JPM23: Editas touts skinnier pipeline after major shake-up to ride out 'unfriendly' markets

Gilmore O’Neill has certainly made an impact in his first six months as CEO of Editas Medicine, culminating in a major pipeline clear out and 20% workforce layoffs this week.

Executives noted the difficult market conditions that have plagued biotechs over the past year were a factor in the decision and said the pipeline reprioritization will free up cash during remarks at the J.P. Morgan Healthcare conference Tuesday afternoon. The company has dropped the likes of EDIT-103 for a degenerative eye condition, as well as its wholly owned multiplexed edited induced pluripotent stem cell (iPSC) derived natural killer (iNK) cell programs. The pipeline retool was announced Monday as JPM was getting underway. 

It may have been a bold move to offload so much of the company’s pipeline in one go, but Chief Financial Officer Michelle Robertson defended the decision as necessary to fund both EDIT-301 and future in-vivo research.

“Given the unfriendly state of the equity markets, we feel really comfortable that this cash extension will put us in a good position for once we get to the value inflection points,” Robertson said in the session.

Judging by CEO Gilmore O’Neill’s comments, we shouldn’t expect things to quieten down at the biotech anytime soon now that the company is working with a slimmed-down pipeline. 

“Last year was a pivotal year in Editas Medicine’s history as we reset our vision, began execution of this vision and hit multiple milestones of progress for this change,” he told the conference. “And we expect this year to be even more eventful.”

In practice, that means prioritizing EDIT-301, which consists of edited, patient-derived CD34+ hematopoietic stem and progenitor cells. The company has set a target of dosing 20 patients with sickle cell disease by the end of this year, with data from the first two patients dosed last year expected by the middle of 2023.

Dosing of the first patient in the EDITHAL trial of EDIT-301 for transfusion-dependent beta thalassemia is due this quarter, with a clinical update expected by the end of the year.

The pipeline and workforce restructuring has freed up cash to push forward earlier-stage research, with a key focus for the year being the exploration of next-generation in-vivo assets. First up will be the editing and targeting of hematopoietic stem cells.

“Why is that a good idea? Well, we have validated by in-human proof of concept the target, which is the gamma globin promoter, and the mechanism for editing [the enzyme] AsCas12a,” O’Neill said. “It enables us to reduce the magnitude of the problem to one of targeted delivery to hematopoietic stem cells in vivo. And we’ve actually started some work on that.”

With Chief Scientific Officer Mark Shearman, Ph.D., also departing the company in the shakeup, deciding on further potential targets for this in-vivo research will have to wait until the missing piece of the biotech’s C-suite puzzle is found.

“Beyond that, I really want to bring in our CSO, who will be partnering with Bruce Eaton, our CBO, and Baisong Mei, our CMO, to really think very robustly about the targets that we will bring forward,” O’Neill said.