Lilly Acquires Prevail and Gene Therapy for Neuronopathic Gaucher

Lilly Acquires Prevail and Gene Therapy for Neuronopathic Gaucher
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Eli Lilly reached an agreement to acquire Prevail Therapeutics, including its clinical program for PR001, an experimental one-time gene therapy for neuronopathic Gaucher disease.

Prevail has a portfolio that includes early and clinical stage gene therapies targeting the genetic causes of several neurodegenerative diseases. These use a modified and harmless adeno-associated virus type 9 to deliver a functional copy of the disease-causing mutated gene to cells, including those in the central nervous system (CNS; the brain and spinal cord).

“Gene therapy is a promising approach with the potential to deliver transformative treatments for patients with neurodegenerative diseases such as Parkinson’s, Gaucher and dementia,” Mark Mintun, MD, vice president of Lilly’s pain and neurodegeneration research, said in a press release.

“The acquisition of Prevail will bring critical technology and highly skilled teams to complement our existing expertise at Lilly, as we build a new gene therapy program anchored by well-researched assets,” Mintun added.

Asa Abeliovich, MD, PhD, Prevail’s founder and CEO, said that Lilly “is an established leader in neuroscience drug development and commercialization who shares our commitment to patients with neurodegenerative diseases.

“With its global scale and resources, Lilly will be the ideal organization to maximize the potential of our pipeline and accelerate our ability to bring these therapies to as many patients as possible,” Abeliovich added.

Under the terms of the agreement, Lilly will acquire all outstanding shares of Prevail, with payments of up to $26.50 per share, or a total of about $1.04 billion, if the first regulatory approval of a pipeline product in the U.S., Japan, U.K., Germany, France, Italy, or Spain happens by Dec. 31, 2024. The minimum agreed payment at closing is $22.50 per share plus another contingent value right.

PR001 is being developed for neuronopathic Gaucher, which affects the CNS and includes types 2 and 3 disease. It works by delivering a fully working version of GBA, the gene that is deficient in Gaucher patients, to nerve cells. 

GBA provides the instructions to produce beta-glucocerebrosidase, an enzyme involved in the disposal and recycling of large fatty molecules inside cells. Beta-glucocerebrosidase deficiency leads to the toxic buildup of these molecules, ultimately impairing the function of several organs.

As such, PR001 is expected to restore beta-glucocerebrosidase activity in the CNS and potentially prevent further neurologic damage. 

PR001’s clinical program includes the Phase 1/2 PROVIDE trial (NCT04411654), which will evaluate the safety and effectiveness of the gene therapy in 15 infants (newborns to age 2) with type 2 Gaucher — the most severe form of neuronopathic Gaucher. Enrollment is ongoing at NYU’s Medical Center, and three other U.S. sites are expected to soon open. More information can be found here.

Participants will receive a single dose of PR001 directly into the cisterna magna, an area located above the spinal canal, together with an immunosuppressive treatment regimen.

Updates on PROVIDE findings are expected by next year.

The therapy received fast track, orphan drug, and rare pediatric disease designations from the U.S. Food and Drug Administration for the treatment of Gaucher disease; all are meant to expedite its clinical development and review. 

Since GBA mutations also increase the risk of Parkinson’s disease, PR001 is also being tested as a potential therapy for Parkinson’s patients with such mutations (PD-GBA). Prevail’s gene therapy portfolio also includes PR006 for frontotemporal dementia caused by GRN mutations (FTD-GRN), and PR004 for certain synucleinopathies — neurodegenerative diseases associated with the toxic accumulation of clumps of a protein called alpha-synuclein.

In addition, the company is working on other potential gene therapies for Alzheimer’s disease and amyotrophic lateral sclerosis.

“I’m incredibly proud of the Prevail team, who have made great progress advancing our pipeline of gene therapy programs for patients with these devastating disorders,” Abeliovich said.

“In just over three years, Prevail has advanced two first-in-class gene therapy programs into clinical development for PD-GBA, [neuronopathic Gaucher], and FTD-GRN, established two manufacturing platforms, and developed a broad pipeline with great potential to impact patients in need of disease-modifying treatment options,” he added.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.
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José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.
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