Biphenyl sulfonamide compounds for the treatment of type IV collagen diseases
Inventors
Komers, Radko • Jenkinson, Celia
Assignees
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Publication Number
US-11207299-B2
Publication Date
2021-12-28
Expiration Date
Abstract
Methods of treating Alport syndrome and other diseases associated with a type IV collagen deficiency, and preventing hearing loss associated the same, are provided, comprising administering a compound having structure (I), or a pharmaceutically acceptable salt thereof, or administering a pharmaceutical composition comprising the compound of structure (I) or pharmaceutically acceptable salt thereof.
Core Innovation
The invention relates to a biphenyl sulfonamide compound, sparsentan, in particular a compound having structure (I), and pharmaceutically acceptable salts thereof. The compound is positioned as a dual angiotensin II (AT1) and endothelin (ETA) receptor antagonist for treating diseases associated with type IV collagen deficiency, including Alport syndrome and hearing loss.
The invention relates to a pharmaceutical composition that comprises a compound having structure (I), or a pharmaceutically acceptable salt thereof, for treating hearing loss in a subject having Alport syndrome. The subject is defined by a mutation in a COL4A3, COL4A4, or COL4A5 gene, and the composition is administered to the subject.
A related aspect provides a method of treating a collagen type IV deficiency by administering the pharmaceutical composition containing the compound having structure (I), or a pharmaceutically acceptable salt thereof. Documented use contexts include preventing or treating hearing loss associated with Alport syndrome and type IV collagen deficiency-associated disease, and the compound is associated with dual AT1/ETA inhibition.
Claims Coverage
The independent claims provide two main coverage areas: treating hearing loss in Alport syndrome subjects with COL4A3/COL4A4/COL4A5 mutations, and treating collagen type IV deficiency in a subject. Across the claim family, inventive features are centered on administering a pharmaceutical composition containing a compound having structure (I) or a pharmaceutically acceptable salt, with dependent refinement by age and dosage ranges.
Treating hearing loss in Alport syndrome subjects with COL4A3/COL4A4/COL4A5 mutations
Administering a pharmaceutical composition comprising a compound having structure (I), or a pharmaceutically acceptable salt thereof, to treat hearing loss in a subject having Alport syndrome and a mutation in a COL4A3, COL4A4, or COL4A5 gene.
Treating collagen type IV deficiency by administering a structure (I) compound composition
Administering a pharmaceutical composition comprising a compound having structure (I), or a pharmaceutically acceptable salt thereof, to a subject to treat collagen type IV deficiency.
Adult subject for the structure (I) compound hearing loss treatment
Treating hearing loss in an adult subject by administering a pharmaceutical composition comprising a compound having structure (I), or a pharmaceutically acceptable salt thereof.
Daily dosing of the structure (I) compound for hearing loss treatment
Administering a compound having structure (I), or a pharmaceutically acceptable salt thereof, at a daily amount within stated mg/day dose levels for the hearing loss treatment method.
Weight-based dosing range of the structure (I) compound
Administering a compound having structure (I), or a pharmaceutically acceptable salt thereof, to a subject using stated dosage ranges in mg/kg.
Overall, the claim coverage centers on administering a pharmaceutical composition containing sparsentan, or a pharmaceutically acceptable salt, to treat hearing loss in Alport syndrome and in subjects with COL4A3/COL4A4/COL4A5 mutations, and also to treat collagen type IV deficiency. Dependent refinements emphasize adult subject limitation and dosing ranges in mg/day and mg/kg.
Stated Advantages
Maintaining eGFR (no clinically meaningful decline), maintained for 12 months or more.
Preventing hearing loss is described as a use case linked to Alport syndrome and type IV collagen deficiency-associated disease.
Documented Applications
Treating hearing loss in a subject having Alport syndrome by administering a pharmaceutical composition comprising a compound having structure (I) or a pharmaceutically acceptable salt.
Treating hearing loss in a subject having a mutation in a COL4A3, COL4A4, or COL4A5 gene using a pharmaceutical composition comprising a compound having structure (I) or a pharmaceutically acceptable salt.
Treating collagen type IV deficiency by administering a pharmaceutical composition comprising a compound having structure (I) or a pharmaceutically acceptable salt.
Preventing hearing loss in Alport syndrome and type IV collagen deficiency-associated contexts.
Assessing renal function endpoints including maintaining eGFR constant and at or above pre-dose eGFR in disclosed Alport syndrome embodiments.
Assessing hearing loss endpoints including ABR thresholds and cochlear/strial basement membrane morphology in disclosed Alport syndrome embodiments.
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