SRRK - Earnings call 3 2023 | Scholar Rock Holding Corporation

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Rushmie Nofsinger

Good morning. I am Rushmie Nofsinger, Vice President of Corporate Affairs and Investor Relations at Scholar Rock. Welcome, and thank you for joining us today for our Q3 2023 business update call. This call is audio only.

You can access the slides that we'll be referring to on the Events and Presentations section of the Investor Relations page on the Scholar Rock website.Moving to Slide 2.

purposes want Scholar that prospects Securities Rock's forward-looking for I various be that Litigation begin, Harbor statements, about Safe XXXX. statements expectations, the Reform we Act to the we'll note making constitute provisions of Before under the Private of plans, and only represent as and of future of date. should representing not Any forward-looking as as views statements be our our views today relied upon any X. all our to the filings the risks.Turning SEC Slide of refer Please full to for disclosure I'd members during team call for like the who will of and available Rock to the Scholar conclusion introduce of be formal the be today's questions remarks. the presenting after will Qatanani, joined Operating will now Head Ted I Jay Jay. of CEO turn Myles, it by over Officer Scholar Financial Chief Chief to Rock; Mo of am and Research.I Backstrom, and Officer;

Jay Backstrom

Thank particular and you. QX year future positioned to are well call. We've to far Slide the for excellent very success.Moving believe business we about in and I'm third update our this Rashmi. progress and excited so X. morning, made in and welcome Good our quarter

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to present its from remain DRAGON who investigators, has We to to Now families, we begin continue and to are continue in, and met in this to the study DRAGON. December, objectives, the activities, benefit who we that from also closeout in on currently study.We their take plan enrollment enclose or therapy plan treat like opportunity and patients those while medical future to data particular ongoing participating meetings. in at staff, participated thank the patients DRAGON would

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broadest of programs. portion cardiometabolic who in of I'd of of the potential to an the apitegromab regulatory will to and is stages including the for Mo the Slide turn of experience follow-on overview the to neuromuscular XXXX. in the and company provide And XX. SMA be of benefit the over planning the be extend a possible.Now will ambulatory to in to commercial perspective, Head and under diseases. brings is X an to age allowing our by from success patient of we this approval, both Research a expect us and children program market.And additional finally, studies, apitegromab leveraged wealth patients SMA team in launch moving population commercial a like established Mo, the Assuming cardiometabolic

in team you quite time, for has cardiometabolic on for I through As remarks, Mo's diseases targeting anti-myostatin my been opening mentioned Mo will this working and Mo? research. some walk

Mo Qatanani

XX. Thank you, Jay, Slide and everyone.Now good on morning,

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in SRK-XXX, proof-of-concept a are of we in plan Phase XXXX. agonist turn trial the moving submission expected To is inform GLP-X We of initiate obesity The summary. development will SRK-XXX to Ted of II that towards trial for it over data in apitegromab receptor in combination mid-XXXX.Now in with readout IND to I XXXX.

Edward Myles

achieve to of We're significant seeing of execution our great XXXX portfolio to where has Slide positioned we're this coming to factors XXXX. out in appears areas, across scientific form exquisite Mo.Moving been cardiometabolic in selectivity patient targeting productivity of approach are as XX. Thanks, outcomes well differentiated. into important going our proving we oncology.We approach our to growth latent our the applying is so a and and highly is and to year platform of SMA unique be excited safety be selectivity believe disease

on last of approximately continue million completed runway business evaluate Our with the $XX promising expertise way second a with programs companies.We and programs the capital offering we that and portfolio. our ended other leveraging advance extends equivalents within programs, view funding believe quarter and the important strong to million. cash our We XXXX. raised for our infrastructure, SMA cash third netted by us we're $XXX non-dilutive opportunities October and well-positioned of puts that bring in capital development other cash we into strategic to financial advancing upsized partnership as of half an public cardiometabolic.Additionally, the position We're month, focused to myostatin potential in we as

strategy support study.Our and capital patients announced in focus, investments SRK-XXX, XXXX to a in on on cardiometabolic we advance we reduction DRAGON as Our SMA conclude newly remaining complete and allocation primary to includes includes of spend apitegromab XXXX, to substantial our look our continue and enrollment as program.

the capital over therapies.This core I'll turn excited inhibition, to With novel important Q&A. it our and serve myostatin promising the can we the ability future patients our are we operator, competency, hand, our in unparalleled the aligned start imperatives of remarks. and formal Operator? strategic our selectivity our programs to about with concludes underway now with so

Operator

the of Yee Michael [Operator Jefferies. question Instructions] from comes line first with Your

Michael Yee

what FDA and be develop. was is traditional path regulatory gain, that an Do how a X had I that also any your Phase SMA We gene ] proportion one the of SMA on the analysis an primary played is [ the therapy that? is at with muscle versus and patients?And younger study there X I appreciate younger for an for on that on then would there weight after -- more that [ questions, was there's just loss? and in how out, in about analysis probably drug have on based decision thoughts say pre-specified ] observing anticipate the hypothetical around the whether question, patients one wondering that you accepted Whether the And if to is the X study, and analysis pre-specified X has interesting trying endpoint believe think patients that cardiometabolic. wondering and you're to any cardiometabolic, the DMD you that happened to older? look if by analysis you obesity, I XX, to III, obesity is older versus

Jay Backstrom

question. the Jay. Yes, good. for is This Thanks

with Let me start SMA.

XX. yes, So, X is analysis the to primary our

ages group know, look the X But juncture across and you have the analysis include proportion we age We opportunity top-line groups. certainly haven't of cohort. as and disclosed this analysis the and that pre-specified our data, are upon older primary X, between of the the at who to

top-line we So, at will results. in certainly look the that

So, cardiometabolic, of the patient yes, to interesting very question the question. I population.With care think that SMA on took respect

of current the respect the amount think primary that for loss with weight to is the I that it weight loss, approval occurs, is endpoint.

mass think think efforts recognizing is evolving future this an But think are and I to need think preserve as demonstrate a I and we now critical inform that know that you function across the will or stay improvements I we a it's to outcomes.So, field, As in point think some regulatory at be and in standard in, need either I that endpoints. as we're will patient-reported I to move the tuned program part, lean will need the really forward. is others help muscle but

be can the proof-of-concept our we're demonstrating to maintain mass. on study, going muscle For preserve we that and certainly focused

think that's mechanistically approach and forward. proof-of-concept our that's going for I sound

Operator

the Our next of Bratzel Piper Allison question line comes Sandler. from from

Allison Bratzel

provide us better So properties and Mo. for on for seems some a Could you a first to XXX color major there. It format on distill go the differentiator just more different suited the XXX question obesity? in apitegromab, why SRK-XXX, compared to forward subcu just maybe of really is is

Just IND And IND a for to curious, and Any And filing explored? could gating on gating is and gives that, is subcu ready just go administration filing of mid-'XX? color confidence be that apitegromab is help then will what's helpful. what in question, to be us you also what something understand for apitegromab same filing XXX, would you to obesity? IND related there you've note,

Jay Backstrom

and a a get IND it's really rationale and we apitegromab and it's because part with to that is the I'm already the turn IND, new for SMA. the is, apitegromab opportunity delighted the asset.So of a over it and a question to cross-reference This the really I'll the Jay. as to lot required to what's the to one take get matter proof-of-concept Why division around apitegromab. of Maybe filed IND we question, together existing for then X-part clinical-stage And I'll of have scientific IND documents here the is putting current submit Mo. agree that

matter of dossier and of that regulatory to kind completing it's engage do FDA. So then the a really

into we work then details. this and talk IND ] frankly had area. required that work I'll for about let even enabling SRK-XXX Mo our And more maybe So prior going the [ started to then, announcing

Mo Qatanani

of as we develop of to and concentrations. a mean, population space. myostatin molecule this the this with an this is I lower the our bit understanding was for talking studies IND-enabling the subcu to mean, has especially as well cetera. property and a only It thanks, novel extensive we population.So amenable as myostatin based overall dosing. developed SRK-XXX. on attractive It utilizing enable [ highly all I'll I Jay. we -- mentioned, for platform. these as designed into of doses is of SRK-XXX studies talks molecule. cell initiating about affinity our that of and Yes, selective, start It next structure high developed studies. we have We're from vitro Again, year, in this efficacy as IND-enabling and higher et with the profile, well it going lower done we IND at to we've mouse when From subcutaneous all design affinity patient population.So these of potentially we line in doses concentration seen very you're and things, to with enabling get-go, typical models ] as continue leading forward and you move we've do also other studies, the forward the models, support in PK patient SRK-XXX high see to target has efficacy we're the patient to it to is developments, as and that with that looking the profile by

Operator

comes Our with the next from Tessa line question of Romero JPMorgan.

Tessa Romero

could. we if us questions from this X morning,

time change powering trial endpoint If the the have primary SAPPHIRE what the not, secondly, related what at before for question, from top-line are are tell next And can this? should us is, you the at around assumptions consider the using baseline year? test one how of as you baseline will the a to and results be you this months? you XX thinking disclosed disclosing we statistical about mean First characteristics the Hammersmith

Jay Backstrom

Yes.

on trial powered then By demonstrate powering Jay. I'll demonstrate not the hierarchical certainly change. From is the take X-point fully point to I put of following to But it. to Hammersmith secondary we're expect And color the able the adequate again, a the really kind that, -- and and to statistical we mean endpoints. the testing disclosed assumptions, we've questions. way have we for be that. doing to we this power So that just

publication So, TOPAZ at regard I [ SAPPHIRE the saw of think we're critically with do ] ] to that try ], in planning then time [ conferences baseline in that well we think as as is important characteristics, study.And to as very data Really Congress over about to our what think positioned plans. in Ally [ the we we're and replicate disclosure we -- to year. sort we it looking

come to cadence given in time not that, any to more likely but occur as the we near-term So disclose the the conferences. of medical

Operator

Our next question comes from Truist Securities. Devarakonda from the line of Srikripa

Srikripa Devarakonda

obesity. couple As SRK-XXX apitegromab and questions basic obesity, a in I develop you and actually maybe myostatin had on of

Some studies obesity. is that have shown myostatin in up-regulated

versus reduced impact? anti-myostatin? you How be the that And treat you with think where that where myostatin Just activity. window wondering potentially as of patients consider an patients how effective is And patients? how well the given of variability? there do is background optimal drug could obesity SMA patients? if also you studies this dynamic has been characterized could the Especially have show in in weight, shown Is you there you lose think these lot a do

Jay Backstrom

Yes.

see start SRK-XXX I'll talking maybe with just and expect about we what So obesity. with in would in to then clinic apitegromab, follow

that what is preservation, say, myostatin think increasingly I muscle think we've shown not believe targeting does clear that I very now, in which can result in TOPAZ the mass. we and is I study,

think in observation consistent there think TOPAZ.I across. functionally we've shown I is that

able good of certainly very that it is setting in muscle. the be normal do a and obesity should myostatin So target, have to where you

The interesting, I believe myostatin, in think that -- as setting. was is to up demonstrated that setting, fact this that's we'll we SMA is an in opportunity in think there their I have I that the case. So demonstrate the high just do signaling but

Operator

the line BMO Etzer comes Darout question Markets. of Capital from Our next from

Etzer Darout

development progress. are on and catalyst How during earlier -- if the to apitegromab color And plans? of development SRK-XXX, of the described I the indication? guess maybe with proof-of-concept steps on opening sort SRK-XXX, in bit on for the for you next the obesity you an on in the little maybe just I data the more update, of thinking given Congrats be earlier a what about will, remarks, maybe is you mean, of future programs And example? and then the program, size for lines maybe for opportunity RCC,

Jay Backstrom

Again, can Yes, XXX we proof-of-concept and so overall shown Phase forward. we just III of studies, and with run designed -- to-date Rock at think, we sort let proof-of-concept Scholar me randomized start we've that can are path trials. I

to horizon. the to on apitegromab Anything my certainly drive cardiometabolic with positions rapidly out, by broader bigger, you I for company for the we open near-term I hand, we're discussions right And really report in really the will, beyond certainly if for proof-of-concept trials. our our think driving to clinical But setting. think what do still how we think in data value, our forward as get getting drive that think which around can that we can space then clinic, so, approach, opinion XXX as proof-of-concept, up also to at proof-of-concept.And into to time so that's the really will get that that we XXX us about open I we'll and validate I

response group treat, a about apitegromab this see how thinking and both I data and this the at to out is try put level a XXX, that on our tough XXX.For the heavily rate really what checkpoint around, that's very I to inhibitor in shared of and to really a it, on pre-treated are I failed proof-of this Mo we're So to laid I immunotherapy, beautifully looking and through way to which major why really so you like treatment, we've place take that really have earlier such progressed so really conclusions. earlier But is therapies want in if much think reached been and particularly shows any up of think therapy, impact.Because lines sense. have patients therapy getting beat any concept, in the that makes those these color

us to Ted And begin logical really steps lay pleased of the Phase any concept kind today, with what behind whole that's really would and FDA is engage then to right for development in meeting, review further an and XXX.And be of the work to data performed. said with so next our of the as out DRAGON end frankly, them now way I

will. our it's It's what's on the done reset, is meantime -- you the in FDA met it's job, a on anti-myostatin look happening -- that our think down, programs. objective. XXXX interaction, its all in at but wind take focus with if its really We'll I

Operator

comes next of Maldonado the Our Wainwright. from line H.C. with Andres question

Andres Maldonado

congratulations on reiterate I'll that all the progress.

quick us question from one So XXX. on

response? So I aims talk goal and in question patient the that be, follow-up of been you ascribe variability to in tolerability which treatment-emergent variance loss driving that response. programs apitegromab, of next-generation this hone guess much the more of really Could muscle to the solve the maybe context about of patient safety in events in the the context, would has how And then are worrisome and do you combination? in given of the variation issue obesity of some positive,

Jay Backstrom

So, maybe a team with ] do [ we'll tag Mo.

some -- plateau of in range a patients think you loss of with the occurs I that sort be some. kind gets your the that a to variability agonist, GLP-X across well, see for question is current receptor do and weight

of lot think there's the a some ability it is I to of may a loss and but it's we've probably say the data, -- weight -- for affect doses.So, be think XX% the what, in very to at that between overall the I seen, that a as and literature review look there adequate well From fairly muscle things patient's related mass loss. that that. range, XX% get is definitely the it's starting it I my to points

we think events, kind related across. have on that should Hence, it's but there.And And mass, think effect I so really this, those we the emphasized our about proof-of-concept of consistent at point really really we'll to I we've thinking look which is interesting preservation are and our a muscle safety because of matters. that's studies believe again, your lean But focus matters. move forward. really to it It

use. been loss therapies, I but time, their continued think So all over about by been that effective, issues and they've safety have weight troubled they've prevented toxicities

nothing with we're to-date been the seeing the that these effects, we've therapies. agonist what warrant patient exposure.We stopping there is GLP-X past seen in would some wide think like has receptor I seeing but really what and are

is other on avoid where will myostatin possible.And areas, at into in that of conversations do and no offset muscle, coming if that's if any add-on the lean -- additional all the risk to mind, going companies really believe think a therapy to that's this that's into additional I hitting the myostatin of other of But therapy look comes some in you toxicities loss thing. negative of I like bring off-target So used kind effects, in mass, an good my that space, needs muscle approach only effects. our by

walked think, I that. Mo through

right, that's look some get start activin when shouldn't could bring far risk and patients see, seeing If approach. we may Phase when hit see in to really [ and GDFXX.So, -- It want to troublesome a on that at not happens B, profile. report like over with true any into also and you very, what TOPAZ, receptors years we our we're out potentially to you III But that that A I profile. things safety you risk-benefit you that with so time be We'll clean additional coming our have for obviously ] very data. that

I certainly we show that. think want that. to I the cardiometabolic continue with we're space, positioned to And think in

Operator

question of Rodriguez-Dumont Ernesto Our the line Cowen. next with comes from

Ernesto Rodriguez-Dumont

the regulatory progress. obesity follow-up Congratulations program for functional just I purposes. the on on endpoints

expect therapy? functional gains with be those hints do you you limit kind conjunction therapy identifying then there the any to also, to other to of chronic And So myostatin therapy be a to settings, GLP-X there their or the in program? the biomarker expect from muscle do in the some and programs obesity other will target changes functional that lead that of endpoint for a way show are programs

Jay Backstrom

Yes.

and SMA with and strength we're grip functional an and as So certain example, assess functional of as ways other get I and function. well, there's back IND regulatory included you're program. tailored think the -- FDA engagement the the we maintaining programs. in begin to you other to going and could preserving underlying were know as show measure other that clinical But would disease.There's measures measures the Again, interaction Those for -- a through specifically we've motor to we things. that couple advance our endpoints measures the agencies that with the more

also something life of consider. we that is that include There at can measures would quality that's to look measures. So

our GI doses. the at As possible I potentially effect for you effects do the to Mo weight that which by and showed, know and getting the with there think associated same approach on current lower are some is potential effect significant some have loss, that therapies.It's weight additive loss what on very have activity, intriguing is we

program lot So that. So, not this. terms we've there's around myostatin at I got ideas. around a really interesting clinical very, kind beginning the effort thinking -- our I of in of but lot to at And and really the a the mean, of the of chronic we've targeting got come, of deep efforts full ideas to registration.But We're therapy, More can out of of beginning very think Right? good then space. that's the we question. do mapping

at team. interesting rebound.If agonist. muscle some which potential of chances rapid As we even on and certainly that you is I'm metabolic the therapy since fact company, I to good restoring that from talking maintaining at we is, can't questions -- could underscoring is receptor I But the program, And organ, GLP-X that, now the [ exciting moved moving that loss take on TOPAZ me. for is progress Mo's a as as think data which some Really there have IND. the is address what least I and really GLP-X our by lot we very patients chronically. look joined with of at preserving clinical maintaining Mo current the from for showing team ] in XXX have therapy. apitegromab a reduced the lower for to have they've years.So a give excited weight really about mentioned effects There on it we're is challenge that perspective, earlier, again, know, toward research the our

Operator

time. further are over the to at There you. no Backstrom, turn Mr. back this Jay I questions call

Jay Backstrom

Yes.

driving team for So to Again, kind of through listen, rest pleased we reinforce, our progress. done. really delighted thank has the where and our I'm into you. XXXX Thank forward work I Very milestones. this just have of to running your some interest. really major with look year the And you really really with

that, we'll thank call. with close So And you. again, once the

Scholar Rock Holding (SRRK) 7 Nov 232023 Q3 Earnings call transcript