AMLX - Earnings call transcript Q3 2024 | Amylyx Pharmaceuticals, Inc.

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Operator

Good morning. My name is Alan, and I will be your conference operator today. At this time, I would like to welcome everyone to the Amylyx Pharmaceuticals Third Quarter 2024 Earnings Conference Call. [Operator Instructions] Please be advised this call is being recorded at the company's request.

I would now like to turn the call over to Lindsey Allen, Head of Investor Relations and Communications. proceed. Please

Lindsey Allen

Cohen thank quarter joining me our XXXX call are and discuss today on Chief all our Officer. today third Klee, Jim our financial Josh Chief Medical you Officer; morning, Camille Frates, us for to the Co-CEOs; and Good Dr. Justin our Bedrosian, Financial and results. With

historical and are statements limited on and with everyone Private expressed include, regulatory we events our Act or make implied statements are our we I thereof statements this clinical materially that developments, our but plans any Before XXXX. These could call any the statements. current Actual begin, runway. those of forward-looking regarding the and and of to Reform not would are statements and that like safe statements results facts thereof are AMXXXXX, on based expectations differ forward-looking the not expected information impact harbor Litigation cash provisions that made to timing presented regarding avexitide, to, are AMXXXXX the remind and expectations respect beliefs, by to Securities or pursuant and from

on You required any place are to undue forward-looking statements any disclaims cautioned and statements, by Amylyx obligation reliance such not update to these unless law.

the Now to turn over I will Justin. call

Justin Klee

our conditions. in and high diseases and bring you to with productive pipeline in few our neurodegenerative advancing all to to mission been thank treatments morning, have late-stage fulfill Good the for needs last joining The endocrine new months us. unmet communities in potential effort

first breakthrough in track receptor of a options. We therapy XXXX, no Camille GLP-X discuss little to avexitide, in approved on the later. our FDA trial treatment which quarter PBH post-bariatric progress and both Phase initiate diseases continue our designation to or in We asset with with a will hypoglycemia lead orphan remain III drug antagonist

participants XX by with in treated primary In this pancreatic the also AMXXXXX II with interactions HELIOS and significantly mid-October, as with our XX measured for endocrine endpoint showed of of syndrome. PBH our excited positive at open-label People data top living Wolfram have the potential line we are in therapy. of from communities trial We improvement reported C-peptide response increased weeks. AMXXXXX the Phase function

or We similar secondary across all also saw overall improvements stabilization endpoints.

Additionally, data the collected improvement being sustained time. longer-term demonstrated over

data and With inform other FDA we a these meet III the positive program. in to stakeholders with to hand, Phase plan

we I to mid antisense And ORION interim Our of with is clinical continue Phase AMXXXXX Calpain-X from year. trial recruiting in and initiating of expect our next AMXXXXX, trial in ALS. forward people looking are analysis data targeting PSP our well, to we oligonucleotide

in position to are a strong runway into expect to our take continue and We us financial cash XXXX.

focused and on strategy clear of addressing preclinical built on data. mechanistic is rigorous Our measurable is orphan and pipeline well-defined rationales, biomarkers and conditions foundation

this data progress the strategy. in positive this quarter, Our we including reported supports Wolfram syndrome,

Our progressing our delivering remains on key focused milestones on ahead. team our pipeline and

I will Camille. over the to now call turn

Camille Bedrosian

progressive avexitide in Calpain-X supranuclear Justin. PSP each post-bariatric AMXXXXX, X including Thanks, in syndrome will PBH, in ALS. hypoglycemia AMXXXXX programs, hyperinsulinemic Wolfram palsy our our briefly I of review or ASO and in hypoglycemia, or and

First, update avexitide treatment an lead the of PBH. our I'll program, provide for on

QX on III to pivotal PBH initiate actively in program year. track We are are a in and planning Phase next of

to We initiation the the trial once and expect share the prior to finalized design of trial.

from data the in program We expect XXXX. top line

study million than PBH Level PBH of surgery GLP-X condition Most of the to of or trials hypoglycemia a X in and p-value from is a pancreatic studied effect beta and cases, clinical PBH. receptor glucose stabilizing in III, and in persistent we a bind to Phase PBH dose, progressive XX-milligram cells events showed on X.XXX. X% GLP-X these the a decade. and events in trial hypoglycemia has be islet Data been an a and Let notably, by X.XXXX mitigate in the Avexitide people some the estimated IIb action. affects is more is hypoglycemia. GLP-X decreasing that X X mechanism a to approximately in caused excessive XX% to the secretion Phase XX% significant of on in X thought events. last bariatric intend Level to on block reduction in reductions of levels. Avexitide further p-value reduction which with insulin trials me bit by touch XXX,XXX hypoglycemia debilitating an hypoglycemia demonstrated leading with designed highly to the in response,

XX Level are and events risk in requiring efficacy X include and as of third-party glucose occur clinically blood these program losing rescue. events. At deciliter. when outcome For milligrams hypoglycemia levels, context, X Level tremors, composite consciousness. reduction X dizziness Level of X below events Level per can primary Phase levels III the of drop will our symptoms defined The the be hypoglycemia

anticipated primary efficacy We forward initiating QX the expected III and data on year this agreed outcome. XXXX. top has FDA look in next line Phase in program to of

to Turning syndrome the Wolfram program. now

positive because as study, control, fatal data disease who otherwise with we participants across all burden or or all meaningful and disease multiple of overall and patient for may progression week-XX the treatment Justin for available they at XX XX-week and primary disease as perspective. showed trial are their in our line an acuity As no stabilization mentioned, the measures options. The measured AMXXXXX in the suggest by as treatment from data These improvements symptom in clinician XX, were pleased reached longer-term measures in C-peptide, top pancreatic of to visual with encouraging function Phase visit. improvement result measures participants results glycemic II approved including week present including data HELIOS week-XX progressive well additional endpoint

longer-term XX addition, showed time. for XX week participants In improvement data over week completed sustained assessments and who

in to inform and the in XXXX. stakeholders we update With Phase and other with these to expect XX-week hand, and longer-term meet the III to program data provide an plan FDA

Enrollment the going well. the in is study program. Now turning ORION-PSP to

described mid-XXXX. an in Phase through PSP top and IIb/III This have to and the analysis planned, analysis for track be of we with approximately we are interim of part with study our As share line living people first week-XX. XXX as of will an data on previously conduct data ORION unblinded

We this also in data Health application for the available we clinical pleased that analyze for plan inform to weeks. clearance share have our received on In to who go/no-go XX from will Canada on trial people ALS. with we participants are ALS, analysis This living beyond decision AMXXXXX proceeded a program.

We ALS the Phase in dose the ascending the to multiple begin trial in coming called safety and activity plan XX evaluate to months. plan placebo-controlled LUMINA approximately biological I Canada We in the We adults living ALS starting and at present with to plans Meeting levels, for with evaluate Annual Consortium XX.X last pleased Northeast the our study month. X milligrams. dose were

in observed toxicology NOAEL, of an clinical greater We application requested our firms. XXx for a dosing also FDA to lower drug at milligrams starting hold additional the FDA resulted proposed no XX.X the level, than observed investigational starting showed effect in on from U.S. Toxicology studies The the or information, by the to AMXXXXX. dose that submitted restricted an dose based and data which amount adverse is independent new a than milligrams margin XX.X of safety

believe if needed. address to be working U.S. can We trial the FDA are completed comments. of We the outside

to LUMINA in from expect XXXX. cohort early continue We data

our expected quarter and for pipeline. this progress remain key on our to by track encouraged milestones are our We achieve

now will Jim? call I the turn Jim. to over

James Frates

investments. Thanks, QX in Camille. July, million of closing out as expected. $XXX.X in we Financially, with Avexitide the and cash we turned QX acquisition After our ended

company to expect continue from AMXXXXX PSP program, our and manage data the the Importantly, of we to line readouts, we cash Avexitide as us clinical from take program readout into expected early top data interim namely from the through XXXX meaningful program. work Phase cohort readout III data the to runway our our

decrease of and Now the turning were trailing due results. the of now restructuring. for to related to administrative QX personnel-related our of sales were and discontinued financial same top for in a from a million expense for $XX.X following both million payroll were and for the The our and trial compared and expenses were line $XX as $XXX,XXX, result PHOENIX a data decline general $XX.X XXXX. Selling, decrease quarter to quarter costs revenues to in million product from development in same clinical a was commercial true-ups decline expenses cost the $XX.X sales Research $XXX,XXX rebates and professional RELYVRIO and compared primarily due services. primarily million our payroll for to ALBRIOZA. the Net in to the a and XXXX. period third personnel-related costs was and The period consulting decrease in and in decrease

of acquisition purchase our XXXX, We Our during spend is to restructuring with move price record to do to of million expectations, and expense we $XX.X Consistent of related and be $XX related stock-based excluding on R&D our prior million expense million to we compensation, SG&A, plan $XX as transaction now expect the complete, any per comprised range of we in QX, amount material forward. and expect the quarter. million combined not $XX.X costs. total into recorded going restructuring of Avexitide

a of believe the a believe position per third we're we share. ahead. solid Finally, the in in Overall, financial on million net quarter, critical in we deliver ability and to loss our recorded $XX.X $X.XX or milestones

remarks. call the turn now closing provide some to to Josh I'll over

Joshua Cohen

ahead. upcoming milestones In about pipeline are we programs, Thank you, excited Jim. closing, X path our and

to Phase FDA the III line we top is on FDA breakthrough orphan of with providing an and track and the hand, in of In advance III into therapy engage II with syndrome, Our program Wolfram and lead in results development asset, Phase look XXXX. XXXX. both our in to the avexitide, as update beginning plan designation inform HELIOS designation with PBH drug has first other quarter stakeholders in positive expectation we to Phase our

next we is The towards data ORION we clinical by build high in and conditions you and the continue neurodegenerative of -- need. year upon our in end I with trial expect analysis early trial XXXX. our year from data in of mid updated keeping of orphan next we working AMXXXXX critical and year. to as forward the well, ALS study look unmet are work expect or sharing to And early We Phase AMXXXXX the PSP we initiating and cohort diseases interim endocrine in recruiting

for Now the open up call would I questions. to like

Operator

from question Goldman Instructions] of first Corinne Johnson [Operator Your Sachs. comes

Unknown Analyst

Corinne. [indiscernible] is This on for

PBH existing III patients market for managed study? to are we So PBH I How initiating And that factors efficaciously Phase about a the are by gating have of therapy? then the the couple what should questions. size think

James Frates

Sure.

XX% tolerance XXX,XXX monitoring, mixed So abnormal testing comes ultimately people in or there glycemic in we show and estimate bariatric people suggests that the market, PBH. This between XX% roughly who will control. of glucose living meal surgery continuous with that of literature are a terms in have from

folks therapy. Some are of those managed be available by able to

persistent have decade to you and roughly to when continue in people PBH So for of that, an the about X% surgeries gets last PBH. million X% living account with you XXX,XXX of people X estimated

on factors, In terms of we're pass III to Camille the more detail. gating to I'll Phase I'll over track, and provide and any say

Camille Bedrosian

very Sure. for Yes. Thank you the question. much

is are stand and our XXXX. initiate -- to actively we There's sites up the going quarter initiation So with and doing very a We're first clinical lot of needed what's well. it's for working enthusiasm. clinical going team trial of

forward. looking we're So

Operator

from America. of question Charlie comes next Your of Bank Yang

Chen Yang

wanted just well. on I PBH to touch as

FDA with given XXXX? know design, a And design? have are previous agreement the potential factors, contemplating to the readout you of what what like on there other how terms are Just the Eiger, this we is trial earlier about in in with

Camille Bedrosian

the for much question. very you Thank Yes.

and having FDA. design. avexitide And on just with the read Level Level there milligrams and therapy the trial The refining with So I'll believe minutes, edges, and the of a as the we're X those to been use composite as have we prior endpoint we initiation. per just agency day the it, the a primary of will in with dose reminder, hypoglycemia. put we'll as the XX Eiger as interactions around summarize number details breakthrough provide a study agreement with PBH, X

Level X with in And notably meaningful significant as showed highly a reminder, PBH events, and hypoglycemia reductions studies statistically and in level. already dose and Level the conducted improvements X, and avexitide the XX-milligram both at the IIb Phase II

hypoglycemia about studying drugs. quite X population, Level evaluate pleased the is to aspect. that that guidances we're trials information are in diabetes the endpoints in So for that that and Level X Furthermore, antidiabetic and the hypoglycemia in used

and endpoint, composite So primary XX-milligram dose the endpoint. Level X hypoglycemia X is Level

it, described a study placebo-controlled XX-participant As Eiger study. parallel randomized for XX again, weeks, had arm they proposed

around it be good we'll refining said, I a as and -- So and that's benchmark. a -- bit from proceed there. be We'll

any at the -- XXXX. soon concluded end XXXX. We of I'm apologies, QX will possible of year be and and enrollment by the the expect beginning as in XXXX as XXXX rate in yes, have Yes, to sorry, data for study then

Operator

Michael DiFiore question comes next of Evercore. from Your

Michael DiFiore

from Two me.

avexitide. one First on is

about of curious antagonists to don't lead hyperglycemia you Just since happening. lots could undesirable the It think how on address sequelae. GLP-X kind of the risk this of

the So to be U.S. safety separately, high? future runs I seems about be could think the if milligram on that. why the still starting amenable see in with dose wanted deems And AMXXXXX, to deemed to U.S.? the kind how be you whether in commercialized be will FDA regard FDA the Just the asset you're on of dose thinking do FDA Canada fine, Like to that just trials thinking this just too to run high? XX.X too allowing the trial color any even and assuming in then

Camille Bedrosian

sure. Sure,

avexitide. about question So first, your

ability receptor case off or It's of GLP-X the is a reversing function a receptor. activate of the the PBH As turning avexitide of excess the to blocking antagonist, receptor. and the it's in not reminder, GLP-X

is it insulin the returning access to really glucose So control glycemic homeostasis.

to don't that the not So Furthermore, trials conducted toxicology that in PBH. we in nor in the hyperglycemia have prior work was clinical starting program observed of we setting. either -- clinical the saying I'm to anticipate used hypoglylycemia, sorry, hyperglycemia

we mechanism not do -- an given is that at. that we we the see issue. do something as of action pathophysiology looked the and So And PBH, of

Justin Klee

things, would peptides, been those too, The been well. but side I of and And principal quite well reactions, well sorts just often with in see it's ones side are injectable you of as tolerated terms site profile effects generally quite effects it's that injection generally, add, tolerated.

Camille Bedrosian

begin Yes. And I'll XXX, comments. may then regard have additional with Josh want and to to

fact more given that FDA, data their XX.X observed that NOAEL independent some the in the So the interactions toxicology and XXx program. milligrams we address nonclinical of FDA with is will milligrams understand who to questions XX.X reviewed have the determine the thinking, and toxicologists the believe their that below the

profile With has regard So confident we're certainly, whether green will and we XXX we're speed. yes, the light that of to very can Canada U.S., with confident in which able proceed, the go do be ultimately, to the overall us Health going to. safety are all to in we given we

Justin Klee

the with Mike, probably similar several there's often U.S. you've as kind level therapeutics, a for And of add, first Yes. we're of encountering just to I'd RNA outside think move very reasons been dose seen do well, I of kind trials as here.

Operator

from Graig Securities. Your next of question comes Mizuho Suvannavejh

Graig Suvannavejh

want on go to to III continued back the for proposed avexitide Congrats I Phase did design the progress. trial in PBH.

primary team, of I are with think you that endpoint composite And get proposing Camille and a that and to include the want just X they thoughts previously clarification had around proposed the go I X. Level is you Level as X. just you've chosen focusing that. I Level around Level in concurrence maybe why and to X, PBH believe Eiger were to on a

trial whether trying including what sense success? increased just of on a giving confidence get maybe Level around does that hypoglycemia, So to X

Camille Bedrosian

Yes, sure.

that Phase CGM X limit to a events of the wanted of the might the Eiger that events look course, hypoglycemia glucose hypoglycemia specifically study a Level to in one II rescue well, The clinically is as world opportunity certain a stick starting to precipitously and diabetes Level endpoint the be see with with the can just dire a even X, blood Level gives are actually, The just X capture hypoglycemia glucose before X drugs a is then in there the for well an that unable us that antidiabetic to the from recall level X finger to Level drop, and at goes meaningful. occurring on IIb are those Level level, And reduction X below realizing. based go composite X is events. to and themselves Level don't composite Level CGM and by all blood Level setting. I X and clinical And So one and alarms. is Level a well-established consequences. so X. as individual problems The from if where And the knows if

Justin Klee

were And both and on add Level Level to strong II on Phase X. seen X effects in the top X, only IIb, Phase comment the other the and

those, we So the drug one. against of promising to distinct both not to be either see

Graig Suvannavejh

PSP PSP, with with And III portion? decision the go/no-go you're would may with apologize the past, to remind moving if with in your respect could then a to you forward asked Great. the us interim maybe follow-up have just trial regards what I this in my I be to just be readout. looking and and considered comfortable what to for enough feel Phase good but

Camille Bedrosian

Sure. Thank Graig. you,

looking of PSP is the clear baseline baseline. there in established a high data -- PSP. at recapitulated shows PSPRS, endpoint a in for The trials function we'll scale number clinical demonstrate are So in function change an in AMXXXXX at strong And activity that data that are and progression previous interim there decline and from is rating to PSP. the be from level, in endpoint, that a placebo very is over primary of

against So have we compare. to benchmark which a will

will we And unblinded be doing look.

also comparing So information looking and will XX as be weeks we at also beyond placebo. biomarker We'll against data well. out be

So potential they PSP. based make data able on AMXXXXX to those and decision up, line be a regarding in we'll the of all how

Justin Klee

our as proteomics we that total we tau this earlier CSF, And very phospho-tau well. year and strong were by that study, in both with just both as of about you reminding well, prior was with I'll everyone rationale -- this in that the study too Graig, the disease. for And AMXXXXX based XXX know did, measured PSP in well that a but Alzheimer's know some ELISA work trial off I published participants in and there XXX reductions of trial as

Operator

Marc Leerink Your next question of comes Partners. Goodman from

Basma Radwan Ibrahim

we ORION study. the Basma Marc. about Can on question have is for This another

total and be tau. you're going said to the measuring You the p-tau

data? CSF-based only you have look to decision also are to your going we about biomarker Or for at be PET is avexitide. it in order biomarker? the a follow-up tau the planning Also, to make So

Camille Bedrosian

Great.

study, study be the that compared was to CSF. baseline. PSP a the signal And Justin disease as both recently primarily the Alzheimer's in very tau strong phospho-tau dramatic we for we'll CSF in saw just published in and So indicated, looking at in reductions the

Basma Radwan Ibrahim

Great.

include you Or arm of III only with it Phase against placebo? is are an to the planning care? standard avexitide, Regarding active

Camille Bedrosian

Yes.

nutrition the PBH really, approved meals small hours, the can high quite What people of cetera, for with that's therapies and sugar, restricting people of carbs, throughout a type eating although couple management draconian eaten, every difficult complex very, used So strive medical follow, PBH. a no food no as to for do. are which to protein, et of there what very diet, a is is they is -- actually therapy, certainly that's be

these are Other or that that the used, who've are And far people tried no drugs approaches are avexitide we so and for with those effect. very which been medications other little but studied success. little, has studying with

So and medical people be therapy, it their placebo nutrition will continue will versus avexitide.

Operator

from comes question Ghosh of next Ananda H.C. Your Wainwright.

Ananda Ghosh

I the That events? to PK? fast respect the about the arexitide. Can one the with based dose how hypoglycemic talk just you for pharmacokinetic is them on avexitide arexitide occur how events post briefly their on XX-milligram and question of those have counters profile symptoms fast

Camille Bedrosian

Yes.

about also range in It just an pharmacokinetically, pharmacodynamically avexitide XX therapeutic of reaches or hours I short post that. day you're and for about a or at hour complete dosing. remains about So range guess therapeutic

of coverage the get So over with day the we dose. course do mg the XX

studies do some single-dose modeling, glucose the onset been statistical PK/PD have In the of endogenous makes again, of you're onset I on with earliest fact, suggest were instantaneously. the pharmacodynamically, is drug pretty And the with of significance happens in in terms the studies crossover quick. met which that and is of a dose, pretty of it where competing effect of and this which And and insulin context that clear essentially sense GLP-X, receptor the single think also fast. there suggesting

Operator

Your Beatty Baird. of next Joel question comes from

Joel Beatty

think say, of success business which programs, kind the remaining probability as your the program, lead How a avexitide, do lower development? other potential balancing as among, you of may cash have potentially well about for

James Frates

profile, clear, at a the Well, as since financial it's made Jim science. a the Thanks as question. getting term. we've Frates. make as its for late made potentially acquired avexitide, in was patients we about table little Joel, hopefully all that impactful lonely in view most clinical the seen, it the it opportunity I talking clear we've that for we've data stage since asset. the think safety near I market, it the here and our clearly we valuable Yes. the of lead -- The development,

That the programs X said, important important already major along we're far And our development scientific of to where done. quite are each have we're all those very validation of in other work close milestones.

us current in belts that. avexitide, But have we've with that places, we programs. the day. making do our plans, tighten in gets data III think data on we'll other if our on sure So work with through as the of cash importantly. be Phase track each we're we seen And we I I runway every that can something our outlined, think on we've it's most to But

Operator

no time. are ahead. Please questions back Klee. call There to the I'll at further turn go this Mr.

Justin Klee

you third today's great to discuss XXXX Thank quarter call joining results. Have us for on financial a day. our

Operator

conference participation, and disconnect. call. this today's Ladies concludes may Thank and you you for gentlemen, your now

Amylyx Pharmaceuticals (AMLX) 7 Nov 242024 Q3 Earnings call transcript