sorrento therapeutics, inc. · on september 22, 2016, scintilla pharmaceuticals, inc....
TRANSCRIPT
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549
FORM 8-K
CURRENT REPORT Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): September 22, 2016
SORRENTO THERAPEUTICS, INC. (Exact name of registrant as specified in its charter)
9380 Judicial Drive San Diego, CA 92121
(Address of principal executive offices)
Registrant’s telephone number, including area code: (858) 210-3700
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Delaware 001-36150 33-0344842(State or other jurisdiction of
incorporation or organization) (Commission File Number)
IRS Employer Identification No.)
Written communication pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
On September 22, 2016, Scintilla Pharmaceuticals, Inc. (“Scintilla”), a subsidiary of Sorrento Therapeutics, Inc. (“Sorrento”), commenced an equity financing process (the “Proposed Financing”). A presentation regarding Scintilla has been posted under the “Investors” section of Sorrento’s website at www.sorrentotherapeutics.com and is furnished as Exhibit 99.1 to this Current Report on Form 8-K and incorporated herein by reference.
The information contained in, or incorporated into, this Item 7.01, including Exhibit 99.1 hereto, is being furnished and shall not be deemed “filed” for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any registration statement or other filing under the Securities Act of 1933, as amended (the “Securities Act”), or the Exchange Act, except as shall be expressly set forth by specific reference to such filing.
This Current Report on Form 8-K, including Exhibit 99.1 hereto, is neither an offer to sell any securities, nor a solicitation of an offer to buy any securities, nor shall there be any sale of any such securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. Any sales of securities in the Proposed Financing will be made solely to “accredited investors,” as defined under Regulation D under the Securities Act (“Regulation D”), pursuant to the exemptions from registration provided by Rule 506(c) of Regulation D.
Safe Harbor for Forward-Looking Statements
Any statements contained in this Current Report on Form 8-K, including Exhibit 99.1 hereto, other than statements of historical fact, including statements about management’s beliefs and expectations regarding the Proposed Financing and Scintilla’s prospects and future results, are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 and should be evaluated accordingly. These statements are made on the basis of management’s views and assumptions regarding future events and business performance. Words such as “estimate,” “believe,” “anticipate,” “expect,” “intend,” “target,” “should,” “may,” “will” and similar expressions and their negative forms are intended to identify forward-looking statements.
Forward-looking statements involve risks and uncertainties that may cause actual results to differ materially from any future results, performance or achievements expressed or implied by such statements. These risks and uncertainties include, without limitation, Scintilla’s ability to complete its proposed acquisition of Semnur Pharmaceuticals, Inc. and to integrate Semnur Pharmaceuticals, Inc.; developments regarding Scintilla’s research and development efforts and clinical trials; regulatory reviews and approvals; and Scintilla’s ability to hire and retain key employees.
These and other risks and uncertainties are discussed in more detail in Sorrento’s filings with the Securities and Exchange Commission (the “SEC”), including Sorrento’s most recent Annual Report on Form 10-K, most recent Quarterly Reports on Form 10-Q and recent Current Reports on Form 8-K. Many of these risks are beyond management’s ability to control or predict. Should one or more of these risks or uncertainties materialize, or should the assumptions prove incorrect, actual results may vary in material aspects from those currently anticipated. Investors are cautioned not to place undue reliance on such forward-looking statements as they speak only as of the date the statement is made. All forward-looking statements attributable to Sorrento or Scintilla or persons acting on behalf of either Sorrento or Scintilla are expressly qualified in their entirety by the cautionary statements and risk factors contained or referenced in this Current Report on Form 8-K and Sorrento’s filings with the SEC. Except as required under the federal securities laws or the rules and regulations
Item 7.01 Regulation FD Disclosure.
of the SEC, neither Sorrento nor Scintilla undertakes any obligation to update or review any forward-looking statement or information, whether as a result of new information, future events or otherwise, except as required by law.
99.1 Scintilla Pharmaceuticals, Inc. Slides, dated September 2016.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
Dated: September 22, 2016
SORRENTO THERAPEUTICS, INC.
By: /s/ Henry Ji, Ph.D. Name: Henry Ji, Ph.D.Title: President and Chief Executive Officer
Exhibit 99.1
Exhibit99
1SC
INTILLA
Corporate
PresentationSeptem
ber2016
D
isclaimer
ScintillaPharm
aceuticalsInc
(“Scintilla”)isasubsidiary
ofSorrentoTherapeuticsInc
(the“C
ompany”)
apublicly
tradedcom
panyon
theN
asdaqStock
Market(“SR
NE”)
Certain
statementscontained
inthispresentation
orinotherdocum
entsoftheC
ompany
alongw
ithcertain
statementsthatm
aybe
made
bym
anagementofthe
Com
panyorally
inpresenting
thismaterial
may
contain“forw
ardlooking
statements”
asdefinedin
thePrivate
SecuritiesLitigationR
eformA
ctof1995These
statementscan
beidentified
bythe
factthattheydo
notrelatestrictly
tohistoric
orcurrentfactsThey
usew
ordssuchas“estim
ate”
“expect”“intend
”“believe
”“plan
”“anticipate
”“projected”
andotherw
ordsandterm
sofsimilarm
eaningin
connectionw
ithany
discussionoffuture
operatingor
financialperform
anceorcondition
Thesestatem
entsarebased
uponthe
currentbeliefsandexpectationsofthe
Com
pany’smanagem
entandare
subjecttosignifi
cantrisksanduncertainties
Statementsregarding
futureaction
futureperform
anceand/orfuture
resultsincludingw
ithoutlimitation
thoserelating
tothe
timing
forcompletion
andresultsof
scheduledoraddition
alclinicaltrialsandthe
FDA
’sorotherregulatoryreview
and/orapprovalandcom
merciallaunch
andsalesresults(ifany)ofthe
Com
pany’sformula
tionsandproductsand
regulatoryfilingsrelated
tothe
same
partneringand
collaborationopportunities
andScintilla’sproposed
acquisitionofSem
nurPharm
aceuticalsInc
may
differfromthose
setforthin
theforw
ardlooking
statements
Peaksales
marketsize
andincidence
estimateshave
beendeterm
inedon
thebasisofm
arketresearchand
comparable
productanalysis
butnoassurancescan
begiven
thatsuchsaleslevelsw
illbeachieved
ifatallorthatsuch
marketsize
estimatesw
illproveaccurate
TheC
ompany
assumesno
obligationto
updateforw
ardlooking
statem
entsascircumstanceschange
Investorsareadvised
toconsultfurtherdisclosuresthatthe
Com
panym
akesorhasmade
onrelated
subjectsinthe
Com
pany’sForm10
K10
Qand
8K
reportsN
ASD
AQ
:SRN
EB
ecauseactualresultsare
affectedby
theseand
otherpotentialriskscon
tingenciesanduncertainties
theC
ompany
cautionsinvestorsthatactualresultsmay
differmaterially
fromthose
expressedorim
pliedin
anyforw
ardlooking
statement
Itisnotpossibleto
predictoridentifyallsuch
riskscontingenciesand
uncertaintiesThe
Com
panyidentifiessom
eofthese
factorsinitsSecuritiesand
ExchangeC
omm
ission(“SEC
”)filingsonForm
s10K
10Q
and8
Kand
investorsareadvised
toconsultthe
Com
pany’sfilingsfora
more
complete
listingofrisk
factorscontingenciesand
uncertaintieseffectingthe
Com
panyand
itsbusinessandfinancial
perform
anceScintilla™
Sorrento™G
MA
B™
CA
RTN
K™
TNK
Therapeutics™and
theSorrento
logoare
trademarksow
nedby
SorrentoTherapeutics
IncA
llothertrademarksand
tradenam
esarethe
propertyoftheirrespective
owners
Logohttp://photosprnew
swire
com/prnh/20150105/167173LO
GO
SCIN
TILLA2
C
ompany
Overview
3
V
isionand
Mission
SCIN
TILLAO
urVision
Tosignificantly
advancetreatm
entsandoutcom
esforpainpatientsw
ithinnovative
nonopioid
painm
anagementsolutions
OurM
issionTo
developand
comm
ercializea
newgeneration
ofpainm
edicationstargetingm
oderateto
severepain
with
improved
efficacyand
reducedrisk
ofopioidside
effectsandabuse
potentialm
eetingsignificantunm
etpatientand
healthcaresystem
needs4
Scintilla
Overview
ScintillaPharm
aceuticalsisam
ajorityow
nedsubsidiary
ofSorrentoTherapeuticsA
nnouncedproposed
acquisitionofSem
nurPharmaceuticalsin
August2016
PortfolioofTw
oInvestigationalProductC
andidatesSpanningInterventional
PainSpectrum
SP102:firstnon
opioidepiduralsteroid
injectablepotentially
fortheSP
102treatm
entofLumbosacralradicularpain
Phase1
/2trialin
chronicback
paincom
pleteddosing
IND
filingtargetin
Q1
2017Projected
tobegin
PivotalPhase3
clinicaltrialsin2017
RTX
:anovelnon
opioidm
oleculetargeted
forthetreatm
entofintractablecancerpain
Phase1
/2clinicaltrialsconfirm
edactivity
consistentwith
animalm
odelsC
ancerPainIN
Dtargetearly
2017R
TXScheduled
tobegin
Phase2
clinicaltrialsin2017
Orphan
Drug
Designation
Semnur
Pharmaceuticals
SCIN
TILLASorrentoSorrento5
Investm
entHighlights
InnovativeN
onopioid
PainM
anagementPortfolio
LargeEstablished
yetUnderserved
TargetMarkets
Worldw
ideC
omm
ercialRightsto
AllProductC
andidatesStrong
ProprietaryPlatform
with
High
Barriersto
EntryEstablished
Reim
bursementA
ccessTw
oProductsw
ithB
lockbusterPotentialPoisedto
Replace
CurrentStandard
ofCare
Sorrento6
The
RightTeam
toD
eliverLongTerm
Value
25+yearsofresearch
&developm
entcorporateexpertise
Henry
JiPhD
Chairm
anM
anagementexperience
asfounderandexecutive
officerofnumerousbiotechnology
companies
CEO
SorrentoTherapeutics
JaisimShah
ChiefExecutive
Officer
25+yearsofpharm
aand
biotechcorporate
comm
ercialandproductdevelopm
entexpertiseC
EOSem
nurPharma;C
BO
Elevation;CB
OPD
LB
ioPharma;V
PB
ristolMyers
20+yearsofclinicaldevelopm
entatpharmasand
biotechsfocusedin
painand
Dm
itriLissinM
DC
NS
therapeuticareas
ChiefM
edicalOfficer
VP
Clinical
Xenoport;V
PC
linicalD
urectG
lenSato
JD20+
yearsoffinancialandlegalexpertise
atpublicbiotechsand
legalfirms
Legal/InterimC
hiefFinancialOfficerSupport
PartnerC
ooley;CFO
andG
CExelixis&
PDL
BioPharm
aSuketu
Desai
PhD20+
yearsofmanufacturing
/CM
Cdevelopm
entatpharma
/biotechC
hiefTechnologyO
fficerV
PM
anufacturing/C
MC
Allergan;V
PC
ephalon/Teva
20yearsofresearch
andoperationsexperience
atpharma
/biotechB
ryanJones
PhDV
PO
perationsV
PO
perationsSorrento;SeniorD
irectorA
mylin
7
The
PainM
arketisExpandingand
Underserved
Chronic
painaffects116
million
oralmostone
inthree
Am
ericans(1)C
oststheU
nitedStatesapproxim
ately$560
to$635
billionannually
(2)N
early30
million
patientssufferfromlow
erbackpain
inthe
US
(3)G
reaterthan80%
ofcancerpatientshaveuncontrolled
painduring
courseordisease
(4)Pain
likelya
majorreason
forhospitalizationsG
rowing
government
physicianand
patientbacklashagainstopioid
basedproducts
High
rateofaddiction
Myriad
ofserioussideeffects(respiratory
depressionG
I)N
otsuitableforlong
termuse
ScintillaFocuseson
aB
roadSegm
entofthePain
Market
PainA
cuteC
hronicC
hronicC
hronicN
onM
alignantmalignantPain
PainScintilla
Focus(1)A
SIPPN
IHSnarr
JaredR
isksB
enefitsand
Com
plicationsfromEpiduralSteroid
Injections:AC
aseR
eportA
AN
AJournalV
olume
75N
o3
June2007
http://ww
waana
com/new
sandjournal/documents/snarr183
188pdf;(2)Institute
ofMedicine
(3)DecisionsR
esourcesGroup
Chronic
Pain:Disease
Landscapeand
Forecast2016
(4)Datam
onitorDecem
ber2009
Strong
Pipelinew
ithB
reakthroughPotential
PositioningM
arketOpportunity
Developm
entMilestones
SP102
Potentiallythe
firstapprovedpreservative
andsurfactantfree
steroidindicated
forepiduraladministration
totreatlum
barradiculopathyN
ovelgelformulation
designedto
havea
prolongedresidency
time
atthesite
ofinjectionN
onparticulate
preservativeand
surfactantfreeProgressive
diseaseoften
leadingto
expensiveback
surgeryC
urrenttherapiesprovidelim
itedpain
reliefandhave
potentialforserioussideeffects
No
currentlyapproved
injectableproductforepiduraladm
inistrationEstim
ated~10
million
epiduralsteroidadm
inistrationsperyearinU
Salone
(2)Potentiallysignificantreduction
inopioid
usePhase
1/2
trialinchronic
backpain
completed
dosingIN
Dfiling
targetinQ
12017
Projectedto
beginPivotalPhase
3clinicaltrialsin
2017R
TXN
ovelnonopioid
potentialfortreatmentofintractable
cancerpainPotentially
singleinjection
efficacyw
ithlongerterm
benefitPainR
eductionPotentially
significantreductionin
opioiduse
IncreaseQ
ualityofLife
particularlym
obilityM
orethan
80%ofcancerpatientsexperience
uncontrolledcancer
relatedpain
duringcourse
ofdiseaseC
hemotherapy
inducedneuropathic
painseen
inup
to/
ofpatientswith
acostofm
anagementof
$23
billion(1)Potentialtoreplace
currentcostlyinvasive
treatments(nerve
stimulation
intrathecalopioidsradiotherapy)C
anbe
usedin
additionto
opiatesPhase
1/2
clinicaltrialsconfirmed
activityconsistentw
ithanim
almodelsC
ancerPainIN
Dtargetearly
2017Scheduled
tobegin
Phase2
clinicaltrialsin2017
FilingforB
reakthroughdesignation
ifgrantedw
ouldallow
forrapidapprovalO
rphandrug
statusgrantedLem
aetal2011
NEJM
July3
2014Editorial:EpiduralG
lucocorticoidInjectionsin
Patientswith
LumbarSpinalStenosis;G
unnarBJ
Andersson
MD
PhD
9 1
2
SignificantN
earTermC
linicalMilestone
TargetsforScintillaProgram
sM
ultipleclinicalm
ilestonesforallprogramsexpected
inthe
next12m
onthsM
idto
latestage
trialscomm
encingforSP
102&
RTX
programs
Inaddition
potentialcorporatedevelopm
entmilestonesm
ayalso
beachieved
with
potentialcollaborationsorlicensingin
exU
Sterritory
in2017
Program2016
20172018
Period2H
1H2H
1H2H
Phase1
/2Phase
3SP
102Phase
3StartPhase
3C
omplete
Results
IND
andPhase
1/2
RTX
Phase1
/2Phase
2R
esultsStart10
SP
102:InnovativeN
onopioid
InjectableforC
hronicPain
11
SP
102C
hronicPain
Opportunity
LargeG
rowing
Unsatisfied
Market
LowTechnical
Risk
ShortenedD
evelopment
ProgramV
eryPositive
Response
toN
ovelSP102
ProfileLarge
numberofinterventionalback
paintreatm
entproceduresinU
S(~10M
)N
ointerventionalprescription
painproductapproved
orinlate
stageforepiduraluse
inU
SFam
iliarityand
rapidacceptance
ofprescribingphysiciansw
ithSP
102treatm
entPositive
FDA
PIND
Meeting
in2014
Established505(b)(2)program
SP102
profiletargeted
toevolving
SUIC
omm
itteeand
FDA
Advisory
Com
mittee
consensusPotentiallyfirstFD
Aapproved
productforthisindicationN
olong
termsystem
ictox
ordosefinding
studiesrequiredPreclinicaland
Phase1
/2clinicaldata
supporthigherprobabilityofsuccess
Scheduledto
beginPhase
3clinicaltrialsin
early2017;very
stronginvestigatorinterest
Physiciansseean
FDA
approvedparticulate
andpreservative
freeproductasnovel
which
couldpotentially
avoidgrow
ingliability
risk12
FD
APre
IND
Meeting
(2014)FD
Aexpressed
interestinsupporting
anESIproductw
ithgood
safetyprofile
FDA
focusonlocaltox;did
notexpressconcernsonsystem
ictox
with
APIand
excipientFDA
stressedm
arketedinjectable
steroidsarenotapproved
forepiduralusebutare
usedextensively
andpatientsare
treatedw
ith3
6injections/year;they
areaw
areofthe
safetyissuesassociated
with
steroidparticulatesand
preservativescausingrare
neurologicdeficitsPotentialforreducing
sizeofsafety
databaseforSP
102isdependenton
toxstudy
andcom
parativePK
studyoutcom
eand
1stPhase3
clinicaltrialresultsA
greeto
505(b)2application
Need
bridging(com
parativebioavailability)study
toreference
drugPediatric
StudyPlan
neededpriorto
ND
Asubm
issionSafety
FollowU
p“6
month
followup
safetyevaluation
evaluationofany
safetyissuesidentified
inthe
nonclinicalstudiesand
evaluationofsafety
issuesthatwere
thesubjectofFD
A’sD
rugSafety
Com
munication
regardingepiduralsteroid
injections”Prim
aryendpointat4
weeks
secondaryat12
weeks
FungalM
eningitisOutbreak
(201214)
New
sweek
0424
2016TH
EK
ILLERPH
AR
MA
CY
INSID
EA
MED
ICA
LM
ASS
MU
RD
ERC
ASE
Meningitisoutbreak
causedby
compounding
pharmacy
producedsteroidsforspiduralinjections
contaminated
with
Aspergillusfum
igatusInfected
more
than800
peopleacross20
states>60
ofwhom
diedB
yK
urtEichenwald
/April16
2015http://w
ww
newsw
eekcom
/2015/04/24/insideone
mostm
urderouscorporate
crimes
ushistory
322665htm
l#V
TLgIv4aBsIm
ailto14
EpiduralSteroid
Injections(ESIs)Are
One
oftheM
ostCom
mon
MedicalProceduresin
theU
SC
omm
onSurgicalProceduresin
Millions(1)
EpiduralSteroidInjections10
000A
llcardiacinterventions7
500O
perationsoftheG
Isystem6
000K
neearthroscopies4
000C
ataractprocedures3600
Breastbiopsies1
6000
2500
5000
7500
10000
CD
CH
CU
PScintilla
dataon
filehttp://updatespain
topicsorg/2012/01/epiduralsteroid
injectionshtml
Accessed:A
SIPPC
onference2012
Manchikanti
Medicare
SlidesM
edicareO
verallESIInjectionV
olume
(2)K
eyPoints
Epiduralsteroidinjectionsare
oneoption
torelieve
painassociated
with
LumbarR
adiculopathyThe
Medicare
marketforthistreatm
entoptionisexpanding
rapidlyw
itha
CA
GR
of95%
ESIsoverallhaveincreased
130%per100
000M
edicarebeneficiariesLum
bar/SacralTransformationalESIshave
increased665%
per100000
Medicare
beneficiarieshighestofany
category15
SP
102M
arketOverview
LumbarR
adiculopathyisa
subtypeofLow
Back
Paincharacterized
bydebilitating
painusually
requiringm
edicalinterventionSpinalStenosisLarge
patientpopulationFrequentR
adiculopathyFrequently
treatedw
ithESIs
Degenerative
Disc
Disease
LumbosacralSpondylolisthesis
OccasionalR
adicularSmallerpatientR
adiculopathypopulation
PainSurgically
Surgicallytreated
treatedLum
barDisc
Herniation
FrequentRadiculopathy
Surgeryand
ESIarecom
mon
LumbosacralR
adicularPainM
arketTrendsSignificantIndication
Overlap
SpinalStenosiscanbe
causedby
aherniated
orbulgingdisc
Disc
degenerationcan
leadto
spondylolisthesisanddisc
herniationInconsistentD
iagnosesIm
agingscan
isrequiredto
determine
thespecific
causeofLum
barRadiculopathy
Some
physicianswilladm
inisterESIwithoutim
agingscan
Undefined
PatientPopulationA
geand
occupationare
majorfactors
Patientabilityand
willingnessto
undergosurgery
oftendeterm
inestypeoftherapy
Epidemiology
Chronic
Pain:116
million
Am
ericans(ASIPP)
Patientswith
LowB
ackPain:
27%orabout30
million
(NIH
)Lum
barRadiculopathy:
Com
mon
conditionw
ithan
estimated
US
prevalenceof~13%
40%*
IfSP102
were
ableto
captureeven
asm
allshareofthislarge
market
therevenue
potentialwould
besignificant
Source:ASIPP
NIH
SnarrJared
Risks
Benefits
andC
omplicationsfrom
EpiduralSteroidInjections:A
Case
Report
AA
NA
JournalVolum
e75
No
3June
2007http://w
ww
aanacom
/newsandjournal/docum
ents/snarr183188
pdf*Stafford
MA
PengP
HillD
ASciatica:a
reviewofhistory
epidemiology
pathogenesisand
therole
ofepiduralsteroidinjection
inm
anagement
BrJA
naesth2007;99(4):461
47316
A
FocusonN
onnarcotic
PainM
anagementW
illDrive
theG
rowth
ofEpiduralSteroidInjections
Prescriptionopioid
abuseisatepidem
icproportionsin
theU
S1
CurrentProblem
Additionally
researchshow
sthatopioidsdonotprovide
clinicallym
eaningfulpainreliefin
patientswith
lowback
pain2M
ultimodalanalgesia
istheuse
oftwo
ormore
analgesicagentsortechniquesto
improve
painM
ultiModalPain
managem
entwhile
minim
izingrisk
ofadverseevents
Multiple
medicalorganizationsrecom
mend
multim
odalanalgesiaforchronic
painm
anagementincluding
Managem
entthe
Am
ericanSociety
ofAnesthesiologists
Am
ericanSociety
ofRegionalA
nesthesia&
theA
merican
Academ
yofO
rthopedicSurgeons
PotentialforThe
SP102
clinicalprogramisintended
todem
onstrateitsutility
asakey
adjuncttreatmentforlum
barSP
102radiculopathy
andpotentialasa
newpain
managem
entstandard“C
onsultantsA
SAm
embersand
ASR
Am
embersstrongly
agreethatepiduralsteroid
injectionswith
orwithoutlocalanestheticsshould
beused
forradicularpainorradiculopathy”
Am
ericanSociety
ofAnesthesiology
PracticeG
uidelinesforChronic
PainM
anagement3
1C
enterforDisease
Controland
PreventionIncreasesin
Drug
andO
pioidO
verdoseD
eaths20020014
MM
WR
2015;64;15
2Efficacy
Tolerabilityand
Dose
EffectsofOpioid
AnalgesicsforLow
Back
PainJA
MA
InternalMedicine
2016Jul1;176
3Practice
GuidelinesforC
hronicPain
Managem
entA
nesthesiology2010;112:N
o4
Apr2010
1717
C
hronicB
ackPain:
SteroidInjectionsB
oldedW
arningsLessU
sedC
urrentlyU
sedD
rugForm
ulationPotentialIssues
Thesafety
andeffectivenessofepiduraladm
inistrationM
ethylprednisoloneacetateofcorticosteroidshave
notbeenestablished
andD
epomedrol
•Containspolyethylene
glycol+benzylcorticosteroidsare
notapprovedforthisuse
alcohol•Benzylalcoholneurotoxicity
Thesafety
andeffectivenessofepiduraladm
inistrationK
enalog•Triam
cinoloneacetonideofcorticosteroidshave
notbeenestablished
andC
ontainsbenzylalcoholcorticosteroidsarenotapproved
forthisuse•N
eurotoxicityofbenzylalcohol
Betam
ethasoneacetate
50%&
Thesafety
andeffectivenessofepiduraladm
inistrationSoluspan
Betam
ethasonesodium
phosphate50%
ofcorticosteroidshavenotbeen
establishedand
Containsbenzalkonium
chloride&
corticosteroidsarenotapproved
forthisuseED
TA•N
eurotoxicityofbenzalkonium
chloride&
EDTA
Thesafety
andeffectivenessofepiduraladm
inistrationD
examethasone
sodiumphosphateofcorticosteroidshave
notbeenestablished
andD
ecadron•B
enzylalcoholcorticosteroidsarenotapproved
forthisuse•B
enzylalcoholneurotoxicityA
llmarketed
productshavebolded
labelwarnings:“seriousneurologic
eventssom
eresulting
indeath
havebeen
reportedw
ithepiduralinjection”
andthatthe
“safetyand
effectivenessofepiduraladministration
ofcorticosteroidshave
notbeenestablished”
AllcurrentESIproductshave
preservativesandsurfactants
SP
102D
ifferentiatedProductProfile
&Positioning
Depo
Medrol
SP102
KenalogC
elestoneIm
portantTreatmentA
ttributes(m
ethylprednisD
examethasone
(Potential)(triam
cinolone)(betamethasone)
olone)FD
Aapproved
forlumbosacral
radicularpainC
linicaldatadem
onstratingsafety
andefficacyFastonsetofeffectin
LRw
ithless
spreadD
urationofefficacy
Reduction
indisability
inLR
SaferrepeatinjectionsLow
erriskofneurologic
AEs
Novelform
ulationw
ithprolonged
residencytim
eatinjection
siteN
oSurfactants
No
PreservativesN
oParticulates
PrefilledSyringe
SP
102C
linicalUtility
ofProductConceptisC
onsistentWith
PainSpecialistsN
eedsPhysiciansreacted
favorablyto
theSP
102productprofile
givingthe
steroidan
averagescore
of62
ona
favorabilityscale
of17
OverallR
eactionSteroid
A’sstated
durationofeffect
nonparticulate
formand
imm
ediateonsetofaction
were
them
ostoftencited
benefitsbyphysiciansO
na
favorabilityscale
of17
sevenphysiciansscored
itwith
a7
fourwith
a6
andfourw
ith5
Costw
ascitedoften
astheonly
negativeaspectofthe
profileA
n8
week
durationofeffectw
ouldincrease
favorabilityin
some
physiciansbutdecrease
favorabilityin
othersdueto
lessrevenuecaused
bylow
ervolume
ofinjections“It’snoveland
disruptiveThe
nonparticulate
andnon
preservativeaspectsare
exciting”A
nesthesiologistSteroid
A’sFavorability
N15
76
5432106
36
16
362
AnesthesiologistsPM
RSpecialistsO
therSpecialistsA
llSpecialistsSpecialty
Group
“Costm
aybe
highbutifelim
inatedneed
for2ndinjection
may
bew
ellworth
it”A
nesthesiologist20
Lifecycle
Managem
entSumm
aryO
pportunitiesPhysiciansindicated
thereispotentialopportunity
forspontaneoususeofSP
102in
theU
Soutside
oflumbarradiculopathy
which
couldrepresentan
additionalupsideof~50
200%*
LRProcedure
Volum
eLR
InjectionProcedure
Forecast55
MTotalProcedure
Volum
e8
316
5M
55
MN
onLR
ProcedureU
psideN
onLR
ProcedureV
olume
~50200%
*2
811
MA
dditionalUses
CarpelTunnel
TriggerPointInjectionsInjectionsforK
neeShoulders
Wrists
Ankles
JointsC
ervicalRadiculopathy
Knee
Arthritis
Hip
andK
neeR
eplacements
Com
plexR
egionalPainSyndrom
es(CR
PS)Lum
barSpinalStenosisA
cuteSpinalInjury
Discogenic
Pain*A
ssumessim
ilardegreeofutilization
foradditionalindications21
SP
102Toxicology
ProgramD
esignPlan
Identicalprogramsin
>100B
eagledogsand
MiniPigs(each
programcostsapproxim
ately$2m
)M
ultiplearm
GLP
studyusing
2routes:
SDEpiduralC
ontrol(saline)1m
LSD
EpiduralPlacebo0
4m
LSD
EpiduralPlacebo1
mL
SDEpiduralLow
dose:m
gin
04
mL
SDEpiduralM
eddose:
mg
in0
4m
LSD
EpiduralHigh
dose:m
gin
1m
LSD
Intrathecalcontrol(saline)SD
Intrathecallowdose
SDIntrathecalM
edD
oseSD
Intrathecalhighdose
Multidose
epiduralcontrol(oncea
week
for4w
eeks)M
ultidoseepiduralm
eddose
(oncea
week
for4w
eeks)22
SP
102PreclinicalStudy
Results
Hydrodynam
icStudy
Results
Addition
ofviscosityagentresultsin
adose
dependentprolongationofthe
residencytim
eofthe
productwithin
theepiduralspace
Com
mercialinjectable
steroidproducts(ie
Kenalog
Depo
Medroland
Decadron)have
anepiduralresidency
halflife
of~15m
inutesandhave
largespread
away
fromeffected
siteH
asanepiduralresidency
halflife
of>110m
inutesIsmore
localizedto
theinjection
sitethan
comm
ercialproductsspread
limited
toone
vertebraein
1sthourforgreaterlocaleffectvs67
with
comm
ercialproductsToxicology
StudiesCom
pletedin
Concurrence
with
FDA
PreIN
DM
eetingM
inutesN
odrug
relatedtoxicity
local(spinalarea)orsystemic
(includingbrain)
macroscopic
orhistopathologyafterboth
single&
repeat(oncea
week
for4Wregim
en)Histopathology
cleanin
bothroutine
andspecialstains
NO
AEL
ishighestdosetested
10m
g/anim
alsingledose
&2
mg
repeatdosePlacebo
armclean
nontoxic
bothroutesin
bothspecies
AdditionalV
asculartoxstudy
mentioned
byFD
Aalso
completed
successfullyD
irectinjectioninto
vertebralarteryofpigs
No
toxicity:vesselocclusionorm
acroscopicbrain
injuryasreported
with
Depo
Medrol
cleanhistopathology:no
brainorvascularinjury
23
SP
102C
linicalDevelopm
entPlanO
verviewC
orporateC
linicalStrategyisbased
ona
two
stageapproach
Stage1:IN
Dto
pivotalPhase3
decisionPhase
1/2
trialinchronic
backpain
completed
dosingA
dequateand
wellcontrolled
Phase3
efficacytrial#1
(USA
)O
penlabel
longterm
safetystudy
Phase3
openlabelsafety
studyStage
2:Com
mercialscale
updecision
throughN
DA
Adequate
andw
ellcontrolledefficacy
trial#2Pediatric
study(plan
torequestdeferralto
conductpostmarketing)
24
SP
102C
linicalDevelopm
ent:Ongoing
Phase1
/2A
nO
penlabel
Singlearm
Two
periodFixed
SequentialdoseStudy
toC
haracterizethe
Pharmacokinetics
Pharmacodynam
icsand
SafetyA
dministered
byEpiduralInjection
Com
paredw
ithD
examethasone
SodiumPhosphate
InjectionU
SP10
mg
Dexam
ethasoneA
dministered
byIV
Injectionin
Subjectswith
LumbarR
adiculopathyN
umberofpatients:12
subjectsdosedEpiduralinjection
procedureC
Tguidance
Avoid
dilution(no
contrastno
localanesthetic)C
ontaindrug
inepiduralspace
(interlaminarapproach
preferred)Interim
Analysiscom
pletedProjected
Enrollmentcom
pletionSeptem
ber2016LPLV
October2016
CSR
deliveredJanuary
201725
SP
102C
linicalDevelopm
ent:CL
EA
RPhase
3O
bjectivesC
orticosteroidLum
barEpiduralAnalgesia
forRadiculopathy
(CL
EA
R)
Evaluatethe
analgesiceffecton
legpain
following
asingle
transforaminalepiduralinjection
compared
toa
singlesham
injectionofplacebo
saline(0
9%sodium
chloride2
mL)in
subjectswith
unilaterallumbosacral
radiculopathyEvaluate
opioidconsum
ptionascom
paredto
placeboEvaluate
painat4
weeksand
degreeofdisability
asmeasured
bythe
Osw
estryD
isabilityIndex
(OD
I)Characterize
durationofanalgesic
effectEvaluatethe
safetyand
tolerabilityofm
ultipleepiduralinjectionsIndication
LumbosacralR
adicularPainSelection
Criteria
Clinicalsym
ptomsconsistentw
iththe
MR
Idiagnosisofnerverootcom
pressionsecondary
toa
pathologicdisc
condition(e
gherniated
discannulartearorfoream
inalstenosis)Leg
pain(5
9)presentforatleast2m
onthsandnotm
orethan
9m
onthsintensity
beingequalorw
orsethan
anyconcurrentlow
erbackpain
26
H
ighB
arrierstoEntry
ProtectSP102
IntellectualPropertyFiled
IPfiling
claiming
novelformulations(viscosity
dissolutionprofile
stabilityresidency
time)and
method
oftreatingU
SA
pplicationN
o14/162
625(“Pharm
aceuticalFormulation”)filed
January23
2014(w
ithpriority
toU
SProvisionalA
pplicationN
o61/755723
(January23
2013)andN
o61/776617
(March
112013)
PublishedD
ecember4
2014U
SProvisionalA
pplicationN
o62/106
045(“Pharm
aceuticalFormulation”)filed
January21
2015B
arrierstoC
ompetition
GenericsU
seofspecific
excipientinform
ulation(notin
label)willrequire
determination
andrepeatlocaltox
studiesrequired
forincreasein
residencytim
efortargetindications
Longterm
Supplyand
Exclusivityagreem
entwith
keyexcipientpharm
aprovidercom
pletedPatentsclaim
ingproprietary
formulation
method
ofuse
possiblein
Orange
Book
FDA
requirementforexpensive
localtoxPK
efficacy/safetystudiesfornew
routeM
anufacturingTrade
secretssubstantialknow
howlearned
overpastfewyearsw
ithform
ulationsteroidsand
viscoussolutiongels
27
R
TX:Firstin
classTherapeuticfor
IntractablePain
28
ProductO
verview:R
TXU
ltrapotentTR
PV1
agonistselectivelytargeting
afferentneuronsinchronic
painstatesH
ighlyspecific:affectsonly
TRPV
1expressing
nerves(Aand
Cfibers)R
TXvs
capsaicinM
echanismof
18B
illionScoville
unitsA
ction2
5000JalapenoD
orsalhorn=
>12
000tim
escapsaicinPharm
acologicalsequenceofeffectsin
vivoTR
PV1
receptoractivationdepolarization
apoptosisPotentially
permanentclinically
meaningfulanalgesia
regardlessoftypeofpain
(nociceptivevisceraland
neuropathic)EfficacyC
oncomitantreduction
inopiate
useIm
provementin
QoL
andfunction
SafetyA
lterationofheatsensation
inthe
targetedarea
No
effectonnorm
alacutepain
perception/sensation
ormuscle
functionTargeted
singleinjection
fortreatmentofintractable
painD
osingO
neinjection
Patientneednotdiscontinue
currentanalgesicm
edicationspriortoinjection
29
R
TXC
ancerPainEpidem
iologyO
verviewIntractable
cancerpainisa
subsetofcancerpainusually
requiringsignificantm
edicalinterventionR
TXispositioned
toreplace
costlyinterventionalpain
managem
entproceduresM
CancerPrevalence
MC
ancerPainM
Moderate
SeverePain
80%ofcancerpatientsexperience
moderate
tosevere
painduring
thecourse
oftheirdiseaseprogression
WH
Oestim
ates1020%
ofcancerpainpatientscannotbe
adequatelym
anagedEV
ENw
ithoptim
alopioidtherapy
(4)1
NIH
/NC
IandScintilla
internalresearchV
anden
Beuken
vanEverdingen
etal2007
Rossetal
20006and
ScintillaK
OL
marketresearch
Datam
onitorDecem
ber2009Estim
atedPatients2019
CancerPain18
million
Am
ericans(1)Patientswith
Pain53%
orabout10m
illion(2)M
oderateSevere
CancerPain
33%orabout3
1m
illion(3)
R
TXB
reaksNeurogenic
Inflamm
ationC
ycle:CancerPain
istheExpected
Regulatory
PathforFirstA
pprovalC
ancerPainTissue
InjuryInflam
mation
FocalNeuropathies
Trigeminal
PostHerpetic
Syndromes
Neurogenic
PhantomLim
bInflam
mation
Cycle
Chronic
RegionalPain
Organ
failurePain
AfferentN
erveA
ctivationC
ongestiveH
eartand
Psubstance
releasefailure
Chronic
PancreatitisC
ardiacA
pplicationC
ardiacsym
patheticafferentdenervation
attenuatescardiacrem
odelingand
improvescardiovasculardysfunction
inratsw
ithheartfailure
Heartisinnervated
byTR
PVR
1expressing
afferentnervesandthese
afferentnervesareessentialforthe
Source:(ZahnerMR
etal2003;Wang
HJ
etal2014)cardiogenic
sympatho
excitatoryreflex
duringm
yocardialischem
ia31
R
TXPO
C:C
anineB
oneC
ancerPainPreclinicalFindingsPerm
anentReduction
InPain
Measured
atWeek
14N
oSide
EffectsAsSeen
With
Opiates
OrH
ighD
oseN
SAID
SSuch
AsSedation
Constipation
OrN
auseam
m)100
toC
ouldSense
Norm
alPainSignals
(0V
AS
No
LossOfM
uscleFunction
FullyA
lertPosttreatment
Return
Of“N
ormal”
PersonalitiesR
eportedB
yO
wners
Source:Brow
netal
Anesthesiology
2005;103:10529
60n=18
Score50
40p=0
0001Pain
foralltime
points30Observational2010
n=18n=8
n=5n=4
02
610
14W
eeks32
FirstIn
Hum
anStudy
Confirm
sActivity
StudyO
verviewR
esultsFromFirstin
Hum
anStudies
APhase
1Study
oftheIntrathecalA
dministration
ofResiniferatoxin
forTreatingSevere
Refractory
PainA
ssociatedW
ithA
dvancedC
ancer(1)FIH
Open
labeldose
escalationsafety
studyPain
inrefractory
cancernotcontrolledby
standardtreatm
ent;>6/10N
RS
(severepain)
12patientshave
beenenrolled
todate
Placedon
clinicalholdby
FDA
inJune
2015afterN
IHPharm
acyfailed
aninspection
inM
ay(no
RTX
patientwasinvolved
orharmed)
Improved
painand
increasedactivity
with
reducedopioids
No
unexpectedtoxicities
MTD
notreachedw
ith1
mL
RTX
over2m
invia
infusionpum
p3
nonorpoorly
ambulatory
patientsableto
become
ambulatory
Reduced
Opiate
Usage
(1)ClinicalTrialsgov
NC
T00804154(IN
D64323)
Studyconducted
attheN
IH(N
eurologicalDivision)
Sw
itchto
EpiduralEpiduralinjection
intoornearthe
dorsalrootganglion(D
RG
)forunilateralordiffusepain
Intrathecalinjectioninto
thecerebrospinalfluid
space(C
SF)targetingboth
DR
Gsand
dorsalhornneurons
Note:G
reen=
SubstanceP
asmarkerofA
fferentnervesU
ntreatedIntrathecalEpidural
34
H
uman
ProgramStatus
GM
PA
PIcompleted
(3registration
batches)CM
CC
omm
ercialsupplychain
establishedInvestigationaldrug
productavailableM
utagenicitystudiescom
pletedToxLum
barGLP
completed
Pathologyand
reportsongoingC
ancerPainIN
Dplanned
early2017
EpiduralMED
study2017
Clinical
Cardiovascular
Preclinical&M
iniPigstudy
atUC
LAongoing
Regulatory2nd
Hypertension
ModelatN
ebraskaongoing
PreIN
Dm
eetingw
ithC
ardioD
ivisionQ
12017
C
urrentTreatmentC
ostEstim
atedC
ostofPatientControlled
Morphine
Treatment*
Year
2000Today**Pum
pR
entalm
edicationsrefillcharges
andsupplies(PerD
ay)$154
$228PerM
onth$4
625$6845
6M
onthtreatm
ent$27
750$41070
Top5
ReasonsforU
nscheduledR
eadmission
toa
CancerC
enter(N
=1351)
Fever15%
Sepsis**
Assum
ing3%
increaseperyear11%
Uncontrolled
Pain8%
Dehydration
6%Pneum
onia5%
Adm
issionC
ostofUncontrolled
Pain$8
025$11877
TotalCostofU
ncontrolledPain
(6m
onthw
indoww
ithone
admission)
$35775
$52947
Source:MSA
InternalResearch
ofthefollow
ing:*
TheC
ostofCom
fort:EconomicsofPain
Managem
entinO
ncologyB
ettyFerrell
City
ofHope
ON
EPublication
2000V
ol1N
o9:56
61**
Assum
ing3%
increaseperyear
36
M
arketResearch(1)
InitialPricePoint
Ourevaluation
suggeststhatthem
arketwillsupporta
potentialpricerange
of$15000
$30000
forRTX
therapyforpain
managem
entinthe
terminal
intractablepain
cancerpatientB
asedon
Key
StudyFindings
Currentcoverage
ofcomparatortherapies(IT
druginfusions)ata
cost($60100K
infirstyear)thatispredictably
higherthanthisrange
forthetargeted
populationPayersare
notexpectedto
limitcoverage
oraccessforeffective
painm
anagementin
theterm
inalpopulationPayershave
supportedpalliative
careand
qualityoflife
improvem
entsinthispopulation
Payershavegiven
afavorable
impression
oftheearly
clinicalprofileand
resultswith
RTX
inthispopulation
Am
ajorityofpayersconsulted
havesuggested
thatarange
of$10$30K
iswithin
acoverage
“comfortzone”
consideringthe
costofITdrug
therapysurgicaland
neurolyticproceduraloptionsin
thispopulation(1)Study
approvedby
Scintillaand
preparedby
Alliance
LifeSciences
37
R
TXM
arketingO
pportunitiesO
pportunitiesInadequate
treatmentforcancerpatientsw
ithintractable
painInnovative
nonopioid
nonaddictive
analgesicthatw
orksbyselectively
killingthe
neuronsthatareresponsible
forcancerpainw
hileleaving
otherneuronsintactSuperiorefficacyand
safety(TB
D)profile
with
potentialcom
petitivecostadvantage
overopioidsW
ithlong
termuse
opioidsgraduallylose
theireffectivenessandpatientsneed
progressivelyhigherdosesto
getpainreliefbutatthe
costofahostofdebilitating
sideeffects
includingim
pairedconsciousness
nauseavom
itingand
constipationIn
additionforpatientsw
hodo
notgetrelieffromm
orphinethere
arenota
lotofoptions
SignificantN
earTermC
linicalMilestonesforScintilla
Programs
Multiple
clinicalmilestonesforallprogram
sexpectedin
thenext12
months
Mid
tolate
stagetrialscom
mencing
forSP102
&R
TXprogram
sIn
additionpotentialcorporate
developmentm
ilestonesmay
alsobe
achievedw
ithpotentialcollaborationsorlicensing
inex
US
territoryrelated
toSP
102and
RTX
in2017
Program2016
20172018Period
2H1H
2H1H
2HPhase
1/2
Phase3
SP102
Phase3
StartPhase3
Com
pleteR
esultsIN
Dand
Phase1
/2R
TXPhase
1/2
Phase2
StartResults
39
Financing
andU
seofProceeds
~$100M
PrivatePlacem
entofCom
mon
StockFund
clinicaltrialsandN
DA
submissions
Pipelineadditions
Key
hiresacrossdepartments
Com
merciallaunch
prepPaym
enttoshareholdersrelated
toSem
nuracquisitionG
eneralcorporatepurposes
Investm
entHighlights
InnovativeN
onopioid
PainM
anagementPortfolio
LargeEstablished
yetUnderserved
TargetMarkets
Worldw
ideC
omm
ercialRightsto
AllProductC
andidatesStrong
ProprietaryPlatform
with
High
Barriersto
EntryEstablished
Reim
bursementA
ccessTw
oProductsw
ithB
lockbusterPotentialPoisedto
Replace
CurrentStandard
ofCare
41