PASG - Earnings call transcript Q2 2021

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Operator

Thank you for holding. Good morning, and welcome to the Passage Bio Second Quarter 2021 Financial and Operating Results Conference Call. At this time, all participants are in a listen-only mode.

Following the formal remarks, we will open the call up for your questions. Please be advised this call is being recorded at the company's request. like President the Strategic of I'd Stuart over Finance. time, Vice Henderson, Investor this to call turn to At Relations and Stuart, please begin.

Stuart Henderson

This Bruce under recent our the key the business to Officer second updates Releases highlights. the will and the Salinas, outlines review Q&A we On Press and and Bio quarter release XXXX our R&D today's Chief our financial portion section. our today. will available Quigley, Officer call, will Jill Passage investors.passagebio.com Difficulty] on discuss President review is at [Technical Officer also website statement available Goldsmith, topics discuss plan clinical results and Chief of Operating for Eliseo Chief call. be Executive the

and by the are strategies, operations; actual these forward-looking suggested matters. uncertainties other company's today's of data statements its and of expectations uses the cash forward-looking on ability number that assumptions of of to impacts our respect significantly and manage ability cause the plans These and include the Before risks trials; candidates on differ clinical ability and not about collaborators lead research the and performance limited from including and please programs; results call statements company's flows; respective financial our subject on, company's but of our and that cash of to ability we based product about of our disorder; along that expectations statements. execution could to to and those clinical execute initiation trials initiatives; with are to comments trends timing note manufacturing CNS a to the development fund partners' including, pandemic milestones, may treat anticipated with availability to to key begin, target the the costs from COVID-XX such

required for not the uncertainties, factors made information this Given filings expectations, or with as you these on actual cause update statements SEC to call. is Except occur including pass events forward-looking to or for reflect circumstances any company its to any now to obligation refer should call. my Bruce? disclaims from the this Bruce statements that company's CEO, forward-looking forward-looking call that results law, statements. to revise could materially or It the after place Please over reliance and concerning undue pleasure risks differ publicly by these account on any Goldsmith. the risk to

Bruce Goldsmith

we us I is strategic Thanks, which begin Stuart. four And joining strong thank our Program Therapy around company. Jim partnership These are by pillars our Wilson. for pillars Dr. all believe that will this Chief our GTP, morning. Scientific Penn's Advisor, my or differentiate remarks you centering by Gene with led our

genetic includes and and develop corporate framework for and which broad our Our focus our company in manufacturing supply a we medicines. a continue our pipeline, a transformative to robust and with solid establish position. chain patients where cash pillars how medicines efforts highly These foundation, experienced strong team our as and integrated CNS for as disorders, provide leader the we

significant existing partnership CNS large excited to and We our amended have I'm agreement this For diseases. license evolution. report pipeline, view GTP scope include broaden our the strategic and as we the that point to to with a in company's collaboration inflection

we option proof-of-concept, with for targets collaboratively be larger GTP collaboration making. we a programs, University ability medicine development program into expansion with our targets. disease pipeline guided to Pennsylvania. We're Temporal will optionality able of Under quick beyond license expand and CNS the expert focusing has this CNS which the these condition, this agreement. option continues genetic the believe of no on an obligation in also our XX advancement. the strengthen to a the exercise the positive to target and decision initiating already new Epilepsy to monogenic team and preclinical indications new Jim dedicated Wilson, mutual to that Passage the support underlying of Bio total Once effort by opportunity have will greater and Lobe targets his In several and candidates be identification additional across robust These of under evaluate select exercise wide to will enable with our for parallel provide advance conditions exploratory research biological our generate for agreement We and and rare productive technologies reached data and populations, indications Alzheimer's but to range upon GTP, to research the and that evaluation programs. large to may option. We'll the bolster then are programs proof-of-concept

monogenic So far, have have of options we seven and diseases, we these in exercised rare remaining. XX

monogenic In driven indications, exercise disorders, and ability in de options, When as current the may focus factoring risk in our rare so addition area into manner, this in additional continue to exercising need, to remaining our XX expansion and data the development a differentiation options we'll on patient programs. clinical well. do we large to maintain product

open have therapy analytical New to our capabilities, manufacturing vector we Therapy and West this Gene we Princeton Jersey. advance and and called development, testing that Development enhanced Hopewell, With goal in analytics facility, development appropriately our For I'm we moved eventually gene assay to our for capabilities, have new met clinical Lab and production. validation production state-of-the-art are essential happy report products our Research scale clinical product as characterizing commercial optimizing to our

is Mark these announcement joins and world-class manufacturing we We're as of caliber talent the Chief have in we across proud Törnsén Commercial of later the by Chief to Financial responsible including and and leading attracted positioning information our illustrated Bio, organization will be first commercialization. Forman appointments, King and tax, Relations commercial technology. of determining leaders for responsible the company's for Investor company's treasury, foundation, as for this strategy pipeline and Passage joins and clinical including joins corporate as successfully new recent and to path Medical our the partnership, solid us us recruited. is strength Maria have Chief Officer Officer and as underpinned three out development executive efforts. Our leading finance, accounting, pillars functions, responsible month building is translational Officer product us and Simona

also Board to we're who call commercialization development strategy, We kind supporting Directors guiding the corporate is company. upon and Porter, Dr. and have level. an in the at Derrell recent example addition a product and able tremendous strong of experience of expertise The brings of talent of our board

despite options. for the the pillars, lead three dementia focused in upliFT-D remain or in patients day which quarter our of of on disorders around development our we're XXXX. XXXX, GALC safety in and first biomarker for fourth mutations, in We hyper in trials the treatment the there of and strong Krabbe strategic the of and XX our Disease execution COVID-XX company to the clinical third Frontotemporal XXXX. patients quarter the study our first XX due trial ongoing build cohort no deliver of treatment in data we As cohort Imagine-X data and dosing treatment several GALC clinical and upliFT-D programs initial limited track CNS in half day for of of to of for the four are Reporting important GMX first for our therapeutic our on milestones. reporting Infantile the for These on with delays study biomarker first patients the include granular initial safety of rare

turn million with by are execution Eliseo who strong supported and at $XXX clinical over it Chief securities marketable Salinas and sheet detail. I'll our our a momentum now R&D in will end. balance more discuss to in cash equivalents Our cash, programs Officer, quarter

Eliseo Salinas

site and lead some programs. patient identification begin overarching comments I'll Bruce. for three initiation you, regarding Thank with our

so multisite continents. weeks. have include First, in to and significant they programs in the counseling expect of with companies and patients out clinic to that nine be our point international ongoing targeted that patient we global sites groups. physician areas made by through our disease have offering three the and collaborations of where we Despite our characteristic also headway are. in COVID-XX, challenges the for a for extinguishing In we we and sites network pleased countries can all with a that across multiple testing advocacy and genetic are were total, network due opening variety and scope, a We identifying partnerships lead our continue patients three clinical service coming initiatives These of of initiatives progress with programs. broad programs,

programs. specific to Now our lead

and course care. we announced pleased severe which trial program, that disease, the gangliosidosis. beyond with lead the trial of treatment the with form is we our rapid know, The XXXX, patient options infantile you for progressing is trial Imagine-X report of in current April dosed GMX the a the I/II patients I focused form of very in the that disease planned. many first most Phase as PBGMXX the is global for supportive treatment am to Imagine-X on no As

have initiation of them We in and to expect begin coming weeks sites sites. in clinical those additional the to the activate enrolment completed

this GMX onset As levels primary label being tolerability allow We're in is also early the of will is we to the measuring XX have for first to CSF biomarker those safety level and infantile quarter the look of dose cohort study discussed study expanding divided to serum, of in trial four escalation PBT fourth beta initial cohorts PBGMXX. distinct globally. cohort and that gallons day forward of the safety late assess One open our dose The our reporting progress and receiving our by cohorts. Imagine-X previously, Cohort we composed with on from data an and and levels, the enroll into initial of year infantile goal patients the age and

Turning to Krabbe Global our program.

we from clearance MHRA Global I/II calls, and prior Health FDA, received our called clinical the initiate regulatory Canada. in our trial Phase GALC discussed we As the have to U.K.'s

multiple site expect of first advance initiation to third continue processes the quarter. to patient the We dose in and sites

trial, cohorts in onset into an a late dose with higher Those GALC goal early dose The label disease. dose early PBKRXX in Imagine-X to PBKRXX this include and escalation study first early in Similar GALC's Krabbe a will cohort cohorts. then open study patients of disease activity of of to our assess that safety with evaluate confirmatory patient assessing and both and patients, will As CSF be dose both followed PBKRXX ascending of will reminder, with initial the serum. onset we infantile progress is Krabbe groups. expansion goal secondary the in infantile primary by age of

of global the in And frontotemporal behavior, of or causing FTD one language early course FTD specifically the of eight of clinical mutation. mutations a caused is Next, let's progression patient rapid of dementia, of for impairment FTD focused younger because FTD-GRN of onset The onset in causes on third after resulting an symptoms. our X% disease granulin XX% GRN old. that We're It for at PBFTXX common gene in causing occurs function. average to Alzheimer's dementia of trial similar executive the years more with FTD-GRN. mutations discuss program XX by and a is programming. treatment about of is frequency survival than estimated years where deficiency of the to disease

global Phase and clinical patients, FTD-GRN received Canada. an trial dose to study open authorization is Our escalation upliFT-D and label clinical the United regulatory in we trials I/II States in have ensure PBFTXX of

years the and on initiation the serum. to Additionally, Phase The programs, regulatory Like reduce potential PBFTXX biomarkers, two protocol the up potential regulatory evaluate other certain provides processes I/II grants two the received increases market dose designation site European advancing quarter. sites, XX for PBFTXX from fees, key our its research with for FTD-GRN we the of on are impact clinical multiple We're XXXX. and the and assess specifically Bio July levels approval. patient to assistance, for Commission orphan exclusivity including following in of benefits, granulin Passage in third of in designation to and the track of safety our remain in study first goals to treatment CSF

future and the turn potential an the and near-term support comments future with we're their to to programs. call saying patients will FTD not grateful With Disease gene excited serve, the the Krabbe it their data we for honor that, Bruce caregivers, ongoing review I'll workers readouts, incredibly ahead my As only healthcare about end to to our and look communities. the of now physicians, we shape I therapy CNS is clinical other in space. by the over financials. of of We're but work to our the GMX the

Bruce Goldsmith

Thank you, Eliseo.

report. us quarterly As I earnings Simona be our new earlier, and is our next shared she month, King, present CFO for later joining will this

report, this update. financial the For will quarterly I provide

our cash reiterate to position. strong First, I want

As we approximately reported in as million XXXX. million we equivalents securities $XXX morning, of compared December as of XX the ended $XXX and cash, release this our to press marketable with quarter cash

costs We at securities increase clinical G&A quarter marketable share expect next to in June associated with ended for and the for $XX.X for June through additional PENN compared the was these incurred the $X.X increase in the of offset XX increase and XX XXXX increase partially $X.X R&D compensation primarily clinical and our increase for expense quarter manufacturing quarter million million in same $X.X filings XX, $X.X were operations $XX.X These function IND XXXX. related pass to development a cash, due preclinical least increases compensation in which million XX, to cash costs. R&D The ended a $XX.X expenses an work months fund by to based to costs. performed research equivalents was share primarily due million million for expense, employee $XX.X were in manufacturing for programs the to expenses for increase in million to in same in the costs increases were decrease three our related a quarter employee development clinical preparation research personnel million million $X.X months. lead in XXXX. relates the other due ended in for The agreement, headcount. an due million increase increase headcount personnel a in and based certain an and $X.X in compared a in to million costs, and costs, the June

address XXXX. quarter and of second for that, million was same the diluted We're fees the team patients compared and tireless date Temporal to with With initiating as loss at end the with therapies shares XXXX, $XX.X presents my expanded research and pillars broader guided treatment especially day population share patients will million I need effort the quarter underscoring quarter we professional also by $X.XX that a in direct disease compared facility of net $XX.X a and basic about diluted with CNS strong in per the that the costs in put disorders. XXXX, forth second increased specifically successes significant. operations the by to of two affect who per second strategic options of Passage populations both diseases loss support for agreement Epilepsy, are and was to loss $X.XX to excited result rare basic efforts. by a remarks and $X and Our for research to our strategic by CNS every to it Bio, XXXX. of quarter our Net discovery million working our are where Net for We development large of our to our patient and large Lobe hard GTP our look transformative Alzheimer's new collaboration deliver disorders. opportunity expanded in is forward adult

maintaining that monogenic rare We our CNS on to disorders. focus also emphasize want we're

clinical GMX, extremely our our for focused team, engagement teams and patients Disease and studies on all Krabbe described, manufacturing FTD. Eliseo patient As with team are

are stage potential We monogenic to advancing and evaluate committed programs to in the options CNS our continue other space. early rare

year, this reminder, quarter in upliFT-D We're Operator? report our trials our a and one One three data biomarker of company first and of of safety made trials the it patients as quarter first that, GALC this biomarker initial safety data Imagine-X as the the third in to dose advancing a day pipeline in report up Cohort GALC two of XXXX. near-term are open our XX leader As we to ourselves our medicines. and in year, the establish we building have upliFT-D questions. fourth of genetic proud the and day in will progress our half and from milestones in study the for key to initial XX I and With for

Operator

Thank brief to you. first question. the [Operator remarks Instructions] will Bruce provide prior taking Goldsmith

Bruce Goldsmith

all you, this us operator. joining morning. thank thank And you Yes, for

who about did the Yamada, few you away suddenly has to Tachi from Before passed want by this now a likely global questions, say opportunity moving words outpouring to as I all take know, to the Bio And to Wilson. Passage few X. that I I as he's had. co-founded a And Squinto also along August minutes Tachi impact and with on know Jim wanted think talk Steve you about

in we me the it's across of is this say by So and demonstrated I And very comments seeing all and the management of speaking inspirational honoring and he the his was today, did our leader. saddened for as knew we're But board shocked yesterday, obviously and Chairman all grateful globe. us and him had to very have and you late terms mentor. all with Tachi words. and fresh And incredibly But releases deeply that incredible known worked a all Tachi. the tell both I to a we're massive and to devastating. impact for the in industry really can we're both what opportunity Founder, course, press and with extremely few team contributions, a very want and an

foundation about and as company pursuing While remain we guide The wisdom continue. the certainly but including we Bio's guidance Tachi's the certain company, of Passage in for the helps the made and that, And very few having and that totally now finally, turn board, patients talked of mission a his insights do over so to in terms do has and patients family impact we do both And we globe, comfort I announcements they we absolutely strong with think think and help talked to will for who and to really And them build incredibly Steve, everybody, an that just commitment or have last do and much I across to we of in for to opportunities And to strong grief as the also Tachi's thoughts. mention committed out I've have. additions. I as back really the today. were want guidance family. operator Passage such the days, in And with And strategy, we've constantly We and a over with set we in partially company. he also Tachi I talked partner, a to deep various the motion the gives massive with obviously and innovation our to work hope I unified to meant management the over made as and set said, foundation Jim commitment strategy for our are time. Tachi's memory think often questions. that operational With mind. will the Bio, the Tachi's the call we today about about and patients

Operator

with Thank from line comes you. JPMorgan. our And Romero of question first Tessa the

line go is open. Your Please ahead.

Tessa Romero

morning patient now? dosed? How sites you remind patient? that identified hoping that dosed question number you're Hi, where all. us much will so site get up and much. I/II be patient to And live GMX many activation? Good you the target And have you do you in the on specifically then in me, can you've to Thanks that if to? first clarifying question. thanks my just what Have that have is Phase in you've And the for April. A for second taking in very go study the infantile noted not, you're

Bruce Goldsmith

turn then Eliseo. comments few and Sure, to it I'll Thanks over the make question. for also a

other many year. perspective have for the as I about patients from on. or of that So we date hope any additional patients forward-looking exactly our day end to two any do, questions guidance we're what activation terms were announce sites dose studies reiterated other will, Eliseo, we coming Eliseo strategy is moving got the not patient and that other we think the is reporting really have what the out biomarker were to your data focusing the around you want when for just did over said that hope on we often of first kind of overall do of I open to the how doses we into that and reemphasize come do the XX more on we And and sites add in the forward. details details comments? across the note first And kind plan about all sites weeks our will really companies is safety of the time overarching of which in by cohort, on and GMX,

Eliseo Salinas

and countries, and to same sites. of in their it continents, site studies that do And one number patient, high that time. you back lose doesn't to the typical patients But at recognized is dosing open a That futile sequential In program, is which to doing, dose technically domain slides. Yes, least a the our first trials vast opening strategy a the days could many sites patient eight An our second. and be the enrolment are one of is in that space dose escalation patient gene plan do would elect where one to what number ineffective for between have XX enroll the a consideration therapy studies in to one public to number sequential aspect case, all open XX we momentum. get to you site. months cell would at be different the with high we time important would you to sites, with at in a next with are. I'll we're wait that of important just the But or of patient sometimes that clinicaltrial.gov high at one. it's time because program term number patients these the as a take is it's with those get of all identified is so

on sequential, those the start have of a move sites. and identified could the and also and activating So deal sites ready of to we our strategy to the are that third second we And opening patient is immediately. go, site then focus

weeks, weeks. the sites next will active we're progress happy with so those the We far. few overall, in very last And have few the initiated in sites become

Tessa Romero

was much. very Thanks That helpful.

And so about passing. to sorry hear Tachi's

Bruce Goldsmith

Thank you, Tessa.

Operator

from our And question Bitritto-Garg the comes of with Neena you. line Thank next Citi.

Your go line ahead. is open. Please

Neena Bitritto-Garg

question. thanks guys, for taking Hey the

guess kind that that get study, a by But maybe regards the of studies, two quarter, end other I the similar I early third there of question. FTD potential first was dosed patient the second last you to think noted quarter So the to in quarter. the or in

I of there, there this perceive study on sort if had per any or should I a guess, help guess and kind kind se? unexpected whether of the site you So is as characterize that of can we of any yes, delay Or activation getting running? kind made you've and progress not Thanks. delays, up you've

Bruce Goldsmith

The moving in simple other seemingly they because negotiations well I of of initiations, of of there differences site study up them because way as changes or before, IRB is trying we're with. as ahead to Neena. that a mentioned getting either are kind as idiosyncratic would just lot characterize are interact studies this us, that hung and were Thanks, earlier. And staff that, every simply timing, of lack the such of filed backlogs I

it step, the we we we're was based of of part were or insights kind said, the the the either that's sites, So step. the that And site now on in and as is what quarter, terms along saying said, activities scheduled, either we of when updated and also the been way cetera. were getting activation when second better we which dose just and when of then those is making. anticipated And completed, second scheduled have we those third initiation because progress having we third patients quarter the better Eliseo guidance, et obviously of enrollment now or have

issue. environment we're the living to it basically the a right think specific specific, one So I don't forward. trying just any in move was and now It's programs

Neena Bitritto-Garg

helpful. you, Thank perfect. Got you. That's

Operator

Werber Thank next from you, question of our Yaron line comes with Cowen. the and

line go Your ahead. Please open. is

Brendan Smith

good This the to Congrats our team Brendan the Hi, yesterday. on on to the in news is morning. everyone condolences Yaron. progress from and for

upcoming more cadences ahead programs ALS, moving just MLD, some and actually timing preclinical your see, one actually a maybe us, what's for we in of see the to looking those, data to relative preclinical Charcot-Marie, might or from and other? think Just when quick efficacy some really MLD new from approach you on looking each forward your them thoughts sense get of specifically who to that space? you seem to very with might on you there, have, advantages hoping actually, others think in to kind And approach like kind of what just get ex-vivo your solid much. there have just Thanks versus pretty to antiviral

Bruce Goldsmith

to Sure. few a make I'll well. as turn then comments it and over Eliseo

certainly the programs do today enough and in so. And So this just that back at the by really year. GTP and when Wilson each so future. move data these don't that. is on the that MLD and give do forward that, But in we're of is think we detailed think position we thinking at differentiation. very exploratory to we're nice continue of the announced we've we points TLA, in a will mature to his that a to the as certainly but the insights, I a I Gene about and more Program, way upfront think do don't share is one share the specific we group is that we're this things earlier the group, of I available, we're model And in obviously, the about programs this been, Jim programs, presented was will this approaching happy is moved the we that's And in have as any updates. programs forward should Alzheimer's to update way that there And Obviously, Therapy point.

Eliseo cell your over to MLD we and about turn I'll about to maybe think therapy, so to differentiation? about and as talk And potential how the point

Eliseo Salinas

definitely. Yes,

started with end we program this in So the sight.

approach. of you existing I construct end I of even gene exciting and potential degree excitement, the we will say in and transplantation a in at around very data cell and of with on balance, at and would potentially as this a has know the certain therapy construct about follow could and time, very have it's certain the approach that, how stem And at is of to think program autologous the existing we course, second it's mind, our two a risks. in the what development of an less we papers others. at of course, our and that interest, day, the is development, things. MLD. the its benefits so when benefits populations add or armamentarium type So strong could, Well, say MLD the with certain is our that ages, And symptoms, in that

we think there high into account the that need, specific on that of exist the we're So gaps to even brought taking need. a the and equation keeping medical what with what MLD is market. And in MLD medical eye overall, still an course,

Brendan Smith

Thanks very much. great. Okay,

Operator

you. from And of Guggenheim. next comes of the our Chattopadhyay line Thank Debjit question

Your line go ahead. open, is please

Debjit Chattopadhyay

and you questions thank condolences on for Hey, taking my passing. Tachi's

assay So April, given measurement the in was be the And dosed what that perspective? you biomarker the first expectations a GMX do from an should perspective, beyond from subject safety? squared have out assays

Bruce Goldsmith

Debjit. Thanks,

getting probably what data. fourth we data. know share appropriate and in data year from look the at what data steps and that updates to just to and we of believe at in we'll XX in-house, quarter. obviously, And quarter as and time. can And as have biomarker share that available look fourth you we at the then this to is appropriate cleaning subsequent So And some safety is that programs, what delivering have we've it requires that the is committed other day to

patients run we specific the follow-up. as to then study can't for appropriate. and will of we and But and with So commit report obviously several to that as data endpoints the follow appropriate continue follow-up years also a

what get can think I quarter. in going we exactly the to fourth we're So into present don't

CSF is biomarkers levels And that kind we've advancing assays, Your assays extrapolating working you second differences question I on being in essays that the were job of the think adult this. sensitivity patient both that tell from before, yes that I found will the serum here to has done mentioned developed and the because the children. fantastic of of were the - and group and sensitive because samples required a on

all are those yes, So, enough. developing

Debjit Chattopadhyay

licenses of vectors? would Do Penn, via vectors on provide need the plan likely just routes? in new be and vectors you leverage or announced to the ICM? that separate from would the to then Good, it existing you you more and is And color on delivery choice investment the going today,

Bruce Goldsmith

not transgene, All it et you Jim I'm others satisfying we've is expand is answer. just Discovery part a therapy the capsids know, sure programs, real saying those perspective. is But this fantastic be very as answer have on to we - gene I'll from going that, a and to with And that pursue by optimization questions, been been and of on and and vectors cetera of new also and programs, promoter certainly investing depends. Discovery

optimization will route said, further administration, you there and first develop, we and Eliseo may And develop, So another exploratory do And that available. agreement evaluate way, as it and be to we there well that be I wants contemplated think have the and areas and to maybe may or we this the or the very is to an to be other characterize Once be identify and that ICM, in in-vivo, have really may in-vivo add be or to they that of the program. not hopefully, you're targets approaches reach essentially the research absolutely would that developed comment may is both proof-of-concepts. proof-of-concept that along to right, vector on certainly part of well. two some that obtain stage, in-vitro

Eliseo Salinas

target. speculate about be to it as said, Yes, Bruce would bit early a

Lobe we're this broad and a that when distribution. you plausible with different other happy a Now, intrathecal, and require we just it's initiating at might probably probably work. the approach. One, Temporal But look signed intraparenchymal, they and disease, agreement, Epilepsy, Alzheimer's the

be open are modalities and exploring to that necessary of administration might routes we the necessary. So

Debjit Chattopadhyay

Appreciate it, thank you so much.

Operator

Thank And next our the you. Chico Wedbush. line from question with of comes Laura

ahead. open. go line Please Your is

Laura Chico

Thank you my on taking loss questions. And very my the condolences much for here.

mentioned the question questions but balance how you UPenn applications is on some prepared I remarks, scale and two of the this a this considering. versus good My quick why now, think respect to With CNS opportunities? I have think And of the follow-up? is I strategically to few orphan then a about the you larger you indications, in scale think do the focus the expanded a mentioned why time first. in that layer the collaboration, you're larger

Bruce Goldsmith

when I'm Sure, is question. I'll is Laura. delivered. what make to and pick exploratory We're about Eliseo programs, space, a This not links to over it going with the rare in for and less of so abrupt a believe links the We need like. now built characterize and to brief consideration. about thanks, generally, work and potentially it turn looks talk the not expand certain there this we Thank target, what has and monogenic believe gene targets. we those extension partnership clinic thought and you actually what The pipeline into first identify and Eliseo. then the moving and indication you is comment, we've to on way I'll what is for characterize this. This to a a more can it is our natural

that, it start we earlier way And we're adding and is, about as advance going prosecute we programs, will stage to exploratory in think programs to continue point, the very research the Jim do to Laura, is to the with discovery So will that. this to continue stage, clinical we're interested your plan. it

these that exactly we've well. as members here overall programs. this few do at and are a brought on approach this think portfolio clinical and it and is in members built a Eliseo, can is But we've both do also want team now so And comment you opportunity that balanced to some Jim the comments? - right the team his for is experts Eliseo and we team add this, where interest doing in expanding to

Eliseo Salinas

balance we non-monogenic, pipeline. leukodystrophies, just opportunities. that moving non-rare to be that adult add - indications indication diversify strong it forward clinic, pipeline complement to now we and one diseases, look this there our to the Yes, pediatric monogenic two rare two it's desire in are at you When decision, is very our particular aspects in provide and of would one to and non-pediatric, felt have are appropriate diversity a the knowing

that the ago. this the what two that And years that Penn that you call reflects The would I these target, where approach those started the of second less and two confidence you are working and aspect discover it need increased partnership to into so gene the the replace, if about This translating quality two working than confidence desire years of together. collaboration aspects less faulty and is leading strength together, explain have to the our one right now. [indiscernible]

Laura Chico

follow-up Okay, then one quick maybe that's helpful. And question.

increased younger like that trend emerging Meeting Virtual in in patients seemed July, the of at disease versus patients one in context therapy. settings was was MPS older of So in improvement couple it a gene the different

for thoughts the So how as I'm we're curious of thinking the about of and kind other here GMX, you Any Thanks. and some window replicating phenomena applications, anticipate kind these intervention. of there? therapeutic

Bruce Goldsmith

Yes, the that diseases, treatment of the confirmed to tends we these Axiom there. advance to more be more pediatric in be earlier intervention the that seems

as So outcomes. humble. to symptoms hand, on we're what agree the less for appear we now more we But is remain treatment, clinical in to We the seems life, affected opening that the But doing that research. compensatory be you patients later emerge very other tend research, general, earlier is better mechanisms. know whose that severely in the know, what

remain exploration said, you I what patients the mentioned. in no, accepting later onset about while reality, effects a humbly the reason optimistic as of So I for

Operator

our of question with the Thank Zhong Yun from BTIG. And next you. line comes

Your is line open, please go ahead.

Unidentified Analyst

of about landscape your questions. the a competitor slowing Tachi sorry know into Yun. shows parts positive for it. actually like have expectation? reported to taking place And on that do seem be about is your had really history. biomarker when And think recently the for the my to the much programs in to program. set, reacting their better? little disease Xion is what competitive read you compared your very on Yamada, just let do out FTD you so program, data because met And some thoughts the on our investors some And this progression can bit Thank Hi, natural first want the of Dr. we're to pass not investors I on that how question

Bruce Goldsmith

and Yes, question. Thank thank the for you. the thoughts you

the outstanding that wants that's mechanisms field some our we've always anything, make introductory you for questions also move that mentioned comments. I'll And the different. completely balanced of think to Frontotemporal of suffering But and dementia I I from so Eliseo would this excitement think the with I - forward. turn are in for that So generated, if we're that think raised. him, to one data patients the over are add And hope

that delivered And monoclonal is around that receptor blocks the intracellularly. a progranulin So progranulin the data is then high mentioned that a intracellular. specifically dose of four weeks relatively site every that a and for antibody you uptakes action

double it think essentially how the that, progranulin. neurofilament how as to some those is not normal. to CSF or data biomarkers, are, is understand into I the III the getting answer. fully enough of and I'll And that in a that running is to question of of and that, benefit the of preclinical that think in models those does And what seen cetera. turn seen to comments complex intra are clinical what we've characterize that for studies serum. get in normal questions is translate Phase level means questions one outside further over as we've II point And the does is from the raises where last preclinical perhaps, progranulin et the both point study the that the very make XX And can And progranulin progranulin. cells approach to the it. earlier The mature is starting So well to we progranulin back Eliseo relatively they're levels level that and was a marker, light is, your that therapy is are investors about try that gene over studies it Phase is, and neuronal to in further and times showed our in the models, was level well data as achieved about as I increase

to that really exogenous much gives drive higher us so opportunity progranulin, progranulin And levels. the potentially to new

And antibody. that of questions I major differentiating the this monoclonal is the so that, think, and involve some point perhaps

thoughts? any other So Eliseo,

Eliseo Salinas

think covered I it. you No,

Unidentified Analyst

much. Thank you very

Bruce Goldsmith

you. Thank

Operator

the from comes Please James, with line Danielle is line ahead. question Brill your open. next our And you. Raymond of Thank go

Danielle Brill

me Hi, extend passing Tachi's guys. condolences the as questions. about allow to well. much And so Good Thanks morning. for first, my

So I have a couple.

you. Do thoughts I'm Vector AAV curious on aware cutoff symptomatic FTD? curious you early about defining your for any potential And the have in upliFT-D NfL CDR all at a at then are on you study? meeting, do the implications correlate I'm about [indiscernible] or program? maximum upcoming you're scores for the and agenda Thank thoughts your of with First, how FTD inclusion

Bruce Goldsmith

the reframe FTD it I'll Yes, program, so it's on And actually, question. think slightly. a great I

So faster a higher level I'm I The for X.X on potentially. is clinicaltrials.gov. clinical I study not believe there actually select the are look levels to is the the things wrong, reason please have to early those we're it level early obviously it's onset, of refer does little serum. showed a defined, perspective, at symptomatic patients. scores. this population but that that final great numbers. high optimal CDR a for onset But NfL If the bit combined NfL we believe And some That's And in do in patients. around X to tend two patient NfL is the terms of to are is that study studies that But correlated taking been with a from it's progression.

the CDR other any But ask generally And so I if slightly off again, may does I'll perfect. incorrectly. cut comments no, that's capture Eliseo, pause and there have that or and and

Eliseo Salinas

that That's X of is why demented, symptomatic been great the is signals, comment up to objective that's same to in already has evolution the great trials patients, mean of most is look indicate of that taking future quick can the a disease. dementia a then patients. great of One last of When reflected all for CDR trial field, that NfL is level that this rate where of for and at might we them patients higher data you shared I the this years dementia rate. and might in of X.X difficult in of is have about things by seeing back, what recently, and of the hopefully the including as faster and XX which FTD in the far. populations and pick the decline emphasis the in see But concept so for patients us. the not absorbing predict remember a general, correct. strong surprises, the population you are in And patients, one that to this pre of therefore, and bring a patients as declined on first putting the them

Bruce Goldsmith

will And on we're the come focused very question, obviously second so, advisory on up. that committee the yes,

to the in amount thing but continue one MRI with nerve leadership of Our try proactively I we've and to evaluate activity understanding subclinical on is we're right the of talked velocity that And toxicities, and manifestations. We've that in on toxicity still some been never studies areas. also these that all there has who will toxicities Jim the now focused on one these kind for group, these and any it's actually to and sensory do the these example, obviously five vanishingly our other DRG here to we'll potential that be is also the seen ICM types then and his risks, of in do clinical, one for is believe just thrombotic and experience neuron monitoring Hepatotoxicity, many of discussions that we of on small. right think events, whether internally absolutely study. be so of neurotoxicity, I three only in have or an is companies the area neuro are mentioned But to genecity, of or potential generally the types patients related about generation DRG. to conduction We're real of kind of then clinical of oncology,

but or So of they're MRI X,XXXs. note have the that patient toxicities populations also there example, X,XXXs in for - have - in noted been, XXXs not XXs, the been in

evolving. So, this is

past of And built thinking talks be couple our and want ICM that systemic into been potential of general these forefront this, the obviously so are But area are, absolutely our DRG of about to some these guess design. also we've which very the and we clear using we on the toxicities. comments limit for does of at aware focuses I us about years now

we're So to obviously be that's perspective, carefully. our that current going at looking very

Danielle Brill

helpful. thank much. you it, Got Very so

Operator

question-and-answer may for Thank conference. Thank well today's you. and you you does participating conclude as disconnect. as today's now This session

Passage Bio (PASG) 8 Aug 212021 Q2 Earnings call transcript