jefferies 2015 global healthcare conference june … 2015 global healthcare conference june 2015...
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Forging the Future of Dermatology
Jefferies 2015 Global Healthcare Conference June 2015
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.
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Unique opportunity Build leading innovator in evolving dermatology landscape
Large, growing, underserved specialty market with
significant unmet needs
Scientific advances creating opportunity for innovative, new
treatment approaches
Consolidating segment with
few companies focused on true
innovation
Value creation via efficient
development and commercialization Bringing biopharma innovation
to skin disease
4
Dermira highlights Uniquely positioned to build leading innovator in dermatology
Mission Improve patients’ lives by bringing innovation to dermatology and important and differentiated new products to dermatologists
Strategy Identify, develop and commercialize innovative, differentiated dermatology products
3 late-stage programs
with positive P2 data
Cimzia for psoriasis: Differentiated TNF inhibitor for growing >$3B market1
DRM04 for hyperhidrosis: Topical anticholinergic for large, underserved population
DRM01 for acne: Topical sebum inhibitor, differentiated MOA for >$3B market2
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.
Strengths Key management behind past dermatology successes Innovative, late-stage portfolio Strong balance sheet
Tom Wiggans Chairman & Chief Executive Officer
Gene Bauer, MD Chief Medical Officer
Luis Peña EVP, Product Development
Andrew Guggenhime Chief Operating Officer & Chief Financial Officer
Experienced leadership Outstanding track record in dermatology and biopharma
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Chris Griffith VP, Corporate Development & Strategy
STANFORD School of Medicine
GENENTECH A Member of
The Roche Group
The next wave of innovation Three late-stage product candidates with positive P2 data
Program Preclinical P1 P2a P2b P3
Next anticipated milestone1
Commercial rights
Topline P3 data 2017
Dermatology rights in U.S.,
Canada
Initiate P3 program
2H15 WW rights
Topline P2b data 1H16 WW rights
Preclinical data 2015 WW rights
Preclinical data 2015 WW rights
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Cimzia Injectable TNF inhibitor (psoriasis)
DRM04 Topical anticholinergic (hyperhidrosis)
DRM01 Topical sebum inhibitor (acne)
DRM02 Topical PDE4 inhibitor (inflammatory diseases)
DRM05 Topical PDT (acne)
1. Estimates provided as of May 12, 2015.
Attractive partnership with UCB Leveraging Dermira expertise to bring Cimzia to dermatology
Structure Dermira promotes to dermatologists in U.S., Canada; UCB retains all other rights International co-development partnership
Profit share Dermira receives share of gross margin1 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%
Development
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
UCB contribution
$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 payments2
CoC provision UCB may terminate if Dermira is acquired by biologic TNF inhibitor company or other non-qualified company3
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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).
Opportunity
~$3.8B psoriasis sales1, 20% CAGR in biologic sales to dermatologists2
~50% of patients remain unsatisfied with available products3
Relatively low biologic penetration, only 10.5% of treated moderate-to-severe patients1
Solution
Status
Differentiated TNF inhibitor with attractive profile for psoriasis
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 20174
Attractive safety, tolerability, P2 efficacy profile
Productive FDA and EMA meetings completed
Cimzia: Differentiated TNF inhibitor Attractive opportunity in large, growing psoriasis market
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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, 2009-13 CAGR in U.S. sales attributed to dermatologists. 3. Armstrong et al., JAMA 2013, data from 2003-2011. 4. Estimate provided as of May 12, 2015.
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 disease2
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
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1. Decision Resources, Immune and Inflammatory Disorders Study, Psoriasis, October 2014. U.S. sales of biologics for psoriasis. 2. National Psoriasis Foundation website – Psoriasis Severity accessed January 2015.
~$3.8B U.S. sales of biologics in 2013 expected to reach $6.2B by 20231
0%
20%
40%
60%
80%
100%
200 mg q2w 400 mg q2w Placebo 50 mg biw Placebo 40 mg q2w PlaceboRes
pons
e ra
te a
t wee
k 12
Cross-study comparison of Cimzia and market leaders1,2
PASI 75 PGA
Attractive competitive profile in psoriasis P2 data support opportunity for differentiated product profile
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.
2. 12-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).
Launch differentiated TNF inhibitor to derms with leading product profile Efficacy comparable to Humira (mAb) with potential safety advantages of
Enbrel (non-mAb) Objectives
Cimzia Phase 2 clinical trial
Pegylated antibody fragment (n=176, p<0.001)
Humira Phase 3 clinical trial Complete antibody (n=1,212, p<0.001)
Enbrel Phase 3 clinical trial
Fusion protein (n=407, p<0.0001)
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DRM04: Topical hyperhidrosis therapy New product opportunity targeting significant unmet need
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Opportunity
~7.8M people suffer from excessive sweating in the U.S.1
Available therapies limited in efficacy, tolerability or administration profile
Highly underpenetrated market (500k annual topical antiperspirant TRx)2
Solution
Status
Reduce sweat production by blocking cholinergic sweat gland receptors
A topical formulation of novel form of anticholinergic, the reference agent3, approved for systemic administration in other indications
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 2H154
1. Strutton et al., Journal of the American Academy of Dermatology, August 2004, data as of 2003. 2. 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.
Hyperhidrosis: Market development opportunity Large, underserved patient population
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 impairment1
~7.8M people in the U.S. have hyperhidrosis, ~50% of whom suffer from axillary disease1
‒ ~33% of axillary patients report sweating that is barely tolerable or intolerable1
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1. Strutton et. al., Journal of the American Academy of Dermatology, August 2004, data as of 2003.
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
Topical formulation for hyperhidrosis
Confirm profile with new form, additional PRO1
Completed P2b bridging trial (n=105)
Supports advancement into P3
Expands IP opportunities
Strategic development program Regulatory path builds on approved systemic reference agent
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Study DRM04-HH01 1st 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)
Study DRM04-HH02 2nd Phase 2b study
Builds on profile of reference agent for hyperhidrosis
1. PRO = patient-reported outcome instrument.
-43.2% -54.4% -60.2%
-73.4% -71.7%
-100%
-80%
-60%
-40%
-20%
0%
Vehicle 1% 2% 3% 4%
22.5%
36.8%
50.0% 52.5% 45.0%
0%
20%
40%
60%
80%
100%
-55.8 -56.6 -67.2
-91.4 -88.4
-130-110-90-70-50-30-10
Vehicle 1% 2% 3% 4%
Positive HH01 results on key efficacy measures Dose-dependence, statistical significance with reference agent
HDSS Response Rate at Week 4 (% of patients w/ ≥2-point improvement)1,3
14 Change in Sweat Production at Week 4
(mg per 5 min.)1,2
p=0.266 p=0.114 p=0.005* p=0.006 (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 non- responders.
Baseline: 110 106 110 120 111
p=0.626 p=0.260 p=0.006* p=0.001*
Abso
lute
Pe
rcen
t
(n=40) (n=38) (n=40) (n=40) (n=40) Vehicle 1% 2% 3% 4%
p=0.194 p=0.011* p=0.006* p=0.036*
27.3%
40.9%
50.0%
0%
20%
40%
60%
80%
100%102 130 97
-48.7%
-79.8% -67.7%
-100%
-80%
-60%
-40%
-20%
0%
(n=22) (n=22) (n=20)
Vehicle DRM04 Dose 1
DRM04 Dose 2
HH02 results consistent with HH01 results Completion of P2 program enables FDA EOP2 meeting
HDSS Response Rate at Week 4 (% of patients w/ ≥2-point improvement)1,3,4
15 Change in Sweat Production at Week 4
(mg per 5 min)1,2,4
p=0.006* p=0.069 (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.
Abso
lute
Pe
rcen
t
-53.9
-105.3
-72.7
-130-110-90-70-50-30-10
p=0.023* p=0.060
Baseline:
p=0.526 p=0.204
Topical treatment well tolerated Low incidence of manageable side effects
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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
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 2H151
Clinical plan leverages positive P2 data Phase 2 program complete, expect P3 initiation 2H151
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1. Estimate provided as of May 12, 2015.
DRM01: Topical sebum inhibitor for acne New MOA for one of the most common skin diseases
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Opportunity
$3.7B global pharmaceutical acne market1, 40-50M U.S. prevalence2
Only 3 major product classes available for >30 years, need for new MOA3
No available topical therapy targets sebum, a key pathogenic factor
Solution
Status
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)
Statistically significant efficacy in FDA-recommended endpoints
P2b dose-finding program initiated April 2015, topline data expected 1H164
1. VisionGain, Dermatological Drugs Market Forecast 2014–2024, April 2014, 2012 sales. 2. American Academy of Dermatology, Acne, Accessed June 2014. 3. IMS Health, VisionGain, Dermatological Drugs Market Forecast 2014–2024, April 2014, acne product package inserts. 4. Estimate provided as of May 12, 2015.
Acne: Large market, significant unmet need $3.7B global market with limited therapeutic options1
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 arthritis3
Acne treatment guidelines recommend targeting multiple pathogenic factors4
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1. VisionGain, Dermatological Drugs Market Forecast 2014–2024, April 2014. 2. American Academy of Dermatology, Acne, Accessed June 2014. 3. Mallon et al., British Journal of Dermatology, April 1999. 4. Gollnick et al., Journal of the American Academy of Dermatology, July 2003. 5. Symphony Health Solutions, Pharmaceutical Audit Suite Data Calendar Year 2007-2012, Gross sales, accessed July 2014.
Product class Sales5 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
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
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Novel molecule with differentiated MOA Targeting ACC, key regulator of sebum production
-13.3
-19.3
-11.2
-19.9
-35
-30
-25
-20
-15
-10
-5
0
Inflammatory lesion count Non-inflammatory lesion count Vehicle DRM01 Vehicle DRM01
N: 54 53 54 53 Avg. baseline lesion count: 28.6 29.7 38.8 40.9
DRM01 improves lesion counts Significant impact on 2 FDA-recommended primary endpoints
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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.
Primary Endpoints: Absolute Changes in Lesion Counts at Week 121
Avg.
abs
olut
e ch
ange
in
lesi
on c
ount
from
ba
selin
e to
wee
k 12
p=0.0003* p=0.0032*
Inflammatory lesion count Non-inflammatory lesion count Avg. % reduction in lesion counts1:
Vehicle DRM01 Vehicle DRM01 45.9% 63.9% 28.8% 48.1%
p-value1 0.0006* 0.0025*
7.3%
24.5%
0%
20%
40%
60%
80%
100%
Vehicle DRM01
DRM01 improves IGA response Significant impact on 3rd FDA-recommended primary endpoint
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Primary Endpoint: IGA Response Rate at Week 121
(n=4/55) (n=13/53)
p=0.0070*
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.
>3-fold improvement
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
23
400-patient P2b study ongoing Standard design based on published FDA draft guidance
0
DRM01 (4% QD)
Week 12
Screening
Randomized, double-blind, vehicle-controlled, dose-ranging trial ‒ ~400 moderate-to-severe adult acne patients1
‒ 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
1. Principal inclusion criteria: Adults with ≥20 inflammatory lesions, ≥20 non-inflammatory lesions and Investigator’s Global Assessment (IGA) score of 3-4.
Vehicle (0% BID)
End
of fo
llow
-up
Treatment period
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Vehicle (0% QD)
DRM01 (7.5% QD)
DRM01 (7.5% BID)
Ran
dom
izat
ion
P2a clinical trial P2b clinical trial (ongoing)
P3 clinical trials (planned)
Objective(s) Clinical POC Dose-finding Confirm safety and efficacy
Population Adult acne patients (n=108)
Adult acne patients (n≈400)
Adult and adolescent acne patients
(n=TBD)
Administration 7.5% gel BID
7.5% gel BID 7.5% gel QD 4% gel QD Vehicle gel BID Vehicle gel QD
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 1H161 TBD
Topline P2b data expected 1H161 Establishing dose(s) for P3 using FDA-recommended endpoints
25
1. Estimate provided as of May 12, 2015.
Strong financial position $158.1M in cash at Mar. 31, 20151
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$ 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 1.9 1.9
Total stockholders’ equity (deficit) 140.9 153.6
Three Months Ended
SELECT STATEMENTS OF OPERATIONS DATA March 31, 2015
March 31, 2014
Operating expenses:
Research and development 10.1 6.7
General and administrative 4.1 1.8
Total operating expenses 14.2 8.5
Net loss (14.0) (8.5)
1. Includes cash and cash equivalents and investments.
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Significant business momentum Key 2015 milestones and expectations
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
Financial
2015 guidance ‒ $80-85M in non-GAAP operating expenses1
‒ ~$90M in cash and cash equivalents and investments as of year-end 2015
24.6M shares outstanding at year-end 2014
1. Excludes impact of stock-based compensation expenses.
Dermira highlights Bringing biopharma innovation to skin disease
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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
Forging the Future of Dermatology
June 2015