With Prevail Acquisition, Lilly Plans New Gene Therapy Program

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by Diana Campelo Delgado |

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Eli Lilly has announced an agreement to acquire biotechnology company Prevail Therapeutics. Following the deal, Lilly plans to create a gene therapy program for people with neurodegenerative diseases, including Parkinson’s.

“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,” Mark Mintun, MD, vice president of pain and neurodegeneration research at Lilly, said in a press release.

Using a disease-modifying adeno-associated virus (AAV9), Prevail is working to create gene therapies that target the root genetic causes of several disorders characterized by neurodegeneration — the progressive atrophy and loss of function of nerve cells.

“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,” Mintun said.

Prevail is developing PR001 for Parkinson’s with GBA1 mutations and for neuronopathic Gaucher.

People with GBA1 mutations have up to a five times higher risk of developing Parkinson’s disease, with 7-10% of all Parkinson’s cases linked to these mutations. The GBA1 gene contains the information necessary to produce the enzyme beta-glucocerebrosidase (GCase) — an important component of cells’ recycling factories, called lysosomes. Lack of this enzyme, or its faulty activity, will result in the accumulation of toxic substances inside cells, which may contribute to the neurodegeneration seen in Parkinson’s.

PR001 uses a modified and harmless version of an AAV9 to deliver a working copy of the GBA1 gene to nerve cells. The therapy’s safety and efficacy is currently being tested in the Phase 1/2 PROPEL trial (NCT04127578), which is continuing to enroll participants at five U.S. locations. More information on these sites can be found here.

Prevail also is developing PR006 for patients with frontotemporal dementia with GRN mutations.

The company also has some therapies in preclinical development, including PR004 for specific synucleinopathies, and potential gene therapies for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s, and other neurodegenerative diseases.

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

“Lilly is an established leader in neuroscience drug development and commercialization who shares our commitment to patients with neurodegenerative diseases, and I’m excited for Prevail to join the Lilly family,” said Asa Abeliovich, MD, PhD, founder and CEO of Prevail.

“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 said.

Under this agreement, Lilly will acquire all outstanding shares of Prevail Therapeutics — up to $26.50 per share in cash or an aggregate of approximately $1.040 billion — if the first regulatory approval of a product from Prevail’s pipeline happens before December 31, 2024, in the U.S., Japan, the U.K., Germany, France, Italy or Spain.

The U.S. Food and Drug Administration (FDA) granted fast track designation to PR001 in July 2019 for the treatment of people with Parkinson’s disease associated with GBA1 gene mutations. This designation accelerates a therapy’s development and may help expedite its approval by providing more frequent meetings with the FDA and discussions about the therapy’s development plan.

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