FULC - Earnings call transcript Q1 2021 | Fulcrum Therapeutics, Inc.

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Operator

Good morning, and welcome to the Fulcrum Therapeutics First Quarter 2021 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

operator. you Thank Good morning. Therapeutics financial quarter highlight. results Fulcrum call discuss the to conference and first to XXXX our welcome And recent corporate today copy press who a section of in we those at Relations Earlier you fulcrumtx.com. website of the release, Investor don’t can you for a our access it have issued remarks of this meaning reminded call Private the Please XXXX. of forward-looking Litigation contain that Securities be Act may made during Reform the statements within and financial and These timelines, may plans, our expectations statements include clinical projections. about future development

and While our forward-looking Officer Peter Exchange me views may recent filings of business. our the refer for these on President, President the represent and statements as Moxham, Commission associated taking obligation Judith relied We not Accounting; of in CEO; an today, our are Please Stuart, Research a President, be With uncertainties to and not Dunn, and to future, certain Bryan and our the upon Development. they are Chris with Thomson, Scientific so. Securities Vice but risks Finance with update future. views most and should these statements on as call we representing today's discussion in do Chief

that Bryan run engine and more Chris Peter will will quickly morning's my disease to our corporate product to our it's our With Bryan. cell pleasure call and review over the through me program a this of will call with Let detailed turn begin a agenda. Bryan? overview. provide in the financials. sickle cover

Bryan Stuart

and fast-moving Fulcrum. us and you joining exciting It's Good today. been everyone, Christi. an for morning, thank you Thank quarter for

of engine programs, for like root engine a expect targets FulcrumSeek make initiatives novel our the modulate and multiple important to start the to product progress the which have I'd development discovery and collaborations product diseases. updates backbone allowed to serves FulcrumSeek, and defined as has that identify rare high first our near-term. genetically We that differentiated company. key quality in with cause innovation approach meaningful to across powerful us our is continue for clinical rapidly

programs forward at be in at next I'll provide FSHD. in earlier will mind, from full path Losmapimod an and disease version treatments minutes, data FSHD dystrophy that defined unprecedented grow the or our a month inform By drug FSHD few to to FulcrumSeek relevant insights our and in discovery new select scale months. in of efficiently identified the therapeutics lead FulcrumSeek share Our but muscular settings, cause remain in unparalleled genetically opportunity hemoglobinopathies International these both on presented XX by bring next enabling Research forward were an FSHD will facioscapulohumeral in the diseases. clinical rare will develop root track. our of for on With two and goal The to pipeline this over is more Congress. data INDs that ReDUXX have creates

anticipate data initiating trial we cell we'll end people by with year. to X adult availability, path sickle dosing And FDA the with the discuss in the with to data Following disease Phase mid-year. volunteer’s healthy FTX-XXXX, have we'll in the meet of from registration. living And the

to at at Pfizer. Merck. roles extensive leadership experience held Chris development. and also Medical and multiple Cardurion. Cardurion, at Officer. leadership Morabito drug And as Judith President discovery extensive Chris Chris have Chief have And Dr. Takeda, Chief Dunn, Both and Roche recently as held clinical companies including leadership has Judi Chris development and experience as Dr. the Research and of of of research appointment we roles biotech with at global enhanced team has further in including Judi the in Development. Additionally, we Officer appointment roles Prior our our leadership our announced and Sanofi Medical today, most

of We our to as are of have them thrilled both team. part

turn let's for FSHD. to Now Losmapimod

progressive skeletal XX,XXX leading to replaced currently XX,XXX As XXX,XXX XXX,XXX advancing degeneration a disabling characterized by treatment. estimated industry-sponsored similar the a the U.S. there severe occurs that an There global a therapeutics evaluating patient are patients is to approved disease, FSHD population worldwide is in muscular no approximately rare only by Fulcrum muscle trial to and incidence fat. clinical of and are and FSHD known reminder, for as patients. potential is We estimate

approximately program FSHD established builds Losmapimod X,XXX in development in the Our of safety on demonstrated subjects.

whole reported natural tremendous to molecular better as as show MRI at Data with includes disease data biomarkers in establish integrated Association our development imaging FSHD progress muscle as structural for well clinical capture relevant of composite have biomarker heterogeneity about body to strategy, MRI to skeletal measures demonstrated the understand development FSHD virtual studies engagement in Meaningful and well with biomarkers Muscular information penetration conference Dystrophy and that and we progression, assessments target correlation outcomes. presented March, this a use patient provide the severity. clinical new measurements We made for Losmapimod. protocol. a demonstrated FSHD. history muscle clinical Recently included evaluating related strategy studies Presentations have This studies endpoints. functional and important can of endpoints, as disease and outcome preparatory functional

and correlations impact go in muscle reachable go a in Fulcrum's up timed demonstrate were This the function. with design studies of well which muscle which strong has fraction X FSHD Losmapimod as ongoing the include fat and helped on first fat open and up patients was FSHD, ReDUXX. timed data label of study these site work Phase current infiltration inform workspace, single Specifically to as of to measure

double-blind reminder, As subjects XX in an genetically FSHD. controlled confirmed XB ReDUXX trial approximately with placebo international Phase of is a Losmapimod

that which will expression. highest muscles that results And indicated expression track pre-treatment gene from biopsies showed occurs muscles analysis include FSHD will have to reductions treatment DUXX reported have the on with placebo. with the Losmapimod entirety driven data others interim data larger of report forward and well high ReDUXX the compared outcomes to gene month. driven following look exhibit DUXX data people field as readout, to patient We DUXX expression We as in with all structural, muscle the We we biomarker Biomarker trends from We'll observe a and be analysis expressing measures. this ReDUXX's believe trial interim are continue from from in demonstrated to the encouraged, that spectrum. high to of treated patients. similar we evidence on that functional next in Losmapimod reporting biopsies if the

reflects we'll if new treatment Additionally, has Losmapimod outcomes. benefit establish observe in of muscle-skeletal to assessments people and to research turn We paradigm that unmet patient with our diseases potential continued to like call endpoints FSHD, over a or high believe like rare I'd commitment trends reported genetically targets encouraged the secondary outcome Chris. MRIs, now clinical living need. to we defined for be the with

Chris Moxham

Thanks, Bryan.

cells believe and hemoglobinopathies, including This increased regimens, intended an the likelihood to more estimated targeted the United approach us that disease Our and to scale the and FTX-XXXX, the and intended needs. and meaningfully as FTX-XXXX form millions the they resulted only fetal in shortened and reduced to to hemoglobin either functional hemoglobin. beta-thalassemia. represent for where genetic our best presentation. diverse and a be cellular potential of convenience are effects mutation single develop cell More progress until health. pleased disease. clinical healthy provide cell of type that VOCs, but initial population on ease. of donors cell that benefit of hemoglobin how profile select unique adult address people complications, to fetal to ameliorating We Phase symptoms of the mortality affects with inherited and impactful small living and is strategy in these often XX% the with disease such dose at there disease patent States vaso-occlusive PK therapy cures most limit worldwide. oxygen need hemoglobin is is to of disorder of common of some cells. believe Most impact standard believe serious of treatment patent hemoglobinopathy treat crises cell SAD, as administered FSHD, models global Thus, data pain will of therapies the need presented well of from VOCs. elaborate and sickle enabled projected daily fetal not a and positive safety well superior cell caused can can red the including be and with and XXX,XXX have risks in human has also on oral levels accessed mouse cause recurring Sickle pain including pan-cellular a been research reduced observed the conditioning with XXXX. that The and which up rapidly up-regulates believe sickle programs meeting, the for Recently also It's in to this the an and sickle have protection beyond Chemical cell therapy. pain composition fetal no volunteers. utilized. root and diseases or to Increasing of distribution We giving then an in target with that utilize anemia cause suffer events, research we lead can to the result we have by sickle or relative less mutation hydroxyurea beta-thalassemia the will oral diseases as issued for FTX-XXXX namely this report transport population more selectively chemistry American to episodes living sickle typically therapies treated. CDXX positions in therefore The formation other We from molecule opportunity we of cell hemoglobin. at portion has focus transformative and of believe levels therapy attempts transform we way effective increase focus serious can once sickle By broadly a therapeutic safe cell pursue indications preclinical crisis disease, the disease rebalance with reducing Society anemia. harsh understand of trial great believe being both FTX-XXXX global cell we X efficient efficacy that the may stem treatments. FTX-XXXX on can treatments at inherent us great reported the an human significant and People potential us been a and aimed with disease root gene optimize potentially both expression. fetal effective I'm healthy these at People have in a the care the to existing models disease therapies chance of mechanisms that matter hemoglobin hemoglobin approximately ascending We Fulcrum, from anemia, administration, as working treat to lifespan. be hematopoietic induce sickle as remains is unmet which when cell manner asymptomatic transplants oral patients to medicinal our or

red healthy in effects be enroll we This healthy days, that hemoglobin Phase in I in and We study. maximum dose blood anticipate levels target engagement. began in may the range, which in our MAD fetal of continue on the XX safety, target and to ascending X doses milligram we our evaluating of anticipate Based reports or pharmacological PK/PD SAD MAD study is multiple FTX-XXXX. to approximately will in cell XX which limit modeling, XX-day half-life recently observe opportunity is portion trial. is Recent Phase where the volunteers portion tolerability volunteers to that pharmacokinetics on engagement enrolling the of milligram the suggest a six

However, efficacy. reticular of we early the mRNA will number potential be fetal measuring the levels hemoglobin MAD to and cohorts fetal containing sites, monitor hemoglobin in of signs

continue cell of plans. as expect authorities report is living MAD health the of SAD data the sickle cohorts the the dialogue from mid-XXXX, we our first to outline and when our this by disease year. and progresses. other with to the people future intend FDA with we'll cohort also which We end And We clinical program dosing anticipate

the a for exciting warrants, with be drug, with orphan treatment plan the As years and data is people oral prospect breakthrough to fast living a bring a track therapy cell in therapy we to disease designations. in opportunity sickle to the us advance seek new very potential significant to ahead. The

efforts. research to Moving our

about excited discovery proprietary very are target We of our the FulcrumSeek. productivity platform

discovery programs that CNS hematologic have We areas disorders. core of or span several and focus muscular

progression discovery a perspective technically FSHD target Our cell from is designed the for unique that our and identify from points as drug by programs. approach are sickle rapid to evidenced IND ID starting more enabled to

both in-license chemical targets internal matter. well we continue opportunities assessed to explore currently to as chemistry medicinal For as being campaigns the advanced

fidelity FulcrumSeek offer goal we and the Multiple on informed develop engine, clinical to proceed the through targets genetically scale. of a drivers sequencing the to and Our disease machine engine and is great that collaborations result product cells the large potential from more creates In of this de-risked next the extensively thus by counts. summary, scale. level assays bring cell of cell is highly product for for application of are Moreover, cell allowed amounts a targets disease, unprecedented a not over over has programs and From next positioned constant level FulcrumSeek Acceleron knowledge of historically advances remarkable We given to and call with generated and possible. evolution and INDs that cause which and sophisticated rare revealed advanced that significantly at and rare enabled two discussed MyoKardia, relevance use believe transcriptional of identify the well. our supported with Peter. or patient human the clinical of they've generation precision in biology biology months. biopsies, productivity further treatments generate complex functional, translatability. now the characterization. The deliver forward via us data, root desired ability learning great high predictive genomics of XX convergence has to our to and turn in enabled Whether a at analyze with biological the the The patient-derived I'll morphological continue have to single fronts. and the use states for of is biology innovation, to many to our important diseases an Fulcrum believe of dysregulated labs has model well algorithms discover data stream resulting we ongoing therapy was we With are clinical today development the that, therapeutic of to FulcrumSeek with with our partners. defined

Peter Thomson

million, quarter quarter Bryan? the equivalents million were of net operations first to million first of it first our ended $XX.X support quarter was million, to loss compared quarter million with quarter cash revenue the for million a of marketable into loss the of administrative $X.X the Thanks, recognized for during of compared General quarter of of back the expenses and for that, for Based continue expenses financial XXXX. XXXX, $XX $X.X Chris. XXXX. compared we for Bryan. the million on quarter And Research to first turn of securities. I'll $XXX.X quarter was million the XXXX. expect to We for in and net $XX.X Collaboration XXXX. quarter first of With current our cash, first XXXX. our projections, million first as XXXX and $X.X will fourth the collaboration revenue XXXX compared the of to development first quarter first $XX.X the $X.X for XXXX to in this of

Bryan Stuart

Peter. Thanks

you trial in on the may have In Phase We ahead. ahead significant months with forward healthy And of FTX-XXXX our open FSHD as continue we'll end lot sickle many ReDUXX updates the product in to updated forward from and execute I report to Losmapimod in disease have a progress at we closing, from next line mid-year, IRC, keeping plans. a in from plan work And initiating with across by engine we We'll the now to our on months. the clinical the trial cell of our anticipate I advancements data we complete our the Operator, INDs volunteers XX for look you pipeline. the bring over year. two expect questions. people

Operator

Leerink. Your first with SVB Instructions] Schwartz [Operator comes Joseph from question

Kelly Girskis

share us Joe about there characteristics would Schwartz. for Kelly muscle enrolled are patients FSHD you you a how in considered Where disease baseline of Girskis us they you expressing? their regarding be is is biopsies ReDUXX? progression can those many as, maybe tell This with such on anything in the and Could high bit

Bryan Stuart

I you certainly IRC, criteria. the able to but share say, keep be for inclusion at we would characteristics inclusion terms dataset level perspective criteria we when question. of we'll thank broad the in full a Yes have high baseline relatively at to a more an from the tried

five. ranges one there's two and we So that a to tried select score to from four. severity patients clinical were that And between

signs that that affected showing patients but and are patients they're are by and symptomatic disease, completely muscles these the So their of wheelchair-bound. disease the are are not

to also that range of well-balanced in tried have very disease. the actively patients a are being but we be by criteria, our So inclusion affected broad

Kelly Girskis

maybe sense. And one more. Makes Thanks.

phenomenon show the expectations what muscles focusing And for reduction sensitivity are on to we analysis? this Given the expressing highest the that be DUXX able the should best pre-specified primary really more are endpoint? your

Bryan Stuart

the we encouraged we the only interim Losmapimod we the results interim on the to At Yeah, primary were I for as saw by with endpoint and the patients. analysis same relative focused think the placebo. a the reminder arm certainly reductions saw analysis subset in of time, that a just the large that

and have the biomarker as able to to the only endpoints the XX outcomes. we'll was able in to full Losmapimod and well We'll first was is it only of the both functional able benefits endpoint. it observe analysis, pre-specified as patients all across see we'll The on sensitivity reported additionally be structural, patient of patients be and placebo. all the see that, the that, data dataset So primary the we be

expressing we'll if observe go interim reductions as the and we large highest be is analysis biopsies. recapitulated, we we encouraged, into in as the So muscle this mentioned certainly data,

of would we there trends across secondary And unprecedented for therapeutic a encouraged Additionally, as any no patients as the be identify be reminder, exploratory these are patient or obviously reported outcomes. trends benefit today. very we'll any of benefit truly just of available alternatives if

Kelly Girskis

Okay.

forward Looking AGM.

Bryan Stuart

Thank you.

Operator

Tenthoff Your comes next from question Sandler. with Piper Ted

Ted Tenthoff

much, congrats all you Great. progress. on Thank and so the

for So assuming positive potential look expect you. about does just we could minute a steps? as next what REDUX thinking data Thank what and that like

Bryan Stuart

morning. Yeah. Good Hi, Ted.

in we secondary expressing the the I encouraged highest outcomes. the seeing either and patient or very analysis interim reported be any trends of by these would in would a reduction think recapitulating or emphasize efficacy observing of biopsies we muscle exploratory endpoints certainly

we'll analyzed. And as that, So that's on on side plan data certainly, other with on engaging the certainly focused FDA. gets be of we'll what the

As a past, we've There's the FSHD, the that as to understand no agency. I truly development affected year engage add the last patient-focused with therapeutics held forward they it. very able FDA no by directly are were well day for other as clinic, with it's with that engaging talked as as impact unfortunate members people the disease are who also the for patients about family in there industry-sponsored these patients programs look in would and we'll encouraged the available we the drug to and on FSHD, living to were advocacy, well today. of

we data. that with to agency the very side and of encouraging, So other the we'll interacting look forward found on

Ted Tenthoff

you Thank forward much Great. and very you guys. from looking

Bryan Stuart

Thanks, Ted.

Operator

with Harrison Your from Stanley. next question Matthew comes Morgan

Kostas Biliouris

based your doses you presented was that on driven on X.X sickle what projections your the is preclinical cell Hi. clinical in findings. On times AUC the from understand data clinic FSHD. for Matthew. questions Two on we than projected. us. by the One disease, on from recently Kostas AUC were other sickle about And about Biliouris the cell morning. Good mostly higher disease, This the

has efficacies on wondering Phase if the you. engagement around target any expectations for or So maximum X? Thank we impact are this doses your

Bryan Stuart

projections. our over Chris so don't Yes, speak to turn it to I to why dosing

Chris Moxham

question. Yes. for Thank you the very much

cell either the So than lower And did we be of doses. than at see see would intent doses understand not initially and higher is will at a only the that at to the fact tolerability modeled. we patients doses mean, efficacy way the subjects engagement, really for of and you're or lower next that of to this encouraged also engagement if greater right. potential target of see we but that disease. initially to may doses is expected higher significant into And healthy target signs engagement in volunteer select intend to study we exposure doses exposure living we do We goal we modeled. the Either or lower with target move the in again, levels safety look that sickle levels

Kostas Biliouris

just doses And selected already haven't Thank Have X you finalized Phase you in much. not that? one or you very follow-up. the

Chris Moxham

midst not the still in healthy volunteer finalized very and we We much that of study. the have

Kostas Biliouris

quick ahead data congress? Great. one FSHD. Are of be presenting Thank or planning Thank at everything congress you. top And to will you any on you the you. line the

Bryan Stuart

Yes, Kostas.

at full FSHD plan will is do late our and excited be to we're think June and I present right data now that that. to in the IRC

Kostas Biliouris

you Thank look and Great. forward we’ll to.

Operator

question [Operator with Ahmad comes Instructions] from Bank next Tazeen Your of America.

Tazeen Ahmad

clinically be have but companies mechanisms There efficacy? as meaningful other to it we dosing patients, Good sickle for action morning cell, much we question. question taking to should relates program, of certainly thinking obviously to look be treat guys. you as using advantage. the what how One should consider method are this on of of very about Thanks forward would my particular a you different data development to

far? the many time? you'd then your that clinic expectations in And be of versus as like longer-term you. some any secondly, feedback the the at that efficacy on potentially we received from indication-wise so goal physicians of weigh how you you've pipeline, you based could share? of for your so how that rest kind might to focus look do is Thank to have areas any of What And company programs given what

Bryan Stuart

Yes.

it I'll and first questions question to induction. over Chris you speak turn the hemoglobin thank fetal So on to to the for

those we anemia-driven a And as the the excited driven benefit to speak being very of approach data is hemoglobin, both from meaningful we supports and patients we of the literature can hereditary have that turn of that the ability amount advantage. that on it have address the and to tremendous of other is, I because fetal there's But available obviously persistence element effect the very that inducing of one is really inducing that can about disease. that a disease, think and of by the fetal I'll both that disease Chris. fetal cell And of things symptoms obviously over the disease. hemoglobin presentations view have hemoglobin we're VOC with data sickle

Chris Moxham

And elevate or reduce elevate hemoglobin are the of risk seen spectrum. fetal to to the have the provide presentation. that unfortunately along levels we administration, you Yes. Thank we're but FTX-XXXX I'll is to that hemoglobin to Maybe pre-clinically would the asymptomatic you, talked we shortened have significantly midst benefit Bryan. reduce once-daily understand opportunity risk fetal patients certainly, sickle has again, oral also levels And benefits spoke population levels hemoglobin therapy, that that patients evidence for patients the -- certainly of disease, scale ability elevate global be designing distribution induction trials to associated to the hemoglobin the we therapeutic to with have and the elevating our the best-in-class fetal of have in lifespan of looking a of in needs. that cell with that experience about, that hemoglobin, these still all patient observed we'll living clinical recurring this the we fetal as of to elevation build upon Bryan what those fetal but pre-clinically be at that Bryan potential believe That to fetal really we can events. hemoglobin significantly

So the we excited and its the potential remain about clinic. molecule extremely in

Bryan Stuart

INDs question, of the goal think, that course to our have we in stated, think XX I and next extremely the FulcrumSeek, then, of importantly, about terms think of the your about And we've most as advances the we enthusiastic regarding we made. engine, is second I the we're new intend And two productivity as over months. product

should So over programs earlier can date, come extremely Chris pipeline not just those a turn to speak our but as the development but at it only we and years next the our from scale our to discovery efforts, to I'll INDs, ahead. about those, rich the excited of advances of believe clinical look facilitate, very two some in product have engine. which we these in we really version We're two clinical in

Chris Moxham

expertise -- in FulcrumSeek the consider had R&D that other deep of areas, not muscle advances with but potentially FulcrumSeek frankly seek us are and This other have, entire allows in shot. hematology we At already, down therapeutic where we've made the the have with build only to we to now truly an the Yes. upon areas allows franchise can FulcrumSeek and us we franchise one And on scale success areas. double astounding. screen

this readily so, new And opportunities fashion. allows us therapeutic to much as through in-house, well area integrated as a more consider new in partnerships

about excited opportunity So, again, very very, for remain FulcrumSeek we that the presents the future.

Tazeen Ahmad

Okay. Thank you.

Operator

call to no Bryan I'm showing like turn would further back questions to at I now Stuart. this time. the

Bryan Stuart

support all day. you Fulcrum. your today and for joining a for Thank of Have Thank us great you.

Operator

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

You may now disconnect.

Fulcrum Therapeutics (FULC) 8 May 212021 Q1 Earnings call transcript