Business Of Biotech

Engineering B Cells With Immusoft's Sean Ainsworth

• Ben Comer • Episode 283

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On this week's episode of the Business of Biotech, Sean Ainsworth, CEO and Chairman at Immusoft, talks about transitioning from the research bench to entrepreneurship, his M&A experiences as a biotech founder, and why engineered B cell therapies offer a better, more patient-friendly administration for lysosomal storage diseases currently treated with enzyme replacement therapies. Sean also discusses FDA pathways, incentives, and agency engagement, and offers thoughts on the regulatory outlook for cell and gene therapies.     

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Ben Comer:

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 Sean Ainsworth, CEO and Chairman of the Board at Immusoft, an autologous B cell therapy company developing redoseable treatments that don't require immune suppression or myeloablation. We'll talk about Sean's journey from the Navy to the lab and into entrepreneurship, his founding of RetroSense and early development work in optogenetics, what he's learned from the acquisitions and deals he's been a part of prior to joining Immusoft, and we'll get into Sean's approach to building teams and raising capital and the opportunity to improve upon enzyme replacement therapies with engineered B cells. We'll also get Sean's thoughts on the regulatory outlook for cell and gene therapies. Thanks so much for coming on the show, Sean.

Sean Ainsworth:

My pleasure. Great to be here. And thank you, Ben. Appreciate it.

Ben Comer:

Glad to have you. You got your undergraduate degree uh in microbiology at the University of California, San Diego, and were in the Navy before that. Um how did you get interested in uh microbiology initially?

Sean Ainsworth:

Great. Uh thank you. Yeah, I actually, you know, going back to my high school days, that was just always my favorite subject was biology. Um, and uh so yeah, as you mentioned, I did a stint in the Navy right out of high school. Uh I guess I still needed to figure out what I wanted to be when I grew up, one might say. So uh after, you know, through the Navy, I I did uh get the GI Bill and uh put myself through college with uh with the GI Bill. Uh and so uh, you know, it was a natural uh progression for me to get into biology and and uh um and UCSD was just you know the perfect school for that, having been in the Navy at in uh in San Diego.

Ben Comer:

Right, at the base there, I assume. Correct, yeah. You did some uh some lab work on stem cells, uh gene modification to stem cells uh in in San Diego. Um can you describe, you know, what I guess what did you learn from that experience?

Sean Ainsworth:

Yeah, thanks. So uh it was uh it was really uh you know exciting science for me, and that's what uh uh drew me into that particular role. Uh, you know, one might actually think of it as kind of an early uh early attempt at gene modified cell therapies. Uh we were looking at uh uh modifying stem cells uh from cord blood to effect proliferation without differentiation. Uh and so the science was just immediately uh compelling uh to me. Uh I learned after a few years, you know, that uh it's the the the old expression 1% inspiration, 99% perspiration. Uh I spent a lot of time at the you know at the bench doing pipetting, uh running PCRs, uh counting colonies and plates, and and uh reached a point where I realized I didn't want to do that for my you know for my whole career. And so decided to uh to go to the business side at that point.

Ben Comer:

Well, tell me about your first experience with uh with entrepreneurship and maybe what the outcome was.

Sean Ainsworth:

Sure. Uh yeah, so that uh you know, fast forwarding a bit, uh I started a company co-founded out of uh University of Michigan uh that was called Compendia Bioscience. And Compendia was a uh gene modifying, pardon me, uh uh cancer gene expression profiling uh uh tool. Uh and so uh yeah, we launched that uh that goes back to 2006. Uh the company was ultimately acquired by uh Thermo Fisher and um $50 million acquisition, but uh the outcome was was good for everybody. We we never had to raise uh a lot of money for that, a couple million dollars. So that was a nice outcome. Uh and uh so yeah, that was you know uh uh an exciting endeavor.

Ben Comer:

So you went to the lab to starting your first company and getting a nice exit. That's you know, a pretty good first experiment, you know, on the entrepreneurial side. How where did you get the skills, you know, that were needed to make that happen? Um, you know, assembling a team, raising capital, uh getting to that exit point.

Sean Ainsworth:

Yeah. Uh well, I I actually um there was a bit of uh work in between the lab and uh my first entrepreneurial uh endeavor. So I I worked actually in a in an intellectual property law office in uh Tokyo for a couple of years. Uh my brother was an IP attorney, and you know, during back in those days, we we used to chat a lot about our respective work, and I was definitely intrigued by the whole uh um concept of of intellectual property and what how that applies in science and and biotechnology most specifically. So uh from there, I went to uh business school at Wash U in St. Louis and uh got into corporate development consulting. And so that kind of brought in uh the intellectual property experience, the lab experience, and uh the uh the international experience living in Japan. So that work was predominantly focused on uh the Asia-Pacific market, uh, but it did give me a lot of experience with uh, you know, the the concepts and the um the ins and outs of of licensing and uh and intellectual property. And so that led to uh you know that first endeavor of licensing a program out of University of Michigan and that that first startup.

Ben Comer:

And where did uh where did the funding come from from that? I mean, were you also kind of building a a network of investors and and meeting you know VCs and stuff like that at that at that time as well?

Sean Ainsworth:

Uh to some degree, yeah. Yeah. So uh I'll start by saying we actually had our first sale before we uh before we were established as a as a company. So I had to run out and open up a bank account to deposit our check, and we had to jump through some hoops to you know to make all that happen. So it's always nice having revenue uh, you know, at the outset. But uh indeed we we did, you know, I'll give credit as well to uh uh the local financing Michigan Economic Development Corporation uh funded us for I want to say a couple million dollars. And then we also did raise uh a couple million dollars in uh in investment capital.

Ben Comer:

Okay. All right, and so um you you had this uh this exit with uh compendium. What what did you do next?

Sean Ainsworth:

So I started a company uh in the meantime called RetroSense Therapeutics. Uh and RetroSense was a um the really was the first optogenetic approach to enter clinical trials. So that was a the gene therapy for vision restoration in in retinal degenerative disorders. Uh we took that into the yeah.

Ben Comer:

No, yeah, sorry, I didn't mean to I cut you off, but that was a big white space at the time, you know, very early company working in optogenetic therapy. And I mean, was that uh certainly it was a risk, but you know, how did you decide to select that particular IP for retro sense and you know take a kind of a big swing in a brand new area?

Sean Ainsworth:

Yeah, great question. Thanks. I mean, it's it's almost like it chose me. So uh the story there is uh, you know, the the the IP came out of Wayne State University, and they were doing some evaluation of a number of different programs and trying to assess whether you know it made sense to protect the intellectual property. And um, they like to talk to industry folks to get uh you know some sense of where where things have uh sticking power and not. And uh, you know, I I met with the inventor, uh Dr. Johal Penn. He presented his data and I was floored. You know, I I went home. I actually was chatting with my wife about it, and uh said, if this is, you know, if this actually works, um, this is one of the most amazing things that I'll probably ever see in my career. Uh and so yeah, we uh, you know, I long story short, I got back to the uh Wayne State uh tech transfer folks. I said, yeah, this is, you know, you definitely need to protect this intellectual property. It's gonna be important. Uh and they came back to me and said, Can you help us, you know, build out the the development plan and the business case? So uh I actually worked on that for about a year. And um I hadn't planned on uh jumping back into the entrepreneurial side of things, but uh, you know, I knew I would never find anything as as interesting, exciting as that. Um and so um yeah, I took the plunge that second time and uh and ultimately uh I think you know we we were acquired by Allergan after you know after dosing our first patient.

Ben Comer:

So yeah, two for two on you know being uh acquired on on companies that you know that that you work to to form and and launch. Um the you know, the in addition to working in a really innovative space and new space at the time, the you know, the other challenge you faced was a broad kind of macroeconomic situation. This is 2008, 2009, I believe, uh where you know kind of kind of similar to some recent years that we've had in terms of uh you know the investment sentiment and situation uh in in life sciences. But what what could you say, Sean, about you know how you built the team at RetroSense uh during a really difficult economic period and how you kind of secured the funding to get you to 2016 and the Allergan acquisition?

Sean Ainsworth:

Yes, uh so the uh the building of the team, you know, I think the uh first and foremost was uh finding people that shared the same passion. Uh so we were uh a small team, uh, but everybody that was on board really uh understood uh the importance of the science, the the potential impact to these uh to these patient populations. And um, and so uh you know that was kind of rule number one for me is let's let's find people that really get the importance of what we're doing, uh, the why, and uh uh really have share you know that that passion with me. Uh so um indeed we uh you know we did build out the team. Uh we did uh have some rough years. We uh, you know, as you point out, 2009 was still kind of a downturn in the market. Uh and looking back, uh there were no gene therapies even approved in the you know in the Western world at that time. So um I had initially expected that you know the first would have been approved in 2010, uh, and that got bumped out to 2012. So we um, you know, we started out with some uh angel investors that uh kept the the company moving forward. And again, credit to the you know to the Michigan Economic Development Uh Corporation and some of the other local uh funds. Uh they kept uh you know they kept us able to move the science forward, do some of those important uh IND enabling studies, which you know are not hugely expensive. And then um we started getting uh institutional investment after, you know, in that 2012 time frame, once uh once uh that first gene therapy was approved in the Western world. And you know, there there was a lot of excitement then around that time uh surrounding gene therapies. So uh we we uh we still didn't raise a tremendous amount of capital, about 15 million all in. So by you know, by today's standards, that's still a small uh small round, but uh you know that was sufficient to take the company through to uh to acquisition.

Ben Comer:

There's a a common refrain in the industry. A number of guests have said it on the show. We've published a byline piece fairly recently that that had it right in the headline. Biotech companies are sold, not bought. Um uh can you talk a little bit about you know how you kind of set up RetroSense to even be an acquisition target uh for Allergan and you know how you thought about that?

Sean Ainsworth:

Sure, yeah. We uh, you know, I I I think that uh any you know responsible biotech entrepreneur has got the end in sight before even founding the company. Uh and so not to say that I knew Allergan would be the buyer, but uh you're you're building to be either acquired or or or taken public. Uh and so we, you know, part of my every day was talking to industry, uh, understanding what they were interested in, what they were looking for, what kind of data we would need to uh you know to produce. And um we had actually uh, you know, if we can go take a stroll down memory lane, that was Allegan in 2015, I believe, was getting ready to do the reverse uh the inversion with Pfizer uh to uh headquarter Pfizer in in in uh in Ireland. And that uh we had had conversations with Allegan into the lead up of that, and then those got uh shut down, obviously, through you know, those those conversations, and that ultimately was uh uh that that uh inversion didn't uh you know didn't go through. So in the meantime, we had dosed that first patient. Uh, we were really excited by the data, and uh we we circled back to Allergan. Uh they were likewise excited by the data. You know, they liked us preclinically, they they liked us better once we had clinical data, and that's what led to the uh to the term sheet. So uh we'd had a couple of other groups that were interested and uh you know had discussed some terms with, and ultimately the Allegan deal made the made the most sense for us.

Ben Comer:

What is the the forum for those kinds of discussions? And I'm I'm thinking of you know a small biotech company who, as a good entrepreneur, as you've said, is thinking about you know an exit for the company, whether it's you know internal development and approval and commercialization or or some kind of deal. Uh I wonder, you know, how are is it happening like at conferences? You're making appointments with like the big players. Is there some other way that you're you know getting feedback from potential acquirers? Uh, how does can you talk a little bit about just how how those conversations happen?

Sean Ainsworth:

Sure, yeah. I mean, it's uh uh a bit of everything, I think, Ben. So uh, you know, I I think every uh CEO, you know, relies on their board to uh make introductions. Um certainly, you know, there's a lot of conferences uh that uh uh that that one attends. Um and there were several uh investment banking uh you know biotech days that uh uh were also really productive. And so I think it's a function of getting in front of the you know people on a consistent basis, familiarizing them with your your science, your progress. Uh eventually that leads to you know the CDA and if things are progressing uh as you hope uh ultimately to the acquisition.

Ben Comer:

And uh the acquisition uh by Allergan was for how much?

Sean Ainsworth:

So that was uh the headline value was 555 million.

Ben Comer:

Nice. Okay. And so what circumstances led you to to Immusoft uh after I think that was your you know your next company um after retro.

Sean Ainsworth:

Correct, yes. So um, yeah, you know, just to put a little bit of the uh concept around what we were doing at RetroSense, it was delivering the gene using an AAV vector to the eye. Uh intravitreal injection. Uh what that means is you know, you're you're delivering a very small amount of vector. And uh so from a safety perspective, I I had a lot of comfort with uh, you know, starting in an ocular uh setting. Um and so to put a little bit of, you know, to add a little more color still, as I mentioned, this was all before the, you know, before the first gene therapy was approved in the Western world. So um I was uh uh maybe taking baby steps, right? Let's let's get something to work in the eye before thinking about going uh beyond uh the eye and into systemic delivery with with virus vectors. So once we were acquired, um I went over to Allegan for a year to help uh transition that technology in. And um I was really you know thinking as my next act, I I wanted to find a modality to deliver uh a gene therapeutic systemically that avoided uh a lot of the issues with virus vectors, uh, namely the immunogenicity uh as well as immunosuppression requirements, um, and uh, you know, as well as some of the issues with with gene-modified stem cells, which are kind of the other primary modality for uh for gene therapeutics. Um and so in the case of gene-modified stem cells, there is, as you mentioned, uh requirement for uh myeloablation, you know, essentially chemotherapy that that wipes out the bone marrow. Um that typically puts kids in the hospital for you know four to six weeks. Uh, there are a lot of uh morbidity uh issues associated, and you know, unfortunately, uh all too frequently mortality associated with that. So uh our approach at Immusoft. Um I probably jumped ahead a little bit, but uh uh through the course of that uh you know scouring the uh the the landscape for uh an approach that overcomes those shortcomings, uh I was introduced to Immusoft and uh you know pretty immediately stricken by the uh the elegance of the approach and how it how it overcomes some of those shortcomings.

Ben Comer:

So you agreed to join um how did you get up to speed uh you know after initially joining uh Immusoft as the new CEO. You joined as CEO, right?

Sean Ainsworth:

I did. I did, yes. So uh it was a trial by fire. My first day on the job was uh the day before JP Morgan, uh 2018. So uh I actually had done uh several months of diligence before I before I joined the company uh and led some you know initial uh investment uh in the company as well. Uh and um uh you know nonetheless, the the founding CEO uh Matt Schultz, uh I I took his place. Uh, but one of the conditions was that he stayed with the company for a few months as uh uh to you know kind of help me help onboard me. So he he uh very graciously uh abided, and that was really key to uh to to getting my arms around what is uh you know very complex uh uh but exciting modality here.

Ben Comer:

Yeah, well, let's talk a little bit about um engineered B cells. Uh why is a uh redosing capability? And you know, we talked about freedom from immune suppression, myeloablation. Why is that so important uh for patients? I mean, you mentioned you mentioned it, I will say, you know, with the myeloablation, ending up in the hospital and even death in some cases. Um, so that's that's an obvious one. Um, but maybe, you know, what could you say about the the avoiding the immune suppression and and what that means?

Sean Ainsworth:

So there's actually uh uh quite a bit of morbidity associated with the immune suppression as well. Uh it uh you know tends to not get much attention, but uh it's certainly important. And uh I guess uh again, unfortunately, there have been headlines of you know patients uh dying in some of these uh AAV clinical trials uh over the last uh you know year plus. Um and so being able to avoid uh those immunogenicity issues, I think is really uh important. And uh and so I again as I was scouring the landscape, I was I was looking for that. And so the tie-in with redosing is um because we don't have that uh you know those concerns, uh, it does give us the opportunity to to go back and redose patients. Um and I I think you know, if we contemplate a use case, in particular, let's think uh, you know, pediatric patients, uh, as they grow, uh to be able to redose and and have your your therapeutic grow with them, uh I think is is very important. Um and that's not something that is currently available with with AAV approaches. Uh and yeah, why can't you redose those? Immunogenicity. So is you know, once you've done that initial dose, your uh that patient has a pretty uh you know strong immune response to that vector. And so to go back and redose, I'll say with the asterisk, redose given current uh technologies. We we we haven't done that yet uh safely. And so um uh you know, to be able to ensure that uh uh that you can redose, uh, I think that's a big uh win for you know for us and and for the patients, for the community.

Ben Comer:

Right. Right. And uh we're talking about um engineered B cells. I mentioned in the intro, these are autologous. So you're taking cells from the patient, engineering them, and administering them back to the same patient, which is I think how you get around having to suppress the immune system. It's the patient's own cells. Can you just describe maybe at a high level, Sean, you know, what that process looks like and and you know, maybe why it hasn't been done before.

Sean Ainsworth:

Sure, yeah. So uh the process, uh we start with uh an aphoresis, which is it's not a blood draw, but uh you you take the patient's uh the the immune cells effectively from uh the patient's blood and you replace the plasma. And um and so that's our starting material. Uh and from that we isolate out uh those those B cells that are ultimately uh this you know the the next uh point in the starting uh materials. So uh in uh culture, we we introduce our gene of interest. Uh we use a transposon system, uh sleeping beauty transposon, and uh that's very uh effective with uh with B cells. Uh we've found it to be um better than uh you know most virus approaches. Uh and and importantly, I'll add that uh you know it's a fraction of the cost. So um I'm getting off uh topic a little bit to to scalability, but I I think that's a big uh important part of the puzzle that uh you know we've we've prospectively addressed as well. Uh but um so getting back to the you know the approach, we we then differentiate the uh the B cells, we expand them, uh, and and so we differentiate toward that plasma cell state. And the plasma cell, we we sometimes use that interchangeably with B cells, but it's an in-stage differentiated B cell, and it's really this powerhouse biofactory of the immune system. So uh any given plasma cell is producing and releasing thousands of antibodies per second into the circulation, and we harness that ability of the plasma cells, uh, but we direct them when we modify them genetically, uh, we direct them to produce therapeutic proteins of of choice. Uh and so that gives us really uh, you know, we think a broad opportunity to address a multitude of different therapeutic indications. So um we're just getting started, you know, seeing great results so far in the clinic. And and so this is this is very exciting.

Ben Comer:

Uh you mentioned uh scalability as a kind of attribute for this process. And I'm curious. Well, I have two questions. One is what is the the time period from when the cells are are extracted from the patient and then delivered back in? And then second, is that done on site, you know, at uh at a clinic, or are the cells mailed and then sent back? How does that work?

Sean Ainsworth:

So yeah, the patient experience, they come in for that aphoresis, um, they go home, we take that aphoresis product. Our culture system is is one week. Uh, and so another factor in scalability. The the shorter the better. There's a lot of uh person hours that go into the culture, you know, the time in culture. So one week is a uh is a nice short time frame. But uh uh from there we we do uh stability, pardon me, sterility uh studies on the on the drug product. And uh we we actually do have about three weeks of sterility testing. Uh and then we ship that back to the patient and uh or pardon me, back to the hospital. And that's uh where the patient had that aphoresis done. So the patient then comes back in and gets the infusion. Uh the infusion takes an hour, which you know is uh for our lead indication, that's actually uh surprisingly short for these patients because they're used to coming in, you know, four hours every week. Uh and so one hour uh you know for a uh a protein uh biofactory that that might be delivering that therapeutic for you know years, that's that's a big uh um eye-opener for these patients.

Ben Comer:

Yeah, and you're you're referencing enzyme replacement therapy for MPS1, which is your your lead indication, your lead candidate is ISP001, uh a rare uh and progressive, I think most of the time, inherited genetic disorder. What can you say, Sean, about the choice to pursue MPS1 as your lead development program?

Sean Ainsworth:

So we actually um, you know, uh put a ton of thought into that. So appreciate the question. We started with north of you know 100 indications where we thought we might have uh application with the B cell approach. And so, you know, the initial cutoff is can it be, is it a protein therapeutic that can be delivered in trans? Uh and that's what kind of get got us to you know 100 indications. Uh from there, we looked at 11 different uh aspects from uh clinical trial complexity, patient uh population, uh proof of concept, whether there's already human proof of concept for the you know, for the existing um that existing protein that we're ultimately looking to deliver intellectual property. So we rank ordered uh across 11 different uh um uh features and and ultimately MPS1 rose to the top. Uh I left out unmet need. There's there's actually still a pretty significant unmet need. And uh, you know, despite uh a current enzyme replacement therapy that's that's that's on the market.

Ben Comer:

Right. And you talked about you know the the patients having to do multiple hours uh uh of administration for um an ERT. Um I m MPS1 is a um lysosomal storage disorder, which was you know an kind of an early focus area for for biologic companies like Biomarin. I I know was a kind of early early leader in that space creating these uh ERTs um aside from you know just the the kind of sheer administration convenience associated with engineered B cells. Um, are there other ways? that this approach could improve upon uh ERTs?

Sean Ainsworth:

Uh we we do believe, yes indeed, that uh we can ultimately um let me rephrase that, that we may ultimately provide benefit above and beyond what uh what ERT can deliver. Uh and so the in the case of uh you know the current standard of care uh it has a plasma half-life on the order of a couple of hours and so uh you know that's part of the reason for the the need for weekly four-hour infusions is to try to keep that enzyme level up. Uh in our case we're delivering 24-7 and we ameliorate or eliminate uh that that sawtooth effect uh by sawtooth effect that pike you know that spike and trough that uh is observed with uh uh pulsatile infusions so um the expectation is that uh you know 24-7 delivery of that therapeutic protein uh may ultimately uh yield better outcomes in in these patients so got it got it and so how um what would you anticipate being the kind of dosing schedule for this product in MPS1 come I guess compared to the weekly four-hour administration that's more or less a standard of care now I think yes um so we're uh we're actually exploring uh an annual dosing regimen annual okay yeah yeah so we think that would be a you know obviously a huge improvement over uh over weekly uh infusions uh we did redose our our first patient uh and that was after 18 months time so he received uh the lowest dose uh that we you know expect to give in this trial um saw some uh very uh I I'll go ahead and say very compelling uh signs of of of efficacy um including a a 240 meter improvement in uh in his six minute walk test uh that was at a year um he saw pharmacodynamic improvements as well as patient reported outcomes importantly uh you know very significant reductions in pain so it's just one patient uh but uh you know I I think that uh having three uh you know from the uh the the the pharmacodynamics the functional outcomes the patient reported outcomes all uh pointing in the you know in the same direction is is super exciting. We have left that patient on standard of care. So uh that was part of our agreement with the FDA is that we'd start out by uh dosing with ISP001 but keeping the patient on standard of care. Uh we removed that uh we referred to it as an enzyme holiday. So he had an enzyme holiday for about a month. Uh we saw those functional improvements continue forward which was uh which was really exciting. Um some of the biomarkers showed uh changes uh which one would expect when you know removing standard of care uh but we we thought that uh you know it would be important to to go back and redose that patient and uh with the objective of ultimately taking him off of of standard of care.

Ben Comer:

Right. Right.

Sean Ainsworth:

Um just looking through your pipeline uh you've got several assets that are in the discovery phase I think right now that target target um larger therapeutic areas in patient populations uh areas like Parkinson's disease uh obesity and diabetes oncology uh autoimmune diseases can you talk a little bit Sean about um Immusoft's clinical strategy and kind of how you're thinking about progressing other assets in into clinical studies you know uh as a kind of second wave to your lead development program in MPS1 so yes yeah we um we we actually uh took a a two-part approach initially uh toward uh a platform uh consideration uh and so what I mean by that is um our next program in line is is MPS2 and and so the strategy there is uh you know with some safety and preliminary uh signs of efficacy uh we went back to the agency and uh uh we requested uh a waiver for definitive talk studies for the MPS2 program. Uh and so they were amenable to that. And so we we think that's a you know a big win. We don't have to kind of reinvent the wheel every time and and do you know these sprawling and expensive toxicology studies which uh you know means ultimately we think we can accelerate uh you know the next program and the next one after that to to the clinic which uh uh will yield um the the you know the best outcomes more more broadly so uh I mentioned our kind of pipeline prioritization strategy uh we actually had a lot of lysosomal storage diseases that uh popped up on our radar and uh in no small part because you know there are existing programs on the market right that are delivered you know that have that same um call it issue with uh you know with with um durability in in terms of the you know the half-life and so the requirement for frequent redosing. Uh moreover they're genetically defined we know exactly what you know protein to deliver and why um and and so the uh you know the plan has been to start there uh build that clinical experience uh and then move from there into um I think of them as frankly higher risk but higher reward opportunities and CNS autoimmune oncology etc yeah and I mean I think there I think I I've read that there's somewhere between 50 or or maybe 70 lysosomal disorder uh lysosomal storage disorders you know what makes you want to not just pursue those you know got you got you got plenty to choose from in in that in that kind of defined category but it you're you're more ambitious you're looking at some of these really broad and large uh disease areas with uh with huge unmet need um what what would you say about that so yes uh we we actually uh it sounds like you were in the boardroom with us because we had a lot of conversations on on that uh on that very topic uh and we still do um but uh what we ultimately decided is uh you know it makes a lot of there there's so much uh application across this platform that uh you know we would be doing ourselves and probably you know so many others uh a disservice by not looking to advance in you know some of these other more ambitious uh therapeutic areas um so what we're actually working towards is uh you know establishing some early POC in in some of those higher risk high reward uh indications and and looking to partner on those uh yes yeah while uh you know continuing forward with the uh the lysosomal storage approaches which you know generally those are orphan indications right um you know many of them a billion plus uh opportunities um and uh but uh continuing forward with those uh you know under the auspices of of Immusoft and um kind of bringing uh you know ultimately uh with the uh the ambition of kind of bringing it all forward and having partners for many of those programs but uh you know working towards uh the clinic with uh several other lysosomal disease approaches and with uh with your mps one program which is your lead and then your your follow-on project uh or program in mps two um are those being developed completely in parallel do you do you wait until mps one reaches a certain you know clinical milestone before then progressing MPS2 how do you think about that managing that so uh we are um you know we've had a lot of dialogue with the agency on MPS2 uh we think that uh you know we've got just a couple of other blocking and tackling items to uh to be able to move that program forward into the clinic um you know right now we are focused on it on MPS1. Uh it's it's in the clinic we want to continue forward with that uh um and uh as we go out and uh you know secure more resources we'll uh look to advance mps two into the clinic got it i i want to follow up on some of your comments about your uh engagement with the FDA and and kind of get a sense of that but before I do Sean um you're you're CEO and chairman of the board at Immusoft uh you're also a board member at Ray Therapeutics uh and at Opus Genetics what what could you say about um the most important role that a board member can play at a company wow that's a that's a a very broad question uh uh you know the uh I think do no harm is uh you know one of the one of the most important things which seems obvious but uh isn't isn't isn't always so much in practice but uh I think uh you know the as a board member uh you've got a fiduciary responsibility to the shareholders which means um you know you've got to work closely with management to uh understand the direction of the company the strategy and uh when and where um you know the the decisions to uh deploy resources come come into play and uh ensure that you know that that's all um um well uh you know well well designed and strategically thought thought through uh and then above and beyond that uh you know bringing resources to the table uh be they you know uh potential investors strategic partners uh new employees um uh advisors etc I think those are all uh important parts of uh being a you know a good uh a good board member

Ben Comer:

Are there any downsides to being both the CEO and the chairman of the board uh at the same company and I ask because you know I've I've had guests talk about you know difficult conversations with you know the CEO between the CEO and and the board where there's disagreement um you're you're CEO and chairman uh so you know you know you're probably not disagreeing with yourself a whole lot but you know what would you say about um you know being both serving on both and are there are there new challenges associated with that?

Sean Ainsworth:

so at the end of the day uh you know whether you're board chair or not you're your vote counts uh you know the same as uh as any other board member so um as CEO uh you know I'm I'm wearing the hat of the the operator uh by and large uh you know coming into the board meetings and um so uh you know it's it's always important to me to coordinate with the board uh before ever getting into the meetings and and um so there's you know there's there's very uh few instances where you know something really uh comes up at the board meeting that's uh that's a surprise to anybody.

Ben Comer:

Right. Okay. Um let's uh let's get back to FDA. Um the current administration in FDA seems uh fairly engaged with gene and cell therapy particularly in rare diseases you know we've seen just as an example the recent idea of a plausible mechanism pathway floated for personalized therapies that would potentially remove the requirement for randomized uh controlled trials in some instances um ISP001 your lead candidate received fast track designation uh from the FDA I think in October um what more could you say uh I guess about your recent interactions uh with the FDA um what what what other color uh could you add there?

Sean Ainsworth:

Thanks so I I uh you know I think that uh the agency is actually doing some uh you know very innovative uh taking some very innovative steps towards removing some of the barriers for for cell and gene therapies um the you know the the uh the the new approach that you mentioned uh that's uh in you know I think really an offshoot from the uh the baby kj uh right exactly uh dosing which you know was was really extraordinary and just uh uh such an amazing uh collaborative effort and and and story behind that so uh you know for the FDA to take a you know close look at that and start to build in mechanisms to enable more of those approaches to move forward I think makes makes a whole lot of sense and you know if you dig in there's there I I mentioned uh you know platform consideration platform designation uh I think a lot of that actually plays into the you know the the the the baby kj story as well that if you've got a platform that's um you know that's working uh from a delivery perspective and you're targeting a a known gene um to be able to uh you know to to make a one off or you know small and off and take those into a clinical setting uh early uh I think that's uh just a huge win for you know for everybody um and beyond that you know the agency in September came out with guidance for uh for orphan diseases as well that uh you know provide a pathway for uh single arm clinical studies um so I I think that a lot of that is um innovative helpful and uh you know for uh for orphan cell and gene therapies in particular those are those are nice um uh you know alignments uh from a regulatory perspective

Ben Comer:

are there have you noticed any changes in style or strategy or or even performance uh good or bad uh maybe thinking back to your time engaging with the agency uh at Retro Sense kind of up through Immusoft uh how has the agency evolved in your in your view?

Sean Ainsworth:

uh so I'll I'll I'll avoid any uh political uh commentary because that's uh you know there's there's obviously been some changes uh uh that are associated uh with the political changes but uh uh you know I I guess what I'd say is going going way back to the early retro sense days uh evolved is is is the right word that uh the agency has evolved uh with the science and and I think that's been uh really important um you know we're we're doing things now with uh uh so many different gene editing gene therapy uh cell therapy modalities car T you know you name it uh I think a lot of the old frameworks just would never be able to keep up and so um there there has been a a fairly consistent evolution and uh you know these last two changes that I I mentioned um they seem to be uh both you know steps in the right direction towards towards keeping up with that evolution

Ben Comer:

yeah and you'll I I think be you engage with with CBER for your products Richard Pazdur now is going to be leading leading CBER.s uh I I believe um is that correct you'll be working with his group?

Sean Ainsworth:

uh my understanding is he's leading CDER.

Ben Comer:

You're right you're right CDER yeah that's that's correct yeah okay Dr.

Sean Ainsworth:

Prasad is uh CBER.

Ben Comer:

Yeah thank you for that for that correction. Um is there anything else uh Sean that you would say kind of uh looking forward in terms of and maybe even not specific to the FDA I don't know how much you're thinking about uh global markets although you probably are given you know your background and and work and kind of uh cross-border corporate development but um is there anything else you would say about the regulatory outlook for cell and gene therapy um next year is there anything you'd like to see that that you're not seeing yet

Sean Ainsworth:

So yeah appreciate the question the um the the there's still a um boy what am I what am I thinking of the uh the rare pediatric uh disease voucher yeah uh that's a program that uh you know I'm hopeful gets re-implemented soon uh I think there's a pretty uh strong sentiment that uh that that will be re-implemented but uh you know it can't be it can't come soon enough uh it's something that is just so important to those of us working in pediatric orphan diseases um and so you know to give a little color for those who may not uh be familiar uh that that voucher program uh upon approval of your rare pediatric indication is a voucher that uh allows for accelerated approval of uh another program and those can be sold on the open market um you know the last few that I'm aware of have been uh purchased for on the order you know of 150 million dollars uh and so for these again rare pediatric diseases uh you know that's been a huge part of the uh of the decision making process and it's a it's a big part of uh you know the value proposition um so I you know I actually sat in on the ENC committee hearing a couple of months ago I got voted 24 nothing to you know for uh to advance um bipartisan support uh I think there's general consensus that that's going to come through and uh you know hopefully uh hopefully soon.

Ben Comer:

Yeah that's a really important incentive I'm glad you mentioned it I think it ex I think that program expired I want to say like last September. Has it been that that long that it's that it kind of sunset and has not yet been reinstated. I know there are a lot of folks out there uh that that want that incentive to come back. Um yeah and you know you mentioned you can use the priority review voucher the the going rate is somewhere north of a hundred million dollars uh recently and it's kind of been been that way for a little while but that that voucher can be used for any development program that priority review it doesn't have to be you know for the for the purchaser uh of the voucher program they can use it you know for for any development program which becomes quite important you know if you're developing a a product and you have some close competitors and you know are kind of trying to be first to market um it's uh it's a valuable incentive and a valuable tool so I think you know I'm I'm with you in hoping that that that one comes back.

Sean Ainsworth:

We're getting to the end of our time Sean I I wanted to just end with you know kind of your top priorities uh you know what's next for MUSoft what you're most focused on right now so we're still uh you know driving forward with our our lead program uh clinically so MPS1 uh you know we we did have a recent uh meeting with the FDA and uh that was important in gaining alignment on what that pivotal trial looks like uh without getting into the weeds uh you know we've we we do have alignment uh on uh on the uh size of the study the pediatric population the endpoints uh and so those are you know super important things for uh for advancing the uh the program from here um and uh you know in the meantime we we keep our uh you know nose to the grindstone we got a really great group of scientists that are continuing to produce uh amazing pardon me amazing preclinical data and so we're uh you know we're always working to advance those those follow-on programs as well yeah and given that you're working in pediatrics uh would you be uh you know assuming that that uh priority review uh program voucher program is reinstated would you potentially be eligible for that with any of your clinical programs so mps one um absolutely yeah thank you for asking so we've actually got uh rare pediatric disease designation already okay and so that uh is what enables you upon approval uh to get uh to get uh to get that voucher uh mps two uh we believe would also be uh uh available and uh or eligible pardon me so uh that's uh across uh you know a number of lysosomal diseases they're they're almost because they're inborn right they're almost all pediatric

Ben Comer:

yeah that'd be pretty nice I think to probably get somewhere over a hundred million dollars no strings attached to to bring back into the the company and um and maybe tackle some of those even more risky assets that you have uh in discovery right now

Sean Ainsworth:

right right definitely fuel more innovation

Ben Comer:

absolutely well uh Sean thank you so much for coming on the show it's a real pleasure speaking with you

Sean Ainsworth:

likewise my pleasure thanks for having me on the show Ben

Ben Comer:

We've been speaking with Sean Ainsworth a CEO and chairman at Immusoft. 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 Science Leader.com We'll see you next week and thanks as always for listening.

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