Editas Medicine, Inc. Q1 FY2023 earnings call

Gilmore O’Neill introduced the company's new strategy focusing on in vivo programmable gene editing and hemoglobinopathies. The company successfully executed the strategy, increasing investment in the EDIT-301 program, divesting the iNK cell franchise, strengthening the discovery engine, and expanding business development. The EDIT-301 RUBY Phase 1/2 study showed promising data with ongoing enrollment and dosing. The FDA granted orphan drug designation to EDIT-301 for sickle cell disease. The EDITHAL Phase 1/2 trial for TDT dosed the first patient with successful neutrophil and platelet engraftment. The drug discovery group began lead discovery work on in vivo therapeutic targets. The company has a robust IP portfolio, including exclusive licenses to Harvard University’s and Broad Institute’s Cas9 patent estates.

Revenue for the first quarter of 2023 was $9.9 million compared to $6.8 million in the same period last year. The increase is related to the previously announced sale of the oncology assets to Shoreline Biosciences and related licenses. G&A expenses for the quarter were $23 million compared with $19.5 million for the first quarter of 2022. The $3.5 million increase is primarily attributable to increased professional services expenses to support business development activity, partially offset by a decrease in stock compensation expense. R&D expenses this quarter were $38 million, which was flat compared to the first quarter of 2022. This reflects a decrease in expenses following the strategic reprioritization of our portfolio, offset by increased investments to accelerate the development of EDIT-301.

Strategic objectives for the year include providing clinical updates from the EDIT-301 RUBY study in June and end of 2023, providing clinical data from EDIT-301 EDITHAL trial for TDT by the end of 2023, dosing 20 total patients in the EDIT-301 RUBY study by year-end, hiring a new CSO with specific expertise, advancing discovery of in vivo editing of hematopoietic stem cells and other tissues, and leveraging the IP portfolio and business development activities to drive value.

Potential risks include litigation appeal outcomes affecting CRISPR-related product launches, clinical development uncertainties, and regulatory approval uncertainties.

Q: Novartis recently terminated their sickle cell disease program after treating a couple of patients to see no benefit. Given your editing strategy is similar to what Novartis said, could you point to some differences why your promoter editing strategy should continue to work when Novartis failed?

A: Gilmore O’Neill stated that in their initial POC readout, they saw robust data with increase in total hemoglobin and robust fetal hemoglobin expression, different from Novartis, and their unique approach of combining AsCas12a effector CRISPR enzyme with targeting a different region of the HBG1/2 promoter.

Q: For the presentation at EHA, is that a late-breaking presentation?

A: Baisong Mei stated it is a normal oral presentation.

Q: Hey, great. Thanks so much for taking the question. And look forward to the data update next month. So, I guess, I was just kind of wondering about your strategy going forward. So, maybe if you can kind of walk us through how you’re thinking about next programs or priorities beyond EDIT-301?

A: Gilmore O’Neill said the large part of their discovery focus is on in vivo, and they are looking beyond HSCs to other tissues.

Q: I guess, I’ll expand a bit on the last question on the path towards registration for EDIT-301. 20 patients is a pretty substantial cohort size in sickle cell disease. So, do you have any sense of how many patients’ worth of data you will need for a registrational filing?

A: Baisong Mei said they need to align with regulatory agencies on the number of patients for registration.

Q: I guess, I’ll expand a bit on the last question on the path towards registration for EDIT-301. 20 patients is a pretty substantial cohort size in sickle cell disease. So, do you have any sense of how many patients’ worth of data you will need for a registrational filing?

A: Baisong Mei said they need to align with regulatory agencies on the number of patients for registration.

Q: I have a couple of questions for you guys. So, with the appeal litigation pending, what does it mean for company such as CRISPR Vertex that already filed a BLA for exa-cel that utilize CRISPR/Cas9 from your IP perspective. They don’t have a license from you or the Broad and could potentially launch the product before we know the outcome of the appeal. So any insight here would be helpful.

A: Gilmore O’Neill said they anticipate the judgment in early to mid-2024 and are confident they prevail, and they have a portfolio of IP not subject to interference covering product in development using Cas9 for human therapeutics.

Q: I guess, I’ll expand a bit on the last question on the path towards registration for EDIT-301. 20 patients is a pretty substantial cohort size in sickle cell disease. So, do you have any sense of how many patients’ worth of data you will need for a registrational filing?

A: Baisong Mei said they need to align with regulatory agencies on the number of patients for registration.

Q: This is Ernie Rodriguez for Phil. Thanks for taking my question. On the on the sickle cell program, have you met with the FDA to gain better visibility on the regulatory path? And then, a second question on the TDT program. For the year-end update, would that include only the sentinel patients that you initially dosed, or will you disclose additional patients? And if you will be disclosing additional patients, are you planning on reporting when you switch from sentinel dosing to parallel dosing?

A: Baisong Mei said they have engagement with FDA and expect to have data by year-end more than sentinel patients and will disclose good initial data by year-end.

Q: I guess, I’ll expand a bit on the last question on the path towards registration for EDIT-301. 20 patients is a pretty substantial cohort size in sickle cell disease. So, do you have any sense of how many patients’ worth of data you will need for a registrational filing?

A: Baisong Mei said they need to align with regulatory agencies on the number of patients for registration.

Q: I have a couple of questions for you guys. So, with the appeal litigation pending, what does it mean for company such as CRISPR Vertex that already filed a BLA for exa-cel that utilize CRISPR/Cas9 from your IP perspective. They don’t have a license from you or the Broad and could potentially launch the product before we know the outcome of the appeal. So any insight here would be helpful.

A: Gilmore O’Neill said they anticipate the judgment in early to mid-2024 and are confident they prevail, and they have a portfolio of IP not subject to interference covering product in development using Cas9 for human therapeutics.

Q: I guess, I’ll expand a bit on the last question on the path towards registration for EDIT-301. 20 patients is a pretty substantial cohort size in sickle cell disease. So, do you have any sense of how many patients’ worth of data you will need for a registrational filing?

A: Baisong Mei said they need to align with regulatory agencies on the number of patients for registration.

Q: This is Rob on for Madhu. We were just wondering how should we think about forward OpEx, particularly forward OpEx, particularly R&D, given -- forward OpEx, particularly spending in regard to transfusions versus follow-up?

A: Michelle Robertson said about half of their spend is on the RUBY and TDT trials and they don't expect an enormous increase quarter-over-quarter.

Q: This is Benjamin on for Joel. Thanks for taking our questions. Looking across other late-stage products in sickle cell and TDT, it appears that data sets of 30 patients could support approval. So, with Editas being on track to dose 20 patients by year-end, how quickly do you think you’ll be able to secure the necessary data to support regulatory approvals?

A: Baisong Mei said they are positive about the momentum and will sit with regulators to agree on data needed for approvals.

Q: So, I had the same question as Steve, but maybe a different way of asking. What percentage of sickle cell patients with hereditary persistence of fetal hemoglobin have mutations along the globin locus versus the BCL11A locus?

A: Baisong Mei said they do not have all specifics but mentioned the difference between HPFH with promoter region mutation and BCL11A's impact on multiple cells