Patent CA-3081946-A1 (CA)
Inventor: PENEBRE ELAYNE
IPC: A61K31/501
Filed: 2018-10-18
Abstract: (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization ION 1 11111111 111111 1111 11111 1111 11111 1 11 11 1111111 1111 1 111 11 111111 11111 1 1111 1 111111 International Bureau (10) International Publication Number (43) International Publication Date WO 2019/079607 Al 25 April 2019 (25.04.2019) VVIPO I PCT (51) International Patent Classification: (74) Agent: ERLACHER, Heidi et al.; Cooley LLP, 1299 A61K 31/137 (2006.01) C07C 211/00 (2006.01) Pennsylvania Avenue, Suite 700, Washington, District of A61P 7/00 (2006.01) C07C 211/04 (2006.01) Columbia 20004 (US). (21) International Application Number: (81) Designated States (unless otherwise indicated, for every PCT/U52018/056530 kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (22) International Filing Date: CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 18 October 2018 (18.10.2018) DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, (25) Filing Language: English HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, (26) Publication Language: English MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, (30) Priority Data: OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, 62/573,876 18 October 2017 (18.10.2017) US SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, 62/574,128 18 October 2017 (18.10.2017) US TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (71) Applicant: EPIZYME, INC. [US/US]; 400 Technology (84) Designated States (unless otherwise indicated, for every Square, 4th Floor, Cambridge, Massachusetts 02 139 (US). kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, (72) Inventors: PENEBRE, Elayne; 26 Evergreen Avenue, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, Auburndale, Massachusetts 02466 (US). GIBAJA, Veron- TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, ica; 86 Walnut Street, Apt. 3, Dorchester, Massachusetts EE, ES, FI, FR, GB, GR, HR, HU, 1E, IS, IT, LT, LU, LV, 02122 (US). RAIMONDI, Maria Alejandra; 22 McBride MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, Street, Jamaica Plain, Massachusetts 02130 (US). TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). __ (54) Title: METHODS OF USING EHMT2 INHIBITORS IN TREATING OR PREVENTING BLOOD DISORDERS Figure 6 i) DMSO 200 %HbF" A %HbF ceus cells 423% 150 0 -103 0 103 104 105 Comp-PE-A: HbF 1 pM CPD 25R 111) 0.3 M CPU D5R 0.4 CPD D5R v) 0.1 oM CPD 35R y) 0.012 pM CMP D5R 2ou %HbF-6t4 %HhF __ 20 11.1.: ibF 4 ceIs 220 = %Hbri %HbF+cells 20 %HbF"I.' %HbF' cells 2 %HbF- %HbF cells cells 90.9% cells 100% ' cells t 1DO% oelis 08.e% cells 98.1% 150 150 150-, 150 150 . = r =E. ,00 100 In: 100 100 50 , = 1. A 50 50-50 50 r, 0 0 __________ j 0: , 4, 0 -103 0 100 10-'' 105 -10 0 10 164 -105 -1'0i 0 103 10' 105 -10 0 10 '10' 10 -103 0 10 '10` 105 =C; Corop-PE-A ':HbF Comp-PE-A:: HbF Comp-PE-A:: HbF Comp-PE-A =:HbF Comp-PE-A :: HbF 01 (57) Abstract: The present disclosure relates to a method of preventing or treating a blood disorder (e.g., sickle-cell disease) via 01 1-1 administering an EHMT2 inhibitor compound disclosed herein or a pharmaceutical composition thereof to subjects in need thereof. G0 The present disclosure also relates to the use of such compounds for research or other non-therapeutic purnoses. [Continued on next page] Date Reçue/Date Received 2020-04-16 WO 2019/079607 Al I 11 111 IIIIIIII II 11111111111 11111111111111111111111111111111111111111111111111111111 111101111 11 Declarations under Rule 4.17: ¨ as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii)) Published: ¨ with international search report (Art. 21(3)) ¨ before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) Date Recue/Date Received 2020-04-16