Theranexus and Inserm Transfert have entered into an agreement to work on the development of candidates for the treatment of neurological diseases such as Batten disease, announce the two companies.
“This landmark agreement fits perfectly with our strategy to develop drugs blocking the progression of rare neurological diseases such as Batten-1, our Phase I/II agent in Batten disease,” said Franck Mouthon, CEO of Theranexus, in a press release.
Under this three-year agreement, Theranexus will have preferential and early access to technologies related to the treatment of neurological diseases developed by Inserm. The companies will also share their expertise, and Theranexus will have the opportunity to grant licenses for the development of the products identified through this partnership.
The company said it aims to have a first ingredient in its pipeline in about 18 months.
Partners aim to develop new treatment for Batten disease
The project will particularly focus on oligonucleotide therapies – drugs that use small chains of nucleic acids capable of modulating gene activity. Nucleic acids are the building blocks of DNA and RNA.
“We are delighted to collaborate with Inserm, a big name in French and global biomedical research, through its technology transfer office, Inserm Transfert. Thanks to this agreement, we have access to Inserm research to identify and characterize new targets and antisense RNA or oligonucleotide candidates for the treatment of patients with neurological diseases,” said Mouthon.
Pascale Augé, president of the management board of Inserm Transfert, said the company “is proud to launch this alliance” with the French biopharmaceutical company.
“It will be an opportunity to undertake joint actions for the benefit of Inserm scientists and the development of their innovative projects,” said Augé.
Theranexus was already developing an experimental treatment for Batten disease called Batten-1, which contains a compound called miglustat. The therapy aims to reduce the toxic buildup of fatty molecules that causes the symptoms of Batten disease.
Research presented at a recent scientific conference showed that the therapy was effective in preclinical models of the disease.
A phase 1/2 trial (NCT05174039) is underway to evaluate the safety and pharmacological properties of Batten-1 in adolescents and adults with juvenile Batten disease. The trial completed participant recruitment 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.
