Astellas paves backloaded biobucks deal to test 4DMT's eye disease gene therapy vector

Astellas Pharma is digging up some cash to use one of 4D Molecular Therapeutics’ gene therapy vectors for at least one rare eye disease—but the genetic medicines company is going to have to wait a while to see the true bounty.

The Japanese pharma has signed a deal to use 4DMT’s intravitreal retinotropic R100 vector for one genetic target in rare monogenic ophthalmic diseases, the companies said Monday. Astellas could later add up to two additional targets. 4DMT will receive $20 million upfront, with milestones that could total $942.5 million. This includes a near-term milestone of $15 million for the initial target. The company could also receive royalties on sales of any licensed products that result.

Astellas will use the vector to deliver its own genetic payloads and is responsible for R&D, manufacturing and commercialization activities for the resulting programs.

The pharma is dipping its toe in the water through the deal to access a vector that 4DMT has used for four different product candidates. The furthest along is 4D-150, which is in phase 1/2 trials for wet age-related macular degeneration. So far, 70 patients have been dosed with therapies that use the vector, according to 4DMT CEO David Kirn, M.D.

R100 is an adeno-associated virus vector designed for intravitreal delivery that has the ability to penetrate the internal limiting membrane barrier and to efficiently transduce the entire retina, according to the release. This results in robust transgene expression in the retinal cells.

4DMT will maintain rights to the use of R100 for large-market non-hereditary ophthalmic diseases, Kirn noted. That includes the pipeline assets 4D-150, 4D-125, 4D-110 and 4D-175.

The deal could help turn around 4DMT’s fortunes after the FDA placed a clinical hold on the biotech’s Fabry disease gene therapy program in February. Enrollment in the program was voluntarily halted in January after three cases of a kidney condition were reported among the first six patients across two trials. An update on that program is expected in the second half, according to 4DMT’s May earnings release.

Astellas, meanwhile, has been busy, with three oncology approvals expected in the next year and a recent deal with Kate Therapeutics that will see the pharma once again enter a fatal neuromuscular disease called X-linked myotubular myopathy. The 4DMT deal fits into Astellas' focus on blindness and regeneration, which is a primary goal of its R&D strategy, the pharma's Chief Strategy Officer Adam Pearson said in the release.