The first company to win regulatory approval for a gene therapy in the US has become the latest acquisition by a large pharmaceutical firm.
On Monday, Spark Therapeutics said it would be bought by Swiss drugmaker Roche for $4.8 billion. The Wall Street Journal reported the deal Saturday. Shares of the Philadelphia-based gene therapy maker were up more than 120 percent in premarket trading on the Nasdaq Monday.
In December 2017, Spark won the first ever Food and Drug Administration approval for a gene therapy, Luxturna (voretigene neparvovec-rzyl), for biallelic RPE65 mutation-associated retinal dystrophy, a rare, genetic form of blindness. Luxturna works by using an adeno-associated viral vector to insert a corrected gene into cells that carry the mutation.
In addition to the approved product, the company also has several other gene therapies in clinical development. The most advanced are SPK-9001 (fidanacogene elaparvovec), for hemophilia B, and SPK-8011, for hemophilia A, both of which are in Phase III clinical trials. In Phase I/II development, the company has SPK-7001, for choroideremia, and SPK-8016, for hemophilia A in the 30 percent of patients whose disease has inhibitors. Therapies in preclinical development include SPK-3006, for the lysosomal storage disorder Pompe disease, and others for retinal and neurodegenerative diseases.
Roche’s purchase of Spark represents one of the biggest acquisitions of the year so far, closely following last month’s $8 billion acquisition of Loxo Oncology by Indianapolis-based Eli Lilly & Co. However, SVB Leerink analyst Joseph Schwartz pointed out that it also indicated a continued interest by big pharma companies in acquiring gene therapy firms. Other, similar deals in recent memory that he noted include Swiss drugmaker Novartis’s acquisition last year of AveXis for $8.7 billion and the 2016 acquisition of Bamboo Therapeutics by Pfizer for about $700 million. Luxturna is partnered with Novartis, while SPK-9001 is partnered with Pfizer.
Schwartz wrote that the amount of money Roche is paying for Spark “seems steep,” but is potentially rationalized by an attraction to genetic retinal diseases, fortification of the hemophilia franchise and an appreciation for having in-house gene therapy manufacturing capabilities. On those bases, the deal is a positive sign, for other gene therapy companies like BioMarin, Audentes Therapeutics, Nightstar Therapeutics, Uniqure and Solid Biosciences, he wrote.
Photo: AndreyPopov, Getty Images
