
Rare Genetic Disease Drug Developer
VCBeat (WeChat Official Account: vcbeat) has learned that pharmaceutical giant Pfizer (NYSE: PFE) recently announced the acquisition of the Swiss biotechnology company Therachon. Under the terms of the agreement, Pfizer will acquire all shares of Therachon for $340 million. Additionally, Pfizer will assume contingent consideration of up to $470 million, payable depending on the research and development and commercialization progress of Therachon’s rare disease therapeutic, TA-46.
Therachon is a Swiss biotechnology company dedicated to the treatment of achondroplasia and short bowel syndrome (SBS). The company is developing TA-46, a protein-based therapeutic designed to promote bone growth in patients with hereditary achondroplasia.
Achondroplasia is an autosomal dominant genetic disorder. It is caused by mutations in the fibroblast growth factor receptor 3 (FGFR3) gene, resulting in short stature and disproportionate limb shortness, accompanied by debilitating skeletal, neurological, and otolaryngological complications. Currently, there are no approved pharmacological treatments; the only available intervention for achondroplasia is limb-lengthening surgery. This highly invasive surgical procedure addresses height deficits but does not resolve the complications associated with achondroplasia.
TA-46 is a recombinant, soluble protein therapeutic that directly modulates FGFR3 function. TA-46 binds to the natural ligands of FGFR3, fibroblast growth factors (FGFs), and prevents excessive activation of mutant FGFR3 by blocking ligand-induced overactivation. Therachon is developing a subcutaneous formulation of TA-46 for the treatment of pediatric and adolescent patients. The drug has completed Phase I clinical trials, and orphan drug designation has been granted by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).
Mikael Dolsten, Chief Scientific Officer and President of Worldwide Research, Development and Medical at Pfizer, stated, “Whether within or outside our laboratories, Pfizer’s strategic focus has always been to drive the further advancement of global medical technology. Through the acquisition of Therachon, we aim to leverage Pfizer’s leading scientific expertise and development capabilities to accelerate the delivery of this promising therapy for patients with achondroplasia.”
Through this transaction, Pfizer aims to compete with other companies treating achondroplasia. Pfizer’s most formidable competitor is the U.S.-based BioMarin Pharmaceutical, which is conducting Phase III trials of the peptide drug vosoritide. Vosoritide is designed to improve bone growth by inhibiting the intracellular activity of the mutated cell-surface protein.
Therachon is not only focused on therapeutic solutions for achondroplasia but also provides solutions for short bowel syndrome, with its product currently in Phase II clinical trials. It is reported that the Swiss company plans to establish a new entity dedicated to developing treatments for short bowel syndrome before Pfizer takes over, and Pfizer will hold equity stakes in the newly spun-off company.
(Compiled by: Wang Yue)