Patent SG-11201808455V-A (SG)
Title: Formulations comprising recombinant acid alpha-glucosidase
Inventor: CHAR HING
IPC: C12N9/26
Filed: 2017-03-30
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization MD 1101111 0 11101 010 11111 010 1111 1 0 111 010 11111 100 1101 100 110 111110 ill OEN International Bureau ... ..... ..Yjd . n. ..1: .....1 (10) International Publication Number (43) International Publication Date WO 2017/173060 Al 5 October 2017 (05.10.2017) IN I PO I P CT (51) International Patent Classification: (72) Inventors: CHAR, Hing; 1 Cedar Brook Drive, Cranbury, A61K 38/47 (2006.01) A61P 3/00 (2006.01) New Jersey 08512 (US). TESLER, Sergey; 1 Cedar Brook Cl 2N 9/26 (2006.01) Drive, Cranbury, New Jersey 08512 (US). SUNDER- LAND, Wendy; 1 Cedar Brook Drive, Cranbury, New Jer- (21) International Application Number: PCT/US2017/024982 sey 08512 (US). DILONE, Enrique; 1 Cedar Brook Drive, Cranbury, New Jersey 08512 (US). (22) International Filing Date: (74) Agent: ALEGRIA, Rory P.; Servilla Whitney LLC, 33 30 March 2017 (30.03.2017) Wood Avenue South, Suite 830, Iselin, New Jersey 08830 (25) Filing Language: English (US). (26) Publication Language: English (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (30) Priority Data: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, 62/315,436 30 March 2016 (30.03.2016) US BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, 62/457,588 10 February 2017 (10.02.2017) US DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, 15/473,999 30 March 2017 (30.03.2017) US HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, (71) Applicant: AMICUS THERAPEUTICS, INC. [US/US]; KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, 1 Cedar Brook Drive, Cranbury, New Jersey 08512 (US). MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. = = = [Continued on next page] = = (54) Title: FORMULATIONS COMPRISING RECOMBINANT ACID ALPHA-GLUCOSIDASE Embodiment 1 (57) : Provided are pharmaceutical formulations comprising a re- combinant acid a-glucosidase, wherein the recombinant acid a-glucosidase is = 5,000 = 4,000 30% = — u. - - 3,000 — ( 1 ;3 2000 = a o _ co 2 TO% + 6 expressed in Chinese hamster ovary (CHO) cells and comprises an increased 5 content of N-glycan units bearing one or two mannose-6-phosphate residues 4 when compared to a content of N-glycan units bearing one or two mannose- ' E 6-phosphate residues of alglucosidase alfa; at least one buffer selected from 3 a ' the group consisting of a citrate, a phosphate and combinations thereof; and 2 at least one excipient selected from the group consisting of mannitol, polyso (.D 1 ,000 vt- __. 1 rbate 80, and combinations thereof, wherein the formulation has a pH of from = 0 about 5.0 to about 7.0. Also provided are methods of treating Pompe disease = rhGAA lacks M6P; rhGAA contains M6P using these pharmaceutical formulations. = cannot be targeted targeted to lysosomes = to lysosomes Embodiment 2 = 5,000 S' 4 4,000 = L., 3,000 — . (1 2,000 1 1-1 c000 ' .4 0 m co 8% ----- -' 92% -* E !, .23 Cis . 1 0 rhGAA tacks M6P; rhGAA contains M6P cannot be targeted targeted to lysosomes 0 en to lysosomes IN 11 FIG. 6 IN 11 © ei O WO 2017/173060 Al MIDEDIMOMOIDEIRMEMOMMEHIMINIMEHOIMI (84) Designated States (unless otherwise indicated, for every Published: kind of regional protection available): ARIPO (BW, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, — GH, SZ, with international search report (Art. 21(3)) before the expiration of the time limit for amending the TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, DE, LT, SE, GA, claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a)) GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG).