AGTC hopes to gain the first approval in X-linked retinitis pigmentosa for its gene therapy AGTC-501, now in a Phase II/III study. It also is advancing development in achromatopsia, another unmet medical need.

BIIB112, the gene therapy candidate Biogen picked up in $877m acquisition of Nightstar, missed its primary endpoint in X-linked retinitis pigmentosa. Phase III data for BIIB111, also from Nightstar, are expected soon.

Biogen will pay Denali $560m up front plus a $465m equity investment in exchange for co-development and co-commercialization rights to clinical-stage LRRK2 inhibitors for Parkinson's disease.

Second quarter performance was better than expected, but commercial weaknesses show the need for R&D wins and M&A deals as Tecfidera generics and aducanumab uncertainty weigh on the company.