Rare Daily Staff

Spark Therapeutics said it has entered into a licensing agreement with Novartis Pharmaceuticals to develop and commercialize its gene therapy Luxturna to treat an ultra-rare genetic eye disease outside the United States for $105 million upfront and up to an additional $65 million in milestones.

Luxturna is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients. The condition affects about 1,000 to 2,000 patients in the United States. The RPE65 gene provides instructions for making an enzyme that is essential for normal vision. There are currently no approved pharmacologic treatment options for IRD due to biallelic RPE65 gene mutations.

Under the agreement, Spark will retain regulatory responsibility for obtaining European Medicines Agency approval for the gene therapy. Spark also entered into a separate agreement to manufacture and supply investigational Luxturna to Novartis. No other programs in Spark Therapeutics’ pipeline are part of this agreement.

In addition to the milestone payments, which are tied to near-term reguatlroy approval in Europe and initial sales in certain markets outside of the United States, Spark will also receive royalty payments on net sales of the gene therapy outside the U.S.

Dan Faga, chief business officer for Spark, said “by leveraging Novartis’ large, existing commercial and medical infrastructure in ophthalmology” and its commitment to commercializing genetic based medicines, will help ensure more patients with the condition outside of the United States will have access to Luxturna.

He said that Spark will use the proceeds from the transaction to continue to develop its pipeline of gene therapies.

January 25, 2018

Photo: Dan Faga, Chief Business Officer for Spark Therapeutics