Astellas Pharma Inc. and 4D Molecular Therapeutics, Inc. (4DMT) have entered into a license agreement for the use of 4DMT’s proprietary, intravitreal R100 vector for rare ophthalmic disease targets.
Refresh me on these companies.
Formed in 2005, Astellas is the second-largest pharmaceutical company in Japan with research and development areas of focus that include mitochondria, genetic regulation, immuno-oncology, blindness and regeneration, and targeted protein degradation.
Also of interest: Astellas just purchased Iveric Bio.
Based in Emeryville, California, 4DMT is a clinical-stage biotherapeutics company focused on the development of genetic medicines for rare diseases in ophthalmology, pulmonology, and cardiology.
Let’s talk about this technology.
4DMT’s R100 is an adeno-associated virus (AAV) vector designed for intravitreal delivery and to penetrate the internal limiting membrane (ILM) barrier and transduce the entire retina—creating a robust transgene expression within retinal cells.
The vector can be used with each of the company’s four ophthalmic product candidates:
- 4D-150 (wet age-related macular degeneration [AMD]; diabetic macular edema [DME])
- 4D-125 (X-linked retinitis pigmentosa [XLRP])
- 4D-110 (choroideremia)
- 4D-175 (geographic atrophy [GA])
Now the agreement.
Under the terms, 4DMT will grant Astellas access to the R100 in order to deliver Astellas’s “unique genetic payloads for the treatment of rare monogenic diseases,” that will start with one genetic target and the potential for two additional targets.
Astellas will also conduct all related research, development, manfuracting, and commercialisation activities.
Give me some numbers.
4DMT is receiving $20 million upfront as well as potential future option fees and milestones of up to $942.5 million—of which includes $15 million of potential near-term development milestones for the initial target.
Is that it?
Nope, 4DMT will also reportedly be entitled to receive “mid-single digit to double-digit, sub-teen royalties on net sales of all licensed products.”
What are the companies saying?
According to 4DMT’s David Kirn, MD, co-founder and CEO, this collaboration “continues to validate R100 for routine intravitreal low-dose delivery of genetic payloads for the treatment of retinal diseases.”
Astellas’s Chief Strategy Officer (CStO) Adam Pearson stated that the agreement will “ultimately lead to the development of new therapeutics for patients with ophthalmic diseases at high risk of blindness.”