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: Marc Frahm - TD Cowen - Analyst
: Thanks for taking the questions and thanks, everybody, for putting together today's session. But maybe for the physicians, just to maybe explain
some of the underlying biology and that's going on that leads to not just -- the fairly rapid change in your epinephrine levels and blood pressure,
but then kind of taking what appears to be kind of several months to fully resolve where it fully impact all of the symptoms on the patient scale?
And then your care and maybe this is also for the company within that scale, what do you view as kind of most impactful to patients and the some
of the biggest unmet need for the patient? Is it really the full breadth of the scale? Are there particular symptoms within there that you think are
particularly important to show an effect on?
Question: Marc Frahm - TD Cowen - Analyst
: It seems that you have a a fairly rapid impact on norepinephrine levels and then have also pretty rapid impact on blood pressure. But the impact
on symptoms seems to be a bit more delayed and take not dasy, but maybe a few months to really reach its maximum. Just kind of explain that
disconnect.
Question: Marc Frahm - TD Cowen - Analyst
: Yes, that's very helpful.
Question: Douglas Tsao - H.C. Wainwright & Co. - Analyst
: Hi, good morning. Thanks for taking the questions. I'm just curious on for the two physicians, if they can offer some perspective in terms of what
percent of the overall MSA population would be appropriate for treatment with ampreloxetine if it was approved and generally at what point in
terms of the patient diagnosis of the patient journey was treatment for their annual average on the needed or desirable?
Question: Douglas Tsao - H.C. Wainwright & Co. - Analyst
: And that's very helpful. And I guess as you think about what this means in terms of a patient's sort of disease progression? Or are there other sort
of secondary benefits for an MSA patient when you treat the nOH with ampreloxetine potentially?
Question: Marc Frahm - TD Cowen - Analyst
: And I guess just as a quick follow-up, I mean, how significant is that the conditioning effect that you spoke of Dr. Kaufmann?
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Question: Douglas Tsao - H.C. Wainwright & Co. - Analyst
: That's great.
Question: David Risinger - Leerink Partners - Analyst
: Thanks very much. And I wanted to add my thanks for you hosting this session. It's super helpful, and thank you to the two KOLs. So I wanted to
start with a couple of questions on the patient. So what does a typical patient profile? What do they look like? Are they obese or they skinny? And
how variable do they appear? I just trying to get a sense for that. And I'm also curious about the variability patient co-morbidities in a small trial.
Question: David Risinger - Leerink Partners - Analyst
: Great. And then if I could, I just wanted to ask a couple of more questions about the trial itself. So I guess first is fluoro cortisone treatment allowed
for placebo patients. Second, regarding the trial timing. The estimated primary completion on clinicaltrials.gov since December of '24, but it sounds
like it's more like May of '25. Can you talk about that change? And then finally, could you provide some more color on enrolling 100 patients to get
60 evaluable patients? Thank you very much.
Question: David Risinger - Leerink Partners - Analyst
: (multiple speakers) Yes, I'm sorry, those. So you mentioned those two drugs, but one of the KOLs listed a third on this slide, which is called fluoro
cortisone. What about that drug is that allowed for placebo patients?
Question: David Risinger - Leerink Partners - Analyst
: Thank you for all the color.
Question: Julian Harrison - BTIG - Analyst
: I thank you for hosting this very comprehensive event and for taking my questions. Dr. Kaufmann, that you highlighted a few minutes ago, the
potential for first line positioning with ampreloxetine. I'm wondering if you see a meaningful opportunity for switching as well? I guess asked
slightly differently of your and if a patient is being treated for nOH now? What percentage would you likely switch to or add ampreloxetine?
Question: Julian Harrison - BTIG - Analyst
: Excellent. That's very helpful. Thank you both. And then I thought the natural history of MSA was very well covered earlier on in this event. Sorry
if I missed it, but I'm wondering how long it usually takes from patients for symptoms to proper diagnosis and could that maybe change in the
future?
Question: Julian Harrison - BTIG - Analyst
: Got it. Thank you. And then finally on again, sorry if I missed it, is there a clear reason why droxidopa is not effective in MSA patients?
Question: Jingming Chen - Evercore ISI - Analyst
: (technical difficulty) thanks for taking our questions. This is Jingming on for Lisa. So I have a question for current treatments for MSA. So As [Rhonda]
mentioned, only 34% of patients are currently treated. So far, the remaining 66% what are the reasons that they're not receiving treatment? Because
given this is a deep (technical difficulty) disease. In a few years, patients will be severely disabled. I would imagine some treatment is better than
no treatment. So is there like some access issue, tolerability or purely because current treatment providing adequate efficacy? Thank you.
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Question: Jingming Chen - Evercore ISI - Analyst
: I'm referring to one of the slides that Rhonda you mentioned that the pie chart, where only 34% of patients receiving treatment. I'm just wondering
what's happening to the other 66%.
Question: Jingming Chen - Evercore ISI - Analyst
: That's very helpful.
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