smarter drug delivery in dermatology

27
S SM MA AR RT TE ER R D DR RU UG G D DE EL LI IV VE ER RY Y I IN N D DE ER RM MA AT TO OL LO OG GY Y 1 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED. M Ma ay y 2 20 01 17 7 C Co om mp pa an ny y O Ov ve er rv vi ie ew w

Upload: others

Post on 24-May-2022

0 views

Category:

Documents


0 download

TRANSCRIPT

Page 1: SMARTER DRUG DELIVERY IN DERMATOLOGY

SSMMAARRTTEERR DDRRUUGG DDEELLIIVVEERRYY IINN DDEERRMMAATTOOLLOOGGYY

1 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.

MMaayy 22001177

CCoommppaannyy OOvveerrvviieeww

Page 2: SMARTER DRUG DELIVERY IN DERMATOLOGY

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.

2

LLEEGGAALL DDIISSCCLLOOSSUURREESS

SSaaffee HHaarrbboorr aanndd CCoonnffiiddeennttiiaalliittyy

© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.

Page 3: SMARTER DRUG DELIVERY IN DERMATOLOGY

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

3

BBiiooPPhhaarrmmXX CCoorrppoorraattee HHiigghhlliigghhttss

© 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

Page 4: SMARTER DRUG DELIVERY IN DERMATOLOGY

BBiiooPPhhaarrmmXX EExxeeccuuttiivvee TTeeaamm

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

Page 5: SMARTER DRUG DELIVERY IN DERMATOLOGY

BPX-Retinoids

BPX-Combinations

BPX-01-Acne

BPX-Rosacea

Research Preclinical Phase 2 Phase 3

5

DDrruugg DDeelliivveerryy PPiippeelliinnee iinn DDeerrmmaattoollooggyy

© 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

Page 6: SMARTER DRUG DELIVERY IN DERMATOLOGY

6

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

Page 7: SMARTER DRUG DELIVERY IN DERMATOLOGY

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

LLeeaadd PPrroodduucctt CCaannddiiddaattee:: BBPPXX--0011 iiss DDiiffffeerreennttiiaatteedd

7 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.

Opportunity to DISRUPT moderate-to-severe acne market

Page 8: SMARTER DRUG DELIVERY IN DERMATOLOGY

UUnnttrreeaatteedd CCoonnttrrooll

8

BPX-01: Confirmed Delivery of Minocycline to Site

© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.

FFlluuoorreesscceennccee MMiiccrroossccooppyy

» 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)

TTwwoo--PPhhoottoonn FFlluuoorreesscceennccee

» Provides evidence of rapid delivery of low concentration of minocycline to target

» Developed in partnership with a leading university

MMuullttiippllee IImmaaggiinngg TTeecchhnniiqquueess DDeemmoonnssttrraattee PPeenneettrraattiioonn

FFlluuoorreesscceennccee MMiiccrroossccooppyy

BBPPXX--0011 ((11%%))

BBPPXX--0011 ((44%%))

Page 9: SMARTER DRUG DELIVERY IN DERMATOLOGY

0.0

2.0

4.0

6.0

8.0

10.0

12.0

14.0

16.0

18.0

0 1 2 3 4

Upt

ake

(μg/

cm2)

Time (hrs)

MMuullttiippllee ddoosseess ooff BBPPXX--0011 sshhooww qquuiicckkeerr ppeenneettrraattiioonn aanndd ssuussttaaiinneedd eeffffeecctt::

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.

BBPPXX--0011 CCoommppaarreedd ttoo LLiippoopphhiilliicc FFoorrmmuullaattiioonn

Page 10: SMARTER DRUG DELIVERY IN DERMATOLOGY

10

OOppppoorrttuunniittyy ttoo DDiissrruupptt GGlloobbaall AAccnnee MMaarrkkeett**

© 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

Page 11: SMARTER DRUG DELIVERY IN DERMATOLOGY

BBPPXX--0011 CCLLIINNIICCAALL DDEEVVEELLOOPPMMEENNTT

11 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.

Page 12: SMARTER DRUG DELIVERY IN DERMATOLOGY

Primary Endpoint Achieved

12

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

DY

DES

IGN

Secondary/Safety Endpoints

Page 13: SMARTER DRUG DELIVERY IN DERMATOLOGY

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.

MMiinnooccyycclliinnee PPllaassmmaa CCoonncceennttrraattiioonnss ((nngg//mmLL))SSttuuddyy 11 –– PPhhaassee 22aa SSttuuddyy 22

OOrraall MMiinnooccyycclliinneeBBPPXX--0011 11%% VVeehhiiccllee

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)

Page 14: SMARTER DRUG DELIVERY IN DERMATOLOGY

» 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

Page 15: SMARTER DRUG DELIVERY IN DERMATOLOGY

15

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

Page 16: SMARTER DRUG DELIVERY IN DERMATOLOGY

» 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

16

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

Page 17: SMARTER DRUG DELIVERY IN DERMATOLOGY

17

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

PLA

NN

ED P

HA

SE

2/3

FEA

SB

ILIT

Y

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

Page 18: SMARTER DRUG DELIVERY IN DERMATOLOGY

18

MMaarrkkeett DDeevveellooppmmeenntt:: CChhaannggiinngg tthhee TTrreeaattmmeenntt PPaarraaddiiggmm

© 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

SSttrroonngg nneettwwoorrkk ooff KKOOLLss//iinnvveessttiiggaattoorrss eennggaaggeedd iinn ppeeeerr--ttoo--ppeeeerr pprrooggrraammss

Page 19: SMARTER DRUG DELIVERY IN DERMATOLOGY

19

22001177 AAnnttiicciippaatteedd MMiilleessttoonneess

© 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

Page 20: SMARTER DRUG DELIVERY IN DERMATOLOGY

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

NNYYSSEE MMKKTT:: BBPPMMXX

Page 21: SMARTER DRUG DELIVERY IN DERMATOLOGY

SSuummmmaarryy

» 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.

Page 22: SMARTER DRUG DELIVERY IN DERMATOLOGY

TTHHAANNKK YYOOUU

© 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.

Page 23: SMARTER DRUG DELIVERY IN DERMATOLOGY

AAPPPPEENNDDIIXX

23 © 2017 BIOPHARMX, INC. ALL RIGHTS RESERVED.

Page 24: SMARTER DRUG DELIVERY IN DERMATOLOGY

24

22001155 UUSS AAccnnee MMaarrkkeett:: $$33..88BB RReevveennuuee

© 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

Page 25: SMARTER DRUG DELIVERY IN DERMATOLOGY

BBPPXX--0011:: >>$$11BB AAnnnnuuaall MMaarrkkeett OOppppoorrttuunniittyy ((UUSS && EEUU55))

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

Page 26: SMARTER DRUG DELIVERY IN DERMATOLOGY

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

PPrroommiinneenntt MMeeddiiccaall AAddvviissoorryy BBooaarrdd

Page 27: SMARTER DRUG DELIVERY IN DERMATOLOGY

27

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)

MMuullttiippllee pphhaassee 22 ssttuuddiieess ddeessiiggnneedd ttoo mmiittiiggaattee pphhaassee 33 rriisskk

Option B