Johns Hopkins Masters Project: Business Case Slides

1. BUSINESS PLAN
Presented by:
Shubhda Roy
PhiNeTiCS
Bringing Movement Back

2. PhiNeTiCSI. Executive Summary
Mission
Unmet Clinical Need
II. Products
Parkinson’s Overview
Patents
PKS1 antibody
LDT and IVD for quantitative determination of PKS1
PKS1 Compound
III. Market
Market Opportunity
Business Model
Evaluation of the Industry
Marketing and Sales Plan
Competitive Advantage
Marketing Communication Plan
IV. Production
Diagnostic Production Process Overview for PARK ELISA
Employees and Subcontractors
GMP/QSR Compliance
Regulatory and Clinical Strategy
V Risk Factors
VI The Financial Plan
Reimbursement
ASR Labeled PKSAAntibody
IVD-cleared PKS1 PARK Elisa Kit
Desired Financing
Exit Strategy
Table of Contents

3. PhiNeTiCSEXECUTIVE SUMMARY - MISSION
 Assist physicians in diagnosing/treating Parkinson’s disease
 More effective treatment option
 Partner with large research hospital to create an LDT
 Develop and commercialize an IVD

4. PhiNeTiCS
EXECUTIVE SUMMARY – UNMET CLINICAL NEED
 Diagnosis very complex, occurs very late in development
 Physical symptoms are the only sign of PD
 Early Prognosis will help improve patient’s life
 Treatment is very limited
 Drugs only mitigate the symptoms and do not cure them
 PKS1 protein biomarker has shown to have potential in aiding with
the diagnosis and treatment of PD

5. PhiNeTiCSPRODUCTS – PARKINSON'S OVERVIEW
 Progressive neurodegenerative disorder that affects mostly
people over the age of 50
 Affects dopamine pathway
 1 Million Americans effected with Parkinson, with 60,000 new
cases diagnosed every year, 10 million worldwide living with it
 Primary motor vs. secondary motor vs. non-motor symptoms
 Treatment options
 Levodopa/cabidopa, MAO-B inhibitors, deep brain stimulation

6. PhiNeTiCS
PRODUCTS - PATENTS
 United States Patent Application 8.675,309
 PKS1 biomarker
 Antibody composition that is immuno specific for PKS1 and
interacts/modulates expression of protein
 Intellectual property rights encourage collaboration and partnerships
to further advance developments

7. PhiNeTiCS
PRODUCTS – PKS1 ANTIBODY AND ELISA
 The invention provides an antibody composition that is immuno-
specific for PKS1 and interacts with or modulates the expression of
PKS1
•PKS1 levels can be quantitatively measured as an LDT or IVD
using an ELISA assay with antibodies specific to PKS1.
PKS1AB
Enzyme-conjugated
polyclonal secondary
antibody to amplify the signal
(99.9% clinical sensitivity)
PKS1 protein

8. PhiNeTiCS
PRODUCTS – PKS1 PROTEIN
 PKS1 protein has been identified as a key protein biomarker for
Parkinson’s disease, and elevated levels have been correlated with the
development and onset of Parkinson’s disease
 The level of PKS1 in Parkinson’s disease patients was on average 50
times the normal concentration (MALDI-MS)
 Patients who currently receive 1st line therapy dopamine
replacement are still eligible to be monitored by PKS1
Patient type Response to treatment Concentration of PKS1
(relative unites)
Parkinson (-) NA 10 +/- 5
Parkinson (+)
treatment (-)
500 +/- 100
Parkinson (+)
Treatment (+)
Responding to
treatment Sinemet
40 +/- 15
Parkinson (+)
Treatment (+)
No response to
treatment with
Sinemet
450 +/- 200

9. PhiNeTiCSMARKET – MARKET OPPORTUNITY
 Parkinson’s caused due
1. Age – greater than age 50
2. Genetic factor – genes - PINK1, LRRK2 and DJ-1
3. Environmental – Toxins like
 – MPTP, CO, metals, chemicals, pesticides, herbicides etc.
 Studies & focus always been on age or genetic factor
 Toxins in environment an untapped market.
 Target Potential for Parkinson’s will be
1. Anyone above the age of 50 in the USA
 People showing symptoms or having family history
2. Parkinson’s Belt – Midwest, Northeast USA
 People worked in an industry with higher exposure to toxins for at least two years, above age
50
 People worked in an industry with higher exposure to toxins for at least two years, below age
50, showing symptoms & family history
3. Industries like agricultural, mining, metallurgy, or manufacturing with
higher exposure to toxins

10. PhiNeTiCS
PhINeTi
C
FDA
510(k)
Denovo
Class II
Device
IVD
MARKET - BUSINESS MODEL
PhINeTiCs Mayo Cleveland LDT
PhINeTiC
Maine
Biotech
Service
Manufacture
IVD
PhINeTiC Labcorp
Commercialization in
USA & Beyond

11. PhiNeTiCS
MARKET – COMPETITIVE LANDSCAPE
 PKS1 Biomarker increase cause onset of Parkinson
 Currently no quick way to diagnose Parkinson
 Diagnosis – complex, lengthy & not 100% accurate
 Competition may create generics in the (future)
 No-one has partnered with top two research clinics for LDT (Mayo &
Cleveland)
 Credibility, Validation more widespread
 Only Diagnostic IVD kit (now)

12. PhiNeTiCS
MARKET – EVALUATION OF THE INDUSTRY
 1 Million in the USA
 60,000 detected every year
 Cost (direct & indirect) in USA $23 Billion annually
 Early detection helpful in slowing progression of disease
 Quality of Life- Healthier, Stress free, save money

13. PhiNeTiCS
MARKETING – MARKETING AND SALES PLAN
 Mayo and Cleveland clinics
 Will have exclusive rights to LDT diagnostic test
 Many locations to speed up validation process
 Once IVD is available, need to only sell IVD
 LabCorp
 Help globalize/commercialize diagnostic test
 Class II using 510(k) de novo approval
 Create own sales and marketing teams to help with distribution and
marketing

14. PhiNeTiCS
MARKET – COMPETITIVE ADVANTAGE
 First IVD diagnostic kit for Parkinson’s disease
 Sole possession of the patent gives us freedom to begin developing
therapeutic treatments (future) in conjunction with IVD Diagnostic Kit
(current)
 GREAT COMPETITIVE ADVANTAGE!!

15. PhiNeTiCS
MARKET – MARKETING COMMUNICATION PLAN
 Brochures and pamphlets
 Utilize LabCorp to distribute
 Walk-ins, physician offices, senior housing, drug stores, minute
clinics
 Florida, West Virginia, Maine and Pennsylvania (highest % of
seniors over 65 years old)
 Create relationship with industrial companies
 Mining, painting, agriculture, metallurgy, construction
 Exposed to toxins that are known to be a factor in onset and
development of PD
 Annual screening test in these industries for everyone above 50
 Attend conventions and conferences

16. PhiNeTiCS
PRODUCTION – DIAGNOSTIC PRODUCTION PROCESS OVERVIEW
 PKS1 monoclonal antibodies produced using transgenic rabbit
technology.
 growth of "fully" human antibodies, without side effects
 Hybridoma technology
 Myeloma cells fused with cells of a rabbit immunized with the desired
antigen.
 Harvested for PKS1 monoclonal antibodies
 PKS1 monoclonal antibodies scaled up using single-use Bioreactor.
 prevents cleaning or sterilization
 preventing cross contamination
 improving biological and process safety
 Number of parts in bioreactor is few
 making validation for large scale production easier and cuts costs
by 60%.

17. PhiNeTiCS
PRODUCTION – EMPLOYEES AND SUBCONTRACTORS
 Executive steering committee to manage all compliance, quality issues,
regulations and relationships with the contractors
 Written contract signed with all contractors establish
 good communication,
 expectations for acceptable service,
 Monitoring
 FDA Audits, reporting non-conformance,performance matrix,
 compliance with (GMP/GDP/GCP)
 Clinical Reseach Org
 Mayo, Cleveland Clinic
 Contract Manufacturing Org
 Maine Biotechnology Services
 Clinical Packaging and Distribution
 Diamond Kitting Solutions Kit Packaging Products
 Commercialization
 Labscorp

18. PhiNeTiCS
PRODUCTION– GMP/QSR COMPLIANCE
 GMP and QSR regulations will be followed as outlined in 21 CFR 820
 Electronic documentation
 Electronic documentation will be kept in an electronic document
management system (EDMS)
 Strict document version control
 Author, review, approval
 Paper documentation
 Kept onsite for two years
 Shipped off site for remainder of the product’s life

19. PhiNeTiCS
PRODUCTION– GMP/QSR COMPLIANCE
 GMP and QSR regulations will be followed as outlined in 21 CFR 820
 Design controls
 Purchasing control
 Identification and traceability
 Production and process controls
 Acceptance activities
 Labeling
 Handling
 Storage
 Accountability
 Audits will be performed prior to contractual agreement, and
periodically throughout the partnership to ensure the partner
company is in compliance with thee regulations and that a consistent
quality product is being produced and distributed.

20. PhiNeTiCS
PRODUCTION – REGULATORY AND CLINICAL STRATEGY
 Partner with Mayo and Cleveland Clinics
 Prominent location for Parkinson’s Disease
 Develop LDT
 Clinically validate LDT
 Partner with LabCorp
 Large corporations with a global reach
 Commercialize diagnostic test
 Submit de novo 510(k)
 90 day review
 Class II Device
 Not making treatment decisions

21. PhiNeTiCS
PRODUCTION – REGULATORY AND CLINICAL STRATEGY
 Potential Life Cycle Management Opportunity
 Partner with a pharmaceutical company
 Drug development targeting PKS1
 Conduct adequate and well controlled clinical trials utilizing the LDT
 Clinical use of the LDT will be conducted under an Investigational
Device Exemption (IDE)
 Submit a PMA for approval of the companion diagnostic
 180 day review
 Typically approved in conjunction with NDA approval

22. PhiNeTiCS
RISK FACTORS
 Our small organization is built around a single patent that is our only
revenue stream
 Uncontrollable regulatory, data, and manufacturing delays can lead
to significant financial problems
 Our largest risk concerns intellectual property
 Our patent includes PKS1 derivatives and analysis techniques
 Validation and publication can lead to the discovery of similar
diagnostic biomarkers

23. PhiNeTiCS
RISK FACTORS
 We are completely reliant on our device manufacturer to produce the
product
 PhiNetics does not have control over manufacturing conditions
and adherence to cGMP
 We do not have the resources for a long drawn- out patent or
regulatory dispute
 Can be from a competing firm, the FDA/ judicial system, or any
contractual disputes with our partners

24. PhiNeTiCS
THE FINANCIAL PLAN - REIMBURSEMENT
 PhINeTiCs selling diagnostic kits for population aged above 50
 Health Insurance either directly or via an employer will be the main
source of reimbursement
 But Medicare, Medicaid definitely plays a role
 Affordable Care Act (ACA) – Every uninsured can get coverage
under preventative care
 Any policy changes, will have major impact on us
 Relationship companies, payers and work
 Provide annual screening for the Parkinson’s Diagnostic
 at an affordable price to all above the age of 50
 as part of base model in all levels of the insurance plan.

25. PhiNeTiCSTHE FINANCIAL PLAN – ASR LABELED PKS1 ANTIBODY
 Cleveland and Mayo Clinic
 PKSA Antibody as analyst specific reagent (ASR)
 sold at $400 per 0.1mg, which is sufficient for 150 tests.
 Develop LDT with limited use license until IVD commercially
available
 Academic Research Laboratories
 ASR for research purposes
 No FDA approval for ASR for research use
 Free
 Allows ASR to be used by all research labs at least once.
 Makes it more validated and known just by increased usage

26. PhiNeTiCS
The Financial plan - LDT (PARK ELISA Kit)
 The LDT test will be performed by Cleveland and Mayo clinic and reimbursed
at 30$ (CPT Code: 83520/Sensory Neuropathy Complete Antibody Panel)
with 1-10% of the total Parkinson’s Disease market reached over 5 years of
and with 15% royalty coming back to the company
Year1
US Overall
cases of PD2
Number of
patients
screened for
PD3
Predicted
market
penetration
CPT cost
of test
Royalty
(15% of test
revenue)
Predicted
Yearly profit
2015 1,000,000 1,000,000 2% $30.00 $4.50 $90,000.00
2016 1100000 1400000 4% $30.00 $4.50 $252,000.00
2017 1210000 1540000 6% $30.00 $4.50 $415,800.00
2018 1331000 1694000 8% $30.00 $4.50 $609,840.00
2019 1464100 1863400 10% $30.00 $4.50 $838,530.00
1Based on 5 years to develop IVD
2Assume 10% rate of increase of PD prevalence per year
3Assume 3x(increase in prevalence year over year) for screening +1x prevalence for prognosis

27. PhiNeTiCS
The Financial plan – IVD (PARK ELISA Kit)
 Currently there is no IVD available as a diagnosis or prognosis tool.
 Project to achieve 95% market penetration rate in 3 years following
marketing clearance of our IVD in 2020
1Based on 5 years to develop IVD
2Assume 10% rate of increase of PD prevalence per year
3Assume 3x(increase in prevalence year over year) for screening +1x prevalence for prognosis
Year1
US Overall
cases of PD2
Number of
patients
screened for
PD3
Predicted
market
penetration
CPT cost
of test
70% of
CPT code
MARGIN
(30% of test
revenue)
Predicted
Yearly profit
2020 1,610,510 2049740 30% $30.00 $21.00 $6.30 $3,874,009
2021 1,771,561 2254714 60% $30.00 $21.00 $6.30 $8,522,819
2022 1,948,717 2480185 95% $30.00 $21.00 $6.30 $14,843,910
2023 2,143,589 2728204 95% $30.00 $21.00 $6.30 $16,328,301

28. PhiNeTiCSThe Financial plan –
Projected profit for LDT to IVD commercialization
LDT IVD

29. PhiNeTiCS
 We are anticipating a large response to the initial fundraising
attempts due to the unique nature of the biomarker, device, and
antibody.
 There have been very few breakthroughs in Parkinson’s
pharmaceutical therapy in recent years
 We intend to raise 26 million in angel funding
 Lack of de novo 510(k) fees and significant partnerships lessen
the start-up capital needed
FINANCIAL PLAN – DESIRED FINANCING

30. PhiNeTiCS
FINANCIAL PLAN – EXIT STRATEGY
 Larger pharmaceutical company acquiring PhINeTiCs
 Enable them to perform more research on biomarker
 Develop other IVD, LDT or therapeutic treatment