Voyager Therapeutics looks to shake off a tough year as it pens Pfizer gene therapy pact worth $630M

Hoping to make gene therapies safer and looking to move on from a troubled 18 months, Pfizer is putting down as much as $630 million in an upfront/milestone-based payment for Voyager’s next-gen gene therapies.

Pfizer, which recently had to change the protocol for its ongoing Duchenne muscular dystrophy gene therapy trial after a series of safety concerns emerged, will pay $30 million upfront and $20 million in exercise fees for two options, with $580 million backloaded to use Voyager’s so-called TRACER AAV capsids specifically for neurologic and cardiovascular gene therapy programs.

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These capsids have been sought out to target desired cells and tissues with greater specificity, at lower doses and with fewer off-target risks than conventional AAV, with enhanced blood-brain barrier penetration.

The hope is that these could prove safer in the long run and follows on from a similar $1.1 billion deal Takeda made this week with Selecta Biosciences, a pact that focuses on the biotech’s tolerogenic therapies that selectively lessen unwanted immune responses and can therefore also potentially make these therapies safer.

This also comes after Astellas has seen four deaths in young children from its now FDA halted gene therapy trial for AT132 in a rare neuromuscular disease, with three of the deaths appearing to be linked to the therapy and its affect on the liver. Gene therapies hold much promise but also come with risk; the future of their use will surely be focused heavily on making them safe for patients, so don’t be surprised to see more deals like this in the future.

“This transaction highlights the potential of our TRACER platform to identify novel AAV capsids that target desired cells and tissues with greater specificity at lower doses and with fewer off-target risks than conventional AAV serotypes,” said Michael Higgins, interim CEO of Voyager.

“We believe that our TRACER platform has the ability to produce not only enhanced blood-brain-barrier penetrant capsids, but also novel capsids with enhanced tropisms across a diversity of tissues and cell types, offering promise to unlock the fullest potential of gene therapies for a wide array of diseases with unmet medical need.”

This comes as Voyager has been dealing with its own issues: Last August, AbbVie axed its Alzheimer's and Parkinson’s disease pacts with Voyager after the Big Pharma paid $134 million to enter into the two partnerships but then dropped the projects after Voyager completed research activities.

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That same year the biotech was slapped with a clinical hold on an IND for its gene therapy program, VY-HTT01, because of issues with chemistry, manufacturing and controls. This marked a miserable time for the program, coming one year after Sanofi’s Genzyme unit pulled out of a collab for it.

But in April, the biotech got a reprieve after the FDA lifted the hold, allowing the trial to continue after the issues were cleared up.

Just one month later, the biotech’s CEO Andre Turenne (replaced by Higgins for the time being) and its R&D chief Omar Khwaja, M.D., Ph.D., both left unexpectedly. This deal with Pfizer is some much needed good news.

“Our collaboration with Voyager will provide Pfizer with access to additional AAV capsids that may help further advance our industry-leading gene therapy portfolio,” added Seng Cheng, Ph.D., senior vice president and chief scientific officer of Pfizer's rare disease research unit.

“We are impressed with Voyager’s results to date and are enthusiastic about the potential to utilize these novel capsids to help accelerate the development of new therapeutic options for patients living with certain neurologic and cardiovascular diseases.”