IMRA - Earnings call transcript Q1 2021 | Enliven Therapeutics, Inc.

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Operator

Ladies and gentlemen, thank you for standing by, and welcome to the Imara Inc. Q1 Earnings Conference Call and Webcast. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question-and-answer session. [Operator Instructions] I would now like to hand the conference over to your speaker today, Mike Gray. go please and you, ahead, sir. Thank

Michael Gray

forward-looking this you, Imara's first Thank various factors, XX-Q well remind our plans forward-looking that provisions other make recent make of on and quarterly result date. materially implied of SEC of and of future the prospects I'd today we we good set to with welcome a most everyone made during we statements Factors as any represent events XXXX. and statements everyone, views Form various call on harbor or relied the of quarter Any expectations, as important as be to about that company's morning, like views representing Okay. forth conference XXXX call. not from SEC. Risk the in Securities upon only or morning Private statements our statements results that the as the report purposes our forward-looking the filed as and as these call this Litigation those subsequent filings differ those Actual by of expressed constitute safe section could with for under including any Reform Act should conference this

return may discussion forward-looking on the review financial we'll for now as if to President our the rely CEO, change. representing over then Imara's questions. quarter at subsequent future, While date views obligation not and statements Rahul's we results, do turn as that, in elect to to these even I'll so the update Ballal. you open any our today. call first and call I'll of we point With any Rahul? our Rahul some forward-looking to following these to specifically disclaim statements Therefore, views should

Rahul Ballal

year of for beginning The period Thanks, you data-rich morning, joining cell sickle and of It for both programs. Imara. marks Mike. been start this morning's and productive everyone, has a beta-thalassemia XXXX clinical for Good call. thank our a

IIb clinical made which We have doses substantial sickle with patients are enrollment disease of beta-thalassemia, test IMR-XXX. for cell trials to higher designed our Phase in progress and

on We XX the screening a trial, clinical XX transfusion-dependent at remain enrolled for to interim and We expect Forte Ardent report and beta-thalassemia across Forte the to complete that countries interim fully second programs. are of subgroup. activities in data the track XXXX closing these now trial the of arm for conducting analysis protocol-driven studies trials sites half both the these for new and

this both by the data excited entire company This XXXX. studies a from report in also these the second half XXXX across to and testament and the is progress of for I'm analysis analysis to We the appreciative expect of first primary function half data from final in very the and momentum.

Committees, Data the clinical following Monitoring up currently IIb recommendation Phase Importantly, treatment both independent the testing opened we trials are of at daily have higher IMR-XXX in XXX milligram. to doses dose of arms and

four of in are active randomized being With an dose both arm. trials, high open to every treatment out the three patients

a a and patients have XXXX one one the which low Both schema, placebo enriched, dose two-to-one-to-one from two being dose high high of will is in high readout the With arm. half second interim dose, the randomization. dose,

or IMR-XXX in VOCs promising Phase in saw we and in variable in reported progress our In vaso-occlusive addition trial crises which from and HbF in clinical clinical data disease, to in was the IIa sickle our the rate tolerated F-cells. cell with well changes IIb trials, IMR-XXX we top-line of Phase reductions of which

XX comprehensive Hematology EHA the tolerated of from and VOC We we're Congress heart in extension XXXX. completed of that and pleased therapeutic in data to studies Association Phase to larger June. increases XXX-milligram and IMR-XXX with the additional IIa ongoing in plan or the F-cells or failure open European well of additional to an later XXXX patient indications. We trial have at ejection parent observed. also as working extension abstract We've label reported trial, Virtual our upcoming was We're study, potential hemoglobin the as in showed successfully IMR-XXX at fraction HFpEF, meeting to IMR-XXX well Phase preliminary have data open-label which present pre-clinical submitted IIa also explore in preserved in been doses cardiovascular fetal data from from which

of to continue with concept formulate of protocol our cardiologists. made a study Advisory a leading for We is proof up Clinical which Board,

annual families affected second disease an launched support challenges of cell disorders. we serving those Lastly, rare XXXX blood to local program the through for XXXX. incredibly community-based and in time organizations, March, sickle difficult many by these and Real Impact beta-thalassemia living Grants as move and remain we with patients marked

including Impact to community-based Real from to three areas support the expect We grant under increase virtual or social capacity. XXX,XXX relief, COVID-XX health, of to program, across determinants Program funding including key CBO XXX,XXX, in XXXX and organization XXXX up

is grant strive of We to Real a and patients first, continue to our mission. put core embodiment program key the our Impact

We with core adult Phase we few the an forward to I details our expect their now Mike spend to with patient sickle XXXX from begin awards on and turning with the look patients ongoing I'll trials like our commitment programs recent clinical overview including Forte results. and to supporting be minutes IIb would progress cell with of beta-thalassemia. disease the the before back recipients a trial community. review Ardent the call in made in adult to to June trial and patients in of to financial IMR-XXX further

global we Forte and transfusion-dependent activation XX across and with both randomized in resulted in countries enrolled, now has arm the centers Forte XX and this Ardent Having have effort in clinical accelerating fully for arm. trials the XX patients Forte expand active enrollment beta-thalassemia trial. our of in We additional over continue respectively XX Ardent clinical This near-term with and operations footprint studies to centers. the and

With and in these team's of effectively for to these XXXX a interim pandemic. remain half we the the the this mentioned second first As primary each expect thank second IIb increasing from complete guidance trials these from commitment we respective of a their from now year. from protocol-driven these Phase ability our the this this to studies. And trials opportunity these for the pleased tireless This enrollment, half of half to each in in accomplishment in studies data I'm XXXX the to and trials analysis earlier, global the demonstrates from them I'd on of of to analysis final track to is data take XXXX. earlier reaffirm backdrop of analysis significant and like conduct

higher these quarter, for each and opening of following once-daily XXX-milligram the trials in the Ardent dose first of is is Committees patient IMR-XXX Monitoring once-daily overdose, independent in the and and based which separate Data patient IMR-XXX dose, arm higher safety During available proceeding Forte in tolerability of pre-specified of each enrollment These of the the study dosing placebo. treatment XXX-milligram weight review XXX-milligram or additional or XXX-milligram data. dose were recommend studies IMR-XXX which at arms on protocols the the two is based on weight. or

day as disease our completed to Phase XXX-milligram in XXX-milligram to day started recently starting and IIb per per or treatment a IIa XX both titration. at on day as starting without escalated trials cell and point, milligram dose and a As dose as higher the sickle is sequentially in Phase XX clinical the through trial over low as period reference weeks. clinical the XXX-milligram high one XX Dosing substantially

patients is adult and approximately first for Phase XX placebo as reminder, endpoints, a as an secondary additional trial with VOCs. indices evaluating the statistical endpoint increase significance data or placebo measures. Ardent blood adhesion, hemoglobin The will XX for evaluate VOC, be the life greater Patients enroll annualized of of secondary of will including this use XX As include IIb for the patients quality trial, by to defined white baseline week as will disease the planned well of and is is versus by objective and cell randomized stratified biomarkers, versus a associated HBF planned primary endpoint. continue red and sickle through HBF treatment cell of powered trial time region. but cell of Other provide response hemolysis, with from hydroxyurea double-blind to to endpoints proportion to patients fetal efficacy secondary IMR-XXX key on weeks the X%

trial safety evaluate and in and XX Forte patients. patients transfusion-dependent will tolerability approximately non-transfusion-dependent approximately of IMR-XXX XX

trial F-cells reductions in OLE We I'll burden, to an prior We transfusion-dependent during which The clinical dosing. to now of treatment patients, pre XX in transfusion HbF clinical IMR-XXX placebo IIa trial. briefly trial. IIa For the patient trial as the reported and and F-cell a PK HbF safety Phase to be program. after March. and for for endpoints load additional important Phase completed this from turn tolerability will in next well I in as look activity IMR-XXX this the very January, historical also new next of extension were was was changes XX month presenting biomarkers after IMR-XXX this VOC clinical Forte transfusion XXX-milligrams IIa variable these rates start. forward from Phase transfusion disclosed as VOCs Safety or trial and in rates examine there on and of certain iron we EHA to data will as variables in of of comprehensive XX will Phase trial hemoglobin, comparison our IMR-XXX promising at assessed believe the tolerated observed. of in from vaso-occlusive seeing well IIa turn to results results the change biomarkers placebo-controlled plan we data new including crises the in data trial, result in exploratory which versus endpoints. help reestablish and topline weeks our open-label study and and weeks We to evaluate remain outcome with effects

treatment IMR-XXX patients demonstrated F-cells the XX in observed at of least a Biomarker of data increases absolute PD the XXXX and in safety December IIa trial. was allows completion in study administered program patients, of the for OLE dose As daily patients of and the quarter increase and enroll a second a designed trial daily initially in the to then profile OLE XXXX of for four evaluable to A review four treatment. trial. program well enrolled of patients tolerability XX HbF after XX, XXX-milligram The that IMR-XXX following of had was Phase these months from clinical and IMR-XXX similar of in months had of Phase on the preliminary protocol safety of and tolerated both reminder, to as XXX-milligram. OLE biomarker long-term IIa the Approximately who the Phase a demonstrated a four-year dose IIa results trial were trial. OLE

in safety. two In data, and addition case an separate our that OLE committee December maintain is of IIb efficient provided trial the XXXX. gain-driven American higher safety line we XXX-milligram being in is narratives weight. minimum Hematology to dose Meeting patients both baseline greater to a approach a most studies, HbF to for Falling this with to arm update four-month the review with exposure equal in at Phase weight approved in to their on baseline escalation we March, in same continue way dose maximize versions. Both the IIb see milligram from and of based F-cell X.X% eligible employed a of Annual an increased has Society XXX programs upon from in reported also dose increases certain of our dose daily or Phase patients recently This and daily

doses on expect mid-XXXX IMR-XXX start the and a MHRA, submitted of and patients revised protocol the have higher transitioning to to OLE study. in We to FDA

back from marked We quarter to the Imara in additional to progress of I data multiple IMR-XXX. eight-month and turn you. administering another transition period our XXXX. enrollment that now you In the forward present biomarker EHA data And readouts look to also Mike We planned Congress. results. Mike? the the to on the we higher at conclusion, financial including review further will for productive upcoming Thank important including PD doses in expect both to believe call data VOC OLE and our gains updating first

Michael Gray

Thank you, Rahul.

compared to other as personnel-related The the to the the also for $X.X General was $X.X in as Our which increased the and we morning. as R&D first result million XXXX. million expenses are as due that compared the included were was costs. and of this administrative increase increase XXXX of filed other or XXXX. million issued personnel-related in XXXX details summarize as XX-Q first The million quarter for first $X.X $XX.X More increased for quarter also can first operating SEC the of XXXX. now. morning, public costs first common primarily $X.XX operating the to per this of of R&D attributable G&A or as were operating with quarter found we of first the of be million our that compared was efforts first IMR-XXX of $XX.X $X.X release $X.X million a to quarter of share company. million primarily million $X.X for dollars stockholders expenses $X.XX development quarter the quarter well earlier Net as XXXX for quarter of press and related I'll to per to XXXX for share loss a results

we and our And first Series be $X.X you equivalents, open remarks. sufficient operations of Could $XX.X stock. the included into stockholders for of The first line with net cash, questions. investments B million with XXXX we're will this charge concludes convertible to quarter common the questions? please associated expect that ended to Brandy, ready our depletion planned quarter million We cash fund and that attributable prepared mid-XXXX. loss a for

the If line. could you open

Operator

the Instructions] of [Operator Nochomovitz Yigal Citigroup. with line comes question first from Your

Yigal Nochomovitz

the and first, Ardent I on could for I the analysis a arms, that. on for Forte. that Rahul have patients analysis confirm and higher Thanks Mike. you At will interim taking Hi, so, very enrolled much the Rahul I say questions. you had interim the that dose include heard few thought,

timing the for interim what what aspect for And triggers Ardent your both the these a finally, protocol trials? constitute interim would the for analysis view the Fote? of and of positive Second, Thanks. of in outcome analysis

Rahul Ballal

patients the one. I analysis Number Thanks, for IMR-XXX. the finally, Yigal. Ardent for I then higher driven following: report outcomes. positive trial the patients will XX higher referencing randomized include your at approximately of arm they those Number both for and slightly trial out XX Forte the weeks analysis we the as XX trial, And Ardent questions. beta-cell confirm interim dose protocol and are I trials the the and They're pre-specified doses two; sickle Yes, think what that can cell appreciate for the different. Forte was to question for see trial.

at so, beta-thalassemia trial, the in a is of will be we'll a and combination, looking combination For be trial we at safety patients this our efficacy. looking first

reduced long to looking subgroup transfusion as addition as hemoglobin transfusion-dependent pre-transfusion -- we'll the for Specifically safety. in be looking and for for burden

a hemoglobin. For seeing trial of biomarker well in is at the non-transfusion as constitute improvements And the as patients, we'll well some dependent dependent transfusion in in me that be IMR-XXX looking some what as of patients. the tolerated in non-transfusion for improvements those starting well PD higher population doses specifically transfusion to burden, patient beta-thalassemia fetal biomarkers in patients that in hemoglobin and success as are see

powered study. cell Sickle a is cell the sickle it study trial beta-thalassemia is study. It's that's different. in So, our and second slightly

arms of say improvements the placebo. and improved certainly in cell active some dose sickle those and rate XX fetal high see as is we efficacy that we'll that and placebo. outcome, what So, see looking hemoglobin sure you dose at those two versus be a response in in the I hopefully interim patients as those the but give understand that trial, we is are endpoint I active for like patients because in a XX [indiscernible] of number would can't making a patients the positive dose of there on weeks, trending upsells terms analysis, specifically at successful low to dependent well are if the of we XX

response fetal placebo as nice be to response, arms. in in it'd improvement hemoglobin dose-dependent see versus the active as So, well

Yigal Nochomovitz

super down you I the opportunity would That's you studies but specifically when avenue in actually from mechanism HFpEF, just Could an failure a have HFrEF a the had road, the more XXX that on in is conclusions for indications additional the great. potential consider quickly for IMR-XXX. just information? suggests or question not helpful. Okay, and you about HFrEF the summarize heart what preclinical

Rahul Ballal

Yes. Sure.

preserved Two run agent preclinical model. phenotype treated creates are as of models. preventative them an Those heart phenotype. the they HFpEF they II insult studies are we have that of an with through three -- require failure angiotensin and mouse II an EF in So, aldosterone preventative two the

with patient caustic So, been the at three you treat those has top specifically IMR-XXX agent mouse the a -- ultimately and on IMR-XXX protective and of in areas.

fibrosis, first inflammation. in and in one the the The second hypertrophy, one in one third

and expression when PDEX a we a expression able as for we and reduce HFrEF were over-expressed in in and in developed stress you HFpEF could so with works found configured cyclic number asked weeks a treat phenotype getting of approach approach key the similar cardiac DB uniquely in a uniquely see it reductions then GMP have Across HFpEF, those model reduction is in mice a is mechanistically had multimodal in about model. versus preventative inhibitor. phenotype mice a IMR-XXX for saw increase BNP in and in why a specifically neprolysin preclinically relevant our to PDEX phenotype showed that -- cardiac in into who it as phenotype finding in the So, we a all for that hyper model Then you we and takeaway and therapeutic step XX after hypertrophy, eight generated works mice weeks our that we question PDEX pathway have a the of be a and reductions fibrosis. and anti-pro those an HFpEF of and the one further in treated that we PDEX models, three bigger took biomarker the three reduction treatment as of models inflammation that eating PDEX in

that is overexpression mechanistic model. a relevance of in mechanistic, direct there PDEX HFpEF So,

positioned in it's at indication HFrEF. as I of meeting. -- why will XXXX this cardiovascular on that fall that's [indiscernible] said And a be we uniquely for call so package versus be we And will the believe whole the

Yigal Nochomovitz

Got it. Thank you, Rahul.

Operator

of next Your the Harrison comes from Matthew with line Morgan question Stanley.

Unidentified Analyst

us This Matthew. Hi, from morning cell on for sickle disease. A [indiscernible] to question good everyone. is on

that your time preclinical PK work ICXX. that the You commented of efficacy remains that have is driver that the above suggested XXX the

efficacy these you be out you. data expectations IIa in We as parameters, such your whether validated the AUC in are Phase have change and the turn or Thank Cmax would wondering the case other PK drivers? how to

Rahul Ballal

you. Thank

couple So and exposure we data it, look determining appreciate and time be or you we additional of presenting that will addition to that above asking AUC. to answers some that ICXX and looking at concept the levels in question, had start I around help response Cmax at that trough

So, parameters. of the increase to to up certainly going part of advantage of different is EHA. on number in data PK us up two, Number at that up box Number going XXX-milligram these be and one, by XXX-milligram. check that dose a you presenting is front we'll of of AUC exposure across the you

increased hours. increase the, certainly levels for trough You Cmax the XX and you past ICXX

of curve. this like the the of Phase for we so IIb response the advantages that play up across not but is part dose just extension the for those the exposure going program, on get reason is parameters we And see and out PK open to label

Phase thalassemia AUC, including IIb understand to realize we'll noted for higher beta interim in so about be dose in you And Cmax. as think the PK the CXX and as additional help potential side continue on to us data things, treatment, the the specifically of analysis work doing you coming of

Unidentified Analyst

Thank you. Very useful.

the to forward data. Looking

Rahul Ballal

Thank you.

Operator

of Your with from line Leerink. Schwartz the next SVP question comes Joseph

Joseph Schwartz

to that asked what question a very my interim you I I on he first and hoping much. and entail? look the same have was similar entails. the that when I thanks interim first Hi, will about Yigal's the is analysis question -- something. maybe and what only But before? through us a do Is new I analysis primary walk final could missed recall analysis

you based these you unblinding seeing the And any And to keeping in with entails, looks of you're intact? so these of make the is in what okay corrections analysis? addition and trial will regulatory agencies each to what course on primary each be and able

Rahul Ballal

question. good a It's Yes.

you me through. Let walk

in So, due -- had the part an a long fact we've primary studies the you that that's in final quite primary and are interim know. always Joe the a to as

And of step analysis well weeks X% will looking It as done for time give at report initial VOCs or statistically and the XX at XX least a to we patients and point, we'll you litany preserve fashion biomarkers half Let treatment HbF and greater. I which second know, biomarker. statistically hierarchical patients both to be at response a XX as both their a looking at will analysis the patients rigorous me till powered a in analysis to of at see at look sickle through PD first exposure and at be to the that exploratory as as VOC The out. to down of studies look snapshot year, analysis, understanding alpha. that and expect a in check is look really is of beta-thalassemia a, you a paradigm. interim of this we've of XX-week maintain will on in and in The XX the time you XX what annualized primary power secondary primary walk box at which fashion and at weeks that is study in as tolerability, powered hierarchical other and safety, as guided then an also

So, will a XX that read are be link. the out at XX, but ones treatment those through go patients week patients We'll continue.

The program primary. a slightly It's XXXX. It's VOCs in be we've and and look The analysis length. final second different. will XXXX. shorter we'll primary half focused beta-thalassemia is the the guided That at which part six-month final in treatment annualized nine-month of point VOC. in And so be will time be the paradigm XX-week half specifically period XX-week look on course the of that as of at first will of a analysis analysis

arm the year. patients, half XX interim subset XX XX-week the for will XXXX half patients first the NTDT be patients we'll months XXXX, so And the a in XX this looking a at those be nine time In and at weeks of will be the of in looking of the second in arm subset analysis in of or at point second we at And again TDT XX weeks. half looking of that beta-thalassemia. in at

the step So, and just will those XXX in back, be TD taking NTD both analysis. final a that subgroup the in patients

so difference And overall timeframe in between for and XX the XX final is beta-thalassemia and XX delineated weeks, versus and already primary is difference between weeks help? sickle the the weeks, in XX I've final primary versus weeks that. Does that

Joseph Schwartz

be Thank That to your MAD decision, exposure makes a a in currently. sense. that that of then response a twice talk being planning And anything on seen queue that lot to far Absolutely. helpful analysis? that appears again. highlights Can study you doses what you. from that you've are based stat at about you're perform up to so studied is That's looking the

Rahul Ballal

Yes. Sure.

if we're believe cell So, we those in higher dose doses doing in when we Those and points doses arms IIb blinded. opened the believe Phase up obviously dose We to arms tolerability. those for to from they're and signals higher both safety well-tolerated. sickle how doses March, see beta-thalassemia January open the that We and we're proceeding. higher are studies the wanted for

but dose to so you of we XXX-milligrams IMR-XXX is MD and that study of part higher the upon the SAD going optionality continue now range had that, start Phase we data beyond and believe as do Will SAD certainly allow opportunity IIb so as that a this through is make study higher us which have dependent expand programs. to will we potentially? later. had why an That study is for started the wanted we we And the IMR-XXX to to MD just are and wanted versus potentially that SAD noted we to study doses rationale sure explore we MD to that doses implement

Joseph Schwartz

Very question. my for Thanks again helpful. taking

Rahul Ballal

Joe. Thanks,

Operator

now And to this there further closing questions I the are turn back no remarks. to the call speakers like for at any over would time,

Rahul Ballal

look later this forward updates data-rich future and joining this we're appreciate a everyone. you. Thank [indiscernible] to that We have us Thank at and you as enrollment year, you, have you excited We'll data see EHA. to year to morning.

Operator

call. conference concludes This today's

You may now disconnect.

Michael Gray	Chief Financial Officer & Chief Operating Officer
Rahul Ballal	President, Chief Executive Officer & Director
Yigal Nochomovitz	Citigroup
Matthew Harrison	Morgan Stanley
Joseph Schwartz	SVB Leerink

Enliven Therapeutics (ELVN) 11 May 212021 Q1 Earnings call transcript