XFOR - Earnings call transcript Q3 2020 | X4 Pharmaceuticals, Inc.

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Operator

Greetings and welcome to X4 Pharmaceuticals Third Quarter Financial and Operating Results Conference Call. [Operator instructions] As a reminder this conference call is being recorded. It is now my pleasure to introduce your host, Candice Ellis, Director of Corporate Communications and Investment Relations at X4.

You may begin.

Candice Ellis

Thank you, Operator. everyone. morning good And for today. much joining so us Thanks Presenting Officer, Dr. call, we Chief Executive have our on today's Paula our Ragan, Mostafa. and Chief Financial Adam Officer,

prepared joined we'll questions. each, and Quality, President remarks call DiBiase. Operations Following your Mary to of open Technical And our by the be Dr. Senior Vice they'll by

Ragan. those forecasted. risks today's may XX-Q. these the we'll These risks making and found development our to as be actual SEC recent research activities. forward-looking can Paula call as now with I'd a differ most plans, Form regarding The our descriptions As from well that the filed call, Form of reminder statements are in to and subjected results turn to to our forthcoming statements on cause like product in our be uncertainties regulatory over XX-K and our CEO,

Paula Ragan

for And stay morning. all hope thank you're you Candace. Thanks us healthy. everyone this call on We and to the continued joining safe

today operating to to are while ongoing Clinical our Pandemic we environment to due Development that the Mavorixafor We remains advance continue challenging COVID-XX pleased report Program. the

Let me most of review accomplishments. begin a recent with our

population. WHIM journal efficacy recognizes the are X in and We our in potential of data significant syndrome, the in Blood, procedures the patient positive for candidate announce of the which this safety we mavorixafor further Phase pleased publication to believe lead our

chemokine is mutations is in role receptor our Mavorixafor healthy antagonist syndrome reminder, being once-daily key WHIM cells of the and as chemokine is a caused CXCRX rare a the molecule a a small trafficking oral effective by developed immunodeficiency a As plays enabling therapy. of primary receptor inherited CXCRX, first-in-class receptor in a that immunosurveillance. immune disease

as data counts. on published doses in well the presented expands and regarding effect presented blood the specific patient-level including data cell of there total-white comprehensive monocytes of previously on as effects mavorixafor were the lymphocytes increasing new results neutrophils, While on data Blood

strategy data to primary pathway, in therapy the and X,XXX U.S. provided XX of for and the WHIM the Fast mavorixafor most be Track diagnosed challenging of is patients the biomarker for and significant than beliefs burdens. clinic supporting of the Development pharmacokinetic the American continue protocols, with key endpoints, eligible clinical has Journal manuscript Hematology of Track the to syndrome. recognizes to WHIM and secondary detailed of infection selection pivotal the the clinical published presented mavorixafor in our down important This up-to-date rates Official work for safety area CXCRX, the our extended clinical to CXCL on validation trial. our including results and data In provides the the regulating of addition third-party Society reinforce dose, benefits analysis of the Phase X a that by therapy clinical publication long-term The further ongoing the and signaling disease. Through support XX Program potential WHIM the the more This Fast the and thrilled to undiagnosed achievement be disease-modifying frequent mavorixafor granted also mavorixafor's the for for Designation by We're the key estimated FDA treat Plan, first data potential meetings and FDA discuss mavorixafor's regulatory WHIM. unmet the would approval potential syndrome need Drug that more endpoints

European of treatment Drug by and status the FDA by Commission mavorixafor the the for WHIM As Syndrome. the granted FDA as as Therapy was a well Designation reminder Breakthrough previously Orphan

an now Development update Let for Program Clinical me ongoing our provide on mavorixafor.

on Clinical focused surrounding COVID-XX we uncertainties our Program. continued advancing Development despite remain Importantly

regulatory and the clinical patients good We our the continue to Trial enroll of trial. Phase X progress across WHIM make in syndrome and aspects various

the sites its COVID-XIX own has regional- and numerous diversified and are countries considerations. each site-level which across world, around We of

services additional maintain enrollment to and the including working patient to impacts sites of visits are with in-home We COVID-XX. in trial such the as momentum mitigate the

further X the We and Phase anticipate syndrome data are in provide to intend to XXXX do top-line timeline WHIM on able as to clarity in so. continue soon we

COVID-XX-related severe In Trial and or this macroglobulinemia disclosed. of neutropenia XX-day XB we are to to we also to congenital data trial further we Concept had XXXX. make lymphoma, provide from to the X soon patients initial we delays form we SCN progress as our WHIM able continues anticipate around impact Our Proof in as some as a enroll to Waldenstrom's rare timeline are WM, do Study in although intend syndrome continuing Phase Trial, recently have Phase so. Similar of clarity

first availability the clinical our of second visits. to Phase of We a half order year, next expect In the half XB home-health we slight implementing initial the year. COVID-XX-related this previous patient-travel of are study in guidance the the of from results focused concerns in mitigate on delay

needs in the given our regular through Pandemic. COVID of with to We patients dialogue with Patient challenges are extended investigators Advocacy and our their the understand Team

confident are impact plan on have that we address the place effective to COVID-XX We study. in perfectly of WM our an

treatment will data study Lymphoma accelerate is discussions and this a of open-label these a this potential enroll and trial regarding collaboration assessing clinical dose-escalation of is and a data inform Clinical XX for between mavorixafor the safety FDA's [indiscernible] safety the the expect as ibrutinib. with with part The The that share trial important of XX being expected mavorixafor As conducted tolerability we combination trial and and in patients multi-center Trial reminder XB a in the registration will WM. and Phase Waldenstrom's. to signals, results and to Society future of as of the with WM development Leukemia [indiscernible]

the and additions experienced Comet, novel a a chief antagonist two officer our next-generation science NME served Art led Taveras, of new from team, Therapeutics as Art where treatment earlier was like us founded week this discovery I is as Lastly of the Dr. Company started to chief officer. cancer. of to and CSO role scientific Held XX joining welcome for to of CSO. he scientific Comet A CXCRX CoEnzyme is Privately his would new Transform on metabolism, Therapeutics, also the he where focus he who leadership Prior an at

Art drug addition global in Biogen, fit a drug chemokine-related roles key earlier evolution further his of experience lead and career. his towards aspiring held leadership, company. to expertise In chemistries, in With discovery decades and at and is our ideal discovery in at Schering-Plough, significant a foster rare-disease our initiative the R&D an becoming Art leadership

Kaleido corporate Board an as Lawton strengthens of our most chief our served fronts. Corporate and goals. consulting previously to as having Board. working In a strategic and Ms. Directors look addition a with financial across veteran officer of of scientific served our to as the unique expertise quarter. her brings operating Company, extended call member we results Advisory Alison who with industry many recently recently of the over Adam? closely again. of core appointment to Lawton, the that Adam forward for XX's I Alison the to I'll Board's Biosciences, CEO discuss With turn XX's understanding now and

Adam Mostafa

And you, to call Thank of today. thanks Paula. all you the on

financial this and As for morning, presented in Quarter press results XXXX. Third our the I activities will summarize of release our

million of September cash in cash, cash. XX XXXX, As restricted and $XX.X had XXth, equivalents,

were and into exercise our proceeds outstanding Hercules guidance proceeds any compared non-cash XXXX. and of in the our Note XXXX. million $XX.X XXXX, expenses quarter compared XXXX. certain cash expenses our million included for $XX early for of that million million in Facility expenses $X.X as third million investors $X as R&D cash XXXX. include the in administrative the equivalents comparable million potential ended warrants. and $X.X expenses September not for this expenses Debt September expenses quarter or our does from the XXth, XXth, XXXX, quarter cash to and from certain $X.X XXXX. ended expect G&A period million for to continue will were holding We Research non-cash included comparable for the $X.X fund operations for the contingent quarter third period the development of to of General

million the loss the for compared Operator? let's call of net net XXXX. million ended included quarter as non-cash to third up period million September for XXXX in Net With questions. for of was the $XX.X our quarter certain XXXX. for that, Finally $X.X comparable for XXth, a the expenses loss open $XX.X loss

Operator

we by Thank the you. Instructions] Please Q&A compile roster. while [Operator stand

Our with Stifel. Willey, Stephen comes first from question

is line now open. Your

Unidentified Analyst

Hi for all. is Steve. on [Helen] This

Just from me. questions two quick

enrolled So that and trials Is the first you the mentioned Waldenstrom's how some home-health patients you're so going? if Thanks. implementing being in COVID. Trial, of well going applied and that WHIM to mitigate for is you're SCN visits focused as from on effects same that the to strategy

Paula Ragan

[Helen] Thanks the for question. Sure.

is coming many Waldenstrom's. home our So implemented sense which Obviously over health is makes being the are dosing yes. for it months. where studies in for both and WHIM chronic patients The

solely health period actual of Waldenstrom's. so For sense two WHIM on makes SCN of home study's the focused weeks the treatments less given it the only and us

Unidentified Analyst

XXP-XXX, other? updates I Thank also two color you of just engineered across barrier XXX blood-brain sense. -- Okay. more That And And regarding know in you. at XX IND just XXP-XXX. any then differentiated each make you on Are then was either a could from you us I is being these assets point? XXX? remind if are can enabling those can this but studies the wondering -- give little how assets provide

Paula Ragan

programs talk going thank are No, really happy to well. Great. We're about that preclinical our you.

get after and is So clinics. the into the XXX typically two. is to It the most that about studies advanced it's year of approaching a IND-enabling then

are The example that is XXX between mavorixafor, been so some extremely the this we've the mavorixafor likely. differentiation engineer food does yes, does have profile meaningful, but a XXX not molecule. out We come seeing with excited it minor with effect for profiles and particular together of that of

but engineered to is of and step of have about again have also some forward Of worry prove for interaction And certainly that. kind everything course an to important looking profiles, patients entered drug-drug although clinicians so makes far minor, very we've to it -- good. out the that out not we

XXX it's development support plasma address. exposure to forward some we further serious of and late-lead moving excited of XXX to in finding nicely. So, brain again making between profile those that is and right we're behind progress that's good advance for terms to that hope cancers really brain and that exposure in continue optimization very to

Operator

Thank you.

Lee Our next question Canaccord. comes from Arlinda with

Your is line open. now

Arlinda Lee

expecting on could the anytime then any on be for on was syndrome? I provide I in programs. you guys. Might data the this enrollment hoping future? you. an update the curious presentations WHIM Thank Hi that we also feel preclinical near And

Paula Ragan

Great.

just to on enrollment respect So with WHIM.

with of happy say are and XXXX to the with not in pandemic kind a we're despite see XX's who to advance respect of site's his we that very preclinical program this With the so track continue specifics nicely chief -- someone enroll were the are to announcement any providing other to to patients the in Art on global that's it in to a trial. commitment our As to than drug commitments we're really to -- week have tremendous the discovery. very patients' seeing our various data great experience hiring we drug scientific and top-line segue and data with consistent development past, the trial officer, even and the to continue question pleased recent

any some year. updates coming So, tuned preclinical I don't we stay while around think on have because into data next our programs, have specific I think rolling we'll

Operator

Thank you.

Our next from Joel with question Citi. comes Beatty

line Your open. now is

Unidentified Analyst

Hi for taking guys. today. Two is you Joel. on from Shawn my Thank for This me questions.

what be. data about And then be just roll into getting [indiscernible] that kind next of talk you year, data? on reason could plan in out the the Waldenstrom's maybe the may First to results first-half how

Aside could that patient combo? efficacy on patients signals follow-up drug there identification, there and you on talk from we to of that kind any and any confidence you're many complete how EAP at are then your program are your seeing you responses, right give and WHIMS, the due see And a about biomarkers maybe if any would now? not where are right

Paula Ragan

Sure.

Shawn, thanks nice you. So from -- to hear

kind to walk of try your through questions. I'll So

as our support are aligned of of format sure in an working can sure will not robust be our we're the And treatments of specifics a people do the the key Waldenstrom's interpret. respect with of can that presenting the profile landscape, with of them appreciate context leaders single-agents there's of that data, we and nice major these fact the they yet drops trials more so greater for is something in communicate in only called opinion stay with of months though treatment actually a results six IgM put of that conjunction to response," the terms at their we which permutations than overall the well-defined important -- help responses making patients in data so of open-label goal date want day on in first but to certainly gets So to three set it for all the to With with unfold. to advances on world the make means to -- "a a present very it's CXCRX trial format we'll ibrutinib in about the tuned data the alone and the baseline. the our end so third XX% this

So like with patients of not mentioned those patients the six dosing, unfold. results finally with think first with to respect the we sites open and so half issues And as that and we we're for due year excuse XX% -- respect in some beginning people out sure to broadly sad to had the to part the like patients booths ID, on getting a I getting Europe, of making patients confident. reaching and just hope we'll obviously come IgM feel We more know, trial so and been the community you WHIM a profound this feel getting feel of also have to number many confident -- data there and showing commitment engage. various where market, actually nice certainly in There's patients is the directly. present continuing I'm think but the these are and and for them education U.S., the again it's COVID clinics that trial company. enroll unfortunately populations able we thinking and obviously patient from virtual of waiting. science-liaison in and months stay having we're the with you be but great respect tuned broader very a that to we've so visit progress pretty deep some the WHIM to to on own I been our then so incredibly us word are at we they awareness, data for of medical Between overall responses, first three to being conferences next teams, groundswell both the we get me about community to are beat is patient long-term we're their their starting With the

long-term the approval support trajectory to you company We're excited make know, progress. these the continue about So we the for patients. commercial mavorixafor in of of of hopeful

Unidentified Analyst

so Paula. you much Thank Great.

Paula Ragan

Okay.

Operator

you. Thank

Our next comes question Mamtani Mayank with Riley B. from Securities.

Your open. line is now

Unidentified Analyst

Congrats Hi. This Good all morning team. is [Sato] on the Mayank. on for progress.

morning. questions Just a us this few from

First XX-to-XX of and program now that sort year, know, a should off to Waldenstrom's responses expect as first-half it than related relates still know, anything the in enrollment of you on more and data you monitoring is this else terms patient date expecting range? more we the next

Paula Ragan

Yes.

I did is shift if sorry. -- So at I'm your that right? So beginning, or understood what's the the I question [indiscernible] what's get

Unidentified Analyst

exactly right. No, That's no. Yes.

Paula Ragan

Yes.

site -- again due enrollment just two issues the the is to U.S. ex-U.S. U.S., Sites. that's definitely primarily So -- the

are COVID, restrictions the travel that own can on are is depending -- to they home-health now eminent with to that good appreciate know, a has why enabling their patients on consistent. also As about you confident more flowing enrolling located with for been in deal where so direction and level we're comfort why there and and alleviate you which

again forward the around level It's see. next wait health just be efficacy partly sharing that we XX we see safety, drops, data first and of the In will their half of patients, an year. we dependent and it open-label to the in It's upon as trial. terms look IgM then have XX signals and to to

Unidentified Analyst

treatment at-home is because mean follow-up don't window the SCN remind Can just ClinicalTrials.gov? relates much the on I that in I brief sense program. do a sort of given compromised still as just of days is the enrollment you it that visits short know, patients maybe here? And you to then plan maybe manage Great. That's meet you XX nature us, acknowledging a helpful. to single-site this And concerns Washington and the you - believe, know, how

Paula Ragan

Yes.

weigh potential University risk clinicians not other and they open. their always I've update. so the patient-lens there's balance appreciation translational to well to the on to The of trickiest from study respect our think of with of something patients whether it's perspective consistent very risk are of for trial think with added or because make has end of -- the really day, terms Washington probably along benefits I need. been a so the to along is to And the this the type it with that But as the try COVID a ClinicalTrials.gov due or COVID, is exploring have need the for delay since due I trickiest sense. this this state a always that's our at and and on been mechanism do patients for in to that that risk these study sites that enthusiasm is We start use SCN's research investigator's open

So again just as we'll more. and forward stay XXXX tuned. of to story sharpening -- that It's look sort we a learn

Unidentified Analyst

Great. for That's taking Thanks congrats questions. the really And helpful. our for progress.

Paula Ragan

Thank you.

Operator

you. Thank

He] question [Arthur comes Wainwright. with next Our H.C. Yu from

open. now Your line is

Unidentified Analyst

Hey, everyone. for good RK. This is morning Arthur

question the I on WM or the just a have strategy. So follow-up quick

can dose we guys remind terms level could read the of across WHIM the the study and for... us, you So dose through the patient level optimal what in syndrome from

Paula Ragan

Great.

Unidentified Analyst

WM? ... Yes. Thank the you.

Paula Ragan

question. Great Sure.

drug the how as of in once X impacted therapy into day, two back] which is and So milligrams in we're Waldenstrom's, mavorixafor WHIM, drugs per ibrutinib and it's that single-agent dose let's consider low to drug-drug is WHIM though XXX we is do for just starting Phase that that [crossed treated the is milligrams are not by make potential, start we've a with that any that the XXX behind those direction between current per sure even at them. co-administering exposure rationale day. The interactions to need obviously

XXX started on day dose we a once-per-day that in WHIM we healthy we're that's at milligrams, milligrams already at saw in confident level hence per So what based level. a highly And active XXX volunteers.

You the the bump mobilization, PK also certainly highly very see known range. and active a robust are from nice white levels in trough blood-cell in

and to So escalation to dose and keep the so dose. starting being terms until clears dose where patient milligrams then after starting that of about of patient, dose, maximum you we're want good the get levels able we After are just to it we kind see activity. dose each clinical in per feel the XXX the XXX classic pushing XXX

Operator

Thank you.

Our next Christianson question from Laura comes with Cowen.

line Your open. is now

Laura Christianson

in if Hi. fit wondering just a Good more for combination might talk about morning paradigm On treatment for if question. for just to into taking my little it's be the potential approved, ibrutinib what's I we was you with guys. CXCRX-positive patients? [indiscernible] expect the first Thanks could mavorixafor line Waldenstrom's, and bit it used how mavorixafor

Paula Ragan

Sure.

with current patients is small combination, naive should -- with and of so of treatment see I our lines are start do, patients could we exploring to a study approach. a So, that handful say will up what front-line pair we'll of -- drug treatment to any start [indiscernible] three as potentially either as this

of is they what data so the most about opportunity with utilizing although in clinicians, for the are approved market more use I based minds combination will how it sets then of where gather treatment deliver. think data, a clinicians typically drug the So our Europe, treatments, there's ibrutinib other achieved consider U.S. clinicians' second treatment little and more for I the primarily data certainly in that in to of current the open versus obviously on the evidence combination Certainly and of bit will the what there's they line lines that drive then patients. makes line so will terms will this used robust and is how think will their does using of of in do sense kind to begin that ibrutinib data how that's

Laura Christianson

place, runway? one a just recently you for the I'm might quick purchase that And Adam. how put that impact then your how in cash agreement that expected -- wondering Perfect.

Adam Mostafa

Laura. Thanks Yes.

a So, to half, progress. agreement the like helpful tool continue the environment. and runway on stock our we business to in in -- little I right ways is way extend I policy, our represents purchase to I a tool the you now milestones or bit given primary based downside objective think opportunistic towards in think it key that more utilize as to the first protection potentially is think Waldenstrom's think financial bag track external about will, of but if we as progress the The insurance

in stack that that's of our up how terms financial So of sort strategy.

Operator

Our instructions] [Operator with comes Zegbeh question you. Thank next Jallah from Capital. ROTH

open. is Your now line

Zegbeh Jallah

update. Good morning Thanks guys. the for

just expect the or the line I'm probably ask preclinical again to some plan mentioned. think just I broad his on with one as of broaden know, to want about do pipeline to going programs? expertise, board, Art preclinical pipeline kind pursue you with your out to I how leverage indications of earlier you you additional

Paula Ragan

Sure.

discovery And given kind much. to incredibly strong so Chemo his overlap and experience Art we thanks have drug world. the So, yes, in feel fortunate biology joined

think when we I milestones. only need but near pre-clinicals our to our of think to really - course and term about also programs hit we operate intend So, two mavorixafor and - not

develop between in there's experience for what of learning that explore - support and and Waldenstrom's clinic our think certain drug. of total expand possibly drug in think, how that tremendous hopefully either in biology shows complementing and benefit to market with activity I a we can leukemias potentially lymphomas, prioritize will mavorixafor mavorixafor to and us CXCRX XXX amount we help and example I a the patients enable For we're or then really the the do could broadly Art's more

Zegbeh Jallah

you more provide try if the pre-treated pre-treated roll can sure noticed pre-treated you capping more enrolling that are even I you weren't going one answer which is But that or this or you comments last for the on to not but think any not I'm we we you study, can enrolling call what into here. were follow-up a you're non-patients? if said. just patients then And Paula. Waldenstrom's on Thanks patients can had, you

Paula Ragan

do - really disclose. do unmet to patients what there's when data. that's - line we profile with they the have remaining double to I need. to yet no their mutant no, of certainly of regardless that that therapy the Oh what no respect literature, like of back the share there's We'll address able point sort we're to we

do I consistency impact that or of data a we'll I mutation think So population the for next to and to homogenic CXCRX forward the year. kind having a us half creates sharing that of think nice study look patient important, that is in the first

Zegbeh Jallah

Thank you.

Operator

Thank you. Ragan back I'm this Paula would not like at questions the remarks. I turn to time. showing closing any further now to over call for

Paula Ragan

sincerely Thank so joining and us. XX for continuing updates to your key in thank interests catalysts you our you approach we again in business. much. look multiple our provide appreciate on as Well today to progress forward We

any have Thank questions, enjoy reach don't you of the hesitate further please day. your If out. to you and rest again,

Operator

conference gentlemen, participating. for and you Thank call. this concludes today's Ladies

disconnect. now may You

X4 Pharmaceuticals (XFOR) 7 Nov 202020 Q3 Earnings call transcript