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Editas Medicine, Inc. (EDIT)

Q4 2016 Earnings Call· Tue, Mar 7, 2017

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Transcript

Operator

Operator

Good afternoon. And welcome to the Editas Medicine’s Fourth Quarter and Full Year 2016 Conference Call. At this time, all participants are in listen-only mode. There will be a question-and-answer session at the end of this call. Please be advised that this call is being recorded at Editas’s request. I would now like to turn the call over to Editas team. Please proceed.

Mark Mullikin

Management

Good afternoon. This is Mark Mullikin, Senior Director of Finance and Investor Relations at Editas Medicine. Welcome to our fourth quarter and full year 2016 corporate update conference call. We issued a press release earlier this afternoon reviewing our fourth quarter and full year 2016 results and updates regarding the company, which will be covered on this call. A replay of today’s call will be available on the investors and media section of our Web site approximately 2 hours after its completion. After our prepared remarks, we will open up the call for Q&A. As a reminder, various remarks that we make during this call about the Company’s future expectations, plans and prospects constitute forward looking statements for purposes of the Safe Harbor provision under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the risk factors section of our most recent annual report on Form 10-K, which is on file with the SEC. In addition, any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to today. Now, let me turn the call over to our Chief Executive Officer, Katrine Bosley.

Katrine Bosley

Management

Thanks Mark. Good afternoon, everybody and thank you for joining us for our corporate update call for the fourth quarter and for the full year of 2016. I am joined today by several members of our executive team, including Andrew Hack, our Chief Financial Officer; Sandra Glucksmann, our Chief Operating Officer; Vic Myer, Chief Technology Officer; Charlie Albright, Chief Scientific Officer; Gerry Cox, Chief Medical Officer; and Tim Hunt, our Senior Vice President of Corporate Affairs. 2016 was a strong year for Editas Medicine and 2017 is shaping up to be a transformative year. On this call, we will review the most important elements of the progress we’ve made and layout our key goals for 2017. As a Company, we have three strategic priorities; first, to rapidly advance our pipeline and extend our technology leadership; second, to build a business for the long-term; and third, to develop an outstanding organization. So first, let’s discuss how we’ve extended our technology leadership in a number of different dimensions. As part of our intellectual property foundation, we have an exclusive license for human therapeutics from the Broad Institute for a portfolio of CRISPR/Cas9 patents and patent application. This portfolio includes patents that were issued to the Broad Institute to cover the use of CRISPR/Cas9 to edit DNA and human cells. A little over a year ago, the U.S. Patent and Trademark Office declared an interference proceeding for several of these issued patents. This proceeding was initiated to determine who first invented the use of CRISPR/Cas9 to edit DNA prokaryoticcells or cells that have nuclease. We are extremely pleased with the patent office’s recent ruling of no interference, in fact, in this interference proceeding. This ruling ends the interference and it upholds Broad’s patent. This is a highly favorable decision and it reaffirms the…

Andrew Hack

Management

Thanks Katrine. As you may have seen, we filed our 2016 Form 10-K late last week and have summarized our financials in the press release that we made available roughly an hour ago. As we've done on previous calls, I will review the most important components of our financials, and won't be walking through all of the detailed results that can be found in our 10-K and in our press release. Our net cash used in operations in 2016 was approximately $50 million, and our spending on capital equipment was roughly $3.5 million. Key non-cash items recorded in our income statement include; roughly $16 million of stocked-based compensation; a $10 million issuance of notes payable; and roughly $12 million of accrued expenses. Over the course of the year, we increased the size of our organization by roughly 50% from a bit under 60 team members at the close of 2015 to nearly 90 at the end of last year. Our team advanced all of our programs successfully in ‘16, including the critical achievement of gene editing in the retina and non-human primates. In addition, we consolidated our operations into a single new facility in the fourth quarter, providing us with an outstanding home to develop and discover important medicines for many years to come. Taking all of this into account, the primary drivers of our growth and spending in 2016, were, our expanding pipeline, advances in our platform, and legal expenses; and we expect this to continue to be the primary drivers in 2017. As of December 31, 2016, we had approximately $185 million of cash and cash equivalents. Based on our year-end cash position as well as research support under our collaborations, we believe we have at least 18 months of capital to fund the advancement of multiple therapeutic programs in parallel, and to further extend our technology leadership. And with, I’ll hand it back to Katrine.

Katrine Bosley

Management

Thanks, Andrew. 2016 was clearly strong year for Editas, and I’d like to comment briefly on our goals for 2017 as well. As we noted a few minutes ago, in our lead pipeline program for LCA10, we plan to initiate a clinical natural history study in the middle of this year and our goal is to submit our IND to the FDA by the end of this year for that program. Next, we are working to achieve pre-clinical proof-of-concept for additional programs and to disclose data from these programs publically. We’re also pursuing focused partnerships and alliances that will help us accelerate, expand and enable our pipeline of platform. And finally, we’ll work to continue to build an outstanding organization culture to attract the best and brightest people to pioneer across the transformative genomic medicines. So thank you for your interest and support. And with that, we’d like to open up to questions and answers. Operator?

Operator

Operator

[Operator Instructions] And our first question comes from the line of Gena Wang with Jefferies. Your line is now open.

Gena Wang

Analyst

Thank you very much for taking my question. The first one is to add my [indiscernible] on the leasing progress, especially the IP part. So, my first question is, you mentioned that you will participate in three upcoming scientific and medical conferences. Should we expect announcement [indiscernible] data to be reported in one of the meetings?

Katrine Bosley

Management

Thanks Gena. We haven’t disclosed exactly where we first plan to presents the data and/or publish the data. But we definitely appreciate everybody’s interest in it, and that is our goal to present it this year. Certainly, for all of these meetings as you know the abstracts get published certain amount of time in advance. And so, we’ll make sure that folks understand when we are planning to disclose it when that becomes public, and that we definitely appreciate or even as we see, we certainly will present it.

Gena Wang

Analyst

And my second question is regarding the Cpf1. I think clinical development early for both Cas9 and Cpf1. How would you plan moving forward with these two systems or clinical programs?

Katrine Bosley

Management

So, I’ll comment on this briefly, and then I’ll also ask Vic Myer, Chief Technology Officer, to comment. Because for any given program our goal is to make the best medicine, make the best molecule. And so both Cpf1 and Cas9 afford very compelling opportunities. And Vic can comment on how we’re working with both of them through our platform.

Vic Myer

Analyst

Yes, I think as Katrine mentioned in the overview piece, we see these two systems as very complementary to one another. As we mentioned, the Cpf1 is a really interesting enzyme. It increases the number of sites that we can cut within the human genome quite significantly. I think with Cpf1 in all of our other licensed variance, we're now able to cut it 10-times the number of size you can cut with the standard, if you will, as [indiscernible] enzyme. Second, it's got a single short guide RNA that directs the protein to its previous site. So this short guide RNA should provide some advances on the manufacturing and delivery side. And finally it leads to staggered cut, which engenders a different cellular response than a blunt cut. So it's going to potentially more useful for achieving different kinds of repair outcomes.

Gena Wang

Analyst

Thank you. And if I may just please my last question. For the LCA10 program, just wondering if you can walk us through the steps you need to achieve in order to file IND by the end of this year? Thank you.

Katrine Bosley

Management

Sure, I'll comment on that. And then Gerry Cox, our Chief Medical Officer, can also comment. In many respects, it's all the normal pre-clinical work that any program has to complete preclinical safety, to complete all of the manufacturing work. And so in some respects, there is nothing deeply differently than -- for example with gene therapy program, we have to do all of the appropriate CMC work, as well as preclinical work. Gerry, do you add on the preclinical perquisite?

Gerry Cox

Analyst

Sure. I’ll just add to that a bit. The genome editing certainly we want to make sure that we're cutting at the appropriate on-target size and not at off target site, so that’s part of the development program. We’re also initiating a natural history study this year to better characterize patients that may benefit from treatment and to make the clinical trials more efficient.

Operator

Operator

Thank you. And our next question comes from the line of Mike King with JMP Securities. Your line is now open. Due to no response we’ll go to the next question [Operator Instructions]. And our next question comes from the line of Cory Kasimov with J.P. Morgan. Your line is now open.

Whitney Ijem

Analyst · J.P. Morgan. Your line is now open.

This is Whitney on for Cory. Just wanted to ask a G&A question, and how we should be thinking about in 2017. I guess, post the PTAP decision, just given that presumably, some of the legal spend will be wrapping up, but then also there’s potential for the other site to file an appeal. So, any color on how we should be thinking about that?

Katrine Bosley

Management

We view intellectual property as an investment. We're building this company for the long-term, and there is a broad portfolio that we're investing in, that includes some of the things that you’ve mentioned with regard to, obviously historically, there has been the interference of this past year. But there is a broad portfolio and the prosecution of that portfolio is critical component of that spend as well. Andrew, do you want to comment on how we're thinking about that going forward to do that externally.

Andrew Hack

Management

There’re a wide ranges of scenarios, so I can't comment in more detail on what we're anticipating. Spending and investment, more broadly, and IP to look like going forward. But IP is a critical asset to the Company. And so we’ve resourced appropriately for the investment, and we feel like it's provided really strong return investment with the success that has come-to-date and we look forward to continue to invest in that.

Whitney Ijem

Analyst · J.P. Morgan. Your line is now open.

And then just looking beyond LCA10, you mentioned this year you hope to achieve some preclinical POC for additional programs. So, you’re expecting that we could, or you could, I guess kind of determine the next program this year, or how should be thinking about when we could learn more about Part D?

Katrine Bosley

Management

Yes. We definitely appreciate that are eager for us to begin to articulate timeline for additional programs, and we look forward to reaching a point where we do that. We haven’t done that just yet externally, but we certainly have consistently presented data on a regular basis and we’ll continue to do that. As you look at the data, we’re focusing in on a bit more the ex-vivo work that you see us discussing here and in number of our scientific presentations with regard to both hematopoietic stem cells and T-cells; is really important area where we’ve made significant progress. We’re working on all the programs that we articulated in our pipeline. But certainly those are programs where I think that we’ll see the progress emerging mix. And there is number of different specific programs within the capabilities of editing those two different cell types. So, specific programs emerge, we’ll certainly keep everybody posted.

Operator

Operator

Thank you. And we have a question from the line of Mike King with JMP Securities. Your line is now open.

Mike King

Analyst

I was asking if you guys can confirm or not confirm whether Cas9 is going to be the enzyme in the LCA10 program, Cpf1 or would you either not specify it?

Katrine Bosley

Management

We talked about this before. The enzyme that we’re using in that particular product configuration is staph aureus Cas9.

Mike King

Analyst

And I know you were asked a question about Cpf1. But just wondering if -- do you have any estimated timeline to one that’s going to be clinic ready?

Katrine Bosley

Management

Maybe slightly different way to think about it is that it's part of our platform at this point. We only announced the deal in December. But obviously, we’ve been working with an understanding at making sure we had familiarity with it; in advance of that, so that as we brought it onboard and then announced the license; it's part and parcel of that platform, at this point. So for any given program we’re working on, it’s really a question of what’s the best way to solve that target. And there may be targets where you could use either Cas9 or Cpf1 there may be targets where one is better, the others is better. Overall, having both of them gives us a much broader and deeper platform. I believe we really want to be data driven in defining for any given therapeutic target with that approach. We’ll try both molecules and make sure we’re finding the best solution based on empirical data.

Mike King

Analyst

And then if I can just ask a quick question about IP, again I understand the sensitivity about commenting in public about it. Obviously, you see was not happy with the outcome on their part they plan to appeal to have decision. They’re also upbeat about their ability to prosecute their IP. And if we want to use Jennifer Doudna’s metaphor of tennis balls, is if they granted IP claim on single guide, is that somehow -- is that an eventuality you guys are prepared for, and how would you view that under those circumstances?

Katrine Bosley

Management

We certainly appreciate people are thinking about a lot of different theoretical future scenarios. But there is couple of things; one things that’s clear is that the growth patents have been uphold. There are number of patents, multiple claims, in those patents that have broad implications for the field. And just it gets worse reiterating that making a CRISPR/Cas9 based medicine is not defined by a particular form of guide RNA or particular form of an enzyme, it's really defined and underpinned by the ability to make the CRISPR/Cas9 system work in eukaryotic cells. So that’s the fundamental requirement to make the medicine. Obviously, we can’t speculate on what the other parties may or may not choose to do, going forward. Certainly, we’ll continue to invest in our portfolio. There is lot of things one could speculate about but we’ll be prepared for all those scenarios. As we said ever since we’ve founded the Company, we want to make sure we’re building Company for the long-term. And part of that means that, from a business standpoint, we have a path and a strategy for it that’s strong and works in every scenario. Certainly, some scenarios are more favorable than others, and we’re very pleased to be in those scenarios where these patents have been upheld because we think they are really important. But we're prepared for every scenario, because just part of it builds in the business for the long-term.

Operator

Operator

Thank you. And I'm not showing any further questions, at this time. I would now like to turn the call back to Katrine Bosley, Chief Executive Officer for any closing remarks.

Katrine Bosley

Management

Great. Thank you. And with that we thank you all for your participating in the call today. And also for your support as we build the Company and we work to bring new medicines for to patients. Have a great evening.

Operator

Operator

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program, and you may all disconnect. Everyone, have a great day.