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: Josh Schimmer - Cantor Fitzgerald - Analyst
: Great. Thanks for taking the questions and for the comprehensive overview. I guess, first question, how do we think about quantifying the dose
that each patient is getting as part of the QTORIN rapamycin gel? I guess, that would be measured as number of pumps per patient. And then how
many pumps per tube would you expect QTORIN rapamycin to be supplied?
Question: Josh Schimmer - Cantor Fitzgerald - Analyst
: Okay, got it. And then given the difference in clinical manifestation, how do we think about the difference, if any, in the primary endpoints between
the cutaneous lymphocystic malformation or the MLM, microcystic lymphatic malformation, trial versus the cutaneous venous malformation trial?
Question: Josh Schimmer - Cantor Fitzgerald - Analyst
: Okay. Thank you.
Question: Joshua Fleishman - TD Cowen - Analyst
: Hi, this is Joshua Fleishman on the line for Ritu. Thanks for taking my question. How should we think about the new QTORIN rapamycin indication
and the new QTORIN product announcement in the second half of this year? And should we expect for the candidate molecules to have similar
pharmacological characteristics to rapamycin? Thank you.
Question: Whitney Ijem - Canaccord Genuity - Analyst
: Hey, guys. Thanks for taking the question. Excuse me. First is a follow-up on the dosing question. Can you, I guess, remind us about lesion size and,
therefore, dosing differences between MLM and CVM?
And I guess, to what -- given you'll have that CVM data by the time you have the Phase 3 data, to what extent might any differences there play into
your pricing discussion in the MLM indication?
Question: Whitney Ijem - Canaccord Genuity - Analyst
: Got it. Okay. And then, I guess, another follow-up on the next QTORIN rapamycin indication and then the next QTORIN program in general. What
is included in the current cash guidance as it relates to progress with those new programs?
Question: Whitney Ijem - Canaccord Genuity - Analyst
: Perfect. Really helpful. Thanks so much.
Question: Annabel Samimy - Stifel Nicolaus and Company, Incorporated - Analyst
: Hi, thanks for taking my question. I'm wondering if you can just help us understand the powering of the trial. What is required for statistical
significance, and more importantly, what is required for clinical meaningfulness? And I'm thinking more in terms of payers to really capture that
premium pricing that you're looking for in light of some off-label usage of rapamycin. Thank you.
Question: Annabel Samimy - Stifel Nicolaus and Company, Incorporated - Analyst
: Great, thank you.
Question: Louise Chen - Scotiabank GBM - Analyst
: Hi, congratulations on all the progress, and thanks for taking my questions. So just two quick commercial questions for you. If your products are
approved, do you think physicians will test patients for the PI3K mutation or just treat patients with CVM and MLM?
And then the second thing is just what type of support programs do you plan to have for your patients and physicians to help drive uptake and
get coverage for QTORIN rapamycin? Thank you.
Question: Dev Prasad - Lucid Capital Markets, LLC - Analyst
: Hi, thanks for taking our question, and congrats on the progress. So I have a question on CVM. You mentioned you will be looking at number of
endpoints in the Phase 2 trial. So what would be the bar in terms of different efficacy endpoint you will be looking? And what type of data will give
you confidence to move into next phase? Thank you.
Question: Dev Prasad - Lucid Capital Markets, LLC - Analyst
: Great. Thanks for that. Thank you.
Question: Catherine Novack - Jones Trading - Analyst
: Hi, good morning. Thanks for taking my questions. Just one more on cutaneous venous malformations in the Phase 2 study. Do you anticipate
breaking down efficacy by whether patients have PIK3CA or TIE2-activated mutations? Would this make any difference in your decisions for the
target commercial patient population? Thanks.
Question: Catherine Novack - Jones Trading - Analyst
: Got it. And then thinking about duration of treatment in microcystic LMs after FDA approval, do you anticipate this is something where patients
are going to require chronic treatment, or they treat it until remission and monitored for recurrent things?
Question: Catherine Novack - Jones Trading - Analyst
: Got it. Thanks so much for taking my questions.
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