Sangamo, Pfizer Deepen Relationship With New $162M ALS R&D Pact

Shares of Sangamo Therapeutics are on the rise this morning after the company announced it signed a  $162 million deal with Pfizer to develop a potential  gene therapy treatment for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD).

Sangamo and Pfizer will use the California-based company’s zinc finger protein transcription factors (ZFP-TFs) to develop the treatments that are linked to mutations of the C9ORF72 gene, which can cause both ALS and FTLD. The C9ORF72 mutation is linked to approximately one-third of cases of familial ALS. Under this collaboration, Sangamo and Pfizer will investigate allele-specific ZFP-TFs with the potential to differentiate the mutant C9ORF72 allele from the wild type allele and to specifically down-regulate expression of the mutant form of the gene, the company said in its announcement.

Under terms of the agreement, Sangamo will receive a $12 million upfront payment from Pfizer. Sangamo will be eligible for an additional $150 million in development and commercial milestone payments. The California-based company will be responsible for the development of the ZFP-TF candidates, according to the agreement.

Also called Lou Gehrig’s disease, ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. The Centers for Disease Control and Prevention estimates that approximately 12,000 to 15,000 Americans have ALS, with about 5,000 to 6,000 diagnosed annually. The average life expectancy of an ALS patient is approximately three to five years after diagnosis and only 10 percent of patients survive for more than 10 years. Death usually is a result of respiratory failure.

FTLD is a neurodegenerative disease that affects the frontal and anterior temporal regions of the brain.

This is the second collaboration that Sangamo and Pfizer have inked. In May 2017, the companies forged a gene therapy development agreement targeting Hemophilia A. That agreement includes SB-525, one of Sangamo's four lead product candidates. SB-525 entered the clinic in August 2017.

Sangamo Chief Executive Officer Sandy Macrae said the company was excited about the continued collaboration with Pfizer.

"The precision and flexibility of zinc finger proteins enables targeting of virtually any genetic mutation. Collaboration with the right partner for a given therapeutic application is a key component of our corporate strategy and enables us to pursue the vast opportunity set of our platform," Macrae said in a statement.

Sangamo’s zinc finger nuclease (ZFN) genome editing technology allows the company to insert a corrective gene designed to identify and bind to a precise sequence of DNA into a precise location to target a specific disease. Once bound to the target sequence of DNA, a transcriptional repressor domain attached to the ZFP suppresses expression of the gene. In November 2017, the company dosed the first patient in its Phase I/II gene therapy trial for people with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

Greg LaRosa, chief scientific officer for Pfizer Rare Disease, said the gene therapy programs developed by Sangamo offer "tremendous promise" to patients and their families.

In addition to the agreements with Pfizer, Sangamo has also entered into a collaborative partnership with Shire PLC for Huntington's disease.

Shares of Sangamo are trading at $18.47 as of 10:15 a.m.