Vertex Pharmaceuticals is getting deeper into in vivo gene editing. Months after partnering with Mammoth Biosciences, the storied biotech is back with another deal, ponying up $60 million to work with Verve Therapeutics on a single undisclosed liver disease.
Interest in in vivo gene editing has ratcheted up since Intellia and Regeneron first presented phase 1 data on their treatment for the rare disease transthyretin amyloidosis a little more than a year ago. Since then, Novartis, Pfizer and Vertex have struck deals to apply the approach to sickle cell and multiple other rare diseases.
Vertex’s latest foray into the space has resulted in a deal with Verve, a biotech that is using gene editing technologies to try to potently and durably control cumulative exposure to LDL cholesterol. Verve’s goal is to shift treatment of cardiovascular disease from chronic management to single-course therapies. With Verve’s lead program, which uses lipid nanoparticle delivery to turn off the PCSK9 gene in the liver, now in the clinic, Vertex has seen enough promise to come to the deal table.
The agreement tasks Verve with taking in vivo gene editing candidates against an undisclosed liver target of interest into preclinical development. Verve’s to-do list includes identifying and engineering specific gene editing systems and in vivo delivery systems directed to the target and evaluating and optimizing candidates to achieve specified criteria. Vertex will take over partway through preclinical development.
Here’s how the figures break down: Vertex made a $25 million upfront payment and bought $35 million of Verve stock on Wednesday. Verve is eligible to receive up to $66 million, split evenly across the three candidates, in success payments upon the achievement of development criteria. The bigger paydays will come later, with Vertex on the hook for up to $340 million in development and commercial milestones.
Verve is eligible to receive tiered single-digit royalties but can forego those payments in favor of a split of the profits. The deal requires Verve to opt in to the profit share agreement before a patient is dosed with the first candidate. Upon opting in, Verve will decide on the size of its share of the costs and profits, up to a ceiling of 40%, and pay a fee of $25 million to $70 million. The size of the fee depends on the profit split and the licensed Verve technology included in the most advanced product candidate.
Shortly after disclosing the deal, Verve put out news of a stock offering. The offering, which was upsized to $225 million, will support the ongoing phase 1 clinical trial and the progression of another candidate toward the clinic.