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Biotechs Partner on Gene Silencing in $1.1B Deal

Retinal gene therapy

(National Human Genome Research Institute, NIH)

8 Apr. 2019. Two biotechnology companies plan to develop new therapies mainly for eye and neurological diseases that silence genes responsible for those conditions. The deal with Regeneron Pharmaceuticals Inc. in Tarrytown, New York could bring Alnylam Pharmaceuticals Inc. in Cambridge, Massachusetts more than $1.1 billion if all aspects of the agreement are completed.

The agreement calls for Regeneron and Alnylam to collaborate on treatments for eye disorders and diseases of the central nervous system, as well as select targets in the liver, based on Alnylam’s RNA interference technology that silences the actions of specific genes. RNA interference is a natural process to silence the expression of genes causing disease. RNA interference targets specific genes, making it a potentially powerful therapeutic technique, while minimizing damage to other genes, thus limiting side effects.

In this case, the interfering RNA molecules are small in size, with limited numbers of amino acids, and are known as small interfering RNAs or siRNAs. And these siRNAs effectively stop the coding and production of proteins responsible for the disease. In August 2018, FDA approved the first siRNA treatment that reverses damage to nerves from a rare inherited neurological disease, made by Alnylam.

Regeneron develops treatments for a wide range of diseases with synthetic proteins derived from mouse DNA. Regeneron’s pipeline includes synthetic targeted human antibodies derived from mice that the company says avoid triggering immune responses. The company is developing other derivatives from mice genetics, such as synthetic T-cell receptor proteins and bi-specific human antibodies that address 2 targets.

Regeneron also has a separate Regeneron Genetics Center that collects and analyzes genomic data from 400,000 exomes, the protein coding regions of the genome, related to non-identified health records from those individuals. Exomes account for only a small percentage of base pairs in the genome, but they represent about 85 percent of all disease causing mutations.

Alnylam and Regeneron are already partnering on discovery of a gene-silencing treatment for nonalcoholic steatohepatitis, or NASH, a chronic liver disease, a deal begun in March 2018. In that collaboration, the companies are developing a gene-silencing solution acting like a natural variation of the HSD17B13 gene that protects some individuals against progression of NASH. Regeneron is contributing insights on the gene from the Regeneron Genetics Center, while Alnylam is identifying gene-silencing candidates addressing that target.

In the new collaboration, Regeneron gains exclusive access to Alnylam’s technology to discover gene-silencing treatments for eye and central nervous system disorders. Regeneron will lead development and commercialization of eye disease therapies, while the two companies will alternate leadership, but still jointly develop central nervous system therapies. Once candidates for neurological treatments are selected, the companies will also have an option to take part in profits of programs led by the other party. Collaboration on therapies for liver diseases builds on the companies’ existing NASH partnership, adds in the companies’ current clinical-stage work on liver diseases, and spells out a roadmap for further partnering on related treatments.

Under the 5-year agreement, Regeneron is providing Alnylam with an initial payment of $400 million, and is taking a nearly $400 million equity stake in Alnylam, gaining 4.4 million Alnylam shares at $90.00. Alnylam is also eligible for $200 million in milestone payments for the companies’ joint efforts in therapies for eye and central nervous system disorders. In addition, for each of the 30 targets addressed during the project, Alnylam is eligible for $2.5 million per target at the start of a program and another $2.5 million when a lead candidate is identified, for a total potential payout of $150 million.

The deal also calls for Alnylam to take part with other life science enterprises in DNA sequencing of 500,000 individuals in the U.K. Biobank, with the data made available to the worldwide research community. In March, Science & Enterprise reported on the first batch of data from the U.K. Biobank, sequenced by Regeneron, showing strong associations between several gene variations and common health conditions.

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