Katrine Bosley - Chief Executive Officer Mark Mullikin - Senior Director of Finance and Investor Relations Andrew Hack - Chief Financial Officer Sandra Glucksmann - Chief Operating Officer Vic Myer - Chief Technology Officer Charlie Albright - Chief Scientific Officer Gerry Cox - Chief Medical Officer Tim Hunt - Senior Vice President of Corporate Affairs.
Gena Wang - Jefferies Whitney Ijem - J.P. Morgan Mike King - JMP Securities.
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..
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..
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 strength of our intellectual property foundation, and it has profound implications for making CRISPR medicines. I’d like to put this ruling into a bit more context.
To quote on the patent office’s decision, “specifically, the evidence shows that the invention is such systems in eukaryotic cells would not have been obvious over the invention of CRISPR/Cas9 systems in any environment, including in prokaryotic cells or in-vitro.” In nature, CRISPR/Cas9 is a bacterial system, enabling the system to work in eukaryotic cells is the fundamental requirement for making medicines.
Notably, making medicines it's not defined for any particularly form or guide RNA or Cas9 enzyme, but rather is defined by the ability to make a CRISPR/Cas9 system work in eukaryotic cells. It’s an important distinction and it is what is encompassed by the patents included in our license from the Broad Institute.
The strength of our overall intellectual property position is built, both on our foundational patents used CRISPR/Cas9 eukaryotic cells, as well as our significantly expanded technology platform, which I’ll discuss shortly. With these two components, we believe our intellectual property position is unmatched by others in the field.
Beyond Cas9, we announced in December the addition of an exclusive global license to Cpf1, which is the other CRISPR nuclease system that’s been shown to work sufficiently and specifically in human cells. Cpf1 meaningfully advances our ability to develop CRISPR medicines.
It’s both highly efficient and specific at least comparable to Cas9 got any abilities, but it also has important distinctions from Cas9. First, it substantially expands the number of genomics sites we can target.
With both Cpf1 and advanced forms of Cas9, we can now reach 10-times more sites in the genome as compared to most commonly used for Cas9 alone. Second, Cpf1 has a guide RNA, which is a shorter than Cas9, and that potentially makes it easier to manufacture and deliver.
And finally, Cpf1 potentially increase the efficiency and accuracy for some forms of gene repair, because it makes a staggered cut to the DNA, which is different from Cas9 cuts. And in addition, the intellectual property for Cpf1 is completely independent of the intellectual property for Cas9s.
Turning then to our scientific progress, our scientist have made a number of critical advancements in 2016.
I think about novel methods to create covalently-coupled dual guide RNAs distinct from approaches others have taken and our approach has the potential to improve manufacturing of CRISPR medicines and also provides opportunities for unique intellectual property.
In addition, our scientists developed a novel approach to characterize specificity, an approach that’s called you to talk. We believe, overall, we apply the most rigorous approach to analyzing the specificity of CRISPR medicines in the field. Shifting gears then to the pipeline.
In our program to treat Leber Congenital Amaurosis type 10 or LCA10, we have successfully edited cells in the retina of non-human primates in vivo. We accomplished this using sub-retinal delivery of our all-in-one AAV products configurations.
We are very excited by this important step towards the clinic, and we remain on track with this program to achieve the goal of filing an IND by the end of this year.
We also plan to initiate clinical and natural history study of LCA10 in the middle of this year, which will help us identify patients, as well as refine our clinical development strategy.
Our progress and allocation provides the fundamental building blocks through developing therapies for other diseases in the eye, as well as other systematically administered therapies. Turning to other products in our pipeline, we’ve also made significant progress in our ex-vivo editing programs in hematopoietic stem cells and also in T-cells.
We believe editing hematopoietic stem cells is a gateway to developing multiple medicines for diseases of the blood and bone-marrow, including sickle cell disease and immunological diseases. To develop medicines in this field, its critical to reconstitute the blood system with edited hematopoietic stem cells.
To accomplish this, the edited HSCs must engraft long-term and give rise to progeria cells that basically carry the therapeutic genetic edit. And that’s exactly what we show we can do with HSCs, and it positions us to develop a broad range of differentiated therapies with the strong and durable effect.
In other areas of the blood system editing, T-cells provide the foundation for products to treat a number of cancers, as well as immunological and infectious disease.
In our collaboration of Juno cancer, we successfully edited several targets and T-cells, including PD1 with greater than 90% efficiency and was not detective off-target activity in our specificity efforts.
In our wholly-owned T-cell work outside of cancer, we've achieved similar high levels of editing, and this provides the foundation for potential therapies for broad range of diseases. Overall, 2016 was a productive year of extending the platform in advancing the pipelines, we believe provides us with stronger going into 2017.
I would also like to comment on how we’re building the business for the long-term. Last year got off to a great start with our initial public offering in February, where we raised $109 million in gross proceeds. It's added to strong investor base that we have developed as a private company.
We also established important strategic alliances to advance the pipeline of medicines and treating disease with the ilium bone and the blood marrow through collaborations Adverum, and also with the San Raffaele Telethon Institute for Gene Therapy in Italy.
Our business development strategy will remain focused on forming collaborations that accelerate, enable and expand our pipeline. And finally, we are continuously focused on developing an outstanding organization.
We’ve made several important additions to our team in 2016, including the addition of Charlie Albright as our Chief Scientific Officer, Gerald Cox as our Chief Medical Officer and Tim Hunt as our Senior Vice President of Corporate Affairs.
We also added Vice Presidents to lead Preclinical Science, Technical Development and Manufacturing, Program and Alliance Management, Human Resources and Information Technology. I would also like to personally thank Xandra Glucksmann, our Chief Operating Officer, who will be moving on from Editas as the end of this month.
She has been an incredibly important part of these first few years of building Editas, and her talents in company formation are exceptional. Her contributions have been critical to building new organization to this point. She will be missed, but her impact to Editas’ success is long lasting.
The lifeblood of our Company is exceptional scientist and company builders, and we couldn’t be more pleased to the colleagues who joined us in 2016. With that, I would like to turn the call over to Andrew Hack for some remarks on our financial position..
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..
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 Instructions] And our first question comes from the line of Gena Wang with Jefferies. Your line is now open..
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?.
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..
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?.
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..
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..
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..
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?.
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..
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..
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?.
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..
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..
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?.
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..
Thank you. And we have a question from the line of Mike King with JMP Securities. Your line is now open..
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?.
We talked about this before. The enzyme that we’re using in that particular product configuration is staph aureus Cas9..
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?.
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..
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?.
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..
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..
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..
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..