smarter drug delivery in dermatology
TRANSCRIPT
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1 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
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This presentation may include “forward-looking statements” within the meaning ofSection 27A of the Securities Act of 1933, as amended, and Section 21E of theSecurities Exchange Act of 1934, as amended. Forward-looking statements arestatements other than historical information or statements of current condition andrelate to future events or future financial performance. These forward-lookingstatements relate to plans, objectives, and expectations for future operations. In lightof risks and uncertainties, there can be no assurance that the forward-lookinginformation contained in this presentation will in fact transpire or prove to beaccurate. BioPharmX, Inc. undertakes no duty to update any forward-lookingstatement 1) to conform the statement to actual results, or 2) to reflect changes inthe expectations of BioPharmX, Inc.
All information in the following slide presentation is confidential and proprietary ofBioPharmX, Inc. By your receipt of this information, you agree to hold suchinformation confidential, unless required to disclose by law. All registration andtrademarks herein are the property of BioPharmX, Inc.
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Unique Drug Delivery Systems in Dermatology» Delivery to precise targets» Maximum efficacy» Fewer undesirable side effects» Applied to known APIs reduces regulatory time, cost and risk
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Phase 2 Product: BPX-01 (topical minocycline)» First non-lipophilic topical antibiotic formulation developed in-house
» Acne: Phase 2b results in Q217» Rosacea: conducting Phase 2 feasibility study
» Defined regulatory pathway via 505(b)2
Experienced Management Team» 28 employees» Commercialization & dermatology experience» Menlo Park, CA
Strong Institutional Support» Franklin Templeton Advisors» Vivo Capital
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4 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Anja Krammer | President20 years of commercialization experience; advisor for Fortune 100 companies & start-ups
Kin Chan, PhD | EVP Technology15 years experience in biomedical sciences, dermatology, wound healing, biophotonics, optical-thermal interaction in biological tissue, medical device
Greg Kitchener | CFO20 years experience in senior level finance and accounting roles
AnnaMarie Daniels | EVP Clinical and Regulatory Affairs30 years of clinical, regulatory & quality experience for a number of large pharmaceutical companies
Marie-Louise Bots | SVP Corporate Development30 years using licensing, partnerships and strategic acquisitions to boost the sales and profitability at several multinationals
BPX-Retinoids
BPX-Combinations
BPX-01-Acne
BPX-Rosacea
Research Preclinical Phase 2 Phase 3
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Injectable SystemBPX-02
Patents in Progress
Encapsulation System
1 Patent Issued, 2 Pending
CO
SM
ETIC
SA
ESTH
ETIC
S
Anhydrous Hydrophilic Topical
System4 Patents Pending
THER
AP
UET
ICS
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BioPharmX Unique Gel Delivery System
© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Allows higher penetration and targeted delivery at lower concentration
STABILIZES» Hydrophilic: non-occlusive, non-oily» Non-lipophilic (oil-based) » Suitable for APIs degraded in water
Anhydrous Hydrophilic Topical Delivery System
SOLUBILIZES» Improves bioavailability» Not a suspension» Miscible w/ sebum
Optimizing Efficacy
and Safety
Strong Efficacy
» Stabilizes & solubilizes minocycline» Delivered directly to sebaceous gland» Targeted penetration
Improved Safety Profile
» Low dose: 1% minocycline» Minimizes side effects» Low systemic exposure
» Rapidly absorbing» Non-staining» Non-oily
Positive Patient Experience
» Non-fluorescing» 100% patient satisfaction in clinical trials
Delivers leading antibiotic for acne, minocycline in unique topical gel formulation
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7 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Opportunity to DISRUPT moderate-to-severe acne market
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BPX-01: Confirmed Delivery of Minocycline to Site
© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
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» Shows penetration of minocycline (in pink)
» BPX-01 1% achieves penetration to sebaceous gland with minimal epidermal residue
» MALDI confirms spatial drug distribution of minocycline in sebaceous gland (target)
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» Provides evidence of rapid delivery of low concentration of minocycline to target
» Developed in partnership with a leading university
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0 1 2 3 4
Upt
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Time (hrs)
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Ex vivo Human Facial Skin Study
» BPX-01 1% has ~ 2-3 fold increased uptake compared to 1% lipophilic formulation
» BPX-01 1% showed similar uptake to 4% lipophilic formulation at 4 hours
4% BPX-011% BPX-014% Lipophilic1% Lipophilic
9 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Source: SSR Health 2016
2015 US Acne Market: $3.8B Revenue
“The holy grail in acne treatment is optimizing efficacy and tolerability.”
Dr. Ted Lain, Board certified dermatologist and BPX-01 investigator
2015 Global Acne Market Nears $10B» Moderate-to-severe acne market
bifurcated and highly fragmented:Orals » High efficacy» Systemic side effects (dizziness, nausea, diarrhea)
Topicals» Less effective» Non-systemic side effects (rashes, irritation, flaking)
» Significant physician and patient demand for innovation
» BioPharmX topical gel formulation of minocycline is opportunity to become nneeww ssttaannddaarrdd ooff ccaarree
Orals4 leading products:» Valeant – Solodyn» Almirall – Acticlate» Galderma – Oracea» Mayne –Doryx
Topicals6 leading products:» Galderma – Epiduo» Allergan – Aczone, Tazorac» Valeant – Ziana, Retin-A-Micro» Bayer – Finacea
$1.6B
$2.2B
* Source data on file
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11 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Primary Endpoint Achieved
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BPX-01: Phase 2a PP.. aaccnneess Reduction Study
© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
» BPX-01 1% topical minocycline gel vs. vehicle control
» 4-week, single-center US, randomized, double-blind study in 33 patients
» Inclusion criteria: patients 18 to 40; >10,000 colonies/cm2 of P. acnes
» First-in-human study limited to measuring reduction in P. acnes and safety due to 4 week duration
» Assessments at baseline, two, four and six weeks
Log10 mean change from
Baseline at 4 weeks
Log10 mean change from Baseline % at 4 weeks
BPX-01 Minocycline (n=17)
-1.04 -90.9%
Vehicle (n=7) -0.46 -65.3%
» No drug-related adverse events » No detectable minocycline in plasma» No cutaneous toxicity » 100% patient satisfactionR
ESU
LTS
STU
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DES
IGN
Secondary/Safety Endpoints
Demonstrated Improved Safety Profile» No drug-related adverse events reported
» No cases of cutaneous toxicity reported by investigators or patients at 2 and 4 weeks» No erythema, edema, peeling, hyperpigmentation
» 100% patient satisfaction with usability and tolerability» No burning, stinging, tightness, or itching
» No detectable levels of minocycline in plasma at 4 weeks
BPX-01: Phase 2a Safety Results
13 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
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Mean Mean Mean
Baseline BLOQ BLOQ BLOQ
Week 2 BLOQ BLOQ 496.25
Week 4 BLOQ BLOQ 421.58
BLOQ=Below Level of Quantification (≤50ng/mL)
» Randomized, double-blind, vehicle-controlled, dose-ranging study in 226 patients with moderate-to-severe acne
» 12-week study evaluating 3 arms: 1% BPX-01, 2% BPX-01, vehicle» Conducted at 15 U.S. sites» Inclusion criteria: patients ages 9 to 40, IGA* moderate-to-severe, 20-60 non-
nodular inflammatory lesions
14 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.* Investigator Global Assessment; based on scale of 0 (clear) to 4 (severe)
Primary Endpoint» Absolute mean
change in number of inflammatory lesions from baseline at week 12EN
DP
OIN
TSS
TUD
Y D
ESIG
NTI
MIN
G
Secondary Endpoint» Proportion of
subjects with at least a two-grade reduction in IGA* (to clear “0” or almost clear “1”)
Safety/Exploratory Endpoints» Reduction in non-
inflammatory and nodular acne lesions
» Cutaneous tolerance» Subject Global Assessment
(SGA)
BPX-01: Phase 2b Acne Vulgaris Study
» Topline results released May 2017» Comprehensive results provided June 1, 2017
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BPX-01: Phase 2b Topline Results
© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
BPX-01 2%(n=72)
BPX-01 1%(n=73)
Vehicle(n=74)
PRIMARYMean change in inflammatory lesions
- 15.4(p-value = 0.022)
- 15.5(p-value = 0.037)
- 11.3
SECONDARYProportion with 2 grade reduction in IGA and clear to almost clear
22.7% 16.0% 17.1%
• Strong primary endpoint results: • Statistically significant reduction in lesion count vs. vehicle at both 1% and 2%
• Clear numerical trend in IGA for 2% dose:• Informs design and powering for phase 3• Phase 2b not powered to demonstrate statistical significance on IGA
• No serious drug-related adverse events; safety consistent with results of phase 2a • Comprehensive data to be presented at June 1 event
RES
ULT
S
» Two randomized, double-blind, vehicle-controlled studies in patients with moderate-to-severe acne
» 12-week study evaluating either BPX-01 1% or 2% minocycline» Trial size TBD after comprehensive Phase 2b data; conducted at U.S. sites» Inclusion criteria: patients ages 9 to 40, IGA moderate-to-severe, 20-60 non-
nodular inflammatory lesions
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BPX-01: Planned Phase 3 Acne Vulgaris Program
© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Co-Primary Endpoint
» Absolute mean change in number of inflammatory lesions from baseline at week 12
» At least a two-point reduction in IGA to clear or almost clearEN
DP
OIN
TSS
TUD
Y D
ESIG
N
» Request end of phase 2 meeting with FDA: 2Q 17» Phase 3 expected to begin in 3Q 17
TIM
ING
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Rosacea Clinical Development Overview
© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Feasibility Study
» Open-label study: 20 patients with moderate-to-severe papulopustular rosacea» 12-week once-daily treatment with BPX-01 1% minocycline» Pending tolerability assessment at 4 weeks, initiate Phase 2/3» Assessment expected in Q1/Q2
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Planned Phase 2/3» Multi-center, 12-week, randomized, double-blind study in 200 subjects with
moderate-to-severe papulopustular rosacea» Inclusion criteria: patients ages >18, IGA of 3 or 4, minimum of 15-20
inflammatory lesions at baseline» Co-Primary Endpoint:
» Change in lesion count from baseline» Two-point improvement in IGA to clear or almost clear
» Timing» Phase 2/3 expected to begin in 3Q 17
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
RReennoowwnneedd mmeeddiiccaall aaddvviissoorryy bbooaarrdd lleeaaddiinngg ppaarraaddiiggmm sshhiifftt::» Ongoing stream of
presentations at leading dermatology conferences
» 9 presentations at AAD
EEssttaabblliisshheedd pprroodduucctt pprrooffiillee ssuuppppoorrtteedd bbyy cclliinniiccaall eevviiddeennccee» Positive safety &
efficacy data
» 15 peer-reviewed publications/posters
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
» AAD poster on BPX-01 acne development program
» Society of Toxicology poster on BPX-01 safety profile
» Rosacea feasibility assessment
» BPX-01 Phase 2b topline data
» Request end-of-phase 2 FDA meeting
» Initiation of BPX-01 Phase 3 trial
» DERM2017 Presentations
» BPX-Rosacea Phase 2/3 begins
» Advance one preclinical candidate into clinic (e.g. retinoids, combination)
Q3Q2Q1 Q4
Financial Information
20 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
1 On a pro-forma basis. Reported Fiscal Q316 cash of $0.5M plus $11.2M in net proceeds of secondary offering
CCaappiittaalliizzaattiioonn» 74.1M shares outstanding» 131M fully-diluted shares» 40% institutional investors - Franklin Templeton Advisors and Vivo Capital
CCaasshh» $6.5 million as of January 31, 2017» + $4.5M raised in April (not included above) in
offering to existing institutional investors» Sufficient cash to fund BPX-01 phase 2b
clinical trial & advance R&D pipeline
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» Experienced management team» Significant opportunity to disrupt standard of care
in the $3.8B acne market» Demonstrated therapeutic dose using 1% minocycline» Positive Phase 2a and Phase 2b study results; established
path toward commercialization» Women’s health asset, Violet, positioned for license
and/or divestiture» Novel delivery system with potential for targeting multiple
indications as well as incorporating additional APIs» Extensive R&D pipeline of other delivery platforms;
biologics & encapsulation
21 | © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
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23 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
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© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
Brand Name Active Pharmaceutical Ingredient(s) Manufacturer Sales ($mm)
Solodyn Minocycline Valeant $572Acticlate Doxycycline Hyclate Almirall 389Oracea Doxycycline Galderma 378
Doryx Doxycycline Actavis(now Mayne) 265
Zithromax Azithromycin Pfizer 15Total: $1,619
Branded prescription ORAL acne products. 2015 WAC market share:
Brand Name Active Pharmaceutical Ingredient(s) Manufacturer Sales ($mm)
Epiduo Adapalene / BPO Galderma $452Aczone Dapsone Allergan 369Tazorac Tazarotene Allergan 211Ziana Clindamycin / Tretinoin Valeant 197Retin-A-Micro Tretinion Valeant 157Finacea Azelaic Acid Bayer 148Acanya Clindamycin / BPO Valeant 129Onexton Clindamycin / BPO Valeant 123Benzaclin Clindamycin / BPO Valeant 101Other 294
Total: $2,181
Branded prescription TOPICAL acne products. 2015 WAC market share:
24.0%
23.3%16.4%
0.9%
35.3%Acticlate
Oracea
Doryx
Zithromax
Solodyn
20.7%
16.9%
9.7%9.0%
7.2%
6.8%
5.9%
5.6%
5.6%
13.5%EpiduoAczoneTazoracZianaRetin-A-MicroFinaceaAcanyaOnextonBenzaclinOther
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The US & EU5 Acne Market: 75 Million» Approximately 75M in US and EU5 Suffer from Acne
25 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
75M
Moderate to Severe: 15 Million» Approximately 20% of acne market suffers from moderate-to-severe acne
15M
Treated Population: 4.5 Million» Assuming only 30% of moderate-to-severe cases are being treated by a physician
4.5M
BPX-01 Market Share: 450K» Assuming a 10% market share capture
450K
Price / Patient / Year: $1,300 - $2,600» ~$500 per month US / ~$300 per month EU5 assuming 12 – 24 week treatment
regimen per patient per year
$600M – $1.2B BPX-01 US & EU5 Market Opportunity» Gross revenue
PATIENTS
26 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
» Past President, American Acne and Rosacea Society
» Medical Director of the Acne Cure Alliance
» Vice-Chair, Dept. of Derm., SUNY Downstate
Hilary Baldwin,MD Co-Chair
Neal Bhatia,MD Co-Chair
» Board of Directors, AAD 2012-2018
» Editorial Boards: Practical Dermatology; The Dermatologist; J Clinical and Aesthetic Dermatology
James Del Rosso,DO
» Past President, American Acne and Rosacea Society
» Past President, American Osteopathic College of Derm.
» Past President, American Society for Mohs Surgery
» Editor-in-Chief, J Clinical and Aesthetic Dermatology
» Founding President of the American Acne and Rosacea Society
» Editorial Board for Dermatology Online Journal
» Editorial Board for Clinics in Dermatology
Guy Webster,MD, PhD
» Founding Director & current President- Elect of the American Acne and Rosacea Society
» Currently serves on AAD’s Acne Work Group
» Past President, Alabama Dermatological Society
Julie Harper.MD
» Past President and Current Director, Women’s Dermatologic Society» Director, American Society for Dermatologic Surgery » Director, Medical Council of the Skin Cancer Foundation» Founder, American Society of Cosmetic Dermatology and Aesthetic Surgery » Founder, the American Acne and Rosacea Society
Diane S. Berson,MD
» Past President, American Acne and Rosacea Society
» Professor of Dermatology, Vice-Chair for Research for Dermatology, Hershey Medical Center
Diane Thiboutot,MD
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BPX-01: Rigorous, Robust Phase 2 Program
© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.
» Phase 2b powered to evaluate primary endpoint of lesion reduction
» Provides insight on IGA endpoint to inform phase 3 size and scope
» Experienced sites with dermatology specialists enable consistent IGA reporting
» Inclusion of multiple patient-reported outcomes measures:
» Cutaneous tolerance
» SGA of severity and improvement
» Evaluation of systemic exposure (plasma drug levels)
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Option B