Company Update with a Focus on Pipeline

Transcription

1 NASDAQ: Company Update with a Focus on Pipeline December 2014

2 Forward Looking Statement Zogenix cautions you that statements included in this presentation that are not a description of historical facts are forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "indicates," "will," "intends," "potential," "suggests," "assuming," "designed" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include: the expected timing of the FDA review and action date and potential approval of the snda for Zohydro ER; the timing of the commencement of Phase 3 clinical trials for Brabafen and a multi-dose clinical and safety study for Relday; and the ability to secure debt financing based on the non-binding term sheet. Actual results may differ from those set forth in this release due to the risk and uncertainties inherent in Zogenix's business, including, without limitation: risks and uncertainties associated with regulatory review and approval of the snda, including the risk that additional information or data requests from the FDA could significantly delay the FDA s review period; Zogenix s dependence on the successful commercialization of Zohydro ER; Zogenix s ability to achieve broad market acceptance and generate revenues from sales of Zohydro ER; public concern regarding the safety of drug products such as Zohydro ER and the impact of negative publicity and political influences relating to the regulation of the pain management market in general and opioids and Zohydro ER in particular; competition from other pharmaceutical or biotechnology companies; risks associated with the acquisition of Brabant and integration of Brabant's operations into Zogenix's business, including an increase in near and long-term expenditures, exposure to unknown liabilities and diversion of Zogenix's management's time and attention; Zogenix s dependence on its contract manufacturers and its ability to ensure an adequate and continued supply of Zohydro ER to meet market demand; Zogenix s dependence on third parties to develop an abuse deterrent formulation of Zohydro ER, Relday and ZX008; the potential that earlier clinical trials may not be predictive of future results; Zogenix s ability to successfully enforce its marketing exclusivities and intellectual property rights, and to defend the patents covering Zohydro ER, including the potential for Paragraph IV litigation relating to the product; the potential product liability exposure associated with pharmaceutical products such as Zohydro ER and other products Zogenix may in-license or acquire; Zogenix s ability to fully comply with numerous federal, state and local laws and regulatory requirements that apply to its commercial activities; the risk that Zogenix will be unable to negotiate and enter into definitive agreements for a potential debt financing, on acceptable terms, or at all; Zogenix's ability to raise additional funding that it may need to continue to pursue its commercial and business development plans; and other risks detailed under "Risk Factors" and elsewhere in Zogenix s periodic reports and other filings made with the Securities and Exchange Commission from time to time. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Zogenix undertakes no obligation to revise or update this presentation to reflect events or circumstances after the date hereof, except as required by law. All forward-looking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of

3 Developing and Commercializing Medicines in Important Disease Areas CHRONIC PAIN DRAVET SYNDROME SCHIZOPHRENIA ZX008 (low-dose fenfluramine) Oral, 12-Hour Extended Release Hydrocodone without Acetaminophen US Launch March 2014 Potential abuse deterrent product approvals in 2015 and 2016 Dravet syndrome Product Candidate With Long-Term Data Orphan Drug designation in USA and EU Phase 3 3Q2015 Subcutaneous Once-Monthly Antipsychotic Worldwide Rights Phase 1b Jan 2015 Co-Development/ Commercialization Opportunity 3

4 Oral, Extended Release Hydrocodone Acetaminophen-Free FDA Approved October 2013 Launched March

5 Zohydro ER Commercial Update 7,000 Zohydro ER TRxs by Month 6,000 5,000 4,000 3,000 2,000 15,134 TRxs 3Q 2014, up 47% over 2Q ,000 0 February March April May June July August September October Over 35,000 Prescriptions and 4,600 Prescribers from Launch Through November 14, out of 10 Prescriptions Approved by Payers Symphony Health Solutions, Source PHAST Prescription Monthly, February October 2014 Symphony Health Solutions, Source PHAST Prescription Weekly, February November 14,

6 Abuse Deterrent (AD) Formulations for Hydrocodone ER in Development TWO Simultaneous APPROACHES Modification of Approved Capsule Formulation (ZX004) Development of Novel Tablet Formulation with Altus (ZX007) Designed to be injection and insufflation resistant Supplemental application to approved Zohydro ER NDA Designed to add oral abuse resistance Separate NDA - referencing current approved NDA for safety and efficacy No Phase 3 Studies Required 6

7 ZX007 Hydrocodone ER Abuse Deterrent Characteristics Coated hard tablets to withstand chewing Tiny pellets of hydrocodone to maintain ER release profile even when tablet is cut or crushed Super absorbent system (SAS) containing polymers quickly and spontaneously gel in many solvents 7

8 Recent Progress and Upcoming Milestones for Abuse Deterrent Formulations Modification of Approved Capsule Formulation (ZX004) Novel Tablet Formulation with Altus (ZX007) snda submitted: Target Action Date: Jan 30, 2015 Launch Q2 2015, if approved Initiate intranasal HAL study January 2015 Further submission seeking additional AD label claims H Pellet and SAS components developed Complete PK study to select final formulation (March 2015 decision) Initiate required PK and HAL studies (2015) NDA submission H Extended IP runway (>2030) 8

9 ZX008 Low-dose fenfluramine for the treatment of Dravet syndrome, a rare and catastrophic form of intractable epilepsy that begins in infancy Phase 3 Candidate Orphan drug designation in Europe and U.S. Acquired from Brabant Pharma 9

10 Dravet Syndrome Overview A rare form of intractable epilepsy in children with incidence between 1:20,000 and 1:40,000 Severe epileptic seizures in children starting in the first year of life Cognitive and developmental impairment Genetic condition associated with mutations of the SCN1A gene encoding a voltage-gated sodium channel High incidence of Status Epilepticus (severe, continuous seizures) and increased risk of Sudden Unexpected Death in Epilepsy (SUDEP) Unmet medical need impacting young children Cost of care is high with children requiring constant supervision and as adolescents long-term institutionalization No effective, long-term treatment for Dravet syndrome exists 10

11 Fenfluramine Fenfluramine, with or without phentermine, was an anoretic agent used in the treatment of obesity mg daily Withdrawn from worldwide markets in late 1990 s / early 2000 s because of an unacceptable risk of valvular heart defects Preclinical and preliminary clinical evidence of abolishing epileptic seizures first described in the 1980 s Academic investigators in Belgium continued under a Royal Decree to use low-dose fenfluramine as an adjunct to baseline antiepileptic drug therapy in Dravet patients 5-10 mg BID Peer reviewed publication 1 highlighted dramatic reduction in seizures 1 Ceulemans et al, Epilepsia (2012) 11

12 Longitudinal Analysis of Fenfluramine Trial in Dravet Patients Enrolled in the Belgian Study Latest assessment in 2014 Longest duration, most robust data set for an investigational drug in DS Average length of treatment >12 years; longest duration = 26 years Effectiveness (see graph): On average patients have been seizure free for approximately 6 years (1-19 years) Dramatic effects in patients stopping/ restarting therapy Safety: Mild/transient side effects loss of appetite, fatigue, somnolence No clinically significant findings related to cardiotoxicity [echocardiography] 100% 80% 60% 40% 20% 0% 93% N=14 ZX008 Responder Analysis at Last Assessment (n=15) 93% N=14 87% N=12 67% N=10 >50% >70% >90% Seizure Free 12

13 Established Regulatory Pathway to ZX008 Approval Meetings with FDA and European agencies provided specific regulatory guidance Phase 3 program to start in 3Q2015 Multicenter, double-blind, parallel group, placebo-controlled trial of fenfluramine as an adjunct therapy in Dravet syndrome One study in EU, one in US (N=40-60) Orphan designation granted in Europe and US Breakthrough Therapy potential Specific REMS to support and maintain a long-term favorable risk-benefit profile 13

14 Once-Monthly Risperidone With A Unique PK Profile, Available as a Subcutaneous Injection for Treatment of Schizophrenia Phase 1b Jan 2015 Proof of Concept Established 14

15 RELDAY Product Profile vs. Current LAI Atypical Antipsychotics (AA) Substantial Market Research Supports Key Features Physicians Want KEY ATTRIBUTES FROM PHYSICIAN RESEARCH RISPERDAL CONSTA (RISPERIDONE) INVEGA SUSTENNA (PALIPERIDONE) ZYPREXA RELPREVV (OLANZAPINE) ABILIFY MAINTENA (ARIPIPRAZOLE) RELDAY (RISPERIDONE) Therapeutic Levels on Day 1 No Loading Dose/ Oral Supplementation 1X Monthly Subcutaneous No Reconstitution Highly Concentrated U.S. Market with 11,000 Psychiatrists Prescribing LAI Products Growing U.S. and EU Market for LAI Products 15

16 RELDAY Program Status REGULATORY STRATEGY PRODUCT ATTRIBUTES DEMONSTRATED IN PROOF OF CONCEPT STUDY Confirmed 505(b)(2) NDA in US Abridged Hybrid MAA likely in EU First IND Study Subjects with Schizophrenia (n=40) Pharmacokinetics (PK) Therapeutic Levels within 8 hours of Dosing First Day Levels Consistent with Comparable Oral Dose Duration Consistent with Once-monthly Dosing Dose-Proportionality Across the Entire Therapeutic Range Safety /Tolerability No Unique Safety or Tolerability Issues with SC Dosing Injection Site Reactions Were Mild and Spontaneously Resolved Multi-dose Simulations Show Steady State Blood Levels Fall within the Therapeutic Range and with No Requirement for Loading Doses 16

17 RELDAY Next Clinical Study Non-clinical chronic toxicology studies successfully concluded Results support conduct of next clinical study Open label, multiple dose safety and PK study 4 Groups: 60, 90, 120mg RELDAY + Risperdal Consta (active control) Subjects with schizophrenia followed for up to 20 weeks Study initiation January 2015; top line data 3Q2015 Targeting End of Phase 2 FDA meeting 4Q

18 Significant Cadence of Potential Value Creating Milestones Through snda Potential Approval Zohydro ER (AD capsules) Relday Multi-Dose Clinical Trial Begins Abuse Liability Studies Zohydro ER AD capsules Begins Potential Zohydro ER Co-Promotion Relday Multi-Dose Trial Data Results Brabafen Phase 3 Clinical Trial Begins Updated Zohydro ER label with AD claims 2016 Brabafen EU Approval & Altus ADF Zohydro ER NDA Potential Approval Zohydro ER (AD tablets) NDA for Altus ADF Zohydro ER ZX008 Phase 3 Data Relday Phase 3 Clinical Trial Brabafen Target NDA 18

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