RU-2023100586-A - COMPOSITIONS AND METHODS FOR THE TREATMENT OF LATE-ONSET POMPE DISEASE

Inventors

• ФИНН, Патрик
• ХАМЕД, Алаа
• АН ХААК, Кристина
• УИЛСОН, Кэтрин

Assignees

• ДЖЕНЗИМ КОРПОРЕЙШН

Dates

Publication Date

20260505

Application Date

20210611

Priority Date

20200614

Claims (20)

1. 1. A method for treating late-onset Pompe disease (LOPD) in a human subject in need thereof, comprising administering to the subject a pharmaceutical composition comprising an oligosaccharide-protein conjugate and a pharmaceutically acceptable carrier, wherein the oligosaccharide-protein conjugate is characterized by the structure of formula I
2. where GAA is acid α-glucosidase, L is a chemical linker connecting the oligosaccharide and GAA, and n is from 1 to 10,
3. wherein the pharmaceutical composition is administered at a dose of approximately 20 mg/kg,
4. in this case, the individual is characterized by one or more of the following characteristics:
5. (a) the age of the individual at the time of initiation of treatment is between 3 and 18 years;
6. (b) the individual has a forced vital capacity (FVC) percentage of predicted value of between approximately 30% and approximately 50% prior to treatment, and
7. (c) the individual can walk approximately 40 to approximately 50 meters without stopping or using an assistive device before receiving treatment.
8. 2. The method of claim 1, wherein the age of the individual at the time of initiation of treatment is from about 3 to about 18 years.
9. 3. The method of claim 1 or 2, wherein the individual has a forced vital capacity (FVC) percentage of predicted value of between about 30% and about 50% prior to treatment.
10. 4. The method of any one of the preceding claims, wherein the individual can walk from about 40 to about 50 meters without stopping or using the assistive device before receiving treatment.
11. 5. The method of any one of the preceding claims, wherein administering the pharmaceutical composition to a plurality of individuals over 49 weeks results in one or more of the following:
12. (a) an average increase in FVC% while standing of at least about 1.1%, at least about 2.3%, or at least about 4.6%;
13. (b) an average increase in 6-minute walk test (6MWT) distance of at least about 12.5 m, at least about 23 m, or at least about 50 m;
14. (c) an average increase in the percentage of 6MWT from the predicted value (6MWT%) of at least about 2%, at least about 3.5%, or at least about 8%;
15. (d) a mean increase in the percentage of maximum inspiratory pressure (MIP%) from the predicted value in the standing position of at least about 4.5%, at least about 8.7%, or at least about 12.5%;
16. (e) a mean increase in the percentage of maximum expiratory pressure (MEP%) in the standing position of at least about 5.2%, at least about 10.9%, at least about 14.3%, or at least about 16.5%;
17. (f) an average increase in the lower limb hand strength (HHD) of at least about 170 newtons (N), at least about 250 N, or at least about 350 N;
18. (g) an average increase in the upper limb HHD of at least about 95 N, at least about 190 N, or at least about 245 N;
19. (h) a mean increase in the total score on the Quick Motor Function Test (QMFT) of at least about 2.7, at least about 3.3, or at least about 5.2;
20. (i) a mean change in the Walk-Stair-Gowers-Chair-Rise (GSGC) test score of at least about -0.9, at least about -0.35, or at least about 0.40;