Hi, everyone. Good afternoon. Thanks for joining us here on the first afternoon of the William Blair Growth Stock Conference. If you don't know me, my name's Andrew Brackmann. I'm the Equity Research Analyst covering the diagnostics vertical for us here at William Blair. We're very happy to have BillionToOne, and the CEO and co-founder, Oguzhan Atay, join us this afternoon. Their first time at the William Blair Growth Stock Conference after IPO-ing successfully in November. We'll run about 30 minutes in terms of prepared remark presentations here, and then we'll head up to the breakout in Jenny A following this. Lastly, for a full list of research disclosures, please visit williamblair.com. With that, I'll turn it over to Oguzhan.
Thank you, Andrew. Thank you for having us here. Is that a clicker?
Yes.
All right. I don't do prepared remarks, so we'll see how this goes. I have a few new slides that might be interesting for all of you. At BillionToOne, we are redefining what is possible with molecular diagnostics, with a technology that achieves a single molecule level sensitivity and precision. We have four pillars of differentiation that we believe make BillionToOne a different category of molecular diagnostics company. With our technology platform, we revolutionize what is possible, and our patented QCT technology enables single molecule level sensitivity and precision. With this technology, we have built category-defining products, both in prenatal and oncology, and grown exponentially. We have scalable rapid growth. Our revenue has grown exponentially even as we scale past $400 million of annualized revenue run rate. Our 2025 full-year revenue was 100% growth over our full year 2024.
We are still scratching the surface of what is possible in these cell-free DNA markets. With our technology, we are solving a fundamental problem in cell-free DNA, building category-defining products that are not just incrementally better, but that are so much better that it changes what is possible with these tests. We are significantly growing and getting adoption in a market that is sized to be $100 billion. We believe that there is so much more growth that we can have over the next few years or even next few decades. We also have been able to combine this rapid growth with superior gross margin profiles. We have achieved 70+% gross margin profiles, even with sub-scaled ASPs and using only a fraction of our total lab capacity.
As we continue to scale, we are seeing significant opportunity for further ASP growth, as well as significant reductions for COGS per test. This is truly remarkable to achieve this level of gross margin, especially as we are building and launching new tests that initially do not have broad coverage and reimbursement. The thing that I am most proud of, the thing that is, I think, truly differentiated in our company, is that we have been able to achieve GAAP profitability and positive cash flows with only 10% of the accumulated deficits of our public competitors. We have a culture of fiscal discipline, efficient operations incorporating AI, and from day one, we have optimized for what is best for the company in the long term, rather than short-term gains in the market share.
It allowed us to build a differentiated company that is not just growing rapidly, but that is also generating significant profits. Going into these pillars one by one, our revolutionary technology platform really determines the next paradigm in molecular diagnostics. Similar to how 1990s were defined by PCR for infectious disease testing and hotspot mutations, which was now supplanted by 2000s with the first Human Genome Project and the early sequencing efforts that resulted in more testing to be done for genetic testing. BRCA1 and BRCA2 are early examples here. In 2010s, as sequencing cost decreased, it has been possible to do more and deeper sequencing. Deep sequencing allowed prenatal and oncology cell-free DNA tests to be done from blood, and we consider these tests to be first-generation tests.
The fundamental problem in cell-free DNA, as you go into lower limits of detection, as you go into more difficult, complex problems, has never been about deeper sequencing. You start with very little cell-free DNA, and you have to amplify that millions to billions of fold using PCR and other methods. These methods incorporate a lot of errors that are indistinguishable from what was originally in the sample. The fundamental limitation in cell-free DNA is the noise. It's not the signal, it's not deeper sequencing. It is what our technology solves. We have been able to achieve single molecule next generation sequencing, which is designed to unlock the full potential of cell-free DNA. We do this with a simple but elegant idea. We realize that the biggest problem here is the noise that is added by the amplification and sequencing process.
Remember, every molecular diagnostics goes through this, especially if it is cell-free DNA. It is 20, 30 cycles of exponential amplification, millions to billions of fold, which add these errors that are indistinguishable from what was originally in the sample. This is also why there are so many tumor-informed MRDs. If you sequence the tumor to begin with, you know exactly what is in the tumor, so you can ignore all these errors that are being introduced by the amplification and sequencing process. That is why it is a very easy problem to solve. That is why all these companies develop tumor-informed MRDs. That is also why this is something that we don't do, because our technology actually is designed to solve the noise problem without having to look at the tumor.
We design and add these artificial synthetic DNA molecules, that are all single molecule, into the sample before any amplification and sequencing happens. These artificial synthetic molecules amplify along with the sample. It allows us to know where the errors are being introduced so that we can remove them from the data that we get from sequencing. It's a little bit like using noise cancellation headphones in electronics. If you know exactly what the noise characteristics are, you can remove that noise and be left with the pure signal. Whereas, deeper and wider sequencing is just increasing the volume, which is also going to increase the noise. We believe that computing molecular diagnostics are significantly limited. In prenatal, this has been the case, has been limited to chromosomal level changes. In oncology, even in late-stage cancer patients, limited by the sensitivity.
This is why tissue is still seen as the gold standard. With a truly sensitive liquid biopsy technology, sometimes you are able to find not just what is in the tumor, because tumor is still looking one section of that tumor. Sometimes we are able to report results that are not even found in tissue biopsies. With UNITY, which is where we first went into, we redefined what it means to do a non-invasive prenatal test. This was a competitive, commoditized market with so many competitors, more than 1,000 sales reps across them, and we went into it without having resources, and we have become the second-largest prenatal lab and continuing to grow faster than any other lab in molecular diagnostics. With UNITY, we did not want to solve what others were working on.
When other companies were focused on aneuploidies and microdeletions, millions of base pair changes, we developed the technology to be able to look at recessive conditions like cystic fibrosis, sickle cell disease, that are caused by a single base pair change. These conditions are very important to screen for. I would argue that they are more important to screen for even than the traditional NIPT conditions, because for these conditions, you can actually change the treatment paradigms and the outcome of the babies. Today, problem is, a lot of these conditions, actually more than half of these conditions, are not being detected due to the difficulties of testing the father. Misattributed paternity and missing paternal screening means that only about 35% of these important single-gene conditions are getting screened, despite the fact that billions of dollars are spent to screen for them in every pregnancy.
We increase this by, without requiring the partner DNA, directly detecting the causal variant from cell-free DNA at a single base pair resolution. It detects three times more affected pregnancies than traditional carrier screening methods that rely on partner testing. One real-world patient case, I think really highlights how this can be extremely impactful. What we are seeing here is incredible because a great diagnostics does not just change the information that you are getting. A great diagnostics can even change treatment paradigms. Just a few years ago, this would have been unthinkable. Patient here, unknown carrier status, for the patient, for patient's carrier, UNITY determines that this is a high risk for cystic fibrosis. Diagnosis was confirmed via amniocentesis, and the insurance company actually approved approved Trikafta on the pregnant mother in utero for the baby. Prenatal therapy initiated at 27 weeks.
Not only the ultrasound findings resolved, this baby did not go to NICU, which again, otherwise, the ultrasound findings wouldn't get resolved, they would have been in NICU. This baby even passed newborn screening. We are seeing every week cases of cystic fibrosis babies that are getting treated in utero. Every week. We are hearing these cases where these babies are passing newborn screening. Some of them have no symptoms of cystic fibrosis, no pancreatitis, some with full vas deferens. Incredible outcomes for these babies, changing the entire treatment paradigm of cystic fibrosis. Just five, 10 years ago, most people with cystic fibrosis would have a lifespan of 30 to 35 years. Now they are having normal lives. The other thing that I want to highlight here is that we were the first to launch single gene non-invasive prenatal test for these recessive conditions. We didn't stop there.
We launched the first RhD NIPT that still today actually equitably serves all of the U.S. populations. The other tests don't work for African American and Asian populations. We launched the first fetal antigen NIPT, which already changed the medical guidelines. This is for fetal-maternal blood incompatibility. We have launched, in Q1, the first FNAIT NIPT for platelet incompatibility between mother and the fetus. After changing the guidelines for fetal antigen testing for alloimmunized patients in the U.S., we have continued to expand our lead here and continued to move the field forward by adding things that are not available through any other laboratory. Most recently, in Q2, we launched UNITY Confirm, which many key opinion leaders agree with us that it is the innovation of the decade in prenatal genetics. This has always been seen as the holy grail of non-invasive prenatal testing.
Because the problem is, today, if you get an NIPT, and 80% of pregnant patients in the country gets an NIPT, a non-invasive prenatal test for aneuploidies. If it is a positive result, the PPVs of these tests are not 80%, 90%, 100%. For some of the conditions, it can be as low as 50% or lower, which means that you are left with an impossible choice. As a pregnant mother, you are counseled that there is a, let's say, 50% chance that your baby is affected. Because of NIPTs, there are not even that many specialized physicians now who'd even do these invasive testing.
Best case scenario, after waiting six, seven, eight weeks, you are going to see a specialist who might be three, four hours away, where they are going to do an invasive testing to get a sample directly from the baby, from the amniotic fluid, to confirm whether it was affected or not. 70%, 70%, of patients decline invasive testing, and they have throughout their pregnancy, this fear of the unknown, this uncertainty, this anxiety that they have to live with. It's truly terrible. This is the problem that we solved here. This has been seen as the endpoint of screening. If we can capture an intact trophoblast, intact fetal cell directly from maternal blood, we would be able to do whole genome sequencing on it. We wouldn't have mixture of maternal and fetal. It would be pure. This is a much more difficult problem.
Cell-free DNA tests that we talk about will have five, 10% of the DNA coming from the fetus. Here we are talking about less than one in a billion cells coming from the fetus. We developed this technology, and it is only available if a patient on the frontline has used our NIPT test. We don't think of this as a revenue driver. We think of this as a big differentiation that solves this critical unmet need for the patients, for the providers, and that will drive the frontline adoption of our screening test, because it's only available for those who have been tested with our tests. In Northstar, we are redefining liquid biopsy for cancer care.
We have two tests, both of them are for late-stage cancer patients, a therapy selection test that determines what mutations are in the tumor, just from a blood sample, and a response monitoring test, after the therapy starts, to determine whether the patient is responding to therapy or not. At single molecule precision, determining, quantifying the changes in tumor burden so that we can know months ahead of scans whether the therapy is working or not. We know that we have come to this field much later than other companies, that is why we realize that for us to be successful here, we need to do something that has not been done before. We have done a prospective head-to-head comparison with other competitors. This was unbiased. We asked physicians across the country to use whatever test that they are using for liquid biopsy of choice.
On the day that they are drawing those patients, to send us a blood sample as well, a research sample. Same day, same patient, physician choice of competitive lab, and same blood draw sent to us on the same day. We detected 50% more actionable alterations for SNVs, and more than 100% for copy number variants. This is not a 5%- 10% difference. This is a dramatic difference that every physician within their clinic can actually easily feel. You need to only run five tests side by side to be able to get better therapies for at least one of your patients. That is why we have been growing very fast in our oncology business. I sometimes get this question, "Okay, you found more alterations. Does this change anything?" 69% of missed alterations by other tests were clinically actionable. This makes a big difference.
This is a real-world patient impact, real-world case. Patient in their 30s, diagnosed with stage two rectal cancer. Provider ordered tissue NGS test. We don't do any tissue testing. We believe that is an easy thing to do, and we don't do easy things. Tissue NGS test results are negative. They couldn't find an actionable alteration, so they are deciding to proceed with chemotherapy. For this particular cancer patient and cancer type, if they proceed with chemotherapy, the outcomes are not very promising. This particular provider didn't really believe in liquid biopsy. It was actually the nurse practitioner who convinced the provider that we should try everything for this patient.
She had heard about Northstar recently, so she said, "Can I send a Northstar Select test?" Provider said, "Sure." The test comes in, and we actually determine that this patient is MSI-high, which is so much more difficult to detect in liquid than tissue. We did. This unlocked the opportunity for immunotherapy, and in clinical trials, it has been shown close to 100% cure with stage two rectal cancers who are getting immunotherapy. This patient went from having a very poor prognosis to essentially getting cured because we found something that was not even found in tissue. I hope I convinced you that we have the most sensitive liquid biopsy out there. We have done an unbiased head-to-head. We actually do those unbiased head-to-heads over the country all the time now, just to prove it in individual clinics and health systems.
We realize that there is another problem with liquid biopsies. White blood cells can actually contribute to cell-free DNA, and that can look like a tumor-derived mutation. It turns out that one in four solid tumor patients in their blood harbor at least one CH, clonal hematopoiesis alteration. This is a big problem. If you think about it from a actionability perspective, one in four patients might be treated by a drug that they have no possibility of responding because it is not derived from the tumor. It's coming from the white blood cells. This can be particularly problematic in PARP inhibitors.
We have built a panel-wide CH filter that combines machine learning with ultrasensitive buffy coat sequencing of CH-prone clinically actionable DNA repair genes recommended by clinical guidelines, and we have achieved 99% accuracy, more than 99% accuracy on CH calling for all clinically actionable variants. With this, I think we went from being the most sensitive test to being most sensitive and most specific test. With Northstar Response, after the therapy starts, at some point, the patient might progress. We have previously released this result that show that in immunotherapy patients, just looking at the baseline to three-month change is a strong predictor of long-term outcome in these immunotherapy patients. We actually had, again, these are late-stage cancer patients, and we are only looking at the first three months, get a separation of more than two years of overall survival.
We also, and this is new data that we presented at ASCO, now we went one step further, and what we showed here is that the molecular progressive disease that we are looking at showed better performance than scans, than . Among the patients classified as non-progressors by scans, if the scan is saying stable disease, if you look at it with our test, we were able to classify them as either responding to therapy or non-responding to therapy. This shows additional value that is not just that we are earlier than scans, not just that we are better than scans, it also adds value to scans.
Most importantly for MolDX as well, you have to show not just for the initial one, because then you would only get reimbursed for the initial testing, you want to be able to show that this is helpful for longitudinal testing as well. Again, here we have released our NORTH study data where we show that patients classified as progressing molecularly associated with significantly worse progression-free survival and overall survival. What this means is that if the patient never sees an increase in Tumor Methylation Score in the tumor burden, they do really well. If they see an increase, those patients progress. With these remarkable products that are quite differentiated, we have been able to grow rapidly with an exponential revenue growth from zero to $434 million annual revenue run rate in six years.
Most recently in Q1, 84% year-over-year growth, which is driven by both the increases in tests that we receive as well as continued increase in contracting and coverage of our tests, which increased our ASPs by 28% year-over-year. As we increase our ASPs and as our scale increase and our COGS come down, we have achieved superior gross margin profile. In Q1, most recently, we have achieved a 73% gross margin profile. With that gross margin profile, we have been able to attain significant GAAP profitability. You can see as a percentage of our revenues, we went from being very highly negative to getting close to breakeven by the end of 2024, being breakeven positive. These are all GAAP profitability margins, not adjusted. 2025 being GAAP profitable for the full year, and in Q1, this increased to 16% positive GAAP operating and net margins.
We are just getting started. We are investing in our business, in our product lines. As I showed you, every quarter, we are either launching a new product, something that further differentiates our platform or new dataset that show how strong our results are. We expect to continue to be able to do that in the future quarters. We believe that our technology platform, our smNGS platform, will drive our continued and rapid growth in hundred-billion-dollar-plus markets.
All these markets that I am listing here are markets where cell-free DNA testing is fundamentally limited by the other technologies and the noise that they have them. That is why we believe that we can have differentiated, more sensitive, more specific products, not just by 5% or 10%, because if you're a latecomer, that is never enough, but by significant amounts so that we can come in and with even less resources, we can capture them. We are transforming healthcare one molecule at a time, one patient at a time, with a unique technology, a patented technology for the first time that achieves single molecule level sensitivity with single base pair resolution. That allows us to build low COGS products that are differentiated, that are growing rapidly. We have built unique paradigm-changing products. We have changed medical guidelines.
We are changing treatment paradigms, and we have seen exponential growth even as we reach scale. There's so much more room to grow in these very large markets. We have also combined all of this with a culture of extremely efficient operations, productivity, and AI, and that allowed us to achieve GAAP profitability at a much lower scale than any other company in molecular diagnostics that are public and profitable. Our goal, in the future, in the next 5+ years, is to build a category-defining company and become the first molecular diagnostics company to enter the S&P 500. With that, happy to take any questions that you might have.