Rocket Pharmaceuticals Inc at Bank of America Global Healthcare Conference Transcript

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

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Great. Yes. Let's talk about the transition to becoming a commercial company should have INDs this year for LAD-1 and Fanconi. How are those preparations going, as you transition to commercial from a clinical company?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. Great. Yes. How should investors think about pricing? There's some analogs out there of previously approved gene therapies, but then when you look at the indications you're going into LAD-1, ultra-ultra-rare and Fanconi is still very rare, but not as rare. Does the prevalence of the patient population play a big role in pricing? And just how should we think about that, especially when people look forward to Danon, which is a much larger population.

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Now with these first 2 indications, LAD-1 and Fanconi Anemia, how is the current state of diagnosis and patient identification? And what sort of work lies ahead of you there?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : And then talking about larger indications, let's change gears and talk a little bit about Danon Disease. For those, who are new to the story, maybe can you just give a little recap of what you've shown today and what impact your gene therapies has been able to have in these patients? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 10, 2023 / 11:20PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Bank of America Global Healthcare Conference

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : That's fantastic. Now you talked about some of these endpoints, which do you think are most important when it comes to a pivotal trial here? And what do you think are the appropriate endpoints that you would like to look at in order to get a trial that would be of a manageable duration.

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. Now I know there's discussions with FDA happening this year on what the trial could be. What other aspects of the trial do you think would be an appropriate design as far as size, patient population, duration those sorts of things?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. Got it. And this is a key point for investors [in the stock] is what's the trial going to look like, how long it's going to take. When could we expect some sort of update on the -- whatever agreement comes into being with the FDA?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Got you. Now since you've been running the current Phase 1, what differences have you noticed, if any, in diagnosis of Danon, general awareness just as a result of there being a potential treatment out there?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Definitely, definitely. So with all those patients lined up, how should we think about timing then once you have agreement on the trial enrollment, I would assume it would be pretty quick and then getting to data...

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. Maybe we can talk a little bit about the PKD program. Where are you out there with PKD? And where would you see it fitting into the treatment landscape, this been, I think, your only indication, where there's really another approved treatment? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 10, 2023 / 11:20PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Bank of America Global Healthcare Conference

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : And what's the path to the market here in terms of clinical progress?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Great. Okay. And then looking at the next wave of Rocket Pharma, you've talked about that, and we'll get into the other program maybe after, but what should we be thinking about in terms of what's next for you guys?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : That's great. Now you mentioned the preference for AAV going forward versus lenti. What's the strategic rationale there? And what are your expectations just around, in general, around durability of AAV?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Yes. Maybe just -- the durability of AAV?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Got it. Now can you maybe just talk us through a little bit your decision-making process when you're evaluating new opportunities and how you land on the programs that you decide to move forward?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. You recently announced that you got IND clearance for PKP2 Arrhythmogenic Cardiomyopathy, it's [mouthful]. What do you see as the potential for this program? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 10, 2023 / 11:20PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Bank of America Global Healthcare Conference

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Great. Now how should we think about timing of data updates here? And I guess, just more generally, I know in the past, I always tell people Rocket is like clockwork every 6 months, every program gets updated, and it's always positive. So should we expect that sort of pace of updates from this program in future programs going forward?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Great. I'd like to hear that. Next week, you have a number of presentations and posters at ASGCT. What should we be focusing on when we're looking at the updates that you're going to provide? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 10, 2023 / 11:20PM, RCKT.OQ - Rocket Pharmaceuticals Inc at Bank of America Global Healthcare Conference

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. Great. And then just one last question sort of about to wrap up. How should we think about the overall commercial strategy ex U.S? You talked about global commercialization in these initial indications. Does that hold for as you get into larger, larger diseases?

Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst : Okay. Great. Well, with that, I think we'll wrap up. So thanks so much, Gaurav, and thanks, everyone out there for listening.