Sanofi Presents Safety Data from Phase 1/2 in Pompe

Last night at the 14^th Annual WORLDSymposium 2018 in San Diego, California, Sanofi Genzyme presented results from the Phase 1/2 trial (NEO1) and its ongoing extension study (NEO-EXT), showing consistent safety data for the investigational avalglucosidase alfa (neoGAA) in patients with late-onset Pompe disease.

The data showed no evidence of inhibition of enzyme activity over the analysis period.

In patients with Pompe disease, a deficiency of the lysosomal acid alpha-glucosidase enzyme causes an accumulation of glycogen in the lysosome. NeoGAA is an enzyme replacement therapy that is being developed for selective receptor targeting and enzyme uptake, intended to enhance glycogen clearance.

The NEO1 study enrolled 24 patients with Pompe disease, including 10 who had not previously been on treatment and 14 who had been treated with alglucosidase alfa for at least 9 months. After completing NEO1, in which patients received avalglucosidase alfa at 5, 10 or 20 mg/kg once every other week, 19 of 24 patients entered the NEO-EXT study, of which 18 are still enrolled.

All patients in the study have been switched to receive avalglucosidase alfa 20 mg/kg every other week. Participants who remain in the study have continued to receive avalglucosidase alfa for an organized 6-year follow-up.

“These results are reassuring and indicate that the safety data for avalglucosidase alfa is consistent in longer term treatment with what we saw in the initial six months of the trial,” said Loren D.M. Pena, M.D., Ph.D, Associate Professor of Pediatrics at Duke University School of Medicine. “These data add to the body of research supporting avalglucosidase alfa

as a potential new treatment option for patients living with Pompe disease.”

Across both studies at all dose levels, treatment-emergent adverse events (AEs) were mostly mild, and the most common included nausea, headache and fatigue. Each occurred in 12.5% of patients. Additionally, 8.3% of patients experienced dizziness, erythema, muscle spasm, myalgia, dyspnea and rash.

“The development of avalglucosidase alfa builds on Sanofi’s decades of commitment to research targeting new treatments for unmet needs in rare diseases, including Pompe disease,” said Rand Sutherland, M.D., Global Head, Rare Disease Development for Sanofi. “These results provide important additional momentum to our efforts to potentially bring avalglucosidase alfa to patients with Pompe disease.”

In addition to the NEO-EXT trial, neoGAA is also being evaluated in the COMET trial, a Phase 3 study to compare the efficacy and safety of bi-weekly infusions of it in combination with alglucosidase alfa in patients with late-onset Pompe disease who have not receiver prior treatment.

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