RYTM - Earnings call transcript Q4 2024 | Rhythm Pharmaceuticals, Inc.

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Operator

Good day, and welcome to Rhythm Pharmaceuticals Fourth Quarter and Fiscal Year 2024 Earnings Conference Call. [Operator Instructions] As a reminder, this call may be recorded.

I would now like to turn the call over to David Connolly, IR and Corporate Communications.

David Connolly

Thank you, Michelle. at here Connolly Dave Rhythm Pharmaceuticals. I'm

of slides can those the section ir.rhythmtx.com. you by participating our call, Investors website our conference the of be to on going For accessed controlled and

press our XXXX press year and release issued we available results morning, release provides our and financial and our update business on that a is full XXXX that This website.

on Our agenda Slide is X. listed

North are Officer Vice Head call the today Executive David Hunter of and President, Europe. from Jennifer the Chairman, Mazabraud, Executive joining Meeker, line Financial On Chief of Chief Executive President; Lee, Head Yann on Officer; America; Smith, our us International and is President, Vice

that those result of Slide forward-looking reports Actual forward-looking discussed on remarks file call a statements. contains expectations, indicated statements the most may as factors, which quarterly important On in you annual concerning materially differ by our constitute I'll recent SEC. results X, these from this those including plans remind various future and with prospects, or

any statements. addition, disclaim not views our of representing as any represent forward-looking update as our should of upon relied to such obligation dates. views We be any subsequent today specifically and statements In

to David Slide that, I'll turn who the call will begin With on Meeker, over X.

David Meeker

Dave. morning, joining. Thank for you, Good everybody. you Thank

earnings So advance we of strong fourth in preannounced quarter the JPM conference.

genetically into incredibly to signaling oral cash rate the million II proceeds from have Phase evolving. study. the about gross run in our bivamelagon pathway we enrollment through entering are impairments in $XX is points, in which having well our We MCXR XXXX extends program, raised We feel opportunity how completed multiple daily and our driven recently capitalized, ATM positive inflection XXXX

longer. precision a will these getting many steady number we a at percentage continue BBS, disease the high can unmet a a times, of need, or medical where diagnosis, a accelerate opportunities there to As pace grow availability of like patients of undiagnosed patients is therapy where the for a significant and highlighted opportunities decade to have ultra-rare

taking is around to opportunity BBS We continue U.S. and business opportunity a could alone. shape, will the we internationally. The expand build

are are RM-XXX. once-daily making compounds, the we pill a weekly next-generation bivamelagon, oral We injection and the about with subcu excited progress our

our next seen. not but preclinical in cardiovascular to and an patent better more to activity significantly appropriate is no Both the there convenient, generation Now the activity of efficacy no our in to improve robust sparing, hyperpigmentation drug. have goal opportunity MCXR extend models. is whether make products to just be opportunity actually have life Both on a models so remains and

in fully the bivamelagon to enroll The quarter, third we'll XXX Phase this study and II read and program out is the this is beginning enrolled quarter. quarter

III will it and for Phase trial and questions that out what BMI? a What to on design So setmelanotide the continue now first and change familiar the acquired We is now half, X slide of reading a guided quarter HO, timing is percent the of readout trial second of we our readout. can be here. be more in the the in have the receive in we is to expected the specific Slide majority

consistent have remain what updates with we Our said.

original They has incredibly I and X not remains open-label rate do below assignment. because XX%, rolling over an dropout only over wanted Patients start rolled Our the extension. blinded patient family. which And remain think patient the strong metric. that into their to are to

BMI, blinded it is in clinical to regard a the Phase percent trial. III With change

always trials across some being general run designs, can imperfect. comparisons trials have comes made GLP-X. this have obesity reminded the people a We that change with of the similar current is space the be out of server These however so around version percent in trials, many although

at look different. in apples HO, setmelanotide is biology comparison. is fundamentally it we oranges When and The an

hormone replacing are provide pharmacologic is GLP-X The intact hormone. doses to a on approach the alpha physiology. melanocyte-stimulating deficit in We top of

Our same the BMI weeks. in and change the we endpoint, X-year-old patients, at XX trial measure percent and patients enrolls XX-year-old

II lost the Phase youngest trial, years patient oldest our XX%. the weeks as approximately In XX BMI of patient XX% X age XX lost approximately old, whereas their

Now it the percent smaller XX-year-old but on basis, a patient actually lost a more weight, was change.

predominant data are the the preapproval patients and variables impact all do primary. date, well our study, data, which said, on which we will adults, of Phase was which to So access French final will early the was That numbers. in II the all suggests these pediatric

and full subsequent enrollment understood. clearly completed regulatory will which The secondaries from in and States timing data independently pediatric the out pivotal break out filings so the Europe. top United be line of Japanese there for no cohort XX recently read we The the and adult patients, so is cohort, on of impact will story can the patients,

in out of first Japanese 'XX. the We the expect half will read cohort

the to Europe. enable data in or where there certain If prevalence a hypothalamic and successful, us than marketing will obesity seek these in U.S. is tumors authorization of higher Japan brain

reminder to in XX,XXX on patient population. reflected U.S. X,XXX Japan expanding are injury understanding significant, who pool specialty believe in X,XXX of There's patients the with We endocrinology and represent HO We be forward appropriately additional population The and diagnosis point. diagnosed is remain which a to an concentrated the X X,XXX more. the to the Europe, hypothalamus estimates, is affirm conservative. with to our so range to the patients undiagnosed. our this may Slide we similar A on in were patients call patients pool and where not of continue in learn to is we in of that look and original they and likely largely clear a the opportunity slides

to Now the path future clear. is Rhythm the building of

is also syndrome but there a can Prader-Willi work the little why half biologic disease, genes there's We pathway. disease. we'll The with that an can more M&A a driven are XXXX, MCXR as and agonist out the which out explore to trial, sound MCXR of in rationale of challenging further trial to genetically in have a enrolled, be opportunities targeted. impairments, is work coming read first Daybreak

we and patients in escalated be and escalated you trial Phase as the This milligrams X day, milligram plan to initiated, quarter, up XX Prader-Willi, to is a II used dose dose will that at finding study as we higher X We to as signal XX-week new than be evaluate they'll first open-label enroll syndrome. dose patients to years in the in attempt to to significantly know, have Prader-Willi setmelanotide old the XX X a tolerated. which

at deep with site under an syndrome, forward U.S. in investigator and that Prader-Willi you conducting look are trial updating a progress. this we to single on experience So we

as exciting opportunity injury the an related emerging hypothalamus The other pillar, to is or failure of which to develop. incredibly that to the is hypothalamus

lead congenital the patients first developmental XX on in with are acquired on associated enroll We XX-week but setmelanotide's treatment, which to independent abnormalities involving and patients of in syndromes the first ways may congenital many the tumors track this more patient substudy XXXX. learning HO. activity injured in with forward an to HO there injury, brain, related to We be and their quarter of the more patient clearly are may we the look with about hypothalamus have to focused leaving area

next-generation work XXXX. one with not with supplemental indication which doing or anticipate our patent if expansion of extend much, the all, compounds for we both Finally, lives, past

We II the and and of HO quarter, anticipate trial half XXXX. readout have to acquired the in enrollment in the completed Phase second this bivamelagon data we be

and with patients HO enrolling this acquired quarter of begin will end trial data the we before I the Also, a Part in aiming readout the of are for Phase of RM-XXX year. C

readouts, prove addition is be some for to summary, year a in could Rhythm to execution was of year trial which and critical XXXX So in XXXX initiations. transformative of a

I will call to Jennifer. over now the turn

Jennifer Chien

David. you, Thank

in for the was the XXXX growth with U.S. throughout a solid year. So consistent year sales IMCIVREE

years, working have potential patients than fine-tuned care an them we with their to put Once we on we to give system patients, accurate help future the is and support in will therapy, years ensure X to work launch, our to maintenance and launches. place from process and More also on providers therapy. Over have therapy. and access diagnosis health the find

quarter-over-quarter. were New prescriptions fourth quarter received consistent in the

We therapy. and are support to tailored our patients patients and in maintaining to of success continued enabling families reauthorizations on seeing benefit approvals and receive are a long-term

increase the quarter, incrementally positive strong fourth patients a on was attributable combined, the growth. the number to therapy reimbursed number a in when added up Within of trends of to that

wins the level on program free from to some switches later-stage reimbursement we approvals to saw steady of during therapy. For who in quarter. are from discontinuations drug for and our appeal from patients Several addition fewer the scripts therapy reimbursed reimburse example, received,

resulted taken which categories, these their none the a in of quarter. each change when as of quarterly it significant a own on in metrics strong out While stand together,

label approved BBS POMC to Turning to pathway sends years message due differentiate a with Slide a obesity PCSKX root include of children to expansion helps these rare age strong X impact cause that FDA as LEPR young us In or of the as This or approval for diseases. XX. December, deficiency. MCXR the IMCIVREE and

of early young health X. critical early develop long-term treating left health lead better obesity hunger, severe age age childhood to Intervention be to at before and onset treated most can mainly insatiable a the Obesity this With complications. and obesity patients outcomes. and as is can

means for Ben We patients excited what are expansion and like this label families. about their

have when X.X And is he until now its with and child BMI not trying hand. impact is IMCIVREE was young was This trial know he X of X knew his right. mother then, a XX.X on was XX. biolilic Then do when from not she pediatric who example not revolve their diagnosed something seen did now was doctors. around had enrolled went outlines the it, LEPR old, III freedom years entire XX snack deficiency doctors His Phase was on story by and Ben in he was family. more a food, positive Ben, and family lives in though years food. to a even constantly his commercial up that making Then our he for his Even different therapy when old. approximately

Moving to XX. Slide

positive are ongoing. and III research preparing a for to insights outcome next physicians, and potential and payers teams hypothalamic launch. Market the Our patients obesity our Phase in is families trial in from gain acquired

groups progress advocacy executing patient and engaging We submission efforts with our details with with We engagement you as and potential to forward sharing are look more teams XXXX. launch. physician in actively FDA are we

the turn call to I'll Yann. Now over

Yann Mazabraud

Thank you, Jennifer.

growth pleased are for region the year the XXXX. with We in and XXXX fourth the for quarter of international the

patients BBS named or access and sales. in We have for achieved more with access outside a for HO countries than reimbursed the either XX IMCIVREE POMC, U.S. through

HO centers and to the the with paid our remain the PBS BBS In Germany, access with established growth is we region. mirrors launch for for France, in launch programs Germany, expand treating U.S., and consistent number of well BBS of steady now pattern main patients. drivers continue BBS and early the POMC, the the

contributing POMC and HO for France paid paid both approval early is BBS. also the pre-EMA and early access the program programs for access with

to setmelanotide. receive patients We or patients hypothalamic treating continue these from programs, the responding under congenital in France either acquire positive to obesity specifically, are feedback will physicians well

than in in the and new the our authorization U.K., announced for Italy. to including an pediatric growth rare for old a in general in Spain unmet rates in prevalence like X led rest case, for opportunity. significant reimbursement Turkey, a is providing greater Also, BBS as U.S., POMC partnership young need, we many and there patients diseases patients of are marketing Europe, us countries, years incremental Today, as strategic the and the has where the

well formalized working began Our disease and known and MCXR experienced Pharma a to partner a a experts pathway us. as years this Trispera with is new Trispera consultancy disease with We rare has Solutions Excellence and relationship recently few Center partnership. of very an is Turkey. back them in deep community entity

the and a significant certain U.S. are hypothalamic has prevalence neuroendocrine but in opportunity in greater groups and not to least, clinicians XXXX Japan as precision among anticipation region, year served advocacy medicine capita unmet of the acquired a with across obesity Hunter? IMCIVREE we scientific diseases. Last patient or enthusiasm foundational in a access a major a than in Overall, to experts, for build represents in recognized Europe. IMCIVREE and international beginning per need Japan, success met in real out and Rhythm. with for expanding medical operations

Hunter Smith

Yann. Thank you,

common of year-end the and cash sheet, Slide in of $XX start QX into during our XXXX. approximately offering of the program to proceeds getting ATM our XXXX into January Before million the with at of want We Rhythm results, balance of XX. of X.X on or quarter shares continuing I fourth equivalents. through price under beginning $XXX.X gross sale financial ended stock $XX.XX cash a XXXX with million and equity raised weighted average million and at-the-market

$XXX.X $XX million hand million $XX gross The on cash of in gross million January. about QX, of proceeds includes the not but proceeds raised on raised additional in

cash HO. data Phase II readout level past planned the pivotal obesity readouts, of in HO We of into readouts and expect data Phase the both and and XXXX, fund C to as RM-XXX to hypothalamic be data the Part this well well milestones III as several bivamelagon and study meaningful the I operations acquired including Phase sufficient trial of for

frame. as We from the also within anticipate data as the well from data III site trial M&A time Prader-Willi Phase this trial single

in in to We full came global million $XX.X for for from million global that unaudited snapshot $XX.X Let's full sales of in at preannounced XX. of the of now revenue review sales full as and preliminary $XXX.X January, million IMCIVREE Slide million and $XX.X year XXXX, QX reporting P&L year and IMCIVREE from year on XXXX. revenue net fourth the the the quarter compared

Cost U.S. revenue higher net net quarter to for quarters product decreased in fourth $X.X inventory percentage XX.X% of X% fourth fourth sold to at third in XXXX. the paid IPSA. versus COGS COGS The versus in largest the or 'XX costs produced both remains the with in the the million quarter Gross the IMCIVREE, of XXXX. revenue XX% and on approximately of remained quarter quarter. product we capitalized products sales of consistent was XX.X% associated during about third net of in as of and component X% of due royalty part of revenues a at which

quarter-over-quarter, due to RM-XXX II costs ongoing associated experienced quarter expenses $XX.X the bivamelagon X% which year-over-year Phase fourth million primarily in Prader-Willi, a of and for we trials acquisition related in and increased due costs to in the quarter were setmelanotide with quarter. bivamelagon. Sequentially, R&D this of fourth new expenses The compared increase trial to $XX.X was the R&D the began million XXXX. to increase

QX, $XX.X the fourth headcount quarter quarter driven primarily were the increased marketing to 'XX by for Sequentially, SG&A almost expenses to million 'XX. $XX.X of compared as for increased costs. SG&A and million expenses of compared by fourth X%

quarters. XXXX, million, the approximately included averaged QX full the outstanding $XX.X approximately prior X quarter quarter XX.X compensation the operating for million stock-based almost and has XXXX. fourth of XX $XX.X For of shares for the in $XX compared used to expenses in total quarter. million weighted average last operations quarter the Cash year common were in million in of was Fourth $XX million million

dividends this quarter per fourth share Reported loss from GAAP share a preferred the net per diluted EPS for $X.X accrued $X.XX. basic of And was and million. of on $X.XX includes stock convertible

quarter. $X.X ongoing this will reminder, million per be a As accrual

the broken on are XX. from Slide quarter highlights Some out and year the

As fourth for of of million. revenue in was U.S. $XX.X million XX% quarter the that, $XX.X was revenue during mentioned, the accounting revenue from of quarter. IMCIVREE sales global QX

U.S. of year, the revenue. for accounted sales XX% For

inventory the As revenue fourth calendar stocking reimbursed Jennifer in we than was U.S. and evenly of unusual number year. in our quarter-over-quarter spoke fourth inventory an patients of Such by the of during detailed $X moves which the in on pharmacy. increased not increase growth January, by sequential to, greater specialty split quarter therapy the the million quarter are

$XX.X Chem. acquisition global for to stock-based that included the of bivamelagon $XXX.X totaled for from in U.S. the million Our well GAAP in compensation as million, $XX.X LG as XXXX consideration rights million OpEx and

OpEx guidance low of at non-GAAP year, at in end $XXX to the $XXX $XXX.X which the is million, our For million came million range.

non-GAAP giving Consistent but with we on years operating not past, are revenue expenses. guidance, we are offering guidance

expenses to of and overall For for Japan. million. to treatment for of launching increase million XXXX, non-GAAP in million United comprised $XXX obesity we the SG&A we XXXX subsequently of and million SG&A in to due anticipated Europe for $XXX R&D in OpEx $XXX to expected increases in non-GAAP million non-GAAP organization, non-GAAP The of $XXX approximately as million IMCIVREE OpEx expenses preparation hypothalamic $XXX is States anticipate our in and in grow the $XXX

business to growth is the and in programs. in of Increased value. setmelanotide level and anticipated HO, this of in spending development new bivamelagon shareholder due R&D will long-term trial trials affairs RM-XXX and investment expansion ongoing medical and Prader-Willi believe our We of our drive increased

point, final Chem This we included guidance, but out This fee in as second the bivamelagon. license XXXX cash not in payment the XXXX. guidance XXXX. pay recognized and cash this to One in million will OpEx for expense our R&D is $XX was tranche in final July as amount our is of LG

to I'll call hand . With Thank that, back you. David. the over

David Meeker

Hunter. Thanks,

So we've hopefully, We're a commercially, up position established to what our really you've we're putting We've and on got significantly what's for strengthened potentially in -- And open really heard opportunities significant programs, us. which a in growing. is sheet, balance all developmental execute Rhythm strong in exciting front is us. good of us place.

So where we good are. we feel about

open we'll that, Q&A. up to With it

Operator

Wells comes Fargo. [Operator Our Archila first question Derek Instructions] from with

Derek Archila

Congrats the progress. on

Just X us. questions from

Phase the could you and just trial the adult in ultimately, same if the HO, could of the SAD And separately? update the going half MAD of with be to the whether us patients first, IMCIVREE pediatric will second that share RM-XXX split in be you then comment I C? Japanese of you on mix portion Just III kind cohort? you're remind wrapped just Or study was Part 'XX the on wondering into and will expect

David Meeker

Yes.

it don't an SAD specific breakout. So have that for independent likely to plans present I as we MAD it, the piece think is of

trial. will you're is C I assume concert bit what there. in SAD do So about as you no can opportunities, you're of well that right, probably say little of are that for things we'll as But MAD it's a Part with that progressing, long that news, going

we -- far good where so on feel we're out XXX. we data with the coming So about that's are

ped did Phase the more split, not we in regulators For the HO III, adult the trial. wanted -- adults

the XX-XX. are split not is number or So roughly I We're lock we participate. adult number the on centers we to and same. be multiple like that, adults to even have about did of the it be peds said, got exactly cap The focused XX-XX. very additional to going to But

Operator

with question Securities. comes from Fernandez Seamus Our Guggenheim next

Seamus Fernandez

you're lot in X,XXX to would the market than a the at sense get or emphasizing patient quite how David, success, opportunity So Interested population, being sort perhaps just more identifiable the genetic patient that uptake even see of of the to BBS HO, range. firm a XX,XXX in with population. you population

So better just interested get there. understanding a to

as pre-existing thought leaders spoken discussion some space, there little because the a then a the bit obesity sort of hormone treatment the point a we've And in with around with set of dynamics setmelanotide. replacement contributor potential as of is of

like Just the given Phase have interested on impact the results an you in the study? that to HO patients about if Phase direction any mix that you III of trial could in III feel know talked just

David Meeker

the Seamus. understand quite second didn't Sorry, I there. question

replacement that outcomes? you're in variations the So impact saying their endocrine

Seamus Fernandez

and set the the of that point More there injury. magnitude prior a could timing was benefit that on of injury, it the sort limit depending that of preexisting to

David Meeker

I question. take that's let interesting really mean that question first. Yes, a me

really in the was and difference underlying When HO way this right? problem again, agonist, understood MCX took biology where And the who learning, melanocortin-X I between pathway. really in And prepared then my worked, of what essentially, this remarks drug, through patients, had the -- that was. think replacing patient major number took I we nothing into fact, have entered nobody we response, what result small suggests one an the deficit. ourselves every we're signaling which we're highlighted GLP-Xs is a the a in HO, is,

be So is restoring teenage we They examples boys, gained our which mass, they to in lean physiology. normal mass, gaining particularly in of did. theory, a expect sense, have well. And in where them muscle you'd that,

physiology the idea you and there's And support are. restore then are going think that so to some you for we're your I who normal

So to drug back and you you to of to obese we were back your drug a developing you were your In tumor, theory, general for could where if is not get our prior your obesity. question before, but getting insult.

your I impacted, deficit so that or true. think normal likely So that be underlying underlying aren't is physiology to

we've with where like is to what internally like think and and get were some I about experts back our we'd we before you have And of the evidence injury. hope your anecdotal is you talked and this to

health. reasonable and would of a restoration I be a that think very that's expectation, huge

So answered question. the that hopefully,

the the I also, all And managed the Okay. are this of reason that pituitary this. majority, mean, here. they we've We're we of said, where of they health like. to different we're also need XX% is that's What say side along sure as highlighted, recognize with concentrated patients, expect learning appropriately vast as Jennifer they're our uptake their guiding then saw is These entity. not fundamentally call early we're endocrinologist and aspect that, do on BBS. insufficiency endocrinology by yes, being point, managed. is Yes, to uptake make as we And concentrated BBS we you own -- and we than -- an different call than can is and research have these of patients a so in XX%, meaning the this be we especially endocrinology -- because know, in point the with

higher patients, percent diagnosis. So concentrated of number larger

will that lead faster of a uptake. All to

counterbalancing write Now that your that, this pharmacy. you to go local always isn't and said, you true, which script is a

not -- the So drugs we're have authorization. go challenges have. like We'll do it's reimbursement to the launch prior that price. some to we'll changing through It's still we have not going

said, what still go accessible caused from a to more are to at expect going us carefully. by specialty that are little from it opportunity. the maybe been bit we easily different that payers to a and priced so But payers -- be a be well. level those initially, to So factors be we've balancing are where at it's feedback encouraged going That looking

Operator

with Cowen. Nadeau Our comes Philip question from next TD

Philip Nadeau

Two which is, In management the from from BMI your remarks, the to well of the expect in pivotal is which investors well. I melanotide do sure in saw of the us is That's more hurdle Diving to question bit well prepared we're what going as a deeply. the you said HO primary data get into to endpoint. I'm you on does think that regulatory spectrum is XX% what reduction, the loss, guess, kind first spectrum weight is the where extension X% you well. in the question. long-term

And Hunter. then second for one

terms down points of therefore, In build QX gave to absorbed to a out trends, slowly? in channel like and trying sounds QX is going be modestly of $X and be the just was it you the in put because in that Or assemble that destocked data more likely Is there is quarter-over-quarter inventory million possible? QX. to

Hunter Smith

So take the this say, in I would the second first. yes, I'll question And anticipate to we occur quarter. absolutely destocking

can be for when like especially a have I little how Phil. it us, always companies all you QX mean, specialty know you a pharmacy is, single So lunky.

David Meeker

I try Yes, fundamentally Phil, think a world to been to world we've by of give this you is as to to comparisons coming had some of how that examples are challenging. we've question those why so conditioned large as it's the out understandable, and highlight and percent does different extent in GLP-X reason, it's endlessly all and The what the and management and a this sense I our tried think I change. highlighted, question

of know? for average -- know in a both early then are experience that X well. at number in who's what taken this said, of small -- responded everybody patients know We we and Phase we II plus France. drug France year the groups. those in XX% And numbers the that that Essentially, in do So the and has

have his of know right? at of XX-year-old percent light basis who individual larger a a overall had lost we launch also percent more you the absolute -- the lost lighter gave a We at BMI XX that patients subsequently, weeks, but I on less a weeks, who XX much very a but smaller and amount. He example

So those predict. are really just to are hard variables that

adults We have a in of larger trial. number this

they Now the have well. adults I fear. have no respond looked And like

HO. was an I out think of coming that part incredibly powerful

is, project answer disappointed works. hurdle. to XX%. what to is likely regulatory to your So this I beyond difficult incredibly percent have change becomes for question be less we've is than We'd the see about drug said we're -- long incredibly the much good I mean I know I X% more what feel ability that. this

Operator

next question Genuity. Our from Whitney comes Canaccord Ijem with

Whitney Ijem

I wanted the the in the Seamus' around I HO. around guess being some just on up been that follow think, have question numbers on data. to call, prevalence on quoted, earlier prevalence And just commentary of I largely based craniopharyngioma

is any there's know there mean work. I work -- I So that

the in might form? prevalence So including of can terms you that work speak to the other come any of from etiologies, ongoing the congenital

David Meeker

Yes.

So -- it's a work in progress.

fact which looking good, we that we Early initial we're put work this the that least that -- we we're things, is of at. highlight, of I You're hearing will numbers modeling, kind at out increased aspects confidence that or supporting that because those around say believe.

There's the contributing not clearly to population. assumption are cases. many which significantly tumors be true. that with We was entered the necessarily That's this majority of more to craniopharyngioma going

which coming work, so ultimate numbers. this out And that's takes we'll early where of see of the claims some that

The a these patients other We thing was we taking our initial injury. post XX-year did period longer. with live calculation know

you're don't live life patients. to age XX, have So on XX, if tend longer much die you the your at -- a tumor not you at you

part there's model the we have see and that I may on. also, the seeing respond we well, conservative that been think So that adults of then

numbers and So get those are look to continue come we'll we more update as things confidence will out there. these at. And we

highlighted -- gaining I briefly say, and see I pool mean we're was today, weight, that and head we so injury can too can the if likely that case more which it's and again to I undiagnosed talked have But measuring here nobody's about acquired report confident clearly last way remarks will a I a and a the population hypothalamic what And you thinking nothing a There's thing maybe the undiagnosed it's it's have an see. quite case in that's today. early system that's obesity, injury start you you am still what we prepared to world is a than suddenly report. say

Operator

Our Ahmad next America. question comes from with of Tazeen Bank

Tazeen Ahmad

other order I want David, in right more what about them to really it doctors said little they'd drug project your But time. detail see to it what you'll what results this a have not than be thoughts that ask in what knowing active using being to I about you about about and away wanted HO terms you to is to over able like an for that get works of -- know told loss in patients? weight percent bit

second then my one. is And financial a more of

completed do You ATM, rule it's readout coming you doing out after raise this HO out, set up soon. positive? a Would have cash dataset but that your data assuming reads very you that

David Meeker

not would general, What was if was it in I'll Hunter it we that this. Tazeen. direct a nothing Thanks, be doctors are XX% doctors the the let telling works had question, course. it if is XX%. XX% and use use if of answer it it XX% would we're use not in you But or or us asking Yes. very honest, you to that question,

quoted will some tends know all doctors XX% we that case and have Now series the to have about be what the XX%. some and I've response and out a of there's to with than HO response small GLP-Xs that there quoted reports GLP-X, less we said patients of

an this difference and where doctors anything are experiencing. for think certainly terms will it and to understand is like actively following today indication, experiencing experience is labeled are indicated the be date, and who them knowledgeable they're in doctors I what this the approved, these is, amazing patients if this of with our problem and for So

doctors to consistently on. the in I So projecting I I just idea when want better can't of patients majority know incredibly question. in trial, give the outcome -- of does work who a think think no it's have percent clinical treating what than past. But are it's had are I not course, they've work what wrong really difficult. does and They the And focused it these it a

Hunter Smith

question, second for very you your thank to question. Tazeen, the and much And

high data, the vagaries that it we runway we hearing, we're, same First prudent to to we're to of And broader want it We're be to position. to here, -- as uncertainty have all, And in the data. market we following the I'm well where given now flexibility. and that's of and as this take readout a absolutely data we the to coming very reactions operational the extend have to very the period up. thought market happy in reiterate sure of benefits conditions that do our overall a time, think I confidence didn't at very, raise you're

program investment Our is RM-XXX, associated and bivamelagon for expansion. years generally the coming indication an with

the So which will those driven by raise need to more and exciting. to is programs, more the be invest need in success largely

have in, to we're said where so where in we to following no, we the position not want position don't a in Having that. we which money And today raising a like would do readout. we're are that, or we done say, raise anything a be

Operator

from with question Joseph Needham Company. next & Our comes Stringer

Joseph Stringer

on Phase XX. trial, groups to also the criteria Phase slightly older olds it's than Just patients where compared and the year label II to different then inclusion including open the HO Phase age has X III III, X

So describe III patients in primary of have overall impact terms each effect of magnitude variability? of inclusion on can both the you in these of groups the could BMI data Phase what endpoint,

David Meeker

Joey. down thanks, X. None, meaning I and that to No, say that we had Yes. cautiously, patients again,

and I is between think the X X difference negligible.

X-year-old. as as well than TOS. if were we'll And average, the at data were you of I on older And so -- patients was some XX insult. years reported think out French significantly we XX, had had age do adult XX. they they remember, were these helpful. from They a X-year-old mean The incredibly X out the their the

we you've to MCXR agonist your hypothalamic got you're helping an And people. those So out And can chance works problem get that are. of you only It with how provided can it and living first intervene we've concept like the you're that a X strong post far in with isn't a it X once injured with for if living to MCXR this impaired support years doesn't this it. the good signaling that looks if you really matter injury. in,

just patient on change sort we're end I is up think don't either their how was do. XX on more That could drug to the any on percent more thing in focused to they patient BMI. to know, in or what I have I'm offer And who people again, who the or not going because only track that had spectrum took by is did So weeks. who going impact of XX% about lose least are decrease going the weeks, to the Phase a thing other the another to meaning XX% appropriately one II did of was And be? remember XX at trial, lose the X% well

it looked a had good at fat in increase XX% they lean decrease That mass were least in like DEXA percent patient, the -- like the XX-plus scan, their responder. you you looked When they their and body mass. when a

arguably, So of our from a standpoint, one that health was response best responders.

more allowing this and So restoring a that do. replacing do we're again, normal what back you to to idea physiology should you deficit,

should boys teenage mass. differently. on behave will Other So muscle lean put groups

Operator

Our Gershell Oppenheimer. comes question next with Leland from

Leland Gershell

We have X questions.

Dave, identified of label younger in And what therapies. of I patients want you component then the since the also are you to extent ask, uptake. look general those -- your to the historical but ages other enroll review to to genetic commercial things days, I ask, similar just want just with to First, the it's extension a fecal early is see to to just could congenital as wanted patients becoming expansion maybe HO, versus

David Meeker

Yes.

can X-year-old comment on and second Let opportunity. to first the Jennifer me the X- then take

are brain, here. congenital how syndromes who brought "depending following the present". not like patients HO with about they of learning area on by populations variety And this us different we're a opportunity. they of was appropriately, So what observed this And there's to doctors again, is and the developing

of suggests the early those of data that that clear have France who percentage respond very obesity, it's some there is these patients and But patients in some to an agonist. MCXR

and just which percent obesity further, hypothalamic is yet, with literature So the who is patients that obesity then of our one understanding very helpful that, have is congenital impairment. X step not and to the these syndromes here challenge related of

and the before course, birth born it pituitary you are is here like congenital,they unlike Their an have challenge of with think not injury picture. they where they're a insufficiencies start I these, It's time. at And full-blown over emerge emerge and setting after, over time.

is today's So that syndrome and in equally they with on it's obesity connect our wouldn't think this likely have world, you congenital you that related obesity might as can a necessarily you emerge, at looking patient doctor have obesity just top. imagine

challenge. that's So our

said, Like of I learn next lot think there's enthusiasm a the we're I around going a to lot this. over year.

have yes, we to patients. again, little would to love like And I these HO. a something have bit to talk them. do As it's physicians, Yes, I for

said. So very early, as you

Jennifer Chien

that Another the for really opportunity would came point the One, an QX, the the in potential indication pediatric late quite about expansion. I say year. in

the So in have impact of released. an revenue it didn't terms that outline we of the

as around can patients X-plus significant And I these of to we Ben's have my years to be outlined forward, age. this happy for the very our impact Moving for option during an story are background, patients. as

in going to terms like market a increase impact revenue. of significant just opportunity feel we it's on or significant be However, don't

as actually additional for the ability in more our differentiates get shown be -- really population are need to plus. piece patient an of this of a able approval to We to further the old is by have X-year happy terms information that which

patient moving and on consistency that what really IMCIVREE in we're of differentiation forward. focused our population So is

Operator

Our Ding with comes Dennis next question Jefferies. from

Unknown Analyst

on Two from Anthea for is Dennis. us. This

the And pivotal anticipate throughout any impact trial to And trial, then Phase from do GLP-X of see the in a the II, trial drop-ins adult how mix? efficacy about III as and On read-through bit efficacy? upcoming melanotide could enrollment well and Phase are terms little as Ho use baseline? pediatric the this if you at allowed talk you you that on

David Meeker

Read Sorry, -- the what's which . say it about trial? through For again, II the Phase trial? biva. biva term in question

Unknown Analyst

the terms and terms of efficacy read Yes, do and then adult setmelanotide also from of any the enrollment you in pediatric in see mix? through trial

David Meeker

Yes.

arm, patients our X X blinded for So that's a XX-patient so trial.

and restrict older. obviously have insight we at XX the We it. It's no didn't efficacy So this into point.

We which we're So need it's population. still part making trial, slightly the part our a next developing we'll population. the great and we're be we of will but older have formulation don't that for -- on, of biva, that progress pediatric

the [indiscernible] can say about at I work now all that's the So patients mix of

prior If had of and/or with previously loss regard or GLP. continuing trial, a the III GLPs XX% in not they could to GLP greater been had they X about to the on Phase weight have clip, continue so patients the months. were X% in a of

weight that you that stable new the trial, to cannot you be and but excluded add And a was during so loss by didn't had And on medication it. protocol. happen,

Operator

Ram Selvaraju H.C. Wainwright Comany. question & next Our from comes with

Raghuram Selvaraju

experience that of patient what U.S. U.S.? you've the regarding place for expansion compares wanted outside on IMCIVREE congrats And how the longer adherence acquisition, and the ex comment all seeing just in ask the a patient could recent little the dynamics label impact been and U.S. to the progress had specific I in on that market you're label you recently. if since for has of that to more bit

this announced, is to that ex-U.S. this Trispera have extent significant likely question of it think to we if to is likely second the ex for a in a Or or future activities? about And to Is template be is, context? extent kind future model very Turkish relationships you BD what inform a relationship U.S. individualized your the the to much territories?

David Meeker

Yes.

opportunity. and then, you see about the So your Yann, we how just can -- distributorships to think -- of and international experience X. the course, commented U.S. the on on you how impacting experience Jennifer and overall X partnerships. that comment with here

Yann Mazabraud

you. So thank yes,

So pediatric Europe. in

of and U.K. Spain, have we than younger So patients in Germany, more to the that. in we'll X in a treated now in and countries, see France, few continue

on also significant Jennifer for and said clinicians impact for the is previously, the be revenues not impact and patients caregivers, a for one. the will the the As but significant,

nutshell, that's can what a say Europe. indication So in in the I about pediatric

David Meeker

And then you on maybe this. build can

be and where country the where practical is so additional mean think can leverage we way need And our about to then and go I we direct to help. smart distributorship, we by about general country. is approach it

that So in might that an bit of and behind represent the how use forward. distributor our or of decision maybe just a little going thinking to indicative Turkey thinking be

Yann Mazabraud

David. said, you as exactly, Yes,

-- by we country. our think I choose So approach country country

Of course, these happy some in such is the territories in we with what Genpharm we partners Middle that believe to where we Israel in the the are with in direct have now the partnerships. main Turkey, European specific and done than expertise been and we with have with medicine that countries, we And very for better Trispera X ground we with have in do. East . say

Operator

Our JMP. Wolleben with Jon next Citizens comes from question

Jonathan Wolleben

Just during due did in if III titration the protocol, AEs of BMI, some reduced see the or reductions dose wondering the period Phase you're a confidence which in But to Phase everyone's AEs, II, so period? if regain allowing set drug us who dose dose we for patients wondering the after XX-week gives the effect.

David Meeker

Yes. No, good question.

escalates to tolerated. as X So is the set dose protocol everybody to

to acclimate can again you little a truly do If having you that then can't hopefully dose most longer are you decrease. dose tolerate patients it, trouble, you stay then at and/or if dose escalate and

allow does tolerance. So to titration it's a dosing paradigm that

world majority to is X. desired get needed to X X, everybody's the X, BBS this the HO up little weighs and between somewhere has a the going vast of Not I to end go patients been do X. and know the to heavier out I to question a dose Now at there. think

X in HO that's will so ended remind far. up drug, one couldn't something lose only trial trial than And yes, had XX% had quite and more that, had and the patients, to -- who tolerate but the the stopping I'll individual you, fewer we've we like firmly uncommon. dropouts may again, we've X be just in think in I it range. the But we

the that seem an So in Phase to III. doesn't be issue

Jonathan Wolleben

one more, And may. Okay. if I

-- between gens difference a less about talk to you at if there. about efficacy You the you MCX and activity is there's TBD, can talk Wondering X little at the a about can MCX. going talked between setmelanotide, those receptor relative But of bit MCX be activity being next and having the specificate say the the kind XXX you bivamelagon candidates.

David Meeker

Yes.

functional setmelanotide So we highly and assays in similar. XXX in vitro -- potency looking are at the

in was results potency comment was assay. -- bivamelagon We actually But a assay it so on that I don't now. same running the assessed -- for can't It We full those now. within comparison. I are wasn't that done done head-to-head. were different have as the

What on milligram I an like. preclinical can the milligram drug, oral base, it's and -- you've per say the a is data and and obviously, absorption again, availability got

a on are of understanding. saw models, so bivamelagon, set extensive that the of being of factors, lot is got as basis similar built XXX. higher of -- or with milligram trial your a an And setmelanotide in in So get we and off much obviously. we dosing results they But using can doses that the dose were with used the

I'm be we're across MC are and in to So get adequate one going agonism for each range, a therapeutic that I we think to of likely expecting these.

Operator

like showing call back over David time. for further closing to turn the Meeker questions to remarks. I'd I'm at any no this

David Meeker

Well, Okay. for in this all tuning Thanks, morning. great. again

heard, forward we're our on you updating all. look excited about call, next to which you some and where Thanks, exciting information. be are, As we'll we we

Operator

participation. your for you Thank

great Everyone, disconnect. now may day. have You a

Rhythm Pharmaceuticals (RYTM) 26 Feb 252024 Q4 Earnings call transcript