Catalent will produce TA-46, a soluble version of a protein called fibroblast growth factor receptor 3 (FGFR3) that is mutated in the disease achondroplasia, also known as short-limbed dwarfism.
The idea is that TA-46 acts as a decoy and prevents fibroblast growth factor (FGF) from binding the mutated receptor, thereby allowing normal growth to resume. Catalent will produce the drug for a Phase I trial due to start next year.
The US CMO has been involved in the project for more than a year, using its GPex expression technology to produce a range of TA-46 variants from which Therachon selected the most promising preclinical candidate.
Manufacture of TA-46 for trials will take place at Catalent’s biomanufacturing facility in Madison, Wisconsin. The CMO is currently increasing production capacity at the site, adding two 2,000 liter single use bioreactors in an expansion due to be completed in October.
At present there are no treatments for achondroplasia, however, Therchon is not the only firm with a candidate in the pipeline.
BioMarin Pharmaceuticals has vosoritide (BMN-111), an analog of C-type natriuretic eptide (CNP), in a Phase III trial. Denmark based Ascendis Pharma A/S has TransCon CNP, which is a sustained-release prodrug of CNP.
US-based BioClin Therapeutics Inc is also developing an achondroplasia candidate. Like Therachon, the Californian biotech is targeting FGFR3.