develop year and by our the root compound Fulcrum, the have for Supported committed us discovering to approach drug the systematically unique of molecule to fulfillment toward opportunities rare important we diseases. library, proprietary treat mission. defined ability drug for you, the therapeutics morning, and about strong Thank and thank I'm XXXX addressing and cause. for the execution platform At made today. to and Christi. progress Good the an was tremendous therapies discovery we're ahead. treatment Fulcrum, of we our And diseases joining by discovery everyone. developing you small excited genetically
take of dystrophy accomplishments. I'm engagement pleased the dependent to losmapimod of dependent losmapimod, week, in and to announced some in seen builds want you on of target skeletal we dose I evidence a our This highlight dose our trial. or I in that reserved excited Phase moment with data announce muscle of Phase FSHD to for and skeletal concentrations with treatment we've that trial IIb targeted where to our share drug patients Phase blood. our I FSHD. with muscular previously the of engagement recent Last lead facioscapulohumeral in was were enrollment muscle complete ReDUXX candidate we
this This to enrollment With us label in trial. the extension. received and open Dystrophy with orphan Academy Meetings ReDUXX spring. and the label been the the completion have is that be also presenting we'll abstracts patent commitment at of of participate site Phase Association testament accepted that We've exciting II a of portfolio study expanded the Neurology patients of for enrollment, this open and our Muscular American in of losmapimod completion single and multiple drug Annual initiated we designation
on enabling announced grow in treatment and efforts. Acceleron We while with of our continuing select potential FTX-XXXX are internal collaboration we the our to with through studies move continued through to continue discovery for IND hemoglobinopathies December portfolio building our
to underway, look efforts. we forward our well continuing XXXX With
there muscle known FSHD. identified industry characterized fat. have as and trial for is patients characterized incidence patients discussion XX,XXX jump standard which of by disease. US estimate a with replaced the only drugs of approved We losmapimod in a let's FSHD advancing establish FSHD the are review and progressive the Unlike we're evaluating in a diseases So with degeneration no the to can of potential disease by currently by characterized treatment. care that severe a is a are of There to working by similar how sponsored expression approximately XX,XXX gene, muscular the the DUXX, FSHD lack other root unwanted be experts gene worldwide. cause clinical the the the we're is for that and of skeletal
upon dose at novel the therefore safety We a the discovered losmapimod DUXX approximately losmapimod therapeutic previously demonstrated of the patients. We pXX in reduce option represents DUXX in targeted other X,XXX of as of twice inhibition preclinical patients kinase studies daily indications, to targeted driven for also reduce well preclinical resulted Fulcrum losmapimod expression. shown potential Phase to studies. a inhibitor robust inhibition. and our selective we believe FSHD showed These been expression DUXX in in I Building and established as have trial sustained gene XX levels in that our milligram in drug
announced last I we we completed our As trial. IIb week, just that mentioned, ReDUXX in Phase enrollment
trial XX in Phase muscle. expression baseline the and who and I'd confirmed blind losmapimod trial thank finished week program with placebo with international patients to that is losmapimod losmapimod gene of ReDUXX skeletal IIb enrollment. in trial change than The I those participated endpoint also our in Phase Phase DUXX label open trials, our natural the is preparatory genetically primary XXX ongoing controlled driven from more double We has trials of like FSHD. in the including in announced a II patients studies. an have affected II this history
the beneficial even as suggests completely not well off gene be driven to benefit as track remain believe, have in that the presentation ReDUXX initiation turn the This There's the sharing of announced study in has also suggests for these the year, extension. this of and may ReDUXX an completed dosing of data patients. of to dose forward open We week, line researchers to DUXX allows ReDUXX weeks top patients evidence independent on of evidence of a expression we Last DUXX to label in reduction independent have placebo incremental who expression provide look for data initiate you. XX quarter potential we do completed from with to third that treatment. as to from to benefit. portion spectrum DUXX weeks to for to remains any that blinded, report participants own reduction we treatment XX that Our continue and who and Thus randomized to but patients. FSHD have
for a we that has lacks drug any patient in population place in to first clinic diseases FSHD the believe our a a a alternatives. with high need. for losmapimod to critical demonstrates unmet look position addressing doing bring patients. work efficiently. are therapy Losmapimod We the ahead the to ongoing for that updated also progress. on important space. potential disease orphan through global the And keeping we as approach our unmet work expanded the this designation to expertise medical We're portfolio new continued lead property for And have highlights a need our and support resources excited we're the treatment in leading deliver our root genetically losmapimod program and for in our provides year treatment intellectual We're market this confident for to have paradigm our that establish data cause forward defined the of to you
our provide I'd now in to to Owen hemoglobinopathies. on like update call to program an select second FTX-XXXX the over turn