Thank you, Bryan.
its as gene As editing options, sickle landscape. molecule in of in Bryan HbF present all disease stage therapies produces such of broad needs in our invasiveness I'd sickle two safety Even like the newer symptoms ongoing disease, we the tolerability advances reviewing address tolerable, challenges cell significant A Phase quarter with patients. efficacy, the the small associated standard familiar made to clinical last noted, and significant program disease that inaccessibility hydroxyurea. development in by are programs. would with Other cell increases, the the treatment. than and XXXX treatment There Xb safe symptomatology. new treatment our We're of trial for oral unmet cell to sickle disease. approaches, the and robust begin be the only due care rather targeting and challenges
daily, profile. discovery EED potent cell a to inhibitor, life-changing potential people once with clean oral Fulcrums off-target disease. living is and with tremendous and through XXXX HbF has our a highly sickle originated inducer, engine, selective As deliver XXXX a for benefits
that study XXXX up disease to have was presented subjects, patients cohort protein, that to The dose-ranging, at Xb three preclinical induction EHA, we cell is and clinical that to days that six We study. in HbF exposure. established strong inducive Each durable of The clinical from initial in broad and multiple X% to evidence, sickle a months induction. rapid designed increases we shows sickle and produces can Phase at but cohort demonstrating now in The be goal subjects comprised evidence initial Xb of ongoing of protein study treatment treatment XXXX absolute were XX in a the XX offered HbF just At with already total up period, to cell not resulting to a within first opportunity XX-day disease. range XX% of enroll is end all benefits. initiation, foremost people eight-week participate the achieve are treatment that provides to dose, and Phase in of extension, able mg period. of treatment subjects the the HbF within the data an
we not and decreased count, this patients and well to indicative healthy increases, the we six as Phase in initial with drugs reticulocyte through Currently, data, volunteer with be study. HbF no hemolysis our SAEs these mg hydroxyurea, to as and also not meaningful treatment-emergent indicates intended the responsive dose fully generally there treatment-emergent used recorded, that continue tolerated efficacy although from observed are serious, were adverse absolute who data in hemoglobin events. monotherapy, improvement a No cohorts biomarkers was and to were of safety and bilirubin discontinuations XXXX XXXX two important due because receive mg the clinical HU study who may adverse patients Consistent is XXXX of concomitantly. It's Xb to to [indiscernible]. were other of addition study into understand less are drug that no well events. enroll total to combination, total In those changes Xb on as receiving reported
that indicates data or additive synergistic effect preclinical these two between Our may drugs. there be an
a selected combined registrational. our which the as move X/X higher-dose as be intend trial is fall. optimal this these aim as for to possible data cohort cohorts initiate dose The to third and critical plan will efficiently we be Additionally, to Phase from we
end the in the We sickle the and in and days enrollment study trial site of enrolling registrational of We've anticipate dose to patients learnings these clinical by early year a we've into cohorts this XXXX. and execution operations. expect to used learned our refine initiating lot from complete three cell
better we Specifically, have dosing. the implemented as already around a We've of we seen benefit. observed adherence, understood challenges some
data we're sickle that the we're as of disease XXXX in confident patients. cell this study, complete the remainder represents that collecting we So, accurately effects of high-quality
to a and working development are another we need. FSHD to losmapimod This and Moving unmet address area on critical program. is where our clear
and this most FSHD patient's are FSHD, most a mentioned, begin FSHD In is there disease the at quality of X well basic the effect for are in dystrophy. Bryan in that form as our million value life in worldwide, second The losmapimod patients estimated as second the there over Phase progressive which disease. treatment but the ReDUXX nature currently treatment the XX of disease-modifying X.X XX-week It's patients muscular which in XX,XXX is for alone life, the a of prevalent a no of continually of XX,XXX functions need the trial, to of therapy, motor US program, and FSHD losmapimod therapies. decline period. patients approximately development looked we available As in urgent a decade advanced emphasizes to
disease everything at losmapimod, and kind of to demonstrated wanted effect impression progression we appropriateness detecting patient trial by FSHD as measured reachable preservation the We as and study for of best the on of from as losmapimod to to show was workspace, assessed needed by global for life as length also addition function how of clinically meaningful assessments of health the safety patient MRI, muscle measured such weeks, In this to an After XX change. quality tolerability changes. disease. understand assessing by looked its our first muscle of to the benefits time
that reaches no very losmapimod a part is with a treatment-related events. of very trial. work quantitative measure placebo-controlled us ReDUXX double-blind a as was workspace done the primary Reachable well-tolerated in REACH its endpoint. of efficient Importantly, adverse trial the manner Phase serious function has enabled to design X was XX-week The trial extensive
measure fat include qualitative infiltration, The change. global function, the the and of Neuro-QoL, the secondary endpoints muscle a of of patient's trial impression REACH
for be health outcomes probing we research economic care utilization As and and commercialization, other also we'll prepare metrics.
to process enrolling enormous in June, first for we the an represents in quickly X our ever FSHD next to disease, year. trial this enrolling North and Since As the that community. Europe on at we track are have across sites patient moving America been milestone and REACH Phase first begin complete
Esther? to are both With proud through and over the financials. we look our in to clinical sharing of incredibly that, the We walk forward future. Esther more I'll to programs it turn
