ELOX - Earnings call transcript Q3 2018 | Eloxx Pharmaceuticals, Inc.

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Operator

Good morning, everyone, and welcome to Eloxx Pharmaceuticals Third Quarter 2018 Earnings Webcast and Conference Call. Today’s call is being recorded. At this time, I would like turn the call over to Barbara Ryan, Eloxx’s Investor Relations Officer. Please begin.

Barbara Ryan

Thank you, Liz. to review you Quarter of Results Business you morning us XXXX thank and a for Eloxx Update. and all joining of this Financial Welcome, Pharmaceuticals Third

Executive Robert our Matthew Williams, Operating Goddeeris, and Weaver, Officer; Chief Scientist. Chairman are our Dr. our Chief morning Snow, Senior Officer; this Eloxx Pharmaceuticals; Principal me and Joining Dr. Chief Business Officer David Chief of Greg Officer; Ward, Greg Financial

call Litigation Eloxx on be any Factors recent these may X, begin, would are statements the available Commission. results I in and Exchange I with to section statements Private Reform discussed important differ the by would result are a made those those to not www.eloxxpharma.com, that following Chief of that considered website, within immediately our various on indicated annual and as Chairman this over most today, filed Officer statements views we company report other replay XXXX. Robert our historical call. and Act reports Executive Before of the as statements remind of during Securities factors, only. our Risk Securities like forward-looking A materially of the Actual will the including of forward-looking Ward, like Form Any XX-K you from represent XXXX, the to in turn now call call Pharmaceuticals. of be this meaning November

Robert Ward

Thank Webcast Eloxx you, and Conference Quarter and Barbara, welcome to Call. Third Earnings XXXX

fibrosis, new disease investigational our compound, we cystinosis make cystic growing progress our to substantial lead continued and We’re report in our program. company inherited advancing and that pleased to retinal in ELX-XX, advancing

the CFTR ELX-XX levels demonstrate data increases new Colorado, ELX-XX today NanoString with Eloxx fibrosis drug designation correlates at wild disease agent to intravitreal that that believe a activity inherited scientist’s community and other the by program focusing with for for a a cause our use key into these in pigmentosa favorable development new initiated demonstrates new Denver, data biologist, to in first advanced retinal several of exciting the review on also RNA the retinal injection is the you. October Messenger these received and leaders to American investigational presented different We’ve Medicines moments, tolerability XX, highly which dose-responsive on that orphan European data from and in interest focused nonsense Conference showed received available with In a forms Fibrosis in the compounds data Leber’s we Scientist well product have Senior potential of disorders report read-through In On candidates studies our Agency. stimulated the specific nonsense positive mutations cystic Dr. disorders. North Cystic opinion mutations. Usher quarter, and on pleased profile. was we cell from We’re and Goddeeris, cystic new syndrome, function, for distinguished Technology. support few fibrosis above amaurosis elevations data caused will or will by library and increased expert, using we at rare program. inherited We currently a these congenital This IND-enabling a retinitis that very and this type Matt that

development. later lead year this on program We’re for ocular nominate advance track the to this clinical to candidate to

treatments for and entire to wide-ranging partnership Blindness. proud cures that Fighting the into provide drive of retinal preventions, that the entered Foundation is urgent mission we a degenerative have the research will today with We’re spectrum announce diseases. Their to

support, through clinical Eloxx will and scientific ocular advisory Our portfolio partnership scientific with engagement the consultation, other the Foundation Fighting broad reviews include protocol development activities. Blindness supporting

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high Society As Phase a Health Federal and been European fibrosis cystic by have II application reminder, Medicines by priority Belgium programs clinical for and our trial ELX-XX the given our in the for to been a second approved for Products of has for GXXXX-CFTR a ranking Agency Phase population. globally which Fibrosis the with cystic Network. cystic accounts the most II patients is common trial Clinical mutation X% fibrosis Cystic initiate plan mutation, fibrosis and Trial clinical We on about

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in now for cystinosis open. IND In addition, U.S. our for is the ELX-XX

XXXX. II data in ensure Phase We’re top protocol finalizing track completion to our enrollment process with investigators rapid and the line on and report of in to

manufactured possible a retinal $XX.X and into previously clinical and disorders. can of rapidly This well programs, million I’m of milestone development be at funded we’ve the convenient bringing ability successfully and and expand highly equivalents to a on need to mission cystic significant is as deliver commercial safe is patients and their quarter who ended and product. final and our reported, that towards and stored lyophilized patients drug our top and at to in for to fibrosis accomplish with which Eloxx ELX-XX, talented inherited time physicians the of do and on are line temperature. room cash confident effective them medicines a and advance We As budget. dosage data cash in form experienced team in as we’ve cystinosis

You in the we’ll North commitment data Goddeeris to should highlights expect Dr. corporate add continue of we delivering like clinical our our and to presented new and now to as at talent discuss ask October. Fibrosis Conference American ELX-XX part to from exciting attract Cystic on the milestones. I’d key

Matthew Goddeeris

Bob. you, Thank

beneficial organization Conference the investigational forskolin At new assay Fibrosis XX, CFTR organoid [indiscernible] compound of earlier effects on these we Therefore, complement RNA RNA shared its cystic This American biology. CFTR Messenger in on University bronchial emerging activity. range using nonsense of to to And aberrant with RNA the the it demonstrates shared, nonsense This that disease organoid We dependence collaborating assay, attributed a codon is first and North organoids mutations a mutations often derived field this occurs action CF new ELX-XX’s in with not inefficient a leads mutations. nonsense stop in significant a patients CF CF with the and the understanding essential mechanism for broadly ELX-XX. series better Cystic ELX-XX Messenger Denver forms a how who these any, ELX-XX introduced to enable have essential like reproducibility to the the activity to have of the and proteins activating of dramatic swelling that data hold options. in we degraded. the underlying concentrations. also nonsense-mediated cell decay, reduction across mutation, is is disease. a October on reductions the of respond the in epithelial if Eloxx double is RNA hit, protein, patients that show production first year, burden and few, shared being rapidly the of As a response used nonsense to nonprofit understanding assay fibrosis different fibrosis as derived in studies which the in human model When organoids the the treatment patient and cystic essential HUB, translation target in other associated lead reduces Messenger in data the time, of of only to science an highest from can mediated of genes reduced a CFTR.

nonsense determine CFTR. to florescent precisely designed swelling mediated assay, Technology, with reduction was to bar consistent interest increasing In applied experiment, CFTR decay. the mRNA of activity CFTR in tags ELX-XX, we the study in levels of which function this measure in in measured changes if increase Messenger RNA Our to NanoString to codes addition would the as also

We wild interruption the to them previous compared increases RNA mutations. with are levels type. mRNA treated, consistent studies. cystic nonsense organoids in RNA following ELX-XX dose untreated Our fibrosis wild-type an observed data nonsense than showed data across demonstrated decay CFTR multiple to mediated These CF controls, these with by patient to in in ELX-XX. increased when healthy equivalent reduced consistent ELX-XX restoring or responsive organoids in CFTR process patient-derived greater Messenger Messenger When

data CFTR found to mutations. in Messenger mutation We swelling call with Phase focusing for the to with RNA, normal We encouraged GXXXX in the CFTR adequate organoid support cell administered. is of on provides depends in now is showed mRNA our Messenger by which that turn are levels. on back protein. to all, derisks substantial data strong to the Bob. by clinical possible function over ELX-XX assay steady-state used an make the After organoid restore substantial believe like the retroactivity that ELX-XX’s pool CFTR the the ELX-XX reduced to the is trial response These design RNA observed nonsense target it would increase patients I when II

Robert Ward

Goddeeris. Thank fibrosis update you cystic Williams our cystinosis. in give Greg an on clinical like programs to for ask Dr. Dr. you I’d and ELX-XX to two now

Greg Williams

and you, pleased to an update Thank programs on our provide the Bob. with very development our ELX-XX opportunity you to for have pipeline. I’m

We published a have soon. cohort I’m of also begun we submitted fifth results completed we our to the be which I to Phase MAD study in that the have SAD expect report study. the pleased manuscript, for

announced, we previously drug have are study through we to additional evaluate different MAD adding cohorts the As concentrations.

the or We for results publication. presentation expect to scientific submit complete study and the MAD

initiate report We data and XXXX. studies II our Phase line expect in top to

ELX-XX and been received approval clinical by fibrosis plan in As Bob cystic fibrosis, European cystinosis. Agency. designation application in mentioned has both II [indiscernible] earlier, Medicines clinical granted Phase to has initiate our and cystic trials final We trial

sweat For fibrosis, in changes approved at CFTR measuring is Phase for consistent programs with biomarker activity. drugs successful be II we’ll of traditional multiple cystic as ascending the doses evaluating chloride which other ELX-XX,

increasing multiple clinical mutation, most prevalent one will carry in at the to [clintrial.gov]. XX with optimal fibrosis into The II posted will planned protocol calls CFTR and in cystic for enroll least Our nonsense patients on further study to Phase doses trial than dose be more no GXXXX order an the on development, identify allele.

report to and results cystinosis, line is in granted in expect designation in Phase ELX-XX. now fibrosis has FDA Similarly, clinical cystic in XXXX. the We II for drug open, our orphan U.S. top IND the

to a total likely the surrogate these approved very drugs cystine be body, FDA alignment II be We biomarker clinical levels clinical on may a levels marker blood have the six cystinosis. the and we the In study, pleased measuring the in blood used cystinosis in ELX-XX burden are to of benefit. on white the Cystine enrolling dose-dependent cells in patients. predict cystine focused will most a with recently development levels in trial for and cells, considered inform white decrease this on of Phase effect

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accomplishment patients form for on As the an Bob both we clinical be using mentioned, and manufacturing lyophilized is convenient previously in final now will trials, the commercial which we and dosage path physicians important product. these are to

have the demonstrated mutations in pipeline, IND-enabling multiple and series. screening our track we to active disease on the in studies, disorders. lead retinal These our year. compounds data XX candidates nonsense profile on the clinical select we of we In retinal positive agents Regarding our show have this a for compound program, and activity these for are on in in drug inherited compounds currently as library ocular have candidates, these read-through inherited most of completed in progressing favorable XXX our data development and one

We continued we X top results I’d and and the progress the concentrations, from patent show the back for global studies ablation. where these pleased year to II compounds, composition now Eloxx with study electroretinogram Phase profile, over MAD forward fibrosis attenuation like with aminoglycosides tolerability and our are XXXX. I which particularly to matter wave portfolio this of holds preserved the has and cystinosis cystic life complete commonly and long of appraised keeping to emerging report of use. at as turn ELX-XX extensive in wave Bob. an With our on look forms in next rights or you has all line call

Robert Ward

now provide the for our I’d the nine our to of Greg. results quarter for Weaver, first Greg with review of and Chief to Officer, a you, ask like XXXX. Thank third months Financial financial you

Greg Weaver

Thank you, Bob.

or for company For XXXX, per XX, September three $X or share. ended months $X.X million a million September loss the $X.XX the stock-based share XX, The included three-month XXXX, $XX.X of $X.XX operating compared loss to noncash of compensation. period ended million per the reported

operating cash third quarter Our XXXX of use the of $X.X million. in was

XXXX, of for share. noncash XXXX, the months per $XX.X September XX, for per September loss nine to share, For XXXX the The included $X.XX $X.XX loss of nine nine reported or million stock-based million, the compared of $XX.X compensation. months ended million XX, net the loss company a a months or ended operating of $X.X

compared cash nine the were of September the R&D million. an of XX, ended XXXX, use million. million. nine operating of October an expenses compared months for $X.X in million expenses $X.X increase million $XX $XX.X September Our as for to million the months year, XX, XXXX months was prior first were $X.X XX, the $X.X ended of XXXX, the million three increase nine ended R&D months XXXX, $X.X for to for

XXXX cystic for IND-enabling with increased in inherited along fibrosis increased and Our to and in R&D CMC activities preclinical costs costs cystinosis retinal in have disease support the and clinical ELX-XX investing our program. studies for

million September million for XXXX, expenses ended months XX, September $X.X G&A compared $X.X increase $X.X million. ended Our the of three to three XX, an the were months for

million. an million expenses in $X.X For $XX.X the XXXX, September a XX, $XX.X months of increase for ended were same the nine compared to year ago, million The period G&A increase add service in capabilities and headcount. and additional headcount G&A other professional and we were build primarily fees. costs our increase have related to an and expenses increased as personnel-related organization salaries,

had and million. equivalents of we XX, September of $XX.X As cash

operations to our And shares fibrosis II line top I’ll product, XXXX was to and is call and million outstanding the Phase lead data in turn back September trials shares. XX.X XXXX now We for that ELX-XX, over sufficient of our your for XX, Bob. current to investigational cystic fund for expect total modeling, cash the cystinosis. as to

Robert Ward

Eloxx. both Greg. time you, It’s busy exciting at a and Thank

to new on on accomplish that medicines place in time, team the them. have patients and need deliver budget important We our the goals for

EMA. that and our orphan designation cystic ELX is been that in drug pleased for CTA We’re fibrosis has Belgium granted by approved

is IND designation. cystinosis granted orphan the ELX-XX open, the U.S. also Our drug and for has on FDA

data is agent top Phase indications, on are trials have remain our bearing track organoids initiate We consistent meaningfully year. positive substantial II the we’re Phase report read-through and data fibrosis studies. believe II as the new in only line We of encouraged nonsense activity cystic to very the expect mutations. We ELX-XX planned in derisk these to patient-derived demonstrated to

from and data our for track cystic studies top both in on We’re line cystinosis. planned fibrosis

initiated program by library. favorable to new compounds demonstrated and diseases have several focusing our tolerability inherited pleased a with the on profile from We’re emerging retinal

Blindness, be partnering We ISI’s Citi this progress. now On may with participating ocular Jaffray Conference. to as Conference [Healthconnex] the Operator, be X, to Healthcare for we’ll a Foundation development the be continuing novel call at the thank look towards we year. Health Conference. gratified Piper December as the in our on this want up Global well to expect We’re earnings clinical open Later for joining on molecule Fighting call, we’ll us and advance to you and third update Care month, to quarter we questions. you our forward

Operator

question first comes Citi. with Joel Instructions] Our from [Operator Beatty

is line Your open. now

Shawn Egan

Good is inherited my to Shawn, your calling pursuing? about differential compared Can retinal bit you you for in populations are And disorder the other the Joel. how compound big maybe nonsense Thank for profile in in indications? morning. populations question. aminoglycosides ocular to a [indiscernible] why This have you’re will safety taking mutation talk a also understand

Robert Ward

today. There genetic Sure, are Shawn. a joining Thanks retinal which us XXX inherited have for diseases component.

across may cases, more it’s disorders, of XX% the some XX% have mutations nonsense the in patient to population. primary different in So as typical those as XX% many inherited of as mutation, the and patients other the diseases,

Now have of or Each that’s those may the that of diagnosis. multiple those XXX diseases remember, means different proteins diseases, mutations.

is underlying often the has where reason for the So because and light, a brought a ability proposition, is talk Spark forth, attractive of the essential its absorb of the to example, therapy the restores role about gene regardless people mutation. LCA, in that why RPEXX retina in gene that

However, subset defect patients actually is the a of underlying where there are mutation. nonsense

have choose injections. intraocular Lucentis much they they would or for an may the gene therapy option read-through at would enabling intravitreal Eylea in protein, look go for by whether like patients infrequent injection, to of those or So of whether

molecules Now talking remember, novel are molecules. the we’re about

So worked based [indiscernible] is site crystallization time the when binding the approach identified on molecules, some developed ago. ribosome the been the Dr. screening that on had

so the the the human as the cytoplasmic about ribosome. activity have reassembled occurring. well been it’s as structure their disassembled They no on naturally inhibition ribosome And – ability we’re in put on for mitochondrial while little screened were molecules constituents or in groups bacterial ribosome active enable reduced the had the that and not read-through having to talking

with the associated aminoglycosides for bleaching or that themselves, in changes be electroretinogram. with in So it’s they the retinal eye, none can

these Now activity reduced waveforms doses retina halted there’s cells amplitude neurons known in and certain are that the it’s that either of the waveforms at just eye. with the measure are that of the suggesting in or interference

not when changes screen no for range for we in aminoglycosides with a So changes profile we whitening. bleaching retinal the and for we at. observations alterations look with waveform. assay. And dosage the looked we we’ve that retinal really do safety, perform the quite electroretinogram. in see of look We the different We waveform, show see that’s Where

that our ribosome the Does Now attributed Shawn? selective that help, that mitochondrial inhibition molecules for fact could with ribosome. to be are cytoplasmic reduced the on

Shawn Egan

very in Yes, additional confidence maybe And bit follow-up. a your both you Can on so studies far? in the brief learned you and these things a ocular Do you’ve indications? they that’s interesting. give talk cystinosis as cystic fibrosis

Robert Ward

is you think the at read-through, Yes, of establish restore for remember protein production. how the of when – to you sufficient time about tissue period question it’s sufficient do a drug that

And – Well, RNA the stem to with transporter from fibrosis, underneath swell. are Messenger in these drug forskolin, and sufficient them in grown the that now fluid example, restores production. understanding at when they cystic allows to we protein of patients So organized transport cells function their is stimulated part culture. a look that for where remember enable individual

separate whole concentration you Now subcu we individual of drug patients, gives a question injections. a that specific achieved in is In experiments development, set in on confidence? that’s based animals what do tissues second the ask drug on what

a where of So study where to dosage has in do dose concentration range inject, see studies activity. the we animal our doing which our look say support subcu cellular there’s the

fibrosis, enable we our So today, range that cystinosis the of dosage comfortable activity And cover we in will range. that to in cystic study. dosage the completed believe I believe be that we’ve of think studies that range the that dosage on we’ll we’re range of completion efficacious MAD

expected that Shawn? activity. So able we’ll work the Does cover through of to ranging dose that studies range make sense, be

Shawn Egan

Absolutely.

Operator

Robinson Our next Edward Nash comes SunTrust with question Humphrey. from

line Your open. now is

Frank Atkins

Hey, tell three My good Edward. questions. cohorts progress. adding about for additional can first MAD Frank, in on morning. the you one have the study? reason on This Congratulations is is, us the for I

Robert Ward

you Sure, to go has at it of site think that the you’re of III, Frank. expect be of XX% to in supply. batch from to drug your pivotal a to of been a When commercial you manufacturing scale. pivotal have least be design, commercial when that’s Phase has produced the trial, And trial made expected

were product So why component having a and trial. of that such pivotal an we was that’s drug why important talking doing about lyophilized

second the the on is and that pivotal you trial to a The sure dose tolerability right drug administered overall is safety pick that want being and make piece you the way optimizes the profile.

is during dose time the the form out ascending drug So of schedule, the Part B I, multiple dosing Phase work study. this the to

added at looking we and look that provides concentration we’ve to understand because some most that So concentrations, confirm we an there’s believe to injection profile the favorable drug that. we’re that tolerability for different optimal cohorts

that able this is the injection we’re we dose the want we is when the concentration and trial pivotal range. volume started say this to study our and of So to

want we questions all before start to we pivotal So the answer MAD in study our those trials.

of have Galapagos these keep the think Spiro X trials. in trial within X, with were speed year. and streamlined patient design we in a And Now they allowed completed their When trials, their escalation. you Spiro finished started mind, which

So we think very efficient designs. that are those trial

II study comfortable looking Phase are the the that of in and that recruit questions we so endpoints. we ensure And designs the been them. appropriate investigators and at we’ve the right that can important to complete that is we’re amount But working that parallel answer have also assessments

the work us top sure our in MAD through to our trial we line So to we study data make that’s where in sense? allows comfortable pivotal the efficient to make go of these and because we’re with design believe guidance right our with Does dose that concentration. questions XXXX

Frank Atkins

very planned reduction proved you question us measured know statistical not second can organoids, Yes, that that to percent of based – help my numerical XXXX. it’s – submission the endpoint you your – also can are remember significance, preclinical not your And And the are but in frame you expecting? primary in study, just in reduction? I from the also probably the what secondary as powered And sweat And FEV give a to in helpful. expectation? endpoint. what’s also probably II show as study on terms also Phase so a us but you that’s you and you chloride given some is so the data trial you size, regard you expectations? to talked of expecting And on I what

Robert Ward

how Sure. designing trials that’s really look for in guide our and at previous Because a question. terrific And I that development. think has a demonstrated been the we is what internally in trial

that the we we a enroll groups. two II a group sweat to really group and is in that Phase So can function chloride a it. at we no patients smaller Remember XX at a we more control type Phase lower III. the pivotal the key look chloride" assessment that it’s have leaders on approximately But then sweat us that there’s sweat in chloride, because Phase are trial, the change. Because biologic III, the of where where confidence data KALYDECO smaller Phase the KALYDECO milliequivalent back know the look a right, go, at think II expect way the "normal endpoint to lung you’re of XX of KALYDECO II milliequivalent measure treatment at range statistical have change Sweat when than trials you versus you healthy moved difference in it back that average if that between change lead of than XX trial, our was drug get into no-go they in has opinion presented, in of we the trials, the is patients look When picking as these Phase was activity. told are the a showed still at – it and chloride the program. a as look for strongest population to $XX seen And trials. that’s many doses,

benchmark in that and is think the looking the discussed values Because dose individual of assessment move looking back because dose an significant it’s course, FEVX, population. the for population measuring more the dose III we’ll that We’re we’re on is biologic and to into same escalation see that look clinically likely small we what sort it the population." we patient it’s but it’s that signs you going range we normal which expect the where trial first "the Now, we Phase from sweat in FEVX for chloride terms identify a about. be perspective, it’s of than population, trial, that of based In patients to range. are of at the activity, then is

used II response. for response that that reimbursement in Now organoid drugs programs approved the Europe in the reason are why we feel that being patients an currently Phase is enable cystic organoid for fibrosis to today derisked our show is

response Phase the to response GXXXX with in is see studies organoid we had of FEVX population the or So in previous of amount drugs. that II focus trials other manufacturer’s in our clinical a The program we’ve substantial thought that would that of trial. confidence seen FIS drug activity to it’s that be the significant proportionate in the response in our response a Phase a we II to us organoids. expect show FIS pronounced and gives size We’ve

activity. we but seeing So clinical also historical a are response it’s in focus would data both suggest based is we’d that what genotype on, the on

Goodyer’s that also the falls cystine in achieve in mouse cystine with intact those dosage to model XX% escalation again Now within dose livers expect able and reduced side, cystinosis on both shown in took the levels animals the cystinosis the changes range. in And model. and we the cause the fiberglass it of been that that we’ve range knockout the have Dr. reduction work the on really fiberglass to a

upcoming And that us so trials. those confidence our are the on give endpoints preclinical clinical

Frank Atkins

I for last If question. may,

presentation, four one how you And your tell us indications? you’re can – on in four it to the there slides. choose, like X going compound indications is prioritize mentioned among to of are going just the indications? to you’re those ocular going those And So be you lead

Robert Ward

– intravitreal we’re and through injection. here about so inherited Yes, accessible talking disorders retinal again

that right As drug that the of over enable earlier, I we in tissue would mutations the can for read-through. that period time. concentration at right mentioned tissue nonsense occurs, the anticipate location, achieving it’s When of we

disorders range So to are an suitable well as design the As that’s large of trial? the clinical as the we II a that ask, patient Are endpoint example endpoints point agency accepted for Phase of is has across population look registration. ranging? to an a dose there enough there where

think a of if one- and that’s us that, of pioneering doses different LCNA, a you an know ranging, we response. endpoint. work, to physiologic you over allows ranging take two-year that time, dose field measure have because of acceptable for visual Sparks So of want of that endpoint think or Because if period dose we

for a long period dose time Some ranging. not monitoring visual would of field over be ideal

different sure tell we Phase concentration drug, one the the look can and endpoint we we why So an what that’s could would have activity How about then achieve can that than do confidence make know right development be for of another. be across us indications, dose measurable path. we what as tissue we the it’s design II, that

we dose for the that prioritizing. criteria So be would use

Frank Atkins

question. again. Thanks the taking appreciate I

Operator

with question Canaccord. next from Lee Arlinda comes Our

open. Your is now line

Ben Shim

age sort be? maybe expected initiation the And patients Good bit legwork the taking fibrosis? bit will Phase of Bob, maybe tell you to the up of us to As And thanks well additional that the little little And Ben on the that treatment going cystic maybe these start. Shim, understand about will of, duration the can lead morning. can It’s what timing for XX ducktails, question. us probably first for you light maybe in what And help our II Arlinda. a trial of the is be? groups more in questions. a as

Robert Ward

be patient would intervening overall in have vertex we’re of year, our many develop before groups. When Ben. development, efficacy asked working with youngest early of drugs to for desired safety for for to approved use this a idea drug way that population moving we goal. cystinosis when disease, cystic regulators study always the to is an approved just work think the youngest age patient’s drugs patients in you Because were clearest establish in ago. the younger years just But and this to whether And portfolio, was development, Sure, ensure drug we’re if we life the profile or older of drugs. us that rare the fibrosis, group the clinical that and in it’s with

we at we’ll actively in So will recruiting the agent patients that’s read-through the first. demonstrate fibrosis, of first think organoids. in working both you we right program the be that cystic populations for the When of to these genotypes ELX-XX anticipate indications, be only now, older patient with the is the look drug activity

show in to failed failed drugs understanding activity encouraged later view, by We’re clinic. have from our that this organoids response have in the show to because very

very recruiting, pleased enroll the us a because move we’re to able be be clip. see anticipate to we’ll company we forward. profile only an activity rapid And to at that that’s encouraging patients So we’ll

coordinated and advocacy a U.S. very enrolled here deal the sophisticated cystic field, being clinical with knowing and Now the Europe a fibrosis and patient credit the patients also in for Fibrosis are fortunately, and in and in in Europe registries their network patient groups. in the here sequence is Cystic both trial those great Foundation really deserves U.S. of

aware And investigators eligible could are well. that disease in accessible so be worked patients as who are and state are investigators the of who’ve available

not forth those brought has point field previously. the rapid are to a all had product reasons So molecular Cystinosis, recruitment. to

the explored. the certainly, being disorders, and is understood. molecular the be is disease still where is still strong And where with Usher some available. fibrosis. University a those has area a leading McGill the But cystic patients high amount program sequences retinitis enabling if areas to basis that’s retinal so that of of again field genetic an charge pigmentosa across the is think means forward as to syndrome we patient sequenced, with mutation, patients for basis a advocacy that’s the compared nonsense with of trial it’s emerging of looking So a are sequencing you that well Other move disease as the

and aware to to So trial we make viable. being identify the investigators get want where pick is forward patients of qualified areas work and enrollment to able moving

Fighting advocacy so the the these believe registry And disorder of in are cases. whether in work here or patient ocular for patient U.S., we Cystic such be it’s critical great in Foundation the believe worked to groups Blindness that Foundation we who has the a and both success partner Fibrosis has

Ben Shim

That’s much. very helpful. very Thank you

Operator

like the I’m turn time. to at Ward showing remarks. in to I’d queue closing questions Mr. no back call for further this

Robert Ward

year expand has with data the for we appreciate new on think as hosting, team fantastic RNA build dose we’re Thank into and the for higher, new biology as Eloxx delighted therapeutic This and the promise now well. never portfolio. you, Liz, a Messenger We read-through the area. and is a response questions a showing of been I

us, with call. and quarter’s you, we look That on our everyone, for talking conclude our joining today. would thank you So for forward call to next

Operator

Ladies and participation your concludes you and This the Everyone, now in day. conference. have may disconnect. for great today’s program, you gentlemen, a thank

Eloxx Pharmaceuticals (ELOX) 9 Nov 182018 Q3 Earnings call transcript