Late-Stage Development And Commercial Planning With CervoMed's Matt Winton, Ph.D.

On this week's episode of the Business of Biotech, Matt Winton, Ph.D., Chief Commercial and Business Officer at CervoMed, talks about preparing for the late-stage development of neflamapimod in dementia with Lewy bodies (DLB) and commercial planning for an anticipated launch. Winton talks about CMC and manufacturing scale-up, partnership, communicating with regulators and the benefits of global regulatory alignment on protocols for the Phase 3 trial. He also talks about building value through HEOR and payer engagement in parallel with clinical development, and pricing in the context of policy changes and shifting global launch dynamics.           

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Transcript

Welcome And Guest Introduction

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 pleased to welcome Matt Winton, Ph.D., Chief Commercial and Business Officer at CervoMed, a clinical stage company developing neflamapimod. I'm gonna get it right by the end of this. Very good.

Matt Winton, Ph.D. 0:24

Very good.

Ben Comer 0:25

Neflamapimod for several age-related neurologic disorders. Its lead indication, which is entering phase three trials, targets dementia with Lewy bodies. Uh, it's received FDA's fast track designation. Matt joined CervoMed in 2025 after a stint as chief operating officer at Inozyme Pharma, culminating in a $270 million acquisition by BioMarin Pharmaceuticals one year ago, last July. Before that, Matt spent nine years at Biogen, where he was steadily promoted and served as Biogen's senior vice president, head of U.S. multiple sclerosis franchise before leaving to join Inozyme. We'll talk with Matt about preparing for late stage development and scaling up operations at CervoMed, what the commercial uh the commercialization planning looks like, uh, and the new nuances of commercializing products for CNS indications. Matt, thank you so much for being here.

Matt Winton, Ph.D. 1:23

Thanks, Ben. Glad to be here and uh looking forward

From Ph.D. Lab To Business Skills

Matt Winton, Ph.D. 1:26

to the discussion.

Ben Comer 1:26

Yeah, glad to have you. Uh, I wanted to start off uh as we do on the Business of Biotech with your background. You got a PhD from the University of Montreal and then went back to school and got an MBA. Is that right? That's right.

Matt Winton, Ph.D. 1:40

So uh too many degrees, but all very uh important, sort of the trajectory of my career. Um was always uh really interested and passionate about science growing up. And so all through university and through grad school, uh was really focused on science. Um, following uh PhD in Montreal, moved down to Philadelphia here in the US and did a postdoctoral fellowship. Um, and after about four or four and a half years, really got more interested in the commercial side of uh biotech, of drug development. Um, had little experiences throughout. We started a small biotech company out of the lab that I was doing my doctorate degree in. So got a little bit of a taste working in the company as well as you know, completing my degree. But was that something that that you invented in the lab that you spoke of? So it was, yeah. So it was based on my uh PhD project. Um, we uh were targeting uh pathways and and developing drugs for spinal cord regeneration. Um and so uh my investigator, based on sort of the technology, uh, did a little bit of a spin-off. And so I got to sort of work in that company and help move it uh through I and D until uh my duo was over and then uh jumped down to Philadelphia for the postdoc. But it was a great experience just to sort of have those two kind of different um levels of science from an academic science lab, you know, to a more sort of rigorous, um, definitely uh a more focused sort of company approach and small biotech, uh just trying to get a drug to I and D and try to license it out. So great, great, really great experience. And then um, yeah, when I made the switch after my postdoc, I realized that you know academic science was really not where I wanted my career to be. Um I was uh at the UPenn and I was actually playing hockey on the Warden team. I had a lot of friends there, um, and they had a biotech of business club, and I ended up through them joining that and got some exposure into consulting um and into hedge fund investing uh on uh on the science side. And so I realized what I was lacking um was really a business foundation uh in my education. So um we ended up moving to Boston. I ended up, you know, switching careers and getting into consulting. And as part of that, uh past math thought it made sense to do an MBA in the evenings. So I was consulting during the day and then running, taking the green line for those that are familiar to in Boston down to BU, doing uh classes at nights and weekends and trying to uh kind of you know grow in in the consulting world as well. But uh really, you know, really helpful to as someone who never took those classes, you know, throughout school to get that education.

Consulting Lessons And Joining Biogen

Ben Comer 4:28

Yeah, yeah. That sounds like a busy time. Uh and I wanted to ask you about um your experience in management consulting. Um, prior to joining Biogen in 2014, I believe, you worked as management consultant at at Lyrink Swan uh and then Inventive Health. Did those experiences advising uh, I'm sure a lot of different companies um help you decide to make the switch over to an internal company role? Yeah.

Matt Winton, Ph.D. 4:56

Um, so both of those experiences were were great and a little different. Um, Lyrink at the time, you know, an investment bank had a consulting arm which sort of sat in the middle of the investment bank side and the equity analyst side. And obviously, as a compliant organization, there's firewalls in between who can talk to who. And sort of the consultants were able to interact um with uh the equity analysts as well as some of the investment bankers. And so really good sort of crash course in working with a lot of private equity firms, venture firms, um, corporate strategy uh to really understand you know what are those critical questions, to be able to synthesize large um amounts of data, uh, ask the key questions and come out with recommendations for you know senior level C-suite executives. Um Biogen was actually my largest client when I was at K. Oh, they were okay, yeah, which became inventive. So I always joke, um, you know, Biogen and I dated for several years uh before we made it official. Um and I worked there in some sort of form as a consultant for about three years or so before, sort of get to know the organization. And, you know, through that they asked me to come inside um if I'd be interested to come inside to help launch one of their multiple sclerosis drugs. And I already knew the organization, and it's a fantastic science-focused um organization that has a really patient-driven mission. So it was sort of an easy decision for me. You know, worst case, I went in for a year, got some launch experience, and came back, and it made me a better consultant. Um, best case, which what it was, it's kicked off a almost a decade-long career, you know, at Biogen. And um, it was a time at Biogen where there was, you know, I think anything that could happen to a pharmaceutical company could happen. There were launches, there were drugs pulled off the market, there were generic entries, um, you know, changes of CEOs, different indications, different strategies being, you know, rolled out. Um, and so, you know, really good um sort of learning and how to work in a larger company through, you know, different uh different matrices of an organization. So um it was a great time there.

Ben Comer 7:09

Yeah, absolutely. And that it strikes me as probably obvious to a lot of people who've done this, but um, kind of getting to know a company from the outside first uh before you join them as a consultant, really understanding and and having a view into a lot of different companies, not just Biogen, um, and ultimately deciding uh to go there. That that makes a lot of sense. Um, and you've talked about the you know, the sort of crash course in a lot of different areas that you received during your time at Biogen.

Leaving Big Pharma For Startup Life

Ben Comer 7:38

Um, what made you decide to leave and join enzyme in in 2023? You were chief operating officer there, I believe.

Matt Winton, Ph.D. 7:46

Yeah. Um, so you know, nine years at Biogen, things were going well. I was leading up the uh multiple sclerosis commercial unit in the US. Uh, so a little north of $3 billion in revenue, five products in the franchise. Um, and um, you know, MS is a very difficult market. It's a very crowded market, uh, a lot of generics, a lot of branded pharmaceuticals, um, but really enjoyed um owning sort of that PL and having sort of that that broad remit. Um and one of uh a colleagues who I used to work for uh while I was at Biogen was actually on the board of Endozyme. And Enozyme startup company uh focused on rare pediatric bone disorders, so completely different space, different, you know, from neuro from what I was used to. And um, they were looking to uh you know bring in a new CEO, and that CEO is looking to bring in a new leadership team. Um and she threw my hat in the ring. And so um, you know, the more I thought about it, Biotron was great, and and I really enjoyed um sort of being the one uh to help support earnings calls and to help support board presentations. And, you know, given MS was one of the largest business units there, um, and sort of being in the room um was great, but I I wanted to sort of you know bet on myself and sort of be in the seat, right? And be the one who's you know delivering those conversations and meeting with investors and you know, uh being part of the earnings calls and being part of those board meetings. And this was a real opportunity from building teams and franchises to building a company. Um, and you know, we had a very innovative product at innozyme, and it's always great if you can sort of help uh, you know, pediatric rare disease patients and this drug uh, you know, is able to do that. Um and so moving over to enzyme, building out the commercial uh and operational side of things, um, getting our phase three up and running and uh and just getting you know back to to a startup environment, which was which was sort of fun. I mean, I joke, I did everything at enzyme from medical affairs to clinical ops, CMC to planning a fire drill. I mean, that's fun of um of you know small biotech startup is that you know you you've got to find a way to get things done and get it done quickly and efficiently and you know, cost effectively. Um sometimes, you know, that that involves lots, lots of different hats.

Ben Comer 10:21

Yeah, I as you were talking about biogen, it made me remember a conversation I had recently about multiple sclerosis and the really dramatic improvement in outcomes that patients have seen over kind of the time that you were there, the last 10 or 15 years. Um I remember the, I can't remember who I was talking to, but they were saying like you really don't see people that often in wheelchairs uh with multiple sclerosis anymore, which goes to show um just how much change has come to that particular disease area in the form of therapeutics that uh have really moved the needle. So that that must have been pretty exciting um for you to watch.

Matt Winton, Ph.D. 10:59

Yeah, it's an amazing space that has sort of progressed not only in terms of available therapies, but in terms of how it's diagnosed and sort of the um how physicians think about treatment. Um and you have uh a broad brush of different therapies. You still have some some new therapies um that are uh still in development. But I think that really goes to show you the power of sort of biotech um and what we can do and really change a disease um in you know 15, 20 years. Um and you know, you you really rewrite the course of the life for these patients and for these families. Um and MS is is a great example of that.

Ben Comer 11:39

Yeah, absolutely. Um, you moved to enzyme, you've taught you just m talked about that a little bit, all the different hats you were wearing. That's an N they they were um the the lead candidate. Is it enzyme replacement therapy? Is that right?

Matt Winton, Ph.D. 11:52

Yeah, it was an enzyme replacement therapy. It was a technology um that came out of Yale University that was licensed by a founder, um, was able to raise financing, took an IPO, and then um, you know, we were a public company uh and built to uh to a global uh phase three, uh, which we were sort of in the middle of. Uh and then as you mentioned, the acquisition from Biomarin came last summer.

Ben Comer 12:18

Yeah, a big act, and that was, you know, to roughly two years, I guess, after you joined Anazyme. Um, I wanted to ask you about, you know, how kind of what that was like uh going through that deal. You mean do you do you work to set up a company for acquisition? Are there things that you can do that that make it more appealing? Was that kind of the ultimate goal? Or are you just sort of working toward, you know, commercializing a drug and remaining opportunistic for you know whatever happens in between? How how did you think about that?

Matt Winton, Ph.D. 12:50

Yeah, I mean, I'll speak for for for us. Um but so our goal was to kind of commercialize this drug. It was um uh rare, maybe you could say ultra-rare pediatric disease, um, you know, about 10,000-ish patients globally. So small footprint, um, really specialist-driven disease. So something that we, you know, thought we could commercial uh commercialize ourselves. Um and that was part of the goal uh of going to Indo Design was to go there and help, you know, build a commercial organization. I've been part of many very successful commercial organizations, but um, there was about tweaking and improving and um, you know, kind of adding on and and getting better um and pushing the team to do more. This would have been building something from scratch. Um and so that really was the goal. Um, and I think the whole team at Enozyne was really um passionate about that. Um I think that's what you get with rare disease. Not only, you know, do we you know almost every patient and you know the families, but to actually invent something that that wasn't there years ago and to bring hope for these families, um, I think that was truly our our mission. Um but you know, you you never know what happens in this uh in this business. And we always say, you know, this is that was our plan, but if someone wanted to wrestle this away from us, you know, we we understand. Um for us, you know, getting this drug to patients um was paramount. Um and if a company came in that had better resources, more resources and and even maybe more ability to get this to more patients, you know, that that made sense. And I think this is an example of that. Um, where ultimately, you know, if you're in this business to develop new therapies and to help patients, sometimes you also have to admit that maybe you're not the best ones to to launch this drug. And that somebody who also um has experience in these spaces and somebody who already has, you know, a network of distribution and who has a field team, um, these aren't trivial things to build. Um, and so you know, that discussion is as we get into thinking about commercial organizations, sort of the buy, build, you know, rent type of discussions is is really an important one. And one you got to be honest with yourself about is that are you truly the best, you know, person to do this? We could have done it. Um, but given the two opportunities in front of us, and you don't always have those two opportunities right in front of you to uh to sort of weigh against each other, was you know, raise more money, potentially um diluted raise for investors um to commercialize this or a potential company who you know may be in the the right fit to take this drug further. And and so that was the decision that uh, you know, that we made. And, you know, it was it was a bittersweet decision because I think we all were excited and and hopeful that that this was something that we could do

Acquisition Reality And Joining CervoMed

Matt Winton, Ph.D. 15:51

ourselves.

Ben Comer 15:51

Well, the good news is it looks like you have another chance uh to do this. We're gonna pull on this thread of uh building uh a commercial organization uh in just a minute. But I want to get a little bit of background on ServoMed. You so enzyme was acquired by Biomarin. You stayed on maybe for a little while, and then um what circumstances brought you to ServoMed?

Matt Winton, Ph.D. 16:13

Yeah. Yeah, no, I stayed on a little while to help with the integration, wanted to make sure uh it was smooth and a successful integration. Um, and then my goal and and what I told my wife um was taking some time off. We're gonna use this as first time to have a a break in in several years. Um obviously Biogen uh is an is an intense um environment, you know, going through the acquisition in a small uh company like Inozyme sort of was an intense environment. So was gonna take a break and relax. And I got a call from somebody who you know I respect and trust. And they said, Oh, I want you to to meet um the folks over at Serbomed. And I gave them the line, I'm not interested, I'm taking time off. I want to sort of, you know, just relax for a bit. And they said, Oh, just one meeting. And so I said, sure, okay, I'll have one meeting. And I met John Allen here, who's the CEO and founder, uh, as well as sort of the broader management team and you know, really uh experienced board of directors here. Um, and the more people I met and the more I learned more about the company, the more I really uh liked what they were doing, like the science, uh something I'm very familiar with. It's in um brain-related uh age disorders, which is what my postdoctoral fellowship and a lot of time um, you know, through my early career was spent in. And so one discussion led to two, led to many more. And next thing I know, I was um actually in the interviewing process, um, which wasn't the goal, but um turned out to be uh a great fit and it's a great company. And so I think technically um it took about a week and a half off between the two. Um my wife said I give you two weeks. I sort of took the under, um, but she was obviously right. And then uh, and then you know, joined here in October. And it's been uh it's been a great uh, I guess nine, 10 months um since, and a lot going on here and and and really uh really great, exciting space to to be in.

Ben Comer 18:14

Yeah, and you came in as chief commercial and business officer. Um you're about to enter uh phase three trials. Uh you may be we'll we'll talk about that in a second, but uh I'm curious just about you know, coming into the

What A Chief Commercial Officer Does

Ben Comer 18:26

company with that title. What are your key responsibilities? You know, what are your immediate priorities?

Matt Winton, Ph.D. 18:32

Yeah, um, it's a good question. Um, I think it it depends the day. Um, someday it's more on the commercial side, some days it's more on sort of the business side. Um, I think on the commercial side, I think it really speaks to, you know, what we're trying to do when we think about commercialization here and making sure that it's tied into our phase three design, make sure it's tied into some early critical decisions that we want to make as a company to set us up for success as we you know move through late stage development. That we're not thinking necessarily of commercialization is something that you bolt on to after you get a positive readout. Um and so, you know, kudos to the board and to um John or CEO for for thinking maybe a little bit differently about how you bring in sort of commercial um thinking and commercial strategy uh early on. Um and so you know, there's there's some of those, but there's obviously we're early. So there's no true sales teams or or marketing teams, right? And and we're being um very smart and staging how we're how we're thinking about that. The business side, though, that really focuses on um partnerships and how we thinking about our corporate strategy and um external discussions with partners, uh, both on the clinical side as well, you know, potentially future partners for this drug, which we've announced recently. We are, you know, looking for a potential partnership to help us with with the B DLB indication as we you know look forward um to um this trial, this this phase three trial, which is a big undertaking. And so I think you know it ultimately varies, but it it is a sort of a rich um rewarding role because you get to kind of wear those those you know different hats um and sort of really be at the excitement of you know building and thinking through a commercial organization, but also how can we partner? How can we find um different ways to do what we need to get done? And again, that goes to that sort of buy, build, rent. You know, maybe it's not always us, maybe there's other people that are doing what we need better or differently, and um thinking about that uh is is how I spend a lot of my day.

Manufacturing And Phase 3 Preparation

Ben Comer 20:45

Yeah, and I imagine one of those partnerships would be for scaling up manufacturing. Is that correct? I mean, is that something that you've you've been involved with for the phase three trial at this time?

Matt Winton, Ph.D. 20:56

Yeah, so uh, you know, manufacturing is one of those um funny kind of uh aspects where you kind of get no credit for it when everything runs smoothly. Yeah. But if you have sort of a hiccup in manufacturing, then you know you're you're sort of the the worst people of the world. And I think as we you know sort of look towards kind of what our phase three um trial is going to be and and in patients, not only for the trial, but you know, post-trial and and long-term kind of commercialization, making sure we do have, you know, a robust CMC strategy, making sure we have sort of the ability not only to um meet the sort of the need and the capacity of the trial, but you know, we also prepare for those critical things that we're gonna need for an NDA, you know, that we're gonna need as you know, we look past the trial and hopefully we do uh have a successful uh result that we can take this to regulatory agents globally, you know, with our CMC package as well, um, and to to to bring this towards approval. I think those are all critical things that you wanted to work through now. Those are things that are um, I wouldn't say cheap to do early, but those are things that are better to do early because they can get really, really expensive when you try to rush them late. And some of those things even learn CMC, you know, drugs take time to manufacture. Um, some of those timelines you can't change. Um, and so doing some of those activities that do have lead time uh to make sure those are in your plan is is sort of critical. And and I think there's uh, you know, a lot of aspects, you know, in partnership with our uh CMC team that we're you know working through now.

DLB Unmet Need And Pipeline Bets

Ben Comer 22:35

Uh we're talking about um Nefla Mappy Mode, uh, which is slated to interface three in the second half of this year. Is there anything more specific on that that you can give at this point?

Matt Winton, Ph.D. 22:47

Yeah, so um I think that's sort of the the guidance that we give. As I mentioned, we're working to find some partnerships to sort of help with that, and that may impact um that you know timeline. But um, you know, we'd love to get this trial you know started as soon as we can. Uh dementia with Lewy bodies, there's extremely high unmet need. Um it's it's a space where there's currently no approved uh therapies for this disease. Uh it's the second most uh common progressive dementia behind Alzheimer's disease. You know, we we use the stat, but roughly about 12% of all dementias are dementia with Louis body. Um so there's a lot of unmet need here, but very high patient and caregiver burden. Um, you know, in addition to sort of the cognitive deficits associated with disease, which are more um activities of daily living and executive function than memory, you also get parkinsonium symptoms. You can have visual hallucinations, attenuation fluctuations, and you know, sleep disorder. So it's it's it's a big burden. And, you know, the sooner we can get uh this drug to patients and the trial started, the uh the better uh for everybody. Um but in the meantime, we also do have a rich pipeline. Um, we have uh ongoing phase two trial and primary progressive alphasia, which is a subtype of frontal temporal dementias. This is the disease that the actor Bruce Willis um unfortunately has been diagnosed with. Um so it's a dementia, but it's really focused on speech and language, um, trouble finding sort of words. Um and we have a phase uh two A that will have uh some initial biomarker data that we said would be um available by the end of this year. Um, and then we announced earlier this year that we are very excited about um we were selected to be part of uh Experts ALS, which is a program in the UK. Um and it's similar to uh the Healy platform trials that we here we have here in the US uh at MTH. So it's a platform trial for ALS, it's across 11, I think soon to be 17 ALS centers in the UK. It's a program that's funded by the UK government uh and UK charities. Uh, and they look for potential promising drugs for the treatment of ALS, which you know is a horrible disease. And um we they have asked us to be to submit, and we submitted, and um, we're selective. Uh and so you know, hopefully our arm is going to start by the end of the year uh in this trial, looking at uh biomarker effects of nephilmath mode and ALS, specifically neurofilament light chain. And uh and hopefully by the second half of next year, we'll we'll also have some data in ALS, which which is really exciting.

Ben Comer 25:32

Oh, that's awesome. I hadn't heard about that. Um, you you also have a preclinical candidate too, I believe EIP200. Uh, and you haven't chosen an indication yet for that one, I think, right?

Matt Winton, Ph.D. 25:43

Yeah, I think as we look at our drug, and and we have, you know, right now one drug, multiple indications, we're also looking at different formulations, different co-crystals, um, different ways which we can, you know, use uh, you know, one drug and multiple indications and do that in a strategic uh and and sort of clean setting. Um and so you know, we're looking at kind of different formulations and and different aspects of uh of the drug to be able to use broadly. And so um some some preclinical work still to do there. But um, you know, we're we're excited to potentially have multiple shots on goal with with a robust pipeline and you know different indications that hopefully could um you know bring uh this drug to market.

Ben Comer 26:26

Yeah, is that kind of uh pipeline and a product clinical strategy one that that you like and that was appealing to you? I guess it was for for Georgia.

Matt Winton, Ph.D. 26:34

Yeah, and it's funny because in a in azyme was very similar. I think it's sort of a common um sort of you know strategy for some biotechs. Um I think here um it's been really um science-driven. And I think it's a good example of science-driven um drug discovery. Um initially, you know, nephew mapmode was was licensed out of vertex pharmaceuticals. Um, and our first uh indication uh was an Alzheimer's disease. Um but over time, I think the science told us and sort of the MOA of this drug and what we've learned more about DLB is that Nephimapmode was uh a great drug to try um in DLB first. Um that sort of progressive nature of dementia with Luli bodies, you know, and the stat I always like to quote is that from diagnosis to um need for full-time care or sort of nursing home care is about two years. Um, so it's a very progressive, very fast-moving disease, which is horrible for patients and families, but from a drug development perspective, allows you to run shorter, more efficient trials. Um, the phase three trial, which we have alignment on with global regulatory agencies, is 32 weeks, 300 patients, um, you know, placebo controlled. So it's not a huge um, you know, trial. It's not thousands of patients in multi-year. So hopefully this will allow us to get an answer quicker. Um, obviously, biotech is a lot about time and and money, you know, do things as quick as you can with with the less amount of money. So being able to run you know shorter, more uh efficient trials, but still get that rigor and that clinical data that you need to take to the FDA is important. But then over time, science has evolved. The literature has shown that you know, P38 alpha, which is the target of nephamath mode, uh, has a strong um you know link to probably progressive alphasia. Uh, it's been shown, which is a tau-mediated subtype of FTD. Um, TDP43, which has really evolved in sort of uh most of forms of ALS, uh sort of non-um sporadic, non-genetic familiar forms. Um there's been a lot of literature recently and a lot of signs showing that there is a direct link between TDP43 and P38 uh and defects in exonal transport. So I think as the field has moved, it's really influenced us to think about where this drug could also be effective and how we can sort of expand our thinking and expand our pipeline and really follow, you know, the science. Um and uh I've had some you know great mentors in my life, and you know, one of them um at Biogen, you know, always told me if you follow, you know, the science and you do right by the patient, you know, you'll always be able to look at yourself in the mirror in the morning. And I and I think that sort of follow of the science is something that we've um really tried to emulate here at at ServoMed and make sure what we're doing is is sort of the right, you know, for the patients. And and I think you see that in in our pipeline expansion.

Science Rationale Behind New Indications

Ben Comer 29:47

Uh was that Al Sandrock by the time? That was Dr. Wallace. We had him on the show. Yeah, we had him on the show recently. It sounded like something he might see.

Matt Winton, Ph.D. 29:54

Yeah, no, Al's full of a lot of wisdoms and a lot of uh you know things. It was uh he was uh a wonderful uh you know colleague and person to get to work with at Biogen along with you know many others.

Ben Comer 30:07

Uh Nephelmepamode is a small molecule drug, is that right? Okay. And you mentioned you you've um you've spoken, I think, with the FDA about phase three trial design, and you also mentioned global

Aligning FDA And Global Regulators

Ben Comer 30:20

regulators. What can you give me a sense of what the I mean, I have a sense of it at FDA, the meetings that you, you know, that you have in the lead up to various uh stages of clinical development, but from a global perspective, what does that kind of engagement look like? And and maybe which global regulators have you interacted with?

Matt Winton, Ph.D. 30:38

Yeah, I I I think it's it's it's a very similar process. So last year in in November, we announced that we had alignment with with FDA. Um and our interactions, you know, I think you know, we were fortunate were were were great interactions, you know, everything was running on time, um, and I and I think you know, very positive from our standpoint and and from the agreement which we received. Um we wanted to go with the FDA first and make sure that you know we we did have alignment and we did have a sense of what this trial could look like, um, you know, what the design was, what the primary endpoint, you know, was, how many patients you know we were gonna need, and and how we were thinking um, you know, about um our statistical um plan for for the trial. And once we got sort of that alignment, then we felt you know confident about taking that to the European Medical Uh Agency as well as the MHRA in the UK, um, and presented them, you know, with that trial that we had alignment on, as well as all of our scientific data, you know, from our phase two, uh two phase twos, our two A and two B study, all the preclinical data, all of our CMC work. Um, and you know, we're we're fortunate enough, and we announced earlier this year that we've got similar alignment, you know, consistent uh uh phase three design, um, with a primary endpoint being CDR sum of boxes, which is you know a key uh sort of the gold standard endpoint in DLB, a key endpoint they use in a lot of Alzheimer's trials as well. Um we were also very excited that we got approval for one clinical trial, which was um sufficient to, if positive, submission to uh to take to registration. Um, and you know, 32 weeks, which is a time point that we have data on before. So we felt comfortable with that time point and um you know the 300 patients, which um allows us to get enough patients we need to reach our safety database and you know, obviously get the uh power um and statistical uh significance that we need. Um from that, we were able to finalize a protocol for the phase three, and then sort of be a consistent sort of global, you know, phase three protocol, um, which is always nice to have. So you're not constantly changing protocols or different parts of the world have different protocols, different endpoints, maybe um, you know, different dosages. Um so we have, you know, we we announced our dose for a phase three, we announced the trial design.

Ben Comer 33:16

Um and then we want to each one of those uh changes or amendments are time and money, right? Correct. They're time and money.

Matt Winton, Ph.D. 33:22

Um, and and for a small organization to also to manage different um, you know, trials and and and you know, from the from the different sites is is difficult. You also this goes back to sort of having kind of a commercial mind, you know, having different labels across different parts of the world can also be very, you know, difficult and how you think about marketing a drug if the label is different in one country versus the other, um, you know, can can cause some sort of complications. Uh, you know, I mean, people um talk to each other and and people see that, you know, maybe if there's a different um timing of how the drug is is used or different doses, you know, it can impact how how you kind of think about you know using this drug for physicians, it can create confusion for patients as well, um, and for payers and and uh health uh technology uh assessments. If you have multiple different um indications going around, it can be difficult. So we're very happy to have one um kind of phase three trial. And then lastly, which is you know, um just sort of recently, uh we announced that we are having discussions with PMDA, which is the regulatory agency in Japan. Um DLB is is uh a well-understood, well-known disease in Japan. Uh a lot of uh the sort of founding um and and fathers of of DLB, if you will, uh are Japanese um physician scientists. So it's a very well uh understood, well-diagnosed disease. And so um we think it's a potentially great commercial market. Um and so we're in discussions now with with PMDA over there to see if we can uh have approval to uh include uh Japan in our global phase three, um, or what would a potential you know Japanese path to registration look like. Um so we're hopefully this will be a full global um trial, uh big undertaking for uh a small company, but I think you know, given the high unmet need, um, it's something that you know we we want to take on. And I think we feel is is the the right thing to do. And in turn, we have the right partners to do that is is going to be an important step.

Building Commercial Strategy In Parallel

Ben Comer 35:37

You uh said a little while ago that you didn't want to bolt on uh a commercial strategy, you know, later on in the process. You you wanted to start it uh early, um, you know, even before you've entered uh phase three. And I I wonder if you could maybe talk a little bit about um the beginning of that commercial strategy and and and to what extent I guess commercial strategy should commercial strategy should influence uh phase three trial design.

Matt Winton, Ph.D. 36:06

Yeah. Um so I always you know like to think of um commercial readiness as something that shouldn't start right after phase three, right? It's it should be a parallel work stream, it's got to start years earlier. It things that can be calibrated on risk and probability of success. And there's a lot of work you can do um without spending a lot of money, right? Um and I think that's a lot of um mistakes you know biotechs make is sort of that treating commercialization as something we can just kick down the road um and just bolt on after you or just throw money at, yeah. Or just throw money at. Um and you know, I like to think of it more of a sort of overlapping tracks. You have um the science, the organization, the market, all of these kind of mature together. Um, and it's important to think about them um together. Um I think the flip side, what uh a mistake a lot of marketers make is that everything has to be done at once, um, or that you have to um have a fixed calendar in terms of what commercialization could look like. Um commercialization, you know, should be built in stages, right? And should be tied to sort of a real inflection points of things that you know that are critical early on to drive those decisions, right? To drive the endpoints, to drive what you think the label could look like, how people are gonna react to a product and really understanding that target product profile work. Um, because that's obviously gonna drive what types of data you're gonna need to not only get regulatory approval, but to ensure you have an optimal pricing and access strategy, to ensure that KOLs um understand you know the disease, what type of education needs are you are you gonna have, um, and and then ultimately, as we talked about, that manufacturing supply chain, you know, scale up and planning. I mean, those are all things um that you know are hard to do uh a couple months before you, you know, you want to approve this truck. There are things you can do, you know, and and I think being confident in knowing what what you can and you can't do is also critical because I think many of your listeners will, you know, be in a situation is like when runway gets tight or when you know there needs to be certain cuts um, you know, to ensure that the the cash burn is enough to get to data or to you know get to regulatory approval, one of the first doors that gets knocked on is commercial. And people, you know, oh, do you need to do that? Do you need to do that? And so, you know, being confident in these are the must-haves, these are the nice haves. And you know, these, you know, are ones that if if we have the money, great, but I can make a decision, you know, with 70, 80% of this work. And, you know, this work just gets us, you know, uh uh uh this 20, 30 percent just gets us maybe a little um you know, tighter or or more refined in that decision. But um know what you can give back and and know what you where you just sort of dig in. And this is sort of what what what you need to be make a successful commercial organization.

Ben Comer 39:19

Yeah. So is it do you think it's the the best way to build a commercial organization is in a kind of iterative way? Uh like let me maybe we can use an example. So let's say, you know, you're the drug enters phase three trials, you have a really positive like midpoint readout. Does that trigger another set of commercial actions at that point, or is that not how you think about it?

Matt Winton, Ph.D. 39:43

Yeah, no, I think that's exactly right. I mean, to me, I think the most critical thing is, you know, to start earlier is the pair and the H E O R grammar.

Ben Comer 39:52

Okay.

Matt Winton, Ph.D. 39:52

Um, and really understanding, right? Because at the end of the day, um, great drugs don't always result in great commercial successes. Right. More and more so. More and more that's the case. More and more so. And that and it used to be great science, great drug, um, you know, and you know, you're giving big commercial splash, yeah. Big commercial splash. Um, especially in crowded markets, you know, thinking about how you differentiate it where there is already existing drugs that have large shares. Um, you know, obviously there's a lot of pressure on thinking about pricing. You know, we have new laws and most favorite nations and and other things that are coming into place that can really impact and affect, you know, how you think about or how you should think about a launch, where you want to launch, what geographies. Um, you know, if you all have other um drugs in your space, you know, what does a new drug in that space mean? Is there, you know, generics are your, you know, orals versus antibodies or infusions? Um, are you first to market, right? And that brings a whole uh lot of positives, but it's also there's a lot of work that you know needs to be done on educating payers and and um uh countries and and governments around it as well. And so to me, getting that right is is critical. Um the marketing, the market research, you know, a lot of that you know can come later. Um and a lot of that you can be creative about at a small company. Um, I'm a big believer of you know getting out there in front of customers in everything you do. And it's amazing when you spend an hour or two with a doctor what you can learn uh without doing a large, expensive uh market research project. Um, just asking them questions and understanding how they see patients and where they get patients from and what their concerns are and what their needs are. Um, so it's not necessarily you don't do it, but you get creative uh about what you do do and you focus on on what's kind of critical. Um, you don't need a big team of marketers early on. You obviously don't need um salespeople, you don't need a lot of those types of activities. But as you move closer and closer to launch and as things get more and more de-risked, um, those are the types of things you you should be thinking about is um, you know, what does that look like? I'll say though, that should not prevent you from thinking about what your commercial footprint should be and how many of those people you may need. Um, and use the time wisely to refine and and think about, you know, how should this drug be sold and what's the physician experience, what's the patient experience, what are the suit uh services and solutions I'm gonna need to think through. Um, but just not necessarily pulling the trigger in the hiring and in sort of the execution of some of those activities.

Payer Evidence And Endpoints That Matter

Ben Comer 42:46

It seems to, you know, you you mentioned like health economics and outcomes, research, um, having conversations, you know, kind of uh very focused conversations with physicians. I I could see how those would be pretty doable at a smaller company. The payer engagement piece, though, strikes me as a bit a little bit of a bigger lift. I mean, is that is that something that um that you have already started, you know, at this stage in in development? Is it something that you know you'll do soon or or or halfway through the trial or or how do you how do you manage that?

Matt Winton, Ph.D. 43:19

Yeah, and I think you know, there's there's sort of the the optimum and sort of the gold standard model. And if I if you know, if I was a biogen um or any other large pharma, you know, there'd be a lot of that going on and you'd have whole teams of that doing that, right? And so, you know, um you have to also be realistic of of what you can and and can't do. Um, and so, you know, I think there's a a little bit of of that, you know, as well as that you know, you do want to do as as much as you can. And this is where for a small company, um, if you you know we're gonna bring on someone, I think you know, there's a good opportunity um for uh an internal pay or HR person. If if that's not necessary in the budget, there's Obviously, you know, consultants, vendors that are really sort of good in this work in the space that can help with this work and help you sort of think through what your value propositions are, what your health economic modeling is going to look like, how you what types of data gaps that you do have that if you go to a UK, if you go to a Germany, or go to a France, you know, that you're going to need. And even here in the US, more and more, although maybe it's not as formalized, you know, some of these activities are important when you think about Medicare populations, when you think about sort of larger, you know, disease states, you know, different pairs, uh and there's several of them now in in the US, both government and private, you know, want some of this information as well. And so, you know, thinking through what's sort of the best way to develop it and you know, what is the amount that you need to help drive some decision is is critical. But ultimately, if you get approval, great, but that you know doesn't necessarily mean that patients are gonna have access to your drug or you know that um reimbursement is is gonna occur. So um, you know, that is kind of also the the critical things that you know you got to be thinking about.

Ben Comer 45:18

And so is that kind of payer engagement work that you just described happening now, like ahead of the start of phase three at Servimed? It is.

Matt Winton, Ph.D. 45:26

So yeah. So I think I think it's a different extent, right? So I think we're thinking too about endpoints, right? And I think this is sort of an interesting, you know, kind of uh um, I wouldn't say it's unique to to CNS, but I think it's unique to several um therapeutic areas. You know, what is your endpoint and how is a payer going to view those endpoints? Um, and what else are you gonna need uh in that trial to show? Um be it biomarker, um, be it sort of, you know, different types of clinical endpoints, you know, is there any um quality of life endpoints, um, scales that you want to include in the in the trial? Because you know, that's kind of what sort of global payers are looking for. Um and I'll give you one example. So CDR summer boxes and and a lot of um kind of CNS endpoints are composite kind of endpoints, right? They're sort of qualitative scales, you know, that maybe look at cognition or look at function. They, you know, if it's depression or neuropsych, it may be around, you know, um, those aspects, hallucinations, agitation. Um, but it's sometimes it's hard for a payer if they're not familiar with this disease. And you got to remember, you know, a payer, a government organization is doing this for thousands of diseases, hundreds of diseases. You know, they're not necessarily experts in there, but what's a 0.5 change in that scale mean? Is that clinically effective? Is it not? You know, is it worth this 0.5 worth, you know, uh a six-figure price tag?

Ben Comer 46:55

Right.

Matt Winton, Ph.D. 46:56

Um, and so thinking about, you know, well, that's my primary endpoint because that's the right primary endpoint for, you know, this disease, but what else do I need as supportive evidence? And so, you know, for us it's progression of disease. So CDR cell boxes, if we show, you know, an improvement, and you know, they're luckily in that is in that endpoint, there is sort of some literature that shows what clinically meaningful is, which is you know, 0.5. Um in our phase two studies, we've shown greater than that. So, you know, we feel confident, you know, there. But progression is something, you know, are you um getting worse? And at what rate are you getting worse, or can we slow down the course of the disease? You know, is something that's important to physicians, it's something that's important to patients, and ultimately it's something that's important to payers. Um, and so having time to progression as a secondary point, you know, is a good example of bringing in something that, you know, can really help you with those discussions. Um, we do have quality of lifescales in there as well to show that, you know, not only, you know, our drug is having sort of clinical effects, but it's also, you know, changing kind of the burden on caregivers. Um, it's changing sort of how these patients are living their lives. These are all things you want drugs to be doing. And these are the things that payers want to see in order to be able to provide reimbursement and to pay for these drugs. Um, and sort of to help with those discussions. We've been we've been thinking a lot about that now. Um over time, you know, when we have data and we'll start some more, you know, discussions and sort of putting some of our models and and our presentations to front of payers. But, you know, I think for now it's it's a lot about kind of having those early discussions on what's necessary to have in the trial. Um, and then that will build on, you know, later discussions on sort of price and reimbursement and you know, patient populations as as we move forward.

Caregivers As The Second Customer

Ben Comer 48:55

Yeah, I think uh one of the unique things about CNS is that it doesn't have some of the same kind of hard biomarkers that you see in other therapeutic areas like oncology and cardiovascular, for example. So it it almost there's a there's a creative element, I think, there and an opportunity to really bring in, you know, the patient perspective to understand like what it really is that that you're trying to to solve for and demonstrate in a clinical trial, which I assume is is exciting to you.

Matt Winton, Ph.D. 49:26

It is. And I think I think the other sort of unique bit, which you know, I think we experience a lot, especially when you're doing age-related um disorders, um, there's a huge caregiver component. Right. And so that caregiver is almost sort of a second customer, right? And many of the caregivers are the ones making the treatment decisions. And so um, you know, thinking through also early on of how the best sort of position this data so a doctor can talk to the caregiver about what it means to be on your drug and what the benefit they're going to get. Um, and you know, to work with that patient and caregiver to make an informed decision. Um, and some of that is the data in the trial. Um, but what data are you gonna have that they're able to uh you know have those discussions? Um, you know, is not always um survival or tumor shrinkage or something that is very intuitive at CNS. And in some cases, there's no other therapy that's that's been there before. Um, and so you know it is it's something new to point to. Um I think the other thing, which as an industry, um, you know, it's our responsibility, um, but it's to ensure that we are supporting um the ecosystem. Um if you're in a fusion and you need fusion center capacity, you know, how are you thinking about that? Yeah, right. If you're bringing a new technology, you know, how are you thinking about that? And so, you know, we can't as an industry develop these great new drugs and these great innovations that, you know, you know, have high value and you know, cost to the system without helping the system be sustainable. Um, and so thinking through about if you're gonna need new aspects of drug delivery, um, you know, what does that look like? And and how do you start having some of those discussions? Because there's costs to health systems in that, um, there's additional costs, you know, to payers in that, and additional costs to patients, which you know, you want to make sure is not uh negatively impacting the adoption of your drug.

Pricing Pressures And Policy Shifts

Ben Comer 51:36

Uh I'm running out of time with you, Matt, but before I I let you go, um, I want to ask you about drug pricing, obviously a key piece of of launch and commercialization. Uh, you have, I think, some pretty deep experience in drug pricing and market access, both you know, before you join Biogen and at Biogen as well. And I'm curious if there are any specific uh pricing and access dynamics that that you're paying attention to or thinking about right now. You you referenced most favored nations earlier in the discussion. Um how how do you think about pricing right now? And what are the some of the key issues?

Matt Winton, Ph.D. 52:13

Yeah, I mean, when I sort of started my career, um pricing and specialty, you know, or or payer control and and sort of specialty diseases like MS was sort of minimal at best, if at all. Um and there was not a lot of focus on that. I think that is sort of dramatically uh has changed, you know, over the last you know, 10, 15 years. Um, I think there's a lot of um focus on overall cost to the system and and being able to um show uh proof and and and why you know uh a new innovative drug should be um you know priced uh uh commiserate with its value. Um and I think more so now the government is is getting involved um and putting some new legislations. I think the IRA and and sort of you know government negotiations is is you know sort of a space to watch.

Ben Comer 53:09

Yeah, that's something you have to think about as like a Medicaid, a Medicare Medicaid journal.

Matt Winton, Ph.D. 53:13

As a Medicaid space, um, I think most favored nations, you know, is is one as well, because you know, there was always the launch playbook. You launch in these countries first, um, and you know, that may change things. But also as a small biotech, um, if you look at sort of the acquisition space and the MA space, you also want to make sure you're not doing any decisions that may impact a large farm's willingness to uh acquire you or or to partner with you because, you know, of the pricing decision that you've made uh in this country or that country may negative impact larger, you know, kind of profit centers for them in the US or in in sort of Europe, UK, Europe four uh areas. And so I think you know, there's just a lot more strategic thinking that needs uh around that. Um and then also I think as you know, especially for drugs of aging, um, we all know sort of population trends and dynamics and where things are going. And so um these are um groups of patients that are increasing over time as patients get older. And so, you know, thinking about what it means to the system now, but also, you know, five, 10 years from now, what's the system going to be able to absorb and to and to take and to ensure that, you know, you're you're being a good citizen and and a good partner in that. Um so I think it's it's definitely changed a lot. And I think there's a lot more um pressure on um biotech companies and pharma companies to get pricing right. Um, I think it's a decision that you see now has been elevated up to the board and gets presented because it is a decision that can, you know, have huge benefit, but also can have a huge negative, you know, to a drug if if you get that wrong. Um and you know, nobody wants to be on the front page of the New York Times or in front of Congress because they they got that wrong. Right.

2026 Priorities And Closing

Ben Comer 55:04

Um, last question what are your top priorities for the remainder of 2026, Matt?

Matt Winton, Ph.D. 55:09

Yeah, so um, you know, from ServerMed here, we're we're really excited to uh have sort of the the interim data from Primary Progressive Althasia. Uh we're excited to start up our trial in ALS, um, sort of new indications for us. Um and we're you know really um interested and and excited to establish you know a partnership and move this uh phase three trial and DLB forward and um really do our best to move this exciting therapy further and and hopefully to a point where we can bring uh something new and you know to patients that are suffering from these horrible diseases.

Ben Comer 55:46

Thank you so much for coming on the show. Yeah, I really appreciate it. It was a great conversation anytime. We've been speaking with Matt Wenton, PhD Chief Commercial and Business Officer at Servimed. 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 weekly video cast of these conversations every Monday under the Business of Biotech tab at lifescience leader.com. We'll see you next week, and thanks as always for listening.