Eli Lilly strikes another gene-drug deal, acquiring global rights to MeiraGTx retinal gene therapy
Eli Lilly and Company is developing an experimental MeiraGTx gene therapy that clinical data shows can reverse blindness in children with a rare genetic eye disease.
Under the terms of the agreement announced Monday, Eli Lilly will pay $75 million upfront for global rights to the gene therapy AAV-AIPL1. MeiraGTx may receive up to $135 million in additional short-term payments related to regulatory and approval milestones. The New York-based biotech company is currently preparing the gene therapy to seek regulatory approval in the United States and Europe.
AAV-AIPL1 was developed to treat Leber congenital amaurosis 4 (LCA4) caused by mutations in the AIPL1 gene. The AIPL1 protein is important for the normal function of the eye's rod and cone photoreceptors. Mutated versions of this protein cause retinal degeneration. People with LCA4 either have severe visual impairment at birth or develop visual impairment soon after birth. There are currently no treatments for LCA4.
MeiraGTx gene therapy uses an adeno-associated viral vector to deliver a functional copy of the AIPL1 gene to photoreceptors in the central retina. The one-time treatment is delivered via subretinal injection. MeiraGTx tested its LCA4 gene therapy in an open-label, single-arm study that enrolled 11 children aged 1 to 4 years. All participants were legally blind at birth; their only vision was the perception of light.
In February, MeiraGTx reported clinical trial results showing that all participants had improved vision in the treated eye four or more weeks after receiving the gene therapy. Additionally, patients experienced improved functional vision and preserved retinal structure compared with untreated eyes. No adverse reactions were reported. Detailed results were published in The Lancet.
Under the agreement, MeiraGTx can receive up to $400 million in milestone payments from Eli Lilly, including up to $135 million in near-term payments. If the gene therapy is approved, the biotech will also receive royalties from sales of Lilly's commercialized product. The AAV-AIPL1 deal kicks off a broader collaboration between the two companies. Eli Lilly also acquired rights to develop and commercialize preclinical treatment candidates MeiraGTx is developing for other inherited retinal dystrophies, according to MeiraGTx's regulatory filing. These illnesses are not disclosed.
Eli Lilly has an exclusive license to the proprietary MeiraGTx intravitreal shell, a protein shell that encapsulates a gene therapy's genetic cargo and delivers it to specific tissues in the body, for use with up to five ophthalmic targets of Lilly's choice, the filing said. The pharmaceutical company also received an exclusive license to a proprietary panretina- or rod-specific promoter for up to five targets selected by Eli Lilly. MeiraGTx has also granted Eli Lilly certain rights to use its proprietary riboswitch technology for ocular gene editing applications. Riboswitches enable the production of therapeutic proteins or gene-editing nucleases in the body. This production is controlled by oral administration of small molecules.
Gene therapies are a growing part of Eli Lilly's product pipeline. In 2021, the pharmaceutical giant acquired Prevail Therapeutics, a developer of gene therapies for neurodegenerative and rare diseases. Prevail has since established a multi-drug R&D alliance with startup Capsida Biotherapeutics, covering gene therapies for central nervous system (CNS) diseases. In 2022, Eli Lilly acquired Akouos, a developer of gene therapies for rare inherited hearing loss. Earlier this year, Eli Lilly entered into an agreement to secure the rights to Sangamo Therapeutics' capsids to deliver genomic medicines to treat central nervous system diseases. Last month, Eli Lilly acquired Adverum Biotechnologies, which leads a Phase 3 trial of gene therapies for wet age-related macular degeneration.
Andrew Adams, Eli Lilly's vice president of molecular discovery, said in a prepared statement that ophthalmology is an emerging area of interest for the company.
“We are excited to partner with MeiraGTx to bring revolutionary treatments to patients suffering from eye diseases around the world, starting with AAV-AIPL1, which has shown an unprecedented ability to help restore vision to children born blind,” he said.
Photo: Karen Bleier/AFP via Getty Images
