FULC - Earnings call transcript Q3 2020 | Fulcrum Therapeutics, Inc.

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Operator

Good morning, and welcome to the Fulcrum Therapeutics Third Quarter 2020 Conference Call. [Operator Instructions]. 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

Thank you, Sonia. you us. thank and morning, for joining Good Earlier our for recent we issued the release outlining a press progress quarter XXXX. this of morning, third and financial results

may can copy and If the have at meaning statements statements Securities plans, this Fulcrumtx.com. include that Private section of call find reminded website in Litigation be of of made development the of you clinical the future our financial the a XXXX. our Reform within release, expectations remarks don’t Please may projections. Relations Investor forward-looking These you about Act during contain and timelines,

While me the these and in Robert our statements Bryan our but taking not Officer; future, certain with Chief views and update obligation with Stuart, our the most discussion relied be today, a recent views Scientific Officer. as may Owen and forward-looking Chief With we should of today's for filings Exchange do to are not Chief uncertainties Officer; they Executive refer our associated an President so. Commission represent Please as and on the call risks Wallace, on future. in Securities Operating representing to statements business. upon We Gould, of are these

cover sickle run our Let Robert. Robert? financials. cell of call With with program over our turn to Robert product this an our through the me on pleasure will to and quickly and overview provide that, will recent agenda. morning's begin will Bryan updates call the it's progress. my engine, Owen

Robert Gould

morning, everyone. Thank Thanks, for you us good Christi, today. joining and

rare in medical to improve a the throughout with areas progress of Losmapimod, research as with evaluating the genetically development, need. program, tremendous made and In strive patients unmet seen we important our we we've quarter, This high our lives of kinase diseases trial. with our Phase clinical MAP LOSVID, progress defined healthy FSHD. sickle We inhibitor, Phase X cell now and of dystrophy or FTX-XXXX, the COVID-XX. Losmapimod with muscular in and for development We're initiated pXX treatment facioscapulohumeral X screening trial selective in volunteers company,

engine continues that executed discovery genetic with we Our grow. therapeutics cardiomyopathies. drivers identify MyoKardia of to July, address In collaboration product a to

in meetings. at made cell to sickle we been key posters scientific our and various progress disease, highlighted in And accepted management appointments multiple Our FSHD team. has

current This the with approximately the treatment is the of subjects dystrophy, turning to Losmapimod characterized the and gene, diseases the call progress the of deterioration. options. that sickle apoptosis, disease. our DUXX, multiple aberrant occurs disabling indications, by of development. previously across muscle be and the clinical to discuss our treatment fetal treatment over is in death to in progressive hemoglobin can Before the the only replaced muscular of is elevate demonstrated and DUXX X,XXX is beta to most Owen muscle by treatment common in like and second thalassemia, the characterized potential FSHD. a disease program review fat. cell I'd as disease program skeletal has Unlike by with safety root potential results that degeneration no muscular tolerability Losmapimod rare, in other expression and expression for FSHD severe for cause

our the Congress patients. of integrated World we Society. at International multiple posters These to This the presented Muscle quarter, posters FSHD highlighted evaluating approach

and include from data clinical compared interim benefits DUXX-driven patients. enabled treatment quarter potential evaluate We reported on biopsies, data clinical track patients. show trials utilizing the in the imaging, The announce baseline gene placebo. gene of DUXX-driven patient Data have reduction expression reductions large the primary muscles that gene made outcomes. Losmapimod all second with of endpoint, full strategy, molecular be to our Losmapimod in and will in expression DUXX-driven design with progress studies suggest remain expression, highest biomarkers, have Losmapimod our to may readouts, encouraging pretreatment analysis, following These tremendous with the reducing from reported also of We the functional of FSHD in to XXXX.

a highest of analysis, and most the pre-treatment believe and assessing report DUXX-driven to report with exploratory will provide gene topline be next the points. expression, FSHD, sensitivity of results separately. pre-specified we'll we in second the in with level and year, end any of we'll able biopsies key Additionally, assessment comprehensive quarter We the secondary totality therapeutic not data

subjects forward look outcome study, the up clinical been to ongoing in year. in also open-label muscle, assessments to analysis who've and change single-center of baseline We the from several which includes treated a biomarker, continued skeletal MRI

recently share host preclinical FTX-XXXX molecule is treat we additional to this the small more thalassemia. XXth, disease For Owen the future cell the a presented cell detail, on discuss December beta patients of the symposium of therapeutic will root supporting for in inhibitor and when cause FTX-XXXX, promising fetal opinion a leader we cell potent, with potentially designed data sickle induce in EED to more sickle and of comparative development highly expression pharmacology hemoglobin sickle disease. experts. and we'll option and key differentiated data

our to program. COVID now Turning

Losmapimod, who top and days, an reminder, at the inflammation. approximately is XXX on with milligram XX Phase twice trial placebo efficacy day, X of trial patients systemic The or primary of elevated to based who age are care older critical of and subjects proportion of day is death oral standard by in endpoint progression a The respiratory COVID-XX. international to illness Losmapimod designed failure for of progress assess is on risk to subjects with compared dose to multi-center the per a As for of LOSVID hospitalized safety XX XX.

September, trial of continue the first saw in on an in enrollment, we opening make progress trial patient update and some and anticipate in in providing delays August XXXX. the to LOSVID sites While we quarter

a served prior of Eli Medical DaVita, expanded Oltmans of years pipeline Novo a Officer, Advisor, of of was of announce our be departure Founder including roles, As brings Alan to variety our made of at a and Research. at covering Diego Ezekowitz our Curtis forward to announce clinical announced Kim Previously, join wealth pleased Curt served to as Merck, as also and And Nordisk, CEO Senior Cadavid, our but part of Kim as I'm Clinical We've search from of since spent launching that President, regulatory Therapeutics. progress, as leadership as and Fulcrum a appointed Alan general since counsel. our where Abide his We'll the management the Fulcrum I'm meantime, in the the release. also Array, team. we've today advancing an our also programs. prior of will experience invaluable Alan pleased end month for property. Dr. Resources. Abide, a Vice has number contributions following joined important has of intellectual legal President in the board XXXX. Human Chief he to to on the in Hazen our look to executive at expertise teams. he serve Lilly, XXXX, to from Clinical been and benefiting in she been team that promoted has Alan's Vice the member has part Senior roles

communities over the amount Owen. to to these challenging of who like everyone through patient at With of continues We've tremendous that, be forefront we keeping phenomenal our Lastly, a what times. progress, made Fulcrum turn I'd all it do. while to at thank I'll

Owen Wallace

add FSHD related I you, to some Thank begin, just our to key that Robert to of want To the points program Robert. mentioned.

many by treatment We’re dataset biopsies, DUXX-driven new expression, large muscles that full potential The following that the the of placebo. to announce reductions gene Losmapimod indicating expression treat the we gene may compared Losmapimod on pretreatment Losmapimod insights in the the highest quarter reducing encouraged second data showed with bring and be DUXX-driven expect will of us to with to FSHD. in XXXX,

team patients to who outstanding our so to of are continue and this the important grateful clinical investigators, We support program. all

adult therapy. of sickled. caused result of mutation drug recently disease sickle a cell we presentation. and hemoglobin, cells has patients, Now form the beta increasing of a across oxygen FTX-XXXX, very suffer mortality, Disease, asymptomatic EDD from September, patients molecule, the Educational are of small XXX,XXX disease believe broad during likelihood raising target. preclinical events such Meeting disorder estimated complications, as the including have States, an to cell transport, a formation in patients disease, and disclosed affects pharmacology red The disease efficient Cell hemoglobin. Sickle patients cell where Cell the comparative We anemia, millions is serious including typically Sickle for fetal capable worldwide. an and as pain also the cell for transformative both Sickle be our Sickle and and cell with impactful our sickle presented vaso-occlusive the of data, hemoglobin in Symposium, genetic a moving in applicability Disease Sickle cell oral United can crises. less Scientific by the that sickle We impact and Research reducing and fetal National to and candidate thalassemia. has is Increasing potential recurring and mutation

that shown was from FTX-XXXX, resulted disease that cells health. cellular of up believe fetal cell models undesirable CDXX healthy could FTX-XXXX, in hemoglobin, fetal both for of mechanisms hemoglobin data human in broad disease. and total levels that We hemoglobin, to standard cell the sickle and effects to profile approximately a have a reported believe sickle preclinical on and pharmacological selectively hemoglobin on treatment impact disease. transformative The Based to profile symptoms. upregulates sickle of XX% potential models could have human we superior cell mouse this that donors fetal minimal positive to-date, disease-modifying We of a FTX-XXXX FTX-XXXX, with on therapy and cell we've supports seen as its therapeutic hydroxyurea, relative impressive other of a care, have induce been that be the with

This sickle more on generated our XX, from we hearing dosing and study cell evaluate as to upcoming With look target KOL now safety, Maureen dose Achebe our a with (indiscernible). in multiple study. healthy trial December has and IMD quarter. forward to our both Dr. and on our adult unique begin in single we're ascending Phase event X Our significant program volunteers action, dose the tolerability, as this sharing well our will open, of and enthusiasm ascending program mechanism X a community, in in pharmacokinetics, at excited XXXX’s engagement Phase trial

report trial as We and X possible. XXXX Phase this as data from mid-XXXX, into cell sickle expect patients advanced quickly in to

application posters Meeting patent three and Hematology of as well. is the our composition matter we next Additionally, at of month have will American published, Society non-provisional

we pleased portfolio. progress the Moving with preclinical of to our our research efforts, very are

we've data druggable Fulcrum of diseased clinical proprietary both genomics probe connectivity addition has This is in has and programs dimensional proprietary new to the cell characterize unique our functional recapitulate MyoKardia. that this from several discovery that genetically of in year. discussed that Our platform chemical of patients. models approach, The human in identification resulted and libraries has today, the programs in diseases. discovery the insights effects gained in and our from collaborations RNA Target Seek Seek and integrates With grown have we complex the identification map to Fulcrum models, imaging that for high quarter. enabling an resulted prioritized and Fulcrum Acceleron with Bryan? emerged and have our this call significantly high for on turn targets seq being currently defined I'll the Bryan and financials platforms, cell over consists pathways biological our in are the which advanced. Seek database content update healthy that, signaling database to

Bryan Stuart

we and hemoglobinopathies updated We operating quarter of FSHD into development well to increase operating to to in for the administrative and increased in expenses costs invest of Losmapimod XXXX, our us second and and were expenses increased third cash, discovery support disease, in advanced the the Fulcrum’s quarter in $XX.X public primarily as Based sickle million with million ended cash as this XXXX compared in million quarter third personnel plan such and our as compared quarter marketable growth were associated COVID-XX, securities. to quarter planned $X.X third a ended organization. of related XXXX. our projections, support of due development equivalents stage of organization. for well The growth Owen. third $X.X personnel as related our the to to September with million $XX.X XXXX, million research increased quarter FTX-XXXX the $X.X million increased will the and XXXX. costs allowing as was clinical support advance costs as and $X.X was XXXX, as General to of operations the XX, on of due $XXX efforts. of trials, of our The support ongoing for believe the company, Research primarily cell million Thanks, increase to costs

continue for Our quarter loss of was several million third net Overall, a we the million $XX of expect net as for third XXXX. $XX.X quarter XXXX, to the loss catalysts. of compared upcoming to

including Operator, may assessments and and we'll line update report to discovery on our cell updated mid-XXXX. from our very XXXX. healthy an with Acceleron our you the plan forward the biopsy, quarter our volunteer work full we product of in LOSVID while up ahead, XXXX, muscle our expect open in to progress report with questions quarter, keeping X continue plans, provide to and on at first We We for trial trial engine, excited sickle in in execute second the on the dosing FSHD, from progress the about ahead. begin of exploratory, continue advance data programs now our We'll you We're in ReDUXX as quarter and this clinical to and to partners disease our outcome FTX-XXXX data MyoKardia. look for Phase months making

Operator

Instructions]. [Operator

first of Piper Ted comes from Our question Sandler. Tenthoff

is line open. Your now

Ted Tenthoff

Great. to the Thanks on, kind to sense progress respect anyway. of updates for important topline particular readout the get think with both won't any refinement data to COVID What should that from get there reason And full and just going I the in first the get se? we the what's expecting really timing, study? and from ReDUX hear the clinical preclinical. on we update. LOSVID per a you quarter from and wanted be both I exciting, that’s part all, Good just of ReDUX. and is the

So, thanks.

Robert Gould

Ted. Thanks,

and of ReDUX end Let providing that on best whole me to FSHD than data muscle then that the assess points includes able endpoint, data FSHD first. expression, patients, totality and out. Losmapimod the take looking as MRI, the complete the comprehensive we'll be to way of it the most secondary rather only are this, patient-reported second We package number of of key assessment MRI that question and exploratory a the with but measurements, a of DUXX- function, at We thought outcomes. also benefit potential believe not driven provide was body in Losmapimod the gene - fragmenting the

Ted Tenthoff

sense. Thank That makes a of you. lot

Robert Gould

Yes. Thanks, Ted.

the as site terms expected of August were in openings mentioned, slower In lawsuit update, than we September. and

in quarter. the on update the we'll the And status so of give an trial first

Ted Tenthoff

the update all thanks Well, Excellent. the on for and congrats good progress.

Operator

next question Schwartz Thank of SVB you. from And our Joseph Leerink. comes

open now Your line is

Joseph Schwartz

means Great. other much. your spec you flow preclinical elevation questions very measures couple given HBF I disclosures. those you the are what I could had you each on ran superior XXXX, walk Thanks recent telling us when that relative potentially could a that of quantification the different was HPLC, you wondering you in describe if have of for like of and and attributes experiments, cytometry mass to agents. us through conclude

Owen Wallace

think with fetal is what - sickle we're Yes, I generated phenotypes hereditary data This of The we've very sickle consistent hemoglobin is excited very persistence in Owen. preclinical the Joe. the individuals. program. Hi, portfolio that and about pre-clinically

who fetal achieve around positive we levels. our attribute fetal variety One mutation of looked people them continue if that have but key the expressing of pet hemoglobin expressing models, also fetal hemoglobin we've is cell used are treatment, the is to the XX% to And fetal these many to with preclinical the causes hemoglobin. flow cells. the of XX particularly another measures hemoglobin. induction. are cells they a of cells at cytometry a of are fetal In have And induction. particularly CDXX of and a XX% in these of asymptomatic, hemoglobin of mutation, So seeing express and sickle cells, the this have cells to we're which estimation of by percent fetal upwards F And measured of cells, cellularity percentage of get F or number XXXX an hemoglobin F

So we very think a that's profile. attractive

is hemoglobin donors fetal And data, levels XX% to consistent two healthy both And using the fetal upwards a technique total standard are we're different and ways. HPLC, measurement that seeing key other measures looked to are the really sickle using techniques, induction made with for which have The regard is spectrometry. of in hemoglobin of we've complimentary hemoglobin. cell multiple One different And induction. that at across is we donor of those which very their fetal cells, as human-derived in two mass XX% again, and well. gold

So we it's profile. a think very attractive preclinical

Joseph Schwartz

healthy to long That's see you have hemoglobin on long Thanks. could that F be volunteer that Great. study, to enough make then cells? enough your coverage context. it helpful signals? interesting Phase more X enough show some be or improvements, should preclinical fetal Do tox And focused we on that will

Owen Wallace

The PK, safety, two is in anticipate target engagement. than Phase tolerability dosing really X designed longer volunteers. don't for healthy We weeks

the we'll there's cell of just of F a HBF, given we red very a cells see So significant healthy likelihood, elevation in formation low in blood healthies. in think increase that kinetics individuals, or even an

as strategy the is patients trial hemoglobin, will opportunity the through in sickle X the Phase healthies. that best F relative and dynamics believe healthy blood sickle increase in rapidly we of volunteers and in cells possible, into given formation progress because cell patients, general to both disease move offer see then production to and cell as fetal increased to an Our

Joseph Schwartz

Yes, sense. that makes

Okay. taking my Thank you questions. for

Operator

comes Thank you. from [Operator Instructions]. question And Matthew Morgan Harrison Stanley. our next of

line Your open. is

Kostas Biliouris

do, trials or monotherapy these cell thalassemia Thank evaluate the Kostas are like quick planning Phase the with sickle to planning for also or bit beta particular, the Can morning, to as a in plans please Matthew. compounds XXXX. on you. Good after little it everyone. This a I about in you disease? other question only? you also In on And you X? is have hydroxyurea, talk evaluate development are combination a

Owen Wallace

profiled combination and standard in last molecule certainly Kostas, we your of potentially hydroxyurea on in have with hydroxyurea. first, touch that the with to question profile is the we believe - We additive pre-clinically, care

We be fetal with used upregulator clinically in combination that for other sickle also believe approaches hemoglobin could cell the of of disease. that are being a some pursued

With on So, absolutely, post all X, development details. regards those we’re radar Phase plan our that's working to screen. through still the of

got We’re be data, thalassemia certainly opportunity measured see to I into sickle considering as well. disease upregulation hemoglobin, just mentioned, thalassemia believe, moving that opportunity fetal an that beta as well. likely in move And We can definitely cell then once effectively the previously patients we've beta initial most into as of an there's patients. to

Kostas Biliouris

you. Thank

Operator

Thank And this does answer now gentlemen, over Robert for any our turn and remarks. you. I would to ladies conclude back and question the session. Gould call further

Robert Gould

safe. I for participating joining programs us I all the Fulcrum been Fulcrum. of patients thank we as support move just thank here with you your want also to clinical The team healthy forward. our to investigators and and you that want Thank us today all have you. for stay all hopes and at

Operator

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

now You may disconnect.

Robert Gould	CEO, President & Director
Diego Cadavid	SVP of Clinical Development
Owen Wallace	Chief Scientific Officer
Bryan Stuart	Chief Operating Officer
Christi Waarich	Director, Corporate Communications & IR
Ted Tenthoff	Piper Sandler
Joseph Schwartz	SVB Leerink
Kostas Biliouris	Morgan Stanley

Fulcrum Therapeutics (FULC) 10 Nov 202020 Q3 Earnings call transcript