Editas Medicine, Inc. (EDIT) Earnings Call Transcript & Summary

Ladies and gentlemen, thank you for standing by. And welcome to the Editas Medicine 2020 Annual Meeting of Stockholders. I would now like to hand the conference to your speaker today, James Mullen. Please go ahead, sir.

Good morning. And welcome to the 2020 Annual Meeting of Stockholders of Editas Medicine, Inc. I'm Jim Mullen, Chairman of the Board of Directors of Editas Medicine, and I will be presiding over this meeting. At this time, I call the meeting to order. Our top priority is safety of our stockholders and employees. And due to the public health impact of COVID-19 pandemic and to support the health and well-being of our stockholders, employees and communities, this year, we are holding our annual meeting in an all virtual format, and we're pleased to have everyone join this live webcast. We have designed this meeting to provide stockholders the same rights and opportunities to participate as they would at an in-person meeting. Before we get to the formal business of the meeting, I would like to make some introductions. Present at the meeting today are Akshay Vaishnaw, a nominee for director; as well as Jessica Hopfield, David Scadden and Andrew Hirsch, each a director of Editas Medicine; and Cynthia Collins, the company's President and Chief Executive Officer and a director. In addition, the following members of management are also with us today: Charles Albright, our Executive Vice President and Chief Scientific Officer; Michelle Robertson, Chief Financial Officer; Clare Carmichael, our Chief Human Resources Officer; Charlene Stern, our Senior Vice President and Chief Legal Officer; and Harry Gill, our Senior Vice President of Operations. Terry Hassett, who will be serving as our inspector of elections, and representatives from Ernst & Young, our registered public accounting firm, are also with us. After we complete the formal part of the meeting, Cindy Collins will give a short presentation on behalf of the company, and then we will take questions. In order to conduct an orderly meeting, we ask that you follow the rules of conduct for the meeting which have been uploaded to our website and include information about participating in the meeting, including asking questions. Please note that various remarks that we make about future expectations, plans and prospects for Editas Medicine constitute forward-looking statements for the 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, these forward-looking statements represent our company's expectation only as of today. While our company may elect to update these forward-looking statements, it specifically disclaims any obligation to do so. Any forward-looking statements should not be relied upon as representing our company's estimates or views as of any date subsequent to today. I have received an affidavit from the company's proxy solicitor, Broadridge Financial Solutions, Inc., certifying the notice of annual meeting and proxy statement were sent to all stockholders of record as of April 28, 2020. This affidavit is available for inspection by any stockholder upon request following this meeting. Terry Hassett has been appointed to act as inspector of election. I would now ask Terry to furnish us with the count of the number of shares present at this meeting in person or through representation by proxy. Terry?

There are present at this meeting in person or through representation by proxy a majority of the outstanding shares of the company's common stock.

There being a majority of outstanding shares of common stock represented at this meeting out of the total of 54,982,559 shares entitled to vote, I hereby declare that a quorum exists. Turning now to the items to be voted on at this meeting. As indicated in the notice of meeting and accompanying documents that were available to stockholders, the first matter to be voted on is the election of Class I directors to serve until the Year 2023 Annual Meeting of Stockholders. The 2 nominees for this Class I director election are myself, James C. Mullen, and Akshay Vaishnaw. The next matter to be voted on is the advisory vote to approve named executive officer compensation. The proxy statement for this meeting contain the text of the resolution that the stockholders are asked to approve. And the final matter to be voted on is the ratification of the selection of Ernst & Young LLP as our independent registered public audit accounting firm for the fiscal year ending December 31, 2020. I hereby declare the polls are now open for each matter to be voted upon today. If you have not yet voted or if you have previously voted by proxy and wish to change your vote, you may vote by clicking the vote button here on the virtual meeting website and following the instructions there. I'll pause for about a minute to allow stockholders to vote. [Voting]

Now that everyone has had an opportunity to vote, this concludes the business items on the agenda for this annual meeting. The polls are now closed. We now have the preliminary report of the results of the annual meeting. Each of the nominees for director has been elected as a Class I director. The advisory resolution approving executive compensation has been approved. The selection of Ernst & Young LLP as our independent registered public accounting firm for the fiscal year ending December 31, 2020, has been ratified. The inspector of elections will tabulate the final votes, and the final votes results will be included in the Form 8-K, which will be filed within 4 business days after this annual meeting. As there is no further business to come before the meeting, I declare the formal part of the meeting adjourned. The company's President and Chief Executive Officer, Cindy Collins, will now give a presentation regarding the company, and members of the company's management will answer appropriate questions from stockholders. Please follow instructions provided on the virtual meeting website to submit questions. Cindy, I'll turn it over to you.

Thank you all for joining our Annual Shareholders Meeting. I am pleased to present to you today. Pushing forward the boundaries of medicine entails risk as well as great promise. This slide tells you about the risks. At Editas, we strive to make differentiated, transformational medicines for diseases of high unmet need. To accomplish this objective, we are pursuing 2 strategic pillars: in vivo CRISPR medicines and engineered cell medicines. With our in vivo CRISPR medicine portfolio, we are leveraging AAV delivery with our proprietary Staph aureus Cas9 enzyme to develop medicines for serious ocular diseases. We then plan to expand into neurological diseases in the near term and other therapeutic areas further out. For our engineered cell medicines, we are developing best-in-class medicines for hemoglobinopathies and cancers with our proprietary Cas12a enzyme. Over the past few years, we have invested heavily in our platform and can now leverage this effort to advance both in vivo and engineered cell medicines. We have a strong IP portfolio and continue to advance our organizational capabilities. Based on this strategy, we are advancing a broad pipeline of medicines. You can see that our most advanced programs are EDIT-101 for LCA10, EDIT-301 for sickle cell disease and beta-thalassemia, and EDIT-201 to treat solid tumors. On the in vivo side, we are also advancing programs for additional ocular indications and expanding into neurological diseases. In engineered cell medicines, in addition to EDIT-301 and EDIT-201, we are making strong progress on our iPSC-derived NK cell program for solid tumors. Now let's turn to our in vivo portfolio, leveraging our unique capability to deliver CRISPR medicines using AAV. We are pioneering in vivo CRISPR medicines with EDIT-101 for patients with Leber congenital amaurosis 10 or LCA10. LCA10 is an inherited form of blindness caused by a mutation in the gene that encodes the CEP290 protein. Without this protein, the photoreceptors rapidly degenerate, robbing young children of their sight. We hope to rescue vision in these patients by removing the genetic mutation to restore the CEP290 protein and thereby rebuild the photoreceptors. Earlier this year, we became the first in history to treat a patient with an in vivo CRISPR medicine when we initiated dosing in the Brilliance clinical trial for LCA10. We aim to complete the adult low-dose cohort and dose at least 1 patient in the adult mid-dose cohort by year-end, providing the potential for clinical data this year. The design of the Brilliance clinical trial is typical for a rare disease. This is an open-label, dose-escalation study with primary end points assessing safety and tolerability and secondary end points measuring efficacy. We are starting in adult patients with light-perception only and with a dose that is on the low end of the range where we expect therapeutic benefits. Based on safety and tolerability in these patients, we intend to escalate the dose in adults and then move into children who have some visual acuity. We are pleased that the study was cleared to continue following a review of safety data on the first patient. In addition to primary safety end points, secondary efficacy end points include visual acuity, a mobility course, macula thickness, pupillometry and electroretinogram. Our second ocular program, EDIT-102, is aimed at USH2A, which like LCA10 is an inherited retinal disease affecting the photoreceptors. As a result, EDIT-102 benefits tremendously from our work on EDIT-101. In particular, we use the same vector, same Cas9 and same promoter as EDIT-101. We believe this derisks and accelerates our path to the clinic and ultimately a transformative medicine for these patients. EDIT-102 is ready for IND-enabling studies, pending a decision from AbbVie on whether to license the program under our 2017 collaboration agreement with Allergan. We expect their decision by the third quarter. As you have seen from our progression of ocular programs, we leverage learnings for subsequent programs, thereby allowing us to create clinical molecules with increased efficiency. We now plan to leverage our ocular learnings into new tissues that are accessible by AAV. We are uniquely positioned to do so with our proprietary Staph aureus Cas9 enzyme, which is small enough to be delivered with a single AAV for increased efficiency. Beyond ocular, we are expanding into neurological diseases in the near term and additional therapeutic areas further out. For these reasons, we view in vivo CRISPR medicines as 1 of our 2 pillars of our therapeutic strategy. Now I'd like to transition to engineered cell medicines, the second pillar of our therapeutic strategy. Let's start with our editing of hematopoietic stem cells for beta hemoglobinopathies, including sickle cell disease and beta-thalassemia. These diseases are areas of high unmet need with a clear understanding of the underlying biology. In particular, we know from human biology that elevated fetal hemoglobin can dramatically reduce the symptoms of these diseases. We recognize that this is a competitive field and believe our candidate, EDIT-301, can be best-in-class, which I will discuss on the next slide. We remain on track to file the IND for sickle cell disease by the end of this year. This slide summarizes why we think EDIT-301 can be best-in-class. Starting at the top, we believe that EDIT-301 has the potential to impact beta globin in the best way. In particular, preclinical data shows that we induce more fetal hemoglobin than BCL11A enhancer approach. And relative to lentiviral gene therapy, gene editing will reduce the sickle globin and, therefore, not have to compete for alpha globin in the same cell. On the safety side, editing at the globin locus but not at the BCL11A locus is supported by human genetics. Our preclinical studies identify one potential concern for BCL11A editing as we found deleterious lineage skewing when editing the BCL11A locus. Finally, gene editing is more specific than lentiviral expression. To get the high levels of beta globin required for efficacy, there will be cells in the CD34 population that carry up to 20 copies of the viral genome. These random integration events have the potential to inadvertently activate or inactivate genes involved in tumorigenesis. This slide includes results from our preclinical in vivo studies using blood from healthy human donors. As we had predicted from our in vitro studies, editing at the beta-globin site with Cas12a caused a robust induction of fetal hemoglobin, exceeding 50% and approximately 45% above background. Encouragingly, fetal hemoglobin induction was pancellular, as shown on the right. Taken together, we believe that EDIT-301 has the potential to be a best-in-class medicine, and we are excited to progress to the IND stage this year. With this vision for the cell therapy space, there are several cell types that are potentially useful for oncology. Alpha-beta T cells are part of our adaptive immune system and have proven therapeutic for liquid tumors. These cells recognize tumors, either with the endogenous alpha-beta T cell receptor or synthetic chimeric antigen receptors or CARs. While we can edit these cells to construct allogeneic medicines, the potential for graft-versus-host disease from residual contaminating cells remains a significant issue. Further, the number of edits required to make these cells allogeneic will consume much of the potential for genetic changes in these cells. For these reasons, we are focusing our efforts on our wholly owned programs on innate immune cells, particularly natural killer or NK cells. NK cells can recognize tumors by a variety of mechanisms, including multiple innate receptors that recognize cells that don't express T cell antigens and cells that express stress ligands. NK cells are also part of the mechanism by which many therapeutic antibodies kill tumor cells in a process known as antibody-directed cellular cytotoxicity. Further, NK cells can be engineered with CARs. And importantly, NK cells do not cause graft-versus-host disease. We recently initiated IND-enabling studies for EDIT-201, an allogeneic NK cell medicine targeting solid tumors. Approximately 90% of all cancers are solid tumors. EDIT-201 is the first of these medicines that we hope will extend and dramatically improve the lives of these patients. While we believe that edited healthy donor-derived NK cells will be a significant advance to the field, we believe that iPSC-derived cells will unlock the full potential of cell medicines for oncology. This slide depicts the process of making iPSC-derived cells and some of the progress we have made. Starting on the left, iPSCs are created from somatic cells by dedifferentiation. We have partnered with BlueRock Therapeutics to both obtain GMP cell lines and transfer the knowledge of this process to Editas. These cells must then be edited and characterized so that a clonal cell line can be selected. We have optimized editing conditions with our proprietary Cas12a derivator. Further, we have built an unparalleled ability to characterize the genome of edited cells. We will then take these edited clonal lines and differentiate them to make iPSC-derived NK cells using a proprietary and scalable process. The end result will be cells that are more highly engineered than is possible with other cell sources with a low cost of goods and available off-the-shelf in perpetuity. On this slide, we show some of our early results with iPSC-derived NK cells. The left graph shows that we can efficiently edit a variety of genomic targets. The right graph shows that edited cells are more potent at killing tumor cells than unedited cells. Partnerships have been and will continue to be an important component of building our leadership position. We have 2 important development and commercialization partnerships. The first is with Allergan, now part of AbbVie, in ophthalmology. The second is with Bristol-Myers Squibb to develop alpha-beta T cell medicines for cancer and autoimmune diseases. We have also formed 3 important research collaborations to enable and accelerate our pipeline. We have partnered with AskBio to access technology and capabilities that support development of an in vivo CRISPR medicine for neurological diseases. We have a nonexclusive cross license and collaboration with BlueRock Therapeutics to access technology and GMP iPS cell lines to enable our iPS-derived oncology program. And earlier this year, we signed a deal with Sandhill Therapeutics to access their proprietary BINATE technology to develop healthy donor NK cell medicines for solid tumors. We also underpin our business with a very strong intellectual property position. To close, we are making progress on both pillars of our therapeutic strategy to create differentiated, transformational medicines for diseases of high unmet need using our proprietary Staph aureus Cas9 and Cas12a enzymes. For the in vivo medicines, we are advancing EDIT-101. Earlier this year, we treated the first patient ever with an in vivo CRISPR gene editing medicine. In addition, the remainder of our ocular program is progressing nicely. We are leveraging these efforts in ocular therapies to pursue additional therapeutic areas, starting with neurological diseases. For engineered cell medicines, our lead program is EDIT-301 for sickle cell disease and beta-thalassemia. We continue to believe that EDIT-301 is a potentially best-in-class medicine. We have made significant progress in our healthy donor NK cell medicine program, and we are excited to advance EDIT-201 for the treatment of solid tumors. In parallel with EDIT-201, we are also advancing programs using CRISPR editing for the potential treatment of both solid and liquid tumors, including engineered iPSC-derived NK cells. We remain incredibly excited about the times ahead, and I look forward to updating you on our future milestones. Thank you for your attention, and I would be happy to take questions.

Cindy, we have just a couple questions. They are related to our EDIT-101 program. And I will just summarize. They are asking for if you could provide any additional detail on potential for when data may be released to the public as well as plans for dosing additional patients particularly around the adult low-dose cohort.

Sure. Thank you. The dosing of the second patient, we hope to occur in the near term. We have, as I mentioned, been cleared to progress to the second patient based on the first patient safety data. It is dependent upon the reopening of our clinical sites under the current COVID situation, but we hope and expect that will be very soon. Regarding the release of clinical data, we are not anticipating to release data on a patient-by-patient basis, but do hope to have data available later this year. And obviously, if there's anything significant to report, we will report that data, but we want to make sure that we are appropriately reporting data and not necessarily on a patient-by-patient basis.

So at this time, I'm showing no further questions.

Okay. Well, thank you, everyone. We appreciate your time and attention have a great day.

Take care. Thank you.