RARE Daily

Lilly to Acquire Gene Therapy Developer Prevail Therapeutics for $1 Billion

December 15, 2020

Rare Daily Staff

Big Pharma’s push into gene therapy and rare diseases is getting another push as Eli Lilly and Prevail Therapeutics announced a definitive agreement for Lilly to acquire Prevail in a $1 billion deal.

Prevail is developing potentially disease-modifying AAV9-based gene therapies for patients with neurodegenerative diseases. The acquisition will establish a gene therapy program at Lilly, anchored by Prevail’s portfolio of neuroscience assets, and will broaden Lilly’s commitment to use novel modalities to attempt to address otherwise fatal genetic forms of neurodegenerative disease.

Prevail’s lead gene therapies in clinical development are PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD), and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN). Prevail’s preclinical pipeline includes PR004 for patients with specific synucleinopathies, as well as potential gene therapies for Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and other neurodegenerative disorders.

“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,” said Mark Mintun, vice president of pain and neurodegeneration research at Lilly. “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.”

Under the terms of their deal, Eli Lilly will pay Prevail’s shareholders $22.50 per share in cash (or approximately $880 million and an 117% premium to Prevail’s 60 day average share price ahead of the announcement) plus one non-tradable contingent value right (CVR) worth up to $4.00 per share in cash (or approximately $160 million), payable upon first regulatory approval of a product from Prevail’s pipeline. The first deadline for realization of the full value of the CVR is the end of 2024, and it drops continuously to zero by the end of 2028. The deal is expected to close in the first quarter of 2021.

“Lilly is an established leader in neuroscience drug development and commercialization who shares our commitment to patients with neurodegenerative diseases,” said Asa Abeliovich, founder and chief executive officer of Prevail. “In just over three years, Prevail has advanced two first-in-class gene therapy programs into clinical development for PD-GBA, nGD, 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.  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.”

Lilly’s neuroscience portfolio includes an approved drug for migraines and a late-stage candidate for Alzheimer’s, an indication fraught with disappointment.  The Big Pharma highlighted two potentially disease-modifying clinical stage AAV9-based gene therapy candidates from Prevail’s pipeline, PR001 for patients with Parkinson’s disease with GBA1 mutations (PD-GBA) and neuronopathic Gaucher disease (nGD), and PR006 for patients with frontotemporal dementia with GRN mutations (FTD-GRN).

The U.S. Food and Drug Administration has granted Fast Track designation for PR001 for the treatment of PD-GBA and for the treatment of nGD. It has also granted Orphan Drug and Rare Pediatric Disease designations for the treatment of nGD.

The FDA and the European Commission have granted orphan designation for PR006 for the treatment of FTD, and the FDA has granted Fast Track Designation for PR006 for FTD-GRN.

Photo: Asa Abeliovich, founder and chief executive officer of Prevail

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