X4 Pharmaceuticals (XFOR) 8 Aug 242024 Q2 Earnings call transcript

Good morning, everyone, and welcome to X4 Pharmaceuticals Second Quarter 2024 Earnings Conference Call. [Operator Instructions] Also today's call is being recorded. [Operator Instructions]

It is now my pleasure to introduce your host, Mr. Dan Ferry from LifeSci Advisors. ahead. go please Dan,

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

and open we Mary Dr. Chief questions Arbet-Engels; by Affairs. the call Officer, Chief Juves, Dr. DiBiase; Christophe Mark Officer, Baldry; José Operating Following and will to Taveras; Corporate be Dr. will Chief of prepared Scientific and Art up remarks, Officer, Officer, joined Commercial your Head Chief Medical Patient

be on forward-looking development As call, well financial a reminder, today's plans commercialization regarding the as product company and will activities and projections. making regulatory research as statements and

from subject These which expected of year's to XXXX which be in for including is risks this and description the risks filed found statements the can to Form the these company's in cause XX, most SEC, are filed differ XX-Q to with forecasted. today. later recent Form those XX-K, on March filings quarter, be second was XX's that A uncertainties may and actual results

I'll now to turn Ragan. it Paula? over Paula

had so far. Thanks and I'll we've everyone. start saying Dan, what year incredible welcome, by an just much, so

help the tenth an orally founded active a weeks we a with advance lead no asset, of us diseases to those our treatment to ago, rare few Just to company had XX. CXCRX When of few called we a group anniversary celebrated who the small options. ago, decade a have mavorixafor, vision antagonist

immunodeficiency. guidance the point. is at a our U.S. Sales approved and mavorixafor, branded XX and of only XOLREMDI is treatment being in therapy with treated years will and full for their a this we patients launch able now realize of the disease. of targeting future receiving older this on cause The come of are rare now vision XOLREMDI underlying commercial WHIM U.S. the April, team place saw approval XOLREMDI past primary were you WHIM syndrome, underway, As as to

highlight However, today, to I'd like our launch progress. strong

with launch date. pleased Overall, we've to very been the

Our completed into education launch quickly weeks had hit our on them was few distribution running. early our product got great channel a and enabling approval. field training to very commercial where teams, within we in and We of our May the first just of meeting success, drug patients product ground the a

through PANTHERx specialty we've with Coordination educator Rare, the already gone and programs. nurse on pharmacy, feedback and provided our received our smoothly, has patient support positive XXConnect services

currently via coming encouragingly, very have patients prior reimbursement receiving therapy with all and decisions And medical authorizations reimbursement almost on been exceptions. through quickly,

is team of commercial to Our fully and relationships are U.S., community. there We deployed for been ability advocacy strong has the with centers campaigns and disease on execute awareness and to continuing groups. calling on our going To date, well education hematologists excellence therefore, WHIM limited the our knew as this across leveraging key in immunologists. establish patient as

Modell Foundation weeks Deficiency physicians, Jeffrey York We participated a where members team in in the of with IDF, meeting New advocates. we engage of the visited with few our And Foundation Immune patients ago. recently or and annual the just

WHIM efforts, these community at treating better patient most importantly, and and we of all Through interest understanding building physicians, have engaging physician with of the questions around U.S. across become a the syndrome

in were extension The Society American that or our in were its Blood, Phase published Journal early continue the of from open-label Society, data annual meeting of the pivotal new in also at and Immunological from April phase III in the of the leverage trial These and Hematology. trial the presented positive data We May. to CIS, Clinical WHIM

no than in longer-term the for participants trial years. and was reductions signals of in more And annualized lymphocyte improvements counts have well time, some phase revealed neutrophil the observed poster OLE CIS the that treatment treated the as XOLREMDI during that at X as been safety durable with associated with new infection reported we rates. As with

We well chronic event this data late positive also our another made tremendous At our demonstrating progress months. with neutropenia, clinical in from once-daily for past G-CSF, presented investor of durably in Phase our mavorixafor immunodeficiency. tolerated monotherapy ANC, factor, June, neutrophil or rare granulocyte we mavorixafor as quarter stable interim that trial, II a development in oral of was clinical ongoing those increased doses up absolute as as generally long participants injectable and with or colony-stimulating both X and counts, to combination

achieved greater in Importantly, XXX% combination months date of on of completed XXX dosing. the and a with of target X valuable per the participants mid-May increases as and as at G-CSF X cells stable once-daily who than the cutoff monotherapy study ANC mavorixafor X-month had microliter

In X,XXX microliter to on XXX above mavorixafor per which per for mean mean the X the increased ANC's experts monotherapy as lower severe with range of cells and by at X normal ANCs monotherapy defined limit also CN. achieving CN addition, participants levels for ANC mavorixafor durably CN, is than less baseline, participants with cells months. targeted ANC achieved severe and patients microliter, And XXX of at ANC

a these on physician with stable the durable monotherapy G-CSF. and of the were from we positive, impact see community mavorixafor interim results has Feedback dose in to been pleased and as combination

the may needs data of physicians. able interim CN strong and belief to our their mavorixafor that be address patients support the These

and with an dose-related lessens risk in treatment and and oral for on increases treatment long-term that also levels described of use, desire G-CSF that severity toxicities the does own not used infections, carry G-CSF. the or ANC Specifically, for combination or with an whether oral its short

the present to Society to the This dose include from American or who Hematology, expecting data set will trial participants the of are those mavorixafor now from results of of CN receiving ahead ASH adjust allowed full in II November, meeting. Phase baseline. group and the data G-CSF from We're

success looks success U.S. III subsequent asked like what in looks know that The from data Phase is and be approval been for our we in these November. WHIM We've what the like already will news good study presented

mavorixafor Phase shared we over study, infection as neutrophils ANCs in the in duration and monotherapy saw lymphocytes June, can period, reducing with we XX-week rate, mavorixafor results that interim infections. meaningfully reduce increase and levels to II a sustained increased again and combination expected From that severity. a to In G-CSF CN

to maintain for we clinically ANCs we'll dosing the physicians' third trial. be be dose the stable June mav saw completed ability all the meaningful looking monotherapy plus at November, replicate across to that groups of in the reduce what who In patient's we'll while mavorixafor and supports From in participants looking data group, on and G-CSF mavorixafor X months G-CSF levels.

in safety programs clinical as for our seen continued date. other of mavorixafor all and of Finally, we'll be looking also to tolerability

efficacy, CN In and congenital global with June, XWARD idiopathic tolerability mavorixafor III clinical people autoimmune without oral once-daily primary pivotal serious the doses trial, of infections. initiation we announced with acquired also or the experiencing evaluating stable of in and/or of recurrent the branded who study and our G-CSF are safety Phase

The in XX-week track XWARD study and in trial XXX double-blinded operations team full the clinical trial fully multi-center are we to swing, The is randomized, is on placebo-controlled aiming enroll participants. mid-XXXX. opening to a enroll up sites,

trial the our decision as data that Phase support presented initiate III date design. strongly We Phase to well the CN believe as this to II interim

III ANC trial infections. our increases derisk XWHIM that those and clinical results trial, believe in the Importantly, in interim ANC given III reducing Phase benefit mirror led that levels completed the severity the the where Phase successfully of by seen frequency, to CN of increases we duration similar to date patients

syndrome. As to the looking we global focus in opportunity XWARD maximize our we CN WHIM on trial, the efforts are also

we submission the MAA to efficiently an XXXX, opportunities while for expect We in syndrome to WHIM Medicines potential FDA European mavorixafor explore leverage U.S. we geographies be where also in additional by our may early Agency approval. able

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

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

of cash. review or recognized million receipt. a we XOLREMDI, upon As sale disease PRV, were gain mavorixafor's designation. after based this third we shortly monetize the we party previously in had in PRV a awarded discussed, very to a pediatric $XXX And for priority voucher, May, FDA on rare on We approval

the these runway into to support So to note XXXX. that late want are not operations as cash, $XXX.X end restricted in XOLREMDI short-term include potential cash the and had second does XX funds cash equivalents, projected this our company I marketable sufficient believe We revenue. of of quarter, million securities. future

costs, intellectual we sale June X including revenues and of property. of $X.X $X.X and approximately includes an and of the XX, cost months million asset revenue XOLREMDI. underlying of of reported amortized license the ended life intangible royalties the capitalized of operational the as sales-based of over cost to For million million related revenue This milestones $X.X

reflected is stocking We and pharmacy in a would small was number. during the sales at amount like inventory to specialty quarter, this that note of the our there

million quarter and expenses of million for second were development compared included and the in second XXXX. $XX.X in expense. general million comparable $X.X the quarter SG&A $XX.X the Research XXXX. expenses period comparable quarter quarter million R&D the to XXXX. for noncash also compares This the million of second expenses for were of period $XX.X expenses administrative included to of for million for the expense. for $XX.X noncash second Selling, $X.X XXXX

related well noncash company's sale net in reported fair net $XX.X the the to liability. $XXX in gain of the value compared the of XXXX. of warrant Class as to as for for $XX.X period XXXX that income included period net current quarter for of the Note of we of a million million comparable Finally, PRV the $XX.X million loss second million income a remeasurement C the

concluding Paula remarks. I'll now to some turn it for back

significant As company. integrated you a XX, step now can accomplishments for see, fully of mark forward these all a biopharmaceutical

uses to for clinical we XX look on the mavorixafor forward X to sales, maximize over we coming As the our and potential reporting regulatory global to and continue for months. progress additional patients, of explore

for We'll your Operator? up open the now call questions.

We'll first Kristen Instructions] to [Operator morning Cantor. of go Kluska this

It's and years. great the milestone XX better year. a reaching this on drug with Congratulations celebrate approval to how

to I primarily it infections patient So success setting around measure you how versus going Will in ask XOLREMDI. for to are believe anecdotes, clinic commercial differently a wanted else anything first, reports be the on or to physicians consider?

Mark it to perspectives field. they're over give to the what turn some I'll hearing Kristen. from on Thanks,

been underway, that Yes. launch patients targets underlying And is well treatment what's of We're a physicians we're cause exciting and Kristen. for disease. the -- waiting the that the hearing have that is

up practice patients help think XOLREMDI, these we then can of now the us patients about in and become help And approval opening really to these so physicians how the conversations the XOLREMDI. disease, it's they recognize aware with allowing and of have their

research from and know of the payers. on for market physicians, the We that value the infections XOLREMDI impact that drives really for it's patients

for that's more experience with will I what as product. they get looking physicians think So be really the

going how us of discussions U.S. give ex sense partnership a stage? you can And this are at

mavorixafor of CN. value your noted to release ways just And press the WHIM curious, syndrome you're beyond and some looking maximize

So curious come in also other in these well. ways indications conversations, this drug partners with they up about potential utilize has that can this potentially as

Yes.

mean about So in thanks, that that regions think there I existing approval, certain when FDA the are can currently Kristen. approval. the leverage we world

exploration some there there's So ongoing. currently

Adam side can comment of potential more the on collaborations. -- strategic more

Yes. Kristen. Thanks,

as we ongoing partners. this interest appropriate interest update we're neutropenia our back But II and is only and in We'll there have certainly, So recent positive Phase growing there's recent if potential I discussions the data, come market as certainly area. chronic going Paula transaction. to think and given approval mentioned, the with a consummate

Piper Tenthoff We Ed Sandler. now to at next go

know hundred some thousand Yes, to that at of the it's will gone see work has get of I here. company a excited lot first that grow dollars, from and to couple it revenues

us patients drug. drug? many And anything My us commercial Can if are XOLREMDI. patient a of with sort of And do patients maybe about you little them to sense able into of journey question get about patients backlog? the direct of give that seeing that had not sort of sort as give how us are you're characterizing the sort you're for a to the tell numbers, on bit of you on use

Thanks so much, Ted.

made be commitment the and won't pleased of But mean, that the the with in we experiencing continue that extremely I down cascade communities any out. on report as then think just receipt of company now I breaking head the overall are to commitment revenue. we're So we has details and

diagnosis can But support of in drug. on some more way the seeing add Mark the how color kind education from through journey they're all and the patients

ultra-rare to is disease. Yes. And this an thing first the think I remember,

campaigns. launched groups through extended key engagement both And begin our we're through refreshed really digital patient XOLREMDI our is with because -- where the advocacy get we've here campaigns our marketing physicians, conversations also so having branded that's awareness, the reach, to awareness our We've and raise our also disease to we with campaign. and can digital

this creating really kind we're of momentum So around conversations. the

have the the -- in the extension the were on in been XOLREMDI that patients as U.S. of mentioned And open-label patients here all Paula prescribed call, the

more have their there, physicians. we're they're treatment And having a that so and from with waiting just targets conversations patients how the for more been learning those and about now disease,

We go at next RK Wainwright. H.C. now to

Congratulations on years the XX the launch. and also

know get-go? of getting you terms what anything better saying from when -- early this terms of adoption it's adopt Immunodeficiency patients of looked go -- to to as hearing physicians such at these is be and conferences, improving, take else the the to have getting to in and launch. also learned of And from stages attend that that -- ready you're needs -- at I'm I terms drug. In are improving you level not But how it the point in Conference, the a

RK. much, Thanks so

kind of to will number we're oral-targeted this and I'll the about development. very certainly, what with excited hearing and cascade thinking the even add one, And is, add a think targeted and therapy. therapy, we're pleased Mark I they're of

a with been field It's study and of of rigor. injectable varying degrees treatment

phenomenally Our infection Phase successful across burden rate III severity duration. reduction and was showing

share. to great set So with a data to story and begin that is a great

disease to of And benefits it they're even Mark the well. XOLREMDI, some journey of over on turn add the all own I'll as about but to their course, the then then in color clinicians, of that learning to understanding terms and

Yes, exactly.

now, in really now really had energized disease. practice patients physicians go these product, treatment have have it's back really it's and who, think for before available their we look I no for to really approved an that their

XOLREMDI. reason And so a the them make back, about now actually have these patients, and then we've go look conversation a to given the for diagnosis with patient

can data indication, then and for so for SCN this between if other said. so presented be the moving that that there program is now any is analysis how will Well forward? understand study, we And XWARD an there is the interim

Sure.

giving in So as year. Phase full we shared set the II data this be of today, we'll November

of pleased course, we was with but interim. the were it So extremely set data already,

the So we've to as already responses G-CSF. responses addition and in drug of then durable seen monotherapy, durable and sustained stable

puzzle is get ] final G in reduced. the circumstances exploring piece just can [ what So

certainly come in November, full be is will So that set data which going timed. well to

ASH, out community at before word Just on our we'll CN meeting trial enrollments. also ASH to help support the be further and the the get

how appropriate study look that excellent XXXX whole giving certainly, an be is to and going. it's then perspective, So timing But a will we'll forward update. as on updates the

We'll Brookline take Cann our next question now from Leah of Capital Markets.

I have a financial question.

at you including help shares the fully end with So quarter, Adam, us warrants? could number of prefunded the diluted the of the

Sure. Thanks.

So there's million outstanding, prefunded shares, about shares warrants. XXX million XX regular basic

warrants million will. XX $XXX equivalents, share that are only. of are then you And So if million there cash

X XXX divided tranche. tranches, into million are XXX tranche, Those million

are are a shares. you'll diluted So in warrants money, overall, some the included, XXX million of today historical piece. fully in accounts that XX-Q see our that There's later for that small typically out but significantly not

Willey Stephen Stifel. to next now go at We'll

bit process. you but required of a little timing And might drugs understand somewhat the I curious a the kinetics what I you just from the that -- experience just higher-priced can look thus early process. far if on have be read these perspective. that early, paperwork disease again, for the process just an it's as and is limited based that Could maybe like a reimbursement sometimes to just lengthy about know rare talk

turn it to over Sure, Stephen. I I'll mean, Mark.

insurance experienced think this. its good disease patients. given naturally team that had a is, I magnitude ahead well of think the But lot typically options provide, for the classic of news we've of very and barriers the I ultra-rare drug a has these companies

to Mark insights So I'll it some give there. to turn over

for Yes. payers Yes, the mentioned Paula drugs. standard are during using call, methods their as new Steve.

to so and looking And But and quite able patients access have PAs they're at process. XOLREMDI. smoothly, are working exceptions these been

Of -- patients we our are ensures course, it's process which reimbursement. of that But our smoothly. through working have this through our quickly access also the full while service programs work we patient to And see to patient quick the product the XXConnect, hub. good is get suite

one guidance forward? communicating Phase we to XWARD actual in a not how Should thinking progress That's But sales. just III related the And curious going about Okay. maybe enrollment you're question, helpful. in about milestones, a just going is to this updates hear of be with about and hear it at conjunction it? when expect Or say, kind moving XX%? we'll like trial certain target we

Steve. Thanks, Yes.

I think meaningful we'll aim always progress. to communicate

this Of and it's course, point. we at a don't commitments any want year to target, just away it's a because XXXX make almost

we However, to to specifics. to certainly, I zone XXXX again, more our would we'll be get that's provide confidence share. belt say But under as a some here, able

maybe SG&A anticipated to about just XX% then in Adam. wouldn't guess for looks launch. have financial down I And undertaking sequentially, another Okay. the of context be which a question I just been

quarter there's impacts you item? So quarter us just unique either to that anything number the line this that can if remind or X number SG&A

Yes. Steve. Thanks,

the of So probably more quarter second a typical number recurring figure. representative is

The related first we sort year sales early number and of the approval. of underway building costs last time, launch quarter, force of a start-up to nonrecurring ahead the onetime had mentioned as a in getting

So was I will, SG&A going a than more if the would that line quarter inflated, little in expect forward. you

drop that's it looks the So does. why as

progress. Congrats on the

to next Zacks to Bautz Research. go now at Small-Cap David We

of Are if when filled. to in how basis? monthly they bit it revenue you a filled? a prescriptions the little are then how filled prescriptions relation on recognized multiple Is on could Question monthly? those is mechanism about talk are And

Sure.

for I line, we of therapy. patients. commercial obviously our are patients individual blend don't disclose the well both inventory play-by-play revenue have with as think now I recognition mean an current build kind for does we I as our it mentioned on to So intended revenue mean, that as

-- helping in interest But to more at this market's do understand think the ready details. we do we're them provide any certainly, don't want I forecast. we So point,

head which So we'll as think as grow later XXXX, year we we about metrics, provide try over benefit some you to bit really more product can into and all to this will the provide help time. a how

Ladies Ragan, to you, ma'am, and we to I'd this gentlemen, for closing back questions Dr. further no comments. it like morning. turn things have any appears

Well, thank rest an care. and joining today, we of everyone, have you day. your for hope the excellent so much, operator. Take you, Thank you call

we a today's again, us, Thank earnings you, wish Again, Ladies conclude Pharmaceuticals thanks you gentlemen, for Ragan. all And quarter call. does joining much so Goodbye. that second again, Dr. and XX great day. everyone.