Building Cell Therapy Companies With Kenai Therapeutics' Nick Manusos

On this week's episode of the Business of Biotech, Nick Manusos, CEO at Kenai Therapeutics, talks about his experiences building cell therapy spinouts from FujiFilm Cellular Dynamics, learning from big pharma decision-making processes, and dosing the first patient with Kenai's allogeneic neuron replacement cell therapy for Parkinson's disease. Nick also talks about funding an early-stage cell therapy company and forging key manufacturing and therapy administration partnerships. 

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Transcript

Meet Nick Manuso And His Journey

Ben Comer 0:03

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 Nick Manusos, CEO at Kenai Therapeutics, a clinical stage biotech with an induced pluripotent stem cell therapy lead candidate targeting Parkinson's disease, a potentially disease-modifying therapy, which is pretty exciting. This isn't Nick's first biotech rodeo. He has been CEO at multiple biotechs and was the founding chief operating officer at Iota Biosciences, facilitating its sale to Astellas for $300 million in 2020. And he has worked in leadership positions at bigger companies like Baxter, uh, Takeda, and Abbott. I'm excited to chat with Nick about company formation, uh, funding and partnership strategy, and what he's up to at Kenai Therapeutics. Uh, Nick, thank you so much for being here. Great to be here. Thanks for the opportunity, man. Uh sure. Um, as we do, I would like to uh ask a couple of questions uh about your background uh before we get to Kenai. Uh I mentioned in the intro you've worked in in larger pharma and small biotech uh and companies in between. What are are some of your favorite professional experiences uh prior to Kenai? And then I'll ask you about the challenging ones.

Nick Manusos 1:30

Yeah, absolutely. So I've been very fortunate. I my first two decades of my 40-year career were at Abbott Laboratories, um, and I had some great sponsors um and mentors at Abbott Laboratories. I started out in manufacturing. I worked my way up quickly in my career to becoming a plant manager, and then went from plant manager to global sales and went from global sales to becoming a general manager, and then finally I was uh ended up being head of business development at a $5 billion joint venture.

Ben Comer 2:00

What kind of man, just out of curiosity, sorry to cut you off there, Nick. What kind of manufacturing? Was that small molecule manufacturing?

Nick Manusos 2:06

Yeah, it was small molecule manufacturing, yeah. Um, but it was also sterile fill and finish to small molecule manufacturing, multiple manufacturing experiences um back in the very, very first part of my career. So you can see what fortunately, what that translates to is being very versatile and diversified, whether that's functional or technologies. And so that's a wonderful, wonderful reward for my career. I enjoyed that very much. I had a great time doing that and obviously grew professionally and personally with all those experiences across functions and also a career track, could career trajectory. That said, that's the challenging part about it, too, right? The challenging part about it is a new function. The challenging part about that is a new division, a new technology. So you had to become proficient at immersing yourself in the new culture, in the new technology, in the new PL. Not every PL is the same financially. So you become good at, if you will, um doing that change. You become good at change. Um, and especially when it comes to assuming leadership positions, that translates well into the startup world if you think about it. Um, because in the startup world, and especially CEO, um, you have to be savvy at everything, everything from strategy to tactics, everything from finance to technology, you have to be versatile at all those things. And so that wonderful early part of my career at Abbott Laboratories, and then on to the joint venture, and then on to Takeda, great companies, great decision-making processes in these companies, great uh companies and how to do analysis and how to make proper decisions, um, and be when to be inclusive and when to be decisive. So learned a lot in my career working for great companies and was fortunate enough to have a lot of different experiences along the way.

Big Pharma vs Startup Decision Making

Ben Comer 4:04

Yeah, and uh I I want to just follow up on the decision-making process. I I would assume the decision-making process at a company like Takeda is a little bit different, you know, than a start, the decision-making process at a startup biotech company. Uh, I could be wrong about that, but are were there are there things that you experienced at some of those larger companies that you then brought forward to some of the you know more nimble and and smaller organizations that that you've led? Absolutely. So, yeah. So, first of all, you're exactly right.

Nick Manusos 4:35

The one of the biggest differences between decision making um at a startup and decision making at a big pharma or a big company is time, right? Um, speed at which you have to make decisions and the amount of analysis that goes into and the amount of people that need to be involved in that decision-making process. So when you're looking at cross-functionally at a large company, um, the state key stakeholders that are involved, you probably have committee levels that you need to go through and governance that you need to go through. At a small biotech, uh, not only do you not have the have the governance, not have the stakeholders, not have the committees, but you're right, in order to be successful, you have to be nimble. But I can speed up those processes. I learned a lot about those decision-making processes, the type of analytics, the type of homework, and those having gone through those experiences themselves already, having gone through those kind of situations already earlier in my career is beneficial towards making those decisions later in your career at a startup.

Ben Comer 5:40

It strikes me as uh an important skill or talent, uh, even to be able to embrace uh new things, whether it's new drug modalities, uh new new operational functions, different parts of the business. I mean, at some point you must have had to decide, you know, I want to learn about cell therapies and and and stem cell therapies. When at what point did you reach that point in your career where you know you you got excited maybe uh about cell therapies? I think you've worked in a couple of different cell therapy companies at this point, but um, but before that, uh you you hadn't, you know, you were you mentioned you started out in you know small molecule manufacturing. But what was it, I guess, about cell therapies in particular that uh attracted you and and how did you kind of get up to speed on on learning about the the potential and possibilities for cell therapies?

Why Cell Therapy And How He Learned It

Nick Manusos 6:35

Yeah, absolutely. You know, the the biggest learning to new technologies or going to new companies or going to new roles, the the beginning is always humbling, right? You have to check your ego, we have to check your ego at the door. Um, and and learning about cell therapy was no different. Uh, was no different. The future, you know, understanding the future and the promise of pluripotent stem cells was compelling for me. It always, like everything in most careers, is it starts with relationships. It starts with a relationship with someone or an executive who I knew at Takeda who'd gone over to this induced pluripotent stem cell technology company that FujiFilm owned, um, and said, you know, Nick, um, I I've moved over here. We could use somebody like you, we could use your skill set. So what I brought to the table from my years of experience, my business development experience, my strategy experience, my operational experience, they found very valuable. What I had to learn was the technology and and the promise of the technology for patients and for the industry. So um it was a you know win-win situation, if you will. Um, the tools that I learned in my at that time, and over 30 years of experience um in big pharma and and and some startup experience at that time as well. What I brought to the table helped the company, and then the company helped me um understand the promise of the technology and where to take it.

Ben Comer 8:06

What uh what circumstances brought you to uh to Kenai Therapeutics, uh, which I believe was was previously called Ryne Therapeutics. Uh is that right? And and you're the founding CEO.

Nick Manusos 8:18

Correct, correct. So the company that I mentioned, um FujiFilm Cellular Dynamics, is a world-renowned induce proponent stem cell company.

Ben Comer 8:28

Yeah.

Nick Manusos 8:28

Um, I was chief operating officer of that company. That comp when I came in around 2018, I remember I was recruited into that role um because they had just recently started the cell therapy division, if you will, of that company.

Ben Comer 8:43

Yeah, I was gonna say I thought that was fairly new, seven years or something. Yeah.

Spinning Out Century And Opsis

Nick Manusos 8:48

Yeah. So around that 2015-2016, they had started that division. I immediately got going and together with um other colleagues, and one of the first companies that we helped create was leveraging the backbone technology, the immune oncology backbone technology of FujiFilm Cellular Dynamics. Together, we got together with very prestigious venture capital, Versant, and we created Century Therapeutics. Then obviously, Century Therapeutics built their team up and took off with the technology and ran with it. And we had an ongoing partnership with Century Therapeutics, and then Century Therapeutics went IPO in in June of 21. Right. So that's that's comp that's company one. Second company, I was CEO of a majority-owned spin-out called Opsis Therapeutics, it was photoreceptor replacement therapy for diseases of the retina. So this is another cell therapy program out of FujiFilm Cellular Dynamics. We ended up partnering that around the same time as Century went public with Bayer/ Blue Rock. While we were doing these things inside of uh FCDI, FujiFilm Cellular Dynamics, when I was chief operating officer, was the Parkinson's program, the neuron cell replacement therapy program was embedded within there. It had yet to find a partner. I um after completing the IPO and the partnering with Bayer/ Blue Rock, I needed a timeout and I decided to go catch my breath. While that happened, entrepreneurs actually was happening while I was there. Two entrepreneurs, um, Jonathan Ye from Pise Ventures and Carter Cliff, together with uh my new my current partner, Howard Federoff, the ex-CEO of Aspen, and Dr. Jeff Kordover, the two scientific founders, were getting together and creating the spin-out. They spun the Parkinson's program out into at that time Ryan. And then six months later, they recruited me to be the first CEO of the company.

Ben Comer 10:53

Oh, okay. I see. Yeah, and um, I think Bayer had not owned Blue Rock for that long at that point because I remember writing about that acquisition. I mean, it that was fairly new at the time. I think Bayer was somewhat new to cell therapy at that time.

Nick Manusos 11:07

Yeah, Bayer was um, well, Bayer was also a big backer of Senti therapeutics as well. So Bayer Leaps by Bayer was starting to immerse themselves um in the technology. You can tell they believed in the promise of the technology. So back to the takeaway, three companies. So this turns out that Fujifilm Cellular Dynamics platform has proven, right? I mean, all three of these programs have ended up in the clinic. A pretty good track record uh for a company who started. So they're very good. Uh they're very good at the early part of the process, at the at the um founding of the program and the lead up um and the building of the from a process development standpoint. And obviously they're great manufacturers too. So that's great, great place to start a program.

Founding Kenai And Rebrand Strategy

Ben Comer 11:54

Yeah, yeah, absolutely. And so you you've you talked about these kind of three separate companies now that have spun out of uh of cellular di FujiFilm Cellular Dynamics. Um, could could you say a little bit more just about you know how how you did those spin outs, maybe with Century or Opsis, or or even you know, moving to Ryne, like how you connect with the scientific founders, how you you know initially put the team in place. I mean, was that your charge to kind of set those companies up once they were spun out?

Nick Manusos 12:25

It was different, it was different for every every one of them. Um we did with Versant and Century, it was company building. Um, together with their colleagues and our colleagues in Tokyo, um, with which helped orchestrate the initial deal um with versant um and Bayer, and then we at FCDI operationalized it and made it happen um with Century, with the new team at Century. Century brought their own scientific founder, um head of RD, and and they championed it from there, and then they actually hired us to perform. When I was um CEO of the majority-owned spin out of um Fuji Film Cellular Dynamics called Opsis, there was already a founding CSO in place, Dr. David Gamm, brillian brilliant pediatric eye surgeon. Um, and he he was my partner. He was my we together, we were partners. Um, and together, um along with other key stakeholders in the company, we were the ones who led the charge and partnered with Bayer Blue Rock. Um he was one of their even innovators, uh, original innovators in the techno on uh photoreceptor replacement therapy. So, and then the last one is Howard, uh Howard, Dr. Howard Federoff, our founding one of the founding scientists for the company. Um he later, after at the beginning of Ryne, he was not with the company, but later came to join us. Um, so we took what we had learned from FujiFilm Cellular Dynamics, brought that in, brought Howard in and built the team around the the company, and then we and we took off and ran from there.

Ben Comer 14:10

You took off and ran. You joined us as founding CEO. Um, what you know, and maybe talk me through the uh, you know, the initial you're you're joining the company, the decision to change the name uh that maybe came with an investor, it sounds like, uh possibly. Um, but take me through that point from your joining the name change all the way up through, you know, the the formal launch of the company, which happened uh last year, I believe, right?

Raising A Series A In A Tough Market

Nick Manusos 14:38

Yeah, so we were Ryne when we first formed the company while with seed investor uh from Sci-State Ventures. They were our seed investor. We were Ryne. Um, and the strategy when we started building the company at that time was well, the path was to get our IND cleared. So it was heavy CMC focused, heavy regulatory focused, and we built a team around that. And we already had um a seed investor in Ryan. So they initially seeded and funded that initial CMC and regulatory work that we had to get accomplished because we were getting ready for our and to be cleared and we needed, if you will, cells to treat patients with um when we started the clinical trial.

Ben Comer 15:21

Right.

Inside RNDP-001 And Donor Source

Nick Manusos 15:22

We started doing that work, we put the strategy together and we started pitching the company. Um, and that was in early 2022. And then by early 2024, obviously during the journey from early 2022 to early 2024, we needed a series A investor. We needed someone to fund the phase one clinical trial that we just announced, the replace trial, the phase one replace trial that we just announced recently. We needed to fund that. So that we were running up until early 2024, heavy CMC, heavy regulatory, built a team around that. Then we transitioned into more of a clinical stage company when we funded the Series A. And then with that, we changed the name of the company at the Series A in 2024 to Kenai. When we had uh a wonderful set of uh prestigious investors came in, the Collin Group, Cure Ventures, and the Alaska Permanent Fund came in. Nobody forced us to change our name. We wanted to change our name, we wanted to rebrand. Um, and we chose um Kenai after uh the Kenai Peninsula in Alaska, um, because we're a connections company and we're about connections and and delivering neurons uh to the brain and connecting those cells together and with the existing environment is one of the um the solutions for Parkinson's patients who've um who've been robbed, if you will, of dopamine production and therefore their quality of life, what you take for granted every day, walking across the room and getting the remote, brushing your teeth, they've been robbed of that. So we want to replace those lost cells and get that dopamine production going again. So that's uh I'm circling all the way back to the importance of connections and that Kenai and the Kenai landscape. So we because of that natural transition and how much it made sense from a company name standpoint, we decided to go with Kenai. And uh that's why we rebranded ourselves and we've been going with that ever since.

Ben Comer 17:27

Well, I want to uh I want to talk about your lead clinical program uh in just a minute, but I I I have to follow up on, you know, funding an early stage cell therapy company in 2024, uh a tall order, you know, not not an easy thing to do. And I wonder what you might, you know, say to to the listeners out there who are, you know, it's kind of similarly or or still a difficult time to find uh find money for early stage uh cell therapy companies. Is there anything you would say about you know how you were able to do that, you know, find the money to to get this asset into the clinic?

Surgery, Dosing, And Immunosuppression Light

Nick Manusos 18:07

Yeah, there's uh there's a lot of factors. Um I don't want this this to sound too simplified, but you know, persistence, persistence, um, unwavering persistence, and believing in the mission, like the mission I just described to you, um, restoring what Parkinson's disease has stolen from patients. We believed and we rallied around that mission. Um, and from everyone that was part of the company at that time, uh dedicated to that mission, whether it was the story, whether it was execution, whether it was um anything that we were, any milestone we were trying to achieve at the time, being resilient around that, being resilient around that. Um, and then and uh we also believed in ourselves. Uh, we believed our cells were different. We have the data that proves our cells are different. They were developed at FCDI, they were developed uniquely, they were optimized for what we call potency. Um, and potency means efficacy. Potency translates into dopamine production, which again, uh circling back to what I had mentioned previously, that's what Parkinson's robs from patients is dopamine production. So our cells uniquely are highly potent because they were developed to be potent when they were at FCDI. They did extensive lead optimization and preclinically and FDA recognized preclinical models, our cells are different. They show superior potency. So that data, you know, we rallied around that preclinical data. Our investors were engaged in that preclinical data. So persistence, dedication, the data, the powerful data that we had, all of that came together. Um, and we were able to put together, as I said, a group. Great, great syndicate uh with great partners. Not only are they great from a funding standpoint, but they add strategic value. They have there, they're great advisors for the company as well.

Ben Comer 20:10

How much did you raise in that series A? 82 million. Okay. And we're we're talking about um RNDP uh 001. Uh this is uh Kine's lead development candidate. Um you talked about you know the potency of these cells. Where do the donor sources come from for these? Because this is uh an allogenaic cell therapy, uh, meaning it's it's not cells from the individual patient uh who's who's receiving the treatment.

Trial Timelines And What Success Looks Like

Nick Manusos 20:38

Yeah, thank you. Um you're exactly right. This is an off what we call off-the-shelf all genant. Those are kind of the same thing, cryo-preserved um cells. The benefits of that, of course, so we as you said, we have a disease-modifying approach. Replacing the lost cells in the brain is uh disease-modifying approach first, because the current treatments for Parkinson's only address symptoms and efficacy wanes over time. So we have a disease modified, disease-modifying uh approach for RNDP001 with um with from a cell replacement therapy standpoint. So, as I mentioned, our and our cells are are different. So we have the features, which is off-the-shelf, allogenaic, cryopreserve versus autologous. Um, we are we're induced pro potent stem cell versus embryonic. But to get back to your original question, we have a single adult healthy donor. Oh, okay. So we were and which we're able to characterize. For example, we know that this donor does not have Parkinson's disease. So we're able to identify, we don't know who the donor is, but we're able to characterize and test the donor. Um, and then we can bank, we we, if you will, we take that blood sample from that healthy donor and we strip away everything that made it a blood cell, and we take it back to a pluripotent state, a very early state. And then from there, and we and we what we call bank those cells. And from there, I can source that bank later and then coax those cells into becoming the neurons through that lead op process and that optimization process that I mentioned previously to be the neurons that we want them to be optimized and optimized for potency.

Ben Comer 22:26

Right, right. Well, I just want to reiterate, you know, what you said um, you know, about the unmet need in Parkinson's disease. I mean, it's been a notoriously difficult to treat um uh condition. Uh there's nothing that is a disease modifying therapy that that's available on the market right now. Uh so if this, you know, RNDP001 were to be successfully approved by the FDA and marketed, I think it's you know, uh not a stretch to say this would be an enormous uh therapy for for patients. Um you dosed the first patient uh this month, uh a major milestone. Um can you uh maybe just describe the dosing and administration process, what that looks like? And I'm also curious because this is an allogenaic cell therapy, does it require uh immunosuppression uh for the patients who receive it?

Nick Manusos 23:25

Yeah. So the process is we start with those uh PROL preserved neurons, um, and we ship them. They're stored in liquid nitrogen, and we ship them to the site which is going to perform the insertion of the cells in the center of the brain, in the putamin of the individual that has Parkinson's disease. Um we thaw those cells, we prepare those cells to be so they're in a in a in a state in which we can actually um insert those cells into the center of the brain surgically with a with a special device. So once the cells get up into the operating room, this is a unique operating room. It has an MRI, or people a magnet. And the reason we're in a magnet is we need to be very, very precise when we insert the cannula into the brain, um, very, very carefully into the center of the brain and deposit the amount of cells that we want to deposit in the center of the brain by uh um a neurosurgeon, a well-established neurosurgeon. Um the patient is under general anesthesia while all this is happening. Um, and then after uh the procedure is over and we've deposited the number of cells that we want to deposit in the butamin, um, the patient um then recovers and is able to go home uh relatively short time after that.

Manufacturing Scale And Tech Transfer

Ben Comer 24:52

And is there immunosuppressant drugs uh co-administered or do they have to take those or or no?

Nick Manusos 24:58

Yeah, there's uh there is immunosuppression. We call it immunosuppression. Dr. Federoff likes to call it immunosuppression light. It's not it's not immunosuppression like you would if you had an organ transplant, for example. Okay. Um the so um yes, and it's 12 months. Um, and then we taper them off um from some between the period of nine and 12 months, we'll taper them off.

Ben Comer 25:24

All right. Okay. Um, so you you've dosed your first pace patient. Uh, when do you expect, I guess, um, I don't know, top line data, or or if you have a kind of ballpark for when you expect that trial to be completed?

Partner Ecosystem And CIRM Support

Nick Manusos 25:38

Yeah, so we plan to finish enrollment next year in 2026. Um, and then we'll have the key dates or key milestones from a reporting of results standpoint will be at six months and 12 months. And the key things we're gonna be looking for, so remember this is a safety study. So that's the most important thing that we're looking for, the fact that this is safe and well tolerated, and they'll report any adverse events if should they have any to their to their clinician. But the key things that we're looking for is one of the things we're gonna be looking for in addition to safety, is we're gonna be taking an image of the brain. So we have an image of their brain pre-surgery, and then we'll take images of the brain post-surgery at those milestone uh time points that I had mentioned. And we're gonna be looking because we can look to see um through imaging whether the brain is producing more dopamine than it was producing pre-surgery. And that'll be evidence of engraftment and most importantly, innervation of the cells within the brain. We'll be looking so we can take a picture and get a pretty good indication of whether that's starting to happen or not. So imaging will be one important thing that we'll be taking a look at. And then the patient has a diary, we'll be looking at to see how the patient feels and the patient reporting whether how they change in in terms of how they feel. And the last thing is the clinician itself will be doing an assessment using standard um rating scales, movement disorder, society, uh rating scales, and they'll be measuring. So those will be the three things the images, how the patient feels, and the movement disorder specialist rating and see looking at the baseline and seeing if there's any changes in how the patient's doing at those at those time points. We'll be reading that out all of next year.

Ben Comer 27:36

Uh, we've talked about the unmet need in Parkinson's. Um, you I believe are targeting not just the earliest cases of Parkinson's, but moderate all the way up to severe patients, which I believe is a you know a large patient population. Uh you also mentioned, you know, a specialist physician administering this drug. Do you do you have a sense? I mean, is this going to be delivered in like an academic medical center or do you have a sense of uh you know just the the number of um uh of care locations where a product like this could be uh administered in the US?

Funding Paths And Market Outlook

Nick Manusos 28:14

So um ClearPoint Neuro, our partner on the device delivery to the brain um partner, um, they have about 50 sites in the US so far, and 90 worldwide, and and that's growing every year. So um we're feeling and that we're feeling good from uh from a starting point. Um your first question was related to yeah, just the size of the population. Uh is it's right. This is um moderate to moderate severe patients. Um there's 90,000 new patients every year. There's 1.1 million patients in the US. We think there's probably 250 to 300,000 patients that'll be eligible for cell therapy in the US in the US alone.

Ben Comer 29:09

Okay. Um, what about manufacturing? As you as you move you know up into later stages of of clinical trials. Um, what what are your plans for scaling up uh manufacturing? Um, yeah, what what do you what are you gonna do there?

Lilly Gateway Labs And Team Hubs

Nick Manusos 29:24

That's one of the benefits we have as a company. So we already have a manufacturing partner, we don't have to tech transfer from academia to a CDMO and then have the CDMO go from their pilot plant to a commercial site. Um we're tech transfer FCDI is tech transferring to itself. Okay, nice. I was I was chief operating officer when we opened the brand new facility that the product is um that our cells are being manufactured in right now. Um, so it's a great state-of-the-art facility. It has all the capabilities to do things clinically, and they're gonna invest to make that facility commercially viable as well. So we don't have to move sites. Um, we're in great, we're in great shape from one of the big technical risks from a CNC standpoint, and that is tech to tech transfer. So from a scale, scale up or scale out standpoint, we have if that those powerful preclinical results that I mentioned earlier translate into human, there's examples in cell therapy where the FDA uh embraces what we're doing because of what you said, because of the the need for uh individuals with Parkinson's. Um the FDA embraces that and it's offering potentially for us if our data looks good in an accelerated path. So we may be able to skip phase two and go right to a pivotal trial if our data um looks translates the way our preclinical data in in human trials. So that's exciting. Now the uh that's kind of like the good news and bad news from a CMC standpoint, right? So now we've got to go faster. And because we got to go faster, um, and we we we think we can do this. Uh Derek High is our chief technology officer. He is um tremendously experienced um in this space. Um, so what we may do strategically is what we may do is scale out first and then scale up instead of just making bigger batches, we may make uh more batches and then go to bigger batches as an interim step to embrace to embrace the potential for an accelerated path.

Pipeline Beyond Parkinson’s

Ben Comer 31:35

So when you say uh more batches instead of bigger batches, does that mean more kind of spread out facilities or or just more? No, it within and also your that facility you mentioned is that that's that's Fujifilm as a partner uh that's manufacturing that or no? It's in yeah, it's in Madison, Wisconsin.

Nick Manusos 31:55

Okay, yeah. So the site is in Madison, Wisconsin. Um Fujifilm Cellular Dynamics. Fujifilm bought cellar dynamics, cellar dynamics was in Madison, Wisconsin. Got it. More batches happens in the same facility. We don't need another facility.

Ben Comer 32:12

Okay. All right. Well, um, you mentioned uh Clearpoint Neuro. That's that's your partner who's actually working on the the administration of the therapy. And then your your third partner, CR uh C I R M. What what did what role do they play?

Nick Manusos 32:28

So that's the California Institute of Regenerative Medicine. That's actually a funding. That's um oh, okay. That's a funding arm. And we we have uh we've been very fortunate um thanks to great relationships um with CERM, and obviously they believe in what we're doing as well. Um we've just been fortunate enough to receive our second um award from from CERM. Um so yeah, so we've got we have this, I call it the Kenai ecosystem. That was one of the first things that Howard and I worked on building. We've got this wonderful ecosystem that we've built of the gold standard in induced protopotent stem cell development and manufacturing with cellular dynamics, the gold standard in delivery of cell and gene therapy safely to the brain with ClearPoint Neuro. We have great, we have a great syndicate um with uh the Collin Group, Cure Ventures, the Alaska Permanent Fund, and we have CERM as great funding partners who add strategic, not only funding partners, but add strategic value to the company as well.

Patient Advisory Board And Design Input

Ben Comer 33:36

Uh you mentioned uh speaking of funding, uh you mentioned the the Series A, 80 million. I'm curious if there's a specific uh kind of funding goal that you're working toward, whether you know you see the kind of endgame as an acquisition, as you know, an IPO and then a deal, or or as just becoming a commercial biotech and and selling this product uh yourselves or or through a partner. I mean, do you have you and I know you're probably going to be opportunistic, uh, of course, but um, do you have, you know, is there kind of a a first path, you know, that that you're pursuing with this company right now? So right now we're incredibly focused on this phase one trial.

Nick Manusos 34:24

Um again, if that preclinical data translates into humans, I I have firm belief if we just take care of these individuals with with Parkinson's and that data translates, the rest will take care of itself. Yeah, we're we're very aware of what's going on in the markets around us. The I IPO market's been challenging for quite a while. We don't know what the future is going to bring there. That said, the XBI was up 30% in the second half of last year. We're looking at $100 billion in follow-ons recently, but and the MA market is hot as well. So um we're aware of all these factors um and conscious of those factors, and we will need to source uh funding, whether that by do dilutive or non-dilutive for for what we hope is an eventual pivotal trial. But in the meantime, we're just trying to trying to execute on that phase one trial, the phase one replace trial that I that I um had just that we just kicked off, then you just read about.

Ben Comer 35:28

Got it. Um, I read, I think it was a press release about uh Kenye being selected to join the Gateway Labs community. Um Lily Gateway Labs. Uh, can you uh I was not familiar with that. Can you explain what that is and maybe how Kenye was selected uh to join this community?

Near‑Term Focus And Pivotal Planning

Nick Manusos 35:48

Yeah, thank you. Yeah, we're honored and pleased um and fortunate to be part of Lily Gateway Labs. Um Lily partnered with Alexandria, um, and together they invested and built a wonderful innovation hub in San Diego. Um, and we're taking advantage of that. Um, and our um cell engineering team is there, and we have other CMC people um there and other scientists that are there um in our that San Diego site. So it's an innovation hub accelerator. I don't know the exact criteria that uh that Lily used to select their partners who get to be part of the companies that um they partner with on that space. Yeah, not everybody gets in, and we feel very fortunate to be a part of it. Um and they're wonderful. Well, first of all, San Diego, what a great place from a talent perspective. Absolutely. One of the legendary, I've been going to San Diego for 25 years, um, and great, great biotechs and successful biotechs have come out, um, come out of San Diego area and the great research institutions there. It's a great, it's a great place to hire talented people. And so not only are we thrilled to be part of the Lily uh system and the Lily Alexandria system, but we're also lucky that San Diego is such a great place to build a team.

Ben Comer 37:15

Where is the company headquarters? You have these operations out in San Diego, but where's home base?

Nick Manusos 37:21

Uh home base is um, I would say home base um administratively is um Boston, um, but operationally we're we're in San Diego.

Personal Priorities And Closing

Ben Comer 37:30

Okay. All right, got it. Um we talked about uh RNDP001. That's your lead um clinical program targeting Parkinson's disease. You also have RNDP002 and 003 uh in in preclinical stages. Um could you uh maybe just uh give me the highlights of those or maybe what's next for for those two candidates?

Nick Manusos 37:57

Yeah, those um we we have um plans. We're in the very early stages. We have plans to engineer our lead program, if you will, um edit um our lead program to perform. So the OO2 program is going to be designed to perform uh an edit that um excretes a protein, that not only will we replace the cells. So we we talk about it uh um from a when we do uh the three Rs, replace, repair, and restore is what our goal is for the LEED program. The OO2 program will take the repair piece not only um to the connections within the environment, but also hopefully resurrect the damaged cells that Parkinson says they're the cells I've not we have not lost the cells yet, but they're damaged. So we can hopefully be able to not only replace what's lost, but resurrect what's there. Wow. Okay. And then the the OO3 program is designed more for enzyme deficiencies and addressed a subset of the inherited form of the population uh of so you know, of the 100% of Parkinson's patients, about 10% have inherited forms of the disease. Our LEED program addresses the 90%, the idiopathic, but there's um subsets of the inherited form, and O3 will address one of the subsets that exhibits an enzyme deficiencies.

Ben Comer 39:25

Okay. So you're uh the company is kind of exclusively focused on Parkinson's disease, it sounds like. Um yeah.

Nick Manusos 39:34

And uniquely. So I describe it as simply as this our our strategy as a company is to address the entire, all the needs of Parkinson's um individuals with Parkinson's disease, whether that's early to late or inherited versus idiopathic. Um, that whole you know three dimensional area of needs, uh we uh we have a long term strategy to address all those needs. Because as you know, Parkinson's patients, yes, what we mean when we think of Parkinson's individuals with Parkinson's disease, we think about the traditional challenges related to gait and tremor. But Parkinson's patients also suffer from cognition, sleep challenges, other inflammation-related challenges as well.

Ben Comer 40:20

Even digestive challenges, I've heard of. We're seeing some research about, you know, the gut-brain axis. That's really fascinating. We're finding out uh more and more uh about that. But it's uh it's uh I like the ambition. It's a very difficult development space. You're you're going all in on you know, kind of the full story of Parkinson's disease from a patient perspective. And and and speaking of patients, I I wanted to ask you about this. I saw on your website that uh that that Kenai has established a patient advisory board uh comprised of Parkinson's disease patients as well as a a clinical researcher. Can you tell me uh, you know, maybe how that how that came to be and and how the board interacts with the company?

Nick Manusos 41:07

Yeah, um that came to be and championed um by Dr. Fedorov and relationships. He's this is not his first Parkinson's company. He's had multiple um, because he has a passion for it, Parkinson. He has relationships with um patients, individuals with Parkinson's disease. So it started uh there, and then we obviously recognize the benefit, the benefit of understanding and having access to the voice of individuals with Parkinson's disease, both um strategically and tactically, right? So how can we whether that's whether that's having input into clinical trial design from from those individuals' perspective, all the way to um how to pursue patients um across a diverse population. So um, and and we've had we've yes, we've had a formal kickoff, but we also have, you know, I would call ad hoc relationships where we participate in charitable events together, um, or we we seek out advice um um for for pursuing uh other milestones from from select individuals. So it's probably as much as it is formal as it is informal as well and ongoing.

Ben Comer 42:27

Yeah, okay. And what will would the the patients that are on the patient advisory board potentially be eligible to participate in in trials, if not a phase one, like you know, later on potentially?

Nick Manusos 42:41

So I can't speak. I don't know the those patients' profiles, but because we have a very select, very select set of criteria, an extensive criteria as you can imagine for a phase one situation. Sure.

Ben Comer 42:52

Yeah, yeah, absolutely. No, but I I think I think that's interesting. I mean, uh uh most CEOs will tell me, you know, we're we care a lot about the patient voice, we do various outreach, but I I don't often see an actual patient advisory board established as uh as part of the company. So, you know, that that was um interesting. And um I I wanted to ask you about it. Uh I think that's great. Um we are uh uh coming up sort of to the end of our our time here, Nick. Um maybe we can end with, you know, what you're I know you're focused on the phase one trial uh right now, but you know, looking ahead to 2026, um, or is there anything else you could say about your professional priorities at Keenai? And then I'd also love to hear about any personal priorities that you have for 2026.

Nick Manusos 43:45

Yeah, thank you. Yeah, the way I would categorize it, I would think short-term and and longer term, as I've already mentioned, short term, we're focused, we're intensely focused on this phase one trial. It's one of the most important things we have going on in the company right now. And uh all of us in the company are focused on that mission, and that mission being to restore what Parkinson's disease has stolen from these individuals. Um, and so we and we believe in ourselves and we believe in our ecosystem and our approach, and we want to make this phase one trial happy. It's what we've been working three years to get to, and it's really important that we want to get this. This the second thing we have to do, and in parallel, and it almost feels like it conflicts sometimes with it, right? Is we're also excited about the future, as I mentioned, whether that be CMC scale up or planning for what could be what could possibly be a pivotal trial. So we have to focus on the near term and execution, but also begin the process of planning because if if given the opportunity, uh, we want to be ready to seize the opportunity again with that same uh mission in mind um to restore what Parkinson's has stolen.

Ben Comer 44:59

Do you have a sense of how big that pivotal trial might need to be?

Nick Manusos 45:04

Um we have watched um the space and what other similar companies have done. I can't specifically say guarantee, but it could be um a hundred patients with a likely with a control R.

Ben Comer 45:19

Manageable. Yeah. All right. What about uh personal priorities? You got any got any personal goals uh for next year uh that that you'll be thinking about?

Nick Manusos 45:29

So I'm very again very fortunate. Um I early next year I should have my fifth grandchild. Oh, congratulations. Thank you. So we'll we'll be embracing that early in the year and having uh enjoying that at the same at the same time we're per pursuing our professional professional goals as well. Um yeah, looking forward to spending time with with family and enjoying that.

Ben Comer 45:54

Absolutely. Um well, great. Uh Nick, thank you so much for coming on the show. It's a real pleasure speaking with you.

Nick Manusos 46:00

Likewise, man. Thank you for the time.

Ben Comer 46:02

We've been speaking with Nick Minusos, CEO at Kenye Therapeutics. 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 our 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.