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

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.

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

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

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

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

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

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

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

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

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

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

Icahn of from Rheumatology Medical Associate School Research bone of chronic Medicine Chief in and K. Akiko hearing at the Newburger, XX and and Boston Tarrant, Director the Vice Dr. pediatrician Shimamura, University of School; School Medicine Mount at coordinator of and of Pediatrics marrow Clinical of syndrome Medical and Teresa Chan UMass Dr. Donadieu, Cell and a at a Department immunologist the French Harvard of Children's Dr. Charlotte Cunningham-Rundles, failure Paris Professor Duke program and myelodysplastic for Translational and of at Professor and the Immunology we importantly, Director registry the Hospital, Pediatrics Biology May Laboratory the Immunology. Hospital Cancer During professor Trousseau School Professor Jean be Molecular Peter commentary Oncologic at at Dr. of event, for Sinai; will Hemato Dr. in neutropenia; and

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

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

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

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

correlation Events published In Infection of this results to the we and we the be morning, This announced that our May poster also at also what severity Neutropenia. be presenting between is of Incidence XX. release the or the, a of we will CIS, on highlighting Serious Chronic the first study will research SIE assess abstract believe

the Central Time, XX is will X:XX our with adding at poster presentation, we p.m. be Concurrent to which on May website. the Saturday, poster

CFO, our the neutropenic to advance Adam? innovation year throughout financials. to our you it disorders. potential to we white in continue on chronic result turn other mission for As be quarter those that elevate to has look updating Mostafa, to blood as need. We with ability bring to believe published of I'll the cells, now its levels development the patient a breakthrough mavorixafor a to first WHIM we efforts populations our to these review to and to of progress syndrome demonstrated data forward our due our and Adam date, over

support quarter ended to company of sufficient cash, end Thanks, and had you XXXX. thanks $XX.X XX, believe being million to us with XXXX, the into At cash. today. for all operations We equivalents second in funds Paula, March on of the quarter and call the these cash first restricted the that are XX of

which million quarter, million the first $XX.X $XX.X period for expenses development compares the XXXX. for comparable in and to research Our were

payment R&D for accrual an quarter certain a of in-licensing $X company the included first million probable expenses $X.X that occurring. milestone noncash deems for the of and million expenses

the administrative selling, included the March quarter. for million first first million million compared $X.X compared and net million in for to period ended expenses were $XX period And XXXX, quarter Our XXXX. we $XX in as reported for loss a SG&A the $X.X quarter as of certain lastly, for noncash for to million general the of XXXX. comparable XX, expenses $X.X the comparable expenses

C the with first of $X.X expense the loss for call don't that, Net we of quarter. liability a and million open in your our the for warrant why $X.X for And Class included compensation change stock-based gain Operator? fair million up questions. value

you. Thank

Instructions]. will now question-and-answer begin [Operator We the session.

from Willey question Stephen first with The Stifel. comes

Julia for This I X just have is ] [ Steve. my quick questions on on end.

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

regulatory quarter accrual occurring. mostly $X probable the a related R&D that is this line to increase of that's be million and So milestone the payment, in-licensing for related in would or an

the change of noncash. that's that which course, is, there, So see you'll

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

And Riley the comes from next Mayank question Securities. with Mamtani B.

on Congratulations questions today. Two Mayank from and for follow-up. then your on William a continued or is one us This success.

So and at infection on see as that both you information we or well the CIS be your if extra presenting X could -- you'll -- the provide as far infection at just these what on might we curious data color presentations? as KOL, rates if any you the as and

then And presentations? to we each these expecting data these cuts in data one be follow-up. are from should of then different And largely be or new overlapping going

Thanks for the question.

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

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

one communities and So the be but similar. more are data investor is effectively, data of for audience to with then sets the be clinical the sort the quite one to going a same. primarily oriented broader communities, sets will Obviously, the is

you during II if relates as how III the guess, discussions you're is forward? very execution. when FDA you've and your I helpful. Maybe then study about terms that data provide Phase That's of plan Phase gained -- And Phase following going your well as, thinking any insights II in upcoming to what your could

for around chronic this studies? neutropenia clarity, So the is

Yes.

that. Sorry about

you. Yes. No, Thank worries. no

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

those now. right are progress in So

correlation counts, sharing neutropenic elevations the blood is infection. with on focused for and that, of durable both patients, data, in these then durability WHIM forward looking to nutrocells all including including additional white counts, the We're be of will the primarily cell neutrophil crosswalk looking which and

that very positive data Phase nice Ib dose a results. after saw came We the neutropenia out chronic with resoundingly single

from that registration to to the which changes the you'll and looking and with will elevated look is benefit infection consistent for of sharing that with and one. seen nicely similar will be data likely have WHIM. forward will sign a we're And then, more durable future of WHIM, course, that be venue trial Now hopefully

Congratulations again.

comes with TD Cowen. question next The Eva from Privitera

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

ahead. Eva, sorry. I'm please go

Yes. What's versus idiopathic and also patients monotherapy the and congenital the roughly of the split the cyclic with patients of combo G-CSF? with dosed split

of have to we've as Phase can't of -- study course, questions Yes. we're And we so can. the population actively a a because broad of a wide we nice the I any just similar to so enrolling, funnel. split still I trying we're mean, answer Ib,

we So more to have come the on that, only data.

for. be to QX. and aiming that's always around able some We robust of terms from patients, really data had couple possible. I for of the we're timing. between trying and we to forward But enough the or new of a it's soon XX. generalizations, you what to that update somewhere count look thought We're set sharing in then can buckets was of because and certainly meaningful number we're as as But of durability Phase as the across we course, patients aiming valuable QX think make recruitment XX as to in get of a Ib about And

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

can we year Breakthrough I saw share agency Phase the we what WHIM agency mean after controls. because which to granted about the on rates. Designation I what It I showed little XX% what -- of a benchmark, and a II, we you Yes. bit with believe data, II their were us a using of in in course, Phase reduction the with the a Therapy was different XX% historicals infection patients

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

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

H.C. The next from Wainwright. RK with question comes

This from H.C. is RK Wainwright.

job I were WHIM XX. together at XX and May on know if the I mavorixafor. my same about on X the should all do But you XX, putting and KOLs time, think I right,

mavorixafor talking where should used. about additional be neutropenic expect So conditions we could folks these to be

the conversations generated And of gets this to to SEM you -- SEM also, due help causes. would various by sense in where initiate regarding subtypes

up come Some that color conversations. around in these

to us potential about mavorixafor? think expansions indication for for So

much, so RK. question. Thanks Great

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

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

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

The next Kristen question with Fitzgerald. from comes Kluska Cantor

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

Sure.

at know in his this it's I a of conference. So participating Mark CIS, think primarily I has immunologist. team

it So I'll over him Mark? to to provide turn additional color.

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

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

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

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

Yes.

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

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

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

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

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

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

conference today's concludes call. This

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