Zogenix (ZGNX) 8-K Regulation FD Disclosure

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

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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

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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