Continuous therapy. Because life goes ON. Jefferies Global Healthcare Conference June, 2016

Transcription

1 Continuous therapy. Because life goes ON Jefferies Global Healthcare Conference June,

2 Disclaimer These slides and the accompanying oral presentation contain forward-looking statements concerning our business and financial performance and condition, as well as our plans, objectives and expectations for our business, operations and financial performance and condition. In some cases, you can identify forward-looking statements by terms including anticipates, believes, could, estimates, expects, intends, may, plans, potential, predicts, projects, should, will, would, and similar expressions intended to identify forward-looking statements. Forward-looking statements reflect our current views with respect to future events and are based on assumptions and subject to risks and uncertainties, including those set forth in our annual report on Form 20-F for the year ended December 31, 2015 filed with the U.S. Securities and Exchange Commission. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. You should not unduly rely on any forward-looking statements. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that future results, levels of activity, performance and events and circumstances reflected in the forward-looking statements will be achieved or will occur. Except as required by law, we undertake no obligation to update publicly any forward-looking statements for any reason after the date of this presentation, to conform these statements to actual results or to changes in our expectations. These forward-looking statements speak only as of the date of this presentation, and we assume no obligation to update or revise these forward-looking statements for any reason. The trademarks included herein are the property of the owners thereof and are used for reference purposes only. Such use should not be construed as an endorsement of the products or services of the Company. 21 2

3 Price/IP protection Who we are New formulations of existing drugs Allow new administration routes that overcome current limitations New drug with significant clinical advantages High Innovative High NCE-like barriers to entry grant de facto exclusivity Generics High price point Low Low Cost / Timeline / Risk High Accelerated lower risk regulatory pathway (505b2), short time to market 21

4 Pipeline: LD/CD expected to launch in 2018 Product Candidate Indication Preclinical Phase I Phase II Phase III ND0612H ND0612L Severe Parkinson s Disease Moderate Parkinson s Disease LD/CD, Subcutaneous LD/CD, Subcutaneous ND0701 Severe Parkinson s Disease Apomorphine, Subcutaneous ND0801 CNS disease Cognition disorders Nicotine and Opipramol, Transdermal 21 4

5 ON/OFF time 21 5

6 Blood Levels of Levodopa Levodopa, the Gold Standard, has a major drawback: short half life Mild Moderate Severe Dyskinesia On Time Fluctuations Off state Levodopa Administration ND0612 The brain needs steady levels of dopamine Oral LD has a short half life Instead of steady LD levels oral LD creates fluctuations Almost all patients develop fluctuations in 2-5 years Continuous delivery the greatest challenge of levodopa therapy 21 6

7 Efforts to improve continuous LD therapy - limited success Attempts to achieve continuity but no Game Changers: Why? LD has only existed to date in solid form that must be administered through the GI tract Why has LD not been developed to enable alternative routes of administration that provide better continuity? Because it was thought impossible; previous attempts to formulate LD into liquid forms have failed NeuroDerm is the 1 st to formulate LD into a liquid formulation 21 7

8 Advanced patients face highly invasive treatment alternatives Deep brain stimulation Intra-duodenal LD/CD pump (Duodopa) Removal post infection 15% Seizures 2.4% Device sales: ~$500M (excluding surgery) Cognition impairments 41% Cost per patient: up to 100,000 1 st yr, ~$25,000 yr/5yr Peritonitis 3% Complication of insertion 40% Europe sales: $220M Cost per patient: $70,000/yr Wound infection 21% 21 8

9 Lead candidates introduce a new paradigm in PD treatment ND0612H Severe PD The 1 st liquid LD/CD replacing surgical treatments Device CE approved in EU >350,000 patients US and EU ND0612L Moderate PD The 1 st liquid LD/CD drug A new SOC in PD Device CE approved EU > 900,000 patients US and EU ND0612L Moderate PD 2 nd generation delivered through a patch pump One and done operation Device in development 21 9

10 Clinical development 21 10

11 Clinical guidance committee & scientific advisory board Warren Olanow, MD Karl Kieburtz, MD, MPH Peter A. LeWitt, MD John Nutt, MD Nir Giladi, MD Olivier Rascol, MD Werner Poewe, MD 21 11

12 ND0612L Moderate patients 21 12

13 Mean Plasma LD Concentration (ng/ml) Phase I and Phase IIa ND0612L achieves steady LD levels with linear dose proportionality Phase I µl/h 120 µl/h 160 µl/h 200 µl/h 240 µl/h n=30 Time after infusion initiation 21 13

14 ND0612L Phase II study design Randomization 30 moderate to severe patients 2:1 Oral SOC Pre-Randomized 16 patients 1:1 ND0612L+oral SOC Period 1: Randomized double blind 2 weeks Placebo + oral SOC ND0612L Period 2: Open, 1 week ND0612L+Ent Primary end points Secondary end points Exploratory efficacy end points Safety Tolerability PK LD dose adjustment Pump usability OFF time ON time w/o troublesome dyskinesia AIMS Quality of sleep (PDSS) Quality of life (PDQ-39) Disease severity (CGI-C) 21

15 Plasma LD (ng/ml) Phase II first period pharmacokinetics ND0612L stabilizes LD plasma concentration above an average of ~800ng/ml Baseline Best Current Oral Therapy (Placebo) ND0612L (Best Current Oral Therapy) (Adjunct to Oral Therapy) Day 0 Day 14 Day n= Time (h) Peak to trough Fluctuation index ND0612L transforms levodopa PK in PD patients 21 15

16 Plasma LD (ng/ml) Phase II second period pharmacokinetics ND0612L stabilizes LD plasma concentration An average steady plasma levodopa concentration of 550ng/ml was maintained with ND0612L alone, and 800ng/ml when combined with oral entacapone Baseline ND0612L ND0612L+Entacapone (Standard of care) Day 0 Day 21 Day n= n=6 n= Time (h) ND0612L transforms levodopa PK in PD patients 21 16

17 Change from baseline (hr) Change from baseline (hr) ND0612L reduces OFF time w/o increasing dyskinesia (in clinic) Improves motor fluctuations without paying the penalty of troublesome dyskinesia OFF time Troublesome dyskinesia Placebo (n=11) ND0612L (n=18) -3.5 Placebo (n=11) ND0612L (n=18) 2 Hours reduction in OFF time 41% vs. 9% in the placebo Reduction in troublesome dyskinesia 21 17

18 % of patients improved (CGI-C score) Mean change in PDSS score Mean change in PDQ-39 score ND0612L improves quality of sleep, quality of life and global clinical disease severity Quality of sleep Quality of life Placebo (n=11) ND0612 (n=19) 30% improvement in quality of sleep 30% vs. 0.9% compared to placebo -10 Placebo (n=11) ND0612 (n=19) 17% improvement in quality of life 17% vs. 5% compared to placebo Global clinical improvement 100% 80% 90% 60% 40% 20% 0% 36% Placebo (n=11) ND0612 (n=19) 90% improvement in disease severity 90% vs. 36% compared to placebo 21 18

19 Good safety profile In volunteers and PD patients so far: No systemic or local irritation, all patients elected to continue to the open label extension phase Draize score slightly elevated, slight pruritus, similar in both groups, normalized at week 3 Skin local safety in healthy volunteers - no local irritation SC nodules (0.5-1cm) resolving spontaneously No particular systemic AE No patient discontinued early Local safety similar to Phase I Transient and reversible local minor reaction 21

20 ND0612H Severe patients 21 20

21 Plasma LD (ng/ml) Plasma LD (ng/ml) Phase IIa results: ND0612H reaches high LD plasma levels - May replace surgery in the majority of severe patients Steady ND0612H levels alone and with oral entacapone (1x/4h) ND0612L ND0612H 1,454 1, Alone With entacapone ND0612H - Average of all patients Time (hr) Oral LD/CD ND /7.5 mg/ml ND /14 mg/ml ND /14 mg/ml +Entacapone 21 21

22 Commercial development 21 22

23 Advanced PD patients a hidden segment with the largest unmet need One in one hundred people over age 60 >6 million PD patients WW >60% Moderate to severe PD, >60% of all patients PD treatment market $3.8B Breakdown by severity (2011) Severe 16% Early 42% Moderate 42% Drugs: ~$3.3B; many are generic Devices: <1% of PD patients are treated Cost of treatment for moderate patient: $6,000/yr Cost of treatment for severe patient: $70,000/yr 21 23

24 ND0612L/H serve moderate and severe PD patients the segments with the largest unmet need Only <1% of severe PD patients in the US and EU are treated by advanced treatments but spend approx. $800M* (excluding surgery) US & EU Total PD patients 2,200,000 Moderate PD patients ~900,000 Severe PD patients ~350,000 DBS new patients/yr ~22,000 Duodopa patient base ~3,500 Duodopa new patients/yr ~700 Apomorphine patient base*** ~4,000 Severe patients not treated with advanced treatments >300,000 *DBS, Duodopa and continuous apomorphine ** 10,261 & 6,986 net yearly patient base growth plus estimated replacement of deletions from the patient base ***Continuous apomorphine treatment 21 24

25 PD gatekeepers ND0612H Number of treating physicians Dozens of KOLs 1500 Movement Disorder Specialists 14K Neurologists ND0612L Number of treating physicians Dozens of KOLs 1500 Movement Disorder Specialists 14K Neurologists 21 25

26 Positive patient, physician and payer feedback Patients I have been living in a dream and I want this dream to continue Phase II clinical trial patient Physicians * This is great, its everything that we re looking for US Neurologist Payers # PD patients face more and more issues related to uniform drug absorption I would expect Medicare will allow both products at the discretion of the doctors US Payer *A company-sponsored third-party market research report of 12 neurologists in the United States, May 2014, Trinity Partners # A third-party sponsored research of 12 payers from Germany, France and United Kingdom, April # A third-party report of 7 payers from US, December

27 Positioning - efficacy without safety tradeoff High efficacy Duodopa DBS ApoGo ROW ND0701 ND0612H ND0612L High invasiveness, Severe side effects Oral LD/CD Improved Oral LD/CD Low side effects, Not invasive Oral DAs Oral MAO inhibitors Limited efficacy 27 21

28 Development plan 21 28

29 ND0612H - clinical development plan C L I N I C A L E F F I C A C Y Study 006 Phase II 36 patients, 4w (ongoing) Study 010 Phase III 80 patients, 16w NDA Study 012 Long term safety* 50 patients, 12m P K S I M I L A R I T Y Study 005 Pilot PK, 24 HV (ongoing) Results expected in Q2/16 * Single safety study for H and for L, total of 100 patients Study 009 Definitive PK HV MAA 21 29

30 ND0612L - clinical development plan C L I N I C A L E F F I C A C Y Study 007 Phase III patients, 12w NDA Study 012 Long term safety 50 patients, 12m * Single safety study for H and for L, total of 100 patients 21 30

31 Development timelines ND0612H Dose finding PK study (pilot), 005 (24 healthy volunteers, ongoing) Definitive PK, 009 (20-40 healthy volunteers) EMA submission Phase II (30 patients, ongoing) Phase III (80 patients) NDA submission ND0612L Long term safety, 012 (100 patients*) Phase III pivotal, 007 ( patients) NDA & EMA submission * Agreed with FDA for US development, will be discussed with EMA 21 31

32 Milestones ND0612H ND0612L Initiation of a long-term safety study (012) Completion of a PK study vs. Duodopa (005) FDA end of Phase II meeting; EMA meeting Completion of a Phase II study (006) Initiation of a BE study vs. Duodopa, EU (009) Initiation of a Phase III (010) EMA submission FDA submission End of Phase II meeting Initiation of pivotal study (007) Initiation of a long term safety study (012) EMA & FDA submission 2018 EU and US approval EU and US approval 21 32

33 ND

34 ND continuous SC apomorphine Continuous SC apomorphine, available outside the US, is indicated for advanced PD patients who don t respond well to levodopa High efficacy major limitations Apomorphine benefits Highly effective Rapid onset (3-5 minutes) Current commercial apomorphine limitations Low local tolerability and high local pain Large daily volume 12hr infusion limit per injection site - inconvenient - no night treatment ND0701 overcomes current products major limitations Photograph of abdomen with apomorphine infusion (Hazel et al., 2008) 70% of dropout in the clinical trials was due to local site reaction (Ribaric., 2012) 21 34

35 ND0701 overcomes major limitations of current commercial apomorphine A new well tolerated, 24hr treatment of apomorphine 0.5% Apo-Go 2.5% ND0701 Low volume - 5x reduction of daily volume (patch pump) A new standard for apomorphine drug therapy Necrosis Active Granulomatous Inflammatory Reaction 21 35

36 Financial profile Raised gross proceeds of $45 million in IPO of 4,500,000 ordinary shares in Nov 2014, and $77M in an over-subscribed follow-on offering of 4,500,000 ordinary shares in July million shares outstanding as of March 31, 2016 NDRM Trades on the NASDAQ Global Market $95.7 million of cash and cash equivalents and short-term bank deposits as of March 31, 2016 Strong Balance Sheet; sufficient cash until mid

37 Experienced management team Oded S. Lieberman, PhD MBA Chief Executive Officer Sheila Oren, MD MBA Vice President Clinical and Regulatory Affairs Roy Golan, CPA LLM Chief Financial Officer Alon Yaar, DVM MBA Chief Operating Officer Oron Yacoby-Zeevi, PhD DVM Vice President R&D Sharon Cohen Vered, PhD Head of CMC Robert Taub, MBA Chairman Revlon Health Care Group (RHCG) 21 37

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