Biogen and Sangamo Therapeutics, a genomic medicine company, have formed a global licensing collaboration agreement for gene-regulation therapies in neurology in a deal worth up to $2.72 billion ($350 million upfront and $2.37 billion in potential milestones).

Under the agreement, the companies have agreed to develop and commercialize two investigational drug candidates (ST-501 and ST-502) of Sangamo’s for treating neurodegenerative disorders, including Alzheimer’s disease and Parkinson’s disease, a third undisclosed neuromuscular disease target, and up to nine additional undisclosed neurological disease targets. ST-501 is in preclinical development for treating tauopathies, including Alzheimer’s disease. Tauopathies are neurodegenerative disorders characterized by the deposition of abnormal tau protein in the brain. ST-502 is in preclinical development for treating synucleinopathies, including Parkinson’s disease. Synucleinopathies are neurodegenerative diseases characterized by the abnormal accumulation of aggregates of alpha-synuclein protein in neurons, nerve fibers, or glial cells.

The companies will leverage Sangamo’s proprietary zinc finger protein (ZFP) technology delivered via adeno-associated virus (AAV) vectors to modulate the expression of key genes involved in neurological diseases. Sangamo’s genome regulation technology, zinc finger protein transcription factors (ZFP-TFs), is currently delivered with AAVs vectors and functions at the DNA level to selectively repress or activate the expression of specific genes to achieve a desired therapeutic effect. Highly specific, potent, and tunable repression of tau and alpha synuclein has been demonstrated in preclinical studies using AAV vectors to deliver tau-targeted (ST-501) and alpha synuclein-targeted (ST-502) ZFP-TFs, according to information from the companies.

Under the terms of the collaboration, Biogen has exclusive global rights to ST-501 for tauopathies, including Alzheimer’s disease, ST-502 for synucleinopathies, including Parkinson’s disease, and a third undisclosed neuromuscular disease target. In addition, Biogen has exclusive rights to nominate up to nine additional undisclosed targets over a target selection period of five years. Sangamo will perform early-research activities, costs for which will be shared by the companies, aimed at the development of the combination of proprietary central nervous system delivery vectors and ZFP-TFs targeting therapeutically relevant genes. Biogen will then assume responsibility and costs for the investigational new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.

Sangamo will be responsible for GMP manufacturing activities for the initial clinical trials for the first three products of the collaboration and plans to leverage its in-house manufacturing capacity. Biogen will assume responsibility for GMP manufacturing activities beyond the first clinical trial for each of the first three products.

Upon closing of this transaction, Sangamo will receive $350 million comprised of $125 million in a license-fee payment and $225 million from the sale of new Sangamo stock, or approximately 24 million shares at $9.21 per share. In addition, Sangamo may receive up to $2.37 billion in other development, regulatory, and commercial 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 to receive from Biogen tiered high single-digit to sub-teen double-digit royalties on potential net commercial sales of products arising from the collaboration. Closing of the transaction is contingent on completion of review under antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the US.

Source: Biogen and Sangamo Therapeutics