Claims for Patent: 12,178,818

Summary for Patent: 12,178,818

Title:	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
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.
Inventor(s):	Steven GOURLAY, Ann Neale, Philip NUNN, Claire LANGRISH, Olga Bandman, Dolca Thomas
Assignee:	Principia Biopharma Inc
Application Number:	US17/946,585
Patent Claims:	1. A method for achieving a platelet count of at least 50,000/μL in a human patient with immune thrombocytopenia (ITP) 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 and pharmaceutically acceptable salts thereof, once a day or twice a day for a treatment period, wherein the human patient has been identified as having a platelet count of less than 30,000/μL for two or more consecutive platelet counts prior to the start of the treatment period, wherein two of the two or more platelet counts were measured no sooner than 7 days apart in the 15 days prior to the start of the treatment period, and wherein the human patient has been identified as having at least one characteristic chosen from: a history of taking at least one prior ITP therapy prior to the start of the treatment period; and splenectomy prior to the start of the treatment period. 2. The method of claim 1, wherein achieving a platelet count comprises achieving at least two platelet counts of at least 50,000/μL. 3. The method of claim 1, wherein achieving a platelet count comprises achieving at least two consecutive platelet counts of at least 50,000/μL. 4. The method of claim 1, 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. 5. The method of claim 1, wherein the human patient has been identified as having a history of taking at least two prior ITP therapies prior to the start of the treatment period. 6. The method of claim 1, wherein the human patient had a splenectomy prior to the start of the treatment period. 7. The method of claim 1, wherein the human patient has chronic ITP. 8. The method of claim 1, wherein the human patient has relapsing ITP. 9. The method of claim 1, wherein the human patient has refractory ITP. 10. The method of claim 1, wherein the treatment period is at least 28 days. 11. The method of claim 1, comprising administering to the human patient 400 mg 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 and pharmaceutically acceptable salts thereof, once a day or twice a day. 12. The method of claim 1, comprising administering to the human patient 400 mg 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 and pharmaceutically acceptable salts thereof, once a day. 13. The method of claim 1, comprising administering to the human patient 400 mg 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 and pharmaceutically acceptable salts thereof, twice a day. 14. The method of claim 1, comprising administering to the human patient 300 mg 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 and pharmaceutically acceptable salts thereof, twice a day. 15. The method of claim 1, wherein the at least one compound is administered as a monotherapy. 16. The method of claim 1, wherein the at least one compound is further administered in combination with at least one concomitant ITP therapy. 17. The method of claim 16, wherein the at least one concomitant ITP therapy is chosen from corticosteroids and thrombopoietin receptor agonists. 18. The method of claim 16, wherein the at least one concomitant ITP therapy is chosen from corticosteroids, eltrombopag, and romiplostim. 19. The method of 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. 20. The method of 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. 21. The method of 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. 22. The method of claim 1, wherein the at least one compound is orally administered to the human patient.