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TherAchon, the start-up born out of Université Côte d'Azur laboratories, acquired by Pfizer for 340 million dollars

TherAchon, a start-up created in 2014 by Elvire Gouze, Inserm researcher at the Valrose Institute of Biology, is developing innovative therapies to restore growth and normal bone proportion in children with dwarfism. The company was recently purchased by the pharmaceutical giant Pfizer for 340 million dollars. An exceptional success for a company whose scientific management is still located in the research buildings of Université Côte d'Azur.

Publication : 13/05/2019
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Achondroplasia is a genetic disease in which a point mutation in the gene coding for FGFR3 causes delayed bone growth and cartilage malformation. It affects about one child in every 15,000 births and is the major cause of dwarfism. It is characterized by small, disproportionate limbs and other skeletal malformations leading to significant comorbidities such as spinal cord compression and reduced life expectancy. There are approximately 200,000 achondroplasia patients in the world, 20,000 in Europe and between 3,000 and 4,000 in France.

Inserm researcher, Elvire Gouze, has been working on achondroplasia since 2009, first at the Mediterranean Center of Molecular Medicine and since 2016 at the Valrose Institute of Biology. With her team, she discovered a soluble receptor that acts as a decoy and keeps the aberrant protein from activating. In achondroplastic transgenic mice, this treatment allows normal growth and especially prevents the most serious complications.
In 2013, after four years of research, the results were published in Science Translational Medicine. Eight months later, with the help of Versant Ventures and Inserm Transfert Initiative, Elvire Gouze created TherAchon, a biotechnology company that aims to develop a treatment for this type of dwarfism.

In 2014, after one year of existence, TherAchon raised 35 million euros through private investors. OrbiMed and New Enterprises Associates (NEA) acquired a stake, in addition to Inserm Transfert Initiative and Versant Ventures, to allow TherAchon to set up clinical trials. In July 2018, Cowen Healthcare Investments and Pfizer Ventures added a 60 million euro investment.
Today TherAchon's clinical trials are in Phase 1, which means that the molecule is tested on volunteer patients. Pfizer decided to purchase the company at this stage of development, which is extremely rare.
The purchase of TherAchon by Pfizer opens up new opportunities for developing a possible treatment and giving hope to patients suffering from achondroplasia.