The University of Montpellier is contributing to the development of a drug candidate for Wolfram syndrome

[Press Release] - Published on November 4, 2026 in Press Room, University Innovation Hub (PUI), Research Hubs

The University of Montpellier has secured 325,000 euros in funding from the National Research Agency (ANR) to develop a drug candidate for the treatment of Wolfram syndrome, a rare and serious disease for which no treatment is currently available. Stemming from research conducted by the Molecular Mechanisms in Neurodegenerative Dementias (MMDN) laboratory (University of Montpellier, Inserm, EPHE) and supported by the services of theUM, the Montpellier Business and Innovation Center (BIC), and the technology transfer company SATT AxLR, the project is scientifically led by Benjamin Delprat and involves a consortium of academic and European partners, as well as the startup Sitera Pharmaceuticals.

SIT3060: A New Therapeutic Approach for Wolfram Syndrome

Wolfram syndrome is a rare genetic disorder characterized primarily by the onset of diabetes mellitus and optic atrophy, which may be accompanied by sensorineural hearing loss and other complications. The disease causes a progressive decline in patients’ health and can significantly reduce their life expectancy. To date, there is no treatment that directly targets the mechanisms underlying the disease.

Researchers at the laboratory have identified a dysfunction in the interactions between two essential cellular structures: the endoplasmic reticulum, which plays a key role in calcium regulation, and the mitochondria, which are responsible for a large portion of cellular energy production. This research, which received financial support and development assistance from SATT AxLR, led to the identification of SIT3060, a drug candidate currently being developed by Sitera Pharmaceuticals. The creation of this startup represents a significant step in commercializing the results of academic research. As part of this initiative, Sitera Pharmaceuticals received support for its establishment and development provided jointly by the Montpellier BIC and SATT AxLR. The project also received the Montpellier PUI Innovation Award in 2026, recognizing its commercialization potential and its ambition to transform a scientific discovery into a therapeutic solution.

Funding to Accelerate the Development of a Drug Candidate

The project is part of the Erdera call for proposals, which aims to accelerate the development of therapeutic solutions for rare diseases and to facilitate the transition of drug candidates into clinical trials. The 325,000-euro grant awarded by the ANR will help consolidate a consortium centered on SIT3060 by bringing together European academic laboratories, patient organizations, and the private sector.

A doctoral student will be recruited to specifically study the effects of chronic treatment with SIT3060 on vision loss in a mouse model of Wolfram syndrome. The research will also aim to identify the signaling pathway through which the observed protective effect occurs. This research will provide a better understanding of the drug candidate’s mechanisms of action and help consolidate the data needed to continue its development.

Over the course of the three-year project, the partners will seek, in particular, to validate the efficacy of SIT3060 in various preclinical models of Wolfram syndrome, with the goal of accelerating its development toward clinical trials. The research will also explore the potential of this approach for other diseases characterized by a similar communication deficit between the endoplasmic reticulum and the mitochondria.

Ultimately, the main expected outcome of the project is to validate SIT3060 as a drug candidate capable of addressing the cellular and symptomatic deficits associated with Wolfram syndrome. Such a breakthrough would represent a major hope for patients, who currently face a progressive disease for which treatment options remain limited.

“I am extremely proud and honored to coordinate this European consortium, which is expected to yield a new drug candidate for the treatment of Wolfram syndrome and related diseases. I would like to extend a special thank you to the University of Montpellier, which mobilized all the necessary resources to help secure this highly competitive funding.”

Benjamin Delprat