Bristol-Myers Squibb Licensing Gene Therapies in $2.3B Deal

Human chromosomes (Genome.gov)

6 April 2015. Drug maker Bristol-Myers Squibb is licensing gene therapies from biotechnology company uniQure N.V. in a deal with a total potential value of $2.3 billion, including an equity stake in uniQure. The agreement gives Bristol-Myers Squibb exclusive access to as many as 10 disease programs being developed by uniQure, including a treatment for congestive heart failure now in development.

The biotech uniQure is developing gene therapies based on a platform with a packaging technique the company calls gene cassettes that combines an engineered gene with substances that direct the gene to the desired organ or tissue. The gene packages are then delivered with adenoviruses: benign, naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome or cause disease, and generate a mild immune response. uniQure also has a manufacturing process harnessing insect cells for its gene therapies, which is part of the deal with Bristol-Myers Squibb.

The lead uniQure product is Glybera, a gene therapy designed as a one-time treatment for lipoprotein lipase deficiency, a rare genetic disorder where a person lacks the lipoprotein lipase enzyme needed to break down fat molecules, causing fat to build up in the blood. The company says Glybera is the first gene therapy approved for marketing in Europe, and is in late-stage clinical trials in the U.S.

The new agreement gives Bristol-Myers Squibb exclusive access to uniQure’s gene therapies for cardiovascular disorders, including one to treat congestive heart failure. This uniQure therapy aims to restore the heart’s ability to synthesize S100A1, a calcium binding protein that regulates heart function, especially for people where the heart muscle does not contract effectively and less blood is pumped to the body. uniQure’s S100A1 therapy is currently in preclinical research and development.

The agreement likewise gives Bristol-Myers Squibb access to uniQure gene therapies addressing 9 more conditions. Under the deal, uniQure is responsible for the discovery phase, as well as manufacturing of products developed under the partnership. Bristol-Myers Squibb will commercialize all products from the collaboration, take the lead on development and regulatory activities, and be responsible for research and development costs.

The deal gives uniQure an initial payment of $50 million, with another $15 million upon selection by Bristol-Myers Squibb of 3 more collaboration targets in the first 3 months. In addition, Bristol-Myers Squibb is making an equity investment equal to 4.9 percent of uniQure stock, valued at about $32 million, with up to another 5 percent stake taken by the end of 2015.

The agreement gives uniQure up to $254 million in regulatory and milestone payments for the S100A1 therapy, as well as up to $217 million for each the 9 following gene therapies developed under the collaboration, bringing the total potential value to uniQure to more than $2.3 billion. Bristol-Myers Squibb may also obtain another 10 percent equity stake in uniQure, based on the disease programs undertaken by the partnership.

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