SGMO - Earnings call transcript Q2 2018 | Sangamo Therapeutics, Inc.

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Operator

Good afternoon, and welcome to the Sangamo Therapeutics Teleconference to discuss Second Quarter 2018 Financial Results. This call is being recorded. I will now pass you over to the coordinator of this event, McDavid Stilwell, Vice President of Corporate Communications and Investor Relations.

McDavid Stilwell

Hello, and thank you for joining us.

As we begin, I’d like to point out that we’ll be referring to a slide presentation today. of a on the page the Investors Media find our presentation of may and www.sangamo.com, website, Events section you link slide to on the And site. the and Presentations that that to like are the forward-looking this everyone I’d available we the today. also during information remind discuss upon have projections that and statements we based call This information will likely change over time. obligation future. the you today, undertaking an with performance the we’re in Sangamo to of future updates By discussing provide not we what our discuss and results are may from Actual a that substantially should one date still at comments today, assume differ no today valid. later from These Exchange and performance and assumptions Commission, report on specifically statements with and that are uncertainties detailed of are files risks, documents our quarterly XX-Q. in annual certain are Form not XX-K reports on that the Form guarantees and subject our Securities future company to the made stated except undertakes update duty statements to today forward-looking applicable The of under are Sangamo and this no required law. information, date, such as as call Gary me Officer; Ed management, Officer; Ed Financial of General Senior afternoon Chief With Vice Heather this Chief Yi, Officer, Therapy. Chief President and Kathy President are on Medical Technology Sandy several Macrae, Rebar, Counsel, including Officer; Conner, Sangamo Lee, Turner, Executive senior Vice of members Chief Cell and this Again, those of section our we during slides presentation on will and Investors refer and Media site. to this are found call, a slide the to be And now I’d like call Sandy. to turn to the over

Sandy Macrae

our we visible. This that from corporate on already genomic to strategy, second in patients’ hosting our good clinical everyone results transform flies; the positive the evolution. for afternoon our preliminary evolve all Ed we In is our announced the and process of data therapy and hard that into the and to AAVX. efficacy us. our McDavid, to a continues specific emblematic a middle Sangamo is gene update, call. a potential to more SB-XXX company, achievements more the in dedicated the becoming have transformation and progress it’s and are Thank first efficacy you, data program this research hemophilia development call, believe novel of call. therapies this Time This Thank quarter in highlight evaluating joining the will the afternoon using to medicine discuss summer you in clinical A. clinical first emerged the of from we from today’s development new On will is trial Sangamo lives. Conner for

our required genome an key we Sangamo of recently published trial genome for the about SB-XXX parameters serve our to will emphasizing In will our recently for on results preliminary building We’ve point of Ed two And editing. immunology been has technology years, ends. data therapeutic discuss II. provide present appointed expertise. SSIEM, evolved development important studies also viewpoint at gene cell interests safe his technical and He’ll effective in CTO, Rebar, therapy of Sangamo our first editing. the fall cohorts MPS therapy for where in Another X few clinical editing vivo September at last our evaluating

through cell intention to build of to the to therapy Our or was to immunology. development CAR-Treg acquire novo de for capability the field lead collaboration,

of update acquisition. Chief the I’ll including to Ed function for is development allow organ to our the Kathy accelerating position closing CAR-Tregs emerging later to transplant to highly turn the years. now in of finances, on timelines will team And as Yi our first TxCell of of and plan call, a our to them leadership their impact Conner, develop solidifies upon continue acquisition in Later subsidiary diseases. an immune call is Sangamo. the proposed a intention in to the Medical provide this setting to high clinical prevalent two solid auto treatment and Our TxCell field, performing up TxCell We for by Officer. our the

Ed Conner

study the spend this has programs; two the our call. release SB-XXX good latest I’m X/X I’m in with pleased A. time Development our Clinical developments top I’d Thanks, the information therapy for vivo Group from II. to from for MPS editing and candidate genome safety And some results like the we some and clinical everyone. made of of discuss Sandy, discussing to quarter SB-XXX pleased the lead since afternoon, on candidate Phase press Earlier this from preliminary announced hemophilia afternoon and progress efficacy gene hemophilia call, line our and alpha A first for

we of per study have to we To-date alpha results stringent an the trial dataset detailed safety tolerability a only at in has annual and ASH year severe The opportunity the and embargo at then conference. present meeting. this to with patients intend patients SB-XXX three preserve cohort. been by designed to subjects adult so therapy and gene levels, seen ranging far, hemophilia treated XXXX Pfizer the will the we investigational discuss so excited and until dose requirements, is We’re the order in and highlights few later have present the open-label to assess A. two five dose clinical ASH at

third reporting activity the well study, receiving in with or effect substantial tolerated of indicate reduction second been XX data therapeutic the no level factor use treatment patient fifth XX% of at serious oral the rate drug. The There study, steady of no SB-XXX the first adverse in Slide of factor the been is the has A normal to above achieved believes rate treated has elimination been steroids. activity top have usage. cohort replacement use Turning patients observed dose in courses with line dependent for levels. tapering dose factor that events. has after spontaneous summary. reduced of associated factor related Epidemiologic with in generally

patients, observations five with will study confirmatory the first in safety escalation. appropriate meet will Next who that month. is scheduled this additional trials. treat – use then Based committee I’m the for or patients expansion this recommend that protocol are we SB-XXX the registrational determine monitoring confident either study clinically for to dose specifies steps later for from six treatment and The the cohort these on

on the the study, dose Society held safety the the SSIEM cover Last CHAMPIONS completing escalation for an also II study. dose Turning of webcast The SB-XXX in available of Phase replay editing that we CHAMPIONS now first the or the is Errors investigators the of portion announced watch our evaluating I who the SB-XXX clinical MPS I’m program. sixth CHAMPIONS from to Joseph the presentation report The cohorts. the the website cohort Study goal with at to genome of the encourage preliminary was and recently II. MPS data second we of which the We it. webcast vivo from Metabolism Athens, annual to initial will of Muenzer, webcast highest be week on Inborn September The we’ll trial. last symposium treated it treatment in in study two the of principal educational to haven’t those X/X dose are XXXX and Dr. for one presenting preliminary the pleased the and X share lens our week patients, Sangamo through seen Greece. efficacy

production important Slide is disease In a replacement is MPS in steady on the subjects, a IDS is internal to normal and therapy state cells the the how or IDS the is transport amount to enzyme enzyme delivered differences cell inside cellular and XX, into that it why A imperfect out on effective, II of understand healthy cell progression with produced receptor not replacement understand circulation system. leak by extra therapy surface. taken enzyme enzyme As it’s the established up and shown small completely may ERT. due back to as

of most enzyme normally in the to concentration create a circulation. in small deficient ERT body is is produced the the contrast, uptake result, to concentration of weekly found a enzyme large bullets of As tissues. circulation allow high the IDS In tissues designed in with in in found infusion

ERT uptake of editing produces window exposure would produced hours In and from mediated cell continuous be sustain exposure SB-XXX you IDS II receptors produce continuous because amounts tissues a our allow for minutes an the genome illustrated has ERT followed of for the stable within Instead, the life circulation, by That maintain as levels MPS transient to half believe goal to taken as through amount to We for to clearance circulation of due in a only prolonged the will therapy for levels XX, XX. a is on surface. is on However, by the the matter tissues. greater by by low and IDS taken the potential large approach short rapid of Slide effectiveness. up short liver. process. Enzyme enzyme exposure receptor Slide to on ideal and its cells see allowing the are of the limits abruptly high a

learn in the IDS. as at CHAMPIONS safety levels various doses liver SB-XXX expect produce of well potential cells of genome for We to editing as to Study of therapeutic about

maintain and that may XEXX the and circulating as ERT enzyme well drive levels data low model clinical cells Our pharmacodynamic data expected sufficient four to expect patients be and could we At two vg/kg. dose of data levels XEXX to next enzyme report using SB-XXX in as preclinical very pharmacokinetics, cohorts; preclinical suppression to month, suggest uptake be circulating across GAGs. from of SSIEM of sufficient

safety, in urine on We expect plasma report to GAGs. IDS

all from Moving has to is six their reviewed work patient to investigators planning them withdrawal ERT with once subjects been and for the Sangamo data provide SMC, appropriate to Slide data XX, by to patients. determine

ERT an SB-XXX. important of have weekly establishing before, urine withdrawal relevance As after stabilization clinical be we parameter will GAG said of for level of the

SB-XXX We’re evaluate also in to our plans patients. younger advancing

once U.S. in are younger amended CTA similar the safety plans the first efficacy for adults. This is our our protocol in recently patient the for populations, allow allowed pediatric the evaluation six UK. treatment review, FDA to of to in and was established Following

MPS active subject trial I’ll treatment progress was treated in were X/X the announce review quickly SB-XXX programs. concluding, three we of at July, In other first X. Phase clinical first the That to in patients for the pleased mid-dose. Before

treating are U.S. and As to with SB-XXX, as are efficacy the UK begin in authorized soon we established as safety in adolescents adults. and

We’re progress our clinical of ST-XXX in the development program. making also beta-thalassemia

across enrolled three the BIVV-XXX We several this disease clinical have patient IND sickle in the openings is study. sites anticipates sites Bioverativ year. for and first now is States and United proved The clinical for open cell our

hemophilia the for SB-FIX challenge to a U.S. B Finally, in recruit remains

I’ll track We year-end. activate the greater clinical technology remain are as These by data capabilities into to grow in to be now excited turn those such sites organization data-driven accumulation for back of Sangamo, times exciting company company. the a UK an providing to transition This efforts time Sangamo editing product focused I’m are initiate certainly call a and into platform. the development. Sandy. clarity at the clinical to a working for and allows is sites clinical part to on from transformational of our on the of these

Sandy Macrae

to Sangamo, introduce been a designing I’d received XX very take strong voice at Zinc development for studied Ed to editing, Technology. Ed years Chief the Ed. use. sequences. has editing for developed the methods genome of our a proteins finger he’s like for optimization to he novel has Officer. for our Ed of setting Finger that standards Rebar core and and field high Technology Thanks, MIT, target recognizes moment just technologies for led being therapeutic he his In a leader In PhD zinc here

to are Ed? CTO. Ed delighted have service our We

Ed Conner

and good afternoon. Sandy, you, Thank

role The this treatments never currently diseases begin into by juncture. how curative have of I important onetime been this Let am saying me for at stepping prospects intractable greater. excited creating to

the editing opportunities and specific. sufficient require sufficiently will realizing reagents are an developed has potential precise these However, of full ability

for target nowhere using Florence, target of Italy. clinically that reminder, modify vivo presented As to site defined in we and stabilities, discrete XX. preference to sequence targeted gene and is relevant provide with modifying we two by of achieved expression. the else. these Sangamo, great within in In to performance create cells indicates results that the Conference editing a assay I of them the efficiency than is Engineering the threshold capabilities. take summary At on-target antibody-based work, ZFN developed here targeted gene our the to completion In critical off having is moment of of Efficiencies analysis off-target specificity Fassett XX% ZFNs and of focus cleavage, Sangamo degree those for like a gauged conditions. assays. less of of to of underscore for at study that Genome and you Recently, a specificity disruption and locus create the specificity on to specificity high X.X%. to of delivery the Slide to presentation, target only two desired T-cells have genome A virtual designed provided the assessments. these if take no With physician only the same exemplify rigor detection within a achieve Sequencing no searched receptor the evidence for we ability no alpha is in this and of our relevant demonstrated would the modification. to with target clinically Since off-targets key platforms finding genome. ability both reviewing with and the pride as respect samples modification intelligence site to down a cleavage context a meaningful

Our stages. specificity assessments two were performed in

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much high, of cleavage Despite seen. these which for than activity to seen shown assessment low no the X.X%, ZFN in carried modification. is threshold in have sense, XX% for were higher on-target assay were typically and in an that the the stages subjected very been that work we significant levels off-target only same of sensitivity have nucleus. of not evidence both Critically, design the down mutations itself than reasons. we evidence study, verified despite off-target studies modifications off-target of sites in Next, candidate cycle the specificity This in but T-cells of absolute less of potential three other was this

reported, clinical First, it activity. conditions have knowledge, target of the cells the using and to with our modification levels complete absence highest and delivery detectable represents, relevance off-target of

of original result for this pXX our not initial application intent study, test mediated ZFNs. the an therapeutic limit although, pervasive provides may the apoptosis of direct Second, whether

ZFN upon and in sure seen. at As manifested response will study be exposed for studies, cells recent can was we such Unlike which to CRISPR/Cas every nucleus, response classes extensive and the X XX%, seen. CRISPR levels exposure widespread studies. was was any crisper therefore, hypothetic virtually modification to study, of our suggested published in no loss our With that that be in cell

to highlighted Treg. editing turning The study efficient very Finally, capabilities Slide highly T-cells, T-cells the or XX. the modification multiplex regulatory by enabling for of high foundation provide including

at achieving with capabilities along nucleus pursue specificity others specific be of our candidates, that engineering autologous as the calls, with our that sophisticated provide the improvements. we as existing oncology including will As precise update. anticipate generating excitement great any discussed my provide to therapeutic our development reaching generation populations therapies sufficient I’ll engineer for financial Treg platform CAR-Treg of our editing and TxCell me full call enabled generally, engineer Kathy based well of More that range today therapy. we’ll to product in our of let editing again of zinc potential next have confidence well prior we nucleus be platform for for opportunity. precision With for patient larger summarized, and in now by highly efficiency In acquisition, able editing have for we prospects to announcement multiplex therapies and as performance as key diseases. further gene required to the turn multiplex finer on highest have transplant highlighted capabilities, and the nucleus allogeneic T-cell broader through will we the indications, and The closing, I projects of potential rejection the express biology. autoimmunity cellular are enable autoimmune

Kathy Yi

per quarter the Slide a on on everyone. detailed financial the today reflects from and press equivalents $XX.X We cash, Thank you, share payment current an Revenue prior $XX.X the million or million XXXX. with the Ed, quarter included second represents period which XXXX to was in afternoon, release million, net of summarized Kite-Gilead in the and the earlier upfront for for the of was million are issued from compared cash of quarter collaboration or and oncology per increase results $XX.X XX. for that for Total $XXX good XXXX $XX.X million $X.XX the which quarter same $X.XX year. ended investments. We current loss second share

of expect our year, of along cost, end with this by this year completion other integration-related fourth proposed acquisition the quarter to cash. to transaction and With TxCell expected least the in the we million with $XXX

this enter potential three necessary ourself asset to cell As would Sandy expertise we we the TxCell greater of part has and of and of novo in strategy. invest is next ready developed more discussed our with EUR more earlier in the believe And therapeutic taken will Treg have in accelerating price believe the have the autoimmune assembling this cultivation far in be timeline. and call, purchase our value much capability as the plenty immunology than are and million to clinic year. of acquisition, manufacturing areas de far We future resources XX assets a longer pipeline.

to to also to the upgrade Sangamo’s have expect operations of invested smoothly. business our infrastructure progress year. into over heavily And We past TxCell integration

excited team build family with to immunology to the to to TxCell working welcome look the them are We Sangamo forward pipeline. our and

continues inherited to Our future and other opportunities value in pipeline. present investments creation for significant disease metabolic CNS

future in We’re areas invest our and continue to are to these expected operating to increase. planning expenses

Brisbane in In year. addition, is this to our occupy by well, going of end our expect and corporate for construction project headquarters the we

on well acquisition. remarks. that, to as cash quarterly call expense with TxCell the milestone financial over on the in transaction for believe We programs next execute closing to operating TxCell fund I’ll development is our now strong position date. And turn Sandy as we’re future to heavily the during this updated a rate our plan provide clinical on the a we by operating call. in guidance run earnings year closing Finally, Therefore, influenced expense

Sandy Macrae

you, us all Thank delighted team has that I’m for this half accomplished the second of excited to come and Kathy. the year. with the in summer

progress is SB-XXX in clinical may group steady our so development these our for will programs we excited making pipeline across apply preliminary for opportunities SB-XXX. soon be and our potential are available where clinical inform readouts new Our and for programs, technology. we review the data and now the of The will

TxCell therapeutic efficiency of finger continue are Group. him We standards capable and Technology genomic only our questions. over will two that our strategy hands and medicines. zinc now Operator? they confident next of set a acquisition precision, And and patients immunology Rebar in accelerates will generate turn your for cornerstone to platforms transcription to for not as Our factor for long-term shareholders proposed the the ZFN both Ed come. believe I’m but and and specificity We’ll years to to act years, value significant

Operator

Our with first of from the Raycroft line question Instructions] comes Maury Jefferies. [Operator

now open. is line Your

Sandy Macrae

Good afternoon, Maury.

Maury Raycroft

afternoon, line mean good if My on first and recent you what Hi, can light top data seen we’ve and contextualize data, congrats is the safety of question is an from perspective? the just, update. it in efficacy

Sandy Macrae

presenting So of as at conference the this full we the we end will of year. details be said,

so the about careful embargo have. that very conferences these we to And be have

there’s to much in recently say. not do on we So want more that field? had you we what can Ed, reflect this

Ed Conner

said reactions product immunogenicity impact mean, of the on gene itself Yes, that potentially the consequence all gene I And important but are because you’re think because in not and targeted of the trying just to for then as produce. therapy I deliver. very liver cells, immune we’ve along,

forward this data are where details a released. news the at medical we obviously, too been but that of conference, to premature that’s a hearing given about there’s some been So limited point more looking it’s recent at the

Sandy Macrae

with exposure. want can and others weeks that And to sure months data make we of of context more curtail patient we and

Ed Conner

right, That’s absolutely.

Maury Raycroft

it. And follow-up. maybe Got a

normal? primates, the it’s been the above those at highest any whether III what using point I and human doses seeing is XX%, provide should know, guess non-human what can that patient a that you’re that at patient the patient state – Or Cohort you since… a dose. perspective – Is are plateau on, pretty is to that corresponding that I potentially patient in the And data? recent If if were for remain at

Sandy Macrae

know to more. allows want you you as soon as it. can we give the know conference I We Maury, So, and

I’m I primate go to now it’s data. think not and sure useful at back the look

of an have able of companies. at response. hope consistency we’ll can to achieved the and within data Now And to the with to levels you we’re are looking as we other kind the in immune we be the the the the coming lack humans, for Those be share therapeutic, year. end things that more human you of the

Maury Raycroft

then, potentially rate? that we you And enhance it. And this one with on enrollment? one see KOLs? with Are it could based maybe quick could sharing And Got you data enrollment the have. more a ramp-up the just

Sandy Macrae

let answer one. Let’s Ed that

Ed Conner

has enrollment clear the year. on remained be track whole to So

pipeline this a continue We on forward to have continue to enroll we’re of we’ll patients so program. available confident and time very that on

Maury Raycroft

again. Okay, perfect. back queue. you, congrats hope Thank the in I’ll

Sandy Macrae

Thank you.

Operator

Baral question next Ritu Our comes with Cowen. from

Your line is now open.

Irina Margine

SB-XXX levels precisely one therapeutic do Irina Hi, taking plan for And general a targeting levels normal window for with pursue level guys. Ritu. XX%? to my is to wondering on I the then, if Thanks just are for intend you aim And This question. have the to program. on comment just you guys above could level that expression you decent you was the I more. of expression try or more

Sandy Macrae

that imagine can call. of in all a and range us all this you if us So there’s around of

a it that’s effective. immunological consistent We’ve has the and opinion, high, And should why that be almost everyone are there about that says, data and of know and therapeutic around over in very always quotes so we way you and And we’ll it’s there’s talked avoiding we been to response. range. is epidemiological XX% not you XX many that And years. be talked saying, that over our too

we the are more are important. characteristics those so think And that

Irina Margine

In potential over waning as any time? do buffering case, you follow-up. how just quick maybe about a effects think that And for

Sandy Macrae

this more we to by over some late-breaking It’s data. we’re very patients more will data this later want us of have clearly and and consistency that have the seen. we and But incorporate encouraged with data publication to allow our year important. And time the we collect

Irina Margine

ASH include you that Sure, data set? far? to question, so And thank the wondering, just And the able by patient an just patients much. from my these be treated then that’s additional fair. very to a or detail then will you maybe data you’ve expect five last introduce presentation Or two into do

Sandy Macrae

Yes.

So enroll month the patient Hence, is on may another we include are said as for this the patients, to been SMC be later but another and enrolling we expectation meet dosed. that patient we well. patient the potential that expect will has on there as or would call, more data patient we after that sixth

Irina Margine

Thank you very much, guys.

Operator

from Our Wang next with question comes Barclays. Gena

now line is Your open.

Sandy Macrae

Gena, how you? are

Xiaobin Gao

Gena. Hey, just this question no use. is different [Indiscernible] for of the there about actually is that Xiaobin Maybe dialing of a oral course use

responses? like did your you would you second and to just selected, question so any about that would of And also Just in the would what any escalate? future see the wondering dose be then be your wondering criteria expansion cytotoxics continue you dose

Sandy Macrae

patients safe us. important is their our really Understanding to treatment and

that? to you talk can Ed, So

Ed Conner

Yes.

course, come immune as back right, that’s two occasionally of those steroids. were because steroids or cytotoxic observed. as we reactively patients were steroids a and with of have no that the they so were have there repeat effects have treated but normal, dose tapering, stopped So liver have function – test, protocol await no we there We

across with cohorts to So all thus regards pleased with seen safety far treated. of we dose are we’ve the very what

Xiaobin Gao

to and dose Got it. potential And escalation? expansion regarding

Ed Conner

a the make Again, of and regarding answer after expansion. treated, a will variety determination and at factors Committee. dose Yes, have it our Does that Monitoring questions? patient will is that sixth your be look Safety discussion we we with

Xiaobin Gao

maybe program. Yes, guess quick yes. a question I for the II MPS just

the aspect. know interesting data. you want – the you Just expect patient that in your see understanding Do effect to from the therapeutics you update if well? CNS that got the any to have I impact any mouse potential, on about as

Sandy Macrae

them the adult is I’m of interesting, are you encouraging. and small. glad so it’s see is are that patients So showing a data treating, expect very These benefit the don’t anything. we mouse We CNS to agree that chance

adolescents European efficacy. in into both the by and of confirm form authorities population said, starting us the as once adult into some American we Ed authorities children, encouraged, that fact allowed the moving children; the safety can with to We’re so have move and the then

a a I and have the the that patients path the it’s understand find that’s So that package quickly as possible. to will the the get think they has put pleased that greatest with sign benefit, that Sangamo to are children authorities very most that helping together are with encouraging need the they us as from

Xiaobin Gao

Got it. you Thank so much.

Operator

Our next Fargo. Jim question Birchenough from Wells comes with

Your now open. line is

Sandy Macrae

afternoon. Good

Jim Birchenough

therapeutic levels thinking that Is IDS urinary we’re from? occur? something investigators GAG? from what still wondering feedbacks Hi, to on and as congrats reductions thing, guys. this in achieving starting your point what’s expert SB-XXX, I But Maybe helpful. with Good afternoon about seeing very one to look typical at from was Just past the got the progress. call dynamics week all was left you’ve shall we for is, that the lag

Sandy Macrae

of also of stopping ERT you GAG three period And what or a what’s then, is very sort you’re rapid probably known to when see about when over So most start is starting decline ERT. GAG two and weeks, as in ERT known four you a period that that weeks. is off decline of GAG it’s rise But on see slow you measurable when come patients about levels remain a to ERT. of patients in two as to start

And so I about. to be that about ERT, just we that talked as effects within able should discontinuing we talk timeframe measure

Jim Birchenough

the again cells indirectly? just there then IDA, any given levels IDS either of is directly or in on rapid your SB-XXX And study measure way to tissue by uptake

Sandy Macrae

essentially has I the overall past, which mean, was done the body. not of in glycosaminoglycans it the GAGs, been tissue measurement done the load So biopsies. in past it – but tissue taking with we’re the in are to correlate

in are. GAG measurements surrogate what useful helpful, for validated the And and levels really very your of cellular GAG tissue clinically and is so urine a

Jim Birchenough

competitive different to target I a Great. different back concern that to deemed than guess, as just from a way. guess advanced activity factory then, a trying to what maybe us the whether is Sangamo it just competitors some threshold Thanks. might more if perhaps, if achieve to you our be And are could be advancing questions about you thinking there maybe differently? I give what Or for sense SB-XXX, ask goal may other programs? And targeting? with is

Sandy Macrae

there whole really So and question the says can right the that. entirely days values. address from be Spark values it’s and what for? of Jim, slight learn we about. of we’ve are past they what immune data to And sure companies over and aiming of one the important data none And thinking field and have they few what what that that down the BioMarin another watched company is reactions move us watch really to having And one or said with it’s reactions. we’ve we the for, aiming And seen they’re

and so is greater we’re that sent. we XX% And looking clear very are And that Ed for than is I designed that also, patients. that the is for something is what about

in consistent, predictable. Ed? it’s it’s So it’s that not and generating a immune reactions

Ed Conner

but think about threshold I important. a to nature treating. And not reaching about not that And an and in about mean to It’s add so clinical that only population reaching a just you’re is threshold it’s data patient patient Yes. individual why, about it’s long-term patients. important of that’s every

So just that. I would add

Jim Birchenough

the question. taking thanks Well, for Great.

Operator

with Our of question comes Bank next Qian from America. Wang

now open. is Your line

Qian Wang

taking Thank Hi. for Hey, how my you question. are you?

Hem So I A. on may. a is first one couple, have I And if

wondering be And about So durability? all we decision? move you’re before could is to more quarter even you that, decision think the make you that or third Or I patient environment, fourth – maybe we just competitive of just six like criteria about, levels but about mentioned thinking mean, you sometime that the given hear want wondering second potential to quarter? do about going especially expansion fast? see when stabilizing of I or of dosing a want kind what

Sandy Macrae

quickly we there So a to as moving you clear always way as is will that sure be can be this but possible, is decided.

Ed Conner

Yes, it safety. It’s I on its just not the just we’re mean, things consistency, talking based is that levels, its overall about.

presenting seen over data decision we of it the presenting pleased occurred after the meeting about as we and thus year. We SMC. has what But talked share we are far very this the that at and will be look details with all or that at be meeting we that’s forward going ASH the end to obviously, we would will

Sandy Macrae

is a most its numbers sure to The provide that other we safety. with Sangamo’s what It’s patient making patients safe. commercial effective thing us not is around to competition important and entities,

Qian Wang

with the – criteria a use. but you great. others? you comparing your And did also, have signals, any I on see confirm your you the shown, have for you trying steroid to dose-response that to about threshold light whether seen cautiously for data and of any see significant higher be potential think you engaged dose? wondering just Okay, what’s any to prophylactic Phase don’t X? Can know follow-up I have also, partner guys And have you Spark conversation potential will in

Sandy Macrae

make Before got sure I I over Ed, we’ve would to questions. just your hand it

continuing some didn’t we are – we is dose-response them. we – were use of any for different steroids? can criteria any operations any the with about see with courses you because use us So planning And these what help of see to Did Did we all prophylaxis? in steroids? Ed anyway? using

Ed Conner

sure. Yes,

We’ve saw first or future that of cohorts. should nothing is reported that, of that that that guess study reduce a today, that after where we regards in patients we use drug. be perspective, at steroids. effect, indicate comment doing a to cohort second this With factor from I safety prophylaxis So dose-dependent on would receiving did replacement the use we seen

Qian Wang

Did for dose you steroid? response see

Ed Conner

No.

Qian Wang

example enzyme The response for not… with liver No.

Ed Conner

any We No. with liver see dose enzymes. didn’t response

Qian Wang

the… any Okay. engaged And have with you your conversation About partner?

Ed Conner

It’s We partner very talked terrific to our a alliance. frequently.

very, in and we’re fortunate them, very working frequently. We’re with to very be communication

Qian Wang

I have may one. If a last

MPS. As

conclusion So potentially of Can SSIEM ERT Have withdrawn I to you talking you removal see that? the about those think KOLs could the the according drawn data for kind potential are meeting? after – what informational right? of in be we considerations most mean, basically, I

Ed Conner

So we then, individual we’ll convenes, will withdrawal meet SMC patients. ERT of the with discuss six investigators them after with the that for about treated. And have been their patients now

Qian Wang

Okay. Thank you.

Ed Conner

you very Thank much.

Operator

from comes question with next JPMorgan. Our Eric Joseph

is line open. Your now

Eric Joseph

thanks little afternoon, Good joined for I a the bit taking questions. late.

– of of it at prior earlier. get clarification interim this point on resumption? the patients to if discontinued and of a clarification on year study. there II to So touched ERT SB-XX allows Is just infusion? What there’s post-withdrawal, overlap trying sort triggers have period. sort an what of just a withdrawal whether MPS of the in Champions But I’m sorry ERT time you Well,

Sandy Macrae

you study. I can description We of he’s help think with Ed, that. the looking for

Ed Conner

be into so all on ERT, have but on don’t patients are coming to the Yes, patients ERT study.

the As is exists. that only this expect, would therapy available you commercially

data Monitoring or that coming ERT six the be with then, will investigators after withdrawal into convene on again study it are until is time. the ERT individual discussion in. the after patients So the ERT And patients remain and Committee occur about will on and Safety data withdrawn discussed at the

Eric Joseph

that It’s sounds basis to – obviously sort or? part And good. would rescue I about guess, imagine, as Okay. of a we need how I patient-by-patient on therapeutic think of discussion then,

Ed Conner

a about you you to rescue you the about patients we’re to who have that you again, in four if would appropriate they do the are and level once generally, to question? therapy to feel who rise, patients Does on you after restart ERT, ERT will criteria But what call it XX to have two typically to not happens you GAG remove theorize answer start want urine your if what so starts fatigue. to market so. taking but if weeks, within was is are allow study Yes, days, that it, that’s

Eric Joseph

Thank you with pleasure.

Operator

Birchenough Instructions] [Operator have Fargo. from Jim a Wells We follow-up question

Your line is open. now

Jim Birchenough

quick Just question. two

therapy? exogenously I ERT first, IDS produced as enzyme your endogenously how from levels, So that’s you’re we by guess, enzyme supplied discern measuring do and between

Sandy Macrae

of modeling different Ed, for some drug? And and clinical on PK/PD to pharmacology as kind spent have going quite of this pass isn’t have of we I’m we spent the – you looking at but So time clinical it? Ed, the really would the –

Ed Conner

minutes ERT It question dose the an is, your literally answer but is to life administration Jim, to hour. half right is Single hours. of around to

these in levels week they So patients there been their detectable activity so clinical right ERT essentially we from contributed ERT, receive of activity what and we IDS the are IDS is study before because in does they no do the are that’s trough dosed have measure their weekly, levels before.

So from the this SB-XXX. allows us liver then to measure what from is production the

Jim Birchenough

Got it.

So of level. level measurement That’s will trough trough we helpful. seeing

Ed Conner

exactly. because Exactly, exactly yes, –

Jim Birchenough

then you one mentioned able medical the anything be antibodies surprising as if AAVX terms meeting whether bad won’t vector a question you or there of you a be in in I can’t that saw terms and you but good novelty neutralizing of say to the you And you other this was to understand Can for on at say AAVX presenting anything so. of screening just neutralizing going antibody?

Sandy Macrae

across all, there and And human the similar patients XX%. populations. We’ve MPS and of so them of all whole we’re antibodies human because and across I, and they’re patients screen the doing about about and it’s very MPS far that A, you looked B all XX% average the II if we

Ed Conner

on And yes, consistent what the literature gemological seen with that. published at we’ve is,

Jim Birchenough

Got it. Thanks for taking call. the

Ed Conner

any questions to I’d at remarks. in the to showing not back for queue I’m you. turn call time. like Thank further closing this management

Sandy Macrae

all Thank you forward encouraging very joining thank for soon. much day and and us, Sangamo we very you you again with look to talk for all it’s

Operator

great and conference. your Ladies and day. may gentlemen, program the disconnect. This participation for now a thank you in you concludes have Everyone, today’s

Sangamo Therapeutics (SGMO) 8 Aug 182018 Q2 Earnings call transcript