Healthcare Business

Why Pfizer Is Getting Deeper Into Gene Therapy

Pfizer Inc. (NYSE: PFE) is getting deeper into gene therapy. The U.S. drug giant has acquired a 15% stake in Vivet Therapeutics. On top of the investment in the French company, Pfizer also now has an exclusive option that would allow it to take full ownership of the company.

Vivet’s experimental gene therapy is currently targeting a rare and potentially life-threatening genetic disorder called Wilson disease. Pfizer paid $51 million for the stake in this private company, and it would have to pay about $636 million to buy the whole company, after factoring in clinical and regulatory milestone payments.

The overall investment here is rather small for Pfizer on the surface, when considering its market cap is $233 billion. That said, Pfizer is not new to gene therapy investing.

Pfizer has a collaboration with Spark Therapeutics Inc. (NASDAQ: ONCE) in hemophilia. Roche reached an agreement in 2019 to acquire Spark Therapeutics for about $4.3 billion.

Pfizer has a more recent deal with Sangamo Therapeutics Inc. (NASDAQ: SGMO). This partnership was a mere $12 million initial investment to target amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration.

Wilson disease is a rare, chronic and potentially life-threatening liver disorder of impaired copper transport that causes serious copper poisoning. It can be fatal, and the current treatments are less than cures, short of a liver transplant. According to the National Organization for Rare Disorders, there are about 2,000 to 3,000 diagnosed cases of Wilson disease in the United States, but one estimate showed that there may be as many as 9,000 people who may be affected.

While the current Vivet target is Wilson disease, Vivet is also said to be advancing liver-directed gene therapy programs for progressive familial intrahepatic cholestasis for bile excretion defects and citrullinemia for defects in the urea cycle.

Gene therapy may become the next frontier for drug companies seeking to make billions of dollars improving or even curing certain diseases. Treatments may run up to $1 million or more, so there are some ongoing cost issues that leave a lot of upside, but also come with regulatory risks, in a climate where the U.S. government and other governments have been pushing back against outrageous drug and treatment costs.

One analyst speculated in February about which gene therapy companies would be the most valuable as potential buyout candidates after the Spark transaction.

Shares of Sangamo traded down 2% at $9.54 Wednesday afternoon. Pfizer shares were down fractionally at $41.95 on a light volume trading day.

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