Therachon doses the first subject in its Phase 1 trial

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14.02.2018
clinical trial

Therachon AG focused on rare genetic diseases announces the dosing of first subject in phase 1 clinical trial evaluating its lead pipeline candidate TA-46, a novel protein therapy for the potential treatment of achondroplasia, the most common form of short limb dwarfism.

The Basel biotechnology company, Therachon has begun its phase 1 clinical trial for TA-46. 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.

“We are excited to announce this important milestone for the company,” said Luca Santarelli, Therachon’s CEO. “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, 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.”

About Achondroplasia and TA-46
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 therefore developing TA-46, which 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. TA-46 works by normalizing the overactive FGFR3 signalling 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).

(press release)

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