A year ago, Sangamo’s shares sank after it posted lackluster data for its genome-editing therapy; 12 months down the line, and it’s a complete reversal for the biotech as Biogen lays down a major upfront and biobucks pact for several of its central nervous system (CNS) meds.
While not the buyout many investors have been hoping for, Biogen is still the white knight for the biotech, which took a big knock in early 2019.
The Big Biotech, which is trying to make Alzheimer’s disease (AD) research great again, is days away (if you believe the analysts) from filing its failed AD drug aducanumab with the FDA. (After some clever data dredging, it thinks it may works in some patients after all.)
But it doesn’t want to rest on its laurels: Already this year it picked up Pfizer’s unwanted phase 1 CK1 inhibitor in AD in a small $75 million deal. Now, it’s boosting its AD and CNS pipeline further, paying a staggering $350 million upfront for a series of Sangamo’s preclinical assets with a $2.37 billion biobucks stream attached.
This broad collab is based on the biotech’s gene-regulation therapies and focuses on neurology. Initially, the deal falls on ST-501 for tauopathies including AD (a different MOA than its current attempt), ST-502 for synucleinopathies including Parkinson’s disease and a neuromuscular target, with exclusive rights for nine additional undisclosed neurological targets.
The nuts and bolts of the pact look like this: Sangamo will be responsible for GMP manufacturing activities for the initial test in the first three products, from which it plans to use its in-house manufacturing capacity.
For its part, Biogen will assume responsibility for GMP manufacturing activities beyond the first study for each of the first three products.
While the $2.37 billion would require everything going perfectly, that $350 million for drugs not even in the clinic is eye-watering and one of the, if not the biggest, preclinical licensing packages ever put together.
RELATED: Sangamo shares jump on Pfizer, Sanofi (and its own) gene, cell therapy updates
Sangamo has set itself up as rivaling CRISPR biotechs when it comes to gene regulation, using the so-called zinc finger platform that can repress or activate the expression of specific genes to achieve a desired therapeutic effect. It already has tie-ups with Gilead in oncology and Pfizer in hemophilia.
It’s had setbacks with the zinc finger approach, but both Sangamo and clearly Biogen believe a gene-regulation approach could help in neurology, a research area that is in desperate need of a research win. It certainly helped Sangamo’s investors: Shares shot up 40% after-hours on the news to $9.31 a share, although still far away from its highs in 2018 when it was in the $23-a-share territory.
Alfred Sandrock Jr., M.D., Ph.D., EVP of R&D at Biogen, said: “As a pioneer in neuroscience, Biogen will collaborate with Sangamo on a new gene regulation therapy approach, working at the DNA level, with the potential to treat challenging neurological diseases of global significance. We aim to develop and advance these programs forward to investigational new drug applications.”
“There are currently no approved disease modifying treatments for patients with many devastating neurodegenerative diseases such as Alzheimer’s and Parkinson’s, creating an urgency for the development of medicines that will not just address symptoms like the current standards of care, but slow or stop the progression of disease,” added Sandy Macrae, CEO of Sangamo.
“We believe that the promise of genomic medicine in neuroscience is to provide a one-time treatment for patients to alter their disease natural history by addressing the underlying cause at the genomic level.”