Company Presentation I September 2018

Transcription

1 NASDAQ: OPNT Company Presentation I September 2018 Development Stage Specialty Pharma Company focused on Addiction and Drug Overdose

2 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These forward-looking statements relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our or our industry s actual results, levels of activity, performance or achievements to be materially different from any future results, levels of activity, performance or achievements expressed, implied or inferred by these forwardlooking statements. In some cases, you can identify forwardlooking statements by terminology such as may, will, should, could, would, expects, plans, intends, anticipates, believes, estimates, predicts, projects, potential, or continue or the negative of such terms and other comparable terminology. These forwardlooking statements are only predictions based on our current expectations and projections about future events. You should not place undue reliance on these forward-looking statements. Actual events or results may differ materially. In evaluating these forward-looking statements, you should specifically consider various factors. These and other factors may cause our actual results to differ materially from any forward-looking statement. We undertake no obligation to update any of the forward-looking statements after the date of this presentation to conform those statements to reflect the occurrence of unanticipated events, except as required by applicable law. 2

3 Investment highlights Development stage specialty pharma company with a focus on addiction and drug overdose and a growing revenue stream via royalties from net sales of NARCAN nasal spray. Lead asset OPNT003 is a long-acting, rapid onset nalmefene nasal spray for opioid overdose reversal. 505(b)(2) development path to NDA filing in $7.4m grant from NIH to support development. Developed & licensed NARCAN Nasal Spray to ADAPT Pharma. NARCAN royalties and sales milestones provide funds to develop pipeline and help de-risk balance sheet. OPNT001, a naloxone nasal spray, in Phase 2 for Bulimia Nervosa. Addressable market of $1B+ with little competition 1. Enrollment completed - top line results expected 1Q19. Emergent BioSolutions announced $735M purchase of ADAPT. Highlights the value of NARCAN and the potential value of our pipeline. OPNT002, nasal naltrexone for Alcohol Use Disorder (AUD) entering Phase 2. On demand dosing without prior abstinence requirement and harm reduction endpoint. 1 Data provided by Return on Focus report for Company 3

4 Management team has extensive addiction and CNS drug development experience Roger Crystal, MD, MRCS, MBA Chief Executive Officer Phil Skolnick, PhD, DSc (hon) Chief Scientific Officer David O Toole, CPA Chief Financial Officer Mark Ellison, PhD VP, Development, Manufacturing and Quality Quan Vu VP, Corporate Development 4

5 We continue to build upon our first marketed product with a robust addiction and drug overdose pharmacotherapy pipeline Indication Product Candidate / Regulatory Pathway Pre-clinical Phase 1 Phase 2 Phase 3 NDA FDA Approval Partner Opioid Overdose NARCAN Nasal Spray / 505(b)(2) Opioid Overdose OPNT003* Nalmefene Nasal Spray / 505(b)(2) Bulimia Nervosa OPNT001 Multi-dose Naloxone Nasal Spray / 505(b)(2) Alcohol Use Disorder OPNT002 Opioid Antagonist Nasal Spray / 505(b)(2) Opioid Use Disorder OPNT006 Opioid Antagonist Implant Opioid Use Disorder OPNT005 Heroin vaccine: hapten + liposome adjuvant *NIDA awarded approximately $7.4m in grant funding for the development of OPNT003 (intranasal nalmefene) 5

6 Opiant s expertise using opioid antagonists and nasal spray technology is being leveraged across many indications Opioid Antagonists Naloxone Naltrexone Nalmefene Opioid Antagonist (undisclosed) Nasal Spray Nasal Spray Nasal Spray Implant Delivery Method Opioid Overdose Eating Disorders Alcohol Use Disorder (AUD) Opioid Overdose Opioid Use Disorder (OUD) Indication 2019 Net Sales > $200M 1 1M Bulimia patients in the US M patients in the US Synthetics involved in >50% overdose deaths more potent, longer acting agents needed 2017 Vivitrol Sales $269M Market highlights Only program licensed to Adapt Pharma OPNT001 Phase 2 study in Bulimia Nervosa ongoing OPNT002 Phase 1 complete; Phase 2 activities in 1Q 2019 OPNT003 Pilot PK data: rapid onset, long half life 505(b)(2) Feasibility studies underway 1 Emergent BioSolutions Press Release dated 8/28/18: 2 T. Udo and C. Grillo: Biological Psychiatry 84: , See slide 24 for sources 6

7 Opioid Overdose

8 Opioid overdose is a significant public health crisis, that has now evolved to a fentanyl crisis Drugs Involved in U.S. Opioid Overdose Deaths, 1999 to ,000 25,000 Synthetic Opioids other than Methadone, 29,406 (predominantly fentanyl) Opioid overdose deaths continue to increase in Dramatic increase in deaths from synthetic opioids, especially fentanyl 1 20,000 15,000 10,000 5,000 0 Heroin, 15,958 Natural and semi-synthetic opioids, 14,958 Methadone, 3,295 Fentanyl ~50-fold more potent than heroin 2 Leading cause of death in American adults under the age of 50 3 Opioids have become the analgesic of choice unlike anywhere else in the world Fentanyl 50x= see slide 12 for sources

9 Substantial treatment population for opioid overdose reversal agents FIRST RESPONDERS 2.5M OPIOID ADDICTS 1.6M taking methadone, Suboxone ** CO-PRESCRIBING MARKET: 245M Opioid painkillers prescribed to over 20M patients*** x $90 per pack* = ~$2B Addressable Market* Patients who are prescribed opioids are at risk of an overdose and safe prescribing should ensure patient access to a reversal agent *Company estimates including Opiant estimate of average selling price of NARCAN Nasal Spray ** Skolnick; Ann Rev Pharm Tox 2018 *** Volkow and McLellan, NEJM

10 Opiant developed the NARCAN Nasal Spray, which saves lives from opioid overdose With 77% of opioid overdose deaths occurring outside of a medical setting, important for everyone to have access to naloxone, as recommended by Surgeon General 1 : CVS 2 and Walgreens 3 pharmacies stock NARCAN Nasal Spray Aetna, Harvard Pilgrim zero copay on commercial policies 4,5 Needleless Effective Reliable Drug Delivery Easy to Use NARCAN Co-prescribing rules already in Vermont, Virginia, and Arizona

11 OPNT003 Nasal Nalmefene for Opioid Overdose

12 As the opioid crisis has evolved into a fentanyl crisis, NIH calling for stronger, longer-acting formulations of antagonists 1 As the opioid crisis has evolved, 55% of FATAL opioid overdoses 2 now involve fentanyl and/or fentanyl analogs Fentanyl is longer acting and 50x more potent than heroin 3 which may require more naloxone to initially resuscitate a patient with a risk of relapse (renarcotization) once naloxone wears off 4 The use of naloxone may be particularly problematic in rural areas (~70% of U.S. land mass) where access to EMS may be delayed 5 Fentanyl has been used as a chemical warfare agent 6 1 Volkow, ND and Collins, F. NEJM 377: , CDC/Health alert network 3 Skolnick, P. Eur J Pharmacol. 835: , Kaplan, et al., Ann Emerg Med. 34:42-50, 1999; Wang, et al. J. Emerg Med. 16: ,

13 OPNT003, Nasal Nalmefene, is a potent, long acting opioid antagonist with rapid onset, better suited for fentanyl overdose OPNT003 has ideal properties to address the fentanyl crisis because: Nalmefene is more potent than naloxone 1 Nalmefene has a 5-fold higher affinity (binds more tightly) to brain mu-opioid receptors and hence, may be more effective at reversing high potency opioids like fentanyl 2. Nalmefene is longer acting than naloxone The FDA approved injectable nalmefene has a half life of ~10h, whereas naloxone, has a half-life of 1-2 hours 3. Data generated in the Phase 1 study demonstrates OPNT003 also has a significantly longer half life ( hours) than naloxone. Nasal Nalmefene combined with Intravail is rapidly absorbed OPNT003 contains Intravail, a proprietary absorption enhancer delivering rapid increases in nalmefene plasma levels. Data generated in the Phase 1 study demonstrated a T max of 0.25 hours. By comparison, the T max of Narcan Nasal Spray (4 mg) is 0.5 hours 3. Onset of action is one of the factors in determining whether a patient survives an overdose. 1 Skolnick, P. Eur J Pharmacol. 835: , Yong, Z., Gao, X., Ma, W., Dong, H., Gong, Z., Su, R., Nalmefene reverses carfentanil-induced loss of righting reflex and respiratory depression in rats. Eur. J. Pharmacol. 738, , Krieter, et al., J Clin Pharmacol, 56, ,

14 Nalmefene Blood Levels (ng Nalfemene/mL Plasma) Phase 1 data for OPNT003, demonstrates rapid absorption and a long half life compared to injectable nalmefene IN + Intravail IN IM Rapid absorption of OPNT003 yet therapeutic levels persist beyond 4h KEY TAKEAWAYS OPNT003 contains nalmefene and Intravail, which accelerates absorption. Opiant has global exclusive license for Intravail. Plasma levels sufficient to achieve ~90% mu opioid receptor occupancy Study Overview Study performed under a CTA with NIDA using 13 healthy volunteers with intranasal arms blinded.* * Company data on file Hours 14

15 Having developed NARCAN Nasal Spray, the same team will aim to deliver OPNT003 to NDA in 2 years Development pathway confirmed and team able to execute Met with FDA, 505(b)(2) confirmed. This is a development plan that parallels that of NARCAN Nasal Spray. Government endorsement and non dilutive funding NIH grant confirms the unmet need and confidence in this approach. $7.4M NIH grant funds the majority of the work required to file an NDA expected in Product protection Very favorable PK profile of OPNT003 appears to be largely dependent on Intravail; Opiant has global exclusive rights for use with all opioids antagonists. Additional patents filed. Large addressable market, confirmed by 2019E net sales of NARCAN Nasal Spray > $200M OPNT003 is an easy-to-use nasal formulation of nalmefene with a rapid onset and long duration of action. Non-medically trained persons will be able to administer. 15

16 The FDA has confirmed a 505(b)(2) pathway for approval of OPNT003, aiming for NDA filing in Q Q Q Q Q Q Q Q Q Q Q Q 2020 NIDA Grant $7.4m (April) Formulation Development Stability testing to ensure suitable product shelf-life FDA Meeting protocol accepted Pivotal PK study NDA Filing Note: timeline events based on Company s expectations 16

17 OPNT001 Nasal Naloxone for Bulimia Nervosa

18 Although there are 1M patients with Bulimia Nervosa in the USA, only one drug is approved Bulimia Nervosa (BN) Bingeing, followed by purging 1M in USA 1 Severe complications if untreated Compulsive bingeing addictive behavior 1M IN THE US Fluoxetine only approved drug limited efficacy 1 T. Udo and C. Grillo: Biological Psychiatry 84: ,

19 Patient from Phase 2 study vignette daily diary 2 Weetabix and skim milk 1 pear 4 hot cross buns with butter 2 packs of Rolos 2 bags McCoy crisps 3 slices toast with Nutella 2 fresh ice cream Eclairs bacon sandwich with 1 x 175g yoghurt 1 x 400ml frijj chocolate milkshake chicken and chips with peas 1 nectarine Approximately 10 chocolate bourbon biscuits 2 bar Kit-Kats Bowl of chocolate shredder with milk and sugar 2 ring doughnuts 4 Cadbury Freddo bars 19

20 OPNT001 potentially offers significant advantages over competitors for Bulimia Nervosa Criteria OPNT001* (Nasal Naloxone) Fluoxetine (Prozac) Vyvanse (often used off label for BN) Overall Efficacy*?? ** Minimal adverse events As needed administration Absorption likely during purging Not a scheduled drug No tapering at discontinuation * - at efficacy specified in target product profile ** - not approved for Bulimia Nervosa Sources: Vyvanse PI, 1/2017, Prozac PI, 1/2017; Nasal naloxone TPP, 2017; McElroy, 2012; Guerdjikova, 2016; Forman 12/2016; FBNCSG, 1992, Crow,

21 OPNT001 is in Phase 2 for Bulimia Nervosa, with data readout expected 1Q 2019 Trial Type Randomized, double-blind, placebo-controlled multi-site study of OPNT001 in patients suffering from Bulimia Nervosa in the UK Study Size N=86 (completed patient enrollment in September 2018) Protocol Patients instructed to take nasal spray, on demand whenever the urge to binge Primary Endpoint Reduction in binge eating days Duration 12 weeks with data readout expected in 1Q

22 Self-commercializing for Bulimia Nervosa is attractive Validated Unmet Need Prescribers Positive Towards OPNT001 Opiant Can Access Prescribers $1B addressable market and market research supports significant potential to increase treatment rates using a therapy that works rapidly, effectively, and prevents relapse 1 Limited competitors and sparse pipeline Strong positive reaction to OPNT001 (vendor research of 20 psychiatrists) Shire s market conditioning efforts using Vyvanse in Binge Eating Disorder have prepared psychiatrists to use branded drugs to address bingeing Prescribers (psychiatrists with eating disorder practice) are welldefined and reachable with a focused sales force Potential for label expansion for Binge Eating Disorder indication Potential to license rights into primary care providers, and expand market 1 Data provided by Return on Focus report for Company 22

23 OPNT002 Nasal Naltrexone for Alcohol Use Disorder (AUD) 23

24 Despite alcohol abuse rates increasing, existing drugs are poorly tolerated and require specialist prescribing 0.4M 1 Patients with AUD currently receiving pharmacotherapy 1.4M 2 Patients with AUD seeking treatment 16.3M 2 Individuals in the US meeting DSM-V diagnostic criteria for AUD More effective & accessible pharmacotherapy expected to increase # of patients taking medication leading to improved outcomes 1 Data generated under contract with The Nemetz Group 2 Sources: - Center for Behavioral Health Statistics and Quality. (2015). Behavioral health trends in the United States: Results from the 2014 National Survey on Drug Use and Health (HHS Publication No. SMA , NSDUH Series H- 50). - Retrieved from estimate (8.4% of AUD); NIAAA: Understanding the impact of alcohol on human health and well-being ( accessed 10/06/16); - Reprinted from Drug and Alcohol Dependence 74(3). Bridget F. Grant, Deborah A. Dawson, Frederick S. Stinson, S. Patricia Chou, Mary C.Dufour, and Roger P. Pickering. The 12-Month Prevalence and Trends in DSM IV Alcohol Abuse and Dependence: United States, and , pp , Percentage change in first-year members of Alcoholics Anonymous, +1.5& CAGR, , used as proxy; source orange-page.org 24

25 PRN dosing, a harm reduction endpoint and no prior abstinence means OPNT002 addresses limitations of existing medication Existing Pharmacotherapies for AUD OPNT002 Advantage Abstinence Requirement Best outcomes require abstinence prior to commencing medication Taken whenever patients have the urge to drink ( as-needed basis ) Abstinence Outcome Abstinence as only acceptable outcome for FDA approval FDA accepts harm reduction as an outcome measure Adherence Low adherence for existing medication On-demand dosing regimen, where improved compliance is expected 25

26 Rapid nasal absorption vs oral ensures that the maximum amount of drug is present when it is most needed Naltrexone Blood Levels: Oral vs Nasal vs Nasal with Intravail Nasal naltrexone absorption improved with Intravail Faster nasal absorption vs oral Absorption of oral naltrexone is minimal at 5-10 minutes Drinking alcohol causes the release of endorphins (endogenous opioids) Naltrexone, an opioid antagonist, blocks endorphins released by alcohol Patients reduce heavy drinking by blocking the endorphin reward Source: study performed under a CTA with NIDA in 14 healthy volunteers. 26

27 Approved drugs for Alcohol Use Disorder have limitations, capping their commercial success potential Opiant Existing Pharmacotherapies* Criteria OPNT002* (Nasal Naltrexone) Acamprosate (Campral) Disulfiram (Antabuse) Naltrexone HCl (Oral: ReVia / Generics) Naltrexone HCl (Injection: Vivitrol ) As-needed use basis Can be used this way, but not current US practice Well-tolerated - Safety - Craving impact potential - Long-term harm reduction potential safe drinking Abstinence recommended prior to treatment initiation *Based on company assessment by Nemetz Group and advisory board input 27

28 FDA supports OPNT (b)(2) development route likely lower cost and quicker than an NCE OPNT002 Development Overview Meeting with FDA completed 505(b)(2) regulatory pathway confirmed 2018 Further optimization of formulation 1Q 2019 Phase 2 activities 2020 Enter Phase 3 Finish commercial assessment potential to selfcommercialize and target treatment centers, and sublicense primary care rights 28

29 Finance and Milestones 29

30 NARCAN Nasal Spray is licensed to Adapt Pharma and Opiant is eligible to receive annual royalties Schedule of Annual Royalty Rates Net Sales Royalty Rate Up to $50M 6% $50M-$75M 7.5% $75M-$100M 9% $100-$200M 10% >$200M 12% Potential annual payments to Opiant based on 2019 revenue guidance given by purchaser of Adapt Purchaser of Adapt provided guidance of $220 million to $250 million for calendar year 2019 One time milestone of $13.5 million based on Net Sales above $200 million, in one calendar year Annual royalty stream of approximately $18 million to $21 million 30

31 Financial highlights ($ millions) Three months ended June 30, 2018 Mar 31, 2018 Dec 31, 2017* Sources of Funds = Strong Financial Position Cash Balance $11.2 $11.3 $8.1 Debt None None None Revenue $3.2 $1.7 $11.7 Common Shares Outstanding Annual Royalty Stream of approximately $20 million (does not include one-time milestone of $13.5 million that should be earned in either 2018 or 2019) NIDA grant of $7.4 million that will be used for development plan for OPNT003 Potential funds from grants by other government agencies Fully Diluted Share Count 6.3 million of which 2.15 million held by Board and Management Current cash on the balance sheet of $11.2 million *In December 2017, Opiant changed its year end from July 31 to December 31 31

32 Expected milestones across the portfolio over next 12 months 3Q Q Q Q H 2019 NARCAN 3Q Royalty 4Q Royalty 1Q Royalty 2Q Royalty 3Q/4Q Royalty OPNT001 (Bulimia) Last patient enrolled Last patient last visit Phase 2 data readout OPNT002 (AUD) Phase 1 formulation selection study Phase 2 activities OPNT003 (OOR) Formulation development & stability Continuation of formulation development & stability Pivotal PK Study NDA preparation 32

33 Investment highlights Development stage specialty pharma company with a focus on addiction and drug overdose and a growing revenue stream via royalties from net sales of NARCAN nasal spray. Lead asset OPNT003 is a long-acting, rapid onset nalmefene nasal spray for opioid overdose reversal. 505(b)(2) development path to NDA filing in $7.4m grant from NIH to support development. Developed & licensed NARCAN Nasal Spray to ADAPT Pharma. NARCAN royalties and sales milestones provide funds to develop pipeline and help de-risk balance sheet. OPNT001, a naloxone nasal spray, in Phase 2 for Bulimia Nervosa. Addressable market of $1B+ with little competition. Enrollment completed - top line results expected 1Q19. Emergent BioSolutions announced $735M purchase of ADAPT. Highlights the value of NARCAN and the potential value of our pipeline. OPNT002, nasal naltrexone for Alcohol Use Disorder (AUD) entering Phase 2. On demand dosing without prior abstinence requirement and harm reduction endpoint. 33

34 NASDAQ: OPNT Company Presentation I September 2018 Focused on Addiction and Drug Overdose