Editas Medicine Inc (EDIT) 2016 Q3 法說會逐字稿

Good day ladies and gentlemen, and thank you for standing by. Welcome to the Editas Medicine Q3 2016 Earnings Conference Call. At this time all participants are in a listen-only mode. Following our prepared remarks we will host a question-and-answer session, and our instructions will follow at that time. (Operator Instructions). Now it is my pleasure to hand the conference over to Mr. Mark Mulligan, Senior Director of Finance and Investor Relations. Sir, the floor is yours.

Good afternoon. This is Mark Mullikin, Senior Director of Finance and Investor Relations at Editas, and I would like to welcome you to our update call for the third quarter of 2016. We issued a press release earlier this afternoon providing our third quarter 2016 financial results, and updates regarding our Company. A replay of today's call will be available approximately two hours after its completion on the Investors and Media section of our website. 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 provisions 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 on 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. Now let me turn over the call to our Chief Executive Officer, Katrine Bosley.

Thank you, Mark. Good afternoon everybody, and thank you for joining us for our third quarter 2016 update call. I am joined by several members of our executive team, including Andrew Hack, our Chief Financial Officer, Xandra Glucksmann, our Chief Operating Officer, Vic Myer, Chief Technology Officer at Editas, and also Charlie Albright, our Chief Scientific Officer, and Jerry Cox, our Chief Medical Officer, who both joined us over this past quarter, and it is great to have them here as part of the team. As we are working to build our Company, we have three key objectives, and as always, we do appreciate the opportunity to update you today on our progress.

As a reminder, these objectives are number one, to advance our pipeline and expand our platform, number two, to build our business for the long-term, and three, to develop an outstanding organization. This has been a particularly active quarter in terms of organizational development. I would like to make a few comments on that. We made a number of key senior hires, including Charlie Albright as CSO, and Gerry Cox as CMO. And in addition Ken LeClair joined us as Vice President of Technical Development and Manufacturing, and Semiramis Trotto joined us as Vice President of Human Resources. These are all important additions, which round out our senior Management Team, and ensure that we have the right leadership in place as we advance Juno's medicines into the clinics, and as we continue to grow the organization.

We have also continued to build Edtas' team across many dimensions more broadly, the quality of the people we have recruited at the most senior levels is reflective of our overall philosophy to hire the best and the brightest, and in line with our plans, we currently have approximately 90 full time employees onboard. In addition, we recently moved into our new headquarters in Kendall Square in Cambridge, and here we are ample room to further build the organization for the long-term. Charlie and Gerry are on the call with us today, and they will share with you their perspectives on Editas, and on the field of genome editing. Building a leading genome editing company means we need scientific and clinical leadership, with a unique combination of insight and experiences, as we were looking for the right people for these leadership roles, we knew it was critical to find individuals with very specific capabilities.

First, significant expertise in genomics and advanced biologics, and second, meaningful experience developing important new medicines at every stage, from discovery to approval. In addition, we sought experience developing medicines for rare diseases, which is quite different from other areas of drug development. We're very fortunate to have found both Gerry and Charlie, who are unique leaders in the industry in all of these regards. So I would like to introduce our Chief Scientific Officer, Charlie Albright, who will then be followed by our Chief Medical Officer, Gerry Cox, and then Andrew Hack, our Chief Financial Officer, will share some brief remarks on our finances. Charlie.

Thanks Katrine. It is a pleasure to be with you today. I joined Editas from Bristol-Myers Squibb, where I was Vice President of genetically defined diseases and genomics. In the genetically defined diseases group, we discovered potential medicines directed towards targets that were identified by human genetics and then tested these molecules in patients with rare diseases. In the genomics group, we provides genomic tools and information for all of the research and development. With the Drug discovery biologists the depth and quality of the discovery platform is one of several things that attracted me to Editas.

In particular Editas is well on its way to industrialize genome editing based on CRISPR technology. The identification of potent and selective guides is but one example of this industrialization. For instance, we developed a combination of a proprietary in silicone methods, cell based assays, and unbiased methods to detect off target cutting, in order to identify potent and specific guide RNAs A recent disclosure of editing of the PD-1 gene in teeth cells illustrates what we can do. Specifically, we were able to [violillically] knockout PD-1 in more than 90% of primary M & T cells without measurable off target setting. The differentiated investment at Editas has made, and platform innovation is also impressive. In addition to the original Cas9 and for sprec term Streptococcus pyogenes, Editas has several cas9 enzymes at its disposal, including high fidelity Cas9, Cas9 with altered PAM specificity, and Streptococcus aureus Cas9. The PAM variant Cas9s increased a number of genomic sites we can access, and we believe this will eventually help us address more disease targets.

In contrast to the more widely used pyogenes Cas9, the smaller staph aureus Cas9 gives us the ability to put two guide RNAs and the Cas9 coating sequence in a single adeno associated virus. We're exploiting that Staph aureus Cas9 to create a therapy for LCA 10, where we are deliver Cas9 and two guide RNAs to the sub-retinal space using NAAV. Importantly, this therapeutic approach would not be possible without the Staph aureus Cas9. Taking all of these proprietary advances together, I think the discovery platform and product candidates put us in an excellent position to deliver transformational medicines to patients with a wide range of diseases. Now I will turn it over to our new Chief Medical Officer, Gerry Cox.

Thanks Charlie. Hello to everyone on the call. I joined Editas from Sanofi Genzyme, where over the course of nearly two decades, I led the development of many of Genzyme's lysosomal storage disorder products as Vice President of rare disease clinical development. I have also continued to practice medicine as a clinical geneticist at Boston Children's Hospital, where I care for children suffering from debilitating genetic diseases every week. Through my work in clinical development, and as a practicing physician, I have witnessed the potential of new therapies to dramatically extend and improve patient's quality of life. Transforming diseases that were once disabling or fatal, into chronic but manageable conditions.

Among the many reasons that I decided to join Editas, is the Company's commitment to developing and applying CRISPR technology broadly, across numerous therapeutic areas. This is evident in our approach to deliver, where we are working across multiple delivery modalities, whether that's adeno associated viruses, lyponano particles, or as ectoporation in the case of our Ex vivo programs. We're not constrained by the delivery modality, which gives us the flexibility to select and optimize whichever works best for each indication. That's really exciting to me, because it means that we can address the broadest range of diseases and patients possible. I will now turn over the call our Chief Financial Officer, Andrew Hack.

Thanks Gerry. As is our convention, I will review the most important components of our financials, but won't be walking through all of the details of the results that can be found in our press release. In the third quarter of 2016, our net cash used in operations was approximately $17 million, and as of September 30th, 2016, we had approximately $200 million of cash and cash equivalents.

The primary drivers of our spending are our expanding pipeline, our platform, and our legal expenses. Based on our current cash position, as well as research support under our collaboration with Juno Therapeutics, and payments from the Cystic Fibrosis Foundation, we believe we have at least 24 months of capital to fund the advance of multiple therapeutic programs in parallel, and to further extend our technology platform leadership. And with that, we'll open it up for your Q&A. Operator.

Thank you. (Operator Instructions). Our first question comes from the line of Gena Wang with Jefferies. Your questions, please.

Hey. This is actually [Shelby] in for Gena. Thank you so much for taking our questions. So maybe start with an IP question. So what would be your expectation for the interference outcome at the motion space, and what would be your expected outcome of strategy after the board ruled that if there is interference, or if there's no interference? Thank you.

Sure. Thanks for your question. We believe the interference is going well, and overall it is proceeding as we had anticipated, there really haven't been any surprises. As you noted we are part way through the procedural phase. There will be some decisions the patent office will make in Q1, and then we will go from there. But overall we remain confident in our overall IP position. The foundations are very strong, and the proceeding is going well. No surprises.

Thank you. Our next question comes from the line of Marc Frahm with Cowen and Company. Your questions, please.

Yes. Thanks for taking my questions. Could you just give us an update on kind of where things stands in LCA10, and whether formal IND enabling phase has started yet, and if not what the schedule is going to be there?

Sure. So we've said before, our aim is to initiate clinical development with this program next year, and we do remain on track for that goal, for that aim. You may recall this year we have publicly shown data showing we have guides of high efficiency, no detectable off targets, and are able to significantly increase the protein expression of LCA10 of, full expressions of protein in patient cells with LCA10. We will continue to provide updates on the data at major scientific conferences, and our quarterly calls. In terms of the steps towards IND, we'll share those data in due course, as the program advances, but I think overall the key point is that we remain on track to initiate clinical development next year, as we have had for a goal for some time.

Okay. Great. And when the kind of formal IND stuff does start-up, do you intend to disclose that immediately, or is that something we should just kind of expect to hear on a quarterly call? What is the disclosure plan there?

Andrew, do you want to talk a little bit about how we share information, and how we take that on?

Yes. Sure. So obviously we update each quarter our progress against our goals, so that you can hear obviously on this call that things are progressing well. Beyond that, we share data at major medical and scientific conferences, and we use that as an opportunity to help bring people along with the progress we are making in our programs. So those are the two primary venues. You should expect us to continue to use those as the places that we communicate.

Okay. Thank you.

Thank you. Our next question comes from the line of Roy Buchanan with Janney Montgomery. Your questions, please.

Hi. Great. Thanks for taking my questions. I had a couple on hash abstracts, one abstract you guys have about the editing and HFPCs, getting up to 12% Homologous recombination, I just had a question as an adult CD 34 plus cells. Is that biolilic, and is that sufficient to go to the clinic and if not what do you need to do to get the to the clinic?

So I am going to ask Charlie Albright, our CSO to speak to that. Charlie.

Sure. That is a combination of mono and biolilic editing, and as you will appreciate, there's a range of opinion on how much recombination you need to go into the clinic. That's on the low end. So we wouldn't want to comment if that were sufficient to go into clinic or not.

Okay. And then there's another abstract, it is not actually yours, but they sort of pretty high level although a small end of errors with Homologous recombination. Can you guys give us a sense of what you might expect to see, as far as errors with Homologous recombination, or is that may be an outlier? Thanks.

Yes. Sure. This is Vic Myer, the Chief Technology Officer. It is probably premature for us to comment on other folks abstracts, but as you know Homologous directive repairs is a complex process that the cell undergoes. In our reported data, and data that is queued up for publication, we have not observed such a high rate of Edits happening using single strand oligos in cell culture systems. We have in fact, our editing, our HDR correction when there is HDR, is solid, and there's not unexpected edits at the site.

Okay. That's helpful. I think it was an academic group. Maybe that is the reason. Thanks.

Thank you. There are no further questions in queue. So now I would like to hand the conference back over to Katrine Bosley, Chief Executive Officer, for closing comments and remarks. Ma'am.

Great. Well, thank you, we definitely appreciate everyone's participation, and your support as we continue to build Editas, and work to bring new medicines to patients. Have a great evening. Thank you.

Ladies and gentlemen, thank you for your participation on today's conference. This does conclude the program, and you may all disconnect. Everybody have a wonderful day.