ELOX - Earnings call transcript Q2 2020 | Eloxx Pharmaceuticals, Inc.

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Operator

Good afternoon, everyone and welcome to the Eloxx Pharmaceuticals Second 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 begin.

Barbara Ryan

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

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

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

Greg Williams

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

clinical ERSG to for continuing our our advance programs scientific are We and library.

under is investigators of global Fee application most families, COVID-XX submit at set Drug just CF the We response employees, in had trials and approval previously that to patients. fibrosis trial II high XX, We've as clinical cystic and were Phase clinical On Europe ensure the our the paused. importantly pleased while with announce possible. Israel level II our sites announced of currently safety User our workers, CF maintained we our health-care our to we trials health required complete to program, The for engagement we investigators, substantial ELX-XX been from fibrosis. that $X an FDA, designation resumed the to topline June had for proof fibrosis believe reach will treatment orphan of announce has point in clinical benefits patient disorders an certain concept we FDA to our be clinical the important postapproval, that US for drug grant for on ELX-XX expenses, people for used value designation pleased support designation rare priority confers Earlier for development US. for including inflection for benefits affect qualifies exclusivity granted funding cystic a for cystic is populations company. marketing the this were drug underserved trials several data highest qualified to US Our drug credits be phase or tax orphan the order remained to of Phase the XXX,XXX X for medicines of protect our trial temporarily X which years the week, that potential and Orphan the a than trial Prescription opportunities to clinical eligibility million. which for soon of their waiver that fewer Eloxx a US can can with in and in paused were pandemic as our that enrollment in program

clarity as to greater continue will update activities. gain on we We you these

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

signal clinical safety there been report no to trials. pleased have also are date We to in these that

X by in Europe fibrosis trial X program investigators focuses The enrolling second CF sites Phase the cystic patients on of for one Israel; trials, Our consists sites open-label to in our Phase program at expansion network. clinical Foundation the US. Trial endorsed funding X the US of and protocol in by part Europe, Cystic Network. the has the possible Fibrosis development In therapeutic Clinical made and by has Our our ECFS the been been the provided protocol endorsement by of

We again deliver The are we XXXX the also has X evaluating the may sites of other our to strategic capital priority, sufficient additional right countries accomplish proof we ELX-XX where flexibility enrollment concept to topline the data end patient clinical Phase have and is of be through to fibrosis. in key resources cystic feasible. which and in company run for believe

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

R&D continues disease Beyond as library team previously our our disorders. retinal advance kidney in ERSG ELX to had cystic ADPKD inherited from compounds you which or other for we such to autosomal polycystic additional fibrosis, spoken indications, dominant and about -XX

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

data, cadence publications there'll expect of a We be as scientific year. we and that move presentations, additional steady through this

Pharmacology additional detailing the in present late We August October. Conference American specificity stop and in towards manuscript published a has ELX-XX codon Fibrosis that at ELX-XXs Experimental North plan edition in The cystic data Therapeutics. are the of premature for to read-through Cystic Journal XXXX been We pleased scientific fibrosis of

in the at As In programs. ARVO continued a Vision or quarter update inherited we for and we from in Ophthalmology moments, retinal inherited Association previously presented few ADPKD Research shared, on the retinal will Dr. virtual meeting. the and program our progress disorders disorders our data Goddeeris second you during

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

nonsense the CEPXXX, the PDEXB or at last MyoXa, Society of of presented photoreceptors the read-through disorders other retinal alleles pigmental and most for demonstrating recently and scientific syndrome Usher data genes. Nephrology week evaluating are American kidney epithelium, We Positive year's common our impairment where the supported patients. modeling adjustment research the a renal areas of renal dose also into expansion other study as completed necessary The kidney mutation for nonsense there's data on based our from of function. high provides ADPKD prevalence such in of

Our ELX-XX new molecules screening programs continue to library evaluate ERSG to and novel for other opportunities advance our from indications.

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

our our our our quarter second and $XX.X the cash where ended accelerate cash partnerships XXXX the of cash continue resources, and expand equivalents progress. We the extends We through therapeutic to footprint realignment pursue XXXX. to of with runway with end in appropriate and of million

our are to We ELX-XX and advance activities well-funded and cystic deliver fibrosis pre-clinical to topline for of in ELX-XX the additional for data indications. molecules library in

Before I of over Goddeeris, programs update President Research, in in call will on the global turn we progress disorders, ADPKD to overview you our the navigating preclinical Counsel, challenges our an successfully Belloff, to Chief provide and with who Dr. Neil ask Officer the you I'd and diligently the pandemic. are like Matt and COVID-XX retinal how Vice of Operating of General to inherited

Neil Belloff

respective not Protecting date, With has working plan and have implementing our to severity We we have had work Europe during access paused workplace of with oversight our while disruption sites not trial that operations material that companies, To to US this of to travel the financial global the or made mitigation extent on crisis will business order from principal which operations trials, pandemic our a patients health remain and which adverse alternative speak predict you, operate and families the and time can Phase of with in Like government staff health and on safety been personnel in temporarily furlough and most of trial we clinical to paramount with hold. are employees guidelines. minimal the for effective and strategies and of risks and clinical We on diligently of had have the our crisis. CROs the on and pandemic. workers, lay our be business and the ensure off in workforce, the impact the Covid-XX of though accordance will Thank determined sites, normal and investigators. Greg. mind, permitting to cannot our our prohibitions to evaluate in with impact their current safety modifications Israel guidelines. regulations our importance patients Corporate meetings, any we were remain flexible possible health-care mitigate communication including to continue CROs, and impact and a Steve employees. ability future shortly, those continuously complying and in trials remotely, our for clinical our largely we operations applicable condition, by X perform and to period protocols even care

As remain focused program Greg feasible. Phase clinical II soon our completing on as mentioned, trial as we

when over relevant Vice Dr. available. the now would turn Matt of like We to I updates additional Research. will President provide call Goddeeris, to

Matthew Goddeeris

Thank you, Neil.

working advance efforts We research ERSG partners continue our with preclinical to library our of our across to molecules, advance programs. our

Therapeutics. the manuscript stop As stop of favorable premature mediates Journal transcripts demonstrates manuscript ELX-XX This and of that at codons August preserved end profile This Pharmacology protein has we the that fidelity codon a stop therapeutic integrity generates of read-through native tolerability and with Greg mentioned, that the across edition the codons, titled is without read-through target is our ELX-XX healthy stop consistent in dataset. our scientific Experimental inducing with exposure maintained. preclinical premature published read-through are of ELX-XX, clinical pleased via codon translation of found indicates protein XXXX while of been

in ocular efforts preclinical continue disorders Our and to kidney progress.

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

order effort growth. formation We evaluating functional in we establishing in models cyst continue and on impact of to observe ADPKD that confirm the an and readthrough has our

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

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

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

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

compounds efforts, in in base. on continue gratifying others we While reporting ocular are to the Eloxx also is observe program that potential exciting this disease it of our

across Human to a how have second example provide through of Madison, of from as of an intravitreal represent to retinal pediatric of our disorders NBXX promising VP cellular of Accounting, our form group proteins. nonsense to of may results. Genetics. application published Leber's that Finance University derived Pattnaik results also function Journal inherited the nonsense American an individual KirX.X related were XXXX the rescue ERSG and known ELX-XX, another ask I blindness, example, restoring Steve now quarter channel production would results allele These sufficient model at For the financial delivery of with review a broad of like congenital demonstrated essential was These Wisconsin, Bikash of in amaurosis. recently in the McDonald,

Stephen MacDonald

Thanks, Matt.

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

million G&A share June as ended incurred a million a with share company quarter net million XXXX, $X in or same per or $X.X expense the the to $X.XX for of loss and R&D. to allocated million per For the $XX.X of XXXX. $X.XX net Non-cash XX, compensation $XXX,XXX totaled $X.X compared stock period loss to

quarter-to-quarter totaled $X.X period quarter lower R&D XXXX. trials pause for Phase headcount in by The and in million same same X the fees, XXXX quarter to period to million, professional for reduced period. for was the lower to recent the due Second decreased driven expense $X.X and largely which the decrease of XXXX related XXXX second our and expense R&D the was expense compared in costs. headcount million salaries from professional due services XXXX, G&A $X.X for $X million

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

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

$XXX,XXX and loan in was CARES XXXX which some remain are XXXX, SBA's of paycheck PPP of via burn the our that of stock proceeds This the your with turn specified other we apply maintaining commencing applied rate to on end for [XX reach will interest quarter-to-quarter for our Greg. The and cash of financial we received April throughout to quarter modeling for were a March. law a back remaining expect signed variation. eligible for its shares forgiveness U.S. costs. into at Also repaid XX, and We outstanding purposes, XXXX. based call the the which in bears the Xth of will XXX,XXX]. headcount, of XXXX, second program X% stable protection total fairly loans the quarter of loan common comments, balance XXXX low component In is late as rate approximately of Act, and the for be partial concludes using of payroll, million June at I'll and the the

Greg Williams

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

our can laser-focused are that soon sites trial as investigators, on we, complete We possible. assuring clinical this and as

that We in have trials. and to signals date that resumed has are enrollment pleased Israel ELX-XX and in there no safety these Europe for been

that for fibrosis. are treatment granted of the for cystic drug We has the ELX-XX FDA orphan designation gratified

the late orphan Cystic drug ELX-XX cystic the in data confirms look we earlier, mentioned ELX-XX North I American As Fibrosis to to program Conference the forward fibrosis, designation presenting important benefits in October. on and at

of these some programs we encouraging advance cystic we're the in Experimental fibrosis, and ERSG and demonstrate scientific read-through in therapeutic data and been the inherited has their tolerability supporting of to shared novel preclinical XXXX Dr. edition activity manuscript Therapeutics. disorders the our of Beyond levels August continue of portfolio our molecules. from Journal further retinal on compounds Goddeeris Several of the development. you ELX-XX with specificity of our that ADPKD pleased and scientific published Pharmacology of recent latest

we August second presentation September next, be XX, will We at will progress. thank We Thursday, our and investors update may On Genuity and virtual XX, conference. one-on-one to investors meetings we call for Annual us Biopharma Growth with call meetings questions. at joining you very you be XXXX you XXth XXth to a making with quarter Thank the you Citi's Annual much. the for on Canaccord continuing one-on-one Conference. up hosting now Virtual hosting forward open earnings look Operator, our on

Operator

with Gilson Canaccord question Our of Instructions] Genuity. first from line Michelle comes the [Operator

Your go line ahead. is open. Please

Veena Anand

would on FEVX is the the and this Europe and study quarter expected Michelle. change a line about guys, meaningful Israel. Can and you studies Veena on question. clinically in just result have and Hi in the in the I us then for remind resuming be here follow-up maybe a specifically Congrats what on

Greg Williams

much for you so today. joining Thank the and asking call Veena. for Sure,

treatment trial kilogram. dose second X.XX, Greg X.X an dose It's the the first X.X, this the milligrams intrapatient is is escalation where dose X and So, is CF milligrams per has design, dose X groups. third top dose the the kilogram, and is is per

through then for by the dose is the administered doses primary pharmacokinetics evaluated period washout where of each duration an are safety top administration. X week by two independent the safety. for first endpoint safety review patients The of at X-week one The and is are first and a committee duration followed

key with chloride you trial related and endpoint which highly small in in The of and pharmacodynamic primary data we intrapatient FEVX. FEVX. more is consistent following comparison know, and FEVX this, tease endpoints. variability trial, signal. to be correlated may sweat As is again is larger we're much more where pharmacokinetics by is amount pharmacodynamic improvements secondary are more with is and a highly with also chloride In fair get is which sweat measuring a this also will In on a and with difficult a endpoint out there correlated FEVX, trial like focus small potentially we chloride a FEVX. our inter a to it Sweat trial it's

chloride. for differences of normalizing primarily looking to liter potentially chloride would [millimoles] to per and improvements be we're for as change that XX for important So XX a therapeutically sweat patients. in could would chloride increments those changes or as in be much would sweat sweat that FEVX differences Smaller We those sweat also correspond in looking be chloride. correspond

to And our based on providing and as we that previous are from dosage learn so results the we cystinosis right are we pretty we data range more. far, in trial look confident forward

Veena Anand

guess, the kind I U.S., then, when of a potential mitigate Okay, be topline to have include the great, we from thank we're gradually trial, thinking XXXX the data case given you when have U.S. in in guess of in of site you clinical of in able data, study to maybe clinical Should seems will your And report guess or the how kind in do interactions you or you I any and just XXXX. Israeli to impacts better to do and with get U.S. then, activity to second U.S. are both resuming changes, as expect anticipated of wave? COVID I you of timeline or plan kind try the thinking when protocol well? of also to sense kind And somewhere resume? trying the timeline. estimate of you be a implemented an we in will trial

Greg Williams

Sure.

Veena Anand

you. Thank

Greg Williams

is a them I at one questions of to there, there out fuse number will a time. So try

things Israel. have with resumed happy level really first, we Across as and world, the in are as can. Since So know, with Europe and their we we enthusiasm in investigators, made in progress with close and we've COVID. our are continuing to CROs, the we've you move trial of touch we've kept Israel, fast as of forward uncertainty in we associated resumed the good Europe spite

We course normally made related -- and more safety. but carefully patient of any carefully our COVID would are as priority we patients we haven't monitoring protecting course protocol we are changes, of is highest

complete possibly right to trials as as the closed ahead As know, with as provide will we can. is soon become now these we they highest is Israel you Europe additional our and in you since available. and priority temporarily updates we U.S. remain

Veena Anand

quarter And the Thank congrats we'll talk you. Got you. okay. next and again, it, Thank on to week. you

Operator

And Thank question of line you. Joel the Beatty Citi. from our comes next with

is Your open. Please go line ahead.

Joel Beatty

overview for thanks the and team Hi today. helpful

patients to safety has able many Are to no have and question First that good safety levels, provide signals you been signals how seen been has there safety many escalating there of doses at been no hear dose how terms there is, no what it's of is so context in in. or signals far. that

Greg Williams

through no for patients great that data, generate protocol additional there this you dose a the at are related and are will It's We've provide in. thank signals updates. across to as calling levels. safety we Joel, ELX-XX question point and we so, you for got proceeding number Hi, there further of see -- your

Joel Beatty

So mean I the four, number mean? of through level. four I guess patients, guess you all they've that dose that does been what with or three Is

Greg Williams

So appreciate a that not you're I asking. of level at sharing, is this point, we're detail that Joel,

patients data a we comprehensive have will provide we we update. as and timelines, generate more number in additional the of on region better As handle and a each total

Joel Beatty

How I that in for be how into enough. accordance further can fair how real seems patient given be more on Okay, like be the you Then, question you many able enough will some there, -- development. variable give patient chloride that to would how or share endpoint, is got can Yeah, guess it the a give if guess patients a that trial design, you confident to guess the I on context to effect from moving is it it. confidence to I are from sweat drug. many that.

Greg Williams

as So XXX. Patients CF with sweat the reportedly per nonsense diagnostic at liter. having millimoles patients chloride, be over XX of look sweat for mutations would had have value values well chloride we

or to incremental a Since have below we an for differences have further intrapatient than significant data happy you in have share response, of that really if statistically inter we're patients. an are to the and and down values, to is more highly statistical the and even trial, make XX patients results. hard available, to confident are will bring me meaningful that the patients at is between that, this information, mathematic be Israel So going are us these for we clinically enough looking potentially monitor really data you. open-label give quantify to come when in to the important room and U.S., difference both there continue and we lot they Until with we'll it Europe a we we that it's but as to

Joel Beatty

much. Great, Thank very that's helpful. you

Greg Williams

welcome. You're

Operator

line Thank our of question next Ted Tenthoff Piper from the you. comes And Sandler. with

Your go line ahead. Please is open.

Ted Tenthoff

you of reported Great, rolling update thank with the wanted and of has some programs the CF of CF, the get so sense for may to just work opportunities. now get on all thanks whether the impacted that a the and those preclinical much. how for that right much of focus Thanks how and advancing on you sort the stuff understanding very they partnering trial. some are an the appreciating wanted to disease, be COVID are preclinical I retinal offer is detail efforts, other specifically that on

Greg Williams

evaluate talking and businesses and we're So, potential has more color. well number preclinical to to a like down ask We you have activities open opportunities slowed COVID but proceeded Matt continue Goddeeris around to are to little very I'd do Ted, certainly routinely us the conversations. as and partners our give will always know, around those so. we we to a of

Matthew Goddeeris

Greg. Thanks, Yeah.

the definitely and the plans on, put in I we with pretty with and labs Early and There to were academic delays Ted. are it in see IACUCs plans the somewhat their observed with up question, and with work So risk, delays place of that did handle difference some appreciate how going regards was up CROs for the COVID getting to we mitigate only projects the in impacted board across again institutions animal to reopening. much on that we studies they came and says of and own safety in our seen being that but some any around hiatus. running studies order we've the dealt

got handle us initial I a say affect it, the as back able get we a did to So, people to right it. pause few would on for and weeks are

usual terms the So, efforts. it's preclinical business as of much pretty in

Ted Tenthoff

update. you thank Great, much very for that

Operator

you. like over back further to to Thank Greg time. turn at any I'm no would I Williams showing questions the remarks. And conference this Mr. for And further

Greg Williams

for questions. Thank Thank your all for today. you you. Thank in you tuning

look to and forward to forward forward Bye-bye. talking XX update again you talking you XX and continually Citi. look We We will Canaccord September Conference then at again again to soon. the on August at look on with

Operator

you in today's This the Everyone you may does conference. participating thank gentlemen, great have for and program all a Ladies day. disconnect. conclude and

Eloxx Pharmaceuticals (ELOX) 8 Aug 202020 Q2 Earnings call transcript