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
: Thank you, Josh. Thank you, Weldon after the presentation. So I might start with a little bit more of a forward-looking question on the Dine one
zero one program in myotonic dystrophy type one that you outlined for us now that we have some initial data in here and how does that inform
what you might be able to see in the next update? And what is the and maybe frame for us what you might think would be a win-win scenario in
the entirety of the trial?
Question: Tessa Romero - J.P. Morgan - Analyst
: And if I could just toggle back to the splicing data that you showed, we have gotten some questions around the low dose. And folks are pleased
to see that consistency between the day 85 and day one 69 dose time point. And the fact that you have consistency there, but what drove the
difference driving from 13% pricing correction to the 7%? Could you just clarify that and what's underpinning that? Because I've gotten that question
a few times?
Question: Tessa Romero - J.P. Morgan - Analyst
: And you know where on May, we PharmAthene halfway through placing R&D at tumor cohorts to go here? So you you achieved 19% splicing
fraction of that second dose, right? So you've been talking about 2020 to 25% as the goal of achieve for a long time. Like where do you think you
might be able to get to for dose range?
Question: Tessa Romero - J.P. Morgan - Analyst
: We may touch on the regulatory strategy or should last point there and how you think about getting this program to have an approval here? And
what conversations have you already had with regulators versus what conversations might you need to have next? And then can you just remind
us the status of the program in the US?
Question: Tessa Romero - J.P. Morgan - Analyst
: What is a framework for us to think about with respect to how you see the potential size of the market in DM1 here.
Question: Tessa Romero - J.P. Morgan - Analyst
: As we may turn the discussion over to Diane to 51 in DMD, you know, I think a similar question that asked and DM1, I think it would be helpful for
everyone listening. Just to better understand again, how do you how does the data that you've generated? So far and form what you think you
will see in the entirety of it?
Question: Tessa Romero - J.P. Morgan - Analyst
: And final question for me in the last minute or two here. How far along in Stein on some of the other exons beyond exon 51? Because we know
that exon 51 is just a portion of DMD business.
Question: Tessa Romero - J.P. Morgan - Analyst
: Gentlemen, maybe you might just give us a quick cash balance update and or what your current expected cash runway would get into Europe.
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