Cantor Global Healthcare Conference

Transcription

1 Nasdaq: CORI Cantor Global Healthcare Conference September 2017

2 Forward-looking Statements This presentation and the accompanying oral presentation contain forward-looking statements that are based on our management s beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, future financial and operating performance, business plans and objectives, potential growth and market opportunities, financing plans, competitive position, industry environment, product pipeline, clinical trial timing and plans, cash and resource requirements, clinical and regulatory pathways for our development programs, the achievement of clinical and commercial milestones, the advancement of our technologies and our proprietary, co-developed and partnered products and product candidates. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors including, but not limited to, those related to our future financial performance, market acceptance of our proprietary technology platforms for transdermal drug delivery, our ability to develop and maintain partnerships, our ability to identify, develop and market new products in a timely manner, our ability to maintain, protect and enhance our brand and intellectual property, and our ability to continue to stay in compliance with applicable laws and regulations. Moreover, we operate in a very competitive and rapidly changing environment, and new risks may emerge from time to time. It is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements we may make. These factors, together with those that may be described in greater detail in our filings with the Securities and Exchange Commission ( SEC ), may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although our management believes that the expectations reflected in our forward-looking statements are reasonable, we cannot guarantee that the future results, levels of activity, performance or events and circumstances described in the forward-looking statements will be achieved or occur. Moreover, neither we, nor any other person, assume responsibility for the accuracy and completeness of the forward-looking statements. These forwardlooking statements speak only as of the date of this presentation, and we undertake no obligation to publicly update 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, except as required by law. 2

3 Leading Transdermal Product Innovation Vision Transdermal solutions that transform care Approach Building a robust pipeline of novel CNS products Core Strengths 15+ years of development & manufacturing expertise Lead Product Streamlined path for donepezil Alzheimer s program 3

4 Proven Leadership Team Peter Staple Chief Executive Officer Robert Breuil Chief Financial Officer Joseph Sarret, MD Chief Business Officer Bobby Singh, PhD Chief Technology Officer Tim Sweemer, CPA Chief Accounting Officer Dan Arsulowicz VP Operations Grand Rapids Christina Dickerson VP Corporate Development Great Pacific Capital 4

5 Strong Track Record in Transdermal Products Commercial and late-stage partnered programs Partner Product Application Status Clonidine TDS Hypertension Marketed Fentanyl TDS Pain Marketed Crest Whitestrips (5 Products) Teeth Whitening Marketed Twirla Contraception NDA filed Undisclosed Motion Sickness ANDA filed 5

6 Our Proprietary Product Focus: CNS Diseases >5 million people in US have Alzheimer s* >1 million people in US have Parkinson s Rationale for transdermal CNS Sustained and consistent drug effect Improve compliance Reduce side effects, especially GI-related Reversibility of patch vs. injectables Alzheimer s Parkinson s Schizophrenia Depression Anxiety Multiple Sclerosis Epilepsy * Alzheimer s Association, 2016 Report American Parkinson s Disease Association 6

7 Enabling Sustained Delivery: Corplex Technology Novel combinations of pressure-sensitive and bioadhesive polymers Can adhere to both dry and wet surfaces Sustained drug delivery up to 7 days Load large amounts of drug/liquid Uniquely well-suited for poorly soluble drugs Enables first-in-patch products Overcomes limitations of other technologies 7

8 Blending Hydrophilic and Hydrophobic Polymers Over 150 Corplex patents issued and pending worldwide New IP created with each product Hydrophobic domains provide: Robust adhesive capability Hydrophilic domains provide: Capability to hold liquids with solubilized drug Excellent moisture handling capability Separation of liquid from adhesive Exceptional flexibility and conformability Hydrophilic Channels Hydrophobic Domains Hydrophobic Polymers Skin Enhanced stability, loading, and delivery Drug Molecule Hydrophilic Domains 8

9 Alzheimer s: Transformational Market Opportunity Alzheimer s Treatments 2015 U.S. TRx Share Peak WW Oral Sales (Billions) Peak WW Patch Sales (Billions) Rivastigmine (Exelon ) 9% $0.5 $1.1 Exelon Daily Patch Donepezil (Aricept ) 50% $4.2 Memantine (Namenda ) 39% $2.3 CNS Specialty Pharma Opportunity Sources: QuintilesIMS NPA Extended Insights data extracted in December 2016 and Novartis SEC filings 9

10 Major Market* Patient Share Donepezil and Memantine are Dominant Therapies Donepezil is dominant agent throughout disease progression Memantine is typically added later in disease * Major Markets include US, Japan, Germany, UK, France, Spain and Italy **Pre-disease indicates 1-2 years prior Source: DR/Decision Resources, LLC, 2014 market data for all formulations 10

11 Physician Intent to Prescribe is High Q: How likely are you to prescribe Corplex Donepezil to your Alzheimer s patients? Likely Definitely (29%) Probably (42%) Undecided Probably not (5%) Definitely not (2%) Not likely U.S. survey of 310 physicians; 235 primary care physicians and 75 neurologists Assumes $40/month copay and no benefit in GI adverse events Interest level increases with severity of disease Copay costs are an important attribute in treatment choice Source: Primary research conducted by AplusA Bell Falla LLC on behalf of Corium 11

12 Caregiver Interest and Intent to Fill is High I N TENT TO F ILL P R ESCRIPTION Survey of 300 U.S. caregivers: numbers in parentheses indicate number of patients in survey being treated with that agent (some patients were on more than one agent). Percentages <1% are not shown in chart. % in top 2 boxes: 87% 87% 87% 94% (n=281) (n=158) (n=175) (n=50) Q: Assume the doctor for the person you care for wrote that person a prescription for a one-month supply of PRODUCT D*. How likely is it that the PRODUCT D prescription would be filled for the person you care for? *Respondents were told to assume a $40/month copay and that PRODUCT D would have no improvement in GI adverse events vs. oral Aricept Source: Primary research conducted by an independent third party on behalf of Corium 12

13 Payer Approach: Consistent with Exelon Patch Interviews with formulary decision makers at 12 U.S. payers* Indicated Coverage Level** Plan Type National Plan National PBM*** Regional Plan/PBM Medicaid-focused Level of coverage similar to branded Exelon patch and Namenda XR > 50% of plans would require minimal utilization management At least one generic prescription in past 180 days 3 of 8 Medicaid plans would cover all with utilization management * Representing coverage of >100 million lives ** Assumes priced below Medicare Part D Specialty Tier ($670/Rx in 2017) *** PBM = Pharmacy Benefit Manager Source: Primary research conducted by Mary Bordeaux Consulting on behalf of Corium 13

14 Validated Pricing for Branded Transdermal Products 2015 TRx* in U.S. for Acetylcholinesterase Inhibitor (AChEI) Class = 13.3M Each 1% in U.S. market share = 133K TRx Branded Exelon patch monthly WAC = $594 Each 1% of branded U.S. AChEI market = $103M * Source: QuintilesIMS NPA Extended Insights data extracted in December TRx = Total Prescriptions (new plus refills) On average, each TRx represents 1.3 months of drug Source: Redbook 14

15 Positive FDA Guidance on Bioequivalence Pathway FDA agreed with proposed BE approach for both donepezil and memantine CY 2016 written responses confirmed clinical study designs and regulatory pathway A Pilot BE to study two patch sizes, followed by a Pivotal BE study of the final patch size Key factor is long half life: donepezil = 70 hours; memantine = hours Both reach steady state in 3-4 weeks Studies target bioequivalence (BE) to oral Aricept and oral Namenda XR Requires statistically similar patterns of blood concentrations over time Evaluated at steady state (during Week 5); healthy subjects Substantially reduces time, cost and complexity of clinical program Recent meeting with FDA reconfirmed Corium s BE pathway 15

16 Donepezil Pilot Study Design Three period, three-treatment, randomized crossover study Week Period 1 Period 2 Period Screening PK PK PK Cohort 1 5mg 10mg Aricept Oral Washout 5mg 10mg Patch B Washout 5mg 10mg Patch A Follow-Up (20 subjects start) Screening PK PK PK Cohort 2 5mg 10mg Patch A Washout 5mg 10mg Aricept Oral Washout 5mg 10mg Patch B Follow-Up (20 subjects start) Screening PK PK PK Cohort 3 5mg 10mg Patch B Washout 5mg 10mg Patch A Washout 5mg 10mg Aricept Oral Follow-Up (20 subjects start) Actual sequence of final two treatments was randomized 16

17 Pilot Study Objectives Accomplished Determine optimal patch size for Pivotal BE Trial Establish intra-subject variability of AUC and C max Determine optimal number of subjects required in Pivotal BE Trial Demonstrate acceptable skin and gastrointestinal tolerability Demonstrate acceptable adhesion over one week Determine required frequency of blood sampling for Pivotal BE Trial Measure elimination half-life for both oral and patch (washout) 17

18 Pilot Study Results: Unexpected De-risking Although not an objective of this study, statistical bioequivalence was achieved 18

19 Mean Plasma Donepezil Concentration (ng/ml) Pilot Study: Observed PK Profiles at Steady State Transdermal Delivery Exhibits Similar PK Profile to Oral Aricept Corplex Donepezil (n=52) 10 mg Oral Aricept (n=54) Oral Aricept Projected Daily PK Oral PK measured only on Day Days at Steady State (Week 5) Plasma sampling for patch treatments taken throughout Week 5 Plasma sampling for Aricept taken only on last day (day 7) of Week 5 19

20 Bioequivalence Assessment Corplex Donepezil is Bioequivalent to Aricept BE requires the 90% confidence intervals for the geometric mean ratios of AUC ss * and Cmax ss ** for Patch A vs. Aricept to each be within % Primary PK Parameters Geometric Mean Ratio (%) (Patch A vs. Oral Aricept) BE Limits (Target %) AUC ss (ng-hr/ml) Cmax ss (ng/ml) *AUC ss - area under the curve at steady state (for patch, observed area under the curve throughout week 5; for Aricept, observed AUC day 7 of week 5 multiplied by 7) **Cmax ss - maximum concentration at steady state (for patch, maximum observed conc. during week 5; for Aricept, maximum observed conc. during day 7 of week 5) 20

21 Cmax AUC (week) AUC (Day 1) AUC (Day 2) AUC (Day 3) AUC (Day 4) AUC (Day 5) AUC (Day 6) AUC (Day 7) Cmin BE Limits* Bioequivalence: Primary and Supportive PK Statistics C max AUC week C min Primary PK Supportive PK** * At 90% confidence intervals The % criteria do not apply to supportive PK parameters; FDA has indicated that these will be tested for no significant difference compared to oral Aricept ** % Fluctuation data not shown; as expected, the patch achieved less fluctuation between C max and C min due to controlled delivery 21

22 Most Common GI * Side Effects Corplex incidence of nausea was nearly 6x less than Aricept Drug-related GI Adverse Events, By Subject Count 19 29% 33% Corplex Donepezil (n=54) Oral Aricept (n=58) * GI = Gastrointestinal 3 7% 6% 1 4% 2% 6 10% 9% 3 7% % 9 21% 19% 17% Nausea Vomiting Diarrhea Constipation 6% 19% 22

23 Percentage of Patches Favorable Skin Tolerability 80% of patches had an irritation score of 0 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% 0% 18% Observed Skin Irritation (based on 340 patch applications) Score of 1* Score of 2* 15% 12% 5% 2% 1% 1% 3% 0% 0% Day 1 Day 2 Day 3 Day 5 Day 7 Days Post Patch Removal * Score on a rating scale of 0 (none) to 7 (strong reaction beyond patch application site) per FDA draft guidance on dermal response Percentage of patches exhibiting no irritation (score of 0) are not displayed in chart above 23

24 Corplex Donepezil Wearability: Thin and Flexible ~150cm 2 Delivers 7 Days of Drug Thinner Than a Dime 24

25 FDA End-of-Phase 2 (EOP2) Meeting Summary FDA provided positive feedback on remaining clinical and CMC plans Confirmed BE path, PK evaluation parameters, and study designs Reduced number and type of ancillary studies required FDA also reviewed whether pilot BE could serve as pivotal study To stay on track, pivotal study will proceed (dosing by early November) NDA filing expected in 4Q CY2018, following 12-month stability data 25

26 FDA Meeting Additional Highlights Reconfirmed pivotal BE study design and PK statistical testing plan: Streamlined version of pilot study (50 completers), but targeting 66 completers Confirmed dose proportionality sufficiently demonstrated between 5 and 10 mg: No separate dose proportionality study required Confirmed study design for two ancillary studies: Skin irritation and patch application site PK study Clarified that patch-to-pill comparative data (including systemic Adverse Events) would be considered for inclusion in the label during NDA review 26

27 Pivotal Study Design More Streamlined Pilot 3-way crossover, randomized Pivotal 2-way crossover, randomized Healthy Volunteers Enroll 60 to complete at least 30 (50 actually completed) 3 treatments (2 patch sizes and oral Aricept) Each Period = 5 weeks of treatment (5mg Week 1 followed by 10 mg Weeks 2-5) 3-week washout Healthy Volunteers Enroll ~86 to complete ~66 2 treatments (Final patch size and oral Aricept) Each Period = 5 weeks of treatment (5mg Week 1 followed by 10mg Weeks 2-5) 5-week washout Primary Objective: Assess C max and AUC Primary Objective: BE for C max and AUC Study Duration: 6 months 5 months 27

28 Donepezil Pivotal Study Design Each healthy subject* will be randomized into one of two treatment sequences Week Period 1 Period PK PK Cohort 1 Screening 5mg Oral 10mg Aricept Oral Extended Washout 5mg Patch 10mg Final Patch Follow-Up PK PK Cohort 2 Screening 5mg Patch 10mg Final Patch Extended Washout 5mg Oral 10mg Aricept Oral Follow-Up * Based on experience of Pilot Study, will enroll ~86 subjects with expectation of completing ~66 Screening ongoing; dosing expected to commence by early November

29 Donepezil: Clear Pathway to Commercialization 2017* Ancillary Studies CMC, Stability, NDA Preparation File NDA PDUFA 2H 2019 Commercialize Studies Pivotal Bioequivalence Irritation & Sensitization Heat Effect Regional Absorption Dermal Toxicity (Animal) * All years are calendar years 29

30 Financial Highlights at June 30, 2017 FY2016 total revenues of $33M ($22.4M first 9 months of FY2017) Sole supplier of Crest Whitestrips for P&G; new 5-year extension $66.6M in unrestricted cash at June 30, 2017 $52M in long-term debt (primarily with CRG, due June 2019) 35.7M shares outstanding as of August 8, 2017 >10% shareholders: Essex Woodlands, Perceptive, Broadfin 30

31 Corium: Growth and Momentum in 2017 An integrated transdermal products engine Building on 15+ years of development and commercial manufacturing Twirla to drive growth in partner business: potential approval 4Q CY2017 Our Corplex Approach: Donepezil Commercialize lead product Transformational in Alzheimer s CNS disease Products Lower-risk bioequivalence path tracking toward launch by late 2019 Major clinical de-risking achieved in Pilot study; Pivotal data in 2Q CY2018 High-value CNS pipeline opportunity and strong IP Corplex Memantine complements Alzheimer s portfolio New Phase 1 programs in CY 2018 to expand CNS specialty franchise 31

32 Thank You Nasdaq: CORI