Pfizer Collaborates with Vivet to Develop Gene Therapy for Wilson Disease

By Rajaneesh K Gopinath, Ph.D.

The US pharma giant struck a deal by buying 15% equity interest in the French biotech company, headquartered in Paris for US$51 million, and also obtained the exclusive option to acquire all of its outstanding shares for US$635.8 million.

Last week, Pfizer released a joint statement with Vivet Therapeutics expressing their decision to develop VTX-801 which is the latter’s proprietary treatment for Wilson disease, an inherited liver disorder characterized by copper poisoning. The disease is a result of a monogenic mutation in the ATP7B gene that causes impaired copper transport leading to its accumulation in the liver and other parts of the body. This results in serious liver damage and could prove fatal if left untreated. The present cure for this condition includes liver transplants or treatments with critical adverse effects.

VTX-801 – Background, and history

VTX-801 is a novel investigational treatment developed by Vivet for this rare disease. It codes for the functional mini-ATP7B copper transporter that restores the copper metabolism thereby maintaining homeostasis. This adeno-associated virus (AAV)-directed gene therapy received Orphan Drug Designation (ODD) both from the US Food and Drug Administration (USFDA) and the European Commission (EC) back in 2017. It now enters clinical trials and could possibly compete with Alexion’s WTX101 which is already in phase III development. VTX-801’s performance in phase I/II trials would determine if Pfizer would exercise its exclusive right to acquire all of Vivet’s shares in the future.

Both parties shared their excitement over the news. Jean-Phillippe Combal, Co-Founder & CEO of Vivet, said, “We welcome Pfizer as a shareholder and partner that can help us advance our efforts to develop therapies for patients burdened with inherited liver disorders. This investment demonstrates the clear value of Vivet’s innovative approaches to gene therapy.”

Mikael Dolsten, CSO of Pfizer said, “Pfizer strives to provide meaningful enhancements to the lives of patients with rare diseases. Our partnership with Vivet offers an important expansion of Pfizer’s commitment to collaborate with the scientific community and to accelerate our leading AAV-directed gene therapy portfolio.”

References
1.https://www.pfizer.com/news/press-release/press-release-detail/pfizer_secures_exclusive_option_to_acquire_gene_therapy_company_vivet_therapeutics
2.https://www.businesswire.com/news/home/20170925006165/en/Vivet%E2%80%99s-Gene-Therapy-Product-VTX-801-Wilson%E2%80%99s
3.http://www.pmlive.com/pharma_news/alexion_adds_to_pipeline_rebuild_with_$855m_wilson_buy_1231516

Author

Rajaneesh K. Gopinath