Attached files
| file | filename |
|---|---|
| 8-K - 8-K - ZOGENIX, INC. | d57715d8k.htm |
 1 NASDAQ: July 14 th , 2015 ZX008 Development Update Recent FDA correspondence (Type C meeting) clarified requirements for ZX008 IND and Phase 3 program Exhibit 99.1 |
 CMC and Non-Clinical Aspects
CMC • Drug substance synthetic pathway, starting material and validation strategy
confirmed • General agreement with drug substance and drug product specification attributes – Phase 3 supplies: drug substance manufactured; liquid oral formulation
manufactured and on stability
Non-clinical
• IND to initiate Phase 3 studies requires histopathology data (particularly on
heart valves) from the recently completed juvenile toxicology dose-range
finding study
– Data complete and clean; full report in July – IND submission on track for end of August • FDA confirmed reproductive and developmental toxicity and carcinogenicity
studies can be conducted as post-marketing commitments
2 |
 Clinical and Regulatory Aspects
Clinical • FDA agreed that Zogenix’s proposal of two double-blind, randomized,
controlled Phase 3 studies plus one long-term, open label extension study
should be sufficient to support registration, pending review of
study outcomes
• FDA favored a fixed, multiple-dose, parallel arm, placebo-controlled design
• Dosing in mg/kg will provide similar exposures across age/weight range Regulatory • Confirmed 505(b)(2) NDA submission is acceptable • Fast Track Designation application encouraged once IND is active 3 |
 ZX008 Phase 3 Study Design 8 WEEKS BASELINE OBSERVATION 12 WEEKS TREATMENT N = 105 Ages 2 – 18 Two identical studies, one US and one multinational Q4 2015 initiation Open Label Safety Study ZX008 High dose ZX008 Low dose Placebo Initial Screen 4 |
 Pivotal Study Objectives
Efficacy Endpoints
• Primary Endpoint – % change in convulsive seizures from baseline • Key Secondary Endpoints – Responder analysis (Primary endpoint for EU study) – Convulsive seizure-free interval • Other Secondary Endpoints – Episodes of status epilepticus – Caregiver Global Impression of Change – QoL Safety Assessment • AEs • Cardiac – Periodic ECG and Doppler echocardiograms 5 |
 Back-up only
6 |
 ZX008 Phase 3 Study Design Submitted to FDA UP TO 28 DAYS 4 WEEKS BASELINE OBSERVATION 4-12 WEEKS TITRATION 12 WEEKS MAINTENANCE Titrate to Next Dose if <75% Reduction in Seizure Frequency N = 56 Ages 2 - 18 Open Label Safety Study Option Placebo Placebo ZX008 ZX008 2.5 mg, 5 mg, 10 mg Screening 7 |