Thursday, December 1, 2016

Zogenix, Inc. (ZGNX) Develops Orphan Drug for Dravet Syndrome Epilepsy

Zogenix, Inc (NASDAQ: ZGNX) is a clinical stage pharmaceutical company committed to the development and commercialization of therapies for central nervous system disorders that address specific clinical needs for people living with orphan diseases. The company’s lead product candidate, ZX008, is currently being evaluated for the treatment of one such disease – Dravet Syndrome.

Despite the name, orphan diseases affect adults as well. The soubriquet ‘orphan disease’ arose because pharmaceutical companies showed no interest in ‘adopting’ them due to their comparatively small market size. However, the Orphan Drug Act (ODA) of 1983 created financial incentives for drug and biologics manufacturers to devote resources to developing treatments for such diseases, including tax credits for costs of clinical research, government grant funding, and assistance for clinical research. Most importantly, the FDA promised to give a seven-year period of marketing exclusivity to the sponsor of an orphan-designated product who first obtains market approval for that indication.

Dravet Syndrome is a rare genetic epileptic encephalopathy (brain disorder) that, typically, begins during the first year of life. Up until 1989, the condition was referred to as either epilepsy with polymorphic seizures, polymorphic epilepsy in infancy (PMEI) or severe myoclonic epilepsy in infancy (SMEI).

In June, Zogenix initiated the second phase III clinical trial, a multi-national study, of ZX008 as an adjunctive treatment of seizures in children with Dravet Syndrome. ZX008 is designated as an orphan drug in both the U.S. and Europe, and received Fast Track designation in the U.S. for the treatment of Dravet syndrome.

Zogenix is also exploring the efficacy of ZX008 (Fenfluramine) in treating Lennox-Gastaut Syndrome (LGS). LGS is a particularly debilitating type of epilepsy that, in many cases, impairs intellectual development. Sufferers exhibit different types of seizures, particularly tonic (stiffening) and atonic (drop) seizures. Although LGS accounts for only two to five percent of childhood epilepsies, its seizures are hard to control and require life-long treatment.

Interim data from that efficacy study will be presented at a poster session, or presentation of research information or findings, on December 3 at the 70th Annual Meeting of the American Epilepsy Society. Data covering eight weeks from the open label dose finding study will be made public in a presentation entitled ‘Effectiveness and Tolerability of Low Dose Fenfluramine (ZX008) in Lennox Gastaut Syndrome: A Pilot, Open-Label Dose Finding Study’.

A recent report from Aegis Capital ( has put a price target of $28.00 on Zogenix. The stock is currently trading at about $12.80 on the NASDAQ under the symbol ZGNX. The current valuation of $329.8 million based on outstanding shares of 24.8 million at $13.30, the market price at November 23, is based on an 80 percent chance of success in the clinical development process of ZX008 for the treatment of Dravet Syndrome.

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