NASDAQ: Company Update. January 2015

Transcription

1 NASDAQ: Company Update January 2015

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," "plans," "expects," "will," "potential" and similar expressions are intended to identify forwardlooking statements. These statements are based on the company's current beliefs and expectations. These forward-looking statements include statements regarding Zogenix s cash position as of December 31, 2014, the expected timing of the FDA approval and the commercial launch of a Zohydro ER formulation with abuse deterrent properties, continued adoption of Zohydro ER and the potential to add a co-promotion partner, and the timing of the commencement of clinical trials for Relday and ZX008. 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 related to changes in estimated financial amounts based on the completion of financial closing procedures and the audit of the financial statements; 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; difficulties or delays relating to the development, testing, manufacturing and marketing of and obtaining regulatory approval for an abuse deterrent formulation of Zohydro ER or any of Zogenix s other product candidates; Zogenix's dependence on third parties to develop an additional formulation of Zohydro ER, Relday and ZX008; 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; difficulties in identifying, negotiating and carrying out a co-promotions agreement for Zohydro ER; and other risks described in the company's prior press releases and filings with the Securities and Exchange Commission. 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 release to reflect events or circumstances after the date hereof. All forwardlooking statements are qualified in their entirety by this cautionary statement. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of

3 Zogenix: Improving the Patient Experience Founded 2006 Roger Hawley and Dr. Stephen Farr as co-founders IPO Nov 2010 California HQ; 200 employees Developing and commercializing medicines in important disease areas CNS Focus including severe chronic pain, schizophrenia, Dravet syndrome (severe epilepsy) One commercial product and four development candidates Clinical development and regulatory expertise Commercial infrastructure in place 3

4 Developing and Commercializing Medicines in Important Disease Areas CHRONIC PAIN SCHIZOPHRENIA ONCE-MONTHLY DRAVET SYNDROME 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 Proprietary Subcutaneous Once-Monthly Antipsychotic Worldwide Rights Phase 1b Jan 2015 Worldwide Co-Development/ Commercialization Opportunity Dravet syndrome Product Candidate With Long-Term Data Orphan Drug designation in USA and EU Phase 3 3Q2015 4

5 Significant Cadence of Potential Value Creating Milestones Through snda Potential Approval Zohydro ER (AD Capsules ZX004) FDA PDUFA Jan 30, 2015 Relday Multi-Dose Clinical Trial Begins Jan 2015 Abuse Liability Studies Zohydro ER AD Capsules Begins Relday Multi-Dose Trial Data Results Q ZX008 Phase 3 Clinical Trial Begins Q Potential FDA approval of updated Zohydro ER label with AD claims 2016 Potential Zohydro ER Co-Promotion NDA for ADF ER Hydrocodone Tablets (ZX007) targeting 1H 2016 Potential Approval ADF ER Hydrocodone Tablets (ZX007) ZX008 Phase 3 Data Relday Phase 3 Clinical Trial ZX008 Target NDA 5

6 Oral, Extended Release Hydrocodone Acetaminophen-Free FDA Approved October 2013 Launched March 2014 Designed to be an alternative to IR-HC/APAP for severe chronic pain patients requiring around-the-clock opioid therapy Reduces risk of liver toxicity related to over-exposure of acetaminophen True 12-hour dosing allows more consistent pain relief Simple transition from IR-HC to ER-HC vs. switching to a different type of extended-release molecule such morphine or oxycodone Supplemental NDA for abuse deterrent formulation PDUFA January 30,

7 Zohydro ER Continued Quarterly Growth Zohydro ER Quarterly Results Total Prescriptions and Net Revenues 10,299 TRxs $2.4M 15,134 TRxs $4.1M ~19,000 TRxs (est) Approximately 26% growth over Q3 1,141 TRxs $0.3M Q1 14 Q2 14 Q3 14 Q4 14 Over 45,000 Prescriptions and 5,100 Prescribers from Launch Through December 31, out of 10 Prescriptions Approved by Payers 110 sales representatives (as of ) Symphony Health Solutions, Source PHAST Prescription Weekly, February December,

8 Abuse Deterrent (AD) Formulations for Hydrocodone ER in Development TWO Simultaneous APPROACHES Modification of Approved Capsule Formulation ZX004 Development of Innovative Tablet Formulation ZX007 Can be introduced to marketplace more rapidly Designed to be injection and insufflation resistant Supplemental application to approved Zohydro ER NDA FDA PDUFA Jan 30, 2015 Designed to add oral abuse resistance Separate NDA - referencing current approved NDA for safety and efficacy Bioequivalent - No Phase 3 Studies Required 8

9 ZX007 Hydrocodone ER Abuse Deterrent Characteristics Coated hard tablets to withstand chewing Micro 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 9

10 Recent Progress and Upcoming Milestones for Abuse Deterrent Formulations Modification of Approved Capsule Formulation ZX004 snda submitted: PDUFA Jan 30, 2015 Launch Q2 2015, if approved Intranasal HAL study recently initiated Further submission seeking additional AD label claims 1H 2015 Development of Innovative Tablet Formulation ZX007 Micro pellet and SAS components developed Complete PK study to select final formulation (March 2015 decision) Initiate required PK and HAL studies (2015) NDA submission (1H 2016) Extended IP runway (>2030) 10

11 ONCE-MONTHLY Once-Monthly Risperidone With A Unique PK Profile, Available as a Subcutaneous Injection for Treatment of Schizophrenia Proof of Concept Established Multi-dose Trial Enrollment Initiated Phase 3 Targeting 2016 Novel formulation with a differentiated product profile compared to existing long-acting injectables 11

12 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 Estimated $3 Billion US Market 2020 (Credit Suisse Securities Research, Dec 9, 2014) 12

13 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 13

14 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 initiated January 2015; top line data 3Q2015 Targeting End of Phase 2 FDA meeting end of

15 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 granted in Europe and U.S. Acquired from Brabant Pharma (Oct 2014) 15

16 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 16

17 Fenfluramine Fenfluramine was an anoretic agent used in the treatment of obesity, often used off-label with phentermine mg daily Withdrawn from worldwide markets in late 1990s / early 2000s 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) 17

18 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 >50% >70% >90% Seizure Free Reduction in Seizure Frequency 67% N=10 18

19 Established Regulatory Pathway to ZX008 Approval Meetings with FDA and European agencies provided specific regulatory guidance Phase 3 program to start in 3Q2015 Therapeutic doses already established in ongoing long-term study 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 19

20 Significant Cadence of Potential Value Creating Milestones Through snda Potential Approval Zohydro ER (AD Capsules ZX004) FDA PDUFA Jan 30, 2015 Relday Multi-Dose Clinical Trial Begins Jan 2015 Abuse Liability Studies Zohydro ER AD Capsules Begins Relday Multi-Dose Trial Data Results Q ZX008 Phase 3 Clinical Trial Begins Q Potential FDA approval of updated Zohydro ER label with AD claims 2016 Potential Zohydro ER Co-Promotion NDA for ADF ER Hydrocodone Tablets (ZX007) targeting 1H 2016 Potential Approval ADF ER Hydrocodone Tablets (ZX007) ZX008 Phase 3 Data Relday Phase 3 Clinical Trial ZX008 Target NDA 20

21 Financial Highlights Q Total Revenue (Zohydro ER revenue of $4.1M) Market Capitalization ( ~153,361,743 Common Stock Outstanding) Cash Balance as of (unaudited) Debt (1) as of $21.5M $8.8M $222M $42.2M plus $8.5M (escrow) Fully Diluted Shares (2) 186,506,823 Employees 200 (1) Includes $21.5 million of the $24 million debt facility and excludes $7m working capital advance from Endo International (2) Fully diluted shares as of 1/9/15 include options to purchase 16,912,883 shares and warrants to purchase 16,232,197 shares with weighted average exercise prices of $2.65 and $2.66 respectively 21

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