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C O N F I D E N T I A L | www.azurrx.com CORPORATE PRESENTATION (NASDAQ:AZRX) January 2018
Transcript
Page 1: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

C O N F I D E N T I A L | www.azurrx.com

CORPORATEPRESENTATION

(NASDAQ:AZRX)

January2018

Page 2: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Certainstatementsinthispresentationconstitute“forward-lookingstatements”withinthemeaningofSection21EoftheSecuritiesExchangeActof1934,asamended.Anystatementsthatrefertoexpectationsorothercharacterizationsoffutureevents,circumstancesorresultsareforward-lookingstatements.Suchforward-lookingstatementsincludeprojections.SuchprojectionswerenotpreparedinaccordancewithpublicguidelinesoftheAmericanInstituteofCertifiedPublicAccountantsregardingprojectionsandforecasts,norhavesuchprojectionsbeenaudited,examinedorotherwisereviewedbyindependentauditorsofthecompany.Suchforward-lookingstatementsinvolveknownandunknownrisks,uncertaintiesandotherfactorswhichmaycausetheactualresults,performanceorachievementsofthecompanytobemateriallydifferentfromanyfutureresults,performanceorachievementsexpressedorimpliedbysuchforward-lookingstatements.

Theviewsexpressedarethoseofmanagementandarebasedoncurrentlyavailableinformation.Estimatesandprojectionscontainedhereinhavebeenpreparedbymanagementandinvolvesignificantelementsofsubjectivejudgmentandanalysisandarebasedoncertainassumptions.Norepresentationnorwarranty,expressedorimplied,ismadeastotheaccuracyorcompletenessoftheinformationcontainedinthisdocument,andnothingcontainedhereinis,orshallbereliedupon,asapromiseorrepresentation,whetherastothepastorthefuture.Theprojectionsarenotintendedtofollowgenerallyacceptedaccountingprinciples.Neitherouraccountantsnorourlegalcounselhavecompiled,audited,prepared,orcontributedtotheprojectionsortheunderlyingassumptions.Noneofthesepartiesexpressanopinionwithrespecttotheprojections.

Youarecautionednottoplaceunduerelianceontheseforward-lookingstatements.Exceptforongoingobligationsofthecompanytodisclosematerialinformationunderthefederalsecuritieslaws,thecompanydoesnotundertakeanyobligationtoreleaseanyrevisionstoanyforward-lookingstatements,toreporteventsortoreporttheoccurrenceofunanticipatedevents.

CompanyDisclaimer

© AzurRx BioPharma < www.azurrx.com <

Page 3: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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§ Biotechnologycompanyfocusedonthedevelopmentoftherapeuticproteins

§MultiplepipelineprojectsaddressinglargeglobalmarketsinGIandinfectiousdiseases

§MS1819fortreatmentofExocrinePancreaticInsufficiency(EPI)inpatientswithChronicPancreatitis(CP)andCysticFibrosis(CF)

– ClinicalproofofconceptforCPdemonstratedinFLIP-110study

– PreliminaryPhase2datasupportsefficacyandsafetyinCP

– Large,immediatelyaddressableEPImarketof~$1BinU.S.,$1.5Bworldwide(1)

– PartneredwithMayoly-SpindlerinEuropecovering30%ofMS1819clinicalcosts

§AZX1101betalactamasesForpreventionofnosocomial(hospitalacquired)andC.difficileinfectionsinpreclinicaltesting

– Addressesa$4.5-$11billionmedicalissue(2)

§Highlycashefficientoperationswith~30%ofR&DspendrebatedbyFrenchgovernment

(1) U.S.marketsize.Abbvie 2013-201710-Ksandannualreports(Creon),Zenpep &Pancreaze basedon2013-2014IMShistoricaldata/analystprojection.ManagementestimatesforglobalEPImarketsize

(2) CDC2016 © AzurRx BioPharma < www.azurrx.com <

InvestmentHighlights

Page 4: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Product Description IndicationDevelopmentPhase

Discovery Pre-Clinical Phase1 Phase 2 Phase3

MS1819

Yeastrecombinantlipase (Yarrowialipolytica LIP2)

TreatmentofEPI inCPpatients

TreatmentofEPI inCFpatients(1)

AZX1101 Synthetic β-Lactamase

Prevention ofnosocomialinfectionsandantibioticassociateddiarrhea

Expectedprogressin2018CurrentStatus

GITherapeuticProductPipelineMS1819

(1) Phase1carriedoutinEPIpatientswithCP

Page 5: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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§ Pancreasfunctioncanbecompromisedduetopancreaticcancer,surgicalexcisionorbehavioralissues(i.e.alcoholism),etc.

TherearetwobroadpatientspopulationswhichsufferfromEPIandrequiretreatment

CysticFibrosis(~30,000patients)

ChronicPancreatitis(~90,000patients)

§ Diseaseisgeneticandmajorityofpatientsrequiretreatmentfromage4onwards

ExocrinePancreaticInsufficiency(EPI)EPIisaconditioncharacterizedbydeficiencyofpancreaticenzymes,resultingintheinabilitytodigestfoodproperly

Page 6: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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FoodDigestionNeedsEnzymes,FatNeedsaLipaseAmylasesandproteasesinsalivaandstomachcompensateinpancreaticinsufficiency,butnobackupexistsforfatdigestion

Fat Fattyacids&glycerol

Protein Aminoacids

Carbohydrate Glucose

Amylase

Protease

Lipase

Ifthepancreasdoesnotfunctionproperly,oralsupplementsaretakentoallowforfatdigestion

Page 7: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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ClearUnmetMedicalNeed

CurrentEPITreatmentLimitations

§ Limitedeffectiveness

§ Lackofstabilityinacidicenvironment

§ Highpillburden

– Inconvenientforpatients

– Non-adherence

§ Sourcingandsupplyofporcine-derivedpancrelipase(PPEs):

– Subjecttopigherdmanagement

– Riskoftransmissionofpathogens

– Manufacturing/supplychaininconsistency

§ Adverseevent:fibrosingcolonopathy athighdoses

Opportunity§ Abilitytoreducepatientdailypillburdenof~25-40capsulesdownto~5-8 DailyDose

StandardofCare(1)

ExpectedDailyDoseMS1819

vs.

MS1819Current

(1) StandardofcareincludesdrugssuchasCreon,Zenpap andPancreaze

Page 8: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Large Established US Market Of ~$1.1 Billion(1)

All lipase products are pig derived and are less active at the pH in humans resulting in a large pill burden

(1) U.S.marketsize.Abbvie2013-201710K’s,10Q’sandannualreports(Creon),Zenpep&Pancreaze&Pertyzebasedon2013-2016 IMShistoricaldata/analystprojection(2) Creon2013-2016A- Abbvie,2017-2021E(3) 2017-2021EZenpepbasedonmedianorequityresearchprojections(4) 2021Pancreaze&Pertyzeequityresearchprojectionsunavailable

Growth, % 2014 2015 2016 2017 2018 2019 2020 2021

Creon(Abbvie)(2) 25.2% 22.5% 15.5% 11.1.% 6.6% 6.0% 4.5% 5.9%

Zenpep(Allergan)(3) - 105.4% 20.4% 6.5% 3.3% 3.2% 3.1% 11.3%

Pancreaze(J&J)(4) 4.0% 5.0% 2.4% -6.7% 4.7% 5.0% 5.1% -

Pertyze(Cornerstone)(4) - - 60.0% 42.3% 27.7% 18.0% 9.0% -

412 516632 730 811 865 917 958 1,015

3738

4042

4446 49 50

72

167201

214221

228 235 262

2013A 2014A 2015A 2016A 2017E 2018E 2019E 2020E 2021E

Historical Projected

$inmillions

1,207 1,259 1,277

1,076980

633

455

1,143

844

Page 9: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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In-Vitro ActivityofMS1819atpHRange

InvitrolipolyticactivityoftheMS1819lipaseinthepresenceofbilesaltsintheEuropeanandUSPharmacopeiatest(U/mg,PureEnzyme)

8

Lipo

lytic

Activ

ity(U

/mgofpurified

enzymeeq

uivalent)

14,000

12,000

10,000

8,000

4,000

2,000

6,000

NormalpH

PathologicalpH

9 7 5 4 36

MS1819Lipaseshowssuperioractivitytoporcinelipase

standardofcareattherelevantintestinalpH

range

MS1819 PorcinePancreaticExtract

0

Note:Innormalsubjects,physiologicalpHinduodenumisbetweenapproximately5and6.InCPandCFpHisloweredtoamore acidicrange,approximatelypH4to5.MS1819notinactivatedbybilesalts.

pHBasic Acidic

Page 10: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Steatorrhea,PerProtocol(absolutedifference12.6%)

CoefficientofFatAbsorption(CFA)**,PerProtocol(absolutedifference16.2%)

CFA(%)Steatorrhea(g/day)

40.3

31.539.6

44.1

01020304050607080

Baseline Treatment

MS1819 Placebo

49.659.18

48.9542.3

01020304050607080

Baseline Treatment

MS1819 Placebo

FLIP110StudyPerProtocolEfficacyResults(1)MS1819suggestsimprovementoftwokeyefficacyparameters

(1) Studynotpoweredforstatisticalsignificance,Pilot,proofofconceptstudy;mainobjectiveofsafetywithexplorationofefficacy**CFA=coefficientoffatabsorption,ameasureofdietaryfatdigestion

Resultsobtainedonthe2mainefficacycriteria(steatorrheaandCFA)demonstratedapositiveeffectofMS1819comparedtoanegativeeffectofthe

placebo.

Page 11: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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ClinicalTrialDesignforMS1819Phase2ainChronicPancreatitisTrialstartedinQ42016inAustraliaandNewZealand;12-15patientsenrollmenttarget

(B)Washout12-15days

(C)Open-labelphase

12-15dayseachstep

(A)Screening0-30days

UsualPPEtreatment

PreviousPPEtreatment

Screening

MS1819-SD2240mg/day

Baseline

Inclusion

MS1819-SD280mg/day

MS1819-SD560mg/day

MS1819-SD1120mg/day

(D)Follow-up12-15days

V1 V2 V3

V4

V5

V6 V7

V8

Fecalelastase-1atscreening<100µg/gInpatientCFAmeasurement(meanof3consecutivedays)OutpatientCFAmeasurement(meanof3consecutivedays)

Page 12: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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0

5

10

15

20

25

30

CFAImprovement

Porcine MS1819Ph1a/b

Bacteriallipase

Abso

lute

% In

crea

se F

rom

Was

hout

Doseresponse MS1819highestdoseshows>21%CFAimprovement

Clinicalactivity Response inverselycorrelatedtodiseaseseverity

Bristolscale Improvement

Nutritionalstatus Favorable(unchanged)

Peakresponse 57%CFAImprovement

Safety Noseriousadverseeventsornotablemildtomoderateevents

DatafromMS1819highestdose

MS1819CFAResponseInitialresultsdemonstratedCoefficientofFatAbsorption(CFA)improvement,solidsafetyprofile,anddoseresponse

Page 13: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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PerProtocol– CFAAbsolute(%)

PerProtocol– RelativeCFAchange(%)

MS1819Phase2aResultsareFavorableFirst6patients’resultsshowCFAimprovement,solidsafetyprofile,anddoseresponse

48.4%55.5%

61.0%68.8% 69.8%

WO 280mg/d 560mg/d 1120mg/d 2240mg/d

-

7.1%

12.6%

20.4% 21.4%

WO 280mg/d 560mg/d 1120mg/d 2240mg/d

Page 14: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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PCT/FR1999/002079 family PCT/FR2000/001148 family PCT/FR2006/001352 familyTitle Methodfornon-homologous

transformationofYarrowialipolytica

Cloningandexpressinganacid-resistantextracellularlipaseofYarrowia lipolytica

Methodforproducinglipase,transformedYarrowialipolyticacellcapableofproducingsaidlipaseandtheiruses

Abstract TheinventionconcernstheintegrationofageneofinterestintothegenomeofaYarrowia straindevoidofzetasequences,bytransformingsaidstrainusingavectorbearingzetasequences

Theinventionconcernsnucleicacidscodingforacid-resistantextracellularlipases,inparticularC.ernobiiorYarrowialipolyticayeastsandtheproductionofsaidlipasesinrecombinantform

MethodforproducingYarrowialipolyticaacid-resistantrecombinantlipaseutilizingaculturemediumwithoutanyproductsofanimaloriginornon-characterizedmixturessuchastryptone,peptoneorlactoserum,inadditiontoitsuses

Prioritydate 01.09.1998(FR98/10900) 28.04.2000(FR00/01148) 15.06.2006(F026900039)

§ MS1819coveredbygrantedpatentsuptoJune15th,2026(1)

§ ApatenttermextensionofuptofiveyearsmaybegrantedbytheUSPTO,resultinginpossibleendoftheprotectiononJune15th,2031(1)

§ TheFDAcurrentlygrants12yearsofexclusivityfornovelbiologicsfromfirstapproval(e.g.through2033ifapprovedin2021)

§ Freedomtooperate:noblockingpatentshavebeenidentifiedsofar

.

IntellectualPropertyLicensedpatentsrelativetotheMS1819program

(1) RelatestoPCT/FR2006/001352family

Page 15: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Competition to date

Approved and marketed – Only PPEs1 – A mix of lipase, protease and amylase

a.CREON®, Abbott

b.ZENPEP®, VIOKASE® and ULTRESA®, Aptalis Pharma

c.PANCREAZE®, Johnson and Johnson

d.PERTZYE®, Digestive Care Inc.

Recombinant products under development for EPI

a.SOLPURA® aka Liprotamase®, Alnara/Eli Lilly (cross-linked bacterial lipase, protease and amylase)

b.NM-BL burlulipase, Nordmark Pharma (bacterial lipase)

1 All PPE’s must go through NDA approval since 4/28/2004 announcement by FDA

Terminated recombinant products for EPI

a.Dog recombinant lipase, rGL, Meristem

b.Recombinant Microbial Lipase, SLV-339, Solvay Pharmaceuticals

c.Human bile-salt stimulated lipase (rhBSSL), Biovitrum AB-for neonates

Page 16: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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ExocrinePancreaticInsufficiencyPrimaryMarketResearchSupportforMS1819fromPhysiciansandPayers(1)

(1) Resultsof10gastroenterologistand5payerinterviewsconductedbyanoutsideresearchfirmin8/2014

87%ofalldiagnosedEPIpatientsaretreatedwithpancreaticenzymereplacementtherapy

Reducingpillburden,increasingpHstability,andprovidingaporcinealternativePancreaticEnzymeReplacementTherapies(PERTs)isseenasasignificantopportunityinmeetingcurrentunmetneeds

PotentialforMS1819tocapture57%ofnewlydiagnosedEPIpatients;howeverthereislikelylimitedswitchingopportunityforcurrentlytreatedpatients

InterviewedpayersdonotactivelymanagecostsacrossPERTs,whiletheyhaveapositiveviewofMS1819,theydonotfeelthattherearegroundsforhigherpriceswithoutmoreclinicaldata

Page 17: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Product Description IndicationDevelopmentPhase

Discovery Pre-Clinical Phase1 Phase 2 Phase3

MS1819

Yeastrecombinantlipase (Yarrowialipolytica LIP2)

TreatmentofEPI inCPpatients

TreatmentofEPI inCFpatients(1)

AZX1101 Synthetic β-Lactamase

Prevention ofnosocomialinfectionsandantibioticassociateddiarrhea

Expectedprogressin2018CurrentStatus

GITherapeuticProductPipelineAZX1101

(1) Phase1carriedoutinEPIpatientswithCP

Page 18: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Mostmodernantibioticskillbothgoodandbadmicrobes,strippingthebodyoffriendlybacteriaintheprocessofadministration

Bothfriendlyandharmful organismsexistnaturally

Theymayunintentionallyupsetthenaturalbalanceofthegutmicrobiomebykillingoffgoodbacteria

Microbeslivingonandwithinthehumanbody

Deathofmicrobes– Badlefttoflourish

IVantibioticsarecarriedtotheliver,transportedtobileandexcretedviathelargeintestine

Antibiotics

TheMicrobiomeandDiseaseTheImpactofModernAntibiotics

Page 19: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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14MPatients

27MPrescriptions

80-400MDoses

$2B-10BMarket

• PatientsrequiringIVantibiotictherapy

• Higherriskpatientswithmultiplescripts

• 15-75%market

• 5daysprescriptiontherapy

• 4Dosesperday

• $25perdose

ScaleoftheC.DiffProblemand14MPatientsAffectedAnnually(1)AZX1101forthePreventionofC.DifficileInfectionsandNosocomialInfections

(1) Sources:2012IMSHealthandCDMHospitaldatabases.Managementestimates.

Page 20: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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§ Applications

– Oral,non-systemicmedicinetoactlocallyintheGItractwiththepotentialtopreventhospital-acquiredinfectionsbyresistantbacterialstrainsinducedbyparenteraladministrationofβ-lactamantibiotics.

– Potentialpreventionofantibiotic-associateddiarrhea(AAD).

§ Hospital-acquired(nosocomial)infectionshaveahugeeconomicimpactonsociety(2) andareamajorpublichealthconcerncontributingtoincreasedmorbidity,mortality,andcost.

– TheCentersforDiseaseControl(CDC)hasestimatedthatroughly1.7millionhospital-associatedinfections(i.e.~5%ofthenumberofhospitalizedpatients),causeorcontributeto99,000deathseachyearintheUSA(1),withtheannualcostrangingfromUS$4.5to$11billion).

§ TheCentersforMedicareandMedicaidServices(CMS)hasbeguntopenalizehospitalsbynotpayingfor“avoidablecosts.”

AZX1101– OpportunityOverviewAddressingNosocomialInfections

(1) CDC2016.Managementestimates.(2) ~2million(HAIs)intheU.S,90,000estimateddeaths.CentersforDiseaseControl.2016OveralldirectcostofHAIstohospitalsrangesfrom$28-$45B.Scott,R.Douglas.

”TheDirectMedicalCostsofHealthcare-AssociatedInfectionsinU.S.HospitalsandtheBenefitsofPrevention”.CentersforDiseaseControlandPrevention.March2009.

Page 21: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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AZ1101Penicillins(withoutbetalactamaseinhibitors)

Penicillins(withbetalactamaseinhibitors)

3rd generation cephalosporins

Methicillin

Aminoglycosides

Somefluoroquinolones

Macrolides

Tetracyclines

Lincosamides

AZX1101TargetsMultipleAntibioticsintheGutFiledIntellectualPropertyCoversMultipleAntibioticClasses

Page 22: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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ManagementTeam

ThijsSpoorCEO

Maged ShenoudaCFO

DanielDupretChiefScientificOfficer

YvesLeblondDirectorResearchandDevelopment

LucLebretonR&DProgramsDirector

MartinKrusinBusinessDevelopment

Page 23: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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Achieved/AnticipatedMilestonesforAzurRxPotentialforMultipleCatalysts

Milestone Timing

InitiationofMS1819Phase2CPstudyinAustralia Q12017

Resultsfromfirst3patients in MS1819Phase2CPstudy Q22017

Resultsfromfirst6patientsinMS1819Phase2CPstudyshowingsafetyandefficacy>21%CFA Q32017

ProofofconceptdataforAZX1101 Q12018

CompletionofenrollmentinMS1819Phase2 CPstudy 1H2018

InitiationofMS1819Phase2CFstudy Mid2018

SubmitIND/CTAforMS1819 2H 2018

FinalresultsofMS1819Phase2CPstudy 2H2018

Initialresultsfrom CFstudy Q42018

Page 24: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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IPO: 2016

Nasdaq: AZRX

Market Cap: $37MM(1)

Shares Outstanding: 11,554,146(2)

Cash Position (9/30/2017): $2.9MM

Locations: New York &Nîmes (France)

FinancialOverview

(1) As of 11/30/2017(2) As of 11/30/2017

Page 25: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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§ Biotechnologycompanyfocusedonthedevelopmentoftherapeuticproteins

§MultiplepipelineprojectsaddressinglargeglobalmarketsinGIandinfectiousdiseases

§MS1819fortreatmentofExocrinePancreaticInsufficiency(EPI)inpatientswithChronicPancreatitis(CP)andCysticFibrosis(CF)

– ClinicalproofofconceptforCPdemonstratedinFLIP-110study

– PreliminaryPhase2datasupportsefficacyandsafetyinCP

– Large,immediatelyaddressableEPImarketof~$1BinU.S.,$1.5Bworldwide(1)

– PartneredwithMayoly-SpindlerinEuropecovering30%ofMS1819clinicalcosts

§AZX1101betalactamasesForpreventionofnosocomial(hospitalacquired)andC.difficileinfectionsinpreclinicaltesting

– Addressesa$4.5-$11billionmedicalissue(2)

§Highlycashefficientoperationswith~30%ofR&DspendrebatedbyFrenchgovernment

(1) U.S.marketsize.Abbvie 2013-201710-Ksandannualreports(Creon),Zenpep &Pancreaze basedon2013-2014IMShistoricaldata/analystprojection.ManagementestimatesforglobalEPImarketsize

(2) CDC2016 © AzurRx BioPharma < www.azurrx.com <

InvestmentHighlights

Page 26: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

C O N F I D E N T I A L | www.azurrx.com

AdditionalCorporateInformationAppendix

Page 27: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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§ Primaryefficacyendpoint

CFAchangefrombaselineandmeasuredattheendofPhaseConstandardizedhigh-fatmealsandstoolcollectionfor3days(dyemarker)

§ Secondaryefficacyendpoints

Keysecondaryendpoints- CFAchangefrombaselineandmeasuredatstep1-3ofPhaseC- Numberofdailyevacuations- ConsistencyofstoolsassessedbytheBristolscale

Othersecondaryendpoints- Bodyweightandbodymassindex- Weightofstoolsandabdominaldiscomfort- Absorptionvariables:nitrogenfecalcontentandsteatorrhea- Fastingglucose- Biochemistryandlipidparameters- Vitamins,Bone-turnovermarkers,andcirculatingnutritionproteins

MS1819CPPhase2a- PrimaryandSecondaryEfficacyEndpoints

Page 28: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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§ Expectedclinicaladversereactionevents:– Allergicreaction– Hypoglycemia– Constipation– Abdominaldiscomfortorpain

§ Biologicalmarkers– Liver(AST,ALT)andmuscularenzymes(CK)– Renal(creatinemia,ureticacid)andpancreasmarkers(lipasemia,amylasemia)

§ SerumLIP2andanti-LIP2antibodies(ADA)detectionassays

§ Inaddition,laboratorytestswillinclude:– Fecalcalprotectinandfecalchymotrypsin– Hematology(completebloodcellcount),– Biochemistry(creatinine,urea,AST,ALT,ALP,GGT,bilirubin,andCK)– Serumvitamins,fastingglycaemia,lipidparameters,circulatingnutritionproteins,andbone-turnovermarkers.

MS1819CPPhase2a- SafetyEndpoints

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0

250,000

500,000

750,000

1,000,000

1,250,000

1,500,000

1,750,000

2,000,000

2,250,000

2,500,000

2,750,000

3,000,000

3,250,000

3,500,000

3,750,000

4,000,000

0 4 8 12 16 20 24 28 32 36 40

EnzymaticActivy(TBU)ByPillBurden

Porcine- 00

35mgFD- 2

140mgFD- 2

212mgFD- 1

300mgFD- 0

400mgFD- 00

MS1819fillbycapsulesize

GreaterActivityEnablesDosingFlexibilityVariationsincapsulesizeallowallpatientstobedosedat1-2capsulespermeal

Page 30: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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0

250,000

500,000

750,000

1,000,000

1,250,000

1,500,000

1,750,000

2,000,000

0 4 8 12 16 20 24 28 32 36 40

EnzymaticActivity(TBU)ByPillBurden

Creon

FD(4x)

Dailycapsuleburdenisproportionaltoenzymaticrequirements

1120mg/dayofMS1819

EnzymaticActivity(TB

UatpH6

)

Capsules

EnzymeRequirementsVary

Inamoderatepatient,theexpecteddailyminimumrequirementforenzymaticactivityisexpectedtobe1,000,000TBU

§ Moderatepatientscurrentlycontroldiseasetaking~25Creoncapsulestogive1,000,000TBU

§ Phase1wasdosedat250,000TBUgenerating~16%CFA

§ Phase2atestshigherdosescloserto1,000,000TBUwhichareexpectedtoprovideagreaterCFAresponse

Porcine

MS1819

DoseResponseThePhase1dailydoseofMS1819wasabout¼oftherequiredamountexpectedforamoderatepatient,higherdosesinphase2areexpectedtoachievebetterfatdigestion

Page 31: AzurRxBioPharma Presentation January 2018 · §AZX1101 beta lactamases For prevention of nosocomial (hospital acquired) and C. difficile infections in preclinical testing –Addresses

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BoardofDirectors

EdBorkowskiMr.Borkowski wastheCFOofConcordiaInternational andpreviouslyservedastheCFOofConvaTec Healthcare,CareFusionCorporationandMylan,Inc.andinavarietyoffinancepositionsatPharmacia,AmericanHomeProducts,CyanamidandatArthurAndersen.Mr.Borkowski holdsaBachelorofScienceinEconomicsandPoliticalSciencefromAlleghenyCollegeandaMasterinBusinessAdministrationinFinanceandAccountingfrom RutgersUniversity.

CharlesCasamentoMr.Casamento hasbeenexecutivedirectorandprincipalofTheSageGroup,ahealthcareadvisorygroup.HewaspresidentandCEOofOsteologix fromOctober2004untilApril2007,thefounder,president,chairmanandCEOofQuestcor Pharmaceuticals,andheldseniorleadershipandboardpositionsatRiboGene Inc,Indevus (formerlyInterneuronPharmaceuticals),GenzymeCorporation,Johnson&Johnson,Hoffmann-LaRoche andSandoz.Heholdsabachelor'sdegreeinPharmacyfromFordhamUniversityandanM.B.A.fromIonaCollegeandservesontheBoardsofDirectorsofInternationalStemCellCorporationandRelmada Therapeutics.

AlastairRiddell,MDDr.AlastairRiddelliscurrentlyChairmanofaprivateUKbiotech,NemesisBiosciencesLtd,ChairmanofaUKAIMlistedmedical imagingcompanyFeedbackplcandChairmanoftheSouthWestAcademicHealthScienceNetwork,andisalsoontheboardofdirectorsofSkyline VetPharma.Dr.Riddellhasover30yearsexperienceinthepharmaceutical,lifescienceandbiotechindustries,atLederle (nowPfizer),Centocor (nowJ&J),AmershamInternational(nowGEHealthcare)andasCEOofPharmagene,ParadigmTherapeuticsandStemCellSciences.Hebeganhiscareerasamedicaldoctor.

VernLeeSchramm,PhDDr.SchrammservedasChairmanoftheDepartmentofChemistryattheAlbertEinsteinCollegeofMedicinefrom1987to2015.Dr.SchrammwaselectedtotheNationalAcademyofSciencesin2007andwastheAssociateEditorforthe JournaloftheAmericanChemicalSociety from2003to2012.HehasbeenanadvisortoPicoPharmaceuticals,Metabalon Biochemistry,Sirtris Scientific,andBioCryst Pharmaceuticals.HeobtainedhisBSinBacteriologyfromSouthDakotaStateCollege,aMaster’sDegreeinNutritionbiochemistryfromHarvard,andaPh.D.fromAustralianNationalUniversity.

Maged ShenoudaMr.Shenouda waspreviouslytheHeadofBusinessDevelopmentandLicensingatRetrophin,Inc.Mr.Shenouda spentthebulkofhiscareerasanequityanalystatStifel Nicolaus,UBS,JPMorgan,CitigroupandBearStearnscoveringU.SandEuropeanpharmaceuticalcompanies.Mr.Shenouda wasamanagementconsultantwithPricewaterhouseCoopers,andamanagedcarespecialistforAbbottLaboratories.Mr.Shenouda earnedaB.S.inpharmacyfromSt.John'sUniversityanM.B.A.fromRutgersUniversityGraduateSchoolofManagement.

ThijsSpoorMr.SpoorwaspreviouslyPresidentandCEOofFluoropharma Medical,Inc.fromFebruary14,2011untilDecember31,2015. HewastheCFOforSunstoneBioSciences,astrategyconsultantatOliverWyman,anequityresearchanalystatJ.P.MorganandCreditSuissecoveringthebiotechnologyandmedicaldeviceindustries.Hespent11yearswithAmersham /GEHealthcareandholdsaPharmacydegreefromtheUniversityofTorontoaswellasanM.B.A.fromColumbiaUniversitywithconcentrationsinfinanceandaccounting.

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