Aim of New ‘Flagship Agreement’ Is Treatment for Batten Disease, Others

Theranexus, Inserm team up to take on rare neurological diseases

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Two people shake hands after making a deal.

Theranexus and Inserm Transfert have entered an agreement to work toward the development of treatment candidates for neurological disorders such as Batten disease, the two companies announced.

“This flagship agreement is perfectly in line with our strategy to develop medicinal products blocking the progression of rare neurological diseases such as Batten-1, our Phase I/II agent in Batten disease,” said Franck Mouthon, chairman and CEO of Theranexus, in a press release.

Under the three-year agreement, Theranexus will have preferential early access to technologies related to neurological disease treatment that are developed by Inserm. The companies also will share expertise, and Theranexus will have the opportunity to license for development products that are identified through this partnership.

The company said it is aiming to have a first ingredient enter its pipeline in about 18 months.

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The project will particularly focus on oligonucleotide therapies — medications that use small chains of nucleic acids that can modulate the activity of genes. Nucleic acids are the building blocks of DNA and RNA.

“We are delighted to collaborate with Inserm, a leading name in French and global biomedical research, through its technology transfer office, Inserm Transfert. Thanks to this agreement, we have access to Inserm’s research for identifying and characterizing novel targets and RNA or antisense oligonucleotide candidates for treating patients with neurological diseases,” Mouthon said.

Pascale Augé, chairman of the Inserm Transfert executive management board, said the company “is proud to launch this alliance” with the France-based biopharmaceutical.

“It will provide an opportunity to undertake joint actions for the benefit of Inserm’s scientists and the development of their innovative projects,” Augé said.

Theranexus already was developing an experimental treatment for Batten disease called Batten-1, which contains a compound called miglustat. The therapy aims to reduce the toxic accumulation of fatty molecules that causes symptoms of Batten disease.

Research presented at a recent scientific conference showed the therapy was effective in preclinical models of the disease.

A Phase 1/2 trial (NCT05174039) is ongoing to assess the safety and pharmacological properties of Batten-1 in  adolescents and adults with juvenile Batten disease. The trial finished enrolling participants earlier this year.

Theranexus has announced plans for a potentially pivotal Phase 2/3 trial to test the therapy, which is expected to launch next year.