Continuous therapy. Because life goes ON. Jefferies 2015 Global Healthcare Conference June, 2015

Transcription

1 Continuous therapy. Because life goes ON Jefferies 2015 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. These 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. 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. 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. For a description of the primary risks to which we are subject, please see the Risk Factors section of the final prospectus for our initial public offering, which was filed with the U.S. Securities and Exchange Commission on November 17, 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 Innovative formulations that significantly increase clinical efficacy in CNS diseases New formulations of existing drugs allow new administration routes that overcome current limitations High Ethical > Oral drugs with short half life; low bioavailability and/or solubility Generics New drug has significant advantages: > Higher efficacy Low Low Development Cost / Timeline / Risk High > Fewer side effects > Improved quality of life and ease of use High NCE-like barriers to entry grant de facto exclusivity Strong patents Innovative Formulations High price point Accelerated lower risk regulatory pathway (505b2), short time to market Few/ limited alternatives Unique indication 21 3

4 Pipeline of products expected to launch by Product Candidate Indication Preclinical Phase I Phase II Phase III TTM ND0612H Severe Parkinson s Disease LD/CD, Subcutaneous H1-2/2018 ND0612L Moderate Parkinson s Disease LD/CD, Subcutaneous H2/2018 ND0680 Severe Parkinson s Disease LD/CD, Intra-Duodenal BE study, 2015 H2/2017 EU ND0701 Severe Parkinson s Disease Apomorphine, Subcutaneous BE study, 2015 H2/2017 EU ND0801 CNS disease Cognition disorders Nicotine and Opipramol, Transdermal Ongoing Proof-ofconcept study 21 4

5 ON/OFF time 21 5

6 Blood Levels of Levodopa Levodopa, the Gold Standard, has a major drawback: short half life The brain needs steady levels of dopamine Brain dopamine replaced by oral LD Oral LD has a short half life Narrowing therapeutic window with disease progression Mild Moderate Severe Dyskinesia Instead of supplying steady LD levels oral LD creates sharp fluctuations, a saw-tooth graph LD fluctuations lead to motor complications OFF time (avg. of 6 hours) and dyskinesia 20% of patients develop after 6 months; 50% of patients develop after 18 months; almost all patients in 2-5 years Constant LD levels may slow disease progression Early wearing off Delayed on Levodopa Administration On Time Off Time ND0612 Fluctuations Oral route may not be the optimal method of delivering levodopa to patients who have a fluctuating response (Bredberg et al. 1994) Continuous delivery has been the greatest challenge of levodopa therapy 21 6

7 To date, efforts to improve continuous LD therapy have had limited success NO GAME CHANGERS Attempts to achieve continuity Controlled release tablets Slow release tablets Gastric retention devices Dispensing devices Why a limited benefit? Because LD has only existed to date in solid form that must be administered through the GI tract, leading to erratic uptake and low bioavailability Why haven t other drug forms been developed to enable alternative routes of administration? 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 Treatment alternatives for advanced patients Deep brain stimulation Intra-duodenal LD/CD pump (DuoDopa) Removal post infection 15% Seizures 2.4% Cognition impairments 41% Peritonitis 3% Complication of insertion 40% Wound infection 21% Device sales: ~$500M (excluding surgery) Cost per patient: up to 100,000 1 st yr, ~$25,000 yr/5yr Europe sales: ~$200M Cost per patient: $70,000/yr 21 8

9 Lead candidates introduce a new paradigm in PD treatment ND0612H belt pump ND0612L belt pump ND0612L patch pump Severe Parkinson s disease Moderate Parkinson s disease Moderate Parkinson s disease The first liquid LD/CD drug, replacing surgical treatments Device CE approved in EU >350,000 severe patients US and EU Current segment treatment costs: ~$70,000 /yr/pt The first liquid LD/CD drug, a new SOC in PD, significantly reducing OFF-time Device CE approved (EU) >900,000 patients US and EU Current segment treatment costs: ~ $6,000/yr/pt 2 nd generation ND0612L, delivered through a patch pump. One and done operation Device in development by a third party H1-2/2018 H2/

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 80 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 ND0612L+oral SOC Period 1: Randomized double blind 2 weeks Placebo + oral SOC Pre-Randomized 16 patients 1:1 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 14

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 Early onset of treatment effect observed 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) -10 Placebo (n=11) ND0612 (n=19) 30% improvement in quality of sleep 30% vs. 0.9% compared to placebo 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 No systemic or local irritation, all patients elected to continue to the open label extension phase In volunteers and PD patients so far: Draize score slightly elevated, slight pruritus, similar in both groups, normalized at week 3 SC nodules (0.5-1cm) resolving spontaneously No particular systemic AE No patient discontinued early Local safety similar to Phase I in healthy subjects Skin local safety in healthy volunteers - no local irritation Transient and reversible local minor reaction 21 19

20 ND0612H Severe patients 21 20

21 Plasma LD (ng/ml) Summary of preliminary results to date of phase IIa of ND0612H Steady ND0612H levels alone and with oral entacapone (1x/5h) ND0612L ND0612H , Alone With entacapone ND0612H should be suitable for the majority of severe (incl. Duodopa) patients 21 21

22 Commercial development 21 22

23 Advanced PD patients a hidden segment with the largest unmet need Parkinson s disease is the second largest neurodegenerative disease Affects one in one hundred people over age 60 (> 6 million WW) Breakdown by severity and cost (2011) Moderate to severe patients - ~60 % of all PD PD treatment market $3.8B Drugs: ~$3.3B; many are generic Devices: <1% of PD patients are treated by DBS but consume approx. $500M excluding surgery costs Duodopa sales exclude US sales and cost of surgery Severe ~$70,000 16% Mild Most moderate and severe patients are inadequately controlled Historical peak sales of leading treatments Mirapex DBS Duodopa Target population Moderate Severe Severe Annual peak sales $0.7B WW $0.5B WW* ~ $0.2B EU Moderate ~$6,000 42% 42% *Excluding surgical costs ($30K-$50K/pt) 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 Global Total PD patients 1,000,000 1,200,000 6,300,000 Moderate PD patients 420, ,000 2,646,000 Severe PD patients 160, ,000 1,008,000 Severe PD patients treated with advanced treatments (2014) DBS new patients/yr ~13,000** ~9,000** ~22,000** Duodopa patient base 0 ~3,500 ~4,000 Duodopa new patients/yr 0 ~700 ~800 Apomorphine patient base*** 0 ~4,000 ~6,000 Severe patients not treated with advanced treatments ~>100,000 ~>120,000 ~>700,000 *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 Future development plan 21 25

26 Clinical development plan 505(2)(b) pathway for both products ND0612L development plan agreed with the FDA End of phase II meeting expected in 2015 Hybrid pathway is anticipated for both products A PK similarity program for ND0612H (vs. Duodopa) was agreed with the EMA EMA agreed to only 1 pivotal trial with no active comparator for ND0612L ND0612H EU Hybrid Phase IIa - 004, PK (16 patients) Phase II BE, 005 (12 patients) Phase II BE, 009 (40 patients) Long term safety ( patients) MAA US 505(b)(2) / EU Hybrid Phase IIb - 006, PK (30 patients) Phase III (120 patients) Long term safety ( patients) NDA ND0612L Phase II 003 (30 patients) End of phase II meeting Phase III pivotal (240 patients) Phase III pivotal (360 patients) Long term safety ( patients) NDA, MAA H2/ EU approval US approval US /EU approval 21 26

27 Expected milestones ND0612H ND0612L 2014 Completion of a PK study (004) 2015 FDA clinical hold lift Completion of a PK study vs. Duodopa (005) Initiation of a BE study vs. Duodopa, EU (009) Initiation of a phase II study, US (006) Initiation of a long term safety study (011/12) FDA clinical hold lift End of phase II meeting Initiation of 2 pivotal studies (007,008) 2016 Completion of BE study, Europe (009) Completion of phase II study,us (006) End of phase II meeting Initiation of phase III, US (010) 2017 Completion of long term safety study (011/12) Completion of phase III, US (010) Completion of pivotal studies (007,008) Completion of long term safety studies (011/012) 2018 EU and US approval EU and US approval 2019/20 Bridging study and approval of a patch pump version 21 27

28 Experienced and cohesive management team Oded S. Lieberman, PhD MBA Chief Executive Officer Sheila Oren, MD MBA Vice President Clinical and Regulatory Affairs Roy Golan, CPA LLM Vice President Finance 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 28

29 NeuroDerm (NASDAQ: NDRM ) Cash: $38.9 million March 31, 2015 Lock-up: 180 days (Expired in Mid May 2015) Analyst Coverage & Price Target: Jefferies ($21.00), Cowen and Company ($30.00), Oppenheimer & Co. ($19.00) & Roth Capital Partners ($17.50) Shares Outstanding: 17 million (18.7 million on a fully diluted basis) 21 29

30 Investment highlights Enabling a paradigm shift in the treatment of PD First ever liquid formulation of LD/CD enables continuous administration, transforms PD therapy, replaces surgery $2M granted from the Michael J. Fox Foundation for Parkinson s Research Low risks, short timelines high value premium products Formulations with low risks, cost and short time to market (2017/18) Innovative technology - high barriers to entry, effective exclusivity NCE-like value proposition and pricing Positive clinical results Significant reduction in LD plasma fluctuations: >800 ng/ml Reduction in OFF-time compared to SOC: >2 hours No increase in dyskinesia Increase in quality of life and sleep quality Safe and well tolerated Numerous near term value driving milestones 2014: 2 phase II study results 2015: 1 phase II study results; 3 pivotal studies initiation 21 30

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