Netia Medical Technologies Prospective Overview
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Transcript of Netia Medical Technologies Prospective Overview
netiamedicaltechnologies
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Our missionis to provide drug development
companies worldwidewith the technology to create
more expansive,much less expensive
pharmaceuticals.
Netia Medical Technologies
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Snapshot: RNA interferenceRNAi suppresses the expression of proteins that cause disease.
1990 - 1st documented in purple petunias
Attempt to make intensely purple RNAi triggered; all purple suppressed = petals white
2006 - Nobel Prize: discovery of “trigger” that activates RNAi
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Netia RNAi Drug Development Platform
Cuts cost of getting new drug to market by >50%
Saves time to market by 3 years Expands ability to treat medical disorders
Much Better Drugs at a Much Lower Cost
TheCriticalIndustryNeed
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65 Year Decline in Pharma R&D Efficiency
Increasing cost/market approval: Unsustainable
Scannell et al., Diagnosing the decline in pharmaceutical R&D efficiency. Nature Rev Drug Discovery 11: 191-200, 2012
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R&D for 1 Marketed Therapeutic: $1B-$21B
High cost of inefficiency: Unsustainable
Tollman et al., Identifying R&D outliers. Nature Rev Drug Discovery 10: 653-4, 2011
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Limits of Main Drug Type =R&D Cost Crisis
Small Molecule Drugs: • Don’t work with most targets of therapeutic
interest• Can’t be rationally designed• Massive screening required = poor
predictability• Manufacturing & behavior in body unique to
each• Determine likelihood of clinical failure late
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High Drug Prices Pressure Big Pharma
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Pharma’s Recognized Solutiona Drug Development Platform that Targets RNA
rather than proteins = more expansive & much less expensive
The Netia Platformremoves the limitations
and blends the best features of both
have been
developed2
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1st RNA-targeting Platform Antisense Oligo Drugs
1992: 1st systemically given to patients -- invented by Dr. Larry J. Smith
2012: 1st clinical successes, industry acceptance
2016: Limitation: Active only in liver & CNS
2016: Best Feature: Pervasive uptake
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Leading Antisense Oligo Drug Company
FirstClinical Successes
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2nd RNA-targeting Platform Double-strand RNAi Drugs
2004: 1st clinical trials, but poor carrier/cellular uptake
2012: 1st impressive trial results with new carrier
2016: Limitation: Uptake in liver only
2016: Best Feature: Active inside all cells
siRNA
target degraded
RNAimechanism
Leading Double-strand RNAi Drug Company
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$750MInupfrontfees
FirstClinical Successes
Remains LimitedtoLiver
MeetingtheCriticalIndustryNeed
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The Netia Platform Sequential RNAi (seqRNAi) Drugs
seqRNAidrug pair
guide strand
active RNAi mechanism
siRNA
RNA target degraded
RNAimechanism engagedRNA target
Single strands given sequentially; no carrier neededStrands combine in cells; form double-strand RNAi drug
Drug activates RNAi mechanism
RNA molecule that promotes disease destroyed
Unsustainable R&D Costs Overcome
Engage nearly all targets of therapeutic interest
Can be quickly & rationally designed
Manufacturing & behavior in body same for each
Results in animals more predictive of human outcomes
Determine likelihood of clinical failure early17
Netia seqRNAi Drugs:
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Netia Poised to Enter Productivity Plateau of Transformative Technology ‘Hype Cycle’
FinancialModeling &BusinessStructure
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Eli Lilly Dynamic Financial Modelof Small Molecule Drug Development
Overall success rate = 4%
Paul et al., How to improve R&D productivity: Pharmaceutical industry’s grand challenge. Nature Rev Drug Discovery 9: 203-14, 2010.
Netia: Major Advantages at Every Stagewith seqRNAi Drug Development
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ROI Streams
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CRO
Netia: Early Target Validation Savingsfor small Molecule Drug Developers
CRO charges $135M (50% gross profit). Net client savings: $945M
Development Stage
Cost(capitalized)
Cumulative SavingsWithout Early Target
Validation(cumulative)
With Early Target Validation(cumulative)
Preclinical $824M $183M $641M
Phase I $1.10B $256M $852M
Phase II $1.42B $391M $1.029B
Phase III $1.73B $650M $1.08B
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Netia Biopharma Subsidiaries for Acquisition
B I O
CRO develops RNAi drugs in-houseTransfers rights to subsidiaries for sale
High ROIs for drugs developed to any stage
Focus: Disorders for Phase II market approvalOption: Partnering
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Venture-backed Biopharma Acquisitions2005-2012
Giniatullina et al., Building for big pharma. Nature Biotech 31: 284-287, 2013.
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Estimated ROI: seqRNAi Drugs Sold by Development Stage
B I O
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Key PersonnelLarry Smith, Ph.D.
President, Founder, Inventor of Netia technology Previous Founder & CEO Hesed Biomed & Eleos Inc.
Samuel Benchimol, Ph.D. Head, lab working with Netia RNAi drugs Chair, Dept. of Biology, York University, Toronto Prior Head Cell & Molecular Biology, U of Toronto, Ontario Cancer
Institute
Randall Gatz, Ph.D. Former: New Opportunities Business Development Dir, Debiopharm,
Lausanne VP of R&D Fournier Laboratories, Dijon, France PI, US National Cancer Institute’s Drug Development Program