Earnings call transcript Q1 2023

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Operator

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

For opening remarks I would like to introduce Chris Calabrese. Please go ahead.

Chris Calabrese

morning. good and you, Thank Welcome to and XXXX Results First Fulcrum Update Conference Therapeutics Business Quarter Call. Financial the Please be Private reminded contain that remarks made during within Act XXXX. meaning Reform forward-looking this the of Litigation the Securities of statements call may company's These expectations including FTX-XXXX, development and of financial and future time may hold about lines, clinical projections. plans, statements clinical include the the

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

Fraser, Dr. Officer, for as the Dr. today Chief during it's discuss we With Q&A Robert. corporate financials a Robert Interim call portion the over will will Fulcrum, answer well to to Chief my overview, open of Q&A. questions that, call call the will as Executive Iain be the who call. updates Gould, to of Officer Leading able provide the turn the And before key Interim pipeline be pleasure Medical

Robert J. Gould

our diseases genetically Dystrophy. everyone and Chris. Losmapimod X in In the of Good with progress to to hold address our advancing and morning. trial in clinical of deep Facioscapulohumeral improving the Thank areas first make patients REACH of like to unmet continued XXXX, you, high medical join the of defined need. Phase we months the I FTX-XXXX taking organization today. Muscular few our to to commitment I'd appreciate lives us time begin reiterating by advance

X, Chief of Officer July pleased also the of announce and Member the to are We position Directors and of of effective President XXXX. Board the Alex the Executive appointment Sapir to

achievement. leader thrilled special world more sector from to public last of industry are the Board to building stage organizations. to we than Alex and of class Friday, commercial of have on As advisor years his propel knowledge to me brings has XX a Alex experience of stature level pharmaceutical deep and Fulcrum joined as the next

and served Directors XX Board in the as of the the Chief Officer ReViral, to Most of years to as Chief career, activities. Therapeutics recent held Executive Directors responsibility on now critical by company’s Executive Pharmaceuticals. Pfizer. this Officer Vice and as dive provide Earlier recently, President this of a Executive record prior Alex's Member Dova of Alex Alex Member of ReViral served President United spent Board and for of fit organization and for brief Prior his into update introduction, and increasing With within juncture. roles the an of at of GlaxoSmithKline. an makes at commercial Marketing Fulcrum programs Sales and acquisition track him ideal our I'll impressive

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

full a notification paused the received trial that on FTX-XXXX. and placed new FDA in February application We FTX-XXXX As the drug from previously a for February the received on they hold XXth. clinical clinical we suspended XXrd of verbal the FDA had dosing from letter formal on and immediately hold announced, investigational we enrollment

clinical and more we'll collaborative, and we resolve work in the forward active agency provide hold. with we update been been to and far productive look thus on as our interactions have regulatory path with and ongoing continuing clarity our dialogue dialogue the to forward. have have we We once the Overall, the an FDA,

define other evidence The that February on and to the Phase any Fulcrum clinical polycomb is April, observed findings safety of December a in XXXX repressor has nonclinical the that agency requested FDA is October, PRCX. X in The In focused population Xb with the result the the and of related outweighs and or was stated initial or the risk. and complex of submitted hold at hold potential the tradeoffs preclinical balancing inhibitors hematologic XXXX, provided malignancies potential clinical and not feedback treatment hold. trial in benefit efficacy ongoing of the the further that with of FTX-XXXX time continued data

a level the dosing we who clinical prior of further baseline, from have For data from subjects FTX-XXXX, of completed days hold. showed HbF XX after Treatment increase a XX-milligram XX.X% resulting to treatment. of a total XX% highest set context, HbF the the dose strong absolute with in

with emergent generally subjects with that adverse adjourned and maintained We showed milligram off to current been and has discontinuations clinically X sickle the clinical and background disease for hydroxyurea, has or events generated date FTX-XXXX benefit the option differentiated well serious therapeutic date to subjects due tolerated of consistent the in XX preclinical FTX-XXXX risk improvements living events. milligram relevant both across demonstrated to cohorts care. All treatment Additionally, adverse provide standard cell treatment-emergent no data on dose people potential drug-related favorable and that to a the profile.

inability to is and States daily life to XX,XXX loss the United population by our program, Losmapimod, due other zonal and a of form significant Losmapimod treatment generic of a of which has the alone. most advanced mobility, selective impairment in inhibitor. accumulating activities loss of extremity FSHD kinase to dystrophy pXX in chronic patient functional characterized X relentless and is estimated activated Phase protein turning dominant to upper in function, and the perform FSHD an development alpha/beta Now mitogen muscular results of muscle XX,XXX for and pain.

of common approved treatments. forms currently of it's Although dystrophy, no the muscular there most one are

our extremely REACH XXX by the XXXX double-blind initiated to are slow for believe Given patients enroll treatment need this At second for complete urgent sites X unmet adults currently safe address XX is therapeutic Europe. preserve potential of expected potential to muscle and innovation, and are approximately disease-modifying in placebo-controlled or need we in progression. to U.S., trial a trial encouraged high June of and the it This the sites. XXXX. XX Losmapimod's Losmapimod that out and half and of disease effective has Canada, the expected in Phase active the function stop time We enrolling are enrollment to can XX-week

are execution. the pleased sites our with team's is trial the engagement in We which by REACH rapid enrollment level Losmapimod Phase pace X to of of our clinical a and strategic trial, testament the both high

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

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

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

top significant to reliable Phase disease trial we Encouragingly, that improvements from label through and the Furthermore, period. ongoing continued ReDUXX advantages at We our initial Losmapimod measures XX-week receive open are placebo results that progression, treatment have effect demonstrated shows placebo. who XX-weeks. line durability participants a selected relative of the ReDUXX hope we of in demonstrated Xb extension to meaningful compared for demonstrate confident RWS arm to in to Losmapimod of

to placebo disease extension of after an of modifying mean ReDUXX support crossed and therapeutic RWS believe who and potential profile. the disease as safety and patients trial and from dosed been showed these over long-term change initial has across improvement measured the Losmapimod. To XX-week benefit Losmapimod over our patients X,XXX We and baseline. Additionally, provides evidence of from tolerability in data from progression by Losmapimod encouraging areas results date, multiple slowing open-label

forward path learnings and from extension we our safety on drive open-label for ReDUXX database we building ongoing our As leveraging Losmapimod, the forward large and clinical look to trial.

to other turning matters. corporate Now

As and company. commitment April appreciate company the previously made announced, are for has grateful our the Rajavelu, We effective from operational and XXXX. she her Financial to our Esther excellence Officer, contribution resigned recently financial positive to Chief XX,

We continues to with team the to Esther our execute work an Advisor, continuing in finance strategy. are while her role financial as

serve As as to of Fraser Senior Alan Fulcrum a to Iain Advisor Dr. CMO February continues reminder, Dr. to as and serve Clinical the Ezekowitz, continuity. of continues XXXX since program ensure Directors Interim Member Board

As Fulcrum’s on focused solidify hand. remain the and to team, our we work we mission continue at leadership realizing

$XXX.X underwritten cash XX, million equivalents, an January common With on update that XXXX, net million of ended public with million $XXX.X compared December our $XXX.X in completed I to securities our an XXXX. provide XXXX XX, marketable offering of approximately we and cash, In March proceeds. financials. on will stock, raising We

We operate to a timely the continue cash expect of expenses marketable FTX-XXXX and our from financial clinical strong hold. we securities projection our fund assumes position, and mid-XXXX. equivalents, resolution into cash, This to operating the

partially million primarily to $X.X $X.X XXXX the and the decreased costs offset XXXX increased and loss research million as million first of of XXXX. as with quarter of was stock-based Collaboration were $X.X the the for $XX.X for compared for due development Net as first the compared of General was $XX.X The $XX.X XXXX. XXXX, to REACH. of increase compared associated for expense. to for of first headcount primarily first administrative million revenue of million development $XX.X of XXXX. decrease to for million as the expenses Research quarter The the quarter for quarter compared quarter and advancement expense million $XX.X for of to XXXX. was the of quarter by was of first due quarter first $XX.X to first million the quarter million increased compensation were million first XXXX, $X.X

exploring position, remain on the path programs am clinical resolve and FSHD enrollment optimistic reiterate the We are track value XXXX of company's REACH solid clinical FDA on execution of confident that executing to driving Phase clinical we upcoming I to a hold. strong a committed X corporate value want objectives. our the provide are hold catalysts full cash the We of opportunities our and leverage complete second resolve the on research our to for I FTX-XXXX. is for working focused to and to remain in forward forward, to and creation. foundation there engine, our the Overall, half with

and and today's first promise Alex of CEO strength next Fulcrum with as the ending announcement XXXX of in is for great Additionally, of our position a President, future. the quarter

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

Operator

Instructions]. [Operator

first The Tenthoff with Sandler. Piper comes question from Edward

open. Your is line

Edward Tenthoff

need thank deeper you the a are dig issues, Great, you taking to wanted you thank I the outstanding order of experiments then you do sort need information for the XXXX Robert, in the sort thorough what questions to for collect what provide as and my update. whole to of see do touch take? into to list you with the they need the to or FDA data

Robert J. Gould

and we EED two FDA related that a pharmacological which get of lines inhibit One see is question around of reversibility we gene the those [ph] we're currently the inhibiting There's them outstanding results as Ed. Thanks, reversibility. with be them. the in non-toxicology They are Those changes, That's consequence the protein. along when primarily to studies course of we discussing are are expression XXXX. reversibility, preclinical, underway AAD well discussions the with a that with with FDA. effect of we'll issues studies

discussions outstanding issues HbF discuss that The evaluating that population we with most the relates from risk and regulatory us in populations define a to we're were the patient of defining population, we're benefit population. set that them in agency elevation to other asked previously, that active than that higher seeing. to The can has

gene know, that number symptomatic a therapy definitions the been of other whether in sure has cell small risk at some you treatments. and patients, there different the you at molecules or of are populations sickle are studies I'm cell As of looking

so prior And to thoughts population studies with can own care and integrating that benefit. that define along our most those we're

Edward Tenthoff

not if some to a That there? is I sense. makes may a there since really is of question, answer. lot one be toxic tough quick it this to last kind the relationship clinical finding, One Is the a that FDA concerns there and dose of may, concerns or has, a relationship dose

Robert J. Gould

about have in give us the been concern So toxicologic we've that not seen would seen that pre-clinically. any findings studies anything clinical the

update the the events milligram clinical studies, And And two we've benefit those the FDA sure during terms mentioned, part we're dose have no XX that preclinical increases defining they malignancies seen both of is be been inhibitors. concerned and serious is adverse appropriately the I ratio around engagement. seen that doses, malignancies that As the And have X at target was types milligram as as of well hematologic dependent we of all. see there in at PRCX with to risk other been want pre-clinically in clinically. that

Edward Tenthoff

the great some, finding job And may in just Yes. congrats of CEO, time company. a sort for Robert, stepping if I this take new helpful. That's during on a transition

Robert J. Gould

Ed. Thanks

Operator

Joseph SVB question. the for from Schwartz standby Please with comes next question Securities. The next

Your line open. is

Joseph Schwartz

was about you PRCX data show of malignancies relative order much. have to any FDA the risk? and help to hematologic help And them wondering hand, you risk how the get distinguish to comfortable follow as thinking so benefit patient are defining high that you're propensity on to other up, can I if disease? cell of population, that the in benefit a can FTX-XXXX in who Thanks XXXX is able with which sickle inhibitors, the

Robert J. Gould

question we've take the second let Maybe the defining that not toxicology we've we've the animal part FDA only of a on the would with is seeing the the that, studies first types course, gene been studies. to you terms I'll to inhibition of we're on data we've changes, effects Among HbF that gene share patient is seen we're asked and FDA that changes population. that done, seeing done, of Joe. expect then the the that's What as and Thanks to -- Iain HPV, is, of that the the studies the of able studies. first are really speak animal number there but also model obviously, focused the you with EED the in there's studies, reversibility a particularly that most gene really, expression robust -- what in expression

on. speak kinds so focusing to the PRCX what gene inhibitors they're of expression Iain, relative of to you defining other And patient those to maybe changes population. want

Iain Fraser

Yes. and Robert question. thanks, for Thanks, Joseph the

subjects range increase been dependence and where on that So and based clinical see potentially FTX-XXXX to data treated the a to into with beneficial. we've the able that we hemoglobin experience generated we've their dose going this that think relevant to in date, relevant clinically is be clinically

FDA in higher forward We the as on, been greatly about a we've replacement has therapies the will editing aspects therapies we in clinical the as including gene path cell these discuss the informed so stem studies. for field, we where been explore and with gene trial, and risk and think population our defined therapies, by populations those

Operator

next The next Kumar standby question Madhu our comes Sachs. for Goldman with question. Please from

open. line Your is

Unidentified Analyst

questions. wondering, cash are any we our Thanks taking is your for were And Rob INDs to runway? new there asset thoughts Madhu. given for Hey, this on just

Robert J. Gould

the Yes, and muscular development on leadership up of and JJ continue Rob. as we're not Officer, status preclinical those come and give programs, hematology on and we goes or on Jeff those as a speed on that progressing the or board chance has to Scientific non-malignant our the updates excited Alex what further are quite space status. are the space focused updates to to about the but of those a under dystrophy Thanks, areas, be programs actually their providing on Chief comes we've programs. ready the of by, options through we number he We're programs got Jacobs to

Unidentified Analyst

we that the for INDs? Is there a of expect can communication new of sort line Okay. time

Iain Fraser

speculate for that on time a It's little to yet. already Not provide premature guidance us on those lines.

Unidentified Analyst

Okay, you. thank

Operator

with for The next Please Biegler comes question. the standby question next Matthew from Oppenheimer.

Your open. line is

Matthew Biegler

Hey resolution to this for a time Rob do event? We line still on Thanks six-month a time possible on team. table the is just curious likely and think the a were still or XXXX lines, that you is questions.

Robert J. Gould

when it's line provide really that very, time cordial continuing them. the we're the dialogue very get ongoing definition conversations, to by dialogue is a in active the with resolved. And of been the will the we're we're As clear encouraged early with and a we've with FDA, interaction them having active. little Certainly,

Exactly guidance of a is timeline. don't that will our I So current assuming that to when resolution yet. get rapid resolved to guide want

Matthew Biegler

the with what are Okay, little and FDA. then that’s as you you that other eligible to are more they kind bit relationship said and patient cordial maybe of ongoing you to interactions into fair. define or just Thanks. about a scenes little or non-toxicology is the you a would your behind I wanted now collaborative describe you like a said, studies, dial this the into of How bit, the some very just working them, run have? population

Robert J. Gould

cordial extremely with actually it's Yeah, the agency. regulatory

with discussed, thoughts risk, our discussion the the It's the in around at of which way. real pleasure with their populations a provide actually with of we a and interacting open populations, that perception contentious population. and true various coincides it's As them partnership thoughts guidance perception not with them, been any on they as it's are

Matthew Biegler

Rob. Thanks

Operator

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

line Your open. is

Judah Frommer

Yeah. questions. taking good morning. Thanks Hi, the for

kind the there thus far risk study curious commercial of about any does clinical profile the more the changing from devalue it benefit here? addressable the just risk perspective, is or you potentially profile or data a patient -- of First, of is population have just

Robert J. Gould

that's in risk on In we all FDA studies continue of opportunity place to of study speak have a of will requesting we'll enhanced. it HbF, which let that think speak in first, They has the which let requested, hand, initial to oral part to for question, data have population, to the we'd we we've we us is higher studies still and activator speak date. at commercial the doesn't see Iain looking of But be to as robust has be an that, price, has value overall based small the I that part the your devalue seen the then that really molecule first pharmacopoeia. importantly, in the would do important these data to me think last that commercial doing. first we up more Yeah, an the like the defining we're doesn't increases HbF subsequent the and

As strategy of trials this kinds a patients. And multiple Iain involves you the multiple often an just population. is data know, kinds speak risk we've into first whether I'll generated today. to of the of higher and commercial let overall the foray Maybe applicability

Iain Fraser

Judah. Yes. Thank thanks you, Robert, and

is think all devalue X of or data patient in way we've sickle this daily. population So X milligrams, an affected to no I date the does our cell sickle at population milligrams, milligrams that clearly XX population. doses generated perceptions once alter at cell and That of the in

Some at and robust of concomitant hydroxyurea fetal seen the in them in also were that hemoglobin time, we've population. on increases

forward as data. previous related somewhat adverse any defined into to slightly don't population the that patient effects move different So think I there's or impact we

Judah Frommer

I relevant risk is in your still induction be higher Okay, cell potentially fair of to be is HbF going you'd arrive higher been that, risk to again, higher has it or editing, contention, assume levels population? historically, this this know that higher of approaches bar XX% against area might gene in that a population you're at up type induction therapy absolute sense an dosing if which

Iain Fraser

risks see procedures we bearing patient our move forward as way gene mind population, using associated going guideline up editing. define their and themselves. populations to as think as those with we don't are of I patient gene a in that they've therapy the defined in against ourselves Yes. that We're significant and

any on induction clarification on how So there's thinking we're don't populations. I for. particular of just how HbF looking we're much those there a bar think

the I XX% mentioned, number think thrown clearly, a that's been there. as you've is out

XX% the combination a benefit with clearly getting approaches using XX% absolute I in clinical or are being associated the is helpful the past. of terms or range either two think of pharmacological in of into genetic to that those percent

little seems to in higher getting to And that some XX% might some functionally beneficial think extent. extent be so and into be range clearly and I curative a --

response So the we'll move be evaluate up beyond on evaluating dose we milligrams HbF. to response as dose the potentially XX

Judah Frommer

Thank helpful. you. That’s

Operator

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

Robert J. Gould

us you Thank I'm we'll everyone And this and and Ken. Please all healthy. talking you, safe operator thanks stay to to all morning. joined be sure later. who Thanks,

Operator

Thank you for participating.

may disconnect. You now Goodbye.

Fulcrum Therapeutics (FULC) 15 May 232023 Q1 Earnings call transcript