US 11,400,067 C1 (253rd)
Pharmaceutical compositions and uses directed to lysosomal storage disorders
Michael Strupp, Munich (DE)
Filed by INTRABIO LIMITED, Oxfordshire (GB)
Assigned to INTRABIO LIMITED
Supplemental Examination Request No. 96/050,055, Oct. 9, 2024.
Reexamination Certificate for Patent 11,400,067, issued Aug. 2, 2022, Appl. No. 16/324,301, Feb. 8, 2019.
PCT Filed Aug. 11, 2017, PCT No. PCT/IB2017/054928
§ 371(c)(1), (2) Date Feb. 8, 2019,
PCT Pub. No. WO2018/029657, PCT Pub. Date Feb. 15, 2018.
This patent is subject to a terminal disclaimer.
Ex Parte Reexamination Certificate from Reexamination Ordered under 35 U.S.C. 257 issued on May 28, 2025.
Int. Cl. A61K 31/198 (2006.01); A61K 31/13 (2006.01); A61P 25/00 (2006.01)
CPC A61K 31/198 (2013.01) [A61K 31/13 (2013.01); A61P 25/00 (2018.01)]
AS A RESULT OF REEXAMINATION, IT HAS BEEN DETERMINED THAT:
The patentability of claims 7 and 32-34 is confirmed.
Claims 1-6 and 28 are determined to be patentable as amended.
Claims 8-27 and 29-31, dependent on an amended claim, are determined to be patentable.
New claims 35-43 are added and determined to be patentable.
1. A method of treating a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD in a subject in need thereof comprising:
administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years, wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria.
2. A method of delaying progression of a lysosomal storage disorder (LSD) or [ one or ] more symptoms associated with the LSD over time as compared to typical disease progression in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least [about] 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years [ , wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria] .
3. A method of reversing progression of a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD over time in a subject in need thereof comprising: administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years [ , wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria] .
4. A method of improving in a subject in need thereof a biochemical marker of a lysosomal storage disorder (LSD) over time comprising:
administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years [ , wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria] .
5. A method of reducing the severity of a lysosomal storage disorder (LSD) or reducing the severity of or eliminating one or more existing symptoms associated with the LSD in a subject in need thereof comprising:
administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject, wherein the LSD is not Niemann-Pick Type C [ , and wherein the LSD is chosen from Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria] .
6. A method of providing neuroprotection in a subject having, suspected of having, or at risk of having a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD comprising:
administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least about 3 months, at least about 6 months, at least about 1 year, at least about 2 years, and at least about 5 years [ , wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS_III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria] .
28. The method according to claim 5, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis.
[ 35. A method of treating Niemann-Pick Type C (NPC) disease or one or more symptoms associated with NPC in a subject in need thereof comprising:
administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least 3 months, at least 6 months, at least 1 year, at least 2 years, and at least 5 years.]
[ 36. A method of treating a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD in a subject in need thereof comprising:
administering a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration chosen from at least 6 months, at least 1 year, at least 2 years, and at least 5 years, wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria.]
[ 37. The method according to claim 36, wherein the acetyl-leucine is acetyl-DL-leucine.]
[ 38. The method according to claim 36, wherein the acetyl-leucine has an enantiomeric excess of the L-enantiomer or the D-enantiomer.]
[ 39. The method according to claim 36, wherein the acetyl-leucine is in a single enantiomeric form of either the L-enantiomer or the D-enantiomer.]
[ 40. The method according to claim 39, wherein the single enantiomeric form is the L-enantiomer.]
[ 41. The method according to claim 36, wherein the method comprises administering the acetyl-leucine to the subject in need thereof at a therapeutically effective amount of from about 1 g to about 15 g per day.]
[ 42. The method according to claim 36, wherein the LSD is chosen from Niemann-Pick type A disease, Niemann-Pick type B disease, and Niemann-Pick type C disease.]
[ 43. The method according to claim 36, wherein the LSD is chosen from Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type C disease, mucolipidosis II, MPS III, and GM1 gangliosidosis.]