The lead asset of Applied Genetic Technologies Corp. has been spun into Beacon Therapeutics Ltd., which launches with $120.9 million to run a phase II/III pivotal trial of AGTC-501 in X-linked retinitis pigmentosa, and to take forward two other in-licensed preclinical programs in age-related macular degeneration and cone rod dystrophy.
Little more than two years ago, Biogen Inc. plunked down about $800 million for gene therapy developer Nightstar Therapeutics plc, which had an X-linked retinitis pigmentosa drug in its pipeline. Top-line results from the phase II/III Xirius study of cotoretigene toliparvovec (BIIB-112), administered by subretinal injection, show the one-time therapy missed its primary endpoint of producing a statistically significant improvement in the proportion of treated study eyes.
DUBLIN – The contest to bring a safe and effective gene therapy for X-linked retinitis pigmentosa (XLRP) to market is intensifying. Two of the three contenders with clinical-stage programs reported initial 12-month data from phase I/II trials and are now looking ahead to pivotal trials and beyond.
Strongly favorable six-month data disclosed on Jan. 9 by Applied Genetic Technologies Corp. (AGTC) from its ongoing phase I/II program with an adeno-associated virus (AAV) gene therapy for X-linked retinitis pigmentosa (XLRP) did more than provide a whopping stock boost.