Editas Medicine, Inc. Q3 FY2023 earnings call

• Gilmore O'Neill outlined the company's strategy pillars: accelerating EDIT-301 clinical development, sharpening discovery focus on in vivo editing, and expanding business development.
• EDIT-301 progress: Enrolled 27 sickle cell and 8 beta thalassemia patients, expects to dose 20th patient in RUBY trial by Jan 2024, FDA granted RMAT designation to EDIT-301 for severe sickle cell disease.
• In vivo pipeline: Linda Burkly joined as CSO to lead in vivo therapeutic targets, working on hematopoietic stem cells and other tissues.
• Business development: Entered agreement with Vor Bio for ex vivo Cas9 gene edited HSC therapies for hematological malignancies.
• Clinical data: Will present RUBY and EdiTHAL data at ASH webinar on Dec 11, including efficacy (total hemoglobin, fetal hemoglobin, vaso-occlusive events) and safety (neutrophil and platelet engraftment) data.

Editas Medicine reported revenue of $5.3 million for the third quarter of 2023, primarily from the upfront payment under the non-exclusive Cas9 license to Vor Bio. Cash, cash equivalents, and marketable securities as of September 30 were $446 million. R&D expenses were $41 million, G&A expenses were $15 million. There are no product segments with revenue contribution percentages as the focus is on specific clinical programs.

• EDIT-301 dosing of 20th patient in RUBY trial expected in Jan 2024.
• On track to present substantive clinical data of sickle cell patients with considerable follow-up in mid-2024.
• Confident in progress with RMAT designation aiding regulatory path, including intensive FDA guidance and possible rolling review.

• Potential differences between forward-looking statements and actual results due to various factors, including those in the Risk Factors section of the 10-K.
• Uncertainties around off-target editing and regulatory approvals for EDIT-301.

Q: Good morning, guys. Congrats on the progress and thanks for taking my questions. [Technical Difficulty] develop off-target stack [ph] and services that you're seeing. Can you give us a workout -- can you give us a sense of the work that you're doing for off-target. Are you doing whole genome sequencing and any other insights that you see as potential to your path to approval? Thank you.

A: Brian thanks very much. I'm afraid it was incredibly difficult to hear you with the background noise and I know you're suffering from some connection problems there. I think I heard you talk about off-targeting, so I'm assuming you're referring to this week's transform. Yes. And so let me just go with that and then we can follow-up if it's incorrect. But, as you know, I think you referred to the AdCom, and I have to say is that the AdCom represented an incredibly and did a great excitement for this week. It was a great day for sickle cell warriors where patients now can think about not battling a disease, but actually living a life. It was a significant milestone for CRISPR genome editing as that AdCom represents sort of almost a penultimate step towards approval for the first CRISPR-based medicine. What struck us in the very focused discussion of the AdCom was the positive tone, but actually also as we listened to the commentary, it really substantially strengthened our confidence in the very robust data package that we've generated about off-target editing. I hope I've answered your question, because with off-target editing is what I heard. I think the only other thing I want to emphasize, of course, is that we have chosen to advance our proprietary owned AsCas12a enzyme, which indeed has higher fidelity, and it's a significant reduction in off-target edits when compared to Cas9.

Q: Good morning. Thanks for taking my questions and congrats on the progress. Maybe the first, the HbF production levels are obviously quite impressive. Is there a total hemoglobin above which you start to grow concerned in sickle cell disease that patients have too much hemoglobin? If so, what that might be? And then maybe a follow-up for Caren. We saw a couple of large pharma gene editing deals this week. Perhaps you could comment on the overall level of interest in potential platform type collaborations. Thanks.

A: Thanks very much, Eric. With regard to the total hemoglobin, we believe that correcting and that's what we've seen hemoglobin physiology range is substantial benefit. I think we haven't -- we're not going to -- it's hard to speculate about a level that is too high. Indeed, there has been an experience in general with polycythemia in sort of a broader patient population with some conflicting data about the risks of same. But as I say, we feel very confident about the data we're getting with regard to a total hemoglobin and the way that we are correcting it to normal physiological ranges. I think you asked a question about some recent gene editing deals just this week that are announced. What I will say is that what is striking about is, it is great to see pharma, I say big pharma now in a period where we've seen some a dark of deals leaning in and increasing their excitement around the genome editing space. In other words, what I would say is this has been a very good week for CRISPR genome editing space with both that sort of critical near to final step towards approval for a first CRISPR-based therapy and to see now pharma actually looking through the lens of substantial de-risking their view of the value of genome editing as they look to grow their portfolios. I don't know. Caren Deardorf: Yes. Thanks, Eric. It's Caren. What I would add is I think Editas is so well-positioned right now, having refocused the portfolio, we are in a great place to be able to move our own programs forward and are very excited by the continued interest and it opens the door for partnering should that be the right path for us.