Earnings call transcript Q1 2023

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Operator

Greetings, and welcome to X4 Pharmaceuticals' First Quarter 2023 Earnings Conference Call. [Operator Instructions] As a reminder, the conference call is being recorded.

It is now my pleasure to introduce your host, Dan Ferry from LifeSci Advisors. Please begin.

Dan Ferry

Thank you, operator. everyone. morning, good And call XX's today's Executive and Officer, Dr. will on President be Chief Presenting Chief Adam Financial and Ragan; Paula Officer, Mostafa.

Officer, by Following prepared questions. Chief Commercial joined your and Baldry; Art Chief Operating call the Stewart; Scientific Medical Officer, Interim Officer, Mark remarks, we And Officer, will we'll Dr. open DiBiase. Dr. Chief Murray Taveras; Dr. to Chief be Mary

activities. differ regarding XX-Q, as the from call, results As Description company will may SEC, which be research statements filed on is well forward-looking risks plans development and today's for these that the statements with company's found the to XXXX, can XX-K regulatory expected reminder, to subject latest to be product a making and those uncertainties are risks forecasted. These as including be the and actual today. filings Form in quarter's XX's year of cause this

call Dr. to like to Paula over now Paula? Ragan. turn I'd the

Dr. Paula Ragan

the call us thank and today on Dan, Thanks, will an what efficient throughout we make our be commercialization of and investigational the to XX continue you, We as call focus Mavorixafor, XXXX. advance disorder, time truly potential This we exciting rest an towards chronic hope first for joining in hope at to milestones this syndrome. the value-building candidate, on neutropenic lead this is WHIM everyone, morning.

the good absolute we neutrophil our versus lymphocyte times significant and clinically in X Phase longer both threshold endpoint Mavorixafor trial. XXXX, placebo. tolerability above levels that counts statistically clinical once announced its but for absolute achieving in late and WHIM with people endpoints, And demonstrating secondary and Mavorixafor in syndrome relevant daily, key evaluating know, oral you had As first primary trial met

Immunology of was late-breaker Subsequently, Clinical abstract data trial meeting at taking the will XXth professor through May of that who and presentation we Dr. our Time place in trial Sunday, Central Phase St. in XXst for Italy the of AM May oral Louis. investigator Badolato, at X pediatrics an present the Brescia year's Society University from WHIM reporting accepted XX:XX at clinical this from in is announced Raffaele XXst. additional on the XWHIM the

will we posting to on the accessible live presentation. session will website this only be concurrent attendees, our the conference the Although slides be with

Following other CIS the hosting the of be the later on XXth, will an additional that endpoints from of publication results day investor on conference outcome event abstracts metrics. PM X:XX on by trial, morning at present including burden among we data secondary infection to May

that provided or on for can earnings the website register our through morning's link press event You in release. this

the will neutropenia the people investigators immunologists, Joining in and the the medical trial. Phase be with comment results of and us expertise whom several of Phase whom in treating all rheumatologists, for XWHIM have panel WHIM on immunodeficiencies and event hematologists, a unmet X and X diverse to needs of of chronic were

in immunology. Program the the at pediatrician Bone Paris University at Associate of Research Clinical Hospital; XXth and and Tarrant, Teresa Duke Peter Cancer rheumatology of of Myelodysplastic Medical School Shimamura, Biology Medical Director Syndrome importantly, for we Dr. Newburger, registry Charlie at of at and UMass and Dr. of Laboratory and Director at Harvard Marrow Akiko Medicine Failure and Dr. May Dr. in and Immunology for hemato-oncology Children's Vice a School Chief Pediatrics be Boston Medicine of from the of Hospital Translational Chan and French Professor School; commentary department Professor School of the Dr. Molecular neutropenia; professor hearing Professor the During and of Donadieu, Jean Sinai; Cell and Pediatrics at immunologist Mount will event, the chronic Icahn Cunningham-Rundles, Trousseau a coordinator

have individuals syndrome been who be We unique symptom also birth. WHIM since from three and experiencing hearing been WHIM with perspective will have diagnosed

Finally, following Q&A the we Shimamura presentation. formal are to doctors for Tarrant live and us join expecting

on continue activities early a an new event, track providing to XXXX. launch the be our US in be to for syndrome half regulatory a as in XXXX of we to During application, for US expect treatment on potential the the drug Mavorixafor we file of and half US the update second of for WHIM in prepare the first

we cyclic, in on continue to CN chronic in on safety Mavorixafor expected of Phase provide program the all efficacy with of of and and track trial data clarity are the Concurrent to and believe Phase ongoing clinical this, QX/QX evaluating announce treatment timeframe. our the we X enroll timing scope the participants and idiopathic, the for X neutropenia, the of clinical and congenital

XXth. SIEs, severity presenting poster In of serious Concurrent with infection to announced we at May events, and the the published be a PM also May highlighting the poster chronic is poster what the we research that assess between Saturday, will on is our study presentation, results be This we release at X:XX Central the the Time, be to website. of incidence our CIF, which will this on of XXth the first also will neutropenia. adding correlation we abstract believe morning, or

to to As demonstrated to elevate levels a potential the blood other the a white bring and populations to and patient syndrome We WHIM cells, result to of innovation data you updating our we chronic our that progress as has disorders. our with year forward efforts date, mission ability we in need. to look development due neutropenic these of continue its our those on be advance for throughout believe to published Mavorixafor to breakthrough

the Adam Mostafa over it to Adam? quarter financials. I'll now CFO, review to turn our

Adam Mostafa

today. all us being Thanks, on with for of to and you call the thanks Paula,

company XX, quarter At cash had the equivalents, that XXXX, in XX restricted support We into million the to sufficient funds operations cash, the first believe XXXX. $XX.X ended of end second quarter of March and are cash. these

R&D $X.X expenses million certain expenses a and compares for XXXX. included of development in million to milestone the for the that deems payments $XX.X company quarter, $X first for comparable Our of probable were the $XX.X the and quarter for non-cash an first accrual period in-licensing occurring. research million expenses which million

as million for the were non-cash the $X.X compared in selling, first SG&A the million quarter and administrative XXXX. expenses expenses for certain of $X.X expenses for Our million comparable period quarter. to $X.X included general,

of for loss and million change million expense we net reported ended our first first quarter. comparable as C for the $X.X $XX period fair of lastly, value liability in March a the $XX Class stock-based loss million the Net for to of for quarter And XX, million in XXXX, gain the XXXX. compensation included a $X.X compared warrant

Operator? that, up And open call don't the we your with why for questions.

Operator

Stephen Instructions] [Operator Stifel. Willey,

Unidentified Analyst

for on my have two just [Chun questions This quick guys. is Steve. morning, Yang] I end. Good

hear color one like it increased little be discussion front? possible And would partnerships the to we'll that's like and much. Thank you you secondly, guys actually my like has be also potential So first partnering still would do is there a you ongoing updates more I think possible, very detailed with give bit when R&D think expense? more on can provide Adam, on been -- it on it or end.

Adam Mostafa

Thanks for the question.

occurring. for accrual to in milestone is we $X-million regulatory-related And related so mostly a increase the line probable this in-licensing that an or of That's deemed R&D payment. quarter the

And on so is a the cash. you'll there, question that's see change which that

we for continue to update, ways could partnerships. do look front, example, And the types an when something at that. partnership we'll report of have And to the we geographic-rights finance beneficial to On material include, certainly company. that

Operator

Riley Mayank Mamtani, Securities. B.

William Wood

Hi, today. your from success. questions on this continued for William on is Mayank Two Congratulations us.

you'll to KOL, rates the as of that and can far data in or we your provide follow as them, we what or new these Thanks a then follow be should we as on might presentations? curious cuts different you I'm presentations? each infection And be on overlapping largely -- if of just One two or data both see up. at and any up. are be one well information infection expecting color these data at the extra CIS if you're these from presenting as then going should

Dr. Paula Ragan

question. for Thanks the

press to other from are data we've their the infection, burden So duration those and metrics. forward call. more relevant around looking on directly severity, can you'll ones sharing and among to that release, to as to today's our in clinicians, all get outlined important hear we And frequency, relate actually the we're and of perspective which highlighted

terms I part question And The train different and data lost venue. on that the the I -- in of second the apologize, different thanks, Mark. then of -- and -- different

investor So similar. the to community. the data same. sets within will data effectively, clinical But communities. Obviously, be going is for oriented the one to primarily sort the be are audience quite sets one's audience -- of then a broader And more

William Wood

FDA your release, I that about and provide maybe your in well helpful. very That's following upcoming you how if Phase as it. X are Got going X could And guess, terms data Phase insight of you're what any as, forward? when plans Phase X gained study during execution, your you thinking then discussions

Dr. Paula Ragan

around study? neutropenia For the clarity, chronic is this

William Wood

sorry Yes, about that.

Dr. Paula Ragan

Thank you. worries. No

So registration continue we'll provide for that or with can have we chronic data well to completed neutropenia, guide that, on agency program. as clarity Phase we in have the additional so as QX QX interactions a we'll X

dose, We're to looking The patients, chronic Phase for nice on primarily durable neutrophil additional in be resoundingly that will given data, correlation Xb, is durability are those after So blood focused which of the positive neutropenic the that looking progress including very neutrophil, a crosswalk right with had then these all infection. count. the counts, of including and single white cell forward thought elevations in and with now. sharing the We both result. WHIM, data neutropenia,

and likely course, is in for that the to hopefully Now the value size nicely which will of with benefit. to sharing WHIM, in registration that data, changes looking similar [CN] look of we'll elevated that be then, that, future testing trial in WHIM. you're durable And infection and forward with more from consistent

William Wood

that, I again. all and our Thank taking appreciate you for Thanks. questions. congratulations

Operator

Cowen. TD Privitera, Eva

Eva Privitera

Hi, good questions. thanks our morning taking and for

in what For and expect duration of the chronic -- how long patients how neutropenia update, can we follow-up X the of Phase many terms

Dr. Paula Ragan

Eva. actively -- I'm enrolling ahead. sorry. sorry, we're So Go I'm

Eva Privitera

split Yeah monotherapy and versus combo dosed with the also the congenital, idiopathic, patients what's And split roughly? the of cyclic and patients G-CSF? with of

Dr. Paula Ragan

I enrolling. Yeah. mean we're actively so still

--similar any can. questions a we've because of Xb,we wide trying we're nice I So be can't to broad just And a as answer Phase funnel. split have population course, the we of to of as the

between We more robust aiming a and from to you data. We're thought that the then of couple we somewhere across of have a count XX get make had the to able of be think some XX. to was to valuable Xb number possible. trying buckets I patients, the we're the as for terms come because set data when really enough of on as And So Phase generalizations. in

or it's Certainly meaningful that. forward sharing durability QX. aiming as and But timing. But meet to what that's look about QX soon update always can recruitment patients course, And we're as of we around in for. we and

Eva Privitera

for think Thanks And meaningful? presented will of Great. X be infection clinically rates and endpoints that presentation, are Phase at the secondary do that. in what on what CIS, level WHIM reduction you burden

Dr. Paula Ragan

mean, I a which X. a -- little share designation believe you saw Phase what the we on in what to The infection year breakthrough bit can data, X agency WHIM a showed I Phase about and us agency reduction XX% the benchmark. granted the with a what rates. in therapy was different Yes. after of we I XX% It

Of course, using their patients historicals were because control. we of

therapy that as sets us perspective, agency certainly and breakthrough from grant the meaningful the relevant view to certainly clinically and So mark they that designation.

to we'll just totality the the forward in of infection data, a information So including all of couple look weeks. providing

Operator

Wainwright. RK, H.C.

Swayampakula Ramakanth

from Paula morning, Good RK H.C. Thank and Adam. Wainwright. This is you.

think all putting if about on the should right the XXst, them But XXth. my May I I on at KOLs that and know same you're Mavorixafor. and five together do XXth I time, job

around additional subtypes be could expect neutropenic we conditions about help would where also, to of -- should in to SCN the this used? gets Will Mavorixafor expansion think these to up conversations these regarding to you in us So expansions Mavorixafor? for conversations, color -- sense where be causes? for folks SCN initiate due various And talking indication generated about come potential some by that

Dr. Paula Ragan

Great RK. questions. much, so Thanks

which in patients and call, the these KOLs, Europe, are of So US cases, think on rheumatology, where our across managed. and some of is then the hematology, the and some KOLs immunology, I breadth

we them not that on only have our felt patients. we in some have number experiences So of wanted nice relevance universe chronic treating neutropenia own to data a of on certainly commenting but of larger WHIM,

future events experience, the an live we be at into experiences folks of will able the bring those forward to they'll from we'll for that that able in have to end, So around few speak so their conversations. be a them to topic, And have indications. their Certainly the perspective. around the Q&A on look they

Swayampakula Ramakanth

Thank you.

to events. forward these two Looking

Dr. Paula Ragan

RK. so you Thank much,

Operator

Fitzgerald. Cantor Kluska, Kristen

Rick Miller

the Rick of is WHIM, the little bit CIS This on conference CIS, taking a morning. focusing questions. understand front of WHIM? could the at setting, community the you could inform about on our kind To in for the Kristen. ahead help Good be set getting talk and what Thank you could for audience this and you're expecting how you you be audience stage prescriber of in could

Dr. Paula Ragan

Sure.

at this him Mark? think CIS I his conference. over I it primarily to a provide team turn participating in additional -- Mark I So to has immunologists. color. know is of will

Mark Baldry

Paula. Thanks, forward Yeah, customers to our CIS a Hi, at Rick. planning looking where of being we're to be. are lot

meetings focused up which we'll on disease and raising with boost have set of a We customers, company is well, have a key there number WHIM. awareness of as

release of be a valuable Phase conference it's as excitement for data. the builds X the us very we around that So to think going

Rick Miller

you on data Could expecting could presentation the talked And on patients any of you on mostly here, be genetic for the patients what on the that we this in from kind potentially expect about? poster this one going Should also Thanks. information set Great. CN study? G-CSF? do background for plan And more you managed into announced just real-world patient we CIS. you stage maybe

Dr. Paula Ragan

than So even We get records. it's higher-level that. don't in medical genetics not sometimes electronic obviously -- into study a or captured

of the different a is that ICD-XX diagnosed histories types infection severe on There's it And in study down their a their to chronic drill events. clinical then populations different with So there's of ability on higher-level terms neutropenia. different codes. are

evidence connect is those in and connecting that mortality. severity potentially So of degrees with neutropenia real-world dots, cases, morbidity really around poster some the

sharing a follow-up the to as poster forward look to question digest. we certainly that So and have that community

Rick Miller

you Excellent. Thank very much.

Operator

back like Dr. to This would Ragan concludes the over turn the conference question-and-answer I any session. for closing to remarks.

Dr. Paula Ragan

XXth everyone on everyone today, Have having rest certainly interest a Thank our and event. hopefully your to care. We look May of attending your much. you so forward we appreciate Take great and big day.

Operator

today's This call. conference concludes

for you disconnect a your day. may pleasant lines. You have Thank participating and

X4 Pharmaceuticals (XFOR) 4 May 232023 Q1 Earnings call transcript