ISEE - Earnings call transcript Q2 2018

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Operator

Good day, and welcome to the Ophthotech Corporation Second Quarter 2018 Earnings Call. Today's call is being recorded. At this time, I would like to turn the call over to Kathy Galante. Please go ahead, ma'am.

Kathy Galante

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

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

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

Glenn Sblendorio

our you morning. Good you, appreciate Kathy. We everybody. call Thank joining this morning,

our diseases of Medical in Gene Therapy academic we X advance enter as patients Zimura, CX degeneration, this agent, with anti-VEGF We in XXXX with uncontrolled, end program the track IIa multicenter report from trial gene broaden by of we trials During clinical this trial from our with implementing followed in University with on of continued starting Horae our to trials, with line of field the enrollment the the top strategy the top Foundation, therapeutic of portfolio XXXX. the first Research our Phase Center. our growth our or with School diseases. year. Massachusetts and leading dose-ranging, factor the half excited for combination emerging institutions, anti-vascular XXXX of complement We're of data retinal in endothelial and Zimura have expect therapy by in programs our with XXXX and advance therapy. well collaborations who been completed that by in and wet to ongoing In Florida of not inhibitor, line various and University XXXX, significant Pennsylvania data made to University LUCENTIS, we age-related to securing retinal AMD, provide initial macular April continue multiple we progress, our open-label, factor Zimura previously XXXX, clinical today are treated Further, patient team as its

monotherapy atrophy of second geographic data the double-masked, of patient half Phase in this to autosomal This the in Stargardt in recruiting Phase patients trial data Zimura of sham-controlled in recessive We randomized, clinical line for with top patients Initial secondary be safety are to disease, AMD dry efficacy expected the expect initial is in began randomized, ongoing efficacy XXXX. for clinical double-masked, in safety currently XXXX. on and monotherapy track patients our top clinical sham-controlled assessing IIb IIb and trial earlier our recruitment and the assessing line with trial, year. Zimura available

and in an possibly entered X therapy of is commercialize candidate on large of intriguing regarding both adeno-associated of prolonged to Gene we market this potential to moving and for to has into approach autosomal-dominant diseases. and University emerge that has a gene gene treatment the to a University patients agreement combat is adRP, Pennsylvania We making, gene orphan potential orphan loss and rhodopsin-mediated class to and believe therapy the as an that begun the global number it both detailed the loss monogenic-inherited presents progressive retinal since of and leading retinitis We'll as for an retinal to more of a to effect administration. characterized rights vision product to for novel therapy Over retinal decades also durable, diseases, XXXX, blindness. of a scientific rationale. treatment in diseases gene therapy a information disease the AAV, June cure attractive by for vision or therapeutic physicians a Florida provide provide pigmentosa, severe treatment therapies develop with therapy. virus, treat In exclusive Now single promising license a option. new

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

before, series I methods gene approach to novel UMass As therapy to earlier their other utilize retinal mentioned year, School of target we Medical the diseases. research sponsored delivery and minigene with in a agreements initiated

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

development forward, we we the as be As in aggressive our business continue sync science-driven, retina-focused to strategy. will selective our that but new opportunities with continue to are move seek outreach company

technologies this execution collaborations with to drug company novel and create tangible uncover for and institutions on and companies. retinal through differentiating strategy We our are strategy and We our development will in experience clinical expertise key to candidates to the and for continue to our and leading academic on diseases product and value that believe build for opportunities shareholders. delivering this

top the ahead line combination look we we therapy to IIa to for in As trial data expect for report clinical AMD. XXXX, Phase our wet of remainder Zimura

to dry atrophy Phase and to recruitment turn into we diseases. to Kourous. And Second, portfolio the call IIb monotherapy secondary for potentially trial now opportunities enter AMD. expand finally, gene therapy both complete to our I'd over our the of further Zimura new clinical retinal in expect to geographic in like therapeutic

Kourous Rezaei

Thank and morning, you, Glenn, good everyone.

total LUCENTIS signal detect different recruited This at is trial. assess in Glenn a month by uncontrolled, As of treatment-naïve of with the open-label, multicenter clinical doses the mentioned, and Zimura our expect dose-ranging, IIa this a we initial safety line clinical year. with anti-VEGF milligram in in XX data this Phase combination to potential wet of Zimura patients X.X in efficacy We agent, trial trial top end LUCENTIS, the of combination from of X. AMD to with patients designed

to trial, Zimura anti-VEGF AMD. clinical a combination trial determine Following of proceed wet be randomized, to the in of analyzed, this data and therapy with will completion clinical sham-controlled whether we will

of accepted you XX% wet generally our for of the are anti-VEGF clinical I/IIa a that in and from what may tolerated meaningful X-month safety treatment-naïve As well Zimura monotherapy. combination greater the is Phase which as gain or milligram intravitreal equal AMD of than previously generally study, seen is X were gain X.X with of XX.X All in which patients lines group, high-dose of gained than doses trials mean concerns most recall completed LUCENTIS visual vision, in of received identified. baseline, to among injections Zimura visual letters with in patients both were higher

Our sample group. I/IIa Phase consistent had a a with trial control small size no study with

entering this to dry efficacy Zimura Phase to trial. XX We Zimura monitored This wet clinical on currently the complete findings in had a third trial was in this we of AMD designed XX. enrollment later our patients quarter secondary this in patients the in month large-scale, will until upcoming is of potentially, to monotherapy validate XXX Approximately geographic over are IIb randomized, ongoing be into continue assess is clinical data we to present, The year. signal these further trial. expect efficacy And recruitment trial geographic Although forward the track. look year. of atrophy an atrophy Patients controlled to months. before enrolled AMD be into and to to Zimura expected safety in

retinal orphan-inherited clinical IIb recessive enroll is our automosal patients an disease. which in Phase We Stargardt continue to trial disease, in Zimura

of enroll We clinical globally expect this in the patients interventional trial, date. to to trials Stargardt making in disease XXX largest this approximately one

In with into for and which of XXXX, current gene activities. treatment conduct with regulatory commence Center pigmentosa novel of a interference Moving on Research preclinical we license replacement product adeno-associated University global research Pennsylvania therapy Foundation the initiate to to into programs. our and June agreement, the rights commercialize endogenous entered Florida Penn In virus a and plan expect human have an for with down develop The RNA in natural autosomal-dominant pursuant and patients identified University on agreement of to Phase entered the in pigmentosa. and disease a Ophthotech clinical facilitated the also IND-enabling retinitis knock gene. rhodopsin-mediated and Rhodopsin-mediated highly master retinitis I/II trial we to construct is manner to parallel therapy exclusive candidate candidate short transgene autosomal-dominant gene target Penn rhodopsin we sponsored for and rhodopsin-mediated autosomal-dominant combines than a to adeno-associated in to pigmentosa transgene designed product novel a agreement, mutation-independent resistant orphan Based single hairpin plan additional XXX sponsored during vector. research rhodopsin a Ophthotech more the a Innovation, rhodopsin retinitis studies. rhodopsin-mediated by for in to Penn autosomal-dominant RNA history made a XXXX. efficient review, studies with mutations the timelines expressing pigmentosa retinitis monogenic a of

As with collaboration, part renowned scientists internationally of partnership retinal therapy work diseases. our in will gene we for orphan in

Artur and Lewin of Pennsylvania. William Cideciyan University Prof. University collaborate Hauswirth and with Beltran, Sam William will from from Jacobson Prof. of the Alfred and Florida the Aguirre Prof. We and Prof. Gustavo Prof. Prof.

Glenn therapy allows School Hermant earlier, As such scientists, us with step UMass with our Gao Guangping collaborate Dr. was high-caliber referenced as to gene collaboration our Dr. into research and Medical that first Khanna.

know, the adeno-associated indications. retinal Dr. technology is virus therapy. Dr. development novel family to pioneer giving a on for Khanna's research in Gao for orphan And minigene you focuses gene treatments of the As develop

vision generally The common eye. is Amaurosus are AAV address is minigene Leber of the larger strategy AAV into LCA to the exceeds vectors any disease by diseases replacement gene a of School of with of or for with impossible vehicle collaboration being approach degenerative agreement EMA-approved would that includes the size limited evaluate target strategy ABCAX but cargo. currently a caused our through Medical of FDA as mutations the AAV therapy, of Congenital is and diseases minigene the delivery gene our inherited interest which most packaging therapy in form the will or as orphan back functional Further, which retinal Stargardt treatments. that scope vectors delivery in to the mutations genetic available type a the UMass size smaller UMass LCAXX, LCAXX caused AAV novel otherwise gene for therapeutic and passage both gene. to We may the the their XX, disease, loss therapy is is focused the This capacity approach delivering indications. seeks on gene School Stargardt recessive where retinal Medical are CEPXXX still UMass by be to or type minigene orphan of developing at vectors. difficult a and gene developed without deliver lead vector. automosal that by conventional offer to of transgene The the AAV

therapy strategic for Zimura its we in lifecycle believe stage a early potentially very improvement for that Stargardt Although in this disease minigene is development, disease. program our

secondary and trials of allow forefront to in geographic is turn diseases. therapy dry gene University technology at in of data And both be School diseases over cutting-edge meantime, strategy this us would our look that the large retinal the field a AMD, and Florida atrophy Dave diverse wet Dave? the collaboration here using of Our to build portfolio with We our stay. market disease. eminent automosal forward to call Zimura indication believe the scientists In Carroll. the to recessive for Medical be from retinal to for and at orphan therapeutics. therapies gene will to AMD the Stargardt upcoming we is of UPenn, treatment treatment of now I and UMass

David Carroll

Kourous, also reaffirm I'd highlight to everyone. you, Thank items and our this cash good a from morning, guidance. morning press release our like and few of year-end

organizational quarter, $XX.X XXXX $XX.X of $X.XX the restructuring. our net share million a Year-to-date, million for loss again for program or XXXX, Fovista the compared million million XXXX share net the totaled organizational to as our of per share QX impacts totaled of compared or per loss to and III of XXXX Phase and $X.XX net our net $X.XX or the our loss clinical the Fovista clinical restructuring. of the discontinuation due For QX $XX.X per $XX.X a III reflected or loss to discontinuation program Phase $X.XX

cash our to Turning expected year-end balance.

RHO-adRP was based therapy $XXX balance our our research at and range collaborative $X product guidance million of cash cash our million decrease March candidate current XX $XXX cash will We Zimura, programs million XX. gene our which gene programs. includes balance million, reaffirm year-end a business and June Our between our therapy our continuation from expect plan, XXXX development for and on $XXX

Of your expenditures technologies acquisition in-licensing of potential company the additional Thank Glenn. I'll that do these call resulting time. pursue. now the to estimates reflect may you for turn from or additional any candidates, or over development product associated back any the course, not

Glenn Sblendorio

you, Dave. Thank

you second for operator. like thank XXXX phone the look of for and the in tell joining to morning, story. with to and continuing on half going lot engage to and forward a our have I'd investors questions, open this We up now everybody

Operator

Instructions]. our [Operator first Yigal take we'll question Nochomovitz from Citi. And with

Yigal Nochomovitz

on So therapy the question unsurprisingly. gene programs, my is

gene that advantage. recent FDA So manufacturing released. the that But the that's may game. because sure of the draft therapy an And obviously, guidance you're that you're aware the I'm you in give of early regarding recently

the into companies be plan I'd nonclinical guidance, cites FDA how the a [indiscernible] tox. also and cycle thoughts so want the that the scaleup they your long-term about that before initiate integrate curious full scale-up They manufacturing they with that on guidance a to reproductive development in well to turned toxicology as talked early planning it's hear And to to you're respect manufacturing safety development see developmental as clinicals. test start for assuming

guidance on curious around approaches make different be and how development have you're how it just overall coordinate and you early I'd that you in consistent, UFlorida, would So at which thoughts UMass the implementing can your very so may least across stage. UPenn,

Glenn Sblendorio

clinical thanks, finally that guidance Yigal. pleased at actually -- this. this we question. And early And way approaching with do are yes, FDA the that's we a the in we're providing And obviously programs Yes, And on is the programs. great clinical we're area. look

major issue. always course, Of CMC, consistency a is manufacturing,

expertise of through therapy building year, So And and side gene a we've the the side. and network a over the the is consultants importantly, environment. taken in lot. the only we then and think in manufacturers, manufacturers And a are And here we've guidance. I more and understand the methodologies number experienced regulatory we've well U.S. But these think internally learned way out since it been last we've on early side, regulatory of the CMC worldwide. changing approached I we're we've but expertise these interviewed experts not that

think of we're I aware it. So

support answers new that in that. even announced be your preclinical we're I hope leading we I And compliance scale think as that have with, in to the and with we experts would guidelines. that to question. yet, manufacturers anything be haven't sure stage, these working all up,

Yigal Nochomovitz

asked learn mean, I thought check. the think you in but -- I've just of Yes. cadence could And I'd others past, Are that now? any about have in on you there that the the satisfied wings with I I new are academic what this three collaborations? of sort the are about? the we that

Glenn Sblendorio

with we UFlorida, the we're that that the to UMass, We're talk able [indiscernible]. institutions time, UPenn, about clearly great happy really institutions. work all They're three

we've we'd new to two, to as one, potential would indicated, criteria science; continue the in we'd look, there's at great gene think applied be that that we programs to think good I the there's looked we if signal a to and could like -- that first us opportunity you thus all what we that for therapy that indication. if far, leading be we out, works And there's I have selected that, space.

as commercial So the we continue side. talk the very both aggressively company to side as the well academic folks or to on

us, for the to the game these has experience being been I creating think with organizations. academics a early good leading relationships by

continue to we answer, look. So

to We continue to talk folks.

actually right now with getting to quite opposed active calls. inbound that very I is outbound pleasing being as is we're think us what calls

a line So anything the I ourselves. bit the leaders to here I that create think you know on add And name for little allows starting if here. to Kourous, want we're of don't a front academic

Kourous Rezaei

Well, Glenn, said. you I agree with everything

look. to to major And regarding without also do any that continue the are stage needing be guidance to good changes. follow And to CMC. able a FDA we at We obviously we the make

Yigal Nochomovitz

Maybe I to are the have to academic avoid? are program? mean, trying of mean, criteria could different. you you ask And what a for that I a there are profile new new it need certain development must-haves collaboration? in what slightly absolutely do I

Glenn Sblendorio

we clear to third, very great therapy go or great Again, Yes, first the market, UPenn. that back. obviously indication an I'll foremost. with UFlorida, It's be important, demonstrated and think And question. clinic. a to the that a science, first path could a And I

transgenes. a with people clear larger we're great research science, in be very and with a important today may important. But programs has looking overcome hurdle very path UMass think that's the that third point Great I the great is to to work Early is here delivering clinic. doing for see. we that

Operator

Instructions]. [Operator

Glenn Sblendorio

We today and... you. listening appreciate Okay, operator. everybody Thank

Operator

take JPMorgan. question Anupam question We'll our come have in. Rama from We a with

Anupam Rama

Just the on is quick you us maybe it I position. the remind end how the position a for $XXX to But million. is year to with know sufficient stands? cash can currently one cash million $XXX this guidance long as

David Carroll

of assumptions, end So have based everything we clear can now terms that our on XXXX. see see, in the we we of right can ourselves to

As the look you months. for next for to XX GAAP, generally out you just have know

You'll and filing. through ourselves we XX-Q But clear see our XXXX that can into XXXX. see in

Glenn Sblendorio

enough consistently Anupam, turn. think card a comment, of to said pretty Dave's I'd I to is current we absolutely have add thing the right, the have to other programs he's bring we that that all capital

example, about 'XX dry and both in later includes So the dry Dave's also as talked the data a said we he in for program. in wet, 'XX well. the Qs, this data getting therapy what's commentary And again his guidance, for that's And gene for Stargardt for milestone. on then and as to in programs

example, to get the for to clinic. the program, adRP hopefully So it in

forward. So I there these move specific guideline. know it's the is programs But capital not to

Operator

no remarks. are to I telephone over Glenn, questions. turn additional back or now you there like conference further And would for the to closing

Glenn Sblendorio

Well, listening on everybody, and our BD look programs to continuing thank you, to update it. you today. for forward and appreciate activities, We

Operator

you conference. your This Thank today's for concludes participation.

disconnect. now may You

IVERIC bio (ISEE) 1 Aug 182018 Q2 Earnings call transcript