EDIT - Editas Medicine Inc. (EDIT) 6th Annual Evercore ISI HealthCONx Conference (Transcript)
2023-11-28 14:36:05 ET
Editas Medicine, Inc. (EDIT)
6th Annual Evercore ISI HealthCONx Conference
November 28, 2023 1:20 PM ET
Company Participants
Erick Lucera - CFO
Cristi Barnett - Head, Corporate Communications & IR
Conference Call Participants
Liisa Bayko - Evercore ISI
Presentation
Liisa Bayko
Thanks for joining us today for a fireside chat with Editas. My name is Liisa Bayko. I'm a biotech analyst at Evercore ISI, and I'm joined by the team here at Editas. So I'll just turn over. You can do some brief introductions and we'll then just drive right in.
Erick Lucera
Hi. My name is Erick Lucera. I'm the CFO. Happy to be here, and thank you for your support both on the research and banking side.
Cristi Barnett
And I'm Cristi Barnett, Head of Corporate Communications and Investor Relations. I'm also happy to be here. Thank you.
Liisa Bayko
Okay. Wonderful. Let's start off with just a one minute overview of Editas.
Erick Lucera
Yes. So I'll take that. So Editas is a gene editing company. We're actually 10 years old this week, this month and our focus is on editing for cures. We have one lead product in the clinic for sickle cell disease, and we've been releasing data at major medical meetings this year, and looking forward to providing another update on that data in a few weeks.
Question-and-Answer Session
Q - Liisa Bayko
Great. So tell us a little bit more about, kind of -- let, we'll start off with sickle cell. I know you got a pipeline behind that, but what is your approach to sickle cell that might be different from what might be, I think, the first, genome edited medicine to be approved, which is, Vertex and CRISPR's Exa-cel? How are the two different?
Erick Lucera
Well, obviously, from a 30,000 foot view, we're really excited about the progress that Vertex is making, not only for the patients but also for the field of gene editing. But as you mentioned, there are a few differences between our approach and their approach. The first is they're using Cas9, and we're using Cas12a, so it's a different enzyme. And we're targeting the gamma globulin promoter, which we believe, based on our research, it leads to a little bit better hemoglobin data. So we think that'll be a point of differentiation that we'll see play out over time, and we're seeing it already, so.
Liisa Bayko
Great. And are you -- how -- you have intellectual property rights to Cas9. Is that correct?
Erick Lucera
Yes.
Liisa Bayko
And then how are you thinking about, I guess your kind of approach when Exa-cel does get approved, curious how you think about it?
Erick Lucera
Yes. Well, I'd say, we can't comment on any specific program or product or company in terms of discussions, but we can talk broadly. We think that, that we have a foundational -- patents for both Cas9 and Cas12. We've got great intellectual property estate, licensed from Harvard, MIT, and the Broad. And we are open and to anyone that wants to talk to us about getting a license, we'll leave it at that.
Liisa Bayko
Okay. So EDIT-301, which you just alluded to, your lead program, we've got the ASH meeting coming up. What can we expect to hear from Editas at the ASH meeting?
Erick Lucera
Maybe I'll put in a little bit of context and then I'll turn it over to Cristi. But as you know, in the beginning of the year, we had data on one patient and that's what I looked at data on 4 patients at EHA of last year. And as you know, we designed this to have -- and we announced in the beginning of the year that we would dose 20 patients this year that will slip a little bit into January, but the intent was based on the experience of Gilmore and Baisong, who have each brought a total of 10 products to market in this area, we thought that would be a good package of data to have that was validated by what we saw from Vertex this summer. So, we are going from the 4 patients at EHA to a nice data set at ASH, and we're also going to have an analyst meeting that day as well, we'll have a little bit more data. I'll turn it over to Cristi to go over some of the specifics.
Cristi Barnett
Sure. Yes, so at ASH and in our analyst meeting, we'll have 11 patients worth of data from RUBY, so that's the sickle cell trial. 2 of those patients will be out past the year, which is great long-term follow-up. Another 4 will be past 5 months, which is important because that's where we really think we're starting see the differentiation play out. And then we're also going to be showing 6 patients from EdiTHAL which is the beta thalassemia trial as well, 2 of those past 5 months. So, should be a nice kind of 17 patients worth of data that we're sharing and hoping to see similar to what we showed this summer and kind of following that same trajectory.
Liisa Bayko
A lot of people who have been following this space, especially from the Vertex perspective, sort of wonder about the market size. I mean, clearly, the approach is medically a huge transformative event for the patients, but it is a big process to undergo, and it's also probably going to be pretty expensive, I would imagine. So, people kind of wonder about the market opportunity. Can you kind of walk us through, like, what are your thoughts on what the size of the market opportunity is? And sort of how do you get there? What are some of your key assumptions?
Erick Lucera
Obviously, there's a prevalence pool of about 20,000 patients, and we don't yet know what ones will be amenable to this kind of procedure, have ability to get -- to endure it or to get even reimbursement for that, so that aside, we do see value in this procedure or there's, I think, the entry criteria for our trial is two plus vaso-occlusive events a year. We have some patients that have had 100 days a year in the hospital and when you think about that over a lifespan, a shortened lifespan, that adds up pretty quickly. So, I think you see from a pharmacoeconomic standpoint, a pretty good justification for whatever prices get put out in the marketplace, and it's obviously -- we all know it's a terrible disease, and I think what you'll see is, as patients see different therapies out there approved and start hearing stories about how people's lives are transformed by this, I think once it's able to be marketed and these stories get out there, you'll start seeing people raising their hand and saying, yes, I will do this.
Liisa Bayko
What have you heard on how like different pricing arrangements? And I'm not talking about the total value, but the way we've been thinking about it in our model is, like, payments over time, kind of equal installments over 5 years. We just kind of use a very simple approach. Is that, like, the right way to think about it? Or are there other models that make sense to you?
Erick Lucera
I think it's the type of thing -- I mean, look, one of the beauties of living in a country like this is that you can have creative approaches to financing, and that approach you mentioned would certainly be something that I think people are going to be looking for flexibility.
Liisa Bayko
What are you doing on kind of the whole concept of moving towards gentler conditioning regimens, because that's a big -- that's one of the things that I think probably is a little bit of a limiting part of the equation for how big this market can be. So what are you doing on that front?
Cristi Barnett
Obviously, yes, getting to a milder conditioning is one thing that we're looking into, whether that's something that we bring in-house or whatnot. Another thing that we're also working on that we've talked about is also can you bring this medicine into an in vivo approach as well, which would also be a much less burdensome process for the patient as well. So, that is one thing that we're working on as well. And we've already solved for 2 of the problems. We have the target, we have the enzyme. So, working on the last piece of the puzzle, which is the delivery part.
Liisa Bayko
So what's the depletion, like, we hear our -- the gentler conditioning regimen, we hear a lot of talk about this, but I haven't seen a lot of tangible progress. Like, where are we really at? What's the latest kind of developments in that field? I know there's companies beyond just, like the players in the actual, like, gene editing space that are working on that, and then I think you guys are probably working on it yourselves as well. I know CRISPR, Vertex has their own efforts there too. What is the sort of latest and greatest there? And is that -- when do you expect it? Do you think in vivo will come before that or like what makes it over the hump first?
Cristi Barnett
That I don't know for -- to speculate on, but what I do know is that right now, since we're working on the development of 301, you kind of don't want to couple 2 new things together in a way. So we're developing 301 kind of as is, as the model as is, while simultaneously working on in vivo. And then, again, just looking at the field in terms of what is there in terms of milder conditioning that could be out there for the future.
Liisa Bayko
And what's next for the program, so you've got the upcoming data at ASH, and then what should you expect after that? How quickly do you think you'll have enough patients to maybe file for approval? Kind of what year use -- what are your sight lines for that, I'm curious?
Erick Lucera
Yes, we think that 2024 is going to be a great year for the company, and we're going to have more data from the 301 trial, if you think about last year at EHA having 4 patients and now having data on 11. We're going to continue to report out more data to the investment community and the physicians and the patients at a regular cadence. And I think as you think about getting towards a midyear data set, as we mentioned on the last call, we'll have 20 patients dosed by January. As you can see from the data in our corporate deck, we get most of the editing done by 3 to 5 months, which is when you see the correction of the anemia and the fetal hemoglobin levels. So we think that by the time you get to the middle of next year, you're going to have a very robust data set of those 20 patients with enough follow-up to see how well the drug is working.
In terms of going from there, we've continued to guide people to the data set that Vertex produced, so they had 17 patients at 18 months follow-up, so if you roll from midyear next year, another 12 months, we'll have a BLA-ready data set and we'll see how things go. As you know, we have RMAT designation, so that's going to give us a lot of flexibility with respect to having discussions with the FDA and we'll learn more about what our final package needs to be.
So I think next year is going to be a great year from a 301 standpoint. And also from a in vivo standpoint, we are looking for, some sort of delivery technology and we expect to have more on that in the coming year.
And then finally, the third pillar of our story, the Cas9, Cas12 licenses, we think there's a lot of companies out there that are probably needing to have discussions with us, and we look forward to having those in coming months and quarters.
Liisa Bayko
What do you think that, that could be worth, that piece of the equation?
Erick Lucera
It's hard to put a dollar value on it. I think as you look at all the companies that are pursuing CRISPR gene editing programs, whether it's a Cas9 or Cas12, they're all in various stages of development, some are early on. And I think -- I'm not talking about our product in particular, but earlier stage bio book deals that generally have a different structure than what you see for later stage companies where certainty is higher and perhaps earlier milestones may have been missed for lack of a better term. So I think there's a range of opportunities that exist out there.
Liisa Bayko
What kind of a royalty arrangement makes sense? What are some benchmarks, I guess, you could point to? Like, not what you're thinking, but just others that could happen?
Erick Lucera
Yes. I mean, obviously, we have the deal with Bristol-Myers which is out there where we announced a deal with Vor, and I think each one of those is a combination of the prototypical upfronts, development milestones and royalties, so I would imagine that this would take a similar format.
Liisa Bayko
Okay. As for your in vivo program, what's the timing on kind of, I guess, getting into clinic on that one, and when should we expect to hear more about that?
Erick Lucera
You know, we haven't really commented yet on that. Obviously, we need to get -- make a decision on which way we want to go with respect to the delivery technology. The team is working their way of trying to identify the targets. And I think as those activities are going in parallel, we'll have more visibility in the year and be able within 2024, and we'll be able to lay out a development timeline from there.
Liisa Bayko
Because that just seems really transformative I think.
Erick Lucera
Yes, I think for the autologous cell therapies, which we all know are sort of one and done, the ability to transform to a in vivo medicine, which not only is less burdensome on the patients, the caregivers, and their families, but allows you then to expand to a broader patient pool. I think it's totally transformative.
Liisa Bayko
And when you look at the kind of competitive landscape, there used to be a lot of players going after sort of ex vivo sickle cell, and beta thalassemia treatments. And then seems to have been some have gone by the wayside. Who do you think about as your sort of key competitors out there? And I know what you're differentiating on hemoglobin, do you see anyone else with any interesting, I don't know, points of a differentiation?
Cristi Barnett
I mean, ultimately, it's good for patients to have lots of things in development, lots of choices, lots of companies working to solve these diseases that have no treatment. So, I think if we can all get there, that's a win for everybody. And then back to the point on the IP, if that is something that is -- we have the foundational IP, then that also is a win for us, so.
Liisa Bayko
On the foundational IP front as you think about how you might approach that, what are some like benchmarks out there? Or kind of like what -- if people want to like kind of model that for you guys as part of your value stream, what would you point to other companies that have had foundational IP and have been able to extract some value from that?
Erick Lucera
Yes. I think when you think about foundational IP and licensing to multiple companies, you're generally talking about non-exclusive licenses. And when you think about business development and licenses of IP-only for an FTO, freedom to operate standpoint versus licensing a product with data and kilograms of material in a manufacturing process, they're completely different. And I think you can see over the history of biotech, there have been several examples of situations where there's been those companies that have licensed to dozens of companies their IP, whether it's the [Diax] or the Cabilly patents and things like that, versus sort of the Vertex, CRISPR deal, which is really a product license and an exclusive license. So I would say, we're generally -- if you're going to license any product to more than one person, it's obviously nonexclusive, so it's a different spectrum.
Liisa Bayko
And just in the last kind of minute or so we have, maybe you can talk about your cash runway and then kind of catalysts that we could expect, and as we turn the clock into the New Year?
Erick Lucera
Yes. We ended the last quarter with $444 million of cash, and we stated at the time that, that gets the end of the third quarter of '25. As I mentioned, we view the license as a potential source of non-dilutive capital, which may help with our fundraising efforts.
In terms of catalyst for next year, we think next year is going to be a great year for the company. Obviously, we're going to continue to show more progress on 301 with getting a wholesome, a really nice data set by the middle of the year.
We're going to have progress on in vivo with some sort of delivery technology put in place and then sort of go from there towards proof of concept, and then with respect to the Cas9, Cas12 licenses, we're going to be open for business and look forward to talking to as many people as want to come by.
Liisa Bayko
Okay, great. Well, thank you.
Erick Lucera
Thank you.
Cristi Barnett
Thank you.
Erick Lucera
Thanks again.
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Editas Medicine, Inc. (EDIT) 6th Annual Evercore ISI HealthCONx Conference (Transcript)