Investor Presentation December 2012

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Avis Jackson
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1 Investor Presentation December

2 Safe Harbor Statement Certain items in this presentation and other matters discussed today or answers that may be given to questions asked could constitute forwardlooking statements, including statements regarding the progress and timing of clinical trials, the safety and efficacy of our product candidates, our collaborators, and estimates of the potential markets for our product candidates. Additional risks and uncertainties are described more fully in Anacor s 10-Q for the quarter ended September 30, 2012 filed with the Securities and Exchange Commission. These statements are subject to risks and uncertainties relating to Anacor s future financial or business performance. Anacor s actual results or achievements could differ materially from those anticipated in these forward-looking statements. Please note that Anacor is under no obligation to update any of the forward-looking statements discussed today. 2

3 A Biopharmaceutical Company Developing Multiple Drug Candidates Using Novel and Proprietary Boron Chemistry 3

Anacor Pharmaceuticals - Developing Multiple Drug Candidates Using

Late Stage Clinical Development Tavaborole Phase 3 Topical

three Phase 2 clinical trials Data from first Phase 3 study

sales AN2728 Phase 2 Topical treatment for atopic dermatitis and

2 clinical trials Additional Phase 2 data in atopic dermatitis

Partnerships Anti-infectives Animal Health Acne Prolific Boron

4 Anacor Pharmaceuticals - Developing Multiple Drug Candidates Using Novel and Proprietary Boron Chemistry Two Proprietary Compounds in Late Stage Clinical Development Tavaborole Phase 3 Topical treatment for onychomycosis Demonstrated safety and efficacy in three Phase 2 clinical trials Data from first Phase 3 study expected in January 2013 NDA filing expected in 1H13 Potential $1B sales AN2728 Phase 2 Topical treatment for atopic dermatitis and psoriasis Demonstrated safety and efficacy in 15 Phase 1 and Phase 2 clinical trials Additional Phase 2 data in atopic dermatitis expected in 1H13 Potential $600M+ sales Key Strategic Corporate Partnerships Anti-infectives Animal Health Acne Prolific Boron Chemistry Platform 8 novel small-molecule compounds have begun development in the last 7 years 4

5 Deep Pipeline of Proprietary Drug Candidates with Phase 3 Data in Early 2013 Anacor Owned Tavaborole Onychomycosis UPCOMING MILESTONES Phase 1 Phase 2 Phase 3 1H13 2H Topical Anti-fungals (target fungal LeuRs) Backup compound (AN2718) Ph 3 results File NDA pending Ph 3 results AN2728 Atopic Dermatitis Psoriasis Topical Anti-inflammatories (target PDE4) 1 st Ph 2 study in adolescents completed Ph 3 ready Ph 2 results in adolescent doseranging study Initiate Ph 2 safety/pk/ efficacy study in children Initiate Ph 3 trial in atopic dermatitis pending Ph 2 results Backup compound (AN2898) AN3365 Gram-negative antibiotic (targets bacterial LeuRS) Currently in transition back to Anacor Partnered Animal Health ANXXXX (anti-parasitic) ANXXXX Neglected Diseases AN5568 (HAT/Sleeping Sickness) 5

6 Our Lead Program - Tavaborole Tavaborole Target Product Profile: Safe and effective topical treatment for onychomycosis 6

Onychomycosis a Highly Prevalent Fungal Infection Affecting 35M People in the US Fungal infection of the nail and nail bed Without treatment, it can damage the nail unit and spread to other toes and

7 Onychomycosis a Highly Prevalent Fungal Infection Affecting 35M People in the US Fungal infection of the nail and nail bed Without treatment, it can damage the nail unit and spread to other toes and skin Affects 1 in 10 people Affects 1 in 3 diabetics, increasing risk of secondary infections, foot disorders and limb amputations Local, non life threatening infection - fundamentally ought to be treated topically 7

8 Currently Approved Products Have Limitations Oral treatments (Mostly Lamisil and Sporanox) Safety concerns limit their use despite effectiveness Liver toxicity, drug-drug interactions and unwanted side effects Sporanox received 2 black box warnings (CHF and drug interactions) Topical treatment (Penlac is the only approved topical) Safe but low efficacy due to inability to penetrate nail Inconvenient to apply To cure onychomycosis, the optimal topical drug must be small, water soluble and not bind to keratin to penetrate the nail and reach the nail bed Fungus resides in nail and nail bed Nail s thickness and construction of compact keratin matrix and water create barrier for most topically applied agents Topical drug must penetrate nail plate to treat fungus in nail bed 8

9 Due to Limitations of Approved Products, Most Patients are Treated with Debridement Untreated ~20 million people Unmotivated to treat Don t like treatment options Uninformed Oral Medication (primarily Lamisil) ~1-2 million people Most effective treatment option Safety concerns and unwanted side effects have limited the use of oral medication 55% Untreated 30% Debridement 5% Oral 10% Topical Debridement ~8-10 million people Visit podiatrists ~3 times per year for debridement (cutting, clipping, scraping or removing the nail) to improve appearance and comfort Does not cure the infection Topical Treatments (Penlac and OTC remedies) ~ 3 million people Demonstrated little to no efficacy Source: Estimated based on Podiatry Today, IMS, AC Nielsen, Medicare and Alpha Detail data 9

10 While Complete Cure is the FDA s s Basis for Efficacy, Mycological Cure is an Important Secondary Endpoint Culture Negative Culture is the attempt to grow a fungus from a nail bed scraping Negative means no dermatophyte was isolated on mycological culture media Negative KOH Microscopic evaluation of material collected from the nail bed and treated with potassium hydroxide (KOH) looking for fungal elements Negative KOH indicates an absence of microscopically detectable hyphae from toenail samples treated with KOH Clinical Cure 100% normal appearing nail Clear Nail Growth (CNG) Measured nail growth (mm) of new, normalappearing nail Best interim evidence of drug efficacy Mycological Cure Complete Cure 10

11 In Spite of Limitations, Currently Approved Products Had Combined Peak Sales Over $2B Lamisil Oral Sporanox Oral Penlac Topical ~$1.2B peak WW sales Most effective but safety concerns ~$600M peak WW sales Low efficacy and safety concerns ~$300M peak WW sales Low efficacy and inconvenient 2010 NRx (a) 1.4M 23K 375K Complete Cure (Mycological Cure + 100% clear nail) Mycological Cure (Negative Culture and Negative KOH) Price per course of treatment (branded) Potential Side Effects Inconvenience/ Other Issues 38% 14% 5.5% - 8.5% 70% 66% 29% - 35% $1,172 (3 months of treatment) Liver toxicity Drug-drug interactions Diarrhea Rash Smell and taste disturbance $2,050 (3 months of treatment) 2 Black Box Warnings Liver toxicity Drug-drug interactions Cardiac complications Rash GI disorders Liver enzyme test at onset and 6 weeks Older / diabetic patients may be on multiple other oral medications and don t want to or can t add an oral treatment for onychomycosis $664 (1 year of treatment) <5% skin irritation Lacquer must be applied daily, allowed to dry and removed weekly Approved as adjunctive therapy to debridement (a) Source: IMS. Includes generics. 11

Our Solution for Onychomycosis Safe Effective Local, targeted therapy Little or no detectable systemic exposure All preclinical

for oil and water (logp = 1.24) Water soluble (0.

effective than current topical options and safer than current oral therapeutics Potent against Baseline broad spectrum of fungi

12 Our Solution for Onychomycosis Safe Effective Local, targeted therapy Little or no detectable systemic exposure All preclinical toxicology completed Penetrates Nail Small molecular weight 152 Da compared to > 300 Da for most antifungals Balanced preference for oil and water (logp = 1.24) Water soluble (0.8 mg/ml) Activity in presence of keratin Tavaborole A safe and effective topical treatment for onychomycosis that is more effective than current topical options and safer than current oral therapeutics Potent against Baseline broad spectrum of fungi and yeast Unique MOA - targets LeuRS to kill fungus Demonstrated efficacy in three Phase 2 trials Easy to Use Apply with dropper once daily Dries in about one minute No special cleansing or preparation prior to application 12

13 Tavaborole Penetrates Nail 250x Better than Penlac to Reach Fungus that Resides in Nail Bed 2.5 In Vitro Human Nail Penetration Model Study Nails washed and dosed daily with Tavaborole or Penlac Flux (mg / sample) Days 0-3 Days 4-6 Days 7-9 Days Days Total Tavaborole Penlac Presented at the Society for Investigative Dermatology 67 th Annual Meeting. Poster is available at 13

Current Marketed Products Achieve Peak Negative Culture of ~70% - 90% After 6 Months of

Time Lamisil Placebo (1) Lamisil (1) Lamisil (2) Lamisil (3) Sporanox (2) 0 Drug Treatment:

Tavaborole 201 Cohort 3 1Drake et al., J Am Acad Dermatol 1997, 37: 740-5 2De Backer et al.

14 Current Marketed Products Achieve Peak Negative Culture of ~70% - 90% After 6 Months of Treatment 100% 90% 80% 70% 60% 50% 40% 30% 20% 10% Patients Achieving Negative Culture Over Time Lamisil Placebo (1) Lamisil (1) Lamisil (2) Lamisil (3) Sporanox (2) 0 Drug Treatment: Days Lamisil, Sporanox Tavaborole 200, 201*, 203 Tavaborole 201 Cohort 3 1Drake et al., J Am Acad Dermatol 1997, 37: De Backer et al., J Am Acad Dermatol 1998, 38: S Schroeff et al. Br J Dermatol 1992, 126: *Cohorts 1 and 2 14

15 In Phase 2, Tavaborole Achieved ~90% - 100% Negative Culture Within 30 Days of Treatment 100% 100% 90% 90% 80% 80% 70% 70% 60% 60% 50% 50% 40% 40% 30% 30% 20% 20% 10% 10% Days Drug Treatment: Lamisil, Sporanox Tavaborole 200, 201*, 203 Tavaborole 201 Cohort 3 Patients Achieving Negative Culture Over Time Lamisil Placebo (1) Lamisil (1) Lamisil (2) Lamisil (3) Sporanox (2) vehicle Tavaborole Vehicle 1% tavaborole Tavaborole tavaborole % Tavaborole 200 5% tavaborole Tavaborole % tavaborole Tavaborole C1 C1 5% tavaborole Tavaborole C3 C3 tavaborole 203 5% Tavaborole 203 tavaborole % Tavaborole 200 tavaborole 201 C2 7.5% Tavaborole 201 C2 0% Drake et al., J Am Acad Dermatol 1997, 37: De Backer et al., J Am Acad Dermatol 1998, 38: S Schroeff et al. Br J Dermatol 1992, 126: *Cohorts 1 and 2 Anacor Phase 2 data presented at the 100 th National APMA Scientific Meeting in August

Marketed Products Achieve Peak Mycological Cure Between 30% - 75%

Achieving Mycological Cure Over Time Lamisil (2) Lamisil (4)

Penlac (3) 30% 20% 10% 0 30 60 90 120 150 180 210 240 270 300 330

203 Penlac Tavaborole 201 Cohort 3 1FDA Summary Basis of Approval

, J Am Acad Dermatol 1998: 38: S57-63 3FDA Summary Basis of

16 Marketed Products Achieve Peak Mycological Cure Between 30% - 75% After More than 6 Months of Treatment 80% 70% 60% 50% 40% Patients Achieving Mycological Cure Over Time Lamisil (2) Lamisil (4) Sporanox (2) Penlac Vehicle (1) Penlac (1) Penlac Vehicle (3) Penlac (3) 30% 20% 10% Days Drug Treatment: Lamisil, Sporanox Tavaborole 200, 201*, 203 Penlac Tavaborole 201 Cohort 3 1FDA Summary Basis of Approval for Loprox (ciclopirox), Trial 312 2De Backer et al., J Am Acad Dermatol 1998: 38: S FDA Summary Basis of Approval for Loprox (ciclopirox), Trial 313 4Schroeff et al. Br J Dermatol 1992, 128: 36-9 *Cohorts 1 and 2 16

In Phase 2, Tavaborole Achieved Greater Mycological Cure Faster than Approved Products in all but 1 Cohort 80% 70% 60% 50% 40% 30% 20% 10% 0 0 30 60 90 120 150 180 210 240 270 300 330 360 0 30 60 90

17 In Phase 2, Tavaborole Achieved Greater Mycological Cure Faster than Approved Products in all but 1 Cohort 80% 70% 60% 50% 40% 30% 20% 10% Days Drug Treatment: Lamisil, Sporanox Tavaborole 200, 201*, 203 Penlac Patients Achieving Mycological Cure Over Time Lamisil (2) Lamisil (4) Sporanox (2) Penlac Vehicle (1) Penlac (1) Penlac Vehicle (3) Penlac (3) Tavaborole Vehicle 1% Tavaborole % Tavaborole 200 5% Tavaborole 200 5% Tavaborole 201 C1 5% Tavaborole 201 C3 5% Tavaborole % Tavaborole % Tavaborole 201 C2 vehicle 1% tavaborole % tavaborole 200 5% tavaborole 200 5% tavaborole 201 C1 5% tavaborole 201 C3 5% tavaborole % tavaborole % tavaborole 201 C2 Tavaborole 201 Cohort 3 1FDA Summary Basis of Approval for Loprox (ciclopirox), Trial 312 2De Backer et al., J Am Acad Dermatol 1998: 38: S FDA Summary Basis of Approval for Loprox (ciclopirox), Trial 313 4Schroeff et al. Br J Dermatol 1992, 128: 36-9 *Cohorts 1 and 2 Anacor Phase 2 data presented at the 100 th National APMA Scientific Meeting in August

Phase 2 tavaborole trials demonstrate generally dose responsive effects on Clear Nail Growth that are lower than the oral competitors at 6 months, but much better than vehicle and placebo 8 mm 8mm 7

Tavaborole Vehicle 1% Tavaborole 203 1% tavaborole 203 2.5% Tavaborole 200 2.

18 Phase 2 tavaborole trials demonstrate generally dose responsive effects on Clear Nail Growth that are lower than the oral competitors at 6 months, but much better than vehicle and placebo 8 mm 8mm 7 mm 7mm 6 mm 6mm 5 mm 5mm 4 mm 4mm 3 mm 3mm 2 mm 2mm 1 mm 1mm Mean Clear Nail Growth Over Time vehicle Lamisil Placebo (1) Lamisil (1) Lamisil (2) Lamisil Placebo (3) Lamisil (3) Sporanox (2) Tavaborole Vehicle 1% Tavaborole 203 1% tavaborole % Tavaborole % tavaborole 200 5% Tavaborole 200 5% tavaborole 200 5% Tavaborole 201 C1 5% tavaborole 201 C1 5% Tavaborole 201 C3 5% tavaborole 201 C3 5% Tavaborole 203 5% tavaborole % Tavaborole % tavaborole % Tavaborole 201 C2 7.5% tavaborole 201 C2 0 0mm -1mm -1-2mm Days Drug Treatment: Lamisil, Sporanox Tavaborole 200, 201*, 203 Tavaborole 201 C3 1Drake et al., J Am Acad Dermatol 1997, 37: De Backer et al., J Am Acad Dermatol 1998: 38: S Goodfield et al., BMJ 1992, 304: *Cohorts 1 and 2 Anacor Phase 2 data presented at the 100 th National APMA Scientific Meeting in August

19 180 Days of Treatment with Tavaborole,, 5% Negative Mycology and >2mm Clear Nail Growth 360 Day Follow-up Negative Mycology and 12.5mm Clear Nail Growth KOH: Positive Culture: Positive Baseline KOH: Negative Day 180 Culture: Negative 10.1mm 2.5mm KOH: Negative Culture: Negative 12.1mm Day 240 KOH: Negative Day 360 Culture: Negative 15.0mm AN2690-ONYC-200 Subject IOG 19

20 Tavaborole Demonstrated Clinical Efficacy in Three Phase 2 Clinical Trials % of Subjects Reaching Primary Endpoint at 6 Months 43% 53% 50% 30% 27% 26% 32% 14% 14% 5.0% n=30 Cohort 1 7.5% n=30 Cohort 2 5.0% n=29 Cohort 3 1.0% n=30 5.0% n=30 Vehicle n=63 2.5% n=33 5.0% n=31 7.5% n=60 Study 201 Phase 2 objectives Evaluate safety and efficacy at various drug concentrations and dosing regimens Study 203 Key inclusion criteria Positive culture and positive KOH At least 2mm clear nail from proximal end 20% - 60% nail involvement Study 200 (p<0.03) Primary endpoint at 6 mo. Negative culture At least 2mm clear nail growth 20

21 Conclusions from Tavaborole Phase 2 Trials Tavaborole is more efficacious than vehicle alone as demonstrated with statistical significance in a 187 patient trial Topical irritation is the only safety signal seen in any of these trials. Irritation appears to be dependent on: Drug concentration Frequency of application Dosing instruction (irritation appears to increase with more liberal dosing) All dosing regimens tested were efficacious, with efficacy dependent on: Drug concentration (1% is less efficacious than higher doses) Dosing instructions (more liberal dosing appears to improve efficacy) Duration of treatment (although not tested in Phase 2, experts believe that re-infection lowers 12 month efficacy if dosing is stopped) Severity of disease in patients at baseline Frequency of application did not appear to impact efficacy, but less frequent dosing creates a potential compliance problem e.g., daily dosing is easier to remember than 3x per week 21

22 Tavaborole is Currently in Phase 3 with Data From First Study Expected in January 2013 Overview of Phase 3 Studies Design ~600 patients per trial, randomized 2:1 Study 301 (US & Mexico) Study 302 (US & Canada) 95% power to show 10 percentage point difference between active and vehicle Assumes 20% patient dropout Expected efficacy between 15% and 35% Dosing 5% concentration Dosed daily (P) (L) Changes from Phase 2 that were made to Optimize Efficacy and Tolerability 5% concentration is more efficacious than 2.5% and had fewer episodes of irritation than 7.5% Dosing Instructions Treatment Duration Inclusion Criteria Primary endpoint Wet toenail to cover entire surface to the edges, drip under top edge of infected nail, and wipe off any excess from surrounding skin Daily dosing easier to remember for patients thus improving compliance More specific instructions to balance maximum efficacy and minimal irritation 48 weeks (P) Longer treatment duration reduces risk of reinfection Positive culture and KOH (P) (L) Minimum 3mm clear nail (L) 20% - 60% nail involvement (P) Negative mycology and 100% clear nail at 52 weeks (P) (L) Increased clear nail at initiation reduces risk that fungus resides in nail matrix Blinded physician panel reviews of photos to ensure patients meet inclusion requirements Required by FDA (P) Similar to or same as Penlac s Phase 3 (L) Similar to or same as Lamisil s Phase 3 22

23 Tavaborole Summary Tavaborole has the potential to be a safe and effective topical treatment for onychomycosis 2 Phase 3 studies progressing well Retention rate and compliance better than assumptions AE s reported are mild skin irritation with few drug holidays or withdrawals Data from study 301 will be announced in January 2013 and from study 302 ~ 6 weeks later Safety, toxicology and carcinogenicity studies are complete Preclinical safety studies demonstrate promising safety profile Not genotoxic Nonsensitizing Minimal systemic exposure when applied topically No evidence of teratogenicity Clean safety profile when given systemically Considered to be non-carcinogenic Recently completed Phase 1 safety studies TQT results demonstrate no effect on heart rate RIPT (Repeat insult patch test) indicate that tavaborole is not a sensitizing agent Phase 3 efficacy expected to be at least twice that of Penlac, or similar to Sporanox, and approaching that of Lamisil 23

24 Timeline Indicates NDA Approval Mid H13 2H13 1H14 January - Phase 3 Study 301 data Later in Q1 Phase 3 Study 302 data Submit NDA FDA Review NDA Approval Mid-2014 Note: timeline based on Anacor s current best estimates for NDA filing and FDA review dates 24

25 Physician Surveys Indicate that Tavaborole Would Meet an Unmet Medical Need 100% Mean % of Patients who Would be Prescribed Tavaborole 80% 60% 50% 55% 50% 40% 20% 35% 25% 25% 0% Podiatrists Dermatologists PCP s Low Case: Efficacy Similar to Sporanox or Twice that of Penlac Podiatrists Dermatologists PCP s High Case: Efficacy Approaching that of Lamisil Note: Percentages based on 29 physicians surveyed. Findings are directional and should not be viewed as conclusive. Source: D.S. Howard and Associates, August

26 Tavaborole Peak U.S. Sales are Estimated to be $700M to $1B+ Podiatrists Dermatologists PCPs and other MDs Estimated number of patients diagnosed annually by type of physician 8 million 0.3 million 2.0 million Assumptions: Low Case High Case Efficacy (complete cure) ~15% (similar to Sporanox or twice ~35% (approaching Lamisil) that of Penlac) Patient Penetration 15% (50% of survey results) 25% (50% of survey results) New Rx s (podiatrists and dermatologists only) 1.3 million 2.1 million Price per course of therapy $75 - $150 per month or $900 - $1,800 per course of therapy Patient compliance 60% 60% Estimated Peak U.S. Sales (podiatrists and dermatologists only) $700M - $1.0B+ 26

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