NEW YORK – Nippon Shinyaku and Atsena Therapeutics on Wednesday said they inked an exclusive license agreement to commercialize ATSN-101, Atsena's investigational gene therapy for GUCY2D-mutated Leber congenital amaurosis (LCA), in the US and to develop and commercialize it in Japan.
In the deal, Kyoto, Japan-based Nippon Shinyaku will gain exclusive commercial rights to ATSN-101 in the US and its home country, and Atsena will maintain commercial rights in all other territories. The gene therapy will be marketed in the US by Nippon Shinyaku's US subsidiary, NS Pharma, located in New Jersey.
Nippon Shinyaku paid an undisclosed sum upfront for these rights to ATSN-101. Atsena is eligible to receive additional milestone payments, downstream royalties based on sales of ATSN-101, and reimbursement for clinical development work it does on the gene therapy, including conducting a pivotal trial. The companies didn't disclose further financial details of the deal.
LCA is an inherited disease of the retina that impairs eyesight, and the most common form of the disease, comprising 20 percent of cases, is caused by mutations in the GUCY2D gene (LCA1). This can be a particularly severe form of the disease and cause blindness in infants. Atsena's ATSN-101 is a subretinal adeno-associated virus 5-based gene therapy that delivers a functional GUCY2D gene to photoreceptors.
"ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist," Nippon Shinyaku President Toru Nakai said in a statement.
In September, Atsena published data from a Phase I/II trial of ATSN-101 and reported that those on the highest dose experienced a 100-fold improvement in vision in the treated eye compared to baseline as measured by the full-field stimulus test, and these improvements persisted for a year. Patients also tolerated the treatment well at the highest dose. Based on this data, Atsena said at the time that it was looking to advance the gene therapy into pivotal trials.
"This collaboration creates a path to accelerate the development of ATSN-101 and validates Atsena's pioneering technology and development capabilities," Atsena CEO Patrick Ritschel said in a statement. "We anticipate this will be the first of many ocular gene therapy treatments from our clinical portfolio to come."