BASEL, Switzerland, February 14, 2018 – Therachon AG, a biotechnology company focused on rare genetic diseases, today announced the dosing of the first subject in its Phase 1 trial for TA-46, a soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) ligand trap. TA-46 works by normalizing the overactive FGFR3 signaling pathways that underlie bone abnormalities associated with achondroplasia, the most common form of short limb dwarfism. TA-46 is being developed as a weekly subcutaneous drug for children and adolescents living with the disease. The investigational therapy has received Orphan Drug Designation from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).
“We are excited to announce this important milestone for the company,” said Luca Santarelli, M.D., Therachon’s Chief Executive Officer. “Our goal is to develop medicines for rare, genetic diseases that currently have no available treatments. TA-46 is a first-in-class recombinant protein therapy in development for children and adolescents with achondroplasia with the potential of having significant therapeutic impact by directly targeting the genetic cause of the condition.”
“We are eager to quickly move into the next phase of development, where we aim to demonstrate the unique disease modifying properties of TA-46 in children with achondroplasia,” said Christian Meyer, M.D., Ph.D., Therachon’s Chief Medical Officer. “We believe TA-46 has the potential to improve anatomical proportions and thereby prevent the most severe disabling complications of the condition.”
The randomized, placebo-controlled, double-blind trial is designed to evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of TA-46 in approximately 70 healthy male and female volunteers. The trial will take place in the Netherlands.
TA-46 directly restores the physiological function of the endogenous fibroblast growth factor receptor 3 (FGFR3) by acting as a ligand trap and thus modulating the activation of the receptor. Achondroplasia is caused by mutations of FGFR3 that result in excessive activation of this receptor, which acts to inhibit normal bone growth. TA-46 prevents the excessive activation of the mutated FGFR3 by binding to its natural ligands and preventing them from over-activating the receptor. TA-46 has not shown any dose-limiting safety or tolerability issues in non-clinical safety studies to date. TA-46 will be evaluated in children who would receive weekly subcutaneous injections to help restore normal bone growth.
Achondroplasia is a rare, genetic condition which affects approximately one in 15,000 children and is caused by a genetic mutation of the FGFR3, which stunts child bone growth. This condition is accompanied by life-altering neurological, orthopedic and ear, nose and throat complications. Currently, the only available treatment option for achondroplasia is limb lengthening surgery, an extremely invasive surgical procedure that addresses height but not specific achondroplasia-associated complications.
Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Therachon is committed to translating the promise of its science into new treatments for patients with high unmet medical needs. For more information, visit WWW.THERACHON.COM.