Editas halts lead CRISPR program after efficacy data underwhelm

Editas Medicine will put a wrap on its lead clinical program after reporting disappointing results from a Phase I/II trial.

The biotech, one of the first to try to use CRISPR gene editing tech to develop new drugs, said Thursday morning that only three of 14 patients responded to the therapy, known as EDIT-101. Execs will try to find a partner for the program, which is designed to treat an inherited form of blindness called CEP290-mediated LCA10.

Investors appeared disappointed with the news, as Editas shares $EDIT fell about 18% in early Thursday trading.

Researchers were testing EDIT-101 in both adults and children at a few different dosage levels. Data from Thursday’s update came from 14 patients — 12 adults and two kids — 11 of whom received the experimental drug at least six months out. Additionally, Editas had at least a year of follow-up data for seven of the 14.

In analyzing the three responders’ results, the biotech found that two possessed the homozygous version of the disease, or two copies of an affected gene. These were the only two homozygous patients in the study. Only one of the other 12 patients, all heterozygous (one gene) individuals, achieved a “clinically meaningful” response.

Given that homozygous LCA10 is much rarer than its heterozygous counterpart, and it’s the only population where a possible effect could prove conclusive, Editas said a final product would not be commercially viable. Only about 300 people in the US suffer from homozygous disease, so Editas decided to halt development and seek a partner.

Analysts peppered Editas’ execs on an investor call Thursday morning, trying to pick out reasons if EDIT-101 might be salvageable and who the biotech might seek for a partner, among other topics. New CEO Gilmore O’Neill largely gave few specifics when it came to business strategy, but continued to tout Thursday’s data as proof the idea behind the drug is sound.

One analyst asked O’Neill whether the data would move Editas toward considering layoffs or a reverse merger, which O’Neill sidestepped while also throwing cold water in touting another clinical program, EDIT-301. For now, the partnership path is the only concrete step Editas is willing to talk about.

Baisong Mei

The data drop comes after a rocky few years for Editas, whose CRISPR therapy was one of the first to be tested directly in people. O’Neill is the company’s third chief executive in as many years after jumping from Sarepta in June. Chief medical officer Baisong Mei also joined this year after former CMO Lisa Michaels was abruptly fired in February.

Earlier data snapshots had indicated Editas would likely need stronger efficacy results, despite the in vivo therapy proving safe. In September 2021, two of five patients showed any sign of their vision improving, both of whom were treated with EDIT-101’s middle dose.

Some of the company’s previous partnerships also did not work out, as AbbVie scrapped an R&D alliance — originally signed by Allergan — back in August 2020. Allergan paid the biotech $90 million in 2017 to team up.

Thursday’s data also boost competitors Intellia and CRISPR Therapeutics, SVB Securities analysts wrote in a note, noting Editas thus far “has yet to execute with the speed necessary to close the gap” with the other biotechs.

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