SLDB - Earnings call transcript Q3 2020

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

Operator

Ladies and gentlemen, thank you for standing by and welcome to the Solid Biosciences Update Call. At this time all participants are in a listen-only mode. Please be advised that today's conference may be recorded. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] I would now like to hand the conference over to your speaker today, Ms. Officer. Financial Chief Ziolkowski, Jennifer Ma'am, you begin. may

Jennifer Ziolkowski

Thank Biosciences third Crystal. conference Today's recorded. Good and morning, Solid the call. being is to welcome call quarter update and business you, XXXX results financial

COVID-XX I uncertainty we extent call, risks about described pandemic business conference of including make inherent the of important section materially the update the in would Before financial quarterly may development the the started, like remind our regulatory Actual and financial XX-Q during product we Risk clinical including everyone no and factors, a statements, periodic and those looking differ reports strategies the on development results, We to forward forward-looking Factors of ongoing with of company's this that and process, results call. any statements future the obligation DMD and date get operations and duration statements other in including to about regulatory other Form of these undertake and statements most in the trial. the IGNITE clinical could this SEC. filed filed to report due forward-looking guidance, recently after from discussed process, impact statements number

I'd For and Ilan Ganot, Biosciences. call Chief Solid introductions Co-Founder, Executive and the remarks, turn like Ilan? President opening to over to Officer of

Ilan Ganot

dystrophy. our you patients dialing Officer. Chief important Also with today's in. Medical company for thank for is of joining are all acting morning, muscular and Officer; living review Technology our call and Cathryn Joel focus and recent Duchenne today Thanks, Schneider, Jen. Clary, advances Good The to Chief us our

SGT-XXX, first the to the the on of The trial IGNITE trial XXXX. first in on track on FDA's us DMD puts quarter hold is Phase in which the the I/II resume dosing technical lifting of

strategic from The Ultragenyx, the second equity collaboration or ATM, financing. we additional a capital brought at-the-market, the company is then with that into and

us Duchenne and living help They us to momentum of XXXX gene combined, close for events patients important shareholders. value developing a the these creating is of further each into therapies deal with our that and individually, we out XXXX. gave they as great for move of forward While potential outcomes also our have goal improve

will recent events in During we overall of today's corporate discuss our call, context these the strategy.

review will We'll provide priorities. then financial quarter our overview our third We near-term questions. XXXX your an and results take of goals and

that reviewing progress, Before our like therapies Solid founded back to recent to on for specifically a everyone and take I'd step transformative focus remind was Duchenne.

we This father rapid urgent of the and As many the experience I'm of these daily are is quality Duchenne. of for deep safety with members need balance can with therapies stark a a the that relationships with know, of other of always a that child have development reminders you of life. need improve that have community patients patient to and There are reminders the that ensuring priority. also outcomes established

potential to overarching achieve Duchenne. manufacturing patients every be advancing Solid of With Ultragenyx, patient future emerging mission to an safety outcomes let for therapy. decision driven into will this with mind, and entering gene to our create therapeutic continue make our in review progress. our much of still that these the current and gene development is The mission has for me of We clinical key candidates. believe therapy now modality, a and additional opportunities driver for will Duchenne collaboration learned to potential the opportunities with therapeutic As about respect drive recent was field with there we for and improving

SAE. trial the a in placed kilogram the a vg November the in event, hold clinical cohort of you DMD experienced IGNITE per XEXX dose most or XXXX, patient As aware, after FDA on are third likely serious the adverse

leading SAE, this submitted and at functional internal on we data information program that inform the FDA. all year. FDA trial improvements, all consulted conducted of of aspects communications manufacturing the efficacy of of date DMD, could it, protocol October and Based lifted clinical safety, with with decision-making amendment to we hold to regarding the the thorough looking clinical and generated IGNITE potential early in review review have in the on the IGNITE a a the experts to this contributed factors our Following and

and as are Our site Data amended activities at Safety the amended including boards has to approvals. necessary patient Joel, IGNITE resume institutional clinical the the well underway Board to completing we the protocol, who made dosing review Resuming following priority. will our towards we top our appropriate submitting protocol Monitoring that screening possible approved in I'll initiating and improvements our the call and dosing, manufacturing review the over to quickly screening is turn DMD process. as to now

Joel Schneider

Ilan. you, Thank

As changes be that part of have viral processes capsids delivered SGT-XXX drug majority viral total from per fewer to with effectively target empty of our allowing achieved commitment implemented years, now process to the load particles, dosing remove vector genomes the to our product, reducing kilogram continuously over viral we patients. improve of manufacturing XEXX the the thereby

with viral capsids. number able XX% microdystrophin viral continue contain delivering reducing XEXX we and differentiated the fewer that as full The reduced As capsids, which the is dosing duration that empty particles are process was full to our expression. microdystrophin can the reduction, comparability do viral would produce patient the This One empty assessment so are of our reduction capsids contribute was that is This for Cathryn administering total without the the our with process of of understand the clinical dosed of regards improvements our number with hold improved safe to will way manufacturing This in in IGNITE a the SGT-XXX pre-purification, required the an trial. the methods, lifting but administered. particles the the assay the now development produces to that protein while levels. our load capsids. of manufacturing combined terms construct all as of changes AAV to to capsids, This SGT-XXX both quantitative expression intended previous means in which protocol of and our key production a total our support DMD well process to mix, dose. to we contain important empty microdystrophin discuss, of and averaging process of FDA criterion reduces

expression that with of the our of This new the to Over vitro process. to expression SGT-XXX we and with were to addition assay our that used We an expressed. microdystrophin years, measures were we've following culture. the last quantify us levels made our process construct, few this between microdystrophin in grown microdystrophin assay amount processes. previous quantitatively developed to cells in And SGT-XXX similar measure enables assay show and rapidly SGT-XXX able from made of expression

in it over We believe in I'll to IGNITE therapies are clinical improved proud significant developing amendments the and process, DMD of will gene that offers review areas require protocol. our advantages the doses. large we HSV-based Cathryn, systemic that who to therapeutic manufacturing call turn now

Cathryn Clary

complement inhibitor, events, DMD in of to between amended adverse inhibitors: Thank also there you, which the together activation. and the we which point CX risk response, the safety more dose system patient utilized cascade, prophylactic that the protocol the mitigate prior believe complement use acts the when be potential drug-related include CX Joel. clinical we esterase and we are each in IGNITE of inhibitor, complement experts an a in scientific immune a After with eculizumab serious activation, carefully about effective in plays the of be with activation believe increase course reviewing the first complement esterase that consulting of advice two post-dosing. onset associated role of To the the loads of correlation eculizumab on different and viral we've the prednisone month complement and may to and data activation may the that Based our time process. experts, and at complement administered consulted the

believe administration potential reduce magnitude will agents both response. of also the these that complement effectively We a or prophylactic of prevent more of

X XX that load in from capsids also per the delivered reduce per We in first with per milligrams reducing Joel next the which empty the are described, post-dosing two to kilograms We're reduction the to kilogram. combination just the total weight increasing dose also dosed month will milligram kilogram prednisone viral of X in to patient, patients. patients the maximum

on limit While the with each review patient dosing and Monitoring Board intend allows patients. we as the dosing won't protocol for possible our patient. Safety and to need to maintaining we weight subsequent two additional Data increase our weight additional the after we'll next X before them patients, dose while After next the XX-day data increase laboratory patient We rapidly that, X approval with from the for focus FDA the of of trial, an strong our a to the patients, totality in as in move clinical move safety. on of

to submission also for patients data FDA to to included Ambulatory As data. functional collect the mentioned dosed the Assessment earlier, updated test IGNITE included date We're walk and continuing turn XX-month we call Star look and to This IGNITE assessments will six-minute an all North Ultragenyx Ilan, collaboration. from and DMD. in our efficacy DMD, results to of these sharing now patients I back six the at a with all dosed the time. in appropriate forward for review you

Ilan Ganot

Cathryn. Thanks,

nitric goals Ultragenyx' chance platform our with a potential which announced two the with neuronal expand for AAV differentiated therapies first, gene is collaboration that Duchenne variants. NOS of options. goal use provide Now with it oxide the of microdystrophin producer we cell me these great to the turn for has patients synthase, strategic or line let containing additional with this pipeline achieving believe manufacturing Ultragenyx combines domain, reasons: for of We ago, therapeutic HeLa to and collaboration the weeks two

a that with the creates used to therapies patients therapies with microdystrophin manufacturing excited vectors disease in are and to Duchenne. of multiple for develop to opportunities be in We working multiple for leader be and with rare additional collaboration our field this processes unique

SGT-XXX, by Ultragenyx through investment under potential to development from provides longer-term developed for royalties move the the provides creation enables with the with us value capital and opportunities generated programs and collaboration. of Second, us forward milestones that

programs in proof or increased participation of of a royalties Upon share $X.XX royalty made net return collaboration up on right sales. has part has upon tiered and worldwide in collaboration, and the pay profit in to achievement of $XX Ultragenyx for As investment $XXX the in in milestones certain agreed to share co-fund the concept, per at million this to payments. achievement opt million so Solid of to it

opportunity as I over exciting will to Importantly, AAVX additional move now quarter Jen construct microdystrophin the we SGT-XXX to the we variant. partnerships outside our collaboration, with third well establish for as call or financial our of results. to a around the rights SGT-XXX as forward this turn retained full review

Jennifer Ziolkowski

the provide has Form in which additional XX-Q approximately September XX, and the activity, runway to XX-Q, financial our is filed Ilan. detailed second how results $XX focusing than we you, of for regarding our cash quarter on provided Thank contains recent detailed Rather XXXX. an Earlier update the today, our available capital that's like on XXXX, half information the million which ended website. Solid resulted of into I'd in extended that

As XX, we cash of million. had equivalents and cash of September XXXX, $XX.X

strengthened As that balance events Ilan noted, in October, we sheet. had additional two our

was The $XX collaboration of with a Ultragenyx, strategic the investment which in first upfront Solid. million execution agreement included the

dosing in turn In an net the our These support XXXX, make clinical to million. the objectives in IGNITE enable now addition, Ilan. I'll to reinitiating $XX.X that and manufacturing cash completed additional financing in back quarter. resulted of resources first including of equity also DMD progress in we call further us in ATM proceeds

Ilan Ganot

the and months continued as to the their several and resolve clinical been has the hold our shared resume the patients and and our support to Duchenne what DMD I last physicians mission. a efforts at opportunity the Solid. dedication everyone like we for trial After community take We is their committed thank to over year, patience remain thank for its time participating of and who employees necessary their advance we exciting our for steps SGT-XXX, dosing are this an in would rather to the challenging to take this members for to support safely. and IGNITE as

Operator

[Operator line of And first from with Instructions] Joseph SVB question Leerink. Schwartz our comes the

is unmute on unmute Your your line is it. please line please open. Mr. Schwartz is mute, if Mr. your line mute, it. on if Schwartz

with from comes Martin Credit question Auster Suisse. next Our

open. is line Your

Mark Connolly

scale goes change you process capsids confident my Hi, are as up taking you'll that as everyone. understanding dosing of curious lines. IGNITE the disclosure first, to Marty. as specifically, a just you this – you on on far before get of And to QX. your total further? – to quality. be DMD mentioned, curious questions. in resume the able to do to it's time data And your Thank Mark change? realize fraction details patients This with how study, well manufacturing could new first then advance I'm manufacturing efficiently early, for then many clinical your plan terms think patients? I'm itself, you. more how you manufacturing of as or the trial, soon And from do but in process you And And you guess, I little is full what the was how better newly dosed plan for Thanks if all product for IGNITE dose how with I investors the two? improves provide specifically, you

Ilan Ganot

questions. Hi, for thanks the

IGNITE take DMD manufacturing going is maybe to about Joel the will one, and think after. in Cathryn I talk

Joel Schneider

question. for thanks Sure, the

manufacturing synergistic was to aimed improvements our the were mitigation clinical As around we really with strategy our highlighted that strategy be FDA. have to previously, proposed

by reducing patients. removing from SGT-XXX the effectively load viral the empty we're specifically, drug so given total capsids product, And to

And having about now capsids number the XX% highlighted GMP full of we're mix. in we that runs our so what averaging belts, a today, under manufacturing

empty. were produce and methods fairly about as forward. to their or experiences as the communicated those be and information we and We're proprietary. a educating mix a at in this think is and bit little trying of not. reminder, Previously, forward-looking isn't often XX% we eliminate really full either a empty. to or path full general, choose about and about all And XX% capsids, It on And shared. So full empty based sponsors, a XX%

of significant really for meaningful removal and capsids and ourselves, really total empty has excitingly the So – the of viral this in a importantly reduction load.

with would continuing back level into So fourteenth going of same that smaller that given. clinic our the vector a but effective be with doing patient kilogram, dose, we're times per total load so genomes X the ten dose to any

and clinical we exciting IGNITE and as way so mitigation to And this assure is think alongside we into go back the try strategy, compelling very a safety DMD.

I our think was question related to the scaling. second in confidence

transitioned we into program adherent importantly, our well to an suspension think clinic. a going method I from small-scale manufacturing before process

manufacturing we're as be both the our And that improve our suspension-based SGT-XXX That's day we in CMC so until methods, as we're with have been the to development. lock the so method. been method. vitro with done going analytical on And the that continue doesn't We're, to the improvements manufacturing had I patients fact, responsibility. the down all our related done. That expression up in think to on dosed how recent we're really accomplishments, mean improvement CMC well forward-looking highlight trying to product product

more clinic fundamentally, lot there's so we've back excited think to and we're and I improved do, looking dramatically hard a a to the forward to of And done certainly with work, going but product.

it hand clinical me to Cathryn your Let over answer to questions.

Cathryn Clary

Thanks, IGNITE Joel, DMD. questions for and about thanks the

of place in activities now to back getting couple a quarter. that, Ilan about happens as there's excited patients. are first the dosing that that truly are ensure into our And clinic and mentioned, next We taking

with the patients are closely appropriate and different weight, send We the identify who and investigators the the are regulatory protocol our IRBs IBCs packages our sites. to we at as to at working

data two first patients, we Our able first we're new strategies, new the each we XX going safely. which patients be with will two and to the that cautiously confidence treat our days on process of risk before us for next be proceed to on move we'll with gives patients manufacturing mitigation board, patient. review of – our and clinical But laboratory DSMB will

from second So on first DSMB, the to evaluate we'll the patient. quarter, that the with patient in patient information move we'll dose first the

decide patients that do we depend year. next dose to So how on many will of some

but – that We actively throughout we've plans, but some yet, do we haven't year. plan got to revealed number haven't dose the

safety time in of currently, the test. all one-year clinical such at evaluation this the is Star to appropriate In that first decide dosed data, to the the most three our will six-minute six we of data, as we're getting the and walk the three And evaluation. SAE, data at the functional and efficacy terms reveal the low compile data trial, in and for had which his week, disclosure, be at when it is patient sixth the looking North hold, patients, led including dose dose, some actually who high that and the clinic

Mark Connolly

Okay. moving the next And on be would some we Got it. just even? get of let's then one, you're it dosing Would say, patient? you're so as one least sounds, disclosure like by you there that at sort potential of signal to would

Jennifer Ziolkowski

patient? terms moving next In terms should… when when I moving we're on we're of the Maybe – in of to on

Ilan Ganot

jump should I in. It's Maybe Ilan.

Jennifer Ziolkowski

Yes.

Ilan Ganot

to We're disclosures. to be the would quarter. important not needs we have providing we say that that Jen. around disclosure everything the patients Solid, not feel dosing to know, guide come are we're on to again in to this excited shy would that's will first level the update of just We be probably that from going I and you and market

more mentioned, behind data Cathryn a is getting gathered lot As it.

on – appropriately provide timing will over understanding something hopefully You on for, dosed. collected new asked Again, to we we're we're going we're looking comparing for very what also it and patients patients, we for to, think will when biopsies. data patients and be year. data a on dosed but have all be those what what already And six what next of will good having years I that shared from certain endpoints that guide two not for clinical the today,

Mark Connolly

for Thanks all great. Okay, the clarity.

Operator

comes [Operator Schwartz SVB Instructions] next with Leerink. Joseph question Our from

is open. line Your

Joseph Schwartz

combination the quantifying or something specifically like congrats just you tools? of different? the very drugs for and including be of would to something Or it ways Is two? Any as could the amount SGT-XXX. it mass describe about I characterize here you're if how learn semiquantitative like insight wondering interesting continues a best western of it some Is to the much agency, was the very Thanks what blot? more Is spec progress. Great. like us on potency microdystrophin, everyone, the

Ilan Ganot

It's glad in This is from the you Joel's you, good work. I'm Thank you. to got hear to phone Joe. corner.

Joel Schneider

Sure. immunofluorescence, how expression to using very muscle methods, idea the We membrane. Thanks, a is multiple protein Joe. microdystrophin good us quantify the the gives localizing of which clinic in

both spec. also have from and use data disclosed previously blot mass of western We the

we're amount of that capture So we're to methods orthogonally really looking at three and all expression collectively protein the fully detecting.

You're absolutely on clinical clearly side. the also just evolving, but right. agency's CMC expectations The not the side, on are

And think been few expression protein releasing view as way of on as something and is envisioned we've setting expression that – to improvement improved protein a our this working lifted. team and And and recently cells, of to in excitingly expression comparability in manufacturing to the GMP expression vitro which really to vitro a way get well. we're method. our hold that a the it's the of vitro assessment of so this most we FDA part actually a especially viable microdystrophin manufacturing in years. how the that assess of a see a during also clinic, to comparability, in quantitative in assay to way expression be in for Similar specifically terms strategy utilized demonstrate have path is quick has product. But of manufacturing variety our we expression characterization excited the I was we that the in setting, developed ways different a would culturing that We the very produced in an very microdystrophin having methods demonstrate

Joseph Schwartz

to rights partnerships and potentially additional additional and And you then helpful. able of at even are you like that that the about other do deal SGT-XXX explore or thinking desire, versus That's still know vectors you if product full around How Ultragenyx partnerships great. retained vectors Okay, you're it. securing to them the just avail to yourselves I following Thanks. to bring explore deliver and that yourself in-house other all, perhaps did?

Ilan Ganot

still access. the I availability to know, made more Joe. opportunity been product, distribution, take of field, that for the and create it. has we're there's very by time I here, very background concerned global And despite us question, drug the mean, many to at look every you others manufacturability, of am going and a around I serious Great lot by patients, in Thanks. an think options access, a progress

a therapies we and including early for been to we next-generation to keep constantly goes on that trials, on identify in promoter are Same them Every we've things. gene were would in the to a a the that potential now, to six improvements showing process. are delivery manufacturability that the mean, of things systems. now five, gene FDA, of They in clinic and showing products front today we us that years ton. that about keep of looking recognize in data, that learning collaborations how very proponent allow also and interactions days. number we We for vector and is how It's also weren't. research do drug patient are muscle and now, world iterative the and next-generation even vectors things. easier with public We're many I do a things biology. delivery, have better but And and vector thing

example, always manufacturing, other impressed is work great teams a of open, great interested very Ultragenyx work and to always with. chemistry then a and that with in-house and are collaborate of but the lot regulative brings people a like-minded somebody with regulatory, are between capabilities, in our labs, we groups. lot delivery excited to also So in that

Joseph Schwartz

Great, thank you.

Operator

any for am conference this showing and call. not your concludes questions I today's further participation. Thank And gentlemen, Ladies at time. you this you. Thank

You a day. now disconnect. may have Everyone good

Solid Biosciences (SLDB) 8 Nov 202020 Q3 Earnings call transcript