Rocket Pharmaceuticals Inc Investor Webinar Transcript

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

Question: Thibaut Pardo-Garcfa - LifeSci Capital LLC - Analyst : Can you hear me?

Question: Thibaut Pardo-Garcfa - LifeSci Capital LLC - Analyst : This is Thibaut Pardo. I'm stepping in for Cory. So my first -- my question would be what information is being precluded by using different imaging techniques, for example, the fibrosis. And will this have an impact when aggregating the data as a whole or making any statements on the benefits of RP-A501 in the heart?

Question: Gregory Harrison - Scotiabank Global Banking and Markets - Analyst : Hey, good afternoon guys, congrats on the update. Looks really good. First, I wanted to ask about patient 1001, and if you've identified any reasons why the patient didn't have any expression detected at month 30 and 36 even though it appears patient is doing well and has expression now. And then if you could provide any additional color on patient five and the grade four adverse events seen with him, that would be great.

Question: Tyler Van Buren - TD Cowen - Analyst : Hey, guys, thanks for the presentation. Great to see the long-term evidence and maintenance of these improvements in these patients. So expression increases up to 36 months in some patients, but LV mass index improvement seems to be largely stable beyond 12 months, which I suppose is good confirmation of the 12-month endpoint for the ongoing pivotal. But curious to get your thoughts on a couple of things. So first, just on the relationship of expression to LV mass index improvements and if you would expect further improvements in some of these patients beyond 12 months that maybe aren't at beyond a year, three or beyond yet? And then second, if you believe it makes sense to wait to report the pivotal data until all patients have past 12 months, or if six to nine months could make sense as we're seeing improvements in most patients by that time range?

Question: Tyler Van Buren - TD Cowen - Analyst : It was just on the pivitol data, if you think you need to wait for the 12 month primary endpoint to report it or if you -- six to nine months could make sense to report it as a lot of these improvements in those patients are being observed by that time range.

Question: Mani Foroohar - Leerink Partners - Analyst : Thanks for all the questions and for the update, very remarkable long-term follow-up data. Certainly for the longest that we've seen in gene therapy coverage. So I have a little bit of a different question, although similar in some ways to Tyler's. So -- it's very reasonable to think the positively releasing data prior to the 12-month defined endpoint could be premature. I hear you, Kinnari. But if you see a number of patients who reached 12 months, cross the defined threshold, which is greater than the number of the patients you need in the study to have a positive outcome, i.e., seven versus required -- seven required, for example, whatever the number is. Could you then potentially engage with regulators proceed with a rolling submission? Would that be an interim analysis? If you have enough responders that a positive result is statistically inevitable. Does it not make sense then to stop the study early, given that there's no risk of unblinding because it's not a blinded study.

Question: Mani Foroohar - Leerink Partners - Analyst : That makes sense to me. I'm going to quickly slide a second one in here. I know that I'm sort of overstaying my welcome a little bit on the Q&A. So you brought up fanconi, I think one of the debates that we have in kind of the current uncertain regulatory environment is, obviously, those companies that are more well capitalized and less subject to capital markets risk tend to do better. And I've had a lot of discussions with people around what is the value of a PRV and how -- on what time horizon you at Rocket may receive one or more PRVs. So could you give us a little bit of an update of where you guys are in terms of addressing LAD-I, progress towards potential PRV eligibility for eventual Fanconi approval, et cetera? Like what are the time lines for those programs? And how should we think about PRV award and your views on that end market for those assets?

Question: Mani Foroohar - Leerink Partners - Analyst : Congrats again.

Question: Michael Ulz - Morgan Stanley - Analyst : Congrats on the long-term update as well. Maybe just a quick one on the LV mass reduction, in particular, for patient 1006 looks like they met the threshold that I think it was 12 months and continued to improve over the longer period, but then sort of reversed a little bit. Anything unique with that patient that you can call out or explain that?

Question: Eric Joseph - JPMorgan Chase & Co - Analyst : Thanks for taking my question. Thanks for updating us, this term follow-up. Maybe I just had a question on the pivotal and the treatment of change in LVMI as part of a co-primary endpoint. Just in calling out to -- trying see a mean of greater than a 10% decline in this study. Can you just talk about sort of how that's being calculated or how your status plan treats that? Are you, just wondering how sort of accommodates for any kind of outsized size performing patients on LV mass decline and whether a responder analysis is also part of that assessment.

Question: Dae Gon Ha - Stifel, Nicolaus & Company, Incorporated - Analyst : I'll add my congrats on the long-term update looks robust. Maybe a question -- a follow-up question and maybe a short clarification. Following up on the earlier question, Jonathan, when it comes to cardiac MR and echo has it been clearly defined with the FDA. I guess, how much of a wiggle room there is between some patients who might be measured with an echo versus cardiac MR. And maybe if you can expand on the sensitivity of these two assessments, understanding that ICD is what's going to move the decision play between using either of these metrics. And then a clarification is with the Phase II fully enrolled, I just wanted to get your latest thoughts on when the late -- the completion of dosing could happen. Thanks. 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 18, 2024 / 5:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc Investor Webinar

Question: Richard Law - Goldman Sachs Group, Inc. - Analyst : Hey, guys, good afternoon. Congrats on the data. Gaurav, you guys presented the data in a few places today, including AHA, this webinar and also publication at the New England Journal of Medicine. Can you highlight in contrast any differences or additional insights of presentation? And then I have a couple of follow-ups.

Question: Richard Law - Goldman Sachs Group, Inc. - Analyst : I got it. And then for the LAMP2 expression, since the band within each grade are pretty wide, can you discuss like how the LAMP2 expression trended over time within each of the grade? Or I don't know if you can provide like numerical values. I think especially for like say, patient two and three that drop from nine months to the later months. And also what is the standard deviation or variability in LV mass reduction or LAMP2 expression in these measurements.

Question: Richard Law - Goldman Sachs Group, Inc. - Analyst : I see. Got it. And then just one last question. Can you discuss any screening efforts that you have implemented so far? And also what other screening programs are you planning in the pediatric or newborn space?

Question: Jason Zemansky - BofA Securities - Analyst : Thank you, good afternoon, congratulations on the data. I wanted to circle back on one of your comments earlier regarding LAMP2 expression. I appreciate there's a level of variability here given a lot of parameters. But after five years, do you have a sense of ultimately how long protein expression is likely to be sustained, especially given that myocyte turnover is low. I guess, fundamentally, ultimately, what level of LAMP do you think is necessary to provide some of these longer-term benefits to kind of maintain them maybe decades into the future.

Question: Jason Zemansky - BofA Securities - Analyst : I guess to kind of layer on another question. Is it not necessarily the extent of production per cell, but overall, the number of myocytes that have been transduced?

Question: Gil Blum - Needham & Company - Analyst : Hey, good afternoon, then. Allow me to add my congratulations, a very impressive long-term follow-up here. So just a couple from us. Specifically as it relates to the high-dose patient, that appears to not have any dose dependence, which supports the use of the lower dose. Any thoughts as to why it kind of seems like a overall response is a little lower? Does this have to do with the patient's baseline? And I have a follow-up.

Question: Gil Blum - Needham & Company - Analyst : And maybe a closely related question. So in your view, is there a patient baseline dependent ceiling effect for left ventricular mass reduction.

Question: Joohwan Kim - Canaccord Genuity Corp - Analyst : Thanks for taking our question, congrats on the positive data. This is Joohwan on for Whitney Ijem and maybe two slightly different Q's cues from us. First, given the great longer-term data shown today, what's your latest thinking around a female Danon trial? And secondly, you touched on this a little bit before, but can you speak on any read-through you might have from these data and durability to the PKP2 program despite the different capsids?

Question: Samantha Corwin - William Blair & Company LLC - Analyst : Congrats on the long-term data. I know it's a bit early to speculating about what the label might look like. But do you think that the label could potentially exclude patients with reduced EF? And what percentage of patients do you think have reduced ejection fraction? And then given 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 18, 2024 / 5:00PM, RCKT.OQ - Rocket Pharmaceuticals Inc Investor Webinar you announced a couple of months ago, the completion of enrollment in the pivotal trial. I guess, what are the current bottlenecks in terms of treating those patients?

Question: Samantha Corwin - William Blair & Company LLC - Analyst : Great, thank you.