Bringing Curative Cell Therapies To Market with Kite Pharma's Cindy Perettie

On this week's episode of the Business of Biotech, we're speaking with Cindy Perettie, Executive Vice President and Global Head at Kite Pharma, a Gilead-owned company focused on curing cancer with cell therapies. Perettie talks about scaling cell therapies in the community setting, global manufacturing strategy, preparing for the launch of late-stage development programs in lymphoma and multiple myeloma, and working with partners like Arcellx. She also discusses cell therapy funding cycles and big pharma, the autologous versus allogeneic divide, why in vivo CAR-T therapies could be a game changer, what the FDA's focus on cell and gene therapies could mean for U.S. competition, and more. 

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Transcript

Cindy’s Path From Lab To Industry

Ben Comer 0:04

Welcome back to the Business of Biotech. I'm your host, Ben Comer, Chief Editor at Life Science Leader, and today I'm speaking with Cindy Perettie, Executive Vice President and Global Head at Kite Pharma, a Gilead company focused on developing and commercializing curative CAR T cell therapies for cancer patients. Cindy is an experienced leader with a background including cancer research and life science industry leadership positions at Genentech, Roche, and Foundation Medicine. I'm excited to speak with Cindy about developing, scaling, and commercializing curative cell therapies at Kite, her approach to partnerships and collaborations, what she's learned from previous industry experiences, and what the future holds for cell therapy funding and product development. Thank you so much for being here, Cindy. Thank you, Ben. I'm really excited to be here. Yeah, I'm really glad to speak with you as well. I think Kite's a really fascinating uh uh organization and company. Um but I want to start out with uh a little bit on your background, Cindy. You began your career, I think, as a research research associate at Johns Hopkins. Uh and then, as I mentioned in the intro, I went on to work for some of the biggest names in biotech, Genentech and then Roach, where you worked as head of global oncology strategy, and then later as head of Roach Molecular Lab Solutions. Uh, you were also CEO at Foundation Medicine. What can you tell me, Cindy, about your initial decision? Uh, I guess to go from the research field uh into the life sciences industry.

Cindy Perettie 1:42

You know, it's interesting. When you're when you're working in basic research at an academic institution, it's really exciting. You're making discoveries, but what you realize is if you want to turn those discoveries into something that's going to scale for patients, then it makes a lot of sense to join industry because industry has the ability to do all of those things, to take sort of amazing basic research and make it scale. Um, and it, you know, for me, it was about how you can have a broader impact. So it was, it was a it was a good decision overall.

Ben Comer 2:12

What drew you to uh to oncology in particular?

Cindy Perettie 2:16

You know, early um early in life I lost a my grandfather to cancer. And um, when I started doing research, there was a lot of interesting, it was at a time where um new genes were being found as it related to cancer. And so your ability to do targeted treatments versus sort of general chemotherapy was just coming to the forefront. And um, so it was an exciting time, and I think that's what drew me both on the professional side.

Why Oncology And Early Targeted Therapy

Ben Comer 2:43

Yeah, that that makes a lot of sense. I I'm also curious if that initial research experience that you had, and then also your time. I didn't mention this in the intro, but also the time you spent at Sarah Cannon Research Institute, do you think that helped you uh as a biotech leader? And if so, how so?

Cindy Perettie 3:03

Yeah, it's interesting. So we had collaborated with Sarah Canon for many years. I was overseeing Avastin, which was one of an anti-Bel Jeff product in the early days of targeted therapies. And Sarah Canon was such a profound institution where we did a lot of our clinical trials, and they were actually the first group to move clinical trials into the community. So prior to that, you were doing them at academic centers. And um it gave you a chance to really get more real life treatment experience. And so in conversations with them, they convinced me to make the leap from Genentech and join Sarah Canon, which was probably one of the best things I had done in my career. And I would say, because when I was in industry, um you had this view that you were really designing trials that were patient-friendly. And so I think we all drank that Kool-Aid and told ourselves every trial design we put forward, we thought about the patient deeply and we were thinking about the care that the physician needed to give. And then when I went on the other side of it on the Sarah Canasi, I'll give you an example. The my first couple of weeks, I was in the phase one clinic. And in phase one studies, you asked patients to do 12-hour PK days, sometimes 24-hour PK days, where you're actually doing blood draws throughout the day. Um, and the patient's sitting in the cancer center in an office essentially for that entire time, often with a family member. And so you got to see, you really got a bird's eye view of what it took for the patient, what it took for the physician to administer that clinical trial. Um, and so, you know, years later when I left and went back to industry, I had a really different perspective on clinical trial design and how you could do it so that it was meaningful for the company, for the regulatory agencies, but also um that you weren't putting a huge burden on the patients, their caregivers, the physicians.

Ben Comer 4:54

Yeah, you know, people don't just get cancer within what, 50 or 60 miles of an academic medical center. The community setting, you know, is is so important. And I wonder if that's something, you know, where we're talking about your background, we'll get to kite, but is that something that you've thought about, you know, when not only, you know, when it comes to clinical trial protocols and and clinical clinical trial design, but when you think about actual commercialization, uh, is it is it something that you yeah.

Lessons From Community-Based Trials

Cindy Perettie 5:22

Absolutely. And let's start with clinical trials on that. I'm a big fan of the community, and I realize that you can get exceptional care in the community. And I think prior to joining Sarah Canon, I often thought that you had to go to a top-notch academic center to get that sort of care. And I can only say wonderful things about the doctors that were associated with Sarah Canon at that time from Tennessee Oncology, Florida Cancer Specialist, just really invested in their patients and offering clinical trials that you would, you know, in the past have to go into an academic center to get access to. And it opened my eyes to how much um opportunity there is for patients to be seen closer to home and still get that great care. And so as I move forward in my career, that's the other thing we think about a lot. And, you know, we've got new products coming to the market. An example might be Anita Cell, where we've really thought about how do we make sure 20% of those patients in our clinical trial are actually enrolled in the community, in an outpatient setting, able to go home. And um and it makes a huge difference.

Ben Comer 6:22

Yeah, it's something that that can be overlooked. Uh, and and I think it's critically important. I agree with you on that point. Um are there any specific, uh just thinking about your your background prior to kite, Sandy, are there any specific accomplishments or or or maybe the biggest challenges that that you would mention um prior to to joining kite that kind of uh I guess demonstrate some of the things you've worked on, you know, uh prior to joining?

Defining Team Wins And Hemlibra Breakthroughs

Cindy Perettie 6:50

Yeah, you know, I I tend to think about this. If you think about major accomplishments, no single person can do anything. It always takes a great team. And I feel really lucky that I've had the privilege to work with many great teams, whether it was at Genentech or Roche or Sarah Cannon or here at Kite Pharma. I think the idea of bringing together a group of talented individuals and then together tackling your biggest challenges and coming up with your biggest accomplishments is something that gives me a lot of pride. So if I talk about the things that, you know, an accomplishment I'm most proud of, please know there's an amazing team behind that. Absolutely. I'm gonna use an example that was both an accomplishment but had big challenges. And it's something that's allowed me when I went into foundation medicine or kite pharma to think about as an example and to think about a market differently, to think about patients differently. When I was at Genentech and Roche, we developed a therapy called Heme Libra. And I'm lucky enough to work with our SVP of development here at Kite, was actually the clinician who led that development. But Heme Libra was a product that was so different than anything you'd ever seen on the market. So for hemophilia patients, they're often young boys that obviously that grow into men, but you start as a child, and many of these patients have to take um factor eight on a daily basis, or now they've got long-acting factor where maybe every three days they would take an infusion of factor. And if you grow um resistant to factor, you go on bypassing agents. But these are things that you're administering daily, or you know, if you're lucky, maybe every two to three days. And we developed a therapy with our colleagues at ShuGuy that would allow a patient to only take an infusion once every 28 days. And this is a big deal. It's a big deal for small kids, it's a big deal for somebody going off to college. You don't want to be infusing yourself with your new roommate in the room every day. And so it was a really a game-changing therapy. But if you grew up taking these agents and somebody says, trust us, don't take this therapy. You don't need or take our therapy and you don't need to take anything else for 28 days. That's really scary because if you bump into something, you could potentially bleed to death. And so looking at a the development of a novel therapy that had no endpoints for something that would be a 28-day, you know, that would work for 28 days, and then asking patients and physicians to trust us. Um, there was a lot that went into that, but we had such an amazing team. And now I look at that product and what it's done for patients. And we had patients in the clinical trial that for the first time rode a roller coaster. Um, and so there was so much profound impact for patients with hemophilia, but there was a lot of learnings along the way. And those learnings of not looking at everything as the same, if that makes sense. What I learned in the past in traditional drug development or traditional commercialization is going to work in the future has been really helpful as I went on to roles in uh foundation medicine or even here at Kite.

Ben Comer 9:46

Well, speaking of uh of working with teams, you're also a board member at uh at Frenome, as well as a board member at Aspen Neurosciences. And uh I wanted to just quickly talk about this. Maybe you could tell me, Cindy, you know, what your kind of objectives are uh as a board member and if there's a a specific skill or talent that people are kind of go to you for uh as a board member, um, you know, when there's something comes up.

Cindy Perettie 10:15

Yeah, no, it's a great question. In fact, I'm no longer on the board at Freenome, but I am on Aspen Neurosciences. But similarly, I would say those boards um were great boards. They were diverse and everybody was bringing something different to the table. The Aspen uh board is no different, right? They've got a really strong board members, everything from investors to people who bring scientific expertise or very specific, specific things. And I think the the piece that um I've enjoyed bringing to the board as well as learning from my peers is this idea of how do you take a product, particularly with aspen, it's um it's a cell therapy product. And how do you scale it? And um, how do you scale it through development? How do you scale it both from a manufacturing and commercial standpoint? Um, and so those are things that I brought to both on the phenome side because of my uh foundation medicine experience and now on the Aspen Neurosciences side, which is how do you think about scaling pivotal trials? How do you think about gearing up uh for a launch? And how do you think about scaling globally?

Ben Comer 11:18

Well, scaling cell therapy manufacturing, I would assume that you are um are probably highly sought out, you know, for anyone who is an expert and and how to do that well, uh it would be in demand, I would think. Uh so that that makes sense. But uh I also want to ask about one other team of sorts that that you're a member of, which is the biotech CEO Sisterhood. Um I saw the photo. I was at JP Morgan in San Francisco last week. I couldn't make it out, unfortunately, to the biotech CEO Pink Al, the sisterhood meeting in in uh Union Square. Uh, but I I mean I saw the photo and it looks like the organization's really expanding. I wonder if you could just take a minute, Cindy, uh, for the the business of biotech listeners to kind of explain what that organization is and what its mission is.

Cindy Perettie 12:09

Yeah, no, I'm happy to. So I would say I got to work with a number of amazing women through my career. And in in early in my career, there weren't a lot of females in science. And so getting to connect with each other is really important. It became important when I was a scientist. It certainly became important when I went into industry. And sometimes you'd almost seek each other out so you could see um maybe somebody who looked like you and uh sort of had similar experiences. And I think the piece that the biotech CEO sisterhood did is formalize that. And so um two of the members, uh Sheila Goose Raji is one of the founding members, and I worked together at Genentech. In fact, when I was running Avastin, she replaced me when I moved to Europe. She took over Avastin. But these are women who um have a variety of different backgrounds and experiences and coming together and finding ways that we can learn from each other, um, share learning, share mistakes, share challenges, share opportunities, and support each other in our development is something that is now, by doing this, has now become even more formalized. And it was the type of thing that maybe you got together in the past over a glass of wine or you'd meet for lunch and do different things. And now we have a chance to really have concerted learnings and share, share experiences with each other. Everything from development to fundraising to running companies to how to be a great mom and a leader. Um and those aren't things you necessarily talk about day to day at work. So I think having having this experience has been fantastic.

Ben Comer 13:39

So it's formalized in the sense that you can kind of reach out. Or I mean, are there scheduled, you know, meetings or or you know, like calls regularly? And it's just do you, you know, do you have an agenda of specific things? It sorry, I'm I'm just curious about this organization, you know. Yeah, how does it work? Yeah.

Cindy Perettie 13:57

Yeah, it's great. So so yes, you can call each other informally, and many of us do on a regular basis. I would say we have a summit every year where we tackle important topics, and they could be things like if I think about last year, you know, how do you do fundraising um most effectively in a difficult market? You know, the markets haven't been exactly where we wanted them to be. And so having an opportunity to share with each other and support um with an agenda that we all put together, you know, we ask each other what's most important to you, what should we be talking about, and sort of codify the agenda in that way. There's also chances when we're at places like ASCO or JP Morgan or many events that we can all get together as a group and we tend to um find opportunities like you saw at JP Morgan to come together. And then the last thing I'd say is I um run the Women at Gilead um ERG group here. And I had a chance to invite Sheila in. Um, my the Women at Gilead invited Sheila to come speak to us and two other women CEOs to talk to all of the women between Gilead and Kite that wanted to come hear about it. In fact, what I what I'd say is we had 20% of the attendees were men, and just talking about um what it takes to be a CEO, what it takes to um rise up in leadership, what were their experiences? And so there's a variety of ways that we get together and support each other.

Boards, Scaling Skills, And Aspen Neurosciences

Ben Comer 15:16

Um thank you for that uh background on uh on the biotech sister at CEO. Uh I think, like I said, it's a really interesting organization, and uh I'm glad you're a part of it. Um you joined Kite in 2023. What were the circumstances behind that move and and what in particular made you want to join Kite?

Cindy Perettie 15:37

Yeah, so a couple of things. So one is when I think about what's your next career move, I'm always thinking about it from a personal, professional journey. What do you need that at that point in your life personally? And what, you know, what's going to help you grow professionally? And I would say joining Kite, I had worked with Dan O'Day, who's the CEO at Gilead for many, many years. In fact, I think I knew him before we actually worked together when we were we were both in Europe. Um, and he's a tremendous leader. So I think for me, it starts with working with or for somebody that I have a lot of respect for that I can learn from. And Dan was definitely a great draw for me coming to kite. I think the second thing that brought me to kite, and I have been watching kite uh very closely to the point that um when I was at Roche and Genentech, we also were interested in kite and Gilead um sort of closed the deal. Um, so kite is a company that I've looked at because of the curative potential of cell therapy. And there's few companies that you work for that are not afraid to cure patients. Genentech was certainly one of them, and Gilead's exactly like that. And so is kite, right? Gilead has had the chance to cure HCV. We are on a road to cure HIV as well and end the epidemic. But then you look at what cell therapy has done for patients with lymphoma, what we're seeing in the myeloma data today, being able to join a company that has so much promise, such great science. And, you know, after getting here, finding out what an amazing group of dedicated employees we have at Kite. Um, so so I think it's a combination of leadership as well as the mission and what we're doing for patients.

Ben Comer 17:10

I wanted to ask about how Kite fits into um the larger company Gilead. I wonder if you could just describe, you know, the kind of uh executive hierarchy and how Gilead supports kite, how the companies work together.

The Biotech CEO Sisterhood

Cindy Perettie 17:27

Sure. So um, you know, when Dan, when Dan came into Gilead, he recognized quickly that kite's different and cell therapy is truly different, all the way down to the manufacturing, if that makes sense. It's not sort of a plug and play model into a traditional pharma company. And so at that point in time, long before I was here, he made a decision to keep kite separate. Um, and then when I came in um and talking with Dan and lessons I learned at Foundation Medicine, because it was a similar model, and I was working with Rush, right? Yeah. Um, was that integrating GNA functions really help. It keeps connectivity between the two companies. Um, you have different reporting systems financially that can make it difficult if you're not working on the same systems. And so prior to my coming in, we agreed that um we would integrate the GNA functions. And I think that was something that was really welcomed by the Gilead team and by the Kite team, and it's allowed uh the communication channels to open up even more because we're physically in different locations. At the time we were, you know, LA versus San Francisco. We've since now um we occupy a portion of the Gilead campus with our with our research team now, too. But um, Dan is an incredibly empowering leader, and he allows for independent thinking and decision making. But I also know to be successful as a company and to grow in our ambitions at Gilead and Kite in oncology and inflammation and HIV, frankly, the more we work together, the better it's going to be for everybody and for patients. So I sit on the GLT, which is the Gilead leadership team or executive team, and my peers are the heads at Gilead. So we work very closely together. We spent a lot of time together. Um, and it's been great bringing the two companies closer together and allowed us to really think about our go forward portfolio and how we can find not just synergies, but how we can build off of some foundational assets that we have in our in our um portfolio, like Yescarta, like Anita Cell. And so um we operate independently, we are uh together from a GNA function, but we do a lot of things together, including strategy.

Ben Comer 19:33

Yeah. And I I would imagine that um that consolidating the GNA function, I mean, it it at the very least reduces a lot of administrative burden and kind of frees you up to do the things that you really want to focus on.

Cindy Perettie 19:46

Absolutely.

Ben Comer 19:47

Absolutely. Um Height is currently conducting three phase three trials. Um, two, and correct me if I'm wrong on any of this, two label expansions of the previously approved a B cell and follicular lymphoma treatment. Yes, CARTA. Uh, and then you've got a third phase three trial for uh relapsed refractory multiple myeloma patients. That's with a different, uh, a different cell therapy CAR T cell therapy. Uh, and that one I believe is in collaboration with uh our CELIX or Arc Celics, is that right? Yeah, okay. Um, can you talk a little bit, Cindy, about what a launch prep looks like, you know, for cell therapy, maybe you know, in the context of a more traditional small molecule or now monoclonal antibody launch?

Why Join Kite And How Gilead Fits

Cindy Perettie 20:36

Sure. No, I'm happy to. And we're as you know, we're gearing up for the anita cell launch, which is the collaboration um with our stelics of the product and multiple myeloma. And I think, you know, if I think about the differences and what I was saying, sometimes you have to get rid of all the things you know because it's not going to help you for the future and think about things differently. Cell therapies today are delivered in authorized treatment centers around the globe. So we have to go in, um, authorize the treatment center. There's accreditations that go along with it that are independent bodies. And so you can't just um push these medicines out today. I think there'll be a future where you can in a widespread way to every community practice. These are these are um these are systems that know how to deliver cell therapy. So for anita cell, we're getting we're gearing up right now. The nice part about anita cell versus when we launched Giscarta is today we have an established footprint. So around the globe, we have over 570 authorized treatment centers just in the US alone. We're approaching 180 treatment centers. And we grow that number in the US, I'll say, by about 20 to 25 new treatment centers a year. So part of it is making sure you've got the global footprint and you're able to deliver your therapy. Importantly, though, and you've we've all learned this in cell therapy, you have to also be able to manufacture the therapy. And that's something that um kite is just extraordinary at. I've learned so much while I'm here, and we have such Committed um colleagues in both the research uh process development and our in our network as well as our manufacturing network. And so we're gearing up right now to launch in the US with a goal to launch XUS as well. So we'll we have been uh we did a tech transfer and moved uh the anidocell product into our Maryland facility. So we went from using a CRO where it was taking 40 to 50 days to get the product to each patient on the clinical trial to really being in line with our current manufactured product, just Garden Dicarta. So we have a turnaround time today of between 15 and 17 days, depending on where you're shipping around the globe. Um, our product is 96% reliability. It means 96% of the patients that send their cells in, we send a product back on the first run. Sometimes it will take two, but it's a very small percentage. And if I look at the competitors in the market today, the turnaround times are 30 to 40 days. The reliability is around 80%. And these are patients that have cancer and need a treatment right away. So we are excited as we're gearing up for this launch together with our CELICs about what we're going to be able to provide for patients globally. And then if you look at the product itself, the efficacy is phenomenal. Um, where you're seeing, you know, 96% of patients having a response, you're seeing very high minimal residual disease, meaning they have no detectable disease, 95% of those patients too. And we have no neurotoxicities, which is something that some of the competitive products have, where you can actually get a great therapy, but in the process of that develop something like a Parkinson's syndrome or Gillian Beret. And so we're excited about the safety profile, we're excited about the efficacy and what we're going to do for patients with our global manufacturing.

Ben Comer 23:45

On the patient access piece, and you you just mentioned uh a minute ago a future where, you know, any patient would be able to access these therapies. Is that um I guess how do you get there? Is it building, you know, just taking the US for example? Is it simply expanding beyond 180 sites to cover, you know, the full geography, or is there a different kind of like step change for access that that you anticipate happening?

Cindy Perettie 24:10

You know, I'll call it a step change. And and from my previous life where I was doing T cell engagers, we had something similar because those products were being administered in a hospital setting for the first two days in a step-up dosing, and then patients were going home. So there's a lot of learnings from that. And what we're seeing today with with therapies like anita cell, where the safety profile is quite pristine, these are therapies that can be delivered in a community setting or an outpatient setting. So as I look at the next generation of cell therapies, I think we've all made improvements to enable that. So everything we're doing in our research portfolio has the lens of this needs to be an outpatient therapy that can be administered in the community. And so it's not just about opening up the community centers. Um, there's a lot that we have done with FACT, which is the accrediting body over the course of last year to get accreditation for community practices. And they released that at Ash this year. So there's a stepwise approach that we're taking, but we're seeing it start to unlock the system. So it's a combination of a therapy that safely can be delivered in an outpatient setting, coupled with removing some of the access hurdles I just talked about.

Ben Comer 25:21

Got it. Great. Um, I wanted to ask a follow-up as well on Arcelix uh and your collaboration with that company. And I'm just curious, you know, kind of how you share responsibilities for uh for Anita Cell, you know, kind of who does what and how that collaboration works in practice.

Cindy Perettie 25:40

Yeah, so we we have a really great collaboration with Arcellix. I can't say enough good things. It's a strong partnership. And the nice part about collaborating with small companies is that you each bring something different. And the pieces that our CELX brought to this is a stellar innovation that I just talked about that has a really heavily differentiated safety and efficacy profile. And then there's things that Kite brings to the table, which is how do you scale some of the things we talked about earlier, which is how do we bring this product into global development and run global phase three studies, work with regulators around the globe? How do we have a global footprint that we can commercialize on? And of course, our industry-leading manufacturing and all of that together is allowing us to take this great innovation from our CELEX and scale it to patients. Um, today, the way thing the today, the way the contract is structured, we co-promote in the US. So you'll see us come out to launch together in the second half of this year, and then the rest of the world we handle because kite is embedded throughout um throughout around the globe through the Gilead affiliates. And we have global development capabilities as well as global manufacturing capabilities.

Ben Comer 26:50

Excellent. And I'll just mention uh as a side note to business of biotech listeners, if you want to learn more about Arcellix, we did have the their CEO on the show. Uh it's been a couple of years now, but um check that out if you're interested. Um coming back to cell therapy manufacturing uh and scale, you've talked to Cindy, you've worked in you know, monoclonal antibodies, you worked at Foundation Medicine and genotyping, um, both of which at the time were were you know new-ish at the very least. And you had to work with scaling those, you know, new technologies as well. And I wonder how cell therapy manufacturing and scale today kind of compares maybe with uh the early days of of manufacturing monoclonal antibodies, maybe as an example.

Launch Prep For CAR T And Manufacturing Edge

Cindy Perettie 27:40

Yeah, no, it's a great, it's a great example that we use a lot of times, in particular um with our own employees or with investors. And uh I was at Genentech in Chiron in the early days where everyone was trying to figure out how to scale a monoclonal antibody. There weren't things like 20,000 liter reactors, and there weren't necessarily all of these things set up. We're in the same boat, and there's a lot of learnings we can take from um what folks did when they were scaling manufacturing of antibodies. And I think about um now how how companies do that is they have usually a global footprint, maybe a manufacturing facility in Europe, one in the US, maybe one other in Asia, and they're able to serve that whole community. This is no different. We've put in three manufacturing facilities, one here in Los Angeles that allows us to serve Asia as well as the western part of the US. We have one in Maryland that serves uh the eastern part of the US, and we have one in Europe uh that allows us, and and sorry, we're also serving Latin America, and the one in Europe allows us to scale nicely through Europe and the Middle East. And that's a lot of the learnings, honestly, from how people set up monoclonal antibody uh production facilities.

Ben Comer 28:45

What would you say is the most difficult piece of scaling up a cell therapy for, you know, late-stage clinical trials and then ultimately commercialization? Is there a kind of singular piece or a handful of issues that, you know, that the not just kite, but the cell therapy development sector uh in total is is dealing with or struggling with?

Cindy Perettie 29:08

Yeah, there's this healthy tension, Ben, between in research, you want to keep agility. So you're looking at things, and I'll use our portfolio today as an example. You know, today's first generation uh cell therapies go after one target often. They're not dual targeted, they may not have um decoys or armoring on them, but our next generation all have those things. And so on the research side, you've got to keep this agility to keep iterating. And we've done this nicely in collaborations with groups like Penn, where we were testing a GBM construct and we realized, hey, it's not hanging around and it's not hanging around in the brain long enough. We're gonna bring it back in and we're gonna armor it and put it back into patients. There's a there's a level of agility that goes with um development of that construct. But then at some point, in order to scale, you've got to lock down. And it's that tension of knowing when to lock down. And different companies approach it differently. I think kite has had some amazing learnings and they're exceptionally good at it, but we still have tensions, right? We have our process development team saying, you've got to lock that. I'm not gonna be able to get it to clinical trials unless it's locked. And our research team thinking, hey, I just want to tinker a little bit and put one more thing on. Um, and it's something that we work closely together to figure out when it is that time.

Ben Comer 30:21

Um, what about uh acquisition strategy as it pertains to manufacturing? Um, you know, kite has has made some purchases in uh interior biotherapeutics, t-immunity. Uh, there's also a pre-gene deal in China I wanted to ask you about. Um, but what can you say about how um acquisitions, you know, play a role in terms of scaling manufacturing or doing different kinds of manufacturing?

Cindy Perettie 30:48

Yeah, maybe I'll start, you know, we were just talking about the glioblastoma construct that we created. That was with T immunity, right? The acquisition of bringing T immunity and allowed us to start our foray into solid tumors. So that was a really critical acquisition. And we got some amazing talent, uh, this tight collaboration with Penn, and it's really allowed us to start exploring a number of different solid tumors. And when I look at, you know, Interior, we have been watching the landscape and realizing aloe, which could be off the shelf, just really hasn't had the impact everybody thought it would. Um, but the early in vivo data looks really incredible, really promising. And so we knew we wanted to play in the in vivo space. There's two ways to play there. There's integrating and there's non-integrating. And we're gonna play them both. We're gonna make sure that we we place both mets. But for an off-the-shelf product, the Interiors acquisition, what we what we got was A, a great team, people who are really um deep in vector manufacturing and really strong IP. And the way they created their vector is in a modular way. So we can insert new binders, we can move things around really easily, and we really like that technology. And so as we look at what we can do internally versus what we want to acquire externally, we're always taking that into account. And we just didn't have the level of deep vector um manufacturing for in vivo type technologies. And so that was a really important deal for us to bring in-house. But we coupled that with look, we're all watching China, and China has just been amazing. Innovation out of China has been rapidly developing over the last five years, and they're really creating these meaningful opportunities for companies like Kite or Gilead to enter partnerships. And so, as part of the interior acquisition, in parallel, we did a strategic collaboration with PreGene. And the PreGene deal has been fantastic because it allows us to rapidly iterate and create in vivo constructs and then work with the pre-Gene team to test these very quickly in China, um, where the regulatory environment's just different. Um, they faster, right? Yeah, it's fantastic. And so when I think about um those pieces coming together, you know, we feel really confident on the in vivo, on the integrating in vivo space. We're going to be able to move a number of um in vivo constructs into patients in the next two years.

Ben Comer 33:12

Excellent. Uh, I think most listeners of the business of biotech will know the difference between allogenaic cell therapies and autologous cell therapies. Allogenaic being the off-the-shelf, the cell therapy is not from the end patient receiving the treatment, whereas autologous is is cells extracted, uh manipulated, engineered, uh reinserted into the patient. I I just I have to ask, you know, uh, I too thought that allogenaic was going to be the thing, you know, because you and you you mentioned that it hasn't so far worked out that way. Do you have a a thought about why that is?

Expanding Access Beyond Treatment Centers

Cindy Perettie 33:46

Yeah, it's interesting. So you need, you know, what we're discovering is for autologists, obviously it's personalized, but it needs to get deep into the tissue to be effective. And when it's not, um if it's not from your body, if that makes sense, there's uh often a number of factors that can impact its ability to have that level of efficacy. Now, the interesting part about aloe, and I think we're all watching closely for this, is to see if it can play a role in the autoimmune space. Is that is that the type of disease versus uh cancer or malignancies where it can really play a deep role? So some of those trials will be reading, some data has come out early data, but those trials should be reading out. And um that's something that we continue to watch closely. But what we saw with with in vivo is you saw really autologous-like um impact for patients, those first six BCMA patients that were published in the Lancet, um, those, you know, that was profound. And pregene actually uh ran that trial. And it's the first time I've seen something autologous-like. Um, we've been looking for that in aloe and haven't seen it.

Ben Comer 34:52

Um it's an interesting time for cell therapy development, uh, kind of following a previous downturn in investor sentiment. First, is that part improving? Do you have a sense of uh of, and you're not now as part of Gilead as beholden as you know, a startup cell therapy biotech? But yeah, it still matters, right? Uh, but do you have a sense of uh kind of where that sentiment sits right now with respect to you know, broadly cell therapy?

Cindy Perettie 35:22

Yeah, it's a great question coming out of JP Morgan as well, because we were, you know, it was a good chance to pressure test that. In addition to meeting some really great small companies, it was good to hear investor sentiment. So, you know, there was a there was a lot published when um Takeda, as an example, made a conscious choice to move out of the cell therapy business. But at the same time, some of these players were um moving out of the business. You saw AstraZeneca and Abby moving in full steam ahead, right? Making it.

Ben Comer 35:51

Yeah, with Capstan Therapeutics and in the case of AstraZeneca, SO Biotech, both billion-dollar acquisitions. Yeah.

Cindy Perettie 35:57

Exactly. And so I think, you know, the thing that I've learned from being in the cell therapy business is it does take a fair amount of investment to put in global manufacturing and to um integrate your own vector manufacturing. And so there's investments that you have to be willing to make. And if you're a small company, um it's going to be really difficult to make those size of investments. And honestly, Kite wasn't able to do it to the scale we have until Gilead acquired us. And so when I think about it, we've got these great innovation engines. Our Celex is a great example of that too, where you've got these small companies that are creating um novel therapeutics and at some point having them come together with larger pharma who can make the important investments necessary to scale this technology. I think that's what we're going to see more of in the future.

Structuring The Arcellx Partnership

Ben Comer 36:45

Do you think that a startup cell therapy company or even early to mid-stage cell therapy company kind of has to partner with a large company when they get into late stage and launch? Uh, is that, you know, in order to just be able to scale the manufacturing, I guess, uh as a as a primary concern, do you is it is it possible? I mean, sure, someone could invest, you know, hundreds and hundreds of millions of dollars in a company and maybe they could do it. But as a general rule, do you think that that part is necessary?

Cindy Perettie 37:17

Yeah, it's an interesting question. I talked to Rami, who's the CEO at Arcellix that you were referencing earlier about this. You know, Lanza, Wuxhi, there's a number of people doing the manufacturing today, but they're not, it's not their sole business. So their turnaround times aren't great, their reliability isn't great. And I think if you really want to see a product that's going to reach the masses around the globe, it probably will require partnership. However, I want to say that there's contract manufacturers, there's certainly CROs now that have a lot of experience in development of cell therapy. So I think you can get to a certain point. But when it comes to commercialization, footprint, all of those things, today it's going to require a partner. And that could change the time.

Ben Comer 38:02

Uh the current FDA administration uh seems to be pretty supportive of cell and gene therapies in general. Uh, they most recently, uh the you know, beginning of JP Morgan announced a more flexible approach for development commercialization and process validation. Um what do you what do you make of that? Uh I guess speaking specifically about this most recent announcement, um, does that benefit Kite uh directly?

Cindy Perettie 38:31

You know, it does. And I think the piece I'd say that I've been impressed with with the agency, and particularly um Marty Macray, is he's really interested in hearing from people, right? He did these listening sessions with CEOs, and I was lucky enough to attend one of them. He understands regenerative therapies are different. He's really thinking with his team about how to make the FDA more competitive. And so you think about the regulatory environment in somewhere like China, we just talked about Australia, is another great example of a regulatory environment that allows you to move regenerative products into phase one more rapidly than you do in the US. And so what I've seen is a real interest in moving in that direction. And some of these new guidances that you're seeing come out, I think are an example of their taking this very seriously and saying, how can we make the US as competitive as some other countries as it relates to development, but also recognizing the value of curative therapies and that these, you know, cell therapy is very different. And the paradigm that it offers in lymphoma is patients to have the potential chance at cure. And I think they take that very seriously.

From Antibodies To Cells: Scaling Parallels

Ben Comer 39:43

In comparison with markets like uh like China or Australia, when you think about, you know, the speed at which you can get, you know, into trials with with patients, is there is there something you would like to see happen uh that hasn't yet in the US that could, you know, perhaps move us closer to parity with some of these other markets?

Cindy Perettie 40:05

Yeah, I think it comes down to the I and D and being able to move these therapies into patients more quickly. And a lot of the steps that they're taking around uh process validation changes and things like that are enabling it. But I think it's been a it's it's a stepwise approach. And I think we're going to need to see um maybe larger steps in the future, but everything that they're showing is that they're moving in that direction.

Ben Comer 40:28

Yeah. Excellent. Um, we are coming up on the end of our time. Cindy, this uh it's flown by, but I wanted to ask you about your primary goals for 2026 uh before we sign off, and maybe what some of the the biggest challenges or or uncertainties are that you anticipate this year.

Cindy Perettie 40:48

Oh, thanks for that. So 2026, we're we're really looking forward to. So we have two marketed products, as you know, Euscarda and Dicardis, and we're continuing to provide those to patients around the globe, and that won't change. I think the the piece that's coming in is we have a launch the second half of the year for our multiple myeloma therapy. So a lot of a lot of kite is gearing up towards that uh for the US launch. And um, we're also rapidly advancing our portfolio. We shared a lot of great data at Ash. We have next generation lymphoma product that would replace Yescarta and Ticartis. And um we are starting a head-to-head trial of that product, kite 753, against actually against Yescarta. So we feel confident enough in our next generation products that we can beat the first generation of cell therapies. So our focus is really to continue to provide our amazing medicines to patients, have a successful launch within EDACL and continue to advance our portfolio.

Ben Comer 41:45

Oh, you've touched on an old hobby horse of mine, Cindy, the need for a head-to-head trial. So I'm I'm glad to hear that you're doing that. That's fantastic. Um, I really appreciate you being here and coming on the show. Thank you so much for the time. Thanks a lot, Ben. I really enjoyed it. We've been speaking with Cindy Peretti, EVP and Global Head at Kite Pharma. I'm Ben Comer, and you've just listened to the Business of Biotech. Find us and subscribe anywhere you listen to podcasts, and be sure to check out new weekly video casts of these conversations every Monday under the Business of Biotech tab at life scienceleader.com. We'll see you next week, and thanks as always for listening.