investor presentation revisedv.6-2 · 2020. 5. 21. · hepatitis b crispr/cas9 human papilloma...
TRANSCRIPT
Antiviral Therapies
cocrystalpharma.com
Forward Looking Statements
This presentation contains forward-looking statements, including the timing of our drug development programs. Risks include delays in manufacturing created by third parties and the ability of clinical research organizations to recruit patients. Forward-looking statements also are prefaced by words such as "expect," "plan," "intend," "anticipate," and similar words. Forward-looking statements are based on our current expectations and assumptions regarding our business, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. Our actual results may differ materially from those contemplated by the forward-looking statements for a variety of reasons, including those contained in our Form 10-K, as amended, for the year ended December 31, 2015, form 10Q for the quarter ending June 30, 2016. We caution you, therefore, against relying on any of these forward-looking statements. They are neither statements of historical fact nor guarantees or assurances of future performance. We do not undertake any duty to update these forward-looking statements.
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cocrystalpharma.com
§ Clinicalstageantiviralcompany
§ Multipleopportunitiesindifferentviraldiseases
Company Highlights
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Influenza PB2inhibitors,PA inhibitors,PB-1inhibitorsHepatitis C NNI,nucleosideinhibitors,helicaseinhibitors,NS5A
inhibitorsNorovirus Nucleoside inhibitors,NNIHepatitisB CRISPR/Cas 9Humanpapillomavirus CRISPR/Cas9
cocrystalpharma.com
Crystallography Technology Platform
§ Abilitytoquicklygrowultra-highresolutioncrystalsofreplicationenzymes
§ Rapidturnaround ofstructuralinformationthroughhighlyautomatedX-raydataprocessingandrefinement
§ Discoveryofnovelbindingsites –overlaystructureoffragmentsboundtonovelsites
§ Provides3Dstructureofinhibitorcomplexesatnear-atomicresolution–providesimmediateinsighttoguidedrugdiscovery
FragmentE
FragmentD
FragmentA
FragmentB
FragmentC
Fragmenthits
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cocrystalpharma.com
Cocrystal Drug Discovery Process
Nearatomicresolution,X-rayqualitycrystalproduction
Drugpocketselection
Hit-to-leadprocess
Leadoptimization
Drugcandidates
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cocrystalpharma.com
Opportunities
Thereexistssignificantunmetmedicalneedsacrossalargevarietyofviralinfectionsincluding…
HepatitisB&C
Leadingcausesofliverfailureandlivercancer
Chronicinfections>100millionHCV>400millionHBV
OpportunityforshorterdurationinHCVanda
cureinHBV
Norovirus
• Chronic(potentiallyorphanindication)
• Acutegastroenteritis
>250millionacutecases/year
NotreatmentavailableEconomiccostintheUS
alone >$5Billion
InfluenzaA&B
Seasonalandpandemic
3- 5millioninfections/year
EstimatedeconomicimpactofseasonalfluinUS:$50Bto$150B
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cocrystalpharma.com
Company Pipeline
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Therapeutic area LeadDiscovery
Preclinical Phase I PhaseII
HepatitisCCC-31244(NNI)
Hepatitis CCC-2069(NS5A)
HepatitisCCC-2850 (Nuc)
Influenza APB2inhibitor
NorovirusNuc&NNI
HepatitisBandHPV(CRISPR/Cas9)
cocrystalpharma.com
HCV Market Dynamics
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Today and in the future
*BloombergIntelligence(BI)projections,asof2016
§ Hugemarket:>10millionpatientsacrosstheUS,Japan,WesternEurope
§ Currentlytreating400,000+patientsperyear
§ Manyundiagnosedpatients
§ Pricingwillbecompetitive,butthemarketwillstillbesignificantintheforeseeablefuture
§ Greatneedforashorterdurationtherapy
cocrystalpharma.com
Cocrystal’s HCV DAA Combinations
Multipleopportunitiesindevelopingcombinationultra-short,alloralpan-genotypiccure(in-houseorwithpartners)
Pan-genotypicNS5BNuc
Pan-genotypicNS5AInhibitor
Pan-genotypicNS5BNNI
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Oral,Pan-genotypic,Ultra-shorttherapy
cocrystalpharma.com
CC-31244: Pan-genotypic NNI
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§ HighlypotentNS5Bpolymeraseinhibitor(EC50=7nM)
§ Pan-genotypicactivity(genotypes1-6)
§ Highbarriertodrugresistance
§ PhaseIa doseescalatingsafetystudycompleted
§ ViralloaddatainHCVsubjectsavailableinQ4,2016
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1 2 3 4 5
IC50foldch
ange
HCV-796
31228 CC-31244 959 985 HCV-796
Cocrystal’sNNI-4Leads
Cocrystal’sBackupLeads
S365T(NNI-4)
N316Y(NNI-4)
L419M(NNI-2)
S282T(Nuc)
Drugresistancevariants
Unique Drug Resistance Property of Cocrystal NNIs
§ CC-31244showspan-genotypicandbroadactivityagainstcommondrugresistantvariants
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cocrystalpharma.com
CC-31244 Phase Ia Clinical Trial Update
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§ Asingle- andmultiple-doseassessmentofthesafetyandpharmacokineticsofpan-genotypicNNI,CC-31244
• Single-dosecompleted:fivecohortsofhealthyvolunteersat10,50,100,200,and400mg
• Multiple-dosecompleted:twocohortsofhealthyvolunteersat200mgx7daysand400mgx7days)
• Noadverseeffectsobserved
cocrystalpharma.com
CC-31244 Phase Ib Clinical Trial Update
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§ Proof-of-conceptPhase1bstudyongoing
• HCVinfectedsubjectswithminimalfibrosisandnosignificantco-morbidities
• Repeat-dose,randomized,monotherapytrial(400mgx7days)
• Viralload,drugresistance,andpharmacokineticpropertieswillbedetermined
cocrystalpharma.com
CC-2069: Pan-genotypic NS5A
§ Novel,highlypotent,pan-genotypic,NS5Ainhibitor(GT1bEC50<10pM)
§ ActiveagainstcommonNS5Adrugresistantvariants
§ Preclinicalcharacterizationongoing
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cocrystalpharma.com
HCV Nucleoside Program: CC-2850
Searchfornextgenerationbackboneforcombinationtherapy
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§ Novel,potent,pan-genotypic nucleosideinhibitor
§ Activeagainstcommonnucleosideinhibitordrugresistant(S282T)
§ Preclinicalcharacterizationongoing
cocrystalpharma.com
HCV Helicase Lead Discovery Program
Providesuniqueopportunitiesfordrugcombinations
§ InhibitsessentialviralRNAunwindingprocess
§ First-in-classpan-genotypicinhibitors(newmechanismofaction)
§ Highlyconserveddrugbindingmodedemonstratedinallgenotypecrystalsdeveloped(Genotypes1-6)
§ PotentiallyanidealcombinationcandidatewithHCVNuc,NNI,NS5A,and/orproteaseinhibitors
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cocrystalpharma.com
Influenza Program
Influenzaleads:PB2,PB1andPAInhibitors
§ Focusonthreedifferenttypesofinfluenzapolymeraseinhibitors:PB2(cap-binding),PB1(polymerase),andPA(endonuclease)
§ Novel,potentstructure-basedinfluenzaAPB2inhibitorsareatpreclinicalstage
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cocrystalpharma.com
Influenza Program
PotentPB2leadsbindtohighlyconservedbindingpocket
H1N12009
Virginia
H1N11918
Spanish
H7N92013
Zhejiang
H5N11996
Guangdong • H1N1 1918
• H1N1 2009• H5N1 1996 • H7N9 2013
InfluenzaPB2inhibitorSelectedinfluenzaAPB2crystals
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cocrystalpharma.com
Norovirus Program
Unmet&underappreciatedmedicalneed
§ Prophylaxis
§ TreatmentØ Acute(foodborne)
Ø Chronic(Immunocompromised)
Ø Chronic(transplantpatients)
19-20millionillnesseachyear(1in14Americansbecomeilleachyear)
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cocrystalpharma.com
Norovirus Program
BroadspectrumNoro PolymeraseInhibitors
• HumanNorwalk• HumanNoro• MurineNoro
Noronucleosides NoroNNI
HumanNoro HumanNorwalk MurineNoro
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§ Drugable pocketidentified§ Activenucleosidecandidatesidentified
§ Animalmodeldatasupportsactivityinvivo
§ Leaddiscoveryisongoing
cocrystalpharma.com
Hepatitis B Market
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§Hugemarket:400millionchronicallyinfectedglobally;asmanyas2millioninfectedwithchronicHBVinUSalone
§ Currenttherapiesonlysuppress,butdonoteliminate
There is no approved cure at this time
cocrystalpharma.com
CRISPR/Cas9 For Potential HBV Therapy
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§ In-licensedfromDukeUniversityandEmoryUniversityforHepatitisBandHumanPapillomaVirus
§ CRISPR/Cas9allowsforeditingofviralDNA
§ PotentialcureforchronicHBV
cocrystalpharma.com
Cocrystal Board of Directors
RaymondSchinazi(Chairman)
GaryWilcox(ViceChairman)
DavidBlock
PhilFrost
JaneHsaio
StevenRubin
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cocrystalpharma.com
Cocrystal Management
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GaryWilcox,Ph.D.,InterimCEOChairmanandCEOofCocrystalPharma2008-2014;BoardofDirectorsandExecutiveVicePresidentofOperationsofIcosCorporation(ICOS-NASDAQ)from1993to2007.ViceChairman,ExecutiveVicePresident,andDirectorofXomaCorporation(XOMA-NASDAQ);Chairman,CEO,andPresidentofIngene(IGEI-NASDAQ)from1982-1989;ProfessorofMicrobiologyatUCLAfor10years.
SamLee,Ph.D.PresidentPresidentofCocrystalPharma2008-present;Anti-infectivedrugdiscoveryprojectmanagementICOSCorporation1999-2008.Dr.LeereceivedhisPh.D.inBiologicalSciencesfromtheUniversityofNotreDameandpostdoctoraltraininginviralbiochemistrywithDr.I.R.LehmanatStanfordUniversity.
CurtisDale,InterimChiefFinancialOfficerandControllerCorporateControllerSolvayPharmaceuticals;ExecutiveFinanceDirector,Stiefel Laboratories;Multiplefinancialpositions,Bristol-MeyersSquibb.
WaltLinscott,GeneralCounselandCorporateSecretaryGeneralCounselandCorporateSecretary,Carestream Health;VicePresident,GeneralCounselandCorporateSecretary,SolvayPharmaceuticals;ChairofLifeSciencesPracticeofaninternationallawfirm.
cocrystalpharma.com
Scientific Leadership
Dr.RaymondSchinaziCo-FounderofPharmasset,Idenix,Triangle;Founder,RFSPharma
Dr.RogerKornbergCocrystalCo-founder2006Nobelprizewinnerinchemistry
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cocrystalpharma.com
Transformational Year: 2016
HCV§ InitiatedclinicaltrialforCC-31244(NNI)§ CompletedPhaseIa doseescalationsafetystudy§ AntiviralactivitydatainHCVsubjectsforCC-31244inQ42016§ PreclinicalcharacterizationofCC-2069(NS5A)- ongoing§ PreclinicalcharacterizationofCC-2850(Nuc)- ongoing
Influenza§ LeadselectionofinfluenzaAPB-2inhibitors
Norovirus§ Nucleoside/NNIindiscoverystage
HBV(CRISPRCas 9)§ InvitroproofofconceptandanimalmodelstudiesofCRISPR/Cas9
forhepatitisB– ongoing
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