Great, thank you.
Let's go XX. slide to
been We today the Syndrome. the content report Alström expand out setmelanotide. and the Alström regulatory FDA work David while meeting both achieved submission. data, data to important data We've the agreed multiple FDA for been totality the of to meeting regulatory pre-SNDA to with we're affect on and we've EMA, negatively given As we said, for are optimistic, did was The the our we Bardet-Biedl busy. and label and as a Alström successful, BBS a to more them of do to review document we're trial and designs, with agreement the that And the independently. that have the cautiously several and and limited the clinical know briefing which we've not will holding submissions submitted FDA meetings discuss steps pleased BBS
supplemental we package submit on are NDA this and quarter. the regular So, to to in a track complete for FDA
two and to in Authorization for But patient the de II to novo patients MCXR address clinical The US to The genetic to meeting. operational excited aged on aspects obesity. an the progress DAYBREAK submission need Phase studies. is where get about high designed rare complete associated had of younger to the better trial track of and XX, MCXR culminating help two genes studies approximately Rhythm, these enable syndrome. for to our The and can protocols, And remain development briefing in unmet about FDA trials to children. seeking a fourth specific of European us expansion the program, may a these population we a made a we've our similar III EMANATE patients representing by positive program, The pathway, five is pediatric studies and weekly a in very lifelong diseases respond compliance amendment children Europe. were a we pivotal and pathway. successful assessment in with Phase agencies our XXX,XXX meetings feedback the with XXX,XXX in Type chronic incorporate we meaningfully which will designed program to today For which buy II additional key Europe, IMCIVREE. trial of program Both we're switch Phase II the the excited the our ready trial earlier is And on trial Slide study. the pathway III care to a the weekly with aspect genes plan we with five agencies, setmelanotide The obesity trials, joint Bardet-Biedl rare long-term very weekly who to broaden setmelanotide, EMA. of we're three-week of our strategy, with Phase X are six and adherence and of in XX genetic A improve addressing with followed documents, conference number transformational all involve both new quarter. Marketing disease the draft diseases. also
significance secondary pivotal data a and on achieving here, BBS adults, and is package. only the strong statistical primary reminder and includes benefit our is reduction. our trial shown clinically weight loss growing. weight supporter children delivering were Here a The however, all loss it that real met does key which hunger trial, endpoints, not and meaningful successful pivotal registrational convey the of The of mean Alström as not but from
XX and setmelanotide adults across XX. patients in patients Recall, we had XX% BBS separate the you of XX were of adults what or really XX of the alone children, two you full benefit children of remaining believe with the patients years included BBS younger the to those XX. and of scores. than total than XX, X% than range to analysis meaningful groups, with Bardet-Biedl withdrew quite primary But and achieve of older prior loss BMI-Z with So, primary remarkable. weight population. when look including BBS greater. look greater we or is in achieved with clinically out when X.X real data the Bardet-Biedl syndrome we week a at Slide at XX% of the syndrome the the XX a who see reduction few not old into adolescents, loss of weight in are enrollment, There or was or Two XX younger the were greater of who XX% at set. XX adults response XX a analysis. XX achieved their data
into pivotal considered enroll age clinically and data medical from more Furthermore, during looking forward XX-week from normal considered period and body of what meaningful. is placebo on and to a quality study. sharing fall, BMI-Z We're patients reduction data including is a trial of BBS control the positive As of data. the to life of from conferences full trial the this extension our on the index based sex, reminder, standard a of X.X deviation of is XX% measure went mass someone's
Registry excited an the deepest Sunday this I we've to for forward life weekend While for history update Virtual setmelanotide and some severe to that lives. what to their no conducting week. Bardet-Biedl the and with the quality examination hear Family this more weight data was XXX video. in as the Bardet-Biedl to hyperphagia was this Foundation hyperphagia been this we active And this Association pleased Bardet-Biedl means affect the options community now even registry, about gain, look the date during on from of excited last therapeutic they excited with and Bardet-Biedl time obesity participants patients present I to are members CRIBBS. greatly the about we're night of Conference. syndrome. community for in of collaboration more to heartened Clinical There now, That natural about by that We're and also Syndrome we have the community, is this announced than Investigating
Moving the XX on trials. detailed clinical our and of to first slide slide
pediatrics. the So,
XX% disease, sequencing obese Uncovering approximately our results younger, obesity of are As children. age a genetic rate we've months from severe diseases these test know The data at genetic these cases the an Obesity these and overall hit tests is children present six early that Rare and old. old or done these from reported. few to in for are with we in consistent a the five hyperphagia just years the of And or six
ready We're their deficiency, leptin this the and baseline The year in dose actually and decrease one as set syndrome. first responders for children second screening. patient BMI-Z several cases is our this For reference, POMC Sydney, the include include this Pediatric we I'd of deficiency in This in which in is clinical Australia, which PCSK at with defined point label five from to reviewed five study score with the Europe the from already POMC is a and analysis, X.X. due conducted primary patients, patients plan a and of edition to long, countries. North greater We achieving Journal responder and open trial with Phase Child being factor in Health, to half America, than III Bardet-Biedl study April obesity year, endpoint to of a will enroll children in year trial two least the XX Asia-Pacific or biallelic with identified study.
a part the our setmelanotide to lifecycle of a drug. strategy corporate and slide of key Moving formulation XX, is weekly advancing
those II formulation daily setmelanotide the well weekly formulation. we compatible daily of setmelanotide And weekly once both The loss general reminder, a weekly weight obesity, setmelanotide tolerated. showed in the data of achieved presented and comparing with hunger reduction a were to to the of setmelanotide. the Phase and formulations from interim observed formulation daily dosing of As generally be study data to treated the that
for X long-term about milligrams of program total switch our formulations been and are a already pharmacokinetics enrolled daily double a in safety, randomized, blind milligrams which ongoing have For we setmelanotide trial. daily a evaluate patients And will study, trial to six Phase on weekly a and registration least label XX with months. they've efficacy, two who trials, of X open II at
daily tolerated. study is The a individuals maintain that show novo want daily point double dosing biallelic will is the that children patients will patients. We placebo. or XX POMC, well years put five gain of analysis years have age. patients of Then trial. trial either and de running with primary The age. patients will greater of weekly weeks. to The BBS the PCSK, endpoint drug label switching arm of with a weight patients XX. to when from all XX patients an on novo XX starts above younger is About increase between on for no blinded we and the X XX Then proportion setmelanotide or And The include safe weekly blind dosing. we basis, weekly daily week other het one randomized a one for week and than de while baseline LEPR randomized, XX to be be responder to weekly blind, will setmelanotide XX. daily receive open and on is weight and between one be X% will placebo, and
randomized XX XX the setmelanotide weekly or XX pediatrics of are puts weeks of placebo States to half for a primary on then The patients XX these go All XXXX. weeks. change to BBS. milligrams weekly track be weight milligrams mean We United in trials in early Europe to comparing for XXXX placebo. will who on submissions XX Both for receive one weekly weekly endpoint the weeks. in enroll at the either further and last or us will in record receive XX one Patients
is which a and two Now an pathway. XX move is are within in II moving towards to setmelanotide DAYBREAK trial, It's the specific with XX genes variants in Phase evaluating to rapidly genes slide registration. stage that XX. designed assess one the the patients This additional of trial
will in a year. an history one For of there's to patient of enroll greater XX. We'll have open They than above this for genes. the of and trial, BMI of first have XX we or the stage label the have design concept. at a allows ages patients stage the end XX proof the rapid hyperphagia childhood least obesity, will to by XX In X expect the variant between running. weight advancement dose history percentile. of and two one, The reported one genetic of They
estimate XXX would go XX XX therapy. weeks baseline approximately at XX than to a are will two. weight those who X.X. We a patients stage stage X% BMI-Z XX XXX criteria patients, greater third, loss decrease gene, recruit patients, per under into there for XX approximately of The or to enter be for We of least two achieving
lose or to who proportion body X.X achieves from reduction XX through baseline, XX% two setting. to of XX under responders. open endpoints. The endpoint compared of an from blind, double two and gene Slide entry of two whereas would stage be to randomized age group. weight patient more the on to will baseline. considered Patients controlled is randomized in X% based greater gain weight responder than increases will setmelanotide or as entry Last is whereas the they in gain placebo therapy and XX at a for weeks And If will patients the BMI those may responders withdrawal. who enter stage weight then are receive by one placebo be genes. are patients by those not weight, expectation rescued on to prevalent reduction and goes compared Rescued Responders a is weight. above years setmelanotide stage that the from weight body Now, continue weight placebo. stratified be active label primary placebo to least achieve XX the greater
scores. their rate, at And BMI-Z Secondary include to also adults, who in weight reduction will safety well include EMANATE XX waist patients trial. further a historic in the and quality is pathway, of randomized, hunger, This average life, placebo we in genes trial This is which percentage compared within evaluating trial which body in change of of slide groups Phase On believe functioning meet measures loss comprised setmelanotide as look we circumference, as concept, a patients. III sub in in overall proportion proof double-blind, evaluating is secondary change weight X% studies announced such will controlled melanocortin-X setmelanotide January. physical we as demonstrated tolerability. five of five of in independent endpoints in children,
group are heterozygous The and variants first POMC/PCSKX.
and heterozygous receptor second the The variants leptin gene. are
The carry third the is SRCX the for XXX fifth and one one. PCSKX patients sub is in variants finally, enroll study in those NXXXD deletion. the sub fourth randomized gene, SHXBX is gene, Each who to designed patients carrying cohort to study
patients each childhood of XX patients receptor based treatment, total get for It's their will history will ACMG will All variant. XXX hets, obesity important patients by leptin And be hyperphagia. in note get sub age. of studies, will that a classification POMC, of then their on PCSKX and and placebo patients. XX study So, to a will have be these stratified stratified
an with for X% in potential weeks, with XX weight of there's Slide treatment and percentile. treatment XX BMI rescue gain XX at individuals from or weeks weight years inclusion increase above in XX above period We a the a XX, will XX. is BMI X%. above XX, if look The
have of been of of date. shows placebo. primary other because to we'll will have primary in body sub reduction failed X% mean not guidance which exercise duration be percentage a regimes than and analysis, responder this XX they're both equal know of setmelanotide something XX% done each diet the a And and five occurs That's development X% the comparing try the reduction and and week XX of patients as Slide and is diet XX the adolescents Anecdotally, successful BMI across The the with provide who secondary setmelanotide. or will to children the on younger XX adults our previously, having we when BMI endpoint Given They hyperphagia, weight studies. for change then we've and achieved a endpoints. who clinical in more defined responders that weeks program XX% trial, at include loss endpoint having our than achieved to identical or timepoint. greater at exercise. in
the this We measures with or weight study, will in the those trial in of and as EMA both comparator. We'll have as secondaries, tolerability, bodyweight The endpoint genes FDA. was particular, study we several each of the interactions the analysis. Initially, losing assess cohort look the score, design hunger FDA small, it responders uncontrolled, a be group and to with studies, at physical opposed also change allows the in these. per separate analysis. score. encouraged the such predict change combined had different the the functioning as as one mean the in with placebo this responder using early the had this weekly at control, mean important in first change a loss primary BMI-Z, life We've of additional of adopted the study. mean for It BMI gene analysis change is at regulatory mean loss sub endpoint. secondary end or responders concern its the On also defined the is in with of allows strength circumference, most mean adults with own greater primary in of look The gene using five early BMI safety, to and and by other the BMI. primary analysis endpoints The we loss as FDA as quality five basket as of across will placebo This weight specifically percentage each more design, in XX waist responder children genes proposed average traditional X% analysis weeks. and to a whether
insight us date we natural include these data genetic will placebo into program, diseases. this strongest this the have set to give will on of Additionally, history and
patients for result pathogenic, meaningful FDA to gene their any in matter allow be and program will to the relevant own both and we've VUS receptor robust is the studies of the approach. by The this interaction where as with and POMC, and a XX, NDAs. subgroups. the addresses agencies. Now, registration. of which need both statistically het sub study have we've the likely pathogenic and be review. And believe independent succeed concerns we In on operational ACMG PCSKX, can agencies, interested and separate is study each developed supplemental classification. constructive support stratified specific will this for study, of always, summary, development our The Slide het as clinically And clinical had patients a leptin
out CRO. well a IQVIA, to have global transfer research was parallel. a underway operational for plan these and selected in onboarding is clinical and as partner, well studies been Contracts the support We two DAYBREAK. thought both EMANATE have executed
than area, East. network treaters more sites trial to – in America, have have referring in as to and a obesity and XX active physicians across the Europe expect X each countries the Middle will North centers. XX have referring We surrounding XX We'll the globe site
months out faster with This read have singular by initiate a Both rare DAYBREAK over may is driven genetic and genes independent to the XX setmelanotide of and disease trials On or onset certain will same turning designed to their may and safe community obesity, before for others. genetic posing to take least of period XX, track each building enrollment treatment of everyone the at these and want to XX-week and years. little we Jennifer, raise to the for But to It a studies DAYBREAK's cohorts be more EMANATE educate on XX first than EMANATE at by testing. on directly end slide year. out and awareness be in flexibility and I advance certain patients the less ahead or and one for and the opportunities is obesity how remind read recruit hyperphagia studies to over treatment early call has trials will two to effort. are the of to as sub we period. is year dose a We others. sub the to screen weeks, for said submission allow earlier, trial.
XXXX, on than out than for three, March as Jennifer. on had for two and we five more than more of for years, is trials. more for great over than than finally, patients, I'll approval. four of setmelanotide With turn the This with As administered therapy us of and, tolerability year, three embark XX XX confidence therapy. years five And these consistent give and XXX a EMANATE to on to numbers DAYBREAK more that, safety on therapy we more XX two
