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- Nachgewiesen in: USPTO Patent Applications
- Sprachen: English
- Document Number: 20210177942
- Publication Date: June 17, 2021
- Appl. No: 17/184561
- Application Filed: February 24, 2021
- Assignees: ASCENDIS PHARMA GROWTH DISORDERS A/S (HELLERUP, DK)
- Claim: 1-20. (canceled)
- Claim: 21. A compound of formula (IIf) [chemical expression included] or a pharmaceutically acceptable salt thereof; wherein the unmarked dashed line indicates the attachment to a nitrogen provided by the side chain of the lysine at position 26 of a CNP moiety of SEQ ID NO:24 by forming an amide bond; and the dashed line marked with the asterisk indicates attachment to —Z having the structure [chemical expression included] wherein each —Za is [chemical expression included] wherein each c1 is an integer independently ranging from 200 to 250.
- Claim: 22. The compound or pharmaceutically acceptable salt thereof of claim 21, wherein c1 is about 225.
- Claim: 23. The compound or pharmaceutically acceptable salt thereof of claim 21, wherein the compound is of formula (IIf′) [chemical expression included] wherein the unmarked dashed line indicates the attachment to a nitrogen provided by the side chain of the lysine at position 26 of the CNP moiety of SEQ ID NO:24 by forming an amide bond; and the dashed line marked with the asterisk indicates attachment to —Z having the structure [chemical expression included] wherein each Za is [chemical expression included] wherein each c1 is an integer independently ranging from 200 to 250.
- Claim: 24. The compound or pharmaceutically acceptable salt thereof of claim 23, wherein c1 is about 225.
- Claim: 25. A pharmaceutical composition comprising the compound or pharmaceutically acceptable salt thereof of claim 21 and at least one excipient.
- Claim: 26. A pharmaceutical composition comprising the compound or pharmaceutically acceptable salt thereof of claim 22 and at least one excipient.
- Claim: 27. A pharmaceutical composition comprising the compound or pharmaceutically acceptable salt thereof of claim 23 and at least one excipient.
- Claim: 28. A pharmaceutical composition comprising the compound or pharmaceutically acceptable salt thereof of claim 24 and at least one excipient.
- Claim: 29. A method of treating a patient suffering from a disease that can be treated with CNP, the method comprising the step of administering an effective dose of the compound or pharmaceutically acceptable salt thereof of claim 21 to the patient.
- Claim: 30. The method of claim 29, wherein the disease is selected from the group consisting of achondroplasia, hypochondroplasia, short stature, dwarfism, osteochondrodysplasias, thanatophoric dysplasia, osteogenesis imperfecta, achondrogenesis, chondrodysplasia punctata, homozygous achondroplasia, camptomelic dysplasia, congenital lethal hypophosphatasia, perinatal lethal type of osteogenesis imperfecta, short-rib polydactyly syndromes, rhizomelic type of chondrodysplasia punctata, Jansen-type metaphyseal dysplasia, spondyloepiphyseal dysplasia congenita, atelosteogenesis, diastrophic dysplasia, congenital short femur, Langer-type mesomelic dysplasia, Nievergelt-type mesomelic dysplasia, Robinow syndrome, Reinhardt syndrome, acrodysostosis, peripheral dysostosis, Kniest dysplasia, fibrochondrogenesis, Roberts syndrome, acromesomelic dysplasia, micromelia, Morquio syndrome, Kniest syndrome, metatrophic dysplasia, spondyloepimetaphyseal dysplasia, neurofibromatosis, Legius syndrome, LEOPARD syndrome, Noonan syndrome, hereditary gingival fibromatosis, neurofibromatosis type 1, Legius syndrome, cardiofaciocutaneous syndrome, Costello syndrome, SHOX deficiency, idiopathic short stature, growth hormone deficiency, osteoarthritis, cleidocranial dysostosis, craniosynostosis (e.g., Muenke syndrome, Crouzon syndrome, Apert syndrome, Jackson-Weiss syndrome, Pfeiffer syndrome, or Crouzonodermoskeletal syndrome), dactyly, brachydactyly, camptodactyly, polydactyly, syndactyly, dyssegmental dysplasia, enchondromatosis, fibrous dysplasia, hereditary multiple exostoses, hypophosphatemic rickets, Jaffe-Lichtenstein syndrome, Marfan syndrome, McCune-Albright syndrome, osteopetrosis and osteopoikilosis.
- Claim: 31. The method of claim 30, wherein the disease is achondroplasia.
- Claim: 32. The method of claim 31, wherein the patient is a pediatric patient.
- Claim: 33. The method of claim 31, wherein c1 of the compound is about 225.
- Claim: 34. The method of claim 31, wherein the compound is of formula (IIf′) [chemical expression included] wherein the unmarked dashed line indicates the attachment to a nitrogen provided by the side chain of the lysine at position 26 of the CNP moiety of SEQ ID NO:24 by forming an amide bond; and the dashed line marked with the asterisk indicates attachment to —Z having the structure [chemical expression included] wherein each Za is [chemical expression included] wherein each c1 is an integer independently ranging from 200 to 250.
- Claim: 35. The method of claim 34, wherein c1 of the compound is about 225.
- Claim: 36. The CNP prodrug or a pharmaceutically acceptable salt thereof of claim 1, wherein the CNP prodrug is of the following formula: [chemical expression included] wherein the unmarked dashed line indicates the attachment to a nitrogen provided by the side chain of the lysine at position 26 of the CNP moiety of SEQ ID NO:24 by forming an amide bond; and the dashed line marked with the asterisk indicates attachment to —Z having the structure [chemical expression included] wherein each Za is [chemical expression included] wherein c1 is ranging from 200 to 250.
- Claim: 37. The CNP prodrug or a pharmaceutically acceptable salt thereof of claim 36, wherein c1 is about 225.
- Claim: 38. A pharmaceutical composition comprising the compound or pharmaceutically acceptable salt thereof of claim 36 and at least one excipient.
- Claim: 39. A pharmaceutical composition comprising the compound or pharmaceutically acceptable salt thereof of claim 37 and at least one excipient.
- Claim: 40. A method of treating a patient suffering from a disease that can be treated with CNP, the method comprising the step of administering an effective dose of the compound or pharmaceutically acceptable salt thereof of claim 36 to the patient.
- Claim: 41. The method of claim 40, wherein the disease is selected from the group consisting of achondroplasia, hypochondroplasia, short stature, dwarfism, osteochondrodysplasias, thanatophoric dysplasia, osteogenesis imperfecta, achondrogenesis, chondrodysplasia punctata, homozygous achondroplasia, camptomelic dysplasia, congenital lethal hypophosphatasia, perinatal lethal type of osteogenesis imperfecta, short-rib polydactyly syndromes, rhizomelic type of chondrodysplasia punctata, Jansen-type metaphyseal dysplasia, spondyloepiphyseal dysplasia congenita, atelosteogenesis, diastrophic dysplasia, congenital short femur, Langer-type mesomelic dysplasia, Nievergelt-type mesomelic dysplasia, Robinow syndrome, Reinhardt syndrome, acrodysostosis, peripheral dysostosis, Kniest dysplasia, fibrochondrogenesis, Roberts syndrome, acromesomelic dysplasia, micromelia, Morquio syndrome, Kniest syndrome, metatrophic dysplasia, spondyloepimetaphyseal dysplasia, neurofibromatosis, Legius syndrome, LEOPARD syndrome, Noonan syndrome, hereditary gingival fibromatosis, neurofibromatosis type 1, Legius syndrome, cardiofaciocutaneous syndrome, Costello syndrome, SHOX deficiency, idiopathic short stature, growth hormone deficiency, osteoarthritis, cleidocranial dysostosis, craniosynostosis (e.g., Muenke syndrome, Crouzon syndrome, Apert syndrome, Jackson-Weiss syndrome, Pfeiffer syndrome, or Crouzonodermoskeletal syndrome), dactyly, brachydactyly, camptodactyly, polydactyly, syndactyly, dyssegmental dysplasia, enchondromatosis, fibrous dysplasia, hereditary multiple exostoses, hypophosphatemic rickets, Jaffe-Lichtenstein syndrome, Marfan syndrome, McCune-Albright syndrome, osteopetrosis and osteopoikilosis.
- Claim: 42. The method of claim 40, wherein the disease is achondroplasia.
- Claim: 43. The method of claim 40, wherein the patient is a pediatric patient.
- Claim: 44. A method of treating a patient suffering from a disease that can be treated with CNP, the method comprising the step of administering an effective dose of the compound or pharmaceutically acceptable salt thereof of claim 37 to the patient.
- Claim: 45. The method of claim 44, wherein the disease is selected from the group consisting of achondroplasia, hypochondroplasia, short stature, dwarfism, osteochondrodysplasias, thanatophoric dysplasia, osteogenesis imperfecta, achondrogenesis, chondrodysplasia punctata, homozygous achondroplasia, camptomelic dysplasia, congenital lethal hypophosphatasia, perinatal lethal type of osteogenesis imperfecta, short-rib polydactyly syndromes, rhizomelic type of chondrodysplasia punctata, Jansen-type metaphyseal dysplasia, spondyloepiphyseal dysplasia congenita, atelosteogenesis, diastrophic dysplasia, congenital short femur, Langer-type mesomelic dysplasia, Nievergelt-type mesomelic dysplasia, Robinow syndrome, Reinhardt syndrome, acrodysostosis, peripheral dysostosis, Kniest dysplasia, fibrochondrogenesis, Roberts syndrome, acromesomelic dysplasia, micromelia, Morquio syndrome, Kniest syndrome, metatrophic dysplasia, spondyloepimetaphyseal dysplasia, neurofibromatosis, Legius syndrome, LEOPARD syndrome, Noonan syndrome, hereditary gingival fibromatosis, neurofibromatosis type 1, Legius syndrome, cardiofaciocutaneous syndrome, Costello syndrome, SHOX deficiency, idiopathic short stature, growth hormone deficiency, osteoarthritis, cleidocranial dysostosis, craniosynostosis (e.g., Muenke syndrome, Crouzon syndrome, Apert syndrome, Jackson-Weiss syndrome, Pfeiffer syndrome, or Crouzonodermoskeletal syndrome), dactyly, brachydactyly, camptodactyly, polydactyly, syndactyly, dyssegmental dysplasia, enchondromatosis, fibrous dysplasia, hereditary multiple exostoses, hypophosphatemic rickets, Jaffe-Lichtenstein syndrome, Marfan syndrome, McCune-Albright syndrome, osteopetrosis and osteopoikilosis.
- Claim: 46. The method of claim 44, wherein the disease is achondroplasia.
- Claim: 47. The method of claim 44, wherein the patient is a pediatric patient.
- Current International Class: 61; 61; 61; 07; 61; 61; 61
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