License agreement covers innovative gene therapy 4D-150 for neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME)
Strategic Partnership to Expand Access to Novel Gene Therapy
Otsuka Pharmaceutical Co., Ltd. has entered into a license agreement with U.S.-based 4D Molecular Therapeutics, Inc. (4DMT) to obtain exclusive development and commercialization rights for the investigational gene therapy 4D-150 in Asia and Oceania, including Japan.
The agreement marks a major step in Otsuka’s efforts to address neovascular age-related macular degeneration (nAMD)—also known as wet AMD—and diabetic macular edema (DME), two leading causes of vision impairment in the region.
Background: Addressing a Growing Vision Health Challenge
In Japan, approximately 1.3% of individuals aged 50 and above are affected by age-related macular degeneration, with nAMD accounting for 1.2%, representing an estimated 690,000 patients.
nAMD occurs when fragile new blood vessels form beneath the retina and leak fluid or blood, causing retinal damage and vision loss. Current treatment typically involves intravitreal injections of anti-VEGF agents every 4 to 16 weeks—an effective but demanding regimen that can be difficult for patients to maintain long term.
Similarly, DME—a complication of diabetic retinopathy—arises when damaged retinal blood vessels leak, causing swelling near the macula and potential vision loss. Both conditions represent significant unmet medical needs in ophthalmology.
4D-150: A Next-Generation Gene Therapy
Developed by 4D Molecular Therapeutics, 4D-150 is a gene therapy candidate designed to provide long-lasting suppression of abnormal blood vessel formation through a single intravitreal injection.
The therapy uses an engineered adeno-associated virus (AAV) vector to deliver multiple therapeutic genes directly into retinal cells. By inhibiting four members of the VEGF family, 4D-150 has demonstrated efficacy lasting up to 130 weeks in clinical studies—potentially reducing the need for frequent injections.
Details of the Licensing Agreement
Under the agreement:
- Otsuka will lead development, regulatory, and commercialization activities in its licensed territories.
- 4DMT will continue to oversee global Phase 3 clinical activities, including in the APAC region.
- Clinical sites for the 4FRONT-2 global Phase 3 nAMD study are expected to open in APAC by late 2025, with Japan sites expected in January 2026.
- Otsuka will make an upfront payment of USD 85 million, reimburse certain R&D expenses, and provide milestone payments tied to development and commercialization goals.
- Tiered royalties on net sales will also be paid to 4DMT.
Leadership Perspectives
“Otsuka has consistently created value through both in-house and collaborative research,” said Makoto Inoue, President and Representative Director of Otsuka Pharmaceutical Co., Ltd.
“With 4D-150, we aim to prevent vision loss in patients suffering from nAMD and DME through a single, potentially lifelong administration.”
Dr. David Kirn, Co-founder and CEO of 4DMT, commented:
“We are thrilled to partner with Otsuka, whose leadership in the Asia-Pacific region will help accelerate development and access to 4D-150. Together, we aim to reduce treatment burden and preserve vision for patients in high-prevalence regions.”
About Otsuka Pharmaceutical Co., Ltd.
Otsuka Pharmaceutical Co., Ltd. is a total healthcare company dedicated to enhancing well-being through innovative treatments and diagnostics that address both physical and mental health. The company continues to pursue partnerships that expand access to transformative therapies globally.
About 4D Molecular Therapeutics (4DMT)
4D Molecular Therapeutics, Inc., based in California, USA, is a late-stage biotechnology company pioneering disease-targeted gene therapies. Its proprietary Therapeutic Vector Evolution™ platform leverages a library of ~1 billion AAV capsid-derived sequences to develop customized, durable therapies for retinal and other serious diseases.
