When Editas CEO Gilmore O'Neill took the reins of the CRISPR gene editing company, he acknowledged that one of the draws was the sizeable cash runway available. But less than six months into the job, the former Sarepta R&D chief is getting a harsh reminder that money isn’t everything.
New phase 1/2 of Editas’ lead asset EDIT-101 did not suggest a broad enough response, forcing the company to shelve its development and seek a partner, according to a company announcement Thursday. Among 14 patients with leber congenital amaurosis 10 (LCA10), a severe eye disease, three had clinically meaningful improvements in corrected vision. Two of the three patients were homozygous for the IVS26 mutation, representing the only baseline characteristic the company identified for identifying an ideal patient population.
The problem is that focusing only on patients who are homozygous for the mutation whittles down an already-small LCA10 patient population—some 1,500 in the U.S.—down to about 300. As a result, the company says it will no longer pursue development independently and is looking for a partner. Until then, the company is pausing enrollment in the BRILLIANCE trial.
No matter how it’s spun, the results are a gut punch to Editas as it tries to keep pace in a fastly burgeoning gene editing field. The company has faced persistent changes atop the executive team and investors have been clamoring for more data to prove its editing concept works.
Thursday's news did nothing to assuage fears that the company is losing ground, with shares down more than 17% shortly after the markets opened, from $12.25 to $10.15 per share.
In a call with investors, Gilmore addressed the decision to pursue such a rare disease in the first place, given the small market size even if the therapy had proved more successful.
“I think the original rationale was that that was actually a reasonable population, a commercially viable patient population,” Gilmore said. “Nevertheless, when you actually look at the data...that patient population is obviously substantially reduced.” He went on to contend that the company is competitive in other disease areas that are seeing initial gene therapy approvals, like sickle cell and beta-thalassemia.
As for who the company plans to partner with on EDIT-101, Gilmore said the ideal match was a company with a track record of working on gene therapies directed at retinal diseases. When asked if this pause will result in layoffs, a spokesperson for Editas said that the company has “other ocular programs in the pipeline heading towards IND enabling studies and ultimately the clinic” that the team will work on.
Of those programs, first on the docket is EDIT-103, a treatment for patients with rhodopsin-associated autosomal dominant retinitis pigmentosa. Gilmore said the zygosity effect seen with EDIT-101 shouldn’t apply to the 103 program given it's already focused on autosomal dominant patients.
Gilmore expects the decision to shelve EDIT-101 will result in cost savings, but he did not have figures available. As of the end of September, Editas had $478.5 million in cash on hand, roughly $50 million less than three months prior.