HRMY - Earnings call transcript Q3 2024 | Harmony Biosciences Holdings, Inc.

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Operator

Good morning. My name is Todd, and I will be your conference operator today. At this time, I would like to welcome everyone to Harmony Biosciences Third Quarter 2024 Financial Results Conference Call. [Operator Instructions], Please be advised that today's conference may be recorded. [Operator Instructions] I will now turn the call over to Brennan Doyle, Head of Investor Relations. ahead. go Please

Brennan Doyle

Good us operator. Quarter and you update. thank financial Thank as morning, we joining business a today and Third review for results you, provide everyone, Harmony XXXX Biosciences

results encourage we website our measures. today, represent At non-GAAP our the better including everyone Before reconciliation find non-GAAP cycle, I to to we our section to our the accompany that business of financial of underlying this life performance. go believe materials a stage of Investors discussion financial the to start, GAAP

Our Dayno, and speakers Budur, Dierks, Officer; Dr. Financial on Chief today's call Chief Kapadia, Kumar Sandip President Chief are Jeffrey and Dr. Officer; Jeffrey and Officer. Officer CEO; Scientific Chief Medical and Administrative Commercial

statements current to on today, which reminder, a and As are expectations are subject beliefs. forward-looking our These certain statements risks uncertainties. we will making and based be

and results in details. encourage these circumstances even may if differ actual the Our factors undertake consult materially update to for no additional statements SEC filings to risk We obligation change. we you our referenced

would call Dr. the Jeff? Dayno. over like now Jeffrey to to I turn

Jeffrey Dayno

Brennan, quarter you, delivering for clear At and event toward developing becoming strong month, Investor thanks, share CNS medical Thank treatments WAKIX we a focused for team conference joining needs. this our and with earlier unmet on revenue outlined clinical path and successful new the our the significant Harmony Day growth were to excited of at data to company patients leading innovative driving momentum was QX advancing everyone, call today. programs. our late-stage and another for development

or or next launches pipeline generating long-term robust is each during year shared and creation shareholders. With poised patients value our more catalyst, X to to are each years. our for we the we As delivering promise X indication durable deliver over presentation, product new on late-stage that

in Harmony current poised net pipeline deliver over driven thus is that billion In team our $X to forward. fact, has success it's successful, with at revenue this going far, our

Also most Investor I what Day, rare for we strongest be highlighted pipelines and believe the promising with our industry to the diseases. living during in neurological X of people

includes X with franchises each potential billion, Phase XX rare now with development assets $X of pipeline a a of sales year-end. across billion III $X X them in to fourth programs Our initiate peak and X trials to pivotal before CNS orphan

call but robust the tremendous go take able not I into to will pipeline, development our our on points you our that the in we these. all call, regarding pipeline, to are Given the growth want depth today programs on be key from away this

leadership First, to we our continue strengthen position in sleep-wake.

preparing are in our or hypersomnia, to We submit IH. for pitolisant sNDA idiopathic

half programs, we second are the We next the then track clinic IND and mid-XXXX the to our potential are submit best-in-class pitolisant advancing next-gen an enter orexin-X on and agonist in year. for of

Clemizole we or the in encephalopathies as class advanced or address EPX-XXX XHTX receptor for most childhood of known late-stage need Hydrochloride, the have the the developmental to unmet with medical and promising serious epilepsies, DEEs. epileptic Second, development onset rare program agonist

syndrome Dravet we EPX-XXX an patients ongoing registrational initiating in with XXXX, syndrome for Phase III EPX-XXX trial with on pivotal patients line and for Lennox-Gaustaut and trial in data year-end. is a in III Phase be before track for top will

recently potential both acquisition initiated a recently the with has significant Phase our have or epilepsy a that selective of just upside addition, in is syndrome. trial formulation, another liquid have in a as XHTXC In which receptor in agonist these market XHTXC a III the we asset pipeline, Dravet EPX-XXX And asset lorcaserin of agonist. acknowledged

RECONNECT ZYN-XXX data year IH to well from top highlights expand FDA's living file share year market the data including and III going Third, acceptance patients in treatment then first line as half trial there are a with are on as If the call. in our Kumar on approved exciting on Fragile of Phase X this in in our I syndrome. in these coming first catalysts toward submission us will for points next sNDA want with bringing near-term later the decision pivotal patients you programs, could to it with later for some put Fragile development the path positive, these X syndrome. key of the

extension X our data foundation the of which year. we at efficacy strengthening from new our robust the the is pitolisant on sleep/wake, patients Investor data demonstrated in with shared and our response out IH. business, long-term First, beyond leadership trial of of Day These in sustained

of for over evidence majority overall pitolisant a real-world IH for on and trial the for to you, normal year-end. This, is pursuing coming our strong Epworth we severe reason conviction with the year into benefit-risk along strong after indication pitolisant before are the submit an and proposition sNDA range within an As for in or at on will moderate the Kumar sleepiness. a X patients were level maintained Scale show of Sleepiness the track

into how pitolisant the positions on the Building and pitolisant and behind high track reflect in HD dose, And extend XXXX. our or make needs molecule action pitolisant off of GR in of next-gen both provide is model the GR PDUFA gastro-resistant WAKIX patient-centric or programs the Dierks color is Jeff medical HD track to for PDUFA development unique patients make more and us on goal with it with the is of by its to market mechanism unmet commercial of on novel formulations in narcolepsy. the optimize a even those first-in-class will the drug XXXX take the pitolisant programs, in Pitolisant these our pitolisant. good -- to and in for innovation XXXXs. opportunity strategy success of for franchise the to and grow ongoing Both Pitolisant better with and drug addressing

and of wave orexin-X hypersomnolence for disorders next agonist. central receptor treatment innovation the narcolepsy the The are of of other

BPX.XXXXX a of Bioprojet, which our the As orexin-X agonist Kumar during with several with be Investor this which in this on diligent the some have the several review could in followed leaders agonist and this them in This earlier partner, sleep/wake, compound. feel compound then years, closely past of programs. our is best-in-class over we we we you call. unique And year, Day, licensed potential shared orexin-X space based of later features will few

an on XXXX. IND and track the towards first-in-human are of We initiating then filing half mid-XXXX studies in second

developmental the recent in Next, franchise the a to in our in-house regarding of must of in value few encephalopathies. share approved our currently safety these a significant treatments of their unmet recently and/or take competitive efficacy, and with living Many for brought leaving therapies I that the limitations the gap some addressed particularly face Therapeutics. landscape, patients caregivers. medical of epileptic which treatment rare be refractory that to light would like This and you with developments discuss for and is rare through the and moments relevant, seizure Epygenix of epilepsy to need serious excitement terms new acquisition we disorders point tolerability of

recent the the involves the of act in Harmony at view and compounds to as tone enhanced XHT space in approach brain. is this be it innovation. receptor proud interest as and our that validation type serotonin X the simply, for space again this To of We serotonergic put forefront

compounds formulation following: and We have want will what which on mechanism Investor and you serotonin his or the and hydrochloride Day, both development clemizole receptors developed to with is compounds has work EPX-XXX that for liquid highlight X validated both and his a DEEs. I positive model our model. action shared been regarding for But XHTX investigational the who efficacy working lorcaserin. at by their predictability predictability, were EPX-XXX, and XXX% more share through you demonstrated in of zebrafish for negative EPX-XXX Kumar for these programs. Baraban, modulation EPX-XXX screened zebrafish in the XXX% Scott products details of XXX%

patients We Lennox-Gaustaut before have clinical with advanced ongoing patients with track pivotal track the trial which registrational in of which initiate as in Phase with on as agonist is syndrome trial development for program to is XXXX Dravet top the for data syndrome, EPX-XXX an III a most for line XHTX well that on compounds that with that successful, What and treatment have our creation for drive and believe we offer patients value away significant to and the we want clinic our most year-end. are advanced of confident new you for most robust, if the options patients I can is portfolio shareholders. promising in portfolio assets DEEs take for

promising see, franchises you our pipelines have believe exciting the expanded industry of X rare pipeline diversified can and pipeline, most diseases. our built and portfolio we and CNS as neurological the on living for with in strategically we rare Lastly, our X orphan is what patients across

the ZYNXXX X of study anticipated in midyear. as Phase highly submission sure acceptance followed in readout first decision top investors. to near-term is These advance for track syndrome, want make long-term first by which are for These pivotal our top I the of year to readout on we III FDA's Importantly, data in pipeline half catalysts RECONNECT are patients exciting catalysts file coming build next quarter and on continue value our IH Fragile the of our mind creation. sNDA for next include line year with that of the

Switching gears.

and While we solid is than years WAKIX advance in our to generated the which This X $X enabled quarter for focused delivered WAKIX on net market, billion of we in cumulative with significant across the revenue revenue Harmony accomplishment. net another execution million. remain surpass $XXX late-stage a company development programs, less on

are a that goal has once pipeline again reiterating We billion-plus expand $XXX XXXX are to we guidance being way million remain in million well achieving $X in our and on narcolepsy even with net a we and team business that. deep With dedicated of alone active is WAKIX and our in to experience development our further. these results, to opportunity confident remain strong revenue $XXX market the

I approximately XX, and And cash, in to getting the opportunities. apply we just we demonstrated built just a of in to have we development of while are is as we With X additional do September that growth strong all so, be equivalents in we far. execute are Harmony continues story. thoughtful cash at And what same thus that started. proud position approach years, first strategic financial say we'll $XXX and a Harmony This if have our on million am business to investments

long-term We patients value because developing we to generate and by on to that when diseases, our have neurological durable this rare our innovative delivering deliver patients with creation living promise shareholders. know for outlook we treatments we

now Dierks, commercial on will I performance. that, Jeffrey an for turn our call Jeff? over Officer, the Chief With Commercial update our to

Jeffrey Dierks

our XXXX year Jeff. since were net in million Thanks, and continued billion-plus quarterly quarter confidence full underlying Net quarter. momentum our of to quarter WAKIX surpassed saw in in for launch. WAKIX million. reaffirms these adult the solid for $XXX and third quarter of fundamentals alone. million with And potential sales $X cumulative billion business sales, another our third sales the $X strength We performance WAKIX $XXX in narcolepsy in in guidance The for net $XXX net sales sales the the

growth saw quarter majority continued applications We the from given our was of WAKIX patient increased on expansion extremely approximately patients opportunity. base, number in in access quarter. narcolepsy growth in but from by X. prescriber the saw WAKIX pleased launch number in in in increase growth patients as third the and larger the our and indication and X WAKIX the of XXX diagnosed pediatric sequential continued on on WAKIX adult approximately on third to the patient favorable in We quarter. vast base QX the the what narcolepsy We're reported last of attributed average seen both we contributions the The market average facilitated to X,XXX with Slide our

extremely in narcolepsy. pediatric are with launch We our pleased

in approval, in WAKIX the the unique care solid In growth REMS first health WAKIX product year. saw health the third the our treatment and in from was oxybate FDA well. in enrolled since guidance by prescriber of quarter option strong as as third seen and prescriber strong facilitate We narcolepsy growth to we've the professional care in the patients WAKIX quarter branded writer the payer end The patients on continues patient professionals, in of the growth with product. expectations the the the to the coverage the reaffirms pediatric beyond non-scheduled expand oxybate. We line only community our the getting of our that in profile X,XXX base and WAKIX beyond the interest and average of confidence quarter base segment approximately market average demonstrating saw patients

growth continue approximately health care enrolled the through of drive XX% now of see are within generic oxybate represent this care of third at the health than We from additional seeing the penetrated to the And WAKIX quarter with highly professionals beyond we're quarter an more we penetrated professionals, segment for the the growth among segment. to new Coupled care utilization in in care each that X,XXX availability of X,XXX and options. oxybate professionals continue oxybate audience segment and this opportunity enrolled prescribed to continues the quarter. approximately with tap we narcolepsy to insulated even REMS of end durable performance. this health health being professionals, each see patients prescriber this and We're oxybate WAKIX REMS into

WAKIX XX,XXX provides and growth for future of universe, the treating across giving meaningfully tap full broad into differentiated a WAKIX. confidence approximately product narcolepsy that allowing patient to opportunity offers utility us X diagnosed profile in professional the health us narcolepsy entire care patients clinical

Investor commercial narcolepsy we progress on we're our in with and with WAKIX on management track October model good plan to commercial alone, a our cycle along that of the Now on strong with new at $X pitolisant. formulations team Day X, life shared making billion plus achieve

products We're patients formulations new the and GR pitolisant, patient patients. provisional we GI and XX% drive benefits GR needs needs extend the benefits developing to are coding on attributes the target disease to out titration to pitolisant X Both prior is franchise. dates and for features XXXX offer to for WAKIX disturbances incremental with additional dose. narcolepsy have of the HD and eliminates to of unmet GR pitolisant a grow unmet to in pathway the quick-to-market due Pitolisant existing has address in bioequivalence LOE, built in their and of filed of patents meaningful way new patient-focused the PDUFA a around a HD underlying as each gastro-resistant know and XXXX XXXX pitolisant

achieve As therapeutic and the all The through $XXX dose new base patients to a have schedules have net sooner. to a representing products a allowing a discontinued not Both to strategy million some activate achieve GR for at clinical do patients dose commercial patient to million patients not to expand pitolisant model, did potential see clinical benefit. either benefit. patient narcolepsy or We titration unique get $XXX benefits who potential cannot to start peak growth in and sales effects our therapeutic is clinical and using to WAKIX previous GI side WAKIX. achieve GR due incremental and

EDS program to cataplexy, of untreated need meaningful indication narcolepsy of pressing drive narcolepsy is fatigue and of more started enhanced with suffer with of Xx up HD to pitolisant no largest an development efficacy the optimized enhanced even dose pitolisant an A in a titration market, coating designed gastro-resistant formulation from to profile patients is WAKIX deliver to is XX% dose. in that with and features address the efficacy. The higher the up narcolepsy to HD therapeutic for and fatigue an address fatigue greater need in PK and narcolepsy targeting a a that which

professional the on the opportunity showed and dystrophy. idiopathic indication as market product pressing health profile the with addressing with grow our CHD to unique out need peak current commercial most health is the previous We myotonic in the net efficacy expected larger hypersomnia pursued mid-XXXXs. potential and pitolisant durable a unique revenue fatigue $X WAKIX narcolepsy to for than to WAKIX and superior HD patients broadens Preliminary sales payers even and research new patients HD market patient patients, care profile, the product in through the plus an more extend other given for due professionals use. and the indications strategy peak billion alone HD in and greater growth being The model of patient a base its target care see

to of HD patients sleep/wake significant new Payers saw offer the majority pre and the the The profile, all growth WAKIX the care utility WAKIX patients the for durable generic in our access of through in and WAKIX deliver reengage current to LOE. into expected broad Health poised HD professionals for favorable and WAKIX strengthens LOE and patients, start revenues stepping post is transition saw majority leadership pitolisant both value also franchise post previous patient mid-XXXXs. to without HD pitolisant and expected and vast patients. product a to position

sales summary, durable in patient and exciting with building our had and franchise. our to in growth in $X patients another quarter net performance So we're narcolepsy. path to an and continue adds Heading towards quarter, plus net strong We of full living sleep/wake in in of ability WAKIX. fourth sales, growth the prescribers guidance, we're help confident into year billion our

Budur, of Medical to Scientific like to turn Kumar? Kumar to development now would and call advances program. clinical the discuss Chief over Officer, our the our I

Kumar Budur

everyone, to progress make and thank day, R&D, joining advancing great today. in our you us In Good for we program. pipeline continue

across with As franchises need. development assets programs we under now Jeffrey high mentioned, rare XX medical on focused have X had indication X neurological unmet

X have potential X ongoing new the or more easier III will X this We products the to studies X Phase launches distinct indication or registration deliver by of years. with the year next end indications in new for

development #XX, Starting clinical shown Slide total and the XX. highlights on are with feed-based is income XX Slide pipeline through Slide franchise. Our our development on

idiopathic track by to an We this end hypersomnia are sNDA of year. the on for submit

based sustained from Day that extension X long-term study. hypersomnia data patients study reported pitolisant INTUNE the idiopathic pitolisant that X in October with consistently Investor patients than registration data efficacy the the more will of of new with robust we our evidence efficacy on X, Phase At III strong and hypersomnia. lines and submission support on Our showed in out of idiopathic be year in the additional

approximately X maintenance sustained now the out similar, normal scale measured sleepiness was points scale shared majority of in as beyond study. improvement from the and in strong long-term ] in fleeting range the zebrafish of with in questionnaires is efficacy the main by idiopathic hypersomnia [indiscernible] year [ and its X The patients extension effort baseline

data a study better patients and also stable XX last patients with with that as of addition, [indiscernible] approximately with Data from IH IH. of patients the XX% over monotherapy. In from remained benefited pitolisant shared got pre-clinical shows independent we this XX% these in pitolisant over with and

dosing in really safety a only with IH. efficacy program established IH the data patients of patients pitolisant, compatibility condition totality The with its regimen triple that unmet drug with controlled for nonscheduled nighttime treatment also alongside use a favorable a X widespread benefit for efficacy was with for observed has the simple ramp a and offers of application Similar safety a approved challenging profile benefited X proposition associated regimen which potential high patient need with very the profile. with to with a address in risk dosing off-label has are the Pitolisant issue. of streamline,

Moving to study PDUFA their pitolisant used XXXX dose GR and initiate in to track the thereby Day in to of for WAKIX, quarter data pitolisant preliminary HD. Dierks at establishing month, development Jeff with pitolisant unit in current formulation, the we pitolisant expected margins of each deliver. formulations earlier this value development safety highest pivotal on safety on With the labeled study and development optimization first are dosing we Investor HD HD program, date GR Xx up the to these are our XXXX. program. With next establishing pitolisant proposition, the of described bioequivalent

narcolepsy and GR IND-related to have half PDUFA the further a pitolisant track XXXX. on on with of for until Permissions study system HD potential we submitted working target with and in both currently XXXX. the pitolisant safety are activity, been illustrate in protection patent second and in XXXX formulation a We optimizing are pivotal

all orexin-X greater PPMXXXX. agonist. phase program, data our as upcoding to data known that orexin-X much disclosed pharmacology agonist formerly The publicly to in-vitro compared on is on Moving receptor BPX.XXXXX, demonstrated

the of potency the you emerging and is parameter and did the to accounts all that orexin-X excellent over orexin-X an most dosing As across demonstrated talk hypersomnolence fold know, flexibility disorders to other us important receptor dose. was [indiscernible] year human target anticipated maximum disorders potency we This than compounds, based at of give the potentially on ability the consistent central The over XXX-fold XXX this at translates have and selectivity greater receptor. margin of .

over its a targets BPX.XXXXX chemical receptor In potential of for selectivity addition, the structure, demonstrated one connectivity, best-in-class a once a and be XXX also has it potential orexin-X robust to with dosing day other novel potential fold for [indiscernible]. agonist. the excellent preclinical X,XXX over and highest has potency, data interest

mid-XXXX on towards are an track filing XXXX. second initiating in and in the IND first-in-human of half We studies

franchise. Today, earlier And we we DEE. are mentioned the advanced in development in as the very excited to have highlight presentation, most epilepsy [just] program our

of X Clemizole efficacy validated new EPX-XXX enhance EPX-XXX the epileptic XXX% epileptic serotonergic that and action moderation The EPX-XXX candidates, is is encephalopathy. serotonin EPX-XXX XXX one treatment a or value. and mechanism mechanism predictive have developmental XXX% that Hydrochloride, [indiscernible]. positive model of the developmental action and [investment] negative in of for the We and of has well-known zebrafish in shows encephalopathy. of

in In defaulter addition, in for EPX-XXX it preclinical X mechanism also a on utility comes protein approximately other significant exposures. validated years for of headcount from with DEE finding and safety tolerability post-marketing suggesting XX clemizole EPX-XXX action safety [indiscernible] no as or When such experiments we to market was the tolerability, in and broad DEE. is have

III is to and simple [indiscernible]. liquid additional especially in with we dosing the monitoring. or a registration ongoing favorable study studied take the need with including regimen formulation EPX-XXX a BID in addition, meaningful syndrome that tolerability [indiscernible] special no compared Phase [indiscernible] Dravet preliminary and for domestic, DEE [indiscernible] In laboratory X patients, drugs in X safety dosing for of

of year. recruiting actively are this III Dravet registration and track in on the Phase start in syndrome, LGS global study a ARGUS to by III are We study Phase registration the our we end globally study for

strong track drug the efficacy, top and XXXX on for dosing investigational BID tolerability and advanced most promising DEEs. most line in safety for promising profile, and the The for evidence makes

well lorcaserin agonist of more in established X have product, pre-IND and EPX-XXX selective liquid [indiscernible] DEE. the We formulation is phase. or is a action investigation in the mechanism Lorcaserin

the [indiscernible] the in And preclinical in in FDA we which expects following encephalopathy. in are interest exploring have XXXX zebrafish neurology model. in in and experiments the lorcaserin developmental cadence, epileptic also We

currently are recognized we important EPX-XXX. and the study It pre-IND safety unmet lorcaserin. the lorcaserin and upon of plan EPX-XXX The for the is DEE phase, potentially to We short-term, long-term note rare [indiscernible]. well-established [indiscernible] [indiscernible] real-world patients offer designation for and EPX-XXX with the FDA. it designation is DF based with with to and designation for need that agencies Accordingly, tolerability with and [indiscernible] and both often LTS an by also outcomes so could and EPX-XXX orphan on regulatory decision by and pediatric EPX-XXX to from refiled for BS the disease has has LCS both the part and received , the [indiscernible] pediatric has FDA. FDA on by

Finally, syndrome. patients X with Fragile III report we in Syndrome ZYN-XXX only RECONNECT new for symptom of approved in this the Behavioral Phase and trial line to X mid-XXXX. franchise, If remain registrational top with treatment track from be approved, data Fragile any the in on our first will

study there We syndrome are in up disorder Xx. which track Phase prominent Behavioral for to deletion with Neuro XXq on by also to rare registrational symptom are now initiate the III the under XXXX

site and us development to our I significant In like who the and On well personnel and look pipeline in our programs. clinical and HARMONY, for in all of families months more we forward progress. summary, helping would their their coming X are advance efforts as to continue our clinical and commitment make participating the investigators to advancing sharing trial our patients in we made behalf years as thank in [indiscernible] across late-stage the as have progress

Kapadia turn I'll now call our the our CFO, update for an financial to Sandip over performance. on Sandip?

Sandip Kapadia

you'll we morning, and This third earnings our quarter morning, the and our results. release XXXX of Kumar, our details operating XX-Q filed quarter financial where you, Thank and good third everyone. issued find

quarter the performance to biotech our top fund Harmony the profitable, Our line financial a is and another fully double-digit unique company, through growth, in achieved continues We're community. on strong growth maintained and delivered We profile have cash-generating balance pipeline also cash of able our sheet. a XX advancement off Slides shown profitability generation. of robust XX. to

commercialization We us excellence balance million sheet well million WAKIX. narcolepsy. third revenues in reported the advancing net Performance prior the for strong quarter along financial in continued $XXX $XXX.X Our of the to underlying in XXXX and driving with performance in positions pipeline, the continue industry-leading for strong position quarter WAKIX our to the demand year compared quarter. reflects of of

of also for the million total third as XX% quarter reported to operating representing million same $XX.X increase. for in compared the We expenses quarter XXXX, $XX.X a

release non-GAAP $XX.X the net $X.XX or The of per adjusted compared to reconciliation continued growth year our third commercialization driven results. for narcolepsy. in along GAAP share our was investments reflects with the We pipeline, million non-GAAP quarter. for of was share net expenses income in in the income adjusted by of press Non-GAAP primarily believe performance. better to quarter for a diluted per prior in XXXX investment WAKIX underlying $X.XX $XX.X business the Please or diluted refer expanding to late-stage million

$XX.X our balance strategy strong to financial our on grow million third and the to us execute provides We investments. of million which reflects ended of pipeline. cash $XXX.X cash continuing quarter with flexibility of the equivalents The cash, operations from generation

to year. Looking this in expect we quarter-over-quarter continue in QX growth ahead, of revenues net

expect fourth also increases as We registrational R&D Phase we during in quarter. our initiate III the trial fourth

towards narcolepsy once alone. progress $XXX revenue net We $XXX $X reiterating adult in XXXX to plus million, billion guidance for highlighting are million our of the opportunity again our

the for with Jeff? I'd to closing And that, like Jeff his remarks. to back call turn

Jeffrey Dayno

Sandip. you, Thank

Harmony stronger. momentum our up that our want to has wrap emphasize we I been never As call, at

We is to unmet portfolio our this that industry We in of in of believe and of advanced portfolio need. space. a represents and by in committed innovation driven the for continue to area assets this DEEs are fueled we the and epilepsies sleep/wake most rare narcolepsy that robust medical the in serious by in our next strength the wave lead foundational WAKIX business of of

in in the And finally, exciting pipeline more to with hypersomnia catalysts first year. X late-stage launches year and over of indication half our Fragile poised is or next next catalyst rich deliver coming X idiopathic X the years products syndrome each or new

and just Harmony's over the we have growth you. We will to operator. getting story. will And And back conviction proven talent, that the call to fuel the started. now turn are Thank continue resources I

Operator

first from come Our Graig Mizuho. question Instructions] [Operator will Suvannavejh with

Graig Suvannavejh

Congrats progress. on the

Your if pipeline but just I X pitolisant GR ask start, to and could don't know HD where the I is so on strategy. deep. maybe

going we the And As can how just you we sense to when in of when to how positioning the like how on manage a time? kind then commercial just look evolve give to products horizon, coexistence a what and is then X in how basically are it kind potentially comes just visualize hopefully the of to us look position is of the if in out on going have the you GR XXXX, HD same products? you're trying at X market XXXX, might WAKIX of that launches those strategy should visualize

Jeffrey Dayno

I Dierks Jeff to unique how turn terms explain on A commercial lot of our handle you that. to think on that to it enabling can to Jeff that I'm further? us going And expand in based is strategy. to model of

Jeffrey Dierks

achieve patients a have product advancing thinking where see converting patients Pitolisant cycle these necessarily to our activate We and side established to not we about a opportunity GR actually WAKIX in but are pitolisant to $X going is opportunity be billion-plus asset, or GI are obviously, likely on WAKIX we our physicians an So using business, effects dropped base, model can who and to clinical in commercial life and foundational formally off going you're target due management going opportunities. to as next-generation benefit. patient as XXXX. may be GR didn't PDUFA where who the GR, be pitolisant if is that WAKIX from to there accretive expand see that that

We're coming patient as new the titration. that tap growing audience as We patients. a coating going ability into office, benefit any and as to having well WAKIX GR look to as of in into no WAKIX a well previous with see patients new physician's with gastro-resistant the

product products indication doesn't to that deliver with That to patients. Then HD fatigue available existing professionals, when profile the of today. opportunity front care in as PDUFA the to XXXX, is in over treat put -- as converting WAKIX saw WAKIX that a that given So any of to an product engage a saw potential superior majority efficacy, they WAKIX patients. not date embraced that we another converting patients enhanced likely profile when potential product the come they previous to health their looks market they have vast this that to a target

new And patients. they for obviously as opportunity existing also an saw this

and seeing end given patients asset are to that's going and where see kind potential end They coexist. is narcolepsy. and opportunities to to and that, So accretive that products. patients to how up to we're is the of these up meaningful benefits able just offer likely are be GR to with to enhancements be going going be WAKIX the we incremental the coexisting. HD most product going able

Graig Suvannavejh

I If just in could Okay. one. squeeze

orexin the program, about. of investors have on Just been asking very interesting, lots

in see might picture Just what are of that I you provide the about to program? additional or present the able And data? you're know that when what that you comments fuller ability preclinical exactly further we on when hoping the to assets, a you data show present Any orexin on see desire on additional have to data the opportunity. perhaps talked

Kumar Budur

At margins into additional intent is and at available XXX selectivity which receptor meetings. XXX and emphasized translates targets over than agonist, data potential where orexin X,XXX-fold presented we on scientific agonist, anything for interest highest to great with dosing. orexin-X structure, The we receptor compared also XXX fold, data better some the the also our the more than fold to disclose that selectivity of is potency our Investor on upcoming chemical novel QD over Day, at receptor publicly orexin-X other

providing We be when information we soon. will but where, that haven't decided and

Jeffrey Dayno

is at target that we're that we're the and asset. with working scientific Bioprojet, think profile partner, to share looking year next of to our preclinical the a for of full I meeting sort

Operator

Our next Needham. from Fadia with question Ami will come

Ami Fadia

for plans petricaserine you to other And your asset Could only LVS/DS some EPX-XXX space? on [indiscernible] ], particularly can and you seen of about terms development plan as beyond you clinical we've you [ this talk for of in gets DEE differentiation strategy how excited safety development the versus not what about approach talk and the

Jeffrey Dayno

strategy to bit. Kumar in try investigational excited the our the will little that what's and now other to we regards we seeing and in program. some of turn market where in compared some Kumar? just I'll to unpack with terms the profile a level EPX-XXX we're are the advanced high to Yes, We are of products. of

Kumar Budur

there. several asked you know questions I

in by So need unmet of saying start space, let [indiscernible] our validated terms tolerability. high me that, recent several docs and look, in developments have encephalopathy, the efficacy, safety developmental significant presence in despite limitations epileptic

the in has and area. in comes EPX-XXX to several said, ability gaps that's this fill So

talk of new signals Clemizole no been years introduction foremost, has was for to generation safety. with develop me asked and consulted over First [indiscernible]. FDA safety It in about the the let chemical of observed. and antihistamine. us two, this market Number were XX second hydrochloride

So start a toxicity Vegalta impact. June include non-chemical and battery in safety study no we of repeat full in that, observed. also X-month [ X repeat [indiscernible] ] dose of dose support that studies, study end signals conducted were month

I study in We healthy also Phase had a volunteers.

and product. being Here, this and U.S. development is of This and further we supported study saw recruited outside in acceptable safety tolerability also of U.S.

So monitor test Echocardiography liver it a comes ] cardiac having Teva, function look drugs not offers disease-area contents monitor also [ went and having Phase and showed us data at for in disclosed of to benefits and any significant like we of protocol safety the study in not regulatory also through it to FDA extension this to to study EMA. when we from III monitoring. lastly, And additional to of some Dravet hypertension. Epidiolex ongoing long-term none cardiac from And syndrome asks the couple do terms pro routinely where the like preliminary value

established early In and while better terms EPX-XXX Bexicaserin from differentiates profile stages in is of gastro evolving. how is profile it perspective, safety still specifically

in meaningful, development. nature along this Second, third clinical the especially and EPX-XXX of because which clinically thing was is And the BID is the important. very BID, disorder. is, is encephalopathy with further in EPX-XXX the severity administered, [indiscernible] epileptic developmental is patients of in

LCM. recognized study study the in designation orphan syndrome. for III given we the syndrome both drug Phase been and are patients mentioned rare actively and call as regulatory pediatric the earlier, in recruiting for And designation have EPX-XXX promise the Dravet we We to during DS in US Lennox-Gaustaut of have and assistants have and and start a about Europe, disease in

Ami Fadia

you other could approach how address DEEs? Kumar, you also might

Kumar Budur

data protein example, call DEEs, I earlier, Binding have the the preclinical mentioned disorder we show to In for X EPX-XXX [indiscernible] in where instant that data utility other of our evidence Right with study is well. to III we LGS. and focus by [indiscernible] CDD Phase Dravet similarly in complete on in the syndrome now, as focus efficacy saw for our and

of evaluating nature disorder terms in epileptic forward, the development terms approaching in go determined In will all study, of be pursuing the those not have that because but opportunity the are DEEs, we to how the in we other of cytogenetic process [ ] are encephalopathy exactly of we EPX-XXX.

Jeffrey Dayno

the and for year-end. and in basket Dravet stage add, the line and Phase to DEEs, strategies just I broader the pivotal on would late is think go think III these study opportunity there. in the absolutely program study focused DEE's in development I that is more sort which are the of but And now, potential broad then I before syndrome trial opportunity the But we with start LGS track to consistent initiate

Operator

comes with Our next Fitzgerald. Cantor from Duncan question Charles

Charles Duncan

percent perspective And the writers? I is end didn't observation. question, percentage and because congrats start of quarter. attainable to of regarding But would a have the will on in and pipeline the that that? what added kind why it narrow curious oxybate your progress the then the because make all where what the I Jeff from tough XXX products. patients, team, patients, modulated with the new non-oxybate quite is I'm and commercial know the of come seems guide on you like to over It's start the versus low

Jeffrey Dayno

are the in of Jeff, from? comments terms patients on coming where

Jeffrey Dierks

pleased the from added obviously, approximately more in health being XX% patients with so in we're both oxybate professionals. enrolled XXX enrolled that's extremely average tend quarter. REMS maybe Charles, lot of we added reported the nonoxybate. care from the of are patient of group. patients probably second they from patient We coming in seeing saw larger sleep segments in professionals. growth specialists, are there's from And we third It's in care health to health the About larger what oxybate we're about have care patients incrementally new Their those the And And quarter. function REMS professional practices. of XX% terms growth a

we're a of depth this in But XXth, We XXth with physicians audience. growth we to Xth, of all group have most new we really patient churn. see that that the finding REMS-enrolled oxybate to continue to meaning They're insulated experience excited Xnd, know of the coming prescribing, see professional continue these WAKIX. health area. with the tap continue patients an even that care nonoxybate we out have We into in

on And continue so tremendous we're forward. to the diagnosed audience growth into and tap extremely moving pleased. patients, XX,XXX we're We that horizon. opportunities see to going

Jeffrey Dayno

comments Yes. WAKIX to patients to universe with calling Charles, continued on. on the and think Sandip, clinical the hold guidance? utility pattern I would true we're with for in I narcolepsy of that just add, regards the broad HCP on position

Sandip Kapadia

QX. call, relatively range even mentioned going I'd continue from quarter-over-quarter which of sort right, the is is the X% sales, range. our growth into right look, million we on narrow, to in I expect Sure. $XXX as mean, was really I What mean, I say start overall

we good about range. the So feel

where good comfortable. see range provide update, to there. and an momentum, up continue and once the the We now, feel we'll about year we're very on ended we right on obviously We closed good

Charles Duncan

question to XX Dravet little And may remote. bit a I III data ask I'm is with Phase a Kumar with mean the population. XXX. study. with quite regard patient the of intrigued ongoing really I

and characterize the And trial going? mTOR to And able provide OLE, you enrollment is then enroll can persistence you into patients on any burden? can is additional how inhibitors. that that how going? you are a color seizure Are Epidiolex on So

Kumar Budur

projection. recruiting the after mean, is trial I

terms standard And syndrome in the recruiting study a study standard any patients design of other are Canada Europe. U.S., the pretty it's We criteria Charles, and inclusion/exclusion and actively Dravet is compared that in design to studies. population, patient

has of terms duration medicines these In titration on seizure the average the the the number criteria, inclusion/exclusion study, patients of [indiscernible]. of an

actually X what to between seeing medications, and they that's X to good therefore anywhere our much average, need we trial Our on be and antiseizure last clinical pretty are tend and of medicine very in and as the impact that things well, of data, well. some in disclosed very promising on is profile tolerability safety we EBITDA as supportive of earlier, of the as X mentioned the

not signal. pretty [indiscernible] As of appetite tolerability have patient with feeding no the help are difficulties there saw many What already of you know, there is or out given with were their appetite weight and safety other loss. population it have we EPX-XXX this challenging, have did us days, that and

recruit as target as So are we to overall, very soon to I possible. line. possible XXXX the Obviously, top as the how to bring pleased is The medication try as and study go recruitment this patients soon with the going. will is

Operator

Amsellem Our Sandler. next Piper question with will come from David

David Amsellem

generics about net So the as better first about question us in price about market. move payer it through you're you're is on you're we the help sometimes and of how thinking landscape, and understand thinking versus sodium where realized the that particularly in oxybate 'XX with is landscape XXXX thinking more particularly now? how how Just

So the that's number market future. not-too-different sometimes -- in in the

So that's one. number

dosing. clarification wanted pitolisant ask for two, And and a question HD then I regarding to number just

in at dose of think to earlier talked that's R&D WAKIX. correct. that have could said than dosing about higher the make you that higher. And I you Day, the safely I highest call, Xx Xx dose sure want think the just I to you up that I

wanted pitolisant legacy think realistically you So the HD relative of could where to get sense dose just a formulation? to better you

Jeffrey Dayno

Jeff, do you first want to question? respond the to

Jeffrey Dierks

we unique So branded of we landscape very started of about the next oxybate. we already what landscape David, to lot lowest Again, a necessary. year. XXXX. be negotiated There's the And payer for we be position next and with incremental have now to similar very the to as contracting see product of a WAKIX. lot relative not respect sort to for the to year's that's right contracts see thinking [accor] have A

on has than cost expensive think contract. have WAC us less oxybate to a generic we're that position the branded opportunity So I the not afforded and and an

based is what the EDS payers in finding it's are oxybates there. the respect in forward generic WAKIX a in think a market favorable opportunities and be have 'XX, place negotiations we that any cataplexy, to or heard for plans new option to anticipate may We XXXX with landscape WAKIX oxybates payers on and So with in only that, great currently moving don't their upon non-scheduled continue coming. branded generic require that very our product. brand. opportunity that are through moving generic. grow discussions to no access formularies the and confidence And of be us us step in and There likely But is gives both we see I oxybate we've to either forward, product 'XX that going for position given to additional treatment to for to from the patients believe the fact is there maintain payers, lot and seen the for a

Jeffrey Dayno

to to regards HD safety what shared WAKIX development But program program to did up -- David, up dose. HD, the at dose clarify current and the been the a Xx maximum margins pitolisant development for pitolisant up and just we dose established is Day program. small to to we label dosed Xx to establish of Investor of dose the label in HD. with the in WAKIX terms terms with And in of the have target for -- safety maximum Xx in margins study The pitolisant the of current

Kumar Budur

Xx viability to accomplish Jeff the optimized decrease means the formulation the impact And what of formulation, have well, David with to mentioned, some a highest the not in lot with be that, like also to label WAKIX will clinical optimized add meaningful only the able higher just that in to of milligram exposure interindividual patients. things dose were WAKIX. And which which to up to as compared milligram we

Operator

James. will Danielle Raymond next with from question Our Brill come

Danielle Brill

you there do there. I better just DEEs. any with drug separate expecting particular And clarity study appreciate why might slight this on I a we guess, patient might indications I Sandip, reason Or strategy in the as be quarter? from as. for ? suited be to dip lorcaserin sequential that LGS? then your guidance on clarification the there decline a leave for next by [indiscernible] versus any X,XXX reason patients a year-end the have applied Is basket Is feel a

Sandip Kapadia

I there think not was our we We're around said thought exactly overall X,XXX. to guiding approximately exactly X,XXX.

think good going there the we any change so see I demand, fourth terms is growth continued significant quarter-over-quarter what seeing quarter. in a we're So be don't reason would momentum why underlying into of

So that's want maybe else add? Jeff there Dierks, to anything is

Jeffrey Dierks

had very Sandip approximation. consistent. double-digit We've said. Guidance very No, exactly It's durable what an strong growth. been I reiterate would growth, is

have confidence. we And of so a lot

around an to approximation. it's And again, average were the yes, XX. number of near So the patients

of anticipate So similar in QX to momentum in what we've seen to look Danielle, I would sort and QX QX.

Kumar Budur

your regulatory basket open In that we study other is for the terms the of regarding look, study, a basket approach. know question

mentioned, in we in and We one with LGS. are posting as one EPX-XXX X DS programs,

will how a based we opportunity as And We on in taking -- disorder, a exploring depending want the that to keep heterogeneous possible, already measured as efficacy are in article are the action, with the LGS on is literature of clinical liquid of excited preclinical population mechanism so have for basket study that XXXX much in the are approach is DEEs. the evidence very EPX-XXX a in humans that of but lorcaserin, rest definitely EPX-XXX evidence the patient we we of the also widely of for neurology. the [indiscernible] experience excited, an we for that EPX-XXX because also in plans published efficacy development given about for the and by formulation clinical were but

Operator

Ash will question from next with come Verma Our UBS.

Ashwani Verma

for study. in RECONNECT Just [Zygel] the

have you the I which you I studying of you're show as likely patients think to to look methylation. have patients why the well. When have understand previously, XX% methylation noncomplete at primary So could been this Do positive. the the is wanted complete just quickly label the patients have the methylation data complete a you quarter. get benefit if complete and FDA endpoint of the also you understanding on any your methylation. study from on status irrespective the And is that methylation the

is the So by up increased X%? -- is build but the there inventory X% the number from sales aggregation here any like so net added X% case sequentially,

Jeffrey Dayno

ZYN-XXX? question on the you could address Kumar,

Kumar Budur

that primary So we methylation. metalletic There's endpoint that's well, see about If and partially the on of partially normal and this supportive data our came population patients FDA. number with leave primary a the patients patients. complete agency study you does patients power. point methylation discuss target terms But broader now, in is a during population data the getting the do with the primary [lay] us complete discussion in with patient we target and in to see like is for as methalytic methylation an with that opportunity primary [indiscernible] regulatory the complete consistent end of the

Sandip Kapadia

mean I that to Just trade was trade see Typically, net. inventory of second see quarter. I not question least had normal you what that in of we terms an year, see do in some significant half mention, would the quarter, at saying gross of a every this the impact for in fluctuations I'm inventory. you improvement

and quarter the of year, second go second to be year first higher, The know, coming tends half into as tends quarter, as net the down you to the the you in gross starts be.

what you're seeing well. a probably to as bit So improvement little net gross in of

some provides that hopefully, So context.

Operator

next Sachs. from come will Our question Goldman Jenkins with Corinne

Corinne Jenkins

narcolepsy narcolepsy? terms fatigue Maybe just in in to of And how how is measure us pitolisant about a meaningful on a how within would and population, helpful. couple you clinically provide those some what maybe from context us. be HD -- just planning the clinical you're be And that around study could of benefit talk population? to would Could common you patient fatigue like

Jeffrey Dayno

HD. and fatigue for plan to Kumar

Kumar Budur

with About with is study Yes, the to the and XX% very narcolepsy. provenance and we of and of impact than a indicated The of narcolepsy with fatigue are more the that think XX% The has underway, is fatigue. preliminary X/X patients data longitudinal doing fatigue patients similar. of with prevalent shows prevalence fatigue XX have literature is patients symptom we X a narcolepsy in is that patients that XX, narcolepsy. is about significant have

terms treatments. of approved measure, there indication is we are how In do which this no for an

that to measure an specifically work So measures the that in because instruments be out Look using what there the terms the appropriateness narcolepsy. A instrument of fatigue dividing -- leading-edge are is in patients discussing the fatigue we with that that and extensive narcolepsy specifically regulatory of can regulatory we'll all lot and done specifically are at in of we an work. had be instrument [indiscernible]. fatigue patients with the that with measure to is agencies

Corinne Jenkins

that on into of terms patents of those you patents XXs? those the in just help and have kind around of could expand pitolisant the nature then HD, And the early you number

Jeffrey Dayno

regards to And I the on are unique that the the in coding. overall and profile. with think patent improvement around the with HD formulation [indiscernible] PK the along

data support is to that PK will that clinical what regards the of XXXX. the the out So to profile that sort HD And will with patents of the demonstrate. basis

Operator

Our Jason next Bank of Gerberry question America. will come from with

Jason Gerberry

Two for me.

in with potential board of to year, disease just First, respect Wondering, next of of is just just into EPX-XXX. at relative or measure no-go state. get a you to zebrafish enough go, have the a tease areas molecule as more of more plan that's some WAKIX patent. sort the of And when we curious that is trial kind something secure get sensitive 'XX. the help viability to can the construction to to that And on How and then in important crystal IP looking differentiation with a if just [indiscernible] of model? out looked rolling long litigation, infringement ultimately of an broad case goes kind hearing. the out key outcomes inform generics of you we'd curious box what markman frame in you to for there's a be of are I'm respect EPX-XXX

Kumar Budur

experience. great extensively showed studied in clinical great was Similarly, which which EPX-XXX this was efficacy, model, EPX-XXX it and model, fish [indiscernible] efficacy. also in the studied showed was

pure our not obviously, had don't XC in is to compound. data some bexicaserin, studied that brain and and communication. In access agonistic published But analog this of terms XD, the we properties some there fish literature the [indiscernible] within by it's were compound that model, that were have

Jeffrey Dayno

question the And litigation. IP about your Jason, terms of think, I in second

basically, I comment the the all, is sets what I of scheduled But next construction. trial think up mean really litigation. that does on that first claims think, we I March. ongoing it can't for [market] is

construction in the informs for sets think the in then I case it XXXX. up plan trial claims overall the and the

that purpose that the in I final. that in as we're those litigation. the there So with terms the we've strength were said of that stage potential were next USPTO think IP decisions patents, the the to, and that of I were of think in process denied is before, And challenges the X regards to of the confident IP,

to is XXXX. IP strengthened out overall position the So our

Operator

Oppenheimer. question with Our come Francois will last Brisebois from

FranÃ§ois Brisebois

help and so difficult? oxybate just to and the gauge it mentioned of percentage Can You on Is you are patients growth also more just Is elaborate that response? been has comes the that with much the REMS the both from trying that reimbursement I'm WAKIX just on that same? there's doctors. time? changed

in has probably do changes And you wondering just reimbursement orexins reimbursing I'm all any one, if these terms changed? of products if market foresee pharmacy come So into and market?

Jeffrey Dayno

in Thanks for hanging there. Jeff.

Jeffrey Dierks

years. been it's are get disease patients. at use It's looking not again, lot this steps additional we consistent. plan the probably that more consistent generic the for concomitant in to low teens And a patients landscape the given the been couple in be this category it's obviously going put there's It's to high relatively number has potentially double of of of reimbursement administrative space, percentage area. of rare obviously, of a for oxybate, introduction last them, patients. like limited the with like and digits, managed "easier" The to of are certain a and on plans oxybate lot modestly been with about But very care not remember, both WAKIX where on orphan is are, an more has costly a

how look that patients. could branded of budget forward, But categories not given be as huge tell a Obviously, there's going a potential It's of in orphan the time will this. a limited to will moving obviously, But categories other overall, there's products introduced at be not number tend be care managed area. as managed rare decade. number likely later highly to

going to HD the Maybe some can Obviously, to think that enhancements. to enhanced are efficacy. provide really be payers. substances to the oxybate, favorable, for of controlled would but amount the extremely potentially also take, reduce patients reduce which the up helping patients stimulants and amount to provide potentially the meaningful is goal end program I only the be able of hopefully obviously continue GR our not

Operator

like time, to Dayno any for this Thank you. Jeff or additional to over At closing back I would call the remarks. turn

Jeffrey Dayno

Thanks, Thanks, our joining the operator. providing Thanks, late-stage everyone, to robust we making pipeline updates. your were to rest a for future We and excited progress great and look day. are have call today. our forward share with of you in

Operator

Quarter Financial This does Biosciences Conference Third conclude today's Harmony Call. XXXX Results

line, your disconnect now wonderful have may You and day. a

Harmony Biosciences (HRMY) 29 Oct 242024 Q3 Earnings call transcript