January 24, 2018 / 10:27 PM / a month ago

Spark licenses blindness gene therapy rights outside U.S. to Novartis

(Reuters) - Spark Therapeutics Inc (ONCE.O), whose breakthrough gene therapy to treat a rare form of blindness was approved by U.S. regulators in December, said on Wednesday that it had licensed rights to the drug outside the United States to Switzerland-based Novartis AG (NOVN.S).

Philadelphia-based Spark said it will keep U.S. rights to Luxturna, or voretigene neparvovec, which is the first approved gene therapy for an inherited disease. It plans to launch the one-time treatment in March, charging an unprecedented $850,000.

Spark will receive $105 million from Novartis and up to another $65 million in milestone payments based on near-term European regulatory approval and initial sales in certain markets. Spark is also entitled to future royalties on net sales outside the United States.

In a statement, Spark said the deal leverages Novartis’ “large, existing commercial and medical infrastructure in ophthalmology, as well as its commitment to commercializing genetic-based medicines.”

Novartis, which sells opthalmology drugs including macular degeneration treatment Lucentis, last year won U.S. approval for Kymriah, the first of a new type of potent gene-modifying immunotherapy for leukemia.

    “We think this deal is a positive for Spark, as the company will be able to focus on the U.S. launch of Luxturna, leaving the ex-U.S. launch and commercialization to Novartis, a leader in the ophthalmology space,” Jefferies analyst Michael Yee said in a note to investors.

    Luxturna treats inherited retinal disease caused by defects in a gene known as RPE65, which affects between 1,000 and 2,000 people in the United States. It works by delivering by an eye injection viral vector particles containing a correct copy of the gene to retinal cells, restoring their ability to make a needed enzyme.

    Shares of Spark, which fell 2 percent to close at $55.36 in regular trading on Wednesday, were up 3 percent at $57 after hours. The shares have lost nearly 25 percent of their value since mid-December when investors were disappointed by trial data for Spark’s experimental gene therapy for hemophilia A.

    Reporting y Deena Beasley; Editing by Susan Thomas and Tom Brown

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