Roche discontinues Phase III clinical study of antisense RNA therapy tominersen

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In response to Huntington's disease, Roche signed a patent license agreement with Ionis as early as 2017 for a new antisense therapy for proteins related to Huntington's disease.

Roche said that after the independent data committee "recommended the potential benefits and risk status of study participants based on investigating therapies," the company has stopped the administration of the third phase clinical study of the antisense drug tominersen for huntingtin and mutants.

Roche obtained the license of tominersen (formerly IONIS-HTTRx) in 2017, and paid Ionis Pharmaceuticals a US$45 million license fee.

Roche also stopped an open-label extension study called GEN-EXTEND, which used the same high-dose and low-dose protocol.

Roche pointed out that the committee's decision did not involve new security risks.

Tominersen is an anti-nucleic acid drug that binds to Huntington mRNA and degrades this mRNA through the RNA degradation system, thus reducing the expression of toxic proteins, which may change the course of the disease.

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