{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T16:23:08Z","timestamp":1787502188819,"version":"build-2736575974"},"reference-count":13,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T00:00:00Z","timestamp":1777507200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T00:00:00Z","timestamp":1777507200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["accp1.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Clinical Pharm in Drug Dev"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Votoplam is a novel, orally bioavailable, small molecule HTT gene splicing modifier that is being developed for the treatment of Huntington's disease. A single\u2010dose, open\u2010label, three\u2010dose level (5, 10, 20\u00a0mg), parallel group ethnicity study was conducted to evaluate the pharmacokinetics and safety of votoplam in healthy Caucasian versus Japanese participants. Six participants per ethnic group were enrolled at each dose (12 participants per dose level overall). Thirty\u2010five participants (N = 18 Japanese; N = 17 Caucasian) completed the study. Study results showed slightly higher exposure for Japanese participants at all dose levels. AUC\n                    <jats:sub>0\u2013last<\/jats:sub>\n                    increased by 1.27\u2010, 1.17\u2010, and 1.29\u2010fold, and C\n                    <jats:sub>max<\/jats:sub>\n                    increased by 1.58\u2010, 1.19\u2010, and 1.61\u2010fold for 5, 10, and 20\u00a0mg, respectively, for Japanese compared with Caucasian participants. Doses proportionality was observed for both ethnicities. Few mild and moderate TEAEs were reported and were comparable between Japanese and Caucasian participants. In summary, votoplam pharmacokinetics and safety profiles are similar between healthy Japanese and Caucasian participants when administered in the single dose range of 5, 10, and 20\u00a0mg. Therefore, it is expected that dose adjustments should not be necessary in future clinical studies enrolling Japanese participants.\n                  <\/jats:p>","DOI":"10.1002\/cpdd.70062","type":"journal-article","created":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T07:59:35Z","timestamp":1777535975000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Ethnic Bridging of Votoplam, a HTT Gene Splicing Modifier, Using Pharmacokinetic Comparison Between Caucasian and Japanese Subjects"],"prefix":"10.1002","volume":"15","author":[{"given":"Lucy","family":"Lee","sequence":"first","affiliation":[{"name":"PTC Therapeutics, Inc.  Warren NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy\u2010Lee","family":"Richards","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc.  Warren NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Prachi","family":"Anand","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc.  Warren NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Beers","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc.  Warren NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lee","family":"Golden","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc.  Warren NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ronald","family":"Kong","sequence":"additional","affiliation":[{"name":"PTC Therapeutics, Inc.  Warren NJ USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,4,30]]},"reference":[{"key":"e_1_2_8_2_1","first-page":"1993","volume-title":"GeneReviews [internet]","author":"Caron NS","year":"1998"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrneurol.2014.24"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.prdoa.2020.100056"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1101\/cshperspect.a024240"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1326-1-19"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41582-020-0389-4"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.0299"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-021-27157-z"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1021\/acs.jmedchem.8b00741"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrn1806"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.0299"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.16202"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/cpdd.1626"}],"container-title":["Clinical Pharmacology in Drug Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/accp1.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/cpdd.70062","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/accp1.onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/cpdd.70062","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/accp1.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/cpdd.70062","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T15:27:24Z","timestamp":1787498844000},"score":1,"resource":{"primary":{"URL":"https:\/\/accp1.onlinelibrary.wiley.com\/doi\/10.1002\/cpdd.70062"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,4,30]]},"references-count":13,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2026,5]]}},"alternative-id":["10.1002\/cpdd.70062"],"URL":"https:\/\/doi.org\/10.1002\/cpdd.70062","archive":["Portico"],"relation":{},"ISSN":["2160-763X","2160-7648"],"issn-type":[{"value":"2160-763X","type":"print"},{"value":"2160-7648","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,4,30]]},"assertion":[{"value":"2026-01-29","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-04-10","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-04-30","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70062"}}