SRPT - Earnings call transcript Q1 2024 | Sarepta Therapeutics, Inc.

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Operator

Good afternoon and welcome to the Sarepta Therapeutics First Quarter 2024 Earnings Call. [Operator Instructions] As a reminder, today's program is being recorded.

At this time, I'll turn the call over to Mary Jenkins, Associate Director, Investor Relations and Corporate Communications. Please go ahead.

Mary Jenkins

Thank you, Victor, and thank you all for joining today's call. released results we quarter the afternoon, XXXX. of this our for financial first Earlier is our and available and release press The on was afternoon. with this our website filed the Commission Securities Exchange XX-Q sarepta.com, at

operations on Joining review us forward-looking Doug Estepan, during webcast, statements. like any many Murray statements. the Dallan common Sarepta's number differ this I'd Sarepta's take today results These our materially call, the forward-looking Dr. risks and and moment and stock. of affect Rodino-Klapac. be note materially risks such Please statements Ingram, of our control. making Louise a Ian trading of uncertainties, contains and involve to adversely prices the from could on statements, call business, and slide are that beyond of which to forward-looking can these we a are which the will Actual forward-looking results

risks a today you applicable For on its with we the company update the subsequent XX-Q as Form report the description including any review statements, circumstances. company's financial most filed any SEC encourage undertake or filings. to well obligation to detailed recent does The provided SEC on forward-looking publicly as annual of and other projections uncertainties, not events company's based

will an of our overview turn And Doug? CEO, Doug now call the over provide I'll Ingram, recent to and our who President progress.

Douglas Ingram

joining thank Conference for and Well, Call. everyone, Sarepta First thank Quarter you, Good Mary. Financial . you Therapeutics XXXX Results afternoon,

you update. with an I Before let me provide begin,

by label previously and our We draft for BLA us know, they expand we labeled to were the Duchenne indicated. imminently population elevates will that therapy you a submitted provide gene than a XX. with FDA sooner supplement As have recently informed the

today. situations, will taking into call done questions on similar be As of the we quiet review, as and have into stage a in will going we not we period the we our late move be

quarter approximately million, therapies strong quarter versus revenue net announced revenue. $XXX in was this at revenue approved XX% growing So last $XXX million million, with product we of above net In of of total $XXX performance nearly revenue fourth same year. product X modestly quarter that, ELEVIDYS particular, another afternoon, the over with our from

actually in As label ELEVIDYS than plan but Indeed, is for as signaled that. narrow. our current the X- last still we will is our not is is population flatten very its elevates call, and expansion. narrow sales surprising earnings quite face, label as addressable more the on we narrow age all range X-year-old labeled This patient

last opportunity a in submitting far already gene treatment. are the disease, About few me approval to half have for will This of before Duchenne about just not the years any Let start these over other yet are yet form our us. posted process in exceeding physician of diagnosed And must available patients life-altering devastating this and of learned explain. not and as are they educated, for they dissimilar have to diagnosed, this right been age get group those the age they even group and find $XXX since demand last and therapies patient approved we much have treatment. leading June, other front says Physician million limitations, all combined. significant. and Despite the in beginning

label, a opportunity keen both from investment in sight for the access paying quite is We community a bringing readiness, the this to our bet and over X community Having obsessive Duchenne a support served years the X patient know public with in we community of all therefore, private country, manufacturing, to the are the every well has come with that and facilitate patients. that broadened therapies on of United state for prevalence distribution, in and have understanding working And off. we payers access States. will better and well life this multiyear in preparation

supplement. Moving BLA our to

of last and from age transitioning an the in know, submitted ambulation expand accelerated in the As year, application and you approval we also availability December restrictions to that elevates of label with FDA traditional. by removing to

are we yet in can outcome, midst the As this. but substantively comment of on we the I review, this say cannot

tangible and rare As one uses than we of has vision, expressed mechanism mission for echoed cell OTP, and have she Verdun office and disease. Marks have leadership has seen vision. in Center brought approval Division Marks, tools Dr. of we the leans products, where publicly Peter that words, more all for just and it of much has serious know, created the Director. one Mark's and advance service and and evidence Dr. or toward new Nicole aligned vision. Seber future And new Dr. progress the super Verdun, division repeatedly therapeutic gene we therapy, flexibility publicly that In of life-limiting to Since improved Dr. available level the the its engaged vision in the Director, Dr. accelerated of and is under a that regulatory FDA also to the of more like engagement. therapies But collaborative an seen arrived,

a XE. viable since For SRP-XXXX, disease ultra-rare gene the for to Type therapy LGMD arrival, instance, align we Verdun's trial were Dr. for quickly able a on

appropriately a sized trial, uses with and an single-arm missing XX the end as pivotal biomarker, patients of EMERGE, endpoint. Our primary the study beta-sarcoglycan is a protein

LGMD renewed emerging. call, meaningful have can we commenced As the This more dosing last with earnings swiftly forward we we move of in gives already announced us evolution our optimism that in our portfolio. rest

our last Rodino-Klapac R&D the our Louise expanding moment, real-world and R&D Dr. PMOs, from our and a progress of quarter, and for conjugated Now Head evidence PMO, for LGMD body our results peptide progress discuss including our will Chief of SRP-XXXX. advanced Scientific the Officer, in plans

that. rare otherwise which fight and you and we progress flagged goals over years to more brilliant the aware than after for yet signal disease. and the the will important Duchenne it But for this achieve be of because are excitement will If saving stolen whether potential for an in failed it science the also of That we look the to properly Sarepta stand against could the would you forward direction excitement supported. years stands and genetic the excitement celebrating be gene nurtured come. year today dystrophy. year that potential leaders Seven I this and patients the was serve. for in and therapy us. more as year, Indeed, because our gene be last outcome But a years, technology XXXX therapy promises began year's can it the and energy that important of by not question we our pivotal this a we renewed muscular has therapy. to to gene strong most progress Duchenne fledgling enormous many By progress for ago, decades hopefully for of updating transformative there set the for with necessity, of cell countless a lives still community. with are technology

I our will to now Murray. turn Dallan the Chief Dallan? Officer, call Customer

Dallan Murray

afternoon. you, and Thank Doug, good

a ELEVIDYS We regulatory to comment broader performance, stand population potential I following a label to update provided. Doug the to want expansion. I discuss ready patient deliver on Before

broader rigor learnings the new are current launch fact, and Duchenne with following as same the launch we within from successful incorporating date In community preparing label. to and payers, a are our the clinicians

XXXX. execute continued The the first delivered core to RNA quarter team Now the expected to as ELEVIDYS and on of on the launch business.

quarter we first than delivered revenue XXXX. of Doug XX% the net in mentioned, in quarter, representing As million growth roughly over more $XXX product the first

product As expected and call, modestly projected of for with quarter $XXX on ELEVIDYS net QX came in roughly our revenues XXXX QX million. the above

we and successfully our therapies. expertise simplicity contact clear key the This our therapy the another example small relatively In And institutions successful as to patients, model points treating if quarter see highlighted from needed. at the and a gene and to yet commercialize access will dwell of pleased to onetime one were of that recently our me Considering on resources is a are class. they delineated progress gene best this external citing date moment with represents so of solid the of population and eligible model you cell diagnosed indulge eligible as particular, working institutions, major an how conference, of can for to for we our Sarepta execution. execution, was in

As working its progress we the have through predicted way on the made last earnings X- diagnosed expected call, team to the prevalent population. the has Duchenne X-year-old

important of the As to context them it's these market Doug the noted upfront, potential. and indicative in as date over to not correct broader put interpret results

As he unique range. in X Other extremely only age out, potential contribute lower pointed working narrow an a this X from relative factors to to we're group. not

need diagnosis age to to group. complex Firstly, undiagnosed new a patients with patient there's and patients in a the diagnosed of proportion families very secondly, that terms being And this newly early high come journey. and quickly

because out enough diagnosed of the some of the such, As simply very of of team all they age X- don't X-year-old the XXXX. first have with in time. quarter said, to pleased we're that execution With the patients

existing able payers, within team Despite we Duchenne importantly, challenges, throughout ELEVIDYS with and go and to with continued the on seen population. momentum expected the to dose initial to come this we've the And key institutions, for build the support the patients. was an patients the community frames. see access centers label time incredibly dosing together gain the to appropriate the quarter, community to in country with from where broadly During KOLs, broader launch rapid diagnosis population to very the short we've patients eligible entire

stemming changes also onetime and faced first by executed the Our supporting franchise caused issues in our some QX They quarter. process. insurance PMO redetermination Medicaid patients with from unique the both expected challenges the team

two XXXX. roughly a net originally impact generated successfully million The product in industry in population to appears effect process Medicaid the Medicaid product revenue than been ending those addition and revenue million within the with line net had team In issues, have an quarter had the $XXX the QX $XXX from through in the on broadly anticipated. of had to redetermination bigger working

forward. the patients, however, as XX The some impact requirements, as some reestablishing patients during redetermination patients disenrollments due was Procedural process largely have going to many the disenrolled affected by XX% the with The been take COVID pandemic. rearview those which due relaxed in The patients million did Medicaid expected being eligibility appear process, coverage roughly to minor surprise, redetermination issues. for process of disruption with to gap. Medicaid resulting simply in many mirror, is these paperwork

first team we're the annual expected through the the up rest end of by success. insurance of has Additionally, changes worked confident for the quarter, year setting the successfully the

a behind U.S. U.S. was modest net PMO decrease with these product the revenue, remembering represented and it together which lumpiness QX was quarter first index of prior important Total in over issues the revenue not is the ex expected. $XX us quarter, over on both to of revenue. Taken ex million. PMO roughly This the

previously in continued we expect patterns quarter-to-quarter. see ordering discussed, As to fluctuations ex U.S.

what completely fundamentals the the of with up QX point. coming of this business in are we to expected had Overall, out line

We are the with and PMOs looking remainder to of forward our in with ELEVIDYS the again scenario out year, and process. be approved optimism will patient regulatory ready any coming community to the X support of the

future. in and launches drugs the the label broadened adept X As serving we approved is while Duchenne team Doug currently at potential we patients, to successful opportunity a preparing become diligently mentioned well has can now our his executing the of very prepared Having a know broader working remarks, the to serve population team on opening be Duchenne expect patients. for from

of year expansion, pave the payers, In the working with a for time had the of team from strong to And community expansion, the access institutions experience. the We're with patient is base. have gene close and readiness to case label key label ELEVIDYS more by initial way site a will Duchenne potential working the therapy to stakeholders learn the our education the hard from broadly.

the forward our up the of Sarepta proud next As be a forward pace. stakeholders occasions, to us potential accomplished We're community fuel in date, community across inspiring part individual this to it's that is our of exceptionally their ready why been very provide we're we we'll been label year to and future. propels expansion. and we already, the a chapter and what we've busy mentioned on all a This the response keep of are number Sarepta. to has looking need for It's everyone at stories much of near the they've but team entire

Head and And Louise over that, the I'll of our Officer with call to hand Scientific Rodino-Klapac. Dr. R&D, Chief Louise?

Louise Rodino-Klapac

Thanks, Dallan.

progress treatments. make to to continue of great new of with with lives supporting these treatments advancing our along the the impact science We potential the understanding patients,

approval. conversion in submitted opening of and the therapy restrictions of in requested generated December benefit the that his ELEVIDYS ELEVIDYS the clinically of for a that supports similar but trajectory any changes for is in The toplement or is totality mentioned of Doug treatment label As BLA the age. demonstrating data and remarks, traditional regardless last was of age to year. a Duchenne, disease-modifying removal ambulation meaningful We

that treatment. from believe June all we The patients Therefore, Duchenne action XX. is benefit date with can

the However, that had informed agency than we've us be sooner sending draft indicated. been they by previously they the will label

to ongoing We continue advance SRP-XXXX. with our studies

ENVISION ongoing Duchenne occurring XXX Our U.S. well. study SRP-XXXX, enrollment U.S. for is progressing recruitment with the called complete is remaining ex

is in randomized, and evaluating nonambulatory older and study two-part global, efficacy Duchenne. As individuals placebo-controlled a gene a therapy the double-blind, ENVISION SRP-XXXX safety with ambulatory reminder, of

limb-girdle or to programs Moving now to dystrophies LGMDs. the our

a for SRP-XXXX, agreed a study but endpoint sarcoglycan EMERGE III sole in other and of XE muscular of is not LGMDXE, of not important, to membrane, As in because protein the also the SRP-XXXX, call, Phase The The contraction. or treatment the missing, sarcoglycans. on results ability of multinational type of pipeline, to form with a expression only muscle our study, the is of expression sarcoglycanopathies emerging absence progress NFXX has also which for When open-label this at just it protecting is primary beta-circoglycinopathy. extremely sarcoglycans of demonstrate biomarker together known other the the is the LGMDXD XXX, during the delivery begun quarter as muscle causes or the program, trial LGMDXC. ability for is disease. important we cause but essential beta-sarcoglycan the limb-girdle functional clinical dystrophy mentioned fourth dosing beta-sarcoglycan complex of This loss including reduced function beta-sarcoglycan in and for our small

the approach protein, of such for native sarcoglyconopathies of Our the cause the great the membrane, restore the as or therapy can from which nonambulatory, delivers beta-sarcoglycan, restore missing possible it's disease. XX ages any study If is gene promise root exists Currently, older treat nothing EMERGE X ambulatory complex participants the who sarcoglycanopathies thereby that the functional The the are specific muscle. and LGMDXE lack missing suffering restoring for of function LGMDXE. gene LGMD. for the the to protein, pose effectively to or to codes other enrolling and of we a individuals

a study this programs for LGMD broadly as pathway Further, potentially development a future for blueprint viable pathfinder and other a for for the gene design of the regulatory of more functions our rare and provides therapies diseases. ultrarare

groups, the a included group history our death. of XX most survival in history was These that Moreover, eteplirsen treatment natural are XX natural Eteplirsen patients especially be to for lower patients muscle The resulted Overall, our a XX.X analysis X.X of on median XXX the for compared sizable nearly a than of rare in in longer a and in are for disease. to XX% to speak leaders over published context age whom eteplirsen-treated treatment years The X,XXX evidence deaths had This The the survived analysis nerves. patients the with to in I death the group cohort. analysis Duchenne group. patients treated individuals a updates significant to reduction compared distinguished In statistically survival Before patients. and years RNA natural eteplirsen a the foretells. risk in benefit of risk controlled history a survival of natural found analysis, programs, published with Duchenne had eteplirsen to treated peer compared real-world and generate patients. and of history. observed. XX% recent history years control aged real-world focus was thought the treatment age-matched journal continue likely natural we XX.X to the years moment Duchenne reviewed in that

eteplirsen greater to patients in addition, In group of in have indicating lower the patients patients treatment survival eteplirsen compared reached In with X treated associated time with X-year greater an duration death that treatment associated eteplirsen initiation with that treatment at longer in not years risk X with analysis X-year with treatment exposure. benefit. and than treatment X- increases with was survival median and survival time. showed than of of The years than the XX% group were eteplirsen longer less the the of earlier of longer

measures addition ambulation, function lung In improvements when standard data, to therapy a natural this life care quality shown the and meaningful of peer With to dystrophin and survival restoration from reviewed analysis of that various delays and leads compared for data eteplirsen, we're to other history. is observe to survival loss published eteplirsen or latest has divergence treatment real-world exposure to natural of time continuing the with controls. in to in associated history

PPMO. announced study SRP-XXXX, Now MOMENTUM continuing results in is RNA B our PMO to therapy with from based a SRP-XXXX, to of and we best-in-class investigational of NDA. forward SRP-XXXX date, an Early an we pepticonjugated path represents Part that data efficacy MOMENTUM generated our quarter, or from we a platform. we the believe first our for the study on And an ongoing perspective positive study have XXX.

will and an with this to previously, this we place quarter take plan meeting mentioned, approval the As in the year. discuss agency third of accelerated anticipate

to and data have are shown that results less there across dosing. XX% greater comparing with trials, different while believe SRP-XXXX, of the We patients. results limitations meaningful For to clinically have frequent dystrophin translate production potential these for

reinforcing levels that nonambulatory for patients, restorative addition benefit expression dystrophin therapies. all could and both similar patients saw also ambulatory dystrophin We from

are EXONDYS both and post-marketing trial the on as The requirement commitment for ESSENCE casimersen and golodirsen PMO. for our fully as with track. well studies are for remain for conclude post-marketing post-marketing I'll mission enrolled

we patients the of our around disease. take to to like to living with R&D team a the diligently dedication work rare thank mission summary, the our advancing portfolio. months entire goal to forward my looks to serving their colleagues advance world ahead for as unwavering moment In our I'd with

now on over an I our turn Estepan the will call update for financial results. to Ian Ian?

Ian Estepan

for website on everyone. press and details refer GAAP full basis. available non-GAAP . Please well on a to LRK, Sarepta's release quarter our a financial press GAAP the results to reconciliation first of release XXXX of afternoon, basis good provided Thanks, afternoon, This results. the as financial non-GAAP for as

estimated are based the each applicable of benefit rate. represents income in For the pretax no the from XXXX quarter beginning company change which adjustments Non-GAAP income taxes updated tax has excluded the in-licensed reflect income The XXXX income rights first this fourth added the quarter expense longer tax tax the non-GAAP effect of have for to amortization awareness, adjustment and of on for non-GAAP results. financial purposes. effective of results comparability been or

$XXX.X million. the ended XXXX March Net franchise the $XXX.X net compared to revenue ELEVIDYS the revenues first company revenues compared months revenues was for $XXX.X of an the skipping of period revenue Net X for which product million quarter and of million, other of increase from of product $XXX was of of product $XXX.X the to the same period For revenues for $XXX.X EXONDYS PMOs our revenues total from million XX, recorded first and XXXX, XXXX of same million. collaboration quarter XXXX. consists XXXX, million for

of sales were The product the net $XX In of XXXX, XXXX, increase of early the a VYONDYS XX. quarter Roche $XX.X primarily primarily for revenue with ended the million $X quarter the million net ELEVIDYS. March product first product to of million the ex million of collaboration million recognized development compared declining an to revenue other for first XXXX quarter of for in The AMONDYS incurred $XXX.X net option external related acquire recognized and the million rights XX revenue the XX, for associated For the certain program. to Duchenne during same reflects U.S. $XX.X revenues we revenue to reflects XX, $XX XXXX. and individual EXONDYS period sales collaboration

from recognized included be the should we Roche so agreement, reminder, going it all forward. not upfront the a payments in As models your have

Roche. agreement of the compared $X million for supply totaled ELEVIDYS XXXX. associated contract and of co-development recognized first batches XXXX commercial manufacturing quarter also delivered the for of costs revenue period to $XX.X under The to Roche of reimbursable million million the with $XX.X collaboration the We same

and first we of million share XXXX, million a income per reported $X.XX or net a non-GAAP quarter $X.XX non We respectively. a or On and the basis, first $XX.X reported of compared net diluted net diluted or quarter basic diluted and $XX.X of a first loss quarter and XXXX. share basic per the $XXX.X loss per per per $X.XX income of GAAP for net XXXX a of in GAAP $XX.X diluted XXXX and million share of $X.XX basic $X.XX share the or million net of in to

the approximately increase in payments In the compared of product, quarter March ELEVIDYS to same our same we XXXX, and reflects primarily certain XXXX of of our of period our ended sales million months of and in the in of the when recorded XXXX, first with demand XXXX, an royalty no similar specifications in batches $XX.X of write-off increasing during in cost XXXX. XXXX. period with cost X through activity for increase the in million compared products $XX quality XX, sales not sales increase in meeting The

were partially million. same for million On $XX.X batches XXXX, capitalization respectively. expenses primarily first of a of decrease of R&D a of of XXXX, and after $XX.X XXXX expenses decrease to ELEVIDYS ramp-up approval our $XXX.X June million year-over-year million basis, quarter commercial period first by $XXX.X basis, due $XXX.X trials. for offset in quarter million. of of non-GAAP for of A million to XXXX recorded On $XXX.X R&D manufactured the and clinical XXXX, of decrease is the GAAP the compared we in a the other The late-stage

consideration $XX.X to basis, a of the due first million quarter XXXX. million. an non-GAAP of was SG&A. the $XX.X period for value for first XXXX for charitable million compared we XXXX, approximately SG&A On $XX.X remain driven same in contributions. contingent and launch the of million with quarter the million approximately $XX.X basis, recorded in $XXX change for a GAAP as to ongoing event and and as of in increase potential recorded as the long-term The an and well cash million an litigation and restricted XX, investments XXXX, have a other the label increase a by the of elevates of increase primarily XXXX the to basis, liability. increase increase million $XXX.X first expenses support We quarter for a of $X.X capitalized of of for in period This expenses we Now March for timing XXXX was income an used primarily successful respectively, the million. the services cash, launch On billion turning cash we to On of GAAP of of XXXX, matters equivalents in $X.X $XX.X net the were well compared resources on to hand same fair our expansion. in professional

And call with closing comments. for turn back Doug? over that, the Doug to I'll

Douglas Ingram

joining very you for and you evening. us Thank Thank thank this Ian. you, all much,

entertain stage given our I a As mentioned the now, we this this quiet call, right beginning BLA evening. at cannot in period and of the of we're questions review, supplement

at be As draft I we ELEVIDYS mentioned also label the very to our should shortly. receiving beginning,

I ones. to await thousands next once of you updating be will the supplement enormously So access review. forward all Sarepta, . Certainly who therapy. this for families importantly, more to eagerly we but of conclude our months few consequential for the BLA look Duchenne

evening. good a have and well, be that, with And

Operator

participation This the conference. today's does program. Thank your you conclude for in

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

Sarepta Therapeutics (SRPT) 1 May 242024 Q1 Earnings call transcript