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This perspective article explores AZP2006 (INN: Ezeprogind), a novel small molecule targeting the Progranulin (PGRN) and Prosaposin (PSAP) axis to enhance lysosomal health in PSP treatment. AZP2006 stabilizes the PGRN\u2010PSAP complex, improving lysosomal function and reducing tau pathology. Preclinical studies in tauopathy models demonstrated AZP2006\u2032s ability to decrease tau hyperphosphorylation, enhance neuronal survival, mitigate neuroinflammation and promote synaptogenesis. Clinical trials have shown AZP2006 to be well\u2010tolerated in healthy volunteers and PSP patients. A Phase 2a study met its primary endpoints, as it provided valuable safety data and even encouraged further investigation of its efficacy in a larger clinical study. An upcoming Phase 2b\/3 trial aims to assess long\u2010term safety and efficacy in a larger PSP cohort. AZP2006\u2032s mechanism of action strongly suggests potential applications in other tauopathies, including Alzheimer's and Parkinson's diseases. By addressing lysosomal dysfunction and tau pathology, AZP2006 represents a promising disease\u2010modifying approach for PSP and other neurodegenerative disorders.<\/jats:p>","DOI":"10.1002\/cmdc.202400891","type":"journal-article","created":{"date-parts":[[2025,3,28]],"date-time":"2025-03-28T01:50:02Z","timestamp":1743126602000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["AZP2006 (Ezeprogind<sup>\u00ae<\/sup>): a Promising New Drug Candidate in the Battle Against Neurodegenerative Diseases"],"prefix":"10.1002","volume":"20","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0184-1990","authenticated-orcid":false,"given":"Philippe","family":"Verwaerde","sequence":"first","affiliation":[{"name":"Alzprotect SAS  85\u2005C rue Nelson Mandela 59120 Loos France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-9905-5607","authenticated-orcid":false,"given":"Olivier","family":"Defert","sequence":"additional","affiliation":[{"name":"Alzprotect SAS  85\u2005C rue Nelson Mandela 59120 Loos France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,3,27]]},"reference":[{"key":"e_1_2_8_1_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-023-11791-2"},{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(17)30157-6"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1002\/9781444341256.ch15"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1098\/rstb.2022.0387"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2021.105360"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.3389\/fnmol.2020.586731"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pgen.1001026"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00401-018-1861-8"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.tips.2021.11.015"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00401-017-1668-z"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1097\/NEN.0000000000000158"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/jnc.14110"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-021-94708-1"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.tins.2014.04.003"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.201502029"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.3233\/JAD-220883"},{"key":"e_1_2_8_17_1","unstructured":"J\u2212C. 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