PEPG - Earnings call transcript Q4 2023

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Operator

Good afternoon, and welcome to PepGen's Fourth Quarter and Full Year 2023 Earnings Call to discuss its financial results and recent corporate developments. [Operator Instructions] As a reminder, this call is being recorded today, Thursday, March 6 2024.

I would now like to turn the conference call over to Emiko Bryant, Chief of Staff of PepGen. Emiko, please go ahead.

Emiko Bryant

Thank you, operator. thank joining and call. everyone, afternoon, you today's for Good corporate for our quarter provided Earlier recent released results an the on and today, we XXXX financial fourth year and update developments. full XX-K filed press outlining financial at are results and website The available our with our on release both pepgen.com. SEC this the afternoon our

Executive of on Mellion, Officer. Senior Michelle the James Clinical Joining Chief Dr. and today me Donnelly, McArthur, PhD, President, Head Officer; are call President Development; Noel and Chief Vice Financial

making be in may recent not will to with SEC. statements regarding events its statements information, and we description development differ outlook reminder, a of these or uncertainties any research cause subject risks forward-looking our undertake financial as to materially new statements from in addition can update product most that on PepGen As results a the XX-K risks be its and does are future regulatory those to to actual changes our activities. found publicly forecasted. A obligation plans of and forward-looking result file expectations. in These

over now our the call to will I James? CEO, James turn McArthur.

James McArthur

unmet the you, need. clinical of or good Emiko, PepGen including everyone. meaningful our medical over peptide platform Enhanced cell-penetrating Thank EDO, diseases the derived XXXX, high of first neuromuscular afternoon, progress inpatient from programs with trials course Oligonucleotide, evaluating made PepGen and initiation Delivery X in

with of approach. disease Our is Muscular program, is treatment XX-skipping an for patients also Duchenne to exon clinical known lead PGN-EDOXX EDOXXX DMD amenable or Dystrophy, the as whose designed

a exon the people individuals mutation U.S. have with EU reminder, DMD XX% approach. or in As an a amenable XX-skipping to X,XXX an and estimated approximately

and or the affects for designed whom U.S. are treatments. EDODMX or in no type myotonic PGN-EDODMX XXX,XXX currently there that DMX more program, clinical estimate We disease-modifying dystrophy is the treatment people approved for Europe DMX. of than second Our X,

that improve for both potentially the be mission for programs DMX the our our believe have developing and and is DMD the potential transformative building outcomes PepGen, meaningfully company. patients. at vision long-term We and Here medicines to foundation of disease-modifying

these clinic possible. through living stock them a the team committed believe diseases puts cash a our commitment. projected this The as on offering quickly our to advancing in of our to Our strong cash into to is financial us to equivalent, recent sense existing important get offering, execute extend and as the for We XXXX. proceeds people with urgency team with position programs these net our cash this together runway with from

clinical Turning to the our programs. from latest updates highlights and

X enrollment CONNECTX-EDOXX, to cohort pleased II completed we first dose Phase trial. our in are announce evaluating X kg for in have patients clinical We the mg DMD per in

safety of review by the safety cohort of the we escalate emerging mg X. advancing and X process to Data assuming cohort Following profile, Board an dose of acceptable EDOXX. Monitoring per XX to The our place plan to cohort prior to second Safety kg at the per the will take data kg mg same

of we earlier MHRA U.K. EDOXX initiate an Phase in clearance to our the quarter We therapy. parallel, we study U.K. the dosing with in evaluating initiating week, kg to in patients CONNECTX-EDOXX, level per expect currently XX-skipping exon announced the X in II the this In amenable to people in mg DMD received cohort in X dose third XXXX. be the from of

open X including the for Following mg in to other kg to regulatory sites cohort, U.S., per the we trial expect authorizations. CONNECTX's CONNECTX preliminary data readout for geographies, subject

of nonclinical own modeling to in in per levels mg for available for patients kg We safety, anticipate data work skipping dystrophin reporting X the treatment protein. PepGen our clinical DMD produce EDOXX and with CONNECTX cohort and including exon expects externally internal data production. Based high mid-XXXX, assumptions, preliminary and upon using dystrophin

we level, as to analysis dose normal mg expect by X in measured At CONNECTX-EDOXX of levels following greater Western protein kg than doses patients. see the background levels repeat trial above of X% EDOXX DMD the in blot dystrophin X per of

cohort, of be EDOXX the to kg a greater if achieve were This For of achieved dystrophin would DMD levels therapy X%. our to XX than highest production per dystrophin exon-skipping date. mg dose by level

support this path Our combined from with modeling regulatory X% accelerated projections safety of authorities. we data approval, towards package suggests dystrophin designed assuming could that normal for protein. greater CONNECTX dose CONNECTX the level is achieve and The levels of expression than a possibility to potential alignment potentially and dystrophin

the Turning to by on our pleased EDODMX XXXX. FDA development DMX program. was October hold We that lifted clinical in the were

same authorities, the EDODMX across including U.S., trial advancing regulatory with X in clinical at starting discussions FREEDOM-DMX we dose our mg are I people Phase all living the DMX. countries, the Following level kg therapy with at for per

diseases to and needs. medical month, Just was granted conditions unmet by designation FDA. designed clear the facilitate review last serious and treat of with designation to EDODMX fast development track potential This therapies designed the is expedite

least ascending and single dose cohort we second correction X allows the with early mg half designation more frequent the track expect fast the to FREEDOM-DMX, patients. In XXXX, splicing in and safety, drug clinical XXXX. FDA, report for December functional measures and announced data, to PepGen patient Phase access the first outcome communication lead including potentially can dosed dose for in per Importantly, which from trial, I preliminary approval of and earlier at was kg

We the and per XX kg active that expect correction mg to both the pharmacologically believe could kg study XX per dose be mg myotonia FREEDOM-DMX dose splicing exhibit clinical mg correction. and in X evaluated dose meaningful per and kg

it expect FREEDOMX-DMX, trial DMX half of the patients of second than in normal rather degrading we well clinical that our our both to in designed differentiator XXXX. as pathogenic the DMPK CUG DMPK RNA. to An our as repeat than PGN-EDODMX is important dose target the is open to RNA addition placebo-controlled ascending In expansion with trial, selectively multiple FREEDOM-DMX pathogenic

at superior that believe potential has a to improved preclinical dose achieve lead As we a outcomes functional well-tolerated could based the levels, which events and selectivity EDODMX data, to result of patients. for splicing of this our on correction

focus to in lead candidates key progress. with CTA-enabling exon skip to addition to to previously primates, of program our for areas therapeutic the our our exon dose team and preclinical reported research X% target designed our evaluate XX details XXXX. preclinical is nonhuman in team DMD. skipping a providing disorders. and dystrophin of for our is In neurologic superior of clinical as neuromuscular PGN-EDOXX patients programs, transcript, in continues and and We approximately look in We commencing repeat IND studies more we forward advance studies

review provide call I trials Michelle the Dr. the the to Michelle? turn PepGen's Clinical an Head designs, of clinical of DMD trial and mentioned. just now in-depth will DMX Mellion, of in ongoing over Development, I to that

Michelle Mellion

approach. at biopsy patients must every week and a Each Thank occur living amenable XX. trials that an Phase XX-week of you, The and exon dosing the dose X young on will least with DMD James. clinical DMD, trial XX-skipping provide EDOXX to of a CONNECTX of years non-ambulatory II in clinical enrolling X XX Starting is muscle for be open-label, multiple-ascending DMD and both enroll men age ambulatory is with weeks. our pre-dose boys and will to weeks once

the the in with before We X cohort. the evaluate mg subjects to kg per will DSMB XX data mg dose dose X progressing from safety per kg cohort

multiple a the dose X placebo-controlled of dose cohort. biopsy for at cohorts. CONNECTX weeks a will months. safety muscle evaluation who proceed the dose data least The non-ambulatory years to old. Participants trial X the Phase boys therapy from baseline before amenable will We are multinational evaluate men prior based at at escalations provide then will is XX-skipping DSMB an both young exon administered XX-week next enroll upon EDXXX will further clinical review double-blind, week data ascending living every with ambulatory II dose will XX. be DMD and and and that we X to

in MHRA we to received the the our open U.K. are February, for to in following planning CONNECTX the the year authorization study and the and U.S. In regulatory clearance. later EU CTA this from initiate

EDODMX our to development Turning plan.

and I in first in of provide trial XX. FREEDOM-DMX DMX global in dose ascending escalation placebo, prior randomized biopsy a single kg to following dose enroll X:X of evaluating patients XXXX. have DMX patients our dosed infusion dose drug in up muscle versus will randomized, December patient with with at of by pleased taken are the XX review data placebo-controlled XX EDODMX safety a FREEDOM-DMX EDODMX We total from cohorts. The per biopsies of subjects double-blind, Phase and muscle will X, of single XX week again mg favor a XX at to baseline followed day

in the anticipate regulatory Phase discussions Our FREEDOM-DMX support multiple ascending a half II with XXXX with Phase regulatory II of clinical to We study is the multinational authorities. planned trial approvals that in inform the subject to EDODMX alignment designed of dose trial DMX, opening potential trial following our second will regulators.

our PepGen's of Financial Officer, now development clinical hand I Noel the review Noel? review plans, line Donnelly, will Chief that results. our financial With latest to to

Noel Donnelly

Michelle. afternoon's the this press SEC and you, results financial will and reflect results period. provided fourth in year the comments corresponding Thank quarter of full financial our details high-level More My are release in XXXX filing.

on December cash XX, held As XXXX, million PepGen XX, to and $XXX.X XXXX. compared of December equivalents million $XXX.X cash in

expected the on million. Based cash for equivalents, operating fund operations to of offering PepGen's the loss February million current into X, on Net XXXX successfully proceeds $XX.X $XX offering X.XX current mentioned of James previously, of of our proceeds was fourth PepGen cash shares for are As million. stock approximately an quarter XXXX, underwritten including common gross XXXX. plans, completed the and

Our year million. loss net $XX.X was full XXXX for the

expenses XXXX, development ended months and $XX.X for XX, December Research million. the X were

the $XX.X XXXX research attributable associated and December primarily in quarter manufacturing XXXX. preclinical, $X.X clinical development personnel-related ongoing and increase was the the and PGN-EDXXX XXXX administrative and development $XX.X company's for related and the months to the administrative trials. General was in research were General the PGN-EDODMX XXXX expenses of the expenses including administrative fourth expenses programs our for expenses in million. full to clinical were due X of million. in future quarter fourth ended million and XXXX, full costs costs and year For general an The to costs. The with year primarily increase of expenses compared advancement XX, to were increase and

February XX.X million had outstanding. of shares XXXX, as Finally, XX, PepGen approximately

call I James. to that, turn will with the back And

James McArthur

the dose ascending patients, successful inpatient the very multiple data I'm preliminary year This ongoing XXXX we DMD. is report middle to our forward from Noel. platform. To invoice provide quickly advance cohort important as DMX operational the the data execution announcements dose the data. XXXX will to from multiple to data trial course per data for year. look anticipated team's continuing first have you, our dystrophin the safety, X expect Thank and of the beyond. clinical programs in and for time clinical in upcoming in proprietary clinical over trials exon an of from DMD XX-skipping PepGen kg CONNECTX-EDOXX, of readouts to and ability our proud we multiple review making plan our with In We EDO production and XXXX, mg clinical

Phase correction and For anticipate mg FREEDOM-DMX patients dose the with half DMX, functional I kg the at outcome of reporting splicing the multinational of per least XXXX. EDODMX X we safety, second cohort from trial preliminary in in measures

With Operator? that, I will open the call questions. for

Operator

Instructions] [Operator

question Leerink Joseph comes first Partners. our And from Schwartz from

Jenny Leigh Gonzalez-Armenta

for is was talk and and to if you Joe. that of more to in how risk if specific more just implications blocking could This on might correction bit Jenny little mechanism read splicing for approach a I any through expect profile PGN-EDODMX? a about a result And safety? you the wondering

James McArthur

you, Jenny. Thank

as and Our mechanism, mouse toxic And patient able and DMPK can CUG of nuclei cell-based the models, similarly are is the employs we as we correct which PMO. very to demonstrate those foci approaches, as as correct our that as we're and targeting to both liberate to able well able that this seeking achievable splicing EDO and nonpathogenic well do levels, correct demonstrate models repeat dramatically peptide chemistry unlike cells were the well of patient in cells. as with This the to DMPK the been conjugation profound splicing technology the both to observable this well healthy volunteers, at the EDOXXX number in same myotonia importantly, we're dose in MBNLX in as response, in as which the reduce model. pathogenic we've degrade electrophysiologic other to also able the a is which

excellent primates technology, exon which the such, drive disease it our skipping. demonstrated following as As with targeting this coupled level to of and specifically toxic are humans believe too, well species a exon nonhuman that platform been XX-skipping we single highest robust in the coupling and mice dose that as EDO in we delivery has reduce

As such, will we believe improvement myotonia us say levels correction of we that demonstrate to strength. [indiscernible], from allow of achieve doses that that correction at splicing can we robust

it's we in knocking avoid [indiscernible] challenge and able that drives by we indiscriminately Although DMPK, a will fundamentals pathogenic disease toxicologic not efficiency the disease. down species to be RNA targeting by the demonstrated, been this is this that believe that of this is

Operator

question next comes from Paul our And Matteis Stifel. from

James Condulis

upcoming is This one on had the I relates to readout. for Paul. James as just it

to Just different do then recently dystrophin unadjusted readout of your dose? mg you're need to measures? looking an get for plus than next hoping there's you about X%, confidence And kg, some greater as dystrophin just can muscle we been kg, then see And discussions including per mg XX to to mentioned above for content. it X% relates much dystrophin. are one, to initial that have guess, at specifically, per X% fat these for dystrophin, two, adjusted dystrophin how X that for get we And above figure accounts the study at cuts I X%

James McArthur

appreciate I the Great. question.

So and reason you're is net study endogenous why dystrophin only reporting with of it can are a of It's this enters gain one on correct elevate The background. is individual by that X.X%. X.X% dystrophin an the important levels we above levels X.X% of dystrophin. really planning

over to per levels X% are kg background to still. And levels have looking gain go X so the we higher level, terms possibility but see dystrophin a of least see at dose at and we the mg in

It mg XX% kg, nonhuman us of approximately a apologize, EDOXX nonhuman monthly per that exon The is dystrophin. produces almost this XX I we've that a our dystrophin looked we And X could to this I exon increased when XX skipping confidence in at patients per give to in would has primates, why at X% skipping. studies, low XX% levels X% X% of produce was kg, that better But or misspoke. in reason doses level we X% single mg skipping. going observed dose following exon of primate

produce dystrophin levels levels from Obviously, sense see the higher will us potential going production. good of a low levels much higher levels of of dystrophin single-digit skipping exon higher we to exon gives and production. it have But that skipping

healthy primates. in of supported, mentioned call mouse is based have also both, model, the our on by done the we the in course, modeling This as I work in work nonhuman cells primary And work volunteers. in

Operator

of question our America. Tazeen from comes And from Bank next Ahmad

Tazeen Ahmad

Just two ones on quick DMD.

when For process X that? the this reasonable started XX expectation cohort data well? a would per you have mg to kg cohort, it on be XX do already based mg timeline you mg And the for the expect from for of expect as the identifying data, your year to think patients

James McArthur

Thank Tazeen. you,

study. the So study we we our for robust of lot clinical and see from a anticipate clinical enthusiasm investigators seeing of recruitment this CONNECTX

year. cohort As per we I've mentioned, mid- kg data this we've set of and mg of reporting already recruited X anticipate out that cohort X

update of an able will XX-mg recruitment, in as readout, fashion study. that a guidance we but the anticipate for terms well to on seeing timing give more based the kg you of have we in as we patients in we're data soon the investigators report for timely those as the as being of per enthusiasm As terms in

Operator

question comes Wedbush. next from our from Chico And Laura

Laura Chico

have I you. for X

a a about exon to little initial of preliminary with respect you're CONNECTX with and that you the the kinetics pace data. doses. bit if discussing could wondering spend James, seeing moment I'm So more skipping

expectation the the then in time. over or increase kind which that you. have you what's samples. the maximal I'm guess mistaken, was skipping of of curious patient effect quick I'm anticipate if seeing of a I plateauing, there can I nonhuman magnitude I the time? that occurred follow-up think in And a not over kind primates, a if for

James McArthur

I appreciate Laura. the question, Terrific.

it in data correct. skipping we've X, to increase dose as for dose When occurring exon dose incrementally to levels X terms observe X, seen X are by from dose appear still from go and very skipping X smaller reaching look our these dose well program, our that of the we You're smaller not that from at can a achieve. one some primate we X that more much and increase dose XX of dose to may greatest nonhuman our XX exon be and that level does that X high a both as is of and there we

we've exon As humans production such, than single-dose on humans. better done that based companies single-dose skipping prior modeling in likely to work skipping in we above do seeing and exon of exon where anticipate level the the X% of done we very skipping dystrophin on from the work other based compare extrapolation we've their our X% and

based modeling lastly, multiple nonhuman at work then we've and primate on both the very And single looking extensive done doses.

such, very as X we of we're reflective exon term. So robust doses. And following believe, long skipping levels this achieve, what be see to do we will expect to of able

Laura Chico

quick color functional on half think FREEDOMX, splicing would here. with update? to will hop you're more wondering second the the to what a And then be the I'm any I'll maybe be remind getting DMX us kind you meaningful that back the in magnitude we share could of respect of if could in respect you level around correction that one just With and kind and assessments of if looking for, of question extent what's you queue

James McArthur

then to be me it we've the first hand that done to speak FreedomX. speak at measures Mellion I'll looking outcome the over that Let Michelle work we'll preclinically to splicing and to correction

we've have myotonia of model the as higher that measurement mouse of of levels higher the [indiscernible] with correction, and engineered as and granted standpoint higher electrophysiology. see which splicing well dragging behind and animal this go by the the both can in we in terms model, the from in So correction higher preclinical model, we that levels observed of work and conducted, is

such, looking see at the approach of at As myotonia. we XX% beginning splicing we're XX% XX%, higher level that myotonia. approach can mouse as of And XX%, in robust splicing correction we're levels at XX% the we correction correction correction, of to of begin model, to levels

demonstrate able disease. of do we So the in correction the be than it's changes robust to better physiology terms of really percent this believe to and pathology important XX-plus see splicing to

we'll With Michelle speak to that in FreedomX. that outcome to over metrics I'll turn the to be looking at it

Michelle Mellion

muscles, assessment myotonia to X, FREEDOM well are in a are full is symptoms risk strength with of outcome such several functional most measures that be placing of these believe or I an the including study done different time So [indiscernible] assess related the walk of which test on and of now and this measures familiar and assessment endpoints at these our include the as other clinical -- outcome the XX-meter And vHoT, correction will DMX. during points assessment assessments, different as assessments. includes impact to as the

James McArthur

said. just I add And Michelle to to want what

the well that robust earlier, ascending as mentioned FREEDOMX was and single dose is our both well as splicing study work myotonia. expect of based both correction to we clinical dose, reasonably as following As we a work single clinical the expect of see others on can in a terms study as changes

mentioned good really of we go change seeing the year. get from we on multiple be molecule very this to this there. will the Some maintain data sense pathology looking doses will presenting start the of before EDODMX power of a meaningful the Michelle other disease. be a change we assessments we'll the that that and that at But

Operator

Instructions] [Operator

Ghosh comes question H.C. from Company. next from & Ananda Wainwright our And

Ananda Ghosh

program to EDOXX? data I DMD does with about and X recent your SRP-XXXX that respect then that James. platform follow-up I program. questions, you the data. one the on What concerning had And and question one tell the is have on DMX the

James McArthur

you, Ananda. I question. appreciate Thank the

dose was exon XX a per higher kg, the skipping mg well reversible kg transient single dose mg done of single grade XX of a tolerated to relatively to at we ability only dose. in at have per mediate cross-trial exon of of single compared levels we X kg. mgs following EDXXX, the XXXX So adverse events. sixfold in molecule comparison demonstrated XX following XXXX humans to the And there, able humans XX per were a XX EDOXX observe skipping

very So tolerated drug. well very,

anticipate, the with We exon momentum greater in we sixfold forward X% as be potential in what A both levels momentum to is studies skipping dystrophin we the could extrapolate contention dystrophin of produce what have than And XXXX. was X-month that patients B clinical doses. observed X-month than that supports producing and this we and the higher X following our levels potentially

in And EDOXXX data, the we'll mouse, in done and that so nonhuman both that the human remain confident, able to has primates we that nonhuman overall and see the modeling work both we've very in based to comparison incorporated X% the animal be extrapolation than with on patients. indeed, cross-trial modeling that work dystrophin primate and data clinical better

production achieve an drug X% dystrophin produced any exon be any will If by produced this and by highest we skipping exon-skipping the of be projected could even with exon-skipping dystrophin level approach, to drug.

be And but feel so X% we -- later be huge that to the done, than on patients. dystrophin greater we based believe work produce potential in a than we success be to would all to have we've the

Ananda Ghosh

with DMX there programs And has primary the a companies terms it. see endpoint program, of the debate kind of might DMX Got as what advance. the been to respect in

been discussions have there a around of the vHoT. so And lot

about agency's in to measurements? DMX there splicing take program? the that endpoint the is correlation between a your question the assay is, vHoT and view the on primary my what So correction is follow-up And

James McArthur

to I could and off, Michelle start Perhaps ask thoughts. to Mellion add then my I'll

with levels and beyond greater to from correction. splicing cannot we we be our ongoing can clinical believe important slicing is so that end are of points we regulators with the studies. mechanistic What is conversations these the an underpinning would that So EDODMX program and what approval say of speak and FREEDOMX

levels this would in see of myotonia levels with We of to of of levels we And the would patients, topologies myotonia as greater many correction. related seen models, greater animal we correction of disease. and correction slicing have, greater such, corrections of see in longer-term movement expect

But of what all ultimately approvable our that. will perhaps Michelle allow add endpoint. these to that would anticipation me let become go So to an it into is

Michelle Mellion

James. right, on believe ascending taken and grip the grip the our be placing that's Yes, these we'll our these And single FREEDOM-DMX the totality strength, strength. endpoints vHoT may endpoints of at impact in together, dose of later likely such walk endpoints looking vHoT full is of we about points these have therapies is test, [indiscernible] which on study, study doses as from that the and able future and as it will the I potentially endpoints functional several potential at selected learn time the potential that over on such impact for our intermediate that to correction population. multiple will a assess be and study impact to appreciate this the disease

Operator

showing McArthur And I to James for the I call to over now turn closing like am further would questions. back remarks. no

James McArthur

and Thank today's in operator, you, everybody, participating call. thank for you,

at you me you If follow-up We a all Have great soon look of do conferences. please you. items, questions any investor evening. have to or with connecting or reach upcoming forward out Thank to Noel.

Operator

for call. conference Thank participating. today's concludes This you

You disconnect. now may

PepGen (PEPG) 6 Mar 242023 Q4 Earnings call transcript