PATENT CLAIM ANALYSIS

Application Number: 16066555
Application Type: Utility
Filing Date: 2018-06
Publication Date: 2019-01
Patent Classification: ["514", "233200"]

Abstract:
The invention provides compounds for activating the activating transcription factor 6 (ATF6) arm of the unfolded protein response (UPR), or activating the transcriptional targets of ATF6, in the endoplasmic reticulum of a cell, the compounds being of any of formulas (I) through (IX) as described herein. The compounds can be used for treatment of conditions involving gain-of-toxic-function and loss-of-function folding disorders including lysosomal storage diseases, antitrypsin-associated emphysema and similar diseases. These molecules are also expected to have disease-ameliorating effects in Alzheimer's disease and diabetes.

Claim (Index 1):
A method of treatment of a protein misfolding disease in a patient afflicted therewith, comprising administering to the patient an effective amount of:\n (1) a compound of formula (I) wherein Q is S, O, CH 2 , CHF, or CF 2 , n=1, 2, 3, or 4, when Q is CH 2 , CHF, or CF 2 ; n=1 when Q is S or O, and V, W, X, Y and Z are each independently hydrogen, halogen, alkyl, alkenyl, alkynyl, or alkoxy; or a pharmaceutically acceptable salt thereof; or (2) a compound of formula (IIA) or (IIB) wherein R alkyl, alkenyl, alkynyl, cycloalkyl, or cycloalkenyl, or is a 5- or 6-membered aryl or heteroaryl; wherein non-hydrogen R group can be unsubstituted or substituted; or a pharmaceutically acceptable salt thereof, or (3) a compound of formula (IIIA) or of formula (IIIB) wherein R is unsubstituted or substituted alkyl, alkenyl, cycloalkyl, cycloalkyenyl, aryl, arylalkyl, heteroaryl, or heteroarylalkyl; Ar is aryl or heteroaryl, wherein any aryl or heteroaryl of Ar can be unsubstituted or substituted with halo, alkoxy, or hydroxyl; or R and R\u2032 together can form a substituted or unsubstituted 5- or 6-membered cycloalkyl or heterocycloalkyl; or a pharmaceutically acceptable salt thereof; or (4) a compound of formula (IV) wherein V, W, X, Y, and Z are each independently H, halo, alkyl, alkenyl, or alkoxy; and wherein the ring bearing W can be a phenyl, or can be a 2-thienyl ring or an analogous 5 membered heteroaromatic ring wherein only groups V, W, and X are present: or a pharmaceutically acceptable salt thereof;\n or \n (5) a compound of formula (V) \n wherein m=1, 2, or 3; Y is cyano or carboxamido; and Ar 1  is any one of wherein Q is O or S, X is halo, alkyl, alkenyl, alkynyl, or alkoxy, and n=0, 1, 2, or 3; and a wavy line indicates a point of bonding or attachment; or a pharmaceutically acceptable salt thereof; or, (6) a compound of formula (VIA) wherein each X is independently H, hydroxyl, halo, amine, nitro, (C1-C4)alkyl, (C2-C4) alkynyl, (C1-C4) alkyl ester, or (C1-C4)alkoxy, (C2-C4) alkenyl, (C1-C4) fluoroalkyl, benzoyl, mesyl; and n=0, 1, 2, or 3; Z is NR, CR2 or S, each R is independently H, (C1-C4)alkyl, (C2-C4) alkenyl, (C2-C4)alkynyl, benzyl, (C1-C4) alkyl ester, (C1-C4)alkoxy, or (C1-C4) fluoroalkyl; a wavy line indicates a point of bonding or attachment of Y, which is any one of, or of formula (VIB) wherein each X is independently H, hydroxyl, halo, amine, nitro, (C1-C4)alkyl, (C2-C4) alkynyl, (C1-C4) alkyl ester, or (C1-C4)alkoxy, (C2-C4) alkenyl, (C1-C4) fluoroalkyl, mesyl; and n=0, 1, 2, or 3; Z is NR, CR2 or S, each R is independently H, (C1-C4)alkyl, (C2-C4) alkenyl, (C2-C4)alkynyl, (C1-C4) alkyl ester, (C1-C4)alkoxy, or (C1-C4) fluoroalkyl; a wavy line indicates a point of bonding or attachment of Y and W, which is any one of, wherein Q is O or S; each V is independently H, hydroxyl, halo, amine, nitro, (C1-C4)alkyl, (C2-C4)alkynyl, (C1-C4) alkyl ester, or (C1-C4)alkoxy, (C2-C4) alkenyl, (C1-C4) fluoroalkyl, mesyl; and n=0, 1, 2, or 3, or a pharmaceutically acceptable salt thereof;\n or \n or of formula (VIC) wherein each X is independently H, hydroxyl, halo, amine, nitro, (C1-C4)alkyl, (C2-C4) alkynyl, (C1-C4) alkyl ester, or (C1-C4)alkoxy, (C2-C4) alkenyl, (C1-C4) fluoroalkyl, mesyl; and n=0, 1, 2, or 3; Z is NR, CR2 or S, each R is independently H, (C1-C4)alkyl, (C2-C4) alkenyl, (C2-C4)alkynyl, (C1-C4) alkyl ester, (C1-C4)alkoxy, or (C1-C4) fluoroalkyl; a wavy line indicates a point of bonding or attachment of Y and W, which is any one of, wherein Q is O or S; each V is independently H, hydroxyl, halo, amine, nitro, (C1-C4)alkyl, (C2-C4)alkynyl, (C1-C4) alkyl ester, or (C1-C4)alkoxy, (C2-C4) alkenyl, (C1-C4) fluoroalkyl, mesyl; and n=0, 1, 2, or 3, or a pharmaceutically acceptable salt thereof;\n or \n (7) a compound of formula (VIIA) \n or a compound of formula (VIIB) or a compound of formula (VIIC) wherein Q is O or S; W\u2550C,N,S,O; R 4  is H, OH, halo or (C1-C4)alkyl, or (C1-C4) alkoxy; X is halo, nitro, (C1-C4)alkyl, (C2-C4)alkynyl, (C1-C4) alkyl ester, or (C1-C4)alkoxy, (C2-C4) alkenyl, (C1-C4) fluoroalkyl; m=1, 2, or 3, and n=0, 1, 2, or 3; R 5  is independently at each occurrence any one of: or a pharmaceutically acceptable salt thereof; or (8) a compound of formula (VIII) wherein R 6  is any one of R7  is X is H, halo, hydroxyl, nitro, (C1-C4)alkyl, (C2-C4)alkynyl, (C1-C4) alkyl ester, (C1-C4)alkoxy, (C2-C4) alkenyl, or (C1-C4) fluoroalkyl; m=0, 1, or 2, and n=0, 1, 2, or 3; or a pharmaceutically acceptable salt thereof; or (9) a compound of formula (IX) wherein X is hydroxyl or amine, and Ar 1  is any one of: or a pharmaceutically acceptable salt thereof.

Metadata:
- Claim Count in Document: 32.0
- Percentile: 94.0
- Lexical Diversity: 1.44928
- Patent Class: 514.0
- Transitional Phrase Type: open
- Component Type: 1
- Foreign Priority: False
- Related Applications: ['14849428', '15614278', '12992128', '12280793', '11217137']

Analysis Scores:
- 35 USC 101 Eligibility (BERT): 0.7387959293134065
- 35 USC 102 Novelty (BERT): 0.5269769780400766
- Combined Prediction Score: 0.7176140341860736
- Mean Citation Score: 301.845864
- Max Citation Score: 336.1769700000001
- Similarity Product: 290.8040629204989

Labels:
- Claim Label 101: 1
- Claim Label 102: 1
- Claim Label 103: 1
- Claim Label 112: 0
- Combined Label: 1
- Label 101 Adjusted: 1

Dataset: test