Developing Orphan Products for Patients with
`Rare Gastrointestinal and Endocrine Disorders
`
`August 2012
`
`nps
`
`pi1arn1:u:oLItic:Lls
`
`NPS EX. 2073
`CFAD V. NPS
`IPR2015-01093
`
`Page 1
`
`
`Rare Gastrointestinal and Endocrine Disorders
`
`August 2012
`
`nps
`
`pi1arn1:u:oLItic:Lls
`
`NPS EX. 2073
`CFAD V. NPS
`IPR2015-01093
`
`Page 1
`
`
`
`Safe harbor statement
`
`.5-'tateinents inade in this presentatinn, which are net histuiicai in nature, such as the tiining cf
`the pntentiai reguiatuiy suhinissiun, apprnlrai pincess and cuinineiriahltatiun at" the cuinpanys
`iate-stage iegistiatiun prc-gian'is, the cuinpanys expected fiiture cash tin ws ti'c-in the
`cuinpanys rnyaity—hased puitt'n;‘iu ut'pn:iducts and pruduct candidates, the cuinpanys iiiture
`upeiatiuns and the cninpanys 2u'I2 cash hurn guidance, cunstitute funuard-iuniring
`stateinents fur purposes at the sate harhur prn Irided hy the Frilrate Securities iitigatinn
`Hefunn Act at 1995. ihese stateinents are hased an the cuinpanyis current eirpectatinns and
`heiiefs and are suhject tn a nuinher ut'i'actni's and uncertainties that cuuid cause actuai
`iesuits tn diifer inatenaiiy frein these descrihed in the fnnuaid-inciting stateinents, such as
`the risk that we du nut receilre reguiatniy appnwai tn inarhet uur iate-stage registiatiun
`piugiains in a tiineijy inannei; er at aifif, the nsh that we iaii tn maintain nur eiiisting
`cuiiahniati'Iie reiatiunships reiated tn the cuinpany’s inyaity-hased puitfiniiu nt'pi'uducts and
`the risk that uur cash fin ws are iuwer than expected due tn increased expenses er in wer cash
`in-tinws finin appiicahie cniiahuiatinns, as weii as ether risir iactnis described in the
`cuinpanys pen'ndic tiiings with the i_i..5‘. Securities and Exchange Cuininissinn, inciuding its
`Annuai iiepnit en i-'eri:n Id-if and i-'crin Id-Qs. Ah’ iniclrinatiun in this presentatiidn is as at the
`date at‘ this piesentatinn and i'iF.5‘ undeitaires nu duty tn update this intnrinatinn.
`
`August 2012
`
`ups
`
`II Ir--uzn-Iluli
`
`Page 2
`
`Page 2
`
`
`
`
`
`NPS value proposition is defined by a near—commercial pipeline,
`significant royalty cash flows, and a strong operational position
`
`Near-commercial,
`orphan product
`candidates with strong
`market potential
`
`- Gattex® (teduglutide) in short bowel svndrome (BBS)
`
`- Expected NBA approval in 2012; EU marketing autJ1ori.=_ation apected 3Q12
`
`-
`
`l*~latpara"" (PTH 1-34) in hypoparathvroidism
`
`- Expected Ei|.i'-‘t filing in 2012 and approval in 2013
`
`- NPSPFEJD and NPSFFEJS (calcilytics): potential in ADHH, an ultra-orphan disorder
`
`- Rovalties on Sensipar®fMimpara® represent significant value
`
`‘valuable mvaltv-based
`portfolio expected to
`significantlv enhance
`cash flows
`
`- FY11 sales of ssosrr {up 13% vov) and vro 2:112 through arse $444M [up 1ss+. Yflf]
`
`- Partial monetized as l'lEIl'I-l'ECflU|'£ debt fi_l92l~'| at fi'&3l]_.-'12, 9% intere1h]|' first $32M per
`vear
`rovalbes withheld to repav debt w $50-El] + flcess paid to P5
`
`- Debt expected to be repaid in mid-2iZI15; MP5 to receive 1ElEI% of rovalties through 2013
`
`Strong operational
`position
`
`- Rovalties on Nucvntaifl and Revestiveifi‘ are unencumbered
`
`ro-fom1a cash and investments at lZIi:'i,!3l],i12; adequate cash to
`$135l*-“I1 in
`launch bot products
`
`Univ recourse debt is ~$1?l*-ll convertible note due in 2EI14
`
`Management team with proven track record of developing and launching
`orphan products
`
`Strategic outsourcing business model maximizes operational efficiencies
`
`1 25M pavment received from Amgen in Julv 21312
`
`3
`
`1 n 4 I - n - r . I - - n In.
`
`Page 3
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`Page 3
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`
`
`NPS' product pipeline is highlighted by two advancing registration
`
`programs and a potential product launch in 2012
`
`Productlproduct
`candidate
`
`.
`.
`Indication
`
`.
`.
`Preclinical
`
`P1
`
`P2
`
`P3
`
`Regulatory
`reviaw
`
`M:':Il'kE|Z-Ed
`
`|‘ilotes_!|:iaI'ti1er
`
`Garret
`
`NATPAHA
`
`|"i|P5P?El{'.|
`,.,F,5,,;,.,;.,5
`
`-EL
`
`Teduglutide
`
`Ewart tmwei sir-drama— ?;“F$fi1’EL‘§”a
`
`Hiriauiiarathirrnldsm o E'§fIfl:i’L"‘§fida
`
`ms-Hur j
`
`Pediatricfother —
`
`.
`wwr-gnu
`
`E‘gEl£:|.fT:.::da
`
`5E"5iF=fiH'Mlmpara
`
`:3;-;=;g;:;,;;:;;;;:,_:v fi;;“.,§'f“ “'””“ 5"‘
`
`REGPARA
`
`Prams-st
`
`Secondary
`hyperparamyroidism
`
`lwowa Halizlto
`Kirin {Asia}
`
`osneupurusis figffigfglfifida
`
`NUCYHTH
`
`Hflderatefseverfi Pal" JHHSSEH {U5}
`
`Reuesfive
`
`Short bowel sirndro me llli_.icon1ed,.FTali:eda
`
`Cinacalcet HCI
`
`Post renal transplant Amgen
`
`l-tonacaleret
`
`Bone disorders _ ESE ['-.-'I.I"ilH’}
`
`I
`
`*i|3iutosorria| dominant hypo-caloernia with hifp-eroaloiuna
`
`-4
`
`IIPS
`""""""'“"
`
`Page 4
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`Page 4
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`
`
`NPS is well—equipped to transition into a commercial company and
`
`deliver significant tialue—dritiers in 2012
`
`GATFEX in short bowel syndrome:
`
`Acceptance of NBA submission
`
`FDA Aidtiisoryr Committee meeting
`
`Fositito CHMP opinion
`
`-:'.'.'omn'iercia| and supply-chain readiness
`
`FDA a|:'+|Jm‘u'a| of GATTEK
`
`-:'.'.'omn'ieiicia| launch of GATFEK
`
`NATPARA in hypoparathyroidism:
`
`I-ldld pro-out meeting with FDA
`
`Report additional REPLACE data
`
`Submit U.5. ELA
`
`NPSPFQD and NPSPFQS (calcilytics):
`
`Define development strategyr
`
`Teduglutide in additional indications:
`
`Define development strategyi
`
`V’
`
`EH12
`
`9’
`
`EH12
`
`2H12
`
`2H12
`
`v”
`
`v"
`
`2H12
`
`21112
`
`21112
`
`Page 5
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`Page 5
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`
`
`GA1'l'EX® (teduglutide) in Short Bowel Syndrome
`
`Page 6
`
`
`
`SBS is a highly disabling disorder that is often managed with
`
`parenteral nutrition (PN) and intravenous (IV) fluids
`
`Typically occurs after erctensive intestinal
`reaction due to disease or injury
`
`Patients are on Pl‘~l,«"I‘r.l fluid for wii nights per
`week; 3 to 12 hours a day
`
`Remaining gastrointestinal tract unable to
`abmrb sufflcient nutrients andfor fluids on a
`conventional diet
`
`Can lead to serious life-threatening
`complications, including infections, blood
`clots, and liver damage
`
`Patients rrray have to rely on parenteral
`nutrition or Iv fluids to survive
`
`Patients are socially and personally impaired
`
`The largest obstacle is the need to be
`‘hooked-up’ to PM for rrrany hours per day
`
`Socially and personally constraining: diarrhea,
`frequent urination, difficulty sleeping and loss
`of functional independence
`
`Reduced 5-year survive|*
`
`3i] to save probability of survival depending
`on age at Phl initiation
`
`Goal of GKITEX therapy:
`
`of the remaining intestine
`
`improve the structural and functional integrity
`
`“P9
`
`Page 7
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`Page 7
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`
`
`GATTEX'ii4?=' (teduglutide) is a GLP—2 analog that could be a first—in—
`
`class treatment for adult SBS
`
`I GLP-2 is a 33-amine acicl enclogenaus pe ticle
`secreted from intestinal L—ce||s after mea ingestien
`
`- Physiological preperties ef GLP-2:
`-
`E:=r;pancls intestinal muccisa
`
`- Enhances nutrientalziscirpticin
`
`-
`
`Stimulates intestinal hlcncicl flciw
`
`-
`
`Increases intestinal barrier functicin
`
`I GATTEX (tedugluticle):
`
`raclaticin resistant reccimbinant human
`- DPP-IV cl
`GLP-2 aria cig
`
`- Differs frcim GLP-2 El|'I|‘:.l' by an N-tem1inus
`substituticin cif glycine fcir alanine in pcisiticin 2
`
`-
`
`Extencls half life frcim Ir‘ minutes tn 2 |'IflU|'5
`
`Cince-clailyr sulzicutanecius injecticin
`
`Patent exclusivity thrciugh April 2iJ2iJ ancl cirphan
`clesignaticin
`
`-
`
`-
`
`HIM“
`
`Haas”
`
`>
`
`Page 8
`
`Page 8
`
`
`
`STEPS Phase 3 registration study and STEPS 2 open—|abe|
`
`continuation study design
`
`-— STEPS: 24-week Phase 3 —- -S STEPS 2: 24-munth cipen-labe|—-
`regisiratican study
`cuntinuatian Study
`
`
`
`
`
`LPLU
`
`11113
`
`ups
`
`Page 9
`
`Page 9
`
`
`
`Phase 3 STEPS data presented at DDW 2011 show GATFEX
`
`reduces parenteral nutrition dependence in adult SBS patients
`
`.
`.
`.
`I Primarv endppint: 2[i% tp 1i]iII% reductipn bf
`PN,r'I‘v' frcum baseline at weeks 2i] 3: 24
`
`I 53% bf GA'|TE.‘nI patients achieved endpoint vs.
`30% for placebo [p=EI.iJi]2}
`
`I At week 24, GATFEX patients achieved a
`mean 4.4L (34%) reducticnn in weeklv PNIIV
`vcilume frcnm 13L baseline vs. 2.3L (13%) fcnr
`placebp (p<:EI.EIiII1)
`
`I Despite the significant reducticnn in weekly
`PN,r‘I’v' vculume, GATFEX patients maintained
`their nutriticunal status
`
`I Epdv weight was stable for GA1TE}{-treated
`patients
`
`I Liver functicin: statisticallv significant
`imprcuvement seen in ALT, A51", and bilirubin
`values in the GATFEX grcnup vs. placebo
`
`
`
`Responderlate
`
`UtersperWeek
`
`Hflflfa
`
`4D “In
`
`E|"r’n
`
`
`
`n=43
`
`n=-43
`
`*P="3'-’-3"-3'2
`
`r~'..:
`
`4_4|_=I===l=
`n~_-39
`
`**p-:fl_[_|-D1
`
`I ;_;A1-|-E;.;
`I H
`ha
`
`355
`
`Baseline PN,!I‘v" vblume:
`GA1TEl(:12.9L
`Placebo: 13.2L ups
`
`Page 10
`
`Page 10
`
`
`
`
`
`In the 24-week Phase 3 STEPS registratien study GATTEX
`significantly reduced PN/IV volume requirements
`
`
`
`PH/IVlitersperweek
`
`-1.5
`
`-1.33
`
`-2.09
`
`I GAT|'EK
`
`I Placebo
`
`__ Baseline vu|un1e:
`GATFEK: 12.ElL
`
`Placebo: 13.2L
`
`-2.29
`
`=-=
`
`in-=
`
`psaas
`
`psa.a1
`
`***
`
`psa.i:ii:i1
`
`Hfiiifiilfi
`
`-3.43
`
`:1: =i=_ :1:
`-3.33
`
`=1: it It
`
`-4.3?
`
`12
`
`1e
`
`20
`
`24
`
`Week
`
`11
`
`nps
`
`lllr-an-Itl-all
`
`Page 11
`
`Page 11
`
`
`
`Gattex achieved greater reductions in fluid composite effect versus
`
`placebo
`
`Change in urinary output, oral intake, and PNIW volume
`
`as
`
`Liters/week
`
`Page 12
`
`Page 12
`
`
`
`And 54% of GATTEX—treated patients achieved one or more
`
`clays eff PN/IV at week 24 of Phase 3 STEPS study
`
`14
`
`13
`
`Reduced days per week
`
`-12-En
`
`I"-Jr
`
`I:
`
`1D-
`III#ufpatients
`
`
`4
`
`3
`
`1 I 1
`
`1
`
`1
`
`ltnv.-:2
`
`2tn-:3
`
`3
`
`4
`
`IGATFEX: 2 1 clay = 21339 (54%)
`
`I Placebo: 2 1 clay = 9,139 (23%)
`
`F'=EI.EIlJ4?
`
`13
`
`IIIIIIIIIII l"‘I
`
`Page 13
`
`
`
`12-month data further supports the potential of GATTEX with some
`
`patients achieving independence from PN/IV
`
`3333333 33,33 333 3.3333
`
`E a3
`
`I STEPS 2 interim analysis for 34 patients
`who had received 12 monlhs of treatment
`
`- 91% of patients achieved a 211% to 1flD"!a
`reduction in PM volume
`
`-
`
`ll-“lean reduction in PNEW volume was 5.2
`liters per week or 4fl".ri~.- from pr'e-treatment
`baseline
`
`- 24% of patients (EH34) reduced their
`infusion davs per week lav three or more
`
`- As of May 2012, seven patients have
`achieved independence from PN/Iv while
`on Gattex in STEPS 2
`
`c: E?-
`
`
`
`a5
`
`a3
`
`Percentageofpatients
`
`EEE$E§afia-Se?
`
`“U ‘=3
`1*“ ‘=3
`.3333-333, 3 3 33.33 3 3333 3333333
`
`3
`
`Page 14
`
`Page 14
`
`
`
`NBA
`submitted
`
`Positive
`CHMP Vote
`
`EMA
`apprcwal
`
`NPS submitted its New Drug Application for GATFEX in 4Q11 and is
`
`preparing for a number of key potential events in 2012
`
`
`
`Adiijsiw
`:5 H _
`
`ll-Irlnla-lulu-Hull
`
`Page 15
`
`
`
`Gattex commercial launch strategy is based on 5 strategic
`
`objectives
`
`I Commercial leadership with extensive experience with orphan disorders
`
`I Commercial build-up gated by key milestones
`
`v’ Positive Phase 3 results
`
`v’ NDA acceptance
`
`I
`
`I
`
`Positive Advisory Committee
`
`NDA Approval
`
`Focus on patient identification
`
`Secure market access and reimbursement
`
`I
`
`I
`
`I Offer personalized patient services
`
`ups
`
`IIIl—lIl:I'IlHtll‘I
`
`Page 16
`
`Page 16
`
`
`
`Patient identification is key in the orphan drug space
`
`
`Gattesg‘
`
`I
`
`Prevalence versus reachable patients
`
`rerluuluriiieirliiiimririiriltree
`
`I Treatment flow from resection to home healthcare with PM/Iv’ fluids
`
`I
`
`Patient—sourcing tactics:
`
`Patients I sites enrolled in clinical development program
`
`Partnering with patient advocacv groups
`
`I MDRD, the Dlev Foundation, ASPEN, etc.
`
`Leveraging intemet and social media activities
`
`I
`
`5hortl::owe|supporLcom
`
`I Cinline forums
`
`Deplovment of ii-*lSLs
`
`I Networlcing with thought leaders and centers of excellence
`
`Potential collaboration with home infusion companies
`
`1?
`
`Page 17
`
`Page 17
`
`
`
`Access and reimbursement critical te a successful launch and
`
`sheuld net be underestimated
`
`f
`
`|EilIi
`
`a
`
`|Li|lflE rillllinri
`[
`
`"n are
`E"
`1
`H
`
`I Market access studies
`I
`13 pavers - 155 MM lives and 12 pavers - 113 MM lives
`I
`555 ,r‘ Gatterr: reimbursement prcrfile:
`
`I
`
`flrphan status - unmet medical need — scrlid clinical evidence
`
`I
`
`Price inelastic with hurdles tcr reimbursement
`
`I
`
`SBS patients’ reimbursement cc-verage
`
`I
`
`I
`
`55% crf 555 patients have ccrverage
`
`wfiflflfa ccrmmercial, ~2EI“.i'a Medicare, and ~2EI“.i'a Medicaid
`
`I
`
`Internal expertise
`
`I
`
`5uccessfi.r| track reccrrd crf securing access and reimbursement tr:r crrphan drugs
`
`I Dperaticrnal elements
`
`I Health eccrncrmics-based value |Ji'EI|J-I:r5itiEil'l readv at launch
`
`I Gatterr: launch with ccr-pav and ccr-insu rance suppcrrt
`
`I Gcr-tcr-market limited tcr specialtv pharmacv netvvcirk
`
`Page 18
`
`Page 18
`
`
`
`Patient HUB will offer personalized service te each Gattex patient
`
`I
`
`Persenalized service will accempanv each SBS patient threughout
`me Gattex Experience
`
`f
`
`|EflHfl|UllflE[lflHFlflllflIn]gnmm
`
`I The patient services HUB
`
`I Manned lav NPS care ccrcrrdinatcrrs
`
`I Ccrcrrclinate clinical services
`
`I Avcrid any additicrnal burden tn the prescrilzrers
`
`I
`
`Expected deliverables
`
`I Mitigate patients’ reimbursement challenges
`
`I Educate patients ahcrut Gatterr: characteristics and use
`
`I Support REM5 (if need be)
`
`Page 19
`
`Page 19
`
`
`
`Patient—centric commercial strategy will focus on burden—free
`access and care through a single NPS contact — Care Coordinator
`
`
`
`- Reimbursement senrioes
`
`HUB; back office support)
` Home health
`Dietitian]
`care nursing
`nutritionist
`
`_
`
`d
`
`______H
`
`Pharmacist
`
`{fin-call nurse
`
` _
`
`I _-
`E .'
`
`I
`
`»:
`
`.;!_
`
`T-.~I:I.;‘:i
`
`xv.’
`
`IIPS
`
`ll-Irlnla-lulu-Hull
`
`Page 20
`
`
`
`
`H ,_
`
`'
`
`;- -
`|-
`. I
`.---_,.rr-_.1'.''..I “
`.-_'.__'. u_
`.-.1"';.‘.
`r.
`.‘
`:'_.:_.-:._'__.'_.|__|L3'-§Hfi'5,];
`.‘._;i §|_:_;5-‘;_::_‘,-.-._?_
`
`Page 20
`
`
`
`Summary of GATFEX in SBS
`
`I
`
`I
`
`SES is a rare and chronic disorder with an estimated U.S. prevalence of ~10-15 thousand
`
`Patients are dependent on PN/Iv’ fluid for nutrient and fluid support
`
`I This supportive care significantlv impacts patients lives
`
`I Associated with serious and sometimes life-threatening complications
`
`I Comprehensive, patient—centric slrategv undenrvav
`
`I
`
`I
`
`I
`
`Limited competition
`
`Positive reimbursement and pricing outlook
`
`|‘~'larl<et is accessible with a relativelv small commercial infrastnrcture
`
`I
`
`GATTEX is a flrst-in-class treatment that address the underlying goal of SBS therapv —
`improving intestinal absorptive capacity and reducing dependence on PM/Iv’ fluids
`
`I
`
`I
`
`Significantlv reduces PN,r'I‘v' fluid volume dependence
`
`Significantly reduces the number of infusion davs per week
`
`I Achieves complete weaning off of PN,lI‘v' infusions for some patients
`
`I
`
`IP errzclusivitv through April 2lJ2i]
`
`Page 21
`
`Page 21
`
`
`
`NATPARA""" in Hypoparathyroiclism
`
`Page 22
`
`
`
`When the parathyroid (PTH) gland cannot produce PTH normallv,
`
`homeostasis of calcium and phosphate cannot be achieved
`
`I There are multiple dimensions of and
`consequences to hvpoparathvroidism
`
`I
`
`Hvpocalcemia
`
`I Hvperphosphatemia
`
`I
`
`I
`
`Renal wasting of calcium which can caum
`hvpercalciuria
`
`Bone, muscular, nerve, and lcziclnev complications
`
`I No approvecl PTH replacement therapy
`
`I
`
`Current approaches aim to reduce severity of
`svmptoms by Iaising semm calcium levels with
`calcium and active vitamin D supplementation
`
`I Active vitamin II} and calcium supplementation can
`worsen complications
`
`I
`
`Hypercalciuria
`
`I Hvperphosphatemia
`
`I
`
`In'eversil:ile complications of the kidnev, eve, heart,
`and tiiain
`
`Page 23
`
`Page 23
`
`
`
`Hypoparathyroiclism complications range from mild or moderate
`
`symptoms to irreversible complications
`
`+-
`
`Symptoms and complications:
`-
`Paresthesias (tingling, numbness}
`- Carpopeoal spasm (violent, painful
`contraction of the muscles in the hanoffeet)
`
`-
`
`'
`
`Tetany (involuntary muscle
`contractionfo-amping)
`
`5EiELiFE
`
`-
`Bradycaroia
`- Hypercalciuria (excessive urinary calcium
`eacrefion)
`_
`_
`Hyperwrnlnerallzeo bone
`‘iiiitam in D intoaicafion
`
`-
`-
`
`-
`
`Irreversible complications can include:
`
`-
`
`-
`
`-
`
`Kidney stona
`
`Encl-stage renal disease
`
`CarcIio~.iaso.Ilar cliseases
`
`_
`
`-
`-
`-
`-
`Calflum depflslts In the mam
`' Cataracts
`
`Hl’P”Par3|_';'!Tl’rF'idi5m ‘:|’f"9”_iti‘""E and
`FEW: Etr": camp ":at'fln5
`
`441%
`
`3flofn
`
`‘E
`-4%
`in 20%
`3:
`fa
`
`1o=a.
`
`9% ‘
`
`Cognitive Nonspecific Neurotic
`Impairment Psychiatric Symptoms
`Symptoms
`
`Pychosis
`
`“=53
`
`ht
`
`* ww-u.I.ma
`
` :
`Faaxa et al,
`" Psg.rcl'iia1:ric Aspects of Farathtrroid Dis-ewe “ Ps1.r-.-'J'ii:-sonwatu -ID:-E-, November-December 1999
`
`-'riIc.-co
`
`Eq
`
`.......a........
`
`Page 24
`
`Page 24
`
`
`
`New research presented at ENDO 2012 undersceres high burden ef
`
`disease for hvpbparathyreidism
`
`I
`
`Investigaters frem Mass General and Brigham and ’v'v‘emen's Hespital presented “Leng-
`rerm fefferreup efpaais-Hts mas hyereparaafiymrdrsm - a ceherf studj/’ by Mitchell et al.
`
`- Detailed chart revievvs crf 120 patients (with ebservatien peried ef T vears (IQR 4-14))
`
`-
`
`?3'%- were female, mean 52 vears (range 2-BF), fiEr% pest-surgical
`
`- Renal effects:
`
`-
`
`33% [2iJ,!53] had a 24-heur urine calcium level 2 3i1Ii]rrrg*
`
`- 31% [1Ir',!54] had renal calcificatiens crr icrnes
`
`- 41% had eGFR -:EuEI mL_r‘min,i1.}'3n'F
`
`-
`
`2% required renal transplantaticrn
`
`52% (16331) had basal ganglia calcificatiens
`
`3% (1l],i12lII) had at least ene hvpecalcemic seizure
`
`33% were evaluated in an emergency department er hespitalized fer hypeparathvreidism
`cemplicatiens, including ?% in the last vear crf ebservatien
`
`-
`
`-
`
`-
`
`I Cenclusien: data show that hvpoparathvreidism and its treatment carrv an unexpectedly
`high burden ef disease
`
`“l'l-::Irr'r'ra| urine Ea 'I2JtCr*ET.I-Ell‘! 5'EI-3EI'EI
`
`r'r'rg,r'2*1- |'rr
`
`I
`
`r-lete: Fer r-latrerral l-’.r:|r'rr:v Feurrdatrerr. 1:Ire t3FRs assecrated wrtlr the stages cat clrrerrrc k.iClI'l'E'g." drsease are as fellewsz stage L er=r'.-:a:r, stage 2 EFF‘. fit:I-
`as, stage 3 am an-as, stage+1--EFF‘. Lses, stage seat-:L5.
`
`25
`
`n S
`
`Page 25
`
`Page 25
`
`
`
`There is a disconnect between patients and physicians about
`
`hvpoparathyroidism...
`
`I
`
`I
`
`I
`
`-
`
`“'It takes a long time to get a
`diagnosis and is hard to centre!”
`
`“'I go from physician to phvsician in
`search of a knowledgeable about
`treating mv disorder”
`
`“Taking multiple pills per day and
`being a calcium roller ooaster is a
`major burden”
`
`“I get brain fog and cognitive
`Efi’ec[5”'
`
`
`
`'
`
`I
`
`I
`
`I
`
`“H'fi?0i33|'3th‘fFflidi5m i5 fair‘? Silfjplfi
`ti? UB9 “DEE and E35?‘ '-'0 CW“-'|‘i3"
`
`“The disease is fairlv simple and
`slraig ht forvvar ” (under value liie
`disease)
`
`“'It's not a big deal to have to take
`calcium and vitamin D"
`
`“The cognitive issues are not a part
`of the disease” (cl isoount cognitive
`effects)
`
`Eli
`
`IIPS
`
`Page 26
`
`Page 26
`
`
`
`New patient research recently launched to better describe
`
`hypoparathyroidism burden of illness and patient challenges
`
`
`
`
`
`I cant“ be eibne eoyeiiore. I
`can T .oln"ii'e.
`Ir'?':ie i'oomi.'i'ig fear
`of (essay anizf Eli-‘El’? e seieure
`‘fling? “"3"” €m5'E”m"
`EffifiEgg
`
`m m:g51:’:Dr5:F$”;":hgp5
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`
`ieeili
`
`£5,
`
`'
`
`i'' H?’
`
`mmgg “,1,-m my Emyi}, fie
`,i:ii'.si»'.ii:i,-:',r iiiitilii me .-::i":i.ii".-:16-*e.r'.I,
`goiirig on weilirs,
`i".iiii'riii'ig—e.-ii’
`ofiiihgs we time to ob-
`beccime olnefieizgiir.-g.
`
`
`
`-- Amy, age 42
`
`Page 27
`
`I Recently launched PARADOX — Eafient
`attitudes and flesponses about Qisorcler
`Qutcomes efiplored
`
`-
`
`In partnership with The Hypoparathyroidism
`Association and the Mayo Clinic
`- Designed with input from physician thought
`leaders and patients
`
`.
`
`.
`
`.
`
`.
`
`.
`
`- Communicate the burden of illness to the
`
`broader community (physician, payer, patient,
`regulatory) in adyance of launch
`
`- Giying patients a yoice to help yalidate the
`physical, emotional, and quality-of-life
`challenges they experience eyeiy clay
`
`I Market research suggests patients are yery
`frustrated; no one understands their plight
`
`Page 27
`
`
`
`NATPARA could be a major advance in treating hvpoparathyroiclism
`
`The first and only bioengineered replica of
`the full-length endogenous H4-amino acid
`parathyroid hom1one
`
`The mechanism of action offers a more
`
`phvsiological outcome with the potential to
`reduce long-term complications
`
`Potential to reduce large fluctuations in their
`
`sen.Im calcium levels
`
`
`I
`
`I
`
`I
`
`I
`
`NATPARA will be available in multiple
`dosages to allow for personalized treatment
`
`I U.S. exclusivity:
`
`- Patent exclusivity through December
`2EI1H
`
`- Cirphan status with BLA market
`excl usivitv expected through 2l]25
`
`.I.
`
`
`
` W
`
`I
`
`I
`
`C - 12lI'I'I"lI'I.lI
`
`I.
`
`I
`
`an
`-_
`.
`
`Page 28
`
`Page 28
`
`
`
`- Ranclcimizecl, clculale-blincl, clcise escalating, placebci-ccintrcilled
`
`- F'n'mar*,r endpciint: maintain serum calcium while reducing
`calcium and vitamin II} analcig supp-len'ientatien by ED%
`
`- Dnce-c|ai|*,r SC injecticin administered with a pen
`
`NATPARAT“ Phase 3 registration study (REPLACE)
`
`
`‘ Insufficient reepcunee — titlate up
`
`
`
`
`
`Dptimize~fl-1{lweeks
`
`1llfl-lJn'icg
`1
`A1'I=-Anal5flIT-=9
`
`}'5n1cg
`
`Jltfiust I.a'ta1n.fir.I D and fifcfum
`
`AZPla
`A IPlacehc: 2
`CE
`|:I|agE|3.u |Ftacel::c: 1
`
`be 3
`
`Randemize
`[Bmelin-e}
`
`H=1a4
`
`Dnee
`titration
`
`Week 24
`4-week
`{endpoint} fellow-up
`
`IIPS
`
`IiIl—l‘I|II'I'I 'IIl"‘I
`
`Page 29
`
`Page 29
`
`
`
`24-week Phase 3 REPLACE study delivered statistically significant
`
`results; data presented at ENDO 2012
`
`I Primary endpoint: maintain serum calcium
`while reducing calcium and vitamin D analog
`supplementation by 5fl“r'a
`
`I Secondarv endpoint: percentage of patients
`who achieved independence from active
`vitamin D and decreased oral calcium to EEIEI
`
`mg or less
`
`I NATPARA was well tolerated and compliance
`was high
`
`. 93% {34],rgm flf M|qTpARJa,L_t|-gated pati.En1_-5
`completed the study: versus sass (35:44) of
`placebo-treated patients
`- Similar incidence of adverse events and serious
`adveri events for both groups
`
`Primarv endpoint: IT|' analysis
`
`50% -
`
`53%
`
`p{fl_mfl1
`
`
`
`Natpa
`“=9?
`
`Fla
`ho
`“$14
`
`“In of patients who achieved independence from
`supnlerrrentatrun at week 24
`43%
`
`%
`‘I: 43%
`
`-E
`E 1fl“»"n
`
`gar,
`
`D
`50 fir
`
`%
`:2 45%
`‘E
`g zoos
`
`:r E?
`
`Page 30
`
`Page 30
`
`
`
`hypeparathyreiclism replacement therapy
`
`
`
`
`NW‘ ?' mu:
`
`F'°*—='“* P,“-‘r 3
`
`Presentation ef
`aclclitienal data
`
`NATPARA is on track for a 2013 launch as the first approved
`
`Gem mereiel
`
`Ieuneh
`
`
`
`FDA Adviser?
`Panel
`
`
`
`FWE-BLA
`
`meeting with
`FD*
`
`Submit U5 ELA
`
`Petential FDA
`
`Approval
`
`ups
`
`lllr-an-Itl-all
`
`Page 31
`
`Page 31
`
`
`
`Market—sizing research estimates a U.S. prevalence of ~65,UOU
`
`insured patients
`
`I
`
`Studv used the IMS Life-Link Health Plan claims database, with 60 million unique U.S.
`patients and two epidemiologic approaches to improve validity
`
`I Captured the number of cases over a 12-month period {October 200?-September 2008)
`
`I
`
`Two methodologies based on claims data
`
`I
`
`I
`
`Prevalence-based approach calculated using hvpopaiathvroidism diagnosis and hvpocalcemia
`diagnosis with known hvpoparathvroidism indicators (neck surgerv, etc.)
`
`Incidence-based approach calculated bv counting the number of parathvroidectomv,
`thvroidectomv and neck dissection surgeries
`
`I Both methods in the studv arrived at a similar estimate of ~ElII,lZIlJlII to ES,l]l]l] insured patients
`with chronic adult hvpoparathvroidism
`
`I Research recentlv presented at American Association of Clinical Endocrinologists (AACE)
`and International Society for Pharmaooeconomics and Outcomes Research (ISPCJR)
`meetings
`
`I
`
`Epidemiological research provides directional support of market size however; market
`development and patient finding activities complement estimates from the “bottom up"
`
`Page 32
`
`Page 32
`
`
`
`Market research underscores the favorable market dynamics for
`
`hvpeparathyreidism
`
`chronic cases
`
`Survey ef 290 physicians
`
`- Treating the underlying cause ranked as the
`mcist impcirtant in terms of unmet needs and the
`greatest advantage cif NP5P55E’s profile
`
`- Over 5D% cif phvsicians interviewed indicated
`thev will be earlv-adapters
`
`- Mild, moderate, and severe cases were largely
`categerized based EH1 svmptcims
`
`-
`
`Langat expecmd use in n1cIdeIate to severe
`
`
`
`.‘fi:mrc'e'.' 351 El‘ third-party nrarfiret re'.s'ev-:rrch firnrfedby NF5"
`
`Page 33
`
`Page 33
`
`
`
`Summary of NATPARA in hvpoparathyroidism
`
`I Hvpoparathvroidism is a rare and chronic disorder with an estimated U.S. prevalence of
`30,000+ (60-65,000 insured)
`
`I There is no approved long-tenn PTH replacement therapy
`
`I Current treatments aim to manage symptoms through calcium and active vitamin D
`supplementation
`
`I Can exacerbate the condition and cause irreversible complications of the kidnevs, heart, brain,
`and eyes
`
`I
`
`I
`
`Patients are often highly symptomatic (tingling, parathesias, tetanv, seizure, etc.) and
`complications range from mild to moderate to irreversible complications, such as renal
`disease
`
`NATPARA could be the first approved long-tenn replacement therapy for
`hvpoparathvroidism
`
`I Dffers a more physiological treatment outcome
`
`I
`
`Significantly reduces calcium and active vitamin II} supplementation
`
`I Achieved complete weaning from supplementation for nearly half of patients
`
`I well tolerated with 93% of NATPARA-treated patients completing the REPLACE study
`
`Page 34
`
`Page 34
`
`
`
`Financials 8: Summary
`
`nps
`
`p|i1:|rm:u:I:LIt'Ic:Ll5
`
`Page 35
`
`
`
`Key financial information
`
`06/30/12
`(in millions)
`
`Pro ftwna cash and investments1
`
`Current liabilities?
`
`5.75% convertible debt due 2014 ($5.44 conversion)
`
`Non-recourse debt3
`
`Shares outstanding
`
`2012 cash burn guidaneel
`
`1Irn:iudes|n'ipai:|: of June 21211.1.‘ Amgen ,i' Senslpar transaction
`2 Exduda amounts associated current portion of non-recourse, Sensloar- and Preotact-secured non-recourse debt of $3M
`3 Long-ienn, Sensloar-secured non-recourse debt is $EI2.3|"«-'I
`
`ttttttttttttt I1
`
`Page 36
`
`
`
`In summary, the NPS investment offers multiple value drivers
`
`I
`
`Two near-commercial orphan product candidates with positive Phase 3 results and
`strong top— and bottom-line potential
`
`I GA'l'l'EX® expected to be approved in late 2E! 12 for the treatment of adult SBS
`
`I U.S. BLA submission for NATPARAT" in hvpopaiathvroidism on track for 2lII12
`
`I Valuable royalty-based portfolio
`
`I
`
`Sensiparfllflimpara royalties expected to significantly enhance cash flows in 2lJ12 through 2l]1B
`
`I
`
`Partially monetized as non-recourse debt [$92M at fi,:‘3-:Zl,i 12, 9% interest}; fiist $32M per year of royalties
`wimheld to repay debt with excess paid to NPS
`
`I Debt expected to be fullv repaid in mid-2£l15; NPS to receive 1Ell]% of royalties tJ1rough 2013
`
`I
`
`Strong operational position
`
`I
`
`I
`
`I
`
`$135M in pro-fon*na cash and investments at EIE.,’3lII,!12; adequate cash to launch both products
`
`Solid management team with a proven track record for delivering on stated objectives
`
`Strategic outsourcing business model maximizes operational efficiencies
`
`Page 37
`
`Page 37
`
`
`
`Developing Orphan Products for Patients with
`Rare Gastrointestinal and Endocrine Disorders
`
`August 2012
`
`Page 38
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