Conference Transcript - EDIT 2026-06-04

Matthew Pecora, Analyst — Jefferies

All righty, everyone. Good afternoon, and welcome to the Jefferies Global Healthcare Conference in New York City. My name is Matthew Pecora, and I'm here with the Jefferies Healthcare Investment Banking Team. It is my great pleasure to introduce you to Gilmore O'Neill, CEO of Editas Medicine. Thank you.

Gilmore O’Neill, CEO

Thanks very much. Matthew, good afternoon, everybody. It's great to be able to tell you about Editas Medicine and update you on where we are today. I'm going to make some forward-looking statements that you can read at some point or rather you can read the disclosure or disclaimer at some other point but the key thing is that Editas Medicine is a company that is focused on the development of therapeutics that are actually going to be creating meaningful clinical difference over and above the standard of care for patients and that's at the core of our strategic interest. In addition, we are using CRISPR editing in a way that is rather unique in a mechanistically differentiated approach to therapeutics by essentially using it to drive the upregulation or the increase in synthesis of proteins that could actually benefit patients or mitigate disease. And because we're using CRISPR editing, we do it in a durable manner and then finally we're formulating the CRISPR editing packages into lipid non-particles so that we can actually create scalable off-the-shelf simple to use single infusion based therapies with a cost of goods that really means that we can actually have these medicines used not just across tissue types but actually in in health care systems in a maximally effective manner to reduce the burden of human disease and edit 401 is our lead asset which checks all the boxes in that strategic intent in that it has the potential to transform the hyperlipidemia treatment landscape because of its potentially best in class with an unprecedented mean 90% reduction in LDL cholesterol and LP little a cholesterol essentially two key atherogenic lipoprotein particles that are critical to human disease and we have demonstrated those reductions in non-human primates. But like what is the need for lipid lowering in human therapeutics? Well the bottom line is that arteriosperotic cardiovascular disease remains the dominant and key killer and cause morbidity in human health around the planet. I think that's a critical point. I think the second point is that lipids, LDL-C, LP little a, are key independent risk factors for the development of cardiovascular disease. And what has happened over the last 30 years, and what continues to go on, is that the field and the data continue to evolve and are a constant trajectory pushing the required levels of cholesterol that we should get to lower and lower. And indeed, the most recently published guidelines, have actually continued that trajectory, where it really has become clear that we need to reduce LDL cholesterol and LpA to levels that we really haven't targeted before, but the data are showing we must do that. And that is important for many millions of patients who, notwithstanding the current standard of care, cannot get to those desired levels of cholesterol and therefore remain at very high risk and I'm just going to describe two particular groups that sit in that very high risk category for cardiovascular disease complications but actually highly motivated and you might say well what do you mean by highly highly motivated well the bottom line is that cholesterol is your stereotypical silent killer there are many of us in this room who probably don't even know what our cholesterol levels are there are a lot of young looking people in this room who probably have never cholesterol tested However, the cholesterol is actually accumulating and creating that silent accumulation of atheroma, which creates the risk of heart attack, stroke, or renal failure or limb loss. So these patients, when I say they're highly motivated, they actually know that they're at risk. So let me take you through them. The HEFH or heterozygous familial hypercholesterolemia patient is a patient who has an inherited lesion of the LDLR receptor and has very high levels of LDL cholesterol from a very young age. Their motivation is that they've actually seen a close family relative have a heart attack or a major cardiovascular event. They may indeed have had one themselves. And the vignette on the left of your screen is an example of just one type of patient. There's another patient population that sits in that very high risk category and those are patients who have established cardiovascular disease. They don't necessarily have a history or family history of hypercholestering but they certainly have established cardiovascular disease. They have had prior cardiovascular events. They've had a heart attack or an MI. They've had a stroke. So they know that this is not just a silent killer for them. This is a very real risk for them and they also can't get to go with the current standard of care so these patients actually remain at very high risk and are very much aware of that high risk and it is clear to them that you know where they can't get to target notwithstanding standard of care that is 60% or 40% reduction is not enough and this is where edit 401 actually has the potential to offer something to those patients and those patients by the way and I sort of skipped over it, do number many millions of patients just in the United States alone. But what Edo401 is the potential to offer is a meaningful reduction, further reduction, of more than 90 percent in LDL cholesterol and epilipolae. And it can be done in a way that reduces the need for chronic therapy because it's maybe a simple single infusion, and it actually provides intensive durable reduction of cholesterol and therefore meaningful reduction in risk going forward. So before I take you through some of the detailed data, let me just summarize where we are with EDIT-401 and what can offer. A single dose of EDIT-401 has achieved a mean 90% reduction in LDL cholesterol and LpA across multiply-dosed non-human primates. The LDL cholesterol reductions have remained durable through six months, and I'll take you through that study in a moment. And we've seen highly correlated reductions in LDL cholesterol and LP little a. And what they again do is highlight the transformative potential for LDLR upregulation to the levels that we're actually seeing with edit 401. And finally, we have a very promising preclinical safety profile with no adverse clinical observations at therapeutically relevant doses. So overall, the unifying mechanism of LDL regulation really gives us the potential to offer a meaningful risk reduction to those patients that really remain needing it notwithstanding today's standard of care. So let me take you through the data. This is a graph from an ongoing non-human primate study and we have taken a data cut at six months and what you can see on the y-axis on the left is the percentage change in LDL cholesterol you capriciate in the treatment arms that all the animals achieve mean reductions of 90% that was robust and it occurred as you can see across the x-axis which measures time in the first 48 hours to eight days and that has been maintained through six months I would say that this is associated with at least a six-fold mean increase in LDLR protein upregulation or expression in the liver and that's important because that is currently more than what preclinical work has demonstrated that PCSK9 inhibitors can achieve. And again, very importantly, we're now seeing really consistent durability over six months for this reduction. But that's just the LDL cholesterol. Beyond just durability, what was also very interesting was that LpA is also reduced. And this is actually biologically and mechanistically plausible in that LDL receptor does actually take up LP little a even if it's a lower affinity for LP little a and also because it massively reduces LDL cholesterol which is substrate for the manufacture or synthesis of LP little a it actually you know results in this again very exciting mean 90% reduction LP little a and again it's rapid as we saw for the LDL cholesterol and as say combined together demonstrates an ability to reduce atherogenic lipoprotein particles in a way that is going to substantially and we hope additively reduce risk for patients. So where did this idea come from? Why did we think to actually use a very different strategy where we actually make a change in the non-coding region of the LDL receptor gene to increase the levels of LDL synthesis directly. And this came from a critical part of our strategy, which I didn't articulate at the beginning, which is the idea that we would look in human genetic databases for naturally occurring gain-of-function variants. And we found one, and this is actually published data, in an Icelandic kindred of which seven members had very low LDL cholesterols, which, by the way, they tolerated very well without adverse effects. But you can appreciate in this graph that in those seven red lines to the left they are well below the LDL levels of their peers in Iceland and so this actually felt was a very interesting target and something that we actually built on in designing and developing our edit 401 molecule so beyond just its potential for efficacy obviously the preclinical safety profile is very important as it's going to inform our ability to move into humans and the doses at which we'll actually be able to explore. And the key takeaway here is that our safety observations at the therapeutically relevant doses demonstrate that it was very well tolerated with no adverse clinical observations. And while we did observe transient ALT elevations, they were very similar to the vehicle control. And these all resolved in less than a week. And we had no liver, absolutely no liver histopathology findings. This is a summation of our clinical or non-clinic experience from our non GLP animal experiments comprising about 45 animals but we have an ongoing GLP talks with an additional 30 or so animals 32 animals in fact but the chemistry while those studies are ongoing we do know that the liver function chemistry is consistent what we're demonstrating here they are GLP so I think there's a lot of confidence in actually moving this compound edit 401 into the clinic and And we're actually in a very good position today where, thanks to a very successful financing just two weeks ago, we're in a very good position, well capitalized, to execute against the strategy that's outlined here. And that is that we are on track to submit a CTN in the middle of this year in Australia. We will actually be dosing patients there this year, and we anticipate having early data from our first cohort by the end of the year. And then we will complete enrollment in 2027. We will complete the enrollment of the dose finding portion, I should say, of our phase one study. And we will have top line data results available in 2027. But as I say, the nice thing is, is now that, you know, sitting here at the back end of a financing driven by a lot of excitement in investors, having seen this data, is that we really have a potential to deliver a best in class therapy, which addresses the needs of a significant at-risk population and ultimately aligns with patients' needs and healthcare system needs. So I say best-in-class therapy with 401, significant at-risk. And I think very importantly, I just want to emphasize that as you articulated our strategy at the beginning, is that one of our goals was to make this accessible, easily usable. And so driving low-cost of goods and using or creating a therapeutic that can be used with a simple, single infusion really reduces the burden on patients and health care systems as we go forward. And with that, I would love to address any questions that you might have. Thanks very much. And if there are no questions, then I'm happy to close the session. That's okay. Great. Well, thank you very much. Thanks very much.