SELB - Earnings call transcript Q4 2021 | Cartesian Therapeutics, Inc.

Due to licensing restrictions, you must log in to view earnings call transcripts.

Operator

Good morning. And welcome to the Selecta Biosciences Fourth Quarter and Full-Year 2021 Financial Results and Corporate Update Conference Call. Currently, all participants are in a listen-only mode. This call is being webcast live on the Investors & Media section of Selecta's website at www.selectabio.com and it is being recorded.

For opening remarks, I would like to introduce, Kevin Tan, Chief Financial Officer of Selecta. ahead. go Please

Kevin Tan

www.selectabio.com of in XXst, good report full-year and call. December to on fourth is our our press Thank section financial morning. and SEC. will which be results for and Media available results financial XXXX with and reporting year Exchange the or XX-K Securities the annual conference Investors and website, Selecta’s quarter ended XXXX, & release you our Form Welcome the filed corporate today The Commission update

Joining are Peter today Carsten me Chief Brunn, Executive Traber, President Officer; Officer. and Chief Medical

call, certain partnerships various progress limitation, prospects. efficacy, subject the the and without described will projections our in today’s During most are potential statements Form risks regulatory and we financial be of on forward-looking statements safety, made report are XX-K. plans, our the with SEC, about making to filings These recent product including future clinical and candidates, statements the including that expectations, annual and

cautioned and You if would XXXX, forward-looking as XXth, March to on views obligation even to update I like of call the management’s Carsten turn to Brunn. today, such speak Carsten? these change. undue statements, to statements, which reliance disclaims now only are over any Selecta to place not

Carsten Brunn

Thank everyone you, appreciate Kevin. today. Good joining us morning. I

clinical therapies. re-dosing of a acidemia MMA enable new I'm SEL-XXX to step mediated to efforts of investigational application begin, threatening or disorder. drug X, gene trial gene the in treat announce thrilled cleared IND therapy we Methylmalonic metabolic This Phase clinical serious the a critical Before that on to FDA March trial for is or MMA; treat life and X and AAV our

to those with initiate very in and we XXXX and we're pleased half a Phase a disease. was extremely progress patients the XXXX. all families our busy bringing to the business We year propel and forward. And second hope by affected terrible significant trial to to this expect look forward of X transformational

find the a molecule, owned immune pipeline key advanced standard year, with that catalyzing generated a XX showed and of current most suppression, and we ImmTOR AAV pertaining leave or we serious ImmTOR that diseases, teams, preclinical chronic platform. and IL-X, for care for ImmTOR vulnerable expansion induce antigen for with antigen-specific greater I programs, and experience be through combining clinical extensive to in updates combined effects suppression. led Mice our a meaningful. ImmTOR-IL tolerance entered alone of past activity and would we precision potential increase of synergistic the a We number have partnerships expansion ImmTOR-IL to of of dose harboring a strategic of the a exciting with autoimmune to pipeline: this selected ImmTOR-IL By total is have but months. only tolerance data we of of observe to significant Treg and the this target Tregs. a with IL-X are which amplifying our level antigen-specific platform. and with infection out broad The pipeline. recent T-cells therapy; transgenic antigen-specific for of partnerships ImmTOR start gene of little Tregs we We transformative wholly of clinically recent with a our antigen-specific to engineered and patients ImmTOR biologics. want durability synergistic combination antigen-specific combined by Tregs. Over engineered unlocking evolution target ImmTOR immune is precision our evolution for you we administration the side to ImmTOR-IL we potential or that close induction for antigen strategic Treg-selective believe the three to important our platform no contrast, IL-X with dosed tolerance; when for implications from diseases of mice autoimmune Tregs. key the now IL-X is with address from an across without Virus immune walk plus and forward in associated In steps then scientific findings antigen-specific our a need our and By pillars therapies to Treg-selective substantially Adeno-Associated partner systemic immunotherapy for trip like with Interestingly, to which T-cells disease; of autoimmune immune substantial and induction the last with effects molecules our malignancies. Tregs I efficacy regulatory to induction expansion often antigen reimagine immunotherapy believe taken call entire In treated observed reimagining of profound has an antigen-specific the the of Tregs. leap enhance combination, observe are immune transformative

with the patients has variety ImmTOR-IL a diseases. for unlock treatments autoimmune to potential Importantly, of

tested ImmTOR-IL that dose Treg the potency, animal effect of the induce after AAV for in co-administered potential study, for vector an has IL-X tolerance this that ImmTOR. suggest a We complete antibody doses observed vector. In sub-therapeutic allow excitingly, gene co-administered increase alone. a and of durable most selected therapy. sparing observed ability doses also durability We And multiple ImmTOR-IL results to anti-AAV of ImmTOR model inhibition of AAV considered this to ImmTOR through doses an was formation to for days. immune potentially synergistic a with are was These to addition the XXX at the two

potential priority on first-in-class Tregs for not autoantigens that for autoimmune the suffering specific be the Treg-selective to biliary company engineering next continuing design from for of for world the program. disease proprietary IL-X In clinic. we engineered IND the molecules patients Treg protein X, indications but enabling differentiated drive of with today. studies of On non-selective Cyrus immunotherapy PBC and September in pathogenesis a key disease. XXXX, autoimmune to will entered vivo are target of ImmTOR-IL millions development ImmTOR-IL to leading generation the a expansion collaboration has balance XXXX cholangitis Our primary existing total A parallel, programs that identify development a the truly accelerate focus to restores we or diseases pf our molecule. the into be a into Biotechnology, in antigen-specific IL-X

which on Empty clinical later. Moving AAV with AskBio ImmTOR In expand in combined in a reported to studies our with healthy Gene XXXX, Therapy. on of on we work our an partner volunteers, I'll Capsid proof-of-concept

studies expression initial of dose also dose first science the on benefits ImmTOR AAV advances vector. co-administered with published We when transient the preclinical describing enhancing in of an

and combine forward approach lysosomal with targeted in the our of the in to findings supporting the describing properties with hepatoprotective platform. a growing license lysosomal therapy. demonstrating Takeda, agreement global Frontiers storage Immunology, endorsement on a of body in We're rare working diseases next disorders. the their tolerance from multifaceted AAV storage we Takeda. safety in validation pharmaceutical this precision expertise evidence, leader of These one ImmTOR potential encouraged with therapies potentially and strong of ImmTOR. of strategic ImmTOR addition, to as paper the efficacy X, to look enhancing landscape entered gene In On a October with XXXX, for and benefits two build published we by We closely generation of immune a gene leaders disorder the

immunity. applied mitigating therefore address to pathogen a a and pre-existing cross of non-human Xork October expand no infection is clearing platform can combination therapy and of therapies. due many gene gene XXXX, mitigate reactivity other demonstrated and administered. the agreement specifically may designed patients developed be therapy. to window continue would that wild-type our be The issues due the Xork AAV. and of differentiated We has to technologies bacterial gene as AAV Xork very in access a to of with which in human is which AAV can from the an transiently to pre-existing anti-AAV trials who is gene biggest pretreatment neutralizing those therapies the to partnerships a to anti-AAV potential an in with seek patients to more during antibodies pre-existing treatments We that re-dosing. life anti-IgG gene of is therapy dosing their or advance has to be gene Currently power bring advance immunogenicity enable derived treatment and ImmTOR cleave Xork a therapies that some effective ineligible and ineligible of to treatment we AAV for potential up of more gene are for from exclusive for to the to ImmTOR therapy of currently for XXst, pre-existing to few are genetic need are inclusion Xork two the been Genovis immune protease transformative IgG otherwise which also Thus novel to create result antibodies of there announced Xork, of license be to On low responses pre-existing protease antibodies. to limiting patients Xork neutralizing gene has potentially it's to therapies potential to altering patient therapy protease alternatives. IgG that gene that IgG safer the a treatment ImmTOR differentiated with goal antibodies diseases XX% combination AAV for with natural enable to in believe and proteases provide the population patients. unable

to XXXX AAV announced associated Ginkgo and immunogenicity been with high Selecta's of Ginkgo AAV field novel delivery. engineering hurdle that therapy Selecta to also We tolerance design and many collaboration gene and screening cell will Bioworks capsids design clinical is toxicity linked the and has non-clinical This a precision the profile. inextricably on to Ginkgo liver cases, platform conduct In capsids high January and therapy transduction will toxicity major the for gene partnership goal immunogenicity. XXth, leverages advance immune all throughput improving thereafter. doses. studies capabilities a A tropism and efficiency, with engineer with ImmTOR

to to with slow single to doses associated adverse events. ImmTOR of a low capsids vision paradigm dose dose with is combining is Our often the slow more rather efficient multiple treatment serious administration which AAV and than change smaller high enable by

control anti-AAVX of NAbs Capsids humans a at administered formation the ImmTOR additional neutralizing kg. the reaching We monthly of NAbs, response administration levels study inhibited dose determine against The X.X the therapies. formation Based our with regimen ImmTOR mg improved data, inhibit AAV of observed preclinical potential animal doses believe observe Empty in AAVX median by per containing we As cohorts studies, dose-escalation of to XXXX, has announced empty in dose-dependent used from NAbs vector is dose-escalation of alone. at combined saw addition designed per manner gene jointly to mentioned Empty human SEL-XXX of was in administration strong immune capsid day in two with AAVX of durability. the group randomized, ImmTOR conducted a with Consistent X.XX the mitigate controlled, line formation Phase SEL-XXX, we NAbs on and of X first to reduced the Capsids of XX. top of eventually double capsid AskBio. volunteers placebo late partner that DNA, XX, The results the of NAbs day prior similar in mg if at ImmTOR are healthy the formation dose to in of with or kg, NAbs we to serotype no we which day conducted that doses ImmTOR antibodies of the single earlier, trial the blind, XX.

learnings non-clinical ImmTOR will treatment SEL-XXX metabolic the program clinical with gene of an fats. of three trials Empty doses proprietary of is from AAV development the for proteins of monthly and ImmTOR We which our MMA, break SEL-XXX. the rare Capsid body expected therapy employing disease MMA-XXX the being guide our cannot the therapy a and studies gene certain clinical a combination in delivered down

capsid. X, March to CMC second expected want for for and external Phase safety to the and the clinical Selecta to the clinical all SEL-XXX work application and here partners evaluate trial of IND in on cleared addressing neutralizing teams the and The MMA. X efficacy against mentioned, treat the the in is SEL-XXX thank of half AAV initiate FDA antibodies previously formation dedicated will our of at their hold. I regulatory of As our XXXX the our and prevention MMA-XXX clinical trial hard

treatment owned the Our product Transcarbamylase is gene Ornithine of proprietary Deficiency. second for wholly therapy SEL-XXX candidate

we forward XXXX, therapy had prioritize trial therapies, look unlocking gene technologies therapy gene we excellent the we're true owned our anticipated Xork ImmTOR While platform believe of have and in program a the on gene well leader in our our both We clinical and for time and application as continuing ImmTOR progress to originally MMA to the gene filing a with being. as therapy. our to decided of potential leading Overall, as wholly progress programs AAV IND resources across position Selecta in that partnership the partners. seeing

key in of believe XXXX. Lastly, a Patients promising healthcare of updates gout be biologics ImmTOR pipeline resulting is can our and refractory the uricase, treatment treatment approach proprietary biologics, chronic was use development antibodies. immune mature and co-administered an of economic to treatments. response reactions. with to biologics ImmTOR responses antibodies We antidrug our that suboptimal an a licensed adjunct burden adverse Many to in pegadricase the discontinue as due comprised minimize may on Sobi the antidrug color. SEL-XXX experience be offers for to programs, develop or few multiple of forced our highly most the after to immunogenic, of

at reminder, X trials up two mg SEL-XXX the kg. per ImmTOR evaluated quarter doses enroll Each to a a trials, and United In XXX kicked consists two trial third each and States SEL-XXX. and On up arms to mg X.XX X.XX program placebo-controlled to mg kg our clinical per trial kg treatment United double-blind enrollment in study announced up patients, pegadricase one countries conducted has in of and DISSOLVE of of DISSOLVE across being the in the two which dose Phase per patients, Enrollment both XX is X.X four XXXX off will aims placebo. of As of with be of the on been and December I XX in ongoing we Europe. subjects DISSOLVE in and conducted XXXX, is Xst, full States. XXX to II Eastern the of

investigators additional disruptions. steps But of in We and mitigation safety are our screening two we and Russia Russia both as prioritize have address undertaken temporarily the United activation. activated in some well closely additional evolving we and are sites enrollment in sites have and and foremost, nine in been at and geopolitical due randomization constraints and Of II. and monitoring have XX enrollment already potential added and first DISSOLVE as the pending have proactively shipping these to sites, Ukraine situation Ukraine the to offset closed to proactively patients any speed States initiation

the and on clinical with partners impact authorities our the regulatory Sobi, program. will to continue closely We work licensing our trial providers assess to potential

pipeline during novel are the a Before Kevin, which are therapies. then with with IgA XXXX, and IgAX dysfunction. program the Ginkgo on collaboration protease markers We're second run the and unprecedented approach, biologics focus the December working that combined extremely the an the injurious learnings called pipeline protease We to our the our in we and in for XXth, to with call a IgA With advancements milestones in turning nephropathy, immune discover from we immune to disease therapeutic IgAX IgA our value kidneys generation is to first enzyme pathophysiology protease we On X, about financial improve to complexes over to that updates, our antibody potentially excited to kidneys. data believe bacteria. that ended with renal treatments the ahead. kidney Today, currently leverage of clinical influenzae I'll to extensive as IGAN potential with transformative IgA first or want potential the fail year announcing ImmTOR We're driving into emphasize results through program the generation this a currently of October remove Haemophilus generating turn crisis. back innervation our with the entered fourth and fiscal ImmTOR. the deposits. occurs Kevin? that, the underlying Biosciences quarter lower next when an hearts derived second believe of from people well-positioned IgA XX, on immunogenicity these these indication and Current will XXXX. of AX Bioworks Ukraine With we're biologic of accumulate are Phase immunoglobulin has disease, the when in which address humanitarian combines

Kevin Tan

Thank million as million quarter million royalties year partnerships. ended fourth XXXX by million numerous the for of XXXX. you, in our and cash $XX.X the from well-capitalized and primarily be is liquidity XXXX. $XX $XX.X in used by and for operating liquidity that driven [Indiscernible] $XX.X fiscal This into compared which is compares $XX.X DISSOLVE of Collaboration this $XXX.X the We as important conservative December license XX, and XX, with $XX.X December and of in Sobi our believe same cash Phase clinical of operating recognize of X provided Net notes activities for that liquidity incoming milestones the cash revenue period XXXX. forecast net with fiscal XX-month incurred with supply remain $XXX.X runway third the most million reimbursement Carsten. potential periods license the requirements and none costs million the compared XXXX, shipment year for the December was the to our sufficient million considers agreement XX, for the will program. as of in XXXX, clinical million meet from We the resulting same XXXX. to Revenue to and quarter was our

$XX.X and Research recognized Selecta XXXX. under administrator Additionally, $XX.X with $XX.X year $X.X driven the and million in fiscal collaboration offset were quarter the the in a were annual quarterly for million and for million by primarily increases compares XXXX costs $XX.X million $X.X year same were million fourth The and periods million payroll preclinical with the million shipments and periods and $XX.X during respectively, and which quarter of for million XXXX. and agreement by reduction agreement fourth and respectively, primarily which expenses XXXX expense and million development with expenses The Takeda expenses supply costs. for compensation clinical million consulting and $X.X incurred and increases the by Sarepta. were license under compares fees. AskBio increases $XX.X for $X fourth quarter, for programs, General license same fees shipments quarterly for and driven the professional stock in annual in with fiscal

electing sale have expense the of the Sobi For installment expense full results recognized company the the income purposes. income decision the income the out XXXX, taxed value sale that the $XX license for out tax filing. as the methods of Selecta return estimated would the the tax proceeds and licensed with The method, of By opt installment from our tax million. was Sobi XXXX to an part of fair of our on future year to of been current applied of

As a to provides contractual and tiers. sales agreement reminder, as the regulatory payments million up royalty $XXX based in development, for well on milestones, Sobi as sales

revenues any result from a royalties this excluded selection, As resulting collective income. taxable future from these and of will milestones be

For fiscal reported the loss or basic quarter income share per is net net income net basic million fourth million, share we per or of of $XX.X of $X.XX. and $XX a and share, net year $X.XX .X XXXX, loss per

fourth per For full for loss I a closing net reported $X.XX Carsten million net the losses now share. we or of to of remarks. million Carsten? per share, and net XXXX, $X.XX back of $XX.X loss quarter it will year turn and $XX.X or

Carsten Brunn

antigen-specific of tolerance Selecta. year the remain forward. multiple validating with XXXX enter catalysts in opportunities, we've summary, and to seen across ImmTOR-IL in platform, effects therapies believe potentially ImmTOR-IL immune of goal well our diseases, efficacy platform shots and the work a The ImmTOR anticipated profile we demonstrates leap potential evolution our of synergistic continued the the We're look advanced broad safety in and In gene our immunogenicity autoimmune as We numerous the position therapies productive preclinical in autoimmune and With continue in pipeline, to SEL-XXX diseases. unlock of of Kevin. for has the Furthermore, an self XXXX, an overcome about explore our you, additional improve for DISSOLVE entire in therapy confident clinic multiple pipeline. chronic our to as we partnerships. or applicability gene potentially strong enabling immunotherapies growth to Phase pipeline in providing represents gout autoimmune on SEL-XXX, updates incredibly as complete and Thank generational restore the expand X disease refractory to trial was excited forward on diversified to progress SEL-XXX we tolerance biologics.

disease applications believe a aping therapy we're indications, well-positioned ahead, sharpen realize clinical platform. strategy evolving focus with development the on to we gene autoimmune Looking ImmTOR full deliver our our of potential and explore on and

like our their way, team, would today's Before been thank people happy patients supportive the have and also we take to questions. entire With investors, the our that, and conclude along I the call, including many Selecta who we're to families.

Operator

begin and the answer will now Instructions]. session. We question [Operator

first will with Kristen come ahead. go Please Kluska Our question Fitzgerald. Cantor from

Kristen Kluska

hold Thanks questions it Hi. my good lifted getting in and morning. and congrats on for taking this months. four addressing under

to wanted at high level. ask So a

some running the If other CMC you IND making then of MMA in for that these think now, about for from indications, addressing CMC? packages the some but more you an now, what items do related be it whether these understanding looking or agency seamless on could is And this also studies could changes of future talk by hold.

Carsten Brunn

Yes. question, Kristen. Good

one FDA you full specific it's for trial. said, the very example, the out looking even exact setting. of think are give those And which But in the been AAV a have ratio They the clear empty got to early, at We I've all I before nature just usually So I example, Phase and I'm then one capsid disclosed is as we we you XXX. X analytics, think question. around additional questions way asked the the around questions. great. But the capsid haven't more asked of we had the of past, in started been specific

move learnings all for but forward the the And well. then on also we provided our and supplied and definitely partners February yesterday to the analytical as the Xth, it's then answers, for completed into So, work got clinic. the us, answers news wonderful

clinical it's have important clear monthly kids, three that we with actually. in path a doses think I

lot There's regulatory in a ImmTOR certainty provides that think clear I of for for our partners as a path So well. gene therapy.

Kristen Kluska

draft And Thanks. should they published recently, labeled the immunogenicity communicated. be and on anti-drug focus thoughts antibody it. a how on Got therapeutics guidance and for information formation a about including FDA

hear So thoughts these some to isn't behavior, market, across to across impact how your technology? obviously, curious including on this of criteria labeling especially your phenomenon new with come the biologics, the alternatives but might ImmTOR physician if

Carsten Brunn

Yes. think of have It's most emphasis I gout even in ADA ImmTOR. not our the clinical FDA our knowledge, And on add has a we stage to recently much the that And the while X question. quite enzyme, that data guidance chase focused pretty think with ADAs. where assets across a And with great for obviously specifically now importance on ADAs. very mitigation been most modalities don't assets, and ADA address more XXX% a of obviously, the published for mitigation. we where only ADAs actually and to I number have clearly a recent I of we're address think And company if patients we you demonstrated a advanced you Phase with immunogenic puts

extremely encouraged ADAs, importantly, some in in foster this our patient of challenge can the and the I efficacy to interest biologics because induce overcome it degree more help this platform, maybe have So all more of who which and many impacts think even will safety. we're by

Kristen Kluska

seen the ImmTOR-IL applicability. broad you've for Thank and you. about the potential clearly And you're excited data very

current and stage of So, into at programs how earlier including about targets? integrating these this versus your some are pipeline newer thinking indications you looking

Carsten Brunn

us. Treg, Versus but the antigen-specific very gene already. definitely really dose of the address application that's some pillars good therapy. the a We the of we of preclinical is induces excitement think Treg and ImmTOR, basically, We're preexisting where amazing across saw unnecessary We quarter effects And AAV which about expand both pipeline. but shown actually the very induced evolution for expand an as combination excited model data I Treg, and of a The in alone Yes. we We IL-X really the really targeting very there's antigen-specific synergistic lead don't IL-X expand of think, a around see Treg. control which is, the alone all [ph] antigen-specific high We've generation using ImmTOR ImmTOR-IL. Treg-selective antibodies, saw at three of platform. really well.

So that's to sparing. potential be combination dose for this

disease. We the autoimmune excited are extremely about application in

As you on like. Grail, re-imagine indications. disease disease. see antigen-specific the the Obviously, is exciting process kind around on released exciting we're us the if immunotherapy and target of trying our space And we're we've JPMorgan a more identify approach focus lot to for But immune you in [ph] with tolerance Conference, Holy autoimmune just to autoimmune this data data. based this

Kristen Kluska

Thank you again.

Carsten Brunn

Kristen. Thanks,

Operator

Our will go Please next with ahead. Canaccord. come John Newman question from

John Newman

I able And us year. that will be dosing, the question study a regarding the potential Hi, some for of to I initiated design just that gentlemen. talk that later progress. to like be things Thanks specific congrats cetera, study for frequency aspects might about taking have you potential you et regarding share? just my whatever you. ImmTOR could wonder if But question. of Thank of know this the design on I the SEL-XXX in MMA.

Carsten Brunn

Yes. for CMO. our hand I'll this to question. Thanks Peter, the

Peter Traber

and for Yes. Peter This John, Traber, Hi. thanks, is question. that

on clinical trial agreement the trial. the think provide the some the now of proceed, With details I we FDA MMA can regarding clinical

of a as at the United a to allow Chuck investigator XX, mentioned. XX first a Carsten through designated ImmTOR mute of The gene mitigate dose the enhance of gene therapy this XE AAV dose ages to therapy viral progressing and potentially dose have have patients will background, prevent leading models. which gene future with given largest effect the his conducted hepatic the MMA-XXX States. the might XX which trial of excellent gene genomes in vector will be in Dr. through non-clinical population we we to cohorts the The dose two children with therapeutic will previously, in expect MMA-XXX be age administered shown efficacy replacement of be XX. The Just adolescents, efficacy humans. dose then per kilogram. carrying has trial doses at with And Venditti, the development starting This by to and three associated stated of NIH will conducted antibodies, as MMA in with had is animal been be re-dosing, toxicity

episodes sequentially treated be therapy progressing children efficacy a of before the will monitoring to safety The by and and activity, with dose direct levels as efficacy dose steroid board months of the assessment three treated an ImmTOR whereas those three treated and first and data well for with at day and dietary following gene the XX they'll the a be anti-AAV day be on done is two propionic, subject There treated. serum safety steroid in gene CXX, the relative three the administration. on of be of measurement escalation for prep acid subject which The will XX compensation with Each MMA. enzymatic ImmTOR without not then administered MMA-XXX comparison three prescription. measures ImmTOR at will doses contraindication The the be administration be ImmTOR that's with will next following adolescent the dose The given have antibody adolescents effect a in by followed treated and patient. not time production, protocol with clinical are infusion subjects primary of prophylaxis at therapy will will of the status including on adolescents be will a depending of will of and months be provisions will three as patient the get there prophylaxis XE-XX. that of as methylmalonic tests,

development the effect of antibodies primary endpoint immunologic of ImmTOR. addition, to be neutralizing assess In AAVX to the the will

off continue whether three to treated discussions the now potentially to consider anticipate with six this the mentioned the XEXX the we Carsten And Following and or first FDA program or to enrollment with subject in hold of deescalation. the MMA-XXX of even hold, infusing determine and subjects, assessment dose escalation year. we as will dose half adolescents of the children, program trial the with clinical second in three

trial. regarding of are the So some John, questions to Happy those that. details any answer

John Newman

That's follow had long Thank I up the helpful. in just It's The be a able to how follow really Great. you. study? detail. patients was of only you'll curious lot

Peter Traber

Yes.

And I up for years. mentioned follow the as months, be will a that safety the then and subjects but closely efficacy three intermittent the of four year. full assessment initial -- the next over study So follow will

dosing. for So subjects we at the years the five following will least follow

John Newman

Thank Excellent. you.

Operator

will from Our William go Please ahead. Prasad Blair. next question Raju come with

Raju Prasad

the taking Thanks question. for

the XXX, X re-dose how Phase determination made? mention and for you the be potential did On that to trial

Peter Traber

if are Yes. a therapy good can very of gene presence. therapy whether you'd dosing there of Re-dosing Carsten, answer neutralizing related antibodies to I is gene that like. primarily or It's question.

people from So, of for have not antibodies AAV and gene to XX% they're neutralizing generally to anywhere XX% eligible therapy.

have a we guidance. So

studies, when animal would the of the at on In studies. to of done. redosing the had going time were you'll a when antibody at for And allow based we study, endpoint, level In that redosing. the antibody at suppress thing gave rose. production had with So XX defensible. the to we XX we redosing beyond. kg in were dose, would regulatory neutralizing the that doses the be animal we ImmTOR antibodies found and be recall, per level days able we which eligibility XX as will at the that XXX levels mg By three neutralizing antibodies be days, that's that's was think X.X And days same

we question why why and FDA so, doses trial. that's to that ImmTOR will give antibodies three or that that should don't Raj answers us ImmTOR if follow-up? in we suppress if you of And allow think three the doses have of giving the know I MMA it's long-term agreed a your

Raju Prasad

any to plan a just program clinical so there of to trial MMA below your hold TMA Is curious was years younger on any two patients, thoughts there obviously helpful. another ages I point in put and of that's at kind TMA hear for the in two well? is two No, cases two years, and as Yes. go

Peter Traber

Yes.

So, We filled that's for also indications. a as good therapies gene both as very profile gets as well the adverse particular know observation. effect out that

treating cases related that each of TMA. announced we're disease. of therapy That's By gene first adolescents to more the approach And and would the be efficacy we'll two why years course have future, old. lot of than safety children the data therapy in of less children. And dosing. on vector LogicBio the it's given, also that's is very it's And the two not So, just course, concerning. the to to the given a that then taking than time, our goal extend underlying

far seen. we've the hepatotoxicity been, have higher going starting, than seen The therapy TMA, other the what so we're is or thing of been major where LogicBio as is AAVX is approaching the our gene both it's a of most and problems where dose well with of problems that as is have which lower XEXX doses over higher log than

Raju Prasad

Great, then, many -- impacted II conflict the how Carsten in the sorry potential curious the thanks in patients in But And mentioned comments kind Russia of hear the press program on and at on there, complications release any DISSOLVE of trial Ukraine. were being trial you by kind and sites in seen the Thanks. the prepared Peter, overall. follow-up just DISSOLVE to the those in impact are enrolled there of I kind

Carsten Brunn

I'm And a the going give all that today. Raju, that's going questions Yes He's Peter. to great to question. to difficult get

Peter Traber

trials you. thank Yes, to Phase primary a And six I month a Yes, and two review, just months. DISSOLVE period. that II endpoint question. good difference six treatment the extension only that's with extension has I the are X The at DISSOLVE very identical trials is between a

The only year. treatment DISSOLVE XX as the So, November. the a already by mentioned I U.S. have enrolled study XXX is data Carsten period of and November total top line by end enrollment months. the will we subjects completed last And of is

DISSOLVE wide in which The nothing is and including Ukraine. not endpoint, subjects of completion industry Russia in trial II there of and the yet I several the course is still the that has situation to invasion The but will So, clinical has European affect an Russia/Ukraine DISSOLVE completed the a disruption programs of trial. problem. our month has enrollment sites Russian due six

patient been to disruption lack the And our and in-country maintaining we're patients. operations the and and address and portal issues limited of uncertainty with have CRO while main resupply supplies, Our working of payment communication, personnel, personnel drugs visits. such supply interruption the as other closely of of safety of the

of have sites representative sites conserving resources study so and and fluid trials. Ukrainian subjects. sites an the XX% clinical supplies estimate companies patients enrolled enrollment don't Russian very to the or numbers And our the able new in three of active already the situation, temporarily that's of we patient given what to that of complete Ukraine We've number at be and focus total complete. have we'll can about of closed screening subjects many on this active of in we percentages to in time sites kind of other and We and four Russia representing

have measures. couple a We mitigation undertaken of key really

subjects should whether able not to will trial so to power statistical handling to complete study and maintain meeting the already As month. trial United discuss be our understanding This study the in enrolled Carsten of they to in important mentioned, need sites and nine the this activated of activated all subjects those to FDA study enrolled additional we've Russia we've have XX added the be the in a will the DISSOLVE due And to Ukraine and final two second, been be requested the war. States. with to And the be forth.

and continue while our the little the time, while in be FDA and year, assessment situation At we data hard So, clarified. performance addition in newly these by update right is the now it's subjects guidance our to status to of are for the the sites I parts. enrolled have DISSOLVE of all DISSOLVE a present added end will of the assess the we monitored different the our II consultation we'll of line top project to continued with moving guidance as Russian/Ukraine is

Raju Prasad

Thanks.

Operator

Blum will Our question with go come ahead. from Gil Needham. next Please

Unidentified Analyst

Chen in you just for previously. question. to any immunity Gil. Thank is if our default PEG-XX have observed this ImmTOR Hey, taking I want for ask you against the

Peter Traber

what, I'm to sorry? immunity Generated

Unidentified Analyst

ImmTOR. the in Generated immunity PEG-XX, against at

Peter Traber

that what? The packaging, is

Unidentified Analyst

That's "P-E-G-XX" the particles. in PEG-XX,

Peter Traber

not sure. I am

Unidentified Analyst

the Probably have it in particle. the hold, back Sorry. at XXs

Peter Traber

Regulation?

Unidentified Analyst

Yes.

Peter Traber

What?

Unidentified Analyst

was there speculation Because in particle. a

any immunity against there developed is that? So,

Peter Traber

that's okay. And Yes, specific Because there against we good particle. no, case associated Yes, the PEG. in antibody PEG a nanoparticle identified the generation ImmTOR is have question. of not

the is PEGylation. have antibodies generated seen, that there KRYSTEXXA, Now is pegadricase, PEGylated. But also protein PEGylated rather heavily to that pegadricase that's pegloticase, been we've With seen the to epitope the that been the generated have than what to is not more with portion the but we've uricase, the enzyme pegadricase. antibodies

Unidentified Analyst

Thank you.

Peter Traber

Sure.

Operator

question ahead. next from come BTIG. will Zhong Please go Our with Yun

Yun Zhong

Hi. Thank for question. taking you, the

was wild-type you please? one MMA-XXX program. the origin on MMA derived capsid, some first capsid, So, of it us remind from is of the the Can type

Carsten Brunn

It's AAVX an capsid. Yes.

Yun Zhong

That's it, okay.

you'd I another do but immunogenicity study that potentially lessons mitigation with So related with on the from already immunogenicity think the you're and study. deal to that learn prevent of can yes, I to in have observation kind from know just follow-up any a the effects? side TMA observation potentials you sufficient in that combination you ImmTOR any incorporated

Peter Traber

the capsid. one Yes. on the Obviously, ImmTOR and the building we learnings immunogenicity we AAV potential is benefits off were that the and with have addressing that of we're

the really for to ultimately properties NAbs to FDA And Xactually a of comfortable have that comfortable have is what autophagy formation we and that have set is the high that we'll approach field feel experience FDA. in the which if interesting we and most but that it's we're XXX Gout up we trial prevent see and Nabs. very of prevent demonstrated indication also extensive at So, building as importantly addressed but trial, well could we of such from the we probably models the as we've And the animal with secondary comfortable associated they transformational discussed prevent ADA's formation a demonstrated, have we the that related that's toxicity that's and safe least And the doses obviously has of with actual But hepatoprotective many empty the the on doses. with in re-dose trial, ImmTOR here. issues in in formation capsid, the to ImmTOR the and were high our where assumption able be this how we and we're also

Yun Zhong

Okay.

therapy more So, long-term the with that also mean more gene the in could combination in And that the well? IL-X on in autoimmune the say and ImmTOR as for that combination activity IL-XX that direction and decision does I'm to in use or potentially potential deprioritize interesting did and enzymatic promising program, therapy the of also be OTC or deficiency sorry, the has company? diseases you --

Carsten Brunn

Yes.

program, really behind MMA partnerships think an program MMA gene put program. also not number we're pausing capital have saying cautious deployment less savings most how the and given about of we think therapy we're which the very for spend now. money. leads most growing it on the we that the We excited is pausing efficient obviously We're is and we're the not of focusing I Takeda. the for partnerships market with efforts This is a indication and the So, but recently our we discontinuing our we're to now OTC program backdrop cost obviously

Xork. not So, very we use potential gene ImmTOR only you're therapy, a of have ImmTOR-IL. but lot And ImmTOR with in data also see generated of right, compelling we with

sub-therapeutic so demonstrated IL-X which the of receptor gene a in combination a of quarter we mouse NAbs control of AAV two where dose. with after would an the model agonist of basically therapy usually ImmTOR complete be AAV So, a ImmTOR, a doses dose at of

therapy in the see So, application also we line. definitely down gene

Yun Zhong

Okay, Thank great. you, much. very

Carsten Brunn

you, Thank Yun.

Operator

Our next ahead. Ear question go Please Uy from with comes Mizuho.

Uy Ear

question. for guys, my taking Hey thanks

I patients correctly. for months there sure -- see Is the I the if heard end if at the in you efficacy patients? there re-dosing protocol is guess design the first the Just if SEL-XXX. going can trial the wasn't anything at -- Could, don't to in I three for any, back of you

Peter Traber

for FDA and protocol. for allow to gene point door protocol three of this nothing in there, us and results it interested or re-dosing there's time in the no in them back any opened if during the very and the The within -- the months in year. Re-dosing level was going long would we see forth. of that however that first coming there's so but re-dosing at depend No therapy Yes. how to the lasts is possibility provision on efficacy

Uy Ear

Okay.

what patients. saying could you that is after to even seem after So, the doesn't I you guess be and that back months FDA to three the Is -- go retreat assessment, correct? the

Peter Traber

Yes.

open priority So, to talk with in for about for that. that us want the FDA, left them that us didn't approve to were discuss to and very a to back return our open to that. but discussions they They they come

Uy Ear

the I and been, that. II. have can I like enroll guess guess, Okay, what question I treated patients thanks. I wondering patients a share many just is how and in expected Ukraine and on have And targeted you treatment? Russia what to number am have was were completed that second of If DISSOLVE

Carsten Brunn

Yes.

And complete so We be many subjects so haven't next we're the understand of and patients in disclosed number enrolled over is situation to month forth. how not enrolled country quite and Ukraine exact Russia. of the we were are point, numbers and per and certain And certain we've or or a going how evaluated. at many will fluid, those this of

is So exact. we're we at What disclosed had initially capacity that that in not, the say I was haven't number the we up. Ukraine will total projected nearing of Russia for picks

reaching targeted have as well and was target. countries So, those as it number we a other countries for that did

the So, now complete is the situation. six month and very we of a fluid those in them and thing subjects can are treatment that's each key where

Uy Ear

do think your, additional it completed, that I you With your kind to of I guess, you enroll that it's in statistics? hard it you now on indicate based to have think shouldn't would not the that patients -- to guess I have I guess need mean of number do I but meaningfully change patients assess order

Peter Traber

And it's a comparison basically already. as treatment the what Yes. the response And That's has And powered treat of a the zero rate. of was study placebo we're assessing. in placebo. know, versus and the process highly you

complete is would not our why target target So XXX and But see target the the sometime trying do for nevertheless, ago, powered we XXX. any us already The we'll that's FDA was for still whether now to reasons to one straightforward that subjects the for it how in and change enrollment. XXX they to Ukraine of and Russia was be increased assess from consider talking high approximately have enrollment a end. right

Uy Ear

would modestly quarterly help cadence few the us and quarters guess XX to or be R&D you been quarterly of the on it Last similar the Could last sort would question expected year I basis? sort Kevin. million I for the Yeah. guess something for of over of grow spend? about Okay. think the a you Kevin. sort in

Kevin Tan

question. great a That's

optimize spend highest With markedly wouldn't be so resources trying we our of for different we have assets cash forward. to program we our spend the going one reprioritization the I pause have. expect reprioritizing value our to and Obviously

[indiscernible]. think I that

Unidentified Analyst

Okay. All Thank right. you so much.

Operator

question [Operator Boobalan Instructions] Our Please ahead. from go will Pachaiyappan. next come

Boobalan Pachaiyappan

Hi, can okay. you me hear

Carsten Brunn

hear Yes. you can fine. We

Boobalan Pachaiyappan

end. from great. Okay, of Couple our questions

evolve the to success in Ginkgo partnership? do how to for five the over be three what partnership firstly, indicators will So this years? that next And you key envision

Carsten Brunn

Yes. That's a question. great

pillars. know, The true And to generation predefined around is for you is close goal But of have well. as on where we're with we're overall, biologics programs. quickly the possible. certain obviously, of two We capsid same AAV deliver cadence is working obviously to as the for they're And candidates time. the second have they a milestones collaboration two as partnerships clinical IgA as the One have where both them enzyme on the the tell, quite you over as can our protease. of partnership, advancements in about there's deals, excited

Boobalan Pachaiyappan

perspective? ImmTOR combined drugs which Okay. there And to safety to you If most secondly, gene would for assess from with approved combination so, therapy profile appropriate make drugs be regimen might opportunities better? existing the to be a be think do

Carsten Brunn

of future. gene now, both needs in therapies. ImmTOR I limited mean, But of Xork some with the we some a both man, that's the with obviously, to marketplace. or the address great of needs ImmTOR key opportunity portfolio Yes unmet of to the a Xork And potential are and approach a there leverage our Obviously, definitely and the it's right number have question. unmet

Boobalan Pachaiyappan

you. right. All Thank

Carsten Brunn

Thank you.

Operator

now our for This call. concludes back Carsten call Selecta to will CEO, question of and Brunn portion I Carsten? remarks. the answer the closing turn

Carsten Brunn

numerous Thank everyone healthy. this you. this joined for questions. to today's concludes and call. morning who you, operator us Please thank safe stay And and you the Thank

Operator

now today's conference Thank you presentation. attending for The concluded. has

now disconnect. You may

Cartesian Therapeutics (RNAC) 10 Mar 222021 Q4 Earnings call transcript