Why is Eli Lilly Seeking Prevail’s Gene Therapy Pipeline?

In a move to expand its footprints in the lucrative field of gene therapy, Eli Lilly, a US drugmaker, is purchasing a New York-based gene therapy developer Prevail Therapeutics in a deal valued at $1.04 billion. The deal comes just weeks after Eli Lilly secured another deal with Precision BioSciences for developing in vivo gene therapies for genetic disorders.

Gene therapy has become a hot pursuit for many pharmaceutical companies. More than 200 companies are operating in North America to develop treatments for diseases difficult to treat. Novartis, Spark Therapeutics, Biogen, and Sarepta Therapeutics are some of the leaders in the field. With rising interest, the gene therapy market is expected to reach USD35.67 billion by 2027- ten-fold higher than last year’s market size. Now, Eli Lilly is set to become a member of this tribe.

Lilly’s commitment to the field of neuroscience dates long back. It started with a series of R&D programs in central nervous system disorders and churned drugs for migraine including Emgality and Reyvow. In 2012, the company established a facility at its UK base for studying conditions, including Alzheimer’s and schizophrenia. Although the company closed the center’s shutters early this year, it made sure that the talent was preserved and moved everything to Cambridge, Massachusetts, where it maintains a hub specializing in the application of siRNA and gene therapy. Eli Lilly also ran a clinical trial for their anti-amyloid beta antibody, solanezumab, which although failed in the phase 2 trial gave the company experience in the field.

Having failed with conventional therapies, Eli Lilly is now planning to use the gene therapy approach to treat patients with neurodegenerative diseases. However, since the company has limited experience in the field, it will need Prevail’s help to turn its dream into a reality.

What is Eli Lilly seeking from this transaction?

Eli Lilly is seeking Prevail’s clinical and preclinical pipeline, which includes gene therapies for Parkinson’s with GBA1 mutations (PD-GBA), neuronopathic Gaucher disease, and frontotemporal dementia with GRN mutations. These gene therapies deliver the correct form of the defective gene in the cells. The company’s lead clinical gene therapy is PR001 that is designed to treat patients with Gaucher disease. There are very limited treatment options for patients with Gaucher disease, which got PR001 Orphan Drug designation from the USFDA. Besides, it was also granted Rare Pediatric Disease Designation, and the USFDA expedited its review process to fill an unmet need in the field. PR001 is currently in Phase 1/ 2 to treat patients with PD-GBA and Type 2 Gaucher disease.

Prevail is also working on a treatment for frontotemporal dementia (FTD-GRN), and its drug PR006 is currently being tested in Phase 1/2 study for the treatment of FTD-GRN patients. Like PR001, PR006 has also been granted Orphan drug designation for the treatment of FTD, and it is currently being reviewed under fast track designation. Besides these treatments, Prevail is also working on a therapy 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.

In just over three, Prevail managed to do a lot of things. The company got two of its therapies in clinical development, established two manufacturing platforms, and developed a broad pipeline with great potential to impact patients in need of disease-modifying treatment options. The speed and expertise are what Eli Lilly needs at the moment if it wants to set a strong foothold in the gene therapy market where others have already made their territory.

What is in it for Prevail?

“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, M.D., Ph.D., founder, and chief executive officer of Prevail. Moreover, 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. We look forward to working together to advance our shared mission.”

For this deal, Prevail will receive an aggregate amount of approximately $880 million from Eli Lilly, who will buy shares for $22.50 apiece. Prevail is also eligible to receive a $4 contingent value right (CVR) payment if a product from the company’s pipeline gets regulatory approval-as long as it happens before the end of 2024. If the company fails to get any approval, the value of CVR will drop steadily until it expires at the end of 2028.

Who else is working in this field?

Eli Lilly will face strong competition from Voyager therapeutics in the future. Voyager is a clinical-stage gene therapy company that focuses on developing treatments for severe neurological diseases, including Parkinson’s and ALS. Its VY-AADC investigational gene therapy for Parkinson’s is currently in the Phase 2 trial, where its safety and efficacy are being evaluated. However, treating brain disorders with gene therapy is very challenging, and it will take a long time before any successful drug comes to the market.

By Ruchi Jhonsa, Ph.D.

Related Article: CRISPR Therapeutics Scores Prestigious Grant for Advancing Gene Therapies for HIV

References

1. https://investor.lilly.com/news-releases/news-release-details/lilly-announces-agreement-acquire-prevail-therapeutics

Author

Ruchi Jhonsa