Following its $5.9 billion Iveric Bio buy, Astellas is leaning into the eye disease space with a licensing agreement for 4D Molecular Therapeutics’ proprietary intravitreal AAV delivery system.
Pictured: Astellas’ Americas headquarters in Illinois/iStock, JHVEPhoto
Astellas Pharma has signed a license agreement with 4D Molecular Therapeutics, gaining access to the California biotech’s proprietary viral vector for a rare genetic eye disease, the companies announced late Monday.
Under the agreement, Astellas will pay $20 million in upfront fees but 4DMT will be eligible for up to $942.5 million in future option fees and milestones. This sum includes a $15 million payout in potential near-term development milestones for an initial target.
4DMT will also be entitled to mid-single digit to double-digit, sub-teen royalties from net sales of licensed products that may come out of the partnership.
In return, Astellas will be able to use 4DMT’s proprietary R100 delivery technology, an adeno-associated virus vector designed for intravitreal administration. R100 can penetrate the internal limiting membrane, a common stumbling block for eye-directed gene therapies, as well as efficiently transduce the entire retina. As a result, R100 promotes the robust expression of transgene constructs in retinal cells.
4DMT uses R100 for all three of its clinical-stage candidates for eye diseases. In April 2023, the company posted positive interim results from its Phase I/II PRISM study in wet age-related macular degeneration (AMD). The candidate, dubbed 4D-150, was well-tolerated and triggered no signs of inflammation in most of the study participants.
There were also no dose-limiting toxicities and serious adverse events linked with 4D-150, which further validates the “potential of our intravitreal R100 vector, for the safe delivery of effective transgene payloads,” 4DMT CEO David Kirn said in a statement at the time.
The company is also trialing 4D-150 in diabetic macular edema. R100 is being utilized in several other of 4DMT’s candidates, including 4D-110 in choroideremia, 4D-175 in geographic atrophy and 4D-125 in X-linked retinitis pigmentosa.
Under Monday’s license agreement, Astellas will use R100 to deliver a unique genetic payload to treat a rare monogenic eye disease. The Japanese multinational will be responsible for all research, development, manufacturing and commercialization activities for this therapy. Astellas can also add two more targets for an additional option exercise fee.
Meanwhile, 4DMT will retain rights to R100 for large-market, non-hereditary ophthalmic conditions, Kirn said in Monday’s announcement.
Astellas has recently set its sights on the eye disease market. In May 2023, the company acquired Iveric Bio for $5.9 billion, gaining access to the New Jersey biotech’s portfolio of eye treatments. Iveric is awaiting the FDA’s decision for its investigational C5 inhibitor avacincaptad pegol for the treatment of geographic atrophy secondary to AMD. The regulatory decision is due August 19.
Tristan Manalac is an independent science writer based in metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.
