Graphite pencils out sickle cell therapy after adverse event, cutting half of workforce loose

Graphite Bio is writing off a sickle cell therapy once heralded as a potential cure and cutting half its staff—including two executives—after a review of the business. 

The biotech is ending development of nulabeglogene autogedtemcel (nula-cel) a month and a half after halting a phase 1/2 trial due to a “serious adverse event in the first patient dosed.” The patient experienced prolonged low blood cell counts, requiring ongoing transfusion and growth factor support, which was deemed to be related to the treatment. The pause was voluntary, and Graphite reported the event to the FDA.

While Graphite will consider external partners for the program, the biotech is shifting priorities to its early-stage non-genotoxic conditioning program and hopes to develop candidates to move into the clinic, according to an after-market release Wednesday.

“We believe that gene correction is the optimal way to treat sickle cell and many other genetic diseases. However, after an extensive assessment of the nula-cel program, we made the difficult decision to discontinue nula-cel development based on the time and resources needed to resume the CEDAR study and the evolving treatment landscape for sickle cell disease,” said CEO Josh Lehrer, M.D.

Half of Graphite’s staff will be laid off as part of the corporate restructuring to reduce cash burn while the company “explores strategic alternatives,” Graphite said in the release. In a related Securities and Exchange Commission filing (PDF), Graphite said the severance costs will reach approximately $4.9 million to be recorded in the second quarter.

The filing also revealed that Chief Business Officer Philip Gutry and Chief Scientific Officer Jane Grogan, Ph.D., will leave at the end of the second quarter. 

The company had $283.5 million in cash and equivalents in the bank as of the end of 2022.

Back in January, when Graphite announced the pause on the nula-cel program, the company also indicated that a request for human testing of GPH102 in beta-thalassemia was no longer expected by mid-2024. Graphite made no mention of that therapy or the early-stage alpha-1 antitrypsin deficiency program in its latest release. 

Editor's Note: An earlier version of this story's headline said that a patient had died in the phase 1/2 trial. The study was shut down after an adverse event in the first patient dosed, not a death. Fierce Biotech regrets the error.