Conference Transcript - RCKT 2026-06-04

Andrew Tsai, Analyst — Jefferies

We're going to get started with our next session. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies. Thanks for listening, and it's my pleasure to have the Rocket team with me. To my direct left is Gaurav Shah, CEO, and to his left, Meg Dodge, SVP of External Affairs. Welcome, both of you.

Gaurav Shah, CEO

Andrew, thanks for having us.

Andrew Tsai, Analyst — Jefferies

Great to be here. Yes, yes. So there may be some people in the audience who are less familiar with the Rocket story, so can you help us level set where you are, what programs you're working on, your strategy, and then catalyst milestones over the next 6 to 12 months would be very helpful.

Gaurav Shah, CEO

Great. We are Rocket Pharma. We are a cardiac-focused genetic medicines company, primarily focused on genetic cardiomyopathies that while each one is a rare disease together, they're not rare. Each disease is rare, but rare disease is not rare. It ends up becoming very common when you add them all together. So our focus is on three genetic cardiomyopathies. One is a hypertrophic one called Dannon disease, a disease of autophagy that primarily affects both boys and girls, but boys in their early years and females later in life. We are in a pivotal trial aimed at an accelerated approval path in agreement with FDA for that program and are actively enrolling patients right now for the safety-running portion of that. The next program is an arrhythmogenic cardiomyopathy called PKP-2, which is the most common type of ACM, which is the second big bucket of cardiomyopathies. In that program, we're in a phase one trial. We've found the pivotal dose, and we're in discussions with FDA regarding the next steps for an accelerated or full approval pathway. The third cardiac program is BAC-3 dilated cardiomyopathy, a devastating disorder, multifactorial in nature with regard to the protein. And in that program, we are entering phase one. We're about to start treating patients. So three big programs in cardiogenetic medicines. Historically, Rocket has been a two-platform company, cardiac being one with an in vivo AAV approach. the original programs in the company were bone marrow programs where we use an ex vivo lente technology led1 fanconia anemia and pyruvate kinase deficiency we're very happy to get the first FDA approval from CBER this year for led1 with a product called Creslati which were we got done through the accelerated approval pathway and we're in the process of getting it that out to patients as soon as possible. That also came with a large PRV sale which allows us to have cash run away for another two years into second quarter of 2028. So we are a genetic medicines company focused on cardio but also have a legacy programs in ex vivo lenti bone marrow derived disorders and moving forward we're going to continue to focus on cardiac build that out. We have other programs that are behind the scenes and are also rare but you add them all together and we're talking about hundreds of thousands of patients and really opening the door to cardiovascular genetic medicines here

Andrew Tsai, Analyst — Jefferies

right hundreds of thousand patients on a gene therapy price it's a big huge opportunity okay and so starting with some come some bigger picture questions you know when when we think about gene therapy you know it's gone through it like this important maturation period over the past few years, safety, manufacturing, clinical execution. So how are you guys thinking about these aspects, and what differentiates you from other gene therapy players out there?

Gaurav Shah, CEO

I would say primarily we are very proud of the fact that we invested early on in an in-house AAV manufacturing facility. And what this allows us to do, in addition to having a long-term path to reduce cost of goods and good margins, is that it allows us to develop the process and the release criteria and potency assays and all those aspects of things in our hands. And control is the most important thing with regard to CMC discussions with FDA, because often there's back and forth with FDA. It's iterative dialogue, and having it in-house really accelerates those pathways and also ensures that we control the product and not somebody else. So we're very proud that the Danon program is being produced in-house and the other programs will be as well. I would say that's one big differentiating factor. The other is just that we have the experience and we've gone through these programs with multiple patients. We've developed a muscle memory for how to treat these patients, how to read labs, how to pivot around safety issues, how to maximize efficacy, and for example, how to read protein expression. So I think out of the companies in this field, we have the most in-depth experience with regard to those key aspects of drug development.

Andrew Tsai, Analyst — Jefferies

Very good. Makes sense. And then...

Meg Dodge, Other

I can add a few words, Andrew. So I think the sector as a whole for gene therapy is also in a period of execution. I mean, we got earlier guidance this week from FDA. And so if you compound what it's going to take with respect to execution, that means that we as an industry we have developed our science tremendously, but it's going to be a matter of manufacturing, as Gaurav said, clinical engagement, and also regulatory engagement. And FDA has really tried to step up, and so I think we're in the next, in a period here next of working with the agency to really bring genetic medicines to the forefront on both the development side, the manufacturing side, as well as the regulatory front.

Andrew Tsai, Analyst — Jefferies

Right, you guys are very thoughtful and careful about all these, thinking things through. And then to your point, like the FDA, I think they released a new guidance document or something?

Meg Dodge, Other

On Tuesday, yes.

Andrew Tsai, Analyst — Jefferies

Okay, so FDA, you know, really wants to collaborate with you guys. Okay.

Gaurav Shah, CEO

Yeah, I would also say that guidance documents are important and they're a good template for us, for the whole field. At the end of the day with the FDA, it's about individual dialogues and collaboration with the review team. We've had a track record of being engaged respectfully both ways and getting to a good landing spot for all of our programs,

Andrew Tsai, Analyst — Jefferies

and I anticipate that'll continue great and so maybe before we touch on the Dannon program which to me is the core thesis of your story but congratulations on Chris Lottie's approval though where you earned a PRV voucher so how are you thinking about you know the sequencing treatment center onboarding and the overall TAM of this product when can we learn about when you might launch the pricing strategy the reimbursement strategy and so forth I can take this

Meg Dodge, Other

one so what we tried to do and what we aimed to disclose to the street and to the community in 2025 was a reprioritization of the pipeline and that's when we defined ourselves as being a cardiovascular focused company and so with that we had Chris Lottie at the finish line with a potential FDA approval kind of on the cusp and so what we really wanted to do was get that on the across the finish line and then be able to talk to partners about getting those lentiviral programs into the hands of someone that's better able to commercialize them so we've gone through the steps to to prepare to make Chris Laudy available to patients we're talking to potential partners about assuming that program however in the event that you can't find the right buyer for what I call a custom-built house we're prepared and available we're prepared and able to make Chris Lottie available to patients and so if you're asking about our preparation to be able to make Chris Lottie available to patients that'll be later in 2026 towards the tail end of the year got it

Andrew Tsai, Analyst — Jefferies

got it and so you know when I think about how this reads across to your other programs on you know should you launch gain payer access reimbursement acts gain experience launching feels like there could be potential scale as your other follow-on programs become approved is that kind of fair and

Meg Dodge, Other

anything else you dad yeah consider it muscle memory once you go through the process of developing your in-house commercial commercialization team and going through the steps to have a company be able to provide access for a therapy you can apply that to other programs so yes it's building the infrastructure but it's also developing the team the know-how and the skill set to be able to do that directly so you can apply it to other programs right

Andrew Tsai, Analyst — Jefferies

okay very good and so shifting gears to your Dan and program it's great to see that it's been resume but bigger picture for help us frame the US market to opportunity of Dan and how many US patients you think they are how many diagnosed identified for instance any just just so we can have a sense of the

Gaurav Shah, CEO

peak sales opportunity here so I would say that we will be able to answer that question in much more depth during a full program update that we anticipate second half of this year we will have a top-down breakdown of the Dan and opportunity that's based on epidemiology as well as genotyping and we'll also have a bottom-up approach going out there and seeing where these patients are and who So we'll have that update. I would say that historically we've stuck by the number of at least 15,000 patients in the U.S. and Europe. It's Dannon is a frequency of hypertrophic cardiomyopathy and you can extrapolate from the HCM population to figure out how much is true Dannon disease out there. There's a big gap between true prevalence of Dannon and actually diagnosed Dannon because because Danone disease was identified as a genetic cardiomyopathy as recently as the early 2000s. So there just hasn't been that much time for people to understand what Danone is, how it manifests, and also even doing genetic testing for cardiomyopathies is a relatively novel thing. So there's going to be a gap between true prevalence and diagnosed. So even beyond this update, our next step ahead of the commercial launch is to find these patients through genetic testing and other means. so we'll answer the question but that's sort of the big picture answer okay very

Andrew Tsai, Analyst — Jefferies

good and so can you remind us in the phase one program which led you to have this accelerated approval pathway within this phase two pivotal that you're doing what did you see in phase one for these Dan and patients how many patients on efficacy on the phase two kind of co-primary endpoints what did you see in phase one that gives you the confidence you'll make it through the goal line in

Gaurav Shah, CEO

phase two? In phase one, we treated six patients who were followed for more than one year. And out of those six patients, we had results that were published in the New England Journal of Medicine about a year and a half ago. And those results showed that all six patients had florid protein expression. In fact, a couple of them out to five years or more. We showed that all of them had a reduction in left ventricular mass index, LVMI, which is now agreed upon as one of our co-primary pivotal endpoints. In fact, protein and LVMI are the co-primary endpoints for the ongoing pivotal trial. In phase one, we showed that all six patients would have met that endpoint at the one-year point if this were a phase two trial. So that gave us a lot of confidence to move forward into phase two. Also, those patients showed massive reductions in troponin and BNP for as long as we followed

Andrew Tsai, Analyst — Jefferies

them until the NHAM paper great so 100% success rate basically in phase one and so then you started the phase two although you faced a little slight hiccup and last year with the you know clinical partial clinical hold but then you got it resolved very quickly within three months and then now you're resuming it with the safety initial safety cohort now and so can you kind describe what happened what what learnings have you had on that with that experience so early in

Gaurav Shah, CEO

the phase 2 trial we saw a higher incidence of thrombotic microangiopathy TMA than we would have anticipated or that we saw in phase 1 and in an effort to further mitigate the risk of TMA and by the way the patients who had TMA and early in the phase 2 trial they've resolved the TMA and those patients are fully resolved with regard to the safety issues and hopefully moving on to look at efficacy. But to address the TMA, what we did is we added a C3 inhibitor. And the issue that we faced as soon as we treated patients with the C3 inhibitor is that there was a paradoxical unlocking of direct endothelial damage with the combination of a torrential rain of AAV9 plus a C3 inhibitor. It's not what we expected, but there's no good preclinical models for cardiomyopathies, and some of these things play out in the real world and the clinics. So we had an unfortunate case of a patient death due to the combination of C3 plus AAV9. So in the resumed trial, which we negotiated with the FDA very rapidly, lifted the hold in about three months. We agreed to remove the C3 inhibitor. That addresses the capillary leak syndrome, the endothelial damage risk that we saw with the C3. It keeps the TMA risk on. In order to mitigate the TMA risk further, we did an analysis of the patients and the product, and we determined together with FDA that the phase two product that we were using was likely higher in potency than the phase one product because it was an enriched product with high or full-empty ratios. And because of the high or full-empty ratios, the potency applied to both efficacy and safety. So we recalibrated the dose to a lower dose to match the phase one potency. So while the new dose appears lower in number, it's around 4E13, it is intended to match the dose of the phase one product, which is closer to 6.77E13. So we're moving forward with a recalibrated dose, absence of C3, and a couple of other tweaks in patient selection, as well as drawing out the doses of rituximab over time to further eradicate B cells ahead of treatment. And that trial has resumed.

Andrew Tsai, Analyst — Jefferies

It makes a lot of sense. We should not expect a capillary or leak syndrome because there's no C3, and then TMA risk is heavily reduced because now we are back to the phase one equivalent dose okay great and so now you're trying that out in the initial batch of three safety patients have you dosed the first patient by chance and when does the

Gaurav Shah, CEO

third patient get dosed exactly we have initiated dosing we haven't guided on exactly when we'll finish but we'll have an update a comprehensive update as

Andrew Tsai, Analyst — Jefferies

mentioned in the second half second half okay and is it fair to say that TMA generally pops up pretty immediately after dosing so if you see something I don't know how to ask it but is it true and so yeah it's no news good news I

Gaurav Shah, CEO

would say no news is good news TMA usually manifests in our experience in the first two weeks or so so yeah great we'll we'll have a comprehensive update

Andrew Tsai, Analyst — Jefferies

as soon as we can so and it makes sense why you'll share a comprehensive update in second half my understanding is the initial three patient cohort are stagger dosing a month apart then there's a strategy to talk to the FDA then you come back to the street in that update that's correct discussions that's

Gaurav Shah, CEO

And to be clear, the FDA has asked us for a three-patient safety run-in. They have not commented on what the rest of the trial would look like or that it should So we don't anticipate it would change, but we do want the optionality of discussing with, again, what the best trial design is moving forward. The original agreement was a 12-patient single-arm trial with a co-primary of protein plus LV mass index. We have no indication that that's going to change, but we were asked to go back after the safety dialogue So we want to make sure that we do our due diligence there before right right understood

Andrew Tsai, Analyst — Jefferies

And so as you dose established what's needed and then you dose more patients Which I understand it can be done not sequentially, but just all all at once Then you know as I think about The likelihood of success, you know sure optically the phase two dose now seems like a lower dose but at the same time it is the phase one equivalent dose where you saw a hundred percent success rate so confidence should remain unchanged is that the line of thinking from you guys we are hopeful both on

Gaurav Shah, CEO

safety and efficacy just seeing what we've seen through our experience that this phase two is intended to match the phase one and if all goes well you know I think the safety overhang is is the main thing to get through in the near

Andrew Tsai, Analyst — Jefferies

term. Right. And should, so once you talk to the FDA, I think, um, can you just remind us then the original plan for an accelerator approval, how many patients out to one year was the original agreement? And so, you know, best case in your minds, how many patients left after the three patients are dosed would you need per, to fill that original agreement? I would anticipate that

Gaurav Shah, CEO

that they probably want to see all the patients in the trial treated with the same product in the same way. So while we have started treating patients at a different dose and slightly different immunomodulation profile, I would anticipate that we might have to treat 12 additional, you know, total of 12, 3 plus 9, but we'll see what happens. It's a discussion.

Andrew Tsai, Analyst — Jefferies

But then there could be an upside surprise given your base case, okay. Yeah, and so second half, 2026, you'll come back with a seemingly major update because it's not only next steps, but also top-down or epidemiology. And then I'd assume you'll share the bar for success, too, from that FDA meeting, to be clear.

Gaurav Shah, CEO

Yes, absolutely. This is an update that's been long in the making, and because of the event last year, It got delayed, but the update would include, certainly, status of the trial, next steps for to finish the pivotal trial, and epidemiology, bottom-up, top-down, and also an understanding of what it's going to look like to have a win on the trial. All of that, we'll have a comprehensive update.

Andrew Tsai, Analyst — Jefferies

That's correct. Thanks. Okay. and so then shifting gears to then PKP2 well we'll just wait for Dan and and fingers crossed everything turns out right and so PKP2 maybe talk to us about the disease indication how big is this indication relative to Dan and we

Gaurav Shah, CEO

can go from there yeah PKP2 now borders on what is in the common common knowledge base or common uh sort of spirit of how we understand cardiomyopathies these are patients who often have sudden cardiac death or palpitations at a young age except especially when exercising so this is becomes a cardiomyopathy that we are familiar with in day-to-day life right athletes and runners and and others so this pkp2 is a disease of the junction between cardiomyocytes which are not in ideal condition because of the lack of PKP2 protein. And because of this, these patients have recurrent arrhythmias manifesting as PVCs, NSVTs, T-wave inversions, and often fatal arrhythmias for which they need ICDs placed. 80% of these patients do have ICDs placed. So the population here is at least 50,000 in the U.S. and Europe. So it's, again, rare, but not so rare. It's one of the more common types of cardiomyopathies out there. And the program is now at the Phase I is ongoing, but we've treated all the patients we need in order to go to the FDA with the Phase II trial design and approach. All three of our Phase I patients had manifestations of protein expression, improvements in arrhythmias, and general improvements in clinical status out to at least a year. So with that information, we've gone to the FDA. We're in discussions with them as to what a pivotal trial would look like, and more on that in the second half as well.

Andrew Tsai, Analyst — Jefferies

And with the phase one data set, how would you say you compare to competitors, actually, on those efficacy measures?

Gaurav Shah, CEO

So I've seen the data from others in the field. I should first of all say that because it's a more common rare disease, there is room for more than one treatment. There's a lot of patients out there with a high-end met need. Having said that, we're very, I guess, we're reassured by the fact that all of our patients that we treat, although they were only three patients, they saw the improvements that I described pretty uniformly. There was no patient who got worse suddenly or certainly. so I think we're positioned well to be in the lead for this program and it's not always about the number of patients treated but having the right types of patients and the right endpoints to lead to a win in a trial if the drug works

Meg Dodge, Other

right and I think we're in pole position for that I'd also add that in the phase one we align with what we feel is the proper dose to proceed with with respect to the pivotal phase two study so we feel that we're moving forward with the phase two with the dosage that we used that was efficacious in the phase one.

Andrew Tsai, Analyst — Jefferies

Gotcha. So the phase one cohort was three patients. Are you dosing more in the meantime as you talk to the FDA to be clear in phase one?

Gaurav Shah, CEO

Yeah we will dose more patients not because we need to do that for the phase two discussion but because it just gives us more information. There are patients who are waiting for the treatment so we're opening the trial up again, but it's not necessary for the FDA dialogues themselves.

Andrew Tsai, Analyst — Jefferies

Male Speaker 2 Right, and I guess for the initial batch of three patients, would you consider sharing data is my question for the additional phase one data, not only on the new patients, but also longer follow-up. Is that the second half event?

Meg Dodge, Other

Female Speaker 2 Potentially, yeah. We would like to show the long-term follow-up results from those three initial patients, And then, as Gaurav was alluding to, having additional patients enrolled and treated in the phase one at the same dose that we plan to proceed with will be further support of a BLA submission later on down the road.

Andrew Tsai, Analyst — Jefferies

And, you know, with your Chris Lottie experience, your Danann experience, so far, just with your FDA discussion, it seems like you have a know-how or advantage or a good relationship with the FDA on study design, long, big picture. so PKP2 is that kind of the strategy of you know I think Dan and the original agreement was 12 patients and that's it and is that kind of the strategy here

Gaurav Shah, CEO

for PKP2 for you I would say Dan and is a one-off very aggressive cardiomyopathy with an easy to understand pathology this is a disease of big hearts so it makes sense that the endpoint here should be shrinking of the heart that That made a lot of sense. It's also a disease that rapidly progresses in natural history unequivocally. So that leads to an easier dialogue when it comes to justifying a single-arm small trial. I don't think that's going to apply to any of the rest of the genetic cardiomyopathies, including PKP2 or BAC3 or others. So it's a different dialogue from Dannon, and I would not expect the same outcome.

Andrew Tsai, Analyst — Jefferies

I see. and any color how FDA discussions are going by chance I would just use the

Gaurav Shah, CEO

word collaborative sure and and I think in all of our programs with the review team that that we've been assigned especially for these programs it's always been sitting at the table figuring out how to design a trial where we can win if the drug works not designing the smallest trial or trying to have the biggest short-term win it's about getting the drug approved in a way that the FDA agrees with and it's also we try to go for written communications there as well so while it takes time I believe that we're gonna get to a good

Andrew Tsai, Analyst — Jefferies

outcome here great and then lastly the back three program sounds like you're starting phase one backtrack one more time back three how many patients you think there are and in this phase one what what is what is a good good no-go

Gaurav Shah, CEO

no-go scenario for you so back three as a patient population is probably somewhere between Dan and PKP two in terms of the the prevalence in the US and Europe our phase one program the IND is cleared we're going to start treating patients soon and the cohorts are designed as three plus three so three patients unless there's a DLT we may elect to treat more than three patients if the first dose looks like it's working so that we can expand the trial and get more information in anticipation of a pivotal phase two discussion like for PKP2.

Andrew Tsai, Analyst — Jefferies

I see. And what would be a reasonable efficacy endpoint?

Gaurav Shah, CEO

So dilated cardiomyopathies are part of a better trodden path with drug development. Because the left ventricle is dysfunctional, it's easier to come up with endpoints where the left ventricle improves, such as EF, peak VO2, and even exercise testing. So there are ways to measure the left ventricular function in a predictable way where there's drug development expertise and regulatory precedent already in place, whereas for Dan and we have to have these discussions from scratch with the FDA and come up with a new endpoint. So we feel good about once we get to that point to have an easier dialogue with the agency.

Andrew Tsai, Analyst — Jefferies

I see. Okay. And then with the pre-resale of Chris Lively, the picture, you have cash, I think you said until second quarter of 2028? So you can execute on a lot of these milestones starting in second half of 2026, it sounds like, across these programs.

Gaurav Shah, CEO

Yeah, lay low and get things done. That's the motto this year.

Andrew Tsai, Analyst — Jefferies

Okay. Was there anything else you wanted to add, but otherwise?

Gaurav Shah, CEO

I don't think so I think we're we're at the beginning of a journey for cardiac genetic medicines and we're very very excited about the future yes much

Andrew Tsai, Analyst — Jefferies

respect to you for pioneering these spaces and you know best of luck into second half thank you Andrew thank you everyone