FULC - Earnings call transcript Q3 2024 | Fulcrum Therapeutics, Inc.

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Operator

Good morning, and welcome to Fulcrum Therapeutics Third Quarter 2024 Financial Results and Business Update Conference Call. [Operator Instructions] This call is being webcast live and can be accessed on the Investors section of Fulcrum's website at www.falcrumtx.com and is being recorded.

Please be reminded that remarks during this call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, may include statements about the company's future expectations and plans, clinical development time lines and financial projections.

While these forward-looking statements represent Fulcrum's views as of today, this should not be relied upon as representing the company's views in the future. Fulcrum may update these statements in the future, but is not taking on an obligation to do so. most discussions with uncertainties of the business. Fulcrum's Exchange Securities associated to company's for Commission certain refer recent the filings and with Please risks and

President will Sapir, Leading be call today CEO the and of Fulcrum. Alex

Alan, will Alex in providing Financial Dr. and Chief answer key updates Pat and Iain on Pat Alan President which Vice there Officer; Officer; Fraser, Medical our the Senior be are be Chief of will Joining Q&A available on questions. Dr. your Musso, Alex, a call brief Horn, Development. Ian programs, After to

the With over that, it's my pleasure Alex. to turn call to

Alexander Sapir

That's upcoming our everyone. end third morning, I'll call the milestones. great. on Alan provide I for call. questions. conference joining updates Today, then I'll review we'll hand us and finally, Thanks, to financials. quarter And Michelle, over by you and appreciate to your progress good taking with you recent

review the in update a with the we of begin on provided September. very I'll back program brief losmapimod

recall, of we the primary efforts As the losmapimod and on order on a not Phase pipeline. in we secondary endpoints these light from key focus III and in reduction results, and group reported the separation research of of did preclinical you'll workforce development suspended trial. placebo or approximately In the that XX% our development losmapimod REACH to announced show our [indiscernible] advancing

the trials advancing of fully administrative reduction losmapimod both of community. to our community. sharing patients, as the my FSHD take full committed research coordinators and your results trial the across benefit who program, the and the support a express FSHD and to remain of positions We do functions. moment the I sincerely for participated society physicians and included want as to appreciate REACH of This FSHD the to gratitude we and all the to in general broader a development well in clinical to

improving areas was what with with not advancing HBF pociredir, to diseases entire the of To need. II, cell potential Phase that shown patients disappointed deeply lives the III of disease. excited that of replicate in Although oral genetically results Fulcrum unmet the inducer defined we organization mission in patients of end, were committed for did treatment to remains Phase sickle are we the continue our high our

Sickle transfusions, disorder is hydroxyurea, approximately cell for severely patients focusing the relief. disease quality XXX,XXX U.S. standard and life primarily for treatment disease cell inherited and the people pain in a million medications X.X lifelong blood with that approximately worldwide. sickle involved on blood of symptom Historically, of has people impacts

Despite the are recent therapeutic approval by approaches, recent of cell for symptomatology, safe protective and oral of that we Oxbryta. withdrawal of a global believe further remains need unmet broadly the sickle accessible gene which is editing there underscored significant options

we HBF As first-in-class inducer, oral need. a to unmet molecule has pociredir believe that the this potential small address

our trial of we in sites. progress Ib enrolling make and PIONEER activating Phase trial, call we Now to the continue patients pociredir, which

PIONEER possible expeditiously the trial remain data track in on focused provide on development the of are progressing to as from as We XXXX. pociredir and

Based site new the more early intend enrollment and our guidance on detailed year. we current our data in to progress activation trends, in share plans to on provide

followed at X dose XX-milligram months reminder, trial of the of the this at once-daily cohort a evaluating dose X duration and for months. dosing the is by with As X pociredir also be once Cohort a X XX-milligram daily will

to are enroll Both patients. cohorts XX to expected up

intended program are These report comprehensive to studies recent I that trials the also support of interactions are for in initiating we pleased the FDA. with addition to ongoing to we pociredir development PIONEER following In healthy are Phase clinical trial, volunteers pociredir. the

patients disease. increase may supports any And disease fetal achieved with levels cell asymptomatic. for hemoglobin their beneficial become high mid- XXs, sickle sickle presentation hemoglobin cell to is literature patients the in when fetal in that most The importantly,

has disease. provide prior the hemoglobin, clinical disease as to inducer fetal in of of magnitude look potential to the building total with We which to fetal cell could that improvements We sickle generated novel a encouraging believe forward for translate hemoglobin the data differentiated a demonstrated severity. on pociredir meaningful that that pociredir hold people therapeutic living a increased option into

DBA; beyond our for advancing SDS; in Syndrome, Fanconi or such focused in studies candidate additional future. development Now diamond information fan our anemia development regarding IND-enabling anemia, anticipate on CampX. pociredir, aplastic agreement also licensing inherited near and under programs plans and we sharing Anemia, a or early stage Schwatman black are with We as Diamond the

our I we CEO announced evolution. CEO to experience and Directors Rachel's the well companies, several served morning, of at Bio, at joined residents Executive in is updates are as well we with has some NEA This biotech as on Directors, provide also early-stage management Fulcrum's needs that of the where having of given aligned front. as Board Board wanted King our Rachel and

She to role excited be and will deep Committee. advisory currently who I'm scientific Jim's to Rachel also have on Board joining and pleased Rachel to continue benefit judgment. from transitioning an Jim will the Science will serves our of Novavax Collins, sound personally acumen that replacing we on us GlycoMimetics. Technology and be and

development our joined Hematology Now hematology Thomas AstraZeneca. to branch has transitioning clinical us PIONEER our President of Vice at within Agios NIH focused in also career management assumed program, numerous has for the Dr. distinguished study. hematology of on the before Winkler a assets where hematology front, he September developing responsibility hematology industry but and the for on both the and clinical -- Thomas as for

this development talent we has at team was building like joined medical and to study Additionally, year. launch Pat this Horn, earlier clinical out hiring of and REACH decided year retire prepare Officer, Fulcrum the for Medical the losmapimod. key Thomas in Chief as the end instrumental leading our Pat the of for

of us appreciation Thomas express bringing for with who here Musso, hematology to in strong the Alan like his to would the Chief today. fulcrum as background as critical over to Pat, I you. a Alan, I through with over you, is turn room at that, to to it my Pat Thank Financial personally will run well time. thank Officer, And such now our numbers.

Alan Musso

Thanks, cash now financial review I'll our Alex. with position. starting our results

cash, activities Sanofi and XXXX. XX, used position quarter, payment $XX September received The upfront XXXX. were December marketable cash partially million the XXXX, as XX, of cash $XXX.X offset the in to securities $XXX.X by equivalents our As in we to our operating second million to compared from in that fund of cash million is due increase

the the the third decrease $X.X for quarter $XXX,XXX our had quarter in completion collaboration We agreement XXXX. was The revenue of of to third research no XXXX quarter to XXXX. our with of the during fourth of under due MyoKardia collaboration services compared million of

$X.X development our XXXX. third research for of Our partially The Sanofi were costs under decrease due trial. $XX.X quarter global collaboration and increased offset was of Ib compared advancement reimbursement to PIONEER million the for the by related our the third expenses losmapimod, sharing development the for of quarter primarily $XX.X million Phase to million with of XXXX cost to

to XXXX. compensation expenses implemented quarter decrease a in primarily employee the for the XXXX. XXXX million in the administrative million to of were quarter The for of costs due and third of workforce of as of result decreased third $XX third quarter million our General $X.X $X.X compared was reduction

of compared million of a XXXX. of loss third loss for $XX.X quarter the net million of the The XXXX to net quarter for third $XX

Finally, to turning cash guidance. our

burn XXXX cash expect end cash be will to that our million $XX of equivalents expect $XX million securities, and We $XXX in with million. to XXXX, we approximately cash, and marketable approximately

our current cash our operating Based cash, on equivalents will that current to be -- XXXX. least expect sufficient requirements at our we marketable operating securities and into plans, existing fund

to Alex. the me call back let that, you, with over And turn

Alexander Sapir

up Thanks Great. overview that for Michelle, ahead and And with and don't go Alan. business open so much, Okay. questions. why the we of the think financials, I it

Operator

will come with [Operator Instructions] our Oppenheimer. Matthew first And Biegler from question

Matthew Biegler

just the there disease Maybe avenues? want you assets, owned focus you versus for kind seat assets. therapeutic with wholly do next-gen that tech Alex, maybe focus new the are of platform And areas the I to it going Or thinking keeping something the in? pipeline are difference new the in hematology partnering open out? to mean, question, How exploring should about certain how you think forward? high-level wholly and of owned a you're between on

Alexander Sapir

It's great, Yes. Thanks Matt. so much the question. for Yes.

Fanconi Swagman development in of DBA, include around or rich program I say pipeline, have most preclinical and we program candidate do that that would which syndrome have near we anemia, more inherited updates remarks, expect mentioned to So preclinical a is X for a as study. the selection have, announce future during the very I aplastic that we advanced SDS, do the Diamond IND-enabling the and do we anemia, prepared syndrome, our

add for with for there advanced that are far those of really global work I then in that U.S., always right efforts. with to ourselves. signed of by And the focus the time in you a And came is very progress discovery losmapimod. mentioned. our rights these in from the is doing that for going that obviously, what of we our those work some think So have I deal we're interesting anemias is now, markets potential notable you outside to our anything similar option to going But we've Canfor most as year. with last on program. for continue the we an with September being, the deal the some the know, potentially did is inherited licensing to progressing We on would do Sanofi Most that? plastic we very, got Iain,

Iain Fraser

pretty comprehensively. I No, around that covers think

Operator

come Stifel. Dae our from And will Gon with question next Ha

Dae Gon Ha

Congrats questions guess on X pociredir the progress. specifically. on I

the request have to in it back in the information healthy filing, seems volunteers. potential information that XX-Q you your be Going conducted been risk language a any risk may section, further having or studies around this on define like for to

healthy intention another our You announcing to do are study. volunteer

sort what in feedback African review. withdrawal curious I not led for FDA from the study. to FDA curious conduct, trial And FDA meets sort second, just terms on exactly the the this latest what the So of the that that whether run having or feedback with guess, the then of Oxbryta of concurrent standards PIONEER the been you post have was

Alexander Sapir

to add for hunt relates content, probably color around over the -- Sure. on Yes. as a little Thanks, to question. I'll it that Dae And of second Ian. African bit first put over Gon, And put so then Pat. question to to much the I'll that over the I'll Yes. that question,

are, remarks, the clinical the to prior plan think to mentioned eventual off prepared we progress we when in package forward was additional that just we to preclinical data with remind that the required bit additional order work into that no will that I the the did inform maybe become the really did let with to going discussions I to require with FDA that There turn the everybody of to healthy on call, into and patients. on as a NDA. me based get in to get are agency, clinical work volunteers. hold, that volunteers the there some FDA need the I was the moving more in more we've So over or us had But clinical think specifically, Iain, do we development little healthy detail. overall And

Iain Fraser

Yes. Dae Gon. Alex. Thanks, Thanks,

or a more they I studies and So look as been clinic that studies development formulations at of healthy the compound. volunteer were me part of and that get routine are delineate find planned into interaction to drug-drug the of of studies their the There all metabolism important along the these that excretion evaluations some think introduced have recognize new to compound. the studies of studies

that are studies So development those always program. drug were of standard part our the

the the side around volunteer risk healthy resuming was at of there course, then time there's studies because, no was volunteer out participating was on individuals differentiation studies. And patient refining and this that population mentioned, studies benefit. and between to segregated of Alex entirely clinical patient the benefit the As hold patient in balancing healthy

strong. link as the development of the And between There's really plan. of no and was the those the so of coincidental we've oxpritit studies now around overall studies It's with timing no progressing and part perform the are those resumption withdral. pride

Alexander Sapir

the maybe then, to will They say just And actually Yes. we've it been seen pleased Gon, with coming we from we've your I of turning of And quickly have relates African question it patients second over for that before enrolled Dae pretty that some that date. countries. to are I think date. as to real the to some Pat, compliance

more maybe provide detail. In terms that over in take bit the coming on from of regulatory patients sites to I'll Africa, little turn a Pat one to

Unknown Executive

Yes.

from data ex history FDA a sites. the of U.S. So long has accepting

In fact, where most of geographies. different history. population now such cell in studies anemia, really a is There's long global are the as in Phase III the concentrated large sickle and size diseases

in of of that select I and sites the cells and about sickle part think currently The in included FDA. them approval by most Phase sites review we in have especially. sites the only our experience regulatory that ongoing the before And audited been are of even ongoing in Phase study, sites at concerned Africa, to progress majority of are that followed of II GCP. have we have some that studies most the they and activities III studies. have previous sickle by FDA really the cell agencies And these have and led in quality as we're data looking experienced is sub-Saharan each

ability acceptable the studies. for the confident feel their and to I we registration So Phase produce then study and even we've for selected data future sites in

Operator

question come Edward Piper next from Tenthoff with will our And Sandler.

Edward Tenthoff

of you following together up Pioneer to to questions. and on provide you the of into a and can mg roll a quickly how do it's kind Gon's available? would Will out on any you envision may year. study? guidance little in And registrational Dae XX as split get next So able that wanted data terms from reported report this you it I how -- being all from transition better XX sense

Alexander Sapir

part to then XX-milligram much it XX milligram. your great. the the address which Pat question. we second the said in part intend turn That's do as I'll question your of the Yes, run the we've question. Yes. first of so so is address to being to the past, Thanks asking share for over and I'll sequential to cohort,

data out at will different X in those sets So in points come time. XXXX.

do share And independent we those X to another. of so intend

we right we've in with are data when We critical that the new We right we'll absolutely back And as and more XXXX, sites now share. patients, enrollment. add are to once everybody sites. said, have the that that progress build in we are we're to pleased with to have now place, we'll seeing specifically continuing of mass with always specificity, come

now, is guidance. more a in give some share to prepared our premature new So my data any But more specific in to do early XXXX. plan as to I think detailed provide of right early year remarks, the bit it on intend said we I guidance in

maybe terms So I think over more study next turn to of Pat. specifically about your I'll what's that one the question this, after in

Unknown Executive

Yes.

define So Regulatory here. Thomas in Designation, Winkler working are been to Santhe's plan use I we regulatory been our FDA, there He's to we program and to Phase of. with the with of really Since our multiple Orphan make study entitled possibilities. following we for which have group development are Phase And Drug clinical the pociredir the Head Ib.

the the be So may to and a be to as ability the development take PIONEER II, will study. that or that the study, may from data and a take the separate be FDA quickly may FDA. III which we'll depend be But data followed to Phase the it plan a study PIONEER then from Phase their III that the Phase a on And by agreement. II Phase get -- clinical feedback move registrational may from

Alexander Sapir

hemoglobin become And patients disease survival. we've once the symptomatology improves Yes. only asymptomatic. that fetal as the that in to many Ted, Their mid- of show then, with not overall I times, high reduction other would is that also increases of XXs. but sort VOCs, in maybe evidence to in shared of you add and abundance terms And thing only over said you get does

right may of think as for to as discussion further a our time well one certainly of discuss in potentially -- is to program. one when that the hemoglobin in development studies going as endpoint surrogate I use fetal we Pioneer the internal So there's development further our

relationship the that's as well there as -- reduction on strongly how fetal noticeable but very on increases just in think ongoing, survival is that show to data based that out important because work to an still impact make point in a have the So very, that is is hemoglobin I VOC. that is

Operator

And our Kluska from comes next Fitzgerald. with Cantor question Kristen

Unknown Analyst

This is for on Kristen. Rick Miller

quick follow-up the volunteer a studies. healthy on Just

trials? about PK/PD in Phase about of a this ad. these will you the measures interested You've look initiating. thinking in safety? trial as talked be at. relates at looking terms dose I of How And are And in mostly kind any dosing of strategy are getting previous you kind look you're to main here, outcomes how this there Or

Alexander Sapir

Yes, a question, answer it's great I to And Rick. think that.

that X Ian. me to kick Let over

Iain Fraser

perspective distribution this gets studies both It's label The PK are drug. done the study of of a Rick. Thanks, case, looking development. absorption, the metabolism predominantly radio the in at in from profiles admin, standard of Yes. that

that's example. on and the the blood the the other So one yard of measuring PK. And teed the focused up studies drug the measuring in the the excretion that's and in primarily are studies for stool, then

So interaction PK new on they formulation and older in is the compared given posed of are evaluate of and the the used to and drug-drug the for you drug. studies order studies pharmacokinetics into giving presence development commonly formulation formulations the performance that typically other plasma in agents be single-dose the impact where volunteers new healthy any to of will come as measure to the

as clinical in to So tolerability doing. back and inform the all important at endpoint be is those the the in around patients studies, the part development We program of those goes of but what are studies of the a that driver safety look the line the are program obviously that as in and PK. we'll overall results key

Operator

Capital. RBC with comes next from Renza our And question Greg

Gregory Renza

programs assessment balance reflect course, discovery experience, programs. early also, inform maybe from if bit bit looking secondly, are but what's value of Maybe a sneak a how a third XXXX. internally runway you journey that couple you've the that be assets. spend externally risk the using the and one, I pan resources you could How future the that I'll programs, versus of of but as about just about How to losmapimod you we us, and with the just should of proposition, early just thinking some with not the to at Just looking to of Alex, you may, with for And thought does on pociredir, as how comment And maybe discovery out? expect then for the talked this, that all implied there? in a related you're Alan. if camp allocation for about

Alexander Sapir

Yes. Thanks Yes, so losmapimod I we think if do much, versus pociredir, contrast they in Greg. of so questions. I Great are very different. set of a think comparison

I losmapimod very III was we dependent. novel is with going endpoint, new Phase the effort of some think knew challenges a study it that the and into was it

patients I postrider we think study abundance as crises pociredir in and what in were we literature that and direct that fetal see about impact increases of a we the in what that as patients, in to there enrolled the terms the initiation as increases of the overall out well know hemoglobin impressive on of hemoglobin the prior to have fetal survival. well hold show with saw as their very

comments, the with different very Obviously, internal some strong the we group closely fetal a cell X forward. in see see the around And sickle as it discussions after given consultants them been of history endpoints potential surrogate ex-FDA agency a endpoint. mentioned agency having I I move to make is, using think use as think, and of we've surrogate we agency sort I very I to them when Pat's working very last have we evidence are the hemoglobin other the to does how as really I development programs disease does as the time of approving products or is approach their within end understand, know. surrogate as point as sense earlier, right

that very Alan think mentioned, as of externally, our balance discovery you with and of we've think sheet success is early got all robust sort least a I programs. versus into as XXXX, at assuming about

Phase completed this III. pociredir So once Phase study the Phase that's into program we Ib II advancing and

bring and we go a were at smart what have but out sheet lot early doing of but selective. If of highly is, a terms, look be judicious going been decide our think have we've things to on of history I and potentially terms The had the bring externally we'll and to So in in the assets, financial discovery. continue has ability opportunities exciting at think, certainly company those in. robust external to we very in-house, been given very balance we've deals, deals, got we to I and on of look how really judicious excellent to sort

would historically of deep we to other something of So that done Alan be And that of level sort in. I maybe with sort third think if do at it and brought to we the assets of just question. then licensing answer were opportunities we've intelligence his diligence

Alan Musso

I million pociredir Yes, funding next you we those preclinical million. and $XX of addressed funding and to seeing be the forward. think Alex, maximizing expect some spend that advance Greg, of guided, does programs our there full as anticipate it. opportunity And and to year's we $XX

we feel position cash good like in at we're shape So with be a time. our to this really fortunate

Operator

next And Joseph from with Schwartz comes our Leerink. question

Unknown Analyst

dialing for Park Drewry I'm in Joe.

call, these what you onboarding. latest XX there more you were on previous call. on U.S. sites were to you X that and EHA, you a That's on thoughts curious plan I of and today's sites up to at ex I map believe with in you've believe said separately, summer was sites which to our activate And I presented what this XX were activate sites. So U.S. mentioned your the

them. if on curious was onboarding you process identified I and the you've X remaining are where the sites for So

Alexander Sapir

start and provide to detail. bit more it Pat over little I'll question, turn great a to a I'll and it's then Yes,

are you yes, So correct.

in And the we're in pleased right some as in again, with the sites some I enrollment. say of seeing We sites, insights -- U.S. of both that that place more early would we activated here as of well activated. recently have XX now. terms that absolutely ex are the are patient the the sites U.S.

ramp but of you and We -- investigator represented so to maybe, expect a Our still that Pat, will enrolled and the past had several XX Dallas, an onboarded. to to more year. do goal XX have sites at when at of many a the existing by sites terms also just want is the Friday, this were in this were I up end meeting both we Pat sites we give little be But those activated sites sites to new months. in specificity in of would coming additional bit

Iain Fraser

Yes.

So we are recruiting we are -- the we actively made your and The that identified to sites will and successful of activate some are majority we working are on but to in there the those. all those believe working Africa activation be have Pioneer. have in we U.S., question, in of

pretty We exact continue but -- sites. and for much the visits the since EHA next many we more couple have the don't and the activation planned the we number, initiation remaining know months, have activated in site we I meeting of had

will to like enroll be sites continue and to I sufficient We Pioneer. -- think we said, these

We continue pociredir to hemoglobin for inducers. engage has an because new there in interest sites and app and increased investigators been studies partly future new

with So if for their possibility some and studies. join quick Pioneer. activation more we also of could But predominantly could it's and be there's enough, new continue future help with sites, them that a to engage

Operator

question comes next And Instructions] our Goldman Corinne Jenkins with Sachs. from [Operator

Corinne Jenkins

patients is you're allowed and the group would be You specific currently higher fully focused this funded. scope risk to trial? apply currently I broader guess does within is to mentioned the on? that program of Or pociredir that a

Alexander Sapir

Yes.

I'll So over Alan and we've specifics Corinne, turn start, of Yes. terms in maybe for then to I'll what funding more allocated. the and it

carcinogenicity population be Pat Phase patient patient a the really So agency, initiation will of had on believe that as at conversation as that work, -- saw this the the really to our we benefits the are thinking whole impetus XX of we're we're additional for the we about of we there we've for hemoglobin I was the as targeting. for data program risk to some there were the Because benefit. patients. that also able fetal some in risk I we able the patients, that of reason from that to impressive beyond we've the we the currently said agency, end obviously, mentioned, look that preclinical in levels show in population the was the based the That future about severe think the these hold. was once first expand to to more And of prior have talked the XX with a that with past, everything the But they're seen.

the the the we where population of around are terms beyond like. Alan, what then, patient additional agency entire X dosing about in is expanding specifics our have then data at get enrolled, XX patient their that funding months a looks and complete and So goal And patients severe the this with approach today. more take population them looks like what study of the to look these that and

Alan Musso

Yes, Corinne.

capital and sort forecast then the we expect beyond toll and will but and towards go budgeting program development where that not in as encompasses plans of next only are to year process the that. taking So the allocating

going guidance of forward. which So for that's we anticipation is pociredir development that the sort complete give,

Operator

further you no does This this show conclude today's conference much so I participating. time. call. Thank at questions for

disconnect. may You now

Fulcrum Therapeutics (FULC) 13 Nov 242024 Q3 Earnings call transcript