Less than six months after Al Sandrock took the helm of Voyager Therapeutics, the company is laying down maps toward the clinic, deciding its best route is through the central nervous system.
As it embarks on a new chapter, Voyager will prioritize its three assets indicated for Alzheimer’s, Parkinson’s and a type of amyotrophic lateral sclerosis (ALS), the company announced in its earnings report Thursday. They’re not exactly the easiest diseases to get back on track with, but Sandrock was bullish that the company “has a clear path” to proof-of-concept data and could “further progress these programs in an efficient manner.”
“We believe our highly differentiated approaches against well-validated CNS targets leveraging Voyager’s breakthrough TRACER capsids can lead to the development of new therapeutic options for these devastating diseases,” Sandrock said. “In addition, we believe each of these targets has a clear path to human proof of biology that should allow us to further progress these programs in an efficient manner.”
The TRACER platform that Sandrock touts is the key to any future success for these programs. Its objective is to better screen for genetic data that Voyager can then use to build adeno-associated virus capsids, creating gene therapies that have better specificity and less toxicity, all at lower doses than conventional alternatives. Capsids are the outer protein layer of the virus vectors where genetic material is held. Voyager touts the platform as having the ability to select capsids that have “robust penetration of the blood-brain barrier.”
The company’s penned-out plan provides clear guidance on what the aspirations are as the Voyager continues to rebuild more than a year after its CEO and R&D chief departed in May 2021. Those departures marked the end of a multi-year skid where the company experienced clinical holds, crumbling collaborations and falling value.
That’s where Sandrock enters the story. He had retired as Biogen’s R&D chief at the end of 2021, but it proved short-lived once he was given the opportunity to revive Voyager from the biopharma graveyard. Biogen, of course, has been pursuing its own CNS ambitions, including its controversially approved Alzheimer’s treatment Aduhelm.
But the reality for Voyager is that it’s still early days, irrespective of the bold new direction it’s selected. In outlining the three priority diseases and assets, the company indicated none of the programs are slated to enter the clinic earlier than 2024. In fact, the Parkinson’s program is not estimated to enter the clinic until 2025, although the company says that it’s “actively reviewing options to accelerate” work on the gene therapy.
The program currently leading the pack is focused on tau antibodies, for which the company recently presented preclinical data at the Alzheimer’s Association International Conference. Voyager’s data indicated that both its vectorized and passive antibody therapeutic approaches were promising in mice. The company says it intends to select a drug candidate in the first half of 2023, initiated toxicology studies later that year, and apply to enter the clinic in 2024.
There’s also the issue of money. Current cash reserves are enough to take the company into 2024 but, as noted, that’s currently before any of the top 3 assets would enter the clinic. In a statement, Sandrock referenced existing partnerships with Pfizer and Novartis around its TRACER platform and said the company was “optimistic about the potential for similar transactions in the future.”