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Healthcare - Biotechnology - NASDAQ - US
$ 2.66
-3.97 %
$ 220 M
Market Cap
-1.05
P/E
EARNINGS CALL TRANSCRIPT
EARNINGS CALL TRANSCRIPT 2017 - Q1
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Executives

Mark Mullikin - Senior Director of Finance and Investor Relations Katrine Bosley - Chief Executive Officer Charlie Albright - Chief Scientific Officer Andrew Hack - Chief Financial Officer Vic Myer - Chief Technology Officer Gerry Cox - Chief Medical Officer.

Analysts

Whitney Ijem - JP Morgan Cyrus Amoozegar - Morgan Stanley Phil Nadeau - Cowen & Co Mike King - JMP Securities Madhu Kumar - Chardan.

Operator

Good afternoon and welcome to the Editas Medicine’s First Quarter 2017 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 Medicine’s request.

I would now like to turn the call over to the Editas Medicine team. Please proceed..

Mark Mullikin

Good afternoon. This is Mark Mullikin, Senior Director of Finance and Investor Relations at Editas Medicine. Welcome to our first quarter 2017 corporate update conference call. We issued a press release earlier this afternoon reviewing our first quarter 2017 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 website approximately two hours after its completion. After our prepared remarks, we will open 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 quarterly report on Form 10-Q, 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, I will turn the call over to our Chief Executive Officer, Katrine Bosley..

Katrine Bosley

Thanks Mark. Good afternoon, everybody, and thank you for joining us for our corporate update call for the first quarter of 2017.

I am joined today by number of members of our executive team, including Andrew Hack, our Chief Financial 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. The first quarter of 2017 was a big quarter for Editas.

We established a major strategic alliance with Allergan. We had a very significant positive decision from the U.S. Patent Office. We’ve raised a substantial capital in a following offering. And we meaningfully advanced both our pipeline and our platform.

With all of these achievements, we are well position to advance our product pipeline while continuing to expand our platform leadership. I'd like to start first by taking you through building business with the Allergan alliance as well as the progress in our LCA10 program.

We entered the strategic alliance with Allergan to develop and commercialize genomic medicines for patients suffering from serious eye diseases. As part of this option and alliance agreement, we received the $90 million upfront cash payment.

And for each program options by Allergan, we have the potential to receive over $200 million in milestone with more than $100 million of that in milestones for development, approval and launch event.

We are also eligible to receive royalties on net sales of optioned products with Editas retaining the option to co-develop and co-commercialize the LCA10 program plus one other program in the United States with equal profit sharing for these programs.

Over the initial seven year research term of the agreement, we’ll pursue development of a wide range of ocular medicines in collaboration with Allergan. And Allergan has an exclusive option to license up to five of these programs including a program to treat LCA10.

It's an exciting line and it's very much in line with articulated business development strategy and we expect will create substantial value for patients, as well as our shareholders that bring together unparalleled crisper platform with Allergan ophthalmology development and commercial expertise.

The program itself continues to advance well, and Charlie will tell you more about the Data for Program in a moment. Importantly, we have decided to make an adjustment to the timeline on this program and there are two primary reasons.

The first is to accommodate certain delays in third party manufacturing and the other is to take full advantage of our newly formed collaboration with alliance with Allergan. Based on this, we decided to move the target IND filing date for this program to mid-2018.

Now we realize this is a shift, but we do think it's the right decision for the program. The manufacturing delay related to production of input materials for AAV manufacturing. This caused us to have to shift to a latest slot at our AAV contract manufacturing site and collectively this pushed out the timeline as I've described.

Importantly, there were no crisp or specific issues that caused this delay. And although no delay is ever good thing, we understand that this is an important event. It does create a window opportunity to corporate elements of Allergan’s ophthalmology pre-clinical development and manufacturing expertise into the program.

Despite this manufacturing delay, we do remain on track to start a clinical natural history study of the LCA10 patients in the middle of this year. And in addition, we presented data from our non-human primate study at the ASGCT Scientific Conference last week demonstrating up to 50% editing in the real in photoreceptors in non-human primates.

And overall a competence in the program does remain high. The second key aspect in the first quarter of this year was advances in our intellectual property foundation. In addition to the Allergan alliance, in the first quarter, we strengthened our unmatched intellectual property position around CRISPR Cas9 with the U.S.

Patent Trial and Appeal Board highly favorable ruling upholding the Broad Institute Foundational Patent claims. In addition the CRISPR Cpf1 patent portfolio is also advancing.

The European Patent Office recently issued a notice of intent to grant to the Broad Institute very broad coverage of CRISPR Cpf1 system for human gene editing, and this has been exclusively licensed to Editas Medicine.

We're the only company with access to foundational patents covering both Cas9 and Cpf1 as well as advanced forms of each CRISPR system. Taking together, our platform has the potential to deliver the best and widest range of CRISPR medicines. And third, deepening our capital foundation.

In the first quarter, we executed a follow-on offering of primary shares of common stock expanding our investor base and raising $104 million including the full exercise of the underwriters over allotment.

Collectively, the combination of the upfront payment from Allergan and the proceeds from the following stock offering roughly doubled our cash run rate and Andrew will talk more about that later in the call. All in all, it was a very productive quarter for building the company.

And now to take you through progress on the pipeline and platform, I’ll hand the call over to Charlie Albright, our Chief Scientific Officer..

Charlie Albright

Thanks Katrine. The first quarter of 2017 was a very productive one in terms of advancing our pipeline and extending our technology leadership.

We just completed a successful meeting the American Society of Gene and Cell Therapy or ASGCT where our scientists delivered six presentations, which showed our progress and translating the promise of CRISPR medicine into the best and broadest class of genomic medicines.

In our LCA10, we demonstrated efficient editing of CEP290 gene in the retina of non-human primates. While others have shown editing photoreceptors in mice, it was very important to show efficient editing in non-human primate for the receptors, so non-human primate eyes are much more similar to human eyes than are the eyes of mice.

In this non-human primate study, we injected in the sub-retinal space and add no associated virus that expressed the two guide RNAs and Cas9 using a photoreceptor specific promoter. Six and thirteen weeks after this injection, we were removed a piece of the retinal tissue that was exposed to the virus and quantified editing.

This analysis showed that we had productively edited 15% of CEP290 alleles in these samples. Now we know that 15% is an underestimate of editing alleles photoreceptors since the editing machinery was only expressed in photoreceptors and they only comprised about a core of the DNA in these samples.

Consequently, we estimate that we productively edited 50% of the CEP290 alleles in these non-human primate photoreceptors, which significantly exceeds our therapeutic target of 10%. Taken together, these results are really a big deal and support our continued development of our experimental medicine for this devastating disease.

Turning to blood diseases, we presented preclinical data for our program to treat sickle cell disease and beta-thalassemia. We showed that we can induce therapeutically relevant levels of fetal hemoglobin protein using an editing strategy that targets novel sites in the globin locus.

We believe this editing strategy has a potential that will be more potent than what has been previously described by others. In these experiments, we also successfully and durably repopulated the blood system in mice with these edited cells, a critical achievement towards establishing a robust experimental medicine.

Taken together these data give us additional confidence that we are on track to develop what we believe will be a differentiated and superior genomic medicine for patients suffering from hemoglobinopathies. Our team also continues to extend our CRISPR technology leadership in a range of unique dimensions.

At the ASGCT meeting, we presented the progress we made on self-inactivating CRISPR systems. We’ll believe these systems will enable us to tightly control Cas9 levels, while maintaining efficacy when using viral delivery methods. This is important since viral delivery methods are currently the best option for some important diseases.

Finally at both the ASGCT and Keystone Genome Editing meetings, we showed data from our research program to develop synthetic covalently dual guide RNAs for Cas9. Guide RNAs for Cas9 are typically more than 100 nucleotides long.

This length presents challenges for current synthetic methods to produce guides that are pure, in particular the sequences the five prime of the guide RNAs are more prone to synthesis errors, but this is the region of the guide RNA the binds target sight of the genomic DNA.

Hence impurities in the guide RNAs due to synthesis errors could lead to undesired off target cutting. To overcome this limitation, we synthesized two shorter RNAs and covalently-coupled these molecules to form a novel kind of guide RNA.

And importantly, we show that these covalently-coupled dual guide RNAs have greater sequence fidelity than the current synthetic guide RNAs. For these reasons, we believe these covalently-coupled dual guides have the potential to set a new standard for manufacturing purity, accuracy and scalability.

All of these advances in self-inactivating nucleases, novel guide RNA designs and other core components of our platform are differentiated proprietary to Editas and they move us well beyond the initial technologies of the mercenary early days of this field.

We will continue to invest in our platforms to solve these kinds of translational medicine challenges and thereby support the rapid and successful advancement of our pipeline. And so with that, I'd like to reiterate what a strong quarter this was for Editas Medicine from a scientific perspective and how excited we are to share our progress with you.

Now I hand it over to Andrew to review the financial that reported today, as well as the impact of our Allergan alliance will have on there our numbers going forward..

Andrew Hack

Great, thanks, Charlie. We filed our Form 10-Q after the close today, and I summarized our financial statements in the press release that we made available roughly an hour ago. In the first quarter of 2017, our cash and cash equivalents increased by approximately $166 million.

Net cash provided by operations was approximately $70 million and our spending on capital equipment was less than $1 million. And cash flow from operations including the $90 million upfront payment from Allergan related to our strategic alliance and auction agreement, which has accrued is deferred revenue.

This revenue will be recognized approximately linearly over the seven year term of the research agreement. Key non-cash items recorded in our income statement include roughly $6 million of stock based compensation and roughly $11 million of accrued expenses.

Net cash provided by financing activities includes approximately $97 million of net proceeds from our follow-on stock offering a $4.6 million shares. As a result of this offering, we had a total of approximately $41.3 million common shares outstanding as of March 31st.

We ended the quarter with approximately $352 million of cash and cash equivalents on the balance sheet.

Based on our current cash position, as well as research support under our collaborations, but excluding any assumption for future business development transactions or milestones, we believe we have at least 24 months of capital to fund the advance in multiple therapeutic programs in parallel and to further extent our technology leadership.

And with that I’ll hand it back to Katrine..

Katrine Bosley

Thanks Andrew. Before we wrap up, we also wanted to mention a new initiative just announced a few hours ago. Today, we are very pleased to share the news that Editas has joined the Duke University Margolis Center Value-Based Payment Consortium. This initiative has led by Dr. Mark McClellan of Duke University.

He's the former administrator of CMS, Centers for Medicare and Medicaid Services, as well as the former commissioner of the FDA; and he's a widely acknowledged respected leader in healthcare innovation, public policy and economics.

The distinguished advisory group brings together experts in regulatory affairs and policy with leaders from payers, healthcare providers, patient advocacy and biopharmaceutical and medical device industry. The group will work to create practical solutions to address common legal and regulatory issues and to develop and pilot novel payment models.

This initiative is an important new pillar of our long term strategy to build Editas Medicine. And we look forward to pioneer in this arena just as for pioneering CRISPR technology. So thank you for all your interest, your support. And with that we will open it up to Q&A.

Operator?.

Operator

Thank you. [Operator Instructions] And our first question is from the line of Cory Kasimov with JP Morgan. Your line is now open..

Whitney Ijem

I guys, this is Whitney on for Corey.

First question, just wanted to follow-up on the timeline delay, and I think you mentioned in addition to the manufacturing AAV related things that you also wanted to take advantage of Allergan’s preclinical expertise, so just wondering, if you can give us any more information on what that means and if anything that might come out of that additional work could lead to potentially changes in the program or any additional delays?.

Katrine Bosley

One of the key reasons we wanted to work with Allergan is their deep expertise in ophthalmology. And as with any partner you want to kind of bring them into the same speed the program is moving. And so they want to see this move quickly and as we do the insights driven in that progress. So certainly the manufacturing delay affected the timeline.

As we described that gives us the opportunity to take full advantage of any further input that Allergan may have in addition to the plan we already have in place. But really that just bringing our partner fully on board and building from the starting point which was a fully diligence when we launched the alliance..

Whitney Ijem

Okay.

And then just on the interference proceeding, I think do you see parties collected have announced intent to appeal, so can you - I guess talk us through the next step and timelines of that?.

Katrine Bosley

So the appeal by - you see was not surprising, we were anticipating that might well happen. I mean certainly the PTAP decision was a very strong decision. I’ll ask Andrew to touch on at a high level of the timeline going forward..

Andrew Hack

Hi Whitney. Thanks for the question. So time lime going forward is that over the next series of years, we’ll have this play out and on the appeal at this point, it’s too early to speculate on exactly what the detailed timeline will be. But we’ve remained really confident in the investments was made in intellectual property here.

We think the decision was extremely well reasoned. It was brought forth by three judge panel. They were substantial decisions 50 plus pages of articulation of their views. So we look forward to what comes ahead. We feel good about our position and we’ll play out over a series of years..

Whitney Ijem

Got it. Thanks for taking the question..

Andrew Hack

You bet..

Operator

Thank you. And our next question is from the line of Matthew Harrison with Morgan Stanley. Your line is open..

Cyrus Amoozegar

Hi. This is Cyrus on for Matt. Thanks for taking my question.

I guess the first one is, there was a lot of data at ASGCT, what piece of the data do you guys is most important when thinking about your next clinical candidate?.

Katrine Bosley

So I’ll ask Charlie to speak to the data that was presented at the conference. Overall though, we have a number of programs we are working on. And - while we haven’t articulated what specific program we think will come next.

We do plan to continue to share data at meetings just as have at STGP [ph] so that you can have a window into the progress that we’re making.

To articulate from the highlights from the meeting?.

Charlie Albright

Sure. We touched on several things at the meeting I thought were important. At the top of the list was the non-human primate data that we disclosed that we talked about our program for hemoglobinopathy are reviewed some of that here.

What I didn’t talk about was advances we’ve made in Alpha-1 Antitrypsin, a liver disease and our ability to deliver in AAV both knock down of Alpha-1 Antitrypsin and hormoligosis recombination a potential repair, that deficit as well.

And then I went through some of the platform initiatives that we had which were the self-inactivating Cas9, the synthetic guide RNAs, which I didn’t touch on a day very instrumental part of us understanding the nature of the edits that are made and proprietary to that Editas Medicine.

And so we thought there were several significant things at the meeting..

Cyrus Amoozegar

Great, thank you.

And the one more, for the IND delay, I was wondering the impression that critical supply was not necessary to filing IND?.

Katrine Bosley

And so the materials that cause of delay was actually to supply the preclinical toxicology study, so it was on a critical path..

Cyrus Amoozegar

Got it. Thank you..

Operator

Thank you. And our next question is from the line of Phil Nadeau from Cowen & Co. Your line is now open..

Phil Nadeau

Good afternoon, thanks for taking my questions.

First just a follow-up on the manufacturing side, can you talk a little bit more about exactly what happened with the material, was there any scientific questions that cause it to be delayed in getting to manufacturing or was it simply somebody’s manufacturing process was slower than anticipated?.

Katrine Bosley

Sure, I’ll actually ask Vic Myer, our Chief Technology Officer to talk about this.

Vic?.

Vic Myer

Yeah, so I’m sure as you know AAV manufacturing requires several steps to happen in perfect sequence for things to all come together. And we’re using several external contracts to perform these steps.

To sort of painting really broad strokes you can think of is a two part problem where we have to produce the input material that all comes together and then create AAV in a cell culture systems. In our case, one of the input materials fails the quality specification and we need to go back and remake that material.

That delay in remaking material cause us to miss the manufacturing slot with the AAV CMO and that combined with the remaking material pushed out the timeline..

Phil Nadeau

And just to be clear, there is really no risk that the contract manufacturer can hit those quality specifications, is this just kind of a onetime setup?.

Vic Myer

The materials actually been remade and pass quality control and it’s ready to be manufactured..

Phil Nadeau

Perfect. That’s helpful. Thanks.

Then secondly, on the non-human primate data just two follow-up questions, one, how precise is we estimate that photoreceptor is at 25% of generic material and for how precise is the estimate of the year, that you’re editing 50% of real? And then secondary to that you mentioned a 10% therapeutic target, where does that 10% number come from?.

Katrine Bosley

Charlie?.

Charlie Albright

Sure. Let me take the first half of that and I’ll pass it to the Gerry take the therapeutic relevant question. And so what we did was take a number of slices through a non-human primate put a receptor and count the number of photoreceptors in that retinal punch and that’s where that number came from.

And obviously there’ll be some inaccuracy in that number depending on exactly where the slice is, but it’s a reasonably accurate number and can totally consistent with what’s in the literature as well. So we feel reasonable about that value. So there’s not a lot of inaccuracy there.

And Gerry, you want to comment on the therapeutic relevance?.

Gerry Cox

Sure. Yeah, the 10% number that comes from studies done in the early 90’s both and healthy volunteers who had increasingly areas of their vision of scared by a grid and was found that about, it took about 90% of the up scaring of the visual field for patients to really lose recognition of objects.

And the other study took place and patients with a Muscular Dystrophy which approximately 90% of their photoreceptors were lost in the region of phobia which is our daytime high acuity vision, but the patients have near normal vision.

So those studies combined give us comfort that if we were ahead at least 10% of the photoreceptors and corrective phenotypes that that would provide a meaningful that are fit to patients..

Phil Nadeau

Great. That’s helpful.

And then Andrew one last question for you, on the R&D expense for this year, would we be corrected using the Q1 level as the base for the next several quarters or was there anything lumpy in Q1?.

Andrew Hack

So, no, until the Q was filed, a little bit after our press release and so you can see in there the values ascribed to the issuance of notes associated with onetime events, you should also assume that the stock and other non-cash items can be lumpy quarter-to-quarter. So I would encourage you to reference the Q for the carve outs from 1Q..

Phil Nadeau

Got it. Thank you..

Andrew Hack

Yeah..

Operator

Thank you. And our next question is from the line of Mike King with JMP Securities. Your line is open..

Mike King

Hey, guys. Thanks for taking the question. Apologize for any background noise. Just wanted to see - a lot of my questions been answered but maybe a follow-up on the thought process from ASGCT.

Can you talk about whether you might be reprioritizing or emphasizing your R&D spend on stem cells and hemoglobinopathy and perhaps pretty more of a focus on antitrypsin as well?.

Katrine Bosley

So with regard to the portfolio and priorities there, the data that we presented essentially a firm all of the priorities that we’ve had. So you’re not going to shift them in priorities.

The results that we presented will continue for the result of future meetings and as we have - as you get to designating a program to reinforce the clinical, certain we will share that. But I think what you’re really seeing with ASGCT result is that the investments we are making in our priority programs have made good progress..

Mike King

Right. Thank you..

Operator

Thank you. [Operator Instructions] And our next question is from the line of Madhu Kumar with Chardan. Your line is open..

Madhu Kumar

Hi, guys. Thanks for taking my questions.

So on the first one is relates to the manufacturing of AAV for LCA10 and Leber Congenital Amaurosis, can you provide some detail of what level of information you’ve given about upstream and downstream processes you guys have used AAV manufacturing? And I have a second question after that?.

Katrine Bosley

So the manufacturing of AAV is something that many have done in many different settings and what we’re doing is not dramatically different from standard AAV manufacturing. So there’s a not really sort of unique difference in what we’re doing from a standard manufacturing process for AAV..

Madhu Kumar

All right, and then I mean then in terms of thinking about LCA10, what kinds of guides and experience, it was similar the differences are there for LCA10 versus the kind of the latest stage AAV gene therapy of REGENXBIO from a Spark, so how do we think about the LCA10 program relative to REGENXBIO?.

Katrine Bosley

So I’ll ask Charlie, our CSO to speak to this question..

Charlie Albright

Well. There are similar that are delivered by AAVs and therefore the AAV delivery that’s happened in humans already has a level of confidence that they can be use of delivery to get the final receptors and other cells in the eye.

But aside from that they are really quite different, we’re at delivering editing machinery to cause a permanent change and a correction in LCA10 gene expression in this case as opposed to introducing a transgene..

Katrine Bosley

And it may be worth remembering, you may know the gene in particular involved in LCA10, the name is CEP290 gene that’s actually too large to fit into AAV.

So you wouldn’t be able to deliver CEP290 gene in a transgene format because that approach to gene therapy approaching AAV wouldn’t be applicable in LCA10, which is the one that we’re focused on a gene editing approach. Gene editing strategy however is feasible with AAV delivery, delivering those editing components rather than transgene..

Madhu Kumar

I guess I mean less in terms of Vectorology in more in terms of the disease how formal is it, how is visual acuity is similar and different between LCA2 and LCA10?.

Katrine Bosley

I see. Okay. Sure. Gerry, you want to speak to that..

Gerry Cox

I think the LCA2 which is RPE65 mutations that tend to be more slowly progressive disease since LCA10 starts up more than night blindness and then later in childhood that’s decline in daytime acuity. The patients of LCA10 tend to be more severely affected from birth and much more impaired visual acuity during the day..

Madhu Kumar

Okay, great. Thanks very much..

Operator

Thank you. And ladies and gentlemen, this concludes the Q&A session for today. I would like to turn the call to Katrine Bosley for any final remarks..

Katrine Bosley

Great. Thank you. And so that we do thank all of you for participating in today’s call and for your support as we continue to work to bring forward new medicines and build out a tough medicine. Have a great evening..

Operator

Ladies and gentlemen, thank you for participating in today’s conference. This concludes the program, and you may all disconnect. Have a wonderful day..

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