Earnings call transcript Q2 2023

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Operator

Good morning and welcome to the Fulcrum Therapeutics Second Quarter 2023 Financial Results and Business Update Conference Call. Currently all participants are in a listen-only mode. This call is being webcast live on the Investor Section of Fulcrum’s website at www.fulcrumtx.com and is being recorded.

Please be reminded that remarks made during this call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These may include statements about the company's future expectations and plans, including the clinical hold on FTX-6058, clinical development time lines and financial projections.

represent relied as company's upon the statements should today, views forward-looking future. be not as these Fulcrum's of view this in While representing the taking the update but statements may obligation in Fulcrum is do these not on so. an to future,

Fulcrum's company's to with filings business. for recent Exchange and uncertainties the certain discussion of most risks and associated Commission refer Please Securities a with the

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

Officer; and Iain our Joining your Medical Greg Alex answer be questions. to Fraser, today Officer. in Accounting and Dr. the key Q&A be Alex, which will on Tourangeau, on Interim Greg After call are there a updates Principal brief available will Iain Fulcrum's Chief providing programs,

to ahead. With pleasure is that, it my Please over Alex. to call the go turn

Alexander Sapir

you at and time commitment the build time honor genetic our our for on of to Thank thanks taking work towards on us rare It's opportunity of improve patients pipeline the this foundation company's Fulcrum have advancing and strong all to to truly delivering for today. lives operator, lead the diseases. important with me an as join we to to you, and to

on over questions. morning turn open losmapimod FSHD like provide then And after couple what do for So disease. that, brief we'll key cell and and After FTX-XXXX to Greg, short Greg of it up updates it for I'll for a two this financial to a provide update Facioscapulohumeral for I'd Muscular or corporate our programs, is to Dystrophy highlights. for sickle

advanced with So losmapimod, let's start most program. our

for of patient the dystrophy of currently inhibitor alpha/beta pXX FSHD, with an a selective in in form a kinase muscular losmapimod, reminder, of quick Phase XX,XXX development X treatment a is the U.S. population estimated As MAP

I activities putting take function listing and years, coffee, life a cup like over Now accumulating of characterized in of to inability daily relentless and resulting activities that the many perform dishes away the by muscle for loss you granted. FSHD is or

disease. the more approved is that find are none. this us These these had drove these fact have patients that to for the sobering on even that patients factors journey no for options embark Now, their treatment options to

for So, initiated call in study. we X REACH the trial June of which our losmapimod, Phase we XXXX,

extremity Let of It's be endpoint primary the motion the intended baseline change the reachable registration background shoulder bit range motion from quantitative workspace function specifically RWS, and is me The give XX-week and sensor upper technology. geographies. measure U.S. or XD study a trial using in REACH. mobility both a that in a evaluate on of enabling ex-U.S. to and in of for arm

independence function perform Now of daily preserving maintain their about these some patients earlier. living their talked to to this upper of extremity these and for I critical activities is that ability

prepare at really our like for we be marker quality pathology of strategy some we'll that self-supported thinking a questionnaires health looking of fat will life utilization is or launch. payer as measures As which MFI, also muscle disease important of our as and as care study, endpoints commercial help secondary part infiltration this other on an inform important well

of REACH this with screening report quarter. this we line plan being I'm a quarter excited complete study, to study in to to in XXXX. share enrollment closed, later the data that expect we And fourth now the really XX-week and has top

first-ever brings us one for us delivering For closer therapy for patients with more importantly, this FDA-approved FSHD. the step and to the patients,

disease. with sickle XXXX Let treatment cell of HbF on me the is for XXXX. to inducer now patients our oral potential move

for well the as paused Phase nonclinical April, in XXXX. hold that provided evidence inhibitors. a the previously of data of letter preclinical stated October was we point, Xb that submitted to XXXX PRCX enrollment the with initial suspended As we and on observed hematological malignancies immediately XXth other that by February FDA other they trial announced, clinical In and from and hold received at as and dosing in clinical related FDA, feedback December

order FDA has I it's restarting study that prerequisite think potential the where with define risk. study, benefit at data this stage, important further mention agency that to in of do to Phase continued requested additional In clinical as not a the Xb patient population preclinical restart requested the Fulcrum outweighs Phase any for patients. Xb the treatment XXXX to potential the us has or the

therapeutic of believe clinical interactions in risk XXXX our to and with we to benefit demonstrated favorable forward work as differentiated towards we to and believe as disease obtained sickle we cell prior data collaborative, potential look oral in and an has We productive potential clinical possible. the clinical hemoglobin lead interactions the hold HbF resolving dose-dependent that has to the to Overall, living continuing disease profile. date patients inducer provide as that a option a generated as a HbF been to data Based preliminary severity. with the clinical has that FDA preclinical have hold, and that the on magnitude improvement demonstrate a fetal we or XXXX increases total for quickly our meaningful

and we provide will specifics regarding with patient update narrowly-defined have more we clarity once on agreement I FDA. population have the intend once to I regulatory more this forward, an path provide

updates on programs. the our two covers key clinical that So

Greg, important let it other turning me to topics. give over a quick update two Before on

X,XXX program for terms year, license for investigational communities, of DBA, global July DBA we we potential remain delivering to an obtained the disorder underserved treatment to the compounds of estimated research for Fulcrum in research worldwide. groundbreaking acquire exclusive intellectual from in arising property CAMPX blood a or congenital that affects individuals therapies Therapeutics preclinical this rare from an agreement, As Under Anemia committed oral Diamond-Blackfan of CAMPX's will short. this

a broad opportunity strengthens Our agreement on has pipeline, and unique work, our are growth. believe further to we applications expand preclinical CAMPX's and discovery we excited with CAMPX for foundational which potential

I team appointment the to announce priorities, Musso, Additionally, X. of my is pleased to August am our Financial Officer Alan Chief position leadership of solidifying one the and key of effective

knowledge have company his experience financial his a time enters leadership that firsthand I development. strategic and Alan biopharma our of character. its and his decisions as face importantly, complex now acumen, of on a of have industry size, known the within of aboard, company range the insights His be for financial Alan. thus Welcome most some keen stage next will invaluable

on me Greg? turn over with Greg financials. to an our And it give update so that, let to

Gregory Tourangeau

June as XX, of $XXX.X cash, XXXX. securities marketable equivalents of Thanks, as XXXX, million Alex. We ended to million December cash XX, with compared and $XXX.X

our marketable of was securities to equivalents position, mid-XXXX. into second the of operating operate and expect for fund clinical cash, cash and second to XXXX. for quarter our This continue timely projection We million from the the XXXX hold. a strong a compared $X.X million revenue quarter of financial as to FTX-XXXX Collaboration resolution we expenses $X.X assumes

a million well during of to achieved REACH $XX.X development quarter of $X hold and XXXX million of of million was $XX for primarily attributable milestone the the to $X.X as costs due FTX-XXXX. decreased second of compared clinical GSK that the as upon of the a result as The quarter expenses to quarter for were XXXX Research XXXX. the as decrease we second million initiation for second

XXXX. due the of professional for of XXXX. the $X.X the costs. expenses decreased primarily The for $XX.X were second XXXX quarter compared as the of second General was million Net $XX.X of for decrease quarter $XX.X $XX.X to second and quarter was million to as loss million million to quarter of administrative for services second million compared XXXX

let me that, turn And with over it Alex. to back

Alexander Sapir

it open That's that ahead great. for overview, Thanks, And Greg. let's and up with go questions. operator,

Operator

stand roster. Instructions] while [Operator by compile our we Please Q&A

first Stifel. question Dae Ha Gon Our from comes with

open. Your is line

Benazir Ali

you okay? This Benazir is hear for Gon. on Dae me Can

Alexander Sapir

you. We cannot hear

Benazir Ali

Hello?

Alexander Sapir

ahead Go again. Yes.

Benazir Ali

Dae Benazir for on hear Gon. Hello, you this Can is me?

Alexander Sapir

let's and just come the to then on Yes. question, next move difficult maybe them. It's hear we and his Operator, to Gon colleagues back Dae too to can there.

Operator

moment next for Schwartz please. question The our next from with one Joseph question Partners. comes Leerink Okay,

Joseph Schwartz

Can hear okay? you me

Alexander Sapir

Yes, Thanks, Joe. and loud clear.

Joseph Schwartz

taking comment not FDA, could hold, a was given for has in doing regarding the Congrats for on hoping I on order my heard the off you I Thanks Does a are bit progress. preclinical question. asked you help Can the data more get off the of great. a solely to FDA preclinical that on to the you to can key any of expand bit lies a just benefit getting more -- more? patient that really population little define where explain studies. just relationship. suggest risk concept that some us there's ability hold non-tox you attractive an Okay, this

Alexander Sapir

thanks and a asking I'll off I'll turn Joe, for start more Yes, to provide over to and Iain insight. question. it the bit absolutely, then

what additional, into study absolutely would right. is restarting or preclinical volunteers. requested What our before they requested some have moving not you're The additional data FDA clinical as, to define prior data Xb has any patients. in Phase So pharmacology healthy I

that can go sort me little broader he just overview, into turn of and it detail. a let bit to more with So Iain, over

Iain Fraser

Yes. Thanks, Alex.

I the the around around as Xb important patient request population. the and two patients sickle make think study, the Alex of the one two said, has between it's hold with that is disease aspects The the just of clinical cell differentiation appropriate the in there defining program. to the aspects and

of around there's healthy in this that's of number done development is already, in we've still getting initial but program. them work back volunteers. piece drug clinical additional the second at XX, healthy a evaluations, studied into The stage over our traditionally Obviously, volunteers

associated there's a preclinical pharmacology a after to healthy target reassurance a to of giving of doses evaluate that that, And engagement is to not provide doses study as long-term of short number order in part alterations. the number volunteers any with small

specifically is work Xb. development it's for as getting the the and priority it's of the our ongoing, nice for we've an part very have around to healthy that's much population. that's So population back the said the population patient volunteer is the volunteer but as into program, but not before, drug healthy requirement a absolute And

Alexander Sapir

Next question operator?

Operator

by question. The Tenthoff with Piper question next Please the Edward Sandler. stand from comes for next

Your open. is line

Edward Tenthoff

if you. I thank questions, just may. Great, two And

Firstly, welcome aboard, Alan.

REACH next sickle likely, can on get report anything I with to patients? the done follow trial there executing late true? specific you do And just to define back and you what And be to you'll up. so quickly wanted losmapimod first into than data other So follow-up Is is be cell back And Thanks. the respect with volunteers healthy you go year? that the long-term needs then that how there? just most into a could think would to question,

Alexander Sapir

great. it's Yes,

happy Let there, take then Thanks Ed. question it for the Iain both. asking first me over for the to turn question, second to I'm

Iain Fraser

way typically are that Thanks, done healthy healthy those at of studies the volunteer supportive The or any which like Yes. are study, a [indiscernible] And getting again, type clinic. dose be stage linked single required. in study, the ADME crossover is that program, terms the in for radiolabeled typically switch are back a are Alex. single-dose things formulation into study to overall not study clear, this volunteers a and not they're a

There's back And so again, the patients. that is healthy any our initiated first. the get be into priority into It's requirement then there'd back not a volunteer getting and to studies patients there proceed if population, back types patient in if can healthy healthy needed. and parallel volunteer in studies done are necessary studies any of in the issues those will as volunteers, getting be the into

requirement. a not it's So

Edward Tenthoff

thanks. Helpful clarification,

Alexander Sapir

great. That's is then, Alex. and Ed this

that terms We of to a comments. very later This in we quarter. close sometime quickly. had earlier your enrollment In that expect would yes, question, screening has delighted has study We're second as closed. our this announced is enrolled

we to the between safety. of across the two we as primary are patient will until That essentially add line release basically that, you see during improvement the all -- that providing would key XX primary as weeks of study. we and secondary in disposition, to the endpoints, report expect provide be see you be the And out would that see similar to what balance you expect endpoints. We'll well sometime the those which would fourth release is -- saw year duration the out in we'll top to secondary arms. if study So put quarter would the what endpoint line us is next top ReDUXX of on would and

looking we're So that day. forward to certainly

Edward Tenthoff

so very you Thank much. Great.

Alexander Sapir

Thanks, Ed.

Operator

next Dae from with comes Please next for stand question. Ha The Stifel. question by the Gon

Benazir Ali

is This guys Can on me you Benazir now? Gon. hear for Dae

Alexander Sapir

Yes, much better. you Thank so much.

Benazir Ali

Great.

workspace, there what been DUXX So with And one occurs and DUXX on how FDA like like but REACH the FSHD a the interpret if benefit data DUXX in biomarker in regarding in with questions feedback was, they a have reachable discussions minimal effect of well? noteworthy score, as additional any EMA about composite would as a our that?

Alexander Sapir

Yes. Great question.

think I to let me that to that, turn over Iain. answer

Iain Fraser

biomarker. as defined REACH regulators. being And not endpoint the evaluated has the said, and reachable been workspace is on of the biomarker discussion all. for with the is in topic REACH Yes. a That not primary obviously has which I for that, not not topic the The of a endpoints study at The focus the as discussion. is study, the been

Operator

comes The Biegler with OPCO. question Matthew next from

Matthew Hagood

This inherent what outcomes. about in I the to on a meaningful line study for FSHD, wanted enough gives you Matt. is confidence to FSHD Matt make variability impact will you one-year given Hagood that, that just talk the long on ask, in be could time

Alexander Sapir

Yes. Iain?

Iain Fraser

Yes. Thanks, Matt.

So you're correct heterogeneity there is FSHD. in that

that I from study on a which in pretty between in the difference encouraged study. think reachable nominally We're XX much statistically well by the significant described. data that's patients ReDUXX losmapimod X very did Phase placebo workspace, the and show

patients of in study. the study REACH encouraged think XXX observed effect as we're that in study on the based a the ReDUXX powering the Phase seen in I we So is move X number that into and larger by

that's with samples, then respect and secondary which it across MRI-based endpoints the endpoint, at muscles primary. the any heterogeneity a muscles each different the accounts given across the And patient broadly to is because MRI is patient. least the assessment for of in study REACH one So an in whole-body

in helpful reachable on evaluation. it's the that's more integrated think upper which purely It's extremity, does. focused I a that So workspace not the

Matthew Hagood

guys. Thanks

Iain Fraser

Thank you.

Operator

Suisse. next The with from Frommer Credit question Instructions] [Operator Judah is

Judah Frommer

those the next hear just the cell. can same and finer would related we would on hear a on be Congrats I next if come expect would in time progress. what be to come would to Just volunteers? goes guess all the And update planned, put we the if point to we as related update trying healthy out to what updates would would at figure first? patients? Maybe sickle the which

Alexander Sapir

Yes. asking. Great question, Judah. Thanks for

hold, set on to of then specifically sort it a I'll our you the come and me into expect bit questions thoughts the get stage more clinical little over to posed. we in Let would of when to turn that terms off Iain

So hold of beginning came when order minimum think to it the February, at March in we would it that least resolution at out first of to with take I a around six the end months the FDA. guided reach was had that of

the So that sort frame. of time until puts us out September

would path resolution the of this sometime next I we in possibly fourth So forward with that the able year. the FDA has be to share even in with of first reached probably expect or the that would quarter the everybody quarter been year

I define with -- I As that the process time. and the to when mentioned, But simply very continue with and FDA, it's process know, FDA there's you conversations collaborative. many well. go would them as takes just a of productive engaging as

that in happy XX-milligram, patients we dose-dependent priorities to is to And with we've the reinitiate XX. continue is belief to see hold. our been patients And obviously, Phase these hold, that then to with again HbF prior of once our study, that key organization increases move off an one then the begin dosing get Ib we'll ideally and XX as the so

premature little to other stage you'd healthy probably data to from we don't have able cohorts Iain, at else I in cohort it's the XX-milligram the the if from when would to either think I share. and But want this you report a guide XX-milligram two anything be study. that out or Ib volunteers Phase bit the know, mentioned I data

Iain Fraser

No.

response and two to that that getting in distinct sickle focus to back are the what to earlier to is the mentioned populations our the be I what healthy of cell we'll quite and back be not we're those the reemphasize achieved study healthy likely study that and primary and when that's patients the the linked, be, for to forward in not in populations, really get required healthy and to is the terms volunteer Just a cell next moving will question with population the they're will agreements they're sickle on It volunteers. until whatever patient likely sequenced. But announced. we've patients announce happens what around volunteer

Judah Frommer

That's helpful. Thank you.

Alexander Sapir

Judah. Thanks

Operator

the over call to CEO, now the remarks. concludes I of Alex call. Alex? turn portion the for will This back question-and-answer closing Fulcrum's

Alexander Sapir

great. Michelle. That's Thanks,

continued encouraged cash ahead. forward We for our executing in priorities on a the until very I the key to and our of clinical many the in programs out half progress first of as would mid-XXXX. by look XXXX the strong two overall, position say with So I'm team, months

patients just the the finally, to each and with, and and therapies one gratitude take defined to our opportunity most Fulcrum me physicians this It's to diseases genetically of bringing closer teammates, us to this clinical treating let finally, And of people studies families. appreciation my root day efforts rare express patients. collective importantly, the and step to and of my advance their fellow the cause work to brings sincere to transformative group we that

thanks fall day. to you look forward for of a So again, at everyone, call investor on the the great morning. Have Thanks. I seeing this many us some of of rest joining upcoming the conferences.

Operator

concludes This call. for participating. Thank conference today's you

may now You disconnect.

Fulcrum Therapeutics (FULC) 3 Aug 232023 Q2 Earnings call transcript