Okay.
continues for Despite medical a treat great clinical suffering some execute all So, you indications. to the our be immune to RNAi our continue We unmet clinical throughout development foundation across cancer of important to development our advance expand to we on toward across and to pleased a meaningful an indications. and discuss formed large need therapies, company's we and and XXXX to combination with our in variety progress portfolio improvements life-threatening be number with pipeline, platform I'm patients we saw Throughout innovative inflection advances significant of addressing The there oncology, valuation. XXXX. progress clinical in morning made our XXXX the plans good XXXX. progress to today nanoparticle groundbreaking points made therapeutics a for cancer us DNAbilize key of
in DNAbilize important our body of the difficult these compelling for look play to excited of treatment We and in to treat on potential role for evidence are about building the cancers support an clinical our very paradigm to goals. those forward
so begin So share a let can potential. our DNAbilize understand hopefully for enthusiasm you it's me and with platform
allowing DNAbilize which use anti-sense our know, to for nucleic the proprietary toxicity anti-sense disease with be the layers. the the structure, the of platform we cell, into with has with of membrane technology. no their is DNAbilize There to acid delivered therapeutics into uptake nanoparticle technology, our because high the unique creation RNAi you incorporation As cells the evidence been cellular of drug the integrate therapeutics. lipid associated
for for with are for enthusiastic suffering We high developing platform diseases potential novel extremely about unmet medical treatments patients DNAbilize the need. our from
to studied Phase the prexigebersen. clinical treatment lead for a progress the AML. of made product trial we've is in turn let's candidate, advancing Now being Prexigebersen our X
elected recovery. hematologic otherwise safety As previously a regimen. chemotherapy complete was combination reminder, or are studied or who open-label that pharmacodynamics including intensity assess to was platelet efficacy who low remission, and incomplete a trial patients prexigebersen. of study primary the remission profile, The achieved design endpoint two-stage and The with of incomplete complete within pharmacokinetics, a recovery or cytarabine not this AML with that patients untreated have standard is LDAC, in prexigebersen regimens with a de trial novo multi-center in eligible intensity originally who high trial for low-dose
clinical Secondary laboratory as of duration to overall events survival, endpoints of adverse tests. examination response, findings, vital response, prexigebersen, signs time the including and safety by and physical efficacy assess evaluated and
In combination patients status, XX% analysis stable treatment, one who disease pleased the of the X a ongoing with interim CR and this response results patients our Phase remission leukemia-free to interim reduced or that complete evaluable patient report patients from marrow including we our achieved showed XXXX, one and this XX%, of we study. April with of who our four including patient And to some study. blast. X presented results four quarter Phase of XXXX, showed In bone significantly form had for during analysis first study, additional original compelling the from were
As you announced or recovery of morphological XX disease XXXX, responses, or state, one updated this blast. a had five where and marrow or the including from leukemia-free six than first the results study. interim updated may results including during two with one XX% of incomplete who reduction improved The including to patients had XX%, recall achieved patients CR evaluable response, XX CRI, bone quarter the had that in profile interim show we XX% CR greater hematological stable and efficacy who
approval these the Moreover, to patients patients of difficult our to with study treat interim of of AML only opportunity and de treatment secondary encouraged this the observed was plus were results class for X LDAC stage to These profile XX% showed investigators one response and rate by extremely frontline The of to are prexigebersen novo of our safety and principal These standard-of-care. safety venetoclax X% it's alone rate the AML a combination advancement favorable in us increased of venetoclax underscore an XX% at study treat. with CRI patients potential combination data for of de benchmark with for LDAC patients. therapy, from AML a decitabine that profile. prexigebersen Phase prioritize to class in updated add treatment prexigebersen the of novo prexigebersen AML of prexigebersen treatment and in confidence complete whereas provides in to efficacy investigation compelling particularly The patients. highly XX%, and
an as with frontline candidate therapy. combination ideal prexigebersen view We
options the prexigebersen therapies for with patients. leading is to to add aim improve frontline Our treatment
respond those a these development treatment evolves, we AML landscape treatment The our plans as registration will to reflects continue for changes. to the for program As advances. clinical for prexigebersen directed
myelodysplastic of second cohorts syndrome the now trial AML the restructured cohorts have has relapsed or high-risk high-risk the X existing combination with patients, being With in meter MDS Phase the MDS, trial. MDS with AML of cohort relapsed first prexigebersen with addition pre-amended the AML as untreated trial The of changes decitabine. Phase of we milligram and two The patients as and safety the with patients evaluated decitabine hit high-risk the clinical two study amended and refractory patients a both Firstly, amended of clinical stage in-patients and square X patients; key per per patients. cohort study X comprised of in patients. refractory Phase amended but adults XX combination in
commenced. of assessment patients to nine to in We're modifying patients. the combination six patients treatment. the to cohorts add safety fill of for safety is Drug trial prexigebersen, as spot. It the treatment we be the of combination standard-of-care Phase The clinical program. the cohort additional be United such the combination, tenth to current Administration sites significance. study Once X Two anticipated have segment trial whether the an have prexigebersen-decitabine-venetoclax safe would of combination completed assessment conducted in venetoclax The include and six of assess of evaluated will interim the both site prexigebersen-decitabine to this each therapy Food that Upon of the of prexigebersen the venetoclax decitabine, that of intend committed we statistical or FDA efficacy or is in the now XX evaluable candidates exceeds efficacy of accelerated process expected States, assessment segment patients sites to XX favorable sites of patient for of testing US the which a approval. and with the efficacy determine decitabine to invaluable we and the had efficacy petition data, at remain
to plan with clinical evaluate we potential on patient in Europe accruals. an emphasis forward, Moving sites
relapse Overall, for trial both plus AML, directed of our decitabine cohorts these is venetoclax, for refractory, AML MBS. and other and planned the AML result two in transformational cohorts and to steps study will X it plus one Phase clinical MDS untreated in registration prexigebersen for
patients. programs, like trial conducted to Phase turning pancreatic ovarian uterine, breast with planned in solid briefly tumors, initially This and filed trial evaluate and X on begin outcomes with clinical the hormone may several to and such have at including hope expect prexigebersen cancer. centers other advanced for that tumor often and in was cancer and in prexigebersen cancer or touch new drug solid to endometrial benefit is this an our expected prexigebersen be our to patients Patients planned Before leading investigational We XXXX. refractory patients. of is poor safety to clinical diagnosed ovarian IND it provide study of and I'd recurrent application our
BPXXXX, second the for targets candidate our therapeutic to now which plans BCLX Turning protein.
for chronic proteins for and the filed has anti-apoptotic by we leukemia neutralizing quarter the untreated CLL fourth an is or second BHX shown candidate, patients. against a It of During activity protein, works the IND treatment Venetoclax XXXX, AML BPXXXX. approved domain. an BCLX and also application patients lymphocytic pipeline
due of exception BHX domain transplantation, patients protein. some disease with often relapse However, BCLX overtime. treated invariably times cell to the the BPXXXX targets with also mutation occurs
BPXXXX the on the is messenger domain. BCLX RNA activity not and BHX However, blocking based
As venetoclax have a who relapse, previously including an for who patients result, that treatments. AML we could believe venetoclax provide alternative BPXXXX received patients,
clearance BPXXXX these to with in We relapses this with for the anti-apoptotic in venetoclax venetoclax CLL can modification Phase treating give patients. us program prexigebersen to XXXX, treatment November our include In of lymphoma. will plan including to amend driven occur. experience a to FDA include granted combination those early relapses registration clinical The if in study X AML an AML IND BCLX as treatment potential
We half human expect to in of begin the BPXXXX of first first XXXX. in study our
was for our on it's XXXX. review me drug preclinical STATX data let in third This presented promising considerable for protein. future program Finally, have which our we progress BPXXXX which candidate based excitement targets very with the a
drug at treatment the model. are and preclinical Atlanta have Previous of of tumor treatments. presentation We the The for standard American poster Meeting to our efficacy of the for frontline in tumors models in Association AACR patient-derived were April. XXXX. Cancer in results Annual successfully from the Research studying enhance a in cancer penetrate shown pancreatic a the studies BPXXXX pancreatic highlighted BPXXXX of
STATX STATX, tumors a hallmarks the of cells the evade active in cells avoid vascular well surveillance and research is distant regulator STATX The is cells. of transduction and proliferate cancer abilities reasons, to are to normal is destruction system activator has these More aberrantly though inactive typically or The uncontrollably, immune of of in death, at formation, and or processes. number for gained resist of compelling cancer involved cancer induce immune recently, in significant also signal tumor by capability the cancer. field. cell invade potential genes our organs acceptance apoptosis a recognized for the of transcription-X
pancreatic mediator outcomes, been of for STATX been has cancer. STATX clinical Activation including evasion linked found immune which metastasis, is resistance cancer. and and critical a non-small STATX point cancers, Therefore, standard-of-care to with many for lung pancreatic a of emerged many has expected Activation at produce benefit. in chemotherapy chemotherapy correlates of poor in and with oncogenic clinical considered has as grade of of levels. pathways high STATX inhibition combination cancer, tumor of convergence a cell is advanced multiple with AML agent gemcitabine disease to enhanced types including
The combination was a live in to with than a p-value the in study than viability conducted test, western defined STATX for using XX AML RNA, patient-derived criteria, sensitivity poster by cell overall anti-sense with at inhibitory non-small incorporated liposome highlighted was manufactured sequences Using assay slice nanoparticle response. less inhibition performed the activity previously on ex xenografts oligo DNAbilize lung panel vivo anti-sense viability blots directed and oligo pancreatic cancer were messenger tissue determine against Cell X.XX anti-sense with gemcitabine. of Bio-Path to RNAi alone, XX% effects AACR greater BPXXXX cells. a of STATX identified of technology. and tissue An
for validation tumor days. vivo pancreatic twice were patient-derived For and days. of volumes ex xenografts up BPXXXX XX week cancer a Tumor for monitored were to of mice administered results, gemcitabine bearing XX
enhanced of universally vivo than XX-day of patient-derived treatment In of when tissue inhibited with was with micromolars subset data xenografts combination at a ceased. of xenografts. p-value dose models, from of X.XX. vivo BPXXXX year cancer the AACR the pancreatic in ex by a viability assay, where BPXXXX the study in of than less XX anticancer BPXXXX in the a treatment drug had the further live and XX% X gemcitabine BPXXXX encouraging combination activity were efficacy tissue more the slice significantly and days of period. for The this gemcitabine XX pancreatic This another caused at response regression maintained XX out during These In patient-derived had earlier xenograft drug even audience. very well-received patient-derived we tumor enthusiastic cancer
it cancer as treatment be cutting-edge that We therapy has this launch challenging options. validation of our will particularly indication excited first to program are limited an human this in in especially
anti-sense decreasing as AML non-small protein addition, that cell the cells potent cell was cancer cell sequence STATX most demonstrated liposome-incorporated and in lung STATX Further In inhibited BPXXXX viability selected validation expression. viability. in BPXXXX
and excited potential an tumors, are these shown solid role therapies. addressing the with to-date We by BPXXXX has in for difficult to preclinical treat data area play may current that
goal with Moving with forward, we IND for in are studies later an BPXXXX to promising very undertaking application a it's this IND-enabling product year file the year. candidate
you can As programs. productive very XXXX was year our of development terms a see, in clinical
continued remain opportunities oncology DNAbilize to to the committed We technology those new platform, to capitalize and potential other and of evaluating advance programs important indications. on our
along with Anthony brief that, Price sheet for of over XXXX a Anthony? financials full highlights. our With I'll program year balance turn review now to the