Rocket Pharmaceuticals Inc at Needham Healthcare Conference (Virtual) Transcript

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

Question: Gil Blum - Needham & Company, LLC - Analyst : So maybe it's a good place to start as an introduction. I mean, investors have become kind of sanguine on gene therapy as of late. So do you think that potential for larger population drug approvals -- there is a filing for SRP-9001 in DMD and Exa-Cel in SCT, and your own drug in Danon disease. Do you think that could change sentiment?

Question: Gil Blum - Needham & Company, LLC - Analyst : So you're saying investors shouldn't lose their imaginative streak here.

Question: Gil Blum - Needham & Company, LLC - Analyst : I do want to discuss a couple of organizational changes internally in Rocket. You recently announced the addition of Mark White as Chief Medical Officer, and Jonathan is now doing a new entirely new position as the Chief Gene Therapy Officer. How does this influence your commercial readiness or just internal workings of Rocket?

Question: Gil Blum - Needham & Company, LLC - Analyst : Great. That makes a lot of sense.

Question: Gil Blum - Needham & Company, LLC - Analyst : Maybe you have started the trend. So I'd like to focus on Danon disease. I mean, this is where we get most of our questions and that's most of the street's focused on. So kind of get that initial question out of the way, when do you expect to disclose the pivotal study design given the studies planned for this quarter now?

Question: Gil Blum - Needham & Company, LLC - Analyst : So it will probably be with this study announcement. Is that a good guess?

Question: Gil Blum - Needham & Company, LLC - Analyst : Good. That's very helpful. And in recent months, the company has talked about different potential endpoints and combinations thereof. Just to help our investors who maybe not as familiar with us, what are some of these endpoints and some potential options?

Question: Gil Blum - Needham & Company, LLC - Analyst : Maybe a bit of a big picture question. Do you feel that there is any kind of change in attitude and the way the FDA views this thing, you know, Peter Marks? And is there any influence from what's going on in other studies and other development pathways? I mentioned you're up there earlier, especially when you're talking about an external control because I think this is kind of the direction that are going in.

Question: Gil Blum - Needham & Company, LLC - Analyst : Yeah. I do have one question that's a bit in the technical leads. So how is expression of LAMP2B currently assessed? I believe it's IHC staining. So is the total amount of protein what matters here? Or is it the expression in many cells or how should we think about this? 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 18, 2023 / 12:45PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Needham Healthcare Conference (Virtual)

Question: Gil Blum - Needham & Company, LLC - Analyst : That makes a lot of sense to me. Maybe shifting gears to the female Danon population. If you can discuss kind of your plans there. I mean it is much larger than the male population, just due to survival.

Question: Gil Blum - Needham & Company, LLC - Analyst : Excellent. And maybe a couple of words on the commercial opportunity here, just to make us better understand how Rocket views the commercial opportunity and kind of how you're getting ready for a potential commercial launch? I mean, it's an accelerated approval pathway. It might not take that long.

Question: Gil Blum - Needham & Company, LLC - Analyst : That all again makes a lot of sense. I mean, this is probably another one of those cases where when you have a drug, you start finding the patients. I mean when we survey, we surveyed the physicians who treat ACM and they have a diagnosis rate of about 2.5%, which is in line with what is in the literature. But as you say, I mean, if you have a drug, people tend to find the patient. The patients tend to find you. It's probably the right way to think about it.

Question: Gil Blum - Needham & Company, LLC - Analyst : So switching gear to the lentiviral programs, which I'm personally very excited about. And I know some investors are happy, super excited about lentivirus -- such a weird angle there. What learnings, if any, have you derived from experiences with other companies launching in the LV gene therapy space? What is there to learn and what is there to unlearn?

Question: Gil Blum - Needham & Company, LLC - Analyst : And maybe talking about what I consider the lead program in this space for you, which is RP-L201 and LAD-I. Are there are some remaining milestones in this program, timing-wise? I mean, I think the data is pretty conclusive at this point.

Question: Gil Blum - Needham & Company, LLC - Analyst : So LAD-I, like I said, really compelling data, but relatively small patient pool. How do you guys think about expanding kind of outside of that initial indication if possible?

Question: Gil Blum - Needham & Company, LLC - Analyst : Excellent. So on Fanconi anemia, maybe a bit of a psychological question when it comes to dealing with patients and physicians. So the treatment has a relatively slow time to engraftment, right? Do you have some patients that start reaching resistance numbers relatively late? How do you think about messaging this particular aspect of the treatment?

Question: Gil Blum - Needham & Company, LLC - Analyst : That's a very important aspect, I think, of this therapeutic and will probably help you with the messaging, particularly when you say that patients you don't need any chemo to get this therapeutic when the standard of care is now ablation and bone marrow transplant. So definitely an appealing concept.

Question: Gil Blum - Needham & Company, LLC - Analyst : Very interesting. Is there any clinical evidence for an outcomes benefit when you compare to standard of care company? I mean, the study has been going on for a while. But that's the premise of this question.

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. And maybe moving on to what I consider a very important lentiviral program at Rocket and that's of pyruvate kinase deficiency. So pivotal study guided for this year. What patient population are you guys considering? Is this pre- or post-splenectomy? Is that mitapivat failures? What is your guiding thinking at this point?

Question: Gil Blum - Needham & Company, LLC - Analyst : So as much as you can disclose, just based on results from your Phase 1 any thinking of what sort of endpoints the company is considering? 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 18, 2023 / 12:45PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Needham Healthcare Conference (Virtual)

Question: Gil Blum - Needham & Company, LLC - Analyst : And for those of us who will look on timing, is it fair to assume that you will tell us about the design when the study starts?

Question: Gil Blum - Needham & Company, LLC - Analyst : And on the data side of it, is it safe to assume we're going to continue to see the regular updates we've seen from the Phase 1?

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. So switching gears -- because I don't want to leave it behind for your cardio franchise expansion, which is a pretty exciting event. So your next day, the program is expected to enter the clinic is the PKP2. Personally, I have no -- I'm not particularly familiar with this indication. I've never heard of it before we started talking about it. Could you walk us through some of the details of this kind of cardiomyopathy and maybe discuss the differences between that and the Danon program if there are any?

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. So definitely some biomarkers to look at. Maybe -- again, a general question here, what type of patients do you see enrolling in an early study?

Question: Gil Blum - Needham & Company, LLC - Analyst : That's fair. And maybe -- you guys have brought in the Renovacor programs. Just an update on where those programs stand? If it's another very interesting target BAG-3.

Question: Gil Blum - Needham & Company, LLC - Analyst : And maybe a question that will be difficult to answer, but any details you can try to provide on the kind of vector you think of using there? Because when it comes to IV administration in AAV, it's pretty important. 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 18, 2023 / 12:45PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Needham Healthcare Conference (Virtual)

Question: Gil Blum - Needham & Company, LLC - Analyst : Okay. That all makes a lot of sense. So kind of a question to round up our discussion today. Given current financial markets, although a couple less days are a little better, what level of spend do you anticipate in the near term on some of these earlier-stage programs?

Question: Gil Blum - Needham & Company, LLC - Analyst : And that actually a decent segue. I mean, we are having a panel with you guys also on Thursday regarding commercial preparedness. In-housing of facilities. So is that really a key differentiator for a gene therapy company?

Question: Gil Blum - Needham & Company, LLC - Analyst : All right, excellent. We are running up on time. As always, it's a pleasure to have you on, Gaurav, very, very clear answers and to the point.