Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference Transcript

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

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. So maybe let's start with 201. So congrats on the data. You anticipate filing first half of '23. Can you just walk us through the next steps? What has to occur there behind the scenes from your point of view? What do you need to do in terms of the process with the regulators? Flesh that out for us.

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : I mean the data themselves are very clean. I think the only question I had was you had one case of grade 4 PAH. Any comments there?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : And can you remind us of the market opportunity for severe LAD? And how well identified are these patients? And you must really be thinking about sort of the commercial infrastructure now and the strategy there. So can you walk us through that?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. And then from a manufacturing point of view, where will this be manufactured in your Cranbury, New Jersey facility? And I guess just as a broader question as you're moving towards being a more commercial company, can you just talk about your broader manufacturing capabilities?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : And again, just given your -- the proximity to your interactions or sort of more formal interactions towards approval with the regulators, I guess what have you observed over the last 12 to 24 months? It's been an interesting time in sort of cell and gene therapy. And have you -- does it feel like there's heightened scrutiny or trepidation on the regulator side? What can you say there?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : That's great. So maybe we should switch gears and talk about 501 and Danon. So what are your expectations here with earlier intervention in the pediatric patients contrasting to what we've seen with adults?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : So it sounds like you are truly age agnostic. Is there -- how early in the disease course would you go? Would you treat a genetic diagnosis at some point? Or...

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. And then, I guess, one consideration around that is the sort of durability of benefit. And so is this a one and truly done therapy? Or do you think the benefit will weigh in over time?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : A question here. So in the literature, there are studies that show steroids can increase autophagy, you see any effects from immunosuppressants on patients in the ongoing trial?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. You have an ongoing natural history study. I'm curious like what are the key observations that have come out of that study to date? 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 24, 2022 / 11:45AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. A few more questions. Can you tell us about the choice of reporting posterior wall thickness and septum thickness over other echocardiogram parameters?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Another question here. Is this protein expression a potential end point? You've shared protein expression quantified by both immunohistochemistry and Western blot and there's quite a lot of variability between the 2 techniques, which do you view as more accurate and why?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. When do you expect the female trials to begin? And could you just contrast that with the male side?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : And then just as we think about the overall opportunity here, sort of 5, 10 years down the line, what is the overall target population in Danon that you expect to be treating?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. Maybe we'll switch to 102 now. So again, congrats on hitting the primary end point. Could you just walk us through the path forward here in terms of the regulatory discussions, et cetera? How are they going? When do you anticipate this being on the market? 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 24, 2022 / 11:45AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. Great. And remind us there, what's the optimum age for intervention? Is there a cutoff?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : And maybe just lastly on Fanconi. Could you just again frame the size of this market for us?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. And then lastly, in the pipeline, 301, what's the next update there? Just -- and when should we expect it? 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 24, 2022 / 11:45AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : And not that you don't have enough going on already, but as we think beyond your current pipeline, what's at the back of your mind, what areas of interest to you?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Very good. And then just can you remind us cash position, cash burn, the runway it gives you?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : And then just the IP for the portfolio for 201, 102, 501, just remind us of where that is?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : For all of the programs. 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 24, 2022 / 11:45AM, RCKT.OQ - Rocket Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. Great. And then one question I've been asking all the companies so far is just the -- this is an interesting time in biotech, just given what's happened with the market, et cetera. And there's a lot of discussions of -- around the strategics and their behavior towards the smaller cap companies. Just -- has there -- from your seat, has there been any change in the sort of tone of interactions with the large caps in terms of what they're looking for? Does it feel like they're more looking to be risk-sharing partners versus potentially just kicking the tires for a more definitive transaction?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Fantastic. I think we're right at the end of time. Any sort of (inaudible) in your point of view, it's -- you've got a lot going on, you've had a lot great data. So...

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Yes, fantastic. We look forward to seeing it Blossom. Thank you.