4DMT Secures $420M Deal with Otsuka for Retinal Gene Therapy Rights in Asia-Pacific

4D Molecular Therapeutics (4DMT) has announced a significant partnership with Otsuka Pharmaceutical, selling the Asia-Pacific rights to its promising eye gene therapy, 4D-150. The deal, valued at up to $420 million, aims to accelerate the development and potential commercialization of the therapy for retinal vascular diseases in key markets across the region.

Deal Structure and Financial Terms

Under the terms of the agreement, 4DMT will receive $85 million in upfront cash from Otsuka. This initial payment is earmarked to fund the phase 3 development of 4D-150, which targets conditions such as wet age-related macular degeneration (AMD) and diabetic macular edema (DME). Additionally, Otsuka has committed to contributing at least $50 million over the next three years for global development activities.

The deal structure includes potential milestone payments of up to $336 million, contingent on meeting regulatory and commercial targets. Should 4D-150 reach the market, 4DMT stands to benefit from tiered double-digit royalties on sales within the Asia-Pacific region.

4D-150: A Novel Approach to Retinal Vascular Diseases

4D-150 represents an innovative approach to treating retinal vascular diseases. The gene therapy encodes aflibercept, an anti-VEGF drug currently marketed by Regeneron as Eylea, along with a sequence that inhibits VEGF-C expression. This dual-action mechanism is designed to provide sustained delivery of anti-VEGF agents, potentially improving patient outcomes while reducing the burden of frequent injections.

Recent phase 2 trial results have shown promising efficacy, with 60-week data demonstrating a 78% reduction in supplemental injections for patients receiving the phase 3 dose of 4D-150 compared to projected on-label use of Eylea 2 mg. These results have paved the way for the ongoing phase 3 trial in wet AMD, with plans for an additional late-stage study in DME.

Strategic Implications and Market Potential

The partnership with Otsuka is a key component of 4DMT's global strategy. While 4DMT will continue to lead phase 3 clinical trials and manufacturing activities globally, as well as pre-commercial and commercial activities outside the Asia-Pacific region, Otsuka's strong presence in the area is expected to navigate the complex regulatory and patient-access landscape effectively.

Chris Simms, 4DMT's Chief Commercial Officer, highlighted the significance of the Asia-Pacific market, noting its large and underserved retina patient population with a high prevalence of wet AMD and DME. The collaboration aims to accelerate both the development and potential patient access to 4D-150 in these critical markets.

This deal comes amidst a busy year for eye-related gene therapies, with several companies reporting clinical progress and attracting significant investment. The partnership between 4DMT and Otsuka underscores the continued interest and potential in gene therapies for ocular diseases, particularly those addressing large patient populations with chronic conditions requiring frequent interventions.