ELOX - Earnings call transcript Q1 2020 | Eloxx Pharmaceuticals, Inc.

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Operator

Good afternoon, everyone and welcome to the Eloxx Pharmaceuticals First Quarter 2020 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. Please proceed.

Barbara Ryan

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

me Matthew General MacDonald, of Counsel; our Tom Haverty, and Greg Belloff, Joining Stephen Medical our Chief Vice Dr. are Finance Officer; President Chief Officer; Research; Dr. our and President Executive our and Neil Williams, Officer Operating our afternoon this Goddeeris, Vice Chief Accounting. Dr. of

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

Williams, Dr. It of is now my over turn Chief Executive Greg Officer the great to pleasure to Pharmaceuticals. call Eloxx

Greg Williams

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

We scientific for committed programs our ERSG remain advancing library. our to and clinical

we believe trials temporarily global clinical previously to That COVID-XX for and value order Phase plans and from healthcare patients. announced trial will is families, the in and and of substantial an fluid response the to we company. This which line is unprecedented employees, paused ELX-XX to our clarity we and that inflection safety our be a Our workers, we on to data you update cystic health their reach proof-of-concept been fibrosis protect as these reopen. our clinical top our situation and gain will point highest the have in sites fibrosis for said, continue investigators, to pandemic their cystic program, X priority our

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working are resume ensure as Phase our clinical and quickly program with our possible. to investigators We closely sites that and can as X we complete

and provide be safe We in shareholders medical high the on while options most continue to mission with our manner. treatment focused value fulfilling needs to to delivering unmet to patients expeditious

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the capabilities in strong research, have with and I'm on and have that confident the our needed basic goals. We highly resources clinical affairs deliver regulatory expertise drug experienced to a and team we development,

top In CF been interest trial has tremendous. clinical groups cystic these patient fibrosis, extremely are we advocacy and the pleased expressed support we level conducting trials at sites and are that from and top trial of sites, investigators,

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Goddeeris a Yesterday, our as in May programs. disorders disorders data update from program the inherited meeting, moments, well our you we Dr. will on inherited retinal this progress retinal X, and few in presented at as ARVO and ADPKD data virtual the

the accepted our still in abstract late we've Fibrosis North Conference been for global Cystic ELX-XX fibrosis October. for at We June, has this presentation to at had in to data Conference the for due Cystic in plan been Fibrosis COVID-XX and canceled withdrawn abstract. cystic American European ELX-XX the While the present pandemic meeting

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provides on for renal dose function. adjustments The based necessary data modeling

eye intravitreally show production encouraging on ophthalmology longer achieved model. across results milestone We've periods. formulation proof-of-concept We our with to animal when and built in across compound compounds in sustain which strategies efforts, demonstrating ERSG an continue multiple milestone important advance this our an restore ongoing that our in to exposure our work injected the team treatment can protein

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enthusiasm and therapies clinical high for potential mutations in there nonsense We are developing our a is programs new and tackling scientific the for in the as of the most of great the interest community community. as advanced well company and business challenge level

therapeutic and accelerate appropriate footprint our continue pursue expand will to We progress. to partnerships our where

of quarter and cash runway the first and XXXX cash realignment through XXXX. our the ended $XX of million with the with in cash extends end our equivalents resources, We of

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of recent progress in discuss our disorders. inherited I our would ADPKD Research, to now like and to Goddeeris, President ask Dr. Matt Vice retinal

Matthew Goddeeris

Greg. you, Thank

programs We taking ERSG within continue partners all operate molecules, working global authority necessary to library preclinical for with steps efforts to our pandemic. advance research advance recommendations combatting COVID-XX across our our to health our the while of

to today accepted generates via ELX-XX target of manuscript the of Experimental scientific This read-through fidelity happy indicates Fast found the journal's protein has therapeutic Pharmacology that of tolerability this announce Therapeutics. for the our the while stop codon end the premature version of codon This Journal publication preclinical healthy that manuscript our stop with are section without integrity maintained. clinical is stop codons, titled, that of read-through preserved inducing profile premature mediates website. native favorable by been and is translation protein, sets. We at stop read-through ELX-XX manuscript the consistent can within transcripts Forward The and across be with data exposure of found of ELX-XX, prepublication codons demonstrates

are Our ocular preclinical efforts in progressing. renal and

using genetics the our focuses a medicine of on applying unique versatile to assays enable Our understanding approach. of protein to read-through evaluating function our with efficacy on enough team disease and each personalized focus

by PKDX the gene. either copy born are ADPKD or the described a two-hit with single individuals As defective PKDX hypothesis, most of with

ADPKD disease, disease. the In hit. and will throughout sporadic primary or life, a formation the a leading is For this potentially trigger renal end-stage kidney, cyst the second mutation hit genetic to

We whose dose-dependent common those reporter primary common across most have hit with a individuals demonstrated our is PKDX Using nonsense alleles alleles. assay, ERSGs PKDX already now dose-dependent the read-through allele. a are most across the have on read-through and observed we focused

We and our efforts that this order confirm we an observe information in has functional to growth. are the read-through impact formation cyst now applying model to on

platform in characteristic that IND of recapitulates manner ADPKD, encouraging organoids a stem from with a of or for model. diversity cyst submission. patient direct evaluate patient-derived allele, induced have number. results additional impact the of fibrosis in cells can ADPKD approach utility common disease cellular PKDX highlighted a towards ADPKD, cystic metric Similarly, We the of cyst we derived pluripotent state. kidney assess the a a translational have on advance demonstrate These Our differentiated generate function the observed read-through a to organoid positive technology results, cells reduced be of Using models of cystogenesis. that meaningful results has can progression, and and to the the with organoid intend most nonsense

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of read-through Our assay library has in using compounds demonstrated production intravitreal model platform, protein and tolerability, via our animal restored intravitreal acceptable in an dose-dependent injection. ERSG vitro

which in were for this these Research the As results for some presented of at ARVO virtual mentioned, yesterday stands Greg year's meeting, Ophthalmology. Vision Association and

activity model with Intravitreal oculocutaneous of present the a model, molecule in a X human albinism gene, nonsense entitled agents studies in administration OCAX mouse type demonstrate mouse nonsense a functional mutation a which occurring presentation, describes read-through in of results our Our small albinism. in mutation naturally form in

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opportunity Our extend achieve approach of team totality To the actively the desired duration formulation. the sustained-release is to the our working of delivery, reach intravitreal to the read-through the retina. provides

the exploring are release with date, to consistent polymer and three technologies are by target encouraged profile several one to rates our are which of vitro in controlled-release months. biodegradable achieved We release

as read-through with is data we in potential efforts disease-causative sustained-release PDEXB. CEPXXX and the against exposure tissue MyoXa, this mutations such our formulation nonsense observed and genes ongoing coupled USHXa, When

or encouraged could across retinal to disorders. nonsense-related restoration ERSG the intravitreal that inherited approach visual We function critical provide proteins are of preserve restore

like ask to review would Steve Accounting, of results. I to our provide first a and MacDonald, VP Finance our XXXX quarter financial now of

Stephen MacDonald

Matt. Thanks,

top will and line XXXX, through and $XX XXXX. equivalents including securities, cash As of million, the company the company's end cash, of cystic total XX, reported believe through marketable data March fibrosis which in the operations we fund of

net million, quarter or million same per the compared $XX.X as the share in company $XX.X March ended or loss of net a XXXX. of the share XX, to per period incurred XXXX, For for a $X.XX loss $X.XX

entered and of first expense compensation $X.X stock million first million QX XXXX fibrosis $X.X toward we Fibrosis from quarter QX with an Foundation $X.X G&A $X agreement with X second restructuring Cystic charge $X.X with into associated we up million study. to million In of we ongoing our XXXX, payment payment. which funding compensation. R&D. XXXX received U.S. In the provide was our realignment, our contributing the the $XXX,XXX The in totaled Non-cash XXXX, cystic to stock one-time allocated to included million results $XXX,XXX a $X.X of received non-cash of and in to million operations Phase

First and The non-cash XXXX expense for for XXXX. in a $X.X expense decrease in million G&A totaled quarter increase to for million was due driven from compensation an expense $X.X period to was for quarter of XXXX first compared period. costs. $X.X by XXXX compensation, in same XXXX a million and $X.X salaries offset the million, lower lower of headcount and non-cash decrease quarter-to-quarter professional R&D period stock fees infrastructure-related R&D by related the stock the portion same the

quarterly to March, and year our move that efficiencies. effective the Given burn reduced realignment we of throughout reflect cash expect decline headcount, program in spending, as elimination the sequentially the will non-priority company's targeted we

in majority in will G&A, spending. reduction will cost there the be of savings While R&D some fall our

that will our low fairly quarter reach burn stable rate remain and throughout cash in of its the expect We fourth XXXX. XXXX

late was we apply March. U.S. applied CARES Paycheck we which into of will XXXX, of eligible a signed received and the for, Program, the Protection the via proceeds based PPP $XXX,XXX partial forgiveness, which loans law April using SBA's XX,XXX,XXX. Also, for interest are for headcount, your Act component approximately In on in to XX, shares a at payroll, bears other of end of of loan XXXX. specified rate were costs. common purposes, is for maintaining The stock at total X% outstanding and the as balance our and remaining repaid of the commencing be for modeling XXXX March of loan the

the first quarter Greg. back This turn and financial the comments, concludes to I'll call

Greg Williams

we and clinical cystic you, point will to believe company. report fibrosis data, value Thank Phase highest inflection for priority resume in top Steve. line our the to which our X major be It's proof-of-concept trials a

clinical this have in that data Cystic sites trial North on and the soon ready ELX-XX We possible. as Fibrosis on complete our look are American as October. our forward presenting We to ensuring at to investigators laser-focused Conference late we

Beyond read-through activity advance of portfolio cystic further to we molecules. and of supporting encouraging continue Several novel levels their these therapeutic development. compounds our of fibrosis, ERSG demonstrate tolerability

Dr. look on to the you progress. and journal's us We data meeting. Pharmacology we be with the can inherited from and May forward recent our manuscript some quarter scientific inherited of presented our ADPKD scientific our some Fast our our from shared and in to you you of Yesterday, section Journal joining on viewed latest virtual preclinical of earnings activities for the preclinical disorder Therapeutics update Xth, programs in Goddeeris Forward of that continuing the the Experimental these retinal disorders. retinal ARVO first call at We're pleased in data XXXX website. thank

Thank you up may much. call you for Operator, the very open now questions.

Operator

Our from [Operator Gilson with comes question Canaccord. Instructions] Michelle first

Michelle Gilson

just give was hoping I clarity. you could us some maybe

the you the the those different and cystic cystinosis fibrosis cystic read-through just in well, differences just to what us of data? then, is important of is you ELX-XX in remind de-risking fibrosis And you program? why can respect maybe cystinosis, so and these well trial as are Given with from data all some Thank for to design remind results most us why you. the saw in as

Greg Williams

Michelle. you, question. Sure. Thank Appreciate your

so the excellent cystinosis first the that patients. we or anticipated that news. in of This We through pharmacokinetics nephropathic employed the daily-dosing have in exactly volunteers our time couple dosage with things. cystinosis, regimen we firstly, was that different a saw So three healthy got is trial, were doses

forward a saw We've that approved of an the design of peds. committee. data we issues continue data. go The second cohort review trial patients with there decided because included us independent clean since to interpretation study safety with the a Secondly, safety They to were trial profile. and complicated that reviewed were not paused that the by

saw second cell significant statistically first see, we patients. reduction the we a the which perspective, pharmacodynamic not cystinosis in in in measure dose was efficacy effective We dose, an that that blood of ELX-XX key that did From the saw was cystine, of study. nephropathic the white

between the those ELX-XX cohort period lengthy because patients time. of and third some that beginning and beginning drift The that been administration and cohort treatment for There excuse of was group. for second third baseline of somewhat had mixed was over a across administration me, the of third group for time -- second treatment

signs we because issue So are the the a nephropathic did related directly We activity. attributable cystinosis encouraging ELX-XX. as exactly associated severe to are We though primarily the some with particularly reductions and with profile. data to patients design safety we They biologic very anticipated. see overall, clear of drift favorable pharmacokinetics saw baseline saw

So those for program. CF de-risk to these all help of our reasons,

group, go advisory what results were nephropathic for forward. these cystinosis we with potentially mean, patients conversation CFF very In a our as fact, they about in enthusiastic

to chloride. our pharmacodynamic going study, be way bounce assessment cystine in contrast in It trial, on chloride white drift. does. cell doesn't to is primary patients Sweat nephropathic around blood really really cystinosis doesn't efficacy CF sweat Now, the the the

different doses. dose issue at cystinosis, going recur They design the in with the we CF not to in three study In that patient. So escalation cystinosis we looked within had trial. is were

then kilogram treatment, We're X escalating dose per X.X up X.X milligram milligrams starting to milligram it per patients per for at those kilogram. per trial, week kilogram. In milligram kilogram in per So a different X the escalating treatment, a kilogram, doses per was X four at weeks. looking to kilogram, duration kilogram week's milligram two milligrams we're X.X milligram CF of per to for X.XX for of [ph] up After a and to escalation.

is cystinosis what is we nephropathic see the not expect very the in can that So cystinosis in trial our in inferred cystic present pharmacokinetics and fibrosis drift CF the had the issue activity trial we with trial of be CF cystinosis of the trial. the and going to indication baseline in the biologic and be to clear supportive safety to from directly

Michelle Gilson

And if you back might, can running? And one that that to thanks. How follow-up. start data? just get going there monitor any and collect up and quickly, anticipate reopen, I changes the patients study cystic you Okay, sites to fibrosis when are in way the be you

Greg Williams

COVID fantastic timing huge global So result in now distancing the only appear the we're reduce is I that not hospitals policies. the time. starting to that starting but COVID It's anticipating established verge great open are social this really starting to the the any manage There It's clear. able that you great to up. that changes be are know, of or on the countries entirely not not to has everybody they pandemic. countries this clinical on patients, unprecedented industry, COVID the as of protocols their resuming trials, mean, the a consider changes dramatically said, in Europe, load societal the to for It's us. fluid, either but as has starting been are in Israel verge to just not is to reduced. and of is patient a U.S., They're

will I only deliver to our as soon is being, top complete line as for and we you know priority to the data So keep tell possible as enrollment, more. you highest that that we informed time can

Operator

Janney. question Our comes Zhong from with next Yun

Yun Zhong

to patients but once believe X you able to patient complete first able So don't have how have reopen cystic tell, in you about of the enrolled Phase able you the disclosed to the that been studies, are study, number will studies are you in is question fibrosis. be I enrollment? soon the

Greg Williams

the Yun. call. Thanks for Hi,

we enrollment but able resume we what that highest really our is it's you tell with say can't is As will shared I we to exactly I to going unprecedented enrollment, a to look can when line just data let time. meeting and Today Michelle, it to forward be certainly know the top more you priority NACFC do as in get presenting completed know We'll report October. data. forward we at

Yun Zhong

that a do the in you Will maybe those you I timing to got still happens or question expect enroll can treatment, enrolled included who yes. than with Okay, regard patients already have what been probably planned you'll is, the of have analysis another those the what patients part additional probably in understand those set? data that but so patients? uncertain, study, be patients to you sufficient to data is had had

Greg Williams

data the so included approach far be in forward certainly additional go and some set fill know collected the Yes, that when we we will we'll of completion we've as our as more on and you in data enrollment, details. of we

Yun Zhong

you the thank for answers. Okay,

Operator

have [Operator We a Janney. do with Yun Instructions] Zhong follow-up from

Yun Zhong

for follow-up you taking Thank the question.

want you the ADPKD confirm likely level in for the that plan same the is the can just you the that so that have ELX-XX ADPKD as you program tested of the will to So program, comfortable efficiency expressed get kidney? use read-through -- already the given And with well? for kidney sufficient in protein you still are

Greg Williams

for opportunity show in safety. again, We've for we in follow-up. other that library. Well, and our would use a for number them compounds we large Yun, favorable It's the of and read-through one your kidney read-through of characteristics compounds thanks anticipate ELX-XX got not with there's upside all a of or the that clear whether will ERSG

we now as with continuing sorting We are clinical we in trials. trials of would compound approach IND-enabling out our which go process the and

Dr. add there? Goddeeris, anything to would care further you

Matthew Goddeeris

within know we're found choosing we so this kidney does the just this good that have its to but out as that right a kidney possible on results these to within mix, good this choice for be and the yet. it and may lead ruling mutations, molecule ELX-XX, we're read-through based the our has because and read-through activity molecule show you that PK point, ADPKD active against really molecules profile we exposure it also relevant not concern the Greg. from think At biggest is Thanks, I in

Yun Zhong

Thank you for the Okay, great. information. again

Operator

conference And back to at like any hosts. turn the showing over further time. to our I'm questions not this I'd

Greg Williams

really to forward. with thank to forward your appreciate reopen quarter Well, information we in It's Eloxx. and trials and as being to going a our we be very providing you Bye-bye. additional go We interest all much. much able fantastic very look to

Operator

Ladies and that gentlemen, presentation. today's concludes

and disconnect now have wonderful may You a day.

Eloxx Pharmaceuticals (ELOX) 11 May 202020 Q1 Earnings call transcript