Pfizer and Sangamo enter gene therapy deal - C&EN Global

Drug giant Pfizer will partner with Sangamo Therapeutics to develop a gene therapy for the neurodegenerative disorders amyotrophic lateral sclerosis a...
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Business Concentrates ▸ First gene therapy costs $850,000

GENE THERAPY The first gene therapy to treat genetic disease approved by the U.S. Food & Drug Administration will cost $850,000. Luxturna, from Spark Therapeutics, is designed to partially restore vision in people with a rare form of inherited blindness. The one-time treatment was expected to cost $1 million or more. Spark will offer rebates to insurance companies if patients do not improve in short- or long-term tests of light sensitivity. Spark is also in talks with the Centers for Medicare & Medicaid Services about letting government or commercial drug payers foot the bill in installments.—RYAN CROSS

PHARMACEUTICALS

▸ Mallinckrodt to buy Sucampo for $1.2 billion Mallinckrodt has agreed to acquire Sucampo Pharmaceuticals, a Rockville, Md.based drugmaker, for $1.2 billion. With the deal, Mallinckrodt will pick up two approved drugs: Amitiza, a treatment for various bowel diseases with 2016 sales of $456 million, and Rescula, a treatment for ocular hypertension and glaucoma with annual sales of about $9 million. Mallinckrodt CEO Mark Trudeau says the deal also

▸ Pfizer and Sangamo enter gene therapy deal Drug giant Pfizer will partner with Sangamo Therapeutics to develop a gene therapy for the neurodegenerative disorders amyotrophic lateral sclerosis and frontotemporal lobar degeneration. The therapy will use a zinc finger protein to bind a region of mutant DNA implicated in familial versions of the diseases. When the zinc finger is present, a second protein attached to it shuts down expression of the disease-causing gene. Sangamo will receive $12 million up front and potential milestones of up to $150 million.—RYAN CROSS

OUTSOURCING

▸ European contractors land deals in the U.S. Two European manufacturers of active pharmaceutical ingredients have signed supply contracts with U.S. biotech firms. France’s Novasep was selected by Tetraphase Pharmaceuticals to manufacture eravacycline, an antibiotic in Phase III development for life-threatening infections.

Business Roundup

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▸ Agrium and PotashCorp have completed their merger, creating the fertilizer maker Nutrien. The firm can make up to 25 million metric tons of potassium, phosphate, and nitrogen fertilizers annually.

▸ BASF and Sinopec will double neopentyl glycol capacity at their joint-venture plant in Nanjing, China, to 80,000 metric tons per year. The plant started production in 2015.

▸ Ineos has made a final decision to build a 120,000-metric-ton-per-year poly(α-olefin) plant in Chocolate Bayou, Texas. The facility, which will mainly serve lubricant markets, is expected to start up during the third quarter of 2019.

▸ Thin Film Electronics has sold intellectual property covering printed rewritable memory labels to Xerox. The two firms inked a partnership to produce the smart labels in 2014. Thin Film will receive an up-front payment plus royalties.

C&EN | CEN.ACS.ORG | JANUARY 8, 2018

Siegfried will manufacture for Keryx at this facility in Pennsville, N.J. Novasep has supplied early-stage clinical quantities since 2014. Meanwhile, the Swiss contractor Siegfried will supply Keryx Biopharmaceuticals with ferric citrate, the active ingredient in the iron deficiency drug Auryxia. Keryx announced a similar agreement last month with the Canadian firm BioVectra.—RICK MULLIN

BIOTECHNOLOGY

▸ BioNTech raises $270 million BioNTech, a German company developing mRNA-based therapies, raised a healthy $270 million in its series A funding, led by Redmile Group. The firm’s mRNA therapies include vaccines for infectious diseases, cancer immunotherapy vaccines, and mRNA to make therapeutic proteins in the body. BioNTech has also added small-molecule and CAR T-cell cancer immunotherapy programs to its pipeline. BioNTech was founded in 2008 by scientists from Johannes Gutenberg University Mainz. Since then it has established pacts with firms such as Bayer Animal Health, Eli Lilly & Co., Genentech, Genmab, and Sanofi.—RYAN CROSS

▸ Nippon Shokubai, PTT, and Mitsui & Co. will study construction of an alcohol ethoxylate surfactants plant at the Hemaraj Eastern Industrial Estate in Rayong, Thailand. PTT produces ethylene oxide, a raw material, at the site. ▸ Gevo will cut 40% of the workforce at its headquarters in Englewood, Colo. The biobased chemicals company seeks to improve its cash flow so it can expand its isobutyl alcohol plant in Luverne, Minn. ▸ Yield10 Bioscience, a plant science spin-off from

biobased polymer firm Metabolix, has raised $14.5 million in a public sale of shares. Yield10 seeks to alter gene activity and carbon flow in oilseeds and rice to improve yields. ▸ Boehringer Ingelheim is more than doubling the size of its corporate venture fund to $300 million. The extra money will go toward early-stage companies focused on regenerative medicine, infectious diseases, and immuno-oncology. The cash will also help Boehringer expand its portfolio in the U.S.

CR E D I T: S IEGF R I ED

GENE THERAPY

bolsters the company’s rare disease drug pipeline with VTS-270, in development for Niemann-Pick disease type C1, and CPP1X/sulindac, in development for familial adenomatous polyposis.—RICK MULLIN