CATB - Earnings call transcript Q2 2019 | Astria Therapeutics, Inc.

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Operator

Good day ladies and gentlemen and welcome to the Q2 2019 Catabasis Pharmaceuticals Inc. Earnings conference call. At this time, all participants are in a listen-only mode. Later we will conduct a question and answer session and instructions will be given at that time. If anyone should require operator assistance during the conference, please press star then zero on your telephone keypad.

to Corporate call Affairs. like Matthews, to now President, Vice would turn over the Andrew I Ma’am, may you begin.

Andrea Matthews

update second Thank Officer; Chief you me Joanne we President corporate Donovan, where Vice Pharmaceuticals today and conference review Sydney. Welcome Chief Finance. Officer;’ of to Noah will today’s are Jill Chief provide Clauser, Executive results. Medical XXXX Scientific financial quarter Catabasis Milne, call and With our Officer; a Nichols, Andrew

update release today’s this QX morning available We and issued corporate we on our financial our reference on a call which results, press XXXX summarizing is our will website. and

We the during Events slides Investors today’s within section Presentations are in the available website. are also and our part call using of that

most mentioned related we be federal results expectations statements by a I result materially would like securities of our to to that statements forward-looking from Actual as those note current as Slide making variety may of will our those required statements on that and statements under undertakes based a laws. call, is law. during on business considered future these differ morning be X, as today may available indicated the and as by today’s uncertainties, quarterly report which obligation including filed no discussed publicly and with SEC in Form represent this forward-looking website. to except our statements our risks Such on also we any on judgment and Catabasis XX-Q, of recent update

with Noah Phase trial. share Jill, PolarisDMD Joanne will with up. provide our wrap Jill open preclinical will Andy the two will clinical financial update. that, on follow pass With GalaxyDMD then over Edsalonexent, things data, III recent extension our provide an let me the update. and the corporate will and call trials will who trial, label the update to ongoing

Jill?

Jill Milne

on in call. Duchenne the our a for Thank will joining Edsalonexent you as well as believe progress lead Duchenne. Today muscular on foundational events. our with thank I’ll X, morning today’s potential everyone you those for update affected Good us with which program, recent Andrea. be therapy corporate Slide an all we Edsalonexent we to and for dystrophy, provide start by has

monotherapy, has to key loss of NF-kappaB, Duchenne for of skeletal designed all leads to and dystrophin Edsalonexent driver co-administered patients therapies. and throughout We in is of as dystrophic-targeted disease progression. potentially and was a believe activation the cardiac mutation a broad benefit applicability which as chronic benefits. with to potential regardless for their NF-kappaB The lifetime potential well muscle of as Edsalonexent the inhibit

preclinical Based potential the that NF-kappaB, improve share recent has and on data, also believe the mechanism inhibiting to of bone our of the data supporting to Edsalonexent and preclinical clinical Andy potential cardiac and Edsalonexent preserve diaphragm as today function, muscle, as bone health. health. skeletal we will well action

Duchenne, including The broad research of treatments community and treatments also their of that aspect life. muscle blinded shared cardiac caregivers in quality reinforced benefits additional observation physicians, from key improve durable hope research go members importance that provide of benefits skeletal effects. positively and prioritize qualitative recently beyond impact pulmonary and and can of the advocacy studies in A patient was that Duchenne the representatives. was Duchenne with important Participants will

be Duchenne, next Additionally, these can treatment Duchenne. few the boys multiple to due predict Edsalonexent to of areas Duchenne by by broad therapies, and potential dystrophin-targeted address We therapy of with of affected physicians impacted to the for that to that improve combination health receive needs believe will benefit co-administered majority its years. the all within

well tolerated has steroid date. to and a Importantly. Edsalonexent is very not been

receiving and screening scheduled in remaining remaining are expect visits. have drug opened trial final activities are progress screening being the The PolarisDMD sites in for progress countries All study patients the our last boys month Phase XX enrolment. completed with on excellent we clinical clinical September. next have the Many made X, thrilled scheduling trial. visits Turning with III across of and we to our Slide all will be are eight sites

mentioned CMC our half expect on current XXXX. registration commercial Based to Edsalonexent in study data application in from for support clinical are our line early XXXX. The and projections non-clinical intended ongoing with results as top the previously, efforts we we the second an to have of of along in and

trial milestone key Phase in is data our additional at completion PolarisDMD scientific look next enrollment to III that to and information III of announce conferences. We Our we trial. from the Phase Edsalonexent upcoming baseline plan forward sharing

In that strategy member brings board board share and a to we Hugh business to and commercial experience directors, addition of added to with phase strong our the board preparations. of commercialization development the enhancement next Cole. our new very and the extensive directors collective our for skills foundation clinical our of Hugh members. am company a of activities, we or are I pleased laying the complements

With three which commercial addition the new to year up of board become company. disease rare set to enabled evolution our to this we board for Hugh, added directors the us as success a of the prepare have has we

is are on strategy believe represents commercial trial underway significant opportunity for commercialization III to We patients Catabasis. and executing our development our with Edsalonexent a what bring to Phase for planning we

formulating its our introduction. market a full patients, well robust Edsalonexent payors commercialization physicians and by the potential rights global as for strategy prepare is insight and benefits of and priorities comprehensive the Edsalonexent key for characterize have research to to informed gathering. product have as been impact its We and for Among market

our provide will Next, Joanne? on update trials. our Joanne CMO ongoing an clinical

Joanne Donovan

this morning eight extremely enrollment, Edsalonexent we Duchenne. to an update Conference and Parent Project have share Annual Slide At the Thank trial Phase on XX you X. opened had good clinical summer, sites as trial Dystrophy shown PolarisDMD all our opportunity pleased across in the countries Muscular that for everyone. III on Jill, I’m

and limited interest excellent well progress the there with Ireland, Sweden, from being Canada rapidly enrolling III and Duchenne additional remaining and month next are the at are We is are that in September. accept the all remaining the to the at and are sites and trial Germany Australia. U.S., patients as trial space community. able The making The high are appreciative Israel are be the the in the for no of final staff remaining screening this patients The the for completed patients longer the sustained capacity scheduled to sites trial. dedicated very Phase site U.K., point as expected visits in

on a randomized boys. XXX to reminder is A trial the which enroll are quick we about X. of planning Slide design in placebo-controlled trial PolarisDMD double-blind

intervene III aged X to action to believe enrolling MoveDMD boys trial, the greater these for X the Phase of earlier their for mechanism trial is Xth birthday. As we up did Edsalonexent, benefit boys. in opportunity our We Phase II given the we for the that

two XX for Phase taken months. is The study III boys chosen milligrams to boys endpoints stand, forced regulatory because edasa efficacy that past two treatment boy it’s The XX North age meter kilogram one for in one, as primary placebo. the have Star Assessment and secondary by of in walk-run. the air who XXX compared for time North this was includes authorities. randomization change endpoint climb, supported Star of placebo. months The receives as the edasa, steroids endpoint per trial well as test, the Ambulatory of receive Key to time boys every not six such include with regardless mutation to the type primary score appropriate that is after function

the to Starting months, the XX to see on circle. and you III clinical experience days patient baseline with here’s left-hand After side, patients an study PolarisDMD, drug within three for can as study trial for here outline Slide PolarisDMD assessments Phase are Turning the performed. X, every yellow beginning trial. of are after screening occur the drug, visits randomized

We a the shown extension new called trial recently open launched GalaxyDMD, on right. label

the months, PolarisDMD right. placebo trial the complete GalaxyDMD and GalaxyDMD function as GalaxyDMD and of well have Our Phase long-term controlled a will on on to in visits as every bone primary siblings the Edsalonexent, can visit observed well six III with with edasa. in participate tolerability you their data, objective study, also to boys monitoring as streamlined we eligible schedule collect date assessments opportunity Based When has is with the as muscle see health. they XX-month are receive the to as excellent safety

importance is permitted therapies XX with recognize in additional the We in approved of forward. therefore landscape, co-administering the trial we EXONDYS the co-administration evolving intend going exon-skipping and Duchenne co-administration GalaxyDMD treatment of therapies allow to

transition the We from trial has the their the brothers in GalaxyDMD conclude The by extension. initiated were affected and of MoveDMD sons now provide families label to participating other the MoveDMD therefore wrapping sons already with MoveDMD eligible MoveDMD up, GalaxyDMD also The from month. their originally Duchenne, we brothers. trial trial enrolling for edasa to and expect by open eligible boys remaining XXXX also is boys to in this and the enrolled are urged trial

An important Jill bone health mentioned in area that Duchenne. is

risk As fractures height by Duchenne these and Neurology in in and large length; average symptomatic age chart. was XX. at of bone growing over that peers. strength and further boys boys their highlights, U.K. their in burden terms of as however, boys in risk affected recently grow increased growth Important are for are behind time in as health is Duchenne delay XX% both bone about are that in affected affected than boys they the by is falls Duchenne. not published that At the and shown bar Slide fracture impacted bone The researchers fractures. a health negatively bone studied higher deflazacort JAMA fracture The growth were Duchenne, data prednisone X probability or cohort their unaffected treatment, first evidence by there’s as well with of in found the bone birth,

This assessments treatment preserve sometimes fat fatal with loss need severe, research for This associated of fracture have that the negatively be be bone Duchenne PolarisDMD of and current Phase III highlights such and can consequences how health. Duchenne trial. emboli. our with bone The impacts for a research health included as complications can and health precipitating bone why can we in therapies ambulation, shows boys

our now Andy? over to Andy Nichols, the preservation data. the to encouraging CSO, share edasa I’ll preclinical bone pass recent call

Andrew Nichols

you Thank Joanne.

osteoblasts with strength. systems the bone and growth impact by effects repair by potential health. multiple We affected maintaining bone mediated continue and osteoclasts, bone organ biomechanical resorption by on dynamic formation a to of while Duchenne, including which learn Edsalonexent is Bone more mediated for allows appropriate about potential organ coordinated to

play Slide an inhibiting by can which NF-kappaB, the resorption Turning the osteoblasts both Edsalonexent and diagram causes and By a bone to regulating bone mechanisms activated loss. this of associated and which inflammation imbalance reduced X, to has bone formation NF-kappaB to inhibit of the strength a role leading osteoclasts, in loss growth. shows of bone function potential

well as activation seen with the birth. increased to Duchenne Inhibiting The fracture of potential shorter that in preserve after bones has contrast, that In NF-kappaB known to as discussed. Edsalonexent Duchenne. NF-kappaB health with stature Duchenne seen with health negative stronger consistent shortly is reviewed with boys Joanne in the bone risk with in boys longer Joanne and corticosteroids impact is as bone are Duchenne, the in

We mdx mouse model Edsalonexent investigating we our health bone also are trial preclinically regarding data bone of in III Duchenne. and the are generating health with Phase in

In we shorter mdx XX, The fragility DMD, June density of in the at maintenance in treatment and femur effects femurs in that to can the month six dose contrast, presented biomechanical a in clinically in resulted DMD. earlier thickness. cortical with bone of and data model Slide mouse a biomechanical treatment Prednisolone dose symposium Edsalonexent this this this and strength reduced on see in the while at muscle at On were In reduced preserved led length, bone density cortical relevant with relevant summer resulted the direction strength. femur. Prednisolone, modest, thickness, of and study clinically and impact

the in boys providing with grew can for and age Duchenne therapy opportunity burden that We not growth. per those bone vertical MoveDMD more could average of appropriate health by that by believe an year, height the two saw an for we of which affected that affected provide is boys than preserve reduce In trial, inches fractures on a Duchenne. Edsalonexent by

pass of to to Finance over share now will Vice I the Noah Clauser, our call financial our update. President Noah?

Noah Clauser

to our and Thanks quarter Andy summary. I provide press a XXXX so provide will brief details, the release morning everyone Turning second and our financials, XX-Q good

have and through XX, we equivalents As $XX.X investments. on to of that fund we current top term Phase short we and XXXX, the beyond operating Based of our line cash June XXXX. had plan, cash, capital million III expect sufficient results operations

million. activities In quarter of the used our $X.X cash second in XXXX, net operating was

QX in million XXXX. QX $X.X $X.X to was compared Our expense R&D XXXX in million

expense quarter was million XXXX. quarter to G&A second Our $X.X $X.X in of in XXXX of compared million second the the

million in in loss QX was compared million to $X.X operating XXXX. QX XXXX $X.X Our

million $X.X share per was loss net Our in $X.XX or QX.

$XX.X weighted the quarter, common had million. For we second of outstanding average shares

we in information SEC available financial the Additional is today. our earlier filed XX-Q, which with

the Jill. now will over call I pass back to

Jill Milne

provide in you is as dystrophin-targeted all Edsalonexent the diaphragm Noah. opportunity that believe what therapy. as Duchenne offers looking to believe health. skeletal mutation as function, therapies. their as this that benefit benefits We patients with has as co-administered potentially well new potential and Thank a know on regardless compelling monotherapy, lifetime We throughout potential for muscle, of Edsalonexent cardiac a community is the bone a and well We to

well excellent as progress the as clinical say recent teams trial. the to at label sites, the extension launch our III for our Catabasis of and the Phase Duchenne want GalaxyDMD the to trial team with open trial I thank community, you

III by of next which board foundation disease preparations the as phase commercialization our We in a enhancement and progress, of becomes Phase we are for excited commercial directors clinical rare company. lay Catabasis the the

open repeat instructions the With for and ask poll your that, up Sydney I’ll call Thank questions? you questions. the you. can Sydney, please for to

Operator

instructions] [Operator

with Our from Oppenheimer. Hartaj first question Singh comes

is open. line Your

Hartaj Singh

thank One for of the you a questions. I question. have health. bone just the around Great, couple is

from Jill you bone you that questions I just and III. Thank the about some think you. be little Joanne. Polaris data Can health talk that will on just mentioned I on had bit then you had also, collecting for and that, a Phase

Jill Milne

over address that. to to it Hartaj. Joanne I’ll hand thanks Great,

Joanne Donovan

to We are potential working steroids, and lateral at as to even able only the months starting out those as vertebral at fractures. the well. and are, at with them not common, to figure now at be to films the been have look We example so best we to these include can six density to look are what we using have know occur experts will DXA measurements of as bone those well for in but within future look look spine

Hartaj Singh

drug? part point but something I Joanne, around successful of or these are Joanne. the assuming know, of is Great the educating patients could become this you’re the does don’t this more filed, and be is that’s the this label if of and talking know physicians of around--you that part Polaris secondaries, when a benefits

Joanne Donovan

clinical goes goes effects this part that said of are the associated the important measuring whether the--actually, with that the standard of of growth, edasa of think care as be and label the profile of that well. Whether it overall in have I of to profile should also into side is yet understanding effect a seen. that publications terms edasa effects I an the side we’re and into so better is of the or absence the

Hartaj Singh

Fantastic.

therapy are much a the on X--actually, going I The Slide let centers Australia. other that back. step is question was North your seems why America me a X trials take How exon that’s from or It and open, still of the just XX, had you’ve sites that what’s X the you’ve got world be and all competition is group, the X other comfortably of gene on skipping, the age whether it’s American closed? that in in to primarily Australia or seems rest North to got similar to whereas

Jill Milne

think a That’s don’t I it’s great that. question.

United sites more there. Australia, than I getting just sites we’re those later. clearly little in countries. the States sites a completing hence focused the--certainly so the came and in think the we’ve Australia that last majority because We are of the patients it’s on initially in all then vast of U.S. have other the open and why we countries, do we U.S. the on seen I demand in would

it have there the robust meet terms a In we perhaps looking that demand to European there Europe additional was at and of now, in sites, could opened sites demand.

Hartaj Singh

visit screening the you Great, period that thank an between difference If just ask and versus the explain question you screening between Is baseline. Jill, actual the Then and baseline to what’s the XX-day was between baseline? I two? screening going the the visit, Jill. can the

Jill Milne

going sure. that. handle to let for Yes, I’m Joanne

Joanne Donovan

and consents, to appropriate other go boys have no It’s sign conditions, the they study, ensure basic so through in for assessments they to are XX genetic the come they that up have days, and can few baseline formal it could assessments be they days, assessments, the then be back, have XX to and which a do confirmation, come then the days, study. could for the up and

Hartaj Singh

conferences something Joanne. be medical Great talking there as preset--you you’re that in expectation future many a know, Is how or is present of that your data? would failures, about screening the baseline patients you’ll

Joanne Donovan

this screening in it’s Yes, but you so will been present group, rate low that, very failure tell we will age that been has the I good.

Hartaj Singh

is last you again expecting more therapies? of and bit color little for skipping any What study, the give on My percentage a thank questions. just agents? patients allowing already on question Fantastic. my you in those the what thoughts are seeing can that terms exon in you are you were Just there

Jill Milne

Yes, now included only that eligible that’s patient in we who right boys If and the Eteplirsen, do that’s that’s the certainly course, an in, of than would is approved XX% on no Eteplirsen, the that trial have skipping at the more overview because Eteplirsen now. so of as we’ll boys, give certainly we is exon so one for we present look the well. Once expect as now. baseline of have the about the right only that, boys as agent we population of of all so are we data,

to agents skipping establish the potential who trial exon are a do think label there’s is boys combination, for of on part exon stable on as based agents that’s our We the dystrophin enhance to approved, plan the safety those the efficacy with NF-kappaB Edsalonexent and dose development for edasa think of Edsalonexent. of Galaxy of Our protocol controlling the open co-dose of extension skipping intention are of allow the agents our to expression. to is other the to an explore--certainly mechanism to important those we amend

Hartaj Singh

are agents secondary of stratifications, the read-outs? one were skipping and there two. I right, for the trial primary Exon the the of believe

Jill Milne

That correct. is

on based use of stratify will We Eteplirsen.

Hartaj Singh

Great. my so Thank you for much questions.

Jill Milne

Thanks Hartaj.

Operator

Thank you.

comes next Citi. Joel the from with line Our of Beatty question

is open. line Your

Joel Beatty

for Great, questions. thanks my taking

this PPMD living and heard steroids side of being steroids, like one express a calling another families is concern the disease. with even their the summer, effects of some earlier first conference patients lot with with at The on I

you’re You’ve at, Edsalonexent provided Phase bone be a there seems on differentiation from steroids the great other differentiate of question measures could that overview health are that is of that steroids. to in and one safety? effect on about looking measures call the this with III My help side that keeping you’re track trial,

Jill Milne

Yes, let boys a thanks Joel. to of set in steroids. at for the we’re and I’ll lot what these good does a that, give compared will Edsalonexent question, parameters address looking safety young Joanne? absolutely us of to that Joanne compare the data

Joanne Donovan

seen, the in addition that will age is In bone year. in and at. weight MoveDMD of We which also weight, be we appropriately in gain trial know significant the What to BMI, in terms first fractures that the there density, actually and looking growth, health, so have something bone we and with weight. that grow boys of reflects is steroids, terms we’re looking at

main in side steroids behavioral that effect of profile captured would things We effects be are and are features. Cushingoid the well. the course also--some Those a Those side like are in you see of as age this group of things other

very the hear of a even look see they that I see are quality puberty. and grow, life. also seeing in the the at lot what around when that appropriately, of are in that That from something is of they of about boys What developing the you conference course grown are not that’s concerned necessarily that where terms they is and like a they time parents also older haven’t terms think effects you

understand that study, heart appropriate have from saw and study to tachycardia initial that experts abnormal so MoveDMD are that decrease rate and that’s We indicators that rate age rate, parameters the the EKG in understand and preclinical in are something look cardiac study one in levels also we Polaris or ambulatory to with we in heart in the variability, better to Duchenne. Duchenne, seen improved at the the measure monitoring cardiac we better sees the a elevated--from working relative heart in know to then

Joel Beatty

it Could development? the to Got potential it, trial discuss to Phase imagine expand sense III for as other is makes in you’ve any that Edsalonexent clinical use on well. into DMD you successful, if make that could that sense. that vectors I in there’s work the done diseases

Jill Milne

and is go done to potential the of for benefits establishing amount talked least enhance Yes, Clearly perhaps treatment we the Edsalonexent we’ve studying we’ve for concept express areas that of patients, that some to certainly of mechanisms that whom believe support something to low dystrophin obvious in dystrophy that’s preclinical to and could work previously of enhance that we’ve of levels the dystrophin, there. certain. expressed. the shared proof ability that at is population muscular presented, one Becker expression, at Becker gives begin that’s our Edsalonexent of play of about of dystrophin Edsalonexent for publicly What perhaps because is us done ability into the

preclinical with show those the the functional data dystrophin, supports in trial data well been to both MoveDMD clinic the able GRMD generated benefits also also--our the the dog, in have while we’ve potential. along that models mdx mouse as lack the as we’ve We

the what with area so hope Becker to and speaking we’ve ongoing into. is to internally been pilot So and the population, certainly that KOLs in looks in doing an that’s that’s study expand vet like best work what we

Of related Edsalonexent well have course may other dystrophies where there as potential. are muscular

Joel Beatty

you. thank Great,

Operator

to Milne closing back I like you. not turn the showing Jill for further at now Thank remarks. this questions I’m any to time. would call

Jill Milne

Sydney. our your joining for Catabasis. morning for all continued call you Thank and this of you Thank support

you we again on progress. PolarisDMD as other for of areas III keep speaking with our and Edsalonexent you share will execute soon. trial Phase We We to look updated forward

Andrea?

Andrea Matthews

page for Thank XX at Investor www.catabasis.com. today’s concludes you. our Relations replay That available be the on webcast days via A website will call.

Operator

you participating all in today’s gentlemen, does program This may have a thank great Ladies and conference. the Everyone you conclude day. disconnect. for and

Astria Therapeutics (ATXS) 8 Aug 192019 Q2 Earnings call transcript