SG11201805591QA - Tacrolimus for treating tdp-43 proteinopathy - Google Patents

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization International Bureau .. .... ..Yejd (43) International Publication Date ..... .....1 13 July 2017 (13.07.2017) WI P0 I P CT IIIM141101110101011111 HO 11111011130111111111111110011111111101111 III N (10) International Publication Number WO 2017/118857 Al (51) (21) (22) (25) (26) (30) (71) (72) (74) International Patent Classification: A61K 31/436 (2006.01) A61P 25/28 (2006.01) International Application Number: International Filing Date: Filing Language: Publication Language: Priority Data: 1600376.6 8 January 2016 (81) Designated States (unless otherwise indicated, for every A61P 25/16 (2006.01) kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, PCT/GB2017/050021 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, 6 January 2017 (06.01.2017) MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, English RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, English TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (08.01.2016) GB (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, LIMITED GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 41 Cornmarket Street, Oxford TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, Pete; 40 Castle Road, Woot- LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, JONES, Huw; The Old SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, Morden, Royston Hert- GW, KM, ML, MR, NE, SN, TD, TG). Helen; 14 Somer- (GB). SHERRIFF, Julia; 5 Published: 8FF (GB). — with international search report (Art. 21(3)) Potter Clarkson LLP, Street, Nottingham NG1 5GG Applicant: CHRONOS THERAPEUTICS [GB/GB]; c/o Mercer Lewin, OX1 3HA (GB). Inventors: APPLEFORD, ton, Woodstock OX20 lEG (GB). School, 45 Church Street, Guilden fordshire SG8 OJD (GB). KUHLMAN, dale View, Bath BA2 2QQ Terrett Avenue, Oxford OX3 Agent: CARLING, David Andrew; The Belgrave Centre, Talbot (GB). Title: TACROLIMUS FOR TREATING TDP-43 PROTEINOPATHY 300 250 W — 200, >+ cu ... as 150 —I 100 50 : The present invention as amyotrophic lateral sclerosis (ALS) and frontotemporal dementias (FTDs), wherein the tacrolimus is for administration at a -effective dose which (54) \" therapeutically Rotarod Performance over 60 weeks V 4 f 4—. - — RDC5 0 25mg/kg — RDC5 1 25mg/kg R . ' T T1 — RDC5 2 5mg/kg I — Vehicle 1 — Non Tg TT T TT f i. l' k'`w r . Il .4 Ir ... ' 11 in GC Il Il IN 1-1 (57) 0 such 0 10 20 30 40 50 60 70 Week FIGURE 3 provides tacrolimus for use in the treatment in a human subject of a TDP-43 proteinopathy, does not cause immunosuppression in the subject.