Novartis Cancels Plans for Clinical Trials of Gene Therapy OAV201

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Novartis announced it is scrapping plans to move OAV201, an investigational gene therapy for Rett syndrome, into clinical trials.

“The totality of the pre-clinical data [on OAV201] does not support a path forward to human clinical trials,” according to the company’s third quarter report for this year.

Novartis is “exploring other options for this indication,” according to the report.

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Nearly all cases of Rett syndrome are caused by mutations in the gene MECP2. This gene provides instructions for making a protein with the same name, which plays an important role in the development and health of brain cells.

The overall intent of gene therapy for Rett syndrome is to deliver a healthy version of the MECP2 gene to the body’s cells, thus allowing the cells to produce a functional version of the MeCP2 protein.

OAV201 was designed to do this using a specifically engineered viral vector called AAV9. The gene therapy was developed originally by AveXis under the name AVXS-201; AveXis has since been acquired by Novartis and rebranded Novartis Gene Therapies.

Novartis had filed an application with the U.S. Food and Drug Administration (FDA) asking for permission to start clinical testing of OAV201. However, that application was withdrawn in 2019 after reports surfaced that data manipulation had led to inaccuracies in the company’s FDA application for another gene therapy for a different disease — Zolgensma, a gene therapy that has since been approved for spinal muscular atrophy.

These reports prompted Novartis to conduct additional preclinical work on OAV201, including new animal studies and other quality control analyses, with the stated goal of having a better data package to present to the FDA.

Novartis announced that this preclinical work had been mostly completed late last year, and in February the company issued a letter to the Rett community saying it expected to again ask the FDA for permission to start clinical testing before the end of this year.

However, with all preclinical testing now completed, the company has made the judgment that the data do not support moving OAV201 into clinical testing in human participants. The specific data that led to this decision have not been disclosed.