Rocket Pharmaceuticals Inc at Needham Healthcare Conference Transcript

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

Question: Gil Blum - Needham & Company, LLC - Analyst : All right. Excellent. That's a very good overview. So maybe another one of these broader question, something that's been on the investors' minds lately a lot. So considering the sort of pressure we've been seeing in the gene therapy space, but even compared to the (inaudible), what are you thinking potentially turning the tide here? What kind of events did make a difference at this point? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 11, 2022 / 2:15PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Needham Healthcare Conference

Question: Gil Blum - Needham & Company, LLC - Analyst : Great. Thank you very much for those insights. So now we can dive in on Rocket's own programs. A good place to start is the program most investors pay attention to and that's the Danon disease program. This is RP-A501 which is a AAV-based gene therapy, and we are expecting some updates in the third quarter. So what expectations can you set for us around this upcoming data readout?

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. So one key question around Danon disease has been design of a regulatory study. What update can you provide around Rocket's thinking for potential endpoint? And just to remind everyone, timing on regulatory update is in the second half of the year.

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. So maybe a couple of things to unpack here. First of all, assuming you're going to look at protein expression that suggests that you're definitely capable of doing an invasive endpoint, right? You take a biopsy there. Would you also include potentially invasive hemodynamics that you're already going in?

Question: Gil Blum - Needham & Company, LLC - Analyst : That makes a lot of sense. And maybe touching again upon the change in heart dimensions, which you mentioned. So it looks like this accelerates in patients that are aged 10 to 15 based on the Danon registry database [they've got to put out]. So how does this information play into your patient selection for a potential pivotal?

Question: Gil Blum - Needham & Company, LLC - Analyst : That makes a lot of sense. So there is a sufficient flexibility around patient selection regardless of their ages. It's more based on homeostasis of their disease condition.

Question: Gil Blum - Needham & Company, LLC - Analyst : Maybe a bit of a recycled question. This is something that we've been asked a lot. What do you guys consider the addressable pool of patients for RP-A501? And due to learnings from the natural history data, because we've been working on it pretty significantly, give you an idea of how many patients may go on treatment initially?

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay, excellent. And now, kind of a last one on the Danon disease: so is a pivotal study likely to be conducted with commercial-grade materials to circumvent any CMC-related updates later on?

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. Excellent. And I'd like to veer to the lentiviral platform, and I actually want to start with the PKD program with RP-L301. And we're going to be seeing some data at the back half of the year for the Phase 2 study. Can you give us maybe a little bit of -- maybe a little bit background on this program, and also what kind of data should we be expecting on the third quarter?

Question: Gil Blum - Needham & Company, LLC - Analyst : So maybe a different way of asking the same question: what is still missing? I mean, you guys did have an agreement with the FDA, and there's actually quite a decent amount of data out there already. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 11, 2022 / 2:15PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Needham Healthcare Conference

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. Maybe you talk --

Question: Gil Blum - Needham & Company, LLC - Analyst : Maybe a good time to kind of talk about another of your LV programs for a larger indication, actually: so RP-L301 in pyruvate kinase deficiency. So there's also ongoing Phase 2 with the fourth quarter update expected. Can you give us a little details what we should be looking for?

Question: Gil Blum - Needham & Company, LLC - Analyst : Excellent. Maybe to kind of sharpen a little bit the point that you made about potential patients kind of help us [triangulate here]. Do you think it would also potentially include patients who are failing through drug, let's say mitapivat, which is recently approved in this indication? I know that certain patients cannot even be treated with mitapivat and will have [any function enzyme], but would that be an interesting population? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 11, 2022 / 2:15PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Needham Healthcare Conference

Question: Gil Blum - Needham & Company, LLC - Analyst : Right. Excellent. Maybe something that's still in the works, but what are your initial considerations for Phase 2 endpoints? Are we talking about heme? Clearly, we're probably talking about hemoglobin levels. But are we also going to look at transfusion independents so that sort of thing? What's your thinking?

Question: Gil Blum - Needham & Company, LLC - Analyst : So again, touching upon this mainly assuming continued success, where do you think you're going to go with the label expansion? But like we said potentially sequencing with mitapivat, any other thoughts?

Question: Gil Blum - Needham & Company, LLC - Analyst : Right. Last but not least, let's talk a little bit about LAD-1. So can you [offer] a remark on timelines for potential filing post the readout, which is actually coming pretty soon? And I think it's in this quarter?

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. So considering Fanconi anemia is pretty far along -- you know LAD-1 is also pretty far along -- you have a potential to become a commercial company by 2023. Where is that commercial production going to take place at this point? I mean you have your facilities in Cranbury, but are you starting?

Question: Gil Blum - Needham & Company, LLC - Analyst : So what level of investment do you think is still required for that commercial step-up, considering only just that?

Question: Gil Blum - Needham & Company, LLC - Analyst : That's some very useful color. So I'd like to end up with maybe a couple of open-ended ideas here. So there've been very limited regulatory filings to the FDA in the last couple of years. Do you think a filing and approval for either of your LV programs or both will move the needle a little bit for the LV gene therapy space broadly? Because I think there's a lot of skepticism -- if you could address -- [your thoughts] there?

Question: Gil Blum - Needham & Company, LLC - Analyst : Bring lenti back?

Question: Gil Blum - Needham & Company, LLC - Analyst : I would like to maybe spend the last minute here talking a little bit about your Wave 2 programs, which you've mentioned. Should we assume that the company is doubling down on the cardiac [in this] space when you recently added Fady Malik to your Board? So it seems like you're going in that direction. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. APRIL 11, 2022 / 2:15PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Needham Healthcare Conference

Question: Gil Blum - Needham & Company, LLC - Analyst : All right. Thank you so much for joining us. This has been super informative, and we will continue to be in touch. And have a good day.