Rocket Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference Transcript

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

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : <_ALACRA_META_ABSTRACT>And maybe we'll just hop into the Q&A and maybe a couple of background questions first. Just given your focus on gene therapy and your broad pipeline of both AAV and LV approaches, maybe you can just talk about how those are different? What are the advantages and disadvantages of each?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Makes sense. And maybe just in the gene therapy space, very innovative and sort of a new area over the past several years with the FDA. But maybe just talk about your experience with the FDA, how supportive are they of these types of therapies?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Great. Maybe we can switch gears and talk about Danon. That's your lead program. Maybe just give us a little bit of brief background there on the program as well as the disease and where the unmet need is, which is pretty high.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. Maybe just talk about -- you walked us through a bunch of these endpoints and dramatic effects across all of them. How did you go about determining which ones to use in the registrational study?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Makes sense. Just on the primary, can you talk about sort of the bars in LAMP2 expression or LV mass reduction and kind of what you saw in your Phase 1?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Sure. Just in the Phase 1, maybe on both those endpoints, LAMP-2 expression, when does that start to occur after treatment? And then what's the sort of time frame of improvements in LV mass and when you see those?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. And remind us when you started enrolling patients in the pivotal study and just maybe how that's been going?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Have you said when you expect to complete enrollment at all or --?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. And just given the fact that it's technically open label, right, it's not randomized, any thoughts about maybe sharing some data early or not? Or just how do you go about sort of determining whether or not you want to do that?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. Makes sense. And maybe talk a little bit about sort of natural history and what you're doing there because that's going to be the comparator for approval. And I think you're running a couple of different studies, if I recall?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. Is there any way to match the baseline characteristics? Or is this too tough because it's such a rare disease?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Okay. How long is that period? Pretreatment? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 05, 2024 / 6:35PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Okay. Got you. Maybe just talk about the market opportunity in Danon disease and how patients currently are managed?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : I guess why are patients misdiagnosed? And I guess, what's the process to diagnose a patient and how do you sort of expand that?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. And how do you sort of push that forward or anything else you can kind of do prior to your data/launch to sort of accelerate that? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 05, 2024 / 6:35PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Can you also talk about manufacturing? You've built your own facility, and maybe just give us a sense of the status of that and the supply of 501.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : No.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Okay. Great. And maybe we can shift gears to just the PKP2 program. Maybe just give us a little bit of a quick background there and the status.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : And I think you recently started the Phase 1, is that right?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : And maybe just talk a little bit about the trial design and maybe some of the key endpoints and what you hope to learn in the initial data readout.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : So sort of similar process with Danon. You kind of look across all the data and sort of pick the best? Okay. When might we see some of that initial data? Is it possible next year? Or is that too aggressive?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. And then maybe just to wrap up AAV, just touch on BAG3? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 05, 2024 / 6:35PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Maybe we can switch to the LV pipeline. And you've got three programs there. You mentioned some early on, but maybe we'll focus on KRESLADI, LAD-I. You recently got the CRL. And maybe just walk us through that a little bit and next steps there.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Maybe talk about just sort of launch prep opportunity. How much investments you're making there?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Got you. Maybe just switching now to just Fanconi anemia, PKD. Maybe just give us a little bit of background on the disease there. And what's unique about that program?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Can you just talk about why elimination of conditioning is such a big deal?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Makes sense. Can you just remind us the status of that program and when we might see the next update?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. Makes sense. And maybe just talk a little bit about the market opportunity for Fanconi anemia and maybe just put that in the context of the other two sort of LV assets. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 05, 2024 / 6:35PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Okay. Maybe just talk beyond the assets we talked about, maybe earlier-stage pipelines or your thoughts there in terms of developing more AAVs or what your thought process is.

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Yeah. Makes sense. And then maybe just a last quick question, just your current cash position and how far that gets you. Is that through the Danon data?

Question: Mike Ulz - Morgan Stanley & Co. LLC - Analyst : Fair enough. Yes. Great. I think we're just about out of time. So why don't we end it there? Gaurav, thanks so much. I appreciate your time.