PepGen (PEPG) 23 Mar 232022 Q4 Earnings call transcript

Good afternoon and welcome to the PepGen Conference Call to discuss PepGen’s Fourth Quarter and Full Year 2022 Financial Results and Recent Corporate Developments. At this time, all participants are in a listen-only mode.

Following management’s prepared remarks, we will hold a question-and-answer session. [Operator Instructions] As a reminder, this call is being recorded today, Thursday, March 23, 2023.

I would now like to turn the conference call over to Noel Donnelly, Chief Financial Officer of PepGen. ahead. go please Noel,

an Good at Earlier and this financials today, and XX-K financial with and fourth Josh. Press filed corporate joining outlining was website release for you pepgen.com today’s afternoon. quarter our provide XXXX year call. we is full SEC on afternoon, you, recent thank developments. available everyone results for the update our Thank the released our our and

Chief James us Preclinical Jaya Executive today’s MD, and will and PhD, Joining Development. President Development; and of President Head Mellion, Officer; be PhD, Research McArthur, Vice on Michelle Executive Senior Goyal, Vice Clinical President call of and

involve management call, statements on current this significant are During uncertainties. based inherently company’s risks be These and making forward-looking statements. the forward-looking and will expectations

a events that the of to results cause materially statements could Our such those in Factors anticipated result be as the from a statements of uncertainties. actual could differ and and include timing forward-looking factors these company in described future different Factors titled results these risks in from publicly earlier SEC of expectations. Form on filed XX-K not section current statements report information, changes in the the PepGen forward-looking as its any new obligation with does update events, undertake to or today. our result its Risk

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

our Thank afternoon, Thank and quarter fourth joining year you, full good investor for and Noel you XXXX call. everyone.

forward. that to does on to like Before currently note regular quarterly PepGen a we would I moving begin, calls hold not basis intend

to our plan we or lead results potential updated to approval However, felt hold for a call. financial EDOXX, development clinical lead PGN-EDOXX could this our with the timing that program, accelerated

double-blind, trial skipping XXXX, X we in single-ascending healthy presented of dose In placebo-controlled, generated volunteers. Phase data in September of exon our EDOXX randomized,

placebo skipping to oligonucleotide XX were biceps. either muscle X, Following XX evaluation EDOXX. of biopsies the in of of and X, or exon-XX taken Needle mg/kg IV of administration a single concentration

into primary medics delivery generally or reported, at PepGen’s the study safety have profile doses we objective potential trial the EDO technology well-tolerated insights a enhanced of As platforms. previously the key of demonstrate peptide provided cell-penetrating oligonucleotide and

dose trial biopsies in Phase accumulation single delivery skip of our for XX levels accumulation a skip available oligonucleotide be Our on dose highest muscle Day Day publicly we skipping human muscular exon-skipping in XX of encouragingly, comparisons of levels, based with the exhibited XX levels the Duchenne from dose to potential EDOXX to of exon-XX and clinical DMD. following mg/kg dystrophy for transcripts observed dystrophin we exon-XX and and the data Perhaps from programs. protein the highest X other functional at patients of trials transcripts accumulation the from supportive of believe repeat most X, the in cross-trial XX which both planned

at single publicly dose As from a healthy an in than we mg/kg, X.X% single XX human to on exon a observed previously dose a volunteers, cross-trial higher data. results example, following levels up reported based available comparison of following mix in skipping sevenfold

earlier this in same meeting we of exon-skipping single method primates, we by human mg/kg of As dose week, assess wild X.X% XX non-human the exon male MDA used Phase trial. Dystrophy in Association, following Muscular at to observed the type or clinical skipping X ddPCR, reported a EDOXX our

to to for and clinically increased Duchenne signals combination primates non-human PGN-EDOXX primates, non-clinical data that therefore in patients. the doses non-human of monthly the skipping Phase of X believe XX-fold We to X levels the of drive exon XX.X%. impactful potential level human in Following skip transcripts the data in

X. platform of dystrophy treatment strong living paradigm activity for of drive indicative dystrophy the and Duchenne that believe EDO muscular our people shift with also Type EDOXX transformational We are to for potential these a results and potential myotonic

towards now EDODMX X EDODMX, in myotonic DMX of December we data non-clinical our XXXX. Type for PGN-EDODMX or announced program, dystrophy IND-enabling or Turning

bind MBLNX hairpin root designed brief the correction, the muscleblind a with is CUG events CUG lead cause misplacing like MBLNX the as X living repeat by of that the expansion, DMX. DMX. EDODMX to repeat observed Liberation or of been has loops sequestered splicing neurologic in transcripts and reminder, muscle regulator caused DMX Just multi-system liberate of transcript to downstream to of to the pathology patients DMPK

cells CUG at MBLNX accumulation meeting The the earlier EDODMX repeats. shown treatment the the with have nuclei MDA patient week, of in toxic X,XXX As this reported we cells. foci in in reduced over CUG-DMPK

EDODMX’s no showed impacts models, no a or transcripts toxicology Moreover, in EDODMX provide XX primates, repeat containing to level highest mouse dose CUG weeks. model other of these EDODMX short-term explored effects. through on DMX tolerability X profile. Additionally, than potential the in clinical non-human acute drive evidence the through studies thought well-tolerated In the in we level non-human levels of long-term dose XX I of a support which off-target was non-clinical observed mg/kg primates. activity were We to more that is believe favorable data in

while Dable the opportunities our achieved Directors shareholders. XXXX, Board meaningful with Laurie appointed In and public PepGen our value In $XXX.X just the Board went raising to XXXX, to for creating I milestones of of Habib on addition and broadening that NASDAQ Laurie our Chair. serving Stock million PepGen May In you. appreciation outlined as to Keating access Exchange, for capital,

future plans. management including President EDODMX over Vice course We Dr. also and of Clinical Mellion, Michelle team clinical members of EDOXX our the Development, Senior who walk the soon will Head senior of through XXXX and added key you

In value planning Phase our PepGen capitalize XXXX. encouraging on efficacy debut of safety assess development DMD. with doses clinical key young Based and inflection well multi-pronged summary, of X EDOXX on non-clinical in clinical boys to plan repeat trials the we PepGen as clinical volunteer men two living and for to positioned data, we initiate to points healthy and is believe is

pending X dose or Health CONNECTX-EDOXX with first in young XXXX, trial approval initiate open clinical the communicated trial DMD previously XX-week have label to ascending half of expect with in four we we the and boys Canada. by clinical multiple of men application MAD monthly doses As Phase living an

We believe from study second dystrophin EDOXX to the conducted X currently Canada. importantly, to feedback study a our in Canada, year, evaluate ascending of efficient to potential Phase regulators. to report EDOXX. is alignment multiple most in with double-blind, subject open the this initiate in X data MAD In patients. of And DMD the protein study this regulators conducting global trial parallel repeat dose in XXXX could XX-week doses CONNECTX-EDOXX based in similar in expect we expect X this parallel approval CONNECTX-EDOXX our EDOXX in to study plan trial half accelerated that a we label dose we this placebo-controlled towards the with potential XXXX. believe EDOXX pathway for on Phase to We from in support of pathway Phase trial that Canada approval

Lastly, data in and anticipate ascending of to and in and we FREEDOM-DMX single dose XXXX. the Canada half trial DMX mis-splicing Health first the reporting, correction in the pending XXXX, patients U.S initiate Canada. the clinical clinical we in U.S. FDA expect other of And safety, by a approval

Dr. summarize Preclinical our to I programs. Jaya will our now and PepGen’s detail phase Jaya? Development Goyal, further non-clinical President of and early non-clinical call our to supporting clinical Research the over to Executive Vice studies turn progress

James. you, Thank

dosing observed with model PGN-EDOXX X of non-human of skipping to our in PepGen mg/kg protein week were demonstrating every the of potential mentioned, mdx in dosed treat mice up our monthly production doses. and doses. in XX after four X.X% with on non-clinical X in presented four James Clinical every clinical dose. four study, data of monthly exon after and cover XX% bicep In per momentarily. we dose received in we to EDOXX DMD dosed the this repeat and XX-fold will to and exon primates XXB weeks after that mg/kg skipping dystrophin XX% at the primates single non-human Michelle mice Dallas, mouse to study mg/kg both species skipping MDA’s Scientific Conference observed tissues mdx mdx weeks, at of In up planning we encouraging doses, mice earlier a data, As a showed study after dystrophin from are XX increased XX non-clinical In Based dose in fourth EDOXX primate for this that & of of which level mdx the stacking NHPs in the accumulation we exon XX% non-human Texas. bicep,

continued to EDODMX. in repeat. a the pathogenic reduce RNA non-clinical This liberated support dose-dependent earlier we with observed Additionally, the nuclear Texas cells data patient MDA that EDODMX manner this and foci MBLNX meeting for of in toxic size announced DMX X,XXX further full development was from week provides in at

dose pleased were we from As towards non-human we preclinical exon-skipping repeat positive programs, our study. primate the data stage earlier deliver turn to PGN-EDOXX

of we healthy accumulated Phase EDOXX, level observed protein of in accumulation In XX.X% study myoblasts, study mean with upon and In of EDOXX exon-skipping traffic for XX.X% our human myoblast supports type also in data with treated mean upon what skipping showed exon level pipeline, with PGN-EDOXX. preclinical dose the a of repeated EDOXX in volunteer we with script our single both is exon-XX a have update vitro the while This dosing. results and repeated exon-skipping and administration. of with in human skipping. wild patients wild-type consistent level high seen previous from non-human a X we primate potentially exon Following our transcripts skip on from shared accumulation dystrophin the potential conjunction PGN-EDOXX the these

Michelle I presentation to our of will forward week Development MDA to our and to informs now over Dr. clinical Michelle? call clinical Head PepGen’s Vice the our and turn earlier this strategy. that Senior moving plan Mellion, summarize further Clinical at President detail

Jaya. you, Thank

levels Phase delivery mouse for said, well-tolerated The Day of in NHP EDOXX data for EDOXX highest week earlier of James at MDA The study in presented volunteers and Phase the at for a PepGen relevant and results the to the and healthy XX in treat that EDOXX, clinical generally Jaya and our healthy X conference. muscle the As resulted was transfer. support the single of this model X potential in development doses. data clinically exon accumulation support mdx exon-skipping potential DMD our oligonucleotide and shows demonstrating of in skips program non-clinical at dose

As for described by of accumulation Jaya supported study. skip our potential is non-clinical transfer this

study approximately healthy meaningful volunteers dose, in potential that increased monthly exon-skipping skipping in to suggesting increase possible to ddPCR scalability. also NHP Xx resulted single volunteer roughly mg/kg pain single X.X% in a our by XX the mg/kg that X.X% we in healthy dose dose Specifically, XXx exon-skipping, amount XX%, with but of not in while doses this After of NHP, only that a XX dosing a the dosing, of of monthly exon as a supporting skipping, showed X single showed exon-XX in achieve regimen.

anticipate trial second We initiated our in EDOXX protein the DMD. potential a first expected on Phase and initiate planning a young be assess Following EDOXX CONNECTX-EDOXX to and reporting boys in living expected men efficacy global in In be of accelerated with we to multiple are clinical healthy dose XXXX. in trial CONNECTX-EDOXX the the ascending of two XXXX half parallel, is trial volunteers, is doses successful dystrophin safety to that XX-week in trials randomized half X repeat of clinical to open in of XX-week Canada label support XXXX. to the approval. a placebo-controlled is of initiated monthly data

from program. EDOXX Based employs study, we clinically will believe able the EDO peptide our clinical informed EDOXX on EDODMX that for development relevant EDODMX, which Phase in of results EDOXX as study technology healthy same concentrations healthy be clinical volunteer muscle. achieve to X we volunteer our our Additionally,

X the in PepGen initiate enabling Canada XXXX. planning in U.S. correction transcript, a Phase to data safety, and are of to in clinical placebo-controlled data study misplacing single-ascending FREEDOM-DMX We and report patients dose

and now our will line Officer Donnelly, hand results. financial Financial Noel review quarter PepGen’s full year to the Chief XXXX I to fourth

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

or same equivalents on is cash expected to December operations of compared cash which $XXX.X position the into XXXX, for EDOXX net EDODMX. in period to dystrophin $XX.X DMD of compared the of million transcript XX, held PepGen data net lead of the XXXX. generation asset loss to loss includes strong was misplacing quarter As a and $X.X $XXX.X million million fourth and our of fund DMX or December downstream early PepGen’s XXXX, production correction asset XX, our million XXXX. PepGen’s XXXX lead the data cash in for

year loss net with loss $XX.X the full net XXXX. XXXX million Our full the for a for was of million year $XX.X compared

Research for $XX.X XXXX. to fourth were quarter and ended December for development expenses the the X million compared of XX, months XXXX $X.X million

compared million were The the to year in quarter year the expenses $XX.X XXXX, for $XX.X in XXXX million For programs fourth as XXXX to R&D attributable preclinical, to XXXX. the compared related R&D the quarter EDODMX clinical EDOXX ended of full increased XX, primarily increase trial increased and December expenses personnel was of with costs. associated cost manufacturing well fourth and our as

million compared fourth $X.X expenses quarter December December to for public million G&A General full operations. million expenses with associated primarily G&A the $XX.X for XX, $X.X for the ended for were XXXX. The months XX, in $X.X XXXX compared were increased ended expenses the XXXX and full of administrative increase to million XXXX. expenses year was supporting due the year to company X

shares December shares million with as outstanding stock PepGen’s XX, XX.X XXXX outstanding. million of Finally, were approximately diluted XX.X common fully shares of

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

Thank you, Noel.

progress with year Boston our into over moving platform headquarters. made advancing We last the have pipeline recent technology are our proud and we and the new

are on our in patient studies the to We two initiate expect continue in we EDODMX with to key clinical we to expected have with call eager referenced XXXX. in readouts the that and XXXX on capitalize data momentum EDOXX XXXX

CNS will on of XX. Bank Days, the be presenting Healthcare Stifel and upcoming America We at Conference April Conference, XX; Healthcare May conferences, Needham investor XX; March

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

comes with you. Our from Paul Thank [Operator first Instructions] Matteis question Stifel.

proceed. may You

this is James for on question. taking for Paul. Thanks Hi, our

to global half this DMD quick set initiate a of one Just regarding second year. in the study

to And the it is has yet? FDA been the like because pretty I talked space of FDA the clinical holds. conservative reason Just the you in this number wondering, ask have a seems with

label much. any before allowing only initiate So you just before it’s wondering given starting open point, the months U.S. the before? that and anything few – for be inform of of to if they kind have so will how on specifically asked kind a data study pivotal in Thanks

have question. of clinical had our you. Thank regulators informed design we that. conversations Yes, the by appreciate and I a has been with lot

The study deliver study label We study. and DMD people needs And are as studies is go a as appreciate dystrophin to toxicology And X-month completing balancing the studies of possible this end integrated we as in desire data we investors for the timing in both the will living with after be us Canada. study. will what second on part interaction to regulators to XXXX by the the associated global to of with both that data reason be this that drives as which do of benefit open into of global dosing And in study support that that year. the is the rapidly be year. for well the the

Thanks.

Thank you.

Our next Schwartz with comes question SVB from Securities. Joe

proceed. You may

and taking X cohorts studies XX about could the that the to you Beth how or the was of Thanks CONNECTX potential is dose for and assess in healthy question. up to on thinking just if mg/kg for volunteers? with this dose above Hi, you assessment planning the curious on Joe. highest to any you dose our color I are provide are

the for question. Thanks

levels the of Let twofold. will think multi-dose our that for dose mg/kg, me this exon-skipping study, level first well adverse a the The X emergent that deliver study. significant tolerated start highlighting we was with dose safe dose truly only believe in of reason events level off treatment Grade we and single by is in that XX

reported level delivered that single dose First of highest study in sevenfold ever XX than humans. was a single is exon-skipping at as the highlighted, dose higher mg/kg following previously the levels

in course, X.X% exon-skipping Jaya by single exon-skipping XX% non-human mg/kg exon saw doses, seeing same a that showing higher being assay. of we with of exon we dystrophin skip of ddPCR tremendous almost accumulation we same would as levels transcripts exon skip, anticipate levels dose four a primates, following But the on XX drug Second, are delivered the study of higher skip at well. higher produced transcript, the highlighted using

So to about XX the we potential is we go are mg/kg. to believe excited very what

those we regulators that in through with opportunity strategy. of we move patient go will and is as said, and being front conversations development safety have our that with above to our do we mind Now believe center an there clinical potentially all

Great. you. Thank

you. Thank

comes with question Tazeen of next from Our Bank America. Ahmad

You may proceed.

is on Hey, This Tazeen. for [indiscernible] guys.

What quick DMD how to gene patients the the enrollment endpoints DMD planning be have how therapy you are might U.S.? you a We affect CONNECTX. of is measure? on are had clarifying then it agency question approval and what is the maybe to going for accelerated And levels? potential approval the for about in – Is thinking about dystrophin how thinking

of first require patients. of patients. can to beneficial, of provide We is is be will second said And than many which the type of with I of part different question baseline Head truly almost I the our that others It a perhaps clearly, endpoint a the and part particularly with that take younger for Development. your question levels length and a the nature polypharmacy delivered therapy it over treatment beneficial gene dystrophin could of then in we therapies exon-skip bit an meaningful approval that the the is to potential a Duchenne muscular that of is dystrophy going your of turn think in So, on for that level in and Clinical a dystrophin microdystrophin. approach obviously of quite to multiple product full believe being deliver could

meaningful over is change here great on turn And that, a So it to for with Michelle. to opportunity that patients. we there deliver will think I

Yes.

as is course design terms controlled in we this looking of be well what that clinical clinical will of in dystrophin the production trial, of randomized safety. global So as trial at

to affect increased leave production of these improve assessments. along broad these these that we evaluation on with maybe information accelerated how we including array timing in us give of can the how also assessments we for of dystrophin an discussions is We will that does And an idea with have and clinical assessments approval. will a endpoints that be performance them and further regulators the is from important

you. Okay. Thank

Our [Operator you. question Laura Instructions] next with from Thank Chico comes Wedbush.

You may proceed.

Thanks good ask landscape. I guess the much one very to the perhaps around I different for Hi, taking a regulatory wanted question. afternoon.

emerge willingness a for on Thanks meeting was changing then And any So am there around wanted it? though an of soon to again you DMD do see dystrophin committee that landscape accept just there be you going what as in could terms that I the I wondering FDA’s timing specifically productions? the are very up is think you Do coming much. to candidate. verify could on will advisory assessments a dystrophin surrogate the you endpoint? reiterate CONNECTX, performing for there be to the discussions

So, again, I readout to their dystrophin the let touch for component. regulatory first And will it me timing. turn on the over Michelle from

committee advisory upcoming on the that focused is is meeting understanding our So microdystrophin.

before, With it patients, normal we turn a enough there to the normal is the mentioned at microdystrophin by product in over to high product know approvals product molecule I exon-skipping, transformative functional will believe been has for XX% in approach have Becker is that an Michelle. than dystrophin that’s dystrophin of that, which we slightly that different be As comprising by native four And to a perhaps size truncated is produced of agent produced XX% exon-skipping to-date. on present our why molecule potential is if that levels. very I most The patients. the the is domains is and the expressed protein

at with XX. baseline at week more muscle we be Yes, performing biopsies dystrophin at and that looking will production,

approximately production So, us weeks of an of this idea dystrophin treatment. will give after XX

be the with properties platform And respect then just driving or are this That XXXX? observed the can at increased helpful. other stick very the just things after in versus that MDA, to it’s much. the you. nuclear would exon-skipping Thank cell some what levels guess, for portion any you presentations us versus maybe quick thanks the access of one other EDOXX recent compartment throughout penetrating the back analog, maybe differences and of follow-up physical remind to just EDO of like And that? I Okay, features the whether to circling RXG the peptide drivers

the Laura. question, appreciate Yes,

between interspersed of So, in which our we molecule oligo linear is acid greater essential much that with confers is three completely the these in synthetic arginines the a peptide hydrophobic by motifs but fundamentally comprising two core amino cell. nature a shorter the them motifs, polyarginine are those two stability sequences arginine, do at XX contain acids, non-natural upon peptide, sandwiched amino peptide. They believe conjunction driving the short into than

biology. better access are think on research different is and not very which the role into basis preclinical course, also function. that to getting perform sequence its a nucleus, very of we of So, with it’s we the but And of believe different cells, better both necessary we to only work, and oligonucleotide the access

very much. Thanks

call you. I’d to And remarks. the this back James McArthur over concludes session. now to like Thank Q&A the closing any turn for

operator in everybody great are. you. today’s Have you, for where evening thank Thank call. Thank on depending afternoon participating a and and you you

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

You may now disconnect.