Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference Transcript

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

Question: Ashwani Verma - UBS Equities - Analyst : Great. Excellent start. So maybe I know like Danon disease is a big focus area for you, but I'll come to that. Let's kind of like save the best for the last. So I guess the recent updates that we have seen, right, like one is just on like the KRESLADI. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 1:00AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference So I know that you had received like a CRL. You're working with the FDA to try to do all that. They had requested some CMC information. And -- but no clinical information, right? So where are the -- where are you in that process? Like do you feel confident that you're marching towards like a potential approval?

Question: Ashwani Verma - UBS Equities - Analyst : Got it. Okay. And then like for Fanconi Anemia, so you are in the process of like this rolling BLA submission. Do you believe that this is some time, like in 2026, like you will be able to launch that just --

Question: Ashwani Verma - UBS Equities - Analyst : Anything to outline in terms of like what's the regulatory path? And what are you expecting there?

Question: Ashwani Verma - UBS Equities - Analyst : Yes. So this would be like sort of like catapult the drug, the company into a commercial stage biotech. And just in terms of like anything that you can mention, where is the -- like the TAM going to be? Or what have you done in terms of like patient identification initiatives? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 1:00AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Ashwani Verma - UBS Equities - Analyst : Yes. All right. Great. So yes, let's focus on Danon. I mean, for this RP-A501, just kind of if you can like remind us like this Phase II potentially pivotal trial, like what's the design? Why are you focusing on these co-primary endpoints, LAMP-2 and LV mass reduction, just to sort of give the rationale for that.

Question: Ashwani Verma - UBS Equities - Analyst : That's good. So how many patients is that? And just like what therapy? Is it like similar endpoint like LV mass reduction that we will be able to take a look at?

Question: Ashwani Verma - UBS Equities - Analyst : Got it. That's great. And then I guess the -- in this type of a setting, I think patient age has a meaningful impact on sort of like the magnitude and the rapidity of how quickly you can start to see the benefit on LV mass improvement. I mean, if you can talk about like so what was sort of those metrics, what have you seen in like Phase I and for Phase II, like if there is any different in terms of like the baseline age or all those sort of nuances to think about?

Question: Ashwani Verma - UBS Equities - Analyst : Got it. So going back to the status of the study. So you mentioned that you have completed the enrollment of these 12 patients by September. And then -- so you have dosed some of them, not all of them, just like what's the cadence of like how long does it take to get to that dosing?

Question: Ashwani Verma - UBS Equities - Analyst : Okay. But when did you -- when did you initiate -- when did you start enrolling the first patient? Or when do you dose the first patient? Have you talked about 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. NOVEMBER 13, 2024 / 1:00AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Ashwani Verma - UBS Equities - Analyst : I see.

Question: Ashwani Verma - UBS Equities - Analyst : And these are like new patients versus what you had in Phase I, sort of like no, sort of, reusing of patients, I'm assuming.

Question: Ashwani Verma - UBS Equities - Analyst : Sites-wise, like seeing similar or --

Question: Ashwani Verma - UBS Equities - Analyst : Okay. All right. So yes, I guess one question that I get from investors on this is that, I think, in the last one year, sort of people have been expecting some sort of like an interim update on the study. And this is really sort of, you're looking at the instant or a delayed gratification on the story, right? Like that's what it comes down to. So when you think about that, like, is there like a single data update that you would get for like the 12-month endpoint or is there some breadcrumbs along the way that you might drop which might be useful?

Question: Ashwani Verma - UBS Equities - Analyst : Yes. I'm sure you get a lot of questions on sort of the curve of enrollment as well and when does that start to like enroll there. And sort of like 12 months out, is it like is it most of the 12 months out, is it going to happen in like second half '25 or --

Question: Ashwani Verma - UBS Equities - Analyst : Right. And so this is for sort of like an accelerated approval, right? Like for the full approval, what level of understanding do you have with the FDA? Yes. Like how much of a follow-up would that require? Is it the same endpoint? Any focus on like LAMP-2 versus the LV mass reduction?

Question: Ashwani Verma - UBS Equities - Analyst : Got it. Is there a scenario in which like, let's say, if the data coming out from this Phase I is really good on certain aspects that you might not need to show like a longer follow up on your pivotal study?

Question: Ashwani Verma - UBS Equities - Analyst : And these trials are like have you disclosed how these trials power, like from an endpoint? Like what is the -- effectively like the delta that you are trying to show? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 1:00AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Ashwani Verma - UBS Equities - Analyst : Is the natural history, the data here, like fairly tight or is there like a lot of noise there that it would be like well accepted?

Question: Ashwani Verma - UBS Equities - Analyst : Yes. And like does that sort of relationship it stays within a tight range as the age of the patient? What I'm trying to get at is like is it like the sort of the patient age demographic that you have in your trial, is that like well comparable to the natural history cohort?

Question: Ashwani Verma - UBS Equities - Analyst : So the natural history data, like when can we see that? I guess it's going to be before or after the Phase II?

Question: Ashwani Verma - UBS Equities - Analyst : Got it. Okay. So we'll have a clarity on that. All right. Anything else in Danon before we talk about like a couple of other things on your pipeline? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 1:00AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Ashwani Verma - UBS Equities - Analyst : Yes. That's great. Yes. Looking forward to it. So maybe just like switching gears and like talking about some of the -- like the ACM program, right, this -- the PKP2 like that you were talking about earlier, 601. So yes, just if you can quickly give some thoughts around like the market opportunity, like where do you expect the Phase I update, like what should we look forward to that, and just in terms of like a clinical development path, like how can that look like in this program?

Question: Ashwani Verma - UBS Equities - Analyst : Yes. I mean as you think about like these indications, right, like this is pretty unchartered waters that we are in, and not a lot of work has been done in this. So like great opportunity. But I guess like when you think about like sort of these like biomarkers and what actually like makes a difference, what gives you conviction that you have some sort of like a unique insight into this, that the hypothesis that you're working with is actually like the right way versus trying to -- how do you get there faster, basically? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 1:00AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Ashwani Verma - UBS Equities - Analyst : Got it. Great. Excellent. So yes, I mean I guess for PKP2, is there any sort of like a read across do you see to Danon or vice versa? Like one approach works, what implication does it have for the other?

Question: Ashwani Verma - UBS Equities - Analyst : That's great. So just as we look at like the Phase I update for this, in first half '25, just curious, anything that you would outline in terms of specifics? Like how much follow up? How many patients? What sort of like endpoint to just like look at more objectively than the others?

Question: Ashwani Verma - UBS Equities - Analyst : Got it. Okay. All right. That's great. So I guess -- so we talked about sort of like four programs, right, LAD-I, Fanconi, Danon and ACM. Just if you can like talk about the remaining two, just to see like where we are.

Question: Ashwani Verma - UBS Equities - Analyst : Good.

Question: Ashwani Verma - UBS Equities - Analyst : Yes. Lots of progress on the pipeline. Looking forward to the updates. I mean, I guess like just to close it off, like I'll ask a question to you about like regulatory landscape. I know there's been lots here and discussed on this. And I get the impression like across my coverage as well, I see there are a lot of like actions by the FDA in the last like one or two years, seem to suggest that the agency was getting more and more flexible, like more adopting like this natural history cohort, like endpoints that are sort of like biomarker-driven. Do you anticipate any kind of like a meaningful change? Or like you're running a business which is very sort of like tied to the -- with what the agency thinks and does, right, so what is your underlying assumption in terms of what might happen in the next administration? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 1:00AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Ashwani Verma - UBS Equities - Analyst : Good. Good. That's a good positive note to end our session in. Thank you so much for taking the time. Thanks for joining our conference. And good luck with all these programs. You have a lot going on. So good luck with 2025.