FULC - Earnings call transcript Q1 2020 | Fulcrum Therapeutics, Inc.

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Operator

Good morning and welcome to the Fulcrum Therapeutics First Quarter 2020 Conference Call. Currently, all participants are in listen-only mode. There will be a question-and-answer session at the end of this call.

I would now like to turn the call over to Christi Waarich, Director of Investor Relations and Corporate Communications at Fulcrum. Please proceed.

Christi Waarich

[Demetria]. you Thank welcome discuss and recent Fulcrum conference Therapeutics highlights. Good first to quarter to our and XXXX call morning the financial corporate results Earlier our release a today progress. outlining we issued recent press

who those the in section don't For relations you have copy access investor at you website our a of it can fulcrumtx.com. of

include within future Act that the Private of and remarks reminded call of be development financial meaning These during this timelines, our may projections. Litigation the Reform statements contain plans, clinical Please made forward expectations XXXX. may looking about statements and Securities

an as While upon these forward-looking our of to in update statements refer of Securities obligation with today, the certain our future, representing in Please so. should represent with most the relied our but be associated Exchange uncertainties statements filings our on Commission a to We are may and views these do business. not risks not for views discussion future. they as the we recent taking and

With Owen Executive Development; Officer; Operating me Senior President and Officer; Clinical Cadavid, of are Chief call Officer. Vice President Chief Bryan on Stuart, Diego Scientific and Chief Gould, Wallace, Robert today's

for will will quickly our our will of this through me agenda. the cover opening sickle cell will Q&A. morning's our call overview efforts discuss Let with Brian call financials FSHD research an our Robert and Diego run progress. Owen recent begin discuss program. and the program before

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

Robert Gould

caregivers courage compassion everyone many to COVID-XX. thank healthcare us I during continue to today. challenges Christi. their support the Good joining and of morning and investigators the first Thank thank and they so for you You, as workers, want for

defined we our develop remain to everyone mission impacted root their you genetically who purpose hope Fulcrum's diseases and communities to go friends, are unchanged serve has we therapeutics hearts of work discover as been Our safe and cause. and by healthy. keeping the addressing colleagues and patient all treat out to to we

COVID-XX proud like recent I'm begin to highlighting risen the by pandemic. I'd employees challenges of accomplishments. our our how to of of have updates and the evolving some

ReDUXX go Today trial amendment or will provide the robust the in our detail. in phase we extend we dystrophy patients amendment the changes and while the addressing FSHD. presented more an will facioscapulohumeral treatment with losmapimod more over believe Diego muscular patient by trial original will announced with to COVID-XX. a data set from design challenges IIb These

to losmapimod we the the FSHD. debilitating orphan losmapimod I'm designation chronically FSHD. Like is for received diseases. pleased we treatment Early or the have U.S. the also orphan European or Food received in to drug the in report from Administration Commission U.S. Drug orphan from of are that for diagnosis that quarter prevention of and for European designation life-threatening intended for by in Commission treatment, the the granted drugs rare designation

extremely have received further are to advancement pleased losmapimod’s supporting FSHD We the program. this designation

meeting. trial engagement a We data trial session skeletal association muscular the virtual recently from X of clinical losmapimod dose dependent phase our during target presented muscle dystrophy with in

induce make FTX-XXXX, fetal small select oral to to with progress of therapeutic continued designed We hemoglobinopathies. an in expression molecule hemoglobin

hear call. the Owen You'll program our later sickle-cell about from in

and product range on building early development activities diseases of our activities drug research to FulcrumSeek, candidates research programs out to our clinical progress continue with collaboration we also in stage genetically and proprietary under identify broad our initiated defined We make including designed Acceleron. targets, engine a

key on like the Diego for turn FSHD an the over update I'd now call Diego? program. to to

Diego Cadavid

Robert. Thanks

approved clinical industry-sponsored is patients XX,XXX trial expression FSHD diseases and replaced known by of similarly the drugs characterized by we gene, to can are potential there fat. of lack currently worldwide. characterized the is evaluating the treatment. occurs the approximately DUXX the incidents and a Unlike As that gene progressive FSHD muscle a reminder muscular by other a the root of are FSHD cause disease. There aberrant estimate are U.S. be disease characterized as in skeletal XX,XXX advancing only the no by that a severe degeneration for We is

at losmapimod inhibitor MAP studies. DUXX novel for patients. discovered of a represents therefore in the reduced We expression Fulcrum affiliate therapeutic preclinical option that efficacy selected losmapimod potential We believe kinase a

even an of as Our that presentation and FSHD to patients. own has reduction Those benefit to DUXX beneficial expression suggests benefit. any is be completely independent not patients. a the evidence who evidence that gene DUXX as potential turn as that have independent spectrum we do that for DUXX incremental to There suggests in well researchers provide to reduction believe may to expression

from double placebo originally expression blind in exceeded driven baseline genetically of is of patients the the affected of IIb had FSHD. enrollment We trial at change international trial The endpoint gene the Phase patients DUXX confirmed planned. losmapimod with end which XX the we is skeletal in in controlled ReDUXX our muscle. XX primary February completed

healthcare the safety programs. the integrity data completed our providers the are we steps. in as paramount. and trial the next dedicated participants enrollment highest clinical and by and clinical patient our of X remains trial is in phase been safety and clinician we planning considering single COVID-XX Fulcrum also site label research We impacted execution standards has open of in well The maintaining which our of collect their to as

a In the our followed week and the the our COVID-XX, treatment plans at design wake clinical XX limit pretreatment of implemented postponed of second the to of program. we of The to included trial biopsy period. XX activities disruption quickly trial original a ReDUXX potential FSHD biopsy week related sites by number a

patients weeks trial, pandemic label week label the open XX completed all opportunity Prior had in the the week biopsy their into open XX extension. an extension. to to XX COVID-XX patients the of the the enroll including Following XX treatment of enrolled and XX

trial subjects XX XX week roll biopsy a XX week XX the at This completing As from XX the COVID-XX protocol under label collaboration the the continued to amended the with the allows open XX in training receive to pandemic weeks. either period our at week approximately extended and ReDUXX in continuing or the team investigators into extension. trial after our currently

To has at from extension XX summarize, XX approximately an completed still and in open their ReDUXX trial receive label biopsy extension extended will while weeks extension. enrolled roll XX trial a with been have muscle to follow. label XX XX having weeks. to open into the XX or the either apply to already Patients will This patients the

trial an signature and we quarter of third interim anticipate We gene top-line primary year their to data of these quarter conduct As to report modification the subjects driven the XX-week this have data DUXX subjects from part interim in in who of of biopsy. will expression the completed endpoint we the the XX of XXXX. approximately also analysis first expect analysis on sharing

muscle disease. we're extension the reachable is as the in the The we the clinical work and well function losmapimod believe root assessment is population a know and design may there in benefit potential stable also from time cause as patients, skeletal a go, signature and to longer the measures controlled independent greater gene times From XX we able the our this XX also have longer preparatory that over up secondary outcomes. own of studies, treat and patient allows placebo the such on to exploratory the researchers of weeks space, various endpoint FSHD muscle MRI for as both points end reported

address to strongly to important community best as opportunity We want study interest are by the we effort believe these to best of this in patient provide ReDUXX presented COVID-XX. the development changes the the the and advance challenges

continue to changes and with of will in All essential and are to support these collect designed enable investigators us to participation continued dialogue to allow data ReDUXX patients regulators.

to Owen. now I'll call the turn over

Owen?

Owen Wallace

potential of we have the indications progress these treatments address where by we to these the we therapies across and challenges efficacy we more we the aim and diseases increase Thanks research Diego. rebalance effective the root posed treated. indications spite can develop COVID-XX, of way our molecule expression. to and believe continued safe broadly being In causes consistently portfolio. At various to early our small on targeted clinical Fulcrum transform gene disease of work In are make to pursue

continue we business lab obvious operations spaces. a limited essential more an As on

the as internal we with our portfolio. continue results advance well collaboration a As Acceleron as to

features advanced small FulcrumSeek, targets clinical also of generated CRISPR scale designed cellular genetically diseases. defined product combining to machine and the monitoring molecular identify sequencing candidates and large imaging proprietary work have programs and drug engine perturbation a By broad RNA are learning, range by more We high-throughput XX,XXX cellular our a probe molecule on libraries. development than and we data in

the with disease. cause strategy, function understanding our only fundamental genetic gene effects on is strategy it the disease. the their modulate Understanding in of expression of to human to identification our how of target group provides altered FulcrumSeek also is us not therapeutic core cellular is unique

like to advance to thank programs, working into crisis have who work our and through coming lab, our guidelines. COVID-XX distancing enhance who research under to health the especially are I'd employees social safety and those continued the diligently

sickle-cell regulation Likewise could advance compensate patients, to and hemoglobin can selective potent mutated By that a filing, our beneficial hemoglobin approach to IND develop be of towards in has has the and therapy deliver levels thalassemia. increasing the disease fetal we on the for sickle-cell our for hemoglobinopathies and believe patients. of program beta continued HbF for focused up which effective both we

have genetics trials. and been where be future increasing to pharmacology surrogate be improved is and in associated endpoint of strategy human This a prognosis shown that therapeutic outcomes levels HbF supported with data HbF may suggesting by clinical

We're progress. with very our pleased recent

thorough humans FTX-XXXX disease vitro seen be research the of accepted had we've at candidate year. concentrations that oral sickle-cell in September broadly educational models robust sickle-cell an disease HbF on symposium clinical and in and scheduled the to the achieved has will in pharmacokinetic believe preclinical We've we of profiling Our for drug vivo at readily been in of profiled abstract presentation and elevation compound. of XXth annual this based

enabling We the trial disease in XXXX the of by the our application and of IND FTX-XXXX. non-provisional have we've IND filed patent the submit year. phase completed studies second We our toxicology and work end our initiate to half sickle-cell with in and plan also X

I that our now the for call will an turn update for on Bryan to With results quarter. the over Bryan? financial

Bryan Stuart

of plan in XXXX discovery will marketable stage our Based FSHD cash, and into operations We're efforts. continuing is with million transformative hemoglobinopathies urgency these quarter $XX.X sense patients a these and In committed such of therapies with Fulcrum first Owen. a on to difference of bring our FTX-XXXX and potentially for clinic operating believe unprecedented this us We losmapimod the quarter third allowing to to making securities. to disease. bring we XXXX FSHD advance to entered the Thanks times while the great projections and cash our into in as sickle-cell proceeding with current equivalents invest patients. select support

development million of and for cost million $XX.X with support compared $X.X associated [rights] quarter Research March costs to to Included one-time development losmapimod costs one-time GSK and the with the in that issuance $XX.X advancement the was these million as the agreement XXXX license of losmapimod, to personnel series company's organization. ended Fulcrum's million of FSHD primarily to of quarter the the were well was expenses XXXX. the of due the for $XX.X as in for cost first treatment the increase stock growth XX, excluding research related increased of preferred under of convertible related B $XX.X million increased

primarily of General XXXX. as company. to of increased our compared as the first million $X.X quarter of to XXXX growth operating This were cost $X.X for quarter with public as related is the million a and for to expenses associated increase organization personnel as increase well cost support the first due administrative

expect upcoming remain Overall in several to we our and undeterred catalysts. continue mission

interim from in year. report We of the will quarter analysis third this the ReDUXX

the We continue to in we progress months will on and will ahead. about look and programs you trial the Acceleron. partners to continue progress drug year our with product to X ahead sickle-cell end by our and our initiate the of making plans disclose work discovery the excited updated advance with forward on at disease from as the execute our target continue in our biochemical keeping the FTX-XXXX phase while engine We're we as we we

Operator line the you for may now questions. open

Christi Waarich

Operator ready we are for Operator? now questions.

Operator

Morgan with [Operator Instructions] Harrison Our Matthew first question comes from Stanley.

may proceed. You

Unidentified Analyst

for my This you Thank taking from our [indiscernible] A couple questions for is Matthew. side. of questions.

have expect XX any think the whether enough have only is Do you power will that to directional or a expect to one guys improvement there you first given to lose not? analysis. you see will subjects signal interim you to or in first the only show data an power you you be The

Diego Cadavid

trial. help XX subjects phase believe This to at the end some at opportunity sample early an the question. give insights size Cadavid. subjects us the and has -- we and data we for of will prepare power into the is us it of is you appropriate initial Thank planning. The Yes. XX a look three Diego answer the question make to believe

Unidentified Analyst

subjects Thank the you recruit follow-up Will additional the point? believe at subjects you or and need all Okay. you need to you question. have this you

A - Diego Cadavid

We the we needed. We subjects have we recruitment. have completed all believe

Unidentified Analyst

Okay. wave second part, wondering of of again? in subjects this and there whether the of subjects you. there second might when have second wave is XX of additional, can infections. have given Thank whether XX or are And expect at to risk then finally, be a was complete you mitigate biopsy the you I you all the that all are some at actions How losing a XX certain pandemic any a the patients second the you weeks biopsy to taking weeks there

Diego Cadavid

amended When to. carefully exactly we we referring consider Yes. protocol you're the what

So windows, have we and the flexibility around build XX windows some week well time patients. sites as as

So right data week or the at we anticipated at regardless. now we week either XX will get that XX

Unidentified Analyst

much. you very Thank Okay.

Operator

question comes from SVB with Leerink. And our Schwartz next Joseph

proceed. You may

Joseph Schwartz

it ReDUX year evaluated about question clinical still second you duration at the hitting Will and in can be at different the ReDUXX weeks on hitting time the Have arrived where doubling the evolve with week in point? this this are to would seems there patients given XX then its a interest an in like to is of how in you when would be point this with The relax and pandemic how talk you patients that you endpoints? consider patients done respect time many half when for your these be, XX social enrolling waves this analysis best most distancing might however respect protocol patients? based might to

Diego Cadavid

across a we XX last by about weeks an for over in months of visits are between six data enrollment what what to the period decided a to because happening will patients the even completed COVID-XX, six we collect extend if August intermittently. all happen trial Therefore study and clinics is across based much to the patients to ReDUXX of give we reopen moving and Yes. with longer year. February of this where believe expect this year additional this We months. on flexibility expect the period

if would are are as even patients events. and come you slowly that some back believe we is basics know overall a later disease, So XX acute FSHD week missed, progressive we not counting

we as site. So time to as we able believe sites be answer impacts pandemic long questions. data are is The efficacy affecting still the open. we'll of the the not every are many collecting over Especially

Joseph Schwartz

are strike able size in now? for have take just might any And seem to protocol the Can you additional in we warmer bring in to are performed some in and is bit being you additional speak it little we while you XX without that or patients that an a adjustments entering speak the talk to making and bring period this XX to as evaluation? ability be iron assessments clinical require patients [helps]? about, you just weeks is at alpha you Could

Diego Cadavid

Yes. build flexibility. These amendment

So all open, are original over extended which provides create at and with the weeks receipts, sites windows. the of XX are opportunities basics the additional protocol coming we are amendment open, patients

to very trial. have what opportunities committed regardless the retention the fortunate capture subject give that's as of So but very aim with only patients high we reflected sites COVID-XX. on happens the are We're really the in data we and not much that

Joseph Schwartz

to suggests any it's It that open sounds next you the you're date very COVID-XX to from evaluating steps been have heard from label the and I you you able been Thank helpful. like That's impacted insights there. trial? and garner then

front? why impacted ReDUXX. So you has Can that sounds provide been more that any than it on color

A - Diego Cadavid

Yes. site. single study a The open-label is

where began learning happens So always look inform trial impact this have has so to is when have you That obviously this in we that site have have goal ReDUXX in don't that trial, believe for, always this that which sense be be helpful. that one heavily therefore of the opportunity we and in will region the we affected if valuable Netherlands, August had the is we course larger. have site in learnings sites we what so to we really] from has been a been trial trial the considered [don’t many

Joseph Schwartz

That's the color. for helpful. Thanks

Operator

And our Tazeen of next America. question Ahmad comes from with Bank

You may proceed.

Tazeen Ahmad

just to you're I powering clarify assumption. wanted

reduction to a that XX. at said powered So be week XX% study DUXX the show you previously of would

Just based and the accelerated about with to about spoken you particular the path how affect approval on does you're changes FDA that [rate] potential the have that item? talking

Robert Gould

is Robert. this Tazeen, Hi

Just don't speak of made. That was XX% original one did we Diego that reduction assumptions power will but believe in I powering -- a let I the we for the slight study to correction on assumption not ReDUXX. the

Diego Cadavid

Yes, Robert, that is correct.

biopsy XX disclosed what the because and on can impacting of The we is assumptions. Therefore be size own power. This expect power. based has some only don't amendment. subjects the flexibility the muscle sample amendment at this week nothing We their have changed about same. adds never loss is week assumptions the it the treatment XX or for not a really are power

Tazeen Ahmad

the buffer time this how Do you and of the study expensive study in in far your this you potential year. into study does to for so are increasing of with are you will What a and account compliance have effectively you this length if taking seeing observation? rates Okay any for increase? the dropouts

Robert Gould

This Tazeen. Yes. is Thanks Robert again.

it's know an taking One struck cooperation losmapimod the really tablets. as X.X is remind just of to daily take the of patients milligram oral with willingness their as and you place the we've that been you things to drug

the a we rate two of evening the high in we but we the the tablets, we and patient believe compliance high increased so, as only the original XX had original and not because had, So we and retention tablets, patients from enrolling The was trial well. the opportunity patients. and patients actually just to XX the two response of community morning had in that

all with time that that we point are So even we're be patients the do of XX patients at believe the the able study we're going them if not change retain have in patients still original able going to to things to to study. in were be this of but currently the in the currently that if to most are we believe

Tazeen Ahmad

Okay. biopsy with between weeks taking how come a the XX. or and you the last the at feel How big in both gap question you about integrity My in at did do XX because of readings is it's time periods up two? XX the confident

A Cadavid - Diego

Yes. Diego. is This

have preparatory Wellstone and that our we weeks variability done apart group done their So with had signature at own and shared data and The of year was a a their over stability look history collaboration natural all very academic us. done DUXX to and the the they it the and were about had gene study apart. of X-X generous

two at we from this very population that these studies stable. signature is know the So level DUXX

between apart within as of interval any argument weeks gives basically as don't year or a that eight it, value we this biopsies repeated greater So loss long us ability signature. impact interval, or on that a collect expect we good

able a building assuming XX there decrease of and to in these that really moves biopsy sites be have pandemic we we'll patients reopen flexibility into [feeds] for and patients the will who had the over not time bring It's weeks terms and as be obtained of window. size a but the biopsies. obtain XX So XX-week came

and and patients power the we collecting to and chance be the believe endpoint sites flexible So the that best can keeping quality. efficacy losing without is

Operator

Sandler. comes And from Instructions] [Operator question next Tenthoff] Piper with [Edward our

You may proceed.

Unidentified Analyst

step this time year that make next this the would how, would what update. wondering next I'm it's and may that this changes but start and for point now for ahead? you Well, you something a study at data what set to and envisioning be thank early still data would potential registrational obviously additional be with some and

Bryan Stuart

Yes, Ed.

trial to So first that our doing insight the it's into might I look what gain interim think data. design a in all analysis, like. order of XX-week three phase We're

can So that earlier. planning begin we

confirmatory a can As can that you you we having really that? to in had top-line would know of top-line that trial of a informed begin process. third data again week and the more projected quarter begin do add point process and so little remind on planning we're any was doing. kind think detail originally conditional be that we interim data analysis approval XX data anyway want you process that a available the Diego And that require we planning by to the this planning

Diego Cadavid

No.

I this think placebo a having as if more study can said and longer make duration treatment informative. anything we only of better against

Unidentified Analyst

Yes. Excellent. Makes sense.

Thanks forward the interim update. for to Looking time. so the much

Operator

I Gould Q&A to would Ladies and concludes today's any remarks. Robert like now to call gentlemen this now closing of portion turn for over conference. the

Robert Gould

for interest to healthy we want all remain your and I thank support again joining your that you to Fulcrum. today. and us your time today families and you and safe are for thanks hoping all and for Please note

Operator

gentlemen today's and you the conclude Ladies program. does conference. for thank This attending

You may day. all have Everyone great a disconnect.

Fulcrum Therapeutics (FULC) 13 May 202020 Q1 Earnings call transcript