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Certain C t i items it in i thi this presentation t ti and d other th matters tt di discussed dt today d or answers that th t may be given to questions asked could constitute forward-looking 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 Anacors Annual Report on Form 10-K for the year ended December 31, 2012 and subsequent quarterly reports filed on Form 10-Q filed with the Securities and Exchange Commission. These statements are subject to risks and uncertainties relating to Anacors future financial or business performance. Anacors 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 y discussed today.
A Biopharmaceutical Company Developing Multiple Drug Candidates Using Novel and Proprietary Boron Chemistry
Anacor Owned
Tavaborole
(topical anti-fungal)
Phase 1
Phase 2
Phase 3
1H13
2H13
2014
Potential FDA Approval
Onychomycosis
Backup compound (AN2718)
Pre-NDA communications with FDA completed successfully File NDA mid 2013
AN2728
AN3365
(gram-negative antibiotic)
Partnered
Animal Health 1 Animal Health 2 AN5568 (HAT/Sleeping
Sickness)
Target Product Profile: Safe and effective topical treatment for onychomycosis
Tavaborole b l
} }
Affects 1 in 10 people
Affects 1 in 3 diabetics, increasing risk of secondary infections, foot disorders and limb amputations
Topical treatment
Penlac
is the only approved topical and was approved with concomitant debridement
Safe but low efficacy due to inability to penetrate nail Inconvenient to apply
To cure onychomycosis onychomycosis, the optimal topical drug must be small small, water soluble and not bind to keratin to penetrate the nail and reach the Fungus resides in nail bed
nail and nail bed Nails thickness and construction of compact keratin matrix and water create barrier for most tt topically i ll applied li d agents
Topical drug must penetrate nail plate to treat fungus g in nail bed
Due to Limitations of Approved Products, Most Patients are Treated with Debridement
55% Untreated
30% Debridement
podiatrists ~3 times per year for debridement (cutting, clipping, scraping or removing the nail) to improve appearance and comfort not cure the infection
} Does
effective treatment option concerns and unwanted t d side id effects ff t have limited the use of oral medication
} Safety
efficacy
little to no
Source: Estimated based on Podiatry Today, IMS, AC Nielsen, Medicare and Alpha Detail data
9
In Spite of Limitations, Currently Approved Products Had Combined Peak Sales Over $2B
Lamisil (terbinafine) Oral
~$1.2B peak WW sales Most effective but safety concerns 2010 NRx (a) Complete Cure (Mycological ( y g Cure + 100% clear nail) Mycological Cure (Negative Culture and Negative KOH) Price per course of treatment (prior to generic entry) 1.4M 38%
70%
66%
Liver enzyme test at onset and 6 weeks patients may y be on multiple p other oral Older / diabetic p medications and dont want to or cant add an oral treatment for onychomycosis
Lacquer must be applied daily, y allowed to dry y and removed weekly Approved as adjunctive therapy to debridement
(a) Source: IMS. Includes generics. (b) AWP of branded Penlac increased to $3,616 per course of therapy as of 3/1/13
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Effective
} } }
Local, targeted therapy Little or no detectable systemic exposure All preclinical toxicology completed
Potent against broad spectrum Baseline of fungi and yeast Unique MOA - targets LeuRS to kill fungus Demonstrated efficacy superior to Penlac in first Phase 3 study
Penetrates Nail
}
A safe and effective topical treatment for onychomycosis that is more effective than current topical options and safer than current oral therapeutics
Tavaborole
Easy to Use
Apply with dropper once daily Dries in about one minute No special cleansing or preparation prior to application
} } }
Small molecular weight 152 Da compared to > 300 Da for most antifungals Balanced preference for oil and water (logP = 1.24) 1 24) Water soluble (0.8 mg/mL) Activity in presence of keratin
} } }
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Tavaborole Met All Endpoints With Statistical Significance In Two Phase 3 Trials
Studies included patients of all ages with no upper age limit and onychomycosis involving 20% - 60% of target great toenail
Study 301 (N = 594) (active / vehicle) Primary Endpoint p at 52 Weeks Completely clear nail and mycological cure (defined as negative ( g culture and negative g KOH) of target great toenail 6.5% / 0.5% (p=0.001) (p ) 26.1% / 9.3% (p<0.001) 31.1% 31 1% /7.2% /7 2% (p<0.001) 15.3% / 1.5% (p<0.001) 87.0% / 47 47.9% 9% (p<0.001) 24.6% / 5.7% (p<0.001) 2.8% / 1.6% Study 302 (N = 601) (active / vehicle) 9.1% / 1.5% (p<0.001) (p ) 27.5% / 14.6% (p<0.001) 35.9% 35 9% / 12 12.2% 2% (p<0.001) 17.9% / 3.9% (p<0.001) 85.4% / 51 51.2% 2% (p<0.001) 25.3% / 9.3% (p<0.001) 0.8% / 0.5%
Secondary Completely clear or almost clear (10% Endpoints at clinical involvement) target great toenail 52 Weeks Mycological M l i l cure of ft target t great t toenail t il Completely clear or almost clear nail + mycological cure Other Negative culture Completely clear or almost clear nail and negative culture Safety Rate of discontinuations as a result of adverse events
Overall safe and well-tolerated across study subjects No serious adverse events related to study drug
12
Clinical Photographs from Tavaborole Phase 3 Studies 26.1% and 27.5% of Patients Reached Completely Clear or Almost Clear Nails in Studies 301 and 302 Baseline
Completely Clear Nail With Mycological Cure
Week 52
Week 52 Baseline
Almost Clear Nail With Mycological Cure
13
Tavaborole Has Demonstrated Greater Efficacy and is More Convenient to Use than Penlac
Penlac (1) (ciclopirox lacquer) Nail debridement, as often as monthly Lacquer painted on nail daily Weekly removal of Penlac with alcohol (Active / Vehicle) 6 5% / 0.9% 6.5% 0 9% (Study 312) 12.0% / 0.9% (Study 313) 29.0% / 11.0% (Study 312) 36.0% / 9.0% (Study 313) 5.5% / 1.0% (Study 312) 8.5% / 0.0% (Study 313) N/A
Phase 3 Efficacy Results Completely clear or almost clear nail + mycological cure Mycological cure Completely clear nail and mycological cure Completely clear or almost clear nail
(Active / Vehicle) 15 3% / 1.5% 15.3% 1 5% 31.1% / 7.2% 6.5% / 0.5% 26.1% / 9.3
(Active / Vehicle) 17 9% / 3.9% 17.9% 3 9% 35.9% / 12.2% 9.1% / 1.5% 27.5% / 14.6%
Tavaborole Could Represent an Important New Treatment Option for Onychomycosis Patients
}
Demonstrated efficacy and safety in two Phase 3 trials conducted under an SPA
No upper age limit on patients Onychomycosis involving 20% - 60% of target great toenail No debridement was allowed
16
Tavaborole NDA on schedule to be filed with the FDA in mid-2013 In pre-NDA communications with FDA, Anacor achieved concurrence from the FDA on all major areas of focus for the NDA: Safety Efficacy Manufacturing Packaging
Phase 3 data to be presented in an oral presentation at the American Podiatric Medical Association Annual Meeting on July 22, 22 2013 Poster authors Lee Zane, MD Richard Ri h d Pollak, P ll k DPM Max Weisfeld, DPM
17
Background
In November 2012, Valeant published Phase 3 data for efinaconazole, a triazole antifungal developed for the topical treatment of onychomycosis
In October 2012, Anacor provided notice to Valeant Pharmaceuticals International, Inc. (successor in interest to Dow Pharmaceutical Sciences, Inc. (Dow)) seeking to commence arbitration of a breach of contract dispute under a master services agreement dated March 26, 26 2004 between Anacor and Dow related to certain development services provided by Dow in connection with our efforts to develop our onychomycosis nail-penetrating anti-fungal product We are seeking injunctive relief and damages of at least $215.0 million We have carefully reviewed our position and we believe that we have meritorious claims We currently estimate that the arbitration will conclude in the second half of 2013
Valeant agreed to postpone launch of efinaconazole until after final arbitration hearing in September 2013
} }
18
Target Product Profile: Safe and effective topical l treatment f for atopic dermatitis and psoriasis
AN2728
19
Existing Therapies Lack Safety Needed for Treating a Disease that Primarily Affects Children
Topical Corticosteroids Topical Calcineurin Inhibitors (Protopic and Elidel)
Recommended Use Low potency steroids safe for 2nd line therapy when (by American children in short intervals continued use of topical Academy of High potency steroids should be steroids is ineffective or use of Dermatology) avoided in high risk areas such steroids is inadvisable as face or skin folds Advantages Di d Disadvantages Anti-inflammatory Fair to good efficacy
L Local l side id effects ff - skin ki Received R i d Bl Black kB Box warning i thinning, acne, stretch marks from FDA in 2005 Systemic side effects HPA Prior to Black Box warning, Axis suppression considered breakthrough ~72.5% 72.5% of parents worry about products due to perceived using them on children (1) safety relative to corticosteroids ~24% of parents admitted to not using them on children due to safety concerns (1)
Sales Ramp of Protopic and Elidel Demonstrates Demand for Safe Treatment Option
Protopic and Elidel Generated Over $500M in Sales Prior to FDA Black Box Warning in 2005
Protopic and Elidel WW Sales
($ in millions)
Elidel
Protopic
Black Box Warning
2005
2006
2007
2008
2009
2010
Effective
}
Topical application limits systemic y exposure p 16 clinical studies to date demonstrate a promising safety profile
AN2728
Target product profile Baseline efficacy equal to topical calcineurin inhibitors Three Phase 2 studies have demonstrated efficacy treating adults and adolescents with atopic dermatitis
AD-202
AD-203
AD-204
Objective
Human proof of concept in adults 25 adults with atopic dermatitis and 2 target lesions of similar severity
Safety, PK, and efficacy in adolescents 23 adolescents (12-17 years) with atopic dermatitis affecting 10% 35% BSA Whole body Open-label p Patients applied AN2728 ointment, 2% to all treatable areas of atopic dermatitis BID for 4 weeks
Dose-ranging study in adolescents 86 adolescents (12-17 years) with atopic dermatitis and 2 target lesions of similar severity Bilateral Double-blind Randomized 1:1 to treat lesions BID or QD Patients treated one lesion with AN2728, 2% and a comparable lesion with AN2728, 0.5% for 4 weeks ADSI assessed at 8,15, 8 15 22, and 29 days
Patients
Design
Bilateral Double-blind Randomized 1:1, active: vehicle Patients treated one lesion with AN2728, 2% and a comparable lesion with vehicle BID for 6 weeks ADSI assessed at 2,4 24 and 6 wks with primary endpoint at 4 wks
Outcomes
24
ISGA (Investigator Static Global Assessment) Used to assess severity of all of the atopic d dermatitis titi on a patients ti t body b d 5 point scale (0-4)
Score 0 Grade Clear Definition Minor residual discoloration; no erythema or induration/ papulation; no oozing/ crusting
(redness) } Pruritus (itch) } Exudation (oozing/crusting) } Excoriation (evidence of scratching) } Lichenification (skin thickening)
Trace faint pink erythema, with barely perceptible induration/ Almost Clear papulation and no oozing/ crusting Mild Faint pink erythema with mild induration/papulation and no oozing/crusting Pink-red erythema with moderate induration/ papulation with or without oozing/ crusting Deep or bright red erythema with ith severe induration/ i d ti / papulation and with oozing/ crusting
Score 0 1 2 3
25
Study ADAD-204: AN2728 Ointment, 2% Applied BID Demonstrates Greatest Improvement from Baseline ADSI Score; Treatments Show Excellent Dose Response
26
Study ADAD-204: AN2728 Ointment, 2% Applied BID Demonstrates Greatest Improvement in the Individual Components of Atopic Dermatitis After 4 Weeks
27
Study ADAD-204: 62% of Lesions Treated with AN2728 Ointment, 2% BID Were Clear or Almost Clear After 4 Weeks
28
AN2728-ADAN2728AD-204 Results in Adolescents Compare Favorably to ADAD-202 Results in Adults at 4 Weeks AN2728 Ointment, 2% Dosed Twice Daily for 4 Weeks
29
Study ADAD-203: AN2728 Demonstrated Efficacy in Adolescents Using Anticipated Phase 3 Efficacy Measure
} }
Open-label study in adolescents with atopic dermatitis 10%-35% BSA 23 patients treated all atopic dermatitis lesions with AN2728 ointment, 2% BID for 4 weeks Investigator Static Global Assessment (ISGA) assessed weekly
Day 8
Day 15
Day 22
Day 29
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Study ADAD-203: Efficacy Compares Favorably to Elidel, an Established Atopic Dermatitis Therapy
Caution is needed when comparing AN2728s open-label Phase 2 data with randomized, controlled Phase 3 data for Elidel } Pivotal Pi t l Ph Phase 3 d data t f for Elid Elidel l categorized t i d outcomes t f for ISGA after 6 weeks of treatment versus the 4-week course of therapy in the AN2728-AD-203 study
}
Elidel / vehicle
(1)
Outcome Clear/Almost Clear Clear/Almost Clear ( ith 2-grade (with 2 d improvement) Clear
Study ADAD-202: AN2728 Demonstrated Safety and Efficacy in Human Proof of Concept Study in Adults with Atopic Dermatitis
} }
Bilateral study in adults with atopic dermatitis 35% BSA 25 patients treated target lesions with AN2728 ointment, 2% vs. vehicle, BID for 6 weeks Atopic Dermatitis Severity Index (ADSI) assessed at 2, 4, 6 weeks (Primary endpoint at 4 wks)
% of Lesions with Greater Improvement in ADSI than Vehicle at Day 28 (p=0.02) % Improvement in ADSI at Day 28 (p=0.01) % of Lesions Achieving g Total or Partial Clearance (ADSI 2) by Day 28 (p=0.0027)
68%
16%
AN2728 Has Demonstrated Safety in the Treatment of Adults and Adolescents with Atopic Dermatitis
Atopic dermatitis is commonly associated with allergies and sensitivity. Many atopic dermatitis patients are sensitive to contact with soaps, fragrances, etc. Therefore, a non-irritating drug is critical, but almost all clinical trials will show some application site reactions.
}
AD-202
No severe adverse events reported that were considered related to study drug
AD-203
Generally G ll safe f and d well-tolerated ll l d with i h most common AEs AE being b i application li i site i reactions i
No serious adverse events (SAEs); one discontinuation due to AE
Pharmacokinetic data:
Blood levels are generally low and proportional to body surface area treated Overall ll pharmacokinetics h k are similar l to those h seen in adults d l after f adjusting d f for percent b body d surface f area treated }
AD204
No serious adverse events and no discontinuations due to drug-related adverse events (AEs) 22 AEs in 86 subjects
Severity: 91% mild, 9% moderate, none severe Relatedness: 82% not related to study drug
33
In three Phase 2 studies in atopic dermatitis, data for AN2728 Ointment, 2% dosed BID continue to support its target product profile p o for o the treatment a of o atopic a op dermatitis d a in the age ag groups g oup tested d to date Next steps in development:
2 additional safety studies prior to initiating Phase 3: MUSE (maximal use systemic exposure) study in ~30 children with atopic dermatitis to measure blood levels when AN2728 is applied under maximal use conditions TQT study - to assess the effects of AN2728 following multiple-dose administration on electrocardiograms (ECGs) in ~180 healthy volunteers Initiate Phase 3 program of AN2728 development in 4Q13 or 1Q14 Timing dependent upon completion of MUSE study Anticipate approximately 500 patients 2-17 years of age in each Phase 3 trial
34
Corporate Partnerships
35
$3.5M upfront, minimum $6M and up to $12M in research funding Research for a variety of animal health applications ($20 billion market) First development candidate, selected in August 2011 and second development candidate selected in December 2012 - $1M milestone payment received for each
Lilly responsible for worldwide development and commercialization Significant development milestones $350M - $500M potential market opportunity per candidate with high single-digit to low-double digit royalties on sales
Over $75M paid to Anacor to date including $12M upfront + $30M investment in Anacor
Option to license compounds at PoC; 6-year research term GSK Licensed AN3365 (also known as GSK 052) in July 2010 - $15M milestone payment received Expanded p work around bacterial LeuRS, , added program p g to fund Anacors TB research
Op
5Preclinical P li i l 5Safety
5Phase
Parasitic Diseases
Af i African Sl Sleeping i Si Sickness k (HAT) Visceral Leishmaniasis Chagas disease Mala ia Lead Se Malaria Series ies Malaria (New Scaffolds)
SCYX7158 / AN5568
Bacterial Diseases
Tuberculosis (TB) LeuRS TB (non-LeuRS) TB new targets
37
38
Low COGS and Specialty Salesforce Costs Should Lead to Strong Operating Margins
Financial Summary
($ in 000s)
March 31, 31 2013 Cash, cash equivalents and short-term investments (1) T t l assets Total t Notes payable Accumulated deficit Total stockholders equity Total shares outstanding (2) $ $ $ $ $ 32,376 37 378 37,378 23,516 (230 289) (230,289) (7,795) 40,494,466
(1) Excludes $5.0M proceeds from Gates equity investment and $21.4M net proceeds from follow-on public offering in April 2013. (2) As of May 3, 2013.
40
Thank you
41
42
Vectical
Active ingredient: Calcitriol (vitamin D)
27%
23% 14%
21%
16%
7%
8 weeks1 of therapy
Active
Vehicle
Active
Vehicle
Active
Vehicle
Active
Vehicle
Taclonex Taclonex
Active ingredients: Betamethasone Dipropionate (high-potency steroid) & Calcipotriene (vitamin D analog) absent or very y mild with 2- grade improvem ment in IGA
48%
26% 17% 8%
4 weeks of therapy
BetamethasoneDipropionate (Highpotencysteroid)
Calcipotriene (VitaminDanalog)
Vehicle
44
Boron-based compound Inhibits PDE4 Reduces production of TNF-a, IL-12, IL-23 Target product profile efficacy is equal to midpotency steroid Four Phase 2 trials have demonstrated this level of efficacy
Baseline
} }
Safety
} } }
Day 84
Topical application limits systemic exposure Potential for long duration of treatment Clinical trial results have shown a promising safety profile
45
AN2728 has demonstrated efficacy in the range of mid-potency steroids and vitamin D analogs Results of Local Tolerability Study confirm potential to expand indication to patients with inverse psoriasis which involves the face, skin folds, recesses, and genitalia AN2728 is Phase 3 ready for the treatment of mild-to-moderate psoriasis
46
Phase 2b (Dose(Dose-ranging Study in Psoriasis) Clear Dose Response Over 12 Weeks of Treatment
47
Key objectives
Provide data to inform plans for anticipated Phase 3 trial design and End of Phase 2 meeting with FDA
Safety and tolerability with larger body surface area treated than previous Phase 2 trials Level of efficacy with subject to subject comparison, rather than bilateral Time to peak efficacy with whole body treatment
Not intended to demonstrate statistical differentiation of treatment from vehicle, but to inform a fully-powered pair of pivotal Phase 3 trials
}
Design
12 week, double-blind, subject-to-subject comparison 68 patients randomized 2:1 AN2728 Ointment, 2%, vs. Vehicle BID
Clinical evaluations
Safety, efficacy and duration of treatment under anticipated Phase 3 conditions
48
Phase 2b (204 Study) AN2728 Demonstrated Improvement Over Vehicle in Psoriasis at Each Recorded Timepoint
Proportion of Subjects Achieving Clear or Almost Clear with 2-Grade Improvement from Baseline (PGA scale)
17% 14%
49
Day 1
Day 42
Day 84
50
AN2728 Ointment, 2% appears to be safe and well-tolerated when applied to sensitive skin areas
Adverse events occurred at a low rate and were generally mild None of the treated anatomic areas appeared to be particularly sensitive to irritation by the study drug
Reduces R d risk i k of f treating t ti areas of f inverse i psoriasis i i in i Phase Ph 3, 3 increasing potential for non-restrictive psoriasis indication Increases probability of safe and well-tolerated treatment of areas typically affected by atopic dermatitis
51