SRRK - Earnings call transcript Q2 2024 | Scholar Rock Holding Corporation

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good morning and welcome to Scholar Rock's Second Quarter Financial Results and Business Update Call. [Operator Instructions] Please note, this event is being recorded.

Before we begin, I'd like to point out that we will be marking various statements about Scholar Rock's expectations, plans and prospects that constitute forward-looking statements for the purpose of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any year-to-date. I encourage you to go to the Investors & Media section of our website to find our most up-to-date SEC statements and filings. event want be later to today's website on date. of you should reward recording will available also our A at a

Jay ahead. I conference of to like Rock. Backstrom, to Scholar go would CEO President please turn Jay, and now over the

Jay Backstrom

Good to welcome to Sandra. joining call. thank Quarter you Rock's our our of and On update. for business behalf morning, team, you, Scholar Second Thank XXXX I'd like our

the XXXX. close X. Slide then open study of will our from focus by Officer, our and of Mo my is Marantz, progress call Jing TOPAZ, with highlight upcoming program milestones in on provide our Officer, The up today's exciting development Qatanani, call review Scientific with a to obesity. in our progress a apitegromab or SRK-XXX data XX-month II programs Turning the After update summary I'll proof-of-concept Medical questions. remarks, introductory including for to Chief followed of Chief review the will Phase who an

and was and apitegromab to and investigators, At now incredible III platform. our the only our of of and allowed a for product, closer support our our results in has teams Scholar continue program Thanks SMA the remain report is Moving over XXXX, the team SAPPHIRE, The top our ahead as approach the clinical toward with the into Phase to commercialization, on to our schedule. SAPPHIRE, study milestones we to advance the goal we view study to their apitegromab for Rock, track dedication us on first few lead terrific all line a QX. participating designing to coming our key achieve are of months study in that registration progress made we're our on hallmark focus of time and progress half differentiated or from is track X. We've in away. foundational Slide of selectivity to families

potential make are and to has differentiated anti-myostatin that focused with medicines areas believe and can with therapeutic at delivered provide Our areas It fuel research time where we with our High-value those industry-leading portfolio care, living unique substantial and creating productive We we been continues advances incredibly is and highly our an new obesity. opportunities team in for programs potential. has of that deliver a SMA Scholar best-in-class possibilities to in growth. Rock. exciting

are for be We months a XX will the at to trajectory transformative. our point XX in next

to Turning X. Slide

reminder, potential potentially that based a harness our right allow TGF-beta hitting insights the the avoiding unwanted maximizing the at As growth efficacy. toxicities superfamily factors and to the on was foundation scientific by deep company target us time, structural therapeutic for of right the of

of Starting a in bringing back pipeline shown therapeutic As a and this highly innovative forefront we've X, with as receptor with Slide on produced agonist programs, best-in-class management selective myostatin anti-myostatin as With Rock into obesity. apitegromab target. and Scholar address the suggest robust to GLP-X associated differentiated weight loss potential our products. approach healthy myostatin applied reestablished we've muscle to was a treatment in SRK-XXX instrumental and in target our data the emerging the with neuromuscular leading sustainable SMA,

for immunosuppressive be first fibrosis, designed as structural deep the TGF-beta X for key armor a before. such done inflammation disease. SRK-XXX best-in-class the TGF-beta now latent monoclonal pierced has our with we immuno-oncology, our antibody X and uncouple applied our IPF insights riddle effects fibrotic pro-inflammatory indications drivers TGF-beta monoclonal the from to inhibitor the with for for pro engineering antibody been X to to that and are solve the SRK-XXX, have in is resistance. as of not programs referred For TGF-beta we in potential overcome to SRK-XXX the And to selective expertise checkpoint antibody

As and internal blocking antibody from to unrestricted next wave are RGMc, further of validated a embark we advance elegant excited capabilities, innovation. target research anemia our as our for candidate monoclonal to an we our development on evidence have another of we neuromuscular program

X. Slide to Moving

look been of We compared for clinically to focused our and endpoint the capture with mean meaningful the for change to to the placebo, difference therapeutic demonstrate score QX. at the X-point in call. upcoming in corner, is readout proof-of-concept SAPPHIRE optimized the Phase advance results was our clinical difference. and at meet top II in both baseline was the all milestones SAPPHIRE a to to pipeline across XX-month requirements developmental disciplined Hammersmith ability with reporting for apitegromab, statistically areas. share the designed from have just primary forward the based line approval in a around and efforts and Starting disorders the for significant out months XX neuromuscular to TOPAZ the to of data program, we excited SAPPHIRE key study to today's lead success and regulatory on TOPAZ we're on least

data months. SMA apitegromab, colleagues As for updated time, the seen over demonstrating more disease sustained detail, as CHERISH of continued The decline the you where therapy. study with at TOPAZ Jing in such sustained benefit impress, initial the and patients nusinersen-treated treatment presented from highlighting the XX a gains SHINE additional over is long-term months and clinical nature of considering muscle-directed hear noteworthy will the benefit to progressive when recently results options a the particularly from the through nusinersen by and show Finkel functional Cure need XX

to over and remaining new safety the and In no treatment updated safety on apitegromab XX% findings. continued addition the reinforce with sustained the tolerability of improvement, to functional data

into well neuromuscular for apitegromab to neuromuscular extend potential X with reinforce confidence study building and apitegromab SAPPHIRE other of our in expanding SMA the foundation under study allow those Taken data for the we and planning improve will together, lives as to in the SMA. serve our franchise, a children are to SAPPHIRE the successful XX-month as the further A indications. living efforts to as

II cardiometabolic healthy We top management. highly our area programs, months to updated study results time ahead have forward. randomized EMBRAZE, in approach our formally assessing and of entry than GLP-X meaningfully pro wasted in obesity announced guidance is the and selective we our and loss cardiometabolic QX we with forms XXXX. to blocking latent no moving weight combination in our schedule, less proof-of-concept QX to Phase for believe positioned of it into early apitegromab Starting with in contribute are ago, our a XX the myostatin the can programs line now and complete For we've to our enrollment agonist,

data data fat to date the receptor or withdrawal SRK-XXX nonclinical best-in-class body muscle As a with weight SRK-XXX approach following with of GLP-X potential to to ADA regain reduced mass continues preserving to novel leading anti-myostatin at loss add mass you'll Mo, generated presented The the mass of hear management. and demonstrating healthy support the evidence an agonist. from for increasingly

programs as for the checkpoint at I with TGF-X resistance. fibrosis, For TGF-beta Phase to and proof excited such we look inhibitor And IPF, where SRK-XXX our latent concept of in meeting. our steps forward and demonstrated we've overcoming of end discussing in antibody we about of proof high-value mechanism indications FDA for with monoclonal selective what best-in-class potential to IND. of the SRK-XXX, are next immuno-oncology, platform The opportunity exciting grow factors. in and these affords into pipeline. X the look TGF-beta possible become targeting superfamily strength key our to growth and our disease, to us drivers program are given to and of consider to opportunities. we advance It inflammation the see our TGF-beta is thoughtfully forward insights advancing has the

of have Oncology X innovation, the by featured validation Turning unique American as conferences our latent Clinical the and scientific proof American Society Diabetes annual most featured our science Association potential at leading further the therapies. being to was X as recently, of of Slide data monoclonal to and our our or signaling is last We TGF-beta alone, insights antibody, selective increasingly the cutting-edge months anti in structural the been community. research external best-in-class our X. In global recognized SRK-XXX,

milestones in time date Scholar we all are As top a milestone, shown forward for exciting delivered our major QX, our on and at looking to key the Rock. line results to have Slide SAPPHIRE next very on of XX,

I'm pleased who will Chief followed Mo an our who Qatanani, Officer, provide update development walk Marantz, from Scientific an on our over will to Jing the programs, that, through Medical team. us turn with And Jing? update call Officer, research by to our Chief our

Jing Marantz

we If can to Jay. you, Thank XX, Molly. turn Slide

study EMBRAZE clinical We including clinic. On obesity. the programs X data on Jay we June, data the oral an I at was program and the TOPAZ at SMA XX-month ASCO. progress on updated an of have XXX featured on from our focus our will today's that as have call, the session mentioned, program, update In in in

based start study to is on into differentiated TOPAZ, on from broad trial we approach we how substantial the the the left, patients. this across saw of targeting Translating SMA deep for our benefit learnings success. of a insights pivotal Leveraging muscle from TOPAZ skeletal slide To designed clinic, durable have and clinical is regulated. the muscle

on over Lastly, the with years SMA potential of observations exposure, our for with of study, XXX safety date to and patients profile use. of remaining has favorable patient importantly, with non-[indiscernible] XX% support over the been both long-term these

XX. Slide to Turning

look therapy. helpful natural Before the I SMN-targeted trajectory with data, it's of share the to at a treated take updated patients

from Finkel presented the As SHINE CHERISH the the initially study, The enrolled in Jay represent and outcomes to nusinersen. with Dr. patients trajectory patients mentioned SMA that meeting recent of treated long-term Cures lines orange data May randomized nusinersen. June, colleagues which

these well not underwent Hammersmith function improved those the not plateau spine and of scoliosis confounding to on faster time, to orange the pathology. abnormal that measured decline. curvature represents progressive study. commonly with known during surgery The an did line those of years, As assessments. graphs, to and patients. is subgroup have patients it's it for you of managed the the from started the After who Scoliosis Scoliosis Therefore, has represents surgery. motor line The Hammersmith, orange by Hammersmith is scoliosis all solid any impacts at disease as with surgery. be whereas to X a is dotted declined SMA the factor condition part surgery, motor started top who surprising clear see X surgery scoliosis that the function, initially. to that's over for

interval scoliosis patients data surgery. prior focus we'll on study this X The years in put in discussion the well those without to of patients. context, For decline. nusinersen reason, our enrolled the TOPAZ To average where had into nusinersen on of expected our TOPAZ the for things of plateau on to is highlights similar treatment Hammersmith blue exposure that area period shaded or that's relative

is within the than a that despite patients without declined increase. for decline orange years of line Focusing surgery, of natural X period, on of here a after greater key this point. progressive the scoliosis The the trajectory therapy, X score the with these course effective treatment initial solid that patients point highly over Hammersmith is

It function in patients our by XX motor represent and ages the outcomes TOPAZ and preview XX for XX X the So graphs updated these to data a gross months. the the on turning how the Slide SMA measured months non-ambulatory like measures for previously specifically pool from to that XX at provide Hammersmith validated left this scale subset shared function. motor is XX. Hammersmith a context, designed motor I'd X the right. to to we SMA on the to of function data, Similar bar is the months XX trial

surgery assessments for the As our mentioned before, factor surgery the is scoliosis confounding censored analysis. after surgery known are a in

treatment we course the increase this from their months, we otherwise knowing of months. would that at see benefit to was XX see graphs that can saw months at X earlier course sustained over XX decline is improve. of the function The over the these Hammersmith that that motor You journey, continues of to that patients was point reassuring it and see these of XX function motor maintained the

both more the continue X Turning strengthen to subset. to to in was months which to months, maintained the the for on The at and next XX and improvement X upper observed basin group and was time over over slide. XX the function. in trend RULM, Similar X XX in focuses limb XX

For to a or their as upper patients non-ambulatory, improvement is who lift living, activities pushing particularly lung such ability a function of ability important are daily to as represents our their button. continued perform cup in it

Slide XX. to Turning

detail belief that benefit, So and taken low rate, safety SMA muscle the meetings. clinical favorable medical benefit potential profile to with consistency sustained the underlying data discontinuation pathology. patients by of additional our has future together, with directly provide substantial plan share TOPAZ with at addressing findings We reinforces the to updated apitegromab to

our TOPAZ, of learnings the on for clinical SAPPHIRE Those pivotal XX. study Slide to Turning designed success. was

XX transformative population see population points. The and primary clinically on Hammersmith and highlight engagement at benefit mg as at ability TOPAZ TOPAZ the dose chosen efficacy statistically TOPAZ. difference demonstrate can a based with placebo. capture XX for same target The is reflects clinical the to key both least The the Hammersmith few that X-point main sustained you showed designed Here, versus to is the trial observed meaningful was from study SAPPHIRE on schematic, months the just to potential. difference endpoint and

of of serve, we improvement to have encouraged Importantly, with decline them to for a objective we're design with the both to study see to trying with trajectory able design. the clinical to clinical patients We're on the therapy. benefit representative despite our and success patient from the of This EMA with initial knowing TOPAZ balances and that broadly the key of hold. engage been aspects natural instead to SMN continues FDA the align time over be seen progressive optimize the population effort SMA

So patients. taken together, about we're medicine to goal optimistic bring to this our potential

to muscle significant and unmet significant about of has The with mass a turning profound the need. is to lean adoption our with key tirzepatide A on recognized that its patients of use. associated clinical Slide benefit brought issue cardiometabolic program risen obesity. XX. as loss Now has is Obesity semaglutide rapid

essential in Aside maintaining homeostasis. an muscle important physical glucose from function, metabolism to plays therefore, healthy levels day-to-day and of muslce lean living. role is appropriate And energy

great lean that uniquely this need. address unmet positioned with to company us team hard proud I'm end, initiate study expertise We're to we're of ahead deep incredibly To seeing our that in to muscle, EMBRAZE worked a the enable of As effect to schedule. enrollment in momentum inhibitor objective expect the top QX the and report myostatin selective of obesity. to of to setting next line demonstrate is primary in a in muscle study preserve our the year. of of now The

the the effect remains use. across potential that confirm metabolic on long-term to end safety number and supportive in function. and favorable, want tolerability in interested profile including effect of we're the lastly, also We that's profile obese a points population And physical explorate of the understanding

the primary endpoint or weeks. either muscle agonist. DEXA shown combination a overweight XX of obese XX GLP to to Treatment schematic duration conclude, scan study the plus are is of change combination or So of the by a from agonist weeks. here. GLP patients the randomized mass apitegromab in X:X lean for combination baseline at placebo The is

endpoints Secondary effect also at includes other a are PK/PD endpoints durability include weight profile get potential of can on safety function and to assessment tolerability gain. an words, In weeks of preliminary metabolic and we physical The effect. the the study weight loss so other attenuate parameters. additional included. Exploratory rebound and read XX

differentiated now to introduce exciting Scientific Officer, to delighted I'm this, the our With behind show signs our Chief you approach.

Mo Qatanani

Thank you, everyone. good Jing, and morning,

Next slide, please.

slide this start earlier. that and to emphasizes with made going points Jing I'm by Jay the

very clinic our to translational focus candidates. and is innovative in highly our therapeutic innovations across benefit producing Combined selective to a translated areas passion, success we expertise, have patients. and that these the platform have We many differentiated with

efficacy a Phase improved for safety and developed And we mouse function forward patients, motor myostatin you the SAPPHIRE in from saw QX. very took data TOPAZ profile, III selective seeing as over transitional XX map substantial months First, SMA, to in superb particular data heard it our our SMA trial where earlier, in anti-latent sustained look we with improvement in promising we to have we model trial and clinic. its our

solid checkpoint selective our to SRK-XXX, in tumors. primary TGF-beta overcome to with developed antibody X we advanced anti-latent inhibitors Second, patients resistance

the Ib types efficacy promising at multiple we where trial, recent clinic, promising We in where our shown highlighted was models took across Phase its efficacy preclinical our presentation translational it showed tumor showed in we in ASCO. to multiple have as quarter DRAGON

Moving in metabolic in anti-latent profile improve to its models. that the to obesity. selective and obese We due weight our in myostatin studies designed for translational SRK-XXX, preserve loss mass is induced multiple mouse showed muscle ability GLP-X antibody

this in we the advancing and be an forward data Association XXXX at clinic. that American IND in call to on into highlighting look recently will We Diabetes the presentation an and we shared oral filing

added differentiated presented the developed that TGF-beta matrix highly our when has we only SRK-XXX, but inhibiting cells. tissues, anti-latent in the Finally, selective, selectivity antibody X X of not TGF-beta of immune

This signaling, this the Given of differentiated recently mechanism XXXX efficacy and advantage lung, we this highlighted mechanism on safety in Time look to featured the We efficacy forward disease cover and potential and multiple of testing showed and the in filing publication IND clinic, was unique molecule translational unique this fibrotic in an fibrosis. and in fibrotic in liver including renal diseases. of models,

XX. Slide

Focusing SRK-XXX. on

scientific confidence strong preclinical during and drive in its management have loss. induced us validation healthier gives evidence rate to weight We that GLP-X potential

top preservation obesity rate shown of We during was in the by XX% a preclinically that profile, model SRK-XXX blood meeting this namely also loss, mouse reduction glucose this of leads in one Keystone mass seen induced have year. a levels accompanied translational alone. improvements This of in at on reduction area. lean treatment shared an was additional to GLP-X data GLP-X earlier with biggest That the in challenges metabolic solving

key an mass composition. GLP-X treatment increase regain We to withdrawal, and showed improved receptor of leads leading treatment of effects namely after preclinically fat cessation. following durability that in lean challenge SRK-XXX also with the attenuation of This to mass addresses treatment GLP-X another body

highlight We data today. that some will of

nonselective the that SRK-XXX the being Finally, is model, that in in diet to currently same we superior study tested a the antibody anti-active in clinic. is preclinical induced showed head-to-head receptor

in highlights loss patient the As in preserve is the management outcomes obese and potential during for of it ability health muscle weight healthier critical and overall highlighted play population. can weight driving better muscle earlier, to SRK-XXX

Next slide.

in incident rate durability key induced weight the GLP-X model. addition cessation to SRK-XXX mass loss left translational treatment is This of shows The treatment. another of challenge loss groups of loss, In during obesity loss graph ability semaglutide upon this mouse and challenge lean upon semaglutide loss the the during withdrawal. highlights the induced or weight upper in or mass of address of effect lean to on of slide different the benefits

can treatment the the expected. mass to bar, mass to led line. XX treatment during the as as in considerable green treatment of phase semaglutide see you lean days see the you semaglutide the led lean in the SRK-XXX Co-treatment of with phase, As preservation loss blue during can

treated semaglutide after day discontinuation XX following mass during for higher the In treatment. SRK-XXX leads SRK-XXX the the Co-treatment of significantly withdrawal, to led to you of versus when and semaglutide end lean as with if line rapid of along fat -- change semaglutide Cessation study. expected. study. as the can mass blue semaglutide, line mass and rebound the has alone the line comparing for attenuation day regain for mass fat line green addition, the graph the of compare on shows The lower green animals the fat fuel regain effect and SRK-XXX treatment XX with you between see

favorable slide blue Next proportion semaglutide In lean graph Animals composition as has after bar. left, mass the bar of versus in on treated the a cessation of animals controls treatment, SRK-XXX the end with the withdrawal see SRK-XXX treated you body at slide. in study. versus green shows This the the higher can the semaglutide upon the of

on induced bar SRK-XXX control green on the with fat a the significantly graph Hence, mice measures percentage mass, if healthier supporting durable to highlights a period. overall the potential has in fat the weight you end composition a rate the composition. regain mass loss withdrawal by key that a the in of but IgG PUR, lower the versus SRK-XXX more addressing more favorable and in challenge treatment a in with look favorable of body in body at addition, life, In management treated SRK-XXX more GLP-X the This the

percent model This competitive upon where data obesity we a of and the our translational nonselective is change highlighted investor module. in graph study lean mass versus the our the showing during event Next head-to-head is SRK-XXX in This in is profile of the semaglutide mass slide. diet recently induced shared we myostatin another a treatment. approach May,

significant can As see the lean to expected, left. bar the as semaglutide led on you in reduction mass, blue in

was liabilities to see and at antibody, bars you led as noting mass bars at kg green. in dose of of highlights worth mass with presentation preservation antibody. anti-Activin was mass of level As highest than X.X mg receptor with fat efficacy lean kg approaches. at grade. XX anti-active This as X kg lower we receptor of This SRK-XXX dose lean mass dependently lean the associated the only preserving avoiding best-in-class can the per with in low versus where achieved is for equivalent with the profile mg enhancing see dose the per treatment nonselective It tested a SRK-XXX. per and mass SRK-XXX the muscle doses can while you as competitive the loss fat the mg of see of as potential Muscle profile loss. dose mg significantly in per preservation kg, SRK-XXX in SRK-XXX doses supports that for enhancement we XX superb mass see in subcutaneous preservation of the Treatment also also

slide. Next

selectivity schedule our the and expertise GLP-X anti-myostatin EMBRAZE is context Day in clinic, lean has during develop well. is influence to of selective proof-of-concept announced test enrolling our to allows induced May quickly back the in Investor to and inform Finally, leveraging hypothesis in a We the clinical ability the weight the study. that that loss. and a really we demonstrate metabolic in we and our trial setting announced preserve trial test ahead our exclusive This mass our to anti-myostatin parameters -- initiated will us of already obesity we antibodies obesity with ability this are Since it to apitegromab hypothesis milestone to in the program.

XXXX. line So we quarter the second data top of anticipate in now

file the accelerate use XXXX. we an will and We to learnings IND SRK-XXX, our the development from where potentially to in of expect EMBRAZE inform trial

it to Jay. back hand Now I'll

Jay Backstrom

thank Well, Mo. you,

it is Scholar exciting So an for time Rock. closing, in

looking or presubmission advance all conducting EMBRAZE, met initiating our goals including exceeded EMA with we're of the prime formal submission we activities a key our preparation commercialization. our SAPPHIRE IND, That concludes for apitegromab, to readout next important in to have milestone European top presentation. forward time continue meeting for to to Rock. and line our a in preparation as SRK-XXX for great date, Overall, for as for We continuing initiate to Scholar

will Sandra questions. for call the open now up

Operator

[Operator Instructions] first We now take line will the Michael Jefferies. the Yee from question from of

Michael Yee

questions. We had X

in and First, SMA, obviously, to competitors' time. understand one wanted all reading I others you is on your are some their the pivotal Roche of I out the around study just antibody guess are also similar. reading same out the view I think because

there and your because and readout. both as have lean understand about I you muscle obesity drugs wanted your do you're if the also sema you're around same wanted that first, stratifying time. it just that those seen study or drug if readout, that on side, are to completely loss coming those any you to so your And will relates noticed in offering to And I wanted think believe and drugs effects. base tirzepatide or And the design do you I have are of your understand, how in eliminate I different case to differences for and to readout both the expect is know that with stratifying

Jay Backstrom

right. All

about upcoming the So ahead and first Jay. readouts. tackle the -- this go I'll Michael, -- I'll question maybe is tackle

space, their recent our aware that early III of the in we which the out opposed the I'm excited they to of study, as as really out to others particular, Roche, so ongoing. seen out Phase The phase. we're from that is report to most that in in report what somewhere is follow line beyond clearly, targeting our we've and top I establishing suggest -- trial, or to going reporting dose they line phase they're think top they're update their So effect XXXX continue

agnostic both be will they're to to interesting where it with to different but place. a them, risdiplam see nusinersen only studying clearly and think we're risdiplam. remind I the So results, in And

didn't semaglutide seeing And We're can answer, just study. or have the lean No, think on in an then effect we with I since respect proof-of-concept both. program, should interested to Jing going to on both. of by in be see, ultimately, amount effect we'll we be muscle really treatment. this interesting. I'm we but comment. And tirzepatide EMBRAZE, will to stratify think we We agnostic think, should preservation SMA for the

I at were impressive Mo that preserve. muscle cessation. maintain actually course, the And saw saw that is in you mass from because continued very did think upon then, data what presented it of did and lean a increase ADA show we we

we're reproduced I proof results concept clinic. be running will in think likely those the But again, of see. to

looking looking the top fast. glad going to and the The So line accelerated we're forward both. forward impressive, to We've top reporting telling enrollments results. you is I'm team for right? our line

Operator

Securities. Nierengarten We will now David from take of the next question line from the Watford

David Nierengarten

apitegromab It's SMA. and on

is effect historical the attributed confident from children aging? data, clinical you be that therapy the differential in look the background Or case are any when should base longer-term studies? from to there at the you from Just the still

Jay Backstrom

David, given can see this wants think safety as historical in be not think Jing Maybe that and right? I improve program, with data. beauty, apitegromab to function, maintaining for treatment I daily I'll children start and the with that it activity for the tolerability and is maybe about if about if the hopefully fact, really optimal of to of continue actually and that children further. it as growth would living enhancing through maintain and they these I and into we interesting, see muscle SMA. be it's adults young the add chronic their development and -- And the physiology get growth

I combination. So it's that think likely a

is of the to add that maintenance encouraging. see what decline Jing very, don't the over very very wanted see as particularly continuing time, treatment sustained Jing, impressed you nusinersen. said, the effect with we to we're I light again, fact if mean think you to it's that that? continue to good. the that we're I in I and And know,

Jing Marantz

Yes.

covered it, then I Jay. you of think So most

pointed really way Jay to the of of One adulthood. things effect So been this I all of that regulated has a from literature life body large add. think throughout development out muscle that the modest is stating a the to couple speaks mechanistically, as early through there well, that mountain

And so that effect, has in true no been well and of animals documented that's negative an had human. myostatin application dented and in has

X point of addressed there that's is Point probably question, is, number that I Jay one. is number component the think, a So development.

really patients. similar Finkel showed and patient very you important what's I the and presented population is However, that colleagues that Dr. are

TOPAZ patients and you duration at prior for out also status, in things patients patient very of enrolled patients are of therapy the If these the our look type baseline -- the similar. of those point sort functional of that all the age,

to otherwise very patients expected we're compared do, hold. benefit see these the what So to are continued to encouraged

Operator

the now from Markets. will next We line the question BMO from Capital Etzer Darout of take

Etzer Darout

Just a here. couple

to you standard any or we guess, potential of the productive you access data, dynamics care? if your to focus the SRK-XXX approach line for SAPPHIRE are data I you more anti-myostatin? the you of kind then you just sort of as As sort advance sort of continue it And look these for sort and assets relative portfolio payer for use to insights top of as will hand, TGF-beta wondered kind in across fibrosis had of thinking updated apitegromab of about on on need Or develop conversations? how of grows,

Jay Backstrom

Very Yes. good.

we preliminary the set I there And that previous in precedent prior the continue payer current is the let start with do course, shared interaction. line of with top So to interactions We've that do we've payer and me think calls. therapy. had that.

ability enhance that some think gives into in So functional I and gains these the the and that. value to children of us insight add do

We've you robust Of And we what do I us data and I've the seen have of the with is interactions heard to need said interactions is It's further the But expect repeatedly recognition pleased information We've so us this talked in course, community far, rich history. that and the the more everything feel out, line data. that we'll seen progressive to point show course, what believe data these that. care. looking bit preliminary to the to the I physicians, nature SHINE. think talked to plateau the it. CHERISH discussion. expected as We've payer nusinersen additional that we And we from really long-term and and that from top very interesting. it by and for from inherent But we've effect. gives fact, a an continue reinforced from disease. more sources with about we've been there And that around children as did updated give a natural

able to you of and that's been with that I we're the the what will payer that data interaction. kind think sustain, into put TOPAZ, believing. that the where evidence translate well And So maintain we've with sharing the we're if together behind

the now Mo And I've I then where with respect love to love opportunity time the been further pipeline. about talking and done really what really call pipeline go on spent have I years. some the here I the advance. pipeline. to and this finding and on next, to X team going it's

see we to I the be anti-myostatin a programs very I think really the lot feel highlight not good think me. spoke very into SMA interesting that. extraordinarily to I've data TGF-beta about I obesity upcoming strategic X touched our prove to And and on, when decisions go as good but will The about. we data, to are started data spaces the that

further will coming I opportunity great that to our pipeline, I as holds think growth into and value. a believe what the behind phase more enormous invest of and really a we're company have promise more we

first company, for very us exciting. SAPPHIRE. It's dedication forward and for So readout more on listening, It's with clear good and to very to to get and the focusing team year. very foremost, very be come really close us. myostatin to their focused the a again, the go for time pleased as really But to everyone the to we I'm deliberately

Operator

now will of next the line from We take JPMorgan. question the from Romero Tess

Tessa Romero

So like months, observations then the up clarification X or morning. of you has curious that you any of done XX needs particularly can it us for us ask the in frame? was then second X kind you percent And we you which I patients been? what Can this the September give XX on just group? us possible had be from that have timing XX that just has XX to know range about let to tell age might of with could a a like XX% the about data what of if more group? first result? give least be this us of think from decreased inputs at meet you bit question in time that to on was as time? a increased the is how line any Can question can the in seen distribution the I XX and think if on to wide last And time improvement Just And longer-term X-point for there between the of be us what out, top patient months. presented And Hammersmith? driving on Is I little SAPPHIRE. would

Jay Backstrom

Yes, Tess, those. Maybe this tackle is I'll Jay.

that with the And really mentioned as really a distribution, medical we to the have percent updated change. go detail, achieved good at can a around granularity X-point data. Jing we more meeting call, You and the the the the much questions on have advance about XX-month that where granularity plans about get that

when the distract from beautifully for incredibly We just to because recall, want SAPPHIRE. to planning do getting critical but important of They're I a XX-month do us know asked tests. is frankly, priority. has important, really the at in so them on didn't to data driving planning. data XX-month this were about shape list team we now. SAPPHIRE Just been priority SAPPHIRE year, an We delivering us. toward just to were That's data data. it beginning set. the the took good it is we We not able now, were were the

to update us allowed the that XX-month So data.

this of a said, a Jing snapshot. as So bit is

Some more of in more give get detail a this front we as to We'll come. meeting. you medical

then we've checks that and be second that data And database to the and lock registration. have team classic where part the and clean they're meets a not get sure of there. distracting to the for that going to how got this the is from goes we quality your question, the all the

some established time So there. there's in frames

of to to just be driving able with say I'm give get QX, to allow date. us get to as us right? in So And from team this as high-quality stand really to quickly energy I data. call, more there me going this They've behind any the to on is timing granularity These possible the the a to not on driving. hope integrity job teams you the done fabulous are really

We are driving.

We delivering. are focused, we're

test I So remarks. as soon, my we'll we as can soon said get as it in coming

Operator

from the of from James. now question take line will We Nachman Raymond Gary the next

Gary Nachman

that's So and if the only you case III after but the that do payers. secondly, TOPAZ just for some then And important potentially SMA patients include study with really SAPPHIRE could Phase is data of help could think months, these from XX positive, compelling pretty longer-term on label the also this the obesity.

EMBRAZE you'll in for of able quarter the If how next but second be? have you data what gating there soon the be the from it's after, file to assuming factor XXX. I'm IND would will soon year,

Jay Backstrom

Yes. Good question.

know, to everything that the and available TOPAZ. FDA start, it's to the contribute find risk will add your have, interest that set safety meaningful interactions, patients data to the I we the data perspectives. experience be past optimize And essential to those meaningful entire data for for it, let can data, we because the these think over I comment the And with safety that and Clearly, is from be regulatory in variety first that and payer think that they're piece, help will and the to tolerability expect continue about review. think benefit. When support EMA publication in you as to time, about additional one profile to for do I opportunity the patients the long-term them data the for for We a think review like that the data sustained data a we assessment. will with -- of From interactions, and we me for to So you application. to move Gary, important. perspective, about the payer regulatory in and for benefit to think, our my is that them of that clearly we that benefit see looking that's the to you, point open do

maximize these interactions. So we'll those in the And definitely SRK-XXX then that are parallel. program, in again, from

the his work. driving Mo the are really and So We're IND. team IND-enabling tox doing towards

beginning it's our targeting sometime space, we're we of year. discussion next on our and the that but to on done, gate of into maybe So And kind track the obesity it. and is that We the schedule. at journey midyear get as shared

which in although in and team report enrolled, out So helpful support advance. which likely there'll the be those be our the in with include we'll speed target that will pretty strategy, certainly EMBRAZE data concurrent, has IND it's we'll will with the the interactions. It

Operator

next the line Srikripa take now from will the of Securities. from We question. Devarakonda Truist

Srikripa Devarakonda

you're the if can semaglutide, also rolling target following started question, the just color, this. you study It bit -- have certain and with on patients? looks on for given earlier little was obese targeting an tirzepatide apitegromab. up it profiles. matter? Does than And like of the a slightly enrollment be they expected. if to And a just what overweight ratio between the and seems different provide patients possible, know wondering better I for sort obesity more you I seeing you're

anything there about distribution tirzepatide trial is terms into assumptions in powering account So you semaglutide? of between and took the

Jay Backstrom

Yes. Good question.

I'll trial start Kripa, to then So the Again, have sort Jing about this it patient in that's design, proof-of-concept I'll and and this with a add being study. is included population study. Maybe the Jay. the talk of is on

So of able kind on lean preservation to mass. muscle we want that of really an to effect be see

decline with both tirzepatide and differs to you semaglutide see have which point, X. the between think that effect speed the your the think I I

there would of be groups detect so you ability to when of think on effect either may difference be in those preserve can But And terms that. some our the helpful. seeing I

the the really that study, believe EMBRAZE that population that from for be came expect by the the we So in -- then earlier we that we demonstrate but but study. able the question call, didn't Jing effect stratify didn't we've to included considered it, And about the on we to see. we we'll

Jing Marantz

Yes.

population. obese versus particular the for not a overweight we strive Kripa, the ratio for So did

the a to question the as to of about GLP importantly, lean kind ability the And of tirzapetide. concept, is the I ratio patient just the to in the your study of objective muscle Just proof quick context really semaglutide second between mentioned, and have really to assess agonist. as preserve of different

fundamental think X. we is there's to do view Our between a difference not those

have you are reduce weight similar between GLP-X similar study study cardiovascular the ability If there's muscle that. I line overall the of degree both greater at the but to of think the X between in the risk are reduction, of quite loss and context look both reduction, Their degree very in shown kind proportion weight of X. -- a profile has the safety of tirzapetde probably the

that more So preserve set in GLP thinking is primary agonism how up view, that it's to really so we that. -- then That's about trial the it's our muscle weight, lose can we along basically those and the do lose do the objective to pathway of muscle. but and counter the

focus. our that's therefore, so And

Srikripa Devarakonda

that. And one question Great. more just then Okay. follow-up on

proof-of-content a is this -- you So study.

or Lima in develop update baseline terms DEXA do preservation to to is Your Or do think you you muscle in of at expect change drugs? from you're primary need have FDA guidance all By how scan. this you you endpoint to ready trial of any by point change in a pivotal time the time?

Jay Backstrom

is this Jay. Kripa, Yes.

going you it's loss to really into take field. strategies to think healthy have management. regulatory as will weight to And I change FDA be an to -- be going the I although, think to consideration begin tends delayed. know, answer is think the be evolving I

we the FDA. the whether feel said very as about we've from we So to good greater be to insight Having get date forward, seen. remains that, go received information I

through foreshadow need metabolism. data, at showed walking We that. gets as preserve team look muscle. we really the from -- to that benefit. a the we benefit a are we're doing and his at where what we me ADA could nonclinical take Mo of is data we It's take effect glucose The starting what think to think medicine, this measure right? clinical you on Mo see and I in I the demonstrate just clinical addition. And That's ask have I'd an lean you on clear to excited

upon semaglutide, withdrawal we muscle fact, rebound. we and weight lean fat in maintain the a the fact, of increase lean In and muscle the rebound, mitigate mass

you're or So the such side critical muscle, think been really opportunities not combination we've in which role saying those in position, plays I sustainable, lose maintenance on what lose to think, to is it we weight expensive a can I and strategies to at where find there trying seeing the metabolism.

Operator

We the will line from Frahm now of TD Cowen. take Marc from last the question

Marc Frahm

maybe to follow A answered. up lot beginning. have But Mike's already the been on right question at

on profile they're I'll you're succeeding able other the to SMA. likely the between are guys Just are how Or and likely of trials subtle these more different question kind that market be company safety big Do just really after signal then profile? follow-up you have that significant these to to of you overall of that. lead for in and outcomes is terms do in market And just very in trial obviously, efficacy in that the significance the able to differences to think or a might each aspects to cases? of differences terms that be statistical reach to molecules view completely designs so to kind that outright of those failing

Jay Backstrom

Yes.

Okay.

to want I I the Sorry, were it Marc, the this asking is you're I programs. got the got talking profiles you in Because Jay. a of differences side so sure Yes, about the correctly. distraction, as question, make

each different terms seen In expected what's from to profiles. of be the of

Marc Frahm

to be? it's subtle it's very important going significant Is how the is or just data do as view failure? is more you it impressive enough going Or it success to to -- to be that

Jay Backstrom

interesting. start you strategy, right? first children foremost. plans Yes, it's Again, earlier our everything I as young and that, about really we mean I think and if even treating safety X, we ages really trumps. have adults, said move we're a we to and under comment into about made about So

to to safe got therapy. a have be able You've

for to growth that be across. able And to be regard got demonstrate be to been it. to and development. It's able got able we've without You've administered to administer

really is So paramount. safety

but each like is there that think the I these on side. our differences safety strategies, in terms of strategy of I

there. start me let So

efficacy other a was with to nusinersen. we've reason. see scale I think Hammersmith selected It against is are motor setting, scales. respect I I forward standard again, we're for honestly, quite for what's of used gold standard, our gold the data There from kind looking reporting what the out But the really to believe field, think that to measures. this the from and

that's So I think important.

be I tolerability, critical the it the to as a tolerability which safety, linked and from think our but think payer commercialize. to ability back is interaction I about will

is little, recall make That doesn't CEO was very setting if what still duration time on. have impressive. drop because and previous any, a that treatment is really of our forward. know patients in I from comment that at median he would from data the TOPAZ a the seen the are going We I

programs if about But I'm implication think doing not the really that's sure think I to other So you true well. the that, we're we'll just I we're see. going. across where like

I the like then agnostic And treatment. is, that do to final point we're

that players. several important. think is established with is an really I This market

choice, an therapy, of really see not as background play I gene to is forward because think the to terms really you'll think to in the and forward. opportunity, a place that going that We I upregulating just opportunity show have maximizes we're SMN neutral therapy this our good go of in

results. to tell where together, able to frankly, like And about things So are. the looking I you you really feel being all forward we top line those good of look

Marc Frahm

you trial and really it are else ready revise be BLA? Okay. that the -- that hoping just just obviously, side writing other to that That's positive. what need just in-house? up? to things to it still Is kind Or you then on to file helpful. the come gather need will be And we're Can may data the there of CMC

Jay Backstrom

Yes.

of you done. their this and sections you can is that and included. stuff. be runs work that to right? the they're being CMC XX do. work PPQ we've Those complete that in all CMC sections preparing reports a is and it The written, getting progress. to is The nonclinical team applications, are As The the over There work been is, with is months, that XX BLA are know, honest, most beauty last doing

application, modules. that raised by clinical So populate definitely, things earlier really then working that's that are we're Topaz but going There that running evolving. well to team is the we was will beautifully. our in include it as data incorporate. is as Gary, some the to And The data

have We there. put in enough to

all in, We submission. getting Head pleased that ready to a coming of he's Regulatory together fortunate and So of have were outstanding beautifully. us just of is job really done for a fabulous very come and

So us that, rest Jane and team hustling up is are flying. team Mo's Marc. trying and the are to of with keep

Operator

There at are questions time. further no this

Jay Backstrom

going we're us interest, all appreciate your today. you you close thank listen, to call. joining I Well, and Thanks the for Okay.

Operator

conference This Thank participating. for concludes call. today's you

You may now disconnect.

Scholar Rock Holding (SRRK) 8 Aug 242024 Q2 Earnings call transcript