Biogen and Sangamo Therapeutics announced a broad global licensing collaboration deal to develop and commercialize several compounds for a range of neurological and neuromuscular diseases.

Under the deal they will work to develop and commercialize ST-501 for tauopathies, diseases caused by abnormal tau proteins, such as Alzheimers disease, and ST-501 for synucleinopathies, neurodegenerative diseases marked by abnormal accumulation of alpha-synuclein proteins, such as Parkinsons disease.

They will also work on a third undisclosed target for neuromuscular disease and up to nine more undisclosed neurological disease targets. The agreement revolves around using Sangamos proprietary zinc finger protein (ZFP) technology that is delivered by way of adeno-associated virus (AAV). In other words, it is a type of gene therapy.

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, said Alfred Sandrock Jr., executive vice president, Research and Development, at Biogen. We aim to develop and advance these programs forward to investigational new drug applications.

Biogen is plunking down $350 million up front, with $125 million a license fee payment and $225 million in new Sangamo stock, coming to about 24 million shares at $9.21 per share. Sangamo will be eligible for up to $2.37 billion in various milestone payments, including up to $925 million in pre-approval milestone payments and up to $1.445 billion in first commercial sale and other sales-based milestone payments. Sangamo will also be eligible for tiered high single-digit to sub-teen double-digit royalties on any sales of products coming out of the partnership.

Zing finger transcription factors (ZF-TFs) modulate genes. By targeting a ZF-TF toward a specific DNA sequence, it is possible to up- or downregulate the expression of the genes. The ZF-TFs are delivered into the cell by a dead adeno-associated virus.

Biogen gains exclusive global rights to ST-501 for tauopathies and ST-502 for synucleinopathies, as well as the third undisclosed target, and up to nine more undisclosed targets for five years. Sangamo will handle early research activities and the expenses will be shared by the companies. Biogen will then take over responsibility and costs for investigational new drug-enabling research, clinical development, regulatory submissions and global commercialization.

Sangamo will handle GMP manufacturing operations for the initial clinical trials for the first three products, with expectations of using its in-house manufacturing capabilities. Then Biogen will take over GMP manufacturing activities beyond the first clinical trial.

In preclinical research, ST-501 and ST-502 have repressed both the proteins tau and alpha synuclein, respectively. Its a very long ways from preclinical work and effective medications for these types of neurodegenerative diseases, however. Still, investors seem pleased with it, with Sangamo shares climbing 39% at the news.

The combination of Sangamos proprietary zinc finger technology, Biogens unmatched neuroscience research, drug development, and commercialization experience and capabilities, and our shared commitment to bring innovative medicines to patients with neurological diseases establishes the foundation for a robust and compelling collaboration, said Stephane Boissel, head of Corporate Strategy at Sangamo. This collaboration exemplifies Sangamos commitment to our ongoing strategy to partner programs that address substantial and diverse patient populations in disease areas requiring complex clinical trial designs and commercial pathways, therefore bringing treatments to patients faster and more efficiently, while deriving maximum value from our platform.

Sangamo is a very busy company, with 17 separate preclinical or early-stage clinical projects ongoing. Five of the compounds are in Phase I/II clinical trials, with BIVV003 in sickle cell disease and ST-400 for transfusion-dependent beta-thalassemia. ST-920 is for Fabray disease and SB-525 is aimed at hemophilia A.

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Biogen and Sangamo Ink $2.7 Billion+ Neurodegeneration Deal - BioSpace