Less than 2 weeks after making headlines with its first ophthalmic company acquisition, Eli Lilly and Company has announced a second development: a partnership with MeiraGTx Holding plc.
Let’s begin with Lilly.
The global pharmaceutical company has long specialized in the biotech and genetic medicine therapeutic areas—primariily researching, developing, and manufacturing an extensive range of medicines.
- See here for its therapeutic areas of focus as of late (which, notably, has excluded ophthalmology).
And about that new ophthalmic acquisition …
Check out the details of its planned purchase of Adverum Technologies.
Next up: MeiraGTx.
This clinical-stage genetic medicines company has developed a pipeline of four late-stage clinical programs targeting:
- Leber congenital amaurosis (LCA4; AIPLI-associated congenital blindness)
- X-linked retinitis pigmentosa (XLRP)
- Parkinson’s disease
- Radiation-induced xerostomia (Rix)
And the basis for these programs?
MeiraGTx’s manufacturing capabilities have led to the development of a proprietary manufacturing platform based on 20 different potent viral vectors
- Specifically, the company’s novel technology provides in vivo delivery of any biologic therapeutics using oral small molecules.
Talk more about this technology.
The company’s riboswitch in-vivo delivery platform is designed for precise control over the in-vivo messenger ribonucleic acid (mRNA) production of therapeutic proteins from any DNA template.
- Click here to learn about the platform, including its mechanism of action.
And take note: The riboswitch technology enables the broad application and in-vivo delivery of any therapeutic protein.
And of those four clinical programs, which should we know about?
For the sake of this latest news: adeno-associated virus (AAV)-AIPL1.
What it is: An investigational genetic medicine for the treatment of LCA owing to a genetic deficiency of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1).
- Its purpose: To deliver the intact AIPL1 gene to cone and rod photoreceptors within the retina to restore vision and slow further degeneration in pediatric patients with LCA4 congenital blindness (see these reported clinical findings so far)
- Its delivery: a one-time subretinal injection.
In the United States: The therapeutic has received Rare Pediatric Disease and Orphan Drug designations from the FDA.
- And outside the U.S.: AAV-AIPL1 was manufactured and released under a Specials license within the United Kingdom (U.K.).
Any clinical data on it so far?
Yes—based on findings from a U.K.-based, non-randomized, single-arm, open-label, and first-in-human (FIH) study published earlier this year.
- More study details can be found here.
But the gist of the data: Early intervention of AIP1-related retinal dystrophy among young pediatric patients (ages 1-4) via a subretinally-administered dose of AAV-AIPL1 led to improved visual acuity and functional vision, some protection against progressive retinal degeneration, and no serious adverse effects.
Nice! Now to AAV-AIPL1 in the context of this collaboration.
Per the companies’ announcement, MeiraGTx will grant Lilly:
- The worldwide exclusive rights to its AAV-AIPL1 program for the treatment of LCA4-AIPL1
- The worldwide exclusive rights to its gene therapy technologies for use in ophthalmology— “with certain targets named by Lilly”
- These include novel intravitreal (IVT) capsids developed in-house and “bespoke promoters” such as artificial intelligence (AI)-generated promoters for specific cells in the retina
- Certain rights to its riboswitch technology for use in gene editing in the eye
Any mention of monetary value?
Yes, actually … MeiraGTx is expected to receive an upfront payment of $75 million as well as be eligible for:
- Over $400 million in total milestone payments
- Tiered royalties on licensed products
So! When will Lilly start making headway on its newfound exclusive rights?
No specific timeframe or plans have been shared by either company as of yet … so stay tuned.