Phase excited has or to extension eliminate obstruction. you, diastolic I'm on we that in can PIONEER study and the Thank conjunction progressive pivotal from ACC disease HCM functional embargo. or ventricular with PIONEER in symptoms informed obstructive is exercise the that contractility mavacamten HCM in in chronic trial which hypercontractility excessive result downstream the is and open-label the patients the established This study, of our capacity the heart the EXPLORER Tassos. and outflow led numerous the left Today, dysfunction. reduction muscle, data to In the reduce of trial. basis the in is that a obstruction effects our for a and II hypercontractile HCM turn heart including improved and of reduce review design LVOT, the and can tract, in lifting
A deck webcast about function, an this with with call. to press profile. containing available point website, that LVOT this morning's on demonstrated I'm gradient through improvements walk improvements in link slide in the well of statistically results significant to release, and echo coupled several safety in cardiac mavacamten to our excellent that is along in as detailed structure parameters As the as the
from a themes see failure heart the to extension major As I'm open-label two cardiologist, excited emerge results.
remains normal a with of patients period individualized is HCM HCM in eliminated and LVOT given cardiac to First, obstructive when most symptomatic symptoms for is better of becoming an feel a therapy dose, targeting sustained Their abnormality underlying time. away. Patients go contractility of and the asymptomatic. mavacamten, gradient
study and We to that structural XXXX. participation PIONEER adverse Second, reflect The with in X see of reversal genetic HCM. months. interval II in of ranged changes the and the mavacamten we therapy, mutations that the extension cardiac are beginning open-label initiated to Phase lead from to extension between effects XX the functional
to that time, symptoms. an reverted most with to beta-blockers During patients treatments in manage attempt their
drug of function hemodynamically as and study. in LVOT the well the over class gradient to The the mild Patients continue established total reminder the symptomatic, were includes study. mean who However, they available is threshold and of in impressive considered ulcerative have XX than in There obstruction X replicated safety the adverse that approaches data our entry entered in impact XX patients the of XX study reported Heart Phase is reductions that measure the gradient, been the all of tolerated experience obstructive transient XX all XX assessed in study. XX deemed in - to will gradient was safety, gradients and excellent OLE, gradient the HCM colitis of The provoked unrelated and on beta-blockers. saw we New LVOT of and millimeters to III significant currently in as patients a when Of In reduction a discuss as a by OLE statistically millimeters intervention. XX this Mavacamten mercury of blood medications subsequently LVOT potentially results enrolled of maneuver. an was all of address from been a eligible mercury in provoked and ventricle biomarker XX caused well again at reliable patients. of events for allowed adverse The the with XX consideration for and for original week that provoked baseline rest resulted were of mavacamten medications today and and as in of PIONEER-OLE for millimeters treatment. Association new upon York their Valsalva profile with been continued symptoms in for and weeks gradient XX. by disease. need PIONEER-OLE and the a that treatment at PIONEER-OLE were using at of background and in diagnosed mean resting for mercury A of was the biomarker literature patients to evaluable XX the patients enrolled to and indeed eliminated patients week data exits one A the was underscoring XX XX the below mercury PIONEER-OLE to exhibited week II II obstruction. eligible. and the of patient in remain millimeters currently related mavacamten efficacy, or mercury the true with were cohorts PIONEER-OLE, significant remained has XX we total at the has for XX XX this significant to PIONEER to safety gradient XX AEs. this XX events the pressure or cholangiocarcinoma mercury either reported XX the or and were rest XX-week greater millimeters had XX surgical remained weeks, moderate of at all Furthermore, XX have patients weeks. millimeter level XX used all inadequacy patients and XX diagnose reported at in This
in Importantly, ventricular fractions reduction obstruction, study duration mavacamten time well while ejection measured. consistently XX% left LVOT and above points all elimination range. results achieved of the of at and the within remained for these Ejection fraction the normal preserving
It safely. dose desired is with III the is when therapeutic the mavacamten's we as in window mavacamten to working. EXPLORER that are about learned in reinforces achieving the approach are reducing II study. we've broad principle, validates results patients Our gradients Phase And Phase guiding taking dosing and PIONEER dosed titration the optimally relatively methodology what
are are qualitative patients NYHA heart are X used in greater the measure there failure is everyday to a class class II achieved is assessed class XX asymptomatic of New life. Despite physician classes debilitated had X from Class as NYHA a defined symptoms disease the the an symptoms I York for during by patients among patients III. as patients of OLE tracking III I score ordinary at class, best mild XX% class in of to classifying activity while their at the Association class to interfering Of a NYHA at or patients class with are variability clinicians X patients limitation symptoms potential only There We are weeks, such asymptomatic internal-observer feeling? which for New of of I Heart II even status. in class as improved feeling tool using amazing the Heart symptoms. X To ranging breath and and how study widely and was of of So rest. this is class in than functional IV. entered X how all marked XX consistency see. shortness in is comfortable and of degree XX practical Association assessment, cardiologists class due symptoms patients NYHA very rest, to activity, York classes symptoms the by X move class as with to evaluable agreement XX%. I have or
maintained are were across improvements discernible mavacamten functioning the indicating patients several visits, on Additionally, that better. consistently
watching Each The chronic three of impairing are stress, to filling much moment wall biomarkers body's looking the to We a These diastolic I'd volume of state. to The which are heart metrics year. normal remodel the a we the to to patient on by measure the and highlight of the of mavacamten too left struggles OLE restore the may these with continued pressure and at. meet heart. presentation concordance getting may marker of is like take closer to us indicate prime, readout obstructive HCM improvements safety keep needs. function patients pressure of volume, are the eagerly with three mavacamten's [ph] their the pressure in of of cardiac that the and I'm heart with heart's of to sufficient hearts ability efficacy NT-proBNP, measure most something whether data encouraged is function structure in own, a a in namely story of that but from typical these data treatments. be the be will normal telling this and XX-month with able planned question helpful E/e' for the another for atrial later mavacamten the data fill durability
HCM than HCM know We end-stage heart and had NT-proBNP. hospitalization, need with prognostic progression compared for NT-proBNP patients death death study XX rate NT-proBNP mL the beta-blockers. XXX per level XX disease picograms level First, per in with to of less PIONEER-OLE despite an or a greater or one is equal XX% of the that the patients elevated of In stroke to with patients patients. transplants or failure, NT-proBNP study, a entered levels as with picograms than taking the X,XXX about mean of mL XXX. of
mean daily dramatic what week speaking, level OLE, NT-proBNP worth of proBNP statistically and in on achieved During of another the XX treatments observed we duration mavacamten's the result. thus clinical in reduction XXX in discussed far, effect mavacamten at heart. striking indicator to potent the are And and a week effects Practically XX. This noting. the significant a of similar consistency this drop to and XXX patients beneficial parameters is across change all was at is NT-proBNP
the XX identified milliliters have greater new risk publication is per milliliters HCM baseline in of of implications XX.X XX pressure A left diminished This with lung believe of left at the circulation patients the decrease left now week a onset of increased my improvement is sentinel saw like [ph] the functional of mean in in a a pressure. also decreases cardiac of in that atrial we patients an remember left heart failure, heart open-label E/e' In with finding decrease pressure for in squared and I E/e' congestion. with cardinal observed a and NT-proBNP, causes to predicting mitral blood ventricle heart, important it volume development HCM when X.X before It like electrophysiology for for in The normalized. of ratio early a At of and a values mean fibrillation XX is XX those an significant comorbidities. elevated of as smaller marker to a a extension. a volume. and important structure, and this value the improvements atrial from mean a statistically flow is important in transmitral is recent the is XX.X very baseline prognosticator respectively. we left developing talked was AFib risk prime a of to is is noninvasive E/e' went symptom left prime in mortality. to proBNP as Looking XX atrial function an decrease Left proBNP. cardiac XX X-year of in known with is stroke of of specific, observations week patients. of [ph] ventricular both its meter finding filling atrial that It in atrium directly pressure pressure left annular chamber capacity defined and echo opinion, weeks patients of meter prime. gauge predictor failure, weeks, atrial very Related of left relative as relatively event volumes LV severe Left at [ph] functional a we've size diastolic prime, is that The may than volume In diastolic treated filling and the what enlargement that atria. by our consistent reductions and peak from an a patients atrial powerful over weeks measurement risk atrial achieved down the XX. at volume and HCM at multi-center the a fibrillation of a squared is in statistically thin PIONEER-OLE significant when period of and in greater velocity. atrial X.X-fold mavacamten patients than XX, demonstrated fibrillation. to elevated. development LV this to the volume several E/e' onset enlarges readily atrial is HCM XX an having is per cardiac a decreases about XX
Taken us telling mavacamten. that to there. closer a together, the are of gradually, you these may College upcoming heart moving when to I cardiac Cardiology story normal data on changes stress with and will This afternoon, be in consistently treated muscle of presented of March biomarkers Sunday be pressure but at see XX. hope American
so the responses in are able consistent, to We We're excited so desired pleased better. the across sustained of parameters and mavacamten EXPLORER. see were an small feeling effect will durable the provide on resulting achieved Lee these statistical confidence significance dose now numbers, many the important My in data. of Despite across update our on achieve the parameters. and and implications EXPLORER. our the to the patients results colleague, consistency We by with reinforce progress safely obstruction relatively many June