OCGN - Earnings call transcript Q4 2024

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Operator

Good morning, and welcome to Ocugen's Fourth Quarter and Full Year 2024 Financial Results and Business Update. Please note that this call is being recorded at this time. [Operator Instructions]

I will now turn the call over to Tiffany Hamilton, Ocugen's Head of Corporate Communications.

You may begin.

Tiffany Hamilton

good and operator, you, morning, everyone. Thank

presentation. be clinical on Musunuri, Huma Chief Ocugen's me a Accounting update of Officer, will today's our more Medical Ramesh who financial call Shankar Dr. business results; our answer overview questions an on is will webcast and operational Chairman, Chief Qamar, to following available provide progress; Ramachandran, and the will Joining and detail CEO our provide Co-Founder, and Dr. and Officer,

This presentation the is full Investors ocugen.com. website we call release listeners associated will recorded Ocugen XXXX. morning, release, a This issued detailing the available encourage press slide business our press for being section highlights approximately on which website fourth and the a XX on is days. to replay for of and We at year operational review be the quarter with accompanying available the and of

outcomes meaning make Act may, statements development could, activities contains of and uncertainty may, forward-looking presentation This use these intends, Securities our in continue, to predicts, potential, or estimates, clinical some and plans, not related regarding XXXX, that preclinical terms but uncertainties. propose, are other of events Litigation expects, anticipates, risks limited or and might, development We are believes, will, Such words to cases, forward-looking include, of should can subject lines. Private time identify future statements. the as which such within to, statements the Reform anticipated statements

our results with more entitled Securities the and file may Factors SEC, to Exchange to reports factors, Risk fully SEC. in with in and other section the including the filings are that cause the quarterly or risks events we the risk the annual uncertainties current factors described and periodic Commission, our and described subject and These important that numerous are in statements expectations. Such differ materially from uncertainties actual risks

that the the law. statements contained make presentation, information, after presentation. result speak or of as presentation to of required forward-looking by date whether this assume in Any a update statements We as only obligation this otherwise events of as future we of new date this no this presentation, in except forward-looking

annual be Finally, today. report Form XXXX XX-K covering Ocugen's on filed will

I will to now Dr. over turn Musunuri. the call

Shankar Musunuri

for you, you Thank Tiffany, today. and thank us all joining

important move to Phase development significant of We save years, therapy potentially with modifier by next our also This progress we platform closer time OCUXXXST alignment are II/III even programs. OCUXXX costs in which possible to to X XXXX. sooner expedite a all week news burden an to than Stargardt line is our X that clinical ongoing anticipated. our in potential confirmatory us X years, to exciting making with eager brings in and gene clinical XXXX of in goal expected the X disease in XXXX, addressing OCUXXXST X trial to targeting BLAs OCUXXX pivotal the to announce share reached across forward It was novel it for last FDA the especially disease, BLA

and Now, commitment. but on along this the have ambition, an is with confident a commitment deliver our patients strategic scientific Board we to that to I'm unrelated expertise,

the lines runway providing the in XXXX. continuously line data forward development, equity enrollment XXXX. support $XX financings pipeline programs efforts drive advance with and continuing are time we XXXX, our secured to that extends million our to During first to half product and validate in cash in dosing are the we the into second our clinic, of quarter Throughout of XXXX debt and revolutionary we in platform in

addresses XXX associated unmet more in of the tremendous genes. gene-agnostic potentially with combined, With globally, see need meet ability the for the a capture OCUXXX effects and and more to one X.X people of and EU U.S. action. market to our can lead Retinitis its these million that share on than X% XXX,XXX through OCUXXX, Pigmentosa candidate mechanism only discuss medical you and Let's population, all detail. patient mutations X% product in the in market

February, regulatory line a opinion is ATMP that Agencies, for potential In product, for Medicines gene advanced disease treatment for therapies accelerates EMA the can provided of onetime to European the the therapy committee of opportunities for ATMP, European Commission this medicines groundbreaking review classification. advanced medicinal time OCUXXX and offer for the classification positive therapy life. granted

assistance. frequently protocol this Additionally, for more classification with EMA advice interact scientific allows and to Ocugen

OCUXXX. X.XXX statistically usual adequacy of Phase ETDRS demonstrated all at that long-term the for sensitive value chart was XX effect with In positive P visual significant fellow luminance, most safety X-year January, LLBA we low the OCUXXX This are These observed with trial treatment X of the are meaningful eyes gain, findings LLBA compared X of measure improvement the the X X-year on at in in a letters which durability clinically consistent equity subjects years, data from demonstrating treated when in of untreated eyes. to announced I/II year. clinical function regardless mutation results line

XX agnostic. X.Xx X:X of study group, with control a receive gene mutations who treatment arm, Phase ratio participants In III respectively. to XX XX remain be an per will The gene are each divided are either participants OCUXXX participants enroll is untreated into which year the in VG X randomized XX X arms, and in XXX will participants spanning eye study to row,

on of MAA filings first liMeliGhT OCUXXX of We're meet to U.S., intend XXXX actively half to the and BLA enrollment track the mid-XXXX. targets complete clinical the Phase remain to in in enrolling patients and III Canada trial and

the in With answer. combined disease, are subritinal associated and the retinopathies treatment OCUXXXST. received has of potentially Stargardt in approved announced we injection future. available fourth in also all received severely the OCUXXXST ABCAX critical treat week, medical needs potential this for address no Europe very orphan up retinopathies. Next quarter, a designation single the from the Stargardt product for unmet with with is which OCUXXX, patients patients with medicinal classification EMA U.S. along Earlier a an near OCUXXXST step ABCAX in ATMP that desperate associated to treatment and for the options to XXX,XXX is

evaluable the X.XX is Phase stabilization measured XXX% OCUXXXST trial untreated growth from versus improvement, corrected in I eyes meaningful data visual or slower visual injection in after patients. eyes X eyes. p-value a untreated X patients at function atrophic treated of significant a in X-month improvement function. clinically XX% XXX% eyes XX best at demonstrated in single in of by which X slower with atrophic fellow demonstrated visual lesion GARDian X-line lesion equity, eyes XX-letter BCVA, treated There of treated and in was versus eyes and OCUXXXST fellow treated growth months months statistically

intraocular maintains including OCUXXXST neuropathy, adverse events, no estimate a vasculitis serious city adverse of favorable and profile Endo cortile with cases special no vascularization optic tolerability and [indiscernible] inflammation, no interest. of events

trial size. eye clinical II/III will Phase single atrophic XX visual of subjects, receive pivotal untreated in of the the an VG will trial in The XX primary of the will worst XX the lesion ML The -- a of OCUXXXST endpoint equity randomize serve VectroGenomes, and subretinal, confirmatory change per X.Xx concentration whom of injection with is OCUXXXST at XXX-microliter whom in

data and We XXXX. compared LLVA as Phase be corrected visual the include to endpoint targeting are X-year utilized equity equity by BLA mid-XXXX initiate BLA to II/III visual best measured will submission controls. the secondary study plan untreated The for filing. and by

pathway, and to specifically developments to on are to OCUXXX, our move of doses accompanied designed current treatment a a intravitreal offer by over pathogenesis Currently, the year and complement in is degeneration, opens options a frequent advantage FDA-approved various in let's target-only dAMD, safety system. require address about per macular injections, which distinct one X XX pathways implicated considerations. Now multiple triangulated treatment

macular thereby with example, related complement to oxtatustress pathways note underlying important of metabolism, geographic roughly is disease approved atrophy, potential the are Europe. of and treatment. the disease. in lipid It activation also system, XX% all OCUXXX there to For has inflammation, to for the addressing regulate of degeneration, following X patients causes progression, therapies no GA developed

given published Approximately data of a slower our baseline at visual GA the of patients monthly suffer GA and showed untreated compared study. follow-up XX-letter in the X data eyes OCUXXX OCUXXX or of patients X untreated portion globally favorably lesions X the months EU and single leading fellow Phase compared inhibitor I retinal in data treated million in form a X-line in with same Phase function, X-month tissue from U.S. LLVA in at of of improvement complement Preservation Preliminary million X XX% in eyes meaningful from considerably from and to advanced to FDA-approved showed treated dAMD. to clinically I X at eyes Subjects lesion eyes million the eyes injection I growth, other and treated or at every months Phase versus month of time trial.

XX XXX-microliter ml, vector the untreated. subretinal X.Xx group ml, study, XX remained XX dose, high In to X rigs medium medium the of XX with while Phase control VG high Xx the dAMD into treatment of in or OCUXXX [indiscernible] which of secondary is single be dose a assessed. a per administration efficacy were gyms In a XX received subjects to and patients or safety dose will GA the patients to group, either treatment or control per II group. randomized or groups,

Corydon no reviewed the of This inflammation, for including XX neuropathy, have week, of adverse treatments subjects, and or estimate portion adverse from convened and date no DSMB neovascularization to No Unlike the events the special an optic safety Endo serious alluded currently Phase the of available were tolerability vasculitis, interest. GA, events reported to OCUXXX XX all II subjects in additional intraocular there cases been study. profile and of

future the XXXX confirmatory clinical Interim us will as help data as XXXX. of the second and will soon filings trial from clinical III study in and for BLA design a enable XXXX. MAA planned half available data Phase pivotal be potential This our ArMaDa

our treatment, that the GA it medical we multifunctional gene modified a effect gold options be believe OCUXXX can for therapy, propone is standard potential treatment the Given a the worldwide. limited of one-and-done unmet as treating distinct and need, fact

trial being to would with OCUXXX our for Edima, for and clinical biologic vaccines Lastly, call and OCUXXX and DME, has in the landscape platform. Phase diabetic clinic DME. to like to treatment into attention the currently macular macular diabetic the unelation its change candidate all to moved of AMD, current I wet active patients are dosed retinopathy, unique potential a crucial holds anti-VEGF promise disease role DMA degeneration, respond binding benefit and to of receptors the patients component patients, who pathogenesis therapies. with action, integrin including to the XX% -- mechanism XX% play not in that do to I

escalation dose dose, X.XX open-label OCUXXX trial OCUXXX I dose, injection. milligram. multicenter, The total X.XXX versus medium where All intravital after of milligram Patients Phase place clinical subjects drug high and take X safety X.X the a X and a injection will X follow-up apart. and weeks up of low last study cohorts, a is to receive X months intravitreal dose, milligrams injections

the of Couple is I company's inhaled of COVID-XX, mucosal by OCUXXX and sponsor of -- are the all United drug tolerability million worldwide the Institute to part National administered The trial AMD. DR Institute by for and different by wet Phase -- people the safety, vaccine, Allergy and into million DME, or for XXX NIAID, cleared to of the people the the expected Diseases, IND Infectious States, assess application immunogenicity routes, National was or FDA. affected The in conduct OCUXXX, inhalation Health

studies will to the the the will XX receive the XX where antibody the to receive each form. second compared high assigned vaccine group. assigned group years. Within a subjects be CADXX group, of subjects secondary vectored and and Malaysian on breakthrough of XX safety dose The Phase COVID-XX to mucosal and antibodies dose immune low-dose intranasal enroll to start technology of aged systemic emulation vaccine Earlier the to the XX the response based clinical same patients. production, up the subjects exploratory XX as The systemic increased is to and to to a primary form is administered administered will is employed intramuscular. well Phase a similar determine anticipated novel trial year durable I X of the subjects be prevent clinical as will and the trial number in COVID-XX that aim XX technology. I endpoints demonstrated will include Panera other the quarter study by OCUXXX muco XXXX. using X/X subjects XX

provide to the to now I'll update. call Ramesh financial the Ramesh? over turn Ramachandran

Ramesh Ramachandran

Thank of full key overview fourth you, quarter an of I Shankar, and good will the now results morning, year XXXX. the and provide everyone. financial of

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

Research year full compared the were ended expenses ended the year For to December $XX.X million $XX.X million and XXXX. XX, December XXXX, for Development XX,

ended loss year for share net December December loss for million $XX was the ended XXXX. expenses quarter loss administrative per XX, net December quarter for of million XXXX, net million were the $X.X were to for and and $XX.X per ended XX, administrative the compared approximately XXXX, share XXXX, expenses or quarter quarter fourth fourth $X.X December XX, to or General year the $XX.X ended Net the million million General of XXXX. million for for fourth compared $XX.X approximately to XXXX. the compared loss $X.XX a $X.XX XX,

was or or loss $XX.X the full net $X.XX net for a $XX.X share net share. year net loss of per year XXXX loss million loss to million Full per compared $X.XX

restricted $XX.X XX, and as XX, of cash XXXX. cash December XXXX, totaled million December to year million of Our as ended $XX.X compared

that into operations XXXX. first fund the restricted of our enable cash expect cash We us to will and quarter

proactively working our opportunities platforms. always, for including partnerships capital, increase As strategy to are long-term that will exploring we shareholder-friendly our drive scientific

That you. to the update concludes Tiffany, for back my quarter.

Tiffany Hamilton

you, Thank Ramesh.

for We open call will Operator? questions. the now

Operator

first Michael question of Our Instructions] from [Operator Maxim the Okunewitch Group. comes line from

Michael Okunewitch

studies all your OCUXXXST potentially off, like more guess, this a good about launching Phase the in housekeeping thinking Congrats the does I on II/III? progress. for When first thinking newly factor question. about runway, of

Shankar Musunuri

Yes, Michael. budgeted. It's already

Michael Okunewitch

a program, that evaluated And that when then All And from idea efficacy being then of Do of guess I... given sort you to right? endpoints have Phase just I expect what in is DME right. see data the could an are to terms of it we I? emerge Phase start

Shankar Musunuri

ask I'll

Huma Qamar

are question. the of BCVA for you safety administration of efficacy as we exploratory endpoints dose looking of year. the and intravitreal unilateral also the the of a of safety assessing at this OCUXXX And looking the Actually, we of Thank towards are response report end we're OCUXXX, OCUXXX. the at and

antibody also end of as are looking points secondary the at well. of and OCUXXX we PK formation However, OCUXXX

Michael Okunewitch

into more for the one and just then And Okay. back queue. me I'll hop

recent at funding have for OCUXXX I really NIH? heard -- a given for program anything you around been core heard hasn't that we've Phase just this just that much Just funding AXXX, right, of know but regarding perhaps, lately, availability I, uncertainty the

Shankar Musunuri

track Yes, still and NIAID meetings Michael. initiate with The the us our after I. had IND approval, they're stating to they're Phase on

Operator

from Wainwright. the Our of Swayampakula comes from next Ramakanth question line H.C.

Swayampakula Ramakanth

me. A couple questions of quick from

So Shankar, BLAs filing team you next target starting certainly your have X made of an for year. aggressive

towards gives we So should be and this? And out you that watching this goal? as team see achieve your confidence what investors, what our progress could to you

Shankar Musunuri

of the cylinders. into and most 'XX, mean are investors our today, the getting running gene at question. all to We of record. I clinic Good all X beginning we from 'XX, therapy in programs programs And have 'XX. Yes. need look started track late the we

for track itself. record speaks So our

And us recruitment. up. follow-up start so to be Phase months lines goal thanks And to ability BLA. Stargardt and should if into mid we for Phase will significant year, mark so once are medical us II/III the trial is, clinical that this I disease. then Phase of reasonably need unmet a that trial for to about X we and FDA X-year so II the BLA It's trial naturally into targeted convert confirmatory allowing a the mean that and -- III estimating up for put or existing it's again, 'XX,

a clinical for we trial geographic completed targeting in that Similarly, our month recruitment recently atrophy. II ago our getting OCUXXX OCUXXX, Phase is we're announced into

our next completed that will Phase II means So by year. early be

in will discussions Our which year well interim design. and as to allow the as is FDA data III us start on half EMA coming out second of Phase conversations this clinical with having

to duration will a have and patients So to that out the us such diseases recruitment. Therefore, Phase initiate compared reaching can including again, orphan where and that hopeful relatively confidence as we we with to that is and clinical next year. easier Stargardt, many so GA II we are trial trial, us. to clinical X-year RP Once commit are recruit patients during gives that

in BLA. we done that If it start for get 'XX lined can relatively can 'XX, and you up by be

that's I And so are why time think reasonable. these lines

next starting atrophy. OCUXXX and XXXX So year, Stargardt be is XXXX, targeting we're disease; a RP, and OCUXXX, and -- going retinitis to geographic OCUXXXST

Swayampakula Ramakanth

Perfect.

GA. XXX in just about we're So for

there that approved two for therapies know this we So indication. are

difference? and point So given would on the to therapy that, OCUXXX to how initiate convince easy price physicians be both it considering patients

Shankar Musunuri

Go ahead, Huma.

Huma Qamar

price provide is can patients here huge for so the I'll ahead the already in of Thank terms things the was of the completed because question. way. way who GA we got mention you clinical time the SIFO to But and recruitment of of requests Shankar wanted provide. X and a for there aspects point that have I number from

first recruit fact, and the and are foremost on of to a products particularly the and the in we are terms certain the had wet of there, tolerability III are progressing number profile whatever AMD. the clinicians, safety they for those of now to Phase of competitors, the and right patients waiting therapies concerning list, approved terms with in XX% the our In the were

-- safety our hallmarks the of were there approved X events tolerability adverse and of a optic that well products approved which the vasculitis, currently the gives us ischemic serious and included CNV as the and extreme and Also, those like In protocol X-month had terms for of no are that profile OCUXXX, confidence period. neuropathy products. for

and tolerability as only well functional is and Europe, are safety product. improvements efficacy in the approved no the of terms in and in And as differences or there recruitment profile, structural outcomes. seeing of terms not the in we So

majority of compliance So to year that of of physicians of really the do to as and not the XX are also patients X injections considerations older. and age the being safety age per most years is are reluctant the XX issues onset see and because prescribing you

add terms let Shankar of I thoughts And in price his the would point, here.

Shankar Musunuri

Thank you, Huma.

RK, stated, X geographic dAMD, get itself than of potentially late point which patients most funded these is million So price U.S. patients And has we a from the CMS. with perspective, atrophy. by as more

other with are either We're after they're targeting ultra-rare the they products need therapy gene that treatments. gene already complex to start them. unmet are after very in how in priced. one and an or of there organization, working to using biologicals we whether replace solution watching or And deliver are therapies, injections diseases multiple medical scientific diseases certain to Obviously, So or a unfortunately, they're date, we instead marketplace, as an going mindful. diseases very getting going a marketplace, need. have which It's to is done platform, Vector of the taking like something they're not

need reasonable, when we to number points. huge one-and-done definitely, it's and one, And a be you population price So a all have pricing this, the treatment. should consider the like

in or have So many of years life years of remaining many, even XXs, mostly the patients still quality they are them. XXs for

need So payers fairly reimburse make get we and it, we'll to that patients do really the sure and the it. can the it our price goal the so pharmacoeconomic analysis, appropriate product, they who will is

Our access. provide goal is to market

them. So patients get our going perspective to work need effort make we our on to product, we're our sure every in

Swayampakula Ramakanth

progress, III with on year? So the in OCUXXX, study should I/II of and additional III we the with study your question what data now me filing last expect -- the from Phase Phase next Phase BLA between from program

Huma Qamar

safety. recently well updated have our as durability as as profile on So we

have That's we to So safety made. will continue there. commitment that be a

parameters continue on to also they LLVA accordingly. functional and the the report points, become other as available. end efficacy parameters will So we

Shankar Musunuri

that's announced we data, perspective, at the important, not too. durability LLVA only The payer Okay. X-year from a recently

the we'll of have definitely filing So time X-year year. next data at

Operator

NOBLE next comes Capital Our from Robert from the question line LeBoyer of Markets.

Robert LeBoyer

trial? there the company the revenue Congratulations be all question I to whether to with OCUXXX do will any has the My grant associated progress. on Phase with and

Shankar Musunuri

Yes.

stated publicly, have company a is completed And sponsoring from we As we perspective. program. this obligations NIAID our

the develop for and clear IND, FDA them. -- getting and for that the all and manufacturing to We doing preclinical approval work are and transfer then responsible filing

done next So program to to supposed are and the and we've our it the part, they level. take I fund clinical Phase

Robert LeBoyer

trial? funding Or agency where the recorded run be just by Will Okay. something the you the will this revenue turned be as company? to over as they the

Shankar Musunuri

over trial. agency, be we'll Yes, the to the run they turning

Operator

from Gataulin comes of question Chardan. Daniil next line from Our the

Daniil Gataulin

A Congrats on the on of progress. couple all questions OCUXXXST.

the what your the is do OCUXXXST? for for Phase And study? using II/III manufacturing First, commercial-grade strategy you product on plan

Shankar Musunuri

I used Good similar question, are same made at RP. a scale II/III. Phase Daniil. for pattern We'll we follow commercial the for We Phase will our scale the and introduced be already into like doing

thing with scale and program, and forward lots FDA our For that. RP, do the for strategy, same trial. -- introducing consistent which we're We'll with commercial X SD we're agreed moving is the

Daniil Gataulin

And it. U.S. of for using And each if sites? and ex you U.S. terms you're looking Got Okay. study, sites the both this at in both? of are the proportion what

Shankar Musunuri

of pigmentoso. no need up sites, we population, the Stargardt. the for for on But patient based rights Canada Canada, I in except we two is for we'll activate only sites There necessary, XX do have course. If patients. mean set

comfortable patients I those and team in her get can very think that quickly. U.S. Huma the they are

Operator

Co-Founder, turn to portion. Chairman, Dr. CEO now over This Q&A call back and concludes Musunuri. the I Shankar the will

Shankar Musunuri

gene continued We for forward diseases. transformative stakeholders to a we as as We the therapies year significant and initiatives. the Ocugen blindness great interest appreciate pioneering you, biotechnology day. our catalysis, operator. a Thank move Have leader involvement key establish in we of of ahead as look forward

Ocugen (OCGN) 5 Mar 252024 Q4 Earnings call transcript