All right, everyone. Well, welcome to day two of Citi's Back to School Biopharma conference. I'm Eric Joseph, senior biotech analyst with the firm, and this session is with BioMarin, and I'm happy to be joined by Chief R&D Officer Greg Friberg and Chief Commercial Officer Cristin Hubbard. Thank you both for joining us here. Maybe just before we dig into questions, it would be good to have some high-level remarks from you both and just what some of the key priorities are at the company looking over the next six , 12 months.
Great. Thank you, and thank you so much for having us, Eric. So, just to take a step back, BioMarin, we're a leading global rare disease company, and we're really focused in and around genetically defined conditions. What we have now is nine commercial therapies across how we're organized, which is in two business units, and that includes skeletal conditions and metabolic conditions. Now, we're a highly profitable company. We have strong revenue growth, and really, we're increasing cash flow, which is fueled in large part by the patient demand that we are creating across the 80-country footprint that we work in. We recently, in our Q2 call, announced that we've got four products that are tracking to blockbuster status, which we're excited about, and VOXZOGO is in the lead.
At our full-year 2026 guide, we had said that VOXZOGO, the range would be $1 billion to $1.05 billion, so that'll be our first blockbuster product this year. What also happened this year that we talked quite a bit about in the Q2 call was the closing of the Amicus acquisition. What that did for us was it brought in two high-growth commercial products, that is GALAFOLD for Fabry, and POMBILITI and OPFOLDA, which we call PomOp for Pompe disease. What we shared in the call was that our peak potential for GALAFOLD is at $1.4 billion in the mid-2030s, and for POMBILITI and OPFOLDA, that's $1.2 billion potential in the mid to late 2030s.
As a company, after this acquisition, we really do expect substantial non-GAAP EPS accretion from these assets in 2027, and we really are generating significant cash flow from it that we can then reinvest in innovation. With that, I'll hand it over to you, Greg.
Thank you, Cristin. Everyone, can you hear me okay? Thank you for the invite. It's fantastic to be able to talk a bit about BioMarin's research and development. We are in the process of building out our pipeline, but there are a couple of highlights I want to share for you. One is actually published just yesterday, so hot off the press, our hypochondroplasia data. This is taking the CNP analog, VOXZOGO, and applying it to a related but distinct disease, hypochondroplasia. Those results actually exceeded our expectations, both from a growth standpoint as well as some of the secondary endpoints. We're very proud of that data. It's actually been submitted to the FDA, and we'll be able to update folks soon once that filing is accepted and we have a PDUFA date.
If you think back more towards the pipeline, we've had two new additions to the pipeline, one that came along with the Amicus acquisition, molecule that was referred to as DMX-200 from Dimerix. We have the U.S. rights. We call it BMN 820. This is for focal segmental glomerulosclerosis, so FSGS. It's a CCR2 oral inhibitor, and that is a phase III program running in that renal indication, which will have phase III data looking at two year proteinuria out in 2028. So thrilled to be able to add that in the U.S. rights to that molecule. Another hot-off-the-press update for you, we closed, just last week, acquisition of a post phase I product from Aleta Therapeutics. This is ALE-01. This is a molecule that's trying to address hypophosphatasia, but rather than being an enzyme replacement, this is more of a substrate reduction therapy.
Again, the promise there is that not only might you have an oral convenient therapy, but there may be an opportunity to reach some of the tissues that the bone-targeting biologics are unable to reach just because of their PK and their distribution. That includes muscle and joint and so forth. I'll just wrap up by saying that we've got internal innovation as well that's continuing to percolate through our pipeline. Probably the star of the show is BMN 333, which is our next generation CNP analog, more long-acting, weekly administration. Our goal there, I think we'll talk about it a little bit more, is going to be to actually deliver a greater pharmacodynamic effect than what VOXZOGO can achieve.
We have a variety of proof points, again, that tell us that we're headed in the right direction, and we have a molecule that can answer this question definitively. That's in phase II right now. We're enrolling, and it will have data available next year before we launch a seamless phase III thereafter.
Okay. Well, great. Lots to dig in there. I think it might bring us all the way back to the start with VOXZOGO and the commercial franchise in achondroplasia today. I guess one of the things that's come across for a couple quarter updates now is just the growth that you are seeing within the under age 2 cohort of the market. Can you just talk about how much headroom for further penetration you are seeing in the under two year-old population, and how much of a growth driver this could be going forward? Then perhaps even just stepping back as we think about the franchise as a whole within achondroplasia, what some of the key growth opportunities are looking forward.
Yeah, thank you for the question. Specifically to VOXZOGO, we did report in our earnings call strong growth. Revenue at 14% year-over-year, and importantly, patient demand at 20% year-over-year. That is our global numbers. What we had said is that in particular, that growth, and especially in the U.S., is being driven by exactly what you just referenced, Eric, and that is that zero to two population. It is such an important population for this therapy. Most importantly, because if you look at medical consensus guidelines, it really does suggest that you treat right at the time of diagnosis, because that is where that child can then benefit the most in the long run.
We are really excited that we have the exclusive label there, and we anticipate being the only product approved in the zero to two population for the next couple of years. That is an area that we really do see exciting growth coming out of. Not to mention, some of the other growth drivers are that we are continuing to expand and accelerate in certain countries globally outside the U.S. As a reminder, 75% of our revenues come from outside the U.S. There we have growth happening in countries where we are already well-established, and this is where you see the highest growth coming from that zero to two population, because it is an incident population that you are catching at birth. In other countries, we are either newly launching or still expanding into those countries, and so you see growth across all age groups.
It really does come down to a kind of country-by-country look at where that growth is coming from. But in total, we are commercialized in 55 markets in our 80-country footprint, and that is an area that we will continue to grow in. So very exciting for achondroplasia. I certainly want to mention the fact that what we also announced since the quarter closed was the settlement that we reached with Ascendis in terms of getting royalties on net sales of YORVIPATH, which was the first competitor to launch in this space. So we have a 20% royalty on U.S. net sales and an 18% royalty in the EU, Brazil, and South Korea. What excites us most about this is that this does just kind of clear an overhang for us.
This is something that enables us to capture value right away, which our ITC proceedings would not have done. We would've been in further litigation in both the U.S. courts as well as the jurisdictions where our IP is. I think most importantly for us, we think that this is a really important moment for both patients as well as BioMarin to kind of clear this overhang and be able to move forward. That's looking at achondroplasia. Another big and exciting growth driver for VOXZOGO is in hypochondroplasia, and I know you'll speak more to this. We're anticipating a launch should the regulatory bodies approve in the coming year, so we have the team certainly gearing up around that. As we look further out, we are looking to, as Greg had mentioned, really bring forward the hopefully best-in-class long-acting CNP.
The durability of that franchise you can see over time growing.
Let's hold off on hypochondroplasia for a second. Just stick with achondroplasia. Two things. One, just picking up on the competitive dynamic a little bit with YORVIPATH now, right? Appreciating that the settlement does allow you to capture some preserve, I guess, value, right? Or capture some upside, I guess, up at the front versus ongoing litigation. I guess that said, from a share standpoint, what do you anticipate in terms of switching away to YORVIPATH from the existing franchise? I'll leave the question there and follow up on a different point in a second.
What we shared, excuse me, so far has been that we've seen 90% retention on VOXZOGO out through July. Mind you that YORVIPATH launched in February. We've still seen 10% have now switched in the U.S., which is where they've launched. 10% have switched over to YORVIPATH. We've retained 90% on VOXZOGO. Primarily what we hear from families around why they've stayed is multifold. One is that they're seeing the benefit of the product. That, for them, is really important when they know that the safety is there, the efficacy is there, and they're seeing their child benefit from it. In addition to that, many have created a daily routine that they're comfortable with, and that's working for them.
And then we've also heard that they've really appreciated the wraparound services that we have put in place with our clinical coordinator team who are there to help them with injection education, et cetera. That has been beneficial. That is in large part what we've heard. What that story turns out to be, we are monitoring very closely. We will continue to provide color on as we see more, but we're quite happy with seeing that 90% have stayed. I think it also does show that this is not, achondroplasia in particular in the U.S., is not an easy market just to come into and just kind of overturn or somehow capture all the market share. It is an area that these are patients that are being treated by multiple specialties. They're dispersed in the communities.
They don't necessarily see their treating physician frequently, and some are still reticent to treat at all. I think that we're seeing, we've seen that certainly, but I think we're also seeing our competitors see the same.
Got it. Okay. Can you touch on any of commercial implications possibly from a full approval of VOXZOGO next year? Could full approval meaningfully influence demand, access or pricing of the product?
Maybe if I can take that first from the regulatory standpoint. I think the full approval provides three changes for the way that I think the world will view the label. Two of them tend to be rather tactical. One has to do with, again, the way that materials are reviewed by the FDA, and it streamlines them and makes them easier. Second would be on a regulatory level, when there is a full approval for an indication, that does create a headwind for new accelerated approvals in the exact same case, though, to be fair, the FDA uses their discretion, and if they feel, again, it's in the best interest of patients, that tends not to be blocking for follow-on. The third is it gives us an opportunity to have our full data set into the label, and that is really where we see the most opportunity.
Yes, there will be some physicians who I think value the idea that this is full approved, it is not on an exploratory endpoint or not on an accelerated endpoint, but more importantly, it is the details of what goes in that label. The kind of things that are being reviewed by the FDA now are the upwards of 10 years of safety data that we have. There is 1,200 patient years of clinical trial data. That is a tremendous amount of evidence. In addition to the safety that that affords, and again, the opportunity to tell that story, we will be telling, of course, the height story. That is not just annualized growth velocity, but there is an opportunity to present something that often is called final adult height, which is a bit of a misnomer. It is a defined time point with five, six years of growth, for example.
Similarly, we will be able to put forward our proportionality data. We will look at body composition. There will be an opportunity to have them review our FRAME and MagNUM data as well. We have got a variety of data sets, including quality of life, that will be in front of them. Again, it will be up to their discretion in our back and forth on what makes it into the label. But that will allow us not just to have medical dialogue, but the opportunity also to have our sales reps also work with the physicians and share that totality of evidence.
What I would say is that it is not necessarily, and we do not expect a change in how the medicine is necessarily used, because our label really already covers children with achondroplasia that have open growth plates. But what I do expect is that it creates more confidence because of all the data that you have just described, Greg. I think that it really does create more of that confidence in the product, given the amount of patient years we have, the safety, the efficacy that we are seeing, et cetera. Just as a reminder, it would be the first product that would be fully approved for the treatment of achondroplasia, which is also exciting.
That is great. Let us shift to hypochondroplasia, right? You made note of it. We see the publication just out yesterday. Maybe just from a market sizing standpoint, it would be good to have you frame the opportunity in hypochondroplasia, maybe perhaps relative to achondroplasia, not only in terms of prevalence, but also rate of diagnosis and really, treatment-seeking interest.
Yeah
in patients.
These are precisely the things that we're working on now. I'll start off with just the prevalence. We anticipate or expect that the prevalence is roughly that of achondroplasia. What we've shared in terms of the total addressable patient population in achondroplasia, we've shared that the global number is estimated, we estimate it around 24,000. However, we've said in hypochondroplasia, we see the total addressable patient population at 14,000. The reason for that big delta is actually the diagnosis rate. Not that the prevalence is necessarily different, but the diagnosis rate is dramatically different. The estimations now in terms of diagnosis are somewhere around 30%. We intend to move the needle on diagnosis, and that's the work that we're doing now. Greg, I can certainly have you speak to that.
There's the work that we have to do on diagnosis, but also what you alluded to there was around also the treatment patterns and the urgency to treat. One of the most important things that we are working on now and will continue to do is to raise awareness around the condition itself so that treaters and families alike understand the comorbidities that come alongside hypochondroplasia, and importantly, why the importance of treatment really is there. So that's work that we're currently doing now. Is there anything you want to elaborate on that, Greg?
No. A lot of what we would call market shaping that's gone on. That includes, again, this idea of education. But also we've been side by side with physicians, a consensus guideline for definition of the disease and how to define it is out now. Similarly, we've been working with pathologists, even on nitty-gritty factors like trying to reclassify so-called variants of uncertain significance. Again, we know that there's a variety of mutations in the FGFR3 protein that cause hypochondroplasia. This is slightly different than what you see in achondroplasia, where one mutation dominates. Here, it's a big, long tail of different mutations. So clearly making sure that that bin is accurate, those mutations, they fit into, again, this diagnosis. That's an important factor. Of course, when we start looking at efficacy, we want to make sure we see activity across all of those subsets as well.
In the publication, we have that data. It looked great.
Really quickly, just while we say the pacing of this will be in large part how fast we can drive the diagnosis rates up as well as the urgency to treat, the benefit that we have here is that we are not starting from scratch. We have built a commercialization capability in and around skeletal conditions, achondroplasia. These are the same treaters, thought leaders, et cetera. This really, I think, does provide a benefit for us as we come out and launch this product.
I was just going to add, we also have a story to tell that is not just about height any longer. This is in the publication, but we actually type 1 error controlled. We were able to show that when treated with VOXZOGO versus placebo during that first year of double-blind placebo control, that there was a highly statistically significant difference in arm span as well. You may say, "Well, yeah, it is arms. How is that different than the legs and the height?" Well, it is absolutely important in the way that these children interact with their environment. Think about combing their hair, reaching up onto a shelf, driving, these sorts of things. From that standpoint, we think both the payers and the patients are going to see this data set.
Like we were, be quite pleased to see that there is a, presuming it gets approved, a safe and effective therapy for patients.
On the issue of potential approval, is full approval on the table here? If not, do you have regulatory clarity of what would be needed to support full approval in, excuse me, hypochondroplasia?
Yeah. It is an excellent question. Of course, we have been talking about the full approval package for achondroplasia, and clearly there are precedents where that could set up a follow-on indication if it is closely enough related for full approval to be considered. Ultimately, that will be at the discretion of the regulators, but I just want to reassure you, we are working very closely with them. We have two files that are in the FDA right now, other regions as well. From that standpoint, looking forward to being able to close the book again on that full approval for achon, and again, see what comes with hypochondroplasia. So more to come, and I think as we get more clarity from our interactions with the FDA, we will be able to share more details on earnings calls.
Before we leave this space with skeletal dysplasia, maybe we could touch on BMN 333, where you have guided to an update from the phase II study portion of the pivotal trial you are currently running. Maybe just you could help with sort of stage setting there, the endpoints that you are most interested in interrogating in that phase II study portion, and what would you hope to see that would satisfy differentiation versus VOXZOGO?
Yeah. What we know about BMN 333, number one, it was engineered to have a longer half-life, so it has got about 100-hour half-life roughly. It is a construct that again, releases the free CNP into the bloodstream. Think of it like a depot in that sense. So it is more of a continuous administration. That is very different than, of course, VOXZOGO, that has got less than an hour half-life, more of a sawtooth pattern for PK. With that continuous exposure, we ask two questions. Can we see more PK coverage safely as compared to, say, YORVIPATH ? Can we see more PD, at least in the bloodstream? The short answer is yes. We published a phase I protocol, it was at ENDO this year, where we saw up to 13-fold increases in AUC for free CNP as compared to what YORVIPATH was able to produce.
Similarly, that was accompanied by conservatively a two to five fold increase in cyclic GMP in the plasma. Again, it's not the growth plate, but again, it shows that there's more biology at least to be tapped into, at least in the bloodstream. So one step closer. The next step, of course, is we need to move out of healthy volunteers, and we need to move into patients. We're running a study in treatment-naive achondroplasia patients, so you need to again go to the countries where those patients exist. We're in seven countries around the globe right now. That's increasing. We're in the steep part of the enrollment curve. More to come in that regard. But that study is a classic dose-ranging study.
It's looking at three different doses of BMN 333, call it high, medium, and low, and we will be looking at an internal control of VOXZOGO there as well. Our goal is to pick a dose as compared to VOXZOGO that will give a high confidence in a phase III to have a superior growth profile. So what are we going to look at in that phase II to determine that? Well, two things. One is we will b e measuring annualized growth velocity. We will cut the data when every patient has at least six months of follow-up. So again, it'll be more than six months in some patients. There'll be a Bayesian approach applied. There's an internal control of 10 patients on VOXZOGO. That will be a quick spot check.
We have hundreds and hundreds of patients of VOXZOGO data that we can put into a model if we need to as well. But the goal there is, again, not only looking at the dose response, but also we'll have the ability to look at exposure response, which across three dose levels of 333 is going to give us hopefully a good spread of exposure there. And with that, we would pick a do se to move forward to phase III. We'll of course, look at safety data as well. But the phase III, as it's currently designed, is a superiority study. There's no placebo, so patients either receive VOXZOGO or 333, and we've powered it to have a 90% power to detect a 50% improvement in attributable AGV. That's not the minimum detectable number. I mean, you can back calculate the math, but it's 60 versus 60 in each arm.
Again, the go signal would come out of the phase II. That phase II data will be available to us, and we would be planning to publish that. That is going to be something that we will announce sometime in 2027.
Okay, great. All right. Let's shift gears a little bit to the pipeline. I want to pick up on the Aleta ALE-01 acquisition. Acquisition closed recently, right?
Last week.
Last week, right. Yeah. Just by MOA, just to be clear here, this is an ENPP1 inhibitor, if I have that correct?
It is. It's a small molecule ENPP1 inhibitor. So in these individuals, they have high pyrophosphate. That's their problem. That's a direct inhibitor of bone mineralization. And fantastic work that's been done for the field with STRENSIQ and the follow-on agents that are basically replacing the deficient enzyme, which is tissue-nonspecific alkaline phosphatase. And a t the bone, it's taking that high pyrophosphate and converting it through to its end products. A lot of great work's been done there. This is a slightly different approach. I guess similar to Gaucher disease is this is upstream. It's trying to deplete the substrate, trying to deplete this pyrophosphate by inhibiting the enzyme that creates inorganic pyrophosphate.
Okay.
And that is ENPP1. A lot of good preclinical data. There are some sort of gene load mouse crosses that suggest that about a 50% reduction in ENPP1 enzyme activity can restore bone mineralization in H PP models, in rodent models. What we have seen with this small molecule inhibitor of ENPP1 is we have seen healthy volunteer data and a limited amount of patient data, again, looking at the PK profile, the PD profile, and the safety.
Again, seeing that the molecule is meeting the requirements that we would want to see to take it forward into patients. So now, again, we are acquiring this molecule. It is finishing up its phase I portion. Thi s is a study that is running outside the U.S., so of course, we have got to finish chronic docs and get it filed in the U.S. But the promise here is that you do not just have a more convenient STRENSIQ.
The promise of the molecule is that it actually will also be able to reach those compartments that are not bone related. So particularly, the HPP patients fall into a couple different bins, a couple different buckets on when the phenotype presents itself, and particularly those where it presents later in life. So the adults with HPP, many of their complaints are not the classic bone-relate d pathology. Instead, it is things like fatigue, malaise, muscle pain. A small molecule without the bone-targeting biologic aspect has the promise to be able to target those compartments as well. So that is where we see the opportunity here.
It is an established commercial space. There are established regulatory endpoints, some great work that has been done by the alkaline phosphatase molecules, really to work on these patient populations, understand the endpoints. We are hoping just like they are, to learn from that, and again, bring patients something of value.
Anything that you want to add, Cristin?
Just as in relation to the ongoing study, any sort of loose guidance you might offer in terms of when we would expect an initial readout and sort of the scope of that readout, understanding that it is
Yeah
both, let me have this right. Is it both in healthy volunteers and in patients, right?
There are HPP patients in it. Since the data's a week old, we're still working through, again, exactly how the phase I to phase II transition's going to play out. We, of course, need to engage with regulators. The short answer, tho ugh, is we plan to publish this data next year.
Okay.
There will be a public disclosure of both the healthy volunteer and some amount of patient data. What you can expect is not only PK profile, but also PD, enzyme inhibition. Again, as I mentioned, the preclinical models suggest something between 30% and 50% reduction in ENPP1 activity could have dramatic influences on pyrophosphate. It's, again, it's been shown quite nicely by the alkaline phosphatase molecules that all of the benefits that they achieve in these older patients, we're not talking about infants, but these children through the adults really tracked with decreases in pyrophosphate. So we think of this as a fairly de-risked approach. Again, we'll be presenting that data more broadly next year.
Just in thinking about the spectrum of disease that you hope to address, particularly the older adult onset that you spoke to earlier. I guess we have a lot of development and regulatory precedent in the juvenile side. I'd imagine a little bit less on the older. Maybe you can just sort of speak to the development outlook or what development plans could look like in that segment and what you would ultimately hope to demonstrate clinically.
I think what the field has learned, and it's been some great work, is that these are distinct populations. You can talk about when the onset of it is. Is it a juvenile onset adult, an adult onset adult, a teen, a pre/post bone growth plate closure? All of this is important, but you have to target not only the dosing, but also the endpoint measurements to the right population. Clustering them together is probably not the right answer. Moving forward, again, we envision teasing these populations out. Then, again, the beauty of the timing of this is that we can look at those individual populations and continue to do endpoint development, particularly with the adults, where once the growth plates are closed, you can't look at the rickets scores and so forth. You don't have that window into bone formation.
The more blunt endpoints, like six minute walk test, some of these functional endpoints have been notoriously fickle in order to see the effect of your drug. There are newer technologies that are measuring motion. There are subsets, sit to rise. There are a variety of different measures that we would intend to explore in our phase II program, so that by the time we reach phase III, again, we would have solved not only the patient population question, but what's the right endpoint and what's that effect size? How do we power that study?
Excellent. Maybe, again, moving further down the pipeline or elsewhere in the pipeline, I should say, I want to pick up on the Dimerix compound, BMN
Yeah.
820 in FSGS. I guess that study is now fully enrolled, and you had talked about a readout in 2028. That being said, and I guess it's on 2-year eGFR that you're looking at. I don't want-
Two year proteinuria.
Excuse me. Excuse me. Sorry.
No worries.
Sorry. Thanks for the correction.
It's a big movement in the field. Again, sparsentan has done some great work opening this up. The FDA now accepts two-year proteinuria as a full approval endpoint.
Okay. Right. Okay. You are right, because the question that is lingering in my mind is whether there's an opportunity for accelerated approval based on one-year endpoints that are being leveraged in like diseases, or in dis eases in the kidney disease category. Any thoughts around the flexibility that you any optionality, I guess, in the conduct of this trial where you might look to either look at data early and follow it on an accelerated timeline?
Our current belief is that the two-year reduction in proteinuria is the endpoint.
Okay.
It's fairly solid. Now, that being said, we have some knowledge of, again, how the study is performing already. That's the study remains blinded. There was a fairly small phase II data set that this phase III study was built upon. That being said, we know that the study has, number one, passed through futility analysis. Number two, we know that it also, from the standpoint of sample size, th at it's adequately powere d to measure a meaningful decrease in proteinuria. There was a formal sample size reassessment done, again, on blinded data, and the study is appropriately sized. It over-enrolled a little bit, I think, which is great. That was by demand. Patients were interested in being on the study. Again, the promise here is that an oral therapy, it targets CCR2. The idea here is it's not necessarily a vaso-targeting agent.
It is targeting this macrophage-induced inflammatory response that, again, leads to the destruction of some of the kidney podocytes. The hope here is that by having a molecule that would have a meaningful reduction in proteinuria and not have the baggage of things like a black box warning, not having the baggage, again, of having to restrict to certain levels of proteinuria, to non-nephrotic syndrome patients, that t hat would be of great interest to the community. Being a different mechanism of action than the pure vascular si de as well, the idea of combining these agents becomes important. This study is designed to add on to ARB therapy as a baseline. It is not displacing, like sparsentan did, a prior ARB inhibitor. It is an ARB inhibitor plus minus the DMX, the BMN 820 compound. From that standpoint, again, you have to make sure you look at it through that lens.
That meaningful decrease in proteinuria is something we know the study is well sized for. Again, a lot of promise with regard to this mechanism of action that it will offer patients something important.
Excellent. I am going to end on a thematic question here. Our colleagues in tech a re also running a conference this week, so perhaps I am asking this question on their behalf. Obviously, we are often thinking about how AI-enabled tools impact our businesses, all of our businesses. How is BioMarin making use of AI tools today, and how are you measuring their impact?
Yeah. The tools can be quite powerful if you put them in the hands of the right people, you feed them the right data in the right format, and then you do something with it. I would say there are two places in R&D that we have really leaned into. One i s reading and writing, right? We have a very document-heavy work. The idea of cutting out the white space between times, for example, when we lock a database and we file a program, it is about using AI tools to create some of those documents, the filings, but also inserting that into the workflows in the right way so people can get these work products out as fast as possible. We had a great example of a success recently with hypochondroplasia. 79 days between database lock and the submission to the FDA.
That does put us in, at least the McKinsey numbers would put that in the top quartile. The whole field is moving fast. That's great for patients. Otherwise, this is busy time, so to speak. You can imagine having that kind of approach all along a drug's life cycle, whether it's filing of an IND or, again, filing for a new clinical trial, all of these factors can add up and reduce cycle time. I would say that the sec ond major area that we're using AI has to do with patient finding. In that regard, using some of these algorithms to look at very complex data sets. They might include genetic data, looking for hotspots around the globe. They may include social listening and, again, the activities that are going on where we know that patients who are quite educated are seeking out information.
They're just not finding their way to a physician. Then, of course, the opportunity to do good old-fashioned looking at claims databases and so forth. That does allow us to find patients not only in the commercial sector, but also for our clinical trials. We have some great examples where we even found a patient who was an in utero patient. It was a mom looking up for hypochondroplasia on the inte rnet, and again, she was forwarded to one of our clinical trials. So they work. It's very helpful. I would say that the area that I would love to see us lean into more, and we're dipping our toe here, is giving these tools to the scientists and allowing them, again, when it comes to molecular design, organic chemistry and amino acids are another form of a language, right?
The large language models do a good job creating some of these reagents. That'll hopefully cut down the time and the number of molecules that we have to test as we go through our own funnels to bring these things into the clinic. So we have the luxury at BioMarin of, we have some resources. Again, with all the work we've been doing, we have about $1 billion of free cash flow a year. That means that we don't always have to be on the bleeding edge of these technologies, and we're finding that there's a very vibrant third-party sector. They battled it out for the winners. But for writing things like clinical study reports, we're able to actually test a couple of them and see which of these tools is the one that works best for us.
We're finding that in addition to some of our work with Veeva and so forth, getting our data lined up, I do think that there's a major opportunity here to do more with less. As an R&D guy, I've got a budget, and I need to get as much activity into it and get as much to patients as I can, and that's really the name of the game right now. Technology is part of it, but it is enabling the people who have the creativity to do the work.
Great. Well, that's a good note to leave it on. Thanks so much, Greg and Cristin, f or your time today. We really appreciate it.
Thank you.