Good afternoon. My name's Jess Fye. I'm a biotech analyst at JPMorgan, and we are continuing the 43rd Annual Healthcare Conference today with Insmed. We're going to start out with a presentation from the company and then go into Q&A. If you have a question in the room, you can raise your hand. Someone will bring you a microphone, or you can send me questions on the iPad. But with that, let me introduce the company's CEO, Will Lewis.
Thanks, Jess. Before I begin, I want to draw your attention to our forward-looking statement slide. Any comments made today should be placed in context of these filings. Last year, we transformed Insmed, and we began a new chapter that we believe will represent our strongest period of growth ever. Looking back in 2024, we delivered an impressive commercial performance in our sixth year after launch. We validated our next two compounds with important clinical data, and we strengthened our balance sheet so that we may accelerate into 2025, where we anticipate launching a blockbuster drug even as we produce further clinical data for multiple additional compounds and indications. Now, it's rare, but when those things combine in biotech, the opportunity can be staggering, particularly when you're talking about the introduction of a novel mechanism of action. Consider companies like Vertex or Argenx, even Eli Lilly.
With just one novel mechanism of action, each of these companies transformed themselves into one of the great biotechnology or pharmaceutical stories of our day. In 2024, Insmed began that journey with the successful phase III Aspen trial. Our DPP-1 inhibitor unlocked this class for impact and action against bronchiectasis. We are exploring its use in two other diseases, and we will have data this year for one of them. Such opportunities in biotechnology are extremely rare. But as all of these precedents prove, the initial clinical data is just the very beginning of the story of value creation. And so we believe 2025 marks the beginning of this next phase of exciting potential growth for Insmed. Let's explore why. First of all, as we enter 2025, we're looking at the execution of the largest commercial launch in our company's history.
We have announced that we expect continued double-digit growth in Arikayce for refractory MAC in its seventh year of launch. We have late-stage clinical data readouts that will catalyze the next wave of growth, and we've expanded the clinical breadth of our offerings with the start of our first gene therapy program in Duchenne muscular dystrophy, which recently cleared IND. All of this is backed by more than $1.4 billion of cash on our balance sheet, and for those that were not following us closely, that balance equates to what we had at the end of the Q3 , thanks to some renegotiation of some debt and the addition of $150 million of additional capital. As we look across our portfolio, one thing is true for every compound we bring forward.
We are looking to bring forward a first-in-class or potentially best-in-class asset across every stage of development in every disease we target. Here you see outlined what we often refer to as the four pillars of the company: Arikayce, brensocatib, TPIP, and what was historically our research group, which has now been broken out to include its own clinical program, gene therapy for the treatment of Duchenne muscular dystrophy. I'll highlight that right behind that, we have several others that will be entering the clinic this year. What this means for us is that since 2018, our company has been targeting roughly 30,000 addressable patients worldwide in the three areas where we have commercial infrastructure, which are the US, Europe, and Japan. During that time, we've managed to produce very good commercial results for Arikayce refractory MAC addressing this target population.
What is about to unfold at our company is that this addressable population, should all of our clinical trials and approvals go as expected, will grow 80-fold so that by the end of 2030, we anticipate addressing a patient population in excess of two and a half million patients across three distinct programs. The drivers for value creation in 2025 and beyond are going to come from both revenue generation and clinical trial execution. What we can see here is that under the revenue generation side, we expect to see continued double-digit growth from Arikayce and refractory MAC lung disease. We anticipate the launch of brensocatib in bronchiectasis in the Q3 of this year, assuming regulatory approval follows under an accelerated pathway, and as we enter into 2026, we anticipate the launch of brensocatib for bronchiectasis in Japan and Europe.
In parallel, we'll be producing clinical data for TPIP, our PAH drug, in the middle of this year. We anticipate initiating our phase III trial for PH-ILD. We will have clinical data from our phase II trial in chronic rhinosinusitis without nasal polyps before the end of the year, and we anticipate the first data from our gene therapy program in the beginning of 2026. On top of that, we will have the second phase III study for the expansion of our Arikayce program into frontline MAC, so-called ENCORE study, in the Q1 of 2026 as well, so this is going to be a very busy year building off the momentum that we created last year. Let's turn our attention to the first of our products, Arikayce, which is amikacin liposomally encapsulated in a suspension that is administered by an ultrasonic nebulizer.
This is approved for the treatment of refractory Mycobacterium avium complex lung disease conditionally in the U.S. and fully approved in Europe and Japan. We were very excited to come in above the high end of our range last year at $364 million. The range was $340-$360. and when you consider across the three regions where we have commercial operations, you can see that the contribution was uniform across all three: double-digit growth, strong performance out of Japan and Europe in particular, and continued double-digit growth out of the U.S. This is what has given us the confidence to indicate that this year's range is $405-$425 million, which, once again, for the seventh year after launch, is a double-digit growth opportunity. but this is just the beginning.
When the ENCORE data reads out next year, we're going to move from this top line of refractory MAC patients indicated on this slide, broken out by region, into the line below for all MAC lung disease. To put a finer point on this, in the U.S., we currently target roughly 15,000 addressable patients. That is what has allowed us to produce revenue that we've just reported. If we look at where that goes with the success of the ENCORE trial, that should take our addressable market from roughly 15,000 patients to roughly 100,000 patients. Given the revenue we've generated to date, you can understand the substantial opportunity this represents, and there are parallel expansion opportunities in Europe and Japan. Our second product is the DPP-1 inhibitor, brensocatib. This is currently targeting three separate diseases: non-cystic fibrosis, bronchiectasis, chronic rhinosinusitis without nasal polyps, and hidradenitis suppurativa.
If we look at just the first indication, bronchiectasis, this is an opportunity that we think represents more than a million patients at launch using the commercial infrastructure that is already in existence in the U.S., Europe, and Japan. As we think about broadening that opportunity to those who are undiagnosed with bronchiectasis or those who are comorbid with COPD and bronchiectasis, these numbers creep comfortably above a million patients in the U.S. alone. We are getting ready for this launch, which we expect to be in the Q3 of this year, pursuant to what we described as our accelerated approval pathway, which we anticipate because we have breakthrough therapy designation. So when the FDA provides that breakthrough therapy designation, in about 89% of the cases, that accelerated approval is granted. So we feel pretty good about securing that.
We had very clean data last year where the side effect profile was comparable to placebo, and the efficacy was statistically significant across both primary and secondary endpoints. This has initiated our dialogue with doctors, patients, and payers, all of which have been incredibly favorable. I cannot emphasize enough for a blockbuster opportunity to have this kind of backdrop as you are beginning the launch. It doesn't get much better. 90% of doctors are likely to prescribe brensocatib for patients that meet the entry criteria of our phase III study. We've had almost a million unique visits to our disease state website, and if we look at payers, we've already spoken with over 90% of Medicare and commercial lives covered by different accounts. Collectively, this gives us the confidence that we are primed for a very strong launch in brensocatib treating bronchiectasis, but this is just the beginning.
That opportunity, which I just outlined, we have said publicly represents more than $5 billion of net sales, will be just the start. Chronic rhinosinusitis without nasal polyps represents an additional potential large patient opportunity, and we are currently 70% enrolled in our phase II study to demonstrate the efficacy of brensocatib in this population. It's important to note there is only generic inhaled steroids available for the treatment of this condition. And when we look at the prevalence in the US, Europe, and Japan, this numbers well in excess of 50 million patients. We will be targeting the narrow end of that, the most severe patients, those who are either eligible for surgery or who are steroid non-responders. In other words, they're taking the available steroid, but it's not doing enough for them.
These first two rows indicated on this slide are the entry criteria patients that are included in our phase II Birch trial that we'll read out at the end of this year. Simply put, this population added to our earlier population for bronchiectasis would add an opportunity that is as big, if not bigger, than bronchiectasis, and we'll have that data by the end of this year. We do not think that there would be a pricing adjustment of any kind needed to add this population to our opportunities. Beyond that, we are also targeting hidradenitis suppurativa. This is the third indication. That trial just kicked off. It's in its early days, but we're excited to add the patients, potentially if this is successful, to that broader group that I just described.
The one thing I'll say about this indication is that the opportunity here is very substantial, but we're going to take a look after the first 100 patients have cleared this trial's primary endpoint, put that data in the hands of an expert panel. If that expert panel is able to look at the efficacy data and safety data and see that there is an opportunity, then they will advise us to continue. If not, we'll simply shut the trial down. We want to bring drugs where we know they're going to have impact, and that's why we're going to have this panel take this early look. All three of these are incredibly substantial opportunities, and they unlock what I referred to at the opening as one of these new mechanisms of action that can apply to a broad range of disease states.
Our third drug is TPIP or treprostinil palmitil inhalation powder. This is treprostinil with a 16-carbon chain appended to it by an ester bond. In dry powder form, it's breathed into the lung with a single inhalation. The bond is cleaved by esterases in the lung, releasing the active moiety, and what we get as a result of that is a lower peak and a longer trough. The consequence of that is 24 hours of coverage for the first time for patients with PHILD or PAH. That's not available today, and the ability to bring that forward, we think, is going to position this drug very competitively against every other prostacyclin out there. When we look at what that means, it means convenience because you're talking about a dry powder inhaled once daily. We think the safety and efficacy will be superior.
Because of that formulation, you're breathing in an inert drug, so the cough reflex is not as intense. And the consequence is you're able to get much more drug to these patients. The goal of prostacyclin therapy is to max out the available drug you can get to the patients because what you're accomplishing with that is vasodilation, and you want that to be sustained over an extended period of time. This drug represents the potential to do that for 24 hours on just one inhalation. We'll be putting out this data earlier than expected. The acceleration of the enrollment through the end of last year, once people began to see and take note of our PH-ILD phase II data that came out last year, means that we can now expect this data in the middle of the year.
The primary endpoint for this is change from baseline and pulmonary vascular resistance at 16 weeks. We're extremely excited about this program, and we're looking forward to the data readout sometime around the middle of this year. This population is well understood because there are many drugs approved to treat PAH and PH-ILD. What we're after here is producing the best-in-class prostacyclin. And when we look at who we're going to compare ourselves to, absolutely we'll compare ourselves to treprostinil in all its various forms, and we expect to be superior from a safety and an efficacy point of view in respect of those other compounds. But we would even challenge ourselves to look at us in comparison to the product sotatercept because we think we can achieve pulmonary vascular resistance levels that approach, potentially even match what that has been able to accomplish.
For those who are not familiar with that product, after phase II, when it produced a pulmonary vascular resistance of 33.9%, it was acquired by Merck for slightly over $10 billion. This is a very, we call this the sleeper in the company, this product, because it doesn't get a lot of credit, but I think that's going to change in the middle of the year. Finally, I want to mention what we used to call our fourth pillar. This is really a series of platforms that we acquired over the last three or four years that include gene therapy, de-immunized protein engineered using artificial intelligence, technology like RNA end joining that allows the gene therapy capsids to be combined intracellularly to create longer transgenes and thus longer-length proteins, and also the novel approach of synthetic rescue.
Each of these is represented by a different geography in our company: synthetic rescue based out of Cambridge, England, de-immunized protein engineering out of New Hampshire, gene therapy based out of San Diego, and along with RNA end joining, and then our next generation of DPP-1 inhibitors and related compounds coming out of New Jersey, where our headquarters is located. The goal here is to produce compounds on the order of one to two new INDs a year, and we are well on track to do that, starting with the DMD program this year, to be followed by ALS and Stargardt either this year or early in 2026. Behind all of this is the single most important thing we have at Insmed, and that is our culture. We've been recognized for this for many years now. This is something we're extremely proud of.
We assemble extremely capable people, and we empower them to do their best work, and while that may sound like a fairly straightforward exercise, to get people to participate in that and celebrate it is really an accomplishment, and the consequence of that has been the fact that we're three for three. Our first three programs are all working. They've either been successfully approved, will shortly be so, or have been validated and de-risked by mid to late-stage clinical data. That's what gives us such conviction that we're able to produce meaningful results and gives us the courage to put out a forecast where the first three programs, assuming they hit what we have outlined, would represent in excess of $8 billion in peak sales.
This is a very substantial change for a company that has currently just produced $363 million in revenue, but it is absolutely within our grasp, and it is all down to execution. So as we look forward to this next era of growth for Insmed, we will start with the PAH top-line data I mentioned in the middle of the year. We'll launch Brensocatib, assuming it's approved as expected, in the Q3 of this year. We will have CRS without nasal polyps top-line data in the Q4 of this year. We will have initial clinical data from the DMD gene therapy program either at the end of this year or beginning of next year, and we will complement that with our Arikayce Encore top-line data.
Backed by the culture I just described and the strong balance sheet, we are at that inflection point that is going to make Insmed, we believe, one of the next great biotechnology companies in our ecosystem. Thank you very much.
Great. Thank you for the presentation. I guess starting with brensocatib heading into that launch in the Q3 , is there anything that you really, really want to make sure is on the label or anything that is kind of like on the bubble or that you're kind of pushing for to get on the label?
Well, so I would say just to remind everyone, the clinical data was extremely strong and consistent between phase II and phase III. And we did see some positive surprises in phase III. I think the first of those, which is not to be overlooked, is the safety.
The profile of both the 10 and 25 milligram dose were almost perfectly comparable to placebo, which is obviously extremely rare and puts us in a strong position as far as inclination to move this product forward and for its use broadly in these patient populations. When we look at some of the primary and secondary endpoints, we showed statistically significant improvement in probability of having an exacerbation, probability of having no exacerbations, time to first exacerbation, all of which is the goal of treatment with any therapy in this class. We also showed preservation of lung function at the 25 milligram dose, and one way or another, I think it's critically important to get that piece of information into the mix because that is certainly capturing the attention of physicians and the market access world.
Most patients lose about 33 milliliters of lung function a year, and this showed a preservation of that at a statistically significant level at the 25 milligram dose, so I'd love to have that a part of the storyline, how that gets in and what that looks like, we'll see. I would say that our dialogue with FDA has been extremely productive so far. We filed in December of this year. We will update everyone on the Day 74 Letter when that's received. Obviously, that'll be in the Q1 at some point, and once we have that, we'll know what the profile is going to look like, but I actually will just finally comment that I don't think the label is going to be our point of resistance in the treatment paradigm. It's going to be the agreement that we reach with market access.
So for example, we don't think we're going to be restricted to two or more exacerbations in the label. If we are, that's fine, but we don't think that's the case. We think it'll be indicated for the treatment of bronchiectasis. We think the market access world will likely limit to use in patients who have documented two or more exacerbations. So for us, the way to navigate this is to get the broadest label we can, get to a reasonable agreement with the market access world so patients who will benefit from this drug can gain access to it.
And the way to do that is to highlight that we're comfortable going with two or more exacerbations as an entry criteria for treatment and just ensure that patients and physicians are as sensitive to documenting those things as they possibly can be and that physicians are able to simply attest to the existence of those two exacerbations versus having to go back and document that in writing.
Got it. Can your kind of reps in the field talk to pulmonologists or your target physicians about kind of like not about the drug, not marketing it, but you talk about documenting bronchiectatic attacks?
So there's a number of different ways we reach out to the community. And I would say that the first point was we're believers in forward funding the resources needed to ensure that that dialogue happens at all levels.
That includes MSLs, that includes therapeutic specialists, it includes market access. So all of those things have been resourced heavily by the company prior to and then subsequent to the data that came out last year. As we think about what that looks like, for our sales force, I think we had roughly 70 sales reps or therapeutic specialists. We added another 120 to their ranks. They were up, fully trained, and deployed as of October 1st last year. So they're going to have nine months to detail for refractory MAC lung disease and also raise disease state awareness with those physicians, build those relationships so that the day that we're approved, we can turn around and provide drug to the patients that will benefit. That resource is complemented by similar efforts in the medical science liaison field and market access world.
And bringing all three of those in parallel over the course of the last year plus has been our focus.
Okay. Not expecting you to unveil price kind of on stage right now, but what are the things that you think about as you kind of get closer to actually having to set price?
So we're feeling very good about our discussions and our analysis in regards to price. And I think what we put out for everyone to understand is a range of $40,000-$96,000 as a range. That's bookended by what effectively is a specialty drug in asthma on the low end and the initial pricing for idiopathic pulmonary fibrosis drugs at the high end. What we've indicated and what everyone who's done their own research has come back to us and said is this should be in the upper end of that range.
And while we haven't made any pricing decisions, certainly this is a value for money argument in the sense that we are providing the needed benefit to these patients and the price can support that kind of benefit that physicians are looking for. So we don't really expect to have a lot of resistance on this front, but we're in dialogue right now to sort of wrestle that one to the ground.
Okay. You mentioned, I think it was like the last quarterly call, some analogs for us to think about when we think about kind of the shape of the initial launch curve for brensocatib. And you picked some pretty ambitious ones, if I recall. Dupixent was one of the ones you named. So how do you—what are the parallels that you see, or how did you land on those analogs?
To put it very bluntly, we want to have a first-in-class, best-in-class launch. We want this to set the standard in the industry, and that's an ambitious target. But I'll just remind everybody, when we were launching Arikayce and refractory MAC, I think the Wall Street estimates were between $40 million and $60 million in our first year, and we did over $130 million. So we did triple what the street thought when we got out of the gate. And we followed many of these same patterns. We had our therapeutic specialists deployed early. They built relationships. And when it came time to launch the drug, we were ready, and the physicians were eager to utilize the drug in appropriate patients. As we think about this, it's the same model, albeit at a much larger scale, and we have some of the very best therapeutic specialists there are.
I can tell you we put out a call for therapeutic specialist applications to fill 120 positions, and we got over 7,000 resumes. This is going to be a big launch, and this is something everybody wants to be a part of. And as a consequence, we were able to interview for culture. We were able to interview for integrity. We were able to interview for capability. And bringing all those capabilities to bear to this patient population makes us very excited for what this team is going to be able to do. I couldn't be more bullish about where I think we're going to go with this group.
What about competition? You've got BI kind of coming up from behind. There's another asset from Chiesi. How does brensocatib compare to those molecules?
So last year, we saw the data from both of them.
I think as excited as we are to see the DPP-1 mechanism validated, neither one of those portrayed itself in a way that anyone, either physician or investor or otherwise, interpreted as truly competitive. We'll see where phase III comes out. Those are phase II data sets that they produced. They've got to run the phase III trials. We can understand that that will be quite a challenging thing to do if our drug is on the market and recommended for use with strong data that is comparable to placebo. It's not entirely clear to me why you would want to test your patient on a different drug, but we welcome that. We want physicians to have alternative choices. We want patients to have effective medicines.
We're pleased that our first medicine in this class of DPP-1 inhibition is really setting the standard for others to follow, and that's what we're seeing in the BI study. Their phase II study mirrors exactly our study design in Willow, and I anticipate that their phase III study will look a lot like Aspen, but it's at least five years behind.
Got it, so there's more to brensocatib than bronchiectasis, right? So we're heading into an update from the Birch trial. How do you define good data, or what are you benchmarking off of?
Yeah, so the important thing about both bronchiectasis, where there's nothing approved to treat patients, and CRS without nasal polyps, where there is only a generic inhaled steroid to treat patients, is that these are massive markets with nothing approved by way of novel medicine to help these patients.
We set a standard for first or best-in-class therapies, and that carries through to the subsequent indications. So for CRS without nasal polyps, we expect patients on a scale of zero to nine for the symptom score to be at least a five, so they're severe enough that they're symptomatic and that is evident. We're targeting those who are eligible or who have received surgery or who have failed that steroid therapy. They have to be on a steroid and on a stable regimen of it, so we won't see variability around that. And we're looking for sort of a one-and-a-half-point improvement on that score, give or take. And we're powered well to be able to demonstrate that. I'm excited to say that I think our instincts tell us, as they did with bronchiectasis, that this drug is working. We haven't seen the data. We don't know.
But certainly, the safety profile continues to be very good. I'll also remind that we studied 10 milligrams and 25 milligrams in bronchiectasis, and in CRS, we're studying 10 milligrams and 40 milligrams. So we've got a higher dose in this patient population, and we're super excited to see what these data show at the end of the year, particularly when you consider that if that were to be something that looked like it was viable, it would effectively be doubling the addressable market in terms of revenue potential for this compound.
And what about HS? You're running a phase II there as well. How do we think about the enrollment timelines?
Yeah. So HS just started, and once we see what that enrollment pattern looks like, we'll give some guidance on the first 100 patients when they'll cross that threshold for evaluation. It raises a great point, though.
Unlocking a class of therapy, and I made reference to this in my opening remarks, is a very big deal. Vertex was built off of the back of CFTR modulation. We are building off of the back of DPP-1 inhibition, having successfully commercialized Arikayce in three major markets. That puts us in a very enviable position, but it also means that having discovered this novel mechanism, we want to own it. We want to dominate in this space. And so what we've done right after our phase II WILLOW study results is we set our medicinal chemists to work in creating additional DPP-1 candidates. And I'm proud to say we have more than 750 of them right now, with the first handful that have cleared all of the screening and are now getting ready for preclinical development. What are we going to be targeting? We're going to be targeting COPD.
We're going to be looking at rheumatoid arthritis, very substantial markets where this DPP-1 will be specifically dedicated for the treatment of these indications. And so behind the success of brensocatib, which we anticipate, will lie a whole bevy of additional DPP-1 molecules.
Great. Maybe switching to TPIP, I noticed that you're now going to get your phase II PAH data before you kick off your phase III in PH-ILD. Does the PAH data at all inform how you pursue PH-ILD, or do you already kind of have what you need to move forward into phase III and PH-ILD?
So PH-ILD, we saw phase II data last year around May, which was very encouraging. It was a small study of roughly 33 patients, randomized three-to-one treatment versus placebo. But what we saw was directional improvement in time to clinical worsening. We saw directional improvement in six-minute walk.
There were a lot of signs within that study that were extremely encouraging, and what we wanted to do before we started phase III was ensure that because the tolerability was so good and the opportunity to increase dose was going to permit us to go above what we originally thought was our max dose of 640 micrograms to 1,280 micrograms, we wanted to make sure that we could do that with just one or two capsules, so we're doing some manufacturing work to get that into single capsule strengths so that a patient would be more convenient and could just use one max two to get to that 1,280 level, so that's part of the reason why PH-ILD will start its phase III at the end of this year. The other reason is we do want to see the PAH data. I think that is probative.
It's not directly relevant in terms of those are two different disease states, but certainly, we're going to learn more from the 102 patients that are in our PAH phase II study, and we're anxious to examine that and see what it might suggest about our approach to PH-ILD as well.
Would you consider exploring TPIP in other indications?
So this is a question we get a lot in light of the upcoming and expected Either data in idiopathic pulmonary fibrosis. I think the jury is very much out on whether that will be effective there, but I will say this: any place Tyvaso is effective or any place that prostacyclin is going to have a positive impact, our drug should dominate. It really should.
It should be the best-in-class prostacyclin there is with once-a-day dosing, much more drug actually delivered to the patient with the consequent benefits that we see from the dose-response curve in those precedent compounds. So all we're really doing with TPIP is unlocking the full potential of prostacyclin therapy, getting the dose up, lowering the peak so the adverse event profile is reduced, and making it convenient for patients to benefit from this proven therapy.
Okay. We'll take one from the iPad here. So you have talked about how you've brought in external assets in the past. What are the kind of indications or modalities that you're most interested in now?
Well, I have to say this. I think many people view last year's moment when ASPEN trial worked as the kickoff or the starting gun for the company. And we've been preparing for this for a long time.
I've been at Insmed for 12 years, and when I started, there were 30 employees, and we had a zero enterprise value. So we sit here today with roughly $12 billion in market cap and 1,300 employees focused on all this range of opportunities, and we've assembled these in a very deliberate way so that this next five-year period really represents a flywheel throwing off one opportunity and innovative medicine after another. We have the infrastructure and the people to accomplish that. I don't see the need to go out and add additional assets. We are always looking. And in such a have-and-have-not biotech market like the one we find today, it is certainly a ripe time to go out and search.
But that hurdle is very high for us right now because we have so much to execute that we know will deliver value to patients and consequently our shareholders.
Okay. So the company's got a really healthy cash balance right now, but I think you've also been clear that you don't expect to get to profitability with the current cash balance, right? You've got a lot of various updates coming, right, whether it's clinical or launch or whatever. How do you think about when the right time is to kind of go back to the capital markets if that's the route you go?
Yeah. So it's an interesting trade-off because our clinical development spend is quite healthy, as you know, and that's because each of these opportunities has been validated. We have a very disciplined, stepwise evaluation about whether a program will go to the next stage of development.
It has to clear very significant hurdles, including being able to continue to be positioned as a first or best-in-class therapy. If it reaches that threshold, then it is definitely worthy of investment. And from that perspective, we'll continue to invest in that innovation because we think it's going to inherent benefit ultimately to patients and therefore our shareholders. But that process is one that we can titrate up or down as we wish. Right now, we're very comfortable with our spend profile. We have many levers we could pull to augment our balance sheet should we wish to do that.
I don't see the need for that in the near future, but I would say that with $1.4 billion of cash in the bank in such a challenging market environment and knowing we could access equity, convertible, royalty, dealmaking, lots of different options out there, it's just not something we feel pressured to pursue right now, and we're very excited to turn over a lot of these cards to prove our ability to execute both clinically and from a commercial standpoint.
Okay, so speaking of kind of one of those cards that wasn't on my radar for kind of the near future, you got the IND cleared for DMD. What's a win as you kind of take this into the first few patients? What do you want to see?
Well, of course, we always start with safety.
What we're talking about here is intrathecal delivery of gene therapy for the treatment of Duchenne Muscular Dystrophy. If you pause and reflect on that for just a moment, that's a counterintuitive approach. Intrathecal delivery for the treatment of a muscle disorder where you're also trying to transduce cells in the cardiac tissue is not apparent how that is exactly happening biologically. I can tell you, clearing the hurdle for intrathecal delivery of any therapy with the FDA is a very tall order, and it speaks volumes about the caliber of the people we have out in our San Diego operation and the hard work they've put in to be able to accomplish that.
It breezed through that process, so we're very proud of the profile we've been able to put forward and the comfort that the FDA has drawn from the safe approach we've taken to this medicine. The advantage of intrathecal delivery is you can deliver less gene therapy. You're not going through the liver, so you're not getting the immune reaction. You're also not wasting, if you will, the gene therapy product, which is extremely expensive as it is cleared by the liver. So we can represent today that the preclinical data is extremely encouraging in terms of its ability to transduce both muscle and cardiac tissue, including the diaphragm, and that is very exciting for the patient community. It's early days.
The first effort here will be, of course, to start with a low dose and then work our way up as we see that there's safety, as we expect there will be, and then to move down in age because the earlier you treat these patients with the highest tolerable dose, the best potential you have for benefit. What we want, and I will share the vision I expressed with Brian Kaspar when we first sat down to talk about this opportunity, is for some distant day to prevent these kids from going into a wheelchair, and we're looking at that through that lens. It's a very audacious goal. We start with microdystrophin. We think about expanding that to multiple RNA end-joining capsids that allow us to create longer transgenes and longer-length dystrophin, potentially someday even full-length dystrophin, although that's in the distant future. We also look at redosing.
We look at manufacturing using algae to lower the cost. Many different approaches to this therapeutic angle so that we can deliver ultimately for the patient what you would want if the patient were your own child.
Okay. Great. We'll leave it there. Thank you.
Thanks very much.