ELOX - Earnings call transcript Q2 2019 | Eloxx Pharmaceuticals, Inc.

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Operator

Good morning, everyone, and welcome to Eloxx Pharmaceuticals’ Second Quarter 2019 Earnings Webcast and Conference Call. Today’s call is being recorded.

At this time, I’d like to turn the call over to Barbara Ryan, Eloxx Investor Relations Officer. Please begin.

Barbara Ryan

Thank you, operator, and welcome and thank you for joining us this morning for a review of Eloxx Pharmaceuticals’ second quarter 2019 financial results and business update.

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

indicated statements result would Before of this and Actual Litigation the be these today, Reform of Form Private during are our Annual various our by SEC. important those forward-looking XXXX like we within represent X, Securities that from may begin, discussed to you differ August meaning statements forward-looking call filed considered that on Commission are Exchange factors, historical most a XXXX. our risk in including Securities the reports as views XX-k Report the Act the results remind materially other not statements in as of factor with only. made any to those statements the and I filed section with recent Any of

available website, and Chief this of turn to Chairman call Ward, A my is Pharmaceuticals. Robert Eloxx the call the to www.eloxxpharma.com. on will Executive replay pleasure now It over of be company’s Officer

Robert Ward

Eloxx’s you, conference and and XXXX call. Thank earnings Barbara, webcast quarter second to welcome

of as X top remainder and be the expect in cystic our ELX-XX to both trials for line Eloxx, fibrosis lead cystinosis. to We for exciting we data extremely clinical from XXXX Phase report expect our investigational agent

the and quarter, second impairment also study the we During renal MAD our successfully both Xb study. completed Phase

from this X cystinosis, programs we to and report line are track on We our these year. cystic in are currently laser-focused studies and fibrosis advancing data clinical rapidly on Phase top

or open, we been Phase clinical U.S., the pleased the where are Foundation, the expanded CFF. cystic has to protocol have fibrosis for our IND by trial Fibrosis the is and Cystic We our endorsed X that program

presenting forward for XX, also at Fibrosis we ELX-XX look this to had meeting. the North October Conference abstracts data we and announce at American new the have that We to accepted three presentation are pleased Cystic for

fibrosis Our the GXXXX most enroll cystic one CFTR about both trial fibrosis cystic or and common fibrosis carrying cystic patients second Phase XX is the globally than of mutation clinical on for will accounts the GXXXX of X% X population. on gene. no more alleles

unmet cystic have patients positive few, the is fibrosis options. and across high only nonsense a to demonstrated in targeted medical read-through if patients need organoids These mutations. ELX-XX any, therapy from derived results majority the have of treatment

positive representing organoid be In mutations, studies clinical of with Phase benefit. the approved for predictive we in our patient-derived ELX-XX data organoids, to the X program. generated highly for therapies model shown derisks And all of fibrosis, cystic have that substantially nonsense believe has XX% we over

X also are cystinosis we We trial to for Canadian nonsense in a mutations. clinical with that have initiated pleased announce Phase patients ELX-XX

funding this this and forward from top to for cystinosis quarter the fourth early We have from of the Phase Canada. X Quebec and Genome received trial non-dilutive reporting this support trial line in in for year. are grateful Genome we that We look data

molecules ELX-XX fibrosis, such evaluate the in as from continued Based our Additionally, new in our work opportunities screening include or to ERSG screening novel other lung, library. to primary cystic our dyskinesia have indications advance advancing and to work our on others. ciliary diseases programs for we’re

also progress to continue with syndrome. We in in applications, initial focus ocular Usher

in research our David where Business our these details nonsense additional disease, kidney from Snow, the autosomal dominant diseases We in on mutation study into cystinosis as kidney renal with a results high such completed our the will that believe provide you Chief impairment Officer, patients. beyond prevalence more the support there’s call. expanding later of polycystic

as communities developing tackling development excitement for clinical business multiple a as for in there’s both community, scientific company in challenge substantial actively we great engaged the business well potential advanced the therapies are of most and where the and new nonsense discussions. in these the are programs, We mutations

cash million ended XXXX $XX.X with cash and of equivalents. quarter the second We in

fibrosis funded advance both of screening and clinical as well cystic in are well into XXXX, to molecules We advance our ELX-XX in the top line programs programs for deliver as our and cystinosis and library XXXX. data

Our and team possible. mission committed as is to and safe talented our experienced effective them rapidly patients who as of medicines to highly bringing need

clinical turn with our Williams, I’d Dr. who’ll an the advancing you update Phase now Gregory like to on provide Operating Officer, X to call programs. Chief our over

Gregory Williams

to Thank provide update in X for you, cystinosis. programs you fibrosis with like on ELX-XX our an Bob. cystic I’d Phase and

ascending XXXX X, North multiple October. dose and for study Fibrosis American the As for abstract has Phase presentation Bob ELX-XX accepted Conference Cystic we’ve for our been mentioned, completed the at this

one X In with our we allele. our Phase Cystic open a mutation, at CFTR clinical patients IND and U.S., Foundation. the least in GXXXX most nonsense is fibrosis In endorsed the for protocol by U.S., trial will eight on has cystic Fibrosis been enroll prevalent the the

at Dr. Pediatrics, Dr. Cystic Children’s Hospital, Head will Center, serve Medical lead of program cystic Hadassah global study Phase Children’s the Kerem, for in trial Hospital, Eitan Division Adult investigator. the U.S., Boston Ahmet the be of the and Director Fibrosis clinical investigator as X Uluer, our the lead will fibrosis of

this and we later We line to forward top these gratified the leading by participation look are two of year. reporting data experts

four optimal per from Europe, will the and milligrams U.S., Phase to patients calls The order include X.X of an identify development. X up doses fibrosis to kilogram into X.X carry up for per day program protocol to to in ELX-XX, in increasing dose Our cystic further Israel. XX ranging

Phase in as the activity. which be sweat consistent While for multiple endpoint at with patient is CFTR other X, drugs approved doses X primary biomarker traditional ascending evaluating ELX-XX, we successful programs of the measuring safety is chloride will in changes Phase

serve pivotal be also dose sweat advance the will in We changes The changes in selection evaluating biomarker primary to for into FEVX. trials. chloride as will

that expectations Kalydeco in responses have Kalydeco and sweat recall asked sweat Please was we to X and Phase development results, a patient’s X are program Phase levels that that a to results findings. FEVX X for often as shown serve normalize able strong correlation We chloride Phase guide. believe can about chloride the

have programs, our the Phase we clinical To clinical support of product. completed our full drug trial manufacturing X lyophilized

also identified have activity. work have commercial clinical key We and completed supporting process X Phase development manufacture

that patients increases nonsense functional protein have We from ELX-XX mutations. demonstrated with organoids in significantly CFTR expression

these have human We shown systems Phase clinical in and these healthy results bronco program controlled believe fibrosis. data We trial epithelial was also to cystic in activity X restored Ussing CFTR derisk our chamber levels. where

in top trial to initiated Europe our and a received X grateful we and have are we Quebec, expect cystinosis, have clinical to trial XXXX. from the funding trial Phase X this in non-dilutive Canada. trial Phase We cystic U.S., in In six-patient report results line from fibrosis for Genome Israel Genome Canada, clinical

hereditary Dr. the dose-dependent ELX-XX Paul in cysteine renal University for and levels the measure Goodyer, effect and cells, development principal investigator used on most in leader in disease The recognized recently the McGill cystinosis. Professor Phase of approved in cystinosis, drugs of white biomarker the will X blood the clinical is Pediatrics at of study trial. the

to study include optimal milligrams per doses carry ELX-XX ranging X.X an development. nominal order The to dose day three of kilogram in from will identify per further to to X.X

fourth report quarter to study cystinosis in X of data year. in top expect Phase We the from this early line this

subjects patients completed renal have renal support we with program, moderate have many where function, of impairment. impairment severe successfully renal In mild, cystinosis a ELX-XX study impaired the and in with

and our as results disease. to disorders, kidney preliminary clinical autosomal the date, support continuing our dominant existing expanding research other programs polycystic To such kidney

We completed expect the the meeting present a to from scientific study at results impairment renal year. this

novel develop new and compounds. to of our library indications pipeline our of continue We

XX on screening completed active agents of novel have We ERSG in read-through the our compound library. most

an patent by new life and advancing We our has are indications. global portfolio these composition molecules on all long of extensive opportunities there to multiple holds these compounds. novel rights pipeline and use of therapeutic new for with by administration matter addressing expand and/or in routes Eloxx of believe

provide to to activities. our opportunities now and ask on in kidney development of well an business as update the I’d as library for like our retinal you with David inherited molecules diseases, Snow

David Snow

Thank you, Greg.

the unmet will progress among fibrosis that remains individuals therapies. medical date. the breakthroughs this of mutations with in continue nonsense the is subset XX% CF need the to need ELX-XX exciting a population, XX,XXX and to cystic the with represent pleased all clinical there new XX% to high We’re that distinct a fibrosis, Despite carrying tremendous cystic

HIT-CF identified development over already to like with is which mutations progress with in XX phase. has and also I’d European now organoid who the program, CF nonsense testing. participating in mention program patients the The screening are further

our Let the expand a are for opportunities of me and library on compounds. ELX-XX we now exploring additional bit

such Greg and nonsense an ultra-rare disease, enable in the evaluating need area, for both suitability very in While ERSG our our continuing as this in polycystic development company’s completed the kidney the disease, results a mutation other kidney mentioned, segment. development support clinical autosomal cystinosis library ADPKD. current renal there’s medical or we and programs diseases, dominant successfully of the high study compounds unmet development is To of preliminary as impairment a

publications as – we variety that substantial XX,XXX mutation and there beyond have the of diseases represents a opportunity a about nonsense-mediated ADPKD mentioned ERSG of as are targets, component. nonsense early with it’s estimated alone. mediated in a disease also patients wide U.S. that many potential showing We’ve orphan in inherited number across with X,XXX academic nonsense a

kidney currently disease the and next such opportunities lung, evaluating inherited are the multiple eye. organs, of across level We as the

unmet need space, disease the more in beyond has XX,XXX ciliary PCD, dyskinesia cystic globally. in individuals important fibrosis, which we than instance, primary population For significant another pulmonary a find inherited estimated at nonsense or

mutation retinitis with than early improve pigmentosa. the the million started rental by Eloxx X,XXX ERSG also has to inherited nonsense portfolio the Usher potential in potential affected lives Across mutations. individuals We’ve more individuals with nonsense represent IND of the U.S. enabling that the disorders these or X.XX more programs, work than affected

Importantly, a more the available. unmet any, and medical and diseases severe momentum business active rapidly of few, update look phenotypes, development continuing these our to developing expanding We are represent driving to the These and variety you specific portfolio. often disease-modifying on need if there discussions. significant forward behind are important populations treatments

turn to over Bob. I’ll the back now call

Robert Ward

Officer, the now you, a I’d quarter second review ask of results Chief Weaver, to of Thank XXXX. our to David. provide for like financial our Greg Financial

Gregory Weaver

Thank you, Bob.

fund XX, and top totaling we believe line in of first equivalents had through cash fibrosis and planned and operations As XXXX, in current maintain cystic company the into cash, securities our company’s XXXX. cystinosis quarter of data the the million, $XX.X and operations June will XXXX marketable which

ended million, of XXXX. XX, million, of quarter recorded of to million, compared a which to or the and share, QX a In XXXX, for R&D. net same XXXX, loss the $XX.X of $XXX,XXX per net G&A or period expense compensation as a was share June $X.XX $X incurred million recorded stock-based the non-cash loss we $XX.X a $X.X company For $X.XX to

period same the R&D $X.X $X.X totaled as quarter Second expense for XXXX. XXXX compared in million, to million

driven studies, Quarter-to-quarter, increases were our Phase study and our Phase CMC X and renal along preclinical preparations in expenditures X R&D multiple clinical growth by operations. with trials, our in for

for the in $X.X compensation stock-based of to the recorded quarter-to-quarter quarter G&A, to G&A million, our change for as compared and The growth costs, reflective XXXX in a significant the XXXX in was $X increased increases decrease XXXX. G&A second by headcount same non-cash expense in due largely salary-related to fees offset period period. was year-over-year million professional

loss six driven $X.XX For the $XX.X a $X.X million, in compared the was expenses. June or period same incurred million of increase of net a for effect XXXX, the share months per The net $X.XX net of R&D per the by or share, million, ended company higher loss as largely in XXXX. loss XX, to $XX

to and million XXXX operations. of to R&D XXXX, $X.X million, expenses for compared R&D expense our was $XX.X which for $X.X million. recorded clinical increase stock-based in XXXX the million The in $X.X to Non-cash of the increase were of $X.X of expenditures six was for first growth million and to same months first primarily totaled an G&A compensation year-over-year months as R&D. six period $X.X in the due preclinical million,

months the for compared for the expense same six $XX G&A XXXX XXXX. was period in of as to million first $XX.X million,

total purposes, approximately million. XXXX modeling XX, outstanding June as XX.X of For shares was

call the turn concludes Bob. to financial the This I’ll back comments.

Robert Ward

In and this first-half efforts clinical clinical we Europe, are development the Israel Thank conduct track data cystinosis, in both our to to our to fibrosis expanding and top X you, on the line trials cystic of and year. Canada Greg. accelerated reach XXXX, Phase U.S.,

Phase X pleased in of with Genome is Genome from that and patients the support very trial Canada. funding our Quebec non-dilutive clinical We’re enrolling cystinosis

the from this expect fourth We in to early year. top data line the report cystinosis trial quarter

Cystic and been Fibrosis We’re pleased Phase Foundation. clinical is in the our for the cystic that IND U.S. fibrosis trial that open by our endorsed protocol X has

Hospital Medical Children’s our lead Dr. will that Eitan serve from Uluer Hadassah global lead the U.S. the will gratified Center Dr. trial Ahmet Boston We’re investigator. as from in and Kerem here the

of we’ve by well basis as the generated and extremely from scientific of of mechanism the understanding consistency breadth The the ELX-XX the of and as expression encouraged We’re action. cystic and changes fibrosis ELX-XX, first data agent in to mutations. advanced messenger substantial bearing protein in have the CFTR CFTR read-through activity RNA, is only its organoids in patient-derived demonstrated increases nonsense FIS

that believe ELX-XX data of meaningfully our Phase We cystic planned the derisks consistency study. the fibrosis X

levels believe also Kalydeco normalize is We a Phase guide. findings. to strong a in that responses program sweat Please Kalydeco’s that sweat the Phase recall chloride correlation demonstrated and as to Kalydeco chloride was development that useful FEVX able patient’s observed XI X

fibrosis and data line year from top Phase studies our cystic in X planned this report both cystinosis. to track on We’re

have and we as preliminary that their encouraging potential of levels supporting development. tolerability therapeutic several compounds read-through data our to activity, molecules these continue believe We portfolio of as ERSG novel well demonstrated of advance

our best standalone portfolio in identifying Snow shared both our across indications. to programs. program partnered David As with activities new and for the development clinical leads generating We screening anticipate enabling continued progress expand driving and you, to is the data

be Canaccord portfolio our business at will We Genuity geographies. in here and both library the one-on-ones across Conference active and development making very Growth indications a presentation variety continue hosting to in be in Tomorrow, Boston. a of we

for you quarter open us Operator, questions. our Thank forward much. to call for you may progress. our update call. for We now continuing look to joining the you second earnings Thank very

Operator

Our comes question [Operator Tenthoff from Ted Jeffery. with Piper first Instructions]

Edward Tenthoff

Thanks go My detail that. these the Maybe little in goal because to of earlier find for do discovery commentary of your a indications? really has pleased of novel update. question And different terms really here efforts, could some stage the to hear on actually for was with agents the the to you I that? is breadth into just

Robert Ward

you’re molecules. Yes, Ted. we a have you, Thank Yes, correct. novel of library

overtime, different XX most of the have compounds view active. synthesized that been XXX over as So which we the

of retina And is at the think so one diseases or of delivery, talk the administration we’re alternative looking about questions as intravitreal eye, of routes you the Ted, of the administration eye. remember, of the the for of when route we is, so back

pulmonary other of for or in When the we we’re topical opportunities cystic route for kidney think fibrosis, think systemic administration, that’s the we or there’s pursuing, delivery.

better unique one different another. that for than their reasons indication believe or molecules there’s of also We why disease because characteristics be may suited

the able So and novel ask I we’re And such we in of situation to indications. think development a David comment. molecules fortunate across how drive of a be I’d having the number different of large molecules then of or question to looking will just Snow at number

David Snow

we’re to the to and at Phase reassuring that very in have a And We there. provides eye. have the in the X that of also compounds been it’s Bob, at see that, to the Yes, the areas, have and these I we long looking lead add with look different development. us IP can kidney portfolio, need we’ve opportunity. each this those areas subsequent progress what really program we organ potential nice three would obviously, of opportunities as the lung, made

Edward Tenthoff

Excellent. exciting Great. much. so Well, Thanks progress. really

Robert Ward

In approved Yes. many And, to to Ted, cases, of or of primary drugs polycystic Usher no cystinuria, these it’s Syndrome when as ciliary remind retinitis there would well, different you populations. the disease, you autosomal are citing I pigmentosa. whether dyskinesia, ability dominant think these currently bridge for

All identification of who understanding with molecular and active very patient groups them have help the advocacy basis. the

as well expand the real well. therapeutic to a think expertise in-house, in there’s that for individuals greater who different ways we companies as for have partner look as opportunity areas or portfolio So to

Edward Tenthoff

lot are a – side. sense. collaborations those so from Yes, And makes important again, scientific are the of

So great see. to that’s

Robert Ward

Thanks, Ted.

Operator

from next Citi. Joel Our question comes Beatty with

Shawn Egan

Good for morning. taking Thank This Joel. for in is you Shawn Egan question. calling my

important sites? systems set cross can clinical read-outs. upcoming that specific previously there particularly assessing immune are then for are meaningful, you that So other But or follow-up, there barrier organ enter you safety, blood-brain does or are privileged the And the as any expectations remind readouts a efficacy watch? are ELX-XX to when

Robert Ward

right, Yes, Shawn. you’re

with into at population which to look the enable to dose" study then and of we a clinical development one studies and tolerability safety across pick establish doses carry range patient when a program, Phase range go begin to X So as every "the forward. to

for look for we to taking be fibrosis, drug-drug when that want at be that there’s we individuals cystic would example, So may who concerned. already other be drugs, sure no interactions

ensure development is area concerns. ELX-XX the one course, of to does So, have during not program that’s drug-drug explored or interactions that other

through the talk we drug function, kidney. the renal is cleared When about

looking change questions to or study, that we’ve one observations change for consistent basic in pleased had the is, very required modeling that were results expect function surprises. would renal the think the – in predictable? with So our I are with we any renal And of impairment and dosing from we’re one in date, is not unusual

So this will a we’ll a of individuals and/or both on in to would to work, be normal function us that able the kidney to demonstrate that around ability based drug range enable dose-adjust safety preserve way kidney with suggest impaired wide in in the function. further

think emerging is program observed serious potential it I and events way for has successfully. date, through in with to clinical the go to one being that that adverse all be ELX-XX the suggests registered profile no the

the the cysteine around to next date. I So white safety, we’ve think FEVX, change able And sweat in in changes observations very both be that we’re assess. is to tolerability understand cell efficacy the and levels to really, data step chloride, encouraged by preclinical seen to blood

are there within crosses that we are the with And at CNS could se, disease. blood-brain molecules barrier. academic do library we’re CNS-driven may our drug library. have but already there our that the believe do we’re molecules that you We currently X, per targeted MPs drug aware from Now targeting look we other be for aware not when be portfolio, publications towards

have considered particularly of much think you a if that system, of we of time immune as immune the with looking in at when of privilege, we intravitreal is concentrations eye, to does production appropriate the an period it site enable understanding proteins. privilege. about immune a restoration intravital would But read-through relates administration for sufficient And fundamental for administration reason that, believe so that of essential not often, tissue agent our the

question be the concentration to of the as period we’re the proteins. production think what restore to able tissues, drug to really these we So essential study different required about is answer looking over of of

So advantage answering questions in able many to before. have ways, it’s being work studied the on been of that

much in what be trial, there the for it’s see as from think we models date, to characteristics. run. When the a dose So dosage data I with they consistent are a impairment, we then question response we’re do the predict with adjustment the we consistency ELX-XX, to the the modeling. renal of being that are results, variety very run trial pleased what And of reserve able that of might with other demonstrate

Shawn Egan

then the study that In that study, were in were Thank you. a follow-up. ADPKD provided in any brief And patients there that enrolled I further renal development renal appreciate issues? as really read-through that. impairment to any

Robert Ward

new three-step at When a process. indication, a we developing look really it’s

the interest screens the generate read-through variation. at amount first, So and that the of look in on we of genetic at vitro specific specific gene we

So amount run of assay a basically assess read-through read-through. we to the

being amount allow of protein in animals that into move has believe there us for a results level is that step reason a produced functional the potential benefit. to clinical that intact assess, models cellular or The second of to is the restoration to

vitro course, and and of which both the for cellular mice have we fibrosis, cystic into and, transgenic screening a of done think we stepped organoids, did then is other. So in the example, model

different are as the We renal of This With the in have do progresses, that have of individuals they ADPKD. population, but with is levels study. not ADPKD renal diagnosed know that the renal individuals impairment, impairment disease increasing levels impairment. particularly

to part impairment disease. adjusted is handle do to specific it. how would that relates understanding a the But studies the drug very we of So on important need to be that our renal ADPKD view enabling to

Shawn Egan

clinical question. provide update know And the early an identification final Perfect. of still I just many And pretty study, worth can efforts patients’ you include? patient it’s but how into frame can the XXXX my maybe proceeded? update you how data will on then have days also,

Robert Ward

have the And as the or of We’ll provide update whether here later cystic U.S. cystic we’re understanding Institute particularly really Fibrosis Cystic Fibrosis Foundation, molecular fibrosis, the Research Foundation or fibrosis Vertex top year. Fibrosis Cystic fortunate in disease. this a all a the in remember, European catalyzed line groups it’s Cystic or that, these

many the what and have when genetic cystic within of aware community talk already databases. fibrosis So the variation in individuals be many, have we GXXXX, about volunteered they to are included may

cystic So fibrosis in. sophisticated to a is work from very area identification, patient

Similarly, a an and and individuals, been sequencing with cystinosis, as University, be cystinosis developed that Dr. identifying so part McGill disease. Paul can molecular Goodyer have important of

it’s have terms genetic to advanced many know their reasonably individuals currently how the as as cystic not identification to own sequences understood of there both understanding are. patients You well patient understand variation. is while cystinosis And in fibrosis, and

we’re program, understood with Retinal which the way the diseases basis. Blindness. over very be helping started and Ocular working a They retinal pleased a the we’re Foundation Fighting to at is that to XXX the support program, molecular for to in inherited My better be Tracker Now

helping catalyze variation from to and each these substantially is then assessed a genetic to pursue Fighting these groups of Blindness which, has possible research it’s to individuals why having their a Foundation So contributed perspective, database, establishing studies.

also contributions of patient of So groups, without be academic advocacy without and institutions. the it the couldn’t participation patient many done

Shawn Egan

you Thank it. appreciate so I much.

Operator

SunTrust. from [Operator with question comes Instructions] Our next Joon Lee

Joon Lee

a bi-weekly my kilo also, Hi, have bi-weekly the NACF, day. at X.XXX daily MAD it appears milligrams any to is accepted the study. on you be tested at per you’ll data Do milligrams the per I title after PK testing X.XXX have But follow-up you And per top in Based exposure the the expect for kilo And be Thank we exposure? that. comparing you. should ELX-XX line for the data thanks presented understanding that, CF of conference? question. to for abstracts

Robert Ward

American just at Yes, North we’ll presentations. Conference, Joon, as Cystic Fibrosis three reminder, have the a so

new basic mediates have on to the presentation in workshop emerging to and RNA, read-through we’ll therapies So, mutation oral example, correct protein for on CFTR translation nonsense messenger defect, an CFTR and the increase how function. ELX-XX

will in study, the single as additional safety abstracts pharmacokinetics and and safety as dose the ascending the be Two well study. in pharmacokinetics MAD and covering tolerability

the So data American North top be anticipate We present well. at discuss our as those able Cystic Fibrosis. presentations will to do this year line being and

Fibrosis Now remember, at in the American the North October. is Cystic of meeting right end October,

the it’s – a us for addressed to in going Could be early little what about to So cystinosis think cystic bit year compare MAD in SAD us, help Joon. you at a understand both And studies? the Williams studied and was then Dr. doses range that’s how fibrosis. better and these dosage

Gregory Williams

Sure.

in comparatively the kilogram. MAD, life multiple hours. to per that per SAD look studied pharmacokinetics, we understand milligrams at we in the study, we milligrams eight And of is of up kilogram. doses So to half the up at studied When it’s short, X we ELX-XX we X.X dosing and about look

cystic moving to dosing, we we’re that exceed volunteers we’re healthy administered the fibrosis studies. exposure using and that consistent over ensure and overall lower in more doses, daily our With when has cystinosis, time. So are does daily daily exposure not dosing the been towards exposure better so moving to prior towards

would cystinosis, the MAD both exposure associated in So be with twice-weekly the study. with and the consistent CF exposure, overall

Joon Lee

go Yes, ahead. Great.

Robert Ward

I was going had another to say you question?

Joon Lee

Yes.

are like that organoid need or you. do there that out on others basket ophthalmology it mutation you are CS ELX-XX organoid similarly But more the appears say, space? for approach, nonsense in amenable nonsense So, Will the assay. mutations Thank is conduct every a exist your to And credible assay? other based there, assay diseases, the using to new studies some story. to for I’m than fairly

Robert Ward

Yes.

think, in Joon, X% total genetic that’s on And of the I of focusing variations study a GXXXD, eight terrific Vertex believe When then different included, development study. that a pioneering ran work Kalydeco about second which patient that in you first was think I population. question. fibrosis, cystic led

and at early cystic then life. very regulatory remember, because asked because occurs was for study establishing younger fibrosis to and in ages, we’re onset expanded younger patients to moving the individuals typically, Over label and a for populations, safety time, older from with first, cystinosis, perspective, efficacy

So genotypes data. by has combination the label then age. becomes has why clinical pioneered included expanded a Vertex to different expanding of and And also progressively preclinical

HUB, Utrecht But organoids remember, collaboration intent European to The of Medicines which the prospective is University, in a a by funded HIT-CF, nonprofit importantly, most run is arm collecting the with the done with European Agency. Union, trial.

when talk or the in organoid it’s an today, first of and So clinical we organoids, about later. correlations people may develop a other been have had who trial individuals

based data. the many of on And organoid so correlations retrospective are

So HIT-CF will response collect trial. first, run the in clinical then a organoid organoid to establish and test the drugs the laboratory prospective

do of test a So expansion? label basis be a ability We test power of from real laboratory a organoids that would establish say, this regulatory the if perspective an predictive believe of the will to be are represent organoids. that the results could positive, that this

population, that think variation are a with of are other do some a research, of There’s variation. genetic There’s because high people four we area there’s genetic we have of being variation, GXXXX, So rare. example, as there X% variants earth for very important population. with where planet one only on as know, quite have that patient identified far the representation that variants been

So small trial. a a consider running that’s very population to certainly

ability that extend the there individuals, bridge So therapy established. – important is it’s all very to to these to

directly some a also a establish And program, more rare these represents way really the into the genetic It is very potential engaged diseases, to but this so take certainly the FDA with of models closely. EMEA to following and advance. HIT-CF important

Joon Lee

wouldn’t assays of to more reasonable. think But like mutations of basket are ophthalmology? Thank what That about of that’s sort sense every exists different quite similar Do the and organoids for burden diseases you. Great. other carry I amenable trials to a approach? have nonsense doing makes of lot where more in and you a

Robert Ward

results show in correct, you’re do protein about of How you the the an Joon. No, proteins, amount you question functional important really we of different right, change? do study same how protein produced that talk first the variant? Whenever being each unique raises

trial. at studies, and where clinical the the in are studies eye, are program, We LCA animal program humans, translation Sparks’ functional variants across developed. that the very vitro ProQR’s readouts then see in good and diseases, similar in believe looked would protein So whole there’s translational path or models there RPEXX you we other a there or being variants, some in inherited protein of whether where retinal models are there cellular

developing translation research, of the So important the serving really and models. up as an Fighting groups connect to Foundation but support is foundation many Blindness also catalyst academic key with

for primary in been these organoids ciliary Now are ADPKD have for are cases, dyskinesia, or in development and validated. previously there that

that think important for an PCD, for role. do we organoids ADPKD, will So play

retinal disorders cups we right important both be translation organoids, now, have stage than inherited a but more do been models. will think to For eye substantial advanced

Joon Lee

you. Thank

Operator

I’m call not Robert further this Ward, at like Executive I’d over the Chairman and turn questions Chief showing Officer. any to time. And back to

Robert Ward

everyone, thanks, joining our Well, for call today. for us

are you tomorrow Boston, of For those in talk who opportunity to at one-on-one.

be call Thanks will a next on A.M., presentation X next earnings seeing look to for our We participating. doing and all you quarter. forward at

Operator

today’s presentation. Ladies and gentlemen, this does conclude

may a disconnect, wonderful have and now You day.

Eloxx Pharmaceuticals (ELOX) 9 Aug 192019 Q2 Earnings call transcript