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Sanofi Acquires Multiple Sclerosis Drug in $800M Deal

Human brain


10 November 2017. Drug maker Sanofi is licensing a drug candidate from Principia Biopharma Inc. designed to treat multiple sclerosis, an autoimmune condition affecting the brain and nervous system. If all aspects of the agreement are fulfilled, Principia would gain more than $800 million from Sanofi.

Multiple sclerosis is also an autoimmune condition, where the immune system attacks the central nervous system and damages myelin, the fatty, protective substance around nerve fibers, as well as nerve cells themselves. Scar tissue from the damaged myelin, known as sclerosis, distorts the nerve signals sent to and from the brain and spinal cord, causing symptoms ranging from mild numbness to loss of vision or paralysis.

Principia Biopharma, in South San Francisco, California, discovers and develops new therapies with a technology called tailored covalency. Compounds in the company’s portfolio are designed to bind to specific cysteine amino acids in their targets. These amino acids have a binding action different from most others, which allows the therapeutic payloads to break the bonds under certain conditions, making many of the company’s drugs highly targeted and reversible, with fewer adverse effects as a result.

The deal would give Sanofi, based in Paris, an exclusive worldwide license to Principia’s candidate code-named PRN2246, a small-molecule, oral drug that blocks Bruton’s tyrosine kinase, or BTK enzymes. These enzymes are active in signaling pathways affecting B-cells in the immune system, particularly signals promoting autoimmunity. PRN2246, says Principia, can also penetrate the blood-brain barrier, a key property for treating multiple sclerosis, and while it inhibits signals from BTK enzymes to B-cells, it does not deplete the supply of these cells in the immune system.

In addition, says the company, PRN2246 works in the peripheral nervous system, as well as the brain and spinal cord, making it a potential treatment for other neurological and inflammatory disorders. Principia is testing PRN2246 in an early stage clinical trial.

Under the agreement, Sanofi is providing Principia with a $40 million initial payment, with Principia eligible for future milestone payments of $765 million. While Sanofi will be responsible for development and commercialization of PRN2246, Principia has the option to provide financial support for late-stage clinical trials of the drug, in exchange for greater royalties or sharing in profits or losses in the U.S.

PRN2246 is expected to supplement Sanofi’s current presence in the market for multiple sclerosis drugs. Sanofi’s Genzyme division now offers Aubagio, a once-daily oral drug for relapsing multiple sclerosis, and Lemtrada, an infused drug for relapsing multiple sclerosis that does not respond to other treatments. Another therapy for multiple sclerosis code-named GLD52/GZ402668, a synthetic targeted antibody, is in an early-stage clinical trial.

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Hat tip: FirstWord Pharma

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