The biopharma company THX Phamra and Biocodex, an independent international pharmaceutical group, have entered into a strategic licencing agreement in order to advance the development of two drug candidates in three rare diseases.
The three diseases, Batten disease with Batten-1, Gaucher disease and Niemann-Pick disease type C with TX01, each have a high unmet need. They typically affect children and have a major impact on quality of life as well as life expectancy.
The conditions have either no, or very limited, therapeutic options, making each development programme a considerable scientific, medical and human progress.
Biocodex will hold two licences: an exclusive global license for the development and commercialisation of Batten-1 and a regional exclusive license covering the United States and Canada for the development and commercialisation of TX01.
Batten-1 is a drug candidate developed by THX Pharma for the treatment of juvenile Batten disease (CLN3) and TX01 is a new formulation of an already approved compound for the treatment of Gaucher disease and Niemann-Pick disease type C.
Under the terms of the agreement, THX Pharma will receive total payments of up to €173m, including a €12m upfront payment and up to €161m in development and sales milestone payments, as well as tiered double-digit royalties on net sales.
As part of the collaboration, THX Pharma will lead the clinical development of the programmes, with financial and scientific support from Biocodex.
The latter will be responsible for compassionate use programmes, market access and commercialisation activities in the territories covered by each license.
The shared ambition of Biocodex and THX Pharma is to advance therapeutic solutions where unmet needs remain significant, drawing on science, partnership and ongoing dialogue with all stakeholders, including healthcare professionals and patient advocacy groups.
Mathieu Charvériat, President and CEO of THX Pharma, stated: "This strategic agreement with Biocodex represents a major step forward and a significant value-creation milestone for THX Pharma."
"It validates the scientific and strategic value of our assets by partnering with a leading French pharmaceutical company with strong clinical development capabilities, recognised industrial and regulatory expertise and a solid international commercial presence in the field of rare neurological diseases."
"Moreover, this alliance with Biocodex enables the launch of a more ambitious project, both in Europe and in the United States, thereby maximising our chances of success for the benefit of patients."
"Together, we will bring forward innovative therapeutic solutions in three rare diseases affecting thousands of people worldwide: Batten disease, Niemann-Pick disease type C and Gaucher disease."
Nicolas Coudurier, CEO of Biocodex, added: "By partnering with THX Pharma, Biocodex is fully aligned with its innovation strategy dedicated to rare diseases with high unmet medical needs."
"Building on the experience gained with Diacomit in Dravet syndrome, we were convinced by THX Pharma’s scientific expertise and the strong alignment with our values of innovation in the service of health."
"We are also pleased to continue this commitment alongside dedicated research organisations, such as Beyond Batten Disease Foundation, in the interest of patients and their families."