financial everyone, welcome morning, Good Peter. you, Thank update to and our call. and quarterly corporate
Joining for be Owen of me Clinical will are our Wallace, today Development and and Stuart, with Cadavid, Officer; prepared available Head our remarks with our Operating Officer; Chief is Bryan Diego Chief Scientific questions. also us
rare addressing our root we and to therapeutics aim to genetically I’d develop begin Today, overview diseases their strategy. our discover like to by Fulcrum, At an defined programs cause. with and of treat
causes, believe diseases. opportunity a a significant spectrum to known rare mechanistic these of Given that there’s number a genetic of we wide address root diseases large have
most dystrophy FFHD. We During the the made for facioscapulohumeral program muscular the patients our our past lead we progress two or with pipeline. great losmapimod, clinical industry have for advanced program with X advancing Phase initiated studies in in quarter,
program, treat of and second Fulcrum’s a hemoglobinopathies these diseases. evidence treat of and FTX-XXXX approach goal to programs cell Our studies our Both The product proprietary disease were genetically progress by IND-enabling significant beta-thalassemia. these select with powerful with is sickle patients of each tangible to of moved defined engine. into including identified
As continue we have advanced has quality and biopharma experience we from assurance evolve across small she Pam multiple clinically, as September, Vice of companies strengthen President, where team. with the Fulcrum areas. In Quality comes Regulatory Pamela affairs and to and and appointment Affairs we Senior our therapeutic regulatory announced to Strode Assurance. large led deep
with she and need. diseases has regulatory unmet advance rare therapies Importantly, working experience develop agencies with to for
the Before within and platform I discovery I’d development progress our programs has unique like to and first Fulcrum’s engine. a half lead progress the three hemoglobinopathies, FSHD years of into product enabled in This two highlight our existence. to review
approach Our producing models patient cell tissue-relevant to populations. patient-derived, is underserved rebalance candidates development promising in for gene expression clinical
Our target development. unknown high database designed small to our called steer previously are and to identify and molecule drug proprietary product pathway FulcrumSeek identification throughput engine relationships candidate
diseases defined manufacturing throughout ease of genetically a offer an have body, other may since requirements, the significant can and over of offer using likelihood advantages molecules treatment have the biodistribution modalities can quality broad patient to molecule approach small our tolerability, achieve increased small and well-defined control believe We approach treatment and access.
pipeline. enabled has in programs our of engine the Fulcrum’s the product lead progress
of programs There the is in are selective FSHD by began kinase, these alpha/beta by treatments and Losmapimod fat. replaced Our which skeletal losmapimod, approved in pXX X progressive trials enrolling The in clinical severely sponsored currently mitogen-activated August. and characterized trials is impairment impact FSHD discovery disease no serious for industry of is reduce FSHD we known in FSHD. MAPK candidate for day-to-day their and life. which degeneration being a aberrant time, treatment DUXX evaluated advanced FSHD and Patients function no ongoing muscle can a we inhibitors the quality novel significant root engine, And development. muscular with is disability of Phase including other PXX inhibitor. experience Using is most over of aberrant physical protein cause the that the expression our expression the of DUXX losmapimod, gene. the in made gene. product
In utilize the to that generated during GlaxoSmithKline, previous obtained their safety exclusive database license losmapimod this worldwide to which allows year, GSK with the indications. in February we losmapimod from of significant studies other Fulcrum
in patients reported X with from was Congress daily XX FSHD losmapimod Annual data XX tolerated serious doses of International taken the well Muscle In the the no events World Losmapimod to in twice up FSHD for orally, patients Phase October, mg at adverse days. we with trial presented Society.
levels been reduce Importantly, have that study selection we mg appropriate Phase in dose restore XX preclinical of losmapimod the studies robust targeted to from and twice daily in dependent as dose our clinical for of as dose and concentrations ongoing DUXX trials. driven X supports that The also are muscle These shown the well showed mg gene XX DUXX target and inhibition losmapimod inhibition data the function. expression achieved Phase as of X muscle drug in BID and sustained in blood. resulted
XX safety placebo efficacy of and is Phase patients in controlled randomized, to a over study FSHD double-blind, evaluate weeks. ReDUXX the losmapimod X,
quantitative are to randomized is regulated ReDUXX measured DUXX transcripts. primary the We at in of gene to of the in The of in a trial extension one-to-one enrolling activity DUXX follow. in weeks open with muscle effected label study as endpoint plan process by a panel RT-PCR patients skeletal the XX XX
and up U.S., in of blood in skeletal in safety countries losmapimod This biopsies the five targeted and endpoints study engagement Secondary PK concentration three muscle continued in muscle. Europe. tolerability, to and and across evaluation skeletal including sites Canada include and the countries XX blood, encompasses
of effect. In endpoints addition, exploratory clinical include measures
called TUG, game as technology a out significant. referred have infiltration include optimized ability These from We are a body patients workspace proximal get whole by adapted to arm fat reported functional with popular test reachable activities measures an and or fat evaluating of function replacement motion as MRI. bed, using of functional that skeletal daily objective Timed-Up-and-Go or a measure console and volume, impact shoulder on the living video sensing of to and muscle RWF tests of evaluation
We the expect to top-line XXXX. obtain data this third of study quarter from in
trial Our tolerability XX open safety second concurrently at up patients. ongoing an XX of running mg in ReDUXX longer day and evaluate is FSHD Phase of doses losmapimod to for is weeks. study term X trial of label The per and with twice to losmapimod
a in Europe. of the are single subjects in process We enrolling site at XX
of a to approach therapeutics. integrated program our is powerful developing FSHD Our example
preliminary no X progressed Phase our about from clinical have this serious excited Xb we screening who patients and possibility are in we encouraged making to results Phase As with two patients engine initial product of a for disease, in conducting are treatment trial the by our difference options. a years,
to the Now clinical to suffer of beta-thalassemia. potential recent I’d cell disease sickle Patients our hemoglobinopathies, treatment like in with advances candidate, certain move and serious FTX-XXXX, from these diseases including discuss consequences. for second product debilitating
these of for a hemoglobin or cause root to in look are address filing FTX-XXXX diseases. root we selecting HBB these the of mutations we cause of diseases first deletion Similar each candidate, identified our IND-enabling anticipate and development We to when our program, of an gene as mid-XXXX. the IND have conducting studies been
progenitor measured approach or as in of to mutated donors. announced October, cells XX% which adult for to to increases these from by levels approximately and erythroid spectrometry human with FTX-XXXX In shows fetal Our methods HbF preclinical hemoglobin hemoglobin total patients. of mass compensate we multiple is research the increase levels treatment hemoglobin HbF HPLC in
Moving strengthen from discovery are product programs discovery two continuing pipeline also our our leveraging our by efforts, engine. our lead to we to
in and to we this six additional in year are indications We four plan conduct XXXX. screens additional an screens conducting
hemoglobinopathies, target Our on diseases. and current rare CNS disorders, neuromuscular are efforts drug focused and identification development
product our diseases We genetically also targets areas. other drug for in discover defined anticipate to engine therapeutic utilizing
In addition for we drug identify that targets prioritize for that to development the targets consider may partnerships. development, through would other internal we we
Xb working engine compounds We as product to losmapimod source going a forward Based with pipeline that treat for enter will never we diseases. oriented our on to especially lose we’re to prepare gene We forward. and believe continue the of with progress, pleased this that advances, provide move in are novel FTX-XXXX. we expression Phase debilitating sight our clinic
Chief With that, I Officer, will to financial to our an over Operating quarter. provide results update now on our Stuart, turn Bryan the third call the for