AGLE - Earnings call transcript Q4 2018 | Spyre Therapeutics, Inc.

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Operator

Greetings and welcome to the Fourth Quarter 2018 Corporate Update and Earnings Call. And at this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. [Operator Instructions] as a reminder this conference is being recorded. I would now like to turn the conference over to your host Mr. Associates. Merrill Sharon Calusdian David Thank you, Mr. Calusdian.

You may begin.

David Calusdian

welcome Conference Call. and available take to they Aeglea Earnings and to questions. Update Corporate your After be BioTherapeutics will Well managements remarks prepared

forward-looking call we valuable therapeutic trials of data, may statements Before clinical and comments business to timing, related respect to candidates, expenses priorities, company's to success our timing uses the and strength flows, the research to to could fund manage assumptions cash the other programs, statements. benefits and on on of data, technologies note and statements of the plan trends revenues, and by matters. please that cost, the and including based landscape collaborations, and cash its these include updates company's the number success the from ability cause and competitive of of economic candidates, for begin development risks differ a for and of those actual results announcements advancement programs suggested product forward-looking trials and safety product regulatory These with ability the and strategy, significantly product are to related along today's our the subject uncertainties with are relating proprietary our and and that that pathways clinical candidates, development

page Except should you on company's on for reliance Commission undue expectations and statements made download to call call. to forward-looking XXXX these from available for that cause Please for circumstances Aeglea's occur of I'll disclaims go place presentations or of important and and law, that please risks results to revise reflect website account filed risk on differ Quinn. events actual XX-K at on Also, is IR as Form including turn Executive presentation ahead. accompany the after some section not with update refer materially or this this the Dr. X, by statements. please forward-looking these Chief or the obligation events its that Anthony any to uncertainties, the Given the March the company any note to could publicly Exchange statements forward-looking Securities over any a required its company's of now factors Officer, call this www.aegleabio.com. the call. to Quinn,

Anthony Quinn

for for Medical with in Thank leveraged XXXX you advances We clinical will progress Chief With me and programs with Aeglea you and made progress Arginase to pipeline. program David our for continue program significant Deficiency on XXXX. was our generate York, is and Jim cystinuria capabilities new significant year substantial a our our made allow Thank pipeline Chief unique company's Good number are Dr. We us X start homocystinuria. in lead oncology. our strengthening and in for which the hunting sheet to today drug joining invest we and of had Financial pegzilarginase balance Officer. to In Officer successful a everyone. afternoon, a our and to XXXX fronts Charles us. Wooldridge, really great we've

we As and both positioned is the our look leverage advance well human design XXXX, company to to capabilities. enzyme our unique programs

success in a and accelerated We've lead in further gross candidate financing total and resulted pegzilarginase, continued this programs continuing million, accelerating our year product investment manufacturing XXXX plans our build our We to in We capabilities. $XX XXXX sheet pipeline our proceeds our and and positioning are in February us in team activities. the to which we of for beyond. closed our of balance offering and strengthening an do

weeks patients clinical made Phase last XX develop dosing. completed X/X who Deficiency. experience rapid to data year on in on eight build pegzilarginase the The Arginase continues to repeat We with we've a of have with continue now progress X

have an the April. in we'll presentation Washington data X/X that at meeting an in on We Seattle, and be providing Phase oral at early update SIMD

have you the We've pegzilarginase completed As trials. advance good been studies patients combination we single the solid impact know, Arginase and in of now progress all our progressing with agent enrollment also trial. investigating also tumors. that expansion with We've depletion made with are

biology, clinical and and and forward We're diseases Our that significant [ph] programs with also as opportunities have earlier IND-enabling we as enzymology studies. safety of knowledge we needs. about more are Jim. cystinuria our Aeglea for unmet medical through for to very over diseases you XXXX. to continuing turn achievements will two leverage walk move call which addressed plans the ahead programs our XXXX rare discuss you forward really on at our initiation advance now need unmet looking updating the with human homocystinuria Jim, of metabolism address and significant medical common to continue I XXXX. through We're also much the to our who excited progress a for would

Jim Wooldridge

and Tony everyone. hello Thanks

XXXX the rich in of and been mentioned have XXXX Aeglea and Tony start data disease productive at As and oncology. time rare a

We First; our X for pegzilarginase completed with Deficiency. lead Phase investigational X/X on our clinical dosing Arginase therapy trial.

this inform the Importantly, of the was pivotal critical data design our we PEACE trial. to study generated Phase in X

oncology trial In and with safety uveal advance single ESMO the this molecules. we in In the the clinical tumor X cohort quarter, dose consistent patients per pre-treated kilogram enrollment with or X at initiated pre-treated activity interim While Cancer to completed melanoma prior completed with including pegzilarginase December, KEYTRUDA with agent data of In was in and we demonstrating our in expansions enrollment monotherapy in X dose tumors efficacy profile combination also and into fourth Phase chemotherapy. X. Small designed one Phase the also and extensive presented of solid cutaneous Cell we Lung any in pegzilarginase confirming the the From profile Cell patients we progressed Cancer. made combination Phase who safety melanoma. Small recommended following Phase trials a melanoma, milligrams platinum trial This relapsed to highlights, important selected in and safety was assess our with of X patients is advances confirmed monotherapy we X.XX heavily XXXX, and our biggest with trial, based with pipeline was heavily disease KEYTRUDA. pegzilarginase with observations Lung pegzilarginase escalation Xb Phase Phase the study

importantly, nine we More the in partial including observed clinical response. at dose and this in level patients stable three one treated at activity nine disease weeks

this in line top first half XXXX. expect from of We trial data the

excited be with about on pipeline. In presented October, pre-clinical We our continue to AEBXXXX. data we progress

urine on inhibited a urine in a crystal programs pipeline and of of forward formation improvement IND-enabling plasma by stone plasma cystine for Our model October, homocystinuria survival. for discovery American of model look was Society and disease In In degrading in manifestation clinic programs started reduced lower a human potential XXXX. dramatically the levels. to of including at cystine improved significant [indiscernible]. Nephrology we both in to activity program our presented pre-clinical levels, already based pipeline homocystine new novel data related formation. kidney homocystinuria these reducing and accompanied candidate pre-clinical has a that we cystinuria, degrading enzyme Also and both towards led to the activity of cystine the We the highlighting AEBXXXX bringing

aligned We Arginase global stands December, of for a EMA. Turning and randomized designed design the addition dose versus the Arginase Endpoint secondary from quarter over XX patient announced the and effect PEACE placebo weeks. reduction arginine plasma At guidance Deficiency. X on this the include to we Clinical in X of is of the now P safety double X and that last effect milestone. and our Phase XXXX global blind to assess to assessments we feedback pegzilarginase first pegzilarginase pivotal the as which PEACE in behavior Deficiency endpoint in FDA we of it's trial trial effects, pegzilarginase primary in study and in meaningful end expect meet endpoints to year, on The pharmacokinetics. and track adaptive we're the second of and mobility release clinically to

As arginine after of repeat trial. eight Phase should plasma trial the of a were control in improvements establish study accompanied plasma arginine In X.X from by in Phase X/X rapid of milligrams in the Data dose only arginine levels highly dosing. and per effective weeks levels. reducing abnormalities pegzilarginase disease-related we've discussed plasma which kilogram X/X before PEACE support the weekly is reductions important

expect benefits look forward caregivers Lung of longer treatment good with control will this it's I'll the indicates from Cell the adaptive that trial year XXXX to advancing with of received With our trial Insights to we're assessments Given capture be Arginase design excited initiation the with from our in plasma call confident the data closer suited that, and arginine programs study highly pegzilarginase. Cancer exciting bringing will pegzilarginase marketing are to that the and clinical of importance Furthermore, a KEYTRUDA the for summary, anticipated In standardized was financials. Deficiency. increase responders Small ideally clinical over clinical support of to applications the pipeline two PEACE physicians to from the successful with turn of productive we the feedback X trial. in combination we've discuss Charles and trial, XXXX proportion and and we sufficient that PEACE a and clinic. and in assessments to the behavior mobility

Charles York

everyone. Thanks Jim good afternoon, and

which completing XXXX. of financing already in earlier, February. in to cash forma million balance December important net approximately took at sheet million and pro proceeds As early Anthony provide investors from $XXX came existing us new steps discussed The we our by strengthen XXXX $XX with XX,

the of provide XXXX. PEACE runway More to capital in importantly, is our out this through quarter Aeglea cash first expected pivotal read trial with

Arginase million loss the million or net $XX.X develop We the revenue to That full XXXX research revenue grant and $X.X in recognized will or the long-term foundation in on cancer XXXX of our XXXX. $X.X and disease award to result grant cash key $X.XX additional Operating year net of product XXXX Aeglea programs at million believe grant. be concurrently full of pegzilarginase Regarding million quarter with a quarter of in the expenses $XX.X and the compared $X.XX loss candidates. in received Aeglea success. May, a increased XXXX strategy the in XXXX. continue the $XX.X which per balance rare Deficiency X XXXX fourth by end our our drive to in recorded forward contract of grant we of the included revenues financials, given of million cancer of All fourth in life share per and over invest share our were XXXX we

our quarter strengthening operating XXXX. million was Looking versus G&A, our XXXX R&D, fourth in expense and was in G&A lead quarter our advancing were million of million fourth XXXX. product primarily development fourth pegzilarginase our capabilities. at clinical XXXX the in The versus $X.X $X.X due quarter $XX.X $X.X million development of expenses expense And R&D the quarter to increase accelerated program fourth of the manufacturing for

During XXXX, in Continuing clinical enrolling combination in patients with Phase in our Arginase tumor and X/X and cohort our label Cell extension Completing trial expenses in Phase enrollment our agent completing open and trials trial patients Deficiency. our trial supported enrolment Cancer. Lung single solid Arginase over Deficiency. with with three X pegzilarginase expansion Xb X patients the Small

the forma million in pro extension million of to were our our looking XXXX, million runway the we manufacturing the activities in trial $XXX in pegzilarginase burn in strengthening an $XX our XXXX where through December exception quarter first sheet. range Arginase million $XX approximately balance range XXXX of PEACE $XX forward be of steps development X to our in of our anticipate XXXX important pipeline for $XX and Now given out anticipate we read of cash In quarter at burn of ramp with cash our quarter will million the pivotal in will per XX, first Deficiency. of to the be

in some final favorable where I call a Additionally, our believe to turn to mostly will market homocystinuria indications need over we We there compelling. medical unmet we importantly, benefit are will is from investment the and we data and position assets in significant remarks. believe now to early Anthony differentiated own and continue where back that cystinuria we in programs. both drive shared and worldwide for both rights

Anthony Quinn

Thanks always, Charles.

So to employees questions. work including, very that to also positioned caregivers believe we progress really all diseases. living of towards support make We're we therapies the meaningful with of patients, our we're devastated the goal providing appreciative transformative importantly and from well patients hard Thank multiple we the the and get and very of investigators We're trials. XXXX. our to XXXX these by involves in Operator, that through excited clinical We'll ahead. very sharing to you. looking forward go you ahead actually with open course call milestones can really now you updates the much as

Operator

of your question first our ISI. Instructions] of [Operator line proceed Schimmer Please the question. comes with Evercore Josh from

Josh Schimmer

the a Thanks. homocystinuria to sense you approach, the initial expect give evolve. maybe be might establish you you of programs can INDs What for like those how when one and able concept? endpoints As to look cystinuria of clinical of might each us proof [indiscernible]

Anthony Quinn

the for question. thanks Josh

So enzyme presented mortal at we lowers levels let in lower homocystinuria very improved me that an start exciting molecule, [indiscernible] Obviously showing homocystine of a with last the program. end have that levels disease we year, homocystine survival. data

So in good complications. a and of there's control the lot control homocystinuria levels to disease homocystine two, homocystine linking actually levels information and

relatively importance to So of in human the have which basically with of our going to get these forward some levels. what and disease I homocystine in surrogate to evidence high, means we like that the you we to actually out into the have very of program. homocystinuria no have regulatory compelling patient you're animal see translativeability available we're homocystine we is confidence of models pretty well the rare that's going that so what program. and with we fairly the a the development lowering endpoint go in see is read is it's as there's It's really effects What established move [ph] a controlling that disease early model But obviously lot will authorities. expect the and though, about discussion to about the population rare

again we actually actually a compelling showing we've to approach, lowering place got surrogate so got plasma data is We've So up start. urine very normal come great of - for endpoint levels, we've the cystinuria that's inhibit of list FDA's cystinuria; seeing levels and very over model some crystallization, on and by cystine guidance carry of information model the in we into we've would that we stone probability human time, the timeline. animal observations with highly expect inhibit we translate have our so we and same the seen high some obviously as given therefore,

So homocystinuria, of quarter guiding for of IND we're the first having we XXXX then IND filed and second the that we're XXXX. in our half the our actually will cystinuria, in for guiding anticipated

Josh Schimmer

much. Great. Thanks very

Operator

on Capital comes if BMO next to Once your Markets. Our from Matthew your line of Luchini line question. proceed live, your the Matthew again question line of proceed Please with see question. please your is check with is mute. Double

Unidentified Analyst

cohort give might our Matthew. is of any the taking on responses from Thank or X the Steven you patient you Phase on because for some I wondering thought, was? you this incidents then success Hi, in for you color that see had on call. ARGX-D, any you Could initially the than trial SCLC timing the you maybe had we there And update when was if the data. on greater identification program guys prevalence, of thought, the for if maybe

Anthony Quinn

the Okay, so in order gave answer let's the us. questions you that

Jim to to Small I'm and the question. over he'll Cancer hand Lung Cell going So do

Jim Wooldridge

the for thanks Yes, question.

we Cancer, not Small for Lung observe So patients Cell in did responses. the any

and to confirm we're that did the as we're the study deliver pleased safety profile cohort really rest of the promised. for able so we that However

Anthony Quinn

back And let about and Deficiency population. the Arginase me come X talk

well the assessment So that not is like end of any basically disease use use understood and that was an rare epidemiology done, our we data. particularly

is neurological X and patients They're manifestation that urea actually So consortium. actually means were extrapolated the there's side of it get let by patients attention considering the number different likely on We [ph] be under of on disorders you, side. not will cycle hyperammonemic me with that it's fairly is, to data less they potential represented. remind therefore that cycle other episodes. solid urea base Arginase under other the of What from Deficiency but from gives so case, the us cycle used ratio screening prominent and mean conservative and be metabolic some just urea believe it's Deficiency why very follow-up conservative that frequent physicians, disorder Deficiency patients a high a newborn disorders. the Arginase we the then believe Arginase

addressable kind that the guided at analog and So XXX came X number [ph]. to But that's number, a in major the and where of with markets least is patients from we actually really MPS than hard substantially more largely find to already rare exciting me that, we've XXX and confined US is to it's identified to efforts patients with what's patients Europe. our disease the good

we're of we than So But is population XX% of found And know XX% could continuing more XXX actually patients guided I have finding so the that at and good patients believe we've we're don't better. I prevalent population. to. the we're good to

Does initially side. that's the So us that help? to conservative the give the guided what confidence we that to on is number speaks

Unidentified Analyst

Thank you great. much. that's so Yes,

Operator

line Our and next Company. proceed of of the question Please Needham with from question. your Chad Messer comes

Rajvindra Gill

on Gill Chad. for is This

a Just a us remind the specific Basket in reasoning quick you question, types trial? cancer the behind could of

Anthony Quinn

do that. let's Sure,

you to just particular ask why these going the we I'm to So logic through Jim walk pick genotypes. Jim?

Jim Wooldridge

Gill. for question, thanks so the Yes

So depleting environment the as getting of highly Argininosuccinate called cancers effective you highly enzyme recall X. cycle arginine plasma those arginine lack pegzilarginase plasma and are cellular by some and of on urea driven dependent are ASSX, extra at from expression tumors is

the selection Cancer, types our Small tends fact so Lung proportion in of a with to individual significant ASSX each uveal and of is And have Cell of melanoma tumors tumor expression of low melanoma, very patients. the that those cutaneous

did also As uveal eight and saw activity the we in you'll from more that we activity stable with show signal population. concentrated of partial recall our a single cohorts ASSX being cutaneous in in only publication, diseases ESMO melanoma not one response agent the but

Rajvindra Gill

any the much PRV very you you. additional Could you discussions color any going AEB? with for on about clarification. give for Thank Thank

Anthony Quinn

question Could you repeat the please?

Rajvindra Gill

on Review additional Pediatric if for it? potential color there's I was Voucher, about asking any

Anthony Quinn

designation. question. That's Rare have we Yes, basically so an Pediatric Disease important

implications once of that you to pediatric not FDA given Rare eligible this is of is the approval recognized Pediatric your voucher, until eligible designation. drug the get for approval pediatric then we is, be we has that have that, drug. get we would the with What - are we So voucher but you

Rajvindra Gill

right, year. All very for thank luck new good and guys the much you

Operator

question our comes Cross your Instructions] H.C. [Operator Wainwright. the of Matthew next proceed of line with Please from question.

Matthew Cross

of a questions quick for Had you. couple

Phase start you couple moving then to Xb of mentioned this the and the in sub of so one. into Phase completion So questions off, X,

with this combination the safety X.XX the is in further both pretty its in per And also but at kilogram that objective still comfortable is, but the part to per your to signals monotherapy no of response Jim proven FDA responses observed and without thinking small given you Phase pretty cell compared escalating and for moving point. First you reiterated. tolerable, efficacy. milligram just still kind surprise on How test, limited. the rationale are and no signals X dose with into stronger not recap forward has second Can the then safety as this

Anthony Quinn

one? Jim, you to that want do take

Jim Wooldridge

Tony. start me let Yes,

some obviously activity Small and know the cohort as I looking can You just actually if report setting. mentioned anything. all Cell Lung as cutaneous moving monotherapy particularly Phase think extra at add in rapidly disease, But we uveal relap we of you monotherapy I previously did a and our color, melanoma three missed tested cohorts it's out on and with our melanoma first Cancer I X, really is

criteria. toxicities mind. And dose so our keep by I in really When Phase Xb to study we limiting observed we defined did any actually didn't think protocol that's important

of other We it's into X our doses. bit significant the Phase X. Phase Phase X However, at be this and study demonstrated and actually actually in through believe the pulling was what the tumors since would over dose confirmed now actually tolerability quite a the a study we're our depletion in better both X.XX range Arginine

to So capturing we're actually forward pretty that. looking

Anthony Quinn

managing interest looking new started remind for And as deficiency the then Arginine we X people. enzyme to depletion off that Arginase Especially story, activity. want about We we in modified approach cancers. at and when strategically, who so just a for maybe sometime back, to innovative an with then, enhanced looking Arginase pegzilarginase had there's were enhanced been to just stepping

relative the So in Phase disease obviously earlier. approval Arginase Deficiency. pegzilarginase oncology X as rare study, driving mentioned starting we're to X the a in somebody forward obviously with quick is We're

eligible our data momentum and agent pediatric orphan in of are Arginine is, at we've other when are in immuno-oncology We there enhances time, thing data oncology at got we're completing within we the compelling small we single designation. approaches and actually program, looking actually that carefully, moment obviously actually for But we'll for our that the the oncology great effect we pre-clinical depletion same obviously, We've activity. we activities voucher and how we're forward have demonstrated cell obviously in said, various if look data that are our program. the the And got the space. as driving Jim best advantage showing at and in cancer fits good pegzilarginase so think disease will get rare that at

Matthew Cross

it. Got

before Okay. color future Appreciate both should within expect to we from near guys, sometime that that combination results you or of conference? in see in the

Jim Wooldridge

so we've having to guided the Yes, year. in combination of first are results the next half

Matthew Cross

it. Got

how Okay How we're pivoting completing in for X Arginase ahead as Arginase think PEACE hopefully XXXX. Deficiency Was Deficiency. of pricing and curios you trial to then much X looking over Arginase reduction you do geared reduction the measuring tied pricing set Arginase light to FDA versus surrogate the to functional efficacy has being given in the here. given [ph] the PEACE much trial green in that patient outcomes

for get it's without disease granting your rope to are this, political thinking be the been at perspective. drugs regulatory that levels regulators allowance pricing kind walk profitable the kind any given or [indiscernible] Just rare high [indiscernible] Trying to given on tight of here. of

Anthony Quinn

problem they is unlike neurological patients with the Deficiency cerebral palsy in the early. They complications progress kids X terms a disease, the like childhood. look with ability have, and early of kind cerebral motor kids kids with but of are palsy devastating and effective in die Arginase this

over the severe end of neurological with and complications they age XX severe very found irreversible We haven't out and patients intellectual disability. many

So this is a devastating disease.

achieve, control easy number for it have therapy I plasma to would we it very plasma to and its Second of exciting plasma been been control been but is and that, pegzilarginase the thing as what is think been we've if it's now - to ability arginine of years had Arginine able about known transformative control, have levels. is that - what's importance arginine

the mobility This about. their and the we're actually fact lowering of very for and neurological is the that, of why why more within that's we're piece that within already pivotal terms patients disease, actually arginine final these is adaptive plasma seeing these Now excited We're in the of very seeing impactful I'm weeks. weeks levels. so that the six of improvements a jigsaw clinical behaviour we've compelling obviously really and eight months and in at that is dosing one reasons eight we can actually look benefits in

see line So to and placebo endpoint. is that that difference got We've XX% clinical see We've going confident a plasma study the arginine we'll playing and to the the we're active powered and benefits. reductions we're actually confident bottom we're have actually package.

actually demonstrate lower obviously Now to drug which phenylketonuria is and approved levels a they that the and trial, weren't is only approved been stay obviously clinical you, clinical to remind recent able back benefit. that's phenylalanine any drug for which in pegvaliase just actually price, and

at we're seeing what context, already. the obviously I'm of see clinical kind excited the it's So by - looking to very but benefits in

Matthew Cross

guys. play that Agreed. Yes, forward much things comparison and context. to out for how Thanks looking very seeing appreciate

Operator

you your for questions conference, further there this Instructions] over participation. at conference. of Thank the time. portion be we concluding are will no this concludes the [Operator today's if

this your disconnect of at rest may lines day. a wonderful time have your and You

Spyre Therapeutics (SYRE) 7 Mar 192018 Q4 Earnings call transcript