SMARTER DRUG DELIVERY IN DERMATOLOGY - Company Overview March 2018 - BioPharmX
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SMARTER DRUG DELIVERY
IN DERMATOLOGY
Company Overview
March 2018
1| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
LEGAL
DISCLOSURES
Safe Harbor and Confidentiality
This presentation may include “forward-looking statements” within the meaning of the Private Securities
Litigation Reform Act of 1995. Forward-looking statements are statements, other than historical
information or statements of current condition, and relate to future events, financial performance,
operations, revenues, costs, expenses or capital requirements, strategies, goals, milestones, prospects,
beliefs, intentions, plans, expectations, forecasts or objectives. In light of risks and uncertainties, there
can be no assurance that the forward-looking information contained in this presentation will in fact
transpire or prove to be accurate. BioPharmX Corporation (the “Company”) anticipates that subsequent
events and developments will cause the Company’s views to change. However, the Company
undertakes no duty to update any forward-looking statement to conform this presentation to actual
results or to reflect changes in the expectations of the Company.
Actual results may differ materially from those indicated by such forward-looking statements as a result
of various important factors, including risks and uncertainties relating to our operating losses, our ability
to obtain additional funding, our limited operating history, our ability to successfully develop, obtain
regulatory approval and commercialize our product candidates, and other factors discussed in the “Risk
Factors” section of our most recent annual report on Form 10-K filed with the Securities and Exchange
Commission (the “SEC”), our most recent quarterly report on Form 10-Q filed with the SEC, and in the
Company’s other filings from time to time with the SEC.
All information in the following slide presentation is the confidential and proprietary information of the
Company. By your receipt of this information, you agree to hold such information confidential, unless
required to disclose by law. All registration and trademarks herein are the property of the Company.
2| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
BioPharmX Corporate Highlights Unique Drug Delivery Systems in Dermatology » Multiple platforms focused on delivery of API to precise targets optimizing safety and efficacy » Hydrophilic Topical Delivery System offers sustainable pipeline of products with known API, reduces regulatory timeline, cost and risk » Technology developed in-house BPX-01 Acne » Completed positive Phase 2b for acne vulgaris » FDA concurrence on endpoints achieved » Evaluating strategic partnerships to fund Phase 3 BPX-04 Rosacea » Established feasibility; planning for phase 2 » Confirmed tolerability in multiple doses Experienced Management Team » 28 employees » Commercialization & dermatology experience » Menlo Park, CA 3| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
BioPharmX Executive Team
Anja Krammer | President
20 years of commercialization experience; advisor for Fortune 100
companies & start-ups
Kin Chan, PhD | EVP Technology
15 years experience in biomedical sciences, dermatology, wound healing,
biophotonics, optical-thermal interaction in biological tissue, medical device
Greg Kitchener | CFO
20 years experience in senior level finance and accounting roles
AnnaMarie Daniels | EVP Clinical and Regulatory Affairs
30 years of clinical, regulatory & quality experience for a number of large
pharmaceutical companies
Marie-Louise Bots | SVP Corporate Development
30 years using licensing, partnerships and strategic acquisitions to boost
the sales and profitability at several multinationals
R. Kehl Sink | SVP Intellectual Property and Legal
15 years experience in medical device and pharmaceutical fields
4| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
BioPharmX Dermatology Pipeline
Research Preclinical Phase 1 Phase 2 Phase 3
BPX-01 Acne
THERAPEUTICS
Hydrophilic Topical
BPX-04 Rosacea
System
1 Patent Allowed
BPX-Retinoids
17 Pending
BPX-Combinations
AESTHETICS
BPX-02
Injectable System
Patents in Progress
COSMETICS
Encapsulation System
3 Patents Issued
5| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
Innovation - Hydrophilic Topical System
STABILIZES
» Hydrophilic: non-occlusive, non-oily
» Non-lipophilic (not oil-based)
THERAPEUTICS
Hydrophilic Topical » Suitable for APIs degraded in water
System +
FULLY SOLUBILIZES
» Improves bioavailability
» Not a suspension
» Miscible w/ sebum
Greater
penetration
and
Targeted
delivery
6| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
BPX-01:
Topical Minocycline
for the treatment of
Inflammatory Lesions of
Acne Vulgaris
7| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
Lead Product Candidate: BPX-01
Delivers leading antibiotic for acne, minocycline,
in unique topical gel formulation
» Low dose minocycline: advancing 2% dose
Improved
» Minimizes side effects
Safety Profile
» No detectable systemic exposure
» Stabilizes & solubilizes minocycline
Strong
» Delivered directly to sebaceous gland
Efficacy
» Targeted penetration
» Rapidly absorbing » Non-fluorescing
Positive Patient » Non-staining » No photosensitivity
Experience
» Non-oily » High patient satisfaction
Opportunity to DISRUPT moderate-to-severe acne market
8| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
Lack of Innovation in Antibiotics for Acne
Topical Antibiotic Development Since 1950…
Topical Minocycline
Erythromycin Clindamycin
(BPX-01)
1950s 1972
Est. 2020
No topical minocycline after 50 years…
Topical Minocycline
Oral Minocycline
(BPX-01)
1966
Est. 2020
9| © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.
Opportunity to Disrupt Global Acne Market*
2015 Global Acne Market Nears $10B 2015 US Branded Rx Acne Market:
$3.8B Revenue
» Moderate-to-severe acne market
bifurcated and highly fragmented:
Orals Orals
» High efficacy 4 leading products:
» Systemic side effects (dizziness, nausea, diarrhea) $1.6B » Valeant – Solodyn
» Almirall – Acticlate
» Galderma – Oracea
Topicals
» Mayne –Doryx
» Less effective
» Non-systemic, cutaneous side effects (rashes, irritation, flaking)
» Significant physician and patient demand for
Topicals
innovation 6 leading products:
» BioPharmX topical gel formulation of $2.2B » Galderma – Epiduo
» Allergan – Aczone, Tazorac
» Valeant – Ziana, Retin-A-Micro
minocycline is opportunity to become » Bayer – Finacea
new standard of care
Source: SSR Health 2015
“The holy grail in acne treatment is Dr. Ted Lain,
Board certified dermatologist
optimizing efficacy and tolerability.” and BPX-01 investigator
* Source data on file
10 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01 Product Positioning
Antibiotics
Isotretinoin Retinoids
Hormonal ORALS Benzoyl Peroxide
Treatment
Steroids
BPX-01 Antibiotics
TOPICALS Azelaic Acid
Salicylic & Other Acids
Dapsone
Combination Products
Effective, but with systemic side effects Less effective and not well tolerated
BPX-01: BOTH Efficacious & Well Tolerated
As dermatologists prefer polytherapy, BPX-01 is well positioned to displace or
co-exist with the above competitive landscape
11 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01: >$2B US Displacement Opportunity
Displacement targets include both topical & oral antibiotics
Leading acne therapies
$700
$624
$600 $572
$500
$452
$389
$400 $369
$300 $265
$200
$100
$-
BRANDED SOLODYN EPIDUO ACTICLATE ACZONE DORYX
CLINDAMYCIN
* Reflects branded market opportunity. Additional upside associated with generic displacement. Source: SSR Health
12 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01:
Clinical Development
13 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01: Phase 2b Acne Vulgaris Study
» Randomized, double-blind, vehicle-controlled, dose-ranging study
STUDY DESIGN
in 226 patients with moderate-to-severe acne
» 12-week study evaluating 3 arms: BPX-01 1% , BPX-01 2%, vehicle
» Conducted at 15 U.S. sites
» Patients ages 9 to 40, IGA of 3 or 4*, 20-60 non-nodular inflammatory lesions
Primary Endpoint Secondary Endpoint
ENDPOINTS
» Absolute mean change in number » Proportion of subjects with at least a
of inflammatory lesions from two-grade reduction in IGA* (to clear
baseline at week 12 “0” or almost clear “1”)
EXPLORATORY /
» Minocycline plasma concentrations
» Safety – adverse events
SAFETY
» Cutaneous tolerance
» Patient satisfaction
» Non-inflammatory (NIN) lesion reduction
* Investigator Global Assessment; based on scale of 0 (clear) to 4 (severe)
14 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01: Primary Endpoint Achieved in BPX-01 2%
BPX-01 1% BPX-01 2% Vehicle
Subjects per arm 73 72 74
Absolute mean
change in
inflammatory lesions
- 15.5 - 15.4 - 11.2
at week 12
p-value 0.0543 0.0352
Absolute mean change in Percent reduction in
inflammatory lesions inflammatory lesions
0 0%
-4 -20% D=
-8 -11.2 -40% -43.8% 15%
-12 -15.4 -58.5%
-16 -60%
-20 -80%
BPX-01 2% Vehicle BPX-01 2% Vehicle
» Statistically significant reduction in lesions at 2% dose (primary endpoint)
» Confidence in design of phase 3 reinforced by 15% separation between BPX-01 2% and vehicle
Reflects ITT population of 219 patients. Imputation for LOCF applied. Dunnett’s procedure of adjustment for multiple comparisons used
15 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01: Effective and Rapid Onset of Inflammatory Lesion Reduction
Percentage Reduction vs. Baseline
0.0%
-10.0%
potentially contributes to higher
compliance
-20.0%
-25%
-30.0%
-40.0%
16
-50.0% BPX-01 1% BPX-01 2% Vehicle
Baseline 0.0% 0.0% 0.0%
BPX-01
Week 2 1% BPX-01
-32.8% 2% Vehicle -25.7%
-27.6%
-60.0%
Baseline 0.0%
Week 4 0.0%
-39.7% 0.0%
-43.3% -25.3%
Week 2 -32.8%
Week 8 -27.6%
-50.5% -25.7% -38.7%
-49.5%
Week 4 -39.7%
Week 12 -43.3%
-54.4% -25.3% -43.8%
-58.5%
-70.0%
Week 8 -50.5% -49.5% -38.7%
Week 12
Baseline
-54.4% -58.5%
Week 4
-43.8%
Week 8 Week 12
BPX-01 2% BPX-01 1% Vehicle
16 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01: Secondary Endpoint - IGA
Clear numerical trend in IGA improvement seen in the 2% treatment arm
BPX-01 1% BPX-01 2% Vehicle
Subjects per arm 73 72 74
Proportion with ≥ two-
grade reduction and 20.5% 25.0% 17.6%
clear or almost clear D=
7.4%
p-value (vs vehicle) >0.9999 0.5445
30%
25%
20%
15%
10%
5%
0%
Baseline Week 2 Week 4 Week 8 Week 12
BPX-01 1% BPX-01 2% Vehicle
Reflects ITT analysis in 219 patients. Imputation for LOCF applied. P-value: Chi Square with Bonferroni adjustment for multiple comparisons used
17 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01: Safety Results from Phase 2b
BPX-01 Phase 2b Minocycline Plasma Concentrations (ng/mL)
BPX-01 2% BPX-01 1% Vehicle
Baseline BLOQ BLOQ BLOQ
Week 4 BLOQ BLOQ BLOQ
Week 12 BLOQ* BLOQ BLOQ
*One patient of BPX-01 2% measured 42 ng/ml but demonstrated no AE’s BLOQ=Below Limit of Quantification
BPX-01 Phase 2b Adverse Events
» Generally safe and well tolerated
» No serious drug-related adverse events
» Most common study-related AEs across all 3 arms include:
» Nasopharyngitis (2.7%)
» Upper respiratory tract infection (2.7%)
» Headache (1.8%)
» No reported photosensitivity or post inflammation hyperpigmentation (PIH)
» No reported staining and/or skin discoloration was observed
18 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-01 Phase 3 Program Readiness
Robust Phase 2
» Large study: 15 sites throughout the US
» Geographic disbursement
» Diverse demographics
» Designed to inform powering in P3
FDA Concurrence
» Endpoint design used in oral minocycline
» Reduction in inflammatory lesions only
» IGA scale specific to inflammatory lesions only
Finalized Phase 3 Study Design
» Powered appropriately
» Only sites with proven track record in successful dermatology studies
» Ongoing training and oversight
Leveraging an established regulatory pathway for antibiotics
19 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-04:
Topical Minocycline
for the Treatment of
Inflammatory Lesions of
Rosacea
20 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-04 Opportunity in Rosacea
» Rosacea Key Symptoms: facial ~ $1B US Opportunity*
redness with swollen red
bumps and small visible blood
vessels Estimated to affect up to
16 million in the US
» Skin sensitivity, physical discomfort, burning
sensation, facial itching, stinging and swelling 2015 US Rosacea Market - TRx
» Challenge: Skin with rosacea easily aggravated by too
Other
much drug or irritating vehicle
Mirvaso Oracea
(Galderma) (Galderma)
» Limited therapeutic options Soolantra
(Galderma)
à BPX-04 was designed to deliver the active, but not Finacea (Bayer)
further irritate the skin
“I’ve never had a topical for rosacea that Dr. Neal Bhatia,
was this tolerable and easy to apply, let alone Board certified dermatologist
and BPX-04-Rosacea investigator
pleasant and patient friendly”
• Source data on file
• There is some overlap between acne and rosacea due to off-label use
21 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-04 Rosacea: Open Label Feasibility Study
» 12-week, open-label, single-site study (Dr. Neal Bhatia; Therapeutics Clinical
Research, San Diego)
DESIGN
» 30 patients with moderate-to-severe papulopustular rosacea (10 at 1%; 10 at 2%;
10 at vehicle) patients aged 18 years and above
» Once-daily application to the face
Efficacy Endpoints: Safety & Tolerability Endpoints:
ENDPOINTS
» 2-grade reduction in IGA to clear » Investigator & patient assessed
or almost clear (0 or 1) cutaneous tolerability
» Change in lesion count from » Hematology & chemistry lab tests
baseline to 12 weeks » Treatment emergent AE’s
1/31/18 INTERIM
» Both 1% and 2% well tolerated
RESULTS
» BPX-04 1% and 2% aggregate (completed, n=19)
» 89% of subjects were clear or almost clear on IGA scale
» 90% mean reduction in lesions
22 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.Rosacea Open-label Feasibility Study – BPX-04 1%
Baseline Week 12
IGA = 4 IGA = 1
23 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.Rosacea Open-label Feasibility Study – BPX-04 2%
Baseline Week 12
IGA = 3 IGA = 0
24 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.BPX-04: Phase 2b Rosacea Study Protocol
» Randomized, double-blind, vehicle-controlled study in 176 subjects with
STUDY DESIGN
inflammatory lesions of rosacea
» 12-week study evaluating 2 arms: BPX-04 1% and vehicle
» Planned at 10-20 U.S. sites
» Subjects age ≥18, IGA of 3 or 4*, 15-70 inflammatory lesions on the face
» Optional interim analysis to allow sample size adjustment, if required
Primary Endpoint Secondary Endpoint
ENDPOINTS
» Absolute mean change in number » Proportion of subjects with at least a
of inflammatory lesions of rosacea two-grade reduction in IGA* (to clear “0”
from Baseline to Week 12 or almost clear “1”) from Baseline to
Week 12
EXPLORATORY /
» Minocycline plasma concentrations
» Safety – adverse events
SAFETY
» Cutaneous tolerance
» Patient satisfaction
» Change in erythema and telangiectasia
25 * Investigator Global Assessment; based on scale of 0 (clear) to 4 (severe) © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.2018 Milestones
Q1 18 Q2 18 2H 18
BPX-04 Rosacea Phase 2 Program » Target phase 3 & commercial » Complete phase 2
partnership BPX-01 for acne enrollment of BPX-04 for
» Submitted protocol to FDA rosacea
» Initiate phase 2 study of BPX-
» Selected CRO 04 for rosacea » Advance preclinical
candidate into clinic
» Identified study sites » Submit new product candidate
Data Presentations to FDA » Phase 3 program ready to
initiate for BPX-01 for acne
» Winter Clinical
» Maui Derm
Patents Granted
26 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.Financial Information
NYSE American: BPMX
Capitalization
» 190M shares outstanding*
» 376M fully-diluted shares
Cash
» Pro-forma cash of $14.2 million
» $7.6 million as of January 31, 2018
» $6.6 million in proceeds from warrant
exercises after the end of the quarter*
* As of March 9, 2018
27 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.Summary
» Experienced management team
» Significant opportunity to disrupt standard of care
in the $3.8B acne market
» Demonstrated therapeutic dose for acne using 2% minocycline
» Positive Phase 2b acne results: 58.5% reduction in lesions; clear
trend in IGA informs design for phase 3; good safety profile
» Confirmed feasibility in second indication for rosacea
» 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
» Women’s health asset, Violet, positioned for license
and/or divestiture
28 | © 2018 BIOPHARMX CORP. ALL RIGHTS RESERVED.You can also read
