A new clinical trial starts for achondroplasia

Therachon announced on the 14th February 2018 to have started the phase 1 of the clinical trial for TA-46.

Phase 1 is known to be the “First-in-Man” trial. TA-46 is a soluble FGFR3 discovered by Elvire Gouze team, which passed to Therachon biotech for development aiming the treatment of children and adolescents with achondroplasia. The company is confident that this drug as potential to have a significant therapeutic impact by helping to restore normal bone growth and may also reduce specific complications related to achondroplasia, as foramem magnun stenosis. The results of the pre-clinical studies, with the mice model,  were very promising: the molecule showed to be able to prevent the excessive activation of the mutated FGFR3 in achondroplasia. TA-46 acts by binding to FGFR3 natural ligands which can prevent the over-activating the mutated receptor, FGFR3.

X-ray radiographs illustrating treatment effect on skeletal growth. Credit: Sci. Transl. Med. 5, 203ra124 (2013)

Therachon stated that TA-46 has not shown any dose limiting safety or tolerability issues in non-clinical safety studies, which is a point of major relevance to advance to a clinical trial with children.

At this stage in Phase 1, approximately 70 healthy adults will receive TA-46, during which it will be evaluated the safety of this compound. In future phases, phase 2 and phase 3, TA-46 will be evaluated in children with achondroplasia by a weekly subcutaneous injection, that is expected to help restore normal bone growth by directly targeting the genetic cause of this condition.

The duration of this phase 1 was not announced, but typically phase 1 can take several months to be completed and then, all the data must be evaluated prior to advance to the following phases.

Here you can read the press release from Therachon on this news.