BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) Q2 2023 Earnings Call Transcript

Jon Stonehouse: But I think at the end of the day, Nicole, it’s all about what do we see in humans. And do we have a safe and effective dose that’s one today? So that’s the whole point behind that trial.

Helen Thackray: Got it. And then just really quick, I know the last quarter there were some unanticipated headwinds with the lack of funding from some of the external charities, helping to provide [indiscernible] for patients on ORLADEYO. I guess where does that situation stand currently?

Charlie Gayer: The situation has stabilized. So we are not losing more patients or having patients having to drop back to free product since the first quarter. Those that we did put on long-term free product in the first quarter have to stay on it for the rest of this year, but for now, the situation has stabilized.

Anthony Doyle: Yeah, and I think it’s just made one big plus for Charlie and his team, despite that they’re making great traction or having great traction in converting patients from pre-product to paid as he said it was at 34% and now it’s down to 30% and my expectation, I think, Charlie as well is that that’s going to continue to go down.

Operator: Our next question comes from Stacy Ku – Cowen & Co. with TD Cowen. Please go ahead.

Stacy Ku: Thanks so much for taking our questions. We have a few follow-ups. So first, regarding 10013 and additional cohort, was this a question from the FDA or just the next natural stop in your development for this program? And then regarding the potential range of doses for the global PNH study, willing to provide some details there. And I know you’ve discussed it in the next year, but their potential to get a sense of the safety profile any earlier. So those are our questions on kind of the pipeline. And then regarding ORLADEYO, I know you’ve, in the past, not discussed exactly the size of the Salesforce, but not even increasing in Q4. Could you just talk about the relative sizing versus other competitors like the Takeda you think about expanding potential efforts and maybe communities that and clinicians that really only treat to those patients? Thanks so much.

Jon Stonehouse: So Helen, you want to take the 10013, and Charlie, you can take the salesforce.

Helen Thackray: Yeah. So, 10013, the dose that we’re adding with the healthy volunteer trial, the 160 milligrams, that is for our own purposes, that is so that we can assess mostly PK from that dose level and those are the entire PK model. We’ll need that once we have our patient data in order to confirm the final dose for pivotal trial initiation. And so that’s why we’re adding that dose level. In terms of the range of doses in that we’re looking in the PNH trial, we’ll be starting at 80 milligrams and dosing up from there. What we’re looking for is complement activity. We’re looking for that to be — we’re looking at that for multiple doses and in the disease state. We’re also looking then for clinical outcomes. And we’re trying to assess, then, for safety, but more importantly, for efficacy and that in the PNH, in the population setting, we’ll be in critical outcomes like looking at hemoglobin and LCH.

Jon Stonehouse: And Helen, it might be good to just talk about roughly how many patients do you think we need for that study to get a sense of that. And how long do we need to follow those patients to get a sense of the safety, at least in the short, this and the short study.

Helen Thackray: Yeah. So that’s a small study. We don’t need many patients and that’s one of the reasons to assess a complement inhibitor in patients with PNH. You can dose escalate and we will be doing that within individual patients and each one will be giving us information on then how that patient’s complement system is affected and their own clinical outcomes and dose escalating with that information. So not very many patients, small trial. In terms of the safety information, we’ll be looking at that as we dose up as well. And so we’ll have, again, an individual patient, the ability to observe safety across the range of doses.