Zogenix, Inc. (NASDAQ:ZGNX), a pharmaceutical company developing therapies for the treatment of central nervous system (CNS) disorders, today announced new data demonstrating sustained effectiveness and cardiovascular-related safety for patients treated with ZX008 (low-dose fenfluramine) as an adjunctive therapy for seizures associated with Dravet syndrome. The data were presented at the 69th Annual American Epilepsy Society Meeting, taking place this week in Philadelphia, PA(see study data here and here). Zogenix expects to initiate a Phase 3 program for ZX008 in 2015. ZX008 is designated as an orphan drug in both the United States and Europe for the treatment of seizures associated with Dravet syndrome.

“In this new cohort of patients, we continue to achieve meaningful seizure control in Dravet syndrome patients using low-dose fenfluramine as adjunctive treatment,” said Professor Berten Ceulemans, from the University of Antwerp, Belgium, and one of the authors of the poster regarding the study. “The most recent data corroborate both the effectiveness and the safety observations demonstrated in the original cohort of patients, which were published in 2012. We look forward to the start of Zogenix’s ZX008 Phase 3 program evaluating low-dose fenfluramine as a potential adjunctive treatment for seizures associated with Dravet syndrome.”

“The improvement in seizures and cardiovascular-related safety data from this ongoing open-label study continues to be quite compelling,” said Bradley Galer, M.D., Chief Medical Officer of Zogenix. “These data further support ZX008’s potential as a safe and effective adjunct treatment for uncontrolled seizures associated with Dravet syndrome. Dravet syndrome patients and their families remain in need of viable treatment alternatives for this devastating condition that causes frequent, severe and potentially life-threatening seizures that are often unresponsive to standard anti-epileptic medications. As such, we remain focused on advancing our preparations for the start of our Phase 3 program for ZX008, which we expect to initiate in 2015.”

The data presented highlight the initial results from a new cohort of 7 Dravet syndrome patients who began add-on treatment with low-dose fenfluramine (5 mg to 15 mg per day) at various starting points between 2010 and 2014.  Median treatment duration was 0.9 years (range 0.2 to 3.9 years). During the 90-day run-in period prior to initiating low-dose fenfluramine treatment, the median frequency of tonic-clonic seizures was 3.0 per month (range 0.4 to 39.7). At the 6-month evaluation after starting low-dose fenfluramine treatment, the median frequency of tonic-clonic seizures was 1.2 per month, and the median decrease was 73% (range 48-100%).  Over the entire observation period, the median frequency of tonic-clonic seizures was 0.9 per month, and the median decrease was 84% (range 55-100%).

A separate poster reported that during this observation period from 2010 to 2015, treatment with low-dose fenfluramine was generally well-tolerated, and in this new cohort of patients, treatment for periods of 0.9 to 3.9 years did not result in any echocardiographic or clinical signs of cardiac valve abnormalities, pulmonary hypertension or any other cardiovascular abnormalities. The most common treatment-related adverse events were mild-to-moderate somnolence (n=6) and anorexia (n=4). There were no fenfluramine discontinuations due to adverse events or lack of effect.

The observed effectiveness, tolerability and cardiovascular-related safety with add-on, low-dose fenfluramine in this new cohort of Dravet syndrome patients extends the findings previously reported in the original cohort in 2012¹.(Original Source)

Shares of Zogenix closed last Friday at $15.22, up $0.24 or 1.60%. ZGNX has a 1-year high of $21.65 and a 1-year low of $8.64. The stock’s 50-day moving average is $13.21 and its 200-day moving average is $15.37.

On the ratings front, Zogenix has been the subject of a number of recent research reports. In a report issued on November 11, Brean Murray Carret analyst Difei Yang maintained a Buy rating on ZGNX, with a price target of $28, which represents a potential upside of 84.0% from where the stock is currently trading. Separately, on November 10, William Blair’s Tim Lugo reiterated a Hold rating on the stock .

According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Difei Yang and Tim Lugo have a total average return of 0.9% and -3.1% respectively. Yang has a success rate of 42.7% and is ranked #1662 out of 3645 analysts, while Lugo has a success rate of 35.7% and is ranked #3030.

The street is mostly Bullish on ZGNX stock. Out of 4 analysts who cover the stock, 3 suggest a Buy rating and one recommends to Hold the stock. The 12-month average price target assigned to the stock is $23.00, which implies an upside of 51.1% from current levels.

Zogenix Inc is a pharmaceutical company engaged in commercializing & developing therapies that address clinical needs for people living with pain-related and CNS disorders that needs treatment alternatives to help return to normal daily functioning.