Drug Development

Sanofi jumps into a pivotal study for next-gen Pompe drug

Sanofi has added another badly-needed drug to its late-stage portfolio. The drug, dubbed neoGAA, is being advanced by its big Genzyme unit in Boston, which has just started a Phase III trial for a next-gen enzyme replacement therapy for Pompe disease.

Sanofi reports that the first of 96 patients have been treated with their therapy, which they describe as a second-generation approach to enzyme replacement, a field Genzyme helped pioneer.

Last March Sanofi reported early-stage data showing that neoGAA made a significant difference in a 6-minute walk test among the small group of patients tested. And there were also encouraging signs that the drug helped patients with lung performance as well.

Investigators for the program expect to take up to three years for the Phase III. And their move from clinical proof-of-concept into a pivotal study underscores the trend in drug development to cut out mid-stage work whenever possible.

Investigators for the company describe this drug as a “second-generation alglucosidase alfa enzyme replacement therapy that has been specifically designed for enhanced receptor targeting and enzyme uptake through greater affinity for the M6P receptors on muscle cells, with the aim of enhancing glycogen clearance and improving on the clinical efficacy achieved with alglucosidase alfa.”

Sanofi has had a hard year thus far in 2016. The FDA just handed its drug sarilumab, partnered with Regeneron, a rejection due to the pharma giant’s manufacturing woes. CEO Olivier Brandicourt tried, and failed, to acquire Medivation. And its all-important diabetes franchise has been under assault as prices declined.

Sanofi, though, still has an ace up its sleeves. Regulators should make a decision on dupilumab soon, which is tipped as a likely win — provided Sanofi’s problems on the manufacturing side don’t prohibit the OK.



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Sr. Manager, Regulatory Affairs, CMC
CytomX Therapeutics San Francisco, CA
Marketing Associate - Demand Generation
Catalytic Data Science Charleston, SC
Associate Principal, Life Sciences Partnerships
Flatiron Health New York City or San Francisco

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