Rocket Pharmaceuticals (RCKT) 10 Aug 212021 Q2 Earnings call transcript

Welcome to the Rocket Pharmaceuticals, Incorporated Investor Conference Call. My name is Vanessa, and I will be your operator for today's call. At this time all participants are in listen-only mode. Later, we will conduct a question-and-answer session [Operator Instructions]. I will now turn the call over to your host, Mayur Kasetty, Director of Business Development and Operations.

Vanessa. you, Thank everyone. Good afternoon, Mayur and Kasetty, Development is of Director Relations lead Operations Investor and at This Business Rocket Pharmaceuticals. joining Thank for us. you The programs. is updates purpose call and of key discuss to on share clinical our this

use use are those our statements of complying Before we like projects, call are the the we such with These statements considered would of facts, begin, Securities by Safe may to current information variations the XXA and Statements statements within the or identified making covered be meaning prospects, include assumptions have intend for this expects, seeks, plans, call. on expressions. words are forward-looking of not similar and currently Harbor and intentions, in discuss conference historical us words based which to for on made. Securities are of make strategies plans, views which statements Section to forward-looking statements the on briefly we Exchange forward-looking I available of the XXE forward-looking this and anticipates, statements may, the which usually forward-looking estimates, Section our the will believes, of these as purposes such We reflect on by expectations, Act contained and Securities and of Laws, intends, Safe Harbor and Provisions. should, provisions Act, are these about

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you, Thank thank today. for you, us joining everyone Mayur, and

updates you several take have We to through.

for four highlight key take me Let homes today.

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Anemia, Fanconi for Now for turning to and and regulatory our our programs we've provided programs, lentiviral our continue LAD-X at updates PKD filings. ASGCT momentum

very patients For with enrollment, our passed all malignant conditioning Phase evaluation especially related the the providing our note are patients also more have therapy comprehensive monitoring away pending other therapy the XXXX. complications diseases, from collaboration trial likely in evidence many had quarter in protocol, transplant. of that committee. programs to immunologic saddened we I paused forward line in pulmonary gene thrombocytopenia that relative autopsy patient deeply treated hemorrhage final likely related pulmonary underlying non-malignant with in and are program, independent in confirmed of osteopetrosis to occur osteopetrosis data related in look fourth an we to complications a the pulmonary Consistent We those non to osteopetrosis. following with who Of updates infantile was undergoing commonly first

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Gaurav. you Thank

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I'll any This concludes on you update. executive Q&A. thank operator the turn Rocket this team the back is Thank happy to now for call Claudine. to prepared over And questions address our remarks. you. The

follow limit time, will session yourself question. interest you one [Operator that Instruction]. our and with we we one In answer your you. up question. now thank And of begin question and please the question to We ask

question Our from first Greg ahead. Please is go of with Bank America. Harrison

on for the This Greg. Hey, is Ashwin guys. Thanks for questions.

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question. your for Thanks

may in week, seven regard moment. now complete FDA the what full Meeting, the Committee With upcoming of as Orphan confirmation of I actually of that's Gayatri the and contacts FDA this Office last So one no the Head we for working have the for will waiting the Advisory at further the of you and members review. process. clinical We team Gayatri? who this. protocol. pass respect as FDA to to was Products say know, can we're And is to Rao, at the their who, years on we Drug executive And of Dr. Rocket over the

Rao question through everybody, Gayatri Hi, advisory the committee answer is this specific Thanks. how this to to meeting. related

have engaging sort to it's inconsistent certainly in we far with the for of to reason to meeting discussion relationship we that the the As this and Meeting. and respect really all clinical we've between believe the the the And no communications and timing know, With hold that there's Committee as any listening meeting the date. from forward with there. to we're itself, learning had agency Advisory looking

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Dae Our next with question Stifel. from Gon is Ha

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has changed since May update? or proposal that view in your changed folks. anything Thanks So that in anything

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very Thank you much.

thank you. And

with Blum and Gil Company. next Needham Our from question is

you taking afternoon, for Good our thank and question.

therapy? the the that regimen IMO about Thank study. there Is you. and seeing happened significant Just in on between gene the these transplants any differences the patients deaths conditioning really versus

versus mean therapies? gene you Okay, other

cell No, sometimes of hematopoietic standard just is versus stem care. which the as known transplant,

between That's used asking. osteopetrosis transplant? there Is the in gene any difference therapy Correct? ostopetrosis deconditioning it. what you're versus Got

Yeah.

Sure, Dr. I'll over pass Schwartz. to Jonathan that

extensive that's immunomodulatory extensive, guidance a regimen is chemotherapy less this this therapy would less lympho-suppressive Hi, of and osteopetrosis and is [ph] cell will the allogeneic therapy, amyloidosis be sometimes Schwartz. conditioning In your utilize cyclophosphamide, The and different gene B for Jonathan regimen tran, suppressive mild then utilized in used an or body therapeutic governed well. this that's amyloidosis is drug by condition. in that TDM monitoring. study general, as combinations irradiation allogeneic also therapies and total regimens cycloquesoc Typically an or transplant utilize they transplant immunosuppressive pharmacokinetic regimens

extensive, this is nonetheless, of part our program. less a but this So amyloidosis is regimen gene conditioning therapy that's

Did answer Do Sir? question? up? a that your have you follow

up. have you. No, follow question. a That I do Thank not answer does my

much. Thank you very

Our from question is Werber next Cowen. Yaron with

for Great, my two taking thanks Gaurav, questions. question. maybe

patients The based first did cardiac volume what what think be a obviously mind, left walk follow for sort ejection you. minute fraction And of testing of then with any what because one on did updates Or you do related And up? see pivotal? it's all Thank when normal essentially with is a baseline? echos, in were seeing, your primary endpoint you're you might you about ventricular And benefits? longer that any output. stroke on six to

Yaron. Hi,

-- fraction have the disease. in the that a the echo On most ejection for This patients many ejection until patients they preserved trial. been of stage enrolled true fraction most patients have is reach

transplant. disclosed I update lose as previously, here criteria. a function. assumption as earlier, mentioned move last we That on our was that have More to We to did, who that starting on patient cardiac patient the was that have

ask And in sorry, to second there. you again, have a couple the one

testing? one, Yeah, cardiac output. And obviously volume what then minute determinant six the stroke walk second Yeah. of it's because about

walk. Stroke six volume, minute get to and then we'll

revealed was So working on that that current that with we're data shared there also December, the is we've the set in data output, the cardiac on agency.

two patients in as having basic the have the by loss patients started increases before do worsening output we in start of to there's of cardiac even mentioned, these of Some output cardiac see fraction. dynamics. three have a ejection measured And

of information is improvement patients. demonstrated dose in In tests, new this we minute walk terms the before walk from all six minute of now have six and three or stabilization test low

that's stabilization, the law your or well consultation improvements of as So site BNP in class. as

information. So that's the

we and imaging as class. [indiscernible], BNP, or cast walk These even that towards clearance, injection life net discussed, help such endpoints, clinical appropriate guide include to endpoints, functional in six of as endpoints a endpoints phase the On such such have as as of fraction, kind quality test, two. wide the we've capturing endpoints terms as us biomarkers NYHA minute well

of the we FDA. X those of any on will an that So can't will these be could speculate be have part of we endpoints, until what end Phase the discussion with

trial been think the same there's about the right with At time, I a here a had FDA. now. delay we've the discussion quarter on collaborative

have conversations hopeful expedite about Sometimes we're trial potentially can right in discussions development. having We're endpoints the you the until we these don't now. that based on later

right, test, that to I walk And six sure make if minute got I it. I'm missed the sorry,

six and But also I'm walk say minute an You art improvement improved? you NYHA mentioned BNP York was class, stabilized New or and and class. saw minute you sorry, in did six walk in it

larger class. Association revealed baseline. patients improvements specifically had three we suppression. h had patients stabilization six -- walk, BNP specifically, patients XX% have parameters. immune and those have those minute All two regiment walk minute test versus had improvements stabilization New closely in go or or output all XX% cardiac minute who York patients sorry data. monitored test monitored, and suppression two drops all and Those three detail. six lung. from Yeah, BNP also in three BNP, two immune the patients Heart test The stabilization in walk so Six improvement. I'll had And they haven't across All patients to have But who are to the heart

but three in fourth at stable in all been of patients improved patients point. this those or will well quarter, as We

Great, thank you.

And thank you.

question is Our next Foroohar from Please Leerink. go ahead. Mani with SVB

eligible trial are for the there taking the changes Danon would protocol that of question. potentially that thanks Hey, for up? My proportion change be can disease is the question first follow to patients any and for the

Mani. Hi,

such We prior onset the therapy probably gene always considered not we never that last in fibrosis the disease. population. affected. be consider Those year We've to part stage to so are be said patients identifying were the that end of transplant, patients of is would that or would treatable

does the change it not market. treatable overall, or answer So the is addressable

Great.

looking not to learning ADCOM. You in and you're or listening the confirm you're Can invited and meeting forward will September yourself? said whether be participating at proceedings you or the

have Today, had we'll we certainly FDA Yeah, have discussions the no but listening. ADCOM. with not been about invited the be we

guys. helpful, That's Thanks.

thank you. And

Prasad Raju is Blair. William question next from Our with

for the Thanks taking question.

the we it agreement into the pediatric FDA on more progression and into program? can dose, re-enroll plan on dose clinical going With of for then the with kind population you do should a the the into high Is adult lower go regimen? had cannot special question patients, trial patients once the the and the what this a or in I population, of pediatric advancing then you expect

Hi, thanks for Raju. question, the

forward away population as Our intent we'll is happens. pediatric to with that logos and update have move the right

the Okay, just goes helpful. immunosuppression far to changes be can great. maybe regimen clarify were as made that And Thanks. you complement on, that'd activation as the

update FDA the consider And types in It's of We trial trial and revisions, the in the affects there we don't nothing or during would made in as nothing that refinements, eligibility tightening the But points There's patients than the will major. will definitively conduct progresses treat want that of we can we really too that yes, many update. the definitely provide quarter. protocol. to anyway. certainly that affects other fourth active dialogue. an specifics I

thank Great, you.

question have our And with UBS. [Indiscernible] next we

taking for just Hey, fibrosis. is patients the thank One question. a on you the couple. have I my with

patients And your to whittling kind are the down in or XX,XXX the about patients with of stages, EU. defined U.S. are there from market advanced XX,XXX enrollment that that of criteria? You've how you is as across kind fibrosis the and

a impact that Yeah, to boys We talked a that's patients haven't which of program the the heart be disease, have in of the or cases way. sorts fibrosis, question. year leading death. small. very really be It's and up the probably of about the Females, and affected gene transplant, we certainly with would good to going number think liable heart really not maybe months same is about unfortunate forward, very a different certainly actual therapy or that's move so we in population and is as the yet,

you're looking XX,XXX, So if going within anything, get there. third I that exact general party. think by if they're We to numbers think are for those those numbers validated a

Now.

of capture we types those a that even more As could mutations, numbers there's possibility expand.

I think to will going sliver much. that eliminate number end of characteristics don't So get of progress state is patient sort that into diseases baseline that too

to you. you've exactly -- the thank you the are of females the of need trial. move forward do -- What see Yes, mentioned patients with are current And of enrolled kind start we that what kind girls? consistently since to in

With females?

female? With

Yeah, we don't much. that really need to see

little want to think need X, closer, we and Phase like will look start to Xdesign into more a in get but really I we further to boys start a what nothing see there's on Phase thinking bit first, about females. the trial

have plans. given it's start, in certainly any medium when term guidance We to near that but trial will not the

it. Got

invoiced you're Okay. then. X clarify, And the just be to planning trial, Phase only will

Phase the upcoming and Phase Phase extrapolate X. in we The current, learned -- we current want would current what Phase X to to X, be X will X on based the the Phase not

to wouldn't So will limited a voice. And parallel, and separate support we trial, in start females trial. be be exploring would a which in nature would that first registration trial we that hope

Okay, thank very you. helpful. That's

you. Thank

with Schimmer is question Evercore. next from Our Josh

taking for thanks the Great, question.

cell to Just how that different Danon, study X considering you're of that outcome are that Thanks. the X? And the in IMO for pediatric -- with is that that you that capture patients about separately be more then X will -- are that whether explained Phase the otherwise that by Phase inform of have are study? patients that than are might determined design unfortunate advanced the passed kind the who or features here still will disease in of data you X? enrolled yet there Phase traditional any they from were think passed informed on who patient do stem away, the the ultimately topic And Phase have the the a transplant? registration for patient older who might gone looking unique continuing parameters what for What unfortunate not

Josh. Thanks, Right

So in the to in need to terms X pediatric [ph] what start LOTOS would we of Phase Phase see X.

end think the meeting. that's with we'll X obviously have the discussion I of that the Phase at agency

of pediatrics, dose of see moving if think direct of X what we I in of Phase prospect potential be confirmation that, low if benefit the then, can relatively the confirm we adults saw and we more rapidly. we'll toward

had soon define to how trial, for discussion Phase final as that's the possible. X of endpoint, will the still think terms In as under I that be

development been think dialogue over last pediatrics, as Phase I primes weeks, accelerates potentially. the several we've end for adults the hopefully, and dialogue of X plans X having as the Phase well

differences On the have osteopetrosis osteopetrosis other any whether were that will patient, Jonathan, there defer predisposed. patients patient might that to I in versus

with question the to the repeat osteopetrosis patient? you Can respect

they any Yeah, are features have just that for had explain was earlier had disease, outcome might maybe wondering portended eligible had they that cell if that who why transplant, advanced it death an that not whether patient might older there been Jonathan, stem more the clinical

information thing this I conferences disclose lung of patients a don't think data much that clinical One the in more comprehensive that do was very the that later ribs this compromised, pattern. identify regarding provide patient restricted of cause we're that to these low that's risk. related abnormalities or we'll osteopetrosis, lung that that as high as necessarily so is chronic many Yeah, bone at aware either have specific we would we there individual anything in year. relevant

then we patients comment of able you're is high many do is a nonetheless, these is in probably lung why these a our pulmonary have why complications we'd in sort don't a chemo patient that pretty underlying pulmonary complex, talking a osteopetrosis typical of or to can this there that have therapy patient. disorder. different in about and made create still severe was bronchitis have intent All the a having chronic fairly think I of you'd say, which abnormalities him the now patients that less transplant Obviously, kids overall from these inflammation -- some allogeneic, about specific sinus degree And environment. settings. her provide to anything of right But transplant. be parenchyma,

older would see Okay, patients unlikely may improvement? I ask BNP on not and of are a improvement you performance in guess trial? patients, Danon phase enrolling one you're that younger If baseline that the meaningful to would I and pediatric are up are quick and they just they have then if abnormalities that next deteriorated see follow unlikely heart degree abnormalities. you they the the could they younger, to patients, baseline And failure, be to for have amenable because to have

is remember Hi, Josh, that the patients off XX of disease treating. here. who [indiscernible]. that cut And is although are the the for to Below Gaurav adolescents. we're it heart here is than of number young considered pediatric inexorably heterogeneous. is And leads even There adolescence earlier around the a cardiac have failure average, adults, age onset disease On pediatric.

want class pediatric higher. ultimately heart to onset patients or sign many will disease. measure, what have cardiac but Those or have or signs some therapy a ones So that symptoms about. that's in move be would preventative disease failure All this symptom, X of enrolled patients do as of who the is to X before pediatric who may some even the there Phase cardiac trial not of are to are pediatric

it. Okay, got very Thanks much.

question, And defined by wanted which Josh, has before protocol add I also for the move we measures we IMO apply to next that trial. to the

have the trial the on based information to put not to It's this and shared asked with clear. have the just pause we We hold. on FDA, self-mandated a been protocol, be

We're to could had history I also, natural how Phase other a pediatric decline, early our trial side about of version And learning the know We're can information a arm we're help us lot forward final a especially into move trial be we time, what we're low And in you of X. that spending could years. single with see developing a trial, the a output. that hope the also asked patients about trial. by teenage a lot dose in starting activity justify side that potentially Josh,

that So the idea that's X. to here, moving Phase

And thank you.

question is SMBC. Hoang David from next Our with

Hi, thanks for taking the questions.

I the remember to mean, ask sense all thought about clearly reasons for it may want to about. discontinue dose, you we I I be it, some part, talked pursued there decision makes benefits. the because cardiac had high But So you the extra in

maybe impacts you think guess the overall have proposition of I do the that dose, now that dose you without So high option any degree. the the that to capturing value significant therapy of gene

disease disease, be as Danon you of multi-organ, is to So the spectrum treat It would if full possible. say. nice

the disease However, mortality this in is cardiac.

especially normal is who or away pass XX. So these being the ultimate would estimation, or transplant address otherwise we confirm can value longevity XX, patients were heart in life and Extending we disease. proposition, age if able by to the cardiac aspects our a of on

think we strategy what major had a So it I that very was way. works works what and we but in a go with in place,

And you'll that. for you approval initial work basically do full -- the for to bring baseline assumption then think use ease what's -- be the X thanks Phase you this clinic do accelerated? Danon? would get, you'd appropriate the Or endpoints that approval? your get the with be able Is Understood, additional after would I approval in that that to

ultimate have that support is success a speculate Yeah, At accelerated also could of clinical plan. here. mortality time, we point, the that of this to marker it's biomarkers same hard the an benefit I approval range recognize think we at that a

answers other the that have speculate. in were we and I points to hope here, So can't Phase also on biological the But weigh credit agency. extra having also there. And manifestation the at moment, to Jonathan, of we ask really some we as comment start will X

I and don't you Thank disease Again, that it's manifestations. and where low patients, think discount male dose here. disease fatal we'll affect in can whether cardiac we Jonathan the build think I emphasize yet can't or all can Gaurav. the Schwartz important around. the that's non-cardiac to essentially death obviously, endpoint is the although

Importantly, manifestations. if patients, extra substantive in at have female cardiac you look them they don't

believe neuromuscular So these disease has longer low well the that take non-cardiac possible the cardiac will substantively aspects likely in of been transaction those treated male the XX% we we bit we determine who by the a the also seen normal little seen this disorder. see or certainly has adults may of have neurocognitive XX% females elements is date. benefit may protein dose we of benefits than expression unaffected that levels But those that that expression to date. the lab aspects have to in just Likely in in are to patients affect largely as of patients, to that Danon to with these

Okay, for questions. thanks taking so the much

thank And you.

is next with Delvaux Our from question LifeSci Capital. Patrick

Hi, thanks my taking questions. for

patient apparent. As it that heart you underwent the was relates to fibrosis transplant, mentioned

response any and myocarditis been from in there heart similar was biopsies that the [ph]. to worsening Thanks. anything the heart there expanded observed has observed And immune so, or patients contributed However, for an if evidence need transplant?

looked on low also no of patients no, no didn't any at. see dose changes. we the that Patrick, other myocarditis, we've inflammatory Hi, And those technologies

Got it? Thanks.

thank And you.

Morgan. from Our Eric JP next Joseph question is with

joined evening. good late. I question. Thanks Hi, the for taking

that Or the it's it So questions a both program just guess already I question being we X with would maybe patient maybe of addresses And to they just protocol, addressed. are in the when parallel? and as sorry incremental have to that what or Should can -- would up meeting? a to X to need data far, you gating extent be Or be the sufficient? the factors just follow Phase in of data single potential protocol? pediatrics? you clarification patient Danon? programs articulate adolescence contemplating the you would with But be to so latter, revised numbers if terms an end Phase the the in you data revised the pivotal some relates see The new Thanks. for

Yeah.

also output protocol We we one, X, are define to add what patients in with in patients of those what So get since figure with actually, there, number a that smaller. need end Phase sure. dose anything, out meeting see data to so is to number likely are can and the we ne the start that We if need for going regard right think that things to to now, cooperation some we've high we going the And Phase out to to pediatrics, be X be from needed of need history the the are natural design the is, cut FDA. engaging. the don't endpoints of see to the more

to So that's point one I want make.

separate question, ones first trial anticipate for and two We adolescence. we one your there. anticipate don't pediatrics For

gating the described are the hope we now, right ones to do… you So and factors

helpful. Okay, taking And for thanks got Very it. the question.

over you Gaurav And thank in And we CEO sir. Shah, would for no questions further call queue. the to remarks. turn I have closing

updating Thank you, look call, we today's forward everyone, and for to you participating again in soon.

and ladies gentlemen. you you, thank And your conference This and thank our we concludes participation. for

You disconnect. now may