ELOX - Earnings call transcript Q1 2019 | Eloxx Pharmaceuticals, Inc.

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Operator

Good morning, everyone, and welcome to Eloxx Pharmaceuticals' First Quarter 2019 Earnings Webcast and Conference Call. Today's call is being recorded. At this time, I would like to turn the call over to Barbara Ryan, Eloxx' Investor Relations Officer. Please begin.

Barbara Ryan

Thank you, Liz. for Pharmaceuticals' this results first update. you review for and thank Eloxx of quarter and joining morning us a business Welcome, XXXX financial

me President, David Chief Snow, and Business Officer; Weaver, Ward, Greg Joining our Schneider, Financial our in Chief are Senior Ophthalmology; Susan Officer; Greg Vice Williams, Officer; Executive Development Dr. Chairman Operating Chief Dr. morning this Clinical Officer. Chief Robert and

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

Robert Ward

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Greg Williams

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Robert Ward

like programs. in I'd Greg. you, ask Thank progress our Dr. Schneider review ocular the Susan to to

Susan Schneider

time pleased I'm programs. you, ocular our Eloxx build Thank we be exciting team at as Bob. very our and to at advance this

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Robert Ward

Thank you, Susan.

really you Our XXXX. our quarter like first one of results has of with a the now a And provide company. to been for quarter review Chief ask Officer, first I'd the our Financial to successful for Weaver, financial Greg

Greg Weaver

Thanks, Bob.

XXXX, in company in of and company's will had current based fibrosis our the of into operations which cash March the and second quarter plans. million, cash, XXXX, cystic As data XX, totaling the fund marketable line believe equivalents top $XX.X operating we upon cystinosis through and securities XXXX

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as again, operations. Quarter-to-quarter same expense $X totaled $X.X noncash to million expense compared first in compensation, to quarter the growth in $X.X preclinical for million in for same other I'll along due fluctuations XXXX. the our increase, salary-related compared million covenants. professional due XXXX, expense And with primarily for quarter financial to the Bob. $X.X R&D largely and concludes This R&D clinical period costs call XXXX. First the The XXXX $X was million period in fees. back and to turn to G&A million our increased the quarter-on-quarter stock G&A

Robert Ward

development built and in Europe the strong for in efforts Greg. a foundation Phase for to beyond. U.S. reach plan trials fibrosis company our this top X in cystic clinical year, data XXXX you, the both Thank successfully the growth This XXXX, accelerate we'll we In and clinical necessary line conduct and year. and trajectory our

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the our disorders currently on increased to Annual Cystic initial a Cell pleased of towards these As in Gene program. an We're IND-enabling industry Usher Therapy response. acceleration efficacy We've program development. very team gratified the build I'm had to Summit, studies partner inherited Foundation opportunity team Last lead efforts focus the conducting to an syndrome and Fighting supporting and with attracted Sixth clinical molecule the introduce have our positive U.S., we week, retinal Dr. with to Blindness. efforts to experts retinal we're our discussed, FFB and cystic the complete community to Foundation Retinal intend at with the patient our Schneider expansion and inherited and Innovation ocular of advance Blindness with scientific received as novel and Fighting we in and ocular Foundation the diseases, Fibrosis our in engagements very fibrosis U.S. a

business be also indications our very become across and/or to as us. to geographies accessible We may a increasingly in new variety portfolio continue active of the development

Cystic now in Next Fibrosis our Las Lynch XX Bank of May scientific in the three America European call. to we're Conference And to our week, questions. for to Liverpool. looking on continuing XXXX the forward Vegas. open our forward look we'll in for you update the presentations We first be up us on you ELX-XX June, in participating on progress. Thank Operator, at quarter for early Merrill call Meeting joining you Society earnings may

Operator

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

now is open. Your line

Joel Beatty

Hi. taking for questions. Thanks the

on multiple The first is study. dose one ascending

cohort, some the this give and you you've expect a final And cohort the of compared of discuss it respective to areas? sense might are play how final you was could amount the therapeutic dosing Now you to able just that reached how selected? dose

Robert Ward

start agencies for regulatory across into – should healthy FDA potential the for one asks and I is Joe, safety previously, purpose to optimal from up the that to then longer the Phase now, want in And to us Phase in covered a when study, bridges X. the talk dose expected we've doses the effective a X. one patient how range be we Williams, doses And that Remember, patient is first there move do what we Dr. safety of different of we MAD dose establish allows MAD be will this and range identify of I'm lower have you the anticipate exposure that take to patient population. in is dose the escalate that about and that often that a benefit at a we the from to X, with we Phase escalate Yes. than for efficacious remember dose X to dose Once discuss is sorry, study mind, the Phase is study range the to when be expected volunteers. the to to used. we believe paramount look

Greg Williams

you, Bob. Thank Sure.

we based to framework our final twice far. study consistent the range of explored weekly. kilogram. export. models for doing X.X per kind study, in kilogram is we doses That cohort with administered of We That's efficacious the the think is doses. milligram with on SAD which per – kilogram a were X what MAD up range the dose. per provided milligrams animal week should for be dose our This to that XX single to up multiple total We've range So original provided a milligrams doses per thus

Robert Ward

correct? dose dose that is trials, fibrosis X, be the the for the then, that cystic cystinosis range that and anticipate X would both covered range in explored Greg, in patient Phase be will both And we same same populations, Phase and

Greg Williams

healthy volunteers to dose in perhaps Bob. Absolutely, any as Yes. patients, And didn't healthy we you as have don't the we because with volunteers, push high mentioned might potential benefit.

further escalation safety full efficacy and a could patients range. we in dose that exploring we're look So at as

Joel Beatty

the used implication the it. there – are been Could other – of for using then models question drugs how that your on what being for are fibrosis. discuss means organoid a agent? organoid Got and cystic for as you And models fibrosis you've for cystic

Robert Ward

I Joel, to medicine. really personalized important about it's about it's the that fact Yes, think think

So then organoids on response in payers in there they've because laboratory Netherlands see. that the we approved the who today, based donated know the that and drugs currently are that the they're label are of organoid they off use screened individuals reimbursing

Snow, already marketplace. now by while European have at HUB, At an And this we and for Amaya that happening organoids a collaboration will It's we conduct test could that level in more future be and the program overview to what HIT-CF, the regulatory see with the into a with studies provide laboratory and just potentially? you to in Union. the funded that's forward, is of in implications expand collaboration sophisticated David clinical that's a So the how environment moving program. and

David Snow

Bob, the of primary disease. we're and Both dyskinesia. there's looking also ciliary think. at, prevalent the looking in U.S., important kidney at kidney are of highly of in We're I a these programs number effect diseases polycystic

For PKD, there that. patients a in is substantial have for There – probably nonsense yes? XXX,XXX to is component U.S. XXX,XXX the who

Robert Ward

organoid confines thinking was of the implication of within HIT-CF. the more I

with So HIT-CF. ahead. Go

David Snow

in clinical in XXX very CF going couple patients. the years they year, identify next organoid important confirm in They'll joined, We've mutations to trial those to program to with Sorry. Europe. rare a between organoids patients. plan effective XXX are do HIT-CF the a is of last as and within collect of

in way that of database get we that. participate good biomarker to really patients a we'll large And this as related use to have So as It work will we able be plan collect mutations a collect that, then EMEA potential expect organoids will with a for be nonsense they use very and good get regulatory information a by be to and path could data to us on therapy. a it to robust will the number with that database. which as

Robert Ward

a enabling an the second ability mutation as is But patients' Joel, grows one be ocular in taking it patients different different mentioning. to it's other footprint David. drugs is they areas. be likely than using cystic as Thanks, use right. alleles in applications the the cups value do was earlier and be HUB increasingly, a know collaborating the model, in in cells it I on for reflect And interest. due see in the those approach a lot you're means, we fibrosis most of will eye genotype, believe, have responsive have but oncology. allele, likely fibrosis eyecups, my structure. And of And mutation application. did It's very translational Organoids would by two likely perhaps area to genotype organoids, used continue figure of physicians we similar if enabling It's more for with thought cystic on underlying to are that sequence matter. I cystic genotype hold of on that out model development. ability has Vertex establishing a it's approach, of based genetic medicine part to fibrosis where that early we we'll our which high their a most the to will a in the other outside HUB disease a is translational drug David add as indications, medicine are interest be – are and applications But personalized of fantastic on work to but and the in of type to be information. would molecular for a And think because selecting which know expanding we but to and of the not the both organoids organoids where eye, a specific and to into think to also personalized allele

us a to platforms. for So advantage it's of these terrific take really opportunity

Joel Beatty

may on Or the an and measure protein those that cystic question and reduction opportunity Is road mRNA that to and fibrosis of the one be areas? cystinosis. trials in for are markers planned needs X be that efficacy last the studies? disease specific to to is then different a there Phase down more And Great. it focus

Robert Ward

that ensuring: about path, correct, registration. and right? and number ensuring you're number you scientific No, have focused regulators benefit one, on So Joel. patient information safety; for potential are we two, are When think the that measures suitable of then the development

path, think endpoint well-established you FEVX be sweat really where answered pivotal blazed and questions of look Phase to so there that Kalydeco of program are some often perspective, at sometimes from in think But path the will the kind a patient-derived dose samples. for X looking of select the sometimes if chloride a to that trial, we fibrosis, So in models, we those of speak. as scientific translational is at cystic pretty a

laboratory. if enables So testing to have organoids you to made, occur are the think of in personalized individuals, cells who the that donating

that and of at the sure but specific variety different a but you're the our the will it's in more clinical questions of we of all in be within ways program samples. program on of the I'm patient thinking looked based answered, context of those So context generally, sometimes not

Joel Beatty

you. Great. sense. Makes Thank

Operator

comes of Tenthoff question with Jaffray. Ted the next from Our line Piper

open. now is line Your

Ted Tenthoff

of and thanks that something get of Or place data up on color that the the we this you're MAD X could Great. would year-end? is update. have lot is be for I follow-up if better I much it NACF? may. then A Thank a release to here. bit more want wrapping a really progress question, you taking kind great And presentation to release. what is now Phase thinking this Is a with I see very quick respect around press likely by little

Robert Ward

No.

You're right, Ted.

European but Meeting the that is And abstracts American meeting later June, this North is advantages the well. the year. of for Fibrosis year as deadline is One later Cystic also this late-breaking the is in

data to meeting. execution scientific of be that know, as with context will rapidly during whenever has share so for that ability in planned to we of the context see possible you the prefer audience a data and trials, the the as possible clinical So meeting that as

looking definitely continue down would certainly level. our we're preference, on opportunities for it to So that timing, comes basically engage the and to be to scientific but

Ted Tenthoff

if that think, is this really where beyond for us Yes, for you sense. I much a Usher therapy? really sense a effort received. then a I be makes well syndrome potential like Can company, think becoming And the which, the of would bigger may, give program, I it lot read-through ocular on sounds just you other see

Robert Ward

the we we to we concentration time, on many to read-through drug, the read-through right right able the ability period feel stop have in when we for of generalized codons. achieve achieve about be premature Ted, should tissue, that a if right of think way,

the premature level. administration, But variation the issue change the if the stop Now the a Exxon other And right of codon the a the in remember right the pieces the share is in intravitreal retinal of it does junction XXX we there terms potential dystrophies is is in there with some context different sequence-related inherited concentration data us said, read-through read-through or because stop is retina in make in in the of around cluster in right on nonsense-mediated position difference we that depending gives premature concentration a Well, where general, decay. there to nonsense-mediated And codon, the period tissues. drug the those for and successfully achieving for read-through. that time of choroid achieve of occur decay.

a as single cell retina. pigment cells, think protein about or eye, is numerous So not make every There the whether that protein functional. nerves the changes we is make the in processing the where it's the difference signal can the retinal types epithelial photoreceptors function. up But various of proteins components in the

visual are So with allows pigment. restoring of RPEXX, remember Spark for an synthesis that enzyme they

believe and function, range can range insufficient or available that that to in be protein that's was clinic. the are tomorrow it's normal function perform to functional physiologic the protein restores measured a there which we So a protein, levels yesterday that something restored back

proteins. could the pigmentosa, bit focus So you retina Usher of think functional the explain syndrome, about medical retinal of you epithelial or our or other Susan, cells then as of Susan disease we to pigment areas want retinitis unmet high Schneider, need that choroideremia? little perhaps choroid efforts would on itself, And really the be a

Susan Schneider

Bob. on you of in proteins to you, the genetic nail the Thank caused We're looking mutations. hit enable full-length by head. Yes. are diseases nonsense that production the And these

retina well-tolerated, spaces from drug in forward inherited at the And total. specialists focused pigment into looking saying, can within Foundation retinal to that other more multitude looking exciting, accepted was on good, at KOL had choroideremia. was that the So there that with Fighting had that amaurosis And also think board and the we the that, great say, or into is Innovation the the space related at, quite Leber's target retinal the through only inherited approximately feedback the level inherent layer injection, in intravitreal look there also a these that RPE account down and way, levels that we're are be a we Bob with move that's intravitreal disorders really across of more to enhance disorders. would are safe, in in Usher the epithelium. could too, disorders, a these though, there, our about during not retina We feedback U.S. meaningful exciting. of retinal XXX,XXX Blindness. ARVO approach the host Summit we pigmentosa, retinal of clinical quite that in And also half and including the that diseases macula, of general, the an retinitis retina from a development. for functional patients where at photoreceptor in approach specifically clinically meetings output treating that approach would congenital blindness in is patients of that to get the cells we're way, neurosensory as could to I I the on our the really but think, photoreceptors, In

Robert Ward

Cystic good framework here thing very research. a U.S. that remember. The many enables and the That's one science Vertex a in in point. Ted, working Fibrosis established really with to Foundation cases basic

opportunity Blindness My database which retinal are Fighting to sequences is similar sequences. leading Retinal right individuals for available. Tracker, inherited now of is donate Foundation called patient an development the diseases The with widely So a

be allow to clinical there models clinical and that when are eyecups about way that molecular conduct those. sequences where to the or there we would population sequences might us because understand frequency, available enough organoids be best So translational their to address might talk understanding there of is that the complexity, trials development or where

Foundation eye helping right those of than a bit Fighting cystic collection but really now sequences. is to So earlier research is catalyze Blindness little fibrosis, certainly

Ted Tenthoff

additional the all Thanks for helpful. That's Super color. great. really

Operator

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

is line Your now open.

Fang-Ke

morning. Edward. is Good Fang-Ke This on for

And to the pharmacologically ranges X.X. then also, the this earlier the from published dose study the we you is, when that paper, I X.X. Phase those mentioned first active mentioned So think, about X year, is and back question X.X you in look SAD you that

including decided clearly remove to the So per kilogram then your dose? study, is – kilogram including what you the you dose? the X.X include to you in MAD decide milligram ask other just range but want make X.X signals only highest I to milligram And are that X.X, the per

Robert Ward

Well, across, recover confident the in think demonstrate to activity. biologic we're we'll first we our Phase I which X range expect think, that designs, I study

to we if sweat was take in there into millimole one and sweat select change that of of example, sweat were normalizing might would for was higher be which be pivotal there XX activity, was why a chloride as example, increased, of chloride, the biologic will Kalydeco millimoles an dose chloride, but program, So as thinking XX in that Kalydeco be an would example change viewed trials. dose

reasons designed believe sure a So to then that that with we're Phase patients. there the to for beginning clinical that looking evidence a the potential support to benefit. make as benefit we and that escalate, dose when dose And X is we're trials, has dose want we for we optimizes

dose. from same prefer minimal the that the the look doses two if we of perspective, remember, regulatory use effect Now they

we approach. additional Dr. And is Williams, a would select dose comments? so a lower typical

Greg Williams

Bob. Thanks, sure. Yes,

were thank in safety We reason volunteers XX-day just dose It us kilogram tolerated. any study That's there to milligram And MAD X.X great any was cause dosing didn't the a expose there you. concern. was So question. that no healthy well to signals that think the at per

Fang-Ke

MAD they healthy only is volunteers, two then going there's to going abstract study it in – Or – to you're to going there? present going be of for are the study? are Great. just It be sense. to June, the present perfect And the that from from really SAD including makes any you

Greg Williams

Yes.

include MAD that volunteer will and SAD reference papers We of healthy both data. the in our some two data

Robert Ward

well presentation to as the profile. Those the to low that Greg had healthy there's provide you're as proportionality shared, updates. administration as then inter-subject referring dose to will, I demonstrates pharmacokinetic also X, ELX-XX opportunity poster and those on as think of the be be June volunteers the a on the linearity will variability. And

Fang-Ke

lastly, that impaired then a more right? ELX-XX And mentioned you renal then for what patients detail bit you think, And that cystinosis, PK I Great. us just study in of you can a more give that that ran I patient, in with the still you I if As moderate Is mentioned remember data consist you have the renal think volunteers, impaired or identified? mild been PK healthy the correctly. looked case?

Robert Ward

as you mild, Well, it's moderate I question and just the want do that's Dr. you comment. believe to today? impairment are renal think when renally is cleared that drug Greg, occurs, renal ahead a what was the William's of increase comment observations study on go predictable our and

Greg Williams

X it saw kilogram good consistent study. time. We've in the study a SAD to the half-life exposure. seen mild on in over results anticipated. but impaired evaluation We've that on a that in very moderate per slight then impact in renal to And predicted, as it renal into we've with severe. pharmacokinetics volunteers. data in dose we was we continue increases exposure, of increases going of slight some study the So was We're patients, some impairment healthy of We a seen use that, and with prolonged We have the dose the what in the would thought potential exactly lot have exposure. dose used milligram MAD

events. adverse pharmacokinetics. So far, Again, no predictable

really us would very experience. it's a it what perspective, gives comforting that idea from good and renal-impaired of So patients

Fang-Ke

you taking our I Thank you appreciate questions details. the Great. for

Robert Ward

Absolutely

Operator

in I'd remarks. like at call this to closing Ward to And questions the back for time. Mr. no further queue showing turn we're

Robert Ward

Well, Conference opportunity first of week you We'll Lynch and in updating call. that Las look Bank for the everyone, in on to quarter host to us the to you thanks, for happy Vegas, quarter forward hopeful America we're are one-on-one our those joining next of our for progress there. participate next have Merrill

to you presentation our That as our year. conclude that be and we the webcasting today. will progress update look on continuing to programs forward through will We call for

Operator

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

Eloxx Pharmaceuticals (ELOX) 10 May 192019 Q1 Earnings call transcript