Forging the Future of Dermatology. Jefferies 2015 Global Healthcare Conference June 2015

Transcription

1 Forging the Future of Dermatology Jefferies 2015 Global Healthcare Conference June 2015

2 Forward-looking statements 2 This presentation contains 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 business strategy, market sizes, potential growth opportunities, product attributes and performance, the timing and results of clinical trials and the timing and outcome of meetings with regulatory agencies. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors including, but not limited to, those related to our ability to obtain regulatory approval for our product candidates, the costs of our development programs, our ability to obtain necessary additional capital, the outcomes of our clinical trials, including related to further analysis of the results of our studies, the outcomes of meetings with regulatory agencies, our dependence on third party clinical research organizations, manufacturers and suppliers, market acceptance of our potential products, our ability to develop and maintain collaborations and license products and intellectual property, the impact of competitive products and therapies including generics and biosimilars, our ability to manage the growth and complexity of our organization, our ability to maintain, protect and enhance our intellectual property, and our ability to continue to stay in compliance with applicable laws and regulations. These factors, and other factors that we cannot predict or assess, 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. Neither we, nor any other person, assumes responsibility for the accuracy and completeness of the forward-looking statements. We undertake no obligation to 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. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our industry. These data involve a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk.

3 Unique opportunity Build leading innovator in evolving dermatology landscape 3 Large, growing, underserved specialty market with significant unmet needs Consolidating segment with few companies focused on true innovation Bringing biopharma innovation to skin disease Value creation via efficient development and commercialization Scientific advances creating opportunity for innovative, new treatment approaches

4 Dermira highlights Uniquely positioned to build leading innovator in dermatology 4 Mission Strategy Strengths 3 late-stage programs with positive P2 data Improve patients lives by bringing innovation to dermatology and important and differentiated new products to dermatologists Identify, develop and commercialize innovative, differentiated dermatology products Key management behind past dermatology successes Innovative, late-stage portfolio Strong balance sheet Cimzia for psoriasis: Differentiated TNF inhibitor for growing >$3B market 1 DRM04 for hyperhidrosis: Topical anticholinergic for large, underserved population DRM01 for acne: Topical sebum inhibitor, differentiated MOA for >$3B market 2 1. Decision Resources, Immune and Inflammatory Disorders Study, Psoriasis, October 2014, 2013 U.S. sales of biologics for psoriasis. 2. Symphony Health Solutions, Pharmaceutical Audit Suite, 2012 U.S. gross sales of prescription acne products.

5 5 Experienced leadership Outstanding track record in dermatology and biopharma Tom Wiggans Chairman & Chief Executive Officer Gene Bauer, MD Chief Medical Officer STANFORD School of Medicine Andrew Guggenhime Chief Operating Officer & Chief Financial Officer Luis Peña EVP, Product Development GENENTECH A Member of The Roche Group Chris Griffith VP, Corporate Development & Strategy

6 6 The next wave of innovation Three late-stage product candidates with positive P2 data Program Preclinical P1 P2a P2b P3 Cimzia Injectable TNF inhibitor (psoriasis) DRM04 Topical anticholinergic (hyperhidrosis) DRM01 Topical sebum inhibitor (acne) DRM02 Topical PDE4 inhibitor (inflammatory diseases) DRM05 Topical PDT (acne) Next anticipated milestone 1 Topline P3 data 2017 Initiate P3 program 2H15 Topline P2b data 1H16 Preclinical data 2015 Preclinical data 2015 Commercial rights Dermatology rights in U.S., Canada WW rights WW rights WW rights WW rights 1. Estimates provided as of May 12, 2015.

7 Attractive partnership with UCB Leveraging Dermira expertise to bring Cimzia to dermatology 7 Structure Profit share Development UCB contribution CoC provision Dermira promotes to dermatologists in U.S., Canada; UCB retains all other rights International co-development partnership Dermira receives share of gross margin 1 from Cimzia sales attributed to dermatologists for all indications in U.S., Canada Dermira share between 90% and, on sales >$150M in any one year, 50% Dermira funds: Development plan cost up to specified amount between $75-95M 50% of any additional development plan or pediatric study cost Dermira internal cost $109.5M in cash and equity investment Invested $20M in equity Up to $36M development milestone payments, of which $7.3M earned Up to $40M commercial + $13.5M EU approval milestone payments 2 UCB may terminate if Dermira is acquired by biologic TNF inhibitor company or other non-qualified company 3 1. Following approval in psoriasis, profit share is based on gross margin after subtracting cost of certain commercialization support services provided by UCB. 2. EU approval milestone payments are contingent on pricing and reimbursement approvals in certain EU countries. 3. Non-qualified companies include any company that (1) is not engaged in the development or commercialization of a pharmaceutical product or does not maintain Dermira as an operating entity with 50% of its executive management team intact for 1 y, (2) lacks capital to complete development obligations or does not have ability to raise capital to fulfill commercial activities and other obligations to UCB, or (3) does not agree to complete development obligations (if no regulatory approval for Cimzia for psoriasis in U.S., Canada or EU at time of acquisition).

8 8 Cimzia: Differentiated TNF inhibitor Attractive opportunity in large, growing psoriasis market ~$3.8B psoriasis sales 1, 20% CAGR in biologic sales to dermatologists 2 Opportunity ~50% of patients remain unsatisfied with available products 3 Relatively low biologic penetration, only 10.5% of treated moderate-tosevere patients 1 Differentiated TNF inhibitor with attractive profile for psoriasis Solution Opportunity for advantages over marketed and development-stage therapies Attractive UCB partnership to develop, commercialize for psoriasis P3 program initiation announced Jan 2015, topline data expected Status Attractive safety, tolerability, P2 efficacy profile Productive FDA and EMA meetings completed 1. Decision Resources, Immune and Inflammatory Disorders Study, Psoriasis, October 2014, 2013 U.S. sales of biologics for psoriasis. 2. IMS Health Solutions, National Prescription Audit, National Sales Perspectives, CAGR in U.S. sales attributed to dermatologists. 3. Armstrong et al., JAMA 2013, data from Estimate provided as of May 12, 2015.

9 9 Psoriasis: Significant U.S. market opportunity Large, unsatisfied, underpenetrated patient population Autoimmune skin disease affecting >9M people in the U.S. 1 Chronic, complex disease with associated morbidities requiring long-term treatment ~20% of patients have moderate-to-severe disease 2 Treatment transformed by TNF inhibitors Established safety record; >15 years of commercial use Attractive efficacy profile; treat skin symptoms and systemic manifestations of psoriasis New biologics (α-il-12/23 and α-il-17) expected to continue to expand market ~$3.8B U.S. sales of biologics in 2013 expected to reach $6.2B by Decision Resources, Immune and Inflammatory Disorders Study, Psoriasis, October U.S. sales of biologics for psoriasis. 2. National Psoriasis Foundation website Psoriasis Severity accessed January 2015.

10 Attractive competitive profile in psoriasis P2 data support opportunity for differentiated product profile 10 Objectives Launch differentiated TNF inhibitor to derms with leading product profile Efficacy comparable to Humira (mab) with potential safety advantages of Enbrel (non-mab) Response rate at week % 80% 60% 40% 20% 0% Cross-study comparison of Cimzia and market leaders 1,2 PASI mg q2w 400 mg q2w Placebo 50 mg biw Placebo 40 mg q2w Placebo Cimzia Phase 2 clinical trial Pegylated antibody fragment (n=176, p<0.001) PGA Enbrel Phase 3 clinical trial Fusion protein (n=407, p<0.0001) Humira Phase 3 clinical trial Complete antibody (n=1,212, p<0.001) 1. PASI 75 = proportion of treated patients who achieved a 75% improvement in the clinical grading scale called the Psoriasis Area and Severity Index. PGA (Physician s Global Assessment) = proportion of patients who achieved clearing or near clearing of psoriasis as rated by the investigator week efficacy data from Cimzia P2 study and largest pivotal Enbrel and Humira P3 studies. Cimzia patients received 400 mg at week 0 followed by 200 or 400 mg q2w (Reich et al., British Journal of Dermatology, July 2012). Enbrel patients received 50 mg biw (Amgen 2013 Enbrel prescribing information). Humira patients received 80 mg at week 0, followed by 40 mg q2w starting 1 week later (Menter et al., Journal of the American Academy of Dermatology, January 2008).

11 11 DRM04: Topical hyperhidrosis therapy New product opportunity targeting significant unmet need ~7.8M people suffer from excessive sweating in the U.S. 1 Opportunity Available therapies limited in efficacy, tolerability or administration profile Highly underpenetrated market (500k annual topical antiperspirant TRx) 2 Solution Reduce sweat production by blocking cholinergic sweat gland receptors A topical formulation of novel form of anticholinergic, the reference agent 3, approved for systemic administration in other indications Status Attractive efficacy, safety, tolerability profile observed in 3 P2 trials (n=341) of topical formulation of reference agent Positive results for proprietary DRM04 product in P2b study (n=105) Productive FDA EOP2 meeting conducted; expect P3 program to start 2H Strutton et al., Journal of the American Academy of Dermatology, August 2004, data as of Symphony Health Solutions, Pharmaceutical Audit Suite, 2013 data. 3. The reference agent is an anticholinergic agent that has been approved for systemic administration in other indications. 4. Estimate provided as of May 12, 2015.

12 Hyperhidrosis: Market development opportunity Large, underserved patient population 12 Hyperhidrosis is excessive sweating beyond what is physiologically required to maintain normal thermal regulation Impedes normal daily activities and can result in occupational, emotional, psychological, social, physical impairment 1 ~7.8M people in the U.S. have hyperhidrosis, ~50% of whom suffer from axillary disease 1 ~33% of axillary patients report sweating that is barely tolerable or intolerable 1 Highly underserved by current treatment options Topical antiperspirants that clog sweat ducts Off-label systemic therapies Injectable, surgical and other procedures Patient with axillary hyperhidrosis 1. Strutton et. al., Journal of the American Academy of Dermatology, August 2004, data as of 2003.

13 13 Strategic development program Regulatory path builds on approved systemic reference agent Study DRM04-HH01 1 st Phase 2b study Study DRM04-HH02 2 nd Phase 2b study Topical formulation for hyperhidrosis Characterize efficacy, safety and tolerability of new route of administration in new indication Completed P2b dose-ranging trial (n=198) Topical formulation for hyperhidrosis Confirm profile with new form, additional PRO 1 Completed P2b bridging trial (n=105) Supports advancement into P3 Builds on profile of reference agent for hyperhidrosis Expands IP opportunities 1. PRO = patient-reported outcome instrument.

14 14 Absolute Percent Positive HH01 results on key efficacy measures Dose-dependence, statistical significance with reference agent % -20% -40% -60% -80% -100% Change in Sweat Production at Week 4 (mg per 5 min.) 1,2 Baseline: % -54.4% % Vehicle p= % p= % p=0.006* 3% p=0.001* 4% -73.4% -71.7% Vehicle p= % p= % p=0.005* 3% p= % (n=40) (n=38) (n=40) (n=40) (n=40) Vehicle 1% 2% 3% 4% 100% 80% 60% 40% 20% 0% HDSS Response Rate at Week 4 (% of patients w/ 2-point improvement) 1,3 22.5% 36.8% p= % 52.5% 45.0% p=0.011* p=0.006* p=0.036* (9/40) (14/38) (20/40) (21/40) (18/40) Vehicle 1% 2% 3% 4% 1. ITT population shown = all randomized patients dispensed study product (n=198). P-values are an indication of statistical significance reflecting the probability of an observation occurring due to chance alone. P-values of 0.05 or less (denoted by *) typically represent statistically significant results. P-values shown above represent comparisons to cohort of patients who received vehicle only. 2. Changes in sweat production reflect average absolute and percent changes from baseline. Last available on-treatment response used to estimate missing data points. 3. HDSS response rate reflects % of patients achieving a 2-point improvement in HDSS score. Patients with missing data points considered nonresponders.

15 15 Absolute Percent HH02 results consistent with HH01 results Completion of P2 program enables FDA EOP2 meeting Baseline: % -20% -40% -60% -80% -100% Change in Sweat Production at Week 4 (mg per 5 min) 1,2, % p=0.023* -79.8% p= % p=0.006* p=0.069 (n=22) (n=22) (n=20) Vehicle DRM04 Dose 1 DRM04 Dose 2 100% 80% 60% 40% 20% 0% HDSS Response Rate at Week 4 (% of patients w/ 2-point improvement) 1,3,4 27.3% 40.9% 50.0% p=0.526 p=0.204 (6/22) (9/22) (10/20) Vehicle DRM04 Dose 1 DRM04 Dose 2 1. ITT population shown = all randomized patients dispensed study product (n=105). P-values are an indication of statistical significance reflecting the probability of an observation occurring due to chance alone. P-values of 0.05 or less (denoted by *) typically represent statistically significant results. P-values shown above represent comparisons to cohort of patients who received vehicle only. 2. Changes in sweat production reflect average absolute and percent changes from baseline. Last available on-treatment response used to estimate missing data points. 3. HDSS response rate reflects % of patients achieving a 2-point improvement in HDSS score. Patients with missing data points considered non-responders. 4. For patients in the two arms treated with the topical formulation of the reference agent, the results were consistent with those observed in Study DRM04-HH01.

16 16 Topical treatment well tolerated Low incidence of manageable side effects Most common AEs: Study DRM04-HH01: Dry mouth, upper respiratory tract infection, dry skin and blurred vision Study DRM04-HH02: Dry mouth, application site pain and headache Dry mouth, blurred vision and dry skin are well-known, reversible anticholinergic effects

17 Clinical plan leverages positive P2 data Phase 2 program complete, expect P3 initiation 2H P2a clinical trial P2b clinical trials P3 clinical program (planned) POC HH01 HH02 Objective(s) Clinical POC Dose-finding Bridge from reference agent to DRM04 API Evaluate new PRO assessment (ASDD) Confirm safety and efficacy Study product Topical formulation of reference agent Topical formulation of reference agent Topical formulation of reference agent DRM04 DRM04 Population Severe, primary axillary hyperhidrosis patients (n=38) Severe, primary axillary hyperhidrosis patients (n=198) Severe, primary axillary hyperhidrosis patients (n=105) Severe, primary axillary hyperhidrosis patients (n=tbd) Administration QD x 4 weeks QD x 4 weeks QD x 4 weeks QD x 4 weeks Key efficacy measures Sweat production (gravimetry) PRO (HDSS) Sweat production (gravimetry) PRO (HDSS) Sweat production (gravimetry) PRO (HDSS, ASDD) Sweat production (gravimetry) PRO (HDSS, ASDD) Status Complete Complete Complete Initiation expected 2H Estimate provided as of May 12, 2015.

18 18 DRM01: Topical sebum inhibitor for acne New MOA for one of the most common skin diseases $3.7B global pharmaceutical acne market 1, 40-50M U.S. prevalence 2 Opportunity Only 3 major product classes available for >30 years, need for new MOA 3 No available topical therapy targets sebum, a key pathogenic factor Solution Novel prodrug designed to deliver well-characterized lipid synthesis inhibitor Targets sebum production topically New MOA to be developed via well-established regulatory pathway Attractive P2a efficacy, safety, tolerability profile (n=108) Status Statistically significant efficacy in FDA-recommended endpoints P2b dose-finding program initiated April 2015, topline data expected 1H VisionGain, Dermatological Drugs Market Forecast , April 2014, 2012 sales. 2. American Academy of Dermatology, Acne, Accessed June IMS Health, VisionGain, Dermatological Drugs Market Forecast , April 2014, acne product package inserts. 4. Estimate provided as of May 12, 2015.

19 19 Acne: Large market, significant unmet need $3.7B global market with limited therapeutic options 1 One of the most common skin diseases (40-50M U.S. prevalence) 2 QOL impact estimated to be comparable to that associated with epilepsy, asthma, diabetes or arthritis 3 Acne treatment guidelines recommend targeting multiple pathogenic factors 4 Product class Sales 5 Target Limitations Topical retinoids $0.9B Follicular hyperkeratinization Skin irritation, moderate efficacy Topical, oral antimicrobials $1.9B P. acnes, inflammation Bacterial resistance, waning efficacy Oral isotretinoin $0.7B Excess sebum production Significant systemic toxicity 1. VisionGain, Dermatological Drugs Market Forecast , April American Academy of Dermatology, Acne, Accessed June Mallon et al., British Journal of Dermatology, April Gollnick et al., Journal of the American Academy of Dermatology, July Symphony Health Solutions, Pharmaceutical Audit Suite Data Calendar Year , Gross sales, accessed July 2014.

20 20 Novel molecule with differentiated MOA Targeting ACC, key regulator of sebum production DRM01 targets sebum production Prodrug specifically targets acetyl coenzyme-a carboxylase (ACC), key regulator of sebum production Sebum production is key aspect of acne pathophysiology not addressed by available topical therapies Opportunity for isotretinoin-like effects without systemic toxicity

21 21 DRM01 improves lesion counts Significant impact on 2 FDA-recommended primary endpoints Avg. baseline lesion count: Avg. absolute change in lesion count from baseline to week Primary Endpoints: Absolute Changes in Lesion Counts at Week 12 1 Inflammatory lesion count Non-inflammatory lesion count Vehicle DRM01 Vehicle DRM01 N: p=0.0003* p=0.0032* -35 Inflammatory lesion count Non-inflammatory lesion count Avg. % reduction Vehicle DRM01 Vehicle DRM01 in lesion counts 1 : 45.9% 63.9% 28.8% 48.1% p-value * * 1. As recommended in published FDA guidance, data are presented from ITT population, defined as all randomized patients dispensed study product, and the last available on-treatment observation is used to estimate missing data points. Average baseline lesion count includes all 108 patients in the ITT population. Missing data for one patient in the vehicle cohort for whom no on-treatment efficacy assessment was available are excluded from the patient population observed at week 12. P-values are an indication of statistical significance reflecting the probability of an observation occurring due to chance alone. P-values of 0.05 or less (denoted by *) typically represent statistically significant results. P-values shown above represent comparisons to corresponding lesion count reductions observed in patients who received vehicle only.

22 DRM01 improves IGA response Significant impact on 3 rd FDA-recommended primary endpoint 22 Primary Endpoint: IGA Response Rate at Week % 80% >3-fold improvement 60% 40% p=0.0070* 24.5% 20% 0% 7.3% Vehicle (n=4/55) DRM01 (n=13/53) 1. IGA (Investigator s Global Assessment) = Investigator s assessment of disease severity based on FDA-recommended 5-point scale ranging from score of 0, representing clear skin, to 4, representing severe disease. IGA response rate reflects % of patients achieving 2-point improvement in IGA score from baseline. As recommended in published FDA guidance, data are presented from ITT population, defined as all randomized patients dispensed study product. Patients with missing data points were considered non-responders. P-values are an indication of statistical significance reflecting the probability of an observation occurring due to chance alone. P-values of 0.05 or less (denoted by *) typically represent statistically significant results. P-value shown above represents comparisons to response rate observed in patients who received vehicle only.

23 23 DRM01 well tolerated Expected, topical side-effect profile Most common AEs Application-site conditions, frequently observed in clinical trials of topical products Upper respiratory tract infections, considered unrelated to treatment

24 patient P2b study ongoing Standard design based on published FDA draft guidance Randomized, double-blind, vehicle-controlled, dose-ranging trial ~400 moderate-to-severe adult acne patients 1 FDA-recommended primary efficacy endpoints (week 12) Inflammatory lesion count: Absolute change from baseline Non-inflammatory lesion count: Absolute change from baseline IGA: Proportion of patients achieving 2-point reduction in IGA score DRM01 (7.5% BID) Screening Randomization DRM01 (7.5% QD) DRM01 (4% QD) Vehicle (0% BID) Vehicle (0% QD) End of follow-up 0 Treatment period Week Principal inclusion criteria: Adults with 20 inflammatory lesions, 20 non-inflammatory lesions and Investigator s Global Assessment (IGA) score of 3-4.

25 Topline P2b data expected 1H16 1 Establishing dose(s) for P3 using FDA-recommended endpoints 25 P2a clinical trial P2b clinical trial (ongoing) Objective(s) Clinical POC Dose-finding Population Administration Adult acne patients (n=108) 7.5% gel BID Adult acne patients (n 400) 7.5% gel BID 7.5% gel QD 4% gel QD Vehicle gel BID Vehicle gel QD P3 clinical trials (planned) Confirm safety and efficacy Adult and adolescent acne patients (n=tbd) Regimen(s) selected based on P2b data Duration 12 weeks 12 weeks 12 weeks Primary efficacy measures Lesion count IGA Lesion count IGA Lesion count IGA Status Complete Topline data expected 1H16 1 TBD 1. Estimate provided as of May 12, 2015.

26 Strong financial position $158.1M in cash at Mar. 31, $ in millions SELECT BALANCE SHEET DATA March 31, 2015 December 31, 2014 Cash and cash equivalents and investments $158.1 $163.6 Total debt: Bank term loan, current and non-current Total stockholders equity (deficit) SELECT STATEMENTS OF OPERATIONS DATA Operating expenses: Three Months Ended March 31, 2015 March 31, 2014 Research and development General and administrative Total operating expenses Net loss (14.0) (8.5) 1. Includes cash and cash equivalents and investments.

27 Significant business momentum Key 2015 milestones and expectations 27 Operating Initiate Cimzia P3 trials DRM04 HH02 Pb2 data Initiate DRM01 P2b trial Initiate DRM04 P3 program Execute on trial enrollment objectives Continue to evaluate portfolio expansion opportunities 2015 guidance Financial $80-85M in non-gaap operating expenses 1 ~$90M in cash and cash equivalents and investments as of year-end M shares outstanding at year-end Excludes impact of stock-based compensation expenses.

28 28 Dermira highlights Bringing biopharma innovation to skin disease Unique opportunity in dermatology Large, growing, underserved specialty market with significant unmet needs Value creation via efficient development and commercialization Scientific advances creating opportunity for innovative, new treatment approaches Consolidating segment with few companies focused on true innovation Uniquely positioned to succeed Singular focus on dermatology Experienced management team Focused strategy to identify, develop and commercialize innovative, differentiated products Innovative, late-stage portfolio of 3 programs with positive P2 data Strong balance sheet

29 Forging the Future of Dermatology June 2015