has a yearly average return of -14.1%, a 28.7% success rate, and is ranked #3797 out of 3827 analysts.