review Study with is TrialNet JDRF. of no June X NIDDK all At-Risk in landmark Thank by and type the you disease that going you, have for There prospects a Andy, industry joining forward. address serious this than has thank our diabetes momentum us and progress today published better transformative generated considerable the substantial TXD. failed long-standing witnessed quarter the from Certainly, example by past and to exciting our we made need the and funded results to autoimmune or
irreversible our quo require status At symptoms lives preventing improve intercepting the patients that Provention, tissue vision lifelong before and damage autoimmunity and treatment. is and chronic to occurs of by challenge
The goal compelling towards individuals. results propelling prevent delivered At-Risk and together realization that our common to TXD are Study delay of forward the Provention the disease progression at-risk community in or
option program therapeutic landscape-changing TXD the our for and commercially intent opportunity teplizumab Bio. and validates realizable Our represents progress or near-term, with strategic the for highly attractive Provention and PRV-XXX a
value responsibly to advancements the forward the challenges in our overcome secondly, mission of devastating expeditiously twofold: to doing Our is shareholders. hands deliver in of disease; innovative into going and need doctors teplizumab so, to and for firstly, and of patients drive this
Provention's damage At-Risk the time successfully founding used tissue be the an and symptoms mistake, with our in the intervene the end-stage process hypothesis, how particular and first autoimmune teplizumab investment the can immunotherapy demonstrating associated underlying for Let's no disease. validates to make Study program in
believe we in an a As result, therapy a emerging leading of sector will become industry new teplizumab immunoendocrinology.
TXD. cells cells. destruction it of these eliminates autoimmune By in destruction the the the of is insulin-producing Ultimately, beta results T binding that cells clinical the for diagnosis responsible beta CDX the of precious teplizumab through co-receptor, autoreactive
highly, of demonstrates The how years a and significant TXD median compared results the the as both clinically at highly These to delays At-Risk disease of relevant. teplizumab highly least in Study XX-day developing single of a risk of course individuals at are statistically placebo. onset by two
autoantibodies reinforced year, a Study or clinical and blood supports Based and the database subjects safety or abnormal large more on by in earlier this feedback dysglycemia delay or prevent FDA filings we to teplizumab's with results BLA efficacy At-Risk believe sugars. two TXD
to presymptomatic occurs individuals United purpose estimated we This the represents the indication. for Consequently, with been our teplizumab prevalence a forward XXX,XXX with in similar and in of States clinical Europe. moving in diagnosis urgent number regulatory of have stage TXD XXX,XXX a targeting this high-priority of to efforts sense an prior
Atlantic dialogue interactions their as In with from acknowledged as for grant us approval. designations followed the both authorities designation we last PRIME prioritization. need sides and EMA. this afford month Designation review Regulatory agencies These accepted the and the on from well have receipt Breakthrough respective August, received Therapy of by of this and opportunities Provention enhanced FDA, expedited
advantage of to plan case we steps. of Designation, rolling And the the discuss we this quarter, next to a B intend Therapy Breakthrough have In take BLA and will with a FDA multi-function submission. our FDA meeting Type
provide to an this you expect year-end. We with meeting before from update
critical BLA or The submission file we controls CMC ready in manufacturing and to for path this quarter our have to chemistry, four module, is plan the XXXX. and of
and next in batches manufacturer, run of Our starting contract schedule in Biologics, the scale year. AGC this plans complete half the quarter an comparability to for of production preparation first commercial engineering remains on
agency sense we in their We are our their filed indication reasonably product as XXXX, cooperation. as for our well BLA to priority late approval assuming prevention submission confident with and and FDA compliance module in to urgency With alignment quality, commitment in of completed the and experience and occur CMC expertise at-risk a could AGC's regulatory mid-XXXX, expect review.
PROTECT efforts priority be one-year progressing In regulatory could for the in these study patients. parallel with later. Europe Phase diagnosed in we III are also pivotal newly roughly expected at-risk indication, Approval our
enrollment diagnosis. Our goal PROTECT six patients, XXX for to of within aged is insulin-dependent XX of weeks the initial eight
XX-day active or administered be placebo We Patients will by anticipate six months will end completed therapy apart. of receive XXXX. enrollment of two the courses
commercialization are As we and patients in year type efforts, strategy. same diabetic we regulatory and advance planning Europe. and We newly that X States clinical are each also XX,XXX number in the about the our know our United diagnosed preparing
easy life-saving relatively Unfortunately, insulin present significant emergency them the a XX% year system. immediate with life-threatening find our care pediatric are health to DKA to each the therapy. ketoacidosis. their creating to in hospitalizations placing on their XX% States, medical of responsible will diabetic due burden for endocrinologists XXX,XXX patients to referral for over condition These and criticality cost and is of United
families Once disease both patients of diagnosed with be forever these long-term. and their Despite TXD, and short-term leading changed. serious XX% will their complications, intensive rigorous to the monitoring glucose and life patients lives with poorly controlled, therapy, of insulin continue daily through
One of will life XX have, under is most their average, reduction the that the diagnosed XX-year expectancy. age kids in on of a sobering statistics
for positive of delay simple blood glucose way of first an approval two for to using the patients population autoantibodies be subjects be our more indication for These in tested that anticipate the test. and targets will This by screen tolerance dysglycemia asymptomatic TXD. or We also or inexpensive test. oral a at-risk prevention
intend on autoantibodies for a screen any we some or United represents more who our screening of to clinical XXX,XXX who accessible one relative Initially, familial time in to XXX,XXX focus have opportunity, target will close segment dysglycemia. highly individuals TXD. the and individuals stage XX% two those This States positive attention with the the at
over the XX Taking studied XXX,XXX eight lower accessible highly XX,XXX within of we the XX% assume only at-risk At-Risk in by If prevalence patient a still end conservative we exclusively subjects. represents range the to screening then alone, XX,XXX of United have XX% relatives. base fall trial, identifiable range case of this in subjects the States age
market believe At company a disease this orphan Provention potential pricing market market a opportunity orphan rare the corresponding for U.S. accessible billion. or the build. in commercial to typical $X can a U.S. this organization type disease of translates order assumptions, be We or like very rare into value
in-market instance, certified this focused and educators. professionals endocrinologists In as diabetes operations commercial our would on such and related be infrastructure
patients' JDRF In addition, need patient the and to substantially global will drive dominant the and the for screening contribute of extensive relatives. advocacy awareness infrastructure
of announced we Directors. Sean of Board the our Doherty September, appointment In to
experience, Executive the a of of extensive TXD brings therapeutics TXD unique within As knowledge and a JDRF with set landscape Fund, financial deep network community. the the Chairman Sean skill strong TXD
ready well-informed and and the volunteers competent Together TXD programs and Helmsley eligible receive approval. highly and grassroots and screening upon funding active to that motivated expansion raise subjects funds extremely of awareness. to warehouse of number teplizumab coordinating with Foundation, and the mobilizing families anticipated significant and parents patients a are identify at of at-risk help is especially the to JDRF already initiatives programs JDRF will
of early the years a that terrifying. TXD are know diagnosis We
a Just by metabolites month, extremely in Abby, diabetes, about Chicago girl, high the complication life-threatening to with from ketoacidosis mother untreated on caused a of to flight media her the blood. medical is toxic who last Halifax. of had Ketoacidosis a reported XX-year-old due levels a emergency
and soon Fortunately, after was Abby survived type with diabetes X quickly landing. diagnosed
and potential important onset medical groups so is sugars. or only screening, is TXD. Preventing This professionals, we patient touched convinced payers, why preventing are avoiding families will be already the avoidance not so those means delaying of therapy the especially high or by diabetic why embraced or it of ketoacidosis, means worry low TXD delaying teplizumab by blood advocacy daily and
to allowing TXD thousands to and night parents of constant for of profound dangerous have and And It health hundreds compelling the lives giving a sleep that and our positive screen relatives it for individuals. the other of step events. entire means system family consequences means wellbeing care reason through the autoantibodies, of hypoglycemic a people will without fear and members
the and/or familial public believe a a populations. screen with explore broader necessary We opportunities. health the resources will market, diabetes provide to potential commitment we partner will partnership pediatrics, Beyond to
including on and realizable, favorable aligned while in a launching North be continue given stand-alone America, To market opportunities, evaluate partnering, we dynamics. basis realistically the potential to is clear, unique teplizumab
that comes to optionality well And from as our optimizing trade-offs between we consider not substantive the of it only having from options options recognize understanding leveraging maximization timing from, the as depends value those we also true selection. and and upon potential partnering, various as select
being have for Following the of and market advantage completion study in and indication already newly with its submission partners patients, approval PROTECT our from potential considerable and/or will the at-risk diagnosed the pediatric networks, of help will and endocrinologists, rapidly and systems. cemented anticipated teplizumab market having advantage diabetes maximize larger health this diagnosed market educators, segment strong payers the This end relationships newly certified with patient and penetrate opportunity. TXD Provention care
Beyond management life launches, and are diagnosed planning other opportunities. for the expansion, additional and we already newly indications cycle at-risk label
the examine redosing of for of teplizumab extend delay TXD progression to plan individuals. we at-risk the to First, potential
with blood, T of delay realized or in response At-Risk the course peripheral courses teplizumab be have only of which Study evaluated with such to clinical-stage diabetes further teplizumab. timing single therapy. predictive XX-day such the be guided a of of been shown additional redosing may by correlate as prevention We Recall patients. believe anticipate We biomarkers in cell can levels exhausted the TXD
and between to diabetes and teplizumab ages of incidence age of use we of one-third age eight. a of X newly XX, in for path children the evaluate juvenile before eight While the indications. Therefore, for diabetes forward diagnosed XX the approximately diagnosed the the of type occurs childhood all peaks plan at-risk of both under
We see backbone for opportunity various unique serving with of also a as therapies, regimens. the combination teplizumab therapeutic
with For transplantation cell medications, example, or metabolic immunomodulatory antigens, beta combination other agents.
approach, with B the as cell PRV-XXXX. combinations mechanistic own anti-interleukin-X as do combinations For our antibodies, latter presents anti-TNF anticytokine such exciting agents, with or including opportunities
therapies certain are diseases, the as biospecific and CDXXB scaffold and of a relevant currently PRV-XXXX, interception gene proteins. as B advancing as management CDXXB cell-mediated associated well autoimmune both We of therapeutic potential targeting clinically with for immunogenicity such lupus,
Phase in top for first Ib/IIa study and progressing we year. plans initiate from results two-part XXXX, of safety the quarter line the trial from This lupus of multiple-ascending is later the We next dose Ib part PREVAIL in was eagerly the patients study Phase part August. which feedback with the Phase IIa expect launched in PRV-XXXX, well, await to
could rheumatoid on activated plan exploration route autoimmune Crohn's, and Lilly of formulation T other a administration autoimmune MacroGenics facilitate formulation, subcutaneous resuming the potentially teplizumab of teplizumab we and cells, had explore. for indications, to disease convenient previously Finally, and initiative example, by more an driven of started provide for development serious hepatitis, arthritis. celiac a the Such
immune-mediated drive it a a provided for The too in indications. strategic shift is broadly as continue teplizumab vision validation be and to our TXD late. or has other extend paradigm Provention, efforts going we intercepting more exciting is to disease on for year XXXX focused to program before very our preventing
the the my thank executives we partners support, in and like would employees, their Before participating patients our and open amazing consultants. trials investigators our for fellow investors all and call questions, for of and to our clinical continued team Provention I
Thank we're Operator, now take ready questions. you. to