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Home Phe Monitor Webinar
Thursday, June 25, 2020
1
__________________
We will start shortly
**Please select the mute option**
WELCOME!
Lex Cowsert, PhD, Director of Research Development, NPKUA
• Major challenge faced by PKU patients is monitoring Phe
levels
• First line of therapy is highly restricted diet
• New therapies result a new challenge of Phe levels too low
• NPKUA has made Home Phe Monitor a priority
2
• History of the Home Phe Project
• More development is needed
• Today we are pleased to introduce 3 companies that are
launching Home Phe Monitors
Today’s Presenters
1 - Michael Boyce-Jacino, PhD, CEO, Aptatek Biosciences
2 - Peter Warthoe, CSO, Egoo Health
3 - Shima Dalirirad, PhD, Scientist & Robert Harper,
President, CEO, In Vitro Diagnostic Solutions
3
Question & Answer Session – the chat will be available at the
end of the presentations only. Please notate your questions for
later. It would be helpful if there are specific questions to
presenters to notate a slide number. Thank you!
Breakthrough
Device
Breakthrough Lab-at-Home System
Michael Boyce-Jacino, PhD, CEO
4
The Team
Key Advisors:
John Adams
Canadian PKUA
Fran White
MDC Associates
Ali Tinazli, PhD
Fluxergy, HP Life
Sciences
Foad Dabiri, PhD
Mike Boyce-Jacino, PhD Tilla Worgall, MD, PhD Milan Stojanovic, PhD
Twitter, Wanda
CEO CMO, Founder CSO, Founder
• US Director for UK fund • Associate Professor of • Professor of Medical
• 4 Life Science startups Pathology and Cell Biology Science and Biomedical
• VP Genomics Beckman • Columbia University Engineering
• Two exits through sale Medical Center • Columbia University
• Two exits through IPO Medical Center
Aptatek BioSciences, Inc 5
5
Portable Monitoring
“Must-haves”
• Trustworthy Results
• Quantitative
• Same accuracy and precision as clinical
lab data
• Validated by peers and regulatory
authorities
• Easy to use
• Minimal finger stick blood
• Automated analysis (one-step)
• Connected
• Communicate to clinical team
• Data storage, trend analyses
Aptatek BioSciences, Inc 6
6
Phenylalanine is a Small Molecule
Small molecules (< 900 daltons)
Amino acids
Hormones
Antibiotics
Drugs
Environmental factors
Toxins
What makes analysis difficult ?
Phe
Too smooth - nothing sticks
Too small for antibodies to bind it well
Metabolites 2014, 4(4), 1007-1017
Aptatek BioSciences, Inc 7
7
Detection Strategies
Direct
• HPLC and Mass spectrometry limited to central
labs
Indirect
Enzymatic Assays
• Phenylalanine ammonium lyase - measure
ammonia
• Phenylalanine dehydrogenase - add nitroblue
tetrazolium (NBT) to NADH then measure color
Fluorescence Assay
• Incubate phe with ninhydrine and dipeptides -
measure fluorescence
Aptatek BioSciences, Inc 8
8
Aptamers
• Short pieces of oligonucleotides (DNA or RNA)
+
• Selected from very large libraries Target
• Bind a target with high sensitivity
• Can be manufactured in a standardized way
• Adaptable to color or electronic readouts
• Conceptually powerful sensors but tricky to Aptamer
make sensitive
Aptatek BioSciences, Inc 9
9
New Approach For Small Molecule Detection
+
Columbia DNA Target
Aptamer
‘Lid‘ [Quencher] Immediate Signal on Contact
Very high affinity ( = sticks very well )
Optimized for human matrix
Amino acids, steroids, drugs, hormones, sugars, neurotransmitters
Aptatek BioSciences, Inc 10
10Bland-Altman – Low Range
Patient Plasma Samples, range 50-100 μM, n = 70
Aptamer vs. Mass spec
150
150
Benchmark 100
D if f e r e n c e ( M )
50
100 28
D if f e r e n c e ( M )
0 5.0
50 -18
24
-5 0
0 0.48
-23 -1 0 0
-5 0
-1 5 0
-1 0 0 0 50 100 150
A v e r a g e ( ( M a s s s p e c + A p t a m e r ) /2 )
-1 5 0
150
0 50 100 150
A v e r a g e ( ( M a s s s p e c + H P L C ) /2 )
100
Aptamer vs. HPLC
D if f e r e n c e ( M )
50
32
4.5
Limits of Agreement Bias 0
-23
Bland-Altman Upper (95 %) Lower (95 %) -5 0
Mass spec / HPLC 24 -23 0.48 -1 0 0
Bland-Altman
Mass spec / Aptamer 28 -18 5.0 -1 5 0 Limits of
HPLC / Aptamer 32 -23 4.5 0 50 100 150 Agreement (95 %)
A v e r a g e ( ( H P L C + A p t a m e r ) /2 ) Bias
Aptatek BioSciences, Inc 11
11The Components
Reagent pellet
Cap and sample collector Blood collection
Buffer
Test cartridge and lateral flow
Dispense valve
Blood sample and reagents are added
to test chamber when cap is screwed back on
Aptatek BioSciences, Inc 12
12Aptatek at Home System
Reader Consumer wants
Battery powered Self management
Bluetooth At home ease of use
Smart home (Zigbee) capable Clinician desires
Test processing and data storage Real time data
Disposable test Digitized EHR support
Reagent cartridge Payors want
Lateral flow cassette Reduced cost per test
Smartphone app Fewer ED/Dr. visits
iOS and Android Improved compliance
Test management Pharma wants
Data reporting Clinical trial support
HIPAA complaint Marketing support
Compliance
Aptatek BioSciences, Inc 13
13Digital Backbone
Connectivity Compliance Communication
• Cell phone app • HIPAA compliant • Step-by-step guided test
• Links patient to system • FDA compliant • Graphic and numeric report
• Links patient to caregiver • System and test status • By last test, date or time
• Error/failure reporting • Exportable
Aptatek BioSciences, Inc 14
14Aptatek Platform Technology
Central lab performance in home use format
400
A p ta m e r
300
200
( M )
100
0
0 100 200 300 400
M a s s s p e c ( M P h e )
Patient plasma samples, Phenylalanine range 50-300 μM, n = 70
Performance equal to central lab gold
standard
Aptatek BioSciences, Inc 15
15Aptatek Home Health System
Patient Sample
Upload to dieticians and clinicians
Real Time Result
Actionable Data:
Medication management
Opportunities Aptatek
Appointment reminders Quality control
Prescription refill Smart home medical hub
Integration with other health data Reimbursement support
HIPAA compliant data storage Clinical trial support
Clinical care messaging Web portal for patient HIPAA compliant data storage
Wellness support management (Future) Cognitive testing integration
Aptatek BioSciences, Inc 16
16Current Development Status
Research Research Clinical Commercial
Feasibility
Prototype Validation Validation Development
Phenylalanine (Phenylketonuria)
Vancomycin
Tobramycin
Urea Inborn Errors of Metabolism
Creatinine Therapeutic Drug Monitoring
Leucine Dialysis Monitoring
Aptatek BioSciences, Inc 17
17Aptatek BioScieneces
• Aptatek is a spin out of Columbia University focused on adapting proprietary
aptamer chemistry to detection of small molecules at point of care or in the
home
• Early targets in metabolic disease
• Aptatek uses unique chemistry to allow centralized lab tests to be done in
home
• DNA aptamers provide sensitivity of mass spectrometry in portable, user-friendly format
• System enables capture of testing data
• Time of test
• Barcode of test
• Results of test
• Patient use of system
• System enables integration into home health hub
• Connect to google/Alexa type devices
Aptatek BioSciences, Inc 18
18Thank You!
Please contact:
Michael T. Boyce-Jacino, Ph.D., CEO
mbjacino@aptatek.com
609-510-7667
www.aptatek.com
Disclaimer: Neither this presentation nor anything in it shall form the basis of any contract or commitment. This presentation is not intended to be relied upon as advice to investors or potential investors and does not take into account the investment objectives, financial situation or
needs of any investor. All investors should consider such factors in consultation with a professional advisor of their choosing when deciding if an investment is appropriate. The Company has prepared this presentation based on information available to it, including information derived
from public sources that have not been independently verified. No representation or warranty, express or implied, is provided in relation to the fairness, accuracy, correctness, completeness or reliability of the information, opinions or conclusions expressed herein. This presentation
may contain forward-looking statements and projections and such statements and projections should not be considered a comprehensive representation of the Company’s performance. Forward-looking statements involve known and unknown risks, uncertainties and other important
factors that may cause the Company’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. All forward–looking statements attributable to the Company
or persons acting on its behalf apply only as of the date of this document,and are expressly qualified in their entirety by the cautionary statements in the preceding sentence. The Company undertakes no obligation to update or revise these forward–looking statements to reflect
events or circumstances that arise after the date made or to reflect the occurrence of unanticipated events. Any financial information included in this presentation is preliminary, unaudited and subject to revision. Inevitably, some assumptions will not materialize, and unanticipated
events and circumstances may affect the ultimate financial results. Projections are inherently subject to substantial and numerous uncertainties and to a wide variety of significant business, economic and competitive risks, and the assumptions underlying the projections may be
inaccurate in any material respect. Therefore, the actual results achieved may vary significantly from the forecasts, and the variations may be material.
Aptatek BioSciences, Inc 19
1920
WHO WE ARE AND WHAT DO WE DO
Founded in Denmark in 2018 – going public March 2 2020
Currently 21 employees
Product: Small medical device that can measure biomarkers at home
Follows requirements from the EU - CE mark
Same test as you get done now – just faster
Focus areas: PKU and Covid-19 (molecular and antibody test)
21THE EGOO HOME SYSTEM
1. Device
2. Capsule
3. App
4. Cloud
22MOVIE
TIME
23Take blood
BLOOD sample
OUR
TEST Run the test
PROCESS
WAIT 5 hours
24BEST CASE SCENARIO TIMELINE
Before Covid-19
25Egoo and Covid-19 test
1200 covid-19 test pr. week
26Testing of Egoo reagents Testing of Phe-spiked blood
Fluorescence Fluorescence
500,000
400,000
450,000
350,000
400,000
R² = 0.9839 R² = 0.9929
300,000 350,000
Fluorescence
Fluorescence
250,000 300,000
250,000
200,000
200,000
150,000
150,000
100,000
100,000
50,000 50,000
- -
0 500 1000 1500 2000 0 500 1000 1500 2000
Phe calibrator conc. (µM) Phe conc. (µM)
27Pre-study Comparison Rest of assay Feasibility
Research Development data for CE
studies study
Pre-study Assay data for
Research From tubes to capsules (7 Danish Comparison studies Feasibility study
Prof CE
Well-known patients) Reproducibility A number of
Transferring the 1) Analysis of dried
reactions broadly User ability: blood spots from Sensitivity patients get their
assay from tubes to
used within the Patients get UMCG in the Interference own device and
capsules and
field of monitoring capsules to try and Netherlands Stability capsules to test at
finalizing the capsule
PKU fill correctly 2) Analysis of home
ERNDIM‘s dried
Comparison: blood spots
• They also fill
blood cards for All dried blood spots
us to analyze are of known PHE
with Egoo conc.
• The hospital
receives the
patients routinely
28
blood samples to
be analyzedBEST CASE SCENARIO TIMELINE
After Covid-19 + at least one year
29IVDS PKU Now
Shima Dalirirad, PhD Scientist
Robert Harper, President, CEO
30
@ivd.solutions
NPKUA Webinar
6/25/2020Overview
• Technology used in the prototype (pages 3-5)
• Current status:
– Testing results with Phe Spike whole blood samples (pages 6-8)
– Regulatory issues: documentation, V&V, Pre- IDE, plans for clinical trial, etc. (pages 9-10)
– Timeline with milestones for regulatory clearance and commercialization (page 11)
– Estimate funding required to complete project and proposed sources & Target pricing (page 12)
– Manufacturing of the device for commercialization (page 13)
– Summary (page 14)
31PKU Now Test Strip
1. Spreading Layer
2. Primary Blood Separation
Membrane
3. Secondary Blood Separation
Membrane
4. Reagent Layer. Turns Blue
Intensity of color is proportional to
blood PHE concentration
32PKU Now Test Kit (unpacked)
PKU Now
Capillary Tubes vial (20 uL 20 per vial)
PKU Now
Test Strips 20 per vial
Capillary Stand
Patent pending PKU Now
Meter
(iXensor)
PKU Now
2 level of controls.
(5 and 14 mg/dL)
33Home Monitoring Testing
1. Unpack kit.
2. Turn on meter
3. Remove capillary tube
and place in holder.
4. Place PKU Now test
strip in meter
5. Wash hands
6. Finger-prick sample
into capillary tube
7. Bandage finger
8. Apply sample
9. Read PHE
concentration on
display (t≤5 min)
34Data PKU Now Accuracy & Precision 60
Determinations
20 μL (< 5 minute Read Time) 1-25 mg/dL
Median SD .48 % CV 2-7%
n=3
*Spiked blood samples
35
** AAA: Amino Acid AnalysisTyrosine Interference. Patent Pending
Technology Eliminates Tyrosine Interference
Samples = 1, 3 & 6 mg/dL PHE + 3 mg/dL TYR
36Stable, Synthetic, Non-Hazardous
Controls
CLIA Waived Designation:
• Point-of-care test designated for home or over-the-
counter use
5 mg/dL 14 mg/dL
• Low risk device
Rep 1 5.22 13.49
• Requirements: 5.26 14.24
Rep 2
– Simple to use
Rep 3 5.43 13.97
– Insignificant risk of erroneous results
• Fail-safe measures Ave: 5.30 13.90
• Risk controls Stdev: 0.11 0.38
• Alert mechanisms
%CV: 2.10 2.73
– Two levels of quality controls
37
*CLIA: Clinical Laboratory Improvement AmendmentsPre-Clinical Studies, 40 patients per Site
Jerry Vockley, MD, PhD Caroline Eggerding, MD
Division Head, Pediatric
Children’s Hospital of Neurology and Development
Pittsburgh Cooper University Health Care
IVDS Pre-clinical Evaluation
IRB Oversight Consultant
38Regulatory Team
Claudia Campbell-Matland MS Marcia Zucker, PhD
Specialist in Quality Specialist in Regulatory
Management Systems Affairs
39Projected timeline
Year 2 (2020) Year 3 (2021) Year 5 (2022)
• File pre-IDE • Pre-Clinical Evaluation • Market Launch
• QMS • Clinical trial
• FDA 510k submission
*We anticipate a faster timeline if strategic partnership obtained
40Manufacturing of device for commercialization
• Meter under negotiating for price point
• Test strips manufactured in house, IVDS
– Machine coating, slitting, laminating and assembly.
Target Pricing
Component Unit Price
Per-use Cost:
$5
Test strip
Base Unit: Meter $300
41Funding & Sources Seeking Licensing
Partners
Commercialization Plan: Seek grants and strategic partnerships
Current grant funding: Estimated funding required for project
• SBIR Phase I “A POC device for completion:
phenylalanine determination” $225,000 $150K: Clinical evaluation ($50K per site, 3
• SBIR Phase II application $2 million sites)
(Pending Review) $200K: design history file, risk management
system, CE Mark and 510K approval
$100K: marketing and dissemination of
product
42Summary
The PKU Now is
Simple to use
Accurate & precise in the range of 1-25 mg/dL Phe
Eliminates Tyrosine interference on Phenylalanine
Dehydrogenase
Designed to meet all NPKUA specifications
43Questions?
1
Please email additional questions to Lex Cowsert,
Director of Research Development at
lex.cowsert@npkua.orgTHANK YOU
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