Pfizer eyes Fierce 15 winner Vivet buy as gene therapy fever heats up

Vivet is in the crosshairs of Big Pharma Pfizer as the U.S. giant looks to get in on the new industry craze of buying up small gene therapy biotechs.

Speaking to me back in 2017 when it won the Fierce 15 award, the small, France-based biotech believed it could use the potential of gene therapy as a curative approach for Wilson disease, as it also looked across the Atlantic for its future, which turned out to be pretty prescient.

The company got off a €37.5 million ($41 million) series A raise in 2016, no small feat for a European biotech, given that the biopharma industry across the continent typically fails to enjoy the same rich funding streams those in the U.S. do.

The VC arms of Roche and Novartis were early backers for a team of executives from Novartis itself, as well as Sanofi and Gensight.

This includes CEO Jean Philippe Combal, Pharm.D., Ph.D., who worked a stint at Fovea, which in 2009 was bought by Sanofi, and in 2014 moved over to GenSight to work on its gene replacement therapy for Leber’s hereditary optic neuropathy, a rare mitochondrial disease that leads to irreversible and often brutal sight loss in teens and young adults.

Its leading drug, VTX801, initially targets Wilson disease, a rare disorder that can lead to organ damage, neurologic symptoms and potentially death. Around 10,000 patients in the U.S. and 15,000 patients in the EU are estimated to have the disease.

The therapy uses a modified AAV vector to move a truncated functional version of the ATP7B gene into liver cells carrying the defective gene, which should treat the underlying cause of the disease.

Vivet’s pipeline comes out of tech developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada, a not-for-profit foundation at the Centro de Investigación Medica Aplicada; the University of Navarra based in Pamplona, Spain; and the Massachusetts Eye and Ear Infirmary in Boston.

Today, it grabbed the attention of one of the world’s biggest pharmas: Pfizer, which is paying for a 15% stake in the company with an option attached to buy out the biotech. The pair will also now work together on TX801.

It is not an expensive bet for Pfizer, which paid just €45 million ($51 million) upon signing its new stake, which could go up to €560 million ($635.8 million), while holding onto the right to buy up the biotech in full, should “certain data” from the ongoing early to midstage trial of VTX-801 go to plan.

Other terms of the deal were not disclosed.

Vivet is also working on liver-directed gene therapy programs for progressive familial intrahepatic cholestasis for bile excretion defects and citrullinemia for defects in the urea cycle, which leads to the build-up of ammonia and other toxic substances in the blood.

RELATED: Biogen-Nightstar deal sheds light on gene therapy feeding frenzy

Despite being just three months in, it’s shaping up to be a big year for small gene therapy biotechs, as biopharma has been snapping them up like flies: We’ve seen Biogen and its $877 million buyout of Nightstar Therapeutics (with other suitors also in hot pursuit); Neurocrine Biosciences paying Voyager Therapeutics $165 million upfront for the rights to a clutch of gene therapies, with $1.7 billion in biobucks also on the table; the $4.3 billion buyout of Spark by Roche; and last year the resetting of Axovant, the once CNS-focused biotech, into gene therapy work.

Pfizer already has an ongoing collaboration with Spark on hemophilia B gene therapy program SPK-9001. After the New York pharma formed that deal, in 2016 it bought up Bamboo Therapeutics, along with the latter’s recombinant adeno-associated virus-based gene therapy platform.