MeiraGTx has one gene therapy in a potentially pivotal clinical trial for cancer patients and another gene therapy ready to start Phase 3 in Parkinson’s disease. Completing the studies will be expensive and raising money is challenging with stock market volatility increasing by the day. MeiraGTx found another way to fund both studies. It’s teaming up with an artificial intelligence startup to form a joint venture responsible for the Parkinson’s program, supported by up to $230 million to finance its development through commercialization.
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The joint venture, Hologen Neuro AI, will combine technologies from both MeiraGTx and AI startup Hologen, the companies announced Thursday. The $230 million in committed cash will come from London-based Hologen. The startup also agreed to pay MeiraGTx $200 million upon the deal’s close, which the New York-based biotech said it will apply to its pipeline.
The Parkinson’s gene therapy, AAV-GAD, delivers the glutamic acid decarboxylase (GAD) gene into the subthalamic nucleus, a part of the brain key to motor control. It’s the same region targeted by deep brain stimulation to treat Parkinson’s. Delivery of the GAD gene is intended to increase local production of a neurotransmitter called GABA. This approach is intended to alleviate dysfunction of parts of the brain responsible for movement.
In blinded clinical tests to date, MeiraGTx reported its Parkinson’s gene therapy showed significantly better improvement compared to a group that received a sham procedure, measured according to scale used to assess symptoms of the neurological disorder. Furthermore, significant decline in the duration of disabling dyskinesia was observed only in patients who received the gene therapy. In an investor presentation, MeiraGTx said it is speaking to regulators with a goal of starting the Phase 3 test this year.
Hologen, a University College London and Kings College London spinout, says it uses multi-modal generative AI models to capture the complexity of biological systems, especially the brain. Based on clinical and investigational data, it creates what it calls large medicine models, or LMMs, that “learn the rich biological diversity of healthy and pathological variation in unprecedented breadth and detail.” The company adds that its technology is applied to the design of Phase 2 and 3 studies, increasing the probability of trial success.
MeiraGTx will lead clinical development of AAV-GAD. Through a subsidiary in England, it will produce the gene therapy for the pivotal test, entering into commercial manufacturing and supply agreements with the joint venture. Hologen will own a minority stake in the MeiraGTx subsidiary and contribute to its funding. The startup will also use its generative AI technology to optimize manufacturing of the gene therapy, leveraging data that MeiraGTx has amassed over the course of nearly a decade developing its manufacturing process. Hologen’s technology will continue to have a role in AAV-GAD’s Phase 3 test, MeiraGTx President and CEO Alexandria Forbes said in the company’s Thursday announcement of fourth quarter and full year 2024 financial results.
“The use of Hologen’s AI to elucidate brain circuitry in this complex heterogeneous disease has already significantly de-risked the AAV-GAD Phase 3 program when applied to MeiraGTx’s Phase 2 clinical data sets and has identified disease modifying changes in the physiology of the brain in response to AAV-GAD treatment,” Forbes said.
Besides AAV-GAD, the joint venture will develop earlier-stage MeiraGTx programs going after central nervous system targets. One of them, AAV-BDNF, is a preclinical treatment for a genetic form of obesity. Meanwhile, MeiraGTx can focus on other programs. Forbes said the $200 million from Hologen will enable her company to accelerate development of its riboswitch platform, technology that uses oral small molecules to regulate production of a protein or peptide in the body.
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The new cash will also expedite the potentially pivotal Phase 2 test of AAV-hAQP1, a gene therapy for radiation-induced xerostomia — dry mouth caused when radiation treatment for head and neck cancer damages the salivary glands. This side effect has no approved drug therapy, though frequent sips of water help. Damaged salivary glands contain ductal epithelial cells that are mostly water impermeable, MeiraGTx said in its annual report. AAV-hAQP1 is intended to introduce a water-conducting channel into the remaining epithelial cells to increase water flow into the mouth.
In the investor presentation, MeiraGTx described AAV-hAQP1 as a “pipeline in a product” opportunity. Other potential applications include prevention of radiation-induced xerostomia; Sjogren’s syndrome, which is an autoimmune disease affecting tear and salivary glands; and xerostomia associated with radioligand therapy. Sanofi has right of first negotiation for the Phase 2 xerostomia program under terms of a 2023 investment agreement.
With the Hologen payment, MeiraGTx projects its cash runway extends into 2027. That estimate excludes potential milestone payments from Johnson & Johnson, which has rights to a MeiraGTx gene therapy in development for a rare, inherited retinal disease. In addition to supporting the pipeline, the new capital will enable the biotech to repay debt that will come due in 2026, according to the annual report.
Image: Yuichiro ChinoImage, Getty Images
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