Roche, like so many others in the field, has seen major setbacks in trying to hunt new drugs for Huntington’s disease, but it’s brushing off a recent flop and signing up with a new partner in the hopes of trying again.
Back in March, Roche had to stop a phase 3 Huntington’s disease test after an interim review of the data: While this review found no new safety signals associated with the Ionis Pharmaceuticals-partnered antisense drug tominersen, it was assumed that efficacy, or lack thereof, may have driven the decision to stop the trial.
It is technically Roche’s Spark unit, the gene therapy biotech it bought out a few years back, that’s penned this new deal, but it’s still the Swiss major’s cash, which is made up of an upfront and biobucks payment worth $190 million to the tiny new Pennsylvania-based startup NeuExcell.
For this, Roche/Spark can tap NeuExcell's “neuro-regenerative gene therapy platform and capabilities.” In a statement, the pair said NeuExcell's research team “will collaborate closely” with Spark to push the early-stage program on.
After the tominersen flop, Roche is looking at this Huntington’s pact a little differently. The current science says that mammalian adult neurons cannot be replaced, meaning that most R&D for brain diseases tend to focus on slowing disease progress. NeuExcell, however, “may have unlocked the method for regenerating neural tissue,” the biotech contends.
It sees its neuroregenerative gene therapy platform as the key to this, building around transcription-factor-based trans-differentiation tech. The platform seeks to reprogram endogenous glial cells like astrocytes, which surround neurons and are often reactive after neurons are injured or die, into functional new neurons.
The biotech, which only came into public view with a quiet $10 million series A last week, is developing adeno-associated virus (AAV)-based neuroregenerative gene therapy to regenerate functional new neurons at the site of the neurodegeneration.
The plan is to complement this platform with Spark’s own AAV vector approach, which is targeted to the central nervous system.
“Using our existing expertise in gene therapy development and NeuExcell's neuro-regenerative gene therapy research and capabilities, together we can progress the potential of gene therapy for patients living with Huntington's disease,” said Joseph La Barge, chief business officer of Spark Therapeutics.