The Power of Plus

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POMBILITI

A naturally derived* bis-M6P–enriched enzyme1

 

Designed to be processed into the most active form of
GAA1

 

Bis-M6P has ~3000x higher binding affinity to
CI-MPR than M6P

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OPFOLDA

An enzyme stabilizer3

 

Binds with and stabilizes POMBILITI in the blood to increase the amount of active enzyme available to reach the muscle1,2,6

 

Reduces the inactivation of POMBILITI in the pH of the blood, which is unfavorable for rhGAA1,3-5

*Enzyme derived from a Chinese Hamster Ovary (CHO) cell line using perfusion methodology, resulting in cellularly (CHO)-derived N-glycans.1

POMBILITI + OPFOLDA was developed to address
3 key challenges:

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Maximizing Uptake
to the lysosome with bis-M6P1

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Increasing Enzyme Activity
in the lysosome6

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Improving Stability
in the blood stream6

Designed to maximize enzyme uptake1

Developed to address key challenges in delivering rhGAA with a naturally derived*
bis-M6P–enriched enzyme and stabilizer1,4†

Increase Active Enzyme in the Blood6

OPFOLDA binds with and stabilizes POMBILITI in the blood and increases the amount of active enzyme that can reach the muscle3,6

Maximizing Uptake1

POMBILITI, a naturally derived* bis-M6P–enriched enzyme, in combination with OPFOLDA, binds with high affinity to CI-MPRs on the cell surface to be transported to the lysosome1,6

Complete Processing in the Lysosome1

Once inside the lysosome, OPFOLDA disassociates from POMBILITI3

POMBILITI is completely processed into the most active form of GAA, like endogenous GAA7-9

POMBILITI in its most active form cleaves glycogen into glucose1

*Enzyme derived from a Chinese Hamster Ovary (CHO) cell line using perfusion methodology, resulting in cellularly (CHO)-derived N-glycans.1

†Based on in vitro data.

Watch POMBILITI + OPFOLDA in Action

Adding OPFOLDA increased the availability of POMBILITI in
the blood1

Pharmacokinetics of POMBILITI in the blood in ERT-experienced adults with LOPD

Pharmakinetics graph

These pharmacokinetics data cannot be directly translated into clinical efficacy.

Mean AUC Graph
Mean AUC Graph

AUC, area under the curve; CV, coefficient of variation.

† One subject was not dosed properly with OPFOLDA and was excluded from the analysis.10

‡ POMBILITI 20 mg/kg + OPFOLDA 260 mg.1

§ 20 mg/kg.1

‖ POMBILITI is not approved for use without OPFOLDA.1

AUC0-inf is area under the curve (from time 0 to infinity), representing total drug exposure over time.1,11

POMBILITI + OPFOLDA. Results from the 104 week OLE study.

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AUC, area under the curve; bis-M6P, bis-phosphorylated mannose 6-phosphate; CI-MPR, cation-independent mannose 6-phosphate receptor; CV, coefficient of variation; ERT, enzyme replacement therapy; GAA, acid alpha-glucosidase; LOPD, late-onset Pompe disease; M6P, mannose 6-phosphate; rhGAA, recombinant human acid alpha-glucosidase.