Rocket Pharmaceuticals, Inc. (RCKT) Earnings Call Transcript
September 10, 2020
Earnings Call Speaker Segments
Great. Good afternoon. Thank you, everyone, for joining us today. My name is Mohit Bansal. I'm one of the biotech analysts here at Citi, and I have a pleasure to have Gaurav Shah from Rocket. He's the CEO of the company with us. Thank you, Gaurav, for joining us today.
My pleasure, Mohit.
Great.
Good to see you and thanks for having us today.
Thank you. And those who can see us, you can see that Gaurav is sitting right next to fireside. So we are having a fireside chat today, literally. So I hope you enjoy the conversation. [Operator Instructions]
So with that out of the way, Gaurav, just why don't you start with a brief overview of the company for those who are new to the company, in terms of your disease focus, the platform approach, multi-platform approach and what -- versus what we generally see with the gene therapy, and you are actually going after both lentivirus as well as AAV virus. So just wanted to get your thoughts on the company, where it is going and other stuff.
Great. Thanks. Yes. I am by a fireside and taken the meeting literally. So the -- this is our fifth year at Rocket Pharma. We started in 2015. We're 5 years in. I'm very happy to have announced recently that we now have 5 clinical programs. All 5 are the ones that we started with, are in the clinic. 2 of them are -- in fact, are in registration-enabling studies and the other 3 in Phase Is. This is an exciting time, I would say the next -- certainly the next few months, but the next 18 months or so of Rocket is a very exciting time because we will have multiple data readouts in these particular programs starting at the end of this year and into next year. And also, we are now building up our internal manufacturing capabilities for AAV and also additional research and development space near Princeton. So those activities take up a lot of our time and are very, very exciting developments for the company over the next 18 months or so. In terms of who we are and why we are, we are a gene therapy company. We are multi-platform or platform agnostic, depending on how you want to think about it. What we try to do is we try to identify clinical assets. We start with the disease first and work backwards. We try to call assets where there's a clear clean, elegant mechanism of action where you can target a particular protein in a particular cell type that is responsible for the full spectrum of disease. So the obvious example is Danon where we target cardiomyocytes. And we target a protein called LAMP2, which is involved in autophagy and the lack of which leads to essentially the full spectrum of Danon disease, not just in the heart, but in other organs as well. Similarly in Fanconi Anemia, it's a disease of bone marrow stem cells, and we directly target the FANC protein in stem cells in the bone marrow. So there aren't many diseases like that. That's the way we select them, however. And in addition to the clear mechanism of action and starting with the unmet clinical need first, we focus on diseases where we can get to a reasonable clinical endpoint in a time that works for regulatory approval. So in the use of thousands of rare diseases, this whittles that you narrow down to few diseases. 5 of these are in the clinic, like I said, and we hope to pan the pipeline in time. So that's the general overview of the company and who we are.
So I mean, this is very good actually, helpful for us. Gene therapy companies are often kind of criticized for basically, many of the companies try same things, and they're going after same diseases. Like if you look at hemophilia, there are 3, 4 players out there. You look at [ DMV, ] there are so many players out there. But you have been able to find these niche indications where not many people are going. So one, just wanted to understand, one, how do you find these targets? And is that your approach that going after being a first-in-class, less traveled paths, those kind of -- that kind of approach? What exactly is the approach there?
Yes, I'm glad you brought that up. So we try to be first, best and only in class. Only in class is an interesting idea. Drug development takes time. Look, gene therapy is complex. It's -- there's so many unknowns. We're learning this field together. A group of academic experts as well as a group of companies like ours, we're all learning this together. And often, we learn from one another. So being only in class allows Rocket to do things the right way, take our time, not cut corners, make sure that we're starting -- at the end, we're starting to think about the number of patients we have to treat where the true unmet need is, how to manufacture product for that population then working back to think about what a pivotal trial's like, what the right endpoints are for that, what patient population you want. Then working back to think about what a proof-of-concept would look like, and what kind of biomarkers and clinical endpoints you would want to put into your Phase I or POC study. And none of this can really happen if you're always trying to rush to prove something. It takes time, and that's why the thesis of being only in class is important. It is true for the current 5 assets, and as we seek to build the pipeline in the future, that will also be an important part of our asset selection thesis.
Got it. And then the other part of the -- which is fascinating about the companies that you are also virus agnostic. I mean, you have AAV programs, you have lentiviral program. And also, I mean, diseases -- areas where you are going in Fanconi Anemia, you're going in various different diseases as well. So is there a common theme because companies are trying to be either neuromuscular disease companies or XYZ companies. But in your case, diseases are different, viruses are different. So what exactly is the common theme there?
Yes. So...
Or do you have to have a common theme? That's the other question, maybe.
Yes. I mean, I think the most important common theme is sort of what we talked about being only in class, clean mechanism of action, where a little protein can go a long way. Many of these diseases are loss of function diseases where you don't have to overuse the protein so much that it's not feasible with the current gene therapy approaches, loss of function-type path. Beyond that, we're not focused on a particular platform. We know the lentiviral ex-vivo platform has worked very well in bone marrow-derived disorders. We know that AAV works well in heart, but also other platform -- other disease, organs liver and brain. And so we're not -- and we all know, there will be other gene therapy modalities in the future that are perhaps not quite enabled technologies yet but will be soon. And so right now, from the platform perspective, we're going after enabled technologies. Right now, the most enabled technologies are -- in our opinion, is ex-vivo lenti as well as some of the AAV capsid technologies, AAV9, certainly, but others like AAV9. They've been proven. We're standing on the shoulders of giants. We've learned a lot from the partners and companies who have gone to such as bluebird and AveXis and others. And we're trying to refine these technologies and then bring them to these unmet need areas. On the disease, we're also not focused on a particular therapeutic area or TA, as they would call it at Novartis. We're not focused on heart or bone marrow. Right now, we only have bone marrow and heart programs. In the future, that may change. Ultimately, we do want to create expertise though in certain domains. So lenti, AAV, heart, bone marrow is our expertise right now, and we'll be very thoughtful about how to expand that expertise into other disease areas because part of the advantage of being a small company is that you can try to hire some of the best people in bigger therapeutic area and platform, put them together, put them around the table to think about these things. The caveat is if we get too broad, we have 10 therapeutic areas and 10 platforms, that's where we actually would lose our expertise advantage. So we don't want to go that far either. So there's a balance in between.
Great. The other aspect is that you are -- the other part where gene therapy companies often get criticized is also like going after very small diseases as well, right? I mean, they're going after 100, 200, 300 patients. And that may not be economically great for the long term. You kind of have found some kind of middle ground here. You're going after rare diseases, but not too rare. So is that also come in the equation when you really think about the diseases to go after?
Yes. So we don't have a particular threshold of prevalence or incidence or addressable market. Certainly, to make an appropriate long-term business space, being able to treat several hundreds or thousands of patients is going to be compelling. As we build our pipeline, that certainly is the aim to aim higher in terms of prevalence numbers and addressable markets. However, some of the diseases that we've already targeted, such as Leukocyte Adhesion Deficiency-I, don't have that many patients around the world. But they have something else, which is an ability to really have proof of concept, maybe get our first approval with LAD-I, perhaps get a PRV in time assuming that we launch when the PRV is active. And it also allows us to build internal expertise, so we can tackle the bigger programs more easily. LAD-I, for example, also is the ultimate program where a little protein can really go a long way. You only need 2% to 4% CD18 expression in order to potentially confer long-term survival for these patients. So it really -- it's not as big as the others, but it made sense. Most of the ones we'll tackle from now on will be on the Danon-like size or thereabouts as we build the pipeline.
Got it. One more big picture theme, which is kind of emerging is that with the gene therapy, we have seen a little bit of regulatory action in last few months or so. Bluebird had issues, BioMarin got rejected as well or delayed basically. We saw something with Sarepta yesterday. So just trying to understand, I mean, what is your thought process around the regulatory environment right now? And do you think it is more case-by-case basis? Or it is more like FDA is little bit conservative when it comes to gene therapy as probably they should be, but I mean, how do you think about that?
Yes. We've been -- and some of these things just come up as surprises, and there's not much any company can do about things. And I think it's very important to have designations such as RMAT, which used to be called breakthrough or prime and others so we can have frequent agency dialogues and try to get ahead of these issues as early as possible. We're going to learn from what happened there. We have in the company folks who have encountered last-minute difficulties just prior to launch, went to CMC, on the production side, manufacturing side. I, myself at Novartis encountered challenges just in converting the -- what I thought, what everyone thought in leukemia was a world-class academic trial out of Penn into an industry-grade trial that ultimately led to the KYMRIAH approval. There is in the company have learned similarly from whether it's a BMS-Celgene or bluebird elsewhere. So we're trying to learn from this expertise and get ahead of it. Now having said that, we've been obsessive from day 1. The thing, at least I went out and did when I took this job in 2015 was to go to MolMed, secure a lot of lentiviral supply. They've done this before. They know what they're doing. They've gotten drugs approved. And it's just then to think 4 to 5 years into the future. Whenever you're starting the program, you have to think about, as we said earlier, the end goal in mind, what is the product going to look like? How are you going to get a consistent, robust, repeatable product out to the numbers of patients that you need and then hire the team that not only has had successes, but some failures. Sometimes we learn best from our failures. So when we hire, we try to -- where didn't something work out, what can we learn from it and apply to this company. That's sort of a broad answer to what you're saying.
No. That completely makes sense. So you have had -- I mean, even before Rocket, you have had experience with cell therapies and gene therapy with KYMRIAH. So the gene therapy world has moved a lot, especially from the manufacturing side in last few years. And it does seem like that even for third parties, the capacity is coming up with -- now big players are in the game now with Thermo and [indiscernible] and all those M&As. So ad -- so the question is you are -- for one part of business, you are developing your own manufacturing. And then for lenti, you are probably relying upon the third parties here. So there are a couple of questions. Number one, how do you balance these 2 external versus internal integration of the manufacturing? And number two, where do you -- how much do you think the external capacity has evolved in the last few years, which used to be very minimal in the beginning?
Yes. Excellent, excellent question, insightful question, Mohit. The lentiviral, the vector part of the lentiviral therapy would consider a more mature production global capacity, sort of global capacity for LVV is a little bit more mature than AAV. A lot of CDMOs as well as companies have figured out how to scale up suspension and acts some through other methods to be able to treat hundreds or thousands of patients. There is, in our estimation a reasonable amount of CDMO capacity for the long-term to support the agents that we've gone after in the lenti space. So we just haven't had the need to scale that up further. And for AAV, I think one question that is going to be interesting and important to every company who's looking to treat a large AAV-based population is how to scale up whether it's by going to a larger 2,000-liter bioreactor, or it's by changing cell lines or others. People are going to have to figure this out. This is an industry-wide question. We're very fortunate to be friends and partners with many similar companies learning and figuring this out together, really as a global team. Now I think it's an advantage to have an eye on the first few years of manufacturing in-house, while the industry figures this out, while CDMOs develop the capacity. That's why as a commitment to patients and making sure that we can get a reproducible product out to potentially hundreds, if not thousands of patients with Danon, we're committed now to building that in-house so that we're not dependent only on CDMO capacity. We will continue to work with CDMOs as either through dual sourcing or for backup reasons. In AAV, I think it's a good idea to develop that in-house, not just for capacity but also for process development. So that you're not always outsourcing process development and then trying to bring it back in. You can do everything in house. The PD feeds directly into the manufacturing. So we've thought about this. I think the cost of goods for lenti, vector as well as cell processing have gone down over the years. Certainly, from the days I was at Novartis, it's gone down substantially as global capacity has increased, and we've taken advantage of that.
Got it. And then how much of this -- the manufacturing changes play in the equation of trying to have manufacturing in-house and integrated from the get go because we have seen examples of Spark where they changed the process after the trial. Well, AveXis actually went all the way back, and then they just changed it before the trial. So how important it is?
Yes. I mean, there's various ways to think about this, and there's no right answer. We prefer to take our time to get the process than a commercial process even before we treat the first patient on a Phase II trial. We've been able to achieve that for actually all the indications right now, except Fanconi, and I'll come back to Fanconi. For LAD-I, for PKD, for osteopetrosis and for Danon disease, the vector and/or cell processing that will go into the patient is as close to commercial-ready as can be. We don't anticipate a lot of changes from here to be able to launch the product. So we don't anticipate a lot of comparability and things like that from here to commercial launch. That just means it takes longer to start up the trial, and that goes back to trying to be only in class so that you can take your time, and you're not punished for that, right? Now these trials for example, in LAD, we'll treat 9 patients. And those 9 patients will be treated by a commercial-ready product. And we don't have to do another trial after that potentially, right? The idea is that we avoid having to do another trial after that because that trial will be the final trial. Now in Fanconi, we inherited a program that was already in the clinic under Process A. We developed our own Process b, which is commercial ready. And therefore, we had to start the program over, we'll be able to harness some of the long-term information from Process A to support the registration. But that's the only exception to the rule here. And again, I think the leadership team here and the whole company, we've just been burdened in the past by comparability studies, and you just end up in a no person's land there. So we've just -- we've taken some time to do it the right way here. And hopefully, we don't anticipate the same hurdles when we go to launch here.
How much does FDA or regulators understand that many a times when you -- when corporates in-license these products, they come from academia, where the process are completely different and not so much cost-effective, and you have to change those processes just to make it commercially viable. So how much do you think regulators understand that part?
Quite a bit, quite a bit. If you look at Juan Bueren and Julian Sevilla's data from CMAT for the Phase I Fanconi program, there's no doubt that many of the children that they've treated over the last several years may be on their way to potential cures in the bone marrow and never needing a bone marrow transplant. That was all with an academic process. We call it Process A and when we went to both EMA and FDA with that information, especially with the blood count stabilizing and even improving in some cases, they got it right away. And they've been very supportive in helping us figure out not how to change it altogether, but how to make it more reproducible, more reliable, a little more robust so that instead of -- so that a greater proportion of patients can benefit. I think the agencies understand this now. I think there's very little difference now between academics and industry. Maybe 15 years ago, when I was still in my fellowship, it was a different story. And there was industry, and there was academics, but I think it's much more integrated now philosophically as well as in the agency's eyes. We often bring our academic partners to the agency dialogues, and it really becomes one team there. So I think there's been a lot of progress in that front.
Got it, completely understand. So maybe switching gears on the specific topics now. This was a great overview. So now let's just start with Fanconi. I mean you've got data as well as a process from academia, and then you are working with the Process B here. So maybe could you just walk us through the changes you have made? And then what should we expect in coming days, or coming quarters from this?
Yes. So as -- just as a reminder, ex-vivo lenti has both a viral production, lentiviral production process as well as a self-processing step. We made changes to both. On the viral front, we're now working with a commercial-grade vector. And why is that important? It's because the vector itself is more purified and more potent. So therefore, we're actually able to raise vector copy numbers more easily. Sometimes if you have an impure vector, you just -- no matter how many transduction enhancers you add, you just can't get the VCNs above a certain number, especially in a tough disease like Fanconi. So we've made the deliberate, sort of, strategic decision many years ago to take our time to develop the commercial-grade vector. We now have that, sourced from MolMed and other places. So pure vector, number one. Number two is, we added transduction enhancers to the culture and to the transduction process overall. Through that, we've been able to increase our vector copy numbers on average by 2 to 3x, and that's been very helpful and should be helpful for Fanconi patients. And then thirdly, we're also sticking to a fresh product. And the importance of a fresh product is that in Fanconi Anemia, when you cryopreserve and then thaw, there's often a lot of cell loss. So to preserve every cell possible going into the patient's bloodstream and bone marrow, we've decided to go with a fresh product. And finally, we've made some tweaks to the cell, the CD34 selection, the enrichment process that should make Fanconi easier to engraft over time. And this Process B, by the way, is also applied to pyruvate kinase deficiency, which is coming up LAD-I and also osteopetrosis and any other lenti programs we have in the future. In terms of data for Fanconi, much of the Process A, the Phase I from CMAT, has those patients, many of them have already gone out 3 years, some of them have stabilized or increased or improved their blood counts. We'll continue to provide probably annual updates to the CMAT Phase I. The Phase II, which is the Rocket-sponsored trial will have additional data at the end of the year from the Phase I/II that's being pursued at both Stanford and at CMAT in Spain and now also at University College, which we announced recently.
Got it.
And I should say that we also announced that University of Minnesota will be coming up. So 2 sites in the U.S.; 2 sites, new, and we'll be announcing preliminary data from that at the end of this year.
And then you are not using conditioning regimen for this program. So that's quite different from other lentivirus approaches we take. And why is that because the protein needed is limited here?
Yes. Fanconi Anemia is interesting. It is a one-off. You have to -- somehow, you have to condition the bone marrow. Somehow in all diseases, one has [indiscernible] the native disease stem cells to make room for new stem cells, the gene-corrected stem cells. Either one can do that through conditioning, busulfan, et cetera. That's what we're going to do in LAD-I and PKD osteopetrosis. And that's also what needs to be done in beta-thalassemia sickle cell and other diseases. Fanconi is an exception because the marrow conditions itself, if given enough time. Fanconi Anemia is a disease of the marrow conditioning itself, right? The whole outcome of Fanconi patients is that the stem cells fizzle out over the first few years of life. So we don't need to give any more conditioning to these marrows that are already conditioning themselves. It's just a question of giving the gene therapy and giving it enough time in to -- for these cells to engraft and to affect clinical outcomes. And that's why the earlier we go in the patient's life, the more chances we have of succeeding well ahead of the need for a bone marrow transplant. So it's a little bit of a different disease compared with all the other bone marrow-derived disorders. The only other close one is probably XSCID, but I'm not sure that, not conditioning at all is a viable approach there either.
Got it. Got it. So 1 question, I mean, before I go back to the specific questions on other diseases. The conditioning regimen is kind of noted as Achilles' heel for most -- many of these lentivirus therapies because doctors get a little bit worried about frying of the bone marrow. And so -- and then there are companies -- I mean, I know Vertex is coming up with thinking about planning -- coming up with some better condition regimens. Where do you think the field is in terms of understanding the need of conditioning lower -- softer conditioning regimen as well as developing something which could be a little bit milder.
Yes. So it depends on the disease side. I think the field has learned recently that for most disease types, you're going to want to do fully myeloablative conditioning. It might be moderated to some extent as we've done in LAD-I, but not so much that you can't get appropriate engraftment.
Okay.
And right, so that's the key. I think for certain diseases, fully myeloablative conditioning is required. I think on the safety front, however, a very interesting development in recent years is the use and the emergence of nongenotoxic conditioning. We have a research program going on with Forty Seven, Gilead in this regard now. And Magenta just came out with some data in another indication recently. And I think ultimately, the nongenotoxic conditioning approach can be applied across gene therapies. I think, in fact, our first start is Fanconi Anemia, but it may even be more applicable to something like pyruvate kinase deficiency, where if we can use nongenotoxic conditioning to suppress the bone marrow, allow for the gene cells to engraft without the long-term side effects of chemotherapy, that could make gene therapy much more palatable than it is currently across the landscape in diseases such as PKD. So we look forward to developing that as part of the life cycle. The current programs, however, all the clinical programs for LAD-I, PKD and osteopetrosis, will employ chemotherapy, fully myeloablative conditioning. Only Fanconi is the one where we don't need anything at all.
Got it. So maybe turning gears towards PKD here. So I mean, Agios has mitapivat, which is reaching probably soon, we'll have Phase III data there. So how do you think about your program versus a small molecule approach? And I mean, again, gene therapy versus the chronic therapy aspect of it, how do you think about the competitive scenario here?
Yes. So Agios is a very valuable thought partner to us. We share some investigators, and there is a common pool of natural history that we will draw from. So let me just say that upfront. I think, ultimately, it's going to become patients' and physicians' choice as to whether a gene therapy approach works or a small molecule or allosteric activator -- enzyme activator approach works. With genes, what I can say is that there are certain patients who have no protein or very little protein where activating the protein may not confer clinical benefit and where the only approach is a gene therapy approach, right? For some patients that are more moderate, there will ultimately be a choice. And as we develop a nongenotoxic conditioning pathway in the future, it'll just open up those options for some of those patients who are much more mild and much more moderate in the future as well. But I would say that thinking about the PKD market overall. Our current trial is focused on patients who are transfusion-dependent post-splenectomy, but the more and more we look at the effects of splenectomy and how much it really benefits patients or not, we think ultimately, the -- during the life cycle of the PKD program, we'll be able to address severe patients not just those post-splenectomy, but pre-splenectomy, who would need a splenectomy and other severe patients. So at least 50% of the population long term, we think would be a [indiscernible] gene therapy, if not more.
Got it. I know, Danon, I mean, I know people would want me to talk a lot about that. And so maybe just give us some color -- some -- your thought process on this program. I mean, I don't know a lot about Danon. So for me and then benefit of investors, what exactly is the program? And where are you in terms of development of this program?
Sure. Yes, Danon is very, very close to me and to many of us. So first of all, Danon is our own AAV program. At the moment. And it is also the largest market in terms of prevalence and addressable market. It's 15,000 to 30,000 EU plus U.S. and so an exciting program from that viewpoint, from a long-term company growth viewpoint. Now Danon disease is a relatively recently discovered disease, affecting heart, muscle and brain, but primarily the heart. The cause of mortality in these patients is the heart. It's an X-linked disorder, but it's X-linked dominant, so it affects both boys and girls. The boys have higher penetrance and more severe disease early on. Most boys with the disease pass away in their 20s without a heart transplant. The females can live into their 40s or 50s, but they also -- the cause of their mortality also tends to be cardiomyopathy for both boys and girls. It's a disease of autophagy. Autophagy is like the recycling center of the cell. When debris builds up, whether it's mitochondria, organelles or apparatus or all those things we learned in ninth grade, when those organelles need to be cleaned out of the cell, autophagy is the process -- one of the processes for doing that. LAMP2 is a protein that is critical for autophagy. And without LAMP2, this debris builds up in the cell and especially affects cardiomyocytes, which need to have this synergized contraction in order to be -- to have an effective ejection fraction and also the ability to -- for the muscle to relax, right? Both things are important. So these patients tend to have gradual onset of cardiomyopathy. By the time they're teenagers they have rapid decline. And as I said, boys who have X-linked and have no LAMP2 protein will not make it into the 20s without a heart transplant, and it's almost invariably the case. The penetrance is really high. It's a true loss of function type of disease, and that's why it was a compelling indication for us. We think it could also represent the first potential curative gene therapy for a monogenic heart condition of any kind so it represents a big step forward for the cardiology field as well. In terms of the clinical trial, we announced recently that we treated our first higher dose patient at 1.1e14 vector genomes per kilogram. We've completed a treatment of the low dose cohort, which was 6.7e13 vector genomes per kilogram. So we stepped it up for this dose. And these are all doses where there was demonstrable efficacy in preclinical models, and we're exploring this in humans now.
Got it. One question we get a lot around whenever we try -- we get to the e^14 doses for these neuromuscular diseases, I mean, in this case, your -- this is a cardio disease, these doses could be a little bit higher in terms of safety point of view. So how are you ensuring that you don't run into those issues? And is there a cap there beyond which it would not be safe?
Yes. That's an excellent question. And I know it's on a lot of people's minds. There's a lot of publications on this coming out that we're learning from as we go. 1.1e14, first of all, is not the highest dose tested. It's the dose where the AveXis program was ultimately approved for SMA Type 1. Cardiomyocytes have more affinity for -- and I should rather say, AAV9 has more affinity for cardiomyocyte, AAV9 loves the heart. So our hope, as we go through these trials, is that we would never have to go up to the 3e14 range where there were more toxicities with other neuromuscular programs. But that being said, look, you treat dozens of patients, there's different toxicities. It's part of drug development. It's very commonplace in oncology to have even more severe toxicities. You look back at CAR-T, which is a program I was fortunate to be involved in, CRS, cytokine release syndrome, was such a big thing, and there were several patients who got sick. There were some unfortunate patients who passed away in the early days. But the industry got around it. They figured it out. I think the AAV space is exactly there. Over time, a lot of people think about platelets drops and complement activation and immunogenicity and think of these as roadblocks, but I don't see them as roadblocks. I see them as challenges to work around in order to get these drugs onto the marketplace ultimately. And I think not just for Danon disease, but the other neuromuscular programs will also get there. We're going to get through these.
Got it. That makes sense. Maybe, I mean, I think one disease, we have not talked about is osteopetrosis. Could you talk a little bit more about that too as well?
Yes. Osteopetrosis is special to me personally, when I first visited Johan Richter in Lund, Sweden, I think it was 2015 or '16. This redefines the word devastating. These are children who might be 1 or 2 years old and sometimes before 1 years old, and you can see the picture, and it's very sad, have bone growth because of the inability to resorb bone because of mutation in TCIRG1. And because of the inability to resorb bone, a lot of these patients are deaf and blind early in life, sometimes by the first year. A lot of them go into bone marrow failure in the single-digit years. So to me, that redefines the word suffering. These are patients and families who are truly truly suffering. So we brought this program in as one of our first programs in 2015 and '16. This was our fifth IND to clear, I'm very happy to say, in the span of maybe 2 years or so. And we are now IND cleared, identified a site here in the U.S., UCLA, which is [indiscernible] site, who will be treating the first patient by the end of the year.
Got it. Very helpful. I think we have 5, 7 minutes left. So I know you have tons of catalysts coming up by the end of the year. So I'm sure investors would hate me if I don't talk about them. So maybe if you can walk us through the time lines of those catalysts? And what should we expect from those catalysts, just to set the expectations here?
Yes, sure. So Fanconi will have preliminary readout from Process B at the end of the year. That's on track. We've been able to continue enrolling patients despite COVID. These are severe diseases. And obviously, we're very cognizant and aware of the need for focus on COVID research at the moment, but we've been able to enroll several patients in an ongoing basis. So the end of the year, we'll be able to show preliminary Process B data. Whatever we have by then, we'll be able to show from the drug product metrics of recent patients. For LAD-I, we'll have a little bit more of a mature update. Last year, we had an update only on one patient at ASGCT. We treated the second patient, and that finished our Phase I. We're starting the Phase II. So we'll have some additional updates at the end of the year in LAD. PKD is, to me, something that's going to be very exciting. And it's a much more straightforward approach than Fanconi Anemia. In some ways, we were lucky to start out with Fanconi Anemia because it's allowed us to iron out the therapy in the most difficult disease of all. PKD is much more like a beta-thalassemia or other red cell disorder. We know about the percent protein expression we need. We know that if you get enough cells and a high enough VCN from other companies and programs that we should be able to get there. It's relatively straightforward. And we'll have some preliminary data readout in PKD by year-end as the trial enrolls and continues. And then Danon disease, I think, is what people have been asking about, perhaps most voraciously, and we will have a data -- a preliminary Phase I data update by the end of the year.
Got it. Very helpful. Maybe one last question before. So yes, fast forward 5 years, 2025 Citi Biotech Pharma Conference, 20th Citi Biopharma Conference, I hope you come. And we are sitting again next to fireside, probably real fireside this time. What would make you feel really happy and satisfied about the company? And you would say, these were good 5 years.
You mean...
Looking back at 5 years, 5 years.
For now or in a few years. Yes. I mean I never thought that we'd get this far. I never thought that we'd get 5 INDs cleared and 5 clinical programs, and 2 of them being in registration phase and a manufacturing facility being built and R&D capabilities to help expand the pipeline in the future. I'm pleasantly surprised. Some days, I'm shocked, and I'm trying to figure out what happened. But this is great. And I also feel that much of our team -- much of the difficult work, I believe, and obviously, there's tons of difficulties ahead in terms of how do we get commercialization. But on the drug development side, CMC was tough. That was something that I have some experience in, but I'm not a CMC person. Now we're in the clinic, and I feel the company is just getting started. That's how I feel about this, right? So we have 5 programs. In the future, I think that we'll have a pipeline that could rival the pipeline of bigger companies. And I also think that the way we're thinking about asset selection here is not just throwing darts in a wall. We're trying to select assets where all, if not almost all of the programs should work, right? Scientifically, they should be derisked. And we just thought about this pretty carefully. So if we have several programs in the future, we're only going to select the types that could work in the same way that some of the programs that in the early company did. So how am I feeling -- I'm feeling more optimistic than ever. I think Rocket is entering stage 2 of launch. And yes, sky is the limit, if I just be cheesy here.
Where do you think Rocket will be in 5 years? What will be the stage 3 or 4?
Yes. So I hope we have 5 programs -- all 5 of these programs approved by then and on the market. I hope we have another -- a similar pipeline like this in development. And by then, I hope we have internal discovery capabilities that have another discovery pipeline in preclinical development. So I was at Novartis, and I always was highly admiring of the leaders in oncology there who could manage these 10 to 15 to 20 programs in the oncology space. And I think gene therapy companies are not far behind, companies who will have sizable pipelines like that. And we hope to be amidst -- amongst those in 5 years.
Great. On that high note, thank you very much, Gaurav. Really appreciate this candid conversation. I enjoyed a lot. I hope you did too.
Thank you. As always, Mohit.
Great, and thank you, everyone, for joining us today. Hope you all have a great time.
Thank you. Bye.
Bye.
Read the full transcript via the API
You're viewing the first half of this call. Get the complete Rocket Pharmaceuticals, Inc. transcript - plus 251,000+ transcripts from 12,000+ companies, speaker segments and full-text search - through the EarningsAPI REST API or hosted MCP server.
Get an API key View API docs →For developers and AI pipelines
Programmatic access to Rocket Pharmaceuticals, Inc. earnings transcripts and 251,000+ others is available through the
EarningsAPI REST API and the hosted MCP server.
Quarterly plans from $105 - full transcripts, speaker segments, full-text search,
and the /api/v1/transcripts/recent polling endpoint for ETL pipelines.