(News Bulletin 247) – The company has obtained approval from the Food and Drug Administration on the design and evaluation criteria of the pivotal phase III study for its potential treatment against Batten disease. Its stock opened more than 65% higher.

Theranexus marks an important milestone for its great drug candidate. The biopharmaceutical company announced on Tuesday that it had obtained the agreement of the Food and Drug Administration (FDA), the American health authority, 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 first symptom of the juvenile form, a progressive loss of vision, appears between the ages of 4 and 6. It is followed by personality changes, behavioral problems and learning difficulties. Seizures appear in the 2 to 4 years after the onset of the disease. Gradually, patients decline physically and mentally,” explains the company.

The pivotal 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.

Twelve months of financial visibility

Theranexus expects to begin recruiting patients by the end of the year and the study will be conducted in parallel in several centers in the United States and Europe.

“To date, the phase III trial provides for six to nine months of recruitment and two years of treatment. We should therefore have the results of this trial on the main cohort at the end of 2026”, indicates Mathieu Charvériat, CEO of Theranexus, in an interview with News Bulletin 247.

“In parallel, a second smaller cohort of nine patients, with an open protocol [le médecin et les patients connaissent la nature du traitement administré, NDLR] will collect efficacy data every six months, with first results in late 2024. This data will be part of the efficacy evidence package that will be submitted to the FDA and EMA [l’agence européenne du médicament,NDLR] for the marketing authorization application”, adds the manager.

Mathieu Charvériat specifies that Theranexus intends to submit this marketing application to the FDA and the EMA in 2027, according to the company’s current schedule.

In the meantime, the company will have to find new sources of financing to ensure the continuity of its activities. “Our cash position, of seven million euros at the end of March, provides us 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 an agreement license with a partner who would take part of the R&D costs on his own) or a mixture of the two”, explains Mathieu Charvériat.

A commercial potential of several hundred million dollars

At the end of the process, the financial fallout for Batten-1 amounts to several hundred million dollars. “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”, explains Mathieu Charvériat.

Recall that Batten-1 has been Theranexus’ flagship drug candidate since the company chose last year to reposition itself on rare neurological diseases after having given up on finding a partner for its THN102 program intended to fight against Parkinson’s disease. .

After having been reserved for a long time, Theranexus shares opened around 11:30 a.m. with a jump of nearly 66% on the Paris Bourse, to 2.30 euros. On Friday, the micro-cap (5.8 million euros) ended up 15%.