RYTM - Earnings call transcript Q3 2024 | Rhythm Pharmaceuticals, Inc.

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Operator

Good day, and thank you for standing by. Welcome to the Rhythm Pharmaceuticals Third Quarter 2024 Earnings Conference Call. [Operator Instructions] After the speakers' remarks there will be a question-and-answer session. [Operator Instructions] Please be advised that today's conference is being recorded.

I would now like to hand the conference over to your speaker today, David Connolly, Head of Investor Relations and Corporate Communications. ahead. go Please

David Connolly

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

Investors you For release controlled our conference the release website, that participating XXXX our call, press third results we of This by of afternoon, ir.rhythmtx.com. website. our to provides and the that press accessed and on on those business is issued and our available be slides going quarter our section financial can update,

ObesityWeek, San are is Meeting the to of today the We Listed our X Annual from The Slide Antonio, Society. coming Obesity of site you on agenda.

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

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

that, begin forward-looking who be turn as dates. Meeker, only as our X. represent and should I'll the disclaim any our David today specifically We call Slide to will such not In views With of update any relied addition, statements. representing statements as subsequent over upon of views on any to obligation

David Meeker

Thank you, Dave.

XXXX. be and of with recognized We Day. quarter of XXXX. start the coming highly XXXX, we lead today, Election anticipated story with joining the the year thank But made readouts we the in this So have at the you pleased really November for realize would execution in progress we're not a today. We probably all are X, both

data readout, in look one We presented we real-world have obesity patients. I'll an at which French TOS little meeting, early the more in unexpected addition expected the and have that the say executed a at shortly. was to readouts hypothalamic today about

remain our we X, Slide drivers. focused on main shown value X on As

continues team execution through strong our strategy. the First, results global to commercial drive of

expand Second, to first hypothalamic have real-world the top from from the this patient this potential indication positioned data we program obesity are on half based of III XXXX. and the trial hypothalamic for and in report the confidence efficacy in data new track to on include obesity access increased early to We France. in data our Phase line we acquired remain opportunity

we potential oral presented pipeline Third, Society's our progress at opportunities continue data MCXR ObesityWeek, weekly RM-XXX daily to next-generation progress molecule indications. small and in And the Obesity continue with agonist to make we bivamelagon. day MCXR our The the rate demonstrating with new agonist, expansion genetic

at sales continues quarter driven BBS $XX.X by in growth revenues primarily third the coming globally. with for Steady IMCIVREE million,

continue to patients, access. continue prescribe identify payers to supporting We physicians are IMCIVREE and

life experienced an new years is commercial span continuing And opportunity. have in of rare we approval disease and post challenging markets We in U.S. to team early in only X of environments. introduce It the are internationally. this note, the executing

to The XXXX. remain first the than dropout XX%. Our are rate HO less trial track we clinical progressing report III in data as programs Phase the half line from remains on top

full enrollment targeting We cohort are by of Japanese of patients the year-end.

screening. the number targeted dosed small of of molecule XX% patients has program in Our or

weeks than primate those and dosing obesity submit patients study, rat and nonhuman will along RM-XXX to FDA. studies more completing the are X toxicology with for with allow an amendment the to For hypothalamic we the

XXXX. targeting first are dosing of hypothalamic the type the of XXX obesity We first with in patients quarter

want said, Dave of doing from We to ObesityWeek, X I as presentations. and highlight our are this call, poster

in sought you from the design full the to Slide linked X any undertaking X, XX we pathway. an of the X% be or see was In X, or MCXR suggested the may genetic after randomized of X-part we where to trial. variants literature This results eligible more enter patients continued weeks weeks. ambitious can XX where setmelanotide which XX to reported On the genes, December, with DAYBREAK were the double-blind are to enroll First, lost randomized trial. who the the X out for patients either X:X withdrawal last patients open-label Part placebo were therapy Part

XX-week patients patient or live X, setmelanotide Slide numbers mean as opposed of to the with part measurements. We the the of Stage the XX entered with only XX continuous pediatric On therapy a results in of BMI initial Slide on for the XX% setmelanotide average, of or XX their the on responder see decrease obesity further based weeks. XX.X% trial. and in can XX a BMI of they during of severe decreased shows X%, placebo the Part total summary X adult BMI BMIZ And randomized maintained their trial. X demographics, on beyond to patients had you patients patients patients of completed equal On and this XX% patients. X

spaghetti to you are lines or represent plots setmelanotide, with period lines these groups. whereas those Slide who towards regressed identified see to patients. the those The that X, the the gene the The genes to represent baseline. for patients in quite blue respond, results. The pleased X trial seem placebo of we mostly placebo individual whereas to randomized the be can true -- open-label On green Overall, worked. randomized trial treatment continue responders design continued

their greatest patients scales won't for graph look decrease the with note if on upper left, patients were you in go pediatric can accommodate in the Also on the see that group. gene group each patients and adult through the the that each I you different panels, the left gene the at adjusted BMI right. PHIP but and for the to of the gene those on

particularly gene continuing learnings X with their was had PHIP signaling impairing The responders, X. is overall trial placebo setmelanotide patients The In in through the key to to on gene pathway. those a the there those the Part whereas weight. from good MCXR seem the highest a is randomized general, response, percentage this who of patients have are to variant that setmelanotide, completed clear response who suggesting regained

future genes, be of VUS significance. will for recognizing for little with most many work as has unknown variants, identifying relatively variants these variants, loss development done patients of the function those challenge leaving classified true of today The different or variants been in

of that do these both we research or of Our be will That our expectation with genes. on one is done programs. one will work second-generation more additional or

we've no medical about patients the Japan. introductory previously, prevalence I and to may therapies. Now of my we HO. a need there believe know described in the numbers in approved of We in each X,XXX finish with talking Europe, be believe to and of similar comments range is is We U.S. significant want there unmet patients the as XX,XXX

diagnosed have most randomized care endocrinologists. and the available BBS, access data, unlike therapies Phase are unusual unmet of the in medical programs. of of the to France setmelanotide Both these placebo-controlled III paid and We out significant trial. Italy XX-week and mid-XXXX of II approved in II recognition under move early Importantly, an need, and Phase strength absence data made in Phase the through the of the directly patients reported moved

on [ and is the patients the program. was began ] at XX. initial enrolling just Real-world Patients under are year X French at Slide patients treatment late surgery XX adult from mean setmelanotide. to followed X earlier data XX today presented a months TOSH on patients brain beginning of who mean last age receive X XX age from for of with undergone years been France shown the have Italian to

XX.X% can their of in time mean have at slide, who mean X.X% X on reached As continued and XX.X% these respectively, see BMI Patients XX. they after X were you initiating decrease from months, X adult severely affected lose On experiencing with the of with BMI average, weight point, had average decrease a patients in to X-month X BMI. have the treatment. patients a a

We few we important original in adult treated II since who than is This the less in long-term HO? of long data unanswered set to in almost answering onset the reasons. leaving have and a question: Phase responsive patients readout XXXX. new their II with for data the We adults would multiple first being It proximity goes see as children us in data relatively had no presented this the the Phase trial closer way an that extension, are in adults be important question.

of the a to their the disorder age conviction is management These strengthening injury. most and in play adults XX time consistency potential remarkable further role response of patients out that's the this mean and hypothalamic robust, things MCXR the are response, average pathway on and XX, setmelanotide of years who may The obesity. of been in were the one our importance of from the consistent, said, has date with of I the the in as

is The hypothalamic end that. opening, III X in obesity -- our of and the for our to patients we a complete of Phase by Jennifer of on include U.S. up initial sites label will ages So enrollment expect acquired our expansion survey upcoming X trial December finally, touch summary Based and cohort on year. XX, on of to progress Slide the old to milestones. this PDUFA XX as years to and the date enrollment Japanese XX-patient

substudies, POMC, of by enrollment the expect SHXBX also the complete We year. end of to M&A in X PCSKX the and

trial from for a quarter times, I and full SRCX. the the patients RM-XXX. evaluating enrollment Phase not first bivamelagon we enrollment in With we year. setmelanotide do and turn of our being the LEPR acquired complete the hypothalamic many begin II our over Jennifer. MCXR Phase with hypothalamic will that, We dosing III I'll line trial as obesity the the achieve And acquired other half top said anticipate of Part in quarter obesity data XXX track C call is In pivotal first on global able to we sub-studies, the Phase agonist first XX in readout our with of patient XXXX, in anticipate and trial also X cohort have to the patient weekly in

Jennifer Chien

will Slide I start David. on you, Thank today XX.

in continuing growth approvals reimbursement in depth approximately as quarter, prescriptions prescriptions, approvals third In approximately see XXX new commercially and and reimbursement, a resulting well increase the XX for prescribers. patients. for to we of We steady increased reimbursed breadth amongst as are received

first-time a with by number of the therapy with number and pleased in root prescriptions to experience growth increases in is addresses increase. and We prescribers growth both X for demand with repeat as cause are With prescribers. hyperphagia sustained that as the This BBS more of well patients or continued patients. continues severe the obesity IMCIVREE, physicians on positive the this of driven

of symptoms of repeat now additional may the prescribers they more point first-time in in BBS, prior quarters practice. tune BBS diagnosing a any or this and are become their quarter patients as time to At in the future, prescriber recognizing

BBS would to optimal regions, patients As a diseases, patients. experts becoming expect city in physicians which experts support the are rare these to create see to network helps of you disease in these the are and BBS -- throughout nation of care their

into through consistent falling with geneticists, half continue which nephrologists with and as or prescribers regain denials small to The patients though other a quarters, with have and the prescribers for prior as specialties appeals reauthorization, payer about maintain breakdown and providers ophthalmologists. we number consistency Consistent therapy. bucket, remain initial the for work small of of by specialty the We at with endocrinologists patients pediatrician approvals reimbursement will a bucket reimbursement. remain medical and allow of with pace, approvals process care remains half there primary to number continue about steady on the a to reauthorizations, of pleased in including

the are patients, expectancy. treating of The appreciated both well obesity as as obesity label impact population the recognition ACPs to as current quality patients potentially payers. of to We looking young include their slide. further lives age forward that of negatively expanding To as believe IMCIVREE from goes years of early patients targeted differentiation, Early families. an to patients differentiation in this lead is positively with the comorbidities of IMCIVREE untreated by as can and of for will age life our from BBS a we differentiation support Next BBS and and the that and therapy the indications. onset life X these general multiple children the U.S. in at affect

accepted Last date European quarter, include for young Commission the review XXXX. X FDA our authorization to same IMCIVREE and Yann has PDUFA XX, And the for as of as children expansion expanded marketing of years the December age. priority reported assigned the with a sNDA

measure a at is X.XX And BMI-Z III reduction score, considered setmelanotide XX.X% XX in months that demonstrated was on of from in Our submission data BMI what a mean on based body XX mass in patients therapy. reduction our mean deviation an normal. in children Phase index

the were the would associated in authorization diseases recognize and in U.S. and EMA MCX obesity. position of Approval market obesity pathway and onset IMCIVREE's severe on early reinforce top unique hyperphagia from and differentiate general

may this driven children will what modest their overall provide Early adults, we potential hyperphagia their in BBS often than and for behaviors in patients actively opportunity community. excited important growth, the means more identifiable and milestone for patient and in seeking engaged numbers the While and answers. about obesity more are meaningful are caregivers are onset an This be families.

On to my final slide.

investing III acquired to for in for our preparing trial launch. and are potential a prepare positive outcome next Phase obesity are We the in hypothalamic

engaging payers patients patient insights from gain actively We and groups. advocacy are engaging and to in with market physicians, families research and

to different get hypothalamic support there hypothalamic to therapies. year submission engagement launch. and prepare provide data education top expand as field approved and significant, obesity to we we are you and in is a line anticipate in Also, The unmet closer for physician teams to We FDA with no potential on acquired we our increasing more as efforts planning need sharing next XXXX details and are forward obesity. look

now I'll region. over provide Yann an international update to on it to the turn

Yann Mazabraud

outside drivers and for launches the both with various main the start is continue more France or sales. for In Jennifer. strong on The quarter, delivered now on than Slide BBS The or through U.S. initial the Italy programs in to international month ] I countries BBS of Germany. growth. region revenue and inpatient our ex contributor United XX reimbursement administered have started you, recent will available we to States, Bardet-Biedl an Spain XX. for is Thank a be but government countries IMCIVREE in [ success. this revenues the important well,

pattern are BBS our Germany, the that expanding BBS of and and of number mirrors steady U.S., In is launch we consistent from centers the treating now the growth are benefiting patients.

for expanded authorization in X the and Committee, reimbursement and Joint children last or EMA X And Germany, Federal exemption [ As for GBA voted ] lifestyle summer, as month, this treatment the make for old full IMCIVREE for to to both Jennifer years the in list, as children old to mentioned, eligible X country's the thereby exclude approved marketing of the PPL BBS. from indications. young years IMCIVREE it

we the pleased topic, this very from to Germany, illustrates IMCIVREE I story about patient. obesity. are distinct the that entertained would this general to expected, disease Talking debate are share pathway little MCXR rare While success was need patients committee on which this that like an German because disease recognizes with a Germany from treat

One several. of

helped started trade She month, she than score overcome and One her develop X.X% inject XX first, we LC refused injection. the pronounced After BMA-Z herself, is within team now weeks, resume normalized needles, with ] At a girl our few plus routine normal weight and has X fears kilos considered nursing at years with old indication. fear now hyperphagia, BBS injection. a and But what the of therapy to began hyperphagia, home ] normalized body has more she see months of for [ ago. X and for our a age. lost her our by to [ X.X a

in did can our and not importantly, and course new the with our this support and has And we independence family patient many lives have Germany make region she like international elsewhere reports ancillary the of from sense a program. of that Anecdotes difference in she also before.

access continue Once while authorities. been reimbursement will activities. to we we negotiate than more the be negotiation, for engagement X complete one the physician ongoing with now, for reimbursed BBS to year France, early through able promote we has In IMCIVREE

on in will We clinical experience. early build we the of insurer be a the expanding to number are strong as centers positive access success program with the position of

slide. Next

France seeing both treating obesity. with programs France, shared. already year, access paid-early David began as reports, place in positive this have also data and patients we earlier Italy We for patients in In and hypothalamic we're

setmelanotide obesity that that are pleased access hypothalamic to therapy, physicians patients We with positive responding reporting are well and have quite outcome. treating the to are

setmelanotide to with decision of is monthly, multidisciplinary approval that are with access which setmelanotide Health increasing with BBS. process early his craniopharyngioma. little the old old through X-years is allowed first an this process or XXX limited Also in caused participate committee a uptake hypothalamic process low The and The the is between been of how joint led patients XX-years her directly where we was by with has different Ministry program. obesity therapy federal means than this a access Italy, begin by enable under program. the program program physician similar patients seeing the hypothalamic France asks for patients is In patients to the a to to as obesity

slide. Next

launch beginning Our the quarter is positive following England from recommendation Wales NICE. BBS in this and

health that as a expect BBS, patients uptake during anticipate In recommendation U.K. and as X IMCIVREE adults a provide X be reimbursement NICE therapy that treat centers U.K., to with handful patients discuss they patients families. the and children. Germany that center in specialized are will treat option these that for who national month, new quarter. younger of therapy treating than physicians for the X service care with to we begin BBS We sees than centers when patients and the the BBS fourth with old, clinics start know IMCIVREE Each each XX-years on limits measured deferred IMCIVREE patients We to BBS the therapy. there the are more

IMCIVREE daily and on they train decide administration. If they proceed, educated on be to will the

in families, experience or our questions efforts. of and and care England, concerns. very comprehensive excited patient program support any in The launch members administration and positive have launch been nurses of addressing are clinical supportive assisting supportive other very approval very community Germany, our one to a the of we In in BBS visiting then physicians with of patients, and have homes, on IMCIVREE. the the bringing from team Rhythm Following commissioned another we

slide slide. Next last and my

On continue and One October diseases. becoming I who up real early the tomorrow MCXR designed of sponsored on for offer details that, where engage growing experts physicians our on to [ strategic in in are network priorities are on of and network. of with scientific pathway the want ] experts engagement educate With the we is focused to of MCXR events supporting with or X we XX, physician diseases. building and XX pathway and TRANSFORM, to meeting career this their part a the rare support

XX Obesity President by himself. This of European have was for we Endocrinology Liverpool. of attended Pediatric Society physicians Association at for Study to week, November for from XX of the strong and was and next the Annual Endocrinology XXnd is the And Obesity, on event XX It presence the XX Society from by Pediatric countries. co-chaired Volkan the Professor European on the Study Yumuk, endorsed the which by for Society Association European Meeting will the European in a Endocrinology, the European

are in other with on a of Onset and diseases, attendance focus Early Hyperphagia Severe expecting Early children Obesity strong MCXR of rare by entitled University We Treatment BBS diseases. Sadaf of Chair, Professor London of Professor pathway MCXR the among will symposium satellite rare the College and others. our be Cambridge joined at of and the Beales she is Farooqi pathway Philip University event with

analysis X program data French data real-world also We hypothalamic access on program as -- the and genetic oral from well presentation, as sequencing for early genetic abstracts obesity. European our accepted all for URO, have our from sequencing

I to therapy. are X pediatric to call from obesity now over These on Hunter. the pediatric with data patients turn X And hypothalamic months setmelanotide new following

Hunter Smith

Thank you, Yann.

of from sales million QX to sequential $XX.X in grow third Net a as to $XX.X represents steadily this continued quarter. basis in Turning in X% quarter quarter second an revenue of was U.S. over on the Slide during QX last revenue for of the over increase came U.S. accounting the million, to basis, compared quarter product and revenue million XX. global growth XX% year. of second during the sequential and IMCIVREE revenue sales in a year. quarter On at XX% third in $XX.X the

represented than the HO to third an XX% growth and revenue therapy was XX% early of come decreased XX% quarter in this quarter continue More which over from of in for was patients dispensed [ both of Germany net to on access from ] number increase patients. access in Gross the increase sales reimbursed slightly an in the ] launch of to Driving volume in the commercial $XX and International of in XX% to of and accounted for bio quarter-over-quarter U.S. million and the [ increase the second France. corresponding year. product half QX. U.S. programs for BBS revenue sales

still solid We Italy contributions named phases early launches are several sales Spain, patient also countries their and in and seeing the in in revenue well. are from which progressing but

now are United revenue more than generating outside XX States. the We countries in

hence, countries twice QX. represented receive international pull basis more million quarter, of revenue a revenue of believe quarterly we we are once the hit on Some or Nonetheless, $XX from of forward demand that and mark a on QX these we QX. shipments excited our in a intermittent some or

XX.X% as under well to XX.X% net setmelanotide the higher net driver XX.X% as of of of capitalized the continues second quarter $X.X based be same and revenue last labor QX shipments. to or primary the licensing approximately sales production and to quarter this million QX product agreement based versus year. with of was to year product was Cost COGS expensed high on which the costs Ipsen on in inventory, royalty overhead COGS in our X% of The quarter during in during revenue

of in the related DAYBREAK million the of quarter increased development increase in expenses those quarter second and million year. additional during third $XX.X QX. expenses to quarter third in million formerly R&D a to to experienced manufacturing due a and to M&A benefit million quarter from increases of last known as were the for work $XX.X bivamelagon, were both trials compared during recorded the XX% we R&D for $XX.X Sequentially, LBXXXXX. cost Plus, changes there scopes this trials $X

quarter were quarter-over-quarter for versus sequentially decrease for compared million quarter was same $XX.X driven equity by the in $XX.X of a SG&A third $XX.X in tax $X expenses the to decrease QX expense a represent SG&A based changes year. loss for second for French quarter largely quarter. options million The million reduction tax last million payroll nonqualified this the XXXX. expenses on

quarter, For the shares $XX.X outstanding were common million. average third weighted

Slide Now let's move to XX.

September used cash and as XX, of QX. As million XXXX, burn in average public cash $XX.X was million. in which approximately than XX million quarterly for was we first The cash approximately used significant reported company equivalents. $XXX.X operations, cash $XX.X This trailing another months million less in the was $XX a operations Cash in quarter milestone. Rhythm in

to revenues grow. So improvements as we leverage generate continue operating in

On used operations of $XX.X period million, basis, XX% XXXX. was a year-to-date cash for of the a comparable reduction versus

EPS the preferred for million included of million quarter accrued compared $XX of loss quarter $X.X the compensation previous for and Reported to $XX.X $X.XX, convertible on GAAP Third total in net basic dividends million. diluted per expenses third quarter stock-based of includes stock share quarter. which the operating a was

the payable prevented per quarter million XXXX. As this end a dividends per of ] are the to of prior share be second share the quarter at current accrual will $X.XX No count. ongoing $X.X cash reminder, or [

is million represent R&D, in our expenses operating guidance the $XXX of comprised reduced have of of Slide range updated from to SG&A, XXXX of guidance and estimated to $XXX these of both updated approximately This year, midpoint our numbers Today, range. components XX. non-GAAP prior guidance million. $XXX range million expenses million the a quarter guidance million, $XXX with remaining operating OpEx to approximately non-GAAP Turning of of $XXX which to X $XXX we only in million

half data continue XXXX, sufficient that, III line we value-creating to I'll operations to fund back first in With including planned be turn expect over beyond into potentially to XXXX. the from for the readout well milestones, currently trial Phase David. cash the obesity planned multiple Lastly, hypothalamic our hand call of to on top

David Meeker

Thanks, Hunter.

And a So and very we're in lot think a heard year momentum. I of entering finishing summary, -- the quarter. with you've XXXX good entering

open for that, So we updates. With look to the call we'll forward future Q&A.

Operator

[Operator Instructions] Nadeau comes. from Our of the Cowen. question TD Phil line with first

Philip Nadeau

quantify prepared question Congrats to some into QX. strong from forward? for quarter. any other on first items you that of from forward pull was In demand be QX And on what IMCIVREE a you the pull the impact remarks, Would there Hunter, included revenue the number? you. was mentioned lumpy QX able in

Hunter Smith

it's attributable a it depends, nature quarter, obviously, But imprecise the these little we in It's late timing and the come the quarter. they sometimes, of the and orders. if on think more to future

So that's -- around be we estimate $X.X million QX. in that could

Philip Nadeau

a to is pivotal continue the decision do a be And of when And make think helpful. populations advance then They That you those Okay. might trial, to strong. very on go, data. to you populations? congrats question on to to no-go Can the study? see position about look second those DAYBREAK you talk one in a on what you need

David Meeker

Yes. Thanks, Phil.

these do are the can to to the be case to remarks, we loss which ] the post in extent In we in improve I'm that the [ right that Because expect driving sure of do that in think answer of of underlying general words, loose which hoc, of you now, disease. variants many I we said -- benign have results. their other So my variance, wouldn't patients, those and defining the function. true variants can the understand genes, each is, I the better as terms that of for

the sense got comment A today. that is better. So like we've gene that's to PHIP, a bad general understand we not have

sort would do again be program. a look earlier of prevalence go numbers, I done. -- to -- the order which would earlier it a That's That's that more that but would might work has the there's we gene I next-generation order gene we with BBS-like think soft, on, a of can in mean on X,XXX. are but

So one need or to NHL. Phase both of I those would have cleared

Philip Nadeau

very on the is again Congrats That helpful. progress.

David Meeker

out. if that. a not to were a do That's XXXX we kind of that activity, XXXX, moves That

Operator

Archila Derek Wells Our next with Fargo. question the of comes line from

Unknown Analyst

in This us is been on see reduction? us. you what the Maybe HO And HO Adam at can ]? terms then of Do a in just be X GLP-Xs think the patients real-world who can Derek. III couple from study Phase the data weight had we for on experienced you on from GLXPs (sic) know on patients [ of predictive France BMI previously Congratulations the quarter. loss X also, let of months, which patients will

David Meeker

Yes.

data, Phase I So say, what the helpful. drug. will One, this would patient this the I we II predictive? take original think X reason is I patients, be only XX do the think, didn't think so incredibly who is

who XX patients the drug. So took

And Now what's we literally patients. a have this X reassuring diagnosis has is had most the response. patient good taken drug who with has every another

through out remarkably, of terms incredibly III, in The decreases of decrease is reassuring. But the to we've Phase in to now, trial in the of race magnitude percent I the BMI. clinical very we're think, consistency So percent any stay reading decrease. discouraged trying seeing good arms around

victory not them. it have be patients these For nothing, simply for gaining way that would

Phase the well III positive for trial. good percentage the more we're the But think is of decrease. important consistency, and a I part again, So seeing this outcome predicts in that

the of patients they breakout, And GLP-X which the whether the they then the doses the for it. the can't they patients, question so GLP-X they X don't were trial. on of answer patients patients, on the X started stopped are before of your the specifically, I on, X have the were But

would So of get -- the more drug, on of But And were were reduction. if program. than in treatment only X losing were an access they this for of losing weight, understood, weight diabetes during. not I they as actively been the on, attempt their to them early a enrolled have specific X obesity they GLP-X a actually beyond the

Unknown Analyst

on how Got us. who it. many this do to Obesity population had -- dinner many from receive Week, XX,XXX then you've we any to with these have this over patients contribute close Is just they also the And [ the above population may up if patient so, a more one that radiation be to who And our HO have believe U.S. of that estimates X,XXX make or up may somewhat patients ]. you [indiscernible] they to in HO aware been and tumors time. maybe end of? brain that At even at that noted

David Meeker

Yes.

have I And are -- the which We will, and when focused you you the other do as very can this those get ways who of surgery. whole of There HO injury, very moment area you injury on benign injure so to go the a of hypothalamus. if this evolution. specific are well-defined how have group. tumors, part they think it they

radiation that patients. a in of similar from anecdotally including see hear We they patients, some those physicians picture

I more to the So learned interested, there's learned describing, more learning in there. think there no estimate that. to anecdote and be, I their number there's to more We're But about have patients be response. very you're might think I that. what of heard obviously, we've be about as

Operator

Our next question Goldman comes of with from the Corinne line Johnson Sachs.

Corinne Jenkins

Maybe a us. couple of questions from

then of about will a it do how Europe we sort look become XXXX? share from larger And sort as that overall of think you the anticipate contribution should get the BBS First, of and we into patient respect you further launch? on we us also seeing with to updates compliance And you're adherence in population? provide as some into revenue what that could

Hunter Smith

the take first I'll So question, Corinne.

think the be a is I continue base, starting -- we to solid we international, to it the from larger which variable going there. I continue to think, in base But and to which keeps pace degree a built U.S., quarter-to-quarter. growth foresee we've is with

David Meeker

Okay. And second adherence. was on the question

rate? So Is are that you're about focusing you what talking the on? discount

Corinne Jenkins

exactly. Yes,

David Meeker

Yes.

ticked time, some have remained do general, and as things. to can what work changed. I in can to good we we're think insight Maybe because that's last little we XX% color on Jen? closer doing more to some a up Jennifer that problem continue we've and -- So we provide hasn't that said

Jennifer Chien

really we setting your focus answer are when terms a with in think and I question, the learned, expectations. we have clear that discounts, we have of early To these different this insight, our like to take really and of look continued coming at lot things many a of educating efforts just there's

Initially, more well. on efficacy the to drug, more the the expectation needed it was also profile we recognized in that provide timing impact but patients of AE we around of as

think a may may reasons patients reasons important So to different from go, different patients interested ]. I potentially [ in able terms lot we other back. a be we there piece is perspective of maintain is make that that coming of therapy. one it life on There learn being And as that that also them are our just those discount in

patients majority of are these vast The consented.

So our been have patient engagement back support them. who teams to maintain well. in getting on therapy with are patients able And have as we interested seen

Operator

comes Guggenheim with of line Securities. from Fernandez question Seamus the next Our

Seamus Fernandez

bit assets. bit the talk HO about a pipeline to wanted little little and So just about -- more a

get data access Just more result to impressive an just the population. color out wanted France, patients on I start obviously, little the adult of to with the in in X coming a bit able something that you're to onto more in sooner therapy that opportunity mobilize then that with result? to patients data. with the opportunity continue early bring that expanding and Europe the Is

very quite is I I view will IMCIVREE to on that the Obviously, certainly physicians. there's then is second the to strong And question receptor from in think setmelanotide void But really with opportunity compelling durable. pipeline. know or be the MCX our a conversations continue the

wanted get progressing if sense I a and and how are the perspective those didn't just you but data? recruitment said are hope execution. of a you I in believe So to past, midyear updates know would any trials those better really there share from when to

David Meeker

Yes.

would steady? to you to Yann, What and the want going stay say? Is do a little on is So expectations? how or ramp process working color provide your HO that you

Yann Mazabraud

thank So yes. you,

for local A will in on help, have data will always it think. help. so to I local it top physician global France, of So sure, data, likes

Italy a data, and help takeoff as experts will and it French lot Italian talk also look in sure, respectively. at well, own For the

there say are are will data patients, early programs at patient likely look countries And and which on smaller there we that this top currently but France other to already not with are in that, named and start so decide like of identified Italy, need. large which some many can have access I countries where sales,

David Meeker

on regard with incredibly Seamus, second And important. which next-generation programs, question, the your to Great. we are agree you with

weeks nonhuman So in more patients X-month an original had sense XXX, trouble. ramping X X reports to to need are studies submit The get at rodent which will didn't new for the previously, it's We to the quite FDA the is said up. the protocol the those there's as need and primate. the the could treat if also, weeks rodent, the could because the no time, how there that physicians allow the drug Those weekly, amendment submit and the X-month we've -- same was to first written. weeks, said, than for intense. they us which And for recruit we only patients are X think we as update

obviously, provide to So to bottom for longer-term the able want those we patients. be is, treatment line

So that's XXX program. the

out we back midyear, we're open Our sites start. patient laundry on the that goal, and The -- ] just -- slow we and bit is a I A have Cothrabo moved had based of in reasons, open the timing we then in but first submission, to sites in, bivamelagon, first on it a the XXX. [ up early we've rolling. list think the and dose summer and now tough getting of an that patients a got amendment had very We is program

than little about either so dosed XX is patients that or more update in a XX% more got we've -- just today than And the screening. of the

give So our which readout to the while complete trial quarter, small still is is in for date you're we enrollment midyear there will, of a that possible means recruiting, it's still molecule always but a program. expectation sure, the firm that that hard for first

Joseph Stringer

you just feel Can to this is is maybe its us Japan that can one still the And of there? something how on, the then that opportunity And past. for HO final take that something The sense confident Rhythm a in HO it is is opportunity as on you've question. give in talked own? just Great. you about emerge likely something that

David Meeker

Yes.

So at with the is Dr. like met today. in who lead ObesityWeek. we're Japan investigator Tanaka, we said I

He in my this this positive enrollment patients end we'll is that Japanese is going. of complete working I how As again, sites highlighted the the script, are year. required about expectation well by The very of together. our XX was

I at that? we handle about our if diseases, it's a point, can Again, Japan, the But not we a if so if think if people, go So it anything, about you optimism the all asking Can not of rare alone. starting of increased. the it's I -- again, size I much you think you're people right have diminished, and and as country do the have right you think function necessarily. it's

plan today. our so And still that's

Philip Nadeau

the quarter. Congrats on

Operator

from Gon Stifel. of question next comes line Our with the Dae Ha

Dae Gon Ha

week. data guess all on I'll And obesity I focus the just the more hypothalamic congrats on side. this

in I questions data, can just intention go hyperpigmentation there, side of well share of XXX there any Part you healthy couple from a the get as Phase to Just there. C we for sense PK the so before for volunteer, guys the a the as is you profile?

about the I I bivamelagon hypothalamic think we in about Phase recall sites of trial participating the you versus SIGNAL III an in there obesity? much between when guess, is the trial think the it I As participating overenrolled. bivamelagon. guess itself, -- that overlap trial how was

into, that and bivamelagon track bleed the And will, some perhaps could so fast enrollment? of if you I would imagine

lastly, thinking about wanted commercial opportunity, get first? DAYBREAK the thinking a hypothalamic something DAYBREAK after you on here. sort to goal question, profitability going M&A going that than of back guess, you Are might would be by guys Phil's broader about obesity to just going I and and like subsets promising. then just maybe your And sense the after be M&A, after looking more Or opportunities expansion opportunity

David Meeker

haven't bit that perfectly saying C, come reasonable any I a just share plans delay the to I'm was about But the have and delay, Part given think think meeting. we Part a may perfectly Okay. question given first do little I that. it's to about parts? in We we would we in I A it's not one, -- made for the back Will XXX, B your last so terms the C. of where won't. question. -- the

come and defer I'll to So that. on you back

for plead question, the second sites, I'm here. your ignorance going to of the and the overlap On bib

a have part of trial. We were our part of III original number not or the of HO sites which not Phase

that overlap sure you are don't But for did over but have As of sites I new, we these tell we there. you can't said, most enroll. some

can you offline. to maybe So get we back

Hunter Smith

I can on. probably take third

we've for firm I and a it's speculate at would the think what from because established and to investment HO a would little time in would we generating profitability, strategy we revenue that M&A it and early trade-off a those a in the of time question line both be. between registrational that be on DAYBREAK invest presumes to when that

I So it's there's say. early a think consistently moving of evaluate but prospectively, little them parts. try We lot too a to to

are terms at dilution-sensitive company we the would under the all I as we say is generating in a shareholders for the area that flow repeatedly, to as opportunity same of biggest for recognize time, curve cash our the we've the And said maximize We're very What here. is, company. shareholders.

trying to So those the of all optimize we we're parts best can.

Unknown Analyst

congrats again. good. Yes, sounds Great

Operator

from line Whitney of comes next Our Ijem Canaccord question with the Genuity.

Whitney Ijem

still on Just one hear the you me, -- guys can follow-up okay?

Hunter Smith

Yes.

Whitney Ijem

Okay.

cohort. of full the Japanese less follow the Follow-up on for overall filing an the Japan Is in us is remind the on there you you the Or to cut Can with study follow-up is the Japan particular? opportunity. -- cohort a of analysis interim the out potential Japanese Japanese up? cohort Sorry. a it I know said based in of year plus

David Meeker

the former. it's Yes,

additional it's plus so form there's XX Japanese filing. was patient U.S. that, an what the XXX be is that clear the Japanese filing. patients. year, the basis first which -- and it's we've the patients will been the will that's gating When about over-enrollment in out for Japanese be then finish a the -- patients EU So and the patients, the the last they XX will at And

Operator

Hung comes Our the Stanley. next with of Jeff from line Morgan question

Lee Hung

time time or with since patients decade And The setmelanotide. therapy any that regardless on score resection? be a benefit French do from hunger an then could resection? over And resection you ideal would a that Are not, BMI seeing real-world of derive correlates that I think still if patterns a study you a since setmelanotide follow-up. had who suggests have ago

David Meeker

Jeff. And -- trial I amazing perfectly BMI is highlighting the difference. make seem changes this. the you the in HO said, most hunger It's saw Yes. and was doesn't the what think are out XX part what of drop that in time XX. patients it's despite the original about trial, do fact is to thanks, No, exactly that you're I the consistent which think The Phase with II pediatric what we a

of matter. seems it, it and So defect. It's interrupted doesn't MCX I defect, solution. when agonist to think biology the to no it somehow the had pathway the signaling you've impaired be matter The the you says yes, MCX

Lee Hung

you a U.K. hope then to Can that registry gain And Great. what just you greater announced you to And additional reach help few access to patients? weeks Axovia partnership BBS. you the with from in ago, talk the about gain will it? BBS

David Meeker

the the access let to in on to in now he's announced so -- the trying going Obviously, one ago Yann the U.K. And doing the Bardet-Biedl. comment eye leading What week to work develop with the world. registry. I'm he's we closely findings experts work We Phil is treatment him. we a for very a know of Beales well.

wanted that. a of Two, shared of terms have in BBS. we we to one, epidemiology understanding support interest the So

sense it to together. made work So

a us, of would think on which put your opportunity just yes, maybe And don't he's deep thoughts this couple got to it data, Yann, he But together. on that. an I is comments deny but

Yann Mazabraud

just already add can support... I No, that we

David Meeker

Speak up a little bit, Yann.

You've your got into speak to phone.

Yann Mazabraud

Is Okay. good it now?

Hunter Smith

Yes.

Yann Mazabraud

years many the the to that on Beales in have so. BBS support just we U.K. will would started I for and effort I worked X with have we ago maybe, Phil No, now, this already say, Sorry. to add we patient -- year continue registry. can do I his team support BBS and association, and it collaboration

Operator

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

Tazeen Ahmad

about of to about maintain? GLP-Xs you secondly, and combine the HO going to terms Phase maybe to to for for that order with guidance of think broad HO, to that then going told need in you're there, expecting expecting happen time? some a to what's able half I year And III data little drug? you're setmelanotide In early bit the condensed that we line be for that think you to guidance that have of XXXX. the is condense? doctors Should the what time given as line And use the Or of a desire time be you've next that first be

studies And would commercially you're but that not know size? on I per doing se, a speaking, be that good

David Meeker

fair. could, we've you we've -- what first which is question, best we given your So as totally mean, math. I just the which is said,

dosed from trial, the the day So then. do first February. weeks last last XX math on XX-week you patient literally so last of out, patient at can It's a

trial like. to got and And out close the then you've the

out an to working So we'll not as efficiently, it can it's instantaneous. aggressively close as be but we

update don't think better that. going it I we're be lot to able than to a So

XX. we going to it's not think I June -- be

not think do that the on I can all math and case. you conclude that's the

there able be more publicly refine helping but we'll beyond not the sure I I'm that, don't honest. math, not to it you than much much be I'm very to know.

in second the gain our mean -- on interest. for think the HO is world whole and world, the combos Your question is I weight on combination reasons. different discussed, general. was therapies, there In I we focused we've as

been for patient So plateau. well another their may gained And setmelanotide, may added any anecdotally, amenable a have who they a GLP-Xs on has on example. deficit and signaling weight for other which to who pathway be in reasons, patients drug, to we does have know They also been have GLP-X, that given MCXR may setmelanotide.

is weight cases, hypothesis. I loss, with that In they've those consistent incremental some had which of think

good me, be patient thing were better that is mouse We anything thing? used the think a model, that additive. your I good is the question together. know medications So gets outcome. in we To can a was, last And know they a that a

that sense it makes So standpoint. from

of nausea, as in complaints part little of that. There's They GI toxicity. the overlap bit have a both

using be done. a but so may that case, the can And work more it together,it's little the X drugs be

Operator

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

Joseph Stringer

in the back were XXX TRx BBS pretty launch, in you're steady seeing new U.S. patient on the quarter. another adds, growth Just

and Just wondering identified the new in if patients the diagnosed? are vast this Where launch. be majority you diagnosed? to drug would helpful. can on Are color describe are and/or how that point at they the BBS newly Any being this

David Meeker

Okay.

go there, but international, Yann, thoughts if any we'll So then have on can with start Jennifer? you Jennifer. you And

Jennifer Chien

Yes.

mix general. think a So it's in I

being in teams have for rare ] understanding patient get and of outreach time and physicians aware terms to I patients to to heightened been a the and getting a way, to those actually through the with contact that that symptoms patient of education are that disease. just as various forth, [ think physicians, back their overall lot they It's over doing a diagnosis. different and in spec actually our and come

tune, more they're it are potentially diagnosis. and So as ] -- more in to becomes it educated for patients get them [ these easier to also to suspect patients

some have understand this have repeat explained, contributing. finally So are the patient I as prescribers prescribers as they initial how, as first-time and of value to IMCIVREE diagnosis as gotten Both well. well is where a we

David Meeker

maybe good? that leave Yes, we'll it there. Is

Joseph Stringer

Great.

Operator

question Raghuram next with H.C. & of the Company. from comes Our line Selvaraju Wainwright

Raghuram Selvaraju

you see to at hyperinsulinism. could activities congenital in are that planned wanted if comment Just all on

there where on expect Over on color the reimbursed XXXX? you also, if front? be course instituted of might course if us updates anticipate early the additional additional you programs XXXX, And of should some we any give could for access over setmelanotide that

David Meeker

Okay.

this second question, if you point. you've other at just countries Yann, the you of back But I'll in come highlight to for So your would know minute. where any don't part want to I a got

hyperinsulinism. And Thank just The on first I the in category, CHI, got Apologies I I question would causes. oh, that. about the earlier question clarification. you. this on we was got congenital congenital think -- rate put

identified. and our said, are waiting develop progress. CHI molecule, which program, on a I we about, good as to So our get We've have not made molecule spoken we lead

in have And and in are We so updates won't good XXXX. we XXXX, I will commit CHI. here to making interest high remains updating progress in our but the further

to to other back sort be where With phone. looking any You your at? additional access that, go we would Yann, of countries early got

Yann Mazabraud

laptop. Is am close my now? I very better it from

Hunter Smith

Yes.

Yann Mazabraud

Okay.

Sorry.

So yes, thank you for your question.

good not but countries, in many So for so reasons. fact,

countries U.S. already good or XX paid First, early we we of countries countries access. than have access ex early are are, in earlier, as chunk these And I programs where a said more

our countries very carefully. we pick Two,

with less that want countries somewhere, could be would more much when make it we come be will start there some time sustainability. first, on to but we also sure more we and important in So countries,

go. So attention we where to we pay really

and your we be So but than by in months, to not and or again, likely design. the choice XX next additional back X based in by that countries that, questions, on more will X

Operator

with of next Our question Citizens the Wolleben line JMP. Jon comes from

Unknown Analyst

on on This for I Katherine reimbursement. X questions ] Jon. is [ have actually

with the improve is of and kind you was The now. did this how discussions much first And payers that which can what much to upon the of early cause and on reimbursement early, how room to color what any and question denials patients just current paid be and is, had for there? to done since that works in just of U.S.? the upon how regarding rate currently have kind there drug -- more have kind HO then in And shown improve the can this -- identify patients it years get diagnosis

David Meeker

want on Hunter, comment rate? to the paid do you

Hunter Smith

there we our statute the -- coverage to what an Medicare indicated therapies. payers loss. commercial is that coverage coverage by for close no consistent do we've are And -- pretty get we afford commercial rate, obesity among coverage plans, because are we coverage full med By where have expensive from to level speaking many paid small been which you do from commercial, not weight for high essentially from -- cannot have a not are although of or

covered in plus lives levels, And then high of Medicaid. very we XX% plus have --

number to transition -- said patients, number where scripts. of drug running of We've been kind to the of free that been that's the the saying. So about free XX% sorry, we've of scripts go has that

David Meeker

of the for don't drug the issue example, covered don't one the -- has we And in understood general, been obesity they more question It's denial. a, get b, for we And the if price by other policy-related. Medicare. I don't se drugs Medicare. Do per not the That's maybe was we been cover thing reason discount.

question? So answer does that your

Unknown Analyst

the answer my to question. that's No,

Operator

conference over I Dr. for to back turn closing you. would Meeker to remarks. Thank like now the

David Meeker

everybody call. busy of earning tuning here to unconventional end again thanks day on Well, an a and in the Okay. for day

are we forward to our and Thanks. Just excited where look next update.

Operator

you today's conference for call. concludes participation. your This Thank

may disconnect. You now

Rhythm Pharmaceuticals (RYTM) 5 Nov 242024 Q3 Earnings call transcript