Biogen, a large biotech selected from among several companies in a competitive process, is partnering with Sangamo, a firm developing cell and gene therapy treatments for neurological diseases like Alzheimer’s and Parkinson’s, in a deal worth up to $2.37 billion.
The announcement was made on February 27th by the two companies, stating that they have a global licensing collaboration agreement to develop and commercialize a number of drugs. Beside ST-501 for tauopathies including Alzheimer’s disease, and ST-502 for synucleinopathies, including Parkinson’s disease, Biogen and Sangamo will work together on some undisclosed therapy targets: a neuromuscular one and up to nine more neurological disease targets. The companies will make use of Sangamo’s proprietary zinc finger protein (ZFP) technology delivered via adeno-associated virus (AAV) to modulate the expression of key genes involved in neurological diseases.
“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,” said Alfred Sandrock Jr., M.D., Ph.D., Executive Vice President, Research and Development at Biogen.
Gene therapeutics are making an impact on the drug markets lately. Beam Therapeutics Inc., a biotechnology company developing precision genetic medicines through base editing, raised $207 million last month to fund its research.
Biogen is among the drug producers who recently increased prices, despite criticism from President Trump and the lawmakers. Biogen, one of the world’s first global biotechnology companies, practiced a price augmentation on the lower end of the spectrum, increasing the price of Tecfidera, a multiple sclerosis drug, by 6 percent.
The company’s association with Sangamo is expected to prove very beneficial, especially for patients suffering from debilitating diseases.
“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,” said 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.”
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. Biogen also 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, in order to develop the combination of proprietary CNS delivery vectors and ZFP-TFs targeting therapeutically relevant genes. After that, Biogen will be responsible for costs and legal aspects of the investigational new drug-enabling studies, clinical development, related regulatory interactions, and global commercialization.
Sangamo will be responsible for GMP manufacturing activities during the initial clinical trials for the first three products of the collaboration, after which Biogen will assume responsibility for GMP manufacturing activities for each of these.
“The combination of Sangamo’s proprietary zinc finger technology, Biogen’s 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 Sangamo’s 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.”
Closing of the transaction is contingent on completion of the review under antitrust laws, including the Hart-Scott-Rodino (HSR) Antitrust Improvements Act of 1976 in the U.S.
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. 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 million in the first commercial sale and other sales-based milestone payments. Sangamo will also be eligible to receive tiered high single-digit to sub-teen double-digit royalties from Biogen on potential net commercial sales of products arising from the collaboration.
Biogen’s mission is clear: they are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics, and is focused on advancing research programs in multiple sclerosis and neuroimmunology, Alzheimer’s disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology, and pain.
Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients’ lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation.