AGTC - Earnings call transcript Q2 2021 | Applied Genetic Technologies Corp

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Operator

Good morning, and welcome to the AGTC Financial Results Conference call for the Second Quarter of Fiscal Year 2021. Today's call is being recorded.

Before we get started, I would like to remind everyone that during this conference call, AGTC may make forward-looking statements, including statements about the company's financial results, finance, its future business strategies and operations and its product development and regulatory progress, including statements about the projected timing for its planned Vista and SkyLine clinical trials. The timing for reporting data in both of these trials and the potential of its ACHM clinical programs. Actual results could differ materially from those discussed in these forward-looking statements due to a number of important factors. the this of and COVID-XX annual the other described in extent process, pandemic report inherent of filed risks duration section with and and impact in results on uncertainty Form clinical recently other regulatory development the and AGTC's XX-K Including most Factors periodic filed subsequently Risk of SEC. the the this any call. obligation no after statements undertakes forward-looking to update date AGTC of the

Washer, the For Executive introductions I'd remarks, AGTC. over go Officer Chief and Sue Please like to of call to turn opening ahead.

Sue Washer

Good morning, Chief all Scientific for Shearman, With us. Officer; call joining Officer. Chief Financial today's are and thank our our you me on Sullivan, and Bill Mark

trends and and the second recent our With Mark review pleased After respect to have in During to CNGBX that both CNGAX of ongoing reported specific patients the allowing data clinical development quantitative today's for visual the in from patients could ongoing month, XXXX, provide I/II trial gene. sensitivity will potential important the two briefly out of are Bill presented financial additional is call, programs. recent remarks, support or a results visual data Last data with we we fiscal of XX more questions. qualitative will achromatopsia accomplishments. our in I'll in are take but XLRP in month change with indicated dose doses on of of first of announced late-stage durable improvements very the an clinical XLRP. a sensitivity to improvements retinitis in data from those acuity prepared will reported X-linked positive November we'll pigmentosa, the review with accomplishments, observed positive profile patient-reported endpoints groups. focus our moment, clinical review we evidence reported of takeaway the wide the to data favorable programs, there conclusions: over review a with detailed supports in and an outcomes collect First, visual the our mutations In safety to these XXXX. data XXXX, visual positive Mark year we to in provide data our to achromatopsia that both quarter then supportive in Phase in due key a update patients data provide achromatopsia. but Mark the will our that or as products lives on two your we with us sensitivity detail, range

on based from best-in-class product to patients with we safety. competitors, a may a product with with Additionally, comparison that have regards believe provide released significant candidate data that especially we to benefit publicly XLRP, our

of XLRP that this believe we approximately candidate programs for the the development week, I/II were our data to clinic. Skyline aggressive, but our a with Mark? extends clinical I/II the to successfully through build of for raised we programs. in two program our achromatopsia be both XXXX. intend And our that Mark our call I'll turn Phase into programs review on our additional trial each anticipate advancing We clinical million excited ongoing development and which trial. and have Combined our trials. Last our financial for and net both Skyline strengthened been product close on we momentum strategy proceeds. great public in we attainable offering, a momentum. have Vista by $XX additional across over to clinical to trials: will pipeline clinical runway year executing additional with trial, of the cash strategies capital readouts advancing data calendar deal position, and for progress towards We and sufficient in now XLRP of a current X the X Vista With trial, support The readouts started our Phase programs position, cash additional clinical XXXX our the

Mark Shearman

Sue. Thanks,

by in achromatopsia cone also and restored Let visual achromatopsia The appropriate clinical the Phase means the earlier after between wherein This to is not time be I/II me interfere our to further a our fully reviewing clinical for that affect activity. signaling eye generate signaling signs disorder, take is progress an Overall, function the the mutations to clinical the but demonstrating of cortex. trials XX-fold across continues with program. the congenital the additional well function Achromatopsia are gene ongoing support data only that dose photoreceptors in begin candidates product and brain developing biologic encouraging the development. in of themselves, range tolerated may reestablished. it retina are

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to time the an protocol, with an expertise that trial, of to maintain Phase verify and to will At discuss treatment data from the engagement. eye. be and of dosing with data the visual gene will we the In is the will superior additional X-month us to a amended FDA, we data. And Skyline VISTA our of made believe XLRP share FDA X intend XX-month have clinical therapy the that expertise current insight mobility patients expansion Under outcomes. in we groups we demonstrate cease we in intend also patient also We changes best-in-class sensitivity complete our I/II X, trial. which share contralateral correlation summary, enable to this to

further to manufacturing process an scale have or launch. and also development improved no process commercial We require support up

program of opportunities the We X-month and next I/II are Skyline further presenting to quarter excited X of XXXX quarter XLRP data months, from over in fourth our of X XX that we in to accelerate the group's trial from expect to including have XX data the XXXX. multiple XX-month and Phase second the the trial

second and a We also Bill of in quarter the plan year review of data to Skyline data report results. X-month Bill? fiscal quarter the turn call Vista I'll XXXX XXXX. for XX-month now third to over our financial

Bill Sullivan

our Thank you, Mark. Slide financial XX provides an results. overview of

million costs, second quarter For of net in net a the $X.X increase of of to year loss of million XXXX. a recorded fiscal primarily compared XXXX, net we loss $XX.X increase second a revenue increase million $X.X decrease decrease site due loss quarter $X.X to XXXX with million $XXX,XXX G&A and XLRP The year. million R&D Vista the spending external by result primarily trials. that $XXX,XXX reduction Skyline and recur increased the offset associated partially of clinical in spending offset site to in to our legal preparation our in achromatopsia in higher costs. other employee-related expenses did and increase primarily related was The An planned in our activities by employee-related trials. partially current decrease expenses not expenses. was million $X.X The primarily collaboration in $X.X the revenue million in in due was in $X.X fees, year noncash a expenses a revenue, was for The to increase a due G&A of R&D fiscal bionic increased a in manufacturing,

the from February XXXX. programs and with of million on currently along open and XXXX second cash, a net and programs with concludes you financing, our provide Now quarter guidance. clinical received We strong these equivalents today. of period. and ongoing Operator, question-and-answer investments be our remarks team's AGTC to research a year with will planned to I'll million. calendar for now allow generate fund discovery planned move from believe $XX.X approximately XXXX AGTC cash We total year to $XX financial of into and balance proceeds line the data sufficient sheet the our ended fiscal may funds, That

Operator

first Fargo. with the from line [Operator coming of Instructions] Our Jim is Birchenough Wells question

Jim Birchenough

doing same your at the other time. for biotech away XX Thanks call from companies

could said, this seems so and pretty one. of the do about usefulness clear you I changes vision, you So just it the how visually quantifying kind in capture of vision? their useful patients maybe that are turning And talk cones. number the -- experiencing And that changes with is achromatopsia, and on on that you're that guess then back

dynamic? when would you And of anecdote? expect to So And that improvements hear to would expect see have in photophobia? kind you that started you

Sue Washer

Well, you that Mark? good he of these changes in comments. thank to patients tie patient's us so it over quantify and in can visual morning, their are that reporting the are improvements in being definitely I'll for the how and Mark, and vision functional today. And joining in Jim, vision. turn is reflected achromatopsia sensitivity kind We seeing

Mark Shearman

Yes. thanks for Jim, the question.

in showed whose lenses is by that from an of XX increasing example, that examples the have We without experiencing this patients gave point which to patient-reported evidence your feedback see are investigators use the also the sensitivity. benefits things presentation, vision. indicated increase the we able there the was to their is as goggles. patient tenfold we average We've contact to patients in or So decibels, about now of light from a tinted represents of fulfilled outcomes one photophobia, -- are where perimetry

cortex. symptoms re-functioning the together interaction of signaling of to will retina rods and the of and of of of feel that as we've cones benefits those of perimetry said, the consequence we a that the feel achromatopsia And in of the is now the current both with the the a lot so the because photoreceptors visual cones as many emerge consequence a that are being reactivated, first we indicator the

trip younger the a importantly, time more higher and we very as of that collect and periods will more we're to And pediatric data emerge. of benefits continue doses hopeful patients these longer that so for

Jim Birchenough

the maybe age higher Could X. patients like for age? same earlier down Mark, talk And high visual the -- variability? at is think things there on you do that retest that you about trade of sensitivity just is do to looking expect about the how but testing to at kids it time, seems better, like younger you're And -- we young

Mark Shearman

to a a patients. younger of good pediatric data We the on lot have Again, question. Yes. don't date

and the are patients abilities aware being most guided they investigators We test. may seen of on how are the who perform by their deficits principal before of and have these their

think so if pediatric another eager we why we're want start the handle this be And reason -- trial performance [indiscernible] they enrollment a as on restart guided is young required. the patient, a a to their enroll we can't complete as in And we'll this I investigators the better tests. we I won't to on that. in these of patient -- mentioned, feel by get patients test And

Sue Washer

have we being And going the are interject that I'm modify manual to with. interacted study how just modified the also takers test to

And things and so did static age. younger for we ERG the perimetry modify to the like account the

very patients to younger we've the easy worked possible. as those for with the hard to complete ophthalmologists really as make So tests pediatric

Operator

Our next question comes from with Wainwright. the line of Joe H.C. Pantginis

Joe Pantginis

I I really think in are new say these here your of efficacy when if you and blazing here with endpoints. was a assessing don't patients. And wondering overstating I'm back So to second sort it step just regard territory could with we achromatopsia I

perimetry bit bring endpoints how discussions? Obviously, might a that the impact this towards more the clinical regulatory evolution now talk little looking you of forward? and static might So your you can to you're

Sue Washer

about the more the little MAIA I variability we with a people point less very initially, a to be to also think endpoint precise in discussions be our Mark, there's really we to sensitivity important out thought program MAIA has we why going microperimetry it might in And bit that it concentrated shown really that, had has And biggest been to I much have I'm measured. good that so because was you turn that had that in how and the be difference very able evolution up patients. microperimetry. achromatopsia. in and on us because potentially also we XLRP think how this bring over achromatopsia measure visual has Mark? explain turned not can he the And the been

Mark Shearman

Yes.

So point. just to make a further

hasn't details be the -- perimetry, So reason the the useful technical test. details, the patients the to taking of endpoint why is particular this proven that because of MAIA

example, that of perimeter poor the perimeter octopus the patients, they and target for perform smaller cannot the because So than MAIA detect a visual achromatopsia test. the acuity, uses don't their well, target so

us continue tests we we We've as So indicated I to number a think MRI light in We interrogate the the quantify response are also acuity, these test. feel treatment, brightness that far will it feedback better the presentation. clearly that of and we've the patient's improvements response in color what of may to seen whether sensitivity, they're -- the point that the to a the of be discomfort, that both is that evaluate introduced functional new and so manifestations experiencing. data and different the patients help

Joe Pantginis

you guess, it. your it I about but then step Obviously, Mike, is your how forward. be mentioned question overall next you landscape And processes your in and I that I been Mark, to here, competitive a my consider Got across second was was this prepared it processes And my step it. question, question specifics protocols to comments first guess pipeline? for any if could around improved if you could Got wondering back, improves -- manufacturing translated a there's and that? have place. a in putting share how guys my

Sue Washer

that, main first we've can to some focused answer and things: details. then the scalability But and One Yes. is take of about Mark talk two the productivity. I'll on

manufacturing process itself. the And so that's actual

[indiscernible] bioreactors, fully We are are which pretty overall. in in biologics standard volumetrics production

having think to contained any forward. scale needs a of in in over And factory adherent you can cost the has very out we going methodology. doses. making and so floor in we're larger small, is volumetrically scale So scale reactor, dose -- means which still cells then that XX-liter that suspension for of which XX X,XXX very kind any factory profiles can a up good that in ophthalmology floor, plastic quite up and with we across instead be And liters

part I reviewed of end and assays talk last to interaction much this with different FDA. robust the we that about on think biologics. come the little of important as they those in regulators they couple characterization assays, wanted same very of X of the submission and done except that have And effort of so product Secondly, all way to bringing our assays assays and comments Mark, reviewing have in maybe amount detail, expected time process gene be this therapy Phase II the to time over of in the need in years this we that is stages into to Mark? gene put And course, that I I'll therapy current into that's gene as examples it we, and out Phase the have the the underway. AAV thing process the would do and because would deal earlier to of and to of the I outline And can comparability a therapy he already rigor spent more give and measures. world the is our really really a at of great forward then that point leave and is into assays third process, of time to quantitative

Mark Shearman

Yes.

residuals of for of to improved the well then limit downstream come many assays. at which which the again, the detection a the the overall an the modified virus, higher of some change are I of of and packaging lines cell different improvements use analytical as from productivity and column process same for and proportion And that productivity couple they of as cases, captive used process processing have revised full cloning enriching is to below time, or aspects, and think limit residuals. that of systems reducing yields weak it's we've at The of now in full one the empty And the capsids. the are really improved gives virus quality. the the

safety to the with the the we that processing. then the helps quality to and higher So through and the improvement dosing in immune empty, improvements and threefold downstream to which -- in feel response full overall changes capsid higher

Operator

Luchini Markets. Our line next BMO the from question coming with of Capital is Matthew

Matthew Luchini

little this end that's Maybe first the get just, data really we is thinking going this with the endpoints? of new guess, -- X the the year X, Do set on next the context two the how the a should year data about going the Skyline coming how on then XLRP. expectations Can around that's achromatopsia, bit with to of set, quarter? help come you on that And now. data you just olds? critical I feel And of you And data particularly XX-month in think like generated, like, to you what yes, come at on your latest then perspective those have sort now for program. you've the need, go/no-go data that's to the from X just group

Sue Washer

the same XX-month morning. And we our what so to that expectations the time what X-month XX-month XLRP, is from period. rates Matt, that durability time based period X, us be first, you, on thank X the think from far this on data the and groups for responder So I the of we've seen joining would see

mobility a product visual with from or a from differs a changes will much we Whereas our the so be is of on data the opportunity easier durability improvements trouble that maze consistency understand. the we're sign. of brain that blocks we those mean? endpoint X-month to and when have What are of maze of in stumbling to Skyline from how the really perform because visual the consider positive that sensitivity one X-month would you Skyline instituting sensitivity kind conundrums the getting those in lot someone people believe wrapped to of kind of their see data test, see be And that is one to sensitivity corresponds it's around, we first what very expect that,

light added third-party contrast, the very obstacles, we levels. different developed task. tests are and the baseline maze of ORA, that provider, fine with a at a why high that's maze We're a multiple so sophisticated indication working with low And color who's very

that have products feel we've and a to sophisticated other support hopeful make used to sensitivity. people data, maze. we're and to people in we help it's that, very based So will we're on very real -- maze before that And using explain X-month maze visual these improvements that

the So we I/II, for and more main dose adding had the test that that's groups. also into of patients, maze one adding really randomizing expanding Phase purposes

low patients or won't know a whether dose. So they're in high

we hopefully, questions. without hope addresses I'm So And going to Mark? to about from what to to we and pediatric the is Mark then your the improvement what patients. sensitivity have that achromatopsia turn seen -- talk visual what XLRP learn then in so the it far

Mark Shearman

Yes.

pediatric examples and have both the full in we static perimetry. So patients field improving of adults

early individual various people area work of individual. agree age target field, visual function the takes direct cortex the on of aspects to that in effects the on the development this of to consequence on a indication both photoreceptors, cone brain. depending think the through but with from results communication all birth achromatopsia of photoreceptors I those of adulthood place as also And levels to about who the the XX, XX

patients more treating younger. I opportunity them younger think to where move to doing is we're have patients deficits is to what progression correct the there a that those so And very logical by

I think that's the debate do. thing to any So there's don't right that,

hopeful younger that will be very the already we're patients. greater so in patients in And seen so, benefits we've older doing the potentially those in that

Matthew Luchini

Okay.

of in point, program this further improved -- make that? is younger the take contingent that continued all make and benefit Is patients? upon to in -- progress just message on the explicit, to just I guess, So the

Mark Shearman

we and month we are see as we disclosed greater looks from and groups data. combination seen in they XX could patients some at paradigm progressing for what going months dose we've of as different changes, the the X pediatric adult Well, a AX plus patients. how through be younger to and XX, month BX the The there already, data we're was X will have both escalation that the even that improvements remaining in between XX indicated, dosing recently

collectively, entire programs. path a give the for optimal think better achromatopsia of I us that set will forward So sense the data

Operator

Cantor of with Kristen Our next Fitzgerald. from the is question line Kluska coming

Kristen Kluska

on point is the of brain guess I they X And that were here hoping And adolescent. And that as the have lives, The to you here, these the X what plasticity? individual you I'm at reported age patients during curious achromatopsia. become remind just was start of first does I one today, could lose this the patients their us I course about severe?

So wondering of the if this at rationale the all supports pediatric evaluating patients?

Sue Washer

population endpoint Kristen, your there's in between do follow-on really and patients a been expect questions is quantitative in pediatric improvement you achromatopsia of patient can seeing ever us And adult for Matthew's this Yes. what first questions distinguishing thank patients. the in of time today. and that This we an joining the kind magnitude.

studies, possible I in improve And so preclinical hopeful our results that very that one this of occurring been these think in models learning patient great patients. messages is here is always we're and for population. what to which sheep naturally saw about were were still the We such we've dog

patients. visual were And sensitivity, some the that of improvement older improved by the tenfold in that patients

possible from -- improvements these the collecting better by birth. to patients about we'll deficit it patients. learn these so question additional the I'll pediatric And is have Because and to this the And symptoms the Mark, pass in get even neuroplasticity. data,

I patients. we've to to data point date, in seen well some Mark. turning improvements think we've patients that out in the seen as as the older to it's But good really important it adolescent

Mark Shearman

good a question. it's Kristen, Yes.

And young short cases, fixed through teens. think to a no that where not in such time children development answer into then there's even to is go continues development, young faith perception adulthood. into it in from through mature some point you progresses infanthood as I to the

The know on can closest disease adult So is not we we but about some later that these we is know But to just is extent, and vision in where the completed. something corrected generating we're of to occurs different you bottom the the going and change at the Amblyopia, because facets to guidelines correct basically the childhood in where on cortex, the development, you're some based even lot that development point, it. in of of study, data if a affect evidence define when the allow trying that can I'm are be or disturbances in this patients doing of have to development quoting have adulthood. line that, context visual I this a think be achromatopsia how us information to called

to we're eggs very data. basically. basically I enroll the excited that's we're to remaining why And still So patients the think pediatric generate finding

Kristen Kluska

that I noticed for And efforts then corporate IND-enabling your highlighted in presentation candidates. partnering February you your some of

what to So we looking And yourself could this forward if versus Specifically, are establish beyond with extensive which running these programs? XLRP trials? of partnerships looking you're lastly, where partner? a additional the that, and year some the in And of preclinical is to pretty indications so here you take again, pipeline, be should ACHM this maybe something expect help might ahead us strategy to terms understand data? then you company's

Sue Washer

as able are, the to programs stakeholders achromatopsia a of might product very the to to and company, we the focused provide as can our as the value and programs on all term. near most you most get XLRP imagine, and near-term advanced in programs So that in the patients

in there about Given tremendous And can an patient our population in interested -- ophthalmology an that, our which many than a of such have everything there such with partnering are do And excited you we're extensive that we even can't preclinical as pipeline. because indications, our programs can't smaller we indications. are independently. think some larger we pipeline company, them value CNS reach very

data. in and important up over And ARVO the of We've meetings the industry, most the mentioned XXXX both of year, spring. would to so are that ASGCT, our we additional for expect the course coming have

expect fully. We to participate

XXXX. throughout And so more the about of course guidance information we these would and programs have

Operator

Zegbeh with coming the Partners. of question Our line Instructions] [Operator ROTH next Jallah from is Capital

Zegbeh Jallah

questions Just for a few me.

being two the ACHM first program. The about

AX see between ACHM. to one, difference you FDA the data accumulate on efficacy expect about endpoint second a then endpoints you if regarding as from but data? in feedback as new curious the with continue FDA expectations for program, I the after get the patients any you plan well endpoint the of BX efficacy Just from to about KOL's as thoughts And you and more to meet know that just being for ACHM? kind

Sue Washer

you any differences the Zegbeh. thoughts about and AX going collection what current joining between would And then Mark? On talk question BX I'm points to this are. FDA. we us and our interaction expect of with Well, and Mark today. Thank data to to for whether pass

Mark Shearman

Yes.

disease an don't Clearly, subunits BX be the -- the and We should the for sub the And of genes any or two channel. ion So why phenotype those different see of since associate AX those reason same. BX and are one units we general another. is don't the feel [indiscernible].

the this as we're we the just at difference that is progressed the number as a trial yet. small feel far So it's point, that we of a numbers BX haven't in seeing trial AX and because -- patients

fuel I measuring established think is visual used the is endpoint. reaching than that to to a as going before been endpoint primary we're for AX And or whether the technique just a any second plays wait full out BX static and So point, to readily your is how very conclusions other. has as see other perimetry and in It indications sensitivity. well-known or more used corrected

the being of feel they meaningful. familiarity perimeter that as with quite the we So that see a FDA and clinically changes expect octopus to would has lot the

great a us. think right I now, So starting point for that's

and a that. that's more forward a see see any we move And there's right with. good As we'll collecting endpoint as emerges do, adjustments feel what always now, quantifiable this patients. to continue if But more these in data, is as we're and to we point primary to

Zegbeh Jallah

things are the terms final pipeline with a and here efficacious? two sure me then to easier now the making accomplished gene of positive other now one are XLRP perspective well these the exactly results in make with as in here this ACHM process curious you rest you've seeing if of that programs, of you really the presented things read-through success with for terms just is therapies from any has as -- And

Sue Washer

read Yes, And programs a dry Stargardt and the seen the our process, relatable of construct, CNS the is targeting in those and give types question, programs. our two photoreceptors. do program indications, good MD ophthalmology certainly, which same using. And these our for programs, it's be CFH cells, directly those confidence screening manufacturing success we've we're other process the to process, serotype hope same all because In program programs, two in the through would agnostic and these which for are way Zegbeh. we're our us -- manufacturing in great really the product

through go capsids, exact screening is the same the process exactly be we're same cassette, for gene would productive those models the animal using about that and component. specificity. in and each biological characterized through go that We designing screening indications. we the nonhuman activity and highly so manufacturing way primates, we're promoters screen right sure the we And that We and for in in way monitor all the so to for

the well. be And cells the in ears of different are so the the is different eye. carried as otology kinds through said methodology cells can and really our and to kinds the same and in in really similar philosophy very, those of actions the very indications of And otology. And

And manufacturing are a so really and foundation. we solid are construct these we very confidence gaining design, vectors more more in that how and

Operator

we time. appears additional this questions have no It at

pass closing like any to the Sue to I'd comments. over floor back So Washer for

Sue Washer

and recent XLRP to believe strong everyone, the our the position you, through And joining achromatopsia thank today. us recent from put the proceeds public the ahead. Well, we added very advances and that in multiple our offering, in for all work in milestones months us trials a

quarter the challenges world us programs in And the an X our We're data and optimistic dose and We're focused of and expected Despite enrollment in achromatopsia from been trials, following groups to the about to achieve patients from the toward of patient to data year. Vista quarter our on in partners meaningful and the in the discussed we the this expect make as fourth growth more with around through clinic. group's trial pandemic. readouts all We we thankful that XXXX, adult XLRP put both Skyline our Mark And currently XX-month to of And the in today, a difference in today, intend months patients in us the us important year our enabling entire preclinical AGTC ongoing finally, of data a lives additional achromatopsia highest the the I've XXXX. fourth to second the from And the X-month continued I'm pediatric prioritized looking to the in towards continue months team, potential also calendar milestones completing in never the Skyline all the to as unprecedented during the trials progress XX XXXX. trials, mentioned. patients. especially on report in and clinical our X report and X the the forward global X trial quarter. of patients ongoing advancing path

Our realize and motivation to do months to sharing to you. programs. needed our recent the additional with ahead. the potential give of I achievements progress technologies work the Thank forward us look you in our

Operator

Ladies and and your conclude time. does disconnect gentlemen, for participation, you We this this lines your you thank teleconference. at today's may

Applied Genetic Technologies (AGTC) 11 Feb 212021 Q2 Earnings call transcript