Theranexus obtains green light from EMA for phase III study of potential treatment against Batten disease

(BFM Bourse) – The biopharmaceutical company has obtained the green light from the European Medicines Agency on the design and evaluation criteria of the pivotal phase III study for its potential treatment against Batten disease. Theranexus obtained similar clearance from the FDA last month.

Theranexus announces a new breakthrough for its potential treatment for Batten disease. The biopharmaceutical company said on Wednesday that it had obtained the positive opinion of the European Medicines Agency (EMA) on the design (“design”) and the evaluation criteria of the pivotal phase III study (last step before potential commercialization) for Batten-1.

This drug candidate, which has been the subject of a license agreement with the Beyond Batten Disease Foundation (BBDF) since 2019, aims to treat Batten disease, a rare, fatal genetic disease of the nervous system for which it is there is currently no treatment.

The EMA’s response is aligned with the terms of the agreement obtained at the beginning of May from the Food and Drug Administration (FDA), the American health authority. The EMA has thus retained the same criteria as its American counterpart, namely visual acuity as the main criterion for phase III and the evaluation of cognitive and motor functions as secondary criteria.

Theranexus recalls that this phase III study will be randomised, double-blind on a main cohort of 60 patients aged between four and sixteen years and will have visual acuity as the primary endpoint. The secondary endpoints will notably include the evaluation of cognitive, motor and visual functions.

The company specifies that in parallel an additional, but more limited cohort of 9 pediatric patients will be treated open-label [le médecin et les patients connaissent la nature du traitement administré, NDLR] in order to represent the different ages of the target population. This secondary cohort will collect interim results every 6 months on the same efficacy and safety criteria as the main cohort of 60 patients, which will allow the company to collect clinical data early.

A potential commercialization in 2027

Theranexus expects to begin recruiting patients by the end of the year and the study will be conducted in parallel at several centers in the United States and Europe. The company plans to apply for marketing authorization in 2027 from the EMA and the FDA, before aiming for the first marketing of the product in these territories the same year.

“We estimate a peak in sales of Batten-1, between 500 million and 800 million dollars per year. It should be noted that the annual treatment costs in similar diseases are high, in the order of 500,000 to 800,000 dollars per year. year in the United States. The prevalence is 2,000 cases in the United States and Europe,” explained Mathieu Charvériat, CEO of Theranexus, in an interview with BFM Bourse last month.

Before, the company will have to find new sources of financing in the meantime to ensure the continuity of its activities. Theranexus has cash of seven million euros at the end of March, providing it with financial visibility for twelve months. “For the future, we are considering several options, either raising funds from the market or industrial partnerships (for example a licensing agreement with a partner who would take on part of the R&D costs on his own account) or a mixture of the two. “, had also specified Mathieu Charvériat to BFM Bourse.

On the Paris Stock Exchange, this validation from the European health authority to begin phase III trials in Europe is a source of hope. Theranexus stock jumped 12.2% to 1.98 euro, around 10:00 a.m. Wednesday morning after a peak of +30.7% around 9:20 a.m. This progress confirms a good stock market performance initiated last month after the FDA’s agreement on the design and evaluation criteria of the pivotal phase III study for its potential treatment against Batten’s disease. On May 9, Theranexus shares soared 74.5% after having been reserved for a long time on the rise, in reaction to the announcement of this important milestone for the company.

Sabrina Sadgui – ©2023 BFM Bourse