The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Tessa Romero - J.P. Morgan - Analyst
: Great. Thank you, Brian, for the presentation and please come on.
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JANUARY 10, 2024 / 3:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at JPMorgan Healthcare Conference
So so I thought I would start the discussion here on the energize study results, which, Brian, thank you for articulating those right now for us. I did
want to just ask about helping to contextualize that hemoglobin response rate that was observed and mechanistically, why mean a patient? Not
bye-bye?
Question: Tessa Romero - J.P. Morgan - Analyst
: And this actually segues really nicely into my next question, which is what should we specifically look for in terms of what we may see in a fuller
medical meeting presentation, of course, now the data itself is, but the types of analyses that we might expect, right?
Question: Tessa Romero - J.P. Morgan - Analyst
: And how should we think about the level of read-through between energize and energize T. business?
Question: Tessa Romero - J.P. Morgan - Analyst
: Jonathan, please raise your hand if you do have a question. I don't think my iPad is working. And so can we talk a little bit about energize P & A
little more detail here, particularly what you are looking to see around the primary endpoint, which is and a 40 well 40 week read on transfusion
reduction, what response rate are you looking for there?
Question: Tessa Romero - J.P. Morgan - Analyst
: And you are enrolling a broader population here of both alpha and beta thalassemia patients.
So how do you think about the relevant clinical threshold here to have to benchmark your results to and perhaps it might make sense to orient
folks in the room on what is currently available for where I see the PET, but not alpha thalassemia?
Question: Tessa Romero - J.P. Morgan - Analyst
: So in the interest of time, if I would do a couple of quick hits on the pipeline in commercial here. So on the mitapivat program in sickle cell disease
comfortable, are you that the sickle cell pain crises observations that you saw in the Phase two portion of rise and that there is a real signal there
that you will discern in a in a longer Phase three study?
Question: Tessa Romero - J.P. Morgan - Analyst
: Please.
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(technical difficulty) Ask I'm not saying in thalassemia on all 30 odd patients who will be eligible, there will be no eligibility criteria for the trial.
Question: Tessa Romero - J.P. Morgan - Analyst
: And another update that, Brian, you talked a little bit about in your presentation was the decision to move forward AG nine four six in lower-risk
MDS. What underpins your confidence there? And it seems like mitapivat is ERM five, nine six is elucidating a benefit on transfusions but not
hemoglobin response. Is it possible that we might see that as the study moves forward?
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JANUARY 10, 2024 / 3:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at JPMorgan Healthcare Conference
Question: Tessa Romero - J.P. Morgan - Analyst
: So maybe I'll squeeze in a commercial question so that we're not leaving anybody out here. So I mean, has been on the market in PKD for some
time now, how do you think about where the next leg of growth comes from for that program?
I think 100 patients are on therapy as of your last update, we have noticed that there has been maybe a little bit of slowing of patient adds. So how
do you think about the dynamic there and in what is that more than 100 patient prevalence pool?
Question: Tessa Romero - J.P. Morgan - Analyst
: And that was that was great. Thank you. And I think we're out of time. I want to thank the entire Rogers team for being here as part of our conference,
and thank you also for all the listeners for taking the time to join us. I hope everyone has a great rest of the week.
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JANUARY 10, 2024 / 3:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at JPMorgan Healthcare Conference
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