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Novartis AG (NYSE: NVS; SIX: NOVN) attains ex-US rights to develop and commercialize new gene therapy treatment for Leber’s congenital amaurosis (LCA).

Spark Therapeutics Inc. (NASDAQ:ONCE) have announced that the company has entered into a licensing and supply agreement for investigational voretigene neparvovec, branded in the US as LUXTURNA, indicated for the treatment of patients with confirmed bi-allelic RPE65 mutation-associated retinal dystrophy. The agreement is understood to include a $105 million upfront payment for Spark with an estimated $65 million in milestones and a royalty stream of around 25% of net sales. In addition, Spark will receive a milestone of $25 million upon drug approval by the EMA. According to the company’s press release, Spark Therapeutics will retain regulatory responsibility for securing EMA regulatory approval while a separate agreement has been completed with Novartis on the manufacture and supply of voretigene neparvovec.


According to Spark, the clinical evidence for the safety and efficacy of Luxturna derives from an open-label, dose-exploration Phase 1 study (n=12) and an open-label, randomized, controlled Phase 3 efficacy study (n=31) conducted in both pediatric and adult participants with biallelic RPE65 mutation-associated retinal dystrophy (age range 4 to 44 years). In the Phase 3 study, 21 participants were randomized to treatment while 10 were randomized to a non-intervention control arm. After a year, participants in the control arm crossed over to treatment. Efficacy of the treatment was based on a multi-luminance mobility test (MLMT) to measure changes in functional vision assessed by navigation of a course at varying luminance from 1 to 400 lux. Results showed a statistically significant difference between intervention (n=21) and control (n=10) at one year in the median bilateral MLMT outcome measure (difference of 1.6; 95% CI, 0.72, 2.41; p =0.001). According to Spark, the outcome measure has been sustained for at least three years in the original intervention arm and at least two years in the crossover arm. The company additionally claims that treated patients had a “marked difference compared to control participants across the first two secondary endpoints: full-field light sensitivity threshold (FST) testing averaged over both eyes (p =0.001) and the mobility test score change for the first injected eye (p =0.001).” The change in visual acuity from baseline to one year was not significantly different between the intervention and control participants.


Commenting on the deal with Novartis Pharmaceuticals, Dan Faga, chief business officer, Spark Therapeutics stated, “by leveraging Novartis’ large, existing commercial and medical infrastructure in ophthalmology, as well as its commitment to commercializing genetic-based medicines, we help ensure that more patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy who live outside the U.S., and importantly outside of Europe, have access to investigational voretigene neparvovec. We intend to use the proceeds from this transaction to continue to develop our robust pipeline of investigational gene therapies to create a path to a world where no life is limited by genetic disease.”