IP Library Patent Application 17069218
Patent Application
App. No. 17/069,218

METHODS FOR TREATING IMMUNE THROMBOCYTOPENIA BY ADMINISTERING (R)-2-[3-[4-AMINO-3-(2-FLUORO-4-PHENOXY-PHENYL)PYRAZOLO[3,4-D]PYRIMIDIN-1-YL]PIPERIDINE-1-CARBONYL]-4-METHYL-4-[4-(OXETAN-3-YL)PIPERAZIN-1-YL]PENT-2-ENENITRILE

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Patent No.
US None
App. No.
17/069,218
Abstract

Methods for treating immune thrombocytopenia comprising administering at least one compound chosen from (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile (PRN1008) and pharmaceutically acceptable salts thereof are disclosed. Pharmaceutical compositions comprising at least one compound chosen from PRN1008 and pharmaceutically acceptable salts thereof are also disclosed.

Claims (50)

1 . A method for treating immune thrombocytopenia (ITP) in a human patient in need thereof comprising administering to the human patient a therapeutically effective amount of at least one compound chosen from (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile (PRN1008) and pharmaceutically acceptable salts thereof once a day or twice a day for a treatment period, wherein the human patient has at least one characteristic chosen from:

an average plasma count of from 3,000/μL to 33,000/μL for a most recent platelet count prior to the start of the treatment period, a second most recent platelet count prior to the start of the treatment period, and a platelet count on the first day of the treatment period;

a history of taking at least 4 prior ITP therapies prior to the start of the treatment period; and

splenectomy prior to the start of the treatment period.

2 - 6 . (canceled)

7 . A method for achieving a platelet count of at least 50,000/μL in a human patient with immune thrombocytopenia (ITP) comprising administering to the human patient a therapeutically effective amount of at least one compound chosen from (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile (PRN1008) and pharmaceutically acceptable salts thereof once a day or twice a day for a treatment period, wherein the human patient has at least one characteristic chosen from:

an average plasma count of from 3,000/μL to 33,000/μL for a most recent platelet count prior to the start of the treatment period, a second most recent platelet count prior to the start of the treatment period, and a platelet count on the first day of the treatment period;

a history of taking at least 4 prior ITP therapies prior to the start of the treatment period; and

splenectomy prior to the start of the treatment period.

8 . The method according to claim 7 , wherein achieving a platelet count comprises achieving at least 2 platelet counts of at least 50,000/μL.

9 . The method according to claim 7 , wherein achieving a platelet count comprises achieving at least 2 consecutive platelet counts of at least 50,000/μL.

10 . The method according to claim 7 , wherein achieving a platelet count comprises achieving a platelet count of at least 50,000/μL in at least 50% of platelet counts measured during the treatment period.

11 . (canceled)

12 . (canceled)

13 . A method for achieving at least one platelet count of at least 50,000/μL and increasing at least one platelet count by at least 20,000/μL in a human patient with immune thrombocytopenia (ITP) comprising administering to the human patient a therapeutically effective amount of at least one compound chosen from (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile (PRN1008) and pharmaceutically acceptable salts thereof once a day or twice a day for a treatment period, wherein the human patient has at least one characteristic chosen from:

an average plasma count of from 3,000/μL to 33,000/μL for a most recent platelet count prior to the start of the treatment period, a second most recent platelet count prior to the start of the treatment period, and a platelet count on the first day of the treatment period;

a history of taking at least 4 prior ITP therapies prior to the start of the treatment period; and

splenectomy prior to the start of the treatment period.

14 - 24 . (canceled)

25 . The method according to claim 1 , wherein the human patient has an average plasma count of from 3,000/μL to 33,000/μL for the most recent platelet count prior to the start of the treatment period, the second most recent platelet count prior to the start of the treatment period, and the platelet count on the first day of the treatment period.

26 . The method according to claim 1 , wherein the human patient has an average plasma count of from 3,000/μL to 15,000/μL for the most recent platelet count prior to the start of the treatment period, the second most recent platelet count prior to the start of the treatment period, and the platelet count on the first day of the treatment period.

27 . The method according to claim 1 , wherein the human patient has a history of taking at least 4 prior ITP therapies prior to the start of the treatment period.

28 . The method according to claim 1 , wherein the human patient had a splenectomy prior to the start of the treatment period.

29 . (canceled)

30 . (canceled)

31 . The method according to claim 1 , wherein the human patient has chronic ITP.

32 . The method according to claim 1 , wherein the human patient has relapsing ITP.

33 . The method according to claim 1 , wherein the human patient has refractory ITP.

34 . (canceled)

35 . The method according to claim 1 , wherein the human patient has a platelet count of less than 30,000/μL prior to the start of the treatment period.

36 . (canceled)

37 . The method according to claim 1 , wherein the human patient has 2 platelet counts of less than 30,000/μL prior to the start of the treatment period, wherein the 2 counts are measured no sooner than 7 days apart in the 15 days prior to the start of the treatment period.

38 . (canceled)

39 . (canceled)

40 . The method according to claim 1 , wherein the treatment period is at least 28 days.

41 . (canceled)

42 . (canceled)

43 . The method according to claim 1 , comprising administering to the human patient 400 mg of at least one compound chosen from PRN1008 and pharmaceutically acceptable salts thereof once a day or twice a day.

44 . The method according to claim 1 , comprising administering to the human patient 400 mg of at least one compound chosen from PRN1008 and pharmaceutically acceptable salts thereof once a day.

45 . The method according to claim 1 , comprising administering to the human patient 400 mg of at least one compound chosen from PRN1008 and pharmaceutically acceptable salts thereof twice a day.

46 . The method according to claim 1 , comprising administering to the human patient 300 mg of at least one compound chosen from PRN1008 and pharmaceutically acceptable salts thereof twice a day.

47 . The method according to claim 1 , wherein the at least one compound is administered as a monotherapy.

48 . The method according to claim 1 , wherein the at least one compound is administered in combination with at least one concomitant ITP therapy.

49 . The method according to claim 48 , wherein the at least one concomitant ITP therapy is chosen from corticosteroids and thrombopoietin receptor agonists.

50 . The method according to claim 48 , wherein the at least one concomitant ITP therapy is chosen from corticosteroids, eltrombopag, and romiplostim.

51 . The method according to claim 1 , wherein the at least one compound comprises at least one compound chosen from the (E) isomer of (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile and pharmaceutically acceptable salts thereof.

52 . The method according to claim 1 , wherein the at least one compound comprises at least one compound chosen from the (Z) isomer of (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile and pharmaceutically acceptable salts thereof.

53 . The method according to claim 1 , wherein the at least one compound comprises a mixture of (E) and (Z) isomers of (R)-2-[3-[4-amino-3-(2-fluoro-4-phenoxy-phenyl)pyrazolo[3,4-d]pyrimidin-1-yl]piperidine-1-carbonyl]-4-methyl-4-[4-(oxetan-3-yl)piperazin-1-yl]pent-2-enenitrile or a pharmaceutically acceptable salt of the foregoing.

54 . The method according to claim 1 , wherein the at least one compound is orally administered to the human patient.

55 - 58 . (canceled)

Assignments (2)
ASSIGNEE CHANGE OF ADDRESS Recorded Sep 16, 2025
From: PRINCIPIA BIOPHARMA INC.
To: PRINCIPIA BIOPHARMA INC.
Reel/Frame 072881/0270 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 9, 2021
From: GOURLAY, STEVEN; NEALE, ANN; NUNN, PHILIP; LANGRISH, CLAIRE; BANDMAN, OLGA; THOMAS, DOLCA
To: PRINCIPIA BIOPHARMA INC.
Reel/Frame 056802/0501 →