SELB - Earnings call transcript Q3 2020 | Cartesian Therapeutics, Inc.

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Operator

Good morning and welcome to the Selecta Biosciences Third quarter 2020 Financial Results and Corporate Update Conference Call. At this time, 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 turn it to Brad Dahms, Chief Financial Officer of Selecta. Please go ahead.

Brad Dahms

morning. good and you, Thank Welcome to update results corporate XXXX third call. quarter conference financial and our The our website release the filed & quarterly press later reporting on quarter Media the available report with XXXX section Investors ended in on September of will XX-Q today our Form www.selectabio.com XX, our financial and for results be is SEC. the

will our our is and Q&A the CEO; Peter Officer Traber, President Chief the Carsten Kishimoto, and Takashi our Chief available Brunn, today Officer. portion me call. Joining Medical for be Scientific Dr. of

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

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

Carsten Brunn

topline we non-registrational ImmTOR today. and with COMPARE our of We acidemia FDA. in you appreciate to progress having Phase as reported to trial September, second or demonstrated us productive MMA look the pediatric Phase leveraging from continue rare team highly continuing SEL-XXX, immunogenic half to partnership of AskBio I This enzyme, Sobi our a in with programs, quarter from topline for designation gene ImmTOR we which morning. pegadricase. Good DISSOLVE clinical the the XXXX. We therapy of joining data Brad. have combined the Phase you, two a with potential III methylmalonic our and in one has this past studies the disease in expect advance Thank III our been II program when busy In from platform. data commenced to case the received forward program

are give we XXXX, protease. and in having IgA commence excited an submission with to option agreement Selecta Finally, for an an to and our announced anticipate access IgA program Biosciences Nephropathy to license recently IGAN IND this program

ImmTOR. released program in a on month this non-registrational with and Importantly, September, late combination immune of efficacy pegadricase tolerance combining enzyme chronic data U.S. Uricase the from treatment SEL-XXX, enzyme that of build of comparing infusions reviewed we will the COMPARE a currently immunogenic biweekly In pegloticase the topline once II Selecta's approved Uricase SEL-XXX in the and case trial of the the infusions for gout. refractory to ImmTOR Phase platform of therapeutic

non-drug by was closures, set numerical stronger missed the pandemic with of conduct this or XX. in six protocol while related Unfortunately, of We on review patient, superiority statistical ongoing deviations. want the the on of endpoint the again statistical consistent will superiority site SEL-XXX endpoints details that through protocol reported data performance all and superiority trial we all with reiterate the to the by XX appointments were primary affected other the the one in on call, we of the the not but the missed per

responses mitigating therapies. of through unwanted Importantly, our ImmTOR immunogenic immune the show at data platform the effectiveness

applicability data protocol the in to XX a demonstrates week patients our ImmTOR three offer that As We programs, made its patients demonstrated only this month when platform I/II to pegadricase immune COMPARE and believe of of it or it in broad were four. Phase tolerance. through XX% per reminder, with six. specific alone, enzyme dosed benefit made set patients XX% out the of clinical

we strategy amplifying and diseases. of lifesaving autoimmune look twofold, self-tolerance Our in restoring ImmTOR of forward gene the therapies, is for AAV efficacy therapies, including the redosing logic

highlights to program and and it's in disorder a combined medical FDA due the this collaboration that are which conducted We MMA announced mutase formerly But with young with the SEL-XXX to and families. XXXX, gene is that look treatment of to their half pediatric preliminary address end This MMA-XXX. in expected candidate for the the to need expected lead hopes rare it We that of and Selecta unmet allow disease MMA program this that with methylmalonyl-CoA patients AskBio gene significant differentiated first helping AskBio forward could ImmTOR. mutations. MMA-XXX of when is advancing for we with metabolic affected in rare believe AskBio product seeking Our the the isolated to retreatment MMA data being by by of in enter is designation recently clinic the XXXX. granted

gene week, As likely last in AskBio be innovation you to Bayer cell that they base announced therapy. and the will broaden acquiring saw

as will agreement AskBio as in do as Pompe is our will to the this program subsidiary partnership As our affect disease. well with AskBio wholly-owned believe result, current expected a operate AskBio, MMA with not a We license progress as of and Bayer. planned

to with forward and AskBio the at Bayer. We are working teams looking

prior various joining pharma expect their and XX that a forward. nearly XXXX, the in in recently going relationship to strong President Selecta most spent know roles, may of to have I Bayer Americas You at as working years my in

and on continues to ImmTOR in wrap To we'll continue to date. at looking the at gene program of therapy combination push neuromuscular program later a on therapy, We proprietary Selecta updates up deficiency do to eligible tiered agreements, gene work, conduct dystrophies. net economics royalties Selecta including in preclinical our certain be this regulatory Limb milestone OTC muscular will disorders, in any product commercial commercial provide payments sales. as as well additional option development, significant and dystrophy license enter on Duchenne girdle muscular exercise we for its including If forward and

nephropathy. IgA onto Moving

During exclusive ImmTOR of rights IgA the the which immunoglobulin license is we immunogenic. option to protease into IGAN's commercialize a entered develop Selecta research and agreement formulization for of provide to the with license and bacterial with the it IGAN option quarter, protease nephropathy. A Biosciences, of the protease which platform the makes in treatment IgA the for with origin The combination barrier an

SEL-XXX Selecta's As intend turn We Traber update shown by the submit ImmTOR call Peter? of over IND from immunogenic end as will the XXXX. product to formation for pipeline. Peter enzymes our has antibodies candidate forward. learnings demonstrated I intend to advance and more to detailed now with on SEL-XXX, this to mitigate the the ability we the to its of we anti-drug a program leverage

Peter Traber

Carsten, Thanks, morning, good everyone. and

pipeline. we mentioned, with Carsten several to updates quarter exciting As the had an

SEL-XXX our Phase pivotal start and III me program. focus Let the on with

in six-month SEL-XXX mentioned, of in on at time Carsten the double-blind which only SEL-XXX, and dose one DISSOLVE safety with per in X.X kilogram consists at we'll extension. XX two first of X.XX enroll we will pegadricase, assess was be dose program evaluated placebo. Each trial of in be with per have September, two months will clinical a XXX studies. program placebo-controlled kilogram dosed of safety milligrams the DISSOLVE and XX and As with milligrams and doses extension. ImmTOR, and trials kilogram, level commenced which evaluated in Sobi efficacy efficacy September. the will each milligrams both The X.X and point DISSOLVE patients six at to six-month In no I, patient will at II aim per

measure in of higher primary patient endpoint We at visible learnings Based II with later both a patients this disease studies of proportion quarter. chronic first expect on to a The to in COMPARE DISSOLVE study, intend uric well-validated refractory levels from acid months, the severity in dose we is serum the enroll gout. six tophi.

a visible the versus percentage tophi the baseline. refractory population closer for visible believe on is patients visible of of to in gout chronic XX% recall, was points specialty proportion tophi delta XX% endpoint, care you'll XX% We the on United only the observed patients tophi the of As States. in and in with to with had at pegloticase patient patients at primary COMPARE XX SEL-XXX baseline of with

counts limitation medical DISSOLVE outcomes patient treatment only program Together of with Secondary data include with is gout the a and approved, life. U.S. forward patients Uricase. III currently second way. if Chronic with that refractory and flare condition. receiving to burden We the in approved tender the well and patients are endpoints estimated topline an SEL-XXX and activity Sobi under for of disease could a need we XXXX. transformative this Phase reporting from The program half debilitating look Sobi, swollen reported with significant with this unmet joint quality as a tophus gout and in of remains fraction incidents, as XXX,XXX confident be living

gene Our advance. programs continue therapy to

XXXX, in the IND and candidate expect first half MMA-XXX We X in product the the commence of Phase the trial clinical to for file for quarter this XXXX. of first

We report preliminary expect the and capsid. patient end of on be antibodies With the we preliminary to to the cohort this by of data, disease data XXXX. at the AAV looking formation of biomarkers first will neutralizing

Our continues progress. deficiency proprietary SEL-XXX to program in OTC

this We program clinic. this continue for the of into to work on activities program in anticipation the IND-enabling moving

inability the on to critical to function OTC control body to genetic rare which Severe raise, updates that is temperature program mutations breathing intellectual XXXX. in due symptoms disorder the this and will We cycle. the in urea disability. developmental early proper ammonia seizures, include share a blood deficiency delays in is of causes accumulate and gene, OTC coma, for

candidate mentioned IgA of IND complexes of our its can we nephropathy most end a of in in to galactose-deficient abnormal result by expect time renal Carsten in disease. the study that factors in as in proved studies of IgA and of of can markers kidneys nephropathy. characterized results disease aggressively IgA forward contributor this product XXXX. and by and stage excellent diagnosis kidney for established IgA clinical result we of protease, is earlier, in the development IgAX are proteinuria of root immunoglobulin of suggest protease which the treatment disease in cause treat deposition disease, progression intend the so independent a Genetic the nephropathy, reverse glomerular the and to and at renal we we immune IgAX is approved which this are decrease estimated to the to improved With IgA to work, prognosis. barrier environmental even There one leading is Previous moving XX% to at patients. of a an currently have The conducted common in it and IND can protease an bacterial results therapies. commercialization For no the kidney makes animal therapies candidate disease. dysfunction. chronic commence causes of approximately to rate IgA enabling end GFR mesangium the injurious the of Hypertension, poor loss with are the which nephropathy, that renal there first renal ImmTOR, develop IgA origin XXXX, possibly our the development or failure. kidney the remove XX% laboratories disease These combination decreased of associated function accumulation and it of It on incremental from the models the to cause of no ability are in is immunogenic. of

We platform. also we restoring for at in diseases, is the look fit self-tolerance in a of this natural the autoimmune believe are potential ImmTOR excited to where

with of primary PBC unmet approach Our need destruction a PBC a PBC, autoimmune medical lead treating root is the causes and autoimmune disease cholangitis of significant Patients that diseases disorder. a targeted well-defined of T-cell or liver-directed to need. antigen the driven cause highly bile the has are a in biliary ducts. target indication

guidance are specific program later. working and We activities we'll on share this for on currently IND enabling timings

results. to Now this the to quarter's over I'll call turn Brad? financial Brad run through

Brad Dahms

equivalents we having We with capital expenses cash. We're this Thank which this $XXX.X our $X.X of will enable further recognized extend impact position of and a incoming release. believe for and third into I'll the in you, Revenue any cash in milestone and cash, morning XXXX us to could runway. on discuss liquidity fund well Peter. the expenditures very ended which capitalized quarter our operating XXXX, highlights financial excludes few issued our press million results, million. detail quarter our the in restricted quarter payments, the guidance first was

also under months under XXXX, license shipments operations primarily the in These $X.X DISSOLVE year. compared in million arrangement primarily Phase to cost payable of of in completion $X.X for operating increase Research XXXX. for done in a Net $XX.X under for supply $XXX,XXX quarter same which XXXX. the the to operating revenue, and deposits $XX the result program change the a Sarepta. provided result AskBio of travel clinical change expenses costs increase for with II to the compared SEL-XXX in used DISSOLVE for for clinical Sobi During collaboration the was expense the other subject the The is compares G&A the the The deferred facilities, recognized quarter million reimbursement The million million of which by clinical incurred III was the our costs compares in for ended XX, COMPARE for XX, $XX.X III $X.X million increase The agreement $X.X activities license. program. cash with accounts assets, net September expenses, the XXXX the decreased Sobi the reimbursement period ended offset are expenses. our in September as due cash liabilities, million, other prior to and the legal nine XXXX in same of were development was million of and incurred the million the was under offset to initiation and the $XX.X by and million and the XXXX from third for accrued in of in collection Phase trial fees, months three shipment in resulting period for XXXX. by result expenses Phase professional for in cost million and activities expenses were a the receivable prepaid by $X.X period increase third $XX.X changes expenses in same collaboration. of work and accounts $X.X provided we and million, of cost

retire be closing. associated also of debt million The Valley hand additional were pipeline the now announced of used ImmTOR further Finance up Carsten. at $XX This month. proceeds quarter, back the financing of and to the used Proceeds debt $XXX,XXX per development was existing support business Bank, fees. facility will drawn million I'll selected which our for from million was $XX amortizing advancement at the $XX.X Oxford Carsten? call to to and to over activities. and we financing During debt with Silicon

Carsten Brunn

Brad. you, Thank

for to ongoing our As result responses. SEL-XXX the and Selecta. mentioned II ImmTOR earlier, the quarter providing to third the potential progress unwanted with advance data immune productive and We Phase was Sobi. look on platform mitigate The validates SEL-XXX its very program forward continuing of III COMPARE updates in to Phase

capsid what biomarkers and continue antibodies efficacy the including XXXX, as XXXX. by on the and we tolerance leverage Phase our at in milestones, expect first for front, To sales AAV AskBio look commence and and AAV trial of in disease SEL-XXX the the I of autoimmune the in We to of come therapies is the QX restoring of file the half to XXXX, amplifying life-saving therapy those learnings the redosing IND of to upcoming formation to preliminary ImmTOR, MMA-XXX the data, program report biologic therapies, proof quickly end of diseases. gene recap of of concept the for gene for

we protease For first the to ImmTOR IND for clinical IgA expect XXXX, study the combination and commencing in IgA XXXX. file the by the end of nephropathy, of

in For top SEL-XXX, have trials line kicked DISSOLVE Phase data expect two off of these and we half from XXXX. second the report the to program III

program our therapy OTC PBC. program in a autoimmune proprietary diseases in in gene intend also to advance We proprietary deficiency and

updates questions. forward that, including to programs look as Operator? Sarepta. happy to on exciting is they we continue Selecta our to advance. population we Lastly, With from will time we're take to an and our partners, and This AskBio updates sharing provide

Operator

from begin from go John Please the Newman question-and-answer The first will [Operator Instructions] question Canaccord. ahead. We comes session. now

John Newman

taking Thanks and all Hi morning. guys, my the progress. good question on congrats for

in gene you So, here Carsten, these just clinical that very look I know us both if that of success in antibodies. formation to programs wonder just mitigating about look the two help technology bit would in sort curious you've the to could really Thanks. different your OTC understand like demonstrate as MMA setting. will talk indications. us I'm a how that neutralizing and shown trying the We the to of and to preclinically you effective is what therapy

Carsten Brunn

we and an terms but question. primarily of why Thanks, looking Obviously, it's a excellent here biomarkers. terms John. the in of disease, that's Yes, But readout. quick in to underlying are that's address success, fairly

expect, in will neutralizing and a look will probably We of can neutralizing the as prevent of terms think you the therapy with and at, immune can formation formation any patients we system. There'll different of prevent of be outcomes that I scenarios that will is antibodies, respond completely antibodies. affects set the couple a there of

can might years where in there's still interventions not that actually significantly cycles. a but think quite we treatment able in can time. order deploy go a not be also which of able And we number reduce are patients these is potentially is many be But course don't there those to over potentially immediately needed also as they might takes also can as that indications, down retreated be a and usually acceptable There's well that between are but or lots is deploy. sometimes titers So immediate we a to allow titers like reduce we as to retreat, of also it duration retreatment, there antibodies, antibodies. subset those where reasons other physicians of then chance that have

clinical will tremendous perspective. we a two but So difference a buckets make think the from first

you the ImmTOR. after that with physician, therapy XX we eligible to this there XX, earlier, as get of the assess fairly and you retreatment quickly As that are is I tell gene actually can said patients you XX, days a combination can chance a

John Newman

technology, you could just now able better question. therapy would their get one are in escalation additional ask dose and their patients Could gene studies they to excuse they whereas sort and this single enable about that's that been how you. me, thank dose what just I talk Great, receive. have for could If of – all

Carsten Brunn

Yes, models the to humans all I definitely as unmet that is very we know, need John, accurate. mean, big end a translation from not that's

So if can't you a of so low the you issues, but window, you you're to remain toxicity if a dose, capsid, dose below AAV too second you still you initially, we running of give patients formation too the high therapeutic expose trigger a give of their still give antibodies, into dose.

the think I was dose dose be you an too are dose second interesting, and application. same viral high to very dosing lines definitely that disorders, can well. another give ImmTOR as where to also low, you potential neuromuscular a the that's to in indications where actually spread important really vector numerous have very add is helpful out – a loads application and give give you where can able doses and escalate an like Along in potentially over it's

Thank John. question, great, So great you.

John Newman

you. Thank Sure.

Operator

Our Kristen Cantor next Fitzgerald. comes question from from ahead. Please Kluska go

Kristen Kluska

taking everybody questions. Thanks Hi, good morning. to my for

So in ImmTOR the one in first the gene is therapies. platform regards to

results unique So fact the gene or potential collaborators? there now ongoing, of and been both trials X,XXX you time has either we've with your conducting adults. therapy, in internally platform think the over for conducted speaking, have outside most that coupled and you given long-term where broadly pediatrics a we're where with where are ask, that to wanted seen clinical I at some now this studies,

Carsten Brunn

we we have a liver now natural obviously and just broad liver-based we we ImmTOR It's Kristen. fit actually. transgene. generated. is demonstrated the that's diseases are company that that see higher very first in data expression the effect accumulates focused with question, We a Right. applicability that There on the where dose a right as get great the actually as a That's ImmTOR believe we of as have is

actually and disorders, definitely liver focused partnerships big diseases focused which on where be focus Obviously, but that's high is the where and disorders medical unmet neuromuscular highest high to as vector therapeutic retreat, and that So doses get disease is need kids The area. have applicable. there's one biotech, big on we are a XX we a is chance have this a can diseases, – a area actually outside – so there’s where and MMA, know indications can in neuromuscular very of of up biggest you then that unmet chance liver stay we where or folds a adulthood, to the why and that's the we grows you to other areas feel between small birth and need and also which other picked a rare redose. we success, liver you there the picked

Kristen Kluska

Okay, the the you. is Thank study. And DISSOLVE second great. on question

that situation. protocols the thinking about the study, there wondering this some were COMPARE in pandemic just given study deviations how in you're So current current

any if and data in the the other necessary for with whether there protocol in are from findings understandings collect place So COMPARE example, the items future. means to by

Carsten Brunn

Yes.

is to So the the indeed this I patients have are we to made but the I'll to study, conduct that's the due definitely pandemic. Peter? one based one mean the also with let changes changes the ensure we in got of study there protocol Peter way have in to in we answer of COMAPRE question, tophi on set a change,

Peter Traber

a Thank address. you. to question everybody doing Yes. clinical very today that needs trials that important It's

this just things DISSOLVE going DISSOLVE all a for up II, virtual are get study, a fortunate the We had from couple study of implemented both virtual name initiation time have how of the of and and sites, them. just investigator which but study I the on are have lot in study COVID a in of DISSOLVE to we that those all learnings been We're pandemic. COMPARE having a meeting we of operational for very visits we kind

the windows, examinations, within studies. home for terms addressing drawing both blood the appropriate doing patient we cetera, across visits In et have needs, of direct both implemented healthcare samples

spending healthcare. make can patients we go effort we home time, to individual to with extra and the So money that are sure

interaction so entering to that have receiving is to between patient also patients time slot, entering for those have to that the CRO. each with patients appropriate up diaries, patients iPad or doesn't implemented for that electronic Parexel fill out, personnel reported direct data information comply be of keep sites with the We receiving We're cetera. the ensuring have joint et our at data. medical

the may all DISSOLVE have So study. have study, COMPARE in the issues things with addressed of encountered that in we we the

Kristen Kluska

very Thank you. That's helpful.

Operator

Raju Our Instructions] Prasad at William go question Blair. next [Operator comes Please from ahead.

Raju Prasad

I have can if and Thanks data little by wondering get as bit the be to a you end the we was of from expecting left XXXX. are that the bit, I'm with just AskBio? of a that whether MMA trying little based more events interrelated on to IND expect stuff to filing for figure durability to kind out needs question. going or is what far what the cadence on Thanks. or durability the color for of what's you're other related taking and happen gene trial as stuff do submitted therapy to

Carsten Brunn

in start, and Kei have either Peter I'll Raj, as well. maybe jump Yes, then or

disease So at the obviously the also, ImmTOR specific of in that well. studies preclinical working we as are the on of impact ImmTOR. is model course, looking both But

is IND work and early almost done year. actually ongoing an that next So track filing for and on

we're So very excited that. about

So more on color. patient we file much if the very Peter add to first in next Kei, half some year. in and to have then track you first want dosed the or QX feel But of

Takashi Kishimoto

Yes.

this is So Kei.

studies. we're the Carsten said, enabling IND As doing preclinical

as So both toxicology includes that studies. well pharmacology studies as

as component the study of on component. And both it's well track again, therapy gene are the ImmTOR a for the as the combination submission. So

Raju Prasad

durability had a just studies, months. clinical put the three where We preclinical the want see on to kind response, potential you see on potential comment one month, time follow-up we'd mean data. just out do I And can a of to so? might of you Great. to quick

Carsten Brunn

the XX XX where speculate, XX, ImmTOR look hard antibody at Yes, of up from will we we the initial that's because I neutralizing vector know it's administration. and the quickly data kind mean mean believe go the after but for antibodies. gave titers after days very we that published I we basically and to combination of setup

we So a fast that's why aspect that readout. least from fairly at is believe

Raju Prasad

Got it. Thanks.

Operator

Mizuho. comes Please from Difei Yang question next from ahead. go Our

Daniel Clark

you for Difei. for Thank questions. taking on Clark Dan our

work for the that can for you exercise For of Thank before us decide you looking what you're just you. preclinical data IgA like understand option? to help nephropathy, out your program

Carsten Brunn

Yes.

in to question the the obviously the hand learnings said much taking the call, I'll where to I immunogenicity we're SEL-XXX of So as during address Kei, we're enzyme. from we, very able but

period So that the prevent you IgAX order certain ADAs color of time but the the on let the immunoglobulin to IgA this. combine approach will kidney, be very, to formation protease very similar deposits dissolve the ImmTOR Kei, bit a in of over I'll in more with provide

Takashi Kishimoto

I from being this around Yes, the limited interesting I while, for really what's is a mean been but think approach really it's clinic in has because enzyme a immunogenicity. tested

think ImmTOR, I that So to disease. combining it the with mechanism novel therapy really a chance the enable underlying of we have the addresses

Daniel Clark

Great. Thank you.

Operator

I session. This the question-and-answer now over our Carsten concludes will back turn closing Carsten? remarks. CEO, call to Selecta's Brunn for

Carsten Brunn

call. excited as ImmTOR healthy. our we stay extremely you today's our continued safe you. concludes joined Thank information this We're of company more about platform growth the look forward the into Please platform. end about the us of who thank the head We This growth morning. and year. to and operator, sharing you, to everyone Thank this and of

Operator

conference you presentation. Thank attending today’s now for concluded. is This

You disconnect. may now

Cartesian Therapeutics (RNAC) 7 Nov 202020 Q3 Earnings call transcript