ONTX - Earnings call transcript Q1 2018

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Operator

Good morning and welcome to Onconova's first quarter 2018 earnings call and webcast. At this time, all participants have been placed on listen-only mode. At the end of the prepared statements, participants will have the opportunity to ask questions. [Operator Instructions]. Please note that the remarks today will include forward-looking statements and that actual results could differ materially from those projected or implied in our forward-looking statements.

note important refer could statements you press and of to actual in description in release we statements differ, to a a factors SEC cause that results today's the For company's forward-looking filings. on the forward-looking my now the turn and to Onconova's call Ramesh Kumar. pleasure to CEO is It over President, Dr. please Kumar, proceed.

Ramesh Kumar

Thank Good welcome and results morning you, XXXX to call. Victor. our first quarter

of public Guerin. from $XX.XX significantly Earlier and team raised higher-risk CFO, The underwritten therapies available this million advance resources there the we position management our to is complete Rigosertib funds FDA-approved Joining leadership to us topline MDS, are Onconova's the provided this Mark no who infusion patients to me hypomethylating III strengthened from a month, enrollment indication completed upsized important for with sheet offering, offering. proceeds agents. strengthens INSPIRE our in our which of IV refractory with needed our are combined balance data. Phase February, for and The in cash

in we're also result advancing the of completing therapy Phase in III Rigosertib, enrolling study. discussions after full trial an a FDA. addition, Phase the development the start interim towards promising we II the Since XXXX, have Phase with made II program after the expansion program and pre-IND combination IND of clinical CDK our inhibitor In progress including INSPIRE announcing

be expanded from XXXX. trial of in with analysis Looking be expect we completion, the INSPIRE which achieved topline ahead, can that we both enrollment could believe concurrent

by regional agreements the to advance our of for We business licensing candidates. strategic objectives, continued have illustrated development also our pipeline securing

footprint Latin is Austria Rigosertib Pharma, agreement to with reminder, wide an portfolio in a into in we has in Latin America. Vienna, As Pint hematology/oncology a which existing based and commercialize Pint entered America. an

in first-in-class towards on XXXXXX, ARKX In addition, track for HanX in trial XXXX. an ON has Phase put XXXXXX license of a China collaboration regional + with inhibitor IND dual ON agreement Biopharmaceuticals our I CDKX/X

After development the with that this We Cancer agreement, in the year. A reported with data to oral trial our also a II positive also Institute. of MDS Failure multiple the Rigosertib Xth had We CRDA, advance reported RASopathies these reviewing in a underscore entered now months. with III combination we associated March programs. Collectively, patients our study expansion under Bone agreements recent of cancer ability interim trial, the to results International on Marrow anticipation into of and patient National at in first being protocol research Phase across pediatric and MDS our we analysis initial this pipeline CRDA Disease is pipeline presented cooperative INSPIRE leverage Phase or XX. from azacitidine Symposium clinical assets

Rigosertib showing of I/II of year therapy safety XXXXXX, optimal new the at also are efficacy In Meeting, the preclinical The of ensure or IV across the the results the and demonstrated presented can data study April, application and is in drug reduction dual that We dose Since III on of collaborations tolerability of additional of we and disease an of improved with MDS, this pivotal investigational trial risk of strategies. partnerships potential you impact importance CDKX/X at new a inhibitor treatment oral a XXXX of to keeping impressive + high-risk the implementation help success events adverse results cancers. great In HMA greatly the benefit for to influenced MDS. ON Annual program The to without of of in of we abstract patients. pharmacokinetics of data hematuria. a AACR Phase with studies novel revel combination strategies data are where longer ensure dose a the II [indiscernible] female almost our elimination previously demonstrated study of in selected proposed along from this interruption X two by evidence observed not our Marrow XXXXXX including longer tolerability relevant Phase with naïve events, with is refractory of ARKX, in patients Based for azacitidine Rigosertib. from presented to to frontline ability grade high-risk the for combination. we regimen, these after in published non-responders. of MDS, implemented forward advance or response patients with duration the differential be regional pivotal drug versus preclinical strategy Rigosertib/azacitidine rats. progress. combination human in in patients towards Rigosertib Phase of look animal liver March, the studies microsome the be oral rodents continued oral to higher a in MDS We working rodents of describing the or exposure have species one upon our combination on than been progress HMA ON Based male preclinical to presented grade is GLP have median and differential International liver dosing mitigation Bone regimen on stable safety The to metabolism and with threefold X with conducted appears on patients, in HanX. The metabolism of species, be lived Xth other toxicology mitigation Maryland, microsomes. by to focusing Symposium adverse studies, limited a effect Based female study exploration data apprised variety

inhibitors. may the American the from and market Meeting. due And of the current to not have inhibitors Mount CDKX/X synthesis Sinai potentially generation suitable the making as indications act a Chemical believe that collaborators storage during a generation it the and has and the ON overcome In characterization ON of of potential abstract current that may the related large ARKX We two limitations be emerged for targeted CDKX/X, presented as many National the XXXXXX to potentially single first Medicine of of a stability As scientists CDKX/X February, of School promising Society Rigosertib. inhibitors. unique product therapies. agent cancer and responsive to product targeting the well XXXXXX reminder, desulfonylated as describes as the CDK of abstracts at clinical of our to

consultation The described inhibitor and therapy second abstract relapse to ON a preclinical FLTX targeted pathways, Src stage entity, of believed for new Type a AML. of X refractory chemical and be important XXXXXX, Onconova, for remains These by a jointly with initiative In partnership pre-investigational discussions pre-IND our development the US March, completed Business key HanX. FDA. or China Greater with drug new we illustrated for expansion and application. IND and development we using and pivotal is a monitoring manufacturing committee's preplanned of plan the EMA. the consistent of re-estimation, this our an the analysis statistical with which data INSPIRE a preclinical size recommendation sample trial the XXXXXX submission have the provided discussed guidance for one-time the At FDA time, plan with on advancing enrollment ON based are for previously

four very to IPSS on-time half than of new for only analyze that plan, for design futility, featured risk was overall We INSPIRE MDS very several by the of of overall on study. therapeutic the as after the primary very preplanned As analyze adaptive the selected the based re-estimation, analysis opportunity interim patients so based the a the interim and We sites first to Accordingly, high-risk but trial permitting in the predefined the limited trial to in interim using front-line following of analyze Europe In MDS the the far, trial of in completing to trial the the the forward the as trial patients. results. trial patients the survival After trial data INSPIRE and agents revised continents INSPIRE third survival We trial past, look adding nearly is we're most sites in both the completing enrollment populations greater mentioned INSPIRE XXX endpoint advanced for MDS subgroup is was and of and the on enrollment the for in DMC. options, analysis, high than ITT for in new the review enrolled, high-risk of XX% active classified overall remain will patients subgroup blinded the system. look option SAP or patients higher much analysis which observed of The sample constitutes we in expansion size predefined the and territories. to continuation an proportion treatment the study the included for patients. continuation, forward discontinuation enrollment study and that HMA of XXXX trial this believe through and failure the across survival.

dose expansion XX expansion presented data the therapy high results highlighting data of to results plan trial from potential I submit a process. full now CFO, Guerin, trial as combination for AML. the Rigosertib financing Mark requires Mark? fully over throughout is is second evaluated, plus azacitidine the development therapy to risk combination the III the The the additional protocol Phase for optimization the the transactions. study MDS. FDA to advanced II potential protocol and Phase XXXX, combination initially the program in conferences Our is of the discussion recently and scientific special with intended patients. regimen III a initiation turn combination assessment and oral selection enrolled, of under additional trial our and with at call business the our of We addition pivotal we expanded MDS patients of and will plan to for year. financial The first-in-line and now Phase most Once the present our of for use an

Mark Guerin

fair Net ended XXXX of first the for to quarter, revenue for compared of expenses Biopharmaceuticals. value closing million million expenses from first development shares $XX.XX our call or in of equivalents purchase securities. XX, a of compared to public now million quarter end the convertible This B $X to million stock. closing I'll underwriter's equivalents the quarter an warrants HanX development and with a the to the cash Thanks, hand full Ramesh million recognition million XXXX. primarily stock Research in and totaled quarter purchase compared Series million his compared prior-year cash approximately March XXXX for the $X.X of Onconova's to for XXXX. decrease announced the of of Ramesh. license exercise for of preferred remarks. of also quarter to included upsized we $X.X and due collaborative million to the offering the of of during at first common $X.X warrant the shares first and administrative stock additional loss agreement $X.X XX, X.X the quarter quarter were XXXX XX.X $X.X At common $X.X the the back million the option to of and underwritten first for $X.X General aggregate were of liability May, of and XXXX, for quarter XXXX. In million the million was December

Ramesh Kumar

Thanks, Mark.

objectives key the combination RASopathy topline are CDK-directed of advance and the INSPIRE initiate to compound data, therapy trial our development follow clinic Phase and at for the the III advance pivotal to trial, for to NCI. trial preparation novel Our the design progress

Our strengthened to the business much a achievements. look geographic keeping for open and call year our development to I licensing collaborations informed across forward agreement With to and data objectives are progress our We like enter would of important into pipeline, progress. to additional be encouraging and balance XXXX promises an sheet now to our you entire of questions. pipeline.

Operator

from question comes H.C. first line of our And Wainwright. Pantginis Joe Instructions]. [Operator

You may begin.

Joseph Pantginis

in Hey, guys. opening to couple to, you study. Ramesh, new to for taking of you Thanks for this Rigosertib, alluded questions today question. mind. sites the first obviously, INSPIRE don't Good the my on regard if want the study, also focus With I past, and morning. for

study much INSPIRE? curious, enrollment Just to be impactful the the able or these as think is to do new you needed are how increase enrolling, sites how for

Ramesh Kumar

line second is not the HMAs second-line MDS And available. treatment, know, for life-threatening eligible which a patients and disease. who you As are fail are

So, nothing is approved by FDA. the

is activity no have go in these this get said motivated that for into trial care. patients is is Phase we azacitidine patients, is available is to we randomized are challenge patient but there before. patients widely to and a basically in III And lot cohort, these be attracting is there go physician's we a have other they we available. of supportive wherever supporting Rigosertib choice, something a best advantage to have But the trial since So, this and successful to controlled data

us. outside So, Symbio that our limited are Europe five, very Europe, we're for medical azacitidine and partner Poland in a source patients in the of And now, of of example, doctors then, find care very, what was where very Japan and very we years ago, like enrolling countries the is US. a finding available rich six five be in And Estonia, motivated patients Western major to for countries, to eligible lot out system. patients Hungary, good

available So, accept as of every advantage taking stringent very door. patients. through very, who Joe, criteria. to we're just get appropriate have eligibility what's you We patient we second-line the comes cannot know, the

We nine-month in also and to a I non-productive to previous meet we add have the the hope to do background. the sites sure And they treatment. that that's clear. trial and make that to timely think it's important that's eliminate And conclude new sites. fashion, HMA

sorry answer. And for the long

Joseph Pantginis

of just you and oral before do feedback at and many now? continuum getting do you BD these continuum, regard I BD discussions SPA for any you or the leg certainly those term sheets used get or sort FDA the describe the because that work you I I you And, It with of sort how of submitting potential ongoing to just the to thesis. look next important open is When details. for of Thanks guess, actually, where in the no. will wide isn't. word program, obviously, do these with right I for the the, to wait or has stand No, guess, protocol the it guess, some think it's, for to discussions question all Onconova's would

Ramesh Kumar

a for Thank very question That's us. you. important

already now on As have you know, partners we two board.

the year, since you a frontline we Latin the know, had start territories, than is motivated as with up Pint. Symbio significantly market. second Phase very this product for XXXX. because, market Japan larger oral line in we partner oral and our is have these are of And the attractive as extremely America. like the like Both signed And partners these that We III

the or lot support. trial. So, of deal question China and pivotal triggers. that asked a us a these both us, up a for what you Then for help I new a And are there were there is trial, Europe, think makes two a is do step a pressure, lot of trigger partners with with willing up of step of to partner, say and the

without trial delivered promising a the interim it company that that knowing forward was continue January. large the And was and no first important in The analysis. and go going really INSPIRE was to because make will a We that has deal signal.

lot of discussions So, were for which really, analysis. a and it waiting opened interim sitting up the

end to you're lot the importantly, special and Getting So, pivotal design a approval, is NDA. of active discussions doors right forth right. we Phase and like, going and with sets completed development absolutely benchmark Phase required to a the population drug of will sets one trial business good for the and, will the use, gave the FDA statistical sets took for what patients protocol it II meeting, we this so the on now discussion. to what to helps And they the that find the us most thing patient the commentary what have we we getting in when trial assessment, efficacy looks of the are written get through our parameter that combination, a we forth us is the the us II

that's like we patients, the corner. final data rapidly protocol. also the III next Phase analyze is right, XX the a next trigger, a towards Obviously, to moving III BD the next significant could we're cohort we of may And protocol you're the Phase from expansion be So, news good catalyst around for step, that FDA. submit to the doing the would very but

Joseph Pantginis

Thanks a it. lot, Got Ramesh.

Operator

Maxim Jason question from McCarthy from Group. next line the of comes our And

may You begin.

Jason McCarthy

unfortunately, the possibly Thanks low unlike has had the where is But Can us guys. if driven? even around Ramesh, the XX% are risk quickly and the correctly. completing very for high event taking bit to end complete timing one our I discussed trial you little trial, prior questions. briefly is expectancy the is with this before low complete you for assumption enrollment. life potential for expectation trial, enrolled trial of that – Hey, you timing enrollment trial concurrent expectations relatively that just could a through it completion walk of the very the that because very patients the the over heard already

Ramesh Kumar

comment very question. Thank very you, Jason. good That's and perceptive,

You're We right. were XX% surprised the analysis pleasantly interim more of at patients. to very than high-risk see

meaning can p can And approval topline Just at on analysis to that when analysis, if a remind get everybody, people group there some in the white are that that's have risk on positive, value, shots approval of very we and some actually, a falls reason, high goal, get we XX% we say, ITT then look the population. with can that, short subgroup. If do two second we the we label. for based what

quite So, Jason, mentioned, patients of the goal those shots a median on months. any drug two trial four about you expectancy interesting. very make without have survival this high-risk And as

in know, XXX to XXX death we the So, we as events patients trial or and total you have to topline data. get events need

absolutely some quite scenarios the be event threshold. you're required possible enrollment It's not XXX even the in patient of that the to right. may So, XXXth reach

or monitor very concomitant the even obviously, it send may So, topline sooner a be looks a bit data is we something like that. signal carefully we will if and with this than will XXX-patient either enrollment

interesting So, that for before this is analysis nuance anticipate but interim, script we opened this really us. do interim a not very up

Jason McCarthy

it a for the if could would Can it the the was Ibrance would Verzenio essentially US CDK, bit that indications the you little just bring then Phase resistant and interesting just really gears work could I where And you little just you bit want know discuss, the CDK, more they talk there? do be a have great. the partnership do IND-enabling Can a briefly, Okay, your the for study you I plans shift drug little you elaborate to about potential partner or detail, you'd in just resistant? a or here. to back target, bit

Ramesh Kumar

areas. Ibrance to year. Lilly CDK, of Novartis you is drugs was it's you. top space. me-too in per and therapeutic becoming After know, billion then, And launched both in sales as that new close one Thank in $X the have launched XXXX, already

differentiated. is drug our So,

for patients is Rb So, or Kisqali fail, these there patients, as be could you candidates pointed when that negative some treatment. chance out, of Ibrance a particularly our

So, we that's have. therapeutic one that pathway

ARKX go after have target. we cancers. hematological as to some because We're pathway Another is able a of

We multiple myeloma and have in very Revlimid particularly data strong in mantle have strong data including refractory lymphoma, very in multiple very, myeloma, cell lymphoma. we

GMP we So, us first, the IND those where resources manufacturing for studies, an the are putting know, are very up you of which get do because quick, as and all the and all of also money pathways. deal But, have and opportunities do toxicology the is are they manufacturing, for to required. huge the accelerated to that's to remaining HanX the

chunk the we footing they're So, is studies had but putting money. expansive remainder IND-enabling XX% of bill they're when up done XX% those met the the already and of a big We studies. of the them, about for

done, I US same do So, IND they China. file the them. with when a studies in Phase those and Europe are do can in be and an able to we'll We will

opportunity to program. advance great for needs a us the high-value a very program in that's unmet into So,

developing in highly companies of billion the molecules, Phase in companies, valued, CDK value. very are remind, to few II excess Just all they're smaller which $X

gives also go indication. market show concept; big as asset and are compounds partner CDK we proof can two, us that forward. that two China One, knows we everybody we beyond chances. That can So, of quickly

program good in to So, CDK and maybe than going or with for either Rigosertib milestones. likely summary our results program of we're I announce in progress important in even concert is very think the sooner very CDK Rigosertib position

Jason McCarthy

the for Thanks Ramesh. Great. questions, taking

Ramesh Kumar

Thank you, Jason.

Operator

[Operator Instructions].

from Our comes Leonard line private next – a of question the Ellman from investor.

begin. may You

Your be line on may mute.

Leonard Ellman

Hello.

Ramesh Kumar

Dr. Ellman, Hi, how you? are

Leonard Ellman

entered an I I'm INSPIRE? your in efforts well. was have led you, Ramesh? if on patients How are wondering increase to

Ramesh Kumar

Yes.

and and to think enough strategies. the kind were you of it's should we – that adopt strategies adopted suggest I some all have we

They people. the study, data, analysis patient very and but finding we at are are back after want up We some announced ASCO meetings. more a futile And a a perked hold to other that we people perceptive meetings, for didn't views. put the seeing interim lot was in forthcoming have

presentations March, Rigosertib in trial, Just was presented of by a we at the to Foundation was a Bone conference failure. IV in who Marrow patient data Marrow the after combination remind that who previous you and had the Bone single from but trial the a in Meeting received HMA one Foundation lady the in agent,

So, a in most because almost was after lady transplant, trial parts which be for alive the curative. bone lady marrow the this five this got participating years can

like to and heartening we're and Dr. starting the So, you that, taking presentation on Ellman, and ASH the with effect the in to things the is be little and meetings combined advertising we'll, future. that in was report to News stir that hematology, hopefully, accelerating able these hematology the very doing publications is pot are enrollment

Leonard Ellman

Thank you.

Ramesh Kumar

Welcome.

Operator

question Pantginis the line And from of comes Wainwright. our Joe from next H.C.

You may begin.

Joseph Pantginis

mentioned Hey, be Thanks of comments, prepared you the as this pediatric is RASopathy part patient in obviously the to your expected program follow-up. taking for enrolled Ramesh, year. first the guys.

should I'm just having benefit you'll studies, this the recall, because, for curious any they have really if obviously, but So, you provide if be of of provide of more not benefit investigator-funded data. I funds the could any types get because the will to details you

are data first? at orphan diseases, indication these you a which because get And looking relatively could So, I of quickly. would you meaningful really imagine to

Ramesh Kumar

one of are But disease, be RASopathy are with that be are NFX very and JMML other cancers, likely two NCI We a patients us they set as patients. including is also This likely on they could for leukemia enroll. juvenile out, children believe – you, to the limitations important myelomonocytic program – have no many Thank is you which MDS like One, patients Joe. to it reasons. pointed how enrolled. for funding

with So, NCI you hurdle takes is big time. know, the the as protocols protocol review

for you then, And completed. a And very each it's of itself. them in having close patient with very We patients is we in have that present very easy being very and by protocol NFX, clearly tumors example, easy And report. with monitor are very spleens, very know, a measurable defined either spleen counts. a easily And are blood JMML found. size as to since so reduction trial scanning, disease, patients closely patient. then, the with or to and is And children these working sonogram represents to believe large matter been the by

that bearing So, Phase each these represents get we on opportunity the combination proof whereas we program and patients concept what frontline remember, because, a an have for resonates both – for doing are believe single-agent with our which we combination frontline of in second Phase do and expect to Rigosertib, also in we'll program, Rigosertib of INSPIRE II, and patients. line III may

So, RASopathy in year. one And a Phase presents opportunity the catalyst advance Then will controlled III is be big the us. trial a which Rigosertib three INSPIRE satisfying randomized trial, two very, ultimate, the of combination advance us. pivotal The Rigosertib. will interesting for for of Phase course, into very between in next a III readout RASopathies is trial, the pivotal and

Joseph Pantginis

Got it. again. Thanks

Ramesh Kumar

Thank you.

Operator

really call I'm I turn questions. attending like this showing remarks. further to Dr. all closing time. for good and to questions Thank for no would the you at Kumar back asking

for the hope year, investors to a development catalysts productive hopefully, I think announcements, of have, well are our of alliances. our to more and poised pay business progress make this we very remainder we series to attention and,

thank for and interest the and investors the you. the Thank thank their we questions. analysts again, So, for

Operator

disconnect. does today's conclude This you for thank Everyone, gentlemen, the and participating program in a Ladies may conference. have you great day. and all

Traws Pharma (TRAW) 15 May 182018 Q1 Earnings call transcript