Earnings call transcript Q4 2022

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Operator

Greetings, and welcome to Fulcrum Therapeutics Fourth Quarter and Full Year 2022 Earnings Conference Call. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce to your host, Chris Calabrese. Thank you, Chris.

You may begin.

Chris Calabrese

morning. good and you, Thank Welcome to XXXX and Year Fourth Fulcrum Results and Therapeutics Financial Call. Quarter Business Full the Update Conference

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

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

Ezekowitz, Robert of financials corporate Interim will Chief the open be Gould, updates; Esther the who Chief call Financial a for call the provide we J. will Rajavelu, Officer, Officer Dr. will Executive overview key who pipeline cover will today Alan before discuss who serve Leading will Q&A; the over Robert. that, and Fulcrum, With a to the Fulcrum Member Board pleasure turn will the adviser, answer of call be to of senior Directors clinical questions to call. the of as able during Dr. portion and Q&A it's my

Robert Gould

time sickle our our team. runway; cohort updated important including suspended everyone appreciate on and XX-K, our the business on release in changes I to of morning data provided hold, color clinical cell us this the cash press FTX-XXXX morning. full Good now additional the taking guidance XX-milligram the Chris. trial; Ib you, disease updates today. join Thank We to management several Phase and from

by disease. HbF recent patients for sickle FTX-XXXX me oral cell with Let discussing our treatment inducer updates potential our most the program, to the of start

the for drug from clinical from hold XX, they verbal received on new we formal we that the full announced and XX, February FDA As had on application on placed FTX-XXXX, received letter notification FDA investigational February XX. we clinical the hold the a February on

dosing suspended In PRCX. paused XXXX, related evidence the observed in data December the that with previously us, clinical communication noted agency to inhibitors the trial. of repressive Phase its April, whole or to October Ib malignancies the both immediately and complex We in and and preclinical enrollment X hematological as other submitted polycomb nonclinical and

to studies define risk. other PRC have of noted inhibitors. that profile PRCX hematologic is FTX-XXXX continued with FTX-XXXX that potential similar observed the that requested agencies other for potential that the population Fulcrum The further the that inhibitors of with with reported clinically nonclinical the treatment hematological specifically observed outweighs agency of been benefit malignancies malignancies in The and X,

a dose not with have subjects XX-milligram dosing and baseline, increases we from treatment clinical X-milligram shared that and background the was hold. completed the the early was and any the we that dosing to XX similar cohort. the and we're responses HbF from cohort and to In up dosing enrolling FTX-XXXX fluid at demonstrated and hydroxyurea. off January, X.X% patients data dose, on hold result hold, X-milligram of Prior patients ongoing at we're cohort finding time the the clinical The in to X-milligram in trial completing Phase at completed Ib of

completed up and We HbF XX achieved suspended dosing, cohort, the the an provided morning, in from of January. also patients ongoing set to now which The we completed updated days dosing the we X-milligram X-milligram This partial cohort enrolled cohort XX-milligram data increases absolute patients data that the X-milligram in shared X two from X.X%. patients. that

XX same patient of suggesting improved of Through in A well of days Data way demonstrated a of efficacious treatment, HbF as with minimally biomarkers an to of X-milligram XX increases average XX% who from absolute while than patients of an early as average a X.X%, completed during comparison up dose absolute at potentially had time treatment, dose. a days, X%. this point HbF hemolysis. absolute cohort, increase dose of adhering like is patients in X-milligram end X-milligram our in the XX-milligram HbF increase less the had

had deciliter support to XX-day new find For of dose X at these study effect. of day of We highly increase clinical with to initial the the XX-milligram All deciliter response and as dose HbF, point in as XX. of time reduction per encouraging, of supportive gram potential data this a per hemoglobin a be at these data least by X-gram continue also achieving a robust the an drug. significant subject increase one further subjects X well

FTX-XXXX on XX-milligram both with has across dose events. treatment-emergent background consistent to hydroxyurea, standard adverse been or cohorts, and in tolerated which generally current to subjects drug-related adverse subjects, no emergent clinically and showed serious is care. well improvement relevant the date due discontinuations X treatment events off adhering of All

forward the concerns that disease a gave favorable benefit the XXXX working a to clinical sickle We FTX-XXXX great all and further These us closely differentiated to potential living therapeutic with remain possible. as cell provide as data committed with outstanding option to FDA development address and has to confidence for rapidly risk people look profile.

forward. once update the on an we have provide path more clarity will We

are Phase now, fourth XXXX. to For the registration suspending dose of enabling we guidance the Ib to select previous and trial, quarter in guidance our the our complete

Losmapimod our to pXX inhibitor. a mitogen kinase advanced turning Now most is protein activated selective program, alpha/beta

is has an estimated to infiltration characterized pain. XX,XXX and results genetic of life of of XX,XXX a population mobility Losmapimod dominant dystrophy, patient an extremity and daily is and States upper is in in in alone. the United function, Phase FSHD. muscle for fat which due of treatment death III facioscapulohumeral muscular autosomal development chronic by inability perform impairment FSHD significant progressive to the It to activities loss the

Phase losmapimod reach common and track to XXXX, progression. potential urgent muscular We initiated trial in disease-modifying effective address III slow can and points enrolling and trial are The the in And approved most dystrophy, enrollment the has there we complete the no expected XXX of the Europe. a half currently one on our placebo-controlled safe need or is losmapimod U.S., we in patients disease it to of the adults, that stop are While June believe are treatment XXXX. for is of enroll Canada currently and to double-blind approximately in second treatments.

extremity from workspace, and range The maintaining of daily proximal baseline for evaluates and upper influence self-care motion specifically other ability is shoulder life change a the the with independence. function and moves primary measure endpoint or quantitative critical and XD of reachable function the arm activities sensor that's technologies. RWS, extremity for that of upper directly in living, is of Preserving quality absolute motion

Patient In outcomes tolerability, pathology and the such that disease care to thinking These as Change, launch. commercial of and and include include our will Impression addition a for as measures. or endpoints potential prepare Muscle secondary inform strategy will about Fat safety marker life questionnaires PGIC, quality utilization self-reported important of or Infiltration, an Global health we MFI, of payer

is geographies. efficient was both REACH a the in in highly registration be ex-U.S. enabling as XX-week and intended trial and to U.S. designed

IIb demonstrate and are we relative to in that progression ReDUXX reliable demonstrated We to compared weeks. confident placebo losmapimod of have RWS we improvement to advantages trial disease for meaningful XX our at hope selected placebo. significant measures Phase Encouraging,

from line extension who the has top in continue durability through Furthermore, shows open-label initial to the period. losmapimod results of treatment participants ongoing XX-week effect that receive arm demonstrated a

mean trial initial placebo crossed over losmapimod, from patients progression XX-week from change disease of slowing the by to Additionally, RWS period, measured after baseline. improvement in who showed as

data therapeutic from long-term losmapimod. profile. extension and We of areas encouraging dosed and safety patients results the benefit ReDUXX and these our believe losmapimod potential open-label multiple disease-modifying been support provide tolerability and has To across X,XXX over trial in evidence of an date,

U.S. the have our safety extension on And for with ongoing forward reached primary from our for large we continue trial. on the we'll an the endpoint alignment and to as REACH. We leverage open-label learnings path database, and Europe build regulators ReDUXX, clinical drive losmapimod, in

company this opportunity. that announced Santiago to November X Now to matters. who from in Arroyo, March the Dr. us last effective another turning our morning joined XXXX late designed Medical Chief corporate other Officer, pursue We week,

We advancing are CMO X deep early effective interim in regulatory expertise decades Fraser, affairs. through in Dr. and appoint brings today. therapies over of Fraser experience as Dr. Iain excited and possesses to late-stage development

by He most clinical previously Fellow He recently President as And Lundbeck and development clinical part held increasing acquired Merck. at responsibility Clinical the Vice of Bio Elevate Therapeutics served of XXXX. prior that was in was AlloVir, that Company. roles at in organization Abide development to an

continuity. will provide senior With addition Directors Dr. call of serve member since over clinical an a to Dr. I ensure update advisor financials. Esther program the XXXX, on to as turn us, Fraser Ezekowitz will In to our joining Alan Fulcrum the of that, Board to February

Esther Rajavelu

provide stock XXXX. and January million XXXX, of results. ended our $XXX.X we million marketable compared an on of an $XX Thank XXXX at cash then per securities to and In and our price and sheet December share. balance you, financial X.X in Robert. offering common update will million I of following public XXXX, equivalents position year of and cash $XXX.X December runway shares XX, public fourth XX, underwritten strengthened a offering cash, We review full quarter cash with guidance, first position

offering-related expenses the approximately proceeds gross company. underwriting resulted in and approximate were proceeds approximately $XXX The before deduction from net million this of of other million -- and to the discounts $XXX offering

cash, a operating of with a mid-XXXX, approximately $XXX expenses our expect cash to marketable fund tiny balance cash assuming financial million to clinical a FTX-XXXX We our operate from securities the and equivalents and continue of position forma strong resolution hold. pro into

fourth of year collaboration $X.X and previously the and of the million to to due for quarter was the XXXX Collaboration year fourth XXXX, compared year decrease 'XX. and million to turning for the the $XX.X financials. revenue full with was fourth for Acceleron Now XXXX announced The Pharma. termination full $X.X million of for full $X.X quarter agreement Fulcrum's million primarily quarter 'XX

million increase 'XX, was milestone costs, REACH fourth the to the to in GSK of employee-related the and of including III $X the including 'XX million $XX.X fourth primarily increases paid $XXX.X $XXX.X a million for 'XX compensation trial. for due were for increased stock-based quarter The R&D, 'XX. increased compared $XX.X initiation to million Phase full year upon quarter the and of for and million and year expenses operating Total full

the Net of $XXX.X to quarter million $XX.X loss loss was $XX.X 'XX a of of full quarter fourth and the year million 'XX. 'XX, full for for for million compared and year 'XX fourth net million for $XX.X

the turn to Robert back remarks. that, With for call I'll closing

Robert Gould

this the our on corporate We executing Thank year, our objectives. working to of focused our you, forward, are to half leverage remain for to clinical value clinical engine FDA track opportunities of remain exploring on and the FSHD We research driving committed on the complete the enrollment programs resolve Esther. on to and hold REACH trial III in Phase second FTX-XXXX. with our

communication the diligently to FTX-XXXX resolve we We there the path that hold will and updates return want are forward to clinical full reiterate FDA working as proceeds. is I provide we to FTX-XXXX, are a to on clinic. with optimistic enable expeditious the

Operator? conclude for today's to the we closer would along root families. sincere questions that foundational including the work many who genetically extend I to of With been gratitude our entire and want I and are to the call, Fulcrum their like [indiscernible] Before patients. that, supportive our bringing and transformative patients cause have we investors us appreciation diseases my and therapies to treating brought to team, defined thank way, happy the take the has people

Operator

a question Morgan first line Stanley. from Harrison Matthew of with take the We'll Instructions] [Operator

Matthew Harrison

Great. I guess two for me.

study I specifically you what first, about little Type I do to a that hematologic expect expecting in FDA? think patients request given the do with full the And on the the and the very be talk clinical do you that may risk? meeting certain RAT potentially your now you So handling that you it's know think you the just -- could the then toxicology have hold? just observe secondly, you And current the letter study is new around plan necessary? study, bit clinical Or from could maybe A a early, exclude change are you malignancies you guess, to

Robert Gould

you, Thank Matt.

benefit/risk we to asking encouraging move may elevations a the Just benefit they in taking your dialogue really us that from then, as ratio. in second therefore, with the that were We question HbF the -- noted forward patient define and us they population the FDA, address first.

studies. As a patients we're that for on other defined those you taking at the at cell arena have studies And VOCs. in numerous probably look close sickle that higher things know, like gone have higher are risk, risk

the an what essentially noted sickle we're like. The higher was oncoming risk And look study forward, that PRCX. cell profile of that observed have population all clinical can moving in of in we XXXX one study observed And the other in is we other to address Our of hand. the things believe will population, sickle malignancies patients. cell considering with the we was questions with the similar studies that data hold inhibitors of that

Matthew Harrison

just And -- the And Type or don't to request follow-up, meeting if you you know Robert. not? planning can you that Thanks, I know yet. a are if A

Robert Gould

keep jump Yes. updating We're forward. the putting had immediately to on what which a Certainly, them, exactly start plan that. we've that the working I as going call still mention had with through letter together. moving meant develops. plan strategy really is to after our we'll us the almost be does And we

Operator

question of line from next the Oppenheimer. the take Matt with Biegler We

Matthew Biegler

as tazemetostat relates It risk? lymphoma. it on the reason here ablates to there cancer might works risk an comp it by EZHX think best recently I approved a XX%, but think Is PRCX Because drug? you're access. that the it inhibitor, that's think it. whereas also What's completely and tazemetostat, was about Is be to reducing with methylation cancer only I think lower your

there. I'm kind curious So are what of just thoughts your

Robert Gould

precisely That's Yes. correct.

So sarcoma follicular relapsed and the that's, been information lymphoma on from that's tazemetostat as well improved in as tazemetostat. therefore, epithelioid provided refractory

dose any PRCX that fully noted most of point volunteers for into effects. out healthy reasons observed population, in the not have label in cell I'd those we've happens We In inhibit insight And thrombocytopenia, tazemetostat. of they when the sickle anemia what as you for the neutropenia, tazemetostat, our complex. modification

versus inhibitor your difference like an is think that. answer how So in is development fundamental how Does [indiscernible] interacting question? and tazemetostat a there's we clinic the that affecting EZHX affecting XXXX and hematologic

Matthew Biegler

with follow-up only patients the identifying that HbF and about to about I with [indiscernible] a also higher maybe Do HbF? including baseline a something prior question wanted there on just makes population between you I foresee HbF link to induction yet? benefit. sense. remind has us, Yes, -- And the patient like been baseline drug

Robert Gould

let before baseline the let Alan higher the to me I risk So question. speak address population,

relationship of on FTX-XXXX. from degree the And higher they HbF about to experience XX% where we to We in with induction XX% start patient and the baseline we're on whether have not we we seen patients or up X-milligram not, a most baseline data have is, maybe population HbF hydroxyurea the about range similar want of and that HbF. now certainly, independent the independent speak of That HbF of whose between see are they have seeing question? to you where higher-risk increases about HbF. Alan, to X%

Alan Ezekowitz

the VOC need and like therapies, other -- of we which done expand by exclude. Ib, we open-label seriousness of therapy Yes. been would the was have that as incidents we study manipulation just The to of baseline parts and a recognized an complications we frequency out in we would along. a that well go with studies with gene did, all-comers that the Phase other because population as the we start study our increasing are be of and gene population strategy that, we like and need that some guided like VOCs as that episodes would of And chest high-risk then syndrome the

Operator

Sachs. the take with line Kumar We Madhu from Goldman next question of the

Unidentified Analyst

Madhu. for on Rob is This

used to does risk? in how do what compared observe dose you First, translate PRCX the cancer? XXXX clinically? malignancy inhibition what hematologic you PRCX does And of to observed other complex then that preclinically drugs And

Robert Gould

Yes.

compare. preclinical to to And in sickle the we inhibitions, But can be we're cell really degree we Trimethylation as that previously, the insight the benefit using. most of discussed that we discussed HXKXX not We've related appears have confident of the malignancies differentiation patients. we've define related clinical have inhibition ability the about best forward PRCX in to the XXX%. as -- dose Tazemetostat, move exposure to that risk to to we're getting XX% mark. XX% the and getting we're XX% we we're that where the So

Operator

take with the from of the line Suisse. We next Credit Frommer Judah question

Judah Frommer

Yes.

profile. defining leaving preclinical precedent had data feel on what is clarity set you working on benefit/risk Have you're for the up are that risk? on are editing that they further it to if up potentially from following definition FDA more programs that And you? gene is of whether Or you have particularly currently? you Just sufficient

Robert Gould

Yes.

them So on which specific editing that is they gene And conversations any we define have that had in think the we've comparison. with Certainly, that greater. risks they're a clarity is discussing not associated indication making provides how population the with the nor there from are we them that benefit can approaches FDA.

Judah Frommer

your that defined into be would cell potentially to higher back population defining to And population? risk that restricted that Okay. or population you a sickle sense be getting broader is could that step the

Robert Gould

for It's that FDA. the yet a course, that to little know with us early because, of involves discussions

with plan as and current beneficial to we the it risk demonstrated demonstrate FDA what ratio higher that provides higher And continue -- beneficial Alan is. once Our will we think what think said, on is population in that risk for what additional discussions population, benefit/risk opportunity define the discuss populations. to us we a

Judah Frommer

general maybe large, still on And communicated programs recently, anything in we've just coincidental just Could incremental related then more from defined. your out there that unmet kind be gene view. seen sickle might pretty then be that, need be sickle any cell risk drop to timing perspective, And Okay. to some to got editing which kind FDA's curious, to in insight those that of benefit needs maybe lastly, seems programs of FDA understandably, be cell just here. you but

Robert Gould

Yes.

received all came indication from the there that is editing was our for same There no in we letter world. coincidental cell read-through unfortunately, the data clinical the gene any this up that at correspondence patients, hold that time. in sickle think, I it's

Operator

We'll Ha question Dae line Stifel. take our next of with from Gon the

Dae Gon Ha

I you more a references I'll the which observations saw little observe detail were bit may. stick with into around guess, go earlier a this? months There if guess, label and the about but nonclinical before observations, exactly point X the I duration that. were I they indicated about of, you Can couple what tazemetostat, that, at

more do Have to when disclose guess, to materiality DOGS, believe potentially So for nonclinical related And a then complete. or portion studies, bar to forward, I you and what the is in RAT public? going I that in-life as the you contemplate seen that? disclosure your you define as

Robert Gould

done from remind the provided in malignancies and that studies as in appeared. the as to healthy the as certainly, Just volunteer you, population. much XX-week dogs. XX with earlier was those of the rats, we had cell study to weeks initial and XX in my report XX, well study sickle I available comfortable certainly, X, was FDA disclosed dogs. initial the the And and the not FDA as just that on when But IND to treatments updates script. in the during XX-week referred the We've the well X of XX-week and received studies draft weeks, at We've material course

We the also will plans continue risk to provide increasing the FDA, and and dialogue the with do we -- risk that benefit the on not would side, updates as they to but only benefit we we and have would address on... like discussions correspondence define what to with and how particularly

Operator

further any have you do Gon, Dan questions?

Dae Gon Ha

you very much. Thank No.

Operator

with line from question of Ed Sandler. the Piper next the Tenthoff take We'll

Edward Tenthoff

we've I So extensively think covered XXXX.

to this able looking really could it's as So differentiate the I in establish progresses. you're broadest important, I'm population. think on that this And if with ultimately safety for I more think hear that. drug population to luck possible good

for Or just as of broadly this with drug see remind workspace where know losmapimod you a to? but the you primary most Is see that appropriate be working reachable I at drug? appropriate? kind where drug again, most endpoint, looking we're the applied would FSHD of So patients maybe as do us this kind that

Robert Gould

results the an FSHD be the It's a white adaptation. has that wheelchair. broadly advocated to that agent potential I'll who you stage a on voice ReDUXX think study, broad in for Yes, workspace of patients this they applicable over at disease crossed -- with where certainly, we were able population remind based in and patient repeatedly then do but yet and declined that patients we're the the they're not were stabilizes were paper population. trial when open-label a that at. who stabilize the a we they is drug you the to and of to that the patients a took and disease if And has extension. patient reachable are placebo population we showing stabilize will, around symptomology, the That saw in

in of the the where to of sort population range bulk that's So the scale FSHD. clinical used lies. X in X that's of And

very So it's across applicable broadly population. we a think broad patient

Edward Tenthoff

Great. next Excited that year. that for year. this hopefully, enrollment data complete And data,

Operator

Schwartz next from the of take We question SVB line with Securities. Joseph

Joseph Schwartz

[SL] have wondering, determine at of related you have blood far? it FTX cell arrest. promoting And done that cultures patients to maturation deformity, what CDXX any that was a And to in via data I looked on treated [indiscernible] the impact red the quality cytometry development FTX-XXXX the of flow about is confident you viscosity question, RBC. and maturation be erythroid on inducing on morphology order isn't so

Robert Gould

Yes.

red in with correction of see studies. we And count. have consistent in we're anemia. in seeing ameliorating don't the with see and is any that the differentiation impact hematopoiesis at didn't focus reduction particular We you that's on the or the any anemia looked of population. that One Certainly, the But in potential effect. erythroid the that's cell really might data vitro consistent maturation site on vivo absolute patient

and no cell the or there's on an red -- differentiation. other So effect evidence

we of sort formability at seeing at showed those In formability. of XX, surrogate red the of as we of parameters. the RDW And work, cell, the is as which X get and the distribution normalization we've the a also for looked what terms is at we're

the in the the still the saw with preventing HbF cells consistent We're were percent was of pancellular. is or that's event preclinical sickling you occurring. in that studies, So the I'll raise [indiscernible] from of remind pancellular all cells the responding analyzing we cells.

Joseph Schwartz

more on Okay. you be will an any reporting [indiscernible] off then patients, partially those in And these drug? data

Robert Gould

Yes.

forward we As how I still and hand as provided of we currently patients date, be all the provided all Certainly, we we're interested -- cutoff still have as we and we moving the have forward. forget following you're exact and but these we've provide we'll updates move data the them. data considering in on

Operator

We'll of take Ahmad next of with the question line Bank from the America. Tazeen

Tazeen Ahmad

next-gen Robert, just in you on profile could. this clarify the correct if Do if you think in you their you think then to time to think with on you a maybe we the clinical calendar be lines, be might be be terms in-house? Do able that you're take in-house? for you'll year? safety side? differentiating that agency Or that's secondly, And questions us what it that what themselves listed something How quantified you can have and in that has answer could remind hold of could while? the of data all assets could the

Robert Gould

a X you hard months as engaging it's timing X least -- as we not we've little the hold will because, quickly in So still the to control. the me to anticipating that be it will be we're at at lifted don't know, earliest. clinical there's We're how for with dialogue FDA, predict

future that IND you as do other muscle number year. we'll recall, as and sickle in we well anticipate probably programs cell the We stage filing preclinical this dystrophy stage, drug have a of in discovery at the the the another be not as those discussing are more both

Tazeen Ahmad

the FDA that all? the plan change from was -- feedback that Were at

Robert Gould

No.

Operator

floor comments. the now would Ladies session. the you, end we Robert over and the sir. reached gentlemen, Over question-and-answer to And have back Gould turn like to of I for closing to

Robert Gould

safe time today's completes call. everyone Thank Please and stay to you. this healthy, attention for your Thank very much and you today. and

Operator

Ladies and teleconference. today's this gentlemen, concludes

at disconnect lines may for your you You your this Thank time. participation.

Fulcrum Therapeutics (FULC) 9 Mar 232022 Q4 Earnings call transcript