Pfizer this morning added to its methodical push into the gene therapy field, paying $51 million for a 15 percent stake in Vivet Therapeutics and the option to buy the Parisian biotech in the future.
Vivet is developing an experimental gene therapy, VTX-801, for the rare genetic disorder Wilson’s disease, which causes a potentially deadly buildup of copper in the liver and other organs. In a statement, Pfizer (NYSE: PFE) noted that the current available medicines for the disease—drugs like trientine (Spyrine) taken chronically to suppress the buildup of copper—have “sub-optimal efficacy or significant side effects” for many patients. The idea is that VTX-801, through a single infusion, would help patients’ bodies properly break down and dispose of copper.
Pfizer can exercise an option to buy Vivet once it sees data from an early stage study of VTX-801. Vivet could get another $635.8 million if Pfizer pulls the trigger and if VTX-801 gets to market and hits certain sales targets.
The deal is the latest in a recent string of investments by large biopharma companies in gene therapy, which offers the potential for a long-lasting, if not permanent effect through a one-time treatment. The field has gone through decades of ups and downs, but has now proven that it can develop approved marketed products. Several are on the way. These investments are betting that gene therapy can answer its most significant remaining questions: that their therapeutic effects will last long enough to justify their high price tags, and that they can succeed commercially.
Big pharmaceutical companies are opening their wallets to get into the gene therapy field. In January, Johnson & Johnson (NYSE: JNJ) agreed to pay $100 million to MeiraGTx (NASDAQ: MGTX) for rights to experimental gene therapies for inherited forms of vision loss. In February, Biogen (NASDAQ: BIIB) inked an $800 million deal to acquire Nightstar Therapeutics, a developer of gene therapies for eye diseases. That same month, Roche paid $4.8 billion for gene Spark Therapeutics (NASDAQ: ONCE), which owns the only approved gene therapy in the U.S., a treatment for inherited vision loss called Luxturna. Investors are also putting more and more cash into gene therapy companies, bullish on the field’s future prospects despite the outstanding commercial questions.
The Vivet investment marks the latest gene therapy move, meanwhile, for Pfizer. It already has partial rights to a hemophilia B gene therapy being developed by Spark. Pfizer is also one of several companies developing a Duchenne muscular dystrophy gene therapy via its 2016 buyout of Bamboo Therapeutics. And it has an alliance with Emeryville, CA-based 4D Molecular Therapeutics to develop next-generation gene therapy delivery tools for heart diseases.