4D Molecular Therapeutics has sold the Asia-Pacific rights to its eye gene therapy to Otsuka Pharmaceutical, securing $85 million in upfront cash to fund the asset’s phase 3 development.
In return for the rights to develop and commercialize the gene therapy, dubbed 4D-150, for retinal vascular diseases like wet age-related macular degeneration (AMD) and diabetic macular edema (DME) in Japan, China, Australia and other Asia-Pacific markets, Otuska is also expected to assist with cost-sharing at least $50 million over the next three years for global development activities.
Further down the line, Otuska could also end up paying out up to $336 million in potential regulatory and commercial milestone payments, as well as tiered double-digit royalties should 4D-150 make it to market.
4D-150 encodes aflibercept—an anti-VEGF drug Regeneron markets as Eylea—and a sequence that inhibits expression of VEGF-C. 4DMT designed 4D-150 to provide sustained delivery of anti-VEGF agents, freeing patients from burdensome injections and potentially improving outcomes.
The deal comes three months after 4DMT posted 60-week data from a phase 2 trial of 4D-150 that demonstrated a 78% reduction in supplemental injections in people who received the phase 3 dose compared to projected on-label use of Eylea 2 mg.
The gene therapy is currently in a phase 3 trial for wet AMD—its lead indication—with a further later-stage study for DME being lined up. The biotech said the upfront cash from the Otsuka deal would help fund this DME study as well as “pre-commercial activities.”
“We are thrilled to announce this strategic partnership with Otsuka, a leading global pharmaceutical company with a strong presence in the APAC region, reflecting our shared long-term commitment to improving outcomes for patients with retinal vascular diseases,” 4DMT CEO David Kirn, M.D., said in the Oct. 30 release.
“This partnership is a key pillar of our global strategy, with 4DMT continuing to lead phase 3 clinical trial and manufacturing activities globally, in addition to pre-commercial and commercial activities outside the APAC region,” Kirn added.
4DMT Chief Commercial Officer Chris Simms said the Asia-Pacific region “represents a large and underserved retina market, with a high prevalence of wet AMD and DME.”
“Navigating the region’s regulatory and patient-access complexities requires a strong local partner, and we expect Otsuka’s deep presence and expertise to accelerate both development of and access to 4D-150,” Simms added.
It’s been a busy year for eye-related gene therapies. While Johnson & Johnson’s $130 million bet on an X-linked retinitis pigmentosa candidate suffered a phase 3 setback in May, the likes of Opus Genetics, MeiraGTx, Beacon Therapeutics and 4D Molecular Therapeutics have seen better news in the clinic.
Meanwhile, the $135 million series B and Big Pharma backing secured by Stargardt disease-focused SpliceBio in June suggests investors can still be enthused, and Eli Lilly swooped in to buy Adverum last week.
Otsuka has been on the lookout for opportunities to pad out its pipeline this year. The summer saw the Japanese company pay $33 million for a Swedish biotech’s IL1RAP antibodies designed to treat autoimmune and inflammatory diseases, as well as hand over $47 million for the ex-China rights to Harbour BioMed’s BCMAxCD3 bispecific candidate.