Rocket Pharmaceuticals Inc at JPMorgan Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : And if there are questions, just wait for the microphone. But let me get -- just by way of getting started with Q&A, you kind of teased a series of updates coming for the Danon disease pivotal program. So Gaurav, I might ask you to just sort of unpack the set up here a little for us, right? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JANUARY 13, 2025 / 11:45PM, RCKT.OQ - Rocket Pharmaceuticals Inc at JPMorgan Healthcare Conference Which is -- I mean, one is probably pretty straightforward in terms of either enrollment or treatment update, right? The trickier one is perhaps the trial design update with some statistical considerations. What is encompassed within that perhaps? And to what extent are we thinking about sizing of the study or changing the size of the trial as a result?

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : Okay. Okay, great. And perhaps on the, call it, natural history or prevalence population patient ID side, I guess, part of that would be patients identified maybe as part of the screening process, but as part of a measure of sort of priming the market to some extent, when commercial, maybe just -- probably orient us around that a little bit. And then how would that sort of also inform your top down estimate when thinking about the overall size of the preference population that you think Danon's disease is, the ability to sort of refine that? And then also how your experience here is going inform or make the ID process -- patient ID identification process perhaps a little more efficient as a commercial stage company?

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : There's both the market sizing consideration related to that and also patient identification for the purposes of building the market. But also those data sort of might incrementally inform the natural history of the course of the disease out there. I guess, is that an expectation? And will your update kind of speak to that? Any change or thinking around the level of unmet need in the, particularly, male Danon disease population?

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : Got it. Just on PKP2. And I think you've kind of touched on perhaps one of the challenges. And certainly one of the things that we're thinking about in terms of the initial readout from this program, which is what the target level expression is that you need to achieve in this patient population, right? Just given the fact that it's a heterozygous genotype.

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : Just looking at your protocol, I believe it has an exclusion criteria or it specifically calls out patients with truncating mutations as eligible for participation. Just talk about sort of what that's meant to distinguish from perhaps other mutations that are present, that emerge within PKP2 and sort of what the phenotype there is. I guess the question is sort of around -- from the population genetics, whether that might also help inform the sufficient amount of protein expression that could be corrected for these patients.

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : Okay. All right. So it's still -- yeah, you can't really use those patients as a way of sort of figuring out what you can get away with. It's really to avoid those that might actually have a pathogenic protein and therefore undermine the efficacy of your agent. So, well, great. So I guess, some kind of a key question on the biomarker side there with PKP2. On the clinical side -- from the biomarker, of course, we'll learn more. On the clinical side, maybe just talk a little bit about sort of what endpoints will ultimately inform kind of clinical benefit. And maybe kind of leading into that, the extent of baseline information that you're gathering, either from their patient histories or screening that would allow you to kind of make an assessment of clinical benefit over time.

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : Maybe the last question. Just thinking about the lentiviral products potentially launching in 2026. Just how to think about the prioritization of those commercial launches relative to everything that's going on the AAV side. And then maybe I'll just try to sneak in, to the extent -- the question about the extent to which you've had interactions with payers and potential pricing considerations of both the LAD-1 and fanconi anemia gene therapy.