ISEE - Earnings call transcript Q3 2018

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Operator

Good day, and welcome to the Ophthotech Corporation Third Quarter 2018 Results Conference Call. Today's conference is being recorded. At this time, I like to turn the conference over to Kathy Galante. Please go ahead.

Kathy Galante

Good morning, and welcome to our third quarter 2018 earnings call. Mr. is, today Ophthotech Representing Glenn Sblendorio, Dr. and Executive Officer President; Chief Dr. David Chairman; Guyer, Executive Officer; Kourous Rezaei, Mr. Chief Medical Dave Carroll, Officer; Chief Financial Mr. Keith Westby, Chief Operating Officer; and Mr. of Head Kapoor, Business Vishal Development. and and be of of the respect and and timing, programs research to product the today, or in-license to development activities; of matters, Ophthotech's I research cash potential candidates; you regarding and clinical business strategy, plan; our expectations making of the progress financial opportunities. our benefits statements for acquisition will our other utility use of the development acquisition implementation future financial including development potential like and the to strategic X, statements and cash operational, any the expectations would regarding to trials us research balances; our and including potential results and our relating our remind therapy Inception collaborative we with of impact that projected gene safe purpose These statements XXXX. constitute the Act Reform Securities forward-looking under the of statements of Private Litigation the harbor for provision consummation statements risks and matters in subject forward-looking that including various the in-license risks trials; those for from and programs the to these financing and/or design results regulatory that additional research programs; acquisition cause materially any many and matters; actual These to could and conduct expressed to statements, are differ events initiation of relating cover and from availability and negotiation; development and expectation of transactions. clinical of data need for a August business. filings, report for detailed Form risk Risk section quarterly Factors our XXXX, on of our I the affecting the refer on in filed you and in description SEC to particular factors to XX-Q our X,

and be forward-looking as date. of any upon representing as subsequent of should views only as addition, statements our views our In not any relied represent today

obligation to the I update views to to call now at like Glenn. even do we the in future, elect some turn disclaim would may our do statements forward-looking change. point to these any so we While if over

Glenn Sblendorio

Kathy, everyone. good and morning, Thanks

appreciate We our this this lot on morning. we joining you to always as have call a report morning, and

announced past year, to retinal for therapeutics two diseases now developing expanded progress the two a only Further, current in for Today are new track therapeutic a XXXX, on the lines new for wet gene time orphan complement excited diversifying addition for have and with an the Stargardt with in inhibitor, programs, both dry also During Zimura significant in we programs, XXXX, and previously of an age-related for for AMD our programs, CX pipeline data we've gene our programs we pipeline to AMD announce made retinal indication. hit XXXX. therapy. in but treating with readout our and our indication, disease therapy therapeutic and

resulted expansion past over X, expands to therapy. I'm Today pipeline This in indications. retinal our with therapeutics with transactions Inception by the to our Our pleased age-related year backed therapeutic in treatment leading and business Ventures. that pipeline the Versant active Ophthotech of privately and acquired a held has development retinal for acquisition both you company gene diseases the outreach multiple of share

to Best excited also retinal gene to inherited disease, pipeline disease. are orphan for dystrophy, adeno-associated virus novel option an we gene known our have product an or with We therapy exclusive report therapy that a candidates also macular Best as expanded AAV vitelliform treat

for therapy the momentum orphan on created therapeutic diversified that a we year to retinal announcement diseases. both build over strategy gene past our programs have portfolio and today and age-related demonstrate Our the continue of executing to

shareholder in of thrilled the creating acquisition dry in that of treatment start-up form protein I degeneration. out to industry as of and atrophy, Before an age-related retinal to potentially gain sharing macular would scientific for we the is on we well the The including diseases. development from a advanced with focused like implicated retinal are geographic that acquisition Versant, X organization you company. companies rights HtrAX Inception merit, of point range this welcome an our details is known Versant of program to With the for a commercial worldwide age-related inhibitor diseases, an

pipeline. major with Versant new opportunities investor a reach, potentially will substantial also expand us exceptional to identify As global help Ventures additional our

on For intravitreal development formulation and lead administration, the for HtrAX followed conduct studies. we development are by and compounds enabling subject plan on planning Based the to our studies current IND for new to are XXXX. successful submit AMD atrophy we an with FDA inhibitor late secondary of program geographic to targeting to dry completion HtrAX our preclinical IND in US for development, initiate the

role share will terms acquisition a to value and this you in scientific of believe few a our the for we will but serve is believe course cure, treatment the in development the the in will drug a shareholders. acquisition opportunity believe We potential create we therapeutics gene compelling diseases moments. a therapy of offering Dave holds important rationale continue for to tremendous promise Of ophthalmic We with review patients. ophthalmic and that an also

disease gene institutions. agreement to signed gene by commercialize We therapy inherited acquire This product is both Pennsylvania series Florida. the and for candidate BESTX Best of therapy exclusive license disease to second impacting orphan rights AAV, eyes an mutation option for our novel gene arrangements treatment the from in the have of the the University disease and is a the Best an also of of typically patients. retinal develop with both caused University is of

disease. and IND- current the FDA addition we natural in to retinal to along The and plan with Florida, activities upon In by to Best XXXX. enabling commitment therapeutics further therapy and on in our submit with novel Best preclinical research develop strategy gene programs treatments studies an commence build HtrAX to and also timelines and agreement expect option conduct plan for [indiscernible] based IND diseases. the our to sponsor We exclusive to to history to our

is together Zimura past complimentary and of age-related macular pipeline gene age-related disease. been include of therapeutics recessive The year degeneration, treatment clinical has began XXXX, and this geographic very announcements, a productive autosomal to diversified market Stargardt In AMD, two and inhibitor involving large for us. secondary today's retinal that wet the dry therapy orphan atrophy trials to AMD; addition for In to diseases. CX our bring programs we

in randomized, safety are trial initial patients assessing data earlier available our We geographic who to anti-VEGF. IIa Phase double-masked, wet are the patient with completed Xb that recruiting our in completed degeneration, controlled, sham XXXX. and with expect trial Phase by dry XXXX. This treated randomized, controlled double-masked, top Zimura in be combination atrophy began and anti-VEGF report sham not initial of excited for our Xb this earlier Zimura clinical to top in previously the recessive efficacy this of year Also the trial Lucentis Zimura end trial with this open-label, initial Stargardt patients in of enrollment AMD been we for to assessing data clinical Phase in XXXX. of top-line line for quarter efficacy with disease agent and And of monotherapy in age-related We of data macular line April secondary year. patient the trail expect patients multi-center the we monotherapy expected safety autosomal dose-ranging, month fourth in in have the with ongoing patients enrollment clinical

disease and therapy XXXX, treatment the excited June for our pigmentosa, entered addition with disease. moving In in we gene our on to Congenital and adRP. announcement, global therapy a gene Stargardt type Best Amaurosus Today to novel innovative of onto orphan XX, diseases. treating efforts the for University AAV Leber we gene also are about gene and rhodopsin-mediated Pennsylvania rhodopsin-mediated are today's agreement Now license first indications of retinitis that exclusive pipeline therapy for include focused of University rights candidate therapy, retinal Florida develop our product commercialize the autosomal-dominant

with agreement also history candidate natural Center the a together entered facilitated in to plan product Innovation, preclinical of the conduct adRP. Pennsylvania by into well studies for Penn We with studies we as the University as sponsored rhodopsin-mediated which pursuant to research

initiated minigene novel in therapy the methods series agreements sponsored year, approach diseases. I/II Medical we utilize other XXXX. to to expect therapy clinical in trial gene initiate with a of delivery Also to We retinal Phase a target their School and earlier UMass research

for to develop a therapy As research. and needs the We unmet sponsored diverse market School diseases. license of an portfolio patents granted has to indications, our large any these treating specialists obtain retinal to research from option that result know-how each of and of to and diseases, of orphan exclusive of both Medical address this condition scientific that candidates believe for reinforces retinal agreement, commitment integrating therapeutics employing our and medical knowledge patients UMass product or a gene latest strategy patent applications the treatment

in opportunities retinal our our for in experience to strategy. business our will we forward, like diseases strategy be delivering the science-driven, sync value tangible over key with move As retina-focused company our to expertise new development we continue clinical are execution to as and Kourous. to that seek opportunities believe to outreach I'd on continue development the now are for creating our selective that drug and turn for this shareholders. and and call We

Kourous Rezaei

everyone. morning, good and Glenn, you, Thank

epithelial Genetic of inhibitor indication by AMD. unmet new foundation you to of correlation strategic our risk diversified HtrAX commercial indications. acquiring is spectrum scientific Molecular large Zimura demonstrated study portfolio between morphology baseline expression set both and certain HtrAX into a BESTX comparing and a Today HtrAX specific cells more program medical correlation retinal in reaching of inhibitor been XXXX one cells, market age-related AMD. pigment a genes of is of matrix. for eyes The findings to for option therapy targets evidence, therapeutic peptidase HtrAX inhibitor presence disruptions have of overexpression Vision are create and therefore [overexpression] linkage intervention in an patients The come treatment therapy function orphan leads is extracellular postmortem our responsible portfolio indications. perception demonstrated serine the light cells. A our and future AMD. which The the would also I detail regarding of of candidate we exclusive membrane HtrAX including goal or broadened inhibitor membrane, photoreceptors, in of normal and closer our studies necessary strong of and this product addition of and regulation of portfolio function the focused like to demonstrated for and macular HtrAX on of this indicate has the overexpression cells between to scientific diseases, and to HtrAX go step age-related HtrAX with age-related impacts inhibition the are cells program. strong These The involved leads RPE Bruch's a in with for for published HtrAX that has in with of degeneration, molecules need demise gene complement physiological which therapeutics program in of on and for RPE for death that is a our retinal HtrAX goal. in have and the in gene both these eyesight. the Our the viability. disease. based and CX the and alterations of The to the program assets

currently of inhibitor affinity which backup program specificity animal of models, as have in lead number new consists and Our engagement shown target high as compounds. in-vitro compounds, well multiple multiple a and HtrAX

program submit this this late is geographic to medical scientific in targeting dry As rationale unmet are by in in high has address atrophy an strong earlier, XXXX. retinal to diseases. the we the age-related AMD to program mentioned supported need IND believe We for secondary that potential and Glenn

attention Now I would our like therapy gene to to turn programs.

eye. minigene subretinal University the select with and therapy scientists of mutation. program rhodopsin-mediated the of consists various is treat University at the due program this initiated go portfolio have more AAV a to the we value the to at of program and would and recessive of collaborations Florida, Massachusetts gene The disease vector I and for Gene to strengthened Therapy intravitreal for disease CEPXXX we Center like for Medical leading portfolio the autosomal School. Horae and a autosomal-dominant Florida, University of Pennsylvania to initiated group regarding further AAV technologies by disease. our BestX is applications therapy in RPE gene comparing RP, the Best of detail ABCAX multifunctional FDA current that inherited protein therapy orphan and therapy portfolio. Stargardt comprehensive [indiscernible] a mutation Our LCA gene to due in gene Further channels and approved in ion or There regulates currently retinal no bestrophin, The the cells. from addition new option disease into AAV Penn EMA this Best

the of of for humans, and as and vision. [AD] Academy tested retinal was product necessary in BestX lesions demonstrated RPE lead In for model, mutations of in Proceedings it of this to in vision. wide results when spectrum their year cells of above, necessary central micro-detachments. the Journal. dystrophinopathy in The loss of BestX mentioned published the As life a disease serve the concept atrophy the February involve survival candidate bestrophinopathy collectively time diseases perceived over reversing [lesions] the anatomical the USA, which are and lead known in of proof this gene were National results photoreceptors the our The work of Science canine to that inherited are of of PNAS macula,

natural well activities parallel we In pre-clinical to by Penn pursuant facilitated mentioned patients which with for entered sponsored earlier, together history an study to can studies, Penn we a study. submit timelines, conduct In XXXX. with additional manufacturing sponsored IND-enabling also with to toxicology on for for plan with research Penn commence we research, Center master addition IND pre-clinical the as current Innovation as agreement by to as we disease. option Best into a agreement, exclusive the including Glenn a plan Based

our study Based and program, rhodopsin-mediated current other conducting to for programs, therapy RP commenced activities. are on gene additional clinical in and initiate expect IND-enabling studies trial history onto clinical natural X-II currently Phase we regulatory Moving XXXX. the timelines we a review autosomal-dominant have and

degenerative lead for is the to diseases the Stargardt mutations without ABCAX which disease, common or type caused vision gene inherited We LCAXX type CEPXXX any retinal by are by FDA treatments. disease severe mutations approved LCA available minigene currently most Amaurosus XX, of caused gene. is Stargardt EMA in the or both approach Congenital and loss that currently and are which orphan is evaluating LCAXX, the in and automosal therapy also recessive Leber

strategic stage Although for in Stargardt disease a has at our improvement early a very therapy development, for program we believe Zimura potentially disease. its of the minigene cycle large this

top Lucentis, different with our Lucentis inhibitor total now in wet AMD Zimura trial index. to IIa a line potential assess in of clinical naïve Zimura enrolled data We in expect trial anti-VEGF to combination with the year. dose-ranging, CX safety designed with in detect complement our a initial this patients Phase treatment clinical Turning program, we efficacy of This open-label, multicenter, of to signal patients by end from XX of trial. the is of this mg doses and agent, combination X.X

vision and Following the therapy of trial trial completion a will our to naïve received gained Phase in that than letters of to from to higher In This of previously generally a baseline, combination completed which AMD. what of mean three X-IIa six clinical with or AMD injection both this patients. with as X of trial gain combination mg, greater meaningful designed X.X lines sham-controlled visual trial, anti-VEGF monthly wet intravitreal of is of [P&L] wet data Zimura will trials is determine patients validate Zimura equal randomized, monotherapy. are anti-VEGF to mg be in seen was treatment clinical vision, XX% in we generally analyzed proceed accepted Lucentis which in gain than whether with XX.X

these were no concerns Although and identified. well-tolerated were safety

to AMD, assess XXX trial now recruitment we I to Carroll. present, XX. designed dry trial. would trial AMD will starter signal in be trial look completed Phase Patients continue This ongoing and Zimura I/IIa secondly this control large-scale disease. of in clinical upcoming of randomized Phase small are the in we safety and a patients data the month recessive our no potential II like over control XX forward this until clinical to atrophy sample and in into call We validate clinical Dave efficacy atrophy this efficacy month enroll patients trial. Dave? monitored continue of trial in year. this with Earlier our over study, We geographic the the in entering enrolled autosomal further Zimura Zimura although to turn wanted to is therapy months. was to Zimura geographic by end wet in IIb patient consistent Our mono to with before size group, currently finding Phase had a a to

David Carroll

everyone. provide a Thank our to morning like you morning review items our good of an estimate update press and highlight I'd our from Kourous also for this cash yearend inception recent few release to transaction. and

per quarter million $X.XX share income compared a net For loss the $XXX.X or of $X.XX $XX.X our totaled million XXXX. share per for or net QX, to

of a commercialization our QX per the $XXX.X As through $XX and to $X.XX to loss or for share totaled share Novartis of QX, licensing impact Year-to-date income the million agreement. Novartis the XXXX licensing million, agreement. XXXX of completion of our per net $X.XX reflected the XXXX net completion again compared

our year-end to Turning cash expected balance.

is and for our Zimura candidate and product gene are that business gene our transaction This best million balance gene expect our programs. million; also $XXX recently at based approximately the increase reflects balance one current of XX, expected million. our We're announced estimate increase million plan research collaborative $XXX September impact company's from less of our $XXX and which $XXX the continuation Our $XXX than estimate to increasing cash to therapy and our decrease Inception our on and an cash million range therapy was our $XX XXXX includes achieve therapy we of RHO-adRP and research year-end and between the the X program the development This other spending cash collaborative QX. cash guidance goals. now programs from million received prior from

candidates licensing the in company Of associated or not technologies estimates do expenditures pursue. from of course potential may acquisition resulting additional any that product additional or the these or any development reflect

small Versant common additional and outstanding molecule sellers, cash our HtrAX acquired our our approximately $X.X with shares Ophthotech of million in portfolio. Inception shares of million to helped expand issued the approximately which X its is inhibitors $X.X to opportunities to the Turning transaction. post stock We've transaction. of identifying has XX.X%

For million and certain clinical of sellers shares. marketing is clarity, receive $XX.X million approximately approximately milestones. The XX.X% also will $X.X approval

However, Ophthotech or call now for not infrastructure Glenn. your I’ll there turn you to time. Thank the personnel no are will from over assume and for. royalties, no inception near-term back milestones any

Glenn Sblendorio

Thank you Dave.

XXXX And in So plan to we and the dry portfolio with being in our leader build with We've gene gene met X. inherited goal data and have therapeutics continue added cash disease. to increased portfolio the report retinal XXXX. the savings AMD in deal and and we've summary, for in in third to guidance of we year been program retinal wet our the novel treating acquisition disease inception therapy and therapy our this target of through

today. also we So we up on a for I want We're only we you can thank remain note goals of up to the completed going overview open of to that an a our short but the website to like also Operator portfolio posting committed of I providing achieving slides for not long and today. for questions. listening that for and everybody to deals two the moment. the couple term focused will summaries in would term. our be the line for questions open

Operator

JPMorgan. Rama our we you. take first [Operator Thank Instructions] question from from And will Anupam

Unidentified Analyst

on stratifications then therapy in the Thanks how populations the to from turn dry may [what’s] could on Phase also Hey This here execution? Zimura IIb Are the Anupam an a to announcement for of one between filling being with differ Maybe indication? much is IND there should of programs second to And or you're the all Thank in progress. how questions GA thinking this morning BD maybe now studied is of consider so this in continue taking acquisition one AMD, more story about any of to and be guys. us on populations we factors the secondary this guys congratulations my guys. may studying? in are if this between not I morning. [indiscernible] X two you gene that terms candidate for addressable how broadly same sort ongoing expecting the you in file or inception which the

Kourous Rezaei

you Thank question. for the

look we Just and additional one as for additional at specific inhibitor an product gene extra at populations it's as to but for HtrAX a the as In atrophy versus target or using inhibition geographic it clarify is complement extra an get therapy either potentially one the therapeutic general it atrophy. for dry at in do monotherapy not we geographic asset combination of but inhibitor treatment as look not a therapy AMD.

Glenn Sblendorio

question business question. a very on development the second good So with

on a a plate I execute think have on. to we lot

very the in We a team, very presentation that is will team have lifeline. our we started as with and because continue said small good before I

I selective. think we'll be very

the the we the portfolio with You'll that with see effort of BD last was filled the that of is today the slide the year. to posted a objective opportunities website, over lot

very and execution So focused we'll on selective really continue. you. these programs be We'll of to Thank forward. all bring

Unidentified Analyst

that you guys. clear. Thank see I

Glenn Sblendorio

Bye. Bye.

Operator

we And Instructions] next [Operator Yigal hear Citigroup. with will from Nochomovitz

Yigal Nochomovitz

there. questions. taking Thank the Hi for you

that's study there? this you about study, three wet dosing? that's monthly reporting the it incentive or AMD few the an prn out quarter is I guess, in doing you What's followed of dosing talk months monthly the by can dosing a just On

Kourous Rezaei

dosing the a short. It's because for this there is no trial. prn Yes. is six very is duration one There no, clinical months

we in change we call past the the did have [indiscernible] in done the letters. mean vision trial indicated of had where the XX% and As a XX.X with

some at ranging can studies dose additional dose on whether but see done look improve we results. So to those we we have that further

one dose this what whether and validate outcome to purposes; ranging if So additional see trial before. can we do find we can we than serves a had previous two, better findings two

Yigal Nochomovitz

the time see no? in to able or are you course data label open this since And real

Kourous Rezaei

No.

Yigal Nochomovitz

Okay.

Kourous Rezaei

not. have We

Yigal Nochomovitz

that's a development for that no-go go-go But bar on Is trial? do – sort to of is letters your XX.X the control say your future you're for? the when or that what sort proper you looking of

Kourous Rezaei

bar to specific at not have go. point set this which a We for

is look just but we at trial, want have to not criteria Phase in is then do, each We you make have is decision – forward. arms remind IIa a see to a pre-specified to of again but control a this this compares whether done how arm. it what since and this is past have individually a a what how these worthwhile moving We the few it

that have this criteria a we We the and we with statistician at again a to world us then is a soft when to additional but consulted leading forward. make that want to have points help look decision all move end

Yigal Nochomovitz

All right.

– candidates clarify work Penn program and Okay. the product And have Best the same then Can two the Florida or this have just product and on does the since which two different clarify can not of licensed you you each groups of how have institution disease candidates the you are set just just sure. and is clear, whole they together, you not academic entirely academically collaborators candidate?

Kourous Rezaei

Best, done in that has the of The actually help at There collaborative of with technology tested of very with both has Sure. of and also the ones They at And all patients University institutions. also them. studies gene the that that have to a at are model. from Best degeneration the use understanding the vector also is animal are [gene] works history the way sort Pennsylvania. collaboration University animal Florida. the the history They and future animal this studies. which is Pennsylvania. technology Pennsylvania. patients’ is in Florida animals Basically Pennsylvania model model effort this who comes University doing therapy University we comes also unique have technology Florida. retinal University animal These University a from But us The University of center. at – our large the – only of of that's the natural is

Yigal Nochomovitz

makes mentioned I Is cadavers there you to That studies that do it's for expression effort something any high of expressions sense. – dry underway the a Okay. Got dry need you in [indiscernible] the on look expressed then this over it. selection. the patients. AMD patient to HtrAX basically highly think protecting about or And HtrAX don't AMD so patients, of is to in it's all going highly the for be thought postmortem

Kourous Rezaei

more if these to at I whether question. broad we AMD be looking decide wants we the going going a this generic be we do stage at wants looking also be are clinical broad think be look in later to are at and all in patients. at selective great It's more one a or more At base stage phases this based. Obviously we to when will trials patients analysis but to

Yigal Nochomovitz

Thank Got it. Okay. you.

Glenn Sblendorio

Thanks Yigal.

Operator

And further questions like over management for with no or would call the I additional turn any to remarks. to closing back

Glenn Sblendorio

Thank you.

done think I to are in we listening everybody would like today. for you. thank Thank I again and

Operator

you participation. call our conclude for does for That today. your Thank

You may now disconnect.

IVERIC bio (ISEE) 31 Oct 182018 Q3 Earnings call transcript