Editas Medicine, Inc. Q1 FY2024 earnings call

• Reni-cel: Pleased with growing interest in reni-cel, completed adult cohort enrollment in RUBY, enrolling in adolescent cohort and continuing EdiTHAL enrollment; on track to present clinical data from RUBY and EdiTHAL studies in mid-2024 and year-end; manufacturing: Greg Whitehead promoted to Chief Technology and Quality Officer.
• In vivo pipeline: Aim to be a leader in in vivo programmable gene editing, remain on track to establish in vivo preclinical proof-of-concept by end of 2024; presented preclinical data at ASGCT Annual Meeting; Editas has in vivo human proof of concept validated by NEJM publication.
• Business development: Extended collaboration with Bristol-Myers Squibb, 13 programs opted into across 11 gene targets, 2 in IND-enabling studies and 4 in late-stage discovery; ongoing IP litigation regarding CRISPR patents, expect decision in second half of 2024; financials: Cash as of March 31, $377 million; revenue $1.1 million in Q1 2024 vs $9.9 million in Q1 2023; R&D expenses $49 million in Q1 2024; G&A expenses $19 million in Q1 2024.

Editas Medicine in the first quarter of 2024 focused on three strategic pillars. For reni-cel: Completed enrollment in the adult cohort of the RUBY trial for severe sickle cell disease, is enrolling patients in the adolescent cohort of RUBY and continuing enrollment in the EdiTHAL trial for transfusion-dependent beta-thalassemia. For the in vivo pipeline: On track to establish in vivo preclinical proof-of-concept for an undisclosed indication by the end of 2024. For business development: Extended collaboration with Bristol-Myers Squibb for 2 years with options to extend further, and ongoing IP litigation regarding CRISPR patents.

• For reni-cel: Provide clinical update from RUBY and EdiTHAL trials in mid-2024 and year-end 2024; complete adult cohort enrollment and initiate adolescent cohort in RUBY, continue enrollment in EdiTHAL and dosing in both studies.
• For in vivo pipeline: Establish in vivo preclinical proof-of-concept for an undisclosed indication by the end of 2024.
• For BD: Leverage robust IP portfolio and business development to drive value and complement core gene editing technology capabilities.

• Uncertainty regarding the outcome of the patent trial and appeal board appeal regarding CRISPR patents, which could impact the company's IP position and potential value.

Q: I'm wondering about your in vivo pipeline and the proof of concept that you're expecting by the end of this year. What would be the bar for success that you're looking for in this program?

A: Linda Burkly: We are looking for proof of concept for high efficiency delivery and editing for our targeted interest in vivo in this preclinical POC that will give us confidence in our ability to target the target of interest. We are going to be sharing more information on this at a future date.

Q: An interesting update on your disclosure is the plan to identify large serine recombinases, which implies sort of a new approach to many of your -- that many of your peers are also developing some [indiscernible]. Does the size of the recombinases, mediated insertion allow for inserting a coding sequence for dystrophin, for example, and are you able to comment on whether DMD is out of the question regarding your in vivo aspirations?

A: Linda Burkly: We're excited about the LSR technology that we're disclosing here. We are identifying many different novel LSRs. We're not disclosing at the moment, the size of the integrations that can be accommodated by the large serine recombinases but we have quite a few novel LSRs that we have identified and we further charactering them.

Q: Looking at the reni-cell program here, how are you guys looking at the midyear any sell update compared to the year-end update here. As follow-up time progresses, what should we look for from treated patients in terms of safety and efficacy moving forward?

A: Baisong Mei: So in this middle year release, we expect to have at least 18 patient data for RUBY study. And within the 18 patients and 4 of them will have 12 longer, 12 to 21 months of exposure, and 7 will have 5 months to 12-month exposure and another 7 with 2 to 5 months exposure. For the EdiTHAL study, we will have patients from at least 7 of those patients with 4 to 12 months of exposure, which also will be very meaningful.