Editas Medicine, Inc.

Good morning and welcome to the Editas Medicine second quarter 2024 conference call. All participants are now in a listen-only mode. There will be a question and answer session at the end of this call. This call is being recorded at the company's request. I would like to turn the call over to Christy Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Thank you, Brittany. good morning everyone and welcome to our second quarter 2024 conference call earlier this morning we issued a press release providing our financial results and recent corporate updates a replay of today's call will be available in the investors 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 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 annual report on Form 10-K, which is on file with the SEC, as updated by our subsequent filings. 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. Except as required by law, we specifically disclaim any obligation to update or revise any forward-looking statements, even if our views change. Now, I will turn the call over to our CEO, Gilmour O'Neill.

Thanks, Christy, and good morning, everyone. Thank you for joining us today on Editas' second quarter 2024 earnings call. With me today are four other members of the Editas executive team, our Chief Medical Officer, Bae Sung Mai, our Chief Financial Officer, Eric Lucera, our Chief Scientific Officer, Linda Berkley, and our Chief Commercial and Strategy Officer, Taryn Deardorff. Because it is summertime, we're going to keep today's prepared remarks brief and leave more time to take your questions. Let me start by saying that we are pleased with Editas' momentum and progress in the second quarter of 2024 as we pursue Editas' goal to deliver life-changing medicines to patients with previously untreatable or undertreated genetic diseases and to position Editas as a leader in in vivo programmable gene editing. Three pillars underpin our strategy. The first of those pillars is to drive Renicel, a gene-edited cell therapy for hemoglobinopathies, formerly known as Edit 301, toward BLA and commercialization. The second, to build a differentiated in vivo editing pipeline. And the third, to increase business development activities with a particular focus on monetizing our very strong IP. At the start of 2024, we announced the following 2024 objective. For Renicel, we would provide a clinical update from the Ruby trial for severe sickle cell disease and the Edithal trial for transfusion-dependent beta thalassemia in mid-2024 and by year-end 2024. We would complete adult cohort enrollment and initiate the adolescent cohort in Ruby and continue enrollment in Edithal. For our in vivo pipeline, we would establish in vivo preclinical proof of concept for an undisclosed indication. And for BD, we would leverage our robust IP portfolio and business development to drive value and complement core gene editing technology capabilities. So how are we executed against this strategy and these objectives in the second quarter? Let's start with Renicel. First, we shared Ruby and Edithal clinical data in June at EHA 2024, the European Hematology Association's annual congress. The Ruby data set included 18 sickle cell patients with 2.4 to 22.8 months of follow-up, and the Edithal data set included clinical data from seven beta thalassemia patients with 4.1 to 12.8 months of follow-up. These data from the Ruby trial support our continued belief that Renicel will be a best-in-class product for sickle cell disease. So why do we think this? All patients treated in the Ruby trial are free from vaso-occlusive events post-Renicel infusion. All patients have robust correction of anemia with a mean total hemoglobin level within the normal range for both genders at greater than 14 grams per deciliter. And all patients have high fetal hemoglobin with levels well above 40% from six months onwards. We also demonstrate fast engraftment with low variability with a mean neutrophil engraftment of 23 days, which may translate to shortened hospital stays for patients. For cell collection, we have a mean of two apheresis cycles with a range of one to four per patient. Finally, in addition to the clinical data shared at EHA on the manufacturing front for any cell, we have a robust manufacturing process with a low failure rate that continues to improve with experience. This low rate of manufacturing failure can decrease patient burden and reduce overall costs as we avoid cell recollection and redundant cost of goods sold, or COGS. And we're on track to share additional clinical data for both the Ruby and Edithal trials by the end of this year, 2024. Second, Ruby enrollment and dosing continues to be strong. Indeed, we have now completed enrollment of the adolescent cohort. As a reminder, we completed the adult cohort enrollment in the first quarter of this year. We are manufacturing drug product and scheduling adolescent and adult dosing concurrently. We also continue to progress Edithel and are pleased to announce completion of the adult cohort enrollment and continue to dose patients. Finally, based on our continued and collaborative conversations with the FDA, we still expect our future BLA package to be similar in number of patients and duration of follow-up to what we've seen in the gene editing medicine field. Before I turn to InVivo, I'd also like to state that we are very focused on the best use of capital as we develop Renicel and map to the future. As part of that focus, we frequently evaluate opportunities, including the potential for partnering Renicel to most efficiently drive Renicel towards commercialization ultimately delivered to patients in need. Now let's turn to our InVivo pipeline, where we continue to strengthen our InVivo discovery capabilities and drive lead discovery work on InVivo therapeutic targets in hematopoietic stem cells and other tissues. Importantly, we remain on track to establish in vivo preclinical proof of concept for an undisclosed indication by the end of the year. We want to take a moment, rather I want to take a moment to reiterate our in vivo strategy to develop a pipeline of gene editing medicines for patients with serious genetic disease. As a reminder, our internal development efforts are differentiated from established modalities and we are not pursuing the same gene editing approach as others. Our strategy is to use our in-del technology for functional upregulation of gene expression to address loss of function or deleterious mutations. Let me be clear. Our strategy is not the knockdown strategy that others in gene editing are pursuing. And it is worth highlighting that we have already demonstrated that our in-del technology can drive functional gene upregulation, thus creating differentiated experimental medicines, as in our ex vivo development of Renicel. With Renicel, We leverage our INDEL CRISPR technology to upregulate the expression of the gamma-globin gene, a functional homologue of the beta-globin gene, through direct editing of the HBG1-2 promoter sites. We are now applying this same approach to in vivo therapeutics by using our INDEL technology to functionally upregulate the wild-type alleles or functional homologue of target disease genes as we build our differentiated pipelines. Why does the difference between our functional upregulation strategy and the knockdown strategy used by other companies matter? Because with our in vivo strategy, we are not competing with existing modalities or technologies in development that are based on knockdown strategies. Second, our indication selection strategy targets rare and orphan diseases that we believe will allow us to be the first or best in class for a given indication. We expect to move into diseases with larger patient populations in the future. Our lead discovery work is in in vivo therapeutic targets in hematopoietic stem cells and other tissues. Third, Editas is well positioned to achieve success with our in vivo strategy and pipeline with our established capabilities in four main components of in vivo gene editing medicine. One, our guide RNA modifications that enable high potency gene editing in multiple cell types, including in the liver, and improve gene editing outcomes in vivo. Two, a superior editing enzyme in ASCAS12A, three, our messenger RNA, and four, delivery technology, where we are currently evaluating lipid nanoparticles for delivery of gene-editing cargo into multiple tissue types with multiple companies, as well as evaluating additional next-generation delivery technology. I look forward to sharing more details about our in vivo pipeline later this year and our in vivo proof of concept. Finally, I'd like to refer you to our press release issued earlier today for a summary of our financial results for the second quarter of 2024. I'll take this opportunity to briefly review a few items for the quarter. Our cash, cash equivalents and marketable securities as of June 30th were $318 million compared to $377 million as of March 31st, 2024. As you will note, our burn rate was slightly higher this quarter than the past. This higher rate is due to increased external research and development expenses primarily related to clinical and manufacturing costs related to the accelerated progression of our Renicel program. We expect our existing cash, cash equivalents, and marketable securities together with the near-term annual license fees and the contingent upfront payment payment under our license agreement vertex to fund our operating expenses and capital expenditures into 2026. Before we turn to Q&A, as always, it must be said that we could not achieve our objectives without the support of our patients, caregivers, investigators, employees, corporate partners, and you. We are energized by and proud of our progress and execution this quarter. With our sharp and strategic focus, our world-class scientists and employees, our keen drive and execution, and strong balance sheet, we continue to progress our strategy to deliver differentiated editing medicines to patients with serious genetic diseases and evolve from a development-stage technology platform company into a commercial-stage gene editing company. Thank you very much for your interest in Editas. We are happy to answer questions now. Thank you.