Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) Q3 2023 Earnings Call Transcript

So just trying to get a better sense of the breakdown of those potential milestones. And then the second question and just — then I’ll jump back in the queue. On OTC, is there a consideration, just given how excruciating frankly the recruitment has been of this patient population? Is there just a view that the demand among patients is just really not there or that they’re just too hard to find and this indication might be smaller than what we thought previously? But just sort of begs the question, is it worth it to keep pursuing this indication given the very challenging nature of recruiting the trial? Thanks.

Joseph Payne: Sure. Thanks, Seamus for the questions. First, I’ll walk through some of the near-term milestones as you’ve asked. So with our internal programs, starting with our OTC deficiency program, we’ve indicated that the database is being locked relevant to the Phase 1 and Phase 1b data for the OTC program. That database lock is going to occur later this quarter. With respect to Phase 2 interim data, we were guiding the first half of 2024. Now shifting to the CF program.

Padmanabh Chivukula: Hey, Joe, let me answer that question. I think he was referring to the financial milestones. And I think what, is that correct, Seamus? You were referring to the financial — trying to understand the various programs and obviously with the cash runway going to.

Seamus Fernandez: Yeah.

Padmanabh Chivukula: Okay. That’s what I thought.

Seamus Fernandez: Correct. In terms of the deal with CSL.

Padmanabh Chivukula: Yeah. No, no, that’s what I thought the question was pertaining to. So we typically don’t guide specifically on the individual milestone because they’re frankly very lumpy, right? And we don’t know when they’re going to start and initiate a certain program and when with the catalyst for that program be achieved, right? So in terms of not disappointing people, I’d rather announce those milestones when we achieve them and more freely be able to articulate how we were able to accomplish that feat. And what’s going to be critical here as we go forward is the guidance that we give you with the amount of cash that we have to give you a perspective of kind of what we’re burning outside of the CSL and the BARDA relationships, as well as the contribution from the Cystic Fibrosis Foundation.

So if you take basically the number of years and divided by our cash, you’re going to come close to about $120 million in burn. And our goal will be to bring that down even more. So consequently you know the guidance that’s going to be critical to understanding the timing of when these milestones come in. And they’re pretty significant obviously those over $1 billion in development milestones that had over three to five programs. So they’re pretty meaningful, they’re going to have a significant impact on our operations. And as we achieve them, we will certainly be able to explain how we earn them and why we did. And hopefully, that’ll provide you enough comfort that we are well funded into at least for the next three years. Without any revenue milestones in our forecast, none, no commercial milestones or revenues are included in our forecast that would be certainly considered you know, supplemental, and we will update the market, assuming we do have revenues in 2024.

Joseph Payne: And Seamus —

Seamus Fernandez: Okay. Thank you very much.

Joseph Payne: I can give you a little bit more color on the OTC program. As you know, rare disease programs are typically slower to recruit compared to some of the work that we’ve done with the vaccines. And it’s a well-known phenomenon and specifically our OTC trial is being conducted in research centers, which can be slower, of course. So we’ve taken quite a bit of action to potentially speed that up. There are two key things that we’ve done, in the near term that’s going to help in recruitment and trying to speed this up. We enhance the patient experience and we’ve added a concierge service so that we can pay for all of the patients’ needs. And then we’ve also implemented a patient stipend to recognize for — so a lot of their efforts to be part of this trial. So I think both of these things and in terms and also opening up more sites is going to help in recruitment in the near term.

Seamus Fernandez: Right. And I guess, just as a follow-up question to that though, is there any concern around the size of the market opportunity for ARCT for through to, but just trying to get a better sense of — usually there is also demand for rare conditions where there can be benefits, obviously, there are other treatment options out there. So I’m just wondering if there is an assessment that would make sense. As it relates to this program just because with the CF program advancing is it — would seem like resources might be better spent to bring for other rare disease opportunities. It’s just been many, many years pursuing that. So just trying to get a sense of how you guys are thinking.